The first treatment for rare tumors is a sensational prospect, but does Precigen have the cash to achieve this?
Tumors caused by recurrent respiratory papillomatosis are benign, but this does not mean they are not serious. In this rare disease, growth formation in the respiratory tract limits the patient's ability to breathe and speak. The FDA recently approved a pubertal drug that explores the root causes of RRP, making immunotherapy the first drug treatment for the disease.
The first step advantage of Precigen therapy, known in development as PRGN-2012 and branded as Papzimeos, brought sensational sales expectations. But regulatory approval is just a barrier. For the past year, Precigen, based in the German town of Maryland, has been balancing the pipeline and financial position to support Papzimeos, now its first commercial product. As the company's regulatory documents point to financial challenges for the coming year, the balance is not over yet.
In RRP, chronic human papillomavirus (HPV) 6 or HPV 11 infection can lead to the growth of papilloma or benign tumors in the respiratory tract. These growth surgical removal is the standard of care, but this is not curative and requires repeated surgery throughout the patient's life. In rare cases, these tumors become cancerous. Precigen estimates that 27,000 adults in the U.S. and more than 125,000 patients outside the U.S. have RRP.
Papzimeos is a ready-made immunotherapy that uses gorilla adenovirus vector to provide a gene payload to patient cells. The inherited cargo causes cells to express a fusion antigen that causes an immune response to HPV6 and HPV11 infected cells. The company said the engineered viruses used for Papzimeos allow for reduced payload capacity for genetic cargoes compared to other viral vectors.
Precigen's submission seeks to accelerate FDA approval Papzimeos is based on a single-arm open-label study that recruited 35 adult RRP patients who require three or more surgeries per year. Immunotherapy was used as four subcutaneous injections within 12 weeks. Papzimeos does not completely exempt patients from surgery. Patients must perform surgical forgery of visible papillomas before receiving immunotherapy.
In a key part of the Stage 1/2 study, 51.4% of patients achieved a complete response, defined as no surgical intervention within 12 months after treatment. Most patients maintained these reactions within two years. Treatment-related issues were classified as mild to moderate, with no serious adverse events reported. During the August 18 call, CEO Helen Sabzevari said that other clinical data will be presented at the upcoming medical conference for up to 36 months.
Precigen said in a speech at the annual JP Morgan Healthcare conference in January that its immunotherapy could become a bombshell seller in RRP and also offers the potential to address other diseases powered by HPV6 and HPV11. After the FDA approved immunotherapy, Procigen sets the price per bottle at $115,000, or $460,000 for full treatment. Sabzevari said it would be up to doctors to determine whether patients need to make up, but she pointed out that the data for two years is evidence of the durability of immunotherapy. List the price is significantly higher than the estimated price of each patient with a citizen JMP. But, like the newly launched drugs, it takes some time before Precigen can start acknowledging income.
Precigen's financial situation is not strong. As of June 30, the company reported $59.7 million in its second quarter financial report released on August 12, with no additional funding source. Precigen said in the report that capital was insufficient to fund operations, and “these conditions and events have raised great doubts about the company’s ability to continue to worry.” Financial forecasts rule out potential revenue from Papzimeos, which will require cash to support the manufacturing and commercialization of newly approved immunotherapy. However, the FDA's decision was a standard approval that saved the original foundation from the regulatory and financial obligations of confirmatory clinical trials.
Stock products are one way Precigen can raise more money. Biotech's stock closed at $4.18 on Friday, 273% higher than stock trading a year ago, when the restructuring suspended preclinical work and cut 20% of its employees to focus the company on RRP treatment. Shortly after the restructuring, Procigen raised $30 million in stock to support its Papzimeos plan. Precigen stock's recent gains have not met Citizen JMP's forecast, which has raised its target target from $6 per share after the FDA approved it to increase its target target from $8.50, while HC Wainright maintains its price target for Precigen stock at $8.50.
By the end of 2024, Precigen raised $79 million in private fundraising with select investors, including company executive chairman Randal Kirk. With the approval of Papzimeos, Procigen's stock price rose, and these shareholders are now taking back their money and then selling the stocks. Precigen will not receive any proceeds from the sale.
Meanwhile, Precigen has the manufacturing of its new treatments. According to regulatory documents, just before the Papzimeos approval announcement, Procigen catalyzed a three-year supply agreement with the contract manufacturer. No financial terms were disclosed. Precigen CFO Harry Thomasian Jr. said in an emailed statement that Precigen has been executing a “very powerful and targeted” Papzimeos launch strategy, adding that it is fully funded and has been ongoing over the past few quarters.
“Our financial situation is related to the launch itself,” Thomas said.
Although Papzimeos is currently the first and only drug treatment for RRP that can be used for competitive drug treatment. Inovio's DNA plasmid-based immunotherapy INO-3107 also involves HPV6 and HPV11. Unlike Precigen's therapy, INO-3107 does not require a viral vector, which reduces the risk of the body producing antibodies to neutralize it.
Inovio plans to complete the rolling submission of INO-3107 “in the next few months”, the company said in its report on its second quarter 2025 financial results. But, like Precigen and many other biotech companies in the current challenging financial environment, Inovio has limited capital. Inovio reported $47.5 million in cash positions at the end of the second quarter, raising an additional $22.5 million in securities products in July. Suburban Philadelphia-based Biotech says it expects consolidated earnings to continue into the second quarter of 2026, a financial timeline similar to Precigen.
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