Q4 2021 BioXcel Therapeutics Inc Earnings Call
Operator: Good morning, and welcome to BioXcel Therapeutics' fourth quarter and full year 2022 financial results conference. At this time, all participants are in a listen-only mode. If during the conference, you require operator assistance, please call 1-800-637-8170. Please press star zero on your telephone keypad.
Good morning.
I'll come to biotech cell therapeutics fourth quarter and full year 2022 financial results conference call.
At this time all participants are in a listen only mode.
If during the conference you require operator assistance. Please press star zero on your telephone keypad.
Operator: After the presentation, there will be a question and answer session. If you would like to submit a question, you may press star 1 on your telephone keypad. Just to remind everyone, certain matters discussed in today's conference call and or answers that may be given to questions asked are forward-looking statements, subject to Risks and Uncertainties, related to future events and or the future financial performance of the company. However, actual results could differ materially from those anticipated in these forward-looking statements.
After the presentation there'll be a question and answer session. If you'd like to register a question you May Press Star one on your telephone keypad.
Just to remind everyone certain matters discussed in today's conference call and or answers that maybe given to questions asked are forward looking statements subject to risks and uncertainties related to future events and or the future financial performance of the company.
Actual results could differ materially from those anticipated in these forward looking statements.
Operator: 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 September 30, 2020, as will be updated by its annual report on Form 10-K for the year ended December 31st, 2020, which can be found at BioXcelTherapeutics.com or on SEC.gov. As a reminder, today's conference is being recorded. Joining us on today's call are Dr. Vimal Mehta, Chief Executive Officer; Richard Steinhart, Chief Financial Officer; Dr. Vince O'Neill, Chief Medical Officer; Dr. Frank Yocca, Chief Scientific Officer; Rob Risinger, Senior Vice President of Clinical Development, and Matt Wiley, Chief Commercial Officer. It is now my pleasure to turn the call over to Dr. Mehta, the CEO of BioXcel Therapeutics. Please go ahead.
Risk factors that may affect results are detailed in the Companys. 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 September 30th 2021 .
As will be updated by its annual report on Form 10-K for the year ended December 31st 2021.
Which can be found at biotech cell therapeutics dot com.
Or on S E C dot Gov.
As a reminder, today's conference is being recorded.
Joining us on today's call our Doctor of demo Mehta, Chief Executive Officer, Richard Steinhart, Chief Financial Officer, Dr. Vince O'neill, Chief Medical Officer.
Dr. Frank JAKO, Chief Scientific Officer.
Dr. Rob Risinger, senior Vice President of clinical development, and Matt Wiley Chief Commercial officer.
It is now my pleasure to turn the call over to Dr. <unk>.
Oh, a biotech cell therapeutics. Please go ahead.
Vimal Mehta: Thank you, Operator. Welcome, everyone, and thank you for joining our call today to discuss BioXcel Therapeutics' financial performance and business highlights for the fourth quarter and the full year of 2020. We have a lot to discuss as we embark on a potentially monumental year for the company, so let's get right into it. As you know, BioXcel Therapeutics is a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immunology.
Thank you operator, welcome everyone and thank you for joining our call today to discuss bias.
<unk> financial performance and business highlights for the fourth quarter and the full year of 2000.
We haven't locked to discuss as we embark on a potentially monumental year for the company.
Get right into it.
And you know, Mike said that had been taken as a clinical stage biopharmaceutical company.
Rising artificial intelligence approaches to transform everything that is in.
Zions element oncology.
Vimal Mehta: As we near our April 5th Paduva day for our lead asset, BXCL501, I would like to reflect on the significance of 2018. A landmark year for the company, numerous achievements across neuroscience and immune oncology, starting with BXCL501, which is currently under FDA review for the acute treatment of agitation associated with schizophrenia and bipolar disorders 1.0.
As we near our April 1st part of our day part our lead asset <unk> and fiber one I would like to reflect on the significance of 2020 months.
A landmark year for the company numerous that Jamie across neuroscience and immuno oncology franchise.
Starting with the XP and viable one which is currently.
The review for the acute treatment of agitation associated with schizophrenia and bipolar disorders.
Vimal Mehta: We capped an unprecedented journey in 2021, having regulatory submission in just under three years from first in human trials. We are not aware of any other AI-driven Doug Discovery and Development Company that has achieved this milestone as efficiently as we have. We view this impressive timeline as further validation of our AI platform and R&D capabilities. We are now less than one month away from potential approval of 501 as our first marketed drug. Since joining as our Chief Commercial Officer, Matt Wiley has led the development of an integrated commercial strategy to support our potential near and long-term commercial vision.
We kept an unprecedented Germany in 2000 and growing anyone having exactly where that is our mission and just under three from.
From Boston to human trials.
Are there any other AI do run drug discovery and development company that has achieved this milestone as I appreciate I really as we have.
We view this impressive timeline as part of the validation of our AI platform.
R&D.
We are now less than one month away from potential approval on fiber, one and plus marketed drug.
He is joining as our chief commercial officer, Matt Wiley had less need of London.
Integrated commercial strategy to support our potential in EMEA and long term commercial division.
Matthew Wiley: Including launch readiness activities and the build out of our national sales force, which is progressing as planned. We have focused on hiring sales representatives with extensive experience and are finalizing our marketing materials and market access and pricing strategies. Our Medical Science Liaison and Medical Managed Care Teams are actively educating Healthcare Providers and Pierce on Unmet Market needs to disrupt the treatment paradigm for their schizophrenia and bipolar patients suffering from ADHD. Our team participated in a number of emergency medicine and neuropsychiatric conferences around the country in 2021, with more events planned through the month.
Launch activity and the build out of our National sales force, which is progressing as planned.
We are focused on hiring preventive they presented data with our students save Expedia and finalizing our marketing materials and market access and pricing strategies.
Our medical science liaison and medical managed care teams are actively educating.
Health care providers and payers on unmet market needs.
The treatment paradigm for schizophrenia, and bipolar patients suffering from agitation.
Our team participated in a number of emergency medicine and neuropsychiatry conferences.
R&R currently in 2021 with more events planned in the months ahead.
Matthew Wiley: We have also seen positive reactions in market research that reflects broad enthusiasm for our proposed brand story and message platform. In a recent study of over 150 healthcare providers, 83% had a positive impression of BXCL501 after reviewing our blinded marketing material and stated they would prescribe BXCL501 for 40% of their schizophrenia and bipolar patients if approved. This prescribing intent for BXCL501 was shown to display its current standards of care broadly, including benzodiazepines, typical and atypical antipsychotics, and other prescription drugs.
We have also seen positive reaction in market research.
Reflects broad enthusiasm for our proposed brand story and message platform.
In a recent study of 150 health care providers.
T part of thing.
Alright, a positive impression <unk> five or one after reviewing our blinded marketing material and it stated they would prescribe <unk> quite well one for 40% of their schizophrenia and bipolar patients if approved.
This prescribing and Danny for <unk> 501 was shown to display its current standards of care broadly, including benzodiazepines. It got.
It could become anti psychotic and other prescription treatment.
Vimal Mehta: If approved, 5.0.1 would be the first new therapy in nearly a decade for these indications, and we are encouraged by the widespread recognition of its potential benefit to patients and care. Beyond potential approval of 501, we remain committed to our three-pillar portfolio expansion strategy, which includes... Indication expansion, extending our geographic reach, and growing the medical setting where 501 is offered. First, regarding our Indication Expansion Strategy 501 represents a pipeline within a product, and we are actively pursuing new indications representing significant future growth opportunities.
If approved fiber one would be the first new therapy.
In nearly a decade for these indications.
By the wide spread recognition of potential benefits to patients and caregivers.
Beyond potential approval on fiber one.
We remain committed to our three pillar portfolio expansion strategy, which includes indication expansion extending our geography.
And growing the medical side, we have 501 is offered.
First regarding how about indication expansion has started the 501 presented a pipeline within a product and we are actively pursuing new indications representing significant future growth opportunities.
Vimal Mehta: This past December, we initiated a phase 3 program evaluating 501 for the acute treatment of agitation in patients with Alzheimer's disease. This program consists of two trials, tranquility to, for patients in assisted living or residential care, and Pragility 3, Populations in Nursing. [inaudible] With an estimated 100 million episodes of education associated with Alzheimer's in the US every year, we believe 501 has the ability to address this higher, high unmet medical need and would mark the first approved therapy in this indication, if possible.
This part of the December we initiated a phase III program evaluating <unk> hundred one for the acute treatment of agitation in patients with Alzheimer's disease. This program consists of two trials.
We can do for patients and assisted living or residential facility.
<unk> three for patients and not singles.
S T music hardenburg million episodes of agitation associated with Alzheimer's in the U S. Every year. We believe 501 has the ability to address these high it high unmet medical need and would mark the first approved therapy in this indication.
Vimal Mehta: We look forward to advancing this potential novel treatment option for the growing population of Alzheimer's patients. We are also expanding 501 into new indications and have recently fired an IND for 501 as a potential adjunctive treatment for major depressive disorders. MDD remains the most common type of depression in the U.S., with approximately 27 million cases a year.
We look forward to advancing this potential novel treatment option for the growing population of Alzheimer's patients.
We are also expanding fiber one into new indications and have recently widened Indy 501, as a potential adjunctive treatment in major.
Depressive disorder.
MDT remains the most common type of depression in the U S with approximately $27 million came through the year.
Vimal Mehta: There are over 30... 300 million anti-depression prescriptions filed annually with limitations to the statement such as low onset of action and incomplete responses. We believe that our own clinical trial suggests PXL501 could be an effective treatment option for the Moving to our second pillar, geographic expansion, we expect to submit a marketing authorization application with the European Medicine Agency in the first half of this year for PXCL 5. With a sizable market opportunity similar to the US, we are working diligently to grow our footprint and reach more potential patients who could benefit from our novel therapy. As part of this strategy, we are actively seeking a partner to potentially commercialize fiber.
They're out of what 30 300 million anti depression.
Sure. This is Greg annually.
Limitation to the statement such as slow onset of action and incomplete responses.
And believe that our own clinical data suggest.
<unk> hundred one could be an effective treatment option for these patients.
Moving to our second pillar geography expansion.
Expect to submit a marketing authorization application with the European Medicines agency in the first half of this year well be next Gen firewall.
With a sizable market opportunity similar to the U S. We are working diligently to grow our footprint and each more potential patients who could benefit from our novel product.
As part of this strategy, we are actively seeking a partner to potentially commercialize valuable ones.
Vimal Mehta: Our third strategy pillar in our land and expand strategy. We want 501 to ultimately be accessible to patients regardless of treatment setting. Rounding out our growth strategy, we continue to evaluate and pursue expansion opportunities into new settings of care for both current and future Indians. As part of our strategy for a sustainable R&D pipeline and using our AI platform, we have expanded our pipeline with our newest product candidate, BioXcel 502. We initiated Formulation for BioXcel 502, which is designed to be a potent and selective antagonist for a GPCR target affecting serotonergic signaling in the cerebral cortex.
Our third strategic pillar.
In our land and expand strategy.
I mean, one five or one two R&D, mainly be accessible to patients regardless of treatment setting.
Rounding out our growth strategy, we continue to evaluate and pursue expansion opportunities into new settings of care for both current and future indications.
As part of our strategy.
<unk> R&D pipeline.
And using our AI platform, we have expanded our pipeline with our newest product candidate VX Seattle firewood.
We initiated formulation for me a good five or two for the chronic treatment of agitation in dementia patients.
Fiber.
Designed to be a potent selective <unk> party GPC, our target affecting shattered on object signaling episodic railcar tanks.
Vimal Mehta: A Differentiated and Novel Mehta, Let me now turn to our Immuno-Oncology franchise, where we continue to make tremendous progress advancing the 501 program. As a reminder, 701 is an orally administered systemic activator of innate immunity being developed for the treatment of aggressive forms of prostate cancer and advanced solidity. In February, we presented positive data in both metastatic castrate-resistant prostate cancer, MCRPC, patient cohorts at ESCO-GU.
Differentiated and novel mechanism.
Let me now turn to our immuno oncology franchise, we continue to make tremendous progress advancing <unk> hundred one program.
And then remind us.
So I don't know one is an orally administered systemic activator of innate immunity being download for the treatment of aggressive forms of prostate cancer.
<unk> advanced solid tumors.
In February we presented positive data in both maritime.
Castrate resistant prostate cancer M CIBC patient cohorts at <unk>.
Vimal Mehta: BioXcel 701 plus Keytruda demonstrated encouraging composite, Pawns Rates of 21% for Adinokarcinoma patients and 33% for SCNs. Together, the results support 501 potential to serve as a differentiated approach for MCRPC patients and possibly extend Checkpoint Inhibitor Therapy into heart-to-treat cancer. We also expect additional advocacy data from the MD Inversion-led Open Label Phase 2 BASQA trial of 701 and Ketruda for advanced solid tumors in the second half of 2022 and continue to be encouraged by the potential of 701.
<unk> 701, plus Keytruda that master data has got anything composite disc.
Bonds, there so Tony one part I didn't know carcinoma patients and 33% for Etsy NSE patients.
Together these results support fiber one potential to serve as a differentiated approach for <unk> patients and possibly extending to checkpoint inhibitor therapy into hard to treat.
Got it.
We also expect additional aggregating data from the MD Anderson open label Phase II basket trial of 701, and Keytruda for advanced solid tumors in the second half of 2022 and continue to be encouraged by the potential of seven.
Vimal Mehta: To summarize, our AI platform continues to drive our innovation. Starting with our two lead product candidates, both have shown promising results through clinical development, including publication in prestigious journals such as JAMA and JITSI, and we couldn't be more excited for the future of our neuro- and oncology field. 2021 was a critical year for BioXcel Therapeutics as we hit many, three milestones on our way towards the potential first product approval.
To summarize our AI platform continues to drive our innovation starting with our two lead product candidates, both have shown promising clinical development, including publication in prestige as Jeremy.
Such a jamba and you'd see and we couldnt be more excited for the future of our neuro and oncology franchises.
2021, while the critical year, whereby it comes out of your days as we hit many key milestones on our way towards the potential plus product approval.
Richard Steinhart: 2022 is shaping up to be a busy and exciting year as we look to build on our many recent successes. Now, I would like to turn the call over to Richard, who will give a financial update. Thank you, Vimal. I will now review our fourth quarter and full year 2021 financial results. Research and development expenses were $12.5 million for the fourth quarter of 2021, compared to $11.4 million for the same period in 2020. The increased expenses were primarily attributable to increased headcount and related costs during a three-month period in 2021.
2022 is shaping up to be a busy and exciting year as we look to build on our many disinfectant.
Richard Steinhart: Research and development expenses were $52.7 million for the full year 2021, as compared to $58 million for the same period in 2020. The decrease for the year ended December 31, 2021, was primarily attributable to decreased clinical trial expenses related to BXCL501, which were partially offset by increased trial expenses related to BXCL 701 clinical trial. General administrative expenses were $13.6 million for the fourth quarter of 2021, as compared to $9.7 million for the same period in 2020.
Now I would like to turn the call over to Richard who will give you a financial update. Thank you limo I will now review, our fourth quarter and full year 2021 financial results.
Richard Steinhart: The increase was primarily due to increased headcount and related costs, including higher stock base compensation, increased marketing and commercial costs related to the potential launch of BXEL 501 in the United States, as well as increased legal and professional fees and insurance costs. General and administrative expenses were $54.2 million for the full year 2021, as compared to $24.3 million for the same period in 2020.
Research and development expenses were $12 $5 million for the fourth quarter of 2021 compared to $11 4 million for the same period in 2020.
The increased expenses were primarily attributable to increased head count and related costs. During the three month period of 2021.
Research and development expenses were $52 $7 million for the full year 2021, as compared to 58 million for the same period in 2020.
The decrease for the year ended December 31, 2021 was primarily attributable to decreased clinical trial expenses related to <unk> 501, which were partially offset by increased trial expenses related to <unk> 701 clinical trials.
General and administrative expenses were $13 6 million for the fourth quarter of 2021.
As compared to $9 7 million for the same period in 2020.
The increase was primarily due to increased head count and related costs, including higher stock based compensation increased marketing and commercial costs related to the potential launch of <unk> five and one in the United States.
As well as increased legal and professional fees and insurance costs.
General and administrative expenses were $54 2 million for the for the full year 2021, as compared to $24 3 million for the same period in 2020.
The increase for the year ended December 31, 2021 was primarily attributable to increased head count and associated costs, including noncash stock based compensation.
We also incurred significant commercial costs in preparation for potential U S launch a bx L 501.
Richard Steinhart: The increase for the year ended December 31, 2021, was primarily attributable to increased headcount and associated costs, including non-cash stock-based compensation. We also encourage significant commercial costs in preparation for the potential U.S. launch of BXEL 501. BioXcel Therapeutics reported a net loss of $26.1 million for the fourth quarter of 2021 compared to a net loss of $21.1 million for the same period in 2020. For the full year, BioXcel reported a net loss of $106.9 million compared to a net loss of $82.2 million for the same period in 2020. As of December 31, 2021, cash and cash equivalents totaled approximately $233 million.
Well it looks out therapeutics reported a net loss of $26 1 million for the fourth quarter of 2021.
Paired to a net loss of $21 1 million for the same period in 2020.
For the full year <unk> reported a net loss of $106 $9 million compared to a net loss of $82 $2 million for the same period in 2020.
As of December 31, 2021, cash and cash equivalents totaled approximately $233 million.
Vimal Mehta: Now I'd like to turn the call back to Vimal. Thank you, Richard. We would now like to open the call for questions. Operator.
Now I'd like to turn the call back to them.
Thank you Richard we would now like to open the call for questions operator.
Thank you.
Operator: Thank you. At this time, we will 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 question. You may press star 2 if you would like to remove your question. For participants using speaker equipment, it may be necessary to pick up your handset before pressing start.
At this time, we'll be conducting a question and answer session.
If you would like to ask a question. Please press star one on your telephone keypad.
Confirmation tone will indicate your line is in the question queue.
You May press Star two 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 star keys.
Operator: One moment, please, while we pull for questions. Our first question today is from Greg Harrison of Bank of America. Please proceed with your question. Hey, good morning, and thanks for taking our question. So now that you've got 501 Padufas right around the corner, are you in label discussions currently, and how would you characterize them so far? Good morning, Greg.
One moment, please while we poll for questions.
Our first question today is from Greg Harrison of Bank of America. Please proceed with your question.
Hey, good morning, and thanks for taking our question.
So now that the 501 producers right around the corner.
Are you in label discussions currently and how would you characterize them so far.
Good morning, Greg.
Vimal Mehta: Our NDA remains under review by the FDA, and we continue to expect a response to our NDA by the April fiscal year. Got it. And then, if I could hit on a different topic, you know, what is the status of any discussions with partners to commercialize 501 in the EU? And what attributes are you looking for in a future partner?
Our NDA remains under review by the FDA and we continue to expect the response of our NDA by the April period.
Got it.
Then.
If I could hit.
You hit on a different topic.
What is the status of that.
Any discussions with partners to commercialize <unk> hundred one in EU and.
What attributes are you looking for in a future partner.
Vimal Mehta: So, as we mentioned, we are getting ready to submit an MA in the first half of this year. That will also facilitate our discussions with our partners, and we are just holding on to submitting the MA unless we are at a point where we have got the approval from the FDA. And the attributes we are looking for in a partner are that who can be a very strategic partner in terms of not only our first two indications, which are schizophrenia and bipolar, but also for our upcoming SNDAs in Alzheimer's and MDD. So if a partner has a neuropsychiatric franchise and they have those kind of capabilities, those are the key attributes we will be seeking in a partner. Great. Thanks a lot. Thank you, Graig.
So as we mentioned that we are getting ready to submit the MAA.
In first half of this year.
That will also facilitate our discussions with our partners and we are.
Just holding on to submitting I mean, unless we get unless we got to a point, where we have got the approval from the FDA.
And the attributes we're looking in that partner is that who can be a very strategic partner in terms of not only our first two indications, which is in schizophrenia and bipolar but also for our upcoming S. N D as in Alzheimer's and MDT. So if a partner that has the noodles that got big franchise and they have those kind of kept.
Those are the key attributes we will be seeking a partner.
Great. Thanks, a lot.
Thank you Greg.
Yes.
Operator: The next question is from Chris Howerton of Jeff. Please proceed with your question. Great.
The next question is from Chris Howerton of Jefferies. Please proceed with your question.
Unknown Attendee: Thanks so much for taking the questions, and I'm super excited to have the PDUFA just about a month away. So for the, I guess the question that I would have with respect to the EU, you know, what indication do you have from the EMEA that the current data package would be acceptable for submission? My recollection is that there was an extensive amount of European patients that were actually evaluated in the pivotal studies. So maybe just a little clarification on that.
Great. Thanks, so much for taking the questions and Super excited to have the Paducah, just about a month away.
So for the I guess the question that I would have with respect to the E U.
What indication do you have that from the EMEA that the current data package would be acceptable.
<unk> submission I just my recollection was there was an extensive amount of European patients. They were actually evaluated in the pivotal studies. So maybe just a little clarity on that.
Vimal Mehta: And then the second question I would have would be, you know, obviously some early encouraging results on 701, but, you know, what is the decision-making process moving forward in terms of deploying capital towards those programs versus expanding 501? And then the third question, if you'll allow me to sneak it in, I think it's a quickie, is for 502, you know, you said you're in the process of formulating work. Could you just tell us about the expected timelines for that and what the final dosage form that you're expecting to have? Thank you. Thank you, Chris.
And then the second question I would have would be obviously some early encouraging results on 701.
But you know what.
What is the decision, making moving forward in terms of deploying capital towards those programs.
<unk> is expanding fiber one.
And then the third question if you'll allow me to sneak it in I think it's a quickie is four or five O. Two you said you're in the process of formulation work could.
Could you just tell us about the expected timelines for that and what the final dosage form that youre expecting to have thank you.
Vimal Mehta: I have noted down all three questions. I will try to go through them one by one. Regarding your question about what package is required for the MA, we had extensive discussions with the authorities, European authorities, and we presented the package that we have presented to FDA, and they were aligned with the data we have currently and the package we have that is sufficient to submit the MA in Europe. Primarily, all our patients in our Serenity 1 and 2 trials were from the U.S., not from Europe, just to clarify that. But the agency is comfortable using the current package to submit the MAs.
Thank you Chris.
All three questions I will try to go through them one by one regarding your cushion that what package is required for the Ami we had extensive discussions with the authority European authority.
And we presented that back and that we have presented to FDA and they would align that data. We have currently and the package. We have that is sufficient to submit them in Europe .
Primarily all of our patients.
In our city near D. One Sydney do you run into drive waterfront U S. Not from Europe , just to clarify on that.
But agencies comfortable in using the current package was that some of it than me.
Vimal Mehta: Coming to the second question. Yeah, go ahead. Coming to your second question regarding the 701, we have already outlined our five-year vision for BioXcel Therapeutics. We want to be a leading neuroscience company, AI-enabled neuroscience company, and we will continue to focus and deploy our resources from AI all the way to the commercialization in the neuroscience space, and that's the commitment we have made, and we have the most advanced product, which is a pipeline within a product, with first two indications very close to the NDA PDUFA date, and then Alzheimer's in Pivotal Phase III program, and then we will be initiating the MDD program shortly.
Coming to the Tech English Yeah go ahead.
Coming to your second question regarding the 701.
We have already outlined our five year vision for buying cell therapeutics, we wanted to be a leading neuroscience company and everybody noticed things company.
And we will continue to focus and deploy our resources.
<unk> all the way to the commercialization in the neuroscience space and that's the commitment we have made and we have the most advanced product, which is a pipeline within a product with first two indications very close to the NBA Purdue holiday and then as I might've seen pivotal phase III program and then we will be in each year.
During the <unk>.
<unk> programs shortly.
Vimal Mehta: So that commitment has already been made in terms of the 7-0-1. Now we have a clinical proof-of-concept in hard-to-treat tumors, so we will continue to explore strategic options, which could include, like you know, potentially a spin-out, it could be a partnership, it could be a JV, and or co-development. We truly believe in the mechanism of 701, and we think we have proven the following things. We have demonstrated that it is safe and tolerable. It can be combined with Ketruda, and we have demonstrated clinical activity. We have published the mechanism in JITSI.
So that commitment has already been made in terms of that seven O. One now we have a clinical proof of concept.
Hard to treat tumors. So we will continue to explore strategic options.
Which could include like you know potentially a spin out it could be a partnering it could be a JV.
And our core development, we truly believe in the mechanism of 701, and we think we have proven following thing we have demonstrated that it is safe and tolerable. It can be combined with Keytruda. We have demonstrated clinical activity. We have published that mechanism in <unk>. So we have achieved.
Vimal Mehta: So we have achieved a lot with 701, and as just to remind everyone, when we became a public company in March of 2018, we selected these two assets, BioXcel 501 and 701. One of them is very close to approval, and another one has demonstrated cleaner proof of concept. So we are super excited about both the assets we have in our portfolio. And I will now go for the 502 formulation. Formulation work has recently begun in the last several months.
With that one on one and add.
Just to remind everyone. When we became a public company in March of 2018, we had selected <unk> five one and 701 one of them is very close to approval and another one has demonstrated clinical proof of concept. So we are super excited for both the assets we have in our portfolio.
Yeah.
And I will now go for the fiber to formulation formulation Wildcats are recently begun in the last several months. So we will develop in timelines that word our timelines on what best we need to do it is going to be an oral therapy and once we have all of that work done.
Vimal Mehta: So we will be developing timelines, that what our timelines are, what tests we need to do. It is going to be oral therapy. And once we have all of that worked out, we'll provide clarity on our timeline to enter the clinic. But we are getting to that point, and then we will be in that position to provide in 2020.
We'll provide clarity on our timeline to enter the clinic, but.
We are getting to that point, then we will be in that position to providing 2022.
Vimal Mehta: Alright, well, thank you Vimal, I really appreciate the answers and thanks for writing down all three; I appreciate it. Okay, thank you, Chris.
Okay.
Alright, well. Thank you for them all I really appreciate the answers and thanks for writing down all three I appreciate it.
Okay. Thank you Chris.
Operator: The next question is from Robyn Karnauskas of Truist Securities. Please proceed with your question. Hi guys.
The next question is from Robin <unk> of <unk> Securities. Please proceed with your question.
Robyn Karnauskas: Thank you for taking my question. I have two questions for you. So, I'm tranquillity questions one and two. Are you planning on reading them at the same time as you saw through?
Hey, guys. Thanks for taking my question two questions for you on shrink quality.
Are you planning on waiting it out at the same time, if he talked through.
How long would be reading out and how you might disclose the data.
And then I have a follow up.
Robyn Karnauskas: How would you be reading out and how you might disclose the data? And then I have a follow-up. Sure. Great question, Robyn. As you know, we initiated this program, got alignment with the FDA, and initiated the program in December. And at that point in time, Omicron was almost at its peak.
Sure Great question Robin we as you know we initiated this program got alignment with FDA initiated the program in December and at that point in time Army ground was almost at the peak. So we started opening clinical sites. Because then there is some.
Vimal Mehta: So we started opening our clinical sites because when there is some COVID or Omicron situation, access can sometimes be limited to the elderly care centers. So we now have very good clarity going forward about how many sites we need. As you know, we have 150 patients in each trial. So it's not a big ask.
Overdog I'll make around saturation access can sometimes be limiting to the elderly care center. So we now.
Have a very good clarity going forward that how many sides. We need as you know we have 150 patients in each trial. So it's not a big <expletive> . So we adjusted determining how many sides, we need and how are we going to stagger to these two programs. So tangled wreck two will be the first one that we will have the data read out.
Vimal Mehta: So we are just determining how many sites we need and how we begin to stagger these two programs. So Tranquility 2 will be the first one that we will have data readout from, followed by data readout from Tranquility 3. But as you can imagine, these trials are conducted in elderly care centers, residential facilities, and nursing homes. And in some of these places, both the facilities are in the same building.
Followed by a data readout from their tank related but as you can imagine these size I've conducted in elderly care and then residential facilities and nursing home and some of these places all both facilities are in the same building. So these sites will be staggered.
Vimal Mehta: So these trials will be staggered, and data readout will come out in a staggered fashion for the Tranquility program. And a follow-up there, I guess, obviously people ask about, like, this label discussions for your initial vacation, but when you think about living facility versus, you know, nursing homes, and for those that are not in the same center, are there any differences in the type of patients that you're dealing with, like one population more miles than the other?
And they don't they don't will come out in a staggered fashion for the tranquility program.
And a follow up there I guess.
I mean, some people asked about like label discussions for your initial vacation, but when you think about living.
Something versus nursing homes.
And for those that are not in the same center are there any differences in the type of patients that you're dealing with I think one population more mild than the other so should we be thinking that there's differences in other datasets that will be seen.
Vimal Mehta: So we should be thinking that there are differences in the data sets that we'll be seeing. Let me pause this question for Rob so he can describe what the patient populations are, what kind of Alzheimer's patients are in these two centers. Yeah, the patient. The fundamental difference between someone who can live by themselves with minimal supervision and someone who requires monitoring 24-7 a day has to do with their ability to sort of complete activities of daily life, as we call it in medicine. So we do not believe that there's any substantial difference in terms of efficacy, however; we want to demonstrate efficacy across the entire spectrum of care for patients with dementia who become agitated. I got it.
Oh, let me parse this cushion to Rob so he can describe what that patient population size, what kind of president Ms patients out in these two centers the patient, but the fundamental difference between someone who can live by themselves with minimal supervision and someone who requires monitoring 24, 7%.
Robert Risinger: Okay, great. Thank you. Thanks Robyn. The next question comes from Sumant Kulkarni of Canaccord.
Today has to do with their ability to sort of complete activities of daily life as we call it in medicine.
So we do not believe that there is any substantial difference in terms of the efficacy. However, we want to demonstrate efficacy across the entire spectrum of care for patients with dementia, who become agitated.
Got it okay, great. Thank you.
Thanks Robyn.
Okay.
Operator: Please proceed with your question. Morning, thanks for taking my questions. I have three.
The next question comes from <unk> Kulkarni of Canaccord. Please proceed with your question.
Good morning, Thanks for taking my questions I have three I'll start with one of the tranquility trial.
Sumant Kulkarni: I'll start with one on the Tranquility Trial. We saw clinicaltrials.gov had an update posted as of yesterday on one of the phase three trials that has a primary and study completion date of December 2022 and for a maximum of 28 doses over 12 weeks. Could you confirm the maximum number of doses, which appears to be more than we thought it might be? And given the update was just posted yesterday in light of your Omicron comments, does that December 22 completion date reflect your latest expectations now that Omicron seems to be behind us? That's my first question.
We saw clinical trials dot Gov.
Posted as of yesterday and one of the phase three trials.
The study completion date of December 22, and put a maximum of 28 doses over 12 weeks could you confirm the maximum number of billions, which appears to be more than what we thought it might be and given the update with just posted yesterday in light of your comments does that December completion date to reflect our latest expectations now that seem to be behind us.
Question.
Two.
Vimal Mehta: Sumant, I will take the first question, and I will pass it on to Rob about the other portions of the question. We have not provided guidance on when the data readout will be, but as I indicated earlier, there are only 150 patients, so it's not a big trial from a patient enrollment perspective. We just want to be sure that we have a sufficient number of sites open; we want to be sure that we can have enrollment, and we can complete the trial. There are two key pieces.
I will take the first question and I will pass it on to Rob about the other portions of the question.
We have not provided the guidance that.
Then like no data readout will be but as I indicated earlier that there are only 150 patients. So it's not a big trial from me.
Enrollment perspective, we just wanted to be sure that we have sufficient number of sites open but we wanted to be sure that we can have that enrollment and we can complete the trial like you know there are two key pieces. One is plus these acute agitation and then as you said or whether it's the P. D. A.
Vimal Mehta: One is, first, acute agitation, and then, as you said, over a three-year period, testing up to 28 doses to see what the repeat dose effect is. As you know, in Alzheimer's, these patients have multiple episodes, so we are trying to capture not only the acute, but the intermittent chronic when they have multiple episodes, so that when we go out, our SND has a broader label. So with that background, I will pass it on to Rob.
Testing up to 28 doses to see what the repeat dose effect is as you know in Alzheimer's.
These patients have multiple episodes. So we are trying to capture not only the acute we are trying to capture the intermittent chronic when they have multiple episodes. So that when we go out our S. N. Do you have a broader label so with that background I will pass it onto Rob Rob.
Yeah I think.
Robert Risinger: The trial completion date will entirely depend upon our ability to enroll the 150 patient subjects. Now no one predicted the pandemic before it began, and it seems people have difficulty predicting the waves of Omicron, and who knows what the next Greek letter is, but that has the potential to slow down enrollment, and that's precisely why we have not given numbers and dates for our readout yet until we have a clear way of moving forward, and we have a bead, solid date on when the final patient will be dosed in that trial.
Uh huh.
Trial completion date will entirely depend.
Upon our ability to enroll the 150 patient subjects now no one predicted the pandemic before it began and it seems people have difficulty predicting the waves of omicron and who knows what the next Greek letter is but.
That has the potential to slow down enrollment and that's precisely why we have not given numbers and dates for our readout yet until we have accrual moving forward and we have a bead.
And solid data on when the final patient will be dosed in that trial. So we have great confidence. After all we initiated trial as omicron was increasing and soon we'll be able to report.
Robert Risinger: So we have great confidence; after all, we initiated the trial as Omicron was increasing, and soon we'll be able to report accrual and enrollment rates and much better information. You cited a market research survey that you conducted. I want to focus a little bit on the little bit of the class that is not full, which is 17% of survey participants that did not seem to have a positive response on 501.
Accrual in enrollment rates and much better information.
Got it and my second question, you cited market they'll be able to conduct it.
I wanted to focus on the little bit of a glass that is not well.
Which is a 17% 17, 5%.
Not seem to have a positive response on <unk> what are the key points that it looks like it's been solid.
Impediments to their use of the product.
On the 40% describing what anybody.
Okay.
Matthew Wiley: What were the key points that those participants saw as impediments to their use of the product? And on the 40% prescribing, what delivery setting was assumed? So, Sumant, thank you for the question; Matt will take this. Yeah, so there was not any qualitative feedback on the 17%.
Uh huh.
Thank you for the question.
Matt will take this question, yes. So there was not qualitative feedback on the 17%. This was just a quantitative survey or part of the survey.
Matthew Wiley: This was just a quantitative survey, or part of the survey, where we found 83% had a positive reaction. So we did not dig in qualitatively on the remaining 17%, but that's something we'll find out over time. How about the prescribing setting?
Where we found the 83% had a positive reaction. So we did not digging qualitatively on the on the remaining 17%, but that's something we'll find over time.
Matthew Wiley: And then I'll ask my last question too, which is 701, what are your latest plans to ascertain single agent activity? So, in relation to the prescribing setting, we would expect our base case to be in the hospital and institutional setting. That's what we're building out the sales team to focus on at. So, Sumant, I would say we are a few weeks away from understanding what our label will look like, and we will have clarity, but as Matt said, our base case has been that, you know, under the supervision of a healthcare provider. Coming back to the single agent activity for 701, let me pass this question to Vince. Thanks, Vimal. So morning, Sumant.
Yeah.
How about the prescribing setting and then I'll ask my last question do which is 701 what are your latest plans drive had been single agent activity.
Okay.
So in relation to the prescribing setting we would expect our base case is in the hospital and institution type setting.
That's what we're building out the sales team to.
To focus on at launch.
Got it.
So I would say, we a few weeks away.
From understanding what our label will look like and we will have that clarity but has.
Matt said, our base case has been under the supervision of a health care provider.
Coming back to the single agent activity for 701, let me.
Pause this question to Vince Vince. Thanks, then the most mornings month, yes, that's exactly the logical next step Brian as we've previously said, we would need to develop.
Vimal Mehta: Yeah, that's exactly the logical next step, right? As we previously said, we would need to develop, or as part of the development plan, test 701 single agent. So at the ASCO EU 2022 presentation, we did announce that we will proceed with a randomization portion for adenocarcinoma. So that is the next step. Thank you.
As part of the development plan tests, then suddenly one single agent. So <unk> M. U 2022 presentation. We did announce that we will proceed into randomization portion for adenocarcinoma. So that that is the next step.
Got it thank you.
Vince O'Neill: The next question comes from Yatin Suneja of Guggenheim Partners. Please proceed with your question. So yeah, good morning, it's Eddie Onprey out.
The next question from comes from it yet <unk> of Guggenheim Partners. Please proceed with your question.
Operator: Thanks for taking my questions and congrats on all the progress. Just a few from us, maybe from Matt. Approval in April. How should we think about the first year? [inaudible] Transcripts provided by Transcription Outsourcing, LLC. Alonso, Rating Factor, Raghuram Selvaraju, Raghuram Selvaraju, So we have identified a number of key performance indicators that we're gonna be tracking internally. And so, what we will do is, in future communications with investors, provide some of those that are relevant.
Yeah. Good morning, it's Eddie on for <unk>. Thanks for taking my questions and congrats on all the progress just a few from US maybe for Matt assuming approval in.
In April how should we think about the first year of launch from both a script number and a revenue perspective and can you talk about what metrics you'll be providing in the first few quarters to help us track.
The launch and what do you see as sort of the key gating factor for that trajectory.
So we are.
We have identified a number of key performance indicators that we're going to be tracking internally and so what we will do is in future communications with investors I'll provide some of those that are relevant.
Got you and then can you talk about what.
Operator: Can you talk about what the average number of agitation episodes is for these patients and sort of how often those episodes bring patients to a care center where 501 would be administered, just in terms of how often. So, as we have indicated, these patients get about a dozen episodes a year, and they end up in the emergency rooms.
What's the average number of agitation episode does for these patients and sort of how often do those episodes bring the patients to a care center, where 501 would be administered just in terms of like how often.
There is a chance for this drug to be used.
So Eddie as we have indicated these patients get about a dozen episodes a year and they end up in the emergency rooms.
Vimal Mehta: That's kind of on average, and every market research we have done, that tends to be the case, where patients basically end up in the emergency room out of 25 million episodes, and one-third get treated at home. And the reason for that dynamic is that there is no current treatment option that patients can take at home. So whenever there is an episode and it escalates, they end up in the emergency room, or they could go, even if they go to their neuropsychiatric physician or go to any of the critical care centers, they send them to the emergency room because not much is available in terms of therapy.
It's kind of been on an average and every market research we have done that.
To be decades, two thirds.
Of the patients basically end up in the emergency rooms out of 25 million episodes.
And one third get treated at home and the reason for that dynamics is that there is no current treatment options that the patients can vacate at home. So whenever there is episode and escalate they end up in the emergency room or they could go even if they go to the neuro psychiatric physician.
They go to any of the critical care centers.
Send them to the emergency room, because not much is available in terms of the therapeutic option.
Vimal Mehta: And our goal with 501 will be to change these dynamics over a period of time so that when they come into the emergency room, they get treated with 501. And as we move, I have always said we have a strategic plan and vision to expand the medical setting for this program. As we move this into the community and home care setting, this dynamic of the patient will change, how many go to the hospital setting and how many are treated at home. So that's pretty much what we know today that there are a lot of episodes, about 25 million episodes for about 7 million people with schizophrenia and bipolar disorder.
And our goal with 501 will be how to change these dynamics over a period of time when they are coming in the emergency room, they get to you David.
101, and as we move.
I have always said, we have a strategic plan and vision to expand the Americas setting for this program as we move this.
Into the community and homecare setting these dynamics of the patient will change how many go to the hospital setting and how many do you do that but that's pretty much. What we know today that there are lot of episodes of about $25 million episode for about $7 million with schizophrenia and bipolar patients.
Thank you.
Yes.
Operator: The next question comes from Corinne Jenkins of Goldman Sachs. Please proceed with your question. Hi, this is Minds Beyond Booker, and I'm here for the Q&A question.
The next question comes from Corinne Jenkins of Goldman Sachs. Please proceed with your question.
Unknown Attendee: First, what is Kevin T's most focused on with respect to the launch preparation inside the one? And secondly, expect any news in the Alzheimer's setting? Thank you for your question. As we have indicated, we are waiting for approval once we have a full understanding of our label, what our label is.
Hi, This is Mike.
Thank you for taking my question.
Waddington.
John did you say could be launched with teaching and hydro one I'm thinking do you expect any.
Yes, I know it's lumpy.
Okay.
Vimal Mehta: Then we will be able to provide what our launch settings are, but as we have said, our trial was conducted under the supervision of a healthcare provider. So if that's the condition, we have prepared to launch. And we will continue to explore additional settings as we go and expand the potential of 50. Did I answer your question?
Thank you for your question as we have indicated we are waiting for the approval once we haven't fallen best training about our label what I would label. It then we will be able to provide what our launch.
I think that but as we have said that like you know our trial was conducted in a.
Under the supervision of a health.
Health care provider. So if that's the condition we are prepared to launch it.
And we will continue to explore additional settings, as we grow and expand the potential of 501.
Did I answer your question.
Vimal Mehta: Yes, I also wanted to ask, in terms of your interpretation, what are the things that you're most focused on right now? I think we were ready to launch the product in January for our original PDUFA date, and we are, in fact, better prepared to launch now. So all the provisions that are needed in terms of having recruitment and build-out of the national sales force, market access, pricing strategies, and marketing materials that have been built so that the sales force is ready to go.
Yes.
I wanted to ask one inbound.
What I'm most focused on.
No.
I think we were ready to launch the product in January .
For our original per DAU per day, and we are in fact better prepare to launch now so all of that provision that are needed in terms of having a recruitment and build out of our national sales force.
Market access pricing strategies.
Marketing materials that have been like you know Barry for that sales force being ready to go so we feel very.
Vimal Mehta: So we feel very good and confident that we have all the pieces in place to launch this product and his children. Thank you. The next question comes from Colin Bristow of UBS. Please proceed with your question.
Good and confident that we have all the pieces in place to launch these products.
Understood.
Yeah.
The next question comes from Colin Bristow of UBS. Please proceed with your question.
Operator: I want to push you guys on something. So, it sounds like you do not yet have a label in hand. I was just wondering, you know, has FDA set an expectation specifically of when this label will arrive? And then, secondly, as we think about the post-approval setting, is it reasonable to expect some sort of launch guidance? And if so, you know, what metrics do you anticipate giving us?
Hey, good morning, and thanks for squeezing me in.
I wanted to push you guys own something so it sounds like you do know yeah.
Table in the high end.
I was just wondering.
CA definitely expectation specifically when this label would arise.
And then just secondly, as we think about the post approval setting.
Is it reasonable to expect some sort of launch guidance.
And if so what metric do you anticipate giving us thanks a lot.
Colin Bristow: Thanks a lot. Thank you, Colin, for your questions. To answer your first question, I already reiterated that, per company policy, we are not commenting on the subject matter of ongoing and potential ongoing conversations at this time with FDA. We continue to expect a response from the FDA with regard to our NDA by the April Feeds. So that kind of answers your first question about the label where we are and regarding the launch matrix. Matt joined us recently in January.
Thank you Colin.
For your questions.
To answer your first question.
I already did a power.
Our company policy, we are not commenting on the subject matter of ongoing and potential ongoing conversation at this time with the FDA.
We continue to expect a response from the FDA with regards to our NDA by the April 5th four to 14.
Colin Bristow: He is building all the launch matrices, and once we have the approval, he will be in a position to share what matrices to track to see the performance of the launch. Matt, would you like to add anything?
So that's kind of answering your first question about the level, where we are.
And regarding the launch metrics.
Matt.
<unk> joined US recently in January He's building all those launch metrics and once we have the approval he will be in a position to share what matrix to track to see the performance of the launch Matt would you like to add anything yeah. That's right. I mean, you know as we as we get into market.
We will share the key performance indicators that are relevant.
That's great. Thank you.
Vimal Mehta: Yeah, that's right. I mean, you know, as we get into more markets, we'll share the key performance indicators that are relevant. That's great, thank you. The next question is from Rob Selvaraju of H.C. Wainwright. Please proceed with your... Thanks very much for taking my questions, three relatively straightforward ones, hopefully. Firstly, I wanted to ask... you could comment on your ex-U.S. partnering strategy with particular emphasis on the European outlook given your stated timeline for filing the MAA, but also any other regions that you may be exploring at this time with respect to 501. So that's a great question, Ram.
The next question is from Ross Silver How're you of H C. Wainwright. Please proceed with your question.
Thanks, very much for taking my questions three relatively straightforward ones hopefully firstly I wanted to ask if you could comment on your ex U S. Partnering strategy with particular emphasis on the European outlook, given your stated timeline for filing the MAA.
Also any other regions that you may be exploring at this time with respect to hydro one.
So that's a great question.
Thank you.
Uh huh.
Vimal Mehta: Thank you. Geographical expansion is a critical part of our growth for the BXcel 5.1 program because there are a lot of patients, or an equal amount of patients outside the U.S. So in terms of the European strategy, we wanted to file an MA once we had clarity on our approval from the FDA, and as I said, we will be ready to file in the first half of. As we file our MA, that basically gives us about 15 months of time window for approval and then the launch, so we will initiate our business development efforts to look for a partner, and we also get approached, obviously, actively by partners.
Geographical expansion is critical part of our growth being fueled by one program because there are a lot of patients are equal amount of patients outside.
So in terms of the European.
Our strategy.
We wanted to file MAA once we have clarity on our approval from the FDA and as I said that we will be ready to file in first half of this year.
As we filed our MAA that basically gives us about 15 months of time window for approval and then the launch so we will initiate our.
Business development effort to look for a partner and also if we get approached obviously by actively by the partners also.
Vimal Mehta: [inaudible] So that process will continue, and our focus will be that we have a partner who has the right kind of attributes, not only for our first two indications, it's esophageal and bipolar, but they have the capability to be able to follow on for our follow-on approval. In terms of other regions, I would say Japan is a very important territory.
So that process will continue and our focus will be that we have a partner who has the right kind of attributes not only for our first two indications it gets ophelia in bipolar, but they have the capability to be able to fall along for our follow on S. N years.
In terms of other regions I would say, Japan is a very important territory.
Vimal Mehta: As you know, particularly in Alzheimer's, there is a very large patient pool, and it's a big, big unmet medical need. So we will be considering those options. And I think after we get approval for our first product, both Europe and Japan will be very viable partnering opportunities for BioXcel 501 from a commercialization perspective.
As you know that particularly in Alzheimer's there is a very large patient pool, and it's a big big unmet medical need. So we will be considering those options. So I think after we get approval of our first product, both Europe , and Japan will be very viable.
Partnering opportunities for <unk> 501 for me.
Alright.
Virtual edition perspective.
Vimal Mehta: Great. Secondly, I wanted to ask about your plans for major depressive disorder and if you could clarify specifically what you envision as the optimum treatment setting. You're talking about adjunctive therapy, but adjunctive to what? And in what specific subgroup of patients within MDD are we talking here about treatment-resistant depression, and how is this being quantified or classified, as well as if you could comment on the nature of the competitive landscape in MDD and how you anticipate being able to obtain optimum positioning within this indication. Sure, Rob. So, this is Rob Risinger again.
Great Secondly, I wanted to ask about your plans in major depressive disorder, and if you could clarify specifically what you envision as the optimum treatment setting you are talking about adjunctive therapy, but adjunctive to walk.
And what specific subgroup of patients, but then M D D.
Are we talking here about treatment resistant depression, and how is this being quantified or classified as well as if you could comment on the nature of the competitive landscape in M. D D and how you anticipate being able to obtain an optimum positioning within this indication.
Robert Risinger: We do plan to take this into major depression. We anticipate that we would be studying patients with simple major depression as an adjunct to an SS or SNRI. We don't intend to specify SS or SNRI, so this would be used in the outpatient setting for patients who have major depression as a sort of accelerant, as an adjunctive, in order to treat patients more rapidly and sort of get them on their feet.
Sure. So this is Rob risinger again.
Do plan to take this into major depression, we anticipate that we would be studying patients with simple major depression, as an adjunct to NSS or S. NRI.
Don't intend to specify that SaaS or SNRI. So this would be used in the outpatient setting for patients who have major depression as a sort of accelerant.
As an adjunct is in order to treat patients more rapidly and sort of get them on their feet.
Robert Risinger: We know that the landscape is changing in terms of treatments for depression, so we're not specifically targeting TRD, or treatment-refractory depression, at this point. Okay, and then lastly, with respect to 502, I was wondering if you could talk a little bit more about potential unique areas of patient evaluation, efficacy-related outcome measures in the chronic context of Alzheimer's disease and dementia, and in particular, if you have plans to include sleep monitoring as an integral part of the clinical development effort for 502. There are additional potential indications rather than simply, I'll call it simply, but that's a pejorative, I suppose, dementia and agitation associated with it.
We know that the landscape is changing in terms of treatments for depression, we're not specifically targeting T. R D or treatment refractory depression at this point.
Yeah.
Okay, and then lastly, with respect to five O. Two I was wondering if you could talk a little bit more about potential unique areas of patient evaluation.
Vacancy related outcome measure in the chronic context.
Oh, Alzheimer's disease, and dementia and in particular, if you have plans to include sleep monitoring as an integral part of the clinical development effort on five O two.
We are let me say it let me take the first part there are additional potential indications.
Rather than simply I'll call it simply but thats the majority of I suppose demur.
Dementia in agitation associated with dementia, there are other potential indications which were exploring.
Frank Yocca: There are other potential indications which we're exploring. So, let me help you. This is Frank. We're dealing with a molecule now with a different mechanism of action. We are approaching it because we have good preclinical data on chronic agitation and the profile that it gives us, but our approach will be very similar to what we did with 501. We'll look at the molecule, see how it behaves, and go in the different directions that it really points us.
So let me let me let me help yes. This is Frank Rob. So we are dealing with a molecule now with a different mechanism of action.
We're approaching it.
Because we have a good preclinical data on chronic agitation and the profile that it gives us but our approach will be very similar to what we did with 501 will look and we will look at the molecule see how it behaves and go into different directions that it really points us to.
Frank Yocca: But the initial approach will be to fit for a fit for chronic agitation. Thank you. The next question comes from Anita Duchant of Berenberg. Please proceed with your question. Hi, good morning. Thanks for taking the questions. Some of them you have already answered.
But the initial approach will be to fit.
Fit for chronic agitation.
Thank you.
The next question comes from Anita Dushyanth Bahrenburg. Please proceed with your question.
Okay.
Hi, good morning, Thanks for taking the questions.
A follow up on.
Some of them that you already answered.
Operator: So, with respect to the study in Alzheimer's, the two phase three studies, could you provide some clarity on how we should think about the number of patients that might actually benefit from the higher dose between the two different settings? Anita, as you know, in our Tranquility Trial, our lower dose as well as the higher dose, it's a dose-dependent response. So, we don't know what the differences will be from the e-doses; we are testing two doses to see how the patients will benefit and what the response looks like, like we exactly did it in our serenity 1 and 2 trials for schizophrenia, bipolar 120 and 1.
With respect to the steady you know.
The two phase III study could you provide some clarity on that.
How we should think about like the number of patients that might actually benefit from the higher dose between the two different settings.
Yeah.
I need to as you know in our tranquility trial.
Our lower dose as well as higher dose, it's a dose dependent response.
So we don't see that like you know.
What are the differences will be from the E. <unk>. We are testing two doses to see how that patients will benefit and what that is sponsored looks like like we exactly did it in our Sydney entity, one and two trials for schizophrenia, and bipolar 120 and 180. So that's the purpose and once we have the data.
Vimal Mehta: So that's the purpose, and once we have the data, we will know, like now, do we take both doses forward, or do we take just one dose forward? So those are the things that will come out of our phase three program. Rob, would you like to add anything else?
No like no do we take Bulldozes Broadwater, we take single dose forward. So those other things.
We'll come out of our phase three program, Rob would you like to add anything else.
Robert Risinger: No, as is typically done, particularly for registration, we simply characterize those who may benefit from a higher dose versus a lower dose. And if we find factors, we'll put that in the label. It helps to guide dosing.
As is typically done, particularly for registration, we simply characterize those who may benefit from a higher dose versus the lower dose and if we find.
Factors will put that in the label.
Helps the guide dosing.
Robert Risinger: Yes, definitely. Thank you. And then a question on the MDD program. I was wondering, like, is there a specific subset of the population from 27 million cases that might actually benefit. This is a young treatment. What I like about clinical development is that we discover things along the way. Brats and mice don't necessarily predict everything in humans, and so we intend to characterize the effects in the general population of those with depression. If we find subpopulations, as you're suggesting, that may well be another development pathway, or it may steer us in that direction.
Yes. It does thank you and then.
On the M D D program.
I was wondering.
A specific subset of populations on the 20 <unk>.
And in many cases that might actually benefit from it.
Hey, Jim.
What I like about clinical development as we discover things along the way rats and mice don't necessarily predict everything in humans and so we intend to characterize the effects in the general population of those with depression, if we find sub.
As you are suggesting that may well be another development pathway, where it may steer us in that direction.
Robert Risinger: So, Anita, let me add a little color. In general, these antidepressants don't work in the first four to six weeks. And that's what we are targeting. How to accelerate the effect of some of these agents.
So Anita let me put a little.
Add more colored.
In general these antidepressants doing work in first four to six weeks and Thats, what we are targeting how to accelerate the effect of some.
Vimal Mehta: So that's the unmet need we are targeting. Great, thank you. The next question comes from Samir Devani of RX Security.
Some of these agents so that that's the unmet need we are targeting.
Great. Thank you.
Okay.
The next question comes from Sameer Giovanni of Rx Securities. Please proceed with your question.
Operator: Hi guys, thanks for taking my question; it's just the one. Congratulations on getting the data published in German. And it's clearly a very positive article.
Hi, guys. Thanks for taking my question and it's just the one.
Congrats on getting the data published in Jama, obviously, that's a.
Ladies got very positive article, but I was just caught by one phrase or one one comment in the paper with the with a discuss is no consensus on the change in PEC score that represents the minimally clinically important difference can you just comment on that price for me. Thanks.
Samir Devani: Quote by one phrase or one comment in the paper where the author discusses no, www.microsoft.com.au Minimally Clinically Important Differences, Comin' on that. Good afternoon, Samir. So, let me pass this question to Robyn. I wish I could delve into the intricacies of defining a, quote, minimally important clinical difference, but there's a lot of discussion about that. I think what the manuscript shows and the publication is that we have far greater than a minimal difference, and arguing over what a minimal clinical difference is a bit pedantic. We showed a robust response that no one argued about. The change in PEC score was quite high. The percent who respond by almost any criteria is, you know, 70, 80, almost 90 percent.
Robert Risinger: It's simply irrefutable. So you know, I'll be happy to play professor and we can talk for an hour, but we showed far greater than a minimal clinically significant difference. Great, thanks very much. If there are no additional questions at this time, I would like to turn the call back to Dr. Vimal Mehta for closing remarks. Thank you everyone for joining us today. I look forward to connecting with many of you in the coming weeks and updating you on our continued progress.
Good afternoon.
So let me parse this question to Rob.
Uh huh.
I wish I could delve into the intricacies of defining a quote minimally you know important clinical difference.
But there was a lot of discussion over that I think what the manuscript shows in the publication is that we have far greater than a minimal.
And arguing over what a minimal clinical differences a bit pedantic.
We showed a robust response that no one.
<unk> argues.
The change in PEC score was quite high as a percent to respond by almost any criteria is.
70, 80, almost 90%.
It's simply irrefutable so.
I'll be happy to play Professor and we can talk for an hour, but we showed far greater than a minimal clinical significant difference.
Great. Thanks very much.
Vimal Mehta: Have a great day. This concludes today's conference. You may disconnect your lines at this time.
There are no additional questions at this time I'd like to turn the call back to Dr. Fred Moll meta for closing remarks.
Operator: Thank you. Copyright 2019 Mooji Media Ltd. All Rights Reserved. No part of this recording may be reproduced without Mooji Media Ltd.'s express consent. Raghuram Selvaraju, www.microsoft.com, www.microsoft.com.ca, Copyright 2019 Mooji Media Ltd. All Rights Reserved. Copyright 2020 Mooji Media Ltd. All Rights Reserved. No part of this recording may be reproduced, BF-WATCH TV 2021, All Rights Reserved. No part of this recording may be reproduced without Mooji Media Ltd.'s express consent. The Bulletproof Executive, 2013
Thank you everyone for joining us today I look forward to connecting with many of you in the coming weeks and updating you on our continued progress have a great day.
This concludes today's conference you may disconnect your lines at this time. Thank you for your participation.
Okay.
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Yeah.
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