Genprex Inc. (NASDAQ: GNPX) Celebrates Receipt of Orphan Drug Designation, Third Fast Track Designation Amid Preparations for Planned Acclaim-3 Clinical Trial

  • Genprex recently received orphan drug designation for its lead drug candidate REQORSA(R) Immunogene Therapy (quratusugene ozeplasmid) for the treatment of small cell lung cancer (“SCLC”)
  • The company also celebrated receipt of its third Fast Track Designation (“FTD”), granted by the FDA, this time for REQORSA Immunogene Therapy, in combination with Tecentriq in patients with extensive stage small cell lung cancer (“ES-SCLC”)
  • The orphan drug designation (“ODD”) in combination with the FTD is an indication of the great need for better treatment options for patients with ES-SCLC, and non-small cell lung cancer (“NSCLC”), according to Genprex President, Chairman, and CEO Rodney Varner
  • With the receipt of the latest FTD and the orphan drug status, Genprex is looking forward to initiating the Acclaim-3 clinical trial, planned for the fourth quarter of 2023

Genprex (NASDAQ: GNPX), a clinical-stage company developing life-changing gene therapies for patients with cancer and diabetes, recently celebrated an important milestone: the award of an orphan drug designation. The company announced August 10 that the United States Food and Drug Administration (“FDA”) had granted Orphan Drug Designation (“ODD”) to its lead drug candidate, REQORSA(R) Immunogene Therapy (quratusugene ozeplasmid), for the treatment of small cell lung cancer (“SCLC”) (https://ibn.fm/J4gpJ).

The FDA grants orphan drug status to a drug or biological product that prevents, diagnoses, or treats a rare disease or condition as part of the agency’s efforts to prioritize the development and evaluation of new treatments for rare diseases (https://ibn.fm/0PN7j). The ODD grants a sponsor – in this case, Genprex – incentives such as tax credits for qualified clinical trials and an exemption from user fees. Perhaps most importantly, the orphan drug designation grants potential seven-year exclusivity post-approval. The global SCLC therapeutics market is projected to grow at a CAGR of 12.3% from $5.12 billion in 2022 to $16.33 billion by 2032 (https://ibn.fm/y50nf).

The Orphan Drug Act (“ODA”) of 1983 defines rare diseases as those affecting “less than 200,000 persons in the United States.”  The FDA interprets this to mean that the prevalence of the disease, that is the number of people affected by the disease at a particular point in time, must be less than 200,000.  This is different than the incidence of the disease, which is the number of people newly diagnosed with SCLC during the course of a year (https://ibn.fm/cpEOA).

Based on the orphan drug definition SCLC is classified as a rare disease because the prevalence of SCLC is less than 200,000 cases in the US. The condition accounts for 13-15% of all lung cancer cases, and in the United States, about 238,440 adults will be diagnosed with lung cancer in 2023. The condition also presents a poor prognosis, partly because a majority of people – more than 60% – with SCLC are diagnosed after the cancer has spread outside the lung (https://ibn.fm/F7jpN). In such cases, the condition is known as extensive-stage SCLC (“ES-SCLC”).

Patients with ES-SCLC are given combination chemotherapy as the first-line treatment, with varying overall response rates of 44-78% and median progression-free survival (“mPFS”) that ranges between 4.3 and 5.7 months, depending on the selected combination (https://ibn.fm/gyNaW). Patients also undergo maintenance therapy, an ongoing cancer treatment, after the initial response to the first recommended treatment. The mPFS for patients receiving Genentech, Inc.’s Tecentriq(R) as maintenance treatment is only 2.6 months from the start of maintenance treatment, suggesting a substantial need for improvement in maintenance therapy, according to Genprex.

Genprex’s efforts to meet this need received a boost in June 2023 when the FDA granted Fast Track Designation (“FTD”) for REQORSA Immunogene Therapy, in combination with Tecentriq in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment (https://ibn.fm/wsBrh).

“We are excited to receive Orphan Drug Designation from the FDA for REQORSA for patients with SCLC,” commented Rodney Varner, President, Chairman, and CEO of 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 non-small cell lung cancer.”

Genprex’s lung cancer programs now have three FDA Fast Track Designations. In January 2020, the company announced that the FDA had granted an FTD for REQORSA in combination with AstraZeneca’s Tagrisso(R) for patients with NSCLC whose condition progressed after treatment with Tagrisso (https://ibn.fm/aSxQG). Later, in January 2022, the company announced it had received an FTD for REQORSA Immunogene Therapy in combination with Merck & Company’s Keytruda(R) in patients with NSCLC whose disease progressed after treatment with Keytruda (https://ibn.fm/L7Z1S).

With the receipt of the latest FTD and the orphan drug status, Genprex is looking forward to initiating the Acclaim-3 clinical trial, expected in the fourth quarter of 2023. Acclaim-3 is a Phase 1/2 open-label, dose escalation and clinical response study of maintenance therapy evaluating REQORSA in combination with Tecentriq in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment.

For more information, visit the company’s website at www.Genprex.com.

NOTE TO INVESTORS: The latest news and updates relating to GNPX are available in the company’s newsroom at http://ibn.fm/GNPX

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