Q1 2021 Brainstorm Cell Therapeutics Inc Earnings Call
Yes.
Thank you for joining us today your teleconference will be beginning shortly.
Thank you for joining us today and your teleconference will be beginning shortly thank you.
[music].
Greetings and welcome to Brainstorm cell Therapeutics first quarter 2020 on an earnings call. At this time, all participants will be in a listen only mode and.
A question and answer session will follow the formal presentation.
If anyone should require operator assistance during the conference. Please press star zero from your telephone keypad.
Please note this conference is being recorded.
At this time I'll now turn the conference over to Michael Wood with lifestyle advisors. Mr. Wood, you may begin.
Good morning, and thank you everyone for joining us and before we begin the opening remarks I'd like to remind listeners that this conference call contains numerous statements descriptions forecasts and projections regarding brainstorm cell therapeutics and its potential future business operations and performance statements regarding the mark.
And the potential treatment of neuro degenerative diseases, such as Alison and M. S. The sufficiency of the company's existing capital resources for continuing operations, and 2021 and beyond safety and clinical effectiveness of the neuro and technology platform and clinical trials of neuro and and the latest clinical development programs on the company.
And with all these and develop strategic collaborations and partnerships to support the business planning efforts.
Forward looking statements are subject to numerous risks and uncertainties many of which are beyond the brand's drops control, including the risks and uncertainties described from time to time and the company's SEC filings. The company's results may differ materially from those projected on today's call and Brainstorm undertakes no obligation to publicly update any forward looking statements.
Joining me on the call. This morning, and will be I believe a widespread with it and see it all brainstorm Doctor Stacy Lindbergh executive VP and head of global clinical research, Dr. Ralph <unk>, President and Chief Medical Officer, Dr. Peter <unk> Shah Executive VP, and Chief Financial Officer, and in addition, Dr. David set born executive VP and Chief operating off.
There will also be on the call and won't be available to answer your questions during the Q&A session.
So now I'd like to turn the call over to Mr. Leibowitz. Please go ahead.
Thank you Mike.
Thanks to all listening and for joining us to discuss our first quarter financial results corporate highlights.
Sorry, sorry on the call today, we will be discussing the current status.
I've already less program, the very exciting and recently announced top line data from our phase two progressive Ms trial.
Our strategic plans for these programs as well as our quarterly financial results.
Starting with Australia less program I'd first like to see debt, we remain confident and the strength of our data.
And plus neuron as a treatment for AOS and.
And it has brought potential as a technology platform and neuro degenerative disease.
In parallel to regulatory interactions, we have been enacted consultation.
Various experts, including principal investigators and less decisions the decisions.
Regulatory advisors as well as a patient that was <unk>.
Absolutely and groups and the United States as well as in other jurisdictions.
Doctor stays on Lindbergh, our VP and head of global clinical research.
We'll provide insight into how we are going about these interactions and gaye.
Feedback on our program.
However, I want to stress that we plan to take all feedback into consideration as we assess the most efficient way to enable the LLS based on success and are on their own including a potential BLA submission and our other regulatory and business options.
We are diligently working to define the we're on path forward and are gaining scientific support.
We can assure you this is a trop day top priority.
For us and we intend to update the patients and investing give me that these as quickly as possible, but we must let additional developments play out before providing a more comprehensive update.
Dr. Ralph current and our President and Chief Medical Officer will discuss our progressive and his program and a few minutes, but let me give you some of the highlights.
In March we announced positive top line data from our open label Phase two trial.
These data showed that the study met its primary endpoint with neuro and being found to be safe and well tolerated. We also observe consistent improvement and key secondary endpoints spanning neurology.
Function permission and the Biomarkers collectively these promising results further supports the utility of the newer and technology platform and neuro degenerative disease and provide additional proof of concept point of sequel neuron therapy and progressive M. S.
We are now and consultation with our principal investigators to align on next steps for this program and putting design a phase two b trial and regulatory pathway.
We're also on discussions with potential strategic partners.
And part of these efforts, we're also preparing and scientific presentations and peer reviewed manuscript on our data, which will allow us to gain valuable feedback from them as community as we work to determine the next steps and all of our clinical development strategy. One the strategy is whole solidified we'll be sure to provide an update to both investors and the broader M. S.
Community.
Before I hand, this off to say Stacy I want to take a moment to acknowledge.
And that all of the progress that we will discuss share today. He has only been possible over the dedication of our patients and caregivers together with our employees business partners and clinical investigators and I'm extremely grateful to all of these individuals for the critical role they have played and successfully advancing neurons political development.
With that I'll now hand, this call over to Stacy our executive Vice President to discuss the status of our weight loss program and Stacey.
Thanks time.
And so I'm just previewed a few minutes ago, we remain confident and the potential of neurons and a less and are currently consulting with a wide range of people, including principal investigators Atlas experts statisticians regulatory advisors and patient advocacy groups.
These discussions have provided important feedback.
Which has been very positive.
They've also allowed us to gain new insights from our data as well as a lot of support and encouragement from the groups that we discussed our clinical trial data with.
We now have a very mature draft of our manuscript on.
On the study prepared and we expect to submit for publication shortly.
Throughout the writing process, we've had an excellent partnership with the study's principal investigators who are all off the authors.
Timely publication of the manuscript will be important and it will allow us to share our data with and collect additional feedback from the broader and less community.
Given that we are still collecting feedback from experts and that our regulatory interactions and need to remain confidential at this time it would be premature for us to share more specific next steps and analysts today.
However, we are very pleased with the progress we've been making behind the scenes.
And look forward to providing an update.
I'll now hand, the call over to Rob to speak about our MSP program.
Thank you Stacey.
I mentioned, we recently announced positive top line data from our open label Phase two trial and progress at MFS.
We pursued this trial based on the growing confidence and our proprietary cellular technology platform.
The well defined unmet need and progressive en masse.
And our beliefs that repeated intra articular administration of neuro and has the potential to simultaneously address neuro inflammation and urged your generation.
And improved functional outcomes and progressive Ms patients.
The clinical trial was conducted at four leading Ms centers of excellence and the United States and enrolled 20 progressive Ms patients who had stable disease.
In other words, they had not relapsed or required rescue medications and the six months prior to study enrollments.
Participants on this trial and received three repeated intra equal administrations of neuron each given two months apart and were followed for 28 weeks after their first treatment.
This was a phase two clinical trial and the primary objective was to which was to assess the safety and tolerability of treatment and importantly, we included multiple secondary endpoints designed to evaluate the preliminary efficacy of neuron and progressive and mouse. These.
These include itself, several well validated clinical endpoints and disability and function.
Cerebrospinal fluid and serum biomarker analyses.
And a validated patient reported outcome to confirm improvements and walking function.
To ensure a robust analysis of our data we set pre specified response criteria for clinical improvements in key clinical efficacy endpoints using benchmarks that are well accepted by the scientific community.
Additionally, we were careful to enroll a patient population that was very similar to other progressive Ms studies in terms of demographics.
<unk> and functional measures, which allowed for further comparisons.
This allowed us to make meaningful comparisons to a 48 patient matched clinical cohorts from the comprehensive longitudinal investigations and M. S.
A cohort that are followed at the Brigham and Women's Hospital also known as the climb study.
The key findings from our progressive and mouse study, whereas follows.
First of all the trial met its primary endpoint as neuron was demonstrated to be safe and well tolerated and progressive Ms patients.
Procedure related adverse events observed and the study were similar to our experience and allies.
In terms of efficacy are pre specified responder analysis showed consistent improvements across all functional measures, including walking vision and cognition and such.
Such improvements were notably not observed in any of the matched client patients, suggesting that they were due to the clinical benefit of neuro and <unk>.
Truly compelling finding.
We also observed consistent increase and delivered neurotrophic factors and a reduction across key inflammatory biomarkers further confirming neurons therapeutic mechanism of action.
So what does this mean collectively.
Collectively these data provide proof of concept for neuron and progressive and mass and strongly support its potential to address the intra thecal central nervous system inflammation and progressive loss of neural function that drive the relentless clinical progression of this disease when discussing the data with our principal.
The Gators and the patient advocacy community. We have received extremely positive feedback and a great deal of encouragement to take next steps.
It was discussed earlier, we've begun collaborating with experts to design a potential phase two b trial, and we are preparing a peer reviewed manuscript and scientific presentations to effectively communicate our results to the broader EMS community.
We will certainly take all feedback into account as we consider potential next steps, which will include regulatory review and or discussion with potential strategic partners. We look forward to providing everyone with an update once our plants have been solidified.
I'll now turn the call over to freedom to discuss the financials.
Thank you Ralph.
My pleasure now to walk you through our first quarter of 2021 and I'm film performance.
Research and development expenses net for the three months ended March 31, and 2021, but 434 million compared to $5 $95 million net for the three months ended March 31, and 2020 net.
This decrease of $1 six 1 million year over year was primarily due to decrease and expenses related to our phase III and phase two clinical trials and a decrease in expenses in connection with stock based compensation material and travel rent and other activity.
The decrease in expenses was partially offset by an increase and costs related to payroll patents preclinical R&D activities and consultants and a decrease and proceeds received in connection with the treatment of patients under the hospital exemption regulatory pathway and a decrease and grant participation by the Israel.
Innovation authority or I E.
Excluding participation from I Eh and other grants and proceeds received under the hospital exemption regulatory pathway research and development expenses decreased by $2 three 3 million from 714 million and the first quarter of 2022 481 million and the first quarter of 2021.
General and administrative expenses for the three months ended March 31, 2021 were $2 $5 9 million compared to $2 36 million and the three months ended March 31 2020.
This increase of 228000 year over year was primarily due to increase and payroll stock based compensation consultants rent and other costs, partially offset by a decrease and PR and travel related expenses.
Net loss for the three months ended March 31, and 2021 was $6 six 6 million or <unk> 19 per share as compared to a net loss of $8 1 million or <unk> 32 per share for the three months ended March 31 2020.
Cash cash equivalents, including short term bank deposits were approximately $40 million as of March 31, and 2021 compares to approximately 42 million as of December 31, and 2020.
Our total available funding as of March 31, and 2021 was up.
Approximately 57 million. This includes cash cash equivalents and short term bank deposits as well as remaining non dilutive grants, which amounts to approximately 41 million. In addition, we have approximately $16 million available to us and untapped ATM capacity.
For further details on our financials. Please refer to our form 10-Q filed with the SEC today.
Back to you operator.
Thank you.
We will now be conducting a question and answer session.
Would you like to ask a question. Please press star one from your telephone keypad and a confirmation tone will indicate your line is and the question queue do.
And you May press star two if he would like to remove your question from the queue.
And so they're using speaker equipment and it may be necessary to pick up your handset before pressing the star keys, one on police on we poll for questions and what's going on at Star one.
Thank you. Our first question is from the line of David Bautz with Zacks small cap research. Please proceed with your question.
Hey, good morning, everybody.
And you're wanting.
For D. A L. S data that we're gonna be seeing and the publication will that include any data from the expanded access program or a hospital and central hospital exemption programs.
No that would not be included in it.
Okay.
And looking.
Looking at the M. S results Dr. Carl I'm curious, if you could talk about which of those data that you're you're most excited about.
Oh.
Yeah, No David Good morning, I think we're most excited about the.
The overall consistency of the functional improvements there has been and.
And if studies and the past that have shown inconsistent changes across different endpoints, what really struck us was that we saw improvements across all functional measures and these include walking.
By the time 25 foot and walk the nine hole peg test measuring upper extremity function.
Vision and that's the test called the low contrast letter acuity and.
And what was most striking where the cognitive changes there was quite a significant improvement and cognitive processing speed. So these were these were the very striking findings and.
And they were I.
I think reinforced by the consistent biomarker changes that we saw in this study between.
Between Neuroprotective and neuro inflammatory biomarkers the parallels what we saw in ALS. So very positive proof of concept study.
Okay and lastly in regards to the next trial for M. S. I know you probably cant go into details but.
And late stage and Ms trials or at least one year and lengths and I'm just curious how you're thinking about dosing since youre just doing three doses right now would you want to increase the number of doses for those patients that the trial does go for at least a year.
Yeah. It's a very good question and I think it's premature for us to give.
More detail because we're still in discussion with some of the experts that are giving us valuable feedback on.
I think the direction that youre going and is.
Directionally correct and the sense that on.
Longer study will be needed and.
And also additional doses about how many and what the interval will be is something that we're still we're still discussing internally and also with experts. So thanks for the question.
Yeah.
Okay. Thank you very much.
Thank you David operator, we have a.
And the Q&A that Investor Centre, and earlier, where the Mic would see if you can read the Qs and will give us answers.
And then we'll go back to the life Yep. Thank you sorry, there were a number of pre submitted questions. So I'll start with this one.
First of all what are your plans for <unk> going forward do you intend to conduct another trial.
Yeah.
Thank you very much our ultimate goal remains to secure approval for neuro and and AOS.
And we remain confident and the effectiveness and safety of neuron.
I'll start and priority is to publish the full phase III data and a peer reviewed journal is we are also meeting with experts and.
And key opinion leaders, who are not part of the trial and do not have first hand experience with neurons are sure the Ada and receive their feedback.
We are receiving and invaluable insight and positive feedback from these world when on the Atlas experts.
There's widespread agreement demand experts, we've spoken with the.
So the data support advancing neuron as a treatment free and less so.
So it depends on salt, it's not a question if there's a question why knowhow.
Next question please Mike.
Will you be submitting a drug.
Approval submission from neuron and a lot.
And and health, Canada, or the U and as a follow up to that are you considering considering bypassing the FDA and getting approvals from neuro and other and other territories.
While we're not considering to bypass we are considering other geographies and why we're still assessing our exact strategy with the FDA.
Next.
The next question can you comment on the public statements that the FDA released about newer on the statement was somewhat unusual.
Stacey.
Yes, it was unusual for the FDA to issue such a statement on.
And the after you did not reach out to us prior to issuing the statements, but did prioritize the call with US quickly following issuance and what we learned is that they were not operating out of concern based on actions of brainstorm.
And I guess to kind of round it back because the F. D. A indicated in their preliminary assessments, while not recommended by them.
We're not precluded from submitting a BLA.
Thank you very much Mike.
Our next question does brainstorm intend to submit a BLA to the FDA following the phase III neuro and.
And if not right at Cabot was granted FDA approval with without very exceptional clinical data and what.
Is the downside of submitting a BLA at this point.
And if he would take this one please.
Sure.
Great.
As a company brainstorm is continuing to weigh the option of a BLA submission our current priorities and as we stated at our publishing the phase three data and appear to be a journal and meeting with analysts and experts and key opinion leaders.
And it's important and we're prioritizing people that are outside of the clinical trial ecosystem to share our data with and to receive feedback on.
And our goal is to secure and approval for near and as a treatment for AOS and if after consulting with key opinion leaders and after the data is published if we determined that the most rapid way to achieve this goal is just and a BLA following our phase II data, we will pursue this path.
In other words, there may not be a downside to submitting a BLA at this time and that's exactly what we're working to determine.
Next question please Mike.
Can you talk about the revenue generating opportunity for neurology and the international and less marketplace.
David do you want to take this one.
Sure.
So.
There is a lot of patients suffering from and that's outside of the U S.
And are these huge tweedie, calling this need in the U S and beyond there is a very strong international business opportunity.
And the reminder, I mean outside of the U S D and that's why do I pronounce is higher than 400000 patients.
Yeah.
Thank you and next question please.
The next question is regarding the patient advocacy group IMA and <unk>.
And that's just calling for a congressional hearing on.
How are you working with them and what is your strategy here.
Thank you. So we are fully engaged and working with a L. S advocacy community, including Gaiam L. S. I.
And I loved and advise us on this meeting there's a growing awareness of the critical unmet need and the alissa and limited treatment options.
And we're certainly open to discussing practical options across a range of stakeholders, including government.
Next question. Please next question, Rick and relates to hospital exemption. This investor said, they've heard that there are patients being treated and and Israel on open label and many patients where the results and why doesn't the company share them with.
With the public and is this debt.
Yes.
[noise] facility still open for treatment.
Yeah very good question and thank you and this will probably on some part of what David asked before and.
So if you want to take this one.
Sure.
At the present time, we don't have additional outcomes to share from this trial and it's really for a couple of reasons.
First COVID-19 travel restrictions has severely limited this program and the ability to continue to collect longitudinal data and the program.
And second you know.
The questions references that this is an open label study while data can be accessed during the conduct of the state of the study.
And many of the established clinical endpoints and a less are subjective in nature and it's an industry practice to not discuss ongoing trials to avoid bringing bias into the trial and referencing back to the question that the day bid ask at the beginning about this study our publication and from.
From our phase III study will be a read out of and the publication of that trial and therefore once one campaign additional additional data from other studies.
Thanks very helpful. Next question. Please and our next question is regarding the extra some platform does treating a patient cells with neuro and a half.
The ability of the exercise to fight disease.
The quality of the message that exosomes are carrying.
Provide the needed support for neuro and to slow down and disease progression.
Thank you Ralph.
Sure.
It's our belief that similar to neuro and.
And the potential therapeutic benefits of Exosomes is mediated through the delivery of biological molecules that reduce inflammation.
Provide tissue support and promote repair.
So to answer this question directly it's the quality of the message or cargo that exosomes are carrying that determine the outcome.
Exosomes also provider a practical option that allows for easier formulation.
It just takes and may have better tissue delivery and other ways. There also potentially less immunogenic supporting what we believe is a very high potential off the shelf.
Allogeneic cell source a treatment option.
Thank you very much and a question.
And what are the plans to partner neuron and AOS.
Are you currently speaking with partners.
Or from Europe.
Thank you David.
We are open to collaborate and and cycling and continuous discussion and we thought by sponsors.
Or are all the different programs and our portfolio.
So for and you're already there that's indication as well as per we're super lice.
There are as well speaking, we bought mers and tourists you know excess and that far and.
And air D S and vacation.
We are actively engaged in discussion and that's for coffee and that's how the the reason you cannot provide details on esports.
Thank you.
And we have remaining question and this is on the Alzheimer's program.
At this point why don't start a clinical trial for Alzheimer's disease.
You're on potentially to help or improve a person's condition with Alzheimer's.
David.
Yeah.
And the process of finalizing regulatory discussion regarding the Alzheimer clinical trial.
The rationale for and you already have that standard disease and strong.
And then David and that narrowing simulation phase and both in school.
And that's basically seek neurotrophic seaports system and is that right.
Is that a decision.
And that is.
Alcohol did even by neuro and.
Including set and neurotrophic factors and micro RNA molecules.
<unk> known to have it be Nikki shortly and second is that Merck preclinical models.
Thanks, very much Ralph do you want to elaborate a little bit on this.
Yeah, I can just say that there is a tremendous interest and and using cellular therapy to modify the and.
Environment with Alzheimer's disease, which includes obviously inflammation and.
And.
I think our recent findings in and that's the cognitive changes and our M. S. A progressive Ms patients may be and important indicators that Alzheimer's is a.
Very good next indications so that's what I would like to share today.
Thank you very much Ralph Rob you cannot reopen for a few more questions from the.
Audience and thank you and the next question is from the line of Neel Farkas with Maxim Group.
I think its Jason Mccarthy.
Hi, Jason Good morning, and then maybe my name is Neil to I don't know that's very busy they came up that way.
Good morning, Hi cell. So it's like a couple of questions first.
On a L. S. A and I think this question is geared towards state keeps on kind of a call on your experience with Biogen.
How do you think about.
The upcoming could do for added can you map and all time or it's completely unrelated obviously to do what you're doing and a L. S here, but the unmet need there's a parallel there and the F. D. A may look at a drug like that is it might've missed and it's trial, but a good something good and it's safe and on.
And I need something and can you look at their own and L. A and it's similar similar words, where it definitely doing something to.
And so the good for these patients and native missed on.
And it's primary and the phase III trial, but there's something there and with regulators and maybe look at it kind of like there. It seems like they're looking at out of can you bet.
Oh that was a good and sort of get question, but.
Although if you do that.
I'm not sure.
And just area and how do you make.
Jack do you think about how you're positioned neuro and now totally understood sure Yeah, Yeah, and I guess, what I would say is there are a lot of and there's similarities like you're pointing out and there are also a lot of things that are quite different.
And including where we're going to a different center of the FDA and are obviously very different diseases, but I think what you're describing really is at the heart of what every biotech or pharmaceutical companies seeking and their research.
And we're here to produce meaningful medicines for patients and trials sometime this result with and and.
You know a primary endpoint that has missed and the job that we had been working to do and really even the information we've already shared and the public domain.
Precisely we have been working to understand what have we learned and what can we conclude about and your own and.
And you know we believe we gained key insights and therefore, we reached the conclusion and which we've shared repeatedly and even today that we believe strongly and the efficacy of neurons and so.
Back to the similarities I think that much like Biogen had to do we had been forced with the results of this trial and the need to book very objectively and transparently and our data to.
And to share that data also very transparently with with regulators and with Alice experts advocacy groups.
Pit to seek that.
Input that is removed from from the company and then to put for the stuff that really serves and the patient community and our investors as best we can and so.
And it's a very interesting connection and analogy and I think where we're doing exactly the same the same thing in terms of trying to make it the best next steps and put forward and what we believe is the most objective and compelling evidence that speaks to near them.
Okay, and so then how do you think about.
Cell therapy from its pure complexity, and we published the high and which I'm sure you saw it means a lot and they did Miss a few phase III trial on the price.
And Barry, but the way you think about how complex.
L therapies are there's a lot of things happening that might not be captured by one snapshot of an endpoint and so how do you think about neuro and going forward into progressive and that and to your to be do you start to look at other things like MRI white matter free water of brain swelling and kind of.
Tying that to inflammation and inflammatory biomarkers versus the typical traditional and net.
Like trials on maybe relapse and remitting.
Appliances.
Very good question, Ralph I'll, let you take this.
Yeah, Thanks, Jason and I'll try to be very brief I think and message the different a different disease and the sense that the outcome measures.
Have a longer history and in some ways are more more stable and easy to measure having said that we believe very strongly that we can evaluate the cerebral spinal fluid as a source of biomarkers.
And and rather than look for new new Biomarkers, we believed on building upon our strength and what we've learned in the past and other words, we're staying very consistent a biomarker changes and preclinical and AOS and humans and now and progressive and mass that speaks to the mechanism of.
Action and as we have a very well defined cellular products similar to the car T World, We believe that and.
The technology is very predictable debt fingerprint or the biomarker changes related to the sales can be measured very accurately and.
And that's really the strength of our conviction that the technology platform and can be tested very reliably and progressive M. S and we believe and Alzheimer's as well, so we're very confident and our technology and.
And what we can measure and how we can draw and legitimate conclusions from what we're doing.
The last question first and I am so are you planning to position brainstorm in all timers disease strategically, meaning you know you have the bow and the out of Canyon map Hadoop is coming up.
And I was meeting over the summer you know where everybody is expecting alzheimers to suddenly be extremely busy and you do have your program.
And here too much about today, but maybe kind of walk us through what you're thinking for Alzheimers and positioning brainstorm in that state.
Well a very good question. So we have a lot to figure out and these few months as you know.
Our priorities are like this the first thing is and we were submitting very soon.
Oh, Oh, Oh, a peer reviewed manuscript ominous going from here.
Viewed publication and.
We got to figure out what's on that stuff pretty unless we're getting close to that and then we have to figure out what's on extra premise, it's going to be probably another trial with our souls and with other partners. We've got to figure that out also.
Old timers, we were thinking maybe to lay off but we have some very interesting biomarker data from the L. S and M. S trials, which gives us strong support and we should continue from Congress.
Net of matters, which you'll see part of our one of our publications and we're going to probably have a separate publication on the biomarker data from your list only.
So we are deciding now on the next quarter how to proceed with the with Alzheimers.
Conversation with the regulatory and the Europe, and we'll see how that goes.
Well then on once we have something to announce final.
Great. Thank you all for taking the questions.
Thank you very much operator, any more questions, we have time for one or two more.
If you'd like to ask a question at this time you May press star one from your telephone keypad will pause a moment to assemble the queue.
Okay.
And what's your net star one to ask a question at this time.
Thank you.
Thank you at this time I will hand, the floor back to management for any additional from remarks.
No. Thank you very much on the again I want to thank everyone for being with US on this call. This morning.
And keep up with the faith, we'll have that this is going to come to and approval one day.
Thank you very much.
Thank you and everyone joining us today. This will conclude today's conference. Thank you for your participation you may now disconnect your lines at this time.
Yeah.