Trial to Test CAR T-Cell Therapy for Refractory Progressive MS

An autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate recently received clearance from the US Food and Drug Administration (FDA) as an investigational new drug for the treatment of patients with refractory progressive multiple sclerosis (MS). Additionally, the FDA granted KYV-101 a fast-track designation for the treatment of refractory progressive MS, biopharmaceutical developer Kyverna Therapeutics Inc. announced.

KYV-101’s clearance as an investigational new drug allows for the enrollment of patients with refractory progressive MS into a phase 2, open-label trial of KYV-101 CAR T-cell therapy.

“This very important study will answer whether CAR T-cell therapy offers a new treatment option for patients living with MS. This therapy holds the promise to alter the treatment paradigm of MS by fundamentally readjusting the immune system,” said Manuel Friese, MD, professor of neurology and director of the Institute of Neuroimmunology and Multiple Sclerosis at the University Medical Center Hamburg-Eppendorf, in Hamburg, Germany. CAR T-cell therapy modifies a patient’s T cells to recognize and remove B cells. According to Kyverna, KYV-101 specifically targets a protein expressed on the surface of B cells called CD19, which is involved in various autoimmune diseases. Multiple sclerosis is a chronic neurodegenerative autoimmune disease.

Kyverna is currently conducting trials of KYV-101 in patients with lupus nephritis, and trials in patients with systemic sclerosis and myasthenia gravis are planned.

The fast-track designation for KYV-101 was announced shortly after its investigational new drug clearance. The fast-track program facilitates expedited development and review of new drugs to address unmet needs of patients with serious conditions.

“We appreciate the FDA’s support to accelerate the development of potentially life-changing CAR T-cell therapies that could greatly benefit patients living with severe and debilitating neurological autoimmune diseases,” said Peter Maag, PhD, chief executive officer of Kyverna. “This marks another important milestone in our endeavor to change the treatment paradigm with KYV-101.”

References

Kyverna's KYV-101 receives US FDA clearance for treatment of patients with refractory, progressive multiple sclerosis in the KYSA-7 phase 2 trial. News release. Kyverna Therapeutics; January 3, 2024. Accessed March 5, 2024. https://www.prnewswire.com/news-releases/kyvernas-kyv-101-receives-us-fda-clearance-for-treatment-of-patients-with-refractory-progressive-multiple-sclerosis-in-the-kysa-7-phase-2-trial-302025646.html

Kyverna Therapeutics granted FDA fast track designation for KYV-101 in the treatment of patients with refractory progressive multiple sclerosis. News release. Kyverna Therapeutics; January 19, 2024. Accessed March 5, 2024. https://www.prnewswire.com/news-releases/kyverna-therapeutics-granted-fda-fast-track-designation-for-kyv-101-in-the-treatment-of-patients-with-refractory-progressive-multiple-sclerosis-302039087.html