Q4 2021 Brainstorm Cell Therapeutics Inc Earnings Call
[music].
Greetings, and welcome to the Brainstorm Cell Therapeutics Full Year 2021 conference call. At this time, all participants are in a listen-only mode. As a reminder,
Greetings and welcome to the Brainstorm cell therapeutics full year 2021 conference call.
At this time all participants are in a listen only mode.
As a reminder, this conference is being recorded.
I would now like to introduce your host for today's conference, Michael Wood of Lifesci Advisors. Mr. Wood, you may begin.
I would now like to introduce your host for today's conference Michael Wood of lifestyle advisors. Mr. Wood you may begin.
Michael Wood: Good morning, and thank you for joining the call today with Brainstorm Cell Therapy.
Good morning.
Thank you for joining the call today with that Brainstorm cell therapeutics.
Michael Wood: Before we begin the opening remarks, I'd like to remind listeners that this conference call contains numerous statements, descriptions, forecasts, and projections regarding brainstormed cell therapeutics and its potential future business operations and performance.
Before we begin the opening remarks, I would like to remind listeners that this conference call contains numerous statements descriptions forecast and projections regarding brainstorm cell therapeutics as potential future business operations and performance statements regarding the market potential for the treatment of neuro to Jan.
Michael Wood: statements regarding the market potential for the treatment of neurodegenerative disorders such as ALS and MS.
Disorders, such as Pls NMS sufficiency of the company's existing capital resources for continuing operations in 2022 and beyond the.
Michael Wood: sufficiency of a company's existing capital resources for continuing operations in 2022 and beyond.
Michael Wood: safety and clinical effectiveness of the NeurOn technology platform, clinical trials of NeurOn and related clinical development programs, and the company's ability to develop strategic collaborations and partnerships to support its business planning efforts.
The safety and clinical effectiveness of the DRM technology platform.
Trials in Europe , and related clinical development programs and the company's ability to develop strategic collaborations and partnerships to support its business planning efforts.
Michael Wood: Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond the brainstorm's control, including the risks and uncertainties described from time to time in its SEC files.
Forward looking statements are subject to numerous risks and uncertainties many of which are beyond the brain's trumps control, including the risks and uncertainties described from time to time in its SEC filings.
Michael Wood: The company's results may differ materially from those projected on today's call. The company undertakes no obligation to publicly update any forward-looking results.
The company's results may differ materially from those projected on today's call. The company undertakes no obligation to publicly update any forward looking statements.
Joining me on the call today will be president and CEO rainstorm time Leibowitz I'll.
Speaker Change: Joining me on the call today will be President and CEO of Rainstorm, Chaim Leibowitz.
Speaker Change: In addition, Dr. Stacey Lindberg, Executive VP and Chief Development Officer, Dr. Ralph Kern, President and Chief Medical Officer, and Dr. David Setburn, Executive VP and Chief Operating Officer, are also on the call and will be available to answer questions during the Q&A session. With that, I'd like to turn the call over to Mr. Leibowitz.
Ill, let populous interim chief financial officer.
<unk> Doctor Stacy Lindbergh Executive VP, and Chief Development Officer, Dr. Ralph current President and Chief Medical Officer, and Dr. David set burn.
Executive VP and Chief operating Officer are also on the call and will be available to answer questions. During the Q&A session.
With that I'd like to turn the call over to Mr. Leibowitz. Please go ahead.
Thanks, Michael.
Thanks to all listeners for joining us to discuss our full year 2021 financial results.
Leibowitz: Thanks to all listeners for joining us to discuss our full year 2021 financial results and provide.
Provide corporate highlights.
Based on the number one priority continues to be pursuing the optimal path forward to provide broad access to new room for patients with <unk>.
Leibowitz: Based on the number one priority, continues to be pursuing the optimal path forward to provide broad access to Neuron for patients with ALS. As we consider our strategy.
As we consider our strategy.
We will take into account any changes.
Leibowitz: in the regulatory environment that may potentially help us and are acutely aware of increased activity among advocacy groups calling for new medications.
On the regulatory environment that may potentially help us.
And there are acutely aware of the increased activity amongst advocacy groups.
Calling for new medications that can help patients.
Leibowitz: We will be paying close attention to the upcoming FDA Advisory Committee schedule to take place this week on March 30.
We will be paying close attention to the upcoming FDA Advisory Committee scheduled to take place. This week on March 30.
Leibowitz: with which a group of independent scientific advisors will convene to review data and testimony from key stakeholders regarding another investigational ALS drug that is currently being reviewed by the FDA.
Thanks Mitra group of independent scientific advisors will convene to review data and testimony from key stakeholders regarding another investigative memorialist truck.
That is currently being reviewed by the FDA.
Leibowitz: While we can't say how the FDA or its panel of outside advisors will view the data package that has been submitted on this particular drug candidate,
While we can't say, how the FDA or the panel of outside advisors will view the data package that has been submitted on this particular drug candidate.
Leibowitz: It is very interesting and important to note that the agency accepted and granted charter review for an NDA that is based on data from a Phase II trial.
It is very interesting and important to note that.
The agency accepted and granted charter review for an NDA that is based on data from our phase II trial.
This together with the convening of the Advisory Committee in May be a sign that the fda's approach to help improve drug.
Leibowitz: This together with the convening of the advisory committee may be a sign that the FDA's approach to how to improve drugs for neurodegenerative disease such as ALS
But in order to generate a disease such as they are less is evolving.
Leibowitz: Our most significant milestone of the fourth quarter was the peer-reviewed publication of our Phase III clinical data on neuron and ALS in the journal Muscle and Nerve.
Our most significant milestone of the fourth quarter was the peer reviewed publication of our phase III clinical data on the enrollment and the journal muscle and nerve.
The paper reported data from the randomized placebo controlled phase III trial that evaluated the safety and efficacy of repeat doses of neurons.
Leibowitz: The paper reported data from the randomized placebo-controlled phase 3 trial that evaluated the safety and efficacy of repeat doses of neurons.
Although previously announced results show.
Leibowitz: Although previously announced results showed the trial did not reach statistical maintenance on their primary or secondary endpoints, pre-specified and post-doc analysis
The trial did not meet statistical significance on the primary or secondary endpoints pre specified and post hoc analysis.
Leibowitz: showed a neuron treatment effect across both primary and secondary efficacy outcomes in those with less advance.
Showed a neuron treatment effect across both primary and secondary efficacy outcomes and those with less advanced disease.
Leibowitz: Having a peer-reviewed publication in this prestigious journal is important for us.
Having a peer reviewed publication in this prestigious journal is important for us.
Leibowitz: as it provides transparency to our full data set and raises awareness of the potential benefits of Neuron in the broader neurology community. We believe it may also...
It provides transparency to our dataset.
As awareness of the potential benefits of neurology and the broader neurology community.
We believe it may also help us and our regulatory activities.
Leibowitz: As part of our efforts to advance neurons, we have an ongoing initiative to present new data to the ALS and broader neurology communities as it becomes available.
As part of our advance efforts to advance neuro, we have an ongoing initiative to present, new data to the other and broader neurology communities as it becomes available.
We had a number of scientific conference presentations in the fourth quarter of 2021 in recent weeks.
Leibowitz: We had a number of scientific conference presentations in the fourth quarter of 2021 and recent weeks.
Leibowitz: Delivered both by my colleagues here as brainstorming by the clinical investigators we work with
<unk> delivered both by my colleagues here as brainstorming by the clinical investigators.
We work with.
The first the most recent presentation I want to highlight.
Leibowitz: was a late-breaking oral presentation at the MDA Clinical and Scientific Conference, which took place earlier in the year.
While the late breaking oral presentation at the MDA clinical and scientific conference, which took place earlier in March.
This conference with.
Leibowitz: Professor Meredith Sivkowitz, Chief of Neurology at Mass General Hospital and Professor of Neurology at Harvard Medical School.
Thus the merits of Cohen chief of Neurology at Mass General Hospital.
Professor of neurology at Harvard Medical School.
Presented genetic analysis.
Leibowitz: that evaluated how ALS-linked genes and single nucleotide polymorphisms correlated with the response to neurons in our phase 3 study.
That evaluated polyethylene genes in a single nucleotide.
Polymer firms is correlated with a response to <unk> our phase III study.
Leibowitz: The results of these analyses show that in patients carrying one or two copies of a gene called UNC30,
The results of these animals that showed that patients carrying one or two copies of the gene called <unk> 13, there was a statistically significant higher response rates on the loan versus placebo.
Leibowitz: there was a statistically significant higher response rate on the ROM versus placebo. In contrast...
In contrast.
The response rates and the patients who are not carriers of this particular gene were similar between the two groups. These results are very interesting.
Leibowitz: who are not carriers of this particular gene were similar between the two.
Leibowitz: These results are very interesting as they suggest neuron treatment may have more of an influence on disease progression in ALS patients who possess certain high-risk genes. This provides a basis for further.
I suggest <unk> treatment may have more of an influence on disease progression.
Patients will possess certain genes.
This provides the basis for further genetic recommendation and may enable that notification of biomarkers that will help us determine which patients are more likely to perform better on the road.
Leibowitz: and may enable the identification of biomarkers that will help us determine which patients are more likely to perform better on
Leibowitz: The last two presentations I'll highlight today are related to biomarker data from the ALS Phase 3 data.
So thats the presentations will highlight today are related to biomarker data from the list.
The data.
Leibowitz: and took place at the International Symposium on ALS MND and the 2021 Northeast ALS Consortium.
And took place at the international Symposium on <unk>.
2021, northeast Payless consortium.
Leibowitz: This presentation was delivered by two of our principal investigators, Dr. Robert Brown from UMass Medical School and Dr. James Berry of Mass General Multidisciplinary ALS Clinic. Collectively, this data show significant neural-driven changes across a range of CSF biomarkers and suggests that the product biological activity across multiple disease pathways could prove important to slowing the progression of ALS.
These are the physical delivered by two of our principal investigator Dr. Robert Brown from Umass Medical School and Dr. Jim Barry Mehserle multidisciplinary clinics collectively this data show significant neuro driven changes across a range of CSF biomarkers and suggest that the product biological activity across multiple disease pathway.
It could prove important to slowing the progression of Airbus.
We also increased our understanding of neurons mechanism of action in ALS and provide additional evidence confirming that north mechanism is linked to its impact on disease progression.
Leibowitz: They also increase our understanding of neurons' mechanism of action in ALS and provide additional evidence confirming that neurons' mechanism is linked to its impact on ALS disease progression.
Leibowitz: alongside our pursuit of the optimal path forward for neurons and ALS.
Hello side, our pursue of the optimal path forward for new roles in AOS.
Leibowitz: We also continue to make progress in establishing our manufacturing preparedness.
We also continued to make progress in establishing our manufacturing preparedness.
Leibowitz: We had previously entered into a partnership with Catalan to provide GMP clinical supply of Neuron in anticipation of the product's potential regulatory approval.
We had previously entered into the partnership with <unk> to provide GMP clinical supply of neuron in anticipation of the product potential of regulatory approval.
Leibowitz: We announced in December that the technology transfer at Catalan's facility had been finalized, a very important step.
Announced in December that the technology transfer of Carolyn facility has been finalized.
A very important step in this process.
Leibowitz: The manufacturing of cellular therapies, such as neurons, is complex and requires careful planning and very specific expertise.
The manufacturing of cellular therapies, such as neuro is complex and requires.
Careful planning, it's very specific expertise.
Leibowitz: We are very pleased with the progress we are making at the Catalan, which has industry-leading capabilities in this area.
We are very pleased with the progress we've been making gift catalog, which has industry leading capabilities in this area.
Leibowitz: Also in December , we provided an update on our Expanded Access Program for Neuron and announced that the FDA has asked us to submit a protocol amendment to provide additional doses for patients who are participating in the EAP under the original EAP protocol. Participants who have completed the PACE Neuron trial and who met specific eligibility criteria.
Also in December we provided an update on our expanded access program for neuron and announced that the FDA has asked us to submit a protocol amendment to provide additional doses for patients who are participating in EAP under the original EAP protocol participants have completed the phase II trial in <unk> specific eligibility criteria.
Leibowitz: had the opportunity to receive three doses of Neuron under the amended EAP protocol, these eligible participants will receive up to three additional doses, and so have the opportunity to receive as many as nine doses of Neuron in total. Three during the Phase 3 trial, three under the original EAP protocol, and three more under the amended protocol.
Had that facility through Q3 doses on the amended protocol. These eligible participants who will receive up to three additional doses and still have the opportunity to receive as many at $9 lower on the total fees during the phase III trial 300 original EAP, which our goal is to be more onto the amendment.
Leibowitz: This will allow for additional data collection that will help us better understand the potential benefits of longer term.
To allow for additional data collection that will help us better understand the potential benefits of longer term treatment.
Finally, there was a recent IP update on want to mentioned in February we were granted a new patent in Brazil covers new ones manufacturing process is a key element of rent from the overall strategy is to establish a broad intellectual property portfolio to protect our proprietary technologies and products, adding good Brazilian pattern to our existing portfolio.
Leibowitz: Finally, there was a recent IP update I want to mention. In February , we were granted a new patent in Brazil. It covers Neuron's manufacturing process. A key element of BrainStorm's overall strategy is to establish a broad intellectual property portfolio to protect our proprietary technologies and products. Adding this Brazilian patent to our existing portfolio of US, Canadian, European, Israeli, and Japanese patents should position us well to enter into new commercial partnerships for Neuron in South America as well and worldwide.
Canadian and European Israeli on Japanese patents should position us well to enter into new commercial partnerships for neuron in South America, as well and the worldwide.
Speaker Change: I'll now turn over the call to Alla Patlis, who will review our financials. Alla.
I'll now turn over the call to all up are pleased who will review our financial follow up.
Thank you hi, It is my pleasure now to walk you through our full year 2021 financial results bring some kick cash equivalents and short term bank deposit Laura approximately $22 1 million as of December 31st 2021. These comparisons approximately $41 9 million on December <unk>.
Alla Patlis: Thank you, Chaim. It is my pleasure now to walk you through our full year 2021 financial results.
Alla Patlis: Brainstorm's cash equivalents and short-term bank deposits were approximately 22.1 million as of December 31st, 2021. This compares with approximately 41.9 million on December 31st, 2020. Our research and development expenditures net in the year and December 31st, 2021 were 15.3 million compared to 22.3 million for the year and December 31st, 2021.
First 2020 already search and development expenditures net in the year ended December 31st 2021 were 15.
<unk> $15 3 million compared to $22 3 million for the year ended December 31st 2020.
Alla Patlis: Excluding participation from IAA and other grants and proceeds received under the hospital exemption regulatory pathway, research and development expenses were $15.8 million in 2021 compared with $24.6 million in 2020.
Excluding participation from IAA and other grant and proceeds received on the hospital exemption that regulatory pathway.
Research and development expenses were $15 8 million in 2021, compared with $24 6 million in 2020.
Alla Patlis: General and administrative expenses for the years 2021 and 2020 respectively were $9.3 million and $9.4 million.
General and administrative expenses for the year 2021, and 2020, respectively about $9 3 million and $9 4 million net loss for the year ended December 31st 2021. Thank.
Alla Patlis: Net loss for the year ended December 31, 2021 was $24.5 million or $0.68 per share, as compared to a net loss of $31.8 million or $1.07 per share for the year ended December 31, 2020.
$24 5 million or 68 per share.
<unk> net loss of 31 8 million or $1 <unk> per share for the year ended December 31st Twenty-twenty.
Thanks for your time.
Speaker Change: Thank you Alaa, Mike Ford from Lifestyle will now read questions we have received.
Thank you Ella mic forwards from lifestyle will now read the questions. We have received from investors.
Mike Ford: Thank you time and the first question is regarding the regulatory strategy for neuron This investor would like to know when do you intend to file a PLA for neuron and ALS?
Alright, Thank you time and the first question is regarding the regulatory strategy for neuron.
This investor would like to know when do you intend to file a BLA for neuron in AOS and can you explain why you did not share more details on our regulatory strategy on AOS up to this point.
Mike Ford: Can you explain why you did not share more details on the regulatory strategy on ALS up to this point?
Yes, Thank you Michael I'll take this one.
Mike Ford: We've had a very productive year and have been good stewards of the talents and resources we have at Brainstorm.
<unk> had a very productive year and have been good stewards of the talents and resources, we have at brimstone.
We're continuing to invest in our future of keeping our sites on the long term, while delivering on the near term priorities.
Mike Ford: We continue to invest in our future, keeping our sights on the long-term, while delivering on the near-term priorities.
Mike Ford: We've given more detail at this time, just two days before a scheduled FDA advisory committee, that will be discussing another product for ALS patients.
To give more detail at this time, just two days before scheduled FDA Advisory committee that will be discussing another product trail of patients.
Mike Ford: may turn out to be counterproductive and not business smart.
May turn out to be counterproductive and not.
And this marks.
We remain steadfast in our belief of neurons ability to combat this terrible disease.
Mike Ford: We remain steadfast in our belief of neurons' ability to combat this terrible disease.
Some of our highly regarded pi.
Mike Ford: Some of our highly regarded PIs, principal investigators, who are close to our data and have cared for participants in our trial firsthand.
Principal investigators who are close to our data and have cared for participants in our trial firsthand.
Mike Ford: have voiced their belief at various scientific conferences on the efficacy and safety of neurons.
Have voids.
Belief at various scientific conferences in the efficacy and safety of neurons.
Mike Ford: It made public statements calling for access to neurons in parallel to generating additional evidence of its clinical benefits.
We have made public statements clothing for access to neuro and in parallel to generating additional evidence of clinical benefits.
We have done the job of allowing the evidence.
Mike Ford: that supports our product to be evaluated by the Scientific Immunity.
That supports our product to be evaluated by the scientific community.
Mike Ford: First, by publishing a full and transparent analysis over face-to-face data and muscle and nerve.
First by publishing a full and transparent balance with our phase III data in muscle and nerve.
Okay respected peer reviewed journal and have guided by the FDA, we have presented and continue to present.
Mike Ford: a respected peer-reviewed journal, and as guided by the FDA, we have presented and continue to present our data and seek input from neurologists, statisticians, and other stakeholders. Thank you.
Our data.
And seek input from neurologists.
That decision.
And advocacy organizations around the world.
We are gaining very valuable and important feedback from this process.
Mike Ford: We are gaining very valuable and important feedback from this process.
We will continue to work with the FDA.
Mike Ford: to determine the best path forward for neurons. Our aim is to seek.
To determine the best path forward for neuron.
Our aim is to seek the fairest way.
Mike Ford: to make Neuron available as soon as possible for as many ALS patients.
To make neuron available as soon as possible for.
For as many patients as possible and at the same time and create value for our stakeholders.
Mike Ford: and at the same time create value for our stakeholders.
As mentioned.
Mike Ford: Later this week, we'll have an opportunity to learn through an advisory committee, how the FDA and the advisory committee will apply the 2019 FDA ALS Guidelines document for therapy development that reiterated the need for urgency and...
Later this week, we will have an opportunity to learn through an advisory committee.
The FDA and the Advisory Committee will apply to 2019 FDA guidelines documents for therapy development.
It reiterated the need of urgency and regulatory flexibility.
We believe this meeting will provide important information to brainstorm and the community at large that may be able to further guide our decisions for the best way forward.
Mike Ford: We believe this meeting will provide important information to brainstorm and the community at large that may be able to further guide our decisions for the best way forward.
In summary.
Let me be clear.
That we do remain committed to advancing this program fully outpaced ALR patients and pursuing the best in <unk>.
Mike Ford: that we do remain committed to advancing this program for ALS patients.
Mike Ford: in pursuing the best and most expeditious path forward to enable patient access.
Expeditious path forward to enable patient access.
Mike Ford: Next question. You referred to the UNC13A gene in your press release about the MDA.
Next question you referred to the U N C. <unk> in your press release about the MDA presentation could you. Please explain exactly what the gene is and what its relevance as to AOS pathogenesis.
Mike Ford: Can you please explain exactly what the gene is and what its relevance is to ALS pathogenesis?
Thank you Ralph to take this one.
Of course.
Mike Ford: Of course, the hallmark feature of neurodegenerative diseases, including ALS, is the depletion of…
Hallmark feature of Neurodegenerative diseases, including AOS is the depletion of.
Mike Ford: the RNA binding protein, TDP43, from the nucleus of neurons in the brain and spinal cord.
The RNA binding protein TDP 43 from the nucleus of neurons in the brain and spinal cord.
Mike Ford: There are genetic variations called single nucleotide polymorphisms in this gene, and they're among the strongest hits associated with ALS in human genome-wide association studies.
There are genetic variations called single nucleotide polymorphisms in this gene and they are among the strongest hits associated with AOS and human genome wide Association studies.
Mike Ford: In fact, a direct link has been found between UNC13A risk allele and the deleterious effects of TDP43. And a recent paper suggests that a single nucleotide polymorphism in this gene may underline differences in treatment response.
<unk> a direct link has been found between <unk> risk allele.
The deleterious effects of TDP 43, and our recent patient paper suggests that a single nucleotide polymorphism polymorphism in this changing my underlying differences in treatment response.
Mike Ford: From a broad look at the literature, we know that UNC13AC allele is associated with a higher age at symptom onset, a more frequent bulbar onset, high incidence of ALS, lower breathing function, shorter survival, and lower scores on ALS-specific cognitive tests.
From.
A broad look at the literature, we know that <unk> is associated with a higher age of symptom onset.
And more frequent ball bar onset high incidents of AOS and lower breathing function shorter survival and lower scores on AOS specific cognitive tests and we know that published data suggests that the <unk> on this genetic variation contributes to the clinical heterogeneity found in AOS.
Mike Ford: And we know that published data suggests that the C allele on this genetic variation contributes to the clinical heterogeneity found in ALS, including the risk of being diagnosed with ALS and having a worse prognosis with the disease.
Including the risk of being diagnosed with AOS and having a worse prognosis with the disease. In fact retrospective analysis from 2017 published in neurology across trials.
Mike Ford: In fact, retrospective analysis from 2017 published in Neurology Across Trials.
Mike Ford: noted that there was patients with a C-risk allele of this gene responded to lithium carbonate compared to those who received placebo. This analysis led us to pre-specify this gene and this genetic variation in our phase three trial. And as Haim just mentioned, Dr. Merit Sutkiewicz from Mass General presented these data at the recent MDA conference in a late-breaking session just a few weeks ago.
Noted that there was a patient with a C risk allele.
This gene responded to lithium carbonate compared to those who received placebo. This analysis led us to pre specified this team and its genetic variation in our phase III trials.
And as I just mentioned Dr marriage, Succor, which from mass General presented these data at the recent MDA conference in the late breaking session just a few weeks ago.
Speaker Change: And what she shared was that neuron-treated participants with the AC genotype were almost nine times more likely to respond to neuron treatment compared to placebo. And these results offer great promise for the development of future treatments for ALS in addition to accumulating evidence for the effectiveness of neuron. If you're interested in reviewing the presentation, you can access it on the investors and media section of our company website.
And what she shared with the neuron treated participants with the AC genotype, where almost nine times more likely to respond to neuron treatment compared to placebo and these results offer great promise for the development of future treatments for AOS. In addition to accumulating evidence for the effectiveness of neurons and if your interest.
In reviewing the presentation you can access it on the investors and media section of our company website.
Thank you Darryl.
Next question can you help us understand what youre gaining from all of these presentations at scientific conferences regarding the Biomarkers and genetic results on UN <unk> and how do you think this helps you with your regulatory path going forward.
Speaker Change: Next question, can you help us understand what you're gaining from all these presentations at scientific conferences regarding the biomarkers and genetic results on UNC 13A, and how do you think this helps you with your regulatory path going forward?
Stacy.
Sure.
Speaker Change: So while our early efforts focused on clinical outcomes from phase three, this question notes that we've continued to broaden the focus of neurons effectiveness by presenting biomarker and genetic data, both of which add to the compelling case for neurons.
So well.
Efforts focused on clinical outcomes from phase III.
Note that we've continued to broaden our focus.
And the effectiveness by presenting biomarker and genetic data.
Both of which add to the compelling case for Gary on the <unk>.
Speaker Change: The bimarker data presented at scientific meetings and to the community shows that neuron decreases markers of neuroinflammation and neurodegeneration, while increasing markers of neuroprotection.
Margaret data presented at scientific meetings and to the community shows that Darren decreases markers of inflammation in neuro degeneration, while increasing Margaret on your protection.
Speaker Change: And furthermore, the changes in biomarkers observed with neuron, which are not observed with placebo, can even help explain with great accuracy the clinical response that we observed in our phase 2 trial using pre-specified statistical models.
And Furthermore, the changes in Biomarkers observed with Darrin, which are not observed with placebo can even help explain the great with great accuracy. The clinical response that we observed in our phase II trial, using Prespecified statistical model.
Speaker Change: Roth just referred to some of the really exciting and emerging literature, and we're living in a very exciting time in terms of the ability to learn and understand insights into ALS. From a genetic perspective, what we're seeing focusing on UNC 13-A certainly is one of
Rob just referred to.
Some of the really exciting and emerging.
Literature, and we're living in a very exciting time.
In terms of the.
The ability to learn and understand and tightened the AOS and <unk>.
The genetic perspective, what we're seeing focusing on an UN <unk> certainly is one of these dimensions.
Speaker Change: And as our analysis was informed by past literature, we felt it was important to share with the scientific community our own pre-specified genetic analyses of our neuron phase 3 trial, which suggested and suggests that the neuron treatment in this trial may influence disease progression in ALS patients who possess UNC13A risk allele and provide a basis for further genetic characterization in future clinical trials.
And as our analysis was informed bypass literature, we felt it was important to share with the scientific community our own pre specified genetic analyses of our neuron phase two trial, which suggested and suggest that <unk> treatment. In this trial may influence and disease progression in ALS patient to possess you in <unk>.
Risk allele and provide a basis for further genetic characterization and future clinical trials.
Speaker Change: If we step back and we look more broadly at our clinical plan, we've delivered a series of four clinical trials in ALS.
If we step back and we look more broadly at our clinical plan, we delivered a series of four clinical trials in AOS, including double blind and controlled phase II and phase III clinical trials.
Speaker Change: including double blind and controlled phase 2 and phase 3 clinical trials.
Speaker Change: So if I think about this question, so why are these presentations important? Well, these pre-specified biomarker and genetic data add additional layers of credibility and scientific rigor to our findings related to the functional clinical endpoints that we've published from these trials.
So if I if I think about this question to why are these these presentations important piece.
These pre specified biomarker and genetic data add additional layers of credibility and scientific quicker to our findings related to the functional clinical endpoints that we've published from this trial.
Thank you so much spacing.
Michael.
Speaker Change: Next question, if AMX0035 is approved by the FDA, how do you think this will affect the market opportunity for Neuron?
Next question is Amex 0035 is approved by the FDA.
How do you think this will affect the market opportunity for <unk>.
Zero.
Stacey.
Speaker Change: It would not be appropriate for us to comment on the likelihood of approval of AMX 0035.
It would not be appropriate for us to comment on the likelihood of approval of Amex is there is there are three five.
And we believe that each therapy should be evaluated on the scientific data and evidence generated individually yet as a part of the analyst community. We will celebrate all success in terms of treatment becoming available patients.
Speaker Change: We believe that each therapy should be evaluated on the scientific data and evidence generated individually. Yet, as a part of the ALS community, we will celebrate all success in terms of treatment becoming available.
Speaker Change: Thanks. The next question is regarding the Progressive MS program. Can you please provide an update on this program?
Thank you. The next question is regarding the Progressive MS program can you. Please provide an update on this program.
Short of the current Ralph.
Speaker Change: Yeah, thanks for the question. So, as you know, we've completed additional analyses of the phase 2 study that will be shared at upcoming scientific meetings and in a peer reviewed publication, just to give you a little, a little color on this issue and 3 examples, the scientific abstracts that will be shared at the upcoming.
Great. Thanks for the question.
So as you know we've completed additional analyses of the phase II study that will be shared at.
Upcoming scientific meetings and in peer reviewed publication just to give you a little a little color on this issue.
Three examples the scientific abstracts that will be shared at the upcoming <unk>.
Speaker Change: CMSC meeting in May provide important information on three areas. One is the quantitative relationship between neuron treatment and CSF inflammatory biomarkers.
MFC meeting in May provide important information on three areas. One is the quantitative relationship between neuro and treatment in CSF inflammatory biomarkers.
Speaker Change: A second is a closer examination of visual outcomes, looking at individual eye responses in special.
Second is a closer examination of visual outcomes looking at individual I responses in.
In study participants and finally, we did preclinical work that we'll be sharing on the interaction of neuron with <unk>, which is S.
Speaker Change: And finally, we did preclinical work that we'll be sharing on the interaction of neuron with staponimod, which is a S1P receptor modulator and a recently approved MS therapy. So, as you can see, we're continuing very active dialogue with the MS scientific community and experts. We're also continuing our dialogue with regulators about the optimal path forward, and we'll be prepared to share details after publication of our phase two data. And thank you, Ralph.
<unk> receptor modulator and a recently approved MS therapy. So as you can see we're continuing very active dialogue with the scientific community and experts. We're also continuing our dialogue with regulators about the optimal path forward and we will be prepared to share details after publication of our phase II data.
Thank you Ralph.
Thanks, and regarding the expanded access program.
Ralph Kern: Did the FDA contact Brainstorm to request the latest EAP, or did this happen the other way around?
The FDA contact brainstorm to request the latest EAP.
Did this happen the other way around.
So, yes, FDA requested and authorize the breath of extended dosing period for the participants who have completed AEP, but by allowing.
Ralph Kern: So yes, FDA requested and authorized advanced stomach standard dosing period for the participants who have completed AAP by allowing three additional doses of Neuron.
Three additional doses of neuron administered every two months.
As a follow up to that question is brainstorm actively collecting the usable data from the EAP.
Ralph Kern: As a follow-up to that question, is Brainstorm actively collecting usable data from the EAP?
Okay.
Hi, Yes, I'm happy to confirm Brainstorm is activated quoting both clinical and biomarker information than the E&P.
Speaker Change: Yes, I'm happy to confirm BrainStorm is actively collecting both clinical and biomarker information.
Speaker Change: Thank you very much, Michael. I think that's done with the written questions we included today. Holly, would you open for any questions from the audience, please? Certainly.
Thank you Barry it's much Michael I think that some of the written questions. We included today.
Hollywood you open for any.
Any questions from the audience.
Certainly.
Ladies and gentlemen, the floor is now open for questions. If you have any questions or comments. Please press star one on your phone at this time.
Holly: If you have any questions or comments, please press star 1 on your phone at this time.
Holly: We ask that while posing your question, you please pick up your handset if listening on speakerphone to provide optimum sound quality. Please hold while.
We ask that while posing your question you. Please pickup your handset listening on speaker phone to provide optimum sound quality. Please.
Please hold while we poll for questions.
Speaker Change: Your first question for today is coming from David Bautz. Please announce your affiliation, then pose your question.
Your first question for today is coming from David <unk>.
Please announce your affiliation then pose your question.
Okay.
David Bautz: This is David Vowles from Zach's Small Cap Research.
David the outs from Zacks small cap research.
David Bautz: Thanks for the update this morning, and Haim, I'm curious, now that the Phase 3 results have been published, are you receiving any additional feedback from physicians, patient advocacy groups? Maybe if you could tell us a little bit about what you're hearing from people that have been able to see the full data set.
Thanks for the update this morning and hi.
Curious now that.
Phase III results have been have been published.
Are you receiving any additional feedback from.
Physicians patient advocacy groups, maybe if you could tell us a little bit about what youre hearing from people.
To see the full dataset.
Alright, Thank you David.
David Bautz: As you can figure out from our call today, I know many investors want us to talk more.
As you could figure out from our call today I know many investors want us to talk more.
David Bautz: But it's business-wise and regulatory-wise better we talk less.
But it's business wise and regulatory wise, better we talked less.
David Bautz: We're at a time that we're working very hard for the ALS community, and it's maybe not obvious exactly what we're doing, but you just mentioned part of it. We are talking to many stakeholders.
We're at a time that we are working very hard for the Lf community.
Maybe not obvious exactly what were doing but you just mentioned part of it we are talking to many stakeholders.
David Bautz: And yes, we are getting very good feedback and vast majority very positive feedback.
And yes, we are getting very good feedback and then vast majority of very positive feedback.
David Bautz: And there's strong recommendations, which we can't share. We don't think it would be right to share. Specifically, you know, we're in a very sensitive week, and we know this for months, that this outcome is happening this week.
And the strong recommendations, which we can share.
We don't think it will be right to share.
Specifically, we are in a very sensitive weekend, we noticed four months. So this outcome is happening this week.
David Bautz: And it doesn't make any sense that we talk more publicly at this time. I know you're an analyst.
It doesn't make any sense that we talk more publicly at this time.
I know Youre, an analyst tell me if I'm wrong.
Speaker Change: No, I completely understand. So, since you can't talk all that much about what the FDA is doing, maybe I could ask, are you considering going to other jurisdictions and approaching them about approving or about filing for the approval based on your results so far?
No I completely understand.
So.
Since you can't talk all that much.
About what the FDA is doing.
Maybe I could ask are you considering going to other jurisdictions.
And approaching them about approving.
Filing for approval based on your results so far.
That's a tricky one.
So.
Yes.
Yes.
Speaker Change: You know, I understand the questions coming from because other companies did go that path.
I understand the question is coming from because other companies did go that path.
Speaker Change: Definitely we're considering all paths. We know what's going on. We're following everyone's path.
Definitely we are considering all paths, we know what's going on with following everyone's path.
Speaker Change: And we're trying to understand what those interactions did to help.
And we're trying to understand what those interactions did to help.
There were forward or not.
Speaker Change: And not what it seems, obviously, always is the back story.
And not what it seems obviously always at the back story as you know.
Speaker Change: So we're learning that very, very diligently, and we think we know where we are.
So we're learning very very diligently and we think we know where we are.
And.
When we will make a move you'll hear it.
Speaker Change: That's our decision. Rather than discussing an up-front strategy, we are, as I said,
That's our decision rather than causing upfront strategy.
<unk>.
We are as I said, we are getting prepared for every scenario.
Okay.
Speaker Change: Okay, and then my last question is, so outside of the expanded access program through the FDA, is anyone else receiving neuron treatment at this time?
Okay.
And then my last question is so outside of the expanded access program through the FDA is anyone else receiving neuron treatment at this time.
No.
Speaker Change: There's huge activities at the Cattleman site just to get them ready for success.
Okay.
Huge activities at the <unk> side, just to get them ready for success.
Speaker Change: as a manufacturing center, so a lot of activity outside of the providing just DAP patients.
As a manufacturing center.
So a lot of activity outside of that providing just a few.
Patients, but only the EAP patients that are getting their own for now.
Speaker Change: but only the AP patients that are getting their own for now. Okay, great. All right, well, thanks.
Okay, Great Alright, well, thank you for taking the question.
Sure next question please.
Speaker Change: Once again, if there are any questions or comments, please press star one on your phone at this time.
Once again, if there are any questions or comments. Please press star one on your phone at this time.
Speaker Change: Your next question is coming from Michelle Lorenz. Please announce your affiliation, then pose your question.
Your next question is coming from Michelle Llorens. Please announce your affiliation then pose your question.
Michelle Lorenz: Hi, I am with Voices for ALS, and I noticed a couple weeks ago that Merit Sikovic talked about your iPSC cells, that you were going to be doing some confirmation of UNC13A. Can you talk a little bit more about that? I didn't hear you discuss it on the call.
Hi, I am with voices for ALS and I noticed a couple of weeks ago that Matt just talked about.
Your Ips sea gulls that youre going to be doing some combination of UMC start Q&A can you talk a little bit more about that I didn't hear you.
You discussed it on the call.
Yes, we did but if you want to take this one.
Speaker Change: Yes, I'm happy. I'm happy to. So I think this was part of an interview that that she participated in and it was a discussion really around how we can leverage genetic insights.
Yes, Im happy Im happy too. So I think this was part of it and interview.
But she participated in and it was the discussion.
Really around how we can leverage genetic insights and further research on too.
Speaker Change: and further research to help us understand the implications of results in clinical trials, but then also to think about the future trials. And the focus was really with the materials that we have now, how can we provide additional confirmation of the results that we're seeing in this trial. And so we can
Help us understand the implications of results in clinical trials, but then also to think about the future.
Future future trials and the focus was really with the with the materials that we have now how can we provide additional confirmation.
The results that we're seeing in this trial until we can.
Speaker Change: can take materials, genetic materials that we have now, and run additional preclinical experiments, which was really the basis of.
Kent can take material genetic materials that we have now an and Brian additional preclinical preclinical experiments, which is really the basis for conversation.
Speaker Change: And so those are plans that we are undertaking. We are certainly exploring how we maximize the resources we have and bring new additional insights to the table from our phased retreat.
And so those are plans that we are undertaking we are certainly exploring how we maximize the resources, we have and bring new additional insights to the table from our phase III trial.
Speaker Change: Yeah, thank you, Michelle, for that opportunity to clarify that part of the interview. People misunderstood it.
Thank you Michelle for that opportunity to clarify that part of the interview people.
People misunderstood it.
Any other question Michelle.
No. Thank you.
Thank you next question please.
Your next question is coming from John Murphy, John Your line is live.
Speaker Change: Your next question is coming from John Murphy. John , your line is live.
Hi, Thank you I was just wondering on the last conference call. You said you were going to meet with the people familiar with.
John Murphy: I thank you. I was just wondering on the last conference call, you said you were going to meet with the people from ALS.
John Murphy: And after that meeting, you were going to immediately submit the BLA. I was wondering, did you meet with the people of ALS? And if you didn't, why not? Thank you.
After that meeting you're going to immediately submit the BLA I was wondering did.
Did you meet with the people.
And if you didn't find that thank you.
Speaker Change: Thank you very much, John . So, as we said, we are meeting with many stakeholders, clinicians, and yes, we are meeting with patients of ALS.
Thank you very much John So as we said we are meeting with many stakeholders clinicians and yes, we are meeting with patients available.
As well.
Speaker Change: But as we explained again and again on this call, that there's a lot of regulatory activity ongoing now. Therefore, we are not choosing to share the exact strategy of our VLA-5.
But as we explained again and again on this call that there is a lot of regulatory activity ongoing now therefore, we are not choosing to share the exact strategy of our BLA filings.
Speaker Change: I understand the need of investors and the urgency to know what our plans are, but in the favor of the program, to get better chances, to have faster access for patients, we're taking this route. And I just want to reassure everyone with ALS, it's very important for me, that we know and feel the urgency.
I understand.
The needs of investors and the urgency to know what our plans are but in the favor of the program to get better chances.
To have faster access for patients with.
We're taking this route.
<unk>.
I just want to reassure everyone with Alice it's very important for me that.
That we know and field urgency.
I think that what drives me and all of my colleagues.
In and day out.
We worked very hard to produce.
Speaker Change: and create the best possible outcome for ALS patients. Sometimes that calls to lay quiet. ALS patients, in my view, couldn't have a more driven team than this team at Brainstorm.
And create the best possible outcome for Ebola patients.
Sometimes that calls.
So required.
<unk> patients.
In my view Couldnt have more driven.
<unk> then just steam at Brainstorm.
I am very proud of this very devoted team.
And I think all of you shouldn't be.
That's what I can say.
Thank you for the question.
There are no further questions in queue I would now like to turn the floor back over to Mr. Lebovitz for any closing comments.
Speaker Change: There are no further questions in queue. I would like to turn the floor back over to Mr. Lebowitz for any closing comments.
Lebowitz: Yes, Holly, please if you can pull for another question, I just got an email that people are looking and also I see John wants to ask another one.
Yes Holly.
Please if you can pull for another question.
So I've got an email that people are looking and also see John what that another one.
Speaker Change: And if we want to allow him to ask another one, so he can say that we're allowing all his questions. Please, John .
And if you separately, we wanted to allow him to ask another one. So you can say that we are allowing all of these questions.
Please John .
John Your line is live.
John Murphy: Yes, thank you. I saw that Mr. Ness invested quite heavily in Brainstorm. Is there any plans for him joining the board or joining the Brainstorm team? Thank you.
Yes. Thank you I sort of missed the next invested quite heavily embraced storm is there any plans for him joining the board who are joining the brainstorm team. Thank you.
Speaker Change: Thank you for that question, but I cannot comment on behalf of Mr. Ness without getting his approval for that. You must understand that we are very appreciative of Mr. Ness being a major shareholder in Brandstorm.
Thank you for that question, but I cannot comment on behalf of Mr. <unk> health.
And getting an approval for that.
Some sand that we are very appreciative.
Being a major shareholder in brand storm.
But thank you for that question.
And on <unk>.
Although if we can pull for another question.
Speaker Change: Paul, if you can pull for another question, I just had an email that someone else is trying to get out. If you can.
E mail that someone else is trying to get out if you can give them information how to.
Absolutely, ladies and gentlemen, if you have any questions. Please press star one on your phone at this time.
Paul: Absolutely. Ladies and gentlemen, if you have any questions please press star 1 on your phone at this
Your next question is coming from Ken Hackel. Please announce your affiliation then pose your question.
Speaker Change: Your next question is coming from Ken Hackle. Please announce your affiliation, then pose your question.
Ken Hackle: Yeah, I'm with Scopus Holdings. And the question is more on the financial side. I see you've burned through something like 47% of the current assets in the past year. Give me your plans for the current year. What are your thoughts with your capital position, your cash position, what you need to do going forward?
Yes, Im with Scopus Holdings and the question is more on the financial side I see burned through.
47% of the current assets in the past year given your plans for the current year. What are you what are your thoughts with your capital position your cash position going forward.
Yeah very good question. So as you may well know, we havent, we havent opened ATM for up to $100 million.
Speaker Change: Yeah, very good question. So as you may well know, we have we have an open ATM for up to 100 million. We didn't tap into it. And with these prices and these volumes, we are very careful, you can see over the last few years, how careful we are with the ATM activation.
Didn't tap into it.
With these prices and these volumes we are very careful you can see over the last few years, how careful we are with the ATM activation.
Speaker Change: On the other hand, we have enough cash for going forward at least over a year and a half. Our burn rate is really down dramatically, about 30% lower than before. The main reason is because we don't have an ongoing clinical trial now. So we do have manufacturing expenses, but no clinical trials for the moment.
On the other hand, we have enough cash for going forward at least over a year and a half our burn rate is really down dramatically.
Lower than before the main reason because we don't have an ongoing clinical trial now so we do have manufacturing expenses, but no clinical trials for the moment.
Speaker Change: And so we're fine as it is, no pressure whatsoever. Thank you for that question.
So we're finding finance there is no pressure whatsoever.
Thank you for that question.
There are no further questions in queue at this time.
Speaker Change: Okay, Holly, so thank you very, very much and thanks for everyone participating and all the questions.
Okay. Holly so thank you very very much and thanks for everyone participating in all the questions.
Speaker Change: And I wish that we're going to have a very good upcoming quarter, a very exciting quarter definitely to see how.
I wish that we were going to have a very good upcoming quarter.
Very exciting quarter definitely to see how things evolve.
Speaker Change: and I look forward to talk to you and be able hopefully to share far more information with you in the next few. Thank you very much.
And look forward to talk to you won't be able to hopefully share far more information with you in the next few thank you very much.
Okay.
Speaker Change: Thank you, ladies and gentlemen. This does conclude today's event. You may disconnect at this time and have a wonderful day. Thank you for your participation.
Thank you ladies and gentlemen, this does conclude todays event you may disconnect at this time and have a wonderful day. Thank you for your participation.
Yes.