A single-arm, open-label, multicenter trial (Study 1; NCT02601313) evaluated the efficacy of a single infusion of TECARTUS in adult patients with relapsed or refractory mantle cell lymphoma (MCL) who had previously received anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody, and a Bruton tyrosine kinase inhibitor (BTKi; ibrutinib or acalabrutinib). Eligible patients also had disease progression after their last regimen or refractory disease to their most recent therapy. The study excluded patients with active or serious infections, prior allogeneic hematopoietic stem cell transplant (HSCT), detectable cerebrospinal fluid malignant cells or brain metastases, and any history of central nervous system (CNS) lymphoma or CNS disorders.
Seventy-four patients were leukapheresed, 5 (7%) of whom did not begin conditioning chemotherapy or receive TECARTUS: 3 (4%) experienced manufacturing failure, 1 (1%) died of progressive disease, and 1 (1%) withdrew from the study. One patient (1%) received lymphodepleting chemotherapy but did not receive TECARTUS due to ongoing active atrial fibrillation. Sixty-eight of the patients who were leukapheresed received a single infusion of TECARTUS, and 60 of these patients were followed for at least 6 months after their first objective disease response, qualifying them as efficacy-evaluable. Among the 60 efficacy-evaluable patients, 2 × 106 CAR-positive viable T cells/kg were administered to 54 (90%). The remaining 6 (10%) patients received doses of 1.0, 1.6, 1.8, 1.8, 1.9, and 1.9 × 106 CAR-positive viable T cells/kg.
Of the 60 efficacy-evaluable patients, the median age was 65 years (range: 38 to 79 years), 51 patients (85%) were male, and 56 patients (93%) were white, 1 patient (2%) was Black or African American, 1 patient (2%) was Pacific Islander and 2 patient (3%) were of “Other” race. Fifty patients (83%) had stage IV disease, 8 patients (13%) had stage III disease and 2 patients (3%) had stage II disease. Twenty patients (33% of 60) had baseline bone marrow examinations performed per protocol; of these, 10 (50%) were negative, 8 (40%) were positive, and 2 (10%) were indeterminate. The median number of prior therapies was 3 (range: 2 to 5). Twenty-six (43%) of the patients had relapsed after or were refractory to autologous HSCT. Twenty-one (35%) had relapsed after their last therapy for MCL, while 36 (60%) were refractory to their last therapy for MCL. Fourteen patients (23%) had blastoid MCL, 35 patients (58%) had Classical MCL, 1 patient (2%) had Other and 10 patients (17%) had Unknown MCL type. Following leukapheresis and prior to administration of TECARTUS, 21 (35%) of the 60 patients received bridging therapy. Sixteen (27%) were treated with a BTKi, 9 (15%) with a corticosteroid, and 4 (7%) with both a BTKi and a corticosteroid.
Among the 60 efficacy-evaluable patients, the median time from leukapheresis to product delivery was 15 days (range: 11 to 28 days), and the median time from leukapheresis to product infusion was 27 days (range: 19 to 63 days). The protocol-defined lymphodepleting chemotherapy regimen of cyclophosphamide 500 mg/m2 intravenously and fludarabine 30 mg/m2 intravenously, both given on each of the fifth, fourth, and third days before TECARTUS infusion, was administered to 53 (88%) of the 60 efficacy-evaluable patients. The remaining 7 patients (12%) either received lymphodepletion over 4 or more days or received TECARTUS 4 or more days after completing lymphodepletion. All treated patients received TECARTUS infusion on Day 0 and were hospitalized until at least Day 7.
The primary endpoint of objective response rate (ORR) per the Lugano Classification (2014) in patients treated with TECARTUS in Study 1 Cohorts 1 as determined by an independent review committee is provided in Table 9. The median time to response was 28 days (range: 24 to 92 days) with a median follow-up time for DOR of 8.6 months.
Table 9. Efficacy Results in Adult Patients with Relapsed/Refractory MCL (Study 1 Cohort 1) | Efficacy-Evaluable Patients N = 60 | All Leukapheresed Patients (ITT) N = 74 |
|---|
| CI, confidence interval; CR, complete remission; DOR, duration of response; NE, not estimable; NR, not reached; PR, partial remission. |
| Response Rate |
| Objective Response Rate, n (%) [95% CI] | 52 (87%) [75, 94] | 59 (80%) [69, 88] |
| Complete Remission Rate, n (%) [95% CI] | 37 (62%) [48, 74] | 41 (55%) [43, 67] |
| Partial Remission Rate, n (%) [95% CI] | 15 (25%) [15, 38] | 18 (24%) [15, 36] |
| Duration of Response (DOR) |
Median in months [95% CI] Range in months | NR [8.6, NE] 0.0+, 29.2+ | NR [8.6, NE] 0.0+, 29.2+ |
DOR, if best response is CR, median in months [95% CI] Range in months | NR [13.6, NE] 1.9+, 29.2+ | NR [13.6, NE] 0.0+, 29.2+ |
DOR, if best response is PR, median in months [95% CI] Range in months | 2.2 [1.5, 5.1] 0.0+, 22.1+ | 2.2 [1.5, 5.1] 0.0+, 22.1+ |
| Median Follow-up for DOR in months
| 8.6 | 8.1 |
A subsequent, open label cohort in Study 1 (Cohort 3) evaluated the efficacy of TECARTUS in patients with relapsed or refractory MCL who had been treated with up to 5 prior treatment regimens but had not received prior therapy with a BTKi. A total of 95 patients were enrolled and leukapheresed, 8 (8%) of whom did not begin conditioning chemotherapy or receive TECARTUS: 3 (3%) experienced an AE, 1 (1%) died due to an AE, 1 (1%) withdrew from the study as the product did not meet specifications, 1 (1%) withdrew consent, 1 (1%) experienced deterioration of their general condition, and 1 (1%) experienced progressive disease. One patient (1%) received lymphodepleting chemotherapy but did not receive TECARTUS due to rapid and uncontrolled disease progression. Eighty-six patients who were leukapheresed received a single infusion of TECARTUS, and were followed for at least 18 months after their first objective disease response, qualifying them as efficacy-evaluable.
Of the 86 efficacy-evaluable patients, the median age was 64 years (range: 40 to 82 years), 67 (78%) were male, and 19 (22%) were females, 78 (91%) were white, 2 (2%) other, 1(1%) Asian and 5 (6%) were not reported. Most (56 patients; 65%) had stage IV disease, 3 (3%) had stage I, 17 (20%) had stage II, and 10 (12%) had stage III disease. All patients had baseline bone marrow examinations performed per protocol; of these, 52 (60%) were negative and 34 (40%) were positive. The median number of prior therapies was 1 (range: 1 to 5). Seventy-four patients (86%) had relapsed after their last therapy for MCL, while 12 (14%) were refractory to their last therapy for MCL. Sixty-five patients (76%) had classical MCL, 6 (7%) had blastoid MCL, 6 (7%) had pleomorphic MCL, and 9 (10%) were other. Following leukapheresis and prior to administration of TECARTUS, 31 (36%) of the 86 patients received bridging therapy.
The median time from leukapheresis to product delivery was 24 days (range: 14 to 43 days), and the median time from leukapheresis to product infusion was 33.5 days (range: 19 to 85 days). The protocol-defined lymphodepleting chemotherapy regimen of cyclophosphamide 500 mg/m2 intravenously and fludarabine 30 mg/m2 intravenously, both given concurrently for 3 days (Day 5 to Day 3) before TECARTUS infusion to all 86 efficacy-evaluable patients. All treated patients received TECARTUS infusion on Day 0 and were hospitalized until at least Day 7.
The primary endpoint was objective response rate (ORR) per the Lugano Classification (2014) as determined by an independent review committee.
The efficacy results are summarized in Table 10 below.
Table 10. Efficacy Results in (Study 1 Cohort 3) | Efficacy evaluable patients
N = 86 | All Leukapheresed Patients (ITT) N = 95 |
|---|
| CI, confidence interval; CR, complete remission; DOR, duration of response; NE, not estimable; NR, not reached; PR, partial remission. |
| Response Rate | |
| Objective Response Rate, n (%) [95% CI] | 78 (91%) [82.5, 95.9] | 78 (82%) [72.9, 89.2] |
| Complete Remission Rate, n (%) [95% CI] | 68 (79%) [69.0, 87.1] | 68 (72%) [61.4, 80.4] |
| Partial Remission Rate, n (%) [95% CI] | 10 (12%) [5.7, 20.3] | 10 (11%) [5.2, 18.5] |
| Duration of Response (DOR)
|
Median in months [95% CI] Range in months | NR [26.2, NE] 0.0+, 35.3+ |
DOR, if best response is CR, median in months [95% CI] Range in months | NR [26.2, NE] 0.5, 35.3+ |
DOR, if best response is PR, median in months [95% CI] Range in months | 13.3 [2.1, NE] 0.0+, 16.5+ |
| Median Follow-up for DOR in months
| 23.0 |
The median time to response was 1 month (range: 0.8 to 1.9 months)