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Q3 2018 Results
Investor Presentation
October 18, 2018
Novartis AG
Investor Relations
Disclaimer
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 2
This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as “potential,”
“expected,” “will,” “planned,” “pipeline,” “outlook,” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, or regarding
potential future revenues from any such products; or regarding the potential financial or other impact on Novartis, and the potential strategic benefits, synergies or opportunities expected as a result of the
proposed spinoff of our Alcon Division or of the proposed divestiture of certain portions of our Sandoz Division in the US or of the proposed acquisition of Endocyte; or regarding potential future sales or
earnings of the Novartis Group or any of its divisions or potential shareholder returns; or by discussions of strategy, plans, expectations or intentions. You should not place undue reliance on these statements.
Such forward looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of
these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward looking statements. There can be no guarantee
that any new products will be approved for sale in any market, or that any new indications will be approved for any existing products in any market, or that any approvals which are obtained will be obtained at
any particular time, or that any such products will achieve any particular revenue levels. Neither can there be any guarantee that the proposed spinoff of the Alcon Division, or the proposed divestiture of certain
portions of our Sandoz Division in the US or the proposed acquisition of Endocyte will receive necessary approvals, or that they will be completed, or completed as currently proposed, or at any particular time.
Nor can there be any guarantee that Novartis will be able to realize any of the potential strategic benefits, synergies or opportunities as a result of the proposed transactions. Nor can there be any guarantee
that shareholders will achieve any particular level of shareholder returns. Neither can there be any guarantee that the Group, or any of its divisions, will be commercially successful in the future, or achieve any
particular credit rating or financial results. In particular, our expectations could be affected by, among other things: global trends toward health care cost containment, including 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 development of the products described in this release or with respect to the proposed transactions; the potential that the strategic benefits, synergies or opportunities expected from the
proposed transactions may not be realized or may take longer to realize than expected; the inherent uncertainties involved in predicting shareholder returns; the uncertainties inherent in the research and
development of new healthcare products, including clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain proprietary intellectual property protection, including the
ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity on key products which commenced in prior years and will continue this year; safety, quality or manufacturing issues;
uncertainties regarding actual or potential legal proceedings, including, among others, actual or potential litigations with respect to the proposed transactions, product liability litigation, litigation and
investigations regarding sales and marketing practices, intellectual property disputes and government investigations generally; uncertainties involved in the development or adoption of potentially
transformational technologies and business models; general political and economic conditions, including uncertainties regarding the effects of ongoing instability in various parts of the world; uncertainties
regarding future global exchange rates; uncertainties regarding future demand for our products; and 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.
Additional Information and Where to Find It
This communication may be deemed to be solicitation material in respect of the proposed acquisition of Endocyte by Novartis AG. In connection with the proposed acquisition, Endocyte intends to file relevant
materials with the SEC, including a proxy statement in preliminary and definitive form. Stockholders of Endocyte are urged to read these materials (including any amendments or supplements thereto)
and all other relevant documents filed with the SEC when such documents become available, including Endocyte’s definitive proxy statement, because they will contain important information
about the proposed acquisition. Investors and security holders are able to obtain the documents (once available) free of charge at the SEC’s web site, http://www.sec.gov, or from Endocyte by going to its
investor relations web site at http://investor.endocyte.com/investor-relations
Participants in Solicitation
Novartis AG and its directors and executive officers, and Endocyte and its directors and executive officers, may be deemed to be participants in the solicitation of proxies from the holders of Endocyte shares of
common stock in respect of the proposed acquisition. Information about the directors and executive officers of Novartis AG is set forth in the excerpts of Novartis AG’s Annual Report for 2017, which was
furnished to the SEC on Form 6-K on January 24, 2018 and incorporated by reference into Novartis AG’s Annual Report on Form 20-F for the fiscal year ended December 31, 2017. Information about the
directors and executive officers of Endocyte is set forth in the proxy statement for Endocyte’s 2018 Annual Meeting of Stockholders, which was filed with the SEC on March 23, 2018. Information regarding
interests of Novartis AG’s and Endocyte’s respective participants in the solicitation, will be set forth in the proxy statement relating to the proposed acquisition and other materials to be filed with the SEC in
connection with the proposed acquisition.
Agenda
1 Group review Vas Narasimhan
2 Financial review Harry Kirsch
3 Closing Vas Narasimhan
4 Q&A Team
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 3
Delivered strong accretive growth in Q3, with key
innovation milestones on advanced therapy platforms
4 Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Group performance, % cc
Sales Core OpInc
+6%
+9%
Innovation milestones
AVXS-101
BAF312
Endocyte1
1. The acquisition of Endocyte is subject to customary closing conditions, including receipt of regulatory approvals and Endocyte stockholders approval. Until closing, Endocyte will continue to operate as a separate and independent company
Q3 9M
SalesUSD million
Growth vs. PYUSD million cc
SalesUSD million
Growth vs. PYcc
750 37% 2,031 37%
271 113% 710 117%
295 32% 844 37%
291 33% 842 31%
Lutathera® 56 nm1 86 nm1
248 27% 721 27%
72 184% 175 nm1
20 nm1 48 nm1
56
67
46
194
47
143
68
20
Strong performance driven by Innovative Medicines
5 Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
1. Not meaningful
Key growth drivers
Cosentyx®
established as leading differentiated
IL-17A inhibitor
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 6
Sales evolutionUSD million, % cc
Q3 2017 Q3 2018
556
750Ex-US
US
+37%
1. Humira is a registered trademark of AbbVie Inc. 2. TRx Share in Rheum specialty, IMS TRx allocated using SHS APLD (June 2018) factors where only PsO/PsA/AS uses are carved in for TNFs
Q3 sales USD 750m (+37% cc)
Strong growth momentum across all indications
and geographies
Cosentyx® surpassed Humira®1 in TRx in PsA2
in US
Five year data in PsA and AS show sustained
benefits, complementing long-term data in
psoriasis
Cosentyx®
gaining share in a growing and
competitive psoriasis market
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 7
10%
13%
12M ending 7/2017 12M ending 7/2018
+31%
Cosentyx® OthersStelara® TNFs Tremfya®
Dermatology US NRx evolution1
Cosentyx® prescriptions (NRx) +64%1 in a
growing US biologics market (+31%)1
Cosentyx® effectively treats the multiple
manifestations of the psoriatic disease
2/3 of patients have - beyond plaque psoriasis -
scalp, nail, palmoplantar and/or joint involvement2
Cosentyx® real-world evidence confirms safety
and efficacy benefits
1. NRx Share in Dermatology from SHS June 2018. NRx Share amongst Dermatology writers 2. Data on File. Corrona LLC. Corrona Report: Real-World Data From the Corrona Psoriasis Registry® June 15, 2018
All trademarks are the property of their respective owners
Entresto®
sales driven by strong uptake in all
launched markets
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 8
Q3 US sales USD 151m (+104% cc),
ex-US sales USD 120m (+126% cc)
TRANSITION data show Entresto® can be
initiated early and safely in hospitalized patients1
PIONEER study to be presented as late-breaker
at AHA in November
PARAGON study continues as planned following
interim analysis, results expected mid-2019
Sales evolutionUSD million, % cc
Q3 2017 Q3 2018
128
271
+113%
1. Wachter R. et al., Initiation of sacubitril/valsartan in hospitalized patients with reduced ejection fraction heart failure after hemodynamic stabilization: Primary results of TRANSITION study. Data presented at: ESC 2018, Aug 25-29
Ex-US
US
Lutathera®
off to a strong start in US and gaining
reimbursement in Europe
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 9
Strong launch momentum with Q3 sales of
USD 56m (YTD sales of USD 86m)
>1,100 patients in US October YTD1
>85 centers in US actively prescribing
Broad US payer coverage with 70% of lives
covered
2 months after UK reimbursement, 18 centers
actively prescribing52
399
1'123
Q2 2018Q1 2018 Q3 2018
1. Data as of October 16, 2018
US – number of doses
Q3 2018 performance
Sandoz continuing to execute strategy of focusing on
complex generics and biosimilars
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 10
Sandoz sales -4% (cc) as US sales
declined -17% (cc) due to continued
industry-wide pricing pressure
Ex-US sales grew +2% (cc)
Global Biopharmaceuticals grew +21% (cc)
Optimizing our core Gx business:
Divesting US oral solids and dermatology
(~USD 0.6bn sales in H1 2018) to Aurobindo1
Expanding biosimilars in Europe:
Ramping up Rixathon® and Erelzi®
Approval for Hyrimoz® and Zessly®
Positive CHMP opinion for pegfilgrastim
Driving portfolio differentiation
1. Announced on September 6th, 2018 agreement to sell selected portions of Sandoz US portfolio (US dermatology business and US oral solids portfolio) to Aurobindo Pharma USA Inc; expected completion 2019
11
Continued momentum with Q3 sales +5% (cc):
– Surgical +7% (cc) mainly AT-IOLs and
consumables
– Vision Care +3% (cc) with double digit
growth of Dailies Total1® and Systane ®
Q3 core operating income grew +1% (cc),
reflecting impact of growth investments
YTD core operating income grew +14% (cc),
core ROS 18.6% (+1.3% pts)
Alcon Q3 net salesUSD million, % cc
788797
Q3 2017 Q3 2018
+3%
924
966
Q3 2017 Q3 2018
+7%
Surgical Vision Care
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Alcon continues to show solid sales growth as a result
of improved operations and customer relationships
Key data readouts and regulatory milestones in Q3
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 12
ADHF – acute decompensated heart failure Copaxone® is a registered trademark of Teva Pharmaceuticals LTD 1. Submission in Japan initiated
Advanced
therapy
platforms
AVXS-101
SMA Type 1
New analysis reiterates rapid onset of action (WMS 2018)
Simultaneous global submissions in US, EU, and Japan1
Kymriah® EU approvals in pALL and DLBCL
Luxturna® CHMP positive opinion
Potential
first-in-class
therapies
BAF312 Filed for SPMS in US and EU
BYL719 SOLAR-1 study met its primary endpoint, ESMO 2018 presentation
Driving
in-line
brands
Gilenya® Superiority vs. Copaxone® (ASSESS study)
Entresto® HFrEF: PIONEER-HF data to be presented at AHA
HFrEF: TRANSITION demonstrated initiation of Entresto® shortly after stabilization of ADHF well tolerated
HFpEF: PARAGON following interim analysis, study continue as planned with top-line results expected mid-2019
Aimovig® EU approval for migraine prevention
Kisqali® MONALEESA 3 and 7 data added to US label
Tafinlar® +
Mekinist®EU approval for adjuvant melanoma
Biosimilar
pegfilgrastim
CHMP positive opinion
ACZ885 Cardiovascular risk reduction: FDA Complete Response Letter October 2018
Speed: Rapid increase in mean CHOP-INTEND compared
to Spinraza® (nusinersen)4
AVXS-101 START demonstrated transformational
efficacy in Type I SMA (>90% unassisted sitting2)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 13
Rapid onset, sustained efficacy regardless of severity
Baseline Mean age 3.4 months; swallow function 7/12 (58%); CHOP-INTEND
28 points
Efficacy 15/15 (100%) alive and without need for permanent ventilation at 24
months4
9/10 (90%) achieved CHOP-INTEND ≥ 40 by 8 months1
11/12 (92%) achieved CHOP-INTEND >50 during the 24-month study2
11/12 (92%) achieved sitting unassisted during the 24-month study
Durability Continue to achieve major motor milestones with gene therapy alone
during long-term follow-up study2,3
Data is from Cohort 2 in clinical trial, final analysis N=12 1. At day 245 (8 mos), 10 out of 12 Cohort 2 patients in pivotal study had an assessment 2. Two-year data presented as an oral presentation at AAN 2018 3. Data on file. LTFU
data presented as an oral presentation at AAN 2018 4. Poster P.182 at WMS 2018 Spinraza® is a registered trademark of Biogen MA Inc.
Simultaneous regulatory license applications show
significant progress for AVXS-101 in SMA Type 1
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 14
Initiated submission in mid-September, anticipate completion by year end
Potential approval in 1H 2019
Sakigake designation
Japan
BLA submitted to FDA at the end of Q3
Potential approval in 1H 2019 (6 months from file acceptance)
Breakthrough Therapy designation
US
MAA submitted to EMA in early Q4
Potential approval in mid-2019 (7 months from file acceptance)
PRIME designation
EU
AVXS-101 clinical development program continues to
enroll rapidly
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 15
2014-2017 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Q1 2019 Completion3
SMA
Type 1
START (CL-101)1
(n=15; Open Label, Dose
Comparison, Single i.v. dose)
2033
2020
2020
SMA
Type 2
2019
Pre-symptomatic SMA
Type 1,2,3
2020-20234
Pediatric SMA
Type 1,2,3
TBC
STR1VE (CL-303 / NCT03306277) - Single i.v. dose
Initiated in Q4 2017, patient enrollment complete
STR1VE EU (CL-302 / NCT03461289) – Single i.v. dose
Initiated Q2 2018, 6 patients enrolled
STRONG (CL-102 / NCT03381729) - Dose Comparison, Single i.t. dose
Initiated in Q1 2018, patient enrollment complete
SPR1NT (CL-304 / NCT03505099) Single i.v. dose
Initiated Q2 2018, 6 patients enrolled
REACHSingle i.t. dose
START Long-term follow-up (LT-001 / NCT03421977)
12 patients enrolled2
i.v. – Intravenous; i.t. – Intrathecal 1. AVXS-101-CL-101 study published in N Engl J Med 2017; 377:1713-1722 2. As of June 22, 2018 3. Expected primary completion as per clinicaltrials.gov 4. 2020 for 2 copy cohort and up to 2023 for 3
and 4 copy cohort
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 16
Novartis announces agreement to acquire Endocyte,
expanding nuclear medicines platform1
High unmet need
in prostate cancer
Prostate cancer expected to be USD 11bn market globally in 20242
177Lu-PSMA-617 expected to provide an additional treatment option, in particular for
refractory patients
First-to-market
potential in lead
product
177Lu-PSMA-617 potentially first-in-class PSMA radioligand therapy (RLT) in mCRPC;
enrollment of Ph3 initiated3
First therapeutic targeting PSMA of any kind, with potential applications for soft tissue,
bone disease and prostate cancer
Significantly de-risked profile given strong Ph2 and preclinical data, and regulatory
discussions4
Expand nuclear
medicines platform
Adds potential second RLT to follow the successful launch of Lutathera®
Potential to further develop 177Lu-PSMA-617 to enter earlier lines of therapy
Potential to expand platform via 225Ac-PSMA-617
mCRPC – metastatic castration-resistant prostate cancer PSMA – prostate-specific membrane antigen 1. The acquisition of Endocyte is subject to customary closing conditions, including receipt of regulatory approvals and Endocyte
stockholders approval. Until closing, Endocyte will continue to operate as a separate and independent company 2. EvaluatePharma, October 2018 3. Endocyte announced enrollment of first patient on June 5, 2018 4. See Endocyte press
releases on June 5, 2018 and September 10, 2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 17
177Lu-PSMA-617 uses a small molecule ligand to target
a radioactive atom to PSMA-expressing cancer cells
Source: Endocyte Investor Presentation October 2018
RLT that utilizes high affinity targeting ligand to direct potent
radiotherapy to prostate cancer cells
177Lu-PSMA-617 pairs PSMA targeting ligand (PSMA-617) to
radioactive atom (177Lutetium)
“Ligand” is a small molecule designed to bind to PSMA, a protein
highly expressed on the cell surface of most prostate cancer cells
Once bound, the 177Lutetium atom releases an energetic beta
particle that kills the cancer cell
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 18
Strong Ph2 clinical data
PSA PFS50 patients
Overall SurvivalFirst 30 patients
Patients
PSA response%
Sustained response rates
in Ph2 trial expansion1
PSA PFS and OS correlate to PSA response
and compare favorably to historical benchmarks1
Months
p-value comparing PSA <50%
group to PSA ≥ 50% group;
Updated data cut-off since
Lancet publication1
Su
rviv
al
Pro
bab
ilit
y
<30%13/50 (26%)
≥30%37/50 (74%)
≥50%31/50 (62%)
50
00
-50
-100
Su
rviv
al
Pro
bab
ilit
y
Source: Endocyte Investor Presentation October 2018 1. Hofman, Michael (2018, June). Lutetium-177 PSMA617 theranostics in mCRPC: interim results of a phase 2 trial. ASCO 2018, Genitourinary cancer P5040
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 19
Pivotal Ph3 VISION trial design with FDA agreement
to rPFS as alternative primary endpoint to OS1
177Lu-PSMA-617
Choice of NAAD3
or not
Best supportive
care2
Best supportive
care2
Choice of NAAD3
or not
2:1
Stratified
for balance
Source: Endocyte Investor Presentation October 2018 1. Endocyte stated demonstrating benefit in rPFS (radiographic Progression Free Survival) versus control, with no detriment to OS, sufficient for full approval; regardless of the
outcome of rPFS assessment, Endocyte intends to continue to follow patients in VISION trial to assess final OS alternative primary endpoint as per Endocyte press release on September 10, 2018 2. Best supportive care - palliative 3.
NAAD - novel androgen axis drug (abiraterone or enzalutamide)
750 patients, enrollment
initiated
In September, FDA agreed
to rPFS as an alternative
primary endpoint to OS as
sufficient for full approval1
Key secondary endpoints:
ORR, time to symptomatic
skeletal events
Patient inclusion:
mCRPC
Bone and/or soft tissue
disease
PSMA-positive scan (~80%)
≥1 prior taxane
≥1 prior NAAD3
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 20
Transaction highlights
Financing Funded through existing cash
Financial
Benefits
Expected to contribute to Group sales in 2021
Attractive IRR
Other
Transaction unanimously approved by the Boards of Directors of both companies
Closing is subject to Endocyte stockholder approval and satisfaction of customary closing
conditions
Endocyte stockholders to receive USD 24 per share in cash
Values Endocyte at approximately USD 2.1bn on a fully diluted equity basisConsideration
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 21
Expected next steps
Endocyte to file a preliminary proxy statement with the SEC relating to a shareholders
meeting to be held to obtain shareholders approval of the transaction
Closing by H1 2019, subject to an affirmative vote by Endocyte’s stockholders to
adopt the merger agreement and satisfaction of customary closing conditions
Novartis
transaction
Publications on other ongoing investigator initiated clinical trials of 177Lu-PSMA-617 in
prostate cancer patients expected in 2018
Endocyte stated rPFS endpoint expected end 2019 and final OS endpoint end 20201
Endocyte
development and
regulatory status
1. Endocyte Investor Presentation October 2018 Note: The acquisition of Endocyte is subject to customary closing conditions, including receipt of regulatory approvals and Endocyte stockholders approval. Until closing, Endocyte will
continue to operate as a separate and independent company
Previewing R&D and investor update on November 5
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 22
Additional detailed updates
Entresto® pEF and PIONEER-HF
Cosentyx® clinical development program
QAW039 program
BAF312, OMB157, Gilenya®
RTH258 2-year data
Oncology late-stage pipeline update
Deep dive
AVXS-101
October 22, 2018: ESMO 2018 investor call including BYL719 SOLAR-1 data
Note: November 5: R&D and investor update and live audio webcast; main presentation starts at 13.00 h UK time and will finish at 17.30 h
Agenda
1 Group review Vas Narasimhan
2 Financial review Harry Kirsch
3 Closing Vas Narasimhan
4 Q&A Team
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 23
% USD % cc % USD % cc
Net Sales 12,779 3 6 38,631 7 5
Core Operating income 3,555 5 9 10,436 8 7
Operating income 1,939 -18 -13 6,870 5 3
Net Income 1,624 -22 -18 11,420 nm nm
Core EPS (USD) 1.32 2 6 3.90 7 5
EPS (USD) 0.70 -21 -17 4.92 nm nm
Free Cash Flow 3,301 8 8,778 10
Change vs. PYGroup
1
USD million
Q3
2018
9M
2018
Change vs. PY
Summary of Q3 and 9M 2018 financial results
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 24
1. Core results, constant currencies and free cash flow are non-IFRS measures. Further details regarding non-IFRS measures can be found starting on page 55 of the Condensed Interim Financial Report 2. Not meaningful due to the current
year benefit from a USD 5.7 billion net gain recognized from the sale of our stake in the GSK consumer healthcare joint venture in the second quarter
2
Innovative Medicines driving Q3 margin expansion;
9M margin increase driven by IM Division and Alcon
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 25
Q3 2018 9M 2018
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
% cc % cc % %pts cc % %pts cc
Innovative Medicines 9 16 33.7 2.1 32.4 1.1
Sandoz -4 -3 22.4 0.2 20.5 0.2
Alcon 5 1 17.1 -0.7 18.6 1.3
Group 6 9 27.8 0.8 27.0 0.5
Alcon 9M 2018 core margin at 18.6%, expanding vs.
prior year trough
26
23.8%18.6%
9M 2015 9M 2016 9M 2017 9M 2018
18.5% 17.3%
1. Alcon Division 9M 2015, 9M 2016 and 9M 2017 net sales and core margin restated to include the Ophthalmic OTC and Diagnostics products, transferred from the Innovative Medicines Division from January 1, 2018
Expected key drivers to expand core
margin to low-to-mid 20’s by 2023:
+ Improved gross margin from sales
uptake, favorable product mix and
manufacturing efficiencies
+ Cost leverage from existing
infrastructure and reallocating
resources to growth drivers
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Alcon core margin1 (% of USD sales)
5.05.1
9M 2015 9M 2016 9M 2017 9M 2018
4.9
5.4
Alcon net sales1 (USD bn)
2018 Group full year guidance: Sales revised
upwards, core operating income confirmed
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 27
Barring unforeseen events (in cc)
Full year guidanceGrowth vs. PY in cc
Group Sales revised upwards to expected to grow mid single digit
IM Division revised upwards to grow mid to high-single digit
Sandoz to decline low single digit
Alcon to grow mid single digit
Group Core operating income expected to grow mid to high single digit
Significant Fx headwinds expected in H2 and 2019
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 28
Currency impact vs. PYIn %pts, assuming mid-October exchange rates prevail in 2018
FX impact on
Net sales
FX impact on
Core operating income
Simulation
-1
62
-3 -3 -2 -1
72
-4 -5
Q2 FYFYQ4FY Q1 FYQ3 Q1 Q2
0
Q3
0
Q4 FY FY
0
-3 to -4
2017 2018
Actual
2019 2017 2018 2019
Agenda
1 Group review Vas Narasimhan
2 Financial review Harry Kirsch
3 Closing Vas Narasimhan
4 Q&A Team
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 29
Conclusion
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 30
Innovation momentum continued
On track for full year guidance
Delivered strong accretive growth
Progression of advanced therapy platform, including agreement
to acquire Endocyte
Agenda
1 Group review Vas Narasimhan
2 Financial review Harry Kirsch
3 Closing Vas Narasimhan
4 Q&A Team
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 31
Appendix
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 33
Plaque psoriasis
34% n=699
with psoriatic arthritis
38% n=784with scalp involvement
38% n=778
with palmoplantar
involvement
11% n = 223
with nail involvement
16% n = 326
2/3 of patients have – beyond plaque psoriasis –
scalp, nail, palmoplantar and/or joint involvement
1. Data on File. Corrona LLC. Corrona Report: Real-World Data From the Corrona Psoriasis Registry® June 15, 2018.
Only 34% have plaque
psoriasis alone
Gilenya 0.5mgGilenya 0.25mgCopaxone 20mg
Gilenya®
ASSESS trial shows superior efficacy over
Copaxone®
in relapsing-remitting MS
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 34
ARR - Annualized relapse rate AE - Adverse events NS – Not statistically significant 1. Post-approval commitment to FDA; randomized, controlled Ph4 study; 1064 patients 2. A formal comparison of fingolimod 0.5mg vs. 0.25mg was
not planned (study was not powered for such a comparison) Copaxone® is a registered trademark of Teva Pharmaceuticals LTD
Gilenya® 0.5mg - significant 40.7% relative reduction in
ARR vs. Copaxone®
Gilenya® 0.25mg - numerical risk reduction (NS) in ARR
vs. Copaxone®
Safety consistent with the known safety profile
Copaxone® had more discontinuations due to AE and
unsatisfactory treatment effects
Gilenya® 0.5mg is the first therapy to show superiority in reducing relapses vs.
Copaxone® in a controlled head-to-head trial1,2
Aggregate ARR up to Month 12
N = 324 N = 366 N = 345
-40.7%p=0.0138
p=NS
Key drivers vs. PY:
+ Cash flows from operating activities
− Higher net intangible investments
9M 2018 free cash flow at USD 8.8bn
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 35
8.8
9M 2017 9M 2018
8.0
+10%
Group free cash flow USD billion
YTD 2018 net debt decreased by USD 1.9bn mainly
driven by net proceeds from M&A transactions
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 36
USD billion
-19.0-17.1
-7.0 -1.4-3.5
-8.3
Dec 31, 2017 Treasury share transactions, net
Dividends AAA1
Acquisition2
0.3
AveXis, Inc. Acquisition2
13.0
Proceeds from sale of GSK consumer
healthcare joint venture
8.8
Free CashFlow
Others Sep 30, 2018
+1.9
1 Advanced Accelerator Applications S.A. 2. Net of cash acquired.
Q3 2018 Core EPS +6% cc, including the impact from
discontinuation of OTC JV core income
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 37
1.23
Q3 2017 Q3 2017Adjusted1
Q3 2018
1.29
1.32
+6%cc
Core EPSUSD, % cc
1 Q3 2017 core EPS adjusted to exclude core income recorded from OTC JV
Key drivers vs. PY:
+ Higher core operating income
+ Lower shares from share buyback
programs
− Discontinuation of OTC JV core income
from April 1st 2018
2018 pipeline milestones
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation 38
H1 2018 H2 2018
Regulatory
decisions and
opinions
Kymriah® DLBCL (US) ✓ Aimovig™2 Migraine (EU) ✓
Tafinlar® + Mekinist® Adjuvant melanoma (US) ✓ Kymriah® Pediatric and young adult r/r ALL (EU) ✓
Lutathera® NET (US) ✓ Kymriah® DLBCL (EU) ✓
Gx Advair®1 Asthma, COPD (US) ✕ Tafinlar® + Mekinist® Adjuvant melanoma (EU) + ATC (US) ✓
Aimovig™2 Migraine (US) ✓ Gilenya® Pediatric MS (US) ✓
Glatopa® 40mg Relapsing MS (US) ✓ GP2017 Adalimumab BS (EU) ✓
LA-EP20065 Peg-filgrastim BS (EU) =
GP1111 Infliximab BS (EU) ✓
GP20136 Rituximab BS (US) ✕
ACZ885 CV risk reduction (US)9 ✕
Submissions ACZ885 CV risk reduction (EU) ✓ BAF312 SPMS (EU) ✓
BAF312 SPMS (US) ✓ RTH258 nAMD (US/EU) =
Kisqali® Advanced BC (US/EU)3 ✓ BYL719 HR+ BC (US/EU) =
Cosentyx® AS (JP) ✓ AVXS-101 SMA7 (US) ✓
CTL019 Pediatric ALL + DLBCL (JP) ✓
Promacta® 1st line SAA (US/EU) ✓
Major trial
readouts
Kisqali® Advanced BC (MONALEESA-3) ✓4 LIK0668 Obesity ✓
LJN452 NASH (Interim Analysis) ✓ BYL719 HR+ BC ✓
INC280 ALK- cMET amplified NSCLC =
Entresto® HFpEF (Interim Analysis) ✓
✓ Achieved ✕ Missed = On track
1. Complete Response Letter received from FDA after Q4 2017 results; Advair® is a registered trademark of Glaxo Group Ltd. 2. Aimovig® is developed in collaboration with Amgen 3. Indication expansion based on MONALEESA-3 & 7 results
4. Data presented at ASCO; 5. Positive CHMP opinion received 6. Complete Response Letter (CRL) received from FDA after Q1 2018 results 7. i.v. formulation in type 1 SMA; submitted in EU October 2018 8. Program discontinued
9. CRL received from FDA October 2018
13. Preserved ejection fraction
14. Graft-versus-host disease
15. Neuroendocrine tumors
16. Chronic spontaneous urticaria / chronic idiopathic urticaria
17. Psoriatic arthritis head-to-head study versus adalimumab
18. Non-alcoholic steatohepatitis
19. Ankylosing spondylitis head-to-head study versus adalimumab
20. Acute myeloid leukemia
21. Chronic Obstructive Pulmonary Disease
22. Secondary Progressive Multiple Sclerosis
23. IV formulation Spinal Muscular Atrophy Type 1
24. 1st line colorectal cancer / 1st line renal cell carcinoma
25. IT formulation Spinal Muscular Atrophy Type 2/3
Planned filings 2018 to 2022
Combination abbreviations:
fulv fulvestrant
tmx tamoxifen
gsn goserelin
NSAI Non-steroidal aromatase inhibitor
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
39Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
ABL001CML4 3rd line
QGE031CSU/CIU16
2021
ACZ8851st Line NSCLC
ACZ8852nd Line NSCLC
Kymriah®r/r Follicular Lymphoma
Kymriah ®CLL22
Kymriah ®r/r DLBCL in 1st relapse
Entresto®
Heart failure (PEF)13
INC280 NSCLC6
Cosentyx®
nrAxSpA12
OMB157Relapsing multiple sclerosis
2019
SEG101Sickle cell disease
LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia
and others (same as originator)
PDR001 + Tafinlar®+Mekinist®
Metastatic BRAF V600+ melanoma
QVM149Asthma
QMF149Asthma
New molecule
New indication
New formulation
Biosimilars
LCI699Cushing’s disease
Lucentis®
ROP10
BYL719a + fulvHR+, HER2 (-) postmenopausal
adv. BC9 2nd line
RTH258nAMD7
2018
LAM320MDR8 tuberculosis
Lucentis®
Diabetic retinopathy
1. Secondary prevention of cardiovascular events
2. Diffuse large B-cell lymphoma
3. Severe aplastic anemia
4. Chronic myeloid leukemia
5. Long-acting release
6. Non-small cell lung cancer
7. Neovascular age-related macular degeneration
8. Multi-drug resistant
9. Breast cancer
10. Retinopathy of prematurity
11. Indolent Non-Hodgkin’s lymphoma
12. Non-radiographic axial spondyloarthritis
2022
BYM338Sarcopenia
VAY736Primary Sjoegren’s syndrome
Kisqali ®HR+, HER2 (-) BC9 (adjuvant)
Cosentyx®
AS H2H19
Rydapt®AML20 (FLT3 wild type)
INC280NSCLC6 (EGFRm)
Kymriah+ pembrolizumab - r/r DLBCL
ACZ885Adjuvant NSCLC
PDR001 comboMetastatic Melanoma
RTH258Retinal vein occlusion
CFZ533Sjorgen’s Syndrome
KAE609Malaria
EMA401Peripheral neuropathic pain
CNP520Alzheimer’s disease
BYM338Hip fracture recovery
KAF156 Malaria
ECF843d
Dry eye
CAD106Alzheimer’s disease
LHW090Resistant hypertension
HDM201Acute myeloid leukemia
CFZ533Solid Organ Transplant
CSJ117Severe Asthma
AVXS-201Rett Syndrome
LJN452Non-alcoholic steatohepatitis
QBW251COPD21
ZPL389Atopic dermatitis
UNR844Presbyopia
LMI070Spinal muscular atrophy
VAY785e
Non-alcoholic steatohepatitis
VAY736Autoimmune Hepatitis
ABL001CML4 1st line
MTV273Multiple myeloma
LOU064Chronic spontaneous urticaria
VPM087CRC 1L/RCC 1L24
LJC242Non-alcoholic steatohepatitis
a) US filing, submitted in EU
awaiting HA acceptance
Entresto®
Post-acute myocardial infarction
RTH258Diabetic macular edema
QAW039Asthma
2020
Jakavi®Acute GVHD14
Cosentyx®
PsA H2H17
Jakavi®Chronic GVHD14
XolairNasal Polyps
AVXS-101SMA Type 2/325
12. Diffuse large B-cell lymphoma
13. Preserved ejection fraction
14. Graft-versus-host disease
15. Multi-drug resistant
16. Retinopathy of prematurity
17. Severe aplastic anemia
18. Acute myeloid leukemia
19. Acute lymphoblastic leukemia
20. Secondary Progressive Multiple Sclerosis
21. Long-acting release
22. Chronic Lymphocytic Leukemia
23. IV formulation Type 1 SMA
24. 1st line colorectal cancer / 1st line renal cell
carcinoma
25. IT formulation SMA Type 2/3
Pipeline of key projects in confirmatory development
Early Clinical Trials Registration Trials – Phase III / Pivotal In Registration
1. Chronic myeloid leukemia
2. Non-small cell lung cancer
3. Chronic spontaneous urticaria / chronic idiopathic urticaria
4. Neuroendocrine tumors
5. Breast cancer
6. Neovascular age-related macular degeneration
7. Secondary prevention of cardiovascular events
8. Indolent Non-Hodgkin’s lymphoma
9. Non-radiographic axial spondyloarthritis
10. Psoriatic arthritis head-to-head study versus adalimumab
11. Ankylosing spondylitis head-to-head study versus adalimumab
40Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
FTY720 Pediatric multiple sclerosis
Cosentyx®
nrAxSpA9
Entresto®
Heart failure (PEF)13
Kisqali®b + tmx + gsn/or NSAI + gsnHR+, HER2(-) premenopausal adv. or metastatic BC5 1st line
Kisqali®b + fulvHR+, HER2(-) postmenopausal
adv. or metastatic BC5 1st/2nd line
Entresto®
Post-acute myocardial infarction
Jakavi®Acute GVHD14
Cosentyx®
PsA H2H10
Cosentyx®
AS H2H11
LAM320MDR15 tuberculosis
Kisqali®
HR+, HER2(-) BC5 (adjuvant)
Jakavi®Chronic GVHD14
Combination abbreviations:fulv fulvestrant
ltz letrozole
tmx tamoxifen
gsn goserelin
NSAI Non-steroidal aromatase inhibitor
Taf Tafinlar® (dabrafenib)
Mek Mekinist® (trametinib)
a) In collaboration with Amgen; companies to co-commercialize in the US,
Novartis to have AMG 334 exclusive rights in rest of world excluding Japan.
b) Approved in US, submitted in EU.
Lucentis®
ROP16
QMF149Asthma
QVM149Asthma
OMB157 Relapsing multiple sclerosis
RTH258Diabetic macular edema
Promacta®/Revolade®
SAA17 1st line
Rydapt®AML18 (FLT3 wild type)
LA-EP2006 (pegfilgrastim, US)Chemotherapy-induced neutropenia and
others (same as originator)
XolairNasal Polyps
SEG101Sickle cell disease
QAW039Asthma
RTH258nAMD6
LCI699Cushing’s disease
BYL719 + fulvHR+, HER2 (-) postmenopausal
adv. BC5 2nd line
ACZ885Adjuvant NSCLC2
ACZ8851st Line NSCLC2
ACZ8852nd Line NSCLC2
CAD106Alzheimer’s disease
KAE609Malaria
BYM338 Hip fracture recovery
EMA401Peripheral neuropathic pain
INC280 NSCLC2
ABL001CML1 3rd line
CNP520Alzheimer’s disease
ECF843Dry eye
ABL001CML1 1st line
KAF156Malaria
LJN452Non-alcoholic steatohepatitis
QGE031CSU/CIU3
QBW251COPD
LMI070Spinal muscular atrophy
LHW090Resistant hypertension
VAY736Primary Sjoegren’s syndrome
BYM338 Sarcopenia
ZPL389Atopic dermatitis
UNR844Presbyopia
INC280NSCLC2 (EGFRm)
VAY785b
Non-alcoholic steatohepatitis
VAY736Autoimmune Hepatitis
Kymriah ®r/r Follicular Lymphoma
Kymriah ®CLL22
GP2013 (rituximab, US)Follicular lymphoma, DLBCL12 and
others (same as originator)
GP2017 (adalimumab) Arthritides, plaque psoriasis and others
(same as originator)
PDR001 comboMetastatic Melanoma
Kymriah ®r/r DLBCL in 1st relapse
PDR001 + Tafinlar®+Mekinist®
Metastatic BRAF V600+ melanoma
LA-EP2006 (pegfilgrastim, EU)Chemotherapy-induced neutropenia and
others (same as originator)
Kymriah ®+ pembrolizumab - r/r DLBCL
HDM201Acute myeloid leukemia
LOU064Chronic spontaneous urticaria
MTV273Multiple myeloma
CFZ533Solid Organ Transplant
ACZ885Sec. prev. CV events1
LTW888db
Retinopathy
New molecule
New indication
New formulation
Biosimilars
AVXS-101SMA Type 2/325
VPM087CRC 1L/RCC 1L24
CSJ117Severe Asthma
RTH258Retinal vein occlusion
Lucentis®
Diabetic retinopathyBAF312 SPMS20
CFZ533Sjorgen’s Syndrome
AVXS-201Rett Syndrome
LJC242Non-alcoholic steatohepatitis
AVXS-101SMA Type 123
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 2018 presentation
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation42
New additions
Trials taken out (operational decision-points achieved)
Study Program Indication Phase Patients
NCT03200717 IO-PAZ (CPZP034A2410) Votrient® Renal cell carcinoma Phase 2 100
NCT02354508 SWITCH (CSOM230C2413) Signifor® Acromegaly Phase 3 123
Study Program Indication Phase Patients
NCT03668613 (CAIN457A2311) Cosentyx® Psoriasis Phase 3 80
NCT03517540 TANDEM (CLJC242A2201J) LJC242 Non-alcoholic steatohepatitis Phase 2 200
NCT03437278 (CQGE031C2202) QGE031 Chronic spontaneous urticaria Phase 2 48
NCT03481634 KESTREL (CRTH258B2301) RTH258 Diabetic eye disease Phase 3 534
NCT03481660 KITE (CRTH258B2302) RTH258 Diabetic eye disease Phase 3 356
NCT03512197 (CPKC412E2301) Rydapt® Acute myeloid leukemia Phase 3 502
NCT03474965 (CSEG101B2201) SEG101 Prevention of VOC in pediatric patients with Sickle Cell Disease Phase 2 100
NCT03484923 (CPDR001J2201) Tafinlar®+Mekinist® Previously treated unresectable or metastatic melanoma Phase 2 135
Cardio-Metabolic
Entresto®
- Angiotensin receptor neprilysin inhibitor (ARNI)
44
Study NCT02678312 PANORAMA HF (CLCZ696B2319) NCT02661217 TRANSITION (CLCZ696B2401)
Indication Heart failure in pediatric patients Heart failure
Phase Phase 2/3 Phase 4
Patients 360 1,002
Primary Outcome
Measures
Part 1: Pharmacodynamics and pharmacokinetics of
LCZ696 analytes
Part 2: Efficacy and safety compared with enalapril
Assessing the percentage of patients who achieve the target
dose of 200 mg bid LCZ696 at 10 weeks after
randomization
Arms/Intervention
• Part 1: LCZ696 0.8 mg/kg or 3.1 mg/kg or both
• Part 2: Enalapril is 0.2 mg/kg bid; LCZ696: 3.125 mg
granules and adult formulation (50, 100, 200 mg bid)
• Pre-discharge treatment initiation - LCZ696 (50, 100,
200 mg bid)
• Post-discharge treatment initiation - LCZ696 (50, 100,
200 mg bid)
Target Patients
Pediatric patients from1 month to < 18 years of age with
heart failure due to systemic left ventricle systolic
dysfunction
Heart failure patients with reduced ejection-fraction
hospitalized for an acute decompensation event
Expected Completion 2021 Q4-2018
Publication TBD
• IA presented at ESC-2018;
• 10-wk data submitted EHJ (not yet accepted);
• 26-wk data presentation planned for ESC-HF May-2019
with goal of simultaneous publication if possible (not yet
accepted)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
45
Study NCT02884206 PERSPECTIVE (CLCZ696B2320) NCT02468232 PARALLEL-HF (CLCZ696B1301)
Indication Heart failure Heart failure, reduced ejection fraction
Phase Phase 3 Phase 3
Patients 520 225
Primary Outcome
Measures
Change from baseline in the CogState Global Cognitive
Composite Score (GCCS)
Time to the first occurrence of the composite endpoint -
either cardiovascular (CV) death or heart failure (HF)
hospitalization
Arms/Intervention
• LCZ696 50, 100, and 200 mg bid with placebo of
valsartan
• Valsartan 40, 80, and 160 mg bid tablets with placebo
for LCZ696
• LCZ696 50 mg, 100 mg, 200 mg bid/placebo of Enalapril
• Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of LCZ696
Target PatientsPatients with chronic heart failure with preserved ejection
fraction
Japanese heart failure patients (NYHA Class II-IV) with
reduced ejection fraction
Expected Completion 2022H1-2019 (primary); 2020 (open-label extension)
Publication TBD TBD
Entresto®
- Angiotensin receptor neprilysin inhibitor (ARNI)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
46
Study NCT02554890 PIONEER-HF (CLCZ696BUS01) NCT02924727 PARADISE-MI (CLCZ696G2301)
Indication Heart failure Acute myocardial infarction
Phase Phase 3B/4 Phase 3
Patients 887 4,650
Primary Outcome
Measures
Percentage change from baseline in N-terminal pro-brain
natriuretic peptide (NT-proBNP)
Time to the first occurrence of a confirmed composite
endpoint (cardiovascular (CV) death, heart failure (HF)
hospitalization, or outpatient heart failure)
Arms/Intervention
• sacubitril/valsartan (LCZ696)
• sacubitril/valsartan (LCZ696) matching placebo
• enalapril
• enalapril matching placebo
• LCZ696 50 mg, 100 mg, 200 mg bid / placebo of
ramipril/valsartan
• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of
LCZ696 / placebo for valsartan
Target PatientsPatients with HFrEF (LVEF<40%) hospitalized for ADHF
and stable for more than 24 hours
Post-AMI patients with evidence of LV systolic dysfunction
and/or pulmonary congestion, with no known prior history of
chronic HF
Expected Completion Q3-2018 (actual) 2020
Publication
• Presentation planned AHA Nov-2018;
• Publication submitted to NEJM Q4-2018 (not yet
accepted)
TBD
Entresto®
- Angiotensin receptor neprilysin inhibitor (ARNI)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
47
Study NCT01920711 PARAGON (CLCZ696D2301) NCT03066804 PARALLAX (CLCZ696D2302)
Indication Heart failure, preserved ejection fraction Heart failure, preserved ejection fraction
Phase Phase 3 Phase 3
Patients 4,800 2,200
Primary Outcome
Measures
Cumulative number of primary composite events of
cardiovascular (CV) death and total (first and recurrent) HF
hospitalizations
Change in NT-proBNP from baseline to week 12
Arms/Intervention• LCZ696 50 mg, 100 mg and 200 mg
• Valsartan 40 mg, 80 mg and 160 mg
• LCZ696 50 mg, 100 mg and 200 mg bid
• Enalapril 2.5 mg, 5 mg and 10 mg bid
• Valsartan 40 mg, 80 mg, 160 mg bid
• Placebo to match LCZ696 sacubitril/valsartan
• Placebo to match enalapril
• Placebo to match valsartan
Target PatientsHeart failure patients (NYHA Class II-IV) with preserved
ejection fraction
Heart failure patients (NYHA Class II-IV) with preserved
ejection fraction
Expected Completion H2-2019 2020
Publication TBD TBD
Entresto®
- Angiotensin receptor neprilysin inhibitor (ARNI)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Ilaris®
- Anti IL-1β
48
Study NCT01327846 CANTOS (CACZ885M2301)
Indication Cardiovascular risk reduction
Phase Phase 3
Patients 10,061
Primary Outcome
MeasuresTime to first occurrence of major adverse cardiovascular event, which is a composite of CV death, non-fatal MI, and stroke
Arms/Intervention
• Canakinumab 50 mg + standard care therapy
• Canakinumab 150 mg + standard care therapy
• Canakinumab 300 mg + standard care therapy
• Placebo + standard care therapy
Target Patients Post-myocardial infarction patients on standard of care with elevated hsCRP
Expected Completion Core portion of study completed Jun-2017; Open-label extension continues till H1-2020
Publication
• Published cancer results in Lancet, Aug-2017
• Published CV outcomes in NEJM, Sept 21, 2017 vol 377 no 12
• Published pre-planned secondary analysis on relationship of CRP and CVRR; presented at AHA as late breaker Nov 2017
• Presented at ACC 2018 and published T2DM secondary endpoint
• Op Ed published in Fortune. V Narashimhan, Novartis CEO, Mar-2018
• Chronic kidney disease data presented at ACC 11 Mar-2018; published in J Am Coll Cardiol, May 29, 2018 vol 71 no 21
• Published On-treatment IL-6 data in the European Heart Journal and ESC, Munich, Aug 26, 2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Immunology, Hepatology
& Dermatology
Cosentyx®
- Anti IL-17
50
Study NCT01544595 (CAIN457A2302E1 – extension study)NCT01640951 SCULPTURE (CAIN457A2304E1 –
extension study)
Indication Psoriasis Psoriasis
Phase Phase 3 Phase 3
Patients 1,146 675
Primary Outcome
Measures
Cumulative rate of subjects with loss of psoriasis area and
Cumulative rate of subjects with loss of Psoriasis Area and
Severity Index (PASI) 75 response up to week 68 (time = 0
being defined as week 52)
The number and percentage of subjects having any adverse
event
Arms/Intervention
• Secukinumab 150 mg
• Secukinumab 300 mg
• Placebo
• Fixed-time interval regimen secukinumab 150 mg
• Retreatment at start of relapse secukinumab 150 mg
• Fixed-time interval regimen secukinumab 300 mg
• Retreatment at start of relapse secukinumab 300 mg
• Open label secukinumab 300 mg
Target Patients
Patients with moderate to severe chronic plaque-type
psoriasis completing preceding psoriasis phase III studies
with secukinumab
Patients with moderate to severe chronic plaque-type
psoriasis
Expected Completion 2017 (actual) 2017 (actual)
Publication• 2-years results: Br J Dermatol. 2017 May 12. doi:
10.1111/bjd.15656
• 3-years results: Br J Dermatol; 5 June 2017. doi:
10.1111/bjd.15706
• 5-years results: Submitted to JEADV; 14 February 2018
doi: 10.1111/jdv.14878
• 5-years presented at EAD Sept 2017 (late-breaker)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
51
Study NCT02471144 (CAIN457A2310) NCT03066609 (CAIN457A2318)
Indication Psoriasis Psoriasis
Phase Phase 3 Phase 3
Patients 169 543
Primary Outcome
Measures
The percentage of Participants achieving a 75%
Improvement from Baseline in PASI Score at week 12
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
Arms/Intervention
• Secukinumab low dose
• Secukinumab high dose
• Placebo
• Etanercept (comparator)
• Secukinumab 300 mg
• Secukinumab 150 mg
• Placebo
Target PatientsPatients from 6 to less than 18 years of age with severe
chronic plaque
Patients with moderate to severe chronic plaque-type
psoriasis with or without psoriatic arthritis comorbidity
Expected Completion 2023 Q1-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
52
Study NCT02826603 CLARITY (CAIN457A2326) NCT03668613 (CAIN457A2311)
Indication Psoriasis Psoriasis
Phase Phase 3B Phase 3
Patients 1,102 80
Primary Outcome
Measures
Psoriasis Area and Severity Index (PASI) 90 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 response at
week 12
Arms/Intervention• Secukinumab 300 mg
• Ustekinumab 45 mg/ 90 mg
• Secukinumab low dose
• Scukinumab high dose
Target Patients Patients with moderate to severe plaque psoriasisPediatric patients of age 6 to <18 years, with moderate to
severe plaque psoriasis
Expected Completion Q3-2018 (actual) 2023
Publication
• Abstract Winter Clin Derm (US) Jan-2018
• Abstract to EADV in 2018
• Submission Journal (16wk 1ry EP IA) Q3-2018
(ongoing)
• Encore Abstract AAD 2019
TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
53
Study NCT02748863 ALLURE (CAIN457A2323) NCT02745080 EXCEED (CAIN457F2366)
Indication Psoriasis Psoriatic Arthritis
Phase Phase 3 Phase 3
Patients 214 850
Primary Outcome
Measures
Psoriasis Area and Severity Index (PASI) 75 response and
Investigators' Global Assessment (IGA) 0 or 1 responseAmerican College of Rheumatology 20 (ACR20) response
Arms/Intervention
• Secukinumab 300 mg (2 mL PFS device)
• Secukinumab 300 mg (2 x 1 mL PFS device)
• Placebo
• Secukinumab 300 mg s.c.
• Adalimumab 40 mg s.c.
Target Patients Adult subjects with moderate to severe plaque psoriasis Patients with active psoriatic arthritis
Expected Completion Q3-2018 (actual) 2020
Publication• Submission Journal TBC Q2-2019
• Abstract at AAD in 2019TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
54
Study NCT03031782 (CAIN457F2304) NCT01863732 (CAIN457F2305E1 – extension study)
Indication Psoriatic Arthritis Ankylosing spondylitis
Phase Phase 3 Phase 3
Patients 80 300
Primary Outcome
MeasuresTime to flare in Part 2
Assessment of spondyloarthritis international society criteria
/ ASAS 20 response
Arms/Intervention• Secukinumab (pre-filled syringe) 75 mg
• Placebo
• Secukinumab 75 mg in PFS
• Secukinumab 150 mg in PFS
Target PatientsJuvenile Idiopathic Arthritis subtypes of Psoriatic and
Enthesitis-related ArthritisPatients with active ankylosing spondylitis
Expected Completion 2021 Q2-2018 (actual)
Publication TBD
• 3-year results: Manuscript published in Clinical and
Experimental Rheumatology in May-2017
• 4-year results: Presented at ACR in Nov-2017
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
55
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
Publication
• 3 year results: ACR 2016; Mease PJ et al. Arthritis
Rheumatol. 2016; 68 (suppl 10)
• 3 years results: Manuscript submitted in Q4-2017
• Primary 52 week results: Baeten D & Sieper J, et al. N
Engl J Med 2015;373:2534–48
• 2 year results: Marzo-Ortega, et al. Arthritis Care Res
2017 Feb 24. doi: - 10.1002/acr.23233
• 3 year results: Marzo-Ortega, et al. RMD 2017
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
56
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 Q2-2019 Q1-2018 (actual)
Publication
• Primary results: McInnes IB, et al. Lancet.
2015;386:1137–46
• 2 years results: McInnes et al, Rheumatology
2017;56:1993-2003
• 3 year results: Abstract to be submitted to EULAR
congress in Jun-2018
• 16 weeks results: PANLAR congress in Apr-2016
• 52 weeks results: Pavelka et al. Arthritis Research &
Therapy 2017
• 2 year results: Presented at ACR in Nov-2017
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
57
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, 20:4752 week results: manuscript to be submitted in Q1-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
58
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) Q2-2019
Publication
• 52 week results: abstract to be presented at PANLAR
congress (Apr-2018)
• 2 year results: manuscript to be submitted in Q3-2018
• 24 week results late breaker presented in ACR in Nov-
2017
• 24 week data; manuscript submitted to Annals of
Rheumatic Disease in Nov 2017
• 52 week data; to be presented at ACR 2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Cosentyx®
- Anti IL-17
59
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 2019 2022
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis 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 at an initial reduced canakinumab dose
or prolonged canakinumab dose interval
Arms/Intervention• Canakinumab
• Placebo
• Canakinumab dose reduction
• Canakinumab dose interval prolongation
Target PatientsPatients with, 3 separate disease cohorts TRAPS, HIDS,
and colchicine resistant FMF (Hereditary periodic fevers )
Patients with Systemic Juvenile Idiopathic Arthritis (SJIA)
(Pediatric)
Expected Completion 2017 (actual) 2017 (actual)
Publication
• Safety & efficacy (w16+40) in NEJM in May 2018
(May 17, 2018: N Engl J Med 2018; 378:1908-1919)
• Additional manuscripts in 2018
Manuscript to be submitted in Q4-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis 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 2020
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
LJC242 - FXR agonist + CCR2/CCR5 inhibitor
62
Study NCT03517540 TANDEM (CLJC242A2201J)
Indication Non-alcoholic steatohepatitis
Phase Phase 2
Patients 200
Primary Outcome
Measures
• Evaluation of safety and tolerability of combination
therapy (tropifexor + cenicriviroc) by monitoring adverse
event profile, vital signs and laboratory parameters
Arms/Intervention
• Tropifexor
• Cenicriviroc
• Tropifexor + Cenicriviroc
Target PatientsAdult patients with non-alcoholic steatohepatitis (NASH) and
liver fibrosis
Expected Completion 2020
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study NCT02477332 (CQGE031C2201) NCT02649218 (CQGE031C2201E1)
Indication Chronic spontaneous urticaria Chronic spontanenous urticaria
Phase Phase 2B Phase 2B
Patients 382 226
Primary Outcome
Measures
Establish dose-response relationship of QGE031 with
respect to achievement of complete hives response at week
12
Long-term safety; number of participants with treatment-
emergent adverse events
Arms/Intervention
• Ligelizumab 24mg q4wks
• Ligelizumab 72mg q4wks
• Ligelizumab 240mg q4wks
• Ligelizumab 120mg single dose
• Omalizumab 300mg q4wks
• Placebo q 4wks
Ligelizumab 240 mg q4wks open label
Target Patients Patients with chronic spontaneous urticaria Patients with chronic spontaneous urticaria
Expected Completion 2017 (actual) H2-2019
PublicationPrimary results: Presentated at EAACI 2018; manuscript
expected in Q4-2018Primary results manuscript submission in Q4-2019
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation63
QGE031 - Anti-IgE
Study NCT03437278 (CQGE031C2202)
Indication Chronic spontaneous urticaria
Phase Phase 2
Patients 48
Primary Outcome
MeasuresChange in the 7 day Urticaria Activity Score (UAS7)
Arms/Intervention
• Ligelizumab 120 mg q4wks
• Ligelizumab 24 mg q4wks
• Placebo / ligelizumab 120 mg q4wks
Target PatientsAdolescents from 12 to <18 years of age, with chronic
spontaneous urticaria
Expected Completion 2020
Publication Manuscript to be submitted in 2021
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation64
VAY736 – Fully human IgG1/κ anti-BAFF-R mAb
Study NCT02962895 (CVAY736A2201) NCT03217422 AMBER (CVAY736B2201)
Indication Primary Sjoegren's syndrome Autoimmune hepatitis
Phase Phase 2B Phase 2
Patients 180 80
Primary Outcome
Measures
Safety and efficacy of VAY736 in primary Sjoegren's
syndrome (pSS)Alanine aminotransferase (ALT) normalzation
Arms/Intervention• VAY736
• Placebo
• VAY736
• Placebo control with conversion to active VAY736
Target PatientsPatients With Moderate to Severe Primary Sjoegren's
Syndrome (pSS)
Autoimmune hepatitis patients with incomplete response or
intolerant to standard treatment of care
Expected Completion 2020 2022
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation65
Neuroscience
Aimovig® – CGRP receptor antagonist
67
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 2020
Publication Planned in 2019 (more details will follow) TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
CNP520 - BACE inhibitor
CAD106 - active beta-amyloid immunotherapy
68
Study NCT02565511 GENERATION S1 (CAPI015A2201J) NCT03131453 GENERATION S2 (CCNP520A2202J)
Indication Alzheimer’s disease Alzheimer’s disease
Phase Phase 2B/3 Phase 2B/3
Patients 1,340 2,000
Primary Outcome
Measures
Time to diagnosis of MCI due to Alzheimer's disease or
dementia due to Alzheimer's disease
Change in the Alzheimer's Prevention Initiative Composite
Cognitive (APCC) Test Score
Time to diagnosis of MCI due to Alzheimer's disease or
dementia due to Alzheimer's disease
Change in the Alzheimer's Prevention Initiative Composite
Cognitive (APCC) Test Score
Arms/Intervention
• CAD106 450 µg + Alum 450 µg i.m.
• Placebo to CAD106 + Alum 450 µg i.m.
• CNP520 50 mg oral
• Placebo to CNP520 oral
• CNP520 15 mg oral
• CNP520 50 mg oral
• Placebo to CNP520 oral
Target PatientsCognitively unimpaired participants aged 60 to 75 years,
with two APOE4 allele (Homozygotes )
Cognitively unimpaired participants aged 60 to 75 years,
with at least one APOE4 allele (Homozygotes or
Heterozygotes) and, if Heterozygotes, with evidence of
elevated brain amyloid
Expected Completion 2024 2024
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
BAF312 - S1P-R modulator
69
Study NCT01665144 -EXPAND (CBAF312A2304)
Indication Secondary progressive multiple sclerosis
Phase Phase 3
Patients 1,620
Primary Outcome MeasuresThe delay in time to confirmed disability progression as
measured by EDSS (Expanded Disability Status Scale)
Arms/Intervention
• BAF312 (5-day titration: 0.25mg to 1.25mg; Maintenance
dose: 2mg (day 6))
• Placebo
Target Patients Patients with secondary progressive multiple sclerosis
Expected Completion Core in 2016/Extension in 2023
Publication• Presentations at ECTRIMS and AAN 2017
• Lancet March 2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
BYM338 - Activin receptor II B
70
Study NCT02333331 InvestiGAIT (CBYM338E2202) NCT02152761 (CBYM338D2201)
Indication Sarcopenia Hip fracture recovery
Phase Phase 2B Phase 2B
Patients 280 245
Primary Outcome
Measures
Evaluate improvement in physical performance (Change
from baseline at week 24 in Short Physical Performance
Battery)
Change from baseline in total lean body mass measured by
DXA at week 24
Arms/Intervention
• Bimagrumab low dose
• Bimagrumab moderate dose
• Bimagrumab high dose
• Placebo
• Bimagrumab low dose
• Bimagrumab moderate dose
• Bimagrumab high dose
• Placebo
Target Patients Older adults with sarcopenia Patients after surgical treatment of hip fracture
Expected Completion Q4-2018 Q4-2018
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
EMA401 - Angiotensin II type 2 receptor antagonist
71
Study NCT03094195 EMPHENE (CEMA401A2201) NCT03297294 EMPADINE (CEMA401A2202)
Indication Peripheral neuropathic pain Peripheral neuropathic pain
Phase Phase 2 Phase 2
Patients 360 400
Primary Outcome
Measures
Dose-response in change in weekly mean of the 24-hour
average pain score from Baseline to week 12
Change in weekly mean 24-hour average pain score
from Baseline to Week 12
Arms/Intervention• 3 doses EMA401
• Placebo
• 1 doses EMA401
• Placebo
Target Patients Post-herpetic neuralgia patients Painful diabetic neuropathy
Expected Completion H2-2019 H1-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Gilenya®
- S1P-R modulator
72
Study NCT01892722 PARADIGMS (CFTY720D2311) NCT01201356 (CFTY720D2399)
Indication Pediatric multiple sclerosis Relapsing multiple sclerosis (RMS)
Phase Phase 3B Phase 3B/4
Patients 215 4,125
Primary Outcome
Measures
Frequency of relapses in patients treated for up to 24
months (using ARR)Long-term safety and tolerability
Arms/Intervention• Interferon beta-1a i.m.
• Fingolimod 0.5 mg/ 0.25 mgSingle-arm study of fingolimod 0.5 mg/day
Target PatientsPediatric patients with multiple sclerosis with five-year
fingolimod extension phasePatients with relapsing multiple sclerosis
Expected Completion Q3-2017 (core phase) / 2023 (extension phase) Q4-2018
Publication
• Primary data presentation: Chitnis T, et al. Presented at
ECTRIMS 2017 (Late Breaker)
• Chitnis T, Arnold DL, Banwell B, et al. Trial of Fingolimod
versus Interferon Beta-1a in Pediatric Multiple Sclerosis..
N Engl J Med. 2018; 379: 1017-1027.
• Primary data presentation: Cohen J, et al presented at
ECTRIMS 2017
• Primary manuscript: TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Gilenya®
- S1P-R modulator
73
Study NCT01633112 ASSESS (CFTY720D2312)
Indication Relapsing remitting multiple sclerosis (RRMS)
Phase Phase 3B
Patients 1,064
Primary Outcome
Measures
Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod
to glatiramer acetate (20 mg) in reducing the annualized
relapse rate up to 12 months
Arms/Intervention
• Fingolimod 0.5 mg orally
• Fingolimod 0.25mg orally
• Copaxone® 20 mg s.c.
Target Patients Patients with relapsing-remitting multiple sclerosis
Expected Completion Q3-2018 (actual)
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
LMI070 - SMN2 RNA splice modulator
74
Study NCT02268552 (CLMI070X2201)
Indication Type 1 spinal muscular atrophy
Phase Phase 1/2
Patients 44
Primary Outcome
Measures
Number of participants with adverse events (AEs), serious
adverse events (SAEs) and deaths
Arms/Intervention • Branaplam oral, once weekly, 3 ascending doses
Target PatientsPatients with type 1 spinal muscular atrophy
Expected Completion H2-2019
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
OMB157 - Anti-CD20
75
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 H2-2019 H2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
OMB157 - Anti-CD20
76
Study NCT03249714 SAKURA (COMB157G1301)
Indication Multiple sclerosis
Phase Phase 2
Patients 60
Primary Outcome
Measures
Reduced cumulative number of Gd-enhanced T1 lesions
across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab
vs placebo)
Arms/Intervention• Ofatumumab 20 mg subcutaneous injections
• Placebo
Target Patients Patients with relapsing forms of multiple sclerosis
Expected Completion 2020
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Oncology
ABL001 – Specific, allosteric Bcr-Abl kinase inhibitor
78
Study NCT03106779 (CABL001A2301)
Indication Chronic myeloid leukaemia (CML)
Phase Phase 3
Patients 222
Primary Outcome
MeasuresMajor Molecular Response (MMR) rate at 24 weeks
Arms/Intervention• ABL001 40 mg
• Bosutinib 500 mg
Target Patients
Patients with chronic myelogenous leukemia in chronic
phase (CML-CP), previously treated with 2 or more tyrosine
kinase inhibitors
Expected Completion H2-200
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
ACZ885 – IL1β inhibitor
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation79
Study NCT03447769 (CACZ885T2301)
Indication Adjuvant NSCLC
Phase Phase 3
Patients 1,500
Primary Outcome
Measures
Disease free survival (primary), overall survival (key
secondary)
Arms/Intervention• Canakinumab 200mg q3w sc for 18 cycles
• Placebo q3w sc for 18 cycles
Target Patients
Patients with:
• High–risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB
(T>5cm N2)) after complete resection
• All histologies
• With/without EGFR mutation
Expected Completion 2022
Publication • TBD
BYL719 - Alpha-specific PI3K inhibitor
80
Study NCT02437318 SOLAR-1 (CBYL719C2301)
Indication HR + aBC
Phase Phase 3
Patients 572
Primary Outcome
Measures
Progression-free survival (PFS) for patients with PIK3CA
mutant status
Arms/Intervention• Fulvestrant 500 mg + alpelisib 300 mg
• Fulvestrant 500 mg + placebo
Target Patients
Men and postmenopausal women with hormone receptor
positive, HER2-negative advanced breast cancer which
progressed on or after aromatase inhibitor treatment
Expected Completion Q3-2018 (actual)
PublicationSubmitted for ESMO 2018
Manuscript to be submitted in Q4-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Kymriah®
– CAR-T therapy
81
Study NCT02445248 JULIET (CCTL019C2201) NCT02435849 ELIANA (CCTL019B2202)
Indication Relapsed / refractory DLBCL Pediatric and young adult Relapsed/ refractory ALL
Phase Phase 2 Phase 2
Patients 128 95
Primary Outcome
MeasuresOverall response rate; efficacy and safety of CTL019
Overall remission rate (ORR) - overall remission rate during
the 6 months after CTL019 administration, which includes
CR and CR with incomplete blood count recovery (CRi) as
determined by IRC assessment
Arms/Intervention Single-arm study of single dose of CTL019 Single-arm study of single dose of CTL019
Target PatientsAdult patients with relapsed or refractory diffuse large B-cell
lymphoma (DLBCL)
Pediatric and young adult patients with relapsed and
refractory B-cell acute lymphoblastic leukemia
Expected Completion 2017 (actual) 2016 (actual)
Publication
• Schuster et al. at ICML 2017; Schuster et al. at EHA
2017; Schuster et al. at ASH 2017
• Borchmann et al. at EHA 2018
• Manuscript submitted in April 2018
• Grupp et al. at ASH 2016
• Buchner et al at EHA 2017
• Maude et al. N Engl J Med. 2018;378:439-48. DOI:
10.1056/NEJMoa1709866
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Exjade®
- Iron chelation of bis-hydroxy-phenyl triazole type
82
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 Congress submission for Q4-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
INC280 - MET Inhibitor
83
Study NCT02414139 (CINC280A2201) NCT02335944 (CINC280X2105C)
IndicationEGFR Wild-type, ALK negative advanced Non-small Cell Lung
Cancer (NSCLC)
Non-small Cell Lung Cancer (NSCLC) Patients With EGFR
Mutation
Phase Phase 2 Phase 1/2
Patients 348 177
Primary Outcome
MeasuresOverall Response Rate (ORR)
Phase II Groups 1, 2 and 3: Overall Response Rate (ORR)
Phase II Group 4: Frequency of treatment-emergent adverse
events
Arms/Intervention
• Pre-treated pts. with MET GCN ≥ 6
• Pre-treated pts. with MET GCN ≥ 4 and < 6
• Pre-treated pts. with MET GCN < 4
• Pre-treated pts. with MET mutations regardless of cMET
GCN
• Treatment-naïve pts. with MET dysregulation
• Pre-treated pts with MET dysregulation – second line
• Group 1: EGFRmut NSCLC developing resistance to
EGFR TKI
• Group 2: EGFR TKI-naïve, EGFRmut NSCLC with denovo
T790M mutation
• Group 3: Treatment-naïve, EGFRmut NSCLC
• Group 4: 1-3L EGFRmut NSCLC (with food)
Target Patients
Adult patients with EGFR wild-type (wt), ALK-negative
advanced non-small cell lung cancer (NSCLC) with either
MET amplification or MET mutations and are either
pretreated with 1 or 2 prior lines of systemic therapy or are
treatment-naïve for the advanced stage of disease
Adult Patients With EGFR Mutated Non-small Cell Lung
Cancer
Expected Completion Q2-2019 Q4-2018
Publication• Congress presentation in Q4-2018
• Manuscript submission Q2-2019Congress presentation in Q2-2019
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Jakavi®
- JAK1/2 inhibitor
84
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 H2-2019 H2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Kisqali®
- CDK 4/6 inhibitor
85
Study NCT02422615 MONALEESA-3 (CLEE011F2301) NCT02278120 MONALEESA-7 (CLEE011E2301)
Indication Advanced breast cancer – 1st / 2nd line (with fulvestrant) Advanced breast cancer - 1st line (pre-menopausal)
Phase Phase 3 Phase 3
Patients 727 672
Primary Outcome
Measures
Progression Free Survival (PFS) - time from the date of
randomization to the date of the first documented
progression or death due to any cause
Progression Free Survival (PFS) - time from the date of
randomization to the date of the first documented
progression or death due to any cause and assessed
according to RECIST 1.1
Arms/Intervention• Riblociclib 600mg + fulvestrant 500mg
• Placebo of Riblociclib + fulvestrant 500mg
• LEE011 600 mg + NSAI/tamoxifen + goserelin 3.6 mg
• Placebo of LEE011 + NSAI/tamoxifen + goserelin 3.6 mg
Target Patients
Postmenopausal women with hormone receptor positive,
HER2-negative, advanced breast cancer who have received
no or only one line of prior endocrine treatment
Premenopausal women with hormone receptor positive,
HER2-negative, advanced breast cancer
Expected Completion Q1-2018 (actual) 2017 (actual)
Publication• Slamon D, et al. Oral presented at ASCO 2018
• Manuscript published in JCO June 2018
• Tripathy D, et al. Oral presented at SABCS 2017
• Manuscript published in Lancet Oncology May 2018
(online)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
LCI699 - Cortisol synthesis inhibitor
86
Study NCT02697734 LINC-4 (CLCI699C2302) NCT02180217 LINC-3 (CLCI699C2301)
Indication Cushing's disease Cushing's disease
Phase Phase 3 Phase 3
Patients 69 132
Primary Outcome
Measures
Demonstrate the superiority of osilodrostat compared to
placebo in achieving a complete response mean urine free
cortisol ≤ upper limit of normal (mUFC ≤ ULN) at Week 12
Compare the complete response rate at the end of the 8-
week randomized withdrawal period
Arms/Intervention• LCI699 / Osilodrostat
• Placebo
Randomized withdrawal design
• LCI699 / Osilodrostat
• Placebo
Target Patients Patients with Cushing's disease Patients with Cushing's disease
Expected Completion 2020 Q2-2018 (actual)
Publication TBD Abstract submitted to congress for Q4-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
PDR001 – PD-1 checkpoint inhibitor
87
Study NCT02967692 COMBI-i (CPDR001F2301)
Indication BRAFV600 mutant metastatic melanoma
Phase Phase 3
Patients
538
Part 1 (safety-run in): 9; Part 2 (biomarker cohort): 27; Part 3
(Phase III, randomized, placebo controlled): 532
Primary Outcome
MeasuresProgression-Free Survival (PFS)
Arms/Intervention
• Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg BID +
Mekinist 2 mg
• Placebo + Tafinlar 150 mg BID + Mekinist 2 mg
Target Patients
Previously untreated patients with unresectable or
metastatic BRAF V600 mutant melanoma
Expected Completion H2-2019
PublicationCongress presentation and manuscript submission planned
H2-2019
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Rydapt®- Multi-targeted kinase inhibitor
88
Study NCT00651261 RATIFY (CPKC412A2301) NCT03280030 (CPKC412A2220)
Indication Acute myeloid leukemia Acute myeloid leukemia
Phase Phase 3 Phase 2
Patients 717 66
Primary Outcome
MeasuresOverall survival Incidence of safety events and event free survival
Arms/Intervention
• Induction and consolidation chemotherapy plus
midostaurin
• Induction and consolidation chemotherapy plus placebo
• Midostaurin 50 mg
• Placebo
Target PatientsNewly diagnosed patients < 60 years of age with FLT3
mutated acute myeloid leukemia (AML)
Newly diagnosed patients with FLT3-mutated acute myeloid
leukemia (AML)
Expected Completion 2016 (actual) 2020
Publication
• Stone RM, Manley PW, Larson RA, and Capdeville R.
published February 27, 2018 in Blood Advances
2018;(2:444-453
• H. Gu Drug Metab Dispos. 2018;46(2):109-121
• Planned: Karin Hartman, Haneke Kluin-Nelemans
Journal of Allergy and Clinical Immunology (TBD)
• Planned: Combine into single paper (maintenance and
CIR): Leukemia
TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Rydapt®- Multi-targeted kinase inhibitor
89
Study NCT03512197 (CPKC412E2301)
Indication Acute myeloid leukemia
Phase Phase 3
Patients 502
Primary Outcome
MeasuresEvent free survival
Arms/Intervention• Midostaurin 50 mg
• Placebo
Target PatientsNewly diagnosed patients with FLT3 mutation negative
acute myeloid leukemia (AML)
Expected Completion 2021
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Promacta®/Revolade
®– Thrombopoetin receptor agonist
Study NCT03025698 (CETB115E2201)
IndicationPreviously untreated or relapsed/refractory severe aplastic anemia or
recurrent aplastic anemia
Phase Phase 2
Patients 60
Primary Outcome
MeasuresPK of eltrombopag at steady state in pediatric patients with SAA
Arms/Intervention
- Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS
- Arm B: previously untreated SAA-hATG/cyclosporine +
eltrombopag
- Arm A: relapsed/refractory SAA or AA: hATG/cyclosporine +
eltrombopag or cyclosporine + eltrombopag
Target PatientsPediatric patients from age 1 <18 years with relapsed/refractory SAA
or recurrent AA after IST or previously untreated SAA
Expected Completion 2024
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation90
SEG101 – p-Selectin inhibitor
Study NCT03264989 (CSEG101A2202) NCT03474965 (CSEG101B2201)
IndicationPrevention of Vaso-Occlusive Crises (VOC) in patients with
Sickle Cell Disease (SCD) Prevention of VOC in pediatric patients with SCD
Phase Phase 2 Phase 2
Patients 55 100
Primary Outcome
MeasuresPK/PD and safety of SEG101 (crizanlizumab) at 5 mg/kg PK/PD and safety of SEG101 at 5 mg/kg
Arms/Intervention
SEG101 (crizanlizumab) at a dose of 5.0 mg/kg (or 7.5
mg/kg for exploratory group) by IV infusion, ±
Hydroxyurea/Hydroxycarbamide
SEG101 (crizanlizumab) at a dose of 5 mg/kg by IV infusion
± Hydroxyurea/Hydroxycarbamide
Target Patients Adult SCD patients with VOC Pediatric SCD patients with VOC
Expected Completion Q4-2018 2020
Publication Planned abstract submission in 2019 TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation91
Tafinlar®+Mekinist
®- BRAFV600 inhibitor and MEK inhibitor
Study NCT01682083 COMBI-AD (CDRB436F2301) NCT02124772 (CTMT212X2101)
Indication BRAFV600 mutant adjuvant melanoma BRAFV600 mutant solid tumors
Phase Phase 3A Phase 1
Patients 874 142
Primary Outcome
MeasuresRelapse-free survival (RFS) Safety, tolerability and pharmacokinetics and clinical activity
Arms/Intervention• Dabrafenib 150 mg BID + trametinib 2 mg
• Placebo
Trametinib (dose based on age and weight)
Dabrafenib + trametinib (dose based on age and weight)
Target PatientsSubjects with BRAFV600 mutation-positive melanoma with
lymph node(s) involvement, after complete resection
Pediatric Subjects Aged 1 Month to <18 Years with
Advanced V600-Mutation Positive Solid Tumors
Expected Completion Q3-2017 (actual) 2020
PublicationLong G.V., et al. N Engl J Med 2017; 377:1813-1823; DOI:
10.1056/NEJMoa1708539TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation92
Study NCT01677741 (CDRB436A2102)
Indication BRAFV600 mutant cancers
Phase Phase 1
Patients 86
Primary Outcome
MeasuresSafety, tolerability and pharmacokinetics
Arms/InterventionSingle-arm study of oral dabrafenib (dose based on age
and weight)
Target PatientsPediatric subjects aged 1 year to <18 years with advanced
BRAF V600-mutation positive solid tumors
Expected Completion H2-2019
Publication TBD
Tafinlar®
- BRAF inhibitor
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation93
Tafinlar®+Mekinist
®- BRAF inhibitor and MEK inhibitor
Study NCT02039947 COMBI-MB (CDRB436B2204)
Indication BRAFV600 mutant metastatic melanoma
Phase Phase 2B
Patients 126
Primary Outcome
MeasuresIntracranial response (IR) rate
Arms/Intervention Dabrafenib 150 mg BID + trametinib 2 mg
Target PatientsPatients with BRAF mutation-positive melanoma that has
metastasized to the brain
Expected Completion Q2-2018 (actual)
Publication• MA Davies G.V., et al. Lancet Oncology. 2017.
DOI:10.1016/S1470-2045(17)30429-
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation94
Tasigna®
- Bcr-Abl, c-Kit and PDGF-R tyrosine kinase inhibitor
Study NCT01698905 ENESTop (CAMN107A2408) NCT01844765 DIALOG (CAMN107A2203)
Indication Second line CML/CML-TFR Newly diag. CML and CML res/intol to imatinib/dasatinib
Phase Phase 2 Phase 2
Patients 163 59
Primary Outcome
Measures
No documented confirmed loss of MR4, no documented loss
of MMR and no re-starting of nilotinib therapy
• Resistant/intolerant Ph+ CML in chronic phase: Rate of
Major Molecular Responder (MMR) at 6 cycles
• Newly diagnosed and untreated Ph+ CML in first chronic
phase: Rate of MMR by 12 cycles
Arms/Intervention • Single-arm study of nilotinib
• Newly diagnosed and untreated Ph+ CML in first chronic
phase
• Resistant/intolerant Ph+ CML in chronic phase
• Resistant/intolerant Ph+ CML in accelerated phase
Target Patients
Adult CML-CP patients who received a minimum of 3 years
of TKI therapy, started off with imatinib treatment for > 4
weeks, then switched to nilotinib for at least 2 years prior to
study entry and achieved MR4.5 on nilotinib, but did not
have documented MR4.5 at the time of switch from imatinib
to nilotinib
Pediatric patients with newly diagnosed Ph+ chronic
myelogenous leukemia (CML) in chronic phase (CP) or with
Ph+ CML in CP or accelerated phase (AP) resistant or
intolerant to either imatinib or dasatinib
Expected Completion 2017 (actual) 2017 (actual)
Publication Mahon FX, et al. Ann Intern Med. 2018,168(7):461-470• Presentation at SIOP October 13, 2017
• Primary manuscript planned Q4-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation95
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 2018 Results | October 18, 2018 | Novartis Investor Presentation96
Tafinlar®+Mekinist
®- BRAF inhibitor and MEK inhibitor
Study NCT03484923 (CPDR001J2201)
Indication Previously treated unresectable or metastatic melanoma
Phase Phase 2
Patients 135
Primary Outcome
MeasuresObjective Response Rate (ORR)
Arms/Intervention
• PDR001 400mg i.v. Q4W + LAG525 600 mg i.v. Q4W
• PDR001 400mg i.v. Q4W + canakinumab 300 mg (s.c)
Q4W
• PDR001 400mg i.v. Q4W + capmatinib 400 mg BID
orally
Target PatientsAdult patients with previously treated unresectable or
metastatic melanoma
Expected Completion 2021
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation97
Zykadia®
- ALK inhibitor
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation98
Study NCT02299505 ASCEND-8 (CLDK378A2112)
Indication ALK activated NSCLC
Phase Phase 2
Patients 306
Primary Outcome
Measures
Part 1: Pharmacokinetics when taken with food
Part 2: Overall response rate (ORR) when taken with food
Arms/Intervention
• Oral LDK378 450 mg once daily taken with food
• Oral LDK378 600 mg once daily taken with food
• Oral LDK378 750 mg once daily fasted
Target PatientsAdult patients with ALK-rearranged (ALK-positive) advanced
non-small cell lung cancer
Expected CompletionPart 1 (PK): 2016 (actual)
Part 2 (ORR): Q2-2018 (actual)
Publication
• Part 1 (PK): Cho BC, et al. Journal of Thoracic Oncology;
2017 Jul; 12(9) 1357-1367
• Part 2 (ORR): Congress presentation Q4-2018;
Manuscript submission Q3-2018
Ophthalmology
Lucentis®
- Anti-VEGF
100
Study NCT02375971 RAINBOW (CRFB002H2301) NCT02640664 RAINBOW Extension (CRFB002H2301E1)
Indication Retinopathy of Prematurity (ROP) Retinopathy of Prematurity (ROP)
Phase Phase 3 Phase 3
Patients 224 180
Primary Outcome
Measures
To achieve absence of active Retinopathy of Prematurity
(ROP) and unfavorable structural outcome, patients must
fulfill all the following criteria, 1) survival, 2) no intervention
with a second modality for ROP, 3) absence of active ROP
and 4) absence of unfavorable structural outcome
To evaluate the visual function of patients by assessing the
visual acuity in the better-seeing eye at the patient’s fifth
birthday.
Arms/Intervention
• Ranibizumab 0.2 mg
• Ranibizumab 0.1 mg
• Laser therapy
• Ranibizumab 0.2 mg
• Ranibizumab 0.1 mg
Target PatientsMale and female preterm infants with bilateral retinopathy of
prematurity (ROP) who require treatment.
Male and female preterm infants with bilateral retinopathy of
prematurity (ROP) who require treatment.
Expected Completion Q1-2018 (actual) 2023
Publication
• EURETINA: Sep-2018
• AAO: Oct-2018
• Primary manuscript: planned submission by end of 2018
TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
RTH258 - Anti-VEGF
Study NCT02434328 HARRIER (CRTH258A2302) NCT02307682 HAWK (CRTH258A2301)
Indication Neovascular age-related macular degeneration (nAMD) Neovascular age-related macular degeneration (nAMD)
Phase Phase 3 Phase 3
Patients 743 1,082
Primary Outcome
Measures
Change in Best Corrected Visual Acuity (BCVA) from
baseline at week 48
Change in Best Corrected Visual Acuity (BCVA) from
baseline at week 48
Arms/Intervention• RTH258 6 mg/50 µL
• Aflibercept 2 mg/50 µL
• RTH258 3 mg/50 µL
• RTH258 6 mg/50 µL
• Aflibercept
Target Patients Subjects with exudative age-related macular degeneration Subjects with exudative age-related macular degeneration
Expected Completion Q1-2018 (actual) Q2-2018 (actual)
PublicationAbstract/presentation at AAO meeting in Nov-2017 and
Nov-2018
Abstract/presentation at AAO meeting in Nov-2017 and
Nov-2018
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation101
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
• Aflibercept 2 mg
• Brolucizumab 3 mg
• Brolucizumab 6 mg
• Aflibercept 2 mg
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 Q4-2018 2021
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation102
RTH258 - Anti-VEGF
Study NCT03481660 KITE (CRTH258B2302)
Indication Diabetic eye disease
Phase Phase 3
Patients 356
Primary Outcome
Measures
Change from baseline in best-corrected visual acuity
(BCVA)
Arms/Intervention• Brolucizumab 6 mg
• Aflibercept 2 mg
Target PatientsPatients with visual impairment due to diabetic macular
edema (DME)
Expected Completion 2021
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation103
Respiratory
QAW039 – DP2 (CRTh2) antagonist
105
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 2020 H2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
QAW039 - DP2 (CRTh2) 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 H2-2019 H2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation106
QAW039 - DP2 (CRTh2) antagonist
Study NCT03052517 SPIRIT (CQAW039A2315)
Indication Asthma
Phase Phase 3
Patients 1,900 – 2,300
Primary Outcome
Measures
Long term safety: treatment emergent adverse event (AE),
SAE and AE leading to discontinuation from study (52 wks
and 160 wks)
Arms/Intervention
• QAW039 Dose 1
• QAW039 Dose 2
• Placebo
Target Patients Patients with moderate to severe asthma
Expected Completion 2019 (for submission); 2022 (final)
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation107
QMF149 - Long-acting beta2 agonist and inhaled corticosteroid
108
Study NCT02892019 (CQMF149G2202)
Indication Asthma
Phase Phase 2
Patients 80
Primary Outcome
MeasuresTrough FEV1
Arms/Intervention• Indacaterol acetate 75 μg (via Concept1 inhaler)
• Indacaterol acetate 150 μg (via Concept1 inhaler)
Target Patients Children ≥ 6 to < 12 years of age with asthma
Expected Completion H2-2019
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
Study NCT02554786 PALLADIUM (CQVM149B2301) NCT02571777 IRIDIUM (CQVM149B2302)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 2,216 3,092
Primary Outcome
MeasuresTrough FEV1 Trough FEV1
Arms/Intervention
• QMF149 150/160 µg od
• QMF149 150/320 µg od
• MF 400 µg od
• MF 400 µg bid
• Salmeterol 50 µg /fluticasone 500 µg bid
• QVM149 150/50/160 µg od
• QVM149 150/50/80 µg od
• QMF149 150/160 µg od
• QMF149 150/320 µg od
• Salmeterol 50 µg /fluticasone 500 µg bid
Target Patients
Adult and adolescent (>12 years) patients with uncontrolled
asthma despite med-/high-dose ICS or low-dose
ICS/LABA(GINA step 3)
Adult patients with uncontrolled asthma despite med/high-
dose ICS/LABA (GINA ≥4)
Expected Completion H2-2019 H2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation109
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
110
Study NCT03100500 (CQVM149B1305) NCT03100825 (CQVM149B1304)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 51 94
Primary Outcome
Measures
Number of patients who reported treatment emergent
adverse events during the 52 weeks of the study
Number of patients who reported treatment emergent
adverse events during the 52 weeks of the study
Arms/Intervention • Single arm: QMF149 150/320 μg od• Single Arm: QVM149 150/50/160 μg od (Concept1
inhaler)
Target Patients Japanese patients with asthma Japanese patients with Asthma
Expected Completion Q1-2019 Q2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
Study NCT02892344 QUARTZ (CQVM149B2303) NCT03158311 ARGON (CQVM149B2306)
Indication Asthma Asthma
Phase Phase 3 Phase 3
Patients 802 1,251
Primary Outcome
MeasuresTrough FEV1
Change from baseline in Asthma Quality of Life
Questionnaire (AQLQ) total score
Arms/Intervention• QMF149 150/80 µg
• MF 200 µg
• QVM149 150/50/80 μg
• QVM149 150/50/160 μg
• Salmeterol/fluticasone 50/500 μ + tiotropium 5 μg
Target PatientsAdult and adolescent (>12 years) patients with in poorly
(i.e., inadequately) controlled asthmaPatients with uncontrolled asthma
Expected Completion Q1-2019 H2-2019
Publication TBD TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation111
QVM149 - LA beta2 agonist, LA muscarinic antagonist, ICS
Study NCT03137784 SILVER (CQVM149B2204)
Indication Asthma
Phase Phase 2
Patients 148
Primary Outcome
Measures
Evaluate the bronchodilator effects of NVA237 (25 ug and 50
ug) compared to placebo in terms of trough FEV1
Arms/Intervention• NVA237 (glycopyrronium bromide) 25/50 μg
• Placebo
Target Patients Asthma patients
Expected Completion Q1-2018 (actual)
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation112
Xolair®
– anti-IgE antibody
113
Study NCT03369704 (CIGE025F1301)
Indication Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis
Phase Phase 3
Patients 337
Primary Outcome
Measures
Mean nasal symptom score, consists of severity of
sneezing, rhinorrhea and nasal congestion.
Arms/Intervention
In addition to standard of care:
• Omalizumab per approved allergic asthma dosing table
for IgE/body weight combinations
• Placebo
Target Patients
Patients with severe Japanese cedar pollinosis, whose
symptoms were inadequately controlled with current
recommended therapies
Expected Completion Q1-2019
Publication
Nov 2018: Late breaking abstract for the American
Association of Allergy, Asthma and Immunology (AAAAI)
annual meeting in Feb 2019
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Sandoz
Biopharmaceuticals
Erelzi®
- Biosimilar etanercept
115
Study NCT02638259 (GP15 301)
Indication Immunology
Phase Phase 3
Patients 376
Primary Outcome
Measures
Change in DAS28-CRP score from baseline to week 24 in
patients treated with GP2015 and patients treated with Enbrel
Arms/Intervention• GP2015 50 mg
• EU-authorized Enbrel® 50mg
Target Patients Patients with moderate to severe, active rheumatoid arthritis
Expected Completion Q4-2017 (actual)
Publication
• Kavanaugh et al. Arthritis Rheumatol 2017; 69 (suppl 10)
• 48 week: Abstract to EULAR 2018
• 24 week: Manuscript to RMD Open (final submission for
publication on 17-Sep-2018)
• 48 week: Manuscript in Q4-2018 (journal Arthritis Research
&Therapy)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Rixathon®
- Biosimilar rituximab
116
Study NCT02514772 (GP13 302) NCT01419665 (GP13 301)
Indication Immunology Oncology
Phase Phase 3 Phase 3
Patients 107 629
Primary Outcome
Measures
Incidence of adverse events and serious adverse events,
anaphylactic reactions, hypersensitivity; immunogenicity Overall response rate in patients with FL
Arms/Intervention• GP2013
• Rituxan® or MabThera®
• GP2013
• MabThera®
Target PatientsPatients with active Rheumatoid Arthritis, previously treated
with Rituxan or MabThera (ASSIST-RT)
Patients with previously untreated, advanced stage follicular
lymphoma (ASSIST-FL)
Expected Completion 2016 (actual) Q2-2018 (actual)
Publication
• ACR Q4-2017 Poster
• Tony, H-P et al, Arthritis Care & Research, 2018
(accepted for publication)
• Amersdorffer J, et al and Jurczak W., et al presented at
ESMO 2017, Published in Lancet Hematology (doi:
10.1016/ S2352-3026(17)30106-0)
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
GP2017 - Biosimilar adalimumab
117
Study NCT02016105 ADACCESS (GP17-301) NCT02744755 ADMYRA (GP17-302)
Indication Immunology Immunology
Phase Phase 3 Phase 3
Patients 465 353
Primary Outcome
Measures
PASI 75 response rate at week 16 in patients treated with
GP2017 and patients treated with Humira®
Change in DAS28-CRP score from baseline to week 12 in
patients treated with GP2017 and patients treated with
Humira®
Arms/Intervention• GP2017
• Humira® Adalimumab
• GP2017
• US licensed Humira® Adalimumab
Target PatientsPatients with moderate to severe chronic plaque-type
psoriasisPatients with moderate to severe active rheumatoid arthritis
Expected Completion 2016 (actual) Q3-2018 (actual)
Publication
• 51 week data and switch data, Blauvelt et al. BJD, 2018,
https://doi.org/10.1111/bjd.16890
• Blauvelt et al. presented at ACR 2017, Blauvelt et. al.
presented at AAD 2017, Blauvelt et.al. presented at
EADV 2017, Blauvelt et al., presented at ACG 2017,
Blauvelt et.al. presented at UEGw 2017
• Abstract, oral presentation at ACR 2018
• Abstract and poster at EULAR 2019
• Manuscript with study results journal TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Global Health*
*Previously classified as ‘Established Medicines and Anti-infectives’
Tobramycin – An aminoglycoside antibiotic
119
Study NCT02712983 iBEST-1 (CTBM100G2202)
Indication Bronchiectasis
Phase Phase 2
Patients 105
Primary Outcome
MeasuresP. aeruginosa density in sputum
Arms/Intervention
Three dose regimens, each of them having 3 treatment
arms:
• Tobramycin inhalation powder
• Tobramycin inhalation powder and placebo
• Placebo
Target PatientsPatients with non-cystic fibrosis bronchiectasis and
pulmonary P. aeruginosa infection
Expected Completion H2-2019
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
KAF156 – Plasmodium Falciparum Inhibitor – PfCARL mediated
120
Study NCT03167242 (CKAF156A2202)
Indication Malaria
Phase Phase 2
Patients 512
Primary Outcome
Measures
PCR-corrected adequate clinical and parasitological
response (ACPR)
Arms/Intervention• KAF156 and LUM-SDF (different combinations)
• Coartem
Target PatientsAdults and children with uncomplicated Plasmodium
Falciparum Malaria
Expected Completion H2-2019
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
KAE609 – Plasmodium Falciparum Inhibitor – spiroindolone against
PfATP4
121
Study NCT03334747 (CKAE609A2202)
Indication Malaria
Phase Phase 2
Patients 150
Primary Outcome
Measures
CTCAE grades increase from baseline in alanine
aminotransferase (ALT) or aspartate aminotransferase
(AST)
Arms/Intervention• KAE609
• Coartem
Target Patients Adults with uncomplicated Plasmodium Falciparum malaria
Expected Completion H2-2019
Publication TBD
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation
Key definitions and trademarks
Novartis Q3 2018 Results | October 18, 2018 | Novartis Investor Presentation122
This presentation contains several important words or phrases that we define as below:
AE: Adverse Event
ALL: Acute lymphatic leukemia
AMD: Age-Related Macular Degeneration
AML: Acute myeloid leukemia
Approval: In Pharmaceuticals and Alcon in US and EU; each indication and regulator combination counts as
approval; excludes label updates, CHMP opinions alone and minor approvals
aRCC: advanced renal cell cancer
AS: Ankylosing Spondylitis
bid: twice a day
BC: Breast cancer
BCMA: B-cell maturation antigen
BCVA: best corrected visual acuity
BS: Biosimilars
BTD: Breakthrough therapy designation
CGRP: Calcitonin gene-related peptide
CLL: Chronic lymphocytic leukemia
CM: Chronic migraine
CML: Chronic myeloid leukemia
COPD: Chronic Obstructive Pulmonary Disease
CR: complete remission
CRC: Colorectal Cancer
CSU / CIU: Chronic spontaneous urticaria / Chronic idiopathic urticaria
CVRR: Cardiovascular risk reduction
DLBCL: Diffuse large B-cell lymphoma
DMC: Data monitoring committee
EF: ejection fraction
EM: Episodic migraine
FL: Follicular lymphoma
FPFV: First patient first visit
GBM: Glioblastoma multiforme
HF: Heart failure
HF-pEF: Heart failure with preserved ejection fraction
HFrEF: Heart failure with reduced ejection fraction
HR+/HER2- mBC:Hormone Receptor positive / Human Epidermal growth factor receptor 2 negative metastatic
breast cancer
LoE: Loss of exclusivity
M/M: Multiple myeloma
MF: Myelofibrosis
MI: Myocardial infarction
MS: Multiple sclerosis
NASH: Non-Alcoholic Steatohepatitis
NET: Neuroendocrine tumor
NSCLC: Non-small cell lung cancer
NTD: New Therapeutic Drug
od: once a day
ORR: Overall response rate
OS: Overall survival
PA: Prior authorization
PASI 90: 90% reduction in Psoriasis Area Severity Index from baseline
PFS: Progression free survival
PSA: Prostate specific antigen
PsA: Psoriatic arthritis
PsO: Psoriasis
PV: Polycythemia vera
PY: Prior year
QoL: Quality of Life
RCC: Renal cell cancer
r/r ALL: relapsed/refractory acute lymphoblastic leukemia
RRMS: relapsing-remitting multiple sclerosis
SCPC: Sickle cell pain crisis
SpA: Spondyloarthropathy
SPMS: Secondary progressive multiple sclerosis
TFR: Treatment-free Remission
TNBC: Triple negative breast cancer
Trademarks
Eylea® is a registered trademark of Regeneron Pharmaceuticals, Inc.
Enbrel®, Epogen® and Neulasta® are a registered trademark of Amgen Inc.
Humira® is a registered trademark of AbbVie Ltd.
Remicade® and Stelara® are registered trademarks of Janssen Biotech, Inc.
Rituxan® is a registered trademark of Biogen Inc