HEALEY-ALS Platform Trial Amended, WVE-N531 Improves Muscle Growth in DMD, Diazoxide Choline Approved for Prader-Willi Syndrome

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

As it gears up to test its novel therapeutic NUZ-001, Neurizon Therapeutics announced an update to the HEALEY-ALS Platform trial’s master protocol, with changes that include longer trial duration, modified inclusion criteria, and a more patient-centric approach to the study. In the same update, the company also reported positive secondary end point data from its phase 1 MEND study of NUZ-001, with the agent demonstrating significant effects on slow vital capacity (SVC), a notable assessment for patients with amyotrophic lateral sclerosis (ALS). The platform trial, a prominent, double-blind, first-of-its-kind study, essentially tests numerous novel therapeutics in development for ALS while using the same placebo-controlled cohort to improve trial efficiency and reduce the number of patients on placebo. In the new update, the randomized controlled trial (RCT) period was extended from 24 to 36 weeks to allow for longer evaluation of treatment effects and increase the power to detect positive treatment outcomes, especially for therapies that take longer to show effects.

Newly announced 48-week positive data from the phase 2 FORWARD-53 study (NCT04906460) showed that treatment with investigational WVE-N531 (Wave Life Sciences) was safe and led to the first-ever improvement in muscle health with exon skipping in patients with Duchenne muscular dystrophy. Following recent feedback from the FDA, the company intends to file a new drug application (NDA) in 2026 for accelerated approval, with data to support monthly dosing at launch. The updated data featured 11 boys with DMD amenable to exon 53 skipping who advanced to the extension portion of the study and were receiving monthly doses of WVE-N531. Results revealed stabilization of dystrophin expression through treatment, with a mean of 7.8% (95%, 5.4-10.3%) between weeks 24 and 48 of dosing. Measured by western blot, the 48-week mean dystrophin expression was 6.4% (95% CI, 3.8-9.0%).

According to a new announcement, the FDA has approved Soleno Therapeutics' diazoxide choline (DCCR) extended-release tablets, marketed as Vykat XR, for the treatment of Prader-Willi syndrome (PWS) for patients 4 years and older who have hyperphagia. Thus, DCCR, a novel, proprietary extended-release dosage form containing diazoxide choline — the crystalline salt of diazoxide — is administered once daily and becomes the first therapy to treat hyperphagia in PWS. In November 2024, the FDA extended its review period for the new drug application (NDA) of DCCR extended-release tablets for PWS.2 The extension was triggered by recent responses to the agency's information requests, which were classified as a major amendment to the NDA. As a result, the PDUFA goal date was pushed back by 3 months to allow the FDA sufficient time to complete its review, including the newly submitted data. Notably, the agency did not highlight any concerns related to safety, efficacy, or manufacturing in its correspondence.

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