BCX9250 is a potent small-molecule inhibitor of Activin A Receptor Type 1 (ACVR1/ALK2) for fibrodysplasia ossificans progressiva
Haitao Wang, Saman Toutounchi, Robert J Pignolo, Xiaogang Cheng, Cynthia Davis Parker, Qingde Liu, Saritha Muppa, Debra Kellogg-Yelder, Kevin J Polach, Pravin L Kotian, Yarlagadda S Babu, Xilin Chen · 2026
Read BCX9250 is a potent small-molecule inhibitor of Activin A Receptor Type 1 (ACVR1/ALK2) for fibrodysplasia ossificans progressiva by Haitao Wang, Saman Toutounchi, Robert J Pignolo, Xiaogang Cheng, Cynthia Davis Parker, Qingde Liu, Saritha Muppa, Debra Kellogg-Yelder, Kevin J Polach, Pravin L Kotian, Yarlagadda S Babu, Xilin Chen (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13574280.txt.
Description
Wang and co-authors present a 2026 study on a drug candidate for an ultra-rare bone disease, fibrodysplasia ossificans progressiva. In this disease, a mutant receptor makes soft tissue turn to bone after injury or even on its own. The drug, BCX9250, is a small pill that blocks that receptor, called ALK2. The team tested it in binding assays, cells, and mouse models.
The drug bound strongly to both normal and mutant forms of the receptor. In cells carrying the disease mutation, it shut down the false signal that activin A wrongly triggers. Mouse tests covered both new bone growth and its return after removal. BioCryst developed the compound as an oral option for patients.
Catalog record
| CREATOR | Haitao Wang, Saman Toutounchi, Robert J Pignolo, Xiaogang Cheng, Cynthia Davis Parker, Qingde Liu, Saritha Muppa, Debra Kellogg-Yelder, Kevin J Polach, Pravin L Kotian, Yarlagadda S Babu, Xilin Chen |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 38 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |