Precigen’s Papzimeos (zopapogene imadenovec-drba) has been approved by the FDA as the first therapy for adults with recurrent respiratory papillomatosis (RRP). Until now, the standard of care consisted mainly of repeated surgeries to remove papillomas, with frequent recurrence. Papzimeos introduces a new treatment option that directly targets the disease mechanism.
Clinical Outcomes
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Papzimeos (PRGN-2012, Phase 1/2, NCT04724980):
- 51% of patients required no surgery for one year after treatment.
- Over 85% experienced a reduction in surgical procedures compared to the year before treatment.
- Side effects were mostly mild to moderate (injection site reactions, fatigue, chills, fever) with no severe treatment-related events.
- A confirmatory Phase 3 trial (NCT06538480) is ongoing.
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Competitive Landscape – INO-3107 (Inovio):
- In Phase 1/2, about 28% of patients were surgery-free in year one, with improvements seen in year two.
- Well tolerated, mostly mild local side effects.
- Currently not approved; a confirmatory trial is in planning.
Commercial Outlook
Precigen is preparing the U.S. launch and global expansion of Papzimeos with several strategic moves:
- Leadership: Appointment of Phil Tennant as Chief Commercial Officer (July 2024). Tennant brings over 30 years of experience from Merck, AstraZeneca, BMS, and Astellas, highlighting Precigen’s focus on execution.
- Financing: A $125 million non-dilutive credit facility secured with Pharmakon Advisors (100M available upfront, 25M through 2027) to fund commercialization and expansion.
- Partners:
- EVERSANA to support commercialization in the U.S. (market access, field deployment, medical affairs).
- Catalent under a multi-year supply agreement to ensure manufacturing capacity.
- Market: Estimated 27,000 adult RRP patients in the U.S.. Pricing is significantly higher than earlier ~$200K per-patient assumptions, reflecting the value proposition of reducing surgeries.
- Challenges: Small patient population, payer negotiations, and the need for physician awareness and diagnosis remain hurdles.
Analyst projections differ widely:
- H.C. Wainwright: around $1.1 billion peak sales (2033).
- J.P. Morgan: about $250 million in the U.S. market.
Conclusion
Papzimeos demonstrates strong efficacy, with half of patients surgery-free for a year, and a favorable safety profile. With FDA approval, an ongoing Phase 3 trial, experienced commercial leadership, strong financing, and strategic partners, Precigen is well positioned to establish itself as the leader in RRP treatment. Competitors like Inovio are progressing, but Papzimeos currently holds the advantage of being both first-to-market and backed by compelling clinical data.