Overview
H.C. Wainwright has reaffirmed a Buy rating and retained a $34.00 price target on Regenxbio Inc. (NASDAQ: RGNX) after the company disclosed that the U.S. Food and Drug Administration placed clinical holds on two of its investigational gene therapies. The firm’s target implies more than 200% upside relative to the company’s last quoted price of $11.01. InvestingPro data cited alongside the development indicates the stock is trading below its Fair Value and has dropped 31.1% over the past week.
Details of the clinical holds
Regenxbio said the FDA paused the programs for RGX-111, intended for mucopolysaccharidosis type I (MPS I or Hurler syndrome), and RGX-121, for MPS II (Hunter syndrome), after a neoplasm was identified in a trial participant. The tumor was discovered during a routine brain MRI in an asymptomatic participant who had received intracisternal RGX-111 four years earlier as part of the Phase 1/2 study. The finding triggered the clinical hold on both programs.
Preliminary findings and ongoing investigation
According to H.C. Wainwright, preliminary genetic testing of the resected tumor identified an adeno-associated virus (AAV) vector genome integration event associated with overexpression of the proto-oncogene PLAG1. The firm emphasized that the probe into whether this serious adverse event is causally related to the investigational therapy remains in progress.
The research firm also noted that no neoplasm has been reported among the nine other participants treated with RGX-111. The affected participant reportedly remains asymptomatic and is showing developmental progress post-treatment.
Financial context and upcoming milestones
InvestingPro’s commentary included a cautionary note that Regenxbio is "quickly burning through cash," while reporting a current ratio of 2.66, indicating liquid assets exceed short-term liabilities. The company is scheduled to report its next earnings on March 3, 2026.
The FDA action has also delayed the Prescription Drug User Fee Act (PDUFA) date for RGX-121, which had been scheduled for February 8, 2026, as additional laboratory analyses are required to inform regulators.
Analysts’ reactions beyond H.C. Wainwright
Despite the regulatory holds, other research houses have maintained favorable stances on Regenxbio’s shares. Baird reiterated an Outperform rating with a $39.00 price target, and Stifel kept a Buy rating with a $45.00 target. Both firms acknowledged the clinical hold while expressing continued confidence in the company’s prospects.
Investor considerations
Investors tracking Regenxbio should weigh the immediate regulatory setback and the ongoing investigation into the tumor against the continued analyst support and the company’s liquidity position as reflected in the current ratio. The clinical holds affect development timelines for two core gene therapy programs and have triggered reassessment by regulators and researchers.
Key takeaways
- H.C. Wainwright reaffirmed a Buy rating and $34.00 price target on Regenxbio despite FDA clinical holds on RGX-111 and RGX-121.
- The clinical holds followed discovery of an intraventricular CNS tumor in a participant who received RGX-111 four years earlier; preliminary tumor testing detected AAV vector genome integration with PLAG1 overexpression.
- Other analysts including Baird and Stifel have maintained positive ratings, even as the FDA delayed the RGX-121 PDUFA date to allow for further laboratory work.