Opus Genetics, Inc 8-K, filed Mar 29, 2022
Opus Genetics, Inc. (IRD)
8-K
2022-03-29
For: 2022-03-29
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Added on
April 09, 2026
About this 8-K
Opus Genetics, Inc filed this 8-K on March 29, 2022.
Items reported
- Item 7.01: Regulation FD disclosure
- Item 8.01: Other events
- Item 9.01: Financial statements and exhibits
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 OR 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): March 29, 2022
(Exact name of registrant as specified in its charter)
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Registrant’s telephone number, including area code: (248 ) 681-9815
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(Former name or former address, if changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2
of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
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Emerging growth company
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If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 7.01 |
Regulation FD Disclosure.
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On March 29, 2022, Ocuphire Pharma, Inc. (the “Company”) posted on its website an informational presentation regarding the results of its MIRA-3
Phase 3 trial in reversal of mydriasis. A copy of the presentation is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated by reference herein.
The information in this Item 7.01, including Exhibit 99.1 attached hereto, is being furnished, shall not be deemed “filed” for any purpose, and
shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, except as expressly set forth by specific reference in such a filing.
| Item 8.01 |
Other Events.
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On March 29, 2022, the Company issued a press release regarding the results of its MIRA-3 Phase 3 trial in reversal of mydriasis. A copy of the
press release is filed as Exhibit 99.2 to this Current Report on Form 8-K and incorporated herein by reference.
Information contained on or accessible through any website reference in the press release is not part of, or incorporated by reference in, this
Current Report on Form 8-K, and the inclusion of such website addresses in this Current Report on Form 8-K by incorporation by reference of the press release is as inactive textual references only.
| Item 9.01 |
Financial Statements and Exhibits.
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(d) Exhibits
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Exhibit
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Exhibit Description
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Investor Presentation Materials, dated March 29, 2022
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Press Release, dated March 29, 2022
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104
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Cover Page Interactive Data File (embedded within Inline XBRL document).
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto
duly authorized.
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OCUPHIRE PHARMA, INC.
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By:
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/s/ Mina Sooch
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Mina Sooch
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Chief Executive Officer
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Date: March 29, 2022
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Exhibit 99.1
MIRA-3 Phase 3 Trial Results Conference Call March 29, 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 regulatory timelines, commercial timelines, cash runway, and future clinical trials in
reversal of mydriasis (RM), presbyopia, night vision disturbance (NVD) and diabetic retinopathy (DR)/diabetic macular edema (DME), and the potential market opportunity in RM. 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 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; (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 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 construed
as an endorsement of such products.
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 Member Mitch Brigell, PhD,
Head of Clinical Development Susan Benton, MBA, Corporate Board Member Bindu Manne, Head of Market Development and Commercialization Charlie Hoffmann, MBA, VP of Corporate Development and Operations Amy Rabourn, MAcc, VP of
Finance Second Phase 3 RM Trial Topline Readout as Planned in 1Q22
Highlights and Overview
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
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 >340Subjects Dosed Patents
to2034+ APX3330 Oral REF-1 Inhibitor New Chemical Entity (NCE) Nyxol® Novel α1/ α2 Blocker 505(b)(2) 10 Completed Phase 1,Phase 2, and Phase 3 Trials >600Subjects Dosed Exposure in Humans 28 Days Patent
Coverage2034+ Exposure in Humans 365 Days Refractive Retina Presbyopia Reversal of Mydriasis Night Vision Disturbances Diabetic Retinopathy Diabetic Macular Edema DR DME RM P NVD US Market Opportunity $2B - $4B $10B -
$20B ~$500 M US Market Opportunity $10+B Oral Rx Revenues*
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 Phase 3 program planned to initiate in mid-2022 Presbyopia (P) Nyxol® + Low-Dose (0.4%) Pilocarpine Eye Drops Dim Light or Night Vision Disturbances (NVD) Nyxol® Eye Drop LYNX-1 Phase 3 data expected in 2Q 2022
(n=145) Diabetic Retinopathy (DR)/ Macular Edema (DME) APX3330 Oral Pill ZETA-1 Phase 2b data expected in 2H22 (n=103) DME or Wet Age-Related Macular Degeneration (wAMD) APX2009 (Intravitreal or Local Delivery) Seeking partner
funding for IND enabling studies and further development ✓ MIRA-3 Ocuphire Pipeline & Clinical Milestones Note: 0.75% Nyxol (Phentolamine Ophthalmic Solution) is the same as 1% Nyxol (Phentolamine Mesylate Ophthalmic
Solution) Multiple Phase 3 & Phase 2 Clinical Data Readouts Anticipated this Year MIRA-2 VEGA-1 Recent Positive Trial Data ★ Ongoing Trial ★ ZETA-1 ★LYNX-1 ★ MIRA-4
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 mesylate is approved for 2
indications: Regitine® (Pheochromocytoma) – intravenous injection approved in 1952 OraVerse® (Reversal of oral anesthesia) – intramuscular injection approved in 2008 505(b)(2) Regulatory Approval Pathway Nyxol blocks α1 receptors only
found on the Iris Dilator Muscle ↓ Decreases Pupil Size (Moderate Miosis) without Affecting the Ciliary Muscle Iris Dilator Muscle Iris Sphincter Muscle
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 No Headaches Nyxol Product
Candidate Profile Nyxol Clinical Trials Novel, Differentiated Alpha 1/2 Blocker Eye Drop for Refractive Indications Favorable Safety Profile Efficacy Data Nyxol: 0.75% Phentolamine Ophthalmic Solution Preservative Free, EDTA Free, and
Stable Effects Last ≥ 24 Hours Chronic daily dosing of Nyxol at bedtime reduces pupil size for up to 24 to 36 hours Durable
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 did not
expect my dilation to last that long. I have to stay indoors. They say it only lasts a few hours, but it lasts all day, and it is very annoying. RM
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) Difficulty Reading and
Driving Halos and Glare 1. GlobalData Market Research Survey; Oraverse and Regitine Label Patients Report Significant Side Effects after Dilated Eye Exam Note - Tropicamide and Cyclopentolate have same MOA NO REVERSAL DROPS COMMERCIALLY
AVAILABLE The Problem 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 review. Am J Health Syst Pharm
62, 691–699. Parasympathetic innervation stimulates the iris sphincter and ciliary muscle Sympathetic (primarily α1) innervation stimulates the iris dilator muscles 2 Classes of Mydriatic Agents Tropicamide
(anti-cholinergic) Phenylephrine (α1 agonist) Reversal via the Ciliary Muscle by Cholinergic Agonists* is Not a ‘Safe’ Option Retinal tear has been reported in some patients, especially high myopes1 Induces accommodation spasm and
reduction in distance vision2 Induced anterior shift of the lens can increase the risk of acute angle-closure glaucoma2 High incidence of brow ache and headache following installation3 * Cholinergic Agonists include pilocarpine, carbachol,
and aceclidine. Note, pilocarpine is rarely used off-label for RM given these safety concerns. Nyxol® is the only eye drop in clinical development for multiple indications with a MOA that does not affect the ciliary muscle RM
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
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 Placebo drop(s) (2
drops study eye, 1 drop fellow eye) Primary: % of subjects (study eye) returning to baseline (within 0.2 mm) pupil diameter (PD) at 90 min Key Secondary: % of subjects returning to baseline at 0min, 30min, 1h, 90 min 2h, 3h, 4h, 6h, 24h
(overall, by mydriatic agent, by iris color) Mean time to return to baseline PD Mean change in pupil diameter at all timepoints Distance-Corrected Near Vision Accommodation (Tropicamide/Paremyd) Safety and
tolerability Endpoints MIRA-3 Started in Nov 2021 Enrolled 368 in Feb 2022 Phase 3 Results Reported March 2022 Screening Randomization 2 1 Treatment (Max Dilation) 0 min 30min 1 Hr 90min 2 Hr 3 Hr 4 Hr 6 Hr 24
Hr Primary Endpoint Follow Up Visit Mydriatic Agents 3:1:1 – A: 2.5% phenylephrine (alpha-1 agonist), B: 1% tropicamide (cholinergic blocker), C: Paremyd® (combination) : Inclusion: Healthy ≥ 12 years of age Exclusion: Clinically
significant ocular trauma, surgery, or non-refractive laser treatment within the 6 months prior to screening; and recent or current evidence of ocular disease, infection or inflammation in either eye Key Eligibility Criteria RM
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 American Indian
or Alaska Native; Native Hawaiian or Other Pacific Islander 182 (74.6%) 38 (15.6%) 22 (9.0%) 0 (0%) 93 (75.0%) 21 (16.9%) 9 (7.3%) 1 (0.8%) 274 (74.5%) 59 (16.0%) 31 (8.4%) 7 (1.9%) Light Iris Color: n (%) 113 (46.3%) 58
(46.8%) 171 (46.5%) Dark Iris Color: n (%) 131 (53.7%) 66 (53.2%) 197 (53.5%) Treatment and Placebo Arms Were Balanced in MIRA-3 Phase 3 Registration Trial Source: MIRA-3 Table 14.1.2.1 (ARP) (mITT). Notes: 32 pediatric subjects
12-17years old were enrolled in the trial. Race is more than 100% given subjects could check more than one category. Demographics represent all randomized population (ARP) of 368 which is the same as Safety Population and
Modified-Intent-to-Treat (mITT). Per Protocol (PP) Population is 345, excludes 23 subjects who did not dilate more than 0.2 mm 1 hour after receiving mydriatic drop. RM
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 =
20/20 65 65 65 IOP (mmHg) 16.2 16.1 16.1 Treatment and Placebo Arms Were Balanced Across Ocular Measures in the MIRA-3 Trial Source: MIRA-3 Table 14.1.2.1 (ARP) (mITT). RM
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
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 Nyxol MIRA-3 Phase 3 Trial MIRA-2 Phase 3
Trial RM
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
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 Starting at 1 Hour Post-Dose Through 6
Hours MIRA-3 Phase 3 Trial 1.5 0.5 RM
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 3 Trial Mean PD - Study Eye
(mITT) RM p=0.001 Mydriatic Max Dilation; Treatment 1.5 1.5
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 in Both Light and Dark
Irides MIRA-3 Phase 3 Trial Mean PD - Study Eye (mITT) 24 0.5 0.5 1.5 1.5 RM Max dilation; Treatment Mydriatic p=0.0007 p<0.0001
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 hrs 3.4 hrs
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
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% placebo) Less than 1% of subjects
reported instillation site discomfort, pain, or irritation Conjunctival hyperemia was observed to be mild and transient Visual acuity (distance and near) was not adversely affected by Nyxol Over 300 subjects have been treated with Nyxol
and evaluated at 24-hours in the MIRA trials satisfying regulatory requirements for drug safety exposure for the acute RM indication Source: MIRA-3 Table 14.3.1.1; MIRA-3 Table 14.3.1.2.2; MIRA-3 Table 14.3.3.2 (Safety Population). Nyxol
was Well Tolerated with a Favorable Safety Profile RM
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 with high statistical
significance Efficacy seen at all timepoints from 60 minutes to 24 hours Similar efficacy for one drop and two drops Efficacy across all 3 mydriatic agents – phenylephrine, tropicamide, and Paremyd® Efficacy in both light and dark iris
colors Accelerated return to normal distance-corrected near visual acuity Favorable safety and tolerability profile No serious AEs, no drop-outs from AEs No systemic or ocular AEs were observed in ≥ 5% of subjects, except for 11% mild,
transient conjunctival hyperemia NDA planned for late 2022 MIRA-3 Topline Reports Confirms Prior Phase 3 Study Showing Substantial Benefit in Accelerating Reversal of Mydriasis RM
Plans to NDA for Nyxol in RM
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 2022 Primary
Endpoint Met ✓ Secondary Endpoints Met ✓ Primary Endpoint Met ✓ Secondary Endpoints Met ✓ Primary Endpoint Met ✓ Secondary Endpoints Met ✓ RM Fully Enrolled 3-11 years old
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 Clinical Trial Completed 3 registration
batches; 1-year CMC stability will be available for NDA Manufacturing Submit NDA by late 2022, with expected approval review of 10 months Regulatory Approval Nyxol® Target Label Indication The treatment of pharmacologically induced
mydriasis produced by adrenergic (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents, or a combination thereof. Preservative-Free Single Unit Vial (5-pack) ✓ Ongoing Ongoing RM
Reversal of Mydriasis Market Opportunity
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 visits, pupils are
pharmacologically dilated, impairing vision for 6-24 hours Dilated eyes experience: Heightened sensitivity to light Inability to focus, headaches Difficulty reading, working & driving Halos and glare Cycloplegia (loss of
accommodation) Current Landscape: Rare off-label use of cholinergic agonists (e.g., pilocarpine) given ciliary muscle safety issues1,2 Optomap® is offered by optometrists to avoid dilations for ~$50 cash-pay, however images may provide
limited view of retina and disease pathology3 No Currently Available Treatments 100M Annual Eye Dilations Nyxol’s MOA Uniquely Suited As A Reversal Drop For Dilations The Problem RM
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 validation assumed each unit (bottle) has ~10
mL fill volume and each patient gets 2-4 drops Number of Providers (X) Average Number of Weekly Exams (Y) Estimated % Patients Dilated(Z) Total(X*Y*Z) * 48 wk/yr Optometrists 46,000 59 40% ~52
M Ophthalmologists 18,000 88 50% ~38 M Retina Specialists 3,000 150 50% ~10 M 100M Annual Dilated Eye Exams Supply Side Validation: Based on the ~2 million total units of mydriatic agents sold in 2020, we calculated the total
number of dilated eye exams to be ~125 million patients, consistent with demand side estimates. Demand Side Validation RM
~$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% of Patients Likely to Request
Drop 58% physicians would start prescribing Nyxol within 1st year 81% patients would be more likely to schedule yearly eye exams with a reversal drop 0 Current Commercially Available Treatments MIRA Trials Represent 95% of Dilation
Drops Used in Practice Source: GlobalData Market Research SurveyCalculation: 100M Annual Eye Dilations X 65% X 80% X $10 per patient = $500+M Opportunity 68% physicians would be willing to use Nyxol even if patients had to still wear
sunglasses within 1st hour GlobalData Market Research Findings 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 payor (reimbursement)
dependence Significant prior authorization & step-edits hurdles with burden to the practices Lengthy sales cycles and touchpoints due to chronic use and market access upkeep Significant product education requirement Complex
distribution channel including specialty and retail pharmacies “One product, one indication” commercial model is inefficient with fixed cost infrastructure Ocuphire’s Nyxol RM Launch Ocuphire’s Nyxol RM Launch No competition or approved
reversal drop potential for Nyxol to be the only safe option Cash pay (no reimbursement barriers) allowing for quicker adoption Offering a significant value proposition to patients and practices Shortened sales-cycle with acute use
product No training given dilations routine in practices No specialty/retail pharmacy direct to physician “One product, several indications” offers efficiencies in commercial operations RM
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 Targeting Pre-Commercial
Activity Market Development (KOLs) Patient Journey Brand Awareness No approved drug/competition; data-mining for high volume practices Landscape No need for pharmacy; no reimbursement, private pay Direct to Physicians Dilations
are a routine part of practice; adoption requires no staff or patient training Easy Adoption Components of an Efficient Launch Retina 3,000 Retinal Specialists Ophthalmology 20,000 Ophthalmologists Optometry 46,000 Optometrists
Potential Options for Commercialization Go-To-Market Strategy Work with strategic or channel partner with existing commercial ophthalmic products Hire contract commercial organization Build own salesforce RM
Upcoming Milestones
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 NVD (LYNX-1) Report Pediatric Data
for RM (MIRA-4) Submit Nyxol NDA for RM Report Phase 2 Data for DR/DME (ZETA-1) Initiate VEGA Phase 3 Presbyopia Program Multiple Late-Stage Data Catalysts Expected in 2022 for Potential First NDA Approval in 2023
Exhibit 99.2
Ocuphire Announces Positive Topline Results from MIRA-3 Phase 3 FDA Registration Trial for Nyxol® in the Reversal of
Mydriasis
Meets Primary Endpoint With 58% Of Nyxol treated Subjects Returning to Baseline Pupil Diameter at 90 Minutes Compared to 6% of Placebo
Subjects (p<0.0001)
MIRA-3 Confirms Prior MIRA-2 Phase 3 Registration Trial Showing Substantial Benefit in Accelerating Reversal of Mydriasis (RM)
NDA Filing for Nyxol in RM Planned for Late 2022
Potential Launch as Only Dilation Reversal Drop in 2H 2023
Conference Call and Webcast Today at 8.30am ET
FARMINGTON HILLS, Mich., March 29, 2022 - Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on
developing and commercializing therapies for the treatment of refractive and retinal eye disorders, today announced positive topline results in the MIRA-3 trial, the company’s second Phase 3 registration trial investigating its product candidate
Nyxol® for the reversal of pharmacologically-induced mydriasis (dilation of pupil). Ocuphire announced positive results from its first Phase 3 trial, MIRA-2, in March 2021.
Nyxol is a proprietary, preservative-free, stable, investigational eye drop formulation of phentolamine mesylate designed to reduce pupil size by
inhibiting contraction of the iris dilator muscle. MIRA-3 was designed as a multi-center, randomized, parallel arm, double-masked, placebo-controlled Phase 3 trial evaluating the safety and efficacy of Nyxol in subjects with
pharmacologically-induced mydriasis. MIRA-3 enrolled 368 subjects from November 2021 to February 2022 at 16 sites in the U.S.
These topline results demonstrated that the MIRA-3 trial met its primary endpoint with 58% of subjects (study eye) treated with Nyxol returning to ≤ 0.2
mm of their baseline pupil diameter (PD) at 90 minutes compared to only 6% of subjects (study eye) treated with placebo (p <0.0001). The effect was also significant at 60 minutes (Nyxol 42% vs. placebo 2%, p <0.0001). In comparison, only
36% of placebo treated subjects returned back to baseline PD at 6 hours. These results showed clinically meaningful differences between Nyxol and placebo for accelerating reversal of pharmacologically-induced mydriasis.
“The successful completion of the MIRA-3 Phase 3 trial is a major milestone in our development program for Nyxol in RM,” said Mina Sooch, MBA, President
and CEO of Ocuphire Pharma. “We are delighted with the positive efficacy and safety outcomes which confirm the results from our prior MIRA-2 Phase 3 trial. We now have over 900 subjects studied across 10 clinical trials of which over 550 have
been exposed to Nyxol. Importantly, today’s announcement means that that we have two FDA registration trials to support potential approval for the RM indication. We intend to file an NDA with the U.S. FDA in late 2022, which, if approved, would
position Ocuphire for commercial launch of Nyxol in RM in the second half of 2023. We want to thank the study participants, physicians, study site personnel, and everyone who was involved in the MIRA-2 and MIRA-3 trials for their contribution in
advancing this program and bringing us closer to potentially delivering an FDA-approved treatment for RM.”
Highlights of MIRA-3 Efficacy and Safety Results
MIRA-3 (NCT05134974) is a Phase 3 registration trial evaluating
the product candidate Nyxol to expedite the reversal of pharmacologically induced mydriasis. In the trial 368 study participants (336 adults and 32 adolescents at or over age 12) were randomized 2:1 to receive Nyxol (0.75% phentolamine ophthalmic
solution) or vehicle control (placebo) 1 hour after receiving one of 3 mydriatic agents. The three mydriatic agents used in this trial were phenylephrine 2.5% (alpha 1 agonist targeting the iris dilator muscle), tropicamide 1% (cholinergic
blocker targeting the iris sphincter muscle), and Paremyd® (a combination of hydroxyamphetamine hydrobromide 1% and tropicamide 0.25%), which are all commonly used in optometry and ophthalmology offices to dilate patients’ pupils for
annual or special exams as well as surgical procedures. The study population was comprised of subjects in the modified Intent to Treat population (mITT).
Summary of MIRA-3 Topline Data
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The primary endpoint was met with 58% of subjects (study eye) treated with Nyxol returning to ≤ 0.2 mm of their baseline pupil diameter at 90 minutes compared to
only 6% of placebo treated subjects (p <0.0001) across the three mydriatic agents.
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Key secondary efficacy endpoints also met statistical significance:
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Early onset of action with 42% of subjects at baseline PD by 60 minutes post-dose (vs. 2% placebo, p<0.001)
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Significantly more Nyxol-treated subjects returned to normal PD or smaller than placebo-treated subjects at all time points from 1 hour to 24 hours
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Similar efficacy was seen with one or two drops of Nyxol (as the study eye was treated with 2 drops and the fellow eye with one)
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Nyxol was effective regardless of iris color or mydriatic agent used
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Approximately 4 hours were gained in time to return to normal pupil diameter overall and across mydriatic agents and iris colors
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Nyxol restored normal distance corrected near vision significantly faster than placebo
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Nyxol demonstrated a favorable safety and tolerability profile.
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Nyxol was well tolerated with no serious adverse events or withdrawals due to adverse events
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The only AE occurring in greater than 5% subjects was mild, transient conjunctival hyperemia (11%)
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Jay S. Pepose, MD, PhD, Director of the Pepose Vision Institute, Professor of Clinical Ophthalmology at Washington University School of Medicine, and
Ocuphire Medical Advisory Board member and Board member commented, “Nyxol's unique MOA makes it an ideal agent for reversal of mydriasis, as it does not have the
potential safety risks of retinal tears, accommodative spasm and angle closure associated with cholinergic agents like pilocarpine. The MIRA-3 and MIRA-2 trials confirm the favorable safety profile and efficacy, showing rapid reversal of
mydriasis following dilation with all mydriatic agents tested and in both light and dark iris colors. In addition, the pupil reduction of 1 to 1.5 mm from baseline through 24 hours is a potential read through for our other clinical indications
for Nyxol including presbyopia and night vision disturbances.”
Edward Holland, MD, Director of Cornea Services at Cincinnati Eye
Institute and Ocuphire Medical Advisory Board member commented, “Pupil dilation is a necessary tool for ophthalmologists and optometrists to screen for and monitor diseases of the eye. However, patients often find dilation problematic, citing
unwanted symptoms including inability to read, photophobia, loss of accommodation, and inability to work effectively. Many patients complain about or refuse dilation for these reasons. There are no approved treatments currently available for
reversal of mydriasis, and with the announcement today of positive results from MIRA-3, I am very pleased to see the continued progress in advancing Nyxol toward potential FDA approval. If approved, I believe that Nyxol would be widely used in
clinical practice, which could increase the overall number of dilated exams as well as improve patient experience, and lead to better eye health for our patients.”
For more information about the MIRA-3 Phase 3 trial design, please visit www.clinicaltrials.gov (NCT05134974). Ocuphire collaborated closely with Oculos Development Services, a Rush, NY based clinical research organization and subsidiary of Iuvo BioScience, on the execution of the MIRA-3 trial.
Nyxol Development Plan and Next Steps in RM
Ocuphire recently completed enrollment of 23 pediatric subjects in the MIRA-4 trial evaluating the safety and efficacy of Nyxol eye drops to reverse
pharmacologically-induced mydriasis. Top line results are expected in the second quarter of 2022. If MIRA-4 meets its endpoints, the results would potentially support a broader label for Nyxol in RM to include children as young as age 3. Ocuphire
is also on track to complete the Chemistry, Manufacturing and Controls (CMC) section of the NDA as three registration batches of Nyxol have been completed and on stability. The company plans to file an NDA that includes the results of MIRA-1,
MIRA-2, MIRA-3, and MIRA-4 with the U.S. FDA in late 2022.
Reversal of Mydriasis Market Opportunity
Every year in the U.S., an estimated 100 million eyes dilations are conducted to examine the back of the eye, either for routine check-ups, disease
monitoring or surgical procedures, across all eye care practice groups. Depending on the individual and the color of their eyes, the pharmacologically-induced dilation can last anywhere from 6 to 24 hours. Dilated eyes have heightened sensitivity
to light and a decreased ability to focus on near objects, causing difficulty reading, working, and driving. Currently, there are no approved or available options to safely reverse mydriasis. Nyxol has the potential to be the first and only
FDA-approved agent for RM.
Market research conducted by GlobalData surveyed several hundred patients and eye care providers (optometrists and ophthalmologists) about Reversal of
Mydriasis. Over 65% of surveyed patients reported moderate to severe negative impact of a dilated pupil. These data underscore the potential value of the role of the investigational product candidate Nyxol in improving comfort and daily function
after pupil dilation. Furthermore, approximately 80% of patients responded that they would be likely to request a dilation reversal drop, and more than 70% of eye care providers would be likely to use a reversal drop. The market research
confirmed patients’ willingness to pay out-of-pocket to reverse their dilations, supporting a market size estimate of over $500M. Ocuphire is currently evaluating partnering options for an effective and cost-efficient commercial launch of Nyxol
targeted for the second half of 2023.
Conference Call and Webcast (with slides)
A more detailed presentation of the topline MIRA-3 results will be discussed on a conference call and webcast at 8.30am EDT this morning and will be
posted shortly thereafter to the Investors section of Ocuphire’s corporate website under the Events heading, where it will be archived and available for 90 days.
Details for the call are as follows:
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1-877-407-4018
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| International: |
1-201-689-8472
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| Conference ID: |
13728061
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| Webcast: |
Link
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About Ocuphire Pharma
Ocuphire is a publicly-traded (NASDAQ: OCUP), clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for
the treatment of several eye disorders. Ocuphire’s pipeline currently includes two small-molecule product candidates targeting refractive and retinal indications. The company’s lead product candidate, Nyxol® eye drops (0.75% phentolamine
ophthalmic solution) is a once-daily, preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size, and is being developed for several indications,
including reversal of pharmacologically-induced mydriasis (RM), presbyopia and dim light or night vision disturbances (NVD), and has been studied in 10 completed clinical trials. Ocuphire has reported positive topline data from MIRA-2 and MIRA-3,
two registration trials for the treatment of RM, and recently completed enrollment in a pediatric safety trial (MIRA-4) in RM. Ocuphire also reported positive top-line data from a Phase 2 trial of Nyxol for treatment of presbyopia, both Nyxol as a
single agent and Nyxol with low-dose pilocarpine (“LDP”) 0.4% as adjunctive therapy. The company recently completed enrollment in its Phase 3 study of Nyxol for NVD (LYNX-1). Ocuphire’s second product candidate, APX3330, is an oral tablet designed
to inhibit angiogenesis and inflammation pathways relevant to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME) and has been studied in 11 Phase 1 and 2 trials. The company recently
announced the completion of enrollment in a Phase 2b clinical trial of APX3330 to treat DR/DME (ZETA-1). Please visit www.clinicaltrials.gov to
learn more about Ocuphire’s Phase 3 registration trial in RM discussed herein (NCT05134974), pediatric safety study in RM (NCT05223478), Phase 3 registration trial in NVD (NCT04638660), and
Phase 2b trial in DR/DME (NCT04692688). Ocuphire previously completed the first Phase 3 registration trial in RM (NCT04620213) and Phase 2 trial in presbyopia (NCT04675151). As part of its
strategy, Ocuphire will continue to explore opportunities to acquire additional ophthalmic assets and to seek strategic partners for late-stage development, regulatory preparation, and commercialization of drugs in key global markets. For more
information, visit www.ocuphire.com.
Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, timing and results in RM, presbyopia, NVD and DR/DME future clinical trials, potential market size of RM, as well as
statements concerning the success and timing of planned regulatory filings and commercialization. These forward-looking statements are based upon Ocuphire’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 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; (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 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 Ocuphire from time to time with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they
were made. Ocuphire undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Ocuphire Contacts
Mina Sooch, President & CEO
Ocuphire Pharma, Inc.
ir@ocuphire.com
www.ocuphire.com
Corey Davis, Ph.D.
LifeSci Advisors
cdavis@lifesciadvisors.com