After disclosing that the Food and Drug Administration is asking for more information before signing off on a phase 1 study of its "Sleeping Beauty" CAR-T for leukemia and lymphoma, shares in Ziopharm Oncology (NASDAQ:ZIOP) were tumbling 17.7% at 3 p.m. EDT today.
A shrinking cash stockpile on Ziopharm's balance sheet has management paring the research and development pipeline to maintain focus on two approaches. Unfortunately, there's uncertainty with both platforms now, following news of a delay in Sleeping Beauty's time line.
Last month the company said it's pausing plans to pursue a registration-ready trial of its most advanced therapy, Ad-RTS-hIL-12.
An IL-12 control therapy, Ad-RTS-hIL-12 boosts IL-12 expression in the tumor microenvironment to spark an immune-system response. The company's seen some intriguing early-stage responses to this therapy, but management postponed pivotal trials while it tackles "technical requirements related to chemistry and manufacturing control (CMC)."
Today, Ziopharm Oncology said that the FDA has not yet given it a green light to begin its Sleeping Beauty study. Ziopharm hopes to overcome delays in producing CAR-Ts that are associated with existing CAR-T treatments by allowing off-the-shelf production within one to two days. Existing, approved CAR-T's can take two to three weeks to be produced at outside laboratories.
Management didn't go into too much detail regarding what information the FDA is requesting before granting a go-ahead, except that it relates to CMC, but said they "know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner."
Responding in a timely manner is all well and good, but investors are right to be disappointed by today's disclosure. The uncertainty associated with pivotal trials of Ad-RTS-hIL-12 raised the stakes for Sleeping Beauty, and a trial start for the therapy this year was a major focus of excitement among bullish investors.
It's absolutely possible that this is a minor delay that's resolved quickly, but unfortunately, Ziopharm Oncology doesn't have the luxury of time. Exiting March, it boasted only $51 million in cash on the balance sheet; according to management, that's only enough to fund the company through the second quarter of 2019.