Opus Genetics, Inc. · NASDAQ:IRD

Opus Genetics — Ocuphire Pharma — Technical Presentation (March 2021)

29 slides from Opus Genetics's technical presentation, Mar 15, 2021

  1. Slide 1: MIRA-2 Phase 3 Trial Results Conference Call

    ()Ocuphire Restore Vision & Clarity MIRA-2 Phase 3 Trial Results Conference Call March 15, 2021

  2. Slide 2: Disclosures and Forward Looking Statements

    Disclosures and Forward Looking Statements Reform Act of 1995. Such statements include, but are not limited to, statements conceming Ocuphire Pharma, Inc.'s (‘Ocuphire" or the "Company’) product candidates and potential. These forward-looking statements are based upon the Company's current expectati

  3. Slide 3: Agenda and Participants

    Agenda and Participants First Phase 3 Trial Topline Readout as Planned in 1Q21 * Topline MIRA-2 Phase 8 Clinical Trial Results for Nyxol in Reversal of Mydriasis Reversal of Mydriasis Market Opportunity Future Milestones * Q&A Participants Mina Sooch, MBA, President and CEO Jay Pepose, MD, Medical A

  4. Slide 4: Ocuphire Pipeline & Upcoming Milestones

    Ocuphire Pipeline & Upcoming Milestones Multiple Phase 3 & Phase 2 Clinical Data Readouts Anticipated Over the Next Year Product Candidate Indication reared] Pe Development Stage Anticipated Milestones 2 0.75% Nyxol® Dim Light or Night Vision Initiated Phase 3 LYNX-1 trial 402020; S Eye Drop Disturb

  5. Slide 5: Nyxol

    Nyxol® @®D Night Vision Disturbances N > Reversal of Mydriasis Ci & Presbyopia *CH,SO,H Phentolamine Mesylate

  6. Slide 6: Topline MIRA-2 Phase 3 Results

    Topline MIRA-2 Phase 3 Results Randomized, Parallel Arm, Double-Masked, Placebo- Controlled Study of the Safety and Efficacy of Nyxol (0.75% Phentolamine Ophthalmic Senition) to Reverse Pharmacologically-Induced Mydriasis in Healthy Subjects

  7. Slide 7: Objectives and Key Eligibility Criteria

    @ Objectives and Key Eligibility Criteria MIRA-2 (OPI-NYX-RM-301) Phase 3 Trial Evaluating Reversal of Mydriasis with Nyxol or Placebo Key Objectives Key Eligibility Criteria PRIMARY + Inclusion — Healthy = 12 years of age + To evaluate the efficacy of Nyxol to + Exclusion expedite the reversal of —

  8. Slide 8: RM MIRA-2 Phase 3 Registration Design

    @ RM MIRA-2 Phase 3 Registration Design 8 Randomized, Double-Masked, Placebo-Controlled, Parallel, One-Day Trial 0.75% Nyxol MIRA-2 Mydriatic Agent Nyxol drop(s) ABorc (2 drops study eye, 12 US , B, OF 1 drop fellow eye) sites Mydriasis Treatment 168 Time -1 Hour Time 0 (Max Dilation) target Placebo

  9. Slide 9: Treatment and Placebo Arms Were Balanced in this Phase 3 Registration Trial

    © Demographics (mITT Population) Treatment and Placebo Arms Were Balanced in this Phase 3 Registration Trial 9 Age (years): Median (Range) 31 (12-70) 30 (13-73) 31 (12-73) Sex: Male n (%) 36 (38%) 36 (40%) 72 (39%) Femalen (%) 58 (62%) 55 (60%) 113 (61%) Race: White n (%) 70 (75%) 74 (81%) 144 (78%)

  10. Slide 10: Baseline Characteristics Study Eye (mITT Population)

    @ Baseline Characteristics Study Eye (mITT Population) 10 Treatment and Placebo Arms Were Balanced Across These Ocular Measurements Baseline Pupil Diameter Mean (mm) 5.09 5.18 5.13 Max Dilated Pupil Diameter 721 7.20 7.20 Mean (mm) Accommodation Median (diopters) 7.28 7.41 7.41 BCDVA letters 55 lett

  11. Slide 11: Primary Endpoint: % of Subjects Study Eye Returning to Baseline PD at 90 Min

    @ Primary Endpoint: % of Subjects Study Eye Returning to Baseline PD at 90 Min Nyxol Met the Primary Endpoint at 90 Min; Additionally at 60 Min and All Subsequent Timepoints + MIRA-2 Phase 3 i Study Eye (mITT Population) Percent of Subjects Returning to < 0.2 mm of Baseline $508 p<0.0001 20% Placebo

  12. Slide 12: Secondary Endpoint: % of Subjects Returning to Baseline PD by Mydriatic Agent

    @ Secondary Endpoint: % of Subjects Returning to Baseline PD by Mydriatic Agent Subjects Dilated with Phenylephrine had a Faster Response to Nyxol than Tropicamide/Paremyd Study Eye (mITT Population) Percent of Subjects Returning to < 0.2 mm of Baseline by Mydriatic Agent Phenylephrine Tropicamide o

  13. Slide 13: Secondary Endpoint: % of Subjects Returning to Baseline PD by Iris Color

    @> Secondary Endpoint: % of Subjects Returning to Baseline PD by Iris Color Evidence of Efficacy in Subjects with Both Light and Dark Irides, with a More Vigorous Response in Light Irides MIRA-2 Phase 3 Percent of Subjects (%) Study Eye (mITT Population) Percent of Subjects Returning to < 0.2 mm of

  14. Slide 14: Secondary Endpoint: Non-Study Eye Returning to Baseline PD

    oO Secondary Endpoint: % of Subjects Non-Study Eye Returning to Baseline PD A Similar Significant Effect was Obtained with a Single Drop of Nyxol in the Non-Study Eye -—______! MIRA-2 Phase 3 a, Non-Study Eye (mITT Population) Percent of Subjects Returning to < 0.2 mm of Baseline 100% p<0.0001 Place

  15. Slide 15: Secondary Endpoint: Mean Pupil Diameter Over Time

    > Secondary Endpoint: Mean Pupil Diameter Over Time Nyxol Treatment Significantly Reduced PD Starting at 1 Hour Post-Dose through 6 Hours MIR. Study Eye, mITT Population Phase 3 9 —Nyxol n=94 Placebo n=91 8 E = E Ra 57 / . - @ f ~ = £ + aé ~ 2 v p<0.0001 . a5 s fax jilation, 3 ment p<0.0001 Sy | Mea

  16. Slide 16: Secondary Endpoint: Mean Pupil Diameter Over Time by Mydriatic Agent

    @> Secondary Endpoint: Mean Pupil Diameter Over Time by Mydriatic Agent Nyxol Reduced Pupil Diameter With All Mydriatic Agents; More Rapidly with Phenylephrine as Expected MIRA-2 Phase 3 Tri Study Eye, mITT Population Phenylephrine Placebo n=55 —Nyxol n=56 ay jameter (mm) ro Pupil © N Tropicamide or

  17. Slide 17: Secondary Endpoint: Mean Pupil Diameter Over Time by Eye Color

    @> Secondary Endpoint: Mean Pupil Diameter Over Time by Eye Color Nyxol Reduced Pupil Diameter More Rapidly in Both and Light Dark Irides MIRA-2 Phase 3 Study Eye, mITT Population Light Irides Dark Irides 9 t] —Nyxol (n=45) Placebo (n=45) —Nyxol (n=49) Placebo (n=46) ES! 8 = + E + E7 f 7 —7 t a th r

  18. Slide 18: Secondary Endpoint: Safety Findings

    @ Secondary Endpoint: Safety Findings Nyxol was Well Tolerated with a Favorable Safety Profile ¢ There were no deaths, serious AEs, or withdrawals due to AEs * Only AEs, occurring in = 5% of subjects treated with Nyxol, were instillation site discomfort (38% Nyxol vs. 9% placebo) and conjunctival hy

  19. Slide 19: Summary of Positive MIRA-2 Phase 3 Results for Nyxol Eye Drops

    €& Summary of Positive MIRA-2 Phase 3 Results for Nyxol Eye Drops Sustained Efficacy with a Favorable Safety Profile in Reversing Mydriasis with Nyxol * Met primary endpoint at 90 minutes with high statistical significance with 2 drops of Nyxol « Metall key secondary endpoints with high statistical

  20. Slide 20: Next Steps For Nyxol RM Indication for NDA

    @ Next Steps For Nyxol RM Indication for NDA NDA Submission Expected Early 2023 « Perform a second Phase 3 RM registration trial (MIRA-3) — Planned 330 subjects randomized 2:1 to Nyxol or Placebo + In addition to confirming efficacy, this trial will satisfy the regulatory requirement for number of s

  21. Slide 21: Reversal of Mydriasis Market Opportunity

    a1 ©)Ocuphire PHARMA Reversal of Mydriasis Market Opportunity

  22. Slide 22: Nyxol Product Candidate Profile

    @ Nyxol Product Candidate Profile Novel Alpha 1/2 Blocker Eye Drop for Retractive Indications (505(b)(2) Pathway) Nyxol: Phentolamine 0.75% Ophthalmic Soluti Preservative Free, EDTA Free, and Stable Efficacy Data Safety Data Improving Vision No Systemic Effects No Changes in Blood Pressure I Pupil S

  23. Slide 23: Reversal of Mydriasis (RM) — Acute Treatment

    @ Reversal of Mydriasis (RM) — Acute Treatment Annual Exams and Specialty Visits Involve Dilation to Monitor Eye Health The Problem + At many annual eye exams and specialty visits, pupils are pharmacologically dilated, impairing vision for 6-24 hours * Dilated eyes: — heightened sensitivity to light

  24. Slide 24: Reversal of Mydriasis (RM) — Acute Treatment

    @ Reversal of Mydriasis (RM) — Acute Treatment Single Use Indication Leveraging a Precedent Approval Pathway Nyxol’s Potential Differentiated Soluti * Regulatory Precedent with Rev-Eyes (an alpha 1 Before After blocker), approved by the FDA in 1990 but shortly thereafter discontinued (not for safety

  25. Slide 25: Nyxol Comparison to Rev-Eyes

    @ Nyxol Comparison to Rev-Eyes Nyxol has a Distinct Commercial Advantage to Rev-Eyes Nyxol Rev-Eyes Tolerability + Mild hyperemia + Severe hyperemia (80%) Comfort * Mild discomfort + Burning/Stinging (50%) 7 (38%), erythema TOs (4%), or instillation pain (3%) Side effects + None reported + 40% (ptos

  26. Slide 26: A Substantial Revenue Opportunity for Nyxol in Reversal of Mydriasis

    © Summary of RM Market Opportunity A Substantial Revenue Opportunity tor Nyxol in Reversal of Mydriasis * ~100M comprehensive and specialty eye exams in US per year * No current commercially available treatment for reversing dilation — Optomap ultra-wide field camera used for a retinal evaluation wi

  27. Slide 27: Future Milestones

    a7 PHARMA Future Milestones

  28. Slide 28: 2021 to 2022 Ocuphire Cadence of Milestones

    2021 to 2022 Ocuphire Cadence of Milestones Multiple Data Catalysts on Path to NDA(s) 2018/2019 ¥ NVD Podium Presentation at AAO 2018 ¥ Initiate/Report Phase 2b Data for ORION-1 ¥ Initiate/Report Phase 2b Data for MIRA-1 Y Expand Patent Estate 1H 2020 Y Completion of ‘APX3330 License ¥ ARVO 2020 Pre

  29. Slide 29: Q&A

    ()Ocuphire Restore Vision & Clarity Q&A www.ocuphire.com ir@ocuphire.com

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