Regenxbio stock holds steady as FDA extends review of Hunter syndrome gene therapy

Published 19/08/2025, 12:28
Regenxbio stock holds steady as FDA extends review of Hunter syndrome gene therapy

Investing.com - Regenxbio Inc . (NASDAQ:RGNX), a $441 million market cap biotech company whose stock has gained over 16% in the past six months, received notice that the FDA has extended its review timeline for clemidsogene lanparvovec (RGX-121), the company’s gene therapy for Mucopolysaccharidosis II, also known as Hunter syndrome. According to InvestingPro data, the company appears undervalued based on its Fair Value analysis.

The Prescription Drug User Fee Act target action date has been pushed from November 9, 2025, to February 8, 2026, following the company’s submission of longer-term clinical data for all 13 patients in the pivotal study, responding to an FDA information request.

According to Regenxbio, the 12-month data are consistent with biomarker and neurodevelopmental data previously submitted in the Biologics License Application, with plans to present these findings at the International Congress of Inborn Errors of Metabolism in September 2025. While InvestingPro data shows the company maintains strong liquidity with a current ratio of 3.13, it’s worth noting that analysts have recently revised their earnings expectations downward for the upcoming period.

H.C. Wainwright maintained its Buy rating and $34.00 price target on Regenxbio stock, noting that the three-month review extension has "no meaningful impact" on its valuation of the company.

If approved, RGX-121 would be commercialized in the U.S. by NS Pharma, with Regenxbio leading manufacturing and supply chain operations while receiving double-digit royalties on net sales in the U.S. and Asia, plus milestone payments and potential proceeds from a Priority Review Voucher that could be worth $150 million. With analysts forecasting significant sales growth of 238% for FY2025, investors seeking deeper insights can access comprehensive analysis and 8 additional ProTips through InvestingPro’s detailed research report.

In other recent news, Regenxbio Inc. reported its second-quarter 2025 earnings, which fell short of analyst expectations. The company posted an earnings per share of -$1.38, missing the forecasted -$0.94, while revenue came in at $21.36 million, below the projected $38.7 million. These results have drawn attention from analysts, including RBC Capital, which subsequently lowered its price target for Regenxbio from $21.00 to $17.00, although the firm maintained an Outperform rating. Additionally, the U.S. Food and Drug Administration has extended its review timeline for Regenxbio’s gene therapy treatment for Hunter syndrome. The decision date has been moved from November 2025 to February 2026 following the submission of additional long-term clinical data. Meanwhile, Regenxbio continues to work on its Duchenne muscular dystrophy pivotal trial, with a Biologics License Application filing anticipated in mid-2026. The company remains confident in its approach to avoiding liver toxicity issues. These recent developments have sparked varied reactions among investors and analysts.

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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