Q4 2020 Brainstorm Cell Therapeutics Inc Earnings Call
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Greetings and welcome to the Brainstorm cell therapeutics fiscal year end 2020 earnings call at.
At this time all participants are in a listen only mode.
A question and answer session will follow the formal presentation. If anyone should require operator assistance. During the conference. Please press star zero on your telephone keypad. As a reminder, this conference is being recorded I would now like to introduce your host Mr. Michael Wood of lifestyle advisors. Mr. Wood, you may be.
Again.
Thank you and good morning, everyone and thank you for joining the brainstorm cell therapeutics call before we begin the opening remarks, we'd like to remind listeners that this conference call contains numerous statements descriptions forecasts and projections regarding brainstorm cell therapeutics.
Potential future business operations or performance statements regarding the market potential for the treatment of Neurodegenerative disorders, such as this.
Is there any other companies the sufficiency of the company's existing capital resources for continuing operations in 2021 and beyond the findings to date regarding the Tolerability and the potential of the deal went technology platform clinical trials of euro on unrelated clinical development programs and the company's ability to develop.
Collaborations and partnerships to support business planning efforts.
Forward looking statements are subject to numerous risks and uncertainties many of which are beyond brainstorm is control, including the risks and uncertainties described from time to time in the company's SEC filings. The company's results may differ materially from those presented on today's call company undertakes no obligation to publicly update any of these forward looking statements.
Joining me on the call. This morning, I believe what's the president and CEO Brainstorm, Dr. Ralph current price.
From the Chief Medical Officer, Dr Shah Executive VP, and CFO addition, doctors Stacy Lindbergh executive VP and head of global clinical research is also on the call will be available to answer your questions. During the Q&A session. So now I would like to turn the call over to Mr. Li.
Please go ahead.
Thank you Michael.
Welcome to Brainstorm fourth quarter and year end 'twenty 'twenty earnings call.
Thank you everyone for joining us.
On the calls a day I want to discuss our plans for the year ahead.
The best year was challenging year for all of us.
As we navigate through the COVID-19 global pandemic.
Our commitment as a company.
To make known and available to patients with ALS.
As well as the patients with all the other neuro degenerative disease.
Through our progressive M S.
And the old timer diseases right.
I'm incredibly incredibly proud of our employees.
The business partners non clinical investigators for their commitment and collaboration and working together to advance our clinical programs.
I would also like to acknowledge our patients and caregivers who showed great dedication towards clinical trial.
Despite the pandemic.
'twenty 'twenty was truly a year of great learnings and community building.
Our number one priority right now is to seek a path forward for potential regulatory approval of neuron in a L. S.
We have met with the FDA.
And I put forward a compelling set of data.
The FDA is now reviewing the data we shared we are waiting for their feedback.
You will appreciate that this is a sensitive period that we're in now we need to let conversations play out between brainstorm on the F. D. A before we can report.
Detailed plans.
I will say that definitely has been a very collaborative and willing to review and discuss the phase III data with us.
We will try to respond to the questions from Investor.
Wireless respecting the F D a N the process.
Therefore at this time, we cannot.
Speculate on the timing of that feedback or the likely outcome of the ongoing discussions.
Since the phase III readout.
Our whole team has been laser focused on gaining a sound understanding of the data.
The profit of a meeting with the FDA and the other important partner sales generated even greater understanding confidence and support from day Youll have community.
We have proactively provided the FDA with data and analysis that we're discussing a review of the study.
Since our last Investor call. We have continued to learn from the B C. T O two phase III trial.
With each step with each type of our confidence in the study results have continued to increase.
Please no we're operating with great urgency.
We have tremendous support from leading gain or loss position.
It's efficient.
We have thought to gain objective.
Objective at the around the evidence generated.
We continue to have high conviction the efficacy of neuron in our single greatest motivation as a company is to make neuro and available to patients with Atlas.
In addition to our clinical data review, we're also in active dialogue with FDA around the chemistry manufacturing and controls portion of neurons and regulatory review.
We recently conducted a type C meeting with the agency to discuss future CMT plans from their own in support of our commercial ramp up asphalt guidance other requirements necessary to move forward with all the plans for converting to a semi automated manufacturing process.
We will provide a further update on the topic in the near future.
We have initiated an expanded access program to provide neuron treatments for <unk> patients, who completed the phase III trial and meet specific codes eligible eligibility requirements. This EAP was developed in partnership with M. D E and will take place at the six clinical centers of excellence.
Anticipated in the trial.
Initiating the expanded access program immediately after the phase III trial.
Completion and speaks to the promise of neurons phase III clinical data the urgent unmet medical need for patients and our unwavering commitment to patients. They left physician advocacy and patient community feedback on V. A P has been extremely positive.
At this point I would like to invite them to Ralph turn all Chief Medical Officer Amdocs for states other than they're well ahead of it a global clinical research on the comment on the L. S program Ralph.
Thank you Carmen and good morning, everyone I wanted to express my deep appreciation.
To ALS patients and their families to all the investigators and their teams who conducted the highest quality clinical trials through the Covid pandemic.
Bringing us to this important momentum.
I have to say that our AOS phase III data demonstrates a clinically meaningful slowing of ALS disease progression.
And our biomarker analyses suggest that targeting multiple AOS pathways.
Essentially required to achieve this important clinical outcome.
Is in a L. S neurologist I truly believe we're at a point where neurons can benefit ALS patients and we'll continue to learn from our trial to advance the science of day L. S. I'd like to hand, it off to my colleague Doctor Stacy Lindbergh now.
Thanks, Ralph and good morning.
The recently completed phase two trial and error last with our company's first pivotal trial.
Is a big step for our company and well and through it we generated a rich set of data from a well run and high quality trial.
The evidence generated from clinical endpoint as well as a robust biomarker data has allowed us to engage in meaningful discussions with F. D. A.
And as we've carried out all of our pre specified analyses and have worked to address new questions from the FDA.
We've been able to demonstrate a consistent and meaningful treatment effect.
My own view of the data as a drug developer of 25 years and as a statistician isn't the evidence clearly suggest that narrowing it effective in Atlas.
I'm proud of what we'll be able to deliver to the analyst community.
In light of the new insights into this disease through the trial.
And it is my earnest hope that we can provide.
<unk> and improve the lives of Atlas patients with no round.
I'll turn it back to him.
Thank you Stacey.
Aside from neuron development than a L. S. We have many exciting elements in our business plan that we are progressing in parallel.
Focus on areas of high unmet medical need where we believe our focused scientific approach has the potential to add meaningful clinical benefit.
We have made excellent progress in our neuron clinical program and progressive M. S. Despite some delays in clinical trial enrollment due to COVID-19 health care restrictions, we completed all dosing in our study participants. This past December is a reminder of the trial is looking for a relationship but.
Spinal fluids biomarker specific brain and spinal cord MRI measures and observe functional improvements in progressive Ms patients.
We anticipate top line data by the end of the first quarter 'twenty to 'twenty, one I look forward to share our findings with you once a day can be communicated with.
We're in the process of initiating a new clinical program focused on the development of neuroma as a potential treatment for Alzheimer's disease.
While many of all time of therapies that focus on a single target.
Plus as the well known targets of power better amyloid.
Don't have the ability to simultaneously target multiple relevant biological pathways to bring a comprehensive approach to this complex and multifactorial disease.
Our plan for old timers will begin with a multinational phase two clinical trial in Europe to evaluate the safety and preliminary efficacy of neuron treatment in patients with.
Prodromal Tomorrow disease, who also have specific baseline biomarker characteristics. This will be a 52 week open label proof of concept clinical trial to evaluate neurons 40 participants and we will focus on key biomarkers and clinical outcomes.
We're actively working with the regulatory agencies to finalize the trial design and will begin clinical trial enrollment once we receive final approval.
Lastly, we're excited to recently announce the peer reviewed publication of a preclinical study in the journal stem cell research therapy that highlighted the potential of a neuron derived exosomes based.
Treatment for COVID-19, acute respiratory distress syndrome Xerox.
From the study showed that interest the interest the keel administration of Exosomes, let the statistically significant reduction of lung disease severity score and improvements in several additional clinical relevant.
Induced ards markers.
We're excited about the publication of the proof of concept data and we are actively assessing next steps determine.
Our path forward. This includes discussions with possible partners for development opportunities for that technology.
I'll now turn it over the call over to preterm to discuss the financial freedom.
Thank you Jaime and good morning, everyone. It is my pleasure now to walk you through our 2020 financial results.
Research and development expenses net for the year ended December 31, 2020 were $22 3 million compared to $17 2 million net for the year ended December 31 2019.
And these amounts are R&D grants from the Israel innovation authority, or Iia, and California, T cell behind a medicine or <unk>.
Well its proceeds received from the Israeli hospital exemption pathway, which are recorded as an offset to the expense.
This increase of approximately $5 1 million in research and development expense net year over year was primarily due to increase of approximately $2 1 million in expenses in connection with materials consulting depreciation payroll and stock based compensation expenses and other activity.
A decrease of $1 5 million in proceeds received in connection with treatment of patients under the hospital exemption regulatory pathway.
Decrease of approximately $2 6 million and participation of IAA in term in 2020 under various awarded grant.
And an increase of approximately 269000 per cost related to travel and other costs.
The increase in expenses was partially offset by a decrease of approximately $1 3 million in connection with the phase III clinical trial.
Excluding participation from I am sorry, I'm under the grant proceeds received under the hospital exemption regulatory pathway research and development expenses decreased by 133000 from from approximately $24 7 million in 2019 to approximately $24 6 million in 2020.
General and administrative expenses for the years ended December 31, 2020, and 2019 were approximately $9 4 million and $5 8 million respectively.
This year over year increase of approximately $3 6 million in general and administrative expenses was primarily due to an increase of approximately $2 6 million in payroll and stock based compensation expenses, an increase of approximately $1 million in rent stock costs consulting costs of our Investor Relations and public relations.
Activity and various other expenses.
This increase was partially offset by a decrease of approximately 84000 and travel costs.
Net loss for the year ended December 31, 2020 was $31 8 million or one.
<unk> or $1.07 per share as compared to a net loss of $23 3 million or $1.06 per share for the year ended December 31 2019.
Cash cash equivalents, including short term bank deposits were approximately 42 million at December 31, 2020, compared to approximately 0.6 million at December 31 2019.
On September 25, 2020, we entered entered into an amended and restated ATM distribution agreement with SBB, Leerink and Raymond James for up to 45 million.
During the quarter ended December 31, 2020, we sold an aggregate of 3 million 564385 shares of common stock pursuant to the September 25, 2020, ATM at an average price of $6.10 per share raising gross proceeds of approximately $21 8 million.
During the month of January 2021, we sold an aggregate of 679443 additional shares of common stock pursuant to the September 25, 2020, ATM at an average price of $6 54 per share raising gross proceeds of approximately 4.45 million.
With these capital raises our cash and liquidity as of January 31, 2021 is approximately $45 million.
In addition, we still have approximately 19 million in available ATM capacity.
For further details on our financials. Please refer to our form 10-K filed with the SEC today.
Back to your line.
Okay.
Yeah.
Thank you very much freedom.
Wood from lifestyle will now read the questions we have received from investors.
I will direct the questions Michael.
Michael.
Second time first question, it's neuro and has granted FDA approval.
Do you see it being authorized for only a specific subset of patients or anyone with AOS and no matter of their diagnosis state ready to progression or current symptoms.
So as noted at the start of the meeting.
I cannot speculate on whether or not neuron will be approved or the conditions of that potential future approval.
I can't say that our investigators have reviewed the day to believe that the use should be should not be restricted.
Next question please.
Thank you. The next question is about the subgroup at which had clinically meaningful results from the phase III trial.
The quantity of the subgroup participants and the positive results are good enough for a conditional approval or will it take an additional trial focused on the subgroup of newly diagnosed patients.
Once the SBA stands now after your meetings with them is there a green light to submit for approval how long haul such approval take.
Considering their fast track status.
Stacey.
And yes, we have presented new analyses to the MTA, which evaluates the consistency of the effects observed in this pre specified sub group.
Patients with baseline <unk> scores greater than 35.
The analyses, which we hope to present at a cable event shortly demonstrate consistency of a clinically meaningful effect across a much larger set of patients from the trial.
As we've stated we're in active dialogue with FDA around the regular regulatory path for nerd and I cannot comment or speculate further around the conditions of the eye.
Principal.
Sure. Thank you Mike.
Mike.
The extra requirement for the phase III are stated on clinical trials Gov was in a L. S F or S score of 25 and above and you got when the data were presented for the first time and there was a high percentage of patients that were under 25 at baseline I cant explain why this is a and in the phase III press.
Elyse you stated this clinical trial included more severely affected and that population compared to other recent clinical.
Clinical studies.
Thank you Ralph.
Sure. Thank you so as we mentioned earlier the entry criteria in this study.
As listed on clinical trials Dot Gov is euro.
France was above 25 at screening and not at baseline and I should point out that the screening was 20 weeks before baseline.
So patients were rent randomized met the inclusion criteria is designed so it's correct to say that our D. C. T. As yours are two of our phase III trial.
Included a broader set of patients as measured by the baseline a L. S. F. RSR score compared to for example studies in the Pro Act database, where <unk> 97 per cent of patients are over 25 at baseline.
Thank you Mike.
Our next question do you have an explanation for the high placebo rate.
Was seen in phase III and do you think the run in period of the trial.
It was an issue with patients reporting that what they thought they needed to get into the trial.
Stacy please.
Yeah, we have reviewed patient level data closely and we do not believe that the run in period created an issue related to patients seeking to get into the trial.
In terms of that the placebo effect and it really could come from a combination of factors, including the randomization of patients with more advanced disease.
And ultimately we're not at Liberty to provide any more details around this at this time.
Thank you.
Mike.
Can you discuss the biomarkers in the phase III trial.
Can you correlate the respondent to the biomarker data and then in your opinion, which Biomarkers were the most important.
Thank you Ralph that's for you.
Yeah. Thank you. This is an area that we're we're very proud of as you know that our phase III trial.
It was unique in that we collected seven serial samples that CSF biomarkers for each patient.
And this dataset will provide critically needed a data for the ALS community and to support the mechanism of action of neurons.
Our main finding them is that we observed consistent.
Highly robust and statistically significant CSF biomarker changes, particularly in biomarkers related to neurotrophic factor delivery in other words, the cargo that themselves deliver them into.
<unk> nation, and neuro degeneration, which ultimately is the cause of the motor neuron loss in disability and hear less so the changes in biomarkers appear to correlate with clinical response and may be useful in predicting who respond to treatment if approved.
Specific biomarkers that we have focused on and continue to focus on in our analyses include that Jeff also known as vascular endothelial growth factor, which is produced by your own.
Zero filament light or NFL, which is a marker of the neuro axonal loss or nerve degeneration.
Also phosphorylated neuro filament heavy which is a cousin of neuro filling that line.
And M C. P. One which is a very important marker of.
Inflammation. So we think that we have a very good path forward to understand both the mechanism of action and are correlated with the treatment effect.
Thank you.
But you're right. If you have submitted the BLA from neuron to the FDA at this point.
If not what is the anticipated approximate dates that you intend to submit in Europe and your own BLA.
So no we have not yet somebody that they'd be L. A because we have already shared on this call. We have presented our data to the F. D. A.
And an ongoing active dialogue with them around the regulatory.
Pathway from neurons.
That's all we can say now.
Next question please.
When you do sit within your own BLA will it be to treat all patients are only a subgroup such as those with the ALS FRS, our score of 35 or higher.
Ralph.
Thank you. So we do expect to submit the application for all patients and of course. This is that's kind of mentioned this is subject to feedback from the FDA and we can't comment on that but as we stated earlier in our call. Our investigators believe strongly that our.
Ultimately all patients should be given access to interrupt.
Thank you.
Next question would you. Please provide an update on discussions with the FDA concerning the approval process for neuro did I hear that.
Will the company be required to do any additional trials.
When my a BLA to be submitted and given the priority review promised by the FDA about how long do you think it will take to receive an answer from the agency.
[laughter] so as we've already shared we have presented our data to the FDA and are in active dialogue around the regulatory path.
No wrong, we're not at Liberty to provide any additional details at this time.
I must say that we are thankful to the F D. A.
To allow us to present, our data and to provide ongoing updates as we have them.
Our demonstrating through their actions that this is a priority for them too.
Thank you.
Our next question.
Has the company analyze the phase III trial data in more depth.
Other for example, any subgroups of patients.
In addition to those within AOS FRS score greater than 35 that responded more closely to your expectations and also have you learned what what caused the highest placebo effect.
And how how do you think the biomarker data strength in the case for approval of neurons.
[laughter].
Alright, yes, we absolutely have analyze the day that it got.
And we have provided these days it to the F T a covering of breath of topics, including insights into the treatment effect between urine and plus Hebrew true additional subgroup analyses.
In addition to alias clinical experts, we've also engaged uhm external experts statisticians as we speak to draw highly credible conclusions about the treatment effective mirror and from this trial.
We were regulars will be sharing update from the data with with S. T. A to ensure that they have as transparent have an understanding of the day that is possible.
And as we work with them to define the regulatory past from your room.
The the volume a good day to provide confirmation of neurons mechanism of action.
Which it really is behind the treatment effect that means consistently observed N as important as the the new Rune biomarker response and the relationship with the efficacy data is the lack of biomarker respond to placebos equally important and provides really important context as a rigorous control for this phase three trial.
Okay.
That's such a a day isn't that we've expanded access program could you. Please provide an update on this program when do you plan to start dosing.
Who selects the patients had hardly notify.
Or the producer day to Farber and how many patients will you treat or do you plan to treat and for this program.
Well.
Yes, the protocol for the E. P was developed in very close partnership with the F. D. A to provide access to your own for phase three clinical trial participants, who obviously meet specific out what your ability criteria initially patience <unk>, let's severely affected by L. S as measured by the.
G E O S of her S. R score will be the first to receive treatment.
This approach is informed by the top line data from the phase three clinical trial.
On your own for this E. A P program will be produced at the Dana Farber Cancer Institute as this was a matter of fact from site for phase three.
And we have a very good experience there the total number of patients for the E. P will be determined.
By the clinical sites assessment of those patients who meet the inclusion and exclusion criteria and have retained higher Alice function is determined by the <unk> score other.
The clinical trial sites are responsible for screening and enrollment.
As we mentioned the a P protocol specifies three doses of neurons. The same dosing is the phase three clinical trial.
Finally, I might add that D. E E. P program was initiated the first week of January <unk>.
And we expect the first patience in this program to be a ghost next week.
Wonderful Thank you Mike.
Well that's a question of switching gears relates to the program is progressive multiple sclerosis.
What is your current expectations for announcing top line data from the face to progressive in that study.
Well the trial provide data on both.
And efficacy what are the endpoints of this study at all the same biomarkers is used in a last thing being evaluated in this study.
Ralph.
Thank you we we expect.
Phase two M S top line data by the end of this quarter.
The study will provide data on clinical efficacy safety and T. C S F and plasma biomarkers.
Including many that were evaluated in our AOS trial, but in addition, we're looking at C O stuff considering biomarkers.
That are unique and very important and progressive M. S. The key endpoints that we will be initially focusing on are obviously safety.
Changes from base line and a test called the time twenty-five put walking test.
Another test of upper extremity function called the nine whole peg test and as I mentioned, the very important biomarker assessments and we'll look at correlations between these between these important clinical and biomarker assessments.
Thank you.
Okay now we have a couple of questions the Alzheimer's program.
First can you tell us if this eight three Alzheimer's trial hasn't begun in Europe.
How many patients have been enrolled and have you begun dosing these patients.
Stacey.
We're in the final steps of initiating our European Alzheimers clinical trial, and we hope to provide one can tell us soon but there have been no no patience.
Next question you.
K O L. Alzheimer's in 2020, but looking at your pipeline. This program is outlet to even as preclinical.
Can you help clarify this just because he decided not to pursue it or for some other reason.
Okay. So we do continue to move forward as we just heard with all clinical development program for old timers in D. U in the pipeline on our website will be updated to reflect the thank you for those comments.
Next question please.
Okay. So one final question and this relates to the COVID-19 Arts program. This investor wanted to congratulate you on the publication of the preclinical results from your Underived Exosomes to treat arts.
Caused by COVID-19 can you tell us provide more information and tell us if you've decided to conduct a clinical trial is this indication.
Fishing.
Well thanks for the kind words, we're excited about the complication as well and we are in the process of reviewing the unmet need in Orange. In addition to other related conditions and based on the very promising preclinical proof of biotech results were considering the optimal business approach uhm, including various part.
Intercept hearing.
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Your first question.
<unk> comes from the line of David thoughts with Zacks investment Research. Please proceed with your question.
Hey, good morning, everybody.
Uhm in regards to the M. S trial. So assuming there are positive resolved from curious if you could talk about what the next steps in that program would be and if you've for C. A possible path to going straight into a pivotal trial.
Very good question. Thank you Ralph.
Oh, Thanks, David and good morning, Yeah. That's a very good question I think we'll be day to driven we'll have to see the the totality of the evidence in other words.
The biomarker changes how they correlate with clinical outcomes and also will be looking at quantitative MRI correlations.
We have pull together probably the top M S experts in the United States and our trial, that's a five sites.
And I think we'll be having conversations with them recently, there's been a lot of interest in cell therapy for M. S N.
And using different types of cells and different approaches realizing that the conventional treatment still leave a huge biological clinical unmet needs. So I think the the interest is there I think it'll be dependent on the strength of the day, then we'll be happy to discuss it with you at a later date once we have the top line data. So look forward to seeing those results.
Okay, Great appreciate the information and for taking the question.
Thank you.
Are there any additional questions.
As a reminder, if you'd like to ask a question. Please press star one on your telephone keypad now as a reminder, if you'd like to ask a question. Please per star one on your telephone keypad now one moment. Please while we pulled for additional questions.
Ladies and gentlemen, we have reached the end of the question and then.
Or session and I'd like to turn the call back to Mr. Heim Lebowitz, Chief Executive Officer for closing remarks.
Yes. So thank you very much not other questions that looks like we have a very good call and we explained everything very well usually we have many additional questions. So I want to thank my team will prepared for the call.
Thank you all for your support for an ongoing support. Thank you very much have a wonderful day.
This concludes today's conference. Thank you for your participation.
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