Q3 2021 Applied Genetic Technologies Corp Earnings Call

Good afternoon, and welcome to the a G T C 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 every.

One that during this conference call a G. T C 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 and these forward looking statements.

Due to a number of important factors, including uncertainty inherent and the clinical development and regulatory process, the extent and duration of the impact of the COVID-19 pandemic and other risks described and the risk factors section of a G. T. Six most recently filed annual report on form 10-K.

And the other periodic reports filed with the SEC.

A G T C undertakes no obligation to update any forward looking statements. After the date of this call.

For them to drive introductions and opening remarks, I'd like to turn the call over to Sue washer, Chief Executive Officer of a G. T. C. Please go ahead.

Good afternoon, and thank you all for joining US with me on today's call are Steve and fodder, Chief Business Officer, Mark Chairman and 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 ground breaking therapies for people with rare diseases.

Other play and our X linked retinitis Pigmentosa or XRP program earlier. This month, we reported new data from the ongoing phase one two axle RP clinical trial that further support the best in class potential of our XR P product candidates.

The key takeaways are that we saw a 50% response rate for patients and group five and six who meet the inclusion criteria for both and that Skyline and Vista trial at month 12, and we also saw a statistically significant difference with respect to visual acuity and treated compared to untreated eyes for patients and grow.

Two for five and six at month 12 based on best corrected visual acuity or B C. D E data for.

Finally, we demonstrated continued durability of response at month 24.

Great for available for valuation that time point.

Last week, we announced that we have initiated plans to lease a build to suit 21000 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 actual ERP and a chromatopsia probes.

Ram 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 actual our P 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.

Buildout of this facility is expected to be completed and the second half of 2020 to.

Importantly, two of robust tenant improvement allowance tiered rental rate and our amended law and that Hercules. We have structured this new investment and such a way that it does not impact a G. T six cash runway in the near term.

Last week, we also presented data at the a S. D C T and you won't meaning that that describe improvements and the manufacturing process for our excellent peak product candidate and these data further demonstrate that we have developed a robust and reproducible scalable and highly productive and you'd be manufacturing process, which will allow.

Now for a modest sized facility to fulfill supply requirements through commercialization.

Earlier this month at ARVO, Dr. Paul Gang, one of the investigators and our ongoing excellent clinical trial gave me 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 six.

And for high dose groups and showed clinically meaningful improvements and retinal sensitivity for essentially treated patient. He also concluded that the B C V. A data remains supportive trend at both time points.

On Friday, Biogen reported data from their serious phase two three trial a study of product candidate being investigated as a one time gene therapy for patients with XR P and which they did not meet their primary endpoint.

There are several key differences between their XR probe XRP program and hours that we believe will ultimately result, and our greater success and late stage development for.

First we designed and exhaustively tested our product construct both in non human primate and naturally occurring dog model early and the development process to ensure that we selected the most appropriate components, which include a proprietary engineered capsid and define the most appropriate.

And it does range, allowing us to dose we believe at a higher level than our competitors in our human clinical programs.

And our phase one two data shows a more robust safety profile over all doses and biological activity over a wider dose range, including a statistically significant improvement and P. C. P. A.

Third we have taking a thoughtful and deliberative approach towards planning the next steps and XRP clinical development, our skyline and Vista trials won't be analyzing activity over a wider area of the retina and patients with better baseline characteristics using a proprietary algorithm to define low side.

Most likely to respond and force we have added a secondary endpoint using the Ora visual navigation challenge, which is a mobility maze test similar to that used and the external approval in summary, we believe our deep scientific approach will provide the highest chance for success we have done.

And early work, we have presented robust data and we have appropriately designed our skyline and Vista trial.

Turning to our Chromatopsia Dr. Mark for me see what are the investigators on our ongoing phase one two of Chromatopsia 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.

George the positive patient reported outcomes that we presented in 'twenty, and 'twenty and providing a path forward to collect additional data to realize fully the potential of our chromatopsia therapies.

We are currently and completing enrollment of pediatric patients those between the ages of four and eight years and the two highest dose groups and the chromatopsia be free and a chromatopsia ace III trials and we will be following all patients through 12 months. In addition, we will be including both functional magnetic rather.

And imaging of the brain and improved color brightness tests to assess improvements and visual function. We are hopeful that these changes combined with longer follow up time will add to the developing body of evidence and support of anecdotal patient reported outcomes.

We expect to report 12 month data for all adult patients and both are Chromatopsia studies and the second quarter of 2021 and to report three month data from the pediatric patients and the fourth quarter of this year and we will use these datasets to further and form the clinical development of our Chromatopsia program.

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

Thank you Sue.

And with our commitment to improving patients' lives. We have also launched three new initiatives to enhance the patient journey.

With guidance from our global advocacy community on low vision, which included the foundation fighting blindness.

Oh I didn't line is Canada, and retina International we improved our website to make them more accessible and welcoming.

And then enhancements for the website, which are intended to make information easier to find for patients with low vision and clear.

And different font sizes and color options to make the pages easier to read and and options for the page to be Red line.

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 XRP information and can be accessed at www dot seen a trial and dot com.

The second patient focused initiative was the launch of a dedicated patient information and call Center and partnership with server health to provide support to patients with potential interest and our XRP clinical trials. This nurse staff and patient engagement center is designed to assist patients and navigating the clinical trial experience.

This partnership provides access to nurses, who can answer questions to help potential participants understand our XRP clinical trial and.

The potential patient is interested and qualified for one or more of our trials for call Center will provide guidance and logistics support to navigate the process.

Third to facilitate patient knowledge and to enhance the experience and participating and the agencies two trial for <unk>.

Series, a visually accessible information and brochures for credit directly for patients and site coordinators.

These materials paid special attention for the use of easy to read from types and sizes and colors and for the pediatric patients are welcome book was created for with a fictional character who is also has a therapy.

Along with the patient Advisory Committee that we launched from August 2020. These initiatives provide tangible evidence of our commitment to the patients we seek to serve and.

And our recognition that we can only achieve our mission if we work collaboratively to address issues that matter to patients.

From a corporate development front, we entered into two licensing agreements that allow our technology and data to support continued development of novel therapies for inherited retinal diseases.

And just last month, we entered into a licensing agreement for <unk>.

<unk>, our proprietary cone specific promoter technology to sparing vision and say, yes or.

For a genomic medicines company developing vision and saving treatments for ocular disease and <unk>.

T C and sparing vision share it come.

And to improve the health and vision of those patients suffering from inherited retinal disorders and.

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 and their efforts to bring much needed therapies to patients.

Under the terms of the agreement sparing vision received non exclusive rights to our proprietary promoter for use and development of two non competing products with.

And with an option to obtain rights to use the promoter for one additional product and the future.

Did you say you will receive an upfront payment and would 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 teamed on international under which we will provide team Don with for clinical trial material preclinical and clinical data generated for the development of our investigational Intraventricular gene therapy candidate for the treatment of excellent retinoschisis or X or X <unk>.

Which is an inherited disease that causes loss of vision due to teach generation of the retina.

Under the terms of the agreement between Dawn will conduct all activities required to Reinitiate clinical development and other program and we will be eligible to receive milestones and royalties based on clinical progress.

Although we discontinued our <unk> clinical program and 2019, because defined efficacy endpoints were not met and using a traditional injection.

License agreement reopens, the possibility of a much needed treatment for individuals with us and curable disease by enabling <unk> to leverage our investment and previous work and our exploration program to bring potential benefit to patients.

And I'm Dawn and tends to take a sub retinal injection approach towards our explorer program, which may overcome the limitations observed with interventional injection.

Both licensing agreements allow us to contribute to the development of a potentially transformative therapies for patients for ocular diseases and recognize the value of our technology, while also creating potential new revenue opportunities for our licensees and be successful and their endeavors.

Finally, I'd like to let everyone know that we will be hosting an R&D day on Thursday July 20, Stockton from 10, a M to one P M and we.

We will provide additional details in the coming weeks.

It has been a busy few months and energy T. C. And we are proud of all that we've accomplished thus far in 2020 one day.

And for your just reviewed give us additional momentum as we move to advance our XRP program towards commercialization and.

And our continued progress and the clinical development for Chromatopsia candidates and a rich preclinical pipeline.

Thank you Steven but for Bill provides a review of the financials I would like to thank him for his contributions to HTC and we announced earlier today Bill has decided to pursue the same position and and early stage oncology company, one whose mission he's very passionate about.

We would not be and our current position of strength without Bill's vision and financial acumen and that helped fund the company on behalf of the company I sincerely wish Bill continued success and thank him for his many contributions to a G T C.

With Bill's departure set for June nine Jerry Reynolds currently our Vice President accounting will serve as our Chief accounting officer, and Treasurer, assuming certain key financial responsibilities for a G T C, including SEC reporting and corporate governance and 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 and E. G. T C working with you and working with Steve and Mark and all my fellow colleagues for a G. T. C. I believe the future is bright for a G. T C and I look forward to following your future success.

Yes, and again and 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 and net loss was primarily due to a $2 7 million increase and R&D expenses.

A 400000 increase and G&A expenses and a.

600000 increase and other expenses.

The $2 7 million increase and R&D expenses was primarily the result of increased external XRP 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 400000 increase and G&A expenses was primarily due to higher legal fees, partially offset by a reduction and 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 and addition to the 10 million of proceeds that we recently received from our amended loan agreement with Hercules will be sufficient to allow E. G. T C to generate data from our ongoing and planned clinical programs.

Build out our new C. GMP 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.

Thank you.

And ladies and gentlemen at this time, we will be conducting our question and answer session.

If you would like to ask a question. Please press star one on your telephone keypad, a confirmation tone will indicate that your line is and the question queue you.

You May press the Star key followed by the number two if you would like to remove your question from the queue for.

For participants using speaker equipment and may be necessary to pick up your handset before pressing the star keys and once again to ask a question press star one on your telephone keypad.

Our first question comes from Joe and Janus with H C. Wainwright. Please state your question.

Hey, everyone and good afternoon, and thanks for taking the question and Bill Best of luck to you and your new endeavors. So first of all I would just have a quick logistical question and that's just I.

I guess XL RP patient availability.

As the competitive landscape you know just shrank a little bit and there are other studies ongoing as well.

Well. Thank you for that question, Joe and and I think it is a question that would be on top of that of everyone's mind, what we have done it and Stephen really talked about this it's really done a great deal for reaching out to patient organizations and our partnership with serve our 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 and our partnership with share but helps that it really.

Put us in a positive position.

That's helpful. Thanks, and then my question is a little broader it first starts with Biogen, but then linked but then links to your own initiatives. So I guess you know obviously, we saw the clinical data around Biogen and we know some of the safety history, obviously from their publications out of night Star. So maybe can you just discuss some of the safety.

<unk>, but I, specifically want to link it to you know as you said already in your prepared comments you know your proprietary capsid 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 Gonna go ahead, and ask Mark share a man who is on the line to address the differentiation of product and the safety profile and then I'll go ahead and follow up with the manufacturing differentiation smart.

Yeah. Thanks for the question Joe So you know.

The primary.

Primary difference between our projects and net of Biogen Nice stories, the capsid and that's it.

Recall, we did a fairly detailed comparison of the ability of the different capex, it's meeting our own <unk> mirror chip penetrate into primate photoreceptors and express to try and machine going there.

Capture other bedroom two to three fold improvement and that ability. So a greater expression levels. 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 and efficacy perspective, and weighted exhaustive studies in nonhuman primates as well as and the naturally occurring dog model to really match efficacy efficacy safety and so that's allowed us in the clinic.

Two centuries reached the maximum cash.

Tolerated dose in a safe way and one which is controlled by concomitant use of steroids and allow us to.

Good for them and and show activity and multiple doses even at.

Joseph five and six which was the latest data we shared recently, so I think that puts us and a great.

Physician 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 and enrichment of full to empty. So we used to total capex at load and.

As well as process residual so I think overall lots of things and all favor and that broke out.

And then just a follow up on the manufacturing facility and and what we've learned I think the big thing. We've learned is that to have control of your time line and have control of your manufacturing and quality control analysis is critically important and.

And without having some measure of internal manufacturing capabilities and it is very stressful and the organization and so bringing that manufacturing and QC and house Ashwin is now moving into late stage development. We thought was 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 you need to be flexible you you need to have the ability to.

And have many different products.

Work and your facility at different scales and as I said. This facility is had been designed and have up to 500 leader and 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.

This is we're able to use other expertise and kind of been there done that.

And people to help us be successful.

Got it thank you very much.

Yeah.

Our next question comes from Dae Gon Ha with Stifel. Please state your question.

Great Good afternoon, and thanks for taking our questions and congrats on all the progress maybe I'll start with my first question on the FRP side, the point of differentiation as part of the follow up to the earlier question and then second and maybe touch on the logistics and time line for the manufacturing.

And Mark if I heard it correct and also sue in your prepared remarks, you'd laid out the number of a differentiation between your product and nice start Biogen, but I guess going to the point about B C. V. A improvement that you saw and your data and I wanted to kind of get your take on the G. R. K one promoter Thats currently in use.

And your cost structure, 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 abusive yea, but also whatever we saw on the visual sensitivity will turn out to be a robust and the skyline and Vista trial.

And then secondly on the manufacturing side in your press release and also in your prepared remarks, you mentioned, it's gonna be online or coming on line and the second half of 'twenty, two which I guess would coincide with a six month interim data from your Vista trial. So once it does come on line can you walk us through.

And I would need to be done and that new facilities, such that by the time and you have the 12 month data from Vista.

The filing would actually be done and 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. Thank you.

Well, thank you dig out and so there are digging a little bit deeper into it and information and allowing us to expand on what it is we're doing and what and as we're planning. So mark I think that you can address both the statistically significant improvements, we've seen and B C D E and F and attributes and the product that could contribute.

To that.

Yeah. So I think your question was on the CL K, one promoted but I'll also expanded to the coast and optimized currency and itself. So the G. L K, one promoter and our hands.

Outperformed another promotion, which was known and the literature to support expression and both rods and cones and that with IPP and they're now handset glk, one promoted with superior and we showed that.

In non human primates for direct comparison.

The GSK one merger is known and the literature. There are versions of it and I always includes the co promote depressed and additional regulatory elements.

And that maybe a difference between ourselves and the competitiveness.

And then yes, each company has used well two of the three companies have used coach and optimize that free Jarrod complementary TX <unk> executed a truncated version.

The truth is the two codes and optimized version.

And what similar but I believe we are the only company that has really done in depth characterization of the stability of our version of the Coke and optimize all pgi and that.

It means both sequencing DNA as well and protein and.

And in vitro and in vivo as well as through the <unk>.

Manufacturing process itself. So I think a combination of that sort of exhaustive scrutiny and testing the fee construct.

And im confidence that.

And we've chosen the right dose and that the transgene and is being effective and that seems to be playing out from your efficacy both intense for the perimetry, but also as you referenced the PCB area and so as of now we have 12 months data for.

All dose groups showing interest.

Statistically significant difference between the treated eye and the untreated eye relative to baseline.

Obviously, we have limited access to walk Biogen and night that bike and nice star and mirror has.

And that clinical trials, but as far as we know based on what's published then.

<unk> using.

The standard piece and the EIA approach any difference between the treated and untreated eyes.

And then just following up on the manufacturing time line, yes, you're you're you're correct that we're saying it'll be on line in the second half of 'twenty, two and buy online. We mean that that facility will be validate it and will have done shake down run 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 and we think the timing and it's really right on point to be able to have no delay going forward.

Great. Thanks for taking my questions.

Our next question comes from Christian and <unk> with Cantor Fitzgerald. Please state your question.

Good afternoon, and thanks for taking my question. The first one is on the otology and in light of some of the recent findings you're presented with your collaborator autonomy could you discuss any synergies you see with some of the ophthalmology condition 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 and this case the ability to hear could be completely apparent from the time of first.

So Christian and thanks for joining us today, and and and thanks for highlighting a program that we're really quite proud of and and excited about and the relationship with the autonomy and is so strong and I'm sure you're picking up on the data the best and presented.

Presented as a S D C T and I'm going to move this to mark to discuss the similarities that we saw and working in the ear and working and the in the eye.

Mark.

Yes, so thanks for the question.

Yeah. So basically there are a lot of similarities in terms of net.

Cell types, the anatomy of the ear the ability to introduce the virus for the appropriate place, but on top of that we really apply the discipline.

Discipline and the trade craft that we've developed and ophthalmology for otology and by that I mean.

Prior to the.

And the recent data announcement, we've over the last several years.

And time and effort in rodents.

Road, and says well non human primates identify the optimal capsid for expression and support sounds like a novel capsid variants that we're using and this program.

We're using the <unk>.

And with his promoted back that's coupled to a coke and optimized for exactly to try and see which again.

We tested multiple variants and selected the best work and so we spent time.

Coming up with and optimized construct and then our colleagues at autonomy have in house that are the appropriate animal models and demonstrated the very encouraging and promising data showing rescue the phenotype. So we were really pleased with the data.

And it had some anticipation or expectation that it should work based on when and how we design the construct and the fact that we knew that we were able to express to try and scheme.

And your coke and cell types, including in non human primates.

And then Christian just speaking to your question about the time of onset in some of these genetic diseases of hearing walnuts and it being a parent from birth, one of the reasons and and Mark and speak more to this one other reasons. We chose G. J P. Two is one and 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 and expand on that.

Yeah. So that was a consideration I think we feel that we can administer this products to these very young.

Patients ahead of when the rescue surgeries would be needed to be done for a coker and plan, which would be net.

No.

And why it just speaks development. So there is a window of opportunity here, where we feel that we can cure.

Correct.

The deficit and give ourselves time to determine if that's.

Happening before these children would have to be treated as a cookie impact that's not the case with all genetics.

Mutations, particularly some other ones that are manifest and hair cells, but you gave me two was one of them and as Sue mentioned. This is also the most prevalent.

And that mutation and so we feel that it will give us an advantage in terms of being able to select and access appropriate patients.

Great. Thank you.

Our next question comes from Matthew Luchini with BMO capital. Please state your question.

Hi, good afternoon, everyone and thanks for taking my question.

So I think first for me on achromatopsia.

You talked in the past about the importance of getting the pediatric data to the program and I would appreciate it if you could provide me and 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 an immediate follow up for that or are you and are positioned to provide any preliminary color on why do you think goes missing kudos remaining patients might be actually enroll and then I have one follow up from there.

Yeah.

Thank you Matt for the question as far as I'll start backwards as far as enrollment we are actively enrolling patients and both the achromatopsia and be three and eight three trials in that age group of the two.

And for end and eight years old so that enrollment is ongoing and we have not changed our guidance for having three months data by the end of the year. So so far that that's going well, despite the pandemic and and we're hopeful that we can continue and that way.

We do still feel it's important to get the pediatric data to have to have a fulsome understanding of what's going on and the interaction of activating.

Cone cells, and being able to get communication and back to the visual cortex and also because we added credit pediatric groups. The additional types of functional MRI and and the additional color brightness test we are very encourage and and that's why we released the data earlier. This year are very encouraged by what we're seeing.

And the octopus parameter and the static for field Perimetry and the adult patients, but we believe that the pediatric data might give us a more fulsome picture.

Due to the neuroplasticity and younger patients.

Okay, great. Thanks, and then.

One sort.

Sort of bigger and the question is.

What is your company and latest position or view on partnering the lead programs.

And that's something that's actively under consideration and something that is sort of ongoing or do you feel perhaps you want to wait and other some comments from the past.

Taking all licensing or partnering from the earlier programs, but I appreciate the perspective on the later ones. Thanks.

Yeah, I think that we're in the same position that we've got and before you know, especially with the recent financing and our.

Our ability to move forward and generate the data from the Skyline and Vista trial, I think we would at this point well want to have that data in hand before entertaining any particular partnering offering.

We are actively looking for for our partners for our preclinical programs, though so guidance hasn't changed there.

Okay, great. Thank you for taking the questions.

And just a reminder to ask a question at this time press star one on your telephone keypad true.

Remove yourself from the queue press star followed by the number too.

Our next question comes from Jan on Schuh with Wells Fargo. Please state your question.

Hi, Thanks for taking my questions. So first of all I wanted to follow up on a earlier question regarding manufacturing and I.

I think you mentioned that you will look forward to conduct a comparator study.

Before you file the BLA.

And my question is do you foresee a possibility that Ah patient dosing might be required for the comparability study.

And so that that's and my question on that and manufacturing and then I have a question on the.

The progress <unk> made with in terms of the pre specification flow side in the primary endpoint analysis for the XL RP study. So could you characterize your current.

Protocol and the one that you've developed with a vendor.

How many loci are do you do you plan to pre specify and.

And when we hear about the Sky line trials data adhere and three month data would that day it'll be in a form of a you know.

And reported.

Under the pre specified and low say Ah method.

And lastly in terms of the Skyline and data because you mentioned is you had mentioned youre doing them masks for analysis at month three are we going to get to the Pud response rate from group II and group five and is that going to be.

A 50% response rate that you've been looking for it to or could a group to have a lower response rate and therefore, the blended response rate and might be lower thank you.

Well, yeah, and thanks for joining us today and and thanks for your questions I'm going to address the.

The comparability question and manufacturing and then Mark I'm going to have you.

Talk about the pre specified certification algorithm and then how we're going to apply that to skyline and how the data will be reported.

So it's for US the comparability for test we are already conducting or have been conducting comparability between the process. We used for the phase one and two trial and the process that's being used in the pivotal trial and.

A 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 and a contract manufacturing with that process and the commercialization of material and the engineering ones would be done and our new facility. So we're really only comparing the exact same process at different facilities. So while we cannot say for sure.

This will depend on FDA interaction, we would not imagine that studies.

And dosing and and now I'll turn it on.

And the question about free specification.

Yeah.

So I think as you know the three specification exercise utilize the patient data from the phase one two dose escalation that satisfies the criteria for inclusion and basically the algorithm that was developed and allows us to select those low side.

And <unk>, which are most likely to respond and by the decibel Delta them we.

We set as a cut off and they also validated this approach by removing certain subsets of that data and what are you running the algorithm, but this is being applied and has been applied to patients that are being enrolled in skyline.

Correct.

At month, three we'll have 12 patients worth of data, we will be blinded to that data. We will just have group a group b whatever whether as group two and group five we won't know, but it will allow us to determine the response rates in the groups now we will be collecting all types.

The data.

So all of the preliminary data from the central 68 for various other analyses on that cluster of secondary endpoints, we have not yet fully decided.

Which day, so we will release all the format and wish that data will be released but we will be performing all the other day analyses that we've committed to including the pre specification exercise.

Just a quick thanks, Mark and thanks to just a quick clarification for the pre specification.

Have you decided and whether you will push the basis of five and low site or a more than five a little site and looking for the.

And then responding lewisite within that a greater number.

A minimum of five to align with the FDA guidance seven decibel five loci.

It depends a little bit on the.

Projections from the algorithm is too high and many of the low side.

Could change by seven Decibel based on the training set from the phase one two but it'll be a minimum of five.

Got it great. Thank you.

Our next question comes from Zach both Gela with Roth Capital Partners. Please state your question.

Hi, Thanks for taking my questions and I'll, just have them questions about manufacturing and.

Firstly, just to clarify that there are no concerns about them.

And for the I spy and the Vista study you know the steps youre, putting place and that is really just to be prepared for commercialization for and then further development as long.

Yes, I mean and in fact, making segment for the question and the manufacturing facility is being planned for the future and it's really a long term strategic play and and that it could and we talked about produce day engineering runs and commercial material for excellent P. It could produce the pain a pivotal.

Things material for our Chromatopsia and certainly we would be able to utilize it for our preclinical programs as they move into the clinic.

But it is not it is not intended to be available or be used for b, a skyline and Vista trial.

Thanks, and it certainly makes sense and does that because I think what you've shown so far is that you know the problem as well and validate and certainly unique relative to competitors. So I think it's exciting that you guys and how putting place the manufacturing plants I think for me since he also mentioned that you know the manufacturers or for others.

Clinical or preclinical programs just wanted to get a better sense of where you all have some of those programs and what you're thinking might be the next one you know out of that out of the pipeline. Because we are kind of getting excited now to see you know what what more is coming up.

Yeah, we haven't provided specific guidance yet.

And on which programs, we're prioritizing ahead of others or what the specific time line bar as we as we move through the year and and really complete our plans on.

Chromatopsia and and and excellent P will provide further guidance, but we're really excited about the autonomy program and as you saw from the data really and that's moving forward very well and you know our C. S H and pro granular and programs moving forward very well and an exciting.

And about some of the technologies, we're putting in play with the other program. So we'll have more information as we work our way through the year.

Thanks, and congrats again on all the problem.

Thank you exactly.

Arndt.

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

Thank you.

So we have accomplished a great deal already in 2020, one and we're committed to translating this momentum into additional progress across all aspects of our business and the months ahead. The growing body of clinical data gives us confidence that our XR P product candidate has best in class potential and we are matching this for.

And our grass with critical advances and our manufacturing capabilities as we chart a path toward commercializing and 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.

And for patients and their efforts to develop ocular disease therapies, we have multiple data readouts and our XRP and Ah Chromatopsia clinical programs expected later in 2020, one and in 2020 two that we believe will further solidify our position as a leading ocular gene therapy company support our efforts.

And commercialized transformative therapies and provide additional opportunities for value creation.

Our continued progress is only possible with and I'm going to support the patients and physicians, who participate in our clinical trials and as I always do I price 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 returned to more normal route.

<unk> and I look forward to updating you on our achievements in the months ahead. Thank you again for joining us today.

Thank you for this concludes today's conference all parties may disconnect have a great evening.