Ladies and gentlemen, thank you for standing by. Welcome to Biohaven Reports Top-Line Pivotal Trial Results for Troriluzole SCA. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star one one on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star one one again. Please be advised that today's conference is being recorded. I would now like to turn the conference over to Matt Buten, Chief Financial Officer. Please go ahead, sir.
Good morning. My name is Matt Buten, and I'm Biohaven's Chief Financial Officer, and I wanted to thank you for joining us this morning. Before we get into the presentation, I wanted to inform the participants on this call that we will be making forward-looking statements, and to please review this slide for the details of those forward-looking statements. We have a lot planned for this call, and as you can see, we'll provide you with some background on SCA, our pivotal study design, the positive pivotal study results, KOL perception of the successful pivotal data and what it means for patients, and then wrap up with a commercial overview. And then we'll open up the call to questions. I'll now turn the call over to our CEO and Chairman, Vlad Coric, who will be making opening remarks.
Thanks, Matt, and thank you to all the investors and analysts joining our conference call this morning. This is an exciting day for the spinocerebellar ataxia community and Biohaven. As reported in our press release this morning, we achieved positive top-line results in our three-year study of troriluzole. This study protocol stemmed from discussions with the FDA regarding the troriluzole development program. The data that Dr. Beiner will present shortly shows clear delay in disease progression in troriluzole-treated subjects, and in fact, troriluzole slowed the disease by 50% on the study's primary outcome measure. This represents a one point five-year treatment gain over the course of the three-year study. Troriluzole demonstrated efficacy in nine pre-specified outcome measures and was also safe and well-tolerated across the development program.
The results presented today are extremely clinically meaningful to patients and are consistent with the other benefits of troriluzole that we have shown, including a reduction in risk for falls in SCA patients. While today represents an important milestone in our journey to advance a new therapy for SCA, it is a journey that continues with urgency. Based upon these top-line results, we plan to submit a new drug application to the FDA for troriluzole in the treatment of all SCA genotypes by the end of the year. We expect there will be an accelerated review of this filing based on Orphan Drug and Fast Track designations previously granted by the FDA. We stand ready to support patients with SCA, and our commercial team is already preparing for success. We are grateful to so many collaborators for the outcome that was reached with troriluzole and SCA today.
We are indebted to the entire SCA community, the clinicians who conducted our studies, the National Ataxia Foundation and other advocacy groups, the patients with SCA who participated in our trial, and the regulatory agencies, including the FDA, that have engaged with us on this program. I would also like to thank and acknowledge Dr. Jeremy Schmahmann from MGH, who is here today to provide his expert clinical interpretation of our data and comments on the clinical implications of the study results. You will be hearing from Dr. Schmahmann shortly. Without any further delay, let me turn it over to Doctors Qureshi and Beiner to walk us through the study design and results. Dr. Qureshi?
Thank you, Vlad. Before we review the study results, I'd like to provide a brief overview of SCA. SCA refers to a group of rare, dominantly inherited neurodegenerative disorders that are all characterized by cerebellar degeneration. Approximately 15,000 people in the U.S. are affected, and 24,000 in Europe and the U.K. There are currently no approved treatments for SCA, so this represents not only a high unmet need, but a large commercial market. Before Dr. Beiner presents, I also want to make sure that everyone is level set on the primary outcome measure. Of critical importance, we work with the FDA to develop the f-SARA, which is an approvable endpoint for SCA. The f-SARA is a neurologist-assessed scale. It measures SCA disease progression, focusing on functional aspects of the disease: gait, stance, sitting, and speech.
Scores for individual items range from zero to four, and the total score is the sum of the four, with a maximum score of sixteen. Importantly, we validated the f-SARA according to FDA guidance. Based on this, any change on the f-SARA is considered clinically meaningful, and we expect about a half point of worsening per year. Now, let me tell you why we are so excited about troriluzole. Glutamate dysregulation is one of the hallmarks of SCA, and mechanistically, the synaptic glutamate modulating activity of troriluzole corrects the widely documented glutamate dysregulation underlying Purkinje cell dysfunction and neurodegeneration in SCA. So troriluzole was designed to restore glutamate homeostasis in SCA. Recognizing the transformative potential of troriluzole in SCA, the FDA has granted us Orphan Drug designation and Fast Track, and EMA has granted orphan.
Of note, we also have very strong IP protection, with NCE composition of matter patent expiration anticipated in 2041 with extensions. I will now turn it over to Dr. Beiner to present our positive top-line results.
Thank you, Irfan, and good morning, everyone. I am thrilled to present top-line results from our trial, which show clinically meaningful delays in disease progression in troriluzole-treated SCA patients. This is the very first therapy to demonstrate a treatment benefit in SCA. At Biohaven, our motto is Days Matter. In progressive neurodegenerative disorders like SCA, time is of the essence. For these patients, a treatment that can slow the relentless loss of function that comes with SCA, giving more time to be able to care for their children, work, and live their lives independently, is invaluable. Our new study protocol utilized phase three data and an external control of matched untreated SCA patients from the two independent natural history studies, one in the U.S., called the CRC-SCA, and one in Europe, called EuroSCA.
All endpoints were pre-specified, and both the study protocol and statistical analysis plan were submitted to and reviewed by the FDA prior to database lock and analysis of our data. The primary objective of the study was to examine the effects of troriluzole after three years of treatment by comparing data on the f-SARA from patients treated with troriluzole in Study 206 to untreated patients from the U.S. Natural History Study. Secondary endpoints included f-SARA change from baseline at one and two years versus matched patients from the CRC-SCA external control, and also analysis of change from baseline in f-SARA scores at years one, two, and three versus matched patients from the EuroSCA study, and also both natural history studies pooled.
Propensity score matching, which is a rigorous statistical method used to replicate the equipoise achieved by randomization and to reduce bias, was used to ensure that untreated patients from the natural history control arm were rigorously matched to treated patients from Study 206 on baseline characteristics. This slide shows the demographic and baseline characteristics prior to implementing propensity score matching. Patients were matched based on each of these baseline covariates: age, gender, age at symptom onset, genotype, including trinucleotide repeat expansion, and baseline severity as measured by the f-SARA. This graph shows the study's primary outcome measure at years one, two, and three. Troriluzole-treated patients demonstrated statistically significant and sustained benefits at all study time points on the f-SARA compared to the CRC-SCA external control.
These sustained treatment benefits translate into a 50% slower rate of decline compared to untreated patients, representing a 1.5-year delay in disease progression over the three-year study period. Analysis of treated patients compared to untreated patients from a completely separate, independent natural history study, EuroSCA, were consistent with the primary analysis and also showed sustained treatment benefits at years one, two, and three. The addition of EuroSCA data increased the external control sample size and added to the robustness of the statistically significant treatment differences out to year three, favoring troriluzole. Troriluzole reduced SCA disease progression by 70%, which is analogous to a 2.2-year delay in progression over the three years, a remarkable treatment effect. Pooling the two natural history studies in the control arm also resulted in consistent, sustained treatment benefits at years one, two, and three.
It's important to note that a rigorous propensity score matching was again applied to this pooled cohort. The pooled analysis showed that differences in change from baseline in f-SARA at all time points were statistically significant. A 60% slowing of disease progression was seen in troriluzole-treated patients, which reflects a 1.9-year delay in disease progression over three years, so collectively, using two separate external control arms and also a pooled control arm, rigorously matched via propensity score matching, sustained and clinically meaningful treatment benefit was seen over three years of troriluzole treatment, reflecting a 50%-70% slowing of disease progression and a 1.5- 2.2-year delay in SCA decline. Falls are a significant cause of morbidity and mortality in SCA patients, and they are common, with the majority of SCA patients reporting falling in the preceding 12 months.
In our double-blind phase, there was a 53% reduction in relative risk of falls in troriluzole-treated patients. We further looked at SCA patients who are ambulatory and therefore more likely to fall. When we analyzed these patients, who, based on FSARA scores, were not dependent on a walker or wheelchair to ambulate, there was a 60% reduction in relative risk of falls in SCA patients on troriluzole. The clinical relevance to patients of a reduction in falls cannot be overstated and reflects yet another important benefit of troriluzole treatment. As you can see from this slide, troriluzole is safe and well tolerated, with an adverse event profile similar to placebo. Adverse events occurring in greater than 5% of patients are listed below in the slide, and the table shows the serious adverse events. As you can see, there were no significant imbalances between troriluzole and placebo.
Troriluzole achieved statistically a significant superiority on a total of nine consecutive pre-specified primary and secondary endpoints, and the bar for this was set very high. As mentioned earlier, the protocol was designed in discussion with the FDA, and we incorporated their suggestions into our protocol in order to apply the utmost rigor to our data analyses. This table highlights some specific examples of FDA feedback incorporated into our protocol. We adhered to FDA guidance for incorporating real-world evidence in our study design. Additionally, the FDA suggested we use only the U.S. Natural History Cohort as an external control for the primary analysis, as this study was conducted by investigators in our own trial over a similar time period, and using similar standardized assessments and study populations. Despite the smaller sample size in the U.S. study, we achieved statistically significant and meaningful treatment effects.
In order to minimize the potential for any bias, propensity score matching was applied to rigorously balance characteristics between the two study arms, including trinucleotide repeat length. As a separate analysis, patients were also matched on progression rates by genotype. The data from this study provide compelling evidence of a clinically meaningful treatment benefit of troriluzole in SCA. We are excited to move forward with submission of an NDA, and now I'm going to turn the presentation over to Dr. Jeremy Schmahmann, who, as an expert in the field for over forty years, will provide clinical commentary on the implications of our study results.
Thank you, Dr. Beiner. Good morning, everybody. Thank you for the opportunity to speak today and to comment on the positive results presented this morning. By way of background, I am a professor of neurology at Harvard Medical School, and I'm the Martha and Robert Fogelman Endowed Chair in Ataxia and Cerebellar Neurobiology at Massachusetts General Hospital, where I'm also the founding director in nineteen ninety-four of the Ataxia Center. I'm a member of the National Ataxia Foundation Medical Research Advisory Board and of the Clinical Research Consortium for the Study of Cerebellar Ataxias, or CRC-SCA. For over forty years, I've been on the front lines caring for and thinking about patients and their families with ataxia and other cerebellar disorders, and I've been a principal investigator for Biohaven and treating patients in the trial for a number of years.
Let me not sugarcoat this about the spinocerebellar ataxias. These, dominantly inherited diseases visit a genetic plague on families. They devastate generation after generation. They come on sometimes later in life, often in the prime of life, and not infrequently in childhood or in the teenage years. The main focus of attack in the spinocerebellar ataxias is the cerebellum. The cerebellum, or little brain, is critical for regulating and modulating a host of neurological functions, from movement to intellect and emotion. Ataxia degrades the ability to speak clearly so people cannot be understood. It affects mobility, so patients become unable to walk independently. They stumble and fall, and they injure themselves, and they end up using canes and walkers, and then they become confined to wheelchairs. Ataxia affects swallowing, so patients develop aspiration pneumonia, which is a threat to life. Fine motor control and dexterity disintegrate.
Their writing becomes illegible. People can no longer perform tasks that we take for granted, like buttoning or putting a key in a door or working around the house, doing a profession, playing with their grandchildren. Patients have double vision and slowed and jerky eye movements. They can't read, and they can't drive. And there are other impacts of the disease as well, which are less appreciated, but just as devastating. Patients with SCA have cramps, they have tremors, spasticity, unusual movements that can be painful and embarrassing and debilitating. They can lose control of their bladder. They develop urinary tract infections, also a threat to life. And when they don't die, they can develop difficulty with high-level executive control and personality change that robs them of the person that they used to be.
And families watch their loved ones in dismay as the disease evolves and destroys them and the life that they had planned. And the SCAs are a scourge, not just for the patient, but also they produce generational disability, despondency, and depression. There's caregiver burden because patients' families are all involved in working with them to try and get their lives to be somehow manageable. These diseases shorten lifespan, so they not only degrade quality of life, but they take life. And because they run in families, families deal with stigma and despair, and exacerbated by the fact that until now, the reality has been that there was nothing available to stop the illness, bend the arc of disease, or give patients hope for a better future.
Then comes troriluzole and the data presented this morning that show that we now have the first medication that bends the arc of the disease. We've had physical therapy and some symptomatic managements, but there was nothing available to give people an opportunity to live life better or longer. When people taking troriluzole under our care had to come off for a period between the earliest stage of the clinical trial, when the 201 trial ended, they were unhappy, and they wanted to go back on drug because the patients and families noted worsening, and so did we, the neurologists taking care of the patients. The FDA set a high bar with the f-SARA as the primary outcome of the three-year study. Remarkably, Biohaven has met this using this non-granular four-item scale that you've heard about, walking, standing, sitting, and speaking.
Each element of the scale is clinically meaningful. It matters to patients. The slowing of progression of SCA by troriluzole, demonstrated in the top-line results that you just heard about, is a watershed in the history of the SCAs and ataxia in general. This is what patients have been waiting for. It is what the doctors who have been powerless have been waiting for. The patient community is thrilled that there is a medication that has been shown to work for them, and this is a game changer, which is likely to become the standard of care in the treatment of patients with spinocerebellar ataxias.
Dr. Schmahmann, thank you for the commentary this morning, and, you know, you have been a critical leader in our troriluzole development program. We can't thank you and your co-investigators enough for working with us the last several years, to the point that now we're sitting here with top-line data that's robust and represents a paradigm shift in the treatment of SCA. So thank you.
Thank you.
Now I'd like to turn it over to John Tilton, who's our Chief Commercial Officer for Rare Diseases, and John brings a unique perspective. He's, you know, well-versed in this rare disease launch playbook, having been one of the early commercial founders at Alexion and has been preparing for this day and this data. John, looking forward to hearing about your pre-launch activities.
Thank you, Vlad and Dr. Schmahmann. A new approved treatment for SCA represents a significant commercial opportunity to serve the estimated 15,000 people living in the U.S. with this progressive and debilitating disease. This is a meaningful-sized orphan disease that has similarities in its benefits to patients and a commercial size to the recently approved Orphan Drugs like Rett syndrome and Friedreich's ataxia. Currently, there are no approved treatments for SCA. Readily available genetic testing, as well as the potential for a new therapy, will drive increased diagnosis, engage patients with strong advocacy support, and a centralized treatment approach will further support a successful launch. We believe the closest analog is Friedreich's ataxia, which has demonstrated the rapid adoption of a new therapy with a high unmet need and a strong system supporting effective treatment.
The treatment of SCA is also centralized through the leading ataxia and movement disorder centers. This allows for an efficient and targeted rare disease commercial strategy with approximately 100 major centers caring for patients in the U.S. SCA often goes undiagnosed for years, and with the advent of an approved treatment, as well as our disease and diagnostic education efforts, we will facilitate early diagnosis and treatment. We have leveraged our experience, have the plans in place for a best and first-in-class launch and excited to serve the SCA community. Our commercial plans are built on four strategic priorities. Number one, identify SCA patients and accelerate confirmed diagnosis. Number two, upon approval, establish troriluzole as the standard of care for SCA. Number three, implement a Biohaven patient services hub.
Number four, ensure that we educate the SCA treaters and patient community on the benefits of continuing troriluzole therapy, as well as providing comprehensive support. The Biohaven team has a proven track record in successful rare disease launches, have our commercial plans in place, and we'll be ready for a potential launch in 2025. Now, I'd like to turn it back to Vlad for questions.
Thank you, John. In closing, the data presented today embody the patient-first focus and determination to advance novel therapies that have long been the hallmark of Biohaven. Across our ongoing pipeline programs, there's a long-standing commitment to collaborate with the brightest minds across academia, advocacy, industry, and regulatory partners. That spirit of partnership underlines our multiyear development journey in SCA, in which the contributions of the National Ataxia Foundation, leading SCA experts from around the world, guidance from regulatory agencies and, of course, the SCA patient community, were also critical to bringing us to this day. From all of us here at Biohaven, we want to thank you for your tireless support. Biohaven's dedication to delivering transformative therapeutic value to patients is only possible through the support of our investors, who embrace our vision and provide the resources that enable us to pursue and fulfill the promise of science.
Thank you all. We will now be happy to answer any questions that you have. Operator, turn it over to you, to the queue.
Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again... And our first question comes from Tessa Romero with JP Morgan. Your line is open.
Good morning, team. This is Caroline Pocher on for Tessa Romero at JP Morgan. Thanks for taking our questions. Just two from us. So first, what gives you confidence these data would lead to an approval for troriluzole for the treatment of all SCA genotypes? And how has your regulatory dialogue post-refusal to file letter convinced you of this? And then just another follow-up. In your 2Q 2024 results, you noted that the EMA application for SCA3 remains under review. To what extent do these results change your strategy there? And do you intend to submit these as a part of your-- these results as a part of your application?
Good morning, Caroline. Thanks for the question. You know, we have always believed in the clinical efficacy of troriluzole, and, in the initial top-line data where efficacy was seen in the SCA-3 genotype, one year was sufficient in that genotype, because of the progression that occurs more rapidly in that specific patient population. And then what we saw in the extension phase was, other genotypes as they began to progress, you're able to pick up the signal. So we worked with the FDA and had discussions the last several months, regarding the ability to run an analysis and protocol like you've seen today, to capture that therapeutic potential.
I think what made the difference is both the sample size and the three-year endpoint, which demonstrated that over the study period, placebos, you have progressed about what we would have expected from the natural history cohort, but patients treated with troriluzole had a significant decline in that rate of progression. So the data is quite robust and compelling, and I think there are a number of factors that boost our confidence, not just the regulatory interactions that led to this protocol, but really the robustness of the clinical meaningfulness of the data and the statistical findings across two separate independent cohorts, as well as the pooled analysis.
So for those reasons, we feel strongly that we have to move with urgency, to submit our NDA filing, which the team is already working on, and get this to patients, as quick as possible. With the EMA, we, we have updated the EMA, regarding our FDA interactions, while we were having them in real time. They were aware of this protocol, and we have shared the study results with the EMA as well, and we will incorporate this, into our ongoing review, in Europe. Thank you for the question. Next question, operator.
The next question will come from Jason Gerberry with Bank of America. Your line is open.
Oh, hey, guys. Thanks for taking my questions. Firstly, just, do you have a sense of what proportion of this prevalent population is diagnosed currently and, you know, is potentially candidates for treatment? And I'm wondering, are those patients that are more, I guess, likely to be treated, do you think, ambulatory in nature? If you can sort of help characterize those patients. And then just one point of clarification. The press release talks about, you know, being that big at years one and two, but the chart does show an asterisk indicating statistical significance at year three. So I was wondering if you can clarify that on the primary endpoint. Thanks.
Oh, yeah, sure. So first of all, on the primary endpoint, it was statistically significant at all time points, years one, two, and three in the U.S. external control, the European and the pool. So it was clearly positive across all of those. I'll turn it over to John Tilton to talk about the identifiable patients diagnosed and eligible for treatment. But you also asked a question about ambulatory patients. Given this data, it's going to be important for patients to be treated as early as possible. What you're seeing is a disease stabilization and a savings in the loss of neurodegeneration. And so it, it's going to be critical that not only we treat people who are more advanced in the disease, but we get to them as early as possible.
John, do you want to talk about prevalence and incidence?
Yeah. So, based on the epidemiology work we've done, we believe there's about 15,000 SCA patients that are in the U.S. There's about 6,000 that we believe are diagnosed, and we've done that through work through claims and data analysis indicating that we believe that, you know, that's an accurate amount. We also know that there's a number of databases available to us that we can have immediate, you know, relatively immediate access to help us identify patients. We also know in rare disease, that it, it's common for rare diseases to go undiagnosed or underdiagnosed. Without a viable treatment and a, you know, a consistent diagnostic pathway, we have the ability to improve that and increase and accelerate diagnosis in the future.
Thanks, John, and maybe just ask Dr. Schmahmann to comment on, because there's no current treatment for SCA, upon approval, how do you think that might relate to identifying other patients?
And one of the reasons that people have not come forward is the long-held convention, notion, fact that there's nothing to do except for physical therapy and the rest of it, the symptomatic management. Now that we can say we have a drug that has been shown to slow the evolution of the disease, people will come forward in the families who have not been coming forward, who've been reluctant to come forward. There is genetic testing that the National Ataxia Foundation is offering so people can have tests done in the families. I think that the opportunity to increase the availability of access to people who have these conditions will be substantially impacted by the availability of a drug that actually does something helpful.
Thank you, Dr. Schmahmann. Operator, next question, and we're running up against time, so we just have time for a couple more questions. Operator?
Okay, our next question comes from Chris Raymond with Piper Sandler. Your line is open.
Hey, thanks for taking the question, and congrats on the, really this remarkable, turnaround. Maybe two questions, if I can. So, at first, are there any discernible, maybe for the doctor, are there any discernible, geographic differences in progression? You know, just looking at the natural history data sets, it looks like the EuroSCA patients maybe progress faster than those in the CRC-SCA scale. And then the second question is, you know, talk about, if you will, you know, differences in genotype, progression. It looks like maybe there are some imbalances between, the, the two natural history cohorts in the treatment arm. Thanks.
Hey, thanks a lot, Chris, for those questions. And, I'm gonna have Melissa Beiner, and then if Dr. Schmahmann has anything to add, but Melissa?
Yeah, thank you for the question. So in the EuroSCA study, the genotypes included were SCA 1, 2, and 3. So we know that SCA 1 is the most rapidly progressive, followed by SCA 2 and 3. In the CRC-SCA cohort, SCA 6 and SCA 8 patients were also included. They progress very slowly by comparison. So there is heterogeneity in progression rates across the different genotypes, and that's largely what we're seeing between those two different cohorts.
Yeah, so that, that explains the differences you were talking about, Chris. And what, again, is, you know, so critical here is using the propensity score matching and doing that in these two separate populations, despite some, you know, minor differences in genotypes, robust data showing efficacy in each of those different cohorts, as well as the pooled. Anything to add, Dr. Schmahmann?
No, I think that you've covered it. There is a variability in the rate of progression in the different diseases, and that's what you're picking up on in the two cohorts.
Great. Thank you. Operator, next question.
The next question will come from Tyler Van Buren with TD Cowen. Your line's open.
Great. Thanks, guys. Good morning, and good for you guys for sticking with troriluzole and SCA, especially for the patients. I have a couple. The first one is, the treatment benefit versus the control arms is striking, of course. So can you just elaborate on the discussions with the FDA and their agreement on using CRC-SCA as an external natural history control as we think about potential approval? Was it originally recommended by you guys or by them, and why is it the best external control to use? And then the second one is pretty simple. It's just, obviously, the 50%-70% slowing of disease progression over three years is impressive, but do these patients, do some of these patients feel better following treatment? And if so, when does that start?
Thanks, Tyler. You know, really appreciate those questions. So I think it was a very high bar by being limited in the primary to only the U.S. natural history cohort. We had proposed the pooled analysis because with our experience in this rare disease, we knew the greater the sample size, the better we'd be able to detect the difference. The rationale of the FDA was to use a concurrent external control in the U.S. and to limit it to U.S. only. I think some of that rationale, you know, had to do with a lot of the same sites. Also, around the same time period, we're enrolling in the natural history cohort, so it was a very relevant comparator in this U.S., you know, driven study.
And so I think those were the main reasons. However, you know, we always thought, look, this is a genotype that defines the diagnosis often in this disease. If you're genotype positive, it shouldn't matter whether you're in the U.S. or Europe, especially since there's no standard of care. So, it. I think it was a higher threshold to meet, and we, you know, beat it. We showed that troriluzole is highly effective in both cohorts studied. And that pooled analysis, I think it's important to say, it's not just combining the two analyses, but you pool the external control patients, and you do a propensity score matching. So it's an even and yet a different kind of cohort that you're comparing it to.
You really have three cohorts that are lining up and showing all consistent, you know, results from that perspective.
Thanks.
And then the last question?
The patient feeling on-
Oh, yeah, on patients, what they might observe in this study, either Dr. Beiner or Schmahmann.
You know, I'll give you a bit of background. When the 201 didn't work and patients came off the study, we heard from them across the country, "I want to go back on this drug." And it was that clear call from the patients, the doctor's impression, that we then took back to Biohaven and said: "Excuse me, I think you need to take another look at this because there's something going on here. And while it didn't work in, you know, in eight weeks, there is a story that you need to go back to." And to Biohaven's credit, they did exactly that, and here we are, you know, many years later.
Yes, you know, to answer that further, we do hear from patients, and we do hear from our movement disorder specialists involved in this trial. We have many, many patients who absolutely feel differently on this drug, including patients who were told at the beginning of the trial, for example, that within a year they'd be using a cane, and five years out on troriluzole, they're ambulating on their own without any assisted devices.
Great. Thanks, Tyler. Operator, last question.
Our last question comes from Charles Duncan with Cantor Fitzgerald. Your line is open.
Yes, super. Good morning, Vlad and team. Congratulations on these results. Thanks for taking the questions. I had a couple of questions for the KOL, Dr. Schmahmann. I wondered if he could speak to the persistence of patients and other medical resource use, what he detected over time, even anecdotally. And then to you, Vlad, in terms of FDA next steps, would you conduct a pre-NDA meeting or just file an NDA?
In terms of the patients, I have to tell you that between the patient community and the National Ataxia Foundation support, this is a remarkably cohesive group that is aware of all the issues they're struggling with, has stuck with the Biohaven. You know, who does a trial that goes on for this long? It's sort of unheard of, and the patients have stuck with it. They've traveled from all over the country, coming to the sites that do the study, because they're noticing a difference, and they want to stick with it, so there's a persistence in the patients and their commitment, and I am thrilled for the patients, and their families, that there is movement in this in a way that we can actually bend the arc of the disease finally.
Thank you, Dr. Schmahmann. And Charles, to your question, you know, we've already initiated the process to submit our NDA. We have had multiple interactions and discussions with the FDA over the last several months. I think, you know, we weren't able to share those with folks until, you know, recently, and obviously, this top-line data. So given the robustness of those discussions and most importantly, this top-line data, we've already communicated to the FDA that we will be resubmitting prior to the end of this year. And we're really excited about that and feel an obligation to move quickly since this is a neurodegenerative disorder. And you can see from the data, you must intervene to save neurons as soon as possible in these patients.
Excellent.
Thank you. Thanks, Charles, appreciate it. Thank you all on the line for questions, and we look forward to giving you further updates, as both the U.S. resubmission and the European filing continues. Thank you all.
This concludes today's conference call. Thank you for participating. You may now disconnect.