Neuren Pharmaceuticals Limited

Neuren's partner Acadia Pharmaceuticals Inc. received Health Canada approval of DAYBUE™ (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older under the Priority Review process. This makes it the first and only drug approved in Canada for the treatment of Rett syndrome. Neuren CEO Jon Pilcher commented: “We are excited to see this first approval outside the United States, which is a significant milestone in the ongoing program to expand access to DAYBUE.” Click below to read more.

Jon Pilcher, CEO of Neuren Pharmaceuticals (ASX: NEU) will present at ASX CEO Connect on Tuesday 22 October 2024 AEDT. Please register at no cost using the link provided below.  https://lnkd.in/gJmb6ByV

Neuren is proud to be named winner of the Australian Growth Company Awards Health & Life Sciences Growth Company of the Year Award for the second consecutive year.

Neuren today announced positive outcomes from an End of Phase 2 Meeting with the US Food and Drug Administration (FDA) to discuss proposals for the first ever pivotal clinical trial program in Phelan-McDermid syndrome (PMS). There are no medications approved for PMS, which has severe quality of life impacts on those living with it, as well as on parents and siblings. At the FDA meeting alignment was reached on key aspects of the NNZ-2591 Phase 3 program, with Neuren to submit further information to confirm endpoints for the primary efficacy assessment. Neuren CEO Jon Pilcher commented: “We are pleased with the outcomes of a very collaborative meeting with the FDA and are eager to move forward in our mission to develop NNZ-2591 as a first approved treatment for Phelan-McDermid syndrome, which has an overwhelming unmet need.” Click to read more.

Neuren Pharmaceuticals Limited has been announced a finalist for the Australian Growth Company Awards for Health and Life Sciences. These awards celebrate and recognise the achievements of high-growth companies across Australia.

Neuren today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). NNZ-2591 was safe and well tolerated as an oral liquid dose and improvements were seen in clinically important aspects of AS. Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from baseline that was statistically significant and considered clinically meaningful. Neuren CEO Jon Pilcher commented “These results provide additional confirmation that NNZ-2591 as an oral liquid dose may address the core symptoms of diverse neurodevelopmental disorders, independent of the origin of the underlying genetics. We are very grateful to the people in the Angelman syndrome community and at the trial sites in Australia who enabled the successful completion of the trial.”

Neuren is delighted to be presenting at at PEAK Asset Management and Monsoon Communications’ #BiotechShowcase. View the program and register to attend: https://peak.contact/

Hear from a panel of top line corporate advisors and 8 biotech companies, including Neuren CEO Jon Pilcher, at the #BiotechShowcase in Melbourne on 21 August. Registration is free, spots are limited - register now: https://peak.contact/

Neuren today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Pitt Hopkins syndrome (PTHS). Statistically significant improvement from baseline was observed by both clinicians and caregivers from treatment, across all 4 efficacy measures that were specifically designed to assess the core characteristics of PTHS. Neuren CEO Jon Pilcher commented “We are very excited about the results of this first clinical trial in Pitt Hopkins patients. This underserved community has such urgent unmet need and we can now continue towards our goal of developing a first approved treatment. We are very grateful to the people in the Pitt Hopkins community and at the trial sites in the United States who enabled the successful completion of this extremely challenging, but groundbreaking trial.” Click below for more:

Neuren is pleased to report that Health Canada has accepted for Priority Review the New Drug Submission (NDS) of trofinetide for the treatment of Rett syndrome, that was submitted by Neuren’s global partner Acadia Pharmaceuticals Inc. Click to read more.