Acceleron stop development of ACE-083 for FSHD

Published Date
17/09/2019
Category
Research
Orange box stating: Breaking news research

Acceleron Pharma has announced topline results from its phase 2 trial testing ACE-083 in adults with facioscapulohumeral muscular dystrophy (FSHD). Although the drug increased the size of the muscles it was injected into, this did not translate into a clinical benefit i.e. there was no improvement in muscle strength or function. Unfortunately this means that Acceleron is discontinuing development of ACE-083 for FSHD. Although this is disappointing news, the learnings from this trial are very valuable to the muscular dystrophy field and will help in designing future trials for FSHD.

For more information, read Acceleron’s press release.

 

Previous news related to this story:

13 July 2018

Acceleron Pharma has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ACE-083 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This designation gives Acceleron certain financial benefits that will help to lower the cost of developing ACE-083.

ACE-083 is currently being evaluated in a Phase 2 trial – the company recently presented positive preliminary results from the ongoing trial.

For more information, please read Acceleron’s press release.

1 May 2018

Acceleron Pharma has announced the US Food and Drug Administration (FDA) has granted Fast Track Designation to ACE-083, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This status will help speed up the development and regulatory review process of the drug.

ACE-083 is currently being evaluated in a Phase 2 trial in individuals with FSHD.

For more information, please read Acceleron’s press release.

20 September 2017

Acceleron Pharma has released an update on its neuromuscular programme. It is currently developing two drugs that aim to increase muscle mass by blocking myostatin. ACE-083 is currently being tested in people with facioscapulohumeral dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) in phase 2 clinical trials. Preliminary results from the FSHD trial are expected in late 2017. ACE-2494, which works in a similar way to ACE-083 but is administered systemically, has shown promising results in preclinical studies.  A phase 1 study testing ACE-2494 is expected to be initiated later this year.

For more information, please read Acceleron Pharma’s press release.

19 July 2016

The pharmaceutical company, Acceleron, recently announced the results of its phase I clinical trial testing ACE-083 in healthy people. ACE-083 is an investigational protein therapeutic that binds and blocks the action of a family of proteins that negatively regulate muscle growth (including the myostatin protein).

The trial results showed that ACE-083 was safe and significantly increased the volume of the muscle that it was injected into. According to their press release, Acceleron intends to advance ACE-083 into a phase II clinical trial for people with facioscapulohumeral muscular dystrophy (FSHD).

To find out more about the latest research into FSHD, please contact our Research Line at research@musculardystrophyuk.org or call 020 7803 4813.

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