July 2016 Corporate Presentation · 2016-07-01 · July 2016 Corporate Presentation. ... Canadian...

19
July 2016 Corporate Presentation

Transcript of July 2016 Corporate Presentation · 2016-07-01 · July 2016 Corporate Presentation. ... Canadian...

Page 1: July 2016 Corporate Presentation · 2016-07-01 · July 2016 Corporate Presentation. ... Canadian Securities Administrators’System for Electronic Document Analysis and Retrieval

July 2016 Corporate Presentation

Page 2: July 2016 Corporate Presentation · 2016-07-01 · July 2016 Corporate Presentation. ... Canadian Securities Administrators’System for Electronic Document Analysis and Retrieval

Forward-Looking Statements

1

Certain of the statements made in this presentation may constitute forward-looking information within the meaning of applicable Canadiansecurities law and forward-looking statements within the meaning of applicable U.S. securities law. These forward-looking statements orinformation 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 dataexclusivity in major markets, the expectation that voclosporin will be the only CNI with a label for LN, the expected progress of the AURIONstudy; the anticipated commercial potential of voclosporin for the treatment of LN; and anticipated interactions with the US Food and DrugAdministration. 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 otherthings, assumptions about: the market value for the LN program; that another company will not create a substantial competitive product forAurinia’s LN business without violating Aurinia’s intellectual property rights; and the size of the LN market. Even though the management ofAurinia believe that the assumptions made and the expectations represented by such statements or information are reasonable, there canbe 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 otherfactors 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 anduncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described inforward-looking statements or information. Such risks, uncertainties and other factors include, among others, the following: the market forthe 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 inforward-looking statements and information, there may be other factors that cause actual results, performances, achievements or events tonot 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 anticipatein 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 thispresentation are qualified by this cautionary statement. Additional information related to Aurinia, including a detailed list of the risks anduncertainties affecting Aurinia and its business can be found in Aurinia’s most recent Annual Information Form available by accessing theCanadian 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.

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Experienced Management Team

2

Over 100 years of combined corporate life sciences industry experience

Charles A. Rowland , MBA, CPA Chief Executive Officer

Michael R. Martin, BSc Chief Operating Officer

Neil Solomons, MD Chief Medical Officer

Dennis Bourgeault, CPA, CA Chief Financial Officer

Lawrence D. Mandt, BS VP Regulatory Affairs & Quality

Robert Huizinga, RN, MSc, CNeph VP Clinical Affairs

Bradley Dickerson, BA, Management GM Americas & Global Commercial Assessment

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Clinical stage pharmaceutical company focused on the global nephrology and autoimmunity markets

Developing a novel Phase 2b lupus nephritis (LN) therapy, voclosporin, a calcineurin-inhibitor (CNI) and NCE with certain advantages over existing therapies

Lupus market is projected to be worth ~$3B* by 2019 in the US/EU

– Lead asset granted FAST TRACK designation by the FDA

Strong safety package with robust clinical rationale:

– Efficacy of multi-targeted LN therapy has been established

– Well characterized safety profile - >2,000 patient exposures – some for multiple years

– Voclosporin is potentially a best-in-class calcineurin inhibitor (CNI) with robust IP*^ exclusivity

– Supportive 8 week proof of concept data - AURION

AURA-LV global clinical Phase 2b trial in LN has completed enrolling at 265 patients with data read-out sometime in Q3/16 → AURION study is ongoing

Aurinia Investment Highlights

3

*^Includes 5 year PTE*Data Monitor

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SLE an autoimmune disorder impacting ~ .5-1.5M* people in U.S.

– Highly heterogeneous; can affect the heart, lungs, skin, joints, vasculature system and kidneys

~40-50% of SLE patients develop lupus nephritis

– LN is inflammation of the kidney caused by SLE

– If left untreated or inadequately treated, LN can lead to end stage renal disease, dialysis, renal transplant and death

No FDA or EMA approved LN therapies

Systemic Lupus Erythematosus (SLE) & Lupus Nephritis

(LN)

4

*Lupus Foundation of America (LFA) estimate

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While at Aspreva, Aurinia Management Executed the ALMS Study (Establishing CellCept® as Current SoC)

5

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Despite CellCept®, Significant Unmet Medical Need Remains:

>90% of Patients Fail To Achieve Complete Remission**

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

Remission Responder Non-Responder

92%

43%

13%

8%

57%

87%

LN patient outcome based on treatment response*

Not on Dialysis @ 10 years On Dialysis at 10 years

20%

40%

60%

80%

100%

6 months

~54%respond

<10% achieve complete remission**

*Chen et al. Clin J. Am Soc Neph. 2008: Response = 50% reduction in proteinuria Remission = Proteinuria <.33g/24hrs**Aspreva Lupus Management Study – ALMS (NEJM/JASN). Complete remission requires proteinuria of <.5g/24hrs and presence of inactive urinary sediment

Rapid control & reduction of proteinuria in lupus patients reduces the need for dialysis*

% o

f P

atie

nts

Higher remission rate reduces the

risk of ESRD*

6 NASDAQ - AUPH

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CNIs are inhibitors of calcineurin phosphatase and the combination of MMF & CNIs are standard of care (SoC) for prevention of rejection in solid organ transplants

The immunology of lupus supports treating LN patients with a similar approach to that used in transplant patients (multi-targeted therapy)

Strong rationale for voclosporin use in LN

– Potent effects on T-cell activation and immunomodulatory effects

– T-cell mediated immune response important feature of pathogenesis of many autoimmune diseases, including LN

Need for MTT with Best-in-Class Calcineurin

Inhibitor (CNI) in Lupus Nephritis - voclosporin

7

Altered functional group

on CsA

Impacts binding to the latch region on

Calcineurin

Voclosporin

N

HO

O

HN

N

OO

N

O

N

O

N O

NH

O

HN

O

N

O

HN

O

N

OMTT

Transplant

Auto-immune diseases

Antivirals

DiabetesHyper-lipidemia

Hyper-tension

Coronary heart

disease

MTT is a Common Way to Treat Complex Diseases

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*Remission rates: CellCept®

vs. IVC after 6 months – ALMS

**Remission rates: multi-target therapy vs. IVC after 6 months –

Bao et al.

Data Suggest Multi-Targeted Therapeutic (MTT) Approach To LN

Will Yield Superior Results to either MMF or IVC Alone

8

Aspreva Lupus Management Study – ALMS (NEJM/JASN). Complete remission required presence of inactive urinary sediment**Bao Hao et al. J Am Soc Nephrol 19:2001-2010, 2008. IVC – Intravenous cyclophosphamide

Rem

issio

n/R

esp

on

se r

ate

20%

40%

60%

80%

100%

8.6%

53%

8.1%

56%

Partial

Response

Complete

Response

CellCept®IVC

6 Months

20%

40%

60%

80%

100%

50%40%

5%

40%

Partial

Response

Complete

Response

Multi-TIVC

6 Months

Rem

issio

n/R

esp

on

se

rate

(“multi-target” = CellCept® +CNI)

^MTT of Lupus Nephritis: A Prospective Randomized Controlled Multi-Center

Trial – (n=362)

40%

60%

80%

100%

38%

25%

38%

Partial

Response

Complete

Response

Multi-T(n=181)

IVC(n=181)

24 Weeks(multi-target = MMF + CNI)

46%

20%25%

p< .001

Japaneseregistration study:Tacrolimus-treated

patients experienced 15% rate of diabetes/ glucose intolerance^

^^Miyasaka et al. Mod Rheumatol. (2009) 19:606-615

^Liu et al. Annals of Internal Medicine 11 November 2014 doi:10.7326/P15-9000

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AURION: Study Design

Primary analysis: Number of patients achieving each of the following biomarkers and the number of these patients who go on to achieve week 24 or week 48 remission.

Biomarkers:– 25% reduction in P/CR at 8 weeks– C3 normalization at 8 weeks– C4 normalization at 8 weeks– Anti-dsDNA normalization at 8 weeks

Secondary analyses: 24, 48 week outcomes, markers of SLE, PK/PD voclosporin in LN subjects

24 week

assessment

End of study

48 weeks Exploratory

Endpoint 8 weeks

Single Arm, Twin Centre Exploratory Study Assessing The Predictive Value Of An

Early Reduction In Proteinuria In Subjects Receiving Multi-Target Therapy With

Voclosporin 23.7 Mg BID, MMF Up To 2g Daily In Patients With Active Lupus Nephritis

AURION Data Release Ongoing at Upcoming Scientific Meetings:

- 7 patients to 24 weeks (June 2016)

- 10 patients to 24 weeks (Sept. 2016)

Voclosporin 23.7 mg bid Voclosporin 23.7 mg bid

MMF 1 - 2 g + oral corticosteroidsScreening

N = 10

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0.00

1.00

2.00

3.00

4.00

5.00

1 (01-02) 2 (01-03) 3 (01-05) 4 (01-06) 5 (02-01) 6 (02-03) 7 (02-04)

UPCr Pre-Treatment to Week 24 (n = 7)

Pre-Treatment Week 24

10

AURION: 24 Week Data – First 7 Patients

Mean proteinuria reduction at 24 weeks is 54%*^ using voclosporin as a component of MTT

57% (4/10) have achieved a Complete Remission at 24 weeks as defined as a urinary protein creatinine ratio of ≤ 0.5mg/mg, eGFR within 20% of baseline and concomitant steroid dose of less than 5mg/day.

*Indicates patient taking MMF at entry (failing MMF)

^ LOCF – subject withdrew from study at 8 weeks

^

*

-1.00

-0.50

0.00

0.50

1.00

1.50

2.00

2.50

3.00

3.50

4.00

UP

Cr

(mg/m

g)

Visit

UPCr Means and 95% Confidence Level Over Time

UP

Cr

(mg

/mg

)

*

*

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11

LN Inflammatory markers are positively impacted after 24 weeks of MTT with voclosporin

eGFR assessed through CKD-EPI formula

Renal function is stable (as measured by eGFR) throughout the treatment period

C3 & C4 Improves after 24 weeks* of therapy, while renal

function as measured by CKD-EPI remains stable

0

20

40

60

80

100

120

140

160

eG

FR

(m

L/m

in/1

.73m

2)

Visit

eGFR Means and 95% Confidence Level Over Time*

0

10

20

30

40

50

60

70

80

90

C3C

3

mg/d

l

C3 – Baseline vs. Week 24*

Baseline 24 weeks

18% increase in mean

C3*

0

5

10

15

20

25

C4

C4

mg/d

l

C4 – Baseline vs. Week 24*

Baseline 24 weeks

37% increase in mean

C4*

*LOCF for subject 7 – subject withdrew from study at 12 weeks

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One subject (subject 4) out of 7 required multiple dose interruptions of MMF and voclosporin for hypertension, anemia, community acquired pneumonia eventually maintained on lower dose of voclosporin therapy

Another (subject 7) failed to respond adequately to drugs and was withdrawn from therapy at week 12

Otherwise combination well tolerated

No unexpected safety signals

24 week Safety/tolerability utilizing voclosporin

as a component of Multitargeted Therapy (MTT)

12

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AURION – 24 week Top-Line Data Summary -

Encouraging

13

24 week AURION data appears to support the hypothesis that MTT utilizing voclosporin has the potential to improve LN disease activity

AURION efficacy data is encouraging:

– 57% (4/7) of patients in remission at 24 weeks as measured by urinary protein creatinine ratio of ≤ 0.5mg/mg, eGFR within 20% of baseline and concomitant steroid dose of less than 5mg/day

– 54% mean reduction in proteinuria (including LOCF data)

– Mean C3/C4/anti-dsDNA trending towards normalization

Safety profile is encouraging while renal function remains stable (as measured by eGFR/CKD-Epi)

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AURA (Aurinia Urinary - protein Reduction in Active nephritis) : Enrolment Complete – 24 week data available Q3/2016

14

Voclosporin 39.5 mg bid

MMF 2 g + oral corticosteroids

1:1

:1 R

and

om

izat

ion

N

=26

5

1º Endpoint 24 weeks - Complete remission (confirmed UPCr ≤ 0.5 and eGFR

within 20% baseline value)

Voclosporin 23.7 mg bid

Voclosporin 39.5 mg bid

End of study 48 weeks - Durability of remission, 48 week outcomes, extra-renal lupus activity (SLEDAI

MMF 2 g + oral corticosteroids

MMF 2 g + oral corticosteroids

Voclosporin 23.7 mg bid

Placebo Placebo

20-25mg/day

15-20mg/day

10-15mg/day

5mg/day

10mg/day

2.5mg/day

Week 2 4 6 12 24 48

AURA - Forced Steroid taper30 mg/day

40 mg/day

50 mg/day

60 mg/day

8

Study design aims to demonstrate rapid,

sustained complete remission in the

presence of extremely low steroids

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Commercial Potential of Voclosporin for the Treatment of LN Could Be Substantial

Total US SLE/Lupus Population ~.5 – 1.5M^^

~200K

~300K

Not diagnosed,

not in the

healthcare

system

SLE patient population

(non-renal)

LN patient population,

elevated proteinuria

Other products used for Lupus

(United States)*

Estimated

Price/year (in US)*

Rituxan® ~$27,000/year

CellCept® (branded 2012) ~$12,000/year

Benlysta® ~$35,000^/year*

*plus infusion costs

Competitor Pricing per LN patient per year

*Datamonitor SLE- Stakeholder insights

^Biotrends – Launch trends - Benlysta®

*IMS Data

^^LFA estimates

15

*Projection based on IMS-NDTI data includes both renal

(65%) and non renal (35%) Lupus patients

**Internal Aurinia projections

Average Annual Cost of Illness Per Patient ***

$0 $10,000 $20,000 $30,000 $40,000 $50,000 $60,000

SLE nephritis

Renal failure

Crohn's disease

Systemic lupus eurythematosus

Ulcerative colitis

SLE without nephritis

Rheumatoid arthritis

Heart disease

Bipolar disorder

Chronic obstructive pulonary disease

Diabetes

Hypertension

Asthma Medical costs

Absence costs

Short term

disability costs$14,909

$64,195

$22,427

$35,687

$25,921

$25,215

$21,161

$19,530

$17,860

$15,407

$14,079

$10,125

$8,907

*** Journal of Occupational and Environmental Medicine, Volume 51, Number 1, January 2009

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The Economic Burden of Patients Suffering from Lupus

Nephritis Can Exceed $60K/year

16

0

10000

20000

30000

40000

50000

60000

70000

SLE (without LN) LN

20

05

US

D

Direct and Indirect Costs of SLE (without LN) and LN*

Inpatient Emergency

Outpatient Rx

Absenteeism Short-term disability

Total = $21,161

Total = $64,195

Indirect

Indirect

Direct

Direct

*Carls et al.,JOEM., Volume 51, No. 1, January 2009

**Li T, et al. Arthritis Rheum. 2009;61(6):755-763.

***Does not include cost of transplant

$0

$20,000

$40,000

$60,000

$80,000

$100,000

$120,000

1 2 3 4 5

Me

an

An

nu

al M

ed

ical C

osts

Year Since Initial Diagnosis

LN with ESRD (n = 156)

LN without ESRD (n = 333)

LN (n = 489)

SLE without LN (n = 1,809)

SLE (n = 2,298)

Reference (n = 2,298)

Mean Annual Medical Costs Associated with

Lupus with and without Nephritis**

NASDAQ - AUPH

******

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Although the cost to treat LN can be greater than other autoimmune diseases the treatment objectives are remarkably similar:

– Crohn’s Disease / Psoriatic Arthritis / Multiple Sclerosis:

Induce/maintain remission – avoid destructive flares leading to permanent tissue damage

Decrease frequency of hospital and ambulatory care visits

Limit long-term disability

Reduce overall steroid burden

Improve QoL and wellbeing of the patients

In LN physicians are trying to avoid, dialysis, renal transplantation and death

Voclosporin pricing potential should be considered within

the context of other autoimmune diseases

17

$10K

$20K

$30K

$40K

$50K

$60K

$70K

$80K

An

nu

al W

AC

Price

*

200K 250K 300K 350K 400K

Estimated Prevalence

BENLYSTA® – nrSLE

ENBREL® – PsA

STELARA® – PsA

GILENYA™ – MS

TECFIDERA™ – MS

450K

CIMZIA® – CD

500K

TYSABRI® – CD

~LN

*WAC price from Analysource – Calculated as per package insert for indication

.

LN is Less Common than other Autoimmune

Diseases and can have a higher

Pharmacoeconomic Burden

HUMIRA® – CD

REMICADE® – CD

COPAXONE® – MS

AUBAGIO® – MS

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Management with track record of industry success & extensive expertise and focus on lupus nephritis

Well-capitalized, efficient public entity (NASDAQ: AUPH TSX: AUP)

– 32.3 million shares outstanding, 5.9 mil warrants

Solid safety and proof of concept package

– > 2,000 patients treated with voclosporin to date >> well characterized safety profile

– Same multi-target therapy (MTT) approach used in transplantation

– Strong clinical evidence to support CNI efficacy as part of MTT

– Supportive 8-week Proof of Concept - AURION

Large and well-defined market opportunity

– Renal lupus has significant unmet medical need with an extremely high pharmaco-economic burden

– Growing market projected to exceed $3B^ in value by the end of this decade

– Robust IP protection with likelihood of data exclusivity in major markets

Investment Summary

18

AURA LV enrollment

complete N=265

Q1 / 2016 Q2 / 2016 Q3 / 2016 Q4 / 2016

AURION 8-Week data

collection/ enrollment

complete

AURA LV 24-week

1° data read-out

AURION 24-Week

data

collection/release

FDA End Ph.II

meeting*

*Following positive AURA results

^Data Monitor

FDA Fast Track

designation