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Celgene Corporation CELG and partner Acceleron Pharma Inc. XLRN announced positive results from a second randomized, double-blind, multi-center clinical phase III study, BELIEVE, on pipeline candidate, luspatercept.
The trial evaluated the efficacy and safety of luspatercept, in addition to best supportive care as compared to placebo plus best supportive care in adults with transfusion-dependent beta-thalassemia.
The results show that luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response, which was defined as at least a 33% reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks (from week 13 to week 24) compared to placebo.
Moreover, luspatercept also met all key secondary endpoints of demonstrating statistically significant improvements in RBC transfusion burden from baseline of at least a 33% reduction during the period from week 37 to week 48. Treatment with the candidate also showed at least a 50% reduction during the period from week 13 to week 24 and at least a 50% reduction during the period from week 37 to week 48. It also demonstrated a mean change in transfusion burden from week 13 to week 24.
Notably, this is the second positive phase III study for the candidate. Last month, both the companies announced positive results from another phase III study, MEDALIST, wherein luspatercept met the primary and key secondary endpoints compared to placebo in patients with IPSS-R very low, low or intermediate risk myelodysplastic syndromes (MDS) with chronic anemia and refractory to, intolerant of, or ineligible for treatment with an erythropoietin-stimulating agent (ESA), ring sideroblast-positive, and require frequent RBC transfusions.
Data from both the studies will be submitted to a future medical meeting in 2018. Both the companies intend to submit regulatory applications for luspatercept in the United States and Europe in the first half of 2019.
A third study is also ongoing, BEYOND, which is evaluating luspatercept in non-transfusion dependent beta-thalassemia.
The successful development and commercialization of luspatercept bode well for Celgene as it makes desperate attempts to revamp its portfolio, given the recent pipeline setbacks. The company suffered a setback in early 2018, when it received Refusal to File letter from the FDA regarding its New Drug Application (“NDA”) for multiple sclerosis candidate, ozanimod. A late-stage study on its lead cancer drug, Revlimid, in combination with Roche Holdings’ RHHBY Rituxan failed. Consequently, shares took a hammering.
Shares of Celgene are down 19.6% in the year so far compared to a 2.7% decline for the overall biotech industry.
Nevertheless, Celgene has a few promising candidates in its pipeline, which should boost prospects. Celgene acquired Juno Therapeutics and added JCAR017 (lisocabtagene maraleucel; liso-cel) to its lymphoma pipeline. JCAR017 is a best-in-class CD19-directed CAR-T candidate, currently in a pivotal program for relapsed and/or refractory diffuse large B-cell lymphoma.
The candidate is expected to obtain regulatory approval in the United States in 2019 with potential global peak sales of approximately $3 billion. Another promising candidate is bb2121, an experimental anti-B-cell maturation antigen chimeric antigen receptor (CAR) T cell therapy in collaboration with bluebird bio, Inc. BLUE.
Celgene carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank stocks here.
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