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| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 2
This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by
words such as “potential,” “expected,” “will,” “planned,” “pipeline,” “outlook,” or similar expressions, or by express or implied discussions regarding potential new products, potential
new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding the potential outcome, or financial or
other impact on Novartis, of the proposed divestiture of certain portions of our Sandoz Division business in the US; or regarding the potential impact of the completion of the up to
USD 5 billion share buyback; or regarding potential future sales or earnings of the Group or any of its divisions or potential shareholder returns; or by discussions of strategy, plans,
expectations or intentions. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant
known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary
materially from those set forth in the forward-looking statements. You should not place undue reliance on these statements. In particular, our expectations could be affected by,
among other things: global trends toward healthcare cost containment, including ongoing government, payor and general public pricing and reimbursement pressures and
requirements for increased pricing transparency; regulatory actions or delays or government regulation generally, including potential regulatory actions or delays with respect to the
proposed transactions or the development of the products described in this presentation; the potential that the proposed divestiture of certain portions of our Sandoz Division
business in the US may not be completed in the expected time frame, or at all; the potential that the strategic benefits, synergies or opportunities expected from the proposed
divestiture of certain portions of our Sandoz Division business in the US, and other transactions described, may not be realized or may be more difficult or take longer to realize than
expected; the inherent uncertainties involved in predicting shareholder returns; the uncertainties inherent in the research and development of new healthcare products, including
clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the
impact on Novartis of the loss of patent protection and exclusivity on key products that commenced in prior years and will continue this year; safety, quality or manufacturing issues;
uncertainties involved in the development or adoption of potentially transformational technologies and business models; uncertainties regarding actual or potential legal proceedings,
including, among others, product liability litigation, disputes and litigation with business partners or business collaborators, government investigations generally, litigation and
investigations regarding sales and marketing practices, and intellectual property disputes; our performance on environmental, social and governance measures; general political,
economic and trade conditions, including uncertainties regarding the effects of ongoing instability in various parts of the world; uncertainties regarding future global exchange rates;
uncertainties regarding future demand for our products; uncertainties regarding potential significant breaches of data security or data privacy, or disruptions of our information
technology systems; and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the
information in this presentation as of this date and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or
otherwise.
Eylea® is a registered trademark of Regeneron Pharmaceuticals, Inc.
Strong Q3 with double digit top and bottom line growth, margin expansion and major innovation milestones
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 4
Q3 operational performanceContinuing operations1 growth vs. PY in % cc 2
Innovation performance
Sales
+13%
Sales & Core OpInc guidance increased
Core OpInc
+18%
Core margin
+1.4%
pts
OS – overall survival RMS – relapsing forms of multiple sclerosis 1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz
(including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be
found on page 56 of the Condensed Interim Financial Report 3. Study narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women and patients with lower ejection fraction
Launched in US
Ofatumumab Compelling efficacy in RMS
Met primary endpoints in nr-axSpA
Achieved OS in 2nd Ph3 study
Clinically important benefit in HFpEF
sub-groups3
QVM149 / QMF149 Positive Ph3 results in asthma
Key growth drivers represent 28% of 9M Innovative Medicines sales and contributed +8%pts to Novartis sales growth
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 5
8%
9%
1%2%
Growth drivers CC growth
9M 2019
Gx erosion
volume
Others
28%
Key growth drivers sales in USD m
(% of Innovative Medicines sales)
9M 20179M 2016 9M 20199M 2018
Promacta®
Piqray®
Zolgensma®
Cosentyx®
Entresto® Xolair®
Tafinlar®+Mekinist®
Lutathera®
Kisqali®
Kymriah®
Xiidra®
10%
16%
21%
Growth contribution to continuing operations sales
(% points)
Strong sales performance of key growth drivers
NM – not meaningful
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 6
SalesUSD Million
Growth vs. PYUSD Million
Growth vs. PYcc
SalesUSD Million
Growth vs. PYcc
937 27% 2,586 30%
160 nm 175 nm
430 61% 1,208 75%
102 nm 102 nm
380 31% 1,036 26%
119 116% 334 nm
79 nm 182 nm
345 22% 982 22%
123 76% 325 92%
299 22% 870 20%
43 nm 49 nm
Q3 9M
Key growth drivers
Lutathera®
2019 2019
85
187
160
159
102
63
59
54
51
44
43
Cosentyx® sales driven by strong demand across indications and geographies
Cosentyx® revenues USD m, % cc
459601
291
336
Q3 2018
US
Ex-US
Q3 2019
750
937
+27%
Strong momentum in growing US dermatology
market1
Dermatology market TRx +17% YoY, driven by novel agents
Cosentyx® TRx +32% YoY, despite intensifying competition
Leading expansion in growing US SpA market2
SpA market TRx +15% YoY
Cosentyx® major contributor to market growth, TRx +36% YoY
Positive CHMP opinion for up-titration to 300mg in AS
PREVENT met primary endpoints at 16 and 52 weeks
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 7
1. IQVIA National Prescription Audit for Dermatology, WE 09/27/2019 2. IQVIA National Prescription Audit for Rheumatology, WE 09/27/2019. Rx for SpA indications. Market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz® and
Cosentyx®
1.7m
PsA AS nr-axSpA
3.2m
1.7m
19-27% 12-21% 4-8%Biologics
penetration3
Nr-axSpA indication builds Cosentyx® leading position in SpA, potentially doubling patient population in axSpA1
Significant potential for growth
across SpA populations (US & EU5)2
Well-positioned for further growth
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 8
Diagnosed
Undiagnosed
Biologics
treated
Please see appendix for references All trademarks are the property of their respective owners HS – Hidradenitis Suppurativa PsO – Psoriasis JIA – Juvenile Idiopathic Arthritis GCA – Giant Cell Arthritis
PREVENT Could add nr-axSpA to label; submissions
ongoing
MAXIMISE Proved for the first-time benefits in axial
manifestations of PsA
EXCEED Assessing benefits in joints vs. Humira® –
expected H1 2020
Other clinical
trials
Including HS, pediatric PsO, pediatric JIA
and Ph2 GCA study, on track
Entresto® solidifying position as standard of care in HFrEFwith >USD 1.2bn sales YTD
Solid QoQ demand growth US and ex-US
PROVE-HF and EVALUATE-HF provide mechanistic support for Entresto® efficacy1
FDA approved pediatric indication in Q4
HFrEF – heart failure with reduced ejection fraction 1. While several structural and functional echocardiographic measures versus enalapril showed improvement, EVALUATE didn’t meet the primary endpoint of change from baseline in aortic
characteristic impedance at 12 weeks
Entresto® revenues USD m, % cc
151220
120
210
Q3 2018 Q3 2019
Ex-US
US
271
430
+61%
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 9
01
/18
30
03
/18
11
/17
05
/18
07
/18
09
/18
05
/19
07
/19
01
/19
03
/19
11
/18
11
/19
0
25
35
20
40
45
09
/19
Entresto® weekly US TRx2017-19, thousands
+51%
Q3
2018
Q3
2019
PARAGON-HF: clinically important effect against active comparator supports expansion of addressable population
Regulatory and payer discussions ongoing. US submission for inclusion of data in label Q4 2019
Largest HFpEF outcomes trial on back of approved
HFrEF indication
Despite narrow miss on primary endpoint, pre-defined
secondary endpoints and subgroup analyses
supportive of benefit
Greater treatment benefit in HFpEF patients with
ejection fraction below median, and in women
HFrEF – heart failure with reduced ejection fraction HFpEF – heart failure with preserved ejection fraction
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 10
Total HF hospitalizations and CV deathN= 4796
Rate ratio 0.87 (95% CI 0.75, 1.01)
p = 0.059
Sacubitril/valsartan
Valsartan
Broad access
Zolgensma® off to a strong start with sales USD 175m since launch
Solid demand Broad patient profile
Even distribution across
age, SMA types and
incident and prevalent
populations
>50% switches from
nusinersen
>50 treating institutions
across US - majority of
leading academic centers
of excellence having
prescribed since launch
~90% commercial
patients and ~30%
Medicaid patients
covered
>99% final approval rate
for on label patients
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 11
Zolgensma® key opportunities to build on US launch momentum
Newborn screening (US): 30% of newborns
screened, expected to reach 70% end of 2020
~2/3 of incident patients treated in states with
newborn screening (vs. ~1/4 in states without)
Medicaid policies: coming in place rapidly
including key states like Florida, New Jersey
and Michigan
New interim STRONG data: reinforcing
efficacy in SMA type II (IT, +5.9pts HFMSE
score, patients 2-5 years old)
USAwaiting FDA feedback on IT filing
approach
EU CHMP opinion anticipated in Q1 2020
Japan Decision anticipated in 1H 2020
Early
accessIncluding France, Portugal, Germany
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 12
Opportunities Regulatory
IT – intrathecal
Strong start to Beovu® US launch, highly competitive label Similar efficacy with fewer injections, supported by visual & anatomical measures
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 13
Longer treatment intervals achievable
without compromising efficacy
Only q12w without compromise to efficacy*
Only treatment with q12w recommended
immediately after loading
Flexibility from q8w to q12w
All trademarks are the property of their respective owners Source: BEOVU [prescribing information] East Hanover, NJ. Novartis: 2019, Dugel P, et al. HAWK and HARRIER: Ph3, multicenter, randomized, double-masked trials of brolucizumab
for neovascular age-related macular degeneration [published online ahead of print]. Ophthalmology. 2019. nAMD = neovascular age related macular degeneration; VEGF = Vascular Endothelial Growth Factor; q12w = every 12 weeks
Supportive label language on
visual and anatomical measures**
Recognition of treatment decisions
according to visual and anatomical
measures of disease activity
Reductions in central retinal subfield
thickness across all treatment arms
Overall safety profile in
line with comparator
*Eylea® prescribing information states “Although not as effective as the recommended every 8-week dosing regimen, patients may also be treated with one
dose every 12 weeks after one year of effective therapy. Patients should be assessed regularly”. Lucentis® prescribing information states: “Although not as
effective, patients may also be treated with one dose every 3 months after 4 monthly doses. Patients should be assessed regularly.”
**According to the FDA label, the utility of these measures has not been established
Most common adverse
reactions (≥5%): vision
blurred, cataract,
conjunctival hemorrhage,
eye pain, vitreous floaters
Beovu® comprehensive clinical trial programs ongoing including potential new indications
DME RVO PDRAMD
A head-to-head superiority study
vs. aflibercept evaluating treatment
interval duration in an identical
Treat-to-Control regimenPhase 3 head-to-head non-inferiority
studies vs. aflibercept to support
global registration
CONDOR
Phase 3 non-inferiority study vs.
aflibercept to support global
registration
20212 ≥20232 ≥20232,32019-20201
A head-to-head non-inferiority study
vs. aflibercept evaluating the efficacy
and safety of q4w treatment
1. First expected approval date 2. first planned submission 3. Study name pending approval AMD = Age related macular degeneration, PCV = Polypoidal Choroidal Vasculopathy, DME = Diabetic Macular Edema, RVO = Retinal vein
occlusion, PDR = Proliferative diabetic retinopathy; q4w = every 4 weeks
PCV studyRandomized, open-label, multicenter
study assessing the safety & efficacy
of two different dosing regimens of
brolucizumab 6 mg in patients with
symptomatic macular PCV
A head-to-head non-inferiority study
vs. aflibercept evaluating the efficacy
and safety of q4w treatment
Phase 3 non-inferiority studies of
brolucizumab q12w/ q8w vs.
aflibercept to support global
registration
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 14
Ofatumumab – high efficacy disease-modifying therapy with the potential to be used early and broadly
1. CDW, confirmed disability worsening and CDP, confirmed disability progression are interchangeable terms, defined by an increase ≥1.5 EDSS points for patients with baseline EDSS of 0, increase of ≥1.0 EDSS points patients with baseline
EDSS of 1.0-5.0 and increase of t ≥0.5 EDSS points for patients with baseline EDSS of 5.5 2. NfL levels at month three measured as adjusted geometric mean levels and difference is geometric mean ratio (GMR)
Strong efficacy results vs. teriflunomide
(ASCLEPIOS I&II)
Superior efficacy for relapses, MRI activity
Substantial reductions in 3- and 6-month
disability progression1
Lower levels of NfL2
Safety profile – no significant signals
concerning infections/ malignancies
Supporting highly competitive
product profile
High efficacy
Favorable safety profile
Convenient subcutaneous option
No need for infusion center
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 15
Initiating worldwide regulatory submissions starting Q4 2019
Market feedback on Mayzent® encouraging – acceleration of diagnosis and onboarding needed for sales ramp-up
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 16
Appreciated by customers
and payers
Supportive label and
additional unique data
90% willingness to prescribe4
>2600 SFs, 2/3 in aSPMS
>150m lives with preferred or
unrestricted access
Accelerating diagnosis and
onboarding
Shift focus from relapses to
progression
Drive urgency to treat
Simplify onboarding
Accelerate time to paid Rx
Active SPMS1 (EDSS 3-6)
Reduces disease progression
Safety and tolerability
Cognitive processing speed2
4 year delay to wheelchair3
Expect CHMP opinion in late Q4 2019
Please see appendix for references aSPMS – active Secondary progressive multiple sclerosis EDSS – Expanded Disability Status Scale SF – Start Form
FevipiprantLUSTER to determine benefit in moderate-severe asthma. ZEAL confirmed clean safety profile but no significant lung function improvement in less severe population
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 17
Comprehensive Ph3 program to define full potential
Exacerbation
52 weeks
GINA 4/5
Core
registration
Results expected Q1 2020
Safety
Up to 3 years
GINA 3/4/5
Core
registration
Interim results expected Q1
2020
Lung function
12 weeks
GINA 3/4
Supportive
studies
No significant improvement of
FEV1 on top of ICS ± LABA1,2
Clean safety profile confirmed3
US patient numbers (≥ 12 years)
Ph3 asthma study populations
ZEAL
1.4m
patients5
BASE CASE
LUSTER: high eosinophils4
1.5m patients
SevereModerate
ASTHMA SEVERITY
EO
SIN
OP
HIL
S4
HIGH
LOW
FURTHER POTENTIAL
LUSTER: low eosinophils
1.5m patients
GINA – Global Initiative for Asthma
1. In combination with inhaled standard of care – predominantly low-medium dose ICS+LABA 2. Detailed analysis of the ZEAL1 & 2 data is ongoing & results to be communicated in H1 2020 3. Based on ZEAL 1 & 2 and a dedicated
thorough QT study which demonstrated absence of a QTcF signal with fevipiprant vs. placebo (Novartis data on file) 4. High eosinophils defined as ≥ 250 cells/µL 5. patients with unresolved symptoms and exacerbations on GINA 3 regimes
Piqray® launch off to strong start with USD 49m YTD sales
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 18
aBC – advanced breast cancer TNBC – triple negative breast cancer HR+/HER2- – hormone receptor positive, human epidermal growth factor receptor-2 negative 1. Alpelisib (BYL719) also being explored in PIK3CA-related overgrowth
spectrum (PROS), a group of rare disorders driven by mutations in the PIK3CA gene
Strong launch in USUSD million
Building foundation for global launch
First and only therapy for aBC patients with PIK3CA mutation
Approximately 40% of the HR+/HER2- aBC population have
a mutation in PIK3CA
Good initial uptake of PIK3CA testing in the US
Foundation Medicine PIK3CA CDx tissue approval
anticipated Q4 2019
CHMP opinion expected H1 2020
Potential to expand1 beyond aBC with programs starting in
HER2+, TNBC, head & neck, ovarian cancer6
43
Q3 2019Q2 2019
Kisqali® is the only CDK4/6 inhibitor to demonstrate consistently superior overall survival (OS) in two Ph3 trials
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 19
MONALEESA-3Post-menopausal
The only CDK4/6 with two positive readouts OS benefit proven across multiple populations
and combination partners
Kisqali® plus fulvestrant achieved statistically significant OS
benefit in postmenopausal patients (MONALEESA-3)
Kisqali® plus endocrine therapy achieved statistically significant
OS benefit in pre- and peri-menopausal patients
(MONALEESA-7)
Across both pivotal Ph3 trials, Kisqali demonstrated 28% / 29%
reduction in the risk of death
Comprehensive OS evidence expected to further differentiate
Kisqali® within CDK4/6 class
MONALEESA-7Pre-menopausal
2019 expected pipeline milestones
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 20
H1 2019 H2 2019
Regulatory
decisions and
opinions
Mayzent®1 SPMS (US) ✓ BYL719 (Piqray®) HR+ Breast Cancer (US) ✓2
Kymriah® Ped / Young Adult r/r ALL (JP) ✓ Beovu® nAMD (US) ✓
Kymriah® r/r DLBCL (JP) ✓ Lucentis® RoP (EU / JP) ✓ (EU)
Promacta® Severe aplastic anaemia (EU) ✕ Lucentis® Diabetic Retinopathy (EU) ✓3
Zolgensma® SMA type I (US) ✓ Mayzent®1 SPMS (EU / JP)
Zolgensma® SMA type I (EU / JP)EU Q1 2020
JP H1 2020Xolair® Pollinosis (JP)
Major
expected
submissions
Brolucizumab (RTH258) nAMD (US / EU / JP) ✓ Cosentyx® nr-AxSpA (US / EU / JP) ✓ (EU)
Crizanlizumab (SEG101) Sickle Cell Disease (US / EU) ✓ Entresto® HF-rEF (JP) ✓Mayzent®1 SPMS (JP) ✓ Entresto® HF-pEF (US / EU)
INC280 NSCLC (US / JP)
Ofatumumab (OMB157) Relapsing MS (US / EU)
PDR001 (combination
with Tafinlar®+Mekinist®) Metastatic Melanoma (US / EU) H1 2020
QVM149 Asthma (EU / JP) ✓
Major
expected trial
readouts
AVXS-101 IT SMA type II IT Formulation ✓ Cosentyx® nr-AxSpA ✓
Zolgensma® SMA type I / II /
presymptomatic✓ Entresto® HF-pEF (✓)4
Fevipiprant (QAW039) Asthma5
Ofatumumab (OMB157) Relapsing MS ✓PDR001 (combination
with Tafinlar® + Mekinist®) Metastatic melanoma
✓ Achieved* ✕ Missed*
*Represents achieving on-time readout of the data, irrespective of trial outcome 1. The name Mayzent® has now been fully approved by the FDA and so has the product. But the name has only been provisionally approved in Europe, and the
product has not yet been approved there 2. Piqray® FDA approval achieved in H1 2019. 3. CHMP positive opinion received 4. Study narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women
and patients with lower ejection fraction 5. ZEAL 1/2 readouts complete (did not meet the primary efficacy endpoint of FEV1 improvement in moderate asthmatic patients), LUSTER 1/2 readouts expected Q1 2020
2020 catalysts expected to maintain 2019 momentum
Potential
catalysts
Selected
examples
Key
approvals1
Ofatumumab (OMB157)
Relapsing MS
Crizanlizumab (SEG101)
Sickle cell disease
Capmatinib (INC280)
NSCLC
QVM149
Asthma
Cosentyx®
nrAxSpA
Key
submissions2
Fevipiprant (QAW039)
Asthma
177Lu-PSMA-617
mCRPC
AVXS-101 IT
SMA type 2/3
PDR001 combo4
Metastatic melanoma
Alpelisib (BYL719)
PIK3CA-related overgrowth
spectrum
Key
readouts3
Phase 3 Select Phase 2
177Lu-PSMA-617
mCRPC
ABL001 (3rd line)
Chronic myeloid leukemia
Entresto®
Post-acute MI
Beovu®
DME
Kisqali®
Breast cancer (MONALEESA-2 OS)
Jakavi®
Chronic GvHD
Tropifexor (LJN452)
NASH
LOU064
Autoimmune diseases
LNP023
Renal diseases and
paroxysmal nocturnal
hemoglobinuria
1. First approval in any market 2. First submission in any market 3. Readouts enabling submission, label change or pivotal trial initiation 4. In combination with Tafinlar® and Mekinist®
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 21
% USD % cc % USD % cc
Net Sales 12,172 10 13 35,042 5 9
Core Operating income 3,748 15 18 10,650 13 18
Operating income 2,358 5 9 7,263 3 10
Net Income 2,041 8 12 6,018 -48 -45
Core EPS (USD) 1.41 16 19 3.97 12 17
EPS (USD) 0.90 11 14 2.62 -47 -44
Free Cash Flow 3,968 26 9,449 13
Change vs. PYContinuing operations
1
USD million
Q3
2019
9M
2019
Change vs. PY
Summary of Q3 and 9M 2019 continuing operations financial results
1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well
as the continuing corporate functions 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 56 of the Condensed Interim Financial Report
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 23
2
2
2
Net sales
change vs. PY
Core operating
income
change vs. PY Core margin
Core margin
change vs. PY Core margin
Core margin
change vs. PY
(in % cc) (in % cc) (%) (%pts cc) (%) (%pts cc)
Innovative Medicines 15 16 34.1 0.4 34.3 2.2
Sandoz 5 18 24.8 2.8 21.8 1.6
Continuing Operations 13 18 30.8 1.4 30.4 2.4
Q3 2019 9M 2019
Continuing operations delivering core margin expansion of 1.4%pts cc vs. PY
1
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 24
2
2
2
2
2
1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well
as the continuing corporate functions 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 56 of the Condensed Interim Financial Report
New focused medicines company 2019 FY guidance: sales and core operating income revised upwards
Barring unforeseen events (in cc)
New focused medicines company | full year guidanceExcl. Alcon and Sandoz proposed US portfolio sale to Aurobindo1 from both 2018 and 2019; growth vs. PY in cc
Sales revised upwards expected to grow high single digit
• IM Division revised upwards to grow high single digit to low double digit
• Sandoz revised upwards to grow low single digit
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Key Assumption: All guidance includes forecast assumption that no Gilenya® generics enter in 2019 in US.
1. Novartis continues to expect the previously-announced divestment of the Sandoz US oral solids and dermatology portfolio to be completed in the coming months, pending closing conditions including regulatory approvals. 2018 FY Sales and
Core OpInc of the Sandoz US Oral solids and Dermatology businesses were approximately USD 1.2bn and 0.3bn, respectively.
25
Core operating income revised upwards expected to grow mid to high teens
Core OpInc growth with continued strong underlying momentum; potential generic headwinds in Q4 2019
26
Key drivers of continuing operations core operating incomevs. PY (cc)
9M 2019
+ Innovative Medicines growth drivers
including Cosentyx®, Entresto® and
Zolgensma®
+ Productivity
+ Valsartan competitor supply
shortage
+ Innovative Medicines growth drivers,
launches uptake1
+ Productivity
− Potential increased Gx erosion2
− Potential resolution of valsartan
competitor supply shortage
− Increased investments in pre-launch
and launches
Q4 2019
1. Including Zolgensma®, Mayzent®, Aimovig®, Piqray® 2. Mainly Afinitor ®, Exjade® / Jadenu® and Ophthalmology brands
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Currency impact vs. PY%pts, assuming mid-October exchange rates prevail in 2019 and 2020
Expected currency impact for full year 2019 and 2020
SimulationActual
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
FX impact on
Net sales
FX impact on
Core operating income
1
-5 -4 -3 -3-1
-9-6
-3-5
-1
FYQ3FY Q1 FYQ2 FYQ4 Q1 Q2 Q3 Q4 FY FY
0
-2 to -3-1 to -2
2018 2019
27
2020 2018 2019 2020
Conclusion
Increased full year sales and core operating income guidance
Excellent Q3 performance with double digit increases in top and bottom line,
and margin expansion
Zolgensma® and Piqray® launches off to a strong start
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 29
Continuing innovation, notably ofatumumab’s remarkable efficacy in RMS
Ofatumumab – high efficacy on key measures of disease activity
32
End point OMB. TER. RRRc p value OMB. TER. RRRc p value
Annualized relapse rate 0.11 0.22 50.5% <0.001 0.1 0.25 58.5% <0.001
Gd+ T1 lesions per scan 0.01 0.45 97.5% <0.001 0.03 0.51 93.8% <0.001
New or enlarging T2 lesions per year 0.72 4.00 82.0% <0.001 0.64 4.15 84.5% <0.001
Serum NfL levels at month 3a 8.8 9.41 7%c 0.011 8.92 10.02 11%c <0.001
Brain volume lossb -0.28 -0.35 0.07 0.116 -0.29 -0.35 0.07 0.129
End point OMB. TER. RRRc p value
3-month CDW* 10.9% 15.0% 34.4% 0.002
6-month CDW* 8.1% 12.0% 32.5% 0.012
6-month CDI 11.0% 8.1% -35.2% 0.094
ASCLEPIOS I ASCLEPIOS II
PRE-SPECIFICED COMBINED ANALYSIS OF ASCLEPIOS I & II
*CDW, confirmed disability worsening and CDP, confirmed disability progression are interchangeable terms, defined by an increase ≥1.5 EDSS points for patients with baseline EDSS of 0, increase of ≥1.0 EDSS points patients with baseline
EDSS of 1.0-5.0 and increase of t ≥0.5 EDSS points for patients with baseline EDSS of 5.5 a NfL levels at month three measured as adjusted geometric mean levels and difference is geometric mean ratio (GMR) b Brain volume loss is
measured as difference in slope between 12 and 24 months, difference measured as adjusted mean difference in slope c RRR is relative risk reduction and in cases where it is different- highlighted in footnote; OMB = Ofatumumab; TER =
Teriflunomide; CDI = Confirmed Disability Improvement.
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Net debt increased by USD 3.2bn driven by M&A transactions and share buybacks
33
-6.6
-3.8
-5.3
Dec 31, 2018 Dividends Free Cash Flow1
2.9
-16.2
0.2
OthersAlcon Net debt2 Sep 30, 2019Treasury share
transactions, net
M&A
transactions1
-19.4
9.4
-3.2
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines (including the Xiidra® acquisition for USD 3.5bn) and Sandoz (including the US
generic oral solids and dermatology portfolio), as well as the continuing corporate functions 2. Includes the net de-recognition of USD 0.6bn cash and cash equivalents and USD 3.5bn of financial debts related to the Alcon spin-off
USD billion
Key drivers vs. PY:
+ Higher operating income
(adjusted for non-cash items)
− Higher working capital
Offsetting one-time effects:
+ Real estate divestment proceeds
− Prior year OTC JV dividend and GSK milestone income
9M 2019 free cash flow at USD 9.4bn
8.39.4
9M 2018 9M 2019
+13%
Continuing operations1 free cash flow2
USD billion
1. Continuing operations as defined on page 44 of the Condensed Interim Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as
the continuing corporate functions 2. Free cash flow is a non-IFRS measure. Further details regarding non-IFRS measures can be found starting on page 56 of the Condensed Financial Report
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 34
Planned filings 2019 to 2022
35 | Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Entresto®
Heart failure (PEF)
INC280 NSCLC
Cosentyx® USnr-axSpA
OMB157Relapsing multiple sclerosis
2019
Xolair EUNasal Polyps
New molecule
New indication
New formulation
Biosimilars
2022
Cosentyx®
AS H2H
Rydapt®AML (FLT3 wild type)
ECF843Dry eye
ACZ885Adjuvant NSCLC
Cosentyx®
Hidradenitis suppurativa
QAW039Asthma
2020
Jakavi®Acute GVHD
Cosentyx®
PsA H2H
Jakavi®Chronic GVHD
PDR001 + Taf/MekMetastatic BRAF V600+ melanoma
AVXS-101 ITSMA Type 2/3
177Lu-PSMA-617mCRPC
ABL001CML 3rd line
QGE031CSU
Entresto®
Post-acute myocardial infarction
Beovu® (RTH258)Diabetic macular edema
2021
LAM320a
MDR tuberculosis
ACZ8851st Line NSCLC
ACZ8852nd Line NSCLC
Kymriah®
r/r Follicular Lymphoma
Kymriah®
r/r DLBCL in 1st relapse
MBG453MDS
PsA H2H Psoriatic arthritis head-to-head study versus adalimumab
ROP Retinopathy of prematurity
RCC Renal cell carcinoma
SAA Severe aplastic anemia
SMA Type 1 Spinal Muscular Atrophy Type 1 (IV formulation)
SMA Type
2/3
Spinal Muscular Atrophy Type 2/3 (IT formulation)
SPMS Secondary Progressive Multiple Sclerosis
TNBC Triple negative breast cancer
MDR Multi-drug resistant
MDS Myelodysplastic syndrome
nAMD Neovascular (wet) age-related macular degeneration
NASH Non-alcoholic steatohepatitis
nr-axSpA Non-radiographic axial spondyloarthritis
NSCLC Non-small cell lung cancer
PDR Proliferative Diabetic Retinopathy
PEF Preserved ejection fraction
Indication abbreviations:
ALL Acute lymphoblastic leukemia
AML Acute myeloid leukemia
AS H2H Ankylosing spondylitis head-to-head study versus
adalimumab
BC Breast cancer
CLL Chronic Lymphocytic Leukemia
CML Chronic myeloid leukemia
CRC Colorectal cancer
CSU Chronic spontaneous urticaria
CVRR Secondary prevention of cardiovascular events in patients
with elevated levels of lipoprotein (a)
GCA Giant cell arteritis
GVHD Graft-versus-host disease
HNSCC Head and neck squamous cell carcinoma
mCRPC Metastatic castration-resistant prostate cancer
2023
ZPL389Atopic dermatitis
UNR844Presbyopia
QBW251COPD
VAY736Autoimmune Hepatitis
ABL001CML 1st line
VPM087CRC 1L/RCC 1L
SAF312Chronic ocular surface pain
BYL719 (Piqray® US)HER2+ adv. breast cancer
TQJ230CVRR
BYL719 (Piqray® US)TNBC2
BYL719 (Piqray® US)HNSCC 2/3L
BYL719 (Piqray® US)Ovarian Cancer
AVXS-201Rett Syndrome
KAE609Malaria
KAF156 Malaria
LJN452NASH
HDM201AML
CFZ533Solid Organ Transplant
CSJ117Severe Asthma
LJC242NASH (+cen)
LMI070Spinal muscular atrophy
LNP023IgA nephropathy
LOU064CSU
MOR106Atopic Dermatitis
LXE408Visceral leishmaniosis
VAY736Primary Sjoegren’s syndrome
Kymriah ® + pembror/r DLBCL10
CFZ533Sjoegren’s Syndrome
LNP023Membranous nephropathy
RTH258Retinal vein occlusion
Kisqali ®HR+, HER2 (-) BC9 (adjuvant)
PDR001 comboMetastatic Melanoma
LNP023C3 glomerulopathy
GP2411 (denosumab)Osteoporosis, skeletal-related in bone
met. pts (same as originator)
KAE609Severe Malaria
Cosentyx®
GCA
RTH258PDR
Combination abbreviations:
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
cen cenicriviroc
pembro pembrolizumab
fulv fulvestrant
BYL719PIK3CA-related overgrowth spectrum
Pipeline of key projects in confirmatory development
Early Clinical Trials Registration Trials – Phase III / Pivotal In Registration
36| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
a) Approved in US, submitted in EU.
LMI070Spinal muscular atrophy
LNP023IgA nephropathy
KAE609Malaria
ECF843Dry eye
KAF156Malaria
HDM201AML
CFZ533Solid Organ Transplant
CSJ117Severe Asthma
AVXS-201Rett Syndrome
LJC242NASH (+cen)
177Lu-PSMA-617mCRPC
ABL001CML 1st line
QAW039Asthma
ACZ885Adjuvant NSCLC
ACZ8851st Line NSCLC
INC280 NSCLC
ABL001CML 3rd line
QGE031CSU
PDR001 + Taf/MekMetastatic BRAF V600+ melanoma
ACZ8852nd Line NSCLC
BYL719 (Piqray® US)HER2+ adv. BC
TQJ230CVRR
LJN452NASH
QBW251COPD
UNR844Presbyopia
VAY736Autoimmune Hepatitis
LOU064CSU
MOR106Atopic Dermatitis
VPM087CRC 1L/RCC 1L
SAF312Chronic ocular surface pain
MBG453MDS21
LXE408Visceral leishmaniosis
VAY736Primary Sjoegren’s syndrome
ZPL389Atopic dermatitis
PDR001 comboMetastatic Melanoma
Kymriah®+ pembro
r/r DLBCL
CFZ533Sjoegren’s Syndrome
LNP023Membranous nephropathy
Zolgensma®SMA Type 2/3
LNP023C3 glomerulopathy
KAE609Severe Malaria
PsA H2H Psoriatic arthritis head-to-head study
versus adalimumab
ROP Retinopathy of prematurity
RCC Renal cell carcinoma
SAA Severe aplastic anemia
SMA Type 1 Spinal Muscular Atrophy Type 1 (IV
formulation)
SMA Type 2/3 Spinal Muscular Atrophy Type 2/3 (IT
formulation)
SPMS Secondary Progressive Multiple Sclerosis
TNBC Triple negative breast cancer
Indication abbreviations:
Cosentyx® USnr-axSpA
Entresto®
Heart failure (PEF)
Entresto®
Post-acute myocardial infarction
Jakavi®Acute GVHD
Cosentyx®
PsA H2H
Cosentyx®
AS H2H
Jakavi®Chronic GVHD
Cosentyx®
Hidradenitis suppurativa
BYL719 (Piqray® US)TNBC
BYL719 (Piqray® US)HNSCC
BYL719 (Piqray® US)Ovarian Cancer
Cosentyx®
GCA
LAM320MDR tuberculosis
OMB157 Relapsing multiple sclerosis
RTH258Diabetic macular edema
Rydapt®AML (FLT3 wild type)
Xolair EUNasal Polyps
RTH258Retinal vein occlusion
Kymriah®
r/r DLBCL in 1st relapse
Kisqali®
HR+, HER2(-) BC (adjuvant)
Kymriah®
r/r Follicular Lymphoma
GP2411 (denosumab)2
Osteoporosis, skeletal-related in bone met. pts (same as originator)
RTH258PDR
GVHD Graft-versus-host disease
HNSCC Head and neck squamous cell carcinoma
mCRPC Metastatic castration-resistant prostate
cancer
MDR Multi-drug resistant
MDS Myelodysplastic syndrome
nAMD Neovascular (wet) age-related macular
degeneration
NASH Non-alcoholic steatohepatitis
nr-axSpA Non-radiographic axial spondyloarthritis
NSCLC Non-small cell lung cancer
PDR Proliferative Diabetic Retinopathy
PEF Preserved ejection fraction
ALL Acute lymphoblastic leukemia
AML Acute myeloid leukemia
AS H2H Ankylosing spondylitis head-to-head
study versus adalimumab
BC Breast cancer
CLL Chronic Lymphocytic Leukemia
CML Chronic myeloid leukemia
CRC Colorectal cancer
CSU Chronic spontaneous urticaria
CVRR Secondary prevention of cardiovascular
events in patients with elevated levels of
lipoprotein (a)
GCA Giant cell arteritis
Combination abbreviations:
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
cen cenicriviroc
pembro pembrolizumab
fulv fulvestrant
Promacta®/Revolade®
SAA17 1st line
SEG101Sickle cell disease
RTH258a EU (Beovu®)nAMD6
Lucentis®
Diabetic retinopathy
BAF312a EU (Mayzent™US)SPMS20
QMF149Asthma
QVM149Asthma
LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia and
others (same as originator)
Zolgensma®a
SMA Type 123
BYL719a (Piqray® US)PIK3CA mutant HR+, HER2 (-)
postmenopausal adv BC5 2nd line (+fulv)
Xiidra®a
Dry eye
Cosentyx® EUnr-axSpA
Xolair USNasal Polyps
New molecule
New indication
New formulation
Biosimilars
BYL719PIK3CA-related overgrowth spectrum
References
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 37
Slide No. Reference
Slide 8 -
Cosentyx®
1. Non-radiographic axial spondyloarthritis (nr-axSpA) and ankylosing spondylitis (AS) are part of the axial spondyloarthritis (axSpA) spectrum.
2. DRG Epidemiology database - axSpA: release Dec’18; PsA: Nov’18; RA: Jan’19. Patients on biologics: PsA and AS - Calculated Patient equivalent based on IQVIA Midas
volume Dec’18, Indication split IQVIA medical data Dec’18; nr-axSpA - DRG disease landscape forecast release Dec’18, RA: Corrona Study 2017; Reference for US data:
Epidemiology - axSpA: DRG (Prevalence), Optiref Study (Diagnosis Rate), Jefferies (Treatment Rate) December 2018; PsA: Jefferies (Prevalence & Diagnosis Rate),
Treatment Rate (Jefferies & DRG) December 2018.
3. Out of patients treated with systemics.
Slide 16 -
Mayzent®1. Largest trial performed in SPMS; KapposL et al. Siponimod vs. placebo in SPMS: double-blinded randomized, Ph3. The Lancet. 2018; DOI 10.1016/S0140-6736(18)30475-6
2. Benedict et al. Effect of Siponimod on cognition in patients with SPMS: Ph3 EXPAND study subgroup analyses. AAN 2019. P2-051
3. Novartis QuickPulse HCP Tracker, reported by InCrowd Inc. fielded May 3-15, 2019
4. Spherix Global Insights RealTime Dynamix – MS Q219
Clinical Trials Update Includes selected ongoing or recently concluded global trials of Novartis development programs/products which are
in confirmatory development or marketed (typically Phase 2 or later).
For further information on all Novartis clinical trials, please visit: www.novartisclinicaltrials.com
Key changes vs. Q2 2019 presentationNew trials added
Study Program Indication Phase Patients
NCT03867201 DRAGON (CAMG334A2304) Aimovig® Migraine Phase 3 550
NCT03781414 CONTRAIL I (CCFZ533A2202) CFZ533 Kidney transplantation Phase 2 128
NCT03905525 TWINSS (CCFZ533B2201) CFZ533 Sjögren's syndrome Phase 2B 260
NCT03974100 (CGP24112301) GP2411 Osteoporosis Phase 3 522
NCT04097821 ADORE (CINC424H12201) Jakavi® Myelofibrosis Phase 1/2 130
NCT04072887 (CQBW251B2201) QBW251 Chronic obstructive pulmonary disease (COPD) Phase 2 900
NCT03802630 RAPTOR (CRTH258C2301) RTH258 Retinal vein occlusion Phase 3 500
NCT03810313 RAVEN (CRTH258C2302) RTH258 Retinal vein occlusion Phase 3 750
NCT03917472 (CRTH258B2305) RTH258 Diabetic macular edema Phase 3 500
NCT04058067 KINGLET (CRTH258B2304) RTH258 Diabetic macular edema Phase 3 268
39 | Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Trials removed (operational decision-points achieved)
Study Program Indication Phase Patients
NCT02565511 GENERATION S1 (CAPI015A2201J) CNP520 Alzheimer’s disease Phase 2B/3 1,340
NCT01544595 (CAIN457A2302E1 – extension study) Cosentyx® Psoriasis Phase 3 1,146
NCT02748863 ALLURE (CAIN457A2323) Cosentyx® Psoriasis Phase 3 214
NCT02826603 CLARITY (CAIN457A2326) Cosentyx® Psoriasis Phase 3B 1,102
NCT03512197 UNIFY (CPKC412E2301) Rydapt® Acute myeloid leukemia Phase 3 502
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
41
Study NCT02678312 PANORAMA HF (CLCZ696B2319) NCT03785405 (CLCZ696B2319E1 – extension study)
Indication Heart failure in pediatric patients Heart failure in pediatric patients
Phase Phase 2/3 Phase 3
Patients 360 240
Primary Outcome
Measures
Part 1: Pharmacodynamics and pharmacokinetics of
sacubitril/valsartan LCZ696 analytes
Part 2: Efficacy and safety compared with enalapril
Number of participants with Adverse Events (AEs) and
Serious Adverse Events (SAEs)
Arms/Intervention
• Part 1: Sacubitril/valsartan 0.8 mg/kg or 3.1 mg/kg or
both; 0.4 mg/kg or 1.6 mg/kg or both (single doses).
• Part 2: enalapril/placebo 0.2 mg/kg bid (ped. formulation
1mg/ml) and adult formulation (2.5, 5, 10 mg bid);
Sacubitril/valsartan (LCZ696)/placebo: Ped. formulation
granules (12.5, 31.25 mg in capsules); liquid formulation
(1mg/ml and 4mg/ml concentration) and adult
formulation (50, 100, 200 mg bid)
• Single arm, open label sacubitril/valsartan (pediatric
formulation granules (12.5, 31.25 mg in capsules); liquid
formulation (1mg/ml and 4mg/ml concentration) and
adult formulation (50, 100, 200 mg bid))
Target Patients
Pediatric patients from 1 month to < 18 years of age with
heart failure due to systemic left ventricle systolic
dysfunction
Pediatric patients with heart failure due to systemic left
ventricle systolic dysfunction who have completed study
CLCZ696B2319
Expected Completion
2021; (Analysis of 110 pts from Part 2 formed the basis for
pediatric submission in Apr-2019 and approval by the US
FDA in Oct-2019 for the treatment of symptomatic HF with
systemic left ventricular systolic dysfunction in children aged
1 year and older)
2022
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
42
Study NCT02554890 PIONEER-HF (CLCZ696BUS01) NCT02661217 TRANSITION (CLCZ696B2401)
Indication Heart failure, reduced ejection fraction Heart failure, reduced ejection fraction
Phase Phase 3B/4 Phase 4
Patients 881 1,002
Primary Outcome
Measures
Percentage change from baseline in N-terminal pro-brain
natriuretic peptide (NT-proBNP)
Assessing the percentage of patients who achieve the target
dose of 200 mg bid LCZ696 at 10 weeks after
randomization
Arms/Intervention
• Sacubitril/valsartan (LCZ696) 24/26 mg, 49/51 mg or
97/103 mg bid or matching placebo
• Enalapril (2.5 mg, 5 mg, and 10 mg) bid or matching
placebo
• Pre-discharge treatment initiation - LCZ696 (50, 100,
200 mg bid)
• Post-discharge treatment initiation - LCZ696 (50, 100,
200 mg bid)
Target PatientsPatients with HFrEF (LVEF<40%) hospitalized for ADHF
and stable for more than 24 hours
Heart failure patients with reduced ejection-fraction
hospitalized for an acute decompensation event
Expected Completion Q3-2018 (actual) Q4-2018 (actual)
Publication
• Mar-2019 ACC: 4wk OLE data, and core study data on
biomarkers, de novo HF, hospitalizations, & prior
exposure
• Apr-2019 Circulation: Research letter on composite
endpoint (Circulation. 2019;139:00–00)
• Q3-2019 ESC: Secondary abstracts submitted
• Planned in Q1-2020: RAAS naive and de novo HF
• 28-May-2019 TRANSITION primary publication -
published EJHF
• Secondary data presentations: de novo HF and
ACEi/ARB naive sub-groups presented at ESC-HF
Congress in May 2019 and submitted to EJHF in Jun-
2019 (expected publication Q3-2019)
• Planned in Q4-2019: 26-weeks data presentation at
ESC-2019 in Paris
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
43
Study NCT02884206 PERSPECTIVE (CLCZ696B2320) NCT02468232 PARALLEL-HF (CLCZ696B1301)
Indication Heart failure Heart failure, reduced ejection fraction
Phase Phase 3 Phase 3
Patients 592 225
Primary Outcome
Measures
Change from baseline in the CogState Global Cognitive
Composite Score (GCCS)
Time to the first occurrence of the composite endpoint -
either cardiovascular (CV) death or heart failure (HF)
hospitalization
Arms/Intervention
• Sacubitril/valsartan 50, 100, and 200 mg bid with
placebo of valsartan
• Valsartan 40, 80, and 160 mg bid tablets with placebo
for sacubitril/valsartan
• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo
of enalapril
• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of
sacubitril/valsartan
Target PatientsPatients with chronic heart failure with preserved ejection
fraction
Japanese heart failure patients (NYHA Class II-IV) with
reduced ejection fraction
Expected Completion 2022 Q1-2019 (actual); H2-2020 (open-label extension)
Publication TBDPlanned in H1-2020: Core study primary manuscript in Circ
J
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
44
Study NCT01920711 PARAGON-HF (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)
Indication Heart failure, preserved ejection fraction Heart failure, preserved ejection fraction
Phase Phase 3 Phase 3
Patients 4,822 2,577
Primary Outcome
Measures
Cumulative number of primary composite events of
cardiovascular (CV) death and total (first and recurrent) HF
hospitalizations
Change in NT-proBNP from baseline to week 12
and change in 6 minute walk distance (6MWD) from
baseline to Week 24
Arms/Intervention
• Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200
mg bid
• Valsartan or placebo 40 mg, 80 mg, and 160 mg bid
• Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and
matching placebo
• Enalapril 2.5 mg, 5 mg and 10 mg bid and matching
placebo
• Valsartan 40 mg, 80 mg, 160 mg bid and matching
placebo
Target PatientsHeart failure patients (NYHA Class II-IV) with preserved
ejection fraction
Heart failure patients (NYHA Class II-IV) with preserved
ejection fraction
Expected Completion Q3-2019 (actual) Q4-2019
Publication
• Sep-2019: Primary manuscript published (Angiotensin–
Neprilysin Inhibition in Heart Failure with Preserved
Ejection Fraction. Solomon S et al; NEJM. DOI:
10.1056/NEJMoa1908655)
• Sep-2019: ESC: Late breaker presentation of primary
results
TBD
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
45
Study NCT03909295 (CLCZ696D1301E1 – extension study) NCT02924727 PARADISE-MI (CLCZ696G2301)
Indication Heart failure chronic Post-acute myocardial infarction
Phase Phase 3 Phase 3
Patients 63 5,650
Primary Outcome
Measures
Number of participants with Adverse Events (AEs) and
Serious Adverse Events (SAEs)
Time to the first occurrence of a confirmed composite
endpoint (cardiovascular (CV) death, heart failure (HF)
hospitalization, or outpatient heart failure)
Arms/Intervention• Sacubitril/valsartan 50 mg,100 mg,200 mg film coated
tablets
• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid / placebo
of ramipril/valsartan
• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of
sacubitril/valsartan / placebo for valsartan
Target Patients
Japanese heart failure patients (NYHA Class II-IV) with
preserved ejection fraction after CLCZ696D2301
(PARAGON-HF)
Post-AMI patients with evidence of LV systolic dysfunction
and/or pulmonary congestion, with no known prior history of
chronic HF
Expected Completion 2021 H2-2020
Publication TBD TBD
Entresto® - Angiotensin receptor neprilysin inhibitor (ARNI)
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
CFZ533 – Blocking, non-depleting, Fc-silent, anti-CD40 monoclonal antibody
Study NCT03663335 (CCFZ533A2201) NCT03905525 TWINSS (CCFZ533B2201)
Indication Kidney transplantation Sjögren's syndrome
Phase Phase 2B Phase 2B
Patients 325 260
Primary Outcome
Measures
Composite event (BPAR, Graft Loss or Death) over 12
months post-transplantation and post conversion (for
maintenance cohort)
Change in EULAR Sjögren’s syndrome Disease Activity
Index (ESSDAI) score and EULAR Sjögren’s syndrome
Patient Reported Index (ESSPRI) score
Arms/Intervention• CFZ533 doses + MMF + corticosteroids
• Control/Standard of Care: TAC + MMF + corticosteroids
• CFZ533 doses
• Placebo
Target Patients Kidney transplant recipients Patients with Sjögren's syndrome
Expected Completion 2021 2022
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
47
CFZ533 – Blocking, non-depleting, Fc-silent, anti-CD40 monoclonal antibody
Study NCT03781414 CONTRAIL I (CCFZ533A2202)
Indication Kidney transplantation
Phase Phase 2
Patients 128
Primary Outcome
Measures
Proportion of patients with composite event (BPAR, Graft
Loss or Death) over 12 months
Arms/Intervention
• Control/Standard of Care: TAC + MMF + Corticosteroids
• CFZ533 dose A + MMF + Corticosteroids
• CFZ533 dose B + MMF + Corticosteroids
Target Patients Liver transplant recipients
Expected Completion 2022
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
48
Cosentyx® - Anti IL-17
49
Study NCT03504852 (CAIN457A2324) NCT03589885 MATURE (CAIN457A2325)
Indication Psoriasis Psoriasis
Phase Phase 3B Phase 3
Patients 331 122
Primary Outcome
Measures
PASI 90 response and IGA mod 2011 0 or 1 response after
16 weeks of treatment
PASI 75 response and IGA mod 2011 0 or 1 response after
12 weeks of treatment
Arms/Intervention
• Secukinumab 300 mg every 2 weeks after weekly doses
till Week 4
• Secukinumab 300 mg every 4 weeks after weekly doses
till Week 4
• Secukinumab 2 mL (300 mg) auto-injector
• Secukinumab 2 x 1 mL (150 mg each) prefilled syringe
• Placebo 2 mL auto-injector
• Placebo 2 x 1 mL prefilled syringe
Target Patients Subjects (≥90kg) with moderate to severe plaque psoriasis Subjects with moderate to severe plaque psoriasis
Expected Completion H2-2020 H2-2020
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
50
Study NCT02471144 (CAIN457A2310) NCT03668613 (CAIN457A2311)
Indication Psoriasis Psoriasis
Phase Phase 3 Phase 3
Patients 162 84
Primary Outcome
Measures
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
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 low dose
• Secukinumab high dose
Target PatientsPatients from 6 to less than 18 years of age with severe
chronic plaque psoriasis
Pediatric patients of age 6 to <18 years, with moderate to
severe plaque psoriasis
Expected Completion 2023 2023
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
51
Study NCT03066609 (CAIN457A2318)
Indication Psoriasis
Phase Phase 3
Patients 543
Primary Outcome
Measures
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
• Secukinumab 150 mg
• Placebo
Target PatientsPatients with moderate to severe chronic plaque-type
psoriasis with or without psoriatic arthritis comorbidity
Expected Completion Q1-2019 (actual)
Publication
• Week 16 results: Poster presented at: 2019 American
Academy of Dermatology (AAD) Annual Meeting,
• March 1–5, 2019, Washington, D.C.
• 52-week results: Poster at EADV 2019, Madrid 9-13
October, 2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
52
Study NCT03031782 (CAIN457F2304) NCT03769168 (CAIN457F2304E1 – extension study)
Indication Psoriatic arthritis Psoriatic arthritis
Phase Phase 3 Phase 3
Patients 80 64
Primary Outcome
MeasuresTime to 33 flares Number of participants with JIA ACR30 response
Arms/Intervention• Secukinumab (pre-filled syringe) 75 mg
• Placebo
• Secukinumab 75 mg/0.5 ml
• Secukinumab 150 mg/1.0 ml
Target PatientsJuvenile idiopathic arthritis subtypes of psoriatic and
enthesitis-related arthritis
Patients with juvenile idiopathic arthritis subtypes of juvenile
psoriatic arthritis and enthesitis related arthritis
Expected Completion 2021 2025
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
53
Study NCT01892436 FUTURE 1 extension (CAIN457F2306E1) NCT01649375 MEASURE 2 (CAIN457F2310)
Indication Psoriatic arthritis 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 published in September
2018 (Mease PJ, et al. RMD Open 2018;4:e000723.
doi:10.1136/rmdopen-2018-000723)
• 5 year results: accepted for publication in ACR open
Rheumatology in September 2019
• Primary 52 week results: Baeten D & Sieper J, et al. N
Engl J Med 2015;373:2534–48
• 2 year results: Marzo-Ortega, et al. Arthritis Care Res
2017 Feb 24. doi: - 10.1002/acr.23233
• 3 year results: Marzo-Ortega, et al. RMD 2017
• 5 year results: EULAR 2019; Marzo-Ortega H, et al.
FRI0379. Annals of the Rheumatic Diseases
2019;78:873.
• 5 year results; manuscript target submission Oct 2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
54
Study NCT01752634 FUTURE 2 (CAIN457F2312) NCT02008916 MEASURE 3 (CAIN457F2314)
Indication Psoriatic arthritis 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 (actual) 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
• 5 year (EOS) manuscript ongoing; to be submitted in
Oct-2019
• 16 weeks results: PANLAR congress in Apr-2016
• 52 weeks results: Pavelka et al. Arthritis Research &
Therapy 2017
• 2 year results: Presented at ACR in Nov-2017
• 3 year (EOS) results: To be presented (ORAL) at
PANLAR April 2019
• 3 year (EOS) manuscript submitted in May-2019;
awaiting journal decision
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
55
Study NCT01989468 FUTURE 3 (CAIN457F2318) NCT02159053 MEASURE 4 (CAIN457F2320)
Indication Psoriatic arthritis 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, 20:47
• Week 104 (EOS) manuscript: Kivitz et al, Rheumatol
Ther https://doi.org/10.1007/s40744-018-0123-5
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
56
Study NCT02294227 FUTURE 4 (CAIN457F2336) NCT02404350 FUTURE 5 (CAIN457F2342)
Indication Psoriatic arthritis Psoriatic arthritis
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) Q1-2019 (actual)
Publication
• 52 week results: abstract presented at PANLAR
congress (Apr-2018)
• 2 year (EOS) results published: Rheumatology
Therapy. 2019 Jun 21. doi: 10.1007/s40744-019-0163-5.
• 24 week results published: Mease P, et al. Annals of the
Rheumatic Diseases 2018;77:890-897.
• 52 week results presented at EULAR and ACR 2018
• 52 week manuscript accepted (Rheumatology, October
2019, in press)
• 2 year (EOS) results presented at EULAR 2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
57
Study NCT01863732 (CAIN457F2305E1 – extension study) NCT02745080 EXCEED (CAIN457F2366)
Indication Ankylosing spondylitis Psoriatic arthritis
Phase Phase 3 Phase 3
Patients 300 850
Primary Outcome
Measures
Assessment of spondyloarthritis international society criteria
/ ASAS 20 responseAmerican College of Rheumatology 20 (ACR20) response
Arms/Intervention• Secukinumab 75 mg in PFS
• Secukinumab 150 mg in PFS
• Secukinumab 300 mg s.c.
• Adalimumab 40 mg s.c.
Target Patients Patients with active ankylosing spondylitis Patients with active psoriatic arthritis
Expected Completion Q2-2018 (actual) H1-2020
Publication
• 3-year results: Manuscript published in Clinical and
Experimental Rheumatology in May-2017
• 4-year results: Presented at ACR in Nov-2017
• 4 year results manuscript published; Rheumatology,
Volume 58, Issue 5, May 2019, Pages 859–868,
• 5 year (EOS) results manuscript published; Baraliakos X,
et al. RMD Open 2019;5:e001005. doi:
10.1136/rmdopen-2019-001005
• Manuscript target submission January 2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
58
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 Week 52: Q3-2019 (actual); Final: 2021 2022
Publication
• Abstract (16 week results) submitted as a late breaker to
ACR 2019
• Manuscript target submission Jan-2020
TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Cosentyx® - Anti IL-17
59
Study NCT03713619 SUNSHINE (CAIN457M2301) NCT03713632 SUNRISE (CAIN457M2302)
Indication Hidradenitis Suppurativa (HS) Hidradenitis Suppurativa (HS)
Phase Phase 3 Phase 3
Patients 471 471
Primary Outcome
Measures
Proportion of participants with Hidradenitis Suppurativa
clinical response (HiSCR)
Proportion of patients with Hidradenitis Suppurativa Clinical
Response (HiSCR)
Arms/Intervention
• Secukinumab 300 mg every 2 weeks
• Secukinumab 300 mg every 4 weeks
• Placebo (every 2 weeks)
• Placebo (every 4 weeks)
• Secukinumab 300 mg every 2 weeks
• Secukinumab 300 mg every 4 weeks
• Placebo (every 2 weeks)
• Placebo (every 4 weeks)
Target Patients Subjects with moderate to severe Hidradenitis Suppurativa Subjects with moderate to severe Hidradenitis Suppurativa
Expected Completion 2022 2022
Publication Preliminary results in EADV (most likely) in 2021 Preliminary results in EADV (most likely) in 2021
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Ilaris® - Anti IL-1β
60
Study NCT02059291 CLUSTER (CACZ885N2301) NCT02296424 (CACZ885G2306)
Indication Hereditary periodic fevers SJIA - Systemic Juvenile Idiopathic Arthritis
Phase Phase 3 Phase 3B/4
Patients 203 182
Primary Outcome
Measures
To demonstrate significant reduction of disease activity
with canakinumab vs. placebo
Proportion of patients in clinical remission on canakinumab
who are able to remain in remission following canakinumab
dose tapering (reduced canakinumab dose or prolonged
canakinumab dosing interval)
Arms/Intervention• Canakinumab
• Placebo
• Canakinumab dose reduction
• Canakinumab dose interval prolongation
Target PatientsPatients with, 3 separate disease cohorts TRAPS, HIDS,
and colchicine resistant FMF (Hereditary periodic fevers )
Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)
(Pediatric)
Expected Completion 2017 (actual) 2018 (actual)
Publication
• QoL overall presented at EULAR in Q2-2019 and will
be presented at ACR in Q4-2019
• Planned manuscripts: QoL overall to be submitted in
Q4-2019 and crFMF efficacy & safety in Q2-2020
• Remission & flexible dosing – presented at ISSAID & EULAR in Q2-2019
• Planned manuscript in 2019: Remission & flexible dosing to be submitted in Q4-2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
LJN452 - FXR Agonist
61
Study NCT02855164 (CLJN452A2202)
Indication Non-alcoholic steatohepatitis
Phase Phase 2
Patients 345
Primary Outcome
Measures
Adverse event profile of different doses; determine the dose
relationship of LJN452 on markers of hepatic inflammation
in NASH (ALT and AST); determine dose-response
relationship of LJN452 on liver fat content by changes in
quantitative MRI; determine effect of LJN452 on liver fibrosis
by biopsy
Arms/Intervention Multiple LJN452 doses and placebo
Target Patients Patients with non-alcoholic steatohepatitis (NASH)
Expected Completion H1-2020
Publication
• Primary (interim) data abstract submitted to AASLD in
Q3-2019
• Manuscript to be submitted in H2-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
LOU064 – Bruton's tyrosine kinase (BTK) inhibitor
62
Study NCT03926611 (CLOU064A2201)
Indication Chronic spontaneous urticaria (CSU)
Phase Phase 2
Patients 308
Primary Outcome
MeasuresChange from baseline in weekly Urticaria Activity Score (UAS7) at Week 4
Arms/Intervention
• LOU064 Arm 1 Low dose of LOU064 orally in the morning (once daily) and
matching placebo in the evening from Day 1 to 85
• LOU064 Arm 2 Medium dose of LOU064 orally in the morning (once daily) and
matching placebo in the evening from Day 1 to 85
• LOU064 Arm 3 High dose of LOU064 orally in the morning (once daily) and
matching placebo in the evening from Day 1 to 85
• LOU064 Arm 4 Low dose of LOU064 orally, twice daily from Day 1 to 85
• LOU064 Arm 5 Medium dose of LOU064 orally, twice daily from Day 1 to 85
• LOU064 Arm 6 High dose of LOU064 orally, twice daily from Day 1 to 85
• Placebo arm Matching placebo, orally, twice daily from Day 1 to 85
Target Patients Adults with CSU inadequately controlled by H1-antihistamines
Expected Completion 2020
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
LJC242 - FXR agonist + CCR2/CCR5 inhibitor
63
Study NCT03517540 TANDEM (CLJC242A2201J)
Indication Non-alcoholic steatohepatitis
Phase Phase 2
Patients 200
Primary Outcome
Measures
• Evaluation of safety and tolerability of combination
therapy (tropifexor + cenicriviroc) by monitoring adverse
event profile, vital signs and laboratory parameters
Arms/Intervention
• Tropifexor
• Cenicriviroc
• Tropifexor + cenicriviroc
Target PatientsAdult patients with non-alcoholic steatohepatitis (NASH) and
liver fibrosis
Expected Completion H2-2020
Publication Manuscript to be submitted in H1-2021
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
QGE031 - Anti-IgEStudy NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)
Indication Chronic spontaneous urticaria / Chronic idiopathic urticaria Chronic spontaneous urticaria / Chronic idiopathic urticaria
Phase Phase 2B Phase 2B
Patients 382 226
Primary Outcome
Measures
Establish dose-response relationship of QGE031 with
respect to achievement of complete hives response at week
12
Long-term safety; number of participants with treatment-
emergent adverse events
Arms/Intervention
• Ligelizumab 24mg q4wks for 20 weeks
• Ligelizumab 72mg q4wks for 20 weeks
• Ligelizumab 240mg q4wks for 20 weeks
• Ligelizumab 120mg single dose
• Omalizumab 300mg q4wks for 20 weeks
• Placebo q 4wks for 20 weeks
Ligelizumab 240 mg q4wks open label for 52 weeks
Target Patients
Adult patients with chronic spontaneous urticaria
inadequately controlled with H1-antihistamines at approved
or increased doses, alone or in combination with H2-
antihistamines or leukotriene receptor antagonists.
Adult patients with chronic spontaneous urticaria
inadequately controlled with H1-antihistamines at approved
or increased doses, alone or in combination with H2-
antihistamines or leukotriene receptor antagonists.
Expected Completion 2017 (actual) Q3-2019 (actual)
Publication
• Primary results: Presented at EAACI 2018, EADV 2018,
and GUF 2018; NEJM publication (Oct. 3rd);
• Secondary results presented in 2019 at: AAD, EAACI,
WCD, EADV, PAAM, ACAAI, UCARE.
• Primary results: AAD 2019;
• Secondary results presented in 2019 at: AAD, EAACI,
WCD, EADV, PAAM, ACAAI, UCARE; manuscript
planned in H1/2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
64
QGE031 - Anti-IgE
Study NCT03437278 (CQGE031C2202)
Indication Chronic spontaneous urticarial / Chronic idiopathic urticaria
Phase Phase 2
Patients 48
Primary Outcome
MeasuresChange in the 7 day Urticaria Activity Score (UAS7)
Arms/Intervention
• Ligelizumab high dose q4wks for 24 weeks
• Ligelizumab low dose q4wks for 24 weeks
• Placebo / ligelizumab high dose q4wks for 8 / 16 weeks
Target PatientsAdolescents from 12 to <18 years of age, with chronic
spontaneous urticaria
Expected Completion H2-2021
Publication• Study design to be presented at PAAM 2019,
• Manuscript to be submitted in 2022
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
65
QGE031 - Anti-IgE
Study NCT03580369 Pearl 1 (CQGE031C2302) NCT03580356 Pearl 2 (CQGE031C2303)
Indication Chronic spontaneous urticaria Chronic spontaneous 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 for 52 weeks
• Ligelizumab dose B q4w for 52 weeks
• Omalizumab 300 mg q4w for 52 weeks
• Placebo q4w from randomization to wk20, then
ligelizumab dose B from wk24 to wk52
• Ligelizumab dose A q4w for 52 weeks
• Ligelizumab dose B q4w for 52 weeks
• Omalizumab 300 mg q4w for 52 weeks
• Placebo q4w from randomization to wk20, then
ligelizumab dose B from wk24 to wk52
Target PatientsAdolescents and adults with chronic spontaneous urticaria
inadequately controlled with H1-antihistamines
Adolescents and adults with chronic spontaneous urticaria
inadequately controlled with H1-antihistamines
Expected Completion 2021 2021
Publication• Study design presented at UCARE 2018
• Manuscript to be submitted in 2022
• Study design presented at UCARE 2018
• Manuscript to be submitted in 2022
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
66
VAY736 – Fully human IgG1/κ anti-BAFF-R mAb
Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)
Indication Primary Sjögren's syndrome Autoimmune hepatitis
Phase Phase 2B Phase 2/3
Patients 180 80
Primary Outcome
Measures
Safety and efficacy of VAY736 in primary Sjögren's
syndrome (pSS)Alanine aminotransferase (ALT) normalization
Arms/Intervention• VAY736
• Placebo
• VAY736
• Placebo control with conversion to active VAY736
Target PatientsPatients with moderate to severe primary Sjögren's
syndrome (pSS)
Autoimmune hepatitis patients with incomplete response or
intolerant to standard treatment of care
Expected Completion H2-2020 2023
Publication
• Late Breaking Abstract to be submitted to American
College of Rheumatology 30-Sep-2019
• Manuscript to be submitted in 2020
TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
67
ZPL389 - H4 receptor antagonist
Study NCT03517566 ZEST (CZPL389A2203)NCT03948334 ZESTExt (CZPL389A2203E1 – extension
study)
Indication Atopic dermatitis Atopic dermatitis
Phase Phase 2 Phase 2
Patients 360 360
Primary Outcome
MeasuresIGA (Investigator's global assessment) response at week 16
Frequency of Adverse Events (AEs) and Serious Adverse
Events (SAEs)
Arms/Intervention
• ZPL389 dose 1
• ZPL389 dose 2
• ZPL389 dose 3
• ZPL389 dose 4
• Placebo
• ZPL389 Dose 1 + Topical Corticosteroids (TCS) and /or
Topical Calcineurin Inhibitors (TCI)
• ZPL389 Dose 2 + Topical Corticosteroids (TCS) and /or
Topical Calcineurin Inhibitors (TCI)
Target Patients Patients with moderate to severe atopic dermatitis Adult patients with atopic dermatitis
Expected Completion H1-2021 2022
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
68
Zolgensma® - SMN1 gene replacement therapy
70
Study NCT03461289 STRIVE-EU (CL-302) NCT03306277 STRIVE (CL-303)
Indication Type 1 spinal muscular atrophy Type 1 spinal muscular atrophy
Phase Phase 3 Phase 3
Patients 30 20
Primary Outcome
MeasuresProportion of participants sitting without support
• Achievement of independent sitting for at least 30
seconds
• Event-free survival
Arms/Intervention Open-label, single-arm, single-dose, intravenous Open-label, single-arm, single-dose, intravenous
Target Patients Patients with spinal muscular atrophy Type 1 Patients with Spinal Muscular Atrophy Type 1
Expected Completion H2-2020 Q4-2019
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Zolgensma® - SMN1 gene replacement therapy
71
Study NCT03505099 SPR1NT (CL-304) NCT03837184 STRIVE Asia Pacific (CL-306)
Indication Spinal muscular atrophy Type 1 spinal muscular atrophy
Phase Phase 3 Phase 3
Patients 27 6
Primary Outcome
Measures
• Percentage of participants achieving functional
independent sitting for at least 30 seconds at any visit
• Percentage of participants achieving the ability to stand
without support for at least 3 seconds at any visit
Proportion of participants sitting without support
Arms/Intervention Open-label, single-arm, single-dose, intravenous Open-label, single-arm, single-dose, intravenous
Target PatientsPre-symptomatic patients with spinal muscular atrophy and
multiple copies SMN2Patients with spinal muscular atrophy Type 1
Expected Completion 2021 2021
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Zolgensma® - SMN1 gene replacement therapy
72
Study NCT03381729 STRONG (CL-102)
Indication Type 2 spinal muscular atrophy
Phase Phase 1
Patients 27
Primary Outcome
Measures
• Safety and tolerability, incidence of adverse events
• Proportion of patients achieving Standing Milestone
• Change in Hammersmith Functional Motor Scale
Arms/Intervention Open-label, single-arm, single-dose, intrathecal
Target Patients Patients with spinal muscular atrophy with 3 copies of SMN2
Expected Completion Q4-2019
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Aimovig® – CGRP receptor antagonist
73
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 H2-2020
Publication
• Planned for Q4-2019 - PROs and prespecified subgroup
analysis (double blind phase)
• Planned for Q1-2020: 1Y OLE
• Planned for Q4 2020: 2Y OLE
Planned for H2-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Aimovig® – CGRP receptor antagonist
74
Study NCT03867201 DRAGON (CAMG334A2304)
Indication Migraine
Phase Phase 3
Patients 550
Primary Outcome
Measures
Change from baseline in monthly migraine days during the
last 4 weeks of the 12-week treatment period
Arms/Intervention• Subcutaneous injection of AMG334 (erenumab) 70 mg
• Subcutaneous injection of placebo
Target Patients Adult chronic migraine patients
Expected Completion 2022 DBT phase; 2024 OLE phase
Publication Planned in Q4-2023 (DBT)
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Gilenya® - S1P-R modulator
75
Study NCT01633112 ASSESS (CFTY720D2312) NCT01201356 LONGTERMS (CFTY720D2399)
Indication Relapsing remitting multiple sclerosis (RRMS) Relapsing multiple sclerosis (RMS)
Phase Phase 3B Phase 3
Patients 1,064 4,125
Primary Outcome
Measures
Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod
to glatiramer acetate (20 mg) in reducing the annualized
relapse rate up to 12 months
Long-term safety and tolerability
Arms/Intervention
• Fingolimod 0.5 mg orally
• Fingolimod 0.25mg orally
• Copaxone® 20 mg s.c.
Single-arm study of fingolimod 0.5 mg/day
Target Patients Patients with relapsing-remitting multiple sclerosis Patients with relapsing multiple sclerosis
Expected Completion 2018 (actual) 2018 (actual)
Publication• Primary data presentation at AAN in 2019
• Primary manuscript – submission planned in Q4-2019
• Primary data presentation: Cohen J, et al presented at
ECTRIMS 2017
• Primary manuscript accepted by Therapeutic Advances
in Neurological Disorders (estimated publication Oct-
2019)
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
LMI070 - SMN2 RNA splice modulator
76
Study NCT02268552 (CLMI070X2201)
Indication Type 1 spinal muscular atrophy
Phase Phase 1/2
Patients 39
Primary Outcome
Measures
Number of participants with adverse events (AEs), serious
adverse events (SAEs) and deaths
Arms/Intervention
Branaplam oral, once weekly:
• Part 1: 5 ascending doses
• Part 2: 2 different dose levels
• Part 3: patients continue on initial dose assigned in Part
1 or Part 2
Target PatientsPatients with type 1 spinal muscular atrophy
Expected Completion H2-2020
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Mayzent® - S1P-R modulator
77
Study NCT01665144 -EXPAND (CBAF312A2304)
Indication Secondary progressive multiple sclerosis
Phase Phase 3
Patients 1,652
Primary Outcome MeasuresThe delay in time to confirmed disability progression as
measured by EDSS (Expanded Disability Status Scale)
Arms/Intervention
• BAF312 (5-day titration: 0.25mg to 1.25mg; Maintenance
dose: 2mg (day 6))
• Placebo
Target Patients Patients with secondary progressive multiple sclerosis
Expected Completion Core in 2016/Extension in 2023
PublicationThe Lancet Neurology, Volume 39, No.10127, p1237-1330,
March 2018
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
OMB157 - Anti-CD20
78
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 (actual) Q3-2019 (actual)
Publication Primary manuscript planned in H1-2020 Primary manuscript planned in H1-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
OMB157 - Anti-CD20
79
Study NCT03249714 APOLITOS (COMB157G1301) NCT03650114 ALITHIOS (COMB157G2399)
Indication Multiple sclerosis Multiple Sclerosis
Phase Phase 2 Phase 3
Patients 60 2010
Primary Outcome
Measures
Reduced cumulative number of Gd-enhanced T1 lesions
across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab
vs placebo)
Evaluate the long-term safety and tolerability of ofatumumab
20 mg subcutaneous (sc) once every 4 (q4) weeks in
subjects with RMS from the first dose of ofatumumab
Arms/Intervention• Ofatumumab 20 mg subcutaneous injections
• Placebo• Ofatumumab 20 mg every 4 weeks
Target Patients Patients with relapsing forms of multiple sclerosis Patients with relapsing MS
Expected Completion H2-2020 2025
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor
81
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 bid
• Bosutinib 500 mg
• ABL001 40 mg QD + 400 mg imatinib
• ABL001 60 mg QD + 400 mg imatinib
• Imatinib 400mg QD (continuation treatment)
• Nilotinib 300mg bid (switch to nilotinib treatment)
Target Patients
Patients with chronic myelogenous leukemia in chronic
phase, previously treated with 2 or more tyrosine kinase
inhibitors
CML-CP patients not reaching DMR (MR 4.5) while on 1L
imatinib treatment
Expected Completion H2-2020 H1-2021
Publication Manuscript submission in H2-2020 (journal TBD) TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
ACZ885 – IL1β inhibitor
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 82
Study NCT03447769 (CACZ885T2301) NCT03631199 CANOPY-1 (CACZ885U2301)
Indication Adjuvant NSCLC 1st Line Non-small cell lung cancer (NSCLC)
Phase Phase 3 Phase 3
Patients 1,500 627
Primary Outcome
Measures
Disease free survival (primary), overall survival (key
secondary)
• Safety run-in part: Incidence of dose limiting toxicities
• Double-blind, randomized, placebo-controlled part:
Progression free survival (PFS)
• Overall survival (OS)
Arms/Intervention• Canakinumab 200mg q3w sc for 18 cycles
• Placebo q3w sc for 18 cycles
• Canakinumab or matching placebo in combination with
pembrolizumab and platinum-based doublet
chemotherapy
Target Patients
Patients with:
• High–risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB
(T>5cm N2)) after complete resection and standard of
care adjuvant cisplatin-based chemotherapy
• All histologies
Patients with
• Histologically confirmed Stage IIIB, IV NSCLC with no
prior systemic anticancer therapy
• Squamous and non-squamous NSCLC
• No EGFR mutation and ALK rearrangement
Expected Completion 2022 2021
Publication TBDJohnson B et al. Abstract accepted for presentation at
AACR-NCI-EORTC Oct 2019 (safety run-in)
ACZ885 – IL1β inhibitor
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 83
Study NCT03626545 CANOPY-2 (CACZ885V2301)
Indication 2nd / 3rd Line Non-small cell lung cancer (NSCLC)
Phase Phase 3
Patients 240
Primary Outcome
Measures
• Safety run-in part: Incidence of dose limiting toxicities.
• Double-blind, randomized, placebo-controlled part:
Overall Survival
Arms/Intervention
• canakinumab in combination with docetaxel
• canakinumab matching-placebo in combination with
docetaxel
Target Patients
Patients with:
• Stage IIIB or IV NSCLCwithout EGFR, ALK, ROS-1 or B-
RAF mutation
• Previously treated with platinum therapy and PD(L)1-
inhibitor
Expected Completion 2021
Publication TBD
BYL719 - Alpha-specific PI3K inhibitor
84
Study NCT02437318 SOLAR-1 (CBYL719C2301)
Indication HR+ advanced breast cancer
Phase Phase 3
Patients 572
Primary Outcome
Measures
Progression-free survival (PFS) for patients with PIK3CA
mutant status
Arms/Intervention• Fulvestrant 500 mg + alpelisib 300 mg
• Fulvestrant 500 mg + placebo
Target Patients
Men and postmenopausal women with hormone receptor
positive, HER2-negative advanced breast cancer which
progressed on or after aromatase inhibitor treatment
Expected Completion Q3-2018 (actual)
Publication
• Andre F, et al. Presentation at ESMO 2018
• Andre et al. Manuscript N Engl J Med 2019;380:1929-
1940.
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Exjade® - Iron chelation of bis-hydroxy-phenyl triazole type
85
Study NCT00940602 TELESTO (CICL670A2302)
Indication Iron overload
Phase Phase 2
Patients 224
Primary Outcome
Measures
To compare deferasirox to placebo with regard to event-free
survival in low and int-1 risk MDS patient with transfusional
iron overload
Arms/Intervention• Deferasirox, iron chelator
• Placebo
Target PatientsPatients with myelodysplastic syndromes (low/int-1 risk) and
transfusional iron overload
Expected Completion Q3-2018 (actual)
Publication• Angelucci E, et al. Presentation at ASH 2018
• Angelucci E, et al. Manuscript submitted Q3-2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
INC280 - MET Inhibitor
86
Study NCT02414139 (CINC280A2201) NCT03647488 (CINC280D2201)
IndicationEGFR Wild-type, ALK negative advanced Non-small Cell
Lung Cancer (NSCLC)Non-small cell lung cancer
Phase Phase 2 Phase 2
Patients 364 105
Primary Outcome
MeasuresOverall Response Rate (ORR)
Run in part: Assess safety and tolerability of capmatinib and
spartalizumab combination.
Randomized part: Overall Survival (OS)
Arms/Intervention
• Pre-treated pts. with MET GCN: ≥ 6; ≥ 4 and < 6; <
4
• Pre-treated pts. with MET mutations regardless of
cMET GCN as second or third line
• Treatment-naïve pts. with MET dysregulation
• Pre-treated pts with MET dysregulation – second
line
• Treatment-naïve pts with cMET mutations
regardless of cMET GCN
• Capmatinib plus spartalizumab
• Docetaxel
Target Patients
Adult patients with EGFR wild-type (wt), ALK-negative
advanced/ metastatic NSCLC with either MET
amplification or MET mutations
Pre-treated adult patients with EGFR wild-type ALK
rearrangement negative advanced/metastatic non-small cell
lung cancer, that has demonstrated progression following one
prior platinum doublet and one prior PD-(L)1 checkpoint
inhibitor
Expected Completion Q2-2019 (actual) 2021
Publication• Wolf J, et al. Presented at ASCO 2019
• Manuscript submission in H2-2019 (journal TBD)TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Jakavi® - JAK1/2 inhibitor
87
Study NCT02913261 REACH2 (CINC424C2301) NCT03112603 REACH3 (CINC424D2301)
Indication Steroid-refractory acute graft vs. Host disease (SR aGVHD) Steroid-refractory chronic graft vs. Host disease (SR cGVHD)
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 (actual) Q3-2019 (actual)
Publication • Manuscript submission in Q4-2019 (journal TBD) • Manuscript submission in H1-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Jakavi® - JAK1/2 inhibitor
88
Study NCT03491215 REACH4 (CINC424F12201) NCT04097821 ADORE (CINC424H12201)
Indication Graft versus host disease Myelofibrosis
Phase Phase 2 Phase 1/2
Patients 45 130
Primary Outcome
Measures
• Measurement of PK parameters
• Overall Response Rate (ORR)
• Incidence of dose limiting toxicities within the first 2
cycles
• Response rate at the end of cycle 6
Arms/Intervention • Ruxolitinib
• Ruxolitinib
• Ruxolitinib+Siremadlin
• Ruxolitinib+Crizanlizumab
• Ruxolitinib+MBG453
Target PatientsPediatric patients with grade II-IV acute graft vs. host disease
after allogeneic hematopoietic stem cell transplantationPatients with Myelofibrosis (MF)
Expected Completion 2022 2024
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Kisqali® - CDK 4/6 inhibitor
89
Study NCT03701334 NATALEE (CLEE011O12301C)
IndicationAdjuvant treatment of hormone receptor (HR)-positive,
HER2-negative, early breast cancer (EBC).
Phase Phase 3
Patients ~4,000
Primary Outcome
Measures
Invasive Disease-Free Survival for using STEEP criteria
(Standardized Definitions for Efficacy End Points in adjuvant
breast cancer trials)
Arms/Intervention• Ribociclib + endocrine therapy
• Endocrine therapy
Target Patients
Pre and postmenopausal women and men with HR-positive,
HER2-negative EBC, after adequate surgical resection, who
are eligible for adjuvant endocrine therapy
Expected Completion 2025
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Kymriah® – CAR-T therapy
90
Study NCT02445248 JULIET (CCTL019C2201) NCT03568461 ELARA (CCTL019E2202)
Indication Relapsed / refractory DLBCL Relapsed / refractory follicular lymphoma (FL)
Phase Phase 2 Phase 2
Patients 128 113
Primary Outcome
MeasuresOverall response rate; efficacy and safety of CTL019 Complete Response Rate (CRR)
Arms/Intervention Single-arm study of single dose of CTL019 Single-arm study of tisagenlecleucel
Target PatientsAdult patients with relapsed or refractory diffuse large B-cell
lymphoma (DLBCL)Adult patients with relapsed or refractory FL
Expected Completion 2017 (actual) H2-2020 (interim analysis)
Publication
• Schuster et al. Presentations at ICML 2017; at EHA
2017; at ASH 2017; at ASH 2018; Borchmann et al.
Presentation at EHA 2018; Bachanova et al.
Presentation at ICML 2019
• Schuster et al. N Engl J Med. 2019;380(1):45-56. doi:
10.1056/NEJMoa1804980. Epub 2018 Dec 1.
TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Kymriah® – CAR-T therapy
91
Study NCT03876769 CASSIOPEIA (CCTL019G2201J) NCT03570892 BELINDA (CCTL019H2301)
Indication 1st line high risk acute lymphoblastic leukemia (ALL) 2nd line Diffuse large B-cell lymphoma (DLBCL)
Phase Phase 2 Phase 3
Patients 160 318
Primary Outcome
Measures5 year Disease Free Survival (DFS) Event-free Survival (EFS)
Arms/Intervention Single-arm study of tisagenlecleucel; retreatment allowed Tisagenlecleucel versus standard of care
Target Patients Pediatric and young adult patients with 1st line high risk ALL
Adult patients with aggressive B-cell Non-Hodgkin
Lymphoma after failure of rituximab and anthracycline-
containing frontline immunochemotherapy
Expected Completion 2025 2021
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
MBG453 – Tim-3 antagonist
92
Study NCT03946670 (CMBG453B12201)
Indication Myelodysplastic syndrome
Phase Phase 2
Patients 120
Primary Outcome
Measures
Complete Remission (CR) rate and Progression Free
Survival (PFS)
Arms/Intervention• Experimental: MBG453 + hypomethylating agents
• Placebo comparator: Placebo + hypomethylating agents
Target PatientsAdult subjects with intermediate, high or very high risk
Myelodysplastic Syndrome (MDS) as per IPSS-R criteria
Expected Completion 2021
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
PDR001 – PD-1 checkpoint inhibitor
93
Study NCT02967692 COMBI-i (CPDR001F2301)
Indication BRAFV600 mutant metastatic melanoma
Phase Phase 3
Patients
538
Part 1 (safety-run in): 9; Part 2 (biomarker cohort): 27; Part 3
(Phase III, randomized, placebo controlled): 532
Primary Outcome
MeasuresProgression-Free Survival (PFS)
Arms/Intervention
• Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg bid +
Mekinist 2 mg
• Placebo + Tafinlar 150 mg bid + Mekinist 2 mg
Target Patients
Previously untreated patients with unresectable or
metastatic BRAF V600 mutant melanoma
Expected Completion H2-2019 (IA); H2-2020 (Final analysis)
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Rydapt®- Multi-targeted kinase inhibitor
94
Study NCT03280030 (CPKC412A2220) NCT03591510 (CPKC412A2218)
Indication Acute myeloid leukemia Acute myeloid leukemia
Phase Phase 2 Phase 2
Patients 66 50
Primary Outcome
MeasuresIncidence of safety events and event free survival
Occurrence of dose limiting toxicities
Event Free Survival ( EFS)
Arms/Intervention• Midostaurin 50 mg
• Placebo• Chemotherapy followed by Midostaurin
Target PatientsNewly diagnosed patients with FLT3-mutated acute myeloid
leukemia (AML)
Newly diagnosed pediatric patients with FLT3 mutated acute
myeloid leukemia (AML)
Expected Completion H1-2020 H2-2022
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
PDR001 - PD-1 checkpoint inhibitor
Study NCT03484923 (CPDR001J2201)
Indication Previously treated unresectable or metastatic melanoma
Phase Phase 2
Patients 230
Primary Outcome
MeasuresObjective Response Rate (ORR)
Arms/Intervention
• PDR001 400mg i.v. Q4W + LAG525 600 mg i.v. Q4W
• PDR001 400mg i.v. Q4W + capmatinib 400 mg bid orally
• PDR001 400mg i.v. Q4W + canakinumab 300 mg (s.c)
Q4W
• PDR001 400mg i.v. Q4W + ribociclib 600 mg p.o QD on
Days 1 to 21 of a 28-day cycle
Target PatientsAdult patients with previously treated unresectable or
metastatic melanoma
Expected Completion 2021
Publication Abstract submission to congress in H1-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
95
Promacta®/Revolade® – Thrombopoetin receptor agonist
Study NCT03025698 (CETB115E2201)
IndicationPreviously untreated or relapsed/refractory severe aplastic anemia or
recurrent aplastic anemia
Phase Phase 2
Patients 60
Primary Outcome
MeasuresPK of eltrombopag at steady state in pediatric patients with SAA
Arms/Intervention
• Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS
• Arm B: previously untreated SAA-hATG/cyclosporine +
eltrombopag
• Arm A: relapsed/refractory SAA or AA: hATG/cyclosporine +
eltrombopag or cyclosporine + eltrombopag
Target PatientsPediatric patients from age 1 <18 years with relapsed/refractory SAA
or recurrent AA after IST or previously untreated SAA
Expected Completion 2025
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
96
SEG101 – p-Selectin inhibitor
Study NCT03264989 SOLACE-Adults (CSEG101A2202) NCT03474965 SOLACE-Kids (CSEG101B2201)
IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with
Sickle Cell Disease (SCD) Prevention of VOC in pediatric patients with SCD
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)H2-2021 (pediatric patients ≥6 year old)
2022 (pediatric patients 6 months – 6 year old)
Publication Abstract submission to congress in H1-2020 TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
97
SEG101 – p-Selectin inhibitor
Study NCT03814746 STAND (CSEG101A2301)
IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with
Sickle Cell Disease (SCD)
Phase Phase 3
Patients 240
Primary Outcome
MeasuresRate of VOC events leading to healthcare visit
Arms/Intervention
• Crizanlizumab 5.0 mg/kg
• Crizanlizumab 7.5 mg/kg
• Placebo
Target Patients Adolescent and adult SCD patients (12 years and older)
Expected Completion H1-2022
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
98
Study NCT01677741 (CDRB436A2102)
Indication BRAFV600 mutant cancers
Phase Phase 1/2
Patients 85
Primary Outcome
MeasuresSafety, tolerability and pharmacokinetics
Arms/InterventionSingle-arm study of oral dabrafenib (dose based on age
and weight)
Target PatientsPediatric subjects aged 1 year to <18 years with advanced
BRAF V600-mutation positive solid tumors
Expected Completion Q1-2020
Publication
• Kieran MW et al. Manuscript Clin Cancer Res; (accepted
Q3-2019, not yet published) (PK analysis)
• Hargrave D et al. Manuscript Clin Cancer Res; (accepted
Q3-2019, not yet published) (safety/efficacy in low-grade
gliomas)
Tafinlar® - BRAF inhibitor
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
99
Tafinlar®+Mekinist® - BRAF inhibitor and MEK inhibitor
Study NCT02684058 (CDRB436G2201)
Indication BRAFV600 mutant gliomas
Phase Phase 2
Patients 142
Primary Outcome
MeasuresObjective response rate
Arms/Intervention Dabrafenib + trametinib (dose based on age and weight)
Target Patients
Children and adolescent patients with BRAF V600 mutation
positive relapsed or refractory high grade glioma (HGG) or
BRAF V600 mutation positive low grade glioma (LGG)
Expected Completion 2021
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
100
Tafinlar®+Mekinist® - BRAFV600 inhibitor and MEK inhibitor
Study NCT02124772 (CTMT212X2101)
Indication BRAFV600 mutant solid tumors
Phase Phase 1
Patients 142
Primary Outcome
MeasuresSafety, tolerability and pharmacokinetics and clinical activity
Arms/InterventionTrametinib (dose based on age and weight)
Dabrafenib + trametinib (dose based on age and weight)
Target PatientsPediatric Subjects Aged 1 Month to <18 Years with
Advanced V600-Mutation Positive Solid Tumors
Expected Completion 2021
Publication Abstract submission to congress in H1-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
101
Zykadia® - ALK inhibitor
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
102
Study NCT02299505 ASCEND-8 (CLDK378A2112)
Indication ALK activated NSCLC
Phase Phase 2
Patients 306
Primary Outcome
Measures
Part 1: Pharmacokinetics when taken with food
Part 2: Overall Response Rate (ORR) when taken with food
Arms/Intervention
• Oral LDK378 450 mg once daily taken with food
• Oral LDK378 600 mg once daily taken with food
• Oral LDK378 750 mg once daily fasted
Target PatientsAdult patients with ALK-rearranged (ALK-positive) advanced non-small cell
lung cancer
Expected Completion
Part 1 (PK): 2016 (actual)
Part 2 (ORR): Q2-2018 (actual)
Final (ORR): Q4-2019
Publication
• Part 1 (PK): Cho BC, et al. J Thorac Oncol. 2017 Sep; 12(9) 1357-1367
• Part 2 (ORR): Cho B et al. J Thorac Oncol. 2019 Jul; 14(7) 1255-1265
• Final (ORR): TBD
177Lu-PSMA-617 – Lu-labelled prostate specific membrane antigen (PSMA)
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
103
Study NCT03511664 VISION (PSMA-617-01)
IndicationPSMA-positive Metastatic Castration-resistant Prostate
Cancer (mCRPC)
Phase Phase 3
Patients 750
Primary Outcome
Measures
• Radiographic Progression Free Survival
• Overall Survival
Arms/Intervention• 177Lu-PSMA-617 plus BS/BSC
• BS/BSC alone
Target Patients
Adult patients with PSMA-positive Metastatic Castration-
resistant Prostate Cancer (mCRPC)
Expected Completion H1 2020
Publication TBD
Lucentis® - Anti-VEGF
105
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
Absence of active Retinopathy of Prematurity (ROP) and
unfavorable structural outcome at Week 24, defined as, 1)
survival, 2) no intervention with a second modality for ROP,
3) absence of active ROP and 4) absence of unfavorable
structural outcome
To evaluate the visual function of patients by assessing the
visual acuity in the better-seeing eye at the patient’s fifth
birthday.
Arms/Intervention
• Ranibizumab 0.2 mg (up to 3 injections max)
• Ranibizumab 0.1 mg (up to 3 injections max)
• Laser therapy
• Ranibizumab 0.2 mg (up to Week 40, if warranted)
• Ranibizumab 0.1 mg (up to Week 40, if warranted)
Target PatientsMale and female preterm infants with bilateral retinopathy of
prematurity (ROP) who require treatment.
Male and female preterm infants with bilateral retinopathy of
prematurity (ROP) who completed RAINBOW.
Expected Completion Q1-2018 (actual) 2023
Publication
• EURETINA: Sep-2018
• AAO: Oct-2018
• Primary manuscript published online by The Lancet in
Sep-2019
(https://www.thelancet.com/pdfs/journals/lancet/PIIS0140
-6736(19)31344-3.pdf)
TBD
| Novartis Q3 2019 Results | October 22, 2019 | 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)
• Year 1 Manuscript: Dugel P, et al. Ophthalmology 2019 Apr 12; HAWK and HARRIER: Phase 3, Multicenter,
Randomized, Double-Masked Trials of Brolucizumab for Neovascular Age-Related Macular Degeneration.
• Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses
(Angiogenesis/Mac Soc in Feb-2019; WRC in Mar-2019; ARVO in April-2019; ASRC July-2019; EURETINA Sept-2019;
AAO Oct-2019 and APVRS Dec-2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
106
RTH258 - Anti-VEGF
Study NCT03386474 (CRTH258A2301E1) NCT03481634 KESTREL (CRTH258B2301)
Indication Neovascular age-related macular degeneration (nAMD) Diabetic eye disease
Phase Phase 3 Phase 3
Patients 150 534
Primary Outcome
MeasuresNumber of treatment-emergent adverse events
Change from baseline in best-corrected visual acuity
(BCVA)
Arms/Intervention• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
• RTH258 3 mg/50 µL
• RTH258 6 mg/50 µL
• Aflibercept 2mg/50 uL
Target PatientsPatients with neovascular age-related macular degeneration
who have completed the CRTH258A2301 study
Patients with visual impairment due to diabetic macular
edema (DME)
Expected Completion Q3-2018 (actual) 2021
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
107
RTH258 - Anti-VEGF
Study NCT03481660 KITE (CRTH258B2302) NCT04058067 KINGLET (CRTH258B2304)
Indication Diabetic eye disease Diabetic macular edema
Phase Phase 3 Phase 3
Patients 356 268
Primary Outcome
Measures
Change from baseline in best-corrected visual acuity
(BCVA)Change in best-corrected visual acuity (BCVA)
Arms/Intervention• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
• Brolucizumab 6 mg
• Aflibercept 2 mg
Target PatientsPatients with visual impairment due to diabetic macular
edema (DME)
Patients with visual impairment due to diabetic macular
edema
Expected Completion 2021 2022
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
108
RTH258 - Anti-VEGF
Study NCT03917472 (CRTH258B2305) NCT03802630 RAPTOR (CRTH258C2301)
Indication Diabetic macular edema Retinal vein occlusion
Phase Phase 3 Phase 3
Patients 500 500
Primary Outcome
Measures
Change in best-corrected visual acuity (BCVA) from
baseline up to week 52
Change from baseline in best-corrected visual acuity
(BCVA) at week 24
Arms/Intervention• Brolucizumab (RTH258) 6 mg/0.05 mL every 4 weeks
• Aflibercept 2 mg/0.05 mL every 4 weeks
• Brolucizumab 6 mg every 4 weeks
• Aflibercept 2 mg every 4 weeks
Target PatientsPatients with visual impairment due to diabetic macular
edema
Adult patients with visual impairment due to macular edema
secondary to branch retinal vein occlusion
Expected Completion 2021 2022
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
109
RTH258 - Anti-VEGF
Study NCT03810313 RAVEN (CRTH258C2302)
Indication Retinal vein occlusion
Phase Phase 3
Patients 750
Primary Outcome
Measures
Change from baseline in best-corrected visual acuity
(BCVA) at week 24
Arms/Intervention• Brolucizumab 6 mg every 4 weeks
• Aflibercept 2 mg every 4 weeks
Target PatientsAdult patients with visual impairment due to macular edema
secondary to central retinal vein occlusion
Expected Completion 2023
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
110
UNR844 - Disulfide bonds modulator
Study NCT03809611 (CUNR844A2203)
Indication Presbyopia
Phase Phase 2
Patients 120
Primary Outcome
Measures
Change in binocular distance-corrected near visual acuity
(DNCVA) from baseline
Arms/Intervention• 1.5% solution UNR844-Cl
• Placebo
Target Patients Patients with presbyopia
Expected Completion Q4-2019
Publication Planned in ASRCS in 2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
111
QAW039 – DP2 receptor antagonist
113
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 Q4-2019 Q3-2019
Publication Planned in 2020 Planned in 2020
| Novartis Q3 2019 Results | October 22, 2019 | 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 Q3-2019 Q3-2019
Publication Planned in 2020 Planned in 2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
114
QAW039 – DP2 receptor antagonist
Study NCT03052517 SPIRIT (CQAW039A2315) NCT03650400 (CQAW039B2201)
Indication Asthma Asthma
Phase Phase 3 Phase 2
Patients 1,900 – 2,300 24
Primary Outcome
Measures
Long term safety: treatment emergent adverse event (AE),
SAE and AE leading to discontinuation from study (52 wks
and 160 wks)
Pharmacokinetics, safety and tolerability
Arms/Intervention
• QAW039 Dose 1
• QAW039 Dose 2
• Placebo
• Fevipiprant Cohort A; Fevipiprant Cohort B; Chewable
tablet
Target Patients Patients with moderate to severe asthma Children aged 6 to < 12 years with asthma
Expected Completion Q4-2019 (for submission); 2022 (final) H2-2020
Publication TBD TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
115
QBW251 - CFTR potentiator
116
Study NCT04072887 (CQBW251B2201)
Indication Chronic obstructive pulmonary disease (COPD)
Phase Phase 2
Patients 900
Primary Outcome
Measures
Trough FEV1 (Forced Expiratory Volume in 1 second)
change from baseline after 12 weeks of treatment
Arms/Intervention
• QBW251 450 mg
• QBW251 300 mg
• QBW251 150 mg
• QBW251 75 mg
• QBW251 25 mg
• Placebo
Target PatientsCOPD patients on background triple inhaled therapy (LABA /
LAMA / ICS)
Expected Completion 2021
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
QMF149 - Long-acting beta2 agonist and inhaled corticosteroid
117
Study NCT02892019 (CQMF149G2202)
Indication Asthma
Phase Phase 2
Patients 80
Primary Outcome
MeasuresTrough FEV1
Arms/Intervention• Indacaterol acetate 75 μg od (via Concept1 inhaler)
• Indacaterol acetate 150 μg od (via Concept1 inhaler)
Target Patients Children ≥ 6 to < 12 years of age with asthma
Expected Completion Q3-2019
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid
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 asthma
inadequately controlled on medium/high-dose ICS or low-
dose LABA/ICS (GINA step ≥ 3)
Adult (≥18 years) patients with asthma inadequately
controlled on medium/high-dose of LABA/ICS (GINA step ≥4)
Expected Completion Q3-2019 Q3-2019
Publication• Planned in H1-2020
• Planned abstract for BTS in Q4-2019Planned in H1-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
118
QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid
119
Study NCT03100500 (CQVM149B1305) NCT03100825 (CQVM149B1304)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 51 94
Primary Outcome
Measures
Long-term safety/tolerability: Incidence and severity of
treatment emergent adverse events during the 52 weeks
study
Long-term safety/tolerability: Incidence and severity of
treatment emergent adverse events during the 52 weeks
study
Arms/Intervention • Single arm: QMF149 150/320 μg od • Single Arm: QVM149 150/50/160 μg od
Target Patients Japanese patients with asthma inadequately controlled Japanese patients with asthma inadequately controlled
Expected Completion Q1-2019 (actual) Q2-2019 (actual)
Publication• Planned in H1-2020
• Planned abstract for ATS in 2020
• Planned in H1-2020
• Planned abstract for ATS in 2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid
Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 802 1,251
Primary Outcome
MeasuresTrough FEV1
Non-inferiority of Asthma Quality of Life Questionnaire
(AQLQ)
Arms/Intervention• QMF149 150/80 µg od
• MF 200 µg od
• QVM149 150/50/80 μg od
• QVM149 150/50/160 μg od
• Salmeterol/fluticasone 50/500 μg bid + tiotropium 5 μg od
Target Patients
Adult and adolescent (≥12 years) patients with mild asthma
inadequately controlled on low-dose ICS or low-dose
LABA/ICS (Gina step 2-3)
Patients with uncontrolled asthma
Expected Completion Q1-2019 (actual) Q3-2019
Publication Planned in Q1-2020 Planned in H1-2020
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
120
Xolair® – anti-IgE antibody
121
Study NCT03369704 (CIGE025F1301)
Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis
Phase Phase 3
Patients 337
Primary Outcome
MeasuresMean nasal symptom score, consists of severity of sneezing, rhinorrhea and nasal congestion.
Arms/Intervention
In addition to standard of care:
• Omalizumab per approved allergic asthma dosing table for IgE/body weight combinations
• Placebo
Target PatientsPatients with severe Japanese cedar pollinosis, whose symptoms were inadequately controlled with current
recommended therapies
Expected Completion Q1-2019 (actual)
Publication
• Late breaking abstract was published at AAAAI (American Association of Allergy, Asthma and
Immunology) annual meeting, Feb-2019.
• Oral/poster published at EAACI (the European Academy of Allergy and Clinical Immunology), Jun-2019
• Planned oral/poster to be submitted for JRS (Japanese Rhinologic Society) in Oct-2019
• Planned manuscript to be submitted to JACI (The Journal of Allergy and Clinical Immunology) in Q4-2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Hyrimoz® - Biosimilar adalimumab
123
Study NCT02744755 ADMYRA (GP17-302)
Indication Immunology
Phase Phase 3
Patients 353
Primary Outcome
Measures
Change in DAS28-CRP score from baseline to week 12 in
patients treated with GP2017 and patients treated with
Humira®
Arms/Intervention• GP2017
• US licensed Humira® adalimumab
Target Patients Patients with moderate to severe active rheumatoid arthritis
Expected Completion 2018 (actual)
Publication• Wiland, P. et al., presented at EULAR 2019
• Wiland, P. et al., BioDrugs, Q4 2019
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
GP2411 - Biosimilar denosumab
124
Study NCT03974100 (CGP24112301)
Indication Osteoporosis
Phase Phase 3
Patients 522
Primary Outcome
Measures
Percent change from baseline (%CfB) in lumbar spine Bone
Mineral Density
Arms/Intervention
• GP2411 60 mg /mL subcutaneous injection every 6
months
• Prolia® 60 mg /mL subcutaneous injection every 6
months
Target Patients Postmenopausal women with osteoporosis
Expected Completion 2022
Publication Study data publications expected for 2024 and beyond
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated
126
Study NCT03167242 (CKAF156A2202)
Indication Malaria
Phase Phase 2
Patients 512
Primary Outcome
Measures
PCR-corrected adequate clinical and parasitological
response (ACPR)
Arms/Intervention• KAF156 and LUM-SDF (different combinations)
• Coartem
Target PatientsAdults and children with uncomplicated Plasmodium
Falciparum Malaria
Expected Completion H1-2021
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
KAE609 – Plasmodium Falciparum Inhibitor – spiroindolone against PfATP4
127
Study NCT03334747 (CKAE609A2202)
Indication Malaria
Phase Phase 2
Patients 210
Primary Outcome
Measures
CTCAE grades increase from baseline in alanine
aminotransferase (ALT) or aspartate aminotransferase
(AST)
Arms/Intervention• KAE609
• Coartem
Target Patients Adults with uncomplicated Plasmodium Falciparum malaria
Expected Completion Q4-2020
Publication TBD
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation
Key definitions and trademarks
| Novartis Q3 2019 Results | October 22, 2019 | Investor Presentation 128
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