On September 14, 2020 Ziopharm Oncology, Inc. ("Ziopharm" or "the Company") (Nasdaq:ZIOP) reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to Ad-RTS-hIL-12 with veledimex (Controlled IL-12) for the investigational treatment of diffuse intrinsic pontine glioma (DIPG), a lethal brain tumor occurring in the pontine region of the brain (Press release, Ziopharm, SEP 14, 2020, View Source [SID1234569925]). DIPG accounts for approximately 10 to 15 percent of all cases of brain tumors in children. The Rare Pediatric Disease Designation program is intended to encourage the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
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"We are delighted to have received the Rare Pediatric Disease Designation for Controlled IL-12 from the FDA. This milestone for Ziopharm emphasizes the significant unmet need for children living with DIPG," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "Currently, there are no viable treatment options for this type of brain tumor. We are working with the FDA to advance Controlled IL-12 as a new gene therapy for this aggressive disease, which has historically been largely seen as incurable."
The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. If Ziopharm’s Biologics License Application (BLA) for Controlled IL-12 in DIPG is approved, the Company may be eligible to receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred to another company for their program.
About DIPG
In children, the incidence of brain cancer is approximately 4.84 per 100,000, according to the National Cancer Institute. Glioma in the pontine region of the brain, or DIPG, accounts for approximately 10-15 percent of all cases of pediatric brain tumors, with about 150-300 new diagnoses per year in the United States.1 Median survival ranges from 8-11 months.2 There are no curative options.
About Controlled IL-12 (Ad-RTS-hIL-12 plus veledimex)
Ziopharm’s Controlled IL-12 platform is an investigational gene therapy designed to induce and control the production of human interleukin 12 (hIL-12), a master-regulator of the immune system. The Company has treated more than 175 patients, including more than 125 patients with rGBM, with Ad-RTS-hIL-12 plus veledimex and administered more than 1,300 doses of veledimex across three types of solid tumors, building a significant safety profile, mechanistic dataset and evidence of anti-tumor effects.
Controlled IL-12 is being studied in a phase 1/2 trial (NCT03330197) designed to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 given 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, Dana-Farber Cancer Institute in Boston and University of California in San Francisco.