FDA advances Precigen’s gene therapy for respiratory disease

Published 25/02/2025, 14:14
FDA advances Precigen’s gene therapy for respiratory disease

GERMANTOWN, Md. - The US Food and Drug Administration (FDA) has accepted Precigen Inc.’s (NASDAQ:PGEN) biologics license application (BLA) for its investigational gene therapy, PRGN-2012, granting it priority review status with a target action date of August 27, 2025. The announcement comes as Precigen, with a market capitalization of $512.52 million, has seen its stock surge over 50% in the past six months, according to InvestingPro data. PRGN-2012 is designed to treat adults with recurrent respiratory papillomatosis (RRP), a rare and potentially fatal disease with no current FDA-approved therapy.

RRP is caused by human papillomavirus (HPV) types 6 or 11 and is characterized by the growth of benign tumors in the airways leading to severe complications. The disease is currently managed through repeated surgical interventions, which do not address the underlying HPV infection and may lead to significant morbidity.

The priority review designation by the FDA is an acknowledgment of the urgent need for an effective treatment for RRP patients, who may undergo numerous surgeries over their lifetime. PRGN-2012, which has received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA, aims to elicit immune responses against HPV-infected cells, potentially reducing or eliminating the need for surgery.

Clinical data from the Phase 1/2 study of PRGN-2012 showed promising results, with more than half of the patients achieving a complete response and a significant reduction in surgical interventions post-treatment. The study, which met its primary safety and efficacy endpoints, reported no dose-limiting toxicities and no treatment-related adverse events above Grade 2. Analysts appear optimistic about the company’s potential, with price targets ranging from $5 to $7, significantly above current trading levels, as revealed by InvestingPro data.

Precigen’s President and CEO, Helen Sabzevari, expressed optimism about the therapy’s potential to provide a significant clinical benefit and an alternative to surgery for the estimated 27,000 adults with RRP in the US.

PRGN-2012 utilizes Precigen’s proprietary AdenoVerse gene therapy platform, which is designed to deliver therapeutic genes effectively and has been shown to generate durable T-cell immune responses.

The FDA’s acceptance of the BLA for PRGN-2012 marks a critical step towards potentially offering the first FDA-approved treatment option for patients with RRP. With the company’s next earnings report due on February 26, 2025, investors can access comprehensive analysis and 14 additional ProTips through InvestingPro’s detailed research reports, which provide deep-dive analysis of Precigen’s financial health and growth prospects. This article is based on a press release statement from Precigen, Inc.

In other recent news, Precigen, Inc. has announced several key developments. The company successfully regained compliance with Nasdaq’s minimum bid price requirement, maintaining a closing price at or above $1.00 per share for ten consecutive business days. This marks a positive shift for the company after previously falling below the required threshold. Additionally, Precigen has entered into a securities purchase agreement for a private placement of its 8.00% Series A Convertible Perpetual Preferred Stock, expecting to raise $79 million in gross proceeds. The funds are intended to extend the company’s cash runway into 2026, potentially beyond the commercial launch of their therapeutic candidate PRGN-2012.

In another strategic move, Precigen completed the sale of certain assets, including intellectual property and royalty rights related to FCX-007, to Innovator 21, LLC for $8.5 million. This transaction concludes Precigen’s involvement with the FCX-007 product candidate. The proceeds from this sale will be used for working capital and general corporate purposes. These recent developments highlight Precigen’s efforts to streamline its portfolio and focus on core areas of expertise.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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