Open-label clinical trial of Yutrepia for PH-ILD enrolls 1st participant
FDA to make decision soon on Yutrepia, inhalation powder of treprostinil
The first participant has entered ASCENT, a clinical study into the safety and tolerability of Liquidia’s Yutrepia, an inhaled dry powder formulation of treprostinil that is under regulatory review for pulmonary hypertension associated with interstitial lung disease (PH-ILD).
The open-label study (NCT06129240) seeks to enroll an estimated 60 adults, ages 18 to 80, who have a diagnosis of PH-ILD, which refers to high blood pressure in the lungs that develops due to diseases that cause the lungs to become scarred. Study sites are not yet known.
FDA expected to make decision before end of January
The U.S. Food and Drug Administration (FDA) is expected to make a decision before the end of January about whether to extend Yutrepia’s tentative approval for pulmonary arterial hypertension (PAH) to PH-ILD.
If given the FDA’s green light, “YUTREPIA will provide practitioners and patients the opportunity to comfortably use a low-resistance dry-powder inhaler across a wide range of doses to a broad range of patients with PAH and PH-ILD with varying lung function and clinical severity,” Rajeev Saggar, MD, Liquidia’s chief medical officer, said in a press release.
Yutrepia contains treprostinil, a lab-made compound that mimics prostacyclin, which occurs naturally in the body. Prostacyclin widens and relaxes the blood vessels, thereby reducing blood pressure. Administered via a palm-sized device, Yutrepia is inhaled directly into the lungs. This is expected to improve blood flow and exercise capacity.
The FDA’s tentative approval of Yutrepia was based on data from a Phase 3 clinical study, called INSPIRE (NCT03399604), that showed the medication not only was safe and well tolerated, but also improved exercise capacity and quality of life in adults with PAH.
Liquidia sought to extend Yutrepia to include PH-ILD following confirmation from the FDA that no additional clinical studies would be needed to support a decision. If the extension is given, the decision would come into effect after the end of an exclusivity period granted to Tyvaso during which the medication is protected from market competition.
Tyvaso, an inhaled solution of treprostinil developed by United Therapeutics, was the first medication to be approved in the U.S. for PH-ILD. Treprostinil is also vailable from United in an oral formulation as Orenitram, in an infusion/injection as Remodulin, and in an inhaled dry powder formulation as Tyvaso DPI.
For this year, Liquidia is also planning a placebo-controlled Phase 3 clinical study testing L606, a sustained-release formulation of treprostinil that the company licensed in mid-2023 from Pharmosa Biopharm. The FDA agreed that a single pivotal study would support both PAH and PH-ILD indications.
Open-label Phase 3 trial of L606 continues recruitment at US sites
Meanwhile, Liquidia continues to enroll an estimated 60 adults with PAH or PH-ILD in its open-label Phase 3 clinical study (NCT04691154) of L606, administered twice daily with a short-duration nebulizer. Recruitment is ongoing at sites in the U.S.
To be eligible to enter the study, people with PAH must have never been treated with a prostacyclin or they must be transitioning from Tyvaso or Tyvaso DPI. Those with PH-ILD must be transitioning from Tyvaso or Tyvaso DPI. The study’s main goal is to assess L606’s safety and tolerability.
“We are … pleased by the increasing interest to enroll patients in the L606 open-label study,” Saggar said. “The real-time data on patient tolerability, dosing titration, and clinical response will greatly inform the pivotal efficacy trial and should provide additional confidence in the benefits of more consistent, sustained exposures over a 24-hour period.”
The study has treated some patients with L606 for longer than one year and at doses comparable to 25 to 27 breaths of Tyvaso, four times daily. The company expects the study to be fully enrolled this year and plans to share interim data at medical conferences later in the year.