In a deal that could bring more than $2.1 billion in payments to Arbor Biotechnologies Inc., 90-year-old Chiesi Group gained exclusive and global rights to develop and commercialize ABO-101 for primary hyperoxaluria type 1, an ultra-rare disease caused by a mutation in the AGXT gene, as well as an option to go after a limited number of additional targets.
Activation of cannabinoid receptors in the peripheral nervous system may help treat inflammatory and gastrointestinal disorders as well as pain, and several full agonists have been reported but they present safety concerns. Partial agonists, such as those that activate receptors to 20%-50% of full activity, may be safer, yet so far such agonists have emerged serendipitously.
Takeda Pharmaceutical Co. Ltd. is exiting all work in cell therapies and will seek an external partner to advance the company’s research and clinic-ready cell therapy programs, the company said, noting that it is not currently running any clinical trials using cell therapy technology.
Daewon Pharm Co. Ltd. has disclosed tricyclic derivatives acting as reversible H+/K+-ATPase inhibitors reported to be useful for the treatment of gastroesophageal reflux disease.
Researchers from Opko Health Inc. and Entera Bio Ltd. recently presented preclinical pharmacokinetic data on OPK-8801003, an oral GLP-2 analogue developed for the treatment of short bowel syndrome.
C4X Discovery Holdings Ltd. has selected a preclinical candidate from its oral α4β7 integrin inhibitor program for the treatment of inflammatory bowel disease (IBD). The potent and selective α4β7 inhibitor supports once-daily dosing. Moreover, the company’s approach can be combined with other targeted therapies for patients with complex or refractory disease where existing approaches have failed to deliver sufficient control.
Haisco Pharmaceutical Group Co. Ltd. has identified pyridone derivatives acting as integrin α4β7 (LPAM-1) antagonists reported to be useful for the treatment of Crohn’s disease and ulcerative colitis.
When it won U.S. FDA accelerated approval more than eight years ago, Intercept Pharmaceuticals Inc.’s Ocaliva (obeticholic acid) was viewed as a breakthrough, becoming the first new treatment in 20 years for rare, progressive liver disease primary biliary cholangitis (PBC) and, for several years, the only second-line treatment for PBC patients failing to respond to ursodeoxycholic acid. More recently, however, Ocaliva has faced regulatory and safety stumbles, with Intercept now voluntarily pulling the farnesoid X receptor activator from the U.S. market.
Lactiga US Inc. has received an award from the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to advance the development of its secretory IgA (sIgA) antibody for inflammatory bowel disease (IBD).
Metabolic dysfunction-associated steatotic liver disease (MASLD) now affects over a third of adults worldwide. Its progressive form, metabolic dysfunction-associated steatohepatitis (MASH), is a leading driver of cirrhosis, cancer and liver transplants while often coexisting with diabetes, obesity and cardiovascular disease.
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