The FDA has granted full approval to Precigen Inc.'s PAPZIMEOS (zopapogene imadenovec-drba), marking a historic milestone as the first and only treatment approved for adults with recurrent respiratory papillomatosis (RRP). The approval sent Precigen's stock soaring over 50% in pre-market trading, reflecting the significance of this breakthrough for both patients and investors.
Addressing an Unmet Medical Need
RRP is a debilitating condition caused by certain types of HPV viruses that leads to tumor growth in the airways, affecting approximately 27,000 adults in the United States. Until now, patients have been trapped in a cycle of repeated surgeries that only manage symptoms without addressing the underlying cause. These procedures are necessary just to maintain breathing function and can cause voice problems that significantly impact daily life, with rare cases becoming life-threatening.
Revolutionary Gene Therapy Approach
PAPZIMEOS represents a paradigm shift in RRP treatment, utilizing an immunotherapy approach that trains the body's immune system to target HPV-infected cells directly. The treatment is administered as four doses over 12 weeks, leveraging Precigen's AdenoVerse platform, which uses a modified virus to deliver therapeutic agents without replication.
In the pivotal clinical study, 51% of patients achieved a complete response, defined as requiring no surgeries for at least one year following treatment. Remarkably, most of these patients remained surgery-free two years later, demonstrating the therapy's durable efficacy. The safety profile was manageable, with side effects primarily consisting of injection site pain, fatigue, and chills, with no major safety concerns identified in the trials.
Market Impact and Investor Response
The approval came faster than expected, as Precigen was initially on an accelerated approval pathway but received full approval without requiring additional confirmatory studies. This development has positioned the Maryland-based biopharma company as a focal point for biotech investors, with the stock jumping from approximately $1.85 at the previous close to about $2.65 in pre-market trading.
Wall Street analysts have responded positively to the company's prospects, with four firms issuing buy ratings and no sell ratings. Recent analyst price targets include $8.50 from HC Wainwright & Co., with a median target of $6.00 across three analysts who have issued targets in the last six months.
Commercial Readiness and Future Outlook
Precigen is already preparing for commercial launch, developing patient support programs and promotional strategies for PAPZIMEOS. The company's CEO described the approval as a "historic turning point," while patient advocates have expressed enthusiasm about having real hope after decades of surgical management approaches.
The approval validates Precigen's precision medicine platform and could serve as a stepping stone for other therapies in the company's development pipeline. However, as with any rare disease treatment, commercial success will depend on factors including insurance coverage adoption, physician uptake, and the relatively small patient population of 27,000 adults in the U.S.
Institutional investors have shown mixed activity, with 69 adding shares and 55 decreasing positions in recent quarters. Notable recent moves include Parkman Healthcare Partners adding over 3 million shares and Schonfeld Strategic Advisors adding nearly 2.8 million shares in Q1 2025.
