Precigen (PGEN) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Leadership and platform advancements

• Seasoned leadership has advanced a diverse gene and cell therapy portfolio over the past four years.

• Proprietary Gorilla AdenoVerse platform enables high-capacity, repeat-dosing gene therapies with strong CD8 responses.

• Manufacturing capabilities include GMP and cGMP facilities, ensuring rapid scale-up for commercialization.

PRGN-2012 for recurrent respiratory papillomatosis (RRP)

• PRGN-2012 is positioned to be the first approved therapy for RRP, a rare, devastating HPV-driven disease with no current treatments.

• Achieved FDA breakthrough designation and accelerated approval pathway; BLA submitted in December 2024.

• Pivotal phase I/II data showed significant efficacy and favorable safety, with 51% complete response and durable surgery-free outcomes up to 36 months.

• Confirmatory single-arm trial is enrolling; FDA approval does not require trial completion, only initiation.

• Commercial launch targeted for second half of 2025, with cGMP readiness and a modest, focused sales force planned.

Market opportunity and commercial strategy

• U.S. RRP prevalence estimated at 27,000 patients, with over 125,000 ex-U.S.; multi-billion-dollar global opportunity.

• Payers recognize high unmet need and value; expect prior authorization with treat-to-label and inclusion/exclusion criteria.

• Pricing will align with gene therapy analogs; final price not yet disclosed.

• Sales force will target approximately 500 key otolaryngologists, mainly in academic and urban centers.