CF Patients in Europe Wanted for Phase 2 Trial of Investigational Therapy RPL554

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

Share this article:

Share article via email
HOPE-1 trial of SPX-101

Verona Pharma recently announced the company’s accomplishments in the last six months, including an update to the company’s clinical trial in cystic fibrosis (CF) and positive interim results for its investigational therapy RPL554.

RPL554 is an inhaled dual inhibitor of two enzymes, phosphodiesterase 3 and 4, and was previously shown to significantly improve lung function when compared to placebo treatment. RPL554 acts as an effective bronchodilator and has potent anti-inflammatory properties.

Verona is conducting a Phase 2a trial (NCT02919995) evaluating RPL554 in up to 10 patients with CF in the U.K. The study will evaluate the effects of escalating doses of the drug versus placebo in adult CF patients to determine the therapy’s tolerability, pharmacokinetics (its movement within the body from absorption to distribution, metabolism, and excretion), and pharmacodynamics (its effects and mechanism of action). The effects of RPL554 on lung function also will be assessed.

The study includes three groups: a high-dose group in which patients will receive a single inhaled dose of 6 mg of RPL554; a low-dose group with a single inhaled dose of 1.5 mg of RPL554; and a placebo control group. All three will be administered via a nebulizer.

The study is currently recruiting participants. Results from this trial are expected to be available in the first half of 2018.

In addition to CF, Verona Pharma also is developing RPL554 for the treatment of chronic obstructive pulmonary disease (COPD) and possibly asthma.

“It has been a transformative six months for Verona Pharma,” Jan-Anders Karlsson, CEO of Verona Pharma, said in a press release. “We now have the team and funding to deliver a comprehensive package of Phase 2b data for nebulized RPL554 as maintenance therapy for both COPD and CF, as well as for the treatment of acute exacerbations of COPD.”

Karlsson said the company looks forward “to updating the market on multiple clinical data points in this and coming years.”