REGENXBIO to advance diabetic retinopathy treatment to Phase IIb/III trial

ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX), currently valued at $414.7 million and showing strong revenue growth of 80.7% over the last twelve months, announced Thursday it will initiate a pivotal Phase IIb/III clinical trial for surabgene lomparvovec in diabetic retinopathy using suprachoroidal delivery, following positive two-year data from its Phase II ALTITUDE trial. According to InvestingPro analysis, the company appears undervalued based on its Fair Value metrics.

The company will receive $100 million from collaboration partner AbbVie when the first subject is dosed in the Phase IIb/III trial, and an additional $100 million when the first subject is dosed in a second Phase III clinical trial, according to a press release statement. InvestingPro data shows REGENXBIO maintains a healthy balance sheet with more cash than debt and a current ratio of 2.93, providing strong financial flexibility for trial advancement.

The Phase II ALTITUDE trial demonstrated that surabgene lomparvovec was well tolerated in subjects with non-proliferative diabetic retinopathy through two years of follow-up. At dose level 3, no intraocular inflammation was observed with short-course topical prophylactic steroids.

The upcoming pivotal two-part placebo-controlled Phase IIb/III trial will use a primary endpoint of greater than 2-step improvement on the Diabetic Retinopathy Severity Scale at one year.

"Advancing our DR program to late-stage development brings sura-vec closer to being a potentially transformative new treatment for the millions of people living with DR," said Steve Pakola, Chief Medical Officer at REGENXBIO.

As part of the amended collaboration agreement, AbbVie will independently advance and fund a new Phase III ACHIEVE trial in wet AMD to assess potential reduction in injection burden compared to standard of care.

Diabetic retinopathy affects nearly 10 million people in the United States and is the leading cause of vision loss in working-age adults worldwide. Surabgene lomparvovec is being investigated as a potential one-time treatment that inhibits vascular endothelial growth factor (VEGF). With analysts projecting profitability this year and setting price targets ranging from $13 to $52, detailed analysis and additional insights are available in the comprehensive Pro Research Report on InvestingPro, which covers this and 1,400+ other US stocks.

In other recent news, REGENXBIO Inc. has released interim data from its Phase I/II AFFINITY DUCHENNE trial, highlighting positive functional, safety, and biomarker results for its investigational gene therapy, RGX-202. The data showed improvements in functional measures and a slowdown in disease progression, with participants performing better than external natural history controls at 9 and 12 months post-treatment. Additionally, REGENXBIO’s preclinical research demonstrated that its microdystrophin gene therapy construct with the C-terminal domain showed improved muscle function in treating Duchenne muscular dystrophy. Analyst firms H.C. Wainwright and Stifel have both reiterated their Buy ratings on REGENXBIO stock, maintaining price targets of $34 and $40, respectively, following the positive trial data. Stifel also highlighted REGENXBIO’s strategic agreement with Healthcare Royalty, which provides up to $250 million in exchange for royalties and milestones from its gene therapy products. This deal allows REGENXBIO to maintain full rights to its key opportunities in Duchenne muscular dystrophy and ophthalmology. These developments underscore the company’s progress in advancing its gene therapy treatments.

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