Phase 2a Trial of Experimental Pulmonary Arterial Hypertension Drug Begins

According to a story from BioSpace, the biopharmaceutical company Altavant Sciences has recently announced that the first patient has been dosed in a phase 2a proof of concept trial. This trial is testing the company’s lead investigational drug candidate, rodatristat ethyl, as a treatment for pulmonary arterial hypertension, a rare lung disease. This trial will evaluate target engagement, biomarker changes, and the overall safety profile of the drug.

About Pulmonary Arterial Hypertension (PAH)

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.

About Rodatristat Ethyl

The goal of this study is to evaluate rodatristat ethyl’s mechanism of action that is explicitly designed to halt or even reverse a distinct characteristic of the illness called pulmonary vascular remodeling. The medication is classified as an inhibitor of tryptophan hydroxylase (TPH) in order to reduce the production of serotonin. Abnormal production of peripheral serotonin has been linked with the progression of pulmonary arterial hypertension in research studies. This mechanism has the potential to act on other diseases as well, such as sarcoidosis and idiopathic pulmonary fibrosis.

The study will eventually include an estimated total of 36 adult patients. Treatment (or placebo) will be administered two times a day for six weeks. Patients who are currently receiving other treatments, such as vasodilators, are also invited to participate in the study.


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