Top-line results from Enanta Pharmaceuticals Inc.'s Argon-1 phase IIa study of EDP-305 for the treatment of nonalcoholic steatohepatitis (NASH) found one of the two doses of the drug tested met the study's primary endpoint, alanine aminotransferase (ALT) reduction at week 12, contributing further evidence for the drug's mechanism of action, farnesoid X receptor (FXR) agonism. But the non-biopsy study appears to have left investors without proof for something more even more valuable: a clear case for differentiation vs. FXR first-mover Ocaliva (obeticholic acid, Intercept Pharmaceuticals Inc.). Appearing to reflect the sentiment, Enanta's shares (NASDAQ:ENTA) fell 15.1% to $60.51 on Thursday.
Corvia Medical Inc. is enrolling patients in a 100-site multinational study of its Interatrial Shunt Device that will measure traditional heart failure endpoints as well as biosensor data transmitted to the cloud for analysis by Napier, Ill.-based Physiq's artificial intelligence analytics. The trial will evaluate Tewksbury, Mass.-based Corvia's device in heart failure with preserved and mid-range ejection fraction.
BOSTON – The FDA's Center for Device and Radiological Health (CDRH) released draft guidance on Monday aimed at encouraging the use of patient input in medical device clinical trials and helping sponsors understand how they can use patient engagement to improve the design and conduct of investigations.
Shares of San Diego-based Tocagen Inc. (NASDAQ:TOCA) fell 77.7% to 93 cents on Sept. 12, and further since, after its two-part immunotherapy for people with recurrent brain cancer failed to surpass standard of care on overall survival (OS), the primary endpoint of the company's phase III Toca 5 trial. Secondary endpoints in the registrational study were also missed, showing no meaningful difference between study arms.
HONG KONG – In one of the latest combination efforts against cancer, Kahr Medical Ltd. is teaming up with Switzerland's Basel-based Roche Holding AG to study the use of the former's lead program, DSP-10, in combination with the latter's PD-L1-blocking checkpoint inhibitor, Tecentriq (atezolizumab), in patients with advanced non-small-cell lung carcinoma refractory to checkpoint inhibitors.
Ritter Pharmaceuticals Inc. is reeling from the phase III failure of its lead candidate, RP-G28, designed for patients with lactose intolerance. RP-G28 failed to demonstrate statistical significance in its primary endpoint as the top-line data showed it had no or little difference on patients compared to placebo. The data also show RP-G28 missed its secondary endpoints, all of which casts a shadow on the company and its pipeline.
Glaxosmithkline plc has won FDA approval to market Nucala (mepolizumab) for use in children as young as 6 with severe eosinophilic asthma (EA). The therapy already had FDA approval as an add-on maintenance treatment for kids with the same condition ages 12 and older. Approval of the sBLA, submitted last November, catches the U.S. market up to the EU, where Nucala has been approved as an add-on treatment for children ages 6 to 17 since August 2018.