Lee Roth – Senior Vice President, The Ruth Group Vimal Mehta – Founder, Chief Executive Officer, President, Secretary and Director Richard Steinhart – Chief Financial Officer Frank Yocca – Chief Scientific Officer Vincent O'Neill – Senior Vice President and Chief Medical Officer.

Keith Tapper – BMO Capital Markets Sumant Kulkarni – Canaccord Genuity.

Good day, and welcome to the BioXcel Therapeutics Third Quarter 2018 Results Conference Call. Today's conference is being recorded. At this time, I would like to turn the call over to Lee Roth. Please go ahead..

Thanks, Leanne, and good morning, and once again, welcome to the BioXcel Therapeutics third quarter 2018 financial results conference Call.

Just to remind everyone, certain matters discussed on today's call or answers that maybe give to questions asked include forward-looking statements that are subject to risks and uncertainties relating to future events and/or the future financial performance of the company.

Our actual results could differ materially from those anticipated in such forward-looking statements. 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, which can be found on our Web site www.bioxceltherapeutics.com or www.sec.gov.

Please note, the company is under no obligation to update any forward-looking statements discussed today and investors are cautioned not to place undue reliance on these statements.

With me today from management are Vimal Mehta, Chief Executive Officer of BioXcel Therapeutics; Richard Steinhart, Chief Financial Officer; Vince O'Neill, Chief Medical Officer; and Frank Yocca, Chief Scientific Officer. With that said, it's my pleasure to turn the call over to the company's Chief Executive Officer Dr. Vimal Mehta.

Vimal?.

Thanks, Lee. Good morning everyone, and thank you for joining our conference call to discuss BioXcel Therapeutics financial results for the third quarter of 2018.

Over the past several months, we have made significant progress in developing both our clinical assays, BXCL501 and BXCL701, our emerging pipeline candidates establishing clinical partnership and expanding our operational capability. I would like to start by highlighting the progress we have made in our neuroscience program with BXCL501.

BXCL501 is a potential first-in-class drug that directly targets the positive mechanism of agitation and is under development for acute treatment of mild to moderate acute agitations resulting from schizophrenia/bipolar and Alzheimer's disease.

[Indiscernible] formulation of BXCL501 is believed to have a record [ph] onset of action and provide ease of administration in agitated patients. Along with its robust safety and pharmacokinetic profile established through use in millions of patients and in over 130 clinical trials, Dex is the most selective alpha2A adrenergic agonist available.

It produces an arousable sedation and has a calming effect on agitated patients. In the U.S., we believe as many as 5 million patients with Alzheimer's disease, schizophrenia and bipolar disorder experience agitation.

Approximately 1.2 million of these patients experience mild to moderate agitation and represent in potential patient population for treatment with BXCL501.

With [indiscernible] first approved inhale antipsychotic treatment for acute agitation as a reference drug, we believe we have a well-characterized regulatory pathway for BXCL501 via FDAs 505B2 pathway along with potential favorable reimbursement.

We recently had our free investigation new drug IND meeting with the FDA and received valuable guidance on the development of BXCL501. Following the approval of our IND, we plan to initiate a first in human pharmacokinetic bioavailability and safety study of [indiscernible] formulation of that prior to year-end.

The aim of this trial is to define the optimal dose of the [indiscernible] and collect value and data to move it into Phase 2/3 agitation trial. The data from this trial is expected to be available in the first-half of 2019.

We are pleased to report that we have completed manufacturing of our proprietary [indiscernible] formulation of dexmedetomidine and it is available for clinical study. Additionally, we appointed a clinical research organization to support the company in conducting and managing the pharmacokinetic as safety clinical study.

Previously, we announced positive results from our Phase 1b trial evaluating intravenously administered Dex. Finding from this study demonstrated that the selected dose and exposure levels of [indiscernible] had a calming effect in healthy subject without clinically-meaningful changes in blood pressure or heart rate and the drug was well-tolerated.

Through this trial, we were able to develop a dosing strategy for the sublingual thin-film formulation of BXCL501. As an extension of the Phase 1b study, we are evaluating the dose and exposure level in schizophrenia and dementia or the Alzheimer's type healthy 80 patients. We support further development of the BXCL501 in valuing disease pathology.

We expect to report data from these studies of [indiscernible] before the end of this year.

Additionally, in order to support the global clinical development of our neuroscience program, especially BXCL501, we formed a world-class neuroscience Clinical Advisory Board, the founding members of the CAB includes Professor Sheldon Preskorn, Professor Stephen Marder, Dr. George Grossberg and Professor Alan Breier.

We are looking forward to working with the CAB and receiving their valuable guidance on clinical development, regulatory, and medical affairs as well as the commercialization strategy for BXCL501 and other emerging neuroscience program. Let's turn to our second clinical program, BXCL701.

BXCL701 is an orderly available systemic inmate immune activator with dual mechanism of action. It has demonstrated single activity in melanoma with an established safety profile from 700 healthy subjects and cancer patients.

Designed to directly stimulate inmate immune system, BXCL701 was inhibiting dipeptidyl peptidases, DPP 8/9 additionally BXCL701 targets fibroblast activation protein, FAP as an immune inhibitor.

Preclinical combination data evaluating BXCL701 [indiscernible] has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological membrane.

BXCL701 primarily mechanism action has been highlighted in multiple peer-review journals including Nature Medicine, providing an important validation of the scientific rationale behind BXCL701.

We have specifically chosen treatment emergent neuroendocrine prostate cancer, tNEPC, and pancreatic cancer as our lead target indication based on BioXcel, BXCL701 dual mechanism of action and a strong underlying biologic rationale.

Additionally, based on results from preclinical studies the combination of the BXCL701 with I-O agent produces a synergistic anticancer research by elevating the infiltration of tumor-killing cells, NK cells and neutrophils while blocking immunosuppressive cells.

We believe the BXCL701 potential return all tumors into all into combination with higher agent can make tNEPC and pancreatic tumors more responsive offering as they potentially viable therapeutic strategy.

We recently received FDA acceptance of the IND application we submitted for BXCL701 in combination with Pembrolizumab, Keytruda for the treatment of tNEPC.

We Phase 1b two studies expected enroll up to 40 patients at multiple trial site we call this single arm [indiscernible] open label study is to examine the safety pharmacokinetic antitumor activity of the combination of BXCL701 and Pembrolizumab in tNEPC patients with efficacy and point of objective to constant.

We also have completed the [indiscernible] development of for BXCL701 with adequate drug supply to conduct upcoming clinical trials as well as other studies with potential partners. We have also selected a leading clinical research organization to support the complain conducting and managing clinical studies.

We anticipate initiation of this trial by year end and expect to record data throughout 2019.

In addition we expanded our existing immuno oncology research collaboration with Nektar Therapeutics into a clinical collaboration to further explore the potential of our BXCL701 in combination with Nektar NKTR-214, a CD122-biased agonist and a checkpoint inhibitor.

This focus of this new clinical collaboration is on developing the triple combination of BXCL701, Nektar Therapeutics and NKTR-217 and a Checkpoint inhibitor for the treatment of pancreatic cancer.

Most companies which has the cost of clinical trial with trial assessing the triple combination pancreatic cancer is expected to be initiated in our lead 2019.

Today we are presenting promising pre-clinical data along with Nextar on the triple combination of BXCI-701, NKTR-214 and a checkpoint inhibitor across multiple tumor type at the 2018 society for immunotherapy of cancer annual meeting.

Finding from this three clinical studies have demonstrated robust antitumor activity resulting in company tumor regression in multiple tumor model and generation of functional immunological, highlighting the utility of the triple combination has the potential therapy for pancreatic cancer and other indications.

In the third quarter we also established than industry leading immuno-oncology clinical advisory board to support the global development of BXCI-701 and our emerging immuno-oncology program. The inaugural member of the immuno-oncology care was Dr. Louis Venus, Dr. Daniel Von Hoff, Dr. Eric Small and Dr. Emmanuel Antonarakis.

These extreme expert in the field of oncology will guide our team in advancing our I-O program to clinical development to improve patient outcome.

I would also like to point out that we are employing approaches to this clinical development of our lead candidate to patient selection optimization, translation research and predictive biomarker discovery, utilizing our artificial intelligence approaches and platform.

Our exclusive AI partnership with BioXcel Corporation gives us the ability to continue to identify additional indication expansion opportunities for both BXCL501 and 701 in terms of new indications and additional geographies for development.

Our goal is to establish a global footprint by filing IND and clinical trial applications for additional indications in multiple patients. Additionally we are also utilizing our AI platform to prioritize and expand our emerging pipeline to addition our candidate in neuroscience and immuno-oncology.

We are excited about the potential of our lead program and our emerging pipeline for just as much promise. On the operational side we strengthened our management team with the appointment of Dr.

Chethan last year as Senior Vice President, and Head of Translational Medicine, Clinical Pharmacology and regulatory affairs and David Hanley as Vice President and Head of Global Pharmaceutical Development and Operations. Dr.

Lathia will be responsible work as supporting the advancement of companies pipeline programs on the clinical pharmacology and regulatory perspective. While Dr. Hanley will oversee the pharmaceutical development and growth global operation initiative associated with our drug part.

We have thoroughly utilized, we are thoughtfully utilized a portion of the capital from our IPO to advance the clinical development of our lead program. BXCL501 and 701 as well as solidify our leadership team.

We expect cash burn to increase through the remainder of the year and in 2019 build on the increasing number of clinical studies in both our program.

As an organization we believe we have made substantial progress over the last several months and we are now well positioned to achieve our clinical and operational objectives in the coming quarters, BioXcel Therapeutics is committed to bringing transformative therapies to the market and helping patients who have few expanding options.

We try everyday to create significant value for our shareholders. With that, I would like to turn the call over to Richard to discuss our financial results..

Thank you, Vimal and once again thank you for all joining us this morning to welcome to our shareholders. The proceeds from our initial public offering from launch of 2018 gave us the necessary capital to advance the clinical development of BXCL501 and 701 including the initiation of multiple studies needed to program in the coming weeks.

The third quarter 2018 we reported a net loss of approximately $4.9 million compared to a net loss of $900,000 for the third quarter of 2017. Research and development expenses totaled $3.8 million for the third quarter of 2018 compared to approximately $600,000 for the same period in 2017.

The increase in research and development expenses reflects increased development activities in both BXCL501 and BXCL701 programs including increased personnel costs, professional fees, clinical trial costs and manufacturing costs.

General and administrative expenses in the third quarter of 2018 were approximately $1.3 million compared with approximately $300,000 in the third quarter of 2017. The increase was primarily due to additional payroll and payroll related expenses, professional fees and costs associated with operating public companies.

We had cash and cash equivalents of $47.1 million as of September 30, 2018 again reflecting the proceeds from our IPO. We believe that our current cash position is sufficient to meet our needs for at least the next 18 months. That concludes the financial review and I would like to turn it back over to Vimal for any closing comments..

Thanks Richard, I like to thank everyone for joining the call and we would like to open it up for any questions..

Thank you. [Operator Instructions] And we will take our first question from Geoff Meacham with Barclays..

Hi guys, thanks for the question and congrats on all the pipeline progress. Vimal, I don't want to get specifics but for 501 was there any subscribers in your FDA meeting in terms of the feedback or the development path and for the PK study of 501 what was your view as an ideal profile? Then I have one follow-up..

So, our goal with the -- behind the meeting was very simple, to get a FDA buy-in on our -- like in our overall development plan. And we focused on two areas, which is schizophrenia and bipolar, and Alzheimer's.

And what we understood, I can be very specific, but in general we understood that FDA was very encouraging for both indications because there is no systemic therapy currently available for effective treatment of agitation.

Overall also, we saw the acceptance of the strategy that we have outlined, that we will do the [indiscernible] like now with multiple doses, we will do the pharmacokinetic bioavailability study, and a safety study in using different dose cells of [indiscernible].

And then using that data we can proceeds forward with Phase II/Phase III registration trial in 2019..

Okay.

And then on the PK profile, what would you say for 501 would be an ideal profile? I know you obviously have to run a bridging study at some point with the PK from the IV, but looking forward to the data coming up, what are your expectations?.

So, I have Frank Yocca. I will pass this question to Frank.

Frank?.

Yes, Geoff, so we think we understand what plasma concentrations we need to get our affect.

So what we're looking for in terms of the PK profile is something that we can achieve, like a [indiscernible] around somewhere between 20 to 30 minutes so that we can get a rapid onset of affect, and achieve those dose ranges where in the smaller IV studies we're seeing the activity of the drug producing these graft spores that would fit in terms of reducing agitation..

And we'll take our next question from Carter Gould with UBS Financial..

Hi, this is [indiscernible] on for Carter. Thanks for taking our questions. I got a couple here on 501. So from the point when you have the 501 [indiscernible] in hand, can you talk a little bit more about just the timelines [indiscernible] registrational study approval.

And then how should we be thinking about the duration of the 501 studies relative to the IV Dex one?.

Sure. So let me address, first, the BXCL501 film trial plan. We are expecting to initiate our pharmacokinetic safety study this year upon approval of the IND. We already had the we are in the meeting with the FDA. We have the CMC for the film, multiple doses, already prepared, available for clinical studies.

We have a CRO who is -- like now will be helping us conduct the study. So all pieces are in place, it's a matter of just obtaining the IND approval from FDA, and the studies will start this year.

These studies end to the short study, like they are not that long because you can finish these studies in a short time, so we expect the results to come out in first-half of 2019.

And using the data, we go back to the FDA with a fuller plan and a defined indication whether it's schizophrenia and bipolar or Alzheimer's or both, and get their approval to initiate Phase II/Phase III studies.

Those studies, from our experience -- the previous experience we know at least for schizophrenia and bipolar they require about nine to 12-month. So if we have the approval from the FDA in first-half of 2019, then those studies will go up to early or first-half of 2020, and then we'll be ready to file a NDA.

Our goal and hope is that we should be able to file an NDA, if possible, by 2020, and that's what we are targeting..

Great. Thank you..

And we'll take our next question from Do Kim with BMO Capital Markets..

Hi, there. This is Keith Tapper on for Do Kim. I had a few questions about 701. So the IND for 701, this is to treat NEPC.

Could you talk a little bit about the study design and what that might look like being that it's a treatment emergent disease in terms of exclusion criteria for background treatment or anything like that and how that might affect measuring the outcome..

Thanks for your question. I will pass it on to Vince, who is our Chief Medical Officer..

Sure, thanks, Vimal. So the study as we previously said is a combination approach with KEYTRUDA, and we chose KEYTRUDA based really on the fact that we really had some data from the KEYNOTE-199 study and how KEYTRUDA works or its efficacy or the lack there of in this patient population. So that's the combination approach.

It's a two-stage [indiscernible] design, so 15 patients plus approximately 15. And it'll be preceded by a safety run-in or escalation prior to that. So fundamentally, the primary endpoint is based on response rate. And we can do that because then you can see, unlike classic [indiscernible] of prostate, typically results in measurable disease.

Typically it results in a visceral mets, for example. So that's the general approach to the study.

And does that answer your question?.

It does. In terms of, I guess, exclusion criteria, is there like certain line of treatment that -- because it generally results from previous chemotherapy.

So is there a certain standardization of lines of treatment prior to being involved in the study?.

Sure. So, clearly by definition, as you just said, this is a treatment emergent disease. So by definition, patients had to have the androgen deprivation therapy. And in addition one line of chemotherapy, so those are the inclusions..

Okay, that's very helpful. Thank you..

And we'll take our next question from Sumant Kulkarni with Canaccord..

Good morning. Thanks for taking my questions. I have one. It's actually a big picture question, but has a very specific focus to it. We had the recent ESMO conference where we saw a lot of products coming out -- combo products in immuno-oncology not doing as well as people might have hoped.

So given yours is an organization that relies on artificial intelligence, how quickly can you adapt your product, which also involves a combo? You might find some new intelligence based off of your approach.

Is that a valid way of looking at things? How nimble can you be?.

Sumant, that's a great question. We always use our AI and machine learning platform whenever we are even going to any conference. We have done this for conference that's happening in Washington, D.C., this idea of immunotherapy. So we use that to understand what the overall landscape looks like. And I call that as a machine-created report.

And our scientists, our team that is [indiscernible], this week, they utilized that report to understand the overall landscape, to understand particularly the combination landscape, and then understanding and drilling it down to what other combinations are going on.

At a very high level, if you look at the immuno-oncology, primarily it is combination of adaptive agents which can change the micro environment using adaptive immune approaches. I think where BioXcel Therapeutics separates itself is in the innate immunity.

I think and -- I'm not aware of that we are the only company which has an orally available systemic innate immunity activator, and we have for this agent 700 patient data.

So our belief right from the beginning has been that to be able to get responses, the kind of responses you are looking, and particularly in hard-to-treat tumor, like neuroendocrine prostate cancer and pancreatic tumor, you need to combine innate immunity agent, which is BXCL701, with other agents.

And we have chose right now NKTR-214, we continue to explore more, and a checkpoint inhibitor.

Combination of these three, basically innate immunity activator does the early surveillance of the immune system, it kind of warns the adaptive immunity agents and the professional killer cells which are T cells, they're already pre-warned by the time like you know, you using and activating [indiscernible] immunity plus [indiscernible].

So I think overall we strongly believe that going forward, you will see more and more immunity activation as a strategy employed by other companies as well and I think we are ahead particularly in terms of the [indiscernible]..

Thanks for that..

And it appears there are no further questions in the queue at this time. I would like to turn things back over to Vimal Mehta for any additional or closing remarks..

I like to thank everyone for joining us today, and also excellent questions, I think they are very relevant. We continue to feel very excited about what we have been able to accomplish and what is ahead of the company in Q4 2018 and throughout 2019. Thank you very much..

And that does conclude today's conference. Thank you for your participation. You may now disconnect..