Dive Brief:
- The Food and Drug Administration slapped a clinical hold on a yet-to-begin Phase 1 trial of a CD19-targeted CAR-T therapy being developed by Ziopharm Oncology, sending shares in the biotech down by over 18% Monday.
- The FDA has requested additional information relating to chemistry, manufacturing and controls of the cell therapy, which is designed to be manufactured at point-of-care using ZioPharm's so-called Sleeping Beauty platform.
- While the clinical hold looks set to delay the start of this study, the company does not foresee any impact on its planned trial at the National Cancer Institute using the platform for TCR-modified T cells in solid tumors.
Dive Insight:
CAR-T-based therapies have the potential to be game changers for the treatment of certain cancers, with some holding out hope for possible cures. Two CAR-T therapies are cleared on the U.S. market — Novartis' Kymriah (tisagenlecleucel) and Gilead Sciences' Yescarta (axicabtagene ciloleucel) — with a number of companies in the wings, including Celgene, which recently acquired Juno Therapeutics.
Solving the supply chain for these personalized and patient-derived therapies presents a major challenge to commercializing CAR-T. Created from a patient's own T cells, most CAR-Ts need to be shipped to a manufacturing facility, where they are genetically engineered and then shipped back for infusion into a patient.
The cells must be kept at the correct temperature throughout the process, which must occur quickly as the eligible patients are often extremely sick. Maintaining chains of custody and identity is crucial to ensure the right treatment gets to the right patient.
The Sleeping Beauty technology, which Ziopharm and Intrexon licensed from the University of Texas MD Anderson Cancer Center in 2015, is designed to be a lower-cost and with fewer steps.
Ziopharma believes that by moving away from centralized manufacturing toward point-of-care manufacturing could allow for CAR-T cells to be produced within two days — much faster than the two to three weeks currently needed by the leading companies.
So far, though, that's just the hope, and the FDA's request for further information on manufacturing suggests the agency wants to double-check this new approach.
"We know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner," Ziopharm CEO Laurence Cooper said in a statement. Investors, however, don't appear so convinced.