Ziopharm Oncology, Inc. (NASDAQ:ZIOP), today announced two publications in peer-reviewed scientific journals supporting the Company’s ongoing clinical programs by reinforcing two of the foundational technologies underlying the Company’s approach to the genetic modification of T cells to target cancer. A paper in the journal Blood enhances the clinical data set surrounding the Sleeping Beauty platform by indicating encouraging long-term survival of patients that received CAR-T. The Clinical Cancer Research publication demonstrates that T-cell receptors (TCRs) targeting genetic changes within TP53 can be obtained from T cells in the peripheral blood, overcoming the need to harvest a patient’s tumor. The Company plans to expand its existing library of TCRs as part of its commitment to advance clinical development for the treatment of patients whose solid tumors have driver mutations, including in TP53, using Sleeping Beauty-modified T cells.
“These articles authored by our partners at MD Anderson Cancer Center and the National Cancer Institute highlight two of the technologies we are developing,” said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. “The Blood publication describes the multi-year survival of patients with non-Hodgkin lymphoma after CD19-specific CAR-T and long-term persistence of the T cells manufactured with the Sleeping Beauty system. The Clinical Cancer Research publication describes how TCRs with specificity to mutations within TP53 present in tumor cells can be obtained from circulating T cells, which overcomes the need to obtain tumor-infiltrating lymphocytes through surgical resection. Taken together, we believe these reports reinforce the potential of our Sleeping Beauty system as an ideal solution for the non-viral gene transfer of T cells and facilitate the manufacture of TCR-T by obtaining neoantigen-specific TCRs from peripheral blood.”
A letter published in Blood,1 the journal of the American Society of Hematology, discussed long-term outcomes of seven patients with relapsed or refractory B-cell lymphoid malignancies, all of whom had received CD19-specific CAR-T cells infused two days following autologous hematopoietic stem-cell transplantation (NCT00968760). Four patients demonstrated sustained persistence of CAR-T (median time of persistence duration was 4.5 years, range 2-5 years). Five-year progression-free survival and overall survival were 71% and 86%, respectively.
Data published online in Clinical Cancer Research2 showed that TP53 mutation-reactive T cells circulating in peripheral blood are a source of neoantigen-specific TCRs for adoptive cell therapy. Last year, Ziopharm announced an exclusive license with the National Cancer Institute to access a library of TCRs against cancer-specific neoantigens in hotspots targeting TP53, KRAS and EGFR, for use with the Sleeping Beauty platform. Ziopharm’s TCR-T cell therapy program is led by Drew Deniger, Ph.D., who joined the company from NCI and is the last author on the article published in Clinical Cancer Research. Additional information regarding this publication, including commentary from Dr. Steven Rosenberg, chief of the Surgery Branch at NCI, is available on the website of the American Association for Cancer Research.3