A Phase 3 clinical trial testing LIQ861 met its primary objective, showing that the investigational inhaled dry powder treprostinil formulation is safe and effective for the treatment of pulmonary arterial hypertension (PAH), according to clinical biopharmaceutical company Liquidia Technologies.
Liquidia’s LIQ861 is an alternative to the current inhaled treprostinil treatment Tyvaso, marketed by United Therapeutics. It lowers the blood pressure in pulmonary arteries by widening the arterial walls and reducing blood clotting.
LIQ861 is designed as an easy-to-use, palm-sized inhaler to effectively deliver the active substance treprostinil. The formulation is produced using the company’s PRINT (Particle Replication in Nonwetting Templates) technology, intended to safely deliver high doses of uniform treprostinil particles, improving deep-lung delivery and increasing its therapeutic benefits.
The open-label Phase 3 INSPIRE trial (NCT03399604) includes PAH patients transitioning from stable doses of inhaled treprostinil to LIQ861, or PAH patients receiving stable treatment with no more than two oral non-prostacyclin PAH therapies and whose treatment regimen is supplemented with LIQ861.
Results showed that all 109 participants enrolled in the trial tolerated doses of LIQ861 ranging between 25 and 150 micrograms, with no serious adverse events. Of the participants, 101 completed at least two months of treatment.
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Most of the reported adverse events were mild to moderate, including cough (reported in 33% of the patients), headache (18%), and throat irritation (14%), among others. The adverse events were often reported during the first two weeks of treatment.
“The top-line analysis of LIQ861 from the INSPIRE study is highly encouraging for physicians and patients. LIQ861 was safely titrated to therapeutic levels across a wide range of inhaled doses and was very well tolerated,” Nicholas Hill, MD, INSPIRE’s principal investigator, said in a press release.
“This means that we are moving closer to having an inhaled therapy available for PAH that is much more convenient than previous ones,” added Hill, who is chief of the pulmonary, critical care and sleep division and a professor of medicine at Tufts University School of Medicine.
According to Liquidia, enrollment is complete in a separate sub-study comparing the bioavailability (drug absorption in the body) and pharmacokinetics (drug route inside the body) of LIQ861 with Tyvaso. The company expects results from the study in the second quarter of this year.
Based on the results obtained so far, Liquidia plans to submit a new drug application to the U.S. Food and Drug Administration by the end of the year.
“We are extremely grateful to the patients participating in the clinical trial and for the effort and speed with which our investigators completed enrollment. We are preparing the new drug application submission, while collecting additional longitudinal data on the benefits from LIQ861,” said Neal Fowler, CEO of Liquidia.