Bristol-Myers Squibb announced that its blood cancer treatment lisocabtagene maraleucel (liso-cel) has achieved its primary and secondary endpoints in TRANSCEND NHL 001 study.
Liso-cel is an investigational CD19-directed chimeric antigen receptor (CAR) T-cell therapy with a defined composition of purified CD8+ and CD4+ CAR T cells in relapsed/refractory large B-cell lymphomas.
In the trial, 344 patients were leukapheresed and 269 patients secured liso-cel at one of three dose levels.
TRANSCEND NHL 001 is an open-label, multicentre and pivotal phase 1 study designed to assess the safety, pharmacokinetics, and antitumor activity of liso-cel in patients with relapsed/refractory B-cell non-Hodgkin lymphoma, including diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal B-cell lymphoma, follicular lymphoma Grade 3B and mantle cell lymphoma.
The treatment-related adverse events, dose-limiting toxicities and objective response rate are the primary outcome measures of the trial, while complete response rate, duration of response and progression-free survival (PFS) are the secondary outcome measures.
The overall response rate (ORR) was 73% among patients evaluable for efficacy in the trial, and up to 53% of patients achieved a complete response (CR).
Median progression-free survival (PFS) was 6.8 months and median overall survival (OS) was 21.1 months, said the company.
The firm plans to complete the submission of a biologics licence application to the US Food and Drug Administration (FDA) by the end of this year, based on results from TRANSCEND NHL 001. Liso-cel has not yet secured approval for any use in any country.
Bristol-Myers Squibb cellular therapy development senior vice president Dr Stanley Frankel said: “These pivotal longer-term results from TRANSCEND NHL 001 continue to give us confidence in the clinical profile of liso-cel. Importantly, these results were demonstrated in a study with more than 250 patients in a broad population reflective of clinical practice, including those with poor prognoses and a range of histologies.
“We look forward to providing these data to support the regulatory approval for this treatment option for these patients with large B-cell lymphomas.”
Recently, the company secured breakthrough therapy status from the FDA for its Orencia (abatacept) to prevent moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors.