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Novartis to highlight long-term data, innovative pipeline for patients with hematologic diseases and breast cancer at ASH and SABCS

  • Multiple studies evaluating deep molecular response with Tasigna® versus Glivec® in Ph+ CML patients
  • Overall survival data from Jakavi® Phase III trials in patients with the debilitating blood cancer, myelofibrosis
  • Hematology pipeline innovations in acute lymphoblastic leukemia, chronic lymphocytic leukemia, advanced systemic mastocytosis and multiple myeloma
  • Updates on advanced breast cancer portfolio at SABCS, including Afinitor® and investigational compounds BKM120 and BYL719

Basel, December 4, 2013 - Novartis will present updates on its broad cancer portfolio with more than 240 abstracts at the upcoming American Society of Hematology (ASH) annual meeting and CTRC-AACR San Antonio Breast Cancer Symposium (SABCS). Presentations will provide key scientific evidence for targeted treatments in chronic myeloid leukemia (CML), myelofibrosis (MF) and advanced breast cancer, as well as emerging information on pipeline compounds[1],[2].

"Cancer treatment has been revolutionized by targeted therapies, which have established a highly successful approach to the management of many cancers," said Hervé Hoppenot, President, Novartis Oncology. "At this exciting time in cancer research, we are presenting new long-term findings on well-established, targeted therapies while also reporting early data on innovative treatment approaches and compounds in areas of high unmet patient need, including acute lymphoblastic leukemia, chronic lymphocytic leukemia and advanced breast cancer."

Novartis Oncology has 24 pivotal trials underway and more than 25 new molecular entities in development targeting key oncogenic pathways and covering a wide range of mutations.

The ASH annual meeting in New Orleans, held December 7-10, will feature data assessing the overall survival rates for Jakavi® (ruxolitinib) in MF, and multiple studies evaluating deep molecular responses with Tasigna® (nilotinib) compared with Glivec® (imatinib)[*] therapy in a variety of appropriate Philadelphia chromosome-positive CML (Ph+ CML) patient populations[1]. Novartis Oncology will also share updated efficacy data on Exjade® (deferasirox) in transfusion-dependent thalassemia major patients with severe cardiac iron overload[1]. Pipeline advances will also be presented, including additional outcomes data in patients with relapsed, refractory acute lymphoblastic leukemia (ALL) and relapsed, refractory chronic lymphocytic leukemia (CLL) treated with chimeric antigen receptor (CAR) modified T cells directed against CD19 (CTL019)[1].

At CTRC-AACR SABCS, held December 10-14, Novartis Oncology will present data for its mTOR inhibitor Afinitor® (everolimus) and provide updates on the clinical development of investigational PI3K inhibitors BKM120 (buparlisib) and BYL719 in breast cancer[2].

Highlights at ASH include:

Tasigna (nilotinib)

  • Long-term, five-year update from the ENESTnd study evaluating superiority of Tasigna versus Glivec in patients with newly diagnosed Ph+ CML in chronic phase (Abstract #92; December 8, 5:15 PM)
  • Three-year data from the ENESTcmr study evaluating sustained deep molecular response following a switch to Tasigna in patients with Ph+ CML in chronic phase who still had evidence of residual disease after two or more years of Glivec therapy (Abstract #94; December 8, 5:45 PM)
  • LASOR, a Phase III study comparing the efficacy of Tasigna to Glivec in patients with Ph+ CML in chronic phase who have a suboptimal cytogenetic response to Glivec (Abstract #95; December 8, 6:00 PM)

Jakavi (ruxolitinib)

  • Long-term pooled overall survival results from the COMFORT-I and COMFORT-II Phase III trials studying the safety and efficacy of Jakavi in patients with MF (Abstract #2820; December 8, 6:30 PM)
  • Long-term overall survival results from COMFORT-I with responses reported through 132 weeks (Abstract #396; December 9, 11:45 AM)
  • New analyses of COMFORT-II survival data examining the impact of Jakavi on patients with low- and high-risk molecular mutations (Abstract #107; December 8, 6:00 PM)
  • An evaluation of overall survival among COMFORT-II patients compared to a historical control group of MF patients (Abstract #4066; December 9, 6:00 PM)
  • Baseline characteristics and symptom burden in the RESPONSE trial, a Phase III study comparing safety and efficacy of Jakavi with best available treatment in polycythemia vera patients (Abstract #4071; December 9, 6:00 PM)

Exjade (deferasirox)

  • Two-year results from CORDELIA, the first head-to-head multicenter, randomized, open-label trial evaluating Exjade compared with deferoxamine for the removal of cardiac iron in patients with beta-thalassemia major (Abstract #1018; December 7, 5:30 PM)
  • Twelve-month data from the HYPERION study investigating the combination therapy of deferasirox-deferoxamine in treating transfusion-dependent thalassemia patients with severe cardiac siderosis (Abstract #2257; December 8, 6:30 PM)
  • Forty-eight-month results from five-year non-interventional registry of lower-risk MDS patients with transfusional iron overload comparing survival and acute myeloid leukemic transformation status in chelated and non-chelated patients (Abstract #2775; December 8, 6:30 PM)

PKC412 (midostaurin)

  • Quality of life results and updated duration of response and overall survival data of PKC412 in patients with advanced systemic mastocytosis (Abstract #106; December 8, 5:45 PM)

CTL019 (CART-19)

  • Additional outcomes from long-term follow-up in children and adults with relapsed, refractory ALL treated with CTL019 (Abstract #67; December 8, 5:00 PM)
  • Outcomes from Phase I/II long-term follow-up and dose optimization studies in patients with relapsed, refractory CLL treated with CAR modified T cells directed against CD19 (CTL019) (Abstract #4162; December 9, 6:00 PM; Abstract #873; December 10, 8:00 AM)

LDE225 (sonidegib)

  • Preclinical study of disease modifying effects of the combination of JAK2 and Hedgehog inhibitors in the treatment of myelofibrosis (Abstract #666; December 9, 5:45 PM)

LBH589 (panobinostat)

  • Progression-free survival and overall survival data from PANORAMA-II, a Phase II study of panobinostat, bortezomib and dexamethasone in patients with relapsed and bortezomib-refractory multiple myeloma (Abstract #1970; December 7, 5:30 PM)
  • Identification of the recommended Phase II dose of ruxolitinib plus panobinostat in patients with primary myelofibrosis, post-polycythemia vera or post-essential thrombocythemia MF (Abstract #4045; December 9, 6:00 PM)

Highlights at SABCS include:

Afinitor (everolimus)

  • Updates on Afinitor in HR+/HER2 negative advanced breast cancer include a BOLERO-2 study analysis characterizing patient responses to everolimus plus exemestane (Abstract #P2-16-17; December 12, 7:30 AM)
  • New BOLERO-3 study analyses looking at the impact of a combination therapy including everolimus, trastuzumab and vinorelbine on safety and quality of life in HER2 positive advanced breast cancer patients who are resistant to trastuzumab and have been pre-treated with a taxane (Abstract #P3-15-03; December 12, 5:00 PM; Abstract #P4-12-18; December 13, 7:30 AM)
  • Additional analysis of BOLERO-3 evaluating efficacy of the everolimus combination therapy in a subset of Asian patients will be presented (Abstract #P4-12-19; December 13, 7:30 AM)


  • A Trials in Progress poster presentation of the Phase II BELLE-4 study of BKM120 in combination with paclitaxel in patients with HER2 negative locally advanced or metastatic breast cancer, with or without PI3K activation (Abstract #OT2-6-07; December 12, 5:00 PM)
  • A Trials in Progress poster presentation of the Phase II NeoPHOEBE study of BKM120 in combination with trastuzumab and paclitaxel in patients with HER2 positive, PIK3CA mutated and PIK3CA wild-type primary breast cancer (Abstract #OT2-6-08; December 12, 5:00 PM)


  • Phase I data of BYL719 in combination with fulvestrant in patients with PIK3CA mutated estrogen-receptor positive (ER+) breast cancer (Abstract #P2-16-14; December 12, 7:30 AM)

Throughout ASH and SABCS, Novartis Oncology will host dedicated webpages (http://www.novartisoncology.com/newsroom/featured-news/ash-meeting-2013.jsp; http://www.novartisoncology.com/newsroom/featured-news/sabcs-2013.jsp) that will provide unique insights and perspectives into emerging areas of cancer care and research.

About Tasigna (nilotinib)
Tasigna® (nilotinib) is approved in more than 110 countries for the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML) in adult patients resistant or intolerant to at least one prior therapy, including Glivec® (imatinib), and in more than 85 countries for the treatment of adult patients with newly diagnosed Ph+ CML in chronic phase.

Tasigna Important Safety Information
Use with caution in patients with uncontrolled or significant cardiac disease and in patients who have or may develop prolongation of QTc. Low levels of potassium or magnesium must be corrected prior to Tasigna administration. Monitor closely for an effect on the QTc interval. Baseline ECG is recommended prior to initiating therapy and as clinically indicated. Uncommon cases (0.1 to 1%) of sudden death have been reported in clinical studies in patients with significant risk factors.

Use with caution in patients with liver impairment, with a history of pancreatitis and with total gastrectomy. Patients with rare hereditary problems of galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption should not use Tasigna. Tasigna may cause fetal harm in pregnant women. Women taking Tasigna should not breastfeed.

The most frequent Grade 3 or 4 adverse events are hematological (neutropenia and thrombocytopenia) which are generally reversible and usually managed by withholding Tasigna temporarily or dose reduction. Monitor blood counts regularly. Pancreatitis has been reported. The most frequent non-hematologic adverse events were rash, pruritus, nausea, fatigue, headache, alopecia, myalgia, constipation and diarrhea. Most of these adverse events were mild to moderate in severity.

Please see full Prescribing Information available at www.tasigna.com.

About Glivec (imatinib)
Glivec® (imatinib) is approved in more than 110 countries for the treatment of all phases of Ph+ CML, for the treatment of adult patients with KIT (CD117)-positive gastrointestinal stromal tumors (GIST), which cannot be surgically removed and/or have metastasized and for the treatment of adult patients following complete surgical removal of KIT+ GIST.

Glivec Important Safety Information
Glivec can cause fetal harm in pregnant woman Glivec has been associated with severe edema (swelling) and serious fluid retention. Cytopenias (anemia, neutropenia, thrombocytopenia) are common, generally reversible and usually managed by withholding Glivec or dose reduction. Monitor blood counts regularly. Severe congestive heart failure and left ventricle dysfunction, severe liver problems including cases of fatal liver failure and severe liver injury requiring liver transplants have been reported. Caution in patients with cardiac dysfunction and hepatic dysfunction. Monitor carefully.

Bleeding may occur. Severe gastrointestinal (GI) bleeding has been reported in patients with KIT+ GIST. Skin reactions, hypothyroidism in patients taking levothyroxine replacement , GI perforation, in some cases fatal, tumor lysis syndrome which can be life threatening have also been reported,with Glivec.  Correct dehydration and high uric acid levels prior to treatment. Long-term use may result in potential liver, kidney, and/or heart toxicities; immune system suppression may also result from long-term use. In patients with hypereosinophilic syndrome and heart involvement, cases of heart disease have been associated with the initiation of Glivec therapy. Growth retardation has been reported in children taking Glivec. The long-term effects of extended treatment with Glivec on growth in children are unknown.

The most common side effects include fluid retention, muscle cramps or pain and bone pain, abdominal pain, loss of appetite, vomiting, diarrhea, decreased hemoglobin, abnormal bleeding, nausea, fatigue and rash. Glivec should be taken with food and a large glass of water.

Please see full Prescribing Information.

About Jakavi (ruxolitinib)
Jakavi® (ruxolitinib) is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases and was approved by the European Commission in August 2012 for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Jakavi is approved in more than 50 countries, including the European Union, Canada and some countries in Asia, Latin and South America. Additional worldwide regulatory filings are underway.

Novartis licensed ruxolitinib from Incyte Corporation for development and commercialization outside the United States. Both the European Commission and the US Food and Drug Administration (FDA) granted ruxolitinib orphan drug status for myelofibrosis. Jakavi is marketed in the United States by Incyte Corporation under the name Jakafi® for the treatment of patients with intermediate or high-risk myelofibrosis.

The recommended starting dose for Jakavi is 15 mg twice daily for patients with a platelet count between 100,000cubic millimeters (mm3) and 200,000 mm3,and 20 mg twice daily for patients with a platelet count of >200,000 mm3. Doses may be titrated based on safety and efficacy. There is limited information to recommend a starting dose for patients with platelet counts between 50,000/mm3 and <100,000/mm3. The maximum recommended starting dose in these patients is 5 mg twice daily and patients should be titrated cautiously.

Jakavi is a registered trademark of Novartis AG in countries outside the United States. Jakafi is a registered trademark of Incyte Corporation.

Jakavi Important Safety Information
Jakavi can cause serious side effects, including a decrease in blood cell count and infections. Complete blood count monitoring is recommended. Dose reduction or interruption may be required in patients with severe hepatic or renal impairment or in patients developing hematologic adverse reactions such as thrombocytopenia, anemia and neutropenia. Dose reductions are also recommended when Jakavi is co-administered with strong CYP3A4 inhibitors or fluconazole. Use of Jakavi during pregnancy is not recommended and women should avoid becoming pregnant during Jakavi therapy. Women taking Jakavi should not breast feed.

The most common adverse drug reactions, occurring at any level of severity (incidence >10%) are urinary tract infections, anemia, thrombocytopenia, neutropenia, hypercholesterolemia, dizziness, headache, alanine aminotransaminase increased, asparte aminotransferase increased, bruising, bleeding and increased blood pressure. Other common adverse drug reactions (incidence 1 to 10%) are herpes zoster, weight gain, flatulence and tuberculosis (1%). Progressive multifocal leukencephalopathy (PML) has been reported. Physicians should be alert for neuropsychiatric symptoms suggestive of PML.

Please see full Prescribing Information available at www.jakavi.com.

About Afinitor (everolimus)
Everolimus is approved as Afinitor® in the European Union for the treatment of hormone receptor-positive, HER2 negative (HR+/HER2 negative) advanced breast cancer, in combination with exemestane, in postmenopausal women without symptomatic visceral disease after recurrence or progression following a non-steroidal aromatase inhibitor. In the United States, Afinitor is approved for the treatment of postmenopausal women with advanced hormone receptor-positive, HER2 negative breast cancer (advanced HR+/HER2 negative breast cancer) in combination with exemestane after failure of treatment with letrozole or anastrozole.

Afinitor (everolimus) tablets is approved in more than 100 countries, including the United States and throughout the European Union, in the oncology settings of advanced renal cell carcinoma following progression on or after vascular endothelial growth factor (VEGF)-targeted therapy, and in the United States and European Union for locally advanced, metastatic or unresectable progressive neuroendocrine tumors of pancreatic origin.

Everolimus is also available from Novartis for use in certain non-oncology patient populations under the brand names Afinitor® or Votubia®, Certican® and Zortress® and is exclusively licensed to Abbott and sublicensed to Boston Scientific for use in drug-eluting stents.

Indications vary by country and not all indications are available in every country. The safety and efficacy profile of everolimus has not yet been established outside the approved indications. Because of the uncertainty of clinical trials, there is no guarantee that everolimus will become commercially available for additional indications anywhere else in the world.

Important Safety Information about Afinitor (everolimus) tablets
Afinitor/Votubia can cause serious side effects including lung or breathing problems, infections (including sepsis), and kidney failure, which can lead to death. Mouth ulcers and mouth sores are common side effects. Afinitor/Votubia can affect blood cell counts, kidney and liver function, and blood sugar, cholesterol, and triglyceride levels. Afinitor/Votubia may cause fetal harm in pregnant women. Highly effective contraception is recommended for women of child-bearing potential while receiving Afinitor/Votubia and for up to eight weeks after ending treatment. Women taking Afinitor/Votubia should not breast feed. Fertility in women and men may be affected by treatment with Afinitor/Votubia.

The most common adverse drug reactions (incidence >=10 percent) are mouth ulcers, skin rash, feeling tired or weak, diarrhea, nausea, decreased appetite, infections (including upper respiratory tract infection), low level of red blood cells, abnormal taste, inflammation of lung tissue, weight loss, swelling of extremities or other parts of the body, nose bleeds, itching, vomiting, high level of blood cholesterol, headache, high level of blood sugar, cough, spontaneous bleeding or bruising, and breathlessness. The most common Grade 3-4 adverse drug reactions (incidence >=2 percent) are mouth ulcers, feeling tired or weak, infections, inflammation of lung tissue, diarrhea, spontaneous bleeding or bruising, low white blood cells (a type of blood cell that fights infection), and breathlessness. Cases of hepatitis B reactivation, blood clots in the lung or legs, and menstruation disorders such as absence of periods have been reported. Abnormalities were observed in hematology and clinical chemistry laboratory tests.

Please see full Prescribing Information available at www.afinitor.com.

About Exjade (deferasirox)
Exjade is an oral iron chelation therapy indicated for the treatment of chronic iron overload due to frequent blood transfusions (>=7 ml/kg/month of packed red blood cells) in patients with beta-thalassemia aged 6 years and older). It is also indicated for the treatment of chronic iron overload due to blood transfusions when deferoxamine therapy is contraindicated or inadequate in the following patient groups: patients with beta-thalassemia major with iron overload due to frequent blood transfusions (>=7 ml/kg/month of packed red blood cells) aged 2 to 5 years; patients with beta-thalassemia major with iron overload due to infrequent blood transfusions (<7 ml/kg/month of packed red blood cells) aged 2 years and older; and patients with other anemias aged 2 years and older. Exjade is also indicated for the treatment of chronic iron overload requiring chelation therapy when deferoxamine therapy is contraindicated or inadequate in patients with non-transfusion-dependent thalassemia syndromes aged 10 years and older.

It is approved in more than 100 countries including the US, Switzerland, Japan and countries comprising the EU. The approved indication may vary depending upon the individual country.

Exjade Important Safety Information
Exjade is contraindicated in patients with an estimated creatinine clearance <60 mL/min, with hypersensitivity to the active substance or any of the excipients, or in combination with other iron chelator therapies. Exjade is not recommended in patients with severe hepatic impairment.

There have been postmarketing reports of acute renal failure, hepatic failure and cytopenias. Renal failure requiring temporary or permanent dialysis, renal tubulopathy and interstitial nephritis have been reported. Upper gastrointestinal ulceration and hemorrhage, sometimes fatal, have been reported. Caution should be used in elderly patients due to a higher frequency of adverse reactions. Exjade is not recommended in patients with a short life expectancy (e.g., high-risk myelodysplastic syndromes), especially when co-morbidities could increase the risk of adverse events.

Severe skin reactions (including cases of Stevens-Johnson syndrome), serious hypersensitivity reactions, decreased hearing and lens opacities have been reported. The most common adverse reactions are nausea, vomiting, diarrhea, abdominal pain, rash, pruritus, non-progressive increases in serum creatinine, increased transaminases, abdominal distension, constipation, dyspepsia, proteinuria and headache.

Please see full Prescribing Information available at www.exjade.com.

About PKC412, CTL019, LDE225, LBH589, BKM120 and BYL719
Because these are investigational treatments, the safety and efficacy profile of PKC412, CTL019, LDE225, LBH589, BKM120 and BYL719 have not yet been established. Access to these investigational treatments is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the treatments. Because of uncertainty of clinical trials, there is no guarantee that PKC412, CTL019, LDE225, LBH589, BKM120 and BYL719 will ever be commercially available anywhere in the world.

The foregoing release contains forward-looking statements that can be identified by words such as "to highlight," "investigational," "will," "emerging," "underway," or similar terms, or by express or implied discussions regarding potential marketing approvals for PKC412, CTL019, LDE225, LBH589, BKM120 and BYL719, potential new indications or labeling for Tasigna, Glivec, Jakavi, Afinitor, and Exjade, or regarding potential future revenues from any of the foregoing investigational compounds and products. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that any of PKC412, CTL019, LDE225, LBH589, BKM120 or BYL719 will be submitted or approved for sale in any market, or at any particular time, or that any of Tasigna, Glivec, Jakavi, Afinitor, or Exjade will be submitted or approved for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that any of the foregoing investigational compounds or products will receive regulatory approval or be commercially successful in the future. In particular, management`s expectations regarding such investigational compounds and products could be affected by, among other things, the uncertainties inherent in research and development, including unexpected clinical trial results and additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company`s ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; unexpected manufacturing issues, and other risks and factors referred to in Novartis AG`s current Form 20-F on file with the US Securities and Exchange Commission. 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 133,000 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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[1] American Society of Hematology. ASH Annual 2013 Meeting Program. Available at: https://ash.confex.com/ash/2013/webprogram/start.html
[2] San Antonio Breast Cancer Symposium. SABCS Annual 2013 Meeting Program. Available at: http://www.sabcs.org/ProgramSchedule/index.asp

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[*]Known as Gleevec® (imatinib mesylate) tablets in the US, Canada and Israel.

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