Gefurulimab Positive Phase 3 Data Released, PoNS Device Eyes Stroke Indication, AD109 Performs in Phase 3 LunAIRo Trial

AstraZeneca has announced positive data from its phase 3 PREVAIL study (NCT05556096), with investigational gefurulimab, a complement C5 inhibitor administered once-weekly, meeting primary and all secondary end points in patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG). The company is planning to share more from the study at an upcoming meeting and discuss the findings with global regulatory authorities. PREVAIL, a global, double-blind, placebo-controlled, parallel study, included 260 patients with gMG across 20 countries who were randomly assigned to gefurulimab or placebo for 26 weeks, followed by an optional open-label study. According to the company, treatment with the investigational agent displayed statistically significant and clinically meaningful differences vs placebo on the primary end point of Myasthenia Gravis-Activities of Daily Living (MG-ADL), a common measure for gMG trials.

Helius has announced positive data from its Stroke Registrational Program (SRP) that supports the use of its Portable Neuromodulation Stimulator (PNS) Device as a beneficial treatment for poststroke patients with gait or balance deficits. Based on these new findings, the company is planning a formal submission to the FDA in the third quarter to expand its indication in this patient population. Stemming from positive conversations with the FDA, the SRP comprised 3 studies, including a double-blind, randomized controlled trial and a single-arm trial. Results revealed that the double-blind randomized trial met its primary end point, with those on the PoNS device demonstrating statistically significantly greater improvements in gait and/or balance deficit due to stroke. Notably, this finding was found with and without including additional data from an open-label study using statistical methods to balance baseline characteristics.

Apnimed has announced positive data from its pivotal phase 3, 12-month LunAIRo trial (NCT05811247) testing investigational AD109, a combination of aroxybutynin and atomoxetine, as a potential treatment for obstructive sleep apnea (OSA). These findings, along with previously announced phase 3 data from the SynAIRgy trial (NCT05813275), are expected to support a new drug application submission to the FDA in early 2026. In LunAIRo, treatment with the AD109 led to significant reductions in Apnea-Hypopnea Index (AHI), the primary end point, relative to those on placebo. At the 26-week mark, patients on the therapy achieved a mean reduction in AHI of 46.8% compared with reductions of 6.8% for placebo, which was statistically significant (P <.001). Notably, these effects were maintained out to the end of the study, around week 51 (P <.001).

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