Pharma Focus America

Precigen's PRGN-2012 AdenoVerse™ Immunotherapy Granted Breakthrough Therapy Designation for Recurrent Respiratory Papillomatosis Treatment

Wednesday, June 21, 2023

Precigen, Inc. (Nasdaq: PGEN) is a biopharmaceutical company dedicated to the development of innovative gene and cell therapies to improve the lives of patients. Recently, the company announced that its investigational PRGN-2012 AdenoVerse immunotherapy has received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) for the treatment of recurrent respiratory papillomatosis (RRP). This designation is granted to medicines that demonstrate substantial improvement on clinically significant endpoints over available therapies for serious or life-threatening diseases.

RRP is a condition characterized by the growth of benign tumors in the respiratory tract caused by human papillomavirus (HPV) infection. The current standard of care for RRP involves repeated surgical interventions, as there are no approved therapeutics available.

PRGN-2012 is a first-in-class immunotherapy that utilizes Precigen's AdenoVerse platform. It incorporates optimized antigen design using gorilla adenovector technology to elicit immune responses against HPV 6 and HPV 11-infected cells. Gorilla adenovectors offer advantages such as repeat administration capability, inability to replicate in vivo, and the ability to deliver a large genetic payload.

The Breakthrough Therapy Designation for PRGN-2012 was based on positive clinical evidence from a Phase 1 study. In this study, patients who had undergone an average of 5.8 RRP surgeries in the year prior to PRGN-2012 treatment showed a strong response at the recommended phase 2 dose (RP2D). Six out of twelve patients achieved a complete response, with no post-treatment surgeries required after a minimum follow-up of 12 months. In the 12 months following treatment, there was a reduction in surgeries for 83% of patients. PRGN-2012 also induced a robust de novo HPV-specific T-cell immune response and demonstrated a favorable safety profile.

Previously, PRGN-2012 had received Orphan Drug Designation from the FDA for the treatment of RRP. Currently, the therapy is being evaluated in a Phase 2 study involving adult patients with RRP. Precigen has completed enrollment in the Phase 2 study and is engaged in discussions with the FDA regarding potential expedited development paths to support a future submission of a Biologics License Application (BLA).

Precigen's mission is to advance medicine with precision by leveraging its innovative technologies in gene and cell therapy. The company focuses on therapeutic areas such as immuno-oncology, autoimmune disorders, and infectious diseases. By progressing therapies from preclinical stages to clinical proof-of-concept and commercialization, Precigen aims to provide innovative and affordable biotherapeutics in a controlled manner.

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