Ziopharm said the collaboration with the vaunted cancer research center will use non-viral gene transfer to stably express and clinically evaluate neoantigen-specific T-cell receptors in T cells.
Four months after Boston-based ZIOPHARM Oncology received regulatory clearance to move its Sleeping Beauty CAR-T platform program forward in solid tumors, the company forged a research and development agreement with MD Anderson based on that platform.
This morning, Ziopharm said the collaboration with the vaunted cancer research center will use non-viral gene transfer to stably express and clinically evaluate neoantigen-specific T-cell receptors (TCRs) in T cells. The partnership with MD Anderson will deepen an existing relationship the company already had with the cancer center. In 2015, the groups established a relationship related to a CD19-specific CAR-T. Earlier this month, the company said, the U.S. Food and Drug Administration cleared an Investigational New Drug Application for a Phase I clinical trial to evaluate CD19-specific CAR-T, manufactured and infused within two days of gene transfer using Ziopharm’s rapid personalized manufacture in patients with relapsed CD19+ leukemias and lymphomas.
According to Ziopharm, “DNA from the Sleeping Beauty system is stably inserted into the genome of resting T cells to co-express a chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch, which is designed to reduce cost, simplify production, and preserve the therapeutic potential of the T cells.” The Phase I trial announced weeks ago will enroll up to 24 patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT. The Sleeping Beauty platform genetically modifies T cells with DNA plasmids to express T-cell receptors to target neoantigens inside and outside hotspots for solid tumors and CAR to target CD19 for blood cancers using the company’s rapid personalized manufacturing to produce and release CAR-T within two days of gene transfer, the company said of its platform.
Ziopharm Oncology Chief Executive Officer Laurence Cooper said he was delighted about the chance to deepen his company’s relationship with MD Anderson.
“This new agreement is a launch point to expand our TCR library and execute two new clinical trials; a trial for utilizing TCRs from the library targeting hotspot mutations in KRAS, TP53 and EGFR, and a second trial for personalized TCRs targeting patient-specific neoantigens,” Cooper said in a statement.
Ferran Prat, head of research administration and industry ventures at MD Anderson, said cell-based immunotherapies have proven to be a “powerful new option” in treating blood-based cancers, but have not yet proven successful in solid tumors. Prat said the hope is that researchers are on the cusp of breaking through that impasse and will be able to advance a new generation of cell therapies that could do so.
Under its prior agreement with MD Anderson, Ziopharm has approximately $20 million of pre-funded R&D, which may now be used under the new agreement, for both the CAR-T or TCR-T initiatives, the company said. Under terms of the new agreement, Ziopharm will provide an additional $20 million for the expanded work through 2023. Undisclosed payments are also included in the deal based on certain development and regulatory milestones.
In addition to the R&D agreement, Ziopharm also entered into a lease agreement with MD Anderson for laboratory and office space in Texas. The new facility will serve as home for Ziopharm’s expanded Houston office, the company noted.
