Acceleron Announces Initiation of PULSAR Phase 2 Trial of Sotatercept in Pulmonary Arterial Hypertension
PULSAR trial expands Acceleron’s pipeline into pulmonary disease
“There is a real need for new treatment options that have the potential
to improve survival for PAH patients,” said
“The initiation of the PULSAR trial is an important milestone in our
newly expanded research and development efforts in pulmonary disease,”
The Company plans to report preliminary results from the 6-month primary treatment period of the PULSAR Phase 2 trial in the first half of 2020.
About the PULSAR Trial
The PULSAR Phase 2 trial is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of sotatercept in PAH patients. The primary endpoint of the trial is the change from baseline in pulmonary vascular resistance (PVR) over a 24-week treatment period. The key secondary endpoint is change from baseline in six-minute walk distance (6MWD). A total of 90 patients will be randomized 1:1:1 into three treatment arms with standard-of-care vasodilator therapies in combination with sotatercept or placebo. Following the 6-month double-blind treatment period, participants in the trial will be eligible to enroll into the 18-month extension period.
For additional information about this clinical trial, please visit clinicaltrials.gov, identifier NCT03496207.
Sotatercept acts as a ligand trap for members of the transforming growth factor-beta superfamily, including those directly involved in the BMP pathway proven critical for maintaining healthy pulmonary vasculature. In multiple preclinical studies in PAH, sotatercept significantly decreased pulmonary vessel muscularization, improved pulmonary arterial pressures, and decreased indicators of right heart failure. Sotatercept is currently being evaluated in the PULSAR Phase 2 trial in PAH.
About Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial Hypertension (PAH) is a rare, chronic, and rapidly progressing disorder characterized by the narrowing of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. PAH results in significant and progressive strain on the right side of 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 primarily 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 all forms of the disease.
Acceleron is a
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
This press release contains forward-looking statements about the Company's strategy, future plans and prospects, including statements regarding the development of the Company's compounds, the timeline for clinical development and regulatory approval of the Company’s compounds and the expected timing for reporting of data from ongoing clinical trials. The words "anticipate," "believe," "could," "estimate," "expect," “goal,” "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Actual results could differ materially from those included in the
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including, but not limited to, that preclinical testing of the Company's
compounds and data from clinical trials may not be predictive of the
results or success of ongoing or later clinical trials, that the
development of the Company's compounds will take longer and/or cost more
than planned, that the Company or its collaboration partner,
The forward-looking statements contained in this press release are based on management’s current views, plans, estimates, assumptions and projections with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.
Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice President, Investor Relations and Corporate Communications
Candice Ellis, 617-649-9226
Manager, Investor Relations and Corporate Communications
Matt Fearer, 617-301-9557
Director, Corporate Communications