FDA grants fast track status to KYV-101 for progressive forms of MS

Newly cleared trial to test cell-based therapy in treatment-resistant patients

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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The U.S. Food and Drug Administration (FDA) has granted fast track status to KYV-101, Kyverna Therapeutics’ cell-based therapy candidate for people with progressive forms of multiple sclerosis (MS) that are treatment-resistant.

The FDA designation is intended to accelerate the development of therapies that aim to address unmet medical needs for people with serious or life-threatening conditions. Among other incentives, it makes Kyverna eligible for more frequent meetings and discussions with the FDA about KYV-101’s clinical developmental plan.

The award of fast track status follows the FDA’s clearance, earlier this month, of a Phase 2 trial (NCT06138132) to test KYV-101 in people with progressive disease types whose condition worsens despite the use of standard MS treatments.

“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,” Peter Maag, PhD, Kyverna’s CEO, said in a company press release.

“This marks another important milestone in our endeavor to change the treatment paradigm with KYV-101,” Maag added.

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Developing KYV-101 as treatment for PPMS or SPMS

In MS, the immune system attacks and destroys the myelin sheath, a protective coating around nerve fibers that helps them transmit signals efficiently, leading to nerve fiber degeneration.

Many immune cell types are involved in the inflammatory responses that drive multiple sclerosis. B-cells, for example, normally produce antibodies that help the body fight off infections and other pathogens, but become abnormally primed to attack the nervous system in MS.

KYV-101 belongs to a class of treatments called CAR T-cell therapies. These essentially involve isolating a patient’s immune T-cells and engineering them in the lab to carry a man-made receptor, called chimeric antigen receptor, or CAR, that recognizes a disease-related target. These cells are then expanded to millions and infused back into the patient to help eliminate that target.

In the case of KYV-101, the CAR receptor is designed to recognize the CD19 protein found at the surface of B-cells, providing the immune system with the ability to target and eliminate these disease-causing, antibody-producing B-cells.

It is believed that it may take only a single treatment of KYV-101 to provide sustained disease control for people with progressive MS.

The therapy was previously tested in people with B-cell cancers in a Phase 1/2 trial (NCT02659943). It was found to have anti-cancer effects, while having a better safety profile than that seen in previously developed CAR T-cell therapies.

The upcoming MS Phase 2 trial, called KYSA-7, is expected to enroll 12 adults with either primary progressive MS (PPMS) or secondary progressive MS (SPMS). Eligible patients will not have experienced relapses or active inflammatory lesions in the last two years, and will have an MS type that is refractory, or resistant, to other MS treatments.

The trial, expected to run through mid-2027, aims as its primary goal to evaluate the treatment’s tolerability at multiple dose levels. Researchers also will monitor the therapy’s safety and pharmacological properties. Further areas for study are KYV-101’s effectiveness at delaying disability worsening and walking function decline, and at slowing brain shrinkage, a sign of neurodegeneration.

KYV-101 also is being evaluated in a Phase 2 study involving people with lupus nephritis, another autoimmune disease. Trials also are being planned for people with systemic sclerosis and myasthenia gravis.