Ocuphire Pharma Results Presentation Deck
CO
6
APX3330 History and Ref-1 Inhibition Mechanism
Ref-1 Involved in Multiple Key Pathways that Contribute to Diabetic Retinopathy and DME
Mechanism of Action - Ref-1 Inhibition
Нурохіа
↓
Ref-1
HIF-1a
VEGF
(Signaling
Cascade)
Anti-VEGF
Lucentis®
EYLEAⓇ
APX3330 —
Neovascularization
Logsdon et al (2018), Li et al (2014).
Inflammation
Ref-1
NF-kB
TNF-a
Chemokines
Other Growth
Factors
(Signaling
Cascade)
Steroids
●
• Ref-1 (reduction-oxidation effector factor-1) is a novel
target discovered by Dr. Mark R. Kelley at Indiana
University School of Medicine and Ocuphire's
Scientific Advisor for APX program
●
APX3330 is a small molecule oral drug candidate and
a first-in-class inhibitor of Ref-1
• APX3330 previously developed by Eisai for multiple
hepatic inflammatory indications and later by Apexian
for advanced solid tumors in 11 Phase 1 and 2 trials
Similar oncology origin as approved anti-VEGFs
●
MOA uniquely decreases both abnormal
angiogenesis and inflammation by blocking
pathways downstream of Ref-1
Extensively studied in over 20 in-vitro and animal
studies with favorable efficacy and safety
Ocuphire
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