Brainstorm Cell Therapeutics Inc.

Greetings and welcome to the Brainstorm Cell Therapeutics third quarter 2020 conference call. At this time, all participants are in a listen-only mode. As a reminder, this call is recorded, and I would now like to introduce your host for today's conference, Michael Wood from LifeSci Advisors. Mr. Wood, you may begin.

Thank you, everyone, for joining the Brainstorm Cell Therapeutics earnings call. 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 and its potential future business operations and performance, statements regarding the market potential for the treatment of neurodegenerative diseases such as ALS and MS, the sufficiency of the company's existing capital resources for continuing operations in 2020 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 its business planning efforts. Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond brainstorms control, including the risk and uncertainties described from time to time in the company's SEC filings. 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 Liebowitz, President and CEO of BrainStorm, Dr. Ralph Kern, President and Chief Medical Officer, Dr. Stacey Lindberg, Executive VP and Head of Global Clinical Research, and Preetam Shah, Executive VP and CFO. They will be available to answer your questions during the Q&A session. I'd now like to turn the call over to Mr. Liebowitz. Chaim, please go ahead.

Thanks, Michael. Welcome to BrainStorm's third quarter 2020 earnings. call and thank you everyone for joining us. So far, 2020 has been very productive for Brainstorm as we advanced Neuron on several neurological diseases, continued to build out our operating infrastructure capabilities and expanded our senior management team. We're focused on the upcoming top line readout for the Neuron phase three trial in ALS, which we expect to happen by the end of November. We believe that a successful outcome for this trial will add hope to the ALS community and will be a transformative moment for brainstorm, setting us on a path to filing a BLA and supporting our transition to a commercial stage company. The phase three ALS trial is being conducted at six neurology centers of excellence in the United States. I would like to once again thank all the investigators and their staff at the participating sites for their dedication, especially for enabling this trial to complete on time in the middle of an ongoing COVID-19 pandemic. I also want to express my complete gratitude to the trial participants and their loved ones who fully devoted themselves to the challenges of bringing an investigational therapeutic forward. Joining me on the call today are our President and Chief Medical Officer, Dr. Ralph Kern, who will update you on our clinical program, including our pivotal ALS trial, as well as recent news relating to our progressive MS and Alzheimer's disease programs. I've also asked Dr. Stacey Lindberg, Executive Vice President, Head of Global Clinical Research, to join the call today. She'll talk for a few minutes about the design of the Phase III ALS trial, Our CFO, Dr. Priyam Shah, will then provide updates on our financial results before turning it back to me for concluding remarks and will then address your questions in the Q&A session. For that session, I would like to also invite two recent additions to our senior team. In September, we appointed Dr. Tony Wachlowski as Executive Vice President and Global Head of Regulatory Affairs. Tony is a recognized leader in regulatory affairs and brings years of multinational experience. He spent 35 years at Bristol-Myers Squibb, and his experience expands a broad range of regulatory capabilities across multiple therapeutic areas and geographies. He has a track record in guiding successful FDA interactions and establishing and executing successful global regulatory strategies. We also appointed Bill White as Senior Vice President for Market Access and Pricing. Bill is also a very accomplished senior executive with more than 25 years' experience in leading product commercialization and securing market access for innovative new medicines. His experience will help us develop market access, patient service and distribution strategies that will be important to the timely and efficient market entry of Neuron for ALS, assuming its approval by the FDA. These two most recent appointments are the latest in a series of key senior hires we made throughout 2020. The reason I'm mentioning them now is that they are part of a strategic initiative to build a senior team with proven development and global commercial capabilities as Brainstorm makes the transition to a commercial organization. Other appointments this year that were previously announced included Dr. Stacey Lindberg as Executive Vice President Head of Global Clinical Research, and David Setbaum as Executive Vice President and Chief Operating Officer, and Professor Jacob Renkel, a distinguished economist and banker, joined us as the chairman of our board earlier in the year. In addition to our preparations for the upcoming data readout, we've been busy planning and executing another pre-BL activities. Our clear focus is to expedite this process We want to be able to submit a BLA with the FDA as soon as possible after top line data is available. At the same time, our clinical trial activities and data preparedness is growing. We are advancing our CMC activities in preparation for the BLA and subsequent launch. With respect to the BLA planning, understanding the urgency of ALS, we are in full dialogue with the FDA We're actively exploring with them opportunities to expedite the information flow to the FDA with the ultimate goal of enabling the data review process itself. I'll now turn the call to Dr. Ralph Kern for a clinical and RD update. Ralph?

Thank you, Chaim, and welcome, everyone. As Chaim just stated, the immediate priority and focus for everyone here at Brainstorm is the completion and preparation of top-line data readouts in the Phase III ALS trial for Neuron. We completed dosing of all patients at the beginning of July, as we previously announced, and we intend to announce top-line results by the end of November. There are several important clinical trial activities remaining that are being completed at this time to support a high-quality database lock. I'm very happy to announce that these are all proceeding as planned. This double-blind placebo-controlled trial will provide data that hopefully can support registration of neuron for ALS. A lot of very thoughtful planning went into the Phase III clinical trial design, including careful selection of clinical and biomarker outcomes, and the detailed statistical analysis plan that will be used to analyze the data. We've incorporated all that we have learned about the neuron treatments, including lessons learned from our prior Phase II randomized placebo-controlled clinical trial. To help you understand our thinking about the Phase III clinical trial design, and particularly how the primary endpoint is calculated, I'm going to turn the call over to Dr. Stacey Lindborg. Stacey, in addition to her experience in global commercialization and regulatory strategy, has a long and deep experience in clinical trial design and biostatistics. It's really great to have Stacey here today to share our thinking with you. Stacey?

Thank you, Ralph. To begin, I want to emphasize the critical role that trial design plays in determining the success of a product. Throughout my career, I've been involved in the advancement of numerous innovative therapies through the development and the regulatory process leading to approval. While the underlying clinical activity and the safety of a new product will always be the most important factors in determining if it meets the standards for approval, very often it's the design and the execution of the supporting clinical trials that become the overriding factor and one that a company can influence for success. The Phase III Neuron trial was obviously well underway before I arrived at Brainstorm earlier this year. However, I can say that from the beginning, I've been impressed by the very careful planning The attention to detail and execution and the sophisticated analysis that went into the setting up of this trial and ultimately has prepared us for the readout that we anticipate shortly. The phase three trial enrolled approximately 200 patients randomized one-to-one to receive three doses of Neuron or placebo administered over four months. It incorporates an 18 to 20-week run-in period. This allows us to ensure that the trial only involves ALS patients defined as rapid progressors. This is important as we observed a greater benefit in rapid progressors from neuron treatment in our Phase II study. Additionally, this trial's run-in period allows us to quantify the ALS decline for each patient prior to treatment. This is very important for a disease like ALS that has great heterogeneity. It brings precision to our estimates for the treatment effect by accounting for interpatient variability. The primary endpoint of this trial is a responder analysis based on the change in the rate of decline of a patient's ALS functional rating score or the ALS-FRS-R score across 28 weeks. And specifically, the way this works is by comparing the rate of decline for each patient in the pre- versus post-treatment period Each patient's ALS-FRSR data are fit with a model providing an estimate of the disease progression over time in the form of a slope, which is expressed in terms of the rate of ALS-FRSR score, either the decline or improvement, per month. We define a responder as a participant whose ALS-FRSR post-treatment score is at least 1.25 points per month greater than his or her pretreatment slope. Those patients who don't see at least a 1.25 points per month improvement in their ALS-FRS-R slope after treatment are defined as non-responders. The response data are then analyzed using a statistical model with important disease covariates. There are a number of secondary efficacy analyses, including the percentage of trial participants or patients with disease progression that's halted or improved, the ALS-FRS-R change from baseline, the combined analysis of function and survival, slow vital capacity, as well as survival analyses. And another critical element of our trial is the collection of CSF biomarker data at seven different time points across the study in all patients. This data will further our understanding of neurons' biological effect in ALS patients. And safety remains paramount in our trials and will be central to our planned data review. Altogether, we believe that these primary and secondary endpoints will generate the evidence we need to assess neurons' efficacy and safety. And for more details on the trial design and how the primary endpoint is calculated, I gave a presentation at the annual Northeast ALS or NEALS meeting on September 30th, and you can find a poster from this event as well as an audio recording of my presentation on Brainstorm's corporate website. I'll now turn the call back to Ralph to complete the review of our clinical and regulatory activities.

Thank you, Stacey, for that great discussion. I want to touch on a few other issues now. The other news relating to our ALS program during the last quarter was a very important publication in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. In this publication, we describe the results from a series of preclinical experiments that demonstrated the ability of neurons to induce T and B regulatory cells and upregulate a molecule called IL-10. This is very important, as we know that decreased T and B regulatory function appear to play a role in disease progression in ALS, and in other neuroinflammatory diseases that are currently under study. These novel observations greatly extend our understanding of how neuron may modulate an overactive immune system, and coupled with its ability to deliver neurotrophic factors, helps advance our understanding of neurons mechanism of action. We believe that neurons ability to simultaneously target these pathways is an important contributor to the proposed mechanism of action. We hope to extend these observations in the plan phase three biomarker analysis. We also announced on our last quarterly call that our trial of Neuron and Progressive MS was fully enrolled. And we're pleased to report today that we expect all study treatments to be completed by the end of the year. In September, we also presented a scientific poster at the MS virtual 2020 meeting. This scientific presentation was given by Dr. Tanuja Chitnis, who is a professor of neurology at Harvard Medical School and senior neurologist at the Brigham and Women's Hospital in Boston. The study evaluated 48 participants in the CLIMB registry, which is a large-scale, long-term, prospective natural history study of MS. Our colleagues conducted an analysis of patients who matched exactly the inclusion criteria of our ongoing Phase II progressive MS trial and serves as an important natural history comparator. What was unique about this presentation is that Dr. Chitnes demonstrated a correlation between specific brain and spinal cord quantitative MRI measures that we're performing in our clinical trial, and she correlated them with the observed functional improvements in progressive MS patients So we believe that these results are very important as they will help us in the analysis and greatly advance the ability to understand the results from our phase two clinical trial in progressive MS. Also, as discussed in the last quarterly call, we are in the process of initiating an innovative clinical program focused on the development of Neuron as a potential treatment for Alzheimer's disease. As part of this program, we're planning a multinational Phase II clinical trial in Europe to evaluate the safety and preliminary efficacy of neuron treatments in patients with prodromal to mild Alzheimer's disease who are biomarker and clinically selected. This will be conducted at the VU University Medical Center in Amsterdam, PTA Salpêtrière Hospital, and other clinical trial sites in the Netherlands and France. We are currently engaged with the relevant regulatory agencies to finalize the design and conduct of this clinical trial, and we will begin clinical trial enrollment once we receive final regulatory and logistical approvals. Lastly, in July, we announced results from a groundbreaking preclinical study of neuron-derived exosomes as a potential treatment for COVID-19 ARDS that we studied in an LPS ARDS mouse model. We showed, for the first time, that intratracheal administration of exosomes extracted from mesenchymal stem cells using our neuron technology resulted in statistically significant improvements in multiple lung parameters. We're in the process of publishing these findings in a peer-reviewed scientific journal, and we're actively considering next steps. I will now turn the call over to Preetam to discuss the financials. Preetam?

Thank you, Ralph. It is my pleasure now to walk you through our third quarter 2020 financial performance. Research and development expenses net for the three months ended September 30, 2020 were 1.87 million compared to 4.01 million net for the three months ended September 30, 2019. This decrease year over year was primarily due to decrease in expenses related to the phase three and phase two clinical trials an increase in participation of IIA and CIRM under various awarded grants, and a decrease in expenses in connection with patents, travel, and other activities. The decrease in expenses was partially offset by an increase in material costs, payroll, and stock-based compensation expense, and a decrease in proceeds received in connection with the treatment of patients under the hospital exemption regulatory pathway. Excluding participation from IIA and CIRM under the grants and proceeds received from the hospital exemption regulatory pathway, research and development expenses decreased by $1.68 million from $5.66 million in the third quarter of 2019 to $3.98 million in the third quarter of 2020. General and administrative expenses for the three months ended September 30, 2020 were $2.62 million, compared to $1.54 million in the three months ended September 30, 2019. This increase year-over-year was primarily due to an increase in payroll, stock-based compensation, PR costs, consultants, and other costs partially offset by decrease in travel expenses. Net loss for the three months ended September 30, 2020 was $4.49 million, or 14 cents per share, as compared to a net loss of 5.63 million, or 25 cents per share, for the three months ended September 30, 2019. Cash, cash equivalents, including short-term bank deposits, were approximately 28.8 million at September 30, 2020, compared to approximately 16.2 million at June 30, 2020. Our total available funding as of September 30, 2020, which includes cash, cash equivalents, and short-term bank deposits, as well as remaining non-diluted CIRM, IIA, and other grants, amounts to approximately $31.4 million. On March 6, 2020, we put in place a $50 million ATM facility. During the quarter ended September 30, 2020, we raised approximately 13.7 million at an average price of $14.48 per share utilizing this ATM facility. As of September 24, 2020, we had utilized approximately 23 million of the 15 million ATM capacity with approximately 27 million of ATM capacity still available to us under the March 6, 2020 ATM. On September 25th, 2020, we amended and restated the March 6 ATM and increased our available capacity from approximately 27 million to sell up to 45 million of our common stock under this ATM facility. To clarify, the aggregate amount of 45 million under the amended and restated ATM facility put in place on September 25th, 2020 with SVB Laring and Raymond James includes the amount of approximately $27 million unsold pursuant to the March 6, 2020 ATM. As of October 14, 2020, we raised an additional $5.1 million under the September 25, 2020 ATM facility at an average price of $17.21 per share. With this additional capital, Our total available funding as of October 14, 2020, which includes cash, cash equivalents, and short-term bank deposits of approximately $33.1 million, as well as remaining non-dilutive funding from CIRM, IIA, and other grants, amounts to approximately $36 million. For further details on our financials, please refer to our Form 10-Q, Files with the SEC, today. Back to you, Haim.

Thank you, Freedom. Good job. We'll now turn over the call for Q&A. Michael Woods from LifeSci will read the questions we have received. Michael?

Thanks, Cain. So the first question we have relates to intellectual property. And this investor says that the company website and various investor presentations indicate the intellectual property portfolio is robust around the New York platform. and they're interested to get a better understanding of the company's competitive advantages. It appears there are competitors in the ALS stem cell space. Would you please provide more details of what comprises the IP portfolio, the number of patents, geographic areas covered, competitive differentiation of neurons specifically protected by your IP portfolio, and for example, are you the only autologous adult stem cell platform that has IP in this area?

Thank you. So Brainstorm's patent portfolio includes granted patents in the United States and in Europe. In addition, we have numerous granted patents in other jurisdictions, including Canada, Israel, Japan, and Hong Kong. These cover both cell compositions and method of treating diseases. We also have several pending patent applications which cover various aspects of the Neuron technology platform. Next question, please.

Next is a regulatory question. Will Brainstorm request the accelerated approval at the EMA in November 2020 when you have the results of the Phase 3 in hand?

David, can you please answer this question?

Sure. So, first of all, Brainstorm has benefited from the assistance which is associated with the SME program, and we've been holding all often direct status in Europe. So Branson cannot speculate on the results of the phase three study, but he's committing and engaging with EU regulators in order to seek approval in a timely manner.

Thank you. Next question.

And how quickly do you think you can get the BLA filed in the U.S. after the phase three results are announced?

Thanks, Ralph. Thank you. The team is actively preparing for the BLA now, as we mentioned in our opening comments. We do not intend to comment on the precise timing of our application, but I can tell you that we're doing all that we can to be ready, should the Phase III data support such an application.

Thanks.

Next question. So, will Neuron treat only specific ALS genetic subtypes, such as SOD1?

Rob, you go again.

Neuron is being evaluated in ALS patients irrespective of their genetic status. As you know, only 10% of ALS has a clear genetic factor identified, of which about 20% are related to SOV1. Our investigational ALS treatment is targeting a much broader population. Having said that, we are evaluating genetics in the Phase III clinical trial, and we do plan to analyze any potential impact specific genes have on the ALS treatment outcomes.

Thanks. And then if approved, how do you foresee physicians utilizing neuron for ALS treatment? Will it just be administered every eight weeks for the rest of the patient's life, or how will that play out?

Ro? We're working on the assumption that physicians will initiate and continue to administer neuron based on their experience obviously ALS patient preferences, and an assessment of benefit-risk, which is a moving target as the disease progresses that is continually reevaluated. One thing that we are able to say is that a single bone marrow harvest, due to our cryopreservation technology, should be able to support at least three years of therapy.

Thanks, Tom. So there's been an instance with another cell therapy company that recently announced a delay in their BLA filing due to an inability to come to agreement with the FDA on a required potency assay. Do you think a similar type of assay is going to be required for neuron BLA filing, and what is the status of your development of such an assay?

Well, we can't comment on another company's interactions with the FDA, but here's what we do know about our BLA filing. First of all, we're aware of the importance of a potency assay aspect, and we're addressing it internally. We plan to discuss this in more detail with the FDA at the appropriate time. And secondly, Brainstorm's BLA will be based on a well-designed and executed Phase III clinical trial with supportive evidence from phase two.

Thanks. How many ALS patients, in your estimation, could be treated in the first year following approval of Neuron?

Yeah, we're working towards treating a maximum number of patients very quickly after an approval. We're in advanced discussions concerning our commercial production. partners, and we should be able to disclose more about this very soon. The next question, please.

Thanks. And the next question relates to the hospital exemption program that's been ongoing in Israel. Do you have any additional information on the patients that were treated through this program, and will the efficacy data that's been collected from those patients be presented or published at some point?

Stacey?

We're happy to take this. All patients have been enrolled and are being treated per the protocol. At Brainstorm, we're committed to advancing science and we'll share the data as soon as we can. What I can tell you is that our communication priorities will go in the following order. First, regulatory agencies and investigators in ongoing trials. And then second, the scientific and the investor community.

Thanks, Stacey. The next question relates to the upcoming data release. First of all, what gives your team confidence that the data release will actually happen by the end of November? Do you think this is an aggressive or conservative target date? And then assuming the data shows strong efficacy as expected, what's the estimated timelining for submitting the BLA, FDA approval, and commercial launch?

Thank you. That goes to you, too.

Sure. We do remain confident in our commitment to share top-line data from our Phase III neuron trial by the end of November. And as shared earlier in this call, the team has actively been preparing for the FDA and are doing so now. We will not comment on the precise timing of our filing, but we are doing all that we can to be ready should the Phase III data support the applications.

Thanks, Stacy. And then another question relating to the BLA filing. Aside from the efficacy data that will be generated in the Phase 3 trial, what other information or data will be needed to support the application?

Yeah, I would ask Tony to please answer this one. And, Tony, please introduce yourself before you answer the question. Talk to Tony Wachlowski.

Thank you, Chaim. And hello, everyone. I'm Tony Wachlowski. I joined Brainstorm in September from Bristol-Myers Squibb. And I'll just say I'm so excited to be here, joining a great team as the Executive Vice President and Head of Global Regulatory Affairs. So again, very excited to be here and do all I can to help with the therapy. So with regard to when information is needed, a biologics license application or BLA is a request for FDA approval to introduce the product into interstate commerce. And as such, it will include a comprehensive summary of all of our data that can address the biologic product in question. FDA has extensive industry guidance regarding the content that's required for a BLA, and that's what we're following. The BLA application for Neuron will contain product and manufacturing information, along with a summary of nonclinical and clinical studies. Our overarching goal is to provide the FDA with the information that they need to make an informed regulatory decision. And we continue to have very productive engagements with the FDA.

Thanks, Tony. Next question relates to availability of Neuron once it's approved. Once Neuron treatment is available on the market, does it require ALS patients to travel to specific countries in order to have access to stem cell therapy, or will it be available, do you think, in all countries?

Tony, want to take this one, too?

Sure. Thank you, Haim. Well, like all therapies that require prior approval for marketing, availability would be limited to where it is approved for commercialization by that relevant authority. And although U.S. is our first country targeted for regulatory approval, we're not aware of any restriction that might prevent a non-U.S. patient to travel to the United States. We will be working diligently to make the therapy available to patients in other countries.

Thank you. Assuming FDA approval is granted, how do you envision the commercial launch of Neuron rolling out? Will the company manufacture Neuron at its own facilities? Will it be ready for large-scale production using bioreactors? Will the launch take place initially just in the U.S., And when do you plan to treat patients in Europe, Israel, Japan, and other markets?

So many countries. David?

Sure. So, as we said before, we are in advanced discussion concerning our commercial production. And we should be able to disclose more about this very soon. We are engaging and we plan to work with regulators outside of the U.S., to seek approval for Neuron in different geographies.

Thank you. And the next investor wanted to simply welcome Mr. William White to the company and recognizing that his expertise will be very important to the commercial launch of Neuron. But the question is, how far along is brainstorming preparedness for the broad market access and reimbursement for private and public insurers? And are these preparations happening just in the U.S., but also in the other target markets?

As you might guess, I would allow Bill White to answer this question himself as he's getting some warm welcome from an investor. Bill, please introduce yourself first and then answer the question.

Sure. Thank you, Haim. I appreciate that. My name is Bill White. I joined Brainstorm in late September following four years out of Vexist, now Novartis Gene Therapy. where we launched the second gene replacement therapy approved in the United States called Zolgensma for spinal muscular atrophy. So I'm very excited to join Brainstorm and do a similar approach in bringing our novel neuron therapy to market. To answer the question, and thank you for that question, it's a good one, our goal is to partner with each of the commercial and public payers well in advance of the launch of Neuron in order to prepare the market for timely access to our therapy. We have just initiated early engagement with the top payers in the country, including UnitedHealthcare, Aetna, Cigna, and we expect to expand our outreach to the bearers once the Phase 3 readout has been completed. Additionally, we expect to launch in the U.S. first. of course, and then followed by a similar approach in the EU5 and some of the other key global markets.

Thank you. And the next question relates to the progressive MS program. Please provide an update on the Phase II trial for Neuron in progressive multiple sclerosis. Is the trial still on track to finish by the end of 2020? When do you expect to announce top-line data? Are there plans for Phase III? and with strong efficacy and safety from phase two, is there a possibility that the company could apply for accelerated approval from the FDA?

Bob? Thanks. I'm happy to take that. So just as an update, as I mentioned previously, the phase two progressive MS trial is fully enrolled, and we expect that all patients enrolled in the trial will have received all the doses of Neuron by the end of the year. Our plan is to fully analyze the clinical and biomarker data and then decide on next steps. And obviously, it will be data-driven.

Thanks. And then regarding the Alzheimer's disease trial, how is enrollment proceeding in the Phase II trials in Alzheimer's disease in Europe? Many patients have been enrolled at this point. Will the company begin dosing patients by the end of 2020? and is there a possibility for interim data being announced at some point during this study?

Thanks. David?

We're actively engaged with the relevant regulatory authorities in Europe regarding both the design and the conduct of the trial. We intend to start dosing patients as soon as the clinical trial is approved.

Thank you. Thanks. And the next question relates to the expanded access program. There have been rumors of Brainstorm providing expanded access to participants in the phase three trials. Does the company have such plans? Are there external financial resources to help with costs? And might the company provide expanded access to additional ALS patients?

Ralph, you want to take the first shot on this?

Yeah, thank you. Happy to. So we appreciate the sensitivity and urgency and importance of this issue. Just to clarify, we're not yet providing EAP at this time. However, we have been very actively considering expanded access as a potential pathway for ALS patients. As you know, Neuron is not a conventional pharmaceutical product, and there are certain logistical steps that are needed to be completed to make this treatment available. We're very dependent on our partners, and at this point, some of them are working through logistical details needed to support such a program, and they're not quite ready yet to participate. We plan to continue this work, and we will provide a further update when we have more information. So please stay tuned.

Yes, thanks, Ralf. And just to conclude on this matter, there was also a question about financing activities. Brainstorm is more than happy to finance this program if only our partners would be ready. So we did put aside money for this, but again, as Ralph just said, we're dependent. And if the partners will be able to speed up and get ready, the finance will not withhold it. Mike, next question, please.

Yeah, one more question submitted from investors. Investigational ALS treatments have been featured on ABC's Nightline show Do you know when the next episode featuring progress in ALS treatments, including Neuron, may potentially be featured on Nightline?

Yeah, so Nightline has continued to follow and film the ALS community since the last segment. They advised us that they are planning to do a full-length documentary and are waiting for the results of our Phase D trial for completion. I think we're done with the written questions. Melissa, you want to reach out if there are any additional verbal questions? We have time to take three, four more questions.

Yes. If you'd like to ask a question over the phone, please press star 1 on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star 2 if you'd like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star key. One moment, please, while we pull for questions. Thank you. Our first question comes from the line of Jack Mayer, private investor. Please proceed with your question.

Hi, Chaim. Congratulations on the progress. With respect to the hospitalization program in Israel, I believe that the last time you spoke about it, the status was that you had been able to treat the Israeli patients, that you had been unable to treat the foreign patients, and that you had gotten approval in Israel to treat additional patients. Can you just update that part of it?

Yeah, so we did treat all 12 patients, even with COVID. We did not start the additional part of the patients. We are focused for the phase three trial and elsewhere. We have a lot of pressure from patients to give them treatment in the interim, even now, between now and the readout. We'll have to decide if we want to do it. We have the approval to do it, obviously, and we'll see. Thank you. Very good question, Jack. Do you have any other questions?

Not at this time, thanks.

Thank you. Melissa, the next.

Once again, it's star one to ask a question at this time. Our next question comes from the line of Ann Schwartz, private investor. Please proceed with your question.

Yes, is there any movement in Latin America that you guys can talk about?

Yes. What do you mean movement in Latin America, Anne?

Like, yeah, is there any movement for brainstorming around for those folks?

If you're asking, I'm sure you may be part of this. There's a huge campaign from patients of Latin America asking us to get ready to provide treatment in Latin America. And as you can hear, we had some questions before also on Europe. So with European regulatory, we're already in contact. In Latin America, we did file for a few patents, but it's taking them a lot of time to respond. But we do know that there is a big ALS, unfortunately, a big ALS population in Latin America. And once we get an approval, if we will get an approval from the FDA, we will consider that market. It's a very big market. It's a big patient population that really wants to make sure that we know about them. So we do know about them. Absolutely.

Can I ask one more question about the variants, SMA, PLS, all the ALS variants? Will there be a time that you guys will, maybe Walter can talk about that one since he was involved with SMA, but we're just wondering about the variants for ALS, if they will be included.

Yes, many in our company were involved in SMA. One of them is Dr. Ralph Kern. Ralph, you want to address this?

Yeah, I would say that PLS has much closer resemblance to ALS from a disease perspective. So I would say that SMA is a different disease, and we're also following very closely the successes in SMA. We really applaud the opportunity for patients and their families to get treatment. PLS is a much slower disease, more difficult to identify and much lower frequency. than traditional ALS. We have had discussions with experts about it, and we're continuing to think about PLS, which has mostly an upper motor neuron involvement. So it's on our radar screen, but nothing to announce today.

I appreciate it because it's left out a lot. So thank you.

You're welcome. Next question, please.

Thank you. Our next question comes from the line of John Newbins with Raymond James. Please proceed with your question.

Good morning, everybody. My question has to do, is there any update you could provide, particularly to David, on the efforts to find a large commercial partner in any geography? Has the company decided whether to market the product themselves in, I should say ourselves, in the U.S., Europe, and potentially Japan, and one specific question. Would a Japanese study, phase three study, be required to get approval in Japan? Thank you.

Well, it's your client.

Yeah. Hi, John. Hope you're well. Yeah, I think that I'll answer the second part, and then I'll return it back to my colleagues about partnerships. Our feeling in Japan is, similar to other advanced therapies, is that there are accelerated pathways, such as the Sakigake pathway, that potentially could help accelerate efforts in that country. Often, a smaller study in Japanese population is all that's needed if there's a large study scientifically valid placebo-controlled trial in the U.S. So those are things that we are aware of. But in terms of progress with other partners, I'll return it to my colleagues, Chaim and David, who are closer to that.

Exactly. David, do you want to talk about the partnerships? It's a good opportunity, I think.

David? David?

Maybe we lost David. So, yeah, David's working very actively with many parties. He's making sure that all the relevant parties are up to date and well aware of where we are. I'm hesitant what else I can share with you, and it looks like David fell off the line. But when we'll have something to announce, of course we will announce. Melissa, we have time for one more question, please.

Thank you. Our next question comes from the line of David Bouts with Zacks Investment Research. Please proceed with your question.

Hey, good morning, everybody. So I wanted to ask a quick follow-up on potential, the potency assay that may be required by the FDA. Basically, what I'm curious to know is what would you be testing to say that, okay, these cells are good to go Basically, what do you think the FDA is going to specifically look for in that type of assay?

A very good question. Ralph or Tony can take this.

Yeah, I think I'll let Tony start, and then maybe I can add to his answer. Sure.

Yeah, so I would, at the time, I would start with the observation that there is guidance on potency assays in the United States and Europe also, for that matter, and there's lots of recent precedent that we've been studying, and we've had some consultants help us understand the nature of this. It is a challenge. We have the benefit of the measurement of many markers of characterization of the cells, as well as the coming biomarkers in the CSF themselves. So I think, you know, we have some framework in place, and as was mentioned in the call already, we will be discussing it with the FDA at the appropriate time. It is an important consideration and emerging as more and more important recently. So we're well aware of it and have some, I think, reasonable approaches to it. Ralph?

Yeah, what I can – thanks, Tony. What I can add is that we've been very thoughtful and deliberate about linking the – biological parameters of our cellular product with preclinical studies, and then showing consistent reflection of this biological activity in biomarkers, which we have spent a lot of effort linking to our clinical results. And we think that those efforts will bear fruit, and that obviously very thoughtful combination of biological properties of the cells, preclinical data, and then biomarker data will be used to address those questions in the future.

Thank you very, very much. I think that would conclude. Melissa, thank you very, very much for running this verbal session. And I want to thank everyone that asked questions previously that we were able to sort them out and make sure that we answer the variety of the issues that investors may have. That includes many analysts that we didn't hear them verbally today, and they did send their questions earlier. So I thank them for their time and hope that we answer the questions to their satisfaction. And I want to thank everyone for being with us today, and we look forward to probably the next time we speak to you after top-line results. So thank you very much. Very exciting times coming.

Thank you. This concludes today's conference. You may disconnect your lines at this time. Thank you for your participation.