Tag: gene expression

   
- Successfully initiated enrollment in advanced Phase 2 ovarian cancer trial

CAMBRIDGE, Mass., Aug. 08, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.^® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced that the company granted non-statutory stock options to new employees. Fulcrum granted stock options to purchase shares of the company’s common stock pursuant to the company’s 2022 Inducement Stock Incentive Plan, as amended, or the plan, as an inducement material to the new employees entering into employment with Fulcrum in accordance with Nasdaq Listing Rule 5635(c)(4).

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®.