Ocuphire Pharma Results Presentation Deck
16
Clinically Meaningful Registration Endpoints in DR
Path Forward to Phase 3: Systemic Drugs Should Evaluate DRSS Change in Both Eyes
In retina, opportunity for
approval to show
improvement OR worsening
(prevention of progression)*
Precedent approvable endpoint
for locally delivered drugs
(non-systemic) in DR:
≥ 2-step DRSS
improvement in study eye
Eylea (Panorama trial)
Lucentis (Rise/Ride trials)
●
●
Oral/systemic drugs are different than anti-VEFG IVT as they treat
both eyes
Therefore, a suitable evaluation is change in both eyes (binocular)
Potential approvable endpoints for systemic drug in DR (to be
confirmed at the EOP2 FDA meeting) include:
●
≥ 3-step binocular DRSS improvement
> 3-step binocular DRSS worsening
ZETA-1 Phase 2 trial for APX3330 evaluated key secondary
endpoints ≥ 3-step binocular DRSS improvement and
worsening to inform design of the Phase 3 registration trial
*Nair P, Aiello LP, Gardner TW, Jampol LM, Ferris FL III. Report From the NEI/FDA Diabetic Retinopathy Clinical Trial Design and Endpoints Workshop. Invest Ophthalmol Vis Sci. 2016 Oct
1;57(13):5127-5142. doi: 10.1167/iovs. 16-20356. PMID: 27699406; PMCID: PMC6016432.
Ocuphire
Source: ZETA-1 Clinical trialView entire presentation