FYI:I found the following paragraph in relation to upcoming efficacy endpoints meeting very reassuring: Patrick DaviesChairJon PilcherCEOFor NNZ-2591 "We were excited that during 2024 thepositive Phase 2 trial results for Pitt Hopkins syndromeand Angelman syndrome were consistent with thePhelan-McDermid syndrome results validating our thesisthat NNZ-2591 can potentially have a broad impact onneurodevelopmental conditions. We were very pleasedwith the outcome of our End of Phase 2 Meeting with theFDA for Phelan-McDermid syndrome enabling us to movestraight to Phase 3 with a similar program to the successfulDAYBUE program in Rett syndrome. Efficacy endpoints arealways the most complex issue when you are leading theway as a first treatment and there is no precedent to follow.We went through a similar journey with FDA to align onefficacy endpoints for Rett syndrome. Overall we believethat the success of DAYBUE de-risks the NNZ-2591 programsgiven the similarities in clinical profile, scientific rationale,trial design and endpoints. Building on the Rett syndromeexperience we are eager to embark on the first ever Phase 3trial in Phelan-McDermid syndrome aiming to provide a firsttreatment option to that community." Kens
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(20min delay)