Acceleron bails on ACE-083 after unsuccessful mid-stage study

Acceleron Pharma (NASDAQ:XLRN) has decided to terminate development of ACE-083 after a Phase 2 study in patients with  Charcot-Marie-Tooth disease, a group of inherited disorders that cause nerve damage, failed to demonstrate functional improvement despite meeting the primary endpoint of a statistically significant increase in mean total muscle volume.

The company will now focus its resources on anemia and pulmonary PAH (with sotatercept) with Reblozyl as well as ongoing preclinical work in TGF-beta protein superfamily-based discovery and research.

In September 2019, it nixed development of ACE-083 for facioscapulohumeral muscular dystrophy.

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https://seekingalpha.com/news/3549925-acceleron-bails-on-aceminus-083-after-unsuccessful-mid-stage-study