Regenxbio (NASDAQ:RGNX) shares gained more than 4% in premarket trading Thursday after investors focused on encouraging clinical trial results for the company’s Duchenne muscular dystrophy therapy, despite weaker-than-expected quarterly financial results.
Earnings and Revenue Miss Expectations
Regenxbio reported a first-quarter loss of $1.72 per share, wider than analyst expectations of a $1.34 loss per share.
Revenue totaled $6.39 million, well below the consensus estimate of $25.8 million.
The company’s revenue declined 93% year-on-year from $89.0 million in the first quarter of 2025.
Regenxbio said the drop was largely due to a $70 million upfront license payment from Nippon Shinyaku recognized in the prior-year period, as well as a $12.2 million reduction in ZOLGENSMA royalty revenue after certain licensed U.S. patents expired in January 2026.
Duchenne Trial Meets Primary Endpoint
Investor sentiment improved after the company announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE trial evaluating RGX-202.
According to Regenxbio, the study achieved its primary endpoint with high statistical significance.
The company said 93% of patients reached RGX-202 microdystrophin expression levels above 10%, with a reported p-value of less than 0.0001.
The treatment also demonstrated a statistically significant relationship between microdystrophin expression and functional improvement.
CEO Highlights Transformational Year
President and chief executive Curran Simpson said the company continues to make progress across its late-stage pipeline.
“REGENXBIO enters a transformative year with positive momentum, reaching significant late-stage milestones to support our potential first- and best-in-class gene therapies,” Simpson said.
Research Spending Continues to Increase
Research and development expenses rose to $57.3 million from $53.1 million in the prior-year quarter.
The increase was mainly linked to clinical trial costs associated with RGX-202, along with higher personnel expenses.
General and administrative expenses also increased modestly to $21.3 million from $20.3 million a year earlier.
Cash Position Expected to Support Operations Into 2027
As of March 31, 2026, Regenxbio held $150.5 million in cash, cash equivalents and marketable securities.
The company said its current liquidity is expected to fund operations into early 2027.
This outlook excludes any potential future milestone payments from partners.
Regenxbio also said it expects to receive a $100 million milestone payment from AbbVie once the first patient is dosed in the Phase IIb portion of the NAAVIGATE study, which is expected during the second quarter of 2026.