TRACON Pharmaceuticals (NASDAQ:TCON) (FRA:T051) revealed on Monday that the US Food and Drug Administration (FDA) granted TRC102 Orphan Drug designation for the treatment of patients with a type of tumor called malignant glioma, including glioblastoma, an aggressive type of cancer that can occur in the brain or spinal cord.
According to the San Diego, California-based company which develops a clinical-stage pipeline of cancer therapies, TRC102 is a small molecule inhibitor of the DNA base excision repair pathway, that causes resistance to alkylating and antimetabolite chemotherapeutics.
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 glioblastoma, who progressed following Temodar and external beam radiotherapy.
Investors reacted positively to the news, sending TRACON shares up nearly 1% to $4.97 in New York.
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 TRACON chief medical officer James Freddo.
“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,” he added.
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 US.
With orphan designation, TRACON qualifies for various incentives with respect to TRC102 for the treatment of glioblastoma, including tax credits for qualified clinical trials.
Among the benefits of Orphan Drug status in the US is a seven-year period of market exclusivity for the indication, if approved.
Contact the author Uttara Choudhury at [email protected]
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