Phase III Trial Finds Pharmaxis's Bronchitol Effective; Two Primary Efficacy Endpoints Met
SYDNEY, Australia, Aug. 28 -- Pharmaxis today announced that its Phase III B301 study of Bronchitol for the treatment of people with bronchiectasis has met its two primary efficacy endpoints: quality of life and mucus clearance.
Data from the 362 subject study demonstrated a highly significant improvement in quality of life after 12 weeks of treatment with Bronchitol as assessed by the St George Respiratory Questionnaire, a patient reported outcome tool for measuring health-related quality of life, (p-value less than 0.005) and a significant improvement in quality of life compared to placebo (p-value less than 0.05).
In addition, there was a highly significant difference in mucus clearance at 12 weeks for patients receiving Bronchitol versus those patients receiving placebo (p-value less than 0.001).
There were no serious adverse events attributable to treatment and the incidence of adverse events did not significantly differ between the placebo and the Bronchitol groups. The dropout rate overall was less than 10% indicating that treatment was well accepted.
The trial was conducted at 22 hospitals across Australia, New Zealand and the United Kingdom. Participants received either Bronchitol or placebo for 12 weeks, after which participants were provided with Bronchitol for a total of 12 months to determine the safety of long term treatment.
"The completion of this study and the achievement of its primary endpoints is a major advance towards our goal of having Bronchitol available for patients with bronchiectasis," said Pharmaxis Chief Executive Officer Dr Alan Robertson.
"This is the largest single study ever conducted in bronchiectasis and we have collected a large amount of data on a variety of secondary endpoints which are still being analyzed. With no products currently indicated for bronchiectasis we look forward to discussing our complete data set and next steps with the regulators."
Bronchiectasis is an incurable, degenerative and chronic lung condition. Pharmaxis expects Bronchitol to be the first targeted medication for this patient group in over 20 years -- addressing an important medical need.
Over 600,000 patients worldwide suffer from bronchiectasis including 110,000 in the United States and 20,000 in Australia. Total U.S. medical care expenditure is US$13,000 per bronchiectasis patient, double that of patients without the disorder; and an increased overall cost of US$630 million. Widespread availability of high resolution scanners is leading to increasing diagnosis and the understanding that bronchiectasis is more common than previously thought. Pharmaxis is developing Bronchitol as a daily treatment administered by inhalation to the patient's lungs.
Earlier this month, Pharmaxis requested a Special Protocol Assessment review with the U.S. Food and Drug Administration to commence a U.S. Phase III trial with Bronchitol.
Full study results will be submitted for presentation at an upcoming international scientific meeting.
The statements contained in this media release that are not purely historical are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential for Aridol and/or Bronchitol. All forward-looking statements included in this media release are based upon information available to us as of the date hereof, and we assume no obligation to update any such forward-looking statement as a result of new information, future events or otherwise. We can not guarantee that any product candidate will receive FDA or other regulatory approval or that we will seek any such approval. Factors that could cause or contribute to such differences include, but are not limited to, factors discussed in the "Risk Factors and Other Uncertainties" section of our Form 20-F lodged with the U.S. Securities and Exchange Commission.
For more information, please contact: Alan Robertson -- Chief Executive Officer Tel: +61-2-9454-7200 Email: [email protected] United States: Brandon Lewis Trout Group Tel: +1-646-378-2915 Email: [email protected] Brad Miles BMC Communications Group Tel: +1-212-477-9007 Email: [email protected] Australia: Virginia Nicholls Tel: +61-417-610-824 Email: [email protected]