Acceleron ditches midstage drug after flop

Fail sign
(Nick Youngson/CC BY-SA 3.0/Alpha Stock Images)

Acceleron Pharma saw its shares in the red Monday night after one of its key pipeline drugs failed.

The experimental med was being tested in a phase 2 trial in patients with Charcot-Marie-Tooth disease (CMT), a group of inherited conditions that damage the peripheral nerves. In a brief update, Acceleron said ACE-083 “did not demonstrate functional improvement” in the study and that it was now “discontinuing development” of the therapy.

Drilling down into the specifics of this failure, the Cambridge, Massachusetts-based company said that while its drug did give a statistically significant increase in mean total muscle volume, the trial’s primary endpoint, that increase did not translate to statistically significant improvements in any of the functional or quality of life secondary endpoints when compared to a dummy treatment.

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“Unfortunately, over the course of multiple clinical trials, our myostatin-plus hypothesis has not resulted in the functional outcomes necessary to provide clinically meaningful benefits for patients with neuromuscular diseases,” said Habib Dable, president and CEO of Acceleron.

“We wish to thank all of the patients, families, caregivers, and investigators for their support and participation in this research. I also want to acknowledge our team’s hard work and commitment to executing robust Phase 2 trials that have provided us the data necessary to make the difficult but informed investment decision to discontinue the program.”

Dable added: “We will now focus our resources on two main disease areas of hematology with Reblozyl in anemia and pulmonary with sotatercept in PAH [which recently got a trial win], along with our ongoing preclinical efforts in TGF-beta protein superfamily-based discovery and research.”

Shares in the company were down more than 6% on the news.

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