Abstract Details

Title Design of KYSA-6, a Phase 2, Open-label, Multicenter Study of KYV-101, a Novel Fully Human Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy in Refractory Generalized Myasthenia Gravis

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P7 - Poster Session 7 (5:00 PM-6:00 PM)

Poster/Presentation
Number 11-034

Objective To evaluate the safety and efficacy of KYV-101 in patients with refractory generalized myasthenia gravis (MG).

Background MG is a B-cell-mediated autoimmune disease for which new therapeutic paradigms are needed that can improve disease activity in refractory patients and enable sustained treatment-free remission. KYV-101 is a first-in-class, fully human autologous anti-CD19 CAR T-cell therapy with a promising clinical safety profile. In the named patient setting, KYV-101 demonstrated rapid, profound improvements in disease severity and was well-tolerated in patients with severe refractory MG. In a case study of a patient with treatment-refractory, anti-acetylcholine receptor (AChR) positive generalized MG, a single infusion of KYV-101 resulted in rapid improvement of symptoms and mobility (Haghikia A, et al. Lancet Neurol. 2023;22:1104-1105). KYV-101 is being investigated in KYSA-6 (NCT06193889), a phase 2 open-label, multicenter study in MG. KYV-101 received FDA Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designation for MG.

Design/Methods

Twenty patients 18–75 years old, with generalized MG (class IIB-IV per MG Foundation of America), the presence of disease-related autoantibodies, an MG-ADL score ≥6, who failed prior immunosuppressive/immunomodulatory therapy, are eligible. After 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. Primary endpoints are the safety and efficacy of KYV-101, assessing adverse events and MG Activities of Daily Living (MG-ADL) at 24 weeks. Secondary endpoints include evaluation of further efficacy, disease-related autoantibodies, and patient-reported outcomes.

Results KYSA-6 is ongoing in the US and Germany.

Conclusions

KYV-101 is a novel therapy with the potential to change the treatment paradigm through deep B-cell depletion and immune reset with a single infusion in patients with B-cell driven autoimmune diseases like MG. KYV-101 is also under investigation in additional neurologic and rheumatologic diseases.

Authors/Disclosures
Aiden Haghikia, MD (Hannover Medical School) PRESENTER	Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck Serono. The institution of Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Galapagos. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Merck Serono. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kyverna. Dr. Haghikia has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Roche. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Janssen. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Bayer. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Neuraxpharm. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Biogen. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Sanofi. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Novartis. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Merck Serono. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Celgene.
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.
Ralf Gold, MD (Neurologische Universitaetsklinik)	Dr. Gold has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen . Dr. Gold has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Gold has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genzyme. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer Vital. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai Pharamaceuticals. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Gold has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for SAGE Publishers. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Novartis. Dr. Gold has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Biogen. The institution of Dr. Gold has received research support from Novartis. The institution of Dr. Gold has received research support from Biogen.

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