Jazz Pharmaceuticals and Actio Biosciences have entered into a definitive agreement under which Jazz will acquire Actio for USD 820 million in upfront consideration, along with up to USD 500 million in contingent payments, strengthening Jazz’s portfolio of treatments for rare and severe epileptic disorders.

The transaction will give Jazz access to ABS-1230, Actio Biosciences’ lead clinical-stage asset and a first-in-class small-molecule precision therapy designed to inhibit the KCNT1 ion channel. The investigational therapy is being developed for KCNT1-positive epilepsy (KCNT1+ epilepsy), a rare genetic Developmental and Epileptic Encephalopathy (DEE).

KCNT1+ epilepsy affects an estimated 2,500 patients in the US and is characterised by a severe and persistent seizure burden. Many patients experience dozens to hundreds of seizures each day that remain resistant to available antiseizure medications. Disease onset occurs during infancy in approximately 80 percent of cases, with many affected children experiencing significant developmental impairment and some not surviving into adulthood. Patients with later-onset disease can experience disruptive nocturnal seizures alongside cognitive and psychiatric complications.

There are currently no FDA-approved therapies specifically for KCNT1+ epilepsy, creating a significant unmet medical need. ABS-1230 has recently demonstrated meaningful seizure reductions in an early clinical proof-of-concept trial involving children with KCNT1 epilepsy.

The therapy is currently being evaluated in the Phase 1b/2a KYRON trial, which is designed to potentially serve as the registrational study supporting a future New Drug Application (NDA) in the US. ABS-1230 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations. The programme has also been accepted into the FDA’s Rare Disease Evidence Principles (RDEP) programme, which is intended to facilitate the development of therapies for ultra-rare diseases.

The proposed acquisition further expands Jazz Pharmaceuticals’ focus on rare neurological diseases and builds on its experience in epilepsy, including its commercial portfolio led by Epidiolex. If successfully developed and approved, ABS-1230 could provide a targeted treatment option for patients with KCNT1+ epilepsy, a population that currently has no FDA-approved therapy specifically addressing the condition.