For the first time, the Food and Drug Administration is allowing a certain kind of cell therapy for epilepsy to be tested in humans.
The therapy, created by Shanghai-based Unixell Biotechnology, is designed to curb the excessive electrical activity that triggers seizures in epileptic patients. It uses donor-derived — or “allogeneic” — stem cells reprogrammed so that they ultimately produce the main chemical messenger, “GABA,” responsible for calming the brain and nervous system.
Unixell said that, in animal testing, its therapy appeared safe and able to suppress seizures by reconstructing inhibitory brain circuits. Now with the FDA’s approval, the company is moving forward with an early-stage study in people with drug-resistant epilepsy. Unixell noted how current epilepsy treatments, though helpful to some patients, carry “significant side effects,” so there’s an urgent need for novel therapies that can not only target the root source of the seizure, but also preserve healthy tissues and avoid safety issues.
The therapy’s progress is yet another example of the rapidly advancing Chinese biotech ecosystem, where, until recently, research focused less on innovation and more on developing drugs that are as good or somewhat better than existing medicines.
Pitchbook, an analytics firm, detailed this shift in a January report that found “emerging modalities” like cell and gene therapy have, over the past decade, continually received a greater portion of the venture dollars flowing into Chinese biotech. Last year, those modalities accounted for almost half of the total venture capital invested. This “highlights investor appetite for high-complexity assets over ‘fast-follower’ models,” wrote Pitchbook analyst Ben Zercher.
Additionally, the report showed that “innovative drugs” submitted for human testing by China’s developers more than tripled, from 688 to just shy of 2,300, between 2019 and 2023. And since 2021, China has registered nearly twice as many first-in-human trials for “next-generation antibodies” as the U.S. and Europe combined.
China’s biotech ecosystem has “gained the lead” generating early, promising drug candidates, and will likely hold onto it, Zercher wrote.
Should Unixell’s therapy eventually come to market, it would diversify a field dominated by older, “small molecule” anti-seizure medications. Those include levetiracetam, lamotrigine and carbamazepine, also known by their respective brand names Keppra, Lamictal and Tegretol.
Yet, Unixell will likely also face newer competition. Decades of research into ion channels — cellular tunnels that often play a role in epilepsy — has finally started to bear fruit.
Just this week, Xenon Pharmaceuticals disclosed that its ion-channel-opening drug azetukalner performed significantly better than a placebo in a study of hundreds of people with focal onset seizures. After 12 weeks of treatment, median monthly seizure frequency was down 53% among participants given a higher dose of azetukalner, compared to down 10% in the control arm.
Xenon now plans to ask for approval later this year. Shares of the Canada-based company rose 50% on the results. Paul Matteis, an analyst at the investment firm Stifel, described the data as a “home run” and “way above investor expectations.” Analysts at RBC Capital Markets, meanwhile, raised their forecast for the drug, and expect peak annual sales in the U.S. to reach at least $1.6 billion.
Biohaven and Quralis, a private biotech, have their own experimental drugs that, like Xenon’s, are meant to regulate the nervous system by activating potassium ion channels. Biohaven’s is in Phase 3 testing for focal epilepsy, while Quralis’ is earlier along and under investigation as a potential treatment for ALS, pain and rarer seizure disorders.
Praxis Precision Medicines has a sodium ion channel inhibitor that researchers are evaluating in mid- to late-stage experiments as a monotherapy and add-on therapy for focal epilepsy.
