Jazz Pharmaceuticals announced on Aug. 10 that it will acquire Actio Biosciences for $820 million in cash, aiming to obtain the California biotech’s lead candidate for a rare form of epilepsy. The agreement also includes up to $500 million in potential milestone payments, with the transaction expected to close by the end of this year.
Through this acquisition, Jazz will gain access to ABS-1230, a clinical-stage small molecule intended to treat KCNT1+ epilepsy, a genetic disorder affecting approximately 2,500 Americans. Patients with KCNT1+ epilepsy often experience frequent seizures from infancy and currently have no FDA-approved therapies available. According to the companies, existing antiseizure medications offer limited benefit.
In an early proof-of-concept trial, ABS-1230 was associated with meaningful seizure reductions in children diagnosed with KCNT1 epilepsy. The ongoing Phase 1b/2a KYRON trial is serving as the registrational study for ABS-1230 and has received Fast Track, Rare Pediatric Disease, and Orphan Drug designations from the Food and Drug Administration. The drug is also part of the FDA’s new Rare Disease Evidence Principles program focused on ultra-rare disease therapies.
After completion of the deal, Actio Biosciences will spin out into a privately held company retaining its remaining assets. Jazz will hold a minority stake in this spinout entity, which plans to focus on developing ABS-0871—a TRPV4 inhibitor—for Charcot-Marie-Tooth disease type 2C.
Renee Gala, CEO of Jazz Pharmaceuticals, said Monday that the purchase “represents a highly strategic expansion of our rare epilepsy portfolio, building upon the global success of Epidiolex and deepening our leadership in rare and severe epilepsies.” David Goldstein, CEO of Actio Biosciences, said, “Guided by our expertise in genetics, we set out to leverage a deep understanding of disease biology to develop highly effective targeted therapies for rare diseases with high unmet need...ABS-1230 is that vision personified, and we are thrilled to partner with Jazz on the next leg of the journey to bring this important medicine to patients.”
