Q1 2021 BioXcel Therapeutics Inc Earnings Call

Vimal D. Mehta: Back in March, we announced that we had completed the submission of our NDA application to the FDA, supported by positive data from the pivotal serenity studies reported last July. This is our first NDA for our neuroscience program, and we expect a response from the FDA this month regarding the submission. On the commercialization front, we are continuing to advance our preparations for commercial readiness. These initiatives have included making key hires for both commercial and medical teams.

Vimal D. Mehta: In March, we deployed our Medical Science Liaison and Medical Managed Care Team. Additionally, we are completing the design of our Salesforce size and structure. If approved, we are confident we would be ready for a commercial launch of BXL 50.

Vimal D. Mehta: Importantly, the infrastructure we are building to support the potential commercialization of BXL 501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders. One and two will be crucial foundations to launching additional potential follow-on in the case, paving the way for our neuroscience business. Recently, in tandem with Mental Health Awareness Month, we launched an education campaign on agitation in neuropsychiatric diseases such as schizophrenia and bipolar disorder, called This campaign intends to shed light on the importance of prompt and cooperative de-escalation in the acute treatment of agitation. Our interactive website, PartnersIncom.com, aims to educate health care providers.

Vimal D. Mehta: Importantly, our strategy for commercializing 501 for the treatment of schizophrenia and bipolar disorder, this order includes agitation associated with dementia as well as other possible uses. We have already benefited broadly from the market research, partnership, and advisory board meeting. We have held with a deeper understanding of the agitation treatment market, and we will continue identifying key learnings and insights as we roll out our commercial plan for schizophrenia and bipolar disorder that will help lay the foundation for the much larger dementia opportunities.

Good morning, and welcome to the bio ex cell Therapeutics first quarter 2021 financial results Conference call. At this time, all participants are in a listen only mode.

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 he would like to register a question you May Press Star one on your telephone keypad.

Just to remind everyone certain matters discussed in today's conference call and our answers that maybe given to questions asked are forward looking statements that are subject to risks and uncertainties related to future events and our future financial performance of the company.

Vimal D. Mehta: With the preparations for U.S. commercialization on track, we are excited to announce that our strategic plans for geographic market expansion in Europe are also underway. Utilizing our NDA package, we expect to file a marketing authorization application with the European Medicines Agency for the treatment of acute agitation associated with schizophrenia and bipolar disorders 1 and 2 in the second half of this. We are committed to delivering transformative neuroscience medicines to the global population through regulatory expansion opportunities and potential strategic partnerships to help commercialize 501 in Europe.

Actual results could differ materially from those anticipated in these forward looking statements. The risk factors that may affect results are detailed in the Companys. Most recent public filings with the U S. Ex U S Securities and Exchange Commission, Inc.

Clothing, its quarterly report on form 10-Q for the quarterly period ended March 31st 2021, which can be found on its website at www dot bio ex cell therapeutics dot com or www Dot S. E C thought G O V.

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

Joining us on today's call are Doctor thermal Mehta, Chief Executive Officer, Richard Steinhart, Chief Financial Officer, well Kane, Chief Commercial Officer, Vince O'neill, Chief Medical Officer.

Vimal D. Mehta: Adding to our geographic expansion strategy, we recently strengthened our BXL 501 IP portfolio with the issuance of two Japanese patents. The first covered a method of treating agitation, which will expire no earlier than 2037, and the second is directed to film design, which will expire no earlier than 2014.

Frank Yucca, Chief Scientific officer, and rain up in Abu <unk> Chief Development Officer.

It is now my pleasure to turn the call over to Dr. <unk>. The CEO of <unk>. Thank you Sir Please go ahead.

Thank you operator, good morning, everyone and thank you for joining our conference call to discuss <unk>.

Vimal D. Mehta: All these achievements serve as foundational building blocks for expanding our global, While we continue to prioritize our dementia and schizophrenia bipolar programs, we believe that 501's differentiated mechanism of action has the potential to treat a wide spectrum of agitation across neuropsychiatric and neurological disorders. We are confident this candidate has the potential to be used across an array of treatment settings where there are large unmet needs. Recently, we reported data from the release study, our Phase 1B2 trial of BXL 501 for the treatment of opiate withdrawal. This trial marked our first time testing 501 as a subchronic treatment, twice daily dosing over 1.

<unk> financial results and business highlights for the first quarter of 2000 and joined the ones we have.

I appreciate everyone's time and attention.

This quarter, we have remain committed to accomplishing our long term goal for our neuroscience candidate.

Well one we then ex let's say on advancing our diminishing our development program as well as the commercial infrastructure needed to support the potential launch of our cluster indication.

Starting off with dementia, we are very pleased with the tremendous advancement. We have made with this indication from successful data readout could they received a breakthrough therapy designation from the FDA.

In first quarter, we announced positive data from the Tranquility study our phase one b trial for the acute treatment of agitation associated with dementia, which included patients with Alzheimer's disease.

Vimal D. Mehta: We were encouraged that we met the primary safety endpoint and were able to identify a dose range that was generally well tolerated and resulted in numerical improvements in retention in this difficult-to-treat fentanyl-enriched patient population. According to the latest estimates from the CDC, synthetic opioid fatalities rose by an unprecedented 55% during the 12 months ending in September 2020. The growing use of fentanyl is becoming a significant national health crisis.

To summarize five one was well tolerated with that is particularly significant reductions in agitation achieved at two hours post dose been born alrighty, and 60 microgram cohort as measured by primary and secondary endpoint.

Also the supplement on 40 microgram dose cohort starting about the initiative.

And is progressing Robert.

David is that is aimed to provide development that pivotal program and provides a strong foundation for our broad diminishing our development strategy we.

We are highly encouraged by favorable ones potential and dementia. We believe it has broad applicability to not just take your vegetation what day.

Vimal D. Mehta: Based on these statistics, we believe there is an important need for new treatment options to help address this mounting crisis. We will continue to analyze these results in collaboration with our highly distinguished advisors regarding potential next steps for this important condition. The release data also supports further investigation of different dosing regimens across additional indications and treatments. As you can see, we have laid a robust clinical foundation for our BXL 501 program, conducting a total of seven clinical trials in over 800 subjects across a range of diseases in just over 2.5 years. We have now demonstrated the potential for robust treatment effects for elevating acute agitation in three distinct indications, schizophrenia, bipolar disorder, and dementia.

Reported a spectrum of graduation.

Escapes that day.

Supported by day Tranquility results, we received FDA breakthrough therapy designation for <unk>, one for the acute treatment of agitation associated with dementia.

Another win for this indication.

This designation highlights the need for alternative treatment options.

For this.

Asian population and potentially offers an expedited development and regular day, they do view for quite a while.

With our end of phase II meeting with the FDA in the near future, we'll look forward to discussing our registrational.

Charlie's many important dementia solidifying the trial design as Robert initiating the late stage program in the second half of this year.

Approximately 6 million individuals in the U S are living with dementia and of that seven people or the same expedia as agitation episode.

Vimal D. Mehta: We have made significant progress on our strategic initiatives in building an integrated organization encompassing artificial intelligence, research, and development, medical, and commercial capabilities to deliver innovative medicines to patients. At BioXel, we are passionate about the potential to bring novel treatments in neuroscience and immunoncology to the millions of patients who lack effective and tolerable options while also creating value for our shareholders. Now, I would like to turn the conversation to our Immunology Clinical Candidate, BXL7.

This market it is a huge opportunity that is largely untapped.

Following decades somebody says that's payable no safe and effective treatment that directly target.

Channel, commonly seen with dementia Sharon Inc.

How does that motivation.

So dan by the potential to be the first company to develop it that'd be designed to address the significant base strength than you have given me.

In parallel we are making great strides with commercial readiness initiative in hopes of bringing the Steve Madden to specifically address in schizophrenia and bipolar disorder.

Vimal D. Mehta: It is an oral immunomodulator designed to stimulate both innate and adaptive immune systems, potentially turning cold tumors into warm ones. 7. It is designed to be a potent inhibitor of DPP 8 and 9 that may activate inflammasomes, resulting in the release of pro-inframatry cytokines in the tumor microintrae. As you know, despite Checkpoint Inimator Therapy's undoubted success, many patients do not respond to them, particularly cold tumors such as aggressive forms of prostitute. We are excited about BXL 501's novel mechanism of action to combine with a checkpoint inhibitor and are encouraged by our IEO program today.

Education to the U S market.

Back in March we announced that we have completed the submission of our NDA application to the FDA supported by positive data from the phase <unk> Sydney.

They didn't need these studies reported last July.

This is our first NDA for our neuroscience program and we expect a response from the FDA. This month regarding the submission.

On the commercialization front, we are continuing to advance our preparation for commercial readiness.

These initiatives have included making key hires for both commercial and medical day in March we deployed our medical science liaison and medical managed care team. Additionally, we are completing the design of our sales force size and structure.

Vimal D. Mehta: We believe 701 is the most advanced orally available innate immunity activator in development that has been optimized for dosing in combination with kids. 7-1 is currently being evaluated in two combination trials with Kedra for the treatment of several advanced cancer types, both cold and hot. In our ongoing open-label phase 1B slash 2 trial in aggressive forms of prostate cancer, we are pleased that the Adinocarsimuma cohort has met its efficacy bar to move to stage. The trial will now continue to full enrollment, and updated efficacy data are expected to be reported this year.

If approved we are confident we would be ready for a commercial launch I'll be ex U S fiber one.

Importantly, the infrastructure we have.

To support the potential commercialization of EXL payable one for acute agitation.

<unk> associated with schizophrenia, and bipolar disorder, one in total will be crucial foundation total.

Launching additional potential follow on indication paving the way for our neuroscience business.

The assembly in tandem with mental health awareness month, we launched an education campaign on agitation in neuropsychiatric diseases, such as schizophrenia and bipolar disorder.

Card boiling point.

Vimal D. Mehta: This is a giant step for 701, as this candidate is showing early promise in cold tumors by potentially making the cancer more recognizable to checkpoint inhibitors. If successful, this innate immune inhibitor could be transformative in immunoncology.

This campaign intend to shed light on the importance of prompt and cooperative deescalation in the acute treatment of HAE.

The addition.

Our interactive website at partners, Inc. Com dotcom aims to educate healthcare providers.

Importantly, our strategy strategy for commercializing five one for the treatment of schizophrenia bipolar disorder includes agitation associated with dementia as well as other possible indications.

Vimal D. Mehta: Finally, the company has a strong cash position of $194 million to achieve our key milestones. With that, I would like to turn the call over to our CFO, Richard Steinhart.

We have already benefited broadly from the market research.

Richard I. Steinhart: Thank you, Vimel. We reported a net loss of $26.4 million for the first quarter of 2021, compared to a net loss of $14.9 million for the same period in 2020. Research and Development expenses were $14.7 million during the first quarter of 2021, as compared to $12.4 million for the same period in 2020. Higher expenses were primarily attributable to an increase in personnel and related costs necessary to enlarge our development and medical teams.

Advisory Board meeting you Havent heard it would be there do you put on this or any of the agitation diekman landscape.

We really continue identifying key learning and insight as we roll our commercial plan price schizophrenia and bipolar disorder.

We'll have laid the foundation for the much larger dementia opportunity.

Yeah.

With the preparations for U S. Commercialization on track we are excited to announce that on what its strategic plans for geographic and market expansion in Europe are also underway.

Richard I. Steinhart: In addition, we experienced increased professional fees in conjunction with higher consulting fees and CMC costs related to BXL 501, as well as increased costs related to our release clinical trials. However, these increases were offset in part by a decrease in serenity 1 and 2 clinical trial costs. General and Administrative expenses were $11.6 million for the first quarter of 2021, as compared to 2.6 million for the same period in 2020. The increase was primarily due to higher personnel-related costs, as well as costs related to our expansion in preparation of the potential launch of BXL 501 in the U.S. and increased legal, professional fees, and insurance costs.

Utilizing our NDA package, we expect to file a marketing authorization application with the European medicines agency for the treatment of acute agitation.

Associated with schizophrenia, and bipolar disorder I was wondering do Inc.

The second half of this year.

We are committed to delivering transformative neuroscience medicine to the global population through regulatory expansion opportunities and potential strategic partnerships to commercialize fiber one in Europe.

Adding to our geographic expansion strategy, we recently strengthened our <unk> firewall, one IP portfolio with the issuance of two Japanese patent.

<unk> got one method of treating agitation, which will expire no earlier than 2037, and then second is directed to frame design, which will expire no earlier than 2014 zone.

Richard I. Steinhart: The first quarter of 2021 results include approximately $5.6 million in non-cash stock-based compensation costs, compared to non-cash stock-based compensation costs of $776,000 for the same period in 2020. As of March 31st, again, our cash and cash equivalents totaled approximately $194 million.

All of these achievements serve as foundational building block for expanding our global footprint.

Okay.

While we continue to prioritize our dementia schizophrenia bipolar program, we believe that fiber one's differentiated mechanism of action has the potential to treat a wide spectrum of agitation across numerous like I'd taken neurological disorders.

Vimal D. Mehta: Now we'd like to turn their call back to Vimel for any further comments. Thanks, Richard. We would now like to open the call for questions, Operator.

Operator: Thank you. The floor is now open to questions. If you would like to ask a question, please press Star 1 on your telephone keypad at this time. A confirmation tone will indicate your line is in the question queue. If you would like to remove your question from the queue, please press Star 2. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.

One for Dan. This candidate has the potential to be used across an array of treatments or day.

Yes.

Large unmet needs.

Recently, we reported data from the release study our phase I wouldn't be too Brian I'll be ex L 501 for the treatment of opioid withdrawal symptoms.

Operator: Once again, that is star one to register a question at this time. We do ask that you please limit yourself to one question and one to two follow-up questions. Our first question today is coming from Jeff Meacham of Bank of America. Please go ahead.

This marked our first time testing 501, as a sub chronic treatment.

Daily dosing or one rig.

Got aged that we met the primary safety endpoint and we're able to identify a dose range that was generally well tolerated and they've done it in numerical improvement in their attention.

Gregory Allen Harrison: Hey guys, this is Greg Harrison. I'm for Jeff. Thanks for taking the question.

Gregory Allen Harrison: So as you look forward to a pivotal year,

Gregory Allen Harrison: in dementia, what are your preferred characteristics you'd target?

Gregory Allen Harrison: in your end of phase two meeting with the FDA. Just wondering if you'd be able to potentially run a smaller trial given the existing

Got it could be Frank in this patient population.

According to the latest estimates from the C. D C synthetic opioid fatalities rose by an unprecedented 55% during the 12 months ending in September 2020.

Gregory Allen Harrison: given the existing safety track record of 501 and get the indication added to the label center.

Vimal D. Mehta: Greg, thank you for the question. This is Wemel.

The growing user friendliness is becoming a significant national health crisis.

Vimal D. Mehta: We are planning a meeting with the FDA to get alignment on the things you exactly mentioned, the clinical trial design, and what is the size of the trial needed, and what is the size of the safety data. So we will get clarity on all of that soon once we have a meeting with the FDA. And as you know, in Tranquility, we use the ALF setting, and we are trying to see if what we want to do in Phase 3 is close to what we have done in Trunquility.

Based on these statistics, we believe they're important and need for new treatment option.

The mounting crisis.

We will continue to analyze these data in collaboration with our highly distinguish adviser regarding potential next steps for this important condition.

We released data also support for that investigation of different dosing regimen across additional indications and treatment settings.

And you can see we have layered a robust clinical foundation quite a lot of <unk> 501 broken out.

Gregory Allen Harrison: Got it. Very helpful. Thanks.

Operator: Thank you. Our next question is coming from Greg Savonay of Goldman Sachs. Please go ahead.

A total of seven clinical trials in over 800 subjects across a range of diseases in just over two and a half years.

Anna: Hi, team. Thanks for taking a question. This is Anna on behalf of Greg Fonich. Just one quick question from us. Could you provide a little bit more color on the placidity enrollment and, you know, the burden of COVID-19? Just a little color would be helpful.

We have now demonstrated the potential for a robust treatment effect, while elevating acute agitation in three distinct indications schizophrenia bipolar disorder and dementia.

Vimal D. Mehta: He initiated the sites

Vimal D. Mehta: sites in Q1, and after opening a couple of sites, we realized that there were operational difficulties in enrolling the patients and opening additional sites. So we decided to voluntarily pause enrollment until we assess what is the optimal step to take to get initiated.

We have made.

Good progress on our strategic any share gains in Brisbane, and integrated organization encompassing artificial intelligent search and development medical and commercial capabilities to deliver innovative medicines to patients.

At <unk>, we are passionate about the potential to bring novel treatment in neuroscience and immuno oncology to the millions of patients who lack effective and tolerable options, while also creating value for our shareholders.

Vimal D. Mehta: Great things. And do you expect that the timelines would be slightly delayed in terms of getting the first signs or signals of data?

Vimal D. Mehta: Once we have assessed what the operational challenges are and how we're going to overcome them, then we will be able to provide guidance on time.

No.

I would like to turn the conversation to our immuno oncology clinical candidate <unk> 701.

Operator: Thank you. Our next question is coming from Brian Mills of Jeffreys. Please go ahead.

Good day than oral immuno modulator designed to stimulate both innate and adaptive immune systems potentially dumping cold tumors to hot.

Brian Mills: Hi, thanks for taking our questions. So I was wondering if you could talk a little bit about the 501 commercial launch preparations, and have you encountered any logistical challenges due to COVID, and when do you expect to hold meetings with the P&T committees?

So I don't know one is designed to be a potent inhibitor of DPP eight and nine that may activate inflammatory <unk> is there anything in the release of pro inflammatory cytokines in the tumor microenvironment.

Unknown Executive: Sure, thanks, Brian. This is Will Kane, and I'll address your question.

As you know despite checkpoint inhibitor therapy undoubted success, many patients do not respond to them, particularly cold tumors, such as aggressive form of prostate cancer.

Unknown Executive: So our preparation for the launch is ongoing, and we're making steady progress. Everything, you know, is coming together. We've completed a good chunk of market research to help inform us, and the product does receive positive reviews by hospital-based clinicians as well as pharmacy directors. So we consider that a positive signal relative to the receptivity to 501. They also clearly recognize the unmet need, particularly in the patients they are treating in the hospital setting. And as I said, the profile does motivate them.

We are excited about being still viable ones novel mechanism affection, two combined with a checkpoint inhibitor and crazed by Wot Io program to date. We believe 701 is the most advanced orally available innate immunity activator day.

In development that has been optimized for dosing in combination with Keytruda.

Unknown Executive: We don't anticipate at the current time, you know, impacts of COVID simply because we're a ways away from the launch, and that's going to require tracking over the next several months. As you know, our base case plan is a launch in Q1 of 2022. And so we will plan for that, continue to assess the opportunity to access hospitals to, to reach hospitals to, to achieve our goal when we build our sales force.

So I don't know one is currently being evaluated in two combination trials with keytruda for the treatment of several advanced cancer types, both cold and hot tumors.

In our ongoing or one open label phase one b slash two trial and aggressive forms of prostate cancer. We are pleased.

Unknown Executive: The goal is obviously to engage and partner with local advocates in hospitals that are high priority for us so that they can be a spokesperson, if you will, in front of the P&T committees to initiate the process and ultimately achieve formulary access for the product.

That would be I didn't know Garcia from a mark Paul Hart has met its fifth.

Casey bar to move to stage two.

The trial will now continue to full enrollment.

<unk> updated efficacy data are expected to be reported this year.

Brian Mills: Great, that's helpful. Thank you.

This is a giant step for 701.

Operator: Thank you. Our next question is coming from Robin Carnacus of Truist Securities. Please go ahead.

This candidate is showing oddly problem is in quality almost by potentially making the candidates have more recognizable to checkpoint inhibitors.

Robyn Kay Shelton Karnauskas: Hi guys, good morning. Thanks for taking the time to ask me a question. I guess one for Will, you know, you talked about the structure of the sales force and for marketing the drugs. Do you have any updated thoughts on either, you know, feedback from, you mentioned payers, like what kind of feedback are you getting from the payers and any updated thoughts on the structure of your sales force? And then, just as a follow-up, can you just give an update on enrollment in the PTSD and delirium trials? Thank you.

If successful this innate immune inhibitor could be transformative to the immuno oncology field.

Finally, the company has a strong cash position of $191 million to achieve our key milestones.

With that I would like to turn the call over to our CFO Richard Steinhart.

Thank you Raimo.

We reported a net loss of $26 $4 million for the first quarter of 2021 compared to a net loss of $14 9 million for the same period in 2020.

Unknown Executive: Sure, hi Robin, thanks for the questions. I'll obviously start.

Research and development expenses were $14 $7 million during the first quarter of 2021.

Unknown Executive: So as it relates to feedback, if you will, from payers. We have, as I said, done some market research with both payers, but more importantly with hospital-based P&T committee members, such as pharmacy directors. As I said, the profile of the drug is very well received. They recognize the unmet need, as I stated previously. Hospitals are a more price-sensitive segment of the market, and so we're looking to understand what, you know, what, you know, what level of discounting, if you will, off the whack price may be necessary to achieve the formulary access that we are shooting for, which is broad-based, right, because the product has applicability across a variety of patients with agitation associated with schizophrenia and bipolar disorder.

As compared to $12 4 million for the same period in 2020.

The higher expenses were primarily attributable to an increase in personnel and related costs.

Necessary to enlarge our development and medical teams.

In addition, we experienced increased professional fees in conjunction with higher consulting fees and CMC costs related to be ex L 501, as well as increased costs related to our to our release clinical trial.

These increases were offset in part by a decrease in serenity one into clinical trial costs.

General and administrative expenses were $11 6 million for the first quarter of 2021.

As compared to $2 6 million for the same period in 'twenty 'twenty.

The increase was primarily due to higher personnel related costs as well as costs related to our expansion in preparation.

Unknown Executive: So what I would say is feedback so far has been positive. We have more work to do, certainly on the pricing front as we get closer, which we will continue to do, and as we engage more and more P&T committee members directly through both the medical managed care team and then ultimately when we field an account management team in the second half of this year, which is on schedule, terms of the structure of the field force, I guess I'm pleased to say that as we continue the analysis to finalize the size and structure, it is pretty much on par with what the initial thinking was, 75 to 100 representatives, probably closer to 75, as we think through the process.

Potential launch of <unk>, five and one in the U S and increased legal and professional fees and insurance costs.

The first quarter of 2021 results include approximately $5 $6 million in <unk>.

Noncash stock based compensation costs compared to non cash stock based compensation cost of $776000 from the same period in 2021 2020.

As of March 31st again, our cash and cash equivalents totaled approximately $194 million.

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

Thanks Richard.

I'd now like to open the call for questions operator.

Thank you the floor is now open for questions.

I'd like to ask a question. Please press star one on your telephone keypad at this time.

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Unknown Executive: We have already identified high-potential hospitals that would constitute the core. That's about, as we indicated previously, that's about 1500 or so. And so the next step is to continue to profile them to understand things like P&T, scheduling processes, who the local advocates are, et cetera, to help us prepare for engagement with them. Robin, this is Vimal regarding the

This happens using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.

Once again that is star one to register a question at this time.

We do ask that you. Please limit yourself to one question and what did you follow up questions.

Our first question today is coming from Geoff Meacham from Bank of America. Please go ahead.

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

So as you look forward to a pivotal trial and dementia.

What are your preferred characteristics you'd target in your end of phase two meeting with the FDA.

Just wondering if you'd be able to potentially run a smaller trial given the existing safety track record of 501 and get the indication added to the label Center.

Vimal D. Mehta: Robin, this is Wemal regarding the update on PTSD. As you know, that is a trial we are conducting with Yale and the VA. It's an IST.

Yeah.

Greg Thanks for the question.

This is at BMO.

Uh huh.

A banding and meeting with the EBITDA to get alignment on the thing you exactly mentioned the clinical trial design and what is the size of the trials needed and what is the size of the safety database.

Vimal D. Mehta: So they are working towards the goal of initiating the trial. As we get updates on what they have initiated, we will be able to be in a position to communicate. For the delirium, which I mentioned, the placidity, we had some operational challenges in the ICU in the current environment to enroll patients, as well as to be able to open additional sites. So we have voluntarily paused delirium, and we continue to be focused on our three priority indications, schizophrenia, bipolar, and dementia.

So we will get the clarity on all of that.

Soon once we have a meeting with FDA.

And as you know in tranquility.

We use yeah lab setting.

And we are.

Trying to see that what we wanted to do and Phd is close to what we have done in tranquility.

Operator: Thank you. Our next question is coming from Ram Svalaju of H.C. Wainwright. Please go

Got it very helpful. Thanks.

Raghuram Selvaraju: Hi, thanks very much for taking my questions. Can you hear me? Yes, we can hear you.

Yeah.

Yeah.

Thank you. Our next question is coming from Gregg Seven day of Goldman Sachs. Please go ahead.

Vimal D. Mehta: Okay, firstly, with respect to the European perspective on 501, can you perhaps elaborate on what you consider to be the most optimal and effective way to commercialize the drug if you anticipate establishing your own commercial infrastructure in Europe, or if you're going to seek a partner? And if you are going to seek a partner, can you give us a sense of what you think is likely to be the optimal timing for that, either ahead of or post-approval? Thank you. So, Ram, thanks for the question.

Hi, Thanks for taking my question. This is Dan on for Greg and just one quick question from US could you provide a little bit more color from the Placidity enrollment and you know that.

The burden of COVID-19.

A little color would be helpful. Thanks.

B K.

In Q1, and after opening a couple of day to be released.

There are operational difficulties in enrolling the patients in opening additional sites. So we decided to voluntarily pause enrollment until b.

What is the optimal after day to initiate that trial.

Vimal D. Mehta: We are very pleased that our recent interactions with the agency in Europe were positive, and they gave us good feedback, and we were encouraged that we can use the NDA package to file in Europe. Having said that, which we are planning to file in the second half of this year, our strategy as a company is to launch this product in the U.S. ourselves and seek a partner in Europe. And with this filing, like, you know, once we get this filing, it definitely creates more interest because now there is a definitive path to regulatory approval.

Oh.

Great. Thanks, and do you expect that the timelines would be slightly delayed in terms of getting first signs are for signals from data.

Once we have that says what the operational challenges that and how are we going to overcome than we will be able to provide a guidance on the time line.

Great. Thanks, so much.

Thank you. Our next question is coming from Brian <unk> of Jefferies. Please go ahead.

Hi, Thanks for taking my questions. So.

I was wonder if you could talk a little bit about federal.

Total one commercial launch preparations and have you encountered any logistical challenges due to COVID-19 and.

Vimal D. Mehta: And we'll continue to explore the opportunity to find the relevant partners. We obviously get approached by some partners and see who has the relevant capabilities, not only for schizophrenia and bipolar disorder, but for our other upcoming indications, including dementia. Okay, and then just a quick follow-up on the delirium cider. Are you envisioning the possibility of conducting a new study somewhat along the lines of placidity but that doesn't involve focusing on COVID-19 patients exhibiting acute delirium?

When do you expect to hold meetings with PMT Committee.

Yeah.

Sure. Thanks, Brian This is will again.

To address your question so our preparation for the launch is ongoing and we're making steady progress.

Everything is coming together, we've completed a good chunk of market research to help inform us and the product has received positive reviews by hospital based clinicians as well as pharmacy directors. So we consider that as a positive signal relative to the receptivity to our 501 they've also.

Clearly recognize the unmet need, particularly in the patients are treating in the hospital setting and as I said the profile does does it does motivate them.

Vimal D. Mehta: Are patients who are exhibiting delirium in the post-surgical setting? And do you think that it would be feasible to conduct such a study without running into the same logistical problems that you previously had?

We don't anticipate at the current time impacts.

Impacts of COVID-19 simply because we're a ways away from the launch and that's going to require tracking over the next several months as you know our <unk>.

Vimal D. Mehta: There are four sets in all.

This case plan is a launch in Q1 of 2022.

Vimal D. Mehta: There are four settings for delirium, ICU, the post-operative setting, medical wards, and ER. So we have a choice to make that in any of those settings, the trial can be conducted. We chose ICU for a very fundamental reason. There was data available with IV Dex about a proof of concept that when Dex was used, all patients who were refractory to Heloparadol responded. So that was the rationale to choose the ICU, but considering the operational difficulties, medical votes could be another very viable option.

And so we will plan for that continued to assess the opportunity to access hospitals too to achieve our goal when we build our sales force.

The goal is to obviously engage and to partner with local advocates and hospitals that are high priority for us so that they can be spokes.

Spokes people, if you will in front of the P&C committees to IND to initiate the process and ultimately achieve.

Achieve formulary access for the product.

Great. That's helpful. Thank you.

Thank you. Our next question is coming from Robyn <unk> of true Securities. Please go ahead.

Operator: Thank you. Our next question is coming from Yatinsenjaya of Guggenheim Partners. Please go ahead.

Hi, guys. Good morning, Thanks for taking the question.

I guess, one for well you said you talked about the structure for.

Yatinsenjaya: Good morning, guys. Thank you for taking my question. With regard to the potential dementia-related agitation study, is there a thought of going after dementia, or could you narrow it to

The sales force from marketing of the drug do you have any updated thoughts on either no feedback from you mentioned payers like what kind of feedback are you getting from the pairs and any updated thoughts on the structure of your sales force and then.

Yatinsenjaya: Could you narrow it to a particular dementia subset like, let's say, Alzheimer's? Just wanted to get a sense of the learning that you might be applying from what happened at Acadia.

As a follow up can you just give a.

Get on enrollments.

Vimal D. Mehta: Yati, yes, thanks for the question. In our tranquility trial, the majority of our patients were Alzheimer's patients. If you look at it, they were about 85% or so, and then the second largest was vascular dimension. When we looked at the data, we did not see, like, in our subgroup analysis, much difference between the two types of dementia population, and the third was FTD. We did not have Parkinson's patients as well as Louisbody.

Of the Pizza Theater Delirium trial. Thank you.

Sure Hi, Robyn thanks for the questions obviously start.

As it relates to feedback if you will from payers.

As I said done some market research with both payers, but more importantly, with hospital based PMT Committee members such as pharmacy directors.

As I said the profile of the drug is very well received they recognize the unmet need as I stated previously hospitals are more price sensitive segment of the market and so we're looking to understand what what level of discounting. If you will off of WAC price may be necessary to achieve the formulary access that we.

Vimal D. Mehta: So we are already narrow in terms of our dementia subpopulation. And what you mentioned, what learning can we have? We can learn from that, and then we will have a conversation with the FDA. What is the optimal path to get to the SNDA? Is it dementia with Alzheimer's as a major subtype and some of the other dementias, which I mentioned, or is it only Alzheimer's; those things need to be discussed with FDA?

We are shooting for which is broad based right because the product has applicability across a variety of.

Spectrum of patients with agitation associated with schizophrenia bipolar disorder. So what I would say is feedback. So far has been positive we have more work to do certainly on the pricing front as we get closer which we will continue to do and as we engage more and more on PMT Committee members directly through both the medical managed care team and then ultimately when we feel that an account management team in this.

Yatinsenjaya: Got it.

unknown: Question on 701: could you maybe talk about what you need to see?

Second half of this year, which is on schedule.

unknown: you need to see in stage two for you to move into, let's say, pivotal development. And then how much data are we going to be expecting later this year?

In terms of the structure of the field force.

I'm pleased to say that as we continue the analysis too to finalize the size and structure. It is pretty much on par with what the initial thinking was 75 to 100 representatives probably closer to 75 as.

Will: Will, do you want to take that question?

Will: Sure, I'll take that. Thanks so much. So, as you may

Will: I may recall the studies divide them into stage one and stage two, so 15 patients in stage one and then a further 13 patients in stage two, so 28 patients in total.

As we as we think through the process, we have already identified high potential.

Hospitals that would that would constitute the core that's about as we indicated previously that's about 500 or so and so the next step is to continue to profile them to understand things like a PNT scheduling processes with a local advocates are et cetera to help us prepare for engagement with them.

Will: I previously said that we're aiming to see an objective response rate around, a composite response rate,

Will: an objective response rate of around, a composite response rate of, I should say, sorry, around 20%. And given that this is an end-stage patient population, we think that's compelling. So were we to see that, then the next step would be actually to expand this study and continue to run the trial essentially to accrue more patients and get a more precise handle on what that response rate is. Hopefully, that answers your question.

Robin. This is reminder, regarding the update on the PTSD as you know that is the trial, we are conducting with Gale and V C.

I S T.

No.

They are working towards the goal of initiating the trial as we get up there that they have initiated we will be in a position to communicate force.

The day, lithium, which I mentioned the plessey duty.

Had some operational challenges in the ICU and the current environment.

Two enrolled patients as well as to be able to open additional sites. So we have voluntarily boss.

Operator: Thank you. Our next question is coming from Colin Bristow of UBS. Please go ahead.

The lithium and we continue to be focused on our three priority indications schizophrenia bipolar and dementia.

Rich: Well, guys, this is Rich calling in for Colin. A couple of questions from us. We only saw the MD Anderson Basket Study presentation at the upcoming ASCO, and I believe you guys mentioned that you would present prostate cancer in mid-year, but it seems to be delayed now.

Thank you. Our next question is coming from Robert supervise you of H C. Wainwright. Please go ahead.

Hi, Thanks, very much for taking my questions can you hear me.

Yes, we can hear you.

Okay, Firstly with respect to the European perspective on fiber one can you perhaps elaborate on what you consider to be the most.

Rich: and also a follow-up question on; you mentioned the cash position after one Q.

Optimal and effective way to commercialize the drug if you anticipate establishing your own commercial infrastructure in Europe, or if youre going to seek a partner and if you are going to seek a partner can you give us a sense of what you think is likely to be the optimal timing for that either ahead of or post approval.

Rich: after 1Q at $194 million. When do you expect to do another race to support commercialization of 501? Thank you.

So.

Thanks for the question we are very pleased there.

Our region.

Interactions with the agency in Europe were positive.

Vimal D. Mehta: You are right that regarding ESCO, our investigator at MD Anderson is going to present the data that is related to the hot tumors like either refractory or treatment eye patients. We have indicated that we will plan to announce the data sometime in mid-year with the prostate. We are with the trial where we have moved to stage two. We feel good and confident that trial enrollment is progressing well.

And they gave us.

Good feedback and we were encouraged that we can use NDA package to file in Europe.

Having said that which we are planning to file in second half of this year our strategy as a company is to launch this product in U S. Our sales and seek a partner in Europe.

And with this filing like no.

Vimal D. Mehta: These are open-level trials. We want to accumulate a sufficient number of patients and see responses over a period of time. And we believe that there will be no material change in our guidance when we are ready to share the data on the 701. Regarding your question, do you have any questions, or does it address your 701 question?

Once we get this filing.

It definitely creates more interest because now there is there definitely additive path to regulatory approval and we will continue to explore the opportunity to find the relevant partners. We obviously get approached by some partners and see who has the relevant capability.

<unk>, not only for schizophrenia, and bipolar, but our other upcoming indications including dementia.

Vimal D. Mehta: Yeah, that answer. Okay, and as we indicated,

Vimal D. Mehta: Okay, and as we indicated, our cash position is $194 million. In our past three quarters, if you look at it, our burn rate has been in the order of about $25 million per quarter. So this cash position gives us a very strong position to achieve the key milestones that we want to achieve that we have highlighted today. So we feel currently that we are in a good position as far as our cash is concerned.

Okay, and then just a quick follow up on the delirium side of things are you envisioning the possibility of conducting a new study somewhat along the lines of placidity, but that doesn't involve focusing on COVID-19 patients exhibiting acute delirium rather.

Insurance, who are exhibiting delirium in the post surgical setting and do you think that it would be feasible to conduct such a study without running into the same logistical problems that you had previously.

Vimal D. Mehta: Thank you. Our next question is coming from Sumant Kilkarni of Kanakord. Please go ahead. Okay.

There are four <unk> four day lithium.

Are you bullish.

Bullets to operative setting medical boards and so.

So we have a choice to make that in any of those settings.

Operator: Thanks for taking my questions. I have a couple.

Sumant Satchidanand Kulkarni: So first, we know you said you expect potential acceptance of your 50-1 NDA this month, and we're right at the 60-day mark since your press release. So could you characterize any new interactions with the FDA that you might have specifically had on this NDA after submission? And the second question is, what are the latest levels you have for non-depletion of funding? And do you have any presence in geography, you versus Japan, or perhaps royalty deals in the U.S.?

Trial can be conducted we chose ICU for very fundamental reason there was a data of a label with the IV Dex.

About the proof of concept.

When <unk> was used.

All patients responded who are refractory to handle it better at all so that was the rationale to choose ICU, but considering the operational difficulties medical what can be very viable option.

Thank you.

Thank you. Our next question is coming from Yadkin from J <unk> of Guggenheim Partners. Please go ahead.

Vimal D. Mehta: So, Suman, you are right that we have completed the 60-day cycle with the FDA. The questions FD asked us prior to the 60 days; all of those questions were addressed. We expect to hear from the FDA on the now 74th day when we will potentially learn about the acceptance as well as about the producer. Your second question is about NANDELU financing, considering we are filing our MAA with EMA.

Good morning, guys. Thank you for taking my question with regard to the potential dementia related agitation study is.

He's there that's sort of going after damage, yeah or could you narrow it to a particular dementia subset like like let's say in Alzheimer's.

Alzheimer.

Just wanted to get a sense I'm you know the learning that you might be applying from what's happened at Acadia.

Vimal D. Mehta: So Europe will be the first priority. As you notice, we got two additional patents issued in Japan. That's a real market opportunity. We will continue to evaluate, and we will schedule that as a next priority for Europe. And sometimes, when you are seeking these partnerships, you do find partners who may be interested in both geographies. So it's very difficult to predict, but I think we are making progress on both fronts outside.

Yes, hey, thanks for the question.

Our tranquility trial, our majority of the patients, whereas zoom reservation.

About 85% or more.

And then second largest was once cooler dementia when we looked at the data we did not see.

Our sub group analysis margin difference between the two types of dementia population.

Operator: Thank you. Our next question is coming from Samir Bhavani of RX Securities. Please go ahead.

And the third was FTE, the we did not have <unk>.

Parkinson's patients as well as lewy body. So we are already narrow in terms of.

Samir Devani: Yeah, hi, thanks for taking

Samir Devani: my questions. I just want to confirm that you mentioned you're using the NDA.

Samir Devani: You're using the NDA package for the European filing, but is there any other data that you may need ahead of that submission? So that's, I guess, the first question.

Dementia sub population.

What you mention what learnings can be had.

Learn from that and then we will have a conversation with the EBITDA. What is the most optimum path to get to this NDA is dementia with Alzheimer's is a major subtype and some of the other dementia, which I mentioned audits only Alzheimer's.

Vimal D. Mehta: That, Samit, thanks for asking the question. That was our question when we had a meeting with the agency, and now we believe we have the alignment that we don't need to generate any additional data than what we already have in our NDA package to submit the MA application.

Things needs to be discussed with FDA.

Samir Devani: Okay, that's great. And then just one follow-up, just on the placidity and delirium opportunity. I'm just wondering if you could maybe just remind us of why doctors would not prefer in that setting to use sort of IV decks rather than the film.

Got it and then just one question on 701 could you maybe talk about what do you need to see in stage two for you to move into pivotal development.

And then how much data are we going to be expecting later this year. Thank you.

Vimal D. Mehta: Thanks very much.

Samir Devani: That's a very relevant question, Samir. As you know, IV-Dex is only available in a surgical suite currently, and it also involves some sort of titration and other difficulties that are associated with administering IV-Dex to treat agitation. So what we have learned is that there are settings like medical wards and other settings where there could be opportunity for a 501. So that's part of the reason we feel that it is across an institutional setting, but there are certain places, like particularly in medical wards and all that. It's a big burden to be able to put an IV line in and do the titration with IV tax.

Bill do you want to take that question.

Sure I'll take that thanks, and thanks for the Moe M.

So as you may recall that the study is divided into stage one stage two so 15 patients in stage, one and then a further 14 patients in stage two so 20 patients in total.

We previously said that we're aiming to see an objective response rate at a road composite response day I should say sorry.

Around 20% and given that this is an end stage patient population, we think that compelling.

So where are we to see that then the next step would be actually to expand up that study.

And continue to run the trial.

Operator: So our research indicates that the delirium opportunity in medical wards and some of the other places in the institution can be very relevant for the film. That's great. Thank you. Our next question is coming from Anita Deschianz of Berrenberg Capital Markets. Please go ahead.

Essentially to accrue more patients and get a more precise handle on what that response is hopefully that answers your questions.

Thank you.

Yeah.

Thank you. Our next question is coming from Colin Bristow of UBS. Please go ahead.

Well, Hey, guys. This is rich calling in for Collyn.

Anita Deschianz: Hi, good morning. Thanks for taking my order.

Couple of questions from us.

We only saw the MD Anderson basket study presentation at the upcoming <unk> and I believe you guys mentioned that you would present the prostate cancer.

Vimal D. Mehta: So in terms of enrollment in the supplemental cohort of 40 micrograms, enrollment is progressing well, and we expect that we will be able to complete that enrollment and have our data in hand to make decisions for our phase three program. Regarding Salesforce, Will mentioned that our range is 75 to 100, but more or less the sizing work that is being done for priority hospitals, about 1,500, it may be more or less like 75%.

But it seems to be delayed now so just any reason for that and also a follow up question on <unk>.

You mentioned the cash position.

<unk> at $194 million.

When do you expect to do another raise to support commercialization of fiber one thank you.

Yeah.

You are right there regarding at school or a lack of new investigators at MD Anderson is going to present the data that is related to the hot tumors.

Either refractory or treatment naive patients.

<unk>.

We.

<unk> indicated that we will plan to announce the day media, sometimes with a broad street, where we are.

With their trial, where we have moved to these changes to be feel good and confident that trial enrollment is progressing well. These are open label trial, we wanted to accumulate sufficient number of patients and see the responses over a period of time and we believe that there is no material change.

Operator: Thank you. We're showing no additional questions in queue at this time. I'd like to turn the floor back over to management for any additional or closing comments.

Our guidance when we will be ready to share that data on the 701 time.

Regarding your question.

Do you have any question or does it address your 701 question.

Yes that answers thank.

Thank you.

Okay, and as we indicated cash position is at or near $94 million.

Our Basel III quarter, if you look at it.

But it has been in the order of about 25 million per quarter. So this cash position gives us a very strong.

<unk> positioned to achieve the key milestones that we wanted to achieve that we have highlighted today. So we feel currently we are in a good position.

As far as our cash is concerned.

Vimal D. Mehta: Thank you, operator. Our number one goal as a company is to deliver transformative medicines to patients in need, and we are confident this can become a reality in the near future. Thank you all for joining our call to me. Gentlemen, thank you for your part. Connect your lines at this.

Thank you. Our next question is coming from two months' Kulkarni of Canaccord. Please go ahead.

Good morning, Thanks for taking my questions I have a couple. So first we know you said you expect potential acceptance of your final one indeed this month and then I think the 60 day Mark since you press release, so could you characterize any new interactions with the FDA that you might've specifically had this indeed after submission and the second question is what are the latest levels you have flow non dilutive funding and do you have any preference.

Operator: Ladies and gentlemen, thank you for your participation. You may disconnect your lines at this time and have a wonderful day. Thee Thank you.

By geography U S, Japan, perhaps royalty deals in the U S.

So two months Youre right that we have completed the 60 day cycle with net debt.

<unk> M. D has asked us prior to the 60 day all of those portions but adverse.

Operator: Thank you. Thank you. Thank you. Thank you.

We expect to hear from the FDA on that now 74 day.

When we will.

Potentially line.

As well as about the Purdue.

Your second question is about nine dilutive financing.

Considering we are filing our.

MAA with EMA, So Europe will be the first priority as you noted that regard two additional patent issued in Japan, that's a real market opportunity, we will continue to evaluate and we will schedule.

The next priority two day auto.

And sometimes when you are seeking these partnerships you do find partners, who may be interested in both geography. So it's very difficult to predict but I think we are making progress on both fronts outside the U S.

Yeah.

Got it thanks.

Thank you. Our next question is coming from Sameer for Bonnie Rx Securities. Please go ahead.

Yeah, Hi, thanks for taking my questions.

I just want to confirm you mentioned youre using the NDA package for the European filing but is there any other day to that.

You may need ahead of that submission.

I guess the first question. Thanks.

Thanks for asking the question that was our question when we had meeting with agency and now we believe we have the alignment that we don't need to generate any additional data than what we already have in our NDA package to submit.

MAA application.

Okay. That's great and then just one follow up just on the Placidity in delirium opportunity I'm just wondering if he can.

Maybe just remind us of.

Why docs would not prefer in that setting to use sort of IV dex rather than the film thanks very much.

That's a very relevant questions from me.

As you know IV Dex is only available in a surgical suite currently and also it didn't wall.

Some sort of titration and other difficulties that are associated and administering IV to extra teeth agitation. So what we have learned is that upsetting.

Like medical Ward and.

Other testing.

There there could be opportunity for a final one so that's part of the reason we feel that it is across institutional setting, but there are certain places like particularly in medical wards and all day and it's a big burden to be able to per day IV line and do their day.

Station with IV day, So our research indicates that the lithium opportunity and medical boards and some of the other places in the institution can be very relevant for the for profit.

That's great. Thanks, Phil.

Yeah.

Thank you. Our next question is coming from Anita to size up Ehrenburg capital markets. Please go ahead.

Hi, good morning, Thanks for taking my questions just pick up from here can you talk about their involvement in that channel could it be expansion study.

Sorry, if I missed this line.

Were you able to measure what the price of the sales force.

Hum.

And I guess from my line.

So in terms of then rollman in the supplemental cohort of 40 microgram enrollment is progressing well.

And we expect that we will be able to complete that enrollment and have our day data in our hands to make decisions for our phase III program.

Regarding the sales force will as mentioned that our range is 75 to 100 day, but more or less the sizing work that is being done for priority hospitals about 1500, it maybe more like 75.

Great. Thank you.

Thank you.

Were showing no additional questions in queue at this time I would like to turn the floor back over to management for any additional or closing comments.

Thank you operator, our number one goal as a company is to deliver transformative medicines to patients in need and we are confident this can become a reality in the near future. Thank you all for joining our call today.

Yeah.

Okay gentlemen, thank you for your participation you may disconnect. Your lines at this time and have a wonderful day.

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Okay.

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