Meta Pharmaceuticals Inc. announced that the FDA has granted rare pediatric disease designation to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH), an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body.
In the wake of Sage Therapeutics Inc.’s recent phase II fizzle with SAGE-324 in essential tremor, Wall Street is waiting for better news in the indication, which has racked up failures across several developers. Among those finding favor lately is Praxis Precision Medicines Inc. with Nav-targeting ulixacaltamide, also known as PRAX-628, which Oppenheimer analyst Francois Brisebois said “warrants particular attention.”
Researchers from Wayshine Biopharm Holding Ltd. published new data regarding the preclinical systemic pharmacokinetics (PK), dose proportionality, and central nervous system (CNS) distribution of the novel ataxia telangiectasia mutated (ATM) inhibitor WSD-0628, being developed for the treatment of brain tumors.
Researchers from Changzhi Medical College and affiliated organizations presented the discovery of DYC-1, a novel dual-target inhibitor of dual-specificity tyrosine phosphorylation-regulated kinase 2 (DYRK2) and histone deacetylase 8 (HDAC8), being developed for the treatment of hepatocellular carcinoma (HCC).
Investigators at Washington University in St. Louis and Umea University have reported that the small molecule PS-757 was effective in culture and animal models against Streptococcus pyogenes, a gram-positive pathogen responsible for more than 500,000 deaths per year globally.
Scientists at the Novartis Institutes for Biomedical Research (NIBR) in Cambridge have discovered a small molecule that could be used as a therapy for sickle cell disease (SCD). The molecular glue oral degraders of the WIZ transcription factor called dWIZ-1 and dWIZ-2, bind to cereblon (CRBN) and WIZ, marking it for degradation and inducing the expression of fetal hemoglobin (HbF).
Tokyo-headquartered Otsuka Pharmaceutical Co. Ltd. said Aug. 1 that it will acquire Boston-based Jnana Therapeutics Inc. through a potential $1.125 billion M&A deal. Under the terms, Otsuka will work to close the transaction by the third quarter of 2024, with $800 million paid out to Jnana shareholders, along with up to $325 million in additional development and regulatory milestones payments.
As the Sept. 21 PDUFA date looms for arimoclomol from Zevra Therapeutics Inc. in Niemann-Pick type C (NPC), the U.S. FDA’s newly formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) decided in favor of the drug.
Researchers from the University of Minnesota have presented the discovery and preclinical characterization of a novel potent inhibitor of multidrug resistance-associated protein 1 (MRP1), ZW-1226, that is being developed as a therapeutic candidate for the treatment of multidrug resistant (MDR) cancers.
Researchers from East China Normal University and Fudan University presented the discovery of a new oral compound, Z-526, being developed as a treatment to alleviate chemotherapy-induced cachectic muscle loss.
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