Polyphor Ltd., today announced the development of an inhaled dosage form of its novel breakthrough antibiotic Murepavadin. In order to support and accelerate the development, Polyphor will leverage a European program dedicated to the development of inhaled antibiotics, iABC - a consortium of leading lung specialists in 18 hospitals and research institutions in eight European countries. These institutions will receive up to EUR 5 million funding for this project from the Innovative Medicines Initiative (IMI), a public-private partnership of EFPIA and the EU, while Polyphor will invest up to EUR 5 million.
Funding up to proof-of-concept in man will be provided for EUR 5 million by Polyphor and for another EUR 5 million by IMI via the funding of a consortium dedicated to the development of Inhaled Antibiotics in Bronchiectasis and Cystic Fibrosis (iABC).
Respiratory infections, especially those caused by drug-resistant bacteria, are the main cause of disease and death in people with cystic fibrosis and bronchiectasis. Thanks to inhaled antibiotics, patients now live longer than ever before and enjoy a better quality of life. However, additional treatment options are needed to reduce the bacterial load and fight resistant pathogens - and especially resistant Pseudomonas strains.
The iABC project is a Europe-wide program funded by the IMI to develop new, life-saving antibiotic treatments to manage chronic lung infection. The two industry partners are Polyphor and Novartis.
Murepavadin (POL7080) is the first representative of the Outer Membrane Protein Targeting Antibiotics (OMPTA), a class of antibiotics against Gram-negative bacteria with a novel mode of action discovered by Polyphor. It is a precision antibiotic specifically targeting Pseudomonas aeruginosa, one of the most difficult to treat pathogens, and is highly potent and active also against multi-drug resistant strains.
Pivotal studies of Murepavadin's intravenous formulation will be initiated in the first months of 2018 for the treatment of patients with nosocomial pneumonia (Ventilator-associated and hospital-acquired pneumonia; VAP/HAP) caused by Pseudomonas aeruginosa, including its resistant strains. The inhaled formulation of Murepavadin could extend the therapy to the treatment of chronic infections by Pseudomonas aeruginosa - affecting for example over 60% of the adult patients with cystic fibrosis and many of those with non-CF bronchiectasis and other rare lung diseases.
Professor Stuart Elborn, Professor of respiratory medicine at the Royal Brompton Hospital in London and Principal Investigator, said: "Just a few decades ago, most cystic fibrosis patients died in early childhood. Thanks to antibiotics, patients born today can expect to reach early middle age. However, this progress is threatened by the fact that current medicines do not fully eradicate the infections." He continued: "The iABC project represents an important contribution to efforts to counter this threat. Our work has the potential to deliver new inhaled antibiotics that will improve the quality of life and survival of patients with cystic fibrosis and bronchiectasis by reducing the number of lung infections, improving lung function, and overcoming antibacterial resistance."
Giacomo Di Nepi, Chief Executive Officer of Polyphor, said: "Cystic fibrosis and bronchiectasis patients are a vulnerable group. They are at particular risk of chronic infection. We are pleased and honored to be selected as one of two industry partners of the iABC consortium to develop an inhaled formulation of Murepavadin as a new treatment option. Our investigational antibiotic reaches high concentration in the lung and shows a high efficacy against Pseudomonas aeruginosa, the main cause of chronic lung infections in cystic fibrosis, affecting the majority of patients. In addition, by targeting only Pseudomonas aeruginosa, Murepavadin does not induce further resistance in other pathogens, which could infect these patients. Murepavadin may therefore provide a new important treatment option for patients with cystic fibrosis and bronchiectasis."
About Cystic Fibrosis and MurepavadinCystic fibrosis is a common inherited disease that affects around 36,000 people in the EU and 70,000 patients worldwide. People with cystic fibrosis face regular respiratory infections with 60% of adults suffering lung infections caused by Pseudomonas aeruginosa. Chronic bacterial pulmonary infections lead to an irreversible decline in lung structure and function. Today, respiratory failure is the cause of death for 95% of patients with cystic fibrosis.
Bronchiectasis refers to a group of diseases in which the airways become damaged and scarred. Patients affected with bronchiectasis experience similar symptoms to cystic fibrosis, including regular respiratory infections, often with Pseudomonas aeruginosa. Bronchiectasis is considered an emerging disease as more and more people in industrialized countries are diagnosed. As a consequence, no reliable indications for prevalence are available. Data from iABC's intended patient registry will increase the understanding of the disease and help to identify measures to assess the clinical effectiveness of potential medicines. Today, there are no therapies approved to treat bronchiectasis.
Murepavadin is a potent precision antibiotic with very low rate of resistance development
Murepavadin (POL7080) is the first representative of the Outer Membrane Protein Targeting Antibiotics (OMPTA) class, a class of antibiotics against Gram-negative bacteria with a novel mode of action discovered by Polyphor. Murepavadin is highly potent and unlikely to negatively impact the patient's native bacterial flora. No cross-resistance with current standard of care antibiotics has been observed.
Earlier this summer, Polyphor received positive guidance from the US Food and Drug Administration (FDA) and the European Medicines Agency for phase III development of Murepavadin for the treatment of patients with nosocomial pneumonia (ventilator-associated and hospital-acquired pneumonia; VAP/HAP) caused by infections through Pseudomonas aeruginosa, including its resistant strains. The Company is currently finalizing the study protocol in collaboration with its investigators and is initiating the pivotal development program. First patient enrolling is expected by Q1/2018.
In addition, the FDA has granted the QIDP (Qualified Infectious Disease Product) status to Murepavadin, which makes the compound eligible for priority review and statutory exclusivity for an additional five years upon approval. Such status is exclusively reserved for breakthrough medicines in life-threatening indications. Polyphor also develops other members of this new class of antibiotics which show excellent activity towards a broad range of Gram-negative pathogens, including isolates resistant to all other antibiotic classes.
IMI is a partnership between the EU and the European pharmaceutical industry
The Innovative Medicines Initiative (IMI) is the world's biggest public-private initiative in life sciences aiming to improve health by speeding up the development of, and patient access to, the next generation of medicines, particularly in areas of unmet medical or societal need. IMI is a joint undertaking between the European Union and the pharmaceutical industry association EFPIA (European Federation of Pharmaceutical Industries and Associations).
IMI supports collaborative research projects and builds networks of industrial and academic experts in order to boost pharmaceutical innovation in Europe. Through the IMI 2 program, IMI has a budget of EUR 3.3 billion for the period 2014 to 2024. Half of this comes from EU's research and innovation program, Horizon 2020. The other half is committed mostly by EFPIA companies.
In November 2011, the European Commission launched its first Action Plan against the rising threat from Antimicrobial Resistance, and called for unprecedented collaborative research and development efforts to bring new antibiotics to patients. The New Drugs for Bad Bugs (ND4BB) program was launched within the IMI in direct response to this call.
More information on IMI is available from http://www.imi.europa.eu/ and www.efpia.eu.
iABC unites Europe's leading lung specialists and researchers
The iABC (inhaled Antibiotics in Bronchiectasis and Cystic Fibrosis) project is part of New Drugs for Bad Bugs (ND4BB) program to develop new antibiotic treatments to manage chronic lung infections and will also establish the first European patient register for bronchiectasis, providing a platform to improve the quality of care for patients across Europe. It is a EUR 50 million pan-European project launched in 2015 funded by the European Commission through IMI and the EFPIA and involves leading lung specialists from 20 organizations in eight European countries including industry partners Polyphor and Novartis.
The iABC consortium involves researchers from Northern Ireland, Scotland, England, Spain, Germany, France, Italy, Belgium, the Netherlands and Switzerland. It is led by the School of Medicine, Dentistry and Biomedical Sciences, and the School of Pharmacy at Queen's University Belfast, Northern Ireland. Additional organizations involved are the Health and Social Care Trust, University Medical Center Utrecht, Belgium; Rijksuniversiteit Groningen, the Netherlands; Servicio Madrileño de Salud, Madrid, Spain; Universitair Ziekenhuis Antwerpen, Belgium; University of Dundee, Scotland; Institut National de la Santé et de la Recherche, Poitiers, France; Università degli Studi di Milano, Italy; Hospices Civils de Lyon, France; Medizinische Hochschule Hannover, Germany; Universiteit Antwerpen, Belgium; University of Edinburgh, Scotland; Royal Brompton and Harefield NHS Foundation Trust, London, England; Vall d'Hebron Institut de Recerca, Barcelona, Spain; Papworth Hospital NHS Foundation Trust, Cambridge, England; Erasmus Medical Center, Rotterdam, the Netherlands, and the EFPIA companies Polyphor Ltd., Allschwil, Switzerland, and Novartis Pharma AG, Basel, Switzerland.
For more information on the iABC Programme, visit http://www.iabcproject.com
Tel: +41 61 567 16 00