ENCOURAGING NEW DATA PRESENTED ON KITE’S YESCARTA (AXICABTAGENE CILOLECUEL) FOR RELAPSED/REFRACTORY CENTRAL NERVOUS SYSTEM LYMPHOMA

-- At Median Follow Up Of 24.2 Months, No Apparent Additional Risks of Adverse Events Related to Axicabtagene Ciloleucel Were Seen --

-- Findings Also Suggest Efficacy Trend with Median Progression-Free Survival and Durability of Response of More Than 12 months --

-- Data Presented Orally At The 2024 American Society Of Clinical Oncology Annual Meeting --

Stockley Park, UK – 3 June 2024 – Kite, a Gilead Company, announced data from a pilot study in collaboration with Dana-Farber Cancer Institute, that demonstrate Yescarta^® (axicabtagene ciloleucel) is well-tolerated in patients living with relapsed or refractory (R/R) primary or secondary central nervous system lymphoma (PCNSL and SCNSL).[i] The findings, which also showed efficacy benefits, were presented today in an oral session (Abstract #2006) at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.  

Central nervous system lymphoma (CNSL) is an aggressive and rare form of non-Hodgkin lymphoma that has either originated in (primary) or spread to (secondary) the brain, eye, spinal cord or cerebrospinal fluid. The prognosis of PCNSL has historically been poor with a five-year survival rate of only 30%.[ii] More than half of the patients experience a relapse after front-line treatment, with a median survival of approximately two months.ⁱⁱ R/R CNSL is considered an area of unmet clinical need with no standard of care treatment options. 

“Patients with central nervous system involvement of large B-cell lymphoma were excluded from the studies that led to the FDA approval of axi-cel in relapsed/refractory large B-cell lymphoma,” said Lakshmi Nayak, MD, Associate Professor of Neurology, Harvard Medical School and Director of the Center for CNS Lymphoma, Dana-Farber Cancer Institute. “We conducted this pilot study to evaluate the safety of axi-cel in CNSL patients and to see if there would be a preliminary signal of efficacy. Not only did we see a high response rate with axi-cel in this heavily pre-treated population, but they were durable. While we need more data to evaluate axi-cel in a larger study, these results are promising and represent a significant breakthrough in the treatment of this particularly rare and aggressive brain cancer for which there are limited options when it recurs.”

In this pilot study of 18 patients with CNSL, at a median follow-up of 24.2 months, no treatment-limiting toxicities and no apparent additional risk of adverse events were reported. Immune effector cell-associated neurologic syndrome (ICANS) were observed among 44% of patients (27.8% were Grade >3).ⁱ 

The objective response rate was 94.4% and the complete response rate was 66.7%.ⁱ The median time to best response was three months.ⁱ The median duration of response was 13.4 months and 9 patients had progressed.ⁱ At a median follow-up of 24 months, the median progression-free survival was 14.3 months (95% CI: 6.3-NR) and median overall survival was 26.4 months (95% CI: 11.2-NR).ⁱ 

Ninety percent of patients developed grade 1 or 2 cytokine release syndrome;ⁱ two patients developed Ommaya-related meningitis requiring explant with subsequent recovery.ⁱ One patient developed grade 3 electrographic focal status epilepticus that resolved with anti-epileptic agents.ⁱ Seven patients have died, all from disease progression.ⁱ

“We are very encouraged by the data presented today that indicate the potential to extend the benefits of axicabtagene ciloleucel to people with both primary and secondary central nervous system lymphoma, whose prognoses are typically very poor” said Ibrahim Elhoussieny, MD, Vice President, Medical Affairs, Kite. “These early data support that CAR T-cell therapy may potentially offer a treatment option to these patients.” 

About Central Nervous System Lymphoma  

Central nervous system lymphoma (CNSL) is an aggressive and rare form of non-Hodgkin lymphoma that has either originated in (primary) or spread (secondary) to the brain, eye, spinal cord or cerebrospinal fluid. The prognosis of PCNSL has historically been poor with a five-year survival rate of only 30%.ⁱⁱ More than half of the patients experience a relapse, after which the average survival is approximately two months.ⁱⁱ R/R CNSL is considered an area of unmet clinical need with no standard of care treatment options. 

CNS lymphoma is more common in men than women. The median age of diagnosis is 55. CNS lymphoma makes up only 4% of all tumours of the nervous system and only four to six percent of all non-Hodgkin lymphomas.[iii]^,[iv] PCNSL accounts for approximately 4% of newly diagnosed CNS tumors[v] and 1% to 2% of all NHLs.[vi]

About the Study 

The pilot study enrolled 18 patients (13 PCNSL, 4 SCNSL, 1 concurrent systemic and ocular lymphoma), of whom the first six patients were observed for treatment-limiting toxicities (TLTs). The primary endpoint was safety, measured by rate of TLTs and grade 3+ adverse events (AEs). Secondary endpoints included objective response rate, complete response rate, duration of response, progression-free survival and overall survival (OS).  

About Yescarta (Axicabtagene Ciloleucel)

Axicabtagene ciloleucel is a CD19-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy, approved by the European Commission (EC) for the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) who relapse within 12 months from completion of, or are refractory to, first-line chemoimmunotherapy; adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy; adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy.^[vii]

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, focused on cell therapy to treat and potentially cure cancer. As the global cell therapy leader, Kite has treated more patients with CAR T-cell therapy than any other company. Kite has the largest in-house cell therapy manufacturing network in the world, spanning process development, vector manufacturing, clinical trial production and commercial product manufacturing.

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. Gilead acquired Kite in 2017.

[i] Nayak L et al. A Pilot Study of Axicabtagene Ciloleucel (axi-cel) for the Treatment of Relapsed/Refractory Primary and Secondary Central Nervous System Lymphoma (PCNSL and SCNSL). Abstract #2006 presented at the American Society of Clinical Oncology (ASCO) Annual Meeting.

[ii] Green K, et al. Central nervous system lymphoma. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan. PMID: 31424729.

[iii] Löw S, Han CH, Batchelor TT. Primary central nervous system lymphoma. Ther Adv Neurol Disord. 2018 Oct 5;11:1756286418793562. doi: 10.1177/1756286418793562. PMID: 30305848; PMCID: PMC6174646.

[iv] Central Nervous System (CNS) Lymphoma. Memorial Sloan Kettering Cancer Center. Available at: https://www.mskcc.org/cancer-care/types/central-nervous-system-cns-lymphoma Accessed: April 2023

[v] Villano JL, Koshy M, Shaikh H, Dolecek TA, McCarthy BJ. Age, gender, and racial differences in incidence and survival in primary CNS lymphoma. Br J Cancer. 2011;105(9):1414-1418. doi:10.1038/bjc.2011.357.

[vi] Tang D, Chen Y, Shi Y, et al. Epidemiologic characteristics, prognostic factors, and treatment outcomes in primary central nervous system lymphoma: a SEER-based study. Front Oncol. 2022;12:817043. doi:10.3389/fonc.2022.817043.

[vii] European Medicines Agency. Yescarta^® (axicabtagene ciloleucel) SPC. Available at: https://www.ema.europa.eu/documents/product-information/yescarta-epar-product-information_en.pdf. Accessed May 2024.