Precigen Announces Progress on PRGN-2012 for Recurrent Respiratory Papillomatosis
核心洞察
Precigen completed a pre-BLA meeting with the FDA, achieving full alignment on the BLA content for PRGN-2012, targeting a rolling submission in Q4 2024.
Commercial and manufacturing readiness campaigns are underway for PRGN-2012, anticipating a potential launch in 2025 for recurrent respiratory papillomatosis (搜索).
A confirmatory clinical trial for PRGN-2012 has been initiated, following FDA guidance, with ongoing patient enrollment to support the BLA submission.
Precigen, Inc. (Nasdaq: PGEN) has announced key advancements in its biopharmaceutical programs, particularly PRGN-2012 for recurrent respiratory papillomatosis (搜索) (RRP), alongside progress in its UltraCAR-T platform, as reported in the company's third quarter 2024 financial results and business update.
PRGN-2012: BLA Submission Imminent
Precigen is on track to submit a Biologics License Application (BLA) to the FDA for PRGN-2012 in Q4 2024 under the accelerated approval pathway. This follows a successful pre-BLA meeting with the FDA, where alignment was achieved on all content modules, including chemistry, manufacturing, and controls (CMC).
"We are excited about our imminent submission of a BLA for PRGN-2012 in RRP as we have finalized our pre-BLA meetings and are aligned with the FDA on the content for all modules and plan for submission in the fourth quarter. Our commercial and manufacturing readiness campaigns for PRGN-2012 are well underway to support a potential 2025 launch," said Helen Sabzevari, PhD, President and CEO of Precigen.
Commercial Readiness and Confirmatory Trial
In anticipation of a potential 2025 launch, Precigen has initiated commercial and manufacturing readiness campaigns for PRGN-2012. A confirmatory clinical trial is also underway, as per FDA guidance, to further support the BLA submission.
PRGN-2012 Clinical Data
Data from the pivotal clinical study of PRGN-2012, presented at the 2024 ASCO meeting, demonstrated significant efficacy and safety. Key findings include:
- 51% of patients (18 out of 35) achieved Complete Response, eliminating the need for surgeries post-treatment.
- 86% of patients (30 out of 35) experienced a decrease in surgical interventions in the year following PRGN-2012 treatment.
- The treatment was well-tolerated, with no dose-limiting toxicities or treatment-related adverse events greater than Grade 2.
- Significant improvement (p < 0.0001) was observed in Derkay scores and quality of life among complete responders.
UltraCAR-T Platform: PRGN-3008
Precigen presented preclinical data at SITC 2024 for PRGN-3008, a next-generation UltraCAR-T targeting CD19 (搜索). This therapy incorporates membrane-bound IL-15 (mbIL15) for enhanced persistence, an intrinsic PD-1 blockade to prevent exhaustion, and a safety/kill switch.
Preclinical data showed that a single administration of PRGN-3008 enhanced expansion and persistence, produced robust antitumor efficacy with complete tumor clearance, and demonstrated significantly longer survival compared to conventional CD19 (搜索) CAR-T cells. In a humanized mouse model of lupus nephritis (搜索), PRGN-3008 showed complete elimination of B-cells as well as a decrease in antibodies to double-stranded DNA (dsDNA), a specific marker of lupus.
Financial Highlights
Precigen closed a public offering of its common stock in August 2024, resulting in net proceeds of approximately $30.9 million. The company has strategically prioritized its clinical portfolio and streamlined resources to focus on the potential commercialization of PRGN-2012.