Gemini Therapeutics announced that GEM103, the company’s investigational treatment for dry AMD, has been granted Fast Track designation by the FDA. Gemini is evaluating GEM103, a recombinant, human complement factor H (CFH), for its ability to address the multiple AMD pathobiologies in genetically-defined subsets of AMD patients caused by CFH loss-of-function variants and slow the progression of their disease. CFH loss-of-function variants are present in approximately 40% of patients with AMD. The ongoing phase 2a ReGAtta study is evaluating GEM103 in patients with geographic atrophy (GA) secondary to dry AMD.
“We are pleased that the FDA has granted Fast Track designation to GEM103, which has the potential to be an important therapeutic option for patients with dry AMD and CFH loss-of- function gene variants,” Jason Meyenburg, CEO of Gemini Therapeutics, said in a company news release. “The FDA’s recognition of the existing unmet need in this patient population and this decision underscores the potential of GEM103 and its unique mechanism of action to treat this large population of AMD patients.”
Fast Track is an FDA designation intended to facilitate development and expedite review of investigational therapies to address unmet medical needs in the treatment of conditions for which nonclinical or clinical data has demonstrated the potential of the therapy to address this medical need. Benefits of this designation include more frequent engagements with the FDA to discuss the drug’s clinical development plan, eligibility for Accelerated Approval and Priority Review, and a Rolling Review, in which Gemini has the opportunity to submit completed sections of a Biologics Licensing Application (BLA) for review by the FDA, rather than waiting to complete the regulatory submission before it can be submitted for review.
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 6 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.