On August 1, 2019 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the second quarter ended June 30, 2019 and provided a business update (Press release, Ophthotech, AUG 1, 2019, View Source [SID1234537986]).

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"We are pleased with the Company’s progress as we achieved several milestones to advance and expand our gene therapy pipeline for orphan inherited retinal diseases, or IRDs," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "We entered into exclusive global license agreements for rights to gene therapy product candidates for the treatment of BEST1-related IRDs and Leber congenital amaurosis type 10, or LCA10. We are encouraged by new research data from our miniCEP290 collaboration that support our plans to move this program forward in LCA10, and we expanded our gene therapy portfolio with the addition of a minigene research program for USH2A related IRDs, including Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. Manufacturing activities for IC-100 and IC-200, our two lead gene therapy product candidates, are currently ongoing using state-of-the-art manufacturing capabilities of Paragon Gene Therapy, part of Catalent Biologics."

Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio added, "With the addition of the USH2A program to our portfolio, IVERIC bio is seeking to treat vision loss associated with what are reportedly the most common form of autosomal dominant retinitis pigmentosa (rhodopsin-mediated) and the most common form of autosomal recessive retinitis pigmentosa (USH2A-related). Retinitis pigmentosa is the most common orphan IRD. We look forward to presenting our gene therapy pipeline and discussing our programs in greater detail with highly-recognized gene therapy scientists and key opinion leaders in retinal diseases at our upcoming Gene Therapy R&D Investor Day."

Second Quarter 2019 and Recent Highlights

Orphan IRD Gene Therapy Programs

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Natural history studies and IND-enabling activities for IC-100 are ongoing. The Company expects to initiate a Phase 1/2 clinical trial for IC-100 in the second half of 2020.

IC-200: BEST1-Related IRDs
In April 2019, the Company entered into an exclusive global license agreement with the University of Pennsylvania and the University of Florida Research Foundation for rights to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of BEST1-related IRDs, including Best vitelliform macular dystrophy, also known as Best disease. The Company expects to initiate a Phase 1/2 clinical trial for IC-200 in the first half of 2021.

miniCEP290: Leber Congenital Amaurosis Type 10 (LCA10)
Encouraging results from the Company’s collaboration with the University of Massachusetts Medical School (UMass Medical School) in its miniCEP290 program led the Company to exercise its option and, in July 2019, the Company entered into an exclusive global license agreement with the University of Massachusetts for rights to develop and commercialize mutation independent novel AAV minigene therapy product candidates for the treatment of LCA10, which is due to mutations in the CEP290 gene and is the most common type of LCA.

miniUSH2A: USH2A-Related IRDs Including Usher Syndrome Type 2A and USH2A-Associated Nonsyndromatic Autosomal Recessive Retinitis Pigmentosa
In July 2019, the Company entered into a sponsored research agreement with UMass Medical School and an exclusive option agreement with the University of Massachusetts for rights to develop and commercialize novel AAV gene therapy product candidates utilizing a mutation independent minigene therapy approach for the treatment of USH2A-related IRDs. This group of orphan IRDs include Usher syndrome Type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa.

Gene Therapy Manufacturing
In June 2019, the Company announced that it had entered into a strategic manufacturing relationship with Paragon Gene Therapy, part of Catalent Biologics. This agreement is for production and manufacturing of AAV vectors for pre-clinical activities and planned Phase 1/2 clinical trials for IC-100 and IC-200.

Gene Therapy R&D Investor Day Scheduled
The Company will host its first "Gene Therapy R&D Investor Day" in New York, NY on Friday, September 13, 2019. The agenda includes updates from IVERIC bio’s management on its gene therapy portfolio in orphan IRDs, and scientific discussions from gene therapy scientists and key opinion leaders in orphan inherited retinal diseases including;

Guangping Gao, PhD, University of Massachusetts Medical School

William A. Beltran, DVM, PhD, University of Pennsylvania

Charles A. Gersbach, Duke University

Hemant Khanna, PhD, University of Massachusetts Medical School

Gustavo D. Aguirre, VMD, PhD, University of Pennsylvania School of Veterinary Medicine

Andreas K. Lauer, MD, Oregon Health & Science University

Bart P. Leroy, MD, PhD, Ghent University and Children’s Hospital of Philadelphia

Alfred S. Lewin, PhD, University of Florida

Therapeutic Programs
The Company expects initial top-line data for its ongoing Phase 2b clinical trial of Zimura (avacincaptad pegol), a C5 complement inhibitor, for the treatment of geographic atrophy secondary to dry age-related macular degeneration to be available in the fourth quarter of 2019. The Company expects initial top-line data for its Phase 2b clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease to be available in the second half of 2020.

Second Quarter 2019 Financial Results

Operational Update
As of June 30, 2019, the Company had $106.9 million in cash and cash equivalents. After taking into account the Company’s recent in-licensing transaction for its miniCEP290 program and sponsored research agreement for its miniUSH2A program, the Company reaffirms its estimate that year-end 2019 cash and cash equivalents will range between $80 million and $85 million. This estimate is based on its current 2019 business plan, including the continuation of its current research and development programs. This estimate does not reflect any additional expenditures, including associated development

costs, in the event the Company in-licenses or acquires any new product candidates or commences any new sponsored research programs.

R&D Expenses: Research and development expenses were $10.0 million for the quarter ended June 30, 2019, compared to $8.5 million for the same period in 2018. For the six months ended June 30, 2019, research and development expenses were $17.7 million compared to $16.2 million for the same period in 2018. Research and development expenses increased primarily due to increases in costs associated with the Company’s gene therapy programs and HtrA1 inhibitor program, offset by decreases in costs associated with the Company’s Zimura programs, personnel expenses and professional and consulting fees.

G&A Expenses: General and administrative expenses were $5.2 million for the quarter ended June 30, 2019, compared to $6.3 million for the same period in 2018. For the six months ended June 30, 2019, general and administrative expenses were $10.7 million compared to $12.0 million for the same period in 2018. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure.

Net Income: The Company reported a net loss for the quarter ended June 30, 2019 of $14.4 million, or ($0.35) per diluted share, compared to a net loss of $13.2 million, or ($0.37) per diluted share, for the same period in 2018. For the six months ended June 30, 2019, the Company reported a net loss of $26.9 million or ($0.65) per diluted share, compared to a net loss of $26.3 million or ($0.73) for the same period in 2018.

Conference Call/Web Cast Information
IVERIC bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for August 1, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-458-4121 (USA) or 323-794-2597 (International), passcode 3528260. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 3528260.