Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Technical Presentation (June 2021)
33 slides from Opus Genetics's technical presentation, Jun 30, 2021
Slide 1: VEGA-1 Phase 2 Topline Results Conference Call
VEGA-1 Phase 2 Topline Results Conference Call June 30, 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: Agenda and Participants
Agenda and Participants Topline VEGA-1 Phase 2 Clinical Trial Results for Nyxol and Low-Dose Pilocarpine in PresbyopiaPresbyopia Market OpportunityFuture MilestonesQ&AParticipantsMina Sooch, MBA, President and CEOMitch Brigell, PhD, Head of Clinical DevelopmentJay Pepose, MD, Medical Advisory Board
Slide 4: 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 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 5: Product Profile: Nyxol + Low-Dose Pilocarpine (LDP) Combo
Product Profile: Nyxol + Low-Dose Pilocarpine (LDP) Combo Moderate Use of Iris Dilator And Iris Sphincter Muscles To Improve Near Vision 0.4% 0.75%Nyxol 0.4% LDP Nyxol LDP P Iris Dilator Muscle Inhibition Iris Sphincter Muscle Activation Active ingredient approved decades ago 505(b)(2)Novel MOA on i
Slide 6: Potential 'Best in Class' Presbyopia Drop
Potential ‘Best in Class’ Presbyopia DropTopline Results From Vega-1 Were Positive… Nyxol + LDP Presbyopia Treatment is Differentiated: Statistically significant efficacy dataFavorable safety profileComfort and tolerabilityFast onsetLong durationMaintain good distance visual acuity (night/day)Novel
Slide 7: Nyxol Phentolamine Mesylate indications
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of Mydriasis 0.4% +
Slide 8: Topline VEGA-1 Phase 2 Results
Topline VEGA-1 Phase 2 Results Randomized, Multi-Center, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) + 0.4% Low Dose Pilocarpine (LDP) for the Treatment of Presbyopia Clinical trial NCT#04675151
Slide 9: Objectives and Key Eligibility Criteria
Objectives and Key Eligibility Criteria Key ObjectivesPRIMARYTo evaluate the efficacy of Nyxol + LDP to improve DCNVA compared to Placebo alone in presbyopia subjectsKEY SECONDARYTo evaluate the ocular and systemic safety of Nyxol + LDP and each component individuallyTo evaluate multiple secondary v
Slide 10: Presbyopia VEGA-1 Phase 2 Design
Presbyopia VEGA-1 Phase 2 Design Clinical trial NCT#04675151 Randomized, Double-Masked, Placebo-Controlled One-Week Trial Primary: % of subjects with ≥ 3 lines of improvement in distance-corrected near visual acuity comparing Nyxol + LDP vs placebo alone at 1 hourSecondary:% of subjects with ≥ 2 and
Slide 11: Patient Population – Subject Disposition
Patient Population – Subject Disposition 148/150 subjects completed the study (mITT)Only a single subject difference between mITT and PP populationPer Statistical Analysis Plan, all analyses performed on PP population with results being nearly identical for mITT Source: VEGA-1 TLR Table 14.1.1 Subje
Slide 12: Demographics (PP Population)
Demographics (PP Population) Treatment And Placebo Arms Were Balanced In This Phase 2 Clinical Trial Source: VEGA-1 TLR Table 14.1.2.2 Demographics and Baseline Characteristics (PP Population) P Placebo Alone N=43 Nyxol Alone N=30 LDP Alone N=31 Nyxol+LDP N=43 Total N=147 Age (years): Median (Range)
Slide 13: Baseline Characteristics Study Eye
Baseline Characteristics Study Eye (PP Population) Placebo Alone N=43 Nyxol AloneN=30 LDP Alone N=31 Nyxol+LDPN=43 TotalN=147 Baseline Characteristic Photopic DCNVA Mean Letters read-Binocular (Snellen Equiv.)70 letters = 20/20 46 (20/63) 45 (20/63) 48(20/63) 46 (20/63) 46 (20/63) Photopic BCDVA Mea
Slide 14: Primary Endpoint: % of Subjects ≥15 Letter Gain in Photopic DCNVA at 1 Hour
Primary Endpoint: % of Subjects ≥ 15 Letter Gain in Photopic DCNVA at 1 Hour Primary Endpoint Was Met For Nyxol + LDP Gaining ≥ 15 Letters Near Vision In PP Population VEGA-1 Phase 2 Trial P Source: VEGA-1 TLR Table 14.2.1.1 (mITT) and 14.2.1.2 (PP)Percent of Subjects With Improvement From Baseline
Slide 15: Secondary Endpoint: % of Subjects ≥10 Letter Gain at 1 Hour
Secondary Endpoint: % of Subjects ≥ 10 Letter Gain In Photopic DCNVA At 1 Hour Source: VEGA-1 TLR Table 14.2.1.1 (mITT) and 14.2.1.2 (PP) Percent of Subjects With Improvement From Baseline in Photopic DCNVA by Time Point. 10 letters is 2 lines. Many Subjects Treated With Nyxol + LDP Gained A Clinica
Slide 16: Secondary Endpoint: % of Subjects ≥15 Letter Gain At All Timepoints
Secondary Endpoint: % of Subjects ≥ 15 Letter 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 Gain From 30 Min To 6 Hou
Slide 17: Monocular Results for Study Eye and Fellow Eye
Source: VEGA-1 TLR Table 14.2.1.2 Percent of Subjects With Improvement From Baseline in Photopic DCNVA by Time Point (PP Population) Similar Results Were Seen Monocularly For Study Eye And Fellow Eye On Primary Endpoint Secondary Endpoint: % of Subjects ≥ 15 Letter Gain DCNVA (Monocular) P VEGA-1 Ph
Slide 18: Near Letter Gain and Distance Letter 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 Als
Slide 19: Change in Photopic and Mesopic BCDVA at 1 Hour
Change in Photopic and Mesopic BCDVA at the 1-Hour Timepoint Source: VEGA-1 TLR Table 14.2.8.1 and 14.2.10.1 Percent of Subjects With Improvement or Loss From Baseline in Photopic and Mesopic BCDVA by Time Point (PP) Treatment With Nyxol And/Or LDP Did Not Reduce BCDVA And Had A Modest Beneficial Ef
Slide 20: 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 P Statistics Compared to Nyxo
Slide 21: 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 22: Tolerability: Conjunctival Hyperemia (Redness) Score
Tolerability: Conjunctival Hyperemia (Redness) Score Source: VEGA-1 TLR Table 14.3.3.2 Continuous Summary of Conjunctival Hyperemia by Time Point (Safety Population) Minor Change (0.5 Point) In Redness Score Over The First 2 Hours In LDP Arms P Conjunctival Hyperemia CCLRU Scale for Reference
Slide 23: Summary of Positive VEGA-1 Phase 2 Results
Summary of Positive VEGA-1 Phase 2 Results for Nyxol Eye Drops Met the primary endpoint with statistical significance for binocular photopic near vision at 1 hour 61% Nyxol + LDP gained 15 letters (3 lines) or more vs. 28% Placebo (33% Placebo Adjusted)Met the Phase 3 co-primary endpoint vs. placebo
Slide 24: Next Steps
Next Steps VEGA-1 Presbyopia Presentation by Dr. Pepose at ASCRS on Sunday July 25, 2021 at 8:45amASCRS Paper ID 76645 SPS-204 Presbyopia Correcting IOL Comparisons, New Treatments and StudiesMBCR - Level 2, Lagoon EFMIRA-2 Reversal of Mydriasis Presentation by Dr. Pepose at ASCRS on Monday July 26,
Slide 25: Presbyopia Market Opportunity
Presbyopia Market Opportunity
Slide 26: 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 27: 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 28: Synergistic Effects of Nyxol + Low-Dose Pilocarpine Combo
Synergistic Effects of Nyxol + Low-Dose Pilocarpine (LDP) Combo Source: Ocuphire Clinical Trials Nyxol + LDP Demonstrated Efficacy and a Favorable Safety Profile in VEGA-1 Trial Average PD in photopic conditions is 3.5 to 4.5 mm 1.5 to 2.5 mm PD reductionmoves toward the pin-hole (1.6 to 2.5 mm, up
Slide 29: 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 30: Potential 'Best in Class' Presbyopia Drop
Potential ‘Best in Class’ Presbyopia DropCompetitive Approaches Limited by Safety/Tolerability, Durability, and Poor Distance Night Vision Nyxol + LDP Presbyopia Treatment is Differentiated: Statistically significant efficacy dataFavorable safety profileComfort and tolerabilityFast onsetLong duratio
Slide 31: Future Milestones
31 HARMA Future Milestones
Slide 32: 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 33: Q&A
www.ocuphire.comir@ocuphire.com Q&A