Precigen Reveals Pivotal PRGN-2012 Study Data: Over Half Achieve Complete Response in Recurrent Respiratory Papillomatosis

Germantown, MD, June 3, 2024 – Precigen, Inc., a biopharmaceutical company focused on advanced gene and cell therapies, has announced successful outcomes from their Phase 1/2 pivotal study of PRGN-2012 for treating recurrent respiratory papillomatosis (RRP). The results were unveiled at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting by Dr. Scott M. Norberg of the National Cancer Institute.

Study Highlights

The study aimed to evaluate both the safety and efficacy of PRGN-2012, an off-the-shelf (OTS) gene therapy. The trial enrolled 38 adult patients diagnosed with RRP, a chronic condition caused by HPV leading to recurrent benign tumors in the respiratory tract. These patients had undergone at least three surgeries in the year prior to joining the study.

Efficacy Outcomes

The primary goal was to assess complete response (CR) rates and compare the number of surgeries required before and after treatment. Remarkably, 51% of the participants achieved a CR, meaning they did not require any surgeries in the 12 months following treatment. The CRs were also durable, with a median follow-up duration of 20 months, and no median durability of response has been reached as of yet.

Furthermore, 86% of the patients experienced a significant reduction in the number of surgeries needed post-treatment compared to the year before. The median number of surgeries dropped from 4 before treatment to 0 after treatment. The therapy also significantly improved Derkay scores, which measure the extent of papilloma growth, and the Vocal Handicap Index-10 (VHI-10), a scale for assessing the quality of life related to vocal issues.

Safety Profile

PRGN-2012 was well-tolerated among participants. There were no severe treatment-related adverse effects, with most side effects being mild to moderate. Common side effects included injection site reactions, fatigue, chills, and fever. Importantly, there were no dose-limiting toxicities, and no significant anti-drug antibody response was observed.

Mechanism of Action

The treatment works by inducing HPV 6/11-specific T cell responses, which are crucial for combating the disease. A significantly greater expansion of these T cells was observed in patients who responded to the treatment compared to non-responders.

Future Prospects

Given the favorable safety profile, strong efficacy, and ease of administration, PRGN-2012 is positioned to potentially become the leading treatment for RRP. Precigen is planning a rolling submission for a Biologics License Application (BLA) to the FDA under an accelerated approval pathway, with the submission expected in the second half of 2024. The company is preparing for a potential market launch in 2025, aiming to offer a long-awaited new treatment option for the RRP community.

Helen Sabzevari, Ph.D., President and CEO of Precigen, expressed optimism about the future of PRGN-2012. She emphasized the significant impact the therapy could have on patients' lives, reducing the need for repeated surgeries and improving quality of life.

Conclusion

The Phase 1/2 study results indicate that PRGN-2012 could revolutionize the treatment of RRP, offering a more effective and safer alternative to the current standard-of-care. With regulatory approval on the horizon, this therapy could soon provide a much-needed solution for patients suffering from this debilitating condition.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!