Good afternoon. My name is Adrienne, and I'll be your conference call operator today. Welcome to the Anavex Life Sciences Fiscal 20 21 Third Quarter Conference Call. As a reminder, this conference call is being recorded. I would now like to turn the call over to your host for today's conference, Clint Tomlinson.
Please go ahead.
Thank you, and good afternoon, everyone. We appreciate you joining us today for Anavex Life Sciences' 3rd quarter conference call call to review financial results and discuss the company's business updates. A taped replay of this call will be available after the call. The call will also be available for replay on Anavex's website at www.anavex.com. With us today is Doctor.
Christopher Missling, President and Chief Executive Officer and Saundra Breunisch, Principal Financial Officer. Call. Following management's remarks, there will be a question and answer session. Before we begin, please note that during this conference call, 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. We encourage you to review the company's filings with the SEC. This includes without limitation the company's forms 10 ks and 10 Q, which identify the specific factors that may cause actual results or events call to differ materially from those described in these forward looking statements. These factors may include, or 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 the call over to Doctor.
Nisling.
Thank you, Clint, and we appreciate everyone joining us on today's conference call to review our financial results and describe the company's growth strategy. Let me start by stating that we can proceed into the remainder of 2021 with a background of a strong balance sheet of over $157,000,000 in cash and no debt. Starting with our lead drug candidate, ANAVEX-two seventy three, we expect to announce top line results from the confirmatory, double blind, placebo controlled, late stage Phase IIbIII study in Alzheimer's disease in second half twenty 22. The double blind placebo controlled 509 patient late stage Phase IIbthree Banavex 273 trial in patients with Alzheimer disease exceeded enrollment beyond 4 50 patients at 52 sites across North America, Europe and Australia using ADAS COG, Cognition and ADCS ADL activities of daily living and function as primary endpoints. This multicenter, double blind clinical trial is measuring efficacy, tolerability and safety of 2 different once daily oral ANAVEX-two seventy three doses or placebo.
ANAVEX-two seventy three is an orally available small molecule activator of the sigma-one receptor. Data suggests that activation of sigma-one results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promote neuroplasticity. The study includes a pre specified precision medicine biomarker, Sigma-one gene expression, which demonstrated correlation with direct measures of clinical benefit, cognition and activities of daily living call. And following the presentation in the previous Phase 2 Alzheimer's disease, TEGAN data previously confirmed dose dependent target engagement of Sigma-1 with Anavex-two seventy three. As a reminder, our clinical strategy is clearly differentiated from other biopharma companies.
In clinical studies in CNS, Anavex is continuing to pioneer the approach of big data in clinical trials to leverage the relevance of phenotypic and genotypic precision medicine analysis of whole exome sequencing and gene expression data in drug development and in particular the potential to identify patients' genetic variants and gene expression changes that may predict increased chances of success of Alzheimer disease, Parkinson's disease and Rett syndrome treatments. Additionally, we can announce that we exceeded the enrollment target for the precision medicine ANavex-two seventy three Phase twothree AVATAR clinical trial in patients with Rett syndrome. And currently top line results from this study are expected in the second half of twenty twenty one. Further clinical milestones are provided in Anavex Life Sciences' latest corporate presentation available at www.arx.com. And now I would like to direct the call to Sandra Boenisch, and Principal Financial Officer of Anavex for a brief financial summary of the recently reported quarter.
Thank you, Christopher, and good afternoon to everyone. We can report a significant increase in cash runway. Our cash position on June 30, 2021 was $157,600,000 which we believe is sufficient cash runway to fund operations and clinical programs beyond 2025. We reported a net loss of $10,200,000 for the current quarter call or $0.14 per share as compared to $6,500,000 or $0.11 per share in the comparable quarter last year. Research and development expenses for the quarter were $9,000,000 compared to $6,700,000 for the comparable quarter of fiscal 2020.
This increase is primarily attributable to the continued advancement of our ongoing clinical trials, most notably call, the full enrollment of our international Phase IIbIII Alzheimer's disease trial and the continued enrollment and advancement of the International Rett Syndrome Program. General and administrative expenses were $2,400,000 for the quarter as compared to $1,400,000 for the prior year period. The increase is associated with the growth of our team and non recurring costs associated with our Annual General Meeting. Thank you. And now I will turn the call back to you, Christopher.
Thank you, Sandra. We are Seemly excited and we believe that Anavex has never been in a stronger position than it is today with our recent double blinded placebo controlled study results correlating with predictive biomarker outcomes and the strongest balance sheet to date and the further strengthening of the Anavex team. In summary, we believe we are positioned for further growth and expect a catalyst bridge upcoming 12 months. So we look forward to providing further updates as advancement continues. I would like We'll now open the call for questions.
Operator, please go ahead.
Thank you. At this time, we'll be conducting a question and answer session. Call. And our first question comes from Charles Duncan from Cantor. Your line is open.
How are you doing? This is Pete Sapropoulos on for Charles. Good afternoon, Christopher and team. Congratulations on the progress and certainly appreciate all the release. So I have one question regarding the RET program.
When you think about the efficacy measures that were made and we're seeing and the efficacy seen in the U. S. Adult study, how do you sort of feel about the sample size or the planned effect size out of AVATAR?
So we have been observed in the U. S. Rett study in a low dose study of 5 milligram daily orally, that effect size in the RCQ was 1.1 Cohen's D, which is considered very large. And for the Adam score, it was even larger, 1.3. So these are very large effect sizes.
In the ABATA study, we even have a larger population in the placebo controlled study And the doses are higher than 5 milligram. So we expect if there is a dose response curve, which Positive dose response curve, which we right now assume it is to be the case that this effect size will be sufficient for a positive readout in the AVATAR study as well.
Thank you All that color. Another question I have regarding the REV program is in clinicaltrials.gov, the pediatric excellence study states that the estimated completion date is about November 2021. Are you still on track to complete the study by that time. And the reason why I'm asking is I know that I believe that you have a large part of it being conducted in Australia. And now unfortunately, they're going back into lockdown in some parts because of COVID.
The study is right now still enrolling well and the ability to continue the trial is still the case. What we cannot know for certainty how much enrollment is impacted by this current situation in Australia, but we still believe as of today that the timelines are accurate to be able to complete the trial in the stated time frame. But if this will change, we will update call? Accordingly.
All right. Thank you. And just one last question on the Alzheimer's program. Can you provide some color on the rollover rates for the Phase IIbIII study into the open label extension?
We were really Very impressed with the very high rollover rate into the open label study, which was over 95%, which is extremely high. And we also have been informed that the patients who now are reaching The end of this extension study open label have requested and the respective caregiver to stay on study drug. And so we have now been able to also extend because of this request the open label study by another period of time for allowing the open extension study to give these patients continued access to study drug. But I want to point out that does not affect the timeline of the placebo controlled study. So it's just in parallel The ability of the patients who finished the placebo console study to stay on study drug without interruption.
That's good. It's probably perceived benefit and probably reflects a lack of tolerability issues. So thank you very much for taking my questions and congratulations
again. Thanks.
And your next question comes from Sumit Roy from Jones Research. Your line is open. Call. I apologize, it's Sumit Roy from Jones Trading.
Hi, thank you for taking my questions. First question on the adult avatar RET study. Do we have an idea if when you're having FDA conversation and if the trial size needs to be modified to turn into to a pivotal trial, if you can give us any color on that. And also some color on the dosing For both the Avatara and Xcellence studies, what kind of dose cohorts are should we expect?
Right. So we are in the dialogue right now with the agency. We have Fast Track designation and orphan drug designation as well as the voucher eligibility for Rett syndrome with Anavex 2CM3. So we're right now in dialogue with the agency and I would like to provide an update With updates as soon as we have that, that would be more appropriate. The second question is regarding the doses.
We have a higher dose than 5 milligram, and We have used a dose of up to 50 milligram in the Parkinson's dementia study and in the Alzheimer study. But the reason why we're keeping this Dosing specifics right now within the blinded information is because the study is in a population which is very new to this trial, And we are concerned that there might be between the family interactions, which could lead to some Unblinding of the study or inadvertently to learning about the effect of the drug and Making a calculation back on the envelope, if more drug is given, how much the effect would be based on the existing data of the 5 milligram arm. So that's the reason why we keep this right now blinded, but we will obviously disclose it when we have the data, But it is higher than the 5 milligram dose.
I see. Very interesting. And One last question on the Alzheimer's trial. Could you give us any color on the baseline MMSE of these patients, it seems like a wide range 20 to 28. What should we be expecting more Early stage or mild to moderate enrolling in your Phase IIbIII trial?
Right. So the rationale for that The range was we have seen in the Phase 2a prior a very positive response across the entire of MMSE baseline score, which started from 16 to 28. But we noticed that the patients above 20 MMSE The ability to improve from baseline on a net positive to reverse The disease symptoms, so the cognition was improved and not only the ability to show a delay of their cognitive decline, while the patients below 20 MMSE were able to stay on a stable score. So the other reason why we included 2020 and higher is that these patients are better, still in a better way to comply with the trial regimen. You have more advanced cognitive impairment, often that is not the case.
But the patient is really like a very broad average within this score of between 2028, which is really also the identified And the highest unmet need with Alzheimer's disease today.
I see. So you would you won't break it up into 2 groups. You would Keep it as the entire 20 to 28 groups and that's how you will present the data?
Right, because we saw that there was no difference of the improvement if you were 28 or you were 20. In both cases, we saw with appropriate dose an improvement of the score.
Got it. Thank you so much for taking the questions. I'll hop back on the queue and congrats again on the progress.
Thank you.
And your next question comes from Ram Selvaraju from H. C. Wainwright. Your line is open.
Thanks very much for taking my questions. First of all, Again, alluding to the potential role of lauquamazine in Alzheimer's disease. Can you comment on some of the preclinical information that's been presented recently and whether this would potentially point to a protective role for Blaucomazine And specifically, if you can speculate on in which population the use of block amazine as a protective might be most relevant from a clinical perspective. For example, in those people who are exhibiting signs of prodromal Alzheimer's disease or mild cognitive impairment. I understand that it's an early stage at which to be thinking about that.
I just wanted to get your thoughts in this regard.
Appreciate the question. We have learned in a Disease in a preclinical animal model of prevention. So it's Basically, you gave to healthy mice the drug for 7 days, every day for 7 call. They stop the treatment and then you inject the data into the brain, which is unknown Animal model to pathology. And then these animals usually, they get sick, they lose cognition.
And you see that in the measures of water maze behavior and so forth. And we noted in the arm which had been given This 7 days treatment, prevention that these animals never developed After the injection with ADETA, any symptoms of cognitive impairment, they believed and cognitively are stood as their wild type part. But those who were given the abeta and without the pretreatment, They developed a cognitive impairment as you expected from the animal model. So this is the observation which led to the potential that DEVA1 activation with Anavex273, which as we know by now is extremely up stream and possibly able to prevent the cellular stress, which is caused by the abeta Interjection and so in the entire challenges of the pathology, which is not limited to A beta, that this could be used as a prevention also in the future, which has to be awfully confirmed. The intelligence we have on the ALCEEMA study seems to be also giving credibility in that direction since we noticed a gradual ability to improve conversion with earlier stage status of the capacities.
So when I mentioned before the cognitive impairment Lower than 20 MMEC and the lower score means more cognition or more cognitive impairment, better cognitive impairment that those patients were better off the earlier you treat them. So we might be able to Continue the trajectory of into the ability to prevent the cognitive impairment to even take place when we give the drug to patients which are either as you point out mildly cognitive impaired or before even Any symptoms of cognitive impairment is present just to be able to always prevent this cellular stress by this activation. And we compete in a way to mini aspirin, which some people take for avoiding cardiovascular problems and they do that every morning for breakfast. So I'm not saying that this is confirmed in human, But eventually, we will be able one day to tackle that and that is the plan in a study.
Great. Thanks. And also I wanted to ask about your thoughts regarding lessons, takeaways from the Rett syndrome experience with larcamizine and their applicability to your potential clinical development initiative with the compound in Fragilex syndrome. And if you could also give us a sense of when you anticipate the Fragile X syndrome program to initiate patient enrollment. Thank you.
We have included in our Rett syndrome clinical trial a measure which is called Adams And that score is interesting enough a secondary score in the Rett syndrome study, but it is often used as a primary endpoint in fragile X studies. And that Adam's score was extremely positive with behavior improvement of general mood, of anxiety and compulsive behavior improvement, which were very significant and very broad and global. So we believe that In a data readout of the preclinical data of predilex with ANAVEX-two seventy three, which we submitted to a Peer reviewed paper of a nature paper, which we expect to come out forthcoming that this could be a very good basis for the rationalizing progressing Fragile X into an indication of choice also for ANAVEX2-seventy three. And the idea is obviously to move this forward as as soon as possible given the unmet need of Fragile X, which is the largest population of autism spectrum disorder. And Tregt syndrome is Also part of the family of the autism spectrum disorders, so these are correlated diseases, although they are originated in different places in the causation and the causality, but they have similar symptoms and overlapping symptoms with each other.
So we expect the trial to be initiated once we are able to showcase The data in a peer reviewed paper. Thank you very much.
And our next question from Tom Bishop from BI Research.
Hi, Christopher. You said that you expected to include a Phase 3 prevention study Or to initiate 1 for the prevention indication. And I was wondering what the status of this and how
So this is something which we would like to discuss with the agency because it's obviously a very important study and we want to get that right. So we will let that happen first before providing more details on that. But I'd like to just point out the potential to be the case gives us also a great hope that Given the broad upstream effect of the Sigma-1 activation, which is I want to remind everybody, It's not something we came up with, but it's really endogenous, the body owned defense, avoid and to prevent cellular stress, which causes diseases like Alzheimer, but also Parkinson's and other cognitive impairment indication and probably also the degenerative and as well as the and your development indications, which is the reason why we see the strong effect beneficial effect with the drug in this Raw therapeutic different indications. So this is something we'd like to discuss with the agency first before making providing more details. But this is the long shot And this is probably the big prize that one day this could be used as a prevention treatment for all degenerative diseases and not only for treatment.
That's a very exciting possibility. What is the status of the meeting To proceed on the Phase 3 in Parkinson's, sometimes it seems like we lose a lot of time between trials. I know you presented the final call. Data. And I'm just wondering what the time line is here.
Have you scheduled a meeting with FDA or I had one more.
That's a very good question. The key background is to know that the trial itself has finished, but the analysis is not limited to the endpoints which we reported. There are more work up ongoing right now. And this is also unique for Anavex and differentiates other companies that we have included in our trials, including the PDD trial, The Tarkas and disease dementia trial, the whole genomic exome analysis, both DNA, but also RNA. And you know that we reported the RNA changes of the Sigma-one gene.
What we have not yet done is they have the intelligence on the RNA of all the other genes, which is the whole genome. So there will be so much more intelligence from this PDD study, which we are right now putting together. And once we have that, then we will have the ability to put this all forward Because this will determine a much more robust Phase 3 design of a pivotal study in Parkinson's disease dementia as well as in Parkinson's, but that requires also the dialogue with the agency first. But before that, we have to have the entire data at hand.
Okay. And the status of the Michael J. Fox $1,000,000 Imaging study, Is Michael J. Fox doing that independently of you? I mean, in the use of flying the drug, but they're doing the imaging.
I mean, how is that going?
No, it's basically a grant that allows us to do the study and we are in the process of starting this year. It's a study which will capture the imaging and the profile of the ANAVEX system 3 of the drug in the brain in the humans with the same pet ligand which we have done already in animals. So this will be a confirmatory of that effect in human brains and that is study, which was fully funded by Michael Fox Foundation, which we are executing and they basically funded that study with a grant.
I see. Okay. And you indicated a mystery indication earlier this year. And also there's a 371 trial, is there not Phase 1 ongoing? Could you comment on those 2?
What are you saying? I don't know.
Right. It's not a mystery indication. We have only said we have not disclosed yet the indication. We have several animal models where Anavex 2CM centimeters
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centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters centimeters confirmatory effect on disease, which is rare in nature, ultra rare in nature. And we want to make sure before we move forward with the clinical trial that we pick the right one to do because we have the choice of several indications And we are doing this right now. So once we have that completed that assessment, we will disclose that indication and start the trial also right away. And regarding ANAVEX371, it's basically another molecule, had its own IP, and that is now in a Phase I, and we expect data readout in the second half of this year. It's progressing well, and we are excited because it confirms the validates the mechanism of action of our platform portfolio to focus on Sigma-1 modulation.
And also, it has received offering drug designation for frontal temporal dementia, which is also an unmet need. So we are now preparing after the Phase I the next stage of that, which will be study in an indication of cognitive impairment, it could very well be a frontal deplazemetra call or another indication with an unmet need with cognitive impairment.
But the Phase I study is are you saying it's done?
It is about to wrap up and we will be able to report this the data in the second half of this year. That's correct.
Will that include any Efficacy data or it's just a safety trial, Leroy?
It's predominantly a safety trial, but I will allow us to basically share the data once we have it and that gives us better visibility on what is included in that outcome measures beyond Phase 1 data or from safety data?
Okay. Well, I've been very encouraged by The breadth of the positive readouts you're getting on a variety of indications, and I think that helps support the idea that A2-seventy three really does something in the bank, Which I think you have a little doubt of and I do too, but it's just good to see so many positive readouts coming. Thank you.
If I may add, it's really important to notice and to highlight the fact that in all clinical trials we have performed so far, Not only was ANAVEX2-seventy three efficacious at the right doses, but it also demonstrated a dose response call, which is always a very clear indication of our effect. And thirdly, we have noticed that all the data, all the trials so far performed had a very strong biomarker of response or predictive biomarker response, which was borne by the level of mRNA expression of the target of our drug itself. So there's really no better way of showing efficacy and confirming efficacy of a drug with these strong biomarker outcomes, which correlated with all the primary and secondary endpoints call of the trials we have performed.
Like I said, it's very encouraging. All right. Thank you.
Thank you.
Thank you, ladies and gentlemen. This concludes today's conference call. You may now disconnect.