Novartis marks World CML Day with update on global research program to evaluate whether Ph+ CML patients can live treatment-free

EAST HANOVER, N.J., Sept. 19, 2013 /PRNewswire/ -- Novartis commemorates World CML Day by announcing the latest milestone in its unique clinical trial program evaluating the potential for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) to maintain undetectable levels of disease after stopping drug therapy – a concept called treatment-free remission. More than 100 study sites are now enrolling patients to the trial program.

Since 2008, organizations around the world have recognized World CML Day¹. The date, September 22 or 9/22, was chosen to symbolically represent the genetic material that is associated with CML – the missing section from chromosome 22 shifts to chromosome 9 and vice versa, in a phenomenon called "translocation²." Known as the Philadelphia chromosome, this genetic mutation is present in about 95% of CML patients³.

"As a leader in CML, Novartis is proud to support World CML Day through our continued dedication to ongoing research in this disease," said Herve Hoppenot, President, Novartis Oncology. "We are now exploring if the disease can be treated to a point where drug therapy is no longer needed, representing the next step in what may be possible for patients living with Ph+ CML."

Over the past decades, Novartis research in Ph+ CML has helped transform the disease from a fatal diagnosis to a chronic condition for many patients. Today, the company continues its long-standing commitment to the global CML community. The Novartis treatment-free remission clinical trial program[*] includes eight studies that are now underway and actively enrolling Ph+ CML patients in more than 100 global sites across 40 countries.

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[*] In the Novartis treatment-free remission clinical trial program, molecular response (reduction of BCR-ABL transcripts in the blood of patients) is measured at four levels, based on an international standard:

• MMR (<= 0.1% BCR-ABL)
• MR4 (<= 0.01% BCR-ABL)
• MR4.5 (<= 0.0032% BCR-ABL)
• Undetectable BCR-ABL (no detectable BCR-ABL transcript level with sample sensitivity of at least 4.5 log)

Globally, Novartis is sponsoring four treatment-free remission studies and providing support for four investigator-initiated studies. More information on the Novartis-sponsored trials, including addresses and contact information for each study site, can be found at:

• ENESTfreedom study: www.clinicaltrials.gov - identifier # NCT01784068
• ENESTop study: www.clinicaltrials.gov - identifier # NCT01698905
• ENESTpath study: www.clinicaltrials.gov - identifier # NCT01743989
• ENESTgoal study: www.clinicaltrials.gov -identifier # NCT01744665

Stopping treatment is not a clinical recommendation and should only be attempted in the context of a well conducted clinical study. A very important part of these treatment-free remission studies is the inclusion of regular molecular monitoring with International Scale Real-Time Quantitative Polymerase Chain Reaction (IS RT-Q-PCR) testing. Once treatment is stopped molecular monitoring is used to identify if a patient's level of disease remains in deep molecular response or if the reintroduction of treatment is needed.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as "to evaluate," "can," "potential," "continued," "dedication," "ongoing," "may," "continues," "commitment," "providing support," or similar expressions, or by express or implied discussions regarding potential new indications or labeling for Novartis Ph+ CML products or regarding potential future revenues from such products. 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 a Novartis Ph+ CML product to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that any Novartis Ph+ CML product will be approved for any additional indications or labeling in any market. Nor can there be any guarantee that any Novartis Ph+ CML product will achieve any particular levels of revenue in the future. In particular, management's expectations regarding such products could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; unexpected manufacturing issues; 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 Pharmaceuticals Corporation researches, develops, manufactures and markets innovative prescription drugs used to treat a number of diseases and conditions, including cardiovascular, dermatological, central nervous system, bone disease, cancer, organ transplantation, psychiatry, infectious disease and respiratory. The company's mission is to improve people's lives by pioneering novel healthcare solutions.

Located in East Hanover, New Jersey, Novartis Pharmaceuticals Corporation is an affiliate of Novartis AG, which 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 131,000 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.     What is World CML Day? CML Advocates Network. http://www.cmladvocates.net/worldcmlday/what-is-world-cml-day

2.     National Comprehensive Cancer Network (NCCN): Clinical Practice Guidelines in Oncology: Chronic Myelogenous Leukemia, V.4.2013.  Available at: http://www.nccn.org/professionals/physician_gls/pdf/cml.pdf.

3.     Faderl, Stefan, et al. "The biology of chronic myeloid leukemia." New England Journal of Medicine.1999; 341.3: 164-172.

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