On August 6, 2020Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported its financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, Ziopharm, AUG 6, 2020, View Source [SID1234563088]).

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"During the second quarter, we were able to report positive clinical and organizational progress, positioning the Company for additional data readouts in the coming year," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer. "With the completion of enrollment in our phase 2 trial of Controlled IL-12 in combination with Libtayo, and launches of two new trials, we have begun to see encouraging signs of our partners re-emerging despite the COVID-19 outbreak."

Recent Corporate Highlights

Sleeping Beauty TCR-T Program

NCI Phase 2 Personalized TCR-T Trial. During the quarter and in recent weeks, the Ziopharm team collaborated with the National Cancer Institute (NCI) to advance manufacturing preparations for dosing the first patient in this first-in-human non-viral TCR-T study (NCT0402436). As NCI laboratory functions gradually re-open, the team there is once again proactively screening patients for neoantigens and T-cell receptors (TCRs) to render them eligible for the trial. Ziopharm has been able to use its newly expanded laboratories in Houston to complete required engineering runs and provided that information to the NCI to help expedite their enrollment to the trial.

Personalized and Library TCR-T Clinical Trials with MD Anderson. Based on interactions with the U.S. Food and Drug Administration (FDA), the Company continues to make progress toward finalizing the design of its TCR-T clinical trials at MD Anderson based on the Sleeping Beauty platform and clearance of an Investigational New Drug (IND) application to the FDA in Q1 2021. The Company plans to evaluate both its personalized TCR-T and its library TCR-T therapies and is focused on completing IND-enabling CMC (chemistry, manufacturing and controls) and nonclinical data package to support those opportunities. Given its progress assembling a TCR library, the Company expects the trial using these allogeneic receptors to begin enrollment first, with anticipated cancer indications of gynecologic, colorectal, pancreatic, non-small cell lung cancer and cholangiocarcinoma.

Controlled IL-12 Program

Phase 2 Combination Study. Ziopharm announced completion of enrollment in June of its phase 2 combination trial of Controlled IL-12 with Regeneron’s Libtayo to treat patients with recurrent glioblastoma (rGBM). Per the study protocol, 40 patients were enrolled at 7 sites, of whom 39 were dosed with the combination. Initial data is expected to be submitted for presentation later this year.

Pediatric Trial. The Company recently announced that the first patient with diffuse intrinsic pontine glioma (DIPG) had been dosed in its phase 1/2 study of Controlled IL-12 for the treatment of pediatric brain tumors. The trial (NCT03330197) 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 gliomas. Up to 12 patients with DIPG may be enrolled in phase 1 of the study, which is being conducted at leading pediatric cancer centers across the United States, including Lurie Children’s Hospital in Chicago, the Dana-Farber Cancer Institute in Boston and the University of California in San Francisco.

ASCO 2020. The Company provided positive clinical updates at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) virtual annual meeting in late May. Continued follow-up from Controlled IL-12 monotherapy studies reinforced encouraging median overall survival, while Controlled IL-12 in combination with a PD-1 inhibitor demonstrated a favorable safety profile and encouraging initial survival data. Additional data are expected to be presented later this year.

Sleeping Beauty CAR-T Program

Eden BioCell CAR-T Study. The Company’s joint venture partner, Eden BioCell, continued to make progress toward filing an IND this year for a clinical trial in Taiwan to assess patient-derived (autologous) CD19-specific membrane bound IL-15 CAR-T cells, produced using Rapid Personalized Manufacturing (RPM) technology designed to reduce cost and simplify production for infusion the day after gene transfer.

Ziopharm CAR-T Study. The Company’s clinical collaborators at MD Anderson Cancer Center were able to complete necessary preparations and commence enrollment in the phase 1 clinical trial infusing donor-derived (allogeneic) CD19-specific CAR-T therapies produced using RPM. Up to 24 patients with advanced CD19+ leukemias and lymphomas who have relapsed after allogeneic bone marrow transplantation will be enrolled in this investigator-initiated trial (NCT03579888).

Operational

Expanded Board of Directors. Ziopharm recently announced the appointment of James Huang to the Company’s Board of Directors. Mr. Huang is a Managing Partner at Kleiner Perkins Caufield & Byers China and has founded and financed several innovative life sciences companies, including GenScript, Legend Biotech and Zai Lab. He is also Founding Partner of Panacea Venture, which formed TriArm Therapeutics, the funding partner for Ziopharm’s joint venture, Eden BioCell.

Scientific Advisory Board. In June, the Company announced the appointment of renowned oncology and immunotherapy pioneer, Carl June, M.D., as Chairman of its newly formed Scientific Advisory Board (SAB). Dr. June is recognized in the oncology field for his groundbreaking work in the development and commercialization of gene therapy and T-cell therapies. The SAB will provide strategic counsel to guide the efficient development of Ziopharm’s innovative technologies and pipeline of immunotherapies.

Second Quarter 2020 Financial Results

Research and development expenses were $12.1 million for the second quarter of 2020, compared to $10.0 million for the second quarter of 2019, primarily reflecting increased clinical trial activity.

General and administrative expenses were $6.6 million for the second quarter of 2020, compared to $4.8 million for the second quarter of 2019. The increase in general and administrative expenses for the second quarter of 2020 is primarily due to increased headcount, legal costs associated with its expanded patent portfolio and facility costs.

Net loss for the second quarter of 2020, was $18.6 million, or $(0.09) per share, compared to a net loss of $14.6 million, or $(0.09) per share, for the second quarter of 2019.

Cash and cash equivalents, as of June 30, 2020 were $153.5 million.

A prepayment of approximately $14.0 million 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, August 6, 2020, at 4:30 p.m. EDT. The conference call can be accessed by dialing 1-877-451-6152 (U.S. and Canada) or 1-201-389-0879 (international). The passcode for the conference call is 13706729. To access the live webcast or the subsequent archived recording, click here or visit the "Investors" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the company’s website for two weeks.