PhIII dead end for celiac drug crushes penny stock biotech

2022-06-21

A compound that’s been touted as the first-ever drug to enter a Phase III trial for celiac disease appears to have run into a dead end. At an interim statistical analysis designed to determine how many more patients it would take to detect a statistically significant effect of larazotide, 9 Meters Biopharma said it may have to shutter the study because “the additional number of patients needed to determine a significant clinical outcome between placebo and larazotide is too large to support trial continuation.” The company will keep looking over the data for any silver linings — subgroup effects, particular symptom alleviation — and talk to the FDA before finalizing plans about the drug. But execs are already preparing investors for the worst case scenario. “Pending the final analysis, financial and human resources previously dedicated to this study will be re-deployed to advance our development program for vurolenatide and our early-stage product candidates,” president and CEO John Temperato said in a statement, referring to 9 Meters’ other lead program, a GLP-1 receptor agonist GLP-1 receptor agonist. Shares of the Raleigh, NC-based company $NMTR , which slid into penny stock status earlier this year, dropped another 56% to $0.23. Focusing on patients with celiac disease who continue to experience gastrointestinal symptoms, the Phase III trial had a 24-week design, including 5 weeks for screening and 12 weeks for double-blind treatment. It would enroll 525 patients, randomized into three dosing arms: larazotide 0.25 mg, larazotide 0.5 mg and placebo. Once the first half of the trial population completed their 12-week efficacy portion, though, 9 Meters hired an independent statistician to re-estimate, based on the data from those treated patients, the actual “treatment group size required to detect a statistically significant clinical effect of larazotide.” The primary endpoint was mean change for celiac symptom severity, as measured by the CeD PRO score. The trial began back in 2019, before Innovate Biopharmaceuticals, the original developer of larazotide, merged with OrbiMed-backed private startup RDD Pharma to become 9 Meters. More recently, the compound’s inventors proposed using it to treat severe post-Covid disease in children. Thanks in part to the RDD deal, 9 Meters said it has other assets in development for rare gastrointestinal disorders, led by vurolenatide, which is in Phase II for short bowel syndrome.

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