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.
JAK2 inhibitors (JAK2i) are the standard treatment for myelofibrosis (MF), offering symptom relief and reducing spleen size. However, all FDA-approved JAK2i are type I inhibitors, which fail to eliminate the mutant MPN clone, leading many patients to treatment discontinuation.
Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.
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.
Novartis AG’s monoclonal antibody, ianalumab (VAY-736), when added to standard-of-care eltrombopag, extended disease control of primary immune thrombocytopenia (ITP) by 45%, according to data presented Dec. 9 during a late-breaker abstract session at the 67th American Society of Hematology’s annual meeting in Orlando, Fla.
ENL-YEATS is an epigenetic reader that sustains transcriptional programs essential for AML, whereas FLT3 mutations, present in approximately 30% of patients, drive malignant proliferation. Dual inhibition of ENL-YEATS and FLT3 may therefore more effectively disrupt complementary drivers of leukemogenesis than FLT3 targeting alone.
Exicure Inc.’s buyout early this year of GPCR Therapeutics Inc. is paying off in a big way with data from the finished phase II trial testing burixafor (GPC-100). The agent is used with propranolol and granulocyte colony-stimulating factor to mobilize hematopoietic progenitor cells in patients with multiple myeloma (MM) undergoing autologous hematopoietic cell transplant.
Impressive results of a potential second-line combination treatment for multiple myeloma from the Majestec-3 trial of teclistamab plus daratumumab raised eyebrows at the American Society of Hematology’s 67th annual meeting, with the combination showing an 83.4% rate of progression-free survival at three years vs. 29.7% for standard of care.
On the heels of mixed phase III data from Agios Pharmaceuticals Inc. with mitapivat to treat sickle cell disease (SCD), Fulcrum Therapeutics Inc. wowed investors by way of initial results from the ongoing 20-mg dose cohort in the phase Ib Pioneer trial testing oral, once-daily fetal hemoglobin inducer pociredir.
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