Latest Neurodevelopmental Disorders News

PYC Therapeutics has announced promising pre-clinical results for its RNA-based drug candidate PYC-002, targeting the genetic root of Phelan-McDermid Syndrome, with clinical trials expected to begin in 2026.

Neuren Pharmaceuticals has been granted a US patent for NNZ-2591 to treat Pitt Hopkins syndrome, bolstered by promising Phase 2 clinical trial results and FDA orphan drug status.

Neuren Pharmaceuticals posted record 2024 financials driven by DAYBUE royalties and advanced NNZ-2591 into Phase 3 trials, while launching a $50 million share buy-back amid volatile market sentiment.

Neuren Pharmaceuticals has approved a significant long-term equity incentive plan tied to critical clinical and regulatory milestones for its drug candidate NNZ-2591, reinforcing management’s commitment to shareholder value.

Neuren Pharmaceuticals has received official FDA minutes confirming the primary endpoints for its pivotal Phase 3 trial of NNZ-2591 targeting Phelan-McDermid syndrome, reinforcing the positive signals from earlier studies.

Neuren Pharmaceuticals reports a robust 11% rise in DAYBUE™ US net sales for Q1 2025, alongside a 17% jump in royalty income, setting the stage for an anticipated European market entry in early 2026.

BlinkLab Limited has raised A$7.66 million through a well-supported placement to accelerate FDA regulatory trials for its autism and ADHD diagnostic platforms, leveraging its innovative smartphone-based technology.

Neurotech International has inked a development deal with RH Farma to produce a pharmaceutical-grade cannabinoid drug targeting pediatric neurodevelopmental disorders, leveraging advanced European extraction technology.

BlinkLab has secured North Shore Pediatric Therapy as the second US clinical site in its autism diagnostic study, enhancing recruitment and data quality with a leading Chicago-based specialist.

Neuren Pharmaceuticals has received a significant cash inflow of approximately US$50 million following the sale of a Priority Review Voucher linked to its Rett syndrome treatment. This milestone underscores the growing value of Neuren’s neurological drug portfolio and its partnership with Acadia Pharmaceuticals.

Neuren Pharmaceuticals has secured a pivotal FDA Type C Meeting to discuss primary efficacy endpoints for its Phase 3 trial of NNZ-2591 targeting Phelan-McDermid syndrome, advancing its rare disease drug development.

Zelira Therapeutics has secured a fourth funding tranche of US$681,000, advancing its HOPE® Autism Spectrum Disorder FDA trial process and bringing total funding to US$3.25 million. The company is progressing clinical validation and product development, including the transformation of Zenivol® into a capsule formulation.