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On Friday, Stifel analysts maintained a positive stance on Regenxbio Inc . (NASDAQ:RGNX), reiterating a Buy rating with a $40.00 price target on the company’s shares. Currently trading at $7.45, the stock sits well below analyst targets ranging from $14 to $52, according to InvestingPro data. The company’s market capitalization stands at $376 million. The firm expressed confidence in the biotechnology company’s prospects for the year 2025, which they believe will be pivotal for Regenxbio’s transition to gene therapy (GTx) commercialization.
The analysts highlighted the anticipated completion of Regenxbio’s first Biologics License Application (BLA) for RGX-121, a treatment for Mucopolysaccharidosis Type II (MPS II), on an accelerated review timeline in the second half of 2025. The approval of RGX-121 is considered foundational for the company’s RGX-202 program for Duchenne muscular dystrophy (DMD). Phase 3 enrollment for RGX-202 is reportedly about 50% complete, bolstered by consistent biomarker data across a broad age range and early functional data that support its unique construct. InvestingPro analysis shows the company maintains a healthy current ratio of 2.69, indicating strong ability to fund its clinical programs.
In the field of retinal disease, Regenxbio’s partner AbbVie (NYSE:ABBV) continues to enroll patients in Phase 3 trials for ABBV-RGX-314 for wet age-related macular degeneration (wAMD), with trial completion expected in 2025 and results anticipated in 2026. AbbVie is also preparing to initiate pivotal studies for RGX-314 in diabetic retinopathy (DR), a condition that currently has limited treatment options. Both programs are seen as potential long-term growth drivers for AbbVie’s ophthalmology franchise.
The Stifel analysts underscored the value proposition of Regenxbio’s stock, noting that it is trading below its cash position of $244.9 million and has more than $450 million pending in fees and Priority Review Voucher (PRV) value. They believe that the realization of value from Regenxbio’s gene therapy pipeline represents a significant opportunity for investors. InvestingPro confirms the company holds more cash than debt on its balance sheet, though it’s currently burning through cash rapidly. Get access to 8 additional ProTips and comprehensive financial analysis with an InvestingPro subscription.
In other recent news, Regenxbio Inc. reported its fourth-quarter 2024 earnings, surpassing analyst expectations with an earnings per share (EPS) of -1.01, compared to the forecasted -1.13. However, the company’s revenue fell short, reporting $21.21 million against a forecast of $23.47 million. Despite this revenue miss, Regenxbio highlighted progress in its gene therapy programs, including a submitted Biologics License Application (BLA) for RGX-121 targeting Hunter Syndrome. The company is also advancing its Duchenne muscular dystrophy (DMD) gene therapy study and collaborating with AbbVie on wet AMD (NASDAQ:AMD) and diabetic retinopathy programs. Regenxbio ended the quarter with a strong cash position of $245 million, which it expects to fund operations into the second half of 2026. The company’s research and development expenses decreased to $209 million in 2024 from $232 million in 2023. Analysts have noted the company’s robust pipeline and potential future milestones, including the anticipated FDA approval of RGX-121 by the fourth quarter of 2025.
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