After decades of trying and dozens of failed trials, amyloid targeting has paid off with the first disease-modifying agents reaching the market. But success does not mean slam dunk. Aduhelm (aducanumab, Biogen Inc.) was dogged by controversy throughout its brief tenure, and Biogen pulled the plug on it in early 2024. Leqembi (lecanemab, Biogen Inc.) has received full approval. In this second installment of a three-part series on Alzheimer’s, BioWorld looks at the nuanced view of amyloid’s role in the disease. Read More
Natural killer (NK) cells play a crucial role in cancer immunosurveillance. Circulating NK cells can kill target cells without prior sensitization. There are some receptors known to impact the activity of NK cells, such as the inhibitory NK cell receptor TIM3, among others, which recognizes phosphatidylserine on the surface of cells. More research has been performed using targeted or genome-wide CRISPR screening to identify cancer cell genes that impact NK cell-killing ability. Read More
Cytomx Therapeutics Inc. has announced the nomination of the second clinical candidate under its Probody T-cell engaging bispecific (TCB) collaboration with Astellas Pharma Inc. The two companies are collaborating on multiple conditionally activated TCB programs, with T-cell engagers offering new possibilities for the treatment of solid tumors. Read More
Alterome Therapeutics Inc. has closed a $132 million series B financing to support the advancement of its pipeline of next-generation, small-molecule targeted cancer therapies into the clinic, including a highly specific AKT1 E17K inhibitor and a KRAS selective inhibitor. Read More
Hypoxia-inducible factor 2α (HIF-2α) is a transcription factor that plays a key role in oxygen homeostasis and response to tumor hypoxic microenvironment of cancer cells. Previous research has suggested that inhibition of HIF-2α is a promising antitumor approach, particularly against tumors associated with mutant von Hippel-Lindau (pVHL). Read More
Discovery of novel Nav1.8 inhibitors capable of achieving high levels of target modulation at low oral doses for the potential treatment of pain was reported by Merck & Co. Inc. Read More
Researchers from China Pharmaceutical University published data detailing the discovery and preclinical evaluation of novel tubulin polymerization inhibitors as potential anticancer agents. Read More
Verismo Therapeutics Inc. has submitted an IND application to the FDA seeking to initiate a phase I trial this year of Synkir-310 for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas (B-cell NHL), including diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma and marginal zone lymphoma. Read More
Neuron23 Inc. has synthesized leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of cancer, neurodegeneration and Parkinson’s disease. Read More
Ono Pharmaceutical Co Ltd. has identified diacylglycerol kinase α (DGK-α, DGKA) and/or diacylglycerol kinase ζ (DGK-ζ, DGKZ) inhibitors reported to be useful for the treatment of cancer. Read More
Conformation-X Therapeutics LLC has closed an oversubscribed funding tranche of $3.65 million to support the company’s development of a pipeline of immunotherapies that activate both the innate and adaptive arms of the immune system. Read More
Bridge Medicines LLC has divulged protein ENL (MLLT1; YEATS1) and/or FLT3 (FLK2/STK1) inhibitors potentially useful for the treatment of acute lymphocytic leukemia and acute myeloid leukemia. Read More
It has been previously demonstrated that while cancer cells with defects in ataxia telangiectasia mutated kinase (ATM) signaling are susceptible to ataxia telangiectasia and Rad3-related (ATR) inhibition following treatment with DNA-damaging drugs, normal cells can tolerate ATR inhibition by activating an ATM-mediated compensatory DNA damage response. Read More
Chiesi Farmaceutici SpA presented data for novel lung-targeted muscarinic M3 receptor antagonists/β2-adrenoceptor agonists (MABAs) as candidates for the treatment of chronic obstructive pulmonary disease (COPD). Read More
Vertex Pharmaceuticals Inc. has described α1-antitrypsin (SERPINA1) (Z-mutant) polymerization inhibitors reported to be useful for the treatment of α1-antitrypsin (AAT) deficiency. Read More
