Q1 2020 Earnings Call
[music].
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Just to remind everyone certain matters discussed in todays conference call, where answers that maybe given to questions asked are forward looking statements that are subject to risks and uncertainties relating 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 U.S. Securities and Exchange Commission.
Including its quarterly report on form 10-Q, four the quarterly period ended March 31st Twentytwenty, which can be found on its website www dot biotech cell therapeutics Dot Com war on Www Dot S E C Dot Gov.
I would now like to turn the conference over to them all.
Keith Executive Officer Biologic cell Therapeutics. Please go ahead.
Thank you operator, and good morning, everyone and thanks for joining our upon friend's car to discuss the buyers so kind of paradox financial disaster and business highlights for the first quarter 2020.
We appreciate everyone's time entertain true.
Joining me for the <unk>, Richard Steinhart, Chief Financial Officer, Frank Yakov, Chief Scientific officer that Envincio needs Chief Medical Officer.
Before we begin I wondered takes some time to address these unprecedented times. They already name didn't make it crises that has created uncertainty at onboard as strongly affected each and every one off what.
I personally want good times, all healthcare workers, who are dialing and that's new fighting on the front line getting for patients and be ramping before that is part of the way that they had hard won't be that was all what everyone lasting grad teacher.
Following the guidance from the U.S. sentence, what do these control and prevention and they stayed up and I think that we have made wiped out I guess my across our company aimed at protecting out what employee health and safety widen maintaining key activities needed to had won the governmental policy under control.
The by itself be remains more <unk> and going back to lead our 2020 miles grows in order to money into the potential impact, though they spend they make on our ongoing Perez rehabbing, the always gone back without getting too because I.
To date, we have not experienced any significant delays in our ongoing clinical died and have made great strides with our clinical problem.
We will continue to close anymore. They turned up over 19 situation and worldwide any updates as needed.
Shifting our discussion back to our corporate highlights.
I wouldn't like to Pfizer disguise, our lead neuroscience clinical program be axiata fiber one.
And then they might not be axiom fiber one is our proprietary penetrating formulation or dexcom, Adam redeemed or that's for the acute deep meant for the treatment of acute education.
We haven't design this candidate to be easily administered and have it rapid onset of action in order to produce Eric I mean in fact without excessive city.
We haven't made you had one sees discord out in our favorite kind of time see they may be one then to.
Reach on phase three studies or be Axiall fiber, one for the acute treatment of vegetation in patients with schizophrenia and bipolar disorder.
Back in March Vietnam that more than one third of the patients inboard tight hadn't been enrolled and treated and Golden Marin continues to be progressing as planned and we have not observed it changing and Gordon mandate you do that will remain in pending.
Riyadh aren't that do report <unk>.
Topline data from that sit energy program in mid 2020, and really communicate update as we get close a two hour clinical data readout.
It is important to note that all these kids every now and bipolar depression and <unk> have successfully satellite and they started the be actually in fiber one piece men guided by your health care provider.
With our favorite at programs eat out only a few months Oh, Hey, you had a whole foods that we will be everybody to submit our first endear for be acted in fiber one during the first half our 2021 being getting and noninvasive fast acting treatment with the millions of patients who suffer from MCU Daddy's <unk>.
No. Thank I think this evidence.
Our plenty good progress continued with the initiation of tranquility. This study a phase one be to try and what they did find out for acute agitation in Judy I think dementia highlighted the potential volatility of neuro all four of our nearest type program, including disorders and beyond.
<unk> neurotic psychiatric conditions.
Since the Bankability time has an adaptive design. We are currently the viewing safety and Tolerability data in order to choose the next stepped into.
This ties enrolled and many of these aren't right and top line data is expected in mid 2000 Grundy.
With off label dog, having a black box warning for the R. Donnelley and no M.D.A. approved therapies to treat agitation in Judy I think dementia that either desperate need fought it safe and effective treatment for patients that goes with the psychological behavior.
Redesign be actually in fiber one to be a fast acting easy to administer therapy to try to fill this unmet.
Need and provided <unk> Mandeep roots, what do you have your was that have thought about managing dementia related agitation.
In parallel we continue to investigate the U.S, albeit at both digital device technologies, such as Apple watch to hopefully in NZ prevention in demand.
Mendo dementia related agitation.
Conceiving. This patient population is among the highest it's got big ready for severe illness and that that should share did with Colby 19, we have taken it gosh anything major it appears that was the safety and hadn't thought Oh, what our daily patient part D. Bancolombia tied it ought to be axiom fiber one go they have all it.
I had been manufactured and have been provided to the long term care facility because I didn't get tranquility study there do you think they they offer exposing the patients.
Additionally.
We are continuing our investigation I'd be actually on fiber ones coming capabilities to the initiation of phase two study being performed by researchers have yielding an industry.
The aim of these studies to assess the biomarkers associated with education, such as can conduct tends to this bonds heart rate variability and blood pressure in patients with schizophrenia and their response to determine we'd be axiom fiberlan.
Managing education any that Alan for both physicians and category rather than being able to use. These biomarkers and then initiate is taken note prior to the onset of visit but same terms could be ready banding fishing. These borderlines, taking notice, but provide sufficient time, but can you give a good deliver I'd be actually if I want to pay insurance.
Before an education episode of become dangerous you. One don't do studies have been perform only on his schizophrenia patients. We believe this side of it had to open the door to investigate the potential use all be axiom fiber one in additional indications that exhibit.
Hey, Mike similar physiological signals overhype I'd, rather than expanding before pension market does this candy that into chronic agitation.
To wrap up this quarter. Its nearest things highlights we received and be a clearance or what I envy application for be acts in five or one four defeat amendable, there I'd withdrawal symptoms they put pension fourth indication for this candidate.
As the number one cause of death in the U.S. why didn't we draws under the age of 50 years or accidental drug overdoses is embedded in this country with the majority of fatalities opioid related.
What are the remote opioid withdraw it can be very debilitating and I'm comfortable and many are they continue to dig. These drugs are they labs to award feeling the same thing same dumb.
There is a desperate need for Neil efficacious treatment options do have a they are nets out population from continuous abuse.
Do you have to be Akcea is my old ones intrinsic potency favorable delivery method and mechanism of action. We feel this can eat it has the potential to off what I'd wanted you to do you think written it all the same time, helping to come back and have you that artists to the lapse.
We are encouraged by the bromine things is that the weakness with intervene as Dan and elevating <unk> same time and be Atlantic doing he shared that elite trial, a phase one beat due time for the <unk> mendo patient Expedia and thinks in terms of opioid withdraws shortly.
[noise] this past quarter.
We have focused on what afterwards on expanding our neuroscience programs linear got into alignment strategy exploding numerous indication in hope so in hopes of reaching an extensive patient population. We believe be act in fiber one off what their differentiated therapy to treating the millions of patients.
That's a lot from I, but I doubt that and it lack effective ordinated.
We have focused on increasing the commercial value of the Skandi, there and plan on exploding be act in fiber, one and their potential feet men for acute agitation and high productivity level as well as for chronic education.
I think the conversation or to our immuno oncology clinical candidate B.S.T. as having a one hour orderly ever live a systemic innate immunity active <unk> equity radar designed with a dual mechanism affection.
Recently, we are now that we had initiating the phase two efficacy portion of the phase one be two trial well be akcea is that when no. One in combination with good food up what Pete and I imagine neuter endocrine prostrate cancer or de NEPC.
Did that from the phase one be safety, leading showed that they split daus totaling 4.6 milligram per day is that recommended doing when used in combination with keytruda.
This big dog has shown aren't tied to get side effects and since then weve site of and activation and has an improved safety profile.
In the phase two efficacy.
Hi, approximately that would be eligible man with D N repeat it.
Well, let's see 0.3 milligram or be Akcea is that when no. One twice daily on days, one to 14 of eastern DRAM. They cycle like 200 milligram of Keytruda administered intravenously on day, one and then every 21 days.
We expect to report initially in theme data from the phase two trial in the fourth quarter of 2020. Currently there are no approved treatments 40, any busy and they had and highly aggressive form of prostate cancer.
We believe we actually had a sad when no one has the potential to be an effectively men for the sub population and it creates an aggressive adaptive immune response, making too much more reactive to immuno therapy, including get to them.
Furthermore, the company's all the third one thing that clinical evaluation of be act is that one on one via the ongoing open label Phase two basket trials conducted at the MD Anderson Cancer Center. This study is evaluating the combination I'd be exits that when no one and paid through the.
In patients with at one started it can said that that either naive to or the frac. They do checkpoint tabby Andy's following the dosing schedule using the phase one be pledged to study for D.N. need be.
The combination fraud or be Axiata, seven no one big impact from mapped out and their value Matt from five that end Mulcahy in pancreatic and said we did start following the completion of net that inphi that phase one be safety study of Empaque and I've allomap and the outcome.
All of that.
In February we raised net proceeds of approximately 16 million in their public offering.
Which had been significantly strengthen our balance sheet. This cash together with our cotton valley, though for a wide buyers that enough cash run.
The way to find preclinical recordati anticipation milestones into 2021.
With that I would like to have done that call over to our CFO Richard Steinhart They check.
Thanks demo once again, thank you all for joining US this morning, and welcome to our shareholders.
VTR reported a net loss of $14.9 million for the first quarter 2020 compared to a net loss of 7.2 million the same period in 2019.
The first quarter 2020 results include about $800000 noncash stock based compensation <unk> I'm sorry compensation.
The increase in net loss was primarily due to cost associated.
With our pivotal phase three trials.
Research and development expenses were 12.4 million for this first quarter 22020, as compared to 5.7 million for the same period in 2019.
The increase was primarily due to an increase in clinical trial costs salaries bonus and related costs professional research and project related costs and chemical manufacturing can controls related to our be XL fiber, one and be actually out seven to one product candidates.
General and administrative expenses were $2.6 million for the first quarter 2020, compared to 1.7 million for the same period in 2019.
The increase was primarily due to professional fees for legal and patent matters.
Total operating expenses for the first quarter of 2020 or approximately $15 million as compared to total operating expenses of approximately 7.4 million the same period in 2019.
We had cash cash equivalents $80.1 million as at March 31st 2020.
That concludes the financial review the first quarter now I'd like to turn the call back to demo for any further comments metal.
Thanks Richard.
We would now like to open the call the questions operator.
Thank you.
This time will be conducting a question and answer session. If he would like to ask a question. Please press star one on your telephone keypad.
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Our first question today comes from Geoff Meacham of Bank of America. Please proceed with your question.
Hey, guys. This is Scott on for Jeff. Thanks for taking my question wanted to ask about the phase one two study for fiber wanted dementia.
Seems the doses are lower at 30, 60, 90 micrograms due to the elderly patient population.
However in the phase one trial in schizophrenia, 60 milligram microgram dose that was not statistically significant. So I was wondering if we should anticipate a tighter window for success given that needs to balance dosing and efficacy in these patients.
And then as a follow up for seven to one.
You recently noted the 0.6 milligram dose is recommended and that's putting the dose can be associated with improved safety profile.
You know there certainly concerns around the safety for seven to one given the drugs. Prior history. So can you give us any insight as to whether splitting that those might impact efficacy for any reason and the potential concerns you're seeing around dosing it as a once daily thanks.
Thanks, Scott this is venmo.
I'm going to positive solution to a probably think a who's our VP of clinical nearby.
Onsite about five of them question and then Vince will pick up the question related to be Akcea is having a one at all.
Sure. Thank you Jeff good question.
The the dose range in schizophrenia.
We're not banking if you will on a thing identical in dimension as you point out they are older. They tend to be more trail and so we are testing at a slightly different dose range.
Simply to determine.
The doses that may be safe, but also affected.
Vince.
Yep.
For the the question so on that on something to one that was really around splitting dose versus our says no I believe so what we saw and a once daily buildings and non split doors.
<unk> 0.6 milligrams of 600 micrograms was a antibody syncope and this was presented at the ASCO Gi poster at just a couple of months ago.
The quad is to expand the cohort or otherwise.
It's actually identify a better tolerated EM there wasn't shed Joe what we find is that by splitting the doors, we no longer so those types of the Ben we certainly still so some on target toxicity I don't colleges you know that's not necessarily a bad thing at all.
So the dog is doing but it's expected to do but the events of syncope or what we're simply no longer than a longer cousins. So that's a you know what we believe we fix the tolerability by splitting those hopefully that answers your question.
Got it thanks guys.
The next question is from Robin Caracas of Suntrust Robinson Humphrey. Please proceed with your question.
Hi, guys. Thanks for taking my question.
First of all <unk> give us some data points on what's happening to be completed.
The opioid withdrawal trial, what are the right let me start.
And then do you think well we still do you think will still we still see data. This year and then on seven to one so we think about.
What you've learned on safety profile, what does the safety.
The other drugs did you learn anything about.
The way it should be working just give us some thought.
So far.
Thank you.
Thanks Robin this is the them all.
Regarding the opioid would draw overtime.
We had all the pieces there due to go to initiate that Todd.
And we were running teatime synergy run into a pivotal phase three and try and go to tranquillity time and discovered situation at all we made a conscious choice as a company do like you know North star the old <unk> light in the middle of code 90, Indeed.
That's helpful employees our partners.
Employees as well as far the patient safety as you might have note is that go over 19, it's creating even a bigger ER opioid crisis is a which is enough, but I thought or what are the blade the number of fish and aplenty and they keep coming to the side and that's what have we have been advised by I want to see outerwear.
unknown: BF-WATCH TV 2021
unknown: Good morning and welcome to BioXcel Therapeutics' first quarter earnings conference call and audio webcast. Before we start, I would like to inform you that this conference is being recorded and that all participants are in a listen-only mode. At the request of the company, we will open the conference for questions and answers after the presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad.
Let me make those that we go to me shared this sturdy anytime I don't think there's any rate limiting.
Step and we will provide the garden shortly as we indicated that we are about to initiate that's not a at that time. So we got into good position to initiate that <unk>.
unknown: Just to remind everyone, certain matters discussed in today's conference call or answers that may be given to questions asked are forward-looking statements that are subject to risks and uncertainties relating 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 U.S. Securities and Exchange Commission, including its quarterly report on Form 10-Q for the quarterly period ended March 31, 2020, which can be found on its website, www.bioxceltherapeutics.com or on www.sec.gov. I would now like to turn the conference over to Vimal Mehta, Chief Executive Officer of BioXcel Therapeutics. Please go ahead.
Regarding the seven no one question I will.
Oh ask a vince to rain or what we have been able to observe beside that safety anything that's something that save a efficacy aspect Vince.
Sure. Thanks, Syed them, all and Wonder woman, So I got I'm going to put it back to the data that we presented on school Gee you in terms of the leveling I think unimportant one is that.
Let's talk seems to be a strong acted each of them. They didn't system and I think that's important because that's far EM many of the although potential approaches to activating EM in each community.
So we do see things like either in Chile, My Algea Puffy ankles, all of which actually funds and be tend to settle down after the first or second cycle.
What we did see as I mentioned some of the previous question was hypotension, then one episode of Simcity by splitting that <unk>.
Vimal D. Mehta: 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 2020. We appreciate everyone's time and attention. Joining me for the call today are Richard Steinhart, Chief Financial Officer; Frank Yocca, Chief Scientific Officer; and Vince O'Neill, Chief Medical Officer. Before we begin, I want to take some time to address these unprecedented times. The COVID-19 pandemic, a crisis that has created uncertainty around the world, has truly affected each and every one of us. I personally want to thank all healthcare workers who are tirelessly fighting on the front lines, caring for patients and preventing the further spread of the virus. Their hard work deserves our everlasting grace.
Retention, they don't seem to have mitigated substantially and yet we see we still see the remainder of those oftentimes it took system. So.
You know I believe is that we dominate activates that is at the same type C. And of course, you know we have a large city, it's behind the dog ready at this point. So that's one important allowing m., we haven't released any detail. So the ASCO do you or since you asked would you presentation, but I would simply calling on efficacy. When this is the one be portion.
To make yes, 50 study the majority of patients were stable and stable didn't does even remained on study at the time of that presentation.
So.
Also give an update on the other trials.
No one and if they're proceeding with any issues.
And just the latest update there and when we get to the next he does that.
Vimal D. Mehta: Following guidance from the U.S. Centers for Disease Control and Prevention and the state of Connecticut, we have made vital adjustments across our company aimed at protecting our employees' health and safety while maintaining key activities needed to advance the development of our clinical system. The BioXcel team remains motivated and on track to reach our 2020 milestones. In order to monitor the potential impact of this pandemic on our ongoing trials, we have been in close contact with our clinical sites. To date, we have not experienced any significant delays in our ongoing clinical trials and have made great strides with our clinical program. We will continue to closely monitor the COVID-19 situation and provide any updates as needed.
Thank you.
Sure. So I'll take them off I mean, I'll take the the Triple combination studies. So I think as we've mentioned the Dublin they've been paid on next up to one slipped plus the Belmont has to be combined before would be iden and seven to one and so that studies. It ends in the hands or find that Pfizer didn't items, maybe six weeks ago pausing old trials.
The anime style. We can go the reopening old trial. So that study was underway at the patients were enrolled EM and there's no reopened again to accrual. So our anticipation is that as we previously guided I didnt seven to one portion to that Dublin.
Once we have seen data later on this year. So that's the triple combination and then be MD Anderson study, that's an important part the development plan as you're aware that simple schumer's both in patients who are keeps it naive and also patients who feel keytruda progressed EM that study is a is it opened a just at the time of the year.
Vimal D. Mehta: Shifting our discussion back to our corporate highlights, I would like to first discuss our lead neuroscience clinical program, BXCL501. As a reminder, BioXcel 501 is our proprietary thin-film formulation of dexmetamidine or dex for the treatment of acute agitation. We have designed this candidate to be easily administered and have a rapid onset of action in order to produce a calming effect without excessive sedation. We have made key advances this quarter in our pivotal trials, Serenity I and II, which are Phase III studies of BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorder. Back in March, we announced that more than one-third of the patients in both trials had been enrolled and treated. Enrollment continues to be progressing as planned, and we have not observed a change in enrollment rates due to the COVID-19 pandemic.
And that's a that's progressing again consistent with guidance, we are anticipating them to present data at the ended the year from <unk>.
Vimal D. Mehta: We are on track to report top-line data from the Serenity program in mid-2020 and will communicate updates as we get closer to our clinical data readout. It is important to note that all schizophrenia and bipolar patients enrolled have successfully self-administered the BioXcel 501 treatment guided by a healthcare provider. With our pivotal program readout only a few months away, we are hopeful that we will be able to submit our first NDA for BioXcel 501 during the first half of 2021, bringing a non-invasive, fast-acting treatment to the millions of patients who suffer from acute agitation associated with neuropsychiatry. Our clinical progress continued with the initiation of TRANQUILITY, this study, a phase 1b2 Since the Tranquility Trial has an adaptive design, we are currently reviewing safety and tolerability data in order to choose the next test. This trial's enrollment is on track, and top-line data is expected in mid-2020.
Great. Thank you.
Oh.
The next question is from do Kim of BMO capital markets. Please proceed with your question.
Good morning, Thanks for taking my questions.
Hoping you could review for US what you could be what would be considered clinically meaningful in terms of reduction.
In the Serenity trial and when you look at the attitude study he saw slightly benefit and bipolar patients versus placebo then for schizophrenia. That's something you'll also expect or is it just within normal variability for agitation studies.
Thanks, Joe for the question.
I've been asked the job to answer that question about schizophrenia, what's his bipolar drop.
Yeah.
So so the difference between schizophrenia effect and I pulled effect for anti Psychotics.
Tends to be relatively minimal.
They're I realize it out of soup, how to slightly better efficacy and bipolar patients than schizophrenia.
We're seeing from the one or two data the phase to serve the the ascending dose trial that we did we saw.
A a clinical benefit some improvement by C.G. I.
<unk> of one point equated to an improvement.
On the Panther I didn't toward components score of around two to three points. So we'd expect to see a similar effect for patients with bipolar as schizophrenia, it's possible, but it'll be greater but we are that remains to be seen.
I could blacks on about potential reasons, why it might be more efficacious and bipolar than schizophrenia, having to related to for example, the autonomic nervous system on the relationship activation in bipolar patients, who are agitated and manic or hypomanic.
But we'll know a lot more once we have the actual data. So short answer I don't know if there will be a difference it would be interesting, but we're not counting on that in terms of the powering of the trial.
Vimal D. Mehta: With off-label drugs having a black box warning for the elderly and no FDA-approved therapies to treat agitation in geriatric dementia, there is a desperate need for a safe and effective treatment for patients that struggle with this psychological behavior. We designed BioXcel 501 to be a fast-acting, easy-to-administer therapy to try to fill this unmet need and provide a treatment, if approved, for caregivers In parallel, we continue to investigate the use of wearable digital device technologies such as the Apple Watch to hopefully enhance the prevention and treatment of dementia-related agitation.
And do you still see a negative three point reduction in tech score as the bar foreign clinically meaningful and either patient population.
Yes, it's about yes, we were able to demonstrate that in the smaller studying whether you expect that.
That degree of change would represent a clinically meaningful improvement.
Great and could you do you have to speak to that no other rising cases and delirium.
Vimal D. Mehta: Considering this patient population is among the highest risk category for severe illness and death associated with COVID-19, we have taken precautionary measures to preserve the safety and health of our elderly patients. For the Tranquility Trial, all BioXcel 501 doses have already been manufactured and have been provided to the long-term care facilities running the Tranquility Study, reducing the risk of exposing patients. Additionally... We are continuing our investigation of BioXcel 501's calming capabilities through the initiation of a Phase II study being performed by researchers at Yale University. The aim of this study is to assess biomarkers associated with agitation, such as skin conductance response, heart rate variability, and blood pressure, in patients with schizophrenia and their response to treatment with BXCL5. Managing agitation is a challenge for both physicians and caregivers, and being able to use these biomarkers as an initial signal prior to the onset of visible symptoms could be very beneficial.
Hospitals are setting due to come in 19, and whether you should actually start looking at any fiber one study a in that setting no worsening later.
It's a dog this is remote [noise] <unk>.
And there's a lot of press at all in like and all these coping 19 patients who are largely due to green do vision as well as forced into vision.
Mechanical vendors.
Use they are getting delayed EM and had a ton. So reason why they're getting it but a lot of patients are getting that so we continue to be fully evaluate how we can have these patients and we talked about what you've been a leader and explore those opportunity.
In terms of Ah.
The Lady and program be already it started to dig part of our denim and so we are trying to speed up that conceiving that didn't see their demand does need in the hospital that thing for eating guys. You do agenda is everything from the lithium.
For the benefit of covering nine didn't patients as well as for our own the strategy could even so Rob would you like to add anything on on that.
Vimal D. Mehta: These bodily signals could provide sufficient time for caregivers to deliver BXCL501 to patients before an agitation episode becomes dangerous. Even though this study is being performed only on schizophrenia patients, we believe this trial will help open the door to investigate the potential use of BXCL501 in additional indications that exhibit similar physiological signals of hyperarousal, expanding the potential market of this candidate into chronic disease. To wrap up this quarter's Neuroscience Highlights, we received FDA clearance of our IND application for BXL501 for the treatment of opioid withdrawal symptoms, a potential fourth indication for this candidate. As the number one cause of death in the U.S. for individuals under the age of 50 years old, accidental drug overdose is rampant in this country, with a majority of fatalities being opioid related. Furthermore, opiate withdrawal can be very debilitating and uncomfortable, and many addicts continue to take these drugs or relapse to avoid feeling these distressing symptoms.
I would just say that.
The <unk> for you feel five or one decks met at nobody is already commonly used to treat delirium and I see you setting.
And so we do see data the sort of low hanging fruit and we're exploring.
Potential development options in that area.
Great. Thanks for taking the questions guys.
Thanks, though.
The next question is from Sumant Kulkarni of Canaccord. Please proceed with your question.
Thanks for taking my questions first one on fiber one and then I have a follow up on seven no. One so we've seen some developments and the competitive side in chronic management agitation Alzheimer's patients. How specifically do you expect to potentially bridge from acute treatment of fiber one the contact management side, if that's possible and on 710.
We've been encouraged to see the best progress into the phase two efficacy part of the trial for the NEPC in combination with Keytruda, but could you give us a sense of what we expect in terms of the body you might need to hit on efficacy and how to potentially bar soaps single agent activity do you have seven no. One what does that give you get the combo.
Vimal D. Mehta: There is a desperate need for new efficacious treatment options to help aid this underserved population from continuous abuse. Due to BioXcel 501's intrinsic potency, favorable delivery method, and mechanism of action, we feel this candidate has the potential to offer advantages in treating withdrawal symptoms, helping to combat an abuser's urge to relax. We are encouraged by the promising results we witnessed with intravenous drugs in elevating withdrawal symptoms, and we are planning to initiate the release trial, a Phase 1b2 trial for the treatment of patients experiencing symptoms of opioid withdrawal shortly.
Hi, Thanks, so much.
Regarding the fiber one question related to.
Or can you. Please repeat the question I'm, just I want to make sure we announced that it.
Right. So the question specifically was we've seen some developments on the competitive site on chronic management decided station Alzheimer's patients.
You have a product like now that has isn't development for acute agitation, how could that potentially be translated to maybe a chronic management does I did they can also in the longer run.
Great question, I think we see that any effects as related to do you think education in dementia flash as I'm is a big problem. It.
Vimal D. Mehta: We have focused our efforts on expanding our neuroscience programs, clinical development strategies, and exploring numerous indications in hopes of reaching an extensive patient population. We believe BioXcel 501 offers a differentiated therapy for treating the millions of patients that suffer from hyperarousal and lack effective alternatives. We are focused on increasing the commercial value of this candidate and plan on exploring BioXcel 501 as a potential treatment for acute agitation in hyperactive delirium as well as for chronic agitation. Now turning the conversation over to our immuno-oncology clinical candidate, BioXcel 701, our orally available systemic innate immunity activator designed with a dual mechanism of activation. Recently, we announced that we are initiating the Phase 2 efficacy portion of the Phase 1b2 trial of BioXcel 701 in competition with Keytruda for the treatment of emerging neuroendocrine prostate cancer or TNE. Results from the Phase 1b safety lead-in showed that a split dose totaling 0.6mg per day is the recommended dose when used in combination with ketones.
Currently the as you know that I know treatments of 11 to do these patients under those circumstances.
We all know neurological conditions as they progress that education episode or the spectrum start changing you may be there, but he agitation stayed then you go to I thought I'd guys. You teach and then it's up chronic education in chronic education, so any kind of a deep men that can be.
We are chronic education will be good because that can help reduce number of episode what is still if you think about the education, that's happening and ER acute agitation, that's still happening in these patients and in the beginning a you have in sport I do you guys you dish and there's no need to use it.
Richard I. Steinhart: This split dose has shown on-target side effects consistent with cytokine activation and has an improved safety profile in the Phase II efficacy trial. Approximately 30 eligible men with TNEPCF will receive 0.3 mg of BioXcel 701 twice daily on days 1 to 14 of each 21-day cycle, plus 200 mg of Ketruda administered intravenously on day 1 and then every 21 days. We expect to report initial entry into data from the Phase II trial in the fourth quarter of 2020. Currently, there are no approved treatments for TNEPC, a rare and highly aggressive form of prostate cancer. We believe BioXcel 701 has the potential to be an effective treatment for this subpopulation as it creates an aggressive adaptive immune response making tumors more reactive to immunotherapies, including ketosis.
Chronic I'll give you tend to run a education can be de degree there or something that'd be the acute treatment as well as in chronic so do we have you think about this is that's already can be two episodes that month and do you really need a chronic education or it could be to appease the week fills up chronic treatment will be sufficient in the one.
It does become a chronic and they they treatment that can be effective introducing number of episode what they still want to have breakout episodes. So that need via fiber one will freight Dan as well as some of these chronic education drugs take quite a long time six to eight weeks.
To become effective and you'd need to manage the beach and so we think that board fiber one fortyk due date morneau vegetation will fit in very valid with any chronic education. It will have in fact support and build the market a deep wendy's overlaid by the for both kind of situation.
So did it on to your question, what I'm happy to embellished more on it I don't know that that's great. Thanks.
So regarding the seven no one question I will.
I see dawn to Vince to describe what Debat is and what to expect from away neuroendocrine Prostrakan surprise.
Sure. Thanks, so much money someone so I think the question was.
Really to food. So one is what might the Bobby in high will be no.
Contribution of seven to one or the into that your contribution like let's say the latter question first and the has been a study with single agent Keytruda. This was really quite we did not include an internal keytruda alone control arm.
Richard I. Steinhart: Furthermore, the company is also advancing the clinical evaluation of BXL-701 via the ongoing open-label Phase II basket trial conducted at the MD Anderson Cancer Center. The study is evaluating the combination of BXL-701 and Keytruda in patients with advanced solid cancer that are either naive to or refractory to checkpoint therapy and is following the dosing schedule used in the Phase I-B-2 study for T and E. A combination trial of BioXcel 701, BAMPAC from Nectar and Avalumab from Pfizer and Merck AGA in pancreatic cancer will In February, we raised net proceeds of approximately $60 million in a public offering, which helped to significantly strengthen our balance sheet. This cash, together with our current reserves, provides BioXcel NF-CASH1 runway to fund key clinical, regulatory, and operational milestones into 2021. With that said, I would like to turn the call over to our CFO, Richard Steinhart. Richard.
And I'm in a similar not identical but similar population EM and then that setting. This was a castrate resistant prostate cancer group of patients who had sweetened with Eaton either underwritten blockers.
And in that setting the response rate data Lewis.
Between three and 5% EM. So I think they have a reasonable handling what keytruda can do in this and this classic cold tumor EM given there is no standard of care here.
We believe the response rate <unk> durable, okay, and that's a feature obviously a back then you know agents and my response rate and the team typically 15% at or above would be of interest to the feel especially with new field don't quite substantially.
Thank you.
The next question is from some of your Divani of Rx Securities. Please proceed with your question.
Hi, everyone. Thanks for taking my questions and it's good to see that the trials are on track. Despite these challenging times.
My question is actually on the delivery and study you're planning, it's like again coming back to dicing.
It looks like you're exploring 20 in 60 micrograms in that study so.
I guess could you, perhaps just elaborate and how that compares tea.
What's currently being used with the intravenous product currently no say, whether there are any extra.
Vimal D. Mehta: Thanks, Vimal. Once again, thank you all for joining us this morning, and welcome to our shareholders. BTI reported a net loss of $14.9 million for the first quarter of 2020, compared to a net loss of $7.2 million for the same period in 2019. The first quarter of 2020 results include about $800,000 in non-cash stock-based compensation. The increase in net loss was primarily due to costs associated with our Pivotal Phase III trials. Research and development expenses were $12.4 million for this first quarter of 2020, as compared to $5.7 million for the same period in 2019. The increase was primarily due to an increase in clinical trial costs, salaries, bonuses, and related costs, professional research and project-related costs, and chemical manufacturing and controls related to our BXL501 and BXEL701 product candidates.
R&D cost associated with that program. This year, thanks very much.
Vimal D. Mehta: General and administrative expenses were $2.6 million for the first quarter of 2020, compared to $1.7 million for the same period in 2019. The increase was primarily due to professional fees for legal and patent matters. Total operating expenses for the first quarter of 2020 were approximately $15 million as compared to total operating expenses of approximately $7.4 million for the same period in 2019. We had cash and cash equivalents of $80.1 million as of March 31st. 2020. That concludes the financial review of the first quarter. Now I'd like to turn the call back to Vimal for any further comments. Okay, Vimal?
[noise] that made thanks for your question regarding the ability we have not it's spelled out what the plan is what does this mean, we'll be using because that's still in their planning stage.
Dementia, obviously, it's on the clinical trials dot com and a delay the m. using their planning C.
Conceding that there is such a big.
Brendan make situation with Govi 19 in these patients that they need to be up kind of speeding up or down a man as part of our overall strategy, we always do where phase one be pledged to these does don't tend to be that much capital intensive.
And we do it on chart number of patients as you have seen without what its schizophrenia and dementia.
Andy size tend to be a like in a enrolled rapidly and finish fat. So because of all goes. He then we don't see much impact on our clinical pad, it's Brian, but there will be though and we are still in the planning state.
We will provide the guidance once we are all pieces come together when the style will start and when we can provide the data readout on on dilutive.
Thanks very much.
Thank you.
There are no additional questions at this time I like to turn the call back the female Mehta for closing remarks.
Thank you again for joining our call today during the first quarter 2020, we remain focused on advancing the development of our two clinical program be axiom fiber one is that when no one with key data read out occurring in the coming on.
unknown: Thanks, Richard. We would now like to open the call to questions, Operator.
unknown: Thank you. At this time, we'll be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the start keys.
Good day and please reach out to us if you have a if you have any additional questions. Thank you.
This concludes todays conference you may disconnect your lines at this time.
unknown: Our first question today comes from Jeff Meacham of Bank of America. Please proceed with your question. Hey, guys. This is Scott on behalf of Jeff.
Thank you for your participation.
[noise].
unknown: Thanks for taking our question. I wanted to ask about the Phase I-II study for 501 in dementia. It seems the doses are lower at 30, 60, and 90 micrograms due to the elderly patient population. However, in the Phase I-B trial in schizophrenia, the 60-microgram dose was not statistically significant. So I was wondering if we should anticipate a tighter window for success given the need to balance dosing and efficacy in these patients. And then, as a follow-up, for 701, you recently noted the 0.6-milligram dose is recommended and that splitting the dose can be associated with an improved safety profile. You know, there are certainly concerns around the safety of 701 given the drug's prior history. So can you give us any insight as to whether splitting the dose might impact efficacy for any reason and the potential concerns you're seeing around dosing it as a once-daily? Thanks.
Vimal D. Mehta: Thanks, Scott. This is Vimal. I'm going to pass this question to Rob Risinger, who is our VP of Clinical Neuroscience, to answer the 5R1 question, and then Vince will pick up the question related to BioXcel 701.
unknown: Rob?
unknown: Sure. Thank you, Jeff. Good question.
unknown: The dose range in schizophrenia, we're not banking, if you will, on it being identical in dementia. As you point out, they are older; they tend to be more frail. And so we are testing a slightly different dose range, simply to determine the doses that may be safe but also effective.
unknown: [inaudible]
[music].
unknown: Yep. Thanks, Scott, for the question. So on 701, it was really around splitting the dose versus not splitting it, I believe. So what we saw on the ones daily dosing, so the non-split dose at 0.6 milligrams or 600 micrograms was an event of syncope. And this was presented on the ASCOGE poster just a couple of months ago. That required us to expand the cohort or otherwise essentially identify a better tolerated. Dose and Schedule. What we found is that by splitting the dose, we no longer saw those types of events. We certainly still saw some on-target toxicity, and in oncology, as you know, that's not necessarily a bad thing at all.
unknown: Got it. Thanks, guys. The next question is from Robyn Karnauskas of SunTrust, Robeth and Humphrey. Please proceed with your question.
unknown: Hi guys, thanks for taking my question. First of all, can you give us some data points on what steps need to be completed in order to start the opioid withdrawal trial? What are the rate-limiting steps? And then, you know, do you think we could still see data this year?
unknown: And then on 701, so when you think about... What is the safety profile? What is the safety sort of hint to you around the efficacy of the drug? Did you learn anything about whether the drug is working the way it should be working? Just give us some thoughts on what you've learned so far from the early data.
Vimal D. Mehta: Thanks Robyn. This is Vimal.
unknown: Regarding the opioid withdrawal trial, we had all the PCs ready to go to initiate the trial, and we were running three trials, Serenity 1 and 2, our Pivotal Phase 3, and Tranquility Trial. And this COVID situation arose.
Vimal D. Mehta: We made a conscious choice as a company to not start the opioid withdrawal trial right in the middle of COVID. In the interest of our employees, our partners, and employees, as well as for the patients. As you might have noticed, COVID-19 is creating even a bigger opioid crisis, which is in the press all over the place; the number of patients is plenty, and they keep coming to these sites. And that's what we have been advised by our CRO and the clinical team. We could initiate this study anytime. I don't think there is any rate-limiting stuff.
unknown: And we will provide guidance shortly, as we indicated that we are about to initiate that trial. So we are in a good position to initiate that opioid withdrawal. Regarding the 701 question, I will ask Vince to weigh in on what we have been able to observe besides the safety, any signals from the efficacy aspect. Vince?
unknown: presented at ASCO-GU. In terms of the learnings, I think an important one is that this drug seems to be a strong activator of the innate immune system, and I think that's important because, thus far, many of the other potential approaches to activating innate immunity have been weak. So we do see things like fever and chills, myalgia, and puffy ankles. All What we did see, as I mentioned from the previous question, was hypotension and one episode of syncope. By splitting the dose, the hypotension events seem to have been mitigated substantially, and yet we still see the remainder of those on target toxicity. So our belief is that we have a strong innate activator that is at the same time safe, and of course, you'll know we have a large safety database behind the dog already at this point. So that's one important learning.
unknown: We haven't released any new data from the ASCO-GU or since the ASCO-GU presentation, but I would simply point out that on efficacy, I mean this is the 1B portion, this is fundamentally a safety study, the majority of patients were stable and had stable disease and remained on study at the time of that presentation. Can you just also give an update on the other trials going with 701? If there are proceeding with any issues due to COVID and just the latest update there and when we can see the next data set from 7-1-1. Sure, so I'll take Vimal. If I may, I'll take the triple combination study first. So I think, as we'd mentioned, the doublet of Benpeg on Nectar 214 plus Belimab has to be combined before we add in 701.
unknown: So that study is in the hands of Pfizer. Pfizer did announce maybe six weeks ago that they were pausing all trials. They announced approximately a week ago that they're reopening all trials. So that study was underway, the patients were enrolled, and it's now reopened again for accrual. So our anticipation is that, as we previously guided, we will add the 701 portion to that doublet once we have safety data later this year. So, that's the triple combination.
unknown: And then the MD Anderson study, and that's an important part of the development plan, as you know, that's in hot tumors, both in patients who are Keytrude nave and also patients who failed Keytrude or progressed through it. That study is open; it opened just at the turn of the year, and that's progressing. Again, consistent with guidance, we are anticipating being able to present data at the end of the year from my trial. Great, thank you.
[music].
unknown: The next question is from Du Kim of BMO Capital Markets. Please proceed with your question. Good morning.
unknown: Thanks for taking my questions. I was hoping you could review for us what would be considered clinically meaningful in terms of reduction in the serenity trials. And when you look at the ADDIS-EW studies, you saw a slightly benefit in bipolar patients versus placebo, then for schizophrenia. Is that something you also expect, or is it just within normal variability for agitation studies?
unknown: Thanks, Joe, for the question. I will ask Rob to answer that question about schizophrenia versus bipolar disorder.
unknown: Yeah.
unknown: So, Doe, the difference between the schizophrenia effect and the bipolar effect for antipsychotics tends to be relatively minimal. I realized that Atasupe had slightly better efficacy in bipolar patients than in schizophrenia. We're seeing from the 102 data, the phase 2 or the 8th dose trial that we did, we saw a clinical benefit, some improvement by CGI of one point equated to an improvement on the PANDIC auditory component score of around two to three points.
unknown: So we expect to see a similar effect for patients with bipolar disorder as for schizophrenia. It's possible that it'll be greater, but that remains to be seen. I could wax on about potential reasons why it might be more efficacious in bipolar than schizophrenia, having to do with, for example, the autonomic nervous system and relationship activation in bipolar patients who are agitated and manic or hypomanic. But we'll know a lot more once we have the actual data. So short answer, don't know if there will be a difference. It would be interesting, but we're not counting on that in terms of the powering of the trial.
unknown: And do you still see a negative three point reduction in the PEC score as the bar for clinically meaningful in either patient population?
unknown: Yes, it's about Yes, we were able to demonstrate that in the smaller study, and would you expect that That degree of change would represent a clinically meaningful improvement?
unknown: And could you also speak to the rising cases of delirium in hospital ER settings due to COVID-19 and whether you should actually start looking at a 501 study in that setting now versus later?
Vimal D. Mehta: Hello, this is Vimal. As there is a lot of press around like, you know, these COVID-19 patients who are elderly due to pre-intubation, as well as.
unknown: For more information, visit www.icliniq.com
unknown: Intubation or Mechanical Ventures use, they are getting delirium, and there are tons of reasons why they are getting it, but a lot of patients are getting it. So we continuously evaluate how we can help these patients, and we talk to our QPNA leaders and explore those opportunities. The Delirium program is already a strategic part of our development, so we are trying to speed that considering that this is a tremendous need in the hospital setting for treating the agitation resulting from delirium for the benefit of COVID-19 patients as well as for our own strategic reasons. So Rob, would you like to add anything to that?
unknown: I would just say that the API for BXCL501, dexmedetomidine, is already commonly used to treat delirium in the ICU setting. And so we do see that as a sort of low-hanging fruit, and we're exploring potential development options in that area.
unknown: Great. Thanks for taking the questions, guys.
unknown: Good morning. Thanks for taking my questions. First one on 501, and then I have a follow-up on 701. So we've seen some developments on the competitive side in the chronic management of agitation in Alzheimer's patients. How specifically do you expect to potentially bridge from the acute treatment of 501 to the chronic management side, if that's possible? And on 701, we were encouraged to see progress in the phase two efficacy part of the trial for TNEPC in combination with Keytruda. But could you give us a sense of what to expect in terms of the bar you might need to hit on efficacy and how to potentially compare single agent activity of 701 versus activity of the combo?
unknown: Thanks, Joe.
unknown: Thanks, Sumant. Regarding the 501 question related to... Can you please repeat the question? I just want to make sure we answer it right.
unknown: The next question is from Sumant Kulkarni of Canaccord. Please proceed with your question.
unknown: Sure, so the question specifically was we've seen some developments on the competitive side of chronic management of agitation in Alzheimer's patients. You have a product right now that is in development for acute agitation. How could that potentially be translated to maybe chronic management of agitation also in the longer run?
Vimal D. Mehta: Great question. I think we see that any success related to treating agitation in dementia slash Alzheimer's is a big problem. Currently, as you know, there are no treatments available to treat these patients. Under those circumstances... As we all know, neurological conditions, as they progress, the agitation episodes or spectrum start changing. You may be in a pre-agitation state, then you go to sporadic agitation, then subchronic agitation, and chronic agitation. So, any kind of treatment that can treat chronic agitation will be good because that can help reduce the number of episodes. But still, if you think about the agitation that's happening and the acute agitation that's still happening in these patients, and in the beginning, you have sporadic agitation, there's no need to use a chronic agitation when agitation can be treated with something that is an acute treatment as well as in sub-chronic.
Vimal D. Mehta: So, the way we think about this is that sporadic can be two episodes a month and do you really need a chronic agitation or it could be two episodes a week, still sub-chronic treatment will be sufficient, in the once it does become a chronic and there is a treatment that can be effective in reducing number of episodes but they still going to have breakout episodes so that needs where 501 will fit in as well as some of these chronic agitation drugs take quite a long time six to eight weeks to become effective and you need to manage the patient so we think that both 501 for acute treatment of agitation will fit in very well with any chronic agitation it will help in fact support and build the market if treatment is available for both kind of, So, did it answer your question or I'm happy to embellish more on it?
unknown: Oh no, that's great, thanks.
unknown: So regarding the 701 question, I will pass it on to Vince to describe what the bar is and what to expect from our Neuroendocrine Project.
unknown: Might the bar be, and how will we know the contribution of 701 or the individual contribution, right? So I'll take the latter question first.
unknown: And there has been a study with single-agent Keytruda. This was really why we did not include an internal Keytruda alone control arm in a similar, not identical, but similar population. And in that setting, this was a cancer-resistant prostate cancer group of patients who'd had treatment with ADT, the antigen blockers. And in that setting, the response rate is very low indeed; it's between 3 and 5 percent. So I think we have a reasonable handle on what Keytruda can do in this classic cold tumor. Given the fact that there is no standard of care here, we believe the response rate, provided it's durable, okay, and that's a feature, obviously, of immunoagents, a response rate in the teens, typically 15 percent or above, would be of interest to the field and actually to patients.
unknown: The next question is from Samir Devani of RX Securities. Please proceed with your question.
unknown: Hi, everyone. Thanks for taking my questions. It's good to see that the trials are on track, despite these challenging times. My question is actually about the delirium study you're planning. Looks like, again, coming back to dosing, looks like you're exploring 20 and 60 micrograms in that study. So I guess, could you perhaps just elaborate on how that compares to what's currently being used with the intravenous product? And also, whether there are any extra R&D costs associated with that program this year? Thanks very much.
Vimal D. Mehta: Samir, thanks for your question. Regarding the delirium, we have not spelled out what the plan is, or what doses we will be using, because that's still in the planning stage. Dementia, obviously, is on clinicaltrials.gov, and delirium is in the planning stage. Considering that there is such a big pandemic situation with COVID-19 and these patients are in need, we are kind of speeding up development. As part of our overall strategy, we always do a phase 1b slash 2. These trials don't tend to be that capital intensive, and we do them on a small number of patients, as you have seen with our schizophrenia and dementia, and these trials tend to be, as you know, enrolled rapidly and finish fast. So because of all those reasons, we don't see much impact on our clinical trial spend, but there will be some, and we are still in the planning stage. We will provide guidance once we have all the pieces come together, when this trial will start, and when we can provide the data readout on delay.
unknown: Thanks very much.
unknown: Thank you.
Vimal D. Mehta: There are no additional questions at this time. I would like to turn the call back to Vimal Mehta for closing remarks.
unknown: Thank you again for joining our call today. During the first quarter of 2020, we remain focused on advancing the development of our two clinical programs, BXCL501 and 701, with key data readouts occurring in the coming months. Have a great day, and please reach out to us if you have any additional questions. Thank you.
unknown: This concludes today's conference. You may disconnect your lines at this time. Thank you for your participation.
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