ALLSCHWIL, Switzerland, Dec. 21, 2004 (PRIMEZONE) -- Actelion Ltd (Other OTC:ALIOF) (SWX:ATLN) announced today that the pivotal study evaluating safety and efficacy of miglustat (Zavesca(R)) in patients with Late Onset Tay-Sachs disease (LOTS) will continue -- as planned -- for another 12 months. A recently concluded safety interim analysis confirmed the existing safety profile for Zavesca(R) also at the high doses used in this trial.
The randomized open-label study is following 30 patients receiving either the current standard care for LOTS (n=10) or miglustat 200 mg three times a day (n=20). According to study protocol, this regimen is followed for 12 months, and then all patients receive miglustat for another 12 months. The dose is the double of the dose recommended for type 1 Gaucher disease, in order to increase brain exposure to miglustat.
The evaluation of the 12-months data shows that miglustat at 200 mg tid dose is well tolerated and confirms the safety profile established in Gaucher's patients. After 12 months follow-up, 29 of the 30 randomized patients continued in the trial.
Several clinical and computerized assessments are conducted throughout the study, focusing for example on potential changes in eye movement or speech patterns. Efficacy data will become available at the end of this study, expected sometime in late 2005 or early 2006.
About Late Onset Tay-Sachs disease (LOTS)
Tay-Sachs disease, one of the lysosomal storage disorders, is an autosomal recessive genetic disease affecting the central nervous system. It is the most common form of GM2 gangliosidoses. GM2 is a component of neuronal cells membranes, and is normally degraded by the enzyme B-hexosaminidase A (B-HexA). In Tay-Sachs disease, this enzyme is absent or deficient causing the deleterious accumulation of GM2 in the brain. In Late Onset Tay-Sachs (LOTS), clinical manifestations generally start in the third decade, progress very slowly, and are very heterogeneous. Neurological symptoms include slurred speech, muscle weakness, tremors, unsteady gait, and psychoses. There is currently no drug therapy available for the treatment of LOTS.
About Zavesca(R)
Zavesca(R) (miglustat) is the first oral treatment option for type 1 Gaucher disease, a rare genetic disorder where an accumulation of GSL leads to liver and spleen enlargement/dysfunction, anemia, thrombocytopenia, bone disease and pain. It is the first in a new class of drugs known as substrate reduction therapy (SRT). Zavesca(R) is approved and available in the European Union, United States, Canada, Israel and Switzerland.
Zavesca(R) safety information
Peripheral neuropathy has been reported in type 1 Gaucher patients treated with Zavesca(R). Patients should undergo a neurological exam at the start of treatment and regularly thereafter. Zavesca(R) should be reassessed in patients who develop symptoms of peripheral neuropathy. Zavesca(R) may cause fetal harm if administered to a pregnant woman and is contraindicated in women who are or who may become pregnant; patients should be apprised of the potential hazard to the fetus. There is a risk of impaired fertility in men. Men should maintain reliable contraceptive methods and not plan to conceive while taking Zavesca(R) and for 3 months thereafter.
Actelion Ltd
Actelion Ltd is a biopharmaceutical company with its corporate headquarter in Allschwil/Basel, Switzerland. Actelion's first drug Tracleer(R), an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets Tracleer(R) through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union as well as Canada, Australia and Switzerland. Actelion, founded in late 1997, is a leading player in innovative science related to the endothelium - the single layer of cells separating every blood vessel from the blood stream. Actelion focuses on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SWX Swiss Exchange (ticker symbol: ATLN).
For further information please contact:
Actelion Ltd, Gewerbestrasse 16, CH-4123 Allschwil
Investor Contact Roland Haefeli +41 61 487 34 58
+1 650 624 6936
Media Contact Peter Engel +41 61 487 36 28
+1 650 624 6996
http://www.actelion.com