Ocuphire Pharma Results Presentation Deck
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ZETA-1 Trial: Key Takeaways
APX3330 achieved statistical significance on a key pre-specified secondary endpoint of preventing
clinically meaningful progression of diabetic retinopathy (as defined by binocular 3 or more steps
worsening on the DRSS¹) after 24 weeks of treatment
Trend toward more efficacy at 24 weeks vs 12 weeks, suggests that the 52-week Phase 3 trial may
generate a larger signal due to an increase in % of placebo subjects who progress
Prevention of 3-step worsening (binocular) is a suitable endpoint for an oral, systemically drug
➜ Ocuphire plans to go forward with this potential registration endpoint in Phase 3 following
confirmation with the FDA in EOP2 meeting
Oral APX3330 demonstrated favorable safety and tolerability
Retinal KOLs feedback suggest that slowing of DR progression with an oral agent would be a useful
treatment in patients with background DR and good visual function
If approved, APX3330 could be an important new primary preventative therapeutic option that
could be used in a large number of diabetic patients who are earlier in their disease
1. diabetic retinopathy severity score
Source: ZETA-1 Clinical Trial
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