Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (November 2021)
57 slides from Opus Genetics's investor presentation, Nov 15, 2021
Slide 1: Restore Vision & Clarity: Ocuphire Corporate Presentation
Restore Vision & Clarity Ocuphire Corporate Presentation November 15, 2021 Mina Sooch CEO Exhibit 99.2
Slide 2: Disclosures and Forward Looking Statements
2 Disclosures and Forward Looking Statements 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 Ocuphire Pharma, Inc.’s (“Ocuphire” or the “Company”
Slide 3: Ocuphire Opportunity: A Late-Stage Clinical Ophthalmic Biotech
3 Late Clinical Stage Company Targeting Large, Unmet Ophthalmic Markets Significant Clinical Data andRegulatory Precedents Significant IP Portfolio and Small Molecule CMC Advantages Multiple Near-Term Data Catalysts with Capital Efficient Plan Nyxol eye drops target multiple chronic and acute front
Slide 4: Ocuphire Management Team
4 Ocuphire Management TeamDecades of Biotech and Drug Development Experience Mina Sooch, MBA President & CEO and Founder Drey Coleman VP, Clinical Operations Amy Rabourn, CPA VP, Finance Charlie Hoffmann, MBA VP Corporate Developmentand Operations Mitch Brigell, PhDHead, Clinical Developmentand Stra
Slide 5: Large Unmet Opportunities for the Aging Eye
5 Large Unmet Opportunities for the Aging EyeDeveloping Drugs to Treat Front & Back of the Eye Diseases Source: GlobalData Market Research Report, 2020; Company Estimates for Market Size Front Back Nyxol® APX3330 Reversal of Mydriasis US Market~100M pupil dilations per year in U.S. Opportunity$325M-
Slide 6: Product Candidate Development Stage and Anticipated Milestones
6 Product Candidate Indication Development Stage Anticipated Milestones Pre-clinical Phase 1 Phase 2 Phase 3 Ocuphire-Focused Development 0.75% Nyxol®Eye Drop Reversal of Mydriasis (RM) Positive Data Readout Initiated Phase 3 MIRA-2 trial 4Q20; Topline data reported in 1Q21 (n=185)Initiate Phase 3 M
Slide 7: Extensive Development on Both Drug Candidates
7 11Phase 1 & Phase 2 Trials >340Subjects Dosed Exposure in Humans365Days Patents to2034+ Extensive Development on Both Drug CandidatesWell-Controlled Phase 1, 2, and 3 Clinical Programs with MIRA-2 Data Leading the NDA Path NCE Development Pathway Studied in inflammation/hepatitis & cancer patients
Slide 8: Nyxol Phentolamine Mesylate
8 Nyxol® Phentolamine Mesylate Presbyopia RM Reversal of Mydriasis NVD Night Vision Disturbances P
Slide 9: Nyxol History & MOA
9 Nyxol History & MOARationale for Differentiated Product Profile & 505(b)(2) Path Nyxol’s active ingredient, phentolamine mesylate (PM), is currently approved for 2 indicationsPheochromocytoma (60+ years ago, Regitine®) – intravenous injectionReversal of oral anesthesia (10+ years ago, OraVerse®) –
Slide 10: Nyxol Product Candidate Profile
10 Efficacy Data Improving Vision↓ Pupil Size (moderate miotic)↑ Contrast Sensitivity (night)↑ Near Visual Acuity (light/dark)↑ Distance Visual Acuity Safety Data No Systemic EffectsNo Changes in Blood Pressure No Changes in Heart RateTolerated Topical EffectsMild / Transient / Reversible Eye Rednes
Slide 11: Nyxol: Reversal of Mydriasis
11 Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Reversal of Mydriasis Night Vision Disturbances 0.4% +
Slide 12: Reversal of Mydriasis (RM) – Acute Treatment
12 Reversal of Mydriasis (RM) – Acute TreatmentAnnual Exams and Specialty Visits Involve Dilation to Monitor Eye Health At many annual eye exams and specialty visits, pupils are pharmacologically dilated, impairing vision for 6-24 hoursDilated eyes:heightened sensitivity to lightinability to focusre
Slide 13: RM: Single Use Indication Leveraging a Precedent Approval Pathway
13 Before After Reversal of Mydriasis (RM) – Acute TreatmentSingle Use Indication Leveraging a Precedent Approval Pathway Regulatory Precedent with Rev-Eyes (an alpha 1 blocker), approved by the FDA in 1990 but shortly thereafter discontinued (not for safety or efficacy reasons)Clinical Effect to po
Slide 14: RM MIRA-2 Phase 3 Registration Design
RM MIRA-2 Phase 3 Registration Design Completed Randomized, Double-Masked, Placebo-Controlled, Parallel, One-Day Trial MIRA-2 0.75% NyxolMydriatic Agent A, B, or C 12 USsites168target healthy subjects Nyxol drop(s)(2 drops study eye, 1 drop fellow eye) Mydriasis Time -1 HourPlaceboMydriatic Agent A,
Slide 15: Primary Endpoint: Subjects Returning to Baseline PD at 90 Min
15 Primary Endpoint: % of Subjects Study Eye Returning to Baseline PD at 90 Min Source: MIRA-2 Trial, mITT Population (same as Safety Population), *Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd) Nyxol Met the Primary & Secondary Endpoints at 90 Min; Additionally at 60 Min &
Slide 16: Secondary Endpoint: Mean Pupil Diameter by Mydriatic Agent
16 3 4 5 6 7 8 9 6 24 Pupil Diameter of Study Eye (mm) -1 0 0.5 1 1.5 2 3 4Time Post-Treatment with Nyxol/Placebo (Hours) Tropicamide and Paremyd Nyxol (n=38) Placebo (n=36) 3 4 5 6 7 8 9 3 4 6 24 Pupil Diameter of Study Eye (mm) Time Post-Treatment with Nyxol/Placebo (Hours) Phenylephrine Nyxol n=5
Slide 17: Secondary Endpoint: Returning to Baseline PD by Iris Color
17 0.5 1 1.5 2 3 4 6 Placebo n=46 7% 4% 11% 15% 22% 35% 41% Nyxol n=49 2% 25% 43% 47% 71% 69% 88% 0% 20% 40% 60% 80% 100% Percent of Subjects (Study Eye) (%) Time Post-Treatment with Nyxol/Placebo (Hours) Placebo n=46 Nyxol n=49 Secondary Endpoint: % of Subjects Returning to Baseline PD by Iris Colo
Slide 18: Secondary Endpoint: Accommodation and Time Savings
18 Secondary Endpoint: Accommodation And Time Savings Nyxol Demonstrates a Faster Return to Baseline Accommodation and Shorter Dilation Time by 4-5 Hours Source: MIRA-2 CSR table #14.2.3.2.1. PP population is the per protocol population. Percent of Subjects with Unchanged Accommodation from Baseline
Slide 19: Summary of Positive MIRA-2 Phase 3 Results
19 Summary of Positive MIRA-2 Phase 3 Results for Nyxol Eye Drops Met primary endpoint at 90 minutes with high statistical significance with 2 drops of NyxolMet all key secondary endpoints with high statistical significance Efficacy for all 3 mydriatic agents – phenylephrine, tropicamide, and Paremy
Slide 20: Reversal of Mydriasis (RM) Market Opportunity
20 Reversal of Mydriasis (RM) Market OpportunityWith No Commercially Available Treatment, Nyxol May Provide Significant Revenue Potential GlobalData market research reportVision Care Market Grows 2.4 Percent in 12-Months Ending September 2019. Vision Monday, January 20, 2020. 65% PatientsReport mode
Slide 21: Nyxol: Presbyopia
21 Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of Mydriasis 0.4% +
Slide 22: 2021: The Time for Presbyopia Drops
22 2021: The Time for Presbyopia DropsHeadlines From Academia and Industry Articles Thru the Year with an Early First Approval Sources: Academic review articles, journals, and publications “The correction of presbyopia remains ophthalmology’s ‘Holy Grail’…”-OIS 10/29/2021 P
Slide 23: Presbyopia – Chronic Opportunity
23 Presbyopia – Chronic OpportunityAging Population Drives Demand for Alternatives to Reading Glasses & Very Large Market Lens loses ability to change shape when viewing objects up close as we ageDependence on reading glasses for intermittent and prolonged useGrowing need for therapies that improve,
Slide 24: Pupil Modulation Eye Drops May Replace Reading Glasses
24 Presbyopia – Chronic OpportunityPupil Modulation Eye Drops May Replace Reading Glasses “Pin-hole” effect of Nyxol and low dose pilocarpine may improve near vision by enhancing depth of field as validated by other devices/therapiesMore durable combination of two miotics affecting different muscles
Slide 25: Product Profile: Nyxol + Low-Dose Pilocarpine Combo
25 Product Profile: Nyxol ® + Low-Dose Pilocarpine (LDP) Combo Moderate Action on Iris Dilator and Iris Sphincter Muscles for Near Vision Improvement 0.4% 0.75%Nyxol 0.4% LDP Iris Dilator Muscle Inhibition Iris Sphincter Muscle Activation Phentolamine (alpha1/2 antagonist) approved non- ocular injec
Slide 26: Presbyopia VEGA-1 Phase 2 Design
26 Presbyopia VEGA-1 Phase 2 DesignRandomized, Double-Masked, Placebo-Controlled, Multi-Center One-Week Trial Clinical trial NCT#04675151. DCNVA = distance-corrected near visual acuity . BCDVA = best corrected distance visual acuity Endpoints Primary: % of subjects with ≥ 3 lines of improvement in d
Slide 27: Primary Endpoint Significantly Met for Nyxol + LDP
27 79% 90%80%70%60%50%40%30%20%10%0% Percent of Subjects (%) Secondary Endpoint: Percent of Subjects with ≥ 10 Letters DCNVA Improvement from Baseline Binocular (PP Population) 1Time (Hours)Placebo (n=43) Nyxol+LDP (n=43) Primary Endpoint: % of Subjects ≥ 15 Letter Gain in Photopic DCNVA at 1 HourPr
Slide 28: Efficacy Endpoints: DCNVA Gain Across Timepoints
28 Efficacy Endpoints: % of Subjects ≥ 15 Letter DCNVA Gain Across TimepointsNyxol + LDP had Strong Response with ≥ 15 Letter Near Gain from 30 Minutes to 6 Hours Source: VEGA-1 TLR Table 14.2.1.2 Percent of Subjects with Improvement From Baseline in Photopic DCNVA by Time Point (PP Population). 15
Slide 29: Near Gain and Distance Loss Endpoint
29 2nd Endpoint: % of Subjects ≥ 15 Letter Gain In Near & < 5 Letter Loss In Distance Source: VEGA-1 TLR Table 14.2.2.2 Percent of Subjects with >= 15 Letters of Improvement in Photopic DCNVA and < 5 Letters of Loss in Photopic Binocular BCDVA by Time Point (PP Population) Phase 3 Approval Endpoint
Slide 30: Secondary Endpoint: Mean Pupil Diameter Over Time
30 Secondary Endpoint: Mean Pupil Diameter Over Time Source: VEGA-1 TLR Table 14.2.12.1 Observed Values and Change from Baseline in Photopic Pupil Diameter by Time Point (PP Population) 4.3 4.5 4.4 4.3 4.2 3.2 2.4 2.1 2.3 2.5 2.7 2.9 4.8 3.1 3.2 3.2 3.4 3.3 3.3 4.4 4.3 3.0 2.5 2.7 3.1 3.3 1.0 2.0 3.
Slide 31: Secondary Endpoint: Safety Findings
31 Secondary Endpoint: Safety FindingsNyxol + LDP Combination Was Well Tolerated with a Favorable Safety Profile Source: VEGA-1 Study Results (Safety Population, n=150); Only a single subject difference between mITT (n=148) and PP population (n=147) No serious AEs, almost all AEs were mild0% headach
Slide 32: Potential Best in Class Presbyopia Drop
32 Product Attributes* Nyxol+LDP compared to VUITYTM Efficacy (all time-points) ✓+ Safety: Maintain Distance Vision (especially at night) ✓+ Safety: Tolerability (no headaches) ✓+ Durability (at least 6 hours) ✓+ Fast Onset (within 30 mins) ✓+ Convenience (daily drops) ✓ Tunable Pupil Modulation ✓+
Slide 33: Presbyopia Eye Drops Competitive Landscape
33 Presbyopia Eye Drops Competitive Landscape Corporate Websites, Grzybowski, A, Markeviciute A, Zemaitiene R. A Review of Pharmacological Presbyopia Treatment. 2020 Validation of Pupil Modulating Drops Achieving Pin-Hole Effect & Efficacy, Many with Pilocarpine Pupil modulation MOA Combination drug
Slide 34: Nyxol: Night Vision Disturbances
34 Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of Mydriasis 0.4% +
Slide 35: Night Vision Disturbances (NVD) – Chronic Opportunity
35 Moderate-to-Severe NVDs US Patients Night Myopia 10.8M Cortical Cataracts 4.1M Post-LASIK 500k Post-IOL Implant 300k Total ~16M Night Vision Disturbances (NVD) – Chronic OpportunityImperfections in the Eye Affect Night Vision in Millions Peripheral imperfections scatter light when pupils enlarge
Slide 36: NVD: Pupil Modulation as a Solution
36 Night Vision Disturbances (NVD) – Chronic OpportunityPeripheral Optical Imperfections Allowing Pupil Modulation as a Solution Moderate Decrease in Pupil Size for scattered light gets blocked by the irisClinical Effect to potentially improve low contrast night vision as seen in trialsTolerable wit
Slide 37: NVD LYNX-1 Phase 3 Registration Design
37 NVD LYNX-1 Phase 3 Registration DesignOngoing Randomized, Double-Masked, Placebo-Controlled Two-Week Trial LYNX-1 Endpoints Primary: % of subjects with≥ 3 lines of improvement in mesopic low contrast best- corrected distance visual acuity (Day 8)Secondary (Days 8 & 15):Pupil diameterVisual acuity
Slide 38: Nyxol Demonstrated Clinical Effect in NVD
38 -0.07-2% -0.05-0.5% -0.11-1% -0.99-20% -1.00-21% -0.88-19% -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0.2 Day 8 Day 15 Day 16 Pupil Diameter Change from Baseline ANCOVA (mm and %) Placebo (n = 20) 14-day Daily Evening Dosing, Last Dose on Day 14 ORION-1 Phase 2 Trial Nyxol Demonstrated Clinical Effect in
Slide 39: APX3330
39 APX3330 APX3330 DR Diabetic Retinopathy DME Diabetic Macular Edema wAMD Wet Age-Related Macular Degeneration
Slide 40: Large, Unmet Need in Diabetic Eye Diseases
40 Large, Unmet Need in Diabetic Eye Diseases (US) DR ~7.7M Patients DME ~750K Patients Diabetic Retinopathy & Macular EdemaNon-Injectable Alternative Therapies are Needed For Earlier Stages of Disease Diabetic retinopathy (DR) and diabetic macular edema (DME) are a leading cause of vision loss worl
Slide 41: APX3330 History and Ref-1 Inhibition Mechanism
41 APX3330 History and Ref-1 Inhibition MechanismRef-1 Involved in Multiple 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 (Signaling Cascade) TNF-αChemokin
Slide 42: APX3330 Down-Regulates VEGF and Inflammatory Cytokines
42 APX3330 Down-Regulates VEGF Protein and Anti-Inflammatory Cytokines In Vivo and In Vitro Evidence of APX Dual Pathway Mechanism of Action Treatment of APX3330 (10mg/kg, oral gavage) in rats with type 1 diabetes and induced stroke shows a significant decrease of VEGF signaling.Increased VEGF is a
Slide 43: Preclinical Data: Oral APX3330 Blocks Neovascularization
43 Preclinical Data: Oral APX3330 Blocks NeovascularizationLesion Volume Decrease with Oral APX3330 in Murine Laser CNV Model Similar to EYLEA® Data Silva et al. Oral APX3330 treatment reduces L-CNV lesions in preclinical mouse model and confirms Phase 2 DR/DME clinical dose with sufficient distribu
Slide 44: Phase 1 PK Clinical Data: Human Drug Exposure
44 Apexian preclinical dataEisai preclinical dataSilva et al. Oral APX3330 treatment reduces L-CNV lesions in preclinical mouse model and confirms Phase 2 DR/DME clinical dose with sufficient distribution to human retina using PBPK modeling. Presented at the ARVO 2021 Annual Meeting Phase 1 PK Clini
Slide 45: APX3330 Product Candidate Profile for Multiple Retinal Indications
45 APX3330 Product Candidate Profile for Multiple Retinal IndicationsFirst-in-Class Ref-1 Inhibitor with Favorable Human Safety Data for Retinal Indications Expected Efficacy Data Improving Eye Health in Diabetics↓ Inflammation↓ Abnormal AngiogenesisEnhance Compliance & ExposureOral pill may reduce
Slide 46: DR/DME ZETA-1 Phase 2b Design
46 DR/DME ZETA-1 Phase 2b DesignOngoing, Randomized, Double-Masked, Placebo-Controlled 24-Week Trial (Similar to Eylea Pivotal P3 DR Trial) NPDR = non-proliferative diabetic retinopathy (which includes non centrally involved diabetic macular edema) PDR = proliferative diabetic retinopathy (which inc
Slide 47: Innovative Approach for Retinal Diseases with APX Platform
47 Innovative Approach for Retinal Diseases with APX PlatformAPX3330 May Treat Patients Across the Spectrum of Retinal Diseases Potential First Oral Rx for Retina DiseasesFirst-line earlier intervention for the diabetic eyeAdd-on therapy to current anti-VEGF treatmentsProven Novel MechanismMay decre
Slide 48: Boards and Milestones
48 PHARMA Boards and Milestones
Slide 49: Ocuphire's World-Class Medical Advisory Board
49 Ed Holland, MDLoyola University Chicago Jay Pepose, MD, PhD UCLA Thomas Samuelson, MD University of Minnesota Paul Karpecki, OD Indiana University Eliot Lazar, MDGeorgetown University Marguerite McDonald, MD Columbia University David Boyer, MD Chicago Medical School Gerald Horn, MD University of
Slide 50: Ocuphire Board of Directors
50 Ocuphire Board of DirectorsSeasoned Directors with Decades of Drug Development, M&A/Financings, and Ophthalmology Sean Ainsworth, MBALead Independent Director,Board Director James Manuso, PhD/MBABoard Director Jay Pepose, MD, PhDBoard Director Richard Rodgers, MBABoard Director Susan Benton, MBAB
Slide 51: 2021 to 2022 Ocuphire Cadence of Milestones
51 2021 to 2022 Ocuphire Cadence of MilestonesMultiple Data Catalysts On Path To NDA(s) Ongoing Partnering Discussions with Leading Ophthalmic Companies (including European and Asian Players) 2021 2022 Report Phase 3 Data for NVD Report 2nd Phase 3 Data for RM Report Pediatric Data in RM Submit Nyxo
Slide 52: Recent FDA Ophthalmology Drug Approvals
52 Recent FDA Ophthalmology Drug ApprovalsFDA Record Number of Drugs Approved for Front and Back of the Eye in 2021 Source: Company websites, 2020 10K annual reports, Q2 2021 quarterly reports Company Drug Indication Date Status Cyclosporine Topical Opthalmic Emulision Severe Vernal Keratoconjunctiv
Slide 53: OCUP – Market Snapshot
53 OCUP – Market SnapshotSufficient Cash Runway Through 2022 Source: FactSet Ticker OCUP Price $4.02 Close on 11-1-21 Market Cap ~$70 M As of 11-1-21 Common Shares Outstanding 17.3 M As of 9-30-21 Cash $22.2 M As of 9-30-21 Cash Runway Sufficient through 2022 Guidance as of 9-30-21 Average Daily Vol
Slide 54: Restore Vision & Clarity
Restore Vision & Clarity www.ocuphire.com ir@ocuphire.com
Slide 55: Appendix
55 ©)Ocuphire PHARMA Appendix
Slide 56: NVD Endpoint: Low Contrast Visual Acuity Chart
56 NVD Endpoint: 5% Low Contrast Visual Acuity (LCVA) ChartFDA Accepted Endpoint for Contrast Sensitivity Assessment Precision Vision Before Treatment* 3 Lines Improvement Illustration Primary Endpoint of Nyxol LYNX-1 TrialPercent of subjects with ≥ 3 lines of improvement in mesopic low contrast bes
Slide 57: DR/DME Endpoint: Diabetic Retinopathy Severity Scale
57 DRSSScore 1(10) 2(20) 3(35) 4(43) 5, 6(47, 53) 7 – 13 (60 , 61, 65, 71, 75,85,90) Description DR Absent Micro- aneurysm only Mild NPDR Moderate NPDR Moderately Severe NPDR PMod DR – Mild, erate, and Severe Retinal Image Healthy blood vessels with no bulges Small bulges in blood vessel walls as we
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