Hello, and welcome to the BioXcel Therapeutics Second Quarter 2020 Financial Results Conference Call. At this time all participants are in a listen-only mode. [Operator Instructions] A question-and-answer session will follow the formal presentation. As a reminder, this conference is being recorded.

It’s now my pleasure to turn the call over to Vimal Mehta, CEO. Please go ahead..

checkpoint naïve patients and patients who are refractory to checkpoint therapy. In June, the safety portion of the trial was complete. And recently, the trial met the efficacy bar in both arms, allowing the study to proceed to completion. We look forward to presenting initial data at a scientific conference later this year.

Furthermore, the combination trial of BXCL701, bempeg from Nektar and avelumab from Pfizer and Merck KGaA in second-line pancreatic cancer, will start following the completion of Nektar and Pfizer Phase Ib safety study of bempeg and avelumab and the outcome of that trial.

We are also further exploring compelling combination with BXCL501 in additional indications with high unmet needs. In July, we raised gross proceeds of approximately $200 million in a public offering, which helped to significantly strengthen our balance sheet.

This cash, together with our current result, provide BioXcel a cash runway well into 2022 to fund key clinical regulatory, operational and commercial activities. With that, I would like to turn the call over to our Controller, Michael Stanton.

Michael?.

Thanks, Vimal. Once again, thank you all for joining us this morning, and a special welcome to our shareholders. BTI reported a net loss of $21.4 million for the second quarter of 2020 compared to a net loss of $8.5 million for the same period in 2019.

The second quarter 2020 results include approximately $2 million in noncash stock-based compensation compared to $1 million for the same period in 2019. Research and development expenses were $17.9 million for the second quarter of 2020 compared to $6.5 million for the same period in 2019.

The increase was primarily due to increases in clinical trial activity. General and administrative expenses were $3.5 million for the second quarter of 2020 as compared to $2.1 million for the same period in 2019. The increase was primarily due to salaries, noncash compensation costs and professional fees.

Total operating expenses for the second quarter of 2020 were approximately $21.4 million compared to total operating expenses of approximately $8.6 million for the same period in 2019. As of June 30, 2020, cash and cash equivalents totaled approximately $65.5 million.

This does not include the $187.5 million in net proceeds generated from our equity offering that closed on July 31, 2020. That concludes the financial review of our second quarter. Now I would like to turn the call back to Vimal for any further comments..

Thanks, Michael. We would now like to open the call to questions.

Operator?.

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions] Our first question today is coming from Geoff Meacham from Bank of America. Your line is live..

Okay, great. Thanks guys for the question and congrats on the progress. I just had a couple. So when you just, as it relates to the SERENITY studies, I know you guys have a time to dig through that.

Is there any data in the elderly population in that study that that could be helpful just as a read-through to the dementia study and just more specifically asking on optimal dosing and moving up on that? And then I have one followup..

Thanks Geoff for the question. We – as you said, we've been digging through the data. And what – wherever we are with the analysis, Rob will provide you some perspective on it..

Yeah. So we've looked at in both trials, the overall AEs for patients aged 65 through 75 that were dose both with 120 or 180 micrograms, and there was no different AEs than the younger patients, really no difference in the profile and also say there were no SAEs in this population or any patients in these trials..

Okay, that's helpful. And then, just as it relates to TRANQUILITY and the dosing. If you assume that, maybe one of the lower doses is what's move forward in Phase III. I know you guys are planning on having several doses available. But if you had an even lower one available for dementia.

Just give us a sense for maybe how you think about that commercially from a – pricing and sort of reimbursement access perspective?.

Sure. Good morning, Geoff. This is Will. So I think obviously we'll wait to see the data and how that plays out relative to the optimal dose for that population. I think it will give us an opportunity then to think through pricing potentially by indication or by dose strength. Again we'll have to wait and see how that plays out.

And then, as you can appreciate for the geriatric dementia population, the Alzheimer's population, that is mostly a Medicare Part D populations. So that would involve interactions with those PDPs and pay organizations.

But I think it would give us a strong position given the lack of approved treatments for acute agitation in dementia, Alzheimer's, et cetera. So I think it would serve as well or – it wouldn't anyway hamper us in terms of launching the product if the dosing were different..

Okay, great. Thanks a lot guys. I appreciate it..

Thanks, Geoff..

Thank you. Our next question today is coming from Robyn Karnauskas from Truist Securities. Your line is now live..

Hi guys. thanks for taking my questions and congratulations. Okay. I have a few. Let me just start with dementia. So you said you've dose up to 60 in the Phase III trial for schizophrenia, you didn't see a stat big benefit there.

And if I recall the IV dose for – in dementia was sort of overlapping, as far as PK/PD with the other indications are going after.

Like, do you have any sense of whether or not, I mean – it's double-blinded, but is there any sort of rule given the adaptive trial design that you've have not seen efficacy or see efficacy at that time point over that bar is. Just trying to get a sense of – if you have any sense yet of how high you could go.

And then the next question would be, so you mentioned that you're going after 90 and if that bet take you into the fourth quarter. What happens if you go even higher, and what is the bar for going even higher above 90? Could that push you into first quarter. Just trying to get a sense on timelines? Thanks..

Great question. Let me address with your second question about the timeline. I think these cohorts generally won't take too long to complete, if COVID-19 situation does not impact our access to their dearly care center. That can become challenging, if cohort go without any like COVID-19 delays, and we can complete it pretty quickly.

So depending on, as you indicated once 90 is completed, do we choose to go further to 120 or 105, whatever dose we will choose, data will guide us. So could it go to Q1? It's very hard to predict, but our goal is – that as soon as we can complete these recruitment and move the program forward.

So it's hard to predict at this time, as we have indicated in our press release also. I will pass it on to Rob, regarding the other aspect of the question, which is related to what activity we saw in SERENITY with a lower doses.

Rob?.

Sure. So we designed the SERENITY trials in the development around agitation and schizophrenia and bipolar, based on our Phase II trial, that Phase II trial was a dose ranging trial. And as you're pointing out, we saw efficacy at a range of doses, it was really remarkably dose responsive.

Now this was obviously patients with schizophrenia and we found some efficacy actually at 60 micrograms in that Phase II trial. We chose to take the higher doses into the Serenity trials because they prove to be greater proportion of response, a greater magnitude of response, and maybe slightly faster in terms of their efficacy.

So we have a lot of things to consider. But the point is that in patients who are much younger, realize patients with schizophrenia, we showed efficacy at doses of 60 micrograms. And that's why we're testing lower doses in patients with dementia. We know that the IV formulation is labeled to use about half the dose in the elderly.

So we actually have a plethora of data to design these trials, and we're using the exact same approach that we've used to generate the pivotal trials that were robustly positive. We're doing the same thing with dementia. .

And Robyn, I like to add also. This is Vimal. Even if you remember in our Phase II schizophrenia trial, 80 was statistically significant. So we start seeing activity with a lower dosing and in schizophrenia and bipolar considering the nature of the patient, then you optimize the dose where you start seeing maximum number of response.

So that's why we had dose escalated to 120 and 180 there. .

Great. Just a quick follow-up, just want to be clear. But I know that yet to start lower for IV, but from what I understood from our conversations is that, the IV-PK look somewhat similar. So we don't really know if that's actually needed for going forward.

I just was asking what is in the protocol, the bar for going even higher, higher than 90, because 90 you could argue that 90, you might have – it could be a better chance.

If it's IV, it looks exactly the same, as far as PK-PD with these older patients than with the younger patients, sort of – as you're sort of alluding to in the schizophrenia trial, then you may need to go 90, you may need to go higher, if you want to have better efficacy.

So what is that bar, just – clear as to the allowance for you to go higher? And then....

Robyn, just – there is no bar for us to escalate..

Okay. I think if you have to be safe, right. You have to have like, then....

Okay. Safety is the bar. If you, of course – we're not going to do anything unsafe. So safety is the bar..

Okay. And then color-wise, anymore clarity, I know you can't predict timing. But have you seen any increase in enrollment, as COVID sort of – shifted like from the beginning of the trial to the end. Has the COVID impact changed or for example, are you enrolling patients faster, as COVID cases come down in certain parts of the country.

Anymore – just – I know you can't give specifics, but just any type of color that would help us feel more comfortable that there couldn't be slowing again potentially?.

We do see our enrollment when there are no impact with the COVID-19, no shutdowns by those state or by the federal level to get access to these facilities. We do see trials do go quite fast. If there is no disruption..

Okay, perfect. Thanks for taking my questions..

Thank you, Robyn..

Thank you. Our next question today is coming from Yatin Suneja from Guggenheim Partners. Your line is now live..

Hey, guys. Thank you for taking my questions. Just a couple from me also. With regard to the safety consideration. Could you maybe talk about how this older patient. Are they even – are they also more sensitive on the safety side. And then with regard to the change in blood pressure or drop in heart rate.

What might be acceptable, like how much drop in heart rate would you anticipate given that you saw that in your earlier trials in schizophrenia? And then I have a follow-up..

Yeah. So we're looking at overall safety, as we continue to escalate the dose and we're particularly, we know that in the elderly and safety of anything with respect to cardiovascular changes can result and for example falls or syncope, where people – if you will pass out and so that's one of the key safety measurements that we're monitoring closely.

And so that's what we're – those are the kinds of events that we're monitoring. But we continue to escalate..

Got it. And then in terms of– can you just comment also on like how many centers? Is it – this consider more of a caregiver setting. Like who is giving a consent.

And then, who is, our patients able to take the films themselves or is it caregiver that is providing the film to them?.

Yes, we haven't – entirely disclosed that. So, it's that typical care center for – where many patients with dementia are residing..

Okay, that's all I had. Thanks..

Thanks, Yatin..

Thank you. Our next question today is coming from Sumant Kulkarni from Canaccord. Your line is now live..

Good morning. Thanks for taking my questions. I have two, the first one is very specific.

Could you please put the data in your May 2019 poster from ASCP into a quantitative context for investors from bridging from an intravenous dose in Alzheimer's patients to a safe and effective dose for the film for agitation and dementia? That's the first question.

The second one is, in the agitation and geriatric dementia setting, clearly, there's a risk-benefit profile that must be considered. What I mean is the risk of someone agitated versus not being treated, even if the treatment has some side effects.

How might you be specifically able to address this aspect when you go to the FDA, for example for approval?.

Yes, the range of plasma concentrations with the IV, kind of proof of concept trials, that produce now a RASS score of minus one. Now, realize that you can treat agitation with the RASS score of zero, but the range that produced a RASS score of minus one was between 100 and 300 picogram per mL.

And so, we know we can achieve that with our sublingual proprietary formulation. With respect to the safety, the risk benefit profile, we know that agitation patients, especially with dementia and agitation, have a number of risks just from the condition. Those risks include physical harm to themselves. They include falls.

They include accidents and falls. So we are – obviously, these are also things that we're monitoring closely. And this is a placebo-controlled trial as most of our trials will be. So we'll have comparator datas what I'm suggesting..

Got it. And just a follow-up to that. What are some of the specific quantitative parameters you set – you're setting as hurdles on the safety when you move to the next cohort..

Because these are – this is a proof-of-concept trial, if you will, or dose ranging. We're looking for any safety signals that would prevent us from testing higher doses..

And that mean, Sumant, that if there is a syncope or there is a syncope and a risk of fall, so those are the key parameters that we monitor for the safety. Obviously, you monitor all the blood pressure and heart rate those changes.

But I think key factor from a dose escalation perspective is whether there is any syncope or whether there is any syncope and a fall..

Got it. Thanks..

Thank you. [Operator Instructions] Our next question today is coming from Do Kim from BMO Capital Markets. Your line is now live..

Hi, this is Jameson on for Do. Thanks for taking the questions and congrats on the progress. One on the delirium program. Given that you have all that additional data from SERENITY, the MGH study, plus the data of IV dexmed, do you think there's a potential for an accelerated path? Or do you still anticipate running the Phase III? Thanks..

So you’re right. If it is COVID-19 patients, there could be some accelerated path including emergency use authorization.

But I think that market is like, you know, that need is sporadic and that market is unpredictable where we are focusing from a commercial opportunity, ICU-based delirium for our first trial, because that's a very well established situation agitation for the delirium patient.

So I'm going to pass it onto Reina, she can put a little more color, the hyperactive delirium patient and what they experienced in terms of agitation..

Yes. So agitation in delirium in several places in the hospital is the reason for higher mortality or outcomes et cetera. So – and there is a lofty track record of IV Dex in successful management of delirium.

So – and keeping our data with the film, I have no doubt that we can certainly pursue and really increase the outcomes of those patients with delirium in several settings..

Great. Thanks. Congrats on the progress, looking forward to the TRANQUILITY readouts..

Thank you..

Thank you. Our next question today is coming from Ram Selvaraju from H.C. Wainwright. Your line is now live..

Thanks very much for taking my question. So, firstly, on the TRANQUILITY program, I had two clarificatory points.

Firstly, if you have great efficacy with the 90 microgram dose, just if you look at the efficacy profile and assuming that the safety is all clear, what exactly would be your incentive to pursue higher doses? In other words, the 90 microgram doses looking very potent and looks like it would give you a high degree of statistical significance.

What would be your specific incentive for looking at higher doses? And then secondly, can you comment on medications that are excluded in the TRANQUILITY study, in particular, those that are known to be associated with high-risk of falls in this population.

And in particular, I'd be interested to know whether NUEDEXTA and NUPLAZID are excluded medications. Thank you..

The only medications right now that are really excluded would be medications that would increase blood pressure, like pressers for example. We have intentionally tried to include patients who are on a range of other medications. We know that the population is treated with polypharmacy unfortunately.

And so we're trying to test this as safely as possible, but nonetheless, and as generalizable a population as is feasible. So the exclusions right now have to do with pressers things that that might alter blood pressure in a negative way..

Okay, thank you. That's helpful. Now, in addition, I wanted to ask about opioid withdrawal efficacy endpoints, which efficacy endpoints you anticipate are likely to be most applicable in that context.

And also if you could enumerate what the potential advantages of a drug like 501 might be in that indication versus, for example, Lucemyra or lofexidine..

We are using the COWS, the clinical opiate withdrawal scale, and the SOWS, the subjective opiate withdrawal scale of Gossop. This is the registrational endpoint that the FDA accepts, for example, to demonstrate efficacy, to treat opiate withdrawal symptoms.

We believe that BXCL501 has both robust effects in terms of treating opiate withdrawal symptoms. We believe it is rapid. We believe it is also – should have a magnitude of effects likely greater than Lucemyra. Now, this is based upon our IV proof-of-concept trial in patients undergoing withdrawal.

And that is also based on preclinical data showing the selective and highly potent agonism relative to Lucemyra..

And Ram, I do want to address your initial question. If we win 90 microgram, we see great efficacy, do we have any incentive to go forward? I think what we are really trying to achieve there is a sweet spot of effectiveness and the tolerability and also the coverage.

We are here not just treating Alzheimer's dementia patients, we are having all kinds of dementia patients and we want to see what dose is working in what kind of patients, so that we can get a good handle and then we can choose for our late-stage development right kind of a dementia population where we see optimal efficacy as well as safety..

Okay, great.

And then just a couple more I was wondering if you could provide us with an update on the COVID-19 associated delirium compassionate use program? And if you are seeing any additional interest, any demand from hospitals beyond MGH in that context? And then if you could provide us with your sort of updated perspective on potential lifestyle indications as it were in the long run with regard to 501? I mean, we've talked previously on other calls about phobias.

I was just wondering if you had any thoughts about conditions like PTSD, middle-of-the-night awakening, things like that, where a calming agent like this might potentially be applicable, of course, in the very, very long run after you get through all of these initial indications. Thank you..

That's right. That's right. As you mentioned, these are quite a bit of potential with 501 to expand and capture the market opportunity. Delirium MGH is a MGA study, and they will provide the update. If they provide us update, then we will provide.

But what that has done coming to your question created interest with a lot of key opinion leaders who are the leaders in this field. And we are working with them to design our Phase II trial in delirium.

So that trial or that visibility like in our interests and FDA’s support to use it for a compassionate use has been helpful and we have speeded up our government for delirium agitation.

I will pass it on to Rob and Reina to talk about more broadly about like how they are thinking about PTSD, alcohol withdrawal, or phobias, there quite a bit of opportunity.

So Rob and Reina?.

And TBI, I mean, phobia, for example, people think of phobias like a fear of flying or a fear of heights. Phobia is actually have a certain amount of background anxiety as well. It does affect their lives. And there is a thing called agoraphobia, which limits people's ability to even leave their home. So it's hard for them to be employed often.

They can't leave the house. There is potential application across a number of these anxiety disorders as well as PTSD, a huge and continuing problem for patients, not just military, but many, many patients develop PTSD from accidents and trauma.

There – again, there's an evidence that dexmedetomidine may have beneficial effects in traumatic brain injury patients, not just in the acute trauma and treating with delirium, there's evidence of that, but there is also potential evidence of long-term recovery. They have, for example, sleep wake disturbances.

It's a classic symptom, almost every patient has that. And obviously, dexmedetomidine can potentially improve sleep as a biomimetic sleep. So it augments sleep in a very specific way that is unique very different than benzodiazepines or other sleep agents. So I'll let Dr. Benabou speak to others..

So thank you very much, Rob. So, honestly, I think we add value in patients' lives if we can either prevent, predict, or – and optimize a disease state. So, there's a lot that we can do in terms of lifestyle on a daily basis as well.

So – and 501 has clearly demonstrated a reliable, consisting, excellent efficacy and safety profile that is due to his central mechanism of action. So we're confident that we also make a difference in all those other indications, as Rob mentioned..

And I would like to also that at some point we will engage the FDA in a conversation where we can seek that there will be opportunity for a broader agitation label if we can prove our drug is working through a central mechanism after having the efficacy in couple of these indications, so there a quite a bit of a strategy that is needed to do the lifecycle management and capture the full potential of this drug.

And I think good news is that we have both clinical team and commercial team and our medical team they're working very closely how to prioritize them and what strategy to use to expand the potential of 501..

Thank you..

Thanks, Ram..

Thank you. We have reached the end of our question-and-answer session. I'd like to turn the floor back over to management for any further or closing comments..

Thank you again for joining our call today. I'm extremely proud of BioXcel's tremendous growth on both the clinical and corporate fronts, and we remain focused on continuing this momentum for the remainder of 2020 and beyond. Have a great day, and please reach out to us if you have any additional questions. Thank you..

Thank you. And that does conclude today’s teleconference and webcast. You may disconnect your line at this time and have a wonderful day. We thank you for your participation today..