Enrolment on phase III cystic fibrosis trial completed

US-based Inspire Pharmaceuticals has completed patient enrolment in TIGER-1, the first of two planned pivotal phase III clinical trials evaluating denufosol inhalation solution for the treatment of cystic fibrosis (CF).

The company says it expects efficacy data from TIGER-1 to be available mid-2008, somewhat earlier than previously announced.

TIGER-1, which was initiated in July 2006, is a double-blind, placebo-controlled, randomised study comparing 60 mg of denufosol to placebo, administered three times daily by jet nebuliser, in approximately 350 patients with mild CF lung disease. The approximate mean age of patients enrolled in the study is 14 years.

The trial includes a 24-week efficacy and safety portion, followed by a 24-week open-label denufosol safety extension. Of the patients who have completed the efficacy portion to date, approximately 98 per cent are currently enrolled in the safety extension. The primary efficacy endpoint is change from baseline in FEV1 (forced expiratory volume in one second) in litres at the 24-week stage.

Inspire also revealed the timings of the second phase III TIGER-2 clinical trial evaluating denufosol. The company said it would work with select CF treatment centres in the US and Canada for the remainder of 2007 to prepare for patient enrolment. The company added that it would begin international site enrolment in the H2 2008, as additional countries become eligible to be included from a regulatory perspective.

Dr Christy L Shaffer, CEO of Inspire, said: ìWe are very excited to have reached this milestone in the phase III development of denufosol, a novel compound that has the potential to treat the underlying cause of CF.î

Dr Frank J Accurso, director of the paediatrics and cystic fibrosis centre at the University of Colorado and lead principal investigator of TIGER-1, said: ìThe smooth and rapid enrolment in TIGER-1 demonstrates a strong interest in denufosol as a potential part of the treatment regimen for mild CF patients. An early intervention treatment for CF could be an important part of treating this disease.î

The incidence of CF is approximately 1 in 2500 live births with the disease currently affecting 8,000 people in the UK (the region with the highest incidence) and 70,000 people worldwide. The estimated global market value for a new CF treatment is in the region of USD 75.2m, according to a report by the University of Dundee.

In September 2007, UK-based Lamellar Biomedical received financing of nearly USD 2m from a consortium of investors to develop a CF treatment. The company said the money came from business angel group Tri-Capital, Barwell and Scottish Enterprise's Co-Investment Fund.