NeurologyLive®'s Conference Coverage, Oveporexton Displays Symptom Improvement in NT1, FDA Allows BLA for AMT-130 in HD

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

This past week, NeurologyLive has been busy covering some of the most important conferences happening across the field of neurology. As our coverage from CMSC and ATMRD comes to a close, our team was already on the ground bringing you the latest updates from PNS in Maastricht, Netherlands, and SLEEP in Baltimore, Maryland.

From expert interviews and in-depth conversations to social media updates and emerging industry data, NeurologyLive continues to bring you the news and insights shaping the future of neurology. This week’s coverage featured everything from promising preclinical data on LRP1 agonists in neuroinflammation, to important phase 3 trial findings in narcolepsy, along with expert Q&As and conversations from leaders across the field. Be sure to visit NeurologyLive.com for the latest updates.

One of the biggest pieces of news to come from our conference coverage is that Newly presented findings from an analysis of 2 randomized, placebo-controlled phase 3 studies, The First Light (NCT06470828) and The Radiant Light (NCT06505031), showed that treatment with the investigational orexin receptor 2 agonist oveporexton (Takeda) was associated with improvements in REM sleep architecture and reductions in several sleep-related symptoms among patients with narcolepsy type 1 (NT1).¹

All told, the analysis of both studies showed that oveporexton normalized several objective measures of REM sleep abnormalities and also reduced patient-reported hallucinations, sleep paralysis, and disturbed nighttime sleep. Presented by lead author Lucie Barateau, MD, PhD, associate professor in the Sleep Unit of Montpellier University Hospital, at the 2026 SLEEP Annual Meeting, held June 14-17 in Baltimore, Maryland, these findings add to a growing body of evidence supporting orexin receptor agonism as a disease-targeted therapeutic strategy for NT1.

In other news, the FDA has indicated that 3-year data from the phase 1/2 clinical program of AMT-130, an investigational gene therapy for Huntington disease (HD), may serve as the primary basis for a planned Biologics License Application (BLA) submission seeking accelerated approval. According to developer uniQure, the company expects to submit the application in the third quarter of 2026.²

The regulatory update follows a recent Type B meeting between uniQure and the FDA, during which the agency requested additional alignment on the design of a confirmatory study that would follow a potential accelerated approval. According to the company, the FDA indicated that the confirmatory study may evaluate AMT-130 against standard-of-care therapy rather than requiring a sham procedure.²

Matt Kapusta, chief executive officer at uniQure, said in a statement.1 "Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA's genuine commitment to addressing the unmet need of Americans living with Huntington's disease. The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study."²

To read the full piece and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. Barateau L, Gong Y, Dauvilliers Y. Effects of Treatment with Oveporexton, an Orexin Receptor 2 Agonist, on Sleep in People with Narcolepsy Type 1: Phase 3 Results. Presented at: 2026 SLEEP Annual Meeting; June 14-17; Baltimore, Maryland. Abstract 0723
2. uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease. uniQure. News Release. June 17, 2026. Accessed June 17, 2026. https://www.uniqure.com/investors-media/press-releases