Phase II bump puts brakes on Acceleron

17 September 2019
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A Phase II trial flop for rare disease candidate ACE-083 has prompted Acceleron Pharma (Nasdaq: XLRN) to abandon development of the program in facioscapulohumeral muscular dystrophy (FSHD).

Acceleron said that while ACE-083 increased muscle volume in the trial, that did not translate into statistically-significant improvements in functional tests.

The announcement caused shares in the TGF-beta specialist to drop by almost a dollar on Monday. Topline results from another Phase II trial of ACE-083, in Charcot-Marie-Tooth disease, are expected at the start of next year.

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