Setback for Novartis’s Kymriah: CAR-T therapy fails to meet primary endpoint in Phase 3 study

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Novartis’s Kymriah CAR-T therapy did not meet its primary endpoint of event-free survival in a phase 3 study in patients with aggressive B-cell non-Hodgkin lymphoma (NHL) after relapse or lack of response to first-line treatment.

Novartis and the trial teams will carry out a full evaluation of the BELINDA study data.

“We were hopeful the BELINDA study would show that Kymriah could improve outcomes and the overall treatment experience for these patients in need.

“The study investigators will work together with Novartis in the coming weeks and months to understand the factors that contributed to this outcome,” said Michael R Bishop, professor of medicine and director of the Hematopoietic Stem Cell Transplantation Program, University of Chicago Medicine and BELINDA steering committee chair.

The trial enrolled patients from over 73 sites in 18 countries worldwide. The primary endpoint was event-free survival (EFS) defined as the time from the date of randomization to the date of the first documented disease progression or stable disease at or after the week 12 assessment, per blinded independent review committee (BIRC), or death at any time. Secondary endpoints include EFS as assessed by local investigator, overall survival, overall response rate, duration of response, time to response and safety. Patients in the control arm, receiving SOC, had the opportunity to cross over to receive Kymriah upon progression determined by BIRC.

“Patients with aggressive B-cell non-Hodgkin lymphoma who are refractory to first-line treatment are vulnerable and we are disappointed that the BELINDA study did not meet its primary endpoint in this setting,” said Jeff Legos, executive VP, global head of oncology and hematology development.

The CAR-T therapy is currently approved for the treatment of relapsed or refractory pediatric and young adult acute lymphoblastic leukemia adult diffuse large B-cell lymphoma (DLBCL).

“Kymriah continues to demonstrate durable responses for patients with certain advanced blood cancers in the third-line setting. We remain committed to accelerating development of Kymriah and our next-generation CAR-Ts and anticipate sharing early clinical results for these therapies at an upcoming medical meeting,” added Legos.