REGENXBIO initiates pivotal phase of RGX-202 Duchenne trial with positive functional data; signs $150M ATM facility

REGENXBIO Inc.

First five patients in Phase I/II showed stable/improved function on NSAA and timed tests, exceeding external natural history controls.
FDA alignment achieved on accelerated approval pathway; BLA submission expected in 2026.
Biomarker data: RGX-202 microdystrophin expression highest reported; no serious adverse events.
New ATM sales agreement with Leerink Partners for up to $150M common stock, replacing prior BofA facility.
Pivotal trial enrolling ambulatory patients aged 1+, first patient dosed; includes younger boys aged 1-3.

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