Pharmacy Daily | Bronchitol phase 3 failure

AUSTRALIAN pharmaceutical research firm Pharmaxis has been dealt a significant blow, with the failure of a phase 3 clinical trial assessing the effectiveness of its respiratory medicine Bronchitol in people with bronchiectasis. According to an ASX release, top-line results of the double-blind, placebo controlled, randomised trial showed an 8% reduction in exacerbation rates among patients treated with Bronchitol compared with control, “which was not statistically significant”. The result saw Pharmaxis shares hammered, trading at just 15c each, down more than 50%. The company said that although the study had failed its primary endpoint, “secondary endpoints on efficacy and safety” had been met. And “the positive trend in the primary endpoint was supported by a number of statistically significant secondary endpoints,” the Pharmaxis statement said. “These included a delay in the time to a first exacerbation, reduced days on antibiotics and improved quality of life”. The 52-week study of 485 subjects was the largest ever undertaken in bronchiectasis, taking place across 84 hospitals in Australia, New Zealand, North and South America and Europe. Bronchiectasis is an obstructive lung disease characterised by localised, irreversible dilation of part of the lung due to destruction of muscle and elastic tissue. Pharmaxis ceo Gary Phillips said that due to the results of the trial the company would not be proceeding immediately with a regulatory submission for bronchiectasis. However an analysis of the large amount of data collected in the trial suggests that Bronchitol performs better in some patients with high disease burden. “It seems likely that a clinically meaningful subgroup of Bronchitol patients can be identified to form the basis for discussions with regulatory authorities on approvable clinical trial designs,” Phillips said. Bronchitol (mannitol) is an inhaled dry powder designed to hydrate the lung and restore normal lung clearance mechanisms, and is approved in Australia and Europe for the treatment of cystic fibrosis. Bronchitol also has orphan drug designation for bronchiectasis in the USA - but does not have US approval for cystic fibrosis.

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