ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX) has shared optimistic results from a Phase I/IIa trial of ABBV-RGX-314, a potential one-time gene therapy for wet age-related macular degeneration (wet AMD (NASDAQ:AMD)). The study, recently published in The Lancet, reveals that patients receiving a single dose of ABBV-RGX-314 experienced stable or improved vision and retinal health up to two years post-treatment.
The company's announcement today indicates that ABBV-RGX-314 could offer a significant advancement in treating wet AMD, a chronic condition that often leads to vision loss and currently requires frequent anti-VEGF injections. The gene therapy aims to reduce the treatment burden by potentially providing long-term benefits from just one administration.
ABBV-RGX-314 employs the NAV® AAV8 vector to deliver an antibody fragment designed to inhibit the VEGF pathway, which is responsible for the growth of leaky blood vessels that contribute to fluid accumulation in the retina. This approach could serve as a new standard of care for managing wet AMD and other chronic retinal conditions.
The therapy is also under evaluation in two pivotal trials, ATMOSPHERE® and ASCENT™, which are on track and expected to support global regulatory submissions by late 2025 to early 2026.
Kenneth T. Mills, President and CEO of REGENXBIO, expressed confidence in the multi-billion-dollar potential of ABBV-RGX-314 as a first-in-class gene therapy. Jeffrey S. Heier, M.D., the primary investigator for the trial, emphasized the need for a treatment that can alleviate the significant vision loss and treatment burden associated with wet AMD.
REGENXBIO is advancing ABBV-RGX-314 in partnership with AbbVie (NYSE:ABBV) and is exploring two delivery methods: subretinal and suprachoroidal, the latter facilitated by an exclusive license with Clearside Biomedical (NASDAQ:CLSD), Inc.
Wet AMD is a leading cause of vision loss in the U.S., Europe, and Japan, affecting up to 2 million individuals. Current treatments, although effective, pose adherence challenges due to the frequency of injections required.
This news is based on a press release statement and provides a glimpse into the potential for gene therapy to transform the treatment landscape for patients with wet AMD.
InvestingPro Insights
As REGENXBIO Inc. (NASDAQ:RGNX) continues to make strides with its gene therapy for wet age-related macular degeneration, the financial metrics and analyst insights provide a broader context for the company's market position and future outlook. Despite the clinical advancements, InvestingPro Tips suggest that analysts are cautious about the company's profitability in the near term. They do not anticipate REGENXBIO will be profitable this year, and the company is quickly burning through cash, with weak gross profit margins reported over the last twelve months as of Q1 2023.
However, it's not all challenging news for REGENXBIO. The company's liquid assets exceed its short-term obligations, indicating a level of financial stability. Additionally, the firm operates with a moderate level of debt, which could be a strategic advantage as it continues to invest in its innovative treatments. On the performance front, REGENXBIO has seen a strong return over the last three months and a large price uptick over the last six months, reflecting investor optimism in the wake of positive clinical trial results.
InvestingPro Data shows a market capitalization of $1.09 billion USD and a negative P/E ratio of -4.17, highlighting the company's current lack of profitability. Revenue growth has declined by approximately 19.94% over the last twelve months, which may be a concern for potential investors. Nevertheless, the company's share price performance, with a 25.45% total return over the last six months, suggests that the market is reacting positively to the company's long-term potential.
For those considering an investment in REGENXBIO, or for current shareholders looking to deepen their understanding of the company's financial health and prospects, additional InvestingPro Tips are available. There are 9 more tips that provide granular insight into the company's performance and forecasts, accessible on InvestingPro. Use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, offering valuable tools for investors to make informed decisions.
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