Inhaled drug shows promise for rare lung disease combination
A therapeutic principle rooted in decades of pulmonary vascular research is entering a new stage of clinical development. The investigational inhaled drug mosliciguat, a soluble guanylate cyclase (sGC) activator, has produced marked and consistent effects in a randomized phase 2 study involving patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD), one of the…
A novel inhaled drug called mosliciguat is showing promise in treating pulmonary hypertension linked to interstitial lung disease, according to a recent phase 2 clinical trial. This sGC activator, which activates a specific pathway in the lungs, has demonstrated consistent and significant effects in patients with PH-ILD. Unlike other sGC stimulators, mosliciguat targets dysfunctional forms of the enzyme, making it effective in conditions associated with oxidative stress.
The drug was delivered via a dry-powder inhaler, allowing for targeted treatment with minimal systemic effects. In the phase 1b ATMOS study, patients experienced reduced pulmonary vascular resistance and improved exercise capacity after a single dose. The larger PHocus phase 2 trial, involving 135 patients across 20 countries, met its primary endpoint, showing a 56.3% reduction in pulmonary vascular resistance.
The treatment effect was seen across various subgroups, and the drug also improved right-heart and pulmonary vascular function. The development of mosliciguat builds upon decades of research focusing on the nitric oxide-sGC-cGMP pathway, ultimately leading to the approval of riociguat for pulmonary arterial hypertension. The success of this trial highlights the importance of long-term translational research and the value of regional, national, and international collaboration in addressing rare diseases with unmet medical needs.
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