Jazz Pharmaceuticals is reaching into its wallet once again, this time buying Actio Biosciences and an experimental therapy for KCNT1+ epilepsy, a form of the disease with no FDA-approved treatments.
Jazz Pharmaceuticals is buying Actio Biosciences for $820 million in cash to get its hands on the California biotech’s lead candidate for a rare form of epilepsy.
Jazz is also offering Actio the chance to collect up to $500 million in additional milestone payments, according to a Monday release, with the deal expected to close by the end of this year.
With its purchase, Jazz gets access to ABS-1230, a clinical-stage small molecule designed to treat a rare form of epilepsy. The candidate holds the potential to become a first-in-class precision therapy for KCNT1+ epilepsy, a genetic form of the disease that impacts about 2,500 Americans, according to the companies.
Patients with KCNT1+ epilepsy, which typically onsets in infancy, can experience dozens to hundreds of seizure episodes daily. Currently, there aren’t any FDA-approved therapies for the disease and antiseizure medications have a limited effect.
In an early proof-of-concept trial, Actio’s ABS-1230 was tied to meaningful seizure reductions in children with KCNT1 epilepsy, according to Monday’s release. The ongoing Phase 1b/2a KYRON trial is designed to serve as the registrational study for the drug, which has secured FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations. ABS-1230 is also part of the FDA’s new Rare Disease Evidence Principles program for ultra-rare disease therapies.
Actio’s ABS-1230 also holds potential as a treatment in additional genetic epilepsy indications, the company said.
Once the deal closes, Actio Biosciences will spin out into a privately held, independent company with its remaining assets. Irish biopharma Jazz will hold a minority stake in the spinout, which will focus on a small molecule TRPV4 inhibitor called ABS-0871 for Charcot-Marie-Tooth disease type 2C.
For Jazz, the buy “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,” CEO Renee Gala said in a prepared statement Monday.
Epidiolex is a cannabidiol that was first approved in 2018 for seizures tied to Lennox-Gastaut syndrome (LGS) and Dravet syndrome, with several label expansions since. Jazz picked up the drug in its 2021 buyout of GW Pharmaceuticals.
Now, Epidiolex “remains the epilepsy anchor at +16% YoY,” Truist Securities analysts wrote last week, bringing in $292 million in the second quarter of this year.
As for Actio, the biotech launched a few years ago and raised $55 million in series A cash in 2023. The biotech’s mission is to identify and target the root causes of rare diseases and develop novel precision medicines.
“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,” Actio CEO David Goldstein said in the release. “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.”
