Kite's Tecartus® Demonstrates High Response Rate in Adults With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia Earning Priority Review Designation

-- 71% of Adult Patients in Phase 2 ZUMA-3 Study Achieved a Complete Response Following a Single Infusion of Tecartus --

-- Findings Support Recent Supplemental Biologics License Application (sBLA) for Tecartus Accepted for Priority Review by the U.S. Food & Drug Administration (FDA) --

-- If Approved, Tecartus Would Be the First and Only CAR T-Cell Therapy Approved for Adult Patients (18 Years and Older) with Relapsed or Refractory B-cell Precursor Acute Lymphoblastic Leukemia --

SANTA MONICA, CA, USA I June 4, 2021 I Kite, a Gilead Company (Nasdaq: GILD), announced today results from the primary analysis of ZUMA-3, a global, multicenter, single-arm, open-label Phase 1/2 study evaluating its chimeric antigen receptor (CAR) T-cell therapy Tecartus® (brexucabtagene autoleucel) in adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The data were simultaneously published in The Lancet and presented during an oral session at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting from June 4 – 8 (Abstract #7002).

“Outcomes in adults with acute lymphoblastic leukemia are poor relative to what is observed in children, with less than half of people over 20 years of age expected to survive the illness. It is on this background that CAR T-cell therapy with brexucabtagene autoleucel was tested in adults with relapsed B-ALL in ZUMA-3,” said Bijal Shah, MD, ZUMA-3 investigator and medical oncologist, Moffitt Cancer Center, Tampa, Florida. “In this international, multicenter study, we observed a response rate of 71%. Importantly, the majority of these responses were associated with undetectable minimal residual disease.”

In the pivotal Phase 2 portion of the trial, 71 patients with relapsed or refractory disease were enrolled. Among treated patients (n=55), 47% had received three or more prior therapies. At a median follow-up of 16.4 months, 71% of treated patients achieved a complete response (CR) or CR with incomplete hematological recovery (CRi), with 31% in ongoing response at data cut-off. 97% of those responders had deep molecular remission, with undetectable minimal residual disease (MRD), and median overall survival (OS) among all responders was not reached. Among 25 patients with prior blinatumomab treatment, the CR/CRi rate was 60%. Among all treated patients, median duration of response (DOR), relapse-free survival (RFS), and OS were 12.8 months, 11.6 months and 18.2 months, respectively.

Grade ≥3 adverse events occurred in 95% of patients, with anemia (49%) and pyrexia (36%) most frequently reported. Grade ≥3 cytokine release syndrome (CRS) and neurologic events occurred in 24% and 25% of patients, respectively, and were generally reversed with treatment. Two Grade 5 treatment-related events occurred (one brain herniation and one case of septic shock).

Based on these data, the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) and granted Priority Review designation for Tecartus for the treatment of adult patients with relapsed or refractory B-cell precursor ALL, with a target action date under the Prescription Drug User Fee Act (PDUFA) of October 1, 2021. If approved, Tecartus would become the first and only CAR T-cell therapy approved for adults (≥18 years old) with relapsed or refractory ALL.

“The data presented at ASCO today validate the response rates seen in the Phase 1 portion of the ZUMA-3 study and the transformative potential of Tecartus in adult patients with ALL,” said Frank Neumann, MD, PhD, Kite’s Global Head of Clinical Development. “We have already seen the impact of Tecartus for patients with relapsed or refractory mantle cell lymphoma, and these new data are a significant next step in our continued commitment in developing our therapies for patients with leukemias and lymphomas.”

In 2016, Tecartus received Breakthrough Therapy Designation in recognition of the unmet medical need in adult patients with relapsed or refractory B-cell precursor ALL. Tecartus is currently approved for the treatment of relapsed or refractory mantle cell lymphoma, as the first and only CAR T-cell therapy to receive accelerated approval from the FDA in this indication. The Tecartus U.S. Prescribing Information has a Boxed Warning in its product label regarding the risks of cytokine release syndrome (CRS) and neurologic toxicities, and Tecartus is approved with a risk evaluation and mitigation strategy (REMS) due to these risks; see below for Indication and Important Safety Information.

Tecartus has not been approved by any regulatory agency for the treatment of adult patients with relapsed or refractory ALL. Its safety and efficacy are currently under review by the FDA for this indication.

About ALL
ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system and other organs. Approximately 1,030 adults are treated annually for relapsed or refractory ALL. Survival rates remain very poor in adult patients with relapsed or refractory ALL, with a median overall survival of approximately eight months with the most commonly used therapeutic agents.

B-cell precursor ALL is the most common form of the disease, accounting for approximately 75 percent of cases. Treatment for this form is typically associated with inferior outcomes compared with other types of ALL.

About ZUMA-3
ZUMA-3 is an ongoing international multicenter, registrational Phase 1/2 study in adult patients (≥18 years old) with ALL whose disease is refractory to or has relapsed following first standard systemic therapy with remission of 12 months or less, after two or more lines of systemic therapy or after allogeneic stem cell transplantation. The objectives of the study are to evaluate the safety and efficacy of Tecartus in this patient population.

About Tecartus
Tecartus is an autologous, anti-CD19 CAR T-cell therapy. Tecartus uses the XLP manufacturing process that includes T cell enrichment, a necessary step in certain B-cell malignancies in which circulating lymphoblasts are a common feature. In addition to adult ALL, Tecartus is also currently being evaluated in pediatric ALL. The use of Tecartus in both cancer types is investigational, and its safety and efficacy have not been established in these cancer types.

Tecartus Indication
Tecartus is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL).

This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About Kite
Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with commercial manufacturing operations in North America and Europe. Kite’s singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit

About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

SOURCE: Kite Pharma

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