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
Precigen (PGEN) has completed its Biologics License Application (BLA) submission to the FDA for PRGN-2012, a potential first FDA-approved treatment for adults with Recurrent Respiratory Papillomatosis (RRP). The therapy received Breakthrough Therapy Designation and Orphan Drug Designation from both FDA and European Commission. The BLA submission includes compelling Phase 1/2 pivotal study results where over 50% of patients achieved Complete Response and more than 85% experienced decreased surgical interventions in the year following treatment. The company has requested priority review, which could reduce the FDA review timeline from 10 to 6 months. PRGN-2012 demonstrated strong safety profile with no treatment-related adverse events above Grade 2.
Precigen (PGEN) ha completato la sua richiesta di licenza biologica (BLA) all'FDA per PRGN-2012, un potenziale primo trattamento approvato dall'FDA per adulti con Papillomatosi Respiratoria Ricorrente (RRP). La terapia ha ricevuto la Designazione di Terapia Avanzata e la Designazione di Farmaco Orfano sia dall'FDA che dalla Commissione Europea. La richiesta di BLA include risultati convincenti dello studio pivotale di Fase 1/2, dove oltre il 50% dei pazienti ha ottenuto una risposta completa e più dell'85% ha sperimentato una riduzione degli interventi chirurgici nell'anno successivo al trattamento. L'azienda ha richiesto una revisione prioritaria, che potrebbe ridurre il tempo di revisione dell'FDA da 10 a 6 mesi. PRGN-2012 ha dimostrato un forte profilo di sicurezza, senza eventi avversi correlati al trattamento superiori al Grado 2.
Precigen (PGEN) ha completado su solicitud de licencia biológica (BLA) ante la FDA para PRGN-2012, un posible primer tratamiento aprobado por la FDA para adultos con Papilomatosis Respiratoria Recurrente (RRP). La terapia recibió la Designación de Terapia Innovadora y la Designación de Medicamento Huérfano tanto de la FDA como de la Comisión Europea. La solicitud de BLA incluye resultados convincentes de un estudio pivotal de Fase 1/2, donde más del 50% de los pacientes logró Respuesta Completa y más del 85% experimentó una disminución en las intervenciones quirúrgicas en el año posterior al tratamiento. La empresa ha solicitado una revisión prioritaria, lo que podría reducir el tiempo de revisión de la FDA de 10 a 6 meses. PRGN-2012 demostró un sólido perfil de seguridad sin eventos adversos relacionados con el tratamiento superiores al Grado 2.
프레시겐 (PGEN)은 반복성 호흡기 유두종증(RRP) 성인을 위한 FDA 최초의 승인 치료제인 PRGN-2012에 대한 생물 의약품 허가 신청(BLA)을 FDA에 제출했습니다. 이 치료법은 FDA와 유럽연합 집행위원회로부터 혁신 치료제 지정 및 희귀약 지정 승인을 받았습니다. BLA 제출에는 50% 이상의 환자가 완전 반응을 보였다는 설득력 있는 1/2상 임상 시험 결과가 포함되어 있으며, 85% 이상이 치료 후 1년 동안 외과적 개입이 감소했다고 보고되었습니다. 회사는 우선 심사를 요청했으며, 이를 통해 FDA의 심사 기간을 10개월에서 6개월로 단축할 수 있습니다. PRGN-2012는 2등급 이상의 치료 관련 부작용이 전혀 없는 안전성 프로필을 입증했습니다.
Precigen (PGEN) a soumis sa demande de licence biologique (BLA) à la FDA pour PRGN-2012, un potentiel premier traitement approuvé par la FDA pour les adultes atteints de papillomatose respiratoire récurrente (RRP). La thérapie a reçu la désignation de thérapie innovante et la désignation de médicament orphelin tant de la FDA que de la Commission Européenne. La soumission de la BLA inclut des résultats convaincants d'une étude pivotale de phase 1/2, où plus de 50 % des patients ont obtenu une réponse complète et plus de 85 % ont connu une diminution des interventions chirurgicales dans l'année suivant le traitement. La société a demandé un examen prioritaire, ce qui pourrait réduire le délai d'examen de la FDA de 10 à 6 mois. PRGN-2012 a démontré un profil de sécurité solide sans événements indésirables liés au traitement de grade supérieur à 2.
Precigen (PGEN) hat seinen Antrag auf Biologics License Application (BLA) bei der FDA für PRGN-2012 eingereicht, eine potenzielle erste von der FDA genehmigte Behandlung für Erwachsene mit wiederkehrender respiratorischer Papillomatose (RRP). Die Therapie erhielt sowohl von der FDA als auch von der Europäischen Kommission den Status einer Durchbruchtherapie und die Orphan Drug Designation. Der BLA-Antrag umfasst überzeugende Ergebnisse einer entscheidenden Phase-1/2-Studie, bei der über 50% der Patienten eine vollständige Rückmeldung erzielten und mehr als 85% eine Verringerung der chirurgischen Eingriffe im Jahr nach der Behandlung erlebten. Das Unternehmen hat eine prioritäre Prüfung beantragt, die den FDA-Prüfzeitraum von 10 auf 6 Monate verkürzen könnte. PRGN-2012 zeigte ein starkes Sicherheitsprofil ohne behandlungsbedingte unerwünschte Ereignisse über Grad 2 hinaus.
- Over 50% of patients achieved Complete Response in Phase 1/2 pivotal study
- More than 85% reduction in surgical interventions post-treatment
- Potential to be first FDA-approved therapeutic for RRP
- Received both Breakthrough Therapy and Orphan Drug Designations
- Strong safety profile with no severe adverse events
- Cash runway extended into 2026, beyond potential commercial launch
- FDA review acceptance still pending
- Commercial success dependent on FDA approval
- Priority review status not yet granted
Insights
The BLA submission for PRGN-2012 represents a major milestone with significant market implications. The clinical data is exceptionally strong, with >
For a company with a market cap of
The clinical profile of PRGN-2012 is remarkable from a medical perspective. RRP has been a treatment challenge for decades, with patients enduring hundreds of surgeries throughout their lifetime. The safety data showing no treatment-related adverse events above Grade 2 and the robust efficacy in reducing surgical burden represent a potential paradigm shift in RRP management.
The immunological mechanism targeting HPV 6/11-infected cells addresses the root cause rather than just managing symptoms. This could fundamentally change the natural history of this devastating chronic disease that currently has no cure.
The regulatory positioning is highly advantageous. The triple designation status (Breakthrough Therapy, Orphan Drug in US/EU, accelerated approval pathway) significantly de-risks the approval process. If priority review is granted, approval could come as early as mid-2025, assuming a 6-month review cycle from potential acceptance in Q1 2025.
The comprehensive Phase 1/2 data package, including quality of life metrics and Derkay scoring, strengthens the submission. The unmet medical need in RRP and lack of approved alternatives further support a favorable regulatory outcome.
– PRGN-2012 has the potential to be the first FDA-approved therapeutic for the treatment of adults with RRP, a rare and devastating chronic disease for which the current standard-of-care is repeated surgeries –
– PRGN-2012 received Breakthrough Therapy Designation from the FDA and Orphan Drug Designation from the FDA and the European Commission –
– The BLA, under an accelerated approval pathway, is supported by data from the Phase 1/2 pivotal study in which more than
– PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2 –
PRGN-2012 is an investigational AdenoVerse® gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11. PRGN-2012 received Breakthrough Therapy Designation, Orphan Drug Designation, and an accelerated approval pathway from the US Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission.
PRGN-2012 has the potential to be the first FDA-approved therapeutic for the treatment of adults with RRP. RRP is a rare, difficult-to-treat, lifelong neoplastic disease of the upper and lower respiratory tracts caused by infection with HPV 6 or HPV 11 that can be fatal. Currently, there is no cure for RRP and the current standard-of-care is repeated surgeries, which do not address the underlying cause of disease and are associated with significant morbidity. As a result, the cycle of recurrences and surgeries continues and patients can require hundreds of lifetime surgeries.1-7
The BLA is supported by data from the pivotal Phase 1/2 clinical study of PRGN-2012 for the treatment of RRP (NCT04724980), which evaluated the safety and efficacy of PRGN-2012 in adult RRP patients. Of the 38 total patients enrolled in the study, three patients received four administrations of PRGN-2012 at 1x 1011 particle units (PU)/dose and 35 patients received four administrations of PRGN-2012 at the recommended phase 2 dose (RP2D) of 5 x 1011 PU/dose over a 12 week treatment period via subcutaneous injection. Primary endpoints included safety and Complete Response rate defined as the percentage of patients who require no RRP surgeries in the 12-month period after PRGN-2012 treatment completion. Key secondary endpoints included HPV-specific immune responses, extent of papilloma growth as measured by Derkay scoring, and quality of life as measured by Vocal Handicap Index-10 (VHI-10). As reported in the groundbreaking results from the pivotal study presented at the 2024 American Society of Clinical Oncology (ASCO) annual meeting, the primary safety and efficacy endpoints were met.
"The impact of this debilitating disease on patients, families, and their caregivers has been overlooked for more than half a century. There is currently no approved therapy for RRP patients and the submission of our BLA is an extremely important step in bringing the first therapy to fight this devastating disease," said Helen Sabzevari, PhD, President and CEO of Precigen. "We look forward to working closely with the FDA on next steps now that we have completed the BLA submission and we are excited by the potential to bring PRGN-2012 to RRP patients as quickly as possible. With our most recent financial transactions announced last week to enhance our balance sheet, we have extended our cash runway into 2026, well beyond potential commercial launch in the second half of 2025."
AdenoVerse®
Precigen's AdenoVerse platform utilizes a library of proprietary adenovectors for the efficient gene delivery of therapeutic effectors, immunomodulators, and vaccine antigens designed to modulate the immune system. Precigen's gorilla adenovectors, part of the AdenoVerse library, have potentially superior performance characteristics as compared to current competition. AdenoVerse gene therapies have been shown to generate high-level and durable antigen-specific T-cell immune responses as well as an ability to boost these responses via repeat administration. Superior performance characteristics and high yield manufacturing of AdenoVerse vectors leveraging UltraVector® technology allows Precigen to engineer cutting-edge investigational gene therapies to treat complex diseases.
Precigen: Advancing Medicine with Precision™
Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on LinkedIn or YouTube.
Trademarks
Precigen, AdenoVerse, UltraVector and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.
Cautionary Statement Regarding Forward-Looking Statements
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon the Company's current expectations and projections about future events and generally relate to plans, objectives, and expectations for the development of the Company's business, including the timing and progress of preclinical studies, clinical trials, regulatory approvals, commercial launches and related milestones, the promise of the Company's portfolio of therapies, and in particular its CAR-T and AdenoVerse therapies. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release. The Company has no obligation to provide any updates to these forward-looking statements even if its expectations change. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. For further information on potential risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in the Company's most recent Annual Report on Form 10-K and subsequent reports filed with the Securities and Exchange Commission.
Investor Contact:
Steven M. Harasym
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com
Media Contacts:
Donelle M. Gregory
press@precigen.com
Glenn Silver
Lazar-FINN Partners
glenn.silver@finnpartners.com
†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. |
References | |
1 | Mounts, P et al. (1982). "Viral etiology of juvenile- and adult-onset squamous papilloma of the larynx." Proc Natl Acad Sci U S A 79(17): 5425-5429. |
2 | Smith, E et al. (1993). "Human papillomavirus infection in papillomas and nondiseased respiratory sites of patients with recurrent respiratory papillomatosis using the polymerase chain reaction." Arch Otolaryngol Head Neck Surg 119(5): 554-557. |
3 | Derkay, CS et al. (2008). "Recurrent respiratory papillomatosis: a review." Laryngoscope 118(7): 1236-1247. |
4 | Derkay, CS et al. (2019). "Update on Recurrent Respiratory Papillomatosis." Otolaryngol Clin North Am 52(4): 669-679. |
5 | Seedat, RY (2020). "Juvenile-Onset Recurrent Respiratory Papillomatosis Diagnosis and Management - A Developing Country Review." Pediatric Health Med Ther 11: 39-46. |
6 | Dedo, HH et al. (2001). "CO(2) laser treatment in 244 patients with respiratory papillomas." Laryngoscope 111(9): 1639-1644. |
7 | Silver, RD et al. (2003). "Diagnosis and management of pulmonary metastasis from recurrent respiratory papillomatosis." Otolaryngol Head Neck Surg 129(6): 622-629. |
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