Q3 2022 Brainstorm Cell Therapeutics Inc Earnings Call
Thank you for holding ladies and gentlemen, and please remain on the line to Brainstorm conference event will begin momentarily. Thank you for your patience.
[music].
Greetings and welcome to the Brainstorm cell Therapeutics third quarter 2022 conference call.
At this time participants are in a listen only mode.
A reminder, this conference call is being recorded and now I would like to introduce your host for today's conference Michael Wood of lifestyle advisors. Mr. Wood you may begin.
Hi, good morning, and thank you for joining us before we begin the call with an opening remarks, we'd like to remind listeners that this conference call contains numerous statements descriptions forecast and projections regarding brainstorm cell therapeutics and its potential future business operations and performance.
Statements regarding the market potential for the treatment of neuro degenerative diseases, such as a lesson in that.
The sufficiency of the company's existing capital resources for continuing operations in 2022 and beyond the safety and clinical effectiveness of the neuron technology platform in clinical trials of neurons and related clinical development programs and the companys ability to develop strategic collaborations and partnerships to support their business planning efforts.
Forward looking statements are subject to numerous risks and uncertainties many of which are beyond the control, including the risks and uncertainties described from time to time in the company's SEC filings.
Company's results may differ materially from those projected on today's call and the company takes no obligation to publicly update any forward looking statements.
Joining us on the call today will be <unk>, president and CEO of Brainstorm Doctor Stacy Lindbergh Executive Vice President and Chief Development Officer, Dr. Ralph current President and Chief Medical Officer, and our interim Chief Financial Officer. In addition, Dr. David Sandberg Executive Vice President and Chief operating <unk>.
Or was it also on the call and will be available to answer your questions during the Q&A session.
So now I'd like to turn the call over to Mr. Lee Boothby. Please go ahead.
Thanks, Michael and thank you to everyone for joining us this morning.
As is our usual practice, we will follow our prepared remarks by addressing questions. We received.
Some investors in advance.
As well as taking live questions from those of you listening on the call today.
The main topic, we want to cover this morning is the fda's issuance of a refusal to file letter for the neuron BLA submission.
The letter we received from the FDA contained two topics.
That led to the issuance of the refusal to file letter.
The first is clinical and statistics and the second is CMC or.
Chemistry manufacturing and controls.
On the first topic clinical and statistics the letter basically summarizes.
What was known prior to the filings.
The trial met the primary endpoint.
On the second topic, the litter item items.
<unk> items that we expect to be able to fully remediate.
Returning to the first and arguably the most important topic in the letter.
It is clear that you have.
Do you still seeks.
Substantial evidence defined by statistically significant result on.
The clinical outcomes.
But the primary endpoints.
And anything short of this.
And their view is insufficient.
Evidence for a filing.
We obviously have a different view on the value of the evidence.
That has been generated from the phase III trial.
We continue to believe that the totality of evidence from the phase III trial.
Sufficient to support an approval and represents a significant contribution trail of therapy.
We also believe there needs to be urgency.
And flexibility in the review and approval of treatments for individuals with Alice.
We will have no time to waste.
<unk> remains a uniformly fatal illness.
<unk> typically within two to five years from diagnosis.
The limited number of available treatments have only a modest impact on disease progression.
We will pursue a type a meeting to progress this application in.
And as part of this meeting we will discuss with FDA a path to an FDA Advisory Committee meeting and outcome.
We believe that it is more than reasonable to expect.
That the FDA will allow a fair hearing in an open and transparent settings.
As an FDA Advisory committee meetings.
And that provides an open and fair hearing in a public venue.
The FDA can frame their review of the evidence and all of the other relevant stakeholders can also expressed their view.
Also here a whole medical expert statisticians patients and other members of the AOS community view our data.
This forum will also allow patients physicians and the broader advocacy community to express their views relevant to an approval.
At this point I will turn the call over to Dr stays at Lindbergh our.
Our executive Vice President and Chief Development Officer for some additional comments.
And to provide a review of data share through recent medical conference presentations Stacey.
Yeah.
Thank you Haim.
Let me begin by reiterating my support for the path for that time just outlined.
We remain committed to people living with AOS, and we'll do everything within our power to allow us to process coronary.
As we continue to believe that the totality of evidence from the phase III trial is sufficient to support an approval and represents a significant contribution to Atlas therapy.
We will press forward independently toward process and take the steps necessary to move towards an advisory Committee.
And we are fortunate to have support from some of the most respected Atlas clinicians and researchers because.
Cause indicated to us that they are frustrated by the news of the FDA refusal to file our BLA.
As we shared in previous earnings calls, we continue to have scientific discussions around our data.
And there's a growing belief that the totality of evidence Springer suggest us taking on what the efficacy that should not be ignored.
While the regulatory process remains the primary focus for the company.
Continue to share new data underwriting with the medical community.
Eric two important recent presentations.
One at the Atlas one research symposia and another at the annual meals meeting that I want to highlight.
Both presentations contained new analyses from your own phase III trial.
At <unk>, one we presented data showing that in your own had a consistent effect on biomarkers.
And people with advanced Atlas at baseline in the trial.
And those with less advanced disease.
With no evidence of a floor effect in the biomarker data.
The Neil's meeting we had a poster on new sensitivity analyses and account for the floor effect and the Atlas functional rating scale revised Atlas functional rating scale on the key end points from year end phase III trial.
Both of these datasets add to the robust body of evidence that we believe supports a clinically meaningful treatment for neuro Daniela.
Let me begin by speaking about our presentation I gave at the fifth annual analyst One research symposia that took place in October .
As we've described before seven CSF samples were collected in all participants in the phase III trial.
Your line was shown to decrease biomarkers associated with neuro inflammation, and neuro degeneration and increase Neuroprotective biomarkers over 20 weeks.
Demonstrating its multi faceted mechanism of action.
The new analyses that were shared at Alice one look at the longitudinal trajectory of Biomarkers for the subgroup of participants with baseline Atlas upfront star scores greater than 25.
<unk> to those with a baseline scores less than or equal to 25.
The latter group being those participants that were most likely to be impacted by the floor effect on the Alice FRS our scale.
Consistent patterns were observed across biomarkers in both subgroups.
Specifically, we observed decreases in pro inflammatory and Neurodegenerative markers.
And increases in Neuroprotective and anti inflammatory markers in participants treated with <unk> compared to placebo.
These results indicate that neuron was having similar biological effects on trial participants regardless of the level of disease progression at baseline.
This is important because while it was difficult to accurately assess the treatment effect of neurons in participants who had more advanced disease.
Baseline because of the limitations with the revised ALS functional rating scale the <unk>.
Higher macro data showed that <unk> was actually producing a biological effect and all trial participants.
Furthermore, Prespecified statistical modeling designed to identify biomarkers that have the potential to predict a clinical response with nerang identified biomarkers that spans the three key pathways of neuro degeneration, neuro inflammation and neuro protection.
And the model had good statistical properties.
I'd now like to move on to discuss the poster presented at the recent meeting which featured the result achieved post hoc sensitivity analysis methods looking at the total score threshold in the item level thresholds.
These methods allow us to take orthogonal approaches to better understand the true effect of treatment with neuron observed in the phase III trial compared to placebo.
Directly addressing the floor effect of the Alice Safra, Saar, which confounds the treatment estimate in the trial.
Results generated with each method showed that after controlling for the impact of the Alice efforts our floor effect.
Participants treated with neuro and had a higher rate of clinical response.
And less function lost across 28 weeks compared to placebo.
The treatment response observed in the sensitivity analyses are consistent with results from the pre specified subgroup from the trial.
Which was statistically significant.
At the peak of point O five O level in the secondary end point average change from baseline to week 28.
This presentation was jointly delivered by myself and Dr merits that quits.
Keith of Neurology at Massachusetts General Hospital.
Julian Dorn Professor of Neurology at Harvard Medical School, and director of the Sean Healey.
Shawn M Healy and AMG Center for Atlas, The Massachusetts General Hospital.
Together these data give us new insights into the neuro <unk> phase III data and provide evidence of neurons clinically meaningful effects.
We are pleased to be able to share them with the analyst community.
We believe that the scientific backing of our data will be important to an AD com discussion and will enable better understanding of the evidence that's been generated.
I'll now turn the call over to our President and Chief Medical Officer, Dr. Ralph correct.
Thank you Stacey Theres one other important piece of recent news that we wanted to mention briefly today, which relates to publications.
Our phase II trial of neurons and progressive MFS.
The study was featured in the multiple sclerosis Journal in September of this year.
And was also presented at the 38 Congress of the European Committee for treatment and research in multiple sclerosis also known as <unk>.
Dr. Jeffrey Cohen, the Haynesville, Pryor, and Dow Chair and professor of Neurology to Cleveland Clinic Learner College of Medicine and.
And director of experimental Therapeutics, Melbourne Center for MFS treatment and research.
These positive data published.
Published in a prestigious peer reviewed journal.
In addition to being presented at <unk> is an important step in the evaluation of neurons potential as an innovative therapy and progressed with MFS, a devastating neurological disease with limited therapeutic options.
The study met the primary endpoint of safety and demonstrated improvements in tests of neurological function and cognition as.
As well as important changes in CSF biomarkers that reflect neurons mechanism of action.
Unimportant to Ms disease pathways.
We believe the clinical and biomarker results that were featured in the manuscript provides strong rationale to continue the evaluation of neurons to potentially address the unmet clinical need.
Of these patients.
Who have few therapeutic options.
Furthermore, the biomarker results provide additional support.
For neurons potential as a platform technology with broad applications in neuro degenerative diseases we.
We are currently collecting feedback from MFS experts and regulators, which will inform our plans to advance neuron in progressive Ms.
Before I turn the call over to Al Let Pat list, who will review our financials. Let me conclude my remarks by expressing our sincere gratitude to the FDA for offering to discuss the path forward for neuro and in AOS through a type a meeting which has my colleagues mentioned could offer a path to an AD com. We believe this is the best.
Comfort for patients and caregivers with AOS.
I will now turn the call over to <unk>, who will review our financials.
Thank you Ross.
It is my pleasure now to discuss our financial results for the third quarter ended September 30, then to 'twenty two.
<unk> cash cash equivalents and short term bank deposits or.
Approximately $7 4 million as of the end of September and this compares with approximately $22 1 million on December 31st 2021.
Research and development expenditures net in the third quarter of 2022, or $3 8 million compared to $3 6 million for the comparable period in 2021 general and administrative expenses for the third quarter were $3 1 million compared with $1 7 million.
In the comparable period of 2021.
Net loss for the third quarter was $6 9 million or <unk> 19 per share as compared to a net loss of $5 3 million or <unk> 15 per share for the comparable period in 2021.
<unk>.
Thank you Ella.
Michael look from Lifesize will now read the questions. We have received from investors Michael.
Thanks, Hi, My first question here.
Can you tell us in as much detail as possible, but without jeopardizing your company the shareholders in the future approval of neuron or why the FDA rejected.
BLA the ILS community needs as much information as we can provide so we can support you and respond or respectively to the FDA.
Thank you.
I believe we have covered this question quite a lot of detail in our prepared remarks.
Obviously, we don't think will jeopardize the company by being transparent with the community as you have seen.
However.
I want to say I truly appreciate the wages question as framed.
Specifically the wording around how you can respond respectfully.
<unk> on social media.
But we believe the people living with ALS and their lyft community deserve to her and open professional debate.
And to have their voice heard booked it.
It is critical.
The trend of your view of the decide.
To reach out to FDA.
Alright comments on social media.
We urge you please be respectful.
Although the language is not fair nor helpful.
I thought it was important to say on this call. Thank you Michael the next question. Please.
If you intend to re file the BLA, what additional information or data can you include that would influence the FDA to change their minds and accept the filing.
Please.
Yes.
As we've already stated our next step will be to pursue a type b meeting.
This meeting allows a discussion of the best path forward, which could include two things number one additional data.
Number two a possible agreement for submission and a request for an AD com as stated in our prepared remarks.
Thank you.
Next question what is the expected timing of the type a meeting.
Thank you for your time.
Per the FDA regulations, we have 30 days from the receipt of the refusal to file letter to request a type a meeting.
And the FDA then should schedule. This type a meeting it should occur within 30 days of the receipt of our meeting request.
Thank you.
As we move forward can you comment on any potential learnings that you would apply from the process that preceded <unk> approval in AOS.
Thank you Michael Raphael, you, meaning <unk> Ralph perspective.
Yes. Thanks for the question one of the major learnings from delivery of <unk> approval process.
Is the importance of having an open and transparent discussion among regulators.
Clinician statistician and the broader ALS community recall that the FDA originally instructed delivery of sponsored not to file on the basis of their single phase III trial after.
After further consideration this decision was reversed.
Leading to the delivery of NDA filing.
Then please recall that after this filing the agency issued briefing documents for both AD comps, which contained quite negative views on delivery of delivery of data.
At this first AD Com the committee voted 624 against religion Rio.
Then followed by continued discussion and analysis of data eventually leading to a second AD com, where the agency again issued negative briefing documents.
However, at the second AD Com senior FDA officials opined that the AD comm members should consider the unmet need for people living with ALS.
And as FDA guidelines allow to vote based on the afforded regulatory flexibility.
To consider when sufficient evidence may suffice.
Ultimately the committee voted 17, two in favor of delivery.
Then subsequently went on to gain FDA approval.
Looking back at these events you can see just how powerful and critically important for patients.
<unk> com process can be.
It provides an open forum for the public to hear opinions from professionals on both sides of the debate around data.
Moreover, we see that this debate can ultimately proved to be persuasive with the agency.
And lead to patients gaining access to new therapies.
We therefore remain fully committed to neurons advancements and we feel strongly that having an opportunity to have our BLA filed so that we can discuss our data in the context of an ad com.
The best interest of the AOS community.
Thank you.
Next question would you consider running an additional clinical trial to convince the FDA of neurons clinical effectiveness.
JC placebo.
Thanks for the question as stated in the prepared remarks, we believe that for diseases, such as AOS, which are uniformly fatal with limited treatment options that individuals should have the chance to gain broad access to therapies that suggested clinically important treatment effect.
If clinical trial results are less in absolute.
We believe neuron Pittsburgh criterion, having.
Having said that part of our type a meeting will be to discuss with FDA a possible confirmatory trial.
Thank you.
That with the FDA, having refused to file the BLA.
Is it possible for an outcome meeting to take place.
Yes, Stacy for singles.
Absolutely there are processes in place at the FDA that can allow for our sponsors application to go from a refusal to file letter.
Oh, the ultimately being filed looking advisory committee meeting to follow.
And not only that there are examples with precedent.
However to be clear our next step will be to request and participated in a type b meeting with the FDA and as part of this meeting to discuss all of these issues.
We'll listen to the feedback from FDA and seek insights that allow us to chart the best path forward.
Thank you.
Given this news from the FDA, we'll brainstorm now consider pursuing approvals in other countries.
Ralph.
Yes. Thank you for this question as we previously stated we continue to evaluate opportunities for filings in other countries and jurisdictions, but.
But for now our primary focus is with the FDA.
Thanks.
Our next question what happened with the biomarker data from the Phase III study you've spoken about peer reviewed manuscript for this in the past.
Stacey.
Great question.
Market data from this study is an important part of the body of evidence from the phase III trial.
By macro data as you know from this trial has been presented at numerous scientific meetings all of which can be found in the events and presentations section of our company's website.
We are actively working with leading biomarker experts on a comprehensive scripts that reports on all biomarkers collected.
An early draft of the manuscript exist and getting the manuscript under review in a prestigious peer reviewed journal as one of the highest priorities for brainstorm.
I wanted to take the opportunity while we're discussing the biomarker data to think three important partners for their support of our biomarker study.
The AOS Association.
And the California Institute of regenerative Medicine.
Each of these partners believed in your neuro believed in the importance of developing <unk> markers and invest at times to generate the rich biomarker data that we now have from our phase two trial.
Thank you.
So the next question is from an <unk> patient family member.
This is the time to be perfectly honest with your shareholders and let patients. If you wanted to fight for neuro.
For some strange reason I'm still waiting to be willing to do that time, even though my son, Andrew who completed the phase III at the Mayo clinic.
Died awaiting October 32002, I don't want any other mother to have to watch for child would die.
Be honest with US. Please this is crucial.
Well I guess I'll have to take this one is addressed to me.
Uh huh.
It is heartbreaking to hurt this question.
I regret to share that we receive heartbreaking emails like this all too frequently.
I think this question.
Courage to share.
So thanks.
Please know that we use.
This unimaginable events.
To propel us forward.
These stories each of which represent loved ones gone.
Far too soon of the reason, we won't give up.
Hearings that your son was in our phase III trial.
I want to let you know how grateful we are to you and your son.
For participating in our phase III trial for hoping to generate evidence that we can take forward to the FDA and so the community with the goal of helping others.
We want you to know that we are always honest with our statements. There are times when we have to be silent to allowed to process.
So there is not one of those times.
Today, we are sure shared openly with investors and with all others listening as you have heard.
Thank you for not giving up.
Thank you for your fight on behalf of other mothers and family members.
The next question is regarding the extended access program what is the status of the EAP and where the EAP data submitted to the FDA.
Stacey.
The original expanded access program was designed to provide three treatments with Marin.
All given two months apart.
During the conduct of the program we were approached by the FDA requested to consider expanding the program to include a second period.
We agreed to their request and amended the protocol to include one additional period.
With three additional treatments two months apart.
Which is a total of six treatments across the entire expanded access program.
Regarding the status the first period of the EPS complete in the second period is ongoing.
And since the EAP was ongoing at the time of the BLA filing.
Submitted a summary at EAP data available prior to the filing of our BLA with us.
Right.
Thank you.
And then that's just a financial question how do you plan to fund the company going forward.
Yes, the $1 billion question.
We have an $8 million to $100 million that we can tap into opportunistically and.
In addition, quite a few of our major shareholders are considering supporting the company at this time.
They felt it might be a good time to even out there investments, while supporting the companys potential pathway to approval.
We're also in talks with institutional investors that might see this as an opportunity to get into the ground floor.
Closer to potential pivotal milestones such as an outcome in a possible approval.
The best strategy for finance remains.
Where the main focus of the company is and that is to arrive at an agreement with the FDA to allow an outcome. Once we have triggered this out our burn rate will be in my belief a non issue.
<unk>.
Just one last question from a list of submitted questions can you comment please on Brainstorms exoskeleton technology.
Sure all these things out.
Thank you for that question, our preclinical program of acute and fibrotic respiratory indications.
He is actively ongoing with more positive results from an in vivo study.
And our Lps induced acute lung injury model treated with aerosolize excess phones.
We observed statistically significant higher blood oxygen saturation and treated mice compared to controls.
Are there more we have initiated an exploratory program to screen for inflammatory indications in which the extra Sunday immuno module sorry effects.
Two potential clinical benefits.
Additionally in line with our view of exit films is a platform technology for targeted delivery of therapeutic molecules. We have initiated a program to buying targeting moiety and load the exosomes with bioactive molecules.
Finally, we have delineated the roadmap for <unk>.
<unk> manufacturing and are in active discussions with leading clinicians for planning.
First clinical trials in inflammatory lung conditions.
Thank you.
Holly.
You opened for questions.
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, we ask that while posing your question you. Please pickup your handset listening on speaker phone to provide optimum sound quality.
Please hold while we poll for questions.
Your first question for today is coming from David ballots at Zacks small cap.
Hey, good morning, everybody.
Hi, I'm curious if you could give just a little bit more information about the CMC issues that the FDA brought up.
And perhaps importantly.
Importantly, how costly if they will be costly will those changes need to be.
Thank you very much David so the CMC issues.
There are more trivial issue it does not really costly it's asking some questions about some elevations and some others. Some are already in the BLA and we have to point out where it is summer we anticipated most of the questions. We would see them as review questions. We don't see outside of the clinical commentary any red flags.
Okay. That's good to hear and then I.
I guess this question is for Stacy so you've talked about the floor effect and I guess some data that you presented.
You guys are accounting for that I was wondering if you could just talk a little bit about more what actually is the floor effect and why it needs to be accounted for in the trials.
Yeah, David that's a great question and it has been part of.
Presentations that we've given to really be very clear with what we mean by by the spring reset floor effect.
So what we observe in participants that are starting at baseline we've chosen to focus on the threshold of 25 and below.
Which really represents a set of trial participants who haven't been in.
Our late phase trials.
And what's the experience with ease and we see a pronounced right.
Alice efforts are items that start at the score of zero.
No individual item scale items go from theory to floor.
When you start it theory, you cannot measure ongoing disease progression.
So when this happens the scale is not able to quantify or to measure any disease progression certainly in those questions as well as across the multiple other items that.
And some patients started at day rates. So the overall effect is that the participants are declining this is.
Pedro illness, we know that disease progression continues but the scale is not able to quantify it.
It misrepresent certainly with the primary endpoint, which when you have a change in the rate of decline is labeled a treatment response on the primary endpoint it actually misrepresent the data as a clinical response when it is simply an inability to measure of disease progression. So it's a critical thing.
That we can actually very objectively quantify.
And it's something that does need to be controlled for and to be able to see the endpoints that were pre specified how they are influenced once we control for that data and in fact see that the treatment effect.
As we expected with the powering of the trial is very consistent with the powering of the trial and it is very consistent across.
The lease that we that we do control control this data with different methods.
Okay, Great that's actually really helpful. So thanks for taking the questions. This morning.
Well thank you David.
Your next question for today is coming from Mira Klingenberg, a private investor.
Yes can you hear me.
Yes.
Yes, I just want to state that my son Matthew.
Was in the phase III trial, and he received EAP, one and EAP to he and his wife and the rest of US in the family had never doubted that neuro and helped him stabilize and improve for periods of time.
An AD com is absolutely necessary, we continue to fight every day for neuron, we think brainstorm for your continued work.
And Tonight and for not abandoning a L S.
We are here to support.
Oh the process. Thank you.
Well I guess, it's not a question of thank you very much for your comments.
Appreciate it.
Your next question for today is coming from Cowen Morris, a private investor.
Good morning, everybody. My name is Laura asked my mom was in that bank term trial as well as the EAP.
I wanted to first thank you all for your help in my family getting to keep my mom for extra a couple of years, we have no doubt along with.
And a million cards that this helped my mom.
Believe in the following and also and her and her breathing and pres.
We Unfortunately lost my mom two months ago that was a that.
August 2008, so my question here because my mom.
Certainly at the tail end of this disease ALS.
A L. S. F Rsi was well below 25, when entering the EAP I just wanted to go over the Biomarkers once more.
Can you confirm that brainstorm found changes in all biomarkers, even for people at Mum's L. L. S FRS.
Jaime do you want me to take that please stay CSI with them yes.
Yes.
You for the question, Colin and yes I am.
April to confirm your understanding is exactly right and it was a very important finding cross to be able to present in the scientific literature that we see consistent pattern.
And in our Biomarkers neuron treated persistence levels of inflammation decreasing levels with no protection increasing.
So I've neuro degeneration, increasing these we're seeing consistently.
And participants that were in the lower end of the scale.
Similar to where your mother into the trial as well as participants that had higher value.
Very consistent patterns across the biomarker data.
Awesome I do have one.
My question, where are there any difference in biomarker Steven people with higher scores.
I guess above 35 versus those below 25 like my mom.
And I think it's similar question to what you asked before and maybe what I'll, what I'll add in.
When we when we uncovered that we don't see differences in the baseline values in the in across the Biomarkers across not only these these important pathways that are really across every time a car that we collected then it becomes very powerful because we're able to present the biomarker data in total for all participants.
If you look at the presentations, we've given youll see that these are all trial participants and we believe that's the best way to represent represent the data.
What's also very important in our in our in our data and I shared this in the prepared remarks.
But when we look at connecting.
Biomarker data with the clinical data and this was a pre specified.
<unk> analysis, which is very important.
We had a separate statistical analysis plan that was submitted to the FDA to govern our analysis of the biomarker data and really the importance of the biomarker data relative to the clinical outcomes.
And when we look at your own treated participants and we look at the biomarker data we find through them.
And powerful statistical model that there are three key biomarkers that explain or predictive of clinical outcomes observed in the trial. So what that means is these changes in these biomarkers are important and they're relevant to the clinical outcome.
Thank you Stacy I appreciate it and again. Thank you to you all truly I believe that you gave us years extra life My mom's that thank you.
Sure. Thank you.
Your next question for today is coming from Tiago Wilkerson.
Hi, Thanks for taking my question.
If there is a <unk>.
<unk> to maybe narrow the scope of the application to support the Alysa first.
Patients with a score of at least 26 and above it seems like the data supports that strongly and if theres any.
If there is any breakdown in your application to the FDA I was just asking if that was part of it.
Or was it just a full requests for approval.
No matter what.
<unk> patients.
Thank you for your question, but legal counsel advised us not to go into those details today.
Can imagine was told before our type a meeting.
Well sure when when when.
I want to be an issue for us to share.
An additional question you might have.
Hello.
I guess, we lost him.
Okay operator.
Your next question for today is coming from Paul Smith.
Thank you.
Hi, My name is Paula Smith, My son, Josh is 33 years old and he was in the neuron trial and both Eap's.
First of all it's another I just wanted to say how grateful I am to brainstorm team as I believe that my son, Josh is still here today because.
Josh is diagnosed in March of 2019 and today he is still walking talking.
Eating breathing and still able to take family vacations.
Well he was in the trial and in the EAP.
Every time that he received from the neuro <unk> halted his progression significantly.
And that's just speculation.
Stipulations stopped.
When he wasn't receiving it declined.
We're now worried that.
E&P is over and there is a loss of function so ominous.
I have two questions today.
During EAP.
Pulmonologist documented a 41% improvement in his breathing function.
His breathing was at 64% and it went to 105%.
I know that the EAP sites weren't collecting this data because of COVID-19.
Several others in the EAP program have also improved and their breathing.
My question is how can we ensure that brainstorm includes this information in the submission to the FDA to show that neuro and it's working.
Listen as these questions from.
Tom patients' families members, calling us.
We're not prepared for this on an investor call, but we really appreciate it number one but as you can imagine we cannot comment about patient data.
I'm sure.
Yeah.
We collect everything professionally through our CFO and only that data, we're able to to summit.
I know <unk> patients.
Their own ways to two.
To.
Perhaps some maybe two doctor in the centers.
They can share it with our CMO, perhaps we don't.
We kind of have direct contact with patients as you know.
Again, this is very warming to hear from them to handle the other ones you know how this change we are regulus.
Regulatory wise to discuss any any patients.
I just wanted to thank you of course in Europe .
Sorry for being part of this and sort of warming to hear what you're saying, but I hope you understand that we cannot really comment to these comments.
Yes.
I just feel like it.
If it's the.
The data that they're looking for we have it we have a proven by.
The trials the EAP and then our outside doctors doctors.
Doctors can't believe the change in my son.
I mean, if you're looking for any information we have the information that's all I'm, saying.
We will pursue all leads that we can professionally offline and we thank you for putting us on this.
Thank you.
How happy I am here.
We're all probably here to hear all of the families benefiting you feel that is benefiting from the wrong yes.
Ah patients, that's very warming to head out, but we cannot comment on outside of that as you can understand.
Okay. Thank you.
We'll take one more.
Question.
Believe we're getting close.
Two.
And it's locked.
So operator.
Your next question for today is coming from Daniel Walker.
Thank you.
Yes, good morning, Daniel Walker with next industries.
Maybe just talk a little bit about this refusal to file letter I guess it seem like in some of your previous comments that you were working very closely with the FDA you spoke very highly of your work that youre doing with the FDA how much of a surprise what this refusal to file I mean.
Daniel you had indicated they were working with great urgency and partnering very closely with you can you just.
Opine on that a little bit.
Thank you.
As you know we showed that with the public when we announced the intention to file.
So we had long ago, we've taken the decision we have to file the BLA, that's the only way to get through to an outcome meeting.
We filed the BLA, we were hoping it will be accepted for review and.
It will be offered an outcome.
The definitely the letter that we got from the FDA. It wasn't what we expected, but the good news is that the type a meeting to allows an outcome requests.
From the FDA, so like it's a bump in the road and that's a big bump in the road, but I think we're still on the right track.
To proceed and I believe the FDA respect the due process here.
We respect their view to different views in our view.
We have huge support from.
Leading key opinion leaders.
And some of the patient community.
And we think as we said on the call you've heard.
The outcomes right venue to be able to have the debate like.
Like you see in other product and AOS.
We're only approved because they've got some got more than one outcome.
So I think we should get out a chance that one outcome and we want to win the first outcome. That's how the plan and we have to prepare for that so that's where we are I think.
The FDA has no reason not to allow an outcome.
Thanks for the great. Thank you so much and maybe just.
To kind of follow that up I guess.
Merit the Doctor at mass General.
That made there had been different understanding of the flexibility between.
What might be considered flexibility between cedar at Ciber can.
Can you comment on that.
Well listen.
Dr. <unk>. She speaks for her so I don't have to explain in our statements.
She is one of the leading doctors in the United States for AOS to chief on the knowledge and Harvard She understands a cvs either better than we do even.
What I could share with you is that our view is similar to many rare disease experts opinion.
And such rare diseases ALS.
Where there are very few treatment options if at all to offer.
The FDA shirt or even must be more concerned with the false negative rather than the concern of a false positive.
We hope to see that I think we have seen some flexibility in the previous approval for these reasons.
With our doctors earnings up and accepted.
And we hope we're very hopeful that we arent the same trucks.
That approval.
So thank you for the discussions.
Thank you so much.
Operator, I think that brings our call to a conclusion today.
Do you have any closing comments.
I just gave I think the closing comments, but thank you very much Holly and this was very well done.
Look forward for our upcoming calls with additional better news. Thank you very very much.
Thank you ladies and gentlemen, this does conclude todays event you may disconnect. Your phone lines at this time and have a wonderful day. Thank you for your participation.