First Use of KYV-101 for Severe Stiff-Person Syndrome Published in PNAS

25 June 2024
Kyverna Therapeutics, Inc., a biopharmaceutical company dedicated to developing cell therapies for autoimmune diseases, announced a significant milestone with the publication of their latest research in the Proceedings of the National Academy of Sciences (PNAS). This study involves the application of KYV-101, an anti-CD19 CAR T-cell product candidate, in a 69-year-old patient diagnosed with treatment-refractory stiff-person syndrome (SPS).

Patient received KYV-101 after conventional therapies failed. The notable results included a significant improvement in walking distance and a 40% reduction in GABAergic medications. The treatment was well-tolerated, showing only low-grade cytokine release syndrome (CRS) and no immune effector cell-associated neurotoxicity syndrome (ICANS), which supports continued investigation of KYV-101 in neuroimmunological diseases.

The patient, previously unable to walk beyond 50 meters, reported an uninterrupted walking distance of several kilometers within three months post-treatment. Simon Faissner, M.D., a lead co-author and professor of translational neuroimmunology at Ruhr University Bochum, expressed optimism about these improvements, suggesting they could herald a paradigm shift in managing debilitating autoimmune diseases if confirmed by further studies.

Ralf Gold, M.D., another co-author and chair of Neurology at Ruhr University Bochum, emphasized the transformational effects observed in a patient unresponsive to standard treatments for several years. He highlighted the absence of neurotoxicity and the reduction in pathogenic anti-GAD65 autoantibodies, indicating potential for further studies to validate these promising findings.

Peter Maag, Ph.D., CEO of Kyverna, noted the broader implications of KYV-101's positive outcomes, including those seen in patients with multiple sclerosis and myasthenia gravis. He emphasized the company's dedication to patient care and scientific progress.

Stiff person syndrome (SPS) is a rare neurological disorder characterized by severe muscle stiffness and spasms, leading to difficulty in movement and walking. It often results in the patient's walking resembling a statue due to severe stiffness. Triggers can include sounds, sudden touches, and conditions that cause anxiety, often leading to misdiagnosis as a primary anxiety disorder. Currently, there is no cure for SPS, and treatments only aim to manage symptoms.

KYV-101, designed by the National Institutes of Health (NIH), is a fully human CD19 CAR T-cell product candidate for B cell-driven autoimmune diseases. Initially tested in a 20-patient Phase 1 trial in oncology, its positive results have been published by the NIH in Nature Medicine. Kyverna is currently conducting open-label Phase 1/2 and Phase 2 trials in the U.S. and Germany for autoimmune diseases in rheumatology and neurology. To date, 50 patients have been treated with KYV-101 across more than 15 locations in Europe and the U.S. The product is also being studied in investigator-initiated trials for various indications globally.

Kyverna Therapeutics centers its efforts on developing innovative cell therapies for autoimmune disease patients. Their lead product, KYV-101, is progressing through clinical trials targeting multiple sclerosis, myasthenia gravis, systemic sclerosis, and lupus nephritis. The company is also developing next-generation CAR T-cell therapies tailored for B cell-driven autoimmune diseases.

The latest findings on KYV-101's efficacy and safety offer promising prospects for its future in treating autoimmune conditions. The ongoing and future research aims to further establish KYV-101's potential to revolutionize treatment paradigms for these challenging diseases.

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