corporate presentation rashes, hair loss, oral ulcers, anxiety & depression! the severity of...
TRANSCRIPT
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HQ: Victoria, British Columbia, Canada
NASDAQ: AUPHTSX. AUP
Founded by former members of the Aspreva Pharmaceuticals team
Targeting patient populations for which there is a high unmet medical need
Highly experienced team with 35 full-time employees
Patient-focused late clinical stage biopharmaceutical company
Company Highlights
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Seasoned management
team which led the development of Cellcept®, the standard of care in the treatment
of lupus nephritis (LN)
Highly differentiated,
next-generation CNI with strong IP and potential
to be used in multiple
indications
Lead Program is in Phase III for
treatment of LN with significant
Phase II data
Additional indications
entering Phase II
STRONG CASH POSITION~182M as of September 30, 2017
Clinical-stage biopharma
company focused on the global
nephrology and autoimmunity
markets
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Our mission is to develop & deliver treatments to people living with debilitating diseases
Product Indication Development Stage
Phase I Phase 2 Phase 3 Market
Voclosporin
Lupus Nephritis(LN)
FSGS & MCD(Nephrotic Syndrome)
VOS(ophthalmic solution)
Dry Eye Syndrome (DES)
SLE & LN Overview & Symptomatology
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1. Lupus Foundation of America website: http://www.lupus.org/about/statistics-on-lupus
2. NIDDK, Lupus Nephritis. https://www.niddk.nih.gov/health-information/health-topics/kidney-disease/lupus-nephritis/Pages/index.aspx. Accessed July 26, 2016.
3. Maroz N, Segal MS. Am J Med Sci. 2013;346(4):319-23. 4. Lupus Foundation of America, http://www.lupus.org/resources/15-questions-
kidney-issues-and-lupus1. Accessed July 26, 2016.
CENTRAL NERVOUS SYSTEM
Headaches, dizziness, memory disturbances,
vision problems, seizures, stroke,
or changes in behavior
LUNGS
Pleuritis, inflammation, or pneumonia
BLOOD
Anemia, decreased white cells, increased
risk of blood clots
HEART
Chest pains, heart murmurs
KIDNEYS
Inflammation
SLE is a chronic, complex and often disabling autoimmune disorder
Affects over 500K people in the US (mostly women)1
Highly heterogeneous, affecting range of organ &
tissue systems1
LN is an inflammation of the kidneys caused by SLE & represents a serious progression of SLE
Up to 60% of SLE patients develop LN2
Straightforward disease outcomes—early response correlates w/long term outcomes; measured by proteinuria2
Debilitating and costly, often leading to ESRD, dialysis, renal transplant, and death2
Severe LN progresses to ESRD within 15 years of diagnosis in 10% to 30% of patients3
Leakage of blood proteins into the urine (proteinuria) is clinical sign of LN4
NO FDA OR EMA APPROVED LN THERAPIES
Widespread
fatigue, fever, joint pain, muscle aches,
photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression
!
The Severity of Lupus Nephritis
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Mok et al, Arthritis Rheum 2013
Standardized
mortality ratio
SLE patients w/renal damage and ESRD have 14-fold and >60-fold increased risk, of premature death, respectively
Cost Burden of Lupus Nephritis
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$0 $10,000 $20,000 $30,000 $40,000 $50,000 $60,000 $70,000
Asthma
Hypertension
Diabetes
COPD
Bipolar Disorder
Heart Disease
Rheumatoid Arthritis
Ulcerative Colitis
Crohns Disease
SLE (no nephritis)
Lupus Nephritis
Average Annual Cost of Illness per Patient by Condition*
Medical Costs Absence Costs Short Term Disability
* Carls G, et al. J Occup Environ Med. 2009;51:66-79.
Getting Disease into Remission Quickly Is Key
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INDUCTION
MAINTENANCE
FLARE REMISSION
IVC or MMFwith high
dose steroids
MMF or AZA
steroidtaper
steroidtaper
*Chen et al. Clin J. Am Soc Neph. 2008: Response = 50% reduction in proteinuria Remission = Proteinuria <.33g/24hrs
Destructive Cycle of LN Outcomes Based on Response*
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
100%
Remission Responder Non-Responder
92%
43%
13%
8%
57%
87%
Not on Dialysis @ 10 years On Dialysis at 10 years
Dollars Can’t Capture the Patient Experience
Recent patient feedback from Lupus Patient Focused Drug Development meeting with FDA confirms desire for better QOL and less steroids
SteroidBurden
Cardiovascular Issues
Osteoporosis and Fractures
Emotional Issues
InfectionsGlaucoma and Cataracts
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http://www.lupuspfdd.org
Control of Active Disease
Rapid Disease Control
Lower Steroid Burden
Convenient Treatment Regimen
Voclosporin—Potential to Address LN Critical Need
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LN Critical Need Voclosporin(based on AURA-LV Phase 2b 48-week results)
Voclosporin Overview
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LN, lupus nephritis; CNI, calcineurin inhibitor; MMF, mycophenolate mofetil; PK/PD, pharmacokinetics/pharmacodynamics.
Calcineurin inhibitors (CNIs) have demonstrated efficacy for a number of
conditions, including transplant patients, lupus nephritis (LN) patients,
keratoconjunctivitis sicca (dry eye) and other autoimmune diseases; however side effects
exist which can limit their long-term use
Predictable concentration effect and tight PK/PD relationship—no therapeutic drug monitoring1,3
Better glucose profile versus tacrolimus2
Increased potency & improved lipid profile vs CsA1
Voclosporin (VCS) is a novel CNI that may offer a number of advantages over the legacy CNI options (cyclosporine A
{CsA} and tacrolimus)
1. Aurinia Data on file2. Busque S, et al. Am J Transplant. 2011;11(12):2675-2684 & AURA LV Data
3. AURA-LV Data on file
Inhibition of calcineurin reduced cytokine activation
Potential disease-modifying podocyte stabilization, which protects against proteinuria
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The Activity of VCS in Renal Disease Involves 2 Separate Mechanisms
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voclosporinActin
cytoskeleton
Dephosphorylated synaptopodin breaks up and destabilizes the actin cytoskeleton of the podocyte
Glomerular basement membrane
CytoplasmT cell
receptor
APC
Nucleus
IL-2INF-gammaTNF-alpha
Cell-mediatedimmuneresponse
voclosporin
Tissuedamage
Calcineurin
LN, lupus nephritis; NFAT, nuclear factor of activated T cells; APC, antigen-presenting cell; IL, interleukin; INF, interferon; TNF, tumor necrosis factor.
Voclosporin LN Clinical Program
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AURION(Proof of Concept)
AURA-LV (Phase IIb)
• UPCR at week 8 is highly predictive of renal response at 24 & 48 weeks
• Renal function remains stable and inflammatory markers continue to normalize
• Statistically significant higher CR, PR, time to CR/PR, UPCR reduction & SLEDAI at 24 & 48 weeks; Achieved highest remission rates of any global LN study at 48 weeks (49.4%, p<.001)
• AE, SAEs & overall trial mortality consistent with other global LN trials
Drug-Drug Interaction (DDI) Study (Phase 1)
AURORA(Phase III)
• Healthy volunteers (US or Canada)
• 2 weeks of treatment (14 days MMF, 7 days VCS)
• Assessments: Bloodwork, PK sampling, and other assessments at various timepoints
• Double-blind placebo controlled study to demonstrate that VCS added to SoC can increase overall renal response rates in the presence of extremely low steroids;
• 2-year Extension StudyPrimary endpoint: Renal response (or CR) at 52 weeks
AURA-LV Study Design: Phase IIb
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VOCLOSPORIN 23.7 mg bid VOCLOSPORIN 23.7 mg bid
MMF 2 g + oral corticosteroids
VOCLOSPORIN 39.5 mg bid VOCLOSPORIN 39.5 mg bid
MMF 2 g + oral corticosteroids
PLACEBO PLACEBO
MMF 2 g + oral corticosteroids
Secondary endpoint
48 weeks
Primary endpoint
24 weeks
1:1
Ra
nd
om
iza
tio
n
N=
26
5
20-25 mg/daily
15-20 mg/daily
10-15 mg/daily
5 mg/daily
2.5 mg/daily
Week 2 4 6 16 24 528
Rapid steroid taper
Study was designed to evaluate whether voclosporin added to SoC can increase speed of and overall remission rates in the presence of extremely low steroidsPrimary endpoint: Complete Remission (or renal response) at 24 weeks
AURA-LV Key Inclusion Criteria & Outcome Measures
Indicative of highly active disease
KEY INCLUSION CRITERIA
Diagnosis of SLE according to ACR criteria
Biopsy proven LN [Class III, IV or Class V (alone or in combination w/Class III or
IV)]
Proteinuria of ≥1.5 mg/mgOR ≥2 mg/mg*
PRIMARY OUTCOME MEASURES
CR is defined as: Urinary protein/creatinine ratio of ≤0.5 mg/mg
Normal, stable renal function (≥60 mL/min/1.73m2 or no confirmed decrease from baseline in eGFR of ≥20%)
The proportion of subjects achieving complete remission (CR) at 24 weeks
KEY SECONDARY OUTCOMES
CR at 48 weeks, Partial Remission, Time to Remission, Time to Partial Remission, Durability of remission, and SLEDAI at 24 & 48 weeks
+
*≥2 mg/mg refers to Class V patients
Presence of sustained, low dose steroids (≤10mg prednisone from Week 16-24)
No administration of rescue medications
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AURA-LV: Complete Remission (CR) Rates at 24 & 48 Weeks
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0%
10%
20%
30%
40%
50%
60%
Baseline 24 weeks 48 weeks
Pat
ien
ts a
chie
vin
g C
R
Progression to Complete Remission
Control Low Dose
p=.045
p<.001
Improving delta over time
23.7mg BID
Control
24 week CR 32.6% 19.3%
Odds Ratio (OR);p-value
2.03;p=.045
48 week CR 49.4% 23.9%
Odds Ratio (OR); p-value
3.21; p<.001
*23.7mg is Phase III dose, so 39.5mg not shown here
23.7mg BID* VCS demonstrates statistically significant CR rates at 24 & 48 weeks
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VCS 23.7mg BID* demonstrates statistically significant complete and partial remission (renal response) rates at 24 & 48 weeks
AURA-LV: Improved Remission Over Time with Voclosporin
*23.7mg is Phase III dose, so 39.5mg not shown here
19%24%
33% 49%
30%24%
37%
20%
0%
20%
40%
60%
80%
24 48 24 48
PR= 49% PR=48%
PR=70% PR=69%
PR=pPR + CR
p<.001
Week
Control
pPR CR
pPR=PR - CR27.3mg BID VCS
pPR CR
100% subjects in CR at 24 weeks remain in CR at 48 weeks
AURA-LV: Pre-specified Biomarker Analyses: UPCR (mg/mg) and Anti-dsDNA (Mean) Over Time
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0
2
4
6
8
10
Baseline Week 2 Week 4 Week 6 Week 8 Week 12 Week 16 Week 20 Week 24 Week 48 Week 50
UP
CR
(m
g/m
g)
Visit
UPCR (Mean) Over Time
p<.001
1.75
51.75
101.75
151.75
Baseline Week 2 Week 4 Week 6 Week 8 Week 12 Week 16 Week 20 Week 24 Week 48
dsD
NA
An
tib
od
y (I
U/m
L)
Visit
Anti-dsDNA (Mean ± SD) Over Time
p=.006
VCS 23.7mg BID demonstrates statistically significant reduction in UPCR & Anti-dsDNA vs patients in the control group; UPCR also remains stable after tx stopped
12.9
8.8
-4.5
7.8
-5.3
12.7
6.2
-6.3
4.7
-7.9-10
-5
0
5
10
15
Mean Change from Baseline to Week 24
Week 24 Week 48
Mean Change from Baseline to Week 48
AURA-LV: Pre-specified Analysis:SELENA-SLEDAI Score 24 & 48 weeks
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p=.003
p<.001
Baseline
Control Voclosporin23.7mg BID
VCS showed statistically significant reduction of SLEDAI score vs. patients in the control group
AURA-LV: Renal Function: eGFR (mL/min/1.73m²) over time
20
0
20
40
60
80
100
Baseline Week 2 Week 4 Week 6 Week 8 Week 12 Week 16 Week 20 Week 24 Week 26 Week 36 Week 48 Week 50
eGFR
(m
L/m
in/1
.73
m²)
Visit
eGFR (Mean) Over Time
Treatment Complete at week 48
Note: Per DSMB, this chart shows raw eGFR values corrected so that values >90 mL/min/1.73 m2 were rounded to 90 mL/min/1.73 m2 (i.e., corrected eGFR). Source: Figure 14.2.6PH.
Renal function remains stable throughout treatment period; eGFR returns to baseline after 48 weeks
AURA-LV: Blood Pressure (BP) over 48 weeks
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80
90
100
110
120
130
140
Baseline Day 1 Week 2 Week 4 Week 6 Week 8 Week 12 Week 16 Week 20 Week 24
Syst
olic
Blo
od
Pre
ssu
re (
mm
Hg)
Visit
Systolic BP (Mean) Over Time
Week 48
50
55
60
65
70
75
80
85
90
95
Baseline Day 1 Week 2 Week 4 Week 6 Week 8 Week 12 Week 16 Week 20 Week 24 Week 48
Dia
sto
lic B
loo
d P
ress
ure
(m
mH
g)
Visit
Diastolic BP (Mean) Over Time
No significant difference in blood pressure over the 48-week treatment period
AURA-LV: Summary of Adverse Events & Historical Comparison
22
2.Furie R. et al., Arthritis and Rheumatology, Vol. 66, No 2, February 20143. Appel GB, et al. J Am Soc Nephrol. 2009;20(5):1103-1112 – Aspreva Lupus Management Study (Induction)4.Mysler, E. et al., Arthritis and Rheumatism, Vol. 65, No 9, September 2013, 2368-23795. AURA-LV Study results – Aurinia data on file
Adverse Event (AE) Summary ControlN = 88n (%)
VCS 23.7 mg BID N = 89n (%)
Any AE1 78 (88.6) 82 (92.1)
Any Serious AE (SAE)1 17 (19.3) 25 (28.1)
Any AE with Outcome of death1 4 (4.5) 10 (11.2)
Any Treatment-Related AE w/ Outcome of death 0 (0.0) 0 (0.0)
Any Treatment-Related AE 15 (17.0) 45 (50.6)
Any Serious Treatment-Related AE 1 (1.1) 4 (4.5)
Any AE Leading to Study Drug Discontinuation 9 (10.2) 16 (18.0)
Any AE Leading to Study Drug Discontinuation (excluding deaths)2 8 (9.2) 11 (12.4)
AURA-LV5
N=265 n (%)
(48 weeks)
ALMS Induction3
N=370n(%)
(24 weeks)
Abatacept Study2
N = 298n(%)
(52 weeks)
Ocrelizumab Study4
N=378n(%)
(48 weeks)
SAE’s, Subjects, n (%) 61 (23.0) 92 (25.3) 92 (30.9) 107 (28.3)
Serious Infections, Subjects n (%) 30 (11.3) 40 (10.9) 38 (19.5) 64 (16.9)
Deaths, Subjects, n (%) 13 (4.9)1 14 (3.8) 14 (4.7) 14 (3.7)
Note: data shown is for treatment-emergent adverse events (i.e., AEs post randomization).1. Data includes three placebo-randomized subjects that died post-study completion.2. For Any AE leading to Study Drug Discontinuation (excluding deaths) denominators are N=87 for control arm and N=79 for VCS 23.7 mg BID arm.
1. Data during study treatment period only; does not include the three placebo-randomized subjects that died post-study completion.
AURA-LV: Results Summary
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EFFICACY
Voclosporin 23.7mg BID (vs control) demonstrated a statistically significant:
➢ Higher CR vs. at Weeks 24 (p=.045) and 48 (p<.001)
➢ Higher PR (50% reduction in UPCR over baseline) at Weeks 24 (p=.007) and 48 (p=.007)
➢ Faster time to CR (UPCR ≤ 0.5mg/mg) (p=.002)
➢ Faster time to PR (p=.001)
➢ Reduction in UPCR at Weeks 24 (p<.01) and 48 (p<.001)
➢ Reduction in SLEDAI at Weeks 24 (p=.003) and 48 (p<.001)
First therapeutic agent to meet the PRIMARY endpoint and key 24- and 48-week pre-specified secondary endpoints in a global clinical trial for active LN
SAFETY
➢ No new or unexpected safety signals were observed with the use of VCS in LN patients; voclosporin was well-tolerated over a 48-week period.
➢ The overall safety profile was consistent with the expectations for the class of drug, the patient population, and concomitant therapies.
➢ Across indications, >2,400 patients have been treated with VCS with no new or unexpected SAEs.
AURORA Study Design: Phase III Mimics AURA Phase IIb
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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.
VOCLOSPORIN 23.7 mg bid
MMF 2 g + oral corticosteroids
PLACEBO
MMF 2 g + oral corticosteroids
Primary endpoint
52 weeks
Secondary endpoint
24 weeks
1:1
Ran
do
miz
ati
on
N=
32
4
20-25 mg/daily
15-20 mg/daily
10-15 mg/daily
5 mg/daily
2.5 mg/daily
Week 2 4 6 16 24 52
Rapid steroid taper
8
2-Y
ear
Ext
en
sio
n S
tud
y
Treatment arm
Control arm
Primary endpoint: Renal response (or CR) at 52-weeks – data expected late 2019
AURORA Key Inclusion Criteria & Primary Endpoint
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*≥2 mg/mg refers to Class V patients; ^Biopsy results over 6 months prior to screening must be reviewed with a medical monitor to confirm eligibility.
Inclusion Criteria Primary Endpoint^
Diagnosis of SLE according to ACR criteria
Renal Response at week 52Urinary protein/creatinine ratio (UPCR) of ≤0.5
mg/mg
+
Kidney biopsy within 24 months^ of study entry confirming histologic diagnosis of LN
Normal, stable renal function (≥60 mL/min/1.73m2 or no confirmed decrease from
baseline in eGFR of >20%)
+
Biopsy proven LN [Class III, IV or Class V (alone or in combination w/Class III or IV)]
Presence of sustained, low dose steroids (≤10mg prednisone from week 16-24)
+
Proteinuria of ≥1.5 mg/mgOR ≥2 mg/mg*
No administration of rescue medications
^primary endpoint is a composite
The AURA-LV Phase IIb and the AURORA Phase III study have nearly identical inclusion criteria and similar primary endpoint
AURORA Phase III Study Status*
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Number of sites initiated
62 48
76
USA / Canada APAC
EU/CIS/Africa
MAJORITYof US sites activated
26 out of 29 Countries have sites initiated
Investigator Meetings Completed
Miami, FL, USA Madrid, Spain Bangkok, Thailand Osaka, Japan
Panama City, Panama Cape Town, South Africa Belgrade, Serbia Sao Paolo, Brazil
*as of January 5, 2018
14 LATAM
LN Clinical & Regulatory Timelines
1H 2018 2H 2018 1H 2019 2H 2019 1H 2020 2H 2020 1H 2021
AURORA Recruitment
Complete
AURORA Data
Potential Approval
Projected FDA
AdCom
DDI Study
Rolling NDA Submission
Non-Clinical Section CMC Section Clinical Section
TARGET LAUNCH
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Clinical Regulatory
AURORA Extension Study
Ideal Commercial Opportunity
Positioning ProductBuilding Relationships
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Aurinia & Voclosporin
Established community
Underdeveloped market
Costly disease burdenLimited competition
Reimbursement for value
& VOCLOSPORIN
Additive vs.
Displacement
Established
Market
Costly Disease Burden
Limited Competition
Reimbursement for Value
Identifying TargetsPricing Assessment
Market Opportunity
29
Initial estimates of voclosporin peak sales potential in lupus nephritis yield global opportunity of
$1.4 billion+
A number of renal diseases are characterized by proteinuria
Reduction in proteinuria correlates with long-term outcomes for renal diseases
Legacy CNIs are effective in reducing proteinuria in renal diseases
Voclosporin may have a number of advantages over legacy CNIs
Renal Franchise Expansion
MAXIMIZE VOCLOSPORIN’S VALUE
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Nephrotic Syndrome (NS) Overview & Symptomatology
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KIDNEYS
Inflammation
NO FDA OR EMA APPROVED THERAPIES FSGS or MCD!
Acute Kidney Injury
Nephrotic syndrome is a collection of symptoms that indicate kidney damage
Proteinuria – large amounts of protein in the urine
Hypoalbuminemia – low levels of albumin in the blood
Hyperlipidemia – higher than normal fat and cholesterol
levels in the blood
Edema, or swelling, usually in the legs, feet, or ankles and
less often in the hands or face
Patients more susceptible to infection and embolism
Straightforward disease outcomes—early response correlates with long term outcomes; measured by
proteinuria
Lack of control of proteinuria results in End Stage Renal Disease, which means dialysis or kidney transplantation
~50% of NS patients have FSGS or MCD on biopsy1;
~60,000 patients in the US
1NephCure Registry 2017
Integrity of the podocyte is key feature of disease progression
1. Cattran et.al J. Am. Soc. Nephrol. 16:1061-1068, 2005
Early Clinical Response is Critical to Maintaining Long-Term Kidney Health in FSGS
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1. Cattran et.al J. Am. Soc. Nephrol. 16:1061-1068, 2005
Rapid control & reduction of proteinuria increases kidney survival1
FSGS/MCD Proof of Concept Potential Study Design
VOCLOSPORIN 23.7 mg bid
ProjectedInterim data readout(s)
6 months –Primary
Endpoint
N=~20
33
Q4 2018
KEY INCLUSION CRITERIA
Biopsy proven FSGS or MCD
Proteinuria of ≥3 mg/mg
Corticosteroid-Free
PRIMARY OUTCOME MEASURE
The proportion of subjects achieving complete or partial remission at 6 months
CR is defined as: Urinary protein/creatinine ratio of ≤0.3 mg/mg
PR is defined as 50% reduction in Urinary protein/creatinine ratio
Study is designed to evaluate the safety and efficacy of voclosporin as a first-line therapy for FSGS/MCD
Voclosporin Ophthalmic Solution (VOS): A New Product
www.webmd.com/eye-health/ss/slideshow-dry-eyes
▪ Unique aqueous, preservative free nanomicellar solution (voclosporin 0.2%)
▪ To be used in the treatment of Dry Eye Syndrome, impacting >20 million patients in the United States
▪ Additional animal safety toxicology studies planned. Studies already completed in rabbit and dog models
▪ Additional human clinical trials plannedPhase 1 study already completed
▪ IP to ~2031
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Dry Eye Syndrome (DES) –Unmet Need
DES is an Inflammatory Disease • Cure is rare or non-existent
• Control of symptoms is inadequate with currently available Rx
• Disease incidence may be growing, independent of improved diagnosis
• Aging population, increasing incidence
with age, menopause
• Growth of prostaglandin / other eye
drop Rx for glaucoma
• Increasing patient demand for better control of symptoms
• Growth of premium IOL cataract surgery, with increased insistence on high grade vision after more expensive surgery
35
VOS: Potential to Be a Low Risk, High Reward Project
36
VOS is ~4 times more potent than CsA and VOS has a 4-fold higher concentration of API
per drop than Restasis® (clear solution vs. microemulsion)
VOS achieved high concentrations in the target tissues of the eye
(Rabbit model preclinical data)
VOS has demonstrated excellent tolerability in a H2H preclinical comparison to Restasis® & in a Phase 1 study – irritation equivalent
to placebo
VOS has the potential to be dosed once daily with a rapid onset of action
(Rabbit study)
VOS has the potential to be a well differentiated and best in class CNI for the treatment of DES
Voclosporin Ophthalmic Solution (VOS) incorporates a unique & patented nanomicellar formulation system with potential clinical advantages vs. Restasis®
VOS Clinical Development Strategy
37
VOS .2%
2-4 week Primary & Secondary endpoints
KEY INCLUSION CRITERIA
A symptom score of ≥30 on a VAS (1-100)
Have a hx of DES in one or both eyes supported by a previous clinical diagnosis
OUTCOME MEASURES
Q2 2018
2H 2018
N = ~60Restasis ®.05%
An anesthetized Schirmer score of ≥5 and ≤10/5min
Evidence of ocular surface staining (fluorescein staining of at least 3 (0-15))
Secondary: OSDI, SANDE, VAS (dryness)
Primary: Ocular tolerability vs. Restasis®
Secondary: Corneal Staining, Conjunctival Staining, Schirmer test
Secondary: Adverse Events
Phase IIa – Randomized Active-Controlled Parallel-group study of the Ocular Tolerability of VOS in DES patients*
*Subject to FDA discussions
Investment Summary
38
MANAGEMENT WITH TRACK RECORD OF SUCCESS & EXTENSIVE EXPERTISE IN LN
Positive PoC and Phase IIB study results
SOLID LN CLINICAL DOSSIER
Extremely high pharmaco-economic burdenLN patients appear to be readily assessable and easily
identified by specialty treaters
LARGE AND WELL-DEFINED MARKET OPPORTUNITIES >$2B
>2,400 patients treated with voclosporin to date (across indications) well-characterized safety profile
Positive interactions with regulatory authoritiesOnly one Phase III clinical trial required by the FDA prior
to a NDA submission; Rolling NDA in preparation
STRONG CASH POSITION
~$182M as of September 30, 2017
FSGS/MCD are synergistic disease areas, representing the same call points as LN; ability to add ~60K patients
in U.S.
VOS presents unique partnering and/or divestiture opportunity
Potential FDA Adcom (LN)
Potential approval (LN)
Milestones
1H 2018 2H 2018 1H 2019 2H 2019 1H 2020 2H 2020 1H 2021
TARGET LN LAUNCH
Confirmatory DDI Study (LN)
Initiate PhIIFSGS/MCD Study
Initiate VOS Phase IIa
Submit IND for FSGS/MCD
FDA Meeting on VOS
DDI study
AURORA Recruitment
Complete
AURORA Extension Study
Begins
FSGS/MCD Ongoing Data
Readouts
VOS Phase II data
Rolling NDA (LN) Initiated:
Non-Clinical
NS data readouts
FSGS/MCD Ph II Ongoing Data
Readouts
AURORA Phase 3 Data (LN)
Rolling NDA (LN):CMC
NDA final submission (LN)
39
Thank You
#1203-4464 Markham Street • Victoria BC V8Z 7X8
www.auriniapharma.com