Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) Q2 2025 Earnings Call Transcript

Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) Q2 2025 Earnings Call Transcript August 14, 2025

Brainstorm Cell Therapeutics Inc. beats earnings expectations. Reported EPS is $-0.34, expectations were $-0.41.

Operator: Greetings, and welcome to the Brainstorm Cell Therapeutics Second Quarter 2025 Conference 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.

Michael Wood: Thank you, Kelly. Good morning, everyone, and thank you for joining us this morning. Before passing it off to the company management for prepared remarks, I would 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 disorders such as ALS, the sufficiency of the company’s existing capital resources for continuing operations in 2025 and beyond, the safety, 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, 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 call, and the company undertakes no obligation to publicly update any forward-looking statements. Joining us on the call this morning will be in Chaim Lebovits, President and CEO of Brainstorm, Dr. Bob Dagher, Executive Vice President and Chief Medical Officer; Hartoun Hartounian, Executive Vice President and Chief Operating Officer; and Alla Patlis, Interim Chief Financial Officer. So I’d now like to turn the call over to Mr. Lebovits. Please go ahead.

Chaim Lebovits: Thank you, Mike. Good morning, everyone, thank you for joining us today. We appreciate your continued interest and support in Brainstorm. I want to reaffirm that our top priority is advancing NurOwn into our planned Phase IIIb clinical trial. This trial called ENDURANCE, is designed to confirm its therapeutic benefit in patients with early stage ALS. As we previously disclosed, we reached a key milestone in May this year with the U.S. FDA granting clearance to initiate this pivotal trial. This allows us to move forward with patient enrollment, and we are preparing to begin that process. The trial for ENDURANCE has been reviewed and agreed upon with the FDA under a Special Protocol Assessment, or SPA, which confirms that the study’s endpoints and statistical methods will appropriate — will be appropriate to support a future BLA.

Assuming the data meets expectations. The SPA provides us with regulatory clarity and a strong foundation, and we believe it derisks this program. An important part of the trial preparations is to ensure we have a robust manufacturing network to supply product for the trial. We announced during the second quarter that we secured a partnership with Minaris Advanced Therapies, a global contract development and manufacturing organization that specializes in cell and gene therapies. We have initiated the technology transfer in preparation for clinical trial manufacturing at Minaris state-of-the-art facility in Allendale, New Jersey. This partnership enhances Brainstorm’s U.S.-based manufacturing capabilities and will complement our previously announced collaboration with Pluri Inc., in Israel, which will also contribute to the production of clinical trial materials.

I want to spend a few minutes talking about a recent important regulatory development, and that is the filing of the citizens petition with the FDA requesting a de novo review of the data supporting NurOwn. This petition was filed by a coalition of ALS patients and family members. As a reminder, a citizens petition filed under the Federal Food, Drug and Cosmetic Act is a formal process allowing any interested party to request FDA action, and this includes reviewing data or issuing new guidance. Although Brainstorm was not directly involved in drafting or submitting this petition or its contents, we, of course, welcome the FDA’s willingness to reevaluate existing data. We have consistently stood by the integrity and scientific validity of the NurOwn data, and we support exploration of potential regulatory pathways that may allow appropriate access to this potentially valuable treatment for patients with ALS.

We believe a comprehensive review of all evidence is essential, and this is even more important today as the regulatory landscape for rare disease with unmet needs continues to evolve. The Citizens petition is a 309-page document that incorporates a decade of data, including the results completed NurOwn’s trials as well as extensive real-world evidence such as new findings from the expanded access program. The new evidence is supported by testimony from top ALS neurologists who served as principal investigators in the prior Phase III trial. Brainstorm is committed to continuing alignment with the FDA, and we intend to proceed the Phase IIIb ENDURANCE trial, as I explained earlier. As previously announced, Brainstorm’s common stock recently transitioned from the NASDAQ Capital Market to the OTCQB.

The listing is a result of the company’s noncompliance with NASDAQ Listing Rule 550(b)(1) pertaining to its minimum shareholder equity requirement. While this was a challenging outcome, I want to be clear that it has no impact on our business operations, our ongoing R&D programs or our commitments to execute on our strategic priorities. We remain fully committed to advancing NurOwn for patients living with ALS and to progressing our clinical initiatives, including the planned Phase IIIb trial. Our vision for the company and focus have not changed. The work being conducted by our team continues, and our dedication to serving both our patients and our shareholders remains steadfast. Brainstorm remains a reporting company under the Securities Exchange Act of 1934, and we’ll continue to provide regular updates on our operational progress and financial position in full compliance with applicable SEC regulations.

We intend to keep stockholders informed of all material developments as we assess our options for relisting on the NASDAQ. Alla?

Alla Patlis: Thank you, Chaim. Our research and development expenditures net for the quarter ended June 30, 2025, were $1.1 million. This compared to approximately $0.9 million for the quarter ended June 30, 2024. General and administrative expenses for the quarter ended June 30, 2025, were approximately $1.4 million compared with $2.1 million in the same period in 2024. Net loss for the quarter ended June 30, 2025 was approximately $2.9 million or $0.34 per share. This compares to a net loss of approximately $2.5 million or $0.60 per share for the quarter ended June 30, 2024. Cash, cash equivalents and restricted cash were approximately $1 million as of June 30, 2025. I will now turn the call back to Chaim.

Chaim Lebovits: Michael, do you want to read the first question, please?

Michael Wood: First question from the investor is, when do you intend to begin the Phase IIIb trial?

Chaim Lebovits: We have made significant progress to prepare for the trial. Our team in partnership with one of the largest CROs has diligently worked to advance the trial to the point of initiation. We’re operationally ready. However, as many of you know, our ability to initiate the trial and remain listed on the NASDAQ was dependent on securing a significant round of funding. Just as we were closing in on that funding, the citizens petition was filed with the FDA. While the petition has the potential to be a powerful tool that could create an opportunity for our near-term accelerated approval while performing another trial, it has also created a new regulatory dynamic. This led many key investors to adopt a wait-and-see approach, and we were unable to close the financing required to both initiate the trial and meet NASDAQ’s minimum shareholder equity requirements.

We are now actively exploring alternative funding and remain ready to accelerate as soon as a signal is given.

Michael Wood: Next question. What happened to the nondilutive funding that you shared you would be applying for?

Chaim Lebovits: Yes. So our outstanding team did an incredible amount of work to prepare the comprehensive application for nondilutive funding. Unfortunately, due to unexpected pause and submission time line from the granting organization. By the time the submission window reopened, we were unable to demonstrate the sufficient co-funding required. The funding that we had planned to raise in June would have provided the necessary co-funding, but as we mentioned, we were unable to close that around at the time, and therefore, couldn’t submit the application yet. The good news is that once we secure that co-funding, we will be able to resubmit the application. We’re actively exploring new and alternative funding resources driven by the belief that our compelling data and the immense unmet medical need for our therapy will ultimately attract the right partners.

Michael Wood: And 1 final question. Do you plan to regain compliance and uplift to NASDAQ?

Chaim Lebovits: Yes, regaining compliance and uplifting to NASDAQ remains a core strategic objective. However, our ultimate priority is to initiate the Phase IIIb trial and get NurOwn to patients as quickly as possible. We believe these 2 goals are linked. A positive conclusion from the FDA regarding the Citizens petition, particularly an announcement that our existing data supports our BLA submission conditioned on a confirmatory trial could be a game-changing event. Such an outcome would likely enable us to easily raise the funds needed for the Phase IIIb trial. This trial design has been granted by the FDA, the first ever SPA agreement for an ALS study, which is a powerful validation of its potential to meet FDA standards for approval.

I would also like to remind everyone that NurOwn’s potential extends well beyond ALS. It’s a true platform product in the CNS space, with encouraging new survival data from our expanded access program and breakthrough pharmacogenomic data presented at the ISCT 2025 meeting. We’ve also shown very promising results in preclinical studies for other neurodegenerative diseases like progressive MS, Parkinson’s and Huntington’s, these milestones along with the foundational science reinforce our long- term vision. There’s real momentum building, and it’s rewarding to be part of something with such strong potential to make a difference. Kelly, would you want to open the call for questions.

Operator: [Operator Instructions] Your first question is coming from David Bautz with Zacks Small-Cap Research.

Q&A Session

David Bautz: Good morning, everyone. Thanks for the update this morning. I’m curious if you could discuss what some of the potential outcomes are from the Citizens petition filing for the company that is.

Chaim Lebovits: I think I just mentioned the potential outcome would be that if the FDA would find the Citizens petitions attractive? And would allow or invite us to file a BLA, that would be a very exciting moment to have. We, as you know, from day one, believed. And therefore, we filed at the time the BLA that this should be approved in conditions to a confirmatory trial and not important how you call it a Phase IV or Phase IIIb trial. And so if the FDA today would see that after rereading the Citizens petition, that would be a wonderful outcome.

David Bautz: Okay. And 1 of the — I think 1 of the most important pieces of data that was in the petition and also the company has talked about this 5-year survival data from the EAP study. I’m wondering if you could kind of put that into context, kind of discuss why that is so important. And then one of the things that I’ve been hearing also well is just a function of the patients who are earlier in their disease? Or is it actually something unique to the treatment that patients were receiving?

Chaim Lebovits: Yes. So I will just begin, but I will let Bob in a moment, give you more detailed answers. It’s in his court. But any way you look at it, and we’ve spoken to many scientists, there’s no question that seeing 10 out of 10 patients over 5 years and 6 out of 10 over 7 years without a trait, it’s impressive, very impressive. But Bob, do you want to give a more detailed answer, my educated answer.

Ibrahim Dagher: I’d love to. Thank you for the question. It’s very important for us. Obviously, we’ve been thinking about it a lot. So it’s important to note 2 important aspects of the regulatory situation for Brainstorm at this time. When — as I mentioned, when the BLA was filed, it was filed based on a 6-month study, Phase III pivotal study with data up to 6 months basically a follow-up and included an entire patient population, not only focusing on the early disease. So these are important characteristics we have today. So today, we understand that we have survival on 10 patients that continued in the EAP. So we have real world evidence beyond 6 months, obviously, as Chaim just mentioned, that 5 years, 10 out of 10 remained alive.

Shortly after 5 years, 1 participant succumbed to euthanasia by choice. And then 7 years later now, and we have survival beyond 7 years, which is unprecedented. We basically understand from the petition. Obviously, we don’t have direct access to the data, of course, that is closed now. But leading into the petition and testimonies of patients and their loved ones that this survival data is going without tracheostomy and invasive support et cetera, respiratory function remaining good, is very encouraging for us, and we hope for the FDA as well for the reviews. So that’s very critical. Also the second aspect is looking at basically the subset of population that started with early disease and did not progress to advanced disease by the time baseline came in that pivotal study is another critical aspect to look at, which is basically the hallmark for the entry criteria into the next study, the Phase IIIb study that we are preparing to start.

And that will become an important consideration and discussion that will become the confirmatory studies have we been asked — to be asked by FDA to resubmit our BLA we’ll be focusing on those 2 aspects, long-term survival, early disease outcomes and basically, hoping for an accelerated approval and followed by confirmatory study that we are committed to execute. Does that help answer all the questions or all the points about it.

David Bautz: Yes. No, that was great. Really appreciate it.

Chaim Lebovits: Kelly, any additional questions?

Operator: Your next question is coming from Jason McCarthy with Maxim Group.

Unidentified Analyst: This is Joanne on the call for Jason McCarthy. So for the first one, could you just walk us through or just perhaps, I guess, remind everyone the distinction between Citizens petition and broader public support or demand for NurOwn approval? And then if you could just outline Brainstorm’s plans that remain unchanged, mainly securing the SPA aligning manufacturing and initiating the Phase IIIb confirmatory study. Just from our perspective, I think it’s important to underscore that the path for NurOwn has always been to advance through the full clinical development process and that can’t be emphasized enough. So if you could just walk us through that, that would be appreciated.

Chaim Lebovits: Yes, Joanne. So to clarify your question, you were asking the difference of Citizens petition versus just a regular petition to the FDA?

Unidentified Analyst: Yes. Just like to the broader public support or is this like demand for NurOwn’s approval?

Chaim Lebovits: Yes. Yes, Citizens petition is a regulatory pathway, not commonly used in this type of situations. And also you don’t see Citizens petition commonly 309 pages. So I must comment that the group that did this competition put in a lot of work, months and months of work. It’s almost as a work put up into filing a BLA. And they also have the possibility which a company doesn’t have and Bob spoke that a moment ago, we can’t follow up patients after the trial is over, the follow-up of the trial. They didn’t view many people, and they are allowed to citizens and their families and they speak to each other. So they introduced a little bit more information than what we had just like we mentioned before, on the EAP patients, we learn from reading the petition that for so many years after treatment patients are stable where they’re breathing without having traits or anything like that. Yes. But Bob, do you want to comment on the second half of the question?

Ibrahim Dagher: Yes. Yes, thank you for the question. I think the key important point here is that there is, should I use the word obligation or the FDA will respond and has to respond to a Citizens petition. So as I mentioned, it’s a pathway established less used as often used. So the outcome from that is basically that the quality of the evidence presented in the over 300 pages will be met by reviewers at FDA, that will have to make a determination what to do next. And as was mentioned repeatedly, that will not change our plans, our clinical development plan is to — for us and for the patients, the community and the clinicians that we want to know the benefits of NurOwn by minimizing all biases and by basically executing this special protocol assessment agreed, the SPA agreed protocol with FDA which hold in all the important aspects to have a clear determination does our therapy benefit patients, what type of patients.

And we understand and we share that earlier in the disease are the strongest signals on the Phase III study that we conducted and the biomarker data and follow-up. So this study will have a double-blind phase at 6 months followed by open-label phase another 6 months so it gives us 1 year of follow-up on survival and biomarker and other aspects. So we’re hoping that we will hear back the FDA will have to render their opinion and that will be — the next step for us will be to meet with them and discuss the plan moving forward.

Unidentified Analyst: Got it. Appreciate the color. And you sort of touched on this, but just turning to the FDA. We’re wondering if they’re open to looking beyond ALSFRS scores as a gold standard end point just given its limitations with such heterogeneous patient population and just sort of considering other meaningful measures such as overall survival, time to ventilation or even changes to — in quality of life as supportive for an approval pathway?

Chaim Lebovits: Thanks for that question. Bob is just back from a conference. Maybe that was discussed there. No, we cannot talk on the behalf of FDA, of course, but what you raised in your question, many are raising. But in our trial, the primary endpoint is ALSFRS-R score. And Bob can elaborate that in the conversations we had this for the SPA agreement. It was very clear that the FDA is still looking at ALSFRS as the gold standard endpoint. Others are only exploratory endpoints for them. Also, we — in another conference that we were there a few months ago, which I participated James Berry, who is the Co-Chair for NEALS while talking about many other end points, I believe that in the next 5 to 10 years, ALSFRS will still be the primary gold star endpoint. But maybe you had your new insights just coming back from the ALS Nexus conference. Bob, please feel free to share?

Ibrahim Dagher: Yes. Thanks for that. Today is the last day of the conference. And obviously, your question is very important, and the entire field is very interested in it. The points are these that it is clear that under our SPA agreed protocol, the change from baseline ALS is the primary endpoint and that will not change. And to change that, we’ll have to go back and rediscuss the protocol with FDA and have basically proposed alternative, which we don’t have today, the field, and we have been investigating this for a long time, does not have a replacement today. in the past 20 years ago, plus when riluzole was approved was based on a long study and having survival as the endpoint. Unfortunately, we cannot do that today. Obviously, we cannot keep people on placebo for a very, very long time to assess survival and basically not able to run 2, 3 year studies to determine that.

So gold standard today of running 6, maybe 9 months studies, short studies, the functional assessment remains the standard. However, there are a lot of talks about adding biomarkers and quality of life or in our case here, we have the benefit of real world evidence that we’ll be discussing with the agency in those 10 EAP patients that we can talk about. But again, it’s not going to change the current plan for us without confirmatory study using the ALSFRS as the primary end point.

Unidentified Analyst: Got it. Very helpful. And looking forward to hearing more updates on NurOwn as things progress.

Chaim Lebovits: Thank you very much, Joanne. Kelly, we have time for 1 more question as we’re coming to the top of the hour.

Operator: Certainly. Your next question is coming from [ Deborah Balena ], a private investor.

Unidentified Analyst: My name is Deb Balena. My son was diagnosed with ALS at just 28 years old. After diagnosis, Matt was unable to qualify for a clinical trial, and he sought to pass the right to Try law, which was named after him. Our family is encouraged by this FDA commissioner’s commitment that they’re going to honor the spirit and the language of the right to Try law. Just as a background, Matt received 7 doses of neuron under the right to Try law. And we were unaware of the Citizens petition, which I’m very interested in. We fully support it. I read it through and through because the FDA never had an opportunity to evaluate Matt’s real-world evidence as documented in his VA records. And as an aside, his NurOwn treatment was performed at the VA under the right to Try law.

Matt’s evidence provides really unique data points for the FDA to consider. Importantly, Matt is the only one in the United States of America, who has received 6 consecutive doses at 2 months interval. He appears to have the largest magnitude and the longest lasting improvements. When he received NurOwn, Matt’s baseline score was [ 21 48 ], he had already lost more than half of its function on the ALSFRS-R score. On NurOwn, he regained and sustained 6 points of function over that period of time. He stopped using a BiPAP to breathe, he was able to stand up out of his wheelchair unassisted for the first time in 2 years, and this has never happened in any other ALS trial that I’m aware of. Applying the commissioner’s common sense test, Matt’s evidence, and I do truly hope they get to see it proves that NurOwn works.

As a mother, I’ve seen it, I’ve watched it. I’m also a Lean Six Sigma Black Belt so everything I do is based on evidence. Matt is a Navy pilot, he, as a Navy pilot had 10x the risk of getting ALS. And as a result of this service to our country, we joined in support of this Citizens position. We believe that Matt has a right to have as compelling evidence, which is very well documented, considered by the FDA. And thank you for your time, and I appreciate this opportunity.

Chaim Lebovits: I don’t know what to say Deb. I was chuckling when they said private investor. Of course, we know you. And we’re very, very proud that we were able to provide treatment to Navy Colonel like your son, very brave person and it’s the only exception we made to go with the right to try even though there is some controversy, as you know, we went with it. It was in his name, he fought for that…

Unidentified Analyst: Yes. I’m not a private investor, by the way. I’m just a mother. So I just want to be sure everybody knows that [indiscernible] I ended up in the queue this way, but here I am with our story and he is still eating, he is still breathing on his own. And his last dose was 5 years ago this month.

Chaim Lebovits: Yes, Deb. As you know, we share your enthusiasm about our product, as you know. And we do thank everyone that’s trying to create the FDA to have a second look into this. All agree that we should try to do another Phase IIIb trial, it depends on funding. FDA allowing a refile BLA, allowing a pathways for an accelerated approval would probably make it very easy to do this trial. So we’re all on the same page here Deb. Thank you for sharing. I know you did that in the panel a few months ago in a conference that I participated, it was very, very moving. You had more time than what on a call with investors. But I appreciate you calling in this morning. I really appreciate that and send our best to Matt.

Unidentified Analyst: Yes. And everyone wants to see the data, I have it. Thank you…

Chaim Lebovits: Thank you very much.

Operator: There are no questions in queue at this time, and the Q&A is concluded. I would now like to turn the floor back over to management for any closing remarks.

Chaim Lebovits: I just want to wish everyone a wonderful day and hopefully in the next call, we’re going to be after very good news. Thank you very much.

Operator: Thank you, everyone. This does conclude today’s conference. You may disconnect your phone lines at this time. Thank you for your participation.