Ann: Phase 2 trial shows significant improvements in Pitt Hopkins, page-42

NEU: Phase 2 Pitt Hopkins Trial Results Exceed Expectations

Neuren Pharmaceuticals (NEU) has released the highly anticipated Phase 2 trial results for NNZ-2591 in Pitt Hopkins syndrome, and the data has significantly surpassed market expectations.

Broker Expectations:A broker analysis outlined three potential outcomes:

• Bull Case: Clear, statistically significant improvements across most/all endpoints, validating the drug's IGF-1 mechanism and its potential as a platform treatment for multiple neurodevelopmental disorders. This scenario anticipated a 15-20% share price increase.
• Base Case: Good but not exceptional data, suggesting some clinical impact and a modest 0-10% share price rise.
• Bear Case: No clinical impact, potentially causing a 20% drop in share price.

Delivered Results:NEU's announcement confirmed statistically significant improvements in all four efficacy measures specific to Pitt Hopkins syndrome, as assessed by both clinicians and caregivers (p<0.05). The Clinical Global Impression of Improvement (CGI-I) mean score was 2.6, and the Caregiver Overall Impression of Change (CIC) mean score was 3.0. Importantly, NNZ-2591 demonstrated an excellent safety and tolerability profile with no severe adverse events.

Impact:The results align with the bull case, suggesting a likely 15-20% increase in NEU's share price. This outcome not only strengthens confidence in NNZ-2591’s potential but also positions NEU favorably for future developments and potential big pharma interest.