BEIJING, China and CAMBRIDGE, Mass., Dec. 22, 2019 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today announced that the China National Medical Products Administration (NMPA) has accepted a supplemental new drug application (sNDA) for REVLIMID (lenalidomide), in combination with rituximab, for the treatment of patients with relapsed or refractory indolent lymphoma (follicular lymphoma or marginal zone lymphoma). REVLIMID was first approved in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone, in adult patients who have received at least one prior therapy, and the label for the combination was expanded in 2018 to include adult patients with newly-diagnosed multiple myeloma (NDMM) who are not eligible for transplant. It is currently marketed in China by BeiGene under an exclusive license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.

This milestone for REVLIMID marks another step in the expansion of our hematology franchise into non-Hodgkins lymphoma (NHL) in China, where significant unmet medical needs remain. Together with the pending approvals of tislelizumab for Hodgkins lymphoma and zanubrutinib for mantle cell lymphoma and chronic lymphocytic leukemia as well as Revlimid for multiple myeloma, Vidaza for myelodysplastic syndromes and acute myeloid leukemia and additional products from the collaboration we have announced with Amgen, we are working to build a market-leading presence in the treatment of hematological cancers in China, said Dr. Xiaobin Wu, General Manager of China and President of BeiGene. We are excited about this opportunity and look forward to working closely with Bristol-Myers Squibb and the NMPA to bring this chemotherapy-free treatment option to patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma in China as soon as possible.

The sNDA is supported by a clinical, non-clinical, and chemistry, manufacturing and control (CMC) data package, including the results from the pivotal Phase 3 AUGMENT study (NCT01938001) sponsored and conducted by Bristol-Myers Squibb. AUGMENT is a randomized, double-blind, multicenter trial in which a total of 358 patients with relapsed or refractory follicular or marginal zone lymphoma were randomized 1:1 to receive REVLIMID and rituximab (R2) or rituximab and placebo. With a median follow-up of 28.3 months (range: 0.1 to 51.3 months), R2 demonstrated clinically meaningful and statistically significant improvement in progression-free survival (PFS), evaluated by an independent review committee (IRC), relative to the control arm with a 54% reduction in the risk of progression or death (hazard ratio [HR] = 0.46; 95% confidence interval [CI]: 0.34, 0.62; p < 0.0001). The median PFS was 39.4 months for the R2 arm and 14.1 months for the control arm with an improvement by more than 2 years. Overall response rate (ORR), a secondary endpoint, was 78% in the R2 arm vs. 53% in the control arm, as assessed by the IRC. Duration of response (DoR) was significantly improved for R2 vs. control with median DoR of 37 vs. 22 months, respectively (P =0.0015; HR: 0.53; 95% CI, 0.36-0.79). The most frequent adverse event (AE) in the R2 arm was neutropenia (58%), vs. 22% in the control arm. Additional commonly observed AEs in more than 20% of patients included diarrhea (31% in the R2 arm vs. 23% in the control arm), constipation (26% vs. 14%), cough (23% vs. 17%), and fatigue (22% vs. 18%). Adverse events that were reported at a higher rate (>10%) in the R2 arm were neutropenia, constipation, leukopenia, anemia, thrombocytopenia and tumor flare.

About follicular lymphoma (FL) and marginal zone lymphoma (MZL)

FL and MZL are two major types of indolent lymphomas;1 FL is the most common subtype, constituting approximately 20% to 25% of all NHL,2 followed by MZL (approximately 5% to 17% of all NHLs).3 NHL incidence in China is 88,090 according to the World Health Organizations Globocan 2018 database.4 Given the incurable nature of relapsed or refractory FL/MZL, the efficacy and safety limitations of current treatment options, and the fact that patients are typically older and with comorbidities, a high unmet medical need exists for the development of novel treatment options with new differentiated mechanisms of action and a more tolerable safety profile that can improve the quality of response and PFS in the setting of previously treated FL/MZL.

About REVLIMID

In China, REVLIMID was approved in combination with dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma (MM) who are not eligible for transplant in 2018. It received approval in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone in adult patients who have received at least one prior therapy.

REVLIMID is approved in Europe and the United States as monotherapy, indicated for the maintenance treatment of adult patients with newly diagnosed MM who have undergone autologous stem cell transplantation. REVLIMID as combination therapy is approved in Europe, in the United States, in Japan and in around 25 other countries for the treatment of adult patients with previously untreated MM who are not eligible for transplant. REVLIMID is also approved in combination with dexamethasone for the treatment of patients with MM who have received at least one prior therapy in nearly 70 countries, encompassing Europe, the Americas, the Middle-East and Asia, and in combination with dexamethasone for the treatment of patients whose disease has progressed after one therapy in Australia and New Zealand.

REVLIMID is also approved in the United States, Canada, Switzerland, Australia, New Zealand and several Latin American countries, as well as Malaysia and Israel, for transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities and in Europe for the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

In addition, REVLIMID is approved in Europe for the treatment of patients with mantle cell lymphoma (MCL) and in the United States for the treatment of patients with MCL whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. In Switzerland, REVLIMID is indicated for the treatment of patients with relapsed or refractory MCL after prior therapy that included bortezomib and chemotherapy/rituximab.

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.

U.S. Indications for REVLIMID

REVLIMID (lenalidomide) in combination with dexamethasone (dex) is indicated for the treatment of adult patients with multiple myeloma (MM).

REVLIMID is indicated as maintenance therapy in adult patients with MM following autologous hematopoietic stem cell transplantation (auto-HSCT).

REVLIMID is indicated for the treatment of adult patients with transfusion-dependent anemia due to low-or intermediate-1risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities.

REVLIMID is indicated for the treatment of adult patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib.

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated follicular lymphoma (FL).

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL).

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.

REVLIMID is only available through a restricted distribution program, REVLIMID REMS.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.

Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.

Hematologic Toxicity (Neutropenia and Thrombocytopenia)

REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.

Venous and Arterial Thromboembolism

REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.

CONTRAINDICATIONS

Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus.

Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide.

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: See Boxed WARNINGS.

REVLIMID REMS Program: See Boxed WARNINGS. Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements.

Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. Patients may require a dose interruption and/or dose reduction. MM: Monitor complete blood counts (CBC) in patients taking REVLIMID + dexamethasone or REVLIMID as maintenance therapy, every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Monitor CBC in patients on therapy for del 5q MDS, weekly for the first 8 weeks of therapy and at least monthly thereafter. See Boxed WARNINGS for further information. MCL: Monitor CBC in patients taking REVLIMID for MCL weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. FL/MZL: Monitor CBC in patients taking REVLIMID for FL or MZL weekly for the first 3 weeks of Cycle 1 (28 days), every 2 weeks during Cycles 2-4, and then monthly thereafter.

Venous and Arterial Thromboembolism: See Boxed WARNINGS. Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on the patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision.

Increased Mortality in Patients With CLL: In a clinical trial in the first-line treatment of patients with CLL, single-agent REVLIMID therapy increased the risk of death as compared to single-agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials.

Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID and in patients with FL or MZL receiving REVLIMID + rituximab therapy, an increase of hematologic plus solid tumor SPM, notably AML, have been observed. In patients with MM, MDS was also observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment.

Increased Mortality With Pembrolizumab: In clinical trials in patients with MM, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with MM with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.

Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID + dexamethasone. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered.

Severe Cutaneous Reactions: Severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. Consider REVLIMID interruption or discontinuation for Grade 2-3 skin rash. Permanently discontinue REVLIMID for Grade 4 rash, exfoliative or bullous rash, or for other severe cutaneous reactions such as SJS, TEN, or DRESS.

Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with REVLIMID. The patients at risk of TLS are those with high tumor burden prior to treatment. Closely monitor patients at risk and take appropriate preventive approaches.

Tumor Flare Reaction (TFR): TFR has occurred during investigational use of REVLIMID for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL, FL, or MZL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion.

Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection.

Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before starting REVLIMID treatment and during therapy.

Early Mortality in Patients With MCL: In another MCL study, there was an increase in early deaths (within 20 weeks); 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L).

Hypersensitivity: Hypersensitivity, including angioedema, anaphylaxis, and anaphylactic reactions to REVLIMID has been reported. Permanently discontinue REVLIMID for angioedema and anaphylaxis.

ADVERSE REACTIONS

Multiple Myeloma

Myelodysplastic Syndromes

Mantle Cell Lymphoma

Follicular Lymphoma/Marginal Zone Lymphoma

DRUG INTERACTIONS

Periodically monitor digoxin plasma levels due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin-stimulating agents or estrogen-containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dexamethasone and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin.

USE IN SPECIFIC POPULATIONS

Please see full Prescribing Information, including Boxed WARNINGS, for REVLIMID.

Please see the rituximab full Prescribing Information for Important Safety Information at http://www.rituxan.com.

About BeiGene

BeiGene is a global, commercial-stage, research-based biotechnology company focused on molecularly-targeted and immuno-oncology cancer therapeutics. With a team of over 3,000 employees in the United States, China, Australia, and Europe; BeiGene is advancing a pipeline consisting of novel oral small molecules and monoclonal antibodies for cancer. BeiGene is also working to create combination solutions aimed to have both a meaningful and lasting impact on cancer patients. In the United States, BeiGene markets and distributes BRUKINSA (zanubrutinib) and in China, the Company markets ABRAXANE (paclitaxel for injection [albumin bound]), REVLIMID (lenalidomide), and VIDAZA (azacitidine) under a license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.5

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGenes plans and expectations for further development and commercialization of REVLIMID in China and the potential implications for patients. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

______________________1 Bello C, Zhang L, Naghashpour M. Follicular lymphoma: current management and future directions. Cancer Control. 2012;19:187-95.

2 Sousou T, Friedberg J. Rituximab in indolent lymphomas. Semin Hematol. 2010; 47(2):133-42.

3 Zinzani, P. L. (2012). The many faces of marginal zone lymphoma. Hematology, 2012(1), 426432.

4 https://gco.iarc.fr/

5 ABRAXANE is registered trademark of Abraxis Bioscience LLC, a Bristol-Myers Squibb company; REVLIMID and VIDAZA are registered trademarks of Celgene Corporation, a Bristol-Myers Squibb company.

See the original post here:
BeiGene Announces Acceptance of a Supplemental New Drug Application in China for REVLIMID in Relapsed or Refractory Indolent Lymphoma - BioSpace

BEIJING, China and CAMBRIDGE, Mass., Dec. 22, 2019 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today announced that the China National Medical Products Administration (NMPA) has accepted a supplemental new drug application (sNDA) for REVLIMID (lenalidomide), in combination with rituximab, for the treatment of patients with relapsed or refractory indolent lymphoma (follicular lymphoma or marginal zone lymphoma). REVLIMID was first approved in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone, in adult patients who have received at least one prior therapy, and the label for the combination was expanded in 2018 to include adult patients with newly-diagnosed multiple myeloma (NDMM) who are not eligible for transplant. It is currently marketed in China by BeiGene under an exclusive license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.

This milestone for REVLIMID marks another step in the expansion of our hematology franchise into non-Hodgkins lymphoma (NHL) in China, where significant unmet medical needs remain. Together with the pending approvals of tislelizumab for Hodgkins lymphoma and zanubrutinib for mantle cell lymphoma and chronic lymphocytic leukemia as well as Revlimid for multiple myeloma, Vidaza for myelodysplastic syndromes and acute myeloid leukemia and additional products from the collaboration we have announced with Amgen, we are working to build a market-leading presence in the treatment of hematological cancers in China, said Dr. Xiaobin Wu, General Manager of China and President of BeiGene. We are excited about this opportunity and look forward to working closely with Bristol-Myers Squibb and the NMPA to bring this chemotherapy-free treatment option to patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma in China as soon as possible.

The sNDA is supported by a clinical, non-clinical, and chemistry, manufacturing and control (CMC) data package, including the results from the pivotal Phase 3 AUGMENT study (NCT01938001) sponsored and conducted by Bristol-Myers Squibb. AUGMENT is a randomized, double-blind, multicenter trial in which a total of 358 patients with relapsed or refractory follicular or marginal zone lymphoma were randomized 1:1 to receive REVLIMID and rituximab (R2) or rituximab and placebo. With a median follow-up of 28.3 months (range: 0.1 to 51.3 months), R2 demonstrated clinically meaningful and statistically significant improvement in progression-free survival (PFS), evaluated by an independent review committee (IRC), relative to the control arm with a 54% reduction in the risk of progression or death (hazard ratio [HR] = 0.46; 95% confidence interval [CI]: 0.34, 0.62; p < 0.0001). The median PFS was 39.4 months for the R2 arm and 14.1 months for the control arm with an improvement by more than 2 years. Overall response rate (ORR), a secondary endpoint, was 78% in the R2 arm vs. 53% in the control arm, as assessed by the IRC. Duration of response (DoR) was significantly improved for R2 vs. control with median DoR of 37 vs. 22 months, respectively (P =0.0015; HR: 0.53; 95% CI, 0.36-0.79). The most frequent adverse event (AE) in the R2 arm was neutropenia (58%), vs. 22% in the control arm. Additional commonly observed AEs in more than 20% of patients included diarrhea (31% in the R2 arm vs. 23% in the control arm), constipation (26% vs. 14%), cough (23% vs. 17%), and fatigue (22% vs. 18%). Adverse events that were reported at a higher rate (>10%) in the R2 arm were neutropenia, constipation, leukopenia, anemia, thrombocytopenia and tumor flare.

About follicular lymphoma (FL) and marginal zone lymphoma (MZL)

FL and MZL are two major types of indolent lymphomas;1 FL is the most common subtype, constituting approximately 20% to 25% of all NHL,2 followed by MZL (approximately 5% to 17% of all NHLs).3 NHL incidence in China is 88,090 according to the World Health Organizations Globocan 2018 database.4 Given the incurable nature of relapsed or refractory FL/MZL, the efficacy and safety limitations of current treatment options, and the fact that patients are typically older and with comorbidities, a high unmet medical need exists for the development of novel treatment options with new differentiated mechanisms of action and a more tolerable safety profile that can improve the quality of response and PFS in the setting of previously treated FL/MZL.

About REVLIMID

In China, REVLIMID was approved in combination with dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma (MM) who are not eligible for transplant in 2018. It received approval in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone in adult patients who have received at least one prior therapy.

REVLIMID is approved in Europe and the United States as monotherapy, indicated for the maintenance treatment of adult patients with newly diagnosed MM who have undergone autologous stem cell transplantation. REVLIMIDas combination therapy is approved inEurope, inthe United States, inJapanand in around 25 other countries for the treatment of adult patients with previously untreated MM who are not eligible for transplant. REVLIMID is also approved in combination with dexamethasone for the treatment of patients with MM who have received at least one prior therapy in nearly 70 countries, encompassingEurope, theAmericas, theMiddle-EastandAsia, and in combination with dexamethasone for the treatment of patients whose disease has progressed after one therapy inAustralia and New Zealand.

REVLIMIDis also approved inthe United States,Canada,Switzerland,Australia,New Zealandand several Latin American countries, as well asMalaysiaandIsrael, for transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities and inEuropefor the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

In addition, REVLIMIDis approved inEuropefor the treatment of patients with mantle cell lymphoma (MCL) and inthe United Statesfor the treatment of patients with MCL whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. InSwitzerland, REVLIMID is indicated for the treatment of patients with relapsed or refractory MCL after prior therapy that included bortezomib and chemotherapy/rituximab.

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.

U.S. Indications for REVLIMID

REVLIMID (lenalidomide) in combination with dexamethasone (dex) is indicated for the treatment of adult patients with multiple myeloma (MM).

REVLIMID is indicated as maintenance therapy in adult patients with MM following autologous hematopoietic stem cell transplantation (auto-HSCT).

REVLIMID is indicated for the treatment of adult patients with transfusion-dependent anemia due to low-or intermediate-1risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities.

REVLIMID is indicated for the treatment of adult patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib.

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated follicular lymphoma (FL).

REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL).

REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.

REVLIMID is only available through a restricted distribution program, REVLIMID REMS.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.

Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.

Hematologic Toxicity (Neutropenia and Thrombocytopenia)

REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.

Venous and Arterial Thromboembolism

REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.

CONTRAINDICATIONS

Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus.

Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide.

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: See Boxed WARNINGS.

REVLIMID REMS Program: See Boxed WARNINGS. Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements.

Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. Patients may require a dose interruption and/or dose reduction. MM: Monitor complete blood counts (CBC) in patients taking REVLIMID + dexamethasone or REVLIMID as maintenance therapy, every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Monitor CBC in patients on therapy for del 5q MDS, weekly for the first 8 weeks of therapy and at least monthly thereafter. See Boxed WARNINGS for further information. MCL: Monitor CBC in patients taking REVLIMID for MCL weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. FL/MZL: Monitor CBC in patients taking REVLIMID for FL or MZL weekly for the first 3 weeks of Cycle 1 (28 days), every 2 weeks during Cycles 2-4, and then monthly thereafter.

Venous and Arterial Thromboembolism: See Boxed WARNINGS. Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on the patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision.

Increased Mortality in Patients With CLL: In a clinical trial in the first-line treatment of patients with CLL, single-agent REVLIMID therapy increased the risk of death as compared to single-agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials.

Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID and in patients with FL or MZL receiving REVLIMID + rituximab therapy, an increase of hematologic plus solid tumor SPM, notably AML, have been observed. In patients with MM, MDS was also observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment.

Increased Mortality With Pembrolizumab: In clinical trials in patients with MM, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with MM with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.

Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID + dexamethasone. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered.

Severe Cutaneous Reactions: Severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. Consider REVLIMID interruption or discontinuation for Grade 2-3 skin rash. Permanently discontinue REVLIMID for Grade 4 rash, exfoliative or bullous rash, or for other severe cutaneous reactions such as SJS, TEN, or DRESS.

Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with REVLIMID. The patients at risk of TLS are those with high tumor burden prior to treatment. Closely monitor patients at risk and take appropriate preventive approaches.

Tumor Flare Reaction (TFR): TFR has occurred during investigational use of REVLIMID for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL, FL, or MZL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion.

Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection.

Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before starting REVLIMID treatment and during therapy.

Early Mortality in Patients With MCL: In another MCL study, there was an increase in early deaths (within 20 weeks); 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L).

Hypersensitivity: Hypersensitivity, including angioedema, anaphylaxis, and anaphylactic reactions to REVLIMID has been reported. Permanently discontinue REVLIMID for angioedema and anaphylaxis.

ADVERSE REACTIONS

Multiple Myeloma

Myelodysplastic Syndromes

Mantle Cell Lymphoma

Follicular Lymphoma/Marginal Zone Lymphoma

DRUG INTERACTIONS

Periodically monitor digoxin plasma levels due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin-stimulating agents or estrogen-containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dexamethasone and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin.

USE IN SPECIFIC POPULATIONS

Please see full Prescribing Information, including Boxed WARNINGS, for REVLIMID.

Please see the rituximab full Prescribing Information for Important Safety Information at http://www.rituxan.com.

About BeiGene

BeiGene is a global, commercial-stage, research-based biotechnology company focused on molecularly-targeted and immuno-oncology cancer therapeutics. With a team of over 3,000 employees in the United States, China, Australia, and Europe; BeiGene is advancing a pipeline consisting of novel oral small molecules and monoclonal antibodies for cancer. BeiGene is also working to create combination solutions aimed to have both a meaningful and lasting impact on cancer patients. In the United States, BeiGene markets and distributes BRUKINSA (zanubrutinib) and in China, the Company markets ABRAXANE (paclitaxel for injection [albumin bound]), REVLIMID (lenalidomide), and VIDAZA (azacitidine) under a license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.5

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGenes plans and expectations for further development and commercialization of REVLIMID in China and the potential implications for patients. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

______________________1 Bello C, Zhang L, Naghashpour M. Follicular lymphoma: current management and future directions. Cancer Control. 2012;19:187-95.

2 Sousou T, Friedberg J. Rituximab in indolent lymphomas. Semin Hematol. 2010; 47(2):133-42.

3 Zinzani, P. L. (2012). The many faces of marginal zone lymphoma. Hematology, 2012(1), 426432.

4 https://gco.iarc.fr/

5 ABRAXANEis registered trademark ofAbraxis Bioscience LLC, aBristol-Myers Squibb company; REVLIMIDand VIDAZAare registered trademarks ofCelgene Corporation, aBristol-Myers Squibbcompany.

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BeiGene Announces Acceptance of a Supplemental New Drug Application in China for REVLIMID in Relapsed or Refractory Indolent Lymphoma - GlobeNewswire

News Release

Tuesday, December 10, 2019

New findings from a clinical trial show that treatment with the immunotherapy drug blinatumomab is superior to standard chemotherapy for children and young adults with high- or intermediate-risk B-cell acute lymphoblastic leukemia (B-ALL) that has relapsed. Those treated with blinatumomab had longer survival, experienced fewer severe side effects, had a higher rate of undetectable residual disease, and were more likely to proceed to a stem cell transplant.

Our study demonstrates that immunotherapy with blinatumomab is more effective and less toxic than chemotherapy as a bridge to curative bone marrow transplant for children and young adults with very aggressive relapse of B-ALL, said Patrick Brown, M.D., who chaired the trial and is director of the Pediatric Leukemia Program at the Johns Hopkins Kimmel Cancer Center, Baltimore. We are thrilled that these patients, whose survival has not substantially improved for decades, now have a new and better standard of care.

The findings were presented as a late-breaking abstract at the American Society of Hematology (ASH) annual meeting on Dec. 10, 2019. The trial was led by the Childrens Oncology Group (COG), part of the National Cancer Institute (NCI)sponsored National Clinical Trials Network. NCI is part of the National Institutes of Health. Amgen reviewed the trial protocol and amendments and provided the study drug under a Cooperative Research and Development Agreement with NCI.

These findings will likely have immediate impact on the treatment of this group of children and young adults with relapsed B-ALL, said Malcolm Smith, M.D., Ph.D., associate branch chief for pediatric oncology in NCIs Cancer Therapy Evaluation Program, which sponsored the trial. These results also reinforce the important role that federally funded clinical trials play in developing more effective treatments for children with cancer.

When children have B-ALL that relapses after their initial treatment, they are typically given chemotherapy. The first four to six weeks of chemotherapy, the reinduction phase, is commonly followed by additional intensive chemotherapy, or consolidation treatment, to further reduce disease levels. Following this, hematopoietic stem cell transplant is considered the best treatment for approximately half of patients, based on factors such as whether relapse occurred during initial treatment or shortly after it was completed.

However, chemotherapy can produce severe side effects in some patients and is sometimes ineffective in reducing leukemia levels to the low levels needed prior to transplant. As a result, patients may not be able to proceed to transplant or transplant may be delayed, which increases the risk that the leukemia will return.

The COG study investigated blinatumomab as an alternative type of consolidation treatment to follow the reinduction phase. Blinatumomab is a type of immunotherapy that works by binding to two different molecules: CD19, a protein, or antigen, expressed on the surface of B-ALL cells, and CD3, an antigen expressed on T cells. By bringing T cells close to leukemia cells, the immunotherapy helps the T cells recognize and kill the cancer cells.

Blinatumomab has been approved by the U.S. Food and Drug Administration (FDA) for adults and children with B-ALL that has returned or has not responded to treatment. FDA has also granted accelerated approval to the drugmeaning confirmatory trials must show it has clinical benefitfor some adults and children undergoing treatment for B-ALL who achieve complete remission but still have small amounts of leukemia detectable using very sensitive methods.

Investigators in this study wanted to see if blinatumomab could increase rates of survival free from leukemia and be less toxic than intensive chemotherapy in children and young adults undergoing consolidation treatment.

The trial report was based on 208 children and young adults aged 130 with relapsed B-ALL who had received reinduction chemotherapy and were considered to have high- or intermediate-risk disease. They were randomly assigned to receive either two rounds of intensive chemotherapy or two 4-week rounds of treatment with blinatumomab before proceeding to a transplant. (A separate part of the study addressed children with low-risk disease.)

After a median follow-up time of 1.4 years, those in the blinatumomab group had higher rates of 2-year disease-free survival, the primary outcome of the study, than those who received intensive chemotherapy (59.3 5.4% vs. 41 6.2%). Those treated with blinatumomab also had higher rates of overall survival (79.4 4.5% vs. 59.2 6%), fewer severe side effects, a higher rate of undetectable residual disease (79% vs. 21%), and a higher rate of proceeding to stem cell transplant (73% vs. 45%).

At a planned interim analysis, an independent data safety monitoring committee concluded that the outcome for children treated with blinatumomab was superior to that of children treated with chemotherapy only and recommended that enrollment to the high- and intermediate-risk part of the trial be stopped.

Future clinical trials will study whether blinatumomabs effects in relapsed B-ALL can be enhanced by combining it with other immunotherapy and will test whether adding the drug to standard chemotherapy for children and young adults with newly diagnosed B-ALL is beneficial.

About the National Cancer Institute (NCI):NCIleads the National Cancer Program and NIHs efforts to dramatically reduce the prevalence of cancer and improve the lives of cancer patients and their families, through research into prevention and cancer biology, the development of new interventions, and the training and mentoring of new researchers. For more information about cancer, please visit the NCI website atcancer.govor call NCIs contact center, the Cancer Information Service, at 1-800-4-CANCER (1-800-422-6237).

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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Immunotherapy drug improves outcomes for some children with relapsed leukemia - National Institutes of Health

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, today announced the launch of the company with a $35 million total Series A financing. Abingworth LLP and Qiming Venture Partners USA served as lead investors, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC. The proceeds will be used to advance the clinical development of the companys lead product candidate, JSP191, which is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplant.

Jaspers development of JSP191 is also supported by a collaboration with the California Institute for Regenerative Medicine (CIRM), which has been funding the program and is committed to providing a total of $23 million in grant support. As part of the Series A financing, Amgen, which discovered JSP191 (formerly AMG191), has licensed worldwide rights to Jasper that also include translational science and materials from Stanford University.

Jasper was co-founded by Judith Shizuru, M.D., Ph.D., a hematopoietic stem cell transplant expert at Stanford University, and Susan Prohaska, Ph.D., a Stanford University-trained immunologist, stem cell biologist and early-stage drug development professional. Dr Shizurus CIRM-funded lab advanced the understanding of the ability of anti-CD117 to impact hematopoietic stem cells and, together with the Lucile Packard Childrens Hospital Stanford and University of California, San Francisco (UCSF) pediatric transplant teams, was the first to study an anti-CD117 antibody in the clinic as a conditioning agent. That humanized antibody, now called JSP191, was first studied for conditioning for transplant in immune-deficient patients in collaboration with Amgen, UCSF and CIRM.

Stem cell transplantation is a potential curative therapy for people with hematologic cancers, autoimmune diseases, and debilitating genetic diseases. However, the pre-transplant conditioning required to prepare patients for transplant involves highly toxic chemotherapy, which can be life-threatening and limits the number of people who are able to benefit, said Dr. Shizuru, co-founder and member of the Board of Directors of Jasper Therapeutics. JSP191 is the only anti-CD117 antibody to demonstrate safety and efficacy in severely ill patients receiving stem cell transplant in the clinic. We plan to expand clinical development to patients receiving transplants for acute myeloid leukemia/ myelodysplastic syndrome or autoimmune diseases and to patients receiving stem cell-directed gene therapies.

Dr. Shizuru added, With an experienced executive team of biotech veterans and a strong syndicate of healthcare-focused investors, Jasper Therapeutics is well positioned to achieve our vision of building a leading biotech company starting with JSP191 and expanding to other novel therapies for immune modulation, graft engineering and cell and gene therapies.

JSP191 is currently being evaluated in an ongoing Phase 1 clinical trial as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that failed. This severe genetic immune disorder leaves patients without a functioning immune system. Interim results of the study will be presented in an oral presentation (abstract #800) on Monday, December 9, at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Fla. Clinical studies to evaluate the safety and efficacy of JSP191 as a conditioning agent in patients undergoing hematopoietic cell therapy for hematologic cancers are planned for 2020.

Founding Management Team

Dr. Shizuru and Mr. Lis are joined on the Jasper Therapeutics Board of Directors by Kurt von Emster, Managing Partner of Abingworth LLP, and Anna French, Ph.D., Principal at Qiming Venture Partners USA. Dr. Prohaska is a Board observer.

With our investment in this program, were able to realize our mission of fast-tracking stem cell treatments by helping academic researchers rapidly advance the most promising discoveries in the lab into the clinics and to drug development with commercialization partners, said Maria T. Millan, M.D., President and CEO of CIRM. Jaspers two co-founders took a novel antibody with unique properties and moved it from the bench to the bedside relatively quickly, and were thrilled to partner with this talented team to potentially impact a broad group of people who could benefit from stem cell therapy.

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic doses of radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

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Jasper Therapeutics Launches with $35 Million Series A Financing to Develop and Commercialize Innovative Conditioning Agents and Therapies to...

The first girl in the trial came in with chronic diarrhea and the immune system of an untreated HIV patient. Born with a rare genetic disease that impeded her ability to make B and T cells, she had once been given a stem cell transplant but it didnt take. Back in the hospital, she was injected with a new experimental antibody and then given a new stem cell transplant. Soon, she gained weight. The diarrhea stopped.

She has normal T cells now, Judith Shizuru, the Stanford scientist who pioneered the antibody, told Endpoints News. Shes in school.

Its the kind of medical story to launch a biotech around, and thats what Shizuruis doing. Today, her company Jasper Therapeutics is emerging out of stealth-mode with $35 million in Series A funding led by Abingworth and Qiming, a molecule from Amgen, and a Phase I trial set for its first readout on Monday at ASH.

Jasper is broadly aimed at making stem cell transplants safer, more accessible and more effective by using antibodies as conditioning agents. Theseagents clear out bone marrow to make room for the new stem cells to graft onto the body.

Their Phase I uses a naked antibody called JSP191 to help patients with severe combined autoimmune deficiency receive stem cell transplants the only possible cure for the life-threatening disease but such transplants are used in a wide variety of conditions and Jasper has broader aims. Those include other autoimmune diseases, acute myeloid leukemia and cell-directed gene therapy.

Theres a significant amount of progress being made in gene therapy, interim CEO William Lis told Endpoints, but no progress being made in a conditioning agent that will help graft gene therapy.

Shizuru path to the new antibody was long and fortuitous. In 1987, Arl Arzst, the legendary ad executive and president of Proctor and Gamble international flew in on a recruiting trip for Stanford business students. There he visited Shizuru, a young biologyPhD candidate, because he knew her roommate. Arzsts daughter had diabetes and as Shizuru explained the work she was doing on pancreatic islet cell transplants, he told her to come to Europe.

Shizuru had never been to Europe, but there Arszt introduced her to Ken Farber and the other founders of the Juvenile Diabetes Foundation (now the JDRF). The founders struck a years-long correspondence and encouraged Shizuru to go to medical school, where she decided that if scientists were ever going to develop transplants that didnt trigger an immune response, it would be through stem cell work. She continued her work at the Irv Weissman Stanford regenerative lab, where eventually a graduate student made a discovery that piqued her interest.

To put new stem cells in, you have to get the old stem cells out. Thats not always easy. The cells sit inthese pockets in the bone marrow, and theyre pretty comfortable there. Doctors have to force them out, often using chemotherapy or radiation, which damage DNA and cause severe side effects. The costs sometimes outweigh the benefits.

There are diseases were not treating because its too dangerous, Shizuru said. And the kids were treating, theyre so, so fragile.

The grad student had shown in mice that antibodies could be used to deplete the stem cells and potentially eliminate the need for chemotherapy or radiation. Shizuru and her team began looking to see if anyone had developed a human version of the antibody, CD117. It turned out Amgen had already developed a version of this antibody for a different use. It also turned out she had a former postdoc and a former advisor who worked there. They began a collaboration.

We set out to cross the valley of death, Shizuru said, using an industry slang term for the jump from animal models to human uses.

After making a variety of tweaks to the treatment, they published a paper inScience Translational Medicine in 2016showing the antibodies created a 10,000 fold reduction in the number of stem cells in mice.

The same year, they began a clinical trial on 90 SCID patients. These patients had received stem cell transplants when they were very young but hadnt been given chemo or radiation for fear the side effects would be too severe. The original transplants boosted their numberof immune cells, but without chemo or radiation, the stem cells dont graft into those pockets and the body wont continue producing T cells. Without those, they are extraordinarily prone to infection. Many pass away before age 2.

The hope is that the antibodies allowed the stem cells to graft, and the preliminary answer to that question will be out on Monday. For the first girl in the trial, life has improved but questions about how long her body will make immune cells remain. Still, for that girl and others, Shizuru is confident.

We see there is stem cell engraftment, Shurizi said. They are actually making new T cells.

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Jasper Therapeutics launches out of Stanford with new approach to stem cell treatment - Endpoints News

SAN FRANCISCO--(BUSINESS WIRE)--Imago BioSciences, Inc., a clinical-stage biotechnology company focused on the treatment of myeloproliferative neoplasms (MPN) and related bone marrow diseases, announced today that preliminary data from its ongoing Phase 2 study of IMG-7289 (bomedemstat) in patients with myelofibrosis (MF) has been selected for an oral presentation at the American Society of Hematology (ASH) Annual Meeting, on December 9 in Orlando, Florida. The abstract will be published November 6, and the presentation will include results updated from those in the abstract.

Kristen Pettit, M.D., assistant professor at the University of Michigan and investigator in the study at the Rogel Cancer Center in Ann Arbor, will present both preliminary results from Phase 2a, as well as initial data from patients from the Phase 2b expansion. The objectives of the study are to evaluate the safety and efficacy of IMG-7289 (bomedemstat) in up to 75 patients at sites in Australia, the US, UK and Europe. In this study, bomedemstat is administered orally once-daily as monotherapy in adult patients with intermediate-2 or high-risk MF resistant to or intolerant of ruxolitinib.

The FDA recently approved a second JAK2 inhibitor but the majority of patients with myelofibrosis will eventually lose the benefit of those treatments, said Dr. Pettit. Patients have an urgent need for new treatments that manage their symptoms. We continue to be encouraged by the bomedemstat data we see in this clinical investigation.

Imago Presentation

Title: A Phase 2 Study of the LSD1 Inhibitor IMG-7289 (bomedemstat) for the Treatment of Myelofibrosis. Session: 634. Myeloproliferative Syndromes: Clinical: Emerging and Novel Targeted TherapiesSession Date: Monday, December 9, 2019Session Time: 7:00 AM - 8:30 AM ESTPresentation Time: 7:45 AM ESTRoom: Orange County Convention Center, W304EFGH

About IMG-7289

IMG-7289 (bomedemstat) is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A). LSD1 is an enzyme regulating both cytokine expression and myeloid differentiation and sustaining self-renewal in malignant hematopoietic stem/progenitor cells. In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutic agents across a range of myeloid malignancy models, including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis. An international Phase 2b study of bomedemstat for the treatment of myelofibrosis remains ongoing (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. Imago is focused on improving the management of malignant and life-threatening diseases of the bone marrow with a focus on the myeloproliferative neoplastic disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. The company is based in California.

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Imago BioSciences Preliminary Data from Ongoing Phase 2 Study of IMG-7289 for the Treatment of Myelofibrosis to be Presented at the 61st American...

Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research 2019 report by Mart Research outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fanconi Anemia (FA) Fanconi Hypoplastic Anemia pipeline landscape is provided which includes the disease overview and Fanconi Anemia (FA) Fanconi Hypoplastic Anemia treatment guidelines. The assessment part of the report embraces, in depth Fanconi Anemia (FA) Fanconi Hypoplastic Anemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Fanconi Anemia (FA) Fanconi Hypoplastic Anemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

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Table of Content for Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report:Chapter One: Report IntroductionChapter Two: Fanconi Anemia (Fanconi hypoplastic anemia, Fanconi pancytopenia, Fanconi panmyelopathy)Chapter Three: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Current Treatment PatternsChapter Four: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Mart Researchs Analytical PerspectiveChapter Five: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline TherapeuticsChapter Six: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia -Products AnalysisChapter Seven: Recent TechnologiesChapter Eight: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Key CompaniesChapter Nine: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Key ProductsChapter Ten: Dormant and Discontinued ProductsChapter Eleven: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Unmet NeedsChapter Twelve: Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Future Perspectives

List of Tables for Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report:Table 1. Diagnostic GuidelinesTable 2. Treatment GuidelinesTable 3. Assessment SummaryTable 4. Company-Company Collaborations (Licensing / Partnering) AnalysisTable 5. Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Acquisition AnalysisTable 6. Assessment by Phase of DevelopmentTable 7. Assessment by Product Type (Mono / Combination)Table 8. Assessment by Stage and Product TypeTable 9. Assessment by Route of AdministrationTable 10. Assessment by Stage and Route of AdministrationTable 11. Assessment by Molecule TypeTable 12. Assessment by Stage and Molecule TypeTable 13. Assessment by MOATable 14. Assessment by Stage and MOATable 15. Late Stage Products (Phase-III)Table 16. Mid Stage Products (Phase-II)Table 17. Early Stage Products (Phase-I)Table 18. Pre-clinical and Discovery Stage ProductsTable 19. Inactive ProductsTable 20. Dormant ProductsTable 21. Discontinued Products

To Check Discount on Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report @ https://martresearch.com/contact/discount/2/42145

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Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report 2019: by Trends, Development, Types,...

Cellular therapy is also known as cytotherapy or cell therapy. Cellular therapy (CT) is the transplantation of cellular material into human body to repair or replace damaged tissue and damaged cells. Advancement in technology, development of innovative products, and growth in the number of research activities have helped in the discovery of several types of cells that are likely to be used in the treatment or therapy of various conditions and diseases. Several cells such as hematopoietic stem cells (HSC), mesenchymal stem cells, skeletal muscle stem cells, dendritic cells, pancreatic islet cells, and lymphocytes can be used in cellular therapy. HSC is extensively used in cellular therapy. Cellular therapy is used to treat various types of cancers, infectious diseases, autoimmune diseases, and urinary problems. The therapy also helps patients rebuild damaged cartilage in joints, improve a weakened immune system, and repair spinal cord injuries. Moreover, it also helps treat neurological disorders. Cellular cancer therapy has various approaches as the cell can be designed to stimulate the patients immune system (T cells or natural killer cells) to kill cancer cells, or to replace most of the patients immune system to enhance their immune response to cancer cells, or to directly find and kill the cancer cells.

The global cellular therapy in oncology market has been classified based on cancer type and geography. In terms of cancer type, the market has been categorized into blood cancer, prostate cancer, pancreatic cancer, brain cancer, and other cancer. The blood cancer segment accounts for a major share of cellular therapy in oncology market. Increasing prevalence of prostate cancer is expected propel the segment in the near future. According to WHO statistics, 8.2 million people die each year due to cancer which estimates about 13% of all death worldwide. There are more than 100 types of cancers that require unique diagnoses and therapies. This increases the demand for cellular therapy in oncology in near future.

Geographically, the cellular therapy in oncology market has been segmented into five major regions: North America, Europe, Latin America, Asia Pacific, and Middle East & Africa. In terms of revenue, North America dominates the cellular therapy in oncology market followed by Europe. The market in Asia Pacific and Latin America is developing. This trend is expected to continue during the forecast period. Availability of large patient pool, expansion of the health care industry, and rise in government investment to improve the health care industry are anticipated to propel the market in these regions. The cellular therapy in oncology market in countries such as Brazil, China, and India are projected to expand at substantial growth rate during the forecast period due to rise in awareness among the population about the usage of cellular therapy to treat various types of cancers and rapid innovations in cellular therapy.

Browse more detail information about this report visit at at https://www.transparencymarketresearch.com/cellular-therapy-oncology-market.html

Increasing prevalence of various cancers, affordability of cellular therapy in cancer drugs, high adoption in developed markets, and development of innovative drugs are other factors driving the cellular therapy in oncology market. High competition among existing players, high risks of failure, severity and complications involved in cellular therapy due to misdiagnosis, and lack of awareness among the rural population in underdeveloped and developing economies are likely to inhibit the market.

Major players operating in the cellular therapy in oncology market include Alkem Laboratories Limited, Amgen, Inc., Bayer AG, Sanofi, Bristol-Myers Squibb, Boehringer Ingelheim GmbH, F. Hoffmann-La Roche Ltd, Cipla, Inc., Merck & Co., Inc., Eli Lilly and Company, GlaxoSmithKline Plc., Johnson & Johnson Services, Inc., Novartis AG, Pfizer, Inc., and Teva Pharmaceutical Industries Ltd.

The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.

The study is a source of reliable data on: Market segments and sub-segments Market trends and dynamics Supply and demand Market size Current trends/opportunities/challenges Competitive landscape Technological breakthroughs Value chain and stakeholder analysis

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The regional analysis covers: North America (U.S. and Canada) Latin America (Mexico, Brazil, Peru, Chile, and others) Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg) Eastern Europe (Poland and Russia) Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand) Middle East and Africa (GCC, Southern Africa, and North Africa)

The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industrys value chain.

A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period.

Highlights of the report: A complete backdrop analysis, which includes an assessment of the parent market Important changes in market dynamics Market segmentation up to the second or third level Historical, current, and projected size of the market from the standpoint of both value and volume Reporting and evaluation of recent industry developments Market shares and strategies of key players Emerging niche segments and regional markets An objective assessment of the trajectory of the market Recommendations to companies for strengthening their foothold in the market

Note:Although care has been taken to maintain the highest levels of accuracy in TMRs reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Cellular Therapy in Oncology Market: Strategic Analysis to Understand the Competitive Outlook of the Industry, 2017 2025 - Online News Guru

San Jose, California (PRWEB) March 25, 2014

Follow us on LinkedIn High prevalence of cancer worldwide and growing number of related casualties is creating an immediate need for effective diagnosis and therapy. Despite continuous research and the development of novel drugs, cancer remains unbeatable in most cases. The discovery of Circulating Tumor Cells (CTCs) and Cancer Stem Cells (CSCs) and their molecular mechanism is forecast to play an indispensable role in the future of cancer diagnostics and treatment. CTCs are cells dispersed from the primary tumor and found in peripheral blood circulation. The detection of CTCs and their numbers present important clues on the presence of cancer and the extent of its spread within the body. Clinical applications of CTC diagnostics are currently limited with high cost being the primary limiting factor. Unmet medical needs in the field of effective screening is however expected result in continuous flow of R&D investments in CTCs and CSCs. CTC based diagnostics involve a simple blood test and is increasingly being preferred over painful bone marrow aspirations and surgical biopsies to diagnose and analyze cancer metastasis.

CTC quantification and analyses based on molecular research also provides the potential to develop personalized cancer treatment regimens, which is garnering interest among scientific communities. Better, faster, and more user-friendly methods to detect and characterize CTCs will witness increased demand in the coming years. PCR-based (nucleic acid-based) identification methods are the most effective and sensitive for CTC genetic profiling, scoring over immunocytometric (protein-based) methods for molecular characterization of CTCs. RT-PCR and qPCR are highly specific techniques that are widely used to identify and amplify CTCs. CellSearch is the only FDA-approved automated system that offer combined enrichment, staining, and scanning of CTCs.

Cancer Stem Cells (CSCs) are the bulk cells within a tumor carrying its proliferative capability. CSCs remain unaffected by cancer treatment strategies, including chemotherapy and cause tumor relapse or re-occurrence thereby creating the need for new therapeutic drugs that destroy CSCs. The technology is still under extensive research. Biotechnology and pharmaceutical companies are increasingly shifting focus to anti-cancer therapeutics that target cancer stem cells and their regenerative mechanisms.

As stated by the new market research report on Circulating Tumor Cells and Cancer Stem Cells Technologies, the United States and Europe are the largest markets worldwide. The United States remains the undisputed leader in CTC diagnostics. Asia-Pacific is forecast to emerge as the fastest growing market driven by developing healthcare infrastructure, growing patient awareness, increasing per capita healthcare spends, focus on quality healthcare services, and the urgent need for advanced cancer diagnostics.

Key players covered in the report for CTC diagnostics include Adnagen GmbH, ApoCell Inc., Biocep LTD, Biocept Inc., Biofluidica Microtechnologies LLC, Celltrafix Inc., Clearbridge Biomedics, Creatv Microtech Inc., Cynvenio Biosystems Inc., Ikonisys Inc., IVDiagnostics Inc., Janssen Diagnostics LLC, Epic Biosciences Inc., Rarecells SAS, Screencell, Stemcell Technologies Inc. Market participants in CSC research include Alchemia Limited, Amgen Inc., Exelixis Inc., Formula Pharmaceuticals, GlaxoSmithKline Plc, Geron Corp, Infinity Pharmaceuticals, Kalobios Pharmaceuticals Inc., Novartis AG, OncoMed Pharmaceuticals Inc., Roche Diagnostics, and Verastem Inc., among others.

The research report titled Circulating Tumor Cells and Cancer Stem Cells Technologies: A Global Strategic Business Report announced by Global Industry Analysts Inc., provides a comprehensive review of market trends, drivers, key issues and challenges. The study also provides insights into CTC biology and CTC detection technologies, including CellSearch, ISET, CTC Chip, FAST, FISH, etc. The report provides market estimates and projections for CTC Diagnostics for all major geographic markets including the United States, Canada, Japan, Europe (France, Germany, Italy, UK, Spain, Russia, and Rest of Europe), Asia-Pacific, and Rest of World. Exclusive coverage is presented on Cancer Stem Cells biology, Surface Markers, Signaling Pathways, and Pipeline drugs.

For more details about this comprehensive market research report, please visit http://www.strategyr.com/Circulating_Tumor_Cells_CTCs_and_Cancer_Stem_Cells_CSCs_Technologies_Market_Report.asp

About Global Industry Analysts, Inc. Global Industry Analysts, Inc., (GIA) is a leading publisher of off-the-shelf market research. Founded in 1987, the company currently employs over 800 people worldwide. Annually, GIA publishes more than 1300 full-scale research reports and analyzes 40,000+ market and technology trends while monitoring more than 126,000 Companies worldwide. Serving over 9500 clients in 27 countries, GIA is recognized today, as one of the world's largest and reputed market research firms.

Global Industry Analysts, Inc. Telephone: 408-528-9966 Fax: 408-528-9977 Email: press(at)StrategyR(dot)com Web Site: http://www.StrategyR.com/

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Need for Advanced Cancer Diagnostics Drives Demand for Circulating Tumor Cells & Cancer Stem Cells Technologies ...

Bloomberg

Monday Sector Leaders: Biotechnology Stocks Forbes In trading on Monday, biotechnology shares were relative leaders, up on the day by about 2.7% as a group, led by Onyx Pharmaceuticals (ONXX), trading higher by about 50.6% and Ariad Pharmaceuticals (ARIA), trading higher by about 12.6% on Monday. Biotechnology company Onyx rejects Amgen's $10 -bn takeover biddomain-B Onyx Rejects Amgen's Takeover Bid, Then Places Itself on the MarketNew York Times ETFs For an Onyx Takeover BattleFox Business Bloomberg all 215 news articles »

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WyoFile

Wyoming senators reap largesse from biotechnology firm Amgen WyoFile A California biotechnology company set to benefit by millions of dollars from a last-minute addition to the January 1 “Fiscal Cliff” bill is among the top campaign contributors to Wyoming U.S. Senators Mike Enzi and John Barrasso. Both senators were ...

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JERUSALEM--(BUSINESS WIRE)--

Gamida Cell Ltd., a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine, announced today that the first patient, enrolled in its pilot study of NiCord as an investigational treatment for sickle cell disease (SCD), has been transplanted at Duke University in North Carolina.

Gamida Cell CEO Dr. Yael Margolin said, The NiCord study is a first step in broadening Gamida Cells pipeline of cell therapies to treat patients who suffer from severe non-malignant diseases with a very large unmet clinical need.

SCD is a group of inherited red blood cell disorders where red blood cells become hard and sticky and look like a C-shaped farm tool called a sickle. According to statistics, SCD affects 90,000 to 100,000 in the US alone, mainly African-Americans and Hispanic-Americans. Symptoms range in type and severity. SCD can be fatal and to date, the only cure for SCD is stem cell transplantation from a family related matched donor.

Dr. Margolin continued, Sickle cell disease can be cured with a successful bone marrow transplantation, especially from a family related fully matched donor. Most patients do not pursue this option, since they do not have the suitable donor. NiCord is intended to reverse this situation and provide a readily available cure.

NiCord is an expanded cell graft derived from an entire unit of umbilical cord blood and enriched with stem cells. NiCord was developed based on Gamida Cells proprietary NAM technology.

The official name of the study is Allogeneic Stem Cell Transplantation of NiCord, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Sickle Cell Disease. http://www.clinicaltrials.gov/ct2/show/NCT01590628?term=nicord&rank=1. A total of 10 patients, ages 2 21, will be enrolled in the NiCord study, a single center, single arm trial evaluating the safety and efficacy of transplanting NiCord together with a second un-manipulated cord blood unit in patients with SCD following myeloablative therapy. The study will also assess transplant-related mortality, event-free survival and overall survival at 100, 180 and 365 days, respectively.

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging (EMITF), Clal Biotechnology Industries (CBI.TA), Israel Healthcare Venture, Teva Pharmaceutical Industries (TEVA), Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

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First Pediatric Sickle Cell Disease Patient Receives NiCord® Stem Cell Transplantation in Gamida Cell Pilot Study at ...

The California stem cell agency faces
no easy task in trying to translate basic research findings into
something that can be used to treat patients and be sold commercially.

Elizabeth Iorns Science Exchange Photo

“One goal of scientific publication
is to share results in enough detail to allow other research teams to
reproduce them and build on them. However, many recent reports have
raised the alarm that a shocking amount of the published literature
in fields ranging from cancer biology to psychology is not
reproducible.”

“First, I think it is important to
accept that there is a crisis affecting preclinical research. Recent
studies estimate that 70% of preclinical research cannot be
reproduced. This is the research that should form the foundation upon
which new discoveries can be made to enhance health, lengthen life,
and reduce the burdens of illness and disability. The
irreproducibility of preclinical research is a significant impediment
to the achievement of these goals. To solve this problem requires
immediate and concrete action. It is not enough to make
recommendations and issue guidelines to researchers. Funders must act
to ensure they fund researchers to produce high quality reproducible
research. One such way to do so, is to reward, or require,
independent validation of results. The reproducibility initiative
provides a mechanism for independent validation, allowing the
identification of high quality reproducible research. It is vital
that funders act now to address this problem, to prevent the wasted
time and money that is currently spent funding non-reproducible
research and to prevent the erosion of public trust and support for
research.”

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Bob Klein is nearly an icon in the
history of the $3 billion California stem cell. And when he appeared
before its governing board last month and aggressively touted a $20
million grant proposal already rejected by agency reviewers, his
actions raised eyebrows.

Robert Klein Elie Dolgin/Nature photo

Irv Weissman Stanford Photo

 “He has
considerable influence with the ICOC(the CIRM governing board), and
is closely associated with biotech in the Bay Area. Even if he
doesn't make a lot of money himself from this, then he certainly has
friends who will.  Irv Weissman would be one of those friends."

"StemCells Inc has been on the
stock market for 20 years, without producing anything of value for
the investors.  The stock price has been sinking fast:  it
was 60 cents this June; last year at this time, it was around $5 a
share.   

“On July 17, when the CIRM Disease
Team Award review results became available, the stock rose from 87
cents to $1.80 – a person who could anticipate the outcome of the
CIRM applications could have made considerable money in that 24 hour
period.”

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SAN DIEGO, CA--(Marketwire -06/07/12)- Bio-Matrix Scientific Group, Inc. (BMSN) (BMSN) announced today that its wholly owned subsidiary Regen BioPharma, Inc. has executed an exclusive option agreement which grants Regen BioPharma an option to license Patent #6,821,513 which patents methods of stimulating blood production in patients with deficient stem cells. The patent, as well as data licensed with the patent, covers methods of stimulating the bone marrow to generate new blood cells. The patent and option agreement are disclosed in the Company's most recent 8K filed with the US Securities and Exchange Commission on June 6, 2012.

"The technology has broad applicability to help cancer patients recover faster following chemotherapy, as well as for recipients of bone marrow and cord blood transplants. Currently, new blood cell production is stimulated by expensive drugs such as Neupogen and Neulasta which replicate the body's growth factors but can cause side effects and rely upon the diminished recuperative powers of an immune compromised patient," stated J. Christopher Mizer, President of Regen BioPharma.

David Koos, Chairman & CEO of Bio-Matrix Scientific Group, added, "We are excited to get this therapy into the clinic. Based on peer-reviewed published animal data, it has the potential to restore immune function faster and more effectively than the existing standard of care."

The licensed technology covers the use of a naturally-occurring cell type for stimulation of bone marrow stem cells. By utilizing cells as opposed to drugs, Regen BioPharma believes it possesses a substantial advantage to existing approaches in terms of safety and economics of production. Currently the market for growth factors that stimulate blood making stem cells is more than $4.84 billion per year (www.wikinvest.com/stock/Amgen).

About Bio-Matrix Scientific Group Inc. and Regen BioPharma, Inc.:Bio-Matrix Scientific Group, Inc. (BMSN) (BMSN) is a biotechnology company focused on the development of regenerative medicine therapies and tools. The Company is focused on human therapies that address unmet medical needs. Specifically, Bio-Matrix Scientific Group Inc. is looking to increase the quality of life through therapies involving stem cell treatments. These treatments are focused in areas relating to cardiovascular, hematology, oncology and other indications.

Through Its wholly owned subsidiary, Regen BioPharma, it is the Company's goal to develop translational medicine platforms for the rapid commercialization of stem cell therapies. The Company is looking to use these translational medicine platforms to advance intellectual property licensed from entities, institutions and universities that show promise towards fulfilling the Company's goal of increased quality of life.

Disclaimer

This news release may contain forward-looking statements. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking statements. The risks and uncertainties to which forward-looking statements are subject include, but are not limited to, the effect of government regulation, competition and other material risks.

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Bio-Matrix Scientific Group's Regen BioPharma Subsidiary Executes Option Agreement to License Stem Cell Intellectual ...

JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that it has closed an internal E financing round of $10 million. All major shareholders participated.

The financing will be used to support the global commercialization of the companys lead cell therapy product, StemEx, in development as an alternative therapeutic treatment for patients with blood cancers, such as leukemia and lymphoma, who can be cured by bone marrow transplantation but do not have a matched bone marrow donor. The company is currently seeking a strategic partner to join in the global commercialization of StemEx.

The financing will also support the continued development of the companys pipeline of products, primarily the NiCord clinical trial for sickle cell disease and thalassemia.

Mr. Reuven Krupik, chairman of the board of Gamida Cell said, The investors were unanimous in their decision to reinvest, understanding the importance of bringing StemEx to market as well as maintaining the companys leadership role in the stem cell industry. Gamida Cell is a game changer.

The international, multi-center, pivotal registration, Phase III clinical trial of StemEx completed enrollment in February 2012. Clinical outcome is expected in Q4/2012. The market launch of StemEx is planned for 2013. StemEx is likely to be the first allogeneic stem cell product in the market. StemEx is being developed by the Gamida Cell-TEVA joint venture.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell said, With the continued support of our shareholders and the analysis of the clinical results of the StemEx trial just around the corner, we are now focused on submitting the BLA.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit. Competing products in development use two units. As the average cost of a cord blood unit in the U.S. is $40K, StemEx is expected to be a significantly less expensive treatment option. StemEx is also expected to be available in the market several years before any of the competing products.

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

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Gamida Cell Closes $10 Million E Financing Round Earmarked to Support the Global Commercialization of the Company’s ...

Belfast Telegraph

US biotechnology giant Amgen to invest $200m in Dún Laoghaire, Co Dublin plant ... FinFacts Ireland By Finfacts Team US biotechnology giant Amgen and the Irish Government today announced the start of a $200 million-plus expansion programme in Ireland that is expected to result in the creation of 100 jobs. The Taoiseach, Enda Kenny TD and the Tánaiste ... Amgen to create up to 100 jobs in US$200m expansionSiliconrepublic.com 100 jobs in pipeline as biotech company expands in DublinInsideireland.ie Amgen Announce Major Expansion Of Dun Laoghaire FacilityeGov monitor Irish Times -Belfast Telegraph -thejournal.ie all 84 news articles »

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Wall Street Journal

Amgen's First CEO Wall Street Journal Amgen was founded as Applied Molecular Genetics in 1980 with funding from venture capitalists amid a rush to exploit the power of gene splicing. The company worked on several products in its early years, including cloned chicken growth hormone, ... George Rathmann, Founding CEO of Amgen and Icos, Dies at 84Xconomy all 24 news articles »

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JERUSALEM--(BUSINESS WIRE)--

Gamida Cell announced today that the Gamida Cell-Teva Joint Venture (JV), equally held by Gamida Cell and Teva Pharmaceutical Industries, has enrolled the last of 100 patients in the international, multi-center, pivotal registration, Phase III clinical trial of StemEx, a cell therapy product in development as an alternative therapeutic treatment for adolescents and adults, with blood cancers such as leukemia and lymphoma, who cannot find a family related, matched bone marrow donor.

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining, non-manipulated cells from the same unit.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell, said, "The JV is planning to announce the safety and efficacy results of the Phase III StemEx trial in 2012 and to launch the product into the market in 2013. It is our hope that StemEx will provide the answer for the thousands of leukemia and lymphoma patients unable to find a matched, related bone marrow donor.”

Dr. Margolin continued, “StemEx may be the first allogeneic cell therapy to be brought to market. This is a source of pride for Gamida Cell, as it further confirms the company’s leadership as a pioneer in cell therapy. In addition to StemEx, Gamida Cell is developing a diverse pipeline of products for the treatment of cancer, hematological diseases such as sickle cell disease and thalassemia, as well as autoimmune and metabolic diseases and conditions helped by regenerative medicine.”

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The company’s pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers such as leukemia and lymphoma, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cell’s therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, which are expanded in culture. Gamida Cell was successful in translating these proprietary expansion technologies into robust and validated manufacturing processes under GMP. Gamida Cell’s current shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

Link:
The Gamida Cell-Teva Joint Venture Concludes Enrollment for the Phase III Study of StemEx®, a Cord Blood Stem Cell ...