Trofinetide Clinical Trial Results Published

New Zealand-Based Biotech Neuren Pharmaceuticals Has Published Results of Phase 2 Fragile X Clinical Trial

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Research just published in the peer-reviewed journal ScienceDirect reports on a phase 2 double-blind placebo-controlled clinical trial of trofinetide in  Fragile X syndrome.

Trofinetide, administered at 35 mg/kg BID and 70 mg/kg BID to adolescent and adult males with Fragile X syndrome (FXS), was shown to be generally safe and was well-tolerated. It also showed preliminary evidence of efficacy for the 70 mg/kg BID group compared with placebo.

What is Trofinetide?

Trofinetide is an example of a new class of drugs based on insulin-like growth factors (IGF-1). IGF analogs are considered the most promising approach for treating Rett Syndrome, a fatal genetic disorder that affects only girls. Researchers at FRAXA-DVI, our preclinical testing facility in Chile, found that this treatment strategy works as well in Fragile X knockout mice as in Rett syndrome mice! Neuren used this data to launch their Fragile X treatment program.

Promising New Design for Fragile X Clinical Trials

The study makes two other important contributions to treatment development research for FXS: it describes an analytical approach that should be considered as a viable alternative to traditional approaches for early phase studies, and it provides evidence for the utility of two novel outcome measures that are FXS-specific.

Explore Current Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.

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