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August 8, 2017

Kite Pharma Submits IND for BCMA-Targeting CAR-T Cancer Immunotherapy

  • Kite Pharma said today it has submitted to the FDA an Investigational New Drug (IND) application to launch a Phase I, first-in-human trial of a chimeric antigen receptor T-cell (CAR-T) therapy that targets B-cell maturation antigen (BCMA) in patients with relapsed/refractory multiple myeloma.

    KITE-585 is designed to work through the engineering of a patient's T cells to express a CAR  that targets BCMA, a protein expressed on the cell surface of multiple myelomas, then redirecting those T cells to kill cancer cells. KITE-585 contains a receptor derived from a fully human monoclonal antibody and a CD28 co-stimulatory domain intended for optimized T-cell expansion and function.

    Kite disclosed its intent to advance KITE-585 into the clinic in April, when it presented preclinical data on the CAR-T therapy at the American Association of Cancer Research (AACR) Annual Meeting in Washington D.C.

    In one study—Abstract #4979, “Development of KITE-585: A fully human anti-BCMA CAR T-cell therapy for the treatment of multiple myeloma”—KITE-585 showed what the company termed potent activity against multiple myeloma (MM) cell lines, both in vitro and in vivo. CAR T cells were active in the presence of soluble BCMA and also eradicated established multiple myeloma tumors in mice.

    KITE-585 polyfunctional activation and proliferation of T-cells in the presence of MM cell lines, with no evidence of tonic signaling in the absence of target cells, since the therapy contains a proprietary linker with the CD28 co-stimulatory domain.

    In a poster presentation at AACR—Abstract #2135, “Selectivity and specificity of engineered T cells expressing KITE-585, a chimeric antigen receptor targeting B-cell maturation antigen (BCMA)”— KITE-585 showed specificity for BCMA-expressing target cells due to the selectivity of its novel single-chain variable fragment (scFv) for BCMA, following an assessment using Retrogenix™ cell microarray technology.

  • Third CAR-T Candidate Advancing to Clinic

    KITE-585 is Kite’s third CAR-T cancer immunotherapy candidate to advance into the clinic and beyond. The company in March completed its Biologics License Application seeking FDA approval for its lead CAR-T product axicabtagene ciloleucel (formerly KTE-C19) in relapsed or refractory aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT).

    The FDA in May designated axicabtagene ciloleucel for Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of November 29, 2017. While Kite acknowledged the death of a patient in an axicabtagene ciloleucel trial in April, the company added that the patient was in poor health with an aggressive form of NHL before the onset of treatment.

    Last month, Kite submitted the first CAR-T application in Europe, a marketing authorization application to the European Medicines Agency seeking approval of axicabtagene ciloleucel for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are ineligible for ASCT.

    Also in Kite’s CAR-T pipeline is a fully human anti-CD19 CAR-based product candidate directed against B-cell malignancies. Last year, the NIH’s National Cancer Institute (NCI), began a Phase I clinical trial of the product candidate in patients with B-cell malignancies under an existing Cooperative Research and Development Agreement (CRADA) between Kite and the NCI. James N. Kochenderfer, M.D., an investigator in the Institute’s Experimental Transplantation and Immunology Branch, is leading the study.

    “KITE-585 has the potential to become Kite's next significant advance in cell therapy for patients with cancer,” David Chang, M.D., Ph.D., Kite’s evp of R&D and CMO, said in a statement.

    Kite and Novartis have emerged as leading developers of CAR-T cancer immunotherapies, with both seeking the first FDA approval of a CAR-T treatment.  

    Last month, Novartis won an FDA advisory committee’s unanimous recommendation of approval for Novartis’ leukemia-fighting treatment CTL019 (tisagenlecleucel), a CAR-T therapy developed through a collaboration between the pharma giant and researchers from the University of Pennsylvania launched in 2012. CTL019 is indicated for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).

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