Ziopharm Oncology Announces FDA Clearance of IND for Rapid Personalized Manufacture of CD19-specific CAR-T
– Phase 1 clinical trial of CAR-T produced and infused in two days or less from gene transfer using Ziopharm’s Sleeping Beauty platform –
– Rapid personalized manufacture of CD19-specific CAR-T for investigation in unaddressed patient population with relapsed leukemias and lymphoma after bone marrow transplantation –
The IND clearance builds upon the Company’s experience with two prior generations of immunotherapy trials using the Sleeping Beauty platform, which it believes is the most clinically-advanced non-viral approach to the genetic modification of T cells. With this third-generation trial, 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.
“There are currently no effective treatment options for patients who relapse soon after allogeneic bone marrow transplantation (BMT), as evidenced by their low rate of remission and poor long-term survival. This trial expands the range of patients with CD19-expressing malignancies that can be treated using the RPM technology,” said
Up to 24 patients will be enrolled to evaluate infusion of donor-derived RPM CAR-T in patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT. This study will be conducted at
Research reveals three-year survival for adults with CD19+ acute lymphoblastic leukemia after allogeneic BMT ranges from 30% to 65%.1 For patients with other CD19+ cancers, allogeneic BMT can provide a three-year survival rates between 30% to 75%.1 Few patients experience a durable remission who relapse in the months following allogeneic BMT, regardless of the treatment modality, with some having a median survival of only 2 to 3 months.2
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1 D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2018. Available at https://www.cibmtr.org
2 Keil F, Prinz E, Kalhs P, et al. Treatment of leukemic relapse after allogeneic stem cell transplantation with cytotoreductive chemotherapy and/or immunotherapy or second transplants. Leukemia 2001; 15:355-361.
Ziopharm Oncology Contact:
VP, Investor Relations and Corporate Communications
Source: ZIOPHARM Oncology Inc