GERMANTOWN, Md. - Precigen, Inc. (NASDAQ:PGEN), a biopharmaceutical company, announced today positive results from a Phase 1/2 pivotal study of its gene therapy product, PRGN-2012. The study, which evaluated the safety and efficacy of the treatment in patients with recurrent respiratory papillomatosis (RRP), met its primary endpoints.
The investigational therapy led to 51% of participants experiencing a complete response, meaning they required no surgeries for RRP in the 12 months following treatment. Additionally, 86% of patients saw a decrease in surgical interventions compared to the year prior to receiving PRGN-2012. The median number of surgeries dropped from four pre-treatment to zero post-treatment.
PRGN-2012 was also reported to be well-tolerated, with no dose-limiting toxicities or treatment-related adverse events greater than Grade 2. Furthermore, the treatment was associated with significant improvements in Derkay scores, which measure RRP severity, and in quality of life scores among complete responders.
RRP is a rare disease characterized by the growth of benign tumors in the respiratory tract, caused by human papillomavirus (HPV) types 6 or 11. The current standard-of-care involves repeated surgeries, with no FDA-approved therapeutic available. PRGN-2012, if approved, has the potential to be the first such therapeutic option.
The study's design included an initial dose escalation cohort followed by a larger group receiving the recommended Phase 2 dose via subcutaneous injection. The treatment induced HPV 6/11-specific T cell responses, with a greater expansion of peripheral HPV-specific T cells in responders compared to non-responders.
The company plans to discuss these results with the FDA as part of a rolling Biologics License Application submission under an accelerated approval pathway, anticipated in the second half of 2024. Precigen is also preparing for a potential commercial launch in 2025, pending approval.
This announcement is based on a press release statement from Precigen, Inc. The detailed results were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting and will be further discussed in a webcast event by the company.
InvestingPro Insights
As Precigen, Inc. (NASDAQ:PGEN) shares promising results from its pivotal study, the financial perspective of the company adds another layer of consideration for investors. According to recent InvestingPro data, Precigen's market capitalization stands at 366.77 million USD, reflecting the market's current valuation of the company.
Despite the positive clinical outcomes, the company's financial metrics suggest some challenges. The firm's gross profit margin over the last twelve months as of Q1 2024 was notably negative at -935.91%, indicating that costs significantly outweighed revenue during this period. Moreover, the company has experienced a substantial revenue decline of 76.6% over the last twelve months as of Q1 2024.
In the context of these financial results, two InvestingPro Tips highlight critical considerations for investors. Firstly, Precigen holds more cash than debt on its balance sheet, which could provide some financial flexibility in the short term. However, the company is also quickly burning through cash, which may raise concerns about its long-term financial sustainability, especially as it prepares for potential commercialization efforts.
Investors interested in a deeper analysis of Precigen's financial health and future prospects can find additional insights on InvestingPro. There are currently 9 more InvestingPro Tips available for PGEN at https://www.investing.com/pro/PGEN. These tips could provide valuable guidance on whether the company's current clinical successes could translate into financial gains. For those considering an InvestingPro subscription, use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.