Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (January 2022)
50 slides from Opus Genetics's investor presentation, Jan 5, 2022
Slide 1: Ocuphire Corporate Presentation
Ocuphire Corporate Presentation Mina Sooch CEO January 5, 2022 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: Differentiated, Late-Stage Pipeline Targeting Large Unmet Ophthalmic Markets
Differentiated, Late-Stage Pipeline Targeting Large Unmet Ophthalmic Markets Of The Front And Back Of The EyeNyxol with > 330 patients treated across 9 trials (505(b)(2) regulatory pathway)APX3330 with > 340 patients treated across 11 trials (NCE development pathway)Nyxol and APX3330 achieved promis
Slide 4: Large Unmet Opportunities For The Aging Eye
Large Unmet Opportunities For The Aging Eye Nyxol To Treat Front Of The Eye And APX3330 For The Back Of The Eye Diseases Nyxol® $9B - $18B $325M - $1B $2B - $4B $3B - $7B $1B - $3B APX3330 128 MPatients 100 MPatients 16 MPatients Presbyopia Reversal of Mydriasis Night Vision Disturbances Diabetic Re
Slide 5: Ocuphire Pipeline & Clinical Milestones
Ocuphire Pipeline & Clinical Milestones Note: 0.75% Nyxol (Phentolamine Ophthalmic Solution) is the same as 1% Nyxol (Phentolamine Mesylate Ophthalmic Solution) Product Candidate Indication Pre-clinical Phase 1 Phase 2 Phase 3 RegulatoryApproval Anticipated Milestones 0.75% Nyxol®Eye Drop Reversal o
Slide 6: Nyxol Eye Drops: RM, Presbyopia, NVD
6 RM Reversal of Mydriasis P NVD Presbyopia Night Vision Disturbance 0.4% + Nyxol®Eye Drops
Slide 7: Nyxol MOA & History
Nyxol MOA & History Phentolamine Mesylate Reformulated As A Proprietary Topical Eye Drop Nyxol Phentolamine Mesylate is Active Ingredient in Nyxol: α1 & α2 Antagonist Blocking α1Reduces Pupil Size Blocking α1Dilates Blood Vessels Phentolamine mesylate is currently approved for 2 indications:Regiti
Slide 8: Nyxol Product Candidate Profile
Nyxol Improves Vision by Decreasing Pupil Size↑ Near & Distance Visual Acuity↑ Contrast Sensitivity (night) No Systemic EffectsNo Changes in Blood PressureNo Changes in Heart RateWell-Tolerated Topical EffectsMild, Transient, Reversible Eye RednessIOP Unchanged or DecreasedNo Headaches Favorable saf
Slide 9: Nyxol for Reversal of Mydriasis (RM)
I have to stay indoors. They say it only lasts a few hours, but it lasts all day, and it is very annoying.RM Patient, Age 51 RM 9 Nyxol®forReversal of Mydriasis (RM)
Slide 10: Reversal Of Mydriasis (RM) Market Opportunity
Reversal Of Mydriasis (RM) Market Opportunity With No Commercially Available Treatment, Nyxol May Achieve Significant Revenue Potential > 65% of PatientsReport moderate to severe negative impact of dilated exams1 $10 - $20Price range for cash pay per patient with room for physician markup1 100M+Gene
Slide 11: MIRA-2/3 Phase 3 Registration Trial Design
MIRA-2/3 Phase 3 Registration Trial Design Randomized, Double-Masked, Placebo-Controlled, Parallel, One-Day Trial Mydriasis-1 Hour 1:1 Mydriatic Agent A, B, or C 0.75% Nyxol Placebo 12 to 16 US sites185 to 330target healthy subjects Nyxol drop(s)(2 drops study eye, 1 drop fellow eye) Mydriatic Agent
Slide 12: MIRA-2 RM Phase 3 Trial Met Primary & Secondary Endpoints
MIRA-2 RM Phase 3 Trial Met Primary & Secondary Endpoints Source: MIRA-2 Trial, mITT Population (same as Safety Population), *Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd) 49% Of Patients Returned To < 0.2mm Of Baseline At 90mins Vs. 7% Placebo Max pupil dilation, Treatmen
Slide 13: Summary Of Positive MIRA-2 Phase 3 Results
Summary Of Positive MIRA-2 Phase 3 Results For Nyxol Eye Drops mITT Population, MIRA-2 Trial Rapid Efficacy With A Favorable Safety Profile In Reversing Mydriasis With Nyxol No serious AEs, no drop-outs from AEs, no systemic AEs were observed in ≥ 5% of subjectsMild, transient conjunctival hyperemia
Slide 14: NDA Submission Targeted In Late 2022
NDA Submission Targeted In Late 2022 Ongoing Activities Sets Ocuphire On Path To A Potential Regulatory Approval In 2023 Complete RM trial with 20 subjects ages 3 to 11 per agreed FDA initial pediatric plan Pediatric Safety Complete a 2nd Phase 3 trial in RM with ~330 subjects which also meets 24-ho
Slide 15: Pre-Commercial & Go-To-Market Strategy
Pre-Commercial & Go-To-Market Strategy Activities Underway To Support Capital-Efficient Nyxol RM Commercial Launch Brand Awareness Across Eye Care Professionals PhysicianTargeting Patient Journey Market Development Engage leading Key Opinion Leaders and Professional Societies to establish OCUP as an
Slide 16: Nyxol for Presbyopia
P 16 Nyxol® forPRESBYOPIA “By Age 45, 80% of Americans will struggle with Presbyopia, and by age 50, nearly everyone will.” NY Times
Slide 17: 2021: The Time For Presbyopia Drops
2021: The Time For Presbyopia Drops Sources: Academic review articles, journals, and publications Headlines From Academia And Industry Articles Thru The Year With An Early First Approval “The correction of presbyopia remains ophthalmology’s ‘Holy Grail’…”-OIS 10/29/2021 P
Slide 18: Presbyopia Is A Burgeoning Opportunity
Presbyopia Is A Burgeoning Opportunity Global Prevalence of Presbyopia, 2018, Fortune Business Insights Reading Glasses Forecast 2016-2027, Cataract & Refractive Surgery Today, 2021, NEI 2010 data.Vitale S. et. Al. JAMA Ophthalmology, 2008, Vision problems, US, Arch. Ophthal, 2014, Vision Monday. La
Slide 19: Product Profile: Nyxol + Low-Dose Pilocarpine Combo
Product Profile: Nyxol ® + Low-Dose Pilocarpine (LDP) Combo Source: 1) Nyxol® data from 9 completed trials; Pilocarpine Product label and Literature 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 Sp
Slide 20: 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 21: VEGA-1 Phase 2 Trial Met Primary & Secondary Endpoints
VEGA-1 Phase 2 Trial Met Primary & Secondary Endpoints 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 Minutes To 6 Hours VE
Slide 22: Secondary Endpoints: Improved DCNVA Without BCDVA Loss
Secondary Endpoints: Improved DCNVA Without BCDVA Loss 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); Table 14.2.1.2 Percent of Subjects With Improvement From
Slide 23: 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 24: Summary Of Positive VEGA-1 Phase 2 Results
Summary Of Positive VEGA-1 Phase 2 Results PP Population, VEGA-1 Trial Nyxol + LDP Had Strong Efficacy Response & Well Tolerated Safety Profile Efficacy Safety Met primary endpoint with statistical significance at 1 hour with Nyxol® plus Low-Dose Pilocarpine (LDP)Met key secondary endpoints with sta
Slide 25: Potential 'Best in Class' Presbyopia Drop
Potential ‘Best in Class’ Presbyopia Drop Product Attributes* Nyxol+LDP VUITYTM 1) Efficacy (3 Line Gain in DCNVA - Primary Endpoint Responders) 61% 26-31% 2a) Safety: Loss of Distance in Mesopic None None 2b) Safety: Tolerability No Headaches >5% Headaches 2c) Safety: Conjunctival Hyperemia <5% red
Slide 26: Presbyopia Eye Drops Competitive Landscape
Phase 2 Phase 1 Presbyopia Eye Drops Competitive Landscape Corporate Websites, Grzybowski, A, Markeviciute A, Zemaitiene R. A Review of Pharmacological Presbyopia Treatment. 2020 Pupil modulation MOA Combination drugs Soften lens MOA Phase 3 Phase 2 Phase 1 Allergan(VUITYTM;1.25% pilo) Orasis(CSF-1;
Slide 27: Nyxol for Dim Light or Night Vision Disturbances
I’m no longer comfortable driving at night, especially with my son in the car. I have a hard time playing beach volleyball in the evenings due to the bright lights at the courts. Post-LASIK, Age 42 Nyxol® forDim Light or Night Vision Disturbances 27 NVD
Slide 28: Market Opportunity In Dim Light Or Night Vision Disturbances
Market Opportunity In Dim Light Or Night Vision Disturbances No Approved Treatments With Ripe Opportunity For Growth No Approved Treatments $2B - $4BEstimated US NVD Market Opportunity Peripheral imperfections scatter light when pupils enlarge in dim light, causing halos, starbursts, and glare that
Slide 29: 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 30: Nyxol Demonstrated Clinical Effect In NVD
ORION-1 Phase 2 Trial NYX-SNV Phase 2 Trial Nyxol Demonstrated Clinical Effect In NVD *NYX-SNV trial was small and not designed for a statistical 3-line improvement in low-contrast visual acuity; the ~20% effect was used for powering and sizing of Phase 3 trial Key Endpoints Observed In Multiple Pha
Slide 31: APX3330 for DR and DME
DR DME 31 Diabetic Retinopathy Diabetic Macular Edema Apx3330tablets
Slide 32: Large, Unmet Need in Diabetic Eye Diseases
Large, Unmet Need in Diabetic Eye Diseases (US) 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 worldwideDiabetes damages small blood vessels within the eye causing leakage, oxygen
Slide 33: APX3330: Drug Development History And Patents
APX3330: Drug Development History And Patents Significant Preclinical & Clinical Data Supporting Human Safety, MOA, and PK 11Phase 1 & Phase 2 Trials >340Subjects Dosed Exposure in Humans 365 Days Patent Coverage 2034+ Studied in inflammation/hepatitis & cancer patients (Studied by Eisai & Apexian,
Slide 34: APX3330 History And Ref-1 Inhibition Mechanism
APX3330 History And Ref-1 Inhibition Mechanism Logsdon et al (2018), Li et al (2014). Ref-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 Fact
Slide 35: APX3330 Down-Regulates VEGF Protein And Anti-Inflammatory Cytokines
APX3330 Down-Regulates VEGF Protein And Anti-Inflammatory Cytokines Tao Yan et al. APX3330 Promotes Neurorestorative effects after stroke in type one diabetic rats. Aging and Disease. Vol 9, Oct 2018Apurinic/Apyrimidinic endonuclease 1 regulates inflammatory response in macrophages. Jedinak A, Dudhg
Slide 36: Preclinical Data: Oral APX3330 Blocks Neovascularization
Preclinical Data: Oral APX3330 Blocks Neovascularization - 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- Publi
Slide 37: Phase 1/2 Clinical Trials: PK Data Supporting ZETA-1
Phase 1/2 Clinical Trials: PK Data Supporting The ZETA-1 Trial 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
Slide 38: APX3330 Product Candidate Profile
Improving Eye Health in Diabetics ↓ Inflammation ↓ Abnormal AngiogenesisEnhance Compliance & ExposureOral pill may reduce the burden of frequent anti-VEGF injections Few Systemic Adverse Effects< 5% Mild Gastrointestinal (diarrhea)< 5% Mild Skin Rash (reversible)Lack of Significant Acute Neurologic,
Slide 39: DR/DME ZETA-1 Phase 2b Design
DR/DME ZETA-1 Phase 2b 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-
Slide 40: Innovative Approach For Retinal Diseases With APX Platform
Innovative Approach For Retinal Diseases With APX Platform Potential First Oral Rx for Retina DiseasesFirst-line earlier intervention for the diabetic eye Add-on therapy to current anti-VEGF treatmentsProven Novel MechanismMay decrease both inflammation and angiogenesisConvenient Daily RegimenFavora
Slide 41: Team/Boards, Milestones, And Financial Data
Team/Boards, Milestones, And Financial Data
Slide 42: Ocuphire Management Team
Ocuphire Management Team Decades Of Biotech And Drug Development Experience Mina Sooch, MBAPresident & CEO and Founder Drey ColemanVP, Clinical Operations Amy Rabourn, CPAVP, Finance Charlie Hoffmann, MBAVP Corporate Development and Operations Mitch Brigell, PhDHead, Clinical Development and Strateg
Slide 43: Ocuphire's World-Class Medical Advisory Board
Ocuphire's World-Class Medical Advisory Board Fortunate For The Insights Of Leading KOLs & Drug Candidate Co-Founders James Katz, MDUniversity of Illinois Jay Pepose, MD, PhDUCLA Thomas Samuelson, MDUniversity of Minnesota Paul Karpecki, ODIndiana University Eliot Lazar, MDGeorgetown University Marg
Slide 44: Ocuphire Board of Directors
Ocuphire Board of Directors Sean Ainsworth, MBALead Independent Director, Board Director James Manuso, PhD/MBABoard Director Jay Pepose, MD, PhDBoard Director Richard Rodgers, MBABoard Director Susan Benton, MBABoard Director Cam Gallagher, MBAChair, Board Director Mina Sooch, MBAVice-Chair, Boa
Slide 45: Ocuphire Cadence Of Milestones
45 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 2nd Phase 3 Data for RM Report Pediatric Data in RM Report Phase 3 Data for NVD Submit Nyxol NDA for RM
Slide 46: Ophthalmology – An Attractive Biotech Sector
Ophthalmology – An Attractive Biotech Sector FavorableRegulatoryEnvironment Source:1.Endpoint Dec 29, 2021- Hitting a new record on drug approvals, the FDA offers a thumbs-up to another atopic dermatitis contender; OIS Year in Review 2021; Company press releases Deal Activity And FDA Approvals In Op
Slide 47: OCUP – Market Snapshot
OCUP – Market Snapshot Source: FactSet Active Trading Volume And Sufficient Cash Runway Through 2Q 2023 Ticker OCUP Price $3.73 As of 12-31-21 Market Cap $64.8 M As of 12-31-21 Shares Outstanding 18.8 M As of 12-31-21 Cash $24.5 M As of 12-31-21 (unaudited) Cash Runway Sufficient into 2Q 2023 Guidan
Slide 48: Ocuphire Pharma
www.ocuphire.comir@ocuphire.com Ocuphire Pharma
Slide 49: NVD Endpoint: Low Contrast Visual Acuity (LCVA) 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 50: DR/DME Endpoint: Diabetic Retinopathy Severity Scale (DRSS)
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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