Ocuphire Pharma Investor Presentation Deck

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#1Glaucoma & Anterior Segment Eyecelerator@AAO 2021 November 11, 2021 Ocuphire Restore Vision & Clarity Mina Sooch CEO and Founder#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 Ocuphire Pharma, Inc.'s ("Ocuphire" or the "Company") product candidates and future milestones, including the potential for Nyxol to be a "best in class" presbyopia drop. 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) timing or ability for the company to achieve its targeted milestones; (ii) the success and timing of regulatory submissions and pre-clinical and clinical trials; (iii) regulatory requirements or developments; (iv) changes to clinical trial designs and regulatory pathways; (v) changes in capital resource requirements; (vi) risks related to the inability of the Company to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; (vii) legislative, regulatory, political and economic developments, and (viii) the effects of COVID-19 on clinical programs and business operations. 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 may not be reproduced or provided to any other person (other than your advisor) without our prior written consent. By accepting delivery of this presentation, you agree to the foregoing and agree to return this presentation and any documents related thereto and any copies thereof to us or to destroy the same if you do not make an investment in any securities. The information contain within this presentation shall not, except as hereinafter provided, without the prior written consent of the Company, be disclosed by you or your representatives in any manner whatsoever, in whole or in part, and shall not be used by you or your representatives other than for the purpose of evaluating the transaction described herein. By accepting delivery of this presentation you further acknowledge and agree aware of the restrictions imposed by the United States securities laws on the purchase or sale of securities by any person who has received material, nonpublic information from the issuer of the securities or any affiliate thereof and on the communication of such information to any other person when it is reasonably foreseeable that such other person is likely to purchase or sell such securities in reliance on such information for so long as the information remains material and non- public. 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. November 6, 2020 NASDAQ CONGRATULATES OCUPHIRE ON RECENT LISTING AND CAPITAL RAISE OCUPHIRE PHARMA Ocuphire PHARMA OCUP NasdaqListed Nasdaq SON#3Ocuphire Overview A Late-Stage Clinical Ophthalmic Biotech (Nasdaq Symbol: OCUP) Nyxol® APX3330 3 Presbyopia U.S. Prevalence: ~128M 0. 4 % Positive Phase 2 Top-line Data 2Q21 Advance Two Phase 3 in 2022 Phase 2 Recruiting Phase 2 Top-Line Data in 2022 Night Vision Disturbances U.S. Prevalence: ~16M adults Phase 3 Recruiting Phase 3 Top-Line Data early 2022 Diabetic Retinopathy U.S. Prevalence: ~ 7M Reversal of Mydriasis ~100M pupil dilations per year in U.S. Positive Phase 3 Top-Line Data 1Q21 2nd Phase 3 Planned 2H21 2nd Phase 3 Top-Line Data early 2022 NDA Submission Late 2022 Diabetic Macular Edema U.S. Prevalence: ~750K Ocuphire PHARMA#4September 22, 2021 | 11 min read Treatment landscape for presbyopia evolving toward noninvasive options 2021: The Time for Presbyopia Drops Headlines From Academia and Industry Articles Thru the Year with an Early First Approval Presbyopia PHYSICIAN JULY 201 In the Face of Presbyopia... A New Conversation is Imminent 4 New options are on the horizon for presbyopia-correcting drops August 30, 2021 Dr Marguerite B. McDonald Ophthalmology Times Europe Journal, Ophthalmology Times Europe September 2021, Volume 17, Issue 07 How ophthalmic leaders view solutions today and those on the horizon SAVE Article 口 By Conni Bergmann Koury July 1, 2021 Presbyopia treatment options now and on the horizon "The correction of presbyopia remains ophthalmology's 'Holy Grail'..." Refractive September 2021 -OIS Presbyopia-correcting drops: The next frontier Pharmaceuticals are poised to enhance near vision for millions of presbyopes. Sources: Academic review articles, journals, and publications Clinical Ophthalmology FDA APPROVAL OF ABBVIE EYE DROP A NEW MOMENT IN PRESBYOPIA 10/29/2021 Presbyopia A Review of Current Treatment Options and Emerging Therapies - CLINICAL UPDATE Dovepress REVIEW Presbyopia-Correcting Eyedrops Move Ahead Presbyopia PHYSICIAN NOVEMBER 2021 HELP ON THE WAY ent options he corner @ A Review of Pharmacological Presbyopia Treatment Andrzej Grzybowski, MD, PhD, MBA", Agne Markeviciute, MD, and Reda Zemaitiene, MD, PhD| Presbyopia Treatment Market Size Projected to Rise Lucratively by 2026 end BioSpace PentaVisien How Presbyopia Correction Drops Will Change My Treatment Regimen CRST Cataract & Refractive Surgery Today Ocuphire PHARMA#5Product Profile: Nyxol® + Low-Dose Pilocarpine (LDP) Combo Moderate Action on Iris Dilator and Iris Sphincter Muscles for Near Vision Improvement 0.75% Nyxol 5 + 0.4% HO 0.4% LDP Iris Dilator Muscle Inhibition Iris Sphincter Muscle Activation Source: 1) Nyxol® data from 8 completed trials; Pilocarpine Product label and Literature Evening drop Daytime drop ● ● ● Phentolamine (alpha1/2 antagonist) approved non- ocular injectable indications decade(s) ago 505(b)(2) Novel MOA on iris dilator with 24+ hour durability Moderate 1+mm pupil reduction No daytime redness w/ chronic evening dosing Nyxol Well-tolerated with no systemic effects Stable, preservative-free, single use vial 1.5 to 2.5 mm PD reduction moves toward the pin-hole (2 to 2.5 mm, up to 3 mm) Pilocarpine (cholinergic agonist) approved decades ago Known MOA on sphincter muscle with potent miotic effects at approved doses (1%, 2%, 4%) Chronic daytime dosing of LDP Low concentration avoids known tolerability issues: ➤ headache and browache redness accommodative spasm causing loss of distance vision especially at night Ocuphire PHARMA#66 ● ● Presbyopia VEGA-1 Phase 2 Design Randomized, Double-Masked, Placebo-Controlled, Multi-Center One-Week Trial VEGA-1 17 US sites 150 presbyopia patients 0.75% Nyxol 4 arms Placebo Randomization Visit 1 Baseline Baseline Baseline Baseline Screening Evening Dosing (3-4 doses) Nyxol Nyxol Placebo Placebo Visit 2 (3-6 Days Later) LDP Drop No Treatment LDP Drop No Treatment Treatment Arms Nyxol + LDP Nyxol Alone LDP Alone Placebo Alone Eligibility Criteria Males or females ≥ 40 and ≤ 64 years of age BCDVA of 0.0 LogMAR(20/20 Snellen equivalent) or better in each eye under photopic conditions DCNVA of 0.4 LogMAR (20/50 Snellen equivalent) or worse in photopic conditions in each eye & binocularly Phase 2 Enrollment Completed Feb to May 2021 – 150 Subjects Reported Topline Results End of 2Q21 Clinical trial NCT#04675151. DCNVA = distance-corrected near visual acuity. BCDVA = best corrected distance visual acuity Endpoints Primary: % of subjects with ≥ 3 lines of improvement in distance- corrected near visual acuity comparing Nyxol + LDP vs placebo alone at 1 hour Secondary: ● • % of subjects with ≥ 2 and ≥ 3 lines gained at time points from 30 min to 6 hours in photopic lighting comparing Nyxol + LDP vs placebo, Nyxol alone, and LDP alone No loss of distance vision Pupil diameter at time points Safety and tolerability (redness) ● Ocuphire PHARMA#77 Efficacy Endpoints: % of Subjects ≥ 15 Letter DCNVA Gain Across Timepoints Nyxol + LDP had Strong Response with ≥ 15 Letter Near Gain from 30 Minutes to 6 Hours Percent of Subjects (%) 70% 60% 50% 40% 30% 20% 10% 0% Percent of Subjects with ≥ 15 Letters Binocular Photopic DCNVA Improvement from Baseline 33% Placebo Adjusted Response p=0.003 Nyxol alone effect (~12 hr data) p=0.09 33% 16% 0 Rapid onset of efficacy p=<0.0001 61% 14% VEGA-1 Phase 2 Trial 0.5 28% 1 61% Primary Endpoint p=<0.0001 63% Placebo (n=43) 16% 2 Time (Hours) p=0.02 47% 21% Nyxol+LDP (n=43) 3 p=0.02 21% 4 47% Durable benefit over 6 hours p=0.06 37% 19% Source: VEGA-1 TLR Table 14.2.1.2 Percent of Subjects with Improvement From Baseline in Photopic DCNVA by Time Point (PP Population). 15 letters is 3 lines. Ocuphire PHARMA#88 Secondary Endpoint: Improvement in DCNVA Baseline vs. 1 Hour Post Nyxol+LDP Nyxol + LDP had a Rapid Improvement on Near Vision for Many Patients Percent of Subjects (%) 45% 40% 35% 30% 25% 20% 15% 10% 5% 0% 0% 7% 20/15 0% 12% 20/20 Binocular Photopic DCNVA Nyxol + LDP Improvement in Snellen Equivalent 84% 20/40 or Better Near Vision 68% 20/32 or Better Near Vision 0% 23% 20/25 0% 26% 20/32 VEGA-1 Phase 2 Trial 0% Baseline -16% 20/40 33% Snellen Acuity №% 20/50 1-Hour 40% 7% 20/63 23% 0% 20/80 Source: VEGA-1 TLR Table 14.2.24.1 Percent of Subjects with Photopic DCNVA by Time Point (PP Population) 2% 0% 20/100 Visual Acuity Chart K CRHN ZKDV C HVORK RHSON KSVRH HNK CD NDVKO DHOSZ VRNDO CZHKS ORZSK Before 20/63 After 20/32 Ocuphire PHARMA#99 Secondary Endpoint: Intermediate Vision Nyxol + LDP Had Significant Improvement in Letters Read in DCIVA Compared to Placebo Change In Letters Read From Baseline (Letters Read) 12 10 8 p<0.05 6 2 0 Change from Baseline in Binocular Photopic DCIVA (Letters Read) p=<0.0001 VEGA-1 Phase 2 Trial 1 2 p<0.05 3 Time (Hours) Placebo (n=43) Nyxol+LDP (n=43) Intermediate Vision Efficacy with Nyxol+LDP 4 LO 5 Source: VEGA-1 TLR Table 14.2.7.1 Observed Values and Change from Baseline in Photopic DCIVA by Time Point (Letters Read) (PP Population) p<0.05 6 Ocuphire PHARMA#1010 Secondary Endpoint: Mean Pupil Diameter Over Time Achieved Pupil Size ~2mm in Nyxol+LDP Consistent with 3-line Improvement in Near Vision Mean Pupil Diameter (mm) 5.0 4.0 3.0 2.0 1.0 4.8 4.4 4.3 4.2 *** Baseline *** 3.1 Daily Evening Nyxol Dosing 12 hr minimum interval to Time 0 0 4.5 *** 4.3 3.2 *** ** 16 4 4.5 3.2 3.0 2.4 *** T *** ** 1 4.4 3.2 2.5 2.1 VEGA-1 Phase 2 Trial Best Eye Mean Pupil Diameter -Placebo (n=43) ***T ***1 ** 4.3 3.4 2.7 2.3 *** *** *** 4.4 3 3.3 3.1 2.5 2 Time (Hours) -Nyxol+LDP (n=43) -Nyxol (n=30) *** *** 1 *** 4.4 3.3 3.3 2.7 Nyxol+LDP arm statistically significant compared to all arms -LDP (n=31) Source: VEGA-1 TLR Table 14.2.12.1 Observed Values and Change from Baseline in Photopic Pupil Diameter by Time Point (PP Population) **p<0.01 ***p<0.0001 5 *** 4.6 6 3.6 ** 3.3 2.9 Ocuphire PHARMA#1111 Additional VEGA-1 Phase 2 Efficacy Results Nyxol + LDP Efficacy Data has Potential for Differentiation ● Met the planned Phase 3 primary endpoint of 15 letters (3 lines) near vision gain with less than 5 letters of distance vision loss Statistical significance and/or trend across multiple timepoints for combination treatment vs. each individual component (Nyxol or LDP) • Met multiple additional key secondary endpoints Nyxol+LDP was numerically better than each component at every timepoint Efficacy data in both light and dark iris colors Near vision efficacy seen monocularly - - Source: VEGA-1 Clinical Study Results Ocuphire PHARMA#1212 Safety Findings Nyxol + LDP Combination Was Well Tolerated with a Favorable Safety Profile • No serious AEs, almost all AEs were mild 0% headaches or brow aches reported for Nyxol+LDP arm ● ● ● ≤ 5% mild, transient conjunctival hyperemia AEs in Nyxol+LDP arm No change in distance vision for Nyxol + LDP arm 0% had ≤ 5 letter distance loss in photopic lighting Only 5% distance loss in mesopic lighting No change in IOP ● ● Source: VEGA-1 Study Results (Safety Population, n=150); Only a single subject difference between mITT (n=148) and PP population (n=147) Ocuphire PHARMA#1313 Summary & Next Steps for Ocuphire Presbyopia Drops Advance Into Phase 3 with a Differentiated Product Profile Important Product Efficacy Attributes Safety: Maintain Distance. Safety: Tolerability Durability Fast Onset Convenient Daily Drops Tunable Pupil Modulation Source: VEGA-1 Clinical Study Results Nyxol+ LDP Clinical Data ✓ ✓ ✓ ✓ ✓ ✓ Ocuphire is differentiated by using both the dilator and sphincter muscles moderately to reach a pin-hole pupil size AAO 2021 Presentations AAO Refractive Surgery Day ePoster VEGA-1 Presbyopia Presentation by Dr. Jay Pepose Abstract # 30068457 AAO Retina Day Scientific ePoster ZETA-1 DR/DME Presentation by Dr. Mike Allingham Abstract #PO332 Advance into Presbyopia Phase 3 With Registration Trials in 1H22 Towards a Potential NDA Filing in 2023 Ocuphire PHARMA

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