–   Pivotal Phase 2 study data of PRGN-2012 for the treatment of patients with recurrent respiratory papillomatosis to be presented at the 2024 ASCO Annual Meeting as a late-breaking oral presentation on June 3rd – –   Company to host a conference call on June 3rd following the PRGN-2012 ASCO presentation to discuss in detail the pivotal study results and provide business updates – –   PRGN-2012 rolling BLA submission, under an accelerated approval pathway, is anticipated in the second half of 2024; commercial readiness activities underway for a potential launch in 2025 – –   Two trial-in-progress presentations for PRGN-2009 in combination with pembrolizumab for the treatment of recurrent/metastatic cervical cancer and oropharyngeal cancer to be presented at ASCO – –   Company and the Recurrent Respiratory Papillomatosis Foundation to co-sponsor the inaugural RRP Awareness Day on June 11th to raise awareness and bring together individuals living with RRP, caregivers, clinicians, and government officials – –   Cash, cash equivalents, and short-term investments totaled $44.8 million as of March 31, 2024 – GERMANTOWN, Md., May 14, 2024 /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 first quarter 2024 financial results and business updates. "We are excited to share the pivotal Phase 2 data for our PRGN-2012 study in patients with RRP at ASCO and look forward to providing additional details regarding the results at our planned conference call following the presentation. We remain on track for a PRGN-2012 rolling BLA submission in the second half of 2024 and we are actively moving ahead with our commercial readiness efforts in anticipation of a potential launch of PRGN-2012 in 2025," said Helen Sabzevari, PhD, President and CEO of Precigen. "Based on the competitive advantages of PRGN-2012, including a favorable route of administration, safety profile and the efficacy demonstrated in the clinical trial results so far, we believe PRGN-2012 has the potential to be the first-in-class and best-in-class treatment for RRP patients. We anticipate PRGN-2012 to overwhelmingly be the treatment of choice for RRP patients, if approved, as indicated by our commissioned research of healthcare providers and key opinion leaders which found PRGN-2012's competitive advantages highly compelling." "With multiple milestones anticipated in 2024 and 2025, we remain steadfastly committed to a strategy of sound financial management," said Harry Thomasian Jr., CFO of Precigen. "We are evaluating various financing opportunities to strengthen our balance sheet as we prepare our lead asset, PRGN-2012, for potential commercial launch in 2025." Key Program Highlights AdenoVerse® PRGN-2012 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 was the first to receive Breakthrough Therapy Designation and an accelerated approval pathway for RRP from the FDA. PRGN-2012 received Orphan Drug Designation from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission.   PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP (clinical trial identifier: NCT04724980). Results from the Phase 1 portion of the Phase 1/2 study were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS). PRGN-2012 demonstrated overall safety and clinically meaningful benefit with 50% of patients (N=12) in Complete Response, which is defined as no surgeries needed during the 12-month period following PRGN-2012 treatment completion. All Complete Responses were durable and ongoing more than two years after PRGN-2012 treatment. 83% of patients had a reduction in RRP surgeries in the 12-month period after PRGN-2012 treatment compared to 12 months pre-treatment.  Correlative data support expansion of peripheral HPV 6 and HPV 11–specific T cell immunological responses as the underlying mechanism of action for PRGN-2012. PRGN-2012 is built using the Company's differentiated gorilla adenovector that allows for repeat dosing. The redosing potential of AdenoVerse has been highlighted in clinical studies where repeat administrations of PRGN-2009 and PRGN-2012 gene therapies led to enhancement of antigen-specific T cell immune responses without generation of significant neutralizing antibodies in contrast to other viral vectors. Results from the pivotal Phase 2 study of PRGN-2012 for the treatment of RRP, including immunological responses, will be 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." Scott M. Norberg, DO, Associate Research Physician, Center for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, will deliver the presentation on June 3, 2024 at 8:30 AM CT. The Company plans to host a conference call on June 3, 2024 to discuss in detail the PRGN-2012 pivotal study results presented and provide business updates. FDA confirmed that the ongoing Phase 1/2 single arm study will serve as pivotal and no additional randomized, placebo-controlled trial will be required to support submission of a Biologics License Application (BLA). A rolling BLA submission under an accelerated approval pathway is anticipated in the second half of 2024. Based on FDA guidance, the Company is on track to initiate a confirmatory study prior to submission of the BLA. Commercial readiness preparations are underway for a potential launch in 2025. The Company and the Recurrent Respiratory Papillomatosis Foundation will co-sponsor the inaugural RRP Awareness Day on June 11, 2024. The multi-stakeholder event will raise awareness and bring together individuals living with RRP, caregivers, clinicians, and government officials to encourage new connections and build community among those interested in and affected by RRP. The inaugural event will be hybrid with in-person participation at the National Press Club in Washington DC and a webcast for virtual participation. PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is an investigational off-the-shelf AdenoVerse gene therapy designed to activate the immune system to recognize and target HPV-associated cancers. The Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) is enrolling patients (clinical trial identifier: NCT05996523). An abstract titled, "Phase II trial of immunotherapeutic HPV vaccine PRGN-2009 with pembrolizumab before standard treatment in subjects with newly diagnosed HPV-associated oropharyngeal cancer" will be presented as a trial-in-progress poster presentation on June 2, 2024 from 9:00 AM to 12:00 PM CT at ASCO. The Phase 2 randomized, open-label study of PRGN-2009 in combination with pembrolizumab in patients with HPV-associated recurrent/metastatic cervical cancer is active and recruiting patients (clinical trial identifier: NCT06157151). An abstract titled, "A Phase 2 study to evaluate efficacy and safety of PRGN-2009, a novel gorilla adenovirus-based immunotherapy, in combination with pembrolizumab versus pembrolizumab monotherapy in patients with recurrent or metastatic cervical cancer" will be presented as a trial-in-progress poster presentation on June 3, 2024 from 9:00 AM to 12:00 PM CT at ASCO. UltraCAR-T® PRGN-3006 in AML/MDS: PRGN-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.  PRGN-3006 is currently under investigation in a Phase 1b dose expansion clinical trial (clinical trial identifier: NCT03927261) for the treatment of patients with r/r AML or higher-risk myelodysplastic syndromes (MDS). An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024.  PRGN-3005 in Ovarian Cancer: PRGN-3005 is an investigational multigenic, autologous CAR-T cell therapy engineered ...