South Korean artificial intelligence-based drug developer Pharos Ibio Co. Ltd. said that the Ministry of Food and Drug Safety (MFDS) granted an orphan drug designation for PHI-101, a second-line therapy for acute myeloid leukemia (AML).
Vor Biopharma Inc.’s trem-cel, a stem cell transplant designed to block the toxicity from cancer treatments, has produced some positive early stage results, including delayed relapse in patients. Phase I/IIa study data showed participants with relapsed/refractory acute myeloid leukemia benefited from trem-cel followed by treatment with Pfizer Inc.’s antibody-drug conjugate cancer fighter Mylotarg (gemtuzumab ozogamicin).
South Korean artificial intelligence-based drug developer Pharos Ibio Co. Ltd. said that the Ministry of Food and Drug Safety (MFDS) granted an orphan drug designation for PHI-101, a second-line therapy for acute myeloid leukemia (AML).
Genetic or pharmacologic inhibition of casein kinase 1α (CK1α) has proven effective in suppressing the proliferation of acute myeloid leukemia (AML) cell lines with wild-type TP53. Researchers from Bristol Myers Squibb Co. recently presented the discovery and preclinical characterization of BMS-986397, a cereblon E3 ligase modulatory drug (CELMoD) designed as a molecular glue degrader of CK1α.
Hemogenyx Pharmaceuticals plc has developed a new and improved version of its bispecific antibody CDX for the treatment of relapsed/refractory acute myeloid leukemia, a subset of acute lymphoblastic leukemia, and potentially for conditioning in bone marrow transplantations.
Both casein kinase 1α (CK1α) and zinc finger protein Helios (IKZF2) are among the targets most recently evaluated for the treatment of acute myeloid leukemia (AML). A growing number of molecules against these targets acting as degraders or inhibitors are actively being investigated.
Data from an interim analysis have convinced Syros Pharmaceuticals Inc. to stop enrollment in its phase II study of treating newly diagnosed acute myeloid leukemia (AML) and retinoid alpha receptor agonist gene overexpression. Analysts seemed unconcerned about the repercussions for Syros’ phase III study in myelodysplastic syndrome.
The possibility of a 2025 approval looks to be off the table for Actinium Pharmaceuticals Inc.’s Iomab-B, at least in the U.S. In a move that H.C. Wainwright analyst Joseph Pantginis dubbed “a major surprise,” the FDA has requested a head-to-head study demonstrating overall survival before it will consider approving the radiotherapy candidate for use in patients with active relapsed or refractory acute myeloid leukemia.
Plexium Inc. has reported the discovery and preclinical characterization of PLX-3618, a novel monovalent direct degrader of BRD4 being developed for the treatment of cancer.
University of Regensburg has described water-soluble FLT3 (FLK2/STK1) inhibitors and their prodrugs reported to be useful for the treatment of acute myeloid leukemia (AML).
