Evaluating CAR-T KYV-101 for Generalized Myasthenia Gravis

Kyverna Therapeutics is currently developing the chimeric antigen receptor T-cell (CAR-T) therapy KYV-101 for the treatment of various autoimmune diseases. Among several trials the company is conducting is the phase 2/3 KYSA-6 clinical trial (NCT06193889), which is evaluating KYV-101 in patients with generalized myasthenia gravis (gMG).

Updated data from KYSA-6, specifically pertaining to the 6 patients who had been treated in the phase 2 portion as of an October 3, 2025, data cutoff, were recently presented at the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 29 to November 1, in San Francisco, California. At the conference, CGTLive® interviewed Naji Gehchan, MD, MBA, the chief medical and development officer of Kyverna, to learn more.

CGTLive: Can you give some background context about the data Kyverna presented?

Naji Gehchan, MD, MBA: We're excited to share here at AANEM the interim results of the 6 patient part of our phase 2 trial, which is a portion of our phase 3 registrational trial, KYSA-6. In this trial, we're looking at the impact of KYV-101, which is a CD19, CD28 CAR T-cell therapy, in gMG.

What were the key data points?

We are really very excited with the impact of this therapy for generalized gMG patients. Usually what you would be looking at in those therapies is an impact on the scores of Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG), which are scores done by patients and physicians. What we've seen with KYV-101 is a profound impact at 2 weeks that was sustained over 24 weeks and 36 weeks after the single dose of therapy.

Also, you're looking at safety, and we've shown a tolerable safety profile with CAR-T. What we are looking at is cytokine release syndrome (CRS) and immune effector cell associated neurotoxicity syndrome (ICANS). In the beginning, we had no ICANS and only low grade CRS in this part of this trial and this is consistent with what we've seen with 100 patients already dosed with KYV-101.

But really the promise of KYV-101 is this durable, drug-free, disease-free remission, and this can only be achieved with a drug like KYV-101. What we've seen with those results is on top of this remarkable impact on the scores, 100% of patients were responders across the different scores we have in the trial. Two out of 3 patients achieved minimal symptom expression. This is something that is not seen with other therapies. But also one of the major changes, a paradigm change in MG, is the ability with KYV-101 after 1 single dose to free patients from their immunosuppressants: 100% of patients were free of immunosuppressants up to 24 weeks and 5 out of 6 patients were free of all their immunosuppressants at their last follow-up. This is the true change in treatment paradigm we see with KYV-101.

How would you summarize the big picture implications of this for doctors and the broader healthcare community?

As a physician, and certainly as a treating physician, the ultimate goal is freeing patients from their chronic disease—freeing them from the therapies they need to take chronically. Being able today with a single dose of KYV-101 to achieve those results, a durable drug-free, disease-free remission, is really a change of paradigm of how you treat MG. Today, currently, the treatment options that exist, and even those that are in investigation don't bring this profound impact, and on top of it, don't achieve a disease-free and drug-free remission. This is like the larger picture that we're looking to do with KYV-101.

Are there any future plans for this program that you can discuss more?

These interim phase 2 results are really, really unprecedented results in this disease. As we look forward, we have agreements with with authorities to move into a phase 3 trial, that is a randomized phase 3 trial in MG where we will be looking at the impact of KYV-101, compared to standard of care. That's a superiority trial that we will be doing, and patients will have the opportunity to cross over to standard of care after their primary end point of MG-ADL and QMG of 24 weeks. We're really looking forward to demonstrate the impact that we've seen in our phase 3 trial now moving forward and bringing this therapy to patients.

Is there anything else you want to add?

The promise of the promise of CAR T-cell therapy, and specifically here KYV-101, is bringing to patients this hope of a disease-free and drug-free remission that is durable. Today, we're getting closer to this hope. This is why I'm at Kyverna, this is why the team is at Kyverna. I wake up every day to bring this hope to patients and we're really a step closer after those very promising phase 2 results, and as we move forward into our phase 3, to making it a reality for patients living with MG.

This transcript has been edited for clarity.