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Shares of Precigen PGEN have soared nearly 43% in the past week after the company provided an encouraging regulatory update for its lead pipeline program.
Last week, Precigen announced that it has completed a rolling submission for a biologic license application (BLA) seeking the FDA’s accelerated approval for PRGN-2012, an experimental gene therapy to treat adults with recurrent respiratory papillomatosis (RRP).
A rare disease, RRP causes benign tumors (called papillomas) to grow in the respiratory tract, most commonly in the larynx (voice box). It is caused by two types of human papilloma virus (HPV) – HPV 6 and HPV 11.
More on PGEN’s BLA Filing for PRGN-2012
The BLA is supported by data from a pivotal phase I/II study that evaluated PRGN-2012 for the treatment of adult patients with RRP. Data from the study showed that more than 50% of patients achieved a complete response (defined as the percentage of patients who require no RRP surgeries). Also, more than 85% of patients experienced a reduction in surgical interventions in the year after receiving PRGN-2012 compared to the year before receiving the therapy.
The regulatory filing is currently in the initial 60-day review period and the FDA will decide whether to accept the application for further review and set the target action by this period. Management has requested a priority review for this filing, which, if granted, will reduce the agency’s review period by four months.
If the FDA approves this filing, PRGN-2012 will be the first FDA-approved therapeutic for adults with RRP. The disease currently has no cure, and the standard treatment involves repeated surgeries. However, these procedures only manage symptoms and do not address the root cause of the disease, often leading to a cycle of recurrences. It has been observed that patients with RRP frequently require surgeries over their lifetime.
Management expects a potential approval for PRGN-2012 before this year’s end. The gene therapy has already received breakthrough therapy and orphan drug designation from the FDA in RRP indication.
PGEN Stock Performance
In the past year, Precigen’s shares have lost 13.9% compared with the industry’s 13.6% fall.
Image Source: Zacks Investment Research
Other Players in the RRP Space
Another company developing its own RRP medication is Inovio INO, which is evaluating INO-3107 in a pivotal phase I/II study. However, the Inovio drug is an investigational DNA medicine designed to generate an antigen-specific T-cell response against both HPV-6 and HPV-11 proteins.
Management plans to submit a regulatory filing with the FDA in mid-2025 seeking accelerated approval for the drug in the RRP indication.