Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (January 2021)
37 slides from Opus Genetics's investor presentation, Jan 6, 2021
Slide 1: Ocuphire Corporate Presentation January 2021
Ocuphire Corporate Presentation January 2021
Slide 2: Disclosures and Forward Looking Statements
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
Late Clinical Stage Company Targeting Large, Unmet Ophthalmic Markets Significant Clinical Data and Regulatory 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 o
Slide 4: Ocuphire Management Team
Ocuphire Management Team Decades of Biotech and Drug Development Experience Amy Rabourn, CPAVP Finance Daniela Oniciu, PhDHead CMC and Global Clinical Supply Kostas Charizanis, PhD, MBASenior Director Market Strategy and R&D Mitch Brigell, PhDHead Clinical Development and Strategy Laura GambinoProj
Slide 5: Large Unmet Opportunities for the Aging Eye
Large Unmet Opportunities for the Aging Eye Source: GlobalData Market Research Report, 2020 Developing Drugs to Treat Front & Back of the Eye Diseases Back Front PresbyopiaU.S. Prevalence: ~120M Night Vision DisturbancesU.S. Prevalence: ~16M adults Reversal of Mydriasis~100M pupil dilations per year
Slide 6: Product Candidate Pipeline 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) Initiated Phase 3 LYNX-1 trial 4Q2020;Data expected in 3Q21 (n=160) 0.75% Nyxol®Eye Drop Reversal
Slide 7: Extensive Development on Both Drug Candidates
11Phase 1 & Phase 2 Trials >340Subjects Dosed Exposure in Humans 365 Days Patents to2034+ Extensive Development on Both Drug Candidates Well-Controlled Phase 1 & Phase 2 Clinical Programs Set Stage for NDA Path NCE Development Pathway Studied in inflammation/hepatitis & cancer patients 7Phase 1 & Ph
Slide 8: Nyxol Phentolamine Mesylate
Nyxol® Phentolamine Mesylate NVD Night Vision Disturbances P Presbyopia RM Reversal of Mydriasis
Slide 9: Nyxol History & MOA
Nyxol History & MOA 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®) – intramuscular injectionPM has been reformulated as a topical e
Slide 10: Nyxol Product Candidate Profile
Improving Vision↓ Pupil Size (moderate miotic)↑ Contrast Sensitivity (night)↑ Near Visual Acuity (light/dark)↑ Distance Visual Acuity No Systemic EffectsNo Changes in Blood PressureNo Changes in Heart RateTolerated Topical EffectsMild / Transient / Reversible Eye RednessIOP Unchanged or Decreased↓ I
Slide 11: Night Vision Disturbances (NVD) – Chronic Opportunity
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 Opportunity Peripheral imperfections scatter light when pupils enlarge in dim light, causing halos, starbursts, and glare that imp
Slide 12: NVD – Nyxol's Potential Differentiated Solution
Night Vision Disturbances (NVD) – Chronic Opportunity Moderate Decrease in Pupil Size for scattered light gets blocked by the iris Clinical Effect to potentially improve low contrast night vision as seen in trialsTolerable with a minimal side effect profile Convenient and Durable with chronic once-d
Slide 13: NVD LYNX-1 Phase 3 Registration Design
NVD LYNX-1 Phase 3 Registration Design Randomized, Double-Masked, Placebo-Controlled Two-Week Trial LYNX-1 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 measures (distance
Slide 14: Nyxol Demonstrated Clinical Effect in NVD
ORION-1 Phase 2 Trial NYX-SNV Phase 2 Trial Nyxol Demonstrated Clinical Effect in NVD Key Endpoints Observed in Multiple Phase 2 Trials Improved Low Contrast Distance Visual Acuity* Source: NYXG-201 Durable > 24 hour Pupil Modulation Effect % of Eyes with Mesopic Low Contrast Visual Acuity Improveme
Slide 15: Reversal of Mydriasis (RM) – Acute Treatment
Reversal of Mydriasis (RM) – Acute Treatment At every annual eye exam and many specialty visits, pupils are pharmacologically dilated, impairing vision for 6-24 hoursDilated eyes:heightened sensitivity to light inability to focus reading, working, and driving are difficult halos and glare The Proble
Slide 16: Reversal of Mydriasis – Regulatory Precedent and Solution
After Before Reversal of Mydriasis (RM) – Acute Treatment 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 reduce pupil size and reverse mydriasis by counteractin
Slide 17: RM MIRA-2 Phase 3 Registration Design
RM MIRA-2 Phase 3 Registration Design Randomized, Double-Masked, Placebo-Controlled, Parallel, One-Day Trial MydriasisTime -1 Hour MIRA-2 1:1 Mydriatic Agent A, B, or C 0.75% Nyxol Placebo 12 US sites~168 healthy subjects Nyxol drop(s)(1 day) Mydriatic Agent A, B, or C Placebo drop(s)(1 day) Treatme
Slide 18: Nyxol Demonstrated Clinical Effect in RM
Nyxol Demonstrated Clinical Effect in RM Key Endpoints Observed from MIRA-1 Phase 2b Trial MIRA-1 Phase 2 Trial Nyxol Reduced More Subjects to Pupil Diameter (PD) Baseline Source: MIRA-1 Nyxol Reduced PD Faster Across Phenylephrine & Tropicamide Mydriatic Agents Source: MIRA-1 RM Percent of Subjects
Slide 19: Presbyopia – Chronic Opportunity
Presbyopia – Chronic Opportunity 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, quality of life Source: GlobalData Market Research Report, 2020 The
Slide 20: Presbyopia – Nyxol's Potential Differentiated Solution
Presbyopia – Chronic Opportunity “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 muscles (iris dilator and sphincter) involved in pupil size mod
Slide 21: Presbyopia VEGA-1 Phase 2 Proposed Design
Presbyopia VEGA-1 Phase 2 Proposed Design Planned Randomized, Double-Masked, Placebo-Controlled One-Week Trial Visit 1 VEGA-1 Eligibility Screening Randomization 4 arms 0.75% Nyxol Placebo 20 US sites~152 presbyopic patients Visit 2 (3 – 5 Days Later) Screening P Primary: % of subjects with ≥ 3 line
Slide 22: Nyxol Demonstrated Clinical Effect in Presbyopia
Nyxol Demonstrated Clinical Effect in Presbyopia Key Endpoints Observed from Multiple Phase 2 Trials Average pupil size in photopic conditions is 3.5 to 4+ mm Source: ORION-1 0.4% 1.5 to 2+ mm PD reductionAchieve the pin-hole (1.6 to 2 mm) effect Nyxol Alone LDP Alone Near VA Line Improvement at Day
Slide 23: Presbyopia Eye Drops Competitive Landscape
Phase 3 Phase 2 Phase 1 Presbyopia Eye Drops Competitive Landscape An ideal formulation for presbyopia treatment would meet the following criteria: Comfort and tolerabilityFast onsetLong durationEfficient pupil size modulationStrong safety profileMaintain good distance visual acuity Corporate Websit
Slide 24: APX3330
APX3330 APX3330 DR Diabetic Retinopathy DME Diabetic Macular Edema wAMD Wet Age-Related Macular Degeneration
Slide 25: APX3330 is a Ref-1 Inhibitor
APX3330 is a Ref-1 Inhibitor Ref-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 Luc
Slide 26: APX3330 Product Candidate Profile
APX3330 Product Candidate Profile First-in-Class Ref-1 Inhibitor Phase 2 Ready for Retina Diabetic Indications Improving Eye Health in Diabetics ↓ Inflammation ↓ Hypoxia Signaling ↓ Abnormal AngiogenesisEnhance Compliance & ExposureOral pill may reduce the burden of frequent anti-VEGF injections Few
Slide 27: Diabetic Eye Opportunity
Diabetic Eye Opportunity DR ~7.7M Patients DME ~750K Patients Diabetic Retinopathy & Macular Edema Diabetic retinopathy (DR) and diabetic macular edema (DME) are a leading cause of vision loss worldwide, especially in working age adults in developed countriesDiabetes damages small blood vessels with
Slide 28: Diabetic Retinopathy & Macular Edema – Potential Differentiated Solution
Diabetic Retinopathy & Macular Edema 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 Novel Mechanism that may decrease both inflammation and VEGF activityConvenient option
Slide 29: DR/DME ZETA-1 Phase 2 Proposed Design
DR/DME ZETA-1 Phase 2 Proposed Design NPDR = non-proliferative diabetic retinopathy (which includes non centrally involved diabetic macular edema)PDR = proliferative diabetic retinopathy (which includes non centrally involved diabetic macular edema) Planned Randomized, Double-Masked, Placebo-Control
Slide 30: APX3330 Generally Well Tolerated with Clinical Signals
APX3330 Generally Well Tolerated with Clinical Signals Observations from Pre-Clinical Studies and 11 Clinical Trials of APX3330 Source: Unpublished Data Dec 2019 Lesion Size and Corresponding Fluorescent Stains in L-CNV Models Treated with APX3330 at 25 mg/kg Human Pharmacokinetics of APX3330 at 120
Slide 31: Boards and Milestones
31 PHARMA Boards and Milestones
Slide 32: Prestigious Ocular Medical Advisory Board
Richard Lindstrom, MDUniversity of Minnesota Ed Holland, MDLoyola University Chicago Jay Pepose, MDUCLA Jack Holladay, MDUniversity of Texas Thomas Samuelson, MDUniversity of Minnesota Paul Karpecki, ODIndiana University Eliot Lazar, MDGeorgetown University Gary Novak, PhDUC Davis Marguerite McDonal
Slide 33: Ocuphire Board of Directors
Ocuphire Board of Directors Seasoned 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 DirectorMFR/IP Advisor Richard Rodgers, MBABoard Director Susan Bent
Slide 34: 2020 to 2022 Cadence of Milestones
2020 to 2022 Cadence of Milestones Multiple 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 2018 Initiate/Report Phase 2b Data for ORION-1In
Slide 35: www.ocuphire.com
www.ocuphire.comir@ocuphire.com
Slide 36: NVD Endpoint: Low Contrast Visual Acuity Chart
NVD Endpoint: 5% Low Contrast Visual Acuity (LCVA) Chart Precision Vision FDA Accepted Endpoint for Contrast Sensitivity Assessment Before Treatment* 3 Lines Improvement Illustration Primary Endpoint of Nyxol LYNX-1 Trial Percent of subjects with ≥ 3 lines of improvement in mesopic low contrast best
Slide 37: DR/DME Endpoint: Diabetic Retinopathy Severity Scale
DR/DME Endpoint: Diabetic Retinopathy Severity Scale (DRSS) EYLEA Product Pamphlet ® FDA Accepted Endpoint for DR (EYLEA® in PANORAMA Pivotal Trial) Patients included in the ZETA-1 Trial Primary Endpoint of APX3330 ZETA-1 Trial Percent of patients with a ≥ 2 step improvement on the DRSS score at wee
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