REGENXBIO swings to Q1 loss, but pivotal Duchenne gene therapy data eyes 2027 launch

REGENXBIO (NASDAQ:RGNX) reported a sharp revenue decline and a widened net loss for the first quarter of 2026, missing consensus estimates.

However, the financial downturn was overshadowed by highly anticipated, positive topline Phase III data for its Duchenne muscular dystrophy gene therapy, sending ripples of optimism through the patient and investor communities.

REGENXBIO announced that total revenue for the first quarter ended March 31, 2026, plummeted to $6.4 million compared to $89 million in the first quarter of 2025.

The steep 93% year-over-year drop was primarily driven by the absence of a one-time $70 million upfront license payment from Nippon Shinyaku recorded in Q1 2025, alongside a $12.2 million decline in Zolgensma® royalty revenues following the expiration of core licensed patents in the United States in January 2026.

The drop in revenue translated to a net loss of $90.1 million, or $1.72 per share, compared to a net income of $6.1 million, or $0.12 per share, in the prior-year period.

The financial numbers missed Wall Street estimates, which had projected a narrower loss per share of $1.34 on revenue closer to $25.8 million.

Despite the financial miss, investor focus locked onto the company's clinical milestone.

REGENXBIO reported that its pivotal Phase III AFFINITY DUCHENNE trial for RGX-202 successfully met its primary endpoint with high statistical significance (p<0.0001).

Data showed that 93% of the 30 participants achieved microdystrophin expression levels above 10% at Week 12.

Crucially, the trial demonstrated a statistically significant correlation between RGX-202 expression and interim functional improvements on the North Star Ambulatory Assessment (NSAA) scale at one year.

The therapy maintained a generally favorable, differentiated safety profile, though the company noted two serious adverse events—one case of myocarditis and one case of asymptomatic liver injury—both of which resolved within weeks.

From a liquidity perspective, REGENXBIO held $150.5 million in cash, cash equivalents, and marketable securities as of March 31, 2026, which it expects will fund operations into early 2027.