Precigen receives Orphan Drug Designation for PRGN-2012 in Europe

Precigen announced that the European Commission, or EC, has granted Orphan Drug Designation for the Company’s first-in-class investigational medicine PRGN-2012 for the treatment of recurrent respiratory papillomatosis, or RRP. RRP is a rare, difficult-to-treat and sometimes fatal neoplastic disease of the upper and lower respiratory tracts caused by human papillomavirus type 6 or HPV type 11. PRGN-2012 received Orphan Drug Designation from the FDA and Precigen was the first company to receive Breakthrough Therapy Designation and an accelerated approval pathway from the FDA for an RRP treatment. PRGN-2012 is an innovative therapeutic vaccine with optimized antigen design that uses gorilla adenovector technology, part of Precigen’s proprietary AdenoVerse platform, to elicit immune responses directed against cells infected with HPV 6 or HPV 11. Gorilla adenovectors have numerous advantages, including the ability for repeat administration, the inability to replicate in vivo, and the ability to deliver a large genetic payload. PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP in the US. A Phase 2 data presentation is anticipated in the second quarter of 2024 and a planned Biologics License Application submission under an accelerated approval pathway with the FDA is anticipated in the second half of 2024. Commercial readiness preparations are underway for a potential launch in the US in 2025.