Applied Genetic Technologies Corp (AGTC) 2021 Q3 法說會逐字稿

Good afternoon, and welcome to the AGTC Financial Results Conference Call for the Third Quarter of Fiscal Year 2021. 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 current and planned clinical trials. 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 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'd like to turn the call over to Sue Washer, Chief Executive Officer of AGTC. Please go ahead.

Good afternoon, and thank you all for joining us. With me on today's call are Stephen Potter, Chief Business Officer; Mark Shearman, Chief Scientific Officer; and Bill Sullivan, our Chief Financial Officer. During today's call, we'll review our recent accomplishments, and then we'll take your questions.

We've had several notable accomplishments since our last quarterly call in February, all of which enhance our vision of using our gene therapy expertise to develop groundbreaking therapies for people with rare diseases. Notably, in our X-linked retinitis pigmentosa, or XLRP program earlier this month, we reported new data from the ongoing Phase I/II XLRP clinical trial that further supports the best-in-class potential of our XLRP product candidate. The key takeaways are that we saw a 50% response rate for patients in Group 5 and 6, who meet the inclusion criteria for both of the Skyline and Vista trials at month 12, and we also saw a statistically significant difference with respect to visual acuity in treated compared to untreated eyes for patients in groups 2, 4, 5, and 6, at month 12 based on best corrected visual acuity or BCVA data. Finally, we demonstrated continued durability of response at month 24 in two of three patients from Group 4 available for evaluation at that time point.

Last week, we announced that we have initiated plans to lease a build-to-suit 21,000 square foot current Good Manufacturing Practices, or cGMP, manufacturing and quality control facility adjacent to our Florida location to prepare for late-stage development of our XLRP and achromatopsia programs as well as to support our preclinical pipeline. Build-out of this GMP facility, which will support up to 500-liter scale manufacturing is part of our strategy to enable more rapid filing of a biologics licensing application and commercial launch of our XLRP product candidate upon potential United States Food and Drug Administration approval. The GMP facility is also expected to support more rapid advancement of our entire product pipeline while providing supply chain redundancy and reducing manufacturing risk. Build-out of this facility is expected to be completed in the second half of 2022. Importantly, through a robust tenant improvement allowance, tiered rental rates, and our amended loan to Hercules, we have structured this new investment in such a way that it does not impact AGTC's cash runway in the near-term.

Last week, we also presented data at the ASGCT Annual Meeting that described improvements in the manufacturing process for our XLRP product candidate. These data further demonstrate that we have developed a robust, reproducible, scalable and highly productive AAV manufacturing process which will allow for a modest size facility to fulfill supply requirements through commercialization. Earlier this month at ARVO, Dr. Paul Gang, one of the investigators in our ongoing XLRP clinical trials gave a presentation of the data initially disclosed in November of last year. He concluded that our product candidate had a well-tolerated safety profile retained through month 12 for low dose groups and month 6 for high-dose groups and showed clinically meaningful improvements in retinal sensitivity for centrally treated patients. He also concluded that the BCVA data remains a supportive trend at both time points.

On Friday, Biogen reported data from their serious Phase II/III trial, a study of product candidate being investigated as a onetime gene therapy for patients with XLRP, in which they did not meet their primary endpoint. There are several key differences between their XLRP program and ours that we believe will ultimately result in our greater success in late-stage development.

First, we designed and exhaustively tested our product construct, both in nonhuman primates and naturally occurring dog models early in the development process to ensure that we selected the most appropriate components, which include a proprietary engineered capsid, and define the most appropriate dose range, allowing us to dose, we believe, at a higher level than our competitors in our human clinical programs. Second, our Phase I/II data shows a more robust safety profile over all doses and biological activity over a wider dose range, including a statistically significant improvement in BCVA. Third, we have taken a thoughtful and deliberative approach towards planning the next steps in XLRP clinical development.

Our Skyline and Vista trials will be analyzing activity over a wider area of the retina in patients with better baseline characteristics using a proprietary algorithm to define low side most likely to respond. And fourth, we have added a secondary endpoint using the aura visual navigation challenge, which is a mobility maze test similar to that used in the Luxturna approval.

In summary, we believe our deep scientific approach will provide the highest chance of success. We have done the early work. We have presented robust data. And we have appropriately designed our Skyline and Vista trials.

Turning to achromatopsia. Dr. Mark Penici, one of the investigators on our ongoing Phase I/II achromatopsia clinical trial gave an Encore presentation at ARVO of the data that we had reported from this trial in January of this year. The key takeaway from these data is that the visual sensitivity data support the positive patient-reported outcomes that we presented in 2020 and provide a path forward to collect additional data to realize fully the potential of our achromatopsia therapies.

We are currently in completing enrollment of pediatric patients, those between the ages of 4 and 8 years in the 2 highest dose groups in the achromatopsia B3 and achromatopsia A3 trial. And we will be following all patients through 12 months. In addition, we will be including both functional magnetic residence imaging of the brain and improved color brightness tests to assess improvements in visual function. We are hopeful that these changes, combined with longer follow-up times will add to the developing body of evidence and supportive anecdotal patient-reported outcomes. We expect to report 12-month data for all adult patients in both the achromatopsia studies in the second quarter of 2021 and to report 3-month data from the pediatric patients in the fourth quarter of this year. And we'll use these data sets to further inform the clinical development of our achromatopsia program.

I will now turn the call over to Steven for comments on exciting new initiatives in patient advocacy and business development.

Thank you, Sue. Consistent with our commitment to improving patients' lives, we have also launched 3 new initiatives to enhance the patient journey. With guidance from the global advocacy community on low vision, which included the Foundation Fighting Blindness, Canada, and Retina International, we improved our website to make it more accessible and welcoming. These enhancements to the website, which are intended to make information easier to find for patients with low vision include different font sizes and color options to make the pages easier to read and an options for the page to be read aloud.

Recognizing the increasing use of mobile devices by patients, these enhanced features are also designed for mobile compatibility. We also launched a new website that is dedicated to XLRP information and can be accessed at www.senictrials.com. The second patient-focused initiative was the launch of a dedicated patient information call center in partnership with Serva Health to provide support to patients with potential interest in our XLRP clinical trials. This nurse staffed patient engagement center is designed to assist patients in navigating in the clinical trial experience. This partnership provides access to nurses who can answer questions to help potential participants understand our XLRP clinical trial and as a potential patient is interested and qualified for one of our -- more of our trials, the call center will provide guidance and logistics support to navigate the process.

Third, to facilitate patient knowledge and to enhance the experience in participating in the AGTC trial, a series of visually accessible information and brochures were created directly for patients and site coordinators. These materials paid special attention to the use of easy to read font type, size and colors; and for the pediatric patients, a welcome book was created for -- with a fictional character who also has XLRP.

Along with the patient advisory committee that we launched in August 2020, these initiatives provide tangible evidence of our commitment to the patients we seek to serve. And our recognition that we can only achieve our mission if we work collaboratively to address issues that matter to patients.

On the corporate development front, we entered into 2 licensing agreements that allow our technologies and data to support continued development of novel therapies for inherited retinal diseases. As announced last month, we entered into a licensing agreement that provides our proprietary cone specific promoter technology to SparingVision SAS, a genomic medicines company developing vision saving treatments for ocular diseases. AGTC and SparingVision share a common mission to improve the health and vision of those patients suffering from inherited retinal disorders, and we are pleased to license access to our promoter to support development of their ophthalmology gene therapy pipeline and give them an important tool in their efforts to bring much needed therapies to patients.

Under the terms of the agreement, SparingVision received nonexclusive rights to our proprietary promoter for use and development of 2 noncompeting products with an opportunity to obtain rights to use the promoter for 1 additional product in the future. AGTC will receive an upfront payment and be eligible to receive milestone payments for successful clinical development and royalties on future sales on a per product basis.

Last month, we also announced a licensing agreement with TeamedOn International, under which we will provide TeamedOn with the clinical trial material, preclinical and clinical data generated for the development of our investigational intravitreal gene therapy candidate for the treatment of X-Linked retinoschisis or XLRS, which is an inherited disease that causes loss of vision due to the degeneration of the retina. Under the terms of the agreement, TeamedOn will conduct all activities required to reinitiate clinical development of the program, and we will be eligible to receive milestones and royalties based on clinical progress.

Although we discontinued our XLRS clinical program in 2018 because defined efficacy endpoints were not met using intravitreal injection, this licensed agreement reopens the possibility of a much needed treatment for individuals with this incurable disease by enabling TeamedOn to leverage our investment and previous work in our XLRPS program to bring potential benefit to patients. TeamedOn intends to take a subretinal injection approach to its XLRS program, which may overcome the limitations observed with intravitreal injection. Both licensing agreements allow us to contribute to the development of potentially transformative therapies for patients with ocular diseases and recognize the value of our technology, while also creating potential new revenue opportunities should our licensees be successful in their endeavors.

Finally, I'd like to let everyone know that we will be hosting an R&D Day on Thursday, July 22 from 10 a.m. to 1:00 p.m., and we will provide additional details in the coming weeks. It has been a busy few months at AGTC, and we are proud of all that we have accomplished thus far in 2021. The achievements we have just reviewed give us additional momentum as we move to advance our XLRP program towards commercialization and make continued progress in the clinical development of our achromatopsia candidates in our rich preclinical pipeline.

Thank you, Steven. Before Bill provides a review of the financials, I would like to thank him for his contributions to AGTC. As we announced earlier today, Bill has decided to pursue the same position at an early stage oncology company, one whose mission, he is very passionate about. We would not be in our current position of strength without Bill's vision and financial acumen that helped fund the company. On behalf of the company, I sincerely wish Bill continued success and thank him for his many contributions to AGTC.

With Bill's departures set for June 9, Jerry Reynold, currently our Vice President, Accounting, will serve as our Chief Accounting Officer and Treasurer, assuming certain key financial responsibilities for AGTC, including SEC reporting and corporate governance. A search for a new CFO is already underway.

I'll now turn the call over to Bill for a review of our financial results.

Thanks, Sue, and thank you for those kind words. I've certainly enjoyed my time at AGTC, working with you, working with Steven, Mark, and all my fellow colleagues at AGTC. I believe the future is bright for AGTC. And I look forward to following your future success. And again, thank you.

With that said, I'll now turn to our financial update. For the third quarter of fiscal year 2021, we recorded a net loss of $14.9 million compared to a net loss of $11.2 million for the third quarter of 2020. The increase in net loss was primarily due to a $2.7 million increase in R&D expenses, a $400,000 increase in G&A expenses and a $600,000 increase in other expenses. The $2.7 million increase in R&D expenses was primarily the result of increased external XLRP spending for planned manufacturing, clinical site preparation and other activities related to the Skyline and Vista trials, partially offset by decreased external achromatopsia spending.

The $400,000 increase in G&A expenses was primarily due to higher legal fees, partially offset by a reduction in employee-related expenses. Following the completion of our successful $75 million financing in February yielding net proceeds of approximately $69 million, we ended the third quarter of fiscal year 2021 with total cash, cash equivalents and investments of $111 million. We believe these funds in addition to the $10 million of proceeds that we recently received from an amended loan agreement with Hercules will be sufficient to allow AGTC to generate data from our ongoing planned clinical programs, build out our new cGMP manufacturing facility 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 Instructions) Our first question comes from Joe Pantginis with H.C. Wainwright.

And Bill, best of luck to you in your new endeavors. So first, I would just have a quick logistical question, and that's just I guess, XLRP patient availability as the competitive landscape just shrank a little bit, and there are other studies ongoing as well.

Well, thank you for that question, Joe. And I think it is a question that would be on the top of everyone's mind. What we have done, and Stephen really talked about this, it's really done a great deal of work reaching out to patient organizations.

And our partnership with Serva Health has really been wonderful. And we are really at the point where it's not patient identification and patient interest that is any kind of rate-limiting factor on our trials moving forward. So we're very proud of the efforts of patient advocacy, patient enrollment, our partnership with Serva Health that have really put us in a positive position.

No, that's helpful. And then my question is a little broader. It first starts with Biogen, but then links to your own initiatives. So I guess, obviously, we saw the clinical data around Biogen. We know some of the safety history, obviously, from their publications out of Nightstar. So maybe can you just discuss some of the safety differentiations, but I specifically want to link it to as you've said already in your prepared comments, your proprietary capsids, your manufacturing that you've had in place and your new manufacturing. And specifically also, what have you learned from others, your current manufacturing that you've had in place for quite some time now that you can apply to this new facility?

So I'm going to go ahead and ask Mark, our Chairman, who is on the line to address the differentiation of the product and the safety profile. And then I'll go ahead and follow-up with the manufacturing differentiation. Mark?

Yes. Thanks for the question, Joe. So the primary difference between our product and that of Biogen Nightstar is the capsid. And as you recall, we did a fairly detailed comparison of the ability of the different capsids, meaning our own Nightstar's well with Meira penetrate into primate photoreceptors and expressive transgene, showing that our capsid at about a 2 to 3-fold improvement in that ability. So a greater expression level. So I think that's important. But I think as Sue referenced in her comments, selecting the appropriate dose is key, both from a safety perspective as well as an efficacy perspective. And we did exhaustive studies in nonhuman primates as well as in the natural recurring dog model to really match efficacy with safety. And so that's allowed us in the clinic to potentially reach the maximum tolerated dose in a safe way and one which is controlled by concomitant use of steroids and allow us to determine and show activity at multiple dose, even at doses 5 and 6, which was the latest data we shared recently.

So I think that puts us in a great position to be able to really know what the most effective dose is and know and have confidence that that can be administered safely. And then on top of that, the improved manufacturing process allows us to have an enrichment of full to empty, so reduce the total capsid load as well as process residuals. So I think overall, lots of things in our favor in that regard.

And then, Joe, just a follow-up on the manufacturing facility and what we've learned. I think the big thing we've learned is that to have control of your timeline and have control of your manufacturing and quality control analysis is critically important. And without having some measure of internal manufacturing capabilities, it is very stressful on the organization. And so bringing that manufacturing and keeping in-house, as we're now moving into late-stage development we thought was in a very important strategic decision to make, and we're very excited about that prospect.

And I think what we've learned by watching others build facilities is that you need to be flexible. You need to have the ability to have many different products, work in your facility at different scales. As I said, this facility is -- has been designed. So we have up to 500-liter batches, but certainly, we can do smaller as well. And we've really been able to bring on board consultants and experts that have already built multiple facilities. So this is -- we're able to use others' expertise and kind of have been there done that set of people to help us be successful.

Our next question comes from Dae Gon Ha with Stifel.

And congrats on all the progress. Maybe I'll start with my first question on the XLRP side. The point of differentiation as part of the follow-up to the earlier question. And then second, maybe touch on the logistics and timeline for the manufacturing.

So Mark, if I heard it correct, and also, Sue, in your prepared remarks, you laid out the number of differentiation between your product and Nightstar Biogen. But I guess going to the point about BCVA improvement that you saw in your data, I wanted to kind of get your take on the GRK1 promoter that's currently in use in your construct.

Perhaps if you can talk a little bit more about what's specifically unique about this promoter? Why you selected it? And what gives you confidence as we go forward that not only are we getting the BCVA, but also whatever we saw on the visual sensitivity will turn out to be robust in the Skyline and Vista trials?

And then secondly, on the manufacturing side, in your press release and also in your prepared remarks, you mentioned it's going to be online or coming online in the second half of '22, which I guess would coincide with the 6-month interim data from your Visa trial. So once it does come online, can you walk us through what would need to be done in that new facility such that by the time you have the 12-month data from Vista that the filing would actually be done in a sort of concurrent manner and without any supplemental submission that would potentially lead to a delay, if you will. So any detail on that would be great.

Well, thank you, Dae Gon, for those -- digging a little bit deeper into the information and allowing us to expand on what it is we're doing and what it is we're planning. So Mark, I think that you can address both the statistically significant improvements we've seen in BCVA and attributes of the product that could contribute to that.

Yes. So I think your question was on the GRK1 promoter, but I'll also expand it to the cogent optimized transgene itself. So the GRK1 promoter in our hands outperformed another promoter, which was known in the literature to support expression in both rods and cones, and that was IRBP. But in our hands, the GRK1 promoter was superior, and we showed that in nonhuman primates or direct head-to-head comparison. The GRK1 promoter is known in the literature. There are versions of it. And ours includes the core promoter plus an additional regulatory element. So that may be a difference between ourselves and the competitors.

And then yes, each company has used -- well, 2 of the 3 companies have used cogent optimized RPGR, the third company may have GTx, GTx has used a truncated version. The 2 cogent optimized versions are somewhat similar, but I believe we are the only company that has really done in-depth characterization of the stability of our version of the cogent optimized RPGR. And that means both sequencing DNA as well as protein in vitro and in vivo as well as through the manufacturing process itself. So I think a combination of that sort of exhaustive scrutiny and testing of the construct gives us confidence that we've chosen the right dose and that the transgene is being effective.

And that seems to be playing out in the efficacy, both in terms of the perimeter, but also as you referenced, the BCVA. And so as of now, we have 12-month data for all dose groups showing a statistically significant difference between the treated eye and the untreated eye relative to baseline. So obviously, it's -- we have limited access to what Biogen Nightstar and Meira have in their clinical trials. But as far as we know, based on what's published, they're not claiming, using the sort of standard BCVA approach, any difference between the treated and untreated eyes.

And then just following up on the manufacturing timeline. Yes, you were correct that we're saying it will be online in the second half of '22. And by online, we mean that the facility will be validated, and we'll have done shake down runs. And what will need to be done at that point is true engineering runs and comparability studies before we could be ready to file the BLA. So we think the timing is really right on point to be able to have no delay going forward.

Our next question comes from Kristen Kluska with Cantor Fitzgerald.

The first one is on otology. In light of some of the recent findings you presented with your collaborator Otonomy, could you just discuss any synergies you see with some of the ophthalmology conditions for a gene therapy approach? And then on the flip side, how do you start to think about some of these gene therapies for congenital indications where the effects, in this case, the ability we hear could be completely apparent from the time of birth?

So Kristen, thanks for joining us today, and thanks for highlighting a program that we're really quite proud of and excited about in the relationship with Otonomy is so strong. And I'm sure you're picking up on the data that's just been presented at ASGCT. I'm going to move this to Mark to discuss the similarities that we saw in working in the ear and working in the eye. Mark?

Yes. So thanks for the question. Yes. So basically, there are a lot of similarities in terms of known cell types, the anatomy of the ear, the ability to introduce the virus in the appropriate place. But on top of that, we really applied the discipline and the tradecraft that we've developed in ophthalmology for otology. And by that, I mean, prior to the recent data announcement, we've, over the last several years spent time and effort in both rodents as well as nonhuman primates, identify the optimal capsid for expression and support sales.

So it's a novel capsid variant that we're using in this program. We're using the -- ubiquitous promoter, but that's coupled to a cogent optimized GJB2 transgene, which again, we've tested multiple variants and selected the best one. And so we spent time coming up with an optimized construct. And then our colleagues at Otonomy have in-house set up the appropriate animal models and demonstrated a very encouraging and promising data showing rescue of the phenotype. So we were really pleased with the data. We had some anticipation or expectation that this should work based on how we design the construct and the fact that we knew that we were able to express the transgene in the appropriate cell types, including in nonhuman primates.

And then, Kristen, just speaking to your question about the time of onset in some of these genetic diseases of hearing loss and it being apparent from birth, one of the reasons, and Mark can speak more to this, one of the reasons we chose GJB2 is, one, it's one of the most common causes of genetic onset hearing loss but also, there is a longer window of where you can potentially intervene and maintain hearing before you really have to go to that kind of end-stage solution of a cochlear implant. So Mark, I don't know if you have a few words to expand on that.

Yes. So that was a consideration. I think we feel that we can administer this product to, obviously, very young patients ahead of when the rescue surgery would be needed to be done for a cochlear implant, which would be prior to speech development. So there is a window of opportunity here where we feel that we can correct a deficit and give ourselves time to determine if that's happening before these children would have to be treated with a cochlear implant. That's not the case with all genetic mutations, particularly some of the ones that are manifesting hair cells, but GJB2 was one of them. And as Sue mentioned, this is also the most prevalent genetic mutations. So we feel that it will give us an advantage in terms of being able to select and access appropriate patients.

Our next question comes from Matthew Luchini with BMO Capital.

So I think first from me on achromatopsia. You talked in the past about the importance of getting the pediatric data to the program. And I'd appreciate if you could provide your latest perspective on the importance of the data from these patients, given sort of everything that you've seen from the other cohorts thus far? And kind of as the immediate follow-up to that, are you in a position to provide any preliminary color on when you think those missing -- remaining patients might be actually enrolled? And then I have one follow-up from there.

Thank you, Matt, for the question. As far as -- I'll start backwards. As far as enrollment, we are actively enrolling patients in both the achromatopsia B3 and A3 trials in that age group of between 4 and 8 years old. So that enrollment is ongoing, and we have not changed our guidance of having 3-month data by the end of the year. So so far, that's going well despite the pandemic, and we're hopeful that we can continue in that way. We do still feel it's important to get the pediatric data to have a fulsome understanding of what's going on and the interaction of activating cone cells and being able to get communication back to the visual cortex. And also because we added for the pediatric groups the additional test of functional MRI and the additional color brightness test. We are very encouraged, and that's why we released the data earlier this year. I'm very encouraged by what we're seeing in the Octopus perimetry, the static full field perimetry in the adult patients but we believe that the pediatric data might give us a more fulsome picture due to the neuroplasticity in younger patients.

Okay. Great. And then one sort of bigger question is, what is the company's latest position or view on partnering the lead programs? Is that something that's actively under consideration? Something that is sort of ongoing? Or do you feel perhaps you want to wait? I know there's been comments in the past that taking our licensing or partnering in some of the earlier programs. I would appreciate your perspective on the later one.

Yes. I think that we're in the same position, as we've guided before, especially with the recent financing and our ability to move forward and generate the data from the Skyline and Vista trials, I think we would, at this point, want to have that data in hand before entertaining any particular partnering offering. We are actively looking for partners for our preclinical programs, though. So guidance hasn't changed there.

(Operator Instructions) Our next question comes from Yanan Zhu with Wells Fargo.

So first, I wanted to follow-up on a earlier question regarding manufacturing. I think you mentioned that you look forward to conduct comparability study before you file the BLA. My question is, do you foresee a possibility that patient dosing might be required for the comparability study? And -- so that's my question on that on manufacturing.

And then I have a question on your -- the progress made with -- in terms of the pre-specification of loci in the primary endpoint analysis for the XLRP study. So could you characterize your current protocol, the one that you developed with a vendor? How many loci do you plan to prespecify?

And when we hear about the Skyline trials data at the year-end, 3 months data, would that data be in a form of reported under the prespecified loci method? And lastly, in terms of Skyline data, because you mentioned -- you're doing a masked analysis at month 3. Are we going to get the pooled response rate from Group 2 and Group 5 And is that going to be a 50% response rate that you've been looking forward to? Or could Group 2 have a lower response rate, therefore, the blended response rate might be lower?

Well, Yan, thanks for joining us today, and thanks for your questions. I'm going to address the comparability question on manufacturing. And then Mark, I'm going to have you talk about the prespecification algorithm and then how we're going to apply that to Skyline and how the data will be reported. So as far as the comparability's test, we are already conducting or have been conducting comparability between the process we use for the Phase I/II trial and the process that's being used in the pivotal trial.

And that comparability plan was discussed and reviewed with the FDA and did not require patient dosing. The comparability study we would have to do with the new facility, it will be the exact same process, but the pivotal material was made at a contact manufacturing with that process and the commercialization material and the engineering ones would be done in our new facility. So we're really only comparing the exact same process at different facilities. So while we cannot say for sure because this will depend on FDA interaction, we would not imagine that study (technical difficulty) patient dosing. And now I'll turn it to Mark on the question about prespecification.

So I think as you know, the prespecification exercise utilize the patient data from the Phase I/II dose escalation that's satisfied the criteria for inclusion, and basically, the algorithm that was developed allows us to select those loci, which are most likely to respond by the decibel delta that we set as a cutoff. And they also validated this approach by removing certain subsets of that data in rerunning the algorithm. But this is being applied and it has been applied to patients that are being enrolled in Skyline, correct?

At month 3, we'll have 12 patients worth of data. We will be blinded to that data. We'll just have Group A, Group B, whatever -- whether A is Group 2 or Group 5, we won't know. But it will allow us to determine the response rate in the groups now. We will be collecting all types of data. So all of the perimetry data from the central 68 various other analyses on that cluster secondary endpoints. We have not yet fully decided which data we will release or the format in which that data will be released, but we will be performing all of the analyses that we've committed to, including the prespecification exercise.

Just a quick -- thanks, Mark. Just a quick clarification. For the prespecification, have you decided whether you will prespecify 5 loci or more than 5 loci? And looking for the responding loci within that greater number?

It's a minimum of 5 to align with the FDA guidance, 7-decibel, 5 loci. It depends a little bit on the projections from the algorithm as to how many of the loci could change by 7-decibel based on the training set from the Phase I/II, but it will be a minimum of 5.

Our next question comes from Zegbeh Jallah with ROTH Capital Partners.

I think I just have questions about manufacturing. And the first is just to clarify that there are no concerns about manufacturing for the Skyline and the Vista study, the stuff you're putting in place now is really just to be prepared for commercializations and then further development as well?

Yes. So manufacturing -- thank you Zegbeh for the question. The manufacturing facility is being planned for the future. It's really a long-term strategic play in that it could -- as we talked about, produce the engineering runs and commercial material for XLRP. It could produce the phase -- pivotal phase material for achromatopsia. And certainly, we would be able to utilize it for our preclinical programs as they move into the clinic. But it is not intended to be available or be used for the Skyline and Vista trial.

And it certainly makes sense at this step because I think what you've shown so far is that the platform is well-validated and certainly unique relative to competitors. So I think it's exciting that you guys are now putting in place the manufacturing plants. I think for me, since you also mentioned that the manufacturers for the clinical or preclinical programs. Just wanted to get a better sense of where you are with some of those programs. And what you're thinking might be the next one out of the pipeline because we are kind of getting excited now to see what more it's coming up.

Yes. We haven't provided specific guidance yet, Zegbeh, on which programs we're prioritizing ahead of others or what the specific timelines are. As we move throughout the year and really complete our plans on achromatopsia and XLRP, we'll provide further guidance. But we're really excited about the Otonomy program. And as you saw from the data release, that's moving forward very well. And our CFH and Progranulin programs moving forward very well and excited about some of the technologies we're putting in play with the other programs. So we'll have more information as we work our way through the year.

At this time, there are no further questions, I'll turn the call back over to Sue Washer for closing remarks. Thank you.

Thank you. So we have accomplished a great deal already in 2021, and we're committed to translating this momentum into additional progress across all aspects of our business in the months ahead. The growing body of clinical data gives us confidence that our XLRP product candidate has best-in-class potential, and we are matching this progress with critical advances in our manufacturing capabilities as we chart a path toward commercializing a potentially transformative treatment for this disease.

We're also proud that we have identified licensing opportunities that will allow our technologies to support other organizations that share our commitment to improving outcomes for patients in their efforts to develop ocular disease therapies. We have multiple data readouts in our XLRP and achromatopsia clinical programs expected later in 2021 and in 2022, that we believe will further solidify our position as a leading ocular gene therapy company, support our efforts to commercialize transformative therapies, and provide additional opportunities for value creation.

Our continued progress is only possible with the ongoing support of the patients and physicians who participate in our clinical trials. And as I always do, I express our profound appreciation for their willingness to take this journey with us. I hope all of you stay safe and healthy as we emerge from the pandemic and return to more normal routine, and I look forward to updating you on our achievements in the months ahead. Thank you again for joining us today.

Thank you. This concludes today's conference. All parties may disconnect. Have a great evening.