Astrazeneca AB has disclosed 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) inhibitors reported to be useful for the treatment of alcoholic liver disease, liver fibrosis, cirrhosis, hepatic steatosis, liver inflammation, hepatitis C virus infection and liver cancer.
Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver diseases.
Researchers from Inipharm Inc. presented preclinical data for INI-822, a small-molecule inhibitor of HSD17B13, being developed for the treatment of liver disease.
Echosens SA obtained clearance from the U.S. FDA to expand the indications for screening with its Fibroscan system. The approval removes contraindications for pregnancy and active implant and includes patients with confirmed or suspected liver disease. It designates Fibroscan as a noninvasive aid for clinical management, diagnosis and monitoring of adult and pediatric patients with liver diseases. The age has been removed as the first selection probe and exam-type step.
Mirum Pharmaceuticals Inc. plans to “move rapidly toward regulatory filings” to expand use of IBAT inhibitor Livmarli (maralixibat) into progressive familial intrahepatic cholestasis (PFIC), President and CEO Chris Peetz told investors during a conference call Oct. 24 to discuss the positive top-line results from the phase III March study, which not only hit its primary endpoint in patients with PFIC2 but showed statistical improvements in pruritis across a range of PFIC subtypes.
Versantis AG has agreed terms with Genfit SA in an acquisition deal that involves CHF40 million (US$41.4 million) up front, plus up to CHF65 million in milestones, and one third of the proceeds from a potential sale of a rare pediatric disease priority review voucher, should the U.S. FDA grant one.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
Syncona Ltd. has added £10 million (US$13.1 million) to the £26.6 million it put into the series A round of Resolution Therapeutics Ltd., enabling the company to advance an allogeneic version of the macrophage cell therapy it is developing for the treatment of compensated liver disease. Since the first close of the round in December 2020, the company has been working on process development for the autologous product, and patients have continued to be recruited to a 50-patient phase II study being run by the academic founders of Resolution.
With the rates of nonalcoholic steatohepatitis (NASH) rapidly rising, Glympse Bio Inc. and Laboratory Corp. of America Holdings Inc. (Labcorp) have announced new tests that can assess the risk of the liver condition without the traditional biopsy.