Precigen Receives FDA Approval for Immunotherapy Treatment

Precigen, a biopharmaceutical company, has received FDA approval for its immunotherapy for a rare respiratory disease. The treatment, developed using Precigen's proprietary technology platforms, targets intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen operates through two segments: Biopharmaceuticals and Exemplar, which focuses on developing research models and services.

Precigen, Inc., a biopharmaceutical company specializing in gene and cell therapies, has received a significant milestone with the FDA approval of its gene therapy candidate, Papzimeos (zopapogene imadenovec-drba), for the treatment of recurrent respiratory papillomatosis (RRP) [1]. This approval marks a pivotal advancement in the company's portfolio, which focuses on developing therapies for intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases.

RRP is a rare, chronic disease caused by persistent human papillomavirus (HPV) 6 or 11 infection, leading to the growth of benign tumors in the respiratory tract, most commonly the larynx. The disease is associated with significant morbidity, including voice changes, breathing difficulties, and airway obstruction. Until now, there were no approved medical therapies that eliminated the need for repeated surgical procedures. The FDA's approval of Papzimeos represents a significant breakthrough for patients suffering from RRP [1].

Papzimeos is administered via subcutaneous injection and is designed to stimulate an immune response against cells infected with HPV types 6 and 11—the causative agents in RRP. The therapy offers a novel mechanism of action distinct from traditional treatments, which have relied primarily on repeated surgical interventions. The approval is based on results from a single-arm, open-label trial evaluating Papzimeos in adult patients with RRP who required three or more surgeries per year. In this pivotal study, 51.4% of patients (18/35) achieved a complete response—defined as no need for surgical intervention in the 12 months following treatment. Follow-up data showed that durable responses were maintained in most patients through two years, with a strong correlation between clinical benefit and the induction of HPV 6/11-specific T cells [1].

The safety profile of Papzimeos was favorable, with most treatment-emergent adverse events being mild to moderate. No dose-limiting toxicities were observed, and no treatment-related serious adverse events were reported. The FDA granted approval of Papzimeos to Precigen under Priority Review and the product received both Orphan Drug designation and Breakthrough Therapy designation [1].

The approval of Papzimeos has significant implications for Precigen and its shareholders. The company's stock surged by approximately 34% in the premarket following the FDA's announcement [2]. Precigen operates through two segments: Biopharmaceuticals and Exemplar. The Biopharmaceuticals segment is primarily comprised of the company's legal entities of Precigen and ActoBio, as well as royalty interests in therapeutics and therapeutic platforms from companies not controlled by the Company. The Exemplar segment is composed of Exemplar Genetics LLC, doing business as Precigen Exemplar (Exemplar), its wholly owned subsidiary focused on developing research models and services for healthcare research applications [3].

References:

[1] https://www.fda.gov/news-events/press-announcements/fda-approves-first-immunotherapy-recurrent-respiratory-papillomatosis
[2] https://seekingalpha.com/news/4486035-precigen-stock-up-fda-nod-gene-therapy
[3] https://www.marketscreener.com/news/precigen-shares-surge-pre-bell-after-recurrent-respiratory-papillomatosis-drug-receives-fda-approval-ce7c51dedd8ff427