Pharmaceutical research company Pharmaxis (ASX: PXS) today announced its recently completed international Phase 3 trial of Bronchitol® (mannitol) in adults with cystic fibrosis (CF) met its primary endpoint.
- The study recruited adult CF patients with all grades of disease that were already on the best standard of care.
- The study demonstrated the superiority of Bronchitol versus the comparator on the primary endpoint (FEV1 change from baseline over 26 week treatment period), with an effect of 54 ml (p=0.020), corresponding to a 2.2% relative change (p=0.025).
- The improvement in lung function was less than that seen in the adult CF population in previously reported phase 3 studies.
- No statistically significant differences between treatment groups in secondary endpoints were recorded, although a trend was observed in favour of Bronchitol for another lung function parameter (FVC).
- Bronchitol had a good safety profile with similar rates of adverse events seen compared to control.
- FDA resubmission is expected in 2018.
Pharmaceutical research company Pharmaxis today announced it has appointed Dr Kathleen Metters to the Board of Directors. Dr Metters has more than 25 years of experience in the discovery and development of novel therapies for the treatment of serious diseases.
Dr Metters spent 9 years as a senior executive with Merck & Co. (Merck Sharp & Dohme (MSD) outside the United States and Canada) which included a 4 year period as senior vice president and head of Worldwide Basic Research. That was followed by an appointment to design and establish External Discovery and Preclinical Sciences, created to expand Merck’s scientific network to the greater research community in academia, biotechnology, and government; building partnerships in life sciences, medicine, engineering, and information technology.
After leaving Merck in 2011 Dr Metters was appointed President and Chief Executive Officer for Lycera Corp., a biopharmaceutical company pioneering innovative approaches to novel oral medicines for treatment of autoimmune diseases and cancer. Under her leadership, Lycera developed a robust pipeline of proprietary and partnered immune modulator programs which formed the basis, in June 2015, of an exclusive global collaboration with Celgene Corporation.
Dr Metters will serve as a Non Executive Director on the Pharmaxis Board – joining current members Malcolm McComas, Simon Buckingham, Will Delaat and Gary Phillips.Read full media release - pdf
Pharmaxis Drug Discovery Set to Generate Further Euro 10m Milestone in 2017 as Boehringer Confirms Second Clinical Study
Pharmaxis today announced positive updated information on Boehringer Ingelheim’s clinical development plans for the Pharmaxis drug discovery asset PXS-4728A. In addition to a phase 2 trial in NASH that is scheduled to start mid-year, Boehringer has confirmed that a phase 2 study for a second disease indication will commence this year. Pharmaxis is due to receive milestone payments of €18m for the start of a phase 2 trial in NASH and €10m for the second indication.
Boehringer acquired PXS-4728A from Pharmaxis in May 2015 with initial clinical development focused on finding a treatment for NASH (Non-alcoholic Steatohepatitis), a debilitating liver disease.
Pharmaxis CEO Mr Gary Phillips said, “PXS-4728A is an anti-inflammatory drug with excellent preclinical data in several disease models. We are delighted to see Boehringer looking to exploit that potential and commence clinical development in a fresh indication in the second half of this year. The structure of the deal with Boehringer anticipated its potential in more than one disease and the €10m we expect for the second indication would bring total expected milestones received for starting phase 2 trials in two diseases to approximately A$42m in this calendar year. This is an important signal about Boehringer’s confidence in the potential of PXS-4728A to help patients.”
Mr Phillips added, “This significant injection of cash into the Pharmaxis business will allow us to strengthen our drug development pipeline in fibrosis and inflammation and add further scientific expertise. We aim to continue to build a company with the capability to translate and commercialise early stage research into assets with world class data sets that are highly valued by large Pharma companies seeking partnerships.”Read full media release - pdf
Pharmaxis today announced it has entered into an exclusive distribution and supply agreement with global pharmaceutical company Chiesi Farmaceutici SpA (Chiesi) for the commercialisation of Bronchitol® (mannitol) for cystic fibrosis in adults aged 18 years and above in Chiesi’s home market of Italy.
Under the terms of the agreement Chiesi will take over responsibility for the marketing, sales and distribution of Bronchitol with immediate effect. Italy has approximately 5,000 cystic fibrosis patients and is one of the top 5 EU markets by value. This new territory has been added as an extension to the agreement signed with Chiesi in 2015 for distribution rights in Germany, Ireland and the United Kingdom.Read full media release - pdf