Investigational product that acts as a reverse-remodeling agent designed to rebalance BMP/activin signaling for patients with Pulmonary Arterial Hypertension.

Sotatercept is being studied in patients with pulmonary arterial hypertension (PAH) – a rare, progressive, and life-threatening blood vessel disorder. In PAH, an imbalance in BMP and activin signaling leads to vascular remodeling. Sotatercept is a ligand trap with high selectivity for multiple members of the TGF-beta superfamily. With its believed ability to block the TGF-beta superfamily ligands which are known to be upregulated in PAH, and based on preclinical data published in Science Translational Medicine, sotatercept could promote a rebalancing of pro-proliferative activin signaling, and anti-proliferative BMP (bone morphogenetic protein) signaling, potentially reversing the remodeling caused by the disease and restoring vascular homeostasis.

The United States Food and Drug Administration (FDA) has granted Orphan Drug designation and Breakthrough Therapy designation to sotatercept for the treatment of PAH; the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to sotatercept for the treatment of PAH.

Additional clinical studies of sotatercept include the SPECTRA Phase 2 trial, which is designed to continue to assess the efficacy and safety of sotatercept in patients with PAH, and we are currently planning multiple Phase 3 trials with the potential to support a long-term vision of establishing sotatercept as a backbone therapy for patients with PAH in all stages of the disease. We recently initiated our main registrational Phase 3 trial, known as STELLAR, as well as additional Phase 3 trials, ZENITH, HYPERION, and SOTERIA.

Sotatercept, which is part of a licensing agreement with Bristol Myers Squibb, is an investigational therapy that is not approved for any use in any country.