REGENXBIO Inc.

REGENXBIO did not state quarterly revenue, EPS, gross margin, or year-over-year financial figures on this call. For RGX-202, the pivotal Phase III portion of AFFINITY DUCHENNE met its primary endpoint with high statistical significance: 93% (28 of 30) of patients exceeded 10% microdystrophin at week 12, and 80% exceeded 40%. Average microdystrophin expression was 71.1% overall and 41.6% in patients aged 8 and older. Interim safety covered 31 dosed patients and included 2 treatment-related SAEs, both resolved without sequelae. Interim 12-month functional data were reported for 9 patients aged 4 and older, with improvement versus external controls across NSAA and timed function tests. Looking ahead, management said it expects to dose 60 patients by midyear, to pursue a potential approval in 2027, and to file a BLA in the early first half of 2027.

Curran Simpson said the company sees RGX-202 as a potential best-in-class Duchenne gene therapy and emphasized the significance of the correlation between microdystrophin and function. He said REGENXBIO has dosed more than 50 patients across pivotal and confirmatory studies, is on track for 60 by midyear, and is planning for potential approval in 2027. He also highlighted the lifting of the partial clinical hold on RGX-121 and said the company remains positioned for three approvals over the next couple of years, including two blockbuster opportunities.

No traditional CFO remarks or financial statements were given in the transcript. The company’s only financial guidance-related comment was operational: in retinal disease, it expects the first patient in the Phase IIb diabetic retinopathy trial in the second quarter, which would trigger a $100 million milestone payment from AbbVie. Management also said it expects a BLA filing in early 2027 and cited having closer to 50 patients in the safety database by filing time based on continued enrollment.

Analysts focused on FDA strategy, asking whether the agency’s apparent interest in a randomized controlled trial could affect accelerated approval, how many 12-month patients would be needed, and whether the company would still file if FDA asked for more. Management said FDA has not reviewed this new data yet, but argued the magnitude of functional effect and the biomarker-function correlation support accelerated approval; it said a randomized trial would likely have to be run ex-U.S. and that the company could pivot the confirmatory study if needed. Questions also centered on the liver SAE and the immune suppression regimen; management explained the liver event was considered an SAE because the patient was hospitalized for administrative reasons, remained asymptomatic, and fully recovered, while investigators said the prophylaxis regimen, including eculizumab and sirolimus, had been generally well tolerated.