Efficacy and safety of lisocabtagene maraleucel in adult Japanese patients with relapsed or refractory follicular lymphoma: 31-month follow-up of the phase 2 TRANSCEND FL study

BACKGROUND: Lisocabtagene maraleucel (liso-cel), a CD19-directed chimeric antigen receptor T-cell therapy, showed high response rates and favorable safety in patients with relapsed/refractory (R/R) FL in the international, pivotal, phase 2 study (TRANSCEND FL). We report results from TRANSCEND FL in Japanese patients with second-line or later (2L+) FL after a median on-study follow-up of 31 months. METHODS: Key eligibility criteria were age ≥18 years, prior combination systemic therapy including an anti-CD20 antibody and alkylating agent, and positron emission tomography-positive, measurable lesions. Primary endpoint was overall response rate (ORR) per independent review committee using Lugano 2014 criteria. Secondary endpoints included complete response (CR) rate, duration of response (DOR), progression-free survival (PFS), overall survival (OS), and safety. RESULTS: Overall, 10 Japanese patients received liso-cel and were evaluated for safety; 9 were efficacy-evaluable. Patients had a median age of 67 years and a median of 3 prior systemic therapies. ORR and CR rate (95% CI) were both 100% (66.4-100). Median DOR and PFS were not reached, 24-month DOR and PFS rates (95% CI) were both 88.9% (43.3-98.4), and only 1 patient had relapsed. All patients were alive at data cutoff. Rates of any-grade cytokine release syndrome (CRS), neurological events (NEs), and infections were 30%, 10%, and 30%, respectively; no grade ≥3 CRS, NEs, or infections were reported. No new safety signals were detected. CONCLUSIONS: These data underscore liso-cel as an effective treatment option with a manageable safety profile for Japanese patients with 2L+ R/R FL. Trial registration number NCT04245839.