Gemini Therapeutics Initiates GEM103 Phase 2a Study as an Add-On to Anti-VEGF Therapy for the Treatment of Wet AMD Patients at Risk for Progressive Vision Loss Due to Macular Atrophy
Gemini Therapeutics announced the commencement of a phase 2a trial advancing GEM103 as a potential add-on therapy for patients suffering from wet AMD who have, or may be at risk for, macular atrophy (MA) but require ongoing anti-vascular endothelial growth factor (anti-VEGF) treatment. Topline data related to safety, tolerability, effect on intraocular CFH levels and biomarkers is expected in the second half of 2021.
“Current anti-VEGF therapies have improved the quality of life for patients with wet AMD,” Marc E. Uknis, MD, FACS, Chief Medical Officer of Gemini Therapeutics, said in a company news release. “However, there is no therapy for the MA that can develop in some patients. In these patients, ongoing anti-VEGF therapy has been associated with a compartmental insufficiency of CFH activity. In this setting, GEM103, a recombinant human CFH, has the potential to be a meaningful add-on therapy.”
“The addition of a clinical research program targeting a selected wet AMD population is a natural next step for Gemini, and builds on our existing focus in genetically-defined dry AMD,” Jason Meyenburg, Chief Executive Officer of Gemini Therapeutics, said in the news release. “Wet AMD affects approximately 1.4 million people in the United States, and GEM103 is uniquely suited to correct CFH insufficiency associated with VEGF inhibition. This study is an important next phase in our understanding of the potential role of GEM103 as an add-on therapy in a subset of patients with wet AMD who go on to have co-morbid macular atrophy.”
The clinical trial will be a randomized, single-masked, and sham-controlled study of patients requiring ongoing VEGF inhibition for wet AMD. The objective of the study is to assess safety, tolerability, effect on CFH levels and disease-relevant biomarkers in the ocular compartment to inform future development strategies.
About GEM103
Gemini’s lead program, GEM103, is a pioneering precision medicine approach, targeting trial enrichment with genetically-defined patients. GEM103 targets a genetically-defined subset of age-related macular degeneration (AMD) patients with complement dysregulation. Of the 15 million dry AMD patients, approximately 40% (or six million) have variants in the complement factor H (CFH) gene. Such loss-of-function variants are associated with increased dry AMD disease risk. GEM103 is believed to be the first ever recombinant native complement modulator, full-length recombinant complement factor H (rCFH) protein. When delivered by intravitreal injection, GEM103 has the potential to address unmet medical need in genetically-defined subsets of AMD patients by circumventing dysfunctional CFH loss-of-function variants and slowing the progression of their retinal disease.