Efgartigimod Meets End Point in Seronegative Myasthenia Gravis Study, Cemdisiran Submission for MG Coming Soon, Phase 3 Studies of IncobotulinumtoxinA Begin

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

Newly announced topline data from the phase 3 ADAPT SERON study (NCT06298552) showed that intravenous (IV) efgartigimod (Vyvgart; argenx) met its primary end point, demonstrating significant and clinically meaningful impacts on patients with seronegative myasthenia gravis (gMG), a patient population with no FDA approved treatments. Based on these findings, the company is planning to submit a supplemental biologics license application (sBLA) to expand its indication in seronegative gMG across 3 subtypes: muscle-antibody specific kinase (MuSK), LRP4+, and triple seronegative. ADAPT-SERON, a randomized, double-blind, placebo-controlled study featured 119 patients across numerous continents who were randomized to receive 4 once-weekly IV infusions of efgartigimod or placebo, followed by a 5-week follow-up and primary analysis. Overall, the treatment with efgartigimod let to statistically significant (P = .0068) impacts on Myasthenia Gravis Activities of Daily Living (MG-ADL) total score, the primary end point, after 29 days in Part A of the study.

Updates from the phase 3 NIMBLE trial (NCT05070858) revealed that investigational cemdisiran (Regeneron) met its primary and key secondary end points, demonstrating statistically significant effects as a monotherapy in patients with generalized myasthenia gravis (gMG). Based on these findings, Regeneron is planning to submit a new drug application (NDA) for cemdisiran as a monotherapy in the first quarter of 2026, pending discussions with the FDA. NIMBLE, a double-blind trial, randomly assigned adults with gMG to either cemdisiran monotherapy (n = 64) every 12 weeks, a combination of cemdisiran and pozelimab (n = 67; “cemdi-poze”) every 4 weeks, or placebo (n = 59) every 4 weeks for a 24-week treatment period. Cemdisiran is a small-interfering RNA therapy that works by silencing the production of C5 complement protein while pozelimab, marketed as Veopoz, works by binding to and inhibiting complement factor C5.

Patient dosing has begun for 2 new, large-scale, phase 3 trials, dubbed MINT-E (NCT07018700) and MINT-C (NCT07018713), that test the therapeutic safety and efficacy of incobotulinumtoxinA (Xeomin; Merz Therapeutics) as an option for episodic and chronic migraine in adults. Each of these trials will include the opposite patient population, with MINT-E comprising those with episodic migraine, defined as less than 15 headache days per month, while MINT-C includes those with chronic migraine. The studies, which are currently enrolling, are expected to include 1770 adults with migraine across 120 sites in North America and Europe.

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