普通外文研报
RGNX Read Through: RGX-202 Pivotal Data Bodes Well For SLDB's SGT-003 in DMD
研报英文原文证据摘录
RGNX Read Through: RGX-202 Pivotal Data Bodes Well For SLDB's SGT-003 in DMD
Quick Note
May 14, 2026 Solid Biosciences Inc. (SLDB)
Rating: RGNX Read Through: RGX-202 Pivotal Data Bodes Well ForOUTPERFORM SLDB's SGT-003 in DMDPrice:
$7.38
12-Month Price Target: The Wedbush View: Remain Outperform rated on SLDB. RGNX (not covered)
$16.00 reported top-line Phase 3 AFFINITY DUCHENNE data for RGX-202. While overall
microdystrophin expression appeared robust (71% via Western Blot at week 12),
RGNX also reported 2 SAEs (subacute myocarditis, liver injury). We see positive
Analysts read through to SLDB. We view SGT-003 holding an easier immune suppression
Laura Chico, Ph.D. regimen with preliminary microdystrophin data that looks at least on par. Pairing(212) 668-9868
Laura.Chico@wedbush.com this with the IMPACT study already underway, and SLDB remains on "solid footing"
Thomas Yip with SGT-003.
(707) 521-2281
thomas.yip@wedbush.com RGNX rolls out pivotal RGX-202 results. The Phase 3 portion of the AFFINITY
DUCHENNE study evaluated 2e14 gc/kg RGX-202 (RGNX, not covered) in ~30
ambulatory DMD patients aged 1yr and older. The primary endpoint of the study
evaluated the proportion of patients with microdystrophin expression at/above
10% at week 12.
● Efficacy. Mean RGX-202 microdystrophin expression (via Western Blot) reached
71% overall at week 12. Higher expression shown in younger (<4yrs) patients
with 4-7yrs cohort achieving 58% (n=9). SRPT's (Outperform, Zhong) Elevidys
generated an average 34% expression in the same age group.
● Safety. Two SAEs were reported (subacute myocarditis, liver injury) with both
fully resolving. RGNX incorporated an immune suppression regimen including
eculizumab (5 doses), and overlapping sirolimus and steroids.
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