Brainstorm Cell Therapeutics - Q2 2024

Transcript

Operator (participant)

Greetings, and welcome to the BrainStorm Cell Therapeutics second quarter 2024 conference call. At this time, participants are in a listen-only mode. As a reminder, this call is being recorded. I would now like to introduce your host for today's call, Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.

Michael Wood (Head of Investor Relations)

Good morning, everyone, and thank you for joining us today. Before passing it off to company management for prepared remarks, I would like to remind listeners that this conference call contains 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 disorders such as ALS, the sufficiency of the company's existing capital resources for continuing operations in 2024 and beyond, the safety and clinical effectiveness of the NurOwn technology platform, clinical trials of 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, and many of these 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 call, and the company undertakes no obligation to publicly update any forward-looking statements. Joining us on the call this morning will be Chaim Lebovits, President and CEO of BrainStorm, and Alla Patlis, Interim Chief Financial Officer. In addition, Dr. Haro Hartounian, Chief Operating Officer, and Dr. Bob Dagher, Chief Medical Officer, are also on the line and will be available to answer questions during the Q&A session. I'd now like to turn the call over to Mr. Lebovits. Please go ahead.

Chaim Lebovits (President and CEO)

Hi. Thank you, Mike. Good morning and afternoon to everyone. Thank you for joining us today. We appreciate your continued interest and support in BrainStorm. We're making substantial progress in our preparations for the Phase 3b trial for NurOwn in ALS. Importantly, NurOwn is now a de-risked asset, having secured a written SPA agreement from the FDA. This significant milestone, which we announced earlier in the year, underscores the agency's recognition of the unmet need that exists in ALS. The FDA continues to be supportive, and we are grateful for their input and advice. In addition to the SPA, during the second quarter, we reached alignment with the FDA on CMC aspects of the Phase 3b trial. In June, we participated in a very constructive face-to-face Type C meeting and have now resolved previously outstanding CMC questions.

Cell therapy products have certain additional complexities in the manufacturing process. Therefore, it's important that the FDA is involved at various steps during the regulatory process, including prior to initiating a pivotal trial. Our collaboration with the CRO has been instrumental in advancing key trial protocols. We have selected a leading CRO and are diligently building a network of over 12 leading clinical centers across diverse geographic regions to ensure broad patient access and representation. The manufacturing processes are well advanced and, I believe, on track to meet production timelines. We remain committed to initiating the trial by the end of 2024 or the first Q of 2025. To bolster our financial position, we're actively pursuing non-dilutive funding, including a promising grant application with the potential to secure up to $15 million. ALS is a devastating disease with limited treatment options.

The potential impact of a successful Phase 3b trial cannot be overstated. It could represent a significant breakthrough for patients and their families. We are committed to transparent communication and providing regular updates about our progress. We are grateful for the support and collaboration of the entire ALS community as we continue this important work. I want to address the items in our proxy statement and the upcoming shareholder meeting. Nasdaq has notified us that we must maintain a $1 share price for 10 consecutive trading days by October 28th, or risk delisting. Additionally, we need to achieve a $35 million market cap. To address these challenges and provide the company with maximal flexibility, we've scheduled an earlier-than-usual annual shareholders meeting.

A key item on the agenda is seeking shareholder approval to authorize the board of directors to affect the reverse stock split if deemed necessary to maintain our Nasdaq listing. It's important to understand that approving this proposal does not automatically trigger a reverse split. It simply grants the board the discretion to implement it if it becomes necessary to comply with Nasdaq's listing requirements. While we'll exhaust all possibilities to avoid a reverse split, including driving up the share price with good news, we believe it's essential to have this option available to protect shareholder value and maintain our Nasdaq listing. The board will carefully consider all options, including a potential reverse split or transition to the OTCQB. If we opt for the OTCQB, we'll work diligently to regain Nasdaq compliance without resorting to a reverse split. Our ultimate goal is to maximize shareholder value.

We're confident in our ability to navigate these challenges and deliver value to shareholders. Your support in approving the proxy is crucial to ensuring we have the flexibility to make the best decisions for the company. Thank you for your continued support. Alla?

Alla Patlis (Interim CFO)

Thank you, Chaim. Cash, cash equivalents, and restricted cash were approximately $3.65 million as of June 30, 2024, compared to $0.75 million as of June 30, 2023. Research and development expenses for the three months that ended June 30, 2024 and 2023, were approximately $0.9 million and $2.8 million, respectively. General and administrative expenses for the three months ended June 30, 2024 and 2023, were approximately $2 million and $2.7 million, respectively. Net loss for the three months ended June 30, 2024, was approximately $2.5 million, as compared to a net loss of approximately $5.3 million for the three months ended June 30, 2023. Net loss per share for the three months ended June 30, 2024 and 2023, was $0.04 and $0.13, respectively.

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

Chaim Lebovits (President and CEO)

Thank you so much, Alla. We'll now open the call for the written questions. Please, Mike, can you please go forward with the written questions?

Michael Wood (Head of Investor Relations)

Yes. First question: Can you please elaborate on the key milestones that investors should be looking forward to in the near term?

Chaim Lebovits (President and CEO)

Thank you, Mike. Absolutely. We're very enthusiastic about the potential catalysts that should drive significant shareholders' value. In the near term, we are focused on several key milestones. Firstly, securing the first few trial site agreements is a crucial step. This demonstrates our ability to execute on the clinical trial timeline effectively, efficiently. Secondly, we're actively pursuing additional non-dilutive funding through grants and strategic partnerships. Successful acquisition of these funds will significantly strengthen our financial position and accelerate our development timeline. And of course, trial initiation and enrolling the first patient in the study are major milestones that will mark significant progress. We believe we can successfully execute on these milestones and deliver substantial value for our shareholders.

Michael Wood (Head of Investor Relations)

Thank you. Next question. With a proposed increase in the authorized shares from 100 million to 250 million, there's a possibility that existing shareholders would be significantly diluted. What is your plan for equity raises over the next several quarters?

Chaim Lebovits (President and CEO)

Thank you for this question. Very good, so we can clarify this. Of course, we understand the concern regarding potential dilution, but it's important to emphasize that an increase in authorized shares does not equate to immediate dilution. This action only provides us with the flexibility to pursue strategic opportunities without resorting to dilutive financing. Our primary focus is non-dilutive funding sources, such as grants and other strategic collaborations. We have a successful track record of securing non-dilutive funding, demonstrating our ability to execute on the strategy. BCLI has a 15-year history of efficient capital management, issuing less than 100 million shares while raising close to $200 million. This demonstrates our commitment to maximizing shareholder value. The proposed increase in authorized shares is a strategic long-term decision to ensure we have the flexibility to capitalize on future growth opportunities without unnecessary dilution.

We will continue to explore all avenues to optimize capital allocation and minimize dilution.

Michael Wood (Head of Investor Relations)

Thanks, Chaim. A related question: What non-equity funding sources currently have a good probability of transacting before initiation of the Phase 3b trial? Can you speak to grants, partnerships, or the potential outsourcing of intellectual property?

Chaim Lebovits (President and CEO)

Thank you. So we're actively pursuing multiple non-equity funding avenues. Our previous grant award from CIRM, the California Institute for Regenerative Medicine, was a good example of our efforts. It was a $60 million non-dilutive grant that supported our Phase III trial. We're also exploring partnerships with pharmaceutical companies and other industry stakeholders. These collaborations can provide both financial support and strategic advantages. Outsourcing of intellectual property is not currently on the table. We believe that maintaining control over our core technology is essential for long-term success.... However, we're open to strategic partnerships that involve technology sharing or licensing on the mutually beneficial terms. Thanks.

Michael Wood (Head of Investor Relations)

The next question regarding the planned clinical trial: Is there a take-a-peek provision that would allow interim analysis of early signs of efficacy are reported?

Chaim Lebovits (President and CEO)

Thank you. An independent data safety monitoring board is being established for the Phase 3b trial design. The board members and their charter are currently under finalization, but the DSMB will be responsible for periodic safety evaluations to monitor patient safety and well-being. Thank you.

Michael Wood (Head of Investor Relations)

The next question: Is the ALSFRS-R at enrollments higher than in the prior Phase III study?

Chaim Lebovits (President and CEO)

Thanks. Bob, you want to take that question, please?

Bob Dagher (EVP and Chief Medical Officer)

Yes, thanks, Chaim. Based on properly conducted simulation, the total score of the ALSFRS-R is expected to be higher than in the previous trial, and we're looking forward to executing the trial based on those assumptions. Thank you.

Michael Wood (Head of Investor Relations)

Also, on the trial, will there be more trial sites than in the prior Phase III, thus allowing the trial slot to be filled more quickly?

Chaim Lebovits (President and CEO)

Thank you. That's for Bob, too.

Bob Dagher (EVP and Chief Medical Officer)

Yes, thanks. I'll take that question as well. Yeah, we are planning to add a higher number of sites for the Phase 3b trial than it was conducted in phase III. For the same roughly number of patients, about 200 patients in the trial, we expect that this will increase the rate of enrollment and will also speed the timelines of the trial. Thank you.

Michael Wood (Head of Investor Relations)

Has a commercial manufacturing partner being locked down at this point?

Chaim Lebovits (President and CEO)

Haro, please take that one.

Haro Hartounian (EVP and COO)

Thank you, Chaim, for the question. We're planning for multiple manufacturing sites for this trial and simultaneously are engaged in advanced discussions with highly qualified potential commercial manufacturing partners. While we cannot disclose the specific detail until a contract is signed, we are confident in our ability to secure a suitable partner in a timely manner to support the commercial launch of our product, if and when approved.

Michael Wood (Head of Investor Relations)

Thank you. Is the Phase 3b inclusion criteria more stringent than the prior Phase III study, thus ensuring a healthier pool of patients or trial participants?

Chaim Lebovits (President and CEO)

Thank you. Bob, you want to take that?

Bob Dagher (EVP and Chief Medical Officer)

Yeah, sure. Thanks for the question as well. Yeah, we designed the phase, the inclusion criteria for the Phase 3b trial, being informed by the phase III, but they are more refined to focus on patients that are earlier in the disease, in the course of their disease. The goal is to enroll patients with a higher likelihood of showing benefit from our treatment, which we believe will be also strengthened by having an overall robust data. Thank you.

Michael Wood (Head of Investor Relations)

Thank you very much.

Chaim Lebovits (President and CEO)

I think that concludes the written questions we received. I'll leave, would you open the call for any additional questions from any investors on the line?

Operator (participant)

Thank you, sir. 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 pick up your handset if listening on speaker phone to provide optimum sound quality. Please hold while we poll for questions. Thank you. Our first question is coming from Jason McCarthy with Maxim Group. Your line is live.

Joanne Lee (Biotech Equity Research Associate)

Hi, this is Joanne Lee on the call for Jason McCarthy. Thanks for taking the question, and, congratulations on the progress made this quarter. Just regarding the CMC alignment, does the company need to implement any new actions or adjustments on the CMC front to reach, reach the alignment with the FDA?

Chaim Lebovits (President and CEO)

Thank you very much. We did. We reached alignment to be able to initialize the trial, and that was very important. But thank you for that clarifying question.

Joanne Lee (Biotech Equity Research Associate)

Got it. And just regarding the upcoming study, could you briefly remind us of the changes made to the ALSFRS-R entry criteria, and how these adjustments may impact the upcoming Phase 3b? And just as a follow-up, if you could provide a mechanistic rationale for why an effect might be more easily identifiable under the new ALSFRS-R criteria.

Chaim Lebovits (President and CEO)

Thank you. Bob, you want to take this question?

Bob Dagher (EVP and Chief Medical Officer)

Sure, I'm happy to do that. Thank you for your question. So basically, there are a number of differences from the previous trial. The main criteria regarding to the ALSFRS is, number one, to make sure that every item, the score is made up of 12 items, every item on the score, is scored at a 2, 3, or 4. Recall, each item can be scored from 0 to 4, 0 being the worst. So we are not allowing any 0 or any 1 answered on each one of the 12 items. That's number one. Number two, we are also not allowing the LS total score to be either 45, 40-- not, sorry, not 45, 46 or 47 or 48. 48 is the maximum, but once you start your first symptom, you drop from 48 typically to 47 or lower.

Reason for that is we didn't want any allowance of the 2 years since first symptom for someone 2 years later to remain without really showing signs of progression by more than 1 or 2 points. So the score allows people to enter the trial at 45 or less. Number 3, we are looking basically at associated entry criteria, such as the typical 2 years from symptom onset, looking very carefully at first symptom with a documented evidence of that, and making sure that we get patients quickly after they get diagnosed with ALS. And we're also looking to get their respiratory function to be relatively spared with looking at slow vital capacity to be above 65%, which is in the healthy side.

Combining all of those together and simulating those entry criteria I mentioned on large databases, one of them is the PRO-ACT database. We got a score, a total score, that puts us at a confidence higher than what was conducted in the previous phase III, and in line with the modern ALS clinical trials that were conducted in recent years. So we have high confidence with these criteria together to get the population of target for that will benefit most from NurOwn, which is the population that's earlier in the disease course. I hope this answers your question.

Chaim Lebovits (President and CEO)

Thank you so much, Bob. Thank you for the questions. Yeah.

Joanne Lee (Biotech Equity Research Associate)

Yeah, that was very helpful. Thanks again. Looking forward to further updates on the launch of the study. Thank you.

Chaim Lebovits (President and CEO)

Thank you so much for being on the call today. Thank you.

Operator (participant)

Thank you. Our next question is coming from David Bautz with Zacks Small-Cap Research. Your line is live.

David Bautz (Senior Analyst)

Hey, good morning, everybody. So in regards to the upcoming phase III trial, it looks like it's gonna have a 24-week double-blind period and a 24-week open-label extension. So in regards to potentially filing the BLA, assuming positive results from the double-blind portion, could the BLA be filed, essentially immediately after that, or will the data from the open-label extension be required for the BLA?

Chaim Lebovits (President and CEO)

No, we would be able to file the BLA after the first half of the double-blinded period. Thank you for that question.

David Bautz (Senior Analyst)

Okay. And I was wondering if you could comment just on the, a general sense of how, patient advocacy groups, maybe even, patients or physicians also, their enthusiasm still for NurOwn. And you've already commented about potentially seeking a non-dilutive funding, but I was gonna ask about, you know, whether the, the patient advocacy groups, would also potentially have any type of, grants or funding available for the Phase III trial.

Chaim Lebovits (President and CEO)

Thank you very much for that question. We are working diligently with many patient advocacy groups, and I can tell you that we're seeing even more excitement than the previous trial. Hopefully, we'll see that publicly and also in ways of grants. But, I can tell you across the board, patient advocacy are so very supportive of this Phase 3b trial. As you know, some of them felt that we didn't have sufficient data in the Phase III, but they're very supportive of the Phase 3b trial. They want to have an answer on NurOwn, a clear answer, and they think that the patient population that we are now focusing on, as Bob described again and again, is the patient population that has the best chance to show efficacy.

We're getting a lot of support, and I think we are getting far stronger support from different groups that wasn't there in the past.

David Bautz (Senior Analyst)

All right. Well, that's great to hear, and thanks for taking the questions.

Chaim Lebovits (President and CEO)

Yeah, okay. Operator, we have time for one more question, please.

Operator (participant)

Understood, sir. We have a question from Christopher Nicholson, who is a private investor. Your line is live.

Christopher Nicholson (Research Anayst)

Hi, good morning, everybody. I'm not a private investor. I'm a research analyst at ACF Equity Research. I wondered if you are in a position to give any color on the cost of the trial at this point, either on a per head basis or overall? And as a follow-up, are you able to give us any further color on the likely costs of production of each course of treatment at this time?

Chaim Lebovits (President and CEO)

Thank you so much for these questions. So the answer is that we are still not in a position, and we will share, of course, in the near future, more exact costs for the trial. The reason for that is that the main cost of the trial, the manufacturing costs, and we're still in the phase of finalizing contracts with different manufacturing centers from different geographies. That will be different prices, and it's not just a small difference. So therefore, we're not able to. The previous trial cost us about $50 million, and with 200 patients only for 6 months, and this time it's for 7 months, sorry, and this time it's for a longer period.

But we'll provide numbers in a later stage, and definitely not able to give you a production cost, what it will be in a few years from now, because the whole industry is changing with the CDMOs and also versus a manual process, and maybe we'll be able to implement automatic process, which significantly will change prices to the product costs. We are really focused to try to bring down the product cost. So while we are doing this huge undertaking of initiating this trial, we're still, at the same time, working very hard with R&D to get our processes cheaper as well. It's very important in gene and cell therapy, and we're very focused for that. But thank you very much for these questions.

Christopher Nicholson (Research Anayst)

Thank you. If I could just follow up slightly on that one. Is it, is it your experience that the current technological innovations that are going on in manufacturing and therapy development do they play well into your space? Is it the expectation that you could see costs reducing, or is it somewhat the other way around because you're very innovative?

Chaim Lebovits (President and CEO)

Thank you very much for that question. So if we can get a validated automated product, definitely will be dramatically decreased. But even in the manual product, we are having many good developments to get the price cheaper. I think even in this trial, we may have a dramatic cheaper price per product than previously, hopefully, in the... If you look on the whole thing of it. So but we are still working out those. Exactly, you're touching on things that we're working on now very hard, and hopefully in the next quarters, you will see different updates on this.

Christopher Nicholson (Research Anayst)

Thank you very much indeed.

Chaim Lebovits (President and CEO)

Thank you very much. So I wanna thank again everyone for being on the call today. We are finishing just on time for the 9:00 A.M., we wanted to finish this call. Thank you very, very much for everyone joining us, and thank you all for your continued support, and we're looking in the next quarter to have additional exciting updates in line to what we discussed today. We hope everything will fall in place, and we'll be in a better place when we speak in the next quarter, hopefully even after initiating a trial. Thank you very, very much.

Operator (participant)

Thank you, ladies and gentlemen. This does conclude today's conference call. You may disconnect your lines at this time, and have a wonderful day, and we thank you for your participation.