Following a late-cycle review meeting with the U.S. FDA in September and the agency’s decision to skip the advisory committee meeting, expectations were high heading toward the PDUFA date for Applied Therapeutics Inc.’s priority NDA for govorestat in galactosemia. So the complete response letter issued by the FDA just ahead of the Nov. 28 PDUFA date, citing deficiencies in the clinical application, caught nearly everyone off guard.
Potent siRNAs against B4GALT1 were designed in silico and screened in vitro (Huh7 cells, primary mouse, human hepatocytes) as well as in vivo (C57BL/6 mice) for the selection of a lead Galomic siRNA.
Researchers at the University of Copenhagen have identified a signaling pathway that simultaneously increased energy expenditure and decreased food intake. In both human and primate studies, agonists of the tachykinin NK2 receptor (NK2R) led to both decreased food intake and increased energy expenditure. And in behavioral tests, they were not aversive, suggesting they do not cause the nausea that is a major side effect of GLP-1 agonists.
The conditional marketing approval for Ocaliva (obeticholic acid) has been revoked with immediate effect, following a standoff between the EMA and Advanz Pharma Ltd., the company that markets the primary biliary cholangitis (PBC) therapy in Europe. On Sept. 5, London-based Advanz won a short reprieve after challenging the EMA’s June 28 ruling that the marketing authorization for Ocaliva should be revoked, when the General Court of the EU granted a temporary suspension of EMA’s decision. However, on Nov. 27, Advanz announced the court had said it would not be extending the suspension.
Wall Street began comparing and contrasting what’s available after Amgen Inc. rolled out phase II data with weight loss candidate Maritide – a disclosure that led shares of the biotech heavyweight (NASDAQ:AMGN) to close Nov. 26 at $280.01, down $13.99.
With the U.S. FDA approval of Attruby (acoramidis) for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Bridgebio Pharma Inc. is taking on an industry giant. The next-generation, oral, small-molecule stabilizer of transthyretin will take on Pfizer Inc.’s Vyndamax (tafamidis, Vyndaqel), a TTR stabilizer approved in 2019 for ATTR-CM but expected to lose exclusivity in late 2028. The approval was based on a phase III study that showed Attruby significantly reduced death and cardiovascular-related hospitalizations. The NDA, which had a Nov. 29 PDUFA date, was approved Nov. 22.
Urocortin-2 (UCN2) is a selective corticotropin-releasing factor CRF2 receptor (CRFR2) agonist, which has been previously shown to reduce fat mass while promoting muscle hypertrophy. Hanmi Pharmaceutical Co. Ltd. presented the discovery and preclinical characterization of a novel CRFR2-selective UCN2 analogue, HM-17321, being developed for the treatment of obesity.
Startup company Pep2tango Therapeutics Inc., unveiled with backing by Versant Ventures, is advancing unimolecular multireceptor peptide agonists that target GLP-1, GIP, amylin and calcitonin receptors.
Fat cells from patients who had lost weight after bariatric surgery, as well as from animals who had gained and then lost weight, were transcriptionally distinct from cells that had not experienced such weight changes at the organism level. In the animal studies, those transcriptional changes were due to epigenetic changes. The findings, which were published online in Nature on Nov. 18, 2024, are a possible molecular-level explanation for the short-term nature of most weight loss.
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