FDA places hold on Ziopharm CAR-T trial

CAR-T is emerging as one of the most potentially exciting therapies in the industry, particularly as a beacon for the collective push towards personalised treatment, so it is unsurprising that many small biotechs are currently pursuing treatments within this space.

However, one such company, Boston-based Ziopharm Oncology, has fallen foul of the FDA in its pursuits, as the US regulator slapped a clinical hold on its Phase 1 trial into its CD19-specific CAR-T therapies manufactured under point-of-care in patients with leukaemias and lymphomas. The news sent the company’s shares falling by 17%.

The hold was placed alongside requests for information relating to the therapy’s chemistry, manufacturing and controls. Ziopharm has since announced that it will work to satisfy these requests in collaboration with its partners, Intrexon's Precigen subsidiary and The University of Texas MD Anderson Cancer Center. It also noted that the commencement of the study could be delayed, but it does not expect the FDA’s move to alter the overall timeline of the trial.

The study will use the firm’s ‘Sleeping Beauty’ platform to rapidly generate CAR-T cells designed to co-express CD19-specific CAR, membrane-bound interleukin 15 and a safety switch, with the goal of targeting solid tumours infusing T-cell receptor-modified T cells.

"We know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner," said Dr Laurence Cooper, Chief Executive Officer of Ziopharm. "We are undertaking cutting-edge science and are on the verge of a paradigm shift based on our approach to very-rapidly manufacture CD19-specific T cells within two days using our non-viral approach to CAR-T therapy based on the ‘Sleeping Beauty’ platform."