Actelion Reports Positive RAPIDS-1 Results; Actelion Reports Highly Significant Study Results With Tracleer in Digital Ulcers
Study Enrolling Scleroderma Patients Meets Primary Endpoint; Prevention of New Digital Ulcers; Application for Orphan Drug Status Forthcoming
ALLSCHWIL/BASEL, Switzerland, Aug. 15, 2002 (PRIMEZONE) -- Actelion Ltd (SWX:ATLN) announced today preliminary results from RAPIDS-1, evaluating the efficacy and safety of Tracleer(TM) in the treatment of digital ulcerations in scleroderma patients. The primary endpoint of the study, the prevention of new digital ulcers, was achieved. This result was highly statistically significant.
Isaac Kobrin, Head of Clinical Development, said: ``I am very pleased with these promising results. We knew already that scleroderma patients benefit from Tracleer(TM) treatment if they suffer from pulmonary arterial hypertension, for which the drug is already approved. Now we have evidence that scleroderma patients suffering from digital ulcers may also benefit from Tracleer(TM) treatment. Moreover, this randomized study has again confirmed the safety and tolerability profile of Tracleer(TM) in the scleroderma patient population.''
Actelion will immediately start the process to obtain a second Orphan Drug designation for Tracleer(TM), this time for digital ulcerations in scleroderma patients. The company will also discuss the full results with authorities worldwide to determine the appropriate procedures for a regulatory filing. Full results of RAPIDS-1 will be submitted for presentation at upcoming scientific meetings, with publication to follow.
Tracleer(TM), the first orally available dual endothelin receptor antagonist, is already approved and available in major markets worldwide for the treatment of pulmonary arterial hypertension (PAH), a chronic life-threatening condition that severely compromises the function of the lungs and heart. Between 15 to 30 percent of all scleroderma patients develop PAH, the leading cause of mortality in these patients.
Tracleer(TM) in Digital Ulcers - Investigator comments Digital ulcerations are also a common complication of scleroderma, occurring in 25 percent or more of patients. Digital ulcerations are very painful and difficult to heal open sores, occurring on fingers and toes, as a result of the blockage of small blood vessels (obliterative vasculopathy). In severe cases, where gangrene might develop, surgery and even amputation may be required.
The lead investigator of Rapids-1, Carol Black, CBE, MD, Professor of Rheumatology, Royal Free Hospital, London, said: ``I am delighted with the results of this study. For the first time in a placebo-controlled study, an oral drug has been shown to prevent new digital ulcers in scleroderma patients. These findings, taken together with the proven efficacy of Tracleer(TM) in pulmonary arterial hypertension, are clearly supportive of the hypothesis that endothelin plays a fundamental role in vasculopathy. In addition, endothelin may also contribute to the pathophysiology of scleroderma itself.''
The lead investigator in the United States, Joseph H. Korn, MD, Professor of Medicine and Biochemistry, School of Medicine, Boston University, said: ``The finding that Tracleer(TM) prevents the occurrence of new digital ulcers in scleroderma patients is both statistically significant and also clinically relevant. The decrease in digital ulcers means that patients will have less pain and improved function as well as a lower risk of serious finger infection.''
The study RAPIDS-1 (RAndomized Placebo-controlled Investigation of Digital ulcers in Scleroderma) was an international, multi-center, double blind, placebo-controlled clinical trial evaluating the prevention of ischemic digital ulcers in 122 patients with systemic sclerosis (scleroderma). Patients were randomized in a 2:1 allocation to either Tracleer(TM) or placebo for a sixteen-week treatment period. Patients had to either have active digital ulcers at entry into the study or to have a history of digital ulcerations in the preceding twelve months. |
