Bullish indicating open at $55-$60, IPO prices at $37
On Thursday, Stifel analysts reaffirmed their Buy rating on Regenxbio Inc . (NASDAQ:RGNX) stock, maintaining a $40.00 price target, significantly above the current trading price of $8.53. According to InvestingPro data, analysts broadly maintain a bullish stance, with price targets ranging from $13 to $52. The decision follows the release of new positive interim data from the Phase I/II AFFINITY DUCHENNE trial.
The trial data revealed improvements in patients treated with Dose Level 2, showing gains compared to baseline and a slowdown in loss compared to natural history. The data highlighted RGX-202’s unique construct, demonstrating efficacy, consistency, and safety in older patients with muscle deterioration. InvestingPro analysis shows the company maintains a "GOOD" overall financial health score, suggesting strong operational stability despite being in the development phase.
Stifel analysts noted the significance of the functional data, which includes TTR, 10MWR, 4SC, NSAA, and caregiver reports. These results are particularly notable for restoring function in patients aged 6 to 12, who are typically considered beyond the usual treatment window.
The analysts emphasized the importance of C-terminus inclusion in RGX-202, which recruits relevant proteins to muscle sarcolemma. This has been crucial for the rapid enrollment in Phase III, with over 50% completed, and the company anticipates filing a supplemental Biologics License Application by mid-2026.
The positive results from the trial position RGX-202 as a standout gene therapy for Duchenne muscular dystrophy, in a market that has experienced several setbacks. With revenue growth of 80.7% in the last twelve months and a strong balance sheet showing more cash than debt, RGNX appears well-positioned to advance its clinical programs. For deeper insights into RGNX’s financial health and growth prospects, InvestingPro subscribers have access to 8 additional ProTips and comprehensive financial analysis.
In other recent news, REGENXBIO Inc. has reported interim data from its Phase I/II AFFINITY DUCHENNE trial, showcasing positive functional, safety, and biomarker data for its gene therapy RGX-202. This investigational therapy for Duchenne muscular dystrophy demonstrated improved performance on functional measures, with high expression levels of microdystrophin observed in participants. Meanwhile, the company secured a $150 million upfront payment from a royalty bond agreement with Healthcare Royalty, potentially extending its cash runway into early 2027. This agreement could provide up to $250 million in total, supporting late-stage activities such as the potential FDA approval of RGX-121 for MPS II and the submission of a Biologics License Application for RGX-202. Stifel analysts have maintained a Buy rating on REGENXBIO, with a price target of $40, noting the company’s strengthened financial position and readiness for commercialization. Additionally, the FDA has accepted for priority review the Biologics License Application for RGX-121, a potential gene therapy for Mucopolysaccharidosis II, with a target action date set for November 9, 2025. This therapy could become the first gene therapy for MPS II, potentially transforming the treatment landscape. The strategic partnership with Nippon Shinyaku positions NS Pharma, Inc. to lead the commercialization of RGX-121 in the U.S. upon FDA approval.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.