EXHIBIT 99.2
Published on August 12, 2021
Ocuphire Corporate Presentation August 12, 2021 Mina Sooch CEO Exhibit 99.2
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 Ocuphire Pharma, Inc.’s (“Ocuphire” or the “Company”) product candidates and future milestones,
including the potential for Nyxol to be a “best in class” presbyopia drop. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect.
Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: (i) timing or ability for the company to
achieve its targeted milestones; (ii) the success and timing of regulatory submissions and pre-clinical and clinical trials; (iii) regulatory requirements or developments; (iv) changes to clinical trial designs and regulatory pathways; (v)
changes in capital resource requirements; (vi) risks related to the inability of the Company to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; (vii) legislative, regulatory,
political and economic developments, and (viii) the effects of COVID-19 on clinical programs and business operations. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed
as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors detailed in documents that have been and may be filed by the Company from time to time with the SEC. All
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the owners thereof and are used for reference purposes only. Such use should not be construed as an endorsement of such products. November 6, 2020
Late Clinical Stage Company Targeting Large, Unmet Ophthalmic Markets Significant Clinical Data and
Regulatory Precedents Significant IP Portfolio and Small Molecule CMC Advantages Multiple Near-Term Data Catalysts with Capital Efficient Plan Nyxol eye drops target multiple chronic and acute front of the eye indications addressing large
markets: Dim Light / Night Vision Disturbances (NVD), Reversal of Mydriasis (RM), & Presbyopia (P)APX3330 tablets target chronic back of the eye indications: Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME), a leading cause of
blindness in diabetic patients Nyxol and APX3330 achieved promising clinical data over multiple Phase 1, 2, and 3 trialsNyxol with > 330 patients treated across 9 trialsAPX3330 with > 340 patients treated across 11 trialsFDA End of
Phase 2 meeting guidance for Nyxol (all indications) in May 2020 US and global issued patents thru 2034 for both assets; new 2039 Nyxol patent issued for presbyopia Stable, small-molecule drugsNyxol = single-use, preservative-free eye
dropAPX3330 = oral pill Initiated 4 late-stage trials (2 Phase 3, 2 Phase 2) with readouts expected in 2021-2022Reported positive P3 data in RM in 1Q21 with Nyxol NDA submission targeted late 2022Reported positive P2 data in Presbyopia in
2Q21 with plans to advance to P3 in 2022Recent $15M offering combined with cash on hand sufficient to run operations into late 2022Analyst coverage by Cantor, Canaccord, Jones Trading, Alliance Global, and Encode Ideas Ocuphire
Opportunity A Late-Stage Clinical Ophthalmic Biotech (Nasdaq Symbol: OCUP) Nyxol® APX3330
Ocuphire Management Team Decades of Biotech and Drug Development Experience Amy Rabourn,
CPAVP Finance Daniela Oniciu, PhDHead CMC and Global Clinical Supply Ronil Patel, MSSenior Director BD and Market Strategy Mitch Brigell, PhDHead Clinical Development and Strategy Chris ErnstQuality Assurance Lead Drey ColemanHead
Clinical Operations Charlie Hoffmann, MBAVP Corporate Development and Operations Mina Sooch, MBAPresident & CEO and Founder
Large Unmet Opportunities for the Aging Eye Source: GlobalData Market Research Report, 2020; Company
Estimates for Market Size Developing Drugs to Treat Front & Back of the Eye Diseases Back Front PresbyopiaU.S. Prevalence: ~120M Night Vision DisturbancesU.S. Prevalence: ~16M adults Reversal of Mydriasis~100M pupil dilations
per year in U.S. Nyxol® APX3330 $10+ to $20+B US Markets $4 to $10B US Markets Diabetic Macular EdemaU.S. Prevalence: ~750K Diabetic RetinopathyU.S. Prevalence:~ 7M $325M- $1B US Market Opportunity $9B - $18B $2B - $4B $3B -
$7B US Market Opportunity $1B - $3B
Product Candidate Indication Development Stage Anticipated
Milestones Pre-clinical Phase 1 Phase 2 Phase 3 Ocuphire-Focused Development 0.75% Nyxol®Eye Drop Reversal of Mydriasis (RM) Initiated Phase 3 MIRA-2 trial 4Q20; Topline data reported in 1Q21 (n=185)Initiate Phase 3
MIRA-3 trial 2H21; Data expected in early 2022 (n=330) Initiate Pediatric trial 2H21;Data expected in early 2022 (n=20) 0.75% Nyxol® + Low-Dose 0.4% Pilocarpine Eye Drops Presbyopia (P) Initiated Phase 2 VEGA-1 trial
1Q21;Topline data reported in 2Q21 (n=150)Initiate Phase 3 program in 1H22 0.75% Nyxol®Eye Drop Dim Light or Night Vision Disturbances (NVD) Initiated Phase 3 LYNX-1 trial 4Q20;Data expected in early 2022 (n=160) APX3330 Oral
Pill Diabetic Retinopathy (DR)/ Macular Edema (DME) Initiated Phase 2 ZETA-1 trial Apr21;Data expected in 2022 (n=100) Partnering-Focused Development APX2009 Intravitreal DME, Wet Age-Related Macular Degeneration
(wAMD) Next steps: IND enabling studies (with partner funding) Ocuphire Pipeline & Upcoming Milestones Multiple Phase 3 & Phase 2 Clinical Data Readouts Anticipated over the Next Year Note: 0.75% Nyxol (Phentolamine
Ophthalmic Solution) is the same as 1% Nyxol (Phentolamine Mesylate Ophthalmic Solution) Positive Data Readout Recruiting Positive Data Readout Recruiting
11Phase 1 & Phase 2 Trials >340Subjects Dosed Exposure in Humans 365 Days Patents
to2034+ Extensive Development on Both Drug Candidates Well-Controlled Phase 1, 2, and 3 Clinical Programs with MIRA-2 Data Leading the NDA Path NCE Development Pathway Studied in inflammation/hepatitis & cancer patients 9Phase
1, Phase 2, and Phase 3 Trials > 330Subjects Dosed Exposure in Humans28Days Patents to2034+ 505(b)(2) Development Pathway Studied in multiple ocular refractive indications Nyxol APX3330
Nyxol® Phentolamine Mesylate Presbyopia RM Night Vision Disturbances Reversal of
Mydriasis NVD P
Nyxol History & MOA Nyxol’s active ingredient, phentolamine mesylate (PM), is currently approved
for 2 indicationsPheochromocytoma (60+ years ago, Regitine®) – intravenous injectionReversal of oral anesthesia (10+ years ago, OraVerse®) – intramuscular injectionPM has been reformulated as a topical eye drop (Nyxol)Nyxol is a
first-in-class non-selective α1 and α2 blocker product candidateMOA of relaxing the iris dilator muscle (α1)Redness is an on-target α1 effect on sclera vessels (transient, mild) Rationale for Differentiated Product Profile & 505(b)(2)
Path Phentolamine Mesylate Reduces Pupil Size Dilates Blood Vessels (Vasodilation) α1: Smooth Muscle Blockade α1: Iris Dilator Blockade
Improving Vision↓ Pupil Size (moderate miotic)↑ Contrast Sensitivity (night)↑ Near Visual Acuity
(light/dark)↑ Distance Visual Acuity No Systemic EffectsNo Changes in Blood PressureNo Changes in Heart RateTolerated Topical EffectsMild / Transient / Reversible Eye RednessIOP Unchanged or Decreased↓ Intraocular Pressure (IOP) at Normal
Baseline Nyxol Product Candidate Profile Novel Alpha 1/2 Blocker Eye Drop for Refractive Indications (505(b)(2) Pathway) Safety Data Efficacy Data Nyxol: 0.75% Phentolamine Ophthalmic SolutionPreservative Free, EDTA Free, and
Stable Chronic daily dosing of Nyxol at bedtime demonstrated no significant daytime redness and durability of effects for more than 24 hours
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of
Mydriasis 0.4% +
Reversal of Mydriasis (RM) – Acute Treatment At many annual eye exams and specialty visits, pupils
are pharmacologically dilated, impairing vision for 6-24 hoursDilated eyes:heightened sensitivity to light inability to focus reading, working, and driving are difficult halos and glare The Problem Annual Exams and Specialty Visits Involve
Dilation to Monitor Eye Health ~100M eye exams / year in US No Current Commercially Available Treatments RM Source: GlobalData Market Research Report, 2020 I have to stay indoors. They say it only lasts a few hours, but it lasts all day,
and it is very annoying. RM Patient, Aged 51
After Before Reversal of Mydriasis (RM) – Acute Treatment Regulatory Precedent with Rev-Eyes (an
alpha 1 blocker), approved by the FDA in 1990 but shortly thereafter discontinued (not for safety or efficacy reasons)Clinical Effect to potentially reduce pupil size and counteract the effect of mydriatic drugs (alpha agonists and
cholinergic blockers) used to dilate the pupilConvenient and Stable eye drop given at the office that may allow vision to return to normal soonerTolerable with a minimal side effect profile (unlike cholinergic agonists such as
pilocarpine) Nyxol’s Potential Differentiated Solution Single Use Indication Leveraging a Precedent Approval Pathway Seeking Treatment Findings Patients likely to request reversal of dilation1 80% Eye care providers likely to use
reversal drops2 70% Source: 1.GlobalData Market Research Report, 2020 – percentage includes those who answered moderately to highly likely (4-7 on a scale of 1-7) 2.GlobalData Market Research Report, 2020 – percentage includes those who
answered moderately to highly likely (6-10 on a scale of 0-10) RM
RM MIRA-2 Phase 3 Registration Design Completed Randomized, Double-Masked, Placebo-Controlled,
Parallel, One-Day Trial MydriasisTime -1 Hour MIRA-2 1:1 Mydriatic Agent A, B, or C 0.75% Nyxol Placebo 12 US sites168 target healthy subjects Nyxol drop(s)(2 drops study eye, 1 drop fellow eye) Mydriatic Agent A, B, or C Placebo
drop(s)(2 drops study eye, 1 drop fellow eye) Treatment Time 0 (Max Dilation) Primary: % of subjects (study eye) returning to baseline (within 0.2 mm) pupil diameter (PD) at 90 minSecondary:% of subjects returning to baseline at 30min, 1h,
2h, 3h, 4h, 6h, 24h (overall, by mydriatic agent, by iris color)Mean change in pupil diameter from mydriatic max at all timepoints (overall, by mydriatic agent, by iris color)Accommodation (Tropicamide/Paremyd)Safety and tolerability
(redness) Endpoints Started and Completed Enrollment in 4Q20 – 185 Subjects Topline Results Expected in 1Q21 Reported in March 2021 Eligibility Screening Randomization Mydriatic Agents 3:1:1 – 2.5% phenylephrine (alpha 1 agonist), 1%
tropicamide (cholinergic blocker), Paremyd® (combination) 1:1 RM
Primary Endpoint: % of Subjects Study Eye Returning to Baseline PD at 90 Min Source: MIRA-2 Trial,
mITT Population (same as Safety Population), *Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd) Nyxol Met the Primary & Secondary Endpoints at 90 Min; Additionally at 60 Min & All Subsequent Timepoints Max
pupil dilation, Treatment Mydriatic p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 Nyxol Reduced More Subjects to Baseline Pupil Diameter (PD) Nyxol Reduced PD Faster Across All Mydriatic Agents* MIRA-2 Phase 3
Trial *Data include all three mydriatics (Phenylephrine, Tropicamide, Paremyd) p<0.0001 RM
Secondary Endpoint: Mean Pupil Diameter Over Time by Mydriatic Agent Source: mITT Population, MIRA-2
Trial, Standard Error bars are shown. Nyxol Reduced Pupil Diameter With All Mydriatic Agents; More Rapidly with Phenylephrine as Expected MIRA-2 Phase 3 Trial Mydriatic Max pupil dilation, Treatment Max pupil dilation,
Treatment Mydriatic Nyxol More Rapidly Reduced PD in Subjects Across All 3 Mydriatic Agents Mean Pupil
Diameter RM p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.0001
Secondary Endpoint: % of Subjects Returning to Baseline PD by Iris Color Source: MIRA-2 Trial mITT
Population,, Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd) Evidence of Efficacy in Subjects with Either Light or Dark Irides, with a More Vigorous Response in Light Irides MIRA-2 Phase 3
Trial p<0.0001 p<0.0001 p<0.01 p<0.0001 p<0.0001 p<0.0001 p<0.0001 p<0.001 p<0.001 p<0.001 p<0.01 p<0.0001 Percent of Subjects Returning to ≤ 0.2 mm of Baseline by Iris Color More Subjects
Returned to PD Baseline with Nyxol in Both Light and Dark Irides RM
Secondary Endpoint: Accommodation And Time Savings Nyxol Demonstrates a Faster Return to Baseline
Accommodation and Shorter Dilation Time by 4-5 Hours Source: MIRA-2 CSR table #14.2.3.2.1. PP population is the per protocol population. Note: Worsening of accommodation was defined as an amplitude decrease of greater than 1 diopter
Percent of Subjects with Unchanged Accommodation from Baseline (Tropicamide or Paremyd)Study Eye, PP population MIRA-2 Phase 3 Trial RM Overall MydriaticAgent Irides Color n=185 n=185 n=97 n=34 n=81 n=84 n=34 Average Time to
Return to ≤ 0.2 mm of Baseline PD Δt= 3.5 hrs Δt= 3.2 hrs Δt= 3.4 hrs Δt= 4.1 hrs Δt= 3.6 hrs Δt= 2.3 hrs Δt= 4.5 hrs
Summary of Positive MIRA-2 Phase 3 Results for Nyxol Eye Drops Met primary endpoint at 90 minutes
with high statistical significance with 2 drops of NyxolMet all key secondary endpoints with high statistical significanceEfficacy for all 3 mydriatic agents – phenylephrine, tropicamide, and Paremyd® Efficacy in both light and dark iris
colorsEfficacy with only one Nyxol drop in non-study eyeFavorable safety profileNo serious AEs, no drop-outs from AEs, no systemic AEs were observed in ≥ 5% of subjectsMild, transient conjunctival hyperemia reported in the first hour and
declined steadily thereafter. Baseline mean of 0.7, the mean hyperemia score increased by approximately 1.0 unit on CCLRU scale mITT Population, MIRA-2 Trial Sustained Efficacy with a Favorable Safety Profile in Reversing Mydriasis with
Nyxol Complete a second RM Phase 3 trial with increased subjects ~330 to also meet 24-hour safety population exposureComplete RM trial with 20 subjects ages 3 to 11 per pediatric planComplete registration batches with 1-year CMC stability
and make commercial batches Path to Registration Submit NDA by Late 2022 Proposed Indication The treatment of pharmacologically induced mydriasis produced by adrenergic (e.g. phenylephrine) or parasympatholytic (e.g. tropicamide) agents,
or a combination thereof. RM
Reversal of Mydriasis (RM) Market Opportunity GlobalData market research reportVision Care Market
Grows 2.4 Percent in 12-Months Ending September 2019. Vision Monday, January 20, 2020. With No Commercially Available Treatment, Nyxol May Provide Significant Revenue Potential > 65% PatientsReport moderate to severe negative impact of
dilated exams1 $5 - $20Price range surveyed for cash pay per patient with room for physician markup1 $325M - $1B+Estimated US RM Market Opportunity 100M+General and specialty eye exams per year1 > $6B Eye Exam MarketExams, the
third-largest category, grew faster than both prescription lenses and frames Use of phenylephrine, tropicamide, Paremyd®, or combinations of such comprise nearly 95% of dilating eye drops used by eyecare professionals.1 OptoMap: Retinal
screening for those wanting to avoid dilations but not a replacement for full dilated eye exam $40-65 paid by patients RM
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of
Mydriasis 0.4% +
Presbyopia – Chronic Opportunity Lens loses ability to change shape when viewing objects up close as
we ageDependence on reading glasses for intermittent and prolonged useGrowing need for therapies that improve, rather than hinder, quality of life Source: GlobalData Market Research Report, 2020 The Problem Aging Population Drives Demand
for Alternatives to Reading Glasses & Very Large Market P 120 M Patients No Currently Approved Drug Therapies Effectively everyone over 40 will have the problems with reading. Physician KOL Seeking Treatment Findings Patients
requesting alternative to reading glasses 40% Patients would consider an eye drop alternative 69% ~$9-$18B Market Opportunity Market Assumptions:Total patients - 120 million patientsPrice per month - $50Patients considering eyedrops -
~50%Refills (Months) - 3 to 6
Presbyopia – Chronic Opportunity “Pin-hole” effect of Nyxol and low dose pilocarpine may improve near
vision by enhancing depth of field as validated by other devices/therapiesMore durable combination of two miotics affecting different muscles (iris dilator and sphincter) involved in pupil size modulationTolerable use with minimal side
effects expected with chronic evening use of Nyxol and daytime use of fractional concentration of pilocarpine Retinaeyedoctor.com, GlobalData Market Research Report, 2020 Nyxol’s Potential Differentiated Solution Pupil Modulation Eye Drops
May Replace Reading Glasses Large Pupil Pin-hole Pupil Near Far In focus In focus This would just become part of my daily routine for my eyes to be able to see things up close. How convenient is that? Presbyopic Patient, age
49 P
Product Profile: Nyxol ® + Low-Dose Pilocarpine (LDP) Combo Moderate Action on Iris Dilator and Iris
Sphincter Muscles for Near Vision Improvement 0.4% 0.75%Nyxol 0.4% LDP Iris Dilator Muscle Inhibition Iris Sphincter Muscle Activation Phentolamine (alpha1/2 antagonist) approved non-ocular injectable indications
decade(s) ago 505(b)(2)Novel MOA on iris dilator with 24+ hour durabilityModerate 1+mm pupil reductionNo daytime redness w/ chronic evening dosing NyxolWell-tolerated with no systemic effectsStable, preservative-free, single use
vial Pilocarpine (cholinergic agonist) approved decades ago Known MOA on sphincter muscle with potent miotic effects at approved doses (1%, 2%, 4%)Chronic daily dosing of LDP, a more moderate mioticLow concentration avoids known tolerability
issues:headache and browacherednessaccommodative spasm causing loss of distance vision especially at night Nyxol LDP 1.5 to 2.5 mm PD reductionmoves toward the ‘pin-hole’ (2.0 to 2.5 mm, up to 3 mm) Source: Nyxol® data from 8
completed trials; Pilocarpine Product label and Literature P
Presbyopia VEGA-1 Phase 2 Design Clinical trial NCT#04675151. DCNVA = distance-corrected near visual
acuity . BCDVA = best corrected distance visual acuity Randomized, Double-Masked, Placebo-Controlled, Multi-Center One-Week Trial Primary: % of subjects with ≥ 3 lines of improvement in distance-corrected near visual acuity comparing Nyxol
+ LDP vs placebo alone at 1 hourSecondary:% of subjects with ≥ 2 and ≥ 3 lines gained at time points from 30 min to 6 hours in photopic and mesopic lighting comparing Nyxol + LDP vs placebo, Nyxol alone, and LDP aloneNo loss of distance
vision Pupil diameter at time pointsSafety and tolerability (redness) Endpoints Visit 1 VEGA-1 Randomization 4 arms 0.75% Nyxol Placebo 17 US sites150 presbyopic patients Visit 2 (3 – 6 Days Later) Screening Treatment
Arms Nyxol + LDP LDP Drop Nyxol Baseline Nyxol Alone No Treatment Nyxol Baseline LDP Alone LDP Drop Placebo Baseline Placebo Alone No Treatment Placebo Baseline Evening Dosing(3-4 doses) Males or females ≥ 40 and ≤ 64
years of ageBCDVA of 0.0 LogMAR(20/20 Snellen equivalent) or better in each eye under photopic conditionsDCNVA of 0.4 LogMAR (20/50 Snellen equivalent) or worse in photopic conditions in each eye & binocularly Eligibility
Criteria P Phase 2 Enrollment Completed Feb to May 2021 – 150 Subjects Reported Topline Results End of 2Q21
Primary Endpoint: % of Subjects ≥ 15 Letter Gain in Photopic DCNVA at 1 Hour Primary Endpoint Was
Significantly Met for Nyxol + LDP Gaining ≥ 15 Letters Near Vision VEGA-1 Phase 2 Trial p=0.006 30% Placebo Adjusted Responders Source: VEGA-1 TLR Table 14.2.1.2 % of Subjects With Improvement From Baseline in Photopic DCNVA by Time
Point (PP Population). 15 letters is 3 lines and 10 letters is 2 lines. Note: PP population differs from mITT by only one subject; results were essentially identical. P
Secondary Endpoint: % of Subjects ≥ 15 Letter DCNVA Gain At All Timepoints Source: VEGA-1 TLR Table
14.2.1.2 Percent of Subjects with Improvement From Baseline in Photopic DCNVA by Time Point (PP Population). 15 letters is 3 lines. Nyxol + LDP Had Strong Response with ≥ 15 Letter Near Gain from 30 Minutes to 6 Hours Durable benefit over
6 hours Rapid onset of efficacy Similar Efficacy was Observed In:Study Eye and Fellow EyeLight and Dark Irides P Nyxol alone effect (~12 hr before Time 0) VEGA-1 Phase 2 Trial
2nd Endpoint: % of Subjects ≥ 15 Letter Gain In Near & ≤ 5 Letter Loss In Distance Source: VEGA-1
TLR Table 14.2.2.2 Percent of Subjects with >= 15 Letters of Improvement in Photopic DCNVA and < 5 Letters of Loss in Photopic Binocular BCDVA by Time Point (PP Population) Phase 3 Approval Endpoint Confirmed Greater Efficacy of Combo
over Components at Multiple Timepoints p=0.0009 Statistics Compared to Nyxol+LDP armPowered for comparison to placebo whereas comparison to component arms were designed to inform the Phase 3 sample size VEGA-1 Phase 2 Trial P Even
with a small sample size, combination arm provided statistically meaningful results at 30 min and 2 hours vs. LDP and Nyxol alone arms
Secondary Endpoint: 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 Pupil Size ~2mm in Nyxol+LDP Consistent with 3-line Improvement in Near Vision Nyxol+LDP arm statistically significant compared to all arms
**p<0.01***p<0.0001 VEGA-1 Phase 2 Trial P
Secondary Endpoint: Safety Findings Source: VEGA-1 TLR Table 14.3.1.1 Overall Summary of Treatment
Emergent Adverse Events (TEAE) (Safety Population)Table 14.3.1.3 Treatment-Emergent Adverse Events (TEAE) by System Organ Class, Preferred Term, and Severity (Safety Population) Nyxol+LDP Combination Was Well Tolerated with a Favorable
Safety Profile Placebo Alonen=45 Nyxol Alonen=30 LDP Alonen=31 Nyxol+LDPn=44 Total Treatment Emergent Adverse Events (n) 4 18 13 50 TEAEs by Severity (n [%])MildModerateSevere 1 (2.2%)1 (2.2%)0 (0%) 6 (20%)0 (0%)0
(0%) 6 (19.4%)0 (0%)0 (0%) 13 (29.5%)1 (2.3%)1 (2.3%) AEs Occurring in ≥ 5% of subjects (n [%])Instillation Site Pain (Mild)Instillation Site Erythema (Mild)Conjunctival Hyperemia (Mild)Eye Disorders (Mild) 1 (2.2%)0 (0%)0
(0%)1 (2.2%) 3 (10%)3 (10%)2 (6.7%)2 (6.7%) 0 (0%)2 (6.5%)0 (0%)4 (12.9%) 4 (9.1%)5 (11.4%)2 (4.5%)5 (11.4%) No deaths, no serious AEs Almost all AEs were mild0% headaches or brow aches reported for Nyxol+LDP arm≤ 5% mild,
transient conjunctival hyperemia AEs in Nyxol+LDP armDistance vision: 100% Nyxol + LDP arm had ≤ 5 letter distance loss in photopic lighting (95% in mesopic)No change in IOP Conjunctival Hyperemia CCLRU Scale for Reference Nyxol + LDP
and LDP alone Only transient 0.5 point mean increase P
Product Attributes* Nyxol+LDP compared to market leader Efficacy (all time-points) ✓+ Safety:
Maintain Distance Vision (especially at night) ✓+ Safety: Tolerability (no headaches) ✓+ Durability (at least 6 hours) ✓+ Fast Onset (within 30 mins) ✓+ Convenience (daily drops) ✓ Tunable Pupil Modulation ✓+ ASCRS (July 2021)
Abstract# 76645 (Phase 2) and 74336 (Phase 3)✓+- Indicates better compared to market leader✓- Indicates comparable to market leader Potential ‘Best in Class’ Presbyopia Drop Nyxol+LDP Combination Outperforms Market Leader in Efficacy,
Safety, Durability and Onset Gemini I Phase 3: AGN-190584 Provides Rapid Improvement of Distance Corrected Near Vision That Is Maintained in Participants with Presbyopia- ASCRS 2021 Presentation Gemini I Phase 3: Safety and Efficacy of
AGN-190584 in Participants with Presbyopia- ASCRS Abstract# 74336 Phase 2 Clinical Trial To Evaluate The Efficacy of Phentolamine Ophthalmic Solution And Low-Dose Pilocarpine For The Treatment of Presbyopia- ASCRS Abstract# 76645
Phase 3 Phase 2 Phase 1 Presbyopia Eye Drops Competitive Landscape Corporate Websites,
Grzybowski, A, Markeviciute A, Zemaitiene R. A Review of Pharmacological Presbyopia Treatment. 2020 Validation of Pupil Modulating Drops Achieving Pin-Hole Effect & Efficacy, Many with Pilocarpine Pupil modulation MOA Combination
drugs Soften lens MOA Phase 3 Phase 2 Phase 1 Allergan(AGN-190584;1.25% pilo) Orasis(CSF-1; Low dose pilo) Ocuphire(0.75% Nyxol + 0.4% pilo) Visus(Brimochol®; brimonidine + carbachol) Other Cholinergic
Agonists* CholinergicAgonist* (pilocarpine) Lenz(PRX-100; aceclidine) Eyenovia(MicroLine;1 or 2% pilo) Novartis(EV-06) Alpha Antagonist& pilocarpine* P NDA *act on sphincter and ciliary muscles in dose-dependent
manner Ocuphire is differentiated by using both the dilator and sphincter muscles moderately to reach a pin-hole pupil size Presbyopia Next Steps:Advance into Phase 3 Registration Trials in 2022; Towards a Potential NDA in 2023
Nyxol® Phentolamine Mesylate NVD P Presbyopia RM Night Vision Disturbances Reversal of
Mydriasis 0.4% +
Moderate-to-Severe NVDs US Patients Night Myopia 10.8M Cortical
Cataracts 4.1M Post-LASIK 500k Post-IOL Implant 300k Total ~16M Night Vision Disturbances (NVD) – Chronic Opportunity Peripheral imperfections scatter light when pupils enlarge in dim light, causing halos, starbursts, and glare that
impair visionThe imperfections may be caused by LASIK surgery, IOL implants, certain types of cataracts (cortical), and natural reasons (especially with age) Symptoms cannot be properly corrected by any type of lens (reading glasses, contact
lenses) or surgical procedures Source: GlobalData Market Research Report, 2020 The Problem Imperfections in the Eye Affect Night Vision in Millions No Currently Approved Therapies NVD 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, aged 42
Night Vision Disturbances (NVD) – Chronic Opportunity Moderate Decrease in Pupil Size for scattered
light gets blocked by the iris Clinical Effect to potentially improve low contrast night vision as seen in trialsTolerable with a minimal side effect profile Convenient and Durable with chronic once-daily evening dose Nyxol’s Potential
Differentiated Solution Peripheral Optical Imperfections Allowing Pupil Modulation as a Solution After Before Once there is a drug and a category, that’s when they start looking for the disease. Physician KOL Seeking Treatment
Findings Patients willing to try a new eye drop treatment 67% Patients avoiding driving at night 25% NVD
NVD LYNX-1 Phase 3 Registration Design Ongoing 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 visual acuity (Day 8)Secondary (Days 8 & 15):Pupil diameterVisual acuity measures (distance and near)Safety and
tolerability (redness) Endpoints Eligibility Screening Randomization 1:1 daily evening dose(14 days) daily evening dose (14 days) 0.75% Nyxol Placebo 15 US sites~160 patients with NVD Phase 3 Initiated in Late 4Q20 Top Line
Expected Early 2022 NVD
ORION-1 Phase 2 Trial NYX-SNV Phase 2 Trial Nyxol Demonstrated Clinical Effect in NVD Key
Endpoints Observed in Multiple Phase 2 Trials Improved Low Contrast Distance Visual Acuity* Source: NYXG-201 Durable > 24-hour Pupil Modulation Effect % of Eyes with Mesopic Low Contrast Visual Acuity Improvement Pupil Diameter Change
from Baseline in Mesopic Conditions (Study Eye) Source NYX-SNV Percent of Subject Eyes p=0.029 p=0.04 p=0.16 Baseline Pupil Diameter: Placebo 4.6mm, Nyxol 4.7mm *NYX-SNV trial was small and not designed for a statistical 3-line
improvement in low-contrast visual acuity; the ~20% effect was used for powering and sizing of Phase 3 trial p = 0.0002 p = 0.0001 p = 0.0004 NVD
APX3330 APX3330 DR Diabetic Retinopathy DME Diabetic Macular Edema wAMD Wet Age-Related
Macular Degeneration
Diabetic Eye Opportunity DR ~7.7M Patients DME ~750K Patients Diabetic Retinopathy & Macular
Edema Diabetic retinopathy (DR) and diabetic macular edema (DME) are a leading cause of vision loss worldwide, especially in working age adults in developed countriesDiabetes damages small blood vessels within the eye causing leakage, oxygen
starvation, and abnormal vessel growth, which can obstruct visionDR patients are not commonly treated with approved injectable anti-VEGF drugs given earlier stage of retinal disease and many are asymptomaticDR progresses in steps and may
result in vision loss if left untreatedCurrent treatment for DME: 25% non-responders and 50% partial responders to anti-VEGF drugs Sources: Global Market Insights Report 2019-2025, Market Watch 2019 Report, Gene.com Retinal Diseases Fact
Sheet The Problem Non-Injectable Alternative Therapies are Needed for Earlier Stages of Disease Injectable Anti-VEGF Approved TherapiesNot Commonly Used for NPDR “Cotton Wool” Spots Aneurysm Hemorrhages Diabetic Eye Abnormal Blood
Vessels DR DME
APX3330 History and Ref-1 Inhibition Mechanism Ref-1 Involved in Multiple 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 Factors(Signaling
Cascade) TNF-αChemokines Neovascularization Lucentis®EYLEA® Anti-VEGF Steroids APX3330 Logsdon et al (2018), Li et al (2014). APX3330 is a small molecule oral drug candidate and a first-in-class inhibitor of Ref-1Ref-1
(reduction-oxidation effector factor-1) is a novel target discovered and characterized by Dr. Mark R. Kelley at Indiana University School of MedicineAPX3330 previously developed by Eisai for multiple hepatic inflammatory indications and later
by Apexian for advanced solid tumorsSimilar oncology origin as approved anti-VEGFsMOA uniquely decreases both abnormal angiogenesis and inflammation by blocking pathways downstream of Ref-1 DR DME
APX3330 Down-Regulates VEGF Protein and Anti-Inflammatory Cytokines In Vivo and In Vitro Evidence of
APX Dual Pathway Mechanism of Action Treatment of APX3330 (10mg/kg, oral gavage) in rats with type 1 diabetes and induced stroke shows a significant decrease of VEGF signaling. Increased VEGF is a hallmark of uncontrolled neovascularization
and inflammation in diabetic retinopathies; current approved treatments successfully decrease VEGF levels in the eye. Tao Yan et al. APX3330 Promotes Neurorestorative effects after stroke in type one diabetic rats. Aging and Disease. Vol 9,
Oct 2018Apurinic/Apyrimidinic endonuclease 1 regulates inflammatory response in macrophages. Jedinak A, Dudhgaonkar S, Kelley MR, Sliva D. Anticancer Res. 2011 Feb;31(2):379-85. PMID: 21378315 In vitro APX3330 suppresses pro-inflammatory
cytokines in LPS stimulated murine macrophage cell lines known to be involved in macular degeneration:TNF-α is a potent cytokine that enhances secretion of VEGF-A and VEGF-B by human choroidal fibroblast cells. J Cell Physiol. 2011Genetic
ablation of IL-6 led to significant suppression of AMD (murine CNV model). Am J Pathol. 2007 APX3330 Reduces VEGF Protein in the Brain of Preclinical Models APX3330 Reduces Pro-inflammatory Cytokines in Murine Cell Lines Involved in Macular
Degeneration DR DME
APX3330 Generally Well Tolerated with Clinical Signals 1. Silva et al. Oral APX3330 treatment reduces
L-CNV lesions in preclinical mouse model and confirms Phase 2 DR/DME clinical dose with sufficient distribution to human retina using PBPK modeling. Presented at the ARVO 2021 Annual Meeting Observations from Pre-Clinical Studies and 11
Clinical Trials of APX3330 DR DME Lesion Size and Corresponding Fluorescent Stains in L-CNV Models Treated with APX3330 at 25 mg/kg oral gavage -55% L-CNV Mouse Retina Model APX3330 Reduces Neovascularization Similar to Eylea in
Preclinical Models Silva et al, 2021 Vehicle 25 mg/kg 50 mg/kg APX3330 Gavage OCTLesion Volume Human Pharmacokinetics of APX3330 at 120 mg/day Phase 1 Clinical Trials Human Drug Exposure Multiple Times Higher than Mouse
Efficacious Levels Human 120 mg/day Mice 25 mg/kg Source: Unpublished Data 300 mg BID (600 mg/day total) dosing strategy for APX3330 is predicted to reach retinal AUC concentrations of 15.4 μg/mL; Significant APX3330 reaches human retina,
folds greater than mice who were given APX3330 25 mg/kg (actual 2hr conc. in retina 0.1 μg/mL)1
APX3330 Product Candidate Profile First-in-Class Ref-1 Inhibitor Phase 2 Ready for Retina Diabetic
Indications Improving Eye Health in Diabetics ↓ Inflammation ↓ Hypoxia Signaling ↓ Abnormal AngiogenesisEnhance Compliance & ExposureOral pill may reduce the burden of frequent anti-VEGF injections Few Systemic Adverse Effects~1% Mild
Gastrointestinal (diarrhea)~1% Mild Skin Rash (reversible)Lack of Significant Acute Neurologic, Cardiovascular, Liver, or Pulmonary toxicityNo Topical EffectsNo observed ocular AEs Safety Data Expected Efficacy Data APX3330: 600mg Oral
Dose Twice a day dosing of APX3330 being developed to providesteady state effectiveness with a tolerable chronic safety profile DR DME
DR/DME ZETA-1 Phase 2 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) Ongoing, Randomized, Double-Masked, Placebo-Controlled 24-Week Trial (Similar to Eylea P3 for
Approval) ZETA-1 Eligibility Screening Randomization 1:1 Twice daily oral dose (24 weeks) Twice daily oral dose (24 weeks) APX3330 600mg Placebo 15 US sites~100 patients with moderate-to-severe NPDR and mild PDR DR DME Primary:
% of subjects with a ≥ 2 step improvement on the DRSS (Diabetic Retinopathy Severity Scale) score at week 24Secondary:Central subfield thickness (CST)BCDVA (ETDRS)Safety and tolerabilityExploratory:Labs / PK Endpoints Phase 2 Start
Initiated in April 2021 Top Line Expected in 2022
Diabetic Retinopathy & Macular Edema Potential First Oral Therapy to be used as an earlier
intervention for the diabetic eye before vision symptoms appear or as add-on therapy to current anti-VEGF treatmentProven Novel Mechanism that may decrease both inflammation and VEGF activityConvenient option for patients to potentially
alleviate the burden of injections and increase complianceTolerable as seen in 11 completed Phase 1 and Phase 2 clinical trials EYLEA Product Pamphlet APX3330’s Potential Differentiated Solution APX3330 to Treat Patients Before Vision Loss
Occurs DR DME DR DME
Boards and Milestones
Richard Lindstrom, MDUniversity of Minnesota Ed Holland, MDLoyola University Chicago Jay Pepose,
MD, PhDUCLA Jack Holladay, MDUniversity of Texas Thomas Samuelson, MDUniversity of Minnesota Paul Karpecki, ODIndiana University Eliot Lazar, MDGeorgetown University Gary Novak, PhDUC Davis Marguerite McDonald, MDColumbia
University David Boyer, MDChicago Medical School Gerald Horn, MDUniversity of IllinoisCo-Founder Ocularis/NyxolPast MAB Member Mark Kelley, PhDIndiana UniversityCo-Founder Apexian/APX3330 Prestigious Ocular Medical Advisory
Board Fortunate for the Insights of Leading KOLs & Drug Candidate Co-Founders elCON Medical Michael Allingham, MD, PhDUniversity of North Carolina Richard Messmann, MDWayne State UniversityCMO Apexian/APX3330 Peter Kaiser, MDHarvard
Medical School Jeffrey Heier, MDBoston University
Ocuphire Board of Directors Seasoned Directors with Decades of Drug Development, M&A/Financings,
and Ophthalmology Sean Ainsworth, MBALead Independent Director, Board Director James Manuso, PhD/MBABoard Director Jay Pepose, MD, PhDBoard Director Richard Rodgers, MBABoard Director Susan Benton, MBABoard
Director Cam Gallagher, MBAChair, Board Director Mina Sooch, MBAVice-Chair, Board Director President & CEO
2021 to 2022 Ocuphire Cadence of Milestones Multiple Data Catalysts On Path To NDA(s) Ongoing
partnering discussions with leading ophthalmic companies (including European and Asian players) Completion of APX3330 LicenseARVO 2020 Presentation for MIRA-1 & ORION-1FDA EOP2 Meeting May 2020Completion of Transaction (Nasdaq: OCUP) and
concurrent $20M financingInitiate Phase 3 RM TrialInitiate Phase 3 NVD TrialComplete Nyxol Market ResearchJournal Publications Enrollment of Phase 3 RM TrialInitiate Phase 2 Presbyopia TrialReport Positive Phase 3 Data for RMInitiate Phase 2
DR/DME TrialEnrollment of Phase 2 Presbyopia TrialNew Patent ClaimsClosed $15M registered direct offeringReport Positive Phase 2 Data for Presbyopia ASCRS 2021 Presentation for MIRA-2 & VEGA-1Initiate 2nd P3 RM and Pediatric RM trial for
NDAEnrollment of Phase 3 NVD TrialEnrollment of Phase 2 DR/DME TrialIndustry Conferences & PublicationsManufacture 3xRegistration Batches for Nyxol Blow-Fill-Seal (BFS) Eye DropsComplete 6-month Rabbit Tox Study Report Phase 3 Data for
NVD Report 2nd Ph3 RM Trial Report Pediatric RM trial Initiate 2 Phase 3 Presbyopia TrialsReport Phase 2 Data for DR/DMEInitiate Chronic Ph3 Safety Trial (Nyxol /LDP)Complete 1 year CMC stability on 3xreg batchesSubmit Nyxol NDA filing for RM
in late 2022 Manufacture Commercial Batches of Nyxol Eye Drop 2020 1H 2021 2H 2021 2022* 2023* Report Phase 3 Data for Presbyopia TrialsPotential NDA for Nyxol in RMPotential Commercial Launch of Nyxol in USSubmit NDA filing for Nyxol
for Presbyopia in 2023 *Additional Studies for NVD and DR based on Data Readouts
OCUP – Market Snapshot Source: FactSet Sufficient Cash Runway Into Late 2022 and Substantial Trading
Liquidity Ticker OCUP Price $4.89 As of 8-10-21 Market Cap $82 M As of 8-10-21 Primary/FD Shares 16.9 M / 18.6 M As of 6-30-21 Cash $24.2 M As of 6-30-21 Cash Runway Into Late 2022 Guidance as of 6-30-21 Average Daily
Volume 930,341 Month-to-date at 8-10-21 Short Interest 1.1% As % of float; as of 7-31-21 Research Analyst Coverage on OCUP James Molloy Alliance Global Partners John Newman Canaccord Genuity Kristen Kluska Cantor
Fitzgerald Prakhar Agrawal Jones Trading
www.ocuphire.comir@ocuphire.com
NVD Endpoint: 5% Low Contrast Visual Acuity (LCVA) Chart Precision Vision FDA Accepted Endpoint for
Contrast Sensitivity Assessment Before Treatment* 3 Lines Improvement Illustration Primary Endpoint of Nyxol LYNX-1 Trial Percent of subjects with ≥ 3 lines of improvement in mesopic low contrast best-corrected distance visual acuity
(7 days) * Inclusion Criteria includes subjects with baseline mesopic LCVA of 20/100 or worse
DR/DME Endpoint: Diabetic Retinopathy Severity Scale (DRSS) EYLEA Product Pamphlet ® FDA
Accepted Endpoint for DR (EYLEA® in PANORAMA Pivotal Trial) Patients included in the ZETA-1 Trial Primary Endpoint of APX3330 ZETA-1 Trial Percent of patients with a ≥ 2 step improvement on the DRSS score at week 24 A 13-point Scale
Outlining the Various Stages of Diabetic Retinopathy DRSS Score 1 (10) 2 (20) 3 (35) 4 (43) 5, 6 (47, 53) 7 – 13 (60, 61, 65, 71, 75, 85,90) Description DR Absent Micro-aneurysm only Mild NPDR Moderate NPDR Moderately Severe
NPDR PDR – Mild, Moderate, and Severe Retinal Image Healthy blood vessels with no bulges Small bulges in blood vessel walls as well as other signs in the retina More changes in the blood vessels in the retina and small
spots of blood can become more visible More blood vessels in larger areas of the retina show changes Many of the blood vessels in the retina show visible changes Increased growth of new, damaged blood vessels