Post on 04-Aug-2020
OIS@SECO Presentation on March 4, 2020
Restore Vision & Clarity
Mina Sooch, MBA CEO and Co-Founder
Late Clinical Stage Biotech Targeting
Large, Unmet Ophthalmic Markets
Decades of Clinical Data & Safety Profile
on 2 Acquired Eye Assets
Significant IP Portfolio &
Small Molecule CMC Advantages
Multiple Phase 3 & Phase 2 Data
Catalysts in Next9-18 Months
Experienced Management, Board, and Medical Advisors
2
Two Completed Nyxol® Phase 2b
Trials in 2019
Nyxol® APX3330
Ocuphire SummaryExecuting on a Vision to Advance Late-Stage Ophthalmic Drug Candidates
Large Unmet Opportunities for the Aging EyeDeveloping Drugs to Treat Both Front and Back of the Eye Diseases
BackFront
Diabetic Macular EdemaU.S. Prevalence: 750K
Diabetic RetinopathyU.S. Prevalence: 7+M
PresbyopiaU.S. Prevalence: 110+M
Night Vision DisturbancesU.S. Prevalence: 15-20M
Reversal of Mydriasis80M In-Office Eye Dilations Per Year
Nyx
ol®
AP
X33
30$4-50B US Markets $4-10B US Markets
3
Patents
to
7Phase 1 &
Phase 2
Trials
Up to
28 Days
Exposure in
Humans
>150Subjects
Dosed
APX3330Nyxol®
Extensive Development on Both Drug CandidatesWell-Controlled Trials Provide Foundation for NDA Path
505(b)(2) Development
Pathway
Studied in ocular refractory diseases (NVD) & elderly glaucoma patients
2034+
Patents
to
11Phase 1 &
Phase 2
Trials
More than
365Days
Exposure in
Humans
>400Subjects
Dosed
NCEDevelopment
Pathway
Studied in inflammation/hepatitis & cancer patients
2034+
4
Unique Mechanism of Action for Nyxol®First-in-Class Ophthalmic Formulation with 505(b)(2) Path
• Active in Nyxol® is a previously approved drug phentolamine mesylate (PM)
• PM is a non-selective a1 & a2 blocker
• PM is a lipophilic, small molecule allowing once-daily dose
• Nyxol® has been formulated as a novel, topical eye drop (patents thru 2034 in US/EU/Japan)
Reduces Pupil Size via
5
Relaxes (Vasodilation) via
α1
Smooth MuscleBlockade
(transient, mild redness is an on-target α1 effect
on sclera vessel)
α1Iris Dilator
Blockade
Reduces IOP via
α1: Reduce episcleral venous pressure (EVP)
α2: Increase TM outflow by relaxing contraction of TM
α1: Increase UV outflow and decrease humor production
Nyxol® Product Candidate ProfileFirst-in-Class Alpha 1/2 Blocker Eye Drop Phase 3 Ready for Refractory Indications
Phase 2 Safety Data
↓ Pupil Size (moderate miotic)
↑ Contrast Sensitivity (night)
↑ Near Visual Acuity (light/dark)
↑ Distance Visual Acuity
Improving Vision
Phase 2 Efficacy Data
No Systemic EffectsNo Changes in Blood PressureNo Changes in Heart Rate
Tolerated Topical EffectsMild / Transient / Reversible Eye Redness
IOP Unchanged or Decreased↓ Intraocular Pressure (IOP)
at Normal Baseline
Chronic daily dosing of Nyxol® before bedtime demonstrated no significant daytime redness and durability of effects more than 24 hours
Nyxol®: Phentolamine Mesylate 1% Ophthalmic SolutionPreservative Free and EDTA Free
6
Reversal of Mydriasis (RM) is an Unmet Market Opportunity Single Use Indication Leveraging a Precedent Approval Pathway
The Problem
• After every comprehensive annual eye exam and specialty exams, your pupils become dilated, impairing vision for 4-8 hours
• High sensitivity to light and inability to focus makes reading, working, and driving difficult
Nyxol’s PotentialDifferentiated Solution
• Drug Precedent: Rev-Eyes (a specific alpha 1 blocker) was approved by the FDA in 1990 and later discontinued (not for safety or efficacy reasons)
• Clinical Effect: Nyxol® can reduce pupil size and reverse mydriasis by counteracting the drugs (alpha agonists and cholinergic blockers) used to dilate the pupil
• Convenient: Eye drop given at the office allows your vision to return to normal sooner
7
>80M Dilated eye
exams per year
>4MOphthalmic
surgical dilations per year
Reversal of Mydriasis
US WW
Total Patients 84M 200M
Sales Potential ~$50-100+M ~$50-100+M
Assume Private Cash Pay (One-Time)
US $400-800M Market Size
MIRA-1 Trial: Nyxol® Phase 2b in RMRandomized, Double-Masked, Placebo-Control Crossover Trial in 2019
Randomization (1:1)
• 1% • placebo
Mydriatic Agent A(2.5% Phenylephrine)
n=16
Mydriatic Agent B(1% Tropicamide)
n=16
1 drop after
1 hour
1 drop after
1 hour
• 18 years and older (average age 28, 40% male / 60% female)• Otherwise healthy subjects with no pre-existing ocular
conditions/procedures• Protocol based on Rev-Eyes
Inclusion/Exclusion Criteria
• Primary: Mean change in pupil diameter from mydriatic max in different timepoints (30min, 1h, 2h, 4h, 6h)
• Secondary:• Return to Baseline Accommodation• Safety and tolerability (redness)
Endpoints
1%
placebo
Treatment
Mydriatic Agent B(1% Tropicamide)
n=16
Mydriatic Agent A(2.5% Phenylephrine)
n=16
1 drop after
1 hour
1 drop after
1 hour
Treatment
Day 1 Day 8
Eligibility Screening
4 US sites
32 subjects
Clinical Sites
MIRA-1 (NCT04024891)
Montaquila – West Bay Eye Associates – RIKarpecki – Kentucky Eye Institute – KY
32/32 Patients Enrolled
(8/12/19-9/17/19)
Foster – Athens Eye Care – OHKannarr – Kannarr Eye Care – KS 8
Positive Data Readout
November 2019
Nyxol® Demonstrated Clinical Effects in RMObservations from MIRA-1 Phase 2b Trial
9
Efficacy
✓
Primary Endpoint Successfully Met
Significant difference between Nyxol® compared to placebo in the change from max pupil diameter (PD) at 2 hours post-treatment (p<0.0001; ranked ANCOVA)
✓
✓
Key Secondary Endpoints Met
Nyxol® significantly returns more subjects to their PD baseline by 2 (p=0.016) and 4 (p<0.0001) hours
Nyxol® returned more subjects to their baseline vision (accommodation) at 2 hours as compared to placebo (p=0.008) after dilation with tropicamide
Safety
✓Well-tolerated with mild-moderate redness in the first few hours; no LUMIFY® needed
✓ No burning, no other AEs, no SAEs
✓Works with both parasympatholytic (tropicamide) and adrenergic (phenylephrine) mydriatic agents
Phase 2b MIRA-1 Results Accepted for Presentation at ARVO 2020 Conference“Phentolamine ophthalmic solution reduces time to recovery of medically-induced mydriasis in a Phase 2 trial”
Nyxol® Comparison to Discontinued Rev-EyesA Potential Tolerable, Efficacious and Convenient Drop for Patients & Physicians
Rev-Eyes Product Label → Nyxol Potential LabelRev-Eyes is indicated in the treatment of iatrogenically induced mydriasis produced by adrenergic(phenylephrine) or parasympatholytic (tropicamide) agents. Rev-Eyes is not indicated for thereduction of intraocular pressure or in the treatment of open angle glaucoma.
Nyxol® Drug Candidate
Rev-Eyes
Burning / Stinging NONE SIGNIFICANT
Eye Redness Mild-Moderate Severe
Stable Eyedrop Formulation
YES NO (mixed at site)
Cost Effective YES NO
Efficacy: Pupil Diameter Change / Return to Baseline
YES @ 2 hrs YES @ 1-2 hrs
Vision Performance: Return to Accommodation
YES @ 2 hrs(tropicamide)
YES @ 2 hrs(tropicamide)
Dosing 1-2 Drops 4 Drops
MIRA-1 Trial FDA Registration Trials*
*Rev-Eyes FDA Registration Trials include D2-2 (n=40) and D2-3 (n=40); additional safety trials include D1-1 (n=32), D6-1 (n=38), D7-1 (n=31), and over 400 subjects across 6 open studies (D3-1 to D3-6) with 24 hour safety. Rev-Eyes was taken off market by the sponsor, not the FDA, for reasons unrelated to the unmet need.
• Rev-Eyes demonstrated safe and rapid reversal of mydriasis produced by phenylephrine and to a lessor degree tropicamide.
• Note: Eye color affects the rate of pupillary constriction. In individuals with brown irides, the rate of pupillary constriction may be slightly slower than in individuals with blue or green irides.
10
Prestigious Ocular Medical Advisory BoardFortunate for the Insights of Leading KOLs and Drug Candidate Co-Founders
Richard Lindstrom MDUniversity of Minnesota
Ed Holland MDLoyola University Chicago
Jay Pepose MDUCLA
Jack Holladay MDUniversity of Texas
Thomas Samuelson MDUniversity of Minnesota
Paul Karpecki ODIndiana University
elCON Medical
Eliot Lazar MDGeorgetown University
Gary Novak PhDUC Davis
Marguerite McDonald MDColumbia University
David Boyer MDChicago Medical School
Gerald Horn MDUniversity of IllinoisCo-Founder Ocularis/Nyxol®
Mark Kelley PhDIndiana University SimonCancer CenterCo-Founder Apexian/APX3330
11
Ocuphire Pipeline and Upcoming MilestonesMultiple Phase 3 & Phase 2 Clinical Data Catalysts Expected in Next 9-18 Months
Product Candidate Indication
Development Stage
Anticipated MilestonesPre-clinical Phase 1 Phase 2 Phase 3
1% Nyxol®Eye Drop
Night Vision Disturbance (NVD)
Initiate Phase 3 LYNX-1 trial 1H2020;Data expected in 12 months (n=200)
1% Nyxol®Eye Drop
Reversal of Mydriasis (RM)
Initiate Phase 3 MIRA-2 trial 1H2020; Data expected in 9 months (n=60-100)
1% Nyxol® + Low-Dose Pilocarpine Eye Drops
Presbyopia (P)Initiate Phase 2 VEGA-1 trial 1H2020;Data expected in 15 months (n=100)
APX3330 Oral Pill
Diabetic Retinopathy (DR)/ Macular Edema (DME)
Initiate Phase 2 ZETA-1 trial 2020;Data expected in 18 months (n=60-100)
APX2009 Intravitreal
DR/DME Initiate IND enabling studies
Combo (1% Nyxol® + Latanaprost) Eyedrops
Glaucoma (14 to 22 mmHG)
Evaluate 2nd line open-angle / normal-tension glaucoma Phase 2 trial with partner
ORION-1 4Q19
ORION-1 4Q19
ORION-1 4Q19
MIRA-1 4Q19
APX-CLN-0011 2Q19
MIRA-2
VEGA-1
ZETA-1
LYNX-1
12 This presentation contains “forward-looking” statements that are based on our management’s beliefs and assumptions and on information currently available to management.
2 Phase 3 and 2 Phase 2 trial readouts over next 9 to 18 months
Global partnering opportunities for APX2009
and for Nyxol® late-stage development /
commercialization
Thank You
Restore Vision & Clarity
Contact us at msooch@ocuphire.comor visit www.ocuphire.com