Fragile X Syndrome Drug Validation Initiative (FRAXA-DVI)

Fragile X Syndrome Drug Validation Initiative (FRAXA-DVI)

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential new fragile X treatments. FRAXA has funded FRAXA-DVI for $50,000 or more per year since 2012.

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Neuren’s trofinetide successful in Phase 2 clinical trial in Fragile X

Neuren’s trofinetide successful in Phase 2 clinical trial in Fragile X

We are pleased to share great news adapted from Neuren’s press release: Neuren’s phase 2 trial has successfully established proof of concept and provides a strong rationale for Neuren to move forward with developing trofinetide for Fragile X syndrome. In this initial small 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, observed in both clinician and caregiver assessments. After only 28 days of treatment, improvements were seen across core symptoms of Fragile X syndrome, including higher sensory tolerance, reduced anxiety, better self-regulation and more social engagement. No serious adverse events were reported. Positive Results in Fragile X Syndrome and Rett Syndrome Beneficial effects of trofinetide have now been observed in both Fragile X syndrome and Rett syndrome. Neuren announced encouraging results of their phase 2 trofinetide trial in Rett Syndrome last year. Trofinetide Tested at FRAXA’s Drug Validation

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

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

New Zealand-based biotech Neuren Pharmaceuticals has announced impressive preclinical results in the Fragile X mouse model with two of their lead compounds, NNZ-2566 and NNZ-2591. These compounds are examples of a new class of drugs based on insulin-like growth factors (IGF-1). IGF analogs are currently considered the most promising approach for treating Rett Syndrome, a fatal genetic disorder that affects only girls, and one of the other leading genetic models for the study of autism (along with Fragile X). The surprising news is that FRAXA researchers have found that this treatment strategy works even better in fragile X knockout mice than in Rett syndrome mice! FRAXA’s strategy is to find and target the critical bottlenecks which block the way to development of treatments. In this case, a small pharma company with interesting compounds approached FRAXA to get it tested in Fragile X. The Drug Validation Initiative, or DVI, a testing

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