TRANSCEND CLL 004 is the first pivotal multicenter trial to evaluate a CAR T cell therapy in heavily pre-treated patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma

Results from the primary analysis show 18.4% of patients treated with Breyanzi achieved a complete response (CR) and median duration of CR not reached at median follow-up of 21.1 months

Breyanzi showed a manageable safety profile, with no new safety signals, in this patient population with relapsed or refractory disease after treatment with a BTK inhibitor (BTKi) and BCL-2 inhibitor (BCL2i)

PRINCETON, NJ, USA I May 25, 2023 IBristol Myers Squibb (NYSE: BMY) today announced the first disclosure of results from the primary analysis of the pivotal TRANSCEND CLL 004 study, a Phase 1/2, open-label, single-arm multicenter study evaluating Breyanzi (lisocabtagene maraleucel, liso-cel) in adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). At a median follow-up of 21.1 months, results show that Breyanzi delivered statistically significant complete response (CR) rates, the study’s primary endpoint, in 18.4% of patients in the primary efficacy analysis set (95% CI: 8.8-32; p=0.0006). Among patients who achieved a CR, no disease progression or deaths were observed, with median duration of response not reached.

TRANSCEND CLL 004 is the first pivotal multicenter study of a CD19-directed CAR T cell therapy for patients with relapsed or refractory CLL after progression on a BTKi and BCL2i. These data will be presented in an oral presentation during the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting on June 6, 10:45 a.m. EDT (Abstract #7501).

There is a critical unmet need for patients with relapsed or refractory CLL or SLL, especially those who have experienced disease progression after treatment with a BTKi and BCL2i. These patients often have high-risk disease features and poor outcomes, with short overall survival. Current treatment options rarely provide complete responses, and durability of response is limited.

“For people living with relapsed or refractory CLL or SLL after treatment with BTKi and BCL2i-based regimens, there is no standard of care treatment. Achieving deep and lasting remission in this situation is challenging as most patients experience disease progression despite continuous treatment,” said Tanya Siddiqi, M.D., lead investigator and Associate Professor, Division of Lymphoma, City of Hope National Medical Center. “The durable complete responses observed with liso-cel in the TRANSCEND CLL 004 trial are remarkable and represent a major step in bringing a personalized, T-cell-based treatment approach delivered as a one-time infusion into clinical practice for a complex and historically incurable disease.”

“Results from TRANSCEND CLL 004 reinforce our relentless commitment to bringing the potential of CAR T cell therapy to more patients and transforming the treatment and outcomes for a broad range of hematologic malignancies,” said Anne Kerber, senior vice president, head of Cell Therapy Development, Bristol Myers Squibb. “Breyanzi has shown clinically meaningful benefit across the broadest array of B-cell malignancies of any CD19-directed CAR T cell therapy and we remain dedicated to advancing innovative treatments for some of the most difficult-to-treat diseases with high unmet need.”

The TRANSCEND CLL 004 trial included a broad population of patients with relapsed or refractory CLL or SLL with high unmet need who had received at least two prior lines of therapy, including a BTKi (n=117). The prespecified primary efficacy analysis set (PEAS; n=49) consisted of a subset of patients who had experienced disease progression following treatment with a BTKi and failure of BCL2i-based regimens, representing a patient population with advanced and aggressive disease, and who were treated with the target dose of 100 x 106 CAR-positive viable T-cells of Breyanzi. High rates of undetectable minimal residual disease (uMRD) were observed across patients treated with Breyanzi, with a uMRD rate 63.3% in the blood (95% CI: 48.3-76.6) and 59.2% in the bone marrow (95% CI: 44.2-73.0), which was associated with an increase in progression-free survival. The overall response rate (ORR) was 42.9% (95% CI: 28.8-57.8; p=0.3931), with a median duration of response of 35.3 months (11.01-NR). Data were consistent between the PEAS and the broad patient population evaluated in the study, including heavily pretreated patients with a median of five prior lines of therapy (2 – 12) and high-risk disease, with a CR rate of 18.4% (95% CI: 10.9-28.1), demonstrating the clinical benefit of Breyanzi for a broad patient population with relapsed or refractory CLL or SLL.

Among all treated patients in the study (n=117), including subgroups of heavily pretreated patients, Breyanzi exhibited a manageable safety profile, and no new safety signals were observed. Any grade cytokine release syndrome (CRS) occurred in 84.6% of patients, with Grade 3 CRS occurring in 8.5% of patients. No Grade 4/5 CRS events were reported. Any grade neurologic events (NE) were reported in 45.3% of patients, with Grade 3 NE reported in 17.9% of patients and one case (0.9%) of Grade 4 NE reported. No Grade 5 NE were reported.

Results from TRANSCEND CLL 004 will be discussed with health authorities. Bristol Myers Squibb thanks the patients and investigators involved in the TRANSCEND CLL 004 trial.

TRANSCEND CLL 004 (NCT03331198) is a Phase 1/2 open-label, multicenter study evaluating Breyanzi in patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma. The Phase 1 dose escalation portion of the study assessed the safety and recommended dose for the subsequent Phase 2 expansion cohort. The Phase 2 portion of the study is evaluating Breyanzi at the recommended dose from the Phase 1 monotherapy arm. The primary endpoint of the Phase 2 portion of the study was complete response rate, including complete remission with incomplete bone marrow recovery, based on independent review committee according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) 2018 guidelines.

About CLL and SLL
Chronic lymphocytic leukemia (CLL) is one of the most common types of leukemia in adults. In CLL, too many blood stem cells in the bone marrow become abnormal lymphocytes, and these abnormal cells have difficulty fighting infections. As the number of abnormal cells grows, there is less room for healthy white blood cells, red blood cells and platelets. Small lymphocytic lymphoma (SLL) also affects the lymphocytes, with cancer cells found mostly in the lymph nodes. While there are several treatments available for CLL and SLL, there is no standard of care for relapsed or refractory CLL or SLL after prior therapy with targeted agents, which raises the need for additional effective therapies. Patients with relapsed or refractory disease have limited treatment options and generally experience shorter periods of response with each subsequent treatment.

About Breyanzi
Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells. Breyanzi is made from a patient’s own T cells, which are collected and genetically reengineered to become CAR T cells that are then delivered via infusion as a one-time treatment. Breyanzi is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with LBCL, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal LBCL, and follicular lymphoma grade 3B who have refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy, or refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplant due to comorbidities or age, or relapsed or refractory disease after two or more lines of systemic therapy. Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.

Please see the Important Safety Information section below, including Boxed WARNINGS for Breyanzi regarding cytokine release syndrome and neurotoxicity.

Breyanzi is also approved in Japan and Europe for the second-line treatment of relapsed or refractory LBCL, and in Japan, Europe, Switzerland, and Canada for relapsed and refractory LBCL after two or more lines of systemic therapy. Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in earlier lines of treatment for patients with relapsed or refractory LBCL and other types of lymphoma and leukemia. For more information, visit

Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.

Bristol Myers Squibb: Creating a Better Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision—transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

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About Bristol Myers Squibb
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SOURCE: Bristol Myers Squibb