August 15, 2025
BIOINVEST NEWS: Precigen (PGEN)
Precigen (PGEN 3.250) – FDA Fully Approves PGEN’s PAPZIMEOS for RRR – Well Before PDUFA Date – BUY
PGEN has received FDA approval for PAPZIMEOS for the treatment of adults with recurrent respiratory papillomatosis (RRP). PAPZIMEOS is the first and only FDA-approved therapy for the treatment of adults with RRP. In a major positive for the Company, the approval came early as the FDA PDUFA date was Aug. 27. PGEN completed submission of the rolling Biologics License Application (BLA) in December under an accelerated approval pathway and received the approval in 8 months (https://www.fda.gov/news-events/press-announcements/fda-approves-first-immunotherapy-recurrent-respiratory-papillomatosis).
Full Approval Is Best Of All Outcome
The fact that the agency is working efficiently despite RFK’s medalling is yet another encouraging sign that the FDA working fine and in some cases – such as this one – may surprise on the plus side! The agency’s confidence in granting PAPZIMEOS full approval, despite a small trial size and more importantly – and unlike most “conditional approvals” – PGEN is not required to performa a confirmatory clinical trial (although they are doing one anyway https://clinicaltrials.gov/study/NCT06538480?term=Precigen). The FDA also approved the new treatment without holding an AdCom panel, another show of the agency’s confidence in both the drug’s safety and efficacy. In addition to leading to complete responses (i.e., no surgeries over the course of one year), the safety profile is unbelievably mild. As another positive reminder – PAPZIMEOS is fully-owned by PGEN and there currently there is NO DRUG Competition for Precigen – just expensive ablative surgeries which result in recurring papillomas.
RRP – Bigger Than People Think – No Need For Repeated Annual Surgeries
RRR is a nice size orphan market with approximately 27,000 adult RRP patients in the US. With an annual incidence of over 1,000. RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. RRP can lead to severe voice disturbance (sounds familiar?), a compromised airway, and recurrent post-obstructive pneumonias. Management of RRP has primarily consisted of repeated surgeries several times per year – an expensive that requires general anesthesia in a hospital setting. The surgeries which do not address the root cause of the disease and can be associated with significant morbidity as well as significant patient and health system burden.
The cost of surgery for Recurrent Respiratory Papillomatosis (RRP) can vary significantly, but it’s generally considered a high-cost condition. In the first two years, the cost difference between RRP cases and controls can range from about $5,000 to $76,000, depending on the patient’s age and insurance type. For juvenile-onset RRP, the cost difference can be much higher than for adult-onset RRP. We believe the U.S. potential market is between $500 million and $1 billion but we will know more as of Monday’s conference call. Insurance will easily cover this treatment as the both the disease and quality of life and overall cost burden (including the patientss need to take off work several times a year) is in the $100,000s per year.
HPV Market Potentials – RRP Is Just The First
PAPZIMEOS is the first and only FDA-approved therapy for the treatment of adults with RRP and the first and only approved therapy to address the root cause of RRP. PAPZIMEOS is a non-replicating adenoviral vector-based immunotherapy designed to express a fusion antigen comprising selected regions of human papillomavirus (HPV) types 6 and 11 proteins. PAPZIMEOS is delivered via four subcutaneous injections over a 12-week interval. The FDA approval also provides validation for PGEN’s AdenoVerse therapeutic platform. The Company is also developing PRGN-2009 in cervical cancer (https://clinicaltrials.gov/study/NCT06157151?cond=HPV&term=PRECIGEN &rank=1) – this trial was just posted on August 1.
Conference Call Monday 8AM EST – BUY
PGEN is executing very well with their first FDA approval. We look forward to the conf call for additional launch details. In fact, we believe the Company may have been surprised by the early approval – hence, the Monday AM conference call. Either way, it is the best news for Precigen that we could imagine. We would not be surprised at all if the Company raises capital on the good news – it is not only needed but deserving after such fundamentally good news. The stock may very well have some room to run as it is heavily shorted. The latest short interest was 28.3 million, 19.71% of the float. The short interest ratio (days to cover) is 12.3 days of average trading volume to cover the short.
We are BUYers into the good news of an under-owned and undervalued biotech stock that’s had a long strange trip and bumpy road to its first approval.