Phase 3 Program of Vidofludimus Calcium Continues, CHMP Recommends Approval for Eplontersen, Patient Dosing Commenced in Phase 1/2 Trial of ATX-01

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

According to an announcement from Immunic, an Independent Data Monitoring Committee (IDMC) review of unblinded, interim data from the phase 3 ENSURE program assessing vidofludimus calcium in patients with relapsing multiple sclerosis (RMS) was positive, advising that the trials continue as planned. ENSURE, which includes 2 phase 3 trials (ENSURE-1; ENSURE-2), uses time to first relapse up to 72 weeks as the primary end point.ENSURE 1 and 2 are 2 identical multicenter, randomized, double-blind trials expected to enroll approximately 1050 adults with RMS representing 15 different countries. ENSURE-1 is anticipated to be completed in the second quarter of 2026, while ENSURE-2 is expected to complete in the second half of 2026. The trials test the efficacy and safety of 30 mg daily doses of vidofludimus calcium, a small molecule drug that is designed to activate the neuroprotective transcription factor nuclear receptor related 1 (Nurr1), which is associated with direct neuroprotective properties.

According to a new announcement, the Committee for Medicinal Products for Human Use (CHMP) has recommended approval for Ionis’ and AstraZeneca’s eplontersen (Wainua) as a treatment for adults with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Eplontersen, the only self-administered treatment for ATTRv-PN via an auto-injector, was first approved in the United States last December. The CHMP’s opinion was based on findings from the phase 3 NEURO-TTRansform trial (NCT04136184), a global, open-label, randomized trial that assessed the therapy over an 85-week period. In the data, published in the JAMA, eplontersen demonstrated benefits across the spectrum of ATTRv-PN at 35, 66, and 85 weeks of treatment. Eplontersen, a once-monthly RNA-targeted medicine that provides upstream suppression of transthyretin (TTR) production, is an injection treatment administered subcutaneously in 45 mg doses.

According to a recent announcement, the first patient has been dosed in ARTHEx’s phase 1/2 trial assessing its investigational agent ATX-01, an antimiR oligonucleotide designed to target microRNA 23b (miR-23b), as a new treatment for myotonic dystrophy type 1 (DM1). The double-blind, placebo-controlled, single- and multiple-ascending dose study will aim to enroll 56 participants with the disease to assess the efficacy and safety of the therapeutic.DM1, a disease with no approved treatments, is caused by sequestration of muscle blind like (MBNL) proteins by toxic dystrophia myotonica protein kinase (DMPK) mRNA in the nucleus, and by translational repression of MBNL production caused by miR23b overexpression, both of which lead to a net decrease of MBNL available to exert its usual regulatory functions. ATX-01 is unique in that it has a dual mechanism of action, working to increase MBNL production while also destabilizing the toxic DMPK foci leading to a reduction of DMPK mRNA and release of sequestered MBNL.

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