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Acceleron rockets on positive mid-stage pulmonary arterial hypertension data

Sotatercept hit primary and all secondary endpoints


Acceleron Pharma has unveiled positive data from a phase 2 trial of sotatercept in pulmonary arterial hypertension (PAH), sending shares sky-high.

The US-based biotech company is not revealing the detailed results just yet, but has said that the PULSAR trial readout was a success, with the drug hitting both its primary endpoint and secondary endpoints.

The primary endpoint of the study – which evaluated sotatercept in 106 patients with PAH – was a significant reduction in pulmonary vascular resistance. The additional secondary endpoints included testing patient functionality, with measures such as a six-minute walking test and the WHO functional class.

“We’re thrilled to report such positive top-line results from the PULSAR trial,” said Habib Dable, president and chief executive officer of Acceleron.

“PAH is a debilitating disease of high unmet medical need, so we’re encouraged by these data that signal that sotatercept could deliver added benefit to patients. We look forward to upcoming interactions with health authorities as we plan to globally develop and, if approved, commercialise sotatercept in PAH,” he added.

The response from investors, even without the full data, was positive – shares soared by 79% on Monday when the news broke.

PAH is a rare and progressive disorder, which occurs when arteries in the lungs constrict, which forces the heart to work harder, often causing heart failure.

Aside from sotatercept, Acceleron’s most advanced drug is its Bristol-Myers Squibb-partnered blood disease treatment Reblozyl, which is under review with the FDA for approval in myelodysplastic syndromes (MDS).

The sotatercept data is of particularly important for Acceleron, as it recently had to abandon its lead candidate ACE-083 from its neuromuscular therapeutic programme.

Despite the drug hitting its primary endpoint in a phase 2 facioscapulohumeral muscular dystrophy (FSHD) trial, Acceleron took the decision to pull ACE-083 after a sift through the secondary endpoint data from the study showed that its clinical profile wasn’t strong enough to support a push into costly late-stage clinical trial testing.

The US biotech is still testing ACE-083 in a mid-stage trial in patients with Charcot-Marie-Tooth disease with results due in the first quarter of this year.

Acceleron is due to present a detailed review of the top-line results from PULSAR trial at an unspecified medical conference later this year.

Article by
Lucy Parsons

28th January 2020

From: Research



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