Gemini Therapeutics Announces GEM103 Meets All Endpoints in Phase 1 Clinical Study
Gemini Therapeutics announced that the phase 1 study of GEM103, the company’s investigational treatment for dry AMD, met all its endpoints. Results demonstrate that in 12 patients receiving a single intravitreal (IVT) injection of GEM103, there were no dose-limiting toxicities or treatment-related adverse events. Pharmacokinetic and exploratory biomarkers as measured in the aqueous humor (AH) from patients enrolled in the phase 1 single ascending dose (SAD) study is being presented during a virtual poster session at the 2020 Annual Meeting of the American Academy of Ophthalmology (AAO).
“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,” Arshad M. Khanani, MD, MA, Director of Clinical Research at Sierra Eye Associates and Clinical Associate Professor of Ophthalmology, University of Nevada, said in a company 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 four 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 post injection measurements. In this single dose, open-label study, visual acuity was generally maintained or improved in the majority of patients with advanced central GA. Procedures (IVT administration and sampling of the aqueous humor) were also well-tolerated. In addition, the study demonstrated that dose-dependent supraphysiological levels of CFH were achieved, and AH biomarker changes following GEM103 administration support the mechanism of action of GEM103. The tolerability, PK 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. “Gemini continues to incorporate its insights in genetics and biology to pioneer precision medicines to restore regulation of the complement system in the eye and throughout the body.”
Gemini is currently evaluating GEM103 in the phase 2a ReGAtta clinical trial, a multicenter, open-label, multiple dose escalation study in patients with GA secondary to dry AMD. The study is being conducted in a genetically-enriched population including individuals who participated in the phase 1 study, as well as a treatment-naïve patients. The study will evaluate the impact of monthly GEM103 dosing on safety and tolerability, pharmacokinets and exploratory biomarkers, and measures of visual function relevant in the population.