REGENXBIO Q3 Earnings Call Highlights

Key Points

• Wet AMD results are imminent: REGENXBIO and AbbVie expect this quarter’s top-line results from two pivotal sura-vec studies enrolling more than 1,300 patients. If the program advances, global regulatory submissions are expected in 2027.
• RGX-202 filing is targeted for Q1 2027: The company plans to submit a complete application for its Duchenne therapy at once, seeking accelerated approval, with potential U.S. approval in the second half of 2027.
• REGENXBIO reported $251 million in cash and securities at the end of September and expects its cash runway to extend into late Q4 2027; the balance excludes a $39 million Sarepta settlement payment received in October.

REGENXBIO NASDAQ: RGNX is approaching pivotal trial results for its eye disease gene therapy and preparing a U.S. application for its Duchenne muscular dystrophy treatment, executives said during the company’s third-quarter earnings call.

The company and partner AbbVie expect top-line results this quarter from two pivotal studies of sura-vec in wet age-related macular degeneration, or wet AMD. REGENXBIO also plans to submit a biologics license application for Duchenne therapy RGX-202 in the first quarter of 2027, seeking accelerated approval.

Chief Financial Officer Mitch Chan said REGENXBIO ended September with $251 million in cash, cash equivalents and marketable securities and expects its cash runway to extend late into the fourth quarter of 2027.

Wet AMD Results Expected This Quarter

The sura-vec studies, ATMOSPHERE and ASCENT, enrolled more than 1,300 patients across 14 countries, Chief Medical Officer Steve Pakola said. Each trial compares two doses of the gene therapy with an existing anti-VEGF treatment. Their primary endpoint is whether sura-vec is no worse than the control treatment on change in best-corrected visual acuity at 54 weeks.

Pakola said the planned joint announcement with AbbVie will address injection burden and retinal anatomy as well as the primary endpoint. REGENXBIO expects global regulatory submissions in 2027 if the program advances.

President and CEO Curran Simpson said frequent injections can be difficult for wet AMD patients to sustain. The company is developing sura-vec as a one-time treatment and has included both newly diagnosed and treatment-experienced patients in the pivotal studies.

Asked what treatment-burden reduction would be competitive, Pakola said the goal was at least a 50% reduction, with a meaningful proportion of patients needing no injections. He cautioned that comparisons across studies should account for differences in how difficult their patient populations are to treat.

Simpson said REGENXBIO and AbbVie have trained more than 500 retina specialists. He also said long-term durability data would be important in reimbursement discussions, adding that some wet AMD patients have been followed for five years after subretinal administration.

Sura-vec is also being studied in diabetic retinopathy. Pakola said a phase IIb/III study called NAAVIGATE is enrolling patients. In the earlier phase II ALTITUDE trial, 60% of patients at the dose level being evaluated in NAAVIGATE improved by at least two steps on a diabetic retinopathy severity scale at three years without additional treatment, he said.

RGX-202 Filing Strategy Changes

REGENXBIO now plans to submit the full RGX-202 application at once rather than through a rolling filing. Simpson said the company changed its approach so it could discuss the proposed data package with the Food and Drug Administration in a pre-application meeting this year. He said the change would not affect the expected decision timeline.

The company is targeting potential U.S. approval in the second half of 2027. The application is expected to include safety data from more than 60 patients across pivotal and confirmatory studies, Pakola said. The confirmatory study has completed dosing.

Executives highlighted a statistically significant correlation between microdystrophin expression and improvement on the North Star Ambulatory Assessment, a measure of motor function, at one year. Simpson said the company intends to discuss with the FDA whether that evidence supports the use of microdystrophin as a marker reasonably likely to predict clinical benefit under the accelerated approval pathway.

Asked about the small number of patients in the initial correlation analysis, Pakola said the company expects to have 14 patients with functional data for the application and will also assess correlations with timed function tests. Simpson said REGENXBIO expects to publish updated data after filing but has not specified a meeting or event.

REGENXBIO also plans to start an international randomized, placebo-controlled RGX-202 study, AFFINITY RISE, in the first half of 2027. The trial is designed to enroll approximately 100 patients, Pakola said.

Cash and Other Pipeline Updates

The company’s quarter-end cash balance rose from $241 million at the end of 2025. Chan attributed the increase to a $100 million AbbVie milestone payment tied to the first patient dosing in NAAVIGATE and $108 million in net proceeds from a July public offering of shares and pre-funded warrants.

The September balance excludes a $39 million payment received in October under a settlement agreement with Sarepta Therapeutics. Chan said the cash-runway forecast does not include potential proceeds from other non-dilutive funding sources.

For RGX-121, Pakola said REGENXBIO continues to collect and evaluate additional patient imaging and long-term follow-up data while working with the FDA and partner NS Pharma. The company will provide an update on the program’s path forward as it progresses, he said.

About REGENXBIO (NASDAQ:RGNX)

REGENXBIO Inc NASDAQ: RGNX is a clinical-stage biotechnology company focused on developing gene therapies for serious diseases. The company's programs use adeno-associated virus (AAV) vectors to deliver genetic material to targeted cells, with the goal of addressing the underlying causes of disease rather than primarily treating symptoms.

REGENXBIO's technology is built around its NAV AAV Technology Platform, which supports the design and development of gene-delivery vectors. Its pipeline has included investigational therapies for conditions such as Duchenne muscular dystrophy, Hunter syndrome, age-related macular degeneration and other rare diseases.

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