To boost its rare-disease drug portfolio, Jazz Pharmaceuticals, a Dublin, Ireland-based bio/pharmaceutical company, has agreed to acquire Actio Biosciences, a San Diego, California-based clinical-stage bio/pharmaceutical company developing small molecules for rare diseases, in a $1.32-billion deal ($820 million upfront and up to $500 million in contigent consideration).  

Actio Biosciences’ lead clinical asset is ABS-1230, a small-molecule drug for treating KCNT1+ epilepsy, a developmental and epileptic encephalopathy, a group of rare, severe brain disorders starting mostly in infancy or childhood. In May (May 2026), the company initiated a Phase Ib/II clinical trial of ABS-1230 for treating KCNT1+ epilepsy. In addition, the drug was accepted into the US Food and Drug Administration’s Rare Disease Evidence Principles process, a framework designed to facilitate development of certain therapies for ultra-rare, genetically defined diseases.  

Under the merger agreement, Actio Biosciences shareholders will receive an $820-million upfront payment and up to $500 million in potential approval and sales milestones. As part of the transaction, and concurrently with closing, Actio Biosciences will spin out a new privately held entity with certain management, employees, and assets (not including ABS-1230 which remains with Actio Biosciences and is being acquired by Jazz Pharmaceuticals). The new, independent private company will be funded by existing investors, with Jazz Pharmaceuticals receiving a minority stake and certain related rights. The focus of the new company will be on genetic rare neurological diseases, which will include a clinical-stage small-molecule drug, ABS-0871, for treating Charcot-Marie-Tooth type 2C, a rare inherited nerve disorder and a form of axonal Charcot-Marie-Tooth disease, a peripheral sensorimotor neuropathy, characterized by vocal cord anomalies, impairment of respiratory muscles, and sensorineural hearing loss with distal hands and feet weakness, as well as other early-stage programs. 

The transaction has been unanimously approved by the boards of both companies and is expected to close by the fourth quarter of 2026, subject to customary closing conditions. 

Source: Jazz Pharmaceuticals