Actio Biosciences' lead clinical asset is ABS-1230, a novel, first-in-class small molecule precision therapy KCNT1 ion channel inhibitor. KCNT1+ epilepsy is a rare genetic developmental and epileptic encephalopathy (DEE) affecting approximately 2,500 patients in the United States. Patients suffering from KCNT1+ epilepsy typically endure a profound seizure burden, with most experiencing dozens to hundreds of episodes daily that remain highly resistant to antiseizure medications. Approximately 80% of individuals with KCNT1+ epilepsy experience disease onset during infancy; many of these children never achieve fundamental developmental milestones, such as walking or speaking, and tragically, some do not survive into adulthood. For those with later-onset disease, the condition typically manifests as disruptive nocturnal seizures, often accompanied by significant cognitive and psychiatric comorbidities.

There are currently no U.S. Food and Drug Administration (FDA) approved therapies for KCNT1+ epilepsy. ABS-1230 recently demonstrated meaningful seizure reductions in an early clinical proof-of-concept trial in children with KCNT1 epilepsy. The ongoing Phase 1b/2a KYRON trial is designed to serve as the registrational study to support a new drug application submission in the U.S. In addition to having FDA Fast Track, Rare Pediatric Disease and Orphan Drug Product designations, ABS-1230 was accepted into the FDA's new Rare Disease Evidence Principles (RDEP) program which aims to facilitate rapid development of ultra-rare disease therapies. 

"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, president and chief executive officer, Jazz Pharmaceuticals. "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. Together, we are committed to working alongside the epilepsy patient community and regulators to bring this important medicine to children and families, who currently have no treatment options for a devastating disease."

"We chose to partner with Jazz because they combine a purpose-led culture with a world-class development engine and the commercial scale to ensure ABS-1230 is brought to patients as quickly and efficiently as possible," said David Goldstein, Ph.D., chief executive officer, Actio Biosciences. "We started Actio with one goal in mind: to bring meaningful therapies to those who need them most. 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."

Key Highlights

  • Strengthens Jazz's leadership in rare epilepsy. Builds on durable Epidiolex franchise and growing epilepsy pipeline.
  • Meaningful early clinical data support developing ABS-1230 for KCNT1+ epilepsy, an ultra-rare disease with no approved therapies.
  • ABS-1230 has been accepted into FDA's RDEP program, which accelerates ultra-rare disease development.
  • Opportunity for development of ABS-1230 for additional genetic epilepsy indications. 

Transaction Terms

Under the terms of 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). The new, independent private company will be funded by existing investors, with Jazz 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 TRPV4 inhibitor, ABS-0871, for Charcot-Marie-Tooth type 2C and other early-stage programs. With the focus on rare disease, this investment in the new entity represents a strong strategic fit for Jazz's long-term strategy.

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. Jazz will fund the transaction through a combination of cash on hand and drawing on existing financing facilities.

Closing Conditions

The transaction is subject to customary closing conditions.