Q2 2021 Applied Genetic Technologies Corp Earnings Call
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Good morning, and welcome to the AGC of financial results Conference call for the second 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 <unk> may make forward looking statements, including statements about the Companys financial results financial guidance.
Its future business strategies and operations and its product development and regulatory progress, including statements about the projected timing for its planned for certain skyline clinical trials the timing for reporting data in both of these trials and the potential of its E. C. H M clinical programs actual results could differ materially from those discussed in these forward looking statements due to the.
Number of important factors, including uncertainty inherent in the clinical development and regulatory process. The extent of duration of the impact of the COVID-19 pandemic and other risks described in the risk factors section of a G. T. Six most recently filed annual report on form 10-K, and other periodic results subsequently filed with the SEC a T.
<unk> 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 E. G. T. C. Please go ahead.
Good morning, and thank you all for joining US with me on today's call are Mark Shearman, Our Chief Scientific Officer, and Bill Sullivan, Our Chief Financial Officer. During today's call I'll briefly review our recent accomplishments Mark will then provide an update on our achromatopsia and X linked retinitis pigmentosa or ex all of our Pea.
The program and Bill will review, our financial results for the second quarter of fiscal year 'twenty 'twenty one after our prepared remarks, we'll take your questions.
With respect to our recent accomplishments we are very pleased to have reported positive data from both of our ongoing clinical program.
Last month, we announced the first reported data to provide the quantitative evidence of improvements in visual sensitivity in patients where the chromatopsia due to mutations in the C and D D three or C. N V. A free Jean Marc will review the data in detail, but the key takeaway is that the visual sense of the pivotal data.
To support the positive patient reported outcomes that we presented in 2020 of them.
Boeing wants to focus on specific quantitative endpoints as we collect additional data that could support late stage development of products with the potential to change the lives of patients with the Chromatopsia.
In November of 'twenty 'twenty, we reported positive data from the ongoing phase one two clinical trial in patients with XL RP.
Mark will provide a more detailed review of these day in a moment, but there are two important conclusion for.
First the data indicated durable improvements observed in both visual sensitivity and support of trends in visual acuity over a wide range of doses with a favorable safety profile out to them on 12 in two of the dose groups. Additionally.
Additionally, based on the comparison with publicly released data from our competitors. We believe that we have the best in class product candidate that may provide significant benefit to patients with ex all our P, especially with regards to product safety.
We have an aggressive but we believe attainable strategy for advancing our excellent product candidate through both our skyline and just the clinical trials.
Last week, we weren't quite at two successfully closed a public offering which raised approximately 69 million of net proceeds combined with our current cash position. The additional capital extends our cash runway into calendar year 'twenty 'twenty three.
We anticipate that this will be sufficient to support for additional data readouts for our ex R. T program across three clinical trials. The phase one two trial, the skyline trial and the Vista trial and two additional data readouts for each of our achromatopsia programs in the phase one two trial.
With progress in both of our ongoing clinical development programs and our strength and financial position. We have started 2021, where the GOR of the deal of momentum and we intend to build on momentum by executing on our clinical development strategies and advancing additional pipeline programs towards the clinic.
I'll now turn the call over to Mark for a review of our clinical programs Mark.
Thanks, Sue let me begin by reviewing the progress on our achromatopsia clinical programs.
Overall, our ongoing phase one two clinical trial of Chromatopsia continue to generate the encouraging for any data that support further clinical development.
The product candidates of well tolerated across from a default dose range on Dr. Demonstrating signs of biologic activity.
The chromatopsia ease of congenital disorder, wherein the gene mutation is not the only affects the function of the code of photoreceptor themselves, but also interfere with signaling to the developing visual cortex.
This means that it may take additional time off the function is restored in the eye for appropriate signaling between the retina on the break to be fully reestablished.
We have previously stated we believe that the additional benefit may be observed by following the patients for longer periods of time, using higher doses and dosing the younger patients.
Last month, we were the first to report static perimetry data demonstrating improved visual sensitivity to the excuse that the test retest, great visibility at patients lots of visits ranging from three to 12 months post treatment for seven of 16 patients in the three highest dose groups in the biscuit of trial.
And three of 16 patients in the fall of highest dose groups in the a free trial.
In the subset of be three patients with the valuable multifocal electroretinogram improvements in electrical signaling where measurable in the same treated area.
We continue to analyze data from both the Patriots day free trial and are on track to report 12 months post treatment data for all other subjects in the study in the second quarter of 2021.
The favorable safety profile on this exciting new evidence of biologic activity together with the robust body of positive preclinical data gives us additional confidence in the potential of our chromatopsia of product candidates.
Here is an example of visual sensitivity on the ERP improvements in one of the adult patients from the base III study.
The graph on the left shows the change in mean sensitivity assessed by the static full field perimetry.
As shown in Gray little changes observed in the untreated eye.
There is a very clear increasing the main sensitivity in the treated eye shown in red.
The chart in the sense of shows the heat map of sensitivities changes assessed by the static fulfill perimetry of baseline and the 12 months post treatment.
Again clear improvement is seen in the areas of the Pea of brighter at the 12 month time point call day thing with the placement of the sub retinal blip.
The panel on the right shows change from baseline in multifocal electro rest of the hot coffee for M. S. E. G, which is an objective measurement of electrical signaling in the retina that isn't subject of patient box here.
Here again, we see areas of improvement is masked by brighter colors within the treated area.
This slide shows changes of stomach fulfill perimetry results over time for the same patient with the treated eyes shown on the top row of the untreated eye on the bottom row.
The improvements of the baseline or observed at month, three and are sustained at month 12 and studied the di corresponding to the placement of the bleb was little to no changes observed in the untreated eye over the same period of time.
Slide nine is an example of the B three pediatric patients with improvements in visual sensitivity.
We see results similar to the bakery adult patient with respect to mean sensitivity change by static fulfilled perimetry and on the sensitivity of change heat map.
This patient also share what these changes meant in terms of day to day activities, noting the ability to see more details without the need for tinted contact lenses or adopt goggles.
This is the third example, which is from the pediatric patient from the Ace III study showing improvements in visual sensitivity when comparing the treated on untreated eyes.
On the sensitivity of change heat map the patients exhibited improvement of about nine as shown by the bright the areas.
We are very excited that the ongoing achromatopsia trials of the first to report improvements in visual sensitivity of following treatment the.
The results of out patient by patient analysis give us additional clarity on our path forward and we will be focusing on the aesthetic perimetry endpoints.
The early indicators of activation of retinal cone cells.
We have amended the study protocol for these trials to allow enrollment of patients actually almost four years of age which May result in a greater degree of restoration of functional vision.
We also intend to collect several new datasets, such as functional magnetic resonance imaging of fmri that kind of directly magic changes in brain activity in the visual cortex in response to the different stimuli and thus potentially provide evidence of neuro pathway activation.
And the improved color brightness test to better quantify the changes in color perception the patients report experiencing following treatment.
We anticipate several important milestones in the Chromatopsia of clinical program in 2021, including dosing of the remaining pediatric patients of the two highest dose groups and assessing the amusing fmri on call of brightness tests.
Well it should be noted that we do not have clarity on the potential for the impacts of the ongoing COVID-19 pandemic might have on our recruitment and screening efforts regarding pediatric patients, which has impacted patient enrollment to date or on any such potential delays would affect our planned data readouts for this cohort we currently anticipate.
Reported three months data from pediatric patients in the fourth quarter of 2021.
We also expect to report full 12 month data from the adult subjects in both achromatopsia trials in the second quarter of this year.
I'll now provide a brief review of our ex of lot P program.
We believe that the data we have presented to date differentiate on ex a lot of P candidate from the competitiveness of candidates and provide a strong foundation on which to continue advancing our clinical development strategies.
As many of you know in November 2020, we presented updated the visual sensitivity visual acuity and the safety data from the centrally dosed patients in groups to for five and six of the ongoing phase <unk> clinical trial.
This included 12 months data for groups to enroll on six months data for groups of five and six.
We provided an in depth review of those day throughout the time and the detailed data are available on the investor page or the GTC Dot com will provide only the top line review today.
We've continued to see a favorable safety profile with no dose limiting toxicity observed in all of 28 patients across six dose groups.
At the 12 month time point for the nine centrally dosed patients of groups to fall, we saw measurable improvements in visual sensitivity for two of the eight evaluable patients while the third patient identified as it responds of at six months fell just below the cutoff.
As a reminder, patients as defined as respond to spend at least five low side within the central 36, low side of the Perimetry grid increased by at least seven decibel loss we.
We believe this represents an encouraging sign of Europe for biologic effect.
At the six month time point, we saw measurable improvements in visual sensitivity for five of the 11 patients dose essentially in groups of five and six.
Three of the 11 patients in these groups, who are not responders would not meet the inclusion criteria for future trials many of them.
The five of eight patients for 62% would be considered responders.
The combined analysis of the best corrected visual acuity data from all of 'twenty centrally dosed patients shows that the majority of patients showed stable or improving the CPA in the treat the die.
<unk> for the untreated eye at month six.
None of the patients in that up to 12 months with the same improvements maintained this results has not been reported in other extra lot piece trials.
To provide additional context on why we believe we are strongly positioned to have an industry, leading ex la <unk> therapy candidate. This slide highlights our strong competitive position in this indication.
Based on comparisons without competitors publicly reported data we have reported data from more patients and I've seen that the efficacy results with respect to best corrected visual acuity and the visual sensitivity.
Importantly from a safety standpoint, we have not observed and the project related serious adverse events and unlike our competitors, we have not seen any secondary inflammation in any of our ex without pes trials that required re dosing with steroids to control.
We also believe that the favorable results. We are seeing in the clinic are a result of all well designed vector construct.
As previously outlined the proposed design of the Vista trial is currently expected to include approximately 60 patients randomized across three arms of.
The low dose group the one point to 11 Phe per mill group two dose from the ongoing phase one two trial a high dose group. The 1.1 of 12 Beachy thumbnail of five dose from the ongoing phase one two trial and an untreated control group.
The primary endpoint will be based on visual sensitivity defined as having it at least the seven decibel improvement in visual sensitivity.
In at least five pre specified low site at month 12.
This primary endpoint was informed by comments that we've received from the FDA.
On what evidence, which help support of showing of a clinically meaningful improvement on micro perimetry at the group two on group five doses.
Importantly, we plan to use this endpoint is one of several secondary measures of visual sensitivity that we believe have the potential to support of clinically meaningful benefit, including the CVA and the or of the NC mobility maze. We will also be using a validated patient reported outcome survey.
To gain additional insight into what patients find meaningful.
We intend to submit a six month interim analysis of the data from the Vista trial to the FDA to obtain feedback on our development plan to support approval.
Based on FDA feedback, we may modify the final trial design enrollment numbers and all the statistical analysis plan.
We also intend to discuss with the FDA treatment of the contralateral eye.
At the time, we share the six month Vista data with the FDA. We also intend to share complete 12 month data from our Skylark trial, which is an expansion of our current phase one two trial.
Under the amended protocol will be dosing additional patients at the group's two and five that will seek to verify the correlation of visual sensitivity of changes to mobility maze outcomes and to maintain patient and site engagement.
In summary, we believe we have of superior expertise and ex <unk> gene therapy and that this expertise will enable us to demonstrate best in class clinical data.
We also have an improved manufacturing process and require no further process development of scale up to support commercial launch.
We are excited that we expect to have multiple opportunities to further accelerate our actual up P program over the next 12 to 18 months, including presented 12 months data from groups five and six of the phase one two trial in the second quarter of 2021, and three months data from the Skyline trial in the fourth quarter of.
Of 2021.
We also plan to report six months with the data on 12 months skylight in data in the third quarter of 2022.
I'll now turn the call over to Bill for a review of our second quarter fiscal year 2021 financial results Bill.
Okay.
Thank you Mark Slide 20 provides.
An overview of our financial results for the second quarter of fiscal year 'twenty 'twenty. One we recorded the net loss of $15 5 million compared to the net loss of $8 6 million for the second quarter of 2020.
The increase in net losses, primarily due to a $2 5 million decrease in revenue.
The $3 4 million increase in R&D expenses and the.
300000 increase in G&A expenses.
The $2 5 million decrease in revenue was primarily due to $2 2 million of noncash bionic sight collaboration revenue in fiscal year 2020 that did not recur in the current year.
The $3 4 million increase in R&D expenses was primarily the result of increased <unk> spending associated with their planned of manufacturing clinical site preparation and other activities related to our skyline and Vista trials.
And increased employee related costs, partially offset by a decrease in external spending for our achromatopsia trials.
The 300000 increase in G&A expenses was primarily due to higher of legal fees, partially offset by a reduction in employee related costs.
Now I'll move on to our financial guidance. We ended the second quarter of fiscal year 2021, with total cash cash equivalents and investments of $53 1 million.
We believe these funds along with net proceeds of approximately $69 million received from our February 2021 financing.
Provide E G T C with the strong balance sheet will be sufficient to allow <unk> to generate data from our ongoing and planned clinical programs and fund currently planned research and discovery programs into calendar year 2023.
That concludes the team's remarks today operator.
Now open the line for a question and answer period.
Thank you we will now be conducting the question and answer session. If you would like to ask a question. Please press star one on your telephone keypad. The confirmation tone will indicate that your line is on the question queue.
On the press Star two if you would like to remove your question from the queue for participants using speaker equipment and may be necessary to pick up your handset before pressing the star keys.
Our first question is coming from the line of Jim <unk> with Wells Fargo. Please proceed with your question.
Hi, guys. Congrats on all of the progress on thanks for doing your call away from 40 other biotech companies at the same time.
So with that said just on achromatopsia could you maybe talk about the changes that patients are experiencing visually.
And the quantifying the this is useful vision on number one and I guess the United.
How do you capture kind of the usefulness of.
The changes in their vision and then two it seems pretty clear that you're turning back on cone's. So would you expect improvements in photophobia and you now have you started to hear that kind of anecdote.
When would you expect to see that dynamic.
Well good morning, Jim and thank you for joining US today, we definitely are seeing debt there come chromatopsia patients are reporting improvements in vision and I'll turn it over the mark So he can kind of tie and quantify these changes investing visual sensitivity and how that is being reflected in the in the patients.
Functional vision in their comments Mark.
Yes, good morning, Jami. Thanks for the question. So as we showed in the presentation. There are examples now of patients whose.
Fulfilled perimetry is increasing by an average of 10 decibel loss, which represents a 10 fold increase in light sensitivity.
<unk> also indicated that we have evidenced from patient reported outcomes for.
Feedback from the investigators that this is the patients.
Experiencing benefits for that vision. We gave one example to your point of bad Photophobia, where the patient was able to see things without the use of.
Contact lenses or goggles, and so we feel that as we as we've said that the perimeter is the first indicator of the current being reactivated and we feel that a lot of the benefits.
Benefits will now emerge as a consequence of the cones re functioning because many of the symptoms of achromatopsia are a consequence of both the.
Interaction of cones and rods in the retina together with the signaling of those photo receptors for the visual cortex, and so we're very hopeful that as we continue to collect data for longer periods of time higher doses and importantly, the younger pediatric patients that more and more of these benefits will emerge.
And Mark maybe just on the on the younger patients that youre looking to treat down for the age of for could you talk about but it seems like earlier is better but at the same time is the higher do you expect higher retest variability.
And how do we think about you know testing kits for the things like visual sensitivity of that young age.
Again, a good question and we don't have a lot of data to date on the younger pediatric patients we are being guided by.
The principal investigators who have.
Seen most of these patients before.
Aware of there.
<unk> and the deficits and have they made for fall on the tests and so I think.
This is another reason why we want we're eager to start we stopped enrollment of the hydro pediatric patients to get a better handle on.
The performance of these tests and we won't get enrolled the patient of young patients in this trial, if we feel that they can't complete the tests such as.
Quiet and if I may.
She will be guided by the investigators on that and.
And I'm just going to interject also that we have modified the.
On the study Emmanuel to modify how the test takers are being interacted with and so we did modify things like the E. R. G. In the static perimetry to account for the younger age. So we've really worked very hard with the pediatric ophthalmologists to make.
Those tests as easy to complete for the younger patients as possible.
Great well, thanks for taking the questions guys.
Thank you. Our next question comes from the line of Joe Pant, Janice with H C. Wainwright. Please proceed with your question.
Hey, good morning, everyone. Thanks for taking the question.
So I was wondering if we could just step back a second here with a chromatopsia in your end points.
And I don't think I'm overstating it when I say you are sort of really blazing new territory here with regard to assessing efficacy in these patients. So can you talk a little bit to the evolution of the clinical end points that you might bring forward, obviously youre looking more towards static perimetry now and how this might impact.
Your regulatory discussions.
Well good morning, Joe and thank you for the question I think you'd pick up of very good point, because we have had discussions with people about how in our axle RP program, we concentrated very much on the buy them micro Perimetry and I think that initially we also thought the potential.
The the my on micro Perimetry, because it was more precise.
And had a little bit less variability that that might also be important in achromatopsia and that has not turned out to be the the the endpoint has shown us the biggest difference and it has been really able to be measured and I'm kind of turn it over the mark So he could really explain why.
There's been this evolution in how we measure visual sensitivity in the in the achromatopsia patients Mark.
Yeah, So just to make.
For the points of the my of Perimetry. The reason why the patients.
This endpoint hasn't proven to be useful is because of the particular.
Details of the technical details of taking the test. So for example in my preliminary uses a smaller target on the octopus perimeter on the achromatopsia patients because of their pool of visual acuity cannot detect that target well and so they don't perform the test. So I think the point is that we continue to evaluate.
A number of different tests that will interrogate the patients.
Response to treatment, whether it be acuity sensitivity light discomfort as we indicated in the.
For the presentation. We've also introduced functional MRI and a new call of brightness test we feel that both of these are in.
In response to the feedback from the data that we've seen so far and May help us better quantify what the patients are clearly matter of fact, the manifestations of the improvements that the experiencing.
Got it got it. Thank you for that and then my second question I guess, if my first question was the step back I guess, Mike for My next question is a step forward and Mark you mentioned in your prepared comments about.
The improved manufacturing protocols and processes that you guys have been putting in place I was wondering if you could share any specifics around that obviously there is a.
Competitive landscape to consider here.
But how this improves your overall processes and how it can be translated to a day across your pipeline.
Yeah I'll take the.
First the answer to that and then Mark can talk about some of the details with the.
We focus on two main things one is scalability.
And productivity and so that's the actual manufacturing process itself.
Our and fully volumetric stirred tank Bioreactors, which are pretty standard in biologics production overall.
So in suspension and so we can scale up volume metrically instead of having the scale out across the factory floor with with adherent cells and in any kind of plastic methodology and that means that in of 40 leader reactor, which is quite small we're making over 2000 ophthalmology doses.
And then you can scale up that 40 leaders for any larger dose needs.
And still be in a very contained mm factory floor, which we think has very good cost profiles going forward.
Secondly, we have reviewed this process as part of our end of phase two submission and interaction with the FDA and they have very little comments and in the way of reviewing that in detail and it's up to talk about different assays that they wanted to be done at earlier stages in the process and to our.
Line debt.
We of course, wouldnt need to do comparability to our phase one process on them and that's already underway and then the third thing I would point out is that we have spent a great deal of time and effort over the last two years on the assays and really bringing gene therapy assays AAV gene therapy assets forward.
Time.
Into much more robust and quantitative measures and I'll leave it to Mark maybe he can give a couple of examples of this because I think this is so important to the regulators debt the gene therapy world come in to the current time and really put the same amount of rigor into those assets.
Characterization of the product as is expected of all biologics Mark.
Yes, I think its really productivity and quality of the productivity improvements of come from a couple of different aspects one of which is we closing of some of the cell lines that are used for packaging on the virus, which gives us higher yields as well as an improved proportion of full two empty capsid.
And then we've modified the downstream processing of the virus to change the column system for the use of Gan enriching for full capsid and at the same time, reducing the overall process residuals and in many cases the process residual for now at the limits of our below the limit of detection of the revised and improved analytical assay. So.
We feel that.
Hi, a threefold higher improvement in full to empty, which helps with dosing on safety and immune response to the capsid and then improvements in the overall quality.
Through the changes to the downstream processing.
Thank you very much guys.
Thank you. Our next question is coming from the line of Matthew Luchini with BMO capital markets. Please proceed with your question.
Hi, good morning, everyone and thanks for taking the the.
The questions.
Maybe first on XL RP.
Can you just help.
Set expectations, a little bit around the 12 months a group of five six data that's come in next quarter.
And how we should be thinking about this dataset.
Particularly on the context of the of the.
The skyline data that's going to come at the end of the year with the the new endpoint.
And then on the Chromatopsia I'll just like the get your latest thinking on sort of the the go no go perspective on this program do you feel like with the data that you have now generated would have what you need how critical I guess really is the the data that's going to come from a four year olds.
And then.
Yeah, just those two for now.
Thank you Matt for joining us this morning and first on the.
The actual RP are.
The 12 month data from groups of five and six I think that what our expectations would be based on what we've seen so far is the we see the same durability of rich.
Bonder rates from the six month time period to the 12 month time period, and so we would consider that consistency and durability of the product to be a very positive sign the what differs from the three month data. We expect from Sky line is that that three month data from the skyline will be our first of all.
Opportunity.
To see how the mobility means that we're instituting corresponds to those improvements in sensitivity because we believe one of the Conundrums are the one of the kind of stumbling blocks with the visual sensitivity endpoint isn't a lot of people have trouble getting kind of their brain wrapped around what are those changes.
Visual sensitivity really mean, whereas when you see someone perform on amaze test it it's much easier to understand and so that's why we added the maze task, we're working with Aura of third party provider, who developed the very sophisticated means.
There are multiple paths at the base line with different high and low obstacles color contrast of very fine Asian of light levels. So we feel we're where we're using a very sophisticated means and it's the three month data.
We're very hopeful based on other products that abuse, the amaze before but that means will support and help to explain the people and make real to people. These improvements in visual sensitivity. So that's really on one of the main purposes, we had for expanding the phase one two adding more per.
Asian, adding that maze test and also randomized against the dose groups of patients won't know whether theyre in the low or high dose so hopefully that addresses your.
X a lot of P. A questions and then I'm going to turn it to mark to talk about.
What is the debt what have we seen so far in line of Chromatopsia without visual sensitivity improvement and then what we hope to learn from the pediatric patients Mark.
Yes. So we have examples of both adult and pediatric patients improving in the full field static Perimetry I think.
People, who work in this area of all agree that the.
The Chromatopsia indication results as a consequence of both direct effects on the current photo receptors, but also on the communication of those photo receptors with the target field individuals' cortex of the brain.
And the development of various aspects of of visual function to agile Hood levels takes place from early bird through to about age 12 to 14, depending on the individual.
And so I think what we're doing is a very logical progression to move to younger patients where there is more opportunity to correct. The deficits that those patients have by treating them younger. So I don't think there's any debate that that's the right thing to do and so we're very hopeful of that in doing so.
So the benefits that we've already seen in the older patients will be.
Potentially greater in those younger patients.
Okay. So just to make the I guess just to make the point explicit.
Further progress on this program is contingent upon.
And continuing on improved benefit in the younger patients is that the take home message of all of that well as we indicated we will have the month 12 adult data the data that we disclosed recently was a combination of between three and 12 months for different patients as they are progressing through the dose escalation part.
For a dime. So we're gonna see half of that looks at month 12, and there could be some changes even greater improvements from what we've seen already plus dosing. The remaining two groups in both <unk> and be free up of the younger pediatric patients. So I think collectively that entire dataset will give us a better sense of the.
The optimal path forward for the achromatopsia programs.
Okay. Thank you very much.
Thank you. Our next question is coming from the line of Christian <unk> with Cantor Fitzgerald. Please proceed with your question.
Hi, everyone. Good morning, and thanks, a lot for taking my questions. The first one I have here is on of Chromatopsia and was hoping you could remind us about the age that these patients start to lose the brain plasticity and at what point during the course of their lives does this become severe and I.
I'm just curious here as two of the three individual patients that you reported today they were out of lessons. So wondering if that's at all supports the rationale of he.
Evaluating the pediatric patients.
Yeah, Good morning, Christian and thank you for joining us today and your questions really do kind of follow on Matthew's questions and the distinguishing between what we can expect in adult patients and pediatric patients.
This is the first time in the envy of Chromatopsia of patient population that there's ever been seen of quantitative and improvement in in an end point of this magnitude and so I think you know one of the message into the areas that we're still learning about what is possible to improve in these patients we saw such great results in our preclinical.
The studies, which were naturally occurring dog and sheep models that we're we've always been very hopeful for this patient population and some of the patients had improved by that 10 fold improvement in visual sensitivity, where the older patients and so by collecting the additional data will learn it.
Is it possible to get even better improvements in the pediatric patients because these patients have the symptoms and the deficit from birth.
And the the.
I'll pass the Mark the question about neuroplasticity. They think it's important to point out that in the data we've seen to date, we've seen really good improvements in some of older patients as well as the adolescent patients, but turning it to mark.
Yes, good morning, Kristen it's a good question.
I think the short answer is that Theres not a fixed point in time, where you go from development to no development at progressive through infant would true to the.
The children that entertains and EBIT in some cases, such as faith perception continues to mature into young adulthood. So I think.
At this point, we have some guidelines on when the different facets of visual development change for all completed but the bottom line is we're going to be doing the studies generating the data too.
To allow us to define.
This occurs in achromatopsia patients because of lot of the evidence on the information that I am Cogent is basically based on just development of the cortex in the context of of disease, where youre trying to correct. It the closest we know of bag of something called Amblyopia Ware.
If you have disturbances in vision that can effect.
The development, but we know that that can be corrected to some extent even in the agile later childhood and agile foot. So I think with sale of finding ex basically and that's why we're very excited to enroll the remaining pediatric patient basically generate the data.
Thank you and then I noticed in your February corporate presentation that you highlighted partnering efforts for some of your R&D, enabling candidates. So with that could you help us understand the company's strategy beyond the XRP on ACTH M programs.
Specifically, which indications here you might be looking to take forward yourself versus where the partner and.
You know again, if these partnerships or something Youre looking to establish ahead of running trials and then lastly, you know this is a pretty extensive pipeline. So what should we expect this year in terms of.
And maybe some additional preclinical data thank you.
So we are as the company as you might imagine very focused on excellent opinion of Chromatopsia as the most advanced program programs and the program most able to provide value to all of our stakeholders in the near term and the programs that can get product to patients.
In the near term given that we are very excited about our preclinical pipeline and there are many programs. There that we think has tremendous value.
Such as our CNS indications, which can reach an even larger patient population than some of our ophthalmology indications.
And we're interested in partnering them because we can't with such an extensive pipeline in a smaller company you can't do everything independently and so we would expect over the course of 'twenty 'twenty one to have additional data we've mentioned that the most important meetings of the year for our industry both are.
And the S. D C. P are coming up in the spring, we expect that the participate fully and so we would have more guidance on information about these programs throughout the course of 'twenty 'twenty one.
Great. Thank you again.
Thank you as a reminder, if you would like to ask a question at this time. Please press star one on your telephone keypad. Our next question is coming from the line of the Gela with Roth Capital Partners. Please proceed with your question.
Good morning, guys just two for your questions for me the free.
Just your opinion about the E. C. Chem program just curious if you expect to see the difference in efficacy between the three.
On the pieces as you continue to accumulate data and then the second one just kind of being about at the new endpoint for each of them I know you plan to meet with the FDA asking you got more data from that program, but any feedback about <unk> thoughts on the endpoint is one of them.
On his expectations from the update the guidance at the kitchen waste each of them.
Well good morning, everyone. Thank for thank you for joining us today and I'm Gonna of pass. This question to Mark to talk about what our current thoughts are on whether we would expect any differences between a three and it would be free and then the collection of data points and interaction with the FDA Mark good.
Good morning day part.
So the phase III on a three genes are two sub units of an ion channel and we don't fail since those channel those happiness at a strike of associate can metric of mass equal amounts. We don't see any reason why phase III or ace we should be different from one another player of the phenotype of the disease.
At the same so at this point, we feel that the difference in numbers that we're seeing is just a.
A because it's a small number of patients and we haven't progressed for the eighth day trial as far as of the piece of the trial yet so we're going to just wait and see how that plays out before reaching any conclusions as to whether <unk> is more.
The more readily corrected on the other then to your second point I think the cash.
The fulfill static perimetry is of very well known and established endpoint Tech.
Technique for measuring visual sensitivity.
It is used or has been used in other indications as a primary end point. So we feel that the FDA has quite a lot of familiarity with the octopus perimeter and the changes that they would expect to see as being clinically meaningful. So I think right now thats, great starting point for us.
As a primary endpoint that is quantifiable in these patients and as we're collecting more and more data as we always do we will continue to see what the emotions and see if there's any adjustments to that but right. Now we feel this is a good point of <unk>.
To move forward with.
Thanks, Mike and then the final one for me you know you are seeing some signs of BT for now.
But that had really positive results with ex MRP and now with these two programs of just curious you know if this has any read through to the rest of the pipeline in terms of you know things like the accomplished here that could make the easier from a process perspective as well as in terms of making sure the gene therapy application.
Yeah, that's a good questions like mine and certainly these the success we've seen in these two programs do gives us great hope and plan and confidence in our two other ophthalmology programs, which are the CF H program for dry AMD and the Star Gertz program because in those.
The programs, we're targeting the same types of cells photo receptors.
And our manufacturing process, our product construct screening process would all be directly relate to pull in and read through to those programs in the same way for the CNS indications on our manufacturing process is really agnostic to which serotype, we're using an <unk>.
So we would be the maintenance.
Exact same highly productive and and characterize manufacturing process and the way that we go about screening for capsid promoters in designing the gene cassette is exactly the same in those indications we screen in nonhuman primates.
The we're sure that we're screening for the right specificity, we go through and all the way through to monitor of biological activity in animal models for each component and so our of philosophy and methodology are really carried through to those indications as well and the same can be said.
In ontology and metrology is really very very similar in the different kinds of cells and the actions of the different kinds of cells in the ear is in the eye and so we are gaining more and more confidence that how we design construct and the manufacturing the these vectors.
Are really in a very solid foundation.
Thank you Steve.
Thank you. It appears we have no additional questions at this time, so I'd like to pass the floor back over to Sue washer for any closing comments.
Well. Thank you everyone for joining us today, and we believe that the recent advances in our excellent P. In Oklahoma tops of the trials and the added proceeds from our recent public offering put us in a very strong position to work through all of the multiple milestones in the months ahead. We expect to report 12 month data from groups five and six.
And the ongoing XRP trial, and all of the adult patients from the ongoing of Chromatopsia trials in the second quarter of 'twenty 'twenty, one and to report that three month data from the Skyline trial in the fourth quarter. We're currently focused on completing the pediatric patient enrollment in the two highest dose groups in both of our Oklahoma.
Topsy of trials as Mark mentioned and following all of the patients who 12 months with the three months of the data expected in the fourth quarter of this year. We're also looking forward to additional Vista in skyline Readouts in the calendar year 'twenty 'twenty, one 'twenty 'twenty two mainly we intend to continue advancing the prioritized preclinical programs.
As we discussed today towards the clinic.
The challenges in the world around us today, I've never been more optimistic about our potential to make a meaningful difference in the lives of for patients and I'm, especially thankful to the entire E. G. T C team, our clinical partners and the patients in our trials, enabling us to achieve important milestones and put us on a path toward continued growth in <unk>.
Grass during an unprecedented global pandemic, our recent achievements give us additional motivation to do the work needed to realize the potential of our technologies and programs I look forward to sharing our progress with you in the months ahead. Thank you.
Ladies and gentlemen, this does conclude today's teleconference. We thank you for your participation and you may disconnect your lines at this time.
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Okay.
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Yeah.