Baird has reaffirmed its Outperform rating on Regenxbio Inc. (NASDAQ: RGNX) and left its price target at $39.00 after the U.S. Food and Drug Administration placed clinical holds on the company’s MPS gene therapy programs. That $39 target implies substantial upside from the stock’s recent level of $11.01. Analyst targets across the coverage universe run from $14 to $52, according to InvestingPro data.
The FDA’s clinical hold affects both the RGX-111 and RGX-121 programs following the discovery of an asymptomatic neoplasm in a patient treated for MPS I (Hurler syndrome) during Phase 1/2 studies. Baird’s note states the agency extended the hold to the MPS II (Hunter syndrome) program as well, citing similarities across the programs.
Baird sees the regulatory action as likely to delay approval of RGX-121. However, the research firm indicates this potential postponement has been factored into standard gene therapy labeling assumptions. Baird does not identify an obvious basis to assign Regenxbio’s programs a higher safety risk than other approved gene therapies, referencing therapies such as Zolgensma as a comparison point in its analysis.
Separate market-moving items have also influenced investor perspectives on Regenxbio. The FDA-imposed holds and the tumor finding in a study participant prompted a delay in the PDUFA date for RGX-121, which had been set for February 2026. Stifel, while acknowledging the regulatory and laboratory review process that underlies the delay, has reiterated a Buy rating with a $45.00 price target, per recent coverage.
Counterbalancing the MPS program setbacks are positive efficacy signals from Regenxbio’s RGX-202 program in Duchenne muscular dystrophy. The company reported 18-month results from the AFFINITY DUCHENNE trial indicating that all four treated patients outperformed expected disease progression on the North Star Ambulatory Assessment. Those results, measured against matched natural history subjects, prompted both H.C. Wainwright and Baird to reaffirm favorable ratings - Buy and Outperform, respectively - with price targets of $34.00 and $39.00.
The near-term picture for Regenxbio is therefore mixed. On one hand, regulatory scrutiny and the extension of clinical holds introduce uncertainty and delay for key MPS assets. On the other hand, encouraging clinical performance in the RGX-202 Duchenne program supports the case for pipeline value outside the MPS franchise and has prompted continued buy-side support from multiple research firms.
Investors and market participants will be watching closely for further communications from the company and regulators about the scope of the holds, laboratory findings, and any implications for study conduct or labeling. For now, the analyst community appears to be balancing the operational and regulatory risks affecting the MPS programs against the positive signals from other trials in Regenxbio’s pipeline.
Summary
Baird has reiterated an Outperform rating and a $39 price target on Regenxbio after the FDA placed clinical holds on RGX-111 and RGX-121 following the discovery of an asymptomatic neoplasm in a Phase 1/2 MPS I patient. The FDA extended the hold to the MPS II program due to program similarities. Baird expects delays to RGX-121 approval but considers that risk priced into gene therapy labels. Other firms including Stifel, H.C. Wainwright, and Baird itself have maintained positive ratings in light of encouraging RGX-202 Duchenne data.
Key points
- Baird reaffirmed Outperform and a $39.00 target for RGNX despite FDA clinical holds on MPS programs; current share price cited at $11.01, with analyst targets spanning $14 to $52.
- The FDA placed holds after an asymptomatic neoplasm was found in an MPS I patient in Phase 1/2 trials and extended the hold to MPS II due to program similarities, per Baird.
- Positive 18-month data from the RGX-202 AFFINITY DUCHENNE trial showed all four patients exceeded expected disease progression, supporting continued Buy/Outperform stances from multiple firms.
Risks and uncertainties
- Regulatory delays: The FDA’s clinical holds and ensuing reviews can postpone approval timelines, exemplified by the delayed PDUFA date for RGX-121 originally set for February 2026 - a near-term risk to program commercialization timelines and valuation.
- Safety signal: Discovery of an asymptomatic neoplasm in a trial participant introduces safety uncertainty for the MPS programs and may affect trial conduct, labeling, or public perception of gene therapies.
- Program linkage risk: The FDA extended the hold from an MPS I program to MPS II because of similarities between programs, illustrating how safety findings in one asset can propagate regulatory action across related programs.
Tags: biotech, gene-therapy, healthcare, clinical-trials