Verona secures cash for midphase cystic fibrosis trial

Verona Pharma (LON:VRP) has secured funding for a Phase IIa trial of its PDE3/PDE4 inhibitor RPL554 in cystic fibrosis patients. The funding, which comes four months after Verona raised £45 million ($55 million), was provided by the Cystic Fibrosis Trust through its venture and innovation award program.

London-based Verona plans to start enrolling patients in the study in the first half of next year. The aim is to deliver data on the pharmacokinetics and pharmacodynamics of the drug, while also confirming its safety and tolerability in cystic fibrosis patients. Verona expects to enroll 10 patients and give them three treatment regimens--two doses of RPL554 and a placebo--in a random sequence.

Verona’s primary focus is on developing RPL554 as a treatment for chronic obstructive pulmonary disease (COPD). But, bankrolled by nonprofit the Cystic Fibrosis Trust, it has also explored the use of the phosphodiesterase inhibitor in cystic fibrosis. Verona used an earlier award from the trust to run preclinical tests on RPL554 in cystic fibrosis. With data from the tests suggesting the drug stimulates CFTR, the collaborators have seen enough to justify moving into the clinic.


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The evidence of CFTR stimulation raises hopes RPL554 can clear phlegm from the airways of cystic fibrosis patients, while also easing breathing and improving symptoms of chronic inflammation. The Phase IIa isn’t focused on assessing this theory, but it will gather data of relevance. Verona will collect data on the levels of inflammatory mediators in the sputum of participants eight and 12 hours after receiving treatment.

If the trial supports the advancement of RPL554 in cystic fibrosis, it will open another R&D avenue for Verona at an important time for the company. The £45 million round saw Vivo Capital, OrbiMed, Edmond de Rothschild Investment Partners and other investors come on board. Fuelled by the cash, Verona plans to run a Phase IIb trial in 300 to 400 COPD patients. A Nasdaq IPO is being explored, too. 

Success in either cystic fibrosis or COPD would provide a coda to the career of the late Sir David Jack, who co-invented RPL554 after decades spent helping Glaxo grow into a significant drugmaker.

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