Latest Neurological Disorders News

A single exploration name blew the doors off the leaderboard, while two well-known uranium plays and a Nevada explorer sank hard. Behind the noise: deals got closer to completion, big raisings reset prices, and several “good news” gaps didn’t survive the week.

A single diagnostics update sparked a breakout week, while a handful of biotech names sold off hard despite busy newsflow. Capital raisings, takeover steps and US market pushes kept healthcare in motion heading into mid-February milestones.

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.

A handful of healthcare names did the heavy lifting this week, driven by FDA wins, guideline upgrades, and big shifts in sales and cashflow. The flip side: several clinical-stage stocks fell hard as investors stayed picky about near-term proof and funding.

Neurotech International has secured a Rare Pediatric Disease Designation from the FDA for its lead drug NTI164 targeting Rett syndrome, alongside publishing promising clinical data and raising $8.7 million to fuel further development.

Neuren Pharmaceuticals’ partner Acadia projects DAYBUE sales to reach around US$700 million by 2028, driven by US market expansion and international approvals. The launch of a new formulation and ongoing clinical trials in Japan underpin this optimistic outlook.

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.

Neurotech International has locked in $4 million through a share placement to advance its promising paediatric neurological drug, NTI164. The raise, supported by both new and existing investors including company directors, aims to fund critical clinical and regulatory milestones.