ph 3 trial fails primary endpoint

Media Release 24 April 2013
PHASE 3 CLINICAL TRIAL IN BRONCHIECTASIS (B305) FAILS PRIMARY ENDPOINT
MEETS KEY SECONDARY ENDPOINTS ON EFFICACY AND SAFETY
Pharmaceutical company Pharmaxis (ASX: PXS) today announced its Phase 3 clinical trial (B305)
assessing the effectiveness of Bronchitol® in people with bronchiectasis had not met the trial’s primary
endpoint of demonstrating a significant difference in the rates of defined pulmonary exacerbations in
patients treated over a 12 month period.
Top line results of the double blind, placebo controlled, randomised trial showed an eight (8) per cent
reduction in exacerbation rates among patients treated with Bronchitol compared with control which
was not statistically significant.
The positive trend in the primary endpoint was supported by a number of statistically significant
secondary endpoints. These included a delay in the time to a first exacerbation, reduced days on
antibiotics and improved quality of life.
The trial results show Bronchitol has an acceptable safety profile in the patient population studied with
no overall difference in the numbers of patients experiencing adverse events or serious adverse events
in the Bronchitol and control groups.
The 52 week study of 485 subjects was the largest ever undertaken in bronchiectasis and the first to
look at the rate of exacerbations over 12 months. The study took place across 84 hospitals in Europe,
North America, South America, Canada, Australia and New Zealand.
Pharmaxis Chief Executive Officer, Mr Gary Phillips said, “It is disappointing not to have achieved the
primary endpoint in this trial. We will therefore not be proceeding immediately with a regulatory
submission for bronchiectasis. This trial, however, was a global first and is valuable both to Pharmaxis
and the health professionals treating patients with bronchiectasis. The very large data set contains
more than 43 thousand electronic pages of patient information. A full analysis will take some time but
encouragingly, available data suggests that Bronchitol performs better in some patients with high
disease burden.
“Bronchiectasis is a heterogeneous disease and, while there is more analysis to be undertaken, 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.
“Over the coming months Pharmaxis will explore the options available for advancing the clinical
development program in bronchiectasis. I would like to thank the patients in the trial, their clinicians
and the team at Pharmaxis. This was a large scale undertaking in a disease that is difficult to assess and
I am proud of the effort made in working towards a treatment for this difficult and potentially fatal
disease.”
Dr Diana Bilton, Consultant Respiratory Physician at Royal Brompton Hospital, London, said,
“Bronchiectasis is a serious and disabling lung condition for which there are very few treatment
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options. Historically, reducing exacerbations in respiratory conditions like COPD and bronchiectasis has
proven difficult. The secondary endpoints in this study of improving quality of life and increasing the
time to suffering an exacerbation are highly clinically relevant and the data suggests that Bronchitol is
worth developing in a well-defined patient population.”
Mr Phillips said, “A presentation of the findings of the B305 study will be made at an international scientific
respiratory conference where I expect it will attract significant attention. B305 is a pioneering study in this
disease which affects 600,000 people in the major pharmaceutical markets but for which no products have
been approved to assist with mucus clearance.”
Bronchitol (mannitol) is an inhaled dry power designed to hydrate the lung and restore normal lung
clearance mechanisms. It is approved in Australia and Europe (adults) for the treatment of the genetic
disease cystic fibrosis. Bronchitol has orphan drug designation for bronchiectasis in the US.