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Precigen Is Pioneering a Nonsurgical Treatment for a Debilitating Respiratory Disease; Positive Data Released on Recurrent Respiratory Papillomatosis Showed a 50% Complete Response
--News Direct--New hope may be on the horizon for sufferers of Recurrent Respiratory Papillomatosis, or RRP, a rare and debilitating disease that typically
About this update from Precigen, Inc.
[{"type":"text","content":"--News Direct--New hope may be on the horizon for sufferers of Recurrent Respiratory Papillomatosis, or RRP, a rare and debilitating disease that typically requires patients to undergo multiple surgeries each year to remove benign tumors called papillomas, that grow in the respiratory tract. Precigen, Inc. (Nasdaq: PGEN) released complete data at a virtual R&D Day event on January 24, 2023 at 4:30 PM ET from a robust, open label Phase 1 trial of its investigational candidate PRGN-2012, an innovative gene-based immunotherapy designed to inhibit or prevent the growth of the dreaded papillomas in the hopes of reducing the number of surgeries. Clinical data presented showed a strong response in RRP patients with 50% of patients in complete response, requiring no post-treatment surgeries, following PRGN-2012 treatment at Dose Level 2. All complete responders remained surgery-free post-treatment with a minimum 12-months of follow up. PRGN-2012 treatment at Dose Level 2 significantly reduced the need for surgeries in severe, aggressive RRP patients with the median number of surgeries in 12-month period being reduced from 6.5 pre-treatment to 0.5 post-treatment. The data also showed that PRGN-2012 was very well tolerated by patients, with no dose-limiting toxicities or treatment-related adverse events above Grade 2. A Phase 2 trial of PRGN-2012 in RRP is also underway and, depending on the results, it may qualify as a pivotal trial to facilitate the filing of a Biologics License Applications (BLA). It is approximately 85 percent enrolled to date. RRP affects both children and adults. Symptoms include a hoarse voice, difficulty sleeping and swallowing, chronic coughing, and breathing problems. In extreme cases, the disease can be fatal because the papillomas cause severe airway obstruction to the lungs and the lungs become flooded with fluids and infection. In a minority of patients RRP can malignantly transform into cancer. The company estimates indicate there are up to 16,000 active cases of RRP in the US and as many as 75,000 total worldwide, although there are believed to be thousands more undiagnosed cases. No therapeutic treatments are currently approved by the FDA. Company estimates suggest PRGN-2012 could have peak annual US sales of approximately $1 billion if approved. There is a high need for safe and effective therapeutic opt...