The FDA has agreed that the ongoing Phase 1/2 single-arm study of Precigen’s (NASDAQ:PGEN) investigational PRGN-2012 AdenoVerse immunotherapy for the treatment of recurrent respiratory papillomatosis will serve as pivotal for the purpose of filing an accelerated approval request for licensure.
The FDA also confirmed no additional randomized, placebo-controlled trial will be required to support submission of a biologics license application (BLA). Based on the FDA guidance, the company plans to initiate a confirmatory study prior to submission of the BLA.
In addition, the FDA confirmed that the current primary endpoint for the ongoing Phase 1/2 study, which is complete response rate (percentage of patients with no surgical interventions during the 12 months following treatment with PRGN-2012), along with an immunological surrogate marker, demonstrating an induction of HPV-specific T-cell responses following PRGN-2012 treatment, is acceptable for the accelerated approval request.
Precigen has completed enrollment and dosing in the Phase 2 portion of the study, bringing the total number of enrolled patients to 35 at the recommended Phase 2 dose. Patient follow up is currently ongoing and data collection is anticipated to be completed by the second quarter of 2024.
“The eligibility of the Phase 1/2 study, which has already been fully enrolled and dosed, as the pivotal study to support accelerated approval has the potential to significantly reduce the product development time for PRGN-2012,” Helen Sabzevari, Ph.D., president and CEO of Precigen, said in a statement.
“We are thankful for the FDA’s decision, which underscores the importance of bringing innovative approaches for the treatment of this serious and rare disease,” she added.
