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1/3
MEDIA RELEASE COMMUNIQUE AUX MEDIAS MEDIENMITTEILUNG

Novartis compound LDK378 receives FDA Breakthrough Therapy
designation for ALK+ non-small cell lung cancer

LDK378 is an investigational selective inhibitor of ALK
1
, a target found in a variety
of cancers including metastatic non-small cell lung cancer (NSCLC)

Breakthrough Therapy designation is based on positive early data in patients with
ALK+ NSCLC who have been previously treated with crizotinib

First filing for LDK378 anticipated in early 2014

Basel, March 15, 2013 Novartis announced today that its investigational compound
LDK378 has received Breakthrough Therapy designation by the US Food and Drug
Administration (FDA) for the treatment of patients with anaplastic lymphoma kinase
positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who had progressed
during treatment with, or were intolerant to, crizotinib. There are limited treatment options
for patients with ALK+ NSCLC, who tend to be non-smokers and younger than NSCLC
patients without an ALK translocation
2
.

According to the FDA, Breakthrough Therapy designation is intended to expedite the
development and review of drugs that treat serious or life-threatening conditions if the
therapy has demonstrated substantial improvement over an available therapy on at least
one clinically significant endpoint. The designation includes all of the fast track program
features, as well as more intensive FDA guidance. The Breakthrough Therapy
designation is a distinct status from both accelerated approval and priority review, which
can also be granted to the same drug if relevant criteria are met
3
.

LDK378 is a strong example of our research approach, which focuses on identifying the
underlying cause of disease pathways, said Alessandro Riva, Global Head, Oncology
Development & Medical Affairs, Novartis Oncology. This Breakthrough Therapy
designation will allow us to collaborate more closely with the FDA and potentially to
expedite the availability of an important new treatment option for patients with ALK+
NSCLC.

Initial results from a Phase I study investigating the maximum tolerated dose, safety,
pharmacokinetics and antitumor activity of LDK378 in 88 patients with ALK+ advanced
malignancies, as detected by an FDA-approved test and who had progressed during
treatment with, or were intolerant to, crizotinib, were presented at the European Society
of Medical Oncology 2012 annual congress. The data showed marked responses in a
majority of patients with ALK+ NSCLC
1
. A response rate (including complete response
[CR], partial response [PR] and unconfirmed PR) of 80% was observed in the patients
who had experienced disease progression after crizotinib treatment
1
.

Novartis has initiated two Phase II clinical trials to further evaluate the compound in this
patient population with plans to initiate several Phase III clinical trials later this year. First
regulatory filing is anticipated by early 2014.


2/3
About LDK378
LDK378 is a highly selective inhibitor of an important cancer target, anaplastic lymphoma
kinase (ALK)
1,4
. Because it is an investigational compound, the safety and efficacy profile
of LDK378 has not yet been established. Access to this investigational compound is
available only through carefully controlled and monitored clinical trials. These trials are
designed to better understand the potential benefits and risks of this compound. Because
of the uncertainty of clinical trials, there is no guarantee that LDK378 will ever be
commercially available anywhere in the world.

About NSCLC
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer accounting
for 85-90% of all cases
5
. Approximately 3-8% of patients with NSCLC have the ALK gene
mutation
1
. There are limited treatment options for patients with ALK+ NSCLC, who tend
to be non-smokers and younger than NSCLC patients without an ALK translocation.
2


Disclaimer
The foregoing release contains forward-looking statements that can be identified by
terminology such as can, will, potentially, plans, potential, or similar expressions,
or by express or implied discussions regarding potential marketing approvals for LDK378
or regarding potential future revenues from LDK378. You should not place undue
reliance on these statements. Such forward-looking statements reflect the current views
of management regarding future events, and involve known and unknown risks,
uncertainties and other factors that may cause actual results with LDK378 to be
materially different from any future results, performance or achievements expressed or
implied by such statements. There can be no guarantee that LDK378 will be submitted or
approved for sale in any market, or at any particular time. Nor can there be any
guarantee that LDK378 will achieve any particular levels of revenue in the future. In
particular, managements expectations regarding LDK378 could be affected by, among
other things, negative or otherwise unexpected clinical trial results, including unexpected
new clinical data and unexpected additional analysis of existing clinical data; unexpected
regulatory actions or delays or government regulation generally; government, industry
and general public pricing pressures; competition in general; the companys ability to
obtain or maintain patent or other proprietary intellectual property protection; the impact
that the foregoing factors could have on the values attributed to the Novartis Group's
assets and liabilities as recorded in the Group's consolidated balance sheet, and other
risks and factors referred to in Novartis AGs 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 anticipated, believed, estimated or expected. Novartis is providing
the information in this press release as of this date and does not undertake any
obligation to update any forward-looking statements contained in this press release as a
result of new information, future events or otherwise.

About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of
patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified
portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic
pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal
health products. Novartis is the only global company with leading positions in these areas.
In 2012, the Group achieved net sales of USD 56.7 billion, while R&D throughout the
Group amounted to approximately USD 9.3 billion (USD 9.1 billion excluding impairment
and amortization charges). Novartis Group companies employ approximately 128,000 full-
time-equivalent associates and operate in more than 140 countries around the world. For
more information, please visit http://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at http://twitter.com/novartis.

References

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1. Shaw A, et al. Results of a First-in-Human Phase I Study of the ALK Inhibitor LDK378 in Advanced
Malignancies. Abstract #4400. 2012 European Society for Medical Oncology (ESMO) Annual Meeting,
Vienna, Austria.
2. Shaw A, et al. Targeting Anaplastic Lymphoma Kinase in Lung Cancer. Clin Cancer Res 2011;17:2081-
2086.
3. U.S. Food and Drug Administration. Frequently Asked Questions: Breakthrough Therapies. Available at:
http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/Signific
antAmendmentstotheFDCAct/FDASIA/ucm341027.htm. Accessed Mar 12, 2013.
4. Mehra R et al. First-in-Human Phase I Study of the ALK Inhibitor LDK378 in Advanced Solid Tumors.
Abstract #3007. American Society of Clinical Oncology 2012 Annual Meeting, Chicago, IL.
5. American Cancer Society. Lung Cancer Non-Small Cell Detailed Guide. Available at:
http://www.cancer.org/Cancer/LungCancer-Non-SmallCell/DetailedGuide/non-small-cell-lung-cancer-
what-is-non-small-cell-lung-cancer. Accessed Mar 12, 2013.

# # #

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