Advertisement
The SPA process allows for FDA evaluation of a clinical trial protocolintended to form the primary basis of an efficacy claim in support of a NewDrug Application, and provides an agreement that the study design, includingtrial size, clinical endpoints and/or data analyses are acceptable to the FDA.
Advertisement
Pharmaxis previously agreed on the trial design with the Europeanregulatory agency (EMEA). This trial will therefore form the basis of amarketing application in both the U.S. and Europe.
The phase 3 trial has been designed in collaboration with internationallyrenowned experts in the field of bronchiectasis and will be a randomized,placebo controlled, double-blind investigation of Bronchitol twice daily inapproximately 350 adults with bronchiectasis. Participants will be treatedfor 52 weeks and the primary endpoints are reduction in frequency ofexacerbations and improvement in quality of life. Secondary endpoints includetime to first exacerbation and duration of exacerbation. Additional secondaryendpoints are antibiotic use, sputum volume, exercise tolerance and lungfunction measurements. The trial includes health economic analysis and willbe conducted in centers across Europe and the U.S.
This trial is the second Phase III study to be undertaken for Bronchitolin bronchiectasis and follows the completion of a successful shorter trialreported last year.
Pharmaxis CEO, Alan Robertson, said the company was pleased to haveconcluded its discussions with the FDA and the EMEA.
''We believe this phase 3 trial design will allow us to thoroughlydemonstrate the clinical benefits of Bronchitol in a patient population forwhich mucus build up and clearance is a daily problem. Our bronchiectasisprogram follows closely behind our work in cystic fibrosis where a phase 3clinical trial is expected to soon close recruitment.''
Pharmaxis is developing Bronchitol as a treatment to improve mucusclearance in the lungs of patients with cystic fibrosis, bronchiectasis andother acute and chronic pulmonary conditions. The U.S. FDA has grantedBronchitol fast track status and it is designated as an orphan drug in theU.S.
Forward-Looking Statements
The statements contained in this media release that are not purelyhistorical are forward-looking statements within the meaning of Section 21E ofthe Securities Exchange Act of 1934, as amended. Forward-looking statements inthis media release include statements regarding our expectations, beliefs,hopes, goals, intentions, initiatives or strategies, including statementsregarding the potential for Aridol and/or Bronchitol. All forward-lookingstatements included in this media release are based upon information availableto us as of the date hereof, and we assume no obligation to update any suchforward-looking statement as a result of new information, future events orotherwise. We cannot guarantee that any product candidate will receive FDA orother regulatory approval or that we will seek any such approval. Factors thatcould cause or contribute to such differences include, but are not limited to,factors discussed in the "Risk Factors and Other Uncertainties" section of ourForm 20-F lodged with the U.S. Securities and Exchange Commission.CONTACT: Alan Robertson Chief Executive Officer Tel: +61-2-9454-7200 Email: [email protected] Released through: United States: Brandon Lewis, Trout Group Tel: +1-646-378-2915 Email: [email protected] Australia: Virginia Nicholls Tel: +61-417-610-824 Email: [email protected]
