Despite varied results, the company will submit a New Drug Application for pegcetacoplan for geographic atrophy to the FDA early next year.
Apellis Pharmaceuticals Inc. this week reported top-line results from the Phase 3 DERBY and OAKS studies evaluating intravitreal pegcetacoplan, an investigational targeted C3 therapy, in 1,258 adults with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
According to the company, its OAKS study for pegcetacoplan met the primary endpoint, as it significantly reduced geographic atrophy (GA) lesion growth, while the DERBY study missed its primary endpoint of GA lesion growth reduction.
Based on results from the studies, the company plans to submit a New Drug Application (NDA) for pegcetacoplan for GA to the FDA in the first half of 2022.
“These results underscore the potential for pegcetacoplan to become the first treatment for geographic atrophy, a progressive and irreversible disease that robs patients of their vision and for which no treatment exists,” Jeffrey S. Heier, MD, principal investigator of the DERBY study and director, retina service and director, retinal research at Ophthalmic Consultants of Boston, said in a statement. “Pegcetacoplan demonstrated a clinically meaningful slowing of disease progression with an even stronger effect in GA patients with extrafoveal lesions.”
OAKS met the primary endpoint for both monthly and every-other-month treatment with pegcetacoplan, demonstrating a significant reduction in GA lesion growth of 22% (p=0.0003) and 16% (p=0.0052), respectively, compared to pooled sham at 12 months,
DERBY did not meet the primary endpoint of GA lesion growth, showing a reduction of 12% (p=0.0528) and 11% (p=0.0750) with monthly and every-other-month treatment, respectively, compared to pooled sham at 12 months.
Federico Grossi, MD, PhD, chief medical officer of Apellis, said in a statement the company’s mission is to “develop transformative therapies for people with complement-driven diseases and now, after decades of challenges in this complex disease, pegcetacoplan is the first investigational therapy to significantly slow the progression of GA in a large Phase 3 study.”
“Across our ophthalmology development program, pegcetacoplan has demonstrated an efficacy and safety profile with both monthly and every-other-month dosing that we believe supports treatment for GA patients,” he added in the statement. “We look forward to working with regulatory authorities to bring this medicine to patients in need as quickly as possible.”
According to the company, pegcetacoplan was well tolerated in both Phase 3 studies. The pooled rate of new-onset exudations was 6% of patients in the monthly pegcetacoplan groups, 4.1% in the every-other-month pegcetacoplan groups, and 2.4% in the sham groups.
Two cases of confirmed infectious endophthalmitis and one case of suspected infectious endophthalmitis were observed in the study eye out of a total of 6,331 injections (0.047%). Thirteen events of intraocular inflammation were observed in the studies (0.21% per injection). No events of retinal vasculitis or retinal vein occlusion were observed. There were no clinically relevant changes in vision for patients who developed infectious endophthalmitis or intraocular inflammation.
The company continues to analyze results from the studies, and detailed data will be presented at upcoming scientific meetings.