Tumors caused by recurrent respiratory papillomatosis are benign, but that doesn’t mean they are not serious. In this rare disease, growths form in the respiratory tract that restrict a patient’s ability to breathe and speak. The FDA recently approved a Precigen drug that addresses the root cause of RRP, making the immunotherapy the first drug treatment for the disorder.
First-mover advantage for Precigen’s therapy, known in development as PRGN-2012 and branded as Papzimeos, brings blockbuster sales expectations. But regulatory approval is just one hurdle. In the past year, Germantown, Maryland-based Precigen has been performing a balancing act with its pipeline and its finances in order to support Papzimeos, now its first commercialized product. The balancing is not over, as the company’s regulatory filings point to financial challenges in the year ahead.
In RRP, chronic human papillomavirus (HPV) 6 or HPV 11 infection leads to growth of papillomas, or benign tumors, in the respiratory tract. Surgical removal of these growths is the standard of care, but it is not curative and requires repeated procedures over a patient’s lifetime. In rare cases, these tumors can 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 an off-the-shelf immunotherapy that uses a gorilla adenoviral vector to deliver a genetic payload to patient’s cells. This genetic cargo gets cells to express a fusion antigen that elicits an immune response against cells infected by HPV6 and HPV11. The company says the engineered virus used for Papzimeos permits redosing and offers a larger payload capacity for genetic cargo compared to other viral vectors.
Precigen’s submission seeking accelerated FDA approval for Papzimeos was based on a single-arm, open-label study that enrolled 35 adult RRP patients who required three or more surgeries per year. The immunotherapy was administered as four subcutaneous injections over 12 weeks. Papzimeos does not completely spare patients from surgery. Patients must undergo surgical debulking of visible papillomas before receiving the immunotherapy.
In the pivotal portion of the Phase 1/2 study, 51.4% of patients achieved a complete response, defined as no surgical interventions in the 12 months following treatment. Most patients maintained these responses through two years. Treatment-related problems were classified as mild to moderate and no serious adverse events were reported. During an Aug. 18 conference call, CEO Helen Sabzevari said additional clinical data up to 36 months will be presented at an upcoming medical conference.
In a presentation at the annual J.P. Morgan Healthcare Conference in January, Precigen said its immunotherapy could become a blockbuster seller in RRP and also offers the potential to address other diseases driven by HPV6 and HPV11. Following the FDA approval of the immunotherapy, Precigen set a $115,000 per vial list price, or $460,000 for a full course of treatment. Sabzevari said it will be up to physicians to determine whether a patient needs redosing, but she pointed to the two-year data as evidence of the immunotherapy’s durability. The list price is significantly higher than the $200,000 per patient price estimate from Citizens JMP. But as is the case with newly launched drugs, it will take time for Precigen to start recognizing revenue.
Precigen’s financial position is not strong. In Its second quarter financial report posted Aug. 12, the company reported a cash position of $59.7 million as of June 30 and no committed source of additional funding. The capital is not enough to fund operations for one year, and “these conditions and events raise substantial doubt about the Company’s ability to continue as a going concern,” Precigen said in the report. The financial projection excluded potential revenue from Papzimeos, and the company will need cash to support manufacturing and commercialization of the newly approved immunotherapy. However, the FDA decision was a standard approval, which spares Precigen from the regulatory and financial obligations of a confirmatory clinical trial.
A stock offering is one way Precigen could raise more money. Shares of the biotech closed at $4.18 each on Friday, which is 273% higher than where its shares were trading a year ago when a restructuring paused preclinical work and cut 20% of staff to focus the company on the RRP therapy. Shortly after the restructuring, Precigen raised $30 million in a stock offering to support its plans for Papzimeos. The recent rise of Precigen’s stock is still short of the projection from Citizens JMP, which raised its price target after the FDA approval from $6 to $8 per share, and H.C. Wainright, which maintained an $8.50 price target for Precigen’s shares.
At the end of 2024, Precigen raised $79 million in a private placement with select investors, including Randal Kirk, the company’s executive chairman. With the rise of Precigen’s stock price following the Papzimeos approval, those shareholders are now getting their money back and then some by selling those shares. Precigen will not receive any proceeds from that sale.
Meanwhile, Precigen has manufacturing of its new therapy secured. Just prior to the Papzimeos approval announcement, Precigen entered into a three-year supply agreement with contract manufacturer Catalent, according to a regulatory filing. No financial terms were disclosed. In an emailed statement, Precigen Chief Financial Officer Harry Thomasian Jr. said Precigen has been executing “a very robust and targeted” Papzimeos launch strategy, which he added is fully funded and has been ongoing for the last few quarters.
“We feel very good with our financial situation related to the launch itself,” Thomasian said.
While Papzimeos is the first and only drug treatment available for RRP right now, competition looms. Inovio’s DNA plasmid-based immunotherapy, INO-3107, also addresses both 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 a rolling submission for INO-3107 “in the next several months,” the company said in its report of second quarter 2025 financial results. But like Precigen and many other biotech companies in the current challenging financial environment, Inovio has limited capital. Inovio, which reported a cash position of $47.5 million at the end of the second quarter, raised an additional $22.5 million in a July securities offering. The suburban Philadelphia-based biotech said it expects the combined proceeds will last into the second quarter of 2026 — a financial timeline similar to Precigen’s.
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