United Therapeutics 获 FDA IND 许可，启动 Treprostinil-Iloprost SMI 治疗 PAH 的 Phase I 试验

The study will assess the pharmacokinetics, safety, and tolerability of Treprostinil-Iloprost SMI in healthy participants.

United Therapeutics has received clearance from the US Food and Drug Administration (FDA) under an investigational new drug application (IND) to initiate a Phase I trial assessing Treprostinil-Iloprost SMI as a potential treatment for pulmonary arterial hypertension (PAH).

The single-ascending dose, open-label study will assess the pharmacokinetics, safety, and tolerability of the therapy in healthy participants.

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If this initial study achieves positive results, the company expects to begin a Phase II trial in patients diagnosed with PAH in 2027.

Treprostinil-Iloprost SMI combines two prostacyclin analogues, iloprost and treprostinil.

It is designed to offer episodic symptom relief on an as-needed basis for individuals experiencing acute flare-ups between scheduled maintenance doses of prostacyclin therapy.

The candidate remains at an investigational stage and has not been approved for any indication in any jurisdiction.

United Therapeutics global patient safety and strategic clinical development senior vice-president Ravi Patel: “Many people living with PAH experience moments when their symptoms flare despite their maintenance medication, and today they have no therapy designed specifically for those moments.

“Treprostinil-Iloprost SMI is intended to explore whether an as-needed, combination prostacyclin therapy could help fill that gap. FDA clearance to begin clinical testing is an important first step, and we look forward to advancing this programme.”

PAH is a life-threatening vascular condition characterised by elevated pulmonary arterial pressure resulting from increased resistance in small precapillary vessels.

The disease causes platelet aggregation, structural alterations in vessel walls, and modified smooth muscle cell proliferation, which strains the right side of the heart and can culminate in heart failure and death.

In August, United Therapeutics completed enrolment for its TETON-PPF study, assessing the safety and efficacy of the Tyvaso (treprostinil) inhalation solution (nebulised Tyvaso) to treat progressive pulmonary fibrosis.