• Inspire Pharmaceuticals Inc., of Durham, N.C., reported results of a 28-day Phase II study of denufosol inhalation solution in cystic fibrosis patients, ages 5 to 7, showing that both the 20-mg and 60-mg doses of the drug given three times daily were well tolerated, with a safety profile similar to placebo. Data were presented at the 2006 North American Cystic Fibrosis Conference in Denver. Inspire has initiated the first of two planned Phase III trials to evaluate 60 mg of denufosol vs. placebo in 350 CF patients.

• Neurochem Inc., of Laval, Quebec, said Neurology published online a version of an article on the Phase II trial with Alzhemed (tramiprosate, or 3-amino-1-propanesulfonic acid) in mild to moderate Alzheimer's disease patients. The drug had no significant impact on vital signs and laboratory test values. The most frequent side effects were nausea, vomiting and diarrhea, which were intermittent and mild to moderate in severity. Alzhemed crossed the blood-brain barrier, and dose-dependently reduced CSF A(beta)42 levels after three months of treatment. Results on psychometric measures showed no differences between groups over the three-month double-blind period, consistent with the proposed mechanism of action, since the drug is not designed to confer short-term symptomatic benefits.

• Protherics plc, of London, and AstraZeneca plc, also of London, disclosed the latter's plan to expand the development efforts for CytoFab, a treatment for severe sepsis, with the addition of a 48-patient Phase II program.

• PTC Therapeutics Inc., of South Plainfield, N.J., disclosed findings from two Phase II trials of PTC124 in patients with cystic fibrosis due to a nonsense mutation. The results suggested that PTC124 can restore function of the cystic fibrosis transmembrane conductance regulator protein in airway cells and significantly reduce blood neutrophil counts that are a hallmark of the CF disease process. The initial data were presented at the North American Cystic Fibrosis Conference in Denver.

• Ziopharm Oncology Inc., of New York, reported updated data from Phase I studies of ZIO-201, a form of the active metabolite of ifosfamide, which demonstrated evidence of clinical activity in sarcoma (two of 11 patients, including at least one who had failed ifosfamide [IFOS] therapy) and mesothelioma (one patient with extended stable disease). Patients received ZIO-201 daily for three consecutive days each four weeks. Data were presented at the Connective Tissue Oncology Society meeting in Venice.

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