Business

Description of Business and Organization

IVERIC bio, Inc. (the “Company”), formerly Ophthotech Corporation, was incorporated on January 5, 2007, in Delaware. The Company is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases ("IRDs"). In April 2019, the Company changed its name from Ophthotech Corporation to IVERIC bio, Inc. as the Company continued to broaden its focus to include gene therapies.

The Company recently announced initial, top-line data from its international, randomized, double masked, sham controlled multi-center clinical trial (the "OPH2003 trial"), assessing the safety and efficacy of its most advanced product candidate, Zimura® (avacincaptad pegol), a complement C5 inhibitor, for the treatment of geographic atrophy ("GA"). GA is the advanced stage of dry age-related macular degeneration ("AMD") and is characterized by retinal cell death and degeneration of tissue in the central portion of the retina, or the macula, which may result in loss of vision. The top-line data confirmed that Zimura met the prespecified primary endpoint in the trial in reducing the rate of GA growth in patients with dry AMD. Based on the data the Company has received from the OPH2003 trial to date, the Company has commenced site selection and planning activities for a second pivotal clinical trial for Zimura in GA with the goal of initiating enrollment in this Phase 3 clinical trial during the first quarter of 2020. The Company plans to continue to explore all options for the future development and potential commercialization of Zimura, including potential collaboration and out-licensing opportunities, while it commences Phase 3 activities.

The Company's therapeutics portfolio consists of two ongoing clinical trials evaluating Zimura and its preclinical development program of High temperature requirement A serine peptidase 1 protein ("HtrA1") inhibitors. In addition to the OPH2003 clinical trial of Zimura in GA, which remains ongoing, the Company has an ongoing randomized, double masked, sham controlled clinical trial evaluating Zimura for the treatment of autosomal recessive Stargardt disease (the "OPH2005 trial"). The Company is developing its HtrA1 inhibitor program for GA and potentially other age-related retinal diseases. The Company previously evaluated Zimura in combination with Lucentis® (ranibizumab), an anti-vascular endothelial growth factor ("anti-VEGF") agent for the treatment of wet AMD, for which the Company completed a Phase 2a safety trial (the "OPH2007 trial") during the fourth quarter of 2018.

The Company's gene therapy portfolio consists of several ongoing research and preclinical development programs that use adeno-associated virus ("AAV") for gene delivery. These AAV gene therapy programs are targeting the following orphan IRDs:


•	rhodopsin-mediated autosomal dominant retinitis pigmentosa ("RHO-adRP") which is characterized by progressive and severe bilateral loss of vision leading to blindness;


•	IRDs associated with mutations in the BEST1 gene, including Best vitelliform macular dystrophy, or Best disease, which is generally characterized by bilateral egg yolk-like lesions in the macula, which, over time, progress to atrophy and loss of vision;


•	Leber congenital amaurosis type 10 ("LCA10") which is characterized by severe bilateral loss of vision at or soon after birth;


•	autosomal recessive Stargardt disease ("STGD1") which is characterized by progressive damage to the macula and retina of young adults, leading to loss of vision; and


•	IRDs associated with mutations in the USH2A gene, which include Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa.

The Company's business development efforts have resulted in the expansion of its research and development pipeline and the transition of the Company to include a focus on gene therapy. The Company initiated multiple gene therapy sponsored research programs with the University of Massachusetts Medical School ("UMMS") in February 2018 and initiated an additional gene therapy sponsored research program with UMMS for USH2A-related IRDs in July 2019. Additionally, the Company in-licensed its novel AAV gene therapy product candidate for the treatment of RHO-adRP ("IC-100") from the University of Florida Research Foundation ("UFRF") and the University of Pennsylvania ("Penn") in June 2018, in-licensed its novel AAV gene therapy product candidate for the treatment of BEST1-related IRDs, including Best disease ("IC-200") from Penn and UFRF in April 2019 and in-licensed its minigene sponsored research program for LCA10 (the "miniCEP290 program") from the University of Massachusetts in July 2019. The Company also acquired its HtrA1 inhibitor program through the acquisition of Inception 4, Inc. ("Inception 4") in October 2018.