Roivant's Mosliciguat Hits Phase 2 Endpoints in PH-ILD, Phase 3 PHrontier Enrolling
核心洞察
Roivant Sciences (搜索) reported that mosliciguat met the primary endpoint of the 135-patient Phase 2 PHocus trial in pulmonary hypertension associated with interstitial lung disease (搜索).
At Week 16, mosliciguat produced a 56.3% placebo-adjusted reduction in pulmonary vascular resistance with a p-value below 0.0001.
The trial also met its six-minute walk distance secondary endpoint, with a 35.2-meter placebo-adjusted gain at Week 16 (p=0.0027), and NT-proBNP (搜索) fell 53.2% versus placebo.
Roivant Sciences (搜索) reported positive Phase 2 results for mosliciguat, its experimental treatment for pulmonary hypertension associated with interstitial lung disease (搜索) (PH-ILD), with the company now advancing the program into a Phase 3 study that is already enrolling patients.
The 135-patient PHocus study met its primary endpoint. At Week 16, mosliciguat produced a 56.3% placebo-adjusted reduction in pulmonary vascular resistance (PVR), with a p-value below 0.0001. According to Roivant, this represents the largest PVR reduction reported in a randomized controlled pulmonary hypertension trial.
Secondary and Exploratory Findings
The study also met its secondary endpoint for six-minute walk distance. Patients receiving mosliciguat walked 35.2 meters farther than those on placebo at Week 16, with a p-value of 0.0027. Roivant reported a 53.2% placebo-adjusted reduction in NT-proBNP (搜索), a biomarker used to assess cardiac strain, with a p-value of 0.0002.
Additional results came from a pre-specified exploratory analysis at Week 24. The placebo-adjusted difference in six-minute walk distance was 52.7 meters, and the placebo-adjusted reduction in NT-proBNP (搜索) was 75.9%. Roivant noted that the Week 24 findings were exploratory and were reported with nominal p-values.
Mosliciguat was well tolerated in the study. Cough was reported in 12.1% of patients receiving mosliciguat compared with 18.2% of those receiving placebo.
Disease Context and Development Path
PH-ILD develops when scarring in the lungs damages blood vessels, increasing pressure and forcing the heart to work harder. Roivant has initiated the Phase 3 PHrontier study, with enrollment underway. The PHrontier study will provide the next major set of clinical evidence for the program.
The readout gives Roivant another late-stage pipeline opportunity shortly after the company won U.S. approval for Lisraya (搜索), its treatment for adults with dermatomyositis (搜索).
Treatment Landscape
Mosliciguat would enter a PH-ILD treatment landscape that already includes other therapies. Reuters identified United Therapeutics' Tyvaso and Tyvaso DPI and Liquidia (搜索)'s Yutrepia (搜索) among available treatments, and some patients also receive PDE5 inhibitors off-label, including drugs such as Viagra and Cialis.
Mosliciguat remains an experimental treatment. The results reported so far come from a 135-patient mid-stage study, and the Week 24 findings were exploratory rather than primary or secondary endpoints.
Commercial Forecasts Remain Estimates
J.P. Morgan analyst Brian Cheng told Reuters that before the readout, the firm estimated risk-adjusted peak PH-ILD sales of $1.5 billion for mosliciguat. Cheng said the latest results suggest the drug's potential could be greater, citing management's comments about possible development in additional indications. That figure is an analyst forecast, not Roivant guidance or current revenue.