Neuren's Announcement of Trofinetide New Drug Submission Acceptance by Health Canada

Neuren Pharmaceuticals (ASX: $NEU) has announced that Health Canada has accepted for priority review the New Drug Submission (NDS) of trofinetide for the treatment of Rett syndrome, submitted by Neuren's global partner Acadia Pharmaceuticals (Nasdaq: ACAD). The NDS is supported by results from the positive pivotal Phase 3 LAVENDER™ study, evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women with Rett syndrome.

Executive Commentary on Trofinetide New Drug Submission Acceptance

Pamela di Cenzo, Vice President, General Manager, Rare Disease, Canada at Acadia, stated, 'Rett syndrome is a profoundly debilitating and complex neurodevelopmental disorder. If granted marketing authorization, trofinetide will be the first option available to treat Rett syndrome in Canada.' Sabrina Millson, President of the Ontario Rett Syndrome Association (O.R.S.A.), expressed, 'Our community of patients, caregivers and supporters are excited at the prospect of having a treatment option for Rett syndrome.'

Summary of Neuren's Trofinetide New Drug Submission Acceptance

Neuren Pharmaceuticals (ASX: $NEU) has reported the acceptance of trofinetide New Drug Submission (NDS) for priority review by Health Canada, facilitated by its global partner Acadia Pharmaceuticals. If granted marketing authorization, trofinetide will be the first treatment option available for Rett syndrome in Canada. The NDS is supported by positive pivotal Phase 3 LAVENDER™ study results. Neuren's focus on developing new drug therapies for serious neurological disorders, including Rett syndrome, reflects its commitment to addressing urgent unmet medical needs. The company's partnership with Acadia Pharmaceuticals underscores its dedication to advancing treatments for rare and serious diseases. Neuren's second drug candidate, NNZ-2591, is also in development for various neurological disorders, demonstrating the company's broad efforts in this field.