Acceleron drops a development program as #2 drug fails to spark functional benefits in patients with a rare neuromuscular ailment
Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.
Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.
Their stock $XLRN slid 7% on the news, which was fairly muted — likely as investors are looking past this effort and on to a near-term FDA decision on their lead drug, tapped as a potential blockbuster.
“As we have stated consistently, for ACE-083 to become an important new therapy for patients with FSHD, it would have to deliver a meaningful functional benefit on top of an ability to grow muscle,” said CEO Habib Dable. “Unfortunately, in this case, the data show no evidence of such a benefit and, therefore, do not support further development of ACE-083 for FSHD.”
But they aren’t finished with the drug, one of the key therapies the biotech turned to 2 years ago when their angiogenesis drug dalantercept failed in Phase II. Researchers now are waiting for Phase II data on ACE-083 in patients with Charcot-Marie-Tooth disease—a neuromuscular disorder of different pathophysiology — in Q1 2020.
Cowen’s Yaron Werber was focused on the half-full part of the glass.
We think the termination of further development is a clearing event as investors have been worrying that further development will be costly and unlikely to yield approval.
The other drug Acceleron focused on back in 2017 was luspatercept, which is partnered with Celgene and now under review for its promise to manage anemia resulting from myelodysplastic syndrome (MDS) as well as beta-thalassemia by boosting the number of red blood cells in patients. The FDA’s decision on beta-thalassemia is coming up in early December following a priority review, with high hopes of blockbuster returns once it hits the market.