Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Ocuphire Pharma — Investor Presentation (August 2022)
46 slides from Opus Genetics's investor presentation, Aug 12, 2022
Slide 1: Ocuphire Corporate Presentation August 2022
Ocuphire Corporate Presentation August 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 success and timing of planned regulatory filings,
Slide 3: Two Lead Assets for Front and Back of the Eye
Two Lead Assets for Front and Back of the Eye Nyxol eye drops with > 650 patients treated across 12 trials (505(b)(2) pathway) APX3330 oral tablets with > 340 patients treated across 11 trials (NCE pathway) Four Large Markets with Unmet Needs in Reversal of Mydriasis (RM), Presbyopia, Night Vision D
Slide 4: Ophthalmology is Ripe for Partnering
Ophthalmology is Ripe for Partnering Prolific FDA Ophthalmology Division Deal Activity April 2021 Novartis / Alcon $355 M September 2021 REGENXBIO / Allergan $1.75 B October 2021 Théa / Curacle ~$2 B December 2021 Rayner / Omidria ~$1 B December 2021 Novartis / Gyroscope ~$1.5B January 2022 Théa / A
Slide 5: Ocuphire Overview: Two Late-Stage Clinical Assets
Source: Eisai and Apexian Data; GlobalData Market Research Report, 2020; Company Estimates for US Market Size; *Ocuphire internal estimates 11 Completed Phase 1 and Phase 2 Trials >340Subjects Dosed Patents to2034+ APX3330 Oral REF-1 Inhibitor New Chemical Entity Phase 2b Data 2H22 11 Completed Phas
Slide 6: 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 Positive MIRA-3 Phase 3 data in Q1 2022 (n=368) Positive MIRA-4 Pediatric data in 2Q 2022 (n=23) File Nyxol NDA for RM in late 2022 Presbyopia (P) N
Slide 7: Nyxol Eye Drops: Three Indications
7 Reversal of Mydriasis P NVD Presbyopia Night Vision Disturbance Nyxol® Eye Drops Three Indications Nyxol as a Single Drop for Presbyopia Nyxol with LDP Adjunctive Therapy 1 0.4% 2 RM
Slide 8: Nyxol's Differentiated MOA as an Alpha-1 Blocker
Nyxol’s Differentiated MOA as an Alpha-1 Blocker No Engagement of Ciliary Muscle No Headaches and Lower Risk of Retinal Detachment Phentolamine is the Active Ingredient in Nyxol: a Non-selective α1 & α2 Antagonist 505(b)(2) Regulatory Pathway Supported by Phentolamine Approval for 2 Indications: Re
Slide 9: Nyxol: 0.75% Phentolamine Ophthalmic Solution
Nyxol: 0.75% Phentolamine Ophthalmic Solution Preservative Free, EDTA Free, and Stable Efficacy Data Favorable Safety Profile Durable Nyxol Improves Vision by Decreasing Pupil (~1-1.5mm) ↑ Near Vision ↑ Distance Vision ↑ Contrast Sensitivity (night) No Systemic Effects No Changes in Blood Pressure N
Slide 10: Nyxol for Reversal of Mydriasis (RM)
10 RM Nyxol®forReversal of Mydriasis (RM) Mydriasis or dilated eyes causes: heightened sensitivity to light inability to focus reading, working, and driving difficulty halos and glare headaches I have to visit my retina MD for my monthly injections, where I am dilated. Being dilated every mont
Slide 11: Summary of MIRA FDA Registration Trial Designs
Summary of MIRA FDA Registration Trial Designs Source: In order from left to rightMIRA-2 Registration Trial NCT# 04620213 MIRA-3 Registration Trial NCT# 05134974 MIRA-2 1st Phase 3 MIRA-3 2nd Phase 3 US Sites 12 16 Subjects Enrolled 185 368 Eligibility Healthy ≥ 12 years old Healthy ≥ 12 years old R
Slide 12: Primary Endpoint Achieved in Two FDA Registration Phase 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 13: Planned NDA Package Incorporates Positive Data from MIRA Trials
Planned NDA Package Incorporates Positive Data from MIRA Trials Nyxol Significantly Reduced PD in Subjects Ages 3 & Over with Favorable Safety and Tolerability MIRA Clinical Trials RM No deaths, serious AEs, or withdrawals due to AEs All treatment related AEs were mild in severity The only AE occurr
Slide 14: NDA Submission Targeted For Late 2022
NDA Submission Targeted For Late 2022 Multiple Positive Clinical Trials To Support Broad Label Target Label Indication The treatment of pharmacologically induced mydriasis produced by adrenergic (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents, or a combination thereof. RM MIRA-
Slide 15: Commercial Strategy With Cash Pay Model
Commercial Strategy With Cash Pay Model Source: GlobalData Market Research Survey Simple, Value-Driven, Capital-Efficient and Accelerates Adoption RM Low Adoption Hurdles: No reimbursement or payors/PBMs No training, dilations are routine No specialty/retail pharmacies No competition 58% physicians
Slide 16: ~$500M+ US RM Market Opportunity
~$500M+ US RM Market Opportunity Reversal of Mydriasis (RM) Market Opportunity With No Commercially Available Treatment, Nyxol Could Achieve Significant Revenues 80 to 100 Average number of patients seen by an ECP per week 40 to 50 Average number patients dilated per week 20 to 25 Number of patients
Slide 17: Nyxol for Presbyopia
P Nyxol® forPRESBYOPIA 17 Nyxol Alone Nyxol with LDP Adjunctive Therapy 88% of patients expressed an interest in presbyopia-correcting drops across all ages and income groups Source: Burke Healthcare Research April 2020, n = 1000 presbyopes ages 40-80 years old. FDA approval of AbbVie Eye Drop a New
Slide 18: 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 19: 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 20: 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 21: VEGA-1: Improvement in Functional Near Vision
VEGA-1: Improvement in Functional Near Vision Similar trend was seen at all timepoints for charts Baseline Inclusion: Photopic DCNVA of 20/50 or worse Source: VEGA-1 clinical trial Nyxol and Nyxol with LDP Both Provide Durable Improvement in Functional Near Vision Nyxol as a Single Drop 1 Nyxol with
Slide 22: Summary Of Positive VEGA-1 Phase 2 Results
No serious AEs, most AEs were mild No headaches, no brow aches, and no blurry vision AEs were reported No loss in distance vision under photopic and mesopic lighting ~5% mild, transient redness No change in IOP Safety Nyxol and Nyxol+LDP Pooled Safety Findings Met planned P3 endpoint at 12 hours (29
Slide 23: Presbyopia Eye Drops Competitive Landscape
Phase 3 Phase 2 Phase 1 Orasis CSF-1 (Low dose pilo) Ocuphire Nyxol + 0.4% pilo Visus Brimochol® (carbachol + brim) Other Cholinergic Agonists* Cholinergic Agonist* (pilocarpine) Lenz Aceclidine; Aceclidine + brim Eyenovia MicroLine (2% pilo) Novartis EV-06 / Dioptin Alpha Antagonist & pilocarpine*
Slide 24: 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 25: 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 26: 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 26 NVD Nyxol® for Dim Light or Night Vision Disturbances
Slide 27: 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 28: LYNX-1: Nyxol Met Primary and Secondary Endpoints in NVD
LYNX-1: Nyxol Met Primary and Secondary Endpoints in NVD Source: LYNX-1 clinical trial; PP population; mLCVA: mesopic low contrast visual acuity; pLCVA: photopic low contrast visual acuity Significantly Higher % of Nyxol Treated Subjects Gained ≥15 Letter and ≥10 Letter From Baseline NVD Peripheral
Slide 29: 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 30: Market Opportunity in Dim Light or Night Vision Disturbances
Market Opportunity in Dim Light or Night Vision Disturbances GlobalData Market Research Report, 2020; Revenue potential based currently on Vuity Presbyopia pricing No Currently Approved Treatments in Development with Ripe Opportunity for Growth NVD Post-IOL Implant Post-Lasik Cortical Cataracts Nigh
Slide 31: APX3330 Oral Tablet: Diabetic Retinopathy and Diabetic Macular Edema
31 Apx3330 ORAL tAblet Diabetic Retinopathy and Diabetic Macular Edema DR DME I could lose my hearing, I could lose talking, but … it's frightening to lose my eyesight. Patient Diagnosed with DR
Slide 32: Diabetic Retinopathy At a Glance
Diabetic Retinopathy At a Glance Larger Disease to Manage with Growing Diabetes The number of people with DR expected to increase more than 14M by 2050 There are 8M adults in the U.S. with DR1 DR is the leading cause of blindness among working-age adults If untreated, DR can rob people of their visi
Slide 33: 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 34: Preclinical Data: Oral APX3330 Blocks Neovascularization
Preclinical Data: Oral APX3330 Blocks Neovascularization Source: Silva et al. ARVO 2021 Annual Meeting; Li 2014;Pasha 2018;Jiang 2011 (Vldlr -/- : Very Low-Density Lipoprotein receptor knock-out mice) Eisai PK clinical data APX_CLN_0002 Apexian preclinical data Eisai preclinical data Lesion Volume D
Slide 35: 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 36: 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 37: Masked Safety Findings from Ongoing ZETA-1 Trial
Masked Safety Findings from Ongoing ZETA-1 Trial 1. DME possibly study medication related (APX3330 or placebo) 2. Cellulitis (2 events in same subject), dyskinesia, transient ischemic event, COVID-19 and acute respiratory failure (same subject), progression of multivessel coronary artery disease, ch
Slide 38: APX3330 Product Candidate Profile for Multiple Retinal Indications
Novel MOA for treating retina ↓ Inflammation ↓ Abnormal Angiogenesis Convenient Oral Dosing for Patient Compliance Allow Daily vs. Episodic Exposure Oral pill may reduce the burden of frequent anti-VEGF injections >9000 Subject-exposure days* at ≥600 mg/day dose Few Systemic Adverse Effects < 5% Mil
Slide 39: Broad Opportunities to Treat Retinal Diseases with APX Platform
Diabetic Retinopathy PDR (DRSS >60) Moderate to Severe NPDR (DRSS 43-53) Mild NPDR 34 Million Diabetics in US 10 M 6M 1M 1M ~$8 M+DR Patients DME 1M Wet AMD 2M Dry AMD 10M+ RVO 2M GA 1M DR DME Broad Opportunities to Treat Retinal Diseases with APX Platform APX3330 May Treat Patients Across Retinal D
Slide 40: Milestones, Team/Boards, Financial Data
40 Milestones Team/Boards Financial Data
Slide 41: Track Record of Achieving Milestones
Track Record of Achieving Milestones Exciting News Cadence Ongoing Partnering Discussions with Leading Ophthalmic Companies (including Europe and Asia) 2021 – 1H 2022 2H 2022 – 2023 Submit Nyxol NDA for RM Report Phase 2b Data for DR/DME (ZETA-1) Initiate VEGA-2 & VEGA-3 Phase 3 Presbyopia Trials
Slide 42: 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 43: 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 44: 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 (Ant. Seg | Retina) James Katz, MD University of Illinois Jay Pepose, MD, PhD UCLA School of Medicine Thomas Samuelson, MD University of Minnesota Paul Karpecki, OD Indiana Universit
Slide 45: OCUP – Market Snapshot
OCUP – Market Snapshot Source: FactSet Active Trading Volume and Sufficient Cash Runway Through 3Q 2023 NASDAQ: OCUP Price/share $2.79 As of 8-11-22 Market Cap $57 M As of 8-11-22 Shares Outstanding 20.6 million As of 10Q (6-30-22) Cash $17 M As of 10Q (6-30-22) Cash Runway Sufficient through 3Q 202
Slide 46: www.ocuphire.com ir@ocuphire.com
www.ocuphire.com ir@ocuphire.com Ocuphire Pharma Click here to view Ocuphire Pharma’s Investor R&D Day Recording
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