Phase 3 data show the C3 inhibitors' sustained benefit for the common form of vision loss. A primary author explains the significance of the new findings.
Complement inhibitor pegcetacoplan is associated with continued geographic atrophy (GA) lesion growth reductions at 2 different regimens over 18 months, according to new phase 3 findings.
In new primary outcome data from the DERBY and OAKS trials, presented by Apellis this week, pegcetacoplan showed up to 22% reduction in GA lesion growth versus sham injection therapy among patients with the irreversible vision loss disease.
The long-term data may now support Apellis’ submission of pegcetacoplan—which targets and inhibits the C3 pathway—to the US Food and Drug Administration (FDA) as the first potential drug indicated for treatment of GA.
Indeed, per the company, patients in the OAK trial receiving pegcetacoplan monthly reported a 22% (P <.0001) mean lesion growth reduction at 18 months, while patients on every-other-month therapy reported a still significant 16% (P = .0018) mean reduction. Among such patients in the DERBY trial, patients reported 13% (P = .0254) and 12% (P = .0332) mean reductions over the same period, respectively.
Patients treated across both trials reported similar treatment-related events through months 6 to 18, Apellis stated and data at trial’s end supported potential for continued treatment benefit with pegcetacoplan for GA lesion growth reduction over a long period of care.
In an interview with HCPLive, DERBY primary investigator Jeffrey S. Heier, MD, director of retina service and retina research at the Ophthalmic Consultants of Boston, discussed the impact of the latest findings for the potential landmark GA agent.
“The findings from the 18-month trials were very reassuring, because what they did is, first of all, show that the limitation in GA progression noted at 1 year is continued,” Heier said. “We also saw that the DERBY arm, which had not shown the same level of efficacy in the first year, actually looks closer to OAKS at 18 months.”
Heier discussed the investigators’ intent to expand assessment to 24 months, and his hope to see a continued increase in absolute GA lesion size difference between pegcetacoplan and sham therapy at 2 years.
“We know that this is a disease that’s not a 1-year, 2-year, 5-year, or even a 10-year disease,” he said. “This is a disease that goes on for the patient life.”