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Gemini Therapeutics (Cambridge, MA) expect to announce Phase 2a results in H12021 for dry AMD therapy.

Gemini Therapeutics (Nasdaq: GMTX), a US-based clinical stage ophthalmic company focused on age-related macular degeneration (AMD), has announced a Phase 2a treatment (“ReGAtta” study) and is expected to share topline data from the dry AMD trial in the first half of 2021. The company was recently granted a Fast Track designation from the FDA to facilitate development and expedite review of their investigational therapy to address unmet medical needs.  The study 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. According to the company, the interventional clinical trial is designed to evaluate safety and tolerability data, as well as measures of intraocular pharmacokinetics (PK) and other disease-relevant biomarkers.


The company’s recombinant CFH protein provides a regulatory protein to restore appropriate complement regulation by blocking the detrimental effects of the pathology, including inappropriate cell lysis and exaggerated immune responses.  The GEM103 product  aims to retain the beneficial effects of the disorder, specifically, the clearance of extracellular debris and the repair of oxidative damage related to the complement pathway activation. According to the company, there are 15 million dry AMD patients globally including 6 million patients who harbour variants in the complement factor H (CFH) gene.  GEM103 is proposed to be “the first ever recombinant native complement modulator, full-length recombinant complement factor H (rCFH) protein”.  A wealth of medical literature is available on complement factor H and AMD at NIH PubMed (


Following on the announcement of the enrolment of an estimated 60 patients, the company stated that. “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.  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.”  The clinical trial is listed under, referenced NCT04643886, and the Phase 2 study will recruit 29 study sites in the US and will be sponsored by Gemini Therapeutics.