Tag: preclinical

– New patent enhances market position in Europe

MELBOURNE, Australia and INDIANAPOLIS, July 30, 2025 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, “Telix”) today advises that it will release its financial results for the half-year ended 30 June 2025 on Thursday 21 August 2025.

Zurich, July 29, 2025 – MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory and infectious diseases, announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA's ODD, underscoring MP1032's potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side effects like osteoporosis, growth stunting, and metabolic issues.

Allschwil, Switzerland, July 29, 2025

SANTA ANA, Calif., July 28, 2025 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced the presentation of a poster entitled, “Mechanism of Action of Troculeucel (Non-genetically Modified Natural Killer Cells with Enhanced Cytotoxicity) in Alzheimer's Disease Confirmed by Corresponding Phase I Biomarker Data” at the Alzheimer’s Association International Conference 2025 (“AAIC 2025”) held in Toronto, Canada and online from July 27-31, 2025.

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®.