Regulatory, Setbacks

Ziopharm gets hit with a clinical hold as FDA red lights human study using their fast-built CAR-T

One of Ziopharm’s $ZIO heavily touted, fast-built CAR-Ts isn’t ready for a human trial just yet. 

Laurence Cooper

The biotech reports that the FDA has dropped a clinical hold on their Phase I study of a CAR-T that can be built in 2 days to express CD-19, IL-15 and a safety switch for CD-19/positive leukemias and lymphomas. But regulators want to know more about the chemistry and manufacturing, according to the company, so they put on a hold that may delay the start of the study.

Ziopharm’s shares slid 11% as the news spread.

The FDA in these cases says nothing, leaving the companies and investigators involved free to say whatever they like. In this case, that means an assurance that they can respond in a “timely manner,” as they know exactly what the FDA is looking for.

Ziopharm also said that the clinical hold wouldn’t affect their timeline on their work involving TCR-modified cells for solid tumors.

Turnaround time has been a big issue for the first two companies to debut a first-generation CAR-T: Novartis and Gilead/Kite. But Ziopharm says they have the problem licked. They just need to prove it. 

Ziopharm CEO Laurence Cooper couldn’t resist plugging their work in reporting the setback. He noted: 

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.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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