Inovio Pharmaceuticals (NASDAQ:INO) said the U.S. Food and Drug Administration granted accelerated approval to VOCERYQ, formerly known as INO-3107, as a nonsurgical treatment intended to reduce the frequency of surgeries in adults with recurrent respiratory papillomatosis, or RRP.
VOCERYQ is the company’s first approved product and, according to Inovio, the first FDA-approved plasmid DNA-based immunotherapy. The treatment targets HPV-6 and HPV-11, viruses associated with RRP, a rare disease in which benign papillomas develop in the airways, throat and larynx. Patients can experience difficulty speaking, swallowing and breathing, and may require repeated surgical procedures to remove the growths.
“With accelerated approval and a broad adult indication to reduce the frequency of surgery, the FDA has recognized that impact and the therapeutic benefit of VOCERYQ,” President and Chief Executive Officer Jackie Shea said during an investor call.
Clinical Data and Product Administration
Chief Medical Officer Mike Sumner said VOCERYQ consists of two DNA plasmids: one encoding HPV-6 and HPV-11 E6 and E7 proteins, and another encoding human IL-12 to enhance immune response. The therapy is administered using Inovio’s CELLECTRA device, which injects the treatment into muscle tissue and applies short electrical pulses intended to facilitate entry into muscle cells.
The FDA’s efficacy analysis focused on 26 patients in the RRP-001 study who had required at least three surgeries in the 12 months before treatment. Those patients had a median of 4.5 surgeries in the prior year, including the surgical debulking procedure immediately before treatment began, Sumner said.
Patients received four doses over nine weeks. The FDA analysis began after the dosing period, and Inovio said 27% of the 26 patients achieved a complete response, defined as requiring no surgeries to manage the disease during the following 12 months.
The company said its treatment protocol and product label do not require scoping or surgery to remove visible papillomas during the nine-week dosing window. Inovio characterized VOCERYQ as generally well tolerated, with mostly low-grade treatment-emergent adverse events in the clinical study, including injection-site pain and fatigue.
Sumner said the FDA approval was based on the Accelerated Approval Program and that continued approval may depend on confirmation of clinical benefit in a post-approval study. The company expects to submit a draft protocol before year-end and has until the middle of next year to finalize the study design with the agency.
- The confirmatory study is expected to enroll at least 50 adult and pediatric RRP patients.
- Participants must have required three or more surgical interventions in the 12 months before treatment.
- The study is expected to measure surgical interventions for at least three years after treatment and compare results with an external control group or scientifically justified threshold.
Inovio said it may also evaluate continued treatment for patients who do not achieve a sustained complete response. The company has not yet finalized details of the confirmatory trial or its pediatric development approach, though Sumner said Inovio expects a stepwise process that may initially include adolescents.
Commercial Launch Plans
Chief Commercial Officer Steve Egge said Inovio estimates, based on its claims-data analysis, that the U.S. has a prevalent adult RRP population of at least 25,000 patients. He said immunotherapy has so far been used by only a small fraction of that population.
Inovio expects VOCERYQ to be available for ordering and shipment in about 60 days and anticipates full commercial field deployment in the first quarter of 2027. The company plans to price the therapy at approximately $115,000 per dose, or about $460,000 for the complete four-dose regimen.
The company said it expects insurers covering roughly 80% of U.S. patients to establish coverage policies within nine months of approval, based on its payer research. It plans to seek a permanent J-code, pursue medical-exception pathways and work with hospital and integrated delivery network pharmacy and therapeutics committees.
Inovio’s launch model will use field deployment partner Syneos Health. The company expects to establish 20 key-account-manager territories, supported by reimbursement specialists, payer capabilities and medical science liaisons. Egge said the commercial footprint is designed to cover approximately 90% of RRP patient potential.
About 400 laryngologists treat most RRP patients, according to the company, while approximately 80% of patients receive care at large integrated delivery networks or academic institutions. Inovio also said it has inventory and manufacturing capacity intended to support the first year of the launch through at least 2027.
Pipeline and Financing
Shea said Inovio plans to explore VOCERYQ in pediatric patients, additional treatment strategies and international markets, including the European Union and United Kingdom. The company also plans to advance its broader pipeline, including investigational protein replacement therapies for diseases such as hemophilia A, Fabry disease and hypophosphatasia.
During the question-and-answer session, Shea said the company’s current financing is expected to fund operations through the launch and into the first quarter of 2027. She added that Inovio will need to raise additional capital to fully fund the commercial launch and is evaluating potential funding options.
About Inovio Pharmaceuticals (NASDAQ:INO)
Inovio Pharmaceuticals, Inc is a biotechnology company focused on developing DNA medicines, including DNA-based vaccines and immunotherapies designed to treat or prevent infectious diseases, cancers and other serious conditions. The company’s platform is intended to stimulate targeted immune responses by delivering DNA medicines into cells.
Inovio’s product pipeline has included investigational therapies such as INO-3107, a DNA immunotherapy being developed for recurrent respiratory papillomatosis, and other candidates targeting cancer and infectious diseases.
