CAR-T Cell Therapy Receives IND Clearance from the FDA to be Studied as Potential Treatment for Myasthenia Gravis

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 as a potential treatment for people with myasthenia gravis (MG). KYV-101 is designed to modify the behavior of T cells to target the CD19 protein expressed on the surface of the B cells involved in MG pathology.

The IND clearance enables Kyverna Therapeutics to initiate a phase 2 open-label, multicenter clinical trial called KYSA-6, with the aim of investigating KYV-101 as a potential therapy for MG. KYSA-6 is part of a pipeline of clinical trials investigating KYV-101, including the ongoing phase 1 study KYSA-1 (NCT05938725) in the United States and the ongoing phase 1/2 clinical trial KYSA-3 in Germany assessing the medication’s use for refractory lupus nephritis, as well as the ongoing phase 1/2 study KYSA-5 in the United States for diffuse cutaneous systemic sclerosis.

“We have seen firsthand the transformative effects of KYV-101 in MG patients treated with the investigational therapy in our clinic,” said Professor Aiden Haghikia, Director of the Department of Neurology at Otto-von-Guericke University. “I welcome the FDA's decision and look forward to more clinical data to further our knowledge about CAR T-cell therapy in patients with severe neurological autoimmune diseases.”

MG is a chronic autoimmune disease resulting in weakness of skeletal muscles, partial paralysis of eye movements, problems with chewing and swallowing, respiratory problems, and speech difficulties. a