Latest Neurodevelopmental Disorders News

Neuren Pharmaceuticals has secured Health Canada approval to expand its pivotal Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome into Canadian sites, complementing ongoing US enrolment.

Neuren Pharmaceuticals’ partner Acadia secures FDA approval for DAYBUE STIX, a new powder formulation of trofinetide offering greater flexibility for Rett syndrome patients. This milestone expands treatment options and sets the stage for increased royalty revenue in 2026.

Neuren Pharmaceuticals reported a milestone quarter with DAYBUE net sales reaching US$101.1 million, driving a 24% jump in royalty income. The expanding patient base and community physician uptake signal strong growth ahead.

BlinkLab has completed its U.S. autism pilot study with strong diagnostic accuracy and expanded its pivotal FDA trial to seven leading research centers, positioning itself for regulatory submission in late 2026.

Neurotech International has initiated an Authorised Prescriber program for its lead therapy NTI164, expanding access for children with neurodevelopmental disorders in Australia. The company also secured a Rare Pediatric Disease Designation from the US FDA for NTI164 in Rett Syndrome, marking a key regulatory milestone.

PYC Therapeutics has reported significant progress across its four RNA therapeutic programs targeting genetic diseases, alongside a strong cash runway supporting ongoing development.

Neuren Pharmaceuticals has secured FDA Fast Track designation for its drug NNZ-2591 targeting Phelan-McDermid syndrome, accelerating its path through clinical development. This milestone underscores the urgent need for treatments in rare neurodevelopmental disorders.

PYC Therapeutics has reported encouraging pre-clinical results for its RNA therapy candidate PYC-002 targeting Phelan-McDermid Syndrome, supporting plans to enter human trials in 2026. The data demonstrate safety, brain distribution, and gene expression restoration in key disease regions.

Neurotech International has secured a key FDA designation for its lead drug NTI164, advancing its potential to treat Rett syndrome with regulatory and financial benefits.

Invex Therapeutics has discontinued exclusive negotiations to acquire a rare neurological disease company due to board instability triggered by a shareholder requisition notice. Despite this setback, the company remains committed to diversifying its neurological treatment portfolio.

Neuren Pharmaceuticals reported a robust half-year with an 88% increase in net profit, driven by rising royalties from DAYBUE™ sales and progress in its NNZ-2591 clinical program. The company also completed a $39.6 million share buy-back, underscoring confidence in its growth trajectory.

Neuren Pharmaceuticals has initiated the first US investigational site for its pivotal Phase 3 trial of NNZ-2591 targeting Phelan-McDermid syndrome, marking a significant milestone in addressing this rare neurodevelopmental disorder.