OIS@SECO Presentation on March 4, 2020

Restore Vision & Clarity

Mina Sooch, MBA CEO and Co-Founder

Late Clinical Stage Biotech Targeting

Large, Unmet Ophthalmic Markets

Decades of Clinical Data & Safety Profile

on 2 Acquired Eye Assets

Significant IP Portfolio &

Small Molecule CMC Advantages

Multiple Phase 3 & Phase 2 Data

Catalysts in Next9-18 Months

Experienced Management, Board, and Medical Advisors

2

Two Completed Nyxol® Phase 2b

Trials in 2019

Nyxol® APX3330

Ocuphire SummaryExecuting on a Vision to Advance Late-Stage Ophthalmic Drug Candidates

Large Unmet Opportunities for the Aging EyeDeveloping Drugs to Treat Both Front and Back of the Eye Diseases

BackFront

Diabetic Macular EdemaU.S. Prevalence: 750K

Diabetic RetinopathyU.S. Prevalence: 7+M

PresbyopiaU.S. Prevalence: 110+M

Night Vision DisturbancesU.S. Prevalence: 15-20M

Reversal of Mydriasis80M In-Office Eye Dilations Per Year

Nyx

ol®

AP

X33

30$4-50B US Markets $4-10B US Markets

3

Patents

to

7Phase 1 &

Phase 2

Trials

Up to

28 Days

Exposure in

Humans

>150Subjects

Dosed

APX3330Nyxol®

Extensive Development on Both Drug CandidatesWell-Controlled Trials Provide Foundation for NDA Path

505(b)(2) Development

Pathway

Studied in ocular refractory diseases (NVD) & elderly glaucoma patients

2034+

Patents

to

11Phase 1 &

Phase 2

Trials

More than

365Days

Exposure in

Humans

>400Subjects

Dosed

NCEDevelopment

Pathway

Studied in inflammation/hepatitis & cancer patients

2034+

4

Unique Mechanism of Action for Nyxol®First-in-Class Ophthalmic Formulation with 505(b)(2) Path

• Active in Nyxol® is a previously approved drug phentolamine mesylate (PM)

• PM is a non-selective a1 & a2 blocker

• PM is a lipophilic, small molecule allowing once-daily dose

• Nyxol® has been formulated as a novel, topical eye drop (patents thru 2034 in US/EU/Japan)

Reduces Pupil Size via

5

Relaxes (Vasodilation) via

α1

Smooth MuscleBlockade

(transient, mild redness is an on-target α1 effect

on sclera vessel)

α1Iris Dilator

Blockade

Reduces IOP via

α1: Reduce episcleral venous pressure (EVP)

α2: Increase TM outflow by relaxing contraction of TM

α1: Increase UV outflow and decrease humor production

Nyxol® Product Candidate ProfileFirst-in-Class Alpha 1/2 Blocker Eye Drop Phase 3 Ready for Refractory Indications

Phase 2 Safety Data

↓ Pupil Size (moderate miotic)

↑ Contrast Sensitivity (night)

↑ Near Visual Acuity (light/dark)

↑ Distance Visual Acuity

Improving Vision

Phase 2 Efficacy Data

No Systemic EffectsNo Changes in Blood PressureNo Changes in Heart Rate

Tolerated Topical EffectsMild / Transient / Reversible Eye Redness

IOP Unchanged or Decreased↓ Intraocular Pressure (IOP)

at Normal Baseline

Chronic daily dosing of Nyxol® before bedtime demonstrated no significant daytime redness and durability of effects more than 24 hours

Nyxol®: Phentolamine Mesylate 1% Ophthalmic SolutionPreservative Free and EDTA Free

6

Reversal of Mydriasis (RM) is an Unmet Market Opportunity Single Use Indication Leveraging a Precedent Approval Pathway

The Problem

• After every comprehensive annual eye exam and specialty exams, your pupils become dilated, impairing vision for 4-8 hours

• High sensitivity to light and inability to focus makes reading, working, and driving difficult

Nyxol’s PotentialDifferentiated Solution

• Drug Precedent: Rev-Eyes (a specific alpha 1 blocker) was approved by the FDA in 1990 and later discontinued (not for safety or efficacy reasons)

• Clinical Effect: Nyxol® can reduce pupil size and reverse mydriasis by counteracting the drugs (alpha agonists and cholinergic blockers) used to dilate the pupil

• Convenient: Eye drop given at the office allows your vision to return to normal sooner

7

>80M Dilated eye

exams per year

>4MOphthalmic

surgical dilations per year

Reversal of Mydriasis

US WW

Total Patients 84M 200M

Sales Potential ~$50-100+M ~$50-100+M

Assume Private Cash Pay (One-Time)

US $400-800M Market Size

MIRA-1 Trial: Nyxol® Phase 2b in RMRandomized, Double-Masked, Placebo-Control Crossover Trial in 2019

Randomization (1:1)

• 1% • placebo

Mydriatic Agent A(2.5% Phenylephrine)

n=16

Mydriatic Agent B(1% Tropicamide)

n=16

1 drop after

1 hour

1 drop after

1 hour

• 18 years and older (average age 28, 40% male / 60% female)• Otherwise healthy subjects with no pre-existing ocular

conditions/procedures• Protocol based on Rev-Eyes

Inclusion/Exclusion Criteria

• Primary: Mean change in pupil diameter from mydriatic max in different timepoints (30min, 1h, 2h, 4h, 6h)

• Secondary:• Return to Baseline Accommodation• Safety and tolerability (redness)

Endpoints

1%

placebo

Treatment

Mydriatic Agent B(1% Tropicamide)

n=16

Mydriatic Agent A(2.5% Phenylephrine)

n=16

1 drop after

1 hour

1 drop after

1 hour

Treatment

Day 1 Day 8

Eligibility Screening

4 US sites

32 subjects

Clinical Sites

MIRA-1 (NCT04024891)

Montaquila – West Bay Eye Associates – RIKarpecki – Kentucky Eye Institute – KY

32/32 Patients Enrolled

(8/12/19-9/17/19)

Foster – Athens Eye Care – OHKannarr – Kannarr Eye Care – KS 8

Positive Data Readout

November 2019

Nyxol® Demonstrated Clinical Effects in RMObservations from MIRA-1 Phase 2b Trial

9

Efficacy

✓

Primary Endpoint Successfully Met

Significant difference between Nyxol® compared to placebo in the change from max pupil diameter (PD) at 2 hours post-treatment (p<0.0001; ranked ANCOVA)

✓

✓

Key Secondary Endpoints Met

Nyxol® significantly returns more subjects to their PD baseline by 2 (p=0.016) and 4 (p<0.0001) hours

Nyxol® returned more subjects to their baseline vision (accommodation) at 2 hours as compared to placebo (p=0.008) after dilation with tropicamide

Safety

✓Well-tolerated with mild-moderate redness in the first few hours; no LUMIFY® needed

✓ No burning, no other AEs, no SAEs

✓Works with both parasympatholytic (tropicamide) and adrenergic (phenylephrine) mydriatic agents

Phase 2b MIRA-1 Results Accepted for Presentation at ARVO 2020 Conference“Phentolamine ophthalmic solution reduces time to recovery of medically-induced mydriasis in a Phase 2 trial”

Nyxol® Comparison to Discontinued Rev-EyesA Potential Tolerable, Efficacious and Convenient Drop for Patients & Physicians

Rev-Eyes Product Label → Nyxol Potential LabelRev-Eyes is indicated in the treatment of iatrogenically induced mydriasis produced by adrenergic(phenylephrine) or parasympatholytic (tropicamide) agents. Rev-Eyes is not indicated for thereduction of intraocular pressure or in the treatment of open angle glaucoma.

Nyxol® Drug Candidate

Rev-Eyes

Burning / Stinging NONE SIGNIFICANT

Eye Redness Mild-Moderate Severe

Stable Eyedrop Formulation

YES NO (mixed at site)

Cost Effective YES NO

Efficacy: Pupil Diameter Change / Return to Baseline

YES @ 2 hrs YES @ 1-2 hrs

Vision Performance: Return to Accommodation

YES @ 2 hrs(tropicamide)

YES @ 2 hrs(tropicamide)

Dosing 1-2 Drops 4 Drops

MIRA-1 Trial FDA Registration Trials*

*Rev-Eyes FDA Registration Trials include D2-2 (n=40) and D2-3 (n=40); additional safety trials include D1-1 (n=32), D6-1 (n=38), D7-1 (n=31), and over 400 subjects across 6 open studies (D3-1 to D3-6) with 24 hour safety. Rev-Eyes was taken off market by the sponsor, not the FDA, for reasons unrelated to the unmet need.

• Rev-Eyes demonstrated safe and rapid reversal of mydriasis produced by phenylephrine and to a lessor degree tropicamide.

• Note: Eye color affects the rate of pupillary constriction. In individuals with brown irides, the rate of pupillary constriction may be slightly slower than in individuals with blue or green irides.

10

Prestigious Ocular Medical Advisory BoardFortunate for the Insights of Leading KOLs and Drug Candidate Co-Founders

Richard Lindstrom MDUniversity of Minnesota

Ed Holland MDLoyola University Chicago

Jay Pepose MDUCLA

Jack Holladay MDUniversity of Texas

Thomas Samuelson MDUniversity of Minnesota

Paul Karpecki ODIndiana University

elCON Medical

Eliot Lazar MDGeorgetown University

Gary Novak PhDUC Davis

Marguerite McDonald MDColumbia University

David Boyer MDChicago Medical School

Gerald Horn MDUniversity of IllinoisCo-Founder Ocularis/Nyxol®

Mark Kelley PhDIndiana University SimonCancer CenterCo-Founder Apexian/APX3330

11

Ocuphire Pipeline and Upcoming MilestonesMultiple Phase 3 & Phase 2 Clinical Data Catalysts Expected in Next 9-18 Months

Product Candidate Indication

Development Stage

Anticipated MilestonesPre-clinical Phase 1 Phase 2 Phase 3

1% Nyxol®Eye Drop

Night Vision Disturbance (NVD)

Initiate Phase 3 LYNX-1 trial 1H2020;Data expected in 12 months (n=200)

1% Nyxol®Eye Drop

Reversal of Mydriasis (RM)

Initiate Phase 3 MIRA-2 trial 1H2020; Data expected in 9 months (n=60-100)

1% Nyxol® + Low-Dose Pilocarpine Eye Drops

Presbyopia (P)Initiate Phase 2 VEGA-1 trial 1H2020;Data expected in 15 months (n=100)

APX3330 Oral Pill

Diabetic Retinopathy (DR)/ Macular Edema (DME)

Initiate Phase 2 ZETA-1 trial 2020;Data expected in 18 months (n=60-100)

APX2009 Intravitreal

DR/DME Initiate IND enabling studies

Combo (1% Nyxol® + Latanaprost) Eyedrops

Glaucoma (14 to 22 mmHG)

Evaluate 2nd line open-angle / normal-tension glaucoma Phase 2 trial with partner

ORION-1 4Q19

ORION-1 4Q19

ORION-1 4Q19

MIRA-1 4Q19

APX-CLN-0011 2Q19

MIRA-2

VEGA-1

ZETA-1

LYNX-1

12 This presentation contains “forward-looking” statements that are based on our management’s beliefs and assumptions and on information currently available to management.

2 Phase 3 and 2 Phase 2 trial readouts over next 9 to 18 months

Global partnering opportunities for APX2009

and for Nyxol® late-stage development /

commercialization

Thank You

Restore Vision & Clarity

Contact us at msooch@ocuphire.comor visit www.ocuphire.com