ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX) has reported significant advancements in the treatment of wet age-related macular degeneration (wet AMD (NASDAQ:AMD)), a common retinal disease. At the American Academy of Ophthalmology meeting, data from a Phase II sub-study demonstrated a 97% reduction in treatment burden for patients with bilateral wet AMD who received ABBV-RGX-314, a gene therapy candidate.
The sub-study treated the fellow eye of patients who had previously received ABBV-RGX-314 in one eye, with the goal of determining the safety and efficacy of a single dose in both eyes. At nine months following treatment, the majority of patients required no or just one supplemental injection, and 78% were completely injection-free, maintaining stable visual acuity and retinal thickness.
Curran Simpson, President and CEO of REGENXBIO, highlighted the potential of ABBV-RGX-314 to transform treatment options for wet AMD, offering a one-time treatment for a condition that typically requires frequent lifelong injections. Dr. Arshad Khanani, who presented the data, emphasized the milestone this represents for gene therapy in treating common retinal diseases.
Safety data up to September 11, 2024, indicated that ABBV-RGX-314 was well tolerated, with no serious drug-related adverse events or cases of intraocular inflammation reported. The most common side effects were mild and resolved quickly.
REGENXBIO, in collaboration with AbbVie (NYSE:ABBV), is investigating ABBV-RGX-314 as a potential treatment for not only wet AMD but also diabetic retinopathy and other chronic retinal conditions. The therapy works by inhibiting VEGF, a factor involved in the growth of leaky blood vessels that contribute to fluid accumulation in the retina.
Wet AMD is a significant cause of vision loss, particularly in the United States, Europe, and Japan. Current treatments require ongoing intraocular injections, making adherence challenging and often leading to vision decline over time.
This news is based on a press release statement from REGENXBIO Inc. and represents a promising development in gene therapy for retinal diseases, potentially offering a new standard of care for millions affected by wet AMD.
In other recent news, Regenxbio has seen significant advancements in its gene therapy programs. The company reported positive long-term results from the CAMPSIITE trial of its RGX-121 gene therapy for Hunter syndrome, showing sustained reductions in a key biomarker of brain disease and enabling a significant number of patients to discontinue standard enzyme replacement therapy. Raymond James resumed coverage on Regenxbio with an Outperform rating, highlighting the potential for growth driven by the company's diverse pipeline of treatments.
Regenxbio also announced the appointment of Mitchell Chan as its new Executive Vice President and Chief Financial Officer. H.C. Wainwright maintained a Buy rating on Regenxbio, reflecting a positive outlook on the company's prospects following the recent trial results. The biotechnology firm is preparing for a rolling Biologics License Application submission in the third quarter of 2024, which could lead to a Priority Review Voucher in 2025.
During its Q2 2024 earnings call, Regenxbio reported a solid financial position with $327 million in cash, cash equivalents, and marketable securities. The company also discussed advancements in other priority programs, including gene therapies targeting Duchenne muscular dystrophy, wet age-related macular degeneration, and diabetic macular edema. These are the latest developments in Regenxbio's research and development of gene therapies.
InvestingPro Insights
REGENXBIO's promising advancements in wet AMD treatment come at a critical time for the company. According to InvestingPro data, REGENXBIO's market capitalization stands at $548.6 million, reflecting investor interest in its innovative gene therapy pipeline. However, the company faces financial challenges, with revenue of $89.04 million in the last twelve months as of Q2 2023, representing a decline of 8.18% year-over-year.
Despite these challenges, InvestingPro Tips highlight that REGENXBIO holds more cash than debt on its balance sheet, providing financial flexibility to continue its research and development efforts. This is particularly important given that the company is quickly burning through cash, a common scenario for biotech firms investing heavily in breakthrough therapies like ABBV-RGX-314.
The recent positive data from the Phase II sub-study could explain why REGENXBIO has seen a significant return over the last week, with a 1-week price total return of 8.82%. This uptick suggests that investors are responding favorably to the company's clinical progress.
It's worth noting that REGENXBIO's path to profitability remains uncertain. InvestingPro Tips indicate that analysts do not anticipate the company will be profitable this year, which is reflected in its negative P/E ratio of -2.32 for the last twelve months as of Q2 2023. However, for a biotech company focused on developing groundbreaking treatments, current profitability may be less important than the potential market impact of its therapies.
Investors interested in a deeper analysis of REGENXBIO's financial health and market potential can access additional InvestingPro Tips, with 8 more tips available on the platform to guide investment decisions in this dynamic biotech stock.
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