Acceleron Announces Preclinical Results in Pulmonary Arterial Hypertension at the American Heart Association 2017 Scientific Sessions
- Preclinical results show potential first-in-class disease-modifying properties of sotatercept in pulmonary arterial hypertension -
- Company expects to initiate a Phase 2 trial in 1H 2018 -
"We are extremely encouraged by these positive preclinical results, which support our mechanistic approach for sotatercept and its potential to be a first-in-class disease-modifying therapy for patients with pulmonary arterial hypertension. We look forward to initiating a Phase 2 trial with sotatercept in PAH during the first half of 2018," said Ravi Kumar, Chief Scientific Officer for Acceleron.
The oral presentation by
- In animals treated early in disease progression, sotatercept blocked the development of pulmonary vascular remodeling and right heart failure, and prevented the increase in pulmonary arterial pressure.
- For animals treated after established disease, sotatercept achieved a marked reduction in intimal-medial thickness of pulmonary arterioles relative to untreated animals, decreased vessel muscularization, improved pulmonary arterial pressures and decreased indicators of right heart failure.
"The sotatercept preclinical results show remarkable biological
activity, particularly on the pulmonary vasculature, across multiple,
well-established animal models of PAH," said
Sotatercept is an investigational product that is not approved for use in any country.
The AHA preclinical presentation is available under the Science page of the Company's website at www.acceleronpharma.com.
Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the transforming growth factor-beta superfamily involved in remodeling and regeneration of a variety of different tissues, including the vasculature and fibrosis.
About Pulmonary Arterial Hypertension
Pulmonary Arterial Hypertension (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
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 pulmonary program with a Phase 2 trial of sotatercept planned in pulmonary arterial hypertension.
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