Even though the treatment options that exist for acute myeloid leukemia (AML) are growing, the clinical outcome of patients is still unfavorable. In AML dysregulation of the tyrosine kinase receptors, including RAS, RAF, MEK and ERK, and of the Aurora kinase family (AURK) exists, are both tied to AML development and progression.
Molecure SA has confirmed the in vitro activity of a molecule binding to a new mRNA target within its mRNA discovery platform. This represents the discovery of another class of molecules binding to a second mRNA biological target, which has been confirmed in in vitro assays.
The U.S. FTC’s campaign against the Orange Book listing of patents claiming device components gained momentum when a federal judge in New Jersey ordered Teva Pharmaceuticals USA Inc. to delist five device patents pertaining to its Proair HFA (albuterol sulfate) inhaler.
After delivering animal data to show that inhibiting the NLRP3 inflammasome induces weight loss in mice, Nodthera Ltd. has now demonstrated this effect translates to humans. In a phase Ib/II study of NT-0796, an orally available, brain-penetrant drug, subjects in both the active and placebo arms all lost weight, due to the calorie-restricted diet they received in the 28-day trial.
Ipsen SA picked up U.S. FDA accelerated approval for its Genfit SA-licensed elafibranor, making it the first new drug in eight years for treating primary biliary cholangitis, though a potential competitor lurks just around the corner.
After the phase IIa failure at lowering intraocular pressure to a statistically significant degree with SBI-100, Skye Bioscience Inc. is dropping work with the ophthalmic emulsion, meant to treat primary open-angle glaucoma or ocular hypertension. Resources are turning to the firm’s metabolic program, which includes nimacimab, targeting the cannabinoid 1 receptor, due to start a phase II trial in obesity during the third quarter of this year.
Washington-based Vanda Pharmaceuticals Inc. has faced a challenging few years involving a federal lawsuit against the U.S. FDA, patent infringement litigation, increasing generic competition and dwindling sales, as well as a complete response letter nixing plans to expand its melatonin receptor agonist Hetlioz (tasimelteon) into insomnia, yet it has recently received a higher, unsolicited acquisition offer of $466 million from a second company, Cycle Pharmaceuticals Ltd.
The discussion that preceded the June 4 U.S. FDA advisory committee vote against the approval of Lykos Therapeutics Inc.’s midomafetamine as a treatment for post-traumatic stress disorder could shine some light on the way forward for other sponsors developing psychedelics for approved medical use.
Bridgebio Pharma Inc.’s data from the phase II study with infigratinib in achondroplasia “swings the pendulum of debate sharply in favor” of the firm, competing with Biomarin Pharmaceutical Inc. in the space, Leerink analyst Mani Foroohar said in a report.
Although several members of the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee said they thought Lykos Therapeutics Inc.’s midomafetamine (MDMA), used in combination with psychotherapy, is a promising treatment for post-traumatic stress syndrome, they were not ready to endorse its approval.
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