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.
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.
"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. 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.
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.
LDK378 is a highly selective inhibitor of an important cancer target, anaplastic lymphoma kinase (ALK),. 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.
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer accounting for 85-90% of all cases. Approximately 3-8% of patients with NSCLC have the ALK gene mutation. 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.
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.
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 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.
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 U.S. Food and Drug Administration. Frequently Asked Questions: Breakthrough Therapies. Available at:
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