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Page 1: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Corporate Presentation

January 2018

Page 2: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

2

HQ: Victoria, British Columbia, Canada and Fort Washington, PA

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 30 full-time employees

Patient-focused late clinical stage biopharmaceutical company

Page 3: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Company Highlights

3

Seasoned management

team which led the development of Cellcept®, the standard of care in the treatment

of lupus nephritis (LN)

Lead Program is in Phase III for

treatment of LN with significant

Phase II data

Additional indications

entering Phase II

Ideal Commercial Opportunity with

Extensive IP

STRONG CASH POSITION~182M as of September 30, 2017

Clinical-stage biopharma

company focused on the global

nephrology and autoimmunity

markets

Page 4: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

4

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)

Page 5: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

SLE & LN Overview & Symptomatology

5

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

!

Page 6: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

The Severity of Lupus Nephritis

6

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

Page 7: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Cost Burden of Lupus Nephritis

7

$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.

Page 8: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Getting Disease into Remission Quickly Is Key

8

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

Page 9: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

9

http://www.lupuspfdd.org

Page 10: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Control of Active Disease

Rapid Disease Control

Lower Steroid Burden

Convenient Treatment Regimen

Voclosporin—Potential to Address LN Critical Need

10

LN Critical Need Voclosporin(based on AURA-LV Phase 2b 48-week results)

Page 11: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Voclosporin Introduction

11

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

Page 12: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Inhibition of calcineurin reduced cytokine activation

Potential disease-modifying podocyte stabilization, which protects against proteinuria

21

The Activity of VCS in Renal Disease Involves 2 Separate Mechanisms

12

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.

Page 13: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Voclosporin LN Clinical Program

13

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

Page 14: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURA-LV Study Design: Phase IIb

14

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

Page 15: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

15

Page 16: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURA Complete Remission (CR) Rates at 24 & 48 Weeks

16

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

23.7mg BID* VCS demonstrates statistically significant CR rates at 24 & 48 weeks

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

Page 17: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

17

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 48Week

Control

pPR CR pPR=CR-PR

27.3mg BID VCS

pPR CR

100% subjects in CR at 24 weeks remain in CR at 48 weeks

Page 18: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURA-LV: Pre-specified Biomarker Analyses: UPCR (mg/mg) and Anti-dsDNA (Mean) Over Time

18

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

A statistically significant reduction in UPCR and Anti-dsDNA vs. patients in the control group; UPCR also remains stable after treatment stopped

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

Page 19: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

19

p=.003

p<.001

Baseline

VCS showed statistically significant reduction of SLEDAI score vs. patients in the control group

Control Voclosporin23.7mg BID

Page 20: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

Page 21: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURA-LV: Blood Pressure (BP) over 48 weeks

21

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

Source: Table 14.3.3

No significant difference in blood pressure over the 48-week treatment period

Page 22: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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.

Page 23: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURA-LV: Results Summary

23

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.

Page 24: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURORA Study Design: Phase III Mimics AURA Phase IIb

24

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

Page 25: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURORA Key Inclusion Criteria & Primary Endpoint

25

*≥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.

The AURA Phase IIB and the AURORA Phase III study have nearly identical inclusion criteria and similar primary endpoint

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

Page 26: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

AURORA Phase III Study Status*

26

Number of sites initiated

57 42

68

USA / Canada APAC

EU/CIS/Africa

MAJORITYof US sites activated

25 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

12 LATAM

Page 27: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Components of NDA Submission for VCS

27

Safety Efficacy

PharmacologyPK / PD

Drug MetabolismToxicology

NON-CLINICAL(2H 2018)

CMC(1H 2019)

CLINICAL (1H 2020)

Drug Substance Manufacture Scaled Up & Validated

Drug Product Manufacture Scaled Up

Drug Product Expiry > 2 yrs

Drug Product Validation

Final Packaging Configuration

Lupus NephritisKidney Transplant

PsoriasisUveitis

AURA-LV AURION

AURORA DDI

Page 28: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

28

Clinical Regulatory

AURORA Extension Study

Page 29: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Ideal Commercial Opportunity

Positioning ProductBuilding Relationships

29

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

Page 30: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Market Opportunity

30

Initial estimates of voclosporin peak sales potential in lupus nephritis yield global opportunity of

$1.4 billion+

Page 31: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

31

Page 32: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

Nephrotic Syndrome (NS) Overview & Symptomatology

32

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

Page 33: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

33

Rapid control & reduction of proteinuria increases kidney survival1

1. Cattran et.al J. Am. Soc. Nephrol. 16:1061-1068, 2005

Page 34: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

FSGS/MCD Proof of Concept Potential Study Design

VOCLOSPORIN 23.7 mg bid

ProjectedInterim data readout(s)

6 months –Primary

Endpoint

1H 2019

N=~20

34

2H 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

Page 35: Corporate Presentation - · PDF fileCorporate Presentation January 2018. 2 HQ: Victoria, British Columbia, Canada and Fort ... Better glucose profile versus tacrolimus2 Increased potency

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

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VOS: Potential to Be a Low Risk, High Reward Project

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Voclosporin Ophthalmic Solution (VOS) incorporates a unique & patented nanomicellar formulation system with potential clinical advantages vs. Restasis®

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

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VOS Clinical Development Strategy

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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

1H 2018

2H 2018

N = ~50Restasis ®.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

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Investment Summary

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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

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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 Interim Data

Readouts

VOS Phase II data

Rolling NDA (LN) Initiated:

Non-Clinical

NS data readouts

FSGS/MCD Ph II Primary

Endpoint Data

Initiate Ph III FSGS/MCD trial

AURORA Phase 3 Data (LN)

Rolling NDA (LN):CMC

NDA final submission (LN)

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Thank You

#1203-4464 Markham Street • Victoria BC V8Z 7X8

www.auriniapharma.com

[email protected]