Rare Disease Report

Phase 2 Open-Label Extension PAH Study Begins Patient Dosing

JANUARY 02, 2017
Andrew Black
In July, Eiger Pharmaceuticals began their Phase 2 Pulmonary Arterial Hypertension (PAH) treatment study. Since then, the first patient has completed the 24 weeks dosing of the double-blind Phase 2 study and has now received treatment in the open-label extension. 
The open-label extension is studying the effects of Eiger’s ubenimex for the treatment of PAH.
The study is a multi-center, randomized, double-blind, placebo-controlled Phase 2 study of dosing ubenimex in patients with PAH. 45 patients were randomized in a 2:1 ratio to receive 150 mg ubenimex or matching placebo, administered orally 3 times a day for 24 weeks.
Treatment is being continued.

About ubenimix 

Ubenimex is an oral, small-molecule inhibitor of leukotriene A4 hydrolase, which regulates the production of leukotriene B4 (LTB4), an inflammatory mediator implicated in PAH. LTB4 is produced from leukocytes in response to inflammatory mediators and is able to induce the adhesion and activation of leukocytes on the endothelium, allowing them to bind to and cross it into the tissue.

About Pulmonary Arterial Hypertension 

Pulmonary arterial hypertension (PAH) is a rare disease characterized by increased pulmonary-artery pressure in the absence of common causes of pulmonary hypertension, such as chronic heart, lung, or thromboembolic disease. According to the American Lung Association, approximately 50% of people with PAH die within 5 years of diagnosis. If not treated, average survival is only about 3 years.
There are several orphan drugs approved for treatment of PAH, including:

Uptravi (selexipag)
 Approved in 2015 for treatment of adults with PAH (WHO Group 1), to delay disease progression and reduce the risk of hospitalization.
Opsumit (macitentan) Approved in 2013 for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1) to delay disease progression. Disease progression included: death, initiation of intravenous (IV) or subcutaneous prostanoids, or clinical worsening of PAH (decreased 6-minute walk distance, worsened PAH symptoms and need for additional PAH treatment). Opsumit also reduced hospitalization for PAH.

Adempas (riociguat)  Approved in 2013 for treatment of adults with PAH (WHO Group 1), to improve exercise capacity, WHO functional class and to delay clinical worsening.

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