YESCARTA® (AXICABTAGENE CILOLEUCEL) IS FIRST CAR T CELL THERAPY TO DEMONSTRATE HIGH RESPONSE RATES AND DURABLE CLINICAL BENEFIT IN A PIVOTAL INDOLENT NON-HODGKIN LYMPHOMA STUDY

-- 92% of Patients with Relapsed/Refractory Follicular Lymphoma or Marginal Zone Lymphoma Achieved a Response to Axicabtagene Ciloleucel, Including 76% with a Complete Response -- 
 
Stockley Park, UK – 5 December 2020 – Kite, a Gilead Company, today announced results from the primary analysis of ZUMA-5, a global, multicentre, single-arm, open-label Phase 2 study evaluating Yescarta® (axicabtagene ciloleucel) in adult patients with relapsed/refractory indolent non-Hodgkin lymphoma (iNHL) after at least two prior lines of therapy. After a single infusion of axicabtagene ciloleucel, 92% of iNHL patients (n=104 evaluable for efficacy) responded, including 76% of patients achieving a complete response (CR) at a median follow-up of 17.5 months. The data were presented in an oral session during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract #700).¹ The presentation is also being considered for Best of ASH and has been selected for inclusion in the ASH Annual Meeting Press Program on December 5 at 12:30 p.m. ET. 

Axicabtagene ciloleucel has not been approved by any regulatory agency for the treatment of indolent NHL, including Follicular lymphoma (FL) or marginal zone lymphoma (MZL). The safety and efficacy have not been established in these types of lymphomas. 

“It is encouraging to see this level of response to CAR T cell therapy in a heavily pretreated and multiple refractory/relapsed patient population, in whom response duration to other available therapies is expected to be short,” said Caron A. Jacobson, MD, MMSc, Medical Director, Immune Effector Cell Therapy Program, Dana-Farber Cancer Institute and Assistant Professor of Medicine, Harvard Medical School. “Our goal with treatment is to prolong overall survival, and the durability following treatment with  axicabtagene ciloleucel is promising for relapsed/refractory patients, who are often at a higher risk for early progression.” 
 
94% of patients with relapsed/refractory FL (n=84) responded to axicabtagene ciloleucel, including 80% of patients achieving a CR and 64% of patients in an ongoing response at a median follow-up of 17.5 months. Of patients with relapsed/refractory MZL (n=20), 85% responded to axicabtagene ciloleucel, with 60% achieving a CR. At the data cutoff, 62% of all treated patients had ongoing responses. Median duration of response (DOR), progression-free survival (PFS) and overall survival (OS) were not reached. 

In the safety analysis (n=146), Grade 3 or higher CRS and neurologic events (NEs) occurred in 7% and 19% of patients, respectively. Lower incidence of Grade 3 or higher NEs was observed in patients with FL (15%) compared to MZL (41%), and CRS rates were comparable between the two groups. There were three Grade 5 adverse events, including two FL patients, one with multisystem organ failure in the context of CRS related to treatment with axicabtagene ciloleucel and one with aortic dissection unrelated to axicabtagene ciloleucel treatment. Coccidioidomycosis infection occurred in a patient with MZL unrelated to axicabtagene ciloleucel treatment. 
 

About Indolent Non-Hodgkin Lymphoma

Follicular lymphoma (FL) and marginal zone lymphoma (MZL) are both forms of indolent non-Hodgkin lymphoma (NHL) in which malignant tumours slowly grow but can become more aggressive over time. 
 
FL is the most common form of indolent lymphoma and the second most common type of lymphoma globally.^{2 3} It accounts for approximately 22% of all lymphomas diagnosed worldwide.⁴ MZL is the third most common lymphoma, accounting for 8% of all B cell NHLs.⁴ 
 
Despite advances in management and substantial improvements in long-term survival, patients living with FL have varied outcomes. Currently, there are limited options for the treatment of relapsed/refractory FL and MZL after two or more lines of therapy. 
 

About ZUMA-5

ZUMA-5 is a single-arm, multicentre, open-label Phase 2 study that aims to enrol patients (≥18 years old) with relapsed/refractory iNHL of either follicular lymphoma (FL) or marginal zone lymphoma (MZL) subtypes, who received at least two prior lines of systemic therapy, including an anti-CD20 monoclonal antibody combined with an alkylating agent. The objectives of the study are to evaluate the efficacy and safety of a single infusion of axicabtagene ciloleucel in this patient population. The primary endpoint of the trial is objective response rate (ORR) as assessed by an independent review committee per the 2014 Lugano Classification. Secondary endpoints include CR rate, DOR, PFS, OS, safety and CAR T cell and cytokines levels. The study is ongoing. 
 

About Axicabtagene Ciloleucel

Axicabtagene ciloleucel is a CD19-directed genetically modified autologous T cell immunotherapy. In August 2018, axicabtagene ciloleucel, a chimeric antigen receptor (CAR) T cell therapy, received European Marketing Authorisation for the treatment of adult patients with relapsed/refractory diffuse large B cell lymphoma (DLBCL) and primary mediastinal large B cell lymphoma (PMBCL), after two or more lines of systemic therapy.⁵
 

About Kite

Kite, a Gilead Company, is a biopharmaceutical company based in Santa Monica, California, with commercial manufacturing operations in North America and Europe. Kite is engaged in the development of innovative cancer immunotherapies. The company is focused on chimeric antigen receptor and T cell receptor engineered cell therapies.  
 

About Gilead Sciences

Gilead Sciences, Inc. is a research-based biopharmaceutical company that discovers, develops and commercialises innovative medicines in areas of unmet medical need. The company strives to transform and simplify care for people with life-threatening illnesses around the world. Gilead has operations in more than 35 countries worldwide, with headquarters in Foster City, California.  
 

Forward-Looking Statement

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 risk that physicians and patients may not see the potential benefits of Yescarta therapy and the possibility of unfavorable results from other ongoing and additional clinical studies involving Yescarta. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. The reader is cautioned not to rely on these forward-looking statements. These and other risks are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, as filed with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation to update any such forward-looking statements. 
 
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Kite, the Kite logo, Yescarta and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies. 

¹ Jacobson, et al. Primary Analysis of ZUMA-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients With Relapsed/Refractory (R/R) Indolent Non-Hodgkin Lymphoma (iNHL). Abstract #700 (Oral) presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.

 ² Ciobanu A, Stanca O, Triantafyllidis I, Lupu A. Indolent lymphoma: diagnosis and prognosis in medical practice. Maedica (Buchar). 2013;8(4):338–342.

 ³ A clinical evaluation of the International Lymphoma Study Group classification of non-Hodgkin's lymphoma. The Non-Hodgkin's Lymphoma Classification Project., Blood, 1997, vol. 89 (pg. 3909-3918).

⁴ Leukemia & Lymphoma Society. NHL subtypes. Available at: https://www.lls.org/lymphoma/non-hodgkinlymphoma/diagnosis/nhl-subtypes. Accessed: December 2020.

⁵  European Medicines Agency. Yescarta® (axicabtagene ciloleucel) SmPC. Available at: https://www.ema.europa.eu/documents/product-information/yescarta-epar-product-information_en.pdf. Accessed December 2020. 

CONTACTS:  

Douglas Maffei, PhD, Investors +1 (650) 522-2739

Nathan Kaiser, Media +1 (650) 522-1853  

Cressida Robson, Media (Europe) +44 7341 789 204 cressida.robson@kitepharma.com 

IHQ-ONC-2020-11-0004 December 2020