MSA Agent ATH434 Shows Promising Phase 2 Data, FDA Approves Suzetrigine for Severe Acute Pain, AXS-07 Gains FDA Approval for Migraine

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

New topline results from the randomized, double-blind, placebo-controlled phase 2 trial (NCT05109091) of Alterity Therapeutics’ ATH434 revealed that treatment with the investigational agent was well tolerated and demonstrated clinical as well as biomarker-based benefits, particularly at the 50 mg dose, among patients with early-stage multiple system atrophy (MSA). These findings support ATH434’s potential as a disease-modifying therapy for MSA to slow disease progression and reduce iron accumulation in key brain regions. The study enrolled 77 participants across 23 sites in 6 countries, who received either 50 mg or 75 mg of ATH434 or placebo twice daily for 12 months. Findings showed that ATH434 was well tolerated and effectively reduced iron accumulation in brain regions impacted by MSA. The 50 mg dose demonstrated the greatest benefit, slowing clinical progression by 48% at 52 weeks compared with the placebo (P = 0.03). Additional analyses also suggested trends in improved motor function and preserved brain volume.

According to a new announcement, the FDA has approved suzetrigine (Vertex Pharmaceuticals), an oral selective NaV1.8 pain signal inhibitor, to treat patients who experience moderate-to-severe acute pain. Marketed as Jor na vix, the novel, non-opioid therapy becomes the first new class of medicine to treat acute pain in over 20 years. The new drug application (NDA) for suzetrigine, formally known as VX-548, was based on data from a phase 3 program that included 2 randomized, double-blind, placebo-controlled trials; 1 post abdominoplasty surgery and 1 post bunionectomy surgery. The program also included a single arm safety and efficacy study that included patients with a wide range of surgical and non-surgical pain conditions.

Months after the FDA accepted Axsome Therapeutics’ resubmission of its new drug application (NDA) in September 2024, the agency has approved AXS-07, an oral, rapidly absorbed, multi-mechanistic agent, as a new acute treatment of migraine with or without aura in adults. Marketed as Symbravo, the company noted that it anticipates AXS-07 to be commercially available in the United States in approximately 4 months. 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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