Great Strides 2007
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Medical News Briefs

Vertex Potentiator Continues to Climb Clinical Trial Ladder

VX-770, a compound in clinical trials designed by Vertex Pharmaceuticals to address a core defect in CF, has entered Phase 2 clinical testing and is currently enrolling approximately 36 adult volunteers with cystic fibrosis at more than a dozen care centers across the country. This trial studies the safety, tolerability and absorption rate of VX-770. Restoration of CFTR activity is also being studied. The compound, which belongs to a category of compounds called CFTR potentiators, is believed to partially restore CFTR protein function and increase the probability that the CFTR channel is open. VX-770 may allow for increased chloride transport across the cell surface, alleviating a fundamental problem in CF. The previous Phase 1 trial, completed in 2006, showed that the potential drug could achieve the expected levels in the blood.

Enzyme Replacement Therapy Enters Final Stage of Testing

Altus Pharmaceuticals has begun enrolling for its Phase 3 clinical trials to evaluate the efficacy and safety of its oral pancreatic enzyme replacement therapy. This latest phase of clinical testing for ALTU-135 involves more than 300 patients at approximately 50 sites in the United States. Pancreatic insufficiency is found in approximately 90 percent of individuals with CF and the consequences are treated through the administration of enzymes that help patients to enhance digestion, improve growth, reduce gastrointestinal problems and contribute to general nutritional health. In a previous Phase 2 study, ALTU-135 was found to be well-tolerated and achieved statistically significant improvements in the absorption of fat and protein, as well as an overall improvement in the absorption of carbohydrates. If results from the Phase 3 trials are positive, Altus will file for Food and Drug Administration approval in the U.S.

Denufosol Phase 2 Results “Inspire” Initiation of Phase 3 Testing

Inspire Pharmaceuticals recently completed enrollment for a Phase 3 clinical trial evaluating the safety and effectiveness of inhaled denufosol, which is designed to enhance the hydration and clearance of mucus in the lungs of patients with CF. The trial enrolled approximately 350 CF volunteers age 5 and older and is being conducted at nearly 100 care centers across the United States. This latest trial compares denufosol to a placebo in patients with mild CF lung disease. Inspire will soon open enrollment for a second international Phase 3 trial, which will include a limited number of U.S. sites. Results of a Phase 2 trial showed that patients receiving the study drug had significantly better lung function than those receiving a placebo. Denufosol targets insufficient chloride movement, a fundamental problem in CF, by stimulating the movement of chloride in and out of CF cells, thus circumventing the faulty CFTR protein channel.