Business

3 Months Ended

Mar. 31, 2020

Organization, Consolidation and Presentation of Financial Statements [Abstract]

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").

The Company's therapeutics portfolio consists of Zimura® (avacincaptad pegol), a complement C5 inhibitor, and its preclinical development program of High temperature requirement A serine peptidase 1 protein ("HtrA1") inhibitors. The Company is currently targeting the following diseases with Zimura:


•	geographic atrophy ("GA"), which is the advanced stage of age-related macular degeneration ("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 is preparing for a second international, randomized, double masked, sham controlled, multi-center Phase 3 clinical trial of Zimura for the treatment of GA secondary to AMD (the "ISEE2008 trial"). In March 2020, because of the novel coronavirus ("COVID-19") global pandemic and out of an abundance of caution for the health and safety of its elderly patients and participating physicians and their staffs, the Company decided to delay the initiation of patient enrollment in the ISEE2008 trial even though it was on track to begin enrollment that month. Although it delayed the initiation of patient enrollment, it has continued trial initiation activities and plans to begin enrolling patients expeditiously once the COVID-19 situation stabilizes and the Company and its investigators determine it is safe to do so. The Company will continue to monitor the situation in the United States and across the world and may commence patient enrollment in stages based on the conditions in different communities and countries. It plans to enroll approximately 400 patients in this trial.

In addition, the Company is expecting to receive, by the end of the second quarter of 2020, 18-month data from its international, randomized, double masked, sham controlled, multi-center Phase 2/3 clinical trial (the "OPH2003 trial") assessing the safety and efficacy of Zimura for the treatment of GA secondary to AMD, for which it announced positive, 12-month data in October 2019. 286 patients were enrolled in this trial across multiple treatment groups, including various Zimura doses and sham control groups. The 12-month data confirmed that Zimura met the prespecified primary endpoint in the trial in reducing the rate of GA growth in patients with GA. 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, based on recent guidance from the U.S. Food and Drug Administration ("FDA"), 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. 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. Based on the 12-month data, the Company believes that the OPH2003 trial qualifies as the first of two Phase 3 trials typically required by the FDA for marketing approval of a pharmaceutical product. In light of the 12-month data, the Company has changed the phase designation of the OPH2003 trial to a Phase 2/3 trial. The primary purpose of the 18-month time point is to gather additional safety data. The primary efficacy analysis for this trial was conducted following the 12-month time point, and the reliability of any such comparison and the ability to rule out a “false positive” result was concentrated in the 12-month analysis. The prespecified statistical analysis plan for the trial, therefore, does not include assessing the statistical significance of 18-month data for rate of GA growth in the treatment groups as compared to the corresponding sham control groups. Any analysis of the 18-month GA growth data will be descriptive only and is intended primarily to evaluate the safety of Zimura.

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 two product candidates in preclinical development and several ongoing collaborative sponsored research programs, each of which uses adeno-associated virus ("AAV") for gene delivery. These AAV mediated 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; and


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

The Company's lead gene therapy product candidate is IC-100, which it is developing for the treatment of RHO-adRP. Subject to successful completion of preclinical development and manufacturing under current good manufacturing practices ("cGMP"), the Company plans to file an investigational new drug application ("IND") for IC-100 by the end of 2020 or early 2021 with the goal of commencing patient enrollment in a Phase 1/2 clinical trial during the first half of 2021. The Company is also developing IC-200, its gene therapy product candidate for the treatment of IRDs associated with mutations in the BEST1 gene. Subject to successful completion of preclinical development and manufacturing under cGMP and regulatory review, it expects to initiate a Phase 1/2 clinical trial for IC-200 during the first half of 2021.