Conference Transcript - KYTX 2026-04-22

Speaker 6

Good morning, and thank you for joining us today. We will be discussing the primary analysis from our KAISA-8 registrational trial of Miflecaptogene autolucel, or MIVCEL, formerly referred to as KYV-101 and stiff person syndrome, as well as longer-term follow-up data from our KAISA-6 phase 2 trial in generalized myasthenia gravis. I'd like to remind everyone that we will be making forward-looking statements during today's call. These statements reflect our current expectations and beliefs and are subject to risks and uncertainties that can cause actual results to differ materially. Please review the risk factors discussed in our press releases, our presentation materials, and in our filings with the SEC for additional information. On the call today are Werner Biddle, our Chief Executive Officer, Najee Ghishan, our Chief Medical and Development Officer, Dr. Amanda Piquet, Professor of Neurology at the University of Colorado Anschutz, Celine Dion Foundation Endowed Chair and Lead Investigator in our KAISA-8 Clinical Trial, and Dr. Shreem Muppetti, Clinical Professor, Adult Neurology of Stanford Medicine and Investigator in our KAISA-6 Phase II Trial. Warner will first provide brief remarks for today's call. Then Najee, Dr. Piquet, and Dr. Muppetti will cover our data for stiff person syndrome and generalized myasthenia gravis. After, Warner will close on our commercial opportunity in stiff person syndrome. Following prepared remarks, we will have a Q&A session with all speakers, including Mark Brasso, our Chief Financial Officer. With that, I'll turn the call over to Werner. Werner?

Warner Biddle, CEO

Thank you, Jessica. Here at Caverna, we're excited to share unprecedented data from our neuroimmunology franchise, including the first registrational primary analysis for an autoimmune CAR-T, marking a significant milestone for Caverna and for the field. In stiff person syndrome and generalized myasthenia gravis, our transformative data continue to strengthen our conviction in MIVCELL's differentiated profile and potential to change the treatment paradigm. Importantly, we continue to demonstrate our leadership in this space by advancing MIVCELL towards a potential first approval in autoimmunity. Our SPS-BLA submission is our top priority as we work towards unlocking a valuable commercial opportunity that not only establishes our first-in-class leadership, but also lays the foundation for expansion into additional indications, including GMG and progressive MS over time. Our Phase III trial in GMG is also advancing and paves the way to a large and growing market opportunity, and one where MIVCELL's differentiated clinical profile may offer meaningful advantages relative to standard of care and other therapies currently in development. Now let's turn to the next slide for highlights of our SPS and GMG data. From a high level, the new data presented here this week continue to reinforce MivCell's differentiated profile and our conviction in our neuroimmunology franchise. Results from a registrational phase two primary analysis in SPS demonstrate MivCell's potential to transform patient care, which we believe further supports our path to approval and confidence in our launch. Importantly, we are showing new data on our secondary endpoints, including unprecedented SPS-specific outcomes, as well as translational data that demonstrate the full spectrum of clinical benefit achieved by MIFCEL. We also saw transformative results from our longer-term follow-up data in GMG. Recall, we shared the interim results from our Phase 2 trial back in October of last year. We are very pleased to see even deeper responses as the data mature, with durability of treatment effects sustained out to one year. Notably, 100% of patients achieved clinically meaningful, rapid and robust responses, and the majority of patients continue to have few or no symptoms as of their last follow-up. These data continue to set a new standard in GMG clinical outcomes, further increasing our confidence in our ongoing phase 3 trial. Importantly, across both data sets, we're continuing to see MIV cells significantly reduced treatment burden from chronic immunotherapies. In addition, MivCell demonstrated a consistent, well-tolerated safety profile, which we believe will support the potential for outpatient administration. Turning to slide six. Today's results are underpinned by our potential first-in-class, best-in-class CAR-T construct. MivCell is designed for enhanced potency, with the only fully human CD19-targeting autologous CAR-T with a CD28 co-stimulatory domain that mediates rapid and robust signaling. More than 100 patients have been treated with MivCell to date across multiple indications. And based on the data thus far, MivCell has demonstrated deep B-cell depletion in the periphery and in targeted tissues, supporting a broad immune reset and durable remissions. In addition, MivCell has been shown to have a positive impact on a broad set of immune cell types, and in particular, regulatory T-cells, which are key for keeping the immune system in check. Finally, MivCell is a fully human car, which is designed to enhance patient tolerability. Across the patients treated to date, MivCell has demonstrated a consistent and manageable safety profile with no high-grade CRS or ICANs. Importantly, the first SPS and GMG patients treated with a single dose of MivCell have achieved durable efficacy beyond 24 months without the need for chronic immunotherapies This is truly a remarkable outcome Now I'd like to share with you a patient from our trial, which brings to light how MivCell is transforming the care for SPS Let's play the video This is a 39-year-old male patient from our trial performing the time 25-foot walk test, which is our primary endpoint for the trial. Prior to receiving MivCell, the patient requires a walker to ambulate, and despite the walking aid, you can see his walk is still slow and unstable. He has a slow posture and is focused on maintaining his balance with each step. And here is a video of him walking at just 16 weeks after a single dose of MivCell. As you can see, this is a remarkable transformation. His time to complete the walk went from 17.3 seconds to 5.4 seconds, comparable to a healthy adult. Importantly, he no longer needs a walker. Now, I'll turn the call over to Najee and Dr. Piquet to cover our registrational primary analysis for SPS. Najee.

Speaker 12

Thank you, Werner. Slide 9. As you saw from the video, SPS is a highly debilitating and progressive autoimmune disease, and unfortunately, there are currently no FDA-approved therapies for patients. SPS impacts the inhibitory signaling pathways that control the breaks on muscle contractions and help muscles relax. Symptoms include severe muscle stiffness and painful spasms due to uncontrolled muscle contractions that impact mobility and gains. Patients often resort to the use of symptomatic therapies such as multiple daily doses of benzodiazepines in addition to chronic off-label immunotherapies. Despite these treatments, most patients have an inadequate or no response. Further, chronic immunotherapies also carry long-term safety risks. SPS has a devastating impact on patients' lives, with a disease often occurring in adults during their prime years in life. Up to 80% of patients lose mobility over time, and only about 19% of patients remain able to work after four years with this disease. In addition, patients experience disease progression, which can lead to permanent disability and place them at risk of increased mortality. Turning to slide 10. The high disease burden of SDS and the urgent need for ineffective therapy is highlighted in a natural history study conducted by the University of Colorado and Johns Hopkins University. For background, this was a large multi-center retrospective natural history study examining the impact of SPS on walking speed. The study included 153 patients treated with off-labeled immunotherapies and with available longitudinal time 25 foot walk test data collected over the course of 10 years. Data demonstrated that despite being on therapies, the majority of patients showed no or minimal improvement in the time 25 foot walk test. In addition, over time, disability did not improve and patients' reliance on walking aids increased. Overall, the natural history study provides important evidence of the underserved SPS patient population and provides greater context to the transformative data that we've generated with Mifidel. Turning to slide 11, we are very excited to present full results from our primary analysis building on the top-line data that we shared last December with data cut off November 26, 2025. Our FDA-aligned CAISA-8 clinical trial is a single-arm, multi-center, open-label, registrational phase 2 trial. We have received both the RMAT and orphan drug designations for Myrcel in SPS. The trial included 26 patients. All patients discontinued their immunotherapies prior to a single dose of Myrcel. Let's turn to slide 12. Before Dr. Piquet shares the details of the data, it is important to understand how mobility is assessed for stiff person syndrome. The Time25 footwalk test is our primary endpoint and a validated tool to assess walking ability, as well as to evaluate stiffness and loss of mobility. To put things into perspective, the time that it takes a healthy individual to walk 25 feet is about 4 to 5 seconds. For patients with SPS, that time can be twice as long or even longer, depending on the severity of their disease. For this endpoint, a 20% improvement in the time 25 foot walk is considered clinically meaningful. Slide 13. Now let's turn to our secondary endpoints, which include the modified Rankin scale, or MRS, that measures the degree of disability, the Hauser Ambulation Index, or HAI, which measures the time and degree of assistance to complete the time 25 foot walk test, the distribution of stiffness index, or DSI, that measures muscle stiffness in various parts of the body. And lastly, the heightened sensitivity scale, or HSS, that measures the number of triggers of muscle spasms. All of these measures are included in our trial to ensure a robust evaluation of MEF cells' efficacy. Now I'll turn the call over to Dr. Amanda Piquet to present our impressive data. Dr. Piquet?

Dr. Amanda Piquet, Analyst — Professor of Neurology, University of Colorado Anschutz

All right, thank you. I'm excited to present this unprecedented data. So the results showed MIPSEL delivered rapid, statistically significant, and clinically meaningful improvements across all primary and secondary endpoints at week 16, with the majority of patients regaining function. Importantly, all patients discontinued chronic immune therapies and remained off as of last follow-up. From a clinical perspective, the magnitude and consistency of functional improvement observed is unprecedented. So to take you through this slide here, the primary efficacy endpoint was met. MibCell resulted in significant improvement in the timed 25-foot walk at week 16, representing a 46% median improvement from baseline. 20% improvement is considered clinically meaningful. You can see here the results were statistically significant. 81% of patients achieved or exceeded this bar, with nearly a third walking at the speed of a healthy adult by week 16. Of the 12 patients requiring a walking aid at baseline, two-thirds no longer needed assistance at week 16. This reflects a meaningful functional independence of these patients. The times 25-foot walk improvement was also sustained through 24 weeks for 16 patients who reached the time point. These findings are compelling and potentially paradigm-changing for the SPS community. Next slide. MIPSEL achieved significant improvements in disability, mobility, stiffness, and hypersensitivity. We saw significant improvements in other measures, as you can see here. The shift plots show a significant reduction in disability using the modified Rankin scale. Additionally, there was significant improvement in mobility using the Helzer Ambulation Index. Now, this incorporates the time and degree of assistance needed to walk 25 feet. There were significant improvements observed in SPS-specific measures. This included the distribution of stiffness index, which demonstrates that reduction in muscle stiffness across body regions, and the heightened sensitivity scale demonstrating that reduced sensitivity to stimuli triggering spasms. To put this in a clinical perspective, even in prior trials looking at SPS, we generally don't see with off-label therapies any reduction in that hypersensitivity. So this is incredible to see this in this population. Importantly, 96% of patients had improvement in at least one primary or secondary efficacy endpoint. Next slide. So, there were substantial improvements observed in other secondary endpoints, including the six-minute walk test and the SF36 at 16 weeks. This really reflects the improvements that we saw on physical and mental functioning in our SPS patients. In the six-minute walk, the patients experienced a median improvement of 89 meters. That's essentially walking almost the length of a football field. That is greater than fourfold from the clinical meaningful change of 20 meters. In the SF36, patients scored scores at week 16 were comparable to healthy adults. In this disease, we can see significant impairments in social functioning, emotional status, and mental health. And to have these patients exceed what we see in a healthy adult is just incredible. Next slide. So here we see robust mid-cell expansion leading to complete peripheral B-cell depletion and significant reduction in autoantibody titers. So the first panel shows this robust T-cell expansion where you can see the CAR T-cells peaking at day 14. And then 54% of patients had B-cell reconstitution at week 16. Efficacy, importantly, was maintained with B-cell reconstitution, again reiterating that no patients were resumed on immune therapies as of this follow-up. And then on the other panel here, we see this 56% reduction in GAD-65 autoantibody levels. Moving on to the next slide. This slide here further demonstrates that broad immune reset after mid-cell treatment. So of those patients that I commented on had B-cell reconstitution. They are listed here as recovery. So looking at baseline to recovery, we see these newly emerging B-cell populations showing a significant increase in the naive phenotype and a decrease in the memory phenotype. So what this means is we're seeing new, healthier B cells reemerging. Again, markers of this broad immune reset that we can see after MIV cell. Additionally, on the right side of the screen, you have significant increase in regulatory T cells at week 16. Again, further evidence demonstrating this broad immune reset extending just beyond the B-cell effects. Next slide. MIV-cell demonstrated a well-tolerated safety profile. Importantly, MIV-cell was tolerated, everything was manageable, and no high-grade cytokine release syndrome or immune factor cell-associated neurotoxicity syndrome was observed. There was grade 3-4 neutropenia, a known adverse event that is associated with lymphodepletion and CAR-T treatment that was observed in four patients, and it was manageable. The serious treatment-related AEs occurred in three patients, all of which fully resolved, and again, emphasizing that this was fully manageable. Next slide. I just want to re-emphasize in this registrational trial, we saw a single dose of MIP cell significantly reverse disability, reduce stiffness, and hypersensitivity. It improves mobility in these patients, and they remained free of chronic immune therapy treatments. These are outcomes we have not seen before in this disease. These findings represent a potentially transformative advancement for the patient population that historically have had a lack of treatments in the past and offers great

Speaker 12

promise for CAR-T. Thank you, Dr. Piquet. Next slide, please, 20. In summary, we're pleased to see a single dose of Myrcel demonstrating the potential to transform patient care in SDS. First, an ability to deliver significant clinical improvement while reversing this ability. Second, the potential to eliminate chronic immunotherapies. Third, a well-tolerated safety profile, fourth and importantly, durable clinical benefit achieved through GP cell depletion and a broad immune reset. Collectively, these outcomes represent a treatment breakthrough for patients and physicians. Now let's turn to Generalis Myrcenia Graves on slide 21. Before we begin, I'd like to share a video of a patient with refractory GMG who received MISC cell through an IH pathway in Germany. The video illustrates the significant disease burden and impact on quality of life prior to treatment, as well as the transformative improvement observed following a single dose of MISC cell. Let's play the video.

Mr. Ackerman, Analyst — Patient

And with the outbreak of the disease, the whole life plan was to end. That was bad for me. I couldn't accept that I couldn't go anywhere else. I couldn't either eat, or drink, or breathe. I could go to the car on the park. That was about 50 meters. And even those were for me too many. My self-image was almost zero. Slowly, but surely, I became more healthy. And since then, I couldn't get rid of the big trees, what I could imagine before. We traveled again. We built our garden again. We rode a bike again. And I was almost more active than before.

Speaker 12

Mr. Ackerman's improvement after MifCell is remarkable. Now two and a half years in remission and of all MGE therapies. His story, along with many others, inspire the work we do here at Kiberna. Turning to slide 23. As you saw from the video, GMG is a serious B-cell and antibody-mediated neuromuscular autoimmune disease. Symptoms are highly disruptive to quality of life and can include muscle weakness and fatigue, difficulty chewing and swallowing, trouble with speech, and in severe cases, respiratory failure, which can be life-threatening. Despite the many treatment options available on the market today, including immunosuppressants and biologists, patients still struggle with symptom control, with few reaching minimal symptom expression or MSC. As a result, the majority of patients require frequent and chronic treatment options in addition to background therapies that carry a significant treatment burden. For these reasons, novel therapies are needed to address the underlying drivers of the disease with the goal of minimizing or eliminating symptoms while reducing the reliance of chronic immunotherapy. Slide 24. This is the study design for our Kaiser 6, phase 2 trial which included 7 patients who had failed prior immunotherapies. It is important to note that all patients discontinued their Mg immunotherapies prior to receiving a single dose of Mifcel. The primary endpoints in phase 2 of this trial were the reduction from baseline in MgATL score at 24 weeks and tolerability. We continue to follow these and other secondary measures on the slide, including QMG and MGC, throughout the 18-month follow-up period, which is ongoing. The data cutoff was February 25, with the longest follow-up up to 52 weeks. Now, I'll turn the poll over to Dr. Mupiti to cover the data in greater detail.

Speaker 11

Good morning. These results are unprecedented and exciting for the field. We've seen even deeper reduction in MgADL-QMG compared to previously approved therapies. The MgADL-QMG reduction seems sustained and further reduces as you go up to week 52. MgADL demonstrated a quick reduction at week 2 of 6.4 points and further reduction of 8.5 by week 24. QMG demonstrated rapid and robust mean reduction as early as week 2 of 8.6, with further deepening up to 11.3 at week 24. While there are a number of therapies that have been approved in myasthenia, this degree of deep and sustained reduction has not been previously noticed. Next slide, please. After a single dose of MIXL, patients achieved sustained and clinically meaningful reductions in all the outcome measures that we use in myasthenic labs. 100% of the patients had clinically meaningful response in MGA-DL. 100% of the patients were responders from baseline and MGA-DL, as well as QMT. 57% of the patients had achieved MSC at the follow-up. 100% of the patients had clinically meaningful response of MGA composite and a dramatic reduction of 16 months. More importantly, all of these patients were able to come off their prior immunosuppressive therapy, including non-steroidal anti-steroid drugs, high-dose steroids, FCR, and complement inhibitors up to B24. And this part needs to be emphasized again. We do not usually take off their prior immunosuppressive therapy in other FG trials and FG agents. The fact that we are able to take these medications off and still retain the clinical benefit is not seen previously. Next slide. MIPS helped demonstrate a well-covid safety profile. There were no high-grade CRS or ICAMS. There was only low-grade CRS that were mostly fevers and were manageable quite easily. Three patients had grade 3 out of 4 treatment-related adverse events of neutropenia, lymphopenia, and low liposide count. These are expected adverse events of lymphodiflation and CAR-T cell therapies. Neutropenia was, again, fully manageable and fully dissolved. There were no serious adverse events. The previously reported treatment serious adverse events was reclassified by the investigator as not serious. Results continue to support a very favorable risk-benefit profile of the cell. Next slide. Robust CAR T cell expansion led to a very deep B cell depletion. As you can see on the slide on the left side, there was a robust increase in CAR T cell expansion. And this reflects on the slide up to the right, this deep B cell expansion. We saw recovery was detected in two patients at week 12 and week 16. In those patients where we had prior antibodies available, you're able to see there are two patients with ACHR and one patient with MASC. There's a robust reduction in antibody levels, and the reduction in antibody levels is sustained. But at the same time, patients still have continued preservation of humoral immunity, as you can see on the right side, to common vaccinations. And this is a critical point of NIL cell, is that it's able to reduce the pathological antibodies and yet preserve other tumor immunity. With that, I'll pass it on to Nadia. Thank you, Dr. Amufadil.

Speaker 12

Let's go to slide 30. As already mentioned earlier, we are truly setting a new standard across clinical outcome measures in GMG that has not been seen to date with any approved or investigational inheritance. As you saw from today's results, a single dose of MIF-cell resulted in robust, rapid, and durable improvements across all endpoints, regardless of prior biologic exposure, and with the majority of patients at MSE as of last follow-up. Opportunity to remove immunotherapies, a well-tolerated safety profile, and finally, evidence of immune reset and preserved humoral immunity. Combined, these transformative outcomes underscore MIF-cell's potential to change treatment paradigm in GMG. Importantly, we believe the significant effect size observed in MGADL and QMG, the co-primary endpoints of our Phase III trial, meaningfully increases the probability of success for the Phase III trial. Turning to slide 31 next. As we outlined during our Q4 earnings, enrollment for our FDA-aligned Phase B trial continues continues to advance with 14 sites activated globally. As we've already shared the trial design, I won't go over this slide in detail, but it's important to note that this is the first CAR-T superiority trial on GMT, evaluating MIFCEL versus standard of care. Standard of care includes traditional agents or complement inhibitors. FCRN inhibitors are excluded due to their fluctuating efficacy over dosing intervals. However, patients who have had an inadequate response to FCRNs can be included in our trial. Turning to slide 32. Before I turn the call over to Werner, I'd like to conclude on an important slide that highlights MIF-Cell's differentiated profile. While cross-trial comparisons are not based on head-to-head studies, you can see that across all primary endpoint measurements for approved and investigational therapies, MIF-Cell is the only product candidate that has demonstrated this deep magnitude of response while freeing patients from chronic background immunotherapies with a single dose. With that, I will turn the

Warner Biddle, CEO

call over to woman. Thank you, Najee. I now like to focus on our valuable market opportunity in SPS, moving to slide 34. With the results from our primary analysis, which are further reinforced by natural history data, we believe we have a strong clinical evidence to support our BLA pathway and a pathway to approval. And as the only company with a late-stage asset in this rare disease, we are uniquely positioned for a compelling commercial opportunity in SPS. As we have previously mentioned, there are approximately 6,000 diagnosed patients in the United States. Our initial priority will focus on patients with the highest unmet need who are ready now for a better treatment option. This accounts for 2,000 to 2,500 patients, or roughly 30% to 40% of diagnosed patients who have had an inadequate response to off-label immunotherapies. In this patient population, we believe MIPSEL can be immediately established as the new treatment standard. In addition, given the inadequate responses associated with symptomatic treatments, we believe that we can also target the rest of the 5,500 patients over time, based on real-world clinical evidence and increased patient education. Turning to slide 35, initial feedback from physicians reflects both excitement in our data, as well as the potential for a strong early adoption of MIB cell. We surveyed 20 high-volumes SPS treaters in the U.S. using a product profile informed by MivCell's top-line data. 80% pointed specifically to the efficacy data and the one-time treatment paradigm as the most compelling attributes of MivCell's product profile. We believe this reinforces what we see as one of the most important aspects of MivCell's value proposition supporting commercial success. 90% of patients viewed the profile as compelling versus current treatment options. Most importantly, 85% said they would use MivCell in moderate to severe patients at launch, which represents a meaningful proportion of patients that could immediately benefit from MivCell. Next slide, slide 36. We have a focused go-to-market strategy, initially targeting approximately 10 centers with immediately addressable patient population and attractive launch dynamics. Importantly, these centers were selected because they all have in common what we believe are critical attributes supporting early adoption and launch execution. First, these are centers with recognized SPS leadership, institutions that include thought leaders and high-volume treaters, along with strong institutional support. Second, there are already a significant number of SPS patients being treated at these centers, with additional patient flow supported by established referral networks. Third, all of these sites already have commercial CAR-T experience and accreditation. And finally, these centers have the potential to realize strong economics through robust inpatient and outpatient treatment models, as well as favorable reimbursement dynamics. Taken together, our launch strategy allows for an effective and efficient market entry, focusing on a relatively small number of high-priority launch centers that are well positioned to treat a meaningful proportion of immediately addressable patients. Moving to slide 37. Before we move to the Q&A, I would like to summarize our excitement about the data we presented today and our promising pathway forward. At Caverna, we're executing on our mission to deliver the curative potential of CAR-T and autoimmunity. We believe MIPSEL positions us to deliver the first approved CAR-T therapy in autoimmune diseases, supported by a potential best-in-class clinical profile and growing evidence of a durable drug-free remission. Our focused neuroimmunology strategy enables a first-to-market opportunity in stiff person syndrome, representing a high-value, commercially attractive launch, and establishing a strong foundation for expansion into generalized Mycenae Gravis and additional indications, including progressive MS. Finally, we remain well capitalized to support our SPS BLA submission, the anticipated commercial launch, and our phase three GMG trial. And with that, I'll turn the

Operator

call over to our operator for the Q&A. Thank you, Warner. I will now open the call up to a question and answer session and to ask a question please press star one one on your telephone and wait for your name to be announced and to withdraw your question please press star one one again and we ask that you please limit to one question and any additional questions return to the queue and the first question comes from thomas smith with leering partners your line is open

Thomas Smith, Analyst — Leerink Partners

Hey, guys. Good morning. Thanks for taking our questions and congrats on these really strong data updates. Great to see the consistency of these data sets as they continue to mature. Just for Dr. PK, if I could, we heard quite a lot of buzz around stiff person syndrome at the meeting. There was a really well-attended symposium on Saturday and then a seminar on Sunday. Can you just comment on the level of interest you're seeing in MIPSEL from other academic and community center colleagues who weren't involved in the CAISAH study, but have SPS patients that are looking for new therapeutic options? Thanks so much.

Warner Biddle, CEO

Yeah, Dr. Piquet, do you want to handle this question?

Dr. Amanda Piquet, Analyst — Professor of Neurology, University of Colorado Anschutz

Yeah, so a ton of excitement. And I think this is just a reflection of the lack of therapies that we've had for this rare disease over decades. And obviously, there's a lot of public interest in this disease over the last couple of years. And it's just been incredible to see the amount of research going into helping these patients. So as you said, there's a lot of excitement at this meeting, just continues to grow. And I think a lot of excitement of a MIPS cell to see something, having a treatment for these patients.

Operator

Thank you. And the next question is going to come from Derek Arcella with Wells Fargo. Your line is open.

Speaker 13

Hey, good morning, and congrats on the updates here. All the data is coming very excellent. So just kind of quick question on, you know, how efficacy kind of tracks the GAD-65 antibody reductions that we see, and then just a follow-up on that in terms of like, you know, and this is for Dr. PK, as we think about, you know, this impressive data that we're seeing in SPS, I mean, are there considerations for which you would not prescribe mid-cell for an SPS patient? Thanks.

Warner Biddle, CEO

Thanks, Derek. Appreciate that. Najee, why don't you start with the correlation of efficacy data versus the antibody titers. And then, Amanda, you can touch on the other subject.

Speaker 12

So the efficacy, as we've seen in this trial, and Dr. Piquet shared here, is really remarkable on all our primary, secondary endpoints. We've seen exploratory endpoints also being really very significant. And when you look at the totality of the titers, actually, it's correlated, right? We have this decrease in the antibody titers that is significant. We know that these titers, and Dr. Kik and comment a little bit more, are not extremely correlated exactly to the disease, but what's really key here is this consistency of results clinically and this decrease of titers that has been seen is really important to look at. Amanda, I'll pass it over to you on the other question.

Dr. Amanda Piquet, Analyst — Professor of Neurology, University of Colorado Anschutz

Yeah, so you had asked who would not be a candidate potentially for MibCell. I just want to say that SPS is a very heterogeneous disease. However, we know from natural history data that 80% of patients progress. So I'd say the vast majority are actually going to benefit from MibCell because in those patients that are progressing, We see increased disability, the need for ambulatory aids. So I think actually that's going to be a fair minority of patients that, you know, are unicorns that tend to do okay. But we know this to be a progressive disease.

Warner Biddle, CEO

Yeah, I think that's... Thank you so much. Thanks for the question. I think it's supported as well from the market research we were doing across a number of SPS treaters where we know that 85% said they would use MipCell in the moderate to severe patient population. And as you pointed out, Amanda, the prognosis of these patients gets worse over time. So the fact that we now have something that can be transformative for these patients and give them some hope, I think, is truly remarkable progress. Thanks, Derek. And Warner, just to

Speaker 13

follow up on that real quick, just on the 10 high volume centers that you guys talked about you know, being the target at launch, how many patients does that represent?

Warner Biddle, CEO

Well, of the immediately addressable patient population, we know there's between two to two and a half thousand patients that we've identified that are already refractory to existing immunotherapies or existing off-label treatments. And of the 10 high value centers that we're targeting at launch, that'll cover roughly half of those patients. But I think it's really, really important to keep in mind that those 10 centers are just the starting point for us. We're targeting that as a launch target for ourselves, but we're going to continue to add authorized treatment centers as we continue to move through the launch sequence. The additional centers that we add will not only provide additional patient potential for SPS, but as you know, we're looking ahead to generalized myasthenia gravis, and because of the synergy between these two indications, the additional centers that we're adding will also allow us to treat more patients with GMG over time as well.

Speaker 13

Excellent. Thanks again, guys.

Operator

Thank you. And our next question is going to come from Brian Chung with J.P. Morgan. Your line is open.

Speaker 9

Hi, guys. Good morning and congrats on the data again. This is Ron on for Brian. Looking ahead into the approval for MIVCELL and SPS, how should we think about the label language? Do you see potential for this therapy to be approved for the broad SPS population or do you anticipate approval will be dedicated only to GAD65 or VLAIR positive patients?

Warner Biddle, CEO

Yeah, thank you for the question. Yeah, the BLA preparations are a top priority for us, and we're moving with a strong sense of urgency. And based on this really robust clinical data, we feel really confident in our clinical package, and we've had some really productive dialogue with the FDA up to this point. Najee, maybe you want to provide a little bit of perspective on how we see the label, because this data is truly unprecedented.

Speaker 12

Yeah, so we're not the disclosing specific label, obviously, at this point. And it's certainly our top priority as we are going through the VLA process. But as you can see, and as Dr. Piquet shared here, there's no current approved therapy for this disease. And the impact we're seeing is truly remarkable and majority of patients would benefit from. So this is something how we're thinking about it as we are going through our VLA filing.

Speaker 9

Thank you, guys.

Operator

Thank you. And the next question will come from Michael. It's with Morgan Stanley. Your line is open.

Speaker 10

Great. Good morning. Thanks for taking my question, and congratulations on all the updates as well. Maybe another question for Dr. PK on SPS. You shared a number of benefits across endpoints that were consistent. Just curious if there's particularly one endpoint that really stood out to you or do you think that might be most meaningful for patients? Thanks. Thanks, Michael.

Warner Biddle, CEO

I think when you take a step back and think about the totality of this data set, I think it's worth reinforcing that we're not only seeing, you know, statistically significant and clinically meaningful impact on just one of these endpoints, but just across the entire spectrum. But Amanda, maybe you want to comment on some of the secondary and exploratory endpoints and what piques your

Dr. Amanda Piquet, Analyst — Professor of Neurology, University of Colorado Anschutz

interest from a clinician's perspective? Yeah, I mean, again, reiterating, all endpoints are amazing. So you're asking me to pick basically my favorite child, which you're trying not to do. But I would say what is really amazing is the six-minute walk test, because I mean, to walk an additional football field um that that's just incredible um so i would say i i think i was most impressed with that end point great thank you thanks thank you thank you and our next question

Operator

comes from matt phipps with william blair your line is open good morning thanks for taking my

Matt Phipps, Analyst — William Blair

question and congrats on all the updates dr pique i was wondering if you know you could talk a little bit of how your center will manage patient demand or how you see that going on approval. Is this something where you, you know, it might be one patient a week, given there's obviously lots of other CAR-T trials and, you know, heme indications, or is it, you know, maybe any idea on how many patients you could process? And then just wanted to confirm that only two patients received tocilizumab in the SPS study. And would you ever think about using prophylaxis to, thank you.

Warner Biddle, CEO

Yeah, I think one of the key learnings we're seeing, Matt, as we set up these centers, both from a clinical trial perspective, as well as looking ahead to commercial, is this partnership between the autoimmune treater, in this case, the neuroimmunology specialists and the CAR-T specialists. And I think that's been one of the keys of success to us as we have enrolled the clinical trials. And it's what we're working on right now as we gear up for launch. But Amanda, if you want to provide a little perspective on how you guys are thinking about it from the University of Colorado, that would be helpful.

Dr. Amanda Piquet, Analyst — Professor of Neurology, University of Colorado Anschutz

Yeah, so we have a cellular therapy program that's been established, and we are partnered with our hematology group in the autoimmune space. So we have representative neurologists like myself, as well as rheumatologists, nephrologists, and GI docs all kind of coming together and having this multidisciplinary cellular therapy program. And basically what that group does is help support our current clinical trials, as well as planning for future commercial implementation. So we're investing in basically increasing the capacity for these patients. And I'm just very excited to be able to bring this CAR-T therapy, potentially if approved, into the commercial setting. And like I said, our university is investing improvements for the treatments of patients with autoimmune diseases, including SPS.

Warner Biddle, CEO

Yeah, and this is very similar to what's happening at Stanford as well, like this partnership between the neuromuscular physicians and with the CAR-T specials as well. So I don't know if you want to provide a little bit of color on the situation at Stanford, which is progressing very nicely, too.

Speaker 11

Yeah, so thank you. We have a similar protocol where we have a DIAD model for this disease expert like myself in different diseases. And then we have a bone marrow transplant, a CAR-T expert from hematology. We work together closely and our teams work together. It really improves the efficiency from point of patient selection all the way down to the time that we are able to offer CAR-T to the patient.

Warner Biddle, CEO

That's great. And I know there's a second part of the question about using prophylactic therapies in order to manage and advance the A-profile. Najee, do you want to start on that? Because I think there's been a tremendous amount of progress on how these protocols have been put in place over the past few years.

Speaker 12

Yeah, so as we've said, with MipCell closing more than 100 patients, we have no high-grade CRS ICANs. And certainly, in the past 15 years in CAR-T, the management of CRS and ICANs have dramatically improved. So for MipCell, we do have DEX dosing to the regimen of how to treat CRS and ICANs. And we are looking into prophylaxis, to your point, prophylaxis DEX. It has shown to be efficacious to avoid CRS and also not impacting the expansion. This is well known. So certainly the way those are managed and then this consistency of the safety profile that we have with MipCell is certainly prone for it to be an outpatient when it gets into clinical mission.

Operator

Thank you. As a reminder, to ask a question, please press star 11 on your telephone. Our next question comes from Mitchell Kapoor with HC Wayne. Right. Your line is open.

Warner Biddle, CEO

Hi, can you hear me? You bet, Mitch. Hello.

Jan Zies, Analyst — HC Wayne

Hi. So, this is Jan Zies, sitting in for Mitchell Kapoor. Thanks for taking my question and for the exciting data. This is great. I have a question on the sustained 52-week response. So, the mean curves look rapid and then durable. But beneath those averages, are you seeing distinct response patterns such as patients who plateau early versus patients who, say, benefit and that continues to deepen over time?

Warner Biddle, CEO

Jan, are you speaking about the stiff-person syndrome or myosinia gravis?

Jan Zies, Analyst — HC Wayne

The MGADL.

Warner Biddle, CEO

Right, sure. Najee, you want to take that because this deepening impact is, I think, one of the most important aspects that we're seeing coming out of the data.

Speaker 12

Okay. So what we're really excited about with this update, we're seeing here six patients at 24 weeks with this profound response on MG-ATL and QMG. And as you can see, we have five patients at nine months and three patients at a year with this continued sustained impact on their disease, whether it's MG-ATL or QMG. And importantly, as Dr. Mopedi shared here, I'll turn it over to him, those strong impact on the disease itself is also achieved with freedom of drug and burden of background therapies. So this is really one of the key points in this disease and how we're changing the paradigm is this ability to free patients from their disease and their background immunosuppressants, and it sustains the patients that have reached this one-year time point. I don't know, Sri, if you want to add something to that.

Speaker 11

Yeah, I think another important thing to add, and sometimes we tend to forget when we look at this data, is we have to always think of where the patients started. So these seven patients were refractory, had tried multiple other therapies, including currently FDA-approved therapies. And so we are able to offer, and they were still symptomatic on those therapies. So we are able to offer something that I hadn't experienced before. And so for me, both the depth of the response and sustained response is quite remarkable. I haven't been appreciated before.

Jan Zies, Analyst — HC Wayne

Thank you so much.

Operator

Thank you. And at this time, there are no further questions in the queue. So now I would like to turn the call back over to Warner.

Warner Biddle, CEO

Thank you, operator. And thank you all for joining us today at AAN. On behalf of my entire leadership team, I want to express our gratitude to the patients and families who participated in the Kaiser VI and AIDS trials. And to our investigators and the clinical site teams, including Dr. Piquet and Dr. Mappetti, who graciously shared their time with us today. I also want to acknowledge the entire Kyvern organization for their hard work and dedication on behalf of these patients. We look forward to updating you on our progress ahead. Thank you.