Apellis Pharmaceuticals has responded to “positive” 24-month data for its pioneering geographic atrophy (GA) therapy published in The Lancet, demonstrating “that SYFOVRE is a clinically meaningful treatment” for the disease.
One of the world’s most prestigious peer-reviewed journals, The Lancet published two-year data from the Phase 3 OAKS and DERBY studies evaluating SYFOVRE (pegcetacoplan injection) for GA secondary to age-related macular degeneration (AMD).
Earlier in 2023, SYFOVRE became the first GA therapy approved by US drug regulators, with five additional applications under review globally. It is now the only treatment approved for use beyond 12 months in GA.
In the publication, Apellis said both every-other-month and monthly SYFOVRE showed a clinically meaningful reduction of GA lesion growth with increasing effects over time and a well-demonstrated safety profile in a broad population of more than 1,200 patients.
In OAKS at 24 moths, SYFOVRE administered monthly and every other month slowed GA 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), respectively, compared with sham.
This was compared to 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 OAKS.
At 24 months in DERBY, SYFOVRE administered monthly and every-other-month slowed GA lesion growth 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), respectively, compared with sham.
This was compared to 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 in DERBY – a result that did not reach significance.
Overall, there were no differences in key secondary visual function endpoints 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,” said Dr Charles Wykoff, senior author and director of research, Retina Consultants of Texas.
“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.”
Apellis chief medical officer Dr Caroline Baumal said the 24-month data demonstrated 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,” she said.
“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.”