With a successful phase III study of plozasiran in hand, Arrowhead Pharmaceuticals Inc. plans to file an NDA with the U.S. FDA for treating the rare genetic disease familial chylomicronemia syndrome. While the data are strong, the company is playing catch-up to Ionis Pharmaceuticals Inc., which has a December 2024 PDUFA for its candidate, olezarsen, in the same indication.
Positive top-line results from Calcimedica Inc.’s placebo-controlled phase IIb of Auxora in acute pancreatitis couldn’t support the stock for the day. The selective small-molecule inhibitor of Orai1-containing calcium release-activated calcium (CRAC) channels hit the randomized, double-blind, dose-ranging Carpo study’s primary endpoint, the median time it took to tolerate solid food, as patients who received Auxora had a statistically significant dose response compared to those who received placebo.
Pancreatic ductal adenocarcinoma (PDAC) has a poor prognosis, in part due to the immunosuppression surrounding the tumor mediated by regulatory T cells (Tregs), thus preventing effective responses to immune therapy.
The pathogenesis of chronic pancreatitis (CP) is complex and involves interactions among different cell types, including pancreatic acinar, ductal and stellate cells (PSC). Researchers from Calcimedica Inc. and the University of Szeged set out to assess the pathogenic pathways in these cells as well as evaluate the potential of the novel calcium release-activated calcium channel protein 1 (Orai1) inhibitor CM-5480 as a potential treatment for CP.
An investor has seen promise in First Wave Biopharma Inc.’s targeted, systemic gastrointestinal disease biotherapeutics, offering $4 million in a private placement as enrollment picks up pace in a phase II trial for the company’s lead candidate, adrulipase.
After an eight-year academic collaboration with Glaxosmithkline plc, Kynos Therapeutics Ltd. has spun out of Edinburgh University with a £9 million (US$11.7 million) financing and a phase I-ready program. The company specializes in kynurenine 3-monooxygenase (KMO), a mitochondrial outer membrane protein that is a critical gatekeeper in the kynurenine pathway of tryptophan metabolism, and which regulates inflammation and immunity.
Investigators at MD Anderson Cancer Center have published data suggesting that activating KRAS mutations may be selected for in pancreatitis, because they protect pancreatic tissue from damage.
TORONTO – An initial investment of $250,000 by MCI Onehealth Technologies Inc. could just be the beginning for Ariel Precision Medicine Inc. and an artificial intelligence-fueled platform that uses genetic and clinical data to identify early symptoms of chronic disease.
