Q1 2022 Anavex Life Sciences Corp Earnings Call
Speaker 1: fiscal 2022 first quarter conference call. My name is Clint Tomlinson, and I will be your host for today's call.
2022 first quarter conference call. My name is Clint Tomlinson and I will be your host for today's call.
Speaker 1: At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session. And during this session, if you would like to ask a question, please use the Q&A box or raise your hand. Please note that this conference is being recorded.
At this time all participants are in a listen only mode. Later, we will conduct a question and answer session and during the session. If you'd like to ask a question. Please use the Q&A box or raise your hand.
Please note that this conference is being recorded.
Speaker 1: The call will be available for replay on Anavex's website at www.anavex.com.
The call will be available for replay on <unk> website at Www Dot <unk> Dot com.
Speaker 1: With us today is Dr. Christopher Misling, President and Chief Executive Officer, and Sandra Bohnish, our Principal Financial Officer.
With us today is Dr. Christopher <unk>, President and Chief Executive Officer, and Sandra <unk>, Our principal financial officer.
Speaker 1: Before we begin, please note that during this conference call, the company will make some projections and forward looking statements. These statements are only predictions based on current information and expectations and involved a number of risks and uncertain
Before we begin please note that during this conference call. The company will make some projections and forward looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties.
Speaker 1: We encourage you to review the company's filings with the FCC. This includes without limitation the company's forms 10K and 10Q, which identify the specific factors that may cause actual results or even events to differ materially from those described in these forward-looking slides.
We encourage you to review the Companys filings with the SEC. This includes without limitation. The Companys forms 10-K, and 10-Q, which identify the specific factors that may cause actual results or even events to differ materially from those described in these forward looking statements.
Speaker 1: These factors may include without limitation, risks inherent in the development and or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. And with that, I would like to turn it over to Dr.
These factors may include without limitation risks inherent in the development <unk> commercialization of potential products uncertainty in the results of clinical trials or regulatory approvals need and ability to obtain future capital and maintenance and maintenance of intellectual property rights.
And with that I would like to turn the call over to Dr. <unk>.
Speaker 2: Thank you, Clint. And we appreciate everyone joining us on today's conference call to review our most recently reported financial results and to provide a business update.
Thank you Clint and we appreciate everyone joining us on today's conference call to review, our most recently reported financial results.
And to provide a business update.
Speaker 2: The first quarter marked significant progress across our portfolio, highlighted by the positive top line results of the randomized placebo controlled avatar phase three study. For the treatment of adult patients.
The first quarter marked significant progress across our portfolio.
Highlighted by the positive topline results of the randomized placebo controlled phase III study for the treatment of adult patients with Ret syndrome, and the positive topline results from the placebo controlled phase one study of <unk> <unk>, which is in development for the treatment of neuro degenerative diseases, including FTB.
Speaker 2: And the positive top line results from the placebo-controlled phase one study of Anavex 371, which is in development for the treatment of neurodegenerative diseases, including FTD frontotens Jim.
Front of the debenture and the clinical data driven evidence of efficacy and safety of our broad Sigma one plex, our portfolio, which allows us to plan and to expand further into the rare disease space, including implementing expanded access for adult patients with ret syndrome and under served.
Speaker 2: and the clinical data-driven evidence of efficacy and safety of our broad SGMA-1 platform portfolio.
Speaker 2: which allows us to plan and to expand further into the rare disease space, including implementing expanded access for dull patients with Rett syndrome, an underserved population.
Population.
Speaker 2: At the same time, we are advancing both ANOVAX273 and ANOVAX271 in the planned studies with a goal to driving meaningful growth across our broad Sigma-1 platform portfolio to deliver transformational treatments for patients with both their genitive and developmental neurological disorders around the world.
At the same time, we are advancing both on <unk> three and <unk> 371 in the planned studies with the goal to driving meaningful growth across our broad Sigma one platform portfolio to deliver transformational treatments for patients with both degenerative and developmental neurological disorders.
Around the world.
Speaker 2: Starting with our lead drug candidate, 273, we reported positive top-light results from the second randomized possible controlled avatar phase free study for the treatment of adult patients with red syndrome. The study met its primary and secondary efficacy and safety end points with consistent and clinically meaningful improvements in all efficacy measures of red syndrome symptoms.
Starting with our lead drug candidate <unk> 273, we reported positive top line results from the second randomized placebo controlled phase III study for the treatment of adult patients with vet syndrome.
The study met its primary and secondary efficacy and safety endpoints with consistent and clinically meaningful improvements in all efficacy measures of ret syndrome symptoms.
Speaker 2: Convenient one, daily oral liquid doses of up to 30 milligram of anodex-7-3 were also well tolerated with good medication compliance.
Convenient once daily oral liquid doses of up to 30 milligrams of analytics to seven three were also well tolerated with good medication compliance base.
Speaker 2: based on the results of the avatar phase 3 study and the prior successful US phase 2 study in adult patients' work for ret syndrome, ANOVAX is now planning to meet with the FDA to discuss the approval pathway.
Based on the results of the Avatar Phase III study and the prior successful U S based phase II study in adult patients with Ret syndrome under.
<unk> is now planning to meet with the FDA to discuss the approval pathway.
Speaker 2: Topline results from the placebo controlled excellence face two, third, three study for the treatment of patriotic patients with retin syndrome are expected in second half of 2022. The extension of this expected duration of enrollment is based on recent country and local government requirements for children to have full COVID-19 vaccination, prior to joining a clinical trial, which includes our patriotic excellence trial.
Topline results from the placebo controlled excellence phase II phase III study for the treatment of patriotic patients with Ret syndrome are expected in the second half of 2022.
The extension of the expected or the expected duration of enrollment is based on recent country in local government requirements for children to have full COVID-19 vaccination prior to joining our clinical trial, which includes our patriotic excellence trial.
Speaker 2: This phase two says three study in the study patients with red syndrome H5 to 17 will evaluate the safe in efficacy of 273 and approximately 84 patients over 12 week treatment period, including on a vex to 73 specific precision medicine biomark.
This phase II III study in pediatric patients with Ret syndrome age five to 17 will evaluate the safety and efficacy of 273 and approximately 84 patients over a 12 week treatment period, including <unk> 273 specific precision medicine Biomarkers.
Speaker 2: Regarding our Alzheimer's disease program, top line results from the randomized placebo controlled phase 2B slash 3 Anavex 273 AD004 study for the treatment of Alzheimer's disease are also expected in the second half 2022.
Regarding our ultimate eases program topline results from the randomized placebo controlled phase II <unk> III and <unk> 2000, 38004 study for the treatment of autoimmune disease are also expected in the second half of 2022.
Speaker 2: The 509 patients late stage phase 2b-3 study in patients with ultimately disease is taking place at 52 sites across North America, Europe and Australia, using others COG for cognition and ADCS ADL for activities of baby living and function is primary endpoint.
The 509 patients late stage phase II phase III study in patients with ultimate disease is taking place at 52 sites across North America, Europe , and Australia, using as Cogs for cognition, and Adcs ADL for activities activities of daily living and function.
Marie endpoints.
Speaker 2: This clinical trial is measuring efficacy, tolerability and safety of two different one-stabii oral 273 doses or placebo.
This clinical trial is measuring efficacy tolerability and safety of two different once daily oral <unk> 273 doses or placebo.
Speaker 2: The current data allows us to expand in parallel our ANAVX platform, pipeline using gene biomarkers of response, applying precision medicine for neurological disorders with unmet medical needs, which are expected in 2022. That includes the planned initiation of ANAVX2M3 imaging focus parking to the developmental study.
The current data allows us to expand in parallel our analytics platform pipeline using gene Biomarkers of response applying precision medicine for neurological disorders with unmet medical needs, which are expected in 2022 that includes the planned initiation of Aneth.
This was an <unk> III imaging focused parkinson disease clinical study.
Speaker 2: a plant initiation of a potential pivot closed phase two, phase three study and fragile X syndrome, the most frequent genetic cost of out there's an spectrum disorder. And a plant initiation of a phase two, phase three, little trial for the treatment of a new red disease indication.
A planned initiation of a potentially pivotal phase II phase III study in fragile X syndrome. The most frequent genetic cause of autism spectrum disorder, and a planned initiation of our phase II phase III pivotal trial for the treatment of a new rare disease indications as.
Speaker 2: As well, last month, anaesthetic reported positive top line results from the placebo control phase one, anaesthetic three-seminone clinical trial. In development for the treatment of neurodegener diseases, including FDD, fronto-teprotemensia, for which anaesthetic three-seminone has been granted orphan drug designation by the FDA.
As well last months <unk> reported positive top line results from the placebo controlled phase one and <unk> one clinical trial in development for the treatment of neuro degenerative diseases, including FTB Frontotemporal dementia, which unlike 371 has been granted orphan drug designation by the FDA.
Speaker 2: The study reached primary and secondary endpoints of safety with no serious adverse events or dose limiting toxicity observed. ANAVX371 was well tolerated in all cohorts receiving ANAVX371 in single doses ranging from 5 milligram to 200 milligram daily. With no serious adverse events and no significant lab abnormalities in any subject.
The study reached primary and secondary endpoints of safety with no serious adverse events or dose limiting toxicity observed.
<unk> hundred 71 was well tolerated in all cohorts, receiving an extra 71 and single doses ranging from five milligram 200 milligram daily with no serious adverse events and those significant lab abnormalities in any subject.
Speaker 2: Based on these results in Unvex-371 pre-clinical profile, we intend to advance 371 into a biomarker driven clinical development dementia program for the treatment of FTD, front-order blood dementia, schizophrenia, and Alzheimer's disease, evaluating longitudinal effect of treatment with Unvex-371. We believe that the results of these studies could serve as the basis for advancing into respective registration studies in the US.
Based on these results and extra 71 preclinical profile, we intend to advance 371 into a biomarker driven clinical development dementia program for the treatment of FTB Frontotemporal dementia, she saphenous and ultimate disease evaluating longitudinal effect of treatment with <unk>.
<unk> 71, we believe that the results of these studies could serve as the basis for advancing into respective registration studies in the U S.
Speaker 2: And now I would like to direct a call to Sandra Bernish, Principal Financial Officer of Anavex for Reef Financial Summary, or recently reported quarter.
I would like to direct call to Sandra Burnish principal financial officer, and Alex for a brief financial summary of the recently reported quarter.
Speaker 3: Thank you for the fun and good afternoon to everyone on the call. Our cash position at December 31, 2021 was 151.1 million, which we believe is sufficient cash runway to fund operations and clinical programs beyond 2025.
Thank you Christopher and good afternoon to everyone on the call.
Our cash position at December 31st 2021 was $151 1 million.
Which we believe is sufficient cash runway to fund operations and clinical programs beyond 2025.
Speaker 3: For the first quarter, cash utilized in operations list 3.5 minutes.
For the first quarter cash utilized in operations was three 5 million.
Speaker 3: We reported a net loss of 10.9 million for the quarter, or 14 cents per share, inclusive of non-cash compensation charges of 3.9 million.
We reported a net loss of $10 9 million for the quarter are 14 cents per share.
<unk> <unk> of noncash compensation charges of $3 9 million.
Speaker 3: compared to net loss of 7.9 million or 12 cents per share, inclusive of non-cash compensation of 0.9 million for the comparable quarter of fiscal 2021.
<unk> to net loss of $7 9 million or 12 cents per share inclusive of noncash compensation.
$9 million for the comparable quarter of fiscal 2021.
Speaker 3: A research and development expenses for the first quarter of fiscal 2022 were 8.7 million as compared to 7.9 million for the first quarter of fiscal 2021.
Our research and development expenses for the first quarter of fiscal 2022, or $8 7 million as compared to $7 9 million for the first quarter of fiscal 2021.
Speaker 3: General and administrative expenses worth 3.1 million compared to 1.5 million but a comparable quarter of fiscal 2021.
General and administrative expenses were $3 1 million compared to $1 5 million and a comparable quarter of fiscal 2021.
Speaker 3: In both cases, the increases are primarily related to an increase in non-cash conversation charges of 1.7 million for research and development and 1.3 million for general and administrative expenses, period over period.
In both cases, the increases are primarily related to an increase in noncash compensation charges of $1 7 million for research and development and $1 3 million for general and administrative expenses period over period.
Speaker 3: These increases in non-cash compensation are mainly driven by the addition of staff to manage and support our clinical studies and development.
These increases in non cash compensation are mainly driven by the addition of staff to manage and support our clinical studies and development.
Thank you and now I'll turn it back over to Christopher.
Speaker 2: Thank you Sandra. Again, we look forward in 2022. We are very excited about the company's potential.
Thank you Sandra again, we look forward into 2022, we are very excited about the company's potential as we build on the successful completion of two important studies that allow us to confidently expand further into the rare disease space and planned extended access for adult patients with Ret syndrome.
Speaker 2: as we build on the successful completion of two important studies that allow us to confidently expand further into the red disease space and plan expanded access for patients with red syndrome. While we're looking forward to further pivotal clinical trial results in patriotic red syndrome and our
While we are looking forward to further pivotal clinical trial readouts in patriotic Ret syndrome, and ultimate disease and pipeline updates. This year I would like now to turn the call back to Glenn for Q&A.
Speaker 2: and pipeline updates this year. I would like now to turn the call back to Clinton for Q&A.
Okay.
Okay.
Yeah.
Speaker 1: Thank you. We will now begin the question and answer session. If you have a question, please raise your hand or enter it into the Q&A.
Thank you we will now begin the question and answer session. If you have a question. Please raise your hand or enter into the Q&A box.
Speaker 1: Our first question is coming from Charles Duncan with Canterbury Shield.
Our first question is coming from Charles Duncan.
And if it's true.
Please go ahead Charles.
Speaker 4: Let's see, can you hear me? We can hear you. Okay, super. Thanks Clint. Hi Christopher and team. Thanks for hosting the call. I had a couple of questions on the recent Avatar readout.
Let's see can you hear me, we can hear you okay Super Thanks Clint.
Christopher and team thanks for hosting the call had a couple of questions on the recent avatar readout.
Speaker 4: one on excellence, and then I wanted to ask Sandra a little bit about the cache runway. So regarding avatar, we were quite intrigued with recent results, but I guess I'm wondering if you had assessed just simply the change from baseline and RSVQ and CGI, and if you intend to present that data here in the near term.
One on extra lines, and then I wanted to ask Sandra a little bit about the cash runway.
So regarding effort Avatar, we were quite intrigued with recent results, but I guess I'm wondering if you had a SaaS just simply the change from baseline in RSP, Q and CGI and if you intend to present that data here in the near term and then regarding the.
Speaker 4: And then regarding the expanded access, is that part of an open label extension study or open label extension to the avatar study?
<unk> access is that part of an open label extension study or open label extension to the Avatar study.
Speaker 2: Right, so get me first on the last question. The idea is, expanded access is to provide not only for those patients which have been participating in the trials to give the drug for free, so they can continue to enjoy the treatment effect, but also those patients which are not part of the trial. So that is the definition of the expanded access. So that's why we're considering and working towards.
Right. So give me first on the last question.
Idea is expanded access is to provide not only for those patients which have been participating in the trials to give the drug for free so they can put you to.
Enjoy the treatment effect, but also those patients which are not part of the trial. So that is the definition of the expanded access so thats why we are considering and working towards.
Speaker 2: Regarding the first question, let me explain again the background of the WISP on analysis. And the results of the animal studies for red syndrome and other neurogenic diseases indicated that Anavex o7-3 has both symptomatic and disease-multifying effects on neurodevelopmental and neurodegenerative diseases.
Regarding the first question.
Let me explain again the background of the responder analysis.
And the result of the animal studies for Ret syndrome, and other neurogenic diseases indicated that <unk> III is both symptomatic and disease modifying effects on neurodevelopmental and Neurodegenerative diseases and.
Speaker 2: And for that reason, the ANAVX 2.3 analysis of the data
And for that reason.
<unk> two <unk> analysis of the data should capture this kind of effect and this is done in the form of the way we have presented it as a responder analysis with the <unk> AUC.
Speaker 2: should capture this kind of effect. And this is done in the form of the way we have presented it as a response analysis with the R-Speed QAUC.
Speaker 2: Now on top of that, we have seen and learned and the paper which was quoted and explicitly mentioned also in the presentation of February 1st pointed out that the R-SpecQ alone, the standalone has just so many flaws that has, for example, 200% upswing and downswing and is not calibrated in a baseline. So it's just not fair to use it.
On top of that we have seen and we've learned in the paper, which was quoted and explicitly mentioned also in the presentation of February 1st pointed out that our Q alone as a stand alone has just so many flaws that has for example, 200 percentage.
Upswing and downswing and is not calibrated in the baseline. So it's just not fair to use it.
Speaker 2: However, what we can say is that we make a reference of what is the CGI requirement of a one-point scale improvement, which is a three or less, which is a minimum improvement of clinical significance. And that represents a RSPQ change.
However, what we can say is that we.
Make.
A reference of what is the CGI requirement of a one point scale improvement, which is a three or four or less which is a minimum improvement of clinical significance and that represents a <unk> <unk> change of average of 12, 5% reduction of the <unk>.
Speaker 2: of average of 12.5 reduction of the RSPQ scale. So knowing now that you have a above average over 72% spawned a which are better than three score of three, so they can have also a score of two, which we have seen in this study. So you can see that the RSPQ average needs to be at least more than 12.5 delta. So I hope that helps to explain this a little bit better. Preferably the simulation is looking at what is on the screen.
Gail.
Knowing now that you have.
Above average over 72% sponge, which are better than three score three so they can have also a score of two which we have seen in this study you can see that we are <unk> average needs to be at least more than $12 five delta. So I hope that helps to explain this a little bit better.
It does Christopher I appreciate that.
Speaker 4: Definitely appreciate linking RSVQ to clinical benefit. Let me turn to excellence. I guess I'm wondering if you'll use the same.
Definitely appreciate linking.
RSP Q2 clinical benefit let me turn to excellence I guess I'm wondering if you'll use the same.
Speaker 4: evaluation as was used in Avatar because I think Clean Trouse has it a little bit different and you might correct that. And then the second thing about excellence is that in terms of the patient enrollment to date, can you give us some color on the number of patients enrolled and when during the second half of 22, you would anticipate that study to read out?
Evaluation as was used in avatar, because I think Clint trials has hit a little bit different and you might correct that and then the second thing about actual launch is that.
In terms of the patient enrollment to date can you give us some color on the number of patients enrolled and when during the second half of 2022 you would anticipate that study to read out.
Speaker 2: So the enrollment is going well. It's just that we learned that because of the COVID vaccines have been approved around the world and also have been approved for children, which are younger than 18. And the requirement is now that all these participants want to need to, and it's a fair proposition to get vaccinated.
Yep. So the enrollment is going well. It is just that we learn that because of the COVID-19 vaccines have been approved around the world and also have been approved for.
Children, which are.
Younger than 2018, and the requirement is now that oldest participants want to or need to and it's a fair.
Proposition to get vaccinated.
Speaker 2: A protocol, however, requires that you have to be on a constant medication several weeks, I think it's six or eight weeks in advance for joining a trial. So that means that you have to get the first dose of the vaccine, but also the second one. In time, and then you have to allow for some time to pass by to be on a stable, you know, new indication on a new medication. And vaccine is a new medication.
E Protocol. However requires that you have to be on a constant medication.
Oral weeks I think it's six or eight weeks in advance before joining a trial. So that means that you have to get the first dose of the vaccine, but also the second one and in time.
And then you have to allow for some time to pass by to be on a stable.
New indications new medications and the vaccine is a new indication so what it does it shifts a little bit out.
Speaker 2: So what it does, it shifts a little bit out the timeline to finish the last patient in this trial.
Timeline to finish the last patients in this trial.
Speaker 4: The, what was the other question, Charles? I just, yeah, I just, I was just wondering if you can provide some color on the number of patients enrolled thus far.
The.
What was the other question Trust.
Yes, I just I was just wondering if you can provide some color on the number of patients enrolled thus far.
Speaker 2: We are doing well from our perspective. I'd like to just mention that right now we are very comfortable with the timeframe to being second half 22. That gives us ample time to complete the study with this additional required vaccination regimen. So this is sufficient for us at this point to share. Okay. And you may narrow the timeframe from, say, second half to a little bit more.
We.
We are doing well from our perspective I'd like to just mention that right now we're very comfortable with the timeframe to being second half 'twenty two that gives us ample.
Ample time to complete the study.
With this additional required vaccination regimen so business.
Sufficient for us at this point to share.
Okay, and you maybe narrow the timeframe from say second half two this was a bit more.
Speaker 2: granularity in the future as you as your enrollment progresses. Exactly right. Yes. Okay. And in regards to the clinical trial of golf, I would like to make again a statement here that the clinical trial of golf is not
Granularity in the future and see us here in Rotterdam.
Right.
<unk>, yes, Okay, and then in regards to the clinical trials Gov.
I would like to make a dentist statements here.
We tend to run with golf is not.
Speaker 2: What we want to refer is to company communication. It will be updated eventually. So I like to, you to be aware of that. So the company communication is, has priority over clinical trial.gov. But it will be updated when we have finalized the study outcome. And then we will also update the clinical trial.gov. So right now it's, it's.
What we are.
Two refers to company communication it will be updated eventually so I'd like to you to be aware of that so the the company communication is has priority over clinical protocol, but it will be updated.
We have <unk>.
Analyzed the study outcome.
We will also update the clinical tradeoff right now.
Speaker 2: might not be completely up to date. So I want to make sure people understand that.
Might not be completely up to date, so I want to make sure people understand that.
Speaker 4: Okay, last quick question for Sandra. I think you mentioned 151 million good through 25. I assume that considering only the current spend and that you are not contemplating an NDA filing or commercial prep for that runway and that the runway may change. If you end up being involved in an NDA filing or commercialization.
Okay last quick question for Sandra Thank.
Thank you mentioned $151 million good through 25, I assume that's a.
Considering only the current spend.
And that you are not contemplating an NDA filing our commercial crap for that runway in that.
The run rate may change.
You end up.
Being involved in an NDA filing and commercialization.
Speaker 2: Yeah, if I can maybe start first. So we have over 150 million in cash, which is a really incredibly large sum of money. And when you calculate our average cash utilization in the past was very consistent in the range of two million per month to 2.5. And now in the last quarter we average 2.3 I think.
Yes, if I can maybe start first so we have over $150 million in cash which is a really.
Incredibly large sum of money.
When you calculate our average cash utilization in the past was very consistent in the range of $2 million per month to two five.
And now in the last quarter, we averaged $2 three I think.
Speaker 2: So this exactly right will be the causalization. If we do have additional plans which are marketing the drug, of course there will be a difference in that regard. So in this regard, the causalization is right now continuing what we have planned, which is the several studies which we mentioned, we are planning to do and executing the studies which aren't going. So that is the assumption of the calculation. So in this regard, we are planning to do and executing the results which are not going to be the same.
So this exactly right will be the capitalization if we do have.
Additional plans, which are marketing the drug of course, there will be a difference in that regard so.
In this regard to the capitalization right now continuing what we have planned which is the several studies, which we mentioned we are planning to do and executing the studies which are ongoing.
<unk>.
The assumption of the calculation.
Okay. Thank you.
Thank you.
Speaker 1: The next question is coming from Sumit Roy at Jones Research.
The next question is coming from Sumit Roy Jones Research.
Speaker 5: Hi everyone, thank you for taking the question and congrats on this quarter and the data. One question Chris, I wanted to understand or get you tape on how do you think FDA would look at two drugs being filed so close to each other with two different endpoints curious if you would make any comment if you think they would ask Kedia to file it with RSBQAUC.
Hi, everyone. Thank you for taking my question and congrats on the on this quarter and in the data.
One question.
Chris I wanted to understand or get your take on how do you think.
FDA would look at too.
Drugs being filed so close to each other with two different endpoints curious if you would make any comment if you think they would ask Katy to file it with RSP QA QC.
Speaker 2: It's an excellent question. I would like to speak just on our data. We really think that, as I mentioned before, every drug needs to be looked at.
Its excellent question.
I would like to speak just.
Our data, we really think that as I mentioned before every drug needs to be looked at in its uniqueness and you want to analyze it with the drug effect in mind and that's what we've done and on top of it came the paper on the <unk>, which really.
Speaker 2: in its unique nest and you want to analyze it with the drug effect in mind. And that's what we've done. And on top of it came the paper on the R-SPEC-2, which really has...
Speaker 2: extremely high variability and baseline inconsistency. And again, I'm not speaking here out of line. It's really in this paper from 2020, which came around the time when we finished.
Has extremely hyper ability and baseline and consistency and again I'm not speaking out of line. Its really in this paper from 2020, which came around the time when we finished.
Speaker 2: our first study in the US study. And that's what we learned from that to adjust to this.
Our first study in.
The U S study and Thats, what we learned from that.
To adjust to this and then the guidelines of the FDA guidance are really specific what to do in those cases, if you have a baseline if you ever endpoint, which has.
Speaker 2: And then the guidelines of the FDA guidance are really specific what to do in those cases. If you have a baseline, if you have a endpoint, which has
Speaker 2: not a totally reliability feature, then you can use what is called the anchoring and use the respond analysis. But that requires, it's very important, and we probably should highlight that stronger, that this endpoint in question need to be correlating with this CGI. And if it doesn't correlate, you cannot use this anchoring method. So, and you are then left with a poise.
Not a.
Totally.
<unk> ability a feature than you can.
Use what is called the anchoring and use a responder analysis, but that requires very important and we probably should highlight the stronger that this endpoint in question need to be.
Correlating with the CGI and if it doesn't correlate you cannot use this enquiry method.
And you're then left with a poor outcome, but since we tested the correlation.
Speaker 2: But since we tested the correlation, and it should correlate because the difference between the two measures are the risk of QS assessed by the parents and the CGII are assessed by the physician.
Correlate because the difference between the two measures are we are secure as assessed by the parents and the CGI as assessed by the physician.
Speaker 2: And while they're measuring different things, they both should basically see a positive change independently of each other. And so that means they should eventually correlate to a certain extent.
And while that measure different things they both should basically see a positive change independently of each other and so that means they should eventually correlate to certain extent and they do in our studies and they did and those studies, which is a good thing and so once you have established correlation.
Speaker 2: and they do in our studies and they did in those studies, which is a good thing. And so once you have established the correlation, then the FDA guidance really explicitly say, please use this anchor based method because you just also take care of what the FDA is always concerned about.
Then if the guidance really explicitly say please use this anchor based method because you just also take care of what the FDA is always concerned about not clinically not statistically significant but clinically meaningfulness and by doing this we basically.
Speaker 2: not clinically not statistical significance but clinically meaningfulness. And by doing this we basically raised the bar.
Raised the bar for us for the drug, but we also did it to make it easier to appreciate the drug effect. Because now you can be assured that everybody who is fonda is a responder also has to have an improvement which is clinically meaningful. So we've made it for the FDA easier that's why I think the guidance.
Speaker 2: for us for the drug, but we also did it to make it easier to appreciate the drug effect. Now you can be assured that everybody who is ponder.
Speaker 2: is a responder also has to have an improvement which is cleanly meaningful.
Speaker 2: So we made it for this FDA EVIA. That's why I think the guidance is very clear because they want to make sure that you just don't have an average statistical improvement of a certain percentage.
<unk> is a very clear because they want to make sure that you just don't have an average.
Statistical improvement of a certain percentage and or score, but it doesn't mean anything to anybody nobody can confirm that it is beneficial the physician, Canada asset or confirm it the patient cannot confirm it and the parents can confirm it.
Speaker 2: and or score, but it doesn't mean anything to anybody. Nobody can confirm that it is beneficial. The physician cannot assess it or confirm it. The patient cannot confirm it. And the parents cannot confirm it.
Speaker 2: but it's statistically significant. So it doesn't help anybody. So by using this approach of the CGI anchored R-SpecQ,
Statistically significant so it doesn't help anybody.
So by using this approach of the CGI anchored urban ARPA Q Youll see we were able to raise the bar make it easy to interpret so it's the analogy, but we also mentioned on the first of February of the story of the way.
Speaker 2: And you see we were able to raise the power, make it easy to interpret. So it's the analogy of what we just also mentioned on the first of February of the story of the way a skin diseases are assessed.
Skin diseases RFS.
Speaker 2: you know, in rash or other features that you have to raise, reduce a certain amount of minimum amount.
In rash or.
Sure.
Other features that you have.
To raise reduce a certain amount of minimum amount for example in the in rashes that 75% or 90% before being considered even that the drug works in this bar with what we have here included and that allows for just more fair and proper assessed.
Speaker 2: for example, in RASH is a 25% or is 90% before being considered even that the drug works. In this,
Speaker 2: with what we have here included. And that allows for just more fair and proper assessment of the drug of the effect of the drug.
Some of the truck of the effect of the drug.
Speaker 5: Thank you, because that really helps. Thank you for getting into the details. One last question is, if there is any formulation difference between the adult or retrial and the pediatric one, and if you could talk about if the liquid versus pills, you see an advantage you have versus your peers.
Thank you Chris that's really helpful. Thank you for getting into the details well last question is if you can remind us.
If there is any formulation difference between the adult alright.
Trial in the pediatric one and.
If you could talk about it.
The liquid versus peers, you see an advantage you have versus your peers.
Speaker 2: So we have for the red trials consistent a liquid formulation across all trials, all three trials in the program. So the US study, the avatar and the accents have exactly the same formulation, a liquid formulation, once daily, early, they take the amount of drug days they need. And so there's no difference.
So we have for the ret trials consistent a liquid formulation across all trials all three trials in the program. So the US study the avatar and the excellence have exactly the same formulation as the liquid formulation once daily orally to take the amount of drug they need.
And.
So there is no difference.
Speaker 2: What maybe you're referring to is the formulation for the Parkinson's disease, dementia study, and the Alzheimer's disease study. So in those cases, we have a one-stayly solid capsule formulation or pill, and that is also different doses, and that is taken in a solid form. In the reason why we formulated or developed a formulation of liquid formulation of the drug was,
You're referring to is the formulation for the Parkinson disease dementia study and the ultimate disease study. So in those cases, we have a once daily.
Solid capsule formulation or pill and that is of the also different doses and that has taken.
In a solid form in the reason why we formulated.
To develop a formulation of liquid formulation of the drug was learned very early on that patients with ret syndrome, sometimes either cannot swallow.
Speaker 2: We learned very early on that patients with red syndrome sometimes either can or swallow.
Speaker 2: you know, capsules or pills or they have not even the ability to take food or liquid normal way. They have a pouch where they have a basically getting that injected. So you need to have a formulation which allows to do that and a liquid formulation is exactly suitable for that.
Capsules or pills or they have not even the ability to take.
The food or illiquid, the normal way they have a pouch with a basically getting debt in injected so unique.
<unk> formulation, which allow us to do that in a liquid formulation is exactly suitable for that.
Speaker 5: Thank you Chris, really appreciate it and congratulations again on the robust data.
Thank you Chris really appreciate it and congratulations again on the robust data.
Thank you.
Speaker 1: The next call is coming from Tom Bishop at BI Research. Go ahead Tom.
Next call is coming from Tom Bishop with Bi research.
Go ahead Tom.
Yeah, Hi can you hear me.
Speaker 6: Okay, if Mary serves me on another call, you estimated that I think that the total rent market could be as much as two to five billion. And I was just wondering if my recollections correct and was that for the US or the world? And maybe even if you can tell us something about the adult rent population versus. All this is incredibly close.
Yes.
Okay.
<unk> serves me.
On another call you estimated that.
I think that the total rent market could be as much as $2 billion to $5 billion and I was just wondering if my recollection is correct.
Was that for the U S for the world.
Maybe even if you can tell us something about the adult.
Population versus.
The pediatrics.
Speaker 2: So the number, I don't think we mentioned it, but it's a back on the angle of calculation, which I just want to point out here.
Net.
So the number I don't think we mentioned it but.
Back on the envelope calculation, which I just want to point out here. So on average there are 11000 patients assumed in the U S. So, let's simplify 10000 and a rare disease pricing per year could be easily in the range and I'm not saying that this is the price of the drug for four red patient, but it could be in the range of 200.
Speaker 2: So, on average, there are 11,000 patients who are seeing in the US.
Speaker 2: So let's simplify 10,000. And already these pricing per year could be easily in the range. And I'm not saying that this is the price of the drug for red patient, but it could be in the range of 200,000 to 5000 per year per patient.
Two 500, thousands per year per patient and if you calculate multiply this by 10000, so the total revs.
Speaker 2: And if you calculate and multiply this by 10,000, so the total revenue per year could be sales in the range of $2 billion to $5 billion. I think that's probably the calculation was made. So that would be addressing only US market at this point.
Revenue per year could be.
<unk> in the range of $2 billion to $5 billion I think thats, probably the calculation was made so that would be.
Addressing only U S market at this point.
Speaker 2: If you also now look at specific to adult patients with red, we estimate this is run about 50% of the patients in the adult patients compared to the total patient amount. So you basically would assume that the adult red population would be run about half of.
You also know look at specific to adult patients with ret.
Estimated run about 50% of the patients in the adult patients compared to the total.
Amount. So you basically would assume that the adult ret population will be run about half of that.
Speaker 2: Okay, and then there's the whole world to boot, huh? And then this whole world, we have, obviously, you know, the markets are large. Some of what we have seen is that the market sizes are in the range of, I would say, but then we've seen and...
Okay, and then there is the whole world to boot Huh and then there's a whole world we have off lease.
The markets are large sample of what we have seen is that.
The market sizes are in the range of let's say.
But then we've seen them.
Speaker 2: and estimates pressure lags. Europe is 13,000. Asia is 37,000 and global 350,000.
And estimates fragile X Europe is 13000.
Asia is 37000 and global <unk> hundred 50000.
Speaker 2: with a fragile X or a red? Yeah, this is, sorry, this is red syndrome only. So red syndrome, Europe is 11,000, 13,000. These are all quoted references from the, from the natural foundations or respective information. And I used the 7,000 red syndrome patient and global 350,000 patients with red syndrome.
Was that fragile X or Rick Yes. This is sorry, those ret syndrome, only so ret syndrome.
Europe is 11 30 thousands.
These are all quoted.
References from from.
Natural.
Asians or respective inflammation and Asia. So the 7000, that's innovation and global 250000 patients with Ret syndrome.
Speaker 2: The fragile axis larger, it's around about six to seven times the size of retinum.
For fragile X is larger it's run about six to seven times the size of Ret syndrome.
Okay.
Speaker 6: I know that the quarterly commentary again skips over Parkinson's disease dementia and physical aspect as well, and I'm wondering why that is.
I note that the quarterly commentary again skips over parkinsons disease, dementia, and the physical aspect as well and I'm wondering why that is and what's.
Speaker 2: No, we want to update when we have updates. So the PDD study was really successful and allowed us to move into now two directions. And we stated that one is to plan a potentially people study in Parkinson's alone, given the strong UPD signal.
What's the status.
We want to update when we have updates so the PDD study was really successful and it allowed us to move into now two directions, and we stated that one is to plan a potentially pivotal study in parkinson alone given the strong <unk> signal.
Speaker 2: as well as in UP2Rs in the Parkinson's dementia, given that the strong cognitive improvement.
As well as in you Peter S. In Parkinson's dementia, given the strong covenant of improvement.
Speaker 2: We are now discussing with the respective experts, designs of such studies, and then we go with these designs to the FDA, to the agency, and ask for their input on how to execute those studies. And that's right now taking place. So once we have feedback, we will come back and revert back on this.
We are now discussing with the respective experts designs of such studies and then we go with this design to the FDA to the agency and ask for their input on how to execute those studies and Thats right now taking place. So once we have feedback we will come back and revert back on.
Speaker 6: Okay, and when do you estimate the last patient will complete the last observation in the 48 week?
Yes.
Oh, good okay, and when do you estimate the last patient will complete the last observation in the 48 week.
Speaker 2: Also I'm just trying to understand that's fully enrolled at 509,509 patients already, right? Yeah, 509 patients. So we expect the last patient to be finished around probably summertime. And so I think then we have to clean data, empty data lock, and then we have the data. So that's right now the expected time line.
Alzheimers trial I understand that's fully enrolled at 509509 patients already right.
<unk> thousand nine patients so we expect to.
The last patient to be finished.
Around about probably a summertime.
So I think then we have to.
In data into data loss and then we have the data. So that's right now the expected timelines.
Speaker 6: Could you just describe the various steps of activities after the trial is over and the cleaning and not, and
Could you just describe the various steps of activities. After the trial is over.
The cleaning and <unk> and.
Speaker 6: and eventually I'm blinding the trial and crunching the numbers. I mean, how low into these various steps? I think it would help everybody a little bit and how long roughly a time range, roughly, do they take?
And the inventory than blinding the trial and the crunching the numbers I mean, how long do these very what are these various steps. So I think it would help everybody a little bit.
And how long roughly a time range roughly do they take alright, so the key bulk.
Speaker 2: Right. So the key, uh, uh, bulk of the time is the data T now.
Bulk of the time is the data cleanup.
Speaker 2: So you have to be aware that a clinical trial wants its data locked, what it's called. You cannot go in there anymore. You cannot even change a comma. It's like locked as the word says.
So you have to be aware that our clinical trial once its data locked once called you cannot go into any more you cannot.
Even a change a comma it's like locked as the words. So what do you want to do you want to double triple quadruple check every entry that's that all the <unk> that all items I explained that there is no missing.
Speaker 2: So what you want to do, you want to double triple, quadruple check every entry. That's all the entries are in there. That all items are explained that there's no missing, you know, cell or whatever, like an excel spreadsheet. So this, everything needs to be checked, double check and triple check. And that often takes several months.
Sure.
<unk> sell or whatever like an extra spectrum, so everything needs to be checked and double check and triple check and that often takes several months.
Speaker 2: And once it's done, it's locked. When the time from lock to the, to the unblind data is very short. It's two weeks because then the zero does that. And they do quality control themselves. The double check that the outcome is truly what they have, the, you know, the calculation is provided. And they double check it with their own internal control. And then they give it to us. So this is run about.
And once it's done it's locked and then the time from lock to the to the Unblinded data is very short it's two weeks because then the zero does that and quality.
Quality control themselves to double check that the Optum is truly what they have.
The calculation is provided and they double check with their own internal control and then they give it to us So we'll business run about how it works.
Speaker 6: Okay, that's helpful. And it had been a lot of estimates of how big the revenue opportunity would be for a drug that works on Alzheimer's disease, and I must confess to have made some myself in the US and in the world. And for the US, I think it sounds to me like many think that could be around a $10 billion annually.
Okay. That's that's helpful and there have been a lot of estimates of how big the revenue opportunity would be for a drug that works on Alzheimers disease, and I must confess to have made some myself.
In the U S and in the World.
For the U S. I think it sounds to me like many things that could be.
Round that $10 billion annually.
Speaker 6: But I'm more interested in what your thoughts are on that market potential size.
But I'm more interested in what your thoughts are on that market potential.
Speaker 2: I mean, it's certainly not a reach or unreasonable. I remember when the
I mean, it's certainly not a reach of unreasonable I remember when the.
Speaker 2: Data or the approval from Biden will announce the stock.
Data or the approval from.
Biogen was announced that the stock jumped to $30 billion. So if that is the expectation of a net present value of a successful trial was successful potential successful drug.
Speaker 2: jump to 30 billion. So if that is the expectation of a net present value of a successful trial with successful potential successful drug, let's go to market then so it be it. And I think it's really like a measure of price, time-patient, which are eligible, and then you have to make the math from that point of view. But certainly it's the largest Absolutely.
Go to market than soybeans and.
I think that's really like.
A measure of price transportation, which are eligible and and then you have to make the math from that.
And from that point of view, but certainly its a largest unmet need there's no doubt about that.
Speaker 6: I noticed that same $20 billion increase in the market cap of biogen, temporarily anyway.
I noticed that same $20 billion increase in the market cap of Biogen temporarily anyway.
Speaker 6: Okay, that's it for me. Thank you. Thanks for
Okay. That's it for me thank you.
Thanks for.
Thank you.
Speaker 2: We have here another question. If there is no other question, I will only if you could comment on a couple of items, will you consider initiating further trials in Angelman Syndrome? Perhaps the basketball trial with multiple gene related disorders?
We have another question if there is no other question.
I was wondering if you could comment on a couple of items would you consider initiating further trials and implement syndrome.
The basket trial with multiple gene related disorders.
Speaker 2: And so the answer to that question is, we are planning already, so let me ask you this, I'll say this. So we want to basically bring home the RECFRIEN trust first, and we're very close to that. And after that, we want to tackle these other indications which we have clinical data, information which is a conductive of moving into a clinical trial, which includes entrance syndrome.
And so the answer to that question is we are planning already.
So let me ask you. This answer this so we want to basically bring home the red franchise for us and we're very close to that and after that we want to tackle. These other indications, which we have preclinical data information, which is a conductive of moving into a clinical trial, which includes insurance syndrome, but also as I mentioned.
Speaker 2: But also, as I mentioned, I think on the last call that we're planning to use a quasi basket file for fragile X because fragile X is a very heterogeneous population.
On the last call that we are planning to use a quasi.
Basket trial for fragile X <unk> is a very heterogeneous population and we just wanted to make sure we are addressing the right population.
Speaker 2: And we just wanted to make sure we are addressing the right population for the cohort. And what we do, we do a quasi, planning a quasi basket trial in fragile X. So each cohort will be addressed and we will homogeneous, we are assessed by how well they do with our drug. So we already basically utilizing this.
For the for the court and what we do we do a quasi <unk>.
Learning a quasi.
Basket trial in fragile X. So each cohort will be addressed and will be homogeneous lia assessed by how well they do with our drug so we already basically utilizing this.
Speaker 2: basket like trial designs to feature in our next trial. Clean down any more questions we see.
Basket like Ah trial designs to feature in our next trial.
And are there any more questions Uzi.
Okay.
Okay.
Okay can you hear me.
Speaker 7: Oh, okay. Thank you for taking our question. I'm with you. She, I'm for you. Congratulations on the quarters progress.
Please go ahead, yes.
Thank you.
Taking our question, obviously sure ample arena.
Congratulations on the quarter as progress and I just want to take it one step back about the rep in jumps on that clinical manifestation.
Speaker 7: And I just want to take a one step back about the rest in terms of my clinical manifestation. Because on the rest in terms of the presence, pretty boring, your clinical manifestation.
I think on the pre that's pretty broad range of clinical manifestation. So do you consider our resident primarily central nervous related or both central and peripheral casino novel clinical multiple clinical trials have been using <unk> primary endpoints, but some experts.
Speaker 7: So do you consider a resident primary residential nourished related or both residential and peripheral? Because you know, a lot of multiple clinical trials have been using RCKU as digested primary and points. But some experts will, this is the lab, IRFQ focused too much on behavior changes, but motor functions for red-sensual patient are also important.
Or do you believe I RSV acute focus too much on behavior changes by motor functions or ret syndrome patients are awesome products. So are you married any multi accumulated endpoints in the trial.
Speaker 7: So are you marrying any like motor action related points? In the trial.
Speaker 2: accent question and that's another reason why the R-SPEQ is basically not a bad endpoint, but just needs to be put in the right perspective and as a standalone, not sufficiently validated to represent the full features of R-SPECT.
Excellent question and that's another reason why the Q is basically <unk>.
Not a bad endpoint, but just needs to be put in the rights perspective, and as I stand alone not sufficiently.
<unk> to represent the full features of rats pathology. So the other that is twofold, yes, we did see a signal also in movement.
Speaker 2: So the other that is twofold, yes, we did see a signal also in movement features. We noticed that in the quality of life assessment that there were some activities noticed in that regard.
We noticed that in the quantity of quality of life assessment that there were some activities noticed in that regard and a quality of life also captures movement and <unk>.
Speaker 2: In the quality of life also captures movement and also for example, family cohesion. So is the family in more happy to deal with things and can the patient do things by themselves? And we also had some investigators that there were the ability of moving the legs again, which we're not able to move before.
Also for example.
Family.
Cohesion, so as a family.
We're happy to deal with things and can the patients do things by itself and will also add from some investigators that they were at the ability of moving the next again, which we're not able to before.
Speaker 2: And to come to the question about the periphery versus central nervous system, I think this is a complex disease. And as we learn more about it, we also have to appreciate that the fact that we have seen in the periphery by measuring blood.
And to come to the question about the periphery versus central nervous system. I think this is a complex disease and as we learn more about it but we also have to appreciate that the fact that we have seen in the periphery by measuring blood biomarkers of pathology like the Gaba and the ELD Triple a.
Speaker 2: biomarkers of pathology like the GABA in the L-3-2-A.
Speaker 2: which responded nicely and we're in line with the outcome of the patient benefit.
Which responded nicely and were in line with the outcome of the patient benefit. So these are also CNS related.
Speaker 2: So these are also CNS related features, but they were measured in the periphery. So we cannot exclude that the periphery effect also is involved. But ultimately, we know that the feature of the drug is really...
Features but they were measured in the periphery. So we cannot exclude that the periphery effect.
Also is involved but ultimately we know that the key feature of the drug is really on the <unk>.
Speaker 2: on the CNF and we have seen that confirmed with the target engagement study with the prior with the PET SCUM sigma one ligand. We definitely know that the key
The CNS and we have seen that confirmed with the target engagements that we did prior with a pet scan Sigma one ligand. So we definitely know that the key target point is in the brain, but again I would not exclude that there is also potential periphery effect, but to keep target is definitely the CNS.
Speaker 2: target point is in the brain, but again, I would not exclude that it is also potential periphery effect, but the key target is definitely this, you know
Speaker 7: Thank you, it's helpful. So regarding your interaction with the regulators, have you already scheduled the meeting with FDA on when should we expect the fee? Yep, so the way it works, we have to send to the FDA clinical trial reports.
Thank you it's helpful. So regarding your interaction with the regulators have you have you already scheduled a meeting with FDA on Wednesday with equity.
So the way it works, we have to send to the FDA clinical trial reports.
Speaker 2: And they require to be put together. So it's not just the slides which we have. So in that right now, it's on the high pressure is taking place. So once we have those slides, those documents done together, which are doing the study reports, the formal reports, then we will submit them and then we'll request a meeting. Okay, so do expect the CF be back to be before the readout of excellent study?
And they are required to be put together. So it's not just the slides, which we have so in that right now.
On the high pressure is taking place. So once we have those slides those documents done together with the clinical study reports.
Formal report then we will submit them and then request the meeting.
Okay.
Specter Fiat feedback to <unk> readout of <unk> study.
Speaker 7: This is very likely because we said second half of 2022, so we expect us to be the case. Okay, sure. Thank you. Also, I can follow up your, you just mentioned your best keep study for three semide1 for three different indications. Just can give us more colors.
This is very likely because we said second half 2022. So we expect this to be the case, yes. Okay. Sure. Thank you are also going to follow up your you.
You just mentioned in a basket study or <unk> can be one illustrated by indications.
Can't give us more colors on that.
Speaker 7: I'm thinking about three different indications. How do you plan to recruit patients and how to make your efficacy? The indication has very different and points or clinical duration. Like, scale of Ramiata and Schokone change very quickly. So, do you have any prioritization or just give me a call round?
I cannot say different indications.
How do you how do you plan to treat patients and how to measure efficacy indication.
And point organic growth duration like Randy Atkins show kind of change pretty quickly. So do you have any prioritization or just any color on that.
Speaker 2: Right. So I think it was a bit of like I may be explained too quickly. We refer to fragile X where we will include fragile X patients. This is all fragile X patients, but they have different features which are not identical. So they have into homogenize them to make them more consistent in their teacher. We grew them in distinct groups of fragile X.
So I think it was a bit of like a maybe explained too quickly.
Refer to fragile X, where we will include fragile X patients is all fragile X patients, but they have different features which are not identical so there.
Into Homo giant homogenize them to make them more consistent in their feature we group them in distinct groups of fragile X.
Speaker 2: and it could be certain behavior, prevalent, certain cognitive impairment, prevalent, and so forth. So that's what I was referring to to a quasi-dastic trial. So we will measure the same endpoint, which is fragile x-related endpoint. When it comes to anode of x-371, which we are planning in Alzheimer's and in front of dementia, and also schizophrenia, this will be not part of the trial, but it will be in...
It could be.
But the behavior prevalent certain cognitive impairment.
Prevalence and so forth. So that's what I was referring to to a quasi basket trial. So we will measure to St endpoint, which is fragile X related endpoint when it comes to <unk> 71, which we are planning in.
Summer in.
And photos of dementia and also should Sapremia. This will be not part of the cost that it would be.
Speaker 2: program, we call it, there will be trials which were done separately because our schizophrenia, for example, requires a relatively short study duration, run about six weeks, for example, and the FDT and also not study require longer studies, at least six months. So you see already from that angle that they are not, that they cannot be put in one trial together.
Program, we call it there will be.
The trials, which were done separately because.
Should subpoena for example requires a relatively short study duration for about six weeks for example in the FTP and ultimately require longer studies at least six months. So you see already from that angle that they are not.
They tend to kind of be put in one trial together.
Okay. Thank you that's helpful.
Okay.
That's all the questions that can easily.
Speaker 2: So then we'd like to thank everybody. And again, as we look forward into 2022, we're very excited about the company's potential as we build on a successful completion of the two important studies that allow us to confidently expand further into the Red Disease Base and plan expanded access for the patients with fractal drops.
So then.
We'd like to thank everybody and again as we look forward into 2022, we're very excited about the company's potential as we build on the successful completion of the two important studies that allow us to confidently expand further into the rare disease space and planned expanded access for adult patients with ret syndrome.
Speaker 2: And while we're looking forward to further digital clinical trial without in patriotic red syndrome and in Alzheimer's disease and as well as pipeline updates to see out. So with that, thank you very much.
And while we are looking forward to further pivotal clinical trial, Readouts and patriotic reps syndrome, and an ulcer disease as well as pipeline updates this year, so with that thank you very much.
Speaker 1: Thank you ladies and gentlemen, that concludes today's conference call. We appreciate you participating. You can now disconnect.
Thank you ladies and gentlemen that concludes today's conference call. We appreciate you participating you can now disconnect.
Okay.
Okay.
Okay.