Pharma Focus America

Genprex Granted FDA Orphan Drug Designation for REQORSA® Immunogene Therapy in Small Cell Lung Cancer Treatment

Friday, August 11, 2023

Genprex, a company focused on developing transformative therapies for cancer and diabetes, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for their leading drug candidate, REQORSA® Immunogene Therapy. This innovative therapy, known by its scientific name quratusugene ozeplasmid, is intended to treat small cell lung cancer (SCLC).

Furthermore, in June 2023, the FDA also awarded Fast Track Designation (FTD) to REQORSA Immunogene Therapy. This designation pertains to its use in combination with Genentech, Inc.'s Tecentriq® for patients with extensive-stage small cell lung cancer (ES-SCLC) who have not experienced tumor progression following initial treatment involving Tecentriq and chemotherapy. ES-SCLC refers to cases where the cancer has spread from one lung to other parts of the body; it represents a common and severe form of SCLC. The FDA also granted Genprex FTD for REQORSA Immunogene Therapy for two additional indications: combined with Tagrisso for non-small cell lung cancer (NSCLC) patients who have progressed after Tagrisso treatment, and in combination with Keytruda for NSCLC patients who have progressed after Keytruda treatment.

Rodney Varner, Genprex's President, Chairman, and CEO, expressed enthusiasm for the FDA's Orphan Drug Designation and Fast Track designation. He emphasized the urgent need for improved treatment options for various types of lung cancer and shared the company's anticipation for initiating the Acclaim-3 clinical trial in the coming months. This trial aims to introduce a promising new therapy for patients struggling with these life-limiting cancers.

Orphan Drug Designation is granted to investigational treatments intended for rare diseases that impact fewer than 200,000 people in the United States. This designation provides valuable benefits to drug developers, including development support, tax credits for qualifying trials, exemption from certain FDA fees, and the potential for seven years of market exclusivity following approval.

Genprex's innovative approach to cancer treatment focuses on reexpressing tumor suppressor genes, a process often disrupted in cancer development. REQORSA, the company's leading candidate, employs a plasmid to express a tumor suppressor gene protein called TUSC2. This protein is commonly absent or reduced in small cell lung cancers, and studies suggest that restoring its expression could yield positive clinical outcomes.

Extensive-stage small cell lung cancer carries a grim prognosis, with a median progression-free survival of merely 5.2 months. Patients receiving Tecentriq as maintenance therapy experience a median progression-free survival of just 2.6 months, underscoring the pressing need for improved treatment strategies.

The upcoming Acclaim-3 clinical trial, a Phase 1/2 study, aims to evaluate REQORSA in combination with Tecentriq for patients with ES-SCLC who haven't experienced tumor progression after initial treatment. The trial's goal is to determine the 18-week progression-free survival rate, with the Phase 1 portion

concentrating on dose escalation and the Phase 2 segment involving more comprehensive patient enrollment for thorough evaluation.

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