The Lancet Publishes 24-Month Results from Phase 3 Studies of Syfovre for GA
Apellis Pharmaceuticals announced that The Lancet has published 24-month results from the phase 3 OAKS and DERBY studies evaluating Syfovre (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Apellis received FDA approval for Syfovre, a targeted C3 therapy, in February, becoming the first FDA approved drug for GA. The drug is the only treatment approved for use beyond 12 months in GA.
Between August 30, 2018, and July 3, 2020, 1,258 patients were enrolled in OAKS and DERBY. The modified intention-to-treat populations comprised 614 (96%) of 637 patients in OAKS (202 receiving pegcetacoplan monthly, 205 pegcetacoplan every other month, and 207 sham), and 597 (96%) of 621 patients in DERBY (201 receiving pegcetacoplan monthly, 201 pegcetacoplan every other month, and 195 sham).
In OAKS, pegcetacoplan monthly and pegcetacoplan every other month significantly slowed geographic atrophy lesion growth by 21% (absolute difference in least-squares mean –0·41 mm2, 95% CI –0·64 to –0·18; P=0·0004) and 16% (–0·32 mm2, –0·54 to –0·09; p=0·0055), respectively, compared with sham at 12 months. In DERBY, pegcetacoplan monthly and pegcetacoplan every other month slowed geographic atrophy lesion growth, although it did not reach significance, by 12% (–0·23 mm2, –0·47 to 0·01; P=0·062) and 11% (–0·21 mm2, –0·44 to 0·03; p=0·085), respectively, compared with sham at 12 months.
At 24 months, pegcetacoplan monthly and pegcetacoplan every other month slowed geographic atrophy lesion growth by 22% (–0·90 mm2, –1·30 to –0·50; P<0·0001) and 18% (–0·74 mm2, –1·13 to –0·36; P=0·0002) in OAKS; and by 19% (–0·75 mm2, –1·15 to –0·34; P=0·0004) and 16% (–0·63 mm2, –1·05 to –0·22; P=0·0030) in DERBY, respectively, compared with sham. There were no differences in key secondary visual function endpoints at 24 months.
Serious ocular treatment-emergent adverse events were reported in five (2%) of 213, four (2%) of 212, and one (<1%) of 211 patients in OAKS; and in four (2%) of 206, two (1%) of 208, and two (1%) of 206 patients in DERBY receiving pegcetacoplan monthly, pegcetacoplan every other month, and sham, respectively, at 24 months. New-onset exudative AMD was reported in 24 (11%), 16 (8%), and four (2%) patients in OAKS; and in 27 (13%), 12 (6%), and nine (4%) patients in DERBY receiving pegcetacoplan monthly, pegcetacoplan every other month, and sham, respectively, at 24 months.
“The increasing treatment effects of Syfovre over time with both monthly and every-other-month dosing are exciting to see and meaningful for the field,” Charles Wykoff, MD, PhD, senior author and director of research, Retina Consultants of Texas, said in a company news release. “The vision loss caused by GA takes a tremendous toll on patients and their families, impacting independence and well-being. Syfovre is a major advance for patients living with this progressive and relentless disease.”
“These 24-month data demonstrate that Syfovre is a clinically meaningful treatment for GA, and we are proud these results were published in one of the world's most prestigious peer-reviewed journals,” said Caroline Baumal, MD, chief medical officer, Apellis. “With Syfovre's approval in the United States and five additional applications under review globally, we are working urgently to bring this important treatment to patients in need worldwide.”
Publication co-first authors are Jeffrey Heier, MD, and Eleonora Lad, MD, PhD.