Precigen, Inc., a biopharmaceutical company specializing in innovative gene and cell therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its groundbreaking investigational immunotherapy, PRGN-2012 AdenoVerse. This designation marks a significant milestone for Precigen's AdenoVerse platform and brings hope to patients suffering from recurrent respiratory papillomatosis (RRP), a debilitating condition with limited treatment options.

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Understanding FDA's Breakthrough Therapy Designation

The FDA's Breakthrough Therapy Designation is a coveted status that accelerates the development and review process of medications designed to treat serious or life-threatening diseases. To receive this designation, a therapy must demonstrate substantial improvement over existing treatments, as supported by preliminary clinical evidence.

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PRGN-2012's Potential to Revolutionize RRP Treatment

Helen Sabzevari, PhD, President and CEO of Precigen, expressed enthusiasm regarding the Breakthrough Therapy Designation, emphasizing its potential to transform the lives of RRP patients. Currently, the standard-of-care for RRP involves repeated surgical interventions, with no approved therapeutics available. PRGN-2012 offers the promise of reducing surgical interventions and improving outcomes for these patients, making Precigen proud to receive this recognition from the FDA.

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The Unique Features of PRGN-2012

PRGN-2012 is an innovative immunotherapy that harnesses the power of Precigen's proprietary AdenoVerse platform. It incorporates optimized antigen design using gorilla adenovector technology, which provides several advantages. These include the ability for repeat administration, the inability to replicate in vivo, and the capacity to deliver a large genetic payload. Previously, PRGN-2012 was granted Orphan Drug Designation by the FDA for RRP patients.

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Clinical Evidence Supporting the Breakthrough Therapy Designation

The Breakthrough Therapy Designation was informed by the clinical evidence generated from the Phase 1 study (NCT04724980), which showcased the remarkable potential of PRGN-2012. The study enrolled patients who had undergone an average of 5.8 RRP surgeries in the year prior to treatment. The results were highly promising, with 50% of patients (6 out of 12) achieving a Complete Response, requiring no further surgeries even after a minimum follow-up period of 12 months. Additionally, PRGN-2012 led to a reduction in surgeries for 83% (10 out of 12) of patients during the 12 months following treatment. The therapy also induced a robust HPV-specific T-cell immune response, signifying its efficacy in addressing RRP. Importantly, PRGN-2012 demonstrated excellent safety, with no dose-limiting toxicities or treatment-related adverse events greater than Grade 2.

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Ongoing Phase 2 Study and Future Prospects

PRGN-2012 is currently undergoing evaluation in a Phase 2 study involving adult patients with RRP. Precigen has successfully enrolled 23 patients, reaching a total of 35 enrolled patients at the recommended phase 2 dose (RP2D). Patient follow-up is underway, and Precigen is actively engaging with the FDA to explore rapid development paths that would facilitate the submission of a Biologics License Application (BLA) in the future.