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Wednesday, Regenxbio Inc . (NASDAQ:RGNX) saw an increase in its price target from $27.00 to $29.00 by Raymond (NSE:RYMD) James, with the firm reiterating an Outperform rating on the stock. According to InvestingPro data, analyst targets for RGNX range from $14 to $52, with the stock currently trading at $8.39, suggesting significant upside potential. The stock has already shown momentum with a 16.94% gain over the past week. The revision followed Regenxbio’s presentation of updated results from the Phase 1/2 segment of their AFFINITY DUCHENNE trial at the Muscular Dystrophy Association annual meeting on March 17, 2025. While the company focuses on advancing its clinical programs, InvestingPro data shows it maintains a strong liquidity position with a current ratio of 2.69, indicating sufficient resources to fund its development pipeline.
The company shared new biomarker data for two additional patients at dose level 2, considered the pivotal dose for the study. While no new functional data was released during the meeting, such information is anticipated in the first half of 2025. The updated dataset reportedly showcases variability in expression levels, yet consistently demonstrates over 10% microdystrophin levels at the pivotal dose level, which is the primary endpoint for the AFFINITY DUCHENNE study’s pivotal cohort.
Raymond James highlighted the safety profile of Regenxbio’s therapy, especially in the wake of a liver toxicity-related fatality of a 16-year-old patient treated with a competing therapy, Elevidys. The firm’s analysis suggests that safety remains a key selling point for Regenxbio’s RGX-202 treatment. InvestingPro analysis reveals the company maintains more cash than debt on its balance sheet, though investors should note it’s currently burning through cash reserves. For deeper insights into RGNX’s financial health and additional ProTips, consider exploring InvestingPro’s comprehensive research report.
The price target adjustment reflects a cautiously optimistic stance, acknowledging the small sample size of the study while also considering a slower uptake of Elevidys and increased royalties from Zolgensma, another gene therapy. The analyst from Raymond James expressed continued confidence in the potential of RGX-202, which contributed to the decision to maintain the Outperform rating and raise the price target.
In other recent news, REGENXBIO Inc. reported its fourth-quarter 2024 financial results, revealing revenue of $21.2 million, which fell short of the forecasted $23.47 million. However, the company exceeded earnings expectations with an EPS of -$1.01, compared to the anticipated -$1.13. The company has completed the Biologics License Application (BLA) for RGX-121, a treatment for Mucopolysaccharidosis II, and is seeking accelerated approval with a potential FDA decision in late 2025. Meanwhile, REGENXBIO is advancing its RGX-202 gene therapy for Duchenne muscular dystrophy, showing positive interim data in its Phase I/II trial.
H.C. Wainwright adjusted the price target for REGENXBIO shares to $34, maintaining a Buy rating, influenced by the company’s recent financial performance and the progress of RGX-121. Stifel analysts also reiterated a Buy rating with a $40 target, expressing confidence in REGENXBIO’s transition to gene therapy commercialization in 2025. The company is collaborating with AbbVie (NYSE:ABBV) on RGX-314 for retinal diseases, with Phase 3 trials underway and results expected in 2026.
REGENXBIO holds a strong cash position of $245 million and anticipates significant milestones in 2025, including potential FDA approval for RGX-121. The company is preparing for multiple gene therapy launches, aiming for sustainable profitability and leveraging its partnerships to drive growth in the gene therapy sector.
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