Q2 2021 Axcella Health Inc Earnings Call
[music].
Okay.
Good morning, ladies and gentlemen, and welcome the book Sellers second quarter of 2021 conference call. Please be advised of today's.
It's called the being recorded in the all participants will be in listen only mode until the question and answer session. After todays presentation well begin the opportunity to ask questions to ask a question you May Press Star then 1 on your Touchtone phone.
Withdraw your question, Please press star and true.
And now for opening remarks, and I would like to call the handle.
Mr. Jason <unk>, Vice President of Investor Relations and corporate communications ex all of them. Please.
Please go ahead.
Yeah.
Thank you very much operator, and good morning, everyone. We would like to advise that certain remarks, we will make on today's conference call such as those relating to our ongoing.
The trials of <unk> of $16, 65% of $11.25, as well as our pipeline expansion work include forward looking statements that are subject to various risks and uncertainties. These.
These risks and uncertainties are detailed in our most recent form 10-Q, and our other SEC filings, which can be accessed on our web site <unk> dot com or on the SEC.
Ccs website all forward looking statements represent our views as of today July 29, 2021, and should not be relied upon as representing our views as of any subsequent date, we undertake no obligation to update these forward looking statements with that let me turn the call over to our president and CEO of Bill Hinshaw to begin the discussion.
<unk> clinic Bill.
Thank you, Jason and thanks to all of you who are of turning in for our update. This morning, we're happy to have the opportunity to bring you up to speed on all of that we've accomplished recently and our core areas of focus for the months ahead.
To briefly recap some of the highlights from the first half of 2020.
1.
Following a set of successful regulatory exchanges and meetings, we cleared 2 investigational new drug or IND applications with the FDA and got the Green light to proceed directly into a phase III clinical trial with acts of $60.65 per OAG and of phase 2 b.
For acts of $11.25 for Nash, we also obtained similar clearances from multiple health authorities in Europe, and Canada, enabling us to advance our global development efforts.
And in the second quarter, our team was able to initiate the both of our trials I am very pleased to report that patient recruitment is.
It is now well underway, putting us in a strong position entering the back half of the year.
So let's get into some of the details beginning with the status of our development program for acts of $16.65, $60.65 is our product candidate for the reduction in risk of recurrent overt hepatic encephalopathy or <unk>.
The severity of this condition and the unmet needs for patients who experiences are truly underappreciated and we believe we're uniquely positioned to make a real difference.
<unk> is a serious neuro cognitive episode that results from cirrhosis patients in the midst of these crises.
Paired to the point, where they have difficulty carrying for themselves and may need to be hospital loans.
These events May also lead to coma and even death.
Now of <unk> involves the cascade of events given the cirrhotic state delivers unable to handle 1 of its primary functions, which is the process.
And detoxify waste products such as ammonia.
As a result muscles within the body tried to assist in the detoxification process, but they do it poorly and end up wasting themselves the.
This results in another serious complication known as <unk>.
Meanwhile, toxins within the blood.
Our <unk> continued to increase and cross the blood brain barrier, causing recurrent neuro cognitive episodes.
The day patients who have experienced the <unk> tend to be treated first with <unk>, which is the laxative Sara.
<unk> is administered orally multiple times of day in order to remove.
Blood stream from the gut via multiple loose stools per day.
And as you might expect there are a number of well understood challenges related to this treatment with a lack of compliance and tolerability of being at the top of that list.
Also nearly 50% of patients on <unk> alone has been shown to experience.
Ammonic through within 6 months.
The other primary treatment, which is approved as an adjunct to latch Lowe's is rifaximin Sim.
Similar to latch lowest rifaximin focuses on ammonia removal.
Unfortunately patients continue to experience OAG breakthrough, even while on combination therapy.
We believe that this is due in part to the fact that these treatments don't in dress, the imbalanced and amino acids and <unk> that are also associated with the condition.
This is where access of $16.65 comes in.
Unlike today's standards of care, which focus on removing ammonia from the gut.
$60.65 has a multi targeted mechanism of that focuses on the gut the liver and the muscle.
It seeks to improve neuro cognition by not only improving ammonia and nitrogen handling, but also restoring amino acid homeostasis and halting the progression of muscle wasting.
Now we've already completed 2 clinical studies in which we were able to demonstrate activity in each of these areas with a very safe and well tolerated profile.
And we were pleased to have Dr. Arun Sonangol, 1 of the world's preeminent chronic liver disease specialists deliver an oral presentation containing data from our most recent study.
<unk> at digestive disease week or <unk> W..2021 in May for.
For those who are interested of video of this presentation is now available on the publications section of our website.
Now these data provide a strong foundation for our M power of Phase III trial.
Empower.
Which starts with an E and double EMS is the branding of this trial because we are leveraging the endogenous metabolic modulators or <unk> with $60.65 in an effort to enable patients and caregivers to overcome the significant challenges that they face today.
Patients.
Training and empower got underway in June and I'm pleased to report we have already initiated a dozen clinical trial sites in the months ahead, we plan to increase this number to more than 70 sites across 9 countries in total.
Actually all of which have already been selected.
Approximately 150 patients who.
Who are unmatched <unk>, plus or minus rifaximin will be enrolled and randomized equally to $60.65 per placebo for a 24 week treatment duration.
We will also stratify patients based on Rifaximin use.
As a reminder of the primary endpoint in the trial as the proportion of patients who achieve at.
At least a 2 point improvement in Phs, which is the neuro cognitive battery that is considered to be the gold standard for diagnosing minimal hepatic encephalopathy key.
Key secondary endpoints include the number of <unk> events timed OAG events, including hospitalization and measures of physical function.
These endpoints should enable us to gain clear insight into 16, 60 five's effects and guide the next steps in its development.
We look forward to continuing our engagement with the medical community and advancing this important program.
So let's shift gears now and discuss our other lead candidate Axa.
11, 25, $11.25 is advancing in parallel as a potential treatment for Nash, which is another serious liver disease that impacts up to 40 million people here in the U S alone.
This differentiated multi targeted composition of the <unk> seeks to improve.
Metabolism, and reduce both inflammation and fibrosis by influencing well known pathways that have been implicated in the pathogenesis of Nash.
Some of these include <unk> Alpha.
K <unk>, 1 alpha and TGF beta.
In 2 prior studies, we have demonstrated.
11, 20, five's ability to reduce liver fat insulin resistance and fiber of inflammation across a range of well established biomarkers and enhanced effects have been seen in subjects with type 2 diabetes, which is of closely associated condition that affects 40% or more of Nash.
Patients.
We were pleased to share findings about the sizable and important subpopulation at the American diabetes Association's 80, <unk> scientific sessions, just a few weeks ago.
And based on our past data and encouraging dialogue with regulatory authorities, we were able to get our impact.
The phase <unk> clinical trial underway in the second quarter.
Just 1 month after opening our IND.
This is the result of strong planning and execution from our team and the high level of interest from investigators similar.
Similar to empower the double impact branding is a reflection.
Of $11.25 potential to provide profound benefits to patients with this chronic disease using <unk> with well established safety.
To date over 30 clinical sites have been initiated across the U S and Canada. This number will be increasing to more than 70 global sites virtually all of which have already.
<unk> selected.
Ultimately the trial will enroll approximately 270 patients who will receive 1 of 2 doses of $11.25 or placebo for a 48 week treatment period.
Consistent with regulatory guidance this will be of paired biopsy trials.
The primary endpoint.
Impact is the proportion of patients achieving at least the 2 point improvement in the Nash score. We will look at the achievement of biopsy confirmed resolution of Nash without worsening of fibrosis and the achievement of at least a 1 stage improvement in fibrosis without worsening of Nash as the secondary endpoints.
And Additionally, a range of noninvasive Biomarkers are being included in the trial.
These will be utilized for an interim analysis after a meaningful number of patients reached 24 weeks of treatment, which will provide progressive data compared to our past 12, and 16 week studies.
We expect to be in a position to read.
The interim analysis in mid 2022.
Now in addition to all of the clinical progress Thats been made our R&D group has been hard at work to define the next potential applications for our <unk> platform and candidates. We're looking forward to sharing more detail about these efforts later in the year.
And finally, we have continued to bolster our team of <unk> 1 of our most recent additions is Virginia, Dean who joined as our Chief people Officer in June.
Junior is leading our human resources function as well as our organizational and cultural development initiatives.
She joins us from the consulting firm.
Firm clear site leadership and has led the HR functions for multiple high growth organizations, including both Tesoro and area of Pharmaceuticals, we're thrilled to already be benefitting from our insights and contributions.
So the first half of 2021 has been a time of tremendous progress here.
Our team continues to execute on or ahead of plan as it relates to empower and impact and we're seeing strong inbound interest in our candidates and trials. We're also readying the company for pipeline expansion as we seek to unlock the potential of <unk> across multiple areas.
Now, let me turn the call over to our CFO, Ron <unk> to provide a financial update.
The wrong.
Thank you Bill and good morning, everyone.
I would like to equal the Bill's comment about the team's strong execution and know that the extent to a financial as well as we continue to manage our.
Our it expenses and maintain a solid balance sheet.
We ended the second quarter was approximately $71 million in cash and marketable securities.
This compares with 170 and the law as of December 31, 2020.
We expect that this cash balance will be sufficient to meet all of operating need into the third quarter of.
Of 2022, while also enabling us.
To advance our empower and impact trials readout of interim data from manpack and expand our pipeline.
Turning to the income statement, our research and development expenses were $10.3 million and $20.5 million for the 3 and 6 months.
Expect June 32021.
This is up from $8.6 million and $18.9 million for the comparable periods of 2020, reflecting the recent initiation of our empower and the impact clinical trials.
G&A expenses were $4.9 and $9.2 million.
For the 3 and 6 months ended.
And the 32021 day.
It is up slightly from 4.6 and $8.7 million the law for the same period of 2020.
These increases were primarily the result of greater noncash stock based compensation and benefit of related cost.
<unk> net loss for the quarter and the 6 months ended June 32.
Ended June 1 the 1 was $15.9 million of <unk> 42 per share and $31.1 million or 83 per share respectively. Our net loss for the second quarter included approximately $1.7 million in noncash stock based compensation expense, our net loss for the 3 of 6 months.
2000.232020.
Was $13.9 million of 48 cents per share and $28.9 million of $1.10 per share.
That concludes our formal remarks now operator would you. Please open the line for questions.
We will now begin the question and answer session.
Ask the question you May Press Star then 1 of them you touched on phone.
Using a speakerphone please pick up your handset before pressing the keys.
Of all of your question. Please press Star then 2 this.
This time, we will pause momentarily to assemble the roster.
First question comes from Yasmine range.
Hypersound low please go ahead.
Felicia. Thank you so much from taking a question Julien today.
So what is the current screen failure rate in the <unk>.
Hi study and when do you expect to report out data per day.
And our second question.
Regarding the non invasive biomarkers.
Recently, we thought it's Gordon between the noninvasive test and the histological end points.
And Jim's alpine data set.
Agents will be you can highlight to us why we should not lose trust in the predictive value of these noninvasive tests and which line in Europe.
Given the MLA.
It's not predictive of histological responses in Nash as we head into the interim results in mid 2022.
Thank you.
Alicia Thank you for the questions I'll start and then I'll turn to Margaret supplement a 1 or 2 of the comments there. So in terms of the screen failure.
We're very pleased with the start of our trials.
But I'll remind you that we just initiated them.
And so consistent with what we've provided you guys before we won't be providing real time updates from that level of detail.
Especially at the early stages of the trial that's underway.
Here we.
We're very pleased with the response that we've seen we're very pleased to have more than 30 sites already initiated in this trial.
Anecdotally, it's great when our principal investigator tells us that investigators are calling them and wanting to get into the trial.
We've been very thorough in how we are examining.
And working with the fact that we have an opportunity to appropriately guide the patients as to which patients should come in use of the right algorithms used the right training and work with groups like the summit network that are very experienced in that so we've already considered and planned for an appropriate screen failure rate and are very pleased.
At our start on that piece in terms of when we expect the data to come in.
This is of 48 week trial and so our initial estimate at this stage is that the second half of 'twenty, 2 will complete enrollment and that puts us in a position to utilize top line data in the second.
Pleased with of 'twenty 3 now in terms of the aspects related to the.
Of the noninvasive markers and other elements of all integrate that because we do plan a 24 week interim analysis middle of next year and we will of course provide an overall update on the enrollment and trajectories that will be doing as.
Hal becomes more advanced at the appropriate time now in terms of the noninvasive markers. So I think there is some general comments here and then the specific situation. This is an area that you guys follow very closely and as we know there are a series of noninvasive markers that have indeed been correlated.
Related to clinical outcome in the emerging that's the MRI pdfs of the L. T along with CET, 1 and a few other measures now the MGM will always refer you to the company that ran the trials et cetera for those aspects. What we can share is that the endpoint in that measurement time.
Was quite early for fibrosis, and so of the anticipation is that that may have been premature in terms of time to see an impact on that type of disease state and it's 1 data point across a number so I'll pause there and asked Margaret if she would like to add any additional context on the noninvasive marker.
<unk>.
Alright. Thank the this is Mary cause the Allenby P of clinical development.
Let me just and pull it back up and today and we all understand the liver biopsy are problematic inherently as an end point and assessing histology too early at some companies have done is kind of against the F. D a guidance.
On this matter. We also look forward to contributing the ongoing and very rich discussion about which noninvasive testing.
Only predict clinical outcome and at the heart of it that's what is missing there. There are a number of groups that are working on that we look forward to the <unk>. We felt we have worked with investigators to collect a rich sweet of noninvasive cats that we believe at the end of the day will be more useful to practicing physicians.
And thank you Margaret and Alicia just to make sure I answer the right question on the screen failure rate was that 4 empower impact just to make sure cause I answered it more for the impact.
Yeah. It was originally asking you that empowers sorry about that okay. Great. Yeah. So let me clarify the the same upfront answer we're pleased with the engagement and the response an hour of proceeding with the population in terms of the timing of that this is the 24 week trial and we are looking at completing the enrollment around the third.
Quarter of next year with a top line day to read out in the first quarter of 23 now this is a N O H E and we it's our first experience recruiting N O H E. We have again, great engagement with the medical community, there's a high unmet medical need here, there's a high.
<unk> for our mechanism and so the medical community is very interested in looking and working with these and we're working with a series of of World leading experts in this field. So we we will update you again on the Timeframes of that so apologies for the.
You got more answers then you ask for help with that way [laughter].
Okay. Thank you.
Thank you. The next question comes from a R. C. H C Y Great and company. Please go ahead.
Hi, good morning, and congratulation all of the progress so far this year.
A couple of questions for me I'll start with.
Power.
For O H E.
As you mentioned in your remarks the.
The primary endpoint is at least a 2 point of improvement N. P. H, yes, I'm wondering if you could walk us through both of the.
The.
The the.
The amount of.
A line that you have with the F D a on that as well as prior data that substantiates.
That well known Uhm biomarker.
Okay. Yeah. So let me ask of Alison Schechter, our president of R&D to address those questions that thank you and thanks for the comments Alison Yeah. Appreciate the question.
In terms of of the FDA, we have confirmed this design, including the statistical assumptions with the FDA.
The trial is powered adequately to assess the primary endpoint at change in th yes.
Powering level of that is consistent with most of their phase 2 trials and we're leveraging as you alluded to our past experiencing data regarding the phs and they're assuming of benefit phs in the placebo group.
In our previous study we did see in the 12 week study.
Mystically significant change and ph share so we're confident.
That our assumptions.
Great.
Helpful and then.
A few questions on the face too.
<unk> study you mentioned the interim analysis in mid 2022.
So 3 questions there first.
Walk us through exactly what you expect to report.
The second why was the primary endpoint.
Of the 2 point of improvement in math score.
Chosen.
Piece of the.
F D a guidance.
Think there's probably some questions around that especially given that you also have the 2.
Uhm sure get endpoints as well that you want to measure.
And then the thirdly uhm given that this is an interim just to be clear is there any capacity to make any adjustments to the trial.
As a result of the day to day you collect thanks.
Ellison so.
Neither of very mindful about.
The the endpoint that we should cherish.
Great or equal to 2 point changing nats is the standard histologic benchmark at that price.
TB trials in this field and we believe that the primary and secondary endpoints biopsy confirmed resolution of Nash without worsening of fibrosis.
Correct.
Later or equal to 1 stage of proving of hypothesis without worsening of Nash from this trial the will enable us to define.
And.
Fishing and.
Powered phase 3.
You also asked about the interim analysis and what we'll be looking at the end and not Kate will be looking at the well characterized.
Noninvasive Biomarkers.
Bill mentioned that of users.
Invalidated to associate with histology, such as Bbss lived the last cause of fee and others share in Biomarkers standard keying the pathogen process.
And I think <unk>. Thank you Ellison and lastly in terms of add what will we use that data for it will help inform our go forward programs because as you know we're stratifying for type 2 diabetics from that population. It will help us Allison said help us prepare for the phase 3 in an efficient.
Way it will help inform us related to the dose response, because we have 2 active doses there the anchor dose as well as the.
<unk> dose that we're looking to evaluate and can help prepare us for pediatrics in combination options as well so in terms of ability to adjust the trial itself debt will depend on what we find in what we want to do but it helps really set us up in the foremost for expanded opportunities in this important field.
Great. Thank you Bill now appreciate it.
Thank you.
Thank you. The next question from Jessica from J P. Morgan. Please go ahead.
Hey, guys. Good morning, Thanks for taking my questions. It sounds like screening is going well in the states to be.
I guess, specifically for the Nash trial, which kind of cheese or you you using to encourage patients to enroll in a pair of biopsy study and.
In the context of the current Covid situations and second how should we think about the trajectory for R&D expenses from the roughly 10 million run right in the last couple of quarters as you progress through these these to the trial.
Okay. Thank you just some good morning, thanks for the confidence I'll start ask Margaret to comment a little bit on the Nash and then the Rand on your second part of your question. So overall in terms of Covid itself well, we're very cognizant of it we have been working with the sites and so far we're seeing limited the no impact on.
The pandemic.
Combination of the fact that the centers have had to work through this over the last year plus the fact of course of the vaccination rates that of ongoing and then we're in a great position to participate here now in terms of how we are strategies I think macro wise, what we've heard from the investigator.
<unk> and from the patients as they loved the concept of our mechanism you have a endogenous metabolic modulator of composition that is designed to work with the body's system that is oral dosing, but the date has demonstrated safety and Tolerability and in fact is something that.
They see as a way to restore their body systems. So you combine that with the techniques that Margaret and the team of put in and so far we've had an excellent start an engagement as I mentioned in 1 of the earlier questions. It's always encouraging when you hear from the principal investigator of that other sites are <unk>.
Following them and saying they want to be part of that trial because of what their understanding of the science and the profile of.
<unk> is there anything detail wise on the on the specific strategies that you guys are in flooring that had been effective today.
No I would I would definitely your comments completely of that the investigator enthusiasm.
In turn it communicated to potential subjects in the trial.
That has been in very strong cost price and adding up the study and of course, we are monitoring the COVID-19 situation clearly and half mitigation plans in place.
Particularly once the trial gets ongoing so we're we're well prepared for that everyone understands that the situation is fluid in terms of COVID-19 in clinical trials in general.
Yeah, I think it goes back to the fact that when we first share the data they spontaneously saw the benefit of this as a potential strong first line agent because of the profile I described and see the need there the raw.
Just the second questions feel free.
Just so to address the financial questions.
Half <unk> managing of resources, so far I mentioning of very.
Strong financial position.
We are not providing express the guidance, but what I can say is that R&D expensive will ramp going forward as we get the Charles ongoing again has been mentioned, we just started the initiation of these trials. So we have to see.
How the patient of wood enrolled at what speed Jenny will remain roughly flat in the near term. So we expect that the cash would be sufficient to fund us to final operation to the third quarter of 2022, which would get us beyond key milestone in 22, including also the extension of pop pipeline.
Thank you.
Ex.
Thank you and the next question comes from mainland and Tammy of the rally Securities. Please go ahead.
The the mining beam congrats on the draw goods and thanks for taking the question. So 2 quick follow ups. The named <unk>, and then add 1 and back so.
The standard of care ploy, but the.
The end dollar study could could you just touch on what the the.
The primary endpoint of them soon die of would that be a G. S.
It's relatively unknown studied domain knowledge, but the.
Got any standard of get them, then what expectation if any of you should have on the.
The information you've gathered on the 2 episodes could you made the comment on that.
Sure. This is great quest.
Question.
Powering the trial.
Adequately to assess the primary endpoint, which is the changing phs, where the powering level of that is consistent with other phase 2 trials of we're leveraging our past experience, where we showed in 12 weeks of statistically significant improvement at Phs and are assuming of benefiting phs.
In the placebo group.
We have confirmed this design, including the statistical assumptions that the FDA.
This is the.
Based on the fact that.
We are looking at the patients.
And enriching them for these patients are even.
The more impair these patients have the base line.
At Phs of -4 with cognitive underlying kind of interest regulation and they're also I already have had by enrollment criteria.
Break in the event in the last 6 months since enrolling so we day based on that we feel that will not only.
Hit our primary endpoint, but then our secondary endpoints of team.
She.
Then.
Will be enriched and this will help us design, our regulatory endpoint LHE breakthrough in our phase III.
And my ex just the build on that I think if if the.
The component of your question with the standards of care related to the Phs. So as you are aware of LHE is the clear.
The sizable market growing inmates as an even larger population and continuing to grow what.
What we did the build on Allison's point about looking at the impact where we had the statistically significant impact we had a greater than 2 point improvement that is comparable to the known data of the standards of care in our previous trial. So we not only have everything that Allison said about the power in in the modeling based on.
That impacts we know that having that 2 point improvement, which we saw in the majority of our high dose subjects and just the 12 weeks is comparable to the understood. The standard of care in that population. So and then the breakthrough part of this is even in the registration trial, the best or the 4 rifaximin.
Facts menu saw almost 50% of patients breaking through on the <unk> mono therapy within 6 months and 22% on combination therapy. So as Alison the outline we got and enrich patient population and we know even in that setting there's breakthrough. So we're confident of our ability to it.
Test and evaluate our chance to make a difference on top of that and stratify for those 2 arms.
Oh, Great day I appreciate the details of it and then on the impact study the mass study the the.
<unk> on the mask or.
An equal weeks isn't something that you see I didn't do a lot.
The couple of example, the deadline you've already discussed this.
Maybe wall to the rationale, but not just the diet blame but also like.
If you can comment on the methodology of how Ya doing biopsies.
The different methodology as you know in any of the fields knowledge is advancing on that and if you can comment on the effects expectation day. All this the again of factoring in the end.
Understanding the having the placebo those funds.
From from your mortgage line Spears, Thanks, again for taking the question.
Yeah. So I'll start with the first question and then turn it to Allison for the next 2 so in terms of of the 24 weeks, Mike here familiar with our program. We've already demonstrated activity across all of these areas and 12 and 16 weeks of data. So we were looking to evaluate a progressive.
Set of information with 24 weeks and this INV setting. Additionally.
Our hypothesis is because of our multi targeted mechanism that we will continue to build our compound positive effects across the different mechanisms. So you're familiar of course with the quality of measures that we utilized in our previous studies, we want to add to that and build on that and the.
That's why we're choosing the 24 week data period at this point in time. It also goes to 1 of the earlier questions. We share. So that's the rationale for the 24 weeks and then is Allison said of the previous question will be looking at the key markers that allow us to understand and plan going forward now in terms of the aspects of bio.
Seeing effects size Alison when we comment on that and I just wanted to go into of.
A little bit is that.
The greater than 2 point change in the next.
Standard hit the logic.
Benchmark used for phase 2 trials across the field and we don't want to make the mistaken.
Given the heterogeneous genius Khedive, we don't end with fast progressing slower progressive and we don't know how to cheat. Each we wanted to make sure that we give enough time for our drive to share with histologic change and we believe that the primary and secondary endpoints the <unk>.
Biopsy confirmed resolution of Nash without worsening of fibrosis with the greater equal to 1 stage improvement of fibrosis without worsening of Nash from the travel enable us to design inefficient phase 3 in terms of of the discussion of biopsy, which is again.
The question is an octave actively discussed.
Question in this field.
The well known that in the US now studies of placebo effect, which is likely due to better of hearing Smith prescribed medications and diet is most patients remain enrollment of trial on top of the inherent intrinsic variability of biopsies were asking patients to adhere to a baseline diet were taking a diet.
Diaries and monitoring all of these variables and impact.
In addition, we are building a comprehensive set as we mentioned in the Biomarkers, which will likely guide clinical practice in the future and from the hit the logic perspective once completing the full biopsy analysis. The central pathologist, who is 1 of the Premier Nash hit the pathologist in the world will be re reading of representative.
Sample of the biopsies to evaluate intermediated variability and ensure reliability.
Kind of the effects us I think was the other question.
Alright.
Yeah.
As we covered I think in terms of the aspect of the powering and now we've got that with the FDA right. Yeah. So this is all.
Our our plans in terms of.
Pathologic can histologic changes have been well reviewed with the F b and they're lining of the fees of the Single-blind essential pathologist.
And as I said, we're going to be looking at.
Representing the portion.
To be reread that.
Of course, all of these and have been on line, but the FDA.
The thank you put all of the detail and the thoughtful answers. Thank you.
Thanks, Mike.
Thank you and the next question comes from came of K of.
Please go ahead.
Yes. Thank you.
Just the.
Terms of the empower study of the clinical site selection.
Talk to us about how.
How the specialist.
Diagnosing and treating the patients.
Are captured to feed those patients into your clinical sites.
Yeah. That's a great question I mean, obviously this is a.
Rare disease for LHE.
And it takes specialized doctors.
And tertiary care of centers to do the therefore of where the vast majority of our site. In this trial will be in March of academic tertiary centers just to address that point.
Uh-huh.
About 1 third of the sites will be in the us with the remainder of of Europe.
Okay and.
Is there also a.
The equivalent network.
That you have with Nash.
It helps to facilitate this.
Yeah, I mean, we've been working with the leaders in the field in in this.
Sector has the does have some.
Impact in.
<unk> as well and we're new thing 1 of his site.
For the study as well, but we have a large K O L network.
8.4 LHE anywhere ex.
Putting that to make sure that we get the right patients in the right Center.
Okay. Thanks.
Thanks, Thank the reinforcement here again I'll go back to the differentiated mechanism behind the medical need here.
We have had a lot of inbound interest on this and building on Allison's comments about these tertiary care centers in the notes ohh patients are largely in the system, meaning that they are diagnosed there from your care and when you get the sites on Board then.
Then they tend to have a larger number of patients per site that are eligible for clinical studies and we're recruiting for 150 patients across 70 sites globally. So we were excited by the engagement in the initiation area of the day and I think it's something that Bill mentioned.
We're really getting inbound interest from the investigators with the which then goes to the patients about going beyond removing ammonia, which is the standard of care for Bolton Saxman and Lactulose, we're doing of Multimodality mechanisms of the vaccine so not only the expecting.
Check on.
Neurocognitive, but also our other effects in terms of our.
Our secondary endpoints of improving muscle mass and frail team.
That occurs in the patients and that also is associated with increased morbidity and mortality.
Okay. Thank you.
And then I'll be able to question. Please press Star then 1.
The next question comes from Paul Choi Goldman Sachs. Please go ahead.
Hi, everyone. Congratulations on the progress of all of that.
The others et cetera regarding the initiation of of the triumph. This is Charlie on for Paul. We just have 1 quick 1 also regarding recruitment of the empower study.
And I'm, sorry that it's the Covid related question, but the Covid backdrop is obviously much improved over the course of this year, but if we go back to 24 weeks ago things from.
What can a lot more different and so if we're considering OAG patients that have had.
Richie events within the past 24 weeks do you think there are any possibilities for the lingering impact of the Covid environment 24 weeks ago with presenting of difficulty in terms of of identifying patients who have had these events around that time. Thank you very much.
The Charlie Thank you for the question and the also the level of sophistication of of the short answer is we have of course, the and coordinating with the centers and as was alluded to in a previous question. The user academic tertiary care centers, who are very accustomed and used to managing their patients and a number of.
<unk> of.
The vehicles, whether that telemedicine, whether that's home health, whether that is having them come in and the structure of elements the.
<unk> events are well documented because these are circumstances, where it requires a visit a change of therapy.
Change of care so from that standpoint, we have not seen that particular issue be of concern.
And as you alluded to the timing of this has actually worked out very well for us in terms of the progress of that's made on the pandemic and how the senators manage it. So we will monitor it and of course as you said, it's dynamic but to date of what we.
We have seen and heard his continued progress in the center.
Awesome glad to hear it thanks, so much.
Thank you Charles.
Thank you next question comes from Andreas other rides Wedbush Securities. Please go ahead.
Well. Thank you operated the morning team congrats on the corner as well since the quick 1 per months can you tell us what.
Any of that you have on.
The discussions with the FDA regarding pediatric Nash and then also when can we expect an update on the next <unk> product candidate. Thanks.
Okay. Thanks under aspects of the compliment.
Compliment in on the progress in terms of Peed Nash's, we've communicated before our first per.
Objective was to really get the phase to be up for the adults and then the re engage with the agency of the right time to talk about the optimal strategy to move forward as we all know Unfortunately this is a high growing need with 1 in 10 Americans shoulder and already considered to have Nash, there's very limited development.
We believe our profile, including our mechanism of safety and dosing set us up well for that and we look forward to updating you at the appropriate time on that.
Of regarding the next step in the company in terms of additional opportunities. We're very excited about the work that are R&D team has done the learnings that we've had from the major number of of studies that we've taken end of the company and we will be updating you guys. Later this year about the next steps that will be taking in terms of the.
<unk>, the learnings and the Biology's that we want to affect so we look forward to that and Jason of course will be reaching out to schedule that of the right time.
Great. Thanks, Congrats again.
Thank you Sir.
This concludes our question and answer session and I would like to turn the conference back over the CEO the holding Shaw. Please go ahead.
So thank you everyone for your time. This morning, we are certainly looking forward to the start of the conference Circuit. This fall and hope to see everyone. In person soon until then please enjoy the rest of the summer and operator of this concludes our call. Thank you everyone.
Well first of all concluded. Thank you for attending today's presentation you may now disconnect.