Pegcetacoplan treatment effect accelerated between months 18-24 at reducing GA lesion growth in phase 3 DERBY and OAKS.
New top-line data from the phase 3 DERBY and OAKS trials at 24 months show increased effects over time with intravitreal pegcetacoplan in geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Pecetacoplan treatment effect accelerated between months 18 - 24, while demonstrating clinically meaningful reduction in GA lesion growth compared to sham, according to Apellis Pharmaceuticals, Inc.
The report showed DERBY had a 36% monthly reduction (P <.0001) and 29% reduction every-other-month (P = .0002), while OAKS had a 24% monthly reduction (P = .0080) and 25% reduction every-other-month (P = .0007).
“I am thrilled to see the increased effects over time, which suggest that pegcetacoplan is preserving more and more photoreceptor cells that are directly responsible for vision,” said Eleonora Lad, MD, PhD, Duke University Medical Center in a statement. “The vision loss caused by GA is devastating for patients, taking away their ability to perform critical daily tasks like driving, reading, and recognizing faces. It is very exciting to finally be on the brink of the first potential GA treatment with pegcetacoplan.”
A pre-specified analysis of GA lesion growth over 24 months additionally suggested both monthly and every-other-month pegcetacoplan showed a clinically meaningful reduction in GA lesion growth from baseline compared to sham.
DERBY had 19% monthly reduction (P = .0004) and a 16% reduction every-other-month (P = .0030) and OAKS had a 22% monthly reduction (P <.0001) and an 18% every-other-month reduction (P = .0002).
Additionally, the reduction of GA lesion growth in patients with extrafoveal lesions (28% monthly, 28% every-other-month) was comparable to the reduction in patients with foveal lesions (34% monthly; 28% every-other-month) in the combined studies between months 18 and 24.
There was no clinically meaningful difference observed between pegcetacoplan and sham in key secondary endpoints measuring visual function at 24 months. Visual function outcomes at 24 months were expected to be due to the limitations of the endpoints used for GA and the relatively early assessment timeframe.
The agent additionally continued to demonstrate a favorable safety profile, consistent with safety data to date and longer-term exposure to intravitreal injections. There were no cases of endophthalmitis reported between months 18 and 24.
"These data further reinforce the breakthrough potential of pegcetacoplan, with both monthly and every-other-month treatment demonstrating increased effects across a broad patient population over 24 months,” said Jeffrey Eisele, PhD, chief development officer, Apellis in a statement.
Patients will be treated with pegcetacoplan in the GALE extension study for an additional three years. The US marketing application is under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action data of November 26, 2022.