More than two years after the U.S. FDA shocked Intercept Pharmaceuticals Inc. with its rejection of obeticholic acid to treat fibrosis due to nonalcoholic steatohepatitis (NASH), the company has announced data it said will form the basis of a NDA refile in the disease for which there is no approved therapy.
Treating sooner in the disease course – “hit them hard, hit them early,” as CEO Tahir Mahmood put it – may change outcomes for the better with AMT-101, the candidate from Applied Molecular Transport Inc. (AMT) tested in a combo trial against ulcerative colitis (UC), officials said during a conference call with investors.
New data confirming that Gilead Sciences Inc.’s Hepcludex (bulevirtide) effectively cures hepatitis delta virus (hepatitis D), one of the most severe viral infections of the liver, and favorable phase III results from Madrigal Pharmaceuticals Inc.’s nonalcoholic steatohepatitis (NASH) contender were among the highest-profile scientific announcements at the International Liver Congress (ILC) 2022.
In this case, more is just too much. The number of participants needed to determine a significant clinical outcome in 9 Meters Biopharma Inc.’s phase III of larazotide in treating celiac disease has grown too large to support the study. The company said an independent statistician’s interim analysis convinced it to take a step back and re-evaluate its options for developing the zonulin inhibitor in the indication.
Poor phase II top-line data for Immunic Inc.’s lead candidate, vidofludimus calcium (IMU-838) hammered the stock June 2 as the study of the selective oral DHODH inhibitor missed its primary endpoint of clinical remission in treating moderate to severe ulcerative colitis. The failure means Immunic won’t pursue a phase III study on its own in the indication, but it won’t affect the company’s planned phase III studies of IMU-838 in treating relapsing multiple sclerosis (MS) and a phase II in treating progressive MS.
New and updated preclinical and clinical data presented by biopharma firms at Digestive Disease Week 2022, including: Ardelyx, Ferring, Ironwood, Janssen, Phathom, Rebiotix, Redhill, Second Genome.
Unveiling further positive data for SER-109 in preventing recurrent Clostridium difficile infection at Digestive Disease Week (DDW) over the weekend, Seres Therapeutics Inc. reiterated plans to file a BLA with the U.S. FDA in mid-2022. Should the FDA accept the application and grant priority review – SER-109 has breakthrough and orphan status – the medicine could be the first microbiome-based therapy to reach the market.
New and updated preclinical and clinical data presented by biopharma firms at Digestive Disease Week 2022, including: Albireo, Cymabay, Evoke, Fractyl, Seres.
Intent on expanding both the reach and approved uses of its farnesoid X receptor agonist, Ocaliva (obeticholic acid), Intercept Pharmaceuticals Inc. is selling both its ex-U.S. operations and rights to the primary biliary cholangitis drug to U.K.-based Advanz Pharma Corp. Ltd. for $405 million up front. The deal, including an additional $45 million earnout tied to EU regulatory advances, represents an important step toward strengthening Intercept's balance sheet and options, said CEO and President Jerry Durso.
Connect Biopharma Holdings Ltd. pointed to positive secondary endpoint data and a numerical trend on the primary endpoint in favor of its S1P receptor modulator, CBP-307, in ulcerative colitis, but those results weren’t enough to keep the stock (NASDAQ:CNTB) from falling by more than 57% to close at 82 cents, as investors focused on the primary endpoint miss and the company’s decision to partner the program going forward.