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.
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, management's
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
company's 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
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 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.
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References
[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/
SignificantAmendmentstotheFDCAct/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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Posted: March 2013
