January 29, 2013

Boston Business Journal

Acceleron begins Phase 2 trial of anemia treatment, earns $10M payment

 

Acceleron Pharma Inc. has earned a $10 million milestone payment for beginning a Phase 2 study of its drug candidate intended to treat anemia in patients with myelodysplastic syndromes (MDS), malignancies of the bone marrow that often result in low levels of blood cells.

In a statement Tuesday, the 85-employee Cambridge company said that the study of ACE-536, which it’s developing in partnership with New Jersey-based Celgene Corp. (Nasdaq: CELG), will test the safety, tolerability and efficacy of ACE-536 in patients with low-risk or intermediate-1 risk MDS. More than 10,000 people are diagnosed with MDS in the United States each year, according to the National Cancer Institute.

Steve Ertel, chief business officer for Acceleron, told Mass High Tech that the study will last about a year, and the goal is to enroll about 30 patients. Acceleron could receive up to $200 million in further milestones from Celgene for the ACE-536 program.

Ertel said that ACE-536, like the other five candidates in the biotech’s pipeline, is based on the transforming growth factor beta protein superfamily, and works by regulating the growth and repair of various tissue. Two of those candidates - ACE-536 as well as ACE-011, also known as Sotatercept, a potential treatment for the treatment of anemia caused by beta-thalassemia, myelodysplastic syndrome, diamond blackfan and chronic kidney disease - are being developed with Celgene, while another one - ACE-031, for Duchenne muscular dystrophy - is in development with Shire.

Ertel said that with four candidates in Phase 2 trials, the company doesn’t know which one is likely to reach approval first. “Our strategy is not to have favorites, and to treat them all the same,” he said. He said that any approval is likely still at least a couple years away.

The nine-year old company is privately held, and has raised $300 million to date. Of that, $100 million is from venture capital firms including from Advanced Technology Ventures, Avalon Ventures, Bessemer Ventures, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. Another $200 million has come from various partnerships with companies including Alkermes and Celgene, he said.