Ocuphire Pharma Investor R&D Day

Ocuphire Pharma Investor R&D Day January 31, 2022

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 regulatory timelines, commercial timelines, cash runway, and future clinical trials in RM, presbyopia, NVD and DR/DME,

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) the success and timing of regulatory submissions

and pre-clinical and clinical trials, including enrollment and data readouts; (ii) regulatory requirements or developments; (iii) changes to clinical trial designs and regulatory pathways; (iv) changes in capital resource requirements; (v) risks

related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; (vi) legislative, regulatory, political and economic developments, (vii) changes in market

opportunities, (viii) the effects of COVID-19 on clinical programs and business operations, (ix) the success and timing of commercialization of any of Ocuphire's product candidates and (x) the maintenance of Ocuphire's intellectual property

rights. 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 forward-looking statements contained in this presentation speak only as of the date on which they were made. The Company

undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. The Company makes no representation or warranty, express or implied, as to the accuracy or

completeness of the information contained in or incorporated by reference into this presentation. Nothing contained in or incorporated by reference into this presentation is, or shall be relied upon as, a promise or representation by the Company

as to the past or future. The Company assumes no responsibility for the accuracy or completeness of any such information. This presentation also contains estimates and other statistical data made by independent parties and by us relating to

market shares and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. The trademarks included herein are the property of the owners thereof

and are used for reference purposes only. Such use should not be construed as an endorsement of such products.

Ocuphire Investor R&D Day 1/31/22 Agenda & Speakers

Company Overview Presenter: Mina Sooch, CEO, Founder of Ocuphire Pharma

Ocuphire Pharma Restoring Vision and Clarity for Your Eyes, Today and Tomorrow

Ophthalmology - An Attractive Biotech Sector Demographics, M&A, Regulatory Approvals and Efficient Trials Favor Ophthalmic

Drugs Source:1.Endpoint Dec 29, 2021- Hitting a new record on drug approvals, the FDA offers a thumbs-up to another atopic dermatitis contender; OIS Year in Review 2021; Company press releases

Nyxol & APX3330: Drug Development History and Patents Source: Eisai and Apexian Data Significant Preclinical & Clinical

Data Supporting Human Safety, MOA, Efficacy, and PK

Ocuphire Pipeline & Clinical Milestones Multiple Phase 3 & Phase 2 Clinical Data Readouts Anticipated Over the Next Year

Differentiated, Late-Stage Pipeline for Front and Back of the Eye Nyxol with > 330 patients treated across 9 trials (505(b)(2)

regulatory pathway) APX3330 with > 340 patients treated across 11 trials (NCE development pathway) Nyxol and APX3330 achieved promising clinical data and favorable safety profile across multiple Phase 1, 2, and 3 trials Poised for

Commercial Success in Multiple Large Unmet Markets Addressing 4 large markets with unmet needs: RM, Presbyopia, NVD, and DR/DME Successful trial execution with 2 recent positive Phase 3 and Phase 2 data read-outs for Nyxol in RM and Nyxol + LDP

Presbyopia, respectively Stable, small-molecule drugs with commercial scalability Robust and growing IP portfolio: US and global issued thru 2034 for both assets as well as new 2039 Nyxol patent issued for presbyopia Many Catalysts in 2022

with Track Record of Execution $24.5 million cash reported at 12-31-21 sufficient for operations into 2Q 2023 Highly experienced management, Board and KOLs with broad ophthalmic and biotech drug development and commercialization success

Lower-cost, fast-enrolling, shorter-duration clinical trials Favorable, precedent regulatory environment for ophthalmic drug approval Analyst coverage by Cantor, Canaccord, Jones Trading, Alliance Global, and HCW

I. APX3330 Program Update

APX3330 Chemistry and MOA

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

Tao Yan et al. APX3330 Promotes Neurorestorative effects after stroke in type one diabetic rats. Aging and Disease. Vol 9, Oct

2018 -Apurinic/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 -Fehrenbacher, J. C., Guo, C., Kelley, M. R. &

Vasko, M. R. DNA damage mediates changes in neuronal sensitivity induced by the inflammatory mediators, MCP-1 and LPS, and can be reversed by enhancing the DNA repair function of APE1. Neuroscience 366, 23-35,

doi:10.1016/j.neuroscience.2017.09.039 (2017).

APX3330 VEGF Effects in Normal Cells Kamba 2007; Girardi 2010; Li 2014; APX3330 Investigator Brochure APX3330 Restores Normal

Levels Unlike Biologic Anti-VEGFs that Reduce VEGF Below Normal

APX3330 Preclinical & IND-Enabling Studies APX3330 Investigator Brochure Extensively Evaluated in Over 20 Studies by Large

Japanese Pharma Eisai

Preclinical Data: Oral APX3330 Blocks Neovascularization Silva et al. ARVO 2021 Annual Meeting *Published data on EYLEA. This

study was performed independently from APX3330 study and is a cross-study comparison. **Li 2014; *** Pasha 2018; ****Jiang 2011 (Vldlr -/- : Very Low-Density Lipoprotein receptor knock-out mice) Lesion Volume Decrease with Oral APX3330 in Murine

Laser CNV Model Similar to EYLEA Data

APX3330 Human PK and Safety Summary

Phase 1 Clinical Trials: PK Data Supporting the ZETA-1 Trial Apexian preclinical data (unpublished) APX3330 Investigator

Brochure Eisai PK clinical data APX_CLN_0002 APX3330 has Oral Bioavailability and a Sustained PK Profile

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 Eisai Preclinical Data Apexian preclinical data APX3330 Reaches the Retina via Oral Administration

Subject Exposure Across 11 Prior Clinical Trials * Eisai and Apexian Phase 1 and Phase 2 clinical trials (subject to final safety

database) Over 2000 Subject-Days of Exposure at 600 mg/day

Safety Summary From Phase 1 and Phase 2 Studies Phase 1 and Phase 2 Clinical Trials performed by Eisai and Apexian

APX3330 Product Candidate Profile for Multiple Retinal Indications Phase 1 and Phase 2 clinical trials by Eisai and Apexian

First-In-Class Ref-1 Inhibitor with Favorable Human Safety Data

APX3330 Addressing Unmet Needs in Retina

Clinical Unmet Need in Diabetic Retinal Diseases DR/DME are major causes of vision loss in working aged adults Diabetic

population expected to increase dramatically worldwide Approved therapies for DR are effective but require IVT injection DR patients are not routinely treated with approved injectable anti-VEGF drugs until they develop center-involved DME or

PDR - DR progresses resulting in vision loss Early, noninvasive intervention targeting DR represents a therapeutic unmet need The Problem

Diabetic Retinopathy is a Progressive, Vision Threatening Disease Source: Patient survey adapted from Lions International

Foundation and International Diabetes Foundation-Europe; Meltzer 2000 Losing Vision is Diabetic Patients Top Concern

Key Clinical Landscape in Diabetic Retinopathy (and DME)

ZETA-1 Phase 2b Clinical Trial (APX3330 in DR)

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) https://clinicaltrials.gov/ct2/show/NCT04692688?term=ZETA-1&draw=2&rank=1 Ongoing, Randomized, Double-Masked,

Placebo-Controlled 24-Week Trial (Similar To Eylea P3 DR Trial)

Key Eligibility Criteria in ZETA-1 INCLUSION Moderately-severe to severe NPDR or mild PDR in study eye as confirmed by reading

center BCVA > 20/63 in study eye EXCLUSION Retinopathy from causes other than diabetes in study eye Presence of center involved diabetic macular edema (DME) defined as a central

subfield thickness (CST) 320 m on SD-OCT or the presence of intra- or subretinal fluid within the central subfield Center involved DME in the fellow eye is allowed Prior treatment in study eye with focal/grid laser photocoagulation

within the past year, PRP at any time, systemic or intravitreal anti-VEGF agents within last 6 months in study eye HbA1c 12.0% Clinically significant systemic disease (e.g., uncontrolled diabetes, myasthenia gravis, cancer, hepatic, renal,

endocrine, or cardiovascular disorders) that might interfere as deemed by Investigator ZETA-1 Clinical Trial Given Bilateral Treatment with Oral, Patient Criteria Allows DME in Fellow Eye

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

Enrollment Update on ZETA-1 ZETA-1 Clinical Trial ~70% Completion of Enrollment in 24-week Phase 2b Trial

Comparison of Subject Exposure Before and After ZETA-1 *Interim Zeta-1 Data as of 01-12-22; * Eisai and Apexian Phase 1 and Phase

2 clinical trials (subject to final safety database) Subject-Days of Exposure at 600 mg/day Substantially Increases Exposure Data

Baseline Characteristics for ZETA-1 Trial (Interim) Source: ZETA-1 Demographics and Baseline Characteristics Typical Demographics

for Diabetic Population

Masked Safety Findings from Ongoing ZETA-1 Trial Source: ZETA-1 trial Favorable Safety Profile (as of 1/12/2022) Observed with

600 mg Oral Daily Doses

Takeaways: Masked Interim Safety Findings from ZETA-1 Trial ZETA-1 Clinical Trial

Broad Opportunities to Treat Retinal Diseases with APX Platform Potential First Oral Rx for Retina Diseases First-line earlier

intervention for the diabetic eye Add-on therapy to current anti-VEGF treatments to reduce intravitreal injection burden Proven Novel Mechanism May decrease both inflammation and angiogenesis Convenient Daily Regimen Favorable Oral Safety

Profile As seen in 11 completed Phase 1 and Phase 2 clinical trials Improve Patient Compliance Potentially alleviate the frequent burden of injections Potential Differentiated Solution APX3330 May Treat Patients Across Retinal Diseases as

Single Agent or Adjunctive Therapy

Large Global/US Market Opportunity in Retinal Disease Market Scope 2020 J Glob Health. 2019 Jun; 9(1): 010427 Tilahun M et. al,

Prevalence of Diabetic Retinopathy and Its Associated Factors among Diabetic Patients at Debre Markos Referral Hospital, Northwest Ethiopia, 2019: Hospital-Based Cross-Sectional Study. Diabetes Metab Syndr Obes. 2020;13:2179-2187 Retinal US

Markets Served by Anti-VEGF Injections Alone are Greater than $10B+ Today

What's Important? Eylea label; APX3330 Investigator Brochure, ZETA-1 clinical trial APX3330 has the Potential to be 1st Line of

Therapy for DR Patients

Summary of APX3330 Program

II. Nyxol Reversal of Mydriasis Overview

Reversing Dilations: Addressing an Unmet Need with 1 Blocker Nyxol

Problem: Dilated Eyes for Exams and Procedures Pharmacologically-induced pupil dilation is a necessary tool for routine

ophthalmoscopy but there is 6 to 24 hours of impaired vision including: Inability to Focus Photophobia (sensitivity to light) Cycloplegia (loss of accommodation) Difficulty Reading and Driving Halos and Glare 1. GlobalData Market

Research Survey; Oraverse and Regitine Label Patients Report Significant Side Effects after Dilated Eye Exam

Current Off-Label Landscape for RM 1. Optician (2012)- Mydriatic Drugs: Practical Considerations 2. Pilocarpine FDA Label (2017)

3. Lee DA, Higginbotham EJ, 2005. Glaucoma and its treatment: a review. Am J Health Syst Pharm 62, 691-699. Physicians AVOID Use of Cholinergic Agonists (Pilocarpine) Due to the Risk on Ciliary Muscle

Nyxol's Differentiated MOA as an Alpha-1 Blocker Ocuphire Phentolamine Mesylate Reformulated as a Proprietary Topical Eye Drop

Nyxol Product Candidate Profile Nyxol Clinical Trials Novel, Differentiated Alpha 1/2 Blocker Eye Drop for Refractive Indications

Nyxol Clinical Data for Reversing Dilations MIRA Program Evaluating Nyxol for the Reversal of Mydriasis Ocuphire Efficient

Clinical Programs have Positioned Ocuphire to Target NDA Filing in Late 2022