Neuren Pharmaceuticals (ASX:NEU) has announced positive outcomes from an End of Phase 2 Meeting with the US Food and Drug Administration (FDA) to discuss proposals for the first ever pivotal clinical trial program in Phelan-McDermid syndrome (PMS) with the FDA. There are no medications approved for PMS, which has severe quality of life impacts on those living with it, as well as on parents and siblings. At the FDA meeting, alignment was reached on key aspects of the NNZ-2591 Phase 3 program, with Neuren to submit further information to confirm endpoints for the primary efficacy assessment.
- Constructive End of Phase 2 meeting with FDA, discussing proposals for the first ever pivotal clinical trial program for PMS
- Alignment reached on key aspects of NNZ-2591 Phase 3 trial program:
- Single randomised, double-blind, placebo-controlled trial of treatment for 13 weeks in children aged 3 to 12 years with PMS
- Participants may continue into an open-label extension study continuing treatment until commercial launch
- One active treatment group versus placebo, with target dose equivalent to dose tested in Phase 2 trial
- Less burdensome safety monitoring plan than Phase 2, subject to FDA review of the final protocol
- Neuren to submit further information to FDA to confirm endpoints for the primary efficacy assessment
- Preparations for Phase 3 advancing, including selection of service providers, identification of potential trial sites and manufacturing of NNZ-2591 supplies.
Neuren Pharmaceuticals CEO Jon Pilcher said: “We are pleased with the outcomes of a very collaborative meeting with the FDA and are eager to move forward in our mission to develop NNZ-2591 as a first approved treatment for Phelan-McDermid syndrome, which has an overwhelming unmet need.”