Gemini Therapeutics Announces Initiation of 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
- Category: Proteins and Peptides
- Published on Monday, 01 February 2021 14:16
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- Topline Data Expected in 2H 2021
CAMBRIDGE, MA, 2021 I February 01, 2021 I Gemini Therapeutics, a clinical stage precision medicine company developing innovative treatments for genetically defined AMD, today 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,” said Marc E. Uknis, M.D., F.A.C.S., Chief Medical Officer of Gemini Therapeutics. “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,” said Jason Meyenburg, Chief Executive Officer of Gemini Therapeutics. “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.
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.
About Wet 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. 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 1.4 million patients with wet AMD in the United States and over 2.5 million 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.
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 multiple indications.
For more information, visit www.geminitherapeutics.com.
SOURCE: Gemini Therapeutics