Neuren Pharmaceuticals Limited

ASX:NEU

Neuren Pharmaceuticals Limited

ASX:NEU
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$19.70 (+2.7%)
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#9
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35
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Pinned straw:
Nnyck777
Last edited 4 months ago

Daybue’s main competitor for the treatment of Rett’s has failed to meet its primary end points.

This provides further Moat and validation of current financial models for Daybue.

Anavex Life Sciences Provides an Update on Rett Syndrome Program

Anavex Announces Topline Results from Phase 2/3 EXCELLENCE Clinical Study in Pediatric Rett Syndrome


Validation from Real World Evidence (RWE) of Rett Syndrome Patients under Compassionate Use Authorization



NEW YORK – January 2, 2024

Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics forthe treatment of neurodegenerative and neurodevelopmental disorders today reported topline results from the randomized, double-blind, placebo-controlled, Phase 2/3 EXCELLENCE clinical trial, which evaluated the clinical efficacy, safety, and tolerability of 30 mg ANAVEX®2-73 in 92 pediatric patients with Rett syndrome (RTT) between the ages of 5 through 17 years. Participants were randomized 2:1 (ANAVEX®2-73 [62 patients] to placebo [30 patients]) for 12 weeks, followed by a week 16 safety visit. As well, Anavex reported positive Real World Evidence (RWE) feedback from Rett syndrome patients under Compassionate Use Authorization.

 

This was the very first study of ANAVEX®2-73 in pediatric patients with Rett syndrome. After 12 weeks, the study showed improvement on the key co-primary endpoint Rett Syndrome Behaviour Questionnaire (RSBQ), which is a detailed 45-item questionnaire for assessing multiple Rett syndrome characteristics by the patients’ caregivers. The other co-primary endpoint, the Clinical Global Impression – Improvement scale (CGI-I), which represents a less granular assessment by the site investigators using a seven-point scoring (one=“very much improved” to seven=“very much worse”), was not met.



https://www.anavex.com/post/anavex-life-sciences-provides-update-rett-syndrome-program
mikebrisy
4 months ago

@Nnyck777 you’d hardly consider the result a failure based on a superficial reading of the company’s release.

However, the market is unequivocal, with SP down a brutal 35%.

I wonder if the small group of patients on compassionate use will now switch to DAYBUE? Is it even ethical to keep them on the Anavex product?

(Note: I have already removed the “Anavex discount” from my valuation update in December, but with so few candidates in development this does indeed further shore up the competitive position of DAYBUE. Happy New Year!)

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