Insmed –
Insmed Inc., a global biopharmaceutical company, Tuesday announced positive topline results from its Phase 2b study evaluating the efficacy and safety of treprostinil palmitil inhalation powder (TPIP), administered once daily in patients with pulmonary arterial hypertension (PAH).
The results of the double-blind, placebo-controlled study demonstrated the durability of TPIP’s therapeutic effect as a once-daily therapy based on efficacy being evaluated about 24 hours after therapy was administered.
Based on these results, Insmed will engage with the Food and Drug Administration regarding the Phase 3 trial design for pulmonary arterial hypertension. Insmed plans to initiate a Phase 3 trial in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) before the end of 2025 and a Phase 3 trial in patients with PAH in early 2026.
Pulmonary arterial hypertension is a progressive, rare disease in which the blood vessels in the lungs narrow or become obstructed, leading to high blood pressure in the pulmonary arteries. The most common symptoms include shortness of breath, chest pain, dizziness or fainting, fatigue, and weakness. It is estimated that about 35,000 patients in the U.S. have been diagnosed with the disease. Untreated, PAH can be debilitating and often fatal.
“The statistically significant and clinically meaningful results shown with TPIP in pulmonary arterial hypertension mark a potential breakthrough for patients and the future of prostanoid therapy,” said Gene Sullivan, chief product strategy officer of Insmed.
The study was conducted at 44 sites globally; 102 patients were randomized to receive either TPIP or placebo for 16 weeks. Once-daily TPIP therapy was well tolerated in the study.
“Today’s outstanding results for TPIP represent more than a decade of hard work and the application of innovative chemistry intended to deliver a safe and effective, once-daily inhaled prostanoid therapy for patients with PAH, a devastating, progressive disease,” said Martina Flammer, chief medical officer of Insmed. “Having met the primary endpoint with high statistical significance, as well as seeing positive results for all secondary efficacy endpoints, we are excited about TPIP’s potential to become the prostanoid of choice.”
