Polyphor develops the elastase inhibitor POL6014 for rare lung diseases using PARI Pharma’s eFlow® Technology inhalation device

10:28 EST 9 Jan 2015 | Instinctif Partners

Allschwil, Switzerland, January 9, 2015. Polyphor Ltd, a privately held pharmaceutical company specialized in the discovery and development of macrocycle drugs, today announced the signing of a licensing agreement with PARI Pharma, Starnberg, Germany, to use the eFlow® Technology nebulizer for the administration of POL6014, a highly potent and selective inhibitor of neutrophil elastase.
POL6014 is a synthetic macrocycle drug based on Polyphor`s proprietary drug discovery platform. It is being developed for the treatment of rare lung diseases such as Alpha-1 Antitrypsin (AAT) Deficiency and Cystic Fibrosis (CF). These severe, inherited diseases frequently lead to life-threatening progressive lung deterioration.

POL6014 has been shown to be efficacious in several animal models of lung diseases and was well tolerated. In feasibility studies using the eFlow® nebulizer, POL6014 has shown excellent suitability for inhalation. The start of Phase I clinical studies is planned in 2015.
Commenting on the news, Polyphor CEO, Jean-Pierre Obrecht said:

“Researchers and patient organizations have strongly encouraged us to expedite the development of POL6014 in light of the urgent medical need for novel therapeutic treatment options. Gaining access to a well-established and proven inhalation technology is very important for the further development  of POL6014 in rare respiratory diseases such as Alpha-1 Antitrypsin Deficiency and Cystic Fibrosis.”

The human neutrophil elastase enzyme is present in the healthy lung where it is inhibited by natural protease inhibitors, in particular Alpha-1 Antitrypsin. In AAT deficient patients this natural inhibitor is produced in insufficient quantities due to a genetic defect, which results in high levels of uninhibited elastase. This often leads to severe and fatal lung deterioration. There are several thousand diagnosed patients, mainly in Europe and the US; however, the number of undiagnosed patients is considered to be much higher.

Progressive lung disease also determines the morbidity and mortality of CF patients and therapeutic strategies to halt or even reverse disease progression, improve quality of life and life expectancy for CF patients are keenly sought. In CF, chronic bacterial infections are considered an important factor in stimulating the inflammatory process. Chronic inflammation triggers the body to produce, among other enzymes, an excess of elastase which, if not controlled, leads to degradation of the lung. Inhibition of elastase by inhaled POL6014 could therefore be a promising therapeutic approach. There are around 70,000 diagnosed CF patients, most of them living in Europe and the Americas.

The eFlow® nebulizer developed by PARI is an innovative device for the treatment of respiratory diseases via aerosol inhalation. Throughout its development, particular care was taken to consider patients' needs for efficient, safe, and above all fast inhalation treatment. With the eFlow® device, medications can be delivered to the deep lung compartments quickly and efficiently. Consequently, the eFlow® nebulizer is already widely used in the CF patient community for several medications.

About Polyphor Ltd

Polyphor is a Swiss Pharma company focusing on the discovery and development of macrocycle drugs addressing high unmet medical needs. Macrocycles represent a new drug class complementary to the classical small molecules and large biopharmaceuticals. Polyphor leverages its unique and proprietary technologies to develop its own product portfolio, to pursue licensing and collaboration opportunities. The company’s lead drug candidates include POL6326, a CXCR4 antagonist, with ongoing and upcoming Phase II clinical trials for stem cell transplantation, tissue repair and combination treatments in oncology; POL7080, a highly specific antibiotic with a novel mode of action to treat Pseudomonas infections (Phase II) which was licensed to Roche in November 2013; and POL6014, an inhaled inhibitor of neutrophil elastase for the treatment of Alpha-1 Antitrypsin Deficiency, Cystic Fibrosis, and other lung diseases (Phase I planned to start in 2015).
Polyphor is a privately held company with about 100 employees, headquartered in Allschwil, near Basel. For additional information, please visit

About PARI Pharma GmbH and the eFlow® Electronic Nebulizer

The device uses eFlow® Technology to enable highly efficient aerosolization of medication via a vibrating, perforated membrane that includes thousands of laser-drilled holes. Compared with other nebulization technologies, eFlow® Technology produces aerosols with a very high density of active drug, a precisely defined droplet size and a high proportion of respirable droplets delivered in the shortest possible period of time. Combined with its quiet mode of operation, small size, light weight and battery use, eFlow® Technology reduces the burden of taking daily, inhaled treatments.

For additional information, please visit:

Michael Altorfer, Ph.D. Head Corporate Communications Polyphor Ltd t: +41 61 567 16 00 e: NEXT ARTICLE

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