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Edited Transcript of AGTC earnings conference call or presentation 12-Nov-19 9:30pm GMT

Q1 2020 Applied Genetic Technologies Corp Earnings Call

ALACHUA Dec 5, 2019 (Thomson StreetEvents) -- Edited Transcript of Applied Genetic Technologies Corp earnings conference call or presentation Tuesday, November 12, 2019 at 9:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Mark S. Shearman

Applied Genetic Technologies Corporation - Chief Scientific Officer

* Susan B. Washer

Applied Genetic Technologies Corporation - President, CEO & Director

* Theresa Heah;Chief Medical Officer

* William A. Sullivan

Applied Genetic Technologies Corporation - CFO

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Conference Call Participants

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* Joseph Pantginis

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

* Matthew W. Luchini

BMO Capital Markets Equity Research - Analyst

* Yanan Zhu

Wells Fargo Securities, LLC, Research Division - Associate Analyst

* Yun Zhong

Janney Montgomery Scott LLC, Research Division - Equity Research Analyst & Director of Biotechnology Research

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Presentation

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Operator [1]

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Good afternoon and welcome to the AGTC First Quarter Fiscal Year 2020 Financial Results Conference Call. Today's call is being recorded.

Before we get started I'd like to remind everyone that during this conference call, AGTC may make forward-looking statements, including statements about the company's financial results, financial guidance, its future business strategies and operations and its product development and regulatory progress. Actual results could differ materially from those discussed in these forward-looking statements due to a number of important factors, including uncertainty inherent in the clinical development and regulatory process and other risks described in the Risk Factors section of AGTC's annual report on Form 10-K for the fiscal year ended June 30, 2019.

For introductions and opening remarks, I'd like to turn the call over to Sue Washer, Chief Executive Officer of AGTC. Please go ahead.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [2]

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Good afternoon, and thank you all for joining us today. With me on today's call is Bill Sullivan, our Chief Financial Officer; Mark Shearman, our Chief Scientific Officer; and Theresa Heah, our Chief Medical Officer.

The first quarter of fiscal year 2020 was a significant one for AGTC and the patients we seek to serve. In September, we reported top line 6-month XLRP dose escalation data as well as preliminary 3-month data from the XLRP dose expansion cohort, showing stability of visual function in the periphery and improvement of visual function centrally. We also provided an early look at data indicating biological activity in both of our achromatopsia trial. While Theresa will provide a brief recap of these data shortly, the bottom line is that data from all 3 trials are compelling.

For XLRP we plan to continue our interaction with the FDA, with a goal of initiating a pivotal trial in the second half of 2020. We are excited to announce that we'll host an R&D Day on January 28, 2020 in New York City. This will be an opportunity for you to get an in-depth review of our clinical programs, interact with clinicians, surgeons and patients and also thoroughly analyze all the clinical data available by that date and our plans to move forward.

We hope to see all of you there and we'll provide more detail as the time approaches. We also expanded our preclinical pipeline, both through the advancement of multiple internal programs and with the establishment of a strategic collaboration with the San Diego-based otology focused company, Otonomy, to develop an AAV-based gene therapy for congenital hearing loss.

Additionally, last week we disclosed that Stargardt disease, an inherited form of macular degeneration that most often results from mutations in the ABCA4 gene is our second internal preclinical ophthalmology program, along with dry MD. Mark will discuss these exciting new opportunities in detail a bit later in the call, but I want to highlight that collectively, they gave AGTC one of the most diverse pipelines in the gene therapy space, and we believe that each of these programs provides the opportunity to significantly, help patients and create value for shareholders.

We intend to build on the substantial expertise we've gained in our clinical programs to date to advance these pipeline opportunities in a manner that will support IND filings and successful clinical trial design and development.

I will now turn the call over to Theresa, who will provide our clinical updates.

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Theresa Heah;Chief Medical Officer, [3]

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Thank you Sue. As Sue noted, reporting data from our ongoing trials in XLRP and achromatopsia was a key first quarter achievement. I will now summarize that data, provide an update on current trial status and highlight anticipated next steps and milestones. I will start with a review of the XLRP program.

In September 2019, we reported data from 17 patients, which continue to demonstrate a favorable safety profile for our XLRP product candidate. Efficacy analysis showed stable visual function in all 8 patients dosed peripherally at a 6-month time point and encouraging evidence of improved visual function from 9 centrally dosed patients at the 3-month time point, both in visual fields and visual acuity.

We believe the combination of the favorable safety profile, the stabilization of the peripheral vision and the improvement in central visual function will be highly meaningful to XLRP patients, who today, have no treatment options that address the underlying cause of their disease. We expect to report interim 6-month data from the dose expansion group of the XLRP study in early January 2020. A total of 24 patients have been dosed in the study, and we will present a comprehensive data set at our R&D Day that will include preliminary data on additional patients, dosed as part of our strategy to develop a comprehensive data set.

We plan to hold an annual Phase II meeting with the FDA in the second quarter of 2020. And our goal coming out of that meeting is to have final agreement with the FDA on pivotal trial design. This would position us to initiate that XLRP pivotal trial in the second half of 2020.

Now let me turn to our Phase I/II clinical trial in achromatopsia. In September 2019, we reported data from 14 patients, dosed across the B3 and A3 Phase I/II trials. Early data indicates that the achromatopsia candidates had biological activity and suggest that they may provide meaningful benefit to patients. One of three patients at a middle dose level in each trial, and 2 of 3 patients at a high dose level in the achromatopsia B3 trial have shown clinically meaningful improvements and light discomfort.

We expect to report interim 6 months data from the dose escalation groups of both achromatopsia studies in January 2020. We have now dosed a total of 29 patients across the 2 studies and will present a comprehensive data set from the 6-month dose escalation groups at our R&D Day, at which time, we'll also provide clarity on forward guidance of the programs.

As we noted during the September call, we believe the collective data from all 3 of our ongoing clinical trials is supportive of our product design, construct and manufacturing process and highlights our ability to design and execute clinical trials that provide clinically relevant data.

Let me close the overview of our clinical programs by noting that we continue to dose additional patients for the ongoing XLRP and achromatopsia Phase I/II trials in order to have the most robust data set possible for our inclusion in our Biologics License Application or BLA filing.

I will now turn the call over to Mark, who will review our research assets and preclinical program priorities.

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Mark S. Shearman, Applied Genetic Technologies Corporation - Chief Scientific Officer [4]

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Thank you, Theresa. First, I'm pleased to report that the optogenetics program conducted by our partner, Bionic Sight continue to advance. Site approval has been obtained and all information has been supplied to the FDA such that Bionic Sight can initiative the Phase I/II trial once the IND is active. During the September call we announced the expansion of our preclinical portfolio to include additional programs in ophthalmology and central nervous system or CNS disorders.

In the first quarter, we advanced our strategy to establish a diverse pipeline in the gene therapy arena with the initiation of a strategic collaboration with Otonomy. Under this collaboration, AGTC and Otonomy, which is a leader in developing innovative therapies for Otology, will co-develop and co-commercialize an AAV-based gene therapy to restore hearing in patients with sensorineural hearing loss caused by mutation in the gap junction protein beta 2 gene or GJB2, the most common cause of congenital hearing loss. Mutations in GJB2 attain for approximately 30% of all cases of genetic hearing loss. Patients with this mutation can have severe to profound deafness in both ears that is identified in screening tests routinely performed in newborns. Gene therapy holds great promise to restore functional and lasting hearing in these cases, with early intervention providing significant benefits in the development of language skills, socialization and overall quality of life of patients and their families. Under the collaboration agreement, the parties will equally share the program costs and proceeds and can include additional genetic hearing loss targets in the future.

Last week, we announced Stargardt disease as our second internal preclinical ophthalmology program. Stargardt disease is an inherited form of macular degeneration that most often results from mutations in the ABCA4 gene and occurs in 1 of 8,000 to 10,000 individuals. Loss of functional ABCA4 protein leads to the accumulation of toxic substrates in photoreceptors, resulting in photoreceptor death and therefore, progressive vision loss.

Consistent with our approach to developing AAV-based gene therapy to XLRP and achromatopsia, the goal of our preclinical Stargardt disease program is to deliver and express a functional copy of the ABCA4 gene in affected retinal cells. A key challenge to developing an AAV-based gene therapy for the Stargardt disease is that the length of DNA sequence needed to encode functional ABCA4 protein exceeds the typical payload capacity of AAV vectors.

AGTC and our academic collaborators have previously shown in mouse models that this change can be overcome by using a dual AAV vector expression system. With this system, the DNA encoding ABCA4 is separated into 2 pieces, each of which can be carried by an AAV vector. Once inside the cell, the DNA piece is recombined with each other to create the full length coding sequence.

AGTC has recently developed and optimized dual vector system, and assessed the efficiency of recombination and expression of the full length ABCA4 protein in the retinas of nonhuman primates.

In a 13-week study, subretinal injection of AAV is prepared at AGTC, encoding the N-terminal fragment and C-terminal fragment of human ABCA4 which was tagged to distinguish it from the naturally occurring nonhuman primate ABCA4 protein, resulted in detection of full-length recombinant human ABCA4 protein. The detected protein was limited to tissue punctures harvested from inside the subretinal bleb. These results in a nonhuman primate are, to our knowledge, the first report of such data and represents an important technical advance for the use of dual vector expression systems in retinal gene therapy. Based on the results of the study, we are continuing development of our optimized dual vector system towards IND-enabling studies.

As AGTC's clinical programs continue to demonstrate the potential of our technology platform, we're also advancing other preclinical programs, including a product for the dry form of age-related macular degeneration or AMD, which accounts for 90% of the 24 million AMD cases globally and 3 programs targeting CNS disorders. These programs include the previously announced program in adrenoleukodystrophy as well as 2 additional rare genetic CNS indications that have substantial patient population, with well-defined clinical phenotypes and represent important opportunities to create unique therapeutics solutions for patients.

In October, we also presented nonclinical immune response data that will support our existing clinical programs as well as our multiple preclinical pipeline opportunities. The data are from studies evaluating the effect of preexisting anti-AAV antibodies on the transduction and expression efficiency of AAV vectors in nonhuman primates. Results show that the presence of neutralizing antibodies to AAV in the serum or in the eye did not affect gene delivery, gene expression or inflammation following ocular administration of AAV vectors. These studies reported in the poster presented at the European Society of Gene and Cell Therapy Annual Meeting exemplified the nonclinical development work that we are conducting to support our future BLA filings and to provide regulated clinicians and patients with robust data that will help to define the optimal use of our potential products in specific indications of population.

I will now turn the call over to Bill, who will briefly review our fiscal results for the first quarter of fiscal year 2020.

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William A. Sullivan, Applied Genetic Technologies Corporation - CFO [5]

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Thanks, Mark. Our first quarter 2020 financials were included in our press release, which was distributed a short while ago. For the first quarter of fiscal year 2020, we recorded a net loss of $11.6 million compared to net income of $1.2 million for the first quarter in fiscal year 2019. The increase in net loss was primarily due to a $14 million decrease in revenues and $0.1 million increase in G&A expenses, which were partially offset by $1.5 million decrease in R&D expenses. The slight increase in G&A expenses was primarily driven by higher employee-related and other G&A expenses, partially offset by decreased share-based compensation expenses. The decrease in R&D expenses was primarily due to decreased XLRP sublicense expenses and decreased discovery spending associated with the company's preclinical ophthalmology programs.

Now I'll move onto our financial guidance. We ended the first quarter of 2020 with a strong balance sheet. Total cash, cash equivalents and investments as of September 30, 2019, were $71.1 million. We believe these funds will be sufficient to allow AGTC to generate data from our ongoing clinical programs, to initiate activities to ensure efficient transition into pivotal trials and to fund our currently planned research and discovery programs into the first half of 2021. We expect total cash, cash equivalents and investments as of June 30, 2020, to be between $30 million and $40 million.

That concludes the team's remarks today. Operator, you may now open the line for question-and-answer period.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Joe Pantginis with H.C. Wainwright.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [2]

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Hopefully, I'm not going to get too much into the weeds but I wanted to focus a little bit on the underlying science here and I'd like to start with your dual AAV approach. Very promising for Stargardt, obviously, and I think my lead question, really, is you talked about optimization of the dual vector system but can you give any more color about how you control for the expression of both proteins to be able to have the right stoichiometry, if you will? And when you look forward, what do you envision the FDA or the regulatory environment being for a dual vector system?

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [3]

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Joe, this is Sue and thank you for joining us today. and I'm going to place that question squarely in Mark's lane. So Mark?

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Mark S. Shearman, Applied Genetic Technologies Corporation - Chief Scientific Officer [4]

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Thanks for the question. So a lot of the preparation work for this program was done in collaboration with Bill Hauswirth at the University of Florida. He's recently published a paper, the first author is Dyka, D-Y-K-A, where they outline the studies that they did to identify the optimal hybrid dual vector system. We have been working with Bill for a number of years now and basically use the optimal system that was generated in the mouse model and applied that for the nonhuman primate study that we mentioned in the recent press release. The only change we made was to put a tag on the C-terminal tail of the ABCA4 so that we can distinguish the human recombinant protein from the endogenous nonhuman protein -- nonhuman primate protein. Beyond that, the dual vector is pretty much treated as a normal AAV vector. They were both manufactured in-house, QCed, and used directly in the nonhuman primates table, we'll be revealing more details of that at a later date. There are still a couple of things that we're working on, potential other optimizations that we may incorporate in the program, as we move forward.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [5]

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And Joe, to your question about -- reg -- to your question about regulatory treatment, I think it's when we start interacting with the agency to develop, in a pre-IND format, to develop the toxicology studies and beyond, is when we'll work through with them, whether this will be a mixed single product or whether it will be 2 products separately. But that is yet to be determined, and I think we're really excited to be at the forefront of the use of these dual vectors.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [6]

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Great. And then maybe just quickly because I know there's a lot of even potential hypothesis around but, when you look at the neutralizing antibody data that you put out, which were certainly encouraging, because you still see the clinical effects, just curious, is this really a function of the gene being delivered at such pharmacological levels that the antibodies can't overwhelm it?

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [7]

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Mark, you want to address that too?

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Mark S. Shearman, Applied Genetic Technologies Corporation - Chief Scientific Officer [8]

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Yes. We -- in the ocular studies, we haven't done that type of detailed analysis. I think what we can say is that based on the poster data that we presented, you really have to get to very high levels to see any kind of interaction of pre-existing neutralizing antibodies with AAV transduction and even then, there's not a consistent effect. So we believe that there are other factors which we are yet to identify that are also coming into play here, but the bottom line is that we saw pretty good transduction in that study with animals that have been pre-inoculated to generate those very high levels. And that was a main point. And then the second main point was -- it was really the -- only the injected eye that developed these antibodies. The uninjected eye remains pretty clear, and so that gives us a lot of encouragement that we will be able to dose the contralateral eye of patients without any issue.

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Operator [9]

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Our next question comes from the line of Yun Zhong with Janney.

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Yun Zhong, Janney Montgomery Scott LLC, Research Division - Equity Research Analyst & Director of Biotechnology Research [10]

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Maybe -- first, a follow-up question on the pre-existing neutralizing antibody, do you think that's specific to the AAV capsid? Or to the organ or eye and -- because you have CNS indication in the pipeline so I'm just a little curious how that will apply to your CNS indications.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [11]

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Thank you for the question and Mark, you can go ahead.

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Mark S. Shearman, Applied Genetic Technologies Corporation - Chief Scientific Officer [12]

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Yes. I don't think I can answer that directly yet. We will do similar types of studies to support our pre-CNS indications. I think the conclusions that we've recently published, we are confident of those based on the nonhuman primate studies we have done for the ocular indications. We're going to have to do something similar to determine the impact or potential impact in CNS indications. We have yet to do that.

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Yun Zhong, Janney Montgomery Scott LLC, Research Division - Equity Research Analyst & Director of Biotechnology Research [13]

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Okay. Maybe a follow-up question, if I may. So you laid out very specific guidance on the XLRP program but the guidance is a little baked on the achromatopsia program. So I wonder what information are you lacking so that you cannot -- still need to determine the pathway going forward.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [14]

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So again, I appreciate the question. And I think the reason we're able to be so very specific with the XLRP guidance is we have a lot more data on XLRP already. So we have dosed more patients, we've released data, we've analyzed data, released 6 months data on the dose escalation, we've gotten early data on the expansion cohort. And then -- so until we see that data that is being crunched right now for achromatopsia, I think it's more difficult for us to be specific. But as we said, as we crunch that data and have it in front of us and are able to analyze, then we'll be able to provide more specific forward guidance.

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Operator [15]

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(Operator Instructions) Our next question comes from the line of Jim Birchenough with Wells Fargo.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [16]

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This is Yanan dialing in for Jim. So you mentioned you are continuing to dose additional patients in XLRP study to collect more data, so the future BLA could be more robust. So just wondering, is there -- do you foresee a possibility that data from the existing study is sufficient for a regulatory decision? And also in terms of the -- if you have to do -- you think, if you have to do a pivotal trial, do you foresee whether it will be sham procedure-controlled trial? Or it is going to be a open-label trial with contralateral eye, for example, as a control?

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [17]

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Yanan, this is Sue. And that's a very good question. I -- We have guided that we expect to begin a pivotal trial in 2020. So I think that certainly indicates that we believe a pivotal trial will be necessary for registration. We're continuing to dose patients just to have the biggest body of data that is possible and in support of data. And we've also stated that we expect to have an end-of-Phase II meeting in the second quarter, and so until we have that end-of-Phase II meeting with the FDA, we would think it would be premature to provide any specific information on what we think that pivotal trial would look like.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [18]

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Got it. And then maybe a question on the XLRP data, we see that the light sensitivity improvement in centrally dosed patients reached kind of a peak at the first time point of 1-month. And that's true for both your data as well as for competitor data. So could you talk about whether that aligns with your expectation dynamics of protein expression and clinical effect? Yes. That will be helpful.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [19]

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Thank you, Yanan. First of all, I would say that we have yet to analyze the 6-month data. So I think that will be very important to understanding the curve and the expression levels that we're seeing but Theresa, maybe you could jump in here and talk a little bit more about the patients that we identified as responders and we're providing that visual fields data? And how that curve developed and what data we'll see in the future at the 6-month time point?

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Theresa Heah;Chief Medical Officer, [20]

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So Sue and Yanan, may I clarify? Are you referring to XLRP? Or achromatopsia? Because I think the question kind of overlapped between the 2 indications.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [21]

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Yes, XLRP, please?

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Theresa Heah;Chief Medical Officer, [22]

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Yes. So the XLRP data, we have released the centrally dosed patients, as Sue mentioned, with the retinal or visual sensitivity at 3 months. So at 6 months, that's what we'll release in the next -- by -- before or end -- near the R&D Day. In comparison to our peer, you can see at the most recent AAO meeting, I think we are -- we have a stronger, more robust and comprehensive data set which also -- we included visual acuity information. So I think we'll have to see what happens at 6 months but we continue to follow that patient and until we have data to analyze them, I won't be able to comment at this stage.

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Yanan Zhu, Wells Fargo Securities, LLC, Research Division - Associate Analyst [23]

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Got it. And maybe lastly, a question on potential possibility for redosing because you mentioned the tighter pre-existing antibody doesn't seem to impact the treatment effect. So just wondering, in patients that you treated before, if you feel like there is additional area in the retina that can be treated again, whether second subretinal treatment can proceed following the first one, which obviously, the first one might elicit a certain level of immune response. But do you think it is possible to redose?

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [24]

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Theresa, that's squarely in your lane.

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Theresa Heah;Chief Medical Officer, [25]

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So -- Mark is also in our nonclinical, we're looking at the nonclinical of multiple bleb subretinal injections. But in real life, if you look at Luxturna, we've spoken to several of our surgeons and retinal specialists for Luxturna. They're actually injecting multiple blebs and we believe that at XLRP, when -- once approved in real life, as we can see here, depending on the discussion between the retinal specialists and the patient, what's the patients need in terms of whether they want to stabilize their peripheral field or whether they want to make sure there's a stabilization or improvement in their central vision, really, we believe that -- and our retinal specialists believe that we are the future in terms of the multiple bleb and with the protein expression. But Mark, I don't know if you want to comment for them.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [26]

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Yes. Certainly, Mark -- Mark's data supports that because Mark, in addition to that antibody test, you have conducted nonhuman primate studies with multiple blebs. So you could talk just a little bit about that.

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Mark S. Shearman, Applied Genetic Technologies Corporation - Chief Scientific Officer [27]

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Yes. For sure. Yes. We have a couple of different studies that we have either finished doing or are in the process of doing. One, as Sue just mentioned, is multiple blebs in one eye. And again, that has been well tolerated and in fact, we've sort of implemented that paradigm in some of the nonclinical studies that we're looking at. But the second still ongoing study, which I think is, even more relevant, is to redose the same eye at a different location with subretinal injection. And to date, that seems to be well tolerated and we will be certainly discussing that data more as it -- as the study is completed and obviously, we'll publish it at some point in the future, too.

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Operator [28]

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Our next question comes from the line of Matthew Luchini with BMO Capital Markets.

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Matthew W. Luchini, BMO Capital Markets Equity Research - Analyst [29]

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Congratulations on the progress. So maybe first, just taking a step back. It's been maybe 6 weeks or so since the initial data were released, and I'll be curious to hear a little bit about, what you've been hearing from physicians on this data, now that you've had the chance to share it a bit more broadly. And specifically, what areas is resonating with them, perhaps versus other XLRP or even earlier achromatopsia data sets they may have seen? And then secondly, recognizing that we're going to have an Analyst Day at the end of next -- at the end of January, how should we be thinking about what shall be communicating at that start of the month, in terms of, will it be just a very basic top line press release and conference call? Will it be something more? How do we just -- how should we -- what's your expectations be going into the early data release versus what we'll get later in the month? And then I have one more follow-up after that.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [30]

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Thank you, Matt, for the questions. I'll start kind of with your second question first. And that is, what we will communicate early. Our plan is to do a press release with top line data as soon as it's -- as the data is available and then hold the detailed discussion for that January 28th day. I think that one thing that we've learned is that we are developing a very comprehensive and detailed data set and it is valuable to people to be able to sit and look through it and ask questions. And so at this research day on the 28th, we will have surgeons, we'll have clinicians, we'll have our team, so that people will really be able to dig into the data, but we won't wait for the 28th to release the top line information. So that's the first question.

And second question you asked, what are we hearing from physicians and others as we've gone around and been talking about this data. And I'll start out with a broad comment but I really want to give this question to Theresa because she's had much more detailed interactions. But the broad comment I will say is that it's been a very, very positive response, that people are very encouraged by the data we are showing and are encouraged by the detailed way in which we are explaining the data. And we've also had a large uptick in the number of patients that have been inbound into our clinical trial sites and are setting up screening visits. So we consider that all to be positive. But Theresa, if you want to add some color? You've had many more one-on-one and group discussions with physicians in our sites than I have.

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Theresa Heah;Chief Medical Officer, [31]

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Sure, Sue. Yes. We actually review our data with the -- our study investigators, our thought leaders, the retinal specialists and also the clinical advisory board members of AGTC prior to our data release, also up to our data release. There's been a lot of excitement from individuals and study sites. In fact, there's been several requests of wanting our data to be presented at local meetings, local conferences. We have a lot of excitement as well from all the patient efficacy groups, actually requiring more information. And as Sue mentioned, there's been a lot of inquiries from patients as well through our patient advocacy group.

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Matthew W. Luchini, BMO Capital Markets Equity Research - Analyst [32]

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Great. That's helpful. And then on Stargardt, I believe Nightstar was also working in that area and they had a dual vector approach as well. I was just wondering if you might be able to provide any color on how, if at all, your approach is different from theirs? And I guess, more broadly, thinking about the program, are you be able to provide any thoughts on when you might -- when we might actually see an IND for this program?

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [33]

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So thanks, Matt, for that question. I'll go ahead and take that question. Especially since Nightstar has been absorbed into Biogen, I don't think there's been any public information about their program. I do think it was a dual vector program, how similar it was to what we've been working on with Dr. Hauswirth, I don't think we have any information on that. I think the key thing is that they had never taken it forward into nonhuman primates. We don't feel anyone in ophthalmology has taken dual vectors forward into nonhuman primates and shown that you could get enough recombination and expression of the protein to have a clinical effect. So we think that that's where the differentiation is, that we've done the work, taking it forward into nonhuman primates and then would be ready to take it into IND-enabling studies. And at least at this time, we're not providing any specific guidance on when that IND would be filed.

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Operator [34]

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Thank you. We have reached the end of the our question-and-answer session. Allow me to hand the floor back to Sue Washer for closing remarks.

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Susan B. Washer, Applied Genetic Technologies Corporation - President, CEO & Director [35]

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Thank you. Our first quarter achievements have positioned us for a potentially transformative year in 2020. As mentioned earlier, we are planning to host an R&D Day in New York on January 28 in order to review from -- data from all 3 trials in greater detail and to include conversations with clinical and surgical experts and patients. We'll provide additional information in the next several weeks and look forward to meeting with many of you at this event.

As Theresa mentioned, we are planning to continue our action -- interaction with the FDA, with the goal of initiating a pivotal trial in XLRP in the second half of 2020, which would be an important validation of our technology platform, our manufacturing and our clinical and regulatory development capabilities.

As I always do, I'll close today's call by thanking the patients, physicians and the AGTC team for their dedication to our cause and their support of our efforts to just transform the treatment for rare ophthalmic otology and CNS diseases.

I look forward to sharing our joint achievements with you in the months ahead. Thank you, again.

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Operator [36]

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Thank you. This concludes today's conference. You may disconnect your lines at this time, and have a great day.