Gemini Therapeutics announced it has initiated enrollment in its phase 1 study of GEM103, a recombinant, native Complement Factor H (CFH) for the treatment of dry age-related macular degeneration. AMD is the leading cause of irreversible blindness in the western world—affecting millions of people in the U.S. alone. At least 90% of AMD patients have dry AMD, for which there are no approved therapies.
“Gemini was founded to deliver precision medicines for eye disorders by combining genetic insights with clinical and biological expertise in complement. We are excited to take GEM103 into the clinic as a novel approach in complement modulation for a devastating disease with no therapeutic options,” Jason Meyenburg, Chief Executive Officer of Gemini, said in a company news release “We look forward to topline data from this study and the potential to initiate a phase 2 study, supported by data from our ongoing CLARITY natural history study examining the pathogenesis and progression of dry AMD in patients carrying CFH risk variants.”
CFH risk variants are reported to occur in greater than 40% of patients with dry AMD and are strongly associated with the risk of developing the disease. 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. GEM103 is a fully functional recombinant native CFH protein intended to address dysregulation caused by loss of CFH function variants in the eyes of these AMD patients.
“The management of dry AMD, due to its prevalence and pronounced impact on the patient’s quality of life, continues to be a challenge as there are no treatment options available,” Arshad Khanani, MD, MA, Managing Partner and Director of Clinical Research at Sierra Eye Associates, Reno, Nevada and an investigator in the study, said in a news release. “Clinicians are looking for breakthrough treatments to disrupt the pathology of dry AMD. The concept of treating genetically selected patients with GEM103 to potentially modulate and normalize the alternative complement pathway is an exciting approach.”
This study is a phase 1 single ascending dose (SAD) clinical study evaluating the safety, pharmacokinetics and pharmacodynamics of GEM103. The study targets enrolment of approximately 9 patients with dry AMD who have known CFH mutations.