Brainstorm Cell Therapeutics Inc. Q1 FY2022 Earnings Call

Greetings and welcome to the Brainstorm Cell Therapeutics First Quarter 2022 Conference Call. At this time, participants are in a listen-only mode. As a reminder, this call is being recorded. And I would now like to introduce your host for today's conference, Tom Galassi of LifeSci Advisors. Mr. Galassi, you may begin.

Good morning and thank you for joining us. Before we begin the opening remarks, we would like to remind listeners that this conference call contains numerous statements, descriptions, forecasts, and projections regarding Brainstorm Cell Therapeutics and its potential future operations and performance, statements regarding the market potential for the treatment of neurodegenerative disorders, such as ALS and MS, the sufficiency of the company's existing capital resources for continuing operations in 2022 and beyond, the safety and clinical effectiveness of the NurOwn technology platform, clinical trials of NurOwn and related clinical development programs, and the company's ability to develop strategic collaborations and partnerships to support 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 presented on today's call. The company undertakes no obligation to publicly update any forward-looking statements. Joining me on the call today will be Chaim Lebovits, CEO of Brainstorm; and Alla Patlis, Interim Chief Financial Officer. In addition, Dr. Stacy Lindborg, Executive Vice President and Chief Development Officer; Dr. Ralph Kern, President and Chief Medical Officer; and Dr. David Setboun, Executive Vice President and Chief Operating Officer, are also on the call and will be available to answer your questions during the Q&A session. Now, I would like to turn the call over to Mr. Lebovits. Please go ahead.

Thanks, Tom. Thank you to all listening for joining us to discuss our first quarter financial results and corporate highlights. Given that we provided the detailed update on our clinical programs during our year-end call only about six weeks ago, I'll keep my prepared remarks brief today as is our usual practice. We'll follow our prepared remarks with addressing questions we received from investors in advance, as well as taking live questions from those of you listening on the call today. I want to emphasize that Brainstorm’s highest priority is pursuing the optimal path forward to provide broad NurOwn for patients with ALS. Our continued efforts towards this goal are moving forward and have been advanced in many ways, including ongoing interactions with ALS experts and leaders in the clinical and patient communities. As I previously stated, valuable feedback and insights gained from these interactions are critical and are guiding the specific steps we will take to move them forward. Our dedicated team has continuously received expert feedback on the NurOwn clinical data set since our Phase 3 trials' initial top line read-outs. However, the insights provided to us throughout the first quarter have been particularly insightful, given that we published our full results near the end of 2021. The availability of the Phase 3 publication enabled us to provide the ALS expert community with a full and transparent view of our randomized placebo-controlled Phase 3 trial data that has been validated through the rigorous peer review process. As a reminder, although the Phase 3 trial did not see statistical significance on primary and secondary endpoints, pre-specified and post hoc analyses showed NurOwn delivering robust clinical benefits to ALS patients with less advanced disease. These clinical findings are further supported by biomarker data, showing significant NurOwn-driven changes across important ALS disease pathways such as those related to neuroprotection and neuroinflammation. In terms of the publication of our paper, we have had the opportunity to present data from our Phase 3 trial at three scientific conferences, including the annual meeting at the American Academy of Neurology, and we will deliver an additional biomarker presentation at the first ALS Drug Development Summit later this month. We have been highly encouraged by the feedback received to date, which we are incorporating into our business and regulatory strategy as we move forward. Given the sensitive and confidential nature of our ongoing communications with regulatory authorities, we are not in a position today to disclose the specifics of these interactions. I’ll stress however that we continue to make tangible progress behind the scenes and I look forward to when we can share a comprehensive update. We appreciate the urgency of the needs facing the ALS community and I assure you that our entire team is working diligently through a coordinated effort to address these needs as expeditiously as possible. In parallel with our efforts to advance NurOwn down its optimal growth path, we are adding talented and dedicated individuals to our leadership team. Just this past week, we announced the appointment of Dr. Netta Blondheim-Shraga as VP of Research & Development and Antal Pearl-Lendner to the newly created position of Chief Legal Counsel. We are thrilled to welcome both Antal and Netta to Brainstorm and expect that their complementary skill sets and experience working within industry leaders such as GE Capital and Teva Pharmaceuticals will drive our continued progress. In regards to our pipeline, we continue to make progress developing our proprietary technology platform, which has applications in multiple disease areas. Dr. Kim Thacker, our Senior Vice President of Medical Affairs and Clinical Innovation, presented new pre-clinical data from this program at the International Society of Cell & Gene Therapy Conference earlier this month. This data builds upon prior pre-clinical studies demonstrating the superior efficacy of NurOwn-derived exosomes in a model of acute lung injury when compared with exosomes produced from naïve mesenchymal cells. With the new data presented at ISCT, we gained important insights into the biological mechanisms underlying the superior efficacy of NurOwn-derived exosomes, which appears to be linked to their anti-inflammatory effect on macrophage populations. We look forward to discussing our exosome program further during an upcoming presentation at the ISCT 2022 Annual Meeting, which will take place in Lyon, France on May 26. I will now turn the call over to Alla Patlis who will review our financials? Alla?

Thank you, Chaim. It is my pleasure now to discuss our financial results for the first quarter ended March 31, 2022. Brainstorm's cash, cash equivalents, and short-term bank deposits were approximately $18.4 million as of March 31, 2022. This compares with approximately $22.1 million on December 31, 2021. Our research and development expenditures in the first quarter of 2022 were $2.6 million, compared with $4.3 million for the comparable period in 2021. General and administrative expenses for the first quarter were $2.8 million, compared with $2.6 million in the comparable period of 2021. The net loss for the first quarter was $5.4 million or $0.15 per share, as compared to a net loss of $6.7 million or $0.19 per share for the comparable period in 2021. Back to you, Chaim.

Thank you, Alla. Wonderful job. Tom Galassi from LifeSci will now read the questions we have received from investors. Tom?

Thanks Chaim. Our first question asked if you could provide an update on the regulatory status of NurOwn and ALS?

Stacy, do you want to take it?

As Chaim mentioned in his prepared comments, we continue to leverage expert feedback as we work collaboratively with regulators to enable NurOwn advancements. Our aim is to seek the most expeditious path forward to enable patient access and at the same time create value for our stakeholders.

Okay. For our second question, we have: what are you gaining from recent presentations at scientific conferences?

Yes. I'll ask Ralph to address that. I'm just sharing with the shareholders and other people listening that it is the first time in a long time since COVID that we're doing the call in the same room in our offices in Boston. Ralph, take it.

Thanks Chaim. As we mentioned, we continue to broaden our understanding of NurOwn’s mechanism of action in ALS and in progressive MS, while at the same time providing key scientific insights into the enhanced immunomodulation and protection of our platform technology. These important scientific insights that we will share over the next few months will support a few activities. First of all, regulatory activities. Secondly, they'll definitely increase scientific credibility with the broad scientific community. And finally, they'll provide support for both internal and external discussions related to strategic partnerships.

Thank you.

Our next question asked: can you provide an update on your progressive MS program?

Yes, that's Ralph.

Thank you. As we mentioned, we've completed additional analyses of the Phase 2 study and we plan to share these insights at the upcoming CMSC meeting in June in D.C. and at the meeting in the Fall, which will be in the Netherlands. These scientific presentations will support continued development of NurOwn and progressive MS in our view, but we've also received valuable feedback from MS scientific experts and regulators over the last two months. And once the Phase 2 study is published in a peer-reviewed journal, we’ll provide a further update.

Thank you, Ralph.

Okay. And the last pre-submitted question asks: can you provide an update on your ALS Expanded Access Program?

Yes, sure. Thank you, Tom. We continue to provide additional treatments through the intermediate-sized EAP protocol that the FDA has approved. We continue to actively collect clinical and biomarker data from this program and hope to provide further updates as they become available. I wish the best success to all the patients getting treatments these weeks. I would like you to please open the call for questions from people on the call.

No problem at all. Okay. Your first question is coming from David Bautz of Zacks Small-Cap Research. David, over to you.

Hey, good morning everybody. Well, I know you can't go into the details. I'm just curious if you've had any further meetings with the FDA or if you guys do decide to go down the path of filing a BLA, would you plan on having additional meetings with the FDA before you do that?

I'll answer on the second part of the question. There are different ways how you can talk to regulators. There are official meetings, and if there are sometimes important programs to the agency, they have ways to talk to us. So, we keep on saying that we have an ongoing conversation with regulatory and that's true. It continues to be true. That’s as far as I can go this morning, David. I know we discussed also offline and also with other investors. Everyone wants to know where we are? We'll share as soon as we can. We are doing the best for shareholders of not yet sharing where we are, but everything is on course. To your question. Yes.

Okay thanks. Now regarding exosomes, I'm curious what potential other indications you could look at for that platform outside of the acute lung injury, which you've already shown pre-clinical data for us today.

This is a wonderful question. Again, I can't share too much, but I will share with you that we just announced that we appointed a new VP of R&D and she has a lot of expertise and that’s exactly to drive from a scientific good indication to drive to, okay, where is this going? What are the diseases? Who are the right partners? That's what she has done in 12 hours to use consortiums and bring things from academic through to company development. So, of course, we are not academic, but our R&D team is more academic-driven and it's going to be more business-focused also to try to figure out with whom to partner and what different indications. It's a very good question. So, we're looking at the whole broad possibilities and she is very professional with the team and she may bring out more people to assist her to do exactly that: be able to outline the company to move very fast in the right direction. So, while we are already focused on lung disease in different ways, this will be an add-on this year. Exosomes are becoming very, very hot in our industry, as you know.

All right. Sounds good. Thanks for taking the questions this morning.

Thank you. Next question is coming from Michelle Lorenz of Voices for ALS. Over to you.

Good morning, everybody. I have four questions. Two about biomarkers and two about patient-reported outcomes. The first biomarker question relates to your CSF biomarkers. At the recent meeting, Dr. Billy Dunn talked about the importance of neurofilament light trending in the right direction. And earlier last year, at the end of last year, Dr. Brown said that NFL in the NurOwn trial was trending — I think his quote was 'eye popping.' So, I'm curious if you can comment on which of the CSF biomarkers you found the most compelling? And did you see a larger magnitude difference in the higher ALS FRS scores just as you did in the clinical trial data that was published in Muscle and Nerve?

Thank you so much. So, the answer for that is that we are not claiming a single biomarker support only. We don't think that ALS, none of our scientists or neurologists think that ALS will be driven only by a single biomarker. We do know, as you mentioned, that Dr. Dunn and others have mentioned they would like to see NFL going in the right direction as an additional credibility to ALS results. We can say that we have that, and not only with NFL. We have many biomarkers supporting our ALS score story that we are claiming shows that our trial and our treatment are effective. We did lay out part of the biomarker story in our manuscript, but there's still so much to share and we are working in additional manner that will lay out actually a lot of what you were asking in more detail.

You may not be able to answer the second question. I noticed that Dr. presented at the conference discussing UNC13A, and I believe the data showed that about 65% of the participants met the primary endpoint, with a P-value around 5. Given the misplacing error related to UNC13A, do you think that NurOwn might demonstrate effectiveness on the other genes with misplacing errors identified in Aaron Gertler’s paper in Nature?

You're asking a very good question and Dr. Sakovich did share whatever was able to be shared at the time. It doesn't mean that we say that only A versus C works. It is far more complex as you know. I think, first of all, you really follow all our presentations very closely, and our scientists are very excited that we are paving the way first in biomarkers. As someone following many ALS trials, I know that you know we are the first with the largest biomarker data to bring forward in the ALS trial. And now, thank God, all other trials are now understanding that biomarkers have to be part of trial collection. Our data set of biomarkers brings important information, and we are showing very interesting data as you mentioned. We can of course include that in now biomarker paper. It will have the generic section, but it's only the beginning. I think our biomarker data will be far more helpful at this moment than the genetic data, but the genetic data is very supportive of what we're seeing, definitely.

To that end, I'm also a believer that the patient-reported outcomes are compelling. And as you know, sadly, COVID halted the collection of breathing data in the Phase 3 trial, but it appears from the people in EAP that some people are having significant improvements in FCC. One person obviously stopped using a trilogy, and it's been two years. I checked with his family; they still aren't using the trilogy two years since his last dose. So, I'm curious: is NurOwn upregulating the epidermal growth factor or is there another biomarker that you believe might be responsible for targeting the phrenic nerve that innervates the diaphragm?

Michelle, I wish all the investors would know what you know in detail, but you know that I can't comment on this EAP open program. Wow, you follow everything. Nice. And I can't really comment on this. I can say, you know what? I will say that we are very happy that we're able to provide, of course, the EAP, that's course, given everyone all that. We've done this not really with all the companies do. We're a small advisory company, investing a lot of our money and resources to provide to these few patients at least, more and more treatments. We're very happy that the FDA is supporting this program in a very strong way, and yes, we see what you see, Michelle. We see the same things of course. You're not seeing wrong things, but we just can't comment on them.

Okay. Then the last quick question is, again in EAP, a lot of people have reported that their fasciculations have stopped immediately after getting their NurOwn injection, that's obviously nowhere reflected in the ALSFRS-R. How is Brainstorm capturing this data to provide it to the FDA in support of how a patient feels and functions?

Again, Michelle, it’s a very, very good question. The whole industry is discussing endpoints and about the scores. Of course, the score is something that we have to hold up. And the results that the scores show are also I think very impressive with our trial. Outside of that, you're asking a good question and there's a lot of answers, but no final answer that we can really share here. I think the industry is going to come up with answers because you're bringing up very important points, so the score does not of course cover every improvement, we know that. It's well written in literature. I'm sure you know that as well. We are talking to many people including editors or authors of the ALSFRS-R score and they want to know our data and they're looking at those things, and then maybe they will share some of their thoughts. Very soon we’ll hear, maybe, but it's not for us to really edit the score. We're trying to bring forward our data and share it as we can with the whole community to help everyone understand better how to measure ALS, but these are all very good questions. Even if you are taking our time, quite a lot of time, I'm very happy to answer these very professional questions. Thank you.

Thank you for your time.

Thank you. Your next question is coming from Daniel Walker. Daniel, please ask your question.

Yes good morning. NurOwn has a very broad mechanism of action in terms of its impact on multiple critical biomarkers. It seems like a lot of other therapies and mechanisms of action are much more narrow or end up targeting a subgroup. Maybe can Dr. Kern just comment on the advantages of NurOwn’s mechanism of action relative to other therapies in development, both past and present? I do have a few other questions.

Yes. Hi, Daniel, we'll let Dr. Kern answer your questions. I spoke too much this morning. Dr. Kern.

Yes. Daniel, thanks for that question. I think there's a couple of answers that would help shed some light on the point that you're raising. The first is that there's not a single neuroprotective factor that has been shown to be effective. And in fact, there's good reason for that because there are different targets that the neuroprotective factors reach, and in the preclinical models, there's pretty good evidence that targeting a single neuroprotective factor isn't as good as targeting multiples. They have something called synergy; so they work together. That's kind of the first cut that I would offer. The second is that offering neuroprotection without managing the inflammation in the disease is probably not going to be effective, and there also is some evidence to suggest that combining treatments that address both neuroprotection, in other words allowing the tissue to recover from the disease, while at the same time reducing inflammation are more likely to be effective. We think that there's good scientific rationale for this. And then finally, the treatment technology platform that we're providing is a way of packaging both of those treatments into a single delivery mechanism. So, we think that we have a unique way of providing effective treatments across multiple pathways, and I think as Chaim mentioned earlier, our biomarker data supports that scientific perspective.

Thank you so much. And NurOwn has shown a highly significant burden of proof in terms of ALSFRS biomarkers, UNC13A, and most importantly, patient-reported outcomes. Maybe can Dr. Lindborg just provide some comments about how this compares to other ALS therapies in development both past and present?

Thank you for the question, Daniel. I don't think it's appropriate to comment relative to other therapies, but I will echo the comments that you're providing regarding what is very exciting about our data, which we have in the public domain. As we understand, the participants that were enrolled in the trial and we are accounting for floor effects from the study, we see very clinically meaningful events that are measured through the traditional scale of ALSFSR. We then also, as Dr. Kern just walked through, have seen some incredibly exciting results, with large magnitudes across a diverse set of biomarkers in our biomarker data. And really, as would have been hoped for, based on literature, we're also seeing participants who carry the risk allele UNC13A having a differentiated response to treatment, almost 9x the response with the AC genotype. So, we really see across a collection of measures, which I think is one of the most important factors in really understanding what we believe about a product, and I think it paints a very important picture for what we believe about NurOwn. The last comment I would make is really intermingling these endpoints. So, we have the ability and we published this in our Muscle and Nerve paper to understand, do the biomarkers help explain the clinical response that we see with ALSFSR? And in fact, as we published, they do, with very high predictive nature. So, I think that counter to maybe comparing to other therapies, really what we're very excited about is the consistency of measures and really how everything ultimately fits together. If we saw clinical response that we couldn't explain through biomarkers or vice versa, we would certainly be scratching our heads a little bit more, but what we actually see is a very strong tie that gives a very strong explanation for the effects observed.

Thank you so much. And can Dr. Setboun, based on his past experiences, maybe just provide some insights into how he has seen companies in similar late-stage development think about outside partnerships and collaboration? And then maybe Chaim for you, how is Brainstorm thinking about this particular issue and topic?

Daniel, thank you very much. I spoke too much this morning and I'm very happy you're allowing my colleagues to speak. I'll let David take this question. Thank you.

Thank you for the question. You're right. There are not so many companies that are very active in the cell and gene space, and as you mentioned, exosome technology as well is very interesting and attractive for partners. There are very few at this level of development in Phase 3, with potential to really bring treatment to patients. To your point, these three elements — the cell and gene interest, the exosome technology, and the fact that there is large set data and a unique set of biomarkers — makes our company and our data interesting and attractive to other partners. So, it has always been part of our strategy. We’ve always been in discussion with potential partners, and we take that into account for the future. Thank you.

Thank you so much. And Chaim, I don't know if you could comment about how Brainstorm is thinking about it?

I can only agree with David. I think he hit it.

And just lastly, given the amount of time that has now lapsed and the ever-increasing number of patients among ALS patients and patient advocates, some might question the motivations of Brainstorm and the team. Just curious if anyone on the Brainstorm team or anyone from their family has ever been personally impacted by ALS? And if so, would they be willing to share a little and how that experience has affected their outlook and perspective on ALS and the urgency?

That's a very personal question. More than one of the senior management team do have family members that had ALS. I can tell you that. So, yeah, the urgency is very high. I think that that's what I would say. I don’t see my colleagues wanting to talk about it at this time.

Okay, great. Thank you so much Chaim and thank you to the Brainstorm team. I'll jump back in the queue.

Thank you so much.

Thank you. Ladies and gentlemen, this does conclude today's conference call. You may now disconnect your phone lines and have a wonderful day. Thank you for your participation.