Ziopharm Oncology (ZIOP) recently announced that the FDA has cleared an investigational new drug application (IND) for a Phase I trial to test their 3rd generation CD19-specific CAR-T, explains biotech sector specialist John McCamant, editor of The Medical Technology Stock Letter.
This drug candidate is produced using a process termed rapid personalized manufacture (RPM), as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas.
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These are very sick patients who have no treatment options left as they have relapsed in the months following allogeneic bone marrow transplant (aBMT), with some having a median survival of only 2-3 months.
The IND clearance builds upon the firm's experience with two prior generations of immunotherapy trials using the Sleeping Beauty platform, which it believes is the most clinically advanced non-viral approach to the genetic modification of T-cells.
With this third-generation trial, DNA from the Sleeping Beauty system is stably inserted into the genome of resting T cells to co-express a chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch, which is designed to reduce cost, simplify production, and preserve the therapeutic potential of the T cells.
Up to 24 patients will be enrolled in the study to evaluate the infusion of donor-derived RPM CAR-T in patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT.
This study will be conducted at The University of Texas’ MD Anderson Cancer Center, one of the world’s premier oncology research hospitals, under an investigator-initiated trial expected to begin later this year.
The initiation of this Sleeping Beauty study is rather important for Zipharm and immuno-oncology as a whole. First, Ziopharm's 3rd generation CAR-T has the potential to significantly enhance efficacy in very late-stage cancers. Next, it will greatly reduce the high costs relative to first generation CAR-Ts (currently in the ~$500,000 per patient) range.
Finally, the IND sign-off by the FDA also validates the firm's manufacturing system as safe. This is very critical as the agency has repeatedly emphasized the importance of having safe manufacturing processes when it comes to cell and gene therapies. Overall, more novel trails are due to start soon and the company's technology promise is beginning to become reality.
In recent days, ZIOP’s stock has been under pressure following an error-filled report on the Internet by a short seller that was nefariously spun to include scientific analysis to confuse and create fear in a stock mostly owned by retail investors.
The report’s attack went after rather ancient history from the old ZIOP days even before Lawrence Cooper joined as CEO from MD Anderson in 2015. In our view, ZIOP will refute the shorts with fundamental progress and the current price will be remembered as a great buying opportunity.
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