Regenxbio's Gene Therapy Outlook Dims As Rival Treatments Gain Ground, Goldman Sachs Warns

Goldman Sachs downgraded Regenxbio Inc (NASDAQ:RGNX), citing gene therapy’s appeal for wet AMD and diabetic retinopathy is uncertain, as most patients are elderly and newer anti-VEGF treatments offer longer-lasting effects at lower costs.

Analyst Paul Choi writes that AbbVie Inc partnered ABBV-RGX-314 faces growing competition, with its invasive delivery method making it less favorable than rival treatments.

Data from the ATMOSPHERE and ASCENT pivotal trials evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with Wet Age-Related Macular Degeneration (wet AMD) are expected in 2026.

AbbVie and REGENXBIO will plan a Phase 3 clinical program for ABBV-RGX-314 in Diabetic Retinopathy with suprachoroidal delivery.

The analyst downgraded Regenxbio to Neutral from Buy and reduced the price forecast to $14 from $38 prior.

Goldman Sachs writes that additional competing treatments are progressing in the clinic, including EyePoint PharmaceuticalsInc.’s Duravyu (vorolanib intravitreal insert), for which the company recently presented topline 6-month results.

The analyst views the appeal of gene therapies in retinal disease overall as decreasing, thus reducing the assumed share of patients who are likely to opt for these treatments. This, in turn, leads to meaningfully lowering projected peak sales for ABBV-RGX-314.

As more people adopt the treatment, the analyst expects prices to keep declining despite changes in the ASP+ system. Ultimately, this could set a lower price benchmark for ABBV-RGX-314 as it nears commercialization.

Separately, the analyst highlights the increasing use of RegeneronPharmaceutical Inc’s 2 mg Eylea biosimilars, which have taken share from the branded version despite the availability of Eylea HD.

Goldman analyst Choi finds the current data on RGX-202 (gene therapy for Duchenne muscular dystrophy) promising but limited. Choi also sees a growing need for better treatments, citing interest in Sarepta Theraputics Inc’s Elevidys and strong enrollment in Edgewise Therapeutics’ FOX study for post-gene therapy care.

Last month, Regenxbio and Nippon Shinyaku announced a strategic partnership to develop and commercialize RGX-121 for Mucopolysaccharidosis II, also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I, also known as Hurler syndrome.

Regenxbio will receive $110 million and up to an additional $700 million if certain milestones are achieved, consisting of $40 million in potential development and regulatory milestones and $660 million in potential sales milestones.

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Price Action: RGNX stock is down 7.29% at $7.315 at last check Tuesday.

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