XML 36 R7.htm IDEA: XBRL DOCUMENT v3.20.2
Business
6 Months Ended
Jun. 30, 2020
Organization, Consolidation and Presentation of Financial Statements [Abstract]  
Business 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").
        The Company's therapeutics portfolio consists of its clinical stage product candidate Zimura® (avacincaptad pegol), a complement C5 inhibitor, and IC-500, its preclinical product candidate from its High temperature requirement A serine peptidase 1 protein ("HtrA1") inhibitors program. 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.
        In June 2020, the Company announced 18-month data from its international, randomized, double masked, sham controlled, multi-center Phase 3 clinical trial (the "GATHER1 trial") assessing the safety and efficacy of Zimura for the treatment of GA secondary to AMD. 286 patients were enrolled in this trial across multiple treatment groups, including various Zimura doses and sham control groups and patients were treated and followed for 18 months. The 18-month data supports the previously announced 12-month data from this trial, which confirmed that Zimura met the prespecified primary efficacy endpoint in reducing the rate of GA growth with statistical significance. The reduction in the mean rate of GA growth over 18 months was 28.11% for the Zimura 2 mg group as compared to the corresponding sham control group and 29.97% for the Zimura 4 mg group as compared to the corresponding sham control group. The pre-specified efficacy analysis for the primary endpoint was performed at month 12 using all of the statistical power in the trial to detect a statistically significant difference. Therefore, the p-values for the 18 month statistical analyses are descriptive in nature. The descriptive p-values for the treatment effects at month 18 were p=0.0014 for the Zimura 2 mg group and p=0.0021 for the Zimura 4 mg group. In this trial, the treatment effect was observed as early as 6 months, with an increase in the absolute difference of the mean change in GA growth for treatment with either Zimura 2 mg or Zimura 4 mg, as compared to sham, at each subsequent time point, suggesting the progressive benefit of continuous treatment with Zimura. Zimura maintained its favorable safety profile at 18 months with no investigator reported Zimura related adverse events, no cases of endophthalmitis and a lower rate of choroidal neovascularization ("CNV") than reported for C3 inhibition. The overall 18 month data may suggest a dose response relationship. GATHER1 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 GATHER1 trial qualifies as the first of two Phase 3 trials typically required by the U.S. Food and Drug Administration ("FDA") for marketing approval of a pharmaceutical product. In light of the 12-month data, the Company changed the phase designation of the GATHER1 trial to a Phase 2/3 trial and believes it counts as a Phase 3 clinical trial.
In June 2020, the Company dosed the first patient in its second international, randomized, double masked, sham controlled, multi-center Phase 3 clinical trial of Zimura for the treatment of GA secondary to AMD (the "GATHER2 trial"). The Company plans to enroll approximately 400 patients in this trial.
The Company recently reopened enrollment in its ongoing, international, randomized, double masked, sham controlled, multi-center Phase 2b clinical trial evaluating the safety and efficacy of Zimura for the treatment of STGD1 (the "OPH2005 trial"). The Company plans to enroll approximately 25 additional patients, with the goal of enrolling a total of approximately 120 patients as was initially intended in the protocol for the OPH2005 trial.
        The Company is developing IC-500 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 non-syndromic 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, the Company plans to file an IND and, subject to regulatory review, initiate a Phase 1/2 clinical trial for IC-200 in 2021.