Regenxbio: DMD GTx expression data are positive, but we think regulatory pathway is key focus for investors to give more credit

Research evidence excerpt

Regenxbio: DMD GTx expression data are positive, but we think regulatory pathway is key focus for investors to give more credit

Equity Research

U.S. Small & Mid Cap Biotechnology

14 May 2026

Regenxbio

DMD GTx expression data are

positive, but we think regulatory

RGNX OVERWEIGHTpathway is key focus for investors

Unchanged

U.S. Small & Mid Cap POSITIVEto give more credit Biotechnology

We think further updates on FDA interactions and clarity on Price Target USD 12.00

the regulatory pathway in DMD will be needed for investors to lowered -68% from USD 37.00

get more confident on RGX-202. For '314 (ABBV-partnered), Price (13-May-26) USD 10.04 Potential Upside/Downside +19.5%

Source: Bloomberg, Barclays Researchwe note Ph3 data in wAMD (subretinal) expected 4Q could be

a key catalyst.

Market Cap (USD mn) 518

Shares Outstanding (mn) 51.62

We think expression & safety data look encouraging, further clarity on path to fileability/ Free Float (%) 90.78

approvability could drive meaningful upside for shares at these levels. RGNX reported 1Q 52 Wk Avg Daily Volume (mn) 0.8

EPS and topline data from the pivotal Ph3 AFFINITY study (not placebo-controlled) of RGX-202 Dividend Yield (%) N/A

for DMD; the company also hosted a call and we caught up with management after. The Ph3 met Return on Equity TTM (%) -107.00

its primary endpoint, with 93% of patients reaching >10% expression and 80% reaching >40% Current BVPS (USD) 2.02

expression (n=30); on safety we note one case of asymptomatic liver injury was reported (n=31 Source: Bloomberg

in safety cohort). In patients with functional data at one year, RGNX reported stat-sig correlation

between expression and NSAA (n=9, correlation 0.94, p=0.0002). We note RGNX sees the FDA Price Performance Exchange-Nasdaq