Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Rexahn Pharmaceuticals — Investor Presentation (November 2020)
37 slides from Opus Genetics's investor presentation, Nov 6, 2020
Slide 1: Ocuphire Corporate Presentation November 2020
Ocuphire Corporate Presentation November 2020
Slide 2: Disclosures and Forward Looking Statements
Disclosures and 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 Ocuphire Pharma, Inc.’s (“Ocuphire” or the “Company”
Slide 3: Ocuphire - New Public Ophthalmic Drug Development Company
Ocuphire - New Public Ophthalmic Drug Development CompanyNasdaq Symbol: OCUP Nasdaq public listing allows for capital access, investor liquidity, and strategic visibilityReverse merger with Rexahn and financing transaction announced in June, closed in NovemberOcuphire management team, expanded board
Slide 4: Ocuphire Management Team
Ocuphire Management TeamDecades of Biotech and Drug Development Experience Mina Sooch, MBA Chief Executive Officer 25 years in pharma/biotech industry as a CEO, entrepreneur, venture capitalist and strategist. Raised over 100 million dollars for 2 biotechs, then led private to public on Nasdaq, Reco
Slide 5: Late Clinical Stage Company Targeting Large, Unmet Ophthalmic Markets
Late Clinical Stage Company Targeting Large, Unmet Ophthalmic Markets Significant Clinical Data Significant IP Portfolio and Small Molecule CMC Advantages Multiple Near-Term Data Catalysts with Capital Efficient Plan Nyxol targets multiple chronic and acute front of the eye indications addressing la
Slide 6: Large Unmet Opportunities for the Aging Eye
6 Large Unmet Opportunities for the Aging EyeDeveloping Drugs to Treat Front & Back of the Eye Diseases Source: GlobalData Market Research Report, 2020 Night Vision DisturbancesU.S. Prevalence: ~16M adultsReversal of Mydriasis~100M pupil dilations per year in U.S.PresbyopiaU.S. Prevalence: ~120M $4-
Slide 7: Product Candidate Development Stage and Anticipated Milestones
Product Candidate Indication Development Stage Anticipated Milestones Pre-clinical Phase 1 Phase 2 Phase 3 Ocuphire-Focused Development 0.75% Nyxol®Eye Drop Dim Light or Night Vision Disturbances (NVD) Initiate Phase 3 LYNX-1 trial 4Q2020; Data expected in 3Q21 (n=160) 0.75% Nyxol®Eye Drop Reversal
Slide 8: Extensive Development on Both Drug Candidates
11 >340Phase 1 & Phase Subjects Dosed 2 Trials Exposure in Humans365Days Patents to2034+ Extensive Development on Both Drug CandidatesWell-Controlled Phase 1 & Phase 2 Clinical Programs Set Stage for NDA Path NCE Development Pathway Studied in inflammation/hepatitis & cancer patients 7Phase 1 & Phas
Slide 9: Nyxol Phentolamine Mesylate
Nyxol® Phentolamine Mesylate NVD Night Vision Disturbances P Presbyopia RM Reversal of Mydriasis 9
Slide 10: Nyxol History & MOA
Nyxol History & MOARationale for Differentiated Product Profile & 505(b)(2) Path Nyxol’s active ingredient, phentolamine mesylate (PM), iscurrently approved for 2 indicationsPheochromocytoma (60+ years ago, Regitine®) – intravenous injectionReversal of oral anesthesia (10+ years ago, OraVerse®) – in
Slide 11: Nyxol Product Candidate Profile
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 RednessIO
Slide 12: Night Vision Disturbances (NVD) – Chronic Opportunity
12 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 13: NVD – Pupil Modulation as a Solution
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 with a
Slide 14: NVD LYNX-1 Phase 3 Registration Design
NVD LYNX-1 Phase 3 Registration DesignPlanned Randomized, Double-Masked, Placebo-Controlled Trial LYNX-1 Endpoints Primary: % of subjects with≥ 3 lines of improvement inmesopic low contrast best- corrected distance visual acuity (Day 8)Secondary (Days 8 & 15):Pupil diameterVisual acuity measures (di
Slide 15: ORION-1 Phase 2 Trial: Nyxol Clinical Effect in NVD
15 ORION-1 Phase 2 Trial Nyxol Demonstrated Clinical Effect in NVDKey Endpoints Observed in Multiple Phase 2 Trials Source: NYXG-201 % of Subject Eyes NYX-SNV Phase 2 Trial Improved Low Contrast Distance Visual Acuity*% of Eyes with Mesopic Low Contrast Visual Acuity Improvementp=0.029p=0.04p=0.16So
Slide 16: Reversal of Mydriasis (RM) – Acute Treatment
16 Reversal of Mydriasis (RM) – Acute TreatmentAnnual Exams and Specialty Visits Involve Dilation to Monitor Eye Health At every annual eye exam and many specialty visits, patients’ pupils are pharmacologically dilated, impairing vision for 6-24 hoursDilated eyes:heightened sensitivity to lightinabi
Slide 17: RM – Single Use Indication Leveraging Precedent Approval Pathway
17 Reversal of Mydriasis (RM) – Acute TreatmentSingle Use Indication Leveraging a Precedent Approval Pathway Regulatory Precedent with RevEyes (an alpha 1 blocker), approved by the FDA in 1990 but shortly thereafter discontinued (not for safety or efficacy reasons)Clinical Effect to potentially redu
Slide 18: RM MIRA-2 Phase 3 Registration Design
18 RM MIRA-2 Phase 3 Registration DesignPlanned Randomized, Double-Masked, Placebo-Controlled, Parallel, One-Day Trial MIRA-2 0.75% NyxolMydriatic Agent A, B, or C 12 USsites~168healthy subjects Nyxol drop(s) (1 day) Mydriasis Time -1 HourPlaceboMydriatic Agent A, B, or C Placebo drop(s) (1 day) Tre
Slide 19: Nyxol Demonstrated Clinical Effect in RM
19 Nyxol Demonstrated Clinical Effect in RMKey Endpoints Observed from MIRA-1 Phase 2b Trial MIRA-1 Phase 2 Trial RM Nyxol Reduces More Subjects to Pupil Diameter (PD) BaselinePercent of Subjects Returning to ≤ 0.2 mm of Baseline after Treatment With either Phenylephrine or Tropicamide Nyxol Reduces
Slide 20: Presbyopia – Chronic Opportunity
20 Presbyopia – Chronic OpportunityAging Population Drives Demand for Alternatives to Reading Glasses 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, rather than hinder,
Slide 21: Presbyopia – Pupil Modulation Eye Drops May Replace Reading Glasses
21 Presbyopia – Chronic OpportunityPupil Modulation Eye Drops May Replace Reading Glasses “Pin-hole” effect of Nyxol and low dose pilocarpine may improve near vision by increasing depth of focus as validated by other devices/therapiesMore durable combination of two miotics affecting different muscle
Slide 22: Presbyopia VEGA-1 Phase 2 Proposed Design
22 Presbyopia VEGA-1 Phase 2 Proposed DesignPlanned Randomized, Double-Masked, Placebo-Controlled Trial Visit 1 VEGA-1 Eligibility Screening Randomization 4 arms 0.75% Nyxol Placebo 20 US sites~152presbyopic patients Visit 2(3 – 5 Days Later) Screening P Endpoints Primary: % of subjects with ≥ 3 lin
Slide 23: Nyxol Demonstrated Clinical Effect in Presbyopia
Nyxol Demonstrated Clinical Effect in PresbyopiaKey Endpoints Observed from Multiple Phase 2 Trials Source: ORION-1 Nyxol Alone LDP AloneAverage pupil size in photopic conditions is 3.5 to 4+ mm ~0.7 to 1+ mm P ORION-1 Phase 2 Trial Pinhole PD Size Illustrated with Addition of LDP to Nyxol1.5 to 2+
Slide 24: APX3330
APX3330 APX3330 DR Diabetic Retinopathy DME Diabetic Macular Edema wAMD Wet Age-Related Macular Degeneration 24
Slide 25: APX3330 is a Ref-1 Inhibitor
25 APX3330 is a Ref-1 InhibitorRef-1 Involved in Multiple 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-αChemokines Neovascularization
Slide 26: APX3330 Product Candidate Profile
26 APX3330 Product Candidate ProfileFirst-in-Class Ref-1 Inhibitor Phase 2 Ready for Retina Diabetic Indications Expected Efficacy Data Improving Eye Health in Diabetics↓ Inflammation↓ Hypoxia Signaling↓ Abnormal AngiogenesisEnhance Compliance & Exposure Oral pill may reduce the burden of frequent a
Slide 27: Diabetic Eye Opportunity
27 Diabetic Eye Opportunity 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 worldwide, especially in wo
Slide 28: APX3330 to Treat Patients Before Vision Loss Occurs
28 Diabetic Retinopathy & Macular EdemaAPX3330 to Treat Patients Before Vision Loss Occurs Potential First Oral Therapy to be used as an earlier intervention for the diabetic eye before vision symptoms appear or as add-on therapy to current anti-VEGF treatmentProven Unique Mechanism that may decreas
Slide 29: DR/DME ZETA-1 Phase 2 Proposed Design
29 DR/DME ZETA-1 Phase 2 Proposed DesignPlanned Randomized, Double-Masked, Placebo-Controlled Trial NPDR = non-proliferative diabetic retinopathy (which includes non centrally involved diabetic macular edema) PDR = proliferative diabetic retinopathy (which includes non centrally involved diabetic ma
Slide 30: APX3330 Generally Well Tolerated with Clinical Signals
APX3330 Generally Well Tolerated with Clinical SignalsObservations from Pre-Clinical Studies and 11 Clinical Trials of APX3330 L-CNV Mouse Retina Model APX3330 Reduces Neovascularization Similar to Eylea in Preclinical ModelsLesion Size and Corresponding Fluorescent Stains in L-CNV Models Treated wi
Slide 31: Boards and Milestones
31 PHARMA Boards and Milestones
Slide 32: Prestigious Ocular Medical Advisory Board
Richard Lindstrom, MD University of Minnesota Ed Holland, MDLoyola University Chicago Jay Pepose, MD UCLA Jack Holladay, MD University of Texas Thomas Samuelson, MD University of Minnesota Paul Karpecki, OD Indiana University elCON MedicalEliot Lazar, MDGeorgetown University Gary Novak, PhD UC Davis
Slide 33: Ocuphire Board of Directors
Ocuphire Board of DirectorsSeasoned Directors with Decades of Biotech Drug Development and M&A/Financings Sean Ainsworth, MBALead Independent Director,Board Director James Manuso, PhD/MBABoard Director Alan R. Meyer, MBABoard Director MFR/IP Advisor Richard Rodgers, MBABoard Director Susan Benton, M
Slide 34: 2020 to 2022 Cadence of Milestones
2020 to 2022 Cadence of MilestonesMultiple Data Catalysts for Value-Building Nyxol NDA filing for RM & NVD in early 2023 Ongoing partnering discussions with leading ophthalmic companies (inc. European and Asian players) NVD Podium Presentation at AAO 2018Initiate/Report Phase 2b Data for ORION-1Init
Slide 35: Restore Vision & Clarity
Restore Vision & Clarity www.ocuphire.com ir@ocuphire.com
Slide 36: NVD Endpoint: Low Contrast Visual Acuity Chart
36 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 37: DR/DME Endpoint: Diabetic Retinopathy Severity Scale
37 DR/DME Endpoint: Diabetic Retinopathy Severity Scale (DRSS)FDA Accepted Endpoint for DR (EYLEA® in PANORAMA Pivotal Trial) EYLEA Product Pamphlet ® 1 2 3 4 5, 6 7 – 13 DRSS (10) (20) (35) (43) (47, 53) (60, 61, 65, Score 71, 75, 85,90) Description DR Absent Micro- aneurysm only Mild NPDR Moderate
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