Gemini Therapeutics said the phase 1 study of GEM103, the company’s investigational treatment for dry AMD, met all of its endpoints. Results demonstrated that in 12 patients receiving a single intravitreal injection of GEM103, there were no dose-limiting toxicities or treatment-related adverse events.

“These results are important, as they demonstrate the promise of GEM103 as a potential treatment for patients with dry AMD who have complement factor H (CFH) loss of function gene variants associated with their disease,” said Arshad M. Khanani, MD, MA, director of clinical research at Sierra Eye Associates and clinical associate professor of ophthalmology, University of Nevada, in a news release. “As a recombinant form of human CFH, GEM103 can precisely target the underlying genetics of these patients. GEM103’s promising safety profile in phase 1 is a critical step forward as therapeutic progresses into phase 2a.”

Results showed that, in the 4 ascending single IVT doses tested (50, 100, 250, and 500 µg of GEM103 in a 50µL dose volume), there were no inflammation, anti-drug antibody, or treatment-related adverse events. All dose levels were well tolerated, with no changes to intraocular pressures in postinjection measurements. In this single-dose, open-label study, visual acuity was generally maintained or improved in the majority of patients with advanced central GA. The tolerability, pharmacokinetics, and impact on biomarkers of complement activity at all dose levels support continued clinical development.

“We are extremely pleased that these results have supported advancing GEM103 into a phase 2a study in a genetically-defined AMD population,” said Jason Meyenburg, CEO of Gemini Therapeutics, in a news release.