Neuren Pharmaceuticals (ASX: NEU) has been ranted a Type C meeting to discuss the primary efficacy endpoints its planned pivotal Phase 3 clinical trial programme for NNZ-2591 to treat Phelan-McDermid syndrome by the US Food and Drug Administration (FDA).
Phelan-McDermid syndrome (PMS) is a rare genetic condition caused by a deletion or other structural change of the terminal end of chromosome 22 in the 22q13 region or a disease-causing (pathogenic) variant of the SHANK3 gene.
The company previously announced the positive outcomes from a Type B End of Phase 2 Meeting, at which alignment with FDA was reached on the other key features of the Phase 3 clinical trial program. A Type C meeting was considered by FDA as the best forum for completion of the remaining efficacy endpoints discussion. The meeting will take place face-to-face in early April.
“We look forward to another constructive discussion with FDA. Having a confirmed Type C meeting now establishes a clear timetable and in parallel we are continuing all our preparations, planning for mid-year commencement of the first ever Phase 3 trial for children with Phelan-McDermid syndrome," CEO, Jon Pilcher, said.
Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. Recognising the urgent unmet need, all programs have been granted “orphan drug” designation in the United States. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases.
DAYBUE (trofinetide) is approved by the US Food and Drug Administration (FDA) and Health Canada for the treatment of Rett syndrome. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide.
Neuren’s second drug candidate, NNZ-2591, is in development for multiple neurodevelopmental disorders, with positive results achieved in Phase 2 clinical trials in Phelan-McDermid syndrome, Pitt Hopkins syndrome and Angelman syndrome.
