On October 26, 2020 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that the U.S. Food and Drug Administration (FDA) granted TRC102 orphan drug designation for the treatment of patients with malignant glioma, including glioblastoma (GBM) (Press release, Tracon Pharmaceuticals, OCT 26, 2020, View Source [SID1234569058]). TRC102 is a small molecule inhibitor of the DNA base excision repair pathway.

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TRC102 is being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA). TRC102 was evaluated in a Phase 2 trial in combination with Temodar chemotherapy in 19 patients with progressive or recurrent GBM who progressed following Temodar and external beam radiotherapy. Extended survival was observed in two patients for more than two years, both of whom demonstrated activation of the DNA base excision repair pathway and demonstrated hyperactivation of DNA damage response genes. TRC102 was also evaluated in combination with chemotherapy and external beam radiotherapy in a separate Phase 1 study of 15 patients with newly diagnosed non-squamous cell non-small cell lung cancer that resulted in a response by the Radiographic Evaluation Criteria in Solid Tumors (RECIST) in all patients, including three patients who had a complete response to treatment.

"Orphan drug designation for TRC102 underscores the high level of unmet medical need in patients with malignant glioma and supports its potential in this indication based on the previously reported data demonstrating prolonged survival in patients retreated with Temodar combined with TRC102," said James Freddo, M.D, Chief Medical Officer of TRACON. "We remain committed to developing TRC102 in collaboration with the National Cancer Institute and believe that the data generated to date provide a rationale to study TRC102 in combination with Temodar and radiotherapy in newly diagnosed patients with malignant glioma."

The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics that are being developed to address rare diseases or disorders that affect fewer than 200,000 people in the U.S. With orphan designation, TRACON qualifies for various incentives with respect to TRC102 for the treatment of glioblastoma, including tax credits for qualified clinical trials and seven years of market exclusivity upon receipt of regulatory approval.

About TRC102

TRC102 (methoxyamine) is a novel, clinical-stage small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA). For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical-trials.php.

About Malignant Glioma and GBM

GBM (also called glioblastoma) is a fast-growing malignant glioma that develops from star-shaped glial cells (astrocytes and oligodendrocytes) that support the health of the nerve cells within the brain. GBM is the most invasive type of glial tumors, rapidly growing and commonly spreading into nearby brain tissue. The National Cancer Institute estimates that approximately 22,850 adults (12,630 men and 10,280 women) are diagnosed with brain and other nervous system cancer annually in the U.S. and approximately 15,320 of these diagnoses will result in death. GBM has an incidence of two to three per 100,000 adults per year in the U.S., and accounts for 52 percent of all primary brain tumors.