Axicabtagene ciloleucel is under clinical development by Gilead Sciences and currently in Phase II for Refractory Acute Myeloid Leukemia. According to GlobalData, Phase II drugs for Refractory Acute Myeloid Leukemia have a 21% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Axicabtagene ciloleucel’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Axicabtagene ciloleucel overview

Axicabtagene ciloleucel (KTE-C19 / Yescarta / Yikaida) is human culture expanded genetically modified autologous T cells for cell-based gene therapy. Cells are derived from isolated blood of the patient and are transduced with non-replicative retroviral vector encoding the FMC63 anti-CD19 single chain variable fragment (scFv) CD28/CD3zeta chimeric antigen receptor (FMC63-28Z CAR). It is formulated as suspension for intravenous route of administration. It is indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. It is also indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. Yescarta is indicated for adult patients with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or relapses within 12 months of first-line chemoimmunotherapy.

It is under development for the treatment of solid tumors, indolent non-Hodgkin lymphoma, diffuse large B cell lymphoma (DLBCL), lymph node and extranodal marginal zone B-cell lymphoma, primary mediastinal B-cell lymphoma(PMBCL), mantle cell lymphoma, recurrent chronic lymphocytic leukemia, refractory chronic lymphocytic leukemia, acute lymphocytic leukemia, burkitt lymphoma, refractory acute myeloid leukemia, relapsed/refractory (R/R) primary and secondary central nervous system lymphoma, follicular lymphoma,  high grade B-cell lymphoma and large cell lymphoma. It was also under development for the treatment acute lymphoblastic leukemia and mantle cell lymphoma.

Gilead Sciences overview

Gilead Sciences (Gilead) is a research-based biopharmaceutical company. It carries out the discovery, development and commercialization of medicines for the treatment of cardiovascular, hematological and respiratory diseases, inflammation, liver diseases such as hepatitis B virus (HBV) and hepatitis C virus (HCV) infections, cancer, and human immunodeficiency virus (HIV) infection. It markets its products through commercial teams, third-party distributors and corporate partners. Gilead’s commercial teams promote its products through direct field contact with physicians, hospitals, clinics and other healthcare providers. The company sells its products through subsidiaries and distributors in Europe, the Americas, Asia-Pacific, the Middle East, and Africa. Gilead is headquartered in Foster City, California, the US.

For a complete picture of Axicabtagene ciloleucel’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.