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Once-Daily Exjade Shown to Remove Toxic Iron From the Heart and Liver, According to Data Presented at ASH

Approximately 78% of Beta-thalassaemia Patients had Decreases in Cardiac Iron and 90% had Decreases in Liver Iron After Six Months, Interim Data Show(1)

FRIMLEY, England, December 12, 2007 /PRNewswire-FirstCall/ -- New data show once-daily Exjade(R) (deferasirox) reduces life-threatening iron levels in the heart and liver in beta-thalassaemia patients. These interim results from an ongoing trial show that at, six months, approximately 78 percent of participants had decreases in cardiac iron and 90 percent of patients had decreases in hepatic iron. These results were reported at the 49th Annual Meeting of the American Society of Haematology (ASH) in Atlanta, 8-11 December 2007.(1)

Chronic iron overload is a potentially life-threatening condition that results from frequent blood transfusions required to treat certain types of chronic blood disorders, including sickle cell disease (SCD), thalassaemia, and myelodysplastic syndromes (MDS) and other anaemias. If left undiagnosed or untreated, excess iron in the body can become toxic, causing damage to the heart and liver, ultimately leading to death.(4)

"These preliminary data are very encouraging. At a dose of 30 mg/kg, Exjade lowered both heart and liver iron levels in most patients," said Dr Farrukh Shah, Consultant Haematologist, The Whittington Hospital NHS Trust. "These data add to the growing body of evidence that Exjade successfully removes iron from the heart. This is particularly important because iron cardiotoxicity remains the leading cause of death in thalassaemia major patients."

The body has no inherent mechanism to remove excess iron, so iron chelation is used as an effective treatment for transfusion-related iron overload.(5) Prior to oral iron chelation, patients relied on chelation therapy that had to be administered by nightly infusions by needle and pump, often lasting 8-12 hours a night for 5 to 7 nights a week. Once-daily Exjade oral monotherapy offers patients effective iron reduction, without the need for any infused chelation therapy.(6)

Long-term efficacy and safety demonstrated in sickle cell disease Patients(2)

Interim results after 2.7 years of a separate four-year extension study demonstrates the long-term, dose-dependent efficacy and safety of treatment with Exjade for chronically transfused patients with SCD. Patients treated in the core study with doses of 20 and 30 mg/kg/day showed continued decline in serum ferritin (SF), an indication of iron buildup in the body. For patients initially treated with 5 and 10 mg/kg/day doses in the core study, SF levels gradually declined following a dose increase to approximately 20 mg/kg/day. There were no significant changes in markers of liver or renal function and no cases of progressive increases in serum creatinine (SCr). Additionally, no new adverse events or safety concerns have been reported so far in the extension study.

Exjade demonstrates safety and efficacy in lower-risk MDS patients(3)

Additional research demonstrated that treatment with Exjade decreased mean SF levels over one year in patients with low- or intermidiate-1 IPSS risk MDS. Additionally, 100 percent of patients experienced a stabilised labile plasma iron (LPI) over 12 months, indicating 24-hour sustained suppression of toxic iron with Exjade. Exjade was shown to have a manageable safety profile in this population. Ongoing assessments of this trial, evaluating cardiac, hepatic and endocrine function, will determine the impact of iron reduction with Exjade on morbidity and mortality in MDS.

These data provide further context to a separate study presented at this year's meeting, which demonstrates that chelation therapy provides a significant survival benefit for heavily-transfused patients with low and intermediate MDS. A prospective study of MDS patients found that the median overall survival from diagnosis was 115 months in chelated patients versus 51 months in non-chelated patients (p less than 0.0001).(7)

    Notes to Editors

    1) For further information, please contact:


    Fiona Turner

    Novartis Pharmaceuticals UK Ltd

    Email: 


    Lucy Twitchin

    Red Health

    Email:

    



    2) About the studies
fiona.turner@novartis.com lucy.twitchin@redconsultancy.com

Exjade(R)Reduces Cardiac Iron Burden in Chronically Transfused Beta-Thalassaemia Patients: And MRI T2xStudy.(1)

This study is a prospective, single-arm, phase II trial of 18 chronically transfused beta-thalassemia patients. During this trial, SF levels are tested monthly and liver and cardiac iron concentration levels are tested every 6 months. This study will enroll 30 patients at 4 U.S. centers, with Exjade administered at 30-40 mg/kg/day for the next 12 to 18 months. The ongoing assessments will determine whether Exjade continues to improve cardiac iron burden and maintain or improve cardiac function in severely iron-overloaded patients. Data from other larger studies measuring the effects of Exjade on cardiac iron reduction and cardiac function will be available next year.

Long-Term Efficacy and Safety of Deferasirox (Exjade(R), ICL670), a once-Daily Oral Iron chelator, in Patients with Sickle Cell Disease (SCD).2

The SCD study, an extension phase of an ongoing phase II study comparing Exjade with desferrioxamine, evaluated the safety and efficacy of Exjade at 20-30 mg/kg/day in 159 patients over a 2.7-year period. Patients in the desferrioxamine arm of the extension trial were allowed to cross over to Exjade for the extension trial.

Deferasirox (ICL670;Exjade(R)) Reduces Serum Ferritin (SF) and Labile Plasma Iron (LPI) in Patients with Myelodysplastic Syndromes (MDS).3

The third study, is a phase II, open-label, 3-year trial in 176 patients with low- or intermediate-1 IPSS risk MDS and transfusional iron overload (SF 1000 g/L and greater than 20 units red blood cell (RBC) transfusions), with SCr within two-fold the upper limit of normal (ULN). Initial Exjade dose was 20 mg/kg/day and could be increased to 40 mg/kg/day based on tolerability and response. SF was monitored monthly and LPI was assessed quarterly.

Positive Impact of Iron Chelation Therapy on Survival in Regularly Transfused MDS Patients. A Prospective Analysis by the GFM.7

The MDS survival results are from a prospective survey of hematological data in 170 MDS patients from 18 Groupe Francophone des Myelodysplasies Centers who were referred for blood transfusions during a one-month period (May 15-June 15, 2005). Survival was analysed two years later, at the reference date of May 15, 2007.

3) About Exjade

Now approved in more than 85 countries, including the EU and the US, Exjade is the first once-daily oral iron chelator approved for use in patients with transfusional iron overload who have a wide range of underlying diseases such as thalassaemia, sickle cell disease and myelodysplastic syndromes. Exjade is administered as a drink after the tablets are dispersed in a glass of water, apple, or orange juice.

4) About thalassaemia

Thalassaemia refers to a group of genetic blood disorders that affect the body's production of haemoglobin in the red blood cells. Without treatment, the most severe form of thalassaemia can be fatal. It can also cause jaundice, weak bones and an enlarged spleen.(8)

5) About sickle cell disease

Sickle cell disease is an inherited blood disorder where red cells become deformed ('sickle' shaped) and tend to get stuck in the small blood vessels. This blockage can cause pain, damage tissue and organs and lead to stroke if it occurs in the brain.(9)

6) About myelodysplastic syndromes (MDS)

Myelodysplastic syndromes (MDS) are a group of blood disorders marked by overproduction of defective blood cells. Approximately one third of cases progress to acute leukaemia, a fatal blood cancer.(10)

7) About Novartis

Novartis AG is a world leader in offering medicines to protect health, cure disease and improve well-being. Our goal is to discover, develop and successfully market innovative products to treat patients, ease suffering and enhance the quality of life. We are strengthening our medicine-based portfolio, which is focused on strategic growth platforms in innovation-driven pharmaceuticals, high-quality and low-cost generics, human vaccines and leading self-medication OTC brands. Novartis is the only company with leadership positions in these areas. In 2006, the Group's businesses achieved net sales of USD 37.0 billion and net income of USD 7.2 billion. Approximately USD 5.4 billion was invested in R&D. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 100,000 associates and operate in over 140 countries around the world. For more information, please visit http://www.novartis.com.

References

1. Wood, J et al. Exjade(R) reduces Cardiac Iron Burden in Chronically Transfused Beta-Thalassaemia Patients: And MRI T2xStudy. Poster presented at the American Society of Haematology, 8-11 December 2007, Atlanta.

2. Vichinsky, E et al. Long-Term Efficacy and Safety of Deferasirox (Exjade(R), ICL670), a once-Daily Oral Iron chelator, in Patients with Sickle Cell Disease (SCD). Poster presented at the American Society of Hematology, 8-11 December 2007, Atlanta

3. List, AF et al. Deferasirox (ICL670; Exjade(R)) /Reduces Serum Ferritin (SF) and Labile Plasma Iron (LPI) in Patients with Myelodysplastic Syndromes (MDS). Poster presented at the American Society of Haematology, 8-11 December 2007, Atlanta

4. Kushner, JP et al, Secondary Iron Overload. Haematology 2001 Jan 1: 47-61

5. Porter, JP. A risk-benefit of iron-chelation therapy. Drug Saf. 1997 Dec; 17(6) 407-21.

6. Exjade summary of product characteristics, Novartis, August 2006

7. Rose, C et al. Positive Impact of Iron Chelation Therapy on Survival in Regularly Transfused MDS Patients. A Prospective Analysis by the GFM. Oral session at the American Society of Haematology, 8-11 December 2007, Atlanta

8. Yaish HM. e Medicine. Thalassaemia. Available at: http://www.emedicine.com/ped/topic2229.htm. Last accessed 10 December 2007

9. National Heart, Kung and Blood Institute at the National Institute of Health. What are the Signs and Symptoms of Sickle Cell Anaemia? Available at: http://www.nhlbi.nih.gov/health/dci/Diseases/Sca/SCA_SignsAndSymptoms.html. Last accessed 10 December 2007

10. Kouides P et al. Understanding the Myelodysplastic Syndromes. The Oncologist. 1997 2:389-401

CONTACT: For further information, please contact: Fiona Turner, NovartisPharmaceuticals UK Ltd, Tel: +44(0)1276-698086, Fax: +44(0)1276 698605,Email: ; Lucy Twitchin, Red Health, Tel:+44(0)207-025-6561, Email: fiona.turner@novartis.com lucy.twitchin@redconsultancy.com

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Posted: December 2007

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