10:34 AM
Jul 10, 2018
 |  BC Extra  |  Clinical News

Latest luspatercept readout a second win for Acceleron, Celgene

Acceleron Pharma Inc. (NASDAQ:XLRN) climbed $1.36 to $48.76 Tuesday after it and partner Celgene Corp. (NASDAQ:CELG) said luspatercept (ACE-536) met the primary endpoint in the Phase III BELIEVE study to treat beta-thalassemia. The companies are planning regulatory submissions for the candidate in the U.S. and EU in 1H19.

The results come less than two weeks after the partners said luspatercept met the primary endpoint in another Phase III trial, MEDALIST, to treat myelodysplastic syndrome (MDS). Celgene and Acceleron are also planning U.S. and EU submissions in that indication in 1H19 (see "Celgene, Acceleron Planning Submissions for Luspatercept").

With Tuesday's move, Acceleron shares are up 43% from their close at $33.98 on June 28, before the partners announced the MDS data.

For Celgene, the pair of readouts deliver a dose of good news following a series of setbacks dating back to fall 2017. In October, the company discontinued development of Phase III Crohn's disease candidate mongersen (GED-0301) and slashed its 2020 revenue guidance. Then, in February, FDA issued a refusal to file letter for ozanimod (RPC1063) to treat multiple sclerosis (see "Rocky Road").

The company expects more than $2 billion in incremental peak sales of luspatercept. Celgene licensed the candidate from Acceleron in 2011 (see "Jump-Starting Growth").

Celgene gained $0.71 to $84.56 on Tuesday. Although its shares are up 10% since closing at $76.66 on June 28, Celgene is still off 43% from its all-time high of $147.17 on Oct. 2, 2017.

BELIEVE's primary endpoint measured erythroid response, defined as a 33% reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units from weeks 13-24. Compared with placebo, luspatercept led to a "highly statistically significant improvement," the partners said. They did not release specific data, which are to be presented at a medical meeting this year.

Luspatercept also met all of BELIEVE's key secondary endpoints, the partners said. Those included 33% improvements in RBC transfusion burden from weeks 37-48; 50% reduction from weeks 13-24 as well as weeks 37-48; and mean change in transfusion burden from weeks 13-24. The partners said luspatercept's safety profile was "generally consistent" with prior results.

Luspatercept is a modified activin receptor type 2B (ACVR2B) fusion protein that inhibits several ligands in the transforming growth factor (TGF) beta superfamily.

BELIEVE tested best supportive care plus either placebo or luspatercept in adults with transfusion-dependent beta-thalassemia. The company is also conducting the Phase II BEYOND trial in non-transfusion-dependent beta-thalassemia.

The partners are preparing another Phase III trial, COMMANDS, to evaluate luspatercept as a first-line treatment for MDS. MEDALIST studied the candidate in patients refractory to, intolerant of, or ineligible for treatment with an erythropoietin-stimulating agent (ESA).

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