Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (May 2023)
27 slides from Opus Genetics's investor presentation, May 15, 2023
Slide 1: Ocuphire Corporate Presentation May 2023
Ocuphire Corporate Presentation May 2023
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 success and timing of planned regulatory filings a
Slide 3: Corporate Highlights
Corporate Highlights Two Lead Clinical-Stage Novel Drugs Addressing Multiple Large Ophthalmology Markets with Limited to No Competition & Extensive Patent Portfolio Global License Agreement with Viatris to Fund the Development and Commercialization of Nyxol for All Indications Strong Financial Posit
Slide 4: Ocuphire Pipeline
Product Candidate Indication Pre-clinical Phase 1 Phase 2 Phase 3 Regulatory Approval Upcoming Milestones APX3330 Oral Pill Diabetic Retinopathy (DR)/ Macular Edema (DME) EOP2 Mtg 2H 2023 to Advance to Phase 3 Nyxol® Eye Drop Reversal of Mydriasis (RM) PDUFA Date Sep 28, 2023 Nyxol® Eye Drop Presbyo
Slide 5: Global Partnership with Viatris for Nyxol
Global Partnership with Viatris for Nyxol Viatris Has Selected Nyxol to be a Key Element of its Global Eye Care Division Fully funded development and commercialization costs for all 3 Nyxol indications Partner for Nyxol global commercialization Allows Ocuphire to focus on APX3330 development Strengt
Slide 6: APX3330 Oral Tablet: DR and DME
6 APX3330 ORAL TABLET DR Diabetic Retinopathy DME Diabetic Macular Edema “ ” “I could lose my hearing, I could lose talking but…. It’s frightening to lose my eyesight.” Patient Diagnosed with DR
Slide 7: Diabetic Retinopathy At a Glance
Diabetic Retinopathy At a Glance Larger Disease to Manage with Growing Diabetes There are 8M adults in the U.S. with DR1 DR is the leading cause of blindness among working-age adults DR/DME affects about 1 in 4 people with type 1 and type 2 diabetes Majority of patients with mild to moderate DR are
Slide 8: DRSS Predicts Vision-Threatening Complications (PDR/DME)
DRSS Predicts Vision-Threatening Complications (PDR/DME) 8 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. Dispa
Slide 9: Broad Opportunities to Treat Retinal Diseases with APX3330
Broad Opportunities to Treat Retinal Diseases with APX3330 Source:1. American Diabetes Association; International Diabetes Federation; Healthline; *Ocuphire internal analysis and assumptions; 2. Das UN. DME, retinopathy and age-related macular degeneration as inflammatory conditions. Arch Med Sci. 2
Slide 10: APX3330 Profile Overview
APX3330 Profile Overview Oral, First-In-Class Ref-1 Inhibitor with Favorable Human Safety Data from 12 Completed Trials Novel MOA for Treating Retina ↓ Inflammation ↓ Abnormal Angiogenesis Good Patient Compliance in ZETA-1 with Convenient Oral Dosing APX3330 Demonstrated Slowing of Progression of Di
Slide 11: APX3330 Ref-1 Inhibition Decreases Abnormal Angiogenesis
Sources: Logsdon et al (2018), Li et al (2014). APX3330 Ref-1 Inhibition – Decreases Abnormal Angiogenesis Ref-1 Involved in Multiple Key Pathways that Contribute to DR and DME Mechanism of Action – Ref-1 Inhibition Hypoxia Ref-1 HIF-1α VEGF (Signaling Cascade) Inflammation Ref-1 NF-κB Other Growth
Slide 12: ZETA-1 Phase 2 Design – APX3330 in DR
Primary: % subjects with ≥ 2 step improvement on DRSS (Diabetic Retinopathy Severity Scale1) at week 24 Secondary: DRSS worsening ≥1, ≥2, ≥3*, ≥4* DRSS worsening ≥1, ≥2, ≥3*, ≥4* Progression to vision threatening complications Central subfield thickness (CST) Best Corrected Distance Visual Acuity (B
Slide 13: Change in DRSS is Regulatory Approval Endpoint for DR
Change in DRSS is Regulatory Approval Endpoint for DR Local Drugs (Intravitreal Injections) Systemic Drugs Precedent approvable endpoint for locally- delivered drugs (Non-Systemic) in DR: ≥ 2-step DRSS improvement in study eye Aflibercept (PANORAMA trial) Ranibizumab (RISE/RIDE/DRCR trials) Potentia
Slide 14: ZETA-1 Baseline Characteristics
Source: ZETA-1 Clinical Trial Key Visual Metrics APX3330 n=51 Placebo n=52 Total n=103 BCVA Study Eye Letters (mean) 81 78 80 (20/25 Snellen) BCVA Fellow Eye Letters (mean) 76 77 77 (20/32 Snellen) OCT CST Study Eye (µm) 270 271 271 OCT CST Fellow Eye (µm) 292 286 289 Intraretinal Fluid in the Cente
Slide 15: ZETA-1: Binocular Improvement/Worsening in DRSS at Week 24
ZETA-1: % of Subjects with Binocular Improvement/Worsening in DRSS at Wk 24 APX3330 Demonstrated Statistical Efficacy on Potential Phase 3 Registration Endpoint p=0.12 8% p=0.04 16% 18% 35% 33% 33% 20% 6% p=0.18 2% 0% 0% 11% 36% 36% 29% 18% 11% 9% 0% 5% 10% 15% 20% 25% 30% 35% 40% ≥ 4 Steps Worsenin
Slide 16: ZETA-1: Binocular ≥3-Step Worsening in DRSS
ZETA-1: Percent of Subjects With Binocular ≥ 3-Step Worsening in DRSS Primary Endpoint for Planned Phase 3 Demonstrates Prevention of Progression 12% 16% 0% 5% 10% 15% 20% Week 12 Week 24 Percent of Subjects (%) Visit Placebo (N=49) APX3330 (N=46) n=49 0% n=45 0% n=45 n=49 p=0.04 p=0.07 Percent of S
Slide 17: ZETA-1: Treatment Emergent Adverse Events
APX3330 SAEs: Dyskinesia, TIA, Chest pain Placebo SAEs: Vertigo, Asthenia, Multiple organ dysfunction, Bradycardia, CAD AEs → Withdrawal APX3330: Presyncope, Dyspnea; Placebo: DME (both eyes) *Preferred Term within Organ Class ZETA-1: Treatment Emergent Adverse Events Oral APX3330 Showed a Favorable
Slide 18: APX3330 - Phase Summary and Next Steps
APX3330 - Phase 2 Summary and Next Steps APX3330 is the most advanced oral program in development for diabetic eye disease APX3330 demonstrated favorable safety with compelling potential to slow progression of diabetic retinopathy ZETA-1 statistically significant results on potential Phase 3 registr
Slide 19: Nyxol Eye Drops: Three Indications
19 Reversal of Mydriasis (RM) P DLD Presbyopia Dim Light or Night Vision Disturbances (DLD) Nyxol as a Single Drop Nyxol with LDP Adjunctive Therapy 1 0.4% 2 RM RM NYXOL® EYE DROPS THREE INDICATIONS NEW PARTNERSHIP WITH VIATRIS
Slide 20: Nyxol's Differentiated MOA as an Alpha-1 Blocker
Decreases pupil size (moderately) without affecting the iris sphincter or ciliary muscles Allows for 3 indications: RM, Presbyopia and DLD 505(b)(2) Regulatory Pathway Supported by Prior Phentolamine Approvals in non-ophthalmic Indications Nyxol’s Differentiated MOA as an Alpha-1 Blocker No Engageme
Slide 21: Nyxol Phase 3 Efficacy and Safety Data
*Trend toward statistical significance even in smaller POS arm from time 0 to time 6 hours (n=30); larger sample size for all arms planned in Phase 3 program Primary Endpoint Efficacy Data Key Secondary Endpoint(s) Safety & Tolerability Return to baseline pupil diameter at 90 minutes after dilation
Slide 22: Presbyopia Competitive Landscape
Phase 2 Phase 1 Ocuphire Nyxol + 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 Allergan VUITYTM; (1.25% pilo) O
Slide 23: Corporate Highlights
Corporate Highlights Two Lead Clinical-Stage Novel Drugs Addressing Multiple Large Ophthalmology Markets with Limited to No Competition & Extensive Patent Portfolio Global License Agreement with Viatris to Fully Fund the Development and Commercialization of Nyxol for All Indications Strong Financial
Slide 24: Restore Vision & Clarity – Appendix: Team and Nyxol Data
Restore Vision & Clarity www.ocuphire.com ir@ocuphire.com Ocuphire Pharma Appendix: Team and Nyxol Data 24
Slide 25: Management Team with Decades of Drug Development Experience
25 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 Development Ronil Patel, MS SVP, Operations and BD Chris Ernst
Slide 26: 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 27: Ocuphire Board of Directors
Ocuphire Board of Directors Seasoned Directors with Decades of Drug Development, M&A/Financings, and Ophthalmology 27 Sean Ainsworth, MBA Lead Independent Director, Board Director Jay Pepose, MD, PhD Board Director Cam Gallagher, MBA Chair, Board Director Mina Sooch, MBA Board Director Susan Bento