This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk02: Greetings, and welcome to the Brainstorm Cell Therapeutics call to provide an update on Neuron. At this time, all participants are in a listen-only mode. 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.
spk07: Thank you. Good morning, and thank you, everyone, for joining us. Before handing it over to the company management today for prepared remarks, I'd like to remind listeners that this conference call 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 for the treatment of neurodegenerative diseases such as ALS, the sufficiency of the company's existing capital resources for continuing operations in 2023 and beyond, the safety and clinical effectiveness of the neurotechnology 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. Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond brainstorm 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 call. and the company undertakes no obligation to publicly update any forward-looking statements. Joining us on the call this morning will be Chaim Liebowitz, President and CEO of Brainstorm, Dr. Stacey Lindberg, Co-CEO and Brainstorm's Executive VP and Chief Development Officer, Dr. Bob Dagger is also on the call and will be available to answer your questions during the Q&A session. So, I'd now like to turn the call over to Mr. Liebowitz. Please go ahead.
spk05: Thank you, Michael. And thanks to all of you who have joined us today. Brainstorm issued a press release this morning, providing a regulatory update on Neuron. The purpose of this call today is to discuss the plans we have to work with the FDA and our strategy to seek approval for Neuron and to make the product available to people living with ALS. As you know, The FDA held an outcome to discuss Neuron on September 27th. Unfortunately, the committee voted that Neuron did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate ALS. While we were disappointed with the outcome and we disagree with the panel's conclusion, it is important for us to anticipate how it's likely to influence the FDA's decision to approve Neuron based on the evidence under review currently in our BLA and to make decisions that are likely to lead to an approval for Neuron for ALS with the most efficient path. We remain confident in the data supporting the value of Neuron for ALS patients and are committed to finding a path forward. We have made the decision to withdraw the BLA for Neuron from FDA review. This decision was coordinated with the FDA and is viewed by the agency as a withdrawal without prejudice. We understand at this point that additional evidence including a confirmatory clinical trial are necessary and we will do everything we can to execute on this as quickly as possible. The FDA has invited us to request a face-to-face meeting to discuss the path forward. We see this as a very positive development, and I'm pleased to say that the agency has been responsive to us and is engaged almost on a daily basis. We intend to use this meeting to discuss the next steps, including discussion of an additional clinical study. Since the outcome, our management and board have given considerable thought to our strategic priorities. And after exploring possible options, we have reached this conclusion that the only path forward for the development of Neuron for ALS is to conduct another trial. Therefore, we are withdrawing our BLA and appreciate the fact that this was coordinated effort with the FDA to enable it to be withdrawn without prejudice. At the same time, we're accepting FDA's invitation to have a face-to-face discussion on a face-to-face trial with the aim to reach an agreed-upon trial design. We understand and acknowledge that our role of the BLA and requirement for another trial will be frustrating or is frustrating and disappointing for so many. But we have determined that unfortunately there is no alternative at this time. to recognize the need to align with the FDA, and we'll do all we can to deliver a valuable treatment option to the ALS community. I'll now turn over the call to Dr. Stacia Lindberg for additional comments.
spk03: Thank you, Haim. Our goal is to agree with the FDA on the most direct path to approval for Neuron. And as Haim has outlined, in the interest of patients, the best action we can take is to conduct another phase three clinical study. We have the advantage of having generated a considerable body of data on Neuron in ALS participants. Our clinical studies, including the completed multi-dose phase three trial, have provided valuable insight into how Neuron is working and what patients are most likely to receive the greatest clinical benefit. In our completed phase three trial, We conducted a pre-specified subgroup analysis in participants with ALS-SPRS-R scores 35 and above at baseline. In this pre-specified subgroup, an enhanced treatment effect was observed with a 19 percent higher response rate with Neuron compared to the placebo on the primary endpoint. Importantly, in this subgroup, we show a clinically meaningful difference in the key secondary endpoint, the average change from baseline to week 28 on the ALS-SPRS-R in neuron-treated participants compared to placebo. The difference was statistically significant with a P of 0.05. As we consider the design for the second Phase III study, we will likely focus on mild or less advanced patient population. I'll now turn it back to Haim.
spk05: Thank you, Stacy. There's one more point I want to cover, as I know there will be questions on this, and that is how Brainstorm plans to fund the second Phase III trial. We're engaging directly with investors to communicate the value proposition. They might be institutions that are interested. Some already expressed some interest. They want to see our alignment with the FDA, of course. And also importantly, we are pursuing various funding options, including options for non-diluted grants. I will now turn this over to Michael for the Q&A session.
spk07: Thanks, Klein. The first question. Why would you withdraw the BLA when the ALS community has fought for years for you and is continuing to fight? And then as a follow-up to that, it's what are you saying to those in the ALS community who are lobbying the FDA for approval of the current BLA by the December PDUFA date?
spk05: Yeah. So, thank you very much, Michael, for filtering those questions. We got many questions. People either anticipating a call or just wanting to hear the answers, so we filtered the main important questions. Thank you for that, Michael. We're grateful to the ALS community for their support, of course, and for people living today with ALS. This is a very difficult decision for us, one that is informed ultimately with our end goal to make Neuron commercially available to ALS patients. We unfortunately see no alternative path at this time. We continue to stand behind the data and believe in Neuron as a viable therapy for ALS. We move forward and filed over the test, requested an actum because of the urgency and unmet need in ALS. The outcome of the outcome and the subsequent discussions with FDA has made it clear that an additional trial is the only path forward and that it's most likely to result in an FDA approval. Thank you.
spk07: Next question. What does it mean that you withdrew the BLA without prejudice?
spk03: Michael, I'll take that. It means that rather than waiting for the badoo-pa-date for a final FDA decision on Neuron, we're acting on the feedback from FDA and the adcom to voluntarily remove the BLA from active review. And our goal is to increase the speed to which Neuron can be made commercially available by generating new data and addressing items identified before proceeding with a BLA for FDA review. As Haim shared, our decision to withdraw the application is viewed by the FDA as being without prejudice to refiling. In other words, it allows the application to be resubmitted at a later date after we've addressed deficiencies and brought the new data to the submission.
spk07: Thanks. Next question. Will you be providing access through the Expanded Access Program while completing the next Phase 3B trial?
spk03: Yeah. We understand the importance of access to investigational products through an EAP and will support this if possible financially. For now, our top priority will be funding and executing the Phase 3B trial. we will pursue funding for an EAP after the Phase IIIb trial.
spk08: And a final question.
spk07: Why have you not unblinded the completed Phase III trial at this point? And why does it take you so long to unblind the study?
spk03: So, the withdrawal of the neuron BLA enables brainstorm to provide the treatment assignments to those who participated in the placebo control portion of our Phase 3 clinical program. Information regarding randomization will be provided to the principal investigators at each of the clinical sites who will provide information directly to participants and their families. And HIPAA and confidentiality laws prohibit making private medical information public. We do want to comment we are thankful for all of the clinical trial participants and their loved ones for their sacrifice to be part of the trial and to move forward ALS science. And we deeply hope that this information will offer some peace of mind or at least closure to those living with ALS who contributed to the trial in immeasurable ways.
spk05: Thank you, Michael. Holly, you want to open the call for some questions?
spk02: Certainly. At this time, we will be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. One moment, please, while we poll for questions. Your first question is coming from David Bouts at Zacks Research.
spk01: Hey, good morning, everyone. Thanks for the update. I don't know if I missed this or not, but how quickly is this meeting with the FDA going to take place? How soon will you be able to do that?
spk05: A very good question. Thank you, David. The truth is they are offering us to ask for an expedited meeting, but we do have to prepare a draft of a trial design to submit. So we will, of course, use the wonderful committee we already have set up months ago, as we even anticipated an approval. We knew we would have to do a phase four confirmatory trial. So we're going to have a meeting again, and as soon as we can have that draft forward, we'll send it. We even heard from FDA more or less what they're looking to see in such a trial. So I think it will be easy. but it's hard for me to say a date today.
spk01: Okay. I don't know how much detail you can get into with what you hope to do with the design that you hope to get for a phase three study, but maybe you could just outline a range of patients and then maybe what you're looking at for the primary outcome in this study.
spk05: Yeah, David, that's premature to say today because As you can hear in our message, we want to align with the FDA how this is going to be done. They want to align with us, which is a very good thing, maybe even a spa. So that's why I want to be careful before throwing out the design. Okay.
spk01: No, I understand. So maybe you can comment on, will this study only be taking place in the U.S., or will you be looking to maybe do something outside the U.S.? And then... do you think most of the PIs that you had in the phase three study, in the previous phase three study, will work with you in this next study?
spk05: Thank you for these questions. So I can say that many of the PIs are urging us to do this additional trial, and I believe that all PIs would be more than happy to work with us. Coming forward, we have a wonderful relationship with all the centers. We may choose some centers over others for geographical reasons. We were getting arguments that Center America and other places don't have a site. So we'll see where we end up. But yeah, the leading PIs are totally behind us and they're pushing us to go forward. They feel that, as you heard on the outcome from some of the PIs, but also others, of course, they strongly feel that this is working for a subset of patients and they don't want to see this not going forward.
spk01: Okay. So you plan on this study taking place in the U.S.? ?
spk05: Yeah, so for that, of course, we will focus on the U.S., but the European sites are interested to be part of another trial as well. So we will discuss with them once we have a design. We will first have to align on the design with our PIs, of course, and also our PIs will also align with other ALS organizations in the States, just like the New Scientific Committee. We'll work with them very closely. They are already speaking to us. and with the FDA, and we may have a conversation with NCALS. We already spoke to them quite a few times, and you may have seen before the outcome, they want to see another trial for Neuron, like many other doctors wanted.
spk01: Okay, and then lastly, as far as funding the trial, do groups like IMALS and other patient advocacy groups, do they have funds available to support trials like the one you're going to be doing?
spk05: Well, I can't speak to specific organizations, but there are organizations that have funding. It will be up to them if they want to fund. We will probably reapply to CIRM, who was a big funder of our Phase 3 trial, close to $17 million. And I think they would be interested to get our application. So we'll see who else we will be asking for funding.
spk01: Okay. Sounds good. Thanks for taking the questions.
spk02: As a reminder, if there are any questions or comments, please press star 1.
spk08: As a reminder, if there are any questions or comments, please press star 1.
spk02: Your next question is coming from Daniel Walker. Daniel, your line is live.
spk04: Hi, I'm in Stacy and Bob. I guess, Haim, any updates on the team? I guess, what's the kind of general feeling of the team? And, you know, do you anticipate most of the teams staying on board for this next sort of phase as you move forward? Or do you anticipate some changes with any key personnel?
spk05: Well, we'll definitely have to reorganize going forward based on funding. And, of course, we have to focus on those people that will most strongly be able to support the trial, the upcoming trial. But, yeah, the whole team is with us unanimously to go forward for another trial. Strong support from everyone. And we're all saying that we're willing to go through whatever is needed to try going forward. We have full support from everyone on the team, you know.
spk04: That's great. And then I guess just short-term, near-term, as far as funding, obviously you're going to look to these organizations to get some grants or funding to move forward with this additional phase three. But I guess near-term, how are you thinking about that as a company? You obviously have to meet with these organizations before getting commitments. How are you thinking about that in terms of the existing resources?
spk05: Yeah, as you may know, there are many institutions that are quite interested to support trials that are aligned with FDA similar to that. So, I think we are very focused to approach those institutions. I think also general investors seeing an alignment with FDA will understand that the risk is lower going forward. On the other hand, there are already some investors that came forward that they will want to assist us in the interim until we have that design and we go forward. Just have to see how the market reacts to our announcement today and to the additional announcements we will have over the course of the next few weeks.
spk04: And just lastly, I guess in hindsight, Many investors believe that there was alignment, or I guess you had remarked as such at various times that there was some alignment with the FDA. Obviously, the outcome was what it was. In terms of looking back at this, obviously it's early, it's only been three weeks, but what would you say is the one sort of key takeaway from all this? I know this has been, you know, quite a long time, you know, that this has been something you've been working on for some time and ultimately decided to do this and move forward in this way. Any learnings from this now looking back? I know, again, it's only been three weeks.
spk05: No, it's not a question of three weeks, you know. There's an easy way to say, well, you know, Can't trust the process and just give up. That's the easy way. We strongly believe in our product. And the FDA reached out to us. They want us to do another trial. And we are going to take this very seriously. And we're going to give it all we can. We owe to ALS patients. Unfortunately, we can't deliver today. We tried. We did everything we can to deliver today. Ultimately, many thought that another trial would be needed. And we're up for that. It's easy to say no. I hear that in your voice. You asked me how do we have the strength, but we have a wonderful team, PI supporting us, and FDA asking for another trial. That's great. I'm not in a position to go into other inside stories within FDA. I don't think it's the right thing for the company to do.
spk04: That's great, Aram. I appreciate all the answers. Thanks so much. Thank you very much. Ali?
spk05: One more question, please.
spk02: Your final question is coming from Richard Robbins.
spk06: Good morning. I have a two-part question. First part is, Mr. Chairman, you have been the chief architect and the manager of the FDA debacle where 17 experts voted against you. one of the participants was even laughing at the data, or at least chuckling at the data. My question to you, sir, is that given that you were the manager of this, how come you have not resigned, or why has the board not fired you?
spk05: Thank you very much for that question. So I cannot speak on behalf of the board. You can ask them questions directly to them, but no one on the board is asking anything like that. As I said, We brought forward a trial that, in the first place, the statistical results were not as we were hoping to be. It wasn't black and white. The agency's position was that it's not enough. We went out of our way after listening to so many scientists in the field, both those that were involved in the trial and those outside, all felt that the flexibility for ALS, as called in the guidelines of FDA, should be applied here. I think we did a wonderful, outstanding job presenting the outcome. I don't know if you listened to that. I did. Okay. And I think whoever listened to that, here's what our case was. Now, whoever listens to the second part will get their own conclusions. I'm not here to blame and complain about the agency. We want to work with the agency for the next trial. My question is about me. You think that you don't have any trust in me. If you're an investor, I get that. I'm very happy that the major shareholders of the company are asking me to stay on. And I'm going to do that as long as needed.
spk08: How come you have not resigned? As I told you, the major shareholders want me to stay on.
spk06: Conference call after conference call, I keep hearing that you have confidence in the data, yet 17 experts from the FDA, and by the way, the FDA holds all the cards, you hold none, did not agree with your data, and in fact, one of the participants was even chuckling at the data. Why do you keep saying you have confidence in the data when the FDA doesn't?
spk05: Well, I think I gave the answer, and just to go around in circles, it makes no sense. Stacey wants to say something, and I'll allow her to say something. Thank you.
spk03: Yeah, I think some of the backing and the importance for us as we evaluate the data is really relying on the experts that we put around us, so world-renowned neurologists, statisticians who are looking at our data, looking very objectively, are independent from us, and are giving us counsel and talking very openly and publicly about our data. That is a very important part of our own evaluation as we take every step.
spk06: Just as a final comment, Stacey, I think you should become chairman and CEO, and I think Chaim should find something else to do. I say this respectfully. Have a good day.
spk08: Thank you.
spk02: We have reached the end of the question and answer session, and I will now turn the call back to Chaim for closing remarks.
spk05: Thank you very much, everyone, for listening today and for your wonderful questions, and have a wonderful day. Thank you very much.
spk02: This concludes today's conference, and you may disconnect your lines at this time. Thank you for your participation.
Disclaimer