Full Press Release Details
Ocuphire KOL Event: APX3330 October 14, 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 success and timing of planned regulatory filings, including
planned NDA filings, pre-commercial activities, commercialization strategy and timelines, business strategy, product labels, cash runway, scalability, future clinical trials in reversal of mydriasis (RM), presbyopia (P), dim light/night
vision disturbance (NVD) and diabetic retinopathy (DR)/diabetic macular edema (DME), and the potential market opportunity in DR/DME. 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 preclinical 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, including the scalability 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
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Ocuphire APX3330 KOL Event: Agenda &
Speakers Speakers Speakers Agenda Time (EDT) Mina Sooch, MBA President & CEO and Founder Introductions & Company Overview 11:00 am - 11:10 am Caroline Baumal, MD Disease of Diabetic Retinopathy 11:10 am - 11:20 am Peter
Kaiser, MD Current DR/DME Treatment Landscape 11:20 am - 11:35 am David Lally, MD APX3330, Paradigm-Shifting Oral Treatment Option 11:35 am - 11:50 am Caroline Baumal, MD ZETA-1, Phase 2b Trial in Diabetic Retinopathy and Masked
Safety Data 11:50 am - 12:00 pm
Speakers Agenda Time (EDT) ZETA-1 Trial Design and Data Expectations 12:00
pm - 12:15 pm Q&A Closing Remarks Q&A Moderator: Corey Davis, PhD 12:15 pm - 12:30 pm Ocuphire APX3330 KOL Event: Agenda & Speakers Peter Kaiser, MD Caroline Baumal, MD David Lally, MD Mina Sooch, MBA Peter Kaiser,
MD Caroline Baumal, MD David Lally, MD President, CEO, and Founder Mitch Brigell, PhD Head, Clinical Strategy Mark Kelley, PhD APX Program Scientific Advisor
Company Overview Presenter: Mina Sooch, CEO and Founder of Ocuphire
Pharma Over 25 years of pharmaceutical and biotech experience as CEO, entrepreneur, venture capitalist, and strategy consultant Successful track record of hundreds of millions of capital raised for leading private/public biotech
companies Experience across multiple diseases (cardiovascular, oncology, renal, NASH, CNS, etc.) prior to ophthalmology Recipient of numerous awards, including Deal Makers of the Year in 2016 and Alumni Commencement Speaker WSU College of
Engineering in 2021 Mina Sooch, MBA Harvard University
Upcoming Catalysts in 4Q22: Topline Results APX3330 ZETA-1 P2b trial for
DR/DME NDA Filing for Nyxol for RM P = Presbyopia RM = Reversal of Mydriasis NVD = Night Vision Disturbances DR/DME = Diabetic Retinopathy/Diabetic Macular Edema Ocuphire PharmaNasdaq: OCUP Founded in 2018, Acquired 2 Lead Assets for
Front & Back of Eye Therapies with Novel MOAs & Patent Coverage to 2034+ Nyxol eyedrops Reversal of Mydriasis ("RM") - eye dilation Presbyopia - age-related blurry near vision Night Vision Disturbance ("NVD") - halos, glares,
starbursts APX3330 oral tablets Diabetic retinopathy ("DR") - diabetes-related retinal (eye) disease Successful Execution of 5 Trials in last 2 Years with 6 Positive Phase 3 & Phase 2 Data Read-outs for Nyxol in RM, Presbyopia, and
NVD Potential 2023 commercialization opportunities in RM Near-term initiation planned for Presbyopia VEGA Phase 3 program with Nyxol alone and Nyxol with 0.4% Low Dose Pilocarpine as adjunctive therapy Four Large Markets (~$20B US total)
w/Unmet Needs and Limited to No Competition
Ocuphire Overview Two Late-Stage Clinical Assets Addressing Unmet Needs in
Multiple Large Markets Source: Eisai and Apexian Data; GlobalData Market Research Report, 2020; Company Estimates for US Market Size; Ocuphire internal estimates 11 Completed Phase 1 and Phase 2 Trials >340Subjects Dosed Patent
Coverage2034+ 12 Completed Phase 1,Phase 2, and Phase 3 Trials >650Subjects Dosed Exposure in Humans 28 Days Patent Coverage2034+ Exposure in Humans 365 Days Retina Refractive Presbyopia Reversal of Mydriasis Night
Vision Disturbances Diabetic Retinopathy Diabetic Macular Edema Phase 2 b Last PatientLast VisitCompletedAug 22 ~128 M ~100 M ~2.4 M ~8 M 1st Phase 3 Positive Data Phase 2 Positive Data Single & Combo 2 Phase 3 Positive
Data & Ped P3 ~36 M APX3330 Oral REF-1 Inhibitor New Chemical Entity Phase 2b Data 4Q22 DevelopmentMilestone Prevalence (US) Nyxol Novel 1/ 2 Blocker 505(b)(2) NDA-Filing Ready DevelopmentMilestone Prevalence (US)
Track Record of Achieving Milestones Multiple Positive Data Readouts with
Multiple Catalysts Ahead Ongoing Partnering Discussions with Leading Ophthalmic Companies (including Europe and Asia) 2021 - 1H 2022 2H 2022 - 2023 Submit Nyxol NDA for RM Report APX3330 Phase 2b Data for DR/DME (ZETA-1) Initiate VEGA
Phase 3 Presbyopia Program Potential Nyxol Approval and Commercialization
Disease of Diabetic Retinopathy Presented by: Caroline Baumal, MD Professor
of Ophthalmology at Tufts Medical Center Co-Director of the Retina Service and Medical Retina Fellowship at New England Eye Center Authored over 170 publications, 33 book chapters on retinal diseases, and edited the book Treatment of
Diabetic Retinopathy Recognized by the American Society of Retinal Surgeons, The Retinal Hall of Fame and received such honors as the Donald J. Gass Beacon of Sight Award from the Florida Ophthalmologic Society and the ASRS Crystal Apple
award from the Vit-Buckle Society. Caroline Baumal, MD University of Toronto
Diabetic Eye Disease is Common Cause of Blindness Diabetes and Diabetic
https://www.mayoclinic.org/diseases-conditions/type-2-diabetes/symptoms-causes/syc-20351193 Two Types of DR Non-Proliferative Diabetic Retinopathy (NPDR) - most common form of DR - early stages of edema and exudates, blurred central
vision Proliferative Diabetic Retinopathy (PDR) - later stage of DR, marked by abnormal blood vessels and scar tissue on retina Diabetic Macular Edema (DME) can occur at any stage of DR Diabetes Mellitus is a group of diseases
characterized by high blood glucose levels. Diabetes results from defects in the body's ability to produce and/or use insulin Diabetic retinopathy (DR) occurs when fluctuations or instability in blood glucose levels damages blood vessels in
the retina Type 1 diabetes (T1D): The body produces very little or no insulin, which means that patients need daily insulin injections to maintain blood glucose levels Type 2 diabetes (T2D): The most common form of diabetes - either the
body does not produce enough insulin, or resists insulin Normal Retina Diabetic Retina
Diabetes is a Growing Global Health Epidemic Diabetes Cost Burden Over $900
Billion Dollars in Worldwide Health Expenditure Source: International Diabetes Federation, Diabetes Atlas 10th Edition, 2021, https://diabetesatlas.org/atlas
Diabetic Patients Usually Present with Complex Co-Morbidities Diabetic Patients
are Young and Face Life-long Systemic and Ocular Complications 1. Petrella RJ, et al. J Ophthalmol 2012;159167; 2. International Diabetes Federation, Diabetes Atlas 6th Edition, http://www.idf.org/diabetesatlas; 3. National Diabetes Fact
Sheet, 2011 http://www.cdc.gov/diabetes/pubs/pdf/ndfs_2011.pdf; 4. Rodbard HW, et al. Endocr Pract 2007;13:4-69; 5. Wong TY, et al. JAMA 2002;288:67-74; 6. Nguyen-Khoa B, et al. BMC Ophthalmol 2012;12:11 Patients with DME have an even
greater risk of complications than diabetes patients without DME5,6 DR is the most common cause of vision loss or blindness in working-age adults, usually affecting both eyes DME is a vision threatening complication caused by DR where
excess fluid leaks near fovea and triggers swelling of the macula Treating DR leads to control of DME Oral options have the potential to reach other vascular beds to treat kidney and neuropathic co-morbidities Diabetic
retinopathy1 Stroke1 Diabetic neuropathy1 Diabetic nephropathy1,2 Cardiovascular disease2 Dyslipidemia2
DR: 11M* DME: 7M* DR: 16M* DME: 6M* DR: 4M* DME: 4M* DR: 7M* DME:
4M* DR: 18M* DME: 8M* DR: 15M* DME: 5M* DR: 32M* DME: 14M* USA DR: 8M2 DME: 0.75M2 North America & Caribbean Global Prevalence of Diabetes-Associated Retinal Disease DR Affects 1 in 3 People with Diabetes; DME Affects 1 in 13
People with Diabetes1 *Global estimates are provided by the National Eye Institute, FactSheet, Global Data, Research and Markets, American Academy of Ophthalmology, and PLOS One 1. Holekamp N. M. (2016). Overview of diabetic macular
edema. The American journal of managed care, 22(10 Suppl), s284-s291. 2. American Diabetes Association; American Journal of Managed Care, International Diabetes Federation; Healthline; Ocuphire internal analysis and assumptions
Measuring the Severity of Diabetic Retinopathy DRSS is Regularly Used For FDA
Approvals; Not As Widely Used in Everyday Practice Arcadu, F., Benmansour, F., Maunz, A. et al. Deep learning algorithm predicts diabetic retinopathy progression in individual patients. npj Digit. Med. 2, 92 (2019).
https://doi.org/10.1038/s41746-019-0172-3. Diabetic Retinopathy Severity Scale (DRSS) was developed to differentiate proliferative DR (PDR) from non-proliferative DR (NPDR)
DRSS Predicts Vision-Threatening Complications (PDR/DME) Percent of Eyes
Progress to PDR at 1-Year, 3-Year, and 5-Year Visits by Baseline DR Severity Early treatment diabetic retinopathy study research group. ophthalmology. 1991;98(5 suppl):823-33. Diabetes control and complications trial research group. N
Engl J Med. 1993;329(14):997-86. Fathy C, Patel S, Sternberg P Jr, Kohanim S. Disparities in adherence to screening guidelines for diabetic retinopathy in the United States: a comprehensive review and guide for future directions. Semin
Ophthalmol. 2016;31(4):364-377. doi: 10.3109/08820538.2016.1154170 Early screening and treatment for DR can reduce vision loss by up to 94% 1 Year Follow-up 3 Year Follow-up 5 Year Follow-up Percentage of Eyes that Worsen to
PDR Regardless of severity, all eyes worsen over time
Vision Loss Other Eye Problems Amputation, Losing a Leg Cardiovascular/Heart
Problems Foot Problems Kidney Problems Vision Loss is #1 Concern of Diabetic Patients Diabetic Retinopathy is a Progressive Vision-Threatening Disease Source: Patient survey adapted from Lions International Foundation and International
Diabetes Foundation-Europe; Meltzer 2000 N=2702 What are the top concerns for diabetic patients?
Early Management of Diabetic Retinopathy Poor Adherence to Medical Management
and Lifestyle Options Worsen DR Source: Zhang X et al. Cell Biosci. 2014;14:4:27 Control of Blood Sugar Control of Blood Pressure Smoking Cessation Control of Lipids Medical and lifestyle management is first line of treatment
Majority of Physicians Use a "Wait and Monitor" Approach for DR Patients Over
90% of DR Patients Are Not Treated Proactively and Anti-VEGF Use is Limited Source: ASRS 2021 Preferences and Trends (PAT) Survey How do physicians treat patients with severe NPDR without DME? Closely monitor retinopathy and encourage
systemic glycemic control Consider anti-VEGF in some patients with poor glycemic control and/or other risks Consider anti-VEGF in some patients with good glycemic control and compliance Consider anti-VEGF therapy in all or most
Diabetic Retinopathy At a Glance Current Treatment Landscape Demonstrates Need
for Less Invasive Therapies Source:1. American Diabetes Association; International Diabetes Federation; Healthline; *Ocuphire internal analysis and assumptions; 2. Das UN. DME, retinopathy and age-related macular degeneration as
inflammatory conditions. Arch Med Sci. 2016;12(5):1142-1157. doi:10.5114/aoms.2016.61918 3. Patient survey adapted from Lions International Foundation and International Diabetes Foundation-Europe; Meltzer 2000 4. Guidehouse Triangulation of
Global Data, Market Scope and Investor Forecasts (2020) AMD = Age-Related Macular Degeneration; DME = Diabetic Macular Edema ; BRVO = Branch Retinal Vein Occlusion The number of people with DR expected to increase more than 14M by
2050 There are ~8M adults in the U.S. with DR1 DR is the leading cause of blindness among working-age adults If untreated, DR can rob people of their vision prematurely2,3 56% of patients reported anxiety related to anti-VEGF
treatment DR/DME affects about 1 in 4 people with type 1 and type 2 diabetes Majority of moderate to severe patients with DR are not treated with anti-VEGF due to injection fear and burden $13B (2020) Global Intravitreal Injection
Revenues in AMD, DME and BRVO4
Current DR/DME Treatment Landscape Presented by: Peter Kaiser, MD Chaney
Family Endowed Chair in Ophthalmology Research, Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine and Cole Eye Institute Clinical research expert, serving as a Study Chairman of 5 major, multi-center, international
trials, and principal investigator for numerous studies for AMD, DR, and other retinal disorders. Major contributions to medical literature having authored 7 textbooks, more than 250 peer-reviewed papers Recognized by American Academy of
Ophthalmology and American Society of Retina Specialist with Senior Achievement Awards. Peter Kaiser, MD Harvard Medical School
2004 2006 2011 2012 Ranibizumab nAMD 2014 2019 Aflibercept DME 2022 MOA
focused on VEGF and local delivery have demonstrated efficacy for approved treatments, are the current standard of care, and have been highly effective for wAMD/DME. However, these therapies have limited use in
DR AfliberceptnAMD Faricimab-svoa nAMD/DME Pegaptanib nAMD $9b+ 2021 Revenue $2b+ 2021 Revenue Ranibizumab DME Brolucizumab-dbll AMD IVT Anti-VEGF Therapies are Standard of Care for AMD/DME Anti-VEGF Therapies Over the Decades;
Limited Use in DR Patients Source: Company websites
Panorama Study Further Emphasizes Need for Proactive Treatment of NPDR Eyes
Treated with Aflibercept Showed a >2-step Improvement in DRSS Level at 24 and 52 Weeks Brown DM, Wykoff CC, Boyer D, Heier JS, Clark WL, Emanuelli A, Higgins PM, Singer M, Weinreich DM, Yancopoulos GD, Berliner AJ, Chu K, Reed K, Cheng Y,
Vitti R. Evaluation of Intravitreal Aflibercept for the Treatment of Severe Nonproliferative Diabetic Retinopathy: Results From the PANORAMA Randomized Clinical Trial. JAMA Ophthalmol. 2021 Sep 1;139(9):946-955. doi:
10.1001/jamaophthalmol.2021.2809. PMID: 34351414; PMCID: PMC8343518. Population: Adults with severe NPDR w/o DME 225 Male; 177 Female Mean Age: 56 years (10.5) Setting: Global, Multi-Center Study Intervention: 402 Eyes randomized to 3