The results of a pivotal phase I/II study for PRGN-2012, an investigational gene therapy for recurrent respiratory papillomatosis (RRP), were recently published in The Lancet Respiratory Medicine.1 The study demonstrated that over 50% of participants achieved a complete response, requiring no surgical interventions for at least 12 months post-treatment. Additionally, the therapy showed a strong safety profile, with no dose-limiting toxicities reported. These findings form the basis of Precigen, Inc.’s Biologics License Application (BLA), which has been submitted to the U.S. Food and Drug Administration (FDA) along with a request for priority review. 1
RRP is characterized by benign tumors in the respiratory tract, which can cause voice changes, airway obstruction, and severe respiratory distress.2 The standard treatment involves repeated surgeries to manage symptoms but does not address the underlying HPV infection.2 PRGN-2012 is an innovative therapy designed to stimulate immune responses against cells infected with human papillomavirus (HPV) types 6 and 11, the primary drivers of RRP. The therapy has received both Breakthrough Therapy Designation and Orphan Drug Designation from the FDA and the European Commission, underscoring its potential to address significant unmet medical needs in treating this rare, chronic disease.3 If approved, PRGN-2012 would become the first FDA-approved therapeutic option for adult patients with RRP, potentially changing the management of this challenging condition.
The BLA submission is bolstered by data from a study involving 38 adult patients with RRP. Over 85% of patients experienced a reduction in surgical interventions in the 12 months following treatment compared to the prior year. The therapy’s tolerability and the absence of treatment-related adverse events greater than Grade 2 further support its safety profile.
The FDA is currently in the initial 60-day review period to determine whether to accept the BLA and grant priority review. If the application is approved, Precigen anticipates a commercial launch of PRGN-2012 in the second half of 2025. Of course, the priority review designation could significantly expedite this timeline.
The therapy’s demonstrated efficacy and safety profile have the potential to provide a new treatment solution for RRP patients, addressing an urgent unmet medical need.
- Norberg, Scott M et al. PRGN-2012 gene therapy in adults with recurrent respiratory papillomatosis: a pivotal phase 1/2 clinical trial, Lancet Resp Med. 2025; epub ahead of print. DOI: 10.1016/S2213-2600(24)00368-0
- Bertino G, Pedretti F, Mauramati S, et al., Recurrent laryngeal papillomatosis: multimodal therapeutic strategies. Literature review and multicentre retrospective study. Acta Otorhinolaryngol Ital. 2023;43(2 Suppl 1):S111–S122. DOI: 10.14639/0392-100X-suppl.1-43-2023-14.
- Precigen Receives Breakthrough Therapy Designation for PRGN-2012 AdenoVerse™ Immunotherapy for the Treatment of Recurrent Respiratory Papillomatosis. Precigen. Available at: https://investors.precigen.com/news-releases/news-release-details/precigen-receives-breakthrough-therapy-designation-prgn-2012 (accessed 27th January 2025).
Disclosures: This article was created by the touchRESPIRATORY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
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