Precigen’s Experimental RRP Treatment Shows Early Signs of Promise
By Alex Keown
January 27, 2023
Precigen’s early-stage immunotherapy treatment for patients with recurrent respiratory papillomatosis (RRP) is showing early signs of promise.
In January, the Germantown-based company announced positive data from a Phase I dose escalation and expansion cohort for the potential first-in-class PRGN-2012 off-the-shelf (OTS) AdenoVerse. Data shared by Precigen during its R&D Day presentation showed treatment with PRGN-2012 significantly reduced the need for surgeries for severe, aggressive RRP patients who received Dose Level 2 of the medication.
Precigen announced 50% of the patients saw a Complete Response, which was defined as no surgeries needed during the 12 month period following treatment. The company noted that the patients at Dose Level 2 saw a 58% overall response rate. Additionally, 83% of patients had reduced need for surgeries during the 12 months following dosing. Overall, the number of surgeries in Dose Level 2 patients following treatment declined from a median of 6.5 surgeries to .5 surgeries.
Helen Sabzevari, Chief Executive Officer of Precigen, expressed excitement at the shared data. She said PRGN-2012 has the potential to improve the lives of patients with severe, aggressive RRP through reduced surgeries.
“Any treatment that reduces the burden of surgeries in RRP is considered meaningful and in the PRGN-2012 Phase 1 study, 50% of patients had a Complete Response, requiring no surgeries as of the data cutoff with a minimum of 12 months following treatment,” Sabzevari said in a statement.
RRP is a rare neoplastic disease of the upper and lower respiratory tracts that is caused by infection of types of the human papillomavirus, specifically HPV 6 or HPV 11. Precigen’s Phase I data also showed treatment with that PRGN-2012 treatment resulted in an increase in HPV 6/11-specific T-cell response, the company noted.
The disease is typically characterized by recurrent wart-like growths on the surface of the vocal cords, trachea and lungs, or tissues around the organs, according to Johns Hopkins. Patients with an aggressive form of the disease can undergo hundreds of surgeries to control the growths. The disease can be fatal due to airway obstruction or loss of lung volume.
Repeated surgeries to treat RRP expose patients to surgical risks. The procedures also pose a significant economic burden to families and the healthcare system overall, Sabzevari said.
PRGN-2012 is an experimental vaccine that uses Precigen’s gorilla adenovector technology, part of Precigen’s proprietary AdenoVerse platform. The technology is designed to provoke immune responses directed against cells infected with HPV 6 or HPV 11. In preclinical models, PRGN-2012 demonstrated “strong and specific immune response” against HPV 6 and HPV 11, the company said. In 2021, the FDA granted Orphan Drug Designation to PRGN-2012.
Precigen is now advancing Dose Level 2 in a Phase II trial. The company has currently enrolled 20 patients and is adding up to 12 more. The trial will assess PRGN-2012 as an immunotherapy following standard-of-care surgery in adult patients with RRP.
According to the Recurrent Respiratory Papillomatosis Foundation, there are more than 15,600 global cases of the disease. In the U.S., there are an average of 115 new patients diagnosed each year. Annual cost of treatment is approximately $72,000. There are currently four clinical trials associated with the disease, according to the foundation.
“There has never been a therapeutic option for the RRP patient community and we are incredibly hopeful that this will change in the near future. Our community faces ongoing risks from hospitalizations and repeat surgeries, coupled with daily quality-of-life challenges, such as obstructed breathing, difficulties swallowing, and impaired speech, and our community bears a tremendous financial burden from the significant lifetime costs to patients and their families affiliated with this disease,” Kim McClellan, president of the RRPF said in a statement.
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Alex Keown is a freelance journalist who writes about a variety of subjects including the pharma, biotech, and life science industries. Prior to freelancing, Alex has served as a staff writer and editor for several publications.
