Kyverna’s CAR T-Cell Therapy Candidate Cleared for Phase 2 Trial in Progressive MS

California-based Kyverna Therapeutics recently announced the FDA approval for its investigational CAR T-cell therapy KYV-101 to proceed to a phase 2 study in patients with refractory progressive multiple sclerosis (MS).

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CAR stands for chimeric antigen receptor. CAR T-cell products are made by re-engineering T cells in a laboratory to produce certain proteins that recognize and attach to antigens on target cells and destroy them. With autologous CAR T-cell therapy, the patient’s own T cells are collected, modified and reinfused back to the patient.

KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate. It targets the CD19 protein ubiquitous on the surface of B cells, which are known to mediate various types of autoimmune diseases, including MS.

Currently, Ocrevus (ocrelizumab) is the only FDA-approved treatment for primary progressive MS (PPMS). For patients who do not respond to available treatments, there are limited options.

“This approval is a critical 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,” Bruce Cree, M.D., Ph.D., clinical research director and professor of clinical neurology at the University of California, San Francisco, commented in a press release.

“This study offers participants a new hope for arresting relentless disability worsening and a potentially durable, treatment-free remission,” Cree added.

The phase 2 open-label KYSA-7 trial will enroll approximately 12 patients with PPMS or secondary progressive MS (SPMS). With PPMS and SPMS, symptoms gradually worsen with no relapsing or recovery periods. Eligible participants must be ages 18 to 55 years with no disease relapses within the past two years.

The trial will evaluate participants for dose-limiting toxicities over the course of one year. Secondary outcomes include safety parameters and response to treatment. In a previous phase 1/2 study, KYV-101 was associated with lower inflammatory activity typically responsible for adverse events in other CAR T-cell therapies.

The investigational treatment is also in another phase 2 study evaluating its use in lupus nephritis. Kyverna is planning future trials for the potential use of KYV-101 in patients with systemic sclerosis and myasthenia gravis. In addition, allogenic forms of CAR T-cell therapies targeting B cell-mediated autoimmune conditions are in the company’s pipeline.