Neuren jumps over +11% on patent win for Pitt Hopkins drug treatment, page-4

I am blown away by the end of that article "Stocks appear to have begun a downward trajectory last August when the company’s Phase 2 results in a trial treating Angelman Syndrome failed to impress".

I did not think that was the case and after reading the article in that link, I was still unsure why it was categorised as failing to impress.

I asked Gemini to review and explain and here is what it said:

The Phase 2 trial results for Neuren Pharmaceuticals' NNZ-2591 in children with Angelman syndrome were indeed positive from a clinical standpoint, showing statistically significant and clinically meaningful improvements.

Here's a breakdown of the trial results:

• Significant Improvements: The trial demonstrated statistically significant improvements in global efficacy measures specifically designed for Angelman syndrome. These improvements were observed after 13 weeks of treatment.
• Key Areas of Improvement: Positive changes were noted in several critical aspects of Angelman syndrome, including:
  • Communication
  • Behavior
  • Cognition
  • Motor abilities
• Clinician and Caregiver Assessments:
  • Clinical Global Impression of Improvement (CGI-I): Clinicians assessed that 11 out of 13 children showed improvement, with a mean score of 3.0 (p=0.0010).
  • Caregiver Overall Impression of Change (CIC): Caregivers reported improvement in 8 out of 12 children, with a mean score of 3.2 (p=0.0273).
• Younger Age Group Performance: All children in the 3-12 years age segment showed improvement as measured by both CGI-I (mean score 2.8, p=0.0078) and CIC (mean score 2.6, p=0.0078).
• Safety and Tolerability: NNZ-2591 was found to be safe and well-tolerated as an oral liquid dose, with no serious adverse events or notable negative trends in safety parameters.

These results were highlighted by Neuren Pharmaceuticals as strengthening confidence in NNZ-2591's potential for multiple neurodevelopmental disorders.

Why the Market Reaction Might Have Been Subdued:

Despite these positive clinical outcomes, the market's reaction, as suggested by the HotCopper article title "fails to impress" and a dip in share price, appears to be due to broader factors beyond the trial results themselves. According to Neuren's 2024 Annual Report to shareholders, the company's share price decline was frustrating despite positive trial results and "buy" recommendations from analysts. This was attributed to:

• Momentum Trading: Negative momentum trading significantly impacted the share price.
• Sentiment on DAYBUE Sales: An initial switch to negative sentiment regarding sales of DAYBUE (another of Neuren's drugs) contributed to the overall market perception.

Therefore, while the trial results for NNZ-2591 were clinically encouraging, external market dynamics and sentiment surrounding other aspects of the company's performance likely influenced the stock's reaction.


I maintain my view that the company could do worse than retain some marketing/PR guru!