Member Contact: Jon Pilcher
Position: Chief Executive Officer
Industry: Biotech & Pharmaceuticals
Neuren is developing new therapies for 5 debilitating childhood disorders which currently have no approved therapies, utilizing synthetic analogs of neurotrophic peptides that occur naturally in the brain.
The lead drug candidate Trofinetide is currently in a Phase 3 clinical trial for Rett syndrome and has completed a Phase 2 clinical trial in Fragile X syndrome. The programs have each received Fast Track designation by the US Food and Drug Administration and Orphan Drug designation in both the United States and the European Union. Neuren has granted an exclusive license to ACADIA Pharmaceuticals Inc. for the development and commercialization of trofinetide in North America, whilst retaining all rights outside North America.
In addition, Neuren is preparing for Phase 2 clinical trials of its second drug candidate NNZ-2591 for Phelan-McDermid, Angelman and Pitt Hopkins syndromes, each of which has received Orphan Drug designation in the United States.