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Autoimmune CAR-T data dump: Kyverna, Cabaletta shares fall after unveiling early-stage results

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In 30 patients with a number of autoimmune conditions who received Kyverna Therapeutics’ CAR-T cell therapy, 21 were able to stop taking their immune disease medications. The results mark one of the largest clinical datasets presented on the use of a CAR-T therapy in autoimmune diseases like the muscle condition myasthenia gravis, lupus and others.

Kyverna’s results are part of a series of studies using CAR-T therapy to treat autoimmune disease presented at the European Alliance of Associations for Rheumatology annual meeting in Vienna this week. The early datasets come at a time of ballooning interest in using cell therapies as a potential one-time treatment for autoimmune conditions.

“It’s a big difference from last year. I would say that last year, it was a relatively new idea,” said Greg Deener, CEO of private autoimmune CAR-T company iCell Gene Therapeutics. “Now it is a dominant theme in many of the presentations.”

However, the unveiling didn’t impress investors.

Kyverna also shared in its presentation that of the 30 patients, 28 saw reduced disease activity. One lupus patient who received a lower dose of Kyverna’s therapy relapsed. And patients didn’t experience grade 3 or higher cases of immune system reactions, such as cytokine release syndrome, that are associated with using CAR-T therapies in cancer patients. However, there were three cases of low-grade neurotoxicities in Kyverna’s study.

Leerink Partners analysts noted “some investor pushback” on the nine patients who didn’t entirely stop taking immune-modulating treatments following CAR-T therapy.

The company’s shares $KYTX fell 34% Friday.

“Across multiple disease indications, multiple immune disease patients, [the safety profile] does look different from oncology, and it looks well-tolerated,” Kyverna chief medical officer James Chung told Endpoints News ahead of his presentation Friday.

University Hospital Erlangen rheumatologist Georg Schett — whose initial study of CAR-T therapy for autoimmune conditions sparked the deluge of interest in the field — serves on Kyverna’s scientific advisory board and also has a collaboration with Cabaletta Bio, which shared highly anticipated initial results on its CAR-T study.

Cabaletta reported results on one patient with immune-mediated necrotizing myopathy, an autoimmune muscle disease, and one patient with a form of lupus. Both were able to discontinue chronic maintenance therapies for their diseases, save for a planned steroid taper for the lupus patient.

There were no signs of cytokine release syndrome or neurotoxicity for either patient.

“We are encouraged by the initial data as it is quite comparable to the previously disclosed CD-19 CAR-T results in both lupus and myositis from academic collaborator Dr. Schett,” wrote TD Cowen analyst Phil Nadeau.

Cabaletta’s shares $CABA fell by more than 10% Friday.

And iCell shared data showing that 11 out of 12 patients in investigator-initiated studies of its CAR-T therapy are now in complete remission. The company plans to file applications to start its own sponsored clinical trials in China and the US this year.


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