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Acceleron Discontinues Mid-Stage Muscular Dystrophy Study

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Acceleron Pharma Inc. XLRN announced the discontinuation of a phase II study evaluating its pipeline candidate, ACE-083, in patients with facioscapulohumeral muscular dystrophy (FSHD). Although the candidate achieved growth in muscle volume in FSHD patients, it failed to deliver a meaningful functional benefit on top of it.

Data from the study showed that ACE-083 achieved statistically significant increase in mean total muscle volume, the study’s primary endpoint. However, the company announced that the increase in muscle volume failed to translate into improvements in functional tests of statistical significance, representing secondary endpoints. This failure resulted in the company’s decision to discontinue further development of the candidate in FSHD patients.

So far this year, Acceleron stock has gained 2.7% against the industry’s decline of 1.2%.


Meanwhile, the company continues to develop ACE-083 as a treatment for Charcot-Marie-Tooth disease, another neuromuscular disorder in a phase II study. Top-line data from this study is expected in the first quarter of 2020.

We note that the company had discontinued development of another pipeline candidate, ACE-2494 in April. The candidate was being developed in a phase I study as a potential treatment of neuromuscular disorders. Higher frequency of anti-drug antibodies generation was observed in patients, which was the primary reason behind the discontinuation of its development.

Acceleron is developing its lead pipeline candidate, luspatercept, in collaboration with Celgene for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia and myelofibrosis.

Two regulatory applications seeking approval for the candidate as a treatment for beta-thalassemia and myelodysplastic syndromes associated anemia are under review with the FDA.

Currently, the company is also developing sotatercept for the treatment of pulmonary arterial hypertension (“PAH”), a rare disease with high unmet medical need. The FDA granted Orphan Drug designation to sotatercept for PAH earlier this month.

In June, Acceleron completed enrolling patients in the PULSAR study and plans to report top-line data from the same in the first quarter of 2020. Also, the company is currently recruiting subjects in the SPECTRA exploratory study to have a better understanding of sotatercept's potential impact on PAH. Preliminary results from this study are expected in 2020.

Zacks Rank & Stocks to Consider

Acceleron currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the healthcare sector include Emmaus Life Sciences EMMA and Acorda Therapeutics ACOR, both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emmaus Life’s loss per share estimates have narrowed 17.7% for 2019 and 26.4% for 2020 over the past 60 days.

Acorda’s loss per share estimates have narrowed 25.9% for 2019 and 3.4% for 2020 over the past 60 days.

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