Jazz to Buy Actio Biosciences in $820 Million Deal Focused on Rare Childhood Epilepsy

Rare Daily Staff

Jazz Pharmaceuticals said it will acquire Actio Biosciences for $820 million upfront, adding an experimental treatment for a rare genetic epilepsy with no U.S. Food and Drug Administration-approved therapy.

The agreement includes up to $500 million in future payments tied to regulatory approval and sales milestones. The companies expect the transaction to close by the fourth quarter of 2026, subject to customary closing conditions. Jazz plans to fund the acquisition with cash on hand and existing financing facilities.

At the center of the deal is ABS-1230, an oral small-molecule medicine designed to treat KCNT1-related epilepsy, a severe genetic condition that often begins in infancy. The disease can cause dozens or even hundreds of seizures a day, many of which do not respond to existing antiseizure medications.

KCNT1-related epilepsy is an early-onset developmental and epileptic encephalopathy driven by gain-of-function variants in the KCNT1 gene. Infants and children with the condition experience severe, frequent seizures that are often resistant to antiseizure medications and may have profound developmental delays and intellectual disabilities. Some patients do not survive into adulthood.

ABS-1230 is designed to block the overactive KCNT1 ion channel, a protein that helps regulate electrical signaling in the brain. In an early clinical proof-of-concept trial, children with the condition experienced meaningful seizure reductions, according to the companies. The treatment is now being evaluated in the Phase 1b/2a KYRON study, which is assessing safety, tolerability, pharmacokinetics, seizure outcomes and neurodevelopment.

The FDA has granted ABS-1230 Fast Track, Orphan Drug and Rare Pediatric Disease designations. The candidate has also been accepted into the agency’s Rare Disease Evidence Principles program, an initiative intended to facilitate the development of therapies for ultra-rare conditions. These designations and programs do not guarantee approval, and ABS-1230 remains investigational.

“This acquisition represents a highly strategic expansion of our rare epilepsy portfolio,” Jazz CEO Renee Gala said, pointing to the company’s existing epilepsy medicine, Epidiolex.

As part of the transaction, Actio will spin out a separate private company that will retain certain management, employees and assets, including ABS-0871, an experimental treatment for Charcot-Marie-Tooth disease type 2C and related neuromuscular disorders. Jazz will receive a minority ownership stake in the new company.

“The acquisition of ABS-1230 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,” said Renee Gala, CEO of Jazz. “The emerging clinical profile of ABS-1230 is highly encouraging, and we look forward to closing the proposed transaction and collaborating with our new Actio Biosciences colleagues to address an urgent patient need.”

Photo: Renee Gala, CEO of Jazz Pharmacueticals