Breakthrough Therapy Designation Granted to Precigen's PRGN-2012 AdenoVerse™ Immunotherapy
Precigen, Inc. (Nasdaq: PGEN) has announced that the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to their investigational PRGN-2012 AdenoVerse immunotherapy for the treatment of recurrent respiratory papillomatosis (RRP). This designation expedites the development and review of medicines that target serious or life-threatening diseases and demonstrate significant improvement over existing therapies.
Currently, the standard-of-care for RRP involves repeated surgical interventions, with no approved therapeutics available. The potential of PRGN-2012 to reduce the need for surgeries and improve outcomes for RRP patients has led to the pride of Precigen in receiving the Breakthrough Therapy Designation. It enables direct engagement with senior FDA leadership to facilitate the most efficient product development pathway, including eligibility for rolling and priority review of a Biologics License Application (BLA) to support potential PRGN-2012 registration.
PRGN-2012 utilizes optimized antigen design through gorilla adenovector technology, a part of Precigen's proprietary AdenoVerse platform, to elicit immune responses against cells infected with human papillomavirus type 6 (HPV 6) or HPV type 11 (HPV 11). Gorilla adenovectors offer advantages such as repeat administration capability, inability to replicate in vivo, and the ability to deliver a large genetic payload. FDA previously granted PRGN-2012 Orphan Drug Designation for RRP patients.
The Breakthrough Therapy Designation is based on clinical evidence generated in the Phase 1 study (NCT04724980), presented during Precigen's recent R&D day. The study demonstrated a strong response at the recommended phase 2 dose (RP2D) in patients who had an average of 5.8 RRP surgeries in the year prior to PRGN-2012 treatment. Notably, 50% of patients achieved a complete response, requiring no post-treatment surgeries, and 83% experienced a reduction in surgeries during the 12 months following treatment. PRGN-2012 also induced robust de novo HPV-specific T-cell immune responses and exhibited a favorable safety profile.
Currently, PRGN-2012 is undergoing evaluation in a Phase 2 study involving adult RRP patients, with enrollment completed for 23 patients at the RP2D. Patient follow-up is ongoing, and discussions with the FDA regarding potential rapid development paths for a future BLA submission are in progress.
Precigen (Nasdaq: PGEN) is a biopharmaceutical company dedicated to advancing gene and cell therapies for immuno-oncology, autoimmune disorders, and infectious diseases. RRP, a condition lacking a cure, necessitates repeated surgical interventions, posing risks and emotional distress to patients. The PRGN-2012 AdenoVerse immunotherapy, utilizing Precigen's gorilla adenovector technology, holds promise in eliciting targeted immune responses against HPV 6 and HPV 11-infected cells.
