Q2 2021 BioXcel Therapeutics Inc Earnings Call

[music].

Good morning, and welcome to the buyer of XL Therapeutics for 2021 financial results Conference call.

Operator: Good morning, and welcome to the BioXL Therapeutics 2020 Financial Results Conference Call. At this time, all participants are in a listen-only mode.

At this time all participants are in a listen only mode. During.

Operator: If, during the conference, you require operator assistance, please press star zero on your telephone keypad. After the presentation, there will be a question and answer session. If you would like to register a question, you may press star 1 on your telephone keypad. Just to remind everyone, certain matters discussed in today's conference call and or answers that may be given to questions asked are forward-looking statements that are subject to risks and uncertainties related to future events and or the future financial performance of the company. However, actual results could differ materially from those anticipated in these forward-looking statements.

During the conference you require operator assistance. Please press star zero on your telephone keypad.

After the presentation, there will be a question and answer session. He would like to register of question You May Press Star 1 on your telephone keypad.

Just to remind everyone certain matters discussed in today's conference call and for answers that maybe given to questions asked are forward looking statements that are subject to risks and uncertainties related to future events and or the future financial performance of the company actual results could differ materially from those anticipated in these forward looking statements.

The risk factors that may affect the results are detailed in the company's most recent public filings with the U S Securities and Exchange Commission, including its quarterly report on form 10-Q for the quarterly period ended June 30 of 2021, which can be found on its website at www Biocryst out therapeutics Dot com.

Operator: The risk factors that may affect results are detailed in the company's most recent public filings with the U.S. Securities and Exchange Commission, including its quarterly report on Form 10Q for the quarterly period ended June 30, 2021, which can be found on its website at www.com or on www.seceltherapeutics.com. As a reminder, today's conference is being recorded. Joining us on today's call are Dr. Vimometta, Chief Executive Officer; Richard Steinhart, Chief Financial Officer; Will Kane, Chief Commercial Officer; Dr. Vince O'Neill, Chief Medical Officer; Dr. Frank Yacan, Chief Scientific Officer; and Dr. Rob Rissinger, Senior Vice President of Clinical Development. It is now my pleasure to turn the call over to Dr. META, the CEO of Bio-Excel. Thank you, sir. Please go ahead.

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As a reminder, today's conference is being recorded George.

That's on today's call our Doctor of criminal matter, Chief Executive Officer, Rochet for Richard Steinhart, Chief Financial Officer will Kane, Chief Commercial Officer, Dr. Vince O'neill, Chief Medical Officer, Dr. Frank JAKO, Chief Scientific Officer, and Dr. Rob Crissinger Senior Vice President of clinical development.

Now my pleasure to turn the call over the Doctor met the the CEO of bio ex out. Thank you. Sir. Please go ahead.

Thank you operator.

Vimal D. Mehta: Thank you, operator. Welcome everyone, and thank you for joining our call today. We had a very exciting and productive second quarter as we continued to execute on our key initiatives that we believe will deliver long-term value to patients, healthcare providers, and our shareholders. As many of you are familiar, we are a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immunology. Our two most advanced programs are BXL 501, our neuroscience investigational candidate that is being developed for the treatment of agitations, and BXL 701, our immunoncology investigational candidate which is being developed for the treatment of aggressive forms of prostate cancer and advanced solidity.

Welcome everyone and thank you for joining our call today.

We had a very exciting and productive second quarter as we continue to execute on our key initiative that we believe will deliver long term value to patients health care providers and others share holders.

As many of you are familiar we are of clinical stage biopharmaceutical company utilizing artificial intelligence the approaches towards all of the bonds for making medicines in neuroscience and immuno oncology.

Our 2 most of advanced programs I'd be ex fuel 5 of 1 of our neuroscience investigational candidate that is being developed for the treatment of agitation N V. Axiom. The 7 O..1 hour of immuno oncology investigational candidate, which is being downloads for the treatment of aggressive form of prostate cancer and advanced solid tumors.

I will begin today's call with an overview of the progress we have made with all of our neuroscience programs and plans to expand into additional neuropsychiatric disorders.

Vimal D. Mehta: I will begin today's call with an overview of the progress we have made with our neuroscience program and plans to expand into additional neuropsychiatric disorders. In just over two and a half years, we were able to go from conducting our first inhuman trial with BXEL 501 to receiving FDA acceptance of our NDA filing for the treatment of acute agitation associated with schizophrenia and bipolar disorders. Thanks to the hard work of the entire BioXL team, we were able to successfully conduct a total of seven clinical trials in over 800 patients across a range of disorders, laying a robust clinical foundation for our 501. This is in alignment with our three-pillar growth strategy for our neuroscience franchise, which is designed to expand follow-on indications, including dementia, across multiple treatment settings and our global business.

In just over 2 and the half years.

We weren't able to go from conducting a cluster of human trial would be ex fuel probably won't want to receiving FDA acceptance of our NDA filing for the treatment of acute agitation associated with schizophrenia and bipolar disorder of London.

Thanks to the hard work of the entire box of DS, We were able to successfully conduct a total of 7 clinical trials you know what.

800 patients across a range of the sort of laying the robust clinical foundation for our 501 program.

This is in alignment with our 3 pillar growth strategy for our neuroscience franchise, which is designed to expand follow on indications, including the message here.

Lots of multiple different detecting and our global reach.

Vimal D. Mehta: I'm pleased to report that we continue to successfully execute on these key initiatives and have made important progress across our clinical, regulatory, and commercial objectives for our lead program, BXL 501. With regard to clinical progress, we continue to advance our plans for expanding the use of BXL 501 into additional indications. We remain on track to commence a Phase C program of BXL 501 for the key treatment of agitation associated with dementia in the fourth quarter of this year, which has been granted breakthrough therapy designation by diabetes. Agitation associated with dementia, including Alzheimer's disease, is a significant market opportunity as the need for better treatment options is immense.

I'm pleased to report that we continue to successfully execute on these key initiatives and have made important progress of cost across our clinical regulatory and commercial objectives for our lead program in beauty of firewall.

With regards to clinical progress we continue to advance our plans the product expanding the use of fiber 1 into additional indications we the.

Remain on track to come out and say of Phase 3 program of <unk> 501 for the acute treatment of agitation associated with dementia in the fourth quarter of this year, which has been granted breakthrough therapy designation by the FDA.

Agitation associated with dementia, including Alzheimer's disease is the significant market opportunity as the need for better treatment options.

We also plan to report top line data from our ongoing 14 microgram dose phase II tranquility trial of <unk> 501 in patients with dementia during the fourth quarter of 2021.

Vimal D. Mehta: We also plan to report top-line data from our ongoing 14 microgram dose, Phase 2, Tranquility Trial of BXL 50501 in patients with dementia during the fourth quarter of 2020. In May, we received FDA acceptance for the filing of our NDA for BXL 501 with a priority action date of January 5th, 2022 for the acute treatment of vegetation associated with schizophrenia and bipolar disorder. This acceptance marks important progress towards our goal of providing a new treatment option for the millions of patients struggling with acute erectus.

In May we received FDA acceptance for the filing of our NDA for the axial fiber 1 with the Purdue for action date of January 5.2000 joined the true for the acute treatment of agitation associated with schizophrenia and bipolar disorder.

This except the marked important progress towards our goal of providing a new treatment option for the millions of patients struggling with the acute agitation.

Vimal D. Mehta: If approved, this would be the first major medical advancement for these two indications in almost a decade. In addition, we initiated a pediatric study 4501 for the acute treatment of agitation associated with schizophrenia and bipolar disorder.

If approved this would be the first major medical advancement for these 2 indication and almost for a decade. In addition, we initiated a pediatric study for fiber 1 for the acute treatment of agitation associated with schizophrenia and bipolar disorder.

While the FDA review of our application we are executing on our comprehensive commercial readiness of strategy to ensure that if we receive FDA approval, we are well positioned to bring <unk> to patients and healthcare providers across the U S.

Vimal D. Mehta: While the FDA reviews our application, we are executing on our comprehensive commercial readiness strategy to ensure that if we receive FDA approval, we are well positioned to bring BXL501 to patients and healthcare providers across the year. Our key initiatives in the last quarter included the deployment of our medical science liaison and medical managed care teams, who are continuing to engage in scientific and medical to medical exchange with healthcare professionals and peers, respectively, to provide key insight to support commercial.

Our key initiatives in the last quarter included the deployment of our medical science liaison and medically managed care teams, who are continuing to engage in scientific and medical to medical exchange with the health care professionals NPS, respectively to provide key insights to support from the show.

Of the strategy.

In may of data from the CDC safety for US were presented at the American Psychiatric Association annual meeting and at the International Society for bipolar disorder Global conference in our niche of data will be presented of leading medical conferences. During the second half of this year.

Vimal D. Mehta: In May, data from the Serenity FACC trials were presented at the American Psychiatric Association Enval Meeting and at the International Society for Bipolar Disorders, Global Conference, and Additional data will be presented at leading medical conferences during the second hour.

In late June we had a day part.

Vimal D. Mehta: In late June, we held a commercial day, where our team shared market insights, commercial opportunities, and highlighted launch readiness plans. In preparation for our go-to market strategy, we have augmented our commercial teams with several key hires and are further defining the designs of our sales force and market access strategy. We are advancing our strategic initiatives for Geographic and Expansion, and our plans are underway for a marketing authorization application with the European Medicines Agency for BXL 501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders one.

Sure day.

Our team share market inside the commercial opportunity in the highlighting launch readiness plan.

In preparation for our go to market. The strategy, we have augmented commercial teams with several key hires and are part of the defining the designs of our sales force and market access strategy.

We are advancing our strategic initiatives for geography.

Expansion, our plans are under way for a marketing authorization application with the European Medicines agency for VX 501 for the acute treatment of agitation associated with the schizophrenia and bipolar disorders wondering.

We believe the European market represents a sizable opportunity similar to the U S, which we estimate is 9 million patients in approximately volume 40 million agitation episodes per year.

Vimal D. Mehta: We believe the European market represents a sizable opportunity similar to the US, which we estimate is 9 million patients and approximately 40 million agitation episodes per year. All of this work will serve as a solid foundation for the commercial launch of additional follow-on indications, paving the way for further growth opportunities in our neuroscience. We remain confident in BXL 501's potential across multiple indications where there is a significant need for new, innovative treatment options.

All of this works well.

The solid foundation for the commercial launch of additional follow on indications paving the way for further growth opportunities.

Our neuroscience business.

We remain confident in VA till probably the 1 potential across multiple indications, where there is of significant need for new innovative treatment options.

We continue to leverage our AI technology, and our proprietary clinical data.

Vimal D. Mehta: We continue to leverage our AI technologies and our proprietary clinical data to strategically expand market opportunity across a broad spectrum of neuropsychiatric disorders where BXL 501 can become a valuable therapeutic option. In the fall of this year, we plan to host an R&D day to provide an update on our BXL 501 program, pipeline within a product, and additional neuroscience pipeline candidates. We look forward to providing more information in the coming weeks. Looking beyond our neuroscience program, our Phase 1, 2B study for Caster Resistant Prostate Cancer with BXL 501 is nearing completion, and a more complete efficacy update from the phase 2 portion of the trial is expected in the third quarter of this year. Based on these results, we plan to design our next phase of the CRPC program and leverage BXL 701's novel mechanism to build our immunoncology.

Strategically expanding market opportunity across a broad spectrum of neuropsychiatric disorders.

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Valuable therapeutic options.

Yeah.

In the fall of this year, we plan to host an R&D day to provide an update on our <unk> 501 program.

Pipeline within the product.

And Additionally, the neuroscience pipeline candidate.

We look forward to providing more information in the coming weeks.

Looking beyond our neuroscience program, our phase 1 to be study for castrate resistant prostate cancer with <unk> 501 is nearing completion and a more complete I appreciate the update from the phase II portion of the trial is expected in the third quarter of this year.

Based on these results we plan to design, our next phase of the Seattle to the program and leverage <unk> Serono ones novel mechanism to build our immuno oncology franchise.

As we move into the second half of this year, we look forward for the building on the important progress we have discussed today and continue to drive forward sustainable growth strategies for our business.

Vimal D. Mehta: As we move into the second half of this year, we look forward to further building on the important progress we have discussed today and continuing to drive forward sustainable growth strategies for our business. Now, I would like to turn the call over to Richard, who will give a financial, Thank you, Vimel. I would now like to review our financial results for the second quarter of 2021. Research and development expenses were $13.5 million in the second quarter of 2021, as compared to $17.9 million for the same period in 2020. The decreased expenses were primarily attributable to a reduction in clinical trial costs related to our serenity trials.

Now I would like to turn the call over to Richard who will give you of financial update. Thank you BMO I would now like to review our financial results for the second quarter 2021.

Research and development expenses were $13.5 million during the second quarter of 2021.

As compared to $17.9 million for the same period in 2020.

The decreased expenses were primarily attributable to a reduction in clinical trial costs related to our serenity trials.

Richard I. Steinhart: These amounts are offset in part by an increase in personnel and related costs necessary to enlarge our development and medical team. In addition, we experienced increased professional fees related primarily to toxicology studies, as well as increased regulatory and consulting fees all related to BXL 501. General and administrative expenses were $14.1 million for the second quarter of 2021, as compared to 3.5 million for the same period in 2020. The increase was primarily due to the overall growth of the company, including higher personnel costs and stock-based compensation.

These amounts were offset in part by an increase in personnel and related costs necessary to enlarge our development and medical teams in.

In addition, we experienced increased professional fees related primarily the toxicology studies as well as increased regulatory and consulting fees all related to <unk> 5 of 1.

General and administrative expenses were $14.1 million for the second quarter of 2021 <unk>.

As compared to $3.5 million for the same period in 2020.

The increase was primarily due to the overall growth of the company, including higher personnel costs and stock based compensation.

In addition, we experienced increased costs related to legal professional and insurance fees.

Richard I. Steinhart: In addition, we experienced increased costs related to legal, professional, and insurance costs, and expenses related to the potential commercial launch of BXL 501 in the U.S. The company reported a net loss of $27.6 million for the second quarter of 2021, compared to a net loss of $21.4 million for the same period in 2020. The second quarter of 2021 results include approximately 6.8 million in non-cash stock-based compensation costs compared to non-cash stock-based compensation of 2 million for the same period in 2020. As of June 30th, 2021, cash and cash equivalents totaled approximately $273.1 million.

Net expenses related to the potential commercial launch of <unk> 5 of 1 in the U S.

The company reported a net loss of $27.6 million for the second quarter of 2021 compared to a net loss of $21.4 million for the same period in 2020.

The second quarter of 2021 results include approximately $6.8 million in noncash stock based compensation costs.

Per to noncash stock based compensation of $2 million for the same period in 2020.

As of June 30 of 2021 cash and cash equivalents totaled approximately $273.1 million.

Vimal D. Mehta: Now I'd like to turn the call back to Vimel for any further comments. Thanks, Richard. We would now like to open the call for questions, Operator. Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star one on your telephone keypad. The confirmation total indicates your line is in the question Q. You may press star 2 to remove your question from the queue.

Now I'd like to turn the call back to demo for any further comments.

Richard We would now like to open the call for questions operator.

Thank you we will now be conducting the question and answer session if.

If you would like to ask a question. Please press star 1 on your telephone keypad.

The confirmation of total indicate your line is in the question queue.

You May press star 2 to remove your question from the queue for participants using speaker equipment. It may be necessary to pick up of your handset before pressing the star keys.

Operator: For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star. One moment, please, while we poll for your questions. Our first questions come from the line of Jeff Meacham with Bank of America. Please proceed with your questions. Hi, this is Greg Harrison. I'm on behalf of Jeff.

Please while we poll for your questions.

Our first questions come from the line of Geoff Meacham with Bank of America. Please proceed with your questions.

Hi, This is Greg Harrison on for Geoff Thanks for taking the question.

Gregory Allen Harrison: Thanks for taking our question. So looking to the upcoming readout of 701, what sort of efficacy profile would you expect to see that would give you confidence both in moving the program forward through the clinic as well as in the commercial potential of the assets? Thank you, Greg, for the question. I will turn this question to Wynn. Thanks, Emil. So, as we previously said elsewhere, we're really looking for a 20% response rate, roughly.

So looking for the upcoming readout of 701.

What sort of efficacy profile would you expect to see that would give you confidence both in moving the program for it through the clinic as well as in the commercial potential of the asset.

Thank you Greg for the question I will turn this question to Vince.

Thanks, Paul.

Previously said elsewhere, we're looking really for the 20% response rate roughly that would probably be.

Gregory Allen Harrison: That would probably be very relevant for adenocarcinoma, CRPC, and arguably a slightly lower response rate, so mid-teens may be relevant in any PC. And I say that based on the fact that both of these flavors of castrate-resistant disease are essentially unmet medical needs. Bear in mind that our patient population has failed three lines of systemic therapy.

Very relevant for adenocarcinoma, CRP C and arguably slightly lower response rates from mid teens may be relevant in any PC.

And I would say that lease from the fact that both of these flavors of castrate resistant disease are essentially unmet medical need bear in mind, our patient population has field 3 lines of systemic therapy.

Unknown Executive: That's helpful, thanks. Thank you. Our next questions come from the line of Ram Savaraju with H.T. Wainwright. Please proceed with your question. Hi, thanks very much for taking my questions. Can you give us an update on the availability of what you deem to be qualified sales personnel to enable you to appropriately support the BXL 501 launch? And relative to Vimal's comments earlier on the call regarding the European opportunity and opportunities and other ex-U.S. territories for 501, can you perhaps give us an overview of your partnering strategy, your overall commercial outreach strategy as it pertains to those regions and, in particular, enumerate forests, which territories beyond the U.S. and Europe might potentially be the second most commercial opportunities for 501.

Okay.

That's helpful. Thanks.

Sure.

Thank you our next questions come from the line of.

From a variety of <unk> with H C. Wainwright. Please proceed with your questions.

Alright, thanks, very much for taking my questions.

Can you give us an update on the availability of <unk>.

What you deem to be qualified sales personnel to enable you to appropriately supports the <unk> 5 of 1 launch.

The relative to the <unk> comments earlier on the call of regarding the European opportunity and the opportunities in other ex U S. Territories for 501 can you, perhaps give us an overview of your partnering strategy of overall commercial outreach strategy as it pertains for those regions and in particular enumerate for us.

<unk> territories beyond the U S and the Europe might potentially be the most of the second.

<unk> opportunities for 5 of 1 thanks.

Unknown Executive: Thank you. Thanks, Ram. So I will just take on the first question about, like, you know, the partnering. As we have indicated, we plan to find a partner in Europe for co-development and co-commercialization of BXL 501. And as I mentioned, we are, plans are underway to file an MAA. So things are progressing well on both fronts. With that, I will pass it on to Will to answer your first part of the question. Will? Thank you, then. Good morning, Rahm.

Thanks, Robert So I will just take on the first question about like on the partnering as we have indicated that we plan to find a partner in the.

Europe for co development and co commercialization of <unk> hundred 1.

And as I mentioned that we our plans are underway to filing the MAA.

Things are progressing well on both fronts with that I will pass it onto will to answer your first part of the equation will thank you for them good morning, Robert.

Unknown Executive: Thank you, and good morning, Rahm. Relative to the build-out of Salesforce, as you've indicated, our timing is to hire sales leadership in the third quarter and then plan for hiring the sales force in the fourth quarter, obviously dependent upon the progression of the NDA review and labeling negotiations. I'm happy to report that we recently hired our head of sales, our VP of Sales, who brings with him significant and substantive experience in the hospital setting and clearly a large network of individuals that he has already reached out to.

Relative to the build out of the sales force as we've indicated our timing is to.

Since 2 higher sales leadership in the third quarter, and then plan for hiring of sales force in the fourth quarter, obviously dependent upon the progression of.

For the NDA review and labeling negotiations I'm happy to report that we recently hired our head of sales our VP of sales who brings with him significant in substance of experienced in the hospital setting and clearly a large network of individuals debt that he has already reached out to so we feel from that perspective, we're in a very good position plus we have a.

Unknown Executive: So we feel, from that perspective, we're in a very good position. Plus, we have a solid plan in place with a recruiting firm in order to access top talent, and that's what we'll be initiating after we bring on the sales leadership team to begin that process. So I think we're right on plan, as we have always indicated. Okay, great, and then, just with respect

Solid plan in place with the with the recruiting firm in order to access top talent and that's what we'll be initiating after we bring all of the sales leadership team to begin that process. So I think we're right on plan as we.

I've always indicated.

Okay, Great and then just for with respect to the mouse earlier comments regarding the broadening of the neuroscience pipeline can you maybe elaborate for us what general indications of sub indications within neuroscience, you intend to focus on most and Thats youre going to primarily prioritize neuro psychiatry.

Unknown Executive: Okay, great, and then just with respect to the email's earlier comments regarding the broadening of the neuroscience pipeline, can you maybe elaborate for us what general indications or subindications within neuroscience you intend to focus on most, and if you're going to primarily prioritize neuropsychiatry-related indications versus neurodegeneration, or if you're going to give equal weight to both? So BXL 501, as you know, is a pipeline within a product.

The related indications versus neuro degeneration, or if youre going to give equal weight to both thank you.

So the XL by everyone. As you know is the pipeline within a product. So we continue to focus on clinical execution.

Unknown Executive: So we continue to focus on clinical execution. In terms of our additional pipeline, which we are using our AI platform to identify additional pipeline candidates, our strategy is to focus on stress-related access. So initially, we are focusing on neuropsychiatric disorders. And as we move along, our platform is very much applicable to other disease areas, as you said, neurodegenerative rare diseases. And we believe that will be our next progression. But our next candidate that we plan to discuss at R&D Day will be in the neuropsychetic space. Thank you. Thank you. Our next questions come from the line of Greg Savanovich with Goldman Sachs. Please proceed with your questions.

In terms of our additional pipeline that we are using our AIA platform to identify additional pipeline candidates our strategies to focus on stress related exit. So initially we are focusing of neuropsychiatric disorders and as we move along our platform is very much applicable.

The other Dizzy the area that you said neuro degenerative diseases and we.

We believe that will be our next progression, but our next candidate that we plan to discuss in the R&D day will be in the neuro psychiatric space.

Thank you.

Yes.

Yes.

Thank you for our next question comes from the line of Greg <unk> with Goldman Sachs. Please proceed with your questions.

Hi, Tim This is Dan on for Greg Salon, the rich thanks for taking our question.

Anna: Hi, this is Anna speaking on behalf of Greg Savanovich. Thanks for taking our question. Just one for us, could you please help us frame the 40 microgram data and, you know, what do you expect to see and how should we think about that data in the context of potentially getting a label that's, you know, for at home use? Thank you. And that's a great question.

Just 1 from could you please help us frame the.

For the micro claim data.

And what do you expect the scene, how should we think about that data in the context of potentially getting in the label of that.

For.

At home use the thank you.

And that's of Great question 40, Microgram cohort was initiated 2 additional of Optionality in our dosing regimen. So we already have $30.60, microgram, which was showing good efficacy as well as safety profile.

Unknown Executive: That's a great question. A 40 microgram cohort was initiated to build additional optionality in our dosing regimen. So we already have 30 and 60 micrograms which were showing good efficacy as well as a safety profile, and we wanted to make sure that in dementia, our opportunity lies in the acute to intermittent chronic, and also it lies in ALF nursing and in the home setting. So data from 40 micrograms will inform us what the efficacy and safety intersection is, and we continue to do blinded analysis of the safety at the midpoint of our 40 microgram

And we wanted to make sure that in dementia, our opportunity lies in the acute to intermittent chronic and alter it lies.

And <unk> been noticing in the home setting so data from 40 Mega ground will inform us what is the efficacy and safety in the section and we continue to do blinded analysis of the safety and the.

Mid point of our 40 microgram dosing and this will also inform of what.

Unknown Executive: And this will also inform us what two doses we should take forward in our phase three program that we plan to start in Q for our So this 40 microgram dose as an additional optionality to what we currently have 30 and 60. Great, thanks so much.

To do this we'll take forward in our phase III program that we plan to start in Q4 of 2021.

No.

This 40 microgram dose.

The additional optionality to what we currently have $13.60 microgram.

Great. Thanks, so much.

Thank you. Our next question is come from the line of the Rx in Asia with Guggenheim. Please proceed with your questions.

Yadis Senecia: Thank you. Our next questions come from the line of Yadis Senecia with Guggenheim. Please proceed with your questions. Hey guys, thanks for taking my question. This is Eddie on for Yacht. These two for me.

Hey, guys. Thanks for taking my questions of Eddie on for <unk>. Just 2 for me how are you thinking about enrollment in the dementia study do you think you'll need us the percentage of patients with Alzheimer's or similar type of dementia to match the real world numbers like how you're proposing for the FDA to avoid any subgroup analysis scrutiny and then for will are there any recently approved drugs that are administered in the.

Unknown Executive: How are you thinking about enrollment in the dementia study? Do you think you'll need a specific percentage of patients with Alzheimer's or a similar type of dementia to match the real-world numbers? Like how are you proposing to the FDA to avoid any subgroup analysis scrutiny? And then for Will, are there any recently approved drugs that are administered in a similar setting to 501 that might be a good comparison to track pair dynamics and launch trends next year? Thanks.

The Miller setting for 501 that might be a good comp to track the payer dynamics of the launch trends next year. Thanks.

For our dementia of program, we are very cognizant that what kind of analysis, we will need to present to get the ads and the approval and that's exactly what we are working with FDA that if we have in our Alzheimer's population, which is expected to be the major population.

Unknown Executive: For our dementia program, we are very cognizant of what kind of analysis we will need to present to get ASNDA approval. And that's exactly what we are working with FDA, that if we have in our Alzheimer's population, which is expected to be the largest population with dementia, and that was almost 85% in our tranquility trial, if we have statistical significance, would we get a broader level, or do we need to demonstrate it first? to the subtypes and having alignment with FDA, we will make the choice and decision about what trial or what the trial design should be to achieve those goals.

In dementia and that was almost 85% in our tranquility trial. If we have started to take the significant would we get the.

Broader level or do we need to demonstrate it into the subtypes and having alignment with FDA for you to make the choice in the decision.

What trial or what the trial design should be to achieve those goals.

And then Eddie on the second question relative to any recently approved drugs that may be useful is comps and as we as we've talked about as we indicated on the commercial day presentation. There isn't a specific drug debt that we believe is a true comp for 5 of 1 in 5 of 1 is bringing an innovative new approach to the treatment of agitation.

Unknown Executive: And then Eddie, on the second question relative to any recently approved drugs that may be useful as comps, as we've talked about, as we indicated in the Commercial Day presentation, there isn't a specific drug that we believe is a true comp for 501. I mean, 501 is bringing an innovative new approach to the treatment of agitation in patients where there hasn't been for a while. I think the more relevant way to look at it is by setting, and, you know, in the hospital setting, which is where we will land, obviously, with the first indication. It is the hospital that kind of controls formula access, and, you know, payers usually defer to them in terms of their decisions there.

And patients for their asthma and 1 for a while I think the more relevant way to look at it is by setting.

And when the hospital setting, which is where we will land obviously with the first indication.

It is the hospital of that kind of controls formulary access and payers usually deferred to them in terms of their decisions they're.

Unknown Executive: Hospitals have their own processes, as we indicated, relative to timing and advocacy needed within their institutions. So we're clearly aware of them, and we'll be building a deeper understanding of them among target hospitals. And then, you know, adoption in hospitals just tends to be a little bit slower, right? There's a little more inertia we need to work through. But, as we've indicated, we believe we have a paradigm-changing treatment. and is a valuable new option for hospitals to consider, and the feedback so far has been very favorable. So we're very motivated that, you know, we will be able to drive this steadily forward. In addition, I would just add that our

The hospitals have their own processes as we indicated realm.

Relative to timing and advocacy needed within their institutions. So we're clearly aware of those and we'll be building them a day.

Our understanding of those <unk>.

The target hospitals, and then adoption of hospitals, just tends to be a little bit slower right. There's a little more inertia, we need to work through but as we've indicated we believe we have of a paradigm changing treatments.

And a valuable new option for hospitals consider and the feedback so far has been very favorable. So we're very motivated that we will be able to drive this steadily forward.

In addition, I would just add that our medical science liaison continue to generate additional market insight.

Unknown Executive: In addition, I would just add that our medical science liaison continues to generate additional market insight, and they always bring it back that there is an opportunity in pre-ED settings, like pre-emergency departments, like those are the settings which are in community hospitals, it could be group homes, it could be like, you know, the EMS, and you might know some of the drugs that are used in those settings before patients are brought in. So we continue to evaluate whatever we are learning from the market in shaping our commercial strategy.

And the always.

Bringing it back that there is opportunity in.

3 of these are things like the emergency department like those are the third thing which are in community hospitals. It could be group homes. It could be like the EMS and you might notice some of the drugs of using those hurting before patients have already so we continue to evaluate what we are learning from the market is shaping out.

Our commercial strategy and once we have done complete work, which is expected in the next several months will provide an update.

Unknown Executive: And once we have done the complete work, which is expected in the next several months, we'll provide an update. Appreciate the color. Thank you. Our next questions come from the line of Sumant, called Karnay, with Kanakort. Please proceed with your. Good morning, thanks for taking my question. I have just one.

I appreciate the color thanks, guys.

Yeah.

Thank you. Our next question comes from the line of Sumatra call Kearney with Canaccord. Please proceed with your questions.

Good morning, Thanks for taking my question I have just 1 we've seen the FDA, becoming more unpredictable. The pending NDA is just the head of action needs in that context could you remind us as to how confident you might be about satisfying the pool.

Sumant Satchidanand Kulkarni: We've seen the FDA become

Unknown Executive: more unpredictable with pending NDAs is just ahead of action dates.

Unknown Executive: In that context, could you remind us as to how confident you may be about satisfying pre-approval requirements for your sublingual indication?

Requirements for something Woodson the site inspections.

Unknown Executive: for your sublingual film, such as site inspections, so that you can receive approval in a timely fashion, and when do you expect labeling discussions to begin if they haven't already begun?

So that you can see the approval in the timely fashion and when do you expect the labeling discussions to begin the behind the company.

Unknown Executive: The pre-approval inspection, we continue to do, like, you know, mock exercises using the third party, and our manufacturer is based in Pennsylvania. So we continue to monitor whether FDA will do an on-site visit or, depending on the pandemic situation, will it be virtual. So we are prepared for both situations, Plan A and Plan 2. About your second question, can you please remind me what the second part of the question was?

Yes.

The pre approval inspection, we continue to do like in the mock exercises of using the third party and our manufacturer is based in Pennsylvania. So we continue to monitor that if FDA will do we answer it with debt and depending on the pandemic situation will it be able true. So we are prepared.

For both situations plan and plan B.

About your second question can you just please remind me what was the second part of the question.

Related to the same free.

Unknown Executive: Related to the same pre-approval type discussions, have the labeling discussions started, or when do you expect them to? We expect the labeling discussions to begin.

The approval of the type of discussions have labeling discussion started or when do you expect in the stock.

We expect the labeling discussions to begin.

Unknown Executive: We expect the labeling discussions to begin towards the end of this year. Thank you. Our next questions come from the line of Anita Deschant, with Berenberg, Capital Mark.

Towards the end of the team.

Thank you.

Okay.

Thank you our next questions come from the line of Anita Dushyanth with Bahrenburg capital markets. Please proceed with your questions.

Anita Deschant: Hi, good morning. Thanks for taking my question. This one just based on what we discussed earlier. I was wondering when 501 is, you know, initially introduced in the hospital setting, but eventually when it's expanded for other treatments, that is, when it could be administered at home, could you sort of describe the market size and opportunity for that?

Hi, good morning, Thanks for taking my question.

Based on the label.

In EMEA.

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I was wondering like 1 final 1 for any.

Any sort of the integration from the hospital.

But once.

Thanks for anyone has expanded for advocacy.

Bob.

It could be.

Home.

Could you describe the market as an opportunity for a while.

Thanks for the question I need us so as we've indicated previously our strategy is 1 of landing and expanding.

Unknown Executive: Thanks for the question, Anita. So, you know, as we've indicated previously, our strategy is one of landing and expanding, and so the hospital becomes our primary focus. But, as Zimal indicated, we cast a wide net to understand opportunity that may present itself, ultimately based on the label we received and the enabling of that label to promote in various settings. So, you know, we will continue to look at that, but our base, if you will, out of the gate, will be the hospital setting. Beyond that, as we've indicated, you know, there is significant opportunity for the community.

And so the hospital becomes our primary starts but the jumbo indicated we cast a wide net to understand the opportunity that may present itself ultimately based on the label, we receive and the enabling of that label to promote in various settings. So we will continue to look at that but our base. If you will out of the gate will be the hospital setting.

Beyond that as we've indicated there is significant opportunity.

Unknown Executive: In bipolar disorder patients in the community, they oftentimes experience agitation at home, and that is in many ways treated or treated with benzodiazepines before it rises to the level where it needs to be treated in an emergency department setting. And so we believe over time there is an opportunity to help those patients. And there's a study out of Europe that was conducted by Roberts, which indicated that that opportunity presents itself in terms of their agitation.

Bipolar disorder patients in the community.

Many of them in times experience agitation at.

At home and Jim any of that is in many ways of treated or treated with benzodiazepines before it rises to the level, where it needs to be treated in an emergency department setting and so we believe over time, there is an opportunity to help those patients and there is the study out of Europe debt was conducted by Robert which indicated that that opportunity presents itself in terms of their agitation.

And then relative to the dimension market, which we've indicated that at the very large market.

Unknown Executive: And then, Relative to, you know, the dementia market, which we've indicated, that is a very large market. You know, with currently 6 million Alzheimer's patients in the U.S., that's expected to double over the next 20 years. There's a high rate of agitation in those patients, and they obviously are cared for across treatment settings, not only in nursing homes but assisted living facilities and, obviously, at home. And given the rise in the size of that population, the home setting will become increasingly important, I think.

With the currently 6 million Alzheimer's patients in the U S. That's expected to double over the next 20 years Theres a high rate of agitation in those patients and they obviously are cared for across treatment settings, not only in nursing homes, but assisted living facilities and obviously it home and given the rise in the size of that population of the home setting will become increasingly.

I think important so the whole plan is to enable helping those patients with 501 and thats what the clinical development plan is looking to do in from a marketing perspective, and the commercial perspective, we will build accordingly to capitalize on those opportunities.

Unknown Executive: So, you know, the whole plan is to enable helping those patients with 501, and that's what the clinical development plan is looking to do. And from a marketing perspective and a commercial perspective, we will build accordingly to capitalize on those opportunities.

Okay.

Thanks for all of that was helpful and just 1 more.

Unknown Executive: Thanks, that was helpful, and just one more. Also, how do you sort of plan to allocate your resources between, you know, 501 and potentially delving into that in the other indications and then the early stage, 701 candidates?

Oh for making of how do you sort of plan to allocate Toby for installs between.

Final 1.

From the dark and Bob.

The <unk> bonds.

The army for Paul.

Yeah.

So strategically we have lack of no made a choice to continue to build our neuroscience franchise and <unk> therapeutics will continue to be 1 backfill therapeutics in 5 years down the road to be a leader in neuroscience taken in addition, neuro.

Unknown Executive: So strategically, we have, you know, made a choice to continue to build our neuroscience franchise, and bioexel therapeutics will continue to, we want Bay, and Axel Therapeutics in five years down the road to be a leader in neuropsychiatric and, in addition, neuro-regenerative diseases. So that's the vision of the company we are building. Regarding the 701, now we will have data in the Q3 time frame this year, and that will provide good confidence in our human proof of concept for this mechanism, and that's what exactly we are trying to prove because it's the activator of innate immunity, and we are testing it for the first time with an orally available such an advanced agent in combination with Kittluda.

Rare diseases. So that's what our vision of the company we are building the.

Regarding the 701 now we will have.

The data in Q3 timeframe this year and that will provide a good confidence in our human proof of concept for this mechanism and that's what exactly we are trying to prove because it's the activator of innate immunity and we are testing it for the first time a combination of aura.

Really of a level such as advanced agent in combination with Keytruda. Once we achieve that we will seek strategic options what will be best in the interest of all of our shareholders. We continue to evaluate various options that will make sense for the business.

Unknown Executive: Once we achieve that, we will seek strategic options that will be best in the interest of our shareholders. We continue to evaluate various options that will make sense for the business.

Unknown Executive: Thank you, most of the people.

Awesome.

Thank you as a reminder, if you would like to ask a question. Please press star 1 on your telephone keypad.

Operator: Thank you. As a reminder, if you would like to ask a question, please press star one on your telephone keypad. Our next questions come from the line of Samir Devani with RX securities. Please proceed with your questions. Hi everyone, thanks for taking my question. It's just, if you could just remind me what the status is of the MD Anderson study with 701, that would be great. Vince?

Our next questions come from the line of <unk>.

Amir Giovanni with our ex Securities. Please proceed with your questions.

Hi, everyone. Thanks for taking my question.

Just.

If you could just remind me what the status is of the MD Anderson study with 701.

That'd be great. Thanks.

Unknown Executive: Sure, thanks. As we previously announced, both arms of the study have moved from stage one to stage two. And that study will now complete, or proceed to completion, I should say. And just a reminder, it's a Bayesian style design, so a fairly small sample size, 15 patients, approximately, in each hour. Just as a follow-up, when would you expect us to see any data from that? Yeah, so just bear with us. This is an IST, and obviously, we're not in charge of the enrollment in this study. We would expect to see enrollment probably complete by the turn of the year, approximately. Again, just to stress, we don't have direct control over the trial. But that's certainly how M.D. Anderson has positioned it for us.

Yes.

Sure.

Sure so.

So I think as we previously announced the.

Both arms of the study have moved from stage 1 to stage 2.

And that study of <unk>.

Please proceed to completion I should say and just the remainder it's a Bayesian style design. So a fairly small sample size of 15 approximately patients in each of them.

Just as a follow up when would you expect for after.

The C any data from that study.

Yes, so just bear in mind of this as an ISP and obviously, we're not in charge of the enrollment of the study and we would expect to see enrollment.

Probably complete by turn of the year approximately again just to stress we don't have direct control over the trial, but that certainly had the MD Anderson has them has positioned it to us.

Okay, that's great thanks very much.

Yeah.

Yes.

Unknown Executive: That's great. Thanks very much. Thank you. There are no further questions at this time. I would now like to hand the call back over to Vimometa for any closing questions. Thank you, everyone, for joining us today. I'm looking forward to connecting with many of you at upcoming conferences this fall. In the meantime, if you have any questions, Please feel free to reach out. Have a great day! Thank you. This does conclude today's teleconference. Thank you for your participation. You may disconnect your lines at this time. Have a great day!

Yeah.

Thank you there are no further questions at this time I would now like to hand, the call back over to the demo Mehta for any closing comments.

Thank you everyone for joining us today.

Looking forward the connecting with many of you at upcoming conferences. This fall in the meantime, you have any questions. Please feel free to reach out have a great day.

Thank you. This does conclude today's teleconference. Thank you for your participation.

You may disconnect your lines at this time have a great day.