Applied Genetic Technologies Q1 2022 Earnings Call Transcript

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Provided by Quartr
November 8, 2021

There are 11 speakers on the call.

Operator

Good morning and welcome to the AGTC Financial Results Conference Call for the Q1 of Fiscal Year 2022. Today's call is being recorded. Before we get started, I would 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, including statements about its ongoing and planned clinical trials and preclinical programs. Actual results could differ materially from those discussed in these forward looking statements due to a number of important factors, including uncertainty and the clinical development and regulatory process, the extent and duration of the impact of the COVID-nineteen pandemic and other risks described in the Risk Factors section of AGTC's most recently filed annual report on the Form 10 ks and other periodic reports filed with the SEC. AGTC undertakes no obligation to update any forward looking statements after the date of this call.

Operator

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

Speaker 1

Good morning and thank you all for joining us. During our last quarterly call in September, I had the pleasure of introducing John Lieber as Senses expertise and a track record of success in ophthalmology drug development, including the approval and launch of Lucentis. Her experience and insights will be invaluable as we work to advance our XLRP and achromatopsia trials towards approval And as we expand our clinical portfolio with the advancement of our 2 lead preclinical programs towards IND smiling. Both Susan and John join me on today's call. Last week, we welcomed Sara DeSalvizio as Vice President of Clinical Operations, and we expect that her strategic leadership and contributions to the FDA approval of multiple rare disease therapies further enhances our robust clinical development capabilities.

Speaker 1

Our ability to attract high quality talent like Susan, John and Sarah reflect the great potential of our pipeline and technology as well as our amazing corporate culture, which is built on a shared commitment to improving patients' lives and values the contributions that each member of our team brings towards achieving that vision. Now turning to our ongoing clinical trials, Our XLRP program is our nearest turn opportunity to bring a potentially transformative therapy to patients living with a disease that today has no treatment option. As many of you are aware, we presented promising data from the ongoing Phase III trial earlier this year. This includes 12 month data from patients in the 2 highest dose groups in this trial, demonstrating a 50% response rate among patients who meet the inclusion criteria for the ongoing Skyline and VISTA trials as well as improvements in visual acuity across a wide range of patients that provides supportive evidence of biological response. Additionally, data from a subset of patients in the high dose group who or available for analysis at 24 months, provide evidence of response durability and continued safety.

Speaker 1

As a reminder, we believe that our Skyline and VISTA trials will be successful if we are able to demonstrate the same 50% as we did in patients in the ongoing Phase onetwo trial that meet the inclusion criteria and assuming that we see a favorable safety profile. In September, our clinical collaborator, Doctor. Paula Yang, Assistant Professor of Ophthalmology at the KCI Institute, presented data from a 12 month analysis of macular structure Using optical coherence to evaluate patients in the Phase onetwo XLRP trial, the data, which Susan will review in a few minutes, identify a correlation between macular structure and visual function that supports the treatment effect of our XRP gene therapy candidate. These data add to the growing body of evidence supporting the industry leading potential of this product candidate and further increases our confidence in the possibility for positive results in both the Skyline and Vista clinical trials. Based on the totality of the data generated to date, improvements in visual sensitivity, improvements in visual acuity, Positive patient anecdotes and Doctor.

Speaker 1

Yang's observations of improvements in macular structure, we believe that our We are currently conducting the Skyline Phase III Faxion trial and the VISTA Phase twothree trial and we expect that data from both trials will support a potential filing of a biologic license application or BLA. We anticipate multiple data readouts from our XLRP clinical trials between now and the end of 2022, which Susan will outline in a moment. Each of these readouts is an opportunity to further demonstrate the potential value of our XLRP product candidate, And we believe that collectively these trials will provide the most robust and differentiated set of data for any XLRP gene therapy currently in clinical development. Now let me turn to our chromatopsia clinical program. The data reported to date support the continued development of our chromatopsia with the clinical development of the B3 program and are preparing an end of Phase 2 briefing document for submission to the FDA on which we expect their feedback in the first half of twenty twenty two.

Speaker 1

The path forward for our A3 product candidate will be determined after additional pediatric patient and preclinical data are available for evaluation. Before moving to our preclinical pipeline, I also wanted to remind about our collaboration with Vionic Sight in optogenetics. Earlier in 2021, Vionic Sight announced Promising initial Phase III data that showed that treated patients, all of whom are complete or near completely blind, can now see light and motion and in two cases can detect the direction of motion. Our diversified preclinical pipeline provides multiple opportunities to expand into additional disease areas outside of inherited retinal diseases, including in central nervous system or CNS indications and otology indications and to address large markets outside of the rare disease space, such as the dry form of age related macular degeneration or dry AMD. As previously reported, We have prioritized our programs in dry AMD and frontal temporal dementia for the advancement to IND filing and are on track to initiate As part of our strategic collaboration with Otonomy, The otology program is moving forward with an IND application expected to be filed in the first half of twenty twenty three.

Speaker 1

Key to our ability to advance our clinical and preclinical candidates as quickly as possible is ensuring access to high quality, Scalable and High Productivity Manufacturing. Our leased build to suit current good manufacturing process or cGMP Manufacturing and quality control facility remains on track to become operational in the Q4 of 2022. We have already made several key hires to support the design and construction of the building and intend to continue to bring an additional Staff to support the validation of the facility in the second half of twenty twenty two. Our goal for this custom manufacturing capacity is to enable an expedited BLA filing and commercial launch of our XLRP product candidate subject to FDA approval and support late stage development of our achromatopsia program. Our investment in and commitment to this facility reflects Both our confidence in the clinical commercial potential of our XRP and achromatopsia clinical programs and our commitment to supporting more rapid advancement Now, I will turn the call over to Susan, who will provide additional detail on our exoRP and achromatopsia clinical program.

Speaker 1

Susan?

Speaker 2

Thank you, Sue, for the warm welcome. I'm pleased to join my first call as AGTC's Chief Medical Officer, and I'm excited to have the opportunity to share several positive updates for both of our ongoing clinical programs. As Sue mentioned, Doctor. Paul Yang presented promising new data from the ongoing Phase III XLRP clinical trial at the 14th International Symposium on Retinal Degeneration in September. The data he presented were from an analysis of macular structure in patients 12 months after receiving a single dose of our XLRP product candidate.

Speaker 2

A hallmark of XLRP is that the ellipsoid zone or EC, which is a defined region within the photoreceptor layer of the retina, She generates over time and is eventually lost. The data Doctor. Yang presented shows that intact retinal anatomy at baseline predicts potential for improvement. Of 20 patients evaluated in our XLRP Phase III clinical trial, 13 had intact baseline retinal anatomy, of which 9% or 69% had either complete recovery or improvement in EZ at 6 months. By contrast, the 7 patients who had end stage retinal anatomy, as evidenced by absence of the EZ, showed no structural or functional improvements post treatment.

Speaker 2

The data also showed that there was a significant association measured by improvement in EZ in groups 4 to 6. Among these patients, 4 of 6 with MYA improvement also had EZ improvement, and there was a statistically significant association between MAIA improvement and EZ improvement with a p value of 0.0212. We believe that these data further support a treatment effect from our XLRP product candidate and continue to differentiate the data set for our product candidate from competitive products in development. As Sue also mentioned, we are actively enrolling and dosing patients in both the Skyline and VISTA trials and expect to report multiple data sets from these trials in 2022, including 3 month interim Skyline trial results In the first half of twenty twenty two, 24 month trial results from the ongoing Phase III clinical trial In the Q3 of 2022, 12 month Skyline trial results in the Q4 of 2022 and 6 month interim VISTA trial results in the Q4 of 2022. Our clinical collaborator, Doctor.

Speaker 2

Robert Sisk of Cincinnati Children's Hospital and University of Cincinnati College of Medicine We'll also make an ENCORE presentation of the 12 month data from the Phase onetwo trial at the American Academy of ophthalmology annual meeting taking place from November 12 to 15, 2021. We're pleased to have this additional opportunity to help educate the ophthalmology community about the potential benefits of our XLRP candidate. With respect to the achromatopsia programs, we recently enrolled 6 pediatric ACHMB3 patients and 5 pediatric ACHMA3 patients in higher dose groups 5A and 6A and identified a maximum tolerated dose. As we have previously reported, to address suspected unexpected serious adverse reaction or SUSAR, Safety events in pediatric patients in the highest dose group, systemic and local steroid doses were increased and patients are being monitored closely. I am pleased to say that the patients are doing well, and our investigators are tapering these patients to lower steroid doses.

Speaker 2

Importantly, we did not see comparable inflammation in the 6 pediatric patients across both trials at dose group 5a nor in any of the adult patients for lowest group 4 pediatric patients on which we previously reported and we plan to continue development of our We expect to report interim 3 month data for the pediatric patients in both achromatopsia trials before the end of 2021. For the B3 program, We are also developing an end of Phase II briefing packet for submission to the FDA and expect to receive feedback from the agency In the first half of twenty twenty two, the agency's input will play a foundational role in our plans for advancing the Achromatopsia Now I'll turn the call over to John for a review of our financial results.

Speaker 3

Thanks, Susan. For the Q1 of fiscal year 2022, we recorded a net loss of $17,100,000 compared to a net loss of $15,400,000 for the Q1 of 2021. The increase in net loss was primarily due to an approximate $700,000 increase in research and development expenses, an approximate $700,000 We ended the Q1 of fiscal year 2022 with a strong balance sheet, including total cash and cash equivalents of 90,500,000 We believe these funds will be sufficient to allow us to generate data from our ongoing and planned clinical programs and Fund currently planned research and discovery programs into calendar year 2023. That concludes the team's remarks today. Operator, you may now open the line for a question and answer period.

Operator

Thank you. We will now be conducting a question and answer session. One moment please while we poll for your questions. Our first questions come from the line of Joe Pantginis with H. C.

Operator

Wainwright. Please proceed with your questions.

Speaker 4

Hey, everybody. Good morning and thanks for taking the questions. So first, I just wanted to ask about or maybe A quick question about the SUSAR events in the achromosatopsia study. Is it safe to assume, sorry, no pun intended, That this is really a function of just the higher concentrations that lead to this in pediatric patients and it really is expected.

Speaker 1

Good morning, Joe. Thanks for your question. And yes, we really do believe that this is a dose effect in very young patients. Remember that these Pediatric patients were between the ages of 48 years old and we didn't see any of this kind of inflammation In any teenagers, in any of the lower dose or in any of the adults. So we really believe it's a dose effect in these very young children.

Speaker 4

Got it. That's very helpful. And then more on the macro front, you guys are obviously very, very busy with regard to the build out of your new facility that you say should come online next year. So I was just curious, have you had any issues or additional planning that you've had to Embark upon regarding the global supply chain issues?

Speaker 1

That's a very good issue because Question, because I think overall, Joe, we're all experiencing some level of constraints due to supply chain issues. For that reason, we did take a very proactive stance on the manufacturing facility. And even though we're breaking ground and removing dirt around, we've actually already ordered the equipment That we're going to need in the facility with the idea that if it comes in on time or early that we can store it in our existing Facility before we move it in there. So we're really trying to do a ton of advanced planning to make sure that we hit our mark Of the engineering runs in late 2022.

Speaker 4

Got it. And my last question, if you don't mind, and thanks for indulging me. I don't know if it's too early to ask this question, but are you willing to take any broader strokes with regard to the design of your future dry AMD program and how you might mix endpoints of drusen geographic atrophy and also visual acuity?

Speaker 1

Yes, I do think, Joe, that it's a bit too early. I can understand the interest there, but we're really right now putting together our package for the pre IND meeting with the FDA and developing the protocol synopsis with experts. And so I think We would like to wait and comment on that until we have that protocol synopsis complete.

Speaker 4

Totally understood. Thanks a lot for the comments, Sue.

Speaker 1

Have a great

Operator

day. Thank you. Our next question comes from the line of Dae Gon with Stifel. Please proceed with your questions.

Speaker 5

Hi, good morning. Thanks for taking our questions and congrats on all the progress. Sue, maybe if I can just go back to the Sustar question, Understanding that it's probably more dose and patient baseline age related, can you, I guess, Maybe talk a little bit about AAVTYF and its function or is this something that's probably going to be More broad to other serotypes of AAV, is there anything unique about AAVTYF or do you think this is going to be applicable to other Perhaps ocular gene therapy programs addressing the pediatric population? And then secondly, just with regards to that Doctor. Yang data that you guys were just Briefing us on very interesting, but I guess just thinking about development forward, I know previously you were thinking about Mobility Maze from Skyline trying to draw correlations to the visual function data and making that as a potential regulatory endpoint.

Speaker 5

I guess, where does EZ come in given the correlation data that Doctor. Dieng presented? Have you guys already interacted with the FDA about perhaps using that also as a supportive measure? Thanks.

Speaker 1

Thanks, Bhagavantam, for the questions. I'll Take the Sustar question and then probably give you a top line on the Sustar versus Mays and then give Susan, an opportunity to weigh in on her thoughts there. As far as the capsid being differences in capsid being potentially We have presented data and I think we've reviewed that reviewed it publicly in our corporate presentation Where we've looked at multiple capsids side by side and when you dose these multiple capsids side by side, manufactured the Same way, titer the same way, purified the same way. We don't see a difference In the inflammatory response, what we see is that despite whatever capsid you have at certain high doses, You do see an inflammation response that tapers over time and resolves itself, sometimes even without any steroids whatsoever. And so we don't believe that anything we're seeing in the CSAAR has anything to do with the specific capsids.

Speaker 1

There are some capsids, There's some engineered capsids that you can screen out of libraries and some more off the beaten track, if you want to say capsids that do Create a higher inflammatory response, but not among the TYF or AAV5 or AAV8 or other Captains that we've looked at over time, we don't see that as a positive effect, just total dose. TYS itself, part of how it was engineered is to actually allow it to dwell within the cell Longer and get into the nucleus in a higher percentage such that the DNA is available To have higher expression of protein and that's exactly what we see in side by side comparisons of our TYF capsid with others is that it does support in non human primates about twice the expression of the intended protein as other capsids. So So now moving on to Doctor. Yang's data, we do we have already written and talked to the FDA about our protocols for Skyline and VISTA. We do intend that visual sensitivity is the primary endpoint and with the mobility maze as a secondary endpoint along with visual acuity, We will be collecting the EZ Line data.

Speaker 1

And so I'll turn it over to Susan for comments on its usefulness and supportive nature Of supporting our product candidates.

Speaker 2

Thank you, Sue. Yes, for Doctor. Yang, the improvements in anatomy Are really quite interesting and we always look for association and alignment with changes in anatomic structure and visual function. So for that sense, the alignment with the functional changes are very important to be both clinically meaningful and And hopefully clinically significant in outcomes. And again, this helps us differentiate our product from others and help us to meet the unmet need in these patients.

Operator

Thank you. Our next question has come from the line of Yan Hsu with Wells Fargo. Please proceed with your questions.

Speaker 6

Good morning. Thanks for taking my questions. So first, I have a Couple of questions on enrollment into the Skyline and VISTA trial, mainly not The enrollment status, I know you generally don't provide update into those on an ongoing basis, But how what about the cadence and whether you think that cadence can give you confidence for the Now Q1 'twenty two, 3 months skyline readout, given that in the past COVID has impacted enrollment? And also, how do you prioritize the enrollment of Vista and Skyline, given the potential bottleneck of COVID intact. Thank you.

Speaker 1

So, Yanan, thank you for the questions, and I hope you are having a good morning. You're right. We don't usually provide exact enrollment status as in numbers and types of patients. But we did report earlier, as you mentioned, that over the summer, we had had a fairly high cancellation rate For screening and initiation of patient visits, and that has largely have gone away. We are not having those issues.

Speaker 1

The sites are seeing patients on a regular basis. We also have Fully used our mobile vision centers. I think many of you have heard us talk about the mobile vision center as a way to Capture data during the pandemic and we use that especially for achromatopsia patient. It's a mobile vision center that Travels directly to the patient's home to be able to test data. We now actually have 2 fully operational mobile vision centers And not only are they doing follow-up visits in certain cases, they're also screening and prescreening patients for us.

Speaker 1

And this is something that The patients and the sites are very appreciative of because when the patients actually have to travel to the sites, They already know that they have been prescreened in the past much of the inclusion exclusion criteria. And so the site knows that its time is going to be well So we're very confident that we've worked through those issues in the summer and we're Confident in our data guidance that we've provided.

Speaker 6

Great. Thanks for that. Very helpful update. And also another question on the competitive landscape I think our competitor reported last month data from intravitually administered AAV gene therapy For XLRP, I know the data set is pretty small and early. Could you share your thoughts on that data set and how Should we think about the competitive profiling of that gene therapy?

Speaker 6

Thank you.

Speaker 1

Again, and thank you for that question. We do believe that the data presented Was limited. We have no insight other than the data that others have seen and we would think it was Way too early to make a statement about that data and we would wait to see more fulsome. We do believe that our product With the subretinal delivery really supports higher expression of the protein needed to have an effect in the photoreceptors. And our data is very strong.

Speaker 1

And as we've talked about, we now see improvements in visual acuity, visual sensitivity, Yes. Patient anecdotes, clean safety profile and then now the encouraging data from Doctor. Yang.

Speaker 6

Got it. Thank you, Susan. That's very helpful. And maybe if I may have one last question that is for the 12 months data readout Update for the ongoing Phase III study at AAO, what incremental data will we see Compared with the last 12 months data readout? Thank you.

Speaker 1

So Doctor. This will not be presenting brand new data. It's really, as Susan described, an ENCORE presentation of the data. But what he will be doing is Linking the data in a way that hasn't been concentrated on before, really showing the visual sensitivity data in conjunction With Doctor. Yang's analysis of the EZ line and also going through some surgical procedures and improvements that have So it's really a way, a capstone presentation to kind of pull together all of the data that's been released over the last 18 months Into one consolidated fashion.

Speaker 6

Great. Thank you, Susan.

Operator

Thank you. Our next questions come from the line of Zaghav Jala with ROTH Capital Partners. Please proceed with your questions.

Speaker 7

Good morning. Thanks for taking my questions and congrats on the new hires. I think just two quick ones for me. The first one This is about Doctor. Yan's data.

Speaker 7

I think Susan noted the intact metinab being productive of a response or even fact. And so I was just wondering If your baseline perimeter threshold that you've set is actually going to Be able to also capture that same patient population.

Speaker 1

So, Sangla, good morning and happy to have you on the call. And that's a good question because we previously talked about how we altered the inclusionexclusion criteria between The Phase onetwo and the Skyline and Vista. And I can say that we were already thinking that there was a correlation here Between that baseline MYA and what we would want to see in a healthy retina, So Skyline and Vista already include a look at this imaging, the EZ line, And we do believe that what we have set for Skyline and Vista will capture the intent and the findings that Doctor. Yang had.

Speaker 7

Thanks. And then the last one here is just about the HCM study and the SUSAR. We're just wondering Beyond treating those patients with steroids, are more patients still being enrolled into the study? How do you plan to proceed regarding dose? And any thoughts on whether or not that CUSAR may have impacted or will impact enrollment into the study?

Speaker 1

So we had completed enrollment in B3 of all intended patients. And I think as we noted previously, Lee, in A3, there was only one more patient intended to be enrolled in that very high dose in the young age group, And we have held off on that. We are not dosing that final patient in that very high dose. We think it's prudent to collect Long term data and make sure those patients are doing well and don't have plans at this time to dose that last patient.

Speaker 7

Thanks.

Operator

Thank you. Our next question has come from the line of Matthew Luchini with BMO Capital Markets, please proceed with your questions.

Speaker 8

Hi, good morning. Thank you so much for taking the question. Maybe just to start 2 with a bigger picture question. I want to ask about the recently announced BCTG, the bespoke gene therapy consortium Focused on, among other things, AAV. Just wanted to get your perspective on that effort And I'm trying to understand how AGTC may be able to leverage whatever findings You know, are eventually resolved from that group.

Speaker 8

And then secondarily and more specifically to the company, one to ask, Following the RSO data the presentation of XLRP data at RSO, One of the larger meetings where the state has been presented. I was just curious if you could provide any anecdotal physician feedback you may have gotten at the meeting. And then lastly on achromatopsia, just housekeeping, wanted to confirm if any of the suicidal patients are now fully off of steroids? Thank you very

Speaker 1

much. As far as the VC GT, we think that this is an excellent program that has been started and we are very much Engaged in participating and learning what they bring forward and will And definitely think that sharing at some level of information and data around gene therapy treatments is positive for everyone in the industry. And so we'll be watching that very carefully. As far as physician Feedback, we've had very positive physician feedback on our XLRP program. And as I have mentioned before, Going from Skyline and Intevista, we're expanding the number of sites dramatically And really have not had an issue with physicians being interested in becoming sites, in going through the surgical training And really expanding our reach across the country and into the EU Based on the data that we have seen so far, so very positive physician feedback there.

Speaker 1

And on the Jim, as Susan noted, all of those patients are tapering off steroids, but none of them are completely to 0 yet. It hasn't been Long enough period of time to get down to baseline.

Speaker 8

Okay. Thanks for taking.

Operator

Thank you. Our next questions come from the line of Kristen Kluszkin with Cantor Fitzgerald. Please proceed with your questions.

Speaker 9

Hi, good morning everybody. Thanks for taking my question. Just a quick one here. As you really had the first back to back 6 month Data in January last year, literally right before the pandemic, I wanted to ask how these in person medical meetings that Are taking place including AAO are really going to be helpful in terms of you getting the word out, especially because you haven't had too many in person opportunities Since this initial data as well as the follow on updates has been presented?

Speaker 1

Thank you for the question, Kristen. I do believe that as meetings return to a more normal cadence And we have more opportunity to interact with people more broadly, but that will be generally helpful. But as I stated, we've been very effective reaching out to physicians and educating them through our key opinion leaders. And we've really dramatically expanded, as I mentioned, the number of sites that are involved in the trial and referring to the trial. So while I think initially there was some lag and concern about getting the word out, I think that That has really we've been able to be very effective later during the pandemic time.

Speaker 1

But you're certainly right. I mean, I went to my first large Conference a couple of months ago and it really reminded me how well you can do interacting with people, Really getting at their questions, really providing good feedback when you're face to face. So we're looking forward to AAO. We're very much looking forward to ARVO and ASGCT in the first half of twenty twenty two, And we do think that this will increase our ability to get the word out on our programs.

Speaker 9

Thank you. And then just in light of this recent data at the international symposium and really starting to put all of the endpoints Together and see some correlation, it seems which is not necessarily unique to the company that of Of course, earlier intervention is really what's going to lead to the best potential outcomes. So as you start to think about this and awareness Increases, what implementations or factors are you considering right now to make sure that the patients that are out They are really getting diagnosed or tested earlier on where there's potential for greater benefit at that point.

Speaker 1

So as far as getting the word out, Kristen, I think that that's another good point that you're making. Not only get the word out to physicians, but get the word Out to patients. And that's where I think our long standing commitment to patient advocacy and our partnership with the Foundation Fighting Blindness as well as a wide range of patient advocacy groups really stands us in good And we do a lot of work on education and outreach within this area. The Foundation Fighting Blindness now has 3 called Myretina where they actually provide services to genotype patients and we're very of that effort. And I think that this is the way the grassroots connections to the different patient advocacy groups.

Speaker 1

This is how you get the word out and make sure people are genotyped and aware of potential clinical trials and then eventually a potential product.

Speaker 9

Great. Thank you very much.

Operator

Thank you. Our next question has come from the line of Yun Zhong with BTIG. Please proceed with your questions.

Speaker 10

Hi, good morning. Thank you very much for taking the question. So a follow-up question on the EC area. Sorry if I missed it, but did you say that you looked at those patients at 6 months? And did you look at untreated ice and how Do they compare to treated eyes?

Speaker 10

And also, did you look at the patient at 12 months and maybe at 24 months? And I have a follow-up question, please.

Speaker 1

Eunan, thanks for the question. And the data that Doctor. Yang analyzed is really very interesting. He was looking in the presentation given and the data that we're describing At the 12 month time point, and certainly based on what we observed at 12 months, we would be very interested to see what How that data matures at the 24 month time point? And then and Susan, do you have any further comments to add?

Speaker 2

No, as I said, it's important to have an alignment with what is functional changes as well as the structural changes. I think some of these and there was a comment made earlier about Treating earlier when we have treatments and I think that that's supported by these data at looking at the baseline retinal anatomy To hopefully predict outcomes, functional outcomes, very exciting data and I agree longer term data Will be very important for us to look at as well.

Speaker 10

Okay. And I remember that Nightstar previously reported virtually very little Change in terms of structure in XLRP patient, but recently another company reported 2 1 probably a little over 10% decrease and the other one probably around 25% decrease from baseline in Treated eye. So it seems to be a lot of inter patient variability. And is that mainly due to baseline disease severity or anything else can contribute to Variability in easy area, please?

Speaker 1

That's another good question. I can't Comment in detail on the data that you're mentioning from Nightstar, which was a program picked up by Biogen. But I can say that XLRP is heterogeneous in its patients and it can sometimes be very age related depending on what age of the patient is compared to the age of another patient. But all of these patients do have a steady decline in their the health of their macula as it's Being measured in our case by the easy line, so it's not surprising that untreated eyes do decline over time. That's very well documented That's what happens in these patients and that's why we're so committed to bringing a product forward because without treatment, All of these patients are going to go blind.

Speaker 1

There is that's what the natural history study says, that's what all these patients expect.

Speaker 10

Okay. And last question, on the briefing document for the B3 program of chromatopsia, any changes in terms of what efficacy endpoints you would like to propose to the FDA?

Speaker 1

No. We're still putting that briefing package together and like my I meant about the protocol for the dry AMD. I think until we have that briefing package put together and have received feedback From the FDA, we'll withhold comments. But as we did with XLRP, we will fully report our interactions with the FDA and the agreed upon protocol.

Speaker 10

Okay, great. Thank you very much.

Operator

Thank you. There are no further questions at this time. I would like to turn the call back over to Sue Washer for any closing comments.

Speaker 1

Thank you. Our Q1 of fiscal 'twenty two reflected continued execution of our clinical trial strategies for our XLRP and our chromatopsia gene therapy candidates. We are generating robust clinical and patient reported data that we believe will support the submission of a BLA for our industry leading XLRP product candidate and will also enable us to engage in productive discussions with the FDA With regards to the next steps in our Chromatopsia B3 program, we are proud of the quality, quantity and diversity of data that we have shared with Medical, scientific, patient and investor communities for our XLRP and achromatopsia programs and we are excited about the potential for each of these products to transform patient care because we're committed to pursuing visionary science to advance the quality of care for our patients. Our investment in our new lease manufacturing and quality control facility are a key part of our strategy to advance the development of both lives for a therapy that could have made a meaningful difference and we're committed to addressing this urgent need. With the addition of John, Susan and Sarah, Our team has never been better positioned to prepare for the multiple opportunities that lie ahead.

Speaker 1

The substantial progress we have made in our clinical programs Would not be possible without the continued support of our clinical partners and our investors and most importantly, without

Operator

Thank you. That does conclude today's teleconference. We appreciate your participation. You may disconnect your lines at this time. Have a great day.

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