PRECIGEN, INC. Q4 FY2023 earnings call

AdenoVerse Platform

• Differentiated platform using gorilla adenovectors with high capacity, no preimmunity in humans allowing repeated dosing, and specialized manufacturing for high titers.

PRGN-2012

• Designed to target HPV 6 and 11 for RRP. Phase 1 trial had favorable safety (Grade 1/2 reactions), 50% complete responders (no surgery needed), 83% reduced surgeries. Phase 2 data to be presented in Q2 2024, BLA submission in H2 2024, and FDA gave breakthrough designation and accelerated path.

PRGN-2009

• In Phase 2 for head and neck cancer (recruiting) and cervical cancer (IND approved, recruiting), with potential in combination with pembro.

Manufacturing and Commercial

• Manufacturing facility in Germantown, MD on track, Head of Commercial Operations hired in September 2023, preparing for PRGN-2012 launch in 2025.

The transcript focuses on the AdenoVerse platform. The lead asset PRGN-2012 is for Recurrent Respiratory Papillomatosis (RRP), with Phase 1 trial showing 50% complete responders and 83% reduced surgeries post-vaccination. PRGN-2009 is in development for HPV-related cancers, with Phase 2 studies in head and neck and cervical cancer ongoing. No absolute financial performance or revenue contribution % is provided, but operational details of the platforms and assets are highlighted.

Phase 2 Data

• To present full Phase 2 data for PRGN-2012 in Q2 2024.

BLA Submission

• Intend to submit BLA for PRGN-2012 in H2 2024.

Manufacturing

• Manufacturing facility on track to have capacity for PRGN-2012 launch in 2025.

Confirmatory Trial

• Consideration of a repeat dosing arm in the confirmatory trial, not a FDA requirement but to expand label.

Regulatory

• Uncertainties in BLA submission process.

Commercial

• Challenges in meeting patient demand at launch.

CAR-T Therapies

• Safety concerns with classical CAR-T in chronic diseases (e.g., autoimmunity) due to potential cancer risks and cost implications.

Q: Compare the trial design of PRGN-2012 Phase 1 and Phase 2 studies.

A: The design is exactly the same, and FDA allowed combining the Phase 1 single arm and Phase 2 single arm as pivotal.

Q: Plan for presenting Phase 2 data of PRGN-2012?

A: Will give more guidance soon, considering various options for presentation.

Q: Commercial readiness on manufacturing for PRGN-2012?

A: Manufacturing facility is on track, with capacity to meet patient needs at launch.

Q: Repeat dosing arm for PRGN-2012 confirmatory trial?

A: Design is up to them, not a FDA requirement, but to expand label and patient population受益 from repeat dosing.

Q: UltraCAR-T data and partnership?

A: Interim data on UltraCAR-T to be presented by end 2024, manufacturing is easy with overnight hospital-based production, and ongoing evaluation of partnership strategies.