The FDA has cleared treprostinil soft mist inhaler for a phase 2 clinical trial to treat pulmonary hypertension associated with COPD, a condition with no currently approved treatments. If successful, this portable inhaler therapy could become the first approved treatment option for PH-COPD patients.
- The phase 2 SUMMIT study will evaluate treprostinil soft mist inhaler in approximately 40 patients with PH-COPD over 16 weeks
- No approved treatments currently exist for pulmonary hypertension associated with COPD, representing a significant unmet medical need
- The soft mist inhaler delivers preserved treprostinil solution directly to the pulmonary vasculature four times daily with customizable dose strengths
- If phase 2 succeeds, phase 3 trials are planned for 2028, potentially leading to the first FDA-approved PH-COPD therapy
- United Therapeutics also plans to develop treprostinil SMI for pulmonary arterial hypertension, interstitial lung disease, and progressive pulmonary fibrosis
A potential new delivery method for treprostinil has received clearance from the U.S. Food and Drug Administration (FDA) to begin a phase 2 clinical trial that evaluates efficacy in treating pulmonary hypertension associated with COPD (PH-COPD). If successful, the treatment has the potential to become the first approved treatment for PH-COPD.
United Therapeutics, the manufacturer of the treprostinil soft mist inhaler (SMI), said in a press release that the phase 2, 16-week, double-blind SUMMIT study will evaluate the safety and tolerability of treprostinil SMI in approximately 40 patients with PH-COPD.
In addition, the study will also evaluate the efficacy of treprostinil SMI on exercise capacity and N-terminal pro-B-type natriuretic peptide (NT-proBNP), a critical biomarker for heart failure. If the phase 2 trial is successful, the company said it plans to launch a phase 3 study in 2028.
Treprostinil is currently used to treat pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease, though not in SMI form. Leigh Peterson, PhD, head of pharmaceutical and xenograft development at United Therapeutics, said there are no approved treatments for PH-COPD.
“We believe that inhaled treprostinil, delivered by a soft mist inhaler, may benefit the PH-COPD patient population by directly targeting the pulmonary vasculature,” she said. “Our hope is that treprostinil SMI will offer a meaningful benefit and reduce disease burden for PH-COPD patients, who deserve a treatment option that addresses their significant and unmet needs.”
Treprostinil SMI includes a portable, hand-held soft mist inhaler designed to deliver a preserved formulation of inhaled treprostinil solution in a single breath. It will be available for the clinical study in multiple dose strengths to support individualized titration and long-term maintenance dosing administered four times daily.
United Therapeutics said it also plans to develop treprostinil SMI for pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease, idiopathic pulmonary fibrosis and progressive pulmonary fibrosis.
