The phase 3 ENSURE program, assessing vidofludimus calcium for relapsing multiple sclerosis (RMS), will proceed as planned following a positive review of unblinded, interim data by an Independent Data Monitoring Committee (IDMC). The ENSURE program includes two phase 3 trials, ENSURE-1 and ENSURE-2.
Vidofludimus Calcium for Relapsing Multiple Sclerosis
The ENSURE trials are multicenter, randomized, double-blind studies expected to enroll approximately 1050 adults with RMS across 15 countries. The primary endpoint is time to first relapse up to 72 weeks. ENSURE-1 is anticipated to be completed in the second quarter of 2026, while ENSURE-2 is expected to conclude in the second half of 2026. The trials are evaluating the efficacy and safety of a 30 mg daily dose of vidofludimus calcium. Vidofludimus calcium is a small molecule drug designed to activate the neuroprotective transcription factor nuclear receptor related 1 (Nurr1), which has direct neuroprotective properties.
CHMP Recommends Eplontersen for ATTRv-PN
The Committee for Medicinal Products for Human Use (CHMP) has recommended approval for eplontersen (Wainua), developed by Ionis and AstraZeneca, for the treatment of adults with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Eplontersen, a self-administered treatment via auto-injector, received its initial approval in the United States in December. The CHMP's recommendation is based on data from the phase 3 NEURO-TTRansform trial (NCT04136184), a global, open-label, randomized study that evaluated the therapy over 85 weeks. Results published in JAMA demonstrated that eplontersen provided benefits across the spectrum of ATTRv-PN at 35, 66, and 85 weeks of treatment. Eplontersen is a once-monthly RNA-targeted medicine that suppresses transthyretin (TTR) production upstream and is administered subcutaneously at a dose of 45 mg.
ATX-01 Phase 1/2 Trial Commences for Myotonic Dystrophy Type 1
The first patient has been dosed in ARTHEx’s phase 1/2 trial assessing ATX-01, an antimiR oligonucleotide targeting microRNA 23b (miR-23b), as a potential treatment for myotonic dystrophy type 1 (DM1). This double-blind, placebo-controlled study, involving single- and multiple-ascending doses, aims to enroll 56 participants to evaluate the efficacy and safety of ATX-01. DM1, for which there are currently no approved treatments, results from the sequestration of muscle blind like (MBNL) proteins by toxic dystrophia myotonica protein kinase (DMPK) mRNA in the nucleus, along with translational repression of MBNL production caused by miR23b overexpression. This leads to a net decrease of MBNL available for its regulatory functions. ATX-01 has a dual mechanism of action, increasing MBNL production while destabilizing toxic DMPK foci, reducing DMPK mRNA, and releasing sequestered MBNL.
