On Jan. 13, 2025 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported highlights for the upcoming company presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2025 at 9:00 AM PT/12:00 PM ET (Press release, Precigen, JAN 13, 2025, View Source [SID1234649719]). Participants may view details for Precigen’s company presentation at Precigen’s website in the Events & Presentations section at investors.precigen.com/events-presentations.

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"Our recent BLA submission for PRGN-2012 in RRP puts our commercial readiness activities in high gear and we are well underway to ensure full readiness in anticipation of a potential launch in the second half of this year. We have made tremendous progress in the past six months on payer and prescriber analysis, clarifying the market opportunity, building out our commercial and distribution infrastructure, and other activities to ensure we fully coordinate the payor, provider, and patient journeys for launch," said Phil Tennant, Chief Commercial Officer of Precigen. "Our updated analysis of the market using claims data and electronic health records underscores the significant potential for PRGN-2012 in RRP with approximately 27,000 adult patients now identified in the US and we expect more than 125,000 patients ex-US. We are confident that we will be ready to commercialize in the US as soon as we receive approval so that patients can gain access to this game-changing treatment."

"With 100% manufacturing success rate demonstrated to date, we anticipate our in-house commercial drug substance cGMP manufacturing facility will be ready to meet the projected demand to support potential commercial launch in the second half of this year," said Rutul Shah, Chief Operating Officer of Precigen.

"We continue to de-risk our PRGN-2012 asset and now that we have submitted the BLA in RRP, we are a step closer to completing our transition to a commercial-stage company. As we start 2025, we are laser focused on working with the FDA and advancing commercial readiness efforts in anticipation of a potential launch in the second half of this year. In addition, we are preparing for submissions to other health authorities in our prioritized global markets. Beyond RRP, we see immense potential for the AdenoVerse platform in other HPV6/11-driven indications, such as genital warts, and HPV16/18-driven indications, such as cervical cancer and head and neck cancers," said Helen Sabzevari, PhD, President and CEO of Precigen. "As communicated previously, we plan to pursue strategic partnerships to advance our UltraCAR-T programs, which deliver autologous, antigen-specific CAR-T cells overnight to a patient at the patient’s medical center. In conjunction, we are preparing for an end of Phase 1b meeting with the FDA for PRGN-3006 to share results for this highly promising program, including new clinical biomarker data that may further enable patient stratification and positively impact efficacy."

Realizing Precigen’s Commercial Vision for PRGN-2012 AdenoVerse Gene Therapy in RRP

PRGN-2012 (INN: zopapogene imadenovec†) 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), a rare and devastating chronic disease for which the current standard-of-care is repeated surgeries. If approved, PRGN-2012 has the potential to be the first US Food and Drug Administration (FDA)-approved therapeutic for the treatment of RRP.
PRGN-2012 received Breakthrough Therapy Designation from the FDA. PRGN-2012 also received Orphan Drug Designation from the FDA and Orphan Drug Designation from the European Commission.
In December 2024, the Company announced that it had completed the submission of a biologics license application (BLA) with a request for priority review for PRGN-2012 for the treatment of adults with RRP to the FDA. The submission is in the initial 60 day review period, during which time the FDA will decide whether to accept the BLA for further review and set the Prescription Drug User Fee Act (PDUFA) action date. The BLA included a request for priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA.
The BLA, under an accelerated approval pathway, is supported by data from the Phase 1/2 pivotal study in which more than 50% of patients achieved Complete Response and more than 85% of patients had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment. PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.
The confirmatory clinical trial of PRGN-2012 was initiated and is enrolling patients 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 2025 launch.
Maximizing the Potential of PRGN-2009 AdenoVerse Gene Therapy in HPV-associated Cancers

PRGN-2009 Phase 2 clinical trials under a cost-effective 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.
Enrollment was paused in the cervical cancer Phase 2 clinical trial at non-NCI sites as part of strategic reprioritization activities in 2024.
Maximizing the Value of the UltraCAR-T Platform through Strategic Partnerships

Enrollment was completed for the Phase 1b trial for PRGN-3006, which received Fast Track designation from the FDA for the treatment of relapsed or refractory (r/r) acute myeloid leukemia (AML).
Based on the results of correlative studies of the patient samples from the Phase 1/1b study, the Company has identified clinical biomarkers that correlate to objective responses after PRGN-3006 treatment in r/r AML patients. This advancement may further enable patient stratification and positively impact efficacy.
The Company is preparing for an end of Phase 1b meeting with the FDA to discuss results and next steps.
The Company plans to focus on strategic partnership opportunities to advance this promising UltraCAR-T program in AML.
*Cash on-hand is preliminary and unaudited and reflects preliminary financial information as of December 31, 2024. In preparing this information, the Company’s actual financial position as of December 31, 2024 has not yet been finalized by management or reviewed or audited by the Company’s independent registered public accounting firm. This information is also not a comprehensive statement of financial position or results of operations as of or for the year-ended December 31, 2024. Subsequent information or events may lead to material differences between the foregoing preliminary financial information and those reported in the Company’s subsequent SEC filings. Accordingly, investors should not place undue reliance on this preliminary financial information.

†zopapogene imadenovec is the international nonproprietary name (INN) for the investigational therapeutic known as PRGN-2012. Zopapogene imadenovec has not been approved by any health authority in any country for any indication.