GEM103 shows potential for dry AMD, geographic atrophy treatment

The phase 1 study of GEM103 and geographic atrophy met all endpoints, according to research presented at the virtual American Academy of Ophthalmology annual meeting.

Arshad M. Khanani, MD, MA, managing partner and director of clinical research, Sierra Eye Associates, clinical associate professor of ophthalmology, University of Nevada, reviewed his findings from the phase 1 single-ascending dose trial.

He reported GEM103 produced extended supraphysiologic levels of complement factor H (CFH) and zero dose-limiting toxicities or treatment-related adverse reactions in patients with dry age-related macular degeneration or central geographic atrophy.

“GEM103 was well-tolerated with no drug reactions, and the pharmacokinetic/pharmacodynamic data for serial aqueous humor analysis showed that we are able to achieve supraphysiologic levels of CFH in these patients, which were maintained up to 8 weeks for a dose level 100 ug or higher,” Khanani said. “We also saw biomarker activity where there were reductions in biomarker of complement activation, … essentially showing that GEM103 has the potential for replenishing the complement factor H levels in patients who have the genetic variant, which is about 35% to 40% of patients with dry AMD.”

Khanani concluded, “The key takeaway is that we can use GEM103, and the hope is that we can decrease or slow down the progression of dry AMD by specifically targeting patients with genetic variants of complement factor H and treating them with GEM103.”