Slide 1: MIRA-3 Phase 3 Trial Results Conference Call
MIRA-3 Phase 3 Trial Results Conference Call March 29, 2022
Slide 2: Disclosures and Forward-Looking Statements
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 r
Slide 3: Agenda and Participants
Agenda and Participants 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
Slide 4: Highlights and Overview
©)Ocuphire PHARMA Highlights and Overview
Slide 5: Key Takeaways from Nyxol's MIRA-3 2nd Phase 3 RM Trial
Key Takeaways from Nyxol’s MIRA-3 2nd Phase 3 RM Trial MIRA-2 and MIRA-3 topline data. MIRA-3 58% vs. 6% p<0.0001 MIRA-2 49% vs. 7% p<0.0001 Key Secondary Endpoints Met Statistical and Clinical Significance
Slide 6: Addressing Unmet Needs in Large Markets
Addressing Unmet Needs in Large Markets Source: Eisai and Apexian Data; GlobalData Market Research Report, 2020; Company Estimates for US Market Size; *Ocuphire internal estimates. Significant Preclinical & Clinical Data Supporting MOA, Efficacy and Safety 11 Completed Phase 1 and Phase 2 Trials >34
Slide 7: Ocuphire Pipeline & Clinical Milestones
Indication Product Candidate Pre-clinical Phase 1 Phase 2 Phase 3 Regulatory Approval Anticipated Milestones Reversal of Mydriasis (RM) Nyxol® Eye Drop Reported MIRA-3 Phase 3 data in Q1 2022 (n=368) MIRA-4 Pediatric safety study data expected in 2Q 2022 (n=23) Presbyopia (P) Nyxol® Eye Drop VEGA Ph
Slide 8: Nyxol's Differentiated MOA as an Alpha-1 Blocker
Nyxol’s Differentiated MOA as an Alpha-1 Blocker Phentolamine Mesylate Reformulated as a Proprietary Topical Eye Drop NyxolTM Phentolamine Mesylate is the Active Ingredient in Nyxol: a Non-selective α1 & α2 Antagonist Blocking α1 Reduces Pupil Size Blocking α1 Dilates Blood Vessels Phentolamine me
Slide 9: Nyxol Product Candidate Profile
Nyxol Improves Vision by Decreasing Pupil (~1-1.5mm) ↑ Near Vision ↑ Distance Vision ↑ Contrast Sensitivity (night) 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 N
Slide 10: Nyxol for Reversal of Mydriasis (RM)
Nyxol®forReversal of Mydriasis (RM) 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 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
Slide 11: Problem: Dilated Eyes for Exams and Procedures
Problem: Dilated Eyes for Exams and Procedures 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)
Slide 12: Nyxol Has Potential To Be The Only Option For RM
Nyxol Has Potential To Be The Only Option For RM Physicians AVOID Use of Cholinergic Agonists (Pilocarpine) Due to Safety Risk on Ciliary Muscle 1 Pilocarpine FDA Label (2017)2. Optician (2012)- Mydriatic Drugs: Practical Considerations 3. Lee DA, Higginbotham EJ, 2005. Glaucoma and its treatment: a
Slide 13: MIRA-3 Topline Phase 3 Results
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
Slide 14: MIRA-3 Phase 3 Registration Trial Design
MIRA-3 Phase 3 Registration Trial Design Randomized, Double-Masked, Placebo-Controlled, Parallel, Multi-Center, One-Day Trial Mydriasis -1 Hour 1:1 Mydriatic Agent A, B, or C 0.75% Nyxol Placebo 16 US sites 368 subjects Nyxol drop(s) (2 drops study eye, 1 drop fellow eye) Mydriatic Agent A, B, or C
Slide 15: Demographics
Demographics Nyxol n=244 Placebo n=124 Total n=368 Demographics Age (years): Mean (Range) 34 (12-80) 36 (12-80) 35 (12-80) Sex: Male n (%) Female n (%) 92 (37.7%) 152 (62.3%) 59 (47.6%) 65 (52.4%) 151 (41.0%) 217 (59.0%) Race: White n (%) African American n (%) Asian n (%) Other^ n (%) ^includes Ame
Slide 16: Baseline Characteristics
Baseline Characteristics Study Eye Nyxol n=248 Placebo n=120 Total n=368 Baseline Characteristic Baseline Pupil Diameter Mean (mm) 5.1 4.9 5.1 Max Dilated Pupil Diameter Mean (mm) 7.2 7.1 7.2 Accommodation Mean (diopters) 7.4 7.6 7.5 BCDVA letters 55 letters = 20/20 57 57 57 DCNVA letters 70 letters
Slide 17: Primary Endpoint: 58% Returned to Baseline at 90 Min
Primary Endpoint: 58% of Subjects’ Study Eye Returned to Baseline at 90 Min Source: MIRA-3 Table 14.2.1.1 (mITT). Data include all three mydriatics (Phenylephrine, Tropicamide, Paremyd). Nyxol Statistically Better Than Placebo Starting At 1 Hour And All Subsequent Timepoints MIRA-3 Phase 3 Trial RM
Slide 18: Primary Endpoint Achieved in Two FDA Registration Phase 3 Trials
Primary Endpoint Achieved in Two FDA Registration Phase 3 Trials 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). Rapid, Consistent and Sustained Reversal of Pupil Dilation with Nyx
Slide 19: Comparison of One Drop with Two Drops
Comparison of One Drop (Fellow Eye) with Two Drops (Study Eye) Source: MIRA-3 Table 14.2.1.1 (mITT). Data includes all three mydriatics (Phenylephrine, Tropicamide, Paremyd). Similar 52% of Subjects Return to Baseline at 90 Minutes with a Single Drop of Nyxol RM MIRA-3 Phase 3 Trial
Slide 20: Mean Pupil Diameter Over Time
Mean Pupil Diameter Over Time Source: MIRA-3 Table 14.2.2.1 (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. Nyxol Treatment Significantly Reduced PD Startin
Slide 21: Mean Pupil Diameter Over Time by Mydriatic Agents
Mean Pupil Diameter Over Time by Mydriatic Agents 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. Nyxol Reduced PD With All Mydriatic Agents; More Rapidly with Phenylephrine as Expected MIRA-3 Phase
Slide 22: Mean Pupil Diameter Over Time by Eye Color
Mean Pupil Diameter Over Time by Eye Color 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. Nyxol Reduced Pupil Diameter Rapidly
Slide 23: Mean Time to Return to Baseline PD
Mean Time to Return to Baseline PD Source: MIRA-3 Table 14.2.3.2 (PP Population). Saving of ~4 Hours in Return to Normal PD Overall and Across Mydriatic Agents Overall Irides (study eye) Mydriatic Agent (study eye) n=230 n=115 n=230 n=115 n=66 n=137 n=26 n=46 n=23 n=47 n=59 n=122 n=56 n=108 RM 5.0 h
Slide 24: Maximum Pupil Dilation Results in Loss of Near Vision
Maximum Pupil Dilation Results in Loss of Near Vision MIRA-3 Table 14.3.6.1.1 (Safety Population) (mITT). DCNVA- Distance-Corrected Near Visual Acuity. Nyxol Returns Near Vision to Baseline Levels Statistically Faster Compared to Placebo RM Baseline Near Vision MIRA-3 Phase 3 Trial 1.5
Slide 25: Summary of Safety Findings
Summary of Safety Findings 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% p
Slide 26: Summary of Positive MIRA-3 Phase 3 Results for Nyxol Eye Drops
Summary of Positive MIRA-3 Phase 3 Results for Nyxol Eye Drops 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 wi
Slide 27: Plans to NDA for Nyxol in RM
27 PHARMA Plans to NDA for Nyxol in RM
Slide 28: MIRA Program Evaluating Nyxol for the Reversal of Mydriasis
MIRA Program Evaluating Nyxol for the Reversal of Mydriasis Efficient Clinical Programs have Positioned Ocuphire to Target NDA Filing in Late 2022 MIRA-1 MIRA-2 MIRA-3 MIRA-4 RM NDA Filing Phase 2b Phase 3 Phase 3 Pediatric Safety NDA Submission 2019 n=32 crossover n=185 n=368 n=23 2021 2022 2022 20
Slide 29: NDA Submission Targeted in Late 2022
NDA Submission Targeted in Late 2022 Potential Regulatory Approval in 2023 Enrolled 23 subjects ages 3 to 11 per agreed FDA initial pediatric study plan Pediatric Safety Completed 2nd Phase 3 trial in RM (enrolled 368 subjects), which also meets 24-hour safety population exposure requirement P3 Clin
Slide 30: Reversal of Mydriasis Market Opportunity
Reversal of Mydriasis Market Opportunity
Slide 31: Reversal of Mydriasis Unmet Need & Landscape
Reversal of Mydriasis Unmet Need & Landscape Source 1. Optician (2012)-Mydriatic Drugs: Practical Considerations 2. Pilocarpine FDA Label (2017) 3. Optos plc Pricing With No Commercially Available Treatment, Nyxol is Uniquely Positioned as a New Reversal Drop At many annual eye exams and specialty v
Slide 32: Bottom-Up Calculation of Annual Dilated Eye Exams
Bottom-Up Calculation of Annual Dilated Eye Exams ~100 M Annual Dilated Eye Exams are Performed in the US *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 validati
Slide 33: ~$500+M Estimated US RM Market Opportunity
~$500+M Estimated US RM Market Opportunity Reversal of Mydriasis (RM) Market Opportunity With No Commercially Available Treatment, Nyxol May Achieve Significant Revenue Potential Patient Willingness to Pay $10 - $20+ 65% Report Moderate to Severe Impact to Daily Function 100M Annual Eye Dilations 80
Slide 34: More Efficient Launch Opportunity for Nyxol in RM
More Efficient Launch Opportunity for Nyxol in RM Launch is Poised to be Disruptive, Cost-Effective and Not Payor-Driven Traditional Ophthalmic Launch Traditional Ophthalmic Launch Highly competitive markets (e.g., dry eye, glaucoma, allergy); little differentiation Launch success takes time given p
Slide 35: Pre-Commercial 2022 & Go-To-Market Strategy 2023
Pre-Commercial 2022 & Go-To-Market Strategy 2023 Sources: ASRS; AMA; AAO; Women in Optometry (WO); AOA Excel and Jobson Medical Information; Physician Interviews Conducted by Ocuphire; GlobalData market research Activities Underway to Support Capital-Efficient Nyxol RM Commercial Launch Physician Ta
Slide 36: Upcoming Milestones
36 ©)Ocuphire PHARMA Upcoming Milestones
Slide 37: Track Record of Achieving Milestones; 2022 News Cadence
Track Record of Achieving Milestones Exciting 2022 News Cadence Ongoing Partnering Discussions with Leading Ophthalmic Companies (including European and Asian Players) 2021 2022 Report Positive Nyxol Alone Phase 2 Data for Presbyopia Report 2nd Phase 3 Data for RM (MIRA-3) Report Phase 3 Data for