Neuren Pharmaceuticals Ltd (ASX: NEU) shares are having a strong start to the week.
In morning trade, the ASX 200 healthcare stock is up over 4% to $14.63.
Why is this ASX 200 healthcare stock charging higher?
The catalyst for this gain has been the release of an announcement this morning in relation to the US Food and Drug Administration (FDA).
According to the release, Neuren has reported positive outcomes from an End of Phase 2 Meeting with the US FDA to discuss proposals for the first ever pivotal clinical trial program in Phelan-McDermid syndrome (PMS).
Management notes that this is a big positive given that 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. This will see Neuren submit further information to confirm endpoints for the primary efficacy assessment.
Phase 3 trial
The ASX 200 healthcare stock notes that the single pivotal Phase 3 trial will be a randomised, double-blind, placebo-controlled trial of treatment for 13 weeks in children aged 3 to 12 years with PMS.
Participants may then continue into an open-label extension study continuing treatment until commercial launch.
There will be one active treatment group versus placebo, with a target dose equivalent to the dose that was tested in the Phase 2 trial.
Based on the safety data from the Phase 2 clinical trial, Neuren proposed a less burdensome safety monitoring plan for the Phase 3 and open label extension trials. This was considered reasonable by the FDA, subject to review of the final protocol.
Management highlights that as this study is the first ever pivotal clinical trial in PMS, there is no precedent for efficacy assessment.
Nevertheless, primary efficacy endpoints for the Phase 3 trial were considered in depth at the meeting, with a range of potential options discussed. The FDA has requested that Neuren submit further existing information before agreeing the final selection of the endpoints.
It is now compiling the further information on efficacy endpoints and updating the Phase 3 trial protocol. Each of these will be submitted to the FDA for review. In the meantime, Neuren is well advanced in the selection of service providers for the Phase 3 program and has commenced identification of potential trial sites.
The ASX 200 healthcare stock's CEO, Jon Pilcher, was pleased with how the meeting went. He commented:
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.
