Trofinetide Phase 3 Trial in 180 girls with Rett syndrome


Neuren Pharmaceuticals is developing what will hopefully be the first real drug for Rett Syndrome. They partnered with Acadia Pharmaceuticals to conduct a phase 3 trial in Rett syndrome.


This Phase 3 randomized, double-blind placebo-controlled study will evaluate Trofinetide and placebo in about 180 girls with Rett syndrome. It will measure the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression of Improvement (CGI-I), a physician assessment, as co-primary efficacy endpoints.


Neuren earlier carried out a phase 2 trial in teenagers and adults aged 16 to 45 years. Laster Neuren also held a Phase 2 double-blind placebo-controlled clinical trial in girls aged 5 to 15 years who suffer from Rett syndrome. Phase 2 showed significant clinical improvement. The improvements were demonstrated on the girls, and also was reported by the caregiver. The final report showed substantial improvements on the Rett Syndrome Behavior Questionnaire (RSBQ; P = .042), the Clinical Global Impression Scale-Improvement (CGI-I; P = .029), and the Rett Syndrome Clinician Domain-Specific Concerns-Visual Analog Scale (RTT-DSC; P = .025).


Daniel Glaze, a pediatric neurologist, and director of the Blue Bird Circle Rett Center at Baylor College of Medicine (Texas Children’s Hospital) said: “The data reported in this study show that females treated with Trofinetide experienced lessened neurobehavioral impairments including social communication deficits, anxiety-like behavior, and mood dysregulation. These are very promising data for the Rett community that is currently without any FDA-approved treatment option.”


About Trofinetide

Trofinetide (NNZ-2566) is a Neuropeptides - small protein-like molecules (peptides) used by neurons to communicate with each other. Trofinetide is a tripeptide (a peptide derived from three amino acids) with sequence Gly-Pro-Glu formed by enzymatic cleavage of the growth factor IGF-1.


Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have given Trofinetide orphan drug designation for treating Rett syndrome. Also, the FDA has granted Trofinetide fast track designation.

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