AstraZeneca has licensed an experimental rare disease therapy to UK-based Mereo BioPharma, as it continues to shed products outside of its area of strategic focus.

Under the deal, Mereo is making an upfront cash payment of $3 million and handing over 490,798 of its shares in return for access to AZD9668, which is being developed as a treatment for the alpha-1 antitrypsin deficiency (AATD).

AATD is a genetic disorder affecting around 100,000 patients in the US and 120,000 patients in Europe. The deficiency can cause severe debilitating conditions such as chronic liver disease but, most notably, pulmonary emphysema.

Pulmonary emphysema results in irreversible destruction of the tissues supporting the function of the lungs and causing severe shortness of breath and wheeze. Patients have both a significantly reduced quality of life and a reduced life expectancy.

Current standard of care for the condition currently varies from country to country. Protein replacement therapy, involving weekly infusions of plasma-derived alpha 1 antitrypsin is available but only reimbursed in the US and some European countries. By suppressing neutrophil elastase through a more easily administered oral treatment, Mereo said it believes AZD9668 has “significant differentiation from the current protein replacement therapy”.

The group plans to carry out a Phase II study in AATD in 2018 to evaluate two doses of AZD9668 in around 150 patients with PiZZ and NULL genetic mutations, which are seen in the more severely affected patients who have very low (PiZZ) or zero (NULL) alpha-1 antitrypsin levels.

“AstraZeneca has generated a substantial clinical data package on AZD9668 which includes extensive Phase II studies in several respiratory conditions that will inform the initial Phase II clinical study we are planning for AATD,” said Denise Scots-Knight, Mereo’s chief executive.

“We believe that the neutrophil elastase inhibitor AZD9668 could provide a new innovative approach for the treatment of AATD.”