Acceleron Pharma has discovered a novel investigational agent ‘sotatercept’ for the treatment of patients with Pulmonary Arterial Hypertension (PAH).
PAH may be a rare and chronic, rapidly progressing disorder characterised by the constriction of small pulmonary arteries and elevated vital signs within the circulation.
PAH leads to significant strain on the heart, often resulting in limited physical activity, heart failure, and reduced life expectancy.
Sotatercept is an investigational compound that works to counteract the abnormal bone morphogenic protein (BMP) signaling within the lungs, believed to be a critical driver of the vascular remodeling processes that contribute to PAH development and progression. It does so by binding and trapping proteins of the transforming growth factor-beta (TGF-beta) family — which control numerous aspects and effects of cell functions — especially those directly involved in the BMP pathway.
By restoring the balance of BMP signaling, sotatercept is believed to improve the health of pulmonary arteries and reduce pulmonary arterial pressure, ultimately lowering the risk of heart failure.
Sotatercept reversed pulmonary vessel muscularisation and improved indicators of right heart failure, in preclinical studies of PAH.
The FDA’s Breakthrough Therapy designation is given to medications that have the potential to provide significant advantages over currently available options. It is intended to accelerate the development, review, and approval of treatments for serious or life-threatening conditions.
In patients with PAH, the current top line analysis of the PULSAR Phase 2 trial of sotatercept, revealed the trial met the main as well as key and other secondary endpoints, with unfavorable events consistent with previously published data on sotatercept in other diseases.