Cenobamate Gets New Approved Administration Routes, FDA Agrees to New Trial Design for NurOwn, ENRICH Results Published in NEJM

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

According to a new announcement, the FDA has granted approval for 2 new administration options for SK Life Science’s cenobamate CV (Xcopri; SK Life Science), a previously approved antiseizure medication (ASM) for adults with partial-onset seizures. The treatment tablets can now be crushed and mixed with water and either administered by mouth as an oral suspension or via a nasogastric tube, providing additional flexibility for dosing. This label update is supported by findings from an open-label, randomized, single-center, 3-period, 6-sequence, balanced crossover study that assessed the pharmacokinetics (PK) and safety of 3 cenobamate administration routes in 24 healthy participants (ages 18-50 years).

According to a new announcement, the FDA has agreed on a new design for a phase 3b trial of BrainStorm Cell Therapeutics agent NurOwn in patients with amyotrophic lateral sclerosis (ALS) using a special protocol assessment (SPA). The phase 3b trial, a 2-part, multicenter study, is expected to commence in 2024 and address objectives that support a future biologics license application (BLA) in ALS. NurOwn, an investigational product, is a technology platform of autologous mesenchymal stromal cells secreting neurotrophic factors cells (MSC-NTF). Under the SPA agreement, the FDA validated the clinical trial protocol and statistical analysis of the planned phase 3b study. Also known as Study BCT-006-US, the trial will enroll 200 participants earlier in the course of their disease, having the onset of ALS symptoms, including limb weakness, within the prior 24 months.

Investigators have published the full dataset of the pivotal ENRICH trial (NCT02880878), the first positive trial to show improved functional and economic outcomes for intracerebral hemorrhage (ICH), in the New England Journal of Medicine. All told, results from the study showed that early intervention of minimally invasive hematoma evacuation using NICO’s technology, BrainPath and Myriad, was safe and resulted in better functional outcomes at 180 days than guideline-based medical management. At 180 days, results showed that the trial met its primary end point, with the surgery group demonstrating a mean utility-weighted modified Rankin scale (UWmRS) of 0.458 compared with 0.374 for MM. This correlated to a posterior probability of superiority of 0.981, exceeding the 0.975 prespecified threshold superiority of surgery.

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