Kyverna Therapeutics, Inc. Common Stock Wells Fargo 21st Annual Healthcare Conference

Sure. Well, at Kyverna, we're really leading the industry, as you know, Derek, bringing these transformative cell therapies to patients and bringing it to them very quickly. Starting with stiff person syndrome, which is our lead indication. We read out our pivotal data earlier this year, and we showed some very transformative results in these patients that have no approved therapy and where the disease continues to progress to a point of disability in a majority of these patients. What we saw for the first time ever is not just an improvement in clinical symptoms, but for the first time ever, a reversal of disability, which has never been seen before in stiff person syndrome, all with a one-time treatment with miv-cel that allowed patients to come off their background therapies and actually live a drug-free, disease-free remission.

On the basis of this data, we've actually started our rolling BLA submission with the FDA, and that's been a really positive milestone for us. In fact, earlier with our Q2 earnings, we announced that we actually finished our CMC filing of the CMC module, which is the key milestone in a cell therapy registry submission, which puts us on track to finish the filing in Q4 of this year and will put us on track, once approved, to be the first therapy ever approved in stiff person syndrome, but more importantly, the first company ever to bring a cell therapy to autoimmune diseases anywhere in the world.

Well, up to this point, we filed all the key modules except for the clinical module, and we've stated this publicly that we'll be reading out our one-year data, which is coming imminently here as a key milestone in Q3. We'll be including that one-year data in the BLA submission, along with additional analysis of our natural history study, which we did in stiff person syndrome. We read out the top-line data on this natural history study earlier this year, but we're providing additional analyses on that for the FDA, and we're going to include those two things in our clinical package. As I say, we're on track to complete the filing in Q4, which puts us on track to be launch-ready in 2027.

Well, if you recall from our primary analysis that we read out earlier this year, we saw a significant clinical response across all the primary, secondary, and exploratory endpoints. So significant improvement in the timed 25-foot walk, which was our primary endpoint, as well as secondary endpoints specific for SPS as well as general movement disorders. We also saw a significant improvement in the six-minute walk test, which is another landmark indicator of improved mobility in patients. In fact, we saw an over 90-meter improvement, which has never been seen before in this disease. On the basis of this and what we're hoping to read out here in the next couple of weeks is a continued progression on that data. Can we show a majority of patients continuing to have this significant remission in their disease and significant improvement in their clinical findings?

Can we also see a significant number of these patients remain off background immunosuppressants like IVIG that they've been chronically burdened from for years? If we're able to show this and continue to demonstrate that durability effect, I think we continue to reinforce that we have a very transformative therapy here for stiff person syndrome. Again, a disease that has no approved therapy and where patients naturally progress over time.

Well, it is difficult to predict on an Advisory Committee, but we do have an RMAT designation, as we have discussed before, and we will be filing with a priority review. We think all of the things that would support that really line up very nicely, including a couple of things that you mentioned, the high unmet need in this disease, the transformative clinical results that we are seeing, the amazing safety profile that we are actually seeing in these patients as well, and the fact that there is no approved therapies in this space. I think all of those things justify a priority review, and we will come back to you and everyone with a status update once we have received that.

Well, through the RMAT designation, we have had regular contact with the FDA, and the dialogue has been very productive. In fact, the review committee that we are working with has been largely unchanged throughout the entire process, including the CMC review committee, and they have been very supportive. There is a strong understanding that there is nothing approved, that the prognosis for stiff person syndrome patients is horrendous. The natural history of these patients at best stays flat, but we know over 80% of them will progress to debilitating disease that requires a walker or a wheelchair or even being bed bound over the course of their diagnosis. There is really, truly a high unmet need here, and like I said earlier, miv-cel is really showing a transformative result, not just an improvement in clinical symptoms, but actually reversal of the course of the disease.

In fact, we know that two-thirds of the patients that required a walker or an assisted walking device at the beginning of the clinical trial didn't need that walking device at the end of the trial. We're doing something here very revolutionary, and something that also allows patients to come off their background immunosuppressants and other chronic therapies that they've been burdened with many, many years and still achieve this dramatic clinical result.