Kyverna Therapeutics, Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

Thank you, Mitchell. It is a pleasure to be here.

Sure. Well, at Kyverna, we're leading the world in bringing the transformative power and curative potential of cell therapies to autoimmune diseases. We've got a unique CAR construct and a well-established manufacturing process, and together we're accelerating this with a focus on neuroimmunology diseases, starting with Stiff Person Syndrome, which is a rare, debilitating disease with no approved therapies and where we've shown transformative results. We are in the process of filing our BLA and on track to complete that BLA filing at Q4 of this year, which would mean we would be the first approved therapy in this condition anywhere in the world, and also the first company anywhere in the world to have an approved cell therapy in autoimmune diseases. This is a really important milestone for us.

This becomes just a starting point for the rest of our pipeline and how we're thinking about the neuroimmunology portfolio. We have really promising data in generalized myasthenia gravis, as well as other conditions like progressive MS, and we're continuing to build a long-term future and potential for how we can bring miv-cel, this transformative therapy, to more patients around the world.

Sure. Well, first thing, let's just acknowledge this is really unfortunate to hear these announcements about these patients. It was really difficult to hear that news. I think it does really underscore what we're doing here at Kyverna is different.

Different in 2 important ways, and you touched on it briefly. First, we have a very unique construct. We're the only CD19 CAR therapy in the autoimmune disease space with a CD28 costimulatory domain, a fully human design, and other modifications to the CAR that have been specifically put in place in order to enhance the safety profile of this CAR. In fact, miv-cel was in-licensed from the NIH with the specific intent to actually be used in autoimmune diseases, and it maintained the potency of earlier generations of CAR T therapy, so there's this potential for this potent B-cell depletion and autoimmune reset and long-term efficacy. But at the same time, a tenfold reduction in the potential for cytokine release as well as ICANS and neurological AEs.

We believe the CAR construct is extremely important, and in the 100-plus patients we've now dosed with miv-cel, we've seen no high-grade CRS, no high-grade ICANS, no reported cases of the IECHS events that some of our peers are reporting. This really underscores the importance of the safety of the construct as the first key pillar. But you touched on the second important point, and I think that is the manufacturing, because in the case of CAR T therapies, the product is the process and the process is the product, and the manufacturing's extremely important. At Kyverna, we're using well-established, well-validated manufacturing process, the traditional manufacturing process. We're not using rapid manufacturing, and I think this is important because that can introduce a different set of variabilities into the CAR itself and could result in a different side effect profile for patients.

Because of our well-established manufacturing, as well as our specifically designed CAR for improved safety, we think this is bearing out in the clinical profile we're now seeing with miv-cel. Like I said, in over 100-plus patients we've now treated, we have a very well-established safety profile and one that we believe will lend itself well when we scale to commercialization.

Sure. Well, stiff person syndrome is a rare condition, but an important one. There are 6,000 patients in the U.S. right now that are suffering from this disease, and this is a progressive disease. It is an autoimmune mediated disease, instigated by GAD65 and other antibodies, but it is a B-cell mediated antibody disease, which is why KYV-101 is so uniquely positioned actually to support and help these patients. We do know the natural history of these patients is horrendous. Over the course of their lifetime, and many of these patients are diagnosed in the middle of their lives, so they have a huge amount of their lives ahead of them, but 80% of them will progress to severe disability where they will need a walker or a wheelchair or even be bed bound.

We know that less than 20% of them will be employed in their jobs just four years after the initial diagnosis, so many of them become homebound. There is a huge cost to the system in terms of lost time from work, but also caregiver costs. There are no approved therapies. There are no approved FDA therapies, and the off-label therapies that these patients are taking do not really work. They help maybe symptomatically for a short period of time, but as I say, most patients progress and become more severely debilitated. Which is why the results that we are seeing with miv-cel in our pivotal clinical study are so remarkable.