Q4 2020 Applied Genetic Technologies Corp Earnings Call
[music].
Good morning.
Welcome to the AGTC fourth quarter fiscal year 2020 financial results Conference call.
Today's call is being recorded before you get started I'd like to remind everyone that during this conference call AGTC may make forward looking statements.
Critics statements about the Companys financial results financial guidance, its future business strategies in operations.
And its product development regulatory progress, including statements about its planned phase two three clinical trial.
Actual results could differ materially from those disgusted. These forward looking statements due to a number of important factors, including uncertainty in her in the clinical development and regulatory process. The extend duration of the impact to the coping I'd seen pandemic and other risks described and risk factor section that Agtcs. Most recently filed annual report.
Form 10-K, and other periodic reports filed with the FCC.
AGTC undertakes no obligations to update any forward looking statements. After the date of this call.
For introductions are opening remarks, Oh, it's a triple over to Sue washer.
<unk> Chief Executive officer of AGTC.
Washer. Please go ahead.
Good morning, and thank you all for joining US with me on today's call our Bill Sullivan, Our Chief Financial Officer, and Mark Sherman, Our Chief Scientific Officer.
Today, we will begin by providing a management update on our plans for X linked retinitis pigmentosa or actual RP clinical program and a brief overview of our other activities.
Next Mark will review, our clinical programs with additional details on our Exlar P. phase two three plan based on that the feedback as well as newco limit every data for the higher dose group five from the ongoing phase one two trial review, our preclinical pipeline and then Bill will review, our fourth quarter and fiscal year end 2025.
Actual results, we will then take your questions.
I would like to begin today's call by noting that we have achieved several critical milestones as we announced in July the after they provide a comprehensive written feedback on our end of phase two submission regarding the protocol design and conduct our next trial to support future regulatory submissions related to our clinical program and pace.
Since with EXL or peak due to mutations in the RPG arching, we continue to move forward as planned with manufacturing clinical site preparation and other activities to enable initiation of a phase two study in the first quarter of 2021.
Mark will provide additional information about the expected design of the pace due to greet trial a bit later in the call.
In parallel we plan to expand the ongoing phase one two trial in order to dose approximately 12 patients into randomized to mass dosing arms to collect additional data, including a functional mobility course added as a supplemental endpoint.
Type does not do mobility course was used to support after FDA approval a block stern. The first gene therapy for an inherited retinal disease, we have already begun prescreening additional patients and expect to begin enrolling in the fourth quarter of 2020.
Mark will also provide details on new preliminary data so the higher dose group size in the ongoing phase one two dose escalation trial, which shows a favorable safety profile in line with the safety profile previously reported in this trial as well as a consistent responder rate when analyzing visual sensitivity.
Given this encouraging data. We currently plan to include the higher dose used in the group five as one of two dose levels, we move forward in our clinical development.
In addition to these exciting developments, we initiated activities designed to support the successful clinical development of both our actual RP enter chromatography, a gene therapy candidate with the launch of a nationwide mobile vision testing program for patients and the ongoing phase one two trials. We're also excited to have formally.
Launched a patient advisory council that will initially focused on ensuring that patient and caregiver voices are incorporated into the Xcellair P. clinical program.
Patient Advisory Council includes advocates on the inherited retinal disease Global community and will also help to inform development of our ongoing up from the top see a clinical programs and programs and our preclinical pipeline.
We remain on track to report data from the two highest dose groups that the ongoing exlar p. clinical trial and our two ongoing phase one two trials in patients with the chroma tops yet due to mutations in the CNG decrease or CNG Athree gene before the end of 2020, we continue to advance our.
Fine across ophthalmology, otology, and CNS and have multiple preclinical programs that are positioned for I'd, enabling studies Mark will provide an update on these programs later in the call.
Our actual RP program is advancing towards a phase two phase three trial, which we expect will commence in the first quarter of 2021, we believe the sustained improvement in visual function that we have observed in the phase one two trial to date gives us an industry leading position in this indication.
We have also seen early Evan it's a biological activity in both on the top gear trials, we believed that our robust technology platform unmatched manufacturing productivity and extensive capabilities to support our preclinical and clinical programs demonstrate that we have the expertise to develop best in class therapies that provide me.
Meaningful benefit to patients.
As discussed during our third quarter 2020 call AGTC has been taking steps to manage through the cobot 19, Pandemics and has been able to limit the disruption to our operations and manufacturing.
I have previously detailed several innovative and creative approaches that we are taking to proactively address potential pandemic related challenges, including a mobile vision testing program that allows us to gather.
Data from patients and our ongoing clinical trials, who are unable to travel to the site, whether they would ordinarily undergo evaluation.
There's a high enthusiasm for this program among patients physicians and clinical investigators, we believe that it validates both our commitment to the patients who participate in our trials and our ability to create innovative solutions that address the real world challenges of gene therapy development, we have conducted more than twice.
He buys visits to date.
I will now turn the call over to Mark for an update on our clinical and preclinical programs Mark.
Thank you so.
Today based on our analysis of the comprehensive written feedback from the FDA.
We'll present several changes we have made to our axa lumpy clinical development plan.
Adjusting the clinical endpoint visual sensitivity, including two last active items in the phase two or three study and extending analysis time project dosing the contractual <unk>.
I will also briefly review the status of their chroma toxic clinical trials.
Preclinical pipeline and provide an update I might add backs and manufacturing powerful.
Let me begin with an update on an ongoing phase one two clinical program in X. lumpy, which I assume no different moving toward a phase two three trials, which we expect will commence in the first quarter 2021.
The data were presented previously demonstrate that our actual up he kind of it provides early I sustained improvements in visual sensitivity and century those patients.
Supported by encouraging trends in visual acuity and quality of life survey results, suggesting that these improvements at positively impacting patients they stay life.
We have completed the initial target enrollment with 28 patients dosed across six dose groups and the patients does today continues to demonstrate a favorable safety profile.
To provide context for our discussion today, let me briefly review those results.
The chart on Slide 10 show sustained increase individual sensitivity in the fall responded a century dose and the valuable patients in groups to unfold with a treaties are shown in red and the untreated eye shown in gray.
This increase individual sensitivity as measured by mean change insensitivity within the trees area will sustain through the six months' time points.
On slide 11, the graphs show a positive trend in visual acuity in seven of nine essentially those patients with treated eye shown on the left untreated eyes on the rights.
Again, this is staying through than a six month time points.
Some patients who in who can either visual sensitivity on visual acuity also anecdotally reported noticeable improvement in visual function, including clear vision and reduced night blindness.
The second away to analyze Microperimetry Daisy <unk> point wise analysis that used to say identified points within a given increase or decrease insensitivity at age locus.
To be consistent with feedback from the FDA and Mira how other research groups in the extra Lucky's gene therapy space, a analyzing visual sensitivities data in future trials, we will define to respond as a change in visual sensitivity of at least seven decibel in at least five looks like my 12, which could revpas.
Then a clinically meaningful benefits.
This ends we presented on slide 12 re analysis of the Maya Microperimetry data from essentially does group treatments for patients that month six.
In addition, we are also reporting for the first time the same analysis from the group five dose group that month six.
When the patient analyze using on your definition of visual sensitive. Good response seven of the 15th century treated patients for all three dose groups combined had at least five no side, that's increased by at least seven decibel.
Patient talk about six and one patient that month right.
Focusing on the group five dose group that does level that we're planning to move forward in this analysis for a seven patients met the response criteria.
Of note if using the inclusion exclusion criteria of visual sensitivity planned at the upcoming phase two or three trial, one group five patient would be removed such that the respond to raise in this case would be for six or 67%.
We do not currently have a complete set a month 12 days were available which will be necessary to 40 evaluate this promising phase one two finding.
In addition, our phase one two did not include a control out the comparison, which will be included in our phase two three unnecessary what do you evaluate efficacy.
Other companies are recorded visual sensitivity using another analytical methods the static fulfilled analysis via the octopus parameter.
This device measures visual sensitivity across a larger area of the rest enough as compared to the Maya to include areas outside than my color on into the periphery.
Slide 13 reports preliminary data as rational sensitivity improvements using point why the analysis.
Locking the points on the perimeter grid, and indicating an increase or decrease in at least seven decibel square given look.
We have provided examples exit from three patients comparing the treated and untreated eyes, and which represent in green all know saw increasing by at least seven decibel at the time point indications.
Well challenging competitive cost research groups due to variations in grid patents used in black Bisons achieved we believe this analysis showed excellent p. part it produces comparable results did those reported by others via the octopus perimeter.
We also believe since the injection of the product is in the Central Mcdonough region that Maya provides a higher resolution measurement retinal sensitivity in the target area.
Based on the publicly available information from our competitors X L. P programs. We are confident that we have a very strong competitive position in this indication.
As shown in the previous slide 11, referring to be C.V.A.. We are the only company to record positive trends in visual acuity you know phase one two clinical study and we have reported early and sustained improvements individual sensitivity.
We have also demonstrated superior gene expression in side by side non human Primate studies conducted a more extensive analysis of the stability profile for the gene constructs.
Finally, we believe that we may have a safety advantage in that unlike states are reported in compared to the studies, we have not seen evidence that late stage inflammation in any patients, allowing for a wider dose range to be invest into late stage trials.
Slide 15, Thislife outlines the proposed design of the phase two or three trial.
We're off them all you studies in general that results in a concern if possible patient bias that could influence and points due to the psychometric nature of the test protocols.
And that's the extent possible there should be minimized.
We plan to enroll approximately 60 patients across to mass active I'm trying to control.
It does concentration issues will be 1.2, 11 effect, if you notice because they'll need to in the phase one two trial and 1.1 East 12 that did you know a male grew five in the phase one two trial, providing a tenfold dose range.
We also plan to as an important secondary endpoints a standardized functional mobility test the catches real would impacts on X lumpy patients by objective reassessing changes in patient mobility, 'cause a secondary to improvements in vision, resulting from therapeutic intervention.
In addition, other planned secondary endpoints Wayne good PCBA.
So feel sensitivity threshold SSG and changes in contrast sensitivity.
We plan to submit the six month interim analysis of the days it to the FDIC to discuss final planning based on those discussions we may modify the final trial design enrollment numbers and statistical analysis plan.
We also want to discuss with yesterday, the possibility of finalizing that dose selection for the treatment of the contralateral are based on the six month interim data.
Hey, Jude you see expects to begin the strong in the third quarter 2021, and because life results from a six month interim analysis in the third quarter 2022.
After any adjustments made based on this feedback if the totality of the data collected shows a compelling benefit risk balance we believe it will support a b L a filing.
Finding a recording of these days and maybe impacted by future effects of the cobot 19 pandemic on clinical trial enrollment, which as previously indicated impacted pediatric can roll it out and call. It helps your trial.
As to noted on slide 16, we also plan to expand the phase one two trials include approximately 12 additional patients and expect to begin enrolling in the fourth quarter Twentytwenty.
We expect these days to provide a near term assessment of the called actually between changes in visual sensitivity and patience abilities and navigate the functional mobility calls.
Slide 17, I like our next steps and moving forward at XL RP clinical development.
We are on track to finalize the protocol and initiate a phase two three and the first quarter of 2021.
We have begun manufacturing the clinical material for this trial at overlapping CZ and those and GLP testing labs, which will support our timeline for initiation.
We expect to report data from the ongoing phase one two trial in the fourth quarter Twentytwenty, which would include 12 months day different groups one through fall evaluating durability of effect and continued safety and interim data from groups, five and six which will inform our understanding of safety and efficacy of our ex Lucky kindred at home.
Got it doesn't.
No I will move on to discuss our ongoing phase one two trials in a chroma tops yet.
As previously announced in both the chroma toxic studies, we have seen early signs that biologic activity supported by patient reported outcomes with no dose limiting toxicity as well as DSMB see approved dosing a pediatric patients to the highest dose.
Enrollment of the I don't cohorts is complete and enrollment of pediatric cohorts is ongoing with for pediatric patients dosed in each trial.
We look forward to sharing additional data with you before the end of Twentytwenty.
I will now move on to discuss our preclinical pipeline progress with our manufacturing platform.
Our preclinical pipeline includes two ophthalmology program, one of which targets the dry form of age related macular degeneration or Andy.
Threed programs targeting central nervous system CNS disorders.
The CNS programs address adrenal leukodystrophies as well as two additional Reggie kinetic CNS indications frontotemporal dementia and I'm your trophic lateral sclerosis.
He's represents diseases with substantial patient populations that have well defined genetic and clinical phenotypes. The targets for these indications that program live and seen Oh RF 72.
We have.
Oh, so collaborations with autonomy and bionic sight, the genetic forms of hearing loss and helps genetics respectively.
Oh pipeline programs build on a industry, leading a being manufacturing technology and expertise, while enabling us to expand into a broader array of indications that have substantial unmet clinical need.
For example, a 50 Lisa manufacturing run using the process, which we are using to provide material for a phase two free trial has produced an estimated 2000 ophthalmology doses.
Based on data generated about facility biopsy demos, we believe that our manufacturing costs and did not only more productive another methods, but also produces material with great purity enough that the sentence, Texas containing the desired genes of interest is nearly 90% and process residual that generally because though 'cause detectability.
Current test method.
I will now turn the call over to Bill who will briefly review our fiscal results for the fourth quarter and Twentytwenty fiscal year.
Thank you Mark.
Fourth quarter 2020, we recorded a net loss of 14.5 million compared to a net loss of 10.5 million for the fourth quarter of 2019th.
The increase in net loss was primarily due to a 2.1 million increase in R&D expenses, a 600000 increase in Genie expenses, and an 800000 decrease from investment and other income.
The 2.1 million increase in R&D expenses was primarily the result of increased XL RP spending associated with Ace two three clinical trial activities, which will enable us to initiate our our studies as quickly as possible.
Increased eight from a top tier spending associated with patient enrollment and increased employee related costs.
Gee expenses increased 600000, primarily due to higher employee related costs and other DNA associated with normal business operations, partially offset by decreased share based compensation expense and legal and professional fees.
Now I'll move on to our financial guidance, we ended the fourth quarter 2020, they strong balance sheet.
Cash cash equivalents in investments as at June Thirtyth, 2024, 80.5 billion.
We believe these funds be sufficient to allow AGTC to generate data from our ongoing clinical programs initiate the xcellair Pete phase two three clinical trial in advanced our preclinical programs into the fourth quarter of calendar year 2021.
That concludes the teams remarks today operator, you may now open the line freight question and answer period.
Thank you end up if you go to your question answer session. If you like to be placed in the question Q. Please press star one under telephone keypad, a confirmation told would indicate your line is in the question Q you made press star to if you'd like true movie question from the Q.
For participants using speaker equipment, maybe necessary to pickup or has that before pressing star one.
One moment, please while we poll for questions.
Our first question today is coming from Joe can give us from H.C. Wainwright. Your line is that a lot.
Hi, Good morning, everyone. Thanks for taking the question and hope you are all doing well two questions first as we're looking at increasing visibility for all the three programs. It's nice to see the competitive slide that you put in your presentation today, one of things I wanted to focus on is when you look at the improvements or lack of in.
Movements in B.C.V.A., depending on the program just curious if you can comment on baseline characteristics of some of these programs that you might be aware of because as we've been talking to a physician's into space. You know, there's a lot of dependency on the baseline characteristics.
Well good morning, Joe. Thank <unk>. Thank you for joining US today, you know first of all we don't have detailed information about the baseline characteristics or the patients or even detailed data from our competitors in the field. When you can only speak to our data and what we can say.
For a b C. B a is that it is not be bass, a and point to look at and axle. Our p. because I think as you may remember from our discussions I actually I P patients do retain central vision into a good central vision, a you know very good central vision late in.
To that disease progression because this is a disease that starts in the periphery, so depending on where those baseline patient starts will really good vision, it's hard to see an improvement in that really good vision and so that's why we've always characterized our PCBA data is very supportive and encouraging but.
Typically significant and why we feel visual sensitivity or visual fields are microperimetry. However, you want to characterize that endpoint is the better and point cracks lucky.
No that is a very good reminder, unhelpful a feedback thing Sue and that's my last question is really just looking forward right. Now obviously these are orphan indications, but oh, you know the unmet unmet need is there. So as you look towards commercialization, you're obviously doing a lot of good background activities with regard to manufacturing that you reminded us today.
As well you know any thoughts around business development around any of your programs.
So as we've stated before we are continuing to have discussions with.
Potential partners, both for our Exlar P. program internationally as we've discussed in previous calls as well as our extensive preclinical programs well we have some that are interested potentially partnering with us on those so that we can move a greater percentage up them forward quickly into the clinic.
Topic looking forward to the data in the fourth quarter. Thanks, a lot.
Thank you Joe.
Thank you next question today for me from battery look any from BMO capital markets are up your line is a lot.
Great. Thanks for taking the questions good morning, everyone.
Congrats on the progress so wanted to first ask when regardless of the phase two three this interim analysis, there will be submitted to FDA and the potential for modification to design numbers start planning et cetera can you just talk a little bit about.
You know I guess, what it could be there would be driving those changes and maybe more importantly.
How confident are how how we should be thinking about the probability that the design.
Trial size et cetera that you're putting par today will be what you're actually able to take across the finish line.
I have a couple of other supply.
Good morning, Matthew Thanks for joining the call. It. So that's a have a very prudent and important question. Obviously, we based our trial design based on both the data from the phase one two and the feedback from the F.D.A. on our submission and obviously we've adjusted.
The the definition of a responder and adjusted that visual sensitivity analysis and for that reason, we thought a six month I hate to or look at the data and an interim time point to just double check that with this new analysis and.
Use of a control arm as a compared or that we got consistent data would be appropriate and then mark do you want to go into some of the more details about what will be looking for their and how we made the decisions on a dose selection that and patient numbers going forward.
Sure Yeah somebody might.
So as you know where we conducted an extensive series a preclinical work to establish an effective dose range and so we've been.
Monitoring the activity in the phase one to try that given the dose escalation phase to see the corresponding stuff that I think the important thing is that we are able.
To select two doses of how that that's a tenfold difference. We you know as something that we don't see clear evidence for dose response, but we want to get myself, the best chance of being able to differentiate the low dose from the high just so that was.
Cost of the rationale for that selecting the group to doesn't that group five dose and then assume mentioned you know we have evidence from the dose escalation phase one too.
Oh, the relative activity a responder rates using my penetrate so we will replicate that in attempt to replicate that in the phase two three and in addition in the phase one two expansion with a two doses book be matched to try to really fair enough. The datasets well have a robust basis on which.
Just to make the final decisions around the phase three trial.
Okay, great. Thank you that's helpful. And then two other questions for me first on the mobility course, it sounds like what you're using is quite similar to what spark used with Lux turn it off can you just remind us.
What I got similarities or differences there should be aware of that are that matter versus what they did.
I guess, what validation work needed on that or not course before it could be you know sort of fully blaster going into the case, two or three and then lastly, just a card you actually yes, no just wanted to confirm that into group five.
There was also no Ah Ah inflammation that require secondary dosage of steroids conceptually I could point of differentiation for your program relative to others.
I will get back into queue. Thank you.
Yeah, So Matthew I start from the bottom and go up but still on the information we have not seen in group five any of that secondary inflammation that others have reported and so the safety profile continues to be the same it's what we've reported for groups one through four and then I'll, let mark address a dress or the details about the mobile.
Realty test year right Matthew that it is very similar onto that used by the external we are working with a third party C. R. O who has already been using a mobility test in other retinitis Pigmentosa trials I'm. So we're quite confident of their ability to move forward with us, but mark do you want to.
Describes it at the test in a little bit more detail.
Yes, so I think it's really as simple concept, it's essentially I obstacle course that patients have to navigate that different light levels and so since we have evidence of improvements in retinal light sensitivity in the phase one two dose escalation. It made a lot of a sense to see.
That my a preliminary data translates to an improvement in performance in them ASE test and so we'll be looking at that.
You know at different levels to see if the two things correspond and we want to get it as quick a read on that as possible and that's why we've also included that in the phase one two expansion.
As well.
Sorry, the last thing that I'll mention is that we thought that the amazed test was a good kinda intermediary staff to connect the quantitative visual sensitivity change and basketball to the patient reported outcome survey so that man's testing.
Middle kind of allows us to correlate and aim in a slightly quantitative way what patients are reporting to us about improvements in visual function and tie up more concrete lead to changes in digital sensitivity.
Great. Thank you so much rather color congrats on the progress.
Thank you with his question today is coming from David Nierengarten from Wedbush Securities. Your line is that a lot.
Thanks for taking the questions on those two first off on the.
Patients the locations, which are having to phase one two is there any chance to use them as a part of the final data set from a phase two three and then the second question is relating to that is are you can you put a finer point I mean search could we expect to you know.
I don't I don't know, if you're going to find significant change the numbers for a phase two three years. Upon further discussion. So yes, there are we talking.
Plus or minus.
Five or 10% their patient number as you refine the plan with you after years, that's impossible to get a finer detail on on your expectations there. Thanks.
So good morning, David I hope, you're doing well and on the question about the six month I think that you know one of the reasons, we're doing that six month I a is because our phase one two did not have randomization and masking and did not have a specific control arm we have done.
Extensive analysis of the data from the phase one two and our are quite confident in the in the trial design and the statistical analysis plan that we put together, but because we didnt have the control arm and because we specifically didn't have the mains test in the phase one two we want to take that opportunity at the six month <unk>.
So we can't say you know what changes there may be there, but we feel that the plan. We've put together has a very good chances of being the final plan, but we just want to base that at that I a on some real data with amazed that we the control arm before we get all the way it did.
The 12 months study and then I'll I'll, let mark address the first question about the phase one two expansion.
Mark you might be a mute.
I'm, sorry, Yeah, Hi, David Yeah, So I think that plan really it to collect as much data as possible. If you recall the phase one two studies using a different clinical trial material lot. So we have a new improved process probably called process three that will be here just in the phase two three study. So we're also doing.
But the luxury between those two lots of material to processes.
I think overall the intention is to collect as much data as possible and to use the totality of the data in any.
Subsequent submission.
Knowing that you know it's generated in slightly different circumstances.
Alright, Thanks to those I was hopeful reminder, for different so processes. Thanks.
Thank goodness question stays coming from zinc was rather from Roth capital Partners. Your line is that a lot.
Good morning can that's in the date and thanks for taking my question does have two quick ones can you speak and what about the enrollment criteria that will be used my face any sense you didn't mention that I've always appreciate its five I thought would have been excluded it based on that that carry out and then after the six month I interim analysis.
For the phase two study, while you're probably going to discuss the potential to Dusty contralateral eye well just wondering how that data may perhaps factoring into the approval thinks it's going to be based on.
Out 12 months back it's gonna be six month data.
And if you all think sloppy contralateral eye dosing in the phase one checks into say.
So I'll make a comment and then and put put it over to mark to provide some additional information we the trial. The phase two three trial is set up to dose. The first one I and it is it is statistically powered by.
Based on the one I data. So we are now the the trial itself does not require the dosing of the contralateral eye.
To be able to see a difference between the active arms and they control arm and by the time of it any submission given that we have a positive discussion with the FDA. The six month part I, a we will have had or additional testing in that contralateral either.
I'll be in April to include in any future submission.
So the powering of the tile the significance of the trials not dependent on that contralateral eye and then over to Mark for the previous question.
Yes, so the design of the two additional studies that we've mentioned obviously based on our experience for the phase one too so.
Most many of the inclusion criteria remain the same which are related to the ability of the patients to perform the various tests as well as.
You know the requirements of them in terms of genetic mutation. So on so we don't have any major changes to that really other than those based on their data that we currently happen how that has influenced the penetrate results and so that's pretty much the path forward we.
I have better this with a what we call that protocol Advisory board and so for both the phase one two expansion as well as a phase two three I think we're pretty much ready and though in that regard.
Thanks, guys really helpful and then for the fees on Tech center, and you're not going to be down some conscious while I just went on.
So from my name is.
Just one I, yes.
Thanks, guys and congrats again on the data.
Thank you said, though.
Thank you as a reminder, that star one three placing the question Q or there's question today's coming from Kristen Cusco from Cantor Fitzgerald. Your line is now but.
Hi, Good morning, everyone. Thanks for taking my questions. The first one I have is with regard to the six month interim analysis with the agency that youre guiding could occur during the third quarter of 2022 do you expect that this will include six months data from the entire patient setting, meaning that you're roughly guiding to full.
We enroll the trial in about a year or are there a certain number of patients. The agency requested to see at this time point.
So to be clear the six month I, a Christian was at our discretion because we wanted to be able to get an early read and with the modified and point as well as the two mast arms compared to a contralateral arm and we didn't want to wait all the way to the.
And 12 months to get that.
You Gotta look at the data so the six month interim analysis was at our discretion and then we'll be able to take the data to the after gay and asked for their feedback and and and double check that we took their recommendations and their feedback into account as we designed the trial we.
Do not plan to weight to the do that six month delay until all patients are doses dose it will be the six month I will be based on a subset.
Okay, great. Thank you and then I know on me recently reported some data in a in the human gene therapy publication. So you know curious as you look at XL RPM General now that you've reported data from five different dose cohorts, how you think.
That matches up with the canine model and the different models that you've looked out in depth in the preclinical development, both on safety and efficacy and how these findings might help you with some of your earlier stage pipeline, where you know you'll be in CNS otology and additional ophthalmology program.
[laughter] potentially looking at dose escalation effects, there as well.
Well. Thank you for that question 'cause thing because we do believe that the breadth and depth of preclinical work that we do to support our clinical programs is one of the things that sets us apart a were a one of the few organizations that works with large animal models on a regular basis not just the naturally occurring.
Dog models that you mentioned, but also extensive nonhuman primate work to make sure we're targeting cells appropriately and we think this is a critical part of our ongoing product development and I'll turn it over to Mark to provide some more details about how we feel that that preclinical work.
Correlates to our work in the clinic Mark.
Hi, My question. So yeah, so you'll see that we've released the dose levels for the planned clinical study and.
Those were really predicated on the biologic activity that we had witnessed in the canine local now accepting that it's the only at muscle of the disease with different characteristics. It I think it was very gratifying to know that dose range that we found activity with in the dog model has essentially held up in the human.
Studies to date, meaning that they groups to Inphi for example of both active doses pre clinically.
So all in all I think we've been very pleased that we feel that there is some predicted validity of to talk model for what we're seeing a in humans.
Great. Thank you.
Thank goodness question today's coming from Jim Birchenough from Wells Fargo. Your line is alive.
Hi, Thanks for taking the questions. A this is E N.
Oh for Jim Congratulations on the data.
So first just wondering for the.
New responder analysis or visual sensitivity.
Could you share the data on the untreated eye.
It's so.
The untreated eye.
We did not see the same kind of responder rate that we saw it with the treated eye and I don't think we have that specific information at hand, as well, but would be happy to pull it and get back to yen.
Got it thanks, Sue and a in the you might have touched on this earlier, so I apologize if I missed that in the phase two three study for the efficacy analysis, what would be used as a the control would it be the out treated eye or the I from.
The untreated control subjects.
Yeah. The the trial again, another phase two three trial as well as the phase one as the phase two study trial is set up to compare eat active arm individually to the control arm. We will also be able to do analysis, obviously between the treated untreated and the active arms.
But the statistical analysis plan is set up to determine a difference between each active arm and the control arm.
I see and Ah because the treaty the arm has a bled, but how how would you match that to the untreated patients. How do you determine I guess, what would that blab be placed in a control patients.
Treated patients.
So going forward in phase two three development as well is in a phase one two expansion we are really trying to standardize as much as is.
Surgically possible the placement of the blood and the central Immaculate region, and so the analysis will be done by the same in the same set of loci within the treated eye at in the active arm as the on treated eyes in the control group.
Got it and last question, how many centers would be included in a phase two three and how do you think about the sergant surgeon training for consistency in the et cetera, no injection. Thanks.
So we're not guiding right now to the exact number of sites, we are still actively contracting and and negotiating contracts with sites both in the United States as well as ex U.S., we plan to conduct this phase two three globally.
And then when it comes to surgeon training, we have a very detailed surgical plan that I know we've discussed with many of you on the phone previously, but we are tailoring the surgical training to the background at each surgeon. So it's a surgeon has worked with US previously obviously.
We then we fully trained them through our program and they are ready to go. It's the surgeon has done extensive amounts of other gene therapy sub retinal injections then they might have been very short training program that they go through with the us and so and so on until we do have a very teared.
Approach to training surgeons.
Got it thanks, Texas.
Thank you we should have our question answer session I like to turn the floor back over to Sue washer for any further closing comments.
Thank you operator, we are really excited to close out our 2020 fiscal year with a path toward reporting data readouts for all three of our clinical programs by the end of this year and initiating a phase two three trials accelerate Pete program in the first quarter 2021, I'm incredibly proud of.
Our team and their ability to continue advancing our clinical programs and working towards our corporate objectives. Despite the challenges of the cobot 19 pandemic.
The progress, we are making and our clinical programs, our manufacturing campaign since us well for the future we as much work to do but we retain the high level of commitment to patients that is always driven us to as cheap our objectives. This commitment has only been enhanced by the productive and insightful interactions we're having with.
Our newly launched patient Advisory Council, and we expect that the patient and caregiver voices. The council provides together with the clinical and scientific feedback from our scientific Advisory Board will help us to develop innovative gene therapies that have clinical and commercial potential and so meaningfully improve patient.
Lives.
As I always do I'll close today's call by thanking the patients physicians and the AGTC team for their dedication to our cause and their support of our efforts to transform the treatment of rare ophthalmic tajiks and CNS diseases. I would also like to thank all of the healthcare workers at first responders, who are carrying tremendous burdens.
In order to protect our health and care for our friends families and colleagues.
I look forward to sharing additional achievements with you in the month ahead and I Hope you and your families are safe and healthy [noise].
Thank you.
Thank you that does conclude today's teleconference and webcast should be disconnect. Your lines is fine and have a wonderful day. We thank you for your participation today.
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