Genprex, Inc., a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, announces that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the Company’s lead drug candidate, REQORSA Immunogene Therapy (quratusugene ozeplasmid), for the treatment of small cell lung cancer (SCLC).
In addition to ODD for the treatment of SCLC, in June 2023, the FDA granted Fast Track Designation (FTD) for REQORSA Immunogene Therapy, in combination with Genentech, Inc’s Tecentriq in patients with extensive-stage small cell lung cancer (ES-SCLC) who did not develop tumour progression after receiving Tecentriq and chemotherapy as initial standard treatment. With ES-SCLC, the cancer has spread from one lung to the other, or to other parts of the body. Extensive-stage is the most common type of SCLC. FDA has also granted Genprex FTD for two other indications of REQORSA Immunogene Therapy, including REQORSA in combination with Tagrisso for non-small cell lung cancer (NSCLC) in patients who have progressed after Tagrisso treatment, and REQORSA in combination with Keytruda for NSCLC in patients who have progressed after Keytruda treatment.
“We are excited to receive Orphan Drug Designation from the FDA for REQORSA for patients with SCLC,” said Rodney Varner, President, Chairman and Chief Executive Officer at Genprex. “This FDA Orphan Drug Designation in combination with our recently received FDA Fast Track designation underscores the great need for better treatment options for patients with SCLC, ES-SCLC and NSCLC. We look forward to initiating the Acclaim-3 clinical trial expected in the fourth quarter of 2023 in order to bring hope of an effective new therapy to patients suffering with this life-limiting cancer.”
The FDA grants ODD status to investigational therapies being developed to treat, diagnose, or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Further, ODD provides benefits to drug developers, including assistance in the drug development process, tax credits for qualified trials, waiver of certain FDA fees, and potential for seven years of post-approval marketing exclusivity.
REQORSA contains a plasmid that expresses a tumour suppressor gene protein called TUSC2. Virtually 100% of small cell lung cancers have reduced or no TUSC2 protein expression, and 41% completely lack TUSC2 protein expression. Pre-clinical studies in mice suggest that re-expressing the TUSC2 protein may lead to clinical efficacy. ES-SCLC has a very poor prognosis, with a median progression free survival (PFS) of only 5.2 months. Importantly, median PFS for patients receiving Tecentriq as maintenance therapy is only 2.6 months from the start of maintenance treatment, so there is a great need for improvement in maintenance therapy.