Verona commences Phase IIa trial of RPL554 to treat CF

UK-based biopharmaceutical company Verona Pharma has commenced the enrolment and dosing of patients in a Phase IIa clinical trial of RPL554 for the treatment of cystic fibrosis (CF).

RPL554 is an inhaled phosphodiesterase 3 and 4 (PDE3 / PDE4) enzymes inhibitor being developed for treating chronic obstructive pulmonary disease (COPD) and CF using its anti-inflammatory and bronchodilatory properties.

The double-blind, placebo-controlled, single-dose trial will evaluate the pharmacokinetic, pharmacodynamics profile and tolerability of RPL554 in approximately ten subjects.

The trial will also assess the effect of the product on lung function.

Verona Pharma chief executive officer Jan-Anders Karlsson said: "Cystic fibrosis is the most common fatal inherited disease in the US and Europe.

"Two recently approved therapies are indicated only for a subset of CF patients, and there is a need for novel, effective anti-inflammatory medications to treat the underlying inflammation in cystic fibrosis.

"RPL554 has a differentiated mechanism of action and pre-clinical data in cystic fibrosis combined with our positive clinical data in COPD leads us to believe it has the potential to be an important new treatment for this debilitating condition."

The data from pre-clinical studies showed that RPL554 induces a protein called CF transmembrane conductance regulator, whose mutation leads to dysfunctional ion channels in epithelial cells associated with CF.

RPL554 is reported to possess the potential to minimise phlegm and airway obstruction, as well as inhibit inflammation.

The results from prior clinical trials in COPD patients indicated that RPL554 causes statistically significant improvements in lung function, either alone or in combination with bronchodilators.