Latest Neurodevelopmental Disorders News

BlinkLab has completed onboarding its tenth and final clinical site for its pivotal FDA 510(k) trial of an AI-powered autism diagnostic aid, setting the stage for patient enrolment to begin this month.

Neuren Pharmaceuticals’ partner Acadia Pharmaceuticals is seeking a re-examination of the European Medicines Agency’s refusal to approve trofinetide for Rett syndrome, aiming to overturn the setback and bring the treatment to European patients.

Neuren Pharmaceuticals reports a 15% rise in 2025 royalties from its partner Acadia’s DAYBUE sales, with strong 2026 guidance signalling further growth. The launch of a new powder formulation and ongoing regulatory efforts in Europe and Japan add to the momentum.

Neurotech International has reported positive long-term safety results for its lead drug candidate NTI164, bolstering its path towards regulatory approval and chronic use in paediatric neurological disorders.

Neuren Pharmaceuticals has initiated a discretionary on-market share buy-back program of up to 5%, citing a strong cash position and undervaluation relative to internal and analyst assessments.

Neuren Pharmaceuticals has dosed the first patient in its groundbreaking Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome, a rare genetic disorder with no approved therapies. The trial marks a significant step forward for the PMS community and rare disease drug development.

The US Congress has reauthorized the Rare Pediatric Disease Priority Review Voucher program through September 2029, a move that could significantly benefit Neuren Pharmaceuticals as it advances its rare disease drug candidates.

Neuren Pharmaceuticals has received detailed feedback from the US FDA on its clinical development plans for NNZ-2591 targeting hypoxic ischemic encephalopathy and Pitt Hopkins syndrome, paving a clearer path forward despite some delays.

Acadia Pharmaceuticals has received a negative vote from the European Medicines Agency’s committee on its application to market trofinetide for Rett syndrome in the EU, prompting plans for a re-examination request.

Neurotech International has secured ethics approval to launch its Beyond Harmony Phase 3 clinical trial of NTI164 for autism spectrum disorder, marking a key step toward regulatory submissions in Australia and the US.

BlinkLab Limited has delivered strong diagnostic results in its U.S. autism pilot study, exceeding FDA performance thresholds and setting the stage for a pivotal 510(k) trial. The company also expanded its clinical trial network and nears completion of its European ADHD study.

PYC Therapeutics has reported significant progress across its four RNA therapeutic programs targeting rare genetic diseases, supported by a strong cash position of $121 million at the end of 2025. Key clinical milestones and regulatory plans set the stage for upcoming trial readouts and potential market entry.