First Take: AFFINITY Hits Primary Endpoint as Expected

Regenxbio reported positive Phase 3 biomarker data for its DMD gene therapy, RGX-202, showing a 71.1% mean microdystrophin expression. However, limited functional data and a case of subacute myocarditis keep the regulatory path to accelerated approval uncertain, maintaining a Neutral rating.

Key Takeaways

• 1.Regenxbio's Phase 3 AFFINITY study met its primary biomarker endpoint with 93% of patients showing microdystrophin expression, yielding a mean expression level of 71.1%.
• 2.While biomarker data was strong and compared favorably to Sarepta’s Elevydis, investors remain focused on safety concerns (one case of subacute myocarditis) and the uncertainty of FDA's regulatory path for accelerated approval.
• 3.Interim functional data showed positive trends but is currently deemed immature due to the small sample size (n=9), making broader efficacy conclusions difficult.

Table of Contents

• High proportion of patients show microdystophin expression; accelerated approval on limited interim functional data remains an FDA review question.
• Additional details
• Valuation and risks
• Disclosure Appendix
• GS Factor Profile
• M&A Rank
• Quantum
• Disclosures
• Company-specific regulatory disclosures
• Distribution of ratings/investment banking relationships
• Price target and rating history chart(s)
• Target price history table(s)
• Regulatory disclosures
• Ratings, coverage universe and related definitions
• Global product; distributing entities
• General disclosures

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