• Zimura also being studied in wet AMD,  idiopathic polypoidal choroidal vasculopathy, and  Stargardt disease
• Initial Top-line Zimura Geographic Atrophy Data Expected in the Fourth Quarter of 2019 -

Ophthotech Corporation (NASDAQ:OPHT) announced  completion of patient recruitment for its Phase 2b clinical trial of Zimura (avacincaptad pegol), the Company’s complement factor C5 inhibitor, monotherapy in patients with geographic atrophy secondary to dry age-related macular degeneration (AMD). Complement factor C5 is a central component of the complement cascade and is believed to be involved in the development and progression of AMD. A total of 286 patients have been enrolled into this randomized, double-masked, sham controlled multi-center clinical trial. This clinical trial is designed to assess the safety and efficacy of various Zimura dosing regimens over 12 months. Patients will continue to be treated and monitored until month 18.

“We are grateful to our principal investigators and their dedicated clinical staffs for their interest and support of our Zimura program, enabling on time completion of recruitment for our clinical trial in patients with geographic atrophy secondary to dry AMD,” stated Kourous A. Rezaei, M.D., Chief Medical Officer of Ophthotech. “Recent clinical data together with the pre-clinical scientific evidence implicating complement in various retinal diseases further invigorates our enthusiasm for the therapeutic potential of Zimura. We look forward to initial top-line data from this clinical trial, which is expected to be available during the fourth quarter of 2019.”

The Company also expects initial top-line data for its open-label Phase 2a clinical trial of Zimura combination therapy with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis (ranibizumab) 0.5 mg in treatment-naïve patients with wet AMD before the end of this year. A total of 64 patients have been enrolled into this randomized, dose-ranging, open-label, uncontrolled, multi-center trial. This trial is designed to assess the safety of different dosages of Zimura in combination with Lucentis and to detect a potential efficacy signal at month 6. Following the completion of this trial, clinical data will be analyzed to assess whether to proceed to a randomized, sham-controlled clinical trial of Zimura combination therapy with anti-VEGF in wet AMD. In January of 2018, the company began a trial of Zimura for the treatment of autosomal recessive Stargardt disease. Additionally it also began an open-label phase 2a trial of Zimura in combination with anti-VEGF therapy for idiopathic polypoidal choroidal vasculopathy.

Zimura is designed to target and inhibit the complement protein C5. Zimura is believed to bind to C5 and inhibit it from cleaving into the terminal fragments, C5a and C5b. By inhibiting the formation of complement system terminal fragments, Zimura may decrease the activation of inflammasomes and decrease the formation of membrane attack complex (MAC), thereby potentially avoiding or slowing the degeneration of RPE cells and providing the rationale as a potential therapy for the retinal conditions that are being targeted.

Dry AMD is a significant cause of moderate and severe loss of central vision, affecting both eyes in the majority of patients. Although dry AMD is the most common form of AMD, there are no U.S. Food and Drug Administration or European Medicines Agency approved therapies to treat this condition. In dry AMD, thinning of the retinal pigment epithelial (RPE) cells in the macula develops, along with other age-related changes to the adjacent retinal and choroidal tissue layers. Geographic atrophy, the end stage of dry AMD, is a disease characterized by degeneration of retinal tissue leading to further loss of vision.