Gemini Therapeutics announced the completion of enrollment in its phase 2a “ReGAtta” study. ReGAtta is a dose escalation trial of GEM103, a recombinant human complement factor H (CFH), in dry AMD patients with CFH loss-of-function gene variants. The trial is designed to evaluate safety and tolerability as well as measures of intraocular pharmacokinetics (PK) and disease-relevant biomarkers to inform the late-stage development program.
“We are pleased to have completed enrollment in our phase 2a ReGAtta trial of GEM103. Our strategy is to provide an intravitreal recombinant human factor H in patients with gene variants resulting in loss of function of their own CFH. There is no approved therapy for the treatment of geographic atrophy (GA) and we know that approximately 40% of dry AMD patients have variants in the CFH gene linked to progression from early AMD to late stage GA,” Marc E. Uknis, MD, FACS, Chief Medical Officer of Gemini Therapeutics, said in a company news release. “We believe that GEM103 offers hope for patients with dry AMD as it precisely targets the underlying genetics of the disease. We look forward to sharing topline data from the trial in the first half of 2021.”
“This is an important achievement for Gemini in our efforts to approach complement-mediated diseases in a novel manner, by restoring regulation of complement system. We want to thank the patients, the ophthalmology community of investigators and the Gemini team for their commitment to this important mission,” said Jason Meyenburg, Chief Executive Officer of Gemini Therapeutics. “We believe that our work in biology and genetics, coupled with the novel mechanism of action of GEM103, can enable us to realize the potential of precision medicine in the treatment of AMD.”
About the Phase 2a ReGAtta Study
The ongoing Phase 2a, multicenter, open-label, multiple ascending dose study of GEM103 in genetically-defined patients with GA secondary to dry AMD is designed to investigate safety and tolerability, PK, exploratory ocular biomarkers, and measures of retinal anatomy and function. GEM103 is delivered monthly by an intravitreal injection and PK and biomarkers of complement regulation are determined from aqueous humor sampling. The study was designed to enroll approximately 60 patients with gene variants that have been linked to the progression of dry AMD from early to late-stage.