Wave Discontinues ALS Agent WVE-004, FDA Pushes Back Decision on SRP-9001, Asundexian Receives Fast Track Designation

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

Despite hitting its target engagement, newly announced topline findings from the phase 1b/2a FOCUS-C9 study showed that treatment with WVE-004 did not result in clinical benefit after 24 weeks, prompting Wave to discontinue its development. The company remains on track to share data later this year from its phase 1b/2a SELECT-HD study assessing its investigational agent WVE-003 in patients with Huntington disease. FOCUS-C9 was a global, double-blind, placebo-controlled trial that assessed the safety and tolerability of single- and multiple-ascending doses of WVE-004, an antisense oligonucleotide, in patients with C9orf72-associated amyotrophic lateral sclerosis and frontotemporal dementia. Over a 24-week treatment period, investigators observed maximal mean reductions of 48% and 50% in poly(GP), a pharmacodynamic biomarker, in the WVE-004 groups dosed every 12 or 4 weeks.

In a company update, Sarepta Therapeutics announced that the FDA has informed it will need additional time to complete the review of SRP-9001, a gene therapy in development for Duchenne muscular dystrophy (DMD). It anticipates that the review will be complete by June 22, 2023. Sarepta noted in its announcement that the agency has indicated that it is working toward potentially granting an accelerated approval for SRP-9001, using the phase 3 EMBARK study (NCT05096221) as a confirmatory trial. If approved, the therapy would become the first gene therapy on the market for DMD, with an indication for patients between 4 and 5 years old; however, the FDA may entertain a non-age-restricted expansion of the SRP-9001 label if EMBARK meets its objectives. EMBARK is set to complete later this year and have data read out in the fourth quarter of 2023.

In a recent announcement, the FDA granted fast track designation for Bayer’s investigational drug asundexian (BAY2433334), an oral direct, potent inhibitor of activated coagulation factor XI (FXIa), as a potential treatment to prevent stroke and systemic embolism in patients with atrial fibrillation (AF). The therapy is currently being evaluated in the OCEANIC clinical trial program, which comprises 2 large multinational studies, OCEANIC-AF and OCEANIC-STROKE, expected to enroll more than 27,000 patients over more than 40 countries. This was the second time asundexian receives fast track designation, the first of which came in 2022 for the prevention of stroke in patients after a non-cardioembolic ischemic stroke.

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