REGENXBIO (NASDAQ:RGNX) reported second-quarter 2026 progress across its late-stage gene-therapy pipeline, highlighting completed enrollment in its Duchenne muscular dystrophy confirmatory study, an agreed path to resubmit its Hunter syndrome therapy application, and the start of a pivotal diabetic retinopathy study under its AbbVie collaboration.
President and Chief Executive Officer Curran Simpson said the company strengthened its balance sheet through more than $200 million in financing and milestone proceeds subsequent to the quarter. REGENXBIO ended the quarter with $106 million in cash, cash equivalents and marketable securities, and said it had more than $310 million on a pro forma basis after receiving a $100 million AbbVie milestone payment and completing a follow-on public offering that generated about $108 million in net proceeds.
RGX-202 Duchenne Program Advances Toward BLA Submission
REGENXBIO said it completed enrollment and dosing in the confirmatory study for RGX-202, its wholly owned gene-therapy candidate for Duchenne muscular dystrophy, ahead of schedule. The pivotal and confirmatory trials have enrolled more than 60 patients in total, providing the safety database intended to support a planned biologics license application.
The company plans to submit the first BLA module to the U.S. Food and Drug Administration during the third quarter of 2026 and expects to complete the BLA filing in the first quarter of 2027. Simpson said potential U.S. approval could come during the second half of 2027.
Management cited pivotal data reported in May, including microdystrophin expression, functional improvement and a favorable safety profile. Chief Medical Officer Steve Pakola said the results included a statistically significant correlation between microdystrophin expression and improvement in North Star Ambulatory Assessment scores.
At the time of the clinical-module submission, REGENXBIO expects roughly half of the 30 patients treated in the pivotal portion of the study to have completed 12-month functional assessments, Simpson said. He added that the FDA has not specified a minimum amount of functional data required for an accelerated-approval submission.
The company also plans to begin AFFINITY RISE, an ex-U.S., double-masked, placebo-controlled randomized study, in the first half of 2027. The study is designed to enroll about 100 patients using a 2-to-1 active-treatment-to-placebo randomization. Pakola said the trial is expected to include a crossover opportunity for patients initially assigned to placebo, though the company did not provide further design details or identify enrollment regions.
REGENXBIO said it is manufacturing intended commercial supply for RGX-202 at its FDA-inspected, commercial-ready Rockville, Maryland, facility and is investing in U.S. launch preparation.
FDA Aligns on RGX-121 Resubmission
For RGX-121, a potential treatment for mucopolysaccharidosis type II, or Hunter syndrome, REGENXBIO said it reached alignment with the FDA following a June discussion and a Type A meeting in July. According to Simpson, the FDA confirmed that the company’s available data are sufficient for review under the accelerated-approval pathway and that no additional studies, including a randomized controlled trial, will be required for BLA resubmission.
The resubmission, planned for the third quarter, will include longer-term efficacy and safety data, including participant imaging. Simpson said the package incorporates two-year biomarker and neurocognitive data, along with updated safety information, rather than requiring newly dosed patients or data beyond the two-year horizon.
Chief Legal Officer Patrick Christmas also addressed REGENXBIO’s royalty portfolio. He said the company’s U.S. patent coverage for Zolgensma has expired, though it retains coverage in about 20 countries outside the United States. He added that the company has coverage for Evrysdi in the U.S. and internationally.
AbbVie Retina Collaboration Reaches New Milestones
REGENXBIO and AbbVie dosed the first patient in the Phase IIb/III NAAVIGATE study of sura-vec for diabetic retinopathy, triggering the $100 million milestone payment to REGENXBIO. The company said the partnership’s near-term focus has shifted to fourth-quarter top-line results from the ATMOSPHERE and ASCENT pivotal studies of subretinal sura-vec in wet age-related macular degeneration.
Pakola said long-term data presented at the American Society of Retina Specialists meeting showed that sura-vec maintained or improved visual acuity and reduced treatment burden through five years in a Phase I/II wet AMD study. In diabetic retinopathy, 2.5-year ALTITUDE data showed durable improvements in disease severity, continued prevention of vision-threatening complications and a favorable long-term safety profile following a single administration, he said.
REGENXBIO said the two wet AMD pivotal trials are designed with 90% power and use a 4.5-letter non-inferiority margin. Simpson said ATMOSPHERE and ASCENT results will be released together because their timing is closely aligned. The companies expect to disclose the primary endpoints, while details on secondary-endpoint disclosure will be determined closer to the data release.
AbbVie will take the primary commercial leadership role for subretinal wet AMD if the program advances, Simpson said. Pakola added that more than 500 surgeons globally have been trained on the procedure.
About REGENXBIO (NASDAQ:RGNX)
REGENXBIO Inc is a clinical‐stage biotechnology company specializing in the development of gene therapies using its proprietary NAV® AAV (adeno‐associated virus) platform. The company engineers next‐generation AAV vectors designed to deliver functional genes to targeted cells, aiming to address a range of rare genetic diseases and ocular, metabolic and neurologic disorders. REGENXBIO’s pipeline features several product candidates in various stages of preclinical and clinical development, including RGX-314 for wet age‐related macular degeneration, RGX-121 for mucopolysaccharidosis II (Hunter syndrome) and RGX-121 for other rare lysosomal storage diseases.
In addition to its internally funded programs, REGENXBIO has established partnerships with major biopharmaceutical companies to advance its NAV technology.
