Shares of REGENXBIO Inc. (RGNX) are up over 20% at $10.43 today. The company recently reported financial results for the fourth-quarter and full-year 2025 and provided details of the upcoming milestones for its investigational candidates in rare disease pipeline.
For the fourth quarter, the firm reported a wider net loss of $67.15 million, or $1.30 per share, compared with $51.19 million, or $1.01 per share, in the prior year.
Revenue jumped to $30.34 million in the fourth quarter of 2025 from $21.21 million in the prior year
For the full year 2025, the net loss narrowed to $193.88 million or $ 3.76 per share from $227.10 million, or $4.59 per share a year ago.
Revenue for the full year climbed to $170.44 million from $83.33 million a year ago.
The company generates revenue by licensing its NAV Technology Platform, including sublicense fees, milestone payments and royalties on net sales of licensed products, Zolgensma and Itvisma, and licensing its intellectual property to collaborators under its license and collaboration agreements with AbbVie and Nippon Shinyaku.
In May 2019, Zolgensma received the FDA approval for the treatment of spinal muscular atrophy (SMA) in pediatric patients who are less than two years of age, and has since been approved in Europe, Japan and other countries.
And in November 2025 FDA has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene, making it the first and only gene replacement therapy available for this broad population.
REGENXBIO continues to receive tiered royalties on sales of Zolgensma and Itvisma.
The firm's late-stage investigational pipeline includes the following.
-RGX-202
RGX-202 is being developed for the treatment of Duchenne muscular dystrophy.
Positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE trial were reported in January 2026.
The company plans to share additional Phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference on March 11, 2026.
In the early second quarter of 2026, REGENXBIO expects to share pivotal topline data.
In mid-2026, REGENXBIO plans to request a pre-BLA meeting to align with the FDA on the BLA submission.
In the first half of 2026, Additional regulatory interactions with the FDA and European Medical Agency (EMA) are planned.
-Clemidsogene lanparvovec (RGX-121)
RGX-121 is in development for Mucopolysaccharidosis (MPS) Type II, also known as Hunter syndrome.
In February 2026, the FDA issued a complete response letter (CRL) for the RGX-121 BLA.
REGENXBIO is addressing the concerns raised in the CRL and plans to resubmit the BLA.
RGX-111 for the treatment of MPS I, also known as Hurler syndrome. Both RGX-121 and RGX-111 are in partnership with Nippon Shinyaku.
Notably, in January 2026, the FDA placed a clinical hold on RGX-111 following preliminary analysis of a single case of neoplasm (intraventricular CNS tumour) in a participant treated in its Phase I/II study.
-Surabgene lomparvovec (sura-vec,ABBV-RGX-314)
ABBV-RGX-314 is being developed for the treatment of wet age-related macular degeneration and diabetic retinopathy, in collaboration with AbbVie.
REGENXBIO expects to share topline data with AbbVie from ATMOSPHERE and ASCENT pivotal trials of ABBV-RGX-314 using subretinal delivery in the fourth quarter of 2026.
Global regulatory submissions are expected in 2027, according to the firm.
ABBV-RGX-314 is also being explored for the treatment of diabetic retinopathy, and REGENXBIO is activating U.S. clinical sites in the pivotal Phase IIb/III NAAVIGATE study.
In the second quarter of 2026, REGENXBIO will receive a $100 million milestone payment from AbbVie upon the first patient being dosed in the Phase IIb portion.
NAAVIGATE is a Phase IIb/III multicenter, randomized, masked, sham-controlled study to evaluate the safety and efficacy of sura-vec in subjects with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME).
If everything goes well, following an interim analysis, REGENXBIO and AbbVie will initiate a Phase III expansion, which will include two Phase III trials, including a U.S. trial and a parallel global trial, led by AbbVie.
With respect to the evaluation of Sura-vec for the treatment of Wet AMD (Suprachoroidal Delivery), enrollment is complete in the Phase II AAVIATE trial.
As of December 31, 2025, the firm reported cash, cash equivalents and marketable securities of $240.9 million, which it expects will fund operations into early 2027.
RGNX has traded between $5.03 and $16.19 in the last year. The stock closed Friday's trade at $8.86.
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April 24, 2026 15:15 ET Economics news flow was relatively light this week even as the conflict in the Middle East continued, raising concerns for policymakers. In the U.S., spending data, initial jobless claims and pending home sales were the highlights. Business confidence in the biggest euro area economy was in focus in Europe. Inflation data from Japan gained attention in Asia.