Great Strides 2008
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Medical News Briefs


Enzyme Replacement Therapy Achieves Good Results in Phase 3 Trials

Altus Pharmaceuticals released positive Phase 3 trial results for Trizytek (formerly ALTU-135), its oral pancreatic enzyme replacement therapy. Trizytek is a combination of three active enzymes designed to improve fat, protein and carbohydrate absorption in patients with pancreatic insufficiency. In the trial, Trizytek demonstrated significant improvement of fat and protein absorption in people with CF. Current enzyme therapies require several pills per meal, while Trizytek provides a one capsule dosing.

PTC Expands Collaboration with Foundation

PTC Therapeutics, Inc. announced positive results from a Phase 2a trial of PTC124, an oral drug for the treatment of CF. The drug improved the function of the CFTR protein and decreased the frequency of cough in CF patients. Following the results, the Foundation expanded its collaboration with PTC. The company will receive up to $25 million to perform additional studies of the drug. PTC124 targets “nonsense mutations,” which are changes in the CF gene that prematurely stop the production of the full-length CFTR protein. Approximately 10 percent of people with CF have nonsense mutations.

FoldRx Achieves Research Milestone

FoldRx Pharmaceuticals, Inc. announced the discovery of a new series of drug prototypes, which demonstrated potential in the laboratory for correcting the protein-folding defect associated with CF. This discovery may pave the way for new drugs capable of treating the basic defect in CF. CFFT will award up to $23.7 million over five years to FoldRx to facilitate the continued discovery and development efforts for this series of compounds.

Transave Antibiotic Formulation Shows Positive Results in Phase 2 Trial

Transave, Inc. released positive results from a Phase 2 clinical trial of Arikace™ (liposomal amikacin for inhalation). The antibiotic treatment aims to address Pseudomonas aeruginosa lung infections. Data from the trial indicate that Arikace produced significant improvement in lung function and was well-tolerated by patients. Those taking the drug also experienced fewer pulmonary exacerbations and hospitalizations. A form of the antibiotic amikacin, Arikace uses a new delivery technology to prolong the release of amikacin in the lungs and minimize systemic exposure.