Acceleron Receives FDA Orphan Drug Designation for Sotatercept in Pulmonary Arterial Hypertension
“We’re pleased that the
Orphan designation is granted by the
Sotatercept is being evaluated in two Phase 2 trials in patients with PAH: the PULSAR trial, which completed its target enrollment in June of this year and the SPECTRA exploratory trial, which is currently enrolling. The Company expects to report top-line results from the PULSAR trial during the first quarter of 2020.
Sotatercept is an investigational therapy that is not approved for any use in any country.
Sotatercept is an investigational agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR2 signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure. Sotatercept is currently being evaluated in the PULSAR and SPECTRA Phase 2 trials in PAH. For more information, please visit www.clinicaltrials.gov.
PAH is 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. The 5-year survival rate for patients with PAH is approximately 57%. Available therapies generally act by promoting the dilation of pulmonary vessels without addressing the underlying cause of the disease. As a result, PAH often progresses rapidly for many patients despite standard of care treatment. A growing body of research has implicated imbalances in BMP and TGF-beta signaling as a primary driver of PAH in familial, idiopathic, and acquired forms of the disease.
Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner,
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Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice President, Investor Relations and Corporate Communications
Ed Joyce, 617-649-9242
Director, Investor Relations
Matt Fearer, 617-301-9557
Director, Corporate Communications