Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS).
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[18-December-2023] |
Highlights:
- Clinical Global Impression of Improvement (CGI-I) - mean score of 2.4, with 16 out of 18 children showing improvement assessed by clinicians - Caregiver Overall Impression of Change (CIC) – mean score of 2.7, with 15 out of 18 children showing improvement assessed by caregivers
MELBOURNE, Australia, Dec. 18, 2023 /PRNewswire/ -- Neuren Pharmaceuticals Limited (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS). Significant improvement was observed by both clinicians and caregivers from treatment, across multiple efficacy measures. Improvements were consistently seen across many of the core PMS characteristics. PMS has severe quality of life impacts for those living with the syndrome, as well as parents and siblings. There are no approved treatments for PMS despite its severely debilitating impact. Neuren CEO Jon Pilcher commented “The strength and consistency of these results has exceeded our expectations and gives us high confidence as we strive to accelerate the development of a potential first therapy to address the overwhelming unmet medical need of PMS. We are very grateful to all the people at the trial sites in the United States and in the PMS community who enabled this groundbreaking trial to be completed successfully.” Elizabeth Berry-Kravis, MD, PhD, Professor Department of Pediatrics at Rush University Medical Center in Chicago and an Investigator in the study, commented: “Although the Phase 2 trial was an open label study, I am very encouraged that both clinicians and caregivers observed pervasive improvements across multiple, clinically important features of PMS including communication, cognition, learning, socialisation and behaviour. Improvements typically considered clinically meaningful were achieved in the clinician rated CGI-I as well as the caregiver rated CIC. I look forward to seeing this program advance to the next stage of development for the PMS community”. Study design The primary endpoints of this first trial in children were safety, tolerability and pharmacokinetics. Secondary endpoints included 14 efficacy measures assessed by clinicians and by caregivers. Efficacy measures included global measures assessing overall change, measures assessing specific symptom areas and measures assessing quality of life. Safety and tolerability Efficacy The results for the global measures rated by both clinicians and caregivers showed a level of improvement typically considered clinically meaningful. 16 out of 18 children showed improvement measured by the Clinical Global Impression of Improvement (CGI-I), an assessment by the clinician of the child’s overall status compared with baseline. The mean CGI-I score was 2.4. 10 children received a score of either 1 (“very much improved”) or 2 (“much improved”). 15 out of 18 children showed improvement measured by the Caregiver Overall Impression of Change (CIC), an assessment by the caregiver of the child’s overall status compared with baseline. The mean CIC score was 2.7. Seven children received a score of either 1 (“very much improved”) or 2 (“much improved”). Other ongoing Phase 2 trials of NNZ-2591 Detailed results are available in the full announcement: https://www.neurenpharma.com/pdf/f9ca13b8-b75f-4ff5-a5b7-1dcb75d547d6/P2-trial-shows-significant-improvements-in-PhelanMcDermid.pdf About Phelan-McDermid syndrome PMS has severe quality of life impacts on those living with it, as well as on parents and siblings. The most common characteristics are moderate to severe developmental and intellectual impairment and developmental delay, delayed or absent speech, symptoms of autism, low muscle tone, motor delays, mild to severe epilepsy, difficulties with toilet training and problems with eating. Further information about PMS is available at: www.pmsf.org About Neuren DAYBUE™ (trofinetide) is approved by the US Food and Drug Administration (FDA) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide. Neuren’s second drug candidate, NNZ-2591 is in Phase 2 development for each of Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome. ASX Listing Rules information Forward-looking Statements View original content:https://www.prnewswire.com/news-releases/neuren-phase-2-trial-shows-significant-improvements-in-phelan-mcdermid-syndrome-302017560.html SOURCE Neuren Pharmaceuticals Ltd | ||
Company Codes: Australia:NEU |