Q3 2019 Results - Novartis

Q3 2019 ResultsInvestor presentation

October 22, 2019

Novartis AG

Investor Relations

Disclaimer

| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 2

This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by

words such as “potential,” “expected,” “will,” “planned,” “pipeline,” “outlook,” or similar expressions, or by express or implied discussions regarding potential new products, potential

new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding the potential outcome, or financial or

other impact on Novartis, of the proposed divestiture of certain portions of our Sandoz Division business in the US; or regarding the potential impact of the completion of the up to

USD 5 billion share buyback; or regarding potential future sales or earnings of the Group or any of its divisions or potential shareholder returns; or by discussions of strategy, plans,

expectations or intentions. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant

known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary

materially from those set forth in the forward-looking statements. You should not place undue reliance on these statements. In particular, our expectations could be affected by,

among other things: global trends toward healthcare cost containment, including ongoing government, payor and general public pricing and reimbursement pressures and

requirements for increased pricing transparency; regulatory actions or delays or government regulation generally, including potential regulatory actions or delays with respect to the

proposed transactions or the development of the products described in this presentation; the potential that the proposed divestiture of certain portions of our Sandoz Division

business in the US may not be completed in the expected time frame, or at all; the potential that the strategic benefits, synergies or opportunities expected from the proposed

divestiture of certain portions of our Sandoz Division business in the US, and other transactions described, may not be realized or may be more difficult or take longer to realize than

expected; the inherent uncertainties involved in predicting shareholder returns; the uncertainties inherent in the research and development of new healthcare products, including

clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the

impact on Novartis of the loss of patent protection and exclusivity on key products that commenced in prior years and will continue this year; safety, quality or manufacturing issues;

uncertainties involved in the development or adoption of potentially transformational technologies and business models; uncertainties regarding actual or potential legal proceedings,

including, among others, product liability litigation, disputes and litigation with business partners or business collaborators, government investigations generally, litigation and

investigations regarding sales and marketing practices, and intellectual property disputes; our performance on environmental, social and governance measures; general political,

economic and trade conditions, including uncertainties regarding the effects of ongoing instability in various parts of the world; uncertainties regarding future global exchange rates;

uncertainties regarding future demand for our products; uncertainties regarding potential significant breaches of data security or data privacy, or disruptions of our information

technology systems; and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the

information in this presentation as of this date and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or

otherwise.

Eylea® is a registered trademark of Regeneron Pharmaceuticals, Inc.

Vas NarasimhanChief Executive Officer

Strong Q3 with double digit top and bottom line growth, margin expansion and major innovation milestones


Q3 operational performanceContinuing operations1 growth vs. PY in % cc 2

Innovation performance

Sales

+13%

Sales & Core OpInc guidance increased

Core OpInc

+18%

Core margin

+1.4%

pts

OS – overall survival RMS – relapsing forms of multiple sclerosis 1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz

(including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be

found on page 56 of the Condensed Interim Financial Report 3. Study narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women and patients with lower ejection fraction

Launched in US

Ofatumumab Compelling efficacy in RMS

Met primary endpoints in nr-axSpA

Achieved OS in 2nd Ph3 study

Clinically important benefit in HFpEF

sub-groups3

QVM149 / QMF149 Positive Ph3 results in asthma

Key growth drivers represent 28% of 9M Innovative Medicines sales and contributed +8%pts to Novartis sales growth


8%

9%

1%2%

Growth drivers CC growth

9M 2019

Gx erosion

volume

Others

28%

Key growth drivers sales in USD m

(% of Innovative Medicines sales)

9M 20179M 2016 9M 20199M 2018

Promacta®

Piqray®

Zolgensma®

Cosentyx®

Entresto® Xolair®

Tafinlar®+Mekinist®

Lutathera®

Kisqali®

Kymriah®

Xiidra®

10%

16%

21%

Growth contribution to continuing operations sales

(% points)

Strong sales performance of key growth drivers

NM – not meaningful


SalesUSD Million

Growth vs. PYUSD Million

Growth vs. PYcc

SalesUSD Million

Growth vs. PYcc

937 27% 2,586 30%

160 nm 175 nm

430 61% 1,208 75%

102 nm 102 nm

380 31% 1,036 26%

119 116% 334 nm

79 nm 182 nm

345 22% 982 22%

123 76% 325 92%

299 22% 870 20%

43 nm 49 nm

Q3 9M

Key growth drivers

Lutathera®

2019 2019

85

187

160

159

102

63

59

54

51

44

43

Cosentyx® sales driven by strong demand across indications and geographies

Cosentyx® revenues USD m, % cc

459601

291

336

Q3 2018

US

Ex-US

Q3 2019

750

937

+27%

Strong momentum in growing US dermatology

market1

Dermatology market TRx +17% YoY, driven by novel agents

Cosentyx® TRx +32% YoY, despite intensifying competition

Leading expansion in growing US SpA market2

SpA market TRx +15% YoY

Cosentyx® major contributor to market growth, TRx +36% YoY

Positive CHMP opinion for up-titration to 300mg in AS

PREVENT met primary endpoints at 16 and 52 weeks


1. IQVIA National Prescription Audit for Dermatology, WE 09/27/2019 2. IQVIA National Prescription Audit for Rheumatology, WE 09/27/2019. Rx for SpA indications. Market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz® and

Cosentyx®

1.7m

PsA AS nr-axSpA

3.2m

1.7m

19-27% 12-21% 4-8%Biologics

penetration3

Nr-axSpA indication builds Cosentyx® leading position in SpA, potentially doubling patient population in axSpA1

Significant potential for growth

across SpA populations (US & EU5)2

Well-positioned for further growth


Diagnosed

Undiagnosed

Biologics

treated

Please see appendix for references All trademarks are the property of their respective owners HS – Hidradenitis Suppurativa PsO – Psoriasis JIA – Juvenile Idiopathic Arthritis GCA – Giant Cell Arthritis

PREVENT Could add nr-axSpA to label; submissions

ongoing

MAXIMISE Proved for the first-time benefits in axial

manifestations of PsA

EXCEED Assessing benefits in joints vs. Humira® –

expected H1 2020

Other clinical

trials

Including HS, pediatric PsO, pediatric JIA

and Ph2 GCA study, on track

Entresto® solidifying position as standard of care in HFrEFwith >USD 1.2bn sales YTD

Solid QoQ demand growth US and ex-US

PROVE-HF and EVALUATE-HF provide mechanistic support for Entresto® efficacy1

FDA approved pediatric indication in Q4

HFrEF – heart failure with reduced ejection fraction 1. While several structural and functional echocardiographic measures versus enalapril showed improvement, EVALUATE didn’t meet the primary endpoint of change from baseline in aortic

characteristic impedance at 12 weeks

Entresto® revenues USD m, % cc

151220

120

210

Q3 2018 Q3 2019

Ex-US

US

271

430

+61%


01

/18

30

03

/18

11

/17

05

/18

07

/18

09

/18

05

/19

07

/19

01

/19

03

/19

11

/18

11

/19

0

25

35

20

40

45

09

/19

Entresto® weekly US TRx2017-19, thousands

+51%

Q3

2018

Q3

2019

PARAGON-HF: clinically important effect against active comparator supports expansion of addressable population

Regulatory and payer discussions ongoing. US submission for inclusion of data in label Q4 2019

Largest HFpEF outcomes trial on back of approved

HFrEF indication

Despite narrow miss on primary endpoint, pre-defined

secondary endpoints and subgroup analyses

supportive of benefit

Greater treatment benefit in HFpEF patients with

ejection fraction below median, and in women

HFrEF – heart failure with reduced ejection fraction HFpEF – heart failure with preserved ejection fraction


Total HF hospitalizations and CV deathN= 4796

Rate ratio 0.87 (95% CI 0.75, 1.01)

p = 0.059

Sacubitril/valsartan

Valsartan

Broad access

Zolgensma® off to a strong start with sales USD 175m since launch

Solid demand Broad patient profile

Even distribution across

age, SMA types and

incident and prevalent

populations

>50% switches from

nusinersen

>50 treating institutions

across US - majority of

leading academic centers

of excellence having

prescribed since launch

~90% commercial

patients and ~30%

Medicaid patients

covered

>99% final approval rate

for on label patients


Zolgensma® key opportunities to build on US launch momentum

Newborn screening (US): 30% of newborns

screened, expected to reach 70% end of 2020

~2/3 of incident patients treated in states with

newborn screening (vs. ~1/4 in states without)

Medicaid policies: coming in place rapidly

including key states like Florida, New Jersey

and Michigan

New interim STRONG data: reinforcing

efficacy in SMA type II (IT, +5.9pts HFMSE

score, patients 2-5 years old)

USAwaiting FDA feedback on IT filing

approach

EU CHMP opinion anticipated in Q1 2020

Japan Decision anticipated in 1H 2020

Early

accessIncluding France, Portugal, Germany


Opportunities Regulatory

IT – intrathecal

Strong start to Beovu® US launch, highly competitive label Similar efficacy with fewer injections, supported by visual & anatomical measures


Longer treatment intervals achievable

without compromising efficacy

Only q12w without compromise to efficacy*

Only treatment with q12w recommended

immediately after loading

Flexibility from q8w to q12w

All trademarks are the property of their respective owners Source: BEOVU [prescribing information] East Hanover, NJ. Novartis: 2019, Dugel P, et al. HAWK and HARRIER: Ph3, multicenter, randomized, double-masked trials of brolucizumab

for neovascular age-related macular degeneration [published online ahead of print]. Ophthalmology. 2019. nAMD = neovascular age related macular degeneration; VEGF = Vascular Endothelial Growth Factor; q12w = every 12 weeks

Supportive label language on

visual and anatomical measures**

Recognition of treatment decisions

according to visual and anatomical

measures of disease activity

Reductions in central retinal subfield

thickness across all treatment arms

Overall safety profile in

line with comparator

*Eylea® prescribing information states “Although not as effective as the recommended every 8-week dosing regimen, patients may also be treated with one

dose every 12 weeks after one year of effective therapy. Patients should be assessed regularly”. Lucentis® prescribing information states: “Although not as

effective, patients may also be treated with one dose every 3 months after 4 monthly doses. Patients should be assessed regularly.”

**According to the FDA label, the utility of these measures has not been established

Most common adverse

reactions (≥5%): vision

blurred, cataract,

conjunctival hemorrhage,

eye pain, vitreous floaters

Beovu® comprehensive clinical trial programs ongoing including potential new indications

DME RVO PDRAMD

A head-to-head superiority study

vs. aflibercept evaluating treatment

interval duration in an identical

Treat-to-Control regimenPhase 3 head-to-head non-inferiority

studies vs. aflibercept to support

global registration

CONDOR

Phase 3 non-inferiority study vs.

aflibercept to support global

registration

20212 ≥20232 ≥20232,32019-20201

A head-to-head non-inferiority study

vs. aflibercept evaluating the efficacy

and safety of q4w treatment

1. First expected approval date 2. first planned submission 3. Study name pending approval AMD = Age related macular degeneration, PCV = Polypoidal Choroidal Vasculopathy, DME = Diabetic Macular Edema, RVO = Retinal vein

occlusion, PDR = Proliferative diabetic retinopathy; q4w = every 4 weeks

PCV studyRandomized, open-label, multicenter

study assessing the safety & efficacy

of two different dosing regimens of

brolucizumab 6 mg in patients with

symptomatic macular PCV

A head-to-head non-inferiority study

vs. aflibercept evaluating the efficacy

and safety of q4w treatment

Phase 3 non-inferiority studies of

brolucizumab q12w/ q8w vs.

aflibercept to support global

registration


Ofatumumab – high efficacy disease-modifying therapy with the potential to be used early and broadly

1. CDW, confirmed disability worsening and CDP, confirmed disability progression are interchangeable terms, defined by an increase ≥1.5 EDSS points for patients with baseline EDSS of 0, increase of ≥1.0 EDSS points patients with baseline

EDSS of 1.0-5.0 and increase of t ≥0.5 EDSS points for patients with baseline EDSS of 5.5 2. NfL levels at month three measured as adjusted geometric mean levels and difference is geometric mean ratio (GMR)

Strong efficacy results vs. teriflunomide

(ASCLEPIOS I&II)

Superior efficacy for relapses, MRI activity

Substantial reductions in 3- and 6-month

disability progression1

Lower levels of NfL2

Safety profile – no significant signals

concerning infections/ malignancies

Supporting highly competitive

product profile

High efficacy

Favorable safety profile

Convenient subcutaneous option

No need for infusion center


Initiating worldwide regulatory submissions starting Q4 2019

Market feedback on Mayzent® encouraging – acceleration of diagnosis and onboarding needed for sales ramp-up


Appreciated by customers

and payers

Supportive label and

additional unique data

90% willingness to prescribe4

>2600 SFs, 2/3 in aSPMS

>150m lives with preferred or

unrestricted access

Accelerating diagnosis and

onboarding

Shift focus from relapses to

progression

Drive urgency to treat

Simplify onboarding

Accelerate time to paid Rx

Active SPMS1 (EDSS 3-6)

Reduces disease progression

Safety and tolerability

Cognitive processing speed2

4 year delay to wheelchair3

Expect CHMP opinion in late Q4 2019

Please see appendix for references aSPMS – active Secondary progressive multiple sclerosis EDSS – Expanded Disability Status Scale SF – Start Form

FevipiprantLUSTER to determine benefit in moderate-severe asthma. ZEAL confirmed clean safety profile but no significant lung function improvement in less severe population


Comprehensive Ph3 program to define full potential

Exacerbation

52 weeks

GINA 4/5

Core

registration

Results expected Q1 2020

Safety

Up to 3 years

GINA 3/4/5

Core

registration

Interim results expected Q1

2020

Lung function

12 weeks

GINA 3/4

Supportive

studies

No significant improvement of

FEV1 on top of ICS ± LABA1,2

Clean safety profile confirmed3

US patient numbers (≥ 12 years)

Ph3 asthma study populations

ZEAL

1.4m

patients5

BASE CASE

LUSTER: high eosinophils4

1.5m patients

SevereModerate

ASTHMA SEVERITY

EO

SIN

OP

HIL

S4

HIGH

LOW

FURTHER POTENTIAL

LUSTER: low eosinophils

1.5m patients

GINA – Global Initiative for Asthma

1. In combination with inhaled standard of care – predominantly low-medium dose ICS+LABA 2. Detailed analysis of the ZEAL1 & 2 data is ongoing & results to be communicated in H1 2020 3. Based on ZEAL 1 & 2 and a dedicated

thorough QT study which demonstrated absence of a QTcF signal with fevipiprant vs. placebo (Novartis data on file) 4. High eosinophils defined as ≥ 250 cells/µL 5. patients with unresolved symptoms and exacerbations on GINA 3 regimes

Piqray® launch off to strong start with USD 49m YTD sales


aBC – advanced breast cancer TNBC – triple negative breast cancer HR+/HER2- – hormone receptor positive, human epidermal growth factor receptor-2 negative 1. Alpelisib (BYL719) also being explored in PIK3CA-related overgrowth

spectrum (PROS), a group of rare disorders driven by mutations in the PIK3CA gene

Strong launch in USUSD million

Building foundation for global launch

First and only therapy for aBC patients with PIK3CA mutation

Approximately 40% of the HR+/HER2- aBC population have

a mutation in PIK3CA

Good initial uptake of PIK3CA testing in the US

Foundation Medicine PIK3CA CDx tissue approval

anticipated Q4 2019

CHMP opinion expected H1 2020

Potential to expand1 beyond aBC with programs starting in

HER2+, TNBC, head & neck, ovarian cancer6

43

Q3 2019Q2 2019

Kisqali® is the only CDK4/6 inhibitor to demonstrate consistently superior overall survival (OS) in two Ph3 trials


MONALEESA-3Post-menopausal

The only CDK4/6 with two positive readouts OS benefit proven across multiple populations

and combination partners

Kisqali® plus fulvestrant achieved statistically significant OS

benefit in postmenopausal patients (MONALEESA-3)

Kisqali® plus endocrine therapy achieved statistically significant

OS benefit in pre- and peri-menopausal patients

(MONALEESA-7)

Across both pivotal Ph3 trials, Kisqali demonstrated 28% / 29%

reduction in the risk of death

Comprehensive OS evidence expected to further differentiate

Kisqali® within CDK4/6 class

MONALEESA-7Pre-menopausal

2019 expected pipeline milestones


H1 2019 H2 2019

Regulatory

decisions and

opinions

Mayzent®1 SPMS (US) ✓ BYL719 (Piqray®) HR+ Breast Cancer (US) ✓2

Kymriah® Ped / Young Adult r/r ALL (JP) ✓ Beovu® nAMD (US) ✓

Kymriah® r/r DLBCL (JP) ✓ Lucentis® RoP (EU / JP) ✓ (EU)

Promacta® Severe aplastic anaemia (EU) ✕ Lucentis® Diabetic Retinopathy (EU) ✓3

Zolgensma® SMA type I (US) ✓ Mayzent®1 SPMS (EU / JP)

Zolgensma® SMA type I (EU / JP)EU Q1 2020

JP H1 2020Xolair® Pollinosis (JP)

Major

expected

submissions

Brolucizumab (RTH258) nAMD (US / EU / JP) ✓ Cosentyx® nr-AxSpA (US / EU / JP) ✓ (EU)

Crizanlizumab (SEG101) Sickle Cell Disease (US / EU) ✓ Entresto® HF-rEF (JP) ✓Mayzent®1 SPMS (JP) ✓ Entresto® HF-pEF (US / EU)

INC280 NSCLC (US / JP)

Ofatumumab (OMB157) Relapsing MS (US / EU)

PDR001 (combination

with Tafinlar®+Mekinist®) Metastatic Melanoma (US / EU) H1 2020

QVM149 Asthma (EU / JP) ✓

Major

expected trial

readouts

AVXS-101 IT SMA type II IT Formulation ✓ Cosentyx® nr-AxSpA ✓

Zolgensma® SMA type I / II /

presymptomatic✓ Entresto® HF-pEF (✓)4

Fevipiprant (QAW039) Asthma5

Ofatumumab (OMB157) Relapsing MS ✓PDR001 (combination

with Tafinlar® + Mekinist®) Metastatic melanoma

✓ Achieved* ✕ Missed*

*Represents achieving on-time readout of the data, irrespective of trial outcome 1. The name Mayzent® has now been fully approved by the FDA and so has the product. But the name has only been provisionally approved in Europe, and the

product has not yet been approved there 2. Piqray® FDA approval achieved in H1 2019. 3. CHMP positive opinion received 4. Study narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women

and patients with lower ejection fraction 5. ZEAL 1/2 readouts complete (did not meet the primary efficacy endpoint of FEV1 improvement in moderate asthmatic patients), LUSTER 1/2 readouts expected Q1 2020

2020 catalysts expected to maintain 2019 momentum

Potential

catalysts

Selected

examples

Key

approvals1

Ofatumumab (OMB157)

Relapsing MS

Crizanlizumab (SEG101)

Sickle cell disease

Capmatinib (INC280)

NSCLC

QVM149

Asthma

Cosentyx®

nrAxSpA

Key

submissions2

Fevipiprant (QAW039)

Asthma

177Lu-PSMA-617

mCRPC

AVXS-101 IT

SMA type 2/3

PDR001 combo4

Metastatic melanoma

Alpelisib (BYL719)

PIK3CA-related overgrowth

spectrum

Key

readouts3

Phase 3 Select Phase 2

177Lu-PSMA-617

mCRPC

ABL001 (3rd line)

Chronic myeloid leukemia

Entresto®

Post-acute MI

Beovu®

DME

Kisqali®

Breast cancer (MONALEESA-2 OS)

Jakavi®

Chronic GvHD

Tropifexor (LJN452)

NASH

LOU064

Autoimmune diseases

LNP023

Renal diseases and

paroxysmal nocturnal

hemoglobinuria

1. First approval in any market 2. First submission in any market 3. Readouts enabling submission, label change or pivotal trial initiation 4. In combination with Tafinlar® and Mekinist®


Harry KirschChief Financial Officer

% USD % cc % USD % cc

Net Sales 12,172 10 13 35,042 5 9

Core Operating income 3,748 15 18 10,650 13 18

Operating income 2,358 5 9 7,263 3 10

Net Income 2,041 8 12 6,018 -48 -45

Core EPS (USD) 1.41 16 19 3.97 12 17

EPS (USD) 0.90 11 14 2.62 -47 -44

Free Cash Flow 3,968 26 9,449 13

Change vs. PYContinuing operations

1

USD million

Q3

2019

9M

2019

Change vs. PY

Summary of Q3 and 9M 2019 continuing operations financial results

1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well

as the continuing corporate functions 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 56 of the Condensed Interim Financial Report


2

2

2

Net sales

change vs. PY

Core operating

income

change vs. PY Core margin

Core margin

change vs. PY Core margin

Core margin

change vs. PY

(in % cc) (in % cc) (%) (%pts cc) (%) (%pts cc)

Innovative Medicines 15 16 34.1 0.4 34.3 2.2

Sandoz 5 18 24.8 2.8 21.8 1.6

Continuing Operations 13 18 30.8 1.4 30.4 2.4

Q3 2019 9M 2019

Continuing operations delivering core margin expansion of 1.4%pts cc vs. PY

1


2

2

2

2

2

1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well

as the continuing corporate functions 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 56 of the Condensed Interim Financial Report

New focused medicines company 2019 FY guidance: sales and core operating income revised upwards

Barring unforeseen events (in cc)

New focused medicines company | full year guidanceExcl. Alcon and Sandoz proposed US portfolio sale to Aurobindo1 from both 2018 and 2019; growth vs. PY in cc

Sales revised upwards expected to grow high single digit

• IM Division revised upwards to grow high single digit to low double digit

• Sandoz revised upwards to grow low single digit

| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation

Key Assumption: All guidance includes forecast assumption that no Gilenya® generics enter in 2019 in US.

1. Novartis continues to expect the previously-announced divestment of the Sandoz US oral solids and dermatology portfolio to be completed in the coming months, pending closing conditions including regulatory approvals. 2018 FY Sales and

Core OpInc of the Sandoz US Oral solids and Dermatology businesses were approximately USD 1.2bn and 0.3bn, respectively.

25

Core operating income revised upwards expected to grow mid to high teens

Core OpInc growth with continued strong underlying momentum; potential generic headwinds in Q4 2019

26

Key drivers of continuing operations core operating incomevs. PY (cc)

9M 2019

+ Innovative Medicines growth drivers

including Cosentyx®, Entresto® and

Zolgensma®

+ Productivity

+ Valsartan competitor supply

shortage

+ Innovative Medicines growth drivers,

launches uptake1

+ Productivity

− Potential increased Gx erosion2

− Potential resolution of valsartan

competitor supply shortage

− Increased investments in pre-launch

and launches

Q4 2019

1. Including Zolgensma®, Mayzent®, Aimovig®, Piqray® 2. Mainly Afinitor ®, Exjade® / Jadenu® and Ophthalmology brands


Currency impact vs. PY%pts, assuming mid-October exchange rates prevail in 2019 and 2020

Expected currency impact for full year 2019 and 2020

SimulationActual


FX impact on

Net sales

FX impact on

Core operating income

1

-5 -4 -3 -3-1

-9-6

-3-5

-1

FYQ3FY Q1 FYQ2 FYQ4 Q1 Q2 Q3 Q4 FY FY

0

-2 to -3-1 to -2

2018 2019

27

2020 2018 2019 2020

Vas NarasimhanChief Executive Officer

Conclusion

Increased full year sales and core operating income guidance

Excellent Q3 performance with double digit increases in top and bottom line,

and margin expansion

Zolgensma® and Piqray® launches off to a strong start


Continuing innovation, notably ofatumumab’s remarkable efficacy in RMS

Appendix

Ofatumumab – high efficacy on key measures of disease activity

32

End point OMB. TER. RRRc p value OMB. TER. RRRc p value

Annualized relapse rate 0.11 0.22 50.5% <0.001 0.1 0.25 58.5% <0.001

Gd+ T1 lesions per scan 0.01 0.45 97.5% <0.001 0.03 0.51 93.8% <0.001

New or enlarging T2 lesions per year 0.72 4.00 82.0% <0.001 0.64 4.15 84.5% <0.001

Serum NfL levels at month 3a 8.8 9.41 7%c 0.011 8.92 10.02 11%c <0.001

Brain volume lossb -0.28 -0.35 0.07 0.116 -0.29 -0.35 0.07 0.129

End point OMB. TER. RRRc p value

3-month CDW* 10.9% 15.0% 34.4% 0.002

6-month CDW* 8.1% 12.0% 32.5% 0.012

6-month CDI 11.0% 8.1% -35.2% 0.094

ASCLEPIOS I ASCLEPIOS II

PRE-SPECIFICED COMBINED ANALYSIS OF ASCLEPIOS I & II

*CDW, confirmed disability worsening and CDP, confirmed disability progression are interchangeable terms, defined by an increase ≥1.5 EDSS points for patients with baseline EDSS of 0, increase of ≥1.0 EDSS points patients with baseline

EDSS of 1.0-5.0 and increase of t ≥0.5 EDSS points for patients with baseline EDSS of 5.5 a NfL levels at month three measured as adjusted geometric mean levels and difference is geometric mean ratio (GMR) b Brain volume loss is

measured as difference in slope between 12 and 24 months, difference measured as adjusted mean difference in slope c RRR is relative risk reduction and in cases where it is different- highlighted in footnote; OMB = Ofatumumab; TER =

Teriflunomide; CDI = Confirmed Disability Improvement.


Net debt increased by USD 3.2bn driven by M&A transactions and share buybacks

33

-6.6

-3.8

-5.3

Dec 31, 2018 Dividends Free Cash Flow1

2.9

-16.2

0.2

OthersAlcon Net debt2 Sep 30, 2019Treasury share

transactions, net

M&A

transactions1

-19.4

9.4

-3.2


1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines (including the Xiidra® acquisition for USD 3.5bn) and Sandoz (including the US

generic oral solids and dermatology portfolio), as well as the continuing corporate functions 2. Includes the net de-recognition of USD 0.6bn cash and cash equivalents and USD 3.5bn of financial debts related to the Alcon spin-off

USD billion

Key drivers vs. PY:

+ Higher operating income

(adjusted for non-cash items)

− Higher working capital

Offsetting one-time effects:

+ Real estate divestment proceeds

− Prior year OTC JV dividend and GSK milestone income

9M 2019 free cash flow at USD 9.4bn

8.39.4

9M 2018 9M 2019

+13%

Continuing operations1 free cash flow2

USD billion

1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as

the continuing corporate functions 2. Free cash flow is a non-IFRS measure. Further details regarding non-IFRS measures can be found starting on page 56 of the Condensed Financial Report


Planned filings 2019 to 2022

35 | Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation

Entresto®

Heart failure (PEF)

INC280 NSCLC

Cosentyx® USnr-axSpA

OMB157Relapsing multiple sclerosis

2019

Xolair EUNasal Polyps

New molecule

New indication

New formulation

Biosimilars

2022

Cosentyx®

AS H2H

Rydapt®AML (FLT3 wild type)

ECF843Dry eye

ACZ885Adjuvant NSCLC

Cosentyx®

Hidradenitis suppurativa

QAW039Asthma

2020

Jakavi®Acute GVHD

Cosentyx®

PsA H2H

Jakavi®Chronic GVHD

PDR001 + Taf/MekMetastatic BRAF V600+ melanoma

AVXS-101 ITSMA Type 2/3

177Lu-PSMA-617mCRPC

ABL001CML 3rd line

QGE031CSU

Entresto®

Post-acute myocardial infarction

Beovu® (RTH258)Diabetic macular edema

2021

LAM320a

MDR tuberculosis

ACZ8851st Line NSCLC

ACZ8852nd Line NSCLC

Kymriah®

r/r Follicular Lymphoma

Kymriah®

r/r DLBCL in 1st relapse

MBG453MDS

PsA H2H Psoriatic arthritis head-to-head study versus adalimumab

ROP Retinopathy of prematurity

RCC Renal cell carcinoma

SAA Severe aplastic anemia

SMA Type 1 Spinal Muscular Atrophy Type 1 (IV formulation)

SMA Type

2/3

Spinal Muscular Atrophy Type 2/3 (IT formulation)

SPMS Secondary Progressive Multiple Sclerosis

TNBC Triple negative breast cancer

MDR Multi-drug resistant

MDS Myelodysplastic syndrome

nAMD Neovascular (wet) age-related macular degeneration

NASH Non-alcoholic steatohepatitis

nr-axSpA Non-radiographic axial spondyloarthritis

NSCLC Non-small cell lung cancer

PDR Proliferative Diabetic Retinopathy

PEF Preserved ejection fraction

Indication abbreviations:

ALL Acute lymphoblastic leukemia

AML Acute myeloid leukemia

AS H2H Ankylosing spondylitis head-to-head study versus

adalimumab

BC Breast cancer

CLL Chronic Lymphocytic Leukemia

CML Chronic myeloid leukemia

CRC Colorectal cancer

CSU Chronic spontaneous urticaria

CVRR Secondary prevention of cardiovascular events in patients

with elevated levels of lipoprotein (a)

GCA Giant cell arteritis

GVHD Graft-versus-host disease

HNSCC Head and neck squamous cell carcinoma

mCRPC Metastatic castration-resistant prostate cancer

2023

ZPL389Atopic dermatitis

UNR844Presbyopia

QBW251COPD

VAY736Autoimmune Hepatitis

ABL001CML 1st line

VPM087CRC 1L/RCC 1L

SAF312Chronic ocular surface pain

BYL719 (Piqray® US)HER2+ adv. breast cancer

TQJ230CVRR

BYL719 (Piqray® US)TNBC2

BYL719 (Piqray® US)HNSCC 2/3L

BYL719 (Piqray® US)Ovarian Cancer

AVXS-201Rett Syndrome

KAE609Malaria

KAF156 Malaria

LJN452NASH

HDM201AML

CFZ533Solid Organ Transplant

CSJ117Severe Asthma

LJC242NASH (+cen)

LMI070Spinal muscular atrophy

LNP023IgA nephropathy

LOU064CSU

MOR106Atopic Dermatitis

LXE408Visceral leishmaniosis

VAY736Primary Sjoegren’s syndrome

Kymriah ® + pembror/r DLBCL10

CFZ533Sjoegren’s Syndrome

LNP023Membranous nephropathy

RTH258Retinal vein occlusion

Kisqali ®HR+, HER2 (-) BC9 (adjuvant)

PDR001 comboMetastatic Melanoma

LNP023C3 glomerulopathy

GP2411 (denosumab)Osteoporosis, skeletal-related in bone

met. pts (same as originator)

KAE609Severe Malaria

Cosentyx®

GCA

RTH258PDR

Combination abbreviations:

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

cen cenicriviroc

pembro pembrolizumab

fulv fulvestrant

BYL719PIK3CA-related overgrowth spectrum

http://deis.novartis.intra/deis.asp?VAK694A


http://deis.novartis.intra/deis.asp?ATI355A






http://deis.novartis.intra/deis.asp?SEB001X



Pipeline of key projects in confirmatory development

Early Clinical Trials Registration Trials – Phase III / Pivotal In Registration

36| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation

a) Approved in US, submitted in EU.

LMI070Spinal muscular atrophy

LNP023IgA nephropathy

KAE609Malaria

ECF843Dry eye

KAF156Malaria

HDM201AML

CFZ533Solid Organ Transplant

CSJ117Severe Asthma

AVXS-201Rett Syndrome

LJC242NASH (+cen)

177Lu-PSMA-617mCRPC

ABL001CML 1st line

QAW039Asthma

ACZ885Adjuvant NSCLC

ACZ8851st Line NSCLC

INC280 NSCLC

ABL001CML 3rd line

QGE031CSU

PDR001 + Taf/MekMetastatic BRAF V600+ melanoma

ACZ8852nd Line NSCLC

BYL719 (Piqray® US)HER2+ adv. BC

TQJ230CVRR

LJN452NASH

QBW251COPD

UNR844Presbyopia

VAY736Autoimmune Hepatitis

LOU064CSU

MOR106Atopic Dermatitis

VPM087CRC 1L/RCC 1L

SAF312Chronic ocular surface pain

MBG453MDS21

LXE408Visceral leishmaniosis

VAY736Primary Sjoegren’s syndrome

ZPL389Atopic dermatitis

PDR001 comboMetastatic Melanoma

Kymriah®+ pembro

r/r DLBCL

CFZ533Sjoegren’s Syndrome

LNP023Membranous nephropathy

Zolgensma®SMA Type 2/3

LNP023C3 glomerulopathy

KAE609Severe Malaria

PsA H2H Psoriatic arthritis head-to-head study

versus adalimumab

ROP Retinopathy of prematurity

RCC Renal cell carcinoma

SAA Severe aplastic anemia

SMA Type 1 Spinal Muscular Atrophy Type 1 (IV

formulation)

SMA Type 2/3 Spinal Muscular Atrophy Type 2/3 (IT

formulation)

SPMS Secondary Progressive Multiple Sclerosis

TNBC Triple negative breast cancer

Indication abbreviations:

Cosentyx® USnr-axSpA

Entresto®

Heart failure (PEF)

Entresto®

Post-acute myocardial infarction

Jakavi®Acute GVHD

Cosentyx®

PsA H2H

Cosentyx®

AS H2H

Jakavi®Chronic GVHD

Cosentyx®

Hidradenitis suppurativa

BYL719 (Piqray® US)TNBC

BYL719 (Piqray® US)HNSCC

BYL719 (Piqray® US)Ovarian Cancer

Cosentyx®

GCA

LAM320MDR tuberculosis

OMB157 Relapsing multiple sclerosis

RTH258Diabetic macular edema

Rydapt®AML (FLT3 wild type)

Xolair EUNasal Polyps

RTH258Retinal vein occlusion

Kymriah®

r/r DLBCL in 1st relapse

Kisqali®

HR+, HER2(-) BC (adjuvant)

Kymriah®

r/r Follicular Lymphoma

GP2411 (denosumab)2

Osteoporosis, skeletal-related in bone met. pts (same as originator)

RTH258PDR

GVHD Graft-versus-host disease

HNSCC Head and neck squamous cell carcinoma

mCRPC Metastatic castration-resistant prostate

cancer

MDR Multi-drug resistant

MDS Myelodysplastic syndrome

nAMD Neovascular (wet) age-related macular

degeneration

NASH Non-alcoholic steatohepatitis

nr-axSpA Non-radiographic axial spondyloarthritis

NSCLC Non-small cell lung cancer

PDR Proliferative Diabetic Retinopathy

PEF Preserved ejection fraction

ALL Acute lymphoblastic leukemia

AML Acute myeloid leukemia

AS H2H Ankylosing spondylitis head-to-head

study versus adalimumab

BC Breast cancer

CLL Chronic Lymphocytic Leukemia

CML Chronic myeloid leukemia

CRC Colorectal cancer

CSU Chronic spontaneous urticaria

CVRR Secondary prevention of cardiovascular

events in patients with elevated levels of

lipoprotein (a)

GCA Giant cell arteritis

Combination abbreviations:

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

cen cenicriviroc

pembro pembrolizumab

fulv fulvestrant

Promacta®/Revolade®

SAA17 1st line

SEG101Sickle cell disease

RTH258a EU (Beovu®)nAMD6

Lucentis®

Diabetic retinopathy

BAF312a EU (Mayzent™US)SPMS20

QMF149Asthma

QVM149Asthma

LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia and

others (same as originator)

Zolgensma®a

SMA Type 123

BYL719a (Piqray® US)PIK3CA mutant HR+, HER2 (-)

postmenopausal adv BC5 2nd line (+fulv)

Xiidra®a

Dry eye

Cosentyx® EUnr-axSpA

Xolair USNasal Polyps

New molecule

New indication

New formulation

Biosimilars

BYL719PIK3CA-related overgrowth spectrum



http://deis.novartis.intra/deis.asp?VAM627A





http://deis.novartis.intra/deis.asp?LBS834A

http://deis.novartis.intra/deis.asp?FTY720D





http://deis.novartis.intra/deis.asp?VAM627A








http://deis.novartis.intra/deis.asp?DNK333B



http://deis.novartis.intra/deis.asp?RFB002D



http://deis.novartis.intra/deis.asp?AHT956A




http://deis.novartis.intra/deis.asp?DNK333B

References


Slide No. Reference

Slide 8 -

Cosentyx®

1. Non-radiographic axial spondyloarthritis (nr-axSpA) and ankylosing spondylitis (AS) are part of the axial spondyloarthritis (axSpA) spectrum.

2. DRG Epidemiology database - axSpA: release Dec’18; PsA: Nov’18; RA: Jan’19. Patients on biologics: PsA and AS - Calculated Patient equivalent based on IQVIA Midas

volume Dec’18, Indication split IQVIA medical data Dec’18; nr-axSpA - DRG disease landscape forecast release Dec’18, RA: Corrona Study 2017; Reference for US data:

Epidemiology - axSpA: DRG (Prevalence), Optiref Study (Diagnosis Rate), Jefferies (Treatment Rate) December 2018; PsA: Jefferies (Prevalence & Diagnosis Rate),

Treatment Rate (Jefferies & DRG) December 2018.

3. Out of patients treated with systemics.

Slide 16 -

Mayzent®1. Largest trial performed in SPMS; KapposL et al. Siponimod vs. placebo in SPMS: double-blinded randomized, Ph3. The Lancet. 2018; DOI 10.1016/S0140-6736(18)30475-6

2. Benedict et al. Effect of Siponimod on cognition in patients with SPMS: Ph3 EXPAND study subgroup analyses. AAN 2019. P2-051

3. Novartis QuickPulse HCP Tracker, reported by InCrowd Inc. fielded May 3-15, 2019

4. Spherix Global Insights RealTime Dynamix – MS Q219

Clinical Trials Update Includes selected ongoing or recently concluded global trials of Novartis development programs/products which are

in confirmatory development or marketed (typically Phase 2 or later).

For further information on all Novartis clinical trials, please visit: www.novartisclinicaltrials.com

Key changes vs. Q2 2019 presentationNew trials added

Study Program Indication Phase Patients

NCT03867201 DRAGON (CAMG334A2304) Aimovig® Migraine Phase 3 550

NCT03781414 CONTRAIL I (CCFZ533A2202) CFZ533 Kidney transplantation Phase 2 128

NCT03905525 TWINSS (CCFZ533B2201) CFZ533 Sjögren's syndrome Phase 2B 260

NCT03974100 (CGP24112301) GP2411 Osteoporosis Phase 3 522

NCT04097821 ADORE (CINC424H12201) Jakavi® Myelofibrosis Phase 1/2 130

NCT04072887 (CQBW251B2201) QBW251 Chronic obstructive pulmonary disease (COPD) Phase 2 900

NCT03802630 RAPTOR (CRTH258C2301) RTH258 Retinal vein occlusion Phase 3 500

NCT03810313 RAVEN (CRTH258C2302) RTH258 Retinal vein occlusion Phase 3 750

NCT03917472 (CRTH258B2305) RTH258 Diabetic macular edema Phase 3 500

NCT04058067 KINGLET (CRTH258B2304) RTH258 Diabetic macular edema Phase 3 268

39 | Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation

Trials removed (operational decision-points achieved)

Study Program Indication Phase Patients

NCT02565511 GENERATION S1 (CAPI015A2201J) CNP520 Alzheimer’s disease Phase 2B/3 1,340

NCT01544595 (CAIN457A2302E1 – extension study) Cosentyx® Psoriasis Phase 3 1,146

NCT02748863 ALLURE (CAIN457A2323) Cosentyx® Psoriasis Phase 3 214

NCT02826603 CLARITY (CAIN457A2326) Cosentyx® Psoriasis Phase 3B 1,102

NCT03512197 UNIFY (CPKC412E2301) Rydapt® Acute myeloid leukemia Phase 3 502

Cardiovascular, Renal and Metabolic

Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)

41

Study NCT02678312 PANORAMA HF (CLCZ696B2319) NCT03785405 (CLCZ696B2319E1 – extension study)

Indication Heart failure in pediatric patients Heart failure in pediatric patients

Phase Phase 2/3 Phase 3

Patients 360 240

Primary Outcome

Measures

Part 1: Pharmacodynamics and pharmacokinetics of

sacubitril/valsartan LCZ696 analytes

Part 2: Efficacy and safety compared with enalapril

Number of participants with Adverse Events (AEs) and

Serious Adverse Events (SAEs)

Arms/Intervention

• Part 1: Sacubitril/valsartan 0.8 mg/kg or 3.1 mg/kg or

both; 0.4 mg/kg or 1.6 mg/kg or both (single doses).

• Part 2: enalapril/placebo 0.2 mg/kg bid (ped. formulation

1mg/ml) and adult formulation (2.5, 5, 10 mg bid);

Sacubitril/valsartan (LCZ696)/placebo: Ped. formulation

granules (12.5, 31.25 mg in capsules); liquid formulation

(1mg/ml and 4mg/ml concentration) and adult

formulation (50, 100, 200 mg bid)

• Single arm, open label sacubitril/valsartan (pediatric

formulation granules (12.5, 31.25 mg in capsules); liquid

formulation (1mg/ml and 4mg/ml concentration) and

adult formulation (50, 100, 200 mg bid))

Target Patients

Pediatric patients from 1 month to < 18 years of age with

heart failure due to systemic left ventricle systolic

dysfunction

Pediatric patients with heart failure due to systemic left

ventricle systolic dysfunction who have completed study

CLCZ696B2319

Expected Completion

2021; (Analysis of 110 pts from Part 2 formed the basis for

pediatric submission in Apr-2019 and approval by the US

FDA in Oct-2019 for the treatment of symptomatic HF with

systemic left ventricular systolic dysfunction in children aged

1 year and older)

2022

Publication TBD TBD



42

Study NCT02554890 PIONEER-HF (CLCZ696BUS01) NCT02661217 TRANSITION (CLCZ696B2401)

Indication Heart failure, reduced ejection fraction Heart failure, reduced ejection fraction

Phase Phase 3B/4 Phase 4

Patients 881 1,002

Primary Outcome

Measures

Percentage change from baseline in N-terminal pro-brain

natriuretic peptide (NT-proBNP)

Assessing the percentage of patients who achieve the target

dose of 200 mg bid LCZ696 at 10 weeks after

randomization

Arms/Intervention

• Sacubitril/valsartan (LCZ696) 24/26 mg, 49/51 mg or

97/103 mg bid or matching placebo

• Enalapril (2.5 mg, 5 mg, and 10 mg) bid or matching

placebo

• Pre-discharge treatment initiation - LCZ696 (50, 100,

200 mg bid)

• Post-discharge treatment initiation - LCZ696 (50, 100,

200 mg bid)

Target PatientsPatients with HFrEF (LVEF<40%) hospitalized for ADHF

and stable for more than 24 hours

Heart failure patients with reduced ejection-fraction

hospitalized for an acute decompensation event

Expected Completion Q3-2018 (actual) Q4-2018 (actual)

Publication

• Mar-2019 ACC: 4wk OLE data, and core study data on

biomarkers, de novo HF, hospitalizations, & prior

exposure

• Apr-2019 Circulation: Research letter on composite

endpoint (Circulation. 2019;139:00–00)

• Q3-2019 ESC: Secondary abstracts submitted

• Planned in Q1-2020: RAAS naive and de novo HF

• 28-May-2019 TRANSITION primary publication -

published EJHF

• Secondary data presentations: de novo HF and

ACEi/ARB naive sub-groups presented at ESC-HF

Congress in May 2019 and submitted to EJHF in Jun-

2019 (expected publication Q3-2019)

• Planned in Q4-2019: 26-weeks data presentation at

ESC-2019 in Paris


43

Study NCT02884206 PERSPECTIVE (CLCZ696B2320) NCT02468232 PARALLEL-HF (CLCZ696B1301)

Indication Heart failure Heart failure, reduced ejection fraction

Phase Phase 3 Phase 3

Patients 592 225

Primary Outcome

Measures

Change from baseline in the CogState Global Cognitive

Composite Score (GCCS)

Time to the first occurrence of the composite endpoint -

either cardiovascular (CV) death or heart failure (HF)

hospitalization

Arms/Intervention

• Sacubitril/valsartan 50, 100, and 200 mg bid with

placebo of valsartan

• Valsartan 40, 80, and 160 mg bid tablets with placebo

for sacubitril/valsartan

• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo

of enalapril

• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of

sacubitril/valsartan

Target PatientsPatients with chronic heart failure with preserved ejection

fraction

Japanese heart failure patients (NYHA Class II-IV) with

reduced ejection fraction

Expected Completion 2022 Q1-2019 (actual); H2-2020 (open-label extension)

Publication TBDPlanned in H1-2020: Core study primary manuscript in Circ

J



44

Study NCT01920711 PARAGON-HF (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)

Indication Heart failure, preserved ejection fraction Heart failure, preserved ejection fraction


Patients 4,822 2,577

Primary Outcome

Measures

Cumulative number of primary composite events of

cardiovascular (CV) death and total (first and recurrent) HF

hospitalizations

Change in NT-proBNP from baseline to week 12

and change in 6 minute walk distance (6MWD) from

baseline to Week 24

Arms/Intervention

• Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200

mg bid

• Valsartan or placebo 40 mg, 80 mg, and 160 mg bid

• Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and

matching placebo

• Enalapril 2.5 mg, 5 mg and 10 mg bid and matching

placebo

• Valsartan 40 mg, 80 mg, 160 mg bid and matching

placebo

Target PatientsHeart failure patients (NYHA Class II-IV) with preserved

ejection fraction

Heart failure patients (NYHA Class II-IV) with preserved

ejection fraction

Expected Completion Q3-2019 (actual) Q4-2019

Publication

• Sep-2019: Primary manuscript published (Angiotensin–

Neprilysin Inhibition in Heart Failure with Preserved

Ejection Fraction. Solomon S et al; NEJM. DOI:

10.1056/NEJMoa1908655)

• Sep-2019: ESC: Late breaker presentation of primary

results

TBD



45

Study NCT03909295 (CLCZ696D1301E1 – extension study) NCT02924727 PARADISE-MI (CLCZ696G2301)

Indication Heart failure chronic Post-acute myocardial infarction


Patients 63 5,650

Primary Outcome

Measures

Number of participants with Adverse Events (AEs) and

Serious Adverse Events (SAEs)

Time to the first occurrence of a confirmed composite

endpoint (cardiovascular (CV) death, heart failure (HF)

hospitalization, or outpatient heart failure)

Arms/Intervention• Sacubitril/valsartan 50 mg,100 mg,200 mg film coated

tablets

• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid / placebo

of ramipril/valsartan

• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of

sacubitril/valsartan / placebo for valsartan

Target Patients

Japanese heart failure patients (NYHA Class II-IV) with

preserved ejection fraction after CLCZ696D2301

(PARAGON-HF)

Post-AMI patients with evidence of LV systolic dysfunction

and/or pulmonary congestion, with no known prior history of

chronic HF

Expected Completion 2021 H2-2020

Publication TBD TBD



Immunology, Hepatology & Dermatology

CFZ533 – Blocking, non-depleting, Fc-silent, anti-CD40 monoclonal antibody

Study NCT03663335 (CCFZ533A2201) NCT03905525 TWINSS (CCFZ533B2201)

Indication Kidney transplantation Sjögren's syndrome

Phase Phase 2B Phase 2B

Patients 325 260

Primary Outcome

Measures

Composite event (BPAR, Graft Loss or Death) over 12

months post-transplantation and post conversion (for

maintenance cohort)

Change in EULAR Sjögren’s syndrome Disease Activity

Index (ESSDAI) score and EULAR Sjögren’s syndrome

Patient Reported Index (ESSPRI) score

Arms/Intervention• CFZ533 doses + MMF + corticosteroids

• Control/Standard of Care: TAC + MMF + corticosteroids

• CFZ533 doses

• Placebo

Target Patients Kidney transplant recipients Patients with Sjögren's syndrome

Expected Completion 2021 2022

Publication TBD TBD


47

CFZ533 – Blocking, non-depleting, Fc-silent, anti-CD40 monoclonal antibody

Study NCT03781414 CONTRAIL I (CCFZ533A2202)

Indication Kidney transplantation

Phase Phase 2

Patients 128

Primary Outcome

Measures

Proportion of patients with composite event (BPAR, Graft

Loss or Death) over 12 months

Arms/Intervention

• Control/Standard of Care: TAC + MMF + Corticosteroids

• CFZ533 dose A + MMF + Corticosteroids

• CFZ533 dose B + MMF + Corticosteroids

Target Patients Liver transplant recipients

Expected Completion 2022

Publication TBD


48

Cosentyx® - Anti IL-17

49

Study NCT03504852 (CAIN457A2324) NCT03589885 MATURE (CAIN457A2325)

Indication Psoriasis Psoriasis

Phase Phase 3B Phase 3

Patients 331 122

Primary Outcome

Measures

PASI 90 response and IGA mod 2011 0 or 1 response after

16 weeks of treatment

PASI 75 response and IGA mod 2011 0 or 1 response after

12 weeks of treatment

Arms/Intervention

• Secukinumab 300 mg every 2 weeks after weekly doses

till Week 4

• Secukinumab 300 mg every 4 weeks after weekly doses

till Week 4

• Secukinumab 2 mL (300 mg) auto-injector

• Secukinumab 2 x 1 mL (150 mg each) prefilled syringe

• Placebo 2 mL auto-injector

• Placebo 2 x 1 mL prefilled syringe

Target Patients Subjects (≥90kg) with moderate to severe plaque psoriasis Subjects with moderate to severe plaque psoriasis

Expected Completion H2-2020 H2-2020

Publication TBD TBD



50

Study NCT02471144 (CAIN457A2310) NCT03668613 (CAIN457A2311)

Indication Psoriasis Psoriasis


Patients 162 84

Primary Outcome

Measures

Psoriasis Area and Severity Index (PASI) 75 response and

Investigators' Global Assessment (IGA) 0 or 1 response at

week 12



week 12

Arms/Intervention

• Secukinumab low dose

• Secukinumab high dose

• Placebo

• Etanercept (comparator)

• Secukinumab low dose

• Secukinumab high dose

Target PatientsPatients from 6 to less than 18 years of age with severe

chronic plaque psoriasis

Pediatric patients of age 6 to <18 years, with moderate to

severe plaque psoriasis


Publication TBD TBD



51

Study NCT03066609 (CAIN457A2318)

Indication Psoriasis

Phase Phase 3

Patients 543

Primary Outcome

Measures



week 12

Arms/Intervention

• Secukinumab 300 mg


• Placebo

Target PatientsPatients with moderate to severe chronic plaque-type

psoriasis with or without psoriatic arthritis comorbidity

Expected Completion Q1-2019 (actual)

Publication

• Week 16 results: Poster presented at: 2019 American

Academy of Dermatology (AAD) Annual Meeting,

• March 1–5, 2019, Washington, D.C.

• 52-week results: Poster at EADV 2019, Madrid 9-13

October, 2019



52

Study NCT03031782 (CAIN457F2304) NCT03769168 (CAIN457F2304E1 – extension study)

Indication Psoriatic arthritis Psoriatic arthritis


Patients 80 64

Primary Outcome

MeasuresTime to 33 flares Number of participants with JIA ACR30 response

Arms/Intervention• Secukinumab (pre-filled syringe) 75 mg

• Placebo

• Secukinumab 75 mg/0.5 ml

• Secukinumab 150 mg/1.0 ml

Target PatientsJuvenile idiopathic arthritis subtypes of psoriatic and

enthesitis-related arthritis

Patients with juvenile idiopathic arthritis subtypes of juvenile

psoriatic arthritis and enthesitis related arthritis


Publication TBD TBD



53

Study NCT01892436 FUTURE 1 extension (CAIN457F2306E1) NCT01649375 MEASURE 2 (CAIN457F2310)

Indication Psoriatic arthritis Ankylosing spondylitis


Patients 460 219

Primary Outcome

Measures

Proportion of subjects that have a positive clinical response

to treatment (individual improvement) in disease activity

according to ACR20 (or ACR50 or ACR 70)

Assessment of SpondyloArthritis International Society /

ASAS 20 response

Arms/Intervention• Secukinumab 75 mg




• Placebo

Target Patients Patients with active psoriatic arthritis Patients with active ankylosing spondylitis


Publication

• 3 year results: ACR 2016; Mease PJ et al. Arthritis

Rheumatol. 2016; 68 (suppl 10)

• 3 years results: Manuscript published in September

2018 (Mease PJ, et al. RMD Open 2018;4:e000723.

doi:10.1136/rmdopen-2018-000723)

• 5 year results: accepted for publication in ACR open

Rheumatology in September 2019

• Primary 52 week results: Baeten D & Sieper J, et al. N

Engl J Med 2015;373:2534–48

• 2 year results: Marzo-Ortega, et al. Arthritis Care Res

2017 Feb 24. doi: - 10.1002/acr.23233

• 3 year results: Marzo-Ortega, et al. RMD 2017

• 5 year results: EULAR 2019; Marzo-Ortega H, et al.

FRI0379. Annals of the Rheumatic Diseases

2019;78:873.

• 5 year results; manuscript target submission Oct 2019



54

Study NCT01752634 FUTURE 2 (CAIN457F2312) NCT02008916 MEASURE 3 (CAIN457F2314)



Patients 399 222

Primary Outcome

Measures

Proportion of subjects achieving American College of

Rheumatology 20 (ACR20) response criteria

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 response

Arms/Intervention

• Secukinumab (AIN457) 150 mg s.c.



• Placebo s.c.

• Secukinumab 10 mg/kg / 300 mg

• Secukinumab 10 mg/kg / 150 mg

• Placebo



Publication

• Primary results: McInnes IB, et al. Lancet.

2015;386:1137–46

• 2 years results: McInnes et al, Rheumatology

2017;56:1993-2003

• 5 year (EOS) manuscript ongoing; to be submitted in

Oct-2019

• 16 weeks results: PANLAR congress in Apr-2016

• 52 weeks results: Pavelka et al. Arthritis Research &

Therapy 2017

• 2 year results: Presented at ACR in Nov-2017

• 3 year (EOS) results: To be presented (ORAL) at

PANLAR April 2019

• 3 year (EOS) manuscript submitted in May-2019;

awaiting journal decision



55

Study NCT01989468 FUTURE 3 (CAIN457F2318) NCT02159053 MEASURE 4 (CAIN457F2320)



Patients 416 350

Primary Outcome

Measures

American College of Rheumatology 20 (ACR20) response in

subjects treated with secukinumab vs. placebo

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 at week 16

Arms/Intervention



• Placebo

• Secukinumab 150 mg s.c. with loading

• Secukinumab 150 mg s.c. without loading

• Placebo



Publication52 week results: Nash et al, Arthritis Research & Therapy

2018, 20:47

• Week 104 (EOS) manuscript: Kivitz et al, Rheumatol

Ther https://doi.org/10.1007/s40744-018-0123-5


https://doi.org/10.1007/s40744-018-0123-5


56

Study NCT02294227 FUTURE 4 (CAIN457F2336) NCT02404350 FUTURE 5 (CAIN457F2342)

Indication Psoriatic arthritis Psoriatic arthritis


Patients 342 990

Primary Outcome

Measures

Assessment of American College of Rheumatology 20

(ACR20)

American College of Rheumatology 20 (ACR20) response at

Week 16

Arms/Intervention

• Secukinumab 150 mg with loading

• Secukinumab 150 mg without loading

• Placebo

• Secukinumab 150 mg load

• Secukinumab 150 mg no load


• Placebo

Target Patients Patients with active psoriatic arthritis Patients with active psoriatic arthritis


Publication

• 52 week results: abstract presented at PANLAR

congress (Apr-2018)

• 2 year (EOS) results published: Rheumatology

Therapy. 2019 Jun 21. doi: 10.1007/s40744-019-0163-5.

• 24 week results published: Mease P, et al. Annals of the

Rheumatic Diseases 2018;77:890-897.

• 52 week results presented at EULAR and ACR 2018

• 52 week manuscript accepted (Rheumatology, October

2019, in press)

• 2 year (EOS) results presented at EULAR 2019



57

Study NCT01863732 (CAIN457F2305E1 – extension study) NCT02745080 EXCEED (CAIN457F2366)

Indication Ankylosing spondylitis Psoriatic arthritis


Patients 300 850

Primary Outcome

Measures

Assessment of spondyloarthritis international society criteria

/ ASAS 20 responseAmerican College of Rheumatology 20 (ACR20) response

Arms/Intervention• Secukinumab 75 mg in PFS

• Secukinumab 150 mg in PFS

• Secukinumab 300 mg s.c.

• Adalimumab 40 mg s.c.

Target Patients Patients with active ankylosing spondylitis Patients with active psoriatic arthritis

Expected Completion Q2-2018 (actual) H1-2020

Publication

• 3-year results: Manuscript published in Clinical and

Experimental Rheumatology in May-2017

• 4-year results: Presented at ACR in Nov-2017

• 4 year results manuscript published; Rheumatology,

Volume 58, Issue 5, May 2019, Pages 859–868,

• 5 year (EOS) results manuscript published; Baraliakos X,

et al. RMD Open 2019;5:e001005. doi:

10.1136/rmdopen-2019-001005

• Manuscript target submission January 2020



58

Study NCT02696031 PREVENT (CAIN457H2315) NCT03259074 SURPASS (CAIN457K2340)

Indication Non-radiographic axial spondyloarthritis Ankylosing spondylitis


Patients 555 837

Primary Outcome

Measures

The proportion of participants who achieved an ASAS 40

response (Assessment of SpondyloArthritis International

Society criteria);

No radiographic structural progression as measured by

modified Stoke Ankylosing Spondylitis Spine Score

(mSASSS)

Arms/Intervention


• Secukinumab 150 mg no load

• Placebo

• Secukinumab 150/300 mg

• Adalimumab biosimilar 40 mg

Target Patients Patients with non-radiographic axial spondyloarthritis Patients with active ankylosing spondylitis

Expected Completion Week 52: Q3-2019 (actual); Final: 2021 2022

Publication

• Abstract (16 week results) submitted as a late breaker to

ACR 2019

• Manuscript target submission Jan-2020

TBD



59

Study NCT03713619 SUNSHINE (CAIN457M2301) NCT03713632 SUNRISE (CAIN457M2302)

Indication Hidradenitis Suppurativa (HS) Hidradenitis Suppurativa (HS)


Patients 471 471

Primary Outcome

Measures

Proportion of participants with Hidradenitis Suppurativa

clinical response (HiSCR)

Proportion of patients with Hidradenitis Suppurativa Clinical

Response (HiSCR)

Arms/Intervention

• Secukinumab 300 mg every 2 weeks


• Placebo (every 2 weeks)






Target Patients Subjects with moderate to severe Hidradenitis Suppurativa Subjects with moderate to severe Hidradenitis Suppurativa


Publication Preliminary results in EADV (most likely) in 2021 Preliminary results in EADV (most likely) in 2021


Ilaris® - Anti IL-1β

60

Study NCT02059291 CLUSTER (CACZ885N2301) NCT02296424 (CACZ885G2306)

Indication Hereditary periodic fevers SJIA - Systemic Juvenile Idiopathic Arthritis

Phase Phase 3 Phase 3B/4

Patients 203 182

Primary Outcome

Measures

To demonstrate significant reduction of disease activity

with canakinumab vs. placebo

Proportion of patients in clinical remission on canakinumab

who are able to remain in remission following canakinumab

dose tapering (reduced canakinumab dose or prolonged

canakinumab dosing interval)

Arms/Intervention• Canakinumab

• Placebo

• Canakinumab dose reduction

• Canakinumab dose interval prolongation

Target PatientsPatients with, 3 separate disease cohorts TRAPS, HIDS,

and colchicine resistant FMF (Hereditary periodic fevers )

Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)

(Pediatric)

Expected Completion 2017 (actual) 2018 (actual)

Publication

• QoL overall presented at EULAR in Q2-2019 and will

be presented at ACR in Q4-2019

• Planned manuscripts: QoL overall to be submitted in

Q4-2019 and crFMF efficacy & safety in Q2-2020

• Remission & flexible dosing – presented at ISSAID & EULAR in Q2-2019

• Planned manuscript in 2019: Remission & flexible dosing to be submitted in Q4-2019


LJN452 - FXR Agonist

61

Study NCT02855164 (CLJN452A2202)

Indication Non-alcoholic steatohepatitis

Phase Phase 2

Patients 345

Primary Outcome

Measures

Adverse event profile of different doses; determine the dose

relationship of LJN452 on markers of hepatic inflammation

in NASH (ALT and AST); determine dose-response

relationship of LJN452 on liver fat content by changes in

quantitative MRI; determine effect of LJN452 on liver fibrosis

by biopsy

Arms/Intervention Multiple LJN452 doses and placebo

Target Patients Patients with non-alcoholic steatohepatitis (NASH)

Expected Completion H1-2020

Publication

• Primary (interim) data abstract submitted to AASLD in

Q3-2019

• Manuscript to be submitted in H2-2020


LOU064 – Bruton's tyrosine kinase (BTK) inhibitor

62

Study NCT03926611 (CLOU064A2201)

Indication Chronic spontaneous urticaria (CSU)

Phase Phase 2

Patients 308

Primary Outcome

MeasuresChange from baseline in weekly Urticaria Activity Score (UAS7) at Week 4

Arms/Intervention

• LOU064 Arm 1 Low dose of LOU064 orally in the morning (once daily) and

matching placebo in the evening from Day 1 to 85

• LOU064 Arm 2 Medium dose of LOU064 orally in the morning (once daily) and


• LOU064 Arm 3 High dose of LOU064 orally in the morning (once daily) and


• LOU064 Arm 4 Low dose of LOU064 orally, twice daily from Day 1 to 85

• LOU064 Arm 5 Medium dose of LOU064 orally, twice daily from Day 1 to 85

• LOU064 Arm 6 High dose of LOU064 orally, twice daily from Day 1 to 85

• Placebo arm Matching placebo, orally, twice daily from Day 1 to 85

Target Patients Adults with CSU inadequately controlled by H1-antihistamines


Publication TBD


LJC242 - FXR agonist + CCR2/CCR5 inhibitor

63

Study NCT03517540 TANDEM (CLJC242A2201J)

Indication Non-alcoholic steatohepatitis

Phase Phase 2

Patients 200

Primary Outcome

Measures

• Evaluation of safety and tolerability of combination

therapy (tropifexor + cenicriviroc) by monitoring adverse

event profile, vital signs and laboratory parameters

Arms/Intervention

• Tropifexor

• Cenicriviroc

• Tropifexor + cenicriviroc

Target PatientsAdult patients with non-alcoholic steatohepatitis (NASH) and

liver fibrosis


Publication Manuscript to be submitted in H1-2021


QGE031 - Anti-IgEStudy NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)

Indication Chronic spontaneous urticaria / Chronic idiopathic urticaria Chronic spontaneous urticaria / Chronic idiopathic urticaria

Phase Phase 2B Phase 2B

Patients 382 226

Primary Outcome

Measures

Establish dose-response relationship of QGE031 with

respect to achievement of complete hives response at week

12

Long-term safety; number of participants with treatment-

emergent adverse events

Arms/Intervention

• Ligelizumab 24mg q4wks for 20 weeks



• Ligelizumab 120mg single dose

• Omalizumab 300mg q4wks for 20 weeks

• Placebo q 4wks for 20 weeks

Ligelizumab 240 mg q4wks open label for 52 weeks

Target Patients

Adult patients with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines at approved

or increased doses, alone or in combination with H2-

antihistamines or leukotriene receptor antagonists.

Adult patients with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines at approved

or increased doses, alone or in combination with H2-

antihistamines or leukotriene receptor antagonists.

Expected Completion 2017 (actual) Q3-2019 (actual)

Publication

• Primary results: Presented at EAACI 2018, EADV 2018,

and GUF 2018; NEJM publication (Oct. 3rd);

• Secondary results presented in 2019 at: AAD, EAACI,

WCD, EADV, PAAM, ACAAI, UCARE.

• Primary results: AAD 2019;

• Secondary results presented in 2019 at: AAD, EAACI,

WCD, EADV, PAAM, ACAAI, UCARE; manuscript

planned in H1/2020


64

QGE031 - Anti-IgE

Study NCT03437278 (CQGE031C2202)

Indication Chronic spontaneous urticarial / Chronic idiopathic urticaria

Phase Phase 2

Patients 48

Primary Outcome

MeasuresChange in the 7 day Urticaria Activity Score (UAS7)

Arms/Intervention

• Ligelizumab high dose q4wks for 24 weeks

• Ligelizumab low dose q4wks for 24 weeks

• Placebo / ligelizumab high dose q4wks for 8 / 16 weeks

Target PatientsAdolescents from 12 to <18 years of age, with chronic

spontaneous urticaria


Publication• Study design to be presented at PAAM 2019,

• Manuscript to be submitted in 2022


65

QGE031 - Anti-IgE

Study NCT03580369 Pearl 1 (CQGE031C2302) NCT03580356 Pearl 2 (CQGE031C2303)

Indication Chronic spontaneous urticaria Chronic spontaneous urticaria



Primary Outcome

Measures

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Arms/Intervention

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

Target PatientsAdolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines

Adolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines


Publication• Study design presented at UCARE 2018


• Study design presented at UCARE 2018



66

VAY736 – Fully human IgG1/κ anti-BAFF-R mAb

Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)

Indication Primary Sjögren's syndrome Autoimmune hepatitis

Phase Phase 2B Phase 2/3

Patients 180 80

Primary Outcome

Measures

Safety and efficacy of VAY736 in primary Sjögren's

syndrome (pSS)Alanine aminotransferase (ALT) normalization

Arms/Intervention• VAY736

• Placebo

• VAY736

• Placebo control with conversion to active VAY736

Target PatientsPatients with moderate to severe primary Sjögren's

syndrome (pSS)

Autoimmune hepatitis patients with incomplete response or

intolerant to standard treatment of care

Expected Completion H2-2020 2023

Publication

• Late Breaking Abstract to be submitted to American

College of Rheumatology 30-Sep-2019


TBD


67

ZPL389 - H4 receptor antagonist

Study NCT03517566 ZEST (CZPL389A2203)NCT03948334 ZESTExt (CZPL389A2203E1 – extension

study)

Indication Atopic dermatitis Atopic dermatitis


Patients 360 360

Primary Outcome

MeasuresIGA (Investigator's global assessment) response at week 16

Frequency of Adverse Events (AEs) and Serious Adverse

Events (SAEs)

Arms/Intervention

• ZPL389 dose 1

• ZPL389 dose 2

• ZPL389 dose 3

• ZPL389 dose 4

• Placebo

• ZPL389 Dose 1 + Topical Corticosteroids (TCS) and /or

Topical Calcineurin Inhibitors (TCI)

• ZPL389 Dose 2 + Topical Corticosteroids (TCS) and /or

Topical Calcineurin Inhibitors (TCI)

Target Patients Patients with moderate to severe atopic dermatitis Adult patients with atopic dermatitis


Publication TBD TBD


68

Neuroscience

Zolgensma® - SMN1 gene replacement therapy

70

Study NCT03461289 STRIVE-EU (CL-302) NCT03306277 STRIVE (CL-303)

Indication Type 1 spinal muscular atrophy Type 1 spinal muscular atrophy


Patients 30 20

Primary Outcome

MeasuresProportion of participants sitting without support

• Achievement of independent sitting for at least 30

seconds

• Event-free survival

Arms/Intervention Open-label, single-arm, single-dose, intravenous Open-label, single-arm, single-dose, intravenous

Target Patients Patients with spinal muscular atrophy Type 1 Patients with Spinal Muscular Atrophy Type 1

Expected Completion H2-2020 Q4-2019

Publication TBD TBD



71

Study NCT03505099 SPR1NT (CL-304) NCT03837184 STRIVE Asia Pacific (CL-306)

Indication Spinal muscular atrophy Type 1 spinal muscular atrophy


Patients 27 6

Primary Outcome

Measures

• Percentage of participants achieving functional

independent sitting for at least 30 seconds at any visit

• Percentage of participants achieving the ability to stand

without support for at least 3 seconds at any visit

Proportion of participants sitting without support

Arms/Intervention Open-label, single-arm, single-dose, intravenous Open-label, single-arm, single-dose, intravenous

Target PatientsPre-symptomatic patients with spinal muscular atrophy and

multiple copies SMN2Patients with spinal muscular atrophy Type 1


Publication TBD TBD



72

Study NCT03381729 STRONG (CL-102)

Indication Type 2 spinal muscular atrophy

Phase Phase 1

Patients 27

Primary Outcome

Measures

• Safety and tolerability, incidence of adverse events

• Proportion of patients achieving Standing Milestone

• Change in Hammersmith Functional Motor Scale

Arms/Intervention Open-label, single-arm, single-dose, intrathecal

Target Patients Patients with spinal muscular atrophy with 3 copies of SMN2

Expected Completion Q4-2019

Publication TBD


Aimovig® – CGRP receptor antagonist

73

Study NCT03096834 LIBERTY (CAMG334A2301) NCT03333109 EMPOWER (CAMG334A2302)

Indication Migraine Migraine


Patients 246 880

Primary Outcome

Measures

Percentage of patients with a 50% response in the reduction

of Monthly Migraine Days (MMD)

Change from baseline in monthly migraine days at the last

month (Month 3) of the double-blind treatment period

Arms/Intervention• Subcutaneous injection of AMG334 (erenumab)

• Subcutaneous injection of placebo

• AMG334 (erenumab) Dose 1

• AMG334 (erenumab) Dose 2

• Placebo

Target PatientsAdult episodic migraine patients who have failed prophylactic

migraine treatmentsAdult episodic migraine patients

Expected Completion 2017 DBT phase (actual); 2021 OLE phase H2-2020

Publication

• Planned for Q4-2019 - PROs and prespecified subgroup

analysis (double blind phase)

• Planned for Q1-2020: 1Y OLE

• Planned for Q4 2020: 2Y OLE

Planned for H2-2020


Aimovig® – CGRP receptor antagonist

74

Study NCT03867201 DRAGON (CAMG334A2304)

Indication Migraine

Phase Phase 3

Patients 550

Primary Outcome

Measures

Change from baseline in monthly migraine days during the

last 4 weeks of the 12-week treatment period

Arms/Intervention• Subcutaneous injection of AMG334 (erenumab) 70 mg

• Subcutaneous injection of placebo

Target Patients Adult chronic migraine patients

Expected Completion 2022 DBT phase; 2024 OLE phase

Publication Planned in Q4-2023 (DBT)


Gilenya® - S1P-R modulator

75

Study NCT01633112 ASSESS (CFTY720D2312) NCT01201356 LONGTERMS (CFTY720D2399)

Indication Relapsing remitting multiple sclerosis (RRMS) Relapsing multiple sclerosis (RMS)

Phase Phase 3B Phase 3


Primary Outcome

Measures

Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod

to glatiramer acetate (20 mg) in reducing the annualized

relapse rate up to 12 months

Long-term safety and tolerability

Arms/Intervention

• Fingolimod 0.5 mg orally

• Fingolimod 0.25mg orally

• Copaxone® 20 mg s.c.

Single-arm study of fingolimod 0.5 mg/day

Target Patients Patients with relapsing-remitting multiple sclerosis Patients with relapsing multiple sclerosis

Expected Completion 2018 (actual) 2018 (actual)

Publication• Primary data presentation at AAN in 2019

• Primary manuscript – submission planned in Q4-2019

• Primary data presentation: Cohen J, et al presented at

ECTRIMS 2017

• Primary manuscript accepted by Therapeutic Advances

in Neurological Disorders (estimated publication Oct-

2019)


LMI070 - SMN2 RNA splice modulator

76

Study NCT02268552 (CLMI070X2201)

Indication Type 1 spinal muscular atrophy

Phase Phase 1/2

Patients 39

Primary Outcome

Measures

Number of participants with adverse events (AEs), serious

adverse events (SAEs) and deaths

Arms/Intervention

Branaplam oral, once weekly:

• Part 1: 5 ascending doses

• Part 2: 2 different dose levels

• Part 3: patients continue on initial dose assigned in Part

1 or Part 2

Target PatientsPatients with type 1 spinal muscular atrophy


Publication TBD


Mayzent® - S1P-R modulator

77

Study NCT01665144 -EXPAND (CBAF312A2304)

Indication Secondary progressive multiple sclerosis

Phase Phase 3

Patients 1,652

Primary Outcome MeasuresThe delay in time to confirmed disability progression as

measured by EDSS (Expanded Disability Status Scale)

Arms/Intervention

• BAF312 (5-day titration: 0.25mg to 1.25mg; Maintenance

dose: 2mg (day 6))

• Placebo

Target Patients Patients with secondary progressive multiple sclerosis

Expected Completion Core in 2016/Extension in 2023

PublicationThe Lancet Neurology, Volume 39, No.10127, p1237-1330,

March 2018


OMB157 - Anti-CD20

78

Study NCT02792218 Asclepios I (COMB157G2301) NCT02792231 Asclepios II (COMB157G2302)

Indication Multiple sclerosis Multiple sclerosis


Patients 900 900

Primary Outcome

Measures

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

of relapses by patient adjusted for time-in-study by patient

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

of relapses by patient adjusted for time-in-study by patient

Arms/Intervention• Ofatumumab subcutaneous

• Teriflunomide oral

• Ofatumumab subcutaneous

• Teriflunomide oral

Target Patients Patients with relapsing forms of multiple sclerosis Patients with relapsing forms of multiple sclerosis


Publication Primary manuscript planned in H1-2020 Primary manuscript planned in H1-2020


OMB157 - Anti-CD20

79

Study NCT03249714 APOLITOS (COMB157G1301) NCT03650114 ALITHIOS (COMB157G2399)

Indication Multiple sclerosis Multiple Sclerosis


Patients 60 2010

Primary Outcome

Measures

Reduced cumulative number of Gd-enhanced T1 lesions

across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab

vs placebo)

Evaluate the long-term safety and tolerability of ofatumumab

20 mg subcutaneous (sc) once every 4 (q4) weeks in

subjects with RMS from the first dose of ofatumumab

Arms/Intervention• Ofatumumab 20 mg subcutaneous injections

• Placebo• Ofatumumab 20 mg every 4 weeks

Target Patients Patients with relapsing forms of multiple sclerosis Patients with relapsing MS


Publication TBD TBD


Oncology

ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor

81

Study NCT03106779 (CABL001A2301) NCT03578367 (CABL001E2201)

Indication Chronic myeloid leukaemia (CML) Chronic myeloid leukaemia (CML)


Patients 222 120

Primary Outcome

MeasuresMajor Molecular Response (MMR) rate at 24 weeks Deep molecular response (MR 4.5) at 48 weeks

Arms/Intervention• ABL001 40 mg bid

• Bosutinib 500 mg

• ABL001 40 mg QD + 400 mg imatinib

• ABL001 60 mg QD + 400 mg imatinib

• Imatinib 400mg QD (continuation treatment)

• Nilotinib 300mg bid (switch to nilotinib treatment)

Target Patients

Patients with chronic myelogenous leukemia in chronic

phase, previously treated with 2 or more tyrosine kinase

inhibitors

CML-CP patients not reaching DMR (MR 4.5) while on 1L

imatinib treatment


Publication Manuscript submission in H2-2020 (journal TBD) TBD


ACZ885 – IL1β inhibitor


Study NCT03447769 (CACZ885T2301) NCT03631199 CANOPY-1 (CACZ885U2301)

Indication Adjuvant NSCLC 1st Line Non-small cell lung cancer (NSCLC)


Patients 1,500 627

Primary Outcome

Measures

Disease free survival (primary), overall survival (key

secondary)

• Safety run-in part: Incidence of dose limiting toxicities

• Double-blind, randomized, placebo-controlled part:

Progression free survival (PFS)

• Overall survival (OS)

Arms/Intervention• Canakinumab 200mg q3w sc for 18 cycles

• Placebo q3w sc for 18 cycles

• Canakinumab or matching placebo in combination with

pembrolizumab and platinum-based doublet

chemotherapy

Target Patients

Patients with:

• High–risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB

(T>5cm N2)) after complete resection and standard of

care adjuvant cisplatin-based chemotherapy

• All histologies

Patients with

• Histologically confirmed Stage IIIB, IV NSCLC with no

prior systemic anticancer therapy

• Squamous and non-squamous NSCLC

• No EGFR mutation and ALK rearrangement


Publication TBDJohnson B et al. Abstract accepted for presentation at

AACR-NCI-EORTC Oct 2019 (safety run-in)

ACZ885 – IL1β inhibitor


Study NCT03626545 CANOPY-2 (CACZ885V2301)

Indication 2nd / 3rd Line Non-small cell lung cancer (NSCLC)

Phase Phase 3

Patients 240

Primary Outcome

Measures

• Safety run-in part: Incidence of dose limiting toxicities.

• Double-blind, randomized, placebo-controlled part:

Overall Survival

Arms/Intervention

• canakinumab in combination with docetaxel

• canakinumab matching-placebo in combination with

docetaxel

Target Patients

Patients with:

• Stage IIIB or IV NSCLCwithout EGFR, ALK, ROS-1 or B-

RAF mutation

• Previously treated with platinum therapy and PD(L)1-

inhibitor


Publication TBD

BYL719 - Alpha-specific PI3K inhibitor

84

Study NCT02437318 SOLAR-1 (CBYL719C2301)

Indication HR+ advanced breast cancer

Phase Phase 3

Patients 572

Primary Outcome

Measures

Progression-free survival (PFS) for patients with PIK3CA

mutant status

Arms/Intervention• Fulvestrant 500 mg + alpelisib 300 mg

• Fulvestrant 500 mg + placebo

Target Patients

Men and postmenopausal women with hormone receptor

positive, HER2-negative advanced breast cancer which

progressed on or after aromatase inhibitor treatment


Publication

• Andre F, et al. Presentation at ESMO 2018

• Andre et al. Manuscript N Engl J Med 2019;380:1929-

1940.


Exjade® - Iron chelation of bis-hydroxy-phenyl triazole type

85

Study NCT00940602 TELESTO (CICL670A2302)

Indication Iron overload

Phase Phase 2

Patients 224

Primary Outcome

Measures

To compare deferasirox to placebo with regard to event-free

survival in low and int-1 risk MDS patient with transfusional

iron overload

Arms/Intervention• Deferasirox, iron chelator

• Placebo

Target PatientsPatients with myelodysplastic syndromes (low/int-1 risk) and

transfusional iron overload


Publication• Angelucci E, et al. Presentation at ASH 2018

• Angelucci E, et al. Manuscript submitted Q3-2019


INC280 - MET Inhibitor

86

Study NCT02414139 (CINC280A2201) NCT03647488 (CINC280D2201)

IndicationEGFR Wild-type, ALK negative advanced Non-small Cell

Lung Cancer (NSCLC)Non-small cell lung cancer


Patients 364 105

Primary Outcome

MeasuresOverall Response Rate (ORR)

Run in part: Assess safety and tolerability of capmatinib and

spartalizumab combination.

Randomized part: Overall Survival (OS)

Arms/Intervention

• Pre-treated pts. with MET GCN: ≥ 6; ≥ 4 and < 6; <

4

• Pre-treated pts. with MET mutations regardless of

cMET GCN as second or third line

• Treatment-naïve pts. with MET dysregulation

• Pre-treated pts with MET dysregulation – second

line

• Treatment-naïve pts with cMET mutations

regardless of cMET GCN

• Capmatinib plus spartalizumab

• Docetaxel

Target Patients

Adult patients with EGFR wild-type (wt), ALK-negative

advanced/ metastatic NSCLC with either MET

amplification or MET mutations

Pre-treated adult patients with EGFR wild-type ALK

rearrangement negative advanced/metastatic non-small cell

lung cancer, that has demonstrated progression following one

prior platinum doublet and one prior PD-(L)1 checkpoint

inhibitor

Expected Completion Q2-2019 (actual) 2021

Publication• Wolf J, et al. Presented at ASCO 2019

• Manuscript submission in H2-2019 (journal TBD)TBD


Jakavi® - JAK1/2 inhibitor

87

Study NCT02913261 REACH2 (CINC424C2301) NCT03112603 REACH3 (CINC424D2301)

Indication Steroid-refractory acute graft vs. Host disease (SR aGVHD) Steroid-refractory chronic graft vs. Host disease (SR cGVHD)


Patients 308 324

Primary Outcome

MeasuresOverall Response Rate (ORR) at 28 Days Overall Response Rate (ORR) at 183 Days

Arms/Intervention• Ruxolitinib 10mg bid

• Best available therapy (BAT)

• Ruxolitinib 10mg bid

• Best available therapy (BAT)

Target Patients Patients with steroid-refractory acute GVHD (SR aGVHD) Patients with steroid-refractory chronic GVHD (SR cGVHD)


Publication • Manuscript submission in Q4-2019 (journal TBD) • Manuscript submission in H1-2020


Jakavi® - JAK1/2 inhibitor

88

Study NCT03491215 REACH4 (CINC424F12201) NCT04097821 ADORE (CINC424H12201)

Indication Graft versus host disease Myelofibrosis

Phase Phase 2 Phase 1/2

Patients 45 130

Primary Outcome

Measures

• Measurement of PK parameters

• Overall Response Rate (ORR)

• Incidence of dose limiting toxicities within the first 2

cycles

• Response rate at the end of cycle 6

Arms/Intervention • Ruxolitinib

• Ruxolitinib

• Ruxolitinib+Siremadlin

• Ruxolitinib+Crizanlizumab

• Ruxolitinib+MBG453

Target PatientsPediatric patients with grade II-IV acute graft vs. host disease

after allogeneic hematopoietic stem cell transplantationPatients with Myelofibrosis (MF)


Publication TBD TBD


Kisqali® - CDK 4/6 inhibitor

89

Study NCT03701334 NATALEE (CLEE011O12301C)

IndicationAdjuvant treatment of hormone receptor (HR)-positive,

HER2-negative, early breast cancer (EBC).

Phase Phase 3

Patients ~4,000

Primary Outcome

Measures

Invasive Disease-Free Survival for using STEEP criteria

(Standardized Definitions for Efficacy End Points in adjuvant

breast cancer trials)

Arms/Intervention• Ribociclib + endocrine therapy

• Endocrine therapy

Target Patients

Pre and postmenopausal women and men with HR-positive,

HER2-negative EBC, after adequate surgical resection, who

are eligible for adjuvant endocrine therapy


Publication TBD


Kymriah® – CAR-T therapy

90

Study NCT02445248 JULIET (CCTL019C2201) NCT03568461 ELARA (CCTL019E2202)

Indication Relapsed / refractory DLBCL Relapsed / refractory follicular lymphoma (FL)


Patients 128 113

Primary Outcome

MeasuresOverall response rate; efficacy and safety of CTL019 Complete Response Rate (CRR)

Arms/Intervention Single-arm study of single dose of CTL019 Single-arm study of tisagenlecleucel

Target PatientsAdult patients with relapsed or refractory diffuse large B-cell

lymphoma (DLBCL)Adult patients with relapsed or refractory FL

Expected Completion 2017 (actual) H2-2020 (interim analysis)

Publication

• Schuster et al. Presentations at ICML 2017; at EHA

2017; at ASH 2017; at ASH 2018; Borchmann et al.

Presentation at EHA 2018; Bachanova et al.

Presentation at ICML 2019

• Schuster et al. N Engl J Med. 2019;380(1):45-56. doi:

10.1056/NEJMoa1804980. Epub 2018 Dec 1.

TBD


Kymriah® – CAR-T therapy

91

Study NCT03876769 CASSIOPEIA (CCTL019G2201J) NCT03570892 BELINDA (CCTL019H2301)

Indication 1st line high risk acute lymphoblastic leukemia (ALL) 2nd line Diffuse large B-cell lymphoma (DLBCL)


Patients 160 318

Primary Outcome

Measures5 year Disease Free Survival (DFS) Event-free Survival (EFS)

Arms/Intervention Single-arm study of tisagenlecleucel; retreatment allowed Tisagenlecleucel versus standard of care

Target Patients Pediatric and young adult patients with 1st line high risk ALL

Adult patients with aggressive B-cell Non-Hodgkin

Lymphoma after failure of rituximab and anthracycline-

containing frontline immunochemotherapy


Publication TBD TBD


MBG453 – Tim-3 antagonist

92

Study NCT03946670 (CMBG453B12201)

Indication Myelodysplastic syndrome

Phase Phase 2

Patients 120

Primary Outcome

Measures

Complete Remission (CR) rate and Progression Free

Survival (PFS)

Arms/Intervention• Experimental: MBG453 + hypomethylating agents

• Placebo comparator: Placebo + hypomethylating agents

Target PatientsAdult subjects with intermediate, high or very high risk

Myelodysplastic Syndrome (MDS) as per IPSS-R criteria


Publication TBD


PDR001 – PD-1 checkpoint inhibitor

93

Study NCT02967692 COMBI-i (CPDR001F2301)

Indication BRAFV600 mutant metastatic melanoma

Phase Phase 3

Patients

538

Part 1 (safety-run in): 9; Part 2 (biomarker cohort): 27; Part 3

(Phase III, randomized, placebo controlled): 532

Primary Outcome

MeasuresProgression-Free Survival (PFS)

Arms/Intervention

• Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg bid +

Mekinist 2 mg

• Placebo + Tafinlar 150 mg bid + Mekinist 2 mg

Target Patients

Previously untreated patients with unresectable or

metastatic BRAF V600 mutant melanoma

Expected Completion H2-2019 (IA); H2-2020 (Final analysis)

Publication TBD


Rydapt®- Multi-targeted kinase inhibitor

94

Study NCT03280030 (CPKC412A2220) NCT03591510 (CPKC412A2218)

Indication Acute myeloid leukemia Acute myeloid leukemia


Patients 66 50

Primary Outcome

MeasuresIncidence of safety events and event free survival

Occurrence of dose limiting toxicities

Event Free Survival ( EFS)

Arms/Intervention• Midostaurin 50 mg

• Placebo• Chemotherapy followed by Midostaurin

Target PatientsNewly diagnosed patients with FLT3-mutated acute myeloid

leukemia (AML)

Newly diagnosed pediatric patients with FLT3 mutated acute

myeloid leukemia (AML)


Publication TBD TBD


PDR001 - PD-1 checkpoint inhibitor

Study NCT03484923 (CPDR001J2201)

Indication Previously treated unresectable or metastatic melanoma

Phase Phase 2

Patients 230

Primary Outcome

MeasuresObjective Response Rate (ORR)

Arms/Intervention

• PDR001 400mg i.v. Q4W + LAG525 600 mg i.v. Q4W

• PDR001 400mg i.v. Q4W + capmatinib 400 mg bid orally

• PDR001 400mg i.v. Q4W + canakinumab 300 mg (s.c)

Q4W

• PDR001 400mg i.v. Q4W + ribociclib 600 mg p.o QD on

Days 1 to 21 of a 28-day cycle

Target PatientsAdult patients with previously treated unresectable or

metastatic melanoma


Publication Abstract submission to congress in H1-2020


95

Promacta®/Revolade® – Thrombopoetin receptor agonist

Study NCT03025698 (CETB115E2201)

IndicationPreviously untreated or relapsed/refractory severe aplastic anemia or

recurrent aplastic anemia

Phase Phase 2

Patients 60

Primary Outcome

MeasuresPK of eltrombopag at steady state in pediatric patients with SAA

Arms/Intervention

• Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS

• Arm B: previously untreated SAA-hATG/cyclosporine +

eltrombopag

• Arm A: relapsed/refractory SAA or AA: hATG/cyclosporine +

eltrombopag or cyclosporine + eltrombopag

Target PatientsPediatric patients from age 1 <18 years with relapsed/refractory SAA

or recurrent AA after IST or previously untreated SAA


Publication TBD


96

SEG101 – p-Selectin inhibitor

Study NCT03264989 SOLACE-Adults (CSEG101A2202) NCT03474965 SOLACE-Kids (CSEG101B2201)

IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with

Sickle Cell Disease (SCD) Prevention of VOC in pediatric patients with SCD


Patients 55 100

Primary Outcome

MeasuresPK/PD and safety of SEG101 (crizanlizumab) at 5 mg/kg PK/PD and safety of SEG101 at 5 mg/kg

Arms/Intervention

SEG101 (crizanlizumab) at a dose of 5.0 mg/kg (or 7.5

mg/kg for exploratory group) by IV infusion, ±

Hydroxyurea/Hydroxycarbamide

SEG101 (crizanlizumab) at a dose of 5 mg/kg by IV infusion

± Hydroxyurea/Hydroxycarbamide

Target Patients Adult SCD patients with VOC Pediatric SCD patients with VOC

Expected Completion Q4-2018 (actual)H2-2021 (pediatric patients ≥6 year old)

2022 (pediatric patients 6 months – 6 year old)

Publication Abstract submission to congress in H1-2020 TBD


97

SEG101 – p-Selectin inhibitor

Study NCT03814746 STAND (CSEG101A2301)

IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with

Sickle Cell Disease (SCD)

Phase Phase 3

Patients 240

Primary Outcome

MeasuresRate of VOC events leading to healthcare visit

Arms/Intervention

• Crizanlizumab 5.0 mg/kg

• Crizanlizumab 7.5 mg/kg

• Placebo

Target Patients Adolescent and adult SCD patients (12 years and older)


Publication TBD


98

Study NCT01677741 (CDRB436A2102)

Indication BRAFV600 mutant cancers

Phase Phase 1/2

Patients 85

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics

Arms/InterventionSingle-arm study of oral dabrafenib (dose based on age

and weight)

Target PatientsPediatric subjects aged 1 year to <18 years with advanced

BRAF V600-mutation positive solid tumors


Publication

• Kieran MW et al. Manuscript Clin Cancer Res; (accepted

Q3-2019, not yet published) (PK analysis)

• Hargrave D et al. Manuscript Clin Cancer Res; (accepted

Q3-2019, not yet published) (safety/efficacy in low-grade

gliomas)

Tafinlar® - BRAF inhibitor


99

Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor

Study NCT02684058 (CDRB436G2201)

Indication BRAFV600 mutant gliomas

Phase Phase 2

Patients 142

Primary Outcome

MeasuresObjective response rate

Arms/Intervention Dabrafenib + trametinib (dose based on age and weight)

Target Patients

Children and adolescent patients with BRAF V600 mutation

positive relapsed or refractory high grade glioma (HGG) or

BRAF V600 mutation positive low grade glioma (LGG)


Publication TBD


100

Tafinlar®+Mekinist® - BRAFV600 inhibitor and MEK inhibitor

Study NCT02124772 (CTMT212X2101)

Indication BRAFV600 mutant solid tumors

Phase Phase 1

Patients 142

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics and clinical activity

Arms/InterventionTrametinib (dose based on age and weight)

Dabrafenib + trametinib (dose based on age and weight)

Target PatientsPediatric Subjects Aged 1 Month to <18 Years with

Advanced V600-Mutation Positive Solid Tumors


Publication Abstract submission to congress in H1-2020


101

Zykadia® - ALK inhibitor


102

Study NCT02299505 ASCEND-8 (CLDK378A2112)

Indication ALK activated NSCLC

Phase Phase 2

Patients 306

Primary Outcome

Measures

Part 1: Pharmacokinetics when taken with food

Part 2: Overall Response Rate (ORR) when taken with food

Arms/Intervention

• Oral LDK378 450 mg once daily taken with food

• Oral LDK378 600 mg once daily taken with food

• Oral LDK378 750 mg once daily fasted

Target PatientsAdult patients with ALK-rearranged (ALK-positive) advanced non-small cell

lung cancer

Expected Completion

Part 1 (PK): 2016 (actual)

Part 2 (ORR): Q2-2018 (actual)

Final (ORR): Q4-2019

Publication

• Part 1 (PK): Cho BC, et al. J Thorac Oncol. 2017 Sep; 12(9) 1357-1367

• Part 2 (ORR): Cho B et al. J Thorac Oncol. 2019 Jul; 14(7) 1255-1265

• Final (ORR): TBD

177Lu-PSMA-617 – Lu-labelled prostate specific membrane antigen (PSMA)


103

Study NCT03511664 VISION (PSMA-617-01)

IndicationPSMA-positive Metastatic Castration-resistant Prostate

Cancer (mCRPC)

Phase Phase 3

Patients 750

Primary Outcome

Measures

• Radiographic Progression Free Survival

• Overall Survival

Arms/Intervention• 177Lu-PSMA-617 plus BS/BSC

• BS/BSC alone

Target Patients

Adult patients with PSMA-positive Metastatic Castration-

resistant Prostate Cancer (mCRPC)

Expected Completion H1 2020

Publication TBD

Ophthalmology

Lucentis® - Anti-VEGF

105

Study NCT02375971 RAINBOW (CRFB002H2301) NCT02640664 RAINBOW Extension (CRFB002H2301E1)

Indication Retinopathy of Prematurity (ROP) Retinopathy of Prematurity (ROP)


Patients 224 180

Primary Outcome

Measures

Absence of active Retinopathy of Prematurity (ROP) and

unfavorable structural outcome at Week 24, defined as, 1)

survival, 2) no intervention with a second modality for ROP,

3) absence of active ROP and 4) absence of unfavorable

structural outcome

To evaluate the visual function of patients by assessing the

visual acuity in the better-seeing eye at the patient’s fifth

birthday.

Arms/Intervention

• Ranibizumab 0.2 mg (up to 3 injections max)

• Ranibizumab 0.1 mg (up to 3 injections max)

• Laser therapy

• Ranibizumab 0.2 mg (up to Week 40, if warranted)

• Ranibizumab 0.1 mg (up to Week 40, if warranted)

Target PatientsMale and female preterm infants with bilateral retinopathy of

prematurity (ROP) who require treatment.

Male and female preterm infants with bilateral retinopathy of

prematurity (ROP) who completed RAINBOW.


Publication

• EURETINA: Sep-2018

• AAO: Oct-2018

• Primary manuscript published online by The Lancet in

Sep-2019

(https://www.thelancet.com/pdfs/journals/lancet/PIIS0140

-6736(19)31344-3.pdf)

TBD


RTH258 - Anti-VEGF

Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)

Indication Neovascular age-related macular degeneration (nAMD) Neovascular age-related macular degeneration (nAMD)


Patients 743 1,082

Primary Outcome

Measures

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Arms/Intervention• RTH258 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• RTH258 3 mg/50 µL

• RTH258 6 mg/50 µL


Target Patients Subjects with exudative age-related macular degeneration Subjects with exudative age-related macular degeneration


Publication

• Oral presentations including both primary endpoint and key 2nd superior anatomic outcomes at AAO meetings in Nov-

2017 (1st year results) and Nov-2018 (2nd year results)

• Year 1 Manuscript: Dugel P, et al. Ophthalmology 2019 Apr 12; HAWK and HARRIER: Phase 3, Multicenter,

Randomized, Double-Masked Trials of Brolucizumab for Neovascular Age-Related Macular Degeneration.

• Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses

(Angiogenesis/Mac Soc in Feb-2019; WRC in Mar-2019; ARVO in April-2019; ASRC July-2019; EURETINA Sept-2019;

AAO Oct-2019 and APVRS Dec-2019


106

RTH258 - Anti-VEGF

Study NCT03386474 (CRTH258A2301E1) NCT03481634 KESTREL (CRTH258B2301)

Indication Neovascular age-related macular degeneration (nAMD) Diabetic eye disease


Patients 150 534

Primary Outcome

MeasuresNumber of treatment-emergent adverse events

Change from baseline in best-corrected visual acuity

(BCVA)



• RTH258 3 mg/50 µL

• RTH258 6 mg/50 µL

• Aflibercept 2mg/50 uL

Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study

Patients with visual impairment due to diabetic macular

edema (DME)


Publication TBD TBD


107

RTH258 - Anti-VEGF

Study NCT03481660 KITE (CRTH258B2302) NCT04058067 KINGLET (CRTH258B2304)

Indication Diabetic eye disease Diabetic macular edema


Patients 356 268

Primary Outcome

Measures


(BCVA)Change in best-corrected visual acuity (BCVA)



• Brolucizumab 6 mg

• Aflibercept 2 mg

Target PatientsPatients with visual impairment due to diabetic macular

edema (DME)

Patients with visual impairment due to diabetic macular

edema


Publication TBD TBD


108

RTH258 - Anti-VEGF

Study NCT03917472 (CRTH258B2305) NCT03802630 RAPTOR (CRTH258C2301)

Indication Diabetic macular edema Retinal vein occlusion


Patients 500 500

Primary Outcome

Measures

Change in best-corrected visual acuity (BCVA) from

baseline up to week 52


(BCVA) at week 24

Arms/Intervention• Brolucizumab (RTH258) 6 mg/0.05 mL every 4 weeks

• Aflibercept 2 mg/0.05 mL every 4 weeks

• Brolucizumab 6 mg every 4 weeks

• Aflibercept 2 mg every 4 weeks

Target PatientsPatients with visual impairment due to diabetic macular

edema

Adult patients with visual impairment due to macular edema

secondary to branch retinal vein occlusion


Publication TBD TBD


109

RTH258 - Anti-VEGF

Study NCT03810313 RAVEN (CRTH258C2302)

Indication Retinal vein occlusion

Phase Phase 3

Patients 750

Primary Outcome

Measures


(BCVA) at week 24

Arms/Intervention• Brolucizumab 6 mg every 4 weeks

• Aflibercept 2 mg every 4 weeks

Target PatientsAdult patients with visual impairment due to macular edema

secondary to central retinal vein occlusion


Publication TBD


110

UNR844 - Disulfide bonds modulator

Study NCT03809611 (CUNR844A2203)

Indication Presbyopia

Phase Phase 2

Patients 120

Primary Outcome

Measures

Change in binocular distance-corrected near visual acuity

(DNCVA) from baseline

Arms/Intervention• 1.5% solution UNR844-Cl

• Placebo

Target Patients Patients with presbyopia


Publication Planned in ASRCS in 2020


111

Respiratory

QAW039 – DP2 receptor antagonist

113

Study NCT02555683 LUSTER-1 (CQAW039A2307) NCT02563067 LUSTER-2 (CQAW039A2314)

Indication Asthma Asthma


Patients 846 846

Primary Outcome

Measures

Reduction in the rate of moderate-to-severe asthma

exacerbations

Reduction in the rate of moderate-to-severe asthma

exacerbations

Arms/Intervention

• QAW039 Dose 1

• QAW039 Dose 2

• Placebo

• QAW039 Dose 1

• QAW039 Dose 2

• Placebo

Target Patients Patients with uncontrolled severe asthma Patients with uncontrolled severe asthma

Expected Completion Q4-2019 Q3-2019

Publication Planned in 2020 Planned in 2020



Study NCT03215758 ZEAL-1 (CQAW039A2316) NCT03226392 ZEAL-2 (CQAW039A2317)



Patients 650 650

Primary Outcome

MeasuresPre-dose forced expiratory volume in 1 second (FEV1) Pre-dose forced expiratory volume in 1 second (FEV1)

Arms/Intervention• QAW039

• Placebo

• QAW039

• Placebo

Target Patients Patients with uncontrolled asthma Patients with uncontrolled asthma


Publication Planned in 2020 Planned in 2020


114


Study NCT03052517 SPIRIT (CQAW039A2315) NCT03650400 (CQAW039B2201)



Patients 1,900 – 2,300 24

Primary Outcome

Measures

Long term safety: treatment emergent adverse event (AE),

SAE and AE leading to discontinuation from study (52 wks

and 160 wks)

Pharmacokinetics, safety and tolerability

Arms/Intervention

• QAW039 Dose 1

• QAW039 Dose 2

• Placebo

• Fevipiprant Cohort A; Fevipiprant Cohort B; Chewable

tablet

Target Patients Patients with moderate to severe asthma Children aged 6 to < 12 years with asthma

Expected Completion Q4-2019 (for submission); 2022 (final) H2-2020

Publication TBD TBD


115

QBW251 - CFTR potentiator

116

Study NCT04072887 (CQBW251B2201)

Indication Chronic obstructive pulmonary disease (COPD)

Phase Phase 2

Patients 900

Primary Outcome

Measures

Trough FEV1 (Forced Expiratory Volume in 1 second)

change from baseline after 12 weeks of treatment

Arms/Intervention

• QBW251 450 mg

• QBW251 300 mg

• QBW251 150 mg

• QBW251 75 mg

• QBW251 25 mg

• Placebo

Target PatientsCOPD patients on background triple inhaled therapy (LABA /

LAMA / ICS)


Publication TBD


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

117

Study NCT02892019 (CQMF149G2202)

Indication Asthma

Phase Phase 2

Patients 80

Primary Outcome

MeasuresTrough FEV1

Arms/Intervention• Indacaterol acetate 75 μg od (via Concept1 inhaler)

• Indacaterol acetate 150 μg od (via Concept1 inhaler)

Target Patients Children ≥ 6 to < 12 years of age with asthma


Publication TBD


QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid

Study NCT02554786 PALLADIUM (CQVM149B2301) NCT02571777 IRIDIUM (CQVM149B2302)




Primary Outcome

MeasuresTrough FEV1 Trough FEV1

Arms/Intervention

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• MF 400 µg od

• MF 400 µg bid

• Salmeterol 50 µg /fluticasone 500 µg bid

• QVM149 150/50/160 µg od

• QVM149 150/50/80 µg od

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• Salmeterol 50 µg /fluticasone 500 µg bid

Target Patients

Adult and adolescent (≥12 years) patients with asthma

inadequately controlled on medium/high-dose ICS or low-

dose LABA/ICS (GINA step ≥ 3)

Adult (≥18 years) patients with asthma inadequately

controlled on medium/high-dose of LABA/ICS (GINA step ≥4)


Publication• Planned in H1-2020

• Planned abstract for BTS in Q4-2019Planned in H1-2020


118


119

Study NCT03100500 (CQVM149B1305) NCT03100825 (CQVM149B1304)



Patients 51 94

Primary Outcome

Measures

Long-term safety/tolerability: Incidence and severity of

treatment emergent adverse events during the 52 weeks

study

Long-term safety/tolerability: Incidence and severity of

treatment emergent adverse events during the 52 weeks

study

Arms/Intervention • Single arm: QMF149 150/320 μg od • Single Arm: QVM149 150/50/160 μg od

Target Patients Japanese patients with asthma inadequately controlled Japanese patients with asthma inadequately controlled


Publication• Planned in H1-2020

• Planned abstract for ATS in 2020

• Planned in H1-2020

• Planned abstract for ATS in 2020



Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)



Patients 802 1,251

Primary Outcome

MeasuresTrough FEV1

Non-inferiority of Asthma Quality of Life Questionnaire

(AQLQ)

Arms/Intervention• QMF149 150/80 µg od

• MF 200 µg od

• QVM149 150/50/80 μg od

• QVM149 150/50/160 μg od

• Salmeterol/fluticasone 50/500 μg bid + tiotropium 5 μg od

Target Patients

Adult and adolescent (≥12 years) patients with mild asthma

inadequately controlled on low-dose ICS or low-dose

LABA/ICS (Gina step 2-3)

Patients with uncontrolled asthma

Expected Completion Q1-2019 (actual) Q3-2019

Publication Planned in Q1-2020 Planned in H1-2020


120

Xolair® – anti-IgE antibody

121

Study NCT03369704 (CIGE025F1301)

Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis

Phase Phase 3

Patients 337

Primary Outcome

MeasuresMean nasal symptom score, consists of severity of sneezing, rhinorrhea and nasal congestion.

Arms/Intervention

In addition to standard of care:

• Omalizumab per approved allergic asthma dosing table for IgE/body weight combinations

• Placebo

Target PatientsPatients with severe Japanese cedar pollinosis, whose symptoms were inadequately controlled with current

recommended therapies


Publication

• Late breaking abstract was published at AAAAI (American Association of Allergy, Asthma and

Immunology) annual meeting, Feb-2019.

• Oral/poster published at EAACI (the European Academy of Allergy and Clinical Immunology), Jun-2019

• Planned oral/poster to be submitted for JRS (Japanese Rhinologic Society) in Oct-2019

• Planned manuscript to be submitted to JACI (The Journal of Allergy and Clinical Immunology) in Q4-2019


Sandoz Biopharmaceuticals

Hyrimoz® - Biosimilar adalimumab

123

Study NCT02744755 ADMYRA (GP17-302)

Indication Immunology

Phase Phase 3

Patients 353

Primary Outcome

Measures

Change in DAS28-CRP score from baseline to week 12 in

patients treated with GP2017 and patients treated with

Humira®

Arms/Intervention• GP2017

• US licensed Humira® adalimumab

Target Patients Patients with moderate to severe active rheumatoid arthritis

Expected Completion 2018 (actual)

Publication• Wiland, P. et al., presented at EULAR 2019

• Wiland, P. et al., BioDrugs, Q4 2019


GP2411 - Biosimilar denosumab

124

Study NCT03974100 (CGP24112301)

Indication Osteoporosis

Phase Phase 3

Patients 522

Primary Outcome

Measures

Percent change from baseline (%CfB) in lumbar spine Bone

Mineral Density

Arms/Intervention

• GP2411 60 mg /mL subcutaneous injection every 6

months

• Prolia® 60 mg /mL subcutaneous injection every 6

months

Target Patients Postmenopausal women with osteoporosis


Publication Study data publications expected for 2024 and beyond


Global Health

KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated

126

Study NCT03167242 (CKAF156A2202)

Indication Malaria

Phase Phase 2

Patients 512

Primary Outcome

Measures

PCR-corrected adequate clinical and parasitological

response (ACPR)

Arms/Intervention• KAF156 and LUM-SDF (different combinations)

• Coartem

Target PatientsAdults and children with uncomplicated Plasmodium

Falciparum Malaria


Publication TBD


KAE609 – Plasmodium Falciparum Inhibitor – spiroindolone against PfATP4

127

Study NCT03334747 (CKAE609A2202)

Indication Malaria

Phase Phase 2

Patients 210

Primary Outcome

Measures

CTCAE grades increase from baseline in alanine

aminotransferase (ALT) or aspartate aminotransferase

(AST)

Arms/Intervention• KAE609

• Coartem

Target Patients Adults with uncomplicated Plasmodium Falciparum malaria


Publication TBD


Key definitions and trademarks


This presentation contains several important words or phrases that we define as below:

AE: Adverse Event

ALL: Acute lymphatic leukemia

AMD: Age-Related Macular Degeneration

AML: Acute myeloid leukemia

Approval: In Pharmaceuticals and Alcon in US and EU; each indication and regulator combination counts as

approval; excludes label updates, CHMP opinions alone and minor approvals

aRCC: advanced renal cell cancer

AS: Ankylosing Spondylitis

bid: twice a day

BC: Breast cancer

BCMA: B-cell maturation antigen

BCVA: best corrected visual acuity

BS: Biosimilars

BTD: Breakthrough therapy designation

CGRP: Calcitonin gene-related peptide

CLL: Chronic lymphocytic leukemia

CM: Chronic migraine

CML: Chronic myeloid leukemia

COPD: Chronic Obstructive Pulmonary Disease

CR: complete remission

CRC: Colorectal Cancer

CSU / CIU: Chronic spontaneous urticaria / Chronic idiopathic urticaria

CVRR: Cardiovascular risk reduction

DLBCL: Diffuse large B-cell lymphoma

DMC: Data monitoring committee

EF: ejection fraction

EM: Episodic migraine

FL: Follicular lymphoma

FPFV: First patient first visit

GBM: Glioblastoma multiforme

HF: Heart failure

HF-pEF: Heart failure with preserved ejection fraction

HFrEF: Heart failure with reduced ejection fraction

HR+/HER2- mBC:Hormone Receptor positive / Human Epidermal growth factor receptor 2 negative metastatic

breast cancer

LoE: Loss of exclusivity

M/M: Multiple myeloma

MF: Myelofibrosis

MI: Myocardial infarction

MS: Multiple sclerosis

NASH: Non-Alcoholic Steatohepatitis

NET: Neuroendocrine tumor

NSCLC: Non-small cell lung cancer

NTD: New Therapeutic Drug

od: once a day

ORR: Overall response rate

OS: Overall survival

PA: Prior authorization

PASI 90: 90% reduction in Psoriasis Area Severity Index from baseline

PFS: Progression free survival

PSA: Prostate specific antigen

PsA: Psoriatic arthritis

PsO: Psoriasis

PV: Polycythemia vera

PY: Prior year

QoL: Quality of Life

RCC: Renal cell cancer

r/r ALL: relapsed/refractory acute lymphoblastic leukemia

RRMS: relapsing-remitting multiple sclerosis

SCPC: Sickle cell pain crisis

SpA: Spondyloarthropathy

SPMS: Secondary progressive multiple sclerosis

TFR: Treatment-free Remission

TNBC: Triple negative breast cancer

Q3 2019 Results - Novartis

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