FDA placed a clinical hold on an upcoming Phase I trial sponsored by Ziopharm Oncology Inc. (NASDAQ:ZIOP), which would be the first for its third-generation, point-of-care CAR T cells that require two days for manufacturing. Ziopharm was down $0.79 (18%) to $3.51 on Monday.
The therapy, produced using Ziopharm's Sleeping Beauty non-viral integration technology, comprises CD19-targeted CAR T cells co-expressing membrane-bound IL-15 and a control switch.
Ziopharm CEO Laurence Cooper previously told BioCentury membrane-bound IL-15 prolongs the survival and subsequent replicative capacity of CAR T cells in vivo, which could reduce the number of engineered cells for patient infusion needed to achieve efficacy. In combination with the rapidity of non-viral gene delivery, the therapy can be manufactured within two days compared with weeks for CAR T therapies that rely on viral CAR integration and lack engineered pro-survival signals.
Ziopharm said FDA requested additional CMC information related to testing the therapy for relapsed or refractory CD19-positive leukemias and lymphomas in an investigator-led study. Ziopharm spokesperson David Connolly declined to disclose details.
A trial start at the University of Texas MD Anderson Cancer Center is expected next half. Connolly said the company's guidance has not changed, but noted the timeline "is in jeopardy."
Ziopharm's second generation CD19-targeted CAR T therapy, which lacks the IL-15 component, is in Phase I testing at MD Anderson for advanced lymphoid malignancies. In the trial, manufacturing has required as little as a week, according to Connolly.
Ziopharm utilizes RheoSwitch technology from partner Intrexon Corp. (NYSE:XON), which regulates IL-15 expression in its third generation CAR T cell pipeline. Last year, Merck KGaA (Xetra:MRK) received an exclusive license to Sleeping Beauty and RheoSwitch to develop CAR T cells against two undisclosed targets for solid tumors and hematologic malignancies.
Intrexon added $0.31 to $16.46 Monday.