REGENXBIO (RGNX) Q3 2025 Earnings Call Transcript

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• ALTITUDE Phase II trial showed greater than 70% reduction in vision-threatening events and meaningful DRSS improvement in NPDR patients

  80% confidence

• Priority Review Voucher market price could extend cash runway to early 2028

  80% confidence

• The confirmatory trial for RGX-202 is open and continues to enroll patients

  80% confidence

• RGX-202 is the only microdystrophin that includes CT domain

  80% confidence

• FDA manufacturing and clinical site inspections resulted in no observations, which is a rare achievement

  80% confidence

• REGENXBIO has more than enough supply of RGX-202 ready and available for the entire confirmatory study

  80% confidence

• Manufacturing facility can produce 2,500 doses of RGX-202 per year

  80% confidence

• All 4 RGX-202 patients at 1 year after dosing exceeded expected functional outcomes

  80% confidence

• Vast majority of prevalent DMD population will remain available in 2027

  80% confidence

• RGX-202 shows clear response across the dose range

  80% confidence

• ATMOSPHERE and ASCENT trials represent the largest gene therapy program ever conducted with over 1,200 patients at 200 sites globally

  80% confidence

• RGX-202 has greater than 80% full capsids representing field-leading purity

  80% confidence

• Cash balance as of September 30, 2025 will fund operations into early 2027, excluding potential non-dilutive financings that could extend runway past 2027

  80% confidence

• Half of approximately 1,000 retina specialist respondents say gene therapy is what they are most interested in

  80% confidence

• Sura-vec demonstrated 4-year durability in wet AMD treatment

  80% confidence

• Non-dilutive opportunities could extend cash runway well beyond 2027

  80% confidence

• Competitor ELEVIDYS has approximately 40% liver injury rate versus virtually nonexistent in RGX-202

  80% confidence

• Process Performance Qualification (PPQ) completion is imminent for RGX-202 manufacturing

  80% confidence

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