Celgene & Acceleron's BLA for Luspatercept Accepted by FDA

Celgene Corporation CELG announced that the FDA has accepted its Biologics License Application (BLA) for pipeline candidate, luspatercept.

Luspatercept, an investigational erythroid maturation agent, is being developed for the treatment of adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia, who have ring sideroblasts and require red blood cell (RBC) transfusions, and for those with beta-thalassemia-associated anemia, who require RBC transfusions.

The candidate is being developed in collaboration with clinical-stage biopharmaceutical partner, Acceleron Pharma Inc. XLRN.

The FDA has granted Priority Review to the BLA for the treatment of beta-thalassemia and set a target action date of Dec 4, 2019. In addition, the FDA has also set a PDUFA date of Apr 4, 2020, for the evaluation of the MDS indication.

The application was submitted on the basis of data from phase III studies, MEDALIST and BELIEVE, which evaluated the ability of luspatercept to effectively treat the anemia associated with MDS and beta-thalassemia, respectively.

The Marketing Authorization Application for the same is under review in the EU. The candidate is also being evaluated in the phase III COMMANDS trial in ESA-naïve, lower-risk MDS patients, the BEYOND phase II trial for non-transfusion-dependent beta-thalassemia and a phase II trial in myelofibrosis.

Celgene also announced encouraging data from the TRANSCEND CLL 004 and TRANSCEND NHL 001 trials at the American Society of Clinical Oncology (ASCO) annual meeting in Chicago in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL). These trials evaluated anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, lisocabtagene maraleucel (liso-cel; JCAR017).

The data included safety and efficacy findings from 23 patients, who received liso-cel infusion at one of two dose levels — 50 × 106 or 100 × 106 total CAR-positive T cells — following lymphodepleting chemotherapy. All patients were previously treated with Johnson & Johnson’s JNJ Imbruvica. The best overall response rate was 82%, with 46% of patients achieving complete remission with or without complete blood count recovery (CR/CRi).

Shares of Celgene have surged 49.2% year to date compared with the industry’s growth of 0.9%.

 

The company has a robust pipeline. Celgene, which is set to be acquired by Bristol-Myers Squibb Company BMY, is focused on the next cycle of innovation with five late-stage candidates — ozanimod, fedratinib, luspatercept, liso-cel and bb2121. It also plans to evaluate fedratinib in combination with luspatercept.

Per the terms of the acquisition, Celgene shareholders will receive $50 plus one share of Bristol-Myers and one tradeable Contingent Value Right, which will entitle the holder to receive a cash payment of $9.00 upon the achievement of FDA approval of three candidates (ozanimod, liso-cel and bb2121) within the specified time periods.

Zacks Rank

Celgene currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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