The stock rally by Tango Therapeutics Inc. over the past month or so has further revved the long-percolating interest in protein arginine methyl transferase 5 (PRMT5) and in synthetic lethality, where a number of parties have programs ongoing.
An investigational dendritic cell vaccine from Northwest Biotherapeutics Inc., administered with standard of care, extended both median overall survival (OS) and "the ‘long tail' of the survival curve" vs. matched external controls in newly diagnosed and recurrent glioblastoma multiforme brain cancer patients, according to results published Nov. 17 in JAMA Oncology.
As Wall Street waits to find out later this year whether Stoke Therapeutics Inc.’s positive results with a low, single dose of STK-001 for Dravet syndrome pans out in more extensive research, a number of players large and small are investigating candidates for the rare but dismal form of epilepsy.
Stimulating the brain via implanted electrodes is used to treat both movement disorders such as Parkinson’s disease, and some psychiatric conditions such as obsessive compulsive disorder. But researchers are also working on ways to make such implanted electrodes listen instead of talk – and translate neuronal signals for people that have lost the ability speak, or the ability to move.
A lackluster efficacy signal has prompted Editas Medicine Inc. to pause enrollment in a phase I/II trial of its CRISPR/Cas9-based gene editing therapy, EDIT-101, which is in development for patients with a particular form of Leber congenital amaurosis type 10.
Astellas Pharma Inc.’s zolbetuximab, a monoclonal antibody targeting Claudin 18.2, met the primary endpoint of progression-free survival in the phase III Spotlight trial in CLDN18.2-positive, HER2-negative, locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma, according to top-line data.
Reistone Biopharma Co. Ltd. said both doses of the JAK1 inhibitor ivarmacitinib, tested against moderate to severe atopic dermatitis (AD) in a multinational phase III trial, met the study’s co-primary endpoints, delivering significant improvements on a common measure of disease severity vs. placebo. It’s the first National Class A new drug for AD that is designed and developed in China, according to the company.
Immutep Ltd.’s lead immunotherapy candidate eftilagimod (IMP-321, efti) met the primary endpoint in its phase II trial evaluating the combination of efti and Merck & Co. Inc.’s Keytruda (pembrolizumab) as first-line treatment of non-small-cell lung cancer (NSCLC), reporting final data that show an overall response rate of 40.4%.
Que Oncology Inc.’s Q-122 significantly reduced the frequency and severity of moderate and severe vasomotor symptoms, or hot flashes, in women taking endocrine therapy for breast cancer, and phase II results showed associated improvement in quality of life, compared with placebo.
GSK plc said it will restrict the second-line maintenance indication for ovarian cancer drug Zejula (niraparib) to only patients with deleterious or suspected germline BRCA mutations, at the request of the FDA, in a sign that U.S. regulators aren’t going to relax scrutiny on PARP inhibitors any time soon.
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