CAMBRIDGE, Mass.--(BUSINESS WIRE)--Gemini Therapeutics, Inc. (Nasdaq: GMTX), a clinical stage precision medicine company developing innovative treatments for genetically defined age-related macular degeneration (AMD) and linked ocular disorders, today 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,” said Marc E. Uknis, M.D., F.A.C.S., Chief Medical Officer of Gemini Therapeutics. “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 opthalmology 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, multi-center, 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.
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. The U.S. Food and Drug Administration (FDA) granted Fast Track Designation for GEM103 for the treatment of dry AMD in patients with CFH loss of function gene variants.
About Dry Age-Related Macular Degeneration (AMD)
Age-related macular degeneration (AMD) is a progressive retinal disease affecting millions of older adults, and the leading cause of irreversible blindness in the western world. Symptoms, which include blurry vision, loss of night vision and loss of central vision, make activities of daily living such as reading, driving and even recognizing faces progressively more difficult. Third-party reports indicate there are approximately 16 million patients with AMD in the United States alone. Dry AMD, which results from an interaction of environmental and genetic risk factors, represents about 90% of that population (or about 15 million) in the US compared to about 1.4 million with wet AMD. Genetic risk of developing dry AMD is significant, with approximately 70% attributable risk of advanced disease to heritability, while aging and smoking confer the strongest non-genetic risk. CFH risk variants occur in approximately 40% of patients with dry AMD and these patients have a significantly increased risk of developing the disease as well as progression from intermediate AMD to GA. The complement system, of which CFH is a modulator, is dysregulated in patients with these risk variants, and results in amplification of aberrant inflammatory responses in the eye. Over time, this dysregulation leads to damage to the macular region of the retina.
About Gemini Therapeutics
Gemini Therapeutics is a clinical stage precision medicine company developing novel therapeutic compounds to treat genetically defined age-related macular degeneration (AMD). Gemini’s lead candidate, GEM103, is a recombinant form of the human complement factor H protein (CFH), and is designed to address both complement hyperactivity and restore retinal health in patients with AMD. GEM103 is currently in a Phase 2a trial in dry AMD patients with a CFH risk variant. The company has generated a rich pipeline including recombinant proteins, gene therapies, and monoclonal antibodies.
For more information, visit www.geminitherapeutics.com.
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