Usama Gergis, MD, MBA Professor of Oncology Director, Bone Marrow Transplant and Immune Cellular Therapy Sidney Kimmel Cancer Center Thomas Jefferson University Hospital Philadelphia, PA, reviewed that difference in acute and chronic graft-versus-host disease (GVHD) and the treatment available for each.

Targeted OncologyTM: How would you treat a patient with GvHD in the second line?

GERGIS: If you [have a patient with] second-line acute GVHD, your answer should be ruxolitinib [Jakafi] because its the only drug that has been tried in phase 3 trials. If you get a [case of] chronic GVHD, your answer should be ibrutinib [Imbruvica].

What is the efficacy of peripheral blood stem cells (PBSC) versus bone marrow from unrelated donors in patients with acute and chronic GvHD?

[Results from] a phase 3 study of bone marrow versus stem cells for unrelated donors [showed] the acute GVHD population [cumulative incidence] was the same between both.1 For the chronic population, the bone marrow did better [PBSC 53% vs bone marrow 41%; P = .01]. This was published almost 8 years ago, [and it] was reported almost 10 years ago, but we still use stem cells.

This has not changed practices, and the reasons are, number 1, there was more primary graft failure on the bone marrow than the PBSC, and number 2, its pretty involved to do bone marrow harvest, although I have done it for 15 years, at least a few every month.

The benefit of bone marrow versus PBSCand this benefit was only studied in unrelated donors, not in matched related donorswas seen across all organs affected with chronic GVHD except lungs, [gut, and serosa].2 So, there was no real benefit in the lungs.

Can you explain the difference between acute and chronic GvHD?

Chronic GVHD is more complicated and involved than acute GVHD. In acute, you have the skin, gastrointestinal organs, and the liver [that may be affected]. Thats it. In chronic, all the patients other organs can be affected. The patients weight can be affected. [Chronic GVHD is] more debilitating over a long time and [can] go unrecognized for a while. [If a patient is] experiencing acute GVHD, you see them twice a week, whereas if the patient has chronic GVHD, you probably see them once a month. So you can see a very stark change in your patients within that month if they lose 10% of their body weight and they already lost a lot of weight in the period right after [transplantation], so that can be obvious to you.

[In my institution], we have the GVHD clinic where we [grade the patient based on] studying the degree of fibrosis, how many organs are affected, the patients range of motion, and the degrees in range of motion. We do frequent pulmonary function tests and various [other] testing. By looking at all the affected organs, you reach a grade, and that can be mild, moderate, or severe [chronic GVHD].

How do you treat moderate-to-severe chronic GVHD at initial presentation and in the second line?

First-line treatment for chronic GVHD are steroids. For second line, there are many agents [to consider]. Ive tried most of them. I like photopheresis because its not pharmacological, but its pretty involved. Your patient will need a permanent catheter, and they will need to come to the transplant center twice a week, and you see a response after a long time. It takes an average of 50 photopheresis sessions for a response. But the beauty of photopheresis [is that] you could try it with other agents, so its not mutually exclusive. You could use it with ruxolitinib, ibrutinib, or any other agents.

The answer will be ibrutinib [for chronic GVHD], and thats based on the [results of a] phase 2 clinical trial that treated 42 patients with steroid-refractory chronic GVHD, and the efficacy was 69% [best overall response rate], and 31% complete response rate.3

What do you think of these poll results?

Everybody agrees on giving ibrutinib. When I gave this talk a couple months ago, lenalidomide [Revlimid] was not included in the poll. I added it because [recently], a nice study in Blood came out from the National Institutes of Health where they tried lenalidomide at a small dose, 2 mg, in steroid-refractory chronic GVHD. Its a large trial; I think its about 100 patients. Theyve seen responses that are comparable with ibrutinib....I treated a patient for multiple myeloma; he received a transplant for multiple myeloma, and now, 6 months later, he has chronic GVHD and some clonal plasma cells. So for him, I was comforted to know the results of the lenalidomide trial.

How does ruxolitinib play a role in this setting?

Ruxolitinib was reported in the REACH3 trial [NCT03112603] with very good responses in chronic GVHD.4 I think it probably will get approved for that indication. Looking at this study about 2 years ago, nothing was studied well in this indication, and ibrutinib was approved.

REACH3 was a large trial, almost 300 patients, and everybody was randomized to ruxolitinib 10 mg twice a day versus best available treatment. They looked at everybody about 6 months later for response.

What should physicians keep in mind when treating?

Chronic GvHD is pretty involved. Your patients will need a multidisciplinary approach. You need to pay attention to their bones. In the first 100 days post transplant, the average bone aging is 17 years.

So although were trying to treat acute GVHD, viruses, and prevent relapses, [by putting] your patients on some steroids, you are aging your patients bones by 17 years only in the first 100 days. No matter what you do, give your patients vitamin D, calcium, and Fosamax [alendronate sodium].

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Gergis Explains the Differences Between Acute and Chronic GVHD - Targeted Oncology

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WESTLAKE VILLAGE, Calif., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT), a late-stage biopharmaceutical company focused on developing and commercializing treatments for unmet needs in immune-mediated dermatological diseases and conditions, or immuno-dermatology, today reported financial results for the quarter and year ended December 31, 2020, and provided a business update.

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Arcutis Announces Fourth Quarter and Full Year 2020 Financial Results and Provides Business Update

WALTHAM, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, today announced that Praveen Tipirneni, M.D., president and chief executive officer of Morphic Therapeutic, is scheduled to participate in a panel discussion on the gastrointestinal microbiome at the 41st Annual Cowen Health Care Conference. The panel discussion will take place at 11:10 AM ET on Tuesday, March 2, 2021.

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Morphic Therapeutic to Participate in GI/Microbiome Panel Discussion at the 41st Annual Cowen Health Care Conference

DEER PARK, Ill., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced that company management will be participating in the 10th Annual SVB Leerink Global Healthcare Conference from February 24th through February 26th.

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Eton Pharmaceuticals to Participate in 10th Annual SVB Leerink Global Healthcare Conference

SALT LAKE CITY, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, announced that it will hold its quarterly earnings conference call for the period ending December 30, 2020 with investors and analysts at 4:30 p.m. EST on Tuesday, February 23, 2021. During the call, Paul J. Diaz, president and CEO, and R. Bryan Riggsbee, Chief Financial Officer, will provide a financial overview and business update of Myriad’s performance for the quarter.

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Myriad Genetics to Announce December 2020 Quarterly Financial Results on February 23, 2021

YAVNE, Israel, Feb. 16, 2021 (GLOBE NEWSWIRE) -- MediWound Ltd. (Nasdaq: MDWD), a fully-integrated biopharmaceutical company bringing innovative therapies to address unmet needs in severe burn and wound management, today announced that the Company will release its financial results for the fourth quarter and year ended December 31, 2020 at 7:00 am Eastern Time on Thursday, February 25, 2021.

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MediWound to Report Fourth Quarter and Fiscal 2020 Financial Results and Host a Conference Call and Webcast on February 25, 2021

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SAN DIEGO, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced it will report fourth quarter and full year 2020 financial results on Monday, March 1, 2021 after the close of the U.S. financial markets. The Company will host a conference call and webcast to discuss the financial results and provide a general business update at 4:30 p.m. ET.

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Travere Therapeutics to Report Fourth Quarter and Full Year 2020 Financial Results

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today announced that the Company will host an Expert Perspectives call on February 22, 2021 at 12:00pm EST. The call will feature a discussion between Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics and Paul Yang, MD, PhD about disease education and endpoints in Usher syndrome and non-syndromic Retinitis Pigmentosa (nsRP).  Areas of focus for the session will include which vision measures are most informative in the context of this disease setting, the role of patient baseline and disease progression, and an overview of the objectives of the Phase 1/2 Stellar trial of QR-421a.

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ProQR Announces Expert Perspectives Call on Disease Education and Endpoints in Usher Syndrome

TORONTO, CHICAGO and MONTREAL, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Medexus Pharmaceuticals Inc. (the “Company” or “Medexus”) (TSXV: MDP) (OTCQX: MEDXF) (Frankfurt: P731) announced today that it will be presenting at the Winter Wonderland Conference, hosted by The MicroCap Rodeo, being held virtually on February 16th – February 19th, 2021.

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CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, announced today that Paul Bolno, MD, MBA, President and Chief Executive Officer, is scheduled to participate in an analyst-led fireside chat at the virtual 10th Annual SVB Leerink Global Healthcare Conference on Thursday, February 25, 2021 at 5:00 p.m. ET.

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Wave Life Sciences to Present at the 10th Annual SVB Leerink Global Healthcare Conference  

CARLSBAD, Calif., Feb. 16, 2021 (GLOBE NEWSWIRE) -- GenMark Diagnostics, Inc. (NASDAQ: GNMK) (“GenMark” or the “Company”), a leading provider of automated, multiplex molecular diagnostic testing systems, today announced that the company plans to participate in the upcoming Cowen 41st Annual Health Care Conference.

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GenMark Diagnostics to Participate in the Cowen 41st Annual Health Care Conference

MARSEILLE, France, Feb. 17, 2021 (GLOBE NEWSWIRE) -- Innate Pharma SA (Euronext Paris: IPH – ISIN: FR0010331421; Nasdaq: IPHA) (“Innate” or the “Company”) announced today that members of its senior management team are scheduled to participate in the following upcoming virtual investor conference:

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Innate Pharma to Participate in the 10th Annual SVB Leerink Global Healthcare Conference

Geneva, Switzerland, February 17, 2021 – Addex Therapeutics (SIX: ADXN and Nasdaq: ADXN), a clinical stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development, today announced that it completed 2020 with a strong cash position of CHF18.7 million of cash and cash equivalents.

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Addex Provides Trading Update and Completes 2020 with Strong Cash Position of CHF18.7 million

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ObsEva to Present at the SVB Leerink 10th Annual Global Healthcare Conference

Basel, February 17, 2021 — Novartis today announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD). The project brings together Novartis drug discovery and gene therapy expertise with the Gates Foundation’s charitable objectives to expand access to healthcare in low-resource settings in an effort to address this potentially life-threatening genetic disease.

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Novartis and the Bill & Melinda Gates Foundation collaborate to discover and develop an accessible in vivo gene therapy for sickle cell disease