Remternetug Significantly Reduces Amyloid Plaque, Phase 3 INTUNE Study Accelerated, Pitolisant Approved for Pediatric Narcolepsy by EMA

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

New interim data from a phase 1 multiple ascending dose study of patients with mild cognitive impairment or mild to moderate dementia due to Alzheimer disease (AD) showed that treatment with remternetug, an investigational agent developed by Eli Lilly, resulted in rapid and robust amyloid plaque reduction. Remternetug, an N3pH-Aß monoclonal antibody, is a follow-on to donanemab, Eli Lilly’s phase 3 antibody also designed against pyroglutamated amyloid-ß. Initiated in July 2020, the newly presented findings showed dose-dependent amyloid plaque lowering across all dosing regimens tested. Through mixed model repeated measures, investigators observed P values of less than 0.001 relative to placebo for each group except those on 250 mg every 4 weeks at day 85.

According to a recent announcement, the timeline for the phase 3 INTUNE study assessing Harmony’s therapy pitolisant (Wakix) in patients with idiopathic hypersomnia (IH) has been accelerated, with enrollment expected to be complete by the second quarter of 2023 and topline data anticipated to be read out in the fourth quarter of this year. Based on a pre-specified interim sample size analysis, a recently convened independent data monitoring committee concluded that the study could continue with the original planned enrollment population. INTUNE, a double-blind, placebo-controlled, randomized, withdrawal study, spans 60 sites in the US, and includes approximately 200 patients with IH. The study, announced in April 2022, uses change in Epworth Sleepiness Scale score as the primary outcome measure to assess the effects of pitolisant vs placebo.

Recently, the European Medicines Agency (EMA) granted approval for the marketing authorization for pitolisant (Wakix, Harmony Biosciences) for the treatment of narcolepsy in pediatric patients age 6 years or older, with or without cataplexy.1 Pitolisant is the first and only drug in the class of antagonist/reverse agonists of the histamine H3 receptor for the treatment of narcolepsy. In a randomized trial investigating pitolisant, 110 pediatric patients with narcolepsy were enrolled, 72 received the treatment and 38 were given placebo. The primary endpoint assessed excessive daytime sleepiness and cataplexy attacks using the UNS. Findings showed a significant decrease of 6.29 points in the UNS score with pitolisant versus -2.60 points with placebo. The secondary endpoints demonstrated that pitolisant significantly reduced sleepiness and cataplexy after 8 weeks compared to placebo.

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