Iveric Bio submitted the first part of a new drug application to the FDA for avacincaptad pegol (ACP; Zimura), a novel investigational complement C5 protein inhibitor for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). This follows Iveric Bio receiving Fast Track designation for ACP in 2020. (via)
How does ACP work?
Overactivity within the complement system and the C5 protein are thought to play key roles in the development and growth of scarring and vision loss linked with GA secondary to AMD. By focusing on C5, ACP may decrease the complement system’s activity that can lead to retinal cell degeneration— thus slowing GA progression.
Tell me about the clinical trials.
The NDA submission is based on the phase 3 GATHER1 and GATHER2 clinical trials. ACP met its primary endpoint in both GATHER1 and GATHER2, which is ongoing.
Both trials measured the safety and efficacy of 2 mg of ACP administered intravitreally on a monthly basis for 12 months in patients with GA secondary to AMD. GATHER1 included 286 participants and GATHER2 included 448 participants.
Primary efficacy endpoints were based on GA area measured by fundus autofluorescence at three timepoints: baseline, 6 months, and 12 months. The mean rate of growth in the GA area from baseline to month 12, based on data, was 35% in GATHER1 and 18% in GATHER2.
Any adverse reactions?
Between both trials, the most frequently reported adverse events were in relation to the injection procedure.
The most common adverse reactions of those patients who received the 2 mg ACP dosage included conjunctival hemorrhage (13%), increased IOP (9%), and choroidal neovascularization (7%).