Latest Rare Diseases News

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.

Neuren Pharmaceuticals has added SYNGAP1-related disorder to its NNZ-2591 development program following encouraging pre-clinical findings, targeting a rare condition with no approved treatments.

Neuren Pharmaceuticals reports a 16% rise in Q2 2025 royalties driven by Acadia’s DAYBUE sales growth and expanding patient base in the US. Full-year sales guidance remains strong with European market entry on the horizon.

Opyl Limited has secured three key partnerships validating its AI-driven TrialKey platform, completed a strategic review to boost AI capabilities, and strengthened its balance sheet with a $1.5 million capital raise and Bitcoin investment.

Cynata Therapeutics is advancing three pivotal clinical trials targeting acute Graft versus Host Disease, osteoarthritis, and kidney transplantation, with major results expected through early 2026. The company maintains a solid cash position supporting its clinical pipeline and strategic growth.

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.