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Corporate Presentation November 2017

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Page 1: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Corporate Presentation

November 2017

Page 2: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Forward Looking Statements

2

Certain of the statements made in this presentation may constitute forward-looking information within the meaning of applicable Canadian securities law and forward-looking statements within the meaning of applicable U.S. securities law. These forward-looking statements or information include, but are not limited to statements or information with respect to the projected worth of the lupus nephritis (LN) market, that voclosporin is potentially a best-in-class calcineurin-inhibitor (CNI) with robust intellectual property exclusivity and the likelihood of data exclusivity in major markets, the expectation that voclosporin will be the only CNI with a label for LN, the expected progress of the AURORA study; the anticipated commercial potential of voclosporin for the treatment of LN, Nephrotic Syndrome, FSGS, Dry Eye and other Autoimmune diseases; and anticipated interactions with the US Food and Drug Administration, including potential dates for submission and approval of marketing applications, and product label . When used in these marketing materials, the words “anticipate”, “will”, “believe”, “estimate”, “expect”, “intend”, “target”, “plan”, “goals”, “objectives”, “may” and other similar words and expressions, identify forward-looking statements or information. We have made numerous assumptions about the forward-looking statements and information contained herein, including among other things, assumptions about: the market value for the LN program; that another company will not create a substantial competitive product for Aurinia’s LN business without violating Aurinia’s intellectual property rights; and the size of the LN market. Even though the management of Aurinia believes that the assumptions made and the expectations represented by such statements or information are reasonable, there can be no assurance that the forward-looking information will prove to be accurate. Forward-looking information by their nature are based on assumptions and involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements of Aurinia to be materially different from any future results, performance or achievements expressed or implied by such forward-looking information. Should one or more of these risks and uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in forward-looking statements or information. Such risks, uncertainties and other factors include, among others, the following: the market for the LN business may not be as estimated; and competitors may arise with similar products.Although we have attempted to identify factors that would cause actual actions, events or results to differ materially from those described in forward-looking statements and information, there may be other factors that cause actual results, performances, achievements or events to not be as anticipated, estimated or intended. Also many of the factors are beyond our control. There can be no assurance that forward-looking statements or information will prove to be accurate, as actual results and future events could differ materially from those anticipated in such statements. Accordingly you should not place undue reliance on forward-looking statements or information. Except as required by law, Aurinia will not update forward-looking information. All forward-looking information contained in this presentation is qualified by this cautionary statement. Additional information related to Aurinia, including a detailed list of the risks and uncertainties affecting Aurinia and its business can be found in Aurinia’s most recent Annual Information Form available by accessing the Canadian Securities Administrators’ System for Electronic Document Analysis and Retrieval (SEDAR) website at www.sedar.com or the U.S. Securities and Exchange Commission’s Electronic Document Gathering and Retrieval System (EDGAR) website at www.sec.gov/edgar.

Page 3: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

3

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 4: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Company Highlights

4

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 5: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

5

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)

Nephrotic Syndrome(FSGS & MCD)

VOS(ophthalmic solution)

Dry Eye Syndrome (DES)

Page 6: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

SLE & LN Overview & Symptomatology

6

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 7: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

The Severity of Lupus Nephritis

7

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 8: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Cost Burden of Lupus Nephritis

8

$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 9: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Getting Disease into Remission Quickly Is Key

9

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 10: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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

10

http://www.lupuspfdd.org

Page 11: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Control of Active Disease

Rapid Disease Control

Lower Steroid Burden

Convenient Treatment Regimen

Voclosporin—Potential to Address LN Critical Need

11

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

Page 12: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Voclosporin Introduction

12

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 13: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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

13

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 14: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Voclosporin LN Clinical Program

14

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 15: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

AURA-LV Study Design: Phase IIb

15

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 16: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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

16

Page 17: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

17

VCS 23.7mg BID* demonstrates statistically significant renal response rates at 24 & 48 weeks; 100% subjects in CR at 24 weeks remain in CR at 48 weeks

AURA-LV: Improved Renal Response Over Time with Voclosporin

*23.7mg is Phase III dose, so 39.5mg not shown here

Treatment* N Response24

weeksOdds ratio

(95% CI)P-

value48

weeksOdds Ratio

(95% CI)P-value

23.7mg BID VCS

89Complete Remission (CR) 33% 2.03 (1.01, 4.05) p=.045 49% 3.21 (1.68, 6.13) p<.001

Partial Remission (PR) 70% 2.33 (1.26, 4.33) p=.007 69% 2.34 (1.27, 4.33) p=.007

Control 88Complete Remission (CR) 19% NA NA 24% NA NA

Partial Remission (PR) 49% NA NA 48% NA NA

19% 24%33% 49%

27%40%

30% 24%

37%20%

39%

32%

0%

20%

40%

60%

80%

24 48 24 48 24 48Week

Control

PR CR

27.3mg BID VCS

PR CR

39.5mg BID VCS

PR CR

Page 18: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

AURA-LV: Pre-specified Biomarkker 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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

AURA-LV: Results Summary

23

EFFICACY

Voclosporin 23.7mg BID demonstrated a statistically significant:

➢ Higher CR vs. the control group 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=.002)

➢ 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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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 treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

AURORA vs. AURA Key Inclusion Criteria

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

AURA-LV (Phase IIb) AURORA (Phase III)

Diagnosis of SLE according to ACR criteria

Diagnosis of SLE according to ACR criteria

Kidney biopsy within 6 months of study entry confirming histologic diagnosis of LN

Kidney biopsy within 24 months^ of study entry confirming histologic diagnosis of LN

Biopsy proven LN [Class III, IV or Class V (alone or in combination w/Class III or IV)]

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*

Proteinuria of ≥1.5 mg/mgOR ≥2 mg/mg*

Page 26: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

AURORA vs AURA Primary Endpoint

26

The AURA Phase IIB and the AURORA Phase III study have nearly identical primary endpoints; however AURORA primary endpoint is at 52 weeks

AURA-LV (Phase IIb) AURORA (Phase III)

Complete Remission at week 24Urinary protein/creatinine ratio (UPCR) of ≤0.5

mg/mg

Renal Response at week 52Urinary protein/creatinine ratio (UPCR) of ≤0.5

mg/mg

+ +

Normal, stable renal function (≥60 mL/min/1.73m2 or no confirmed decrease from

baseline in eGFR of >20%)

Normal, stable renal function (≥60 mL/min/1.73m2 or no confirmed decrease from

baseline in eGFR of >20%)

+ +

Presence of sustained, low dose steroids (≤10mg prednisone from week 16-24)

Presence of sustained, low dose steroids (≤10mg prednisone from week 16-24)

+ +

No administration of rescue medications No administration of rescue medications

Page 27: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

AURORA Phase III Study Status

27

Number of sites initiated

45 2754 12

USA / Canada APAC

Europe LATAM

MAJORITYof US sites activated

23 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

Upcoming Investigator Meetings

Sao Paolo, Brazil

Page 28: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

Components of NDA Submission for VCS

28

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 29: Corporate Presentation treatment of LN, Nephrotic Syndrome, FSGS, ... photosensitivity, rashes, hair loss, oral ulcers, anxiety & depression! The Severity of Lupus Nephritis 7

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

29

Clinical Regulatory

AURORA Extension Study

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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

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Gated Commercial Hiring upon Key Inflection Points

Prepare ahead of inflection points to quickly onboard new resources.

FDA ApprovalPhase III Data (Additional Commercial Leadership)

Ongoing Phase III

Leadership

Market Access

Marketing

Payer Awareness

Analytics / Market Research

Commercial Operations

Sales Leadership Sales Team

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Corporate Accounts

Patient Support

Distribution

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Market Opportunity

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

$1.4 billion+

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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

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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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Rapid control & reduction of proteinuria increases kidney survival1

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

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FSGS/MCD Proof of Concept Study Design

VOCLOSPORIN 23.7 mg bid

Interim data readout

Interim data readout

Interim data readout

6 months –Primary

Endpoint

KEY INCLUSION CRITERIA

Biopsy proven FSGS or MCD

Proteinuria of ≥3 mg/mg

PRIMARY OUTCOME MEASURE

CR is defined as: Urinary protein/creatinine ratio of ≤0.5 mg/mg

The proportion of subjects achieving complete or partial remission at 6 months

PR is defined as 50% reduction in Urinary protein/creatinine ratio

Q1 2019

Corticosteroid-Free

N=~20

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2H 2018

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FSGS/MCD Clinical Strategy Timeline

• Submit IND; Initiate & Complete Proof of Concept Study

Establish Approval Pathway

Initiate Phase III Program

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1H 2018 2H 2018 Q1 2019

Execute a Proof of Concept (PoC) study while conducting Regulatory interactions and preparing for a global Phase III study

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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

Q2 2018

2H 2018N = ~50

Restasis ®.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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Clinical Supply Available

FDA MEETING

Clinical Trial Supply

IND OPEN

Phase 2a: Comparative Tolerability H2Hvs. Restasis®

Q4 2017 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Q1 2019 Q1 2019

1

2

Planned VOS Development Activities and Timelines

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Phase 2b:CLINICAL STUDY or Asset Monetization

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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 Nephrotic

Syndrome (NS)

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]