Thank you and good afternoon everyone. We appreciate you joining us today for Anavex Life Sciences conference call and webcast. Our agenda is to review the company's financial results for the Q2 of its fiscal 2021 financial year and provide a clinical study and business update. A taped replay of this call will be available approximately 2 hours after the call's conclusion and will remain available for 1 month. 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 Sandra Boenisch, Principal Financial Officer. Doctor. Missling and Ms. Boenisch will make prepared remarks and then we will take questions from equity analysts.
Before we begin, Please note that during this conference call, the company will make some projections and forward looking statements regarding future events. 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 to differ materially from those described in these forward looking statements. These factors may include, without limitation, risks inherent in development and or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, the 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.
Missling.
Thank you, Clint. We appreciate everyone joining us on today's conference call to review our financial results and business updates. Let me start with our lead drug candidate, ANAVEX-two seventy three, which is currently in a Phase IIbIII Alzheimer's disease clinical trial utilizing differentiated patient selection criteria. The study is presently over 98% enrolled and randomized. So we are very close to complete enrollment.
What is important is that in that regard is that the independent data safety monitoring board DSMB for the Phase 2bthree Alzheimer disease study of ANAVEX-two seventy three has completed its recent preplanned Review of the preliminary Phase IIbthree study data. As specified in the protocol, the DSMB reviewed the interim safety data For the Phase 2bthree Alzheimer study, AD004 and its open label extension attention AD study. Upon review of the interim safety data, the DSMB made the following recommendation. The DSMB recommendation is to continue the This is very encouraging news and it indicates we are on the right track with these studies. Additionally, Anavex 2CM3 has been featured recently in a peer reviewed publication in the Journal of Neuropharmacology titled Future avenues for Alzheimer's disease detection and therapy from the series of the special issue on the quest for disease modifying therapies for new degenerative disorders.
Separately, the Independent Data Safety Monitoring Board, PSMB, also That's for the ongoing clinical trial program including the late stage AVATAR, RS002, excellence RS003 And US Rett Syndrome Extension Study RSEP001 of ANVEX2-seventy three has also completed its recently planned Review of the respective interim safety data for these 3 separate clinical studies. Upon review of the interim safety data, The DSMB made the following recommendation for each of the 3 studies. The DSMB recommendation is to continue the studies Without modifications, this is very good news and indicates we are on the right path with all these studies. As a reminder of our clinical strategy is to clearly differentiate from other biopharma companies and clinical studies in CNS. Anavex is continuing to pioneer the approach of big data, including omics in clinical trials to leverage the level Our phenotypic and genotypic precision medicine analysis of whole genome sequencing and gene expression data in drug development And in particular, potential to identify patients' genetic variance and gene expression changes that may predict increased chance of success for Rett syndrome, Parkinson's disease and Alzheimer disease treatments.
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 quarter.
Thank you, Christopher, and good afternoon, everyone. We continue to apply fiscally responsible management of cash utilization with moderate increases within budget. We reported a net loss of $8,200,000 for the quarter or $0.12 per share as compared to $7,200,000 also $0.12 per share in the comparable quarter of last year. Research and development expenses were $6,700,000 for the quarter compared to $6,100,000 for the comparable quarter of fiscal 2020. The increase is primarily attributable to the continued advancement of our ongoing clinical trial.
General and administrative expenses were 2,200,000 for the quarter as compared to $1,700,000 for the prior year period. The increase is associated with the growth of our team. Our cash position on March 31, 2021 was $75,900,000 which we believe is sufficient cash runway for up to 3 years. Thank you. And I will now turn the call back over to Doctor.
Miss Lane.
Thank you, Sandra. So in summary, we expect a very data rich Current quarter with data readouts from multiple clinical trials and also the remainder of 2021 to be a We look forward to providing further updates as advancements continue. And I would now like to open the call for questions. Operator, please go ahead.
Our first question comes from Charles Duncan, Kenter Fitzgerald. Please go ahead.
Hey, good afternoon, Chris, and team congratulations on the progress. Certainly appreciate the update on enrollment in the Alzheimer's program. So I had a quick question regarding the DSMB decision recently to continue this study without modification. I assume that not only did they look at safety, but did they look at any efficacy endpoints And or was there an opportunity to change the sizing, upsize the trial due to say Data, I'll call it noise or dispersions And that was not done, so that data is tracking as you would have anticipated on a blinded basis?
The ADASIMA study DSMB related question?
Yes.
Right. So the DSMB has access to all data and we understand that this is DSMB Made the decision to continue without changes according to their review. We do know also though that the focus on the review was on safety.
Okay. So they're looking at safety Adverse events, but were they also considering efficacy? Were there any kind of futility analyses? Or did they have the opportunity to recommend upsizing the trial based on data dispersion?
The DSMB has usually a very independent ability of recommendations, But the D and B itself, the priority is on the safety assessment.
Okay. So It sounds like in this case they weren't really looking at efficacy at this point.
Again, they have access to all the data. So That is the best way I can answer this question. So there was not a specific request to look into something like futility.
Okay. Can you provide some color on the rollover rates for that study Into, say, the open label extension, can you give us perspective on whether or not patients are going beyond the Planned dosing period?
Yes. So we have a very high rollover rate from the placebo controlled Part of the study into the attention AD extension study, which is a 2 year 96 A week long extension open label. And we happen to reach the close to the end of this open label extension study. We have been received already requests for continuation of patients on this extension study And we will very likely seek extension of the extension study because of that. And that reminds us of what happened in the Phase 2a, which was also originally limited to a extension period.
And then because of requests from participants and caregivers and physicians investigators, It was extended multiple times to end up a 5 year extension in total. And after the 5 year, as you know, The participants of the Phase 2a study ended up being allowed to continue studies the drug On a human to heart, the exception procedure. And so this is The feedback we received from the larger Alzheimer study Phase IIbIII.
It seems like the positive relative to the perceived benefit that the patient is getting or lack of Tolerability issues, but when you mentioned high rollover, could I assume that's like 90% Rollover rate or something like that. And then secondarily, in terms of the failure rate within Or excuse me, dropout rate within the placebo controlled portion, what were your assumptions and how well did the trial do thus far? Yes. So,
right. So, the percentage is in the range of what you indicated Our retention, so extension into the open label study from what we understand. And we have modeled The dropout rate similar to other drugs in this field with Alzheimer disease, it's a long study. So there is in patients With the impairment like in Alzheimer with multiple diseases, not only Alzheimer, but also cardiovascular features. So there is often this phenomenon that patients do not finish an entire study.
So we have assumed similar rates as in the Other studies in comparable lengths and I think we are not in any form or fashion Different than or worse than these numbers.
Okay. That's helpful. Last Question regarding the Parkinson's disease dementia program. Were you thinking about going to the agency with Say an End of Phase 2 meeting and if so, can you provide some color on when that might be?
Yes. So we said that we would do that. And once the data is completed, we are about to wrap up. You have to understand in the PDD Study, there were a lot of measures included, the CVR system, the UPRS, The actigraphy, the sleep paradigms and most importantly what have been the most time consuming one, The gene analysis and all this together will be put together in the respective report and that will be shared with the FDA And then we will be able to get the recommendation for the pivotal study in PDD.
Could that be this quarter or in
We will do it as soon as we can. We will do it as soon as we can.
Thank you. Our next question comes from Ram Selvaraju, H. C. Wainwright. Please go ahead.
We will go to the next question. It comes from Jeffrey Cohen from Ladenburg Thalmann.
Please go ahead. Hi, Christopher and Sandra. How are you?
Hello. How are you?
Doing fine. Just a few questions. So first on the income statement. Can you help us out on the incentive income for quarter and what that might look like for the balance of the year as far as our modeling purposes?
What is the question exactly?
I wanted to know about the R and D development incentive income of 1.27 for the quarter And how that may look like for the balance of the year?
I think it was $960,000 for the quarter. And we expect that that will remain consistent throughout the remainder of the year.
Okay. Okay, got it. And Christopher, can you give us some insight into the 273 trial more specifically as far as any With the agency as far as fast track or accelerated approval?
For Rett syndrome?
Yes.
Yes. So we have the first study finished, which was the very first study in Rett syndrome, the U. S. Rett syndrome to ARIES-one study and that we had seen a very positive outcome where we presented top line data at recently last year. And we will now be able to share the entire data of the study.
And with that data, we will go to the FDA and discuss with them the path forward given that we have 2 ongoing studies. 1 is the 2 in the Sage age group with higher doses and RS-three which is Also high doses with younger patient population. And we believe that the first study, the RS002 Could be potentially pivotal together with the U. S. Study.
And that is the discussions we like to have with the agency. We'll explore the approval for the younger patient population with the RSV-three.
Okay, got it. And any update on the PDD Phase 2 And the Avatar Phase twothree in RTT, are those timelines still on track? Exactly. So the RS-one
full data will be this quarter and the PDD study will be this quarter In the release of the top line data of the RS002 is expected by mid of this year.
Okay. And that's in the adult population, correct?
That's the adult population higher dose. That's right.
Thank you. And we have Mr. Ram Selvaraju back online. Please go ahead.
Sorry, thank you very much for taking my questions. I apologize. I must have had a problem with the signal before. First of all, I was wondering if you could Comment on strategic thoughts regarding the potential commercialization of bloxamazine in Rett syndrome versus PDD And what implications that may have in terms of Anavex's overall positioning? Are you looking at those indications as opportunities to self commercialize entirely?
Or are you looking at each of them differently in terms of the degree to which Anavex is going to be directly involved in the actual sale of the drug?
That's a very good question. I think we are not the would be not the first company to market a rare disease ourselves if approved. And that is right now the going plan for Rett syndrome. So we have put in place Steps in order to prepare for that. For the PDD, we have to be aware we have to do another study, which is Not which we didn't.
We have planned anyway, a pivotal study because the first study was our Phase 2 proof of concept study. And so we have some more time to think about the second indication TDD in terms of marketing, if we would like to license it Or how to structure a partnership if at all needed going forward. So this will be something we don't have to decide right now. But Tourette syndrome, we would be able to market it ourselves to this current plan in the U. S.
And pursuant to that, Have you made a definitive decision regarding the disposal of the putative priority review voucher that you would be eligible for if you won Approval of Blaucomaecine and Rett syndrome, would you in other words apply the PRV to something else in your own pipeline Or would it be definitive that you would monetize it?
Right. This is the same flexibility we are able to retain Given that we are planning to also open another study shortly after the Rett study In fragile X, where we have very good preclinical data and the drug also is within the family of autism spectrum disorder And where the drug is expected to be also superior to current treatments or symptoms of treatments to current Opportunities in fragile X, but also we have another rare disease, ultra rare disease, which we have not disclosed. So there are different ways to think about that, but we don't have to make the decision yet regarding how to address the voucher.
Okay, great. And one last question. The clinical development timeline with respect to Fragilex, can you just Walk us through what you expect the near term clinical development milestones to be in the Fragilex indication?
So what Rudolfi, you mean?
Start with the near term clinical development The next clinical study, when that is likely to start and when it is likely to report data?
Right. So the RS-one full data will be this quarter. The RS-two top line will be mid of the year and the 3 will be in the second half of this year. And in between discussion with the agency, which will determine How the data can be utilized in order to get approval for the drug for patients which have no therapeutic available for Rett syndrome.
Thank you. Our next question comes from Tom Bishop from Bio Research.
Hi, Christopher. I got a couple of little questions, but I have always assumed that open label extension study means that everyone gets the drug. Is that Great assumption.
Everybody who had participated in the study previously that is open label extension for those who had finished the study Either in placebo or an active drug, they are able to get the drug. Not everybody without any association or being Not involved in the study.
Right. And they get the drug for free, so?
That's right, yes. Nobody has to pay for anything.
Okay. Sandra, could you just tell me the number of shares outstanding now, now, About average for the quarter, but now.
I think we disclosed it in the Q, it should be about 70,000,000
It should be what?
$70,000,000 $70,000,000
Okay. And I was wondering if the how many Alzheimer's patients still From the original A273 study, which was 32 patients are still on the drug. And there's a famous graphic that I like that shows the number of patients that We're on the medium and low dose that showed a sharp decline in MMSC and etcetera. And those on the high dose showing very little decline, but the total of the 2 as I recall was like 21 or 22 patients and I'm Sort of wondering where the others went.
Yes. So that was in the 3 year interim analysis. We will be able to share the remainder timeframe Possibly in the future, but the patients who have been in the study, they have now been Switch over to a eligibility of a humanitarian device exemption. That means they're not in the clinical trial anymore because the The trial has completed officially the extension, but they continue to take the drug. And I understand that Run about it's less than 21.
I think it's between 10 21 patients, if I'm not mistaken.
And so that's
a very encouraging number that there's still people after 5 years taking the drug.
Yes, yes, it's true. Also with regards to I've read some articles where other Companies that are working on the Alzheimer's indication, one guy one of them has a partner and the other one's got NIH funding. And of course, Anavex has neither, but I think that there is a good reason for both, but I'd rather hear from you than me.
I think we're moving very quickly. When you look at those who get the NIH grants, you have to have published data and it takes a long time to get a grant. And even If you get the grant, it takes a long time until you get the money. So that's not preferred way of moving forward the asset. We've been very quick in moving forward the assets.
And the downside of partnering too early is that you are kept to the upside. So as a biotech company, you just have not retained the upside for shareholder. And by pushing this That is the advantage of having more upside for shareholders of Anavex.
Right. And I was just wondering if you've ever had some interest from other people in partnering or if you just shut them down because you want to keep the 100%?
We have always been called upon and knocked on the door To discuss this and we participate regularly at the conferences like BIO early. So we are in discussions With the big pharma companies, but again, it has to be a mutually beneficial structure for striking something. And we are very excited about our projects, our pipeline and we like to retain the upside for that reason we are proceeding as we speak right now. It does not mean that we will not do a partnership eventually at the right time. What we are aiming for is shareholder value and to maximize it And that requires to have this flexibility in mind.
Okay. And last question, the RET-three trial The adolescent trial, is that 3 or 4? 3. Is that due I was wondering how the enrollment is coming on that and what is the size if you could remind us?
Yes. We increased the size from we have said more than 68, 69, so it's going to be more than 69. And we have stated that it will be in the 2nd hour this year and then enrollment is going well.
Thank you. We have a follow-up question from Charles Duncan, Cantor Fitzgerald. Please go ahead.
Hey, Christopher, thanks for taking the follow-up. Had a couple of questions, one back on the PDD and the other is on RET. Going back to the PDD, your answer before, I'm just trying to figure it out. First of all, do you intend to present the data or more findings from that study in the near term? And then with regard to the genomic analysis, I know that lab function has been a little bit COVIDified, if you will, in the last year, but would you Call it days or weeks or months or quarters in which you'd be able to move forward with Perhaps end of Phase 2 meeting with the agency.
Right. So the PDD data will have A lot of data because the entire CGR system has multiple measures. We're talking about total of It's a lot of the cognitive domains have been captured in the system very granularly And there is no total CVR system score and they all capture different levels of cognitive domains like Picture recognition, word recognition, picture accuracy, memory, word accuracy and Short term memory, long term memory, real and reaction time, A short reaction time, memory reaction time. So there are different levels of cognitive tests which are there. So that is Already a lot of measures, which we will present.
And then comes the entire battery of what we call more participating Measures of the Parkinsonian disease like the UBRS, UBRS 1, 2, 3, And then we have the actigraphy, the sleep paradigms. We have 2 different sleep paradigms. And then on top of it, we have the genomic analysis of how the sigma-one moves and what it does for patients in regarding his But despite that, we are now there to finish this up and we expect this data to be announced, presented this quarter, this quarter as we speak. And the discussion with the agency obviously will follow-up very rapidly once we have that. Just understand The agency requests a report.
Their report has to be completed, has to be audited and reviewed and signed. And that's basically requires also some additional time. But this is now all done. And this will be all done when we submit to the agency. And so we expect it really to be, I would say, more question of months, not days, but not too many months.
That's probably the best way to describe it.
Okay. That's helpful. And then my follow-up on the Rett program, just Quickly on the safety database in terms of its sizing given the range of age groups that you're studying, What has the agency said to you in terms of what's going to be required for that safety database? It is Rare disorders, so I imagine it's not too onerous.
Yes. So we included in all our RET Clinical studies and extension study. So in the RS-one, it's a 36 months 36 week study Of extension open label that was already originally extended before, we have a 1 year extension In the RS002 and also the same 1 year extension RS003 and given the history and we believe these are sufficient Time lines for safety for safety extension and from the feedback Also from the European AU side of the equation, but we believe that what will happen very likely and that's What Anavex is committing to patients and that's what we're here for is that those patients who started a study with Anavex We'll also have always a chance to continue to stay on the study drug of ANAVEX-two seventy three. So it's very likely we'll also extend these extensions beyond the time You're welcome. Thank you.
Thank you. And at this moment, I would like to hand the call back to Christopher Mitzlin.
So thank you very much. And I want to reiterate, we're very excited about our program. We believe we have a really strong pipeline And a platform which allows us to address multiple unmet needs. And in summary, we expect a very data rich current quarter With data readouts from multiple clinical trials and the remainder of 2021 to be also a catalyst rich year for Anavex. With that, we look forward to providing further updates as advancements continue and stay tuned and thank you very much and have a good week good afternoon.
Thank you.