Tolebrutinib Shows Promise in Non-Relapsing Secondary Progressive MS, Fenebrutinib Suppresses Relapses in New Data, FDA Accepts Resubmitted NDA for AXS-07

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

Sanofi has announced topline data from its phase 3 HERCULES study (NCT04411641) of its investigational agent tolebrutinib, with results showing that treatment with the agent met its primary end point of reduction in disability accumulation among patients with non-relapsing secondary progressive multiple sclerosis (nrSPMS). The company plans to present study results from HERCULES, as well as its phase 3 GEMINI 1 and 2 studies (NCT04410978; NCT04410991) of tolebrutinib in relapsing MS at the upcoming European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in Copenhagen, Demark, September 20, 2024. In HERCULES, the study met its primary end point, which was 6-month confirmed disability progression (CDP) defined as an increase of more than 1 point from baseline on EDSS when the baseline score is less than 5, or the increase or at least 0.5 point when the baseline EDSS score was greater than 5.0.

New data expected to be presented at the 40th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Copenhagen, Denmark, show that treatment with investigational fenebrutinib (Roche) resulted in significant suppression of relapses in patients with relapsing multiple sclerosis (MS), in addition to no change in disability over 48 weeks. Phase 3 trials testing the reversible and non-covalent Bruton’s tyrosine kinase (BTK) inhibitor in both progressive MS and relapsing MS remain ongoing.1

The new data stems from the phase 2 FENopta study (NCT05119569), a randomized, double-blind, placebo-controlled 12-week trial that included an open-label extension (OLE) in which all patients receive fenebrutinib up to 192 weeks. In total, 91 patients entered the OLE and 96 remained in the OLE after 1 year. At 1 year, 96% of fenebrutinib-treated patients were free of relapses, equating to an annualized relapse rate of 0.04. In addition, as mentioned, patients on the investigational drug showed no change in disability over that time, as measured by the Expanded Disability Status Scale (EDSS).

According to a new announcement, the FDA has accepted Axsome Therapeutics’ resubmitted new drug application (NDA) for its novel agent AXS-07 as a treatment for acute migraine. The agency has given the oral, rapidly absorbed, multi-mechanistic investigational medicine a Prescription Drug User Fee Act (PDUFA) action goal date of January 31, 2025.AXS-07, a novel agent, is thought to act by inhibiting calcitonin gene-related peptide (CGRP) release, reversing CGRP-mediated vasodilation, and inhibiting neuroinflammation, pain signal transmission, and central sensitization. The agent, which consists of meloxicam and rizatriptan, is enabled by Axsome’s MoSEIC (Molecular Solubility Enhanced Inclusion Complex) technology, which results in faster absorption and longer plasma half-life.

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