Johnson & Johnson and Legend Biotech on Tuesday released interim results from the Phase III CARTITUDE-4 study, touting significant survival improvement in multiple myeloma patients after treatment with their CAR-T therapy Carvykti (ciltacabtagene autoleucel).

The partners did not provide specific data from the second prespecified interim analysis of CARTITUDE-4, only announcing that Carvykti treatment led to a “statistically significant and clinically meaningful improvement” in overall survival, compared with standard therapies. The Phase III study also found that Carvykti’s safety profile in this setting was consistent with its approved label.

Jordan Schecter, disease area leader of multiple myeloma at J&J Innovative Medicine, said in a statement that with these findings Carvykti has become “the first cell therapy to significantly improve overall versus standard of care for patients with multiple myeloma as early as second line.”

J&J and Legend will present findings from CARTITUDE-4 at an upcoming medical congress and will share them with global regulatory authorities.

Carvykti is a genetically modified autologous T-cell immunotherapy that targets the BCMA protein, which is typically expressed by multiple myeloma cells. This mechanism of action allows Carvykti to direct T-cells to BCMA-positive cells and activate their anti-cancer activity. The CAR-T therapy won its first FDA approval in February 2022 for the treatment of patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy.

In April 2024, the regulator expanded its label for use in patients who had undergone at least one prior treatment. The supplemental approval was backed by progression-free survival data from CARTITUDE-4, which showed that Carvykti reduced the risk of disease progression or death by 74% versus standard of care. These findings were published in June 2023 in The New England Journal of Medicine.

Carvykti, along with other CAR-T therapies, has in recent months been at the center of an FDA safety probe. The regulator announced in November 2023 that it was looking into reports of secondary malignancies in patients treated with CAR-T treatments. In April 2024, the FDA required manufacturers of these therapies to reflect the risk of secondary cancers in their products’ boxed warnings.

Last month, findings from a Stanford study published in The New England Journal of Medicine showed that such risk of secondary malignancies was very low.

Of 724 patients who had undergone CAR-T treatment at Stanford Health Care between 2016 and 2024, only 6.5% developed secondary cancers over a median follow-up of three years. One patient died from a secondary malignancy, though this was more likely due to the immunosuppression associated with CAR-T treatments rather than a wrong insertion of the gene in the CAR construct, according to Stanford researchers.

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