After U.S. markets closed for trading yesterday afternoon, biopharmaceutical company Acceleron Pharma Inc. (XLRN:NASDAQ), which endeavors to discover and develop TGF-beta superfamily therapeutics to treat serious and rare diseases, announced that "the PULSAR Phase 2 trial of sotatercept met its primary and key secondary endpoints in patients with pulmonary arterial hypertension (PAH)."
The firm advised that the PULSAR Phase 2 trial is a randomized, double-blind study of 106 people designed to evaluate the efficacy and safety of sotatercept in patients with PAH. The company reported that in patients on stable background PAH-specific therapies, sotatercept successfully met both the trial's primary endpoint of the baseline change in pulmonary vascular resistance over a 24-week treatment period and the key secondary endpoint of six-minute walk distance, a measure of functional capacity and endurance.
The company's President and CEO Habib Dable commented, "We're thrilled to report such positive topline results from the PULSAR trial...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, commercialize sotatercept in PAH."
PULSAR clinical trial investigator and Acceleron paid consultant Dr. Marc Humbert, professor of medicine and director of the French Pulmonary Hypertension Reference Center at the Université Paris-Saclay, remarked, "Approved therapies for patients with PAH target three main pathways of endothelial cell dysfunction to primarily promote pulmonary vasodilation...As a selective ligand trap for members of the TGF-beta superfamily, sotatercept is designed to rebalance BMPR-II signaling, which is a key molecular driver of PAH. The PULSAR data demonstrate that this novel approach has the potential to provide significant benefit on top of currently available therapies."
Dr. Vallerie McLaughlin, professor of medicine and director of the Pulmonary Hypertension Program at the University of Michigan who also consulted on behalf of the firm in the trial, added, "These results are particularly impressive given the patient population, the majority of whom were on background combination therapy, including parenteral prostacyclins, had advanced hemodynamics and lengthy duration of disease...Exceptionally notable was the concordance of the effects across the prespecified subgroups. These clinically meaningful data raise the exciting possibility that sotatercept could potentially shift the treatment paradigm for patients with PAH."
The company explained that sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signaling, a key molecular PAH driver. The firm indicated that "in preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure."
The firm characterized PAH in the report as "a rare and chronic, rapidly progressing disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant strain on the heart, often leading to limited physical activity, heart failure, and reduced life expectancy."
Acceleron is a biopharmaceutical company based in Cambridge, Mass., focused on the discovery, development and commercialization of therapeutics to treat rare and serious diseases. The firm's research efforts are concentrated on harnessing the power of the TGF-beta protein superfamily, the target-rich area of biology behind the body's remarkable capacity for cellular growth and repair. The company's research is mostly centered on addressing hematologic, pulmonary and neuromuscular diseases.
Acceleron began the day with a market cap of around $2.8 billion with approximately 53.08 million shares outstanding and a short interest of about 7.4%. XLRN shares opened nearly 49% higher today at $78.74 (+$25.87, +48.93%) over yesterday's $52.87 closing price and reached a new 52-week high price this morning of $81.99. The stock has traded today between $73.51 and $81.99 per share and is currently trading at $75.66 (+$22.79, +43.11%).
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