The FDA Clears United Therapeutics' Investigational New Drug Application To Proceed With SUMMIT Trial Of Treprostinil Soft Mist Inhaler In Pulmonary Hypertension Associated With Chronic Obstructive Pulmonary Disease

United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that the U.S. Food and Drug Administration (FDA) has granted clearance under the company’s investigational new drug application to proceed with the SUMMIT clinical trial of treprostinil SMI in PH-COPD.

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. The study’s secondary endpoints will 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. Eligible patients in the SUMMIT study will be able to enroll in an open-label extension study to evaluate the long-term safety, tolerability, and efficacy of treprostinil SMI.

Assuming a successful outcome in the SUMMIT study, United Therapeutics anticipates commencing a subsequent phase 3 registration study for treprostinil SMI in PH-COPD in 2028.

"PH-COPD is a serious, life-threatening condition with no approved treatments. We believe that inhaled treprostinil, delivered via a soft mist inhaler, may benefit the carefully defined SUMMIT PH-COPD patient population by directly targeting the pulmonary vasculature. Our hope is that treprostinil SMI will offer meaningful benefit and reduce disease burden for PH-COPD patients who deserve a treatment option that addresses their significant unmet needs," said Leigh Peterson, Ph.D., head of pharmaceutical and xenograft development at United Therapeutics."

"I believe this development will lead to an exponential inflection point for United Therapeutics," said Martine Rothblatt, Ph.D., its Chairperson and Chief Executive Officer. "We estimate PH-COPD could impact five to ten times the number of patients than the conditions all of our current therapies are approved to treat today. Upon successful completion of the SUMMIT program and approval by the FDA, we expect United Therapeutics will become a several times larger company than it is today."

Treprostinil SMI includes a portable, handheld soft mist inhaler designed to deliver a preserved formulation of inhaled treprostinil solution in a single breath. The product will be available for the clinical study in multiple dose strengths to support individualized titration and long-term maintenance dosing, administered four times daily. Each single-load cartridge is designed to support up to two weeks of use, and the product can be stored at room temperature, offering patients a convenient, easy-to-carry delivery option.

United Therapeutics also plans to develop treprostinil SMI for pulmonary arterial hypertension, pulmonary hypertension associated with interstitial lung disease, idiopathic pulmonary fibrosis, and progressive pulmonary fibrosis.

Treprostinil SMI is an investigational-stage product, and is not yet approved in any jurisdiction for any indication.