Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (August 2023)
42 slides from Opus Genetics's investor presentation, Aug 1, 2023
Slide 1: Restore Vision & Clarity: Ocuphire Corporate Presentation
Restore Vision & Clarity Ocuphire Corporate Presentation August 2023
Slide 2: Forward-Looking Statements
2 This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the success and timing of planned regulatory filings and approvals, pre-commercial activities,
Slide 3: Corporate Highlights
3 Corporate Highlights Strong Financial Position to Advance APX3330 Late-Stage Clinical Candidates for Retinal Diseases Represent Multi-Billion Dollar Opportunity APX3330: Paradigm Changing, Non-invasive, Safe Oral Tablet for millions of NPDR patients that are currently left untreated Ref-1, a novel
Slide 4: Ocuphire Pipeline
4 Ocuphire Pipeline Product Candidate Indication Pre-clinical Phase 1 Phase 2 Phase 3 Regulatory Approval Upcoming Milestones APX3330 Oral Pill Diabetic Retinopathy (DR) EOP2 Meeting EOP2 Mtg Q4 2023 APX3330 Local Delivery Retina Select retinal drug delivery technology APX2009 and APX2014 Local Deli
Slide 5: Diabetic Retinopathy Market and Unmet Need
5 Diabetic Retinopathy Market and Unmet Need
Slide 6: Diabetic Eye Disease is Common Cause of Blindness
6 Diabetic Eye Disease is Common Cause of Blindness Diabetes and Diabetic Retinopathy (DR) https://webeye.ophth.uiowa.edu/eyeforum/tutorials/diabetic-retinopathy-med-students/Classification.htm https://www.mayoclinic.org/diseases-conditions/type-1-diabetes/symptoms-causes/syc-20353011 https://www.ma
Slide 7: Diabetic Retinopathy Market Opportunity
7 American Diabetes Association; International Diabetes Federation; Healthline; *Ocuphire internal analysis and assumptions; Das UN. DME, retinopathy and age-related macular degeneration as inflammatory conditions. Arch Med Sci. 2016;12(5):1142-1157. doi:10.5114/aoms.2016.61918 Patient survey adapte
Slide 8: U.S. Diabetic Retinopathy Market and Target Population
8 American Diabetes Association; International Diabetes Federation; Healthline; *Ocuphire internal analysis and assumptions; Spherix Global Insights Patient survey adapted from Lions International Foundation and International Diabetes Foundation-Europe; Meltzer 2000 Estimates are provided by the Nat
Slide 9: NPDR Patients Rarely Treated; Early Intervention Unmet Need
9 Spherix Global Insights: DR Market DYNAMIX October 2022 Early treatment diabetic retinopathy study research group. ophthalmology. 1991;98(5 suppl):823-33. Diabetes control and complications trial research group. N Engl J Med. 1993;329(14):997-86. Fathy C, Patel S, Sternberg P Jr, Kohanim S. Dispar
Slide 10: ASRS Survey: Wait and Monitor Approach for DR
10 10% 20% 30% 40% 50% 60% 70% International US 59.5% 60.5% 17.0% 24.9% 7.7% 7.8% 4.9% 3.1% 10.9% 3.7% ASRS 2021 Preferences and Trends (PAT) Survey ASRS PAT Survey: Majority of Physicians Use a “Wait and Monitor” Approach for DR NPDR Patients Are Not Treated Proactively and Anti-VEGF Use is Limited
Slide 11: Diabetic Retinopathy Treatment Landscape
11 Diabetic Retinopathy Treatment Landscape
Slide 12: Landscape of Non-Invasive Therapies for Diabetic Retinopathy
12 Landscape of Non-Invasive Therapies for Diabetic Retinopathy Ocuphire’s APX3330 is the Most Advanced and The Only Dual Mechanism Oral Drug Candidate Company websites and www.clinicaltrials.gov (as of July 31, 2023) ✓ Completed ◌ Ongoing ✕ Discontinued Company Drug Target/MOA Indication Route of A
Slide 13: Landscape of Invasive Therapies for Diabetic Retinopathy
13 Landscape of Invasive Therapies (IVT/Suprachoroidal) for Diabetic Retinopathy Eylea®/Lucentis® Approved, But Not Used in Patients with NPDR; Rarely Used in Mild PDR Company Drug Target/MOA Route of Administration Phase 1 Phase 2 Phase 3 Commercial Eylea® (aflibercept) VEGF-A/B; PIGF Intravitreal
Slide 14: APX3330 Background
14 HARMA APX3330 Background
Slide 15: APX3330 History and Ref-1 Inhibition Mechanism
15 Logsdon et al (2018), Li et al (2014). APX3330 History and Ref-1 Inhibition Mechanism Ref-1 Involved in Key Pathways that Contribute to Diabetic Retinopathy and DME Mechanism of Action – Ref-1 Inhibition Hypoxia Ref-1 HIF-1α VEGF (Signaling Cascade) Inflammation Ref-1 NF-κB Other Growth Factors (
Slide 16: APX3330: Drug Development History and Patents
16 APX3330: Drug Development History and Patents Significant Preclinical & Clinical Data Supporting Human Safety, MOA, and PK 12* Phase 1 & Phase 2 Trials Exposure in Humans >10,000 Subject Days at 600mg/day Patents to 2034+ Studied in inflammation/hepatitis & cancer patients (Studied by Eisai & Ape
Slide 17: In-vitro Validation of Mechanism of Action
17 Tao Yan et al. APX3330 Promotes Neurorestorative effects after stroke in type one diabetic rats. Aging and Disease. Vol 9, Oct 2018 Apurinic/Apyrimidinic endonuclease 1 regulates inflammatory response in macrophages. Jedinak A, Dudhgaonkar S, Kelley MR, Sliva D. Anticancer Res. 2011 Feb;31(2):379
Slide 18: APX3330 VEGF Effects in Normal Cells
18 APX3330 VEGF Effects in Normal Cells Abnormal Conditions (e.g., hypoxic): Increased level of VEGF activity Normal Conditions: Physiological level of VEGF activity Kamba 2007; Girardi 2010; Li 2014 APX3330 Investigator Brochure Biologic anti-VEGF agents inactivate VEGF directly and reduce VEGF lev
Slide 19: APX3330 ZETA-1 Clinical Trial
19 APX3330 ZETA-1 Clinical Trial
Slide 20: ZETA-1 Phase 2 Trial Design
20 ZETA-1: Phase 2 Trial of Oral AP3330 in Subjects With Diabetic Retinopathy Multi-center, Randomized, Double-Masked, Placebo-Controlled 24-Week Trial Primary: % subjects with ≥ 2 step improvement on DRSS (Diabetic Retinopathy Severity Scale1) at week 24 Secondary: DRSS improvement ≥1, ≥2, ≥3, ≥4 s
Slide 21: ZETA-1: Baseline Demographics and Characteristics
21 ZETA-1: Baseline Demographics and Systemic Characteristics Well-Balanced Across Arms ZETA-1 Clinical Trial APX3330 n=51 Placebo n=52 Age (years) mean (range) 54.3 58.3 (26-81) (24-78) Sex: Male n (%) 24 (47%) 26 (50%) Race: White n (%) 40 (78%) 41 (79%) Ethnicity: Hispanic or Latino n (%) 28 (55%
Slide 22: Percent of Subjects With ≥2-Step Improvement in DRSS
22 Percent of Subjects With ≥ 2-Step Improvement in DRSS From Baseline ZETA-1 Did Not Meet the Week 24 Phase 2 Primary Endpoint (based on Anti-VEGF Precedence for DR) 30% 25% 20% 15% 10% 5% 0% 35% Percent of Subjects (%) (N=49) (N=46) p=0.98 p=0.27 4% 7% 8% n=41 n=39 0% n=48 n=47 Week 12 Visit Week
Slide 23: Clinically Meaningful Registration Endpoints in DR
23 Clinically Meaningful Registration Endpoints in DR Systemic Drugs Should Evaluate DRSS Change in Both Eyes; To Be Formally Confirmed at EOP2 FDA Meeting 10 81-85 71-75 Local Drugs (Intravitreal Injections) Systemic Drugs Precedent approvable endpoint for locally- delivered drugs (Non-Systemic) in
Slide 24: Binocular Improvement or Worsening in DRSS at Week 24
24 8% 16% 18% 32% 20% 6% 0% 0% 11% 36% 36% 34% 34% 28% 17% 11% 9% 0% 5% 10% 15% 20% 25% 30% 35% ≥ 4 Steps Worsening ≥ 3 Steps Worsening ≥ 2 Steps Worsening ≥ 1 Steps Worsening No Change ≥ 1 Step ≥ 2 Step ≥ 3 Step ≥ 4 Step Improvement Improvement Improvement Improvement Percent of Subjects (%) Placeb
Slide 25: Change in DRSS Score by Patient: APX3330
25 Change in DRSS Score by Patient by Eyes 0% Patients in APX3330 Treatment Group had Binocular 3-Step Worsening DRSS Bar Legend Green: Both Eyes Improving Blue: 1 Eye Improving and 1 Eye Unchanged Purple: 1 Eye Improving and 1 Eye Worsening Orange: no change OU Gray: 1 Eye Worsening and 1 Eye Uncha
Slide 26: Change in DRSS Score by Patient: Placebo
26 Change in DRSS Score by Patient by Eyes 16% Patients in Placebo Treatment Group had Binocular 3-Step Worsening Patients (n=50) DRSS Bar Legend Green: Both Eyes Improving Blue: 1 Eye Improving and 1 Eye Unchanged Purple: 1 Eye Improving and 1 Eye Worsening Orange: no change OU Gray: 1 Eye Worsenin
Slide 27: Binocular 3-Step Worsening and Progression to PDR
27 % of Subjects With Binocular ≥ 3-Step Worsening in DRSS and Progression to PDR APX3330 Prevented Progression of Structural Retinal Abnormalities 16% 0% 0% 0% 5% 10% 15% 20% Percent of Subjects (%) Visit Placebo (N=49) APX3330 (N=46) Week 12 n=50 n=47 12% p=0.07 Percent of Subjects With Worsening
Slide 28: ZETA-1: Treatment Emergent Adverse Events
28 APX3330 SAEs: Dyskinesia, TIA, Chest pain Placebo SAEs: Vertigo, Asthenia, Multiple organ dysfunction, Bradycardia, CAD, Cholelithiasis, COVID-19 pneumonia, Cellulitis, Respiratory failure, Skin ulcer, Peripheral embolism AEs → Withdrawal APX3330: Presyncope, Dyspnea; Placebo: DME (both eyes) *Pr
Slide 29: APX3330 Phase 2 Summary and Next Steps
29 APX3330 - Phase 2 Summary and Next Steps APX3330 demonstrated favorable safety and tolerability with compelling potential to slow progression of diabetic retinopathy ZETA-1 statistically significant results on potential Phase 3 registration endpoint: 0% APX3330-treated patients had a binocular ≥
Slide 30: APX3330 Summary
DR is one of the largest markets in retina with 10M patients in US and over 100M worldwide Majority of the NPDR patients are not candidates for approved biologics treatments and are left untreated APX3330 first-in-class oral drug that inhibits Ref-1 which reduces VEGF and inflammatory cytokines to n
Slide 31: Pharma
31 ©)Ocuphire PHARMA
Slide 32: Global Partnership with Viatris for Nyxol
32 Global Partnership with Viatris for Nyxol Viatris Has Selected Nyxol to be a Key Element of its Global Eye Care Division Fully funded development and commercialization costs for all 3 Nyxol indications Partner for Nyxol global commercialization Allows Ocuphire to focus on APX3330 development Stre
Slide 33: Nyxol Eye Drops: Three Indications
33 Reversal of Mydriasis (RM) P DLD Dim Light or Night Vision Disturbances (DLD) Presbyopia Nyxol as a Single Drop Nyxol with LDP Adjunctive Therapy 1 0.4% 2 RM R M NYXOL® EYE DROPS THREE INDICATIONS NEW PARTNERSHIP WITH VIATRIS
Slide 34: Summary of Nyxol Trial Results
34 Summary of Nyxol Trial Results Comprehensive Body of Clinical Data Supporting Efficacy and Safety Across 3 Indications Indication & Status Primary Endpoint Efficacy Data Key Secondary Endpoint(s) Safety & Tolerability RM PDUFA 9/28/23 Return to baseline pupil diameter at 90 minutes after dilation
Slide 35: Corporate Highlights
35 Corporate Highlights Late-Stage Retinal Pipeline Represents Multi-Billion Dollar Opportunity in Unmet NPDR Patients APX Pipeline Driven by a Paradigm Changing, Dual Target Ref-1 Platform for Retinal Diseases Global License Agreement with Viatris to Fund Development and Commercialization of Nyxol
Slide 36: Restore Vision & Clarity
Restore Vision & Clarity www.ocuphire.com ir@ocuphire.com Ocuphire Pharma Appendix 36
Slide 37: Preclinical Data: Oral APX3330 Blocks Neovascularization
37 Silva et al. ARVO 2021 Annual Meeting *Published data on EYLEA. This study was performed independently from APX3330 study and is a cross-study comparison. **Li 2014; *** Pasha 2018; ****Jiang 2011 (Vldlr -/- : Very Low-Density Lipoprotein receptor knock-out mice) Preclinical Data: Oral APX3330 Bl
Slide 38: 505(b)(2) Regulatory Pathway and Nyxol Mechanism
38 505(b)(2) Regulatory Pathway Supported by Prior Phentolamine Approvals in non-ophthalmic Indications Illustration for educational purposes Nyxol’s Differentiated MOA as an Alpha-1 Blocker No Engagement of Ciliary Muscle, No Headaches and Lower Risk of Retinal Detachment Phentolamine is the Active
Slide 39: Presbyopia Competitive Landscape and Nyxol Differentiation
39 Phase 2 Phase 1 Ocuphire Nyxol + 0.4% pilo Visus Brimochol® (carbachol + brim) Other Cholinergic Agonists* Cholinergic Agonist* (pilocarpine) Lenz Aceclidine; Aceclidine + brim Eyenovia MicroLine (2% pilo) Alpha Antagonist & low dose pilocarpine* Alpha Antagonist NDA Allergan VUITYTM; (1.25% pilo
Slide 40: Management Team with Decades of Drug Development Experience
40 Management Team with Decades of Drug Development Experience Drey Coleman VP, Clinical Operations Amy Rabourn, CPA SVP, Finance Charlie Hoffmann, MBA SVP, Corporate Development Mitch Brigell, PhD Head, Clinical Development and Strategy Daniela Oniciu, PhD Global Head, R&D, Chemistry and Product De
Slide 41: Ocuphire's Medical Advisory Board
41 Ocuphire's World-Class Medical Advisory Board Chief Medical Advisor, Ocuphire Refractive Specialist Jay Pepose, MD, PhD UCLA School of Medicine Refractive Specialist Y. Ralph Chu, MD Northwestern University Refractive Specialist Zaina Al-Mohtaseb, MD Baylor College of Medicine Refractive Speciali
Slide 42: Ocuphire Board of Directors
42 Ocuphire Board of Directors Sean Ainsworth, MBA Lead Independent Director, Board Director Jay Pepose, MD, PhD Board Director Susan Benton, MBA Board Director Cam Gallagher, MBA Chair, Board Director James Manuso, PhD/MBA Board Director Richard Rodgers, MBA Interim President & CEO Board Director
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