Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.
Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.
Taigen Biotechnology Co. Ltd. and its Taigen Biopharmaceuticals subsidiary have entered into an exclusive in-licensing agreement with Insilico Medicine Inc. for ISM-4808, a prolyl hydroxylase domain (PHD) inhibitor.
The EMA has rejected the Alzheimer’s disease therapy Kisunla (donanemab) from Eli Lilly and Co., saying the benefits of the anti-amyloid antibody do not outweigh the risks of edema and hemorrhage in the brain, known as amyloid-related imaging abnormalities (ARIA).
Recordati SpA is shelling out $825 million up front for global rights to Enjaymo (sutimlimab), the only therapy approved for treating the rare disease cold agglutinin disease. In the deal with Sanofi SA, which won U.S. FDA approval of the antibody drug in 2022, the Italian pharma agreed to pay up to $250 million more should net sales reach certain thresholds.
Oxford University Innovations Ltd. has divulged Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of anemia, ischemia, cardiovascular disorders, inflammatory disorders, irritable bowel syndrome, chronic kidney disease, Parkinson’s disease and Alzheimer’s disease, among others.
RNA Therapeutics Inc. has received a written response from the FDA to its pre-IND application questions regarding the development of its lead asset, RNAT-89 (BLA-761423), an mRNA LNP formulation to express darbepoetin.
After hearing two conflicting presentations of the safety and efficacy of Geron Corp.’s imetelstat, the U.S. FDA’s Oncology Drugs Advisory Committee (ODAC) voted 12-2 March 14 that the drug’s benefit outweighed its risks as a treatment for transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes in patients who have failed or no longer respond to erythropoiesis stimulating agents (ESAs), or who are not eligible for ESA treatment.
Seoul-based Genexine Inc. filed a BLA to gain domestic approval of its follow-on biologic for chronic kidney disease (CKD)-induced anemia, Efesa (efepoetin alfa, GX-E4), to South Korean health regulators on Jan. 25.
