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Meet Novartis Management
Global Drug Development
May 31, 2017
Global Drug Development
Disclaimer
| Meet Novartis Management | May 31, 2017 | Investor Presentation2
This presentation contains forward-looking statements that can be identified by terminology such as such as “potential,” “expected,” “will,” “planned,” 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; potential shareholder returns or credit ratings; or regarding the potential outcome of the announced review of options being undertaken to maximize shareholder
value of the Alcon Division; or regarding the potential financial or other impact on Novartis or any of our divisions of the significant reorganizations of recent years, including
the creation of the Pharmaceuticals and Oncology business units to form the Innovative Medicines Division, the creation of the Global Drug Development organization and
Novartis Operations (including Novartis Technical Operations and Novartis Business Services), the transfer of the Ophthalmic Pharmaceuticals products of our Alcon Division
to the Innovative Medicines Division, the transfer of selected mature, non-promoted pharmaceutical products from the Innovative Medicines Division to the Sandoz Division,
and the transactions with GSK, Lilly and CSL; or regarding the potential impact of the share buyback plan; or regarding potential future sales or earnings of the Novartis Group
or any of its divisions; or by discussions of strategy, plans, expectations or intentions. You should not place undue reliance on these statements. 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. There can be no guarantee that any new products will be approved for sale in any market, or that any new indications will be
approved for any existing products in any market, or that any approvals which are obtained will be obtained at any particular time, or that any such products will achieve any
particular revenue levels. Nor can there be any guarantee that the review of options being undertaken to maximize shareholder value of the Alcon Division will reach any
particular results, or at any particular time. Neither can there be any guarantee that Novartis will be able to realize any of the potential strategic benefits, synergies or
opportunities as a result of the significant reorganizations of recent years, including the creation of the Pharmaceuticals and Oncology business units to form the Innovative
Medicines Division, the creation of the Global Drug Development organization and Novartis Operations (including Novartis Technical Operations and Novartis Business
Services), the transfer of the Ophthalmic Pharmaceuticals products of our Alcon Division to the Innovative Medicines Division, the transfer of selected mature, non-promoted
pharmaceutical products from the Innovative Medicines Division to the Sandoz Division, and the transactions with GSK, Lilly and CSL. Neither can there be any guarantee
that shareholders will achieve any particular level of shareholder returns. Nor can there be any guarantee that the Group, or any of its divisions, will be commercially
successful in the future, or achieve any particular credit rating or financial results. In particular, management’s expectations could be affected by, among other things:
regulatory actions or delays or government regulation generally; the potential that the strategic benefits, synergies or opportunities expected from the significant
reorganizations of recent years, including the creation of the Pharmaceuticals and Oncology business units to form the Innovative Medicines Division, the creation of the
Global Drug Development organization and Novartis Operations (including Novartis Technical Operations and Novartis Business Services), the transfer of the Ophthalmic
Pharmaceuticals products of our Alcon Division to the Innovative Medicines Division, the transfer of selected mature, non-promoted pharmaceutical products from the
Innovative Medicines Division to the Sandoz Division, and the transactions with GSK, Lilly and CSL may not be realized or may take longer to realize than expected; the
inherent uncertainties involved in predicting shareholder returns or credit ratings; 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 which commenced in prior years and will continue this year; safety, quality or
manufacturing issues; global trends toward health care cost containment, including ongoing pricing and reimbursement pressures, such as from increased publicity on
pharmaceuticals pricing, including in certain large markets; uncertainties regarding actual or potential legal proceedings, including, among others, actual or potential product
liability litigation, litigation and investigations regarding sales and marketing practices, intellectual property disputes and government investigations generally; general
economic and industry conditions, including uncertainties regarding the effects of the persistently weak economic and financial environment in many countries; uncertainties
regarding future global exchange rates; uncertainties regarding future demand for our products; and 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.
Broad, high value late stage pipeline with depth and quality assets in each
therapeutic area2
Focus on operational agility and productivity with goal to invest ~20% of
Innovative Medicines sales in R&D while driving dynamic growth1
Strong and diverse set of emerging Phase II assets from NIBR research as
well as from external deals3
Key Messages
| Meet Novartis Management | May 31, 2017 | Investor Presentation2
Continued strong track record of R&D
excellence
| Meet Novartis Management | May 31, 2017 | Investor Presentation3
1. Since the introduction of Breakthrough Designation pathway by the FDA, Novartis pipeline included a total of 16 breakthrough designations cumulatively of which 13 are currently actively under development or in approvedindications. Includes products in-licensed (with BTD granted prior to the acquisition of the asset) or out-licensed (with BTD granted to Novartis prior to divestment) 2. In number of new molecular entities (NMEs) approvedincluding fixed dose combinations 2007-2016
175+Projects in the
clinic
90+New molecular
entities in the
clinic
16Breakthrough
Therapy
designations1
#1In US/EU
approvals past
10 years2
40Potential filings
in US and EU
2017-2020
Progressing across 4 pillars of transformation
to achieve a step change in performance
| Meet Novartis Management | May 31, 2017 | Investor Presentation4
Driving efficiency
and lowering our
cost base
Best-in-class
portfolio
prioritization
Leading
technology
Inspired
culture and
leaders
1
2 4
3
Rigorous disease area and portfolio
prioritization
| Meet Novartis Management | May 31, 2017 | Investor Presentation5
• Evaluation of disease areas,
decision on future focus
• Examples: Prioritized liver diseases,
de-prioritized transplant
• Evaluation based on science,
risk, and valuation
• Funding consistent with priority
categories
• 70% of resources focused on
top projects
• Increasing number of
projects with blockbuster
potential
• Complementing portfolio
through deals and
alliances
ILLUSTRATIVE
Project prioritization
Race Go Pace Retire
Prioritizing bigger betsDisease/therapy area reviews
Un
me
t n
ee
d/a
ttra
cti
ve
ne
ss
Novartis strength
1
Driving operational performance across an
integrated organization
| Meet Novartis Management | May 31, 2017 | Investor Presentation6
~10,000 associates
90+ NMEs in
clinic across 7
therapeutic areas
1 regulatory
submission
every 6 minutes
~175 programs
in clinic
Leveraging global
scale (3 major centers;
52 countries)
~500+ active trials with ~80,000+ patients
in ~70+ countries 300+ million observations
INTEGRATED
ORGANIZATION
2
Scaling digital development: Building
capabilities for data centered innovation
| Meet Novartis Management | May 31, 2017 | Investor Presentation7
Digital TherapiesData and Trial Operations
Dramatically increase the speed and
quality of trials; with a step change
reduction in cost
Digital technologies as therapeutics
or to support the patient journey
Note: All trademarks are property of the respective owners
3
Progressing late stage development of
potential blockbusters1
| Meet Novartis Management | May 31, 2017 | Investor Presentation8
Therapeutic area Molecule Indication MoAExp. pivotal
trial readout
Onco Oncology
LEE011 (Kisqali®) HR+ advanced or metastatic breast cancer CDK4/6 inhibitor ✓
CTL019 (CAR-T) r/r B-Cell ALL, DLBCL CAR-T Q2 20172
SEG101 (crizanlizumab) Sickle cell pain crises Anti-P-selectin 2018
CM Cardio-metabolicLCZ696 (Entresto®) Heart failure with preserved EF ARNI 2019
ACZ885 (canakinumab) CV risk reduction Anti-IL1β H2 2017
NS Neuroscience
OMB157 (ofatumumab) Relapsing multiple sclerosis CD20 2019
BAF312 (siponimod)3 Relapsing multiple sclerosis S1P receptor modulator ✓
AMG 334 (erenumab)4 Prophylaxis of migraine CGRP receptor antagonist ✓
I&DImmunology&
DermatologyAIN457 (Cosentyx®) Non-radiographic axial SpA Anti-IL17A 2018
Resp Respiratory
QVM149 (indacaterol, glycopyrronium,
mometasone)Asthma LABA + LAMA + ICS 2018
QAW039 (fevipiprant) Asthma CRTh2 antagonist 2019
Oph Ophthalmology RTH258 (brolucizumab) Neovascular AMD Anti-VEGF (scFv) H1 2017
Bios Biosimilars Multiple Multiple Multiple Ongoing
1. Blockbuster potential refers to specified indication 2. Ped. r/r B-cell ALL filed and priority review granted; Breakthrough Therapy designation granted for DLBCL 3. Next steps to be evaluated in consultations with health authorities 4. In collaboration with Amgen; Novartis has AMG 334 rights outside of Japan and co-commercialization in the US
Phase 2 pipeline deep with mix of internal and
external assets
| Meet Novartis Management | May 31, 2017 | Investor Presentation9
Therapeutic area Molecule Indication Externally sourced
Onco
Oncology 18 IO assets incl. combos Multiple Co-stim; Others
ABL001 CML
BYL719 Breast Cancer
INC280 NSCLC Incyte
Jakavi® steroid refractory acute GVHD Incyte
CM
Cardio-metabolic APO(a)-LRx1 High risk CVRR Ionis Pharmaceuticals / Akcea
TherapeuticsAPOCIII-LRx1 High risk CVRR
LHW090 Resistant hypertension
LIK066 Weight loss Advinus
MAA868 Stroke prevention
NS
Neuroscience BYM338 Sarcopenia hip fracture
CNP520 Alzheimer’s
LMI070 Spinal muscular atrophy
EMA401 Neuropathic Pain Spinifex Pharmaceuticals, Inc.
I&D
Immunology-
Dermatology
CJM112 Multiple immune disorders
VAY7852 NASH/Cirrhosis Conatus Pharmaceuticals
LJN452 Non-Alcoholic Steatohepatitis
QGE031 Chronic spontaneous urticaria Tanox/Genentech
VAY736 Sjoegren’s syndrome / AIH
ZPL389 Atopic dermatitis Ziarco
Resp
Respiratory ACZ885 Sarcoidosis
CJM112 Asthma
QBW251 Cystic fibrosis/COPD
OphOphthalmology UNR844 Presbyopia Encore Vision
ECF843 (Lubricin) Dry eye Lubris Biopharma
1. Option to license 2 Emricasan
Trial Indication Opportunity Status
Planned next
milestone
MONALEESA-3 1st & 2nd line in combination
with Fulvestrant
CDK 4/6i in Phase 3
study with Fulvestrant
Fully enrolled;
readout H2 2017
Filing in H1
2018 if positive
MONALEESA-7 Pre-menopausal women 1st line
in combination with goserelin +
ET2
CDK 4/6i in Pre-
menopausal setting
Fully enrolled;
readout H2 2017
Filing in H1
2018 if positive
Adjuvant Trials
EarLEE-1
EarLEE-2
High risk of recurrence trial
Intermediate risk of recurrence
trial
Focus on populations
with higher risk of relapse
Trial initiation Trials to start
Q2-Q3 2017
Kisqali®1
: clinical trial programs advancing across
indications for HR+/HER2- advanced breast cancer
| Meet Novartis Management | May 31, 2017 | Investor Presentation10
Onco
CM
NS
I&D
Resp
Oph
Bios
1. Formerly known as LEE011 2. Endocrine therapy
FDA approved Rydapt®1
in newly diagnosed
FLT3+ AML2
and advanced SM3
| Meet Novartis Management | May 31, 2017 | Investor Presentation11
1. Formerly known as midostaurin (PKC412) 2. Acute Myeloid Leukemia 3. Systemic Mastocytosis 4. R. Stone et al. presented at American Society of Hematology (ASH) Annual Meeting, December 6th 2015, Orlando, FL, USA (Plenary Presentation, Abstract #5) 5. RYDAPT US Prescribing Information April 28, 2017 6. Swissmedic Rydapt Prescribing Information May 05, 2017 7.USA: as detected by an FDA approved test. FDA approved FLT3 mutation testing as a companion diagnostic for Rydapt is performed by The Laboratory for Personalized Molecular Medicine, a subsidiary of Invivoscribe Technologies, Inc. 8. Kantar Health EPI G7 (US, DE, FR, IT, UK, SP, JP); 9. Patel JP, Gönen M, Figueroa ME, et al. Prognostic relevance of integrated genetic profiling in acute myeloid leukemia. N Engl J Med. 2012; 22;366(12):1079-1089;
Onco
CM
NS
I&D
Resp
Oph
Bios
• Targeted therapy approved for newly
diagnosed FLT3 mutated AML in combination
with standard chemotherapy5,6,7
• About 34k8 patients in G7. ~ 1/3 of patients
have a FLT3 gene mutation9 and ~60% are fit
for chemotherapy, translating to ~6000
Rydapt®-eligible patients in the G7
• Worldwide regulatory submissions and HA
reviews ongoing
High unmet need Differentiated label
• First new treatment in more than 25 years for
AML patients eligible for standard therapy
• Rydapt® treatment regimen in FLT3-mutated
AML demonstrated a significant improvement
in overall survival with a 23% reduction in the
risk of death4
• Rydapt® is the first and only approved therapy
for three types of SM collectively known as
advanced SM, a group of ultra-rare, life-
threatening conditions
Progressing our Immuno-Oncology strategy
| Meet Novartis Management | May 31, 2017 | Investor Presentation12
Advancing CAR-T PD-1 update Ready for IO 2nd Gen
18 second generation agents in
mono or combo progressing in
early studies
• Manufacturing optimization
• Filed in pediatric/young adult r/r ALL
in US, priority review granted, filing
in Europe targeted for H2 2017
• Breakthrough Therapy designation
awarded for DLBCL, planned filing in
US and EU in H2 2017
• CLL and Multiple Myeloma
progressing
• Solid tumors in FIH trials
Onco
CM
NS
I&D
Resp
Oph
Bios
Tumor Type PDR001 (PD-1 Antagonist)
Melanoma Phase 3 trial in combination with
Tafinlar®+Mekinist®: FPFV
achieved for run-in
NET Pivotal Phase 2 FPFV achieved
HCC Phase 1b in combination with
sorafenib FPFV achieved
NSCLC Phase 1b FPFV achieved
CRC Phase 1b FPFV Q3 2017
SEG101 (crizanlizumab) – planned filing in 2018
| Meet Novartis Management | May 31, 2017 | Investor Presentation13
1. SEG101 (formerly SelG1) 5mg/kg monthly Source: Ataga et al, NEJM Dec 3, 2016 (online)
SUSTAIN trial (Phase 2)SEG1011 significantly (p=.010) reduced Sickle Cell Pain Crises (SCPC) and generally well tolerated
• Planning FDA submission in 2018;
assuming successful PK/PD
comparability study to final
manufacturing process
• Discussions progressing in Europe
• Additional long term safety and
efficacy data expected to be
generated in adult and pediatric
studies after submission
SEG101
Onco
CM
NS
I&D
Resp
Oph
Bios
11.0
6.9
2.95.1
1.43.0
24.0
4.0
1.1
10.3
4.1
1.6
Median
annual rate of
uncomplicated
pain crisis
+2.9x
Number of
patients with
SCPC rate
of zero at
end of study
+2.2x
Median annual
rate of days
hospitalized
+2.0x
-63%
-42%
-45%
Median time to
first pain crisis
(months)
Median
annual rate
of pain crises
Median time to
second pain
crisis (months)
Placebo (N=67)
SEG101 (N=65)
Advancing ABL001, a potent, allosteric
inhibitor of BCR-ABL1
| Meet Novartis Management | May 31, 2017 | Investor Presentation14
• Effective and overall well-tolerated in
Phase 1 in heavily treated CML patients
resistant to or intolerant of prior TKIs
‒ Pivotal study in ≥3rd CML-CP patients planned to
start in 2017
‒ Phase 1 study ongoing to test other regimens and
TKI combination treatments
• Combination treatments: in vitro data of
ABL001 with nilotinib, imatinib and
dasatinib suggest synergistic effects for
BCR-ABL inhibition2
a. Patients had ≥ 6 months of treatment exposure or achieved response within 6 months b. BCR-ABL1IS reduction achieved c. Patients had ≥ 12 months of treatment exposure or achieved
response within 12 months 1. B. Sellers: AACR 2016 2. T. Hughes and D. White et al; ASH 2016; Abstract 1121 Abbreviations: CCyR = complete cytogenetic response; CHR = complete
hematologic response; CML-CP = chronic myeloid leukemia - chronic phase; MR = molecular response; Ph+ CML = Philadelphia chromosome positive chronic myeloid leukemia; TKI = tyrosine
kinase inhibitor
Responses in CML-CP patients treated
with ABL001 with ≥3 months exposure
on study
ABL001
Onco
CM
NS
I&D
Resp
Oph
Bios
0
10
20
30
40
50
60
70
80
90
100
Hematologic relapse
(n = 16)
Cytogenetic relapse
(> 35% Ph+; n = 12)
Pati
en
ts W
ith
Resp
on
se (
%)
CHR:
88% (14/16) CCyR:
75% (9/12)MR:
42% (16/38)
Molecular relapse
(no MMR; n = 38)
Hematologic Response
Within 6 Months
Cytogenetic Response
Within 6 Monthsa
Status at Baseline
Molecular Response
Within 12 Monthsb,c
BYL719 shows promising data in PIK3CAmut
breast
cancer patients in combination with Fulvestrant
| Meet Novartis Management | May 31, 2017 | Investor Presentation15
• Pre-clinical data demonstrated
synergistic impact of alpelisib +
fulvestrant compared to fulvestrant
alone1
• Pivotal clinical trials currently enrolling
for alpelisib combination in advanced
breast cancer (BC) patients harboring
the PIK3CA mutation
• PI3K/mTOR pathway is most
frequently altered pathway in breast
cancer with PIK3CA mutations present
in approx. 35% of hormone-receptor
positive (HR+) breast cancer patients2
• FDA filing planned in 2019
Onco
CM
NS
I&D
Resp
Oph
Bios
BYL719
Source: 1. Bosch et al. Sci Transl Med. 2015 Apr 15;7(283):283ra51; 2. The Cancer Genome Atlas Network. Nature. 2012;490:61–70
INC280 (capmatinib): Multiple potential first-
in-class indications
| Meet Novartis Management | May 31, 2017 | Investor Presentation 16
Lung
• cMET amplified and/or mutation
− 5-7% of lung cancer
− Potentially 1st in class
• EGFR mutations
- 16% of lung cancer (up to 40% in Asia)
- 1L in combination with EGF816 to prevent
T790/cMET resistance
- 2L in combination with erlotinib to treat cMET
resistance
- Potentially 1st cMET/EGFR combination
• Combinations with immuno-oncology (IO) being
explored
- INC280 enhances dendritic cell activity, thereby
potentially enhancing IO treatments when used in
combination
Hepatocellular Carcinoma (HCC)
• Accounts for 70-90% of liver cancers worldwide.
Approx. 745,500 deaths caused by liver cancer
globally in 20121
• 2L HCC in combination with PDR001 (IO)
1. American Cancer Society. Global Cancer Facts & Figures 3rd Edition. Atlanta: American Cancer Society; 2015
Onco
CM
NS
I&D
Resp
Oph
Bios
Entresto®
- investigating potential treatment for
~9m chronic HF patients without effective therapy
| Meet Novartis Management | May 31, 2017 | Investor Presentation17
1. Potential patients are defined by the indications studied in the ongoing / planned trials in HFpEF and post acute MI. Potentially eligible population dependent on trial results and label. Estimate for
US+EU5 is ~4.2m Current estimates based on Decision Resources; Diller et al. Archives of Family Medicine. 1999; 8(5):414-420; Novartis analyses;
Abbreviations: HF-rEF=heart failure-reduced ejection fraction; HF-pEF=heart failure-preserved ejection fraction; AMI=acute myocardial infarction
Onco
CM
NS
I&D
Resp
Oph
Bios
HF-rEFApproved
HF-pEF Post-AMI
Potentially
eligible patients1
~8.5m ~8.5m ~0.3m
Pivotal trials PARADIGM PARAGON PARADISE
Key supportive
trials
TRANSITION
PIONEER HF
Chronic Heart Failure
Trial Indication Status
Next expected
milestone
HF-pEF1 Fully enrolled 4 months
ahead of plan
Interim analysis 2018
Filing in 2019
Post-AMI2 EnrollingCompletion 2019
Filing in 2020
Pre- vs. post-discharge
following ADHF (HF-rEF)Enrollment on track Completion 2018
In hospital initiation vs.
Enalapril following ADHF3 Enrollment on track Completion 2018
Entresto®
expansion studies all on track
| Meet Novartis Management | May 31, 2017 | Investor Presentation18
1. HF-pEF: heart failure with preserved ejection fraction 2. AMI: acute myocardial infarction 3. ADHF: acute decompensated heart failure
Onco
CM
NS
I&D
Resp
Oph
Bios
Entresto®
- new evidence could enhance standard
of care status in HF patients with diabetes
| Meet Novartis Management | May 31, 2017 | Investor Presentation19
Onco
CM
NS
I&D
Resp
Oph
Bios
• PARADIGM-HF post-hoc analysis1
suggests that Entresto® has favorable
metabolic benefits in HF patients with
diabetes:
- New use of Insulin was reduced by 29%, vs.
enalapril (p=.005)
- HbA1c reduction of -0.26% vs. -0.16% at 1
year vs. enalapril (p=.002)
• Engagement with health authorities for
potential label enabling studies initiated
1. JP Seferovic et al. www.thelancet.com/diabetes-endocrinology. Online Mar 18, 2017
On track for readout mid-2017
• CANTOS Phase 3 trial of ACZ885 in
cardiovascular risk reduction
• 1,400 events reached as agreed with
regulatory authorities, LPLV reached
• Study has passed 3 futility analyses
and 22 DMC safety reviews
• Potentially addressing ~4m patients in
G7 with MI history with high
inflammation burden
Population
• History of MI
• hsCRP≥2mg/L
• On SoC treatment
Primary
endpoint• Composite of CV death, MI or Stroke
Key
secondary
endpoints
• Primary + unplanned revascularization
• New onset of Type 2 Diabetes
Sample size
• 1,400 primary events provides ~90% power to detect 20% RRR vs. placebo
• 10,065 patients randomized
CANTOS trial design
CANTOS has reached the protocol defined
number of events, on track for mid-2017 readout
| Meet Novartis Management | May 31, 2017 | Investor Presentation20
ACZ885
Onco
CM
NS
I&D
Resp
Oph
Bios
Ionis/Akcea Option Deal: Targeting two highly
validated CV targets in Phase 2b studies
| Meet Novartis Management | May 31, 2017 | Investor Presentation21
Source: Novartis Qualitative Research Note: option deal subject to customary closing conditions and regulatory approval
Lp(a)
>50mg/dl
Patients on optimized LDL-C therapy at high risk
Routine or Post Event Testing of Residual Risk Factors
Lp(a)-LRX
• Genetically validated
• Established biomarker
• Phase 2b ongoing
• End of Phase 2 ~2018
APOCIII-LRx
• Genetically validated
• Established biomarker
• Phase 2 planned
• End of Phase 2 2019
ApoCIII
>20mg/dl
and/or TGs
>200mg/dl
CVRR
Onco
CM
NS
I&D
Resp
Oph
Bios
LIK066, an SGLT1/2 inhibitor, with potential
for best-in-class weight loss
| Meet Novartis Management | May 31, 2017 | Investor Presentation22
• Inhibition of both SGLT11
and SGLT21
– Blocks reabsorption of filtered
glucose in the kidney
– Reduces intestinal glucose
absorption
• Aim to achieve weight loss
through dual mechanism of
action
• Proof-of-concept data projected
more than 10% placebo-adjusted
weight loss at 52 weeks
• Additional expected benefits
include improved glycemic control,
lipid profile, blood pressure
• Phase 2b trial planned to start in
H1 2017
SGLT1/2 dual mode of action
1. SGLT = sodium-glucose co-transporter
LIK066 development
LIK066
Onco
CM
NS
I&D
Resp
Oph
Bios
BAF312 filing planned in US H1 2018 in relapsing
MS; labeling of population studied a review issue
| Meet Novartis Management | May 31, 2017 | Investor Presentation23
RRMS SPMS
FREEDOMS I
N=1272
DECIDE
N=1841
EXPAND
N=1651
Fingolimod Daclizumab Siponimod
Age, years (mean) 37.0 36.3 48.0
Time since onset, years (mean) 8.2 / 6.7 7.0* 16.8
Time since (R)MS diagnosis, years (mean) 5.1 4.2 12.6
% relapse-free prior 2 years 0% 0% 63.9%
EDSS (mean) 2.4 2.5* 5.4
EDSS (% pts ≥6.0) 0% 0% 55.6%
Acute MRI lesions (T1 Gd+, % of patients) 61.9% 44%* 21.3%
MRI disease burden [T2 lesion vol (mean)] 6.4 cm3 9.7 cm3 15.3 cm3
Primary Endpoint ARR ARR 3m Disability
progression
FREEDOMS I: Novartis, data on file; DECIDE: Kappos et al 2015; EXPAND: Kappos, et al. AAN 2016
*daclizumab arm only
• Filing based on EXPAND study in
SPMS and Phase 2 study in RRMS
• Novartis to propose labeling to
describe unique population studied
• Timing driven by collection of
additional safety data and finalizing
manufacturing submission
• Safety profile is in line with other
compounds in this class
BAF312
Onco
CM
NS
I&D
Resp
Oph
Bios
0
2
4
6
8
10
12
Placebo OMB157
99.8%
(p<0.001)
OMB157 (ofatumumab) demonstrated strong
efficacy in Phase 2 studies in RRMS
| Meet Novartis Management | May 31, 2017 | Investor Presentation24
• Two parallel Phase 3 studies vs.
oral teriflunomide (Aubagio®2) in
relapsing MS started 3Q16
• Planned filing 2019
OMB157 suppressed >90% of new MS
lesions in the brain as measured by MRI1
Me
an
Nu
mb
er
cu
mu
lati
ve
Ga
d le
sio
ns
we
ek
s 8
-24
OMB157
1. Sorensen PS et al., Neurology 2014;82(7):573-81; MRI = Magnetic Resonance Imaging; Pooled analysis of all doses levels in the Sorensen study (Ph IIa); 100 mg, 300 mg, 700 mg - i.v.
2. Aubagio® is a registered trademark of Sanofi
Onco
CM
NS
I&D
Resp
Oph
Bios
AMG 3341: first-in-class submitted in the US
and in the EU
| Meet Novartis Management | May 31, 2017 | Investor Presentation25
1. Development in collaboration with Amgen, Companies to co-commercialize in the US, Novartis to have exclusive rights in rest of world excluding Japan 2. Global Burden of Disease Study 2015
Collaborators. Lancet. 2016
Onco
CM
NS
I&D
Resp
Oph
Bios
AMG 334
• Migraine in the TOP TEN world
leading of all causes of years lived
with disability2
• 9.4 million episodic and 4.2 million
chronic migraine patients in EU-5
• Fully human, potent, selective
CGRP antagonist targeting
receptor
• Consistent data in phase 2 and 3
clinical studies
AMG 334 with unique profile Robust market access strategy
• Filing activities on track for first-in-class
submission in Q2 2017
• Encouraging subpopulation efficacy data,
recently presented at AAN
• Access strategy focuses on subpopulation
with high unmet medical need. Dedicated
LIBERTY clinical trial underway
EMA401: Novel treatment option for refractory
peripheral neuropathic pain (PNP)
| Meet Novartis Management | May 31, 2017 | Investor Presentation26
1. Lancet (2014), Rice et al.
• PNP is excruciating, appearing e.g. after
herpetic infection, in diabetic patients or in
patients treated with chemotherapy
• SoC medications partially effective with CNS
adverse effects.
• EMA401 is a novel, peripherally acting AT-II
antagonist
• Phase 2 proof-of-concept (PHN): strong
efficacy already seen at 4 weeks
- Efficacy potentially increasing over longer time
period (plateau not reached at 4 weeks) or with
higher dosing
• Phase 2 dose-finding to begin in 2017
EMA401 improved pain significant
in patients with post-herpetic
neuralgia
Time course of mean change in pain intensity from baseline by week of treatment
Data are mean change from baseline in average weekly numerical rating scale pain score
per week of treatment (last observation carried forward imputation). Error bars are standard
error of the mean. *EMA401 vs placebo p=0.0184. †EMA401 vs placebo p=0.0066
Onco
CM
NS
I&D
Resp
Oph
Bios
EMA401
CNP5201
program focused on genetically
identified population at risk of Alzheimer’s
| Meet Novartis Management | May 31, 2017 | Investor Presentation27
• BACE inhibitor
• Clinical program aims to
prevent onset of AD symptoms
in healthy subjects with strong
genetic pre-disposition 2, 3
• Second pivotal study being
initiated in 2H2017
• Completion expected >2022
• FDA Fast Track designation
received
1. CNP520: co-developed with Amgen 2. In collaboration with Banner Alzheimer’s Institute; includes Novartis investigational immunotherapy CAD106 3. APOE4 Homozygotes and amyloid-
positive APOE4 Heterozygotes, 60-75 years old
CNP520Cognitively normal
Preclinical ADMild Cognitive Impairment /
Prodromal AD
Dementia / Mild-moderate-severe AD
Aβ
Tau-meditated neuronal injury
and dysfunction
Clinical function
- 20 years 0Dementia diagnosis
Years
AD
bio
mark
ers
Norm
al
Ab
no
rmal
Most competitors
CNP520
Onco
CM
NS
I&D
Resp
Oph
Bios
Strengthening leadership by targeting new frontiers
in dermatology, rheumatology and hepatology
| Meet Novartis Management | May 31, 2017 | Investor Presentation28
Onco
CM
NS
I&D
Resp
Oph
Bios
Now 2019-22 2022-23 >2024
Rheumatology
Hepatology
& Transplant
Dermatology
nrAxSpA,
superiority H2H vs.
adalimumab
primary Sjoegren's
syndrome
(VAY736 etc)
Early pipeline
Next generation
Bx in CSU
QGE031
(ligelizumab
Atopic dermatitis
(ZPL389)
Hidradenitis suppurativa
Early pipeline
vitiligo etc
Psoriasis
PsA , AS
Orphan
Early pipeline
Combo NASH
treatments
Auto-immune hepatitis
(VAY736)
NASH (LJN452)
(VAY785)
CSU
Tx portfolio
Cosentyx®
continues to deliver strong data
across indications
| Meet Novartis Management | May 31, 2017 | Investor Presentation29
Psoriasis AS & PsA Future Indications
• New analysis shows 21%
of patients did not relapse
more than one year after
treatment discontinuation1
• Early treatment correlated
with lower relapse rate1
• STEPIn trial evaluating
disease modification
potential
1. Lebwohl M et al. Long-term psoriasis control following secukinumab discontinuation indicated disease modification of moderate to severe psoriasis. Presented as a poster presentation at the 13th
Annual Maui Derm for Dermatologists 2017. 20-24th March 2017 2. In physical function, quality of life, and inflammation 3. Marzo-Ortega H, et al. Arthritis Care Res 2017. doi:
10.1002/acr.23233 4. Humira® is a registered trademark of AbbVie Ltd.
• MEASURE 2 data supports
sustained 2 year
improvements in signs and
symptoms2 of AS3
• H2H study in PsA enrolling
versus Humira®4
• Non-radiographic Axial
Spondyloarthritis Phase 3
trial on track for 2019 filing
Onco
CM
NS
I&D
Resp
Oph
Bios
ZPL389 once daily oral for atopic dermatitis
with strong Phase 2 data
| Meet Novartis Management | May 31, 2017 | Investor Presentation30
• Significant unmet need and lack of treatment
options
• ~17 m patients with mod-to severe AD in G73
– ~2/3 pediatric patients with ~20% mod-severe
– ~1/3 adult patients with ~70% mod-severe
• Potential first in class H4 receptor antagonist
• Blockbuster opportunity for pre-Biologics oral
with pediatric-suitable safety profile
• Compelling 8-week PoC data
– ZPL389 strong efficacy already at week 4 1,2
– Efficacy plateau not reached by week 8
ZPL389
Please note that figure above does not show intervals equidistantly
1. Werfel T. et al, Late breaking oral abstract (1346), EAACI 2016; 2. Beck LA et al, NEJM, 2014; 371; 130-139 3. Decision Resources report and internal analysis, Herd RM et al, 1996 and internal analysis
Onco
CM
NS
I&D
Resp
Oph
Bios
Novartis continues to lead the charge in
chronic spontaneous urticaria (CSU)
| Meet Novartis Management | May 31, 2017 | Investor Presentation31
• CSU: under-diagnosed & under-treated
• Xolair®1: building CSU market from zero
– Estimated 3 million patients in G72
– Prolonged itchy hives & angioedema with
QoL impact
• ~1/3 of patients with poor response to anti-
histamines
– Anti-IgE mAb (Xolair®) only approved
therapy
• Next generation mAb QGE031 with increased
potency
– Potential to deliver faster and more
complete remission in more patients
– Phase 2b data in 2017
– Phase 3 start in 2018
1. Novartis co-promotes Xolair® with Genentech in the US and shares a portion of operating income, but we do not record any US sales. Novartis records all sales of Xolair® outside the US 2.
Decision Resource Group
Onco
CM
NS
I&D
Resp
Oph
Bios
CSU
New Allergan collaboration strengthens
Novartis portfolio in NASH
| Meet Novartis Management | May 31, 2017 | Investor Presentation32
• LJN452 in Phase 2 in NASH (FLIGHT-FXR1)
• VAY7852 progressing in four Phase 2 studies
including NASH Fibrosis and NASH Cirrhosis
with collaborator Conatus
• Combining Allergan’s CVC with a Novartis
FXR agonist in Phase 2 studies
• Multiple assets in Phase 2 studies for other
liver indications
1. NCT02855164 2. Emricasan 3. Collaboration with Allergan
LJN452FXR
LIK066SGLT-1/2
VAY7852
Pan-caspase
CVC3
CCR2/5
Multimodal
Clinical
Pre-clinical
Early phase research on
anti-fibrotic mechanisms
Building a pipeline of NASH
combination therapies
Several assets progressing
NASH
Onco
CM
NS
I&D
Resp
Oph
Bios
Developing leading portfolio in moderate to
severe asthma
| Meet Novartis Management | May 31, 2017 | Investor Presentation33
Primary Care
Specialist Care
QMF149
QVM149
Xolair®
QAW039
QVM1491: inhaled combination therapy
• Once-daily triple combination for Ex-US
• Pivotal Phase 3 trials ongoing; planned filing 2019
Moderate/severe asthma portfolio
QAW039: potential first-in-class oral treatment for severe asthma
• CRTh2 antagonist; add-on to ICS/LABA or ICS/LABA/LAMA2
• Pivotal Phase 3 trials ongoing; planned filing 2019
Xolair® : first choice biologic for allergic asthma
• Xolair® with US3 approval for pediatric moderate to severe persistent allergic asthma
1. Fixed-dose combination of indacaterol, glycopyrronium and mometasone 2. LABA = long-acting β2-agonist; ICS = inhaled corticosteroid; LAMA = long-acting muscarinic antagonist
3. Novartis co-promotes Xolair® with Genentech in the US and share a portion of operating income, but we do not record any US sales. Novartis records all sales of Xolair® outside the US
Source: Novartis; Decision Resources, Epidemiology Database
Asthma
Potentially
eligible patients
7.0m (EU5+JP)
4.0m (G7)
2.2m (G7)
Onco
CM
NS
I&D
Resp
Oph
Bios
QAW039 - Phase 3 ongoing based on supportive
Phase 2 data; readout expected in 2019
| Meet Novartis Management | May 31, 2017 | Investor Presentation34
Fold reduction from baseline in eosinophil count
Geometric mean (95% CI); LOCF
0 6 12 18
0.5
1
2
4
8
Time (weeks)
QAW039Placebo
2.6xp=0.008
3.5xp=0.001
1.0xp =0.918
Treatment Wash out
Source: S Gonem, R Berair et al, Phase 2a randomized placebo-controlled trial of the oral prostaglandin D2 receptor (PD2/CRTH2) antagonist QAW039 in eosinophilic asthma, ERS congress 2014
Abbreviations: BID=twice daily; LOCF=last observation carried forward; QoL=quality of life
• Pre-clinical data demonstrate potential for
high potency
• QAW039 (225 mg BID) induced significant
suppression of sputum eosinophilia at week
12 and significant improved QoL
• Phase 3 pivotal trials ongoing; expected
readout 2019
QAW039
Onco
CM
NS
I&D
Resp
Oph
Bios
Ongoing Phase 3 studies to assess q12w/q8w
RTH258 vs. q8w aflibercept1
to readout in H1 2017
| Meet Novartis Management | May 31, 2017 | Investor Presentation35
Source: Holz F, EURetina 2015 oral presentation; NCT02307682; NCT02434328
1. In Phase 3 program, brolucizumab patients are selected for q12 week or q8 week maintenance dosing based on investigator masked assessment of disease activity. Aflibercept patients receive
q8 week maintenance dosing per the Eylea® label 2. Predicated by Phase 2 data (OSPREY trial) and designed to confirm in HAWK and HARRIER trials
nAMD Phase 3 trial designTrials designed to show differentiation vs. aflibercept
Brolucizumab 3 mg (q12w or q8w)*
Brolucizumab 6 mg (q12w or q8w)*
Aflibercept 2 mg (q8w)
92 weeks
(both trials)
Brolucizumab 6 mg (q12w or q8w)*
Aflibercept 2 mg (q8w)
N=1082
N=741
Potential differentiators / clinical
endpoints
• High proportion of patients successfully
maintained on q12w dosing regimen2
• Greater anatomical efficacy: greater
proportion of retinal fluid resolution, higher
central retinal fluid (CRT) reduction and
better CRT stability2
• Individualized treatment regimen assigned as
early as week 16 based on disease activity
assessment
• On track for read-out in H1 2017
Onco
CM
NS
I&D
Resp
Oph
Bios
RTH258
UNR844 (EV06) for potential treatment of
presbyopia
| Meet Novartis Management | May 31, 2017 | Investor Presentation36
82% of patients achieved DCNVA* of 20/40 with EV06
* DCNVA= distance corrected near visual acuity Encore Vision, Inc. press release, May 2016
UNR844
• UNR844 improved lens flexibility
through twice daily drops for 3
months
• Effects sustained over 6 months
• UNR844 was well tolerated, no
sight-related AEs or other safety
concerns
• Phase 2 dose finding now in
planning
Onco
CM
NS
I&D
Resp
Oph
Bios
Recently licensed ECF843 could be first-in-
class treatment for Dry Eye patients
| Meet Novartis Management | May 31, 2017 | Investor Presentation37
• ECF843, a recombinant form of human Lubricin, a naturally
occurring component of normal tears
• Potential first-in-class therapeutic to provide relief of dry eye
signs and symptoms within 4 weeks
• Phase 2 trial showed significant improvement in both signs
and symptoms of dry eye1
• Along with recent acquisition of UNR844 in presbyopia,
expands Front of the Eye pipeline
1. The Ocular Surface, Vol. 15, Issue 1, p77–87
Activity across all three layers of tear film
ECF843
Onco
CM
NS
I&D
Resp
Oph
Bios
Biosimilars: Two CHMP positive opinions in
April
| Meet Novartis Management | May 31, 2017 | Investor Presentation38
1. Launch delay due to litigation 2. As measured by ORR (Overall Response Rate) 3. MabThera® is a registered trademark of Roche
• CHMP positive opinion
• Confirmatory study
demonstrated equivalent
efficacy2 /safety to
MabThera®3
• Erelzi® approved in US1
• CHMP positive opinion
Etanercept Rituximab
Onco
CM
NS
I&D
Resp
Oph
Bios
Molecule Indication1 Originator2 Agency Filing
Etanercept Rheumatoid Arthritis FDA2015(approved)
✓
Etanercept Rheumatoid Arthritis EMA2015(CHMP positive opinion)
✓
Epoetin
subcutaneousAnemia EMA
2015(approved)
✓
Rituximab Non-Hodgkin’s Lymphoma EMA2016(CHMP positive opinion)
✓
Adalimumab Rheumatoid Arthritis FDA 2017
Adalimumab Rheumatoid Arthritis EMA2017(EU file accepted)
✓
Rituximab Non-Hodgkin’s Lymphoma FDA 2017
Infliximab Inflammatory Bowel Disease EMA2017(EU file accepted)
✓
Pegfilgrastim Neutropenia EMA 20173
Pegfilgrastim Neutropenia FDA 20183
Biosimilars filing milestones through 2018
| Meet Novartis Management | May 31, 2017 | Investor Presentation39
1. Main indication only 2. All trademarks are the property of the respective originator companies 3. Reflects re-filing as communicated in Q1
Onco
CM
NS
I&D
Resp
Oph
Bios
H1 2017 H2 2017
Regulatory
decisions
and opinions
Kisqali® (LEE011) HR+/HER2- adv. BC (US) ✓ LEE011 HR+/HER2- adv. BC (EU) =
Rydapt® (PKC412) AML and ASM (US) ✓ PKC412 AML and ASM (EU) =
Tafinlar®+Mekinist® BRAF+ NSCLC (EU) ✓ Tafinlar®+
Mekinist®BRAF+ NSCLC1 (US) =
GP2015 Etanercept BS2,3 (EU) ✓ GP2013 Rituximab BS2,3 (EU) ✓
Zykadia® ALK+ NSCLC (US) ✓ Zykadia® ALK+ NSCLC2 (EU) ✓
Submissions AMG 334 Migraine4 = GP2013 Non-Hodgkin’s Lymphoma (US) =
CTL019 Pediatric/young adult ALL
(US)1
✓ RLX030 Acute heart failure ✕
GP2017 Adalimumab BS3 (EU) ✓ ACZ885 CV risk reduction5 =
GP1111 Infliximab BS3 (EU) ✓ CTL019 DLBCL5 (US) =
GP2017 Adalimumab BS3 (US) =
LA-EP2006 Pegfilgrastim BS3 (EU) =
Major trial
readouts
RLX030 RELAX-AHF-2 (AHF) ✕ CTL019 JULIET (DLBCL) =
ACZ885 CANTOS (CVRR) =
RTH258 HARRIER, HAWK (nAMD) =
Strong progress on key 2017 milestones
| Meet Novartis Management | May 31, 2017 | Investor Presentation40
✓ Achieved ✕ Missed = On track
1. FDA Priority Review 2. Positive CHMP opinion, final EMA approval pending 3. Biosimilar 4. Submitted in the US and EU, Novartis has US co-commercialization rights
5. If results from Phase 3 trials are supportive
Summary
| Meet Novartis Management | May 31, 2017 | Investor Presentation41
• Focus on operational excellence and productivity with goal to achieve R&D
spend of approximately 20% of Innovative Medicines sales in the near
term. Digital development also a top priority
• Seven development franchises with broad, high value late stage pipeline
portfolio and expanding therapeutic breadth and depth
• Strong and diverse set of emerging Phase 2 assets from internal research
and recent external deals
Appendix
1. Secondary prevention of cardiovascular events
2. Diffuse large B-cell lymphoma
3. Multiple sclerosis
4. Severe aplastic anemia
5. Chronic myeloid leukemia
6. Long-acting release
7. Relapsing multiple sclerosis
8. Non-small cell lung cancer
9. Neovascular age-related macular degeneration
10. Multi-drug resistant
11. Breast cancer
12. Retinopathy of prematurity
13. Non-Hodgkin’s lymphoma
14. Non-radiographic axial spondyloarthritis
15. Preserved ejection fraction
16. Graft-versus-host disease
17. Neuroendocrine tumors
18. Chronic spontaneous urticaria / chronic idiopathic urticaria
19. Psoriatic arthritis head-to-head study versus adalimumab
20. Diabetic macular edema
21. Non-alcoholic steatohepatitis
22. Ankylosing spondylitis head-to-head study versus adalimumab
23. Acute myeloid leukemia
Planned filings 2017 to 2021
| Meet Novartis Management | May 31, 2017 | Investor Presentation43
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) EU filing, submitted in US
c) US filing, submitted in EU
d) US re-filing following withdrawal in Q1 2017, submitted in EU
e) US filing, submitted in EU with positive CHMP opinion
f) Lubris LLC transaction announced in April 2017
New molecule
New indication
New formulation
Biosimilars
Combination abbreviations:
fulv fulvestrant
tmx tamoxifen
gsn goserelin
NSAI Non-steroidal aromatase inhibitor
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
LA-EP2006 (pegfilgrastim, EU)
Chemotherapy-induced neutropenia and others (same as originator)
KAE609Malaria
CAD106Alzheimer’s disease
LIK066Weight loss
CJM112Immune disorders
ABL001CML5 3rd line
EMA401Neuropathic pain
CNP520Alzheimer’s disease
BYM338Hip fracture
QGE031CSU/CIU18
BYM338Sarcopenia
PIM447Hematologic tumors
VAY736Primary Sjoegren’s syndrome
Entresto®
Post-acute myocardial infarction
QAW039Atopic dermatitis
RTH258DME20
CTL019b
Pediatric/young adult acute lymphoblastic leukemia
Tasigna®c
CML5 treatment free remission
LCI699Cushing’s disease
BAF312RMS7
QAW039Asthma
Entresto®
Heart failure (PEF)15
Lucentis®
ROP12
INC280 NSCLC8
KAF156 Malaria
QVM149Asthma
QMF149Asthma
Kisqali® + fulvHR+, HER2 (-) postmenopausal
adv. BC11 1st/2nd line
Kisqali® + tmx + gsn/or NSAI + gsnHR+, HER2 (-) premenopausal
adv. BC11 1st line
Zykadia®
ALK+ adv. NSCLC8
(Brain metastases)
Cosentyx®
nrAxSpA14
BYL719 + fulvHR+, HER2 (-) postmenopausal
adv. BC11 2nd line
OMB157RMS7
ACZ885Sec. prev. CV events1
CTL019DLBCL2
Arzerra®
NHL13 (refractory)
Tafinlar® + Mekinist®BRAF V600+ melanoma (adjuvant)
RTH258nAMD9
2021201920182017 2020
Jakavi®Acute GVHD16
FTY720Pediatric MS3
LJN452NASH21
GP2017 (adalimumab, US/EU)
Arthritides, plaque psoriasis and others (same as originator)
GP1111 (infliximab, EU)Autoimmune diseases (same as
originator)
GP2013e (rituximab, US)Follicular lymphoma, DLBCL2 and
others (same as originator)
QBW251Cystic fibrosis
AMG 334a
Migraine
Kisqali®
HR+, HER2 (-) BC11 (adjuvant, intermediate risk)
Cosentyx®
PsA H2H19
Cosentyx®
AS H2H22
LAM320MDR10 tuberculosis
Promacta®/Revolade®
SAA4 1st line
ZPL389Atopic dermatitis
PKC412AML23 (FLT3 wild type)
UNR844Presbyopia
SEG101Sickle cell disease
LA-EP2006 (pegfilgrastim, US)
Chemotherapy-induced neutropenia and others (same as originator)
GP2018 (infliximab, US)Autoimmune diseases (same as
originator)
INC280NSCLC8 (EGFRm)
Jakavi®Chronic GVHD16
Kisqali®HR+, HER2 (-) BC11 (adjuvant,
high risk)
Signifor® LAR6,d
Cushing’s diseasePDR001 + Taf/Mek
Metastatic BRAF V600+ melanoma
ECF843f
Dry eye
PDR001NET17
Pipeline of key projects in confirmatory
development
| Meet Novartis Management | May 31, 2017 | Investor Presentation44
a) Lubris LLC transaction announced in April 2017.
b) In collaboration with Amgen; companies to co-commercialize in the US,
Novartis to have AMG 334 exclusive rights in rest of world excluding Japan.
c) Submitted in US.
d) Approved in US, submitted in EU.
e) Approved in EU, submitted in US.
f) Submitted in EU.
g) Submitted in EU, US filing withdrawal in Q1 2017 with refiling in 2017.
h) Positive CHMP opinion.
New molecule
New indication
New formulation
Biosimilars
1. Chronic myeloid leukemia
2. Non-small cell lung cancer
3. Non-alcoholic steatohepatitis
4. Chronic spontaneous urticaria / chronic idiopathic urticaria
5. Neuroendocrine tumors
6. Relapsing multiple sclerosis
7. Breast cancer
8. Neovascular age-related macular degeneration
9. Secondary prevention of cardiovascular events
10. Non-Hodgkin’s lymphoma
11. Non-radiographic axial spondyloarthritis
12. Psoriatic arthritis head-to-head study versus adalimumab
13. Ankylosing spondylitis head-to-head study versus adalimumab
14. Diffuse large B-cell lymphoma
15. Preserved ejection fraction
16. Multiple sclerosis
17. Graft-versus-host disease
18. Multi-drug resistant
19. Retinopathy of prematurity
20. Acute myeloid leukemia
21. Severe aplastic anemia
22. Diabetic macular edema
23. Acute lymphoblastic leukemia
24. Advanced systemic mastocytosis
25. Long-acting release
PDR001NET5
SEG101Sickle cell disease
Post-PoC Phase III / Pivotal In Registration
ACZ885Sec. prev. CV events9
BAF312 RMS6
FTY720 Pediatric MS16
Lucentis®
ROP19
QAW039Asthma
RTH258nAMD8
Cosentyx®
nrAxSpA11
QMF149Asthma
QVM149Asthma
AMG 334b
MigraineOMB157
RMS6
Entresto®
Heart failure (PEF)15
RTH258DME22
LCI699Cushing’s disease
Kisqali® + ltzd
HR+, HER2(-) postmenopausal adv. BC7 1st line
PKC412AML20
Tafinlar® + Mekinist®e
BRAF V600+ NSCLC2
Arzerra®
NHL10 (refractory)
Kisqali® + tmx + gsn/or NSAI +
gsnHR+, HER2(-) premenopausal
adv. BC7 1st line
Kisqali® + fulvHR+, HER2(-) postmenopausal
adv. BC7 1st/2nd line
Tafinlar® + Mekinist®BRAF V600+ melanoma (adjuvant)
PKC412ASM24
Signifor® LAR25,g
Cushing’s disease
Zykadia®
ALK+ adv. NSCLC2
(Brain metastases)
Zykadia®
ALK+ adv. NSCLC2
(1st line, treatment naïve)
BYL719 + fulvHR+, HER2 (-) postmenopausal
adv. BC7 2nd line
Entresto®
Post-acute myocardial infarction
CTL019c
Pediatric/young adult ALL23
CTL019DLBCL14
GP2013 (rituximab, US)Follicular lymphoma, DLBCL14 and
others (same as originator)
GP2017 (adalimumab, US/EU)
Arthritides, plaque psoriasis and others (same as originator)
GP1111 (infliximab, EU)Autoimmune diseases (same as
originator)
Tasigna®f
CML1 treatment free remission
GP2015h (etanercept, EU)Arthritides, plaque psoriasis and
others (same as originator)
GP2013h (rituximab, EU)Follicular lymphoma, DLBCL14 and
others (same as originator)
VAY736Primary Sjoegren’s syndrome
KAF156Malaria
QAW039Atopic dermatitis
CAD106Alzheimer’s disease
KAE609Malaria
BYM338 Hip fracture
LJN452NASH3
BYM338 Sarcopenia
EMA401Neuropathic pain
QGE031CSU/CIU4
INC280 NSCLC2
ABL001CML1 3rd line
PIM447Hematologic tumors
Jakavi®Acute GVHD17
CJM112Immune disorders
CNP520Alzheimer’s disease
LIK066Weight loss
QBW251Cystic fibrosis
Cosentyx®
PsA H2H12
Cosentyx®
AS H2H13
ZPL389Atopic dermatitis
LAM320MDR18 tuberculosis
Kisqali®
HR+, HER2(-) BC7 (adjuvant, intermediate risk)
Promacta®/Revolade®
SAA21 1st line
PKC412AML20 (FLT3 wild type)
UNR844Presbyopia
LA-EP2006 (pegfilgrastim, US/EU)
Chemotherapy-induced neutropenia and others (same as originator)
GP2018 (infliximab, US)Autoimmune diseases (same as
originator)
INC280NSCLC2 (EGFRm)
Jakavi®Chronic GVHD17
Kisqali®
HR+, HER2(-) BC7 (adjuvant, high risk)
PDR001 + Taf/MekMetastatic BRAF V600+ melanoma
ECF843a
Dry eye
Combination abbreviations:
fulv fulvestrant
ltz letrozole
tmx tamoxifen
gsn goserelin
NSAI Non-steroidal aromatase inhibitor
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
Key definitions and trademarks
| Meet Novartis Management | May 31, 2017 | Investor Presentation45
This presentation contains several important words or phrases that we define as follows:
ADHF: Acute decompensated heart failure
AE: Adverse Event
ALK: Anaplastic lymphoma kinase
ALL: Acute lymphatic leukemia
AMD: Age-Related Macular Degeneration
AMI: Acute myocardial infection
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
ARNI: Antiogensin receptor neprilysin inhibitor
AS: Ankylosing Spondylitis
BTD: Breakthrough therapy designation
CGRP: Calcitonin gene-related peptide
cITP: Chronic immune thrombocytopenia
CM: Chronic migraine
CML: Chronic myeloid leukemia
COPD: Chronic Obstructive Pulmonary Disease
CR: complete remission
CRC: Colorectal Cancer
CRi: Complete remission with incomplete blood count recovery
CSU / CIU: Chronic spontaneous urticaria / Chronic idiopathic urticaria
CVRR: Cardiovascular risk reduction
DLBCL: Diffuse large B-cell lymphoma
DMC: Data monitoring committee
EDSS: Expanded Disability Status Scale
EF: ejection fraction
EM: Episodic migraine
GvHD: graft vs. host disease
HbA1C: Glycated hemoglobin
HCC: Hepatocellular carcinoma
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
MF: Myelofibrosis
MI: Myocardial infarction
MS: Multiple sclerosis
NASH: Non-Alcoholic Steatohepatitis
NET: Neuroendocrine tumor
New assets: Assets acquired in the GSK transaction which closed on March 2, 2015
NSAI: Nonsteroidal aromatase inhibitor
NSCLC: Non-small cell lung cancer
NTD: New Therapeutic Drug
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: Psoriatic arthritis
PsO: Psoriasis
PV: Polycythemia vera
PY: Prior year
QoL: Quality of Life
RCC: Renal cell cancer
ROP: Retinopathy of prematurity
r/r ALL: relapsed/refractory acute lymphoblastic leukemia
RRMS: relapsing-remitting multiple sclerosis
SAA: Severe aplastic anemia
scFv: Single chain variable fragment
SCPC: Sickle cell pain crisis
SM: Systemic mastocytosis
SpA: Spondyloarthritis
SPMS: Secondary progressive multiple sclerosis
Trademarks
Aubagio® and Lemtrada® are registered trademarks of Genzyme Corporation
Enbrel® is a registered trademark of Amgen Inc.
Humira® is a registered trademark of AbbVie Ltd.
MabThera® is a registered trademark of Roche, Ltd.
Remicade® and Stelara® are registered trademarks of Janssen Biotech, Inc.
and Simponi® are registered trademarks of Johnson & Johnson
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