Ocuphire Pharma : ZETA-1 Topline Results Conference Call Presentation

Restore Vision & Clarity

January 25, 2023

ZETA-1 APX3330 Topline Results

Investor Webcast

Disclosures and Forward-Looking Statements

This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the success and timing of planned future clinical trials for APX3330, timing and occurrence of an end of phase 2 meeting with the FDA, the potential of a Phase 3 registration path for APX3330, the success and timing of

planned regulatory filings, business strategy, cash runway, scalability, the potential for APX3330 to be the first line of therapy for DR patients, and the potential market opportunity for the slowing of DR progression. 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, costs, 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;

1. legislative, regulatory, political and economic developments, (vii) Nyxol partnership may not facilitate the commercialization or market acceptance of Ocuphire's product candidates; (x) the success and timing of commercialization of any of Ocuphire's product candidates, including the scalability of Ocuphire's product candidates and (xi) 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.

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Agenda and Speakers

Topic

ZETA-1 Key Takeaways and APX3330 Oral MOA

ZETA-1 Trial Design and Demographics

ZETA-1 Efficacy Findings

ZETA-1 Safety Findings

Overall Summary and Next Steps

Q&A

Mina Sooch, MBA	Mitch Brigell, PhD
Founder and Chief	Head of Clinical
Executive Officer	Development and Strategy

Charles Wykoff, MD, PhD	Mark Kelley, PhD
Vitreoretinal Specialist	APX Scientific Founder
	and Medical Advisor

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ZETA-1 Key Takeaways and APX3330 Oral MOA

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ZETA-1 Trial: Key Takeaways

APX3330 achieved statistical significance on a key pre-specified secondary endpoint of preventing clinically meaningful progression of diabetic retinopathy (as defined by binocular 3 or more steps worsening on the DRSS1) after 24 weeks of treatment

• Trend toward more efficacy at 24 weeks vs 12 weeks, suggests that the 52-week Phase 3 trial may generate a larger signal due to an increase in % of placebo subjects who progress

Prevention of 3-step worsening (binocular) is a suitable endpoint for an oral, systemically drug

• Ocuphire plans to go forward with this potential registration endpoint in Phase 3 following confirmation with the FDA in EOP2 meeting

Oral APX3330 demonstrated favorable safety and tolerability

Retinal KOLs feedback suggest that slowing of DR progression with an oral agent would be a useful treatment in patients with background DR and good visual function

If approved, APX3330 could be an important new primary preventative therapeutic option that could be used in a large number of diabetic patients who are earlier in their disease

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1. diabetic retinopathy severity score Source: ZETA-1 Clinical Trial