Inovio’s DNA-Based Immunotherapy Lands Speedy FDA Nod for Rare Type of Tumor

A rare type of tumor that requires frequent surgical intervention now has a new FDA-approved drug, an Inovio Pharmaceuticals therapy that prompts an immune response against the disease.

The regulatory decision announced after Friday’s market close covers the treatment of adults with recurrent respiratory papillomatosis (RRP) as way to reduce the frequency of surgeries to manage the chronic condition. Inovio’s therapy, known in development as INO-3107, will reach the market bearing the brand name Voceryq. It’s the first FDA-approved product for Plymouth Meeting, Pennsylvania-based Inovio and just the second drug approval in this rare indication. But Inovio executives claim their product has a key competitive advantage.

RRP develops from infection by human papillomavirus (HPV) -6 or HPV-11. Patients develop small tumors called papillomas in the respiratory tract. While benign, the papillomas impair the ability to speak and cause airway obstruction that can become life threatening. Surgical removal has been the standard of care for years, but does not address the underlying infection. Consequently, papillomas return and require repeated surgeries, sometimes multiple times per year. In addition to the medical costs, each procedure introduces the risk of damage to the respiratory tract and vocal cords.

Inovio developed Voceryq to address the infection driving RRP. The product is an immunotherapy composed of two plasmids, which are DNA molecules. One plasmid codes for antigens to the virus and the other codes for the signaling protein IL-12, which enhances the immune response. Together the plasmids are intended to spark an immune response against HPV-6 and -11 proteins in RRP patients. The course of treatment is one dose every three weeks for a total of four doses.

Inovio’s FDA submission was based on data from an open-label Phase 1/2 study. Of the 32 adults with RRP enrolled in the trial, 26 required three or more surgeries in the 12 months prior to receiving the experimental treatment. Inovio reported seven of these 26 showed a complete response, defined as requiring no surgery after 12 months of follow up. Overall, the trial showed a statistically significant reduction in surgical interventions compared to the prior year before patients received the study drug. Detailed results were published last year in Nature Communications and The Laryngoscope.

The FDA decision for Voceryq is an accelerated approval based on Phase 1/2 data. Inovio must conduct a confirmatory study to verify the therapy’s clinical benefit. The FDA also approved a proprietary Inovio device used to administer the DNA-based medicine.

Voceryq does not eliminate the need for surgery. Patients still needed to have debulking surgery to remove papillomas before receiving the immunotherapy. That’s also a requirement for Papzimeos, the Precigen immunotherapy that last year became the first FDA-approved drug for RRP. Papzimeos uses a gorilla adenoviral vector carrying a genetic payload intended to get cells to express a fusion antigen that elicits an immune response to HPV-6 and -11.

While the regulatory decision for Precigen’s drug was a traditional FDA approval versus the accelerated approval for Voceryq, Inovio CEO Jacqueline Shea contends her company’s product has a competitive advantage. Precigen’s label calls for additional surgeries before the third and fourth doses if papillomas are visible. Shea noted that Voceryq does not require surgeries prior to doses three or four.

“We designed the trial this way because every surgery matters to patients,” Shea said during a Monday morning conference call.

Based on an analysis of insurance claims data, Inovio estimates that the U.S. market for RRP totals about 25,000 adults. Shea noted that Voceryq’s label covers adult RRP patients broadly and does require a set number of prior surgeries to determine eligibility. Acknowledging the availability of Precigen’s product, Shea said that Inovio’s market analysis found many RRP patients are seeking alternatives to surgery, yet only a small fraction of these patients have received treatment with immunotherapy. Those patients remain available for Inovio to reach with Voceryq, she said.

Inovio set a $115,000 per dose list price for Voceryq, or $460,000 for the complete four-dose series. Chief Commercial Officer Steve Egge said that price noted is consistent with Precigen’s RRP immunotherapy. The company expects Voceryq will become available in the next 60 days. Inovio is also looking at potentially seeking additional regulatory approvals in Europe, the United Kingdom, and other markets.

As of the end of June, Inovio reported its cash position was $36.7 million. In July, the company raised $20 million in a securities offering. Shea said Inovio is fully funded for Voceryq’s product launch with enough money to last into the first quarter of 2027. She acknowledged that Inovio will need more capital and said the company is evaluating its options.

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