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Disclaimer
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 2
This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by
words such as “potential,” “expected,” “will,” “planned,” “pipeline,” “outlook,” or similar expressions, or by express or implied discussions regarding potential new products, potential
new indications for existing products, or regarding potential future revenues from any such products; or regarding the potential outcome, or financial or other impact on Novartis, of
the proposed spin-off of our Alcon Division, or of the proposed divestiture of certain portions of our Sandoz Division business in the US; or regarding the potential impact of the share
buyback plan; or regarding potential future sales or earnings of the Group or any of its divisions or potential shareholder returns; or regarding potential future credit ratings of the
Group; 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, payer and general public
pricing and reimbursement pressures and requirements for increased pricing transparency; regulatory actions or delays or government regulation generally, including potential
regulatory actions or delays with respect to the proposed transactions or the development of the products described in this Annual Report; the potential that the strategic benefits,
synergies or opportunities expected from the proposed transactions may not be realized or may take longer to realize than expected; the inherent uncertainties involved in predicting
shareholder returns; the uncertainties inherent in the research and development of new healthcare products, including clinical trial results and additional analysis of existing clinical
data; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity
on key products that commenced in prior years and will continue this year; safety, quality or manufacturing issues; uncertainties regarding actual or potential legal proceedings,
including, among others, actual or potential litigation with respect to the proposed transactions, product liability litigation, litigation and investigations regarding sales and marketing
practices, intellectual property disputes and government investigations generally; uncertainties involved in the development or adoption of potentially transformational technologies
and business models; our performance on environmental, social and governance measures; general political, economic and trade conditions, including uncertainties regarding the
effects of ongoing instability in various parts of the world; uncertainties regarding future global exchange rates; uncertainties regarding future demand for our products; uncertainties
regarding potential significant breaches of data security or data privacy, or disruptions of our information technology systems; and other risks and factors referred to in Novartis AG’s
current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this presentation as of this date and does not undertake any
obligation to update any forward-looking statements as a result of new information, future events or otherwise.
Vas NarasimhanCompany overview
Harry KirschFinancial review
Paul HudsonPharma highlights
Susanne SchaffertOncology highlights
Agenda
We aim to become a leading medicines company Powered by advanced therapy platforms and data science
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 5
Go big on data and digital
Passionate about
productivity and margins...
Embrace operational excellence
Build trust with society
While building a new culture
and lasting impact
Unleash the power of our people
Transform Sandoz
We are focusing
the company
Spin Alcon
Building advanced therapy platforms
Driving growth through
cutting-edge platforms...
Leading pipeline
We delivered strong growth with operating leverage in Q4 and FY 2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 6
1. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 53 of the Condensed Financial Report
Q4 (% cc) Full year (% cc)
Sales Core OpInc Sales Core OpInc
Group1 +6% +11% +5% +8%
Innovative Medicines +9% +13% +8% +11%
Sandoz -2% -5% -3% -3%
Alcon +4% 0% +5% +10%
We aim to become a leading medicines company Powered by advanced therapy platforms and data science
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 7
Go big on data and digital
Passionate about
productivity and margins...
Embrace operational excellence
Build trust with society
While building a new culture
and lasting impact
Unleash the power of our people
Transform Sandoz
We are focusing
the company
Spin Alcon
Building advanced therapy platforms
Driving growth through
cutting-edge platforms...
Leading pipeline
We took major steps to focus the company in 2018, while building leading advanced therapy platforms
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 8
All trademarks are the property of their respective owners 1. Announced or closed in 2018 2. The planned 100% spin-off of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary
authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February 2019; completion expected in H1 2019 3. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo is subject to
the completion of customary closing conditions expected in 2019 4. Sale of our anti-bacterial portfolio to Boston Pharmaceuticals is one example of portfolio prioritization. Others include out-licensing of BJG398 to QED Therapeutics, FGF401
to EverNov, LXS196 to IDEAYA.
Deals1 to build new platformsExits1 to focus the company
Gene
therapy
Cell
therapy
Radioligand
therapy
2
3
4
Alcon delivered margin expansion and consistent growth; on track for spin-off H1 20191
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 9
2016 2017 2018
17.6%17.3%
17.9%
1. The planned 100% spin-off of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February
2019; completion expected in H1 2019 2. Alcon Division 2016 and 2017 net sales and core margin restated to include the Ophthalmic OTC and Diagnostics products, transferred from the Innovative Medicines Division from January 1, 2018;
figures above represent Alcon results as a division of Novartis and as such do not include costs associated with establishing Alcon as a separate company, which are expected to be approximately 1% of sales
Strong 2018 results
Sales growth driven by innovation; ATIOLs, Systane®
Complete and Dailies Total1® Multifocal
Margin expansion driven by higher sales and
improved gross margin
Capital markets days held Q4 2018; further investor
engagement planned Q1 2019
Shareholder vote on spin-off February 28 at AGM1
Share ratio of 5 Novartis shares: 1 Alcon share
On track for spin-off Q21; see shareholder brochure:https://www.novartis.com/sites/www.novartis.com/files/2019-novartis-agm-alcon-en.pdf
Alcon core
margin2
% of USD sales
2016 20182017
-1%
4%5%Alcon full
year sales
growth
cc
Our Sandoz transformation will help enable us to compete in a more challenging environment
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 10
Reshaping the portfolio... ...while driving efficiency
Geographic focusLeading in biosimilars: 8 marketed products, more to come Lean cost structure
SKU
rationalization
Manufacturing
footprint
optimization
Regional
consolidation
All trademarks are the property of their respective owners 1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo is subject to the completion of customary closing conditions expected in 2019
1
1. Investments in organic business
2. Growing annual dividend in CHF
3. Value-creating bolt-ons2
4. Share buybacks
We remain disciplined and shareholder-focused in our capital allocation
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 11
100% tax-neutral spin1 of Alcon to shareholders
to enable focus on core medicines business
Proposing 22nd consecutive dividend increase3
~USD 15bn M&A bolt-ons in 2018
100+ collaboration and licensing deals completed
~USD 17bn of net share buybacks over past 5 years,
including purchases under new USD 5bn program4
Novartis
priorities
1. The planned 100% spin-off of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in
February 2019; completion expected in H1 2019 2. Includes M&A and BD&L 3. Proposed dividend increase for 2018 at AGM 2019 4. Share buyback of up to USD 5bn announced on June 29, 2018
We aim to become a leading medicines company Powered by advanced therapy platforms and data science
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 12
Go big on data and digital
Passionate about
productivity and margins...
Embrace operational excellence
Build trust with society
While building a new culture
and lasting impact
Unleash the power of our people
Transform Sandoz
We are focusing
the company
Spin Alcon
Building advanced therapy platforms
Driving growth through
cutting-edge platforms...
Leading pipeline
Our in-line growth brands provide a solid foundation for continued growth
13 Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
FY Sales USD million
vs. PYcc
Growth vs. PYUSD million
766
200
167
521
307
282
119
159
70
2,837
1,028
1,174
1,155
977
167
235
1,039
76
36%
102%
35%
31%
24%
nm1
nm1
12%
nm1
1 Not meaningful
4 additional blockbusters in 2018
Lutathera®
With 10+ potential blockbuster1 launches planned in the next 2 years
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 14
1. Individual assets with expected peak sales >USD 1bn across all indications 2. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312) , but the product itself has
not been approved for sale in any country 3. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received
marketing authorization or BLA approval from any regulatory authorities
2020
INC280 NSCLC
OMB157Relapsing MS
PDR001 comboMetastatic Melanoma
QVM149Asthma
SEG101Sickle Cell Disease
Entresto®
HFpEF
Cosentyx®
nrAxSpA
2018
Lutathera®
NET
Kymriah®
DLBCL
Aimovig®
Migraine
2019
RTH258nAMD
Zolgensma™3
SMA Type 1
BYL719Advanced breast cancer
Mayzent™2
SPMS
Launched 2018
3
4
7
Planned launches
2019 expected catalysts to continue the momentum
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 15
1. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from
any regulatory authorities 2. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product itself has not been approved for sale in any country
Catalysts Selected examples
Key
approvals15 Zolgensma™1
SMA Type 1 (US/EU/JP)
Mayzent™2
SPMS (US/EU/JP)
Brolucizumab (RTH258)Neovascular AMD (US)
Alpelisib (BYL719)Breast Cancer (US)
Major
submissions20 Ofatumumab (OMB157)
Relapsing MS (US/EU)
Crizanlizumab (SEG101)Sickle Cell Disease (US/EU)
Brolucizumab (RTH258)Neovascular AMD (US/EU/JP)
INC280NSCLC (US/JP)
PDR001 comboMetastatic Melanoma (US/EU)
Major
late-stage
readouts
6 Zolgensma™1
SMA Type 2
Fevipiprant (QAW039)Asthma
Entresto®
HFpEF
Cosentyx®
nrAxSpA
Ofatumumab (OMB157)Relapsing MS
PDR001 comboMetastatic Melanoma
Advanced therapy platforms with the potential to expand the game-board
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 16
Small
molecules
Large
molecules
Cell
therapy
Gene
therapy
Radioligand
therapy
Oncology
Cardio-Metabolic
IHD
Neuroscience
Ophthalmology
Respiratory
(Illustrative)
1. Partnership with the Wyss Institute for Biologically Inspired Engineering at Harvard University and the Dana-Farber Cancer Institute 2. Collaboration with Xencor 3. Collaborations with Intellia Therapeutics and Caribou Biosciences
4. Proposed acquisition; transaction subject to the completion of customary closing conditions 5. Collaboration with Cellular Biomedicine Group in China
AAA EndocyteCAR-TCBMG5
Cell for
Cure4
Intellia &
Caribou3
Bispecific
antibodies2Novel
bio-materials1
Transcription
factors
Targeted
protein
degradation
Inhaled
biologics
Novel
bio-materials
AveXis
NIBR
Portfolio
Luxturna®
7 programs in clinic over next yearManufacturing expansion ongoing for AAV-based
gene therapies
Gene therapy platform with rapidly expanding pipeline, deep manufacturing expertise in AAV
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 17
Flexible, disposable
manufacturing platform
Operational facility in
Chicago; ongoing build-
out in North Carolina
Capabilities across AAV
vectors; deep CMC
expertise
Selected assets Indication Stage Next milestone
AVXS-101 (AAV9) SMA Filed Regulatory approval(s) 1H19
CGF166 (Ad5) Hearing loss Phase 1
CPK850 (AAV8) Retinitis pigmentosa Phase 1
AVXS-201 RTT (AAV9) Rett Syndrome Preclinical IND 1Q19
AVXS-301 SOD1 (AAV9) InheritedALS-SOD1 Preclinical IND 2Q19
AVXS-401 Undisclosed Preclinical IND 2H19
AVXS-501 Undisclosed Preclinical IND 4Q19 / 1Q20
We aim to become a leading medicines company Powered by advanced therapy platforms and data science
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 18
Go big on data and digital
Passionate about
productivity and margins...
Embrace operational excellence
Build trust with society
While building a new culture
and lasting impact
Unleash the power of our people
Transform Sandoz
We are focusing
the company
Spin Alcon
Building advanced therapy platforms
Driving growth through
cutting-edge platforms...
Leading pipeline
We are committed to driving consistent margin expansion
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 19
1. Source: Novartis analysis of average 2016 core margin of Large Pharma peer companies
+ Acceleration of key growth drivers
+ Resource allocation and productivity
programs in commercial units
+ Cross-divisional synergies: Novartis
Technical Operations, Novartis Business
Services, Procurement
− Generics (mainly Afinitor®, Exjade®, Gilenya®)
− Launch investments for potential future
blockbustersIM Division
2017
IM Division
2018
Large Pharma
average1
3132
Mid 30s
Innovative MedicinesCore margin (%)
With an aggressive productivity agenda
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 20
Initiative Goal Progress
NTONovartis Technical
Operations ~USD 2bn
savings by 2020
primarily driven by
these functions1;
increased focus on
cash conversion cycle
Announced 16 plant transformations in 2018, including 8 exits
Announced significant restructuring in Switzerland
Developing predictive capabilities to reduce inventory, increase efficiency & automation
NBSNovartis Business
Services
Driving transformation through standardization, simplification & footprint optimization
Announced global restructuring plans
Launched effort to automate high-impact global processes
ProcurementCross-Divisional
Savings targets in all units across major spend categories
Recruited new Head of Procurement from low-margin industry
1. Includes USD 1bn of savings from the NTO network transformation announced in January 2016.
Advancing an enterprise-wide digital transformation
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 21
We’re reimagining medicine with data and digitalOur digital lighthouse projects are kick-starting our enterprise-wide transformation but we need the
commitment of all 125,000 of us to make it a success. Let’s #GoDigital!
Click on the hotspots below for more information
Mining clinical trial
data for new insights
DATA42
Bringing clinical trials
to the patient
Patient-centric remote
trials
Combining medicines
with cutting-edge
technology
Digital therapeutics
Transforming Pharma
commercial models
Pharma ACT
Data repository
for Oncology
DROID
End-to-end tender
optimization
AI tender
Innovative digital
solutions for customers
Around/beyond the pill
End-to-end
process automation
NBS 2.0
Building digital
capabilities
HR
More ef cient
manufacturing
NTO 2.0
Launch excellence
along the patient funnel
Meeting patients and
HCPs along the journey
innovateStrengthening our
innovation core
operateWorking smarter to deliver greater
value
engageMaximizing the return
of our commercial operations
Digital community Tech enablers Data & advanced analytics External partners Digital awareness hub
More efficient
manufacturing
Data & advanced analytics
All trademarks are the property of their respective owners
We aim to become a leading medicines company Powered by advanced therapy platforms and data science
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 22
Go big on data and digital
Passionate about
productivity and margins...
Embrace operational excellence
Build trust with society
While building a new culture
and lasting impact
Unleash the power of our people
Transform Sandoz
We are focusing
the company
Spin Alcon
Building advanced therapy platforms
Driving growth through
cutting-edge platforms...
Leading pipeline
Culture transformation is key to our successInitiated 5-year journey in 2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 23
Reshaped to a more diverse Executive Committee (over 50% new members)
Rolled out new culture vision to create an empowered organization
100% of top leaders going through leadership “academy”; 100% of managers
to receive digital upward feedback on an ongoing basis
Digitally enabled tools
Inspired
Curious
Unbossed
Focused effort to build lasting trust with society
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 24
Established Ethics, Risk &
Compliance function, led by
Executive Committee member
Embedding principles-based
decision-making in the
organization
Ethical
Standards
Integrating Access
Principles into overall
business strategy
Improved ranking in Access
to Medicines Index to #2
Pricing and
Access
Renewed commitment to
malaria with USD 100m
investment
Established Global
Partnership for Zero Leprosy
Global Health
Challenges
Approved new environmental
sustainability targets, incl.
carbon neutrality by 2025
Helped lead Pat-INFORMED
initiative, making patents
available online
Corporate
Citizenship
Continued to improve
transparency and evolve
reporting
Increased reporting on
Financial, Environmental and
Social (FES) impact on society
Stakeholder
Engagement
Well-positioned for top and bottom-line growth
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 25
In-market growth drivers
Cosentyx®, Entresto®, Kisqali®, Lutathera®, Kymriah®, Promacta®, Tafinlar®+Mekinist®, Jakavi®, Biosimilars
15 in-market
blockbusters
as of YE 2018
10+ potential
blockbusters expected
to launch by 2020
Savings of ~USD 2bn
expected by 20201
Patent expiries
primarily in near term
Key launches
Zolgensma™, Mayzent™, RTH258, Aimovig®, OMB157, BYL719, SEG101, Biosimilars
Productivity improvements
NTO transformation, NBS transformation, Procurement initiative, Digital initiatives
Generic erosion
Exjade®
+
+
+
–
2019 2020 2021
Gilenya® (timing TBC)2Afinitor®
NTO – Novartis Technical Operations NBS – Novartis Business Services The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product
itself has not received marketing authorization or BLA approval from any regulatory authorities. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product
itself has not been approved for sale in any country. 1. Includes USD 1bn of savings from the NTO network transformation announced in January 2016 2. Gilenya US compound patent expiration August 2019; dosing regimen patent expiration
December 2027
(Illustrative)
2018 financial results in line with guidance
FY 2018
vs. PYIn cc
Group full year guidance (January 2018)In cc
“Sales are expected to grow low to mid single digit” 5%
8%“Core operating income expected to grow mid to high
single digit”
27
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
% USD % cc % USD % cc
Net Sales 13,269 3 6 51,900 6 5
Core Operating income 3,387 5 11 13,823 8 8
Operating income 1,299 -37 -29 8,169 -5 -5
Net Income 1,194 -40 -32 12,614 64 64
Core EPS (USD) 1.25 3 9 5.15 6 6
EPS (USD) 0.52 -39 -32 5.44 66 66
Free Cash Flow 2,939 20 11,717 12
Change vs. PYGroup
1
USD million
Q4
2018
FY
2018
Change vs. PY
Summary of Q4 and FY 2018 financial results
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 28
1. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 53 of the Condensed Financial Report
Net sales
change vs. PY
Core operating
income
change vs. PY Core margin
Core margin
change vs. PY Core margin
Core margin
change vs. PY
(in % cc) (in % cc) (%) (%pts cc) (%) (%pts cc)
Innovative Medicines 9 13 30.7 1.1 32.0 1.0
Sandoz -2 -5 19.6 -0.6 20.3 -0.1
Alcon 4 0 15.7 -0.7 17.9 0.8
Group 6 11 25.5 1.2 26.6 0.7
Q4 2018 FY 2018
Innovative Medicines driving Q4 margin expansion; FY margin increase driven by IM Division and Alcon
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 29
2018 Key drivers vs. PY:
+ Cash flows from operating activities, mainly:
+ Higher core operating income
+ GSK milestone receipt (divested Vaccines business)
− Higher net intangible investments
FY 2018 free cash flow at USD 11.7bn
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 30
FY 2016 FY 2017 FY 2018
10.49.5
11.7
+12%
Group free cash flow USD billion
Novartis proposes 22nd consecutive dividend increase to the AGM: 2.85 CHF / share1
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 31
1. Proposal to shareholders at the 2019 Annual General Meeting, taking place on February 28, 2019 2. Converted at historic exchange rates at the dividend payment dates as per Bloomberg; assumes an exchange rate of USD / CHF of
0.9862 as of December 31, 2018 for 2018
0.0
0.5
1.0
1.5
2.0
2.5
3.0
20101996 1997 1998 1999 200220012000 2003 2004 2005 2006 20092007 2008 2011 2012 2013 2014 2015 2016 2017 2018
CH
F 2
.80
US
D 2
.94
CH
F 2
.85
US
D 2
.89
CHF USD
2019 Novartis full year guidanceBarring unforeseen events (in cc); Growth vs PY in cc
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 32
Current Group structureNo change to current Group Structure
New focused medicines companyExcl. Alcon1 & Sandoz proposed US portfolio sale to
Aurobindo2 from both 2018 and 2019
1. The planned 100% spinoff of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February
2019; completion expected in H1 2019 2. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during 2019, is subject to the completion of customary closing conditions. Estimated 2018 FY
Sales and Core OpInc of the Sandoz US Oral solids and Dermatology businesses were approximately USD 1.2bn and 0.3bn, respectively.
Key Assumption: All guidance includes forecast assumption that no Gilenya® generics enter in 2019. However, generic competitors may still launch at risk
Net Sales Expected to grow low to mid single digit
IM Division to grow mid single digit
Sandoz to decline low single digit
Alcon to grow low to mid single digit
Expected to grow mid single digit
IM Division to grow mid single digit
Sandoz to be broadly in line with prior year
Core Operating
Income
Expected to grow mid single digit
Alcon Core OpInc margin expected to expand
Expected to grow mid to high single digit
FY 2019 Guidance on other financial KPIsBarring unforeseen events (in cc)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 33
New focused medicines companyExcl. Alcon1 & Sandoz proposed US portfolio sale to Aurobindo2 from both 2018 and 2019
Core
net financial
expense
FY expected to be broadly in line vs. 2018 current Group structure
Core
tax rateFY core tax rate expected to increase slightly to around 16%
1. The planned 100% spinoff of Alcon remains subject to certain conditions precedent, such as no material adverse events, receipt of necessary authorizations as well as tax rulings and opinions and shareholder approval at the AGM in February
2019; completion expected in H1 2019 2. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during 2019, is subject to the completion of customary closing conditions
Expected currency impact for full year 2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 34
Currency impact vs. PY%pts, assuming late-January exchange rates prevail in 2019
SimulationActual
FX impact on
Net sales
FX impact on
Core operating income
-3
1
-5-2
-6 -7-3
FYQ4 FY Q4Q1 FY Q1 FY
0
20182018 2019 2019
Cosentyx®: Strong growth driven by demand, well positioned across indications
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 36
Q4 sales USD 806m (+33% cc) with consistent growth
US (+34% cc) and ex-US (+32% cc)
- Continued demand growth, US YoY TRx +29% Dermatology,
+49% in Rheumatology1
- Gaining share in a growing and competitive psoriasis market
2019: expected to maintain strong access in US
Continue to advance science in psoriatic disease
- ARROW (readout expected end of 2019) expected to confirm
importance of IL-17A vs. IL-23
- Cosentyx® has demonstrated efficacy in the multiple
manifestations of psoriatic disease
PREVENT (nrAxSpA) read-out and submission
expected end 2019
Quarterly sales evolutionUSD million
Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4
0
20
40
60
80
100
120
2.1bn
410490
556615
2017 2018
580
701 750
2.8bnEx-USUS
806
US TRx1 (quarterly in 000s)
1. IMS NPA TRx, Cosentyx® restated in Aug 2017 to include free product, Q4 estimated with WE 12/21/2018 data
Entresto® achieves blockbuster status – reinforcing strong therapy position in heart failure1
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 37
Early indicators of accelerated hospital initiation (PIONEER & TRANSITION)
Significantly reduces NT-proBNP in stabilized ADHF patients (PIONEER)
Strong sales growth driven by execution & new data PIONEER data2 supports early Entresto® useComposite of Death, HF re-hospitalization, LVAD, Listing for Transplant
USD 318m (+76% cc) Q4 sales
Blockbuster in 2018 and doubling sales vs. 2017
ADHF – Acute Decompensated Heart Failure FIR – First Interpretable Results LVAD – Left Ventricular Assist Device 1. Entresto® is approved in HFrEF and ongoing HFpEF trial expected to read out 2019. 2. Published in New England
Journal of Medicine, Nov 2018: DOI: 10.1056/NEJMoa1812851
Global sales, USD million
84 110 128 185 200
239 271
318
Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4
ex-US US
2017 2018
507m
1,028m
Expected newsflow 2019 FIR: PARALLEL-HF (Japan registration trial HFrEF) Q2 2019; PARAGON-HF in HFpEF Q3 2019
46% reduction,
driven by a 44%
reduction in HF
hospitalization
SPMS SMA Type 1 nAMD
EXPAND demonstrates
unprecedented efficacy in SPMS
Targeting MS patients when
progression becomes noticeable,
regardless of relapses
FDA action date expected H1
CHMP opinion expected H2
Launch expected in both
geographies in 2019
2019: Preparing to launch next wave of growth drivers
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 38
Mayzent™1 (BAF312)
Rapid improvement in milestone achievement,
previously unseen survival benefit, and durable
effect
ICER draft report affirms value of Zolgensma™ up
to USD 4 to 5m at the appropriate ultra-rare
disease threshold3
Label discussion on-going with FDA; US and Japan
approvals expected H1 2019, and Europe mid-2019
SMA Type 2 STRONG data readout planned at
AAN 2019
Zolgensma™2 (AVXS-101)
Brolucizumab has the potential to
address important unmet needs in
nAMD – dries better
HAWK and HARRIER year-2 data
reaffirm superiority in key secondary
endpoints from year one versus
aflibercept in patients with nAMD
– Less retinal fluid4
– Fewer injections5
– Uncompromised vision6
On track for launch end 2019
RTH258 (brolucizumab)
See slide 58 for references
Oncology grew +9% (cc) FY 2018, behind continued strong performance of key growth drivers
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 40
Promacta®/Revolade®
USD m, % cc
867
1,174
FY 2018FY 2017
+35%
Tafinlar® + Mekinist®
873
1,155
FY 2017 FY 2018
+31%
Thrombopoietin receptor agonist with
the broadest indication
Strong SAA growth in Japan and 1L
SAA approval in US
Market leader in BRAF+ targeted therapy
Approved for adjuvant treatment of
melanoma across major geographies
including US & EU
50 – 75% of eligible myelofibrosis patients
treated with Jakavi®
Double digit growth for both myelofibrosis
and polycythemia vera indications
Jakavi®
777
977
FY 2017 FY 2018
+24%
Novartis building leadership in radioligand therapy with highly-complex, scaled, on-demand manufacturing capability
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 41
Lutathera® strong uptake continues
Strong launch momentum, Q4 sales USD 81m
In 2018 US: >1,400 new patients; >100 centers actively prescribing
Broad US payer coverage with >80% of lives covered
Following UK reimbursement in Q3, 19 centers actively prescribing
52
399
1,123
1,726
Q3 ’18Q1 ’18 Q2 ’18 Q4 ’18
US, number of doses
2018 FY sales:
USD 167m
HospitalIsotope supply Production
177Lu Isotope
Produced in nuclear reactors
Precursor Isotope176Lu
Target Isotope177Lu
Octreotide
DOTATATE
Lutathera®
labeling
DOTATATE
Aseptic vial filling
Lutathera®
Aseptic conditions
On demand production
Patient
delivery
Patient dose
Standard dose of 7.4GBq/cycle
Aseptic conditions
Requires unique manufacturing centers, supply chain
expertise, and relationships with nuclear medicine centers
Radioligand therapy platform with growing pipeline in solid tumors
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 42
Product Disease (target) Status
177Lu
PSMA-617
Prostate cancer
(PSMA)Ph3 VISION study
initiated 2Q 2018
177Lu
PSMA-R2 Ph1 / 2 study initiated 2Q 2018
68Ga
PSMA-R2Ph1 / 2 study initiated 2Q 2018
18F
CTT1057Ph1 study completed
177Lu
NeoBOMB1
Breast cancer
Colorectal cancer
Lung cancer
Prostate cancer
GIST (GRPR)
Ph1 study to open 1H 2019
68Ga
NeoBOMB1Ongoing Ph1 ISS in GIST
Ph2 study initiated 2Q 2018
177Lu
FF-10158
Glioblastoma
(Integrin
Alphavbeta 3/5)
Preclinical
68Ga
FF-10158 Preclinical
Preclinical Phase I Phase II Phase III Filing Marketed
Therapeutic
PET Diag.
PET Diagnostic
Therapeutic
PET Diagnostic
Therapeutic
PET Diag.
Therapeutic
Expands Novartis nuclear medicine platform
– 177Lu-PSMA-617 potentially first-in-class PSMA radioligand
therapy following successful launch of Lutathera®
– Opportunity to further develop 177Lu-PSMA-617 to enter
earlier lines of therapy
Ph3 VISION trial enrollment initiated for 177Lu-PSMA-617 in mCRPC
– FDA agreed to rPFS as an alternative primary endpoint to OS
– Expected rPFS read-out and filing in 2020
Endocyte further establishes leadership positionRadioligand therapy being explored across full range of solid
tumors
rPFS – radiographic progression free survival
Pipeline of 10 clinical programs and 4 FIH, with large
pre-clinical effort
CAR-T type Indication Phase 1 Ph 2/Pivotal Phase 3 Submitted Approved
CD19 CAR-T Pediatric & young adult r/r ALL
CD19 CAR-T r/r DLBCL
CD19 CAR-T DLBCL in 1st relapse
CD19 CAR-T r/r FL
CD19 CAR-Tr/r DLBCL in combination with
pembrolizumab
CD19 CAR-T Adult r/r ALL
CD19 CAR-T r/r CLL combination with ibrutinib
CD19 CAR-T Pediatric NHL
CD19 CAR-T1st L high risk pediatric and young
adult ALL
CD19 CAR-T r/r DLBCL combo with ibrutinib
Other targets
(UPenn partner)
BCMA&CD19, CD22&CD19,
CD123, EGFRv3
US, EU
US, EU
Starting 2019
Started 2018
Starting 2019
Starting 2019
Started 2018
Started 2018
Novartis cell therapy platform with broad pipeline and building global manufacturing scale for cell processing
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 43
MORRIS
PLAINS
CELL FOR
CURE1 FRAUNHOFER
STEIN
1. Currently operating through manufacturing agreement; proposed acquisition and transaction subject to the completion of customary closing conditions
Starting 2019
Starting 2019
Starting 2019
Preparing to manufacture
Currently manufacturing
CBMG
FBRI
Building global scale for lentiviral vector-based therapies;
established collaborations, working to deliver globally
Sickle cell
disease (SCD)
Preparing to launch the next growth drivers
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 44
PIK3CA mutation in ~40% of
HR+/HER2- aBC patients and
associated with poor prognosis;
currently no targeted treatments
Alpelisib in combination with
fulvestrant is the first and only
therapy specifically for
HR+/HER2- aBC patients1 with a
PIK3CA mutation
Potential US launch H2 2019
HR+/HER2-
breast cancer
Alpelisib (BYL719)
Vaso-occlusive crises (VOCs) remain
the most important unmet need in
SCD – associated with decrease in
QoL and increase in risk of organ
damage and death
Crizanlizumab significantly reduced
frequency of VOCs regardless of SCD
genotype and/or hydroxyurea use
Received FDA Breakthrough Therapy
designation in 2018; planned US and
EU filings H1 2019
Crizanlizumab (SEG101)
aBC – advanced breast cancer 1. Post-menopausal women who progressed on or after an aromatase inhibitor with or without a CDK4/6 inhibitor (study included one man)
Alpelisib
PI3K-α inhibitor
We will continue to drive our priorities in 2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 46
Deliver
financialsSales growth
and margin
expansion
Deliver pipeline targets,
incl. Zolgensma™,
Mayzent™, RTH258
and BYL719 approvals
Maintain high proportion
of first-in-class /
transformative assets
Extend leadership in
cell, gene and
radioligand therapies
Breakthrough
Innovation
Execute Alcon spin and
progress Sandoz
transformation
Drive productivity
through NTO and NBS
transformations
Deliver performance of
in-market growth drivers
Prepare for 10+ potential
blockbuster launches,
incl. 4 in 2019
Operational
Excellence
Scale top 5 digital
initiatives across the
company
Upskill digital
capabilities in all
units and functions
Build Novartis
position within digital
ecosystem
Data & Digital
Leadership
Continue to embed
principles-based
decision-making (P3)
Implement Novartis
Access Principles, with
every new innovative
medicine launch
having an access plan
Progress efforts in
global health
Building Trust
& Reputation
Continue 5-year
journey to transform
culture, with a focus on
strengthening
leadership capabilities
Further increase
diversity and inclusion
Culture
Transformation
Our in-line growth brands provide a solid foundation for continued growth
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 49
Q4 Full Year
SalesUSD million
Growth vs. PYUSD million cc
SalesUSD million
Growth vs. PYcc
806 33% 2,837 36%
318 76% 1,028 102%
Lutathera® 81 nm1 167 nm1
330 32% 1,174 35%
313 31% 1,155 31%
256 17% 977 24%
60 71% 235 nm1
28 nm1 76 nm1
268 14% 1,039 12%
191
75
25
133
81
67
28
22
21
1. Not meaningful
Key growth drivers 4 additional blockbusters in 2018
Cosentyx®: Gaining share in a growing psoriasis market
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 50
1. NRx Share in Dermatology from SHS November 2018. NRx Share amongst Dermatology writers All trademarks are the property of their respective owners
Cosentyx®
Tremfya®
Stelara®
TNFs
Others
Dermatology US NRx evolution1
Cosentyx® prescriptions (NRx) +65%1 in a growing US
biologics market (+25%)1 with 10+ treatment options in 20191
12M ending 11/2018
11%
12M ending 11/2017
14%
+25%
+65%
2018 net debt decreased by USD 2.8bn mainly driven by strong FCF, partly offset by the dividend payment
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 51
USD billion
-19.0-16.2
-7.0 -1.3
-13.9
Dec 31, 2017 Dividends M&A transactions1Treasury share
transactions, net
13.0
Proceeds from sale
of GSK consumer
healthcare joint
venture
11.7
Free Cash
Flow
0.3
Others Dec 31, 2018
+2.8
1. Including mainly USD 8.3bn for AveXis, Inc., USD 3.5bn for Advanced Accelerator Applications, S.A. and USD 1.8bn for Endocyte, Inc., all net of cash acquired
FY 2018 Core EPS +9% cc, excluding the impact from discontinuation of OTC JV core income
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 52
5.08
FY 2017 FY 2018 FY 2017
Adjusted1
FY 2018
Adjusted1
4.86
5.15
4.65
+6%+9%
Core EPSUSD, % cc
1. FY 2017 and 2018 core EPS adjusted to exclude core income recorded from OTC JV
Core EPS as reported
Key drivers vs. PY:
+ Higher core operating income
+ Lower shares from share buyback
programs
− Discontinuation of OTC JV core income
from April 1st, 2018
Advancing pipeline with 30 potential blockbusters1
Potential blockbusters by planned submission year
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 53
1. New molecular entity / new indication with expected peak sales >USD 1bn 2. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but
the product itself has not received marketing authorization or BLA approval from any regulatory authorities 3. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod
(BAF312), but the product itself has not been approved for sale in any country 4. Including combos
202120192018 202232020
INC280 NSCLC
OMB157Relapsing MS
PDR001 comboMetastatic Melanoma
RTH258nAMD
SEG101Sickle Cell Disease
QVM149Asthma
Entresto®
HFpEF
Cosentyx®
nrAxSpA
Zolgensma™2
SMA Type 1
Mayzent™3
SPMS
BYL719Advanced breast cancer
CNP520Alzheimer’s Disease
ECF843Dry Eye
HDM201Acute Myeloid Leukemia
CFZ533Multiple
CSJ117Severe Asthma
ZPL389Atopic Dermatitis
UNR844Presbyopia
VAY736Multiple
VPM087CRC / RCC
LJN4524
NASH
LOU064CSU
LNP023Nephropathy
MOR106Atopic Dermatitis
177Lu-PSMA-617mCRPC
QAW039Asthma
QGE031CSU / CIU
ACZ885Lung cancer
ABL001CML
Zolgensma™2
SMA Type 2/3
2019 expected pipeline milestones
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 54
H1 2019 H2 2019
Regulatory
decisions and
opinions
Mayzent™1 SPMS (US) Alpelisib (BYL719) HR+ Breast Cancer (US)
Kymriah® Ped / Young Adult r/r ALL (JP) Brolucizumab (RTH258) nAMD (US)
Kymriah® r/r DLBCL (JP) Lucentis® RoP (EU / JP)
Promacta® Severe aplastic anaemia (EU) Lucentis® Diabetic Retinopathy (EU)
Zolgensma™ SMA Type 1 (US / EU / JP) Mayzent™1 SPMS (EU / JP)
Xolair® Pollinosis (JP)
Submissions Brolucizumab (Q1 2019) nAMD (US / EU / JP) Cosentyx® NRAxSpA (US / EU / JP)
Crizanlizumab (SEG101) Sickle Cell Disease (US / EU) Entresto® HF-rEF (JP)
Mayzent™1 SPMS (JP) Entresto® HF-pEF (US / EU)
INC280 NSCLC (US / JP)
Ofatumumab (OMB157) Relapsing MS (US / EU)
PDR001 (combination with
Tafinlar® + Mekinist®) Metastatic Melanoma (US / EU)
QVM149 Asthma (EU / JP)
Major trial
readouts
Zolgensma™2 SMA Type 2 Cosentyx® nrAxSpA
Entresto® HF-pEF
Fevipiprant (QAW039) Asthma
Ofatumumab (OMB157) Relapsing MS
PDR001 (combination with
Tafinlar® + Mekinist®) Metastatic Melanoma
✓ Achieved ✕ Missed = On track
1. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product itself has not been approved for sale in any country 2. The brand name Zolgensma™
has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from any regulatory authorities
2018 pipeline milestones
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 55
H1 2018 H2 2018
Regulatory
decisions and
opinions
Kymriah® DLBCL (US) ✓ Aimovig™2 Migraine (EU) ✓
Tafinlar® + Mekinist® Adjuvant melanoma (US) ✓ Kymriah® Pediatric and young adult r/r ALL (EU) ✓
Lutathera® NET (US) ✓ Kymriah® DLBCL (EU) ✓
Gx Advair®1 Asthma, COPD (US) ✕ Tafinlar® + Mekinist® Adjuvant melanoma (EU) + ATC (US) ✓
Aimovig™2 Migraine (US) ✓ Gilenya® Pediatric MS (US) ✓
GP2017 Adalimumab BS (EU) ✓
LA-EP20065 Peg-filgrastim BS (EU) ✓
GP1111 Infliximab BS (EU) ✓
GP20136 Rituximab BS (US) ✕
Submissions ACZ885 CV risk reduction (US/EU) ✓ Mayzent™ SPMS (EU) ✓
Mayzent™ SPMS (US) ✓ RTH258 nAMD (US/EU) Q1 2019
Kisqali® Advanced BC (US/EU) ✓3 BYL719 HR+ BC (US/EU) ✓/✓
Cosentyx® AS (JP) ✓ Zolgensma™ SMA7 (US / EU / JP) ✓
Kymriah® Pediatric ALL + DLBCL (JP) ✓
Promacta® 1st line SAA (US/EU) ✓4
Major trial
readouts
Kisqali® Advanced BC (MONALEESA-3) ✓ LIK0668 Obesity ✓
LJN452 NASH (Interim Analysis) ✓ BYL719 HR+ BC ✓
INC280 ALK- cMET amplified NSCLC ✓
Entresto® HFpEF (Interim Analysis) ✓
✓ Achieved ✕ Missed
The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312) , but the product itself has not been approved for sale in any country. The brand name Zolgensma™ has
been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from any regulatory authorities
1. Complete Response Letter received from FDA after Q4 2017 results; Advair® is a registered trademark of Glaxo Group Ltd. 2. Aimovig™ is developed in collaboration with Amgen; 3. Indication expansion based on MONALEESA-3 & 7 results.
MONALEESA 3/7 trials approved in US in July, Europe in December; 4. Approved in US in November; 5. Positive CHMP opinion received 6. Complete Response Letter received from FDA after Q1 2018 results; 7. IV formulation in type 1 SMA;
8. Program discontinued
Added 2018
17. Psoriatic arthritis head-to-head study versus
adalimumab
18. Non-alcoholic steatohepatitis
19. Ankylosing spondylitis head-to-head study versus
adalimumab
20. Acute myeloid leukemia
21. Chronic Obstructive Pulmonary Disease
22. Secondary Progressive Multiple Sclerosis
23. IV formulation Spinal Muscular Atrophy Type 1
24. 1st line colorectal cancer / 1st line renal cell
carcinoma
25. IT formulation Spinal Muscular Atrophy Type 2/3
26. Metastatic castration-resistant prostate cancer
Planned filings 2018 to 2023
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 56
ABL001CML4 3rd line
QGE031CSU/CIU16
Entresto®
Post-acute myocardial infarction
RTH258Diabetic macular edema
QAW039Asthma
Entresto®
Heart failure (PEF)13
INC280NSCLC6
Cosentyx®
nrAxSpA12
OMB157Relapsing multiple sclerosis
RTH258nAMD7
2023202120202019 2022
Jakavi®Acute GVHD14
Combination abbreviations:
fulv fulvestrant
tmx tamoxifen
gsn goserelin
NSAI Non-steroidal aromatase inhibitor
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
Cosentyx®
PsA H2H17
Cosentyx®
AS H2H19
LAM320MDR8 tuberculosis
ZPL389Atopic dermatitis
Rydapt®AML20 (FLT3 wild type)
UNR844Presbyopia
SEG101Sickle cell disease
LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia
and others (same as originator)
INC280NSCLC6 (EGFRm)
Jakavi®Chronic GVHD14
PDR001 + Tafinlar®+Mekinist®
Metastatic BRAF V600+ melanoma
ECF843Dry eye
XolairNasal Polyps
ACZ885Adjuvant NSCLC
ACZ8851st Line NSCLC
ACZ8852nd Line NSCLC
Kymriah®
r/r Follicular Lymphoma
Kymriah®
CLL22
Kymriah®
r/r DLBCL in 1st relapse
QVM149Asthma
QMF149Asthma
PDR001 comboMetastatic Melanoma
RTH258Retinal vein occlusion
1. Secondary prevention of cardiovascular events
2. Diffuse large B-cell lymphoma
3. Severe aplastic anemia
4. Chronic myeloid leukemia
5. Long-acting release
6. Non-small cell lung cancer
7. Neovascular age-related macular degeneration
8. Multi-drug resistant
9. Breast cancer
10. Retinopathy of prematurity
11. Indolent Non-Hodgkin’s lymphoma
12. Non-radiographic axial spondyloarthritis
13. Preserved ejection fraction
14. Graft-versus-host disease
15. Neuroendocrine tumors
16. Chronic spontaneous urticaria / chronic idiopathic urticaria
AVXS-201Rett Syndrome
a) US filing, submitted in EU
awaiting HA acceptance
AVXS-101SMA Type 2/325
KAE609Malaria
EMA401Peripheral neuropathic pain
CNP520Alzheimer’s disease
VAY736Primary Sjoegren’s syndrome
KAF156Malaria
LJN452Non-alcoholic steatohepatitis
QBW251COPD21
Kisqali®HR+, HER2 (-) BC9 (adjuvant)
LMI070Spinal muscular atrophy
VAY785Non-alcoholic steatohepatitis
CAD106Alzheimer’s disease
VAY736Autoimmune Hepatitis
Kymriah+ pembrolizumab - r/r DLBCL
ABL001CML4 1st line
HDM201Acute myeloid leukemia
LOU064Chronic spontaneous urticaria
CFZ533Solid Organ Transplant
VPM087CRC 1L/RCC 1L24
CSJ117Severe Asthma
CFZ533Sjorgen’s Syndrome
LJC242Non-alcoholic steatohepatitis
LNP023IgA nephropathy
LNP023Membranous nephropathy
MOR106Atopic Dermatitis
LCI699 USCushing’s disease
Cosentyx®
Hidradenitis suppurativa
177Lu-PSMA-617mCRPC26
LJN452Non-alcoholic steatohepatitis
QBW251COPD
LMI070Spinal muscular atrophy
UNR844Presbyopia
VAY736Autoimmune Hepatitis
LOU064Chronic spontaneous urticaria
LNP023IgA nephropathy
MOR106Atopic Dermatitis
177Lu-PSMA-617mCRPC26
ABL001CML1 1st line
VAY736Primary Sjoegren’s syndrome
ZPL389Atopic dermatitis
INC280NSCLC2 (EGFRm)
VAY785b
Non-alcoholic steatohepatitis
PDR001 comboMetastatic Melanoma
Kymriah®
+ pembrolizumab - r/r DLBCL
VPM087CRC 1L/RCC 1L24
CFZ533Sjorgen’s Syndrome
LNP023Membranous nephropathy2
LCI699 EUCushing’s disease
Pipeline of key projects in confirmatory development
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 57
Early Clinical Trials Registration Trials – Phase III / Pivotal In Registration
Combination abbreviations:
fulv fulvestrant
ltz letrozole
tmx tamoxifen
gsn goserelin
NSAI Non-steroidal aromatase inhibitor
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
a) In collaboration with Amgen; companies
to co-commercialize in the US, Novartis
to have AMG 334 exclusive rights in rest
of world excluding Japan.
b) Approved in US, submitted in EU
c) US Submitted. EU submission Jan 2019
.
Lucentis®
ROP16
Promacta®/Revolade®
SAA17 1st line
SEG101Sickle cell disease
QAW039Asthma
RTH258nAMD6
LCI699 USCushing’s disease
ACZ885Adjuvant NSCLC2
ACZ8851st Line NSCLC2
INC280NSCLC2
ABL001CML1 3rd line
QGE031CSU/CIU3
PDR001 + Tafinlar®+Mekinist®
Metastatic BRAF V600+ melanoma
Lucentis®
Diabetic retinopathy
BAF312SPMS20
LAM320MDR15 tuberculosis
QMF149Asthma
QVM149Asthma
OMB157Relapsing multiple sclerosis
RTH258Diabetic macular edema
Rydapt®AML18 (FLT3 wild type)
LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia
and others (same as originator)
XolairNasal Polyps
RTH258Retinal vein occlusion
Kymriah®
CLL22
Kymriah®
r/r DLBCL in 1st relapse
CAD106Alzheimer’s disease
KAE609Malaria
EMA401Peripheral neuropathic pain
CNP520Alzheimer’s disease
ECF843Dry eye
KAF156Malaria
HDM201Acute myeloid leukemia
CFZ533Solid Organ Transplant
AVXS-101SMA Type 2/325
CSJ117Severe Asthma
AVXS-201Rett Syndrome
AVXS-101SMA Type 123
1. Chronic myeloid leukemia
2. Non-small cell lung cancer
3. Chronic spontaneous urticaria / chronic
idiopathic urticaria
4. Neuroendocrine tumors
5. Breast cancer
6. Neovascular age-related macular
degeneration
7. Secondary prevention of cardiovascular
events
8. Indolent Non-Hodgkin’s lymphoma
9. Non-radiographic axial spondyloarthritis
10. Psoriatic arthritis head-to-head study versus
adalimumab
11. Ankylosing spondylitis head-to-head study
versus adalimumab
12. Diffuse large B-cell lymphoma
13. Preserved ejection fraction
14. Graft-versus-host disease
15. Multi-drug resistant
16. Retinopathy of prematurity
17. Severe aplastic anemia
18. Acute myeloid leukemia
19. Acute lymphoblastic leukemia
20. Secondary Progressive Multiple Sclerosis
21. Long-acting release
22. Chronic Lymphocytic Leukemia
23. IV formulation Type 1 SMA
24. 1st line colorectal cancer / 1st line renal cell
carcinoma
25. IT formulation Spinal Muscular Atrophy Type 2/3
26. Metastatic castration-resistant prostate cancer
BYL719a/c + fulvHR+, HER2 (-) postmenopausal
adv. BC9 2nd line
Cosentyx®
nrAxSpA9
Entresto®
Heart failure (PEF)13
Entresto®
Post-acute myocardial infarction
Jakavi®Acute GVHD14
Cosentyx®
PsA H2H10
Cosentyx®
AS H2H11
Kisqali®
HR+, HER2(-) BC5 (adjuvant)
Jakavi®Chronic GVHD14
ACZ8852nd Line NSCLC2
Kymriah®
r/r Follicular Lymphoma
Cosentyx®
Hidradenitis suppurativa
LJC242Non-alcoholic steatohepatitis
References
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 58
Slide 38 – 2019: Preparing to launch next wave of growth drivers
1. The brand name Mayzent™ has been provisionally approved by the FDA and EMA for the investigational product siponimod (BAF312), but the product itself has not been
approved for sale in any country 2. The brand name Zolgensma™ has been provisionally approved by the FDA for the investigational product AVXS-101 (onasemnogene
abeparvovec-xxxx), but the product itself has not received marketing authorization or BLA approval from any regulatory authorities 3. Institute for Clinical Economic Review (ICER)
Draft Evidence Report, page 79 https://icer-review.org/wp-content/uploads/2018/07/ICER_SMA_Draft_Evidence_Report_122018-1.pdf 4. Week 48 – demonstrated superiority in
three secondary endpoints considered key markers of nAMD in clinical practice: central subfield retinal thickness, retinal fluid (intraretinal fluid and/or subretinal fluid) and disease
activity; advantages maintained at Week 96 5. Week 48 – majority of patients (56% and 51%) were maintained on q12w injection interval in HAWK and HARRIER respectively with
remaining patients on q8w regimen (key secondary endpoints); greater than 75% of these patients continued on q12w dosing up to Week 96 6. Met primary efficacy endpoint of
noninferiority to aflibercept in mean change in BCVA with comparable safety to aflibercept; vision gains comparable to aflibercept up to Week 96
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 2018 presentation
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 60
New additions
Trials taken out (operational decision-points achieved)
Study Program Indication Phase Patients
NCT02152761 (CBYM338D2201) BYM338 Hip fracture recovery Phase 2B 245
NCT02333331 InvestiGAIT (CBYM338E2202) BYM338 Sarcopenia Phase 2B 280
NCT01327846 CANTOS (CACZ885M2301) ACZ885 Cardiovascular risk reduction Phase 3 10,061
Study Program Indication Phase Patients
NCT03578367 (CABL001E2201) ABL001 Chronic myeloid leukaemia (CML) Phase 2 120
NCT03663335 (CFZ533A2201) CFZ533 Kidney transplantation Phase 2 325
NCT03701334 NATALEE (CLEE011012301C) Kisqali® Early breast cancer Phase 3 4,000
NCT03568461 ELARA (CCTL019E2202) Kymriah® Relapsed / refractory follicular lymphoma (FL) Phase 2 113
NCT03580369 PEARL 1 (CQGE031C2302) QGE031 Chronic spontaneous urticaria Phase 3 1,050
NCT03580356 PEARL 2 (CQGE031C2303) QGE031 Chronic spontaneous urticaria Phase 3 1,050
NCT03517566 ZEST (CZPL389A2203) ZPL389 Atopic dermatitis Phase 2 360
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
62
Study NCT02678312 PANORAMA HF (CLCZ696B2319) NCT02661217 TRANSITION (CLCZ696B2401)
Indication Cardiac failure congestive Cardiac failure congestive
Phase Phase 2/3 Phase 4
Patients 360 1,002
Primary Outcome
Measures
Part 1: Pharmacodynamics and pharmacokinetics of
LCZ696 analytes
Part 2: Efficacy and safety compared with enalapril
Assessing the percentage of patients who achieve the target
dose of 200 mg bid LCZ696 at 10 weeks after
randomization
Arms/Intervention
• Part 1: LCZ696 0.8 mg/kg or 3.1 mg/kg or both
• Part 2: enalapril 0.2 mg/kg bid; LCZ696: 3.125 mg
granules and adult formulation (50, 100, 200 mg bid)
• Pre-discharge treatment initiation - LCZ696 (50, 100,
200 mg bid)
• Post-discharge treatment initiation - LCZ696 (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
Heart failure patients with reduced ejection-fraction
hospitalized for an acute decompensation event
Expected Completion 2021 Q4-2018 (actual)
Publication TBD
• IA presented at ESC-2018;
• 10-wk data submitted EHJ (not yet accepted);
• 26-wk data presentation planned for ESC-HF May-2019
with goal of simultaneous publication if possible (not yet
accepted)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
63
Study NCT02884206 PERSPECTIVE (CLCZ696B2320) NCT02468232 PARALLEL-HF (CLCZ696B1301)
Indication Cardiac failure congestive Cardiac failure congestive
Phase Phase 3 Phase 3
Patients 520 225
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
• LCZ696 50, 100, and 200 mg bid with placebo of
valsartan
• Valsartan 40, 80, and 160 mg bid tablets with placebo
for LCZ696
• LCZ696 50 mg, 100 mg, 200 mg bid/placebo of enalapril
• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of LCZ696
Target PatientsPatients with chronic heart failure with preserved ejection
fraction
Japanese heart failure patients (NYHA Class II-IV) with
reduced ejection fraction
Expected Completion 2022Q4-2019 (primary); 2020 (open-label extension)
Publication TBD TBD
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
64
Study NCT02554890 PIONEER-HF (CLCZ696BUS01) NCT02924727 PARADISE-MI (CLCZ696G2301)
Indication Cardiac failure congestive Myocardial infarction
Phase Phase 3B/4 Phase 3
Patients 887 4,650
Primary Outcome
Measures
Percentage change from baseline in N-terminal pro-brain
natriuretic peptide (NT-proBNP)
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 (LCZ696)
• Sacubitril/valsartan (LCZ696) matching placebo
• Enalapril
• Enalapril matching placebo
• LCZ696 50 mg, 100 mg, 200 mg bid / placebo of
ramipril/valsartan
• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of
LCZ696 / placebo for valsartan
Target PatientsPatients with HFrEF (LVEF<40%) hospitalized for ADHF
and stable for more than 24 hours
Post-AMI patients with evidence of LV systolic dysfunction
and/or pulmonary congestion, with no known prior history of
chronic HF
Expected Completion Q3-2018 (actual) H2-2020
Publication
• Presentation planned AHA Nov-2018;
• Publication submitted to NEJM Q4-2018 (not yet
accepted)
TBD
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
65
Study NCT01920711 PARAGON (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)
Indication Cardiac failure chronic Cardiac failure chronic
Phase Phase 3 Phase 3
Patients 4,800 2,200
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
Arms/Intervention• LCZ696 50 mg, 100 mg and 200 mg
• Valsartan 40 mg, 80 mg and 160 mg
• LCZ696 50 mg, 100 mg and 200 mg bid
• Enalapril 2.5 mg, 5 mg and 10 mg bid
• Valsartan 40 mg, 80 mg, 160 mg bid
• Placebo to match LCZ696 sacubitril/valsartan
• Placebo to match enalapril
• Placebo to match valsartan
Target PatientsHeart failure patients (NYHA Class II-IV) with preserved
ejection fraction
Heart failure patients (NYHA Class II-IV) with preserved
ejection fraction
Expected Completion Q3-2019 H1-2020
Publication TBD TBD
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
CFZ533 – Blocking, non-depleting, Fc-silent, anti-CD40 monoclonal antibody
Study NCT03663335 (CFZ533A2201)
Indication Renal transplant
Phase Phase 2b
Patients 325
Primary Outcome
Measures
Composite event (BPAR, Graft Loss or Death)
over 12 months post-transplantation and post
conversion (for maintenance cohort)
Arms/Intervention
Target PatientsDe novo and maintenance kidney transplant
recipients
Expected
Completion2021
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 67
Cosentyx® - Anti IL-17
68
Study NCT01544595 (CAIN457A2302E1 – extension study)NCT01640951 SCULPTURE (CAIN457A2304E1 –
extension study)
Indication Psoriasis Psoriasis
Phase Phase 3 Phase 3
Patients 1,146 675
Primary Outcome
Measures
Cumulative rate of subjects with loss of psoriasis area and
Cumulative rate of subjects with loss of Psoriasis Area and
Severity Index (PASI) 75 response up to week 68 (time = 0
being defined as week 52)
The number and percentage of subjects having any adverse
event
Arms/Intervention
• Secukinumab 150 mg
• Secukinumab 300 mg
• Placebo
• Fixed-time interval regimen secukinumab 150 mg
• Retreatment at start of relapse secukinumab 150 mg
• Fixed-time interval regimen secukinumab 300 mg
• Retreatment at start of relapse secukinumab 300 mg
• Open label secukinumab 300 mg
Target Patients
Patients with moderate to severe chronic plaque-type
psoriasis completing preceding psoriasis phase III studies
with secukinumab
Patients with moderate to severe chronic plaque-type
psoriasis
Expected Completion 2017 (actual) 2017 (actual)
Publication• 2-years results: Br J Dermatol. 2017 May 12. doi:
10.1111/bjd.15656
• 3-years results: Br J Dermatol; 5 June 2017. doi:
10.1111/bjd.15706
• 5-years results: Submitted to JEADV; 14 February 2018
doi: 10.1111/jdv.14878
• 5-years presented at EAD Sept 2017 (late-breaker)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
69
Study NCT02471144 (CAIN457A2310) NCT03066609 (CAIN457A2318)
Indication Psoriasis Psoriasis
Phase Phase 3 Phase 3
Patients 169 543
Primary Outcome
Measures
The percentage of Participants achieving a 75%
Improvement from Baseline in PASI Score at week 12
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
Arms/Intervention
• Secukinumab low dose
• Secukinumab high dose
• Placebo
• Etanercept (comparator)
• Secukinumab 300 mg
• Secukinumab 150 mg
• Placebo
Target PatientsPatients from 6 to less than 18 years of age with severe
chronic plaque
Patients with moderate to severe chronic plaque-type
psoriasis with or without psoriatic arthritis comorbidity
Expected Completion 2023 Q1-2019
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
70
Study NCT02826603 CLARITY (CAIN457A2326) NCT03668613 (CAIN457A2311)
Indication Psoriasis Psoriasis
Phase Phase 3B Phase 3
Patients 1,102 80
Primary Outcome
Measures
Psoriasis Area and Severity Index (PASI) 90 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
Arms/Intervention• Secukinumab 300 mg
• Ustekinumab 45 mg/ 90 mg
• Secukinumab low dose
• Secukinumab high dose
Target Patients Patients with moderate to severe plaque psoriasisPediatric patients of age 6 to <18 years, with moderate to
severe plaque psoriasis
Expected Completion Q3-2018 (actual) 2023
Publication
• Abstract Winter Clin Derm (US) Jan-2018
• Abstract to EADV in 2018
• Submission Journal (16wk 1ry EP IA) Q3-2018
(ongoing)
• Encore Abstract AAD 2019
TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
71
Study NCT02748863 ALLURE (CAIN457A2323) NCT02745080 EXCEED (CAIN457F2366)
Indication Psoriasis Psoriatic arthropathy
Phase Phase 3 Phase 3
Patients 214 850
Primary Outcome
Measures
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 responseAmerican College of Rheumatology 20 (ACR20) response
Arms/Intervention
• Secukinumab 300 mg (2 mL PFS device)
• Secukinumab 300 mg (2 x 1 mL PFS device)
• Placebo
• Secukinumab 300 mg s.c.
• Adalimumab 40 mg s.c.
Target Patients Adult subjects with moderate to severe plaque psoriasis Patients with active psoriatic arthritis
Expected Completion Q3-2018 (actual) H1-2020
Publication• Submission Journal TBC Q2-2019
• Abstract at AAD in 2019TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
72
Study NCT03031782 (CAIN457F2304) NCT01863732 (CAIN457F2305E1 – extension study)
Indication Psoriatic arthropathy Ankylosing spondylitis
Phase Phase 3 Phase 3
Patients 80 300
Primary Outcome
MeasuresTime to flare in Part 2
Assessment of spondyloarthritis international society criteria
/ ASAS 20 response
Arms/Intervention• Secukinumab (pre-filled syringe) 75 mg
• Placebo
• Secukinumab 75 mg in PFS
• Secukinumab 150 mg in PFS
Target PatientsJuvenile Idiopathic Arthritis subtypes of Psoriatic and
Enthesitis-related ArthritisPatients with active ankylosing spondylitis
Expected Completion 2021 Q2-2018 (actual)
Publication TBD
• 3-year results: Manuscript published in Clinical and
Experimental Rheumatology in May-2017
• 4-year results: Presented at ACR in Nov-2017
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
73
Study NCT01892436 FUTURE 1 extension (CAIN457F2306E1) NCT01649375 MEASURE 2 (CAIN457F2310)
Indication Psoriatic arthropathy Ankylosing spondylitis
Phase Phase 3 Phase 3
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
• Secukinumab 150 mg
• Secukinumab 75 mg
• Secukinumab 150 mg
• Placebo
Target Patients Patients with active psoriatic arthritis Patients with active ankylosing spondylitis
Expected Completion Q1-2018 (actual) Q4-2018 (actual)
Publication
• 3 year results: ACR 2016; Mease PJ et al. Arthritis
Rheumatol. 2016; 68 (suppl 10)
• 3 years results: Manuscript submitted in Q4-2017
• Primary 52 week results: Baeten D & Sieper J, et al. N
Engl J Med 2015;373:2534–48
• 2 year results: Marzo-Ortega, et al. Arthritis Care Res
2017 Feb 24. doi: - 10.1002/acr.23233
• 3 year results: Marzo-Ortega, et al. RMD 2017
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
74
Study NCT01752634 FUTURE 2 (CAIN457F2312) NCT02008916 MEASURE 3 (CAIN457F2314)
Indication Psoriatic arthropathy Ankylosing spondylitis
Phase Phase 3 Phase 3
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.
• Secukinumab (AIN457) 75 mg s.c.
• Secukinumab (AIN457) 300 mg s.c.
• Placebo s.c.
• Secukinumab 10 mg/kg / 300 mg
• Secukinumab 10 mg/kg / 150 mg
• Placebo
Target Patients Patients with active psoriatic arthritis Patients with active ankylosing spondylitis
Expected Completion Q1-2019 Q1-2018 (actual)
Publication
• Primary results: McInnes IB, et al. Lancet.
2015;386:1137–46
• 2 years results: McInnes et al, Rheumatology
2017;56:1993-2003
• 3 year results: Abstract to be submitted to EULAR
congress in Jun-2018
• 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
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
75
Study NCT01989468 FUTURE 3 (CAIN457F2318) NCT02159053 MEASURE 4 (CAIN457F2320)
Indication Psoriatic arthropathy Ankylosing spondylitis
Phase Phase 3 Phase 3
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
• Secukinumab (AIN457) 150 mg s.c.
• Secukinumab (AIN457) 300 mg s.c.
• Placebo
• Secukinumab 150 mg s.c. with loading
• Secukinumab 150 mg s.c. without loading
• Placebo
Target Patients Patients with active psoriatic arthritis Patients with active ankylosing spondylitis
Expected Completion Q2-2018 (actual) Q2-2018 (actual)
Publication52 week results: Nash et al, Arthritis Research & Therapy
2018, 20:4752 week results: manuscript to be submitted in Q1-2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
76
Study NCT02294227 FUTURE 4 (CAIN457F2336) NCT02404350 FUTURE 5 (CAIN457F2342)
Indication Psoriatic arthropathy Psoriatic arthropathy
Phase Phase 3 Phase 3
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
• Secukinumab 300 mg load
• Placebo
Target Patients Patients with active psoriatic arthritis Patients with active psoriatic arthritis
Expected Completion Q1-2018 (actual) Q2-2019
Publication
• 52 week results: abstract to be presented at PANLAR
congress (Apr-2018)
• 2 year results: manuscript to be submitted in Q3-2018
• 24 week results late breaker presented in ACR in Nov-
2017
• 24 week data; manuscript submitted to Annals of
Rheumatic Disease in Nov 2017
• 52 week data; to be presented at ACR 2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
77
Study NCT02696031 PREVENT (CAIN457H2315) NCT03259074 SURPASS (CAIN457K2340)
Indication Non-radiographic axial spondyloarthritis Ankylosing spondylitis
Phase Phase 3 Phase 3
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 load
• 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 2021 2022
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Ilaris® - Anti IL-1β
78
Study NCT02059291 CLUSTER (CACZ885N2301) NCT02296424 (CACZ885G2306)
Indication Autoimmune disorder Juvenile idiopathic arthritis
Phase Phase 3 Phase 3B/4
Patients 203 182
Primary Outcome
Measures
To demonstrate significant reduction of disease activity
with canakinumab vs. placebo
Proportion of patients in clinical remission on canakinumab
who are able to remain at an initial reduced canakinumab dose
or prolonged canakinumab dose interval
Arms/Intervention• Canakinumab
• Placebo
• Canakinumab dose reduction
• Canakinumab dose interval prolongation
Target PatientsPatients with, 3 separate disease cohorts TRAPS, HIDS,
and colchicine resistant FMF (Hereditary periodic fevers )
Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)
(Pediatric)
Expected Completion 2017 (actual) 2017 (actual)
Publication
• Safety & efficacy (w16+40) in NEJM in May 2018
(May 17, 2018: N Engl J Med 2018; 378:1908-1919)
• Disease specific publications on TRAPS, FMF and
HIDS in 2019
• Additional manuscripts in 2019
Manuscript to be submitted in Q2-2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
LJN452 - FXR Agonist
79
Study NCT02855164 (CLJN452A2202)
Indication Non-alcoholic steatohepatitis
Phase Phase 2
Patients 345
Primary Outcome
Measures
Adverse event profile of different doses; determine the dose
relationship of LJN452 on markers of hepatic inflammation
in NASH (ALT and AST); determine dose-response
relationship of LJN452 on liver fat content by changes in
quantitative MRI; determine effect of LJN452 on liver fibrosis
by biopsy
Arms/Intervention Multiple LJN452 doses and placebo
Target Patients Patients with non-alcoholic steatohepatitis (NASH)
Expected Completion 2020
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
LJC242 - FXR agonist + CCR2/CCR5 inhibitor
80
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 2020
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)
Indication Urticaria Urticaria
Phase Phase 2B Phase 2B
Patients 382 226
Primary Outcome
Measures
Establish dose-response relationship of QGE031 with
respect to achievement of complete hives response at week
12
Long-term safety; number of participants with treatment-
emergent adverse events
Arms/Intervention
• Ligelizumab 24mg q4wks
• Ligelizumab 72mg q4wks
• Ligelizumab 240mg q4wks
• Ligelizumab 120mg single dose
• Omalizumab 300mg q4wks
• Placebo q 4wks
Ligelizumab 240 mg q4wks open label
Target Patients Patients with chronic spontaneous urticaria Patients with chronic spontaneous urticaria
Expected Completion 2017 (actual) Q2-2019
PublicationPrimary results: Presented at EAACI 2018, EADV 2018, and
GUF 2018; manuscript expected in Q1-2019
Primary results: Late breaker abstract (showing 1 year
treatment results) submitted for AAD in Q1-2019; manuscript
submission expected in Q4-2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 81
QGE031 - Anti-IgE
Study NCT03437278 (CQGE031C2202)
Indication Urticaria
Phase Phase 2
Patients 48
Primary Outcome
MeasuresChange in the 7 day Urticaria Activity Score (UAS7)
Arms/Intervention
• Ligelizumab high dose q4wks
• Ligelizumab low dose q4wks
• Placebo / ligelizumab high dose q4wks
Target PatientsAdolescents from 12 to <18 years of age, with chronic
spontaneous urticaria
Expected Completion H2-2020
Publication Manuscript to be submitted in 2021
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 82
QGE031 - Anti-IgE
Study NCT03580369 Pearl 1 (CQGE031C2302) NCT03580356 Pearl 2 (CQGE031C2303)
Indication Urticaria Urticaria
Phase Phase 3 Phase 3
Patients 1,050 1,050
Primary Outcome
Measures
Absolute change from baseline in UAS7 (Urticaria Activity
Score) at week 12
Absolute change from baseline in UAS7 (Urticaria Activity
Score) at week 12
Arms/Intervention
• Ligelizumab dose A q4w
• Ligelizumab dose B q4w
• Omalizumab 300 mg q4w
• Placebo q4w from randomization to wk20, then
ligelizumab dose B from wk24 to wk48
• Ligelizumab dose A q4w
• Ligelizumab dose B q4w
• Omalizumab 300 mg q4w
• Placebo q4w from randomization to wk20, then
ligelizumab dose B from wk24 to wk48
Target PatientsAdolescents and adults with chronic spontaneous urticaria
and inadequately controlled with H1-antihistamines
Adolescents and adults with chronic spontaneous urticaria
and inadequately controlled with H1-antihistamines
Expected Completion 2021 2021
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 83
VAY736 – Fully human IgG1/κ anti-BAFF-R mAb
Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)
Indication Sjoegren's syndrome Autoimmune hepatitis
Phase Phase 2B Phase 2
Patients 180 80
Primary Outcome
Measures
Safety and efficacy of VAY736 in primary Sjoegren'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 Sjoegren's
Syndrome (pSS)
Autoimmune hepatitis patients with incomplete response or
intolerant to standard treatment of care
Expected Completion H2-2020 2023
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 84
ZPL389 - H4 receptor antagonists
Study NCT03517566 ZEST (CZPL389A2203)
Indication Atopic dermatitis
Phase Phase 2
Patients 360
Primary Outcome
MeasuresIGA (Investigator's global assessment) response at week 16
Arms/Intervention
• ZPL389 dose 1
• ZPL389 dose 2
• ZPL389 dose 3
• ZPL389 dose 4
• Placebo
Target Patients Patients with moderate to severe atopic dermatitis
Expected Completion H1-2020
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 85
Aimovig® – CGRP receptor antagonist
87
Study NCT03096834 LIBERTY (CAMG334A2301) NCT03333109 EMPOWER (CAMG334A2302)
Indication Migraine Migraine
Phase Phase 3 Phase 3
Patients 246 880
Primary Outcome
Measures
Percentage of patients with a 50% response in the reduction
of Monthly Migraine Days (MMD)
Change from baseline in monthly migraine days at the last
month (Month 3) of the double-blind treatment period
Arms/Intervention• Subcutaneous injection of AMG334 (erenumab)
• Subcutaneous injection of placebo
• AMG334 (erenumab) Dose 1
• AMG334 (erenumab) Dose 2
• Placebo
Target PatientsAdult episodic migraine patients who have failed prophylactic
migraine treatmentsAdult episodic migraine patients
Expected Completion 2017 DBT phase (actual); 2021 OLE phase H1-2020
Publication Planned in 2019 (more details will follow) TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
CNP520 - BACE inhibitorCAD106 - active beta-amyloid immunotherapy
88
Study NCT02565511 GENERATION S1 (CAPI015A2201J) NCT03131453 GENERATION S2 (CCNP520A2202J)
Indication Alzheimer’s disease Alzheimer’s disease
Phase Phase 2B/3 Phase 2B/3
Patients 1,340 2,000
Primary Outcome
Measures
Time to diagnosis of MCI due to Alzheimer's disease or
dementia due to Alzheimer's disease
Change in the Alzheimer's Prevention Initiative Composite
Cognitive (APCC) Test Score
Time to diagnosis of MCI due to Alzheimer's disease or
dementia due to Alzheimer's disease
Change in the Alzheimer's Prevention Initiative Composite
Cognitive (APCC) Test Score
Arms/Intervention
• CAD106 450 µg + Alum 450 µg i.m.
• Placebo to CAD106 + Alum 450 µg i.m.
• CNP520 50 mg oral
• Placebo to CNP520 oral
• CNP520 15 mg oral
• CNP520 50 mg oral
• Placebo to CNP520 oral
Target PatientsCognitively unimpaired participants aged 60 to 75 years,
with two APOE4 allele (Homozygotes )
Cognitively unimpaired participants aged 60 to 75 years,
with at least one APOE4 allele (Homozygotes or
Heterozygotes) and, if Heterozygotes, with evidence of
elevated brain amyloid
Expected Completion 2024 2025
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
BAF312 - S1P-R modulator
89
Study NCT01665144 -EXPAND (CBAF312A2304)
Indication Secondary progressive multiple sclerosis
Phase Phase 3
Patients 1,620
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
Publication• Presentations at ECTRIMS and AAN 2017
• Lancet March 2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
EMA401 - Angiotensin II type 2 receptor antagonist
90
Study NCT03094195 EMPHENE (CEMA401A2201) NCT03297294 EMPADINE (CEMA401A2202)
Indication Peripheral neuropathic pain Peripheral neuropathic pain
Phase Phase 2 Phase 2
Patients 360 400
Primary Outcome
Measures
Dose-response in change in weekly mean of the 24-hour
average pain score from Baseline to week 12
Change in weekly mean 24-hour average pain score
from Baseline to Week 12
Arms/Intervention• 3 doses EMA401
• Placebo
• 1 doses EMA401
• Placebo
Target Patients Post-herpetic neuralgia patients Painful diabetic neuropathy
Expected Completion H2-2020 H1-2020
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Gilenya® - S1P-R modulator
91
Study NCT01892722 PARADIGMS (CFTY720D2311) NCT01201356 (CFTY720D2399)
Indication Pediatric multiple sclerosis Relapsing multiple sclerosis (RMS)
Phase Phase 3B Phase 3B/4
Patients 215 4,125
Primary Outcome
Measures
Frequency of relapses in patients treated for up to 24
months (using ARR)Long-term safety and tolerability
Arms/Intervention• Interferon beta-1a i.m.
• Fingolimod 0.5 mg/ 0.25 mgSingle-arm study of fingolimod 0.5 mg/day
Target PatientsPediatric patients with multiple sclerosis with five-year
fingolimod extension phasePatients with relapsing multiple sclerosis
Expected Completion Q3-2017 (core phase) / 2023 (extension phase) Q4-2018 (actual)
Publication
• Primary data presentation: Chitnis T, et al. Presented at
ECTRIMS 2017 (Late Breaker)
• Chitnis T, Arnold DL, Banwell B, et al. Trial of Fingolimod
versus Interferon Beta-1a in Pediatric Multiple Sclerosis..
N Engl J Med. 2018; 379: 1017-1027.
• Primary data presentation: Cohen J, et al presented at
ECTRIMS 2017
• Primary manuscript: TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Gilenya® - S1P-R modulator
92
Study NCT01633112 ASSESS (CFTY720D2312)
Indication Relapsing remitting multiple sclerosis (RRMS)
Phase Phase 3B
Patients 1,064
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
Arms/Intervention
• Fingolimod 0.5 mg orally
• Fingolimod 0.25mg orally
• Copaxone® 20 mg s.c.
Target Patients Patients with relapsing-remitting multiple sclerosis
Expected Completion Q3-2018 (actual)
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
LMI070 - SMN2 RNA splice modulator
93
Study NCT02268552 (CLMI070X2201)
Indication Type 1 spinal muscular atrophy
Phase Phase 1/2
Patients 44
Primary Outcome
Measures
Number of participants with adverse events (AEs), serious
adverse events (SAEs) and deaths
Arms/Intervention • Branaplam oral, once weekly, 3 ascending doses
Target PatientsPatients with type 1 spinal muscular atrophy
Expected Completion Q3-2020
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
OMB157 - Anti-CD20
94
Study NCT02792218 Asclepios I (COMB157G2301) NCT02792231 Asclepios II (COMB157G2302)
Indication Multiple sclerosis Multiple sclerosis
Phase Phase 3 Phase 3
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
Expected Completion Q3-2019 Q3-2019
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
OMB157 - Anti-CD20
95
Study NCT03249714 SAKURA (COMB157G1301)
Indication Multiple sclerosis
Phase Phase 2
Patients 60
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)
Arms/Intervention• Ofatumumab 20 mg subcutaneous injections
• Placebo
Target Patients Patients with relapsing forms of multiple sclerosis
Expected Completion H1-2020
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor
97
Study NCT03106779 (CABL001A2301) NCT03578367 (CABL001E2201)
Indication Chronic myeloid leukaemia (CML) Chronic myeloid leukaemia (CML)
Phase Phase 3 Phase 2
Patients 222 120
Primary Outcome
MeasuresMajor Molecular Response (MMR) rate at 24 weeks Deep molecular response (MR 4.5) at 48 weeks
Arms/Intervention• ABL001 40 mg
• Bosutinib 500 mg
• ABL001 40 mg QD + 400 mg imatinib
• ABL001 60 mg QD + 400 mg imatinib
• Imatinib 400mg QD (continuation treatment)
• Nilotinib 300mg BID (switch to nilotinib treatment)
Target Patients
Patients with chronic myelogenous leukemia in chronic
phase, previously treated with 2 or more tyrosine kinase
inhibitors
CML-CP patients not reaching DMR (MR 4.5) while on 1L
imatinib treatment
Expected Completion H2-2020 H1-2021
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
ACZ885 – IL1β inhibitor
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 98
Study NCT03447769 (CACZ885T2301)
Indication Adjuvant NSCLC
Phase Phase 3
Patients 1,500
Primary Outcome
Measures
Disease free survival (primary), overall survival (key
secondary)
Arms/Intervention• Canakinumab 200mg q3w sc for 18 cycles
• Placebo q3w sc for 18 cycles
Target Patients
Patients with:
• High–risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB
(T>5cm N2)) after complete resection
• All histologies
• With/without EGFR mutation
Expected Completion 2022
Publication • TBD
BYL719 - Alpha-specific PI3K inhibitor
99
Study NCT02437318 SOLAR-1 (CBYL719C2301)
Indication HR + aBC
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
Expected Completion Q3-2018 (actual)
PublicationAndre F, et al. Presentation at ESMO 2018
Manuscript submitted Q4-2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Kymriah® – CAR-T therapy
100
Study NCT02445248 JULIET (CCTL019C2201) NCT02435849 ELIANA (CCTL019B2202)
Indication Relapsed / refractory DLBCL Pediatric and young adult Relapsed/ refractory ALL
Phase Phase 2 Phase 2
Patients 128 95
Primary Outcome
MeasuresOverall response rate; efficacy and safety of CTL019
Overall remission rate (ORR) - overall remission rate during
the 6 months after CTL019 administration, which includes
CR and CR with incomplete blood count recovery (CRi) as
determined by IRC assessment
Arms/Intervention Single-arm study of single dose of CTL019 Single-arm study of single dose of CTL019
Target PatientsAdult patients with relapsed or refractory diffuse large B-cell
lymphoma (DLBCL)
Pediatric and young adult patients with relapsed and
refractory B-cell acute lymphoblastic leukemia
Expected Completion 2017 (actual) 2016 (actual)
Publication
• Schuster et al. at ICML 2017; Schuster et al. at EHA
2017; Schuster et al. at ASH 2017
• Borchmann et al. at EHA 2018
• Schuster et al. N Engl J Med. 2018; epub ahead of print.
DOI: 10.1056/NEJMoa1804980
• Grupp et al. at ASH 2016
• Buchner et al at EHA 2017
• Maude et al. N Engl J Med. 2018;378:439-48. DOI:
10.1056/NEJMoa1709866
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Kymriah® – CAR-T therapy
101
Study NCT03568461 ELARA (CCTL019E2202)
Indication Relapsed / refractory follicular lymphoma (FL)
Phase Phase 2
Patients 113
Primary Outcome
MeasuresComplete response rate (CRR)
Arms/Intervention Single-arm study of tisagenlecleucel
Target Patients Adult patients with relapsed or refractory FL
Expected Completion H2-2020
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Exjade® - Iron chelation of bis-hydroxy-phenyl triazole type
102
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
Expected Completion Q3-2018 (actual)
PublicationAngelucci E, et al. Presentation at ASH 2018
Manuscript submitted Q4 2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
INC280 - MET Inhibitor
103
Study NCT02414139 (CINC280A2201) NCT02335944 (CINC280X2105C)
IndicationEGFR Wild-type, ALK negative advanced Non-small Cell Lung
Cancer (NSCLC)
Non-small Cell Lung Cancer (NSCLC) Patients With EGFR
Mutation
Phase Phase 2 Phase 1/2
Patients 348 177
Primary Outcome
MeasuresOverall Response Rate (ORR)
Phase II Groups 1, 2 and 3: Overall Response Rate (ORR)
Phase II Group 4: Frequency of treatment-emergent adverse
events
Arms/Intervention
• Pre-treated pts. with MET GCN ≥ 6
• Pre-treated pts. with MET GCN ≥ 4 and < 6
• Pre-treated pts. with MET GCN < 4
• Pre-treated pts. with MET mutations regardless of cMET
GCN
• Treatment-naïve pts. with MET dysregulation
• Pre-treated pts with MET dysregulation – second line
• Group 1: EGFRmut NSCLC developing resistance to
EGFR TKI
• Group 2: EGFR TKI-naïve, EGFRmut NSCLC with de novo
T790M mutation
• Group 3: Treatment-naïve, EGFRmut NSCLC
• Group 4: 1-3L EGFRmut NSCLC (with food)
Target Patients
Adult patients with EGFR wild-type (wt), ALK-negative
advanced non-small cell lung cancer (NSCLC) with either
MET amplification or MET mutations and are either
pretreated with 1 or 2 prior lines of systemic therapy or are
treatment-naïve for the advanced stage of disease
Adult Patients With EGFR Mutated Non-small Cell Lung
Cancer
Expected Completion Q2-2019 Q4-2018 (actual)
Publication• Wolf et al. Presentation at ESMO 2018
• Manuscript submission Q2-2019Abstract submission Q2 2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Jakavi® - JAK1/2 inhibitor
104
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)
Phase Phase 3 Phase 3
Patients 308 324
Primary Outcome
MeasuresOverall Response Rate (ORR) at 28 Days Overall Response Rate (ORR) at 183 Days
Arms/Intervention• Ruxolitinib 10mg BID
• Best available therapy (BAT)
• Ruxolitinib 10mg BID
• Best available therapy (BAT)
Target Patients Patients with Steroid-refractory Acute GVHD (SR aGVHD) Patients with steroid-refractory chronic GVHD (SR cGVHD)
Expected Completion Q3-2019 Q4-2019
PublicationManuscript Submission Q4-19
Congress presentation Q4 2019 TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Kisqali® - CDK 4/6 inhibitor
105
Study NCT02422615 MONALEESA-3 (CLEE011F2301) NCT02278120 MONALEESA-7 (CLEE011E2301)
Indication Advanced breast cancer – 1st / 2nd line (with fulvestrant) Advanced breast cancer - 1st line (pre-menopausal)
Phase Phase 3 Phase 3
Patients 727 672
Primary Outcome
Measures
Progression Free Survival (PFS) - time from the date of
randomization to the date of the first documented
progression or death due to any cause
Progression Free Survival (PFS) - time from the date of
randomization to the date of the first documented
progression or death due to any cause and assessed
according to RECIST 1.1
Arms/Intervention• Riblociclib 600mg + fulvestrant 500mg
• Placebo of Riblociclib + fulvestrant 500mg
• LEE011 600 mg + NSAI/tamoxifen + goserelin 3.6 mg
• Placebo of LEE011 + NSAI/tamoxifen + goserelin 3.6 mg
Target Patients
Postmenopausal women with hormone receptor positive,
HER2-negative, advanced breast cancer who have received
no or only one line of prior endocrine treatment
Premenopausal women with hormone receptor positive,
HER2-negative, advanced breast cancer
Expected Completion Q1-2018 (actual) 2017 (actual)
Publication
• Slamon D, et al. Oral presentation at ASCO 2018
• Slamon D, et al. J Clin Oncol 36:2465-2472;
DOI:https://doi.org/10.1200/JCO.2018.
• Tripathy D, et al. Oral presentation at SABCS 2017
• Tripathy D, et al. Lancet Oncol 2018; 19: 904–15;
DOI:https://doi.org/10.1016/S1470-2045(18)30292-4
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Kisqali® - CDK 4/6 inhibitor
106
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 2025
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
LCI699 - Cortisol synthesis inhibitor
107
Study NCT02697734 LINC-4 (CLCI699C2302) NCT02180217 LINC-3 (CLCI699C2301)
Indication Cushing's disease Cushing's disease
Phase Phase 3 Phase 3
Patients 69 132
Primary Outcome
Measures
Demonstrate the superiority of osilodrostat compared to
placebo in achieving a complete response mean urine free
cortisol ≤ upper limit of normal (mUFC ≤ ULN) at Week 12
Compare the complete response rate at the end of the 8-
week randomized withdrawal period
Arms/Intervention• LCI699 / osilodrostat
• Placebo
Randomized withdrawal design
• LCI699 / osilodrostat
• Placebo
Target Patients Patients with Cushing's disease Patients with Cushing's disease
Expected Completion H2-2020 Q2-2018 (actual)
Publication TBD Pivonello et al. presented at ICE 2018
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
PDR001 – PD-1 checkpoint inhibitor
108
Study NCT02967692 COMBI-i (CPDR001F2301)
Indication BRAFV600 mutant metastatic melanoma
Phase Phase 3
Patients
538
Part 1 (safety-run in): 9; Part 2 (biomarker cohort): 27; Part 3
(Phase III, randomized, placebo controlled): 532
Primary Outcome
MeasuresProgression-Free Survival (PFS)
Arms/Intervention
• Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg BID +
Mekinist 2 mg
• Placebo + Tafinlar 150 mg BID + Mekinist 2 mg
Target Patients
Previously untreated patients with unresectable or
metastatic BRAF V600 mutant melanoma
Expected Completion H2-2019
PublicationCongress presentation and manuscript submission planned
H2-2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Rydapt®- Multi-targeted kinase inhibitor
109
Study NCT00651261 RATIFY (CPKC412A2301) NCT03280030 (CPKC412A2220)
Indication Acute myeloid leukemia Acute myeloid leukemia
Phase Phase 3 Phase 2
Patients 717 66
Primary Outcome
MeasuresOverall survival Incidence of safety events and event free survival
Arms/Intervention
• Induction and consolidation chemotherapy plus
midostaurin
• Induction and consolidation chemotherapy plus placebo
• Midostaurin 50 mg
• Placebo
Target PatientsNewly diagnosed patients < 60 years of age with FLT3
mutated acute myeloid leukemia (AML)
Newly diagnosed patients with FLT3-mutated acute myeloid
leukemia (AML)
Expected Completion 2016 (actual) H1-2020
Publication
• Stone RM, Manley PW, Larson RA, and Capdeville R.
published February 27, 2018 in Blood Advances
2018;(2:444-453
• H. Gu Drug Metab Dispos. 2018;46(2):109-121
• Planned: Karin Hartman, Haneke Kluin-Nelemans
Journal of Allergy and Clinical Immunology (TBD)
• Planned: Combine into single paper (maintenance and
CIR): Leukemia
TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Rydapt®- Multi-targeted kinase inhibitor
110
Study NCT03512197 (CPKC412E2301)
Indication Acute myeloid leukemia
Phase Phase 3
Patients 502
Primary Outcome
MeasuresEvent free survival
Arms/Intervention• Midostaurin 50 mg
• Placebo
Target PatientsNewly diagnosed patients with FLT3 mutation negative
acute myeloid leukemia (AML)
Expected Completion 2021
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Promacta®/Revolade® – Thrombopoetin receptor agonist
Study NCT03025698 (CETB115E2201)
IndicationPreviously untreated or relapsed/refractory severe aplastic anemia or
recurrent aplastic anemia
Phase Phase 2
Patients 60
Primary Outcome
MeasuresPK of eltrombopag at steady state in pediatric patients with SAA
Arms/Intervention
- Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS
- Arm B: previously untreated SAA-hATG/cyclosporine +
eltrombopag
- Arm A: relapsed/refractory SAA or AA: hATG/cyclosporine +
eltrombopag or cyclosporine + eltrombopag
Target PatientsPediatric patients from age 1 <18 years with relapsed/refractory SAA
or recurrent AA after IST or previously untreated SAA
Expected Completion 2024
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 111
SEG101 – p-Selectin inhibitor
Study NCT03264989 (CSEG101A2202) NCT03474965 (CSEG101B2201)
IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with
Sickle Cell Disease (SCD) Prevention of VOC in pediatric patients with SCD
Phase Phase 2 Phase 2
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)H2-2020 (pediatric patients ≥6 year old)
2021 (pediatric patients 6 months – 6 year old)
Publication Abstract submission Q1-2019 TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 112
Tafinlar®+Mekinist® - BRAFV600 inhibitor and MEK inhibitor
Study NCT01682083 COMBI-AD (CDRB436F2301) NCT02124772 (CTMT212X2101)
Indication BRAFV600 mutant adjuvant melanoma BRAFV600 mutant solid tumors
Phase Phase 3A Phase 1
Patients 874 142
Primary Outcome
MeasuresRelapse-free survival (RFS) Safety, tolerability and pharmacokinetics and clinical activity
Arms/Intervention• Dabrafenib 150 mg BID + trametinib 2 mg
• Placebo
Trametinib (dose based on age and weight)
Dabrafenib + trametinib (dose based on age and weight)
Target PatientsSubjects with BRAFV600 mutation-positive melanoma with
lymph node(s) involvement, after complete resection
Pediatric Subjects Aged 1 Month to <18 Years with
Advanced V600-Mutation Positive Solid Tumors
Expected Completion Q3-2017 (actual) H1-2020
PublicationLong G.V., et al. N Engl J Med 2017; 377:1813-1823; DOI:
10.1056/NEJMoa1708539TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 113
Study NCT01677741 (CDRB436A2102)
Indication BRAFV600 mutant cancers
Phase Phase 1
Patients 86
Primary Outcome
MeasuresSafety, tolerability and pharmacokinetics
Arms/InterventionSingle-arm study of oral dabrafenib (dose based on age
and weight)
Target PatientsPediatric subjects aged 1 year to <18 years with advanced
BRAF V600-mutation positive solid tumors
Expected Completion Q3-2019
Publication TBD
Tafinlar® - BRAF inhibitor
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 114
Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor
Study NCT02039947 COMBI-MB (CDRB436B2204)
Indication BRAFV600 mutant metastatic melanoma
Phase Phase 2B
Patients 126
Primary Outcome
MeasuresIntracranial response rate
Arms/Intervention Dabrafenib 150 mg BID + trametinib 2 mg
Target PatientsPatients with BRAF mutation-positive melanoma that has
metastasized to the brain
Expected Completion Q2-2018 (actual)
Publication• MA Davies G.V., et al. Lancet Oncology. 2017.
DOI:10.1016/S1470-2045(17)30429-
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 115
Tasigna® - Bcr-Abl, c-Kit and PDGF-R tyrosine kinase inhibitor
Study NCT01698905 ENESTop (CAMN107A2408) NCT01844765 DIALOG (CAMN107A2203)
Indication Second line CML/CML-TFR Newly diag. CML and CML res/intol to imatinib/dasatinib
Phase Phase 2 Phase 2
Patients 163 59
Primary Outcome
Measures
No documented confirmed loss of MR4, no documented loss
of MMR and no re-starting of nilotinib therapy
• Resistant/intolerant Ph+ CML in chronic phase: Rate of
Major Molecular Responder (MMR) at 6 cycles
• Newly diagnosed and untreated Ph+ CML in first chronic
phase: Rate of MMR by 12 cycles
Arms/Intervention • Single-arm study of nilotinib
• Newly diagnosed and untreated Ph+ CML in first chronic
phase
• Resistant/intolerant Ph+ CML in chronic phase
• Resistant/intolerant Ph+ CML in accelerated phase
Target Patients
Adult CML-CP patients who received a minimum of 3 years
of TKI therapy, started off with imatinib treatment for > 4
weeks, then switched to nilotinib for at least 2 years prior to
study entry and achieved MR4.5 on nilotinib, but did not
have documented MR4.5 at the time of switch from imatinib
to nilotinib
Pediatric patients with newly diagnosed Ph+ chronic
myelogenous leukemia (CML) in chronic phase (CP) or with
Ph+ CML in CP or accelerated phase (AP) resistant or
intolerant to either imatinib or dasatinib
Expected Completion 2017 (actual) 2017 (actual)
Publication Mahon FX, et al. Ann Intern Med. 2018,168(7):461-470• Presentation at SIOP October 13, 2017
• Manuscript Q1-2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 116
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)
Expected Completion 2021
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 117
PDR001 - PD-1 checkpoint inhibitor
Study NCT03484923 (CPDR001J2201)
Indication Previously treated unresectable or metastatic melanoma
Phase Phase 2
Patients 135
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
Target PatientsAdult patients with previously treated unresectable or
metastatic melanoma
Expected Completion 2021
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 118
Zykadia® - ALK inhibitor
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 119
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 CompletionPart 1 (PK): 2016 (actual)
Part 2 (ORR): Q2-2018 (actual)
Publication
• Part 1 (PK): Cho BC, et al. Journal of Thoracic Oncology;
2017 Jul; 12(9) 1357-1367
• Part 2 (ORR): Cho B et al. Poster Presentation at ESMO
2018; Manuscript submission Q1 2019
Lucentis® - Anti-VEGF
121
Study NCT02375971 RAINBOW (CRFB002H2301) NCT02640664 RAINBOW Extension (CRFB002H2301E1)
Indication Retinopathy of Prematurity (ROP) Retinopathy of Prematurity (ROP)
Phase Phase 3 Phase 3
Patients 224 180
Primary Outcome
Measures
To achieve absence of active Retinopathy of Prematurity
(ROP) and unfavorable structural outcome, patients must
fulfill all the following criteria, 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
• Ranibizumab 0.1 mg
• Laser therapy
• Ranibizumab 0.2 mg
• Ranibizumab 0.1 mg
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 require treatment.
Expected Completion Q1-2018 (actual) 2023
Publication
• EURETINA: Sep-2018
• AAO: Oct-2018
• Primary manuscript: planned submission by end of 2018
to NEJM
TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
RTH258 - Anti-VEGF
Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)
Indication Neovascular age-related macular degeneration (nAMD) Neovascular age-related macular degeneration (nAMD)
Phase Phase 3 Phase 3
Patients 743 1,082
Primary Outcome
Measures
Change in Best Corrected Visual Acuity (BCVA) from
baseline at week 48
Change in Best Corrected Visual Acuity (BCVA) from
baseline at week 48
Arms/Intervention• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
• RTH258 3 mg/50 µL
• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
Target Patients Subjects with exudative age-related macular degeneration Subjects with exudative age-related macular degeneration
Expected Completion Q1-2018 (actual) Q2-2018 (actual)
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)
• Manuscript on 1st results was submitted to Journal of Ophthalmology Dec-2018; 2nd year result manuscript will be
submitted to J. of Ophtha in Q2-2019
• Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 122
RTH258 - Anti-VEGF
Study NCT03481634 KESTREL (CRTH258B2301) NCT03481660 KITE (CRTH258B2302)
Indication Diabetic eye disease Diabetic eye disease
Phase Phase 3 Phase 3
Patients 534 356
Primary Outcome
Measures
Change from baseline in best-corrected visual acuity
(BCVA)
Change from baseline in best-corrected visual acuity
(BCVA)
Arms/Intervention
• RTH258 3 mg/50 µL
• RTH258 6 mg/50 µL
• Aflibercept 2mg/50 uL
• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
Target PatientsPatients with visual impairment due to diabetic macular
edema (DME)
Patients with visual impairment due to diabetic macular
edema (DME)
Expected Completion 2021 2021
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 123
RTH258 - Anti-VEGF
Study NCT03386474 (CRTH258A2301E1)
Indication Neovascular age-related macular degeneration (nAMD)
Phase Phase 3
Patients 150
Primary Outcome
MeasuresNumber of treatment-emergent adverse events
Arms/Intervention• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
Target PatientsPatients with neovascular age-related macular degeneration
who have completed the CRTH258A2301 study
Expected Completion Q3-2018 (actual)
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 124
QAW039 – DP2 receptor antagonist
126
Study NCT02555683 LUSTER-1 (CQAW039A2307) NCT02563067 LUSTER-2 (CQAW039A2314)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 846 846
Primary Outcome
Measures
Reduction in the rate of moderate-to-severe asthma
exacerbations
Reduction in the rate of moderate-to-severe asthma
exacerbations
Arms/Intervention
• QAW039 Dose 1
• QAW039 Dose 2
• Placebo
• QAW039 Dose 1
• QAW039 Dose 2
• Placebo
Target Patients Patients with uncontrolled severe asthma Patients with uncontrolled severe asthma
Expected Completion H1-2020 Q3-2019
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
QAW039 – DP2 receptor antagonist
Study NCT03215758 ZEAL-1 (CQAW039A2316) NCT03226392 ZEAL-2 (CQAW039A2317)
Indication Asthma Asthma
Phase Phase 3 Phase 3
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
Expected Completion H1-2020 H1-2020
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 127
QAW039 – DP2 receptor antagonist
Study NCT03052517 SPIRIT (CQAW039A2315)
Indication Asthma
Phase Phase 3
Patients 1,900 – 2,300
Primary Outcome
Measures
Long term safety: treatment emergent adverse event (AE),
SAE and AE leading to discontinuation from study (52 wks
and 160 wks)
Arms/Intervention
• QAW039 Dose 1
• QAW039 Dose 2
• Placebo
Target Patients Patients with moderate to severe asthma
Expected Completion Q4-2019 (for submission); 2022 (final)
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 128
QMF149 - Long-acting beta2 agonist and inhaled corticosteroid
129
Study NCT02892019 (CQMF149G2202)
Indication Asthma
Phase Phase 2
Patients 80
Primary Outcome
MeasuresTrough FEV1
Arms/Intervention• Indacaterol acetate 75 μg (via Concept1 inhaler)
• Indacaterol acetate 150 μg (via Concept1 inhaler)
Target Patients Children ≥ 6 to < 12 years of age with asthma
Expected Completion Q3-2019
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
Study NCT02554786 PALLADIUM (CQVM149B2301) NCT02571777 IRIDIUM (CQVM149B2302)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 2,216 3,092
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 uncontrolled
asthma despite med-/high-dose ICS or low-dose
ICS/LABA(GINA step 3)
Adult patients with uncontrolled asthma despite med/high-
dose ICS/LABA (GINA ≥4)
Expected Completion Q3-2019 Q3-2019
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 130
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
131
Study NCT03100500 (CQVM149B1305) NCT03100825 (CQVM149B1304)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 51 94
Primary Outcome
Measures
Number of patients who reported treatment emergent
adverse events during the 52 weeks of the study
Number of patients who reported treatment emergent
adverse events during the 52 weeks of the study
Arms/Intervention • Single arm: QMF149 150/320 μg od• Single Arm: QVM149 150/50/160 μg od (Concept1
inhaler)
Target Patients Japanese patients with asthma Japanese patients with Asthma
Expected Completion Q1-2019 Q2-2019
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 802 1,251
Primary Outcome
MeasuresTrough FEV1
Change from baseline in Asthma Quality of Life
Questionnaire (AQLQ) total score
Arms/Intervention• QMF149 150/80 µg
• MF 200 µg
• QVM149 150/50/80 μg
• QVM149 150/50/160 μg
• Salmeterol/fluticasone 50/500 μ + tiotropium 5 μg
Target PatientsAdult and adolescent (>12 years) patients with in poorly
(i.e., inadequately) controlled asthmaPatients with uncontrolled asthma
Expected Completion Q1-2019 Q3-2019
Publication TBD TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 132
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
Study NCT03137784 SILVER (CQVM149B2204)
Indication Asthma
Phase Phase 2
Patients 148
Primary Outcome
Measures
Evaluate the bronchodilator effects of NVA237 (25 ug and 50
ug) compared to placebo in terms of trough FEV1
Arms/Intervention• NVA237 (glycopyrronium bromide) 25/50 μg
• Placebo
Target Patients Asthma patients
Expected Completion Q1-2018 (actual)
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 133
Xolair® – anti-IgE antibody
134
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 Patients
Patients with severe Japanese cedar pollinosis, whose
symptoms were inadequately controlled with current
recommended therapies
Expected Completion Q1-2019
Publication
Nov 2018: Late breaking abstract for the American
Association of Allergy, Asthma and Immunology (AAAAI)
annual meeting in Feb 2019
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Erelzi® - Biosimilar etanercept
136
Study NCT02638259 (GP15 301)
Indication Immunology
Phase Phase 3
Patients 376
Primary Outcome
Measures
Change in DAS28-CRP score from baseline to week 24 in
patients treated with GP2015 and patients treated with Enbrel
Arms/Intervention• GP2015 50 mg
• EU-authorized Enbrel® 50mg
Target Patients Patients with moderate to severe, active rheumatoid arthritis
Expected Completion Q4-2017 (actual)
Publication
• Kavanaugh et al. Arthritis Rheumatol 2017; 69 (suppl 10)
• 48 week: Abstract to EULAR 2018
• 24 week: Manuscript published in RMD Open (20-Nov-
2018)
• 48 week: Manuscript in Q4-2018 (submitted to Journal
Arthritis Research &Therapy on 08-Nov-2018)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Rixathon® - Biosimilar rituximab
137
Study NCT02514772 (GP13 302) NCT01419665 (GP13 301)
Indication Immunology Oncology
Phase Phase 3 Phase 3
Patients 107 629
Primary Outcome
Measures
Incidence of adverse events and serious adverse events,
anaphylactic reactions, hypersensitivity; immunogenicity Overall response rate in patients with FL
Arms/Intervention• GP2013
• Rituxan® or MabThera®
• GP2013
• MabThera®
Target PatientsPatients with active Rheumatoid Arthritis, previously treated
with Rituxan or MabThera (ASSIST-RT)
Patients with previously untreated, advanced stage follicular
lymphoma (ASSIST-FL)
Expected Completion 2016 (actual) Q2-2018 (actual)
Publication
• ACR Q4-2017 Poster
• Tony, H-P et al, Arthritis Care & Research, 2018
(accepted for publication)
• Amersdorffer J, et al and Jurczak W., et al presented at
ESMO 2017, Published in Lancet Hematology (doi:
10.1016/ S2352-3026(17)30106-0)
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Hyrimoz® - Biosimilar adalimumab
138
Study NCT02016105 ADACCESS (GP17-301) NCT02744755 ADMYRA (GP17-302)
Indication Immunology Immunology
Phase Phase 3 Phase 3
Patients 465 353
Primary Outcome
Measures
PASI 75 response rate at week 16 in patients treated with
GP2017 and patients treated with Humira®
Change in DAS28-CRP score from baseline to week 12 in
patients treated with GP2017 and patients treated with
Humira®
Arms/Intervention• GP2017
• Humira® adalimumab
• GP2017
• US licensed Humira® adalimumab
Target PatientsPatients with moderate to severe chronic plaque-type
psoriasisPatients with moderate to severe active rheumatoid arthritis
Expected Completion 2016 (actual) Q3-2018 (actual)
Publication
• 51 week data and switch data, Blauvelt et al. BJD, 2018,
https://doi.org/10.1111/bjd.16890
• Blauvelt et al. presented at ACR 2017, Blauvelt et. al.
presented at AAD 2017, Blauvelt et.al. presented at
EADV 2017, Blauvelt et al., presented at ACG 2017,
Blauvelt et.al. presented at UEGw 2017
• Abstract, oral presentation at ACR 2018
• Abstract and poster at EULAR 2019
• Manuscript with study results journal TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Tobramycin – An aminoglycoside antibiotic
140
Study NCT02712983 iBEST-1 (CTBM100G2202)
Indication Bronchiectasis
Phase Phase 2
Patients 105
Primary Outcome
MeasuresP. aeruginosa density in sputum
Arms/Intervention
Three dose regimens, each of them having 3 treatment
arms:
• Tobramycin inhalation powder
• Tobramycin inhalation powder and placebo
• Placebo
Target PatientsPatients with non-cystic fibrosis bronchiectasis and
pulmonary P. aeruginosa infection
Expected Completion Q3-2019
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated
141
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
Expected Completion H2-2019
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
KAE609 – Plasmodium Falciparum Inhibitor – spiroindolone against PfATP4
142
Study NCT03334747 (CKAE609A2202)
Indication Malaria
Phase Phase 2
Patients 150
Primary Outcome
Measures
CTCAE grades increase from baseline in alanine
aminotransferase (ALT) or aspartate aminotransferase
(AST)
Arms/Intervention• KAE609
• Coartem
Target Patients Adults with uncomplicated Plasmodium Falciparum malaria
Expected Completion Q3-2019
Publication TBD
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation
Key definitions and trademarks
Novartis Q4 and FY 2018 Results | January 30, 2019 | Novartis Investor Presentation 143
This presentation contains several important words or phrases that we define as below:
AE: Adverse Event
ALL: Acute lymphatic leukemia
AMD: Age-Related Macular Degeneration
AML: Acute myeloid leukemia
Approval: In Pharmaceuticals and Alcon in US and EU; each indication and regulator combination counts as
approval; excludes label updates, CHMP opinions alone and minor approvals
aRCC: advanced renal cell cancer
AS: Ankylosing Spondylitis
bid: twice a day
BC: Breast cancer
BCMA: B-cell maturation antigen
BCVA: best corrected visual acuity
BS: Biosimilars
BTD: Breakthrough therapy designation
CGRP: Calcitonin gene-related peptide
CLL: Chronic lymphocytic leukemia
CM: Chronic migraine
CML: Chronic myeloid leukemia
COPD: Chronic Obstructive Pulmonary Disease
CR: complete remission
CRC: Colorectal Cancer
CSU / CIU: Chronic spontaneous urticaria / Chronic idiopathic urticaria
CVRR: Cardiovascular risk reduction
DLBCL: Diffuse large B-cell lymphoma
DMC: Data monitoring committee
EF: ejection fraction
EM: Episodic migraine
FL: Follicular lymphoma
FPFV: First patient first visit
GBM: Glioblastoma multiforme
HF: Heart failure
HF-pEF: Heart failure with preserved ejection fraction
HFrEF: Heart failure with reduced ejection fraction
HR+/HER2- mBC:Hormone Receptor positive / Human Epidermal growth factor receptor 2 negative metastatic
breast cancer
LoE: Loss of exclusivity
M/M: Multiple myeloma
MF: Myelofibrosis
MI: Myocardial infarction
MS: Multiple sclerosis
NASH: Non-Alcoholic Steatohepatitis
NET: Neuroendocrine tumor
NSCLC: Non-small cell lung cancer
NTD: New Therapeutic Drug
od: once a day
ORR: Overall response rate
OS: Overall survival
PA: Prior authorization
PASI 90: 90% reduction in Psoriasis Area Severity Index from baseline
PFS: Progression free survival
PSA: Prostate specific antigen
PsA: Psoriatic arthritis
PsO: Psoriasis
PV: Polycythemia vera
PY: Prior year
QoL: Quality of Life
RCC: Renal cell cancer
r/r ALL: relapsed/refractory acute lymphoblastic leukemia
RRMS: relapsing-remitting multiple sclerosis
SCPC: Sickle cell pain crisis
SpA: Spondyloarthropathy
SPMS: Secondary progressive multiple sclerosis
TFR: Treatment-free Remission
TNBC: Triple negative breast cancer
Trademarks
Eylea® is a registered trademark of Regeneron Pharmaceuticals, Inc.
Enbrel®, Epogen® and Neulasta® are a registered trademark of Amgen Inc.
Humira® is a registered trademark of AbbVie Ltd.
Remicade® and Stelara® are registered trademarks of Janssen Biotech, Inc.
Rituxan® is a registered trademark of Biogen Inc