Neuren Advances Phase 3 Trial for Phelan-McDermid Syndrome

Neuren Pharmaceuticals (ASX: NEU) has opened its first U.S. site to begin a Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome, a rare neurological condition that currently lacks approved therapies. The randomized, double-blind, placebo-controlled study will enroll around 160 children aged 3–12, with an optional 12-month open-label extension. The trial design was finalized in consultation with the FDA, and funding will be supported entirely by Neuren’s existing cash reserves.

PMS, caused by genetic changes affecting the SHANK3 gene, leads to developmental delays, intellectual disabilities, autism symptoms, motor impairments, and epilepsy. With no current therapies, the unmet medical need is significant.

Neuren is dedicated to developing treatments for serious childhood-onset neurological disorders. Its approved drug DAYBUE™ (trofinetide) addresses Rett syndrome, while NNZ-2591 has already shown positive Phase 2 results in PMS, Pitt Hopkins, and Angelman syndromes

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