RGX-202 Puts A Little Pressure On Elevidys, But Just A Little

Research evidence excerpt

RGX-202 Puts A Little Pressure On Elevidys, But Just A Little

Quick Note

May 15, 2026 Sarepta Therapeutics (SRPT)

Rating: RGX-202 Puts A Little Pressure On Elevidys, But Just A LittleOUTPERFORM

Price:

$18.68 REGENXBIO (RGNX, Not Covered) reported positive top-line data from the pivotal

12-Month Price Target: Ph3 portion of the Ph1/2/3 study of RGX-202 in DMD, and we believe that

$35.00 the most important finding is the statistically significant correlation between

microdystrophin expression and functional improvement as measured by NSAA

score. We believe that this correlation, by providing strong validation for

Analysts microdystrophin as a surrogate endpoint that predicts clinical benefit, should

Yun Zhong, Ph.D. benefit all microdystrophin gene therapy programs/products. We see no other

(917) 608-0166 big impact on Elevidys, since 1) we do not believe that the relatively smallyun.zhong@wedbush.com

patient number in RGNX's study allows for a fair comparison of microdystrophin

expression, and 2) the regulatory pathway for RGX-202 remains to be defined

although RGNX management plans to convince the FDA that a randomized

controlled trial (RCT) should not be warranted. In fact, KOLs' positive comments on

RGNX's conference call about the comfort level towards the immunosuppression

regimen for RGX-202, which includes not only sirolimus but complement inhibitor

eculizumab, support adoption of SRPT's enhanced regimen to address the acute

liver injury (ALI) side effect in non-ambulatory patients. Therefore, we remain

optimistic in Elevidys' improved market uptake in 2H26 and beyond.

Elevidys Treatment Had Led to High Microdystrophin Production in Younger

Patients as Well. The microdystrophin expression levels that RGNX reported appear