FDA Clears Kyverna's KYV-101 for Refractory Stiff-Person Syndrome Trial

Kyverna Therapeutics, Inc., a clinical-stage biopharmaceutical company based in Emeryville, California, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. The approval allows the company's fully human anti-CD19 chimeric antigen receptor (CAR) T-cell product, KYV-101, to be used in treating stiff-person syndrome (SPS) as part of the Phase 2 KYSA-8 clinical trial.

Stiff-person syndrome is a rare and progressive neurological autoimmune disorder characterized by muscle stiffness, primarily affecting the torso, arms, and legs. This condition severely impacts patients' mobility, making walking or moving difficult, and can be exacerbated by sudden stimuli such as loud noises or unexpected touches. Current treatments focus only on alleviating symptoms, as there is no cure for SPS.

KYV-101 is an autologous CAR T-cell therapy designed specifically for B cell-driven autoimmune diseases. This therapy was developed by the National Institutes of Health (NIH) and tested in a Phase 1 trial involving 20 oncology patients, with results published in Nature Medicine. Kyverna has been evaluating KYV-101 in Phase 1/2 and Phase 2 trials in the United States and Germany, covering a range of autoimmune diseases in both rheumatology and neurology.

Leading the clinical effort is Dr. Marinos Dalakas, a prominent neurologist and researcher in SPS at Thomas Jefferson University School of Medicine in Philadelphia. Dr. Dalakas emphasized the importance of the KYSA-8 trial, noting its potential to offer long-lasting benefits to SPS patients who do not respond to existing therapies.

Sham Dholakia, M.D., Kyverna's business unit head for rare diseases, expressed confidence in the IND clearance. He highlighted the collaborative approach taken by the FDA and Kyverna's commitment to advancing a potentially transformative treatment for SPS. The approved target dose for KYV-101 in the trial is 100 million cells.

Kyverna's broader pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats. These therapies are designed to be suitable for a variety of B cell-driven autoimmune diseases. The company is conducting multiple clinical trials, including Phase 2 trials for conditions such as multiple sclerosis, myasthenia gravis, and lupus nephritis, in addition to the ongoing trials for SPS.

With over 50 patients treated with KYV-101 across more than 15 locations in Europe and the U.S., the therapy has shown promise in both oncological and autoimmune settings. Kyverna believes that the unique properties of KYV-101 could be crucial for the success of CAR T-cell therapies in treating autoimmune diseases.

Kyverna Therapeutics continues to focus on developing advanced cell therapies to address unmet medical needs in autoimmune diseases. The company aims to bring innovative and effective treatments to patients, with KYV-101 being a leading candidate in their therapeutic arsenal. The ongoing trials and future research efforts are expected to further define the efficacy and safety of KYV-101, potentially offering new hope to patients suffering from debilitating conditions like stiff-person syndrome.