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spk07: Greetings, and welcome to the Brainstorm Cell Therapeutics Full Year 2021 Conference Call. At this time, all participants are in a listen-only mode. As a reminder, this conference is being recorded. I would now like to introduce your host for today's conference, Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.
spk04: Good morning, and welcome.
spk03: Thank you for joining the call today with Brainstorm Cell Therapeutics. Before we begin the opening remarks, I'd 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 and MS, sufficiency of the company's existing capital resources for continuing operations in 2022 and beyond, the safety and clinical effectiveness of the Neuron technology platform, clinical trials in Neuron and related clinical development programs, and the company's ability to develop strategic collaborations and partnerships to support its business planning efforts. Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond Brainstorm's control. including the risks and the certainties 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 me on the call today will be President and CEO of Rainstorm, Chaim Leibowitz, Ala Patlis, Interim Chief Financial Officer. In addition, Dr. Stacey Lindberg, Executive VP and Chief Development Officer, Dr. Ralph Kern, President and Chief Medical Officer, And Dr. David Setburn, Executive VP and Chief Operating Officer, are also on the call and will be available to answer questions during the Q&A session. With that, I'd like to turn the call over to Mr. Leibowitz. Please go ahead.
spk04: Thanks, Michael.
spk05: Thanks to all listeners for joining us to discuss our full year 2021 financial results and provide corporate highlights. Brainstorm's number one priority continues to be pursuing the optimal path forward to provide broad access to neuron for patients with ALS. As we consider our strategy, we will take into account any changes in the regulatory environment that may potentially help us and are acutely aware of increased activity among advocacy groups calling for new medications that can help patients. We will be paying close attention to the upcoming FDA Advisory Committee scheduled to take place this week on March 30, at which a group of independent scientific advisors will convene to review data and testimony from key stakeholders regarding another investigational ALS drug that is currently being reviewed by the FDA. While we can't say how the FDA or its panel of outside advisors will view the data package that has been submitted on this particular drug candidate. It is very interesting and important to note that the agency accepted and granted priority review for an NDA that is based on data from a phase two trial. This together with the convening of the advisory committee may be a sign that the FDA's approach to how to improve drugs for neurodegenerative disease such as ALS is evolving. Our most significant milestone in the fourth quarter was the peer-reviewed publication of our Phase III clinical data on neurone and ALS in the journal Muscle and Nerve. The paper reported data from the randomized placebo-controlled Phase III trial that evaluated the safety and efficacy of repeat doses of neurone. Although previously announced results showed the trial did not reach statistic significance on the primary or secondary endpoints, pre-specified and post-hoc analysis showed a neurone treatment effect across both primary and secondary efficacy outcomes in those with less advanced disease. Having a peer-reviewed publication in this prestigious journal is important for us as it provides transparency to our full data set and raises awareness of the potential benefits of Neuron in the broader neurology community. We believe it may also help us in our regulatory activities. As part of our advance efforts to advance NEURON, we have an ongoing initiative to present new data to the ALS and broader neurology communities as it becomes available. We had a number of scientific conference presentations in the fourth quarter of 2021 and recent weeks, delivered both by my colleagues here as brainstorming by the clinical investigators we work with. The first and most recent presentation I want to highlight was a late-breaking oral presentation at the MDA Clinical and Scientific Conference, which took place earlier in March. At this conference, Professor Mered Sofkowitz, Chief of Neurology at Mass General Hospital and Professor of Neurology at Harvard Medical School, presented genetic analysis that evaluated how ALS-linked genes in single nucleotide polymer forms has been correlated with the response to neuron in our Phase V study. The results of these analyses showed that in patients carrying one or two copies of a gene called UNC13, there was a statistically significant higher response rate on neuron versus placebo. In contrast, the response rates in the patients who were not carriers of this particular gene were similar between the two groups. These results are very interesting, as they suggest neuron treatment may have more of an influence on disease progression in iOS patients who possess certain high-risk genes. This provides a basis for further genetic characterization and may enable the identification of biomarkers that will help us determine which patients are more likely to perform better on neuron. The last two presentations I'll highlight today are related to biomarker data from the ALS phase V data and took place at the International Symposium on ALS MND and the 2021 Northeast ALS Consortium. These presentations were delivered by two of our principal investigators, Dr. Robert Brown from UMass Medical School and Dr. James Berry of Mass General Multidisciplinary ALS Clinic. Collectively, these data show significant neural-driven changes across a range of CSF biomarkers and suggest that the product's biological activity across multiple disease pathways could prove important to slowing the progression of ALS. It also increased our understanding of Neuron's mechanism of action in ALS and provide additional evidence confirming that Neuron's mechanism is linked to its impact on ALS disease progression. Alongside our pursuit of the optimal path forward for Neuron and ALS, we also continue to make progress in establishing our manufacturing preparedness. We had previously entered into a partnership with Catalent to provide GMP clinical supply of Neuron in anticipation of the product's potential regulatory approval. We announced in December that the technology transfer at Catalan's facility had been finalized, a very important step in this process. The manufacturing of cellular therapies such as Neuron is complex and requires careful planning and very specific expertise. We are very pleased with the progress we have been making at Catalan, which has industry-leading capabilities in this area. Also in December, we provided an update on our expanded access program for Neuron and announced that the FDA had asked us to submit a protocol amendment to provide additional doses for patients who are participating in EAP under the original EAP protocol. Participants who have completed the phase three Neuron trial and who met specific eligibility criteria have had the opportunity to receive three doses of Neuron. Under the amended EAP protocol, these eligible participants will receive up to three additional doses and so have the opportunity to receive as many as nine doses of Neuron in total, three during the Phase III trial, three under the original EAP protocol, and three more under the amendment. This will allow for additional data collection that will help us better understand the potential benefits of longer-term treatment. Finally, there was a recent IP update I want to mention. In February, we were granted a new patent in Brazil that covers Neuron's manufacturing process. A key element of Brainstorm's overall strategy is to establish a broad intellectual property portfolio to protect our proprietary technologies and products. Adding this Brazilian patent to our existing portfolio of U.S., Canadian, European, Israeli, and Japanese patents should position us well to enter into new commercial partnerships for Neuron in South America as well and worldwide. I'll now turn over the call to Alaa Patlis, who will review our financials. Alaa?
spk01: Thank you, Chaim. It is my pleasure now to walk you through our full year 2021 financial results. Brainstorm's cash equivalent and short-term bank deposits were approximately $22.1 million as of December 31, 2021. This compares with approximately $41.9 million on December 31, 2020. Our research and development expenditures net in the year ended December 31, 2021 were $15.3 million compared to $22.3 million for the year ended December 31, 2020. Excluding participation from IAA and other grants and proceeds received under the hospital exemption regulatory pathway, research and development expenses were $15.8 million in 2021, compared with $24.6 million in 2020. General and administrative expenses for the years 2021 and 2020 respectively were $9.3 million and $9.4 million. Net loss for the year ended December 31, 2021, was $24.5 million, or $0.68 per share, as compared to a net loss of $31.8 million, or $1.07 per share, for the year ended December 31, 2020. Back to you, Haim.
spk05: Thank you, Alaa. Mike Colwood from LifeSci will now read questions we have received from investors.
spk03: Thank you, Haim. And the first question is regarding the regulatory strategy for Neuron. This investor would like to know, when do you intend to file a BLA for Neuron and ALS, and can you explain why you did not share more details on the regulatory strategy on ALS up to this point?
spk05: Thank you, Michael. I'll take this one. We've had a very productive year and have been good stewards of the talents and resources we have at Brainstorm. We're continuing to invest in our future, keeping our sights on the long-term while delivering on the near-term priorities. To give more detail at this time, just two days before a scheduled FDA advisory committee that will be discussing another product trail of patients may turn out to be counterproductive and not business smart. We remain steadfast in our belief of neurons' ability to combat this terrible disease. Some of our highly regarded PIs, principal investigators, who are close to our data and have cared for participants in our trial firsthand, have voiced their belief at various scientific conferences in the efficacy and safety of Neuron. They've made public statements calling for access to Neuron in parallel to generating additional evidence of its clinical benefits. We have done the job of allowing the evidence that supports our product to be evaluated by the scientific community, first by publishing a full and transparent analysis of our phase III data in muscle and nerve, a respected period journal, And as guided by the FDA, we have presented and continue to present our data and seek input from neurologists, statisticians, and advocacy organizations around the world. We are gaining very valuable and important feedback from this process. We will continue to work with the FDA to determine the best path forward for neuron Our aim is to seek the fairest way to make neuron available as soon as possible for as many ALS patients as possible and at the same time create value for our stakeholders. As mentioned, later this week we'll have an opportunity to learn through an advisory committee how the FDA and the advisory committee will apply the 2019 FDA ALS guidelines document for therapy development that reiterated the need for urgency and regulatory flexibility. We believe this meeting will provide important information to brainstorm and the community at large that may be able to further guide our decisions for the best way forward. In summary, let me be clear that we do remain committed to advancing this program for ALS patients and pursuing the best and most expeditious path forward to enable patients access. Thank you.
spk03: Next question. You referred to the UNC13A gene in your press release about the MDA presentation. Can you please explain exactly what this gene is and what its relevance is to ALS pathogenesis?
spk12: Yes. Thank you.
spk05: Ralph, would you take this one?
spk12: Of course. A hallmark feature of neurodegenerative diseases including ALS is the depletion of the RNA binding protein TDP43 from the nucleus of neurons in the brain and spinal cord. There are genetic variations called single nucleotide polymorphisms in this gene, and they're among the strongest hits associated with ALS in human genome-wide association studies. In fact, a direct link has been found between UNC13A risk allele and the deleterious effects of TDP43. And a recent paper suggests that a single nucleotide polymorphism in this gene may underline differences in treatment response. From a broad look at the literature, we know that UNC13A C allele is associated with a higher age at symptom onset, a more frequent ball bar onset, high incidence of ALS, lower breathing function, shorter survival, and lower scores on ALS-specific cognitive tests. And we know that published data suggests that the C allele on this genetic variation contributes to the clinical heterogeneity found in ALS, including the risk of being diagnosed with ALS and having a worse prognosis with the disease. In fact, retrospective analysis from 2017 published in Neurology Across Trials noted that there was patients with a C risk allele of this gene responded to lithium carbonate compared to those who received placebo. This analysis led us to pre-specify this gene and this genetic variation in our phase three trial. And as I just mentioned, Dr. Merit Sutkiewicz from Mass General presented these data at the recent MDA conference in a late-breaking session just a few weeks ago. And what she shared was that neuron-treated participants with the AC genotype were almost nine times more likely to respond to neuron treatment compared to placebo. And these results offer great promise for the development of future treatments for ALS in addition to accumulating evidence for the effectiveness of Neuron. If you're interested in reviewing the presentation, you can access it on the investors and media section of our company website. Thank you, Raul.
spk03: Next question. Can you help us understand what you're gaining from all these presentations at scientific conferences regarding the biomarkers and genetic results UNC 13A, and how do you think this helps you with your regulatory path going forward?
spk05: Casey?
spk09: Sure. So, while our early efforts focused on clinical outcomes from Phase 3, this question notes that we've continued to broaden the focus of neurons effectiveness by presenting biomarker and genetic data, both of which add to the compelling case for neurons. The biomarker data presented at scientific meetings and to the community shows that neuron decreases markers of neuroinflammation and neurodegeneration, while increasing markers of neuroprotection. And furthermore, the changes in biomarkers observed with neuron, which are not observed with placebo, can even help explain with great accuracy the clinical response that we observed in our phased-through trial using pre-specified statistical models. Ralph just referred to some of the really exciting and emerging literature, and we're living in a very exciting time in terms of the ability to learn and understand insights into ALS. From a genetic perspective, what we're seeing focusing on UNC 13A certainly is one of these dimensions. And as our analysis was informed by past literature, We felt it was important to share with the scientific community our own pre-specified genetic analyses of our neuron phase-through trial, which suggested and suggests that the neuron treatment in this trial may influence disease progression in ALS patients who possess UNC13A risk allele and provide a basis for further genetic characterization in future clinical trials. If we step back and we look more broadly at our clinical plan, We've delivered a series of four clinical trials in ALS, including double-blind and uncontrolled, phase two and phase three clinical trials. So if I think about this question, so why are these presentations important? Well, these pre-specified biomarker and genetic data add additional layers of credibility and scientific rigor to our findings related to the functional clinical endpoints that we've published from these trials.
spk05: Thank you so much, Stacey. Michael?
spk03: Next question. If AMX0035 is approved by the FDA, how do you think this will affect the market opportunity for Neuron? Stacy?
spk09: It would not be appropriate for us to comment on the likelihood of approval of AMX0035. We believe that each therapy should be evaluated on the scientific data and evidence generated individually. Yet, as a part of the ALS community, we will celebrate all success in terms of treatments becoming available to patients.
spk03: Thanks. The next question is regarding the progressive MS program. Can you please provide an update on this program?
spk05: Sure, Dr. Kern.
spk12: Ralph. Thanks for the question. So, as you know, we've completed additional analyses of the Phase II study that will be shared upcoming scientific meetings and in a peer-reviewed publication. Just to give you a little color on this issue and three examples, the scientific abstracts that will be shared at the upcoming CMSC meeting in May provide important information on three areas. One is the quantitative relationship between neuron treatment and CSF inflammatory biomarkers. A second is a closer examination of visual outcomes, looking at individual eye responses in study participants. And finally, we did preclinical work that we'll be sharing on the interaction of neuron with ceponimod, which is a S1P receptor modulator and a recently approved MS therapy. So, as you can see, we're continuing very active dialogue with the MS scientific community and experts. We're also continuing our dialogue with regulators about the optimal path forward, and we'll be prepared to share details after publication of our Phase 2 data. Thank you, Ralph.
spk03: Thanks. And regarding the Expanded Access Program, did the FDA contact Brainstorm to request the latest EAP, or did this happen the other way around?
spk05: Yes, FDA requested and authorized the Brainstorm extended dosing period for the participants who have completed the EAP by allowing three additional doses of Neuron administered every two months.
spk03: As a follow-up to that question, is Brainstorm actively collecting usable data from the EAP? Lacey?
spk09: Yes, I'm happy to confirm Brainstorm is actively collecting both clinical and biomarker information in the EAP protocol.
spk05: Thank you very much, Michael. I think that's done with the written questions we included today. Holly, would you open for any questions from the audience?
spk07: Certainly. Ladies and gentlemen, the floor is now open for questions. If you have any questions or comments, please press star 1 on your phone at this time. We ask that while posing your question, you please pick up your handset if listening on speakerphone to provide optimum sound quality. Please hold while we poll for questions. Your first question for today is coming from David Bouts. Please announce your affiliation, then pose your question.
spk11: Hey, this is David Vowles from Zach's Small Cap Research. Thanks for the update this morning. And, Haim, I'm curious, now that the Phase 3 results have been published, are you receiving any additional feedback from physicians, patient advocacy groups? Maybe if you could tell us a little bit about what you're hearing from people that have been able to see the full data set.
spk05: Thank you, David. Well, as you could figure out from our call today, I know many investors want us to talk more, but it's business-wise and regulatory-wise better we talk less. We're at a time that we're working very hard for the ALS community, and it's maybe not obvious exactly what we're doing, but you just mentioned part of it. We are talking to many stakeholders, and yes, we are getting very good feedback and vast majority very positive feedback. And there's strong recommendations which we can't share. We don't think it would be right to share. Specifically, you know, we're in a very sensitive week, and we know this for months that this outcome is happening this week. And it doesn't make any sense that we talk more publicly at this time. I know you're an analyst. Tell me if I'm wrong.
spk11: No, I completely understand. So... Since you can't talk all that much about what the FDA is doing, maybe I could ask, are you considering going to other jurisdictions and approaching them about approving or about filing for the approval based on your results so far?
spk05: That's a tricky one.
spk04: So, yeah.
spk05: You know, I understand the questions coming from because other companies did go that path and definitely we're considering all paths. We know what's going on. We're following everyone's path and we're trying to understand what those interactions did to help their way forward or not. And not what it seems obviously always is the backstory as you know. So we're learning that very, very diligently and we think we know where we are and uh, When we'll make a move, you'll hear it. That's our decision. Rather than discussing upfront strategy, we are, as I said, we're getting prepared for every scenario.
spk11: Okay. And then my last question is, so outside of the expanded access program through the FDA, is anyone else receiving neuron treatment at this time?
spk05: No. There's huge activities at the Catalan site just to get them ready for success as a manufacturing center. So a lot of activity outside of the providing just DAP patients, but only DAP patients that are getting their own for now.
spk11: Okay, great.
spk04: All right. Well, thank you for taking the question. Sure. Next question, please.
spk07: Once again, if there are any questions or comments, please press star 1 on your phone at this time. Your next question is coming from Michelle Lorenz. Please announce your affiliation, then pose your question.
spk10: Hi. I am with Voices for ALS. And I noticed a couple weeks ago that Merit Sikovich talked about your IPSC cells, that you were going to be doing some confirmation of UNC 13A. Can you talk a little bit more about that? I didn't hear you discuss it on the call.
spk04: Yes, we did. But Stacey, you want to take this one?
spk09: Yes, I'm happy to. So I think this was part of an interview that she participated in, and it was a discussion really around how we can leverage genetic insights and further research to help us understand the implications of results in clinical trials, but then also to think about the future trials. And the focus was really with the materials that we have now, how can we provide additional confirmation of the results that we're seeing in this trial? And so we can can take genetic materials that we have now and run additional preclinical experiments, which was really the basis of her conversation. And so those are plans that we are undertaking. We are certainly exploring how we maximize the resources we have and bring new additional insights to the table from our phase three trial.
spk05: Yeah. Thank you, Michelle, for that opportunity to clarify that part of the interview. People misunderstood it.
spk04: Any other question, Michelle?
spk08: No, thank you.
spk04: Thank you. Next question, please.
spk08: Your next question is coming from John Murphy.
spk07: John, your line is live.
spk13: I thank you. I was just wondering, in the last conference call, you said you were going to meet with the people from ALS. And after that meeting, you were going to immediately submit the BLA. I was wondering, did you meet with the people of ALS? And if you didn't, why not? Thank you.
spk05: Thank you very much, John. So, as we said, we are meeting with the many stakeholders, clinicians, and yes, we are meeting with patients of ALS as well. But as we explained again and again on this call, that there's a lot of regulatory activity ongoing now. Therefore, we are not choosing to share the exact strategy of our BLA filings. I understand the need of investors and the urgency to know what our plans are, but in the favor of the program to get better chances to have faster access for patients, we're taking this route. I just want to reassure everyone with ALS, it's very important for me, that we know and feel the urgency. I think that's what drives me and all of my colleagues day in and day out. We work very hard to produce and create the best possible outcome for ALS patients. Sometimes that calls to lay quiet. ALS patients, in my view, couldn't have a more driven team than this team at Brainstorm. I am very proud of this very devoted team, and I think all of you should be. That's what I can say.
spk04: Thank you for the question.
spk07: There are no further questions in queue. I would like to turn the floor back over to Mr. Lebowitz for any closing comments.
spk05: Yes, Holly. Please, if you can pull for another question. I just got an email that people are looking, and also I see John wants to ask another one. Absolutely. We want to allow him to ask another one, so he can say that we are allowing all his questions. Please, John.
spk07: John, your line is live.
spk13: Yes, thank you. I saw that Mr. Ness invested quite heavily in Brainstorm. Is there any plans for him joining the board or joining the Brainstorm team? Thank you.
spk05: Thank you for that question, but I cannot comment on behalf of Mr. Ness without getting his approval for that. You must understand that we are very appreciative of Mr. Ness being a major shareholder in Brainstorm, but thank you for that question. I'll pass it on to Mr. Ness. Paul, if you can pull for another question, I just had an email that someone else was trying to get out. If you can give them information how to.
spk07: Absolutely. Ladies and gentlemen, if you have any questions, please press star 1 on your phone at this time. Your next question is coming from Ken Hackle. Please announce your affiliation, then pose your question.
spk02: Yeah, I'm with Scopus Holdings. And the question is more on the financial side. I see you burned through something like 47% of the current assets in the past year. Give me your plans for the current year. What are your thoughts with your capital position, your cash position, what you need to do going forward?
spk05: Yeah, very good question. So as you may well know, we have an open ATM for up to $100 million. We didn't tap into it. And with these prices and these volumes, we were very careful, you can see, over the last few years. how careful we are with the ATM activation. On the other hand, we have enough cash for going forward at least over a year and a half. Our burn rate is really down dramatically, about 30% lower than before. The main reason is because we don't have an ongoing clinical trial now. So we do have manufacturing expenses, but no clinical trials for the moment. So we're fine as it is, no pressure whatsoever.
spk06: Thank you for that question. There are no further questions in queue at this time.
spk05: Okay, Holly, so thank you very, very much, and thanks for everyone participating and all the questions, and I wish that we can have a very good upcoming quarter, a very exciting quarter, definitely, to see how things evolve, and I look forward to talk to you and be able, hopefully, to share far more information with you in the next queue. Thank you very much.
spk07: Thank you, ladies and gentlemen. This does conclude today's event. You may disconnect at this time and have a wonderful day. Thank you for your participation.
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