Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (May 2022)
59 slides from Opus Genetics's investor presentation, May 19, 2022
Slide 1: Ocuphire Corporate Presentation
Ocuphire Corporate Presentation May 19, 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, produc
Slide 3: Differentiated, Late-Stage Pipeline for Front and Back of the Eye
Differentiated, Late-Stage Pipeline for Front and Back of the Eye Nyxol with > 650 patients treated across 12 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 p
Slide 4: Ophthalmology – An Attractive Biotech Sector
Ophthalmology – An Attractive Biotech Sector Favorable Regulatory Environment Source:1. Endpoint Dec 29, 2021- Hitting a new record on drug approvals, the FDA offers a thumbs-up to another atopic dermatitis contender; 2. OIS Year in Review 2021; 3. Company press releases Demographics, M&A, Regulator
Slide 5: Pipeline by Indication and Anticipated Milestones
Indication Product Candidate Pre-clinical Phase 1 Phase 2 Phase 3 Regulatory Approval Anticipated Milestones Reversal of Mydriasis (RM) Nyxol® Eye Drop Reported positive MIRA-3 Phase 3 data in Q1 2022 (n=368) Reported positive MIRA-4 Pediatric data in 2Q 2022 (n=23) File Nyxol NDA for RM in late 202
Slide 6: Nyxol Eye Drops: RM, Presbyopia, NVD
6 Reversal of Mydriasis P NVD Presbyopia Night Vision Disturbance Nyxol® Eye Drops Nyxol as a Single Drop for Presbyopia Nyxol with LDP Adjunctive Therapy 1 0.4% 2 RM
Slide 7: Nyxol's Differentiated MOA as an Alpha-1 Blocker
Nyxol’s Differentiated MOA as an Alpha-1 Blocker Phentolamine Mesylate Reformulated as a Proprietary Topical Eye Drop Nyxol® Phentolamine Mesylate is the Active Ingredient in Nyxol: a Non-selective α1 & α2 Antagonist Blocking α1 Reduces Pupil Size Blocking α1 Dilates Blood Vessels Phentolamine mes
Slide 8: Nyxol Product Candidate Profile
Nyxol Improves Vision by Decreasing Pupil (~1-1.5mm) ↑ Near Vision ↑ Distance Vision ↑ Contrast Sensitivity (night) No Systemic Effects No Changes in Blood Pressure No Changes in Heart Rate Well-Tolerated Topical Effects Mild, Transient, Reversible Eye Redness IOP Unchanged or Decreased Minimal to N
Slide 9: Nyxol for Reversal of Mydriasis (RM)
9 I have to visit my retina MD for my monthly injections, where I am dilated. Being dilated every month is a huge burden on my day. I had a premium cataract procedure by my MD, and I was unable to see clearly for two days. My doctor said it was due to my dilation. I did not expect my dilation to las
Slide 10: Problem: Dilated Eyes for Exams and Procedures
Problem: Dilated Eyes for Exams and Procedures Pharmacologically-induced pupil dilation is part of standard care for annual and specialty eye exams… …but there is 6 to 24 hours of impaired vision including: Inability to Focus Photophobia (sensitivity to light) Cycloplegia (loss of accommodation)
Slide 11: Nyxol Has Potential To Be The Only Option For RM
Nyxol Has Potential To Be The Only Option For RM Physicians AVOID Use of Cholinergic Agonists (Pilocarpine) Due to Safety Risk on Ciliary Muscle 1 Pilocarpine FDA Label (2017)2. Optician (2012)- Mydriatic Drugs: Practical Considerations 3. Lee DA, Higginbotham EJ, 2005. Glaucoma and its treatment: a
Slide 12: Reversal of Mydriasis Unmet Need & Landscape
Reversal of Mydriasis Unmet Need & Landscape Source – Optos plc Pricing With No Commercially Available Treatment, Nyxol is Uniquely Positioned as a New Reversal Drop At many annual eye exams and specialty visits, pupils are pharmacologically dilated, impairing vision for 6-24 hours Dilated eyes expe
Slide 13: MIRA-3 Phase 3 Registration Trial Design
MIRA-3 Phase 3 Registration Trial Design Randomized, Double-Masked, Placebo-Controlled, Parallel, Multi-Center, One-Day Trial Mydriasis -1 Hour 1:1 Mydriatic Agent A, B, or C 0.75% Nyxol Placebo 16 US sites 368 subjects Nyxol drop(s) (2 drops study eye, 1 drop fellow eye) Mydriatic Agent A, B, or C
Slide 14: Primary Endpoint Achieved in Two FDA Registration Phase 3 Trials
Primary Endpoint Achieved in Two FDA Registration Phase 3 Trials Source: (Left panel) MIRA-3 Table 14.2.1.1 (mITT); (Right panel) MIRA-2 Table 14.2.1.1 (mITT). Data include all three mydriatics (Phenylephrine, Tropicamide, Paremyd). Rapid, Consistent and Sustained Reversal of Pupil Dilation with Nyx
Slide 15: MIRA-3: Mean Pupil Diameter Over Time
MIRA-3: Mean Pupil Diameter Over Time Source: MIRA-3 Table 14.2.2.1 (mITT). The p-values are change from max pupil dilation treatment compared to placebo. Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd). Standard Error bars are shown. Nyxol Treatment Significantly Reduced PD
Slide 16: Summary of MIRA Registration Trial Designs
Summary of MIRA Registration Trial Designs Source: In order from left to right MIRA-2 Trial NCT# 04620213 MIRA-3 Trial NCT# 05134974 Randomized, Double-Masked, Placebo-Controlled, Parallel, Multi-Center, One-Day Trials MIRA-2 Phase 3 MIRA-3 2nd Phase 3 Number of US Sites 12 16 Subjects Enrolled 185
Slide 17: Summary of Three Positive Late-Stage MIRA Clinical Trials
Summary of Three Positive Late-Stage MIRA Clinical Trials Source: mITT Population, MIRA-2, MIRA-3 and MIRA-4 Trial Confirms Phase 3 Trials with Favorable Safety and Tolerability Profile and Rapid Mydriasis Reversal No deaths, serious AEs, or withdrawals due to AEs All treatment related AEs were mild
Slide 18: NDA Submission Targeted in Late 2022
NDA Submission Targeted in Late 2022 Potential Regulatory Approval in 2023 Completed trial with 23 subjects ages 3 to 11 per agreed FDA initial pediatric study plan Pediatric Safety Completed 2nd Phase 3 trial in RM (enrolled 368 subjects), which also meets 24-hour safety population exposure require
Slide 19: ~$500+M Estimated US RM Market Opportunity
~$500+M Estimated US RM Market Opportunity Reversal of Mydriasis (RM) Market Opportunity With No Commercially Available Treatment, Nyxol May Achieve Significant Revenue Potential Patient Willingness to Pay $10 - $20+ 65% Report Moderate to Severe Impact to Daily Function 100M Annual Eye Dilations 80
Slide 20: More Efficient Launch Opportunity for Nyxol in RM
More Efficient Launch Opportunity for Nyxol in RM Launch is Poised to be Disruptive, Cost-Effective and Not Payor-Driven Traditional Ophthalmic Launch Traditional Ophthalmic Launch Highly competitive markets (e.g., dry eye, glaucoma, allergy); little differentiation Launch success takes time given p
Slide 21: Pre-Commercial 2022 & Go-To-Market Strategy 2023
Pre-Commercial 2022 & Go-To-Market Strategy 2023 Sources: ASRS; AMA; AAO; Women in Optometry (WO); AOA Excel and Jobson Medical Information; Physician Interviews Conducted by Ocuphire; GlobalData market research Activities Underway to Support Capital-Efficient Nyxol RM Commercial Launch Physician Ta
Slide 22: Nyxol for Presbyopia
P 22 “By age 45, 80% of Americans will struggle with Presbyopia, and by age 50, nearly everyone will.” NY Times Effectively everyone over 40 will have the problems with reading. Physician KOL Nyxol® forPRESBYOPIA
Slide 23: 2021: The Time for Presbyopia Drops
2021: The Time for Presbyopia Drops Sources: Academic review articles, journals, and publications between July 2021 to December 2021 Headlines from Academia and Industry Articles with an Early First Approval for VuityTM “The correction of presbyopia remains ophthalmology’s ‘Holy Grail’…” -OIS 10/29/
Slide 24: Nyxol and Nyxol + Low Dose Pilocarpine Presbyopia Eye Drops
Nyxol® and Nyxol + Low Dose Pilocarpine Presbyopia Eye Drops Source: Nyxol® data from 12 completed trials; Pilocarpine product label and literature Differentiated MOA with Two Potential Product Labels for Functional Near Vision Improvement 0.4% 0.75% Nyxol 0.4% LDP Iris Dilator Muscle Inhibition Iri
Slide 25: Presbyopia VEGA-1 Phase 2 Trial
Presbyopia VEGA-1 Phase 2 Trial Clinical trial NCT#04675151. DCNVA = distance-corrected near visual acuity . BCDVA = best corrected distance visual acuity Completed Randomized, Double-Masked, Placebo-Controlled, Multi-Center One-Week Trial Primary: % of subjects with ≥ 3 lines of improvement in dist
Slide 26: VEGA-1: Nyxol+LDP Met Primary & Secondary Endpoints
VEGA-1: Nyxol+LDP Met Primary & Secondary Endpoints VEGA-1 TLR Table 14.2.1.2 60% Patients with Nyxol+LDP had ≥ 15 Letter Near Gain with Fast Onset & Durable Responses VEGA-1 Phase 2 Trial Note: PP population differs from mITT by only one subject; results were essentially identical. P p=0.09 p≤0.000
Slide 27: VEGA-1: Planned P3 Efficacy Endpoint Met by Nyxol and Nyxol+LDP
VEGA-1: Planned P3 Efficacy Endpoint Met by Nyxol and Nyxol+LDP VEGA-1 TLR Table 14.2.2.2.1; Table 14.2.2.2; Table 14.2.1.7; Table 14.2.1.2 Nyxol Single Drop and LDP Combination Provide Statistically Significant 3-line Near Vision Gain Nyxol as a Single Drop for Presbyopia Nyxol with LDP Adjunctive
Slide 28: VEGA-1: Improvement in Functional Near Vision
VEGA-1: Improvement in Functional Near Vision Similar trend was seen at all timepoints Baseline Inclusion: Photopic DCNVA of 20/50 or worse Source: VEGA-1 TLR Table 14.2.24.1 Percent of Subjects with Photopic DCNVA by Time Point (PP Population) Nyxol and Nyxol with LDP Both Provide Durable Improveme
Slide 29: 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 30: 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 and Nyxol Alone Consistent with Near Vision Gains VEGA-1 Phase 2 Trial Daily Evening Nyx
Slide 31: Summary Of Positive VEGA-1 Phase 2 Results
Met planned P3 endpoint at 12 hours (29%; p=0.02) Efficacy Met primary endpoint at 1 hour (60%; p=0.004) Durable near vision gain at 12 and 18 hours Durability Durable near vision gain through at least 6 hours 56% (12 hours post-dose) Functional Vision (20/40 or better) 84% (1-hour post-dose) Sustai
Slide 32: Potential 'Best in Class' Presbyopia Drop(s)
Potential ‘Best in Class’ Presbyopia Drop(s) Product Attributes* 1) Efficacy (> 3-Line Gain w/o loss of 1 line in DCNVA - Primary Endpoint Responders)* 2) Safety: Loss of Distance in Mesopic 3) Tolerability: Headaches and Conjunctival Hyperemia 4) Durability (% responders at the longest timepoint) P
Slide 33: 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
Slide 34: Presbyopia is a Burgeoning Market Opportunity
Presbyopia is a Burgeoning Market Opportunity Source: 1. Global Prevalence of Presbyopia, 2018, Fortune Business Insights Reading Glasses Forecast 2016-2027, Cataract & Refractive Surgery Today, 2021, NEI 2010 data.2. Vitale S. et. Al. JAMA Ophthalmology, 2008, Vision problems, US, Arch. Ophthal, 20
Slide 35: 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 35 NVD Nyxol® for Dim Light or Night Vision Disturbances
Slide 36: Market Opportunity in Dim Light or Night Vision Disturbances
Market Opportunity in Dim Light or Night Vision Disturbances GlobalData Market Research Report, 2020; Photos are illustrative No Approved Treatments with Ripe Opportunity for Growth NVD No Approved Treatments $2B - $4B Estimated US NVD Market Opportunity Peripheral imperfections scatter light when p
Slide 37: NVD LYNX-1 Phase 3 Registration Design
NVD LYNX-1 Phase 3 Registration Design Note: Inclusion Criteria includes subjects with baseline mesopic LCVA of 20/63 or worse 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 visu
Slide 38: LYNX-1: Nyxol Met Primary Endpoint
LYNX-1: Nyxol Met Primary Endpoint Source: LYNX-1 topline data Significantly Higher % of Nyxol Treated Subjects Gained ≥15 Letter and ≥10 Letter From Baseline LYNX-1 Phase 3 Trial n=73 n=70 n=73 n=68 NVD p<0.05 p<0.01
Slide 39: LYNX-1: Improvement in Distance Vision
LYNX-1: Improvement in Distance Vision Source: LYNX-1 topline data Nyxol Provides Meaningful Low Contrast Vision Benefit Across Lighting Conditions at Day 15 NVD p<0.05 p<0.05 LYNX-1 Phase 3 Trial 3 Lines Improvement FDA Accepted Endpoint For Contrast Sensitivity Assessment Tested in Dim Light (Meso
Slide 40: Summary of Positive LYNX-1 Phase 3 Results
Summary of Positive LYNX-1 Phase 3 Results For Nyxol Eye Drops Source: mITT Population, LYNX-1Trial Data Support a Favorable Benefit/Risk Profile For Subjects with NVD No deaths or serious AEs AEs occurring in >5% of Nyxol treated subjects included: Instillation site irritation (9% vs 0% placebo), I
Slide 41: APX3330 Oral Tablet for DR and DME
DR DME 41 Diabetic Retinopathy Diabetic Macular Edema Apx3330 ORAL tablet
Slide 42: Growing Incidence of Diabetes and DR
Growing Incidence of Diabetes and DR Diabetes 34 M US >450 M WW DR 7 M US >150 M WW 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 Losing vision is one of diabetic patients
Slide 43: 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 Fac
Slide 44: APX3330 Reduces VEGF and Inflammation Preclinically
Source: 1. Tao Yan et al. APX3330 Promotes Neurorestorative effects after stroke in type one diabetic rats. Aging and Disease. Vol 9, Oct 2018 2. Apurinic/Apyrimidinic endonuclease 1 regulates inflammatory response in macrophages. 3. Jedinak A, Dudhgaonkar S, Kelley MR, Sliva D. Anticancer Res. 2011
Slide 45: Preclinical Data: Oral APX3330 Blocks Neovascularization
Preclinical Data: Oral APX3330 Blocks Neovascularization Source:1. Silva et al. ARVO 2021 Annual Meeting 2. *Published data on EYLEA. This study was performed independently from APX3330 study and is a cross-study comparison. 3. **Li 2014; *** Pasha 2018; ****Jiang 2011 (Vldlr -/- : Very Low-Density
Slide 46: Phase 1/2 Clinical Trials: PK Data Supporting ZETA-1
Phase 1/2 Clinical Trials: PK Data Supporting the ZETA-1 Trial Source: Eisai PK clinical data APX_CLN_0002 (left panel) Apexian preclinical data Eisai preclinical data Silva et al. Presented at the ARVO 2021 Annual Meeting APX3330 is Bioavailable and Reaches the Retina via Oral Administration Human
Slide 47: 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) ZETA-1 Clinical Trial is Sponsored by Ocuphire Pharma htt
Slide 48: 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 ETDRS Severity Level Steps Very Mild Mild Mod. Sev. VerySev. Mild Mod. High Risk Non-proliferative disease Proliferative disease PA
Slide 49: Masked Safety Findings from Ongoing ZETA-1 Trial
Masked Safety Findings from Ongoing ZETA-1 Trial Favorable Safety Profile (as of 3/17/2022) Observed with 600 mg Oral Daily Doses in Diabetic Subjects 103 Randomized Diabetic Subjects >4500Subject-Days of at 600mg/day APX3330 Exposure 43 Subjects with AEs (96 total events) APX3330 Masked Safety Data
Slide 50: Improving Eye Health in Diabetics
Improving Eye Health in Diabetics ↓ Inflammation ↓ Abnormal Angiogenesis Enhance Compliance & Exposure Oral pill may reduce the burden of frequent anti-VEGF injections >6600 Subject-exposure days* at ≥600 mg/day dose Few Systemic Adverse Effects < 5% Mild Gastrointestinal (diarrhea) < 5% Mild Skin R
Slide 51: 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 52: Large Global Market Opportunity in Retinal Disease
Large Global Market Opportunity in Retinal Disease Source: Nancy M. Holekamp, Overview of Diabetic Macular Edema, 2016. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6513508/pdf/jogh-09-010427.pdfhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC6513508/pdf/jogh-09-010427.pdf; https://www.ncbi.nlm.nih.gov
Slide 53: Team/Boards, Milestones, and Financial Data
Team/Boards, Milestones, and Financial Data
Slide 54: Ocuphire Management Team
Ocuphire Management Team Decades of Biotech and Drug Development Experience Mina Sooch, MBA President & CEO and Founder Drey ColemanVP, Clinical Operations Amy Rabourn, CPA VP, Finance Charlie Hoffmann, MBA VP Corporate Development and Operations Mitch Brigell, PhD Head, Clinical Development and Str
Slide 55: 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, MD University of Illinois Jay Pepose, MD, PhD UCLA School of Medicine Thomas Samuelson, MD University of Minnesota Paul Karpecki, OD Indiana University Eliot Lazar, MD Ge
Slide 56: Ocuphire Board of Directors
Ocuphire Board of Directors Sean Ainsworth, MBA Lead Independent Director, Board Director James Manuso, PhD/MBA Board Director Jay Pepose, MD, PhD Board Director Richard Rodgers, MBA Board Director Susan Benton, MBA Board Director Cam Gallagher, MBA Chair, Board Director Mina Sooch, MBA Vice-Cha
Slide 57: Track Record of Achieving Milestones
Track Record of Achieving Milestones Exciting 2022 News Cadence Ongoing Partnering Discussions with Leading Ophthalmic Companies (including European and Asian Players) 2021 2022 Report Positive Nyxol Alone Phase 2 Data for Presbyopia Report 2nd Phase 3 Data for RM (MIRA-3) Report Pediatric Data fo
Slide 58: OCUP – Market Snapshot
OCUP – Market Snapshot Source: FactSet Active Trading Volume and Sufficient Cash Runway Into 2Q 2023 Ticker OCUP Price $2.21 As of 5-17-22 Market Cap $43 M As of 5-17-22 Shares Outstanding 19.3 M As of 10Q (5-13-21) Cash $19.2 M As of 3-31-22 (unaudited) Cash Runway Sufficient into 2Q 2023 Guidance
Slide 59: Ocuphire Pharma Contact
www.ocuphire.comir@ocuphire.com Ocuphire Pharma Click here to view Ocuphire Pharma’s Investor R&D Day Recording