LIQ861 FOR PULMONARY ARTERIAL HYPERTENSION (PAH)
Our lead product candidate, LIQ861 is an inhaled dry powder formulation of treprostinil designed using our PRINT technology to enhance deep-lung delivery using a convenient, palm-sized, disposable dry powder inhaler (DPI) for the treatment of pulmonary arterial hypertension (PAH). We believe LIQ861 can overcome the limitations of current inhaled therapies and has the potential to maximize the therapeutic benefits of treprostinil in treating PAH by safely delivering higher doses into the lungs.
PAH is a rare, chronic and progressive disease caused by the hardening and narrowing of the pulmonary arteries that can lead to right heart failure and eventually death. Prostacyclin is a vasoactive mediator essential to normal lung function that is deficient in patients with PAH. With PAH, the elevated pressure in the pulmonary arteries strains the right side of the heart as it pumps blood to the lungs. The extra stress causes the heart to enlarge and become less flexible, compromising its ability to push blood out of the heart through the lungs and into the rest of the body. PAH initially presents as exertional dyspnea, lethargy and fatigue and may be confused with other disease states with similar symptoms. PAH often goes undiagnosed or misdiagnosed until symptoms become severe, with the mean time from onset of symptoms to correct diagnosis being more than two years in the U.S. There is no cure for PAH; current treatments rely on symptom management.
Approximately 15-20% of patients with PAH have heritable forms of PAH. People with heritable PAH have either: (1) an autosomal dominant genetic condition associated with mutations in the BMPR2 gene or other recently identified genes now associated with heritable PAH (HPAH) or other forms of PAH or associated conditions such as pulmonary capillary hemangiomatosis or pulmonary veno-occlusive disease, or (2) are members of a family in which PAH is known to occur as primary disease. (Link)
After consultation with the FDA, we advanced from our Phase 1 trial into our current single, pivotal Phase 3 trial, known as INSPIRE, or Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil, evaluating LIQ861 for the treatment of PAH in the U.S. INSPIRE is an open-label trial enrolling over 100 patients with PAH across multiple U.S. sites. Primary endpoints are long-term safety and tolerability of LIQ861.
Visit https://clinicaltrials.gov/ct2/show/NCT03399604 for more information on INSPIRE.
We will seek approval of LIQ861 under the 505(b)(2) pathway, which will allow us to rely in part on the FDA’s previous findings of efficacy and safety of Tyvaso® (U.T.C.) and the active ingredient treprostinil.
LIQ861 particles are a precise, uniform size and trefoil pollen-like shape designed to have ideal aerodynamic properties for deep-lung delivery. For more information on our data for LIQ861, please visit Publications.