On July 12, 2016 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, reported plans for a Phase I adoptive cellular therapy clinical trial utilizing autologous T cells transduced with lentivirus to express a CD33-specific chimeric antigen receptor (CAR) in patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Ziopharm, JUL 12, 2016, View Source [SID:1234513844]). The upcoming trial will be the second initiated at The University of Texas MD Anderson Cancer Center under the research and development agreement among ZIOPHARM, Intrexon Corporation (NYSE:XON), and MD Anderson to expeditiously move promising treatments from the bench to the clinic.

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"By targeting myeloid cells with a CD33-specific CAR, we have the opportunity to leverage T-cell mediated cytotoxicity in a highly directed manner," said Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM. Dr. Lebel continued, "Following a recent review by the Recombinant DNA Advisory Committee, we anticipate progressing plans for this Phase I trial in the second half of 2016 to move this promising treatment closer to the clinic for patients with relapsed or refractory AML who have poor prognosis."

The American Cancer Society estimates that there will be approximately 20,000 new cases of AML and over 10,000 patient deaths from AML in the United States in 2016. AML is a rapidly progressing cancer of the blood and bone marrow characterized by uncontrolled proliferation of immature blast cells with multiple associated gene mutations. A majority of AML patients relapse or present with refractory disease and have overall poor prognosis.

Preclinical studies demonstrated that lentiviral transduced CAR-T cells targeting CD33 exhibit specific cytotoxic activity for CD33+ AML cells. A proof-of-concept study utilizing an in vivo mouse model for AML showed that these CAR-T cells were able to eliminate disease burden and significantly enhance survival as compared to control groups. These positive preliminary results indicate biological activity and are suggestive of potential therapeutic effect for the treatment of AML.

Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM Oncology commented, "As we progress through the remainder of 2016 with our Intrexon Corporation and MD Anderson collaborators, we believe our clinical stage assets and promising preclinical pipeline will position the company at the forefront of those utilizing the immune system in a controlled manner to combat cancer." Dr. Cooper added, "We believe that our CD33 CAR-T cell approach has the potential to positively impact a disease area that has been largely unexplored with this type of immune-therapy and overall this malignancy has seen inadequate improvement in treatment options. We look forward to moving into the clinic as soon as possible to help patients with advanced AML urgently in need of new therapies."

This planned Phase I trial will add to ZIOPHARM’s three active trials at leading institutions, two employing controlled gene therapy to express a powerful anti-cancer effector in interleukin-12 and the other incorporating the innovative non-viral Sleeping Beauty gene delivery system in CAR-T cell therapy for advanced lymphoid malignancies. This new program targeting CD33 is part of the Exclusive Channel Collaboration with Intrexon and is the next CAR-T target the companies are pursuing in addition to the first two targets already selected by the biopharmaceutical division of Merck KGaA.

ZIOPHARM intends to further expand its clinical programs in the area of natural killer (NK) cells and a combination approach to its Ad-RTS-hlL-12 gene therapy using an immune checkpoint inhibitor through the remainder of 2016. Additionally, ZIOPHARM and Intrexon continue to advance preclinical efforts leveraging the Sleeping Beauty platform, the clinical-stage RheoSwitch (RTS), and improving cell manufacturing for the clinical applications of T cells engineered to express CAR and TCR, as well as off-the-shelf approaches.