Kyverna's CAR-T Therapy Shows Promise in Autoimmune Disease Trial

Kyverna Therapeutics has reported encouraging results from a small-scale trial of its CAR T-cell therapy, KYV-101, in patients with generalized myasthenia gravis (gMG). The phase 2 data suggest that the treatment could offer significant benefits for those with moderate to severe autoimmune conditions who have not responded to existing therapies.

Impressive Efficacy in gMG Patients

The trial, which is part of a larger study to support KYV-101's registration for gMG treatment, included six patients with an average disease duration of 5.3 years. These individuals had previously tried immunosuppressant therapies, including FcRns and complement inhibitors, without success.

Key findings from the trial include:

• An eight-point improvement on the MG-ADL scale in patients with 24 weeks of follow-up
• A 7.7-point drop on the quantitative myasthenia gravis (QMG) scale
• Two out of three patients showing minimal symptom expression by Week 24

Dr. Srikanth Muppidi of Stanford Medicine, commenting on the results, described the data as "quite promising," though he noted that the higher disease burden in Kyverna's trial participants could affect cross-trial comparisons.

Safety Profile and Side Effects

The safety data from the trial revealed:

• All six patients experienced cytokine release syndrome, a common side effect of CAR-T therapies
• Two cases were grade 2, while four were grade 1, indicating relatively mild effects
• No cases of immune-effector-cell-associated neurotoxicity syndrome were reported
• Some patients experienced grade 3 and 4 cases of low neutrophils, linked to lymphodepletion

Competitive Landscape in Autoimmune CAR-T Therapies

Kyverna's results appear favorable when compared to other treatments in development for gMG:

• KYV-101's 8-point MG-ADL improvement surpasses the 3.1 to 4.6-point reductions seen in trials of argenx's Vyvgart and AstraZeneca's Ultomiris
• The therapy also outperformed Amgen's Uplizna and Cartesian Therapeutics' Descartes-08, both of which showed 4.2-point changes on the MG-ADL scale

Other companies developing CAR-T therapies for gMG include Cabaletta Bio and Novartis, both working on CD19-directed treatments. Cartesian's Descartes-08, an mRNA CAR-T therapy targeting BCMA, represents a different approach in the field.

Warner Biddle, Kyverna's CEO, expressed increased confidence in the potential for phase 3 success, noting that the current results exceeded the assumed magnitude of effect for the pivotal study's primary endpoint. The company is now enrolling 60 participants for the phase 3 portion of the study, which will compare KYV-101 to the standard of care in gMG treatment.