Business

6 Months Ended

Jun. 30, 2021

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Business

Business

Description of Business and Organization

IVERIC bio, Inc. (the “Company” or “IVERIC”) 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 believes that both therapeutics and gene therapy serve important roles in drug development and providing potential treatment options for patients suffering from retinal diseases.

The Company's therapeutics portfolio consists of its clinical stage product candidate Zimura® (avacincaptad pegol), a complement C5 inhibitor, and its preclinical product candidate IC-500, a High temperature requirement A serine peptidase 1 protein (“HtrA1”) inhibitor. 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;

•drusen secondary to AMD (“drusen”), which is an earlier stage of AMD and is characterized by yellow deposits under the retina which may grow larger and more numerous over time and potentially lead to GA and loss of vision; and

•autosomal recessive Stargardt disease (“STGD1”), which is an orphan inherited condition characterized by progressive damage to the central portion of the retina (the “macula”) and other retinal tissue, leading to loss of vision.

In July 2021, the Company completed patient enrollment for GATHER2, its Phase 3 clinical trial evaluating the safety and efficacy of Zimura for the treatment of GA secondary to AMD. The Company also received a written agreement from the U.S. Food and Drug Administration (“FDA”) under a Special Protocol Assessment for the overall design of GATHER2. The Company expects top-line data from the GATHER2 trial to become available during the second half of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis.

The Company plans to begin clinical development of Zimura for drusen in 2022. The Company is developing IC-500 for GA secondary to AMD and potentially other age-related retinal diseases.

The Company's gene therapy portfolio consists of two product candidates in preclinical development (IC-200 and IC-100) and several ongoing 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:

•autosomal recessive bestrophinopathy (“ARB”), which is characterized by loss of central vision and abnormal changes in electrophysiology, and other IRDs associated with mutations in the BEST1 gene;

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

•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.

The Company is developing IC-200 for ARB and other BEST1-related IRDs, and IC-100 for RHO-adRP.