Acceleron Announces Preclinical Presentations on Sotatercept in Pulmonary Arterial Hypertension at the 2018 American Heart Association Scientific Session

Acceleron Pharma Inc. XLRN, a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced plans to deliver three preclinical presentations on sotatercept in Pulmonary Arterial Hypertension (PAH) at the 2018 American Heart Association (AHA) Scientific Session in Chicago, IL, November 10-12, 2018.

"The preclinical research we'll be presenting at AHA describes sotatercept's ability to exert positive effects on key measures of disease activity—including pulmonary vascular remodeling, pulmonary arterial pressures, and ventricular hypertrophy—in well-established animal models of PAH," said Ravi Kumar, Chief Scientific Officer of Acceleron. "Collectively, these findings support our belief that sotatercept could not only improve overall pulmonary and cardiac function but may also have disease-modifying potential."

Presentations
Title:			ActRIIA-Fc Rebalances Activin/GDF and BMP9 Signaling to Attenuate Experimental Pulmonary Hypertension (Abstract: 320)
Session:			Cournand and Comroe Young Investigator Award Competition
Date:			Saturday, November 10th
Time:			2:45 - 2:55 p.m. CST (McCormick Place, S106a)
Title:			Sotatercept for rebalancing BMP/TGF-beta/activin signaling in PAH
Session:			Novel Therapies for PAH: The next generation of Clinical Trials
Date:			Sunday, November 11th
Time:			9:45 - 9:55 a.m. CST (McCormick Place, N230a)
Title:			RAP-011, a Murine Ortholog of ActRIIA-Fc (Sotatercept), Improves Pulmonary Hemodynamics and Restores Right Ventricular Structure and Function in a Preclinical Model of Severe Angio-Obliterative Pulmonary Arterial Hypertension (Abstract: 16179)
Session:			New Insights into Right Ventricular Failure and Endothelial Dysfunction in Pulmonary Hypertension
Date:			Sunday, November 11th
Time:			10:30 – 11:45 a.m. CST (McCormick Place, Zone 4)

The presentations will be available in the "Science" section on Acceleron's website, www.acceleronpharma.com, immediately following their presentations at the conference. Acceleron will also host and webcast a PAH Research and Development Deep Dive event on Friday, November 16, 2018.

Sotatercept is an investigational product that is not approved for any use in any country.

About Sotatercept

Sotatercept is a ligand trap that is believed to increase BMPR2 signaling—the key molecular driver in all forms of PAH— through its Activin+ approach of inhibiting multiple TGF-beta proteins. In multiple pre-clinical and nonclinical studies, sotatercept demonstrated the ability to attenuate pulmonary vascular muscularization, proliferation, and right ventricular dysfunction. Sotatercept is currently being evaluated in the PULSAR Phase 2 trial in PAH. For more information, please visit www.clinicaltrials.gov. Acceleron is also planning to initiate an exploratory PAH study known as SPECTRA.

About PAH

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.

About Acceleron

Acceleron is a Cambridge-based, 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 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, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular franchise with two distinct Myostatin+ agents, ACE-083 and ACE-2494, and a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.

For more information, please visit www.acceleronpharma.com. Follow Acceleron on social media: @AcceleronPharma and LinkedIn.

Forward-Looking Statements

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 forward-looking statements due to various risks and uncertainties, 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, Celgene, will be unable to successfully complete the clinical development of the Company's compounds, that the Company or Celgene may be delayed in initiating, enrolling or completing any clinical trials, and that the Company's compounds will not receive regulatory approval or become commercially successful products. These and other risks and uncertainties are identified under the heading "Risk Factors" included in the Company's most recent Annual Report on Form 10-K, and other filings that the Company has made and may make with the SEC in the future.

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