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spk03: Good morning and welcome to the Iverik BIO first quarter 2021 earnings conference call. All participants will be in listen only mode. Should you need assistance, please signal a conference specialist by pressing star then zero on your telephone keypad. After today's presentation, there will be an opportunity to ask questions. Please note this call is being recorded. I would now like to turn the conference over to Kathy Galante, Senior Vice President, Investor Relations. Please go ahead.
spk09: Good morning and welcome to Ibericbio's conference call. Representing Ibericbio today are Glenn Slandorio, Chief Executive Officer, Praveen Dougal, President, David Carroll, Chief Financial Officer, Davil Desai, Chief Development Officer, Abraham Skaria, Chief Scientific Officer, and Keith Westby, Chief Operating Officer. I would like to remind you that today we will be making statements relating to IbericBio's future expectations regarding operational, financial, and research and development matters, including statements regarding our expectations for patient enrollment and patient retention in GATHER-2, our second Phase III clinical trial evaluating Zamora for the treatment of geographic atrophy secondary to age-related macular degeneration, our expectations to use GATHER-1, our previously announced clinical trial of Zamora, for the treatment of GA secondary to AMD as a Phase III clinical trial, our development and regulatory strategy for Zamora, and our other product candidates, including our expectations for additional indications for which we may pursue the development of Zamora and IC500, our hypothesis regarding complement inhibition and H-1 inhibition as mechanism of actions for the treatment of GA and potentially other stages of AMD. Our projected use of cash and cash balances, the timing, progress, and results of clinical trials and other research and development activities and regulatory submissions. The potential utility and development potential of our product candidates and the potential for our business development strategy and our personnel, advisory committee members, and human capital resources. These statements constitute forward-looking statements for the purposes 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 related to the future progression of the COVID-19 pandemic, responsive measures to the pandemic, and their impact on our research and development programs. operations and financial position, initiation of the progress of research and development programs and clinical trials, including enrollment and retention in clinical trials, availability of data from these programs, reliance on contract development and manufacturing organizations, universities, collaborators, and other third parties, establishment of manufacturing capabilities, expectations for regulatory matters, developments from our competitors and the marketplace for our products, need for additional financing and negotiation and consummation of business development transactions and other risks. I refer you to our SEC filings and, in particular, to the risk factors included in our annual report on Form 10-K, filed on March 4, 2021, 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.
spk05: Thanks, Kathy, and good morning, everybody. I thank you for joining us for our first quarter conference call. The first quarter of 2021 has been very productive for us at IVARIC-BIO. I am pleased to share that we are excited to be executing and moving towards completing enrollment in the GATHER2 clinical trial, our second phase three clinical trial for Zamora, a novel complement C5 inhibitor for the treatment of geographic atrophy or GA, secondary to age-related macular degeneration. We expect to complete this trial in the third quarter of this year. To date, both recruitment and retention of patients in GATHER2 are exceeding our expectations. we are on track for initial top-line data from Gather 2 to be available approximately one year after the enrollment of the last patient in the Gather 2 clinical trial. Of course, we have to add some time for database closure and analysis of the initial top-line data. If the 12-month results from Gather 2 are positive, We've planned to file applications with the US FDA and the European Medicines Agency for marketing approval of Zamora for GA. I also wanted to mention some organizational changes that further strengthen our management team. I am thrilled to announce that Dr. Praveen Dougal has been promoted to president effective May 1. Praveen has played a critical role in our execution of strategy. He's been a key part of the executive management team leading the company's strategy, and I want to also congratulate Kathy Galante on her well-deserved promotion to Senior Vice President, Investor Relations. I look forward to continuing to work with both of them at this exciting time in our company. Finally, I want to acknowledge David Geyer, my friend, who will be stepping down from our board after 14 years for his leadership as co-founder and executive chairman. I sincerely thank David for his hard work and significant contributions to the company. We wish David well as he rejoins SB Health Investors as a venture partner. I would now like to turn the call over to Keith, who will review enrollment and retention for Gather 2 and update you on our gene therapy pipeline in orphan inherited retinal diseases. Following Keith, Praveen will discuss our strategy in AMD and the formation of our Gene Therapy Inherited Retinal Disease Scientific Advisory Committee. Keith?
spk07: Thank you, Glenn, and good morning, everyone. As Glenn mentioned, our main priority is to aggressively drive patient recruitment and retention in the GATHER2 clinical trial. The enthusiasm, resiliency, and dedication of patients physicians, and their staff are exceeding our expectations. With the reduction in the mean rate of GA growth over 12 months at 27.38%, a p-value of 0.0072 for the Zamora 2 milligram group as compared to the corresponding sham control group in the Gather 1 clinical trial, we believe many principal investigators are enthusiastic about enrolling patients for the Gather 2 clinical trial. leveraging the quality of the positive GATHER1 results to maximize both patient recruitment and retention for GATHER2. These positive 12-month data are further supported by positive 18-month efficacy results and a favorable safety profile that was maintained throughout the 18-month trial. Further, we believe that the early and continuous treatment effect demonstrated in GATHER1 is a key motivator for patient retention in GATHER2. In total, we are planning to enroll approximately 400 patients in the GATHER-2 clinical trial. We continue to be on track and look forward to completing enrollment in GATHER-2 in the third quarter of this year. Our Phase 2b screening clinical trial of Zimora for the treatment of autosomal recessive Stargardt disease, referred to as the STAR trial, is ongoing with the goal of enrolling approximately 120 patients. Results from this clinical trial are expected after the 12-month initial top-line data from GATHER2. There are currently no therapies approved for Stargardt disease in either the U.S. or the European Union. Turning to our gene therapy programs, we are excited about the progress in the development of our product candidate for the treatment of best one related inherited retinal diseases, or IRDs, IC200. We are completing a toxicology study in the naturally occurring canine model of best disease. As a reminder, we have data demonstrating long-term rescue in this model following a single subretinal injection. We are on track to release the recently manufactured GMP batch of IC200 in preparation for the planned IND filing and plan to move IC200 into a Phase 1-2 clinical trial in the second half of 2021. We are not aware of any ongoing competitive programs for BEST1-related IRDs and believe IC200 has the potential to be both first and best in class. Regarding IC100, our product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa, the company continues to evaluate the results from its preclinical toxicology studies. In our preclinical efficacy and toxicology study in a naturally occurring canine model of RhoADRP, efficacy was demonstrated at all three doses tested. We also tested the same three doses in a GLP toxicology study in nonhuman primates. Ocular inflammation on clinical exam was observed in the high dose group in canines and to varying degrees at different dosing levels tested in nonhuman primates. Due to the different findings in two different species, and our high commitment to the safety of our patients, we are planning to discuss the design of our planned first-in-human clinical trial with regulators prior to submitting an IMD. We now believe that IC100 will likely be delayed from entering into a Phase I-II clinical trial this year. We are thrilled about the progress of our mini-gene programs in collaboration with the University of Massachusetts Medical School. We are pleased to report that we have recently identified a lead construct for our Leber congenital amaurosis type 10 or LCA10 mini CEP290 program and currently considering development plans for this program. Also during the second quarter, we expect to obtain additional results from our Stargardt disease mini ABCA4 program, and we expect to obtain preliminary results from our USH2A-related IRD program during the second half of this year. We will continue to keep you updated on our mini-gene programs. Thank you for your time. I will now turn the call over to Praveen. Thank you, Keith.
spk08: Thank you all for joining the call this morning. I hope that you are all well. A key goal of ours is to expand and advance our footprint in multiple stages and types of AMD. We intend to execute a development strategy that will involve both Zemura and IC500 in a complementary fashion to impact multiple forms and stages of AMD. I would like to let everyone know that we are planning to host a DRY-AMD virtual symposium for investors and analysts on Friday, June 18th, from 10 a.m. to noon Eastern Time. The event will include presentations and discussions with retinal specialists and key opinion leaders on Zimora and IC500 and the DRY-AMD landscape. Guest speakers will include Dr. Frank Holtz, University of Bonn. Dr. Peter Kaiser, Cleveland Clinic Lerner College of Medicine Cole Eye Institute. Dr. Arshad Kanani, Sierra Eye Associates and University of Nevada, Reno. Dr. Anat Lohnstein, Sackler Faculty of Medicine at the Tel Aviv University and Tel Aviv Medical Center. Dr. Charles Wyckoff, Retina Consultants of Texas and Blanton Eye Institute Houston Methodist Hospital, and Dr. Trent Woodruff, the University of Queensland. We hope you will be able to join us at our virtual symposium on June 18th. Please reach out to Kathy if you have any questions. Turning to IC500, we announced in March 2021 that we revised our development plans for IC500 to include the investigation of multiple dosing schedules for our product candidates. In April 2021, we commenced our first preclinical tolerability study, and we're currently planning additional preclinical studies, including pharmacokinetic and target engagement studies. Formulation, optimization, and other manufacturing activities are also ongoing. we continue to remain excited about the development potential of IC500 and expect to submit an IND to the FDA for IC500 in GA secondary to AMD in the second half of 2022. As a reminder, we believe IC500 has the potential to be best in class as it inhibits HTRA1 both intra and extracellularly. As Keith mentioned, while IC100 is delayed, our IC200 and mini gene programs are progressing well. We are excited to announce today that in addition to our visionary steering and imaging advisory committees, the formation of an iveric bio gene therapy Inherited Retina Disease Scientific Advisory Committee, which brings together a renowned group of thought leaders who bring extensive clinical experience and a deep understanding of gene therapy to treat inherited retinal diseases. The advisory committee will work closely with senior management as we advance our gene therapy IRD programs. Advisory committee members include Dr. Elias Trabulsi, Cole Eye Institute, Cleveland Clinic Lerner College of Medicine. Dr. Andreas Lauer, KCI Institute, University of Oregon. Dr. Bart Lerwa, Ghent University Hospital and Children's Hospital of Philadelphia. Dr. Mark Panese, KCI Institute, University of Oregon. And Dr. Eleonora Ladd, Duke Reading Center, Duke University Medical Center. We remain committed to our mission statement to develop transformative therapies in retinal diseases. We look forward to keeping you updated on our progress in the months to come. Thank you for your time. I will now turn the call over to Dave.
spk06: Thank you, Praveen, and good morning, everyone. I'd like to highlight a few items from our press release of this morning and update our expected year-end cash balance and confirm our expected cash runway. For the quarter, our net loss totaled 26.8 million, or 29 cents per share, compared to a net loss of 15.1 million, or 28 cents per share, for Q1 2020. This increase in net loss was driven primarily by increases in both R&D and G&A expenses, and the Q1 2020 impact of a favorable settlement of a state corporate tax audit. R&D expenses increased due to the progression of our Zamora clinical programs and the progression of our preclinical gene therapy and IC500 programs. During the quarter, we continued our recruitment and retention activities for Gather 2 and continued our Zamora manufacturing scale-up activities. G&A expenses increased primarily due to an increase in legal costs associated with our ongoing litigations Turning to our expected year-end cash balance and cash runway, as Keith and Glenn have described, we are aggressively driving Gatitude recruitment and retention. We're also investing in the manufacturer's Zamora drug substance and drug product, including required starting materials, as we aim to scale up and validate the manufacturing process. As expected, both of these activities have impacted our Q1 cash burn. However, we expect our quarterly cash burn to decrease as the year progresses. We now expect our year-end cash balance will range between $125 and $135 million. Our long-term cash forecast is unchanged. We estimate that our cash will be sufficient to fund our capital expenditures and operating expenses as currently planned into 2024, excluding any potential approval or sales milestones payable to Archimix or any commercialization expenses for Zamora. These estimates are based on our current business plans. which includes the continuation of our ongoing clinical development programs for Zamora, the progression of our IC100 and 200 programs into the clinic, and the advancement of our IC500 development program. These estimates also assume that we will enroll approximately 400 patients in the GATHER-2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zamora in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of any new sponsored research programs or any associated development that we may pursue. I'll now turn the call back over to Glenn. Thank you for your time.
spk05: Well, thanks, Dave, and thank you, everyone, for your time this morning. and your continued support. I'll now ask the operator if he can open up the line for questions. Thank you.
spk03: We will now begin the question and answer session. To ask a question, you may press star then one on your telephone keypad. If you are using a speakerphone, please pick up your handset before pressing the keys. If At any time your question has been addressed and you would like to withdraw your question, please press star then two. At this time, we will pause momentarily to assemble our roster. The first question comes from Tiago Falk with Credit Suisse. Please go ahead.
spk00: Okay, thanks for taking the question. So just a quick confirmation here. So in addition to GADR1 data, is the 12-month GADR2 data sufficient to support the filing from a safety or exposure perspective, or do you need any additional follow-up there? And another follow-up on the broader competitive landscape in GA. So again, there's a lot of focus on C3 and C5 inhibition because there is clinical data for those approaches. But curious about your thoughts on longer-term potential of approaches like gene therapy targeting CD59 or recombinant complement factor H proteins and other emerging therapies out there. What are some pros and cons? Thank you.
spk05: Tiago, thank you. It's Glenn, and thank you for the question. So I'll take the first question on the GADL1 trial, and I'll ask Praveen to take the second question. Yes, we believe the combination of Gather 1 and Gather 2 will be sufficient for a regulatory filing. I think as we have said in the past, we've seen in our disclosures, we've had numerous conversations, informal conversations with the FDA that has given us comfort in our strategy with the two trials. So yes, we're comfortable. And as I said today, on completion of Gather 2, our intent is to analyze the top-level data, and if it's sufficient, we would file for regulatory approval.
spk08: Graveen? Thank you, Glenn, and good morning, Tiago, and thank you for the question. You know, what I would say is we're delighted that there are so many companies that are looking at the complement pathway because it validates the target. We remain quite convinced that our target has a great deal of scientific backing and scientific evidence. In fact, we feel that our results reflect what would be consistent with previously known science and previously published science. In terms of gene therapy, I think it's important to understand that there's a different challenge to gene therapy for chronic disease as opposed to an orphan disease. Gene therapy is very exciting, but again, the challenges remain for a chronic disease, which It may be more different than orphan disease, as we've seen. So we support all of these companies. We're delighted that there are more people that are in the complement pathway, as I say, because it validates the target. However, we remain convinced that science stands with our target selection in the inhibition of C5. Got it.
spk00: Understood.
spk04: All right. Thank you very much.
spk03: The next question comes from Stacy Ku with Cowan. Please go ahead.
spk02: Good morning. Thanks for taking my questions, and congratulations on the continued progress. I have a few. So first, wondering if you guys would be willing to provide any additional commentary on retention or enrollment. For instance, have you seen an even greater acceleration in the past few months, or should we just expect kind of a steady addition of patients? And then my second question is another competitive one. As we approach a pellucid data readout, can you remind us main differences in study design and specifically the reasoning behind the key inclusion criteria for extra foveal lesions? Thank you.
spk05: Well, Stacy, thanks for the compliment on execution. Thanks for the questions. So on the first retention, and updates on that in color, I'll ask Keith to do that, and on the second question, Praveen will take that.
spk07: Thanks, Glenn. Thanks, Nacy. So, yes, the enrollment was good from the onset, so it's been continued to be quite, we're quite happy with that, and I think there's a number of things that really drive that. One is the results from the Gather 1, which were published, the 12-month results, as well as the 18-month follow-on results from Gather 1. which really helped drive the enrollment and as well as the retention in Gather 2. So we're quite pleased with that and very much on track for the third quarter to complete that.
spk05: The only thing I'd add in terms of color, I think it goes back to a lot of what we said in the past on the preparation in this COVID environment. And Keith and his team work very closely, not only with the sites, but took into account also the impact potentially on patients and how do we keep them safe. So I think that early planning, and if you recall, we did delay the trial in early 2020 until we could better understand what was happening with the pandemic, and that was about a three-, four-month delay, and that allowed us really to take a step back, analyze what was happening with the docs, plan well, and I think we're seeing the fruits of that planning and the execution now. And as you know, we said at the end of the year that we did move up the completion date from, you know, the end of the year or closer to fourth quarter to the third quarter. So that may be a little bit more color for you at this point in time.
spk08: So, Praveen, I'll give you the second question. Yeah, thank you, Glenn. And, Stacy, thank you for your question. So what has been known for quite a long time, more than half a century, is that there's a very stereotypic way that geographic atrophy advances. And what it does is that it advances fairly rapidly in a circumferential fashion. And by rapidly, I mean in a matter of several months. And you can see this in the excellent studies that Genentech has published in regards to their Lampelizumab program. And then once it advances relatively rapidly circumferentially, Once the fovea is involved, the geographic atrophy tends to slow down once the fovea is involved. So the bottom line is that there are lots and lots of patients out there with excellent visual acuity. The visual acuity may be refractible to 20-20, but they're visually dysfunctional because of the geographic atrophy that they have, this extra foveal that may not allow them to function. They may not be able to see a straight line. If you're an architect or certainly an engineer, they may not be able to finish reading a spreadsheet or a sentence if you're an accountant or a lawyer. So there are lots of those patients out there. And the logic behind the selection of extra foveal geographic atrophy is not only to address this enormous unmet need, but also to be able to note that if we're able to slow down the fastest growing geographic atrophy, we've met a higher bar of scrutiny than slowing down a slower growing geographic atrophy. And finally, what I'd also say is that the ability to protect the fovea from being affected by geographic atrophy is terribly valuable. And we believe that we'll be able to demonstrate that as well. So for all of those reasons, important to understand why we selected this patient population. And also, as you've noticed, Stacey, important to note that our patient population is quite different than the Pellis patient population and should be regarded that way as well when you analyze the data. But thank you for your question.
spk04: Thank you.
spk03: The next question comes from David Nearingarden with Wedbush Securities. Please go ahead. Mr. Nierengarten, your line is open. Please go ahead with your question. Go ahead. Thanks.
spk01: The old mute button. I'll ask gene therapy questions today. On the two programs, are there any differences in manufacturing or anything else that would lead to differences in the animal outcomes or you know, obviously the gene being inserted is different. So essentially, do you think it's isolated to, you know, maybe inflammation caused by the gene product or, you know, again, are there differences in manufacturing or other things that might be leading to those cases of inflammation in the animals? Thanks.
spk05: David, thank you for your question. Keith? Sure.
spk07: Thanks, David. Very good question. So in terms of the manufacturing, you know, we feel confident that we did produce good quality material for these. And it's a similar manufacturing process for both. You know, I think looking at the data, we see the discrepancy in the two different species. So we just thought it would be prudent to take a step back and really analyze that data. and have some discussions with regulators prior to initiating the IND filing for the program. That's for IC100. For IC200, we are very much on track. We've completed the manufacturing of the GMP batch for the clinical trial, and that's in the process of being released now.
spk01: And could you remind us on the rhodopsin protein being delivered by the vector? Is it What species is that coming from in those studies? Is it the human rhodopsin protein or a different source?
spk07: It is. So that's a great question. It is the human rhodopsin that's being used for both the non-human primate studies as well as the canine studies, which is the same material we plan to move forward into a clinical trial. Got it.
spk04: Thank you.
spk03: There are no other questions. I would like to turn the conference back over to Glenn Splendorio for any closing remarks.
spk05: Thank you, operator, and we're very happy to have the opportunity to update you this morning on our continued execution and progress, and I'd like to wish everybody a good morning, and thanks for listening. Bye-bye.
spk03: The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.
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