Latest Rare Diseases News

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.

Dimerix Limited’s lead drug candidate DMX-200 has been granted Orphan Drug Designation in Japan for treating Focal Segmental Glomerulosclerosis (FSGS), enhancing its regulatory and commercial prospects in a key market.

Telix Pharmaceuticals has secured FDA agreement on a resubmission plan for its glioma imaging agent TLX101-CDx, aiming for a Q4 2025 filing that includes additional efficacy data. The FDA signals likely expedited review, underscoring the unmet medical need.

Percheron Therapeutics posted a 25% increase in net loss for FY2025 amid termination of its Duchenne Muscular Dystrophy programs. The company pivots strategically to oncology with the in-licensing of HMBD-002, a monoclonal antibody targeting VISTA, aiming to initiate Phase II trials in 2026.

Clinuvel Pharmaceuticals has reported a robust financial year ending June 2025, marked by solid revenue growth and sustained profitability, while advancing its clinical pipeline and maintaining a strong balance sheet.

Invion Limited has affirmed its compliance with ASX disclosure rules following the US FDA’s granting of Orphan Drug Designation for its anal cancer treatment candidate INV043. The company detailed the timing and verification process behind its announcement.

Dimerix Limited reported a 22% reduction in its FY2025 loss to $13.25 million, driven by strong licensing revenues and advancing its pivotal Phase 3 clinical trial for DMX-200. The company secured new global partnerships valued at up to AU$1.4 billion, bolstering its cash reserves and commercial prospects.

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.

Cogstate Ltd has reported a strong FY25 with 22% revenue growth and a near doubling of profit before tax, declaring its first fully franked dividend and preparing to launch its inaugural AI-powered product in FY26.

Cogstate Limited has reported a landmark FY25 with revenue up 22% and net profit before tax nearly doubling, driven by a surge in clinical trials contracts and strategic innovation. The company also declared its maiden fully franked dividend and outlined ambitious growth plans including AI product launches and an expanding Medidata partnership.

Invion Limited secures a pivotal FDA Orphan Drug Designation for its lead candidate INV043, targeting anal cancer with promising preclinical results and a fast-tracked development path.