ELARA Study Design

ELARA was a pivotal, global, open-label, multicenter, single-arm, phase II trial of tisagenlecleucel in 97 adult patients with r/r FL.^1,2

• Patients in the trial were refractory to or relapsed within 6 months after completion of 2 or more lines of systemic therapy, relapsed during or within 6 months after completion of an anti-CD20 antibody maintenance therapy following at least 2 lines of therapy, or relapsed after autologous hematopoietic stem cell transplant (HSCT)

• The trial excluded patients with active or serious infections, transformed lymphoma or other aggressive lymphomas, prior allogeneic HSCT, or disease with active CNS involvement

• One week before infusion of KYMRIAH, all patients in the trial received one cycle of lymphodepleting (LD) chemotherapy. Patients could receive either fludarabine (25 mg/m²) and cyclophosphamide (250 mg/m²) IV daily for 3 days or bendamustine (90 mg/m²) IV daily for 2 days

• Bridging chemotherapy between leukapharesis and LD chemotherapy was permitted as needed

The primary end point of the ELARA trial was complete response rate by an independent review committee. Key secondary end points included overall response rate, duration of response, progression-free survival, overall survival, safety, and cellular kinetics.²

ELARA Baseline Characteristics

ELARA enrolled a broad range of adult patients with r/r FL, including high-risk patient subgroups²

• Most patients had bulky disease (64%), advanced-stage disease (86%), or a Follicular Lymphoma International Prognostic Index (FLIPI) score of ≥3 at study entry (60%)—all of which are risk factors for poor outcomes

• Patients in the trial had a median of 4 prior lines of therapy (range: 2 to 13)

• More than half of the patients had progression of disease within 24 months (POD24) of initiating their first anti-CD20 mAb-containing therapy

CD, cluster of differentiation; ECOG PS, Eastern Cooperative Oncology Group performance status; FL, follicular lymphoma; FLIPI, Follicular Lymphoma International Prognostic Index; HSCT, hematopoietic stem cell transplant; IQR, interquartile range; mAb, monoclonal antibody; PI3K, phosphatidylinositol 3-kinase; POD24, progression of disease within 24 months; r/r, relapsed/refractory.

^aRefractory is defined as failure to respond to previous treatment (stable disease/progressive disease as best response) or progressive disease within 6 months of prior therapy completion.
^bDouble refractory is defined as failure to respond or relapsed within 6 months following therapy with anti-CD20 and alkylating agents, any regimen.

Overall Response Rate

CR, complete response; ORR, overall response rate; PR, partial response.

^aThis included the first 90 patients with measurable disease who received KYMRIAH consecutively and had at least 9 months' follow-up from first objective response or discontinued earlier.^1
bTwo patients with best overall response of CR had their disease relapse more than 6 months after the last line of therapy.^1
cOf the 30 patients who initially achieved a PR, 14 patients (47%) converted to a CR, including 10 patients at the next subsequent visit and within 6 months post infusion.^1  
dCRR was the primary end point in the ELARA trial; ORR was a secondary end point.^4
eOne patient in CR downgraded to PR due to confirmatory bone marrow biopsy performed out of window.^4
fORR is defined as the proportion of patients with a best overall disease response of CR or PR.⁴

Duration of Response

ELARA Study (ASH 2022)

DOR was a secondary end point in the ELARA trial.⁴

Among patients whose best response was CR, 85% were estimated to remain in response at 12 months¹

For responders, median DOR was not reached in ELARA (USPI) with 9.1-month median follow-up for responders^1,g

^gThe first disease assessment was scheduled to be performed at Month 3 post infusion. The median follow up is the time from first objective response to last disease assessment.¹

Progression-Free Survival

ELARA Study (ASH 2022)

PFS was secondary end point in the ELARA trial.⁴

PFS data should be interpreted with caution in a single-arm trial as the statistical significance is unknown.

Overall Survival

Median follow-up of all patients (N=94): 29 months

OS was a secondary end point in the ELARA trial.²

Time to next anti-lymphoma treatment was an exploratory end point in the ELARA trial and should be interpreted with caution.⁴

OS data should be interpreted with caution in a single-arm trial as the statistical significance is unknown.

For information about the safety profile, continue to the next page