Acceleron Pharma's shares established a new 52-week high after the company reported that sotatercept achieved both primary and secondary endpoints in the Phase 2 PULSAR study in patients with pulmonary arterial hypertension.
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%).
Disclosure:
1) Stephen Hytha compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.
2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.
3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the interview or the decision to write an article until three business days after the publication of the interview or article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.
6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.