Acceleron plans to launch Phase 2 FSHD trial this year

Acceleron Pharma, from
Acceleron Pharma, from
Acceleron Pharma, from

According to a Boston Business Journal story, Cambridge, Massachusetts-based biotech Acceleron Pharma has plans to test its ACE-083 compound in facioscapulohumeral muscular dystrophy patients in a phase 2 clinical trial later this year: “The company presented top-line data from a Phase 1 trial in healthy volunteers showing the drug, which is injected into the muscle, was able to increase the size of a patients thigh muscle by up to 14 percent versus placebo. CEO John Knopf described the results as “unprecedented” and said that the company plans to advance ACE-083 into a phase 2 trials in patients with facioscapulohumeral muscular dystrophy in mid-2016.”


9 responses to “Acceleron plans to launch Phase 2 FSHD trial this year”

    • Hard to say. Acceleron is planning a Phase 2 trial, which could take 1-2 years depending on how fast they can recruit participants. They then need to analyze the data and if it looks promising, make a case to the FDA to allow a Phase 3 trial, which is a larger study to prove efficacy. That could take an additional 1-2 years, and then they need to apply for FDA approval.

  1. No matter how long it takes. The mere fact that it is a priority and checked it’s a good news. By the way also we will be happy to take a part in the plan.

  2. An increase of muscle mass doesn’t mean a gain in muscle strength…
    It as been shown in trials on inhibition of myostatin if I remember well (I’m not sure…).
    What’s your idea on that subject ?
    Thank you in advance for your answer.
    Sylvie, from AFM FSH group, France

  3. There is a myostatin inhibitor trial that is further ahead. Some results available below: Keep track of the trial on the ppmd website. Yes, I know that it is being tested on DMD boys but I participated in a previous webinar and was assured it would also work on FSH.

    Location: Online Webinar – Eastern Time Zone
    Website or Map:
    Phone: 1.866.740.1260 — Access code: 9449985
    Event Type: webinar
    Organized By: PPMD
    Latest Activity: 41 minutes ago

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