Tag: treatment options

WALTHAM, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI^® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.¹

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®.

The combined company will operate under the name “ImageneBio, Inc.” and will begin trading on Nasdaq under the ticker symbol “IMA” at market open on Monday, July 28, 2025

The combined company will operate under the name “ImageneBio, Inc.” and will begin trading on Nasdaq under the ticker symbol “IMA” at market open on Monday, July 28, 2025

COPENHAGEN, Denmark, July 25, 2025 - Evaxion A/S (NASDAQ: EVAX) (“Evaxion”), a clinical-stage TechBio company specializing in developing AI-Immunology™ powered vaccines, will be presenting two-year clinical efficacy data from its phase 2 trial with lead compound EVX-01 at the European Society for Medical Oncology (ESMO) Congress 2025 to be held in Berlin, Germany, from October 17-21, 2025.

PARIS, FRANCE, 24 July 2025 - Ipsen announced today that the European Commission has approved Cabometyx^® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues.

Wuppertal, Germany, July 24, 2025 - Aicuris Anti-infective Cures AG today announced the completion of patient enrollment in its pivotal Phase 3 trial, PRIOH-1, evaluating pritelivir for the treatment of refractory and/or treatment resistant herpes simplex virus (HSV) infections in immunocompromised patients. The company’s lead candidate is a small molecule specifically inhibiting the viral helicase-primase complex, which represents a novel mechanism of action distinct from current HSV therapies.

Hennigsdorf/ Berlin, July 23, 2025 – 4TEEN4 Pharmaceuticals GmbH today announced that the first patient has been dosed in a Phase 1b/2a PROCARD1 clinical trial evaluating its monoclonal antibody, procizumab, in patients with shock caused primarily by cardiogenic events. Shock is a life-threatening condition characterized by a sudden breakdown in circulatory function due to end-stage disease or acute events, which often leads to organ failure. With only symptomatic treatment options available, shock is associated with a high mortality rate of over 50%. The Phase 1b/2a trial is designed to identify a target dose for procizumab in preparation for further clinical development. In addition, the trial will provide signals of efficacy. In a Phase 1 study in healthy volunteers, procizumab was well tolerated.