Ziopharm Oncology Reports Third Quarter 2020 Financial Results and Provides Corporate Update
– Company plans to file IND for Ziopharm TCR-T program in Q1 of next year for its Library “hotspot” trial –
– Eden BioCell on track for IND filing in
– Three abstracts accepted at
– Controlled IL-12 receives Rare Pediatric Disease Designantion for DIPG; all three clinical sites active in phase 1/2 pediatric brain tumor trial –
– Strengthens leadership with two new Directors;
“During the third quarter, we again made progress in all three programs and strengthened the Board of Directors,
Recent Corporate Highlights
Sleeping Beauty TCR-T Program
Personalized and LibraryTCR-T Clinical Trials with MD Anderson Cancer Center. During the third quarter, Ziopharm further expanded its library of T-cell receptors (TCRs) targeting shared neoantigens in hotspots for use with the Sleeping Beauty platform. The Company remains on track to file an Investigational New Drug (IND) application with the FDA in the first quarter of next year, seeking clearance to begin its TCR-T trial utilizing allogeneic TCRs from its library. The Company is working closely with MD Anderson, the initial site for this trial, which is expected to commence mid-2021 to treat patients with gynecologic, colorectal, pancreatic, non-small cell lung and cholangiocarcinoma cancers. The Company continues with its planning for a clinical trial evaluating its Personalized TCR-T platform, which will be initiated at MD Anderson after the Library TCR-T trial.
- NCI Phase 2 Personalized TCR-T Trial. This phase 2 study, under the direction of
Steven Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the National Cancer Institute(NCI), and his team, is a first-in-human, non-viral TCR-T study that will enroll patients with a range of solid tumors. The NCI controls the timing of patient treatment for this trial, which is now estimated for next year. During the quarter, data regarding the engineering runs conducted by Ziopharm were successfully transferred to the NCI and the manufacturing process was authenticated by their team and is being validated in their GMP facility. Despite this, the NCI has recently informed Ziopharm that enrollment to the trial will additionally be delayed by regulatory requirements being implemented by the National Institute of Healthas well as the impact of COVID-19. The ongoing pandemic led to the depletion of patients available to be treated on TCR-T trials at the NCI due to disease progression while the facility was largely shut down and has added time needed to accrue new patients. As previously mentioned, the NCI has been proactively screening patients for neoantigens and TCRs to render them eligible for the trial (NCT0402436).
Sleeping Beauty CAR-T Program
- Eden BioCell CAR-T Study. The Company’s joint venture partner, Eden BioCell, has commenced filing of an IND for a clinical trial in
Taiwanto assess patient-derived (autologous) CD19-specific membrane bound IL-15 (mbIL15) CAR-T cells, produced using our Rapid Personalized Manufacturing (RPM) platform. The team expects the filing to be complete before year end, as planned. In addition, Eden BioCell and partners have dosed several patients with relapsed CD19+ malignancies under compassionate use, infusing autologous CAR-T the day after gene transfer per RPM. They report initial data showing the presence of infused T cells, measured weeks after infusion, in peripheral blood and bone marrow. Preliminary observations appear to indicate that mbIL15 supports the manufacturing of CAR-T under RPM which can be safely infused without unexpected toxicities. Additional follow-up is underway in Asia.
- Ziopharm CAR-T Study. The Company’s clinical collaborators at MD Anderson are actively screening and evaluating patients for enrollment in the phase 1 clinical trial infusing donor-derived (allogeneic) CD19-specific CAR-T therapies produced using RPM. Up to 24 patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic bone marrow transplantation will be enrolled in this investigator-initiated trial (NCT03579888).
Controlled IL-12 Program
- SNO 2020. Three abstracts detailing clinical data and observations of Controlled IL-12 have been accepted for presentation at the upcoming 2020
Society for Neuro-Oncology(SNO) virtual annual meeting next month. Updates to be presented on Controlled IL-12 in DIPG (NCT03330197) and combination studies of Controlled IL-12 with Opdivo as a phase 1 trial (NCT03636477) and with Libtayo as a phase 2 trial (NCT04006119) in recurrent glioblastoma (rGBM).
- Pediatric Trial. In the Company’s phase 1/2 study of Controlled IL-12 for the treatment of pediatric brain tumors, all three clinical sites are now active. The trial is designed to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 with up to 14 days of oral veledimex in children with diffuse intrinsic pontine glioma (DIPG). Up to 12 patients with DIPG may be enrolled in phase 1 of the study, which is being conducted at: Lurie Children’s Hospital, the Dana-Farber Cancer Institute and the University of California in San Francisco. The FDA recently granted a Rare Pediatric Disease Designation to Controlled IL-12 for the investigational treatment of DIPG.
- Expanded Board of Directors. During the third quarter, Ziopharm announced the appointment of two new members of the Company’s Board of Directors. Biotech entrepreneur
James Huang, Managing Partner at Kleiner Perkins Caufield & Byers China, was added to the Board in July, and in September, Kevin Buchi, biotech industry veteran and former CEO of Cephalon joined the Board. Directors will continue to actively review the Board membership, in coordination with a retained national search firm, to ensure the skills and experience of directors support the progress and future prospects of the business.
- Scientific Advisory Board. Following the appointment of immunotherapy pioneer,
Carl June, M.D., as Chairman of Ziopharm’s Scientific Advisory Board(SAB), the Company announced population of the SABin September with the addition of Adi Barzel, Ph.D., Gavin Dunn, M.D., Ph.D., Matthew Porteus, M.D., Ph.D., and Kole Roybal, Ph.D. The SABwill provide strategic counsel to guide the efficient development of Ziopharm’s innovative technologies and pipeline of immunotherapies.
- Executive Leadership. Ziopharm is pleased to welcome former Gilead executive
Adam Levy, Ph.D., M.B.A., as EVP, Investor Relations and Corporate Communications. Most recently, Dr. Levywas Executive Director and Head, Corporate Strategy and Investor Relations for Gilead Sciences. Previously, Dr. Levywas VP, Corporate Strategy for Alexion and Executive Director, Corporate Strategy for Bristol-Myers Squibb. He had prior leadership positions with Novartis and McKinsey & Company. Adam holds a Ph.D. in Molecular Biology from the University of Illinoisand an MBA in Finance and Strategy from Northwestern University Kellogg School of Management.
Third Quarter 2020 Financial Results
- Research and development expenses were
$14.0million for the third quarter of 2020, compared to $8.6 million for the third quarter of 2019, primarily reflecting increased manufacturing activity and headcount.
- General and administrative expenses were
$6.4 millionfor the third quarter of 2020, compared to $4.8 million for the third quarter of 2019. The increase in general and administrative expenses for the third quarter of 2020 is primarily due to increased headcount, legal costs associated with its expanded patent portfolio and facility costs.
- Net loss for the third quarter of 2020, was
$20.3 million, or $(0.10)per share, compared to a net loss of $74.0 million, or $(0.43)per share, for the third quarter of 2019 (which reflected a $60.8 million, $(0.36)per share, non-cash charge for an inducement warrant).
- Cash and cash equivalents, as of
September 30, 2020were $135.5 million.
- Additionally, a prepayment of approximately
$11.4million remains for work to be conducted by the Company at MD Anderson under the Company’s research and development agreements.
Conference Call and Webcast
Ziopharm will host a conference call and webcast for the investment community today,
Ziopharm is developing non-viral and cytokine-driven cell and gene therapies that weaponize the body’s immune system to treat the millions of people globally diagnosed with a solid tumor each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology with a goal to treat any type of solid tumor. Ziopharm’s pipeline is built for commercially scalable, cost effective T-cell receptor T-cell therapies based on its non-viral Sleeping Beauty gene transfer platform, a precisely controlled IL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T program. The Company has clinical and strategic partnerships with the
Forward-Looking Statements Disclaimer
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the Company's business and strategic plans and the progress, design and timing of the Company's research and development programs, including the anticipated dates for the clearance of the IND for its TCR-T clinical trial and the submission of the IND by Eden BioCell, timing for the treatment of patients in the NCI’s clinical trial, enrollment expectations for its CAR-T and DIPG clinical trials, and the timing for the data readouts for its Controlled IL-12 trials. Although Ziopharm’s management team believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Ziopharm, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, changes in our operating plans that may impact our cash expenditures, the NCI’s ability to complete the requirements prior to treating patients, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Ziopharm’s product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the
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− Financial Tables Follow −
|Statements of Operations|
|(in thousands except share and per share data)|
|Three Months Ended|
|Research and development||$||13,968||$||8,641|
|General and administrative||6,353||4,807|
|Total operating expenses||20,321||13,448|
|Loss from operations||(20,321||)||(13,448||)|
|Other income, net||6||203|
|Noncash inducement warrant expense||-||(60,751||)|
|Basic and diluted net loss per share||$||(0.10||)||$||(0.43||)|
|Weighted average common shares outstanding used|
|to compute basic and diluted net loss per share||212,837,367||170,613,712|
|Balance Sheet Data|
|Cash and cash equivalents||135,471||79,741|
|Total stockholders' equity||144,918||95,010|
Source: ZIOPHARM Oncology Inc