Business

Description of Business and Organization

IVERIC bio, Inc. (the “Company” or “IVERIC”) 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 therapeutics 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 it continued to broaden its focus to include gene therapies.

The Company believes that both therapeutics and gene therapy serve important roles in drug development and providing potential treatment options for patients suffering from retinal diseases. Its most advanced therapeutic product candidate is Zimura® (avacincaptad pegol), a complement C5 inhibitor. In October 2019, the Company announced initial, top-line data from a randomized, controlled clinical trial ("OPH2003 trial") of Zimura in geographic atrophy ("GA") secondary to dry age-related macular degeneration ("AMD"). 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. The reduction in the mean rate of GA growth over 12 months using the square root transformation was 0.110 mm (p-value = 0.0072) for the Zimura 2 mg group as compared to the corresponding sham control group and 0.124 mm (p-value = 0.0051) for the Zimura 4 mg group as compared to the corresponding sham control group, indicating an approximate 27% relative reduction in the mean rate of GA growth over 12 months when compared with sham for both dose groups. These data for both dose groups were statistically significant and the Company believes demonstrate a clinically relevant reduction in rate of GA growth. Based on its review of the safety data to date, Zimura was generally well tolerated over 12 months of administration in this clinical trial. Over this 12-month period, there were no investigator-reported ocular serious adverse events, no Zimura-related adverse events, no cases of Zimura-related intraocular inflammation, no cases of Zimura-related increased intraocular pressure, no cases of endophthalmitis, and no discontinuations attributed by investigators to Zimura. OPH2003 was designed to be a Phase 2b screening trial, with the potential to qualify as a pivotal trial depending on the magnitude and statistical significance of the potential benefit observed. The Company believes that the safety and efficacy results from the OPH2003 trial could potentially satisfy the U.S. Food and Drug Administration's ("FDA's") requirements as one of the two pivotal clinical trials typically required for marketing approval of a pharmaceutical product. The Company is preparing for an international, randomized, double masked, sham controlled, multi-center Phase 3 clinical trial of Zimura in this indication ("ISEE2008 trial") with the goal of beginning patient enrollment during the first quarter of 2020.

The Company currently has two gene therapy product candidates in preclinical development and several collaborative gene therapy sponsored research programs ongoing. Subject to successful completion of preclinical development and manufacturing under current good manufacturing practices, and regulatory review, the Company plans to initiate a Phase 1/2 clinical trial for IC-100, its lead gene therapy product candidate, during the fourth quarter of 2020.

The Company's therapeutics portfolio consists of Zimura and its preclinical development program of High temperature requirement A serine peptidase 1 protein ("HtrA1") inhibitors. The Company is targeting the following diseases with Zimura:


•	GA, which is the advanced stage of AMD, and is characterized by marked thinning or atrophy of retinal tissue, leading to irreversible loss of vision; and


•	autosomal recessive Stargardt disease ("STGD1"), which is characterized by progressive damage to the central portion of the retina (the "macula") and other retinal tissue of young adults, leading to loss of vision.

The Company previously also 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 clinical trial during the fourth quarter of 2018. The Company is developing its HtrA1 inhibitor program for GA and potentially other age-related retinal diseases.

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 ("Best disease"), which is generally characterized by bilateral egg yolk-like lesions in the macula that, 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; and


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