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Abstract Details

Design of KYSA-8, A Phase 2, Open-Label, Multicenter Study of KYV-101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor (CAR) T-Cell Therapy, in Treatment-Refractory Stiff-Person Syndrome
Autoimmune Neurology
P2 - Poster Session 2 (8:00 AM-9:00 AM)
8-018
To evaluate the safety and efficacy of KYV-101 in patients with refractory stiff-person syndrome (SPS).
SPS is a progressive neuroinflammatory disease in which autoreactive B cells play a pathogenic role, resulting in disability due to impaired mobility. KYV-101 is a first-in-class, fully human autologous anti-CD19 CAR T-cell therapy  with a promising clinical safety profile. KYV-101 demonstrated acceptable tolerability and promising clinical benefits, including marked improvement of stiffness, walking speed, and reduction of symptomatic medications in a patient with SPS (Faissner S, et al. PNAS 2024;121:e2403227121). KYV-101 is being investigated in KYSA-8 (NCT06588491), a phase 2 open-label, single-arm multicenter study of KYV-101 in refractory SPS. KYV-101 received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for SPS. 
Twenty-five patients 18-75 years old, with active seropositive SPS and inadequate response to at least 1 immunomodulatory therapy (IVIG, rituximab, or plasmapheresis), and stiffness index ≥2, are eligible. Patients bedridden >3 months or with a history of stem cell transplant will be excluded. Following lymphodepletion (fludarabine 30 mg/m2/day, cyclophosphamide 300 mg/m2/day; 3 days), patients will receive a single infusion of KYV-101 at the target dose of 1×108 CAR T cells. The primary endpoints are change in the timed 25-foot walk from baseline to week 16 and safety events. Secondary endpoints include other evaluations of SPS-related clinical outcomes and disease-related biomarkers, including modified Rankin scale, distribution-of-stiffness index, heightened sensitivity scores, and quality of life. 
KYSA-8 is actively enrolling patients in the US.
KYSA-8 is the first phase 2 study of anti-CD19 CAR T-cell therapy in refractory SPS. KYV-101 may potentially change the immunotherapy paradigm through deep B-cell depletion and immune reset in autoimmune diseases like SPS. 
Authors/Disclosures
Amanda L. Piquet, MD, FAAN (University of Colorado)
PRESENTER 		The institution of Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech/Roche. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Kyverna . The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech/Roche. The institution of Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyverna. The institution of Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Sands Anderson PC. Dr. Piquet has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Joe Jones Law Firm. Dr. Piquet has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Cortez & Associates. Dr. Piquet has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Falk Waas. The institution of Dr. Piquet has received research support from Rocky Mountain MS Center. The institution of Dr. Piquet has received research support from Roche/Genentech. The institution of Dr. Piquet has received research support from NYU. The institution of Dr. Piquet has received research support from Anokion. The institution of Dr. Piquet has received research support from UCB . The institution of Dr. Piquet has received research support from Foundation for Sarcoidosis. The institution of Dr. Piquet has received research support from Kyverna . Dr. Piquet has received publishing royalties from a publication relating to health care. Dr. Piquet has received publishing royalties from a publication relating to health care. Dr. Piquet has received personal compensation in the range of $10,000-$49,999 for serving as a Litigative Consultant with US-Dept HHS/DICP. Dr. Piquet has a non-compensated relationship as a Medical Advisory Board Member with Autoimmune Encephalitis Alliance (AEA) that is relevant to AAN interests or activities. Dr. Piquet has a non-compensated relationship as a Medical Advisory Board Member with Stiff Person Syndrome Research Foundation (SPSRF) that is relevant to AAN interests or activities.
Anastasia Zekeridou, MD, PhD, FAAN (Neuroimmunology Laboratory, Mayo Clinic) The institution of Dr. Zekeridou has received research support from Roche/Genentech. Dr. Zekeridou has received intellectual property interests from a discovery or technology relating to health care. Dr. Zekeridou has received intellectual property interests from a discovery or technology relating to health care. Dr. Zekeridou has received intellectual property interests from a discovery or technology relating to health care. Dr. Zekeridou has received intellectual property interests from a discovery or technology relating to health care.
Esther H. Nie, PhD (Stanford Healthcare Center for Academic Medicine (CAM)) Dr. Nie has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Nie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Medidata, Acorn AI. The institution of Dr. Nie has received research support from Roche/Genentech.
Dominic Borie (Kyverna Therapeutics Inc.) Dominic Borie has received personal compensation for serving as an employee of Kyverna Therapeutics. Dominic Borie has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Kyverna Therapeutics. Dominic Borie has stock in Kyverna.
James Chung, MD, PhD Dr. Chung has received personal compensation for serving as an employee of Kyverna Therapeutics. Dr. Chung has stock in Kyverna Therapeutics.
Marinos C. Dalakas, MD, FAAN (Thomas Jefferson University) Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols, . Dr. Dalakas has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Dysimmune Diseases Foundation. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ARGENX. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN. Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Therapeutic Advances in Neurology (TAND). Dr. Dalakas has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Medlink.