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27-05-2025
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BrainStorm Secures Key Manufacturing Partnership with Minaris for Upcoming NurOwn® Phase 3b ALS Clinical Trials
NEW YORK, May 27, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that it has signed a Letter of Intent (LOI) with Minaris Advanced Therapies, a global contract development and manufacturing organization (CDMO) specializing in cell and gene therapies, to manufacture NurOwn® for its upcoming clinical trial. The LOI marks the beginning of a strategic collaboration between BrainStorm and Minaris to initiate the technology transfer of NurOwn® in preparation for clinical trial manufacturing at Minaris' state-of-the-art facility in Allendale, New Jersey. This partnership supports BrainStorm's readiness for its planned Phase 3b clinical trial. "We are excited to support BrainStorm in the next phase of NurOwn development," said Eytan Abraham, Ph.D., Chief Commercial and Technology Officer at Minaris Advanced Therapies. "Our team looks forward to applying our expertise in cell therapy manufacturing to enable BrainStorm to conduct its clinical trial to the highest standards and move efficiently to commercial manufacturing." This partnership enhances BrainStorm's U.S. based manufacturing capabilities and supports its multicenter Phase 3b clinical trial in the United States. It complements BrainStorm's recently announced collaboration with Pluri Inc. (Nasdaq: PLUR) in Israel, which will also contribute to the production of clinical trial material. Together, these strategic relationships reinforce BrainStorm's commitment to building a robust and flexible manufacturing network to advance the clinical development of NurOwn®. Chaim Lebovits, President and Chief Executive Officer of BrainStorm Cell Therapeutics, commented, "This partnership reflects our ongoing commitment to the ALS community and to advancing a therapy with the potential to meaningfully impact patients' lives. NurOwn® represents a scientifically advanced and differentiated approach to treating ALS, and by joining forces with Minaris Advanced Therapies, we are ensuring that our clinical and manufacturing strategies are tightly aligned to support a successful Phase 3b trial and beyond," About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTSInvestors:Michael Wood Phone: +1 646-597-6983mwood@ Media:Uri Yablonka, Chief Business Officer Phone: +1 917-284-2911 uri@ Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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20-05-2025
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Q1 2025 Brainstorm Cell Therapeutics Inc Earnings Call
Joyce Lonergan; Investor Relations; LifeSci Advisors LLC Chaim Lebovits; President, Chief Executive Officer; Brainstorm Cell Therapeutics Inc. Hartoun Hartounian; Chief Operating Officer, Executive Vice President; Brainstorm Cell Therapeutics Inc. Ibrahim Dagher; Executive Vice President, Chief Medical Officer; Brainstorm Cell Therapeutics Inc. Netta Blondheim-Shraga; Senior Vice President, Research and Development; Brainstorm Cell Therapeutics Inc. Jason McCarthy; Analyst; Maxim Group David Bautz; Analyst; Zachs SCR Operator Greetings and welcome to the Brainstorm Cell Therapeutics first-quarter 2025 conference call. At this time, all participants are on a listen-only mode. As a reminder, this call is being recorded. I would now like to introduce your host for today's call, Joyce Lonergan of LifeSci Advisors. Joyce, you may begin. Joyce Lonergan Thank you, Holly. Good morning and thank you for joining us today. Before passing the call to 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. 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 and clinical effectiveness of the neuron technology platform, clinical trials of neuron, and related clinical development programs, and the company's ability to develop strategic collaborations and partnerships to support their 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 its SEC filings. The company's results may differ materially from those projected on today's call. The company undertakes no obligation to publicly update any forward-looking statements. Joining us on the call today will be Chaim Lebovits, President and Chief Executive Officer of Brainstorm; Dr. Haro Hartounian, Executive Vice President and Chief Operating Officer; Dr. Bob Dagher, Executive Vice President and Chief Medical Officer; and Dr. Netta Blondheim-Shraga, Senior Vice President of Research and Development. I would now like to turn the call over to Mr. Lebovits. Please go ahead. Chaim Lebovits Thank you, Joyce. Good morning or good afternoon, everyone. Thank you for joining us today. We appreciate your continued interest in supporting Brainstorm. We published our Q1 2025 earnings release after the market closed last Thursday, and then colleagues and I are pleased to provide a corporate update today. Our unwavering primary focus remains the execution of the clinical development plan for neuron and the initiation of our pivotal phase 3B trial designed to confirm its therapeutic benefit for individuals in the early stages of ALS. Hopefully, you will have seen the press release released this morning announcing that the U.S. FDA has cleared us to initiate the trial. This follows the amendments we submitted to our investigational new drug application. Which included comprehensive technical transfer documentation. Robust quality assurance and stringent quality control processes. This regulatory clearance marks a significant milestone, bringing us closer to commencing patient enrollment. As previously disclosed, the trial design has been carefully agreed upon with the FDA under a special protocol assessment or SPA. This spa is a crucial element, as we believe it substantially des the regulatory pathway for neuro. It provides confirmation that the trial's endpoints and our statistical analysis plan are deemed appropriate for the FDA to potentially support approval contingent upon the trial meeting its pre-specified expectations. We also announced previously. That in a face to face type C meaning we had achieved alignment with the FDA and CMC, a particularly vital aspect for an advanced cell therapy like neuron. Our CMC team is always advancing its development and continues to work as regulatory guidance directs. To provide further detail on our operational readiness for the upcoming trial. I'd like to turn the call over to our Chief Operating Officer, Dr. Haro Hartounian. Haro? Hartoun Hartounian Thank you. As previously discussed, we plan to initiate our initial manufacturing for the phase 3B trial at the Tel Aviv Sosky Medical Center. To scale up our manufacturing capabilities, we'll then proceed with the technology transfer to Pleury, which will provide additional clean room facilities. We have signed a letter of intent with Flui and anticipate moving forward to a definitive contract soon. Furthermore, The team and I have secured a leading US clinical site that has successfully passed FDA inspection. We are in the process of finalizing an LOI and we'll be announcing the details of this important site shortly. We are all very excited about the progress and remain hopeful that we can begin treating patients soon. Back to you. Chaim Lebovits For that important update on our manufacturing and site selection progress. Currently, we are actively engaged in negotiations for the clinical trial agreements with approximately 15 leading clinical centers across the United States. Each poised to serve as a site for a phase 3 trial. The strong interest we are getting from numerous renowned ALS clinicians and researchers underscores their conviction in the potential of neurons. We will make announcements regarding these agreements as they are finalized. As noted in our earnings specialty, the details of the trial, which we are calling Endurance, have now been posted on On this website you will see the study plan. Including primary and secondary endpoints. As well as a list of clinical sites that we expect will participate. Publication of these details is important for transparency and allows the medical community and mainly ALS patients who are interested in participating, as well as caregivers, to review the structure of the study. We fully recognize the urgency felt by patients and clinicians for innovative therapeutic options in ALS. Our commitment is absolute in executing this trial with the highest level of scientific rigor. With the limited treatment options currently available for ALS patients. We firmly believe that Neuron, if successful in the study and subsequently approved, holds the promise of becoming a significant and valuable treatment. In parallel with our dedicated neuro development efforts. Our scientific team remains actively engaged. With the academic community and our industry peers sharing the latest data and insights. Last week we participated in the annual AL Drug Development summit in Boston. A crucial gathering that brought together over 200 scientific leaders to address the most pressing challenges in the therapeutic development for this devastating disease. The discussions at this year's meeting were centered on critical areas such as target validation. The effective utilization of biomarkers and the optimization of clinical trial design. I want to take a moment to speak about our incredible team at. Despite facing significant financial constraints. A reality for many small biotech companies in the current environment. Their dedication and tireless efforts are truly remarkable. We heard a very powerful message at the ALS summit from Wendy. A courageous individual living with ALS. Who eloquently referred to every ALS patient as a warrior. Brainstorm, we see it as part of our mission to join the ranks of these warriors working every single day to advance preparations for the critical trial. We are succeeding in making substantial progress with the limited financial resources we currently have, thanks in large. To the outstanding support of our dedicated partners. This positions us to move forward with significant agility once we secure a strategic funding deal, which remains our priority. We understand that members of the investment community and the ELF community are eager to know precisely when the first patient will be enrolled. Please note that our focus remains squarely and diligently completing the necessary steps, including securing adequate funding as we are working to advance various funding opportunities that include potential strategic investments and non-dilutive grants. We are simultaneously proceeding on many fronts to be able to initiate the trial as swiftly and responsibly as possible. We are deeply committed to this endeavor endeavor, and we will continue to provide updates as they become available. I will now turn the call over to Dr. Bob Dagher, Grass Chief Medical Officer, who will give a brief summary of his presentation at the ELA summit last week. Bob? Ibrahim Dagher Thank you, Chaim. Hi everyone. As part of my presentation last week, I provided a detailed overview of the design of our planned phase 3 trial and explained the significant changes we made versus our trial phase 3 trial in order to increase the probability of success. As we have previously disclosed, the new trial will be conducted in two parts. Part A is a 24 week double-blind period which will be followed by Part B, a 24 week open label extension designed to evaluate long-term effects on survival and biomarkers. We have set the entry criteria to enroll patients who have early stage ALS. In other words, those with less advanced level of functional decline. The primary endpoint will be the change from baseline to week 24, so at the end of Part A, in the ALSFRSR total score, which is now considered the gold standard in recent registrational trials. The results from Part A at 24 weeks, if they meet our expectations, should be sufficient to support a new BLA. This is covered specifically in our SA agreement with the FDA. In the phase 3D trial, we eliminated the 3 month running period from the previous study and also shortened the screening period from 20 weeks to now 9 weeks to minimize changes between screening and baseline. I will now turn the call over to my colleague, Dr. Blondheim-Shraga, to discuss the ALS biomarkers analysis. Netta Blondheim-Shraga Thank you, Bob. My presentation at the ALS summit focused on biomarkers in ALS and specifically how the experiments we have conducted with biomarkers support the potential multimodal mechanism of action of neuron. There is currently no established universal marker for ALS, as it is a complex disease that may require combinations of biomarkers for accurate assessment. We hypothesize that neuron exerts its biological effects across multiple pathways. Analysis of CSF samples from patients who participated in the prior phase 3 phase 3A study provided us with valuable insight into how neurons may be exerting its effect. CSF samples were collected at 7 time points from all participants in the study, and analysis of these samples showed significant changes in biomarkers that are relevant to ALS pathology. Treatment with neuron was associated with a reduction in neuroinflammatory and neurodegenerative biomarkers and with increase in anti-inflammatory and neuroprotective biomarkers. At the ALS summit, we presented the results from 3 sets of preclinical experiments to investigate the immunomodulation, neuroprotectant, and neuroregenerative properties of neurons. The first of these was an in vitro experimental model of immune activated peripheral blood mononuclear cells, or PBMCs that were co-cultured with neuron cells. We demonstrated that neuron inhibits the secretion of pro-inflammatory cytokines and decrease the proliferation of certain types of T cells, CD4 cells and CD8 cells that are involved in the inflammatory process. The second was an in vitro hypoxia model that examined the effect of neuron on a motor neuron cell line that had been subjected to hypoxic stress in a low oxygen environment and resulted in about 1 in 3 cells dying. We showed here that when these cells were co-cultured with conditioned media collected from neuron cell cultures, viability was restored to 96.5% of normoxic conditions or 6.5% of normal. Providing evidence of a neuroprotective effect. Finally, we studied an experimental neurite outgrowth model in which human neuroblastoma cells were co-cultured in a no contact transweal system with neuron cells. We showed that neurons enhanced growth of neurites supporting a neuroregenerative role for neurons. As disclosed previously, we reviewed the clinical utility of neurofilament light, or NFL as a biomarker of disease progression and treatment monitoring in ALS. 10 patients who completed the prior phase 3 trial enrolled in an open label expanded access program that spanned 228 week periods. We showed that early treatment with neuron resulted in greater reductions in NFL levels. Among the 6 participants who received neuron in both both phase 3 and the EAP, a continual group level reduction in NFL was observed. In contrast for the 4 patients who received placebo in the phase 3. The group median NFL change was higher at the end of the study, indicating worsening neurodegeneration. After these patients switched to neuron in the EAP, the majority showed a stabilization in NFL levels. As these results were from a very small group, we view them as hypothesis generating and will continue to explore long term effects of treatment in our upcoming phase 3D study. At the conference, we also shared results from a genetic substudy conducted during our phase 3 study. We have examined the underlying genetics of ALS and how it may determine the clinical response to neuron. We are particularly interested in a gene called untertennae, which has been widely studied in ALS. Patients who were enrolled in the prior phase 3 trial had the option to participate in a genetic substudy, and 124 consented. We showed in these patients that the UNC-13A, the M13A genotype, appeared to influence the response to neuron therapy. Patients who were heterozygous carriers of oyolal, had a statistically significant response rate to neuron treatment compared with placebo, supporting further investigation of the uncertain A and other genetic factors and their correlation to treatment effect of neuron in our next trial. I will now turn the call back to Chaim for closing comments. Chaim Lebovits Thank you. For the details on Brainstorm's financials for the quarter ended March 31, 2025. I would refer you to the press release we issued on Thursday. And also to our thank you filed with the SEC. We're now ready for the Q&A, Joyce. Joyce Lonergan Thank you, Chaim. We have four written questions. The first one, can you start the trial without proper funding? Chaim Lebovits Thank you. That's a very good question. While our financials over the past year and a half have reflected a very challenging environment. We have nonetheless been able to make significant strides in preparing for the trial, including technology transfer. FDA regulatory submissions, site selections, and CRO engagements. However, initiating and successfully executing a clinical trial of this nature. Demands a robust and sustainable cash flow. Therefore, while we have diligently progressed to this point with relatively limited resources, securing proper funding is an essential to com commence the trial. As communicated in our recent press release, we are actively pursuing multiple funding avenues to ensure the timely commencement of the trial. These efforts are in various stages. Encompassing a promising $15 million dollar non grant currently will be under review, alongside ongoing negotiations for a strategic partnership. We are focusing on strategic partnerships. Our priority is to secure the necessary capital through such partnerships to confidently initiate and complete this study. Joyce Lonergan Thank you, ho. We have a second question. We see you call the trial endurance. What's the meaning of that? Chaim Lebovits Thank you for that. The name Endurance was carefully chosen. To deeply resonate With ALS community. It stands as a tribute. To the remarkable strength. Tenacity and unwavering spirit demonstrated by individuals living with ALS and their families. For Brainstorm Endurance also underscores our steadfast commitment to persevere in our scientific endeavors and generate the robust data required for regulatory approval of neurons. We believe that this trial embodies the collective resilience of patients and our determined mission to deliver a potentially meaningful therapeutic option for ALS. Thank you. Joyce Lonergan Thank you for that, Hayam. The third question is, will the company also be producing in the US? Chaim Lebovits Thank you, Harold, you want to take that? Hartoun Hartounian Sure. Thank you so much for the question. Absolutely, expanding our manufacturing footprint to the United States is a key strategic objective for us. We're pleased to share that we will be announcing a letter of intent in the coming days with the US-based facility that has a proven track record, having already successfully passed FDA inspection for the production of other other products. This signifies an important step in our plans for future commercialization and supply chain security. Chaim Lebovits Thank you. Joyce Lonergan Thank you for that. Question 4 is, can you update on any advances in the exosome program, or are you not proceeding with that at this time? Chaim Lebovits Thank you, please take that one. Netta Blondheim-Shraga Sure, thank you for this question. We are highly encouraged by the progress of our exosome program as the field of exosome-based therapeutics continues to show strong potential in the treatment of respiratory and inflammatory diseases. Our proprietary allogeneic exosome platform has yielded promising preclinical data demonstrating both therapeutic and preventative effects and models of lung disease. To support these findings, we are preparing a manuscript detailing the efficacy of our exosomes in a preclinical model of COPD. Which would join our existing publication about the efficacy of exosomes in a model of ARDS. Together, these works outline the wide potential of our allogeneic exosome technology, which is derived from neuron cells. Briefly, our new findings demonstrate that early treatment with exosomes significantly reduces lung inflammation in response to bleomycin, as a chemotherapy agent with known lung toxicity, and significantly reduced lung fibrosis two weeks later compared to untreated controls. In light of these exciting new results, we are actively pursuing strategic partnerships to advance the exosome program towards clinical development. In parallel, we are expanding our global intellectual property portfolio and anticipate announcing the issuance of additional patents that will further strengthen the protection of our innovations. Chaim Lebovits Certainly. Operator (Operator Instructions) Jason McCarthy, Maxim Group. Jason McCarthy Good morning, everybody. Thanks for taking the questions. If you can go back to the ANC 13 a discussion that you're having, have you had any communications with FDA in terms of being able to stratify by un 13A in the upcoming phase 3B or was that? Association that you showed through the EAP more of an exploratory study. Kind of, are you locked in with the spy? You don't really have a lot of wiggle room around the ALS FRS to go for on 13A stratification as well? Chaim Lebovits Thank you very much for that question, Jason. In general, the FDA is not yet approving any biomarker as a surrogate, but I would like Bob to elaborate more on that, Bob. Ibrahim Dagher Oh yeah, thank you Jason for the question. So under the special protocol assessment agreement, FDA, the protocol. I wouldn't use the word lock, but It's agreed on with all the details, including the population. It's not, Impossible or difficult to go and and add and make changes will require obviously discussions. However, scientifically speaking, the and we're very excited with the A larger ALS community about these new genetic discoveries and the young 13A story is evolving. However, it remains exploratory and not definitive at this stage in the game of the trial design innovations, etc. We are acutely aware of what's going on. I present the data on our on 13A. Two weeks ago in New Orleans at ISCT it was very well received in oral presentation. It was chosen for that for that target audience as well. So excitement is there but not yet at the level where it will rise to become a stratification point. However, we're going to be obviously exploring. We do post hoc analysis and we cut the populations in however many ways we need to eva evaluate further the effect of neuron in the next study in the phase 3B. Thank you for your question. I really appreciate it. Jason McCarthy Got it. And another, I guess somewhat technical question. You had talked a bit about the hypoxic stress co culture with neuron cells or the neuro media if I call it that, right? And did you say that it restored noroxic activity? And if so, could that Mechanism of action be used as part of a data package when you refill or ffi the BLA the next time around, because I remember last time a lot of questions around Growth factors and levels of this and levels of that came up, but is this more defining of the mechanism of action for neuron that would be supportive of the next BLA. Chaim Lebovits A very good question. Yes, and that she can answer the first part and can answer the second part. Netta Blondheim-Shraga Thank you. Yes, you're correct. What we saw was that the media that was enriched by our cells had a protective effect and a rescue effect really on cells under hypoxic conditions and restored them back to almost 100% of normoxic condition. So 96.5%. This is a cell culture, so it's a simplistic model. It's not a human, live model. It's not a clinical model, but it is supportive, I think of and was included in our ID as well. Jason McCarthy Got it. And just last question on the manufacturing. I know you're aiming to open up additional clean rooms, but currently the Tel Aviv facility, how many excuse me, therapies can it handle? What produced. Chaim Lebovits It depends on how many rules we are using. It will be a rolling enrollment, as with the phrases of the CTA, so it will serve us for the first few months and then we'll start with Pluri, as Harold had specified before, and I believe that next year we'll have the US side also up and running. Jason McCarthy Got it. Thanks for taking the question time. Chaim Lebovits You're very welcome. We have time for one more question, Ali. Operator David Bautz, Zachs Small Cap. David Bautz Hey, good morning, everyone. Thanks for the update this morning. So Haim, I just want to make sure I heard correctly that you guys are looking to open up, is it 15 clinical trial sites and then for each of those sites, obviously financing aside, what needs to occur to TRY to get or to get those sites up and running so that they can enroll patients and then lastly, do you have any estimation of how many patients you can enroll say per month just based on manufacturing capacity. Chaim Lebovits Yeah, so thank you very much for that question. So on you will see a listing of the sites. It's out there, but we didn't yet sign the CTAs. We'll be announcing very soon gradually after we sign CTAs, and yeah, as I just mentioned, we'll start to grow side by side based on a GAN of patient population that we're going to be enrolling based on the manufacturing. So we're working at the final steps steps of those of that GANT, so I can't share exact numbers now. But the plan is, as the 200 patient trial within 2 to 3 years to everyone enrolled and everyone treated. The first part and we'll be deep into the second part as well. And I want to remind you that the BLA would be filed if we have statistical significant result of the first part of the trial. David Bautz Okay great thanks thanks for taking the question. Chaim Lebovits Yeah, so thank you very much. I want to thank everyone for being on the call today. We're hoping to close at 9 o'clock. I see it's exactly 9 o'clock, so thank you very much and have a wonderful day. Operator This concludes today's conference. You may disconnect your lines at this time. Thank you for your participation. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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19-05-2025
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BrainStorm Receives FDA Clearance to Initiate Phase 3b Trial of NurOwn® for ALS
Investor call and webcast scheduled for today at 8:30 a.m. ET NEW YORK, May 19, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company to initiate its Phase 3b clinical trial of NurOwn® (autologous MSC-NTF cells) for the treatment of amyotrophic lateral sclerosis (ALS). The trial design was previously agreed upon with the FDA under a Special Protocol Assessment (SPA), confirming the study's endpoints and statistical methodology are appropriate to support a future Biologics License Application (BLA) submission. This clearance allows the company to proceed with patient enrollment. "This FDA clearance is a defining milestone for BrainStorm and the ALS community," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "We are now positioned to swiftly activate clinical sites in a phased manner, diligently preparing to enroll the first patient in the Phase 3b trial. At the same time, we are actively working to secure funding through multiple avenues, including non-dilutive grants, to ensure the timely and successful launch of this critical study." The Phase 3b trial will enroll approximately 200 participants at leading academic medical centers and will consist of a 24-week randomized, double-blind, placebo-controlled phase followed by a 24-week open-label extension where all participants will receive NurOwn®. The primary endpoint is the change from baseline to week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R). For more details on the trial, refer to ID NCT06973629. BrainStorm management will discuss this important development on its corporate update conference call and webcast, taking place today, May 19 at 8.30am ET. To access the call, refer to the events page on the company's website here. The company will provide further ongoing updates as the trial progresses and key milestones are achieved. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTS Investors:Michael WoodPhone: +1 646-597-6983mwood@ Media:Uri Yablonka, Chief Business OfficerPhone: +1 917-284-2911uri@ Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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15-05-2025
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BrainStorm Cell Therapeutics Announces First Quarter 2025 Financial Results and Provides Corporate Update
Conference call and webcast rescheduled for 8:30 a.m. Eastern Time on Monday, May 19 NEW YORK, May 15, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update. "BrainStorm continues to make meaningful progress in advancing our NurOwn® development program. Our priority continues to be initiation of a Phase 3b trial, designed to confirm the product's efficacy in early stage ALS patients and support a new BLA," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "We have a clear path forward and ongoing support from the ALS community. We recently submitted an amendment to our IND, which includes updated documentation that is essential for regulatory compliance and trial integrity. We are in the process of finalization clinical trial agreements with leading academic centers and completing other necessary steps on trial execution and manufacturing. We believe that NurOwn, if approved, has the potential to become a valuable treatment option for ALS patients. Our team is fully aligned and executing with discipline to position BrainStorm for success." Recent Highlights NurOwn (MSC-NTF) for ALS IND amendment on NurOwn submitted to FDA BrainStorm submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration for NurOwn. This important milestone sets the stage for the initiation of the planned Phase 3b clinical trial, which has been designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The trial will have a primary efficacy endpoint assessing changes in ALSFRS-R scores from baseline to week 24, and is designed to enroll approximately 200 ALS participants with early stage disease. Successful completion of the double blind part of the study (Part A) is expected to generate the clinical data to support a new BLA submission. Phase 3b trial listed on Details of the trial, known as ENDURANCE, are now available on ID NCT06973629. Included is a list of 15 clinical trial sites that are expected to participate in the trial. NurOwn® data selected as Breakthrough Science for Presentation at ISCT 2025 Meeting The new pharmacogenomic were delivered in a oral presentation at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for ALS patients treated with NurOwn. The presentation was featured in the ISCT public announcement regarding the meeting, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." Biomarker insights supporting NurOwn's mechanism and clinical impact presented at the 2025 ALS Drug Development Summit BrainStorm's senior leadership team (Dr. Bob Dagher, Dr. Netta Blondheim-Shraga and May Kay Turner) participated in sessions at the summit that highlighted insights and expertise gained throughout the NurOwn® development program. These sessions included a presentation on cerebrospinal fluid (CSF) biomarker pathways associated with NurOwn treatment, including their relationship to clinical outcomes and disease heterogeneity in ALS. Financial Results for the First Quarter Ended March 31, 2025 Cash, cash equivalents, and restricted cash were approximately $1.8 million as of March 31, 2025. Research and development expenditures, net, for the quarter ended March 31, 2025 were $1.3 million, compared to $1.0 million for the quarter ended March 31, 2024. General and administrative expenses for the quarter ended March 31, 2025 were approximately $1.8 million, compared to approximately $1.5 million for the quarter ended March 31, 2024. Net loss for the quarter ended March 31, 2025, was approximately $2.9 million, as compared to a net loss of approximately $3.4 million for the quarter ended March 31, 2024. Net loss per share for the three months ended March 31, 2025, and 2024 was $0.45 and $0.75, respectively. Conference Call and Webcast Monday, May 19, 2025, at 8:30 a.m. U.S. Eastern Time Participant Numbers: U.S. dial in: 888-506-0062 International: 973-528-0011 Participant Access Code: 621608 Webcast URL: The replay of the conference call can be accessed by dialing the numbers below and will be available for 14 days. Teleconference Replay Number: Toll Free: 877-481-4010 International: 919-882-2331 Passcode: 52457 About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTSInvestors:Michael WoodPhone: +1 646-597-6983mwood@ Media: Uri Yablonka, Chief Business OfficerPhone: +1 917-284-2911uri@ BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIES INTERIM CONDENSED CONSOLIDATED BALANCE SHEETS U.S. dollars in thousands (Except share data)March 31,December 31, 20252024 UnauditedAudited U.S. $ in thousands ASSETSCurrent Assets: Cash and cash equivalents$ 1,644$ 187 Other accounts receivable 67 63 Prepaid expenses and other current assets 621 135 Total current assets$ 2,332$ 385Long-Term Assets: Prepaid expenses and other long-term assets $ 22$ 22 Restricted Cash 182 184 Operating lease right of use asset (Note 4) 653 807 Property and Equipment, Net 382 434 Total Long-Term Assets$ 1,239$ 1,447Total assets$ 3,571$ 1,832LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT)Current Liabilities: Accounts payables$ 6,797$ 6,080 Accrued expenses 538 619 Short-term loans (Note 7) 1,200 300 Operating lease liability (Note 4) 443 549 Employees related liability 1,923 1,430 Total current liabilities$ 10,901$ 8,978Long-Term Liabilities: Operating lease liability (Note 4) 127 171 Warrants liability (Note 5) - 447 Total long-term liabilities$ 127$ 618Total liabilities$ 11,028$ 9,596Stockholders' Deficit: Stock capital: (Note 6) 15 14 Common Stock of $0.00005 par value - Authorized: 250,000,000 shares at March 31, 2025 and at December 31, 2024 respectively; Issued and outstanding: 7,911,204 and 6,141,762 shares at March31, 2025 and December 31, 2024 respectively (*) Additional paid-in-capital 222,144 218,974 Treasury stocks (116) (116) Accumulated deficit (229,500) (226,636) Total stockholders' deficit$ (7,457)$ (7,764)Total liabilities and stockholders' deficit$ 3,571$ 1,832 The accompanying notes are an integral part of the consolidated financial statements. BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIES INTERIM CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (UNAUDITED) U.S. dollars in thousands (Except share data) Three months ended March 31, 20252024Unaudited Operating expenses:Research and development, net $ 1,304$ 961 General and administrative1,785 1,513 Operating loss(3,089) (2,474) Financial income, net46 13 Loss on change in fair value of Warrants liability (Note 6)(179) (940) Net loss $ (2,864)$ (3,401) Basic and diluted net loss per share from continuing operations $ (0.45)$ (*) (0.75) Weighted average number of shares outstanding used in computing basic and diluted net loss per share6,342,002 (*) 4,315,903 The accompanying notes are an integral part of the consolidated financial statements. Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc.
Yahoo
15-05-2025
- Business
- Yahoo
BrainStorm Reschedules First Quarter 2025 Financial Results Release to Post-Market Close Today and Sets Investor Call for Monday, May 19
NEW YORK, May 15, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it has rescheduled the release of its financial results for the first quarter ended March 31, 2025, to post-market close today, May 15. In addition, the Company has rescheduled its previously announced investor conference call, which will now take place on Monday, May 19, 2025, at 8:30 a.m. Eastern Time. BrainStorm's President & Chief Executive Officer, Chaim Lebovits, will present a corporate update to be followed by Q&A. Joining Mr. Lebovits to answer investment community questions will be Haro Hartounian, Ph.D., Chief Operating Officer, Bob Dagher, M.D., Chief Medical Officer, and Alla Patlis, CPA, MBA, Interim Chief Financial Officer. Participants are encouraged to submit their questions in advance of the call by sending them to: q@ Questions should be submitted by 5:00 p.m. Eastern Time on Friday, May 16, 2025. Investors may access the conference call by dialing the following numbers: Participant Numbers: Toll Free 888-506-0062 International 973-528-0011 Participant Access Code 621608 Webcast The replay of the conference call can be accessed by dialing the numbers below and will be available for 14 days. Replay Numbers: Toll Free 877-481-4010 International 919-882-2331 Reply Passcode 52457 About Brainstorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTSInvestors:Michael WoodPhone: +1 646-597-6983mwood@ Logo - View original content: SOURCE BrainStorm Cell Therapeutics Inc.
