Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) has announced its preparation for the Phase 3b trial of NurOwn in Amyotrophic Lateral Sclerosis (ALS) set for the second quarter of 2024. The company has secured a Special Protocol Assessment (SPA) agreement from the FDA and resolved critical manufacturing questions.
With approximately $3.65 million in cash as of June 2024, Brainstorm is actively seeking non-dilutive funding options and is in discussions with potential commercial manufacturing partners. Despite a net loss of $2.5 million in Q2 2024, the company remains focused on reducing production costs and is confident in initiating the trial by late 2024 or early 2025.
Key Takeaways
- Brainstorm has aligned with the FDA on the Phase 3b trial of NurOwn in ALS, with a SPA agreement in place.
- The company is preparing to launch the trial by the end of 2024 or early 2025.
- Brainstorm is building a network of over 12 clinical centers and has selected a leading CRO for broad patient access.
- Non-dilutive funding is being pursued, with a grant application that could provide up to $15 million.
- The upcoming shareholder meeting will discuss a potential reverse stock split to maintain NASDAQ listing.
- Brainstorm reported $3.65 million in cash and equivalents and a net loss of $2.5 million for Q2 2024.
Company Outlook
- Brainstorm is committed to maximizing shareholder value and maintaining its NASDAQ listing.
- The company is actively seeking non-equity funding sources and partnerships to support its financial position.
Bearish Highlights
- The net loss for Q2 2024 was approximately $2.5 million.
- Specific costs for the upcoming trial and production are not yet available as contracts with manufacturing centers are still being finalized.
Bullish Highlights
- The FDA's SPA agreement indicates regulatory support for Brainstorm's trial.
- Brainstorm is confident in securing a commercial manufacturing partner for product launch.
- Technological innovations are expected to help reduce production costs in the future.
Misses
- Brainstorm has not provided specific numbers regarding the cost of the upcoming trial and production due to ongoing contract negotiations.
Q&A Highlights
- Chaim Lebovits discussed the cost-related challenges and the company's efforts to reduce expenses through automation and cheaper processes.
- The Phase 3b trial will have more stringent inclusion criteria, focusing on patients in earlier stages of the disease.
Brainstorm Cell Therapeutics continues to make strides in the development of NurOwn for ALS treatment. The company's proactive approach to securing FDA alignment and non-dilutive funding illustrates its dedication to advancing its clinical program while being mindful of shareholder interests. As Brainstorm navigates the complexities of trial preparation and cost management, it remains optimistic about its ability to deliver on its promises to investors and patients alike.
InvestingPro Insights
Brainstorm Cell Therapeutics Inc. (BCLI) is progressing towards the Phase 3b trial of NurOwn with strategic planning and financial diligence. As investors monitor the company's trajectory, here are some pertinent insights from InvestingPro that may have an impact on their assessment:
InvestingPro Data shows a market capitalization of $25.28 million, which reflects the company's current valuation in the market. The negative P/E ratio, both standard and adjusted for the last twelve months as of Q1 2024, stands at -1 and -1.57 respectively, indicating that the company is not generating profits at this time. This aligns with the reported net loss of $2.5 million for Q2 2024. Moreover, the 1-week price total return as of a recent 2024 date is 16.04%, showing a significant return in the short term, which could be of interest to traders looking for quick gains.
Two key InvestingPro Tips for BCLI are particularly relevant given the article's context:
1. The company is quickly burning through cash, which is a critical consideration for investors as Brainstorm seeks non-dilutive funding options to support its upcoming trial. This tip underscores the importance of the company's financial strategy in the near term.
2. Analysts do not anticipate the company will be profitable this year, which aligns with the company's own reported net loss. This projection is essential for investors who are considering the long-term profitability and viability of Brainstorm.
For those seeking more in-depth analysis, InvestingPro offers additional tips on Brainstorm Cell Therapeutics Inc., providing a comprehensive view of the company's financial health and market performance. There are 10 more InvestingPro Tips available, which can be accessed for further guidance on investing in BCLI.
Full transcript - Brainstorm Cell Therapeutics Inc (BCLI) Q2 2024:
Operator: 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. And I would now like to introduce your host for today's call, Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.
Michael Wood: 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. So I'd now like to turn the call over to Mr. Lebovits. Please go ahead.
Chaim Lebovits: 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 derisked 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 FDA, during the second quarter, we reached alignment with FDA on CMC aspects of the Phase 3 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 quarter 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 28 or risk delisting. Additionally, we need to achieve a $35 million market cap. To address these challenges and provide the company with maximum 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 tax split if deemed necessary to maintain our NASDAQ listing. It's important to understand that approving this proposal does not automatically trigger a risk fit. It simply grants the Board the discretion to implement if it becomes necessary to comply with NASDAQ 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 resplit. Our ultimate goal is to maximize shareholder value. We are 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: 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 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 $4.13 respectively. I'll turn it back to Chaim to close the call.
Chaim Lebovits: Thank you so much, Alla. I'll now open the call for the written questions. Please, Mike, can you please go forward with the written questions?
Michael Wood: Yes. First question, can you please elaborate on the key milestones that investors should be looking forward to in the near term?
Chaim Lebovits: 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 side agreements is a crucial step. This demonstrates our ability to execute on the clinical trial timeline efficiently. Secondly, we're actively pursuing additional non-diluted 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 story 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: Thank you. Next question. With the 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: Thank you for this question. Very good so we can clarify this. Of course, we understand the concern regarding potential dilution. What'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 this 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: Thanks, Chaim. And 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: Thank you. So we're actively pursuing multiple non-equity funding revenues. Our previous grant award from CIRM, the California Institute for Regenerative Medicine was a good example of our efforts. It was a $16 million non-dilutive grant that supported our Phase 3 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: The next question regarding the planned clinical trial. Is there a take a peak provision that would allow interim analysis of early signs of efficacy or reported?
Chaim Lebovits: Thank you. So 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 DSMP will be responsible for periodic safety evaluations to monitor patients' safety and well-being. Thank you.
Michael Wood: Next question, is the ALS FRS or at enrollment higher than in the prior Phase 3 study?
Chaim Lebovits: Thanks. Bob, you want to take that question, please?
Bob Dagher: Yes. Thanks, Chaim. Based on properly conducted simulation, the total score of the ALS FRS 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: And also on the trial, will there be more trial sites than in the prior Phase 3, thus allowing the trial slots to be filled more quickly.
Chaim Lebovits: Thank you. That's for Bob too.
Bob Dagher: Yeah, 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 3 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: And has a commercial manufacturing partner being locked down at this point?
Chaim Lebovits: Haro, please take that one.
Haro Hartounian: 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 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. Thank you.
Michael Wood: Is the Phase 3b inclusion criteria more stringent than the prior Phase 3 study, thus ensuring a healthier pool of patients or trial participants?
Chaim Lebovits: Thank you. Bob, you want to take that?
A - Bob Dagher: Yeah, sure. Thanks for the question as well. Yeah, we designed the inclusion criteria for the Phase 3b trial, being informed by the Phase 3, 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 benefits from our treatment, which we believe will be also strengthened by having an overall robust data. Thank you.
Michael Wood: Thank you very much. I think that concludes the written questions we received. Ali, would you open the call for any additional questions from any investors on the line?
Operator: Thank you, sir. Ladies and gentlemen, the floor is now open for questions [Operator Instructions] Thank you. Our first question is coming from Jason McCarthy with Maxim (NASDAQ:MXIM) Group. Your line is live.
Joanne Lee: 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 the alignment with the FDA?
Chaim Lebovits: Thank you very much. We did. We reached alignment to be able to initialize the trial. That was very important. Thank you for that clarifying question.
Joanne Lee: Got it. And just regarding the upcoming study, could you briefly remind us of the changes made to the ALS FRS 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 ALS FRS criteria?
Chaim Lebovits: Thank you. Bob, you want to take this question?
Bob Dagher: 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 ALS FRF 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 enter 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. The reason for that is we didn't want an allowance of the two years since first symptom for someone two 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 three, we are looking basically at associated entry criteria, such as a typical two-year term symptom onset, looking very carefully at first symptoms 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 stable, looking at low 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 ProAct database, we got a score, a total score that puts us at a confidence higher than what was conducted in the previous Phase 3 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 4 that will benefit more from NurOwn, which is the population that's earlier in the disease 4. I hope this answers your question.
Joanne Lee: Thank you so much, Bob.
Chaim Lebovits: Thank you for the questions.
Joanne Lee: Yeah. That was very helpful. Thanks again. Looking forward to further updates on the launch of the study. Thank you.
Chaim Lebovits: Thank you so much for being on the call today. Thank you.
Operator: Thank you. Our next question is coming from David Bautz with Zacks Small-Cap Research. Your line is live.
David Bautz: Hey, good morning, everybody. So in regards to the upcoming Phase 3 trial, it looks like it's going to 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: 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: Okay. And I was wondering if you could comment just on the 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 going to ask about whether the patient efficacy groups would also potentially have any type of grants or funding available for the Phase 3 trial?
Chaim Lebovits: 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 will see that publicly and also in ways of grants, but I can tell you across the board, patient efficacy are very supportive of this Phase 3b trial. As you know, some of them felt that we didn't have sufficient data in the Phase 3, but they're very supportive of the Phase 3b trial. They won't 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. So 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: All right. Well, that's great to hear and thanks for taking the questions.
Chaim Lebovits: Yeah. Okay. Operator, we have time for one more question, please.
Operator: Understood, sir. We have a question from Christopher Nicholson, who is a private investor. Your line is live.
Christopher Nicholson: 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: Thank you so much for these questions. So the answer is that we are still not in a position now. 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 will provide numbers in a later stage and definitely not able to give you a reduction 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 would change prices to the product cost. 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 on that. But thank you very much for these questions.
Christopher Nicholson: Thank you. If I could just follow-up slightly on that one. 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: 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, if you look on the whole thing. But we are still wearing 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: Thank you very much indeed.
Chaim Lebovits: Thank you very much. So I want to thank again everyone for being on the call today. We are finishing just on time for the 9 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: 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.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.