Transforming Molecules into Breakthrough Therapies. Timothy Wright, M.D. Global Head Pharma Development Investor Day London, 22 November 2013

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Timothy Wright, M.D.

Global Head Pharma Development

Investor Day

London, 22 November 2013

Transforming Molecules into

Breakthrough Therapies

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Disclaimer

This presentation contains forward-looking statements that can be identified by terminology such as such as “potential,” “expected,” “will,” “planned,” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, or regarding potential future revenues from any such products; the potential completion of the divestiture of the Novartis blood transfusion diagnostics unit; potential shareholder returns or credit ratings, the potential outcome of the share buyback being initiated; or regarding potential future sales or earnings of the Novartis Group or any of its divisions; or by discussions of strategy, plans, expectations or intentions. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of the Group regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that any new products will be approved for sale in any market, or that any new indications will be approved for any existing products in any market, or that any approvals which are obtained will be obtained at any particular time, or that any such products will achieve any particular revenue levels. Nor can there be any guarantee that the proposed divestiture of the blood transfusion diagnostics unit will be completed in the expected form or within the expected time frame or at all. 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 divestiture. Neither can there be any guarantee that shareholders will achieve any particular level of shareholder returns or regarding the potential outcome of the share buyback being initiated. Nor can there be any guarantee that the Group, or any of its divisions, will achieve any particular financial results or any particular credit rating. In particular, management's expectations could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally, including an unexpected failure to obtain necessary government approvals for the transaction, or unexpected delays in obtaining such approvals; the potential that the potential strategic benefits, synergies or opportunities expected from the transaction may not be realized or may take longer to realize than expected; the inherent uncertainties involved in predicting shareholder returns or credit ratings; unexpected clinical trial results, including additional analyses of existing clinical data or unexpected new clinical data; the Group's ability to obtain or maintain patent or other proprietary intellectual property protection, including the ultimate extent of the impact on the Group of the loss of patent protection and exclusivity on key products which commenced last year and will continue this year; unexpected product manufacturing and quality issues, including the resolution of the Warning Letter issued to us with respect to three Sandoz manufacturing facilities, and the completion of efforts to restart production of certain products formerly produced at the Consumer Health manufacturing facility at Lincoln, Nebraska, and the restructuring efforts at that site; government, industry, and general public pricing pressures; uncertainties regarding actual or potential legal proceedings, including, among others, actual or potential product liability litigation, litigation and investigations regarding sales and marketing practices, shareholder litigation, government investigations and intellectual property disputes; competition in general; uncertainties regarding the effects of the ongoing global financial and economic crisis, including the financial troubles in certain Eurozone countries; uncertainties regarding future global exchange rates; uncertainties regarding future demand for our products; uncertainties involved in the development of new healthcare products; the impact that the foregoing factors could have on the values attributed to the Group's assets and liabilities as recorded in the Group's consolidated balance sheet; and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected. 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.

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Results of our R&D Investments: Leading Total NME

Approvals Across Key Regions

Novartis

Pfizer/Wyeth

Merck/SGP

BMS

AstraZeneca

J&J

US

EU

Japan

China

New chemical/molecular entity (NCE/NME) approvals for selected companies

2007 – 2013 (Q3)

Notes: Does not include vaccines. Novartis includes Alcon, co-developed or co-marketed products from Ciba Vision, QLT, Idenix, Genentech; Pfizer includes products of Pharmacia + Wyeth; Merck includes products of Schering Plough + Organon; Roche includes products of Genentech and Chugai (JP); J&J includes products of Janssen + Centocor + RWJohnson. EU approvals for all companies are inclusive of fixed-dose combinations

Source: FDA, EMA, PMDA, SFDA/CDE databases (snapshot as of 7 October 2013)

GSK

Roche/Genentech

Sanofi-Aventis

Eli Lilly

Bayer

2

1

5

4

5

9

10

10

11

12

20

5

1

4

5

1

5

8

9

9

4

9

8

5

5

9

2

3

9

15

21

13

15

6

4

3

3

5

4

6

4

8

6

14

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3

new

formulations

25

new

indications

29

new

molecules

2013

OUR PIPELINE

1

2014

2015

2016

2017

1Includes GenMeds and Oncology

Industry-Leading Pipeline & Aspiring Higher

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Three FDA Breakthrough Therapy Designations

RLX030 (Q2)

Acute Heart

Failure

BYM338 (Q3)

Sporadic

Inclusion Body

Myositis

LDK378 (Q1)

ALK+ Non Small

Cell Lung Cancer

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Following the Science in Immune-Mediated Diseases

Single target – multiple indications

Psoriasis

(Submitted)

Psoriatic

Arthritis

(Ph. III)

Ankylosing

Spondylitis

(Ph. III)

Rheumatoid

Arthritis

(Ph. III)

Asthma

(Ph. II)

Uveitis

(Ph. II)

Multiple

Sclerosis

(Ph. II)

IL-17

IL-1, TLRs, IL-4, TSLP, GM-CSF, TNF 

Dendritic

Cells

Antigens: Foreign, Self

Th17

Th1

IL-12, IL-23, … Processed Antigens

Tc1

Pathogen Defense

Immune Surveillance

IFN-

IL-2,

IL-15

IL-21

IL-22

IL-17F

IL-17A

Amplification of

Immune Responses

(esp. Autoimmune)

Secukinumab

AIN457 (Secukinumab)

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Psoriasis

(Submitted)

Psoriatic

Arthritis

(Ph. III)

Ankylosing

Spondylitis

(Ph. III)

Rheumatoid

Arthritis

(Ph. III)

Asthma

(Ph. II)

Uveitis

(Ph. II)

Multiple

Sclerosis

(Ph. II)

IL-17

Patient 1

Patient 2

Week 0

Week 8

Results in chronic plaque psoriasis

Following the Science in Immune-Mediated Diseases

Psoriasis

AIN457 (Secukinumab)

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Psoriasis

(Submitted)

Psoriatic

Arthritis

(Ph. III)

Ankylosing

Spondylitis

(Ph. III)

Rheumatoid

Arthritis

(Ph. III)

Asthma

(Ph. II)

Uveitis

(Ph. II)

Multiple

Sclerosis

(Ph. II)

IL-17

Following the Science in Immune-Mediated Diseases

Psoriatic Arthritis

-0.8

-0.7

-0.6

-0.5

-0.4

-0.3

-0.2

-0.1

0.0

0

4

12

24

36

52

*

*

Placebo (N=27) Etanercept (N=20) Secukinumab 150 mg (N=20) Secukinumab 300 mg (N=27)

Change from baseline HAQ-DI

*P < 0.05 vs. placebo

HAQ-DI, Health Assessment Questionnaire–Disability Index; LOCF, last observation carried forward. Source: Data from Phase III study in Psoriasis (A2303)

HAQ-DI reduction in Psoriasis patients

indicates underlying activity in Psoriatic

Arthritis

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Psoriasis

(Submitted)

Psoriatic

Arthritis

(Ph. III)

Ankylosing

Spondylitis

(Ph. III)

Rheumatoid

Arthritis

(Ph. III)

Asthma

(Ph. II)

Uveitis

(Ph. II)

Multiple

Sclerosis

(Ph. II)

IL-17

Following the Science in Immune-Mediated Diseases

Ankylosing Spondylitis

61

85

17

0

All patients

Biologic-naive

patients

AIN457

Placebo

ASAS 20 (% of patients)

Week 6

ASAS 20: ASsessment in Ankylosing Spondylitis,

improvement in 20% from baseline

First non-TNF-

α

biologic to show

activity in Ankylosing Spondylitis

AIN457 (Secukinumab)

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Sporadic Inclusion Body Myositis (Ph. III) Hip Fracture Recovery (Ph. II) Sarcopenia (PoC) Cancer Cachexia (PoC) Mechanically Ventilated Patients (PoC) COPD Cachexia (PoC)

Act RII

blockade

BYM338

Following the Science in Muscle-Wasting Conditions

Single target – multiple indications

Rationale for Activin RII Blockade

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Sporadic Inclusion Body Myositis (Ph. III) Hip Fracture Recovery (Ph. II) Sarcopenia (PoC) Cancer Cachexia (PoC) Mechanically Ventilated Patients (PoC) COPD Cachexia (PoC)

Act RII

blockade

Following the Science in Muscle-Wasting Conditions

Sporadic Inclusion Body Myositis

sIBM* patient

pre-treatment

8 weeks after

single infusion of

BYM338

5% increase in

muscle volume

Improvement in muscle function also observed

sIBM: Sporadic Inclusion Body Myositis

Data from Proof of Concept study in sIBM patients

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Novartis Committed to Advancing Cell Therapies

Three different approaches using cell therapies

CTL019

Target Indications

Leukemias

Lymphomas

Cell based therapy of isolated

T-cells via lentivirus transfection and

infusion into leukemia patients

Immune cells from patients Re-inject into patient Re-engineer to target cancer

HSC835

Infusion of processed and

expanded cord blood stem cells

into patients lacking stem cells

Target Indications

Leukemias

Leukodystrophies

Hemoglobinopathies

FCRx

Infusion of processed donor

leukophoresis product enriched for

facilitator cells and progenitors

Target Indications

Solid organ transplant

Facilitating cell

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Expanded Cord Blood Stem Cell Infusion for

Hematopoietic Stem Cell Transplants

Source of hematopoietic

stem cells

Cord blood

Leukemia

Leukodystrophies

Hemoglobinopathies

1. HLA mapping (readily

available for banked

cord blood)

2. Harvesting of stem

cells

3. Expansion using

proprietary

technology

4. Patient infusion

Target population

Steps involved

HSC835: Expansion of Cord Blood Stem Cells

Note: This is an illustration of how HSC835-based stem-cell expansion is assumed to work in patients requiring stem cell transplants or others

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Processed Donor Hematopoietic Stem Cells to Build

Immune Tolerance in Transplant Patients

Processed donor

leukopheresis product

enriched for:

HSC

Facilitator cells

Progenitors (FCRx)

Organ transplant

Avoid lifelong

immunosuppression in

some patients and

reduce risk of rejection

by building tolerance

FCRx: Use of Cell Therapy in Transplant Patients

Note: This is an illustration of how FCRx technology is assumed to work in transplant patients

CD56

low

Support HSC

reconstitution

Promote HSC survival

and homing

CD56

high

Protect donor HSC

from recipient

immune attack

Prevent GVHD

Infusion

Donor

Recipient

Recipient with

bone marrow

chimera

Facilitating

cell

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Innovations and Portfolio Prioritization to Drive R&D

Productivity Going Forward

1.

Invest in priority programs

2.

Re-evaluate non-priority programs

3.

Contain fixed costs by monitoring R&D

hiring

4.

Other processes to improve productivity,

e.g., Risk-Based Monitoring (RBM)

 

?  

? ?

Strength of evidence

Unmet need

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Novartis: One of MIT’s Top 50 Disruptive Innovators

Only pharmaceutical company in 2013

Novartis listed as one of

the top 50 Disruptive

Innovators

Only pharmaceutical

company in 2013

Recognized for

developing a

continuous drug

manufacturing process

and other initiatives

Figure

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References

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