FRAXA Drug Validation Initiative (FRAXA-DVI)

December 13, 2025

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing to validate investigational and repurposed compounds for Fragile X.

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Trofinetide Clinical Trial Results Published

May 29, 2020

Neuren Pharmaceuticals has published results of a Phase 2 Fragile X Clinical Trial of Trofinetide ih Fragile X syndrome. This trial tested a new design which can be used in future trials.

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neuren Fragile X trial result graph

Neuren’s Tofinetide Successful in Phase 2 Clinical Trial in Fragile X

December 7, 2015

In this initial trial with a relatively short treatment period, trofinetide was very well tolerated, with the high dose (70 mg/kg twice daily) demonstrating a consistent pattern of clinical improvement.

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Fragile X compound from Neuren

Neuren’s NNZ-2566 Shows Clinical Benefit in Rett Syndrome Trial

November 26, 2014

This isn’t a Fragile X trial, but the Neuren compound, NNZ-2566, that is in trials now for Fragile X has shown significant positive effects in a Phase 2 trial for Rett syndrome.

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IGF-1

Fragile X Treatment Strategy Emerges from FRAXA Research: IGF-1

July 30, 2013

Neuren’s Trofinetide, part of a promising IGF-1 drug class, showed standout results in Fragile X mice—outperforming Rett syndrome models.

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FRAXA Funded Research

Current Research Grants (37)