Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (November 2023)
42 slides from Opus Genetics's investor presentation, Nov 2, 2023
Slide 1: Ocuphire Corporate Presentation November 2023
Ocuphire Corporate Presentation November 2023
Slide 2: 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 success and timing of planned regulatory filings and approvals, pre-commercial activities, co
Slide 3: Ocuphire Pipeline
Ocuphire Pipeline Product Candidate Indication Pre-clinical Phase 1 Phase 2 Phase 3 Regulatory Approval Upcoming Milestones APX3330 Oral Pill Diabetic Retinopathy (DR) EOP2 Mtg October 2023 Special Protocol Assessment (SPA) Submission APX3330 Local Delivery Retina Select retinal drug delivery techno
Slide 4: Management Team with Decades of Drug Development Experience
Management Team with Decades of Drug Development Experience Drey ColemanVP, Clinical Operations Amy Rabourn, CPA SVP, Finance Charlie Hoffmann, MBA SVP, Corporate Development Mitch Brigell, PhD Head, Clinical Development and Strategy Daniela Oniciu, PhD Global Head, R&D, Chemistry and Product Devel
Slide 5: Corporate Highlights
Corporate Highlights Late-Stage Clinical Candidate for Retinal Diseases Represents Multi-Billion Dollar Opportunity Phentolamine Ophthalmic Solution 0.75% (POS) for Refractive Disorders Global license agreement with Viatris to fund all development and commercialization for phentolamine indications:
Slide 6: Diabetic Retinopathy Market and Unmet Need
Diabetic Retinopathy Market and Unmet Need
Slide 7: Diabetic Eye Disease is a Common Cause of Blindness
Diabetic Eye Disease is a Common Cause of Blindness Diabetes and Diabetic Retinopathy (DR) https://webeye.ophth.uiowa.edu/eyeforum/tutorials/diabetic-retinopathy-med-students/Classification.htm https://www.mayoclinic.org/diseases-conditions/type-1-diabetes/symptoms-causes/syc-20353011 https://www.ma
Slide 8: Diabetic Retinopathy Market Data
American Diabetes Association; International Diabetes Federation; Healthline; *Ocuphire internal analysis and assumptions Das UN. DME, retinopathy and age-related macular degeneration as inflammatory conditions. Arch Med Sci. 2016;12(5):1142-1157. doi:10.5114/aoms.2016.61918 Patient survey adapted f
Slide 9: U.S. Diabetic Retinopathy Market
American Diabetes Association; International Diabetes Federation; Healthline; *Ocuphire internal analysis and assumptions; Spherix Global Insights Patient survey adapted from Lions International Foundation and International Diabetes Foundation-Europe; Meltzer 2000 Estimates are provided by the Natio
Slide 10: NPDR Patients Rarely Treated; Progression of DR Severity
Spherix Global Insights: DR Market DYNAMIX October 2022 Early treatment diabetic retinopathy study research group. ophthalmology. 1991;98(5 suppl):823-33. Diabetes control and complications trial research group. N Engl J Med. 1993;329(14):997-86. Fathy C, Patel S, Sternberg P Jr, Kohanim S. Disparit
Slide 11: Diabetic Retinopathy Treatment Landscape
Diabetic Retinopathy Treatment Landscape
Slide 12: Current Standard of Care Based on Severity
Current Standard of Care Based on Severity Currently, There Are No Non-Invasive Treatments Approved for Early Intervention or Slowing the Progression Wait and Monitor for Progression Mild to Moderate NPDR Anti-VEGF Injections PDR Jan Feb Mar Apr May Jun Jul Aug Sep Oct Nov Dec 10 81-85 71-75 Jan Feb
Slide 13: Landscape of Investigational Non-Invasive Therapies for Diabetic Retinopathy
Landscape of Investigational Non-Invasive Therapies for Diabetic Retinopathy Ocuphire’s APX3330 is the Most Advanced Oral Drug Candidate Company Drug Target/MOA Indication Route of Administration Phase 1 Phase 2 Phase 3 Primary Endpoint/ Secondary Endpoints APX3330 Ref-1 inhibitor (Anti-angiogenesis
Slide 14: Landscape of Invasive Therapies for Diabetic Retinopathy
Landscape of Invasive Therapies (IVT/Suprachoroidal) for Diabetic Retinopathy Eylea®/Lucentis® Approved, But Not Used in Patients with NPDR; Rarely Used in Mild PDR Company Drug Target/MOA Route of Administration Phase 1 Phase 2 Phase 3 Commercial Eylea® (aflibercept) VEGF-A/B; PIGF Intravitreal ✓ ✓
Slide 15: APX3330 Background
15 HARMA APX3330 Background
Slide 16: APX3330 Mechanism of Action Targeting Ref-1 Inhibition
Logsdon et al (2018), Li et al (2014). APX3330 - Mechanism of Action Targeting Ref-1 Inhibition Ref-1 Involved in Key Pathways that Contribute to Diabetic Retinopathy and Diabetic Macular Edema Ref-1 (reduction-oxidation effector factor-1) A novel target for retinal diseases, is a transcription fact
Slide 17: APX3330: Drug Development History and Patents
APX3330: Drug Development History and Patents Significant Preclinical & Clinical Data Supporting Human Safety, MOA, and PK 12* Phase 1 & Phase 2 Trials Exposure in Humans >10,000 Subject Days at 600mg/day Patents to2035+ Studied in inflammation/hepatitis & cancer patients (Studied by Eisai & Apexian
Slide 18: In-vitro Validation of Mechanism of Action
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):379-85
Slide 19: APX3330 VEGF Effects in Normal Cells
APX3330 VEGF Effects in Normal Cells Abnormal Conditions (e.g., hypoxic): Increased level of VEGF activity Normal Conditions: Physiological level of VEGF activity Kamba 2007; Girardi 2010; Li 2014 APX3330 Investigator Brochure Biologic Anti-VEGF Treatment APX3330 Anti-Ref-1 Treatment Biologic anti-V
Slide 20: APX3330 ZETA-1 Clinical Trial
APX3330 ZETA-1 Clinical Trial
Slide 21: ZETA-1: Phase 2 Trial of Oral APX3330 in Diabetic Retinopathy
ZETA-1: Phase 2 Trial of Oral APX3330 in Subjects With Diabetic Retinopathy Multi-center, Randomized, Double-Masked, Placebo-Controlled 24-Week Trial Primary: % subjects with ≥ 2 step improvement on DRSS (Diabetic Retinopathy Severity Scale1) at week 24 Secondary: DRSS improvement ≥1, ≥2, ≥3, ≥4 stu
Slide 22: ZETA-1: Baseline Demographics and Systemic Characteristics
ZETA-1: Baseline Demographics and Systemic Characteristics Well-Balanced Across Arms ZETA-1 Clinical Trial APX3330 n=51 Placebo n=52 Age (years) mean (range) 54.3 (26-81) 58.3 (24-78) Sex: Male n (%) 24 (47%) 26 (50%) Race: White n (%) Ethnicity: Hispanic or Latino n (%) 40 (78%) 28 (55
Slide 23: Clinically Meaningful Registration Endpoints in DR
Clinically Meaningful Registration Endpoints in DR Systemic Drugs Should Evaluate DRSS Change in Both Eyes; Formally Confirmed at EOP2 FDA Meeting Source: ZETA-1 Clinical trial 1. Nair P, Aiello LP, Gardner TW, Jampol LM, Ferris FL III. Report From the NEI/FDA Diabetic Retinopathy Clinical Trial Des
Slide 24: End-of-Phase 2 Meeting Outcome
End-of-Phase 2 Meeting Outcome
Slide 25: FDA Accepts the Binocular DRSS Person Scale
FDA Accepts the Binocular DRSS Person Scale For Phase 3 APX3330 DR Program In the binocular Person Scale, the worse eye is weighted instead of calculating the sum of both eyes A 3-step change on this scale is considered clinically meaningful by FDA Level (worse eye/better eye) Description Scale Step
Slide 26: ZETA-1: DRSS Improvement or Worsening at Week 24 (LOCF)
DRSS Worsening DRSS Improvement ZETA-1: Percent of Subjects with Improvement or Worsening in DRSS at Wk 24 on the Binocular Person Scale (LOCF) FDA EOP2 Meeting October 2023, Data Using Person Scale
Slide 27: ZETA-1: DRSS Improvement or Worsening at Week 24 (Observed Cases)
ZETA-1: Percent of Subjects with Improvement or Worsening in DRSS at Wk 24 on the Binocular Person Scale (Observed Cases) DRSS Improvement DRSS Worsening FDA EOP2 Meeting October 2023, Data Using Person Scale
Slide 28: APX3330 Reduced % of Subjects Developing PDR and Losing BCVA
APX3330 Reduced % of Subjects Developing PDR and % Losing BCVA Percentage of Subjects Developing PDR (mITT Population) at week 24 APX3330 reduced the percentage of subjects who developed PDR over the course of 24 weeks Percentage of Subjects with ≥ 5 Letters of BCVA Lost at Week 24 (Safety Populatio
Slide 29: ZETA-1: Treatment Emergent Adverse Events
APX3330 SAEs: Dyskinesia, TIA, Chest pain Placebo SAEs: Vertigo, Asthenia, Multiple organ dysfunction, Bradycardia, CAD, Cholelithiasis, COVID-19 pneumonia, Cellulitis, Respiratory failure, Skin ulcer, Peripheral embolism AEs → Withdrawal APX3330: Presyncope, Dyspnea; Placebo: DME (both eyes) *Prefe
Slide 30: APX3330 Summary and Milestones
APX3330 Summary Successful EOP2 FDA meeting completed in October 2023; agreement that a 3-step change on the binocular person scale is an approvable registration endpoint Submit Special Protocol Assessment (SPA) Advance APX3330 into Phase 3 program with long-term exposure (up to 2 years) APX3330 Mil
Slide 31: DR is One of the Largest Markets in Retina
DR is one of the largest markets in retina with 10M patients in US and over 100M worldwide Majority of the NPDR patients are not candidates for approved biologics treatments and are left untreated APX3330 first-in-class oral drug with unique MOA that inhibits Ref-1 which reduces VEGF and inflammator
Slide 32: Phentolamine Ophthalmic Solution 0.75%
Phentolamine Ophthalmic Solution 0.75%
Slide 33: Global Partnership with Viatris for Phentolamine Ophthalmic Solution 0.75%
Global Partnership with Viatris for Phentolamine Ophthalmic Solution 0.75% Viatris Has Selected POS to be a Key Element of its Global Eye Care Division Fully funded development and commercialization costs for all 3 phentolamine indications Partner for global commercialization Allows Ocuphire to focu
Slide 34: Phentolamine Ophthalmic Solution 0.75%: Three Indications
Treatment of Pharmacologically-Induced Mydriasis P DLD Presbyopia Dim Light or Night Vision Disturbances (DLD) POS as a Single Drop POS with LDP Adjunctive Therapy 1 0.4% 2 RM Phentolamine Ophthalmic Solution 0.75% THREE INDICATIONS NEW PARTNERSHIP WITH VIATRIS APPROVED RYZUMVl™ (Phentolamine Ophtha
Slide 35: Summary of Phentolamine Ophthalmic Solution 0.75% Trial Results
Summary of Phentolamine Ophthalmic Solution 0.75% Trial Results Comprehensive Body of Clinical Data Supporting Efficacy and Safety Across 3 Indications Primary Endpoint Efficacy Data Key Secondary Endpoint(s) Safety & Tolerability Return to baseline pupil diameter at 90 minutes after dilation Met Ph
Slide 36: Corporate Highlights
Corporate Highlights Strong Financial Position to Fund Operations into 2025 Late-Stage Retinal Pipeline Represents Multi-Billion Dollar Opportunity in Unmet NPDR Patients Global License Agreement with Viatris to Fund Development and Commercialization of Phentolamine Ophthalmic Solution 0.75% for All
Slide 37: 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 receptor knock-out mice) Preclinical Data: Oral APX3330 Block
Slide 38: Restore Vision & Clarity
Restore Vision & Clarity www.ocuphire.com ir@ocuphire.com Ocuphire Pharma Appendix 38
Slide 39: Phentolamine Ophthalmic Solution 0.75% Differentiated MOA as an Alpha-1 Blocker
Decreases pupil size (moderately) without affecting the iris sphincter or ciliary muscles 505(b)(2) Regulatory Pathway Supported by Prior Phentolamine Approvals in Non-Ophthalmic Indications Illustration for educational purposes Phentolamine Ophthalmic Solution 0.75%’s Differentiated MOA as an Alpha
Slide 40: Presbyopia Competitive Landscape
Phase 2 Phase 1 Ocuphire Phentolamine Ophthalmic Solution 0.75% + 0.4% pilo Visus Brimochol® (carbachol + brim) Other Cholinergic Agonists* Cholinergic Agonist* (pilocarpine) Lenz Aceclidine; Aceclidine + brim Eyenovia MicroLine (2% pilo) Alpha Antagonist & low dose pilocarpine* Alpha Antagonist NDA
Slide 41: Ocuphire's World-Class Medical Advisory Board
Ocuphire's World-Class Medical Advisory Board Chief Medical Advisor, Ocuphire Refractive Specialist Jay Pepose, MD, PhD UCLA School of Medicine Refractive Specialist Y. Ralph Chu, MD Northwestern University Refractive Specialist Zaina Al-Mohtaseb, MD Baylor College of Medicine Refractive Specialist
Slide 42: Ocuphire Board of Directors
Ocuphire Board of Directors Sean Ainsworth, MBA Lead Independent Director, Board Director Jay Pepose, MD, PhD Board Director Susan Benton, MBA Board Director Cam Gallagher, MBA Chair, Board Director James Manuso, PhD/MBA Board Director Richard Rodgers, MBA Board Director George Magrath, MD, MBA
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