Applied Genetic Technologies Corporation
Q1 2022 Earnings Call Transcript

Published: 9 Nov 2021

[Operator:]

Good morning and welcome to the AGTC financial results conference call for the First Quarter 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 inherent in the clinical development and regulatory process. The extent and duration of the impact of the COVID-19 pandemic and other risks described in the Risk Factors section of AGTC 's most recently filed Annual Report on the Form 10-K and other periodic reports filed with the SEC. AGTC undertakes no obligation to update any forward-looking statements after the date of this call. For introductions and opening remarks, I would like to turn the call over to Sue Washer, Chief Executive Officer of AGTC. Please go ahead.

[Sue Washer:]

Good morning, and thank you all for joining us. During our last quarterly call in September, I had the pleasure of introducing Jon Lieber as our new Chief Financial Officer. Today, I am pleased to introduce Susan Schneider, our new Chief Medical Officer, who brings extensive expertise and a track record of success in ophthalmology, drug development, including the improvable and launch of Lucentis per experience and insight will be invaluable as we work to advance our XLRP in a chromatopsia trials towards approval, and as we expand our clinical portfolio with the advancement of our two lead preclinical programs towards IND filings. Both Susan and Jon joining me on today's call. Last week, we welcomed Sarah DiSalvatore, Vice President of Clinical Operations, and we expect that her strategic leadership and contributions to the FDA approval of multiple rare diseases therapies further enhances our robust clinical development capability. Our ability to attract high-quality talent like Susan, Jon, 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 value 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 term opportunity to bring a potentially transformative therapy to patients living with the disease that today has no treatment options. As many of you are aware, we presented promising data from the ongoing Phase 12 trial earlier this year. This includes 12 months 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 provide supportive evidence of biological response. Additionally, data from a subset of patients in the high dose group who were available for analysis at 24 months, provide evidence of response durability, and continued safety. As a reminder, we believe that our Skyline and Vista trials will be successful if we are able to demonstrate the same 50% response rate as we did in patients in the ongoing Phase 1-2 trial that meet the inclusion criteria in assuming that we see a favorable safety profile. In September, our clinical collaborator, Dr. Paul Yang, Assistant Professor of Ophthalmology at the Casey Eye Institute, presented data from a 12-month analysis of Macular Structure using optical coherence to evaluate patients in the Phase I/II XLRP Trial. The data, which Susan will review in a few minutes, identify a correlation between maximum (ph) structure and visual function that supports the treatment effect of our XLRP gene therapy candidate. These data add to the growing body of evidence supporting the industry-leading potential of this product candidates, 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 Dr. Yang's observations of improvements in macular structure, we believe that our XLRP Gene Therapy may provide meaningful and durable benefits to patients. We are currently conducting the Skyline Phase 1-2 expansion trial and the Vista Phase 2-3 trials 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 EXL RPGN 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 clinical candidates and have also allowed us to identify a maximum tolerated dose in pediatric patients. As previously reported, we are moving forward with the clinical development of the B3 program and are preparing an End-of-Phase II briefing document for submission to the FDA, on which we expect their feedback in the first half of 2022. The path forward for our A3 product candidate will be determined after additional pediatric patient and pre -clinical data are available for evaluation. Before moving to our pre -clinical pipeline, I also wanted to remind everyone about our collaboration with Bionic Sight in optogenetics. Earlier in 2021, Bionic Sight announced promising initial Phase 1-2 data that showed that treated patients, all of whom are complete or near completely blind, can now see light and motion. And in 2 cases, can detect the direction of motion. Our diversified pre -clinical 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 H-related macula degeneration or dry AMD. As previously reported, we have prioritized our programs and dry AMD and frontal temporal dementia for the advancement to IND filing and are on-track to initiate toxicology and bio-fusion studies for both programs in 2022. As part of our strategic collaboration with autonomy, the otology program is moving forward with an IND application expected to be filed in the first half of 2023. Key to our ability to advance our clinical and pre -clinical 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 quality control facility remains on track to become operational in the fourth quarter 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 2022, Our goal for this custom manufacturing capacity is to enable an expedited BLA filing and commercial launch of our XLRP product candidates 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 XLRP and achromatopsia clinical programs, and our commitment to supporting more rapid advancement of our entire product pipeline. Critically, this facility will also provide supply chain redundancy and reduce manufacturing risks. Now, I will turn the call over to Susan who will provide additional detail on our XLRP and achromatopsia clinical programs. Susan?

[Susan Schneider:]

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 As Sue mentioned, Dr. Paul Yang presented promising new data from the ongoing Phase 1-2 XLRP clinical trials, at the 14th International Symposium on retinal degeneration in September. The data he presented were from an analysis of macula structure in patients 12 months after receiving a single dose of our XLRP product candidate. A hallmark of XLRP is that the Ellipsoid Zone or EZ, which is a defined region within the photo receptor layer of the retina, degenerates over time and is eventually lost. The data Dr. Yang presented shows that intact retinal anatomy at baseline predicts potential for improvement. Of 20 patients evaluated in our XLRP Phase 1-2 clinical trials, 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 EC shows no structural or functional improvements post-treatment. The data also showed that there was a significant association between improvements in visual sensitivity measured by Maya and improvements in retinal health measured by improvement in EC in groups 4 to 6. Among these patients, 4 of 6 with Maya improvement also had EC improvement. And there was a statistically significant association between Maya improvement and EC improvement with a P value of 0.0212.12. 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 datasets from these trials in 2022, including 3-month Interim Skyline trial results in the first half of 2022, 24-month trial results from the ongoing Phase 1-2 clinical trial in the third quarter of 2022. Well, month Skyline trial results in the fourth quarter of 2022. And 6 months interim Vista trial results in the fourth quarter of 2022. Our clinical collaborator, Dr. Robert Sisk of Cincinnati Children's Hospital and University of Cincinnati College of Medicine, will also make an on-core presentation of the 12-month data from the Phase 1-2 trial at the American accounting of Ophthalmology Annual meeting taking place from November 12 to 15, 2021. We are 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 ACHM B3 patients and 5 pediatric ACHM A3 patients, and higher dose groups 5A and 6A, and identified a maximum tolerated dose. As we have previously reported, to address suspected unexpected serious adverse reaction for 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. 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 or lowest group for pediatric patients on which we previously reported. And we plan to continue development of our achromatopsia product candidate. We expect to report interim three-month data for the pediatric patients in both a chromatopsia trials before the end of 2021. For the B3 program, we're also developing an end of Phase 2 briefing package for submission to the FDA, and expect to receive feedback from the agency in the first half of 2022. The agency's input will play a foundational role in our plans for advancing the achromatopsia B3 candidate toward a pivotal trial. Now, I'll turn the call over to Jon for a review of our financial results.

[Jon Lieber:]

Thanks, Susan. For the first quarter of fiscal year 2022, we recorded a net loss of $17.1 million compared to a net loss of $15.4 million for the first quarter of 2021. The increase in net loss was primarily due to an approximate $700,000 increase in research and development expenses, an approximately $700,000 increase in general and administrative expenses, and an approximate $300 thousand increase in interest expense. We ended the first quarter of fiscal year 2022, with a strong balance sheet, including total cash and cash equivalents of $90.5 million. 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 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. Wainwright. Please proceed with your questions.

[Joe Pantginis:]

Hey, everybody. Good morning and thanks for taking the questions. So first, I just wanted to ask about or maybe a logistical question about the SUSAR events in the chromatopsia 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?

[Sue Washer:]

Morning Joe, thanks for your question. We really do believe that this is a dose effect in very young patients. Remember that these pediatric patients who are between the ages of 4 and 8 years old and we didn't see any of this kind of information in any teenagers, in any of the lower dose or any of the adults. So we really believe it's a dose effect in these very young children.

[Joe Pantginis:]

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?

[Sue Washer:]

That's a very good issue because I -- question, because 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 manufacturing facility. And even though we're breaking ground and then moving dirt around, we've actually already ordered the equipment that we're going to meet and 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.

[Joe Pantginis:]

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 drops in geographic atrophy and also visual acuity.

[Sue Washer:]

Yeah. I do think Joe that it's a bit too early. I can understand the interest there, but we're 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.

[Joe Pantginis:]

Totally understood. Thanks a lot for the comments Sue.

[Sue Washer:]

Have a great day.

[Operator:]

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

[Dae Gon Ha:]

Hi. Good morning. Thanks for taking our questions, and congrats on all the progress. Sue, maybe if I can just go back to the SUSAR question, understanding that it's probably more dose and patient - baseline age related. Can you I guess talk a little bit about AAV-TYF and its function, or is this something that's probably going to be broader to other stereotypes of AAV. Is there anything unique about AAV-TYF, 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, Dr. Yang data that you guys were just debriefing us on, very interesting, but I guess just thinking about development forward. I know previously you were thinking about mobility major and Skyline trying to draw correlations to the visual function data, making that as a potential regulatory endpoint. Where does come in given the correlation data that Dr. Yang presented? Have you guys already interacted with the FDA about perhaps, using that also as a supportive measure? Thanks.

[Sue Washer:]

Thanks Dae Gon for the questions. I'll take the SUSAR question and then probably give you a top line on the SUSAR versus maze and then give Susan an opportunity to weigh in on her thoughts there. As far as the capsid being -- differences in capsid being potentially more or less inflammatory, we have advantaged data and I think we've reviewed that -- reviewed it publicly in our corporate presentation, where we've looked at multiple capsid side-by-side. And when you dose sees multiple capsid side-by-side, manufacturers the same way, tighter 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 SUSAR has anything to do with the specific capsid. There are 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 EB -5 or EB -8 or other capsids that we've looked out over time. We don't see that as a causative effect, just total dose. Pys 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 Pys capsid with others, is that it does support a non-human primates about twice the expression of the intended protein as other capsids. So now moving onto Dr. Yang 's data, we do -- we have already written and talked to the FDA about our protocols were Skyline and Vista we do intend that visual sensitivity is the primary endpoint with the mobility maze as a secondary endpoint along with visual acuity. We will be collecting the easy line data. And so I'll turn it over to Susan for comments on its usefulness and supportive nature of supporting our product candidates.

[Susan Schneider:]

Thank you Sue. For Dr. Yang, 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 chance, the alignment with the functional changes is very important to be both clinically meaningful and hopefully clinically significant in outcomes. And again, this helps us differentiate our products from others and help us to meet the unmet need in these patients.

[Operator:]

Thank you. Our next questions come from the line of Yanan Zhu with Wells Fargo. Please proceed with your questions.

[Yanan Zhu:]

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 what about the cadence and whether you think that cadence can give you confidence for the now first quarter '22, 3-months Skyline readout, given that in the past, COVID impacted enrollment? And also, how do you prioritize the enrollment of Vista and Skyline given the potential bottleneck of COVID impact? Thank you.

[Sue Washer:]

Hey, 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 the numbers of -- 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 gone away. We are not having those issues. 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 patients. It's a Mobile Vision Center that travels directly to the patient's home to be able to test data. We now actually have two fully operational mobile vision centers and not only are they doing follow-up business in certain cases, they're also screening and pre -screening patients for us, and this is something that the patients in the sites are very appreciated about, because when the patients are actually having to travel to the sites, they already know would that they have been pre -screened in the past much of the inclusion-exclusion criteria. And so the site knows that its time is going to be well-spent with these patients. 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.

[Yanan Zhu:]

Great. Thanks for that very helpful update. And also another question on the competitive landscape front. I think a competitor reported last month data from intravitreally administered AAV gene therapy for XLRP. I know the dataset is pretty small and early. Could you share your thoughts on that dataset and how should we think about the competitive profiling of that chain therapy? Thank you.

[Sue Washer:]

Again, I 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 photo receptors. And our data is very strong and as we've talked about, we now see improvements in visual acuity, visual sensitivity, and patient anecdotes, clean safety profile, and then now the encouraging data from Dr. Yang.

[Yanan Zhu:]

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 1-2 study at AAO. What incremental data will we see a competitor with the last 12 months data readout? Thank you.

[Sue Washer:]

So Dr. Assist will not be presenting brand new data. It's really Susan described an on-core presentation on the data. But what he will be doing, is blinking the data in a way that hasn't been concentrated on before. really showing the visual sensitivity data in conjunction with Dr. Yang analysis as easy line. And also going through some surgical procedures and improvements that have been there. So it's really a way of Capstone presentation to kind of pull together all of the data that's been released over the last 18 months into one consolidated fashion.

[Yanan Zhu:]

Great. Thank you, Susan.

[Operator:]

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

[Zegbeh Jallah:]

Good morning. Thanks for taking my questions and congrats on the new hires. I have just 2 quick ones for me. The first one which is Dr. Yang is data. I think Susan noted intact not being productive, a response or aiding fast. And I was just wondering if your baseline perimeter threshold that you've said is actually going to be able to also capture that same patient populations.

[Sue Washer:]

Good morning and happy to have you on the call. And that's a good question because we've previously talked about how we altered the inclusion, exclusion criteria between the Phase 1 - 2 and the Skyline and Vista. And I can say that we were already thinking that there was a correlation here between that baseline Maya 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 Dr. Yang had.

[Zegbeh Jallah:]

Thanks and then the last, when you're just about the Scheme study and the SUSAR, which is wondering beyond treating those patients with steroid. Is more patient is still being enrolled into the study? How do you plan to proceed regarding those? And any thoughts on whether or not that SUSAR may have impacted or will impact enrollment into the study?

[Sue Washer:]

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

[Zegbeh Jallah:]

Thanks.

[Operator:]

Our next questions come from the line of Matthew Luchini with BMO Capital Markets. Please proceed with your questions.

[Matthew Luchini:]

Hi. Good morning. Thank you so much for taking the question. Maybe just to start to the bigger picture question. 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 try to understand how AGTC may be able to leverage whatever findings eventually result from that group. And then secondarily, and more specifically to the Company, wanted to ask, following the RSO data -- the presentation of XLRP data and RSO, one of the larger meetings where the state was 10%. 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 SUSAR patients are now fully off of steroids. Thank you, very much.

[Sue Washer:]

As far as the BGTC team, we think that this is an excellent program that has been started. And we are very much engaged in participating and in learning what they bring forward and will define -- 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 programs. And as I have mentioned before, going from Skyline and into Vista, we're expanding the number of sites dramatically and really have not had an issue with physicians being interested in the coming 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. And on the SUSAR in the achromatopsia patients, 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.

[Matthew Luchini:]

Okay. Thanks for taking.

[Operator:]

. Our next question has come from the line of Kristen Kluska with Cantor Fitzgerald, please proceed with your questions.

[Kristen Kluska:]

Hi. Good morning, everybody. Thanks for taking my question just a quick one here. As you really had the first back-to-back 6 months 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?

[Sue Washer:]

Thank you for the question, Kristen. I do believe that as meetings, return to a more normal cadence, and we have more opportunities to interact with people more broadly, that 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 has really we've been able to be very effective later during the pandemic times, but you're certainly right. 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 question, 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 2022. And we do think that this will increase our ability to get the word out on our programs.

[Kristen Kluska:]

Thank you. And then just in light of this recent data at the International Symposium, and really starting to fill all of the endpoints together and see some correlation, it seems, which is not necessarily unique to the Company, that 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 there are really getting diagnosed or tested early on where there's potential for greater benefit at that point?

[Sue Washer:]

As far as getting the word out, Christian, that's another good point that you're making. Now, we get the word out to physicians, but get the word out to patients. And that's where our long-standing commitment to the 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 stand. And we do a lot of work on education and outreach within this area. The foundation fighting blindness now has a registry called my retina, where they actually provide services to genotype patients and we're very supportive of that effort. And I think that this is the way the grass roots on connections to the different patient advocacy groups. 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.

[Kristen Kluska:]

Great. Thank you very much.

[Operator:]

Our next questions come from the line of Yun Zhong with BTIG, please proceed with your questions.

[Yun Zhong:]

Hi, good morning. Thank you very much for taking the question. So a follow-up question on the 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 eyes and how do they compare to treated eyes? And also, did you look at the patient at 12 months and maybe at 24 months. And I have a follow-up question, please.

[Sue Washer:]

Ian thanks for the question and the data that seeing analyses really very interesting. He was looking in the presentation given and the data that we're describing at the 12 months’ time points. 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 Susan, do you have any further comments to add?

[Susan Schneider:]

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 made earlier about treating earlier when we have treatments and 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.

[Yun Zhong:]

Okay. And I remember then, Nightstar previously reported virtually very little change in terms of structure in XLRP patient. But recently, another Company reported any -- 2 patients, but one probably a little over 10% decrease, and the other one probably around 25% decrease from baseline in untreated 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 then the variability in easy area, please?

[Sue Washer:]

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

[Yun Zhong:]

Okay. Last question, on the briefing document for the B3 program from a top seer, any changes in terms of what efficacy endpoints you would like to propose to the FDA?

[Sue Washer:]

No, we're still putting up briefing package together. And like my comments about the protocol for the dry AMD, 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 in the agreed upon protocol.

[Yun Zhong:]

Okay. 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.

[Sue Washer:]

Thank you. Our first quarter of fiscal '22 reflects the continued execution of our clinical trial strategies for our XLRP and achromatopsia 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 programs. We are proud of the quality, quantity, and diversity of data that we have shared with a medical scientific patient and investor communities for our XLRP and achromatopsia programs. And we're 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 controls facility are a key part of our strategy to advance the development of both XLRP and our chromatopsia programs as rapidly as possible towards potential approval and commercialization. We know that patients with these diseases have waited their whole lives for a therapy that could have make a meaningful difference. And we're committed to addressing this urgent needs. With the addition of John, Susan, and Sarah, our team has never been better positioned to prepare for the multiple opportunities that lie ahead. 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 the continued face of the patients who believe in our technology and our commitment to their cause. I thank all of them and I look forward to sharing our progress with them and with you in the months ahead.

[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.