STAR-0871

STAR-0871 (formerly ABS-0871) is an investigational, potential first-in-class oral small-molecule inhibitor of the transient receptor potential vanilloid 4 (TRPV4) ion channel. It is being developed by Setara Biosciences, a spinout company formed following Jazz Pharmaceuticals' acquisition of Actio Biosciences. STAR-0871 is designed to selectively inhibit TRPV4, directly addressing the underlying genetic cause of Charcot-Marie-Tooth disease type 2C (CMT2C), a rare inherited neuromuscular disorder caused by gain-of-function mutations in the TRPV4 gene that over-activate the channel. The drug is also being evaluated for the treatment of overactive bladder. A Phase 1 clinical trial in healthy volunteers has been completed, and Phase 1b (THERON) and Phase 2a clinical trials are planned to initiate in 2027 for CMT2C and overactive bladder, respectively. The FDA has granted STAR-0871 Rare Pediatric Disease, Orphan Drug, and Fast Track designations.