Roivant Sciences (ROIV) Study result summary

Disease background and unmet need

• PH-ILD patients face high comorbidity rates and a 3-year mortality rate of ~60%.

• Median survival is only 1.5–2 years despite best available standard of care.

• No non-treprostinil therapies are approved, and current options require frequent dosing and have significant side effects.

• The PH-ILD treatment domain remains in its infancy, with significant unmet needs for efficacy, tolerability, and convenience.

Study background and design

• PHocus was a large, double-blind, multicenter global trial in PH-ILD, enrolling 135 patients across nearly 90 sites in 20 countries, randomized 2:1 to mosliciguat or placebo.

• Key inclusion: PH WHO Group 3 with ILD, PVR ≥4 WU, and defined fibrosis/emphysema by CT.

• The study population was severely ill, with significant comorbidities and high mortality risk, representative of the broader PH-ILD community.

• Baseline characteristics were well balanced across study arms, with most patients having moderate-to-severe disease.

• The primary endpoint was change in PVR at week 16; key secondaries included six-minute walk distance and NT-proBNP.

Mechanism and administration

• Mosliciguat is a once-daily inhaled sGC activator, targeting impaired NO-cGMP signaling in PH-ILD.

• Optimized for distal lung delivery, potentially reducing fibrosis and inflammation.