Q2 2019 Results - Novartis

Q2 2019 ResultsInvestor Presentation

July 18, 2019

Novartis AG

Investor Relations

Disclaimer

Novartis Q2 2019 Results | July 18, 2019 | Novartis 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 share buyback plan; 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 strategic benefits, synergies or opportunities expected from the Alcon and Sandoz transactions 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 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; uncertainties involved in the development or adoption of potentially transformational technologies and business models; 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.

Vas NarasimhanChief Executive Officer

Launched

Launched

Launched

Acquired in July

SEG101 Filed with priority review

Achieved overall survival3

Novartis delivered strong Q2 with margin expansion and transformative innovation


Q2 operational performanceContinuing operations1 growth vs. PY in % cc 2

Transformative Innovation

Sales

+8%

Sales & Core OpInc guidance increased

Core OpInc

+20%

Core margin

+3.2%

pts

1. Continuing operations as defined on page 42 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 55 of the Condensed Interim Financial Report.

3. Pre- and perimenopausal women with HR+/HER2- advanced or metastatic breast cancer

Key growth drivers

Lutathera®

Sales performance driven by Innovative Medicines

1. Not meaningful

Novartis Q2 2019 Results | July 18, 2019 | Novartis Investor Presentation

42

52

56

57

85

157

29

45

182

5

1

1

1

1

SalesUSD Million

Growth vs. PYUSD Million

Growth vs. PYcc

SalesUSD Million

Growth vs. PYcc

421 81% 778 83%

858 25% 1,649 32%

109 nm 215 nm

349 23% 656 24%

340 25% 637 21%

111 94% 202 103%

284 26% 542 23%

58 nm 103 nm

290 18% 571 19%

Q2 H1

http://www.novartisoncology.com/products/jakavi.jsp

Cosentyx® continued growth in competitive environment

US Dermatology1

NBRx %pts share gains/losses Q2 2019 vs. Q2 2018

US Rheumatology1

Weekly TRx YTD

Rheumatology market TRx2 +14% YoY

Cosentyx® TRx +38% YoY

Cosentyx® NBRx growth +11% YoY and gaining share

Dermatology market TRx +10% YoY driven by new launches

Cosentyx® TRx +28% YoY

Cosentyx® NBRx growth +17% YoY and gaining share


All trademarks are the property of their respective owners 1.Source: IQVIA National Prescription Audit for dermatologists/ rheumatologists, WE 28/06/2019 2. Rx for SpA indications: market includes Cimzia®, Enbrel®, Humira®, Simponi®,

Stelara®, Taltz®, Otezla® and Cosentyx®

09/1807/1803/18 03/1905/18 01/1901/18 07/19

1.000

3.000

2.000

05/1911/18

5.000

4.000

0

Stelara®Humira®Enbrel® Taltz®Cosentyx®

1.5

1.3

0.8

0.0

-1.7

-2.4

0.5

TNFs

Stelara®

Tremfya®

Other*

Taltz®

Cosentyx®

Skyrizi®

Cosentyx® now the only biologic with proven benefits across PsA domains including axial2 manifestations


The only product with evidence of benefit

in axial manifestations2

PsA – Psoriatic Arthritis 1. Baraliakos X. et al. OP0235 Secukinumab improves axial manifestations in patients with psoriatic arthritis and inadequate response to NSAIDS: primary analysis of the MAXIMISE trial. Presented at the annual

European League Against Rheumatism 2019 2. Cosentyx is the only biologic with a randomized clinical trial in axial PsA

51.657.8

66.3

42.9

56.463.1

26.232.8 31.3

0

20

40

60

80

100

0 4 8 12

Secukinumab

300mg s.c.

(N=164)

Secukinumab

150mg s.c.

(N=157)

Placebo

(N=164)

Weeks

% r

es

po

nd

ers

P<0.001 vs placebo (multiple

imputation at Week 12). N,

number of patients randomized

MAXIMISE: rapid and significant improvement in ASAS20 response rate through Wk13

(full analysis set)

Up to 70% of PsA patients

have axial manifestations1

70%

Entresto® delivered strong Q2 performance, solidifying position as first choice HFrEF treatment


ESC-HFA expert consensus supports Entresto®

first-line use in HFrEF1

Expert consensus meeting report of the Heart Failure Association of ESC

Sacubitril/valsartan is recommended as a replacement for ACE-I/ARBs to

reduce the risk of HF hospitalization and death in ambulatory patients with

HFrEF who remain symptomatic despite optimal medical treatment with an ACE-I,

a beta-blocker and a MRA.

Initiation of sacubitril/valsartan rather than an ACE-I or an ARB may be

considered for patients hospitalized with new-onset or decompensated CHF

to reduce the short-term risk of adverse events and to simplify management (by

avoiding the need to titrate ACE-I first and then switch to sacubitril/valsartan).

ESC-HFA – European Society of Cardiology - Heart Failure Association; HFrEF – Heart Failure with reduced ejection fraction; pEF – preserved ejection fraction; CHF – Chronic heart failure 1. Seferovic et al. Clinical practice update on heart

failure in in-patient setting 2019: pharmacotherapy, procedures, devices and patient management. An expert consensus meeting report of The Heart Failure Association of the European Society of Cardiology. EJHF, 2019.

129

110

Q2 2019

239

Q2 2018

421

US

Ex-US

221

200

+81%

Entresto® revenues: strong momentum

leveraging PIONEER-HF data USD m, % cc

PARAGON-HF HFpEF data expected to be presented at ESC in September

Zolgensma® rapid launch and access post-approval


MAY 24 MAY 27 JUN 3 JUN 7 JUN 21

FDA approval Promotion in

market

First commercial

policy

Product ready

to ship

First US patient

treated

First international

patient treated via

French ATU

Rapid launch

Payer AccessMedical

policies

>20 commercial plans representing 40% of commercial lives and 4 Medicaid plans have policies

on coverage (not all have been posted externally)

Majority of policies are in line or close to the label. Common limitations: 4 SMN2 copies,

combination use with nusinersen

Approval

rates

Very high approval rates for on-label patients via policy or medical exception1

Wide range of patients approved for treatment: age 1 to 23 months, weight 4 to 12 kg, 2 and 3

SMN2 copy numbers, treatment-naï ve and previously on nusinersen

Contracting 17 commercial plans representing more than 40% commercial lives have signed Letter of Intent

on contracting terms

1. Approval rates so far >95% for patients utilizing the OneGene Program

Expected Zolgensma® newsflow in H2

Regulatory

Data

US: initiating FDA discussions on path for intrathecal dosing for older populations

EU: approval in infants expected in Q4 2019

Japan: approval in infants expected in Q4 2019

Other country filings initiated in Q3 for broad global roll-out

SPR1NT (pre-symptomatic, IV): enrollment expected to be completed Q3;

updated data at World Muscle Society (October 2019)

STRONG (Type 2, IT): updated data at World Muscle Society (October 2019)

STR1VE Global (Type 1, IV, US + EU): update to data at European Paediatric

Neurology Society (September 2019)


Zolgensma® presymptomatic patients areachieving age-appropriate motor milestones


Patients with 2 copies of SMN2 within

WHO windows of normal achievement

4 patients

could sit without support for ≥30 secs

1 patient

could stand with assistance for ≥2 secs

WALKING ALONE

STANDING W/ ASSISTANCE

SITTING W/O SUPPORT

“AVXS-101 Gene-Replacement Therapy in Presymptomatic Spinal Muscular Atrophy: SPR1NT Study Update,” K. Strauss, et al. AAN, 2019.

Open label, data as of March 8, 2019

2 copies (n=8): median 5.4 months of follow-up

3 copies (n=8): median 2.2 months of follow-up

Presymptomatic

Piqray® is off to a solid start

US: received FDA approval on May 24

EU: CHMP opinion expected in H2 2019

Engaged with payers covering over 80% of the target population in the US

Strong Rx momentum driven by PIK3CA mutation testing

Latest NCCN guidelines1 recommend PIK3CA mutation testing and use of

fulvestrant + alpelisib for PICK3CA-mutated tumors

Entered in agreement with Foundation Medicine to develop plasma and tissue test

Expanding programs to explore Piqray® in other tumor types:

- H2 2019 trial starts for HER2+ aBC and TNBC

- H1 2020 trial starts for head & neck and ovarian cancer

aBC – Advanced breast cancer TNBC – Triple negative breast cancer 1. NCCN guidelines have been updated to Version 2 2019


Building foundation for long term success of Mayzent®

RMS – Relapsing multiple sclerosis SPMS – Secondary progressive multiple sclerosis FDO – First dose observation 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. FDO is only recommended for patients with certain pre-existing cardiac conditions -sinus bradycardia, first or second-degree[Mobitz type I] AV block, or a history of myocardial

infarction or heart failure 3. Benedict et al. Effect of Siponimod on cognition in patients with SPMS: Ph3 EXPAND study subgroup analyses. AAN 2019. P2-051 4. Novartis QuickPulse HCP Tracker, reported by InCrowd Inc. fielded May 3-

15, 2019. 5. Spherix Global Insights RealTime Dynamix – MS Q219


CHMP opinion expected H2 2019

1st choice in active SPMS product for HCPs4

90% neurologists willing to prescribe Mayzent®5

>70m lives with preferred access to Mayzent®

MSProDiscuss™ launched to help target patient

identification

Unique clinical data and supportive label

Full range of RMS indication

Active SPMS1 (EDSS range: 3.0 to 6.0)

Efficacy, reduces disease progression

Safety and tolerability

No FDO (~70%)2

Cognitive processing speed3

Priorities are driving Mayzent® differentiation

and the urgency to treat in active SPMS

Beovu™ (brolucizumab, RTH258) differentiated clinical profile – on track for launch upon approval

nAMD – neovascular age-related macular degeneration Brand name BeovuTM provisionally approved by FDA for brolucizumab. the product has not received regulatory approval in any jurisdiction 1. Met primary efficacy endpoint of

noninferiority to aflibercept in mean change in BCVA with comparable safety to aflibercept; vision maintained up to Week 96 2. At Week 48, demonstrated superiority in three secondary endpoints considered key markers of nAMD in clinical

practice: central subfield retinal thickness, retinal fluid (intraretinal fluid and/or subretinal fluid) and disease activity; advantages maintained at Week 96 3. At Week 48, the majority of patients (56% and 51%) were maintained on q12w

injection interval in Hawk and Harrier respectively with remaining patients on q8w regimen (key secondary endpoints); greater than 75% of these patients continued on q12w dosing up to Week 96. 4. FirstWord Pharma Physician Views

snap-poll results, 26 April 2019 5. https://clinicaltrials.gov/ct2/show/NCT04005352


US approval expected Q4 2019; CHMP opinion

expected Q1 2020

Strong awareness of clinical data (HAWK &

HARRIER)4

US operations ready – commercial and medical

teams hired and trained; supply readiness

EU market readiness in line with local

reimbursement timelines

Preparing for launchStrong clinical program

HAWK and HARRIER demonstrated

uncompromised vision, less retinal fluid, fewer

injections1,2,3

Expanded clinical program including TALON

head-to-head study of brolucizumab vs.

aflibercept in a treat-to-control regimen5

Accelerating Xiidra® activities while laying foundation to maximize potential


0

5,000

10,000

15,000

20,000

25,000

30,000

35,000

40,000

45,000

50,000

55,000

10/1807/18 01/1904/18 04/19 07/1901/18

Dry eye disease weekly TRx2

MoA - Mechanism of action. SoV – Share of Voice. DTC – Direct To Consumer 1. Nichols KK et al. Inv Ophthalmol & Vis Sci. 2016;57:2975-2982 2. IQVIA National Prescription Audit 3. Paulsen AJ et al. Am J Ophthalmic

2014;157(4):799-806 4. US Census Bureau. Annual estimates of the resident population for selected age groups by sex for the United States, States, Counties, and Puerto Rico Commonwealth and Municipios: April 1, 2010 to July 1, 2014

5. Schaumberg et al, 2013, Prevalence of diagnosed dry eye in the US, Marketscope 2018 report – Diagnosed Dry Eye patients in the US 6. Novartis Dry Eye market forecasts in the US, Mar 2019, validated with IQVIA TRx and NBRx claims

data Restasis® is a registered trademark of Allergan

Dry eye disease underdiagnosed,

undertreated1, increasing in

incidence

US dry eye

patients3,4

Diagnosed

by ECP5

Prescribed

Rx6

~50% self-

diagnose

~10% treated

with Rx

34 m

17 m

1.6 m

Restasis®

Xiidra®

Opportunities to re-ignite Xiidra®

Only drug to address signs and symptoms of

dry eye through unique anti-inflammatory MoA

Re-engage sales force to increase SoV on

value proposition

Optimize medical education and promotion

including DTC – new campaign Q4 2019

Next steps to maximize Xiidra®

potential

Expand access for Part D patients beginning

in 2021

Crizanlizumab (SEG101) submitted in US and EU – ready for a successful launch in anticipation of potential approval

Ready for a successful launch in US

Commercial field organization in place

Value proposition and account level plan designed to

maximize payer coverage and reimbursement

Post-launch patient support program planned

Innovative disease awareness & patient engagement ongoing


Submitted in US Q2 2019

Priority review granted on 15 July 2019

Approval anticipated on or before Q1 2020

Submitted in EU Q2 2019 for conditional

approval, expected in Q3 2020

Rest of World submissions planned for

after US approval

NotAloneinSickleCell.com

Q3 Q4 Q1 Potential

First approved therapy in HFpEF

First subcutaneous B-cell therapy

in RMS

First IL-17 treatment for all axial

SpA manifestations

Triplet Tafinlar® and Mekinist® /

PD-1 filing

First oral DP2 inhibitor in asthma

Catalyst-rich H2 with 5 major program readouts

Entresto®

PARAGON-HF

Cosentyx®

PREVENT

Fevipiprant (QAW039)

LUSTER1&2

Ofatumumab (OMB157)

ASCLEPIOS 1&2

PDR001 Combo1

COMBI-i

1. Combination with Tafinlar® and Mekinist®


Fevipiprant (QAW039)

ZEAL1&2

PARAGON-HF potentially the first positive confirmatory trial in HFpEF – results to be presented at ESC in September

Intentionally designed to assess impact of Entresto® on overall burden of disease

Potentially first positive morbidity / mortality study in HFpEF

Reduction in total HF hospitalizations, with or without significant effect on CV mortality, would be a

significant breakthrough

Indication/population Primary endpoint (novel) Next expected milestones

LVEF >45%

N=4,822

vs. valsartan

CV death and total (first &

recurrent) HF hospitalization

Results and filing planned H2 2019

ESC late-breaking presentation

HFpEF- heart failure with preserved ejection fraction LVEF - left ventricular ejection fraction CV – Cardiovascular HF – heart failure FIR - First interpretable results ESC - European Society of Cardiology


Ofatumumab (OMB157): potential to provide access to high efficacy b-cell therapy for broad RMS patient population


Loading dose induces rapid and profound B-cell depletion

Monthly dosing mimics the natural recovery cycle of B cells

keeping them at very low levels consistently, avoiding

rebound at end of cycle

Subcutaneous, therefore:

– Flexibility for quick replenishment

– Self-administered, therefore not limited to centers of excellence

– Improved lymph node targeting (vs. IV)

ARR – Annualized relapse rates. 1. Bar-Or et al., April 2018, Neurology, 2018; 90:e1805-e1814. 2. Modelling data on file based on PK-PD model developed from Ph2b.

Me

an

nu

mb

er

of

ne

w T

1 G

d+

le

sio

ns

Dosing regimen2 expected to provide rapid & sustained

B-cell depletion

ASCLEPIOS submitted for presentation at ECTRIMS 2019: potentially high efficacy on T1 Gd+ lesion, ARR and disability

Ph2 ofatumumab in relapsing MS MIRROR data1

Moderate to Severe asthma patientsUS patient numbers

Fevipiprant (QAW039): Comprehensive Ph3 program to address a treatment gap in asthmaTrial readouts on track. Full dataset required to assess product potential


Trial Population / primary endpoint

LUSTER 1 & 22 GINA 4/5, high eosinophils

Exacerbations

1,692 patients1

ZEAL 1 & 23 GINA 3/4

Lung Function (FEV1)

1,300 patients1

SPIRIT4 GINA 3/4/5

Safety

1,570 patients1

GINA – Global Initiative for Asthma. 1. Number of patients per protocol (for LUSTER & ZEAL combined number). 2. Anticipate readout Q1 2020; LUSTER 1 & 2 together with SPIRIT interim analysis could represent the core submission

package. 3. Anticipate readout Q4 2019. 4. Interim analysis readout expected end 2019.

Marie-France Tschudin appointed President of Novartis Pharmaceuticals

Member of Executive Committee of Novartis

25+ years experience in pharma and biotech

industry, including 10 years at Celgene

Purpose-driven leader; consistently built a culture

of inclusiveness and integrity

Joined Novartis in 2017 as Head Pharma Region

Europe, later appointed AAA President


Harry KirschChief Financial Officer

% USD % cc % USD % cc

Net Sales 11,764 4 8 22,870 3 8

Core Operating income 3,648 14 20 6,902 12 19

Operating income 2,663 10 17 4,905 2 11

Net Income 2,109 -73 -71 3,977 -59 -56

Core EPS (USD) 1.34 14 20 2.55 9 17

EPS (USD) 0.91 -73 -71 1.72 -59 -55

Free Cash Flow 3,612 11 5,481 6

Change vs. PYContinuing operations

1

USD million

Q2

2019

H1

2019

Change vs. PY

Summary of Q2 and H1 2019 continuing operations financial results


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 9 22 35.4 3.7 34.4 3.2

Sandoz 3 10 20.5 1.4 20.2 0.9

Continuing Operations 8 20 31.0 3.2 30.2 2.9

Q2 2019 H1 2019

Continuing operations in Q2 2019, delivering core margin expansion of 3.2%pts cc vs. PY

1


2

2

2

2

2



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

Barring unforeseen events (in cc)

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 mid to high single digit

IM Division revised upwards to grow mid to high single digit

Sandoz revised upwards to be broadly in line to low single digit growth

Core operating income revised upwards expected to grow low double digit to mid-teens


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

1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during 2019, is subject to the completion of customary closing conditions. 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

+ Innovative Medicines growth drivers

and launches uptake1

+ Productivity

− Potential increased Gx erosion2

− Potential resolution of valsartan

competitor supply shortage

H2 2019

Core OpInc growth with continued strong underlying momentum; potential generic headwinds in H2 2019

26

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

H1 2019

+ Innovative Medicines growth drivers

including Cosentyx® and Entresto®

+ Productivity

+ Valsartan competitor supply

shortage

1. Including Zolgensma®, Mayzent®, Aimovig®, Piqray®. 2. Mainly Exjade®, Afinitor® and Ophtha brands.


Illustrative

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

Expected currency impact for full year 2019

SimulationActual


2018

FX impact on

Net sales

FX impact on

Core operating income

1

-5 -4-1

-3

-9-6

-1-4

FY Q2 Q4Q1 Q4 FY Q2Q3 FY Q1 Q3 FY

00 0

2018 2019 2019

27

Vas NarasimhanChief Executive Officer

Conclusion: very strong first half 2019

Transformative innovation including the launches of Mayzent®,

Zolgensma® and Piqray®

Strong sales and margin expansion driven by double digit core operating

income growth

Catalyst rich second half, including readouts for

Entresto® PARAGON-HF, ofatumumab in MS and fevipiprant in asthma


Appendix

Net debt increased by USD 1.7bn mainly due to the annual dividend payment in Q1, partly offset by FCF


-16.2-17.9

-6.6 -0.4-2.4

-0.7

Alcon Net debt2Dec 2018 M&A transactions

from Continuing Ops1Jun 30, 2019Dividends Free Cash Flow from

Continuing Ops1

5.5

Treasury share

transactions, net

Others

2.9

-1.7

USD billion


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.

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

H1 2019 free cash flow at USD 5.5bn

5.2

H1 2019

5.5

H1 2018

+6%

Continuing operations1 free cash flow2

USD billion


1. Continuing operations as defined on page 42 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 measures. Further details regarding non-IFRS measures can be found starting on page 55 of the Condensed Financial Report.

2019 expected pipeline milestones

H1 2019 H2 2019

Regulatory

decisions and

opinions

Mayzent®1 SPMS (US) ✓ BYL719 HR+ Breast Cancer (US) ✓3

Kymriah® Ped / Young Adult r/r ALL (JP) ✓ Brolucizumab (RTH258) nAMD (US)

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

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

Zolgensma® SMA Type 12 (US / EU / JP) ✓ (✕2) Mayzent®1 SPMS (EU / JP)

Xolair® Pollinosis (JP)

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

Crizanlizumab (SEG101) Sickle Cell Disease (US / EU) ✓ Entresto® HFrEF (JP)

Mayzent®1 SPMS (JP) ✓ Entresto® HFpEF (US / EU)

INC280 NSCLC (US / JP)

Ofatumumab (OMB157) Relapsing MS (US / EU)

PDR001 (combination with

Tafinlar® + Mekinist®) Metastatic Melanoma (US / EU)

QVM149 Asthma (EU / JP) ✓4

Major trial

readouts

Zolgensma® SMA Type 2 IT Formulation ✓ Cosentyx® nr-axSpA

Zolgensma® SMA Type 1 / 2 /

presymptomatic✓ Entresto® HFpEF

Fevipiprant (QAW039) Asthma

Ofatumumab (OMB157) Relapsing MS

PDR001 (combination with

Tafinlar® + Mekinist®) Metastatic melanoma

✓ Achieved ✕ Missed

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. Zolgensma EU / JP approvals

expected in H2 2019. 3. Piqray® FDA approval achieved in H1 2019 4. QVM149 submitted in EU


17. Psoriatic arthritis head-to-head study versus

adalimumab

18. Non-alcoholic steatohepatitis

19. Ankylosing spondylitis head-to-head study versus

adalimumab

20. Acute myeloid leukemia

21. Chronic Obstructive Pulmonary Disease

22. Secondary Progressive Multiple Sclerosis

23. Myelodysplastic syndrome

24. 1st line colorectal cancer / 1st line renal cell

carcinoma

25. IT formulation Spinal Muscular Atrophy Type 2/3

26. Metastatic castration-resistant prostate cancer

Planned filings 2019 to 2023

ABL001CML4 3rd line

QGE031CSU16

Entresto®

Post-acute myocardial infarction

RTH258Diabetic macular edema

QAW039Asthma

Entresto®

Heart failure (PEF)13

INC280 NSCLC6

Cosentyx®

nr-axSpA12

OMB157Relapsing multiple sclerosis

2023202120202019 2022

Jakavi®Acute GVHD14

Combination abbreviations:

fulv fulvestrant

tmx tamoxifen

gsn goserelin

NSAI Non-steroidal aromatase inhibitor

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

35 Novartis Q2 2019 Results | July 18, 2019 | Novartis Investor Presentation

Cosentyx®

PsA H2H17

Cosentyx®

AS H2H19

LAM320a

MDR8 tuberculosis

ZPL389Atopic dermatitis

Rydapt®AML20 (FLT3 wild type)

UNR844Presbyopia

Jakavi®Chronic GVHD14

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanomaECF843

Dry eye

XolairNasal polyps

ACZ885Adjuvant NSCLC6

ACZ8851st Line NSCLC6

ACZ8852nd Line NSCLC6

Kymriah®

r/r Follicular lymphoma

Kymriah®

r/r DLBCL10 in 1st relapse

New molecule

New indication

New formulation

Biosimilars

1. Secondary prevention of cardiovascular events in patients with

elevated levels of lipoprotein (a)

2. Triple negative breast cancer

3. Paroxysmal nocturnal hemoglobinuria

4. Chronic myeloid leukemia

5. Long-acting release

6. Non-small cell lung cancer

7. Neovascular age-related macular degeneration

8. Multi drug resistant

9. Breast cancer

10. Diffuse large B-cell lymphoma

11. Head and neck squamous cell carcinoma

12. Non-radiographic axial spondyloarthritis

13. Preserved ejection fraction

14. Graft-versus-host disease

15. Neuroendocrine tumors

16. Chronic spontaneous urticaria

AVXS-201Rett syndrome

Zolgensma®

Spinal muscular atrophy type 2/325

Cosentyx®

Hidradenitis suppurativa

177Lu-PSMA-617mCRPC26

KAE609Malaria

KAF156 Malaria

LJN452NASH18

CAD106Alzheimer’s disease

HDM201Acute myeloid leukemia

CFZ533Solid organ transplant

CSJ117Severe asthma

LJC242NASH18

LMI070Spinal muscular atrophy

LNP023IgA nephropathy

QBW251COPD21

VAY785NASH18

VAY736Autoimmune hepatitis

ABL001CML4 1st line

LOU064CSU16

VPM087CRC 1L/RCC 1L24

MOR106Atopic dermatitis

SAF312Chronic ocular surface pain

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

TQJ230CVRR1

VAY736Primary Sjoegren’s syndrome

Kymriah+ pembrolizumab - r/r DLBCL10

CFZ533Sjoegren’s syndrome

LNP023Membranous nephropathy

BYL719 (Piqray® US)TNBC2

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)

a) WHO pre-qualification submission achieved April 2019

BYL719 (Piqray® US)HNSCC11 2/3L

BYL719 (Piqray® US)Ovarian cancer

MBG453MDS23

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


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





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


LJN452Non-alcoholic steatohepatitis

QBW251COPD

LMI070Spinal muscular atrophy

UNR844Presbyopia

VAY736Autoimmune Hepatitis

LOU064CSU3

LNP023IgA nephropathy

MOR106Atopic Dermatitis

177Lu-PSMA-617mCRPC26

ABL001CML1 1st line

VAY736Primary Sjoegren’s syndrome

ZPL389Atopic dermatitis

VAY785Non-alcoholic steatohepatitis

PDR001 comboMetastatic melanoma

Kymriah®

+ pembrolizumab - r/r DLBCL12

VPM087CRC 1L/RCC 1L24

CFZ533Sjoegren’s syndrome

LNP023Membranous nephropathy

Pipeline of key projects in confirmatory development

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


Combination abbreviations:fulv fulvestrant

ltz letrozole

tmx tamoxifen

gsn goserelin

NSAI Non-steroidal aromatase inhibitor

Taf Tafinlar® (dabrafenib)

Mek Mekinist® (trametinib)

a) Approved in US, submitted in EU.

Lucentis®

ROP16

SEG101Sickle cell disease

QAW039Asthma

RTH258nAMD6

ACZ885Adjuvant NSCLC2

ACZ8851st Line NSCLC2

INC280 NSCLC2

ABL001CML1 3rd line

QGE031CSU3

PDR001 + Tafinlar®+Mekinist®

Metastatic BRAF V600+ melanoma

New molecule

New indication

New formulation

Biosimilars

Lucentis®

Diabetic retinopathy

BAF312a EU (Mayzent™US)SPMS20

LAM320MDR15 tuberculosis

QMF149Asthma

QVM149Asthma

OMB157 Relapsing multiple sclerosis

RTH258Diabetic macular edema

Rydapt®AML18 (FLT3 wild type)

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

others (same as originator)

XolairNasal polyps

RTH258Retinal vein occlusion

Kymriah®

r/r DLBCL12 in 1st relapse

CAD106Alzheimer’s disease

KAE609Malaria

ECF843Dry eye

KAF156Malaria

HDM201Acute myeloid leukemia

CFZ533Solid organ transplant

Zolgensma®

Spinal muscular atrophy type 2/325

CSJ117Severe asthma

AVXS-201Rett syndrome

Zolgensma®a EUSpinal muscular atrophy type 123

1. Chronic myeloid leukemia

2. Non-small cell lung cancer

3. Chronic spontaneous urticaria

4. Triple negative breast cancer

5. Breast cancer

6. Neovascular age-related macular

degeneration

7. Secondary prevention of cardiovascular

events in patients with elevated levels of

lipoprotein (a)

8. Head and neck squamous cell carcinoma

9. Non-radiographic axial spondyloarthritis

10. Psoriatic arthritis head-to-head study versus

adalimumab

11. Ankylosing spondylitis head-to-head study

versus adalimumab

12. Diffuse large B-cell lymphoma

13. Preserved ejection fraction

14. Graft-versus-host disease

15. Multi-drug resistant

16. Retinopathy of prematurity

17. Severe aplastic anemia

18. Acute myeloid leukemia

19. Acute lymphoblastic leukemia

20. Secondary Progressive Multiple

Sclerosis

21. Myelodysplastic syndrome

22. Chronic Lymphocytic Leukemia

23. IV formulation Type 1 SMA

24. 1st line colorectal cancer / 1st line

renal cell carcinoma

25. IT formulation Spinal Muscular

Atrophy Type 2/3

26. Metastatic castration-resistant

prostate cancer

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

postmenopausal adv BC5 2nd line (+fulv)

Cosentyx®

nr-axSpA9

Entresto®

Heart failure (PEF)13

Entresto®

Post-acute myocardial infarction

Jakavi®Acute GVHD14

Cosentyx®

PsA H2H10

Cosentyx®

AS H2H11

Kisqali®

HR+, HER2(-) BC5 (adjuvant)

Jakavi®Chronic GVHD14

ACZ8852nd Line NSCLC2

Kymriah®

r/r Follicular lymphoma

Cosentyx®

Hidradenitis suppurativa

LJC242Non-alcoholic steatohepatitis

SAF312Chronic ocular surface pain

BYL719 (Piqray® US)HER2+ adv. BC5

BYL719 (Piqray® US)TNBC4

TQJ230CVRR7

LNP023C3 glomerulopathy

GP2411 (denosumab)2

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

BYL719 (Piqray® US)HNSCC8

BYL719 (Piqray® US)Ovarian cancer

MBG453MDS21

Promacta®/Revolade®

SAA17 1st line

Xiidra® EUDry eye


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






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

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



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



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





http://deis.novartis.intra/deis.asp?CAD106A


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




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






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. Q1 2019 presentation (1/2)

New trials added

Study Program Indication Phase Patients

NCT03769168 (CAIN457F2304E1) Cosentyx® Psoriatic arthritis Phase 3 64

NCT03785405 (CLCZ696B2319E1) Entresto® Heart failure in pediatric patients Phase 3 240

NCT03909295 (CLCZ696D1301E1) Entresto® Post-acute myocardial infarction Phase 3 63

NCT03570892 BELINDA (CCTL019H2301) Kymriah® 2nd line Diffuse large B-cell lymphoma (DLBCL) Phase 3 318

NCT03876769 CASSIOPEIA (CCTL019G2201J) Kymriah® 1st line high risk acute lymphoblastic leukemia (ALL) Phase 2 160

NCT03926611 (CLOU064A2201) LOU064 Chronic spontaneous urticaria Phase 2 308

NCT03946670 (CMBG453B12201) MBG453 Myelodysplastic syndrome Phase 2 120

NCT03650400 (CQAW039B2201) QAW039 Asthma Phase 2 24

NCT03814746 STAND (CSEG101A2301) SEG101Prevention of Vaso-Occlusive Crises (VOC) in patients with Sickle

Cell Disease (SCD)Phase 3 240

NCT03809611 (CUNR844A2203) UNR844 Presbyopia Phase 2 120

NCT03948334 ZESTExt (CZPL389A2203E1) ZPL389 Atopic dermatitis Phase 2 360


Key changes vs. Q1 2019 presentation (2/2)

Trials removed (operational decision-points achieved)

Study Program Indication Phase Patients

NCT03131453 GENERATION S2 (CCNP520A2202J) CNP520 Alzheimer’ s disease Phase 2B/3 2,000

NCT02435849 ELIANA (CCTL019B2202) Kymriah® Pediatric and young adult Relapsed/ refractory ALL Phase 2 95

NCT02180217 LINC-3 (CLCI699C2301) LCI699 Cushing's disease Phase 3 132

NCT02697734 LINC-4 (CLCI699C2302) LCI699 Cushing's disease Phase 3 69

NCT01682083 COMBI-AD (CDRB436F2301) Tafinlar®+Mekinist® BRAFV600 mutant adjuvant melanoma Phase 3A 874

NCT01698905 ENESTop (CAMN107A2408) Tasigna® Second line CML/CML-TFR Phase 2 163

NCT01844765 DIALOG (CAMN107A2203) Tasigna® Newly diag. CML and CML res/intol to imatinib/dasatinib Phase 2 59

NCT02712983 (CTBM100G2202) Tobramycin Bronchiectasis Phase 2 105


Cardio-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 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

• Nov-2018 AHA: Primary data presentation /

simultaneous publication (Velazquez E, et al. NEJM

2019)

• 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)

• 3Q-2019 ESC: Secondary abstracts submitted

• ESC 2018: Primary presentation of 10wk txt period data;

Primary manuscript submitted EJHF; if accepted,

planned publication 2Q-2019

• Secondary data presentations: 2Q-2019 ESC-HF: 26wk

extended treatment data, and “ de novo HF” and

ACEI/ARB naive subgroups; 3Q-2019 ESC: target doses

achieved, 30-day re-hospitalization, NTproBNP

biomarker, and signs and symptoms / NYHA class at

10wk and 26wk time points


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 TBD TBD



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 Q4-2019

Publication Planned in Q3-2019 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)

Indication Kidney transplantation

Phase Phase 2B

Patients 325

Primary Outcome

Measures

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

months post-transplantation and post conversion (for

maintenance cohort)

Arms/Intervention• CFZ doses + MMF + corticosteroids

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

Target Patients Kidney transplant recipients

Expected Completion 2021

Publication TBD


Cosentyx® - Anti IL-17

48

Study NCT01544595 (CAIN457A2302E1 – extension study) NCT03589885 MATURE (CAIN457A2325)

Indication Psoriasis Psoriasis


Patients 1,146 120

Primary Outcome

Measures

Cumulative rate of subjects with loss of psoriasis area and

Cumulative rate of subjects with loss of Psoriasis Area and

Severity Index (PASI) 75 response up to week 68 (time = 0

being defined as week 52)

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

12 weeks of treatment

Arms/Intervention

• Secukinumab 150 mg


• Placebo

• 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

Patients with moderate to severe chronic plaque-type

psoriasis completing preceding psoriasis phase III studies

with secukinumab

Subjects with moderate to severe plaque Psoriasis

Expected Completion 2017 (actual) H2-2020

Publication

• 2-years results: Br J Dermatol. 2017 May 12. doi:

10.1111/bjd.15656

• 5-year results presented at AAD Mar-2019 (Late-

breaking Research) - Secukinumab Maintains

Improvements in Psoriasis through Five Years of

Treatment: A Randomized Extension of the Phase III

ERASURE and FIXTURE Trials

TBD



49

Study NCT02471144 (CAIN457A2310) NCT03066609 (CAIN457A2318)



Patients 169 543

Primary Outcome

Measures

The percentage of Participants achieving a 75%

Improvement from Baseline in PASI Score at week 12

Psoriasis Area and Severity Index (PASI) 75 response and

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

week 12

Arms/Intervention

• Secukinumab low dose

• Secukinumab high dose

• Placebo

• Etanercept (comparator)



• Placebo

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

chronic plaque psoriasis

Patients with moderate to severe chronic plaque-type

psoriasis with or without psoriatic arthritis comorbidity

Expected Completion 2023 Q1-2019 (actual)

Publication TBD

Week 16 results: Poster presented at: 2019 American

Academy of Dermatology (AAD) Annual Meeting,

March 1– 5, 2019, Washington, D.C.



50

Study NCT02826603 CLARITY (CAIN457A2326) NCT03668613 (CAIN457A2311)


Phase Phase 3B Phase 3

Patients 1,102 80

Primary Outcome

Measures



week 12



week 12

Arms/Intervention• Secukinumab 300 mg

• Ustekinumab 45 mg/ 90 mg

• Secukinumab low dose

• Secukinumab high dose

Target Patients Patients with moderate to severe plaque psoriasisPediatric patients of age 6 to <18 years, with moderate to

severe plaque psoriasis

Expected Completion Q3-2018 (actual) 2023

Publication

• Abstract Winter Clin Derm (US) Jan-2018

• Abstract to EADV in 2018

• Submission Journal (16wk 1ry EP IA) Q3-2018

(ongoing)

• Encore Abstract AAD 2019

TBD



51

Study NCT02748863 ALLURE (CAIN457A2323)

Indication Psoriasis

Phase Phase 3

Patients 214

Primary Outcome

Measures


Investigators' Global Assessment (IGA) 0 or 1 response

Arms/Intervention

• Secukinumab 300 mg (2 mL PFS device)

• Secukinumab 300 mg (2 x 1 mL PFS device)

• Placebo

Target Patients Adult subjects with moderate to severe plaque psoriasis

Expected Completion Q3-2018 (actual)

Publication• Submission Journal TBC Q2-2019

• Abstract at AAD in 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 submitted in Q4-2017

• 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



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) results: Abstract to be submitted to ACR

2019

• 5 year (EOS) manuscript to start in Jun-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 targeted for submission end

April 2019



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: manuscript targeted for submission

end of Apr-2019

• 52 week result abstracts presented at EULAR and ACR

2018

• 52 week manuscript targeted for submission end of April

2019

• 2 year (EOS) results to be submitted as late-breaker for

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

TBD



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: H1-2020, Final: 2021 2022

Publication TBD 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

• Manuscripts in 2019: submit both QoL overall and AIDAI in Q3-2019; crFMF efficacy & safety in Q4-2019

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

• Manuscript in 2019: Remission & flexible dosing to be submitted in Q3-2019


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 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 matching placebo in the evening

from Day 1 to 85

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

Day 1 to 85

• 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-IgE

Study NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)

Indication Chronic spontaneous urticaria Chronic spontaneous 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



• Ligelizumab 120mg single dose

• Omalizumab 300mg q4wks

• Placebo q 4wks

Ligelizumab 240 mg q4wks open label

Target Patients Patients with chronic spontaneous urticaria Patients with chronic spontaneous urticaria

Expected Completion 2017 (actual) Q2-2019 (actual)

Publication

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

GUF 2018; manuscript submitted (publication expected Q3-

2019)

Primary results: Late breaker abstract (showing 1 year

treatment results) presented at AAD in Q1-2019; re-

treatment data and safety overview presented at EAACI

2019; manuscript submission expected in Q1-2020


QGE031 - Anti-IgE

Study NCT03437278 (CQGE031C2202)

Indication Chronic spontaneous urticaria

Phase Phase 2

Patients 48

Primary Outcome

MeasuresChange in the 7 day Urticaria Activity Score (UAS7)

Arms/Intervention

• Ligelizumab high dose q4wks

• Ligelizumab low dose q4wks

• Placebo / ligelizumab high dose q4wks

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

spontaneous urticaria


Publication Manuscript to be submitted in 2021


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

• Ligelizumab dose B q4w

• Omalizumab 300 mg q4w

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk48

• Ligelizumab dose A q4w

• Ligelizumab dose B q4w

• Omalizumab 300 mg q4w

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk48

Target PatientsAdolescents and adults with chronic spontaneous urticaria

and inadequately controlled with H1-antihistamines

Adolescents and adults with chronic spontaneous urticaria

and inadequately controlled with H1-antihistamines


Publication TBD TBD


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

Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)

Indication Primary sjogren's syndrome Autoimmune hepatitis


Patients 180 80

Primary Outcome

Measures

Safety and efficacy of VAY736 in primary sjogren'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 sjogren's

Syndrome (pSS)

Autoimmune hepatitis patients with incomplete response or

intolerant to standard treatment of care

Expected Completion H2-2020 2023

Publication TBD TBD


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


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 H1-2020 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


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 H1-2020

Publication Planned in 2019 TBD


CNP520 - BACE inhibitorCAD106 - active beta-amyloid immunotherapy

74

Study NCT02565511 GENERATION S1 (CAPI015A2201J)

Indication Alzheimer’ s disease

Phase Phase 2B/3

Patients 1,340

Primary Outcome

Measures

Time to diagnosis of MCI due to Alzheimer's disease or

dementia due to Alzheimer's disease

Change in the Alzheimer's Prevention Initiative Composite

Cognitive (APCC) Test Score

Arms/Intervention

• CAD106 450 µ g + Alum 450 µ g i.m.

• Placebo to CAD106 + Alum 450 µ g i.m.

• CNP520 50 mg oral

• Placebo to CNP520 oral

Target PatientsCognitively unimpaired participants aged 60 to 75 years,

with two APOE4 allele (Homozygotes )


Publication TBD


Gilenya® - S1P-R modulator

75

Study NCT01633112 ASSESS (CFTY720D2312) NCT01201356 LONGTERMS (CFTY720D2399)

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



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 – planned in 2019

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

ECTRIMS 2017

• Primary manuscript submitted in May 2019 (estimated

publication August 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


OMB157 - Anti-CD20

77

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 TBD TBD


OMB157 - Anti-CD20

78

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

80

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

Expected Completion H2-2020 H1-2021

Publication 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 TBD Abstract submitted Q2-2019 (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 Abstract submission to congress in H2-2019

BYL719 - Alpha-specific PI3K inhibitor

83

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 (accepted Q1-

2019, not yet published)


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

84

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 submitted Q1-

2019


INC280 - MET Inhibitor

85

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-naï ve pts. with MET dysregulation

• Pre-treated pts with MET dysregulation – second

line

• Treatment-naï ve 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


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

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


Jakavi® - JAK1/2 inhibitor

86

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 H1-2020 (journal TBD) • Manuscript submission in H1-2020


Jakavi® - JAK1/2 inhibitor

87

Study NCT03491215 REACH4 (CINC424F12201)

Indication Graft versus host disease

Phase Phase 2

Patients 39

Primary Outcome

Measures

• Measurement of PK parameters

• Overall Response Rate (ORR)

Arms/Intervention • Ruxolitinib

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

after allogeneic hematopoietic stem cell transplantation


Publication TBD


Kisqali® - CDK 4/6 inhibitor

88

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

89

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

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

90

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

91

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

92

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

93

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)

Expected Completion H1-2020 H2-2022

Publication TBD TBD


Rydapt®- Multi-targeted kinase inhibitor

94

Study NCT03512197 UNIFY (CPKC412E2301)

Indication Acute myeloid leukemia

Phase Phase 3

Patients 502

Primary Outcome

MeasuresEvent Free Survival ( EFS)

Arms/Intervention• Midostaurin 50 mg

• Placebo

Target PatientsNewly diagnosed patients with FLT3 mutation negative

acute myeloid leukemia (AML)


Publication TBD


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


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


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


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 TBD


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

Expected Completion Q1-2020

Publication TBD

Tafinlar® - BRAF inhibitor


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


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 TBD


Zykadia® - ALK inhibitor


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): 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

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


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: submitted in Dec-2018 to NEJM,

resubmitted and accepted by Nature in May-2019

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 µ L

• Aflibercept 2 mg/50 µ L

• RTH258 3 mg/50 µ L

• RTH258 6 mg/50 µ L


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


RTH258 - Anti-VEGF

Study NCT03386474 (CRTH258A2301E1)

Indication Neovascular age-related macular degeneration (nAMD)

Phase Phase 3

Patients 150

Primary Outcome

MeasuresNumber of treatment-emergent adverse events

Arms/Intervention• RTH258 6 mg/50 µ L


Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study


Publication TBD


RTH258 - Anti-VEGF

Study NCT03481634 KESTREL (CRTH258B2301) NCT03481660 KITE (CRTH258B2302)

Indication Diabetic eye disease Diabetic eye disease


Patients 534 356

Primary Outcome

Measures

Change from baseline in best-corrected visual acuity

(BCVA)

Change from baseline in best-corrected visual acuity

(BCVA)

Arms/Intervention

• RTH258 3 mg/50 µ L

• RTH258 6 mg/50 µ L

• Aflibercept 2mg/50 uL

• RTH258 6 mg/50 µ L


Target PatientsPatients with visual impairment due to diabetic macular

edema (DME)

Patients with visual impairment due to diabetic macular

edema (DME)


Publication TBD TBD


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


Respiratory

QAW039 – DP2 receptor antagonist

111

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


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



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


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

114

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 µ g od

• QMF149 150/320 µ g od

• MF 400 µ g od

• MF 400 µ g bid

• Salmeterol 50 µ g /fluticasone 500 µ g bid

• QVM149 150/50/160 µ g od

• QVM149 150/50/80 µ g od

• QMF149 150/160 µ g od

• QMF149 150/320 µ g od

• Salmeterol 50 µ g /fluticasone 500 µ g 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 in H1-2020



116

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 in H1-2020



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 µ g od

• MF 200 µ g 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


Xolair® – anti-IgE antibody

118

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 will be submitted for EAACI (the European Academy of Allergy and Clinical Immunology), Jun-

2019

• Oral/Poster will be submitted for JRS (Japanese Rhinologic Society), Oct-2019

• Manuscript will be submitted to JACI (The Journal of Allergy and Clinical Immunology), Q2-2019


Sandoz Biopharmaceuticals

Hyrimoz® - Biosimilar adalimumab

120

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


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

• Manuscript with study results journal TBD, Q3 2019


Global Health

KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated

122

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

123

Study NCT03334747 (CKAE609A2202)

Indication Malaria

Phase Phase 2

Patients 150

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

Q2 2019 Results - Novartis

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