Q2 2023 Brainstorm Cell Therapeutics Inc Earnings Call
Participants
Chaim Lebovits; President & CEO; Brainstorm Cell Therapeutics Inc.
Stacy Lindborg; Co-CEO; Brainstorm Cell Therapeutics Inc.
Alla Patlis; Interim CFO; Brainstorm Cell Therapeutics Inc.
Kirk Taylor; EVP & Chief Medical Officer; Brainstorm Cell Therapeutics Inc.
Michael Wood; IR; LifeSci Advisors, LCC
Jason McCarthy; Analyst; Maxim Group LLC
Presentation
Operator
Greetings, and welcome to the Brainstorm Cell Therapeutics second-quarter 2023 earnings call. (Operator Instructions) As a reminder, this call is being recorded.
And I would now like to introduce your host for today's call, Mr. Michael Wood of LifeSci Advisors. Mr. Wood, you may begin.
Michael Wood
Good morning, and thank you for joining us this morning. Earlier today, Brainstorm issued a press release with its financial results for the second quarter of 2023, including a corporate update.
Before passing it off the company for prepared remarks, I'd like to remind listeners that this conference call will contain numerous statements, descriptions, forecasts, and projections regarding Brainstorm Cell Therapeutics and its potential future business operations and performance statements regarding the market potential for the treatment of neurodegenerative diseases such as ALS, the sufficiency of the company's existing capital resources for continued operations in 2023 and beyond, the safety and clinical effectiveness of NurOwn technology platform, clinical trials of NurOwn and related clinical development programs, and the company's ability to develop strategic collaborations and partnerships to support its business planning efforts.
Forward-looking statements are subject to numerous risks and uncertainties, many of which are beyond Brainstorm's control, including the risks and uncertainties described from time to time in the company's SEC filings. The company's results may differ materially from those projected on today's call, and the company undertakes no obligation to publicly update any forward-looking statements.
Joining us on the call this morning will be Chaim Lebovits, President and CEO of Brainstorm; Dr. Stacy Lindborg, Co-Chief Executive Officer; and Alla Patlis, Interim Chief Financial Officer. In addition, Brainstorm's Executive VP and Chief Medical Officer, Dr. Kirk Taylor, is also on the call and will be available to answer questions during the Q&A session. So I'd like to turn the call now to Mr. Lebovits. Please go ahead.
Chaim Lebovits
Thank you, Michael. Thank you all who have joined us to discuss our Q2 2023 financial results and recent progress. Our main priorities right now are to prepare for the forthcoming advisory committee for NurOwn, our investigational therapy for the treatment of ALS, and to make sure the company is prepared to make NurOwn available to patients.
As announced in June, the FDA will convene a meeting of the cellular tissue and gene therapies advisory committee to review our BLA for NurOwn on September 27. In addition, the BLA has been assigned a PDUFA action date of December 8, 2023.
NurOwn's regulatory process, as we have mentioned previously, will be the same as it is for any other investigational therapy that is subject of a filed BLA. The upcoming outcome will be guided by an agenda that includes detailed presentations, a brainstorm, and the FDA, which will allow our team and the agency to discuss the clinical evidence supporting NurOwn's safety and efficacy as an ALS therapy.
Other key stakeholders, including independent medical experts, statisticians, and patient advocates, will then have the opportunity to provide their own unique perspective on NurOwn's clinical dataset as well as the unmet needs of people living with ALS. Members of the AdCom will then have the opportunity to vote on the response to the question set forth by the FDA.
The agency will take the results of this vote or these votes, as well as the preceding discussions at the meeting under advisement when coming to a decision on the BLA, which will be made by the December 8 PDUFA date.
Given the strength of the clinical evidence we have generated at NurOwn, we remain confident in our ability to achieve a successful outcome from the AdCom and are preparing for success to ensure we can make NurOwn available to patients as quickly as possible if approved later this year.
Our team is focused on being fully prepared in advance of the upcoming AdCom. These preparations began months ago and continue today as we work with our expert consultants to ensure we can present and respond to all questions that the FDA and AdCom members might raise.
We are grateful to the FDA for its cooperation throughout the review process and recognize that the agency is taking our application very seriously.
I'll now turn the call over to my colleague, Dr. Stacy Lindborg, for additional comments, Stacy?
Stacy Lindborg
Thank you, Chaim. We are looking forward to the AdCom in September and cannot overstate the importance of this meeting in NurOwn's regulatory path given the scientific and policy issues that need to be understood.
The FDA has rightly shown a willingness to apply regulatory flexibility when evaluating investigational ALS therapies over the last year, and we look forward to having NurOwn's full data set discussed in the context of the need for new ALS therapies in the public forum offered by an AdCom.
As we move towards the FDA's decision, we continue to have full confidence in our data and believe that a comprehensive analysis of our results strongly support NurOwn's clinical efficacy and safety. We remain committed to the scientific and regulatory process, which includes continuing research to confirm the results of the NurOwn clinical program.
We also remain committed to ongoing learning about the safety and the clinical effectiveness of NurOwn. For this reason, we have assembled a steering committee to gather input on goals and the core design elements of a confirmatory trial. We look forward to providing an update prior to the AdCom.
We continue to be active with the ALS community at scientific conferences. We delivered an important presentation featuring new biomarker data and at the recent 2023 Gordon Research Conference for ALS and related motor neuron diseases.
As we've described previously, during the NurOwn Phase 3 trial, we collected CSF fluid from all trial participants and examined biomarkers spanning three pathways important ALS pathology, neuroinflammation, neurodegeneration, and neuroprotection.
The study is the most robust CSF biomarker study conducted in people living with ALS. And the new data presented at the Gordon Conference features analysis of a biomarker known as neurofilament light in addition to more broadly providing evidence of the importance of using baseline disease characteristics in the analysis of biomarker data, which I'll provide the rationale for.
I believe we all appreciate how heterogeneous ALS is as a disease. Because of this, it is common practice to include ALS disease characteristics of [covariates] in the analysis of clinical data, which we also did our Phase 3 trial. The goal is to include information that could influence an individual's prognosis in addition to therapy so that you're drawing appropriate conclusions relative to the treatment effects in the trials.
When we decided to include these as covariates in our biomarker analyses, we drew on the ALS literature and the final guidance released in 2023 by the FDA on adjusting for covariates in randomized clinical trials for drugs and biologic products.
What's the goal of exploring the importance of ALS disease characteristics in our biomarker data? From Phase 3, we employed five disease covariates that were prespecified and used in a primary efficacy model in the trial. These covariates, which include baseline ALSFRS-R score, the baseline rate of decline, use of riluzole, fight of ALS disease onset, and time since symptom onset to treatments are well supported in the literature.
As highlighted in our poster presentation, all five disease covariates, in addition to biomarker data, had a significant impact on clinical outcomes. Therefore, the analysis of biomarker data can reflect the treatment effect more accurately when accounting for the baseline heterogeneity of participants. Including these covariates in the analysis across all biomarkers simply adds precision to the results.
In the poster, we highlighted the longitudinal trajectory of four biomarkers, including neurofilament light as examples of the improvements observed in biomarkers following treatment with NurOwn compared to placebo across all participants in the trial.
Neurofilament light has been getting a lot of attention in the scientific community and with drug developers as evidenced by the exponential growth in publications in recent years. In NurOwn-treated participants, we observed an 11% decrease from baseline to week 20 in neurofilament light, with the change of around 1% with placebo and a significant treatment difference of a P less than 0.05.
The other biomarkers highlighted in the poster also showed significant treatment differences with the decreases in pro-inflammatory biomarker MCP-1 and large increases in neuroprotective biomarkers, VEGF and galectin-1.
I also presented two other results in the poster. First, neurofilament light baseline levels appear to be prognostic of ALS disease progression. This means that participants with higher baseline neurofilament light values had greater decline from baseline to week 28 as measured by the ALSFRS-R. This finding confirms the results seen in other ALS trials, which is promising for the field.
The last analysis we presented was motivated by the literature and has been used in the review and approval products by the FDA in diseases such as pulmonary arterial hypertension and triple-class refractory multiple myeloma in addition to ALS through the review of tofersen.
And while NurOwn's mechanism of action is very different from tofersen, as we have a broad multi-modal mechanism faction simultaneously targeting biological deficiencies associated with ALS, we felt these analyses were important to conduct and understand the insights derived into our data based on the relationship between neurofilament light and clinical outcome.
The analysis used an approach called causal inference, sometimes referred to as outcomes regression, and it allows us to explore the relationship between the change in neurofilament light and the change in ALSFRS-R due to NurOwn alone by adjusting the observed outcome with the change we would have expected due to the natural progression of the disease.
The analysis presented confirm that NurOwn-driven reductions in neurofilament light are associated with less decline in the ALSFRS-R from baseline to week 28. Taken together, data from the literature and the NurOwn Phase 3 trials, support the hypothesis that treatment-driven reduction in neurofilament light are reasonably likely to be associated with clinical benefit in ALS.
We believe these results are timely given the regulatory precedent that was set in ALS this year. In April, the FDA granted accelerated approval to Biogen and Ionis's drug, tofersen, to treat a genetic form of ALS known as SOD1 ALS, which represents approximately 2% of ALS patients.
The approval decision was based in part on tofersen's ability to lower plasma levels of neurofilament light, establishing the view that reductions in neurofilament light are reasonably likely to be associated with clinical benefit in ALS.
Specifically, when the AdCom that reviewed tofersen was asked whether the available evidence supported a reduction in plasma neurofilament concentration as reasonably likely to predict clinical benefit, the committee voted unanimously nine to zero. This is the first time an ALS drug was approved by the FDA based on biomarker data.
I'll now turn the call back to Chaim for some additional comments.
Chaim Lebovits
Thank you, Stacy. Our second main priority, as I mentioned earlier, is to ensure commercial preparedness and execute on the various activities that we need to complete in order to make NurOwn available to patients if approved.
These include activities across manufacturing, commercial and medical affairs to engage with the physicians who treat ALS, and also early discussions with payers. In terms of how NurOwn would actually be delivered to ALS patients, the first step is to collect bone marrow from the patient, and this is then shipped to our manufacturing facility where the [MSEs] would undergo a series of steps to create the therapeutic product.
We're currently in discussions to be able to sign contracts with centers of excellence across the United States so that they will be set up to collect bone marrow from patients, and we can initiate the manufacturing process for each person's personalized treatment. We'll begin with 8 to 10 centers and then move to broader engagement with more centers.
As we have outlined before, we're in the process of a targeted capability build to expand our team in preparation for anticipated growth. We want to be able to move quickly. So if we're successful in achieving approval for NurOwn, we will have the infrastructure in place, and the wait for patients and families to gain access will be as short as possible.
We have made a number of hires and management changes so far in 2023. At the beginning of the year, Dr. Stacy Lindborg was promoted to co-CIO. Then early in the second quarter, we appointed Dr. Kirk Taylor as Executive Vice President and Chief Medical Officer. Kirk will lead the global medical affairs function and launch activities, including planned product launches, post-approval commercialization efforts, and deepening relationships with the medical community.
More recently, in July, we appointed Dr. Bob Dagher as Executive Vice President and Chief Development Officer. Bob will be responsible for the portfolio strategy and advancement of clinical development plans towards regulatory approval, including the expansion of NurOwn into new diseases and the translation of pre-clinical research in the first-in-human trials.
He brings approximately 20 years of industry expertise in the development and approval of treatments for challenging neurological and rare diseases. He began his career at GSK and has served in leadership positions of science and medicine at companies such as Sanofi Genzyme and LabCorp Covance.
Finally, we also made an important addition to our Board with the appointment of Nir Naor a Board member and the Chairman of the audit committee and member of the governance, nomination, and compensation Committee.
Nir brings over 20 years of global work experience as a CFO and Senior Finance Leader. He has a broad background that includes large pharma and biotech and has overseen organizations with up to $2.5 billion in sales and $1 billion in annual spend.
We are excited to expand our team with these and other talented individuals, and I know they share our vision and excitement around NurOwn prospect. This expanded team is fully focused to prepare Brainstorm for the exciting future ahead.
I'll now turn the call over to Alla to discuss our financials. Alla?
Alla Patlis
Thank you, Chaim. Cash, cash equivalents, and short-term bank deposits were approximately $0.75 million as at the end of June 2023, compared to approximately $3 million as of the end of December 2022. In July 2023, subsequent to the end of the quarter, the company raised net proceeds of approximately $7 million in the registered direct offering.
Research and development expenses for the three months ended June 30, 2023, and 2022 were approximately $2.8 million and $5.1 million, respectively. General and administrative expenses for the three months ended June 30, 2023, and 2022 were approximately $2.7 million and $2.5 million, respectively.
Net loss for the three months ended June 30, 2023, was approximately $5.3 million or $0.13 per share as compared to net loss of approximately $7 million or $0.19 per share for the three months ended June 30, 2022.
I'll turn it back to Chaim. Chaim?
Question and Answer Session
Chaim Lebovits
Thank you, Alla. I'll ask I'll ask Michael Wood from LifeSci to read the questions we have received from investors. Michael?
Michael Wood
Thanks, Chaim. The first question, is the submitted BLA for NurOwn definitely seeking full approval or accelerated approval?
Chaim Lebovits
The BLA filed is seeking full approval. Thank you.
Michael Wood
Thanks. And does the BLA includes data collected from patients that have been treated under the expanded access program and from the [Israeli HE] pathway?
Chaim Lebovits
Yes, that is correct. Data from both programs were included in the BLA.
Michael Wood
Do you still intend to publish your biomarker manuscript?
Chaim Lebovits
Absolutely. The biomarker data is a compelling part of our evidence, which provides strong support of the clinical data. And we can confirm the paper is under review at a highly regarded journal. The senior authors are Dr. Bob Brown and Dr. Merit Cudkowicz, and the paper includes other leading researchers and scientists.
Michael Wood
Thanks. The next question relates to the Gordon Conference. Why did you only recently decided to look at baseline characteristics of patients and make this presentation at Gordon? And did the results change substantially once you've counted the baseline characteristics?
And then having listened to Stacy's discussion this morning, I have an additional question I'm going to add, and that is we're intrigued by the word precision that Stacy used in her prepared remarks. Can you please explain what this means?
Chaim Lebovits
Stacy, I'll have you answer this question.
Stacy Lindborg
Okay. So to the first question, why adjust for baseline characteristics, now basically, this is an example of emerging science.
If you look at the public documents from tofersen to AdCom, you'll see that the FDA did a lot of work exploring the importance of disease covariates, including all of the baseline disease covariates that we prespecified in our efficacy analyses that I actually spoke about in my prepared remarks. And they did this as they were analyzing tofersen data.
Also, as I referenced earlier, the FDA issued a final guidance on adjusting for covariates in randomized clinical trials, which was very timely and provided important perspective. In fact, when we reviewed the guidance, which focused on prognostic baseline covariates, what stood out to us was that sponsors should prospectively specify covariate-adjusted analyses.
Therefore, we thought it logical to use the covariates specified in our Phase 3 statistical analysis plan. So the guideline also noted that covariate adjustment was acceptable even if baseline covariates are strongly associated with each other. So this guidance, combined with the importance the FDA placed on these covariates in tofersen's review, led us to explore the importance of these covariates in our data.
As a side note, in the analysis that we ran, we also used the model that FDA used with tofersen data. And in the cases where this was done, the [significance that has helped] across our data is quite robust.
Michael, if I understood your question at the end, you wanted to know what I meant by the word precision. Let me first recall this statement I made earlier. So I referenced that in the analysis of our biomarker data, we can reflect the treatment effect more accurately when we account for baseline heterogeneity of patients.
So in other words, by adding these characteristics, which really help identify ways that the disease is variable across patients, it brings precision to the estimate of treatment.
So here, when I use this word, precision, what I mean is that the model with disease covariates included in it and these covariates' influence can influence the rate of disease progression. This will have a better ability to capture variability observed in the data. And the significance of these covariates tells us that this is the right way to analyze the data. Otherwise, you're ignoring important information in the analysis.
The last part of the question was how the results compared across the model that had baseline covariates versus one, the model that did not. The biomarker results from the model -- from both models actually are very similar with no conclusions changing substantially for any biomarker.
There were two biomarkers that were already trending towards a significant treatment effect. One was neurofilament light; the other was neuroprotective biomarker HGF. And accounting for these disease characteristics resulted in the P value dropping just below the conventional level of 0.5.
But even for these two biomarkers, the overall pattern in the treatment effect as well as the percent change from baseline in both arms is very similar to the results of the model that didn't adjust for the terms. The estimate of the treatment effect just had more precision because it could take into account important information that also includes clinical outcomes in addition to treatment.
Michael Wood
Thanks, Stacy. Next question, do you intend to proceed with a confirmatory clinical trial?
Chaim Lebovits
Yes, we have definite plans to proceed with a confirmatory trial. It's for months now that we have been meeting with a steering committee of leading clinicians and statisticians. We intend to share more after we get input from the FDA.
In this regard, I would like to really share that we're very thankful for the California Institute for Regenerative Medicine firm that reached out to us, and they asked us that they would be interested that we submit an application for such a trial. Thank you.
Michael Wood
Is there a reason that you're granted a different AdCom from the AdCom that oversaw the Amylyx and Biogen drugs? Do you think this is a good or bad sign that you are being reviewed at a different AdCom?
Chaim Lebovits
Yes. So the designation of the advisory committee that advises FDA for an application under review is determined by the specific center of the FDA center that the application is filed with. The RELYVRIO and QALSODY were both submitted as new drug applications NDA to CDER or the Center for Drug Evaluation and Research, while NurOwn, being a biological product -- a stem cell product, was submitted as a biologic license application to CBER or the Center for Biologics Evaluation and Research.
Each center has multiple AdComs For example, CBER has four. NurOwn will be reviewed by CBER's cellular tissue and gene therapies advisory committee.
If anyone is interested to look in deeper to this, there's a document called the inter-center agreement between the CDER and the CBER assigned to each center's jurisdiction for regulation of drug and biological products and combination of drugs and biological products. And it describes those product characteristics or medical indications that would require a collaborative review of effort by the two centers.
Michael Wood
Thanks. The next question relates to clinical manufacturing controls or CMC. Have you been able to submit amendments to address the CMC items identified by the FDA in their initial RTF letter? And if so or if not, do you anticipate any impact on the PDUFA date?
Chaim Lebovits
So yes, definitely, as we have shared, and I'll confirm this again, the FDA has allowed us to submit amendments, and we have submitted those amendments. Sure. Thank you.
Michael Wood
And have you been having any conversations directly with the FDA while the BLA has been under review?
Chaim Lebovits
Yes. As is typical for a BLA under review, we have regularly occurring interactions with the FDA. We received quite a few requests for information, which we have responded to in a timely fashion. We also were able to share presentations in addition to other interactions.
Michael Wood
You reported that around 25% of patients in your Phase 3 study had a baseline value below 25. And in these patients, further declines could not be measured because the items reached zero. You referred to this as the floor effect. Can you please expand on the floor effect, and do you have any biomarker data for these participants?
Chaim Lebovits
Thank you. Stacy, please?
Stacy Lindborg
Sure. I want to start by just reflecting on the fact that the ALSFRS-R remains the best outcome measure that we have today. But like any bounded scale, it has limitations. And in the group of participants asked about in this question who were in the bottom half of the scale, there was a high rate of participants with ALSFRS-R items, specifically in the fine and gross motor subdomain that started at zero with approximately 40% on starting at zero across all six items.
The rate of zero values, especially on the fine and gross motor, is problematic to measuring a treatment effect in a trial because it's expected that the fine and gross motor domains account for about 70% of decline observed in trials. This therefore confounds the ability to show a treatment effect as the ALSFRS-R can't measure further decline once items reached zero.
So the question about biomarker data in these participants, we have looked at biomarker data in these participants. And in fact, at the Gordon Conference, we reported first that we observed significant improvements on ALS biomarkers with NurOwn versus placebo in all trial participants. And this was important across the three important pathways that I referenced in my opening remarks, neuroinflammation, neurodegeneration, and neuroprotection.
We in fact see very similar treatment patterns in participants with baseline scores 25 and below on the ALSFRS-R. And what this suggests is that NurOwn is biologically active in the overall population that was studied in the trial, which includes participants with advanced ALS disease where the scale, the ALSFRS-R scale, demonstrated measurement challenges.
Michael Wood
Thanks, Stacy. Next question relates to your clinical pipeline. Brainstorm has said that it's working on the use of its product for other indications. Investors have been hearing about this now for years. Please provide some specificity with respect to what working on it means.
Chaim Lebovits
Well, thank you. I'll ask Dr. Kirk Taylor to take this question, please.
Kirk Taylor
Great, Chaim, thank you. Sure. Well, we completed a Phase 2 study evaluating NurOwn as a treatment for progressive MS and announced positive results in 2021. We have worked with neurologists and statisticians with deep expertise in MS to design the next trial and have a solid protocol concept prepared that builds on the completed Phase 2a study.
We've also prepared a protocol concept designed to study the impact of NurOwn in Alzheimer's disease in the context of the unmet need that remains with the approval of treatments that remove amyloid plaque, consulting with leading experts, and that's NurOwn in Alzheimer's disease.
Designed to the question addressed here is, though, in patients for amyloid plaque has been removed, could NurOwn increase cognitive function above baseline levels? That's the question. NurOwn's mechanism of action supports our view that it may have broad applications in neurodegenerative disease. However --
Chaim Lebovits
Kirk?
Kirk Taylor
I'm sorry -- however, like many of our peers in biotech industry, we need to prioritize resources and focus those programs that could potentially benefit patients in the near term and create value for our stakeholders. At this point, we are focused primarily on the ALS program and getting approval in that indication. We intend to move forward with other programs as resources allow. Thank you.
Michael Wood
Thank you. I have one final question. Did Brainstorm finalize on the follow-on offering with Maxim for $7.5 million?
Chaim Lebovits
Yes, in addition to the PR on this matter, as is common practice, we did publish an 8-K. Just to provide some more color on this. We will continue to explore the best ways for finance. We'll have multiple options to finance going forward, and the company will be quite opportunistic to utilize the most favorable opportunities that comes our way at the time of need. Michael?
Michael Wood
And that's the final question.
Chaim Lebovits
Thank you so much. Jenny, would you open for any additional questions?
Operator
Yes, no problem. (Operator Instructions) Jason McCarthy, Maxim Group.
Jason McCarthy
Hi, all. Thanks for taking the question. Really looking forward to that AdCom in September. And about that AdCom, do you expect -- based on your covariate analysis of the five covariates and what you've shown around NFL, particularly in that presentation in July, do you expect the same questions that Biogen had gotten where there was the vote on NFL, nine to zero, but there was differences in the voting, three yes, five no, on the full approval, I guess, requiring a confirmatory study. Do you expect a similar question?
Chaim Lebovits
Thank you very much. Stacy?
Stacy Lindborg
Hi, Jason. It's great to hear from you. My thinking about that is this is a different AdCom, a different FDA center, a different application, very different mechanism of action. I think what each of the drug programs was presenting is their evidence also is quite different. So we both bring interesting insights into neurofilament light and the association with the ability to actually mention and show that reductions in neurofilament light are results in an association with the improved clinical outcomes.
It is a strength, but I've learned over the course of my career to not assume anything with regard to the regulators. We'll actually get the questions out there, close to the time of the AdCom. And I think the questions that we're asked were relevant, but we'll know for certain what our questions are right before the AdCom.
Jason McCarthy
During that AdCom, as part of the presentation, will it be made more of a point on the safety aspects of NurOwn, given that it is an autologous cell therapy, it's not genetically manipulated and that it is a far safer approach apparently than other drugs for ALS, including the Biogen drug/
Chaim Lebovits
Stacy?
Stacy Lindborg
Yes, so we will present the full set of data, efficacy and safety. And Jason, we share your confidence in the safety of NurOwn, not only in the data we've generated but as a result of the way that our product is made. And we will provide a compelling overview of all data at our presentation.
Jason McCarthy
And just lastly, a brief question on the potential commercialization plan. I know it's early, but from a pricing perspective, tofersen, priced at around $14,000 or so just north of that per treatment, but it's about $200,000 all called for a year, is that a similar pricing strategy that you could expect for a cell therapy?
And just from a launch perspective, Chaim, you had mentioned getting possibly 8 to 10 centers initially for bone marrow collection. How large of a sales force would you need to complete your initial commercialization plans?
Chaim Lebovits
Yes. So thank you very much. Many of these questions is premature for us to answer for regulatory reasons, as you know. But I like the assumptions you're laying out. And I think what will be very important is to find the centers of excellence doing not only the bone marrow aspirations but the intrathecal injections, which some of these, like [Sarepta] treatments and [Topican] treatments, really helps out.
And more and more centers have that expertise. And that's what we'll be focusing and probably will start with the centers of the trial, which already have a lot of experience with our product. But we're also talking to many other geographical centers and make sure that patients from other geographies have a center close nearby to where they are.
Just a comment to a previous question you asked, and Stacy answered a little bit, is of course, we believe that our clinical data set, even though the primary endpoint did not hit statistical significance, we think that the body of evidence, moving forward, we are able to show statistically significant results once we're able to show and dive into -- more deeper into the data set, which you spent a lot of time and I don't want to repeat it for you.
Therefore, I think that the question will be more focused on clinical, why the biomarker data gives strong support to what we're seeing in the clinical as it covers even for the part of the score where we think it's not sensitive in the more advanced patients. But when you are able to eliminate the advanced patients, we see both in the primary and secondary endpoints statistical significant results.
Of course, we're not going to lay it out here. That's what we're going to do at the AdCom. But thank you very much for those questions, Jason.
Michael Wood
Chaim and Stacy, thank you.
Chaim Lebovits
Sure.
Operator
Thank you very much. (Operator Instructions) Okay, we don't appear to have any further questions in the queue. I can hand back over for closing comments.
Chaim Lebovits
Thank you. I really appreciate that. It looks like Stacy did a wonderful job laying out the plan, and there's no additional questions. So let's -- we had a long call this morning, so let's give back the time to everyone listening. I want to thank you again for listening in in August -- middle of August.
To have so many investors listening in, it shows the importance to our investors of our plan forward, and we really thank you for listening in today. Thank you very much. Jenny, back to you.
Operator
Thank you, Chaim. This does conclude today's conference call. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your participation.
