AD109 Accepted NDA, AXS-12 FDA Submission, Phase 2 CELIA Study Results

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

According to an announcement, the FDA has accepted for review the new drug application (NDA) for Apnimed’s AD109, an investigational once-nightly oral combination agent targeting the neuromuscular basis of upper airway collapse, for the treatment of adults with obstructive sleep apnea (OSA), with a PDUFA target action date of February 28, 2027. The NDA is supported by data from 2 positive phase 3 randomized, double-blind, placebo-controlled trials, dubbed SynAIRgy (NCT05813275) and LunAIRo (NCT05811247), which both met their primary end point compared with placebo among adults with mild-to-severe OSA. Designed for use across varying levels of disease severity, AD109 offers the promise of a safe, effective, and more user-friendly alternative to current OSA treatments, which are often invasive or difficult for patients to tolerate.

According to a new announcement, the FDA has accepted for filing a new drug application (NDA) for Axsome Therapeutics’ investigational agent AXS-12 (reboxetine), a selective norepinephrine reuptake inhibitor, for the treatment of cataplexy in narcolepsy. The company noted that the agency has set a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027, and does not currently plan to convene an advisory committee to review the submission. The NDA is supported by positive data from 2 phase 3 controlled trials, dubbed SYMPHONY (NCT05059223) and ENCORE (NCT05113745), both of which met their primary end point of reducing the frequency of cataplexy attacks in patients with narcolepsy. AXS-12, previously granted FDA orphan drug designation, is designed to regulate noradrenergic activity to promote wakefulness, sustain muscle tone, and improve cognition.

Biogen presented full Phase 2 data from the CELIA study evaluating diranersen (BIIB080), an investigational intrathecally administered antisense oligonucleotide (ASO) targeting MAPT mRNA, in 416 adults with early Alzheimer's disease (AD) at the 2026 Alzheimer's Association International Conference (AAIC) in London.¹ Based on the data, Biogen plans to advance diranersen into confirmatory Phase 3 development. Overall, diranersen demonstrated treatment effects on 5 of 6 clinical endpoints across all dose arms at 18 months, with the 60 mg dose given every 24 weeks (Q24W) showing the most consistent and largest clinical benefit.¹ Compared with placebo (n = 115), the 60 mg dose (n = 60) slowed clinical decline by 0.54 points (26%) on CDR-SB, 42% on ADAS-Cog13 (P = .01), 50% on MMSE (P = .02), 30% on modified iADRS, and 23% on ADCOMS.

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