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Polyphor extends clinical development portfolio by advancing its inhaled elastase inhibitor POL6014 for lung diseases to Phase I

Allschwil, Switzerland, September 1, 2015. Polyphor Ltd, a privately held pharmaceutical company specialized in the discovery and development of macrocycle drugs, today announced the initiation of recruitment in a first in man clinical Phase I study with inhaled POL6014, Polyphor's proprietary macrocycle drug candidate. POL6014 is a highly selective, potent and reversible inhibitor of human neutrophil elastase (hNE), which is implicated in a number of severe respiratory diseases that frequently lead to life-threatening progressive lung deterioration as, for instance, in the orphan lung diseases Alpha-1 Antitrypsin (AAT) Deficiency and Cystic Fibrosis (CF).

Inhibition of excess hNE may stop or slow lung destruction and the progressive deterioration of lung function, characteristic of many obstructive lung diseases, resulting from the proteolytic (breakdown of proteins) activity of hNE in the lung. In addition, it may also help improve lung function, exacerbation profiles and overall quality of life of patients by reducing the pro-inflammatory effect of hNE in the lung.

The randomized, double-blind, placebo-controlled dose-escalation Phase I study is designed to investigate safety, tolerability and pharmacokinetics of inhaled single doses of POL6014 in healthy volunteers. The study aims to establish a safe maximum tolerated dose for future clinical studies.

"The entry of POL6014 into clinical development is an important milestone for Polyphor. Our clinical stage pipeline now includes three proprietary macrocycle drug candidates in our focus areas of hospital care and rare diseases," said Michael Altorfer, Ph.D., CEO of Polyphor. "POL6014 exemplifies the significant potential of Polyphor's unique macrocycle platform to provide a sustainable flow of drug candidates for challenging target product profiles, which are difficult to address with other drug classes. POL6014 has been optimized for local delivery to the lung, which is the preferred route of administration for many respiratory diseases."

Potential use of POL6014 in Alpha-1 Antitrypsin (AAT) deficient patients:
The hNE enzyme is present in the healthy lung where it is inhibited by natural protease inhibitors, in particular AAT. 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 or even 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.

Potential use of POL6014 in Cystic Fibrosis (CF) patients:
Progressive lung disease determines the morbidity and mortality of CF patients. Therapeutic strategies to halt or even reverse the progression of this orphan disease, improve quality of life and life expectancy for CF patients are keenly sought. Chronic bacterial infections are considered an important factor in stimulating inflammatory processes in CF. The resulting chronic inflammation triggers the body to produce, among other enzymes, an excess of elastase, which, if not controlled, leads to lung deterioration. Inhibition of elastase by inhaled POL6014 could therefore be a promising therapeutic approach. There are approximately 70,000 diagnosed CF patients, most of them living in Europe, North and South America.

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 traditional small molecules and large biopharmaceuticals. Polyphor leverages its unique and proprietary technology platforms to develop its own product portfolio and to pursue licensing and collaboration opportunities with industry partners. The company's lead drug candidates include POL6326, Balixafortide, (Phase II), a CXCR4 antagonist, for stem cell transplantation, tissue repair and combination treatments in oncology; POL7080 (Phase II), a highly specific antibiotic with a novel mode-of-action to treat Pseudomonas infections; and POL6014 (Phase I), an inhaled inhibitor of neutrophil elastase for the treatment of Alpha-1 Antitrypsin Deficiency, Cystic Fibrosis, and other lung diseases.

Polyphor is a privately held company with about 110 employees, headquartered in Allschwil, near Basel. For additional information, please visit www.polyphor.com