Q2 2021 Otonomy Inc Earnings Call
[music].
Ladies and gentlemen, thank you for standing by and that can true autonomy incorporated second quarter 2021 financial results Conference call. At this time all participants are in a listen only mode. After the speaker's presentation, there will be a question and answer.
True session to ask a question during the session you will need to press star 1 on your telephone. Please be advised today's conference is being recorded if you require any assistance press star zero for the operator now it's my pleasure to hand, the conference over to our first speaker today, Robert <unk>, managing director of Investor Relations. Please.
Go ahead Sir.
Thank you operator, good afternoon, and welcome to autonomy second quarter 2021 financial results and business update conference call. Joining me on the call from autonomous are Dr. David Weber, President and Chief Executive Officer, and Paul Cayer, Chief Financial and business Officer before I turn.
On the call over to Dr. Weber I would like to remind you that today's call will include forward looking statements based on current expectations such statements represent managements judgment as of today and may involve risks and uncertainties that could cause actual results to differ materially from expected results.
Please refer to Autonomies filings with the SEC, which are available from the SEC or on the autonomy website for information concerning the risk factors that could affect the company.
The autonomy, specifically disclaims any obligation to update any forward looking statements, except as required by law I will now turn the call over to Dave Weber, President and CEO of autonomy.
Thank you Robert Good afternoon, everyone and thank you for joining us on this call to discuss the economy's recent business updates as well as our financial results for the second quarter of 2021.
We are making solid progress across our multiple programs for tinnitus and hearing loss and the key takeaways from this update includes the following <unk>.
Enrollment for the OTO 313 phase II trial in tinnitus, and the OTO 413 phase 2 extension study in hearing loss off to good starts with both studies on track for top line results in mid 2022.
We have achieved several important milestones for our <unk> gene therapy program, including demonstration of preclinical proof of concept in 2 independent models of congenital hearing loss and completion of a pre IND meeting with the FDA.
We have initiated IND, enabling activities for this program and expect to file on A&D in the first half of 2023.
We're also also continuing to progress our 2 other preclinical programs on all 510 for OTO protection and autos 6 X X for severe hearing loss.
Finally, we significantly strengthened our financial position during the second quarter by completing a financing in April and modifying our debt facility to extend the interest only period.
These actions provide us with the cash runway into the second half of 2023.
During this call I'll provide a brief update on our programs and then Paul will recap our financial results. We can then open up the line for any questions.
Beginning with OTO 313 enrollment in the phase II trial is going well. The design of this trial is based on the successful phase 1.2 trial that demonstrated a higher proportion of responders in the OTO 313 group versus placebo based on the tinnitus functional index.
<unk>.
The phase II will enroll approximately 140 patients with persistent unilateral tinnitus or at least moderate severity based on the <unk>.
As in the prior trial, where we observed a high correlation between the various metrics in responders. We are also tracking tinnitus loudness.
And patient global impression of change.
To enrich the study population, we're excluding patients with severe hearing loss, who we believe may be less likely to respond to treatment.
And we have increased the minimum severity of tinnitus required for entry.
We have also expanded the patient population eligible for enrollment by increasing the time from tentative onset from 6 months to 1 year.
Finally, while we will continue to use response at both months that both months, 1 and 2 following a single treatment for primary efficacy.
We are extending the total observation period out to 4 months to assess durability of the treatment effect.
This is because patients who responded to OTO 313 in the phase 1.2 trial, we're still improving at the end of this study.
We are encouraged by the level of investigator and patient and patient interest in this trial multiple centers are enrolling in the U S and we are making good progress with our site initiation activities in Europe in order to have top line results in mid 2022.
Our next clinical stage program.
Is the OTO 413, a sustained exposure formulation of brain derived neurotrophic factor or bdnf, which is an endogenous nerve growth factor.
This therapeutic approach is highly relevant for the treatment of a broad hearing loss population based on the growing body of evidence, indicating that damage to neuronal connections in the cochlea due.
Due to aging or noise occurred earlier than hair cell loss.
Delivery of beating App to the inner ear can induce neurite sprouting and promote the reconnection of other Tory nerve fibers to hurt sales.
And by repairing these cochlea synapses OTO 413 can improve functional hearing which has demonstrated using speech in noise hearing tests.
During the second quarter, we initiated an expansion of the positive phase 1.2 trial reported in December.
This phase II expansion cohort will randomize approximately 30 hearing loss patients 'twenty will be treated with a single inter tympanic injection of OTO 413, and 10 will receive placebo.
Patients will be followed for 3 months and assessed using the same 3 clinically validated speech in noise hearing test used in the prior cohorts.
As the American English matrix phrase test.
Their words and noise test and the digits and noise test.
In these tests the subject is presented with this set of phrases words or numbers at varying loudness with the constant background noise level that it's typically set to the loudness of a normal conversation.
A significant advantage of speech in noise tests over conventional testing in quiet is that they test overall speech intelligibility mimicking the real world setting for patients who complain that they can't here in a noisy setting.
This is especially important because it's been it has been well established that neither audiometry, nor award recognition inquire predict hearing in a noisy setting.
The enrollment criteria of the expansion study will continue to target a broad hearing loss pop patient population to support the design of the phase III trial, we expect results to be available in mid 2022.
Our third development program is <unk> <unk>, 5 a gene therapy targeting <unk>.
<unk>, our GAAP junction beta 2 <unk>, which is the most common cause of congenital hearing loss.
Patients born with this mutation can have severe to profound deafness in both year that is identified in screening tests now performed routinely in newborns.
Together with our partner <unk>, we presented preclinical data demonstrating that <unk> provides excellent expression.
Web connection 26, the gene product of G JV too in the non sensory cells of the cochlea.
Okay.
In May we achieved an important milestone for this program by presenting preclinical proof of concept results for auto 825 at the American Society for gene and cell therapy meeting.
These results demonstrate.
These results demonstrate the successful recovery of hearing and cookware morphology, and 2 independent mouse models of GE JV to deficiency.
In these models connection 2006 expression is knocked out which results in hearing loss and structural changes of the cochlea that mimic the human condition.
A single interact.
Administration of OTO 825 rescued connection 26 expression in both models.
Furthermore, low 85 induces significant improvement in hearing across multiple frequencies and normalizes cochlear morphology and both of these models.
A summary of these impressive results is provided in the corporate slide deck available on our website.
Yeah.
We're very excited about these results because they validate the therapeutic potential of OTO 825 across a range of hearing loss levels observed in patients and support its advancement into clinical development.
To this end we've also completed a productive pre IND meeting with the FDA. This.
This meeting provided guidance for the design and the IND, enabling non clinical studies.
Requirements for the manufacturing and testing of clinical drug product and helpful considerations for our design of the clinical trial program.
Based on this feedback we have initiated IND, enabling activities and anticipate filing an IND in the first half of 2023.
Our remaining 2 programs our OTO 510 on Ono protectant for patients at risk for cisplatin induced hearing loss and <unk>, 6 X X, which targets hairstyle repair our regeneration for patients with severe hearing loss preclinical.
Development continues on both of these programs.
In summary, we are focused on advancing our multiple programs for treating hearing loss and tinnitus, which represent large untapped markets with significant unmet medical need.
Patient enrollment in the OTO 313, and OTO 413, clinical trials is going well and our ongoing review of results from the prior studies with key opinion leaders is very encouraging.
They support the endpoints, we have chosen and view the results from these trials is demonstrating a clinically meaningful improvement for patients.
We believe that both of these programs are well positioned to address blockbuster market opportunities.
Additionally, we have a first and only company to present preclinical proof of concept results for gene therapy targeting <unk> 2 deficiency, the largest congenital hearing loss patient population.
We have clear evidence from the FDA on what is required to move this program into clinical development and are excited to have initiated our IND enabling activities.
With that I'll turn the call over to Paul Kerr, our Chief financial and business Officer, who will provide a summary of our financial results and plans.
Thank you, Dave and good afternoon, everyone.
As with our development programs. We also made significant progress with our financing and business activities during the second quarter on.
Ill review those updates for you, but first let me recap the second quarter financial results, which are more fully reviewed in our 10-Q filing today.
We reported GAAP operating expenses totaling $12.8 million in the second quarter of 2021, and non-GAAP operating expenses of $10.2 million.
Primary adjustment for non-GAAP expenses is the exclusion of stock based compensation. So this is the financial metric that best approximates our spending level.
These results are on track with our financial guidance for the year that include GAAP operating expense is expected to be in the range of $46 million to $48 million.
Non-GAAP operating expenses of $38 million to $40 million.
A detailed reconciliation of GAAP to non-GAAP numbers can be found at the end of today's press release posted on the Investor Relations page of our website.
From a cash perspective, we finished the second quarter with a cash balance, including cash cash equivalents and short term investments of $97.9 million.
This reflects an underwritten public offering we completed in April that raised gross proceeds totaling $34.7 million.
Following this financing we were able to modify our term loan with Oxford finance to extend the interest only repayment period from the end of this year to June of 2023.
The combined effect of these 2 transactions is to extend our cash runway into the second half of 2023.
1 other business update to note in May we completed the sale of our temporary related assets to our former co promotion partner Al Cabello.
This enables us to fully focus on advancing our multiple development programs for hearing loss tinnitus and eliminates the remaining cash burn we had related to commercial activities.
With that I'll turn the call back over to Dave.
Thank you Paul.
As you can see we are in a strong position with financial runway into the second half of 2023, and our broad clinical and preclinical pipeline targeting the largest patient populations in neuro otology.
Both of our clinical programs have progressed based on promising proof of concept data and we have reported the first preclinical data demonstrating the potential to treat the most common.
Form of congenital hearing loss.
Our focus is on executing against these programs and achieving important clinical catalysts during the coming year.
Operator, we are now ready for questions.
Thank you as a reminder, if you'd like to ask a question simply press Star then 1 on your telephone keypad. Once again Thats star 1 to queue for a question. If you would like to withdraw your question press the pound key.
And our first question is going to come from the line of Stacy <unk> with Cowen and company.
Okay.
Hi, good afternoon, thanks for taking my questions.
Would you be willing to give us an update when enrollment is completed first day 1.
3 and 401 day, and then I have 2 questions on <unk>.
Can you speak a little more to the pre NDA meeting.
Maybe the key consideration for safety.
Think about gene therapy program and particularly on.
Young pediatric or the need for an indication.
Can you talk about what the FDA might require there.
And then as it relates to maybe the best curious thoughts you've seen so far.
The knockout mice, what's your base case expectations regarding durability of effect given that you see.
Gene expression in preclinical models with a single administration. Thanks, so much.
Thank you Stacy first in terms of the enrollment update we will keep investors apprised to our timing to top line results as is our practice, we don't we do not disclose the actual status of enrollment in terms of specific numbers of patients that way all the clinical sites are working towards a common goal of completing enrollment.
But we will keep investors informed as has been our practice on the timing to top line results, which as I've mentioned is on track for middle of 2022, and we have a high level of confidence in that expectation.
For both of US for both of the programs with regard to <unk>. The pre IMD meeting that really focused on a number of areas that we discussed with the FDA. It was very productive.
Meeting.
First and foremost was discussion of our proposed preclinical program in which we did achieve agreement with the FDA.
On that on our proposal to support.
What is our intended patient population.
For this clinical program, which is as you've mentioned is added in the pediatric population.
Our interest is in trying to treat these patients in the early stages of.
Of this disorder, particularly among those who obviously are prelingual in development. So our focus there was in that non clinical program was in that patient population and we did reach.
Agreement on our proposal for that.
We also received.
I think very.
Good feedback on the proposed on the proposed clinical program again coming specifically to the patient population, we intend to treat.
And we had no surprises there I think we felt that the guidance and considerations were very good from the FDA.
And it was really a matter of just details around what we will track.
In the clinical trial.
And then finally, the third part was on manufacturing and very good collaborative interactions there as well.
The manufacturing program.
Of course, it's based on the very solid and improving the experience of our partner AGC and their capabilities in this area and as we had expected.
The FDA was very non.
<unk> about the work that has previously been done by a GTC and the other area and obviously that filter through into the program. So we feel very good about on the manufacturing as well. So I think it was a very productive meeting.
And no surprises from our point it was really mean, mainly just.
Confirmation of what our intentions were on what our proposals were and then just some.
Some additional details that will add to the quality and conduct of the program.
I think your third question was on the rescue on the durability of gene expression clearly this is something that.
We are seeing good durability in the non human primate, we continued to explore that as we continue we.
We have also seen that in the in our preclinical rescue models, we see good duration of effect, but this is clearly something that we will look at it long term in the clinical program as well.
Thank you so much.
Thank you.
Again to ask a question. Please press Star then 1 on your telephone keypad.
And our next question is going to come from the line of Chris Raymond with Piper Sandler.
Hey, Thanks, maybe.
Follow up question on to your answer on day 25, So just I wanted to just clarify.
So.
There is a decent amount of time between now and an IND filing I guess.
With all of the preclinical data you've already generated.
Should we expect maybe.
Presentations between now and when you file your R&D.
I guess, that's the first question on day, 25% and then maybe on 313 just to clarify the phase.
To update you.
The primary analysis I think will be Tsi response at month, 1 and 2.
But I think you follow on these patients out to 4 months when we get that update.
Next year should we expect that longer term follow up.
Initial top line and I guess, what I'm getting at is will that tell us more about potential re dosing frequency.
Thanks, Chris and happy to speak with you so with regard to <unk> <unk> 5 in the IND filing.
And the timeline that we have to there yes, I think we're continuing to do more work in our animal models and as well as on an administration and.
So we're continue to provide updates as we go in and indeed, we're also be presenting at upcoming meetings. So we have more information that will be forthcoming, but I think clearly the the data that we have now on the rescue models is extremely powerful and important obviously demonstrating proof of concept.
In the animal models for this.
For this therapy.
So I think you can expect more from us, but I think the most important data. We think is what we have now presented with this which is the rescue data.
The with regards to $3.13, and the Phase III program you are correct. The tsi at month, 1 and 2 and the responders that is patients who have a 13 point or greater change in the tsi from baseline at month, 1 and at month.
2.
The only sets a very high hurdle 1 that we've heard very positively from from Kols, We think thats, a very significant hurdle to hip.
That will remain our primary.
And we will have that data for the top line.
The additional data that you mentioned, which is looking at patients beyond that second month to month, 3 and 4 is additional data that will be coming as following the top line. We will have some patients likely who have completed their fourth month. So we will provide what we have but that importantly will look at the durability.
The effect and the idea there will be to understand 2 things really do patients continue to get better because that's what the data is telling us from the phase 1.2 that patients were still improving.
Following that single administration. So we wanted to understand what happens to the patients.
Post that single dose because if many of those patients come to mind tentative. The question would be do you even retreat do you even need to retreat.
As opposed to maybe there are patients who do not get all the way to miles.
And there that might set up a re treatment scenario. So I think it will lap actually answer a number of questions for us both in terms of the number of patients.
<unk> MAU.
Tinnitus results from a single administration as well as then are there is there a patient population that requires re treatment and if so what kind of interval that might be at so I think it will be very.
Informative for us.
But again I'm very encouraged by the fact that in the phase <unk>. We saw patients that were still improving following that single administration. So I think most importantly, we got to see what happens with those patients.
Thank you.
Thank you Chris.
Thank you once again to ask a question. Please press Star then 1 on your telephone keypad again star 1 to queue for your question.
And.
No further questions I would like to turn the call over to autonomy Incorporated's, President and Chief Executive Officer, Dave Weber.
Alright, Thank you everyone for participating on our call today, and we hope to meet with many of you at the upcoming Cantor Fitzgerald and HC Wainwright healthcare conferences in September have a good evening everyone. Thank you.
Okay.
Right.
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Ladies and gentlemen, thank you for standing by and welcome to autonomy incorporated second quarter 2021 financial results Conference call. At this time all participants are in a listen only mode. After the speaker's presentation. There will be a question and answer session to ask a question.
During this session you will need to press star 1 on your telephone. Please be advised today's conference is being recorded if you require any assistance press star zero for the operator now it's my pleasure to hand, the conference over to our first speaker today Robert.
Managing director of Investor Relations. Please go ahead Sir.
Thank you operator, good afternoon, and welcome to autonomy second quarter 2021 financial results and business update conference call. Joining me on the call from autonomy are Dr. David Weber, President and Chief Executive Officer, and Paul Cayer, Chief Financial and business Officer before I.
I turn the call over to Dr. Weber I would like to remind you that today's call will include forward looking statements based on current expectations such statements represent managements judgment as of today and may involve risks and uncertainties that could cause actual results to differ materially from expected results.
Please refer to Autonomies filings with the SEC, which are available from the SEC or on the autonomy website for information concerning the risk factors that could affect the company.
Autonomy, specifically disclaims any obligation to update any forward looking statements, except as required by law I will now turn the call over to Dave Weber, President and CEO of autonomy.
Thank you Robert Good afternoon, everyone and thank you for joining us on this call to discuss the economy's recent business updates as well as our financial results for the second quarter of 2021.
We are making solid progress across our multiple programs for tinnitus and hearing loss and the key takeaways from this update includes the following <unk>.
Enrolment for the OTO 313 phase II trial in tinnitus and the other 413 phase 2 extension study in hearing loss are off to good starts with both studies on track for top line results in mid 2022.
We have achieved several important milestones for our own O 825 gene therapy program, including demonstration of preclinical proof of concept in 2 independent models of congenital hearing loss and completion of a pre IND meeting with the FDA we.
We have initiated IND, enabling activities for this program and expect to file on R&D in the first half of 2023.
We're also also continuing to progress our 2 other preclinical programs OTO 510 for OTO protection and autos 6 X X for severe hearing loss.
Finally, we significantly strengthened our financial position during the second quarter by completing a financing in April and modifying our debt facility to extend the interest only period.
These actions provide us with the cash runway into the second half of 2023.
During this call I'll provide a brief update on our programs and then Paul will recap our financial results. We can then open up the line for any questions.
Beginning with OTO 313 enrolment in the phase III trial is going well. The design of this trial is based on the successful phase 1.2 trial that demonstrated a higher proportion of responders in the OTO 313 group versus placebo based on the tinnitus functional index.
Our <unk>.
The phase II will enroll approximately 140 patients with persistent unilateral tinnitus or at least moderate severity based on the <unk> as.
As in the prior trial, where we observed a high correlation between the various metrics on responders. We are also tracking tentative loudness.
And patient global impression of change.
To enrich the study population, we're excluding patients with severe hearing loss, who we believe may be less likely to respond to treatment.
And we have increased the minimum severity of tinnitus required for entry.
We have also expanded the patient population eligible for enrollment by increasing the time from tentative onset from 6 months to 1 year.
Finally, while we will continue to use response at both months at both months, 1 and 2 following a single treatment for primary efficacy.
We are extending the total observation period out to 4 months to assess durability of the treatment effect.
This is because patients who responded to OTO 313 in the phase 1.2 trial, we're still improving at the end of this study.
We are encouraged by the level of investigator and patient and patient interest in this trial multiple centers are enrolling in the U S and we are making good progress with our site initiation activities in Europe in order to have top line results in mid 2022.
Our next clinical stage program.
It's OTO 413, a sustained exposure formulation of brain derived neurotrophic factor or bdnf, which is an endogenous nerve growth factor.
This therapeutic approach is highly relevant for the treatment of a broad hearing loss population based on the growing body of evidence, indicating that damage to neuronal connections in the cochlea due.
Due to aging or noise occurred earlier than hair cell loss.
Delivery of beating App to the inner ear can induce neurite sprouting and promote the reconnection of auditory nerve fibers to hair cells.
And by repairing these cochlea synapses OTO 413 can improve functional hearing which has demonstrated using speech in noise hearing tests.
During the second quarter, we initiated an expansion of the positive phase 1.2 trial reported in December.
This phase II expansion cohort will randomize approximately 30 hearing loss patients 'twenty will be treated with the single engine tympanic injection of OTO 413, and 10 will receive placebo.
Patients will be followed for 3 months and assessed using the same 3 clinically validated speech in noise hearing test used in the prior cohorts that is the American English matrix phrased test.
Their words and noise test and the digits and noise test.
In these test the subject is presented with this set of phrases words on numbers at varying loudness with the constant background noise level that it's typically set the loudness of a normal conversation.
A significant advantage of speech in noise tests over conventional testing in quiet is that they test overall speech intelligibility mimicking the real world setting for patients who complain that they can't here in a noisy setting.
This is especially important because it has been it has been well established that neither audiometry, nor award recognition inquire predict hearing in a noisy setting.
The enrollment criteria of the expansion study will continue to target a broad hearing loss pop patient population to support the design of a phase III trial, we expect results to be available in mid 2022.
Our third development program is <unk> <unk> 5 a gene therapy targeting G. G JV to our GAAP junction beta 2 <unk>, which is the most common cause of congenital hearing loss.
Patients born with this mutation can have severe to profound deafness in both year that is identified in screening tests now performed routinely in newborns.
Together with our partner a GTC, we have presented preclinical data demonstrating that <unk> provides excellent expression.
While the connection 26, the gene product of G JV too in the non sensory cells of the cochlea.
Okay.
In May we achieved an important milestone for this program by presenting preclinical proof of concept results for auto 825 at the American Society for gene and cell therapy meeting.
These results demonstrate.
These results demonstrate the successful recovery of hearing and cookware morphology, and 2 independent mouse models of G JV to deficiency.
In these models connection 2006 expression is knocked out which results in hearing loss and structural changes on the cochlea that mimic the human condition.
A single interact.
Administration of <unk>, 2.5 rescues connection 26 expression in both models.
Furthermore, low 85 induces significant improvement in hearing across multiple frequencies and normalizes cochlear morphology and both of these models.
A summary of these impressive results is provided in the corporate slide deck available on our website.
Yeah.
We're very excited about these results because they validate the therapeutic potential of OTO 825 across a range of hearing loss levels observed in patients and support its advancement into clinical development.
To this end we've also completed a productive pre IND meeting with the FDA. This.
This meeting provided guidance for the design on the buy IND, enabling non clinical studies.
Requirements for the manufacturing and testing of clinical drug product and helpful considerations for our design of the clinical trial program.
Based on this feedback we have initiated IND, enabling activities and anticipate filing an IND in the first half of 2023.
Our remaining 2 programs, our OTO 510, and OTO protectant for patients at risk for cisplatin induced hearing loss and <unk>, 6 X X, which targets hairstyle repair our regeneration for patients with severe hearing loss preclinical.
Preclinical development continues on both of these programs.
In summary, we are focused on advancing our multiple programs for treating hearing loss and tinnitus, which represent large untapped markets with significant unmet medical need.
Patient enrollment in the OTO 313, and OTO 413, clinical trials is going well and our ongoing review of results from the prior studies with key opinion leaders is very encouraging.
They support the endpoints, we have chosen and view the results from these trials is demonstrating a clinically meaningful improvement for patients.
We believe that both of these programs are well positioned to address blockbuster market opportunities.
Additionally, we are the first and only company to present preclinical proof of concept results for a gene therapy targeting <unk> 2 deficiency, the largest congenital hearing loss patient population.
We have clear evidence from the FDA on what is required to move this program into clinical development and are excited to have initiated our IND enabling activities.
With that I'll turn the call over to Paul Kerr, our Chief financial and business Officer, who will provide a summary of our financial results and plans.
Thank you, Dave and good afternoon, everyone.
As with our development programs. We also made significant progress with our financing and business activities during the second quarter.
A review of those updates for you, but first let me recap the second quarter financial results, which are more fully reviewed on our 10-Q filing today.
We reported GAAP operating expenses totaling $12.8 million in the second quarter of 2021, and non-GAAP operating expenses of $10.2 million.
The primary adjustment for non-GAAP expenses is the exclusion of stock based compensation. So this is the financial metric that best approximates our spending level.
These results are on track with our financial guidance for the year that include GAAP operating expense is expected to be on the range of $46 million to $48 million with non-GAAP operating expenses of $38 million to $40 million.
A detailed reconciliation of GAAP to non-GAAP numbers can be found at the end of today's press release.
On the Investor Relations page of our website.
From a cash perspective, we finished the second quarter with a cash balance, including cash cash equivalents and short term investments of $97.9 million.
This reflects an underwritten public offering we completed in April that raised gross proceeds totaling $34.7 million.
Following this financing we were able to modify our term loan with Oxford finance to extend the interest only repayment period from the end of this year to June of 2023.
The combined effect of these 2 transactions is to extend our cash runway into the second half of 2023.
1 other business update to note in May we completed the sale of our temporary all related assets to our former co promotion partner Al Cabello.
This enables us to fully focus on advancing our multiple development programs for hearing loss tinnitus and eliminates the remaining cash burn we had related to commercial activities.
With that I'll turn the call back over to Dave.
Thank you Paul.
As you can see we are in a strong position with financial runway into the second half of 2023, and our broad clinical and preclinical pipeline targeting the largest patient populations in neuro otology.
Both of our clinical programs have progressed based on promising proof of concept data and we have reported the first preclinical data demonstrating the potential to treat the most common.
Form of congenital hearing loss.
Our focus is on executing against these programs and achieving important clinical catalysts during the coming year.
Operator, we are now ready for questions.
Thank you as a reminder, if you'd like to ask a question simply press Star then 1 on your telephone keypad. Once again net star 1 to queue for a question. If you would like to withdraw your question press the pound key.
And our first question is going to come from the line of Stacy <unk> with Cowen and company.
Yeah.
Hi, good afternoon. Thanks for taking my questions first would you be willing to give us an update when enrollment is completed first day, 1.3 and 4 at 1.3 and then I have 2 questions on 2 sides.
Can you speak a little more to the pre NDA meeting.
Maybe the key consideration for safety.
Think about gene therapy program and particularly on.
Young pediatric or the need for an indication.
Can you talk about what the FDA might require there.
And then as it relates to maybe the best curious are you seeing so far.
The knock out mice, what's your base case expectations regarding durability of effect given that you see.
Gene expression in preclinical models with a single administration. Thanks, so much.
Thank you Stacy first in terms of the enrollment update we will keep investors apprised to our timing to top line results as is our practice, we don't we do not disclose the actual status of enrollment in terms of specific numbers of patients that way all the clinical sites are working towards a common goal of completing enrollment.
But we will keep investors informed as has been our practice on the timing to top line results, which as I've mentioned is on track for middle of 2022, and we have a high level of confidence in that expectation.
For both of US for both of the programs with regard to <unk>. The pre IMD meeting that really focused on a number of areas that we discussed with the FDA. It was a very productive.
Meeting.
First and foremost was discussion of our proposed preclinical program in which we did achieve agreement with the FDA.
On that on our proposal to support.
What is our intended patient population.
For this clinical program, which is as you've mentioned is added in the pediatric population.
Our interest is in trying to treat these patients in the early stages of.
Of this disorder, particularly among those who obviously are prelingual in development. So our focus there was in that non clinical program was in that patient population and we did reach.
Agreement on our proposal for that.
We also received.
I think Barry.
Good feedback on the proposed on the proposed clinical program again coming specifically to the patient population, we intend to treat.
And we had no surprises there I think we felt that the guidance and considerations were very good from the FDA.
And it was really a matter of just details around what we will track.
In the clinical trial.
And then finally, the third part was on manufacturing and very good collaborative interactions there as well.
The manufacturing program.
Of course, it's based on the very solid and improving the experience of our partner AGC and their capabilities in this area and as we had expected.
The FDA was very non.
<unk> about the work that has previously been done by a GTC and the other area and obviously that filter through into this program. So we feel very good about on the manufacturing as well. So I think it was a very productive meeting.
And no surprises from our point it was really mean, mainly just.
Confirmation of what our intentions were on what our proposals were and then just some.
Some additional details that will add to the quality and conduct of the program.
I think your third question was on the rescue on the durability of gene expression clearly this is something that.
We are seeing good durability in the non human primate, we continued to explore that as we continue we.
We have also seen that in the in our preclinical rescue models, we see good duration of effect, but this is clearly something that we will look at it long term in the clinical program as well.
Thank you so much.
Thank you.
Again to ask a question. Please press Star then 1 on your till the final key pad.
And our next question is going to come from the line of Chris Raymond with Piper Sandler.
Hey, Thanks, maybe a follow up question on to your answer on day 25, So just I wanted to just clarify.
So.
There is a decent amount of time between now and an IND filing I guess.
Yeah.
With all of the preclinical data you already generated.
Should we expect maybe more presentations between now and when you file your R&D.
I guess, that's the first question on day, 25% and then maybe on 313 just to clarify.
Phase.
To update.
The primary analysis I think will be Tsi response at month, 1 and 2.
But I think your follow on these patients out to 4 months when we get that update.
Next year should we expect that longer term follow up.
The initial top line and I guess, what I'm getting at is will that tell us more about potential re dosing frequency.
Yeah, Thanks, Chris happy to speak with you so with regard to <unk> 5 and the IND filing.
And the timeline that we have to there yes, I think we are continue to do more work in our animal models and as well as on the administration.
And so we're continue to provide updates as we go in and indeed, we're also be presenting at upcoming meetings. So we have more information that will be forthcoming, but I think clearly the the data that we have now on the rescue models is extremely powerful and important obviously demonstrating the proof of concept.
In the animal models for this.
For this therapy.
So I think you can expect more from us, but I think the most important data. We think is what we have now presented with this which is the rescue data.
The with regards to $3.13, and the Phase III program you are correct the tsi.
At month, 1 and 2 and the responders that is our that is patients who have a 13 point or greater change in the tsi from baseline at month, 1 and at month 2.
Not only sets a very high hurdle 1 that we've heard very positively from from Kols, We think thats, a very significant hurdle to hip.
That will remain our primary.
And we will have that data for the top line.
The additional data that you mentioned, which is looking at patients beyond that second month to month, 3 and 4 is additional data that will be coming as following the top line. We will have some patients likely who have completed their fourth month. So we will provide what we have but that importantly will look at the durability of.
The effect and the idea there will be to understand 2 things really do patients continue to get better because that's what the data is telling us from the phase 1.2 that patients were still improving.
Following that single administration. So we wanted to understand what happens to the patients.
Post that single dose because of many of those patients come to mind tentative. The question would be do you even retreat do you even need to retreat.
As opposed to maybe there are patients who do not get all the way to miles.
And there that might set up of re treatments scenario. So I think it will lap actually answer a number of questions for us both in terms of the number of patients.
<unk> MAU.
Tinnitus results from a single administration as well as then are there is there a patient population that requires re treatment and if so what kind of interval that might be at so I think it will be very.
Informative for us.
But again I'm very encouraged by the fact that in the phase <unk>. We saw patients that were still improving following that single administration. So I think most importantly, we got to see what happens with those patients.
Thank you.
Thank you Chris.
Thank you once again to ask a question. Please press Star then 1 on your telephone keypad again star 1 to queue for your question.
And so on.
No further questions I would like to turn the call over to autonomy Incorporated's, President and Chief Executive Officer, Dave Weber.
Alright, Thank you everyone for participating on our call today, and we hope to meet with many of you at the upcoming Cantor Fitzgerald and HC Wainwright healthcare conferences in September have a good evening everyone. Thank you.