CAMBRIDGE, Mass. and COPENHAGEN, Denmark — Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced positive results from its completed Phase 2 multiple ascending dose (MAD) portion of the CL-101 study of sutacimig for the prophylactic treatment of Glanzmann thrombasthenia...
treatment News
OSLO, Norway — Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for GLIX1, a first-in-class small molecule targeting DNA repair vulnerabilities in glioblastoma and other solid tumors....
OSLO, Norway — Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for GLIX1 for the treatment of malignant glioma, a category of devastating brain cancers that includes glioblastoma. Significant clinical benefit beyond current therapies. The...
OSLO, Norway — Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation (ODD) for GLIX1, the company’s lead molecule, for the treatment of glioma, one...
LONDON, UK – Hemogenyx Pharmaceuticals plc (LSE: HEMO) is pleased to announce that the second patient has been successfully treated as part of the Company’s ongoing Phase I clinical trial of HG-CT-1, its proprietary CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia (“R/R AML”) in...
LONDON, UK – Hemogenyx Pharmaceuticals plc (LSE:HEMO) is pleased to announce that it has been informed by the U.S. Federal Food and Drug Administration (FDA) that it has lifted the clinical hold on the Investigational New Drug (IND) application for HEMO-CAR-T for the treatment of acute myeloid leukemia (AML). The FDA...
Second Patient Treated with HG-CT-1 CAR-T Therapy Successfully Passes Initial Safety Evaluation LONDON, UK – Hemogenyx Pharmaceuticals plc is pleased to announce that the second patient has been successfully treated in the ongoing Phase I clinical trial of HG-CT-1, the Company’s proprietary Chimeric Antigen Receptor T-cell (CAR-T) therapy for relapsed/refractory...
Among patients with hemophilia A who receive primary prophylaxis, quality of life (QoL) may be lower compared with the general population, according to research published in Haemophilia. Among patients with severe hemophilia, prophylaxis with clotting factor replacement therapy is the standard treatment, though patients vary in when they initiate therapy. In...
CHARLOTTESVILLE, Va. – HemoShear Therapeutics, Inc., a clinical stage company developing treatments for rare metabolic disorders, today announced that the first two patients have been dosed in the HERO (HElp Reduce Organic Acids) Phase 2 clinical trial of HST5040, an oral small molecule drug being investigated for the treatment of patients with methylmalonic...
FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that Hepatology published an analysis demonstrating a statistically significant improvement in six-year event-free and transplant-free survival in patients with Alagille syndrome (ALGS) treated with LIVMARLI® (maralixibat) oral solution when compared with a natural history control group (p<0.0001). The analysis...