Kyverna's CAR T Therapy Breakthrough in Myasthenia Gravis Treatment

Kyverna Therapeutics has achieved a significant milestone in the treatment of generalized myasthenia gravis (gMG) with its investigational CAR T cell therapy, KYV-101. The company reported a 100% response rate in a Phase II study, setting a new efficacy standard in the field and paving the way for Phase III trials.

Impressive Phase II Results

In the mid-stage portion of the Phase II/III KYSA-6 study, KYV-101 demonstrated remarkable efficacy in patients with moderate to severe gMG. All six evaluable patients, who had previously failed immunosuppressant therapies, achieved clinically meaningful improvements in activities of daily living at 36 weeks compared to baseline.

The study defined treatment response as a minimum 3-point reduction in two disease inventories, a criterion met by all treated patients. Additionally, KYV-101 showed potential in reducing treatment burden, with all patients able to discontinue non-steroidal immunosuppressants, high-dose steroids, and other therapies after 24 weeks.

Safety Profile and Next Steps

KYV-101 was generally well-tolerated, with no reported cases of high-grade cytokine release syndrome or immune effector cell–associated neurotoxicity syndrome. One patient developed grade 4 neutropenia, which was expected and improved to grade 1 by the data cutoff.

Naji Gehchan, Kyverna's Chief Medical and Development Officer, announced plans to begin enrollment for the Phase III portion of KYSA-6 by the end of this year, marking a significant step forward in the development of this promising therapy.

Expanding Horizons for CAR T Therapies

Kyverna's success with KYV-101 is part of a broader trend of applying CAR T therapies, traditionally used in cancer treatment, to autoimmune diseases. Other companies exploring this approach include:

• Cabaletta Bio: Recently reported promising efficacy signals for its cell therapy rese-cel in multiple autoimmune conditions.
• Bristol Myers Squibb, CRISPR Therapeutics, and Autolus Therapeutics: Also leveraging CAR T platforms for autoimmune diseases.

This development represents a potential paradigm shift in the treatment of autoimmune disorders, offering new hope for patients with conditions that have been challenging to manage with existing therapies.