Apellis Pharmaceuticals announced that it has commenced its phase 3 clinical program for APL-2 in patients with geographic atrophy (GA), consisting of two phase 3 trials (DERBY & OAKS.)
The first patient has been dosed in the OAKS trial and the first patient has been enrolled in the DERBY trial. Both trials will assess the safety and efficacy of APL-2 in patients with GA, an advanced form of age-related macular degeneration. There are approximately 1 million patients in the United States with GA and there are currently no FDA-approved treatments for the disease, which leads to loss of visual function and blindness.
“Dosing the first patient in our phase 3 program in geographic atrophy is an exciting milestone for Apellis, physicians, and most importantly patients suffering from GA,” Cedric Francois, MD, PhD, co-founder and CEO of Apellis, said in a company news release. “APL-2 offers hope for patients currently without an approved treatment option.”
“Patients living with GA over time lose their ability to drive, read and perform daily functions, so a treatment that could potentially reduce the rate of GA lesion growth and save visual function is important to this population,” said Nathan Steinle, MD, Retina Specialist at California Retina Consultants.
The phase 3 program consists of two 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled studies (DERBY & OAKS) to assess the efficacy and safety of multiple intravitreal (IVT) injections of APL-2 in patients with GA. The Phase 3 trials are similar in design to Apellis’ Phase 2 FILLY trial, including the eligibility criteria and primary endpoint of change in GA lesion size from baseline to month 12, compared to sham. In DERBY and OAKS, patients will continue on therapy for an additional 12 months beyond the 12-month primary endpoint. APL-2 met its primary endpoint in the phase 2 FILLY trial. At 12 months, APL-2, administered monthly via intravitreal injection, showed a 29% (P=0.008) reduction in the rate of GA lesion growth compared to sham. With every other month administration, a 20% (P=0.067) reduction compared to sham was observed.
Apellis recently received Fast Track Designation from the FDA for APL-2 for the treatment of GA.