H.C. Wainwright has reduced its target price for Regenxbio Inc. (RGNX) to $32.00 from $34.00 and retained a Buy rating following the company's receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration. The CRL relates to Regenxbio's Biologics License Application for RGX-121, a gene therapy candidate for Mucopolysaccharidosis II (MPS II, or Hunter syndrome).
The company’s share price has reacted sharply to the regulatory news. RGNX is trading at $8.85 and has fallen 17.7% over the past week. Analyst price targets on the stock now range widely, from $12 to $50.
The FDA’s rejection of the BLA centered on several issues. Regulators cited concerns about the study eligibility criteria, the comparability of the natural history external control used in analyses, and whether the surrogate endpoint chosen was appropriate for demonstrating clinical benefit. In its correspondence, the agency laid out several possible paths Regenxbio could pursue, including conducting a new clinical study, treating additional patients and collecting longer-term follow-up data, or employing an untreated control arm in future studies.
Regenxbio has said it will request a Type A meeting with the FDA to discuss the CRL and options for a potential BLA resubmission. The company is also contending with clinical holds placed by the FDA on its RGX-111 and RGX-121 programs after a neoplasm was found in a trial participant, according to the reporting on these developments.
Financial and valuation metrics included in the reporting indicate that Regenxbio is burning through cash at a rapid pace while carrying a moderate level of debt. H.C. Wainwright estimated the firm's market value at $1.64 billion. InvestingPro metrics cited in the report suggest the stock may appear undervalued on Fair Value measures despite recent setbacks.
While H.C. Wainwright has adjusted its price target down to $32 and kept a Buy rating, other analysts have also moved their forecasts. Goldman Sachs lowered its target from $14.00 to $12.00 and maintained a Neutral rating. Separately, Baird continues to rate the stock as Outperform with a $39.00 target. The reporting also notes, in another mention, that H.C. Wainwright reiterated a Buy rating with a $34.00 price target.
H.C. Wainwright highlighted the ultra-rare nature of MPS II, noting there are approximately 500 diagnosed patients in the U.S. and that, to date, no approved therapy addresses the neurocognitive decline associated with the disease. The firm indicated that the delay in the RGX-121 program does not affect Regenxbio's RGX-202 program.
Investors looking for further analysis have access to Regenxbio’s detailed Pro Research Report through InvestingPro, which covers more than 1,400 U.S. equities and includes financial metrics and Fair Value calculations. The company is scheduled to report quarterly earnings in roughly 20 days, on March 3, 2026.
Summary
Regulatory concerns raised by the FDA over study design and endpoints led to a CRL for RGX-121. H.C. Wainwright cut its price target to $32 while keeping a Buy rating as the company plans to request a Type A meeting. The stock has fallen significantly and analyst targets now span a wide range.
Key points
- H.C. Wainwright lowered its target for RGNX to $32 from $34 and maintained a Buy rating following an FDA Complete Response Letter concerning RGX-121.
- The FDA identified issues with study eligibility, the comparability of the natural history external control, and the suitability of the surrogate endpoint; it suggested possible next steps including a new study, additional treated patients with longer follow-up, or an untreated control arm.
- Regenxbio plans to request a Type A meeting with the FDA; the company faces clinical holds on RGX-111 and RGX-121 after a neoplasm was discovered in a trial participant.
Risks and uncertainties
- Regulatory risk - The FDA's CRL and clinical holds introduce uncertainty about the timing and pathway to potential approval for RGX-121, affecting the biotech sector and investors in gene therapy programs.
- Financial risk - Reports indicate rapid cash burn and moderate debt, which could pressure the company’s balance sheet and access to capital amid extended development timelines, impacting market and biotech investors.
- Clinical risk - The discovery of a neoplasm in a trial participant has prompted clinical holds, adding uncertainty to program continuity and timelines for both RGX-111 and RGX-121.
No additional disclosure provided.