Phase 2 clinical trial of sotatercept for Group 2 PH succeeds

Pulmonary hypertension (PH) due to heart failure -- Group 2 PH -- has, until now, been treated by treating the underlying heart failure. However, a recent Phase 2 clinical trial, called CADENCE, tried treating PH due to heart failure with preserved ejection fraction (HFpEF) with sotatercept. Phase 2 trials are small trials which look at the safety of a drug and at whether it seems to work.

Full data have not been published yet, but CADENCE successfully met its primary endpoint -- participants who took sotatercept showed a significant reduction in pulmonary vascular resistance after 24 weeks of treatment, compared to participants who received a placebo. Lower pulmonary vascular resistance means the heart needs to use less energy to pump blood through the lungs.

Because this Phase 2 trial was successful, a Phase 3 trial is planned. Phase 3 trials are larger trials which look at whether or not a drug treatment is better than the current standard treatment and whether the drug is reasonably safe. They are the last clinical trials before a manufacturer applies for drug approval.

While much work remains, the CADENCE results offer a welcome sign that new options for Group 2 PH may finally be within reach.