REGENXBIO targets wet AMD data, 2027 filings and cash runway

By Sophie Lindqvist

$251 million in cash, cash equivalents and marketable securities at September’s end compared with $241 million at the end of 2025. At the Q3 earnings call, CFO Mitch Chan attributed the increase to a $100 million AbbVie milestone after first-patient dosing in NAAVIGATE and $108 million in net July public-offering proceeds from shares and pre-funded warrants.

He expects the cash runway to extend into late Q4 2027. The September balance excludes $39 million received from Sarepta Therapeutics in October under a settlement agreement, while the forecast excludes proceeds from other potential non-dilutive funding sources.

What does the wet AMD readout require?

More than 1,300 patients across 14 countries enrolled in ATMOSPHERE and ASCENT, two pivotal studies of sura-vec in wet age-related macular degeneration (wet AMD), Pakola said. REGENXBIO and AbbVie expect top-line results this quarter.

Each trial compares two sura-vec doses with an existing anti-VEGF (vascular endothelial growth factor inhibitor) treatment. The primary endpoint asks whether sura-vec is no worse than the control on change in best-corrected visual acuity (a measure of eyesight) at 54 weeks.

The planned joint announcement will also address injection burden and retinal anatomy, not merely the primary endpoint. If the program advances, REGENXBIO expects global regulatory submissions in 2027.

President and CEO Curran Simpson said: “Frequent injections can be difficult for wet AMD patients to sustain.” He said sura-vec is being developed as a one-time treatment, and the pivotal studies include both newly diagnosed and treatment-experienced patients.

Asked what treatment-burden reduction would be competitive, Chief Medical Officer Steve Pakola said: “The goal was at least a 50% reduction, with a meaningful proportion of patients needing no injections.” He cautioned that cross-study comparisons require adjustment for differences in how difficult patient populations are to treat.

Simpson said REGENXBIO and AbbVie have trained more than 500 retina specialists. He added that long-term durability will matter in reimbursement discussions, and some wet AMD patients have reached five years of follow-up after subretinal administration (delivery beneath the retina).

What can NAAVIGATE establish?

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

What changes in the RGX-202 filing?

Simpson said REGENXBIO now plans to submit RGX-202’s full biologics license application (BLA) at once in Q1 2027, rather than through a rolling filing, in which sections are submitted in stages. The company seeks accelerated approval and targets a potential U.S. approval in the second half of 2027.

He said the switch will let REGENXBIO discuss the proposed data package with the Food and Drug Administration at a pre-application meeting this year. He said the change will not affect the expected decision timeline.

Pakola said the application should include safety data from more than 60 patients across pivotal and confirmatory studies. The confirmatory study has completed dosing.

At one year, executives highlighted a statistically significant correlation between microdystrophin expression and improvement on the North Star Ambulatory Assessment, a measure of motor function. Simpson said REGENXBIO intends to ask the FDA whether that evidence supports microdystrophin as a marker reasonably likely to predict clinical benefit under accelerated approval.

Pakola said the initial correlation analysis involved a small number of patients, while the company expects functional data from 14 for the application. It will also assess correlations with timed function tests. Simpson said updated data should follow the filing, although no meeting or event has been specified.

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

What remains unresolved?

Pakola said REGENXBIO continues collecting and evaluating additional patient imaging and long-term follow-up data. The company is working with the FDA and partner NS Pharma and will update the program’s path forward as it advances.

REGENXBIO (NASDAQ: RGNX) describes itself as a clinical-stage biotechnology company developing gene therapies for serious diseases. Its programs use adeno-associated virus (AAV) vectors to deliver genetic material to targeted cells, with the goal of addressing underlying causes rather than primarily symptoms.

Its NAV AAV Technology Platform supports the design and development of gene-delivery vectors. The pipeline has included investigational therapies for Duchenne muscular dystrophy, Hunter syndrome, age-related macular degeneration and other rare diseases.

The milestone schedule is clear. The clinical outcomes remain pending.