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Bristol Myers & Acceleron Obtain EC Approval for Reblozyl

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Bristol Myers Squibb (BMY - Free Report) and partner Acceleron Pharma Inc. (XLRN - Free Report) announced that the European Commission (EC) has approved Reblozyl (luspatercept) for two indications — the treatment of adult patients with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy, and those with transfusion-dependent anemia associated with beta thalassemia.   

The drug supposedly regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions.

Per the companies, Reblozyl is the first and only erythroid maturation agent to be approved in the European Union (EU), representing a new class of therapy.
The approval was based on positive data from the phase III MEDALIST and BELIEVE studies, which evaluated the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

Approximately, 25 million blood transfusions occur every year in the EU, some of which are needed by patients with anemia due to hematologic diseases like MDS and beta thalassemia.

The drug is already approved in the United States for the treatment of anemia in adult patients with beta thalassemia who require regular RBC transfusions, and anemia failing an erythropoiesis stimulating agent and requiring 2 or more RBC units over 8 weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

Approval in other geographies should boost sales of the drug as well as Acceleron’s growth prospects.

Bristol Myers is currently conducting a phase II study, BEYOND, on luspatercept-aamt in non-transfusion-dependent beta-thalassemia patients. The company expects to release preliminary top-line results by the end of 2020. It is also conducting a phase III study, COMMANDS, in first-line, lower-risk MDS patients. In myelofibrosis, Bristol Myers is conducting a phase II study in patients with myelofibrosis-associated anemia. Initial results from this study showed that luspatercept-aamt improved anemia in patients receiving and not receiving RBC transfusions with more profound effects in patients treated with Incyte’s (INCY - Free Report) Jakafi. Both companies plan to initiate a phase III study, INDEPENDENCE, in patients with myelofibrosis-associated anemia who are being treated with JAK inhibitor therapy and require RBC transfusions.

The drug was added to Bristol Myers’ portfolio following Celgene’s acquisition in November 2019 and the label expansion of the drug is likely to drive sales. Bristol Myers is responsible for paying 100% of the development costs for all studies on luspatercept-aamt.

The company’s shares have dropped 9% so far this year compared with the industry’s decline of 2.4%.


However, competition can be stiff from Novartis's (NVS - Free Report) oral iron-chelating agents — Exjade and Jadenu.

While Bristol Myers carries a Zacks Rank #2 (Buy), Acceleron currently has a Zacks Rank #3 (Hold). You can see the complete list of (Buy) today’s Zacks #1 Rank (Strong Buy) stocks here.

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