PRINCETON -
'This NEJM publication recognizes the importance and robustness of the MEDALIST study, as well as the impact and potential clinical benefit of Reblozyl in MDS patients who have ring sideroblasts,' said
MEDALIST achieved a statistically significant improvement in the primary endpoint of red blood cell transfusion independence (RBC-TI) for 8 or more weeks during the first 24 weeks of the study. The study also met the key secondary endpoint of RBC-TI for 12 or more weeks during the first 24 or 48 weeks of the study, as well as the additional secondary endpoint of hematologic improvement-erythroid (HI-E) for 8 or more weeks as assessed by
The most common treatment-emergent adverse events (TEAEs) of any grade in greater than 10% of patients in either the treatment or placebo arm were fatigue, diarrhea, asthenia, nausea, dizziness, and back pain. TEAEs of Grade 3 or 4 were reported in 42.5% (65/153) of patients receiving luspatercept-aamt and 44.7% (34/76) of patients receiving placebo. Progression to acute myeloid leukemia (AML) occurred in three patients (2.0%) receiving luspatercept-aamt and one patient (1.3%) receiving placebo. Five patients receiving luspatercept-aamt (3.3%) and four patients receiving placebo (5.3%) experienced one or more TEAE that resulted in death.1
Results from MEDALIST were initially presented during the Plenary Scientific Session at the 60th
'It is truly an honor to see work that began in Acceleron laboratories more than a decade ago and advanced successfully through a collaboration with
The
In
About MEDALIST
MEDALIST is a phase 3, randomized, double blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept in patients with IPSS-R-defined very low-, low-, or intermediate-risk non-del(5q) myelodysplastic syndromes (MDS). All patients were red blood cell transfusion-dependent and were either refractory or intolerant to prior
At
Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.
About Reblozyl (luspatercept-aamt)
Reblozyl is an erythroid maturation agent that promoted late-stage red blood cell maturation in animal models.2
Additional Clinical Investigation
A phase 2 trial (BEYOND) in adult patients with non-transfusion-dependent beta thalassemia3; a phase 2 trial in pediatric patients with transfusion-dependent beta thalassemia4; a phase 3 trial (COMMANDS) in
Reblozyl has not been approved as safe and effective for use in patients with MDS or myelofibrosis in any country.
Indication
REBLOZYL is indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions
REBLOZYL is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia
About
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, Acceleron and its global collaboration partner,
Cautionary Statement Regarding Forward-Looking Statements
This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that future study results will be consistent with the results to date, that Reblozyl (luspatercept-aamt) will be successfully commercialized for the indication for which it is currently approved, that Reblozyl may not achieve its primary study endpoints or receive regulatory approval for the additional indications described in this release in the currently anticipated timeline or at all and, if approved, whether such product candidate for such additional indications described in this release will be commercially successful. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect
Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's compounds, the expected timing for reporting of data from ongoing clinical trials, and the potential of REBLOZYL (luspatercept-aamt) as a therapeutic drug. The words 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'goal,' 'intend,' 'may,' 'plan,' 'potential,' 'project,' 'should,' 'target,' 'will,' 'would,' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, that preclinical testing of Acceleron's compounds and data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that the results of any clinical trials may not be predictive of the results or success of other clinical trials, that regulatory approval of Acceleron's compounds in one indication or country may not be predictive of approval in another indication or country, that the development of Acceleron's compounds will take longer and/or cost more than planned, that Acceleron or its collaboration partner, BMS, will be unable to successfully complete the clinical development of Acceleron's compounds, that Acceleron or BMS may be delayed in initiating, enrolling or completing any clinical trials, and that Acceleron's compounds will not receive regulatory approval or become commercially successful products. These and other risks and uncertainties are identified under the heading 'Risk Factors' included in Acceleron's most recent Annual Report on Form 10-K, and other filings that Acceleron has made and may make with the
The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.
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