KITE’S CAR T-CELL THERAPY TECARTUS® (BREXUCABTAGENE AUTOLEUCEL) GRANTED EUROPEAN MARKETING AUTHORISATION FOR THE TREATMENT OF RELAPSED OR REFRACTORY ACUTE LYMPHOBLASTIC LEUKAEMIA (r/r ALL)

KITE’S  CAR  T-CELL  THERAPY  TECARTUS^®  (BREXUCABTAGENE  AUTOLEUCEL)  GRANTED EUROPEAN MARKETING AUTHORISATION FOR THE TREATMENT OF  RELAPSED  OR  REFRACTORY  ACUTE  LYMPHOBLASTIC  LEUKAEMIA  (r/r ALL)

-- Approval Marks Kite’s Fourth Indication in Europe for its Two Cell Therapies and First in Leukaemia --

-- Overall Complete Remission of 71% and a Median Overall Survival of More Than Two Years for All Patients and Almost Four Years for Responders Demonstrated in ZUMA-3 Trial --

Stockley Park, UK – 7 September 2022 – Kite, a Gilead Company, today announced that the European Commission (EC) has approved its CAR T-cell therapy Tecartus^® (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukaemia (ALL).

“This approval makes brexucabtagene autoleucel the first and only CAR T-cell therapy indicated for this population of patients, addressing a significant unmet medical need,” said Christi Shaw, CEO, Kite. “This is also the fourth indication in Europe for which a Kite cell therapy is approved, clearly demonstrating the benefits they offer to patients, especially those with limited treatment options.”

ALL is an aggressive type of blood cancer; the most common form is B cell precursor ALL.[1] Globally, approximately 64,000 people are diagnosed with ALL each year.[2]^,[3]  Half of adults living with ALL will relapse, and median overall survival (OS) with current standard-of-care treatments is approximately just eight months.[4]^,[5]

“Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy and stem cell transplant, creating a significant burden on a patient’s quality of life,” said Max S. Topp, MD, professor and head of Hematology, University Hospital of Wuerzburg, Germany. “Patients in Europe now have a meaningful advancement in treatment. Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.”

The approval is supported by data from the ZUMA-3 international multicentre, single-arm, open-label, registrational Phase 1/2 study of adult patients (≥18 years old) with relapsed or refractory ALL. This study demonstrated that 71% of the evaluable patients (n=55) achieved complete remission (CR) or CR with incomplete haematological recovery (CRi) with a median follow-up of 26.8 months.[6] In an extended data set of all pivotal dosed patients (n=78) the median overall survival for all patients was more than two years (25.4 months) and almost four years (47 months) for responders (patients who achieved CR or CRi).^6,[7] Among efficacy-evaluable patients, median duration of remission (DOR) was 18.6 months.⁶

Among the patients treated with brexucabtagene autoleucel at the target dose (n=100) safety results were consistent with the known safety profile for brexucabtagene autoleucel. Grade 3 or higher cytokine release syndrome (CRS) and neurologic adverse reactions occurred in 25% and 32% of patients, respectively, and were generally well managed. ^[8],[9]^,[10] 

About ZUMA-3 

ZUMA-3 is an ongoing international multicentre (US, Canada, EU), single arm, open label, registrational Phase 1/2 study of brexucabtagene autoleucel in adult patients (≥18 years old) with ALL whose disease is refractory to or has relapsed following standard systemic therapy or hematopoietic stem cell transplantation. The primary endpoint is the rate of overall complete remission or complete remission with incomplete haematological recovery by central assessment. Duration of remission and relapse-free survival, overall survival, minimal residual disease (MRD) negativity rate, and allo-SCT rate were assessed as secondary endpoints.

About Acute Lymphoblastic Leukaemia 
ALL is an aggressive type of blood cancer that develops when abnormal white blood cells accumulate in the bone marrow until there isn’t any room left for blood cells to form.[11] In some cases, these abnormal cells invade healthy organs and can also involve the lymph nodes, spleen, liver, central nervous system and other organs.[12] 

About Tecartus^® (Brexucabtagene Autoleucel)

Tecartus^® (brexucabtagene autoleucel) is an autologous anti-CD19 directed chimeric antigen receptor (CAR) T-cell therapy, an individualised method of treatment that harnesses the body’s own immune system to target cancer cells.⁷ In December 2020, the European Commission granted conditional Marketing Authorisation for brexucabtagene autoleucel, the first CAR T-cell therapy approved in Europe for adult patients with relapsed or refractory mantle cell lymphoma after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor.⁷

About Kite  

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with 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.  

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.  

Forward-Looking Statements  

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Kite and Gilead to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving brexucabtagene autoleucel; the risk that physicians may not see the benefits of prescribing brexucabtagene autoleucel for the treatment of blood cancers; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation and disclaim any intent to update any such forward-looking statements. 

[1] Leukaemia Care. B-cell Acute Lymphoblastic Leukaemia (ALL). Available at: https://media.leukaemiacare.org.uk/wp-content/uploads/B-cell-Acute-Lymphoblastic-Leukaemia-ALLWeb-Version.pdf. Accessed September 2022.

[2] Dong, Y, et al. Leukemia incidence trends at the global, regional, and national level between 1990 and 2017. Exp Hematol Oncol 9, 14 (2020). https://doi.org/10.1186/s40164-020-00170-6. Accessed September 2022.

[3] Leukemia and lymphoma Society Canada. Facts and Statistics. About Blood Cancers. Available at: https://www.llscanada.org/disease-information/facts-and-statistics#Leukemia. Accessed September 2022.

[4] Kantarjian H, et al. Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia. N Engl J Med (2017) 376: 836-847.

[5] Kantarjian H, et al. Inotuzumab Ozogamicin Versus Standard of Care in Relapsed or Refractory Acute Lymphoblastic Leukemia: Final Report and Long-Term Survival Follow-Up From the Randomized, Phase 3 INO-VATE Study. Cancer (2019) 125(14):2474-2487.

[6] Bijal D, et al. Two-year follow-up of KTE-X19, an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, in adult patients (Pts) with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) in ZUMA-3. Journal of Clinical Oncology (2022) 40:16_suppl, 7010-7010.

[7] Data on file. Figure 14.2.10.8.9. Kaplan-Meier Plot of Overall Survival with CR+CRi vs. Others using Independent Review (Phase 1, 1e6 Dose Level and Phase 2, Safety Analysis Set)

[8] Data on file. Table 14.3.2.31.8. Ad-hoc: Subject Incidence of Treatment-emergent Cytokine Release Syndrome Adverse Events by Preferred Term and Worst Grade (ZUMA-2 and ZUMA-3) (Safety Analysis Set)

[9] Data on file. Table 14.3.2.31.10. Ad-hoc: Subject Incidence of Treatment-emergent Neurologic Events by Preferred Term and Worst Grade - Method 1 (ZUMA-2 and ZUMA-3) (Safety Analysis Set)

[10] European Medicines Agency. Tecartus® (autologous anti-CD19-transduced CD3+ cells) SPC. Available at: https://www.ema.europa.eu/en/documents/product-information/tecartus-epar-product-information_en.pdf.  Accessed September 2022.

[11] NHS. Acute lymphoblastic leukaemia (ALL). Available at: https://www.nhs.uk/conditions/acute-lymphoblastic-leukaemia/. Accessed September 2022.

[12] Leukaemia Care. B-cell Acute Lymphoblastic Leukaemia (ALL). Available at: https://media.leukaemiacare.org.uk/wp-content/uploads/B-cell-Acute-Lymphoblastic-Leukaemia-ALLWeb-Version.pdf. Accessed September 2022.