Jazz Pharmaceuticals, a company that built its place in epilepsy drugs through business deals, just struck another one: $850 million to buy Actio Biosciences, a startup whose lead program is in development for a particular rare type of epilepsy with limited treatment options.
The centerpiece of the deal announced Monday is an Actio small molecule in development for treating epilepsy driven by mutations to a gene called KCNT1. While anti-seizure drugs can be used to treat some types of epilepsy, these medicines typically don’t work for KCNT1-related epilepsy, which has no FDA-approved therapies.
KCNT1-related epilepsy affects an estimated 2,500 patients in the U.S. who can experience dozens to hundreds of seizure episodes daily. In some cases, this disease starts showing in infancy, leading to problems that keep patients from achieving developmental milestones, such as walking and speaking. For those who have a later-onset form of the disease, nocturnal seizures are common along with cognitive and psychiatric problems.
Inside an Automated Healthcare Practice: Redesigning Care Around People, Not Paperwork
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
KCNT1 is a gene that codes for potassium channels in the membranes of cells. In KCNT1-related epilepsy, mutations to the gene lead to overactive channels that allow electric charges to flow across the membrane, sparking seizures. The lead drug of Diego-based Actio is ABS-1230, a KCNT1 ion channel inhibitor. This once-daily oral small molecule has reached the Phase 2 portion of a Phase 1b/2a trial that could serve as the registrational study to support a submission seeking FDA approval.
Dublin-based Jazz focuses on drugs for rare cancers and rare neurological disorders. The company’s top neuroscience product is the epilepsy drug Epidiolex, which crossed the $1 billion revenue threshold last year. This cannabidiol-derived drug, approved to treat the rare epilepsies Dravet syndrome and Lennox-Gastaut syndrome, came from the 2021 acquisition of GW Pharmaceuticals. The Jazz neuro pipeline also includes JZP053 (formerly SAN2355), a potential epilepsy drug licensed from Saniona nearly a year ago for $42 million up front. This small molecule activator of two ion channels is in preclinical development.
Actio has not publicly disclosed data for ABS-1230. In a prepared statement, Jazz President and CEO Renee Gala described the emerging clinical profile of Actio’s epilepsy drug as “highly encouraging.”
“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,” she said.
From Blind Spots to Better Outcomes: How SNF Data Transparency Strengthens LTSS
What if health plans could identify member decline before an avoidable hospitalization occurs? Real-time clinical visibility into long-stay SNF members uncovers risk earlier and drives better outcomes.
Beyond the $850 million upfront payment for Actio, Jazz is responsible for paying up to $500 million more if the epilepsy drug achieves milestones. With the Actio acquisition, Jazz joins other companies developing drugs for KCNT1-related epilepsy. Servier has reached Phase 1b/2 testing with an antisense oligonucleotide. UCB has a preclinical small molecule from a collaboration with epilepsy drug developer Praxis Precision Medicines. The pipeline of startup Atalanta Therapeutics includes a preclinical small-interfering RNA therapy designed to reduce levels of KCTN1 protein.
In a research note, Leerink Partners analyst Marc Goodman said the Actio acquisition is reminiscent of Jazz’s $935 million purchase of Chimerix last year. That deal brought a drug that went on to receive the first FDA approval for treating a rare type of brain cancer affecting children and young adults. In addition to premium drug pricing, approval of this product, Modeyso, came with a priority review voucher, which the company sold for $200 million. If approved, the Actio drug may also qualify for a voucher, Goodman said.
“We have known Actio for a few years and have always been intrigued by this program, but we have not seen the small [proof of concept] dataset yet,” he said. “Management indicated that the seizure reduction was ‘meaningful,’ and given the company’s solid track record of deals, we think management deserves the benefit of the doubt that this asset is worth the considerable upfront payment.”
San Diego-based Actio emerged in 2023 with $55 million in Series A financing. As its epilepsy drug approached the clinic last year, the biotech closed a $66 million Series B round of funding co-led by Regeneron Ventures and Deerfield Management. Actio has other programs that will continue their development in a privately held spinoff company. This company will take certain Actio executives, employees, and assets focused on rare neurological disorders. These assets include Actio’s ABS-0871, an oral small molecule designed to target TRPV4. This program is in early clinical development for Charcot-Marie-Tooth disease. There are currently no FDA-approved drugs for this rare, inherited disorder in which nerve damage leads to muscle weakness and muscle atrophy.
When the spinout happens, Jazz will hold a minority stake. Jazz said this new company’s rare disease focus fits the pharma company’s long-term strategy.
Image: Iaremenko, Getty Images