LIQ861 is a dry-powder, inhaled formulation of treprostinil that Liquidia developed to deliver deep into the lungs. U.S. regulators have already approved other formulations of treprostinil, which is a synthetic version of prostacyclin, a blood-vessel-widening substance that PAH patients lack.
Liquidia will recruit 100 patients for the trial at several U.S. locations. The first patient is expected to enroll in the next few weeks.
The trial’s main goals are to assess the therapy’s long-term safety and patients’ ability to tolerate it. Results are expected in 2019.
“We are committed to improving the daily experience of those living with PAH by advancing the clinical development of LIQ861,” Neal Fowler, the chief executive officer of Liquidia, said in a press release.
Liquidia used its proprietary PRINT technology to create LIQ681.
“LIQ861 shows the potential of our PRINT technology to optimize safety, efficacy and convenience of therapeutics,” Fowler said. “The initiation of our trial marks an important milestone for Liquidia, for those living with PAH and for the PAH community at large. We believe that we are well positioned to advance LIQ861 through Phase 3 clinical development.”
“While treatment options exist for PAH, there is still a strong need for innovation to make it easier for patients to self-administer some of the more complex therapies,” said Dr. Nicholas Hill, a professor at Tufts University School of Medicine.
“Leveraging Liquidia’s PRINT technology to enable deep-lung delivery of inhaled treprostinil, LIQ861 offers the convenience of a dry-powder inhaler, which greatly simplifies the delivery of inhaled treprostinil and I believe may likely improve the lives of such patients,” Hill added.
The U.S. Food and Drug Administration has already approved oral and mist-delivered formulations of treprostinil.
Liquidia has designed the Phase 3 trial to generate information that regulators need to approve LIQ861.
Last year a Phase 1 trial of the therapy in healthy volunteers generated promising results. A key finding was that participants tolerated all doses of the drug. Liquidia plans to report additional results from the trial at scientific conferences in 2018.