United Therapeutics Receives FDA Clearance to Begin Clinical Trial of Inhaled PAH Treatment

United Therapeutics Corporation (NASDAQ:UTHR) has received clearance from the U.S. Food and Drug Administration (FDA) to begin clinical testing of Treprostinil-Iloprost SMI, an investigational inhaled treatment for pulmonary arterial hypertension (PAH).

The FDA clearance, granted under an Investigational New Drug (IND) application, allows the company to proceed with an initial clinical study evaluating the treatment’s safety, tolerability and pharmacokinetic properties.

Treprostinil-Iloprost SMI is being developed as an as-needed therapy for patients experiencing episodes of worsening PAH symptoms between doses of their existing maintenance medication.

Investigational Therapy Combines Two Prostacyclin Analogues

Treprostinil-Iloprost SMI combines two prostacyclin analogues, treprostinil and iloprost, delivered through a soft mist inhaler.

The treatment is designed to provide episodic symptom relief for PAH patients who experience acute symptom flare-ups despite receiving ongoing prostacyclin therapy.

Unlike maintenance treatments administered according to a regular dosing schedule, the investigational therapy is intended for use when symptoms worsen between scheduled doses.

United Therapeutics is developing the combination to address a treatment need among patients who continue to experience intermittent symptoms while receiving established PAH medications.

Phase 1 Trial to Evaluate Safety and Tolerability

Following FDA clearance, United Therapeutics plans to initiate a phase 1 clinical trial involving healthy volunteers.

The study will use an open-label, single ascending dose design to evaluate the safety, tolerability and pharmacokinetics of Treprostinil-Iloprost SMI.

The pharmacokinetic assessment will examine how the investigational treatment is absorbed, distributed and eliminated by the body.

The initial trial will provide information to support decisions about subsequent clinical development.

If the phase 1 study produces satisfactory results, United Therapeutics expects to begin a phase 2 trial involving patients with pulmonary arterial hypertension in 2027.

United Therapeutics Targets Unmet Needs in PAH Treatment

Pulmonary arterial hypertension is a condition involving elevated blood pressure in the arteries supplying the lungs, which can place additional strain on the heart.

According to United Therapeutics, approximately 500,000 people worldwide are affected by PAH, including around 50,000 individuals in the United States.

Ravi Patel, PharmD, Senior Vice President of Global Patient Safety and Strategic Clinical Development at United Therapeutics, said patients can experience worsening symptoms even while receiving maintenance medication.

He highlighted the absence of treatments specifically designed to address these episodes between regular doses.

The investigational inhaled therapy is intended to provide an additional treatment option for these circumstances, subject to successful clinical development and regulatory approval.

Regulatory Status and Development Outlook

Treprostinil-Iloprost SMI remains an investigational therapy and has not received regulatory approval for any indication in any jurisdiction.

The FDA’s IND clearance permits clinical testing but does not establish the treatment’s safety or effectiveness in patients with PAH.

The next development milestone will be the planned phase 1 study in healthy volunteers, with results expected to inform the company’s decision on further testing.

United Therapeutics has indicated that a phase 2 study could begin in 2027, depending on the outcome of the initial trial.

The programme adds to the company’s development activities in pulmonary arterial hypertension, with the objective of evaluating an inhaled treatment for intermittent symptom relief.

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