KYV-101 CAR-T Cell Therapy Receives IND Clearance to be Studied as Potential Treatment for Multiple Sclerosis

The Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for KYV-101, (Kyverna Therapeutics, Emeryville, CA), an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell therapeutic candidate, to be studied as a potential treatment for people with multiple sclerosis (MS). In 2023, KYV-101 received FDA Fast Track Designation for investigation as a possible treatment for myasthenia gravis (MG). 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 IND clearance enables Kyverna Therapeutics to initiate a phase 2 open-label clinical trial, KYSA-7, with the aim of investigating KYV-101 as a potential therapy for MS. KYSA-7 is part of a pipeline of clinical trials investigating KYV-101, including the phase 2 open-label, multicenter trial KYSA-6, which will investigate KYV-101’s efficacy in MG. Additionally, both the ongoing phase 1 study KYSA-1 (NCT05938725) in the United States and the ongoing phase 1/2 clinical trial KYSA-3 in Germany are assessing the medication’s use in refractory lupus nephritis.

"This approval is a critically necessary step that paves the way to enroll patients with treatment-refractory progressive MS for whom there are no currently available treatment options in the KYSA-7 trial. This study offers participants a new hope for arresting relentless disability worsening and a potentially durable, treatment-free remission," said Bruce Cree, MD, PhD, MAS, Clinical Research Director and Professor of Clinical Neurology at the University of California, San Francisco, CA.