Ophthotech Completes Patient Recruitment in the Phase 2a Clinical Trial of Zimura® in Combination with Anti-VEGF Therapy in Wet Age-Related Macular Degeneration


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- Initial Top-line Data Expected by the End of 2018 -

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Ophthotech Corporation (NASDAQ:OPHT) today announced the completion of patient enrollment in its Phase 2a clinical trial of Zimura® (avacincaptad pegol), the Company's complement factor C5 inhibitor, in patients with wet age-related macular degeneration (AMD). Zimura is administered in combination with Lucentis® (ranibizumab), an anti-vascular endothelial growth factor (anti-VEGF) agent, in treatment naïve patients with wet AMD. A total of 64 patients have been enrolled into this dose-ranging, open-label, multi-center trial. This uncontrolled trial is designed to assess safety at different dosages and to detect a potential efficacy signal. The Company will evaluate data at month 6 and expects initial top-line data to become available before the end of 2018.

"We are pleased with the enthusiasm shown by the principal investigators in the recruitment for this clinical trial leading to on time enrollment of patients," stated Kourous Rezaei, M.D., Chief Medical Officer of Ophthotech. "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 in wet AMD."

Zimura is also currently being investigated as monotherapy in a Phase 2b clinical trial for patients with geographic atrophy secondary to dry AMD and in a Phase 2b clinical trial in patients with autosomal recessive Stargardt disease (STGD1). Further, Zimura is being evaluated as combination therapy with Eylea® (aflibercept), an anti-VEGF agent, in patients with idiopathic polypoidal choroidal vasculopathy (IPCV).

About Zimura in Wet AMD

Zimura is designed to target and inhibit the complement protein C5. Zimura binds to and inhibits C5 from being cleaved into C5a and C5b, potentially preventing the formation of inflammasomes and the accumulation of membrane attack complex (MAC), preventing cell death. Further, when used in combination with anti-VEGF therapy, Zimura may counteract the anti-VEGF induced complement upregulation, thereby providing the rationale as a potential combination therapy for patients with wet AMD and IPCV.


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About Ophthotech Corporation

Ophthotech is a science-driven biopharmaceutical company specializing in the development of novel therapies to treat ophthalmic diseases, with a focus on age-related and orphan retinal diseases. For more information, please visit www.ophthotech.com.

Forward-looking Statements

Any statements in this press release about Ophthotech's future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about Ophthotech's strategy, future operations and future expectations and plans and prospects for Ophthotech, and any other statements containing the words "anticipate," "believe," "estimate," "expect," "intend", "goal," "may", "might," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. In this press release, Ophthotech's forward-looking statements include statements about the implementation of its strategic plan, the timing, progress and results of clinical trials and other research and development activities. Such forward-looking statements involve substantial risks and uncertainties that could cause Ophthotech's clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the initiation and the conduct and design of research programs and clinical trials, availability of data from these programs, other factors discussed in the "Risk Factors" section contained in the quarterly and annual reports that Ophthotech files with the Securities and Exchange Commission. Any forward-looking statements represent Ophthotech's views only as of the date of this press release. Ophthotech anticipates that subsequent events and developments will cause its views to change. While Ophthotech may elect to update these forward-looking statements at some point in the future, Ophthotech specifically disclaims any obligation to do so except as required by law.

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