Drug for Cystic Fibrosis, Other Lung Diseases Reaches Successful Phase 1 End

Drug for Cystic Fibrosis, Other Lung Diseases Reaches Successful Phase 1 End

Polyphor, a Swiss pharmaceutical focused on macrocycle drug discovery and development, recently announced the successful conclusion of its Phase 1 clinical testing of POL6014 for cystic fibrosis (CF), Alpha-1 Antitrypsin Deficiency (AATD), and other lung diseases.

Macrocycle drugs represent a new class complementary to traditional small molecules and large biopharmaceuticals. The macrocycle candidate POL6014 is a highly selective, potent and reversible inhibitor of neutrophil elastase (hNE).

CF, a life-threatening disease that affects the lungs and digestive systems of about 70,000 people worldwide, is caused by a mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene, which leads to either no CFTR protein or abnormal CFTR protein. Dysfunction or absence of the protein results in the accumulation of thick mucus in the lungs, which progressively inhibits the patients’ ability to breathe.

The Cystic Fibrosis Foundation Therapeutics (CFFT) is collaborating with Polyphor to accelerate the clinical development of the inhaled elastase inhibitor POL6014 in CF.

The compound was found to be well tolerated in the randomized, double-blind, placebo-controlled, dose-escalation Phase 1 clinical trial across all treatment groups.  No serious adverse effects were reported.

The study was designed to assess the safety, tolerability and pharmacokinetics (movement of the drug in the body) of inhaled single doses of POL6014 in healthy volunteers. The measured pharmacokinetics parameters increased in accordance with the dose administered, using a customized Pari eFlow aerosol inhaler.

In a press release, Polyphor’s Chief Medical Officer Dr. Leon Hooftman, said the study confirms that the drug can be safely delivered to the human lung by inhalation.

“Following the successful completion of this study, we have now initiated two clinical Phase Ib trials investigating the safety, pharmacokinetics, and pharmacodynamics of POL6014 in individuals with cystic fibrosis and non-cystic fibrosis bronchiectasis,” Hooftman said.

Other Polyphor’s lead drug candidates include POL6326 (balixafortide) – a CXCR4 antagonist, developed for combination treatments in oncology and other indications; and POL7080 (murepavadin) – a highly specific antibiotic with a novel mode of action to treat pseudomonas infections.

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