The FDA has awarded rare pediatric disease designation to Orphagen Pharmaceuticals Inc.'s OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small-molecule antagonist to steroidogenic factor-1 (SF-1; NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
Maia Biotechnology Inc. has advanced its telomere-targeting molecule program (Project T3) with the nomination of a lead new molecular entity candidate (MAIA-2021-20) and a back-up new molecular entity candidate (MAIA-2022-12) for progression into preclinical GLP-toxicity and other studies. One of these candidates may then be advanced into clinical trials upon completion of the preclinical evaluations.
Being able to detect cancers early can substantially improve survival, but most early detection tests for cancer rely on expensive and sophisticated molecular techniques that might be difficult to implement in resource strapped environments. Two new studies published last week attempt to overcome this problem.
Fochon Biosciences Ltd. has disclosed mitogen-activated protein kinase (ERK) inhibitors, particularly ERK1 and/or ERK2 reported to be useful for the treatment of cancer.
Immunos Therapeutics AG has received full ethical institutional approval from the Human Research Ethics Committee (HREC) and regulatory approval from the Australian Therapeutic Goods Administration (TGA) to conduct a phase I trial of its lead program IOS-1002 in Australia.
Major histocompatibility complex class I (MHC-I) gene promoters are bivalently modified during development, which can be exploited in cancer cells to silence MHC-I expression and evade CD8+ T cells; in cells that exhibit bivalent modification of MHC-I genes, lower levels of cell surface MHC-I can be induced either by PRC2 inhibition or exposing the cells to interferon γ (IFNγ) in conjunction with polycomb inhibition to allow a permissive chromatin status.
Pancreatic cancer is an exceptionally lethal cancer that is notoriously treatment resistant, in part due to poor vascularization in the tumor microenvironment. Investigators working at the Moores Cancer Center at the University of California, San Diego (UCSD), reported in the Jan. 16, 2023, issue of Nature Cell Biology on the discovery of a pathway that was initiated by isolation stresses (e.g., hypoxia, nutrient deprivation, and/or lack of extracellular matrix, ECM) leading to this cellular transformation in the tumor-initiating pancreatic cancer cell.
University of Maryland has identified proteolysis targeting chimeras (PROTACs) acting as apoptosis regulator Bcl-2, Bcl-2-like protein 1 (BCL2L1) and induced myeloid leukemia cell differentiation protein Mcl-1 degradation inducers.
Carrick Therapeutics UK Ltd. has synthesized cyclin-dependent kinase 12 (CDK12) and/or cyclin-dependent kinase 7 (CDK7) inhibitors reported to be useful for the treatment of autoimmune diseases, cardiovascular disorders, fragile X syndrome, muscular dystrophy, neurodegeneration, renal disorders and cancer.
Scorpion Therapeutics Inc. has disclosed urea derivatives acting as phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of cancer.
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