Q4 2021 Anavex Life Sciences Corp Earnings Call
Speaker 1: Welcome to the Anovac Life Sciences Fiscal 2021 fourth quarter conference call. My name is Adrienne and I will be your operator for today's call. At this time, all participants are in a listen-only mode. Later, we'll conduct a question and answer session. During the question and answer session, if you have a question, please press star, then one on your touchtone phone. Please note this conference call is being recorded. I'll now turn the call over to your host, Clint Tomilson. Clint, sir, you may begin.
Walk up to the end of a.
Life Science fiscal 2021 first quarter conference call My.
My name is Adrian and I'll be your operator for today's call. At this time all participants are in a listen only mode. We will conduct a question and answer session. During the question answer session. If you have a question. Please press Star then one on your Touchtone phone. Please note. This conference call is being recorded.
I'll turn the call over to your host Cliff Hamilton clinched Sir you may begin.
Thank you and good afternoon, everyone.
Speaker 2: Thank you, and good afternoon, everyone. We appreciate you joining us today for Anavex Life Sciences' fourth quarter conference call to review financial results and discuss the company's business update.
We appreciate you joining us today for <unk> Life Sciences fourth quarter Conference call to review financial results and discuss the Companys business updates.
Speaker 2: A taped replay of this call will be available after the call and the call will also be available for replay on Anavex's website at www.anavex.com.
A taped replay of this call will be available after the call and the call will also be available for replay on <unk> website at www Dot <unk> Dot com.
Speaker 2: With us today is Dr. Christopher Missling, President and Chief Executive Officer, and Sondra Bronish.
With us today is Dr. Christopher misleading, President and Chief Executive Officer, and Sandra Burnish.
Principal financial Officer.
Following management's remarks, there will be a question and answer session. 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 2: Following management's remarks, there will be a question and answer session. 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 2: We encourage you to review the company's filings with the SEC.
We encourage you to review the company's filings with the SEC.
Speaker 2: This includes, without limitation, the company's forms 10-K and 10-Q, which identify the specific factors that may cause actual results or events to differ materially from those described in these forward-looking statements.
This includes without limitation, the company's forms 10-K, and 10-Q, which identify the specific factors that may cause actual results or events to differ material materially from those described in these forward looking statements.
Speaker 2: 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'd like to turn it over to you.
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 of intellectual intellectual property rights.
And with that I'd like to turn the call over to Dr. Mozley.
Speaker 3: Thank you, Clint. We really 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 Glenn we really appreciate everyone joining us on today's conference call to review, our most recently reported financial results and to provide a business update.
Speaker 3: We concluded an exceptional fiscal year 2021 while continuing our momentum, highlighted by the efficient execution and full enrollment of three precision medicine clinical trials, including the phase 2b slash 3 ANOVAX 273 clinical trial in Alzheimer's disease, the AVATA 273 clinical trial in Rett syndrome, as well as the phase one study of ANOVAX 371.
We concluded an exceptional fiscal year 2021, while continuing our momentum highlighted by the efficient execution well enrollment of three precision medicine clinical trials, including the phase II <unk> III art of excellence.
Seven three clinical trial at somebody's needs.
Other top 273 clinical trial in <unk> syndrome, as well as good phase one study of VX 371.
Speaker 3: Starting with our lead drug candidate, 273. We expect top-line results from the second placebo-controlled avatar study for the treatment of outpatients with Brett syndrome, which are expected to be announced around calendar year end 2021.
Starting with our lead drug candidate <unk> 273, and we expect topline results from the second placebo controlled study for the treatment.
Our patients with breast syndrome, which are expected to be announced around calendar year end.
Do you want.
Speaker 3: This study took place in Australia and the United Kingdom using a higher dose than the U.S.-based Phase II study and enrolled 33 patients over a seven-week treatment period, including RNAVX273-specific precision medicine biomarker.
This study took place in Australia, and the United Kingdom, using a higher dose than the USDA phase II study enrolled 33 patients of a seven week treatment period, including analytics to seven Threep specific precision medicine biomarker.
Speaker 3: Top line results from the placebo-controlled excellence phase two slash three study for the treatment of patriotic patients with Rett syndrome are expected in the first half of 2022. This phase two slash three study in patriotic patients with Rett syndrome, age five to 18, will evaluate the safety and efficacy of 273 in approximately 84 patients over a 12-week treatment period, including 273 specific precision medicine biomarkers.
Topline results from the placebo controlled excellence phase III phase III study for the treatment of patriotic patients with Ret syndrome I expected in the first half of 2022.
This phase II phase III study in pediatric patients with Ret syndrome age five to 18.
Evaluate the safety and efficacy of $2 73, and approximately 84 patients.
12 week treatment period, including $2 73 specific precision medicine biomarker.
Speaker 3: Regarding our Alzheimer's disease program, top-line results from the placebo-controlled phase II B-slash-III RLX273AB004 study for the treatment of Alzheimer's disease are confirmed and are expected in the second half of 2022.
Regarding our somebody this program topline results from the placebo controlled phase twos.
<unk> undertakes.
300, <unk> four study for the treatment of ultimate disease.
Confirmed are expected in the second half of 2022.
Speaker 3: The double-blind, placebo-controlled, 509-patient, late-stage phase IIb slash III study.
The double blind placebo controlled 509 patients late stage phase <unk> study in.
Speaker 3: in patients with Alzheimer's disease exceeded enrollment of the targeted patient number at 52 sites across North America, Europe , and Australia, using ADAS-CoG for good medicine and ADCS-ADL for activities of daily living and function as primary and...
Patients with ultimate disease exceeded enrollment of the targeted patient number at 52 sites across North America, Europe, and Australia using at his call for cognition.
ABL to activities of daily living and function as primary endpoints.
Speaker 3: This multi-center, double-blind clinical trial is measuring efficacy, tolerability, and safety of two different once-daily oral 273 doses or placebo.
This multi center double blind clinical trial is measuring efficacy tolerability and safety of two different once daily oral 273 doses or placebo.
Speaker 3: As reported last month, the Independent Data Safety Monitoring Board for the company's Phase IIb-3 study completed its most recent pre-planned review of the preliminary Phase IIb-3 study data in asthma patients.
As reported last month.
Independent data safety monitoring board for the company as things to be strengths feed study.
But at its most recent pretend with you of the preliminary phase <unk> study data in asthma patients.
Speaker 3: As specified in the protocol, the DSMB reviewed the interim safety data for the 273 phase 2b-3 under Alzheimer's disease clinical study and its open-label extension attention AD study.
Testified in the protocol.
<unk> reviewed the interim safety data for the 273 phase <unk>.
Somebody these clinical study and its open label extension attention <unk> study.
Speaker 3: upon review of the interim safety data, the BSMB recommended to continue the study without modification.
Upon review of the interim safety data.
These can be recommended to continue the study without modification.
Speaker 3: And we're very excited also to report that the top line data from another pipeline compound, ANAVEX-371, which had received orphan drug designation by the FDA for frontal teplodementia, a placebo-controlled phase one study in ANAVEX-371, evaluating ANAVEX-371 in humans, are expected around calendar year 2021.
And we're very excited also to report that the topline data from another pipeline compounds.
371, which had received orphan drug designation by the FDA for Frontotemporal dementia, a placebo controlled phase one study <unk> hundred 71 evaluating on the next 271 units are expected around calendar year end.
2021.
Speaker 3: During 2022, we will also move in closer to further expanding the pipeline for 273 using gene biomarkers of response, applying precision medicine for another neurological disorder with unmet medical need, including a planned initiation of 273 imaging focus, Parkinson disease clinical study.
During 2022, we will also moving closer to further expanding the pipeline for <unk> using gene Biomarkers of response applying precision medicine for another neurological disorder with unmet medical need including the planned initiation of 273 imaging forecast I consider disciplined.
The study.
Speaker 3: A planned initiation also of a Phase II-3 clinical trial for the treatment of a new rare disease indication. And lastly, a planned initiation of a pivotal Phase II-3 study in Fragile X syndrome, the most frequent genetic cause of autism spectrum disorder.
A planned initiatives initiation also of our phase II clinical trials for the treatment of a new rare disease indications and lastly, our planned initiation of a pivotal phase <unk> study in fragile X syndrome. The most frequent genetic cause of autism spectrum disorder.
Speaker 3: In Fragile X, we recently announced in August of this year, strong preclinical data of 273 and Fragile X syndrome published in the peer-reviewed journal Scientific Reports.
In total ex we recently announced in August of this year strong preclinical data of.
273, and fragile X syndrome published in the peer reviewed journal scientific reports.
Speaker 3: The study evaluated 273 in FMR1 knockout mice, a validated animal model for the disease, which resulted in the reversal of hyperactivity and restoration of associative learning. Furthermore, 273 demonstrated dose-dependent sigma-1 receptor occupancy in a positron emission tomography PET study.
The study evaluated 273, and FMR, one knockout mice, a validated animal model for the disease, which resulted in the reversal of pipe activity and restoration of associative learning. Furthermore to sell III demonstrated dose dependent Sigma one receptor occupancy positive positron emission.
Mcguffey Pet study.
Speaker 3: And now, I would like to direct the call to Sandra Boenisch, Principal Financial Officer of Anavex, for a brief financial summary of the recently reported year-end.
Now I would like to direct the call to subtract the Finnish principal financial officer for analytics for a brief financial summary of the recently reported year end.
Speaker 4: Thank you, Christopher, and good afternoon to everybody.
Thank you Christopher and good afternoon to everybody.
Speaker 4: We continue to maintain a strong balance sheet and fiscal responsibility. Our cash position on September 30, 2021 was 152.1 million, which we believe is sufficient cash runway to fund operations and clinical programs beyond 2025
We continue to maintain a strong balance sheet and fiscal responsibility.
Our cash position on September 30th 2021.
It was $152 1 million, which we believe is sufficient cash runway to fund operations and clinical programs beyond 2025.
Speaker 4: During fiscal 2021, cash utilization in operations was $30.4 million.
During fiscal 2021 cash utilization utilization in operations was $30 4 million.
We reported a net loss of $37 9 million for the full fiscal year, which is 54 per share as compared to $26 3 million or <unk> 45 per share in the comparable year 2020.
Speaker 4: We reported a net loss of $37.9 million for the full fiscal year, which is $0.54 per share, as compared to $26.3 million, or $0.45 per share, in the comparable year 2020.
Research and development expenses for the year was $33 million compared to $25 2 million for the comparable fiscal year.
Speaker 4: Research and development expenses for the year were $33 million, compared to $25.2 million for the comparable fiscal year.
Speaker 4: The increase is primarily attributable to the continued advancement of our ongoing clinical trials, most notably the full enrollment of our international phase 2b slash 3 Alzheimer's disease trial and the related open label extension and the continued enrollment and advancement of the Rett syndrome studies and expansion of these trials internationally.
The increase is primarily attributable to the continued advancement of our ongoing clinical trials.
Most notably the full enrollment of our international Phase two these last three alzheimers disease trials.
And the related open label extension.
And the continued enrollment and advancement of the Ret syndrome studies and expansion of these trials internationally.
Speaker 4: General and administrative expenses were $9 million for the year, as compared to $5.9 million in the prior year.
General and administrative expenses were $9 million for the year as compared to $5 9 million in the prior year.
Speaker 4: The increase is mostly significantly associated with an increase in personnel and associated non-cash stock option compensation charges.
The increase was mostly significantly associated with an increase in personnel and associated non cash stock option compensation charges.
Thank you and now I'll return the call back over to you Christopher.
Speaker 3: Thank you, Sandra. And as we look to the remainder of 2021 and into 2022, I'm very excited about the company's potential as we continue to advance and expand our precision medicine program.
Thank you Sandra and as we look to the remainder of 2021 and into 2022 and very excited about the company's potential as we continue to advance and expand our position method two programs.
Speaker 3: As we look ahead, we will continue to focus on driving meaningful growth across our broad Sigma 1 platform.
As we look ahead, we will continue to focus on driving meaningful growth across our broad Sigma one platform.
Speaker 3: portfolio to deliver transformational treatments for patients with both degenerative and developmental neurological disorders around the world.
But for you to deliver transformational treatments for patients with both degenerative and development neurological disorders around the world.
Speaker 3: So we look forward to providing further updates as advancement continue. And at this point, I'd like to also wish everyone a happy Thanksgiving. I would like now to open the call for questions. Operator, please go ahead.
So we look forward to providing further updates and advanced and continue and at this point I'd like to also wish everyone a happy Thanksgiving.
I'd like now to open the call for questions. Operator. Please go ahead.
Speaker 1: Thank you. You will now begin the question and answer session. If you have a question, please press star, then 1 on your touchtone phone. If you wish to be removed from the queue, please press the pound sign or the hash key. If using a speakerphone, you may need to pick up the handset first before pressing the numbers. Once again, if you have a question, please press star, then 1 on your touchtone phone. And our first question comes from Charles Duncan from Cantor Fitzgerald. Your line is open.
Thank you well now begin the question and answer session.
Have a question. Please press Star then one on your Touchtone phone.
If you wish to be removed from the queue. Please press the pound sign or are they asking.
If you're using a speaker phone.
They need to pick up the handset first before pressing the numbers. Once again, if you have a question. Please press Star then one on your Touchtone phone and our first question constant Charles Duncan from Cantor Fitzgerald. Your line is open.
Speaker 5: This is Pete Stavropoulos on for Charles. Good afternoon, Christopher and team, and congratulations on the progress and appreciate all the updates.
This is Pete stavropoulos on for Charles Good afternoon, Christopher and team and congratulations on the progress and I appreciate all the updates.
Speaker 5: I have a couple of questions regarding Rett syndrome. When you think about the efficacy measures that were made and the efficacy seen in the U.S. adult study, you know, how do you feel about the sample size or the planned effect size out of Avatar and also the excellence study? And we saw that you upsized the sample size for excellence from 69 to 84 subjects. What drove that decision, if you can give us some color on that?
I have a couple of questions regarding ret syndrome when.
When you think about.
Efficacy measures.
And the efficacy seen in the U S. Adult study how do you feel about the sample size or the planned effect size out of Avatar and also the excellent study.
We saw that you upsize the sample size for excellence from 69% to 84 subjects.
What drove that is that decision if you can give us some color on that.
I appreciate it and thank you so the efficacy.
Speaker 3: I appreciate it and thank you. So, the efficacy effect size was really significant in the first US study which was using a low dose, as you remember, and the effect size was in the range 1.3 to 1.2, 1.3 quins D, which is considered very large.
Effect.
Really significant in the ER.
First U S study, which was using a low dose as you remember and the effect size was in the range of one three.
One point to 1.3.
Cohen's D, which is considered very large.
Speaker 3: And since we expect those response based on higher doses, we basically are inclined to believe that the second study, the avatar study, might show a similar, if not higher effect size given that we're using a higher dose in the avatar study compared to the U.S. study.
And since we expect those response based on the higher doses, we basically are inclined to believe that the.
Second study the Avatar study might show a similar if not higher effect size given that we are using a high dose and the other tough study compared to the U S study.
Speaker 3: The extension of patient number in the Excellence Study from originally 69 to 84 was based on a request, a regulatory request, to also have an additional sub-analysis of the number of the patient in different.
The extension of patient number in the excellence study from originally 69 to 84 was based on their request.
The regulatory request to also have an additional subs.
Analysis of the.
Number of patients of the patient.
In different age groups for example from 5% to 18 to also have additional analysis of patients from five to 12, and then from 12 or 13 to 18, we wanted to make sure we have the powerful put this additional.
Speaker 3: for example, from five to 18, to also have a additional analysis of patients from five to 12, and then from 12 or 13 to 18.
Speaker 3: In order to make sure we have enough power to put this additional calculation, we thought it is prudent to just add additional number of patients, which ended up to the total number of 84 as we calculated.
Calculation, we thought it was prudent to just add additional number of patients ended up to the total number of <unk> 84, as we co located.
Speaker 5: Thank you. Very helpful. We also saw that you made a few changes to the primary and secondary endpoints in the excellent study. You know, can you give us a, you know, help us understand on what drove those decisions? You know, was it a result of advice or interactions with the FDA or any other regulatory agency?
Thank you very helpful.
We also saw that you made a few changes to the primary and secondary endpoints and the excellent study.
You gave us.
Help us understand what drove those decisions.
A result of advice or interactions with the FDA or any other regulatory agency.
Speaker 3: Right, so we have noticed that the RSPQ is really the most rigorous, more rigorous endpoint. It is really going through 45 very dedicated questions and detailed questions which can be answered very precisely. There's also the ability which we have seen and we have demonstrated that in our presentation of doing a sub-analysis of the sub-scores of the entire score, the RSPQ score.
Right. So we have noticed that the artist acuity is really the most rigorous more rigorous endpoint. It is.
Really going through 45, very dedicated questions and detailed question, which can be answer very precisely there's also the ability, which we have seen and we've demonstrated that in our presentation of doing.
Analysis of the sub scores of the entire school at the RSP to score I wish I when we looked at the CGI I, we noticed that there was a weaker ability to make this because it's really a global assessment and it also has a very.
Speaker 3: However, when we looked at the CGI, we noticed that there was a weaker ability to make this because it's really a global assessment.
Speaker 3: And it also has a very known and it's published.
<unk> known and it's published.
Speaker 6: weak, I would say, reliability.
Uh huh.
I would say reliability.
Speaker 3: But we basically are including that still, but we didn't want to overemphasize that score. So that was the background for the focus on the RSPQ.
But we basically.
Including that still but we didn't want to over emphasize that score. So that was the background for the focus on the <unk>.
Alright, thank you.
Speaker 5: All right, thank you. And my last question, regards to the Parkinson's Disease Dementia Program. You know, can you provide any updates on that program? You know, have you met with the agency to discuss it? And, or do you plan to? Anytime in the near future? Yeah, we plan.
My last question.
Going to the Parkinson's disease dementia program.
You provide any updates on that program have you met with the agency to discuss it and do you plan to anytime.
Anytime in the near future we plan.
Speaker 3: Right, thank you very much. Yes, we plan to do that. We actually are in the process of now discussing the data with the foundations, and we are expecting to get valuable feedback for input on the design of pivotal studies for Parkinson, and also pivotal study for Parkinson dementia. And with that,
Right. Thank you.
Yes, we plan to do that we actually are in the process of now discussing the data with the.
Foundations.
And we are expecting to get valuable feedback.
For input on the design of pivotal studies for Pakistan and also pivotal study for Parkinson dementia and with that.
Speaker 6: you know, in our package, if you so like, we then feel more robustly educated and fully informed to go to do the discussion with the agency about a proper pivotal study in the respective areas.
In our package if you feel like we've been feel.
More robustly.
Educated and.
Fully informed too.
To go to.
The discussions with the agency about prop.
Pivotal study in the respective to indications.
Speaker 5: Do you think by some chance you're going to wait for the imaging study results or it'll
Do you think by some chance you're going to wait for the imaging study results or meeting.
We incurred beforehand.
This will be before the imaging study, but definitely we are still including which we have not yet reported the total gene analysis of the PDD study. So that means we not only looked at the Sigma one gene expression changes of the mrna, but also the gene expression of all genes.
Speaker 3: This will be before the imaging study, but definitely we are still including, which we have not yet reported, the total gene analysis of the PDD study. So that means we've not only looked at the sigma-1 gene expression changes of the mRNA, but also the gene expression of all genes.
Speaker 3: of all participants that is in the active arm as well as in the placebo arm and we believe that additional intelligence can be drawn out of that to make an informed decision.
All participants that is in the active arm as well as in the placebo arm.
Lead states additional intelligence can be drawn.
To make an informed the design of a study which increases the chances of a pivotal study down the road.
Speaker 3: design of a study which increases further the chances of a pivotal study down the road.
Alright, alright.
Speaker 5: All right, thank you. Thank you very much for taking my questions and congratulations again on the progress and happy thanks.
Thank you very much for taking my questions and congratulations again on the progress and a happy Thanksgiving.
Likewise I appreciate it.
Okay.
Speaker 1: And our next question comes from Raj Selaju from H.C. Wainwright. Your line is open.
And your next question comes to Us.
<unk> from H C. Wainwright your line is open.
Speaker 7: Hi Crescent team, this is Mazan Faram, congrats on the progress and thanks for taking our questions. So firstly, how early in 2022 do you envisage filing an NDA for Blanc-Comazine in Rett syndrome, providing you positive results from the three trials?
Hi, Chris and the team this is Muslim for ROM Congrats on the progress and thanks for taking our questions. So firstly how early in 2022 do you envisage filing an NDA.
Full Blanca, Compazine and ret syndrome, providing yet positive readouts from the three trials.
It's we need our collective data first come out and then we can talk about that but oddly.
Speaker 6: It's really, I would let the data first come out and then we can talk about that, but only pending data as soon as possible.
Pending data as soon as possible.
Excellent.
Speaker 7: Excellent. And then with regards to 273, what learnings are you transitioning into your Fragile X syndrome development? For example, are you planning on cross-analyzing data from the Rett syndrome and the Fragile X syndrome trials, given that these two syndromes sort of share overlapping symptoms as well as underlying cellular mechanisms?
And then with regards to $2 73.
What learnings are you transitioning into your fragile X syndrome development. For example are you planning on cross analyzing data from the rest syndrome, and the fragile X syndrome trials given that these two syndromes, so shed overlapping symptoms as well as underlying cellular mechanism.
Yes.
Yeah.
That's exactly right to search tool.
Speaker 3: pockets here to look at. One is the preclinical pocket, and we see a very strong response in the animal model of a fragile egg.
Pockets here to look at one is the preclinical and.
And we see a very strong response in the animal model of a fragile X and leading to even reversal of the pathology.
Speaker 3: leading to even reversal of the pathology. And then we look also at the clinical study of the Rett syndrome, and we see that the phenotypes are overlapping between these two indications, and there are some endpoints which are included in the Rett syndrome study. For example, ADAMS is one of them.
We look also at the clinical study of the Ret syndrome, and we see that the phenotypes are overlapping between these two indications and there are some endpoints which are included in the Ret syndrome Studies for example items is one of them.
Speaker 3: and that had been responding very well with the drug.
And that had been.
Funding very well with the drug.
Speaker 3: and we believe this could be also used as a key measure for the rectum for the Fragile X study, since that has been also even used prior in Fragile X study as a primary endpoint. The decision is not yet made exactly about how to use that endpoint, but this could be one potential venue.
We believe this could be also used as a key measure for the Rexam. The fragile X study since that has been also even used prior in fragile X study.
Our primary endpoint the decision has not yet made exactly about how to use that endpoint, but this could be one a potential venue.
Okay very helpful and we read with interest your recent published paper in expert opinion on therapeutic targets, where you described.
Speaker 7: Okay, very helpful. And we read with interest your recent published paper in Expert Opinion on Therapeutic Targets, where you described 273 and 371 as hand-in-hand targets for Alzheimer's disease. We were wondering if you've, number one, benchmarked these drugs together pre-clinically, and number two, tested a combination. If not, do you plan to do so?
Three and $3 71 as hand in hand targets for Alzheimer's disease. We were wondering if you've number one benchmark. These drugs together pre clinically and number two tests the combination.
If not do you plan to do so.
Speaker 3: So the two compounds came from different angles in different labs, but they are now moving more into what we call, we try to learn as we go. But so far, we could not find a comparable assay other than a very early preclinical assay of target engagement. And there are differences in the affinities of the Sigma-1 receptor and also difference to the muscarinic receptor, which we believe
So the two compounds came from different angles and different labs.
They are now moving more into what we call them.
Then.
As we go but so far we could not find a comparable SA other than a very early.
Preclinical assay of target engagement and the differences in the affinity to the Sigma one receptor and those are different to the muscarinic receptor, which we believe is also important and.
Speaker 3: And ultimately, we will be only able to see really the difference of the two if we run really both drugs in the same indication, in the same trial. So we think that each drug has its own merits, and it could very well be that 371 is really good at...
Ultimately, we will be only able to see really the difference of the two.
We run really both indications goldstrike and the same indication in the same trial so.
We think that each each drug has its own merits and it could very well be that 371, it's really good.
Speaker 3: focusing more on frontotemporal dementia, which we have offered a designation for, and could also be very good at Alzheimer's, but right now we have 273 more advanced, so we will eventually find out.
Focusing more in on Frontotemporal dementia, which we have orphan designation for and could also be very good at Ultima, but right now we have 273 more advanced so we will eventually find out.
Speaker 7: appreciate the color. And then just finally, you've talked about 273 using it in a prophylactic manner. We saw a recent Science Advances article which describes a unique kind of brain proteomic signature in younger cohorts mean age of 39. Are you currently working on or planning a genetic or protein test in a younger cohort to kind of help your 273 administration in the future? Yeah, we'll look into that.
I appreciate the color and then just finally, you've talked about 273 using in a prophylactic manner.
We saw a recent science advances at co which describes.
Kind of brain proteomics signature in younger cohorts mean age of 39 are you currently working on or planning genetic or protein test.
In a younger cohort to kind of.
Help your 273 administrations in the future.
Yes, we look into that and it's a very good point because.
Speaker 3: While the disease is showing up correlating with age, it's clear that a early intervention is really most likely the.
Diseases, showing up correlating with age it is clear that a early intervention is really most likely the.
Speaker 3: the most efficient way to incubate or inoculate this disease.
The most efficient way to ink.
Incubator and alkylate this disease.
Speaker 3: And since our preclinical data indicates that potential, which was done very successfully in animals that who got the drug before they got injected with a toxic A-beta,
Since our preclinical data indicates the potential which was done very successfully in animals that will go up the drug before they got injected with a toxic a better.
Speaker 3: load and they never developed the symptoms of Alzheimer's disease.
Load and data to develop the symptoms of out some of these east so it is really high.
Speaker 3: So there's really high likelihood that we should, or encouragement to also proceed. And we said, and we committed to eventually do that with the appropriate, in the appropriate time down the road. So we're committed to look into that, what you described early on.
The likelihood that we should or encouragement to also proceed when we said and committed to eventually do that with the appropriate an appropriate time down the road. So we're committed to look into that what you described.
Leon.
Okay excellent that's it from us Congrats again and happy Thanksgiving to you and everyone on the call.
Speaker 7: Okay, excellent, that's it from us. Congrats again and happy Thanksgiving to you and everyone on the call. Thanks.
Likewise.
Speaker 1: And the next question comes from Sheldon Roy from Jones Trading. Your line is open. Hello, everyone.
The next question comes from Ryan from Jones trading your line is open.
Hello, everyone and thank you for taking my question.
Could you give us a little color around the upcoming.
Adult Tourette study, so as I understand there will be different dose cohorts. So.
Is could you give us idea of the size and.
Is there going to be in truckload our dose escalation.
What should we expect.
So let me state it does not a different dose. It's just a target dose is higher than the US Study. So there's only one dose and one placebo arm and that one dose will be a target dose.
So that is just higher than the U S study. So there will be not multiple doses technically it's just one higher dose.
Okay got it.
Speaker 8: Okay, got it. And you're not disclosing if it's going to be 10, 20 or 30.
Clothing, if it's gonna be 10, 20 or 30.
Right, we will find out when we disclose the data and we will be.
Speaker 8: Right. We will find out when we disclose the data and then we will be able to learn about that. Got it. And...
To be able to learn about that.
Got it.
And.
The.
Speaker 8: Any color on the Alzheimer's trial, what are you planning as patients are coming out of these 48 weeks? Are they going to go into maintenance trial or what is the plan there?
Any color on the asthma trial, what are you planning as patients are coming out of <unk>.
They're going to go into maintenance trial or what is the plan there.
Speaker 3: Right, so we have an extension study called ATTENTION-AD, which is a two-year study of following up as an open label after the 48 weeks, which has started, you know,
Right. So we Havent extension study called attention a D, which is a two year study following up is the open label. After the 48 week, which has started.
After the first patient finished at 48 weeks and because these patients have.
Speaker 3: after the first patient finished the 48 weeks. And because these patients have actually, some of them finished this phase two open label extension, they had requested to continue to stay on the study drug. And so what we did, we...
Actually some of them finished this phase II open label extension.
They had requested two continues to stay on study drug and so what we did we.
Initiated and were successful in expanding now this attention study open label extension from two years to three years. So patients who finished the study a placebo controlled study.
Speaker 3: initiated and were successful in expanding now this attention AD study, open label extension from two years to actually three years. So patients who are finished the study, the placebo control study, entered into the extension study, finished the two years, will now continue to go into the third year. And that is because of requests by the patients, the caretakers, and the physicians.
The extension study finished two years will now continue to go into the third year.
And that is because of request by the.
Patients that can't take us in the position.
Speaker 3: I'd also like to add that the
I'd also like to add that the.
Speaker 3: I'd like to add also that the conversion from the placebo-controlled part of the study to the open-label is very high, it's above 94% currently.
I'd like to add also that the conversion from the placebo controlled.
Part of this study to the open label is.
Very high it's.
Above 94% content.
Which is a good sign.
Speaker 8: Great, it's really good to hear. And one last question, on the Back to the Red program, where are you with AFD on the conversation turning whether these kind of BD registration trial data and
Great that's really good tissue and one last question on the back to the Rec program, where are you with empty on the conversation.
Turning with at these kind of BD registration trial at the time.
Speaker 3: It's really a question now of the data and the data has to speak for itself.
It's really a question of the data and that data has to speak for itself.
Speaker 3: And we also said it's a potential pill study, so the word really weighs in. And the data really will determine this, how this will be looked at. We have to point out that for adults, there is no treatment available, and the patient population's also harder to bring into a trial because they're more advanced.
We also set it's a potential pivotal study so what really ways.
And the data we will determine this how this will be looked at we have to point out that for adults. There is no treatment available and.
The patient populations also.
Part of it too.
To be bringing into the trial because they are more advanced they also are.
Speaker 3: They also are, because of the disease early passing on, they're not that many patients available to find in a trial. So the focus is really on the...
Cause of the disease.
Early.
Passing on there not that many patients have been able to find in a trial. So the focus is really on the.
Speaker 3: study for that reason, but still there's an unmet need for patients of all ages for rectum dropout.
Patriotic study for that reason, but still there is an unmet.
Unmet need for patients of all ages for Ret syndrome.
So we could expect you could even start rolling.
Speaker 8: So we could expect you could even start a rolling submission with the adults trial and if need be, that could be approved first before the pediatric really gets filed.
Submission with the adult trial, and if need be that that could be approved first before the pediatric all really gets filed.
Speaker 3: I would say it cannot be excluded, and, but I cannot promise it either. So, that's why I said we would have with Avatar study and the U.S. REC study two independent placebo-controlled study in a rare disease, which usually is beyond the request from, for rare diseases usually. So, this would be, if successful, a very powerful pact.
I would say it cannot be excluded and but I cannot promise that either so that's why I said, we would have with avatar study in the U S recipe to independent.
Placebo controlled study in a rare disease, which usually is beyond the request from for rare diseases, usually so this would be.
If successful a very powerful package.
Got it.
Speaker 8: Congratulations again on all the progress and Happy Thanksgiving.
Congratulations again on all the progress and happy Thanksgiving.
Happy Thanksgiving. Thank you.
And your next question comes the yen sung from <unk>. Your line is open.
Speaker 1: And our next question comes from Yun Zong from BTIG. Your line is open.
Speaker 9: Hi, thanks very much for taking the question. So the first one is on Rett syndrome. And so the definition of a responder is that on each efficacy endpoint, is that going to be the same in pediatric patients as compared to an adult patient? And also, the definition of a responder, is that consistent with how clinicians are viewing as clinically meaningful improvement?
Hi, Thanks, very much for taking my questions. So the first one is on ret syndrome.
So the definition of a responder is that each efficacy endpoint is that going to be the same in pediatric patients as compared to an adult patient and also the definition of a responder is that consistent with how clinicians are viewing us.
Clinically meaningful improvement.
Speaker 3: That's correct. It's consistent with the first study and it's consistent with the assessment of the physician. That's correct.
That's correct, it's consistent with the first study consistent with your assessment of optimization that's correct.
Speaker 9: Okay, then I think, I didn't see an update on the 371 program in 2020, sorry, 2022 in terms of upcoming milestones, so I just wanted to check if frontal temporal dementia is still going to be the first indication for that program and when do you expect a study potentially to?
Okay, then I think of.
I didn't see an update on the.
371 program in 2020, sorry, 2022 in terms of upcoming milestones. So just wanted to check if front.
Frontal temporal dementia is still going to be the first indication for that program and when do you expect a study potentially two to start.
Speaker 3: Right, so we have mentioned that we would move ahead with prototype dementia, but we'd like to have really the solid phase one data in hand before we say we commit to this. And, but we definitely will move forward with FTP or any other related dementia indication.
Right. So we have mentioned that we would move ahead with prototypical bencher, but we'd like to have really solid phase one data in hand, before we say we commit to this end.
But we definitely will move forward with <unk> or any other related dementia indication.
Speaker 9: Okay. And then on the pipeline, I think, well, it's very encouraged to be able to target multiple indications, but just wonder which indications do you think you would like to prioritize going forward? Of course, the Rett syndrome is going to be the lead indication, but which indication do you think might be suited for partnerships?
Okay, and then on the pipeline I think it's very encouraging.
So to be able to target multiple indications, but just.
I, just wonder, which indications do you think you would like to prioritize going forward of course, the ret syndrome and.
It's going to be done.
Lead indication for <unk>.
Which indications do you think might be suited for partnerships.
So we believe that we have.
Speaker 3: have with the rare disease franchise, which is Rett Syndrome, Fragile X, and others, the ability, and it's not the first time that a company has built this into a commercial entity with rare disease, targeting rare diseases.
I have with the rare disease franchise, which is Brexit drove X and others.
Ability and Thats not in the first time that our company has built.
This into a commercial entity with rare disease targeting rare diseases, so that seems to be very durable pumps.
Speaker 3: So that seems to be very doable. It comes to the indication like Alzheimer's disease and Parkinson's disease, which requires also the involvement of a detailing general practitioners.
So the indications.
The disease, and Parkinson's disease, which requires most of the involvement of a detailing general practices practitioners physicians of general positions than it might be more powerful to penetrate the market with the support of a large pharma partner.
Speaker 3: positions of general positions, then it might be more powerful to penetrate the market.
Speaker 3: with the support of a large pharma partner and, at the right time, to make sure that we retain most upside for Anadex and for our shareholders.
At the right time.
To make sure that we retain most upside for <unk> and for our shareholders.
Speaker 3: This will be done at the right time and not prematurely to give up too much of the upside. But it is no doubt that these large indications require additional support. Okay. Great. Thank you.
This will be done at the right time.
And not prematurely.
Give up too much of the upside, but it is no doubt that these large indications require additional support.
Okay, great. Thank you very much and happy Thanksgiving.
Thank you likewise.
And our next question comes from Paul Nori from Nobel Equity Your line is open.
Speaker 1: And our next question comes from Paul Norrie from Noble Equity. Your line is open.
Hi, My questions were on the Parkinson's programs and Theyre already answered so thanks for taking the question though.
Speaker 10: i have my questions were on the parkinson's programs and uh... they're already answered so thanks for taking the question
Thank you.
And that concludes the question and answer question.
Speaker 1: And that also concludes the conference call. Thank you for participating. You may now.
And then also concludes the conference call. Thank you for participating you may now disconnect.
Speaker 11: The.
Okay.
Yes.
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Sure.
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