A Disease-modifying Treatment for Pulmonary Arterial Hypertension
Pulmonary Arterial Hypertension, or PAH, is a rare disease of the pulmonary microvasculature characterized by high blood pressure in the arteries of the lungs. This increased pressure can damage the blood vessels and the right ventricle of the heart. PAH mostly afflicts women between the ages of 30 and 60 and usually arises spontaneously. PAH can also be caused by genetic mutations, connective tissue disease, congenital heart disease or drugs and toxins. PAH has just a 61% 5-year survival rate, indicating the poor prognosis of patients afflicted and treated with currently available medicines.
In the early 2010s, imatinib mesylate (as Gleevec®) was evaluated as a treatment for PAH because imatinib can inhibit receptor tyrosine kinases including PDGFRa/b and c-KIT associated with the abnormal remodeling of the lung vasculature that leads to the disease. In that clinical study, imatinib was shown to be a highly effective therapy, but was plagued with side effects that prevented its approval in this indication. IkT-001 was designed to improve the side effect profile of imatinib, coupled with changes in the treatment paradigm since the initial clinical study, suggests that IkT-001 could be a successful treatment for this rare, but rapidly fatal disease.