PALM BEACH, Fla., Dec. 21, 2021 /PRNewswire/ -- FinancialNewsMedia.com News Commentary - Systemic mastocytosis is rare disease which affects very few people and it causes due to C-kit mutation which leads to higher number of mast cell production in the body resulting in accumulation of excessive mast cells in the internal body organs such as spleen, liver, bone marrow and small intestine etc. Recently, the World Health Organization (WHO) updated the prognosis, diagnosis and systemic mastocytosis treatment guidelines for the disease which in turn helped to bring uniformity in the approach by healthcare professionals. The manufacturers in the systemic mastocytosis treatment market are focusing on evaluating possible treatment options to cure the disease by investing heavily in the research & development. Various leading manufacturers are focusing on gaining FDA approval to respective drugs for the systemic mastocytosis treatment to enhance their revenue generation. A reportfrom Future Market Insights said:"Increasing awareness about the systemic mastocytosis treatment as well as symptoms of the disease due to extended effort by non-profit organizations, governmental associations and through other platforms expected to drive the growth of the systemic mastocytosis treatment market Increasing approvals and launches of drugs for the systemic mastocytosis treatment expected to drive the growth of the market Increasing spending on research & development by various pharmaceutical companies to develop novel systemic mastocytosis treatment expected to further fuel the growth of market. Increasing early diagnosis rate subsequently followed by increasing treatment seeking rate further expected to drive the growth of the systemic mastocytosis treatment market." Active companies in the markets today include: Hoth Therapeutics, Inc. (NASDAQ:HOTH), Longeveron Inc. (NASDAQ: LGVN), Bristol Myers Squibb (NYSE: BMY), Takeda Pharmaceutical Company Limited (NYSE: TAK), Amgen (NASDAQ: AMGN).

Future Market Insights continued:"The global systemic mastocytosis treatment market is expected to experience significant growth due to increasing novel treatment options. By drug class, systemic mastocytosis treatment market is expected to be dominated by the mast cell stabilizers due to superior efficacy. By indication, systemic mastocytosis treatment market is expected to be dominated by indolent systemic mastocytosis (ISM) due to higher prevalence. By route o administration, systemic mastocytosis treatment market is expected to be dominated by injectables. By distribution channel, systemic mastocytosis treatment market is expected to be dominated by the retail pharmacies due to higher patient footfall. The global systemic mastocytosis treatment market is expected to be dominated by the North America due to comparatively higher prevalence of the disease. Europe systemic mastocytosis treatment market is expected to be second most lucrative market due to higher treatment seeking rate. Latin America expected to show gradual growth in the systemic mastocytosis treatment market due to steadily increasing diagnosis. Asia-Pacific is emerging systemic mastocytosis treatment market due to increasing diagnosis subsequently followed by treatment. Middle East & Africa is the least lucrative systemic mastocytosis treatment market due to least diagnostic rate and lower awareness about the symptoms."

Hoth Therapeutics, Inc. (NASDAQ:HOTH) BREAKING NEWS: Hoth Therapeutics Announces Submission of Orphan Designation Application for HT-KIT to Treat Mastocytosis Hoth Therapeutics, Inc., a patient-focusedclinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Application to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocyctosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting, resulting in apoptosis of neoplastic mast cells. The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN)), acute myeloid leukemia, gastrointestinal stromal tumors, and anaphylaxis.

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US)are eligible to apply for Orphan Drug Designation (ODD), which provides multiple benefits to the sponsor during development and after approval. Hoth intends to pursue these benefits as part of the drug development for HT-KIT for treatment of mastocytosis, pending designation of the ODD application.

Benefits of Orphan Drug Designation - Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug. A tax credit of 50% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and provide special assistance to companies that develop drugs for rare patient populations. In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.

Key benefits of ODD:

Hoth recently announcedthat its novelanti-cancer therapeuticexhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma. CONTINUED Read the Hoth Therapeutics full press release by going to: https://ir.hoththerapeutics.com/news-releases

In other news and developments of note in the markets this week:

Amgen (NASDAQ: AMGN) recently announced that the U.S. Food and Drug Administration (FDA) has approved Amgen and AstraZeneca'sTezspire (tezepelumab-ekko) for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.

Tezspirewas approved following a Priority Review by the FDA and based on results from the PATHFINDER clinical trial program. The application included results from the pivotal NAVIGATOR Phase 3 trial in whichTezspiredemonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

Bristol Myers Squibb (NYSE: BMY) recently announced thatOrencia(abatacept) was approved by the U.S. Food and Drug Administration (FDA) for the prophylaxis, or prevention, of acute graft versus host disease (aGvHD), in combination with a calcineurin inhibitor (CNI) and methotrexate (MTX), in adults and pediatric patients 2 years of age and older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated donor (URD).

"Orenciais the first FDA-approved therapy to prevent acute graft versus host disease following hematopoietic stem cell transplant, a potentially life-threatening complication that can pose a comparatively higher risk to racial and ethnic minority populations in the U.S. due to difficulty finding appropriately matched donors," said Tina Deignan, senior vice president, U.S. Immunology, Bristol Myers Squibb. "With this fourth indication forOrencia,Bristol Myers Squibb draws on its legacy and expertise in both immunology and hematology to deliver an important treatment option for patients in a disease with high unmet need.

Takeda Pharmaceutical Company Limited (NYSE: TAK) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of intravenous (IV) vedolizumab for the treatment of adult patients with moderately to severely active chronic pouchitis, who have undergone proctocolectomy and ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), and have had an inadequate response with or lost response to antibiotic therapy. The CHMP opinion will now be reviewed by the European Commission. If approved, vedolizumab will become the first treatment indicated for active chronic pouchitis across the European Union.

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates Financialnewsmedia.com and MarketNewsUpdates.com, is a third- party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM was compensated twenty five hundred dollars for news coverage of current press release issued by: Hoth Therapeutics, Inc. by a non-affiliated third party.

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Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options - PRNewswire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Imago BioSciences, Inc., (Imago) a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced the expansion of its global Phase 2b clinical study evaluating bomedemstat (IMG-7289) for the treatment of advanced myelofibrosis (MF) into Hong Kong, where the first patient has now been enrolled and dosed at the Department of Medicine, Queen Mary Hospital and the University of Hong Kong. Myelofibrosis is a rare bone marrow cancer that interferes with the production of blood cells.

In addition to Hong Kong, the Phase 2b study continues to actively enroll patients in the U.S., U.K., and E.U. The study is in the final stages of completing enrollment and continues to dose patients to evaluate safety, tolerability and efficacy.

Patients with myelofibrosis around the world are still in need of new treatment options, said Hugh Young Rienhoff, Jr. M.D., Chief Executive Officer, Imago BioSciences. We are progressing well with enrollment and are pleased to continue expanding our global Phase 2 study into new geographies like Hong Kong. We are encouraged by the signs of clinical activity and safety of bomedemstat as a treatment alternative for patients who do not benefit from the current standards of care.

Bomedemstat is an inhibitor of lysine-specific demethylase 1 (LSD1), an epigenetic regulator critical for self-renewal of malignant myeloid cells and the differentiation of myeloid progenitors. Data presented at the 25th European Hematology Association (EHA) Annual Congress in June demonstrated that the first-in-class LSD1 inhibitor was well tolerated with no dose-limiting toxicities or safety signals. Furthermore, recent data demonstrates the potential of bomedemstat as a monotherapy in intermediate-2 and high-risk patients with myelofibrosis who have become intolerant of, resistant to or are ineligible for a Janus Kinase (JAK) inhibitor.

Bomedemstat was recently granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA) for the treatment of MF. The EMA reviewed bomedemstat non-clinical and clinical data from the ongoing Phase 2 study. The PRIME initiative was launched by the EMA in 2016 to provide proactive and enhanced support to the developers of promising medicines with the view of accelerating their evaluation to reach patients faster.

About Bomedemstat (IMG-7289)

Bomedemstat is an orally available small molecule discovered and developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, bomedemstat demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other therapeutic agents. Bomedemstat is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185, NCT04262141, NCT04254978 and NCT04081220).

Bomedemstat has U.S. FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, Orphan Drug Designation for treatment of acute myeloid leukemia and PRIME designation by the European Medicines Agency for the treatment of MF.

Bomedemstat is being evaluated in two open-label Phase 2 clinical trials for the treatment of advanced myelofibrosis (MF) and essential thrombocythemia (ET), bone marrow cancers that interfere with the production of blood cells. MF patients who are resistant to a Janus Kinase (JAK) inhibitor are eligible for the study of bomedemstat. ET patients who have failed one standard of care treatment are eligible for the bomedemstat ET study.

About Imago BioSciences

Imago BioSciences is a clinical-stage biopharmaceutical company focused on discovering and developing novel therapeutics for the treatment of hematologic disorders targeting epigenetic enzymes. Imago has developed a series of compounds that inhibit LSD1, an epigenetic enzyme critical for cancer stem cell function and blood cell differentiation. Imago is advancing the clinical development of its first LSD1 inhibitor, bomedemstat, for the treatment of myeloid neoplasms. Imago BioSciences is backed by leading private, corporate, and public investors including Farallon Capital Management, LLC., funds and accounts advised by T. Rowe Price Associates, Inc., funds and accounts managed by Blackrock Advisors, LLC., Surveyor Capital (a Citadel company), Irving Investors, Kingdon Capital Management, a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital. The company is based in South San Francisco, California. To learn more, visit http://www.imagobio.com, http://www.myelofibrosisclinicalstudy.com, http://www.etclinicalstudy.com and follow us on Twitter @ImagoBioRx, Facebook and LinkedIn.

Originally posted here:
Imago BioSciences Expands Phase 2 Clinical Trial of Bomedemstat (IMG-7289) for the Treatment of Myelofibrosis into Hong Kong - Business Wire

Detroit Lions senior VP of business development Kelly Kozole works with her daughter, Morgan, who has a rare neurological disorder called beta-propeller protein-associated neurodegeneration, or BPAN.Michael Rothstein

TROY, Mich. -- Wearing a white T-shirt with a massive star in sparkling shades of pink, yellow and seafoam green on the front, Morgan Kozole sits in front of a fold-up chalkboard in the living room of her family's Detroit-area home and starts to draw.

Using pink and yellow chalk, she sketches Mickey and Minnie Mouse. The Disney characters are dominant fixtures in the 5-year-old's life and therefore become a soundtrack for the Kozole family: Morgan constantly saying "Mickey," with her long, blond ponytail bouncing to whatever song happens to be playing on the Mickey Mouse Club.

"These are the two Mickeys," Morgan says, pointing to the chalkboard. Her mother, Detroit Lions senior vice president of business development Kelly Kozole, explains that this is her way of communicating that she would like a visitor to draw Mickey too. If it's close, Morgan accepts it. Another Mickey to fawn over.

For Morgan's birthday earlier this year, the family went to Disney World. On this trip, the Kozoles saw what they had longed for: the potential of progress.

"She knew where we were. She knew Mickey Mouse," Kelly said. "Before, she wouldn't go to the characters, and now she's jumping up and down, hugging. She really, along those lines, is also really into birthdays.

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"The 'Happy Birthday' song. Before that, she was just kind of looking. Sometimes it was too much for her with everyone singing -- sometimes loud noises are too much. This year, we had to sing 'Happy Birthday' to her three times."

Birthdays, for children, are happy occasions -- reasons for grand celebrations of progress toward adulthood. For the rest of Morgan's family it is more complicated.

Morgan has a rare neurological disease called beta-propeller protein-associated neurodegeneration, known as BPAN. It's a disorder, more prevalent in girls than boys, that causes delayed development and seizures, communication issues and, sometimes, motor dysfunction. It's unclear exactly how many people are living with BPAN worldwide due to its rarity, although Dr. Sami Barmada, a scientist at the University of Michigan studying BPAN, estimates roughly 500 to 600 people.

It's rare enough that Dr. Henry Paulson, the director of the Michigan Alzheimer's Disease Center, said there are experts in neurodegeneration who are unfamiliar with BPAN. While Kelly is trying to advocate for her daughter and others with BPAN through fundraising for research, science moves only so fast.

The Kozoles understand that. So birthdays for the family aren't always happy. They are a reminder of what could come.

"That ticking time clock," Kelly said. "Every birthday isn't exciting for me for her. Because it's one year closer to when this bomb is going to go off."

BPAN's rarity makes the reality heartbreakingly simple: There are very few effective treatments, little research and no cure. As Morgan learns how to organize her Peppa Pig characters and learns new words on her iPad -- her future looms.

At some unpredictable point in Morgan's teen and adult years -- the average is around age 25, according to Barmada -- development will just stop. Progress will decline and, in some cases, disappear. Those afflicted with BPAN begin suffering from progressive dystonia parkinsonism -- making it difficult to walk, talk or stand.

"Any day," Kelly said, "it could be like, 'Oh, your daughter's gone.'"

WHEN MORGAN WAS born on Jan. 12, 2015, she was, largely, a healthy baby. She was a little jaundiced but nothing worrisome.

When she would go to the doctor's office for shots, Morgan didn't cry. It was a little abnormal, but when you're a parent of a young child, no crying is viewed as a minor miracle. Kelly and her husband, Kevin, took this as a sign of a tough kid. Nurses even said how great it was.

Looking back, it was a warning sign that something was wrong. BPAN causes a high pain tolerance. Before long, more concerns popped up. Morgan wasn't crawling at nine months, wasn't walking at a year. Expected milestones passed without Morgan reaching them. Kevin and Kelly put her in therapy in late 2016 to work up to these childhood progressive traits and began researching potential causes. They wouldn't find an answer for more than two years.

"She was diagnosed with cerebral palsy at first. One doctor diagnosed her with that, and then another, our neurologist, said she doesn't have that," Kelly said. "Then there was speculation but not a full diagnosis she had autism, so we did all the tests for that.

"So through this kind of journey of trying to find out what was wrong, it was exciting that she didn't have something that you were going to this test for, but you still had so many more questions as you were eliminating all these potential diseases that she could have."

Befuddled, they began genetic testing and in November 2018 received a letter about a mutation on Morgan's WDR45 gene. Kelly Googled it, stumbled upon BPAN and freaked out, calling their neurologist. The neurologist told Kelly not to worry -- BPAN was very rare, and Morgan didn't have it.

Doctors diagnosed her with epilepsy because of seizures. Morgan took Keppra, which helped accelerate her vocabulary to about 50 words, typical for a 1-year-old, when she was 3. Then doctors said no, it wasn't epilepsy either.

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Another meeting with another neurologist led to a different diagnosis. Three days after she and Kevin returned to Michigan from Super Bowl LIII in February 2019, they received a call. Doctors figured out what was wrong.

It was BPAN.

"In my mind, it's worse than cancer," Kelly said. "How is this even possible? That this can even be so painful for kids later on in life. You try so hard to gain all these abilities, and then early adolescence or early adulthood, it's just [gone] one day, and I've seen a lot of these stories.

"There's a BPAN Facebook website, and that's where the doctors sent us. There's no cure. There's no therapy. 'Go to this website.' That's what I was told."

FOR MONTHS KELLY cried, angry and heartbroken. The Kozoles initially told their families and no one else.

In May 2019, Kelly went to her first Neurodegeneration with Brain Iron Accumulation (NBIA) conference. She met other parents, heard their stories and began the new normal.

She used her skills -- organization, fundraising and business -- to brainstorm ways to help. Hardly anyone had researched BPAN. Without it, there would be no chance for a cure -- not in Morgan's lifetime, which could reach her 40s, and not in the lifetime of those who might come after.

She shared what was happening with her boss, Detroit Lions president Rod Wood, and his wife, Susan, using a website link to explain BPAN. Wood knew something was wrong because of texts and emails saying they had to take Morgan to this specialist or that appointment.

"As that was confirmed and became her reality, she is now able to talk about it, in a way," Wood said. "Because she's full bore on trying to help generate awareness and financial resources to find a cure for it.

"She went from the unknown to the very tragic known to, 'OK, what are we going to do about it?'"

Kelly consulted her aunts, both of whom worked in medicine. Linda Narhi worked in biotechnology for Amgen for more than 30 years; Dr. Diane Narhi was the first female chief of staff at Simi Valley (California) Hospital. From talking with another group of fundraising BPAN parents -- BPAN Warriors -- Kelly found a guide.

If her aunts had not been resources, she might have joined BPAN Warriors. But Kelly admittedly needs to be in control, and this was her daughter. She needed to manage this herself. She created a nonprofit called Don't Forget Morgan.

Kelly's aunts provided guidance, and Wood offered contacts he had in the finance industry and Silicon Valley. Wood and Lions general counsel Jay Colvin sit on the board. Other Lions coworkers -- with Wood's blessing -- built the website, designed the logo and created social media plans and the first pitch video for Don't Forget Morgan's rollout in 2020.

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Progress started with a $15,000 grant to help with a mouse model study at Sanford Research in South Dakota, with another, larger, potential grant to come. In recent months, Kelly has focused largely on fundraising, and another parent of a child with BPAN, Christina Mascarenhas Ftikas, has focused on the medical side of the nonprofit.

"This is why I'm here," Kelly said. "I'm supposed to be a vehicle to get all of this awareness and hopefully a cure for BPAN so the child one, two, three, five years from now, there is hope.

"There is no, 'Go to Facebook.' There is something where you can actually give a parent, 'Here's the symptoms to look for.'"

ABOUT AN HOUR away in Ann Arbor, Michigan, Kaci Kegler and her husband, Brian, had been in the same Facebook community. Kelly, new to the group and looking for a nearby connection, wrote Kaci a message.

"Hey, my daughter was just diagnosed, could we connect?"

Kaci understood. She did the same thing, reaching out without success in 2016 after her daughter, Elle, was diagnosed. Kaci wanted to be a resource.

They talked for an hour. There wasn't much Kaci could say to soothe her. Kelly pinged a year later with another message: I'm starting a nonprofit. Kaci offered to help.Despite suffering from BPAN, Morgan is like any other 5-year-old who enjoys playing with her brother, Connor.Michael Rothstein

Days later, on Feb. 28, Kaci and her husband, Brian, an assistant athletic director for development at the University of Michigan, had their yearly fundraiser for BPAN research on Rare Disease Day at Pizza House in Ann Arbor. They met a doctor who had a connection to researchers at Michigan.

"I literally came home and texted [Kelly] and was like, 'Oh my gosh, we may have inroads,'" Kaci said. "We just started texting. I have never met Kelly face-to-face. We still haven't. But we've texted a lot and we've emailed quite a bit.

"It just kind of started."

By summer, they went from nothing to putting pieces in place for a full-fledged research project with a two-year, $140,000 grant for Barmada and Dr. Jason Chua to help start to solve BPAN.

Chua was working on the regulation of autophagy, which is the cleaning out of damaged cells, and studying BPAN became a natural extension of the work he had already been putting in. BPAN alters that in neurons. Barmada said Chua's research provided a "rare win-win situation" to potentially help with BPAN and other diseases too.

"There are a set of questions in BPAN that nobody has the answer to," Barmada said. "And Jason and myself, we just seem to be in the right position, the right place to be able to help out."

The goal is to understand what is happening within BPAN itself and how people end up with it, while also trying to find therapies for existing patients. Within a year, they are hoping to grow stem cells from people with BPAN in their lab, allowing for the creation of their own stem cells missing the WDR45 gene. Then they will try to either replace the gene or "stimulate autophagy through genetic or pharmacologic means," Barmada said. The hope is this can prevent neurodegeneration.

So far, they've hired a research assistant to work with Chua, developed tools to manipulate the gene using the genome-editing tool CRISPR and applied for approval from Michigan and the institutional review board to get skin biopsies to obtain stem cells from BPAN patients.

It's a process, but it's also a start.

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After partnering with Michigan and Sanford, Don't Forget Morgan also began working with Dr. Kathrin Meyer, a researcher at the Center for Gene Therapy at Nationwide Children's Hospital at Ohio State.

"Solving this disease is going to require more than Jason and Sami," Paulson said. "It's going to be a first shot across the bow, but it's going to require more than that. I'll say this, being in the field for a long time. Scientists who are coming up the pike say they want to look at Alzheimer's, want to look at epilepsy. They don't say, 'I want to look at a rare disease.'

"The only way to solve a rare disease is to get someone hooked. Sometimes when you hook a really good one, as I think we have with Jason here, you hook them for life and they make a difference."

MORGAN IS BOUNCING around the Kozoles' suburban Detroit home on this late August day. They just returned from northern Michigan, and having two kids, especially one with special needs, makes tidiness unrealistic.

COVID-19 changed things. Morgan hadn't been to many of her therapies for months. Online school barely kept her attention. There was concern she would have regression in her learning. Instead, her speech advanced by being around Kelly, Kevin and her older brother, Connor, all day. She has sung more songs recently to help increase her vocabulary. Sometimes, she'll listen 20 times in a row.

"Even more than that," Connor said. They aren't sure how much she's truly learning versus memorization. But it is something.Morgan Kozole has inspired her mother, Detroit Lions VP Kelly Kozole, to marshal researchers and other advocates to develop a cure for BPAN, and perhaps help future generations of children who live with the disorder.Michael Rothstein

The family gathers inside Morgan's bedroom -- complete with a special Haven Bed with a zipper to keep her safe from wandering around at night, when she could accidentally turn on the stove and hurt herself or others -- sleep disorders are another BPAN issue. She sits on the floor and starts playing with her small, yellow dollhouse and a fake ice-cream maker. Kelly asks for an ice cream. Morgan makes one for herself instead and pretends to eat it.

Later, outside, Morgan kicks a soccer ball and plays a modified game of catch with a squishy football. Football, no surprise, is big. She says "hike" a lot. "She knows that term," Kevin says, laughing.

In these moments, Morgan seems like any other young child. She attends St. Hugo of the Hills Parish School in Bloomfield Hills, Michigan, but has a one-on-one para nanny to help. She interacts with people, often overly affectionate.

Sitting at the kitchen table after playtime outside, she plays with Starfall, a children's learning app, on her iPad. They hope it accelerates her word recognition. Morgan is entranced watching "Farmer in the Dell" and using her hands to eat orange slices and Cheerios. She needs a mirror in front of her to provide her a target for her mouth. She listens to books, another way to try absorbing information.

Morgan can now count to 20 and say three sentences in a row. Kelly and Kevin have tried to give Morgan a normal life in an abnormal situation, but they worry about the future -- what she won't have and won't be able to experience.

But Morgan has changed some of that outlook too.

"Focus on how she is so loving and has so much pure joy. A lot of parents of special needs [kids] say you can learn so much from these kids, and you really can," Kelly said. "She is, every morning, just so happy, and 'Mama!' Hugs and kisses to strangers. She has none of those behaviors you learn as an adult where you're not kind to people or you don't want to talk to someone.

"She is just open arms, will give you a hug and is so loving, and it's like, 'Wow, this is really what life is about.'"

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'This is why I'm here': A Detroit Lions VP tries to save her daughter from rare disease - ESPN

DUBLIN, Nov. 9, 2020 /PRNewswire/ -- The "Regenerative Medicine Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

The global regenerative medicine market grew at a CAGR of around 16% during 2014-2019. Regenerative medicine refers to a branch of biomedical sciences aimed at restoring the structure and function of damaged tissues and organs. It involves the utilization of stem cells that are developed in laboratories and further implanted safely into the body for the regeneration of damaged bones, cartilage, blood vessels and organs. Cellular and acellular regenerative medicines are commonly used in various clinical therapeutic procedures, including cell, immunomodulation and tissue engineering therapies. They hold potential for the effective treatment of various chronic diseases, such as Alzheimer's, Parkinson's and cardiovascular disorders (CVDs), osteoporosis and spinal cord injuries.

The increasing prevalence of chronic medical ailments and genetic disorders across the globe is one of the key factors driving the growth of the market. Furthermore, the rising geriatric population, which is prone to various musculoskeletal, phonological, dermatological and cardiological disorders, is stimulating the market growth. In line with this, widespread adoption of organ transplantation is also contributing to the market growth. Regenerative medicine minimizes the risk of organ rejection by the body post-transplant and enhances the recovery speed of the patient.

Additionally, various technological advancements in cell-based therapies, such as the development of 3D bioprinting techniques and the adoption of artificial intelligence (AI) in the production of regenerative medicines, are acting as other growth-inducing factors. These advancements also aid in conducting efficient dermatological grafting procedures to treat chronic burns, bone defects and wounds on the skin. Other factors, including extensive research and development (R&D) activities in the field of medical sciences, along with improving healthcare infrastructure, are anticipated to drive the market further. Looking forward, the publisher expects the global regenerative medicine market to continue its strong growth during the next five years.

Competitive Landscape:

The report has also analysed the competitive landscape of the market with some of the key players being Allergan PLC (AbbVie Inc.), Amgen Inc., Baxter International Inc., BD (Becton, Dickinson and Company), Integra Lifesciences Holdings Corporation, Medtronic plc, Mimedx Group Inc., Novartis AG, Osiris Therapeutics Inc. (Smith & Nephew plc) and Thermo Fisher Scientific Inc.

Key Questions Answered in This Report:

Key Topics Covered:

1 Preface

2 Scope and Methodology 2.1 Objectives of the Study2.2 Stakeholders2.3 Data Sources2.3.1 Primary Sources2.3.2 Secondary Sources2.4 Market Estimation2.4.1 Bottom-Up Approach2.4.2 Top-Down Approach2.5 Forecasting Methodology

3 Executive Summary

4 Introduction4.1 Overview4.2 Key Industry Trends

5 Global Regenerative Medicine Market5.1 Market Overview5.2 Market Performance5.3 Impact of COVID-195.4 Market Forecast

6 Market Breakup by Type6.1 Stem Cell Therapy6.1.1 Market Trends6.1.2 Market Forecast6.2 Biomaterial6.2.1 Market Trends6.2.2 Market Forecast6.3 Tissue Engineering6.3.1 Market Trends6.3.2 Market Forecast6.4 Others6.4.1 Market Trends6.4.2 Market Forecast

7 Market Breakup by Application7.1 Bone Graft Substitutes7.1.1 Market Trends7.1.2 Market Forecast7.2 Osteoarticular Diseases7.2.1 Market Trends7.2.2 Market Forecast7.3 Dermatology7.3.1 Market Trends7.3.2 Market Forecast7.4 Cardiovascular7.4.1 Market Trends7.4.2 Market Forecast7.5 Central Nervous System7.5.1 Market Trends7.5.2 Market Forecast7.6 Others7.6.1 Market Trends7.6.2 Market Forecast

8 Market Breakup by End User8.1 Hospitals8.1.1 Market Trends8.1.2 Market Forecast8.2 Specialty Clinics8.2.1 Market Trends8.2.2 Market Forecast8.3 Others8.3.1 Market Trends8.3.2 Market Forecast

9 Market Breakup by Region9.1 North America9.1.1 United States9.1.1.1 Market Trends9.1.1.2 Market Forecast9.1.2 Canada9.1.2.1 Market Trends9.1.2.2 Market Forecast9.2 Asia Pacific9.2.1 China9.2.1.1 Market Trends9.2.1.2 Market Forecast9.2.2 Japan9.2.2.1 Market Trends9.2.2.2 Market Forecast9.2.3 India9.2.3.1 Market Trends9.2.3.2 Market Forecast9.2.4 South Korea9.2.4.1 Market Trends9.2.4.2 Market Forecast9.2.5 Australia9.2.5.1 Market Trends9.2.5.2 Market Forecast9.2.6 Indonesia9.2.6.1 Market Trends9.2.6.2 Market Forecast9.2.7 Others9.2.7.1 Market Trends9.2.7.2 Market Forecast9.3 Europe9.3.1 Germany9.3.1.1 Market Trends9.3.1.2 Market Forecast9.3.2 France9.3.2.1 Market Trends9.3.2.2 Market Forecast9.3.3 United Kingdom9.3.3.1 Market Trends9.3.3.2 Market Forecast9.3.4 Italy9.3.4.1 Market Trends9.3.4.2 Market Forecast9.3.5 Spain9.3.5.1 Market Trends9.3.5.2 Market Forecast9.3.6 Russia9.3.6.1 Market Trends9.3.6.2 Market Forecast9.3.7 Others9.3.7.1 Market Trends9.3.7.2 Market Forecast9.4 Latin America9.4.1 Brazil9.4.1.1 Market Trends9.4.1.2 Market Forecast9.4.2 Mexico9.4.2.1 Market Trends9.4.2.2 Market Forecast9.4.3 Others9.4.3.1 Market Trends9.4.3.2 Market Forecast9.5 Middle East and Africa9.5.1 Market Trends9.5.2 Market Breakup by Country9.5.3 Market Forecast

10 SWOT Analysis10.1 Overview10.2 Strengths10.3 Weaknesses10.4 Opportunities10.5 Threats

11 Value Chain Analysis

12 Porters Five Forces Analysis12.1 Overview12.2 Bargaining Power of Buyers12.3 Bargaining Power of Suppliers12.4 Degree of Competition12.5 Threat of New Entrants12.6 Threat of Substitutes

13 Price Analysis

14 Competitive Landscape14.1 Market Structure14.2 Key Players14.3 Profiles of Key Players14.3.1 Allergan PLC (AbbVie Inc.)14.3.1.1 Company Overview14.3.1.2 Product Portfolio 14.3.1.3 Financials 14.3.1.4 SWOT Analysis14.3.2 Amgen Inc.14.3.2.1 Company Overview14.3.2.2 Product Portfolio14.3.2.3 Financials 14.3.2.4 SWOT Analysis14.3.3 Baxter International Inc.14.3.3.1 Company Overview14.3.3.2 Product Portfolio 14.3.3.3 Financials 14.3.3.4 SWOT Analysis14.3.4 BD (Becton, Dickinson and Company)14.3.4.1 Company Overview14.3.4.2 Product Portfolio 14.3.4.3 Financials 14.3.4.4 SWOT Analysis14.3.5 Integra Lifesciences Holdings Corporation14.3.5.1 Company Overview14.3.5.2 Product Portfolio 14.3.5.3 Financials 14.3.5.4 SWOT Analysis14.3.6 Medtronic Plc14.3.6.1 Company Overview14.3.6.2 Product Portfolio 14.3.6.3 Financials14.3.6.4 SWOT Analysis14.3.7 Mimedx Group Inc.14.3.7.1 Company Overview14.3.7.2 Product Portfolio14.3.7.3 Financials 14.3.8 Novartis AG14.3.8.1 Company Overview14.3.8.2 Product Portfolio 14.3.8.3 Financials14.3.8.4 SWOT Analysis14.3.9 Osiris Therapeutics Inc. (Smith & Nephew plc)14.3.9.1 Company Overview14.3.9.2 Product Portfolio14.3.10 Thermo Fisher Scientific Inc.14.3.10.1 Company Overview14.3.10.2 Product Portfolio 14.3.10.3 Financials14.3.10.4 SWOT Analysis

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Originally posted here:
Worldwide Regenerative Medicine Industry to 2025 - Featuring Allergan, Amgen and Baxter International Among Others - PRNewswire

Dublin, Oct. 30, 2020 (GLOBE NEWSWIRE) -- The "Regenerative Medicine Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

The global regenerative medicine market grew at a CAGR of around 16% during 2014-2019. Regenerative medicine refers to a branch of biomedical sciences aimed at restoring the structure and function of damaged tissues and organs. It involves the utilization of stem cells that are developed in laboratories and further implanted safely into the body for the regeneration of damaged bones, cartilage, blood vessels and organs. Cellular and acellular regenerative medicines are commonly used in various clinical therapeutic procedures, including cell, immunomodulation and tissue engineering therapies. They hold potential for the effective treatment of various chronic diseases, such as Alzheimer's, Parkinson's and cardiovascular disorders (CVDs), osteoporosis and spinal cord injuries.

The increasing prevalence of chronic medical ailments and genetic disorders across the globe is one of the key factors driving the growth of the market. Furthermore, the rising geriatric population, which is prone to various musculoskeletal, phonological, dermatological and cardiological disorders, is stimulating the market growth. In line with this, widespread adoption of organ transplantation is also contributing to the market growth. Regenerative medicine minimizes the risk of organ rejection by the body post-transplant and enhances the recovery speed of the patient.

Additionally, various technological advancements in cell-based therapies, such as the development of 3D bioprinting techniques and the adoption of artificial intelligence (AI) in the production of regenerative medicines, are acting as other growth-inducing factors. These advancements also aid in conducting efficient dermatological grafting procedures to treat chronic burns, bone defects and wounds on the skin. Other factors, including extensive research and development (R&D) activities in the field of medical sciences, along with improving healthcare infrastructure, are anticipated to drive the market further. Looking forward, the publisher expects the global regenerative medicine market to continue its strong growth during the next five years.

Key Market Segmentation:

The publisher provides an analysis of the key trends in each sub-segment of the global regenerative medicine market report, along with forecasts for growth at the global, regional and country level from 2020-2025. Our report has categorized the market based on region, type, application and end user.

Breakup by Type:

Breakup by Application:

Breakup by End User:

Breakup by Region:

Competitive Landscape:

The report has also analysed the competitive landscape of the market with some of the key players being Allergan PLC (AbbVie Inc.), Amgen Inc., Baxter International Inc., BD (Becton, Dickinson and Company), Integra Lifesciences Holdings Corporation, Medtronic plc, Mimedx Group Inc., Novartis AG, Osiris Therapeutics Inc. (Smith & Nephew plc) and Thermo Fisher Scientific Inc.

Key Questions Answered in This Report:

Key Topics Covered:

1 Preface

2 Scope and Methodology 2.1 Objectives of the Study2.2 Stakeholders2.3 Data Sources2.3.1 Primary Sources2.3.2 Secondary Sources2.4 Market Estimation2.4.1 Bottom-Up Approach2.4.2 Top-Down Approach2.5 Forecasting Methodology

3 Executive Summary

4 Introduction4.1 Overview4.2 Key Industry Trends

5 Global Regenerative Medicine Market5.1 Market Overview5.2 Market Performance5.3 Impact of COVID-195.4 Market Forecast

6 Market Breakup by Type6.1 Stem Cell Therapy6.1.1 Market Trends6.1.2 Market Forecast6.2 Biomaterial6.2.1 Market Trends6.2.2 Market Forecast6.3 Tissue Engineering6.3.1 Market Trends6.3.2 Market Forecast6.4 Others6.4.1 Market Trends6.4.2 Market Forecast

7 Market Breakup by Application7.1 Bone Graft Substitutes7.1.1 Market Trends7.1.2 Market Forecast7.2 Osteoarticular Diseases7.2.1 Market Trends7.2.2 Market Forecast7.3 Dermatology7.3.1 Market Trends7.3.2 Market Forecast7.4 Cardiovascular7.4.1 Market Trends7.4.2 Market Forecast7.5 Central Nervous System7.5.1 Market Trends7.5.2 Market Forecast7.6 Others7.6.1 Market Trends7.6.2 Market Forecast

8 Market Breakup by End User8.1 Hospitals8.1.1 Market Trends8.1.2 Market Forecast8.2 Specialty Clinics8.2.1 Market Trends8.2.2 Market Forecast8.3 Others8.3.1 Market Trends8.3.2 Market Forecast

9 Market Breakup by Region9.1 North America9.1.1 United States9.1.1.1 Market Trends9.1.1.2 Market Forecast9.1.2 Canada9.1.2.1 Market Trends9.1.2.2 Market Forecast9.2 Asia Pacific9.2.1 China9.2.1.1 Market Trends9.2.1.2 Market Forecast9.2.2 Japan9.2.2.1 Market Trends9.2.2.2 Market Forecast9.2.3 India9.2.3.1 Market Trends9.2.3.2 Market Forecast9.2.4 South Korea9.2.4.1 Market Trends9.2.4.2 Market Forecast9.2.5 Australia9.2.5.1 Market Trends9.2.5.2 Market Forecast9.2.6 Indonesia9.2.6.1 Market Trends9.2.6.2 Market Forecast9.2.7 Others9.2.7.1 Market Trends9.2.7.2 Market Forecast9.3 Europe9.3.1 Germany9.3.1.1 Market Trends9.3.1.2 Market Forecast9.3.2 France9.3.2.1 Market Trends9.3.2.2 Market Forecast9.3.3 United Kingdom9.3.3.1 Market Trends9.3.3.2 Market Forecast9.3.4 Italy9.3.4.1 Market Trends9.3.4.2 Market Forecast9.3.5 Spain9.3.5.1 Market Trends9.3.5.2 Market Forecast9.3.6 Russia9.3.6.1 Market Trends9.3.6.2 Market Forecast9.3.7 Others9.3.7.1 Market Trends9.3.7.2 Market Forecast9.4 Latin America9.4.1 Brazil9.4.1.1 Market Trends9.4.1.2 Market Forecast9.4.2 Mexico9.4.2.1 Market Trends9.4.2.2 Market Forecast9.4.3 Others9.4.3.1 Market Trends9.4.3.2 Market Forecast9.5 Middle East and Africa9.5.1 Market Trends9.5.2 Market Breakup by Country9.5.3 Market Forecast

10 SWOT Analysis10.1 Overview10.2 Strengths10.3 Weaknesses10.4 Opportunities10.5 Threats

11 Value Chain Analysis

12 Porters Five Forces Analysis12.1 Overview12.2 Bargaining Power of Buyers12.3 Bargaining Power of Suppliers12.4 Degree of Competition12.5 Threat of New Entrants12.6 Threat of Substitutes

13 Price Analysis

14 Competitive Landscape14.1 Market Structure14.2 Key Players14.3 Profiles of Key Players14.3.1 Allergan PLC (AbbVie Inc.)14.3.1.1 Company Overview14.3.1.2 Product Portfolio 14.3.1.3 Financials 14.3.1.4 SWOT Analysis14.3.2 Amgen Inc.14.3.2.1 Company Overview14.3.2.2 Product Portfolio14.3.2.3 Financials 14.3.2.4 SWOT Analysis14.3.3 Baxter International Inc.14.3.3.1 Company Overview14.3.3.2 Product Portfolio 14.3.3.3 Financials 14.3.3.4 SWOT Analysis14.3.4 BD (Becton, Dickinson and Company)14.3.4.1 Company Overview14.3.4.2 Product Portfolio 14.3.4.3 Financials 14.3.4.4 SWOT Analysis14.3.5 Integra Lifesciences Holdings Corporation14.3.5.1 Company Overview14.3.5.2 Product Portfolio 14.3.5.3 Financials 14.3.5.4 SWOT Analysis14.3.6 Medtronic Plc14.3.6.1 Company Overview14.3.6.2 Product Portfolio 14.3.6.3 Financials14.3.6.4 SWOT Analysis14.3.7 Mimedx Group Inc.14.3.7.1 Company Overview14.3.7.2 Product Portfolio14.3.7.3 Financials 14.3.8 Novartis AG14.3.8.1 Company Overview14.3.8.2 Product Portfolio 14.3.8.3 Financials14.3.8.4 SWOT Analysis14.3.9 Osiris Therapeutics Inc. (Smith & Nephew plc)14.3.9.1 Company Overview14.3.9.2 Product Portfolio14.3.10 Thermo Fisher Scientific Inc.14.3.10.1 Company Overview14.3.10.2 Product Portfolio 14.3.10.3 Financials14.3.10.4 SWOT Analysis

For more information about this report visit https://www.researchandmarkets.com/r/ywnlq5

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Link:
Outlook on the Regenerative Medicine Global Market to 2025 - Impact of COVID-19 on the Market - GlobeNewswire

Company Announcement

Copenhagen, Denmark; August 20, 2020 Genmab A/S (Nasdaq: GMAB) announced today that the U.S. Food and Drug Administration (U.S. FDA) has approved the use of DARZALEX (daratumumab) in combination with carfilzomib and dexamethasone (DKd) for the treatment of adult patients with relapsed/refractory multiple myeloma who have received one to three previous lines of therapy. A supplemental Biologics License Application (sBLA) for this indication was submitted by Genmabs licensing partner, Janssen Biotech, Inc. (Janssen), in February 2020. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

We are extremely pleased that multiple myeloma patients in the U.S. will now have yet another treatment option as this is the eighth overall U.S. FDA approval for DARZALEX and the fifth in the relapsed/refractory setting. In addition, DARZALEX is now the first CD38 antibody approved for use in combination with carfilzomib, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The combination has been approved in two carfilzomib dosing regimens, 70 mg/m2 once weekly and 56 mg/m2 twice weekly, based on positive results from the Phase 3 CANDOR and Phase 1b EQUULEUS studies. CANDOR was an Amgen-sponsored study, co-funded by Janssen Research & Development, LLC. EQUULEUS was sponsored by Janssen Research & Development, LLC.

About the CANDOR studyThe Phase 3 trial (NCT03158688) was a randomized, open-label study that included 466 patients with multiple myeloma who had relapsed after 1 to 3 prior therapies. Patients were randomized to receive either DKd or carfilzomib and dexamethasone (Kd) alone. In the daratumumab treatment arm, patients received 8 milligrams per kilogram (mg/kg) on days 1 and 2 of cycle 1, then 16 mg/kg once weekly for the remaining doses of the first 2 cycles, then every 2 weeks for 4 cycles (cycles 3 to 6), and then every 4 weeks for the remaining cycles or until disease progression. In both treatment arms carfilzomib was dosed twice weekly (20 mg/m2 on cycle 1 days 1 and 2 and 56 mg/m2 beginning on cycle 1 day 8 and thereafter) and dexamethasone was given weekly (40 mg orally or via IV infusion). The primary endpoint of the study was progression free survival (PFS).

About the EQUULEUS (MMY1001) Study The Phase 1b EQUULEUS (NCT01998971) study was an open label, multi-cohort trial that evaluated the safety, tolerability, and dose regimen of daratumumab when administered in combination with various treatment regimens for the treatment of multiple myeloma. Among the regiments evaluated, the combination of DKd compared to Kd alone was studied in 85 patients with relapsed/refractory multiple myeloma who had received one to three prior lines of therapy using a once-weekly dosing regimen. DKd was evaluated at a starting dose of 20 mg/m2, which was increased to 70 mg/m2 on Cycle 1, Day 8 and onward.

About multiple myelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 26,000 new patients were expected to be diagnosed with multiple myeloma and approximately 13,650 people were expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX (daratumumab)DARZALEX (daratumumab) has become a backbone therapy in the treatment of multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with carfilzomib and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma who have received one to three previous lines of therapy; in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma.

Story continues

DARZALEX is indicated for the treatment of adult patients in Europe via intravenous infusion or subcutaneous administration: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy7. Daratumumab is the first subcutaneous CD38 antibody approved in Europe for the treatment of multiple myeloma. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S.

In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, is approved in the United States for the treatment of adult patients with multiple myeloma: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for ASCT; in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy; and as monotherapy, in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.8 DARZALEX FASPRO is the first subcutaneous CD38 antibody approved in the U.S. for the treatment of multiple myeloma.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,9,10,11,12

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis and T-cell acute lymphocytic leukemia (ALL). Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company is the creator of the following approved antibodies: DARZALEX (daratumumab, under agreement with Janssen Biotech, Inc.) for the treatment of certain multiple myeloma indications in territories including the U.S., Europe and Japan, Kesimpta (subcutaneous ofatumumab, under agreement with Novartis AG), for the treatment of adults with relapsing forms of multiple sclerosis in the U.S. and TEPEZZA (teprotumumab, under agreement with Roche granting sublicense to Horizon Therapeutics plc) for the treatment of thyroid eye disease in the U.S. A subcutaneous formulation of daratumumab, known as DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) in the U.S., has been approved in the U.S. and Europe for the treatment of adult patients with certain multiple myeloma indications. The first approved Genmab created therapy, Arzerra (ofatumumab, under agreement with Novartis AG), approved for the treatment of certain chronic lymphocytic leukemia indications, is available in Japan and is also available in other territories via compassionate use or oncology access programs. Daratumumab is in clinical development by Janssen for the treatment of additional multiple myeloma indications, other blood cancers and amyloidosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with sites in Utrecht, the Netherlands, Princeton, New Jersey, U.S. and Tokyo, Japan.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs most recent Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra and Kesimpta are trademarks of Novartis AG or its affiliates. DARZALEX and DARZALEX FASPRO are trademarks of Janssen Pharmaceutica NV. TEPEZZA is a trademark of Horizon Therapeutics plc.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 National Cancer Institute. "A Snapshot of Myeloma." Available at http://www.cancer.gov/research/progress/snapshots/myeloma. Accessed June 2016. 3 Globocan 2018. United States of America Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/840-united-states-of-america-fact-sheets.pdf.4 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.5 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20166 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20197 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed June 20208 DARZALEX FASPRO Prescribing information, May 2020. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761145s000lbl.pdf Last accessed May 20209 De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.10 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.11 Krejcik, MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.12 Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

Company Announcement no. 38CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

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Genmab Announces Janssen Granted US FDA Approval for DARZALEX (daratumumab) in Combination with Carfilzomib and Dexamethasone in Relapsed or...

Kyprolis and Velcade have 14 to 16 claims per patient per year for Multiple Myeloma compared to Darzalex and Empliciti which have 9 claims Kyprolis and Velcade have 14 to 16 claims per patient per year for Multiple Myeloma compared to Darzalex and Empliciti which have 9 claims

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Dexurs analysis of Medicare claims data showed that on an average, Kyprolis and Velcade have 14 to 16 claims per patient per year for Multiple Myeloma (MM) compared to Darzalex and Empliciti which have 9 claims per patient. The analysis was based on a sample of Medicare patients between Jan 2019 and Dec 2019, and looked at the J code usage of the drugs for the condition.The number of claims per patient data is a proxy for the number of injections / IV infusions / doses required by a patient. The study also tries to contrast the usage of these medications across three diagnosis categories- MM patients who have not achieved remission (C9000), MM patients in remission (C9001), and MM patients in relapse (C9002).

Multiple myeloma is a form of blood cancer that involves the neoplastic proliferation of plasma cells, a type of white blood cells formed within the bone marrow. While the earlier stages of the disease tend to be asymptomatic, patients may experience symptoms like bone pain, bleeding, frequent infections, and anemia with progression of the cancer. Although there is no cure for MM, a number of treatment options including chemotherapy, stem cell transplantation, radiation therapy, and targeted therapy, can help in managing the progression of the disease and relieving the symptoms.

The drugs considered under this study are targeted therapies approved for the treatment of adult patients with multiple myeloma, alone or in combination with other medication. Unlike chemotherapy, these drugs specifically target the cancer cells and the mechanisms that support their growth, promising better results and fewer adverse effects. Kyprolis (carfilzomib) and Velcade (bortezomib) are proteasome inhibitors that can trigger apoptosis in cancer cells by blocking the action of proteasome, an enzyme complex that is critical in the regulation of cell-cycle. Darzalex (daratumumab) and Empliciti (elotuzumab) are monoclonal antibodies that enable the immune system to identify and kill cancer cells by targeting specific proteins on the cell surface.

The average usage of drug per patient was seen to be highest among MM patients who had not achieved remission, and least among MM patients in remission. An exception to this trend was Kyprolis, which had similar usage among patients in remission and patients in relapse, with the former having a marginally higher number of claims per patient.

Amgens Kyprolis had an average of 15.9 claims per patient in a year. The proteasome inhibitor indicated for the treatment of patients with relapsed/refractory MM, is used as a monotherapy, or as a combination therapy along with dexamethasone, or lenalidomide and dexamethasone.

Velcade, manufactured by Millennium Pharmaceuticals/Takeda Oncology in the U.S, had an average of 13.8 claims per patient in a year. It was noted to have the largest share of claims among the drugs, across the three diagnosis categories for multiple myeloma. Approved for the treatment of newly diagnosed and relapsed/refractory myeloma, the drug is used alone or as a part of combination therapies.

Darzalex, a CD38-directed cytolytic antibody by Janssen Biotech, had an average of 9.9 claims per patient in a year for MM. It is indicated for the treatment of MM as a monotherapy or in combination with other drugs including lenalidomide, dexamethasone, and bortezomib.

Bristol-Myers Squibbs Empliciti had an average of 9.3 claims per patient. It is a SLAMF7-directed immunostimulatory antibody approved for the treatment of MM in combination with lenalidomide and dexamethasone, or pomalidomide and dexamethasone

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Kyprolis and Velcade have 14 to 16 claims per patient per year for Multiple Myeloma compared to Darzalex and Empliciti which have 9 claims - Dexur

DUBLIN--(BUSINESS WIRE)--The "Dilated Cardiomyopathy (DCM) - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines report has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the Dilated Cardiomyopathy, historical and forecasted epidemiology as well as the Dilated Cardiomyopathy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The Dilated Cardiomyopathy market report provides current treatment practices, emerging drugs, Dilated Cardiomyopathy market share of the individual therapies, current and forecasted Dilated Cardiomyopathy market size from 2017 to 2030 segmented by seven major markets. The report also covers current Dilated Cardiomyopathy treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Epidemiology

The Dilated Cardiomyopathy epidemiology division provides the insights about historical and current Dilated Cardiomyopathy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

The disease epidemiology covered in the report provides historical as well as forecasted Dilated Cardiomyopathy epidemiology segmented as [Total Prevalent Population of Dilated Cardiomyopathy, Total Diagnosed Population of Dilated Cardiomyopathy, Familial and non-familial cases of Dilated Cardiomyopathy, Gender-Specific Cases of Dilated Cardiomyopathy, and Total Treated Cases of Dilated Cardiomyopathy] scenario of Dilated Cardiomyopathy in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Key Findings

Drug Chapters

Drug chapter segment of the Dilated Cardiomyopathy report encloses the detailed analysis of Dilated Cardiomyopathy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Dilated Cardiomyopathy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Approved Drug

Corlanor (ivabradine): Amgen

Corlanor (ivabradine) is a hyperpolarization-activated cyclic nucleotide-gated channel blocker that reduces the spontaneous pacemaker activity of the cardiac sinus node by selectively inhibiting the If current, resulting in heart rate reduction with no effect on ventricular repolarization and no effects on myocardial contractility.

The US FDA approval of Corlanor (ivabradine) for the treatment of stable symptomatic heart failure (HF) due to dilated cardiomyopathy in pediatric patients aged 6 months to 18 years was based on a randomized, double-blind, placebo-controlled trial in 116 patients aged 6 months to less than 18 years with symptomatic DCM in sinus rhythm, NYHA/Ross class II to IV HF, and left ventricular ejection fraction 45%. The primary endpoint of the study was 20% reduction in resting heart rate from baseline without bradycardia or symptoms after an initial titration period.

Emerging Drugs

PF-07265803/ARRY-371797/ARRY-797: Pfizer

ARRY-371797 which is also known as ARRY-797 is an oral, p38 mitogen activated protein kinase (MAPK) inhibitor discovered by Array scientists. Compared to other p38 MAPK inhibitors ARRY-797 has unique and differentiated properties: it is highly selective, retains exceptional potency in whole blood and possesses a favorable pharmacokinetic profile. It is currently under phase III trial for the treatment of patients affected with dilated cardiomyopathy due to a Lamin A/C gene mutation. In the year 2019, Pfizer completed the acquisition of Array Biopharma to expand its pipeline and currently this drug is in phase III pipeline drugs of Pfizer with name PF-07265803 for the treatment of patients affected by dilated cardiomyopathy.

Ixmyelocel-T: Vericel

Ixmyelocel-T is an investigational autologous expanded multicellular therapy manufactured from the patient's own bone marrow using Vericel's proprietary, highly automated, fully closed cell-processing system. This process selectively expands the population of mesenchymal stromal cells and alternatively activated macrophages, which are responsible for production of anti-inflammatory and pro-angiogenic factors known to be important for repair of damaged tissue. Ixmyelocel-T has been designated as an orphan drug by the U.S. Food and Drug Administration for use in the treatment of DCM. However, currently the development of this drug is at halt because as per the recent news the company do not have current plans to initiate or fund a phase III trial for this drug at their own.

BC007: Berlin Cures GmbH

BC007 is a DNA aptamer-based compound that binds to and eliminates pathogenic autoantibodies directed against the beta-1 adrenoceptor, a receptor belonging to the large family of cell surface receptors known as G-protein coupled receptors that regulate the heart's rate and contraction strength.

Ifetroban: Cumberland Pharmaceuticals

Ifetroban is a potent and selective inhibitor of the thromboxane receptor (TPr), preventing fibrosis and an inflammatory response. It was initially developed by Bristol-Myers Squibb as an anti-platelet agent to prevent blood clots (blood thrombus), and was acquired by Cumberland in 2011. It is believed that this drug molecule is able to stop important molecular signals that mediate inflammation and fibrosis (tissue scaring) mechanisms in the heart, triggered by the loss of dystrophin protein.

Danicamtiv/MYK-491: MyoKardia

MYK-491 is an orally-administered small molecule designed to increase the number of myosin-actin cross-bridges formed during cardiac muscle contraction while having minimal impact on diastolic function. In the heart, myosin is the motor protein that binds to actin to generate the force and movement of contraction. In patients with dilated cardiomyopathy and systolic heart failure, in which the left ventricle of the heart is too distended and weak to adequately pump blood to meet the body's needs, MYK-491 is intended to increase myosin-actin engagement, thereby targeting the biomechanical defects underlying disease and improving cardiac contractility.

CAP-1002: Capricor Therapeutics

CAP-1002, Capricor's lead product candidate, is a proprietary allogeneic adult stem cell therapy for the treatment of heart disease. The product is derived from donor heart tissue. The cells are expanded in the laboratory using a specialized process and then introduced directly into a patient's heart via infusion into a coronary artery using standard cardiac catheterization techniques. CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that has been shown to exert potent immunomodulatory activity and alters the immune system's activity to encourage cellular regeneration.

Market Outlook

Besides treating any recognizable and reversible underlying causes, the management and treatment of DCM are in concordance with the standard heart failure guidelines. Currently, the treatment pattern of DCM is mainly dependent on pharmacological therapy, pacing therapy, surgical options, and Corlanor (ivabradine).

The pharmacological therapies consist of diuretics, inotropic agents, afterload reducing agents, beta-blockers, anticoagulation medications, anti-arrhythmia medications. The main diuretics that are prescribed for the treatment are furosemide, spironolactone, bumetanide, and metolazone. Common side effects of diuretics include dehydration and abnormalities in the blood chemistries particularly potassium loss. Inotopric agents that are prescribed for the treatment are digoxin, dobutamine, dopamine, epinephrine, norepinephrine, vasopressin, and milrinone. Some afterload reducing medications include angiotensin-converting enzyme inhibitors (ACE inhibitors) such as captopril, enalMay, lisinopril, monopril, angiotensin I blocker such as losartan. Losartan and milrinone are inotropic agents that also relax the arteries. Stronger anticoagulation drugs are warfarin, heparin, and enoxaparin; these drugs require careful monitoring with regular blood testing.

Angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARB) have shown benefit in the treatment of heart failure with reduced ejection fraction and are suggested for the patients affected with DCM. Aldosterone receptor blockade with spironolactone or eplerenone also is recommended in patients with New York Heart Association (NYHA) heart failure class II-IV and systolic dysfunction. Similarly, beta-blockade with carvedilol, bisoprolol, or long-acting metoprolol is recommended in all patients with heart failure with reduced ejection fraction without any contraindications. The addition of isosorbide dinitrate plus hydralazine also has shown to increase survival amongst those with advanced disease.

In some cases, beta-blockers allow an enlarged heart to become more normal in size. Common beta-blockers include carvedilol, metoprolol, propranolol, and atenolol. Side effects include dizziness, low heart rate, low blood pressure, and, in some cases, fluid retention, fatigue, impaired school performance, and depression. The choice of anticoagulation drugs depends on how likely it is that a blood clot will form. Less strong anticoagulation medications include aspirin and dipyridamole. Common anti-arrhythmia medications include amiodarone, procainamide, and lidocaine. Also, Corlanor (ivabradine) is an approved therapy for the treatment of 6 months to 18 years old patient affected by Dilated Cardiomyopathy.

Key Findings

According to the report, Dilated Cardiomyopathy market in the 7MM is expected to change in the study period 2017-2030. The total therapeutic market of Dilated Cardiomyopathy in seven major markets was found to be USD 244 million in 2017 which is expected to increase during the study period (2017-2030).

The United States Market Outlook

In 2017, the total market size of Dilated Cardiomyopathy therapies was estimated to be USD 142.9 million in the United States which is expected to increase in the study period (2017-2030).

EU5 Countries: Market Outlook

In 2017, the total market size of Dilated Cardiomyopathy therapies was found to be USD 74.4 million in the EU5 countries which is expected to increase in the study period (2017-2030).

Japan Market Outlook

The total market size of Dilated Cardiomyopathy therapies in Japan was found to be USD 27.1 million in 2017 which is also expected to increase during the study period (2017-2030).

Pipeline Development Activities

The drugs which are in pipeline include:

1. PF-07265803/ARRY-371797/ARRY-797: Pfizer

2. Ixmyelocel-T: Vericel

3. BC007: Berlin Cures GmbH

4. Ifetroban: Cumberland Pharmaceuticals

5. Danicamtiv/MYK-491: MyoKardia

6. CAP-1002: Capricor Therapeutics

Access and Reimbursement Scenario

The record published in United HealthCare Services, in the United States, stated that reimbursement is eligible for the CPT codes related to various genetic testing for cardiac disease. CPT code 81439 includes indications such as hereditary cardiomyopathy (e.g., hypertrophic cardiomyopathy, dilated cardiomyopathy, arrhythmogenic right ventricular cardiomyopathy), genomic sequence analysis panel - must include sequencing of at least five cardiomyopathy-related genes (e.g., DSG2, MYBPC3, MYH7, PKP2, TTN). Moreover, cardiomyopathies that present primarily as neuromuscular disorders and related genetic testing are also covered in the Medical Policy.

KOL Views

To keep up with current market trends, we take KOL's and SME's opinion working in Dilated Cardiomyopathy domain through primary research to fill the data gaps and validates our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns and Dilated Cardiomyopathy market trend. This will support the clients in the introduction of potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

The publisher performs Competitive and Market Intelligence analysis of the Dilated Cardiomyopathy Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

Report Highlights

Companies Mentioned

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/qfjown

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Worldwide Dilated Cardiomyopathy (DCM) Market Insights, Epidemiology and Forecast - 2030 - ResearchAndMarkets.com - Business Wire

Global Multiple Myeloma Treatment Marketsize was valued US$ XX Mn. in 2019 and the total revenue is expected to grow at 11.34% from 2019 to 2027, reaching nearly US$ XX Mn.

The report study has analyzed the revenue impact of COVID -19 pandemic on the sales revenue of market leaders, market followers, and market disrupters in the report, and the same is reflected in our analysis.

Multiple myeloma, also known as Kahlers disease, is a type of blood cancer of plasma cells that are found in the bone marrow. Multiple myeloma causes cancer cells to accrue in the bone marrow, where they attack the strong blood cells.

Multiple myeloma treatments have developed significantly above the last decade. New multiple myeloma treatments have provided efficient survival rates between myeloma patients. It has been also observed that the future drug pipeline of multiple myeloma is promising, biological drugs and stem cell-based therapies are likely to fuel the multiple myeloma treatment market in the upcoming years. On the other hand, the costs of radiotherapeutic equipment implementation, a limited number of target patients population, strict legal regulations are expected to hamper the market growth. Likewise, the MMR report contains a detailed study of factors that will drive and restrain the growth of the multiple myeloma treatment market globally.

Multiple Myeloma accounts for approximately 2.5% of the cancer-related deaths globally and is the second most major type of blood cancer next to Hodgkins Lymphoma. According to the World Cancer Research Fund, in 2018, above 159500 cases of multiple myeloma were diagnosed with the condition, where the occurrence rate among women and men was found in the ratio 1.2:1. The onset of the disease occurs after the age of 60. In recent times, the age of onset is drastically decreasing. In the year 2001, only two medications were available for treating multiple myeloma but now in 2020, 18 medicines are available. Moreover, there are over 25 FDA-approved drugs for treating multiple myeloma with therapeutics such as pomalidomide, carfilzomib, panobinostat, and ixazomib. The availability of new medications has given new hope for better treatments and better results and thus affecting the growth of the market as well. However, the survival of patients with a limited response while receiving treatment with primary immunodeficiency therapy remains poor and is one of the major challenges.

The MMR report covers the segments in the multiple myeloma treatment market such as type and application. By application, the hospital is expected to continue to hold the largest XX.85% share in multiple myeloma treatments market thanks to growing specialist doctors providing the best chance of long term survival.

North Americas multiple myeloma treatments market was valued at US$ XX.26 Mn. in 2019 and is expected to reach a value of US$ XX.13 Mn. by 2027, with a CAGR of 9.3%. The number of patients in the U.S is growing YoY with nearly 14600 new cases diagnosed annually. In 2017 alone there were approximately 142000 patients diagnosed for multiple myeloma.

Europe and the South African population are prone to develop multiple myeloma when compared with Asian economies. Though, the population in the APAC region outwits Europe and Africa. Further, growing the adoption rate of novel therapies, coupled with the support from the government along with non-government organizations and improving the survival of multiple myeloma patients.

The research study includes the profiles of leading players operating in the global multiple myeloma treatment market. Eli Lilly Company acquired ARMO Biosciences to develop immunotherapies for the treatment of cancer, hypercholesterolemia, inflammatory, and fibrosis diseases.

The objective of the report is to present a comprehensive analysis of the Global Multiple Myeloma Treatment Market including all the stakeholders of the industry. The past and current status of the industry with forecasted market size and trends are presented in the report with the analysis of complicated data in simple language. The report covers all the aspects of the industry with a dedicated study of key players that includes market leaders, followers, and new entrants. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors of the market has been presented in the report. External as well as internal factors that are supposed to affect the business positively or negatively have been analyzed, which will give a clear futuristic view of the industry to the decision-makers.The report also helps in understanding Global Multiple Myeloma Treatment Market dynamics, structure by analyzing the market segments and projects the Global Multiple Myeloma Treatment Market size. Clear representation of competitive analysis of key players by Application, price, financial position, Product portfolio, growth strategies, and regional presence in the Global Multiple Myeloma Treatment Market make the report investors guide.Scope of the Global Multiple Myeloma Treatment Market

Global Multiple Myeloma Treatment Market, by Applications

Hospitals Clinics Cancer Treatment and Rehabilitation CentersGlobal Multiple Myeloma Treatment Market, by Type

Proteasome Inhibitors Immunomodulatory Agents (IMiDs) Histone Deacetylase (HDAC) Inhibitors Immunotherapy Cytotoxic ChemotherapyGlobal Multiple Myeloma Treatment Market, by Region

Asia Pacific North America Europe South America Middle East & AfricaKey players operating in Global Multiple Myeloma Treatment Market

Celgene Corporation Janssen Biotech, Inc. Bristol-Myers Squibb Company Novartis AG Cellectar Biosciences Inc. Millennium Pharmaceuticals Amgen, Inc. bbVie Genzyme Corporation Juno Therapeutics Eli Lilly and Company Glenmark Pharma

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Global Multiple Myeloma Treatment Market-Industry Analysis and forecast 2019 2027: By Application, Type, and Region. - Good Night, Good Hockey

Hematopoietic stem cell transplantation (HSCT) may offer the chance of a cure for patients with leukemia and other blood cancers, but the process of preparing the body to receive such a transplant can be brutal, involving whole body irradiation as well as chemotherapy conditioning. New results show that both steps are needed: a trial that omitted whole body irradiation in young patients with acute lymphoblastic leukemia (ALL) was stopped early because of significantly poorer outcomes.

The multicenter, global FORUM (For Omitting Radiation Under Majority Age) trial involved 75 centers in 17 countries between 2013 and 2018.

"Our study shows significantly better outcomes for total body irradiation compared to myeloablative chemo-conditioning arms, with no differences between the [two]chemo-conditioning groups," concluded Christina Peters, MD, professor of pediatrics in the Department of Stem Cell Transplantation at St Anna Children's Hospital in Vienna, Austria.

The findings in favor of total body irradiation were pronounced enough that the study was halted early by a safety committee, she added.

Peters presented the findings as part of the virtual European Hematology Association (EHA) 25th Annual Congress 2020.

Describing the results as "sobering," session co-moderator Shai Izraeli, MD, director of the Department of Hematology-Oncology at Schneider Children's Medical Center, in Petah Tikva, Israel, said an online comment from the virtual meeting audience reflected the reaction to these unwelcome results: "So we are still stuck with total body irradiation?"

Peters responded the good news is that the number of patients needing to undergo stem cell transplants is low, and with research advances, may hopefully drop even further.

"Only 10% of patients under the age of 18 nowadays undergo allogeneic HSCT, and perhaps in the future that will become even less if we are able to rescue some of the groups with other immunological measures such as CAR-T cells and antibodies," she said.

"I think it is very important to better identify those who really need total body irradiation in the future," she added.

Commenting for Medscape Medical News, Izraeli agreed.

"The prognosis of children after bone marrow transplantation is excellent the majority are cured from their leukemia," he said. "And we have to remember that those who undergo bone marrow transplant have the worst leukemias."

He pointed out that, in fact, contemporary chemotherapy alone is effective in the treatment of more than 90% of patients with ALL younger than aged 18.

For the 10% of patients who do not respond to chemotherapy alone and undergo allogeneic HSCT, about 50% to 80% of pediatric patients who have resistant leukemia are cured. However, the total body irradiation used to prepare the body to receive the transplant is linked to potentially serious consequences later in life, including sterility, lung problems, growth retardation, and secondary cancer.

To determine if the irradiation component could be safely replaced with a chemotherapy-based conditioning approach, Peters and colleagues conducted the FORUM trial.

In total 413 patients undergoing HSCT were enrolled and randomized to pretransplant conditioning with total body irradiation and etoposide (n = 202) or a chemotherapy-only approach with fludarabine/thiotepa/busulfan (flu/thio/bu; n = 99) or fludarabine/thiotepa/treosulfan (treo; n = 93).

Most patients (72%) had B-cell precursor ALL and 23% had T-cell ALL. Just over half (54%) were transplanted in first complete remission (CR1), 40% in CR2, and 4% in CR3.

The source of stem cells was bone marrow for most patients (82%); peripheral blood stem cell for 12%, and cord blood for 4%.

The aim of the study was to demonstrate noninferiority with the chemotherapy approach.

However, the significantly inferior outcome observed in the chemotherapy-only group led to randomization being halted in March 2019.

The 2-year overall survival in the intent-to-treat (ITT) analysis, with a mean observation time of 2.1 years, was 0.75 0.04 for chemo-conditioning versus 0.91 0.02 for total body irradiation/etoposide (ITT P < .001).

The ITT analysis showed relapses were significantly higher in the chemo-conditioning group (2-year cumulative incidence of relapse [CIR], 0.33) compared with the total body irradiation group (CIR, 0.12; P < .001).

The 2-year event-free survival (EFS) rate was also significantly higher in the total body irradiation group (0.86 vs 0.58; P < .001), and transplant-related mortality over 2 years was lower with total body irradiation (0.02 vs 0.09; P = .02).

A per-protocol analysis showed the 2-year overall survival to be the same in the two chemotherapy groups (both 0.77 0.05) compared with 0.91 0.02 in the total body irradiation group (P = .003).

"In this cohort [the 91% overall survival rate] may even be lower than contemporary intensive frontline therapy results that are achieved nowadays," Peters said.

In looking at subgroups, there were no significant differences according to age group or cancer phenotype, while MLL rearrangement was associated with higher relapse incidence.

Remission status was found to notably influence EFS, dropping from 0.91 in CR1 patients with total body irradiation to 0.76 in CR2 patients. However, total body irradiation remained significantly higher compared with the chemo-conditioning groups in CR1 (P = .004) and CR2 (P < .001).

Transplant-related mortality was not significantly different between the total body irradiation and chemo-conditioning groups in the CR1 or CR2 groups (P = .09 and P = .18, respectively), despite the significant difference when remission status was not included.

Overall, "we tried to identify subgroups in which total body irradiation might be eliminated, however in all analyses, total body irradiation was better than chemo-conditioning in all arms," Peters said.

Meanwhile, the findings underscore that even when patients cannot receive total body irradiation, the alternative chemo-conditioning therapy in fact shows favorable efficacy on its own, Izraeli said.

"The prognosis of the chemotherapy group is also quite remarkably good, although less than the total body irradiation arm. This means that if for some reason total body irradiation cannot be given, the chemotherapy is a very reasonable alternative."

Peters has reported relationships with Amgen, Novartis, Pfizer, Medac, Jazz, and Neovii. Izraeli has reported no relevant financial relationships.

EHA 2020 Congress. Presented June 12, 2020. Abstract S102.

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Trial Omitting Total Body Irradiation Before HSCT Stopped Early - Medscape

SOUTH SAN FRANCISCO--(BUSINESS WIRE)--Imago BioSciences, Inc. (Imago), a clinical stage biopharmaceutical company developing innovative treatments for myeloid diseases, today announced that positive Phase 2 data from its lead pipeline program bomedemstat (IMG-7289), will be presented at the Virtual Edition of the 25th EHA Annual Congress beginning June 12, 2020.

Title: A PHASE 2 STUDY OF BOMEDEMSTAT (IMG-7289), A LYSINE-SPECIFIC DEMETHYLASE-1 (LSD1) INHIBITOR, FOR THE TREATMENT OF LATER-STAGE MYELOFIBROSIS (MF)

Session Topic: 16. Myeloproliferative Neoplasms

Final Abstract Code: EP1080

The data demonstrates the potential of bomedemstat as a monotherapy in intermediate-2 and high-risk patients with myelofibrosis who have become intolerant of, or resistant to, or are ineligible for a Janus Kinase (JAK) inhibitor.

Imago is currently conducting a Phase 2 study of bomedemstat in five countries. Clinical endpoints include spleen volume reduction, reduction in total symptom scores, and improvement in circulating inflammatory cytokines, anemia, bone marrow fibrosis and blast count. For additional information, visit cliniciatrials.gov (NCT03136185).

About Bomedemstat (IMG-7289)

Bomedemstat is being evaluated in an open-label Phase 2 clinical trial for the treatment of advanced myelofibrosis (MF), a bone marrow cancer that interferes with the production of blood cells. The endpoints include spleen volume reduction and symptom improvement at 12 and 24 weeks of treatment. Bomedemstat is used as monotherapy in patients who are resistant to, intolerant of, or ineligible for a Janus Kinase (JAK) inhibitor.

Bomedemstat is a small molecule developed by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme shown to be vital in cancer stem/progenitor cells, particularly neoplastic bone marrow cells. In non-clinical studies, IMG-7289 demonstrated robust in vivo anti-tumor efficacy across a range of myeloid malignancies as a single agent and in combination with other chemotherapeutic agents. Bomedemstat (IMG-7289) is an investigational agent currently being evaluated in ongoing clinical trials (ClinicalTrials.gov Identifier: NCT03136185 and NCT02842827). Bomedemstat has FDA Orphan Drug and Fast Track Designation for the treatment of myelofibrosis and essential thrombocythemia, and Orphan Drug Designation for treatment of acute myeloid leukemia.

About Imago BioSciences

Imago BioSciences is a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer therapeutics targeting epigenetic enzymes. Imago has developed a series of compounds that inhibit LSD1, an epigenetic enzyme critical for cancer stem cell function and differentiation. Imago is advancing the clinical development of its first LSD1 inhibitor, bomedemstat, for the treatment of myeloid neoplasms including myelofibrosis and essential thrombocythemia. Imago BioSciences is backed by leading strategic and venture investors including a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital. The company is based in South San Francisco, California. To learn more, visit http://www.imagobio.com.

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Imago BioSciences To Present Update on Phase 2 results of Bomedemstat (IMG-7289), a Lysine Specific Demethylase-1 (LSD1) Inhibitor for the Treatment...

Leukaemia is the cancer of blood cells. Blood cells originate from HSCs, hematopoietic stem cells, in the bone marrow. Thereafter they undergo maturation process called hematopoiesis. Multipotent hematopoietic stem cells often undergo a process of differentiation while in maturation stage to give rise to progenitor cells of myeloid and lymphoid origin. These Myeloid cells include neutrophils, basophils, monocytes, macrophages, erythrocytes, dendritic cells, eosinophils, and megakaryocytes or platelets. While, Lymphoid cells include B cells, T cells and natural killer cells.

Recently in 2016, Global Leukaemia Therapeutics Market was valued at nearly USD 9.44 billion and is expected to grow at a CAGR of 4.1% between 2017 and 2022, accounting to market worth USD 11.97 billion by end of 2022.

The Final Report will cover the impact analysis of COVID-19 on this industry.

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Normally, the blood forming cells in the bone marrow produce leukocytes, that protects against viruses and bacteria. If these leukocytes get damaged and if they are left untreated they get accumulated in the body and invade in other parts like liver, spleen and central nervous system, hence damaging the entire body. The main reasons causing leukaemia are ionizing radiation, smoking, prior chemotherapy and Down syndrome.

Market Dynamics

Recently in 2016, Global Leukaemia Therapeutics Market was valued at nearly USD 9.44 billion and is expected to grow at a CAGR of 4.1% between 2017 and 2022, accounting to market worth USD 11.97 billion by end of 2022.

Global Leukaemia Therapeutics market is majorly driven by the growing number of incidences of target disease across the globe. Also, development of novel agents, advancements in technology and combination therapy with reduced side effects and better survival conditions are some other key factors that drives the Leukaemia Therapeutics Market.

However, the high cost of combination therapies and clinical trials coupled with post-treatment complications, adverse events and side effects are the major constraints that limit the growth of the market.Nevertheless, initiatives like increasing focus on healthcare and personalized medicine along with huge govt. investment & R&D in anti-leukaemia therapeutics research are sure short to boost the market growth in the near future.

Market SegmentationGlobal Leukaemia Therapeutics Market can be segmented as follows :Segmentation by TypeChronic leukaemiaChronic myeloid leukaemiaChronic lymphatic leukaemiaAcute leukaemiaAcute myeloid leukaemiaAcute lymphatic leukaemia

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Segmentation by TherapyBiological TherapyRadiation therapyChemotherapyTargeted therapy

Regional/Geographic AnalysisEurope, North America, Latin America, Asia-Pacific, Middle East & Africa are key market segments of global Leukaemia Therapeutics. North America is the leading region and is anticipated to remain one in the near future, over the forecast period. Demand for leukaemia therapeutics was highest in North America especially in the U.S attributing to increasing geriatric population and increased number of cases. While, Asia Pacific region along with Middle East, Africa and Latin America is expected to grow at moderate pace.

Key Players

The key players in global leukaemia therapeutics market includeF. Hoffmann-La Roche Ltd., Bristol-Myers Squibb, Amgen, Pfizer, Teva Pharmaceuticals, Novartis International AG., GlaxoSmithKline plc., Genzyme Corporation, AbbVie Inc. and others.

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Originally posted here:
Leukaemia Therapeutics Market is expected to grow at a CAGR of 4.1% between 2017 and 2022 - WaterCloud News

Akin to cystic fibrosis (CF), scientists understand that certain mutations contribute to the development of the fatal neurological disorder amyotrophic lateral sclerosis (ALS). And much like CF drugmaker Vertex, a small Cambridge, Massachusetts-based biotech is forging a path to engineering precision therapies to treat the disease that killed visionary physicist Stephen Hawking.

The company, christened QurAlis, now has $42 million in its coffers with three preclinical programs and 5 employees (including senior management) to combat an illness that has long flummoxed researchers, resulting in a couple of approved therapies over the course of decades, neither of which attacks the underlying cause of the rare progressive condition that attacks nerve cells located in the brain and spinal cord responsible for controlling voluntary muscles.

ALS garnered international attention when New York Yankees player Lou Gehrig abruptly retired from baseball in 1939, after being diagnosed with the disease. In 2014, ALS returned to the spotlight with the Ice Bucket Challenge, which involved people pouring ice-cold water over their heads, posting a video on social media, and donating funds for research on the condition.

QurAlis chief Kasper Roet, whose interest in ALS was piqued while he was working on his PhD at the Netherlands Institute for Neuroscience focusing on a treatment for spinal cord paralysis and moonlighting at the Netherlands Brain Bank as an ad-hoc autopsy team coordinator, saw an opportunity to combat ALS when Harvard scientists Kevin Eggan and Clifford Woolf pioneered some new stem cell technology.

Essentially, they found a way to take skin cells from a patient, turn them into stem cells, and turn those into the nerve cells that are degenerating. Thats the missing link, Roet said. So now we can finally use patients own cells to both do target discovery and develop potential therapeutics.

So Roet packed up his things and shifted base to Boston to learn more, with plans to head back to Europe to start a company. He never left. QurAlis was born in 2016, working out of a co-working space called LabCentral after winning a spot via an Amgen-sponsored innovation competition. The company was carved out of a collaboration with Eggans startup Q-State Biosciences, which developed laser technology to examine cell behavior examining how a neuron fires was imperative in the drug discovery process for ALS.

QurAlis, which counts Vertexs founding scientist Manuel Navia as an advisor, now has three preclinical programs. The furthest along is a therapy designed to target a specific potassium channel that is implicated in certain ALS patients the plan is to take that small molecule into the clinic next year, Roet said.

It has become really clear that if you understand why a specific tumor is developing you can develop very specific targeted therapies, he explained in an interview drawing a parallel between ALS and oncology. Thats exactly the same strategy that we are following for ALS. The genetics have shown that over 25 genes are causing the (ALS) mutations. Some of them work together, some of them are very dominant and work alone what we are doing is trying to get those specific proteins that are tied to very specific ALS populations, where we know that that specific target plays a very important and crucial role in the development of the disease.

In 2018, QurAlis scored seed funding from Amgen, Alexandria, and MP Healthcare Venture Management. The Series A injection was led by LS Polaris Innovation Fund, lead seed investor Mission BioCapital, INKEF Capital and the Dementia Discovery Fund, and co-led by Droia Ventures. Additional new investors include Mitsui Global Investment and Dolby Family Ventures, and existing investors Amgen Ventures, MP Healthcare Venture Management, and Sanford Biosciences also chipped in.

Roet is not sure how long these funds will last, particularly given the uncertainty of the coronavirus pandemic. But some of the capital will be used in hiring, given that the QurAlis team is comprised of a mere five people, including Roet.

Weve been very productive, he said. But we can definitely use some extra hands.

Read more:
Precision therapy approach secures small biotech $42M haul to combat disease that inspired the Ice Bucket Challenge - Endpoints News

XconomyBoston

Few drugs exist that treat amyotrophic lateral sclerosis, a progressive disease that kills the nerve cells that allow patients to initiate and control muscle movement.

QurAlis, a Cambridge, MA-based startup, has an ambitious plan to develop a number of precision therapies for the disease based on forms of the condition identified by genetic mutation or a biomarker that CEO Kasper Roet (pictured) hopes to could one day, in combination, help most ALS patients.

Now the company has raised $42 million from investors in the US, Europe, and Japanmoney that will fund a move from the LabCentral incubator in Kendall Square to its own office, more than double the companys headcount by years end, and get at least one of its programs into human testing sometime next year.

The company is leveraging stem cell research from company co-founders Kevin Eggan and Clifford Woolf, Harvard University professors whoby harvesting normal skin cells from ALS patients and turning them into cells such as the motor neurons that damages as the disease progresseshave created models with the same DNA and gene mutations as those patients in an effort to identify new therapeutics for known ALS genes.

Mutations in more than 25 human genes have been implicated in ALS, the company says, and its strategy is to systematically investigate treatments targeting specific disease-causing mechanisms in patient subgroups. Some of those genes are also believed to cause frontotemporal dementia, a common cause of dementia that QurAlis also plans to treat.

One program QurAlis is advancing is intended for patients whose neurons are damaged and killed by the overactivation of certain receptors for glutamate, a key neurotransmitter, in a process known as excitotoxicity.

The company is also working on a treatment intended to return the autophagy process, through which cells recycle unwanted or damage components, to normal functioning. To do so, QurAlis is looking to target the enzyme TBK1, which plays a key role.

Roet, in an interview, said the company views its strategy as analogous to that pursued by Bostons Vertex Pharmaceuticals (NASDAQ: VRTX), which has developed multiple drugs for forms of cystic fibrosis (CF) caused by certain mutations, and late last year received approval for a combination of those drugs for about 90 percent of all CF patients.

We have identified ALS as a disease that we think we understand now, at least for specific subgroups of patients, he said. We understand what is driving the disease and we are able to develop very specific therapies for those patients.

Eggan, Woolf, Roet, and Jonathan Fleming launched QurAlis just over two years ago with seed funding from investors including MP Healthcare Venture Management, the investment arm of Mitsubishi Tanabe Pharma; the investment arm of Amgen (NASDAQ: AMGN); and Alexandria Venture Investments. Mitsubishi Tanabe markets edaravone (Radicava), one of four FDA-approved treatments for ALS. The FDAs 2017 nod for the drug made it the only ALS therapy OKd in the past 20 years.

The Cambridge, MA-based company said the new capital, a Series A financing round, brings the total it has raised to $50.5 million. The investment was led by LS Polaris Innovation Fund, Mission BioCapital, Dutch firm Inkef Capital, and the Dementia Discovery Fund. New investors including Droia Ventures, which operates from Luxembourg and Belgium, Mitsui Global Investment, and Dolby Family Ventures also participated, as did earlier investors including Amgen, MP Healthcare, and Sanford Biosciences.

As part of the deal, LS Polariss Amy Schulman, Inkef Capitals Roel Bulthuis, Dementia Discovery Funds Jonathan Behr, and Droia Ventures Luc Dochez join Mission BioCapitals Johannes Fruehauf on the QurAlis board.

Earlier this year some of the same investors, including Amgen and Dolby Family Ventures, backed a Series A financing for EnClear Therapies, a spinout of QurAlis. That company raised $10 million to advance the development of a dialysis-like medical device designed to filter out harmful proteins in the cerebral spinal fluid of patients with neurodegenerative diseases.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

Excerpt from:
QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing - Xconomy

DetailsCategory: AntibodiesPublished on Saturday, 02 May 2020 12:42Hits: 149

- Innovative, fixed-dose formulation significantly reduces treatment time from hours to minutes and demonstrates consistent efficacy with a reduction in administration-related reactions compared to DARZALEX (daratumumab) for approved indications

- DARZALEX FASPRO is the only subcutaneous CD38-directed antibody approved in the treatment of multiple myeloma

HORSHAM, PA, USA I May 1, 2020 I The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) approved DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a new subcutaneous formulation of daratumumab. DARZALEX FASPRO is approved in four regimens across five indications in multiple myeloma patients, including newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients.As a fixed-dose formulation, DARZALEX FASPRO can be administered over approximately three to five minutes, significantly less time than DARZALEX,which is given intravenously over hours. In the Phase 3 COLUMBA study supporting the approval, DARZALEX FASPRO demonstrated a consistent overall response rate (ORR) and pharmacokinetics and a similar safety profile compared with intravenous DARZALEX in patients with relapsed or refractory multiple myeloma. In addition, there was a nearly two-thirds reduction in systemic administration-related reactions (ARRs) for DARZALEX FASPRO compared to intravenous DARZALEX (13 percent vs. 34 percent, respectively).

"This approval exemplifies Janssen's mission and commitment to bringing together passion, science and ingenuity to advance novel solutions for patients," said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC. "We are excited about the potential of this meaningful innovation in transforming the treatment experience for patients with multiple myeloma where DARZALEX FASPRO can be administered in approximately three to five minutes, significantly less time than intravenous DARZALEX, which is given over hours. Based on its favorable profile, we are accelerating the development of DARZALEX FASPRO and evaluating its potential in multiple ongoing studies."

Click to Tweet: #NEWS: #FDA approves subcutaneous CD38-directed antibody for the treatment of multiple #myeloma. See here for more details: https://bit.ly/2VozhzY

The approval is based on data from the Phase 3 COLUMBA (MMY3012)and Phase 2 PLEIADES (MMY2040) studies.1,2 In the COLUMBA study, the ORR was non-inferior for patients taking DARZALEX FASPROas monotherapycompared to those taking intravenous DARZALEXas monotherapy (41 percent vs. 37 percent, respectively). In addition, there were fewer systemic ARRs with DARZALEX FASPRO versus intravenous DARZALEX (13 percent vs. 34 percent, respectively). In a pooled safety population of 490 patients who received DARZALEXFASPRO as monotherapy or in combination, the ARR rate wFas 11 percent. The safety profiles of intravenous DARZALEX and DARZALEX FASPRO were otherwise similar.1 Additionally, in the Phase 2 PLEIADES study evaluating the efficacy and safety of DARZALEX FASPRO in combination therapies, objective responses were demonstrated in combination with bortezomib, melphalan and prednisone (D-VMP) in newly diagnosed transplant ineligible patients. In addition, objective responses were demonstrated in combination with lenalidomide and dexamethasone (D-Rd) in relapsed or refractory patients who received one prior line of therapy.2

"The Multiple Myeloma Research Foundation shares a common goal with Janssen in advancing treatments for multiple myeloma and addressing the unmet needs of this patient community," said Paul Giusti, President and CEO of the Multiple Myeloma Research Foundation (MMRF). "The approval of DARZALEXFASPRO marks an important milestone which will help make a positive difference in the lives of patients who depend on this effective therapy."

Click to Tweet: .@theMMRF talks about advancing treatments for multiple #myeloma and addressing patient needs with latest #FDA approval. Read more here: https://bit.ly/2VozhzY

"Since the approval of daratumumab, a robust body of evidence has established its use as a treatment for multiple myeloma in both the frontline and relapsed and refractory settings," said Saad Z. Usmani, M.D., Division Chief of Plasma Cell Disorders, Levine Cancer Institute. "With DARZALEX FASPRO there may be fewer administration-related reactions compared to intravenous DARZALEX, providing an additional treatment option that may help patients, oncologists and nursing staff."

DARZALEX FASPROis co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) [Halozyme'sENHANZEdrug delivery technology].DARZALEX FASPRO will be available to patients and physicians as soon as the week of May 11, 2020. The intravenous DARZALEX formulation will also remain available as an option for patients and their physicians.

DARZALEX FASPROis approved in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant, in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy, as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.

The U.S. FDA approval of DARZALEX FASPRO marks the first approval for this innovative subcutaneous formulation globally, and Janssen continues to work with health authorities around the world in an effort to bring this new treatment option to patients living with multiple myeloma.

Access to DARZALEX FASPRO (daratumumab and hyaluronidase-fihj)Janssen offers comprehensive access and support information, resources and services to assist U.S. patients in gaining access to DARZALEX FASPROthrough the Janssen CarePath Program. Through the program, eligible commercial patients pay no more than $5 per injection, regardless of individual income level. Information on the enrollment process is available online atwww.CarePathSavingsProgram.com/DARZALEX.

For more information, healthcare providers or patients can contact: 1-844-55DARZA (1-844-553-2792). Information will also be available atwww.DARZALEX.com. Dedicated case coordinators are available to work with both healthcare providers and patients.

About the COLUMBA Study 1The randomized, open-label, multicenter Phase 3 COLUMBA study (MMY3012) included 522 patients (median age of 67 years) with multiple myeloma who had received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD), or whose disease was refractory to both a PI and an IMiD. In the arm that received DARZALEX FASPRO(n=263), patients received a fixed dose of DARZALEX FASPRO1,800 milligrams (mg), co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) 2,000 Units per milliliter (U/mL), subcutaneously weekly for Cycles 1 2, every two weeks for Cycles 3 6 and every four weeks for Cycle 7 and thereafter. In the intravenous DARZALEXarm (n=259), patients received DARZALEXfor intravenous infusion 16 milligrams per kilogram (mg/kg) weekly for Cycles 1 2, every two weeks for Cycles 3 6 and every four weeks for Cycle 7 and thereafter. Each cycle was 28 days. In the arm that received DARZALEX FASPRO, itwas given in a fixed volume of 15 mL over three to five minutes; the median injection time was five minutes. In the arm that received theintravenous administration, the median durations of the first, second and subsequent intravenous DARZALEXinfusions were 7.0, 4.3 and 3.4 hours, respectively.Patients in both arms continued treatment until disease progression or unacceptable toxicity.

About the PLEIADES Study 2The non-randomized, open-label, parallel assignment Phase 2 PLEIADES study (MMY2040) included more than 240 adults with multiple myeloma, including 67 patients with newly diagnosed multiple myeloma who were treated with 1,800 mg of DARZALEX FASPROin combination with bortezomib, melphalan, and prednisone (D-VMP) and 65 patients with relapsed or refractory disease who were treated with 1,800 mg of DARZALEX FASPROplus lenalidomide and dexamethasone (D-Rd). The primary endpoint for the D-VMP and D-Rd cohorts was overall response rate.

About DARZALEXand DARZALEX FASPROJanssen is committed to exploring the potential of DARZALEX (daratumumab) for patients with multiple myeloma across the spectrum of the disease. DARZALEX has been approved in seven indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.

DARZALEX has become a backbone therapy in the treatment of multiple myeloma, having been used in the treatment of more than 58,000 patients in the U.S. alone since its U.S. FDA approval in 2015. DARZALEX is the first CD38-directed antibody approved globally to treat multiple myeloma and in 2020, DARZALEX FASPRO(daratumumab and hyaluronidase human-fihj) follows as the only subcutaneous CD38-directed antibody approved to treat patients with multiple myeloma.2

CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.4 DARZALEX binds to CD38 and inhibits tumor cell growth causing myeloma cell death.5 DARZALEX may also have an effect on normal cells.3 Data across seven Phase 3 clinical trials, in both the frontline and relapsed settings, have shown that DARZALEX-based regimens resulted in significant improvement in progression-free survival and/or overall survival. 4,5,6,7,8,9,10,11 Additional studies are underway to assess the efficacy and safety of DARZALEXFASPRO in the treatment of other malignant and pre-malignant hematologic diseases in which CD38 is expressed, including smoldering myeloma and in amyloidosis.12,13

Key DARZALEX Milestones:

Please see full Prescribing Information at http://www.DARZALEX.com.

About Multiple MyelomaMultiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.21,22When damaged, these plasma cells rapidly spread and replace normal cells with tumors in the bone marrow. In 2020, it is estimated that 32,270 people will be diagnosed and 12,830 will die from the disease in the U.S.24 While some patients with multiple myeloma have no symptoms, most patients are diagnosed due to symptoms, which can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.23

Please see full Prescribing Information at http://www.DARZALEX.com.

About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at http://www.janssen.com. Follow us at http://www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

ENHANZEis a registered trademark of Halozyme.

1Mateos M-V et al. Efficacy and Safety of the Randomized, Open-Label, Non-inferiority, Phase 3 Study of Subcutaneous (SC) Versus Intravenous (IV) Daratumumab (DARA) Administration in Patients (pts) With Relapsed or Refractory Multiple Myeloma (RRMM): COLUMBA. 2019 American Society of Clinical Oncology Annual Meeting. June 2019.

2Janssen Research & Development, LLC. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 [cited July 5, 2019]. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565. Identifier: NCT03412565.

32020Fedele G et al. CD38 Ligation in Peripheral Blood Mononuclear Cells of Myeloma Patients Induces Release of Protumorigenic IL-6 and Impaired Secretion of IFN Cytokines and Proliferation. Mediators Inflamm. 2013;564687.

4Janssen Research & Development, LLC. A Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02076009?term=mmy3003&rank=1 Identifier: NCT02136134 .

5Janssen Research & Development, LLC. Addition of Daratumumab to Combination of Bortezomib and Dexamethasone in Participants With Relapsed or Refractory Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02136134?term=mmy3004&rank=1 Identifier: NCT02076009.

6Janssen Research & Development, LLC. A Study to Evaluate Daratumumab in Transplant Eligible Participants With Previously Untreated Multiple Myeloma (Cassiopeia). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02541383?term=mmy3006 Identifier: NCT02541383.

7Janssen Research & Development, LLC. A Study of Combination of Daratumumab and Velcade (Bortezomib) Melphalan-Prednisone (DVMP) Compared to Velcade Melphalan-Prednisone (VMP) in Participants With Previously Untreated Multiple Myeloma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02195479?term=mmy3007&rank=1 Identifier: NCT02195479.

8Janssen Research & Development, LLC. Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Participants With Previously Untreated Multiple Myeloma. In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT02252172?term=mmy3008&rank=1 Identifier: NCT02252172.

9Janssen Research & Development, LLC. A Study of VELCADE (Bortezomib) Melphalan-Prednisone (VMP) Compared to Daratumumab in Combination With VMP (D-VMP), in Participants With Previously Untreated Multiple Myeloma Who Are Ineligible for High-Dose Therapy (Asia Pacific Region). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24]. Available at: https://clinicaltrials.gov/ct2/show/NCT03217812?term=MMY3011&rank=1 Identifier: NCT03217812.

10European Myeloma Network. Compare Progression Free Survival Btw Daratumumab/Pomalidomide/Dexamethasone vs Pomalidomide/Dexamethasone (EMN14). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24] Available at: https://clinicaltrials.gov/ct2/show/NCT03180736?term=MMY3013&rank=2 Identifier: NCT03180736

11Amgen. Study of Carfilzomib, Daratumumab and Dexamethasone for Patients With Relapsed and/or Refractory Multiple Myeloma. (CANDOR). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 July 24] Available at: https://clinicaltrials.gov/ct2/show/NCT03158688?term=NCT03158688&rank=1 Identifier: NCT03158688.

12Janssen Research & Development, LLC. A Study to Evaluate 3 Dose Schedules of Daratumumab in Participants With Smoldering Multiple Myeloma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 March 19]. Available at: https://clinicaltrials.gov/ct2/show/NCT02316106?term=smm2001&rank=1 Identifier: NCT02316106.

13Janssen Research & Development, LLC. An Efficacy and Safety Proof of Concept Study of Daratumumab in Relapsed/Refractory Mantle Cell Lymphoma, Diffuse Large B-Cell Lymphoma, and Follicular Lymphoma In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000-[cited 2018 March 19]. Available at: https://clinicaltrials.gov/ct2/show/NCT02413489?term=lym2001&rank=1 Identifier: NCT02413489

14Janssen Biotech, Inc. "Janssen Biotech Announces Global License and Development Agreement for Investigational Anti-Cancer Agent Daratumumab." Issued August 30, 2012.

15Janssen Biotech, Inc. "DARZALEX (daratumumab) Approved by U.S. FDA: First Human Anti-CD38 Monoclonal Antibody Available for the Treatment of Multiple Myeloma." Issued November 16, 2015.

16Janssen Biotech, Inc. "DARZALEX (daratumumab) Approved by U.S. FDA in Combination with Two Standard of Care Regimens for the Treatment of Patients with Multiple Myeloma Who Have Received At Least One Prior Therapy." Issued November 21, 2016.

17Janssen Biotech, Inc. "DARZALEX (daratumumab) Approved by the U.S. FDA in Combination with Pomalidomide and Dexamethasone for Patients with Multiple Myeloma Who Have Received At Least Two Prior Therapies." Issued June 16, 2017.

18Janssen Biotech, Inc. "Janssen Announces DARZALEX (daratumumab) U.S. FDA Approval for Newly Diagnosed Patients with Multiple Myeloma who are Transplant Ineligible." Issued May 7, 2018.

19Janssen Biotech, Inc. "Janssen Announces U.S. FDA Approval of DARZALEX (daratumumab) in Combination with Lenalidomide and Dexamethasone for Newly Diagnosed Patients with Multiple Myeloma Who Are Transplant Ineligible." Issued June 27, 2019.

20Janssen Biotech, Inc. "Janssen Announces U.S. FDA Approval of DARZALEX (daratumumab) Combination Regimen for Newly Diagnosed, Transplant-Eligible Patients with Multiple Myeloma." Issued September 26, 2019.

21Kumar, SK et al. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012 Jan; 26(1):149-57.

22American Cancer Society. "What Is Multiple Myeloma?" Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed June 2019.

23American Cancer Society. "Key Statistics About Multiple Myeloma." Available at: https://www.cancer.org/cancer/multiple-myeloma/about/key-statistics.html. Accessed January 2020.

SOURCE: Janssen

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US Food and Drug Administration Approves DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a New Subcutaneous Formulation of Daratumumab in the...

By Guy Adams and John Naish Apr 25, 2020

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London - Modern science has scarcely presented a more crucial goal, offering untold riches and perhaps even a Nobel prize to the victor, as well as the chance to return the world to normal.

Yet the search for a cure for coronavirus is also uniquely perilous thanks to the dangers inherent in rushing new medical products to market.

Across the globe, thousands of researchers employed by governments, laboratories and drug firms are working flat-out to crack this all-important riddle.

As are endless armchair experts, whose numbers now evidently include Donald Trump.

Yesterday, the worlds most powerful man used a press conference to propose various highly unorthodox new treatments for coronavirus, including injecting disinfectant into the body and blasting patients with ultraviolet light.

Back in the real world, researchers are working tirelessly to combat the virus.

But while bullish claims have been made for a host of possible treatments, the truth is that scientists have little idea which ones might end up being of use.

Four months into the pandemic, we have had millions of people infected but no data yet to show that any particular treatment is especially effective, says Professor Babak Javid, the Principal Investigator at Tsinghua University school of medicine in Beijing, and a consultant in infectious diseases at Cambridge University Hospitals.

Nonetheless, here are some of the most likely candidates.

In mid-March, the American President used Twitter to amplify reports that Covid-19 patients could be successfully treated via a combination of the malaria drug hydroxychloroquine and a common antibiotic, Azithromycin.

In a later press conference, the President insisted that common sense qualified him to make such a claim, urging Americans to take it and asking them: What have you got to lose?

Well, quite a lot, as it turned out. The drugs have a catalogue of nasty side-effects, including an increased risk of cardiac arrest, skin blistering, hearing loss and an inability to move the eyes.

A study this week of 368 male coronavirus patients found that 28 per cent of those treated via hydroxychloroquine alone and 22 per cent who received both drugs (the malaria treatment and azithromycin) in U.S. hospitals died. This compares to a death rate of just 11 per cent for patients who received standard care without either drug.

Hope and hype about the drug Remdesivir have set Californian biotech firm Gileads share price soaring in recent weeks.

The medicine, which is administered via a drip, was originally developed to fight the ebola virus and works by interfering with its genetic makeup.

Although other drugs were soon found to be more effective at treating ebola, subsequent tests suggested Remdesivir had some effect on respiratory viruses such as Sars and Mers. It also appears to be relatively safe.

Gilead has so far given Remdesivir to 1,700 coronavirus patients, with dramatic effects reported in some cases. Two major studies, in the U.S. and Europe, are currently underway.

Sadly, a full-scale clinical trial in China, which has been running for longer, found no evidence it improved the fate of hospital patients, according to documents accidentally leaked this week by the World Health Organisation.

Gilead responded that these leaks contained inappropriate characterisations and insisted their findings were inconclusive.

One reason that the Chinese trials outcome was so disappointing could be that Remdesivir is likely to work best if administered early.

With Covid-19, the virus mostly replicates soon after infection, says Professor Javid. If you give the anti-viral later in the course of an illness, when a patient is hospitalised, there is not much virus left for the drug to attack.

The golden goose for pharmaceutical firms is to discover a new antibody treatment that can attach to individual particles of coronavirus and stop them wreaking havoc.

Two US drug companies, Amgen and Adaptive, recently announced a partnership to study recovered Covid-19 patients in an effort to identify and manufacture crucial virus-killing antibodies.

Another firm, Regeneron, has been conducting a study using the viruss genetic material in mice. Dozens of other companies are pursuing similar projects.

Although an antibody strategy was used against ebola, most new drugs take more than five years to develop, thanks among other things to rigorous testing designed to ensure they do not have dangerous side-effects.

This fashionable, if highly experimental, field involves harvesting blood from people who have recently recovered from coronavirus and donating it to patients who are still suffering from the disease.

Because blood plasma contains antibodies that have learned how to detect and destroy the virus, the theory is that a transfusion will kick-start the recipients ability to fight it.

Its quite easy to harvest plasma from surviving patients, says Professor Javid. It was done even during the Spanish Flu epidemic of 1918-19.

About 600 patients in America have already received this treatment. The NHS is also said to be preparing to carry out an extensive trial in the UK.

However, some experts have pointed out that many Covid-19 victims die because of an overactive immune response to the virus, causing inflammation of lungs and other vital organs. These experts worry that boosting a patients immunity via plasma therapy could actually worsen their condition.

Another problem with plasma therapy is the old-fashioned issue of supply and demand. A limited number of recovered patients are prepared to give blood, and only a finite amount can be taken from them.

However, technology now exists to manufacture synthetic antibodies using the blood of Covid-19 survivors.

Such monoclonal antibodies have become the standard treatment for ebola. Several laboratories have identified monoclonal antibodies that can apparently inhibit coronavirus in test-tubes.

Professor Javid warns, however: No one has yet worked out which of the promising monoclonal antibodies work best for Covid-19, or what might be the best way to use them in combination. This is why they are not in production yet for Covid-19.

Contracting HIV was once a virtual death sentence, but after decades of research, its now mostly a highly manageable condition thanks to a raft of well-tolerated drugs.

Indeed, many HIV drugs are now being touted as possible treatments for coronavirus, including Lopinavir and Ritonavir.

These are being trialled on coronavirus patients in studies at the Universities of Oxford and Nebraska.

But so far there is little evidence of them working outside of a laboratory setting.

The so-called master cells that develop into blood, brain, bones and organs have been touted as the basis for cures for cancer, heart disease and arthritis for years.

Little wonder, then, that the pioneering field of stem cell therapy is now being targeted at coronavirus. Among firms exploring this modish area are Mesoblast, whose boffins are testing bone-marrow cells to establish whether they can help patients develop immunity to the virus. In Wuhan, meanwhile, doctor Dongcheng Wu last month claimed hed treated nine patients by injecting umbilical stem cells. He said they all made a complete recovery within days. The success has not yet been convincingly replicated, however. Stem cell treatments are often risky, too when trialled on Parkinsons, they caused brain tumours.

Around half the patients who die with coronavirus suffer a so-called cytokine storm, in which their immune system goes into overdrive, causing, among other things, acute lung inflammation that stops them from taking on enough oxygen. With this in mind, many products designed to combat inflammation are now being trialled on Covid patients.

They include Tocilizumab, used to treat rheumatoid arthritis, and Dexamethasone, a steroid used against asthma.

The World Health Organisations Solidarity trial is meanwhile testing interferon-beta, which is used to treat multiple sclerosis.

Professor Javid believes immune drugs should be accompanied by effective antiviral treatment: We know from treating flu patients suffering cytokine storms that if you dampen their immune response without also giving them an antiviral drug, it can reduce the patients virus-fighting defences and allow the virus to run wild.

These are the cholesterol-lowering drugs prescribed to millions at risk of heart disease. Now scientists wonder if statins should be given to patients with severe Covid-19 symptoms, for two reasons.

Harvard University investigators recommend their use because they have anti-inflammatory powers.

Scientists added last month in the journal Drug Development and Research that statins have also shown an ability to moderate the immune system and thus protect patients lungs from cytokine storm damage.

Professor Jon Cohen, emeritus professor of infectious diseases at Brighton and Sussex Medical School, argues that while statins have shown potential in test-tube trials, in living patients they have only really shown benefits for the cardiovascular system.

In normal circumstances viruses develop a key that enables them to pick a cells locks and break in, hijacking the cells machinery to make more copies of the virus. Peptide inhibitors stick to this key, rendering it unable to do its job.

We already know that Covid-19 invades human cells through a protein receptor, ACE2.

The big question, therefore, is: what might block the coronavirus attaching to the ACE2 receptor?

Chemists at Illinois University used high-powered computers to identify the amino-acid chemicals in the ACE2 receptor that the virus targets. They then constructed a drug with amino-acids that should stick to the viruss key, rendering it useless.

But as the scientists acknowledge in journal ACS Nano this month, they have tested their chemical in computer simulations not in the lab and certainly not on humans. A lot of hurdles lie ahead.

Ultimately, most scientists agree that coronavirus prevention a vaccine is better than any treatment or cure.

Thats why governments are throwing the proverbial kitchen sink at efforts to create one, with more than 140 projects currently running in parallel all over the world.

Everyone expects a vaccine to appear in the end. But much hinges on how quickly that happens.

Thats why there was so much excitement this week when it emerged that scientists at Oxford University have already begun to conduct their first human vaccine tests.

Other clinical trials in humans are already underway in China.

Nevertheless, Professor Chris Whitty, Englands Chief Medical Officer, says the chance of a vaccine becoming available in the next calendar year are incredibly small.

Why? Because we can move only as quickly as extreme caution will allow, says inoculation expert Dr Kai Hu of Imperial College. He reports Imperials lab has already created a harmless pseudo-virus that carries a coronavirus protein. But, he stresses: Safety is our number one priority. We dont yet know how toxic the vaccine would be to humans.

Given Covid-19s devastating infectiousness, the world will have to keep its fingers crossed that the dash for an effective jab proves a success rather than inadvertently plunging humanity into further peril.

Three members of the same family are taking part in a groundbreaking coronavirus vaccine trial. Mum Katie, dad Tony and daughter Rhiannon Vinney are among more than 1,000 participants taking part in the Oxford University trials.

Teaching assistant Katie, 46, saw the plea for healthy volunteers and urged her husband, 53, who runs two pubs, and their 18-year-old student daughter to sign up with her.

The mum-of-four, from Oxford, said she was not worried about health risks from taking part, because she believes the team have done everything they could to make it safe.

She added: I just want to help so life can return to normal. You have to live in a cave not to know somebody who is affected by this. I really do hope this is the cure.

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Will the race for a Covid-19 cure end in triumph or tragedy? - IOL

The "Orphan Drugs Market Global Report 2020-30" report has been added to ResearchAndMarkets.com's offering.

The global orphan drugs market was worth $132.61 billion in 2019. North America is expected to be the largest region during the period 2015-2023. Major players in the market are Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche, Amgen, Biogen, Bayer, Novartis, GlaxoSmithKline, Johnson & Johnson and AbbVie.

This report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. It places the market within the context of the wider orphan drugs market, and compares it with other markets.

The rising prevalence of rare diseases is a key factor driving the growth of the orphan drugs market.

Orphan diseases or rare diseases occur rarely among the people (i.e. 7 out of 10,000). However, globally, the prevalence of rare diseases is increasing in recent years. In 2017, there were 7,000 identified rare diseases, including hemophilia, Gaucher disease, Hunter syndrome and many types of rare cancer. Some cases of aplastic anemia, caused by damage to stem cells in the bone marrow that are diagnosed in around 500 to 1,000 individuals in the USA each year, are inherited. Thus, the rising prevalence of rare diseases is driving the growth of the orphan drugs market.

Lack of supportive government policies hinders the orphan drugs market.

Due to the lack of relevant policies for orphan drug, certain drugs do not receive any special recognition or priorities for approval by regulatory authority. Medgenome Labs Ltd., global research partner in accelerating insights into complex genetic diseases, pointed out that companies manufacturing orphan drugs frequently drop out in foreign markets due to a lack of government funding. For example, orphan medical products (OMPs) in India, due to lack of proper regulations and clear guidelines, do not obtain tax cuts or exemptions from customs duties. Therefore, lack of supportive government policies limits the growth of the orphan drugs market.

Approval of biological orphan drugs for multiple indication act as a key trend driving the growth of the orphan drugs market.

The biological drugs are used for treating rare diseases such as cancer with fewer side effects that have a high prevalence rate in the developed world. For Instance, in 2018, in order to launch the company's biological orphan drug development program Cardax, Inc. announced that it has been engaged with biological orphan drug expert Frederick D. Sancilio, Ph.D. For the development of commercial products, the companies are focused on obtaining biological orphan drugs to increase their revenue.

In November 2019, Bristol-Myers Squibb, a biopharmaceutical company whose mission is to discover, develop, and deliver innovative medicines, acquired Celgene for an undisclosed amount. Through this acquisition, Celgene shareholders received for each share, 1 share of Bristol-Myers Squibb common stock, $50.00 in cash without interest and one tradeable Contingent Value Right (CVR), which will entitle the holder to receive a payment of $9.00 in cash if certain future regulatory milestones are achieved. Celgene, a biopharmaceutical company positioned to address the needs of the patients with serious diseases.

Key Topics Covered

1. Executive Summary

2. Orphan Drugs Market Characteristics

3. Orphan Drugs Market Size and Growth

3.1. Global Orphan Drugs Historic Market, 2015-2019, $ Billion

3.1.1. Drivers Of The Market

3.1.2. Restraints On The Market

3.2. Global Orphan Drugs Forecast Market, 2019-2023F, 2025F, 2030F, $ Billion

3.2.1. Drivers Of The Market

3.2.2. Restraints On the Market

4. Orphan Drugs Market Segmentation

4.1. Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Orphan Drugs Market Regional and Country Analysis

5.1. Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Story continues

5.2. Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/vm9iey

View source version on businesswire.com: https://www.businesswire.com/news/home/20200417005515/en/

Contacts

ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.com

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Orphan Drugs Market Insights, 2020: Approval of Biological Orphan Drugs for Multiple Indications is Driving Market Growth - ResearchAndMarkets.com -...

I'm just going to come out and say it: Everyone has been complimenting my skin lately. My co-workers, random peopleat the grocery store, my friends and familyeveryone. While I've never dealt with any major skin woes like acne, I still never considered myself to be someone with particularly good skin (whatever that means). My skin has always been on thedry side, and like many women of color, I've dealt with my fair share of stubborn hyperpigmentation. The sudden influx of complimentshasn't just been a nice little boost to my newly 30-year-old ego but also a testament to my current skincare routine, which I've tweaked to perfectionover the course of several months.

As a beauty editor, I have access to every product under the sun. But ever since last fall, I felt like my skin had just lost something. When I think back, it makes sense, as there was lot was happening at the time. I had moved, turned 30, gotten engaged, andmade a major professional moveall in a matter of months, and while each of these life developments was exciting and positive, I found myself overwhelmed with stress. I wasn't sleeping well, I wasn't eating properly, my skincare routine had fallen by the wayside, and all of that was showing up on my face. I was getting pimples, my skin tone was blotchy and uneven, and my skin texture was less than smooth.

But then, something started to happen around January: Every time I posted a photo of my mug on Instagram, a sea of adulation would flood into my comments. I started catching glimpses of my makeup-free face and being truly happy with what I saw staring back at me. Maybe it was the newfound self-love I'd been practicing in therapy, or maybe it was my skincare regimen, whichhadadmittedly reached an all-time level of bougie, even for me. Now, I'm at a place where I'llfreely leave the house without makeup on and am genuinely pleased with how healthy and smooth my skin looks. I'm not perfect, by any means, but Iamgenuinely happy, and I have to believe that my fresh, smooth skin has something to do with it. So here it goes: the exact skincare routine thatdelivered smooth, glowing skin in a month's time and continues to do so to this day. Try it out for yourself and let me know what you think.

Klur Gentle Matter ($22)

I thoroughly cleanse my skin at night, so I don't always use a cleanser in the morning. Most days, I find that warm water is enough. When I do feel the need to cleanse in the morning, though,this gel cleanser is the onlyone I'll reach for. It's so gentle and actually adds moisture and nutrients like green tea, dandelion, and olive oil into my skin instead ofjust pulling everything out.

SkinCeuticals C E Ferulic ($166)

Vitamin C is probably the most important component of my skincare routine right now. While there's a lot of debate around L-ascorbic acid and whether or not its potency is actually good for the skin (jury's still out on that one), I find thatmy skin responds really well to it. Vitamin E and ferulic acid round out this formula with extra skin lipid and antioxidant protection. I can alwaystell when I've gotten lazy with my vitamin C regimen because marks from old blemishes will start to deepen, and my skin will lose some of the glow and refinement that earns me an insane amount of compliments.

Bioderma Sensibio Eye Contour Gel ($20)

I'll admit that I didn't take eye cream seriously until about a year ago, and this non-intimidating tube is to thank for that change of heart. The cream inside is lightweight and easy to lightly tap into my eye area. When I'm using eye cream consistently, I notice that any fine lines in the area soften over time, giving me that smooth, even texture I'm always after.

Kiehl's Ultra Facial Cream ($32)

This moisturizer has been an on-again, off-again staple on my vanity for years now. It's unscented, lightweight, and super effective. If I'm feeling extra dry, I'll even add afew small drops of marula oil to give it an extra hit of moisture.

Victoria Beckham by Augustinus Bader Cell Rejuvenating Priming Moisturizer ($145)

This moisturizing primer is basically like a blurring filter for your skin. It has tiny sparkly particles and theproprietary TFC-8 technology found in Augustinus Bader's other famous creams (more on those later). It makes my skin look way smoother, even when I don't layer any makeup on top.

Elta MD UV Clear Broad-Spectrum SPF 46 ($28)

Say it with me: SPF, all the time, no matter what. UV protection is important for so many reasons, but for me, it's all about mitigating hyperpigmentation and making sure any scars or blemishes on my face aren't getting exposed to the sun. This sunscreen by Elta MD is a dermatologist favorite, and it's one of my favorites, too. It doesn't irritate my skin or leave an unsightly white cast.

Farmacy Green Clean Makeup Removing Cleansing Balm ($34)

I'm a makeup wearer, so my nighttime cleansing ritual has always been serious. I need every stitch ofgunk off of my face before I can relax for the evening. This cleansing balm melts even the most stubborn eye makeup with ease. I usually massage it into the rest of my face for about 30 seconds before concentrating on my eyes. After just a few seconds of gentle rubbing, any makeup is melted down to an inky oil that rinses away without leaving any residue behind.

Reflekt Daily Exfoliating Wash ($48)

This exfoliator is said to be gentle enough for daily use, and I've found that to be true for me. Although I've backed off from using it every single day, I still love how clean and soft my face feels after use. The multitasking jojoba beads are small and smooth, so they aren't at all harsh on the skin and also meltdown to impart moisture instead of stripping the skin.

IS Clinical Cleansing Complex ($44)

This slippery cleanser clings to every trace of grime to remove it while also retexturizing. When I want a flat wash at night instead of a gritty, exfoliating one, this is the cleanser for the job. I used to have a serious attitude about paying more than $20 for cleanser (come on, it's literally money down the drain), but this is the one that taught me the power of investing in a high-quality cleanser.

U Beauty Resurfacing Compound ($148)

I've been using the U Beauty Resurfacing Compound pretty consistently since it launched last winter, and I can honestly say that it's ascended to my skincare top five. It's so good. If smooth skin is your goal, you need to try this stuff. It's patent-pending siren capsules are designed to carry active ingredients wherever your skin needs them and bypass the healthy skin cells that don't. That's why you won't experience any redness, irritation, or peeling that typically arises when starting a retinoid. This has definitely been the hero product in my smooth-skin journey.

Moon Juice Beauty Shroom Plumping Jelly Serum ($58)

It was love at first pump with this magical, hyaluronic acid and mushroom-packed elixir. There aren't many products that make a big difference in your skin's texture after just one use, but this one does. Every time I use it, my skin instantly looks plumped and smoother.

IS Clinical Youth Eye Complex ($105)

This eye cream plumps and moisturizes my delicate under-eye skin before bed. It has a little retinol in it, which honestly freaked me out at first, but over time has resulted in major refinement of fine lines.

Augustinus Bader The Rich Cream ($170)

Are you sick of editors telling you how much they love this cream? Well, I'm sorry to tell you that I'm about to do it, too. When I'm running on fumes and can only manage to get my makeup off and slap one product on my face before bed, this is the indispensable one I can't ever skip. Maybe it's the stem cellstimulating TFC-8 technology, maybe it's some sort of sorcery, but all I know is my skin has legitimately changed in texture since I started using this cream. Real talk: It's worth every penny.

Dr. Dennis Gross Clinical Grade Resurfacing Liquid Peel ($95)

I love a good resurfacing peel, but I have to admit that I've calmed way down on the acids. I found my skin becoming more sensitized and reactive, and while I can't say for sure that my nightly resurfacing toners were to blame, I'm way better off since scaling back. Now, once a week, I'll do a pass of this two-step, clinical-grade lactic and glycolic acid peel, and it immediately makes my skin look smooth, bright, and alive. As with any super-potent acid compound, it's a good idea to patch test this one to make sure your skin doesn't have an adverse reaction.

Goldfaden MD Facial Detox ($65)

I love this clean detox mask because it's cooling and tingly on the skin but doesn't dry down so hard that it makes my face feel dry or depleted. Rinsing off the sulfur-infused paste feels like taking the biggest breath of fresh air.

Dr. Dennis Gross Hyaluronic Marine Hydrating Modeling Mask ($48)

If you know me at all, then you already know how obsessed I am with this modeling mask. I firmly believe that I could stay awake for three days straight, not drink any water the whole time, and still look fresh as a daisy after 20 minutes with this goop slopped on my face.

Klorane Smoothing and Relaxing Patches ($24)

Whether I'm prepping for a photo shoot, getting ready for a night out, or just looking to minimize puffy eyes after a couple of glasses of wine, these cornflower eye patches by Klorane get the job done like no other. The soothing hydrogeleye masks actually stay put so I can move around without them slipping off, which is a huge plus.

Pai Rosehip BioRegenerate Oil ($44)

Not only is this fatty acidrich rose-hip oil an ultra-luxe finishing touch to my nighttime routine, but it also helps to get rid of imperfections caused by an imbalance in my skin's pH. I know that using oil to treat breakouts sounds counterintuitive, but this oil does just as much to calm and soothe the skin as it does to moisturize it.

Osea Malibu Blemish Balm ($48)

Speaking of soothing salves, this coolingbalmfeels so good on top of congested skin. Whenever I notice a pimple or that my pores are looking rough, I'll spot-treat with this clean cream and let it penetrate into my skin tocalmany inflammation that's plaguing me. I'll work it in as the first step in my routine whenever I need it, and it really sets the tone for the entire day.

Up next, the 25 best products to keep your skin right and tight well past your 20s.

This article originally appeared on Who What Wear

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My Skin Is Kind of Perfect Right Now Thanks to This Exact 30-Day Routine - Yahoo Lifestyle

GIOSTAR/HEAMGEN has developed and secured patented technology to manufacture lifesaving mature red blood cells from stem cells. The red blood cells are made utilizing a bioreactor that permits the production of mature red blood cells, under strictly controlled conditions, for transfusion therapy and replaces the need for a human blood donor. GIOSTAR/HEAMGEN mature red blood cells are safe and not compromised by inadequate pathogen detection and inactivation of diseases such as hepatitis C, HIV, hepatitis B and syphilis. The red blood cells are O-Negative (Universal Donor) to eliminate incompatibility and allosensitization reactions.

ATLANTA (PRWEB) January 29, 2020

GIOSTAR/HEAMGEN has developed and secured patented technology to manufacture lifesaving mature red blood cells from stem cells. The red blood cells are made utilizing a bioreactor that permits the production of mature red blood cells, under strictly controlled conditions, for transfusion therapy and replaces the need for a human blood donor. GIOSTAR/HEAMGEN mature red blood cells are safe and not compromised by inadequate pathogen detection and inactivation of diseases such as hepatitis C, HIV, hepatitis B and syphilis. The red blood cells are O-Negative (Universal Donor) to eliminate incompatibility and allosensitization reactions. Trauma situations often do not allow for adequate blood typing due to time restrictions, so the GIOSTAR/HEAMGEN red blood cells address that need effectively.

"There are three main problems for blood transfusions," stated Dr. Anand Srivastava, Founder and Chairman of GIOSTAR. "First we have to match the blood type. Second, there's not enough blood available every single time. And third, when we transfer blood from one person to another person, there is always a chance of the transfer of disease."

Watch a feature interview with Dr. Anand Srivastava on The DM Zone with host Dianemarie Collins.

The World Health Organization (WHO) published the first detailed analysis on the global supply and demand for blood in October 2019 and found that 119 out of 195 countries do NOT have enough blood in their blood banks to meet hospital needs. In those nations, which include every country in central, eastern, and western sub-Saharan Africa, Oceania (not including Australasia), and south Asia are missing roughly 102,359,632 units of blood, according to World Health Organization (WHO) goals. While total blood supply around the world was estimated to be around 272 million units, in 2017, demand reached 303 million units. That means the world was lacking 30 million units of blood, and in the 119 countries with insufficient supply, that shortfall reached 100 million units.

The global market opportunity for GIOSTAR/HEAMGEN technology presents not only a profitable and scalable business opportunity but also a significant social and environmental impact. The global market is estimated to be at least $ 85 Billion/year.

GIOSTAR/HEAMGEN has identified early entry global markets to include Military, Trauma, Asia (replace Hepatitis C contaminated blood products), Africa (AIDS contaminated blood), Newborns, Thalassemia patients, Allosensitized sickle cell disease patients. South Sudan was found to have the lowest supply of blood, at 46 units per 100,000 people. In fact, the country's need for blood was deemed 75 times greater than its supply. In India, which had the largest absolute shortage, there was a shortfall of nearly 41 million units, with demand outstripping supply by over 400 percent. Strategic investments are needed in many low-income and middle-income countries to expand national transfusion services and blood management systems. Oncology is a major user of blood transfusion but if countries don't have the capacity to manage the bulk of oncology, it will limit complex surgery options.

GIOSTAR/HEAMGEN has acquired the exclusive license to the patent for the technique for stem cell proliferation from University of California San Diego (UCSD). The founding team of GIOSTAR/HEAMGEN is comprised of the scientists and clinicians who were involved in creating the Intellectual Property at UCSD and has already achieved PROOF OF CONCEPT - the optimized lab scale proliferation of mature red blood cells - at UCSD as part of their research.

GIOSTAR/HEAMGEN is currently looking for strategic partnerships (Contact Doug@DMProductionsLLC.com) to accelerate the development of donor-independent red blood cells manufacturing capabilities and advance the proof of concept work already done (patented) around the manufacture of safe, universal donor, human red blood cells. GIOSTAR/HEAMGEN will also develop a full automated proprietary bioreactor using robotic technology to produce abundant quantities of red blood cells with a goal for cost-effective commercialization of fresh, human, universal donor Red Blood Cells (RBCs).

ABOUT GIOSTAR

Dr. Anand Srivastava is a Chairman and Cofounder of California based Global Institute of Stem Cell Therapy and Research (GIOSTAR) headquartered in San Diego, California, (U.S.A.). The company was formed with the vision to provide stem cell based therapy to aid those suffering from degenerative or genetic diseases around the world such as Parkinson's, Alzheimer's, Autism, Diabetes, Heart Disease, Stroke, Spinal Cord Injuries, Paralysis, Blood Related Diseases, Cancer and Burns. GIOSTAR is a leader in developing most advance stem cell based technology, supported by leading scientists with the pioneering publications in the area of stem cell biology. Company's primary focus is to discover and develop a cure for human diseases with the state of the art unique stem cell based therapies and products. The Regenerative Medicine provides promise for treatments of diseases previously regarded as incurable.

GIOSTAR is world's leading Stem cell research company involved with stem cell research work for over a decade. It is headed by Dr Anand Srivastava, who is a pioneer and a world-renowned authority in the field of Stem Cell Biology, Cancer and Gene therapy. Several governments and organizations including USA, India, China, Turkey, Kuwait, Thailand, Philippines, Bahamas, Saudi Arabia and many others seek his advice and guidance on drafting their strategic and national policy formulations and program directions in the area of stem cell research, development and its regulations. Under his creative leadership, a group of esteemed scientists and clinicians have developed and established Stem Cell Therapy for various types of autoimmune diseases and blood disorders, which are being offered to patients in USA and soon it will be offered on a regular clinical basis to the people around the globe.

For the original version on PRWeb visit: https://www.prweb.com/releases/giostar_announces_medical_breakthrough_in_biotechnology_and_lifesciences_to_manufacture_abundant_safe_red_blood_cells_from_stem_cells/prweb16854975.htm

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GIOSTAR Announces Medical Breakthrough in Biotechnology and Lifesciences To Manufacture Abundant, Safe Red Blood Cells From Stem Cells - Benzinga

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the development of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET), a bone marrow disease associated with high platelet counts and potentially catastrophic vascular complications. Bomedemstat inhibits the enzyme LSD1 (lysine-specific demethylase 1), thus preventing excess platelet and neutrophil production.

"The Fast Track designation by the FDA recognizes the need for novel therapeutics for myeloid diseases and mirrors our own urgency in addressing these devastating conditions," said Hugh Young Rienhoff, Jr. M.D., CEO, Imago Biosciences. "ET is a quiet bone marrow cancer than can linger for years. In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infractions, each having a significant impact on these patients. With only one FDA approved therapy, one that does not increase overall survival, patients are in desperate need of new options. Based on its mechanism and safety data obtained to date, we believe bomedemstat has the promise to be that new treatment."

The FDA grants Fast Track designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track designation can benefit from early and frequent communication with the agency, in addition to a rolling submission of the marketing application, with the objective of getting important new therapies to patients more quickly.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of platelets and cytokines that drive essential thrombocythemia pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent, and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera.

The FDA has also granted Fast Track designation to bomedemstat for the treatment of myelofibrosis, which is currently being studied in an international Phase 2b study. In this study IMG-7289 was effective in reducing spleen volumes and substantially improved symptom scores in a majority of evaluable patients. For more information visit http://www.clinicaltrials.gov (NCT03136185). Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, venture-backed pharmaceutical company whose investors include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital, as well as other corporate and venture investors.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005164/en/

Contacts

Ian StoneCanale Communicationsian@canalecomm.com (619) 849-5388

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Imago Receives Fast Track Designation from U.S. FDA for Bomedemstat for Treatment of Essential Thrombocythemia - Yahoo Finance