Regenxbio stock rating reiterated at Buy by H.C. Wainwright on positive trial data

Investing.com - H.C. Wainwright reiterated its Buy rating and $34.00 price target on Regenxbio Inc. (NASDAQ:RGNX), whose shares have surged over 53% in the past six months. The renewed confidence comes following new 12-month data from the Phase 1/2/3 CAMPSIITE trial of clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis II treatment. According to InvestingPro data, analysts’ targets for RGNX range from $14 to $52, reflecting significant upside potential.

The data, presented at the International Congress of Inborn Errors of Metabolism 2025, showed subjects in the pivotal phase maintained an 82% median reduction of cerebrospinal fluid levels of heparan sulfate D2S6 through one year, consistent with previously reported topline results that met the trial’s primary endpoint with statistical significance (p

Pivotal subjects demonstrated neurodevelopmental skill acquisition or stability on all sub-scales of the Bayley Scales of Infant and Toddler Development at one year, showing a strong correlation between measured CSF HS D2S6 levels at Week 16 and neurocognitive outcomes at one year.

Regenxbio has submitted these longer-term pivotal data to the FDA for the ongoing Biologics License Application review of RGX-121, with the FDA having completed a pre-license inspection and bioresearch monitoring information inspection in August 2025 with no observations.

H.C. Wainwright noted that the positive 12-month data should help RGX-121 secure U.S. regulatory approval in February 2026. With revenue growth forecasts exceeding 200% for fiscal year 2025, investors seeking deeper insights into RGNX’s potential can access comprehensive analysis and additional ProTips through InvestingPro’s detailed research reports, which transform complex Wall Street data into actionable intelligence.

In other recent news, REGENXBIO Inc. announced significant progress in its experimental gene therapy for Hunter syndrome, with positive one-year data showing an 82% median reduction in cerebrospinal fluid levels of a key biomarker. This development strengthens the company’s case for potential regulatory approval. However, the U.S. Food and Drug Administration has extended the review timeline for RGX-121, moving the Prescription Drug User Fee Act goal date to February 8, 2026, after requesting longer-term clinical data. The company has already submitted the required information for all 13 patients involved in the pivotal trial. Meanwhile, Raymond James reiterated its Outperform rating for REGENXBIO, maintaining a $29 price target based on the promising MPS II data. Similarly, Goldman Sachs maintained its Neutral rating with a $14 price target following the positive clinical data presentation at an international congress. These recent developments reflect ongoing interest and cautious optimism from analysts regarding REGENXBIO’s gene therapy program.

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