trofinetide

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

FRAXA Research Foundation Clinical Trials • Research Updates , , ,
Neuren’s Tofinetide Successful in Phase 2 Clinical Trial in Fragile X
One of the outcome measures - the new Fragile X Syndrome Rating Scale - showing positive results. Blue: placebo; Yellow: low-dose trofinetide; Green: high-dose trofinetide 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 FragileRead more

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

Michael Tranfaglia, MD Clinical Trials • Research Updates , ,
Neuren’s NNZ-2566 Shows Clinical Benefit in Rett Syndrome Trial
Promising Results in Phase 2 Clinical Trial 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. The results of the trial are interesting, in that improvement was seen a Rett syndrome-specific rating scale compared to placebo, and there was also improvement noted on the CGI-I (Clinical Global Impression of Improvement) and Caregiver Top 3 Concerns. However, there was no effect seen on ABC scores (Aberrant Behavior Checklist) compared to placebo. Many in the Fragile X field have noted the inadequacies of the ABC; indeed, it was never designed or intended to be an outcome measure for clinical trials. In this case, a Rett-specific rating scale called the Motor-Behavior Assessment (MBA) showed a statistically significant and clinically meaningful treatment effect at the highest dose of the Neuren compoundRead more

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

FRAXA Research Foundation Research Updates , , ,
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 Trofinetide. 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 facility in Chile funded by FRAXA, wasRead more