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2
Building the Franchise
Richard M. Glickman
Chief Executive Officer & Chairman of the Board
www.auriniapharma.com
Agenda
Topic Speaker
1 Welcome Celia Economides, VP, Corporate & Public Affairs
3 Lupus Nephritis (LN) Program Update Neil Solomons, MD, Chief Medical Officer
8 Q&A All
4
HQ: Victoria, British Columbia, Canada and Fort
Washington, PA
NASDAQ: AUPH
TSX. AUP
5
Management Team
Robert Huizinga
Executive Vice President,
Corporate Development
6
Our mission is to develop and deliver treatments to people living with
debilitating diseases.
5-Year Vision
Global Biopharma Company
Focused Renal / Autoimmune Franchise
Commercialization Capacity
Core Values
7
Corporate History of Aurinia
Late clinical
stage company
in Phase 3
IP consolidated
under one Well-capitalized
entity and
Vifor retains LN private-held
expertise creates Building
Aurinia &
Galenica Aurinia publicly held
Commercial
integrates spin-out and Isotechnika competencies
Aspreva into licenses VCS in merge to
Aspreva
Vifor Pharma; lupus & publicly-held Pipeline
Acquired by
proteinuric Aurinia expansion
Galenica for CellCept (MMF)
kidney disease
>$1B established as
from Isotechnika
new Standard of
Care for LN
8
Building Our Business: Strategic Goals
9
Lupus Nephritis Program Update
Neil Solomons, M.D.
Chief Medical Officer
www.auriniapharma.com
VoclosporinPotential to Address LN Critical Need
Of note, voclosporin is an investigational drug and is NOT currently approved as safe or effective by
any regulatory agency for any use. 11
Components of NDA Submission for VCS
NON-CLINICAL
(2H 2018)
PK / PD Pharmacology
CMC
(1H 2019)
Drug Product
Drug Substance Drug Product Validation
Manufacture Scaled Up
Manufacture Scaled Up
Drug Product Expiry > 2 yrs & Validated Final Packaging Configuration
CLINICAL
(1H 2020)
Safety Efficacy
Kidney Transplant Lupus Nephritis AURA-LV AURION
12
Voclosporin LN Clinical Program
13
AURORA Study Design: Phase 3 Mimics AURA-LV Phase 2b
52-week global, double-blind, placebo-controlled study to evaluate whether
voclosporin added to SoC can increase overall renal response rates in the presence of
low steroids.
Primary endpoint: Renal response (or CR) at 52-weeks data expected late 2019
Secondary endpoint Primary endpoint
24 weeks 52 weeks
Treatment arm
PLACEBO
Control arm
10-15 mg/daily
5 mg/daily
2.5 mg/daily
Week 2 4 6 8 16 24 52
14
AURORA Phase 3 Study Status
15
Clinical & Regulatory Timelines
AURORA
AURORA
DDI Study Recruitment
Data
Complete
Potential
Approval
TARGET
LAUNCH
16
Commercial Strategy
Bradley Dickerson
Global Commercial Lead
www.auriniapharma.com
Ideal Commercial Opportunity
Additive vs.
Established community
Displacement
Reimbursement Established
Reimbursement for value Underdeveloped market
for value market
18
US Lupus and Lupus Nephritis Population
~300,000
~200,000
20%
* Data on file from Aurinia internal market research (epidemiology papers, physician market research and medical claims review)
19
Getting Disease into Remission Quickly Is Key
100%
INDUCTION 8%
90%
80%
IVC or MMF
with high 70% 57%
dose
steroids 60%
87%
FLARE REMISSION 50%
92%
40%
MMF or
30%
AZA steroid
steroid taper 20%
43%
taper
MAINTENANCE 10%
13%
0%
Remission Responder Non-Responder
20
Cost Burden of Lupus Nephritis
Asthma
Hypertension
Diabetes
COPD
Bipolar Disorder
Heart Disease
Rheumatoid Arthritis
Ulcerative Colitis
Crohns Disease
SLE (no nephritis)
Lupus Nephritis
Cardiovascular Osteoporosis
Issues and Fractures
Steroid
Burden
Glaucoma
Infections and Cataracts
Hydroxychloroquine
Rx Prednisone
Mycophenolate
Outpatient Hospital
Facilities Physician Office
Inpatient Hospital
Rheumatology
Providers Nephrology
Acute Care Hospital
Males: 15%
Demographics Females: 85%
Claims leveraged the the MarketScan Research Databases [1] supplied by Truven Health Analytics. The databases contain cleaned,
anonymized insurance claims data from multiple major healthcare insurers.
23
Minimal Branded Competition at Launch
2017 2018 2019 2020 2021 2022 2023 2024 2025 2026+
Market Adoption
Lupus
Nephritis* Voclosporin
Aurinia
24
Current Therapies Are Expensive
Reimbursement Established
Reimbursement for value Underdeveloped market
for Value Market
26
Engaging with Key Stakeholders to Build Relationships
27
Working to Confirm Most Important Targets
Rheumatology
Physician Universe* Nephrology
~14,000
Treater (~85%)
Treaters** Non-Treater (~15%)
~11,900
80% 20%
Patient Concentration*** (40% of Treaters) (60% of Treaters)
~4,800 ~7,100
28
Gated Commercial Hiring upon Key Inflection Points
Payer Awareness
Corporate
Market Access Accounts
Distribution
Leadership Marketing
Patient Support
29
Market Opportunity
$1.4 billion +
30
IP in Lupus Nephritis
Michael Martin
Chief Operating Officer
www.auriniapharma.com
Voclosporin Has a Robust IP Portfolio
32
Hatch-Waxman Review: Drug Price Competition & Patent
Term Restoration Act Public Law 98-417
Allows protection for drug innovators while facilitating and providing
incentives for companies to file ANDAs.
*The European Union has a similar program, but the PTE is called Supplementary Protection Certificates (SPC)
33
Patent Protection of the Voclosporin Product
Composition of Matter
34
Composition of Matter Patent Coverage
2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 2031
October April
USA 2027 2028
Isomeric Patents Patent Term Extension Pediatric
Extension
(Trans Formulation) (5 years) (6 months)
TARGET
LAUNCH
35
Data Exclusivity Expectations for VCS in Ex-US Markets
2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 2031 2032
TARGET
LAUNCH
Voclosporin will be considered a New Chemical Entity (NCE) across global markets
This allows for significant Data Exclusivity across various global markets.
NCE Data Exclusivity is the strongest type of protection 3rd party challenges
are extremely rare.
36
Pursuing Avenues to Expand the VCS Patent Portfolio
AURA-LV Data
37
Composition of Matter & Data Exclusivity Provide Robust
Protection in Key Global Markets
Japan:
United States: CoM Patent 2028*^
CoM Patent 2028*^ Data Exclusivity 8
Data Exclusivity 5 years years from approval
from approval
European Union:
CoM Patent 2028*^ China+:
Data Exclusivity 10 CoM Patent 2028*^
years from approval Data Exclusivity 6
years from approval
38
Voclosporin The Next Generation
Calcineurin Inhibitor (CNI)
Robert Huizinga
Executive Vice President, Corporate Development
www.auriniapharma.com
Voclosporin Introduction
MTT, multi-targeted therapy; LN, lupus nephritis; CNI, calcineurin inhibitor; MMF, mycophenolate mofetil; PK/PD, pharmacokinetics/pharmacodynamics. 40
Calcineurin Binding Occurs Through Latch Region of VCS
voclosporin
Latch Region
voclosporin voclosporin
Actin
cytoskeleton
APC
Cytoplasm
T cell
receptor
Dephosphorylated
Calcineurin synaptopodin breaks
up and destabilizes the
Nucleus actin cytoskeleton of
IL-2 the podocyte
Cell-
INF-gamma mediated
TNF-alpha immune
response
LN, lupus nephritis; NFAT, nuclear factor of activated T cells; APC, antigen-presenting cell;
IL, interleukin; INF, interferon; TNF, tumor necrosis factor.
42
Voclosporin Key Benefits 1,2,3
VOCLOSPORIN
43
Predictable Concentration Effect: No TDM
%CNI
%CNI
%CNI
50 50 50
40
30
25 25 r = 0.8
20 r = 0.5 r = 0.7
10
0 0 0
0 10 20 30 40 50 60 0 250 500 750 1,000 1,250 1,500 0 100 200 300 400 500 600 700 800 900
Concentration Concentration Concentration
(ng/mL) (ng/mL) (ng/mL)
Poor relationship with Variable and impaired Only CNI with predictable
calcineurin activity ability to achieve max concentration-effect
Lower signal calcineurin inhibition relationship (PK/PD)
More noise Less noise
Higher signal
PK/PD, pharmacokinetics/pharmacodynamics; CNI, calcineurin inhibitor. 3. Mayo PR, et al. J Clin Pharmacol. 2013;53(8):819-826.
1. Data on file. 44
2. Ling SY, et al. J Clin Pharmacol. 2013;53(12):1303-1312.
Voclosporin Better Lipid Profile at Therapeutic Doses1
Cholesterol levels with CNI treatment Hypertriglyceridemia levels with CNI treatment
Total cholesterol (Study ISA05-25) %PASI versus % patients with high TG
Mean 95% CI
6.5 VCS 50 VCS
Drug therapy ends
CsA CsA
Mean Total Cholesterol
40
Hypertriglyceridemia
30
5.5
20
5.0
10
4.5 0
10 0 10 20 30 40 50 60 70 80 40 50 60 70 80 90 100
Week %PASI
CNI, calcineurin inhibitor; PASI, psoriasis area and severity index; CI, confidence interval; TG, triglycerides; VCS, voclosporin; CsA, cyclosporine A.
1. Kockx M, et al. 2012, ISBN: 978-953-51-0773-6, InTech, DOI: 10.5772/47866. Available from: http://www.intechopen.com/books/lipoproteins-role-in-health-and-diseases/cyclosporin-a-induced-
hyperlipidemia.
2. Data on file.
45
Across Study Comparisons of Hba1c of VCS & TAC in
Longer-Term Autoimmune Diseases
Long-Term CNI use in
Autoimmune Diseases
HbA1C > 6.5%
25
46
Functional Differences Lead to Lower Rates of NODAT
Tacrolimus voclosporin
NFATc signaling appears to play a
key role in regulating glucose and
insulin homeostasis1-4.
Cyclophilin
T cell
receptor
Tacrolimus and voclosporin
inhibit calcineurin and prevent FKBP
Calcineurin
Nucleus
different mechanisms4,5.
Active NFATc
FKBP, FK506 binding protein; NFATc, nuclear factor of activated T cells, cytoplasmic; IL, 3. Yang TTC, et al. Mol Cell Biol. 2006;26(20):7372-7387.
interleukin; IFN, interferon; TNF, tumor necrosis factor. 4. Heit JJ, et al. Nature. 2006;443(7109):345-349.
1. Auer VJ, et al. Clin Exp Immunol. 2012;170(2):238-247. 5. Busque S, et al. Am J Transplant. 2011;11(12):2675-2684.
2. Pereira MJ, et al. J Clin Endocrinol Metab. 2014;99(10):E1885-E1894.
47
Expanding the Renal Franchise
Robert Huizinga
Executive Vice President, Corporate Development
www.auriniapharma.com
Why Are We Looking Beyond LN?
49
Indication Approach:
VCS New Indications Must Fulfill These Four Criteria
Strategic Clinical
Indications with high disease Validated highly objective
morbidity and/or mortality endpoints
Limited treatment options Biologic plausibility based on
available product and disease
characteristics
Treated by specialty physicians
Small patient enrollment
Aligned with corporate focus required
Regulatory Commercial
Potential for a straightforward Clearly defined patient
regulatory pathway population
Potential for expedited Limited approved competition
regulatory review programs Specialty pricing
Strengthen IP / Data exclusivity
50
Voclosporin Differentiation in Novel Indications
The attributes demonstrated by voclosporin in prior indications (LN, Renal Tx, PsO)
studied should apply to new indications
The advantage of a CNI with an improved PK-PD relationship should allow for:
51
Potential Future Uses and Indications for VCS
(US Patient Estimates)
AURA data
Existing Level of Evidence That CNIs Are Effective
~ 150,000
Systemic Sclerosis
52
Better Diagnosis Leads to Increased Prevalence1
53
~50% of NS Patients Have FSGS or MCD on Biopsy
29%
41% FSGS
MCD
NS (Not Biopsied)
1% 13% Membranous
16% Other
54
The Opportunity in Nephrotic Syndrome
MCD and FSGS are often considered together because both are PODOCYTOPATHIES.
55
Nephrotic Syndrome: Focal Segmental
Glomerulosclerosis and Minimal
Change Disease
James A. Tumlin MD
Professor Medicine/Nephrology
Director GA Nephrology Center for
Glomerular Diseases
56
I am an investigator in the AURA, AURORA
studies
Laboratory Data:
BUN-21 Cr-2.4 Hgb 9.1
Adhesion of
Glomerular
Open-normal
Tuft to
Capillary loops
Bowmans
Capsule
Clinical Case of Severe
Nephrotic Syndrome
65
Classic FSGS
Pathologic Features
Segmental occlusion glomerular
capillaries by accumulation of
extra cellular matrix proteins
Hyalinosis: accumulation of
eosinophilic, trichrome-red
staining amorphous material
beneath basement membrane
70
Podocyte Injury: Multiple Pathways Contributing
to Cell Injury & Apoptotic Cell Death
Reduced
TGF-b Induced Hyperglycemia Production
Podocyte Podocyte
Apoptosis/Detachment
RAGE Receptor VEGF-A
ROS Injury
What is Consequence of
Podocyte Damage or
Depletion?
Podocyte depletion correlates
with Glomerulosclerosis
Albumin a3-Integrin
a3-Integrin a3-Integrin
a3-Integrin
Advanced
Sclerotic Total Glomerular
Lesion Collapse
80
KDIGO Treatment Recommendations
Idiopathic Focal Segmental Glomerulosclerosis
50%
33%
32%
26%
17%
12%
10%
Total
52.0%
Complete
36.0% Recurrence
32.0%
Partial
16.0% 14.2%
7.1% 7.1%
84
Long Term Cyclosporine Therapy for Steroid
Resistant Nephrotic Syndrome: Histologic Changes
CsA Toxicity: 17% at 14 months
FSGS
100% IgM
93%
Complete
85%
MCGN
Time to 66%
Response
58 Days
Partial
13%
91
DIRECT Trial: New Onset Diabetes or Glucose
Intolerance Following Renal Transplant1
New Onset Post-Transplant Diabetes: CsA-26.0%
P=0.046
New Onset Post-Transplant Diabetes: TAC-33.6%
PO4
PO4
Synaptopodin Synaptopodin
Intact Glomeruli
Cultured Podocytes
Low Power
Islet Transplant
High Power
Islet Transplant
1. Auer VJ, et al. Clin Exp Immunol. 2012;170(2):238-247. 5. Busque S, et al. Am J Transplant. 2011;11(12):2675-2684.
2. Pereira MJ, et al. J Clin Endocrinol Metab. 2014 Jul 8. [Epub ahead of print]
3. Yang TTC, et al. Mol Cell Biol. 2006;26(20):7372-7387..
4. Heit JJ, et al. Nature. 2006;443:345-349.
Blocking Podocyte
Detachment and Apotosis
Calcineurin: Role Apoptosis
101
Voclosporin: Benefits
Increased potency vs. cyclosporine A,
allowing lower dosing requirements 1
103
Tacrolimus Induced Inhibition of Collecting Duct
NaK-ATPase Activity: Contribution to Clinical Hyperkalemia
20
N a-K -A TP A cti vi ty (pmol es/ mm/ mi n)
NaCl
Cotransporter
Na+ Cl-
10
NaKATPase
0 Reduced K+
0.5 1.5 3.0 6.0 Excretion
Tacroli mus Concentrati on (ng/ml)
Tumlin et.al. Presented EDTA Meeting Madrid Spain, June 2017 105
Renal Function Following Prolonged (48
wk) Course of Voclosporin
eGFR remains stable over time
Tumlin et.al. Presented EDTA Meeting Madrid Spain, June 2017 106
Summary
Nephrotic Syndrome remains an important unmet need
107
107
Clinical Strategy for Nephrotic
Syndrome
Robert Huizinga
Executive Vice President, Corporate Development
www.auriniapharma.com
VCS Acts Rapidly in Proteinuric Kidney Disease (Based
On LN Data)
5
UPCR (mg/mg)
3 Placebo
2
1
VCS p<0.001
0
Baseline Week 2 Week 4 Week 6 Week 8 Week 12 Week 16 Week 20 Week 24
Visit
Source: Table 14.2.19
109
FSGS/MCD Clinical Strategy Timeline
Execute a Proof of Concept (PoC) study while conducting Regulatory interactions and
preparing for a global Phase 3 study.
Initiate Phase
3 Program
110
FSGS/MCD Proof of Concept Study Design
6 months
Interim data Interim data Interim data
Primary
readout readout readout
Endpoint
111
Indication Approach:
FSGS & MCD Meet Our Strategic Criteria
Strategic Clinical
Indications with high disease Validated highly objective
morbidity and/or mortality endpoints
Limited treatment options Biologic plausibility based on
available product and disease
characteristics
Treated by specialty physicians
Small patient enrollment
Aligned with corporate focus required
Regulatory Commercial
Potential for a straightforward Clearly defined patient
regulatory pathway population
Potential for expedited Limited approved competition
regulatory review programs Specialty pricing
Strengthen IP/Data exclusivity
112
Minimal Branded Competition at Projected Launch
2017 2018 2019 2020 2021 2022 2023 2024 2025 2026+
Lupus Voclosporin
Aurinia
Nephritis*
Nephrotic Voclosporin
Aurinia
Syndrome*
Sparsentan Bleselumab
Retrophin Astellas
113
The Opportunity in Expanding the Franchise
114
New Product Development
Voclosporin Ophthalmic Solution (VOS)
www.auriniapharma.com
Voclosporin Ophthalmic Solution (VOS): A New Product
5. IP to ~2031
1. www.webmd.com/eye-health/ss/slideshow-dry-eyes
116
Dry Eye Syndrome
I have no financial interest in any of the products discussed. I have been paid to be an
investigator in clinical trials, member of Advisory Boards and have received honoraria for
lecturing as part of the Speakers Bureau for:
118
Harvard Medical School 1982
Past Clinical Professor at University of Missouri / Kansas City, and University of Kansas
Principal Investigator in over 100 multicenter clinical trials, wrote Restasis protocols, lead PI on Xiidra
119
The eye surface requires lubrication to
adequately perform its primary functions
of:
1. Barrier protection
2. Visual function
120
TFOS DEWS II Revised Definition
121
What Does a Dry Eye Patient Look Like?
122
Prevalence of Dry Eye
Involved in 40% of Eye Care Visits
Women:
4% @ 40 15% @ 65
Men:
4% @ 50 7.7% @ 80+
Worldwide distribution:
US 5.8%
Market Scope. 2010 Comprehensive Report on The Global Dry Eye Products Market.
Western Europe 8%
China 21%
India 12%
123
The Dry Eye Market
124
Economics of Dry Eye (Ophthalmic Practice)
125
Patient Cost of Treating
Moderate to Severe Dry Eye Symptoms
126
Dry Eye Is an Inflammatory Disease
CD4+ T Cell Infiltration
MHC Class II Upregulation
ICAM-1 Upregulation
CHRONIC Antigen
INFLAMMATION Presentation
T Cell Homing to the
Ocular Surface
T Cell Activation
InDoctor Speak
Supplementation Stimulation Retention
In Patients Terms
Add Tears Stimulate Block tears
More Tears from leaving
128
Cyclosporine for Dry Eye
Cytokines
Disrupt Neural Arc
129
Blocks ICAM-1/LFA-1 Interaction and Inhibits T Cell
Recruitment, Activation, and Cytokine Release
130
LFA-1 Mediates T Cell Functions
Cytokine Cytokine
Induced Release
Inflammation
131
Unmet Needs in Dry Eye
132
Clinician Concerns with Currently Available
Pharmaceuticals for Dry Eye
Restasis Xiidra
15% intolerance 15% intolerance, sometimes severe
High cost High cost, espec. Medicare Part D
Onset 2-4 months Onset 2 8 weeks
Rare WOW response 20% WOW response
Lack of clinical trial evidence for 5-10% blurred vision, 5% dysgeusia
symptoms, stain Weak clinical trial evidence for
Multidose bottle = $$$$ signs
Vials contain 3-5 gtt each
133
Dry Eye What Measures Improvement?
SYMPTOMS
134
Dry Eye Therapeutics Regulatory Issues
135
Clinical Trial Metrics - Symptoms
CATEGORICAL SCALES 0 - 3
0 100
0 50 100
136
Clinical Trial Metrics Signs
Grading Schemes for Staining
137
Measurement of Tear Production - Schirmers
Test
138
Dry Eye Development Many Failures, Some
Success
SYMPTOMS SIGNS
139
OCULAR SURFACE DISEASE Rx ALGORITHM
140
Why are Calcineurin Inhibitors Valuable in Dry
Eye Treatment?
141
What ocular clinical indications would merit
investigation for an compound that was shown to be
an effective inhibitor of T cell activation?
Dry Eye
142
Closing Thoughts
143
Introduction to Voclosporin
Ophthalmic Solution (VOS)
Michael Martin
Chief Operating Officer
www.auriniapharma.com
Risk Mitigated Approach
IMS data & market research suggests that topical CNI will remain a mainstay of DES
treatment (as monotherapy or polytherapy).
145
Restasis (CsA) Was 1st Rx Approved for DES in the US
Unmet Need Remains High
UNMET NEED: RESTASIS Sales (IMS) 2012-2016
Label: 2000
Indication for treatment of a sign only: increased
1800
tear production
Onset of effect: 1600
Millions
Increased tear production observed after 3- 6 1400
months of treatment
1200
Efficacy:
1000
{responder analysis}: 15%-20% of patients in
clinical trial setting, 5% placebo (label) 800
Market Research showed slightly higher perceived 600
clinical efficacy in practice (~25%)
400
Adverse effects:
17% of patients experience ocular burning (per 200
label) 0
Dosing: BID 2012 2013 2014 2015 2016
Treatment success:
High drop-out rates 277 million units in the United States for
Market Research showed 70% drop-out by end of the Restasis in 2016 (MAT)
1st year
Sales growth was ~19% over 2015 despite
Xiidra launch in July of 2016 (MAT)
146
Perceived Significant Unmet Medical Need Remains for
the Treatment of DES Despite Approved Products*
PRODUCT ATTRIBUTE
Efficacy for all DES agents is poor, although
there are no H2H comparisons, only about
Efficacy 20-30% of patients respond to therapy
Restasis Xiidra
*based on Aurinia market research; H2H comparisons have not been completed
147
CNI Inhibition Is a Validated Mechanism for the Treatment
of Ocular Surface Diseases
Cyclosporin A .05% in the US (DES)/Allergan
Calcineurin is a
Cyclosporin A .1% in Japan (Spring Catarhh)
validated target for
Cyclosporin A .1% in EU(DES with Keratitis)/Santen
chronic ocular
Cyclosporin A .1% in EU (V.Keratoconjunctivitis)/Santen
surface diseases. Tacrolimus .1% in Japan (Allergic Conjunctivitis)/Sanju
148
Why Voclosporin Ophthalmic
Solution (VOS)?
www.auriniapharma.com
VOS Is a Unique Formulation
150
VOS Has Robust Tox and Safety Package
Oral Voclosporin (LN Program): Voclosporin Ophthalmic Solution:
SAFETY AND TOXICOLOGY: SAFETY AND TOXICOLOGY:
Current toxicology package supporting the VOS Ophthalmic Ocular Safety and pK work
oral formulation in rabbits and dogs
EFFICACY: EFFICACY:
Current clinical package supporting the oral VOS Efficacy Studies in DES dogs (Proof
formulation multiple indications including of Principles)
LN
HUMAN Phase 1 Dose Escalation Study
VOS Efficacy Study vs. Standard of Care
in DES dogs (MAH under MTA)
15.0
Normal STT in Dogs >15mm/min
10.0
Discontinuation of
Optimmune (2% CsA)
5.0 initiation of VOS .2%
Expected STT value
without therapy
0.0
Baseline Day 7 Day 14 Day 28
152
VOS Has the Potential for Improved Dosing vs. Restasis
Ocular pK in animals supports QD dosing of VOS 0.2%.
Tissue levels after single dose in NZW rabbits over 24 hours
All tissues above therapeutic target concentration at 24 hours
VCS Concentration in
Conjunctiva
VCS Concentration in
Cornea
Therapeutic concentration
of CsA (50-300ng/g) in
Conjunctiva & Cornea*
*R.L. Kaswan., Intraocular penetration of topically applied cyclosporine, Transplantation Proceedings, vol 20, no. 2, supplement, pp. 650-655, 1988
153
VOS Tolerability in Healthy Volunteers Similar to Placebo
Phase 1 dose escalation study to assess the safety and tolerability
of VOS in health volunteers.
30 healthy patients, placebo vs. 2 doses VOS, over an 8 hour period four applications per
eye
Lemp, M.A., Beckman, R., Weiss, S., Originally published by Lux BioSciences
154
VOS Tolerability in Healthy Volunteers Similar to Placebo
Phase 1 dose escalation trial, 30 healthy patients, placebo or
2 doses VOS, over an 8 hour period four applications per eye
Lemp, M.A., Beckman, R., Weiss, S., Originally published by Lux BioSciences
155
VOS Also Has the Potential for Improved Onset of Action
Cohort 3 of Phase 1 safety and tolerability study: Promising early improvements in Signs and
Symptoms in 2 weeks.
OSDI scores were improved in all subjects at all time points while on drug
Phase 1 data (Cohort 3): 5 patients with DES dosed BID for 14 days
(Sign) (Symptom)
Schirmers I-test at Mean Change in OSDI & Symptom Subscales
Screening and Day 14
Tear Production Increase Reduction in Score from Baseline
+31% + 68% - 32% - 48% - 27% - 15%
9 90
7 70
6 60
5 50
4 40
3 30
2 20
1 10
0 0
Left Eye Right Eye OSDI Total Score Vision Related Ocular Symtom Environmental
Function Subscale Subscale Triggers Subscale
Screening Day 14 Baseline Day 14
Lemp, M.A., Beckman, R., Weiss, S.,
Originally published by Lux BioSciences 156
VOS Clinical Development Strategy
Phase 2a Randomized Active-Controlled Parallel-group study of the Ocular
Tolerability of VOS in DES patients*
2-4 week Primary &
Q2 2018 Secondary endpoints
VOS .2%
N = ~50
Restasis .05%
2H 2018
Clinical Trial
1 Supply
2 Phase 2a:
Clinical Supply Comparative
Available Tolerability H2H
vs. Restasis
Phase 2b:
CLINICAL STUDY or Asset Monetization
158
VOS: Potential to Be a Low Risk, High Reward Project
VOS has the potential to be a well differentiated and best in class CNI for the
treatment of Dry Eye Syndrome
159
Wrap-Up
Richard Glickman
Chief Executive Officer
www.auriniapharma.com
Summary
161
Milestones
AURORA TARGET LN
Initiate NS Proof NS Final Proof of
of Concept
Extension Study
Concept Data
LAUNCH
Begins NS data readouts
DDI study
Initiate VOS NS Interim Data
Phase 2a Readouts
VOS Phase 2a
data
162
Q&A Session