q4 and fy 2019 results - novartis

Q4 and FY 2019 ResultsInvestor Presentation

January 29, 2020

Novartis AG

Investor Relations

Disclaimer

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,” “may,” “could,” “would,” “anticipate,” “seek,” 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 development or adoption of potentially transformational technologies, treatment modalities and business models; or regarding potential future or pending transactions,

including 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 share buybacks; 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; uncertainties regarding potential significant breaches of data security or data privacy, or disruptions of our information

technology systems; regulatory actions or delays or government regulation generally, including potential regulatory actions or delays with respect to the proposed transactions or the

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

acquisition of the Japanese operations and associated assets of Aspen Global Incorporated, may not be completed in the expected time frame, or at all; the potential that the

strategic benefits, synergies or opportunities expected from the acquisition of The Medicines Company, the proposed divestiture of certain portions of our Sandoz Division business in

the US, or the planned acquisition of the Japanese operations and associated assets of Aspen Global Incorporated, and other transactions described, may not be realized or may be

more difficult or take longer to realize than expected; the successful integration of The Medicines Company into the Novartis Group and the timing of such integration; potential

adverse reactions to the transaction by customers, suppliers or strategic partners; dependence on key personnel of The Medicines Company; dependence on third parties to fulfill

manufacturing and supply obligations; the uncertainties involved in predicting shareholder returns; the uncertainties 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 is expected to continue this year; safety, quality, data

integrity, or manufacturing issues; uncertainties involved in the development or adoption of potentially transformational technologies and business models; uncertainties regarding

actual or potential legal proceedings, including, among others, product liability litigation, disputes and litigation with business partners or business collaborators, government

investigations generally, litigation and investigations regarding sales and marketing practices, and intellectual property disputes; our performance on environmental, social and

governance measures; general political, economic and trade conditions, including uncertainties regarding the effects of ongoing instability in various parts of the world; uncertainties

regarding future global exchange rates; uncertainties regarding future demand for our products; 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.

Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation 2

Participants

Vas NarasimhanChief Executive Officer

Harry KirschChief Financial Officer

Marie-France TschudinPresident, Novartis Pharmaceuticals

Susanne SchaffertPresident, Novartis Oncology

John TsaiHead of Global Drug Development and CMO

Richard SaynorCEO, Sandoz

Shannon Thyme KlingerGroup General Counsel


In 2019, we kept executing on our strategyFocus Novartis as a leading medicines company powered by advanced therapy platforms and data science

Our focus

Strengthen our core

Accelerate

key geographies

Our priorities

Unleash the power of

our people

Deliver transformative

innovation

Embrace operational

excellence every day

Go big on data and

digital

Build trust with society

Focus our company

and capital


We have focused Novartis as a medicines company

1920 - 1996 1996 - 2009 2009 - 2017 2018 - 2019

Medicinal chemistry

and industrials

Portfolio

transformation

Diversified

healthcare groupFocused medicines company powered by

advanced therapy platforms and data science

1. OTC – Consumer Healthcare 2. Alcon market capitalization on close of 1st day of trading 3. USD 3.4bn upfront + potential milestone payments of up to USD 1.9bn

Acquired Divested OTC1 Acquired Acquired Spun off AcquiredAcquired

USD 8.7bnUSD 3.9bn USD 2.1bn USD 3.4bn3 USD 9.7bnUSD 28bn2USD 13bn


Leading pipeline with strong replacement power

Scale Value Innovation

114 PHASE 1 / 2

37 PHASE 3

13 REGISTRATION

Estimated 2024 sales from

products launched between 2019-242

# of projects1

1. Including Global Health, excluding Sandoz. 2. Innovative medicine product sales excl. Vaccines and LCM products (e.g. new formulations, combinations with off patent molecules); compound-based analysis (Phase 2 and 3) with additional

indications allocated to 1st launch. Inclisiran included. Source: Novartis peer group analysis based on data from Evaluate Pharma (download from November 27, 2019)

16Advanced platform

therapies

in clinical development#1 Replacement power

~90%Pipeline potentially

first-in-class /

first-in-indication

~80% Target areas of high

unmet need

Company A

Company B

Company C

Company G

Company D

Company E

Company F

Company H

Company I

Transformative innovation


2019 was a breakthrough year for innovation

30+ clinical data readouts

Select examples

Zolgensma®

Cosentyx®

Ofatumumab

Entresto®

Fevipiprant

Kisqali®

INC280

Inclisiran1

30+ major submissions

Select examples

Entresto® (JP)

Cosentyx® nr-AxSpA (US/EU)

Ofatumumab (US)

Adakveo® (US/EU)

Beovu® (US/EU/JP)

INC280 (US)

QVM149 / QMF149 (EU/JP)

Inclisiran (US)1

5 NME approvals of potential blockbusters

aSPMS

SMA

Breast cancer

Wet AMD

Sickle cell disease

aSPMS – Active secondary progressive multiple sclerosis SMA – Spinal muscular atrophy AMD – age-related macular degeneration 1. Readout / submission by The Medicines Company



Potential catalysts Select examples

Major approvals1 Ofatumumab (OMB157)

Relapsing MS

Capmatinib (INC280)

NSCLC

Cosentyx®

nr-axSpA

QVM / QMF 149

Asthma

Entresto®

HFpEF (US)

Inclisiran (KJX839)

Hyperlipidemia (US)

Major submissions2 Inclisiran (KJX839)

Hyperlipidemia (EU)

AVXS-101 IT4

SMA

Alpelisib (BYL719)

PROS

177Lu-PSMA-617

mCRPC

Spartalizumab (PDR001) combo

Metastatic melanoma

Entresto®

HFpEF (US)

Major readouts3

(Phase 3)

177Lu-PSMA-617

mCRPC

Beovu®

DME

Entresto®

Post-acute MI (IA5)

Asciminib (ABL001)

Chronic Myeloid Leukemia

Kisqali®

Breast cancer (MONALEESA-2 OS)

Jakavi®

Chronic GvHD

Phase 3 starts TQJ230

CVRR

LNP023

PNH

MBG453

MDS

Tropifexor (LJN452)

NASH

Alpelisib (BYL719)

Multiple indications6

Beovu®

PDR

2020 catalysts maintaining long-term momentum

1. First approval in any market. 2. First submission in any market 3. Readouts enabling submission, label change or pivotal trial initiation 4. FDA placed a partial clinical hold based on findings in a small preclinical animal study 5. Planned

interim analysis expected Q1 2020 (full readout 2021) 6. HER2+ aBC, TNBC, ovarian cancer, head and neck cancer, PROS



Innovation driving growth in China

1. NDA approvals of new compounds and new indications

Number of NDA approvals1

Growth driven by regulatory approvals from innovative

pipeline, market access and optimizing resources

13 NME approvals over the past 5 years, and 22 NRDL

listings since 2017

Average # of NDAs expected to double in the next 5 years

5 average NDA approvals / year 2015-2019

10 average NDA approvals expected / year 2020-2024

50+ NDA approvals planned over the next 5 years

Goal to deliver >90% of 2024+ China submissions

simultaneously with global submission

25

50+

2015-2019Actual

x2

2020-2024Expected



Delivered strong performance in 2019

47.4 bnNET SALES (USD)

+9%vs. 2018 (cc2)

33.5%IM CORE MARGIN2(%)

+1.8% ptsvs. 2018 (cc2)

12.9 bnFREE CASH FLOW2 (USD)

22.3%1-YEAR TSR3 (%)

Top tierRANKING 3

+15%vs. 2018 (USD)

Continuing operations1, FY 2019, TSR as of YE 2019

1. Continuing operations as defined on page 45 of the Condensed 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 58 of the Condensed Financial Report. 3. TSR in USD from

Jan 1st 2019, using 1 day average price at start and 3 month average price at end; ranking when compared to the global HC peer group as defined in the Novartis 2019 Annual Report

14.1 bnCORE OPERATING INCOME2 (USD)

+17%vs. 2018 (cc2)

5.28CORE EPS2 (USD)

+17%vs. 2018 (cc2)

Operational excellence


Strong operational performance from growth drivers

Selected growth driver sales

nm – not meaningful

Lutathera®

Growth drivers and recent launches

as % of Innovative Medicines sales

2019201820172016

Adakveo®

Jakavi®

Ilaris®

Lutathera®

Mayzent®

Zolgensma®

Kisqali®

Xiidra®

Promacta®

Luxturna®

Tafinlar®+Mekinist®

Kymriah®

Piqray®

Beovu®

Cosentyx®

Entresto®

Aimovig®

Xolair®

117

134

137

183

116

192

245

698

361

274

242

714

202

SalesUSD Million

Growth vs. PYUSD Million

Growth vs. PYcc

3,551 28%

1,726 71%

361 nm

441 160%

480 111%

1,416 23%

278 nm

192 nm

1,338 20%

1,114 20%

1,173 19%

671 25%

116 nm

14%

20%

27%

35%



Focused on launch excellence for 15 ongoing and upcoming major launches

Adakveo®

Sickle cell disease

Beovu®

Wet AMD

Piqray®

Breast Cancer

Mayzent®

aSPMS

Zolgensma® IVSMA

2019 2020

QVM / QMF 149Asthma

Cosentyx®

nr-axSpA

Capmatinib (INC280)NSCLC

Ofatumumab (OMB157)Relapsing MS

Ongoing Upcoming

2021

PDR001 comboMetastatic melanoma

177Lu-PSMA-617mCRPC

Alpelisib (BYL719)PROS

AVXS-101 ITSMA

Entresto®

HFpEF

Inclisiran (KJX839)Hyperlipidemia



Zolgensma® strong launch (June) with FY 2019 sales of USD 361m

Broad access, strong patient demand

Intrathecal formulation on partial clinical

hold – regulatory discussions ongoing

CHMP opinion anticipated Q1 2020

Approval anticipated in H1 2020

OthersDecisions anticipated late 2020 or early

2021 in Switzerland, Canada, Australia

Patients treated commercially

Commercial lives covered

Medicaid lives covered

Newborns screened1

Approval rate for on-label patients

Global Managed Access Program initiated

Next steps

100 200

90% 97%

30% >50%

30% 39%

>99%

1. Expected to reach 70% by end of 2020

Q3 end (~) YE (~)



Committed to driving consistent margin expansion

Innovative MedicinesCore margin (% rounded)

+ Sales momentum of key growth drivers and

operational excellence on upcoming launches

+ Productivity programs in Novartis Technical

Operations and Novartis Business Services

+ Resource allocation in commercial units

‒ Generic erosion

‒ Launch investments for potential future

blockbusters, including inclisiran2019

3231

20182017

Mid

30s

near term medium

term

34

Mid to high

30s



Advancing deep transformations in NTO and NBS

NTO – Novartis Technical Operations; Baseline EOY 2016 NBS – Novartis Business Services; Baseline EOY 2018 NGSCs – Novartis Global Service Centers API – Active pharmaceutical ingredients REFS – Real estate and facility services

On track for ~USD 2bn savings by end of 2020; new efforts expected to deliver additional ~USD 1.5bn savings medium term

Strategic levers NTO NBS

Organization Reduced ~1,800 FTEs across global functions and site operations (net of site exits)

On track to reduce ~600 FTEs by 2022

Associates in NGSCs from <40% to >50%

Footprint 10 sites exited

9 additional exits announced

102 out of 210 warehouses consolidated

Footprint reduction through sale, lease-back, and Activity-Based Working (20+ offices)

Moved to single service provider for REFS

Procurement 28% reduction in suppliers for finished product, API

45% reduction in suppliers for indirect materials

New Chief Procurement Officer

Optimizing terms with top 100 suppliers

Data & Digital Delivering advanced analytics solutions on asset, material and inventory management

Continued investments in tech enablers

Re-design of key enterprise processes



Sandoz delivered accretive growth in 2019 by implementing refined strategy

Geographic

priorities

EU: Solidifying #1 position

JP: Closing Aspen acquisition and investing

US: Stabilizing the business

In the process of concluding oral solids

business divestment

Launching pegfilgrastim

Increasing

autonomy Creating Sandoz TechOps organization

Portfolio

update

Building biosimilar pipeline further -

trastuzumab / natalizumab deals

Appealing US Erelzi® decision

Gx Advair® discontinued further development

Sales growth (vs. PY, cc)

+2%

+16%

EX-US

GLOBAL

BIOPHARMACEUTICALS

+7%

+10%

Core operating income growth (vs. PY, cc)

Refined strategy



Four core elements to our digital transformation

Scale 12 digital

lighthousesMake Novartis digital

Become the #1 partner

in tech ecosystemPursue bolder moves

Spanning the entire value

chain, from development to

commercial operations

In full flight with 2-3 year

implementation horizon

Investing in technology

platforms, including CRM,

MDM, API

>1,500 associates mobilized

Rapidly build Data Science

and AI capabilities

Move to One Digital global

collaboration platform

Dedicated leadership

capability program

Scale novel partnership

accelerator: the Novartis

Biome

Complement internal skills

and capabilities

Closely linked to business

priorities

Getting ready for disruptive

healthcare scenarios through

large-scale alliances, e.g.:

Microsoft: AI Innovation Lab

AWS1: TechOps optimization

Tencent: Heart Failure

patient solution in China

1. AWS: Amazon Web Services

Go big on data and digital


Broad set of initiatives to drive culture change

Connect to our purpose

and provide an inspiring

working environment

Build leadership

self-awareness and

capabilities

Inspired Unbossed

Go big on learning

Curious

18 countries visited by CEO in 2019

Unbossed Leadership Experience

(ULE) to be completed in 2020 for the

top 300 leaders in the company

Coursera: ~3,500 courses completed

by 7,000+ users (~85,000 hours)1

LinkedIn Learning: ~14,000 courses

available, 12,500+ users1

Spark live to 83,000 associates,

230,000 recognitions given in 2019

Minimum 14 weeks paid leave for all

parents, regardless of gender

Unleash the power of our people

1. As of YE 2019


In 2019, we made important progress on our efforts to build lasting trust with society

Ethical Standards

Rolled out globally the new Third

Party Risk Management system

Drafting the new Code of Ethics,

co-created with our associates

Pricing & Access

Brought LIC & LMIC prices in

line with EU5 average

Outlined new access strategy

for Sub-Saharan Africa

Global Health

Signed partnership for SCD

with the Government of Ghana

Joined Global Chagas Disease

Coalition

Corporate Citizenship

Joined the UN Equal Pay

International Coalition

Achieved 44% female

representation in management



Introducing ambitious 2020 ESG targets which are deeply embedded in our operating model

Holistic set of ESG targets for 2020... ... deeply embedded in our operating model

Systematically reviewed

Tracked bi-monthly at the Trust & Reputation

Committee, a sub-committee of the Executive

Committee of Novartis (ECN) chaired by the CEO

Linked to compensation

Cascaded into ECN personal objectives, and directly

impacting compensation

Transparently disclosed

To be included in 2020 Annual Report, providing

disclosure on our goals and progress

Pillar Target

Ethical

Standards

Transparency on clinical trials

Strengthen Third Party Risk Management

Fully integrate Human Rights into TPRM

Pricing

& Access

Increase patient reach

Enhance access

Implement pricing principles

Global

Health

Malaria: Advance development of new drugs

Sickle cell disease: Expand coverage

Chagas: Progress on clinical trial

Corporate

Citizenship

Reduce energy & carbon

Reduce waste

Reduce water



Bold long-term aspirations across the ESG spectrum

Reduce launch time lag

to 3 months in LMICs

Implement access

strategy for advanced

therapies in LMICs

Transform treatment of

malaria with USD 100m

committed in R&D1

Holistically address

sickle cell disease in

Ghana

Achieve carbon

neutrality in own

operations by 2025

Deliver on UN EPIC

and LGBTI equity

pledges

1. Commonwealth Heads of Government Meeting April 2018, commitment over the next 5 years LMICs – low and middle income countries


Select examples

www.novartis.com/nisreport2019


Inclisiran

TQJ230

LNP023

Iscalimab

Ligelizumab

AD portfolio2

LNA043

Tropifexor

Ofatumumab

UNR844

QVM / QMF149

MBG453

Asciminib

Canakinumab

Capmatinib

Spartalizumab177Lu-PSMA-617

In-market growth drivers, major launches and a rich pipeline expected to sustain our long-term growth

In-market

growth drivers

Major launches

New indications

Novel assets

15 ongoing / upcoming major launches

80+ major submissions planned to 2022

50+ late stage programs1Cosentyx® HS

Cosentyx® GCA

Cosentyx® LP

Cosentyx® JIA

Cosentyx® LN

Entresto® post-AMI

Beovu® DME

Beovu® RVO

Beovu® DR

Beovu® PDR

Ofatumumab pediatric

AVXS-101 IT

Alpelisib PROS

Piqray® TNBC

Piqray® HER2+ aBC

Piqray® ovarian cancer

Piqray® HNSCC

Kisqali® HR+/HER2- BC (adj)

Kymriah® FL

1. Ph3 / in registration 2. AD portfolio – atopic dermatitis portfolio incl. ZPL389, CEE321

SELECT EXAMPLES


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

Strong Pharmaceuticals growth driven by Cosentyx®, Entresto® and recent launches

Strong sales momentum driven by growth drivers

Mainly Cosentyx® and Entresto®

Maintaining solid performance of established products

Focus on launches to deliver next phase of growth

Launched Zolgensma®, Beovu® and Mayzent® in US

Xiidra® acquired and integrated into Ophtha franchise

Added inclisiran to CRM pipeline

Pharmaceuticals net sales USD billion, growth in % cc

0.7

15.5

2019

3.9

16.1

2017

16.0

5.5

2018

23.3

7.1

20.021.5

+7%

+12%

Established products3Key growth drivers1 Recent launches2

CRM – Cardiovascular, Renal and Metabolism 1. Cosentyx®, Entresto®, Xolair®, Ilaris® 2. Zolgensma®, Xiidra ®, Aimovig®, Luxturna®, Mayzent® and Beovu® 3. All other brands


Cosentyx® Q4 sales driven by strong demand and broad access across indications and regions

Strong performance, above market

Q4 sales up +21% cc

PsO TRx outperform market (+27% YoY vs. +12%)1

SpA TRx growing >2x faster than market2

Maintain momentum

nr-axSpA submitted to FDA in Dec

Pediatric PsO submitted to EMA in Nov

Expected to maintain broad access in 2020

Upgrading expected peak sales >USD 5bn

Cosentyx® sales evolutionUSD million

Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

2.8bn

580701

750806

2018 2019

791858

937

3.6bnEx-US

US

965

1. IQVIA US National Prescription Audit for Dermatology WE 12/20/2019; market includes Enbrel®, Humira®, Siliq®, Skyrizi®, Stelara®, Taltz®, Tremfya® 2. IQVIA US National Prescription Audit for Rheumatology WE 12/20/2019; SpA market

includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz® All trademarks are the property of their respective owners


Entresto® solidifying leadership position in 2019 as an essential first-choice treatment in heart failure

200239

271318

357421 430

518

Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

1.0bn

1.7bn

2018 2019

AHA – American Heart Association; QoL – Quality of Life; NRDL – National Reimbursement Drug List; HFpEF – Heart Failure with preserved Ejection Fraction

Entresto® sales evolutionUSD million, cc

Ex-US

US

Strong momentum

Q4 sales +65% driven by increased demand in hospital

and ambulatory settings

US TRx growth (+48% vs. PY)

Poised for further growth acceleration

New data on reverse cardiac remodeling, in-hospital use

and QoL

Inclusion on China NRDL, supporting expanded use

US regulatory submission for HFpEF on track for Q1

Japan launch expected in H2


Broad access and reimbursement

Strong start to Beovu® US launch with excellent customer feedback and broad access

Strong uptake in 3 months since launch1

EU approval expected Q1 2020, JP approval expected Q2 2020

90%

of US retina specialists have

Beovu® available in their office84%

of retina specialists who don’t

currently use Beovu® plan to

use it in next 6 months

permanent J-Code received,

providing greater reimbursement

confidence

Jan 1

positive benefits verification

outcomes to date295%

1. Novartis commissioned Retina Specialist Panel Online Survey, Dec 2019. 2. Covered or covered with restrictions


Ofatumumab (OMB157) is poised to set a new standard for simple, broad and early B-cell therapy adoption

CDW – Confirmed Disability Worsening Source: ASCLEPIOS I & II data presentation available here 1. Adapted from the OPERA trials, Hauser et al. 2017. Different to the ASCLEPIOS study, a disability “progression” was defined as an

increase from the baseline EDSS score of at least 1.0 point (or 0.5 points if the baseline EDSS score was >5.5) that was sustained for at least 12 (24) weeks, used in OPERA trials

HR (95% CI): 0.541

(0.381; 0.768)

TER 10.5%

OMB 5.6%

Study month

K-M

estim

ate

of cu

mu

lative

eve

nt ra

te [%

]

TER 14mg

OMB 20mg24

22

20

18

16

14

12

10

8

6

4

2

0

0 3 6 9 12 15 18 21 24 27

Regulatory file submitted in US and EU

Easy-to-use autoinjector available upon launch,

enabling simple at-home self-administration and

improved patient experience

Strong scientific presence at major congresses

and in markets through 2020

Engaging with payers on rapid, broad early

access

45.9% risk reduction (p<0.001) in 24-week CDW post-hoc

analyses with revised definition used in OPERA trials1

Unsurpassed efficacy and favorable safety supporting broad and early use in RMS


Efficacy Potent, durable, consistent LDL-Creduction >50%

Safety Profile similar to placebo (no liver, muscle, renal nor platelet signals)

in entire clinical program

Convenience Durable efficacy with only 2 subcutaneous injections per year,

less patient abandonment

Adherence Payers confidence reinforced by physician administration dosing

regimen

Inclisiran (KJX839), preparing to launch potentially transformative cholesterol-lowering therapy

1. Regulatory submissions filed by The Medicines Company

Organizational MDCO now subsidiary of Novartis, expected to be fully integrated end March 2020

Regulatory US and EU files submitted1

JP bridging program in progress

CN local development aligned with CFDA

Commercial Finalizing hiring and training of US teams

Value-based pricing and flexible access strategy

Engaging with payers and health systems to

enable broad and affordable access, including

population models (e.g. UK NHS)

Scaling up supply

Preparing for launchDifferentiated asset


Cosentyx®: nr-axSpA launch

doubles addressable axSpA

population

Entresto®: NRDL-driven expansion

in CN, JP approval expected

Beovu®: Global rollout and DME /

RVO expansion

Ofatumumab: Broad and early

access in CoEs and community

Inclisiran: Broad & affordable access

Mayzent®: Urgency to treat and

patient services optimization

Xiidra®: Return to growth

TQJ230: Lp(a) awareness and

testing

Iscalimab: Educate on unmet need,

leveraging transplant legacy

Ligelizumab: Build US infrastructure

Tropifexor: Drive disease awareness

given asymptomatic nature of NASH

LNP023: Educate physicians on

complement-driven renal &

hematologic diseases

Focus in 2020 will be launches and preparing for next big bets, building on the strong foundation

Maximize growth drivers

Deliver on new launches

Prepare for next big bets


Strong Oncology growth driven by recent launches and growth drivers

+ Strong uptake of recent launches

+ Growth drivers deliver double-digit performance

+ Resource allocation/productivity to fuel strategic

investment (i.e. launches, China)

- Generic impact4

- Healthcare cost containment / pricing

Oncology net sales USD billion, growth in cc

Potential future growth

Established products3Key growth drivers1 Recent launches2

3.3

14.4

2017 2018 2019

13.41.3

0.5

9.7

2.5

0.1

9.6 9.2

3.9

12.3

+9%+10%

1. Including Promacta®/Revolade®, Jakavi® (marketed by Novartis ex-USA), Tafinlar®+ Mekinist® 2. Including Kisqali®, Kymriah®, Lutathera®, Piqray®, Adakveo® 3. All other brands, including those with generic competition in the market

4. Afinitor®, Exjade®, Glivec® and Sandostatin® LAR in EU


Accelerated sales and innovation reinforce the long-term growth of our breast cancer portfolio

~40% of HR+/HER2- breast cancer patients have a PIK3CA mutation,

associated with poor prognosis

NCCN guidelines updated, PIK3CA testing rate ~25% FY 2019

Foundation Medicine PIK3CA CDx tissue approved Q4 2019, plasma

anticipated Q2 2020

"EPIK" development program for 5 new indications1 with potential

opportunity to serve an additional ~100k patients

Only CDK 4/6 inhibitor that consistently demonstrated superior OS in 2

pivotal Phase 3 studies in 2019

Early signs of OS accelerating US growth momentum in Q4

Strong growth across key geographies ex-US in Q4 2019

Potential registration for high and intermediate adjuvant BC as early as 2022

based pre-planned interim analysis (NATALEE, 3-year treatment duration)

Q4Q3Q2

2019 sales in USD m

6

4367

34

91111 123

155

Q2Q1 Q3 Q4

1. TNBC, HER2+ aBC, ovarian cancer, HNSCC; alpelisib PROS

Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation

Adakveo® is off to a solid start in US (approved Nov)

The only approved monthly therapy for reduction in

frequency of VOCs in SCD

Commercial product available within 2 days of approval

Payer engagement ongoing:

– Community centers driving initial uptake

– Permanent J CODE anticipated July 2020

– Medicaid coverage criteria approved in Florida, Kentucky,

Maryland, Washington, Delaware and Alabama

60K

54K

100KSCD Patients in US

60% are 16yr old +

90% have >1 VOC +

VOCs – Vaso-occlusive crises SCD – Sickle cell disease


Kisqali®: Continued strong growth driven

by M3 and M7 OS data

Lutathera®: Leverage potential to grow in

earlier lines of treatment

Promacta®/Revolade®: Growth in ITP and

uptake in 1L SAA in the US and Japan

Piqray®: Maximize US launch momentum

and expand further to EU markets

Adakveo®: Enable access in larger US

accounts and continue education and

patient activation

Kymriah®: Drive further capacity increase

to meet strong demand in pALL and

DLBCL

Capmatinib: Establish awareness of

MET’s role in NSCLC among HCPs /

patient community

Spartalizumab (PDR001) combo: Build

on Taf/Mek leadership position in

metastatic melanoma

177Lu-PSMA: Further evolve commercial

infrastructure for best in class launch

Canakinumab: Focus on medical

education on tumor-promoting

inflammation

Maximizing momentum for our growth drivers and new launches, while preparing for next big bets

Maximize growth drivers

Deliver on new launches

Prepare for next big bets


2019 financial results in line with upgraded guidance

FY 2019 vs. PYin cc

Group full year guidance (as revised in October 2019)in cc

“Sales expected to grow high single digit” 9%

17%“Core operating income expected to grow mid to high teens”


% USD % cc2 % USD % cc

2

Net Sales 12,403 8 9 47,445 6 9

Core Operating income 3,462 11 13 14,112 12 17

Operating income 1,823 34 37 9,086 8 14

Net Income 1,129 -7 -6 7,147 -44 -41

Core EPS (USD) 1.32 14 15 5.28 12 17

EPS (USD) 0.50 -6 -4 3.12 -43 -40

Free Cash Flow 3,488 20 12,937 15

Change vs. PYContinuing operations

1

USD million

Q4

2019

FY

2019

Change vs. PY

Strong sales growth drove double digit increases in core operating income and free cash flow

1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business

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 58 of the Condensed Financial Report

2

2

2


Key drivers vs. PY:

+ Higher operating income

(adjusted for non-cash items)

Offsetting one-time effects:

+ Real estate divestment proceeds

− Prior year OTC JV dividend and GSK milestone income

2019 free cash flow increased to USD 12.9bn driven by higher operating income

20192018

12.911.3

+15%

Continuing operations1 free cash flow2

USD billion

1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business. 2. Free cash flow is a non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the

Condensed Financial Report


Novartis proposes 23rd consecutive dividend increase to the AGM: 2.95 CHF / share1

1. Proposal to shareholders at the 2020 Annual General Meeting, taking place on February 28, 2020 2. Converted at historic exchange rates at the dividend payment dates as per Bloomberg; assumes an exchange rate of USD/CHF of

0.9690 as of December 31, 2019 for 2019 3. In CHF

0.0

0.5

1.0

1.5

2.0

2.5

3.0

200620032001 2004 201820001998 2009 20111997 2016201020051996 2002 20172013 2019201420071999 201520122008

USDCHF

CH

F 2

.85

US

D 2

.84

US

D 3

.04

CH

F 2

.95

2019 dividend yield 3.2%

2019 dividend growth 3.5%3


Core margin

Core margin

change vs. PY

Net sales

change vs. PY

Core operating

income

change vs. PY Core margin

Core margin

change vs. PY

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

Innovative Medicines 31.5 0.7 11 18 33.5 1.8

Sandoz 20.8 1.5 2 10 21.5 1.5

Continuing Operations 27.9 0.8 9 17 29.7 1.9

Q4 2019 FY 2019

Core margin expansion of +1.9%pts

1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business. 2. Core results, constant currencies and free cash flow are non-IFRS measures. An explanation of non-IFRS measures

can be found on page 58 of the Condensed Financial Report

1

2

2

2

2

2

2 2 2

Continuing operations


2020 Novartis full year guidanceBarring unforeseen events; growth vs. PY in cc

Focused medicines company | full year guidanceExcl. Sandoz US oral solids & dermatology businesses1

Sales expected to grow mid to high single digit

IM Division expected to grow mid to high single digit

Sandoz expected to grow low single digit

1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during Q1 2020. 2019 FY sales and core operating of the Sandoz US oral solids and dermatology businesses were approximately

USD 1.1bn and 0.3bn, respectively.

Core operating income expected to grow high single to low double digit

Key assumption: Guidance above includes the forecast assumption that no Gilenya® or Sandostatin® LAR generics enter in 2020 in US


FY 2020 Guidance on other financial KPIsBarring unforeseen events (in cc)

Core net

financial result

Expenses expected to increase by around 0.2bn vs. 2019 reflecting

additional financing costs to acquire The Medicines Company

Core tax rate FY core tax rate expected to be broadly in line with 2019

Focused medicines company | full year guidanceExcl. Sandoz proposed US portfolio sale to Aurobindo1 from both 2019 and 2020

1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during Q1 2020. 2019 FY sales and core operating of the Sandoz US oral solids and dermatology businesses were approximately

USD 1.1bn and 0.3bn, respectively.


Delivered strong performance in 2019

47.4 bnNET SALES (USD)

+9%vs. 2018 (cc2)

33.5%IM CORE MARGIN2(%)

+1.8% ptsvs. 2018 (cc2)

12.9 bnFREE CASH FLOW2 (USD)

22.3%1-YEAR TSR3 (%)

Top tierRANKING 3

+15%vs. 2018 (USD)

Continuing operations1, FY 2019, TSR as of YE 2019

1. Continuing operations as defined on page 45 of the Condensed 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 58 of the Condensed Financial Report. 3. TSR in USD from

Jan 1st 2019, using 1 day average price at start and 3 month average price at end; ranking when compared to the global HC peer group as defined in the Novartis 2019 Annual Report

14.1 bnCORE OPERATING INCOME2 (USD)

+17%vs. 2018 (cc2)

5.28CORE EPS2 (USD)

+17%vs. 2018 (cc2)


Inclisiran

TQJ230

LNP023

Iscalimab

Ligelizumab

AD portfolio2

LNA043

Tropifexor

Ofatumumab

UNR844

QVM / QMF149

MBG453

Asciminib

Canakinumab

Capmatinib

Spartalizumab177Lu-PSMA-617

In-market growth drivers, major launches and a rich pipeline expected to sustain our long-term growth

In-market

growth drivers

Major launches

New indications

Novel assets

15 ongoing / upcoming major launches

80+ major submissions planned to 2022

50+ late stage programs1Cosentyx® HS

Cosentyx® GCA

Cosentyx® LP

Cosentyx® JIA

Cosentyx® LN

Entresto® post-AMI

Beovu® DME

Beovu® RVO

Beovu® DR

Beovu® PDR

Ofatumumab pediatric

AVXS-101 IT

Alpelisib PROS

Piqray® TNBC

Piqray® HER2+ aBC

Piqray® ovarian cancer

Piqray® HNSCC

Kisqali® HR+/HER2- BC (adj)

Kymriah® FL

1. Ph3 / in registration 2. AD portfolio – atopic dermatitis portfolio incl. ZPL389, CEE321

SELECT EXAMPLES



Q&A session

Vas NarasimhanChief Executive Officer

Harry KirschChief Financial Officer

Marie-France TschudinPresident, Novartis Pharmaceuticals

Susanne SchaffertPresident, Novartis Oncology

John TsaiHead of Global Drug Development and CMO

Richard SaynorCEO, Sandoz

Shannon Thyme KlingerGroup General Counsel


Appendix

SalesUSD Million

Growth vs. PYUSD Million

Growth vs. PYcc

518 65%

186 nm

965 21%

155 166%

90 nm

96 nm

67 nm

380 16%

356 15%

293 17%

303 16%

107 31%

178 16%

Sales performance driven by Innovative Medicines

Key growth drivers Q4

nm – not meaningful

Lutathera®

23

186

200

67

26

159

95

90

43

68

50

37

35



Currency impact vs. PY%pts, assuming late-January exchange rates prevail in 2020

Expected currency impact for full year 2020

FX impact on Net sales FX impact on Core operating income

-1

-3-2

-5

-3

Q4Q4 FYFY Q1 FYFY Q1

-1 to -20 to -1

-1 to -2

2019 2020 2019 2020

SimulationActual


Net debt slightly reduced by USD 0.3bn in 2019 mainly driven by strong FCF

1. Continuing operations 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. 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.

-16.2 -15.9

-6.6-3.8

-5.3

Dec 31, 2018 Dec 31, 2019Treasury share

transactions, net

Dividends M&A transactions

12.9

Net debt Alcon2Free Cash Flow1

2.90.2

Others

+0.3


2019 pipeline milestones

H1 2019 H2 2019

Regulatorydecisions and

opinions

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

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

Kymriah® r/r DLBCL (JP) ✓ Lucentis® RoP (EU / JP) ✓Promacta® Severe aplastic anaemia (EU) ✕ Lucentis® Diabetic Retinopathy (EU) ✓

Zolgensma® IV SMA (US) ✓ Mayzent® SPMS (EU / JP) ✓2

Zolgensma® IV SMA (EU / JP)EU Q1 2020

JP H1 2020Xolair® Pollinosis (JP) ✓

Major expected

submissions

Brolucizumab (RTH258) nAMD (US / EU / JP) ✓ Cosentyx® nr-axSpA (US / EU / JP) ✓Crizanlizumab (SEG101) Sickle Cell Disease (US / EU) ✓ Entresto® HF-rEF (JP) ✓Mayzent®1 SPMS (JP) ✓ Entresto® HF-pEF (US / EU) Q1 2020

Capmatinib (INC280) NSCLC (US / JP) ✓

Ofatumumab (OMB157) Relapsing MS (US / EU) ✓3

PDR001 (combination

with Tafinlar®+Mekinist®) Metastatic Melanoma (US / EU) H2 2020

QVM / QMF 149 Asthma (EU / JP) ✓

Majorexpected trial

readouts

AVXS-101 IT SMA ✓ Cosentyx® nr-axSpA ✓Zolgensma® IV SMA presymptomatic ✓ Entresto® HF-pEF (✓)4

Fevipiprant (QAW039) Asthma (✓)5

Ofatumumab (OMB157) Relapsing MS ✓PDR001 (combination

with Tafinlar® + Mekinist®) Metastatic melanoma H2 2020

✓ Achieved* ✕ Missed*

*Represents achieving on-time readout of the data, irrespective of trial outcome 1. Piqray® FDA approval achieved in H1 2019. 2. Positive CHMP opinion Nov 2019, EMA approval Jan 2020 3. EU submission early January 2020 4. Study

narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women and patients with lower ejection fraction 5. LUSTER 1&2 readouts completed and did not meet the clinically relevant threshold for reduction

in rate of moderate-to-severe exacerbation.


H1 2020 H2 2020

Regulatory decisions and

opinions

Beovu® nAMD (EU/JP) Adakveo® Sickle cell disease (EU)

Cosentyx® nr-axSpA (EU/US) Capmatinib (INC280) NSCLC (US/JP)

Cosentyx® AS (CN) Cosentyx® Pediatric psoriasis (EU)

Ofatumumab (OMB157) Relapsing MS (US) Cosentyx® nr-axSpA (JP)

Piqray® HR+/HER2- aBC with PIK3CA mutation (EU)

Entresto® HFpEF (US)

QVM149 Asthma (EU/JP) Inclisiran (KJX839) Hyperlipidemia (US)

Tafinlar® & Mekinist® Adjuvant melanoma (CN) Xolair® Nasal Polyposis (US/EU)

Xiidra® DED (EU)

Zolgensma® IV SMA (JP/EU)

Major expected

submissions

Entresto® HFpEF (US) Alpelisib (BYL719) PROS (US)

Inclisiran (KJX839) Hyperlipidemia (EU) ✓ AVXS-101 IT SMA (US)

Cosentyx® Juvenile PsA / enthesitis-related arthritis (US/EU)

Spartalizumab (PDR001) and Tafinlar® & Mekinist®

Metastatic melanoma (US/EU)

177Lu-PSMA-617 mCRPC (US)

Majorexpected trial

readouts*

Entresto® Post-acute MI1 Asciminib (ABL001) CML 3L

Tropifexor (LJN452) NASH Beovu®

DME

UNR844 Presbyopia Jakavi® chronic GVHD

Kisqali® aBC (MONALEESA-2 OS)

177Lu-PSMA-617 mCRPC

✓ Achieved ✕ Missed

2020 expected pipeline milestones

*Achieved = on-time readout of data, irrespective of trial outcome. 1. Interim analysis readout


Capitalizing on the rich pipeline, faster and broader access in China

Pataday®

Allergic Conj.

Mayzent®

RMS


Adj. Melanoma

Zykadia®

ALK+NSCLC 1st line

Cosentyx®

AS

Eucreas®

T2D

Xolair®

Asthma

Lucentis®

DME/RVO/CNV

Ultibro®

COPD

Vigamox®

Bacterial Conj.

Exelon® PatchAD

Entresto®

Chronic HFRevolade®

ITP

Afinitor®

TSC-SEGA and GI/LUNG NET

Exjade®

IOL

Jakavi®

Myelofibrosis


BRAF+ mM

Sandostatin® LARAcromegaly and GEP NET

Tasigna®

Adult CML

Tasigna®

Pediatric CML

Votrient®

aRCC

Zykadia®

ALK+NSCLC 1st line

Zykadia®

ALK+NSCLC 2nd line

Gilenya®

MS

Cosentyx®

PsO

Gilenya®

MS

Cosentyx®

PsO


BRAF+ mM

Tasigna®

Pediatric CMLNDA

Approval

NRDL

Access

Achieved Planned2019 2020FY 2019 sales:

USD 2.2bn


Building depth across our core therapeutic areas…

Spartalizumab comboMetastatic Melanoma

177Lu-PSMA-617mCRPC

Canakinumab (ACZ885) Lung

Asciminib (ABL001) CML

TQJ230CVRR

LNP023Renal diseases

Tropifexor (LJN452) NASH

LOU064CSU

Adriforant (ZPL389)AD

Ligelizumab (QGE031)CSU / CIU

Iscalimab (CFZ533)Transplant / Sjögren's

LMI070SMA

Ofatumumab (OMB157)MS

UNR844Presbyopia

ECF843Dry Eye

SAF312Chronic Ocular Pain

QVM149Asthma

CSJ117Asthma

QBW251COPD

ONCOLOGYCRM IHD Neuroscience Ophthalmology Respiratory

PHARMACEUTICALS

Select

commercial

assets

1

2

Select

pipeline

assetsMBG453 MDS, AML

LNA043Primary Osteoarthritis

Inclisiran (KJX839)Hyperlipidemia

CRM – Cardiovascular, Renal & Metabolism IHD – Immunology, Hepatology & Dermatology 1. Aimovig® is developed in collaboration with Amgen 2. Luxturna® marketed ex-US



...while strengthening our innovative platforms

1

GENE THERAPY CELL THERAPY RADIO-L IGAND THERAPY

Zolgensma®

SMACPK850Retinis Pigmentosa

CD19 CAR-TDLBCL in 1st relapse

CD19 CAR-Tr/r CLL in combo with ibrutinib

CD19 CAR-T1st line high risk pediatric and young adult ALL

CD19 CAR-TBCMA&CD19

CD19 CAR-TCD123

CD19 CAR-Tr/r DLBCL in combo with pembro

CD19 CAR-Tr/r Follicular Lymphoma

CD19 CAR-TPediatric NHL

CD19 CAR-Tr/r DLBCL in combo with ibrutinib

CD19 CAR-TCD22&CD19

CD19 CAR-TEGFRv3

CD19 CAR-TAdult r/r ALL

177Lu-PSMA-617mCRPC

177Lu-NeoBVarious cancers

177Lu-PSMA-R2Prostate Cancer

Select

commercial

assets

Select

pipeline

assets

AVXS-101 ITSMA

AVXS-201Rett Syndrome

AVXS-301ALS

AVXS-401Friedreich’s Ataxia

AVXS-501Undisclosed

AVXS-601Undisclosed

1. Luxturna® marketed ex-US

Includes preclinical and launched programs


Our pipeline projects at a glance

Phase 1/2 Phase 3 Registration Total

ONCOLOGY 57 20 3 80

PHARMACEUTICALS 57 17 10 84

Cardiovascular, Renal, Metabolism 9 5 1 15

Immunology, Hepatology, Dermatology 22 6 2 30

Neuroscience 9 0 2 11

Ophthalmology 5 3 1 9

Respiratory 7 3 3 13

Global Health 5 0 1 6

BIOSIMILARS 0 1 0 1

Total 114 38 13 165


Novartis submission scheduleNew molecular entity: lead and new indications

2020 2021 2022 2023 ≥2024

asciminibABL001

CML 3L

Lead

MBG453HR-MDS

Lead

ligelizumabQGE031

Chronic urticaria

Lead

ECF843Dry eye

Lead LOU064Chronic spontaneous urticaria

Lead

LNP023PNH

Lead

denosumabGP2411

anti RANKL mAb

BioS

SAF312COSP

Lead

CPK850RP

Lead

LMI070SMA

Lead

MIJ821Depression

Lead

ianalumabVAY736

pSjS

LCM

adriforantZPL389

Atopic dermatitis

Lead

AVXS-201OAV201

Rett syndrome

Lead

LNA043Osteoarthritis

Lead

tropifexorLJN452

NASH

Lead

tropifexor&cenicrivirocLJC242

NASH

Lead

177Lu-NeoB177Lu-NeoB

Multiple Solid Tumors

Lead

VPM0871st line CRC / 1st line RCC

Lead

CEE321Atopic Dermatitis

Lead LXE408Visceral leishmaniasis

Lead

ganaplacideKAF156

Malaria

Lead

TQJ230CVRR-Lp(a)

Lead

cipargaminKAE609

Malaria

Lead

UNR844Presbyopia

Lead QBW251COPD

Lead

CSJ117Severe asthma

Lead

spartalizumabPDR001

m BRAF V600+ melanoma (+Taf/Mek)

Lead

177Lu-PSMA-617177Lu-PSMA-617

mCRPC

Lead

LOU064SjS

LCM

ianalumabVAY736

AIH

Lead

ofatumumabOMB157

Ped MS

LCM

iscalimabCFZ533

SjS

LCM tropifexorLJN452

NASH (combos)

LCM

MBG453Unfit AML

LCM

crizanlizumabSEG101

Sickle cell anaemia w ith crisis

LCM

LNP023iMN

LCM

cipargaminKAE609

Malaria

LCM

LNP023C3G

LCM

LNP023IgAN

LCM

canakinumabACZ885

Adjuvant NSCLC

LCM spartalizumabPDR001

Malignant melanoma (combo)

LCMcanakinumabACZ885

NSCLC 2L

LCM

canakinumabACZ885

NSCLC 1L

LCM

crizanlizumabSEG101

Sickle cell anaemia new formulations

LCM

LE

AD

IN

DIC

AT

ION

SN

EW

IN

DIC

AT

ION

S

capmatinibINC280

Solid tumors

LCM

MBG453Fit AML

LCM

MBG453AML

LCM

iscalimabCFZ533

Renal/Liver Tx

Lead

inclisiranLNP023

Hyperlipidemia

LCM

Compared to past reports, we have categorized submission schedules into NMEs (lead & new indications) and supplementary indications for existing brands


Novartis submission scheduleSupplementary indications for existing brands

2020 2021 2022 2023 ≥2024

Cosentyx USsecukinumab, AIN457

Ped Psoriasis

LCM

Entrestovalsartan+sacubitril, LCZ696

HFpEF

LCM

Xolairomalizumab, IGE025

CSU (for CN)

LCM

Cosentyxsecukinumab, AIN457

PsA H2H

LCM

AVXS-101onasemno-gene abepar-vovec, OAV101

SMA IT

LCM

alpelisib, BYL719PROS

LCM


Psoriasis 300mg AI

LCM

Entrestovalsartan+sacubitril, LCZ696

Post-AMI

LCM

Lampreneclofazimine, LAM320

Tuberculosis

LCM


Auto-injector

LCM


Food allergy

LCM

Beovubrolucizumab, RTH258

DME

LCM

Jakaviruxolitinib, INC424

Acute GVHD

LCM

Promactaeltrombopag, ETB115

Food effect free formulation

LCM


Chronic GVHD

LCM

Kymriahtisagenlecleucel-T, CTL019

r/r DLBCL 1st relapse

LCM


r/r Follicular lymphoma

LCM

Tafinlardabrafenib, DRB436

Neoplasm Pedia

LCM


AS H2H

LCM


Hidradenitis suppurativa

LCM


SpA IVIV

LCM

Kisqaliribociclib, LEE011

HR+/HER2- BC (adj)

LCM

Piqrayalpelisib, BYL719

Ovarian cancer

LCM

Promactaeltrombopag, ETB115

Radiation sickness syndrome

LCM


RVO

LCM

Tafinlardabrafenib, DRB436

Tyroid cancer

LCM


Diabetic retinopathy

LCM


TNBC

LCM


HER2+ adv BC

LCM


Adult r/r ALL

LCM


Lupus Nephritis

LCM


Myelofibrosis (combination)

LCM


Pediatrics Acute GVHD

LCM Cosentyxsecukinumab, AIN457

GCA

LCM


1L high risk ALL, pediatrics & young adults

LCM Jakaviruxolitinib, INC424

Pediatrics Chronic GVHD

LCM

177Lu-PSMA-R2177Lu-PSMA-R2

Prostate cancer

LCM


HNSCC 2/3L

LCM


r/r DLBCL (+ pembro)

LCM Cosentyxsecukinumab, AIN457

Ankylosing spondylitis

LCM


Lichen Planus

LCM Mayzentsiponimod, BAF312

Ped MS

LCM

a) Approved in US

Entresto EUa

valsartan+sacubitril, LCZ696

Pediatric HF

LCM

Compared to past reports, we have categorized submission schedules into NMEs (lead & new indications) and supplementary indications for existing brands


Novartis pipeline in registration

Oncology

Code Name Mechanism Indication(s)

BYL719 Piqray PI3Kα inhibitorPIK3CA mutant HR+, HER2 (-) postmenopausal adv BC5 2nd line (+fulv)

INC280 capmatinib Met Inhibitor NSCLC

SEG101 Adakveo® P-selectin Inhibitor Sickle cell disease

Immunology, Hepatology, Dermatology


AIN457 Cosentyx IL17A Inhibitor Ped Psoriasis nr-axSpA

Ophthalmology


RTH258 Beovu VEGF Inhibitor nAMD

Neuroscience


OAV101 Zolgensma® Gene therapy SMA IV

OMB157 ofatumumab CD20 Antagonist r MS

Respiratory Disease


IGE025 Xolair IgE Inhibitor Nasal polyps

QMF149 Indacaterol acetate +mometasone furoate

Long acting b2-adrenergic agonist + inhaled corticosteroid

Asthma

QVM149 Indacaterol acetate +mometasone fuorate

+glycopyrrnium bromide

Long acting b2-adrenergic agonist + long-acting muscarinic

antagonist + inhaled

corticosteroid

Asthma

Cardiovascular, Renal, Metabolism


KJX839 inclisiran siRNA (PCSK9) Hyperlipidemia

Global Health


LAM320 Lamprene® SMPD1 Inhibitor Tuberculosis

6 lead indicationsLead indication


Novartis pipeline in Phase 3

Oncology


177Lu-PSMA-617 177Lu-PSMA-617 Targeted Radioligand Therapy mCRPC

ABL001 asciminib BCR-ABL Inhibitor CML 3L

ACZ885 canakinumab IL-1b Inhibitor NSCLC 1L NSCLC 2LAdjuvant NSCLC

BYL719 Piqray® PI3Kα inhibitor HER2+ adv BC TNBC HNSCC 2/3L Ovarian cancer

CTL019 Kymriah CD19 CART r/r Follicular lymphoma

1L high risk ALL, pediatrics

and young

adults

r/r DLBCL 1st relapse

Adult r/r ALL

ETB115 Promacta® Thrombopoietin receptor (TPO-R) Agonist

Radiation sickness syndrome Food effect free formulation

INC424 Jakavi JAK1/2 Inhibitor Acute GVHD Chronic GVHD

LEE011 Kisqali® CDK4 Inhibitor HR+/HER2- BC (adj)

PDR001 Spartalizumab PD1 Inhibitor m BRAF V600+ melanoma (+Taf/Mek)

SEG101 crizanlizumab P-selectin Inhibitor Sickle cell anemia new formulation



AIN457 Cosentyx IL17A Inhibitor Lupus Nephritis

Psoriasis 300mg AI

Hidradenitis suppurativa

AS H2H PsA H2H

QGE031 ligelizumab IgE Inhibitor Chronic spontaneous urticaria

Ophthalmology


RTH258 Beovu® VEGF Inhibitor Diabetic retinopathy RVO DME

Respiratory Disease


IGE025 Xolair® IgE Inhibitor CSU (for CN) Auto-injector Food allergy



KJX839 inclisiran siRNA (PCSK9) Hyperlipidemia

LCZ696 Entresto® AT-II / NEP,NEP,AGTR1,AGTR2 Inhibitor Post-AMI HFpEF Pediatric HFa

TQJ230 TQJ230 Anti-Apo(a) ASO targeting Lp(a) CVRR-Lp(a)

Biosimilars


GP2411 denosumab anti RANKL mAb Denosumab BioS


a) Approved in US


Novartis pipeline in Phase 2

Oncology


ACZ885 canakinumab IL-1b Inhibitor Sickle cell anaemia

BYL719 alpelisib PI3Kα inhibitor PROS

CTL019 Kymriah CD19 CART r/r DLBCL (+ pembro)

EGF816 nazartinib+capmatinib Opdivo EGFR Inhibitor NSCLC

INC280 capmatinib Met Inhibitor Solid tumors

Met Inhibitor + spartalizumab HCC NSCLC

INC424 Jakavi® JAK1/2 Inhibitor Myelofibrosis (combination)

LAG525 LAG525 LAG3 Inhibitor Solid Tumors

MBG453 MBG453 TIM3 Antagonist HR-MDS Unfit AML AML Fit AML

NIR178 NIR178, spartalizumab Ad2AR Inhibitor, PD1 Inhibitor Cancers

PDR001 spartalizumab PD1 Inhibitor Nasopharyngeal cancer Metastatic melanoma (combo)

SEG101 crizanlizumab P-selectin Inhibitor Ped sickle cell anaemia with crisis



AIN457 Cosentyx® IL17A Inhibitor SpA IVIV GCA Lichen Planus

CFZ533 iscalimab CD40 Inhibitor Renal/Liver Tx SjS HS T1DM

LJC242 tropifexor&cenicriviroc CCR2 Inhibitor, FXR agonist NASH

LJN452 tropifexor FXR agonist NASH Nash (combos)

LNA043 LNA043 ANGPTL3 Agonist Osteoarthritis

LOU064 LOU064 BTK Inhibitor Chronic spontaneous urticaria SjS

LYS006 LYS006 Anti-inflammatory Acne

VAY736 ianalumab BAFF-R Inhibitor AIH pSjS SLE

ZPL389 adriforant HRH4 Antagonist Atopic dermatitis

Ophthalmology


CPK850 CPK850 RLBP1 AAV RP

ECF843 ECF843 rh-Lubricin Dry eye

LKA651 LKA651 EPO Inhibitor DME

SAF312 SAF312 TRPV1 Antagonist COSP

UNR844 UNR844 disulfide bonds Modulator Presbyopia

Neuroscience


AFQ056 AFQ056 mGluR5 Antagonist Addiction

BAF312 Mayzent® S1P1 Modulator Ped MS Stroke

BLZ945 BLZ945 CSF-1 Inhibitor ALS

LMI070 branaplam Survival motor neuron protein SMA

MIJ821 MIJ821 NR2B Inhibitor Depression

OMB157 ofatumumab CD20 Antagonist Ped MS

Respiratory Disease


ACZ885 canakinumab IL-1b Inhibitor Sarcoidosis

CJM112 CJM112 IL-17A Inhibitor Asthma

CSJ117 CSJ117 TSLP Inhibitor Severe asthma

LOU064 LOU064 BTK Inhibitor Asthma

QBW251 QBW251 CFTR Potentiator COPD

VAY736 ianalumab BAFF-R Inhibitor IPF



CFZ533 iscalimab CD40 Inhibitor Lupus Nephritis

LMB763 nidufexor FXR Agonist Diabetic Nephropathy

LNP023 LNP023 CFB Inhibitor PNH IgAN C3G iMN

Global Health


KAE609 cipargamin PfATP4 inhibitor Malaria Malaria severe

KAF156 ganaplacide - Malaria

LXE408 LXE408 Protozoan Inhibitor Visceral leishmaniasis



Novartis pipeline in Phase 1 (1 of 2)Lead indication

Oncology


177Lu-NeoB 177Lu-NeoB Radioligand therapy target GRPR Multiple solid tumors

177Lu-PSMA-R2 177Lu-PSMA-R2 Radioligand therapy target PSMA Prostate cancer

ADPT01 NIR178, LAG525, spartalizumab, canakinumab, capmatinib LAG3 Inhibitor,PD1 Inhibitor TNBC

BLZ945 BLZ945 + spartalizumab CSF-1 Inhibitor + PD1 Inhibitor Solid tumors

CSJ137 CSJ137 Growth Factor Inhibitor Anaemia

CTL019 Kymriah® CD19 CART Lymphoma

DKY709 DKY709 + spartalizumab - Cancers

EGF816 nazartinib + LXH254, ribociclib, capmatinib, Opdivo, Mekinist EGFR Inhibitor NSCLC

HDM201 HDM201 + MBG453, venetoclax MDM2 Inhibitor Haematological malignancy

JEZ567 JEZ567 CD123 CART AML

JJO686 JJO686 CD22 CART ALL

LHC165 LHC165 + spartalizumab TLR7 Agonist Solid tumors

LSZ102 LSZ102, ribociclib, alpelisib SERD BC

LXF821 LXF821 EGFR CART, PD1 Inhibitor Glioblastoma multiforme

LXH254 LXH254 (combos) cRAF Inhibitor Solid tumors Solid tumors

MAK683 MAK683 EED Inhibitor Cancers

MAS825 MAS825 - Inflammatory diseases

MBG453 MBG453 (combos) TIM3 Antagonist Cancers

MCM998 MCM998, LXG250 BCMA CART, CD19 CART Multiple myeloma

MIK665 MIK665 MCL1 Inhibitor AML Haematological malignancy AML (combo)

NIS793 NIS793, spartalizumab TGFB1 Inhibitor, PD1 Inhibitor Solid tumors

NIZ985 NIZ985, spartalizumab IL-15 Agonist Solid tumors

NJH395 NJH395 - Solid tumors

NZV930 NZV930, spartalizumab, NIR178 CD73 Antagonist Solid tumors

PDR001 spartalizumab, CJM112, LCL161 PD1 Inhibitor, TIM3 Antagonist AML Solid tumors (combo) Solid tumors (combo) Solid tumors (combo)

SQZ622 SQZ622 CD123xCD3 Modulator AML

TNO155 TNO155 SHP2 Inhibitor Solid tumors (single agent) Solid tumors (combo)

VAY736 ianalumab + ibrutinib BAFF-R Inhibitor,BTK Inhibitor Haematological malignancy

VOB560 VOB560 - Cancers

VPM087 VPM087 IL1B Antagonist 1st line CRC / 1st line RCC

WNT974 WNT974 + spartalizumab Porcupine Inhibitor Solid tumors

WVT078 WVT078 - Multiple myeloma

YTB323 YTB323 + ibrutinib CD19 CART Haematological malignancy

33 lead indications


Novartis pipeline in Phase 1 (2 of 2)Lead indication

34 lead indications



DFV890 DFV890 - Multiple Indications

LRX712 LRX712 - Osteoarthritis

MHS552 MHS552 - Autoimmune Indications

MHV370 MHV370 - SLE

Neuroscience


OAV101 AVXS-101 Survival motor neuron protein gene therapy

SMA IT1

OAV201 AVXS-201 MECP2 gene therapy Rett syndrome

Respiratory Disease


CMK389 CMK389 IL-18 Inhibitor Sarcoidosis



HSY244 HSY244 - Atrial fibrillation

LTW980 LTW980 - Hypertriglyceridemia

MBL949 MBL949 - Diabetes

Global Health


KAF156 ganaplacide - Malaria prophylaxis

1. FDA placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study


Abbreviations

IT Intrathecal formulation

IV Intravenous formulation

JIA Juvenile idiopathic arthritis

LP Lichen planus

LN Lupus nephritis

mCRPC Metastatic castration-resistant prostate cancer

MDR Multi-drug resistant

MDS Myelodysplastic syndrome

MS Multiple sclerosis

nAMD Neovascular (wet) age-related macular degeneration

NASH Non-alcoholic steatohepatitis

nr-axSpA Non-radiographic axial spondyloarthritis

NRDL National reimbursement drug list

NSCLC Non-small cell lung cancer

PDR Proliferative diabetic retinopathy

PEF Preserved ejection fraction

PNH Paroxysmal nocturnal haemoglobinuria

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

RCC Renal cell carcinoma

PROS PIK3CA related overgrowth spectrum

RA Rheumatoid arthritis

rMS Relapsing multiple sclerosis

ROP Retinopathy of prematurity

RP Retinitis pigmentosa

RVO Retinal vein occlusion

SAA Severe aplastic anemia

SjS Sjögren’s syndrome

SLE Systemic lupus erythematosus

SMA Spinal muscular atrophy type 1 (IV formulation)

SpA Spondyloarthritis

SPMS Secondary progressive multiple sclerosis

TNBC Triple negative breast cancer

T1DM Type 1 Diabetes melitus

AIH Autoimmune hepatitis

ALL Acute lymphoblastic leukemia

ALS Amyotrophic lateral sclerosis

AMI Acute myocardial infarction

AML Acute myeloid leukemia

AS H2H Ankylosing spondylitis head-to-head study versus adalimumab

BC Breast cancer

C3G C3 glomerulopathy

CDx Companion diagnostics

CCF Congestive cardiac failure

CLL Chronic lymphocytic leukemia

CML Chronic myeloid leukemia

CRC Colorectal cancer

COPD Chronic obstructive pulmonary disease

COSP Chronic ocular surface pain

CSU Chronic spontaneous urticaria

CVRR-Lp(a) Secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein (a)

CVRR-LDLC Secondary prevention of cardiovascular events in patients with elevated levels of LDLC

DME Diabetic macular edema

DLBCL Diffuse large B-cell lymphoma refractory

FL Follicular lymphoma

GCA Giant cell arteritis

GVHD Graft-versus-host disease

HCC Hepatocellular carcinoma

HFpEF Chronic heart failure with preserved ejection fraction

HF-rEF Chronic heart failure with reduced ejection fraction

HNSCC Head and neck squamous cell carcinoma

HS Hidradenitis suppurativa

IgAN IgA nephropathy

iMN Membranous nephropathy

IOL Iron overload

ITP Immune thrombocytopenia

IPF Idiopathic pulmonary fibrosis


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. Q3-2019 presentationNew trials addedStudy Program Indication Phase Patients

NCT04156620 INVIGORATE-1 (CAIN457P12301) Cosentyx® Axial spondyloarthritis Phase 3 500

NCT04065841 ELIVATE (CLJN452D12201C) LJN452 Non-alcoholic steatohepatitis (NASH) Phase 2 210

NCT03373461 (CLNP023X2203) LNP023 IgA nephropathy Phase 2 146

NCT03439839 (CLNP023X2201) LNP023 Paroxysmal nocturnal hemoglobinuria Phase 2 15

NCT03832114 (CLNP023X2202) LNP023 C3 glomerulopathv Phase 2 27

NCT03896152 (CLNP023X2204) LNP023 Paroxysmal nocturnal hemoglobinuria Phase 2 10

NCT03955445 (CLNP023B12001B) LNP023 C3 glomerulopathy Phase 2 27

NCT04154787 (CLNP023D12201) LNP023 Idiopathic membraneous nephropathy Phase 2 72

NCT04109313 (CLOU064A2201E1) LOU064 Chronic spontaneous urticaria (CSU) Phase 2 250

NCT03988608 (CETB115E2202) Promacta®/

Revolade®

Previously untreated or relapsed/refractory severe aplastic anemia

or recurrent aplastic anemia

Phase 2 20

NCT04047472 HOBBY (CRTH258A2307) RTH258 Macular degeneration Phase 3 494

Trials removed (operational decision-points achieved)Study Program Indication Phase Patients

NCT02059291 CLUSTER (CACZ885N2301) Ilaris® Hereditary periodic fevers Phase 3 203

NCT03578367 ASC4MORE (CABL001E2201) ABL001 Chronic myeloid leukaemia (CML) Phase 2 80


Cardiovascular, Renal and Metabolic

Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)

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

H2-2021; (Analysis of 110 pts from Part 2 formed the basis

for pediatric submission in Apr-2019 and approval by the US

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

systemic left ventricular systolic dysfunction in children aged

1 year and older)

2022

Publication TBD TBD


Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)Study NCT02554890 PIONEER-HF (CLCZ696BUS01) NCT02661217 TRANSITION (CLCZ696B2401)

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

Phase Phase 3B/4 Phase 4

Patients 881 1,002

Primary Outcome

Measures

Percentage change from baseline in N-terminal pro-brain

natriuretic peptide (NT-proBNP)

Assessing the percentage of patients who achieve the target

dose of 200 mg bid LCZ696 at 10 weeks after

randomization

Arms/Intervention

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

97/103 mg bid or matching placebo

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

placebo

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

200 mg bid)

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

200 mg bid)

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

and stable for more than 24 hours

Heart failure patients with reduced ejection-fraction

hospitalized for an acute decompensation event

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

Publication

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

biomarkers, de novo HF, hospitalizations, & prior

exposure

• Apr-2019 Circulation: Research letter on composite

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

• Q3-2019 ESC: Secondary abstracts submitted

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

• 28-May-2019 TRANSITION primary publication -

published EJHF

• Secondary data presentations: de novo HF and

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

Congress in May 2019 and submitted to EJHF in Jun-

2019 (expected publication Q3-2019)

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

ESC-2019 in Paris


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); H1-2021 (open-label extension)

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

J



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


Publication

• Sep-2019: Primary manuscript published (Angiotensin–

Neprilysin Inhibition in Heart Failure with Preserved

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

10.1056/NEJMoa1908655)

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

results

TBD



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 Q4-2019 (actual) H1-2021

Publication TBD TBD



Study NCT03373461 (CLNP023X2203) NCT04154787 (CLNP023D12201)

Indication IgA nephropathy (IgAN) Idiopathic membranous nephropathy (iMN)


Patients 146 72

Primary Outcome

Measures

Change from baseline of log transformed UPCR derived

from the 24h urine collections at Baseline and Day 90

Change from baseline of UPCR derived from 24hr urine

collections at Baseline and Week 24

Arms/Intervention

• Placebo

• LNP023 Dose 1 – 10mg bid



• LNP023 Dose 4 – 100mg bid (Part 2 only)

• LNP023 Dose – 200mg bid

• LNP023 Dose – 50mg bid

• Rituximab

Target Patients Patients with biopsy-verified IgA nephropathy

Patients with biopsy proven iMN who are at high risk of

disease progression defined on the basis of antibody anti-

PLA2R titre and proteinuria

Expected Completion 2021 2022

Publication TBD TBD

LNP023 – Factor B inhibition of the complement alternative pathway


Study NCT03832114 (CLNP023X2202) NCT03955445 (CLNP023B12001B)

Indication C3 glomerulopathy (C3G) C3 glomerulopathy (C3G)

Phase Phase 2 Phase 2 (open-label extension)

Patients 27 27 (from ongoing Phase 2, potential patient from Ph3)

Primary Outcome

Measures

Cohort A: Ratio to Baseline of UPCR to Week 12 derived

from 24hr urine collection

Cohort B: Change from Baseline in C3 Deposit Score

(based on immunofluorescence microscopy) at Week 12

Characterize the effect of LNP023 treatment on a composite

renal response endpoint at 9 months (1. a stable or

improved eGFR and, 2. a reduction in proteinuria and 3. an

increase in C3 compared to the CLNP023X2202 baseline

visit)

Arms/Intervention

Increasing doses of LNP023 up to 200mg bid:

• Cohort A: Native kidney patients

• Cohort B: Kidney transplanted patients

• Open-label LNP023 200mg bid

Target Patients Patients with C3 glomerulopathy Patients with C3 glomerulopathy


Publication TBD TBD



Study NCT03439839 (CLNP023X2201) NCT03896152 (CLNP023X2204)

Indication Paroxysmal nocturnal hemoglobinuria (PNH) Paroxysmal nocturnal hemoglobinuria (PNH)


Patients 15 10

Primary Outcome

Measures

Reduction of chronic hemolysis, based on LDH level at

Week 13

Reduction of PNH associated hemolysis, based on

percentage of patients with 60% reduction in LDH or LDH

below upper limit of normal up to 12 weeks of treatment.

Arms/Intervention

• Cohort 1: 10 patients receiving LNP023 200mg bid, in

addition to SoC, for 13 weeks with 3yr treatment extension

period

• Cohort 2: 5 patients receiving LNP023 50mg bid, in

addition to SoC, for minimum 2 weeks with 3yr treatment

extension period. Dose may be increased D15 onwards to

200mg bid if LDH not within limit of normal or reduced by at

least 60% compared to Baseline.

• Arm 1: 4wks treatment LNP023 25mg bid followed by

8wk treatment LNP023 100mg bid and 2yr extension

LNP023 100mg bid

• Arm 2: 4wks treatment LNP023 50mg bid followed by

8wk treatment LNP023 200mg bid and 2yr extension

LNP023 200mg bid

Target Patients

Patients with PNH, showing signs of active hemolysis

despite treatment with SoC (defined as an antibody with anti

C5 activity).

Patients with PNH, showing signs of active hemolysis, not

treated with any other complement inhibitor less than 3

months prior to study start Day 1

Expected CompletionPrimary endpoint: 2020

Extension period: 2023

Primary endpoint: 2020

Extension period: 2022

Publication In preparation TBD



Immunology, Hepatology & Dermatology

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

Study NCT03663335 CIRRUS I (CCFZ533A2201) NCT03905525 TWINSS (CCFZ533B2201)

Indication Kidney transplantation Sjögren's syndrome

Phase Phase 2B Phase 2B

Patients 676 260

Primary Outcome

Measures

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

months post-transplantation and post conversion (for

maintenance cohort)

Change in EULAR Sjögren’s syndrome Disease Activity

Index (ESSDAI) score and EULAR Sjögren’s syndrome

Patient Reported Index (ESSPRI) score

Arms/Intervention

• Two cohorts: de novo TX and maintenance

• Test Arms: CFZ533 + MMF + corticosteroids

• Standard of Care: TAC + MMF + corticosteroids

• Three dose arms of CFZ533

• Placebo

Target Patients Kidney transplant recipients Patients with Sjögren's syndrome


Publication Manuscript of PoC trial to be submitted in Q1-2020 Manuscript of PoC trial to be published in Q1-2020


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

Study NCT03781414 CONTRAIL I (CCFZ533A2202)

Indication Liver transplantation

Phase Phase 2

Patients 128

Primary Outcome

Measures

Proportion of patients with composite event (BPAR, Graft

Loss or Death) over 12 months

Arms/Intervention

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

• CFZ533 dose A + MMF + Corticosteroids

• CFZ533 dose B + MMF + Corticosteroids

Target Patients Liver transplant recipients

Expected Completion 2022

Publication TBD


Cosentyx® - Anti IL-17

Study NCT03504852 (CAIN457A2324) NCT03589885 MATURE (CAIN457A2325)

Indication Psoriasis Psoriasis

Phase Phase 3B Phase 3

Patients 331 122

Primary Outcome

Measures

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

16 weeks of treatment

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

12 weeks of treatment

Arms/Intervention

• Secukinumab 300 mg every 2 weeks after weekly doses

till Week 4

• Secukinumab 300 mg every 4 weeks after weekly doses

till Week 4

• Secukinumab 2 mL (300 mg) auto-injector

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

• Placebo 2 mL auto-injector

• Placebo 2 x 1 mL prefilled syringe

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

Expected Completion Q3-2020 Q4-2020

Publication TBD TBD



Study NCT02471144 (CAIN457A2310) NCT03668613 (CAIN457A2311)

Indication Psoriasis Psoriasis


Patients 162 84

Primary Outcome

Measures

Psoriasis Area and Severity Index (PASI) 75 response and

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

week 12



week 12

Arms/Intervention

• Secukinumab low dose

• Secukinumab high dose

• Placebo

• Etanercept (comparator)

• Secukinumab low dose

• Secukinumab high dose

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

chronic plaque psoriasis

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

severe plaque psoriasis


Publication TBD TBD



Study NCT03066609 (CAIN457A2318)

Indication Psoriasis

Phase Phase 3

Patients 543

Primary Outcome

Measures



week 12

Arms/Intervention

• Secukinumab 300 mg


• Placebo

Target PatientsPatients with moderate to severe chronic plaque-type

psoriasis with or without psoriatic arthritis comorbidity

Expected Completion Q1-2019 (actual)

Publication

• Week 16 results: Poster presented at: 2019 American

Academy of Dermatology (AAD) Annual Meeting,

• March 1–5, 2019, Washington, D.C.

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

October, 2019



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

Expected Completion H1-2021 2025

Publication TBD TBD



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

Indication Psoriatic arthritis Ankylosing spondylitis


Patients 460 219

Primary Outcome

Measures

Proportion of subjects that have a positive clinical response

to treatment (individual improvement) in disease activity

according to ACR20 (or ACR50 or ACR 70)

Assessment of SpondyloArthritis International Society /

ASAS 20 response

Arms/Intervention• Secukinumab 75 mg




• Placebo

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


Publication

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

Rheumatol. 2016; 68 (suppl 10)

• 3 years results: Manuscript published in September

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

doi:10.1136/rmdopen-2018-000723)

• 5 year results: accepted for publication in ACR open

Rheumatology in September 2019

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

Engl J Med 2015;373:2534–48

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

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

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

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

FRI0379. Annals of the Rheumatic Diseases

2019;78:873.

• 5 year results; manuscript target submission Oct 2019



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



Patients 399 222

Primary Outcome

Measures

Proportion of subjects achieving American College of

Rheumatology 20 (ACR20) response criteria

Assessment of Spondyloarthritis International Society

criteria / ASAS 20 response

Arms/Intervention

• Secukinumab (AIN457) 150 mg s.c.



• Placebo s.c.

• Secukinumab 10 mg/kg / 300 mg

• Secukinumab 10 mg/kg / 150 mg

• Placebo



Publication

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

2015;386:1137–46

• 2 years results: McInnes et al, Rheumatology

2017;56:1993-2003

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

Oct-2019

• 16 weeks results: PANLAR congress in Apr-2016

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

Therapy 2017

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

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

PANLAR April 2019

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

awaiting journal decision



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


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

Indication Psoriatic arthritis Psoriatic arthritis


Patients 342 990

Primary Outcome

Measures

Assessment of American College of Rheumatology 20

(ACR20)

American College of Rheumatology 20 (ACR20) response at

Week 16

Arms/Intervention

• Secukinumab 150 mg with loading

• Secukinumab 150 mg without loading

• Placebo

• Secukinumab 150 mg load

• Secukinumab 150 mg no load


• Placebo

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


Publication

• 52 week results: abstract presented at PANLAR

congress (Apr-2018)

• 2 year (EOS) results published: Rheumatology

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

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

Rheumatic Diseases 2018;77:890-897.

• 52 week results presented at EULAR and ACR 2018

• 52 week manuscript accepted (Rheumatology, October

2019, in press)

• 2 year (EOS) results presented at EULAR 2019



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) Q1-2020

Publication

• 3-year results: Manuscript published in Clinical and

Experimental Rheumatology in May-2017

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

• 4 year results manuscript published; Rheumatology,

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

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

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

10.1136/rmdopen-2019-001005

• Manuscript target submission Jan-2020



Study NCT02696031 PREVENT (CAIN457H2315) NCT03259074 SURPASS (CAIN457K2340)

Indication Non-radiographic axial spondyloarthritis Ankylosing spondylitis


Patients 555 837

Primary Outcome

Measures

The proportion of participants who achieved an ASAS 40

response (Assessment of SpondyloArthritis International

Society criteria);

No radiographic structural progression as measured by

modified Stoke Ankylosing Spondylitis Spine Score

(mSASSS)

Arms/Intervention


• Secukinumab 150 mg no load

• Placebo

• Secukinumab 150/300 mg

• Adalimumab biosimilar 40 mg

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

Expected Completion Week 52: Q3-2019 (actual); Final: H1-2021 2022

Publication

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

ACR 2019

• Manuscript target submission Jan-2020

TBD



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

Expected Completion H2-2021 H2-2021

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



Study NCT04156620 INVIGORATE-1 (CAIN457P12301)

Indication Axial spondyloarthritis

Phase Phase 3

Patients 500

Primary Outcome

Measures

The proportion of subjects achieving an ASAS40

(Assessment of SpondyloArthritis International Society

criteria) response

Arms/Intervention• Secukinumab intravenous (i.v.) regimen

• Placebo intravenous (i.v.) regimen

Target Patients Patients with active axial spondyloarthritis


Publication TBD


Ilaris® - Anti IL-1β

Study NCT02296424 (CACZ885G2306)

Indication SJIA - Systemic Juvenile Idiopathic Arthritis

Phase Phase 3B/4

Patients 182

Primary Outcome

Measures

Proportion of patients in clinical remission on

canakinumab who are able to remain in remission

following canakinumab dose tapering (reduced

canakinumab dose or prolonged canakinumabdosing

interval)

Arms/Intervention• Canakinumab dose reduction

• Canakinumab dose interval prolongation

Target PatientsPatients with Systemic Juvenile Idiopathic Arthritis (SJIA)

(Pediatric)

Expected Completion 2018 (actual)

Publication

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

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


LJN452 - FXR Agonist

Study NCT02855164 (CLJN452A2202) NCT04065841 ELIVATE (CLJN452D12201C)

Indication Non-alcoholic steatohepatitis (NASH) Non-alcoholic steatohepatitis (NASH)


Patients 345 210

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

Proportion of patients with resolution of NASH and no

worsening of fibrosis OR improvement in fibrosis by at least

one stage without worsening of NASH at Week 48

compared with baseline

Arms/Intervention • Multiple LJN452 doses and placebo

• Arm A: combination therapytropifexor + licogliflozin

• Arm B: tropifexor monotherapytropifexor (+ licogliflozin

placebo)

• Arm C: licogliflozin monotherapylicogliflozin (+ tropifexor

placebo)

Target Patients Patients with non-alcoholic steatohepatitis (NASH)Adult patients with non-alcoholic steatohepatitis (NASH)

and liver fibrosis

Expected Completion Q2-2020 2022

Publication

• Primary (interim) data abstract submitted to AASLD in

Q3-2019

• Manuscript to be submitted in H2-2020

TBD


LOU064 – Bruton's tyrosine kinase (BTK) inhibitor

Study NCT03926611 (CLOU064A2201) NCT04109313 (CLOU064A2201E1)

Indication Chronic spontaneous urticaria (CSU) Chronic spontaneous urticaria (CSU)


Patients 308 250

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

4• Long-term safety and tolerability

Arms/Intervention

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

matching placebo in the evening from Day 1 to 85

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


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


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

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

• 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

• Selected dose of LOU064 taken orally twice a day

(morning and evening) from day 1 to week 52

Target Patients Adults with CSU inadequately controlled by H1-antihistaminesPatients with CSU who have participated in preceding

studies with LOU064


Publication TBD TBD


LJC242 - FXR agonist + CCR2/CCR5 inhibitor

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

Expected Completion Q4-2020

Publication Manuscript to be submitted in H1-2021


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

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

Phase Phase 2B Phase 2B

Patients 382 226

Primary Outcome

Measures

Establish dose-response relationship of QGE031 with

respect to achievement of complete hives response at week

12

Long-term safety; number of participants with treatment-

emergent adverse events

Arms/Intervention

• Ligelizumab 24mg q4wks for 20 weeks



• Ligelizumab 120mg single dose

• Omalizumab 300mg q4wks for 20 weeks

• Placebo q 4wks for 20 weeks

Ligelizumab 240 mg q4wks open label for 52 weeks

Target Patients

Adult patients with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines at approved

or increased doses, alone or in combination with H2-

antihistamines or leukotriene receptor antagonists.

Adult patients with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines at approved

or increased doses, alone or in combination with H2-

antihistamines or leukotriene receptor antagonists.

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

Publication

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

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

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

WCD, EADV, PAAM, ACAAI, UCARE.

• Primary results: AAD 2019;

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

WCD, EADV, PAAM, ACAAI, UCARE; manuscript

planned in H1/2020


QGE031 - Anti-IgE

Study NCT03437278 (CQGE031C2202)

Indication Chronic spontaneous urticarial / Chronic idiopathic urticaria

Phase Phase 2

Patients 48

Primary Outcome

MeasuresChange in the 7 day Urticaria Activity Score (UAS7)

Arms/Intervention

• Ligelizumab high dose q4wks for 24 weeks

• Ligelizumab low dose q4wks for 24 weeks

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

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

spontaneous urticaria

Expected Completion H2-2021

Publication

• Study design was presented at PAAM (Peds Allergy &

Asthma Meeting) and at UCARE meeting 2019

• Primary results to be presented in 2022 (e.g. EAACI,

PAAM, EADV)

• Manuscript to be submitted in 2022


QGE031 - Anti-IgE

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

Indication Chronic spontaneous urticaria Chronic spontaneous urticaria



Primary Outcome

Measures

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Absolute change from baseline in UAS7 (Urticaria Activity

Score) at week 12

Arms/Intervention

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

• Ligelizumab dose A q4w for 52 weeks

• Ligelizumab dose B q4w for 52 weeks

• Omalizumab 300 mg q4w for 52 weeks

• Placebo q4w from randomization to wk20, then

ligelizumab dose B from wk24 to wk52

Target PatientsAdolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines

Adolescents and adults with chronic spontaneous urticaria

inadequately controlled with H1-antihistamines


Publication

• Study design presented at UCARE 2018

• Primary results to be presented in 2022 (e.g. EAACI, PAAM, EADV)



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

Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)

Indication Primary Sjögren's syndrome Autoimmune hepatitis

Phase Phase 2B Phase 2/3

Patients 180 80

Primary Outcome

Measures

Safety and efficacy of VAY736 in primary Sjögren's

syndrome (pSS)Alanine aminotransferase (ALT) normalization

Arms/Intervention• VAY736

• Placebo

• VAY736

• Placebo control with conversion to active VAY736

Target PatientsPatients with moderate to severe primary Sjögren's

syndrome (pSS)

Autoimmune hepatitis patients with incomplete response or

intolerant to standard treatment of care


Publication

• Late Breaking Abstract to be submitted to American

College of Rheumatology 30-Sep-2019


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

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

Indication Type 1 spinal muscular atrophy Type 1 spinal muscular atrophy


Patients 30 20

Primary Outcome

MeasuresProportion of participants sitting without support

• Achievement of independent sitting for at least 30

seconds

• Event-free survival

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

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

Expected Completion H2-2020 Q4-2019 (actual)

Publication TBD TBD



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



Study NCT03381729 STRONG (CL-102)

Indication Type 2 spinal muscular atrophy

Phase Phase 1

Patients 27

Primary Outcome

Measures

• Safety and tolerability, incidence of adverse events

• Proportion of patients achieving Standing Milestone

• Change in Hammersmith Functional Motor Scale

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

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

Expected Completion Q4-2019 [Cohort B]

Publication TBD


Aimovig® – CGRP receptor antagonist

Study NCT03096834 LIBERTY (CAMG334A2301) NCT03333109 EMPOWER (CAMG334A2302)

Indication Migraine Migraine


Patients 246 900

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); H1-2021 OLE phase (final DBL) Q2-2020

Publication

• Planned for Q1-2020 (Neurology): PROs and

prespecified subgroup analysis (DBT phase)

• Planned for Q2-2020: 1Y OLE

• Planned for Q4 2020: 2Y OLE – TBC. Potentially

abstract only

Planned for H2-2020


Aimovig® – CGRP receptor antagonist

Study NCT03867201 DRAGON (CAMG334A2304)

Indication Migraine

Phase Phase 3

Patients 550

Primary Outcome

Measures

Change from baseline in monthly migraine days during the

last 4 weeks of the 12-week treatment period

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

• Subcutaneous injection of placebo

Target Patients Adult chronic migraine patients

Expected Completion 2022 DBT phase; 2024 OLE phase

Publication Planned in Q4-2023 (DBT)


Gilenya® - S1P-R modulator

Study NCT01633112 ASSESS (CFTY720D2312) NCT01201356 LONGTERMS (CFTY720D2399)

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

Phase Phase 3B Phase 3


Primary Outcome

Measures

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

to glatiramer acetate (20 mg) in reducing the annualized

relapse rate up to 12 months

Long-term safety and tolerability

Arms/Intervention

• Fingolimod 0.5 mg orally

• Fingolimod 0.25mg orally

• Copaxone® 20 mg s.c.

Single-arm study of fingolimod 0.5 mg/day

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

Expected Completion 2018 (actual) 2018 (actual)

Publication• Primary data presentation at AAN in 2019

• Primary manuscript – submission planned in Q4-2019

• Cohen J et al. Extended treatment with fingolimod for

relapsing multiple sclerosis: the 14-year LONGTERMS

study results (Therapeutic Advances in Neurological

Disorders 2019.12:eCollection 2019, doi:

10.1177/1756286419878324)


LMI070 - SMN2 RNA splice modulator

Study NCT02268552 (CLMI070X2201)

Indication Type 1 spinal muscular atrophy

Phase Phase 1/2

Patients 39

Primary Outcome

Measures

Number of participants with adverse events (AEs), serious

adverse events (SAEs) and deaths

Arms/Intervention

Branaplam oral, once weekly:

• Part 1: 5 ascending doses

• Part 2: 2 different dose levels

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

1 or Part 2

Target PatientsPatients with type 1 spinal muscular atrophy


Publication TBD


Mayzent ® - S1P-R modulator

Study NCT01665144 -EXPAND (CBAF312A2304)

Indication Secondary progressive multiple sclerosis

Phase Phase 3

Patients 1,652

Primary Outcome MeasuresThe delay in time to confirmed disability progression as

measured by EDSS (Expanded Disability Status Scale)

Arms/Intervention

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

dose: 2mg (day 6))

• Placebo

Target Patients Patients with secondary progressive multiple sclerosis

Expected Completion Core in 2016/Extension in 2023

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

March 2018


OMB157 - Anti-CD20

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

Indication Multiple sclerosis Multiple sclerosis


Patients 900 900

Primary Outcome

Measures

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

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

Annualized Relapse Rate (ARR) - number of confirmed

relapses in a year calculated based on cumulative number

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

Arms/Intervention• Ofatumumab subcutaneous

• Teriflunomide oral

• Ofatumumab subcutaneous

• Teriflunomide oral

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


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


OMB157 - Anti-CD20

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

Study NCT03106779 ASCEMBL (CABL001A2301)

Indication Chronic myeloid leukaemia (CML)

Phase Phase 3

Patients 233

Primary Outcome

MeasuresMajor Molecular Response (MMR) rate at 24 weeks

Arms/Intervention• ABL001 40 mg bid

• Bosutinib 500 mg

Target Patients

Patients with chronic myelogenous leukemia in chronic

phase, previously treated with 2 or more tyrosine kinase

inhibitors


Publication Manuscript submission in H2-2020 (journal TBD)


ACZ885 – IL-1β inhibitor

Study NCT03447769 CANOPY-A (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

Expected Completion 2022 H1-2021

Publication TBDJohnson B et al. Abstract accepted for presentation at

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


ACZ885 – IL1β inhibitor

Study NCT03626545 CANOPY-2 (CACZ885V2301)

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

Phase Phase 3

Patients 240

Primary Outcome

Measures

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

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

Overall Survival

Arms/Intervention

• canakinumab in combination with docetaxel

• canakinumab matching-placebo in combination with

docetaxel

Target Patients

Patients with:

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

RAF mutation

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

inhibitor


Publication TBD


BYL719 - Alpha-specific PI3K inhibitor

Study NCT02437318 SOLAR-1 (CBYL719C2301)

Indication HR+/HER2- advanced breast cancer with PIK3CA mutation

Phase Phase 3

Patients 572

Primary Outcome

Measures

Progression-free survival (PFS) for patients with PIK3CA

mutant status

Arms/Intervention• Fulvestrant 500 mg + alpelisib 300 mg

• Fulvestrant 500 mg + placebo

Target Patients

Men and postmenopausal women with hormone receptor

positive, HER2-negative advanced breast cancer which

progressed on or after aromatase inhibitor treatment


Publication

• Andre F, et al. Presentation at ESMO 2018

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

1940.


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

Study NCT00940602 TELESTO (CICL670A2302)

Indication Iron overload

Phase Phase 2

Patients 224

Primary Outcome

Measures

To compare deferasirox to placebo with regard to event-free

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

iron overload

Arms/Intervention• Deferasirox, iron chelator

• Placebo

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

transfusional iron overload


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

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


INC280 - MET Inhibitor

Study NCT02414139 (CINC280A2201) NCT03647488 (CINC280D2201)

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

Lung Cancer (NSCLC)Non-small cell lung cancer


Patients 364 105

Primary Outcome

MeasuresOverall Response Rate (ORR)

Run in part: Assess safety and tolerability of capmatinib and

spartalizumab combination.

Randomized part: Overall Survival (OS)

Arms/Intervention

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

• Pre-treated pts. with MET mutations regardless of

cMET GCN as second or third line

• Treatment-naïve pts. with MET dysregulation

• Pre-treated pts with MET dysregulation – second line

• Treatment-naïve pts with cMET mutations regardless of

cMET GCN

• Capmatinib plus spartalizumab

• Docetaxel

Target Patients

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

advanced/ metastatic NSCLC with either MET

amplification or MET mutations

Pre-treated adult patients with EGFR wild-type ALK

rearrangement negative advanced/metastatic non-small cell

lung cancer, that has demonstrated progression following one

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

inhibitor

Expected Completion Q2-2019 (actual) 2022

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

• Wolf J, et al. Manuscript submitted Q3-2019TBD


Jakavi® - JAK1/2 inhibitor

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 SR aGVHD Patients with SR cGVHD

Expected Completion Q3-2019 (actual) Interim Analysis: Q3-2019 (actual); Final: Q3-2020

Publication• Manuscript submission in Q4-2019

• Zeiser R, et al. Abstract submitted Q4-2019

• Manuscript submission in H2-2020

• Abstract submission to congress in H2-2020


Jakavi® - JAK1/2 inhibitor

Study NCT03491215 REACH4 (CINC424F12201) NCT04097821 ADORE (CINC424H12201)

Indication Acute graft versus host disease Myelofibrosis

Phase Phase 2 Phase 1/2

Patients 45 130

Primary Outcome

Measures

• Measurement of PK parameters

• Overall Response Rate (ORR)

• Incidence of dose limiting toxicities within the first 2

cycles

• Response rate at the end of cycle 6

Arms/Intervention • Ruxolitinib

• Ruxolitinib

• Ruxolitinib+Siremadlin

• Ruxolitinib+Crizanlizumab

• Ruxolitinib+MBG453

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

after allogeneic hematopoietic stem cell transplantationPatients with Myelofibrosis (MF)


Publication TBD TBD


Kisqali® - CDK 4/6 inhibitor

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

Expected Completion Interim Analysis: H1-2021; Final: 2026

Publication TBD


Kymriah® – CAR-T therapy

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) Interim Analysis: Q3-2020

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

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

Expected Completion 2025 H2-2021

Publication TBD TBD


MBG453 – TIM-3 antagonist

Study NCT03946670 STIMULUS MDS-1 (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

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


Publication Abstract submission to congress in H2-2020


Rydapt®- Multi-targeted kinase inhibitor

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) from pan-Asia countries

Newly diagnosed pediatric patients with FLT3 mutated acute

myeloid leukemia (AML)


Publication Abstract submission to congress in H2-2020 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


Promacta®/Revolade® – Thrombopoetin receptor agonist

Study NCT03025698 (CETB115E2201) NCT03988608 (CETB115E2202)

IndicationPreviously untreated or relapsed/refractory severe aplastic

anemia or recurrent aplastic anemia

Previously untreated or relapsed/refractory severe aplastic

anemia or recurrent aplastic anemia


Patients 60 20

Primary Outcome

Measures

PK of eltrombopag at steady state in pediatric patients with

SAAHematologic response rate

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

• Eltrombopag 25 mg film-coated tablets

Target Patients

Pediatric patients from age 1 <18 years with

relapsed/refractory SAA or recurrent AA after IST or

previously untreated SAA

Chinese patients with refractory or relapsed severe aplastic

anemia


Publication TBD 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


Study NCT01677741 (CDRB436A2102)

Indication BRAFV600 mutant cancers

Phase Phase 1/2

Patients 85

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics

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

and weight)

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

BRAF V600-mutation positive solid tumors


Publication

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

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

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

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

gliomas)

Tafinlar® - BRAF inhibitor


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


Tafinlar®+Mekinist® - BRAFV600 inhibitor and MEK inhibitor

Study NCT02124772 (CTMT212X2101)

Indication BRAFV600 mutant solid tumors

Phase Phase 1/2A

Patients 142

Primary Outcome

MeasuresSafety, tolerability and pharmacokinetics and clinical activity

Arms/InterventionTrametinib (dose based on age and weight)

Dabrafenib + trametinib (dose based on age and weight)

Target PatientsPediatric Subjects Aged 1 Month to <18 Years with

Advanced V600-Mutation Positive Solid Tumors


Publication Abstract submission to congress in H1-2020


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): Q4-2019

Publication

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

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

• Final (ORR): TBD


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

Study NCT03511664 VISION (PSMA-617-01)

IndicationPSMA-positive Metastatic Castration-resistant Prostate

Cancer (mCRPC)

Phase Phase 3

Patients 814

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)


Publication TBD


Ophthalmology

Lucentis® - Anti-VEGF

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.

Expected Completion Q1-2018 (actual) 2023

Publication

• EURETINA: Sep-2018

• AAO: Oct-2018

• Primary manuscript published online by The Lancet in

Sep-2019

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

-6736(19)31344-3.pdf)

TBD


RTH258 - Anti-VEGF

Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)

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


Patients 743 1,082

Primary Outcome

Measures

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Change in Best Corrected Visual Acuity (BCVA) from

baseline at week 48

Arms/Intervention• Brolucizumab (RTH258) 6 mg/50 µL

• Aflibercept 2 mg/50 µL

• Brolucizumab (RTH258) 3 mg/50 µL



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


Publication

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

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

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

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

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

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

AAO Oct-2019 and APVRS Dec-2019


RTH258 - Anti-VEGF

Study NCT03386474 (CRTH258A2301E1) NCT03481634 KESTREL (CRTH258B2301)

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


Patients 150 534

Primary Outcome

MeasuresNumber of treatment-emergent adverse events

Change from baseline in best-corrected visual acuity

(BCVA)





• Aflibercept 2mg/50 uL

Target PatientsPatients with neovascular age-related macular degeneration

who have completed the CRTH258A2301 study

Patients with visual impairment due to diabetic macular

edema (DME)

Expected Completion Q3-2018 (actual) H2-2021

Publication TBD TBD


RTH258 - Anti-VEGF

Study NCT03481660 KITE (CRTH258B2302) NCT04058067 KINGLET (CRTH258B2304)

Indication Diabetic eye disease Diabetic macular edema


Patients 356 268

Primary Outcome

Measures


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





Target PatientsPatients with visual impairment due to diabetic macular

edema (DME)

Patients with visual impairment due to diabetic macular

edema


Publication TBD TBD


RTH258 - Anti-VEGF

Study NCT03917472 KINGFISHER (CRTH258B2305) NCT03802630 RAPTOR (CRTH258C2301)

Indication Diabetic macular edema Retinal vein occlusion


Patients 500 500

Primary Outcome

Measures

Change in best-corrected visual acuity (BCVA) from

baseline up to week 52


(BCVA) at week 24





Target PatientsPatients with visual impairment due to diabetic macular

edema

Adult patients with visual impairment due to macular edema

secondary to branch retinal vein occlusion


Publication TBD TBD


RTH258 - Anti-VEGF

Study NCT03810313 RAVEN (CRTH258C2302) NCT04047472 HOBBY (CRTH258A2307)

Indication Retinal vein occlusion Macular degeneration


Patients 750 494

Primary Outcome

Measures


(BCVA) at week 24


(BCVA) at week 48





Target PatientsAdult patients with visual impairment due to macular edema

secondary to central retinal vein occlusion

Chinese patients with neovascular age-related macular

degeneration


Publication TBD TBD


UNR844 - Disulfide bonds modulator

Study NCT03809611 (CUNR844A2203)

Indication Presbyopia

Phase Phase 2

Patients 124

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

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 For Submission: Q4-2019 (actual); Final: 2022 Q3-2020

Publication TBD TBD


QBW251 - CFTR potentiator

Study NCT04072887 (CQBW251B2201)

Indication Chronic obstructive pulmonary disease (COPD)

Phase Phase 2

Patients 900

Primary Outcome

Measures

Trough FEV1 (Forced Expiratory Volume in 1 second)

change from baseline after 12 weeks of treatment

Arms/Intervention

• QBW251 450 mg

• QBW251 300 mg

• QBW251 150 mg

• QBW251 75 mg

• QBW251 25 mg

• Placebo

Target PatientsCOPD patients on background triple inhaled therapy (LABA /

LAMA / ICS)


Publication TBD


QMF149 - Long-acting beta2 agonist and inhaled corticosteroid

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 Planned in H2-2020


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

Study NCT02554786 PALLADIUM (CQVM149B2301) NCT02571777 IRIDIUM (CQVM149B2302)




Primary Outcome

MeasuresTrough FEV1 Trough FEV1

Arms/Intervention

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• MF 400 µg od

• MF 400 µg bid

• Salmeterol 50 µg /fluticasone 500 µg bid

• QVM149 150/50/160 µg od

• QVM149 150/50/80 µg od

• QMF149 150/160 µg od

• QMF149 150/320 µg od

• Salmeterol 50 µg /fluticasone 500 µg bid

Target Patients

Adult and adolescent (≥12 years) patients with asthma

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

dose LABA/ICS (GINA step ≥ 3)

Adult (≥18 years) patients with asthma inadequately

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


Publication• Planned in H1-2020

• Abstract: van Zyl-Smit et al, presented at BTS Dec-2019

• Planned in H1-2020

• Abstract ATS Q2-2020



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

• Abstract for ATS in Q2-2020


• Abstract for ATS in Q2-2020



Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)



Patients 802 1,251

Primary Outcome

MeasuresTrough FEV1

Non-inferiority of Asthma Quality of Life Questionnaire

(AQLQ)

Arms/Intervention• QMF149 150/80 µg od

• MF 200 µg od

• QVM149 150/50/80 μg od

• QVM149 150/50/160 μg od

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

Target Patients

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

inadequately controlled on low-dose ICS or low-dose

LABA/ICS (Gina step 2-3)

Patients with uncontrolled asthma


Publication• O. Kornmann et al. Respiratory Medicine 161 (2020)

• Abstract: D’Andrea et al, presented at ERS Sep-2019


• Abstract ATS Q2-2020


Xolair ® – anti-IgE antibody

Study NCT03369704 (CIGE025F1301)

Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis

Phase Phase 3

Patients 337

Primary Outcome Measures Mean 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

• Poster published at EAACI (the European Academy of Allergy and Clinical Immunology), Jun 2019

• Oral presentations were made at JRS (Japanese Rhinologic Society), Oct 2019, and Asian Pacific Society of

Respirology congress, Nov 2019

• Planned oral/poster presentation at Japan society of Immunology & Allergology in Otolaryngology, Feb 2020

• Planned manuscript to be submitted to JACI in Practice, Q1 2020


Sandoz Biopharmaceuticals

Hyrimoz® - Biosimilar adalimumab

Study NCT02744755 ADMYRA (GP17-302)

Indication Immunology

Phase Phase 3

Patients 353

Primary Outcome

Measures

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

patients treated with GP2017 and patients treated with

Humira®

Arms/Intervention• GP2017

• US licensed Humira® adalimumab

Target Patients Patients with moderate to severe active rheumatoid arthritis

Expected Completion 2018 (actual)

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

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


GP2411 - Biosimilar denosumab

Study NCT03974100 (CGP24112301)

Indication Osteoporosis

Phase Phase 3

Patients 522

Primary Outcome

Measures

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

Mineral Density

Arms/Intervention

• GP2411 60 mg /mL subcutaneous injection every 6

months

• Prolia® 60 mg /mL subcutaneous injection every 6

months

Target Patients Postmenopausal women with osteoporosis


Publication Study data publications expected for 2024 and beyond


Global Health

KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated

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

Study NCT03334747 (CKAE609A2202)

Indication Malaria

Phase Phase 2

Patients 210

Primary Outcome

Measures

CTCAE grades increase from baseline in alanine

aminotransferase (ALT) or aspartate aminotransferase

(AST)

Arms/Intervention• KAE609

• Coartem

Target Patients Adults with uncomplicated Plasmodium Falciparum malaria


Publication TBD


q4 and fy 2019 results - novartis

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