IVERIC bio, Inc.
Q4 2020 Earnings Call Transcript

Published: 3 Mar 2021

[Operator:]

Good day, and welcome to the IVERIC bio Fourth Quarter and Year-End 2020 Results Conference Call. Today's conference is being recorded. At this time, I'd like to turn the conference over to Kathy Galante of Investor Relations. Please go ahead.

[Kathy Galante:]

Good morning, and welcome to IVERIC bio's conference call. Representing IVERIC bio today are Mr. Glenn Sblendorio, Chief Executive Officer and President; Dr. David Guyer, Executive Chairman; Mr. David Carroll, Chief Financial Officer; Dr. Pravin Dugel, Chief Strategy and Business Officer; Dr. Abraham Scaria, Chief Scientific Officer; and Mr. Keith Westby, Chief Operating Officer.

[Glenn Sblendorio:]

Thanks, Kathy, and good morning, everyone, and thank you for joining us for our fourth quarter and year-end conference call. I hope you and your families are well as we get closer to turning the corner on this COVID-19 pandemic. We start 2021 with significant momentum as we are excited to share that patient enrollment and retention for GATHER2, our second Phase 3 clinical trial for Zimura for the treatment of geographic atrophy, or GA, secondary to age-related macular degeneration, or AMD, is progressing well, with enrollment trending ahead of schedule. We are accelerating our timeline for completing enrollment in GATHER2 to the third quarter of 2021. If the 12-month results from GATHER2 are positive, we plan to file applications with the results from GATHER1, our first Phase 3 clinical trial for Zimura in GA, and GATHER2 with the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval of Zimura for GA. We are extremely encouraged by the progress of enrollment in GATHER2. We believe patient retention is an important -- patient retention is as important as patient recruitment and are encouraged by the early impact of the programs we have put in place to retain patients in GATHER2. Since the initiation of GATHER2 in June 2020, our team has been proactive and creative, have put in place multiple initiatives to tackle the many challenges that the COVID-19 pandemic has brought to conducting clinical trials. These initiatives are designed to reduce the risk and exposure of COVID to patients and the staff treating them. Keith will review the details of our programs in more detail in a few moments. 2020 was a transformative year for IVERIC bio. Please let me recap some of the highlights from 2020 that we're most excited about. First, we reported positive 18-month results from GATHER1. These data support our previously announced 12-month data from this trial, at which time point Zimura met the prespecified primary efficacy endpoint with statistical significance. Importantly, we believe GATHER1 is currently the only completed Phase 3 clinical trial showing suppression of GA growth with continuous treatment effect over time.

[Keith Westby:]

Thank you, Glenn, and good morning, everyone. We are extremely pleased to accelerate our timeline for completing enrollment in GATHER2 to the third quarter of 2021. We are in an unusually fortunate position of having already published a positive Phase 3 study, GATHER1, in a major peer review journal. We believe this is our most impactful recruitment tool with investigators in GATHER2. Further, the early and continuous treatment effect demonstrated in the GATHER1 trial, along with Zimura's well-tolerated safety profile that was maintained throughout the 18-month trial, are key motivators for patient retention in the GATHER2 trial. We continue to work with our investigators to provide a safe environment for patients which we believe increases the patient's comfort and confidence to participate in the GATHER2 clinical trial during the COVID pandemic. We implemented a number of initiatives to reduce the risk and exposure to COVID for our patients and the staff treating them. We are committed to continuing patient enrollment and retention aggressively in the GATHER2 clinical trial while prioritizing patient safety. I would like to outline some of the steps our clinical operations team has taken to drive recruitment and retain patients throughout the trial. We increased the number of clinical trial sites participating in GATHER2 as compared to GATHER1. We are providing private transportation for patients to and from their scheduled office visits. Sites are set up for social distancing, and we are providing PPE to patients and site personnel. As Glenn mentioned earlier, retention is as important as patient recruitment. We are proactively monitoring visit scheduling and patient follow-through to ensure scheduled treatments are maintained. We also continue to monitor the COVID-19 situation closely and explore additional ways we can support our patients and clinical trial sites. In total, we are planning to enroll approximately 400 patients in the GATHER2 clinical trial. Patients are randomized one-to-one into 2 cohorts

[Pravin Dugel:]

Thank you, Keith. Thank you all for joining the call this morning. I hope that you are all well. Looking ahead, we plan on building upon our position in GA by exploring the potential development of Zimura and IC-500 for other forms and stages of AMD. We expect that our strategy will involve both C5 and HtrA1 inhibitions in a complementary fashion. Let me provide some further details.

[David Carroll:]

Thank you, Pravin, and good morning, everyone. I'd like to highlight a few items from our press release of this morning and provide some guidance on our expected year-end cash balance and our expected cash runway. For the quarter, our net loss totaled $25.4 million or $0.27 per share compared to a net loss of $17.5 million or $0.39 per share for Q4 2019. This increase in net loss was driven primarily by an increase in R&D expenses associated with our Zimura clinical programs and manufacturing activities, our gene therapy programs and the progression of our IC-500 program. During the quarter, we continued our startup and recruitment activities for GATHER2 and continued our manufacturing scale up. Our net loss for 2020 totaled $84.5 million, $1.14 per share, compared to a net loss of $58.9 million or $1.39 per share for 2019, again, primarily due to an increase in R&D expenses. Turning to our expected year-end cash balance and cash runway. We estimate our year-end cash balance will range between $130 million and $140 million. We also 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 Archemix or any commercialization expenses for Zimura. These estimates are based on our current business plan, which includes the continuation of our ongoing clinical development programs for Zimura, the progression of our IC-100 and 200 programs into the clinic and the advancement of our IC-500 development program. These estimates also assume that we will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially setting Zimura in any other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies, the 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.

[Glenn Sblendorio:]

Thank you, Dave. 2021 will be another important year for IVERIC bio. It's all about continued execution to finalize enrollment in our GATHER2 trial in Q3 and, also importantly, as we talked about a lot today, to retain these patients in the trial. So thanks for your time this morning and your continued support. We'll now turn the call over to the operator so that we can open up the line for any questions. Operator?

[Operator:]

. We'll go first to Tiago Fauth with Credit Suisse.

[Tiago Fauth:]

So I just have one on Zimura. So as we're thinking about the potential safety differentiation relative to other complement approaches, can you help us contextualize the CNV rates seen in GATHER1 and potential baseline characteristics of the population enrolled that might have influenced that somehow? And perhaps how that could look like in GATHER2? Also wondering if there are any relevant protocol or methodology changes in how investigators report the events or how the event is confirmed that could also result in differences between GATHER1 and GATHER2 on the safety as we're talking about CNV rates?

[Glenn Sblendorio:]

Tiago, thanks for the questions. Pravin, I think that's one for you.

[Pravin Dugel:]

Sure. Thank you, Glenn. And Tiago, thank you for the question. A lot of questions within that, but a very important question. But let's just talk about the fellow CNVM eye first. The first thing I'd like to share with you is why we excluded patients with CNVM in the fellow eye, it was entirely due to concern about compliance and protection of the integrity of the data. Such a patient would have likely had to return to our clinic multiple times for more than a month -- more than a year -- multiple times a month for more than a year. And we thought that was unreasonable, especially in elderly patients. We wanted to minimize the patient dropout and protect the integrity of the data, and that's the reason that we excluded patients with CNVM in the fellow eye. In regards to conversion to wet macular degeneration, we are starting to learn about the risk factors. There are many such risk factors, we know that. And having a CNVM in the fellow eye may be one of many risk factors. However, having a geographic atrophy that has a higher rate of metabolism because it grows faster, and recall that we recruited patients with extrafoveal geographic atrophy, the fastest-growing geographic atrophy, may also be theoretically a greater risk factor. So it's important to recognize, as you well know, the limitations of comparing results across trials when the patient populations are different, but it's also important to remember the magnitude of difference. For instance, the ratio of conversion between drug and sham in GATHER1 was about 4 times. However, with C3 inhibition, there are reported rates of 17 times. So comparing the magnitude is also important. So as we've mentioned many times before, we believe that there's solid science to support the beneficial efficacy and safety profiles that we have demonstrated with C5 inhibition to-date. Now your question regarding GATHER1 and GATHER2, the inclusion criteria are exactly the same. We are also studying patients with extrafoveal geographic atrophy. The only difference is in the exclusion criteria, as you recall, is that we are continuing to follow those few patients who developed a neovascular membrane for the entirety of the study. Recall that in GATHER1, the Duke Reading Center was not certain whether those patients could have an adequate measurement of the geographic atrophy growth. And then looking back at the GATHER1 study, they're confident that those patients can be accurately measured, so we will continue to follow those patients for the entirety of the study. And hopefully, Tiago that answers your question, but thank you for the question.

[Operator:]

We'll go next to Stacy Ku with Cowen & Company.

[Stacy Ku:]

Great to hear about the enrollment and retention progress. I have just one. We're wondering if there's any reason that Zimura can't be formulated in an extended-release formulation. We've been seeing a number of different extension release technologies for wet AMD, DME. I'm just curious about any progress here.

[Glenn Sblendorio:]

Thank you for that question. Keith, do you want to take that question?

[Keith Westby:]

Sure. Happy to do that. Thank you. Great question. To answer your question, no, there's no reason why we actually think that the properties of Zimura, which is an aptamer, chemically synthesized aptamer, lends itself quite well to those type of technologies. And of course, we are always looking at those types of technologies in the background.

[Glenn Sblendorio:]

And I'll just add that it's early days. Our focus is on getting the trial completed and hopefully being able to file with the regulatory agencies for the approval of this product that's greatly needed by patients. Clearly, extended release is a key part of life cycle. I think we have mentioned that life cycle is in the plan for Zimura. We're looking at a number of things. But more to come on that in the future. But thank you for the question.

[Operator:]

We'll go next to David Nierengarten with Wedbush Securities.

[David Nierengarten:]

Maybe just a quick question from me. Maybe I missed it earlier, but do you attribute the enrollment, the positive trends in enrollment, is it purely enrollment patient recruitment or how is the dropout rate looking compared to your expectations? In other words, is the dropout rate better than what you had anticipated? And then on IC-100, again, maybe I missed this, but that's a little bit of a pushout on the IND filing. Is it manufacturing-related? Or are you wrapping up preclinical data assays? Or kind of what's the cause there?

[Glenn Sblendorio:]

Yes. David, it's Glenn. Thank you for the 2 questions. I'll take the first one, and I'll ask Keith to take the second one. As we said on the call, very happy with the enrollment in the trial. It's trending ahead of schedule. Early days. So as it relates to dropouts, that's not something we had talked about. But I think the important point is that we also spoke about retention, and that retention was just important. And Keith covered a number of the initiatives there. So we have thought about retention right from the beginning. If you remember, we had deferred the start of this trial, which was a difficult decision at the beginning of COVID and tried to pick the right point. So obviously, our efforts around retention is to minimize missed visits, et cetera. But those type of metrics, it's early to report those. I think you should take away from this that at this point that we're happy with the progress of the trial. Keith, do you want to take the second question on IC-100?

[Keith Westby:]

Sure. Thanks, Glenn. So we had provided general guidelines initially as we complete our preclinical development for our first gene therapy asset to hopefully enter the clinic. We just want to be sure that we're bringing forward a plan that has a high probability of success in an area with significant unmet medical need. So we remain very excited about the collaboration with the University of Penn and University of Florida. And we'll continue to develop the unique retinal asset that's capable of simultaneously knocking down and replacing the toxic mutant gene. So as we discussed, we would be wrapping up the -- we completed the non-clinical studies, and we're in the process of site selection and starting up activities. We'll plan to meet with the regulatory authorities to discuss the selected doses for this first-in-human trial before we file the IND. And then that's why the IND is now in the second half as opposed to the first half.

[Glenn Sblendorio:]

David, I think this will be our first clinical trial in gene therapy. So we want to be sure that we're taking all the right steps and doing all the right things. Importantly, as Keith said, the preclinical tox work done. I do want to mention and congratulate our operations team and the work they've done with our outside with CMO on manufacturing. So the materials are there. But I think since this will be our first gene therapy program, we want to be sure that we're taking all the right steps.

[Operator:]

At this time, there are no further questions. I will turn the call back to Glenn for closing remarks.

[Glenn Sblendorio:]

Operator, thank you for hosting the call. And again, thank you to everybody for listening today. We look forward to continued dialogue in the second quarter. Thank you. Goodbye.

[Operator:]

This does conclude today's conference. We thank you for your participation.