S&P 500 falls as traders turn sour on tech
Investing.com - Goldman Sachs maintained its Neutral rating and $14.00 price target on Regenxbio Inc. (NASDAQ:RGNX), a $491 million biotechnology company, following positive 12-month clinical data for the company’s Hunter syndrome treatment. According to InvestingPro data, analysts’ targets range from $14 to $52, with the stock currently trading at $9.71.
The data for clemidsogene lanparvovec (RGX-121) was presented Thursday at the International Congress of Inborn Errors of Metabolism in Kyoto, Japan, which runs from September 2-6.
Goldman Sachs noted the consistency of longer-term data makes the firm "incrementally positive" ahead of the FDA’s upcoming PDUFA date for the therapy.
Safety data beyond one year was comparable with previous findings from the dose-finding portion of the study, with only one treatment-related adverse event of elevated liver enzymes reported, which resolved with steroid therapy.
The research firm observed no apparent dose-response relationship in the safety data, but maintained its Neutral stance on Regenxbio shares despite the positive clinical results.
In other recent news, REGENXBIO Inc. reported positive one-year data for its experimental gene therapy targeting Hunter syndrome, also known as MPS II. The company announced that patients in the CAMPSIITE trial experienced an 82% median reduction in cerebrospinal fluid levels of a key biomarker, heparan sulfate D2S6, after a year of treatment with RGX-121. This development is seen as a step towards potential regulatory approval. Meanwhile, the U.S. Food and Drug Administration has extended its review timeline for RGX-121, moving the Prescription Drug User Fee Act target action date from November 9, 2025, to February 8, 2026. This extension follows the FDA’s request for longer-term clinical data, which REGENXBIO has submitted for all 13 patients in the pivotal study. Additionally, Raymond James has reiterated its Outperform rating and set a $29.00 price target on REGENXBIO, citing the company’s recent data update. The firm emphasized that the results align with previous findings, reinforcing the potential of RGX-121. These recent developments highlight significant progress in REGENXBIO’s efforts to advance its gene therapy program.
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