IkT-001Pro for PAH

A Disease-modifying treatment for Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension, or PAH, is a rare disease of the pulmonary, or lung, microvasculature, characterized by hypertension that affects the functioning of the right-side of the heart, the side of the heart that sends and receives blood to and from the lungs. PAH mostly afflicts women between the ages of 30 and 60 and usually arises spontaneously. Less frequently, PAH can be caused genetic mutations or by drugs or toxins, or associated with connective tissue disease (CTD), congenital heart disease, HIV or schistosomiasis. PAH has just a 61% 5-year survival rate, indicating the poor prognosis of patients afflicted with this rare disease.

In the early 2010s, imatinib mesylate (as Gleevec®) was evaluated as a treatment for PAH because imatinib can inhibit the Abl enzyme c-Kit and PDGFRa/b, enzymes associated with the remodeling of the lung vasculature that leads to the disease. At that time, imatinib was shown to be highly effective therapy, but plagued with side effects that prevented imatinib’s approval in this indication. IkT-001Pro anticipated improvement in the side effect profile of imatinib coupled with the changes in the treatment paradigm for PAH standard of care suggests IkT-001Pro could be a successful treatment for this rare, but rapidly fatal disease.

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