Regenxbio Says Diabetic Retinopathy Gene Therapy Shows Meaningful Improvements In Disease Severity
- Regenxbio Inc (NASDAQ: RGNX) announced additional interim data from the ongoing Phase 2 ALTITUDE trial of RGX-314 for diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME).
- At six months, patients treated with RGX-314 demonstrated clinically meaningful improvements in disease severity versus observation control as measured by Diabetic Retinopathy Severity Scale (DRSS).
- 20% of patients achieved ≥2-step DRSS improvement vs. 10% in control.
- 54% of patients achieved any DRSS improvement vs. 20% in control.
- 0% of patients worsened ≥2 steps vs. 20% in control.
- Related: Despite Solid Efficacy From Gene Therapy, This Analyst Cuts REGENXBIO's Price Target.
- Regenxbio announced that the ALTITUDE trial has been expanded to include a higher third dose level of 1x1012 GC/eye.
- The trial is enrolling two new cohorts (Cohorts 4 and 5) at the third dose level.
- RGX-314 continues to be well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious adverse events.
- The company reported $617 million in cash, cash equivalents, and marketable securities as of September 30, with an operational runway into 2025.
- Price Action: RGNX shares are down 16.10% at $20.33 on the last check Thursday.
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