KYV-101 CAR-T Cell Therapy for Stiff-Person Syndrome Receives Clearance for Further Study

The Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for KYV-101 (Kyverna Therapeutics, Emergyville, CA), an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell therapeutic candidate, to be studied as a potential treatment for people with stiff-person syndrome (SPS). KYV-101 is designed to modify T cells to target CD19, a protein expressed on the surface of the B cells involved in autoimmune diseases.

The new IND clearance enables Kyverna Therapeutics to conduct a phase 2 trial, KYSA-8, with the aim of investigating the efficacy of KYV-101 as a potential therapy for people with SPS. KYSA-8 is part of a pipeline of clinical trials investigating KYV-101, including the phase 2 open-label KYSA-7 and KYSA-6 clinical trials, investigating the therapeutic candidate’s efficacy as a treatment for multiple sclerosis (MS) and myasthenia gravis (MG), respectively.

"CAR T-cell therapy has already shown preliminary but promising results in patients with SPS treated outside of the US," said Marinos Dalakas, MD, FAAN, Professor of Neurology and Director of the Neuromuscular Division at Thomas Jefferson University School of Medicine. "I find the KYSA-8 trial of extraordinary importance as a promising novel therapy for patients with stiff person syndrome who do not respond to current therapies, with implications in providing potentially long-lasting benefits."