Rare Disease Report

First Patient Dosed in Phase 2 PAH Study

JULY 17, 2016
Rare Disease Report
Eiger Biopharmaceuticals has dosed its first patient in the Phase 2 LIBERTY Study for treatment of Pulmonary Arterial Hypertension (PAH). The Phase 2 trial is studying the effects of Eiger’s ubenimex in patients with PAH (WHO Group 1).
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.

LIBERTY Phase 2 Study

The study is a multi-center, randomized, double-blind, placebo-controlled Phase 2 study of dosing ubenimex in patients with pulmonary arterial hypertension. Fourty-five patients will be randomized in a 2:1 ratio to receive 150 mg ubenimex or matching placebo, administered orally 3 times a day for 24 weeks. Patients who complete treatment through Week 24 will be eligible to enroll in an open-label extension study to receive continued treatment.
To find out more on the trials or sign up for recruitment, click here.

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.
Symptoms include
  • dyspne
  • fatigue
  • syncope
  • edemas in the legs
  • cyanosis
  • heart palpitations
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.

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