As NGM Biopharmaceuticals’ phase 2 trial misses the mark, dreams for its Merck & Co.-partnered macular degeneration treatment may be disappearing from sight.
The Californian biotech’s stock dropped 70% in morning trading, from $11.55 per share at close on Friday to $3.09 at 10:45 a.m. ET today.
The phase 2 study, dubbed Catalina, aimed to measure NGM’s sole eye disease asset, a humanized immunoglobulin G1 monoclonal antibody (mAb) called NGM621. The trial failed to reach its main goal of producing statistically significant reduction rates in lesion areas over 52 weeks among patients with secondary to age-related macular degeneration (AMD).
The mAb was administered to 212 patients over 52 weeks via intravitreal injection, with one group of patients receiving the shot every four weeks and another every eight weeks. The investigational treatment demonstrated a 6.5% reduction rate of change in lesion area for the four-week cohort and a 6.3% reduction for the eight-week group—neither of which was statistically significant. Another 106 patients received sham treatment, with no significant results reported.
The company is still examining pre-specified secondary endpoints and post-hoc analyses, NGM CEO David Woodhouse, Ph.D., said in an Oct. 17 release.
NGM621 did show a favorable safety and tolerability profile, with no evidence of increased treatment-related severe adverse events or choroidal neovascularization (CNV), another late form of AMD.
“While the Catalina trial did not meet its primary endpoint, the notable CNV finding combined with signals from secondary and exploratory analyses suggest the possibility that NGM621 may have a role in treating GA,” Charles Wykoff, M.D., Ph.D., director of research at Retina Consultants Texas and Catalina investigator, said in the release. “I look forward to evaluating further details from this trial and presenting the findings at the upcoming Retina Society Annual Meeting in November.”
The results will be unwelcome news for Merck, which has an option to license NGM621 and its related compounds. The big pharma still has three months to make a decision, before the rights return to NGM.
It isn’t the biotech’s first flunk—in 2021, its phase 2b trial Alpine 2/3 missed its primary endpoint assessing aldafermin in nonalcoholic steatohepatitis (NASH) patients. Given the results, NGM pulled the plug on phase 3 plans to evaluate the FGF19 analog among F2/F3 NASH patients.
The biotech hasn’t given up on the NASH asset though, as it is currently conducting Alpine 4, a phase 2b 48-week study for patients with biopsy-confirmed NASH cirrhosis. The company’s pipeline currently consists of seven programs across cancer, retinal diseases, and liver and metabolic diseases.