Roivant Reports Positive Results from PHocus Trial for Mosliciguat in PH-ILD

Roivant has announced positive results from its Phase 2 PHocus clinical trial evaluating mosliciguat for the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD). The study, which involved 135 patients across 87 sites in 20 countries, demonstrated a significant reduction in pulmonary vascular resistance (PVR) and improvements in other key biomarkers.

The primary endpoint of the PHocus trial was met, showing a 56.3% reduction in PVR at Week 16, the largest reported PVR reduction in any randomized controlled trial for PH. This was accompanied by a 35.2-meter improvement in six-minute walk distance (6MWD) and a 53.2% reduction in N-terminal pro-B-type natriuretic peptide (NT-proBNP), a biomarker of cardiac strain, both of which were statistically significant and clinically meaningful.

Pre-specified exploratory analyses at Week 24 showed further improvements, with the 6MWD increasing by 52.7 meters and NT-proBNP reducing by 75.9%, again demonstrating a consistent and strong benefit. Mosliciguat was well tolerated, with a lower incidence of cough compared to placebo, a common tolerability challenge with inhaled prostacyclins.

The results of the PHocus study have positioned mosliciguat as a potential first-in-class, once-daily, inhaled sGC activator with a differentiated mechanism of action. This new treatment approach is designed to deliver targeted pulmonary vasodilation with limited systemic side effects, addressing a significant unmet need in PH-ILD, a condition that can be life-threatening with a median survival of just 1.5 to 2 years despite best-available standard of care.

Roivant plans to initiate the Phase 3 PHrontier study, a randomized, double-blind, placebo-controlled global trial, to evaluate the safety and efficacy of mosliciguat in adult patients with PH-ILD. The company aims to rapidly bring mosliciguat to patients with this condition, which affects up to 200,000 individuals in the U.S. and Europe, and has limited or no approved treatment options.

Professor Marc Humbert, Director of the French National Reference Center for Pulmonary Hypertension, commented, "PH-ILD remains one of the most challenging forms of pulmonary hypertension to treat. The PVR reduction observed in PHocus is remarkable and among the largest reported in a randomized controlled PH trial to date. Together, these results represent a clinically meaningful advancement in this field."

Mayukh Sukhatme, President and Chief Investment Officer at Roivant, added, "Our thesis was that the ATMOS study actually understated the potential of mosliciguat, and when dosed chronically, it would do considerably more. These PHocus results proved that out as clearly as we could have hoped. This data set puts mosliciguat in a league of its own on PVR reduction, 6MWD improvement, NT-proBNP % reduction, cough rate, and ease of use."

Roivant's commitment to expedite mosliciguat's development and bring it to patients who need effective treatment options is evident in the quick transition from Phase 2 to Phase 3. The PHrontier study is expected to enroll approximately 375 patients worldwide, further validating the potential of mosliciguat as a novel treatment for PH-ILD.