14 May 2021 - - US-based precision medicine company Gemini Therapeutics, Inc. (NASDAQ: GMTX) has completed enrollment in its Phase 2a trial advancing GEM103 as a potential add-on therapy for patients suffering from wet AMD and requiring continued anti-vascular endothelial growth factor (anti-VEGF) treatment who have, or may be at risk for, macular atrophy, the company said.

Topline data related to safety, tolerability, effect on intraocular complement factor H levels and disease-relevant biomarkers of complement regulation is expected in late 2021.

The ongoing Phase 2a, multicenter, multiple-dose study in subjects with Neovascular Age-related Macular Degeneration with or at risk for macular atrophy, is designed to investigate the safety and tolerability of GEM103 as an adjunct to standard of care aflibercept therapy.

The study is designed to enroll approximately 45 patients randomized 2: 1 between GEM103 plus aflibercept and sham plus aflibercept arms, with treatment administered via intravitreal injection every other month for twelve months.

CFH levels and disease relevant biomarkers of complement regulation are determined from aqueous humor sampling throughout the study with visual acuity and macular atrophy size measured at defined dosing dates.

Gemini's lead program, GEM103, a full-length recombinant complement factor H protein, is believed to be the first ever recombinant native complement regulator.

GEM103 delivered by intravitreal injection is designed to address both complement hyperactivity and restore retinal health in patients with AMD.

In a genetically-defined subset of AMD patients, GEM103 may circumvent dysfunctional CFH loss-of-function variants and slow the progression of their retinal disease.

In patients undergoing anti-VEGF treatment, GEM103 may help regulate the hyperactive amplification of the alternative pathway believed to lead to cell loss and macular atrophy.

The US Food and Drug Administration granted Fast Track Designation for GEM103 for the treatment of dry AMD in patients with CFH loss of function gene variants.

Age-related macular degeneration 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 16m patients with AMD in the United States alone.

Wet AMD is the most severe form characterized by neovascularization of the retina, leading to significant loss in visual acuity and rapid progression to blindness.

There are approximately 1.4m patients with wet AMD in the United States and over 2.5m patients in the European Union. Wet AMD is most common in individuals over the age of 50, with increasing incidence every decade thereafter.

Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients.

However, there is still no approved therapy for patients with wet AMD who have or may be at risk for vision loss from macular atrophy associated with ongoing need for anti-VEGF treatment.

Gemini Therapeutics is a clinical stage precision medicine company developing novel therapeutic compounds to treat genetically defined age-related macular degeneration.

Gemini's lead candidate, GEM103, is a recombinant form of human complement factor H protein 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 and a Phase 1/2a study in patients with neovascular age-related macular degeneration with or at risk for macular atrophy.

The company has generated a rich pipeline including recombinant proteins, gene therapies, and monoclonal antibodies and is advancing a potentiating antibody for CFH, GEM307, into clinical development for treatment of systemic diseases.