Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (March 2024)
30 slides from Opus Genetics's investor presentation, Mar 27, 2024
Slide 1: Ocuphire Investor Presentation March 2024
O c u p h i r e I n v e s t o r P r e s e n t a t i o n M a r c h 2 0 2 4 Exhibit 99.1
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 End-of-Phase 2 meeting with the FDA to align on late-stage registration endpoints and study par
Slide 3: Positioned to Transform the Treatment of Diabetic Retinopathy
Positioned to Transform the Treatment of Diabetic Retinopathy 3 NPDR market calculated based on total DR market size of 8.9B in 2023 and NPDR revenue share of 70.38% in 2023. AMD, age-related macular degeneration; DR, diabetic retinopathy; DRSS, Diabetic Retinopathy Severity Scale; FDA, Food & Drug
Slide 4: APX3330 is the Foundation of Our Retina Pipeline
APX3330 is the Foundation of Our Retina Pipeline PRODUCT CANDIDATE INDICATION PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 REGULATORY APPROVAL MILESTONES APX3330 Oral pill Diabetic Retinopathy EOP2 meeting ✓ SPA submission ✓ APX2009 Geographic Atrophy Preclinical proof-of-concept APX2014 Retina Select drug
Slide 5: Diabetic Retinopathy is the Leading Cause of Vision Loss in Working-Age Adults in the US
Diabetic Retinopathy is the Leading Cause of Vision Loss in Working-Age Adults in the US 5 C L I N I C A L P R E S E N T A T I O N Blood vessels weaken, bulge, close off, or leak into the retina Growth of new abnormal blood vessels in the retina (neovascularization), vitreous hemorrhage, and scar ti
Slide 6: DR Progression Can Have a Significant Impact On Functional Vision
DR Progression Can Have a Significant Impact On Functional Vision NOTE: The severity of vision loss varies between individuals with DR DR, diabetic retinopathy; NPDR, non-proliferative diabetic retinopathy; PDR, proliferative diabetic retinopathy. 1. [No authors listed]. ETDRS report number 12. Opht
Slide 7: NPDR Represents a Large Segment of the Growing DR Market
NPDR Represents a Large Segment of the Growing DR Market 2M P D R 2 38 M DI ABETI CS 1 1 in 10 Americans have diabetes; 98M (over 1 in 3) have pre-diabetes1 10 M DR 2 ~1 in 4 diabetics (aged 40+) have DR1,2; Projected to increase to 19M+ by 20503 8M N P D R 2 NPDR market calculated based on total DR
Slide 8: APX3330 has the Potential to be the First Oral Treatment for DR
COMPANY DRUG PHASE TARGET ROA APX3330 Phase 2/3 Ref-1 inhibitor Oral Runcaciguat Phase 2 Guanylate cyclase activator Oral OPL-0401 Phase 2 ROCK 1/2 inhibitor Oral VX-1 Phase 2 AOC-3 inhibitor Oral RG7774 Discontinued CB2 receptor (cannabinoid) Oral OTT166 Phase 2 Missed efficacy endpoint Integrin in
Slide 9: APX3330: most advanced oral program in development for diabetic retinopathy
APX3330 The most advanced oral program currently in development for diabetic retinopathy
Slide 10: Ref-1 Mediates Multiple Pathways Involved in DR
Steroid injections / implants ONLY target inflammation Anti-VEGF injections ONLY target angiogenesis Ref-1 Mediates Multiple Pathways Involved in DR Current Invasive Treatments Only Target a Single Pathway 10 NOTE: Ozurdex®, Xipere®, and Iluvien® are not indicated for the treatment of DR. Lucentis®
Slide 11: APX3330 Inhibits Ref-1-mediated Angiogenesis, Oxidative Stress, and Inflammation
APX3330 Inhibits Ref-1-mediated Angiogenesis, Oxidative Stress, and Inflammation Nrf2, nuclear factor erythroid 2–related factor 2; Ref-1, reduction-oxidation effector factor-1; TNF-α: tumor necrosis factor alpha; VEGF, vascular endothelial growth factor. 1. Logsdon DP, et al. Sci Rep. 2018;8:13759.
Slide 12: In Vitro Data Validates Three Clinically Meaningful Pathways in DR
95 100 105 110 115 120 0 12.5 25 50 500 450 400 350 300 250 200 150 100 50 0 VEGF concentrations (% of control) * p<0.05 APX3330 restores physiologic VEGF levels1 APX3330 (30 μm) — ＋ — ＋ oxLDL (150 μg/mL) — — ＋ ＋ TNF-⍺ (pg/ml) IL-6 (pg/ml) * * * *p<0.05 *p<0.05 * % increase in APE1 repair activity *
Slide 13: ZETA-1 Clinical Trial
ZETA-1 Clinical Trial A Phase 2 Randomized, Placebo-Controlled, Double-Masked Study of APX3330 in DR is Complete
Slide 14: ZETA-1 Trial Design and Baseline DRSS Scores
Primary endpoint: % of subjects with a ≥ 2 step improvement in monocular ETDRS DRSS at week 24 Study eye: DR graded moderately severe to severe NPDR or mild PDR (monocular DRSS 47, 53, or 61) Fellow eye: No exclusion* Baseline DRSS Scores Placebo (n=52) APX3330 (n=51) DRSS Score – Study Eye 47 Moder
Slide 15: Binocular DRSS is a Validated and Well-Established Scale
ETDRS monocular severity scale1 Binocular person-level scale1 Moderately severe Severe or very severe Mild Subjects graded based on fundus photographs (images of the retina taken with a fundus camera). DR, diabetic retinopathy; DRSS, Diabetic Retinopathy Severity Scale; ETDRS, Early Treatment Diabet
Slide 16: ZETA-1 Analysis: Fewer APX3330-treated Subjects Worsened and More Improved
7% 4% 12% 12% 0% 5% 10% 15% Percent of Subjects ≤ -3 steps ≤ -4 steps DRSS Change from Baseline Placebo APX3330 Percentage of Subjects with Improvement at Week 24 on the Binocular DRSS Person-Level Scale (Observed Cases) 20% 13% 13% 5% 0% 10% 5% 15% Percent of Subjects 0% ≥ 4 steps ≥ 3 steps DRSS Ch
Slide 17: ZETA-1 Analysis: Fewer APX3330-treated Subjects Worsened
20% 15% 12% 12% 10% 7% 5% 4% 0% ≤ -3 DRSS ≤ -4 DRSS Percent of Subjects Percentage of Subjects with Improvement in DRSS at Week 24 on the Binocular Person-Level Scale (Observed Cases) Change from Baseline Placebo APX3330 ZETA-1 Analysis: Fewer APX3330-treated Subjects Worsened 17 Observed difference
Slide 18: APX3330 Decreased Rates of Developing PDR and Vision Loss
Percentage of Subjects Developing PDR by Week 24 (mITT Population) Percentage of Subjects with ≥ 5 Letters of BCVA Lost at Week 24 (Observed Cases) 26% APX3330 Decreased Rates of Developing PDR and Vision Loss APX3330 prevented progression of structural retinal abnormalities Observed differences bet
Slide 19: APX3330 Demonstrated a Favorable Safety and Tolerability Profile
APX3330 Demonstrated a Favorable Safety and Tolerability Profile Consistent with Prior Studies Total AEs Placebo (n=52) APX3330 (n=51) 120 91 Total treatment-related AEs 14 14 Subjects with treatment- related AEs 10 (20%) 10 (19%) Withdrawals due to treatment-related AEs 1 (2%) 1 (2%) AEs in >5% of
Slide 20: ZETA-2 Clinical Trial
ZETA-2 Clinical Trial A Phase 2/3 Randomized, Placebo-Controlled, Double-Masked Study of APX3330 in NPDR is Planned
Slide 21: Optimizing ZETA-2 for Success
Optimizing ZETA-2 for Success D u r a t i o n 24 weeks (6 months) 48 weeks (12 months) E l i g i b i l i t y Study eye (1 eye) Binocular (2 eyes) S a m p l e s i z e N=103 N=300 P r i m a r y e n d p o i n t ≥ 2-step DRSS improvement in the study eye ≥ 3-step DRSS worsening on a binocular person-lev
Slide 22: High-Risk NPDR Patients are More Likely to Progress
Patients in ZETA-2 will have high-risk NPDR N O D R N P D R P D R ETDRS monocular severity scale1 Binocular person-level scale1 Very mild Moderate Mild Moderately severe Severe or very severe Mild DR, diabetic retinopathy; DRSS, Diabetic Retinopathy Severity Scale; ETDRS, Early Treatment Diabetic Re
Slide 23: Observed Rates of Progression Increase as DR Severity Increases
Observed Rates of Progression Increase as DR Severity Increases Based on Landmark NEI ETDRS Study of Over 3,700 Patients 23 DRSS level 1-year progression rate to ANY PDR1 1-year progression rate to HIGH-RISK PDR2 43 Moderate NPDR 12% 3% 47 Moderately severe NPDR 26% 9% 53a to d Severe NPDR 44 – 51%
Slide 24: Optimized Study Design Positions ZETA-2 for Success
Optimized Study Design Positions ZETA-2 for Success APX3330 600 mg/day BID (n=150) 1:1 randomization (N=300) Primary Endpoint Placebo BID (n=150) Week 0 Week 24 Week 12 Week 48 BID, twice-daily; NPDR, non-proliferative diabetic retinopathy. ≥3 step worsening is associated with a higher likelihood of
Slide 25: Globally Recognized Retina Specialists Support APX3330 Development
Jeff Heier, MD Ophthalmic Consultants of Boston “If ZETA-1 results are repeated in Phase 3, I would place virtually all of my diabetic patients on oral APX3330 and treat locally only as needed." "Ref-1 biology targets three pillars of diabetic eye disease: angiogenesis, inflammation, and oxidative s
Slide 26: Partnership with Viatris
Partnership with Viatris Ryzumvi (phentolomine ophthalmic solution) 0.75% RYZUMVI is a trademark of Ocuphire Pharma, Inc.
Slide 27: Global Partnership with Viatris for Ryzumvi
Global Partnership with Viatris for Ryzumvi Ryzumvi approved for the reversal of pharmacologically- induced mydriasis and expected to launch in 1H 2024 Licensing agreement provides funding for 2 additional indications, with Viatris responsible for commercialization Two Phase 3 studies ongoing in pre
Slide 28: All Indications Have Sizeable Potential US Patient Populations
All 3 Indications Have Sizeable Potential US Patient Populations *RYZUMVI is indicated for the treatment of pharmacologically-induced mydriasis produced by adrenergic agonists (eg, phenylephrine) or parasympatholytic agents (eg, tropicamide). RYZUMVI is a trademark of Ocuphire Pharma, Inc. 1. Ryzumv
Slide 29: Highly Experienced Team with Meaningful Expertise
Highly Experienced Team with Meaningful Expertise Over 60 years of proven clinical, commercial, and transaction experience Involved in the research, development, and approval of numerous Ophthalmic products: Ophthalmic Experts 29 Ash Jayagopal, PhD, MBA Chief Scientific & Development Officer Vabysmo
Slide 30: Ocuphire is Positioned to Transform the Treatment of Diabetic Retinopathy
DR, diabetic retinopathy; FDA, Food and Drug Administration. Ocuphire is Positioned to Transform the Treatment of Diabetic Retinopathy Extensive understanding of large, underserved DR market Addressing unmet needs by targeting multiple DR pathways with oral treatment Demonstrated efficacy in slowing