ISEE (2019 - Q2)

Good day, and welcome to the IVERIC bio Second Quarter 2019 Results Conference Call. Today's conference is being recorded. And now at this time, I would like to turn the conference over to Kathy Galante. Please go ahead. Kathy Ga

Good morning, and welcome to IVERIC bio's Second Quarter 2019 Earnings Call. Representing IVERIC bio today are: Mr. Glenn Sblendorio, Chief Executive Officer and President; Dr. David Guyer, Executive Chairman; Dr. Kourous Rezaei, Chief Medical Officer; Mr. David Carroll, Chief Financial Officer; Mr. Keith Westby, Chief Operating Officer; and Mr. Vishal Kapoor, Chief Business Officer. I would like to remind you that today, we will be making statements relating to IVERIC bio's future expectations regarding operational, financial and research and development matters, including statements regarding the implementation of our strategic plan, including our focus on developing gene therapy for orphan inherited retinal diseases; our projected use of cash and cash balances; the timing, progress and results of clinical trials and other research and development activities; the potential utility of our product candidates; and the potential for our business development strategies, including our collaborative gene therapy-sponsored research programs and other potential in-license or acquisition opportunities. These statements constitute forward-looking statements for the purpose of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. These statements cover many events and matters that are subject to various risks that could cause actual results to differ materially from those expressed in any forward-looking statement, including risks relating to the initiation and the progress of research and development programs and clinical trials; availability of data from these programs; reliance on university collaborators and other third parties; establishment of manufacturing capabilities; expectations for regulatory matters; need for additional financing and negotiation; and consummation of in-license and/or acquisition transactions. I refer you to our SEC filings and in particular to the Risk Factors section in our quarterly report on Form 10-Q filed on May 8, 2019, for a detailed description of the risk factors affecting our business. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we disclaim any obligation to do so as required by law. I would now like to turn the call over to Glenn.

Thanks, Kathy, and good morning, everybody. We appreciate you joining our call this morning. First, we're excited to be hosting our first Gene Therapy R&D Investor Day on September 13. We hope you all will be able to join us at this event, where we have several highly recognized gene therapy scientists and key opinion leaders in retinal diseases. And we'll also provide an update on the state of inherited retinal diseases in our gene therapy programs. Kourous will talk more about this in a few moments. We're pleased with the company's progress as we continue to advance and expand our orphan inherited retinal diseases, or IRD, gene therapy pipeline. We're on track to bring our portfolio of IRD programs into the clinic, starting in the second half of 2020 with our lead candidate, IC-100, for rhodopsin-mediated autosomal-dominant retinitis pigmentosa. We also 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 gene therapy products for candidates for the treatment of BEST1-related IRDs, including Best vitelliform macular dystrophy, also known as Best disease. We're also encouraged by new research data from our minigene CEP290 collaboration for Leber Congenital Amaurosis type 10, or LCA10, with the University of Massachusetts Medical School, which we believe supports our plans to move forward with this program. We just announced that we expanded our gene therapy portfolio with the addition of minigene sponsored research program for USH2A-related IRDs, including Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. And we have also engaged Paragon to do our gene therapy manufacturing. Paragon, now a part of Catalent Biologics. And they will manufacture both our IC-100 and IC-200, our 2 lead gene therapy candidates, for preclinical and planned Phase I/II supply. We continue to make progress with our minigene programs. Last week, we announced that we exercised our option and entered into an exclusive global license agreement with the University of Massachusetts for rights to develop and commercialize mutation-independent, novel, adenosine-associated virus minigene therapy product candidates for the treatment of LCA10 due to CEP290 gene mutations. LCA10 is the most common of all LCAs. Further, we announced that we are expanding our gene therapy portfolio by entering 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 mutation-independent, novel, adenosine-associated virus minigene therapy products for the treatment of USH2A-related IRDs. This is a group of orphan IRDs that include Usher syndrome type 2 and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. A cornerstone component for advancing our gene therapy pipeline is to have a solid GMP manufacturing strategy in place. In June, we announced that we have entered into a strategic manufacturing relationship with Paragon Gene Therapy, now a part of Catalent Biologics. This agreement is for the manufacture of AAV vectors for preclinical and planned Phase I/II clinical supply of IC-100 and IC-200. Our relationship with Paragon has given us access to state-of-the-art manufacturing capabilities that we believe will pave the way to enter the clinic with IC-100 in the second half of 2020 and with IC-200 in the first half of 2021. The execution of our therapeutic programs continues to stay on track as we look forward to receiving and reporting initial top line data from our ongoing randomized, double-masked, sham-controlled Phase IIb clinical trial assessing the safety and efficacy of Zimura monotherapy in patients with geographic atrophy secondary to dry AMD in the fourth quarter of this year. As we stated in the past, if this data from this trial is positive, we plan to seek partnership opportunities for further development of Zimura. I'd now like to turn the call over to Kourous to provide more details about the Gene Therapy R&D Investor Day and to review our programs in more detail. Kourous?

Thanks, Glenn, and good morning, everyone. As Glenn mentioned, I would like to remind everyone that we are hosting our first Gene Therapy R&D Investor Day on Friday, September 13 in New York from 8:30 a.m. to 11:30 a.m. We will provide an update and review our gene therapy portfolio in more detail. We will be joined by highly recognized gene therapy scientists and key opinion leaders in retinal diseases, who will provide their insights regarding our gene therapy programs and their potential for the treatment of orphan inherited retinal diseases. We are excited to share with you a list of our speakers, which include; Dr. Guangping Gao, President of the American Society of Gene & Cell Therapy, Director of Horae Gene Therapy Center at UMass; Dr. William Beltran from UPenn; Dr. Charles A. Gersbach from Duke University; Dr. Hemant Khanna from UMass; Dr. Gustavo Aguirre from UPenn; Dr. Andreas Lauer, Chair, Department of Ophthalmology at Casey Eye Institute; Dr. Bart P. Leroy, Chair and Head of Department of Ophthalmology at the Ghent University and Director of the Retinal Degenerations Clinic, Children's Hospital of Philadelphia; and Dr. Alfred Lewin from University of Florida. We continue to cultivate, strengthen and expand our scientific relationship with the investigators at UMass and its Horae Gene Therapy Center, the UPenn and the University of Florida. We have the privilege of collaborating with leading scientists and pioneers in retinal gene therapy, and we're delighted with the progress we have made in our programs. You will have the opportunity to meet many of them during our Gene Therapy R&D Investor Day. The preclinical development program for IC-100 is on track, and we continue to progress with IND-enabling activity. In collaboration with Penn, we are conducting preclinical study and a natural history study in rhodopsin-mediated adRP patients. As Glenn mentioned earlier, we have engaged Paragon as our manufacturer for preclinical and planned Phase I/II clinical supply of GMP-grade materials for IC-100. Process development and manufacturing activities are currently on track and ongoing. Our clinical strategy is to select a few leading clinical trial sites in the field of retinal degenerative diseases while also have experienced retinal specialists in ocular gene therapy surgery. We have identified our potential clinical trial sites and continue to advance IC-100 towards an IND filing and to initiate a Phase I/II clinical trial during the second half of 2020. Currently, there are no FDA or EMA-approved treatment options available for the treatment of patients with rhodopsin-mediated adRP. Regarding IC-200. In collaboration with Penn, we are conducting additional preclinical study and natural history study in patients with BEST1-related IRD. We have commenced process development, manufacturing and other IND-enabling activities. Paragon is also our manufacturer for our preclinical and planned Phase I/II clinical supply of GMP-grade material for IC-200. We expect to initiate a Phase I/II clinical trial for IC-200 in the first half of 2021. Currently, there are no FDA or EMA-approved treatment options available for patients with BEST1-related IRDs. Although AAV vectors are the leading vector of choice for retinal gene therapy, one of the main limitations is their transgene packaging capacity. Employing a minigene strategy is an attractive option to overcome this limitation and deliver a smaller but functional portion of the larger transgene packaging in standard-sized AAV delivery vector. A primary research in retinal minigene therapy is headed by Drs. Khanna and Gao at the Horae Gene Therapy Center at UMass. And we are excited to collaborate with them. As Glenn pointed out, we have expanded our pipeline and are currently assessing this mutation-independent technology in LCA10, autosomal recessive Stargardt disease and most recently in USH2A-related inherited retinal diseases, including Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive RP. We believe that our new encouraging results from the miniCEP290 program supports the potential of minigene AAV vectors for the treatment of LCA10. We continue to optimize the construct with the goal of identifying a lead product candidate, which would be advanced through preclinical development and IND-enabling activities. We plan to update you further during our Gene Therapy R&D Investor Day. Our miniABCA4 minigene research program for autosomal recessive Stargardt disease is also currently ongoing, and we expect to receive results in 2020. USH2A-related inherited retinal diseases are a global orphan IRD associated with mutations in USH2A gene. These include retinitis pigmentosa associated with Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive RP. Usher syndrome type 2A is an autosomal recessive genetic condition characterized by hearing loss from birth and progressive vision loss due to retinitis pigmentosa that begins in adolescence or adulthood. USH2A-associated nonsyndromatic autosomal recessive RP is a genetic condition that manifests as vision loss without associated hearing loss. We are currently focused on the developing a deliverable mutation-independent minigene treatment option for the vision loss associated with USH2A mutation. There are currently no FDA or EMA-approved therapies to treat LCA10, autosomal recessive Stargardt disease, Usher syndrome type 2A or USH2A-associated nonsyndromatic autosomal recessive RP. 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 inherited retinal disease. We look forward to updating investors as our portfolio continues to progress. Thank you for your time. I will now turn the call over to Dave Carroll. Dave?

Thank you, Kourous, and good morning, everyone. I'd like to highlight a few items from our press release of this morning and also reaffirm our year-end cash guidance. For the quarter, our net loss totaled $14.4 million or $0.35 per share compared to a net loss of $13.2 million or $0.37 per share for Q2 2018 as Q2 2018 reflected $1 million tax benefit associated with the settlement of a local tax audit. Year-to-date, our net loss totaled $26.9 million or $0.65 per share compared to a net loss of $26.3 million or $0.73 per share for 2018. This increase in net loss is driven again primarily by the aforementioned tax benefit associated with our settlement of a local tax audit. Turning to our expected year-end cash balance. Our cash balance at June 30 was approximately $107 million, a $10 million decrease from Q1. The company reaffirms its estimate that year-end cash will range between $80 million and $85 million based on our current 2019 business plan, which includes the expansion of our gene therapy programs, including our recent UMass agreements, expansion of our HtrA1 development program and the continuation of our clinical development programs for Zimura. Of course, these estimates do not reflect any additional expenditures, including any associated development costs in the event that the company in-licenses or acquires any new product candidates or technologies or commences any new research programs. I now turn the call back over to Glenn. Thank you for your time.

Well, thanks, Dave. In conclusion, I want to reiterate our commitment to progressing our company to our goal to potentially become a leader in the development of transformative gene therapies for inherited retinal diseases. We believe we are well positioned to bring our gene therapy orphan IRD portfolio forward. We look forward to keeping you updated on the execution and progress of our company. We also hope to see you at our Gene Therapy R&D Day on September 30 -- 13. And if anybody has questions about that or would like an invitation, please contact Kathy with any questions that you may have about this event. I want to again thank you for joining our call today and would like to open the lines for questions.

Thank you again. Look forward to engaging everybody throughout the summer, and see you on September 13. Goodbye.

Ladies and gentlemen, this will conclude your conference for today. We do thank you for your participation, and you may now disconnect.