Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (August 2021)
53 slides from Opus Genetics's investor presentation, Aug 12, 2021
Slide 1: Ocuphire Corporate Presentation
Ocuphire Corporate Presentation August 12, 2021 Mina Sooch CEO Exhibit 99.2
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 Ronil Patel, MSSenior Director BD and Market Strategy Mitch Brigell, PhDHead Clinical Development and Strategy Chris ErnstQuality Assurance L
Slide 5: Large Unmet Opportunities for the Aging Eye
Large Unmet Opportunities for the Aging Eye Source: GlobalData Market Research Report, 2020; Company Estimates for Market Size 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 Mydr
Slide 6: 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 Reversal of Mydriasis (RM) Initiated Phase 3 MIRA-2 trial 4Q20; Topline data reported in 1Q21 (n=185)Initiate Phase 3 MIRA-3 trial 2H21; Data e
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, 2, and 3 Clinical Programs with MIRA-2 Data Leading the NDA Path NCE Development Pathway Studied in inflammation/hepatitis & cancer patient
Slide 8: Nyxol Phentolamine Mesylate
Nyxol® Phentolamine Mesylate Presbyopia RM Night Vision Disturbances Reversal of Mydriasis NVD P
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: Nyxol: Reversal of Mydriasis
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of Mydriasis 0.4% +
Slide 12: Reversal of Mydriasis (RM) – Acute Treatment
Reversal of Mydriasis (RM) – Acute Treatment At many annual eye exams and 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 Problem Ann
Slide 13: Reversal of Mydriasis – Nyxol's Potential Differentiated Solution
After Before Reversal of Mydriasis (RM) – Acute Treatment 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 potentially reduce pupil size and counteract the effect of mydriat
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 MydriasisTime -1 Hour MIRA-2 1:1 Mydriatic Agent A, B, or C 0.75% Nyxol Placebo 12 US sites168 target healthy subjects Nyxol drop(s)(2 drops study eye, 1 drop fellow eye) Mydriatic
Slide 15: Primary Endpoint: % Returning to Baseline PD at 90 Min
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 & Al
Slide 16: Secondary Endpoint: Mean Pupil Diameter by Mydriatic Agent
Secondary Endpoint: Mean Pupil Diameter Over Time by Mydriatic Agent Source: mITT Population, MIRA-2 Trial, Standard Error bars are shown. Nyxol Reduced Pupil Diameter With All Mydriatic Agents; More Rapidly with Phenylephrine as Expected MIRA-2 Phase 3 Trial Mydriatic Max pupil dilation, Treatment
Slide 17: Secondary Endpoint: Returning to Baseline by Iris Color
Secondary Endpoint: % of Subjects Returning to Baseline PD by Iris Color Source: MIRA-2 Trial mITT Population,, Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd) Evidence of Efficacy in Subjects with Either Light or Dark Irides, with a More Vigorous Response in Light Irides MI
Slide 18: Secondary Endpoint: Accommodation and Time Savings
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. Note: Worsening of accommodation was defined as an amplitude decr
Slide 19: Summary of Positive MIRA-2 Phase 3 Results
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 significanceEfficacy for all 3 mydriatic agents – phenylephrine, tropicamide, and Paremyd® E
Slide 20: Reversal of Mydriasis Market Opportunity
Reversal of Mydriasis (RM) Market Opportunity GlobalData market research reportVision Care Market Grows 2.4 Percent in 12-Months Ending September 2019. Vision Monday, January 20, 2020. With No Commercially Available Treatment, Nyxol May Provide Significant Revenue Potential > 65% PatientsReport mode
Slide 21: Nyxol: Presbyopia
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of Mydriasis 0.4% +
Slide 22: 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 23: Presbyopia – Nyxol's Potential Differentiated Solution
Presbyopia – Chronic Opportunity “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 (iris dilator and sphincter) involved in pupil size modu
Slide 24: Product Profile: Nyxol + Low-Dose Pilocarpine Combo
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 injectabl
Slide 25: Presbyopia VEGA-1 Phase 2 Design
Presbyopia VEGA-1 Phase 2 Design Clinical trial NCT#04675151. DCNVA = distance-corrected near visual acuity . BCDVA = best corrected distance visual acuity Randomized, Double-Masked, Placebo-Controlled, Multi-Center One-Week Trial Primary: % of subjects with ≥ 3 lines of improvement in distance-corr
Slide 26: Primary Endpoint: Letter Gain in Photopic DCNVA at 1 Hour
Primary Endpoint: % of Subjects ≥ 15 Letter Gain in Photopic DCNVA at 1 Hour Primary Endpoint Was Significantly Met for Nyxol + LDP Gaining ≥ 15 Letters Near Vision VEGA-1 Phase 2 Trial p=0.006 30% Placebo Adjusted Responders Source: VEGA-1 TLR Table 14.2.1.2 % of Subjects With Improvement From Base
Slide 27: Secondary Endpoint: DCNVA Gain at All Timepoints
Secondary Endpoint: % of Subjects ≥ 15 Letter DCNVA Gain At All Timepoints 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 letters is 3 lines. Nyxol + LDP Had Strong Response with ≥ 15 Letter Near Gain from 30 M
Slide 28: Near Gain and Distance Loss Endpoint
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 Con
Slide 29: Secondary Endpoint: Mean Pupil Diameter Over Time
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) Achieved Pupil Size ~2mm in Nyxol+LDP Consistent with 3-line Improvement in Near Vision Nyxol+LDP arm statistically s
Slide 30: Secondary Endpoint: Safety Findings
Secondary Endpoint: Safety Findings Source: VEGA-1 TLR Table 14.3.1.1 Overall Summary of Treatment Emergent Adverse Events (TEAE) (Safety Population)Table 14.3.1.3 Treatment-Emergent Adverse Events (TEAE) by System Organ Class, Preferred Term, and Severity (Safety Population) Nyxol+LDP Combination W
Slide 31: Potential 'Best in Class' Presbyopia Drop
Product Attributes* Nyxol+LDP compared to market leader 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 32: Presbyopia Eye Drops Competitive Landscape
Phase 3 Phase 2 Phase 1 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
Slide 33: Nyxol: Night Vision Disturbances
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of Mydriasis 0.4% +
Slide 34: Night Vision Disturbances – 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 35: 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 36: NVD LYNX-1 Phase 3 Registration Design
NVD LYNX-1 Phase 3 Registration Design Ongoing 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 (d
Slide 37: 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 38: APX3330
APX3330 APX3330 DR Diabetic Retinopathy DME Diabetic Macular Edema wAMD Wet Age-Related Macular Degeneration
Slide 39: 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 40: APX3330 History and Ref-1 Inhibition Mechanism
APX3330 History and Ref-1 Inhibition Mechanism 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 Neov
Slide 41: APX3330 Down-Regulates VEGF and Inflammatory Cytokines
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 ha
Slide 42: APX3330 Generally Well Tolerated with Clinical Signals
APX3330 Generally Well Tolerated with Clinical Signals 1. Silva 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 Observa
Slide 43: 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 44: DR/DME ZETA-1 Phase 2 Design
DR/DME ZETA-1 Phase 2 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) Ongoing, Randomized, Double-Masked, Placebo-Controlled 24-W
Slide 45: APX3330's 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 46: Boards and Milestones
46 PHARMA Boards and Milestones
Slide 47: Prestigious Ocular Medical Advisory Board
Richard Lindstrom, MDUniversity of Minnesota Ed Holland, MDLoyola University Chicago Jay Pepose, MD, PhDUCLA Jack Holladay, MDUniversity of Texas Thomas Samuelson, MDUniversity of Minnesota Paul Karpecki, ODIndiana University Eliot Lazar, MDGeorgetown University Gary Novak, PhDUC Davis Marguerite Mc
Slide 48: Ocuphire Board of Directors
Ocuphire Board of Directors Seasoned 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, MB
Slide 49: 2021 to 2022 Ocuphire Cadence of Milestones
2021 to 2022 Ocuphire Cadence of Milestones Multiple Data Catalysts On Path To NDA(s) Ongoing partnering discussions with leading ophthalmic companies (including European and Asian players) Completion of APX3330 LicenseARVO 2020 Presentation for MIRA-1 & ORION-1FDA EOP2 Meeting May 2020Completion of
Slide 50: OCUP – Market Snapshot
OCUP – Market Snapshot Source: FactSet Sufficient Cash Runway Into Late 2022 and Substantial Trading Liquidity Ticker OCUP Price $4.89 As of 8-10-21 Market Cap $82 M As of 8-10-21 Primary/FD Shares 16.9 M / 18.6 M As of 6-30-21 Cash $24.2 M As of 6-30-21 Cash Runway Into Late 2022 Guidance as of 6-3
Slide 51: www.ocuphire.com
www.ocuphire.comir@ocuphire.com
Slide 52: 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 53: 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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