#1Ocuphire Restore Vision & Clarity MIRA-3 Phase 3 Trial Results Conference Call March 29, 2022#22 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 reversal of mydriasis (RM), presbyopia, night vision disturbance (NVD) and diabetic retinopathy (DR)/diabetic macular edema (DME), and the potential market opportunity in RM. 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 PHARMA IS HOSTING A VIRTUAL INVESTOR R&D DAY MONDAY, JANUARY 31ST, 2022 10:00AM - 12:00PM EST www.ocuphire.com Ocuphire OCUP NasdaqListed Nasdaq Ocuphire PHARMA#33 Agenda and Participants Second Phase 3 RM Trial Topline Readout as Planned in 1Q22 ● ● ● Highlights and Overview Topline MIRA-3 Phase 3 Clinical Trial Results for Nyxol in Reversal of Mydriasis (RM) Reversal of Mydriasis Market Opportunity Upcoming Milestones Q&A Participants Mina Sooch, MBA, President and CEO Jay Pepose, MD, PhD, Medical Advisory Board and Board Member Mitch Brigell, PhD, Head of Clinical Development Susan Benton, MBA, Corporate Board Member Bindu Manne, Head of Market Development and Commercialization Charlie Hoffmann, MBA, VP of Corporate Development and Operations Amy Rabourn, MAcc, VP of Finance Ocuphire PHARMA#44 Highlights and Overview Ocuphire PHARMA#5LO 5 Key Takeaways from Nyxol's MIRA-3 2nd Phase 3 RM Trial MIRA-3 Met Primary Endpoint Key Secondary Endpoints Met Statistical and Clinical Significance MIRA-2 and MIRA-3 topline data. Completed 2 Confirmatory FDA Registration Trials in RM MIRA-3 58% vs. 6% p<0.0001 ✓ MIRA-2 49% vs. 7% p<0.0001 On Track to File Nyxol NDA in RM in Late 2022 Ocuphire PHARMA#6Addressing Unmet Needs in Large Markets Significant Preclinical & Clinical Data Supporting MOA, Efficacy and Safety Refractive i Retina 10 Completed Phase 1, Phase 2, and Phase 3 Trials CO 6 >600 Subjects Dosed RM P NVD Exposure in Humans 28 Days Reversal of Mydriasis Presbyopia NyxolⓇ Novel a1/ a2 Blocker 505(b)(2) Night Vision Disturbances Patent Coverage 2034+ US Market Opportunity -$500 M $10B - $20B $2B-$4B 11 Completed Phase 1 and Phase 2 Trials >340 Subjects Dosed DR DME Source: Eisai and Apexian Data; GlobalData Market Research Report, 2020; Company Estimates for US Market Size; *Ocuphire internal estimates. APX3330 Oral REF-1 Inhibitor New Chemical Entity (NCE) Exposure in Humans 365 Days Diabetic Retinopathy Diabetic Macular Edema Patents to 2034+ US Market Opportunity $10+B Oral Rx Revenues* Ocuphire PHARMA#77 Ocuphire Pipeline & Clinical Milestones Multiple Phase 3 & Phase 2 Clinical Data Readouts Anticipated this Year Indication Reversal of Mydriasis (RM) Presbyopia (P) Dim Light or Night Vision Disturbances (NVD) Diabetic Retinopathy (DR)/ Macular Edema (DME) DME or Wet Age- Related Macular Degeneration (WAMD) Product Candidate NyxolⓇ Eye Drop Nyxol® Eye Drop Nyxol® + Low-Dose (0.4%) Pilocarpine Eye Drops NyxolⓇ Eye Drop APX3330 Oral Pill APX2009 (Intravitreal or Local Delivery) Pre-clinical Phase 1 Phase 2 VEGA-1 ✓ Phase 3 ✓ MIRA-3 MIRA-2 ★ MIRA-4 Regulatory Approval ★LYNX-1 ★ZETA-1 Recent Positive Trial Data Ongoing Trial Note: 0.75% Nyxol (Phentolamine Ophthalmic Solution) is the same as 1% Nyxol (Phentolamine Mesylate Ophthalmic Solution) Anticipated Milestones Reported MIRA-3 Phase 3 data in Q1 2022 (n=368) MIRA-4 Pediatric safety study data expected in 2Q 2022 (n=23) VEGA Phase 3 program planned to initiate in mid-2022 LYNX-1 Phase 3 data expected in 2Q 2022 (n=145) ZETA-1 Phase 2b data expected in 2H22 (n=103) Seeking partner funding for IND enabling studies and further development Ocuphire PHARMA#88 Nyxol's Differentiated MOA as an Alpha-1 Blocker Phentolamine Mesylate Reformulated as a Proprietary Topical Eye Drop →Nyxol™ Phentolamine Mesylate is the Active Ingredient in Nyxol: a Non-selective a1 & a2 Antagonist Blocking a1 Blocking a1 Reduces Pupil Size Dilates Blood Vessels Iris Dilator Muscle Iris Sphincter Muscle Nyxol blocks a1 receptors only found on the Iris Dilator Muscle Decreases Pupil Size (Moderate Miosis) without Affecting the Ciliary Muscle A Phentolamine mesylate is approved for 2 indications: ● Regitine® (Pheochromocytoma) - intravenous injection approved in 1952 OraVerse® (Reversal of oral anesthesia) - intramuscular injection approved in 2008 505(b)(2) Regulatory Approval Pathway Ocuphire PHARMA#99 CO Nyxol Product Candidate Profile Novel, Differentiated Alpha 1/2 Blocker Eye Drop for Refractive Indications Nyxol: 0.75% Phentolamine Ophthalmic Solution Preservative Free, EDTA Free, and Stable Favorable Safety Profile Efficacy Data Nyxol Improves Vision by Decreasing Pupil (~1-1.5mm) ↑ Near Vision ↑ Distance Vision ↑ Contrast Sensitivity (night) Nyxol Clinical Trials No Systemic Effects No Changes in Blood Pressure No Changes in Heart Rate Well-Tolerated Topical Effects Mild, Transient, Reversible Eye Redness IOP Unchanged or Decreased Minimal to No Headaches Durable Effects Last > 24 Hours Chronic daily dosing of Nyxol at bedtime reduces pupil size for up to 24 to 36 hours Ocuphire PHARMA#1010 NyxolⓇ for Reversal of Mydriasis (RM) (1 I had a premium cataract procedure by my MD, and I was unable to see clearly for two days. My doctor said it was due to my dilation. I did not expect my dilation to last that long. RM Fra I have to visit my retina MD for my monthly injections, where I am dilated. Being dilated every month is a huge burden on my day. "} I have to stay indoors. They say it only lasts a few hours, L }} but it lasts all day, and it is very annoying. Ocuphire PHARMA#11RM 11 Problem: Dilated Eyes for Exams and Procedures Patients Report Significant Side Effects after Dilated Eye Exam The Problem Pharmacologically-induced pupil dilation is part of standard care for annual and specialty eye exams.... ...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 Physician's Use of Mydriatic Agents¹ Cyclopentolate 5% Paremyd 9% Phenylephrine Alone 16% Tropicamide and Phenylephrine 18% Tropicamide Alone 52% Note - Tropicamide and Cyclopentolate have same MOA NO REVERSAL DROPS COMMERCIALLY AVAILABLE Ocuphire PHARMA#12RM 12 Nyxol Has Potential To Be The Only Option For RM Physicians AVOID Use of Cholinergic Agonists (Pilocarpine) Due to Safety Risk on Ciliary Muscle 2 Classes of Mydriatic Agents Phenylephrine (a1 agonist) Sympathetic (primarily a1) innervation stimulates the iris dilator muscles Tropicamide (anti-cholinergic) Parasympathetic innervation stimulates the iris sphincter and ciliary muscle Lens Cornea Pupil Iris Ciliary muscle Reversal via the Ciliary Muscle by Cholinergic Agonists* is Not a 'Safe' Option 1 Pilocarpine FDA Label (2017) 2. Optician (2012)- Mydriatic Drugs: Practical Considerations 3. Lee DA, Higginbotham EJ, 2005. Glaucoma and its treatment: a review. Am J Health Syst Pharm 62, 691-699. X X X Retinal tear has been reported in some patients, especially high myopes¹ Induces accommodation spasm and reduction in distance vision² Induced anterior shift of the lens can increase the risk of acute angle-closure glaucoma² X High incidence of brow ache and headache following installation³ * Cholinergic Agonists include pilocarpine, carbachol, and aceclidine. Note, pilocarpine is rarely used off-label for RM given these safety concerns. Nyxol® is the only eye drop in clinical development for multiple indications with a MOA that does not affect the ciliary muscle Ocuphire PHARMA#1313 Ocuphire PHARMA MIRA-3 Topline Phase 3 Results Randomized, Parallel Arm, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Solution) to Reverse Pharmacologically-Induced Mydriasis in Healthy Subjects#14RM 14 MIRA-3 Phase 3 Registration Trial Design Randomized, Double-Masked, Placebo-Controlled, Parallel, Multi-Center, One-Day Trial 16 US sites 368 subjects Screening 0.75% Nyxol 2 21 Placebo Randomization Mydriatic Agent A, B, or C Mydriasis -1 Hour Treatment (Max Dilation) 0 min Mydriatic Agent A, B, or C 1 Hr 30min Nyxol drop(s) (2 drops study eye, 1 drop fellow eye) Primary Endpoint 2 Hr 90min 3 Hr Placebo drop(s) (2 drops study eye, 1 drop fellow eye) 4 Hr Follow Up Visit MIRA-3 Started in Nov 2021 Enrolled 368 in Feb 2022 Phase 3 Results Reported March 2022 6 Hr 24 Hr Key Eligibility Criteria Inclusion: Healthy ≥ 12 years of age Exclusion: Clinically significant ocular trauma, surgery, or non-refractive laser treatment within the 6 months prior to screening; and recent or current evidence of ocular disease, infection or inflammation in either eye Mydriatic Agents 3:1:1-A: 2.5% phenylephrine (alpha-1 agonist), B: 1% tropicamide (cholinergic blocker), C: Paremyd® (combination) Endpoints Primary: % of subjects (study eye) returning to baseline (within 0.2 mm) pupil diameter (PD) at 90 min Key Secondary: % of subjects returning to baseline at Omin, 30min, 1h, 90 min 2h, 3h, 4h, 6h, 24h (overall, by mydriatic agent, by iris color) Mean time to return to baseline PD ● ● ● ● ● Mean change in pupil diameter at all timepoints Distance-Corrected Near Vision Accommodation (Tropicamide/Paremyd) Safety and tolerability Ocuphire PHARMA#15RM 15 Demographics Treatment and Placebo Arms Were Balanced in MIRA-3 Phase 3 Registration Trial Demographics Age (years): Mean (Range) Sex: Male n (%) Female n (%) Race: White n (%) African American n (%) Asian n (%) Other^ n (%) ^includes American Indian or Alaska Native; Native Hawaiian or Other Pacific Islander Light Iris Color: n (%) Dark Iris Color: n (%) Nyxol n=244 Source: MIRA-3 Table 14.1.2.1 (ARP) (MITT). 34 (12-80) 92 (37.7%) 152 (62.3%) 182 (74.6%) 38 (15.6%) 22 (9.0%) 0 (0%) 113 (46.3%) 131 (53.7%) Placebo n=124 36 (12-80) 59 (47.6%) 65 (52.4%) 93 (75.0%) 21 (16.9%) 9 (7.3%) 1 (0.8%) 58 (46.8%) 66 (53.2%) Total n=368 35 (12-80) 151 (41.0%) 217 (59.0%) 274 (74.5%) 59 (16.0%) 31 (8.4%) 7 (1.9%) 171 (46.5%) 197 (53.5%) Notes: 32 pediatric subjects 12-17years old were enrolled in the trial. Race is more than 100% given subjects could check more than one category. Demographics represent all randomized population (ARP) of 368 which is the same as Safety Population and Modified-Intent-to-Treat (mITT). Per Protocol (PP) Population is 345, excludes 23 subjects who did not dilate more than 0.2 mm 1 hour after receiving mydriatic drop. Ocuphire PHARMA#16RM 16 Baseline Characteristics Study Eye Treatment and Placebo Arms Were Balanced Across Ocular Measures in the MIRA-3 Trial Baseline Characteristic Baseline Pupil Diameter Mean (mm) Max Dilated Pupil Diameter Mean (mm) Accommodation Mean (diopters) BCDVA letters 55 letters = 20/20 DCNVA letters 70 letters = 20/20 IOP (mmHg) Source: MIRA-3 Table 14.1.2.1 (ARP) (mITT). Nyxol n=248 5.1 7.2 7.4 57 65 16.2 Placebo n=120 4.9 7.1 7.6 57 65 16.1 Total n=368 5.1 7.2 7.5 57 65 16.1 Ocuphire PHARMA#17RM 17 Primary Endpoint: 58% of Subjects' Study Eye Returned to Baseline at 90 Min Nyxol Statistically Better Than Placebo Starting At 1 Hour And All Subsequent Timepoints Percent of Subjects (%) 100% 80% 60% 40% 20% 0% 4% 4% 0.5 MIRA-3 Phase 3 Trial Percent of Subjects Returning to ≤ 0.2 mm of Baseline Pupil Diameter Study Eye (mITT) p<0.0001 2% 42% 1 p<0.0001 58% 6% p<0.0001 7% 66% p<0.0001 2 14% 79% p<0.0001 Source: MIRA-3 Table 14.2.1.1 (mITT). Data include all three mydriatics (Phenylephrine, Tropicamide, Paremyd). 17% 1.5 3 4 Time Post-Treatment with Nyxol/Placebo (Hours) Placebo (n=124) Nyxol (n=244) 86% p<0.0001 36% 91% 6 p<0.0001 72% 89% 24 Ocuphire PHARMA#18RM Percent of Subjects (%) 18 100% 80% 60% 40% 20% 0% Primary Endpoint Achieved in Two FDA Registration Phase 3 Trials Rapid, Consistent and Sustained Reversal of Pupil Dilation with Nyxol Percent of Subjects Returning to ≤ 0.2 mm of Baseline PD Study Eye (mITT) 4%4% 0.5 MIRA-3 Phase 3 Trial p<0.000 42% 2% 1 p<0.0001 58% 6% 1.5 p<0.0001 7% 66% 2 p<0.0001 79% 14% 3 p<0.0001 86% 17% 4 p<0.0001 p<0.0001 91% 89% 36% 6 72% 24 Placebo (n=124) Nyxol (n=244) Time Post-Treatment with Nyxol/Placebo (Hours) 100% Percent of Subjects (%) 80% 60% 40% 20% 0% 3% 1% 0.5 Placebo n=91 Percent of Subjects Returning to ≤ 0.2 mm of Baseline PD Study Eye (mITT) p<0.0001 90% p<0.0001 28% 2% 1 MIRA-2 Phase 3 Trial p<0.0001 49% 7% ■Nyxol n=94 1.5 p<0.0001 59% 11% 2 p<0.0001 80% 18% 3 p<0.0001 82% 30% 4 45% 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). 6 p<0.0001 92% 66% 24 Time Post-Treatment with Nyxol/Placebo (Hours) Ocuphire PHARMA#19RM 19 Comparison of One Drop (Fellow Eye) with Two Drops (Study Eye) Similar 52% of Subjects Return to Baseline at 90 Minutes with a Single Drop of Nyxol Percent of Subjects (%) 100% 80% 60% 40% 20% 0% 4% 4% 0.5 5% Percent of Subjects Returning to ≤ 0.2 mm of Baseline PD Fellow Eye (mITT) p<0.0001 28% 1 5% MIRA-3 Phase 3 Trial p<0.0001 52% 1.5 7% p<0.0001 65% p<0.0001 2 15% 75% p<0.0001 Source: MIRA-3 Table 14.2.1.1 (mITT). Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd). 11% 84% 3 Time Post-Treatment with Nyxol/Placebo (Hours) Placebo (n=124) ■Nyxol (n=244) p<0.0001 31% 90% p<0.0001 65% 91% 24 Ocuphire PHARMA#20RM Mean Pupil Diameter Over Time Nyxol Treatment Significantly Reduced PD Starting at 1 Hour Post-Dose Through 6 Hours Mean Pupil Diameter (mm) 9 8 7 6 5 4 3 p<0.0001 Max dilation; Mydriatic Treatment rT 0 -1 0.5 p<0.0001 Mean PD - Study Eye (mITT) p<0.0001 MIRA-3 Phase 3 Trial KH p<0.0001 Placebo (n=124) p<0.0001 1.5 2 3 4 Time Post-Treatment with Nyxol/Placebo Source: MIRA-3 Table 14.2.2.1 (mITT). The p-values are change from max pupil dilation treatment compared to placebo. 20 Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd). Standard Error bars are shown. -Nyxol (n=244) KH p<0.0001 6 p<0.0001 24 Ocuphire PHARMA#21RM Mean Pupil Diameter (mm) 21 9 8 4 Mydriatic 3 Mean Pupil Diameter Over Time by Mydriatic Agents Nyxol Reduced PD With All Mydriatic Agents; More Rapidly with Phenylephrine as Expected -1 p=0.001 H p<0.0001 0 0.5 Mean PD Phenylephrine ㅏㅓ Max Dilation; Treatment p<0.0001 1 1 p<0.0001 2 H p<0.0001 3 p<0.0001 4 Placebo (n=74) -Nyxol (n=146) 1 1.5 Time Post-Treatment with Nyxol/Placebo (Hours) 5 p<0.0001 6 MIRA-3 Phase 3 Trial Mean PD - Study Eye (mITT) p<0.0001 24 Mean Pupil Diameter (mm) 9 8 LO 4 Mydriatic 3 -1 Max Dilation; Treatment p<0.0001 Mean PD - Tropicamide or Paremyd Placebo (n=50) -Nyxol (n=98) 0 0.5 H p<0.0001 p<0.0001 H FH p<0.0001 p<0.0001 3 HA 1 1.5 2 Time Post-Treatment with Nyxol/Placebo (Hours) 4 5 Source: MIRA-3 Table 14.2.2.3. (mITT). The p-values are change from max pupil dilation treatment compared to placebo. Standard Error bars are shown. FH p<0.0001 6 p<0.0001 24 Ocuphire PHARMA#22RM Mean Pupil Diameter (mm) 00 22 A p<0.0001 p<0.0001 p<0.0001 Max dilation; Treatment CO 4 Mean Pupil Diameter Over Time by Eye Color Nyxol Reduced Pupil Diameter Rapidly in Both Light and Dark Irides Mydriatic 3 -1 Mean Pupil Diameter - Light Irides 0 0.5 1 HA p<0.0001 p<0.0001 Placebo (n=58) -Nyxol (n=113) p<0.0001 1.5 2 3 4 5 Time Post-Treatment with Nyxol/Placebo MIRA-3 Phase 3 Trial Mean PD - Study Eye (mITT) # p=0.0007 6 24 9 8 4 3 Mydriatic -1 p<0.0001 Max dilation; Treatment p<0.0001 Mean Pupil Diameter - Dark Irides 0 0.5 Source MIRA-3 Table 14.2.2.5-(mITT). The p-values are change from max pupil dilation treatment compared to placebo. Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd). Standard Error bars are shown. 1 p<0.0001 p<0.0001 p<0.0001 Placebo (n=66) -Nyxol (n=131) p<0.0001 3 4 5 1.5 2 Time Post-Treatment with Nyxol/Placebo 6 p<0.0001 24 Ocuphire PHARMA#23RM Mean Time to Return to Baseline PD Saving of ~4 Hours in Return to Normal PD Overall and Across Mydriatic Agents Overall Mydriatic Agent (study eye) Irides (study eye) 23 Study Eye Fellow Eye Phenylephrine Tropicamide Paremyd Dark Irides Light Irides 0 n=115 n=230 n=115 n=230 n=66 n=137 n=26 n=46 n=23 n=47 n=59 n=122 n=56 n=108 Source: MIRA-3 Table 14.2.3.2 (PP Population). 1 Placebo 1.3, p=<0.001 2.1, p=<0.001 2 2.5, p=<0.001 2.7, p=<0.001 2.3, p=<0.001 2.0, p=<0.001 ■ Nyxol 3 4 4.0, p=<0.001 5 5.6 5.7 6 6.3 6.4 Time to Return to Baseline PD (Hours) 7 Return to normal 4.2 hrs 7.1 7.0 3.9 hrs 4.3 hrs 7.6 3.6 hrs 4.4 hrs 3.4 hrs 5.0 hrs 8 Ocuphire PHARMA#24RM 24 Maximum Pupil Dilation Results in Loss of Near Vision Nyxol Returns Near Vision to Baseline Levels Statistically Faster Compared to Placebo Change from Baseline (Letters Read) 2 -2 -6 -8 Mydriatic -10 -1 Baseline Near Vision Max dilation; Treatment 0 p=0.0002 MIRA-3 Phase 3 Trial DCNVA Letters Read Study Eye (mITT) p=0.009 1.5 1 2 Time Post-Treatment with Nyxol/Placebo MIRA-3 Table 14.3.6.1.1 (Safety Population) (mITT). DCNVA- Distance-Corrected Near Visual Acuity. 3 4 -Nyxol (n=244) Placebo (n=124) 5 p=0.03 6 Ocuphire PHARMA#25RM 25 Summary of Safety Findings Nyxol was Well Tolerated with a Favorable Safety Profile There were no deaths, serious AEs, or withdrawals due to AEs ● • 48 of 244 (20%) Nyxol treated subjects reported 101 AEs All treatment related AEs were mild in severity ● ● ● ● - The only AE occurring in ≥ 5% of subjects treated with Nyxol, was conjunctival hyperemia (11% Nyxol vs. 0% placebo) Less than 1% of subjects reported instillation site discomfort, pain, or irritation Conjunctival hyperemia was observed to be mild and transient Visual acuity (distance and near) was not adversely affected by Nyxol Over 300 subjects have been treated with Nyxol and evaluated at 24-hours in the MIRA trials satisfying regulatory requirements for drug safety exposure for the acute RM indication - Source: MIRA-3 Table 14.3.1.1; MIRA-3 Table 14.3.1.2.2; MIRA-3 Table 14.3.3.2 (Safety Population). Ocuphire PHARMA#26RM 26 Summary of Positive MIRA-3 Phase 3 Results for Nyxol Eye Drops Confirms Prior Phase 3 Study Showing Substantial Benefit in Accelerating Reversal of Mydriasis ● ● ● ● ● Met primary endpoint at 90 minutes with 58% of subjects returning to pre-dilation pupil diameter vs. 6% of placebo treated subjects (p < 0.0001) Saving of ~4 hours in time to return to normal pupil diameter Met key secondary endpoints with high statistical significance Efficacy seen at all timepoints from 60 minutes to 24 hours Similar efficacy for one drop and two drops Efficacy across all 3 mydriatic agents - phenylephrine, tropicamide, and ParemydⓇ Efficacy in both light and dark iris colors Accelerated return to normal distance-corrected near visual acuity - Favorable safety and tolerability profile No serious AEs, no drop-outs from AEs No systemic or ocular AEs were observed in ≥ 5% of subjects, except for 11% mild, transient conjunctival hyperemia NDA planned for late 2022 MIRA-3 Topline Reports Ocuphire PHARMA#2727 Plans to NDA for Nyxol in RM Ocuphire PHARMA#28RM 28 MIRA Program Evaluating Nyxol for the Reversal of Mydriasis Efficient Clinical Programs have Positioned Ocuphire to Target NDA Filing in Late 2022 MIRA-1 2019 Phase 2b n=32 crossover Primary Endpoint Met ✓ Secondary Endpoints Met ✓ MIRA-2 2021 Phase 3 n=185 Primary Endpoint Met ✓ Secondary Endpoints Met ✓ MIRA-3 2022 Phase 3 n=368 Primary Endpoint Met ✓ Secondary Endpoints Met ✓ MIRA-4 2022 Pediatric Safety n=23 Fully Enrolled 3-11 years old RM NDA Filing 2022 NDA Submission Ocuphire PHARMA#29RM NDA Submission Targeted in Late 2022 Potential Regulatory Approval in 2023 29 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. Preservative-Free Single Unit Vial (5-pack) AAAAA 3000 NyxolⓇ P3 Clinical Trial Completed 2nd Phase 3 trial in RM (enrolled 368 subjects), which also meets 24-hour safety population exposure requirement Ongoing 4141 Pediatric Safety Enrolled 23 subjects ages 3 to 11 per agreed FDA initial pediatric study plan Ongoing Manufacturing Completed 3 registration batches; 1-year CMC stability will be available for NDA Regulatory Approval Submit NDA by late 2022, with expected approval review of 10 months Ocuphire PHARMA#3030 Ocuphire Reversal of Mydriasis Market Opportunity#31RM 31 Reversal of Mydriasis Unmet Need & Landscape With No Commercially Available Treatment, Nyxol is Uniquely Positioned as a New Reversal Drop The Problem At many annual eye exams and specialty visits, pupils are pharmacologically dilated, impairing vision for 6-24 hours Dilated eyes experience: Heightened sensitivity to light Inability to focus, headaches Difficulty reading, working & driving Halos and glare Cycloplegia (loss of accommodation) Source 1. Optician (2012)-Mydriatic Drugs: Practical Considerations 2. Pilocarpine FDA Label (2017) 3. Optos plc Pricing No Currently Available Treatments 100M Annual Eye Dilations ● Current Landscape: Rare off-label use of cholinergic agonists (e.g., pilocarpine) given ciliary muscle safety issues1,2 Optomap® is offered by optometrists to avoid dilations for -$50 cash-pay, however images may provide limited view of retina and disease pathology³ Nyxol's MOA Uniquely Suited As A Reversal Drop For Dilations Ocuphire PHARMA#32RM Demand Side Validation TON LPED FECFD EDICEP ******** Bottom-Up Calculation of Annual Dilated Eye Exams ~100 M Annual Dilated Eye Exams are Performed in the US 32 Optometrists Number of Providers (X) 46,000 Ophthalmologists 18,000 Retina Specialists 3,000 Average Number of Weekly Exams (Y) 59 88 150 Estimated % Patients Dilated (Z) *IQVIA 2020 sales data; KOL Interview; GlobalData market research; and AOA Excel and Jobson Medical Information 'Bottom-Up Calculation' assumes 48 total work weeks in a year Supply side validation assumed each unit (bottle) has ~10 mL fill volume and each patient gets 2-4 drops 40% 50% 50% Total (X*Y*Z) * 48 wk/yr ~52 M -38 M ~10 M Supply Side Validation: Based on the ~2 million total units of mydriatic agents sold in 2020, we calculated the total number of dilated eye exams to be ~125 million patients, consistent with demand side estimates. 100M Annual Dilated Eye Exams Ocuphire PHARMA#33RM 33 Reversal of Mydriasis (RM) Market Opportunity With No Commercially Available Treatment, Nyxol May Achieve Significant Revenue Potential GlobalData Market Research Findings 100M Annual Eye Dilations 80% of Patients Likely to Request Drop MIRA Trials Represent 95% of Dilation Drops Used in Practice Patient Willingness to Pay $10 - $20+ 65% Report Moderate to Severe Impact to Daily Function ~$500+M Estimated US RM Market Opportunity Source: GlobalData Market Research Survey Calculation: 100M Annual Eye Dilations X 65% X 80% X $10 per patient = $500+M Opportunity 58% physicians would start prescribing Nyxol within 1st year 81% patients would be more likely to schedule yearly eye exams with a reversal drop 0 Current Commercially Available Treatments 68% physicians would be willing to use Nyxol even if patients had to still wear sunglasses within 1st hour Ocuphire PHARMA#34RM 34 More Efficient Launch Opportunity for Nyxol in RM Launch is Poised to be Disruptive, Cost-Effective and Not Payor-Driven XX XXXX Traditional Ophthalmic Launch Highly competitive markets (e.g., dry eye, glaucoma, allergy); little differentiation Launch success takes time given payor (reimbursement) dependence Significant prior authorization & step-edits hurdles with burden to the practices Lengthy sales cycles and touchpoints due to chronic use and market access upkeep Significant product education requirement Complex distribution channel including specialty and retail pharmacies "One product, one indication" commercial model is inefficient with fixed cost infrastructure Ocuphire's Nyxol RM Launch No competition or approved reversal drop → potential for Nyxol to be the only safe option Cash pay (no reimbursement barriers) allowing for quicker adoption Offering a significant value proposition to patients and practices Shortened sales-cycle with acute use product No training given dilations routine in practices No specialty/retail pharmacy → direct to physician "One product, several indications" offers efficiencies in commercial operations Ocuphire PHARMA#35RM Pre-Commercial 2022 & Go-To-Market Strategy 2023 Activities Underway to Support Capital-Efficient Nyxol RM Commercial Launch Pre-Commercial Activity Market Development (KOLS) Physician Targeting Patient Journey Brand Awareness 35 Go-To-Market Strategy Potential Options for Commercialization Work with strategic or channel partner with existing commercial ophthalmic products Hire contract commercial organization Build own salesforce Easy Adoption Dilations are a routine part of practice; adoption requires no staff or patient training Landscape No approved drug/competition; data-mining for high volume practices Components of an Efficient Launch Direct to Physicians No need for pharmacy; no reimbursement, private pay Retina 3,000 Retinal Specialists Ophthalmology 20,000 Ophthalmologists TOZ LPED PECFD Optometry 46,000 Optometrists Sources: ASRS; AMA; AAO; Women in Optometry (WO); AOA Excel and Jobson Medical Information; Physician Interviews Conducted by Ocuphire; GlobalData market research Ocuphire PHARMA#3636 Upcoming Milestones Ocuphire PHARMA#37JJ 는 37 Track Record of Achieving Milestones → Exciting 2022 News Cadence Multiple Late-Stage Data Catalysts Expected in 2022 for Potential First NDA Approval in 2023 2021 Report Positive Phase 3 Data for RM (MIRA-2) Report Positive Nyxol+LDP Phase 2 Data for Presbyopia (VEGA-1) New Patent Claims for Presbyopia ASCRS 2021 Presentation for MIRA-2 & VEGA-1 Manufacture 3xRegistration Batches for Nyxol Blow-Fill- Seal (BFS) Eye Drops Initiate 2nd Phase 3 RM AND Pediatric RM trial 2022 Report Positive Nyxol Alone Phase 2 Data for Presbyopia Report 2nd Phase 3 Data for RM (MIRA-3) Report Phase 3 Data for NVD (LYNX-1) Report Pediatric Data for RM (MIRA-4) Submit Nyxol NDA for RM Report Phase 2 Data for DR/DME (ZETA-1) Initiate VEGA Phase 3 Presbyopia Program Ongoing Partnering Discussions with Leading Ophthalmic Companies (including European and Asian Players) Ocuphire PHARMA#38Ocuphire Restore Vision & Clarity Q&A www.ocuphire.com [email protected]