H.C. Wainwright reduced its price target for Regenxbio Inc. to $21 from $23 while maintaining a buy rating, following the U.S. Food and Drug Administration's clinical hold on the company's gene therapy RGX-121.
The FDA imposed the hold on Monday after identifying asymptomatic findings on spinal MRI scans in five participants of the CAMPSIITE trial, which administers RGX-121 via intracisternal or intraventricular routes. Investigators deemed the findings non-serious, and radiologists assessed them as likely benign, with no brain nodules or masses detected. The trial participants had received the therapy 3 to 6 years prior, and the hold follows a prior suspension related to RGX-111.
Regenxbio's shares fell nearly 26% over the past week, trading at $8.91 at the time of the report. The company's Q2 2026 results showed adjusted earnings per share of $0.43, exceeding the $0.25 forecast, though revenue totaled $108.02 million, below the expected $113.74 million. Revenue included a $100 million milestone payment from AbbVie, contributing to cash reserves exceeding $310 million after a public stock offering.
Barclays also adjusted its outlook, setting a $12 price target and downgrading Regenxbio to neutral weight. Regenxbio stated that its other pipeline assets, including therapies for Duchenne and retinal diseases, remain on track, as they utilize different capsids and administration routes. The company does not expect to resubmit the Biologics License Application for RGX-121 in the near term and has removed the candidate from its financial model.
The FDA's clinical hold on RGX-121 follows an expanded MRI monitoring plan implemented after the prior hold on RGX-111, reflecting heightened regulatory scrutiny on gene therapy safety.
