Precigen Reports Full Year 2024 Financial Results and Business Updates
FDA granted priority review to Company's BLA for PRGN-2012 for the treatment of adults with recurrent respiratory papillomatosis and set PDUFA target action date for August 27, 2025
Results from pivotal clinical study of PRGN-2012 were published in The Lancet Respiratory Medicine
PRGN-2012 treatment demonstrated durable complete responses with median duration of response of two years, with some complete responders surgery-free beyond three years as of August 28, 2024 data cutoff
Company continues to rapidly advance commercial and manufacturing readiness campaign in anticipation of 2025 commercial launch
Company ended 2024 with $97.9 million in cash, cash equivalents, and investments, extending its cash runway into 2026, beyond potential 2025 commercial launch of PRGN-2012
GERMANTOWN, Md., March 19, 2025 /PRNewswire/ -- Precigen, Inc. (NASDAQ:PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced full year 2024 financial results and business updates.
"Last year, we achieved several milestones on our path to potentially bring our innovative PRGN-2012 therapy to RRP patients, including presentation of groundbreaking pivotal data and BLA submission to the FDA. Through prioritizing our portfolio and financing activities, we strengthened our financial position, enabling ongoing commercial and manufacturing readiness efforts for PRGN-2012 in anticipation of a potential 2025 launch. Our commercial organization has been working to scale up quickly, right-sizing the organization to rapidly capitalize on the immense demand from patients and treating physicians for a new treatment paradigm that may finally address the underlying cause of the disease," said Helen Sabzevari, PhD, President and CEO of Precigen. "Already this year, our BLA for PRGN-2012 received priority review from the FDA with an August 2025 PDUFA action date, bringing us a step closer to launching the first and only FDA-approved treatment to the approximately 27,000 adult RRP patients in the US. FDA approval for PRGN-2012 would fundamentally change Precigen, enabling the transition from clinical to commercial stage with the real and imminent potential to begin realizing product sales this year and providing financial tailwinds to enable potential expansion of PRGN-2012 to new indications and geographical markets."
"As a result of the preferred stock offering and sale of intellectual property and related royalty rights for a non-core asset, we extended our cash runway into 2026, beyond the potential commercial launch of PRGN-2012 this year. We are preparing for the transition to a commercial stage company and the potential to add product-related revenue," said Harry Thomasian Jr., CFO of Precigen.
Key Program Highlights
PRGN-2012 (nonproprietary name: zopapogene imadenovec†) AdenoVerse® Gene Therapy in RRP PRGN-2012 is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 received Breakthrough Therapy Designation, Orphan Drug Designation, and an accelerated approval pathway from the FDA, and Orphan Drug Designation from the European Commission.
In February 2025, the US Food and Drug Administration (FDA) accepted the company's biologics license application (BLA) for PRGN-2012, and granted priority review to the BLA with a Prescription Drug User Fee Act (PDUFA) target action date set for August 27, 2025. The FDA has indicated that they are not currently planning to hold an advisory committee meeting to discuss the BLA.
Results from the pivotal clinical study of PRGN-2012 for the treatment of RRP were presented at the 2024 American Society of Clinical Oncology (ASCO) annual meeting in a late-breaking oral presentation titled, "PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients" and published in The Lancet Respiratory Medicine.
Pivotal study successfully met its primary safety and pre-specified primary efficacy endpoints.
PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.
51% (18 out of 35) of patients achieved Complete Response, requiring no surgeries after treatment with PRGN-2012. Complete Responses have been durable beyond 12 months with median duration of follow up of 24 months, with some complete responders surgery-free beyond three years as of the August 28, 2024 data cutoff.
86% of patients (30 out of 35) had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment; RRP surgeries reduced from a median of 4 (range: 3-10) pre-treatment to 0 (range: 0-7) post-treatment.
PRGN-2012 treatment induced HPV 6/11-specific T cell responses in RRP patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with non-responders.
PRGN-2012 significantly (p < 0.0001) improved anatomical Derkay scores and VHI-10 scores in complete responders.
Patient enrollment continues to advance in the confirmatory clinical trial of PRGN-2012 in accordance with the guidance from the FDA to initiate the study prior to submission of the BLA.
The Company continues to rapidly advance its commercial and manufacturing readiness campaign in anticipation of a potential launch in 2025.
Based on recently updated internal analysis derived from review of claims data, the market opportunity for PRGN-2012 in RRP is estimated to be approximately 27,000 adult patients in the US and more than 125,000 patients outside of the US.
PRGN-2009 AdenoVerse® Gene Therapy in HPV-associated cancersPRGN-2009 is an investigational off-the-shelf AdenoVerse gene therapy designed to activate the immune system to recognize and target HPV-associated cancers.
PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer are ongoing.
PRGN-3006 UltraCAR-T® in AML and MDSPRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA.
The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the FDA, and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps.
Financial Highlights
In December 2024, the Company raised $87.5 million, of which $79.0 million was from a private placement offering of convertible preferred stock, and an additional $8.5 million was from the sale of certain intellectual property and royalty rights related to a non-core asset.
By year's end 2024, cash, cash equivalents, and investments totaled $97.9 million.
Full Year 2024 Financial Results Compared to Prior Year PeriodTotal revenues decreased $2.3 million, or 37%, and cost of products and services decreased $1.8 million or 30% compared to the year ended
