As part of its ongoing new drug application (NDA) process with the FDA, Apellis Pharmaceuticals plans to submit 24-month data from the phase 3 DERBY and OAKS studies on pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
First off, what is APL-2?
APL-2 is an investigational targeted C3 therapy that works by regulating excessive activation of the complement cascade, which can lead to the onset and progression of diseases. The FDA previously granted it a Fast Track designation for GA treatment.
Tell us about the studies.
The DERBY and OAKS studies were randomized, double-masked, and sham-controlled, with 621 (OAKS) and 637 patients (DERBY) enrolled. Both studies compared APL-2’s efficacy and safety with the use of sham injections. Investigators tracked the efficacy of monthly and bimonthly APL-2in patients with GA assessed by change in the total area of GA lesions from baseline measured by fundus autofluorescence at 12 months. Patients continued to receive masked treatment for 24 months. (via)
What did the data show?
Both monthly and bimonthly injections showed a clinically meaningful reduction—36% in DERBY and 29% in OAKS—in GA lesion growth when compared to the sham injections from months 18 to 24
What would this mean for patients?
If approved, APL-2 would be the first FDA-approved treatment therapy for patients with GA.
What’s next?
Apellis plans on submitting the phase 3 efficacy data to the FDA later this month. The company plans on meeting a Prescription Drug User Fee Act (PDUFA) target action date in February 2023.