Why Regenxbio (RGNX) Shares Are Falling Today - Regenxbio (NASDAQ:RGNX)

Regenxbio Inc 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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Regenxbio Says Diabetic Retinopathy Gene Therapy Shows Meaningful Improvements In Disease Severity