Neuren Pharmaceuticals' NNZ-2591 Shows Promising Results in Phase 2 Trial for Angelman Syndrome
NEUREN PHARMACEUTICALS LIMITED (NEU) Share Update August 2024 Friday 9th
Neuren Pharmaceuticals Announces Positive Phase 2 Trial Results for Angelman Syndrome Treatment
Neuren Pharmaceuticals (ASX: NEU) has announced encouraging results from its Phase 2 clinical trial of NNZ-2591, showing significant improvements in children with Angelman syndrome.
Instant Summary:
- Statistically significant improvements in global efficacy measures for Angelman syndrome.
- 11 out of 13 children showed improvement in Clinical Global Impression of Improvement (CGI-I).
- 8 out of 12 children showed improvement in Caregiver Overall Impression of Change (CIC).
- Every child in the younger age segment (3-12 years) showed improvement.
- NNZ-2591 was safe and well-tolerated with no serious adverse events.
Phase 2 Trial Overview
Neuren Pharmaceuticals conducted an open-label Phase 2 trial of NNZ-2591 in children aged 3 to 17 years with Angelman syndrome (AS). The trial, held at three hospitals in Australia, focused on safety, tolerability, pharmacokinetics, and efficacy over a 13-week treatment period. NNZ-2591 was administered as an oral liquid dose twice daily, with dose escalation in two stages up to 12 mg/kg.
16 participants were enrolled, with 13 completing the dosing regimen. The primary endpoints were safety and tolerability, while secondary endpoints included efficacy measures assessed by clinicians and caregivers.
Safety and Tolerability
NNZ-2591 demonstrated a good safety profile, with most treatment-emergent adverse events (TEAEs) being mild or moderate and unrelated to the study drug. There were no serious adverse events, and no significant trends in laboratory values or other safety parameters were observed.
Efficacy Results
Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed statistically significant improvements. The mean improvement from baseline was significant, whether calculated for the subjects that completed dosing or including discontinued subjects.
11 out of 13 children showed improvement in the Clinical Global Impression of Improvement (CGI-I), with a mean score of 3.0 (p=0.0010). In the younger age group (3-12 years), all 8 children showed improvement, with a mean score of 2.8 (p=0.0078).
8 out of 12 children showed improvement in the Caregiver Overall Impression of Change (CIC), with a mean score of 3.2 (p=0.0273). In the 3-12 years age group, all 8 children showed improvement, with a mean score of 2.8 (p=0.0078).
Development of NNZ-2591
Neuren is developing NNZ-2591 for multiple neurodevelopmental disorders. Positive results have been reported in Phase 2 trials for Phelan-McDermid syndrome and Pitt Hopkins syndrome. An End of Phase 2 Meeting with the US FDA for NNZ-2591 in Phelan-McDermid syndrome is scheduled for September 2024.
Manufacture of supplies for Phase 3 clinical trials is in progress. Neuren is also conducting pre-clinical studies for NNZ-2591 in other undisclosed indications.
The positive Phase 2 trial results for NNZ-2591 could significantly boost Neuren Pharmaceuticals' stock price, as the treatment shows promise for addressing a critical unmet need in Angelman syndrome. The safety and efficacy demonstrated in this trial may attract investor interest and increase confidence in the company's broader neurodevelopmental disorder pipeline.
Investor Reaction:
Analysts are likely to react positively to the news, given the lack of approved treatments for Angelman syndrome and the promising trial results. The statistically significant improvements and good safety profile of NNZ-2591 may lead to increased investor optimism about Neuren's future prospects.
Conclusion:
Investors should keep an eye on Neuren Pharmaceuticals as the company progresses to Phase 3 trials for NNZ-2591 and seeks further regulatory guidance. The promising results from the Phase 2 trial for Angelman syndrome position Neuren as a key player in the neurodevelopmental disorder treatment space.