zopapogene imadenovec (PRGN-2012) / Precigen - LARVOL DELTA

Disease Control and Voice Outcomes Following Treatment With PRGN-2012 in Adults With Recurrent Respiratory Papillomatosis. (PubMed, J Voice) - "These data link greater papilloma disease control with reduced need for clinically indicated procedures and reduced voice handicap after PRGN-2012 treatment independent of short- or long-term measures of disease severity."

Journal • Oncology

Precigen Reports Full Year 2024 Financial Results and Business Updates (PRNewswire) - "Key Program Highlights: PRGN-2012:...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."

Commercial • Launch US • Infectious Disease

FDA Grants Priority Review to Precigen's BLA for PRGN-2012 for the Treatment of Adults with Recurrent Respiratory Papillomatosis with PDUFA Target Action Date Set for August 27, 2025 (PRNewswire) - "Precigen...announced the US Food and Drug Administration (FDA) has accepted the company's biologics license application (BLA) for PRGN-2012 (nonproprietary name: zopapogene imadenovec), an investigational AdenoVerse gene therapy for the treatment of adults with recurrent respiratory papillomatosis (RRP). The FDA granted priority review to the BLA and set a Prescription Drug User Fee Act (PDUFA) target action date of August 27, 2025. The FDA has indicated that they are not currently planning to hold an advisory committee meeting to discuss this application....The BLA is supported by data from the pivotal Phase 1/2 clinical study (NCT04724980)..."

FDA filing • PDUFA • Priority review • Infectious Disease

PRGN-2012 gene therapy in adults with recurrent respiratory papillomatosis: a pivotal phase 1/2 clinical trial. (PubMed, Lancet Respir Med) - P1/2 | "PRGN-2012 treatment resulted in complete response in 51% of the patients treated and was safe. Based on these positive pivotal study results, a biologics license application to the US Food and Drug Administration (FDA) is planned, positioning PRGN-2012 to be an FDA-approved medical treatment for adult patients with RRP."

Journal • P1/2 data • Fatigue • Gene Therapies • Infectious Disease • Oncology

Precigen Completes Submission of BLA with Request for Priority Review to the FDA for PRGN-2012 for the Treatment of Adults with Recurrent Respiratory Papillomatosis (PRNewswire) - "Precigen, Inc...announced the completion of the rolling submission for a biologics license application (BLA) to the US Food and Drug Administration (FDA) for PRGN-2012 (INN: zopapogene imadenovec) for the treatment of adult patients with recurrent respiratory papillomatosis (RRP)....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 is supported by data from the pivotal Phase 1/2 clinical study of PRGN-2012 for the treatment of RRP (NCT04724980)..."

FDA filing • Infectious Disease

Update on HPV-associated head and neck cancers-highlights of the 2024 ASCO Annual Meeting (PubMed, HNO) - "A large number of studies on liquid biopsies (LB) were presented. It was shown that although the methods for analyzing LBs for HPV-positive patients are well developed and can be used for diagnostics, risk classification, treatment management, or tumor follow-up, the methods vary considerably, and their clinical application has not yet been sufficiently validated. With regard to therapeutic HPV vaccination, three large studies were presented for the treatment of recurrent/metastatic HPV-positive oropharyngeal squamous cell carcinoma (OPSCC). The only randomized study was on the vaccine ISA101b (peltopepimut-S) and did not reach its primary endpoint; however, the vaccine seemed to be highly effective in patients with a combined positive score (CPS) ≥ 20. Furthermore, data from a phase I study on PRGN2012, an adenovirus-based immunotherapy used therapeutically for the treatment of recurrent respiratory papillomatosis (RRP), were presented. PRGN2012 led..."

Journal • Review • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Precigen Strategically Prioritizes Portfolio to Focus on First Potential Gene Therapy Launch (PRNewswire) - "Precigen, Inc...announced a strategic reprioritization of the Company's clinical portfolio and streamlining of resources, including a reduction of over 20% of its workforce, to focus on potential commercialization of the PRGN-2012 AdenoVerse gene therapy for the treatment of recurrent respiratory papillomatosis (RRP). These strategic changes substantially reduce required resources for non-priority programs and will enable the Company to focus on pre-commercialization efforts on PRGN-2012, including supporting submission of a rolling biologics license application (BLA) under an accelerated approval pathway anticipated in the second half of 2024, conducting the confirmatory clinical trial, and manufacturing of commercial product. Additionally, the Company will continue acceleration of commercial readiness efforts for a potential launch in 2025..."

FDA filing • Launch • Infectious Disease

Confirmatory Study of PRGN-2012 in Patients With RRP (clinicaltrials.gov) - P3 | N=42 | Recruiting | Sponsor: Precigen, Inc

New P3 trial • Infectious Disease • Oncology

PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients. (ASCO 2024) - P1/2 | "PRGN-2012 is a gorilla adenovirus-based gene therapy/immunotherapy designed to generate HPV6/11-specific T cell immunity. These data demonstrate the overall favorable safety profile and significant clinical benefit of PRGN-2012 in adult RRP patients. These findings support PRGN-2012 as a potential therapeutic option for this patient population where no FDA-approved therapeutics exist."

Clinical • Late-breaking abstract • Fatigue • Gene Therapies • Infectious Disease • Musculoskeletal Pain • Oncology • Pain

Precigen to Announce First Quarter 2024 Financial Results on May 14th and Host a Conference Call on June 3rd following the Late-breaking Oral Presentation for PRGN-2012 at the 2024 ASCO Annual Meeting (PRNewswire) - "The Company will host a conference call on Monday, June 3, 2024 to discuss in detail the pivotal Phase 2 study data of PRGN-2012 for the treatment of recurrent respiratory papillomatosis (RRP) presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Additional details for the June 3rd conference call will be announced at a later date."

P2 data • Infectious Disease

Significant clinical benefit and enhanced T-cell responses with repeated administration of PRGN-2012, a novel gorilla adenoviral vector based immunotherapy, in adult patients with severe Recurrent Respiratory Papillomatosis (ESGCT 2023) - P1/2 | "Based on these findings, PRGN-2012 has been granted FDA's Breakthrough Therapy Designation for the treatment of RRP. The Phase 2 portion of the study has completed enrollment and treatment at the RP2D (n = 23), and interim data will be presented."

Clinical • IO biomarker • Viral vector • Fatigue • Infectious Disease • Oncology

Precigen to Present Late-breaking Abstract for Pivotal Phase 2 Study Data for PRGN-2012 AdenoVerse Immunotherapy for the Treatment of Patients with Recurrent Respiratory Papillomatosis at the 2024 ASCO Annual Meeting (PRNewswire) - "Precigen, Inc...announced clinical data from the pivotal Phase 2 study of PRGN-2012 AdenoVerse immunotherapy for the treatment of patients with recurrent respiratory papillomatosis (RRP) will be presented in a late-breaking oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting taking place from May 31 to June 4, 2024 at McCormick Place in Chicago, Illinois."

P2 data • Infectious Disease

The tumor microenvironment state associates with response to HPV therapeutic vaccination in patients with respiratory papillomatosis. (PubMed, Sci Transl Med) - P1/2 | "PRGN-2012 is a gorilla adenovirus immune-therapeutic capable of enhancing HPV 6/11-specific T cell immunity...Conversely, nonresponders were characterized by greater HPV and CXCL8 gene expression, increased neutrophilic cell infiltration, and reduced T cell papilloma infiltration. These results suggest that papilloma HPV gene expression may regulate interferon signaling and chemokine expression profiles within the tumor microenvironment that cooperate to govern clinical response to therapeutic HPV vaccination in patients with respiratory papillomatosis."

Biomarker • Journal • Tumor microenvironment • Infectious Disease • Oncology • CXCL10 • CXCL8 • CXCL9

Adjuvant PRGN-2012 in Adult Patients With Recurrent Respiratory Papillomatosis (clinicaltrials.gov) - P1/2 | N=38 | Active, not recruiting | Sponsor: Precigen, Inc | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2024 ➔ Jun 2026

Enrollment closed • Trial completion date • Infectious Disease • Oncology

Adjuvant PRGN-2012 in Adult Patients With Recurrent Respiratory Papillomatosis (clinicaltrials.gov) - P1/2 | N=48 | Recruiting | Sponsor: Precigen, Inc | Trial completion date: Oct 2023 ➔ Mar 2024 | Trial primary completion date: Oct 2022 ➔ Mar 2024

Trial completion date • Trial primary completion date • Infectious Disease • Oncology