Precigen (PGEN) Stifel 2024 Healthcare Conference summary

Company overview and platforms

• Focused on innovative cell and gene therapies for unmet needs since 2020, with unique Gorilla AdenoVerse and UltraCAR-T platforms.

• Gorilla AdenoVerse enables repeat dosing and immune enhancement, currently in phase II for oncology and infectious diseases.

• UltraCAR-T offers overnight, lower-cost, autologous cell manufacturing with safety switches, aiming for rapid patient treatment.

• Exciting new data for UltraCAR-T expected in 2025.

• PRGN-2012 (PAPZIMEOS) is advancing toward BLA submission and potential commercialization by year-end.

Disease background and patient landscape

• Recurrent Respiratory Papillomatosis (RRP) is a rare, devastating HPV 6/11-driven disease with no current treatment except repeated surgeries.

• U.S. prevalence is estimated at 20,000, with 125,000–150,000 cases in EU4, UK, Japan, and China; China has especially high prevalence.

• Diagnosis is often delayed or missed, leading to underestimation of true prevalence; increased awareness and advocacy are improving identification.

• Patients are treated in both academic centers and physician offices, with a trend toward concentration in specialized centers.

• Patient advocacy groups play a key role in education, awareness, and regulatory progress.

Current standard of care and unmet needs

• Standard care is repeated surgery, often starting in childhood, with some patients undergoing hundreds of procedures.

• Surgeries are costly, disruptive, and associated with significant quality-of-life and productivity losses; annual U.S. surgery costs alone are in the hundreds of millions.

• Existing prophylactic vaccines like Gardasil are ineffective for RRP, as they cannot generate sufficient high-affinity T cells in already-infected patients.

• Checkpoint inhibitors show limited efficacy and high toxicity, with no complete responders and only temporary reduction in surgeries.