A review recently published in Pulmonary Circulation brings together academic and industry researchers (via the PVRI Innovative Drug Discovery Initiative) to consolidate current knowledge on activin signaling inhibitors (ASIs), the role of specific Bone morphogenetic protein (BMP) ligands, efficacy/safety data, and considerations for developing next-generation activin signaling inhibitors.
Key points:
- Sotatercept is the first US Food and Drug Administration (FDA)-approved activin signaling inhibitor for pulmonary arterial hypertension —a fusion protein that binds circulating TGF-β superfamily ligands (activins A/B, GDF8, GDF11, some BMPs), showing efficacy across the disease’s risk spectrum
- Other approved activin signaling inhibitors were originally developed for blood disorders (anemias, cytopenias in β-thalassemia and myelodysplastic syndromes/neoplasms).
Open questions remain about: the basic biology of how approved activin signaling inhibitors work, short- and long-term side effects, and whether they could help other forms of pulmonary hypertension beyond pulmonary arterial hypertension.
Read more at this link on Pulmonary Circulation
Citation
S. Rajagopal, J. Abueg, K. M. Hart, et al., “ Activin Signaling Inhibitors in Pulmonary Hypertension: A State-of-the-Art Review,” Pulmonary Circulation 16 (2026): e70353. https://doi.org/10.1002/pul2.70353.