Ziopharm Oncology (ZIOP) recently held an R&D Day to provide an outlook for the company’s future with the new management team in place, notes John McCamant, a biotech sector specialist and editor of The Medical Technology Stock Letter.
The company showed extremely encouraging early data in China from the ‘compassionate use’ cohort in Taiwan from their CD-19 CAR-T program. The company is also rumored to having a BCMA (melanoma) CAR-T program which along with CD-19 would give ZIOP two of the best targets in oncology.
ZIOP is very close to moving into the clinic in China for an autologous CD-19 RPM CAR-T trial and we should expect a press release announcing dosing of the first clinical trial patients in the next few weeks.
From what we understand, there is a line of patients already clamoring for the treatment in China. The company is motivated to show results within the first 30-90 days of treating the first patients and will announce data in “real time” and not wait for a medical conference.
This is important as we have said in the past if the cell therapy is going to work, it will be clear within the first 30-90 days if the therapy is effective.
This transparency is what we expect from the new management team led by James Huang, a seasoned oncology investor and executive. In our view, he is finally moving the company to operational excellence.
Using their non-viral delivery and rapid manufacturing platforms (RPM) the company met their dream claims which have been elucidated for more than three years:
• Treated patients with CAR-T with cells manufactured in one day, not weeks like viral solutions like KITE/ GILEAD and CELGENE/ JUNO
• Cell manufacturing costs 5% of competition
• Injected CAR-T cell payloads of ½-1% of viral solutions, the cells were grown in the body, not in a lab. Body acted as its own bioreactor
• Company has already shown complete remission in at least one patient — the other patients outcomes not certain as bodies may still be growing more cells
In our view, ZIOP’s technology is disruptive and transformative as the non-viral platform slashes costs through completely simplifying the process. The ability to manufacture in house overnight allows for broad based treatment at virtually every single community hospital and oncology office in the U.S.
This cost and time savings is what is required to make CAR-T therapy ubiquitous and a standard of care. Existing viral solutions will never show the same throughput or cost efficiencies and cost $500K per patient.
The upcoming hotspot mutation TCR trial for solid epithelial cancers is scheduled to treat patients in the H2:21, however the company is working hard to make this happen even faster. This data could serve as significant proof-of-concept (POC) for the entire platform if positive, which we expect it to be.
We also expect the CD-19 data from China will also be positive and “tee-up” the TCR solid tumor data. Thus, by year end ZIOP will deliver two potential POC data points that would fully validate both their CAR-T and TCR programs.
In our view, 2021 will be a transformative year for ZIOP as the new management team diligently delivers POC data. ZIOP is a buy under $5 with a target price of $12 a share.
