Acceleron Pharma Science & Pipeline

Science & Pipeline

Sotatercept is an investigational product being studied in patients with pulmonary arterial hypertension (PAH) – a rare, progressive, and life-threatening blood vessel disorder.

Sotatercept is a ligand trap with high selectivity for multiple proteins within the TGF-beta superfamily, including activins, GDFs, and others. With its believed ability to block the TGF-beta superfamily signaling pathway, sotatercept could promote a rebalancing of BMPR-II signaling and, potentially, restore vascular homeostasis.

PAH is characterized by a thickening of the pulmonary vasculature, leading to restricted blood flow and elevated blood pressure within the pulmonary vessels. To compensate, the heart must work harder to push blood through the pulmonary arteries.

Currently approved PAH treatments target several main pathways that promote dilation of the pulmonary vessels to reduce pulmonary vascular resistance. These therapies have been used alone or in combination to improve exercise capacity and slow the progression of the disease. However, no existing therapy directly addresses the underlying cellular proliferation that leads to pulmonary arterial pressure. In PAH, signaling via the bone morphogenetic protein receptor type II (BMPR-II) is imbalanced, allowing greater influence from the adjacent activin receptor pathway, further promoting cellular proliferation and vascular remodeling.

Acceleron recently announced results from the sotatercept Phase 2 PULSAR trial in patients with PAH. We are also conducting our second Phase 2 trial, SPECTRA, which is designed to continue to assess the efficacy and safety of sotatercept in patients with PAH. The United States Food and Drug Administration (FDA) has granted Orphan Drug designation and Breakthrough Therapy designation to sotatercept for the treatment of PAH.

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


Pulmonary

Sotatercept
Phase 1Ph1Phase 2Ph2Phase 3Ph3ApprovedApp

Pulmonary

Sotatercept
 
Pulmonary Arterial Hypertension
Pulmonary Arterial Hypertension
PULSAR TrialPULSAR TrialPULSAR Trial
SPECTRA Exploratory TrialSPECTRA Exploratory TrlSPECTRA Trial
ACE-1334
Pulmonary Disease
Pulmonary Disease