Regenxbio Stock Price, News & Analysis

REGENXBIO (Nasdaq: RGNX) announced completion of enrollment in the ATMOSPHERE and ASCENT pivotal trials of subretinal surabgene lomparvovec for wet age-related macular degeneration (wet AMD).

Combined enrollment exceeded 1,200 participants across >200 sites worldwide, making this the largest global gene therapy program reported for an ocular indication. ATMOSPHERE (U.S.) compares sura-vec to ranibizumab; ASCENT (U.S. + 13 countries) compares sura-vec to aflibercept. Primary endpoints are non-inferiority in BCVA at 54 weeks and one year. Company cites durable effects up to 4 years in long-term follow-up and no drug-related serious adverse events in a 60-patient Phase II study. Topline pivotal data expected Q4 2026.

REGENXBIO (Nasdaq: RGNX) announced that Chief Medical Officer Steve Pakola, M.D., will present new analysis of 12-month functional data from the Phase I/II trial of RGX-202 at the International Congress of the World Muscle Society in Vienna, Austria, October 7-11, 2025.

The presentation will highlight that RGX-202 demonstrated a favorable safety profile with no serious adverse events in the Phase I/II study. Notably, pivotal dose participants showed superior performance compared to baseline-matched external natural history controls across all functional measures, suggesting RGX-202's potential as a best-in-class gene therapy for Duchenne muscular dystrophy.

REGENXBIO (NASDAQ:RGNX) has announced positive 12-month pivotal data from its Phase I/II/III CAMPSIITE® trial of RGX-121 for treating MPS II (Hunter syndrome). The trial demonstrated an 82% median reduction in CSF levels of HS D2S6, a key biomarker of MPS II brain disease, sustained through one year.

The study met its primary endpoint with statistical significance (p < 0.0001), showing strong correlation between biomarker levels and neurodevelopmental improvements. Patients demonstrated neurodevelopmental skill acquisition or stability across all sub-scales of the BSID-III assessment at one year.

The FDA completed pre-license and bioresearch monitoring inspections with no observations, and RGX-121 has been well-tolerated in all 26 patients. A decision on the Biologics License Application is expected by February 8, 2026. If approved, RGX-121 would become the first one-time therapy targeting the genetic cause of Hunter syndrome.

REGENXBIO (Nasdaq: RGNX) has announced its participation in four major upcoming investor conferences in September 2025. The company will attend the Wells Fargo Healthcare Conference for one-on-one meetings in Boston on September 4, followed by three fireside chat presentations in New York: the Morgan Stanley Global Healthcare Conference on September 8, and both the Baird Global Healthcare Conference and H.C. Wainwright Global Investment Conference on September 9.

Select fireside chat presentations will be available via webcast through REGENXBIO's investor relations website, with recordings accessible for approximately 30 days after each event.

REGENXBIO (Nasdaq: RGNX) announced that the FDA has extended its review timeline for RGX-121, a potential treatment for Hunter syndrome (MPS II). The PDUFA date has been pushed from November 9, 2025 to February 8, 2026 following the company's submission of additional 12-month clinical data for all 13 patients in the pivotal study.

The FDA's extension comes after completing a pre-license inspection with no observations, and notably, no safety concerns have been raised during the BLA review. RGX-121 holds multiple FDA designations including Orphan Drug, Rare Pediatric Disease, Fast Track, and RMAT. If approved, it would become the first one-time therapy targeting the genetic cause of Hunter syndrome.

REGENXBIO (NASDAQ:RGNX) reported Q2 2025 financial results and significant progress across its gene therapy pipeline. The company's cash position strengthened to $363.6 million, expected to fund operations into early 2027. Key developments include accelerated enrollment in the RGX-202 Duchenne muscular dystrophy pivotal trial, expected to complete by October 2025, with BLA submission planned for mid-2026.

The company reported Q2 2025 revenues of $21.4 million and a net loss of $70.9 million ($1.38 per share). Notable achievements include successful FDA inspections for clemidsogene lanparvovec (RGX-121) for MPS II, with a PDUFA date of November 9, 2025, and advancement of surabgene lomparvovec (sura-vec) for retinal diseases. The company secured a $250 million royalty agreement with Healthcare Royalty and amended its AbbVie collaboration with potential milestone payments of $200 million.

REGENXBIO (NASDAQ:RGNX) announced the advancement of surabgene lomparvovec (sura-vec) into a pivotal Phase IIb/III clinical trial for diabetic retinopathy (DR) using suprachoroidal delivery. The decision follows positive two-year data from the Phase II ALTITUDE® trial, demonstrating durable safety and efficacy with a single injection.

Under an amended collaboration with AbbVie, REGENXBIO will receive $100 million upon first subject dosing in the Phase IIb/III trial and another $100 million for the first subject in a second Phase III trial. The Phase II ALTITUDE trial showed favorable safety results, with no drug-related serious adverse events or intraocular inflammation observed through two years at dose level 3.

REGENXBIO (Nasdaq: RGNX) has scheduled a conference call for Thursday, August 7, 2025, at 8:00 a.m. ET to discuss its second quarter 2025 financial results and recent operational developments. The company will host a webcast that can be accessed through a registration link.

Analysts can participate in the Q&A session by dialing (646) 307-1963 (domestic) or (800) 715-9871 (international) with passcode 9571992. A replay will be available on REGENXBIO's investor website approximately two hours after the call concludes. Participants are encouraged to join 15 minutes before the start time.

REGENXBIO (NASDAQ:RGNX) has published significant preclinical results for RGX-202, their novel gene therapy for Duchenne Muscular Dystrophy. The research, published in Molecular Therapy Methods and Clinical Development, demonstrates that their unique microdystrophin construct containing the C-terminal (CT) domain shows superior performance compared to versions without it.

The studies in mdx mice revealed that the CT domain-containing construct achieved higher protein levels, increased muscle force, and improved resistance to damage. RGX-202 is currently the only microdystrophin gene therapy candidate that includes the CT domain, making it closest to natural dystrophin. These findings support positive interim results from the ongoing Phase I/II AFFINITY DUCHENNE® trial, with REGENXBIO planning a BLA submission in mid-2026 using the accelerated approval pathway.