Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Day (January 2022)
114 slides from Opus Genetics's investor day presentation, Jan 31, 2022
Slide 1: Ocuphire Pharma Investor R&D Day January 31, 2022
Ocuphire Pharma Investor R&D Day January 31, 2022
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 the regulatory timelines, commercial timelines, cash r
Slide 3: Agenda & Speakers
Ocuphire Investor R&D Day 1/31/22 Agenda & Speakers
Slide 4: Company Overview
Company Overview Presenter: Mina Sooch, CEO, Founder of Ocuphire Pharma
Slide 5: Ocuphire Pharma: Restoring Vision and Clarity for Your Eyes
Ocuphire Pharma Restoring Vision and Clarity for Your Eyes, Today and Tomorrow
Slide 6: Ophthalmology – An Attractive Biotech Sector
Ophthalmology – An Attractive Biotech Sector Demographics, M&A, Regulatory Approvals and Efficient Trials Favor Ophthalmic Drugs 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
Slide 7: Nyxol & APX3330: Drug Development History and Patents
Nyxol & APX3330: Drug Development History and Patents Source: Eisai and Apexian Data Significant Preclinical & Clinical Data Supporting Human Safety, MOA, Efficacy, and PK
Slide 8: Ocuphire Pipeline & Clinical Milestones
Ocuphire Pipeline & Clinical Milestones Multiple Phase 3 & Phase 2 Clinical Data Readouts Anticipated Over the Next Year
Slide 9: Differentiated, Late-Stage Pipeline for Front and Back of the Eye
Differentiated, Late-Stage Pipeline for Front and Back of the Eye Nyxol 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 promising clinical data and favorable safety pr
Slide 10: Associates
00 vote Associates Medical Group O) ¢ David Boyer, MD Chicago Medical School EJ Cleveland Clinic Cole Eye Institute aE Mark Kelley, PhD Founder Apexian/APX3330 Peter Kaiser, MD Harvard Medical School 10
Slide 11: APX3330 Chemistry and MOA
00 1 APX3330 Chemistry and M Presented by: Mark Kelley, PhD * Chief Scientific Officer and Founder of Apexian Pharmaceuticals ANA UNIVERSITY Discovered and has developed the redox-specific inhibitors of Ref- ome 1 for over 20 years Mark Kelley, PhD + Associate Director of Basic Science at Indiana Un
Slide 12: 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
Slide 13: References
Tao Yan et al. APX3330 Promotes Neurorestorative effects after stroke in type one diabetic rats. Aging and Disease. Vol 9, Oct 2018 -Apurinic/Apyrimidinic endonuclease 1 regulates inflammatory response in macrophages. Jedinak A, Dudhgaonkar S, Kelley MR, Sliva D. Anticancer Res. 2011 Feb;31(2):379-8
Slide 14: APX3330 VEGF Effects in Normal Cells
APX3330 VEGF Effects in Normal Cells Kamba 2007; Girardi 2010; Li 2014; APX3330 Investigator Brochure APX3330 Restores Normal Levels Unlike Biologic Anti-VEGFs that Reduce VEGF Below Normal
Slide 15: APX3330 Preclinical & IND-Enabling Studies
APX3330 Preclinical & IND-Enabling Studies APX3330 Investigator Brochure Extensively Evaluated in Over 20 Studies by Large Japanese Pharma Eisai
Slide 16: Preclinical Data: Oral APX3330 Blocks Neovascularization
Preclinical Data: Oral APX3330 Blocks Neovascularization Silva et al. ARVO 2021 Annual Meeting *Published data on EYLEA. This study was performed independently from APX3330 study and is a cross-study comparison. **Li 2014; *** Pasha 2018; ****Jiang 2011 (Vldlr -/- : Very Low-Density Lipoprotein rece
Slide 17: APX3330 Human PK and Safety Summary
APX3330 Human PK and Safety Summary
Slide 18: Phase 1 Clinical Trials: PK Data Supporting the ZETA-1 Trial
Phase 1 Clinical Trials: PK Data Supporting the ZETA-1 Trial Apexian preclinical data (unpublished) APX3330 Investigator Brochure Eisai PK clinical data APX_CLN_0002 APX3330 has Oral Bioavailability and a Sustained PK Profile
Slide 19: APX3330 Reaches the Retina via Oral Administration
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 Eisai Preclinical Data Apexian preclinical data APX3330 Reaches t
Slide 20: Subject Exposure Across Prior Clinical Trials
Subject Exposure Across 11 Prior Clinical Trials * Eisai and Apexian Phase 1 and Phase 2 clinical trials (subject to final safety database) Over 2000 Subject-Days of Exposure at ≥600 mg/day
Slide 21: Safety Summary From Phase 1 and Phase 2 Studies
Safety Summary From Phase 1 and Phase 2 Studies Phase 1 and Phase 2 Clinical Trials performed by Eisai and Apexian
Slide 22: APX3330 Product Candidate Profile for Multiple Retinal Indications
APX3330 Product Candidate Profile for Multiple Retinal Indications Phase 1 and Phase 2 clinical trials by Eisai and Apexian First-In-Class Ref-1 Inhibitor with Favorable Human Safety Data
Slide 23: APX3330 Addressing Unmet Needs in Retina
APX3330 Addressing Unmet Needs in Retina
Slide 24: Clinical Unmet Need in Diabetic Retinal Diseases
Clinical Unmet Need in Diabetic Retinal Diseases DR/DME are major causes of vision loss in working aged adults Diabetic population expected to increase dramatically worldwide Approved therapies for DR are effective but require IVT injection DR patients are not routinely treated with approved injecta
Slide 25: Diabetic Retinopathy is a Progressive, Vision Threatening Disease
Diabetic Retinopathy is a Progressive, Vision Threatening Disease Source: Patient survey adapted from Lions International Foundation and International Diabetes Foundation-Europe; Meltzer 2000 Losing Vision is Diabetic Patients Top Concern
Slide 26: Key Clinical Landscape in Diabetic Retinopathy (and DME)
Key Clinical Landscape in Diabetic Retinopathy (and DME)
Slide 27: ZETA-1 Phase 2b Clinical Trial (APX3330 in DR)
ZETA-1 Phase 2b Clinical Trial (APX3330 in DR)
Slide 28: 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) https://clinicaltrials.gov/ct2/show/NCT04692688?term=ZETA
Slide 29: Key Eligibility Criteria in ZETA-1
Key Eligibility Criteria in ZETA-1 INCLUSION Moderately-severe to severe NPDR or mild PDR in study eye as confirmed by reading center BCVA > 20/63 in study eye EXCLUSION Retinopathy from causes other than diabetes in study eye Presence of center involved diabetic macular edema (DME) defined as a cen
Slide 30: Why DRSS is an Important Endpoint?
Why DRSS is an Important Endpoint? Eylea® Panorama study FDA Accepted Endpoint for EYLEA® in PANORAMA Pivotal DR Trial - 2 Step Improvement on the DRSS Score at Week 24
Slide 31: Enrollment Update on ZETA-1
Enrollment Update on ZETA-1 ZETA-1 Clinical Trial ~70% Completion of Enrollment in 24-week Phase 2b Trial
Slide 32: Comparison of Subject Exposure Before and After ZETA-1
Comparison of Subject Exposure Before and After ZETA-1 *Interim Zeta-1 Data as of 01-12-22; * Eisai and Apexian Phase 1 and Phase 2 clinical trials (subject to final safety database) Subject-Days of Exposure at 600 mg/day Substantially Increases Exposure Data
Slide 33: Baseline Characteristics for ZETA-1 Trial (Interim)
Baseline Characteristics for ZETA-1 Trial (Interim) Source: ZETA-1 Demographics and Baseline Characteristics Typical Demographics for Diabetic Population
Slide 34: Masked Safety Findings from Ongoing ZETA-1 Trial
Masked Safety Findings from Ongoing ZETA-1 Trial Source: ZETA-1 trial Favorable Safety Profile (as of 1/12/2022) Observed with 600 mg Oral Daily Doses
Slide 35: Takeaways: Masked Interim Safety Findings from ZETA-1 Trial
Takeaways: Masked Interim Safety Findings from ZETA-1 Trial ZETA-1 Clinical Trial
Slide 36: Broad Opportunities to Treat Retinal Diseases with APX Platform
Broad Opportunities to Treat Retinal Diseases with APX Platform Potential First Oral Rx for Retina Diseases First-line earlier intervention for the diabetic eye Add-on therapy to current anti-VEGF treatments to reduce intravitreal injection burden Proven Novel Mechanism May decrease both inflammatio
Slide 37: Large Global/US Market Opportunity in Retinal Disease
Large Global/US Market Opportunity in Retinal Disease Market Scope 2020 J Glob Health. 2019 Jun; 9(1): 010427 Tilahun M et. al, Prevalence of Diabetic Retinopathy and Its Associated Factors among Diabetic Patients at Debre Markos Referral Hospital, Northwest Ethiopia, 2019: Hospital-Based Cross-Sect
Slide 38: What’s Important?
What’s Important? Eylea® label; APX3330 Investigator Brochure, ZETA-1 clinical trial APX3330 has the Potential to be 1st Line of Therapy for DR Patients
Slide 39: APX3330 for Diabetic Retinopathy: Question & Answer
APX3330 FOR DIABETIC RETINOPATHY . Question & Answer
Slide 40: Summary of APX3330 Program
J Summary of APX3330 Program 40 APX3330 is a novel orally administered drug initially being developed for DR/DME APX3330 targets Ref-1 which plays a role in signaling under both ischemic and inflammatory conditions, both of which are relevant to diabetic eye disease; resulting in inhibiting clinical
Slide 41: II. Nyxol Reversal of Mydriasis Overview
II. Nyxol Reversal of Mydriasis Overview
Slide 42: Reversing Dilations: Addressing an Unmet Need with α1 Blocker Nyxol
Reversing Dilations: Addressing an Unmet Need with α1 Blocker Nyxol
Slide 43: Problem: Dilated Eyes for Exams and Procedures
Problem: Dilated Eyes for Exams and Procedures Pharmacologically-induced pupil dilation is a necessary tool for routine ophthalmoscopy… …but there is 6 to 24 hours of impaired vision including: Inability to Focus Photophobia (sensitivity to light) Cycloplegia (loss of accommodation) Difficulty Readi
Slide 44: Current Off-Label Landscape for RM
Current Off-Label Landscape for RM 1. Optician (2012)- Mydriatic Drugs: Practical Considerations 2. Pilocarpine FDA Label (2017) 3. Lee DA, Higginbotham EJ, 2005. Glaucoma and its treatment: a review. Am J Health Syst Pharm 62, 691–699. Physicians AVOID Use of Cholinergic Agonists (Pilocarpine) Due
Slide 45: Nyxol’s Differentiated MOA as an Alpha-1 Blocker
Nyxol’s Differentiated MOA as an Alpha-1 Blocker Ocuphire Phentolamine Mesylate Reformulated as a Proprietary Topical Eye Drop Nyxol
Slide 46: Nyxol Product Candidate Profile
Nyxol Product Candidate Profile Nyxol Clinical Trials Novel, Differentiated Alpha 1/2 Blocker Eye Drop for Refractive Indications
Slide 47: Nyxol Clinical Data for Reversing Dilations
Nyxol Clinical Data for Reversing Dilations MIRA Program Evaluating Nyxol for the Reversal of Mydriasis Ocuphire Efficient Clinical Programs have Positioned Ocuphire to Target NDA Filing in Late 2022
Slide 48: MIRA-2/3 Phase 3 Registration Trial Design
MIRA-2/3 Phase 3 Registration Trial Design Mydriatic Agents 3:1:1 – 2.5% phenylephrine (alpha-1 agonist), 1% tropicamide (cholinergic blocker), Paremyd® (combination) Randomized, Double-Masked, Placebo-Controlled, Parallel, One-Day Trial
Slide 49: MIRA-2: Participant Characteristics
MIRA-2: Participant Characteristics Source: MIRA-2 TLR table #14.1.2.3 (mITT) MIRA-2 Study was Balanced Across Both Nyxol and Placebo Groups
Slide 50: MIRA-2: Phase 3 RM Trial Met Primary Endpoint
MIRA-2: Phase 3 RM Trial Met Primary Endpoint Source: MIRA-2 Trial Table 14.1.2.1, mITT Population (same as Safety Population) *Data includes three of the most common mydriatics used in practice (Phenylephrine, Tropicamide, Paremyd) 49% of Patients Returned to < 0.2mm of Baseline at 90 Minutes
Slide 51: MIRA-2: Study Eye and Non-Study Eye
MIRA-2: Study Eye and Non-Study Eye Source: MIRA-2 Trial Table 14.1.2.1, mITT Population (same as Safety Population) *Data includes three of the most common mydriatics used in practice (Phenylephrine, Tropicamide, Paremyd) Similar Rapid Return to Baseline Pupil Size Results with 1 or 2 Drops of Nyxo
Slide 52: MIRA-2: Responders Returning to Baseline Pupil Size by Iris Color
MIRA-2: Responders Returning to Baseline Pupil Size by Iris Color Source: MIRA-2 TLR table #14.2.1.6 (mITT) *Data includes three of the most common mydriatics used in practice (Phenylephrine, Tropicamide, Paremyd) Nyxol Works in Subjects with Both Light and Dark Irides, with a More Vigorous Response
Slide 53: MIRA-2: Responders Returning to Baseline Pupil Size by Iris Color
MIRA-2: Responders Returning to Baseline Pupil Size by Iris Color Source: MIRA-2 TLR table #14.2.1.6 (mITT) *Data includes three of the most common mydriatics used in practice (Phenylephrine, Tropicamide, Paremyd) Nyxol Works in Subjects with Both Light and Dark Irides, with a More Vigorous Response
Slide 54: MIRA-2: Mean Pupil Size Over Time After Maximum Pupil Dilation
MIRA-2: Mean Pupil Size Over Time After Maximum Pupil Dilation Source: MIRA-2 TLR table #14.2.2.1 (mITT). Standard Error bars are shown. *Data includes three of the most common mydriatics used in practice (Phenylephrine, Tropicamide, Paremyd) Nyxol Treatment Significantly Reduced PD Starting at 1 Ho
Slide 55: MIRA-2: Mean Pupil Diameter Over Time by Mydriatic Agent
MIRA-2: Mean Pupil Diameter Over Time by Mydriatic Agent Source: MIRA-2 TLR table #14.2.2.3 (mITT). Standard Error bars are shown. Nyxol Reduced Pupil Diameter with All Mydriatic Agents; More Rapidly with Phenylephrine as Expected
Slide 56: MIRA-2: Gain of Visual Function (Accommodation)
MIRA-2: Gain of Visual Function (Accommodation) Source: MIRA-2 CSR table #14.2.3.2.1. PP population is the per protocol population. Patel (2011). Pseudoaccommodation. International Ophthalmology Clinics, 51(2), 109–118. Nyxol Demonstrates a Faster Return to Baseline Accommodation
Slide 57: MIRA-2: Safety Findings
MIRA-2: Safety Findings (After Dilation with Mydriatic Agent) Source MIRA-2 Safety Population TLR table 14.3.1.1.; MIRA-2 table 14.3.1.2.2 System Organ Class; MIRA-2 table 14.3.3.2 Hyperemia Score by Time Point Nyxol was Well Tolerated with a Favorable Safety Profile
Slide 58: 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
Slide 59: What’s Important?
What’s Important? Nyxol has the Potential to be the only FDA-Approved Treatment Option to Reverse Dilation
Slide 60: RM Market Opportunity and Commercialization
RM Market Opportunity and Commercialization Patients are Vocal About the Negative Effects of Mydriasis Quotes are from anonymous patients
Slide 61: Patients are Vocal About the Negative Effects of Mydriasis
Patients are Vocal About the Negative Effects of Mydriasis Quotes are from anonymous patients
Slide 62: Importance of Dilations
Importance of Dilations Source: American Academy of Ophthalmology, Comprehensive Adult Medical Eye Evaluation Preferred Practice Pattern, 2020 Dilated Eye Exam Remains the Recommended Standard of Care
Slide 63: Bottom-Up Calculation of Annual Dilated Eye Exams
Bottom-Up Calculation of Annual Dilated Eye Exams *IQVIA 2020 sales data; KOL Interview; GlobalData market research; and AOA Excel and Jobson Medical Information ‘Bottom-Up Calculation’ assumes 48 total work weeks in a year Supply side validation assumed each unit (bottle) has ~10 mL fill volume and
Slide 64: Market Research Methodology by GlobalData
Market Research Methodology by GlobalData GlobalData Market Research Survey Market Research Conducted in 2H 2020 for RM, Presbyopia, and Night Vision Disturbances
Slide 65: Reversal of Mydriasis (RM) Market Opportunity
Reversal of Mydriasis (RM) Market Opportunity With No Commercially Available Treatment, Nyxol may Achieve Significant Revenue Potential
Slide 66: Why 1990’s Rev-Eyes is Not a Benchmark for Nyxol Future RM Sales
Why 1990’ Rev-Eyes is Not a Benchmark for Nyxol Future RM Sales Rev-Eye label; Nyxol clinical trials Nyxol’s Broad Differentiation Addresses the Unmet Need for Reversal Drops in New Era
Slide 67: RM Market Opportunity and Commercialization
RM Market Opportunity and Commercialization
Slide 68: Perspective from Practice Administrators on Reversing Dilations
Perspective from Practice Administrators on Reversing Dilations Anterior Segment Practice in Southeast “We’ve explored and offered several options over the years to reverse mydriasis – both as a tool to elevate the patient experience and to reduce liability when a patient with poor accommodation wal
Slide 69: Pre-Commercial Activities in 2022
Pre-Commercial Activities in 2022 Activities Underway to Support Capital-Efficient Nyxol RM Commercial Launch
Slide 70: Go-To-Market Strategy for 2023
Go-To-Market Strategy for 2023 Nyxol as RM Allows Efficient Pharmaceutical Launch Across Eye Care Practices
Slide 71: Practice Implementation Models
Practice Implementation Models Physician Interviews Conducted by Ocuphire Positive Feedback from Physicians on Integration of a Reversal Agent into Practice
Slide 72: Nyxol for Reversal of Mydriasis (RM): Question & Answer
Nyxol® for Reversal of Mydriasis (RM)
Slide 73: Summary of Nyxol Reversal of Mydriasis Program
Summary of Nyxol Reversal of Mydriasis Program
Slide 74: III. Nyxol Presbyopia Program
III. Nyxol Presbyopia Program
Slide 75: Pupil Modulation Eye Drops for Presbyopia
Pupil Modulation Eye Drops for Presbyopia
Slide 76: The Time for Presbyopia Drops
The Time for Presbyopia Drops Sources: Academic review articles, journals, and publications Headlines from Academia and Industry Articles with an Early First Approval for VuityTM
Slide 77: What is the Optimal Pupil Size?
What is the Optimal Pupil Size? “A fixed 2- to 3-mm small pupil or a 30% pupil miosis can both produce near visual acuity gains without significant losses to distance acuity or image quality, and therefore can be considered as optimal for a presbyope experiencing a wide range of light levels.” - Opt
Slide 78: Nyxol with LDP as Adjunctive Therapy in Presbyopia
78 i ie — ©)Ocuphire Nyxol with LDP as Adjunctive Therapy in Presbyopia Presented by: Jay Pepose, MD, PhD
Slide 79: Nyxol with Low-Dose Pilocarpine (LDP) as Adjunct Therapy
Nyxol® with Low-Dose Pilocarpine (LDP) as Adjunct Therapy 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
Slide 80: VEGA-1: Presbyopia Phase 2 Trial Design
VEGA-1: Presbyopia Phase 2 Trial Design Clinical trial NCT#04675151. DCNVA = distance-corrected near visual acuity . BCDVA = best corrected distance visual acuity; *3-4 evenings; VEGA-1 Study Design Randomized, Double-Masked, Placebo-Controlled, Multi-Center One-Week Trial
Slide 81: VEGA-1: Demographics and Baseline Characteristics
VEGA-1: Demographics and Baseline Characteristics Source: VEGA-1 TLR Table 14.1.2.2 Demographics and Baseline Characteristics (PP Population). Snellen Conversion Chart. Treatment and Placebo Arms were Balanced in the VEGA-1 Phase 2 Clinical Trial
Slide 82: VEGA-1: Nyxol+LDP Met Primary & Secondary Endpoints
VEGA-1: Nyxol+LDP 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. 61% Patients with Nyxol+LDP had ≥ 15 Letter Near Gain with Fast Onset & Durable Responses
Slide 83: VEGA-1: Planned P3 Efficacy Endpoint Met by Nyxol+LDP
VEGA-1: Planned P3 Efficacy Endpoint Met by Nyxol+LDP 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 84: VEGA-1: Improvement in Functional Near Vision
VEGA-1: Improvement in Functional Near Vision Source: VEGA-1 TLR Table 14.2.24.1 Percent of Subjects with Photopic DCNVA by Time Point (PP Population) Nyxol+LDP had a Rapid Improvement in Near Vision to 20/32 for many Patients
Slide 85: VEGA-1: Mean Pupil Diameter Over Time
VEGA-1: 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 Optimal Pupil Size in Nyxol+LDP (& Nyxol) Consistent with 3-line Near Vision Gain
Slide 86: VEGA-1: Photopic and Mesopic Distance Vision Effects
VEGA-1: Photopic and Mesopic Distance Vision Effects 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) Nyxol and/or LDP Demonstrated No Loss of Distance Vision in Photopic and Mesopic Lighting
Slide 87: VEGA-1: Safety Findings Across All Arms
VEGA-1: Safety Findings Across All Arms 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 Combinat
Slide 88: What’s Important?
What’s Important? Vuity Label 2021 Nyxol+LDP has the Potential to be “Best in Class” Presbyopia Eyedrop
Slide 89: Nyxol Alone in Presbyopia
89 Nyxol Alone in Presbyopia Presented by: James Katz, MD + President of the Midwest Center for Sight the Midwest >) + Board-certified Ophthalmologist with specialties in Conese Cornea, Cataract, and Refractive Surgery James Katz, MD University of Illinois, + Well-Published in Distinguished Ophthalm
Slide 90: VEGA-1 Study Design Assessed Nyxol Alone Efficacy
VEGA-1 Study Design Assessed Nyxol Alone Efficacy *3-4 daily evening doses and then visit 2 on Day 5 Source: VEGA-1 Study Design Nyxol as Single Drop vs. Placebo Met Pre-Specified Endpoints in the Trial
Slide 91: VEGA-1: Nyxol Meets Planned P3 Efficacy Endpoint
VEGA-1: Nyxol Meets Planned P3 Efficacy Endpoint at 12 Hours Source: VEGA-1 TLR Table 14.2.1.7 Percent of Subjects with Improvement in Photopic DCNVA at 0 Minutes for Nyxol and Placebo Treated Subjects (PP Population) Nyxol as a Single Drop Provides a Statistical 3 Line or More Gain Compared to Plac
Slide 92: VEGA-1: >10 Letter Gain in DCNVA with Nyxol
VEGA-1: >10 Letter Gain in DCNVA with Nyxol at 12 Hours Source: VEGA-1 TLR Table 14.2.1.7 Percent of Subjects with Improvement in Photopic DCNVA at 0 Minutes for Nyxol and Placebo Treated Subjects (PP Population) Nyxol as a Single Drop Provides a Clinically Meaningful >10 Letter Gain Compared to Pla
Slide 93: VEGA-1: Improvement in Functional Near Vision
VEGA-1: Improvement in Functional Near Vision Source: VEGA-1 TLR Table 14.2.24.1 Percent of Subjects with Photopic DCNVA by Time Point (PP Population) Nyxol Single Drop Significantly Improves Functional Near Visual Acuity
Slide 94: VEGA-1: Functional Vision Durability
VEGA-1: Functional Vision Durability VEGA-1 TLR Table 14.2.1.2 Percent of Subjects with Photopic DCNVA by Time Point (PP Population) and Table 14.2.24.1 Percent of Subjects with Photopic DCNVA by Time Point (PP Population) Single Drop of Nyxol has Durable Response for 18 Hours
Slide 95: VEGA-1 (Post-Hoc): Efficacy Across Presbyopia Ages
VEGA-1(Post-Hoc): Efficacy Across Presbyopia Ages Source: VEGA-1 TLR Table 14.2.1.7.1 & 14.2.1.7.2 Percent of Subjects with Improvement in Photopic DCNVA by Age Group (PP Population) Nyxol Highest Efficacy in Young Presbyopes as Expected; Also Efficacy Seen in Older Presbyopes
Slide 96: Presbyopia Drops: Ocuphire Next Steps and Competitive Landscape
Presbyopia Drops: Ocuphire Next Steps and Competitive Landscape
Slide 97: Two Treatment Options for Spectrum of Presbyopic Patients
Two Treatment Options for Spectrum of Presbyopic Patients Two NDA Submissions Targeted in 2023: Nyxol Alone and Nyxol+LDP
Slide 98: Potential ‘Best in Class’ Presbyopia Drop(s)
Potential ‘Best in Class’ Presbyopia Drop(s) Placebo Adjusted Values for Vuity were 15-23% in Gemini1 & 2;Placebo Adjusted Nyxol was 16% and Nyxol+LDP was 33% (all stat significant) Source: Nyxol Data: ASCRS (July 2021) Abstract# 76645 (Phase 2) and VEGA-1; Abstract 74336 (Phase 3). VUITYTM Data FDA
Slide 99: Presbyopia Eye Drops Competitive Landscape
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
Slide 100: Large and Growing Presbyopia Market
Large and Growing Presbyopia Market
Slide 101: 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. N
Slide 102: Key Findings from GlobalData Market Research on Presbyopia
Key Findings from GlobalData Market Research on Presbyopia GlobalData Market Research Survey Insights Very Consistent with Other Competitors Market Research Surveys
Slide 103: Presbyopia Market Segments
Presbyopia Market Segments Global Prevalence of Presbyopia, 2018, Fortune Business Insights Reading Glasses Forecast 2016-2027, NEI 2010 data. *GlobalData Market Research 2020 Vitale S. et. Al. JAMA Ophthalmology, 2008, Vision problems, US, Arch. Ophthal, 2014, Vision Monday. Tens of Millions of Lik
Slide 104: Vuity is the First FDA-Approved Eyedrop for Presbyopia
VuityTM is the First FDA-Approved Eyedrop for Presbyopia 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
Slide 105: Nyxol for Presbyopia: Question & Answer
Nyxol® for PRESBYOPIA QUESTION & ANSWER
Slide 106: Summary of Nyxol and Nyxol+LDP Presbyopia Program
Summary of Nyxol and Nyxol+LDP Presbyopia Program
Slide 107: Closing Remarks
Closing Remarks Mina Sooch, CEO, Founder of Ocuphire Pharma
Slide 108: Ocuphire Management Team
Ocuphire Management Team Decades of Biotech and Drug Development Experience
Slide 109: 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
Slide 110: Ocuphire Board of Directors
Ocuphire Board of Directors Seasoned Directors with Decades of Drug Development, M&A/Financings, & Ophthalmology Mina Sooch, MBA Vice-Chair, Board Director President & CEO Sean Ainsworth, MBA| Lead Independent Director, Board Director | HaRvanp ; @ David Geffen » Cam Gallagher, MBA evelaneeecueoe Ne
Slide 111: Thanks To Our Network of Partners
Thanks To Our Network of Partners A Strong Foundation has been Built to Efficiently Grow and Deliver Our Vision for Patients
Slide 112: Track Record of Achieving Milestones
Track Record of Achieving Milestones Exciting 2022 News Cadence
Slide 113: Overall Highlights from Ocuphire Investor R&D Day
Overall Highlights from Ocuphire Investor R&D Day Nyxol, if approved in 2023, would be the only Rx drop for reversing dilations and positively impact the patient experience in an eye care practice Nyxol® eye drops, as a platform, is uniquely positioned to address growing markets in refractive disord
Slide 114: Ocuphire Pharma Investor R&D Day January 31, 2022
Ocuphire Pharma Investor R&D Day January 31, 2022