Brainstorm Cell Therapeutics Inc (BCLI) 2022 Q4 法說會逐字稿

Greetings, and welcome to the BrainStorm Cell Therapeutics' fourth-quarter 2022 earnings call. (Operator Instructions) As a reminder, this call is being recorded. And I would now like to introduce your host for today's call, Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.

Good morning, and thank you for joining us. Earlier today, BrainStorm issued a press release with its financial results for the full year 2022, including a corporate update. Before passing off to the company management for prepared remarks, I'd like to remind listeners that this conference call and webcast will contain numerous statements, descriptions, forecasts, and projections regarding BrainStorm Cell Therapeutics and its potential future business operations, and performance, statements regarding the market potential of the treatment of neurodegeneration such as ALS, the sufficiency of the company's existing capital resources for continuing operations into 2023 and beyond, the safety and clinical effectiveness of the NurOwn technology platform, clinical trials in NurOwn and related clinical development programs, and the company's ability to develop strategic collaborations and partnerships to support its business planning efforts.

Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond BrainStorm's control, including the risks and uncertainties described from time to time in the company's SEC filings. The company's results may differ materially from those projected-on today's conference call, and the company undertakes no obligation to publicly update any forward-looking statements.

Joining us on the call today will be Mr. Chaim Lebovits, President and CEO of BrainStorm; Dr. Stacy Lindborg, Co-Chief Executive Officer; and Alla Patlis, Interim Chief Financial Officer. In addition, David Setboun, Executive VP and Chief Operating Officer is also on the call and will be available to answer your questions during the Q&A session.

I'd now like to turn the call over to Mr. Lebovits. Please go ahead.

Good morning. Thank you, Michael. I want to thank all of you that joined us to discuss our 2022 financial results and recent developments.

Throughout 2022, BrainStorm had a number of important clinical and operational achievements, which we believe position the company for further success in the year ahead. Our priority in 2023 is to advance NurOwn through the regulatory processes as expeditiously as possible. On this round, we were very excited to announce earlier this week that the FDA intends to hold an Advisory Committee Meeting to discuss our BLA seeking NurOwn's approval as a treatment for ALS.

We held a conference call on Monday to discuss the planned ADCOM. But given the importance of this news, as well as some of the follow-up questions we have received since then, I think it's worthwhile to summarize the cost content and emphasize a few key points.

First, the information we communicated on Monday is that NurOwn's BLA is back under review, and the FDA has committed to an Advisory Committee Meeting. The date of ADCOM is unknown at this time, but we know the FDA is actively working on it. We will share it with you once we receive it from the FDA.

I want to emphasize a few additional points from our investor call from Monday. Point number one relates to the review process for NurOwn's filed BLA. Now that the BLA is under review, the review process should be the same as any other regulatory mechanism we could have utilized to get to this point. We look forward to working with them as we work throughout additional stats in the review process, including the regulatory response.

There will be an ADCOM guided agenda, and a set of questions put forward by the FDA. These questions or question will allow experts at FDA and BrainStorm to frame the efficacy and safety of NurOwn in ALS.

Independent medical, and statistical experts, members of the ALS community, and other key stakeholders will then have the opportunity to participate in an open discussion on the prospectus shared by the FDA and BrainStorm as well as the need for new ALS therapies. The agency will then render a decision on the BLA by specified PDUFA dates. We anticipate that we will have the date of ADCOM soon. And when we receive that, it will also have a clarity on the PDUFA date.

The next point I'll emphasize relates to the regulatory mechanism we utilized, which is formerly known as the File Over Protest pathway. Despite its name, this mechanism is a standard regulatory procedure and is not inherently agonistic. In fact, we collaborated constructively with the FDA before the agency provided us with several regulatory options that would reactivate the BLA in order to allow NurOwn to be discussed at an ADCOM.

As mentioned on our call on Monday, this pathway was ultimately chosen over the other options because this allows the fastest regulatory path in ADCOM. This was the driving factor in our decision making. We recognize the urgency that patients deserve.

Next, I'd like to remind those listening to the content of the Refusal to File Letter we received from FDA late last year and speak about how we plan to address these points. The RTF letter included one item related to the trial not meeting the standard for substantial evidence of electiveness, or the primary endpoint [meeting] statistical in the business, and the remaining items related to chemistry manufacturing and controls in short CMC.

Given our exemplary record of high-quality manufacturing, we have full confidence that we'll be able to remediate each of these points in a straightforward manner. In fact, we already have conducted much of the necessary work in a short timeframe and submitted an amendment to the BLA on March 7, 2023, which responds to the majority of the manufacturing items raised in the letter.

Work to respond to the few remaining manufacturing items is ongoing and would be complete in due time. The remaining items of RTF letter and the only item related to our clinical data, that will be the focus, that will be the key point of the discussion of our upcoming ADCOM.

We view the opportunity to discuss NurOwn's full data set in the public forum offered by ADCOM as an extremely positive development for BrainStorm and the entire ALS community. As this forum offers an open conversation among agency reviewers, BrainStorm's experts, clinical program investigators, and all other relevant stakeholders can take place. Each of these groups has its own expertise, experience, and points of view to contribute. And it's critical that all voices are heard given the complex scientific and policy issues at hand.

Finally, I want to once again thank the FDA for their collaboration throughout this process that led them to this decision. Given our data set and incredibly urgent need for novel therapies that can improve the lives of individuals living with ALS, we believe an Advisory Committee Meeting is the most prudent and appropriate next step in NurOwn's regulatory path.

With this, I'll now turn the call over to my colleague, Dr. Stacy Lindborg.

Thank you, Chaim. Once again, I want to make the point that securing an ADCOM has been central to our strategy because we have a robust and compelling data set that will benefit from a deep and thoughtful discussion at this public meeting.

While our Phase 3 trial of NurOwn NALs did not reach statistical significance on the primary endpoint, we firmly believe that the totality of evidence from the trial will ultimately support approval. This belief has only grown as we've continued to analyze the trial results and discuss our learnings with leading experts in the ALS community.

Along these lines, I'd like to highlight a presentation that was made at the Annual Muscular Dystrophy Association Clinical and Scientific Meeting, which was held in Dallas last week. As we've explained before, we now understand that the results from our Phase 3 trial were influenced by participants who entered the trial with advanced ALS and who fell victim to the floor effect of the ALS Functional Rating Scale. A floor effect is observed when scale items reach zero, and ongoing progression cannot be measured on the queried item. The rate of the ALSFRS-R decline appears to slow and plateau in many patients despite further deterioration of the participant's function and health.

While the floor effect is certainly a limitation of the ALS Functional Rating Scale, the good news is its presence can be objectively measured, demonstrated, and accounted for. This is what we've done with various techniques, including with the new analyses presented at MBA last week. This analysis focused on trial participants who were not impacted by the floor effect at baseline, or in other words, participants who did not have a zero on any item of the ALS Functional Rating Scale at baseline, which means that the scale was able to measure a decline that occurred in participants across the entire scale.

The number of participants with no evidence of the floor effect at baseline in this trial was 106 out of 189 or just over half the participants in the trial. When looking at these participants, there was a significantly higher response rate with NurOwn compared to placebo on the primary endpoint, with 41% of NurOwn participants reaching this substantial definition of clinical response versus 23% of placebo or a difference of 18%. And this had a significant p-value of 0.035.

Additionally, in these participants with no evidence of floor effect, we see significantly less disease progression on the endpoint average change from baseline in the ALSFRS-R to week 28. There was a difference of 2.31 with a p-value of 0.04. A copy of this presentation is on the Events and Presentations section of our website, and I would encourage those interested to read it.

These important findings not only give us more confidence in our clinical data, but also address an area where there appears to be a misconception among some observers. There's a perception that the Phase 3 trial had a higher-than-expected placebo effect with approximately 20% of placebo participants responding on the primary endpoint at week 28. While it's true that we expected only 15% of placebo participants to be classified as responders on the primary endpoint, which is based on numerous post-hoc sensitivity analyses, we can objectively say that this was a floor effect that impacted the primary endpoint and not a placebo effect.

While the ALSFRS-R floor effect impacts all analyses that leverage the ALSFRS-R scale data, in the primary endpoint, when the floor effect occurs, it results in a misclassification of clinical response. The plateau also occurs due to the inability to measure further decline on impacted items and also results in a difference from the pretreatment rate of decline and participants meeting the definition of response. Thus, this is a scale phenomenon and not a halting of clinical symptoms.

In fact, in the same trial participants who had the highest rate of floor effect in the study, driving their ALSFRS-R scores to meet the criteria on the primary endpoint of clinical response, these same participants had the lowest SBC scores, the highest rates of pretreatment decline, and the lowest baseline ALSFRS-R scores. And while I'm focusing on these analyses that controls the floor effect, which include these new analyses presented last week, let's not forget that in a prespecified group of participants with the ALSFRS-R score above 35, it was a larger treatment effect across all endpoints with NurOwn compared to placebo with a statistically significant difference on a key endpoint to the average change from baseline in the ALSFRS-R.

Lastly, in addition to these prespecified and sensitivity analyses, biomarker data collected during the trial also demonstrate the importance of accounting for the ALSFRS-R floor effects when evaluating clinical endpoints. These data showed NurOwn positively affected multiple pathways and are intimately involved in ALS, including those that are related to neurodegeneration, neuroinflammation, and neuroprotection.

Importantly, the changes observed were consistent regardless of participants levels of disease progression at baseline, with positive effects seen in trial participants with less advanced ALS and with those with more advanced ALS disease at baseline. One of the reasons we've been able to drive such valuable learnings from our trial's biomarker data is because we employed an extensive specimen collection protocol in the study. This protocol, which called for the matched collection of up to seven serum and CSF samples from trial participants over a 20-week period, is to the best of our knowledge, the largest longitudinal specimen collection protocol ever employed in an ALS trial.

To maximize the value of these samples for the ALS community, we partnered earlier this year with NEALS to provide the public with access to samples from placebo-treated trial participants. This was done in connection with a $500,000 grant previously awarded to BrainStorm by the ALS Association and I AM ALS to support biomarker research in the Phase 3 trial. By providing the ALS community with access to these clinical samples, we hope to drive additional research that will facilitate the discovery of new breakthroughs for patients with ALS.

I'll now turn the call over to Alla to discuss our financials.

Thank you, Stacy. It is my pleasure now to walk you through our full year 2022 financial results.

BrainStorm's cash, cash equivalents and short-term bank deposits were approximately $3 million as of December 31, 2022. This compares with approximately $22 million on December 31, 2021. Our research and development expenditures net in the year ended December 31, 2022, were $14 million compared to $15.2 million for the year ended December 31, 2021.

General and administrative expenses for the year 2022 and 2021, respectively, were $10.9 million and $9.3 million. Net loss for the year ended December 31, 2022, were $24.3 million or $0.66 per share as compared to a net loss of $24.5 million or $0.68 per share for the year ended December 31, 2021.

Now I'll turn it back to Chaim to close the call.

Also for Q&A, but thank you very much, Alla. And Michael would you please -- can you read the prespecified questions we have before we will open the call for callers' Q&A. Michael?

First question. Can you tell us about the biomarker response in the trial versus what happened in the expanded access program?

Very good question. Stacy, that's for you.

Sure. Let me start by summarizing what we've collected in the expanded access program. So we collected both clinical data and CSF samples and collect them on a schedule that was similar to the Phase 3 randomized clinical trial. As a matter of update, the second period of the EP has been completed, which now completes the EAP, and we've begun to analyze the clinical data.

The final biomarker samples need to be collected from the sites, and then samples from all of the EAP participants will be set to labs, the same labs that were used in the Phase 3 trial for analysis. And we will present the data in a scientific forum like we do with all new data.

Thanks. As a follow-up question, have you submitted your biomarker data to a public academic journal yet?

Yes, Stacy?

The manuscript is fully written, and it's in its final stages of review with authors prior to submission. We're really looking forward to getting it under review and generating what we believe will be an important publication for the ALS community in the near future.

Question is regarding the timeline on the PDUFA date and the advisory committee. Did BrainStorm ask for or receive six-month priority review?

We're waiting for the FDA to provide us this information. And that said, we will communicate when we have this information.

And then regardless if you're on a 6- or a 10-month review timeline, can you clarify when the clock started running? Did this happen at the time of the refusal file date? Or is it running a new as of last [one] noted regarding the -- from the FDA?

Good question. So the BLA is back under review. It had to be back under review for the FDA to have an ADCOM. The timeline with the date of RTF is as follows: February 6, 2023, BrainStorm notified the FDA of the decision to request the FDA to file NurOwn BLA for ALS.

And the following day, on February 7, we received confirmation from FDA that the BLA has been refiled. Therefore, February 7 is the date that the review timeline became active again. While we made this point during our prepared remarks, but it's worth reiterating. We chose the procedure of File Over Protest because it allows for the quickest path to ADCOM and started the clock again.

The only of the regulatory path we were seriously considered was to revise the BLA and resubmit. And we did not select this pathway that could present substantial delays in the overall regulatory path, as exemplified by the journeys of other sponsors who experienced unexpected delays.

Individuals living with ALS deserve the urgency that comes with the regulatory clock associated with a BLA filing and an ADCOM. So again, on February 7, it was reactivated file, meaning the first two months already happened from the first day when we (technical difficulty) submitted September 9. And then it paused after the RTF letter, and then the clock started to tick again on February 7.

And one last question, this is a financial question. Has the company been active with its ATM in the last few days? And then what is the capacity of that ATM?

So no, we're not active, of course, in these prices. We have confidence that we'll be able to activate the ATM for better return and less dilution.

As we have disclosed, of course, we do have an ATM in place. It's sponsored by Raymond James and (inaudible). But we did to a single cross investment made by single institutional investor, but we are not activating, and we don't intend to activate the ATM in these current prices.

And the capacity of the ATM?

$100 million.

Okay. That's the final question.

Holly, would you open the call for any questions from any callers?

(Operator Instructions) Jason McCarthy, Maxim Group.

Just a clinical-focused questions. First, did the floor effect impact the biomarker changes in the total Phase 3 trial population? And if not, can the biomarkers be used to show that while the floor effect impacted the ALSFRS-R score in the total population? Can you still see the impact of NurOwn just through the biomarkers, in particular the NFL biomarker, which came up as a big deal during the Biogen ADCOM as a therapeutic marker?

The short answer is yes, but I would let Stacy to elaborate.

Yeah, Jason, it's a great question, and it is a really important point. Every time we've talked about our biomarker data, every presentation we've given in the public domain is focusing on all trial participant because everybody that was in the trial, we see the same biological response with NurOwn across all patients in the trial.

And importantly, we also see no changes as we would expect in these same biomarkers with participants treated with placebo. So there is a very important finding that is brought to this overall study that comes from the biomarker data. And it allows us to have even more confidence that the impact that we're seeing in ALS functional rating scale is, in fact, due to a limitation of the scale and does not reflect on something further about these participants in the trial.

We simply can't measure the decline in participants that progress to advance of the stage of disease and to where the scale has zeros and can't measure differences. So that's the very important point. And we did very specifically look at the participants' data, the biomarker data from the thresholds that define participants that have a floor of the scale versus those that don't, and we see the same patterns across all of our biomarkers. So that will become very clear in our manuscript that will describe our biomarker data very fully.

The second question about -- I'm sorry?

No, I'm sorry, go ahead. I didn't mean to interrupt.

You brought up neurofilament light. And I think there was a very broad and wonderful rich discussion at the two-person ADCOM. And a lot of competence displayed about the ability to the importance of this marker in ALS disease, specifically it refers to the mechanism of action with their products.

What I would say is that when we look at our biomarker data, and we want to understand the relevance of these very large changes that we see across markers of neurodegeneration and neuroinflammation in addition to markers and neuroprotection, we wanted to understand and appreciate how important they were to the clinical response observed. This is something, as a company, we certainly thought about well in advance; we knew it would be extremely important. We don't want to just change biomarkers. We actually want to know that there's a direct effect on the clinical outcomes.

And so this was the subject of a second analysis plan that was submitted to the FDA before we unblinded our trial, where we outlined the proper ways to actually explore these questions. And these models that were prespecified, identified three markers that the knowledge -- the changes that were observed in the trial and where their participants were starting at baseline, that they were very important to predicting the clinical response that was observed in the trial.

And one of those markers actually was neurofilament light. So the changes and the knowledge of baseline values are important to understanding the clinical response, a very relevant to clinical response in addition to a marker of neuroinflammation and neuroprotection. So it's very important.

We know that these three pathways are very central to our mechanism of action. We've also uncovered data that suggests that these are very critical. The ability to modulate, these pathways are very relevant in the clinical outcomes that we observed in this trial.

Can you address also the floor effects drove in part the 28% or so placebo group response rate in the total population in the Phase 3? You'd mentioned in the 106 patients data that was presented a couple of weeks ago at the Muscular Dystrophy Conference, where the -- you removed the floor effect and the placebo response was 23%, while NurOwn was, I think, you said 40% or higher.

The 23% was still above that 15% statistical benchmark that you designed the trial around. Is that a function of something else? Or is that just the small end value in a subgroup that created some noise, and it looks a little bit higher than that 15%?

Yeah. I mean -- so I would say that that is within the ballpark of what we expected based on historical data. So when we design trials, we look at historical data, and we really try to estimate based on other studies, in other settings, natural history, and also clinical trials.

And given the heterogeneity of the disease or the progression rate, all of that can basically interpret and lead to find these in trials. And you really try to estimate what would you expect in a group of participants randomized to placebo.

So the primary endpoint and the assumption that we'd see about 15% response rate on the placebo arm, a 35% response rate on the NurOwn treated participant arm. Those were our assumptions going into it. And in this sample of about half of the data, the NurOwn responders were also about 5% higher than what we anticipated going in. So it was 41% and placebo around 20%.

So I mean, these are -- we were expecting it's about a 20% difference in response rate, and we see that in a sample. And I would say that when you take small samples, you expect some variability (multiple speakers)

The last question. Briefly, can you just talk little bit about the safety aspects of an autologous cell therapy being incredibly safe. I think it goes relatively unnoticed with everyone's focused on efficacy versus other types of therapies. And how something that's very safe like this could really impact to the good, an ADCOM decision?

Yeah, that's also great question. And with every single study, we take very seriously the safety of the trial participants, and we monitor safety very closely. We had a data safety monitoring board monitoring the patient safety from [that live] across the study.

And we also go to great lengths to summarize what was observed in the trial. So we can certainly talk hypothetically as you're asking about the anticipated safety in a -- with an autologous cell therapy. But what's important is to also come back to the evidence and in what we generated.

We've summarized that very completely, very thoroughly. And certainly, that will be summarized at our ADCOM. This is a dimension that the physicians [closest] to our trial. Including our data safety monitoring board have continued to express that there were no safety concerns that were existed in the trial or that emerged as a -- from the profile of NurOwn, and that safety has been satisfied to their complete satisfaction.

Great.

David Bautz, Zacks Small-Cap Research.

So Chaim, you're going to have ADCOM date soon or PDUFA dates. And so when does the company begin discussions, say, with third-party payors and maybe discussions about patient access? I don't want to get too far ahead of things, but at what point of the company begin to have those discussions?

We're able to start in a bit, and I would like to bring in our Chief Operating Officer. He is working on this over a year now. We are preparing for success, as you can imagine. David, do you want to answer this question?

Sure. We've obviously been working on it for quite some time. We have as well the market access and pricing capability and expertise in-house. So we've been engaged with payors for quite some time now.

Okay. And I guess a follow up, without holding you to any hard and fast numbers here, but how quickly would NurOwn be available say, if it was approved? And then where does your manufacturing capabilities stand at this point? For how many patients you could treat, say, in the first year or so?

Of course, we're not going to go into hard numbers, but I will generally tell you that we have an outstanding partner with Catalent. And the Princeton site is very dedicated to us. And we have additional sites here in Israel as well. So we will be able to treat patients immediately after approval.

And of course, we'll have to enroll those patients, et cetera. But for the manufacturing capabilities, we technically are able to treat now patients if we were to have an approval.

Okay.

Mira Klingenberg, Private Investor.

As you know, my son, Matt was in Phase 3 and received six doses and expanded access.

I'm sorry, what was your name? Sorry, I don't mean to interrupt. Yes?

I'm racing back. (multiple speakers)

Sorry, yeah. (inaudible)

My name is Mira Klingenberg.

Okay, thank you.

So I was wondering if you were -- my son, Matt, was in Phase 3 and in expanded access. We are very pleased and thank you so much.

So Matt's symptoms started over five years ago, and he's still walking, keep talking, eating, greeting, all of that. He has not been [trached]. So I was wondering, is there any information about people in expanded access during the trial, and whether or not they're trached, and how long they live? And are you planning on releasing any more information about this study like you have through manuscripts and presentations prior to the ADCOM?

We did speak about it before, but I will let Stacy follow up and give you more specifics.

We're really pleased to hear your son is still doing so well. Five years into the disease, it's quite remarkable. From the expanded access program -- well, first, in terms of traches, we did not have any traches during the Phase 2 trial.

We've just brought the data in-house, and I actually haven't seen yet the rate of any traches that were given during the expanded access programs. I won't comment there. But this data becomes important to ongoing learning.

And it will be something that we will want to present in not only scientific forums but also understand the body of evidence and how it relates to the approval of our product. So we're working internally to analyze the data, and then we'll proceed with discussions with regulators as well as sharing data in the public domain.

Yes. Just to add, and again, I also want to first comment the wonderful news you're sharing about your son. We're very happy to hear that.

But in manuscript wise, our next manuscript would be a biomarker manuscript. And as Stacy just said, even though you're son got probably treatment quite a few months ago, but we got the final data set just a few weeks ago, and that's what we have to analyze.

And I don't know how fast this is able to be published in the manuscript because it depends on, as you know, this journals. But of course, we'll try to have it out as soon as possible. And in our comments, we said in the opening, Stacy said that we will definitely find the platform to share the EAP data and hopefully even before ADCOM.

Candy Simon, Private Investor.

I'm calling in on behalf of -- my son was also in the Phase 3 trial. He was 21 at the time of the Phase 3 trial and probably one of your youngest trial participants. He was diagnosed on December 10, 2018. And we couldn't find a family who was last prepared for this diagnosis, much less who knew anything about any trial regarding ALS.

We did not know that NurOwn was the treatment that everyone was speaking within the ALS community. We knew nothing about the Phase 1 conducted in Israel. We knew nothing about the Phase 2 conducted here in the US.

What we do know is that our 21-year-old son is positive that NurOwn worked for him. We witnessed the benefits with our own eyes as well as what he told us. However, we have basically been called liars by people who can't seem to grasp that NurOwn does work. And despite having email BrainStorm twice requesting to be unblinded, we have never wavered in our advocacy for the approval of this treatment; from day 1, we have believed in it.

Cabe was not offered EAP in round one or two. So sadly, not being unblinded, we do not know with 100% certainty if he received it in the trial. I do plan on testifying at the ADCOM, but it's been 3.5 years since Cabe received his third and final trial injection with no ability to access more treatment in something that works for him.

He's 25 years old now. He is still breathing on his own despite being a life-long asthmatic. He's still eating anything he chooses to eat. Obviously, he has declined sense being in the trial, not being offered EAP 1 or 2, but it would be lovely to be able to speak with confidence.

And I'm curious if you are planning to unwind any of the trial participants, so that the data that I present at the ADCOM is more believable. It feels like BrainStorm, and I'm not trying to be rude, has thrown us to the world to speak highly about the treatment that works. But then we're called liars because we are not unblinded, and people were assuming it was the placebo effect.

So my question is, is there any intention to unblind?

Just a clarification. First of all, we're very, very happy to hear about your son's stability in this disease. This is a wonderful news.

But we didn't have 2 EAP programs. It was the same EAP program that went twice for the same patients that may have been [concluded] -- unfortunately, we couldn't include more patients. We would love to if we would have the possibility. I'm so sorry, we couldn't include more patients.

Really, unblinding policy, we know that some of you have not had. And of course, we are very, very strong in our policy that it is not the best interests for the trial in regulatory process to unblind. And I will love Stacy to really give you a detailed explanation on that, which we already, I think, passed through your doctors.

But we're very happy to this opportunity to tell you directly. But you should know we have the best interests of ALS patients, and we want to do what's best to get this product in access to patients yesterday.

We understand the urgency. And I heard you -- a lot of you saying that you want to be to talking stronger at an ADCOM, I get that. But we have this conflict of interest, like what's the best interest of getting this product approved. And that's what we have to focus.

But Stacy, please give those professional insights you have from your 25 years in the industry, what all trial do usually. There are, of course, some that choose a little bit different way, but they know what the norm is. Stacy?

Yeah, Candy, we thank you very much for sharing the background of your son. And we are encouraged every time when we hear update in terms of the stability of patients. So I'm thrilled he's breathing on his own and able to eat.

But let me step back and provide insights into the conversations that we've had and really what drives our position right now. So from the time -- you're speaking to actually be able to describe with confidence the results that you've seen, and with your son the stability that he has experienced which is unusual in a disease like this.

For others, it's information that will offer a peace of mind or closure on the trial, and we have to say that our first and strongest inclination is to honor these requests. The dilemma that we must confront comes with a desire to provide this level of data has the potential to be detrimental to the overall community, in the sense that it could interfere with the regulatory review.

So we believe this would be the case if we provided patient level randomization while the regulatory review is underway. And for this reason, we made the difficult decision to maintain confidentiality of randomization.

We have evaluated the merits of this decision at multiple times since the trial completed. And we continue to believe that maintaining confidentiality of randomization remains the best decision until the regulatory review process is complete, as it facilitates a discussion of the evidence at a treatment group level.

And we know this is an industry best practice. And I know perhaps it's confusing from the questions you're asking. But fundamentally, when we look back to the forefront and even the chairperson in ADCOM from very recently, there were important scientific benefits from preserving the confidentiality of treatment assignments as they did as well in the interpretation of future data, as it reduces bias in the open-label period of their study. These same parallels exist for us with the expanded access program.

So I hope this gives you some insight. We are absolutely motivated by what is best and would be in the best interest of the entire set of people living with ALS who could benefit from an approval, and that is at the forefront of our action.

Okay.

We have reached the end of our question-and-answer session. And I will now turn the call over to management for any closing remarks.

Just thank you very much. Thanks, Holly. You very well done this call. And thanks, everyone, for all the questions and have a nice day.

This concludes today's conference, and you may disconnect your lines at this time. Thank you for your participation.