Kyverna's KYV-101 Gains FDA RMAT Status for Progressive Myasthenia Gravis Treatment

Kyverna Therapeutics, Inc., a biopharmaceutical company dedicated to developing cell therapies for autoimmune diseases, has announced that its leading CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). This designation is aimed at accelerating the development and review processes for promising therapies designed to treat serious conditions. The decision was based on positive clinical outcomes observed in Germany under the named-patient program.

The RMAT designation will facilitate more rapid consultations and increased involvement from senior FDA officials throughout the development of KYV-101. This is a significant milestone for Kyverna, as it underscores the FDA's interest in novel treatments for severe autoimmune diseases, such as myasthenia gravis.

Myasthenia gravis (MG) is an autoimmune disorder that causes muscle weakness, affecting various tissues throughout the body. Patients with MG develop antibodies that attack essential signaling proteins at the nerve-muscle junction, impairing nerve communication with muscles. Symptoms can include partial paralysis of eye movements, difficulties in chewing and swallowing, respiratory problems, speech challenges, and skeletal muscle weakness. Although symptoms can be transient and may remit spontaneously in the early stages, they tend to worsen over time, with exacerbations potentially lasting for months. About 20% of MG patients experience at least one episode of respiratory crisis.

KYV-101 is an autologous, fully human CD19 CAR T-cell product designed for B cell-driven autoimmune diseases. Developed by the National Institutes of Health (NIH), the CAR in KYV-101 has shown improved tolerability in a Phase 1 oncology trial involving 20 patients, with results published in Nature Medicine. The therapy is currently being evaluated in several Phase 1/2 and Phase 2 clinical trials in both the United States and Germany, addressing a variety of autoimmune conditions in the fields of rheumatology and neurology.

With 50 patients treated so far across more than 15 locations in Europe and the U.S., KYV-101 has demonstrated unique properties that are crucial for the success of CAR T cells in treating autoimmune diseases. The therapy is also being tested in investigator-initiated trials for multiple indications across different regions.

Kyverna Therapeutics, headquartered in Emeryville, California, focuses on developing advanced cell therapies for autoimmune diseases. Their pipeline includes next-generation CAR T-cell therapies in both autologous and allogeneic formats, tailored for B cell-driven autoimmune conditions. Alongside KYV-101, which is advancing through clinical development for conditions such as stiff person syndrome, multiple sclerosis, systemic sclerosis, and lupus nephritis, Kyverna is committed to creating transformative treatments for patients.

The RMAT designation marks a pivotal achievement for Kyverna, potentially enhancing the development and regulatory pathways for KYV-101, and ultimately benefiting patients who suffer from severe autoimmune diseases. The ongoing collaboration between Kyverna and the FDA promises to accelerate the availability of innovative therapies that could significantly improve patient outcomes.