Brivaracetam

Through an acquisition announced Friday, Belgian drug company UCB is wagering more than $1 billion on a cutting-edge medicine thats being tested as a potential therapy for a few seizure disorders as well as Alzheimers disease.UCB offered to buy privately held Neurona Therapeutics for $650 million up front while dangling another $500 million in future, milestone-based payments. If completed, the deal would hand UCB an experimental therapy that uses pluripotent stem cells engineered to calm the brain and repair neural networks.The therapy, codenamed NRTX1001, is currently being evaluated in clinical trials as a treatment for patients with hard-to-treat forms of a common epilepsy rooted in the mesial temporal lobe part of the brain.Such an asset would diversify UCBs arsenal of seizure treatments, which are all small molecule drugs. That list includes Vimpat, Keppra, Briviact and Fintepla with the last being the most recent to secure U.S. approval, for seizures associated with a rare, severe type of childhood epilepsy that often causes long-term disabilities. In 2025, UCB recorded close to 1.2 billion euros, or roughly $1.3 billion, in net sales from just Briviact and Fintepla.In a statement, the company said this acquisition marks a strategic expansion into regenerative medicine and advanced therapies, while showcasing a commitment to inorganic growth and to epilepsy solutions that go beyond symptomatic management.We believe this therapy has the potential to provide durable targeted repair of the nervous system following a single dose and could represent a major step forward for people living with mesial temporal lobe epilepsy, CEO Jean-Christophe Tellier said in the statement.Though often expensive to make and challenging to administer, cell therapies have proven to be powerful tools for fighting certain cancers. A growing body of evidence also indicates these treatments can be helpful combating diseases tied to the immune and nervous systems, which in turn has piqued investor interest. Back in 2024, for instance, Neurona raised $120 million to support its research, through a funding round co-led by Viking Global Investors and Cormorant Asset Management.More recently, Aspen Neuroscience, a biotech developing a cell therapy for Parkinsons disease, closed a $115 million Series C round. And just last month, Oryon Cell Therapies, another startup making a cellular medicine for Parkinson's, emerged from stealth with $42 million in funding.Early this year, analysts at the investment bank Stifel met with two prominent doctors in the epilepsy space, who said around one-third of patients with focal onset seizures a broad category that encompasses mesial temporal lobe epilepsy remain treatment-refractory despite a significant increase in the number of FDA-approved anti-seizure medications over the past 20 years.The statistic underscores a significant unmet need among this population, the analysts wrote.Additionally, the doctors described how community neurologists often prefer to prescribe drugs that are easy to use, like Vimpat or Keppra. They viewed azetukalner, a closely watched, ion-channel-opening medicine from Xenon Pharmaceuticals, as having many of the same attributes that made Vimpat or Keppra commercial successes. Xenon, notably, has become a top takeover target to some on Wall Street.A cell therapy like NRTX1001 may therefore face a bit of an uphill battle. But UCB sees an opportunity. Among other potentially advantageous characteristics, like being a durable, one-off treatment, the therapy is administered to the brain through a minimally invasive surgical procedure.UCB confirmed the deal wont impact the companys 2026 revenue guidance, which is expected to grow in a high single-digit to low double-digit percentage range at constant exchange rates. Underlying profitability, meanwhile, is now expected to grow in a high single-digit to mid-teens percentage range at constant exchange rates. '

Vancouver and Boston-based Xenon Pharmaceuticals announced positive topline results from the Phase III X-TOLE2 study of azetukalner, a KV7 potassium channel opener, in adults with focal onset seizures. The X-TOLE2 study results position azetukalner as the first drug in its mechanistic class to reach a potential US FDA filing since retigabine (from GSK/Valeant Pharma) was withdrawn from the market in 2017 due to safety concerns, and the data exceeded those from the earlier Phase IIb X-TOLE trial. X-TOLE2 was a randomized, double-blind, placebo-controlled, multicenter Phase III study evaluating azetukalner as adjunctive oral therapy, administered once daily with food, in adults with focal onset seizures. The trial randomized 380 participants across three arms — azetukalner 25 mg, azetukalner 15 mg, and placebo — with 374 entering the modified intent-to-treat population. Participants had treatment-resistant epilepsy, with a median of five prior antiseizure medications and a baseline seizure frequency of 12.75 per month; 51.3% were taking three concomitant antiseizure medications. The primary endpoint was median percent change in monthly focal onset seizure frequency from baseline to week 12. The 25 mg group achieved a -53.2% reduction (p=0.000000000006) and the 15 mg group achieved -34.5% (p=0.00007), compared with -10.4% for placebo. The placebo-adjusted reduction for the 25 mg dose was -42.7%, compared with -34.6% in the Phase IIb X-TOLE study. The key secondary endpoint, the proportion of participants with at least 50% seizure reduction, was 54.8% at 25 mg and 37.6% at 15 mg, versus 20.8% for placebo. The most common adverse events across azetukalner groups were dizziness (20.5%), headache (8.8%), somnolence (8.8%), and fatigue (7.6%). Discontinuation due to adverse events occurred in 14.5% of the 25 mg group, 4.8% at 15 mg, and 3.2% on placebo. Of 332 participants completing the double-blind period, 322 entered the open-label extension. Xenon anticipates submitting a New Drug Application to the US FDA in Q3 2026. If approved, it would be the only KV7 potassium channel opener available for epilepsy treatment. Azetukalner opens KV7.2/KV7.3 potassium channels in the central nervous system, enhancing the M-current that stabilizes neuronal resting membrane potential and reduces excessive firing — the core pathophysiology of seizure generation. This mechanism was clinically validated by retigabine (ezogabine), which received US FDA approval for focal seizures in 2011 but was voluntarily withdrawn after reports of skin and retinal discoloration linked to off-target activity at KV7.4/KV7.5 subtypes. Azetukalner was designed with selectivity for KV7.2/KV7.3 to avoid those liabilities. Epilepsy affects approximately three million adults in the US, and focal epilepsy is its most common form. Despite more than 30 approved antiseizure medications, up to half of patients with focal epilepsy continue to experience uncontrolled seizures. Most available therapies act through sodium channel blockade or GABAergic modulation, and few mechanistically distinct options have emerged in recent years. Key competitors and pipeline assets include: Cenobamate (SK Life Science), a dual sodium channel blocker and GABAA receptor modulator approved by the US FDA in 2019 for focal onset seizures in adults, currently in additional Phase III studies. PRAX-562 (Praxis Precision Medicines), a selective persistent sodium current blocker in Phase II development for focal onset seizures. Brivaracetam (UCB Pharma), an SV2A ligand approved by the US FDA in 2016 for focal onset seizures, with ongoing Phase III trials in pediatric populations. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website