Regenxbio, Inc.: Efficacy for RGX-202 looks solid, safety/regulatory path now questions

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Regenxbio, Inc.: Efficacy for RGX-202 looks solid, safety/regulatory path now questions

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Regenxbio, Inc.

Efficacy for RGX-202 looks solid,

safety/regulatory path now questions

Reiterate Rating: BUY | PO: 14.00 USD | Price: 6.21 USD

Data supportive, regulatory overhang a question 14 May 2026

We view today’s RGX-202 AFFINITY DUCHENNE topline as directionally encouraging Equity

despite some questions about safety and regulatory path. The study met its primary

endpoint with the microdystrophin/functional correlation also compelling; however, the

Key Changesfunctional dataset remains limited at n=9, relies on external controls, and the FDA has

previously recommended an RCT even while leaving the door open to review alternative (US$) Previous Current

proposals. While the readout supports continued development and potential disruptive Price Obj. 25.00 14.00

positioning, the key investor debate remains whether the FDA will accept 2027E Rev (m) 437.5 401.3

microdystrophin expression plus interim external-control functional data as adequate for 2028E Rev (m) 562.7 467.3

accelerated approval. We reiterate Buy and $14 PO (from $25) as we reduce peak 2026E EPS -4.87 -4.91

penetration/PoS for DMD on regulatory uncertainty despite an objectively positive 2027E EPS 0.61 -0.24

topline result from the study (now $5/sh from $14) and update cash (now $3/sh). 2028E EPS 2.26 0.20

Solid microdystrophin expression and functional benefit Alec W. Stranahan

Research Analyst

RGX-202 achieved the primary endpoint, with 93% of evaluable patients above 10% BofAS

microdystrophin expression at week 12 and mean expression of 71.1% across all +1 646 743 2109 alec.stranahan@bofa.com

patients. The 12-month functional data are also encouraging directionally, with n=9

Matthew Guggenbiller