Kyverna's KYV-101 Gets FDA RMAT Status for Refractory Stiff-Person Syndrome Treatment

Kyverna Therapeutics, Inc. (Kyverna), a clinical-stage biopharmaceutical company based in Emeryville, California, has received the Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for its CAR T-cell product candidate, KYV-101. This designation applies to the treatment of patients suffering from refractory stiff-person syndrome (SPS), a rare autoimmune disorder with severe symptoms.

Stiff-person syndrome is a debilitating and progressive neurological condition that causes significant muscle stiffness in the torso, arms, and legs, severely influencing mobility. The disease often manifests through muscle spasms and an abnormal posture that can be triggered by sudden stimuli such as loud noises, unexpected touches, or emotional stress. Due to its symptoms, SPS is frequently misdiagnosed as a psychological disorder.

KYV-101, the product in focus, is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell therapy designed specifically for B cell-driven autoimmune diseases. The CAR in KYV-101 was developed by the National Institutes of Health (NIH) to enhance tolerability and underwent initial testing in a Phase 1 trial involving 20 cancer patients, with results published in Nature Medicine.

Kyverna's CEO, Peter Maag, Ph.D., expressed optimism about the potential of KYV-101 to offer significant benefits to patients with SPS. He emphasized the importance of generating data from the KYSA-8 trial to explore the possibility of resetting the immune system of affected patients. Dr. Amanda Piquet, director of the Autoimmune Neurology Program at CU Anschutz Medical Campus, also highlighted the potential of KYV-101 to transform the treatment landscape for SPS, a disease known for its debilitating impact.

Up until now, KYV-101 has been administered to 50 patients for both oncological and autoimmune conditions across more than 15 locations in Europe and the U.S. The ongoing clinical evaluations include Phase 1/2 and Phase 2 trials in the U.S. and Germany, with a focus on various autoimmune disorders in the fields of rheumatology and neurology.

In addition to sponsored trials, KYV-101 is also being assessed through investigator-initiated studies across multiple regions for a variety of indications. The promising results and differentiated properties of KYV-101 are seen as crucial for advancing CAR T-cell therapies as effective treatments for autoimmune diseases.

Kyverna Therapeutics is committed to advancing cell therapies for autoimmune diseases, aiming to provide innovative treatments that can potentially offer durable, immunosuppressant-free remission. Alongside KYV-101, the company is developing next-generation CAR T-cell therapies in both autologous and allogeneic formats. These therapies are designed to be effective in managing B cell-driven autoimmune conditions, further underscoring Kyverna's dedication to addressing unmet medical needs in this area.

Overall, the RMAT designation from the FDA is a significant step for Kyverna, allowing the company to receive expert guidance on efficient drug development and the use of surrogate endpoints from senior FDA officials. This development is expected to expedite the clinical advancement of KYV-101, bringing hope to patients with refractory stiff-person syndrome and potentially other autoimmune diseases.