Quaratusugene Ozeplasmid Earns FDA ODD in Small Cell Lung Cancer

The FDA previously granted fast track designation to quaratusugene ozeplasmid for treating patients with ES-SCLC in June 2023.

The FDA has granted orphan drug designation to quaratusugene ozeplasmid (Reqorsa) for patients with small cell lung cancer (SCLC), according to a press release from Genprex, Inc.¹

According to the press release, quaratusugene ozeplasmid contains a plasmid expressing tumor suppressor gene protein TUSC2, which has reduced or no protein expression in many SCLC cases. It is hypothesized that re-expressing TUSC2 via quaratusugene ozeplasmid may produce clinical efficacy.

“We are excited to receive orphan drug designation from the FDA for [quaratusugene ozeplasmid] for patients with SCLC,” Rodney Varner, president, chairman, and chief executive officer at Genprex, said in the press release. “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, extensive stage-SCLC [ES-SCLC], and [non–small cell lung cancer].”

According to Varner, investigators plan to initiate the phase 1/2 Acclaim-3 trial (NCT05703971) assessing quaratusugene ozeplasmid in combination with atezolizumab (Tecentriq) as a treatment for patients with ES-SCLC in the fourth quarter of 2023. Patients who have a complete response, partial response, or stable disease after receiving initial treatment consisting of 3 to 4 cycles of carboplatin plus etoposide and atezolizumab are eligible to receive the study regimen.

In phase 1 of the study, approximately 12 patients will receive quaratusugene ozeplasmid intravenously at 0.09 mg/kg or 0.12 mg/kg once every 21 days plus 1200 mg of atezolizumab intravenously every 21 days until disease progression or unacceptable toxicity. After identifying a recommended phase 2 dose (RP2D), investigators will continue to administer quaratusugene ozeplasmid plus atezolizumab to approximately 50 patients in phase 2.

The primary end points of the study are identifying the maximum tolerated dose or RP2D in phase 1 and progression-free survival per RECIST v1.1 criteria in phase 2. Secondary end points include overall survival, safety, and pharmacokinetics.

Patients 18 years and older with histologically or cytologically confirmed ES-SCLC and an ECOG performance status of 0 to 1 are eligible for enrollment on the trial. Additional inclusion criteria include having adequate renal and hepatic function as well as stable cardiac condition with a left ventricular ejection fraction of at least 40% within 21 days of entry.

Those who received prior gene therapy or prophylactic cranial irradiation or consolidation thoracic radiotherapy are unable to enroll on the study. Patients are also unsuitable for enrollment if they have active viral, bacterial, or fungal infections requiring intervention; serious concurrent illness or psychological, familial, sociological, geographical, or other concomitant conditions; or a history of autoimmune disease requiring immunosuppression.

The FDA previously granted fast track designation to quaratusugene ozeplasmid for treating patients with ES-SCLC in June 2023.²

“This fast track designation for the Acclaim-3 patient population is another validation of [quaratusugene ozeplasmid’s] potential to treat lung cancer,” Marker Berger, MD, chief medical officer at Genprex, said in a press release at the time of the fast track designation.

References

1. Genprex granted FDA orphan drug designation (ODD) for REQORSA® immunogene therapy for the treatment of small cell lung cancer. News release. Genprex, Inc. August 10, 2023. Accessed August 10, 2023. bit.ly/3ODCAyG
2. Genprex receives U.S. FDA fast track designation for REQORSA® immunogene therapy in combination with Tecentriq® for the treatment of small cell lung cancer. News release. Genprex, Inc. June 28, 2023. Accessed August 15, 2023. https://bit.ly/3NsdZfP