Good morning and welcome to the BioXcel Therapeutics First Quarter 2021 Financial Results Conference Call. Currently, At this time, all participants are in a listen-only mode.

[Operator Instructions] 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.

Actual results could differ materially from those anticipated in these forward-looking statements.

The risk factors that may affect results are detailed in the company’s most recent public filings with the US Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2021, which can be found on its website at www.bioxceltherapeutics.com or on www.sec.gov.

As a reminder, today’s conference is being recorded. Joining us on today’s call are Dr. Vimal Mehta, Chief Executive Officer; Richard Steinhart, Chief Financial Officer; Will Kane, Chief Commercial Officer; Vince O’Neill, Chief Medical Officer; Frank Yocca, Chief Scientific Officer; and Reina Benabou, Chief Development Officer.

It is now my pleasure to turn the call over to Dr. Mehta, the CEO of BioXcel. Thank you, sir. Please go ahead..

Thank you, operator. Good morning, everyone and thank you for joining our conference call to discuss BioXcel Therapeutics financial results and business highlights for the first quarter of 2021. We appreciate everyone's time and attention.

This quarter, we have remained committed to accomplishing our long-term goals for our neuroscience candidate, BXCL501 with an emphasis on advancing our Dementia Development Program as well as building the commercial readiness infrastructure needed to support the potential launch of our first two indications.

Starting off with dementia, we are very pleased with the tremendous advancement we have made with this indication, from successful data readout to the receipt of breakthrough therapy designation from the FDA.

In first quarter, we announced positive data from the TRANQUILITY study, our Phase 1b/2 trial for the acute treatment of agitation associated with dementia, which included patients with Alzheimer's disease.

To summarize, BXCL501 was well tolerated with statistically significant reductions in agitation achieved at two hours post dose with both 30 microgram and 60 microgram cohort as measured by primary and secondary end point. Also, the supplemental 40 microgram dose cohort study was initiated and is progressing.

These results aim to provide development path to a pivotal program and provides a strong foundation for our broad dementia development strategy. We are highly encouraged by BXCL501’s potential in dementia. We believe it has broad applicability in treating not just acute agitation but the full spectrum of agitation across care setting.

Supported by the TRANQUILITY results, we received FDA breakthrough therapy designation for BXCL501 for the acute treatment of agitation associated with dementia. another win for this indication.

With this designation, highlight the need for alternative treatment options for this underserved patient population and potentially offer an expedited development and regulatory review for BXCL501.

With our end of Phase 2 meeting with the FDA in the near future, we'll look forward to discussing our registrational development strategy for dementia, solidifying the trial design as well as initiating the late program in the second half of this year.

Approximately 6 million individuals in the US are living with dementia and of that 70% experience agitation episodes. This market is a huge opportunity that is largely untapped. Following decades of research, there are still no safe and effective treatments that directly target agitation commonly seen with dementia patients including as most patients.

We are thrilled by the potential to be the first company to develop a therapy designed to address this significant patient and caregiver need.

In parallel we are making great strides with commercial readiness initiative in hopes of bringing the first treatment to specifically address schizophrenia and bipolar disorder related agitation to the US 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 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. 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.

If approved, we are confident we would be ready for a commercial launch of BXCL501.

Importantly, the infrastructure we are building to support the potential commercialization of BXCL501 for acute treatment of agitation associated with schizophrenia and bipolar disorders 1 and 2 will be crucial foundation to launching additional potential follow-on indication, paving the way for our neuroscience business.

Recently, in tandem with Mental Healthcare Awareness Month, we launched an education campaign on agitation in neuropsychiatric diseases such as schizophrenia and bipolar disorder called Boiling Point.

This campaign intends to shed light on the importance of prompt and cooperative de-escalation in the acute treatment of agitation, our interactive website at Partners in calm.com aimed to educate healthcare providers.

Importantly, our strategy, strategy for commercializing BXCL501 for the treatment of schizophrenia bipolar disorder includes agitation associated with dementia as well as other possible indication.

We have already benefited broadly from the market research, we had an advisory board meeting, we have had with a deeper understanding of the agitation treatment landscape. We will continue identifying key learning and insight as we roll our commercial plan.

For schizophrenia and bipolar disorder, that will have laid the foundation for the much larger dementia opportunity. With the preparations for US 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 year.

We are committed to delivering transformative neuroscience medicines to the global population through regulatory expansion opportunities and potential strategic partnerships to help commercialize BXCL501 in Europe. Adding to our geographic expansion strategy, we recently strengthened our BXCL501 IP portfolio with the issuance of two Japanese patent.

We first covered method of creating agitation which will expire no earlier than 2037. And the second is directed to film design, which will expire no earlier than 2041. All these achievements serve as foundational building blocks for expanding our global footprint.

While we continue to prioritize our dementia and schizophrenia bipolar programs, we believe that BXCL501’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 1b/2 trial of BXCL501 for the treatment of opioid withdrawal symptoms.

This trial mark our first time testing BXCL501 as a sub-chronic treatment twice daily dosing over one week.

We were encouraged that we met the primary safety endpoints and were able to identify a dose range that were generally well tolerated and resulted in numerical improvement in retention in the difficult-to-treat Fentanyl and this patient population.

According to the latest estimates from the CDC, synthetic opioids 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.

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 step for this important condition.

The RELEASE data also supports further investigation of different dosing regimens across additional indications and treatment settings. As you can see, we have laid a robust clinical foundation for our BXCL501 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 effect for alleviating acute agitation in three distinct indications; schizophrenia, bipolar disorders and dementia.

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 BioXcel we are passionate about the potential to bring novel treatments in neuroscience and immune-oncology 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 immune-oncology clinical candidate BXCL701.

It is an oral immunomodulator designed to simulate both innate and adaptive immune systems potentially turning cold tumors to hot. BXCL701 is designed to be a potent inhibitor of DPP 8/9 that may activate inflammasomes resulting in the release of pro-inflammatory cytokines in the tumor microenvironment.

As you know, despite checkpoint inhibitor there will be undoubted success, many patients do not respond to them particularly cold tumors such as aggressive form of prostate cancer. We are excited about being BXCL501 novel mechanism of action to combine with a checkpoint inhibitor and courage by our IO program to date.

We believe BXCL701 is the most advanced orally available innate immunity activator in development that has been optimized for dosing in combination with KEYTRUDA. BXCL701 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 open label Phase 1b/2 in aggressive forms of prostate cancer we are pleased that the adenocarcinoma cohort has met its efficacy bar to move to Stage 2. The trial will now continue to full enrollment and updated efficacy data are expected to be reported this year.

This is a giant step for BXCL701 as this candidate is showing early promise problem in cold tumors by potentially making the cancer more recognizable to checkpoint inhibitor. If successful this innate immune inhibitor could be transformative to the immune-oncology field.

Finally, 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 Richard?.

Thank you, Vimal. 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. In addition, we experienced increased professional fees in conjunction with higher consulting fees and CMC costs related to BXCL501 as well as increased costs related to our, to our released clinical trial.

These increases were offset in part by a decrease in SERENITY I and II 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 BXCL501 in the US, an increased legal, professional fees and insurance costs.

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 31, again our cash and cash equivalents totaled approximately $194 million.

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

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

Operator?.

Thank you. The floor is now open for questions. [Operator Instructions] Our first question today is coming from Geoff Meacham of 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 in dementia, what are your preferred characteristics you'd target in your end of Phase 2 meeting with the FDA? Just wondering if you'd be able to potentially run a smaller trial, given the existing safety track record of BXCL501 and get the indication added to the label center?.

Greg thanks for the question. This is Vimal. We are having a planning and 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 base needed.

So we will get that clarity on all of that soon once we have a meeting with the FDA and as you know in tranquility, we use ALF setting and we are trying to see that what we want to do in Phase 3 is close to what we have done in TRANQUILITY..

Got it. Very helpful. Thanks..

Thank you. Our next question is coming from Graig Suvannavejh of Goldman Sachs. Please go ahead..

Hi team. Thanks for taking the question. This is Suzana on for Graig Suvannavejh. Just one quick question from us, could you provide a little bit more color on the placidity enrollment and the burden of COVID-19? Just a little color would be helpful. Thanks..

We initiated that site sin Q1 and after opening couple of sites we realized that there were operational difficulties in enrolling the patients and opening additional sites. So we decided to voluntarily form enrollment until we assess what is the optimal step to take to get initiated the drug..

Great. Thanks. And do you expect that the timelines would be slightly delayed in terms of getting first signs or for significant data..

Once we have assessed what the operational challenges are and how we are going to overcome then we will be able to provide guidance on the timeline?.

Great. Thanks so much..

Thank you. Our next question is coming from Brian Mills of Jefferies. Please go ahead..

Hi. Thanks for taking my questions. So I was wondering if you can talk a little bit about the start and commercial launch preparations. And have you encountered any logistical challenges that of COVID. And when do you expect to hold meetings with P&T Committees. Thanks..

Sure. Thanks Brian. This is Will Kane And I’ll address your questions. 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 a positive signal relative to the receptivity to BXCL501. They also clearly recognize the unmet need, particularly in the patients are treating in the hospital setting. And as I said the profile does motivate them.

We don't anticipate at the current time 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 launch in Q1 of 2022.

And so, we will plan for that, continue to assess the opportunity to access hospitals 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 spokespeople if you will in front of the P&T committees to initiate the process and ultimately achieve formulary access for the product..

Great. That’s helpful. Thank you..

Thank you. Our next question is coming from Robyn Karnauskas of Truist Securities. Please go ahead..

Hi, guys. Good morning. Thanks for taking the question. I guess one for Will. You said you talked about the structure for the sales force and for marketing of the drug.

Do you have any updated thoughts on either feedback from you've mentioned payers, and what kind of feedback are you getting from the payers? And any updated thoughts on the structure of your sales force? And then as a follow-up, can you just give a update on enrollments of the PTSD and delirium trials? Thank you. trial? Thank you..

Sure, hi, Robyn thanks for the questions. As we start, 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’ve recognized the unmet need as I stated previously.

And hospitals are more price-sensitive segment of the market; and so we're looking to understand what level of discounting if you will off a 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 a 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, it’s certainly on the pricing front as we get closer, which we will continue to do.

And as we engage more and more on P&T committee members directly through both the medical managed care team and then ultimately when we feel that account management team in the second half of this year which is on schedule.

In terms of the structure 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, we have already identified high potential hospitals that would constitute the core.

That's about as we indicated previously that's about 1,500 or so. And so the next step is to continue to profile them to understand things like a 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 update on the PTSD as you know that is a trial we are conducting with Gale and VA if they [ph] IST.

So they are working towards the goal of initiating the trial as we get update that they have initiated we will be in a position to communicate for the delirium which I mentioned the PLACIDITY, we had some operational challenges in ICUs in the current environment to enroll patients as well as to be able to open additional sites.

So, we have voluntarily forced delirium and we continue to be focus on our three priority indications; schizophrenia and bipolar and dementia..

Thank you. Our next question is coming from Ram Selvaraju 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 BXCL501, 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? Thanks..

Ram, thanks for the question. 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 an 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 US 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 the regulatory approval, and we'll continue to explode 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, for our other upcoming indications including dementia..

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 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?.

ICU, post-operative setting, medical wards and ER. So, we have a choice to make that in any of those settings. Trial can be conducted. We chose ICU for very fundamental reason. There was a data available with IV decks about a proof-of-concept that when that was used, all patients responded who were refracted to Haloperidol.

So, that was the rationale to choose the ICU. But conceding the operational difficulties, medical wards can be another very viable option..

Thank you..

Thank you. Our next question is coming from Yatin Suneja of Guggenheim Partners. Please go ahead..

Good morning, guys. Thank you for taking my questions.

With regard to the potential dementia related agitation study, is there a thought of going after dementia or could you narrow it to a particular dementia such as like, like let's say Alzheimer, just wanted to get a sense the learning that we might be applying from what happened at Acadia?.

Yatin, thanks for the questions. In our TRANQUILITY trial, our majority of the patients were Alzheimer's patient. If you look at it, they were about 85% or more. And then second largest was vascular dementia. When we looked at the data, we did not see in our sub-group 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 Lewy body. So, we are already narrow in terms of our dementia sub-population.

And what you mentioned, what learnings can we have, we learn from that and then we will have a conversation with the FDA, what is the most optimal path to get to the sNDA, is the dementia with Alzheimer's as a major subtype and some of the other dementia which I mentioned or it’s only Alzheimer's, those things needs to be discussed with our team..

Got it, and then just one question on BXCL701. Could you maybe talk about what you need to see in Stage II for you to move into, let's say, pivotal development. And how much data are we going to be expecting later this year? Thank you..

Wills, do you want to take that question?.

Sure. I'll take that. Thanks, Vimal. So as you may recall, the study is divided into Stage 1, Stage 2. So 15 patients in Stage 1, and then a further 13 patients in Stage 2. So 28 patients in total. We previously said that we’re aiming to see an objective response rate at around -- competent response rate, sorry, around 20%.

And given that this is an end-stage patient population, we think that's compelling. So, where we to see that then the next step would be actually to expand out the 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..

Thank you..

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

Hey guys. This is Rich calling in for Colin. Couple of questions from us. We only saw the M.D. Anderson basket study presentation at the upcoming ASCO, and I believe you guys mentioned that you would present the prostate cancer in mid-year. But it seems to be delayed now.

So just any reason for that? And also a follow-up question on you mentioned the cash position after one 1Q $94 million. When do you expect to do another raise to support commercialization of BXCL501? Thank you..

You’re right that regarding ASCO, our – like investigator at MD Anderson is going to present the data, that is related to the heart Is going to present the data that is related to the hot tumors that either refractory or treatment naïve patients. And we have indicated that we will plan to announce the data mid-year sometimes with the prostate.

Where we are with the trial where we have moved to the Stage 2. We feel good and confident that trial enrollment is progressing well. These are open label trial.

We want to accumulate sufficient number of patients and see the responses over a period of time and we believe that there's no material change in our guidance when we will be ready to share the data on the BXCL701 trial.

Regarding your question do you have any question or does it address your BXCL701 question?.

Yeah, that answers it. Thank you..

Okay. And as we indicated cash position is $194 million, 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 we are in a good position as far as our cash is concerned..

Thank you. Our next question is coming from Sumant 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 [indiscernible] NDA this month and the rights of the 60-day mark since your press release.

So, could you characterize any new interactions with the FDA that you might have specifically had in this NDA after submission? And the second question is what are the latest levels you have for non-diluted finance funding? what are the latest levels you have for non-dilutive finance funding.

And do you have any preference for geography, EU versus Japan or perhaps royalty deals in the US..

So Sumant you are right that we have completed the 60-day cycle with the FDA. The questions FDA has asked us prior to the 60-day, all of those questions were addressed. We expect to hear from the FDA under now 74th day when we will potentially learn about the acceptance as well as about the PDUFA dates.

Your second question is about the non-dilutive financing. Considering we are filing our MAA with EMA, so Europe will be the first priority. As you notice that 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 the next priority to the Europe.

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 US..

Thank you. Our next question is coming from Samir Devani of Rx Securities. Please go ahead..

Yeah hi. Thanks for taking my questions. I just want to confirm, you mentioned 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. Thanks..

That's – Samir thanks for asking the question. That was our question when we had 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 MAA application..

Okay. That’s great. And then just one follow-up just on the PLACIDITY and delirium opportunity, I'm just wondering if you 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 question Samir. As you know IV Dex is only available in a surgical suite currently. And also, it involves some sort of titration and other difficulties that are associated in administering IV Dex to treat the agitation.

So, what we have learned is there are settings like medical ward and other settings, where they could be opportunity for a BXCL501.

So that's part of the reason we feel that it is across a 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 a IV line and do their titration with IV Dex.

So our research indicates that delirium opportunity in medical wards and some of the other places in the institution can be very relevant for the film..

Thank you. Our next question is coming from Anita Dushyanth of Berenberg Capital Markets. Please go ahead..

Hi, good morning thanks for taking my questions, just a couple here.

Can you talk about the enrollment rate in the TRANQUILITY expansion study? And also, study for [indiscernible] will you able to mention what the size of the sales force is for the commercialization of the BXCL501?.

So, in terms of the enrollment 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 data in our hand to make decisions for our Phase 3 program. Regarding the sales force, Will has mentioned that our range is 75,200.

But more or less the sizing work that is being done for priority hospitals about 1,500, it may be more or like 75..

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..

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..

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