Apellis Initiates Registrational Programs of Pegcetacoplan in Patients with C3G / IC-MPGN and ALS, Rare Diseases with High Unmet Need - Seite 2

With these trials, Apellis will be the only company actively studying a potential treatment for patients with IC-MPGN, a disease where both the alternative and classical pathways have been implicated, which supports targeting the complement cascade centrally at C3 as a potential therapeutic option.

The company is advancing pegcetacoplan in C3G / IC-MPGN based on data in C3G from the Phase 2 DISCOVERY trial. Data at week 12 were positive; results at 48 weeks will be presented at a scientific congress later this year.

Neurology: Potentially Registrational Phase 2 Study in ALS

Apellis is expanding the development of pegcetacoplan into neurology with a potentially registrational Phase 2 study in ALS, a disease that impacts approximately 225,000 patients worldwide.⁴

In individuals with ALS, high levels of activated C3 are present at the neuromuscular junction⁵ where the neurons communicate directly to muscle cells. Multiple studies suggest that the elevated levels of C3 present in ALS may be responsible for the chronic neuroinflammation that leads to motor neuron death.^5,6,7

Apellis has initiated the MERIDIAN study, a randomized, placebo-controlled trial of pegcetacoplan in approximately 200 adults with sporadic ALS, and expects to dose the first patient by the end of 2020. The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores at week 52.

About the Phase 2 NOBLE study in C3G / IC-MPGN
The Phase 2 NOBLE study (APL2-C3G-204; NCT04572854) is a multicenter, open-label, randomized, controlled study designed to evaluate the efficacy and safety of pegcetacoplan in up to 12 adults who have post-transplant recurrence of C3G or IC-MPGN. Study participants will be randomized in a 3:1 ratio to receive pegcetacoplan or maintain standard of care for 12 weeks and then all patients in the study will receive pegcetacoplan from week 13 to week 52.

The primary endpoint of the study is the proportion of patients with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan. Secondary endpoints include an evaluation of safety, the proportion of patients with reduction in C3c staining on renal biopsy after 52 weeks of treatment, and the proportion of patients achieving at least a 50% reduction in proteinuria.

About the Phase 2 MERIDIAN Study in ALS
The Phase 2 MERIDIAN study (APL2-ALS-206) is a potentially registrational, randomized, placebo-controlled, multicenter study that was designed to evaluate the efficacy and safety of pegcetacoplan in approximately 200 adults with sporadic ALS. Study participants will be randomized in a 2:1 ratio to receive pegcetacoplan or placebo while continuing to receive their existing standard of care treatment for ALS. After one year, all patients in the study will receive pegcetacoplan. To reduce the burden on people living with ALS and their caregivers, the trial has been designed to minimize the number of in-clinic visits, with approximately six clinic visits in the first year and four in the open-label second year.