Safety Data Looks Good in Phase 3 Trial of Potential Pulmonary Arterial Hypertension Drug

According to a story from BioSpace, the biopharmaceutical company Liquidia Technologies, Inc., has recently released interim safety data for its open label Phase 3 clinical trial testing LIQ861. This data indicates that the experimental drug, which is a powdered formulation of treprostinil, has a favorable safety profile. LIQ861 is in development as a treatment for the rare disorder pulmonary arterial hypertension. 

About Pulmonary Arterial Hypertension

Pulmonary arterial hypertension is a condition in which the blood pressure in the arteries of the lungs is abnormally high. The cause of pulmonary arterial hypertension is often unknown in many cases. However, there are a variety of potential causes, such as certain heritable genetic mutations, exposure to certain toxins, and drug use (ex. methamphetamine). It can also appear as a symptom or complication in a number of other diseases, such as heart disease, connective tissue disease, and infection with HIV. The arteries in the lungs are often inflamed. Symptoms of this condition include rapid heartbeat, poor exercise tolerance, shortness of breath, fainting, leg swelling, fatigue, and chest pain. Treatment may include a number of medications and surgical operations, including lung transplant. A transplant can cure the condition, but it can cause many complications. Survival rate is often only about two or three years without treatment, but the latest drugs can prolong life by several years or more. Click here to learn more about pulmonary arterial hypertension.

Interim Safety Findings

This safety data was reported at a two-week timepoint as specifically requested by the US Food and Drug Administration (FDA) and will be included in the company’s New Drug Application. At this two week timepoint, the drug was quite well tolerated in patients. There was no indication of serious toxicity that could limit dosing and no serious drug related adverse events. In this time frame, patients were dosed at 125mcg.

Since that time, patients have received doses of up to 150mcg without any changes in safety profile. Side effects of LIQ861 appear to be mild and include coughing, headaches, and throat irritation. LIQ861 is intended to surpass the current limitations of many inhaled therapies and so far appears capable of delivering higher doses into the lungs more safely.

 


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