The event, to take place at the Lotte New York Palace Hotel, includes presentations by Stanford University’s Dr. Mark Nicolls and Dr. Roham Zamanian. Both will focus on the role of autoimmunity and inflammation in PAH, the potential of disease modifiers and the development of novel PAH treatments. After lunch, both men will be available to answer questions.
Eiger will also provide an update on patient enrollment in its Phase 2 LIBERTY study (NCT02664558), which is currently evaluating the efficacy, safety and tolerability of ubenimex administered orally for 24 weeks, in patients with PAH (WHO Group 1).
Patients who complete the trial may be eligible to enroll an extension study and get an additional 24 weeks of treatment, which will let researchers further investigate the drug’s efficacy and safety. Endpoints of the study include changes in pulmonary vascular resistance, improvements in exercise capacity (measured by the 6-minute walk distance test) and changes in the Borg dyspnea score (a measure of shortness of breath).
Ubenimex is a well-characterized, oral small molecule inhibitor of leukotriene A4 hydrolase (LTA4H), a protein that contributes to the formation of the pro-inflammatory mediator leukotriene B4 (LTB4). LTB4 levels are increased in PAH patients, as well as in animal models of the disease.
“The LIBERTY study represents a clinical translational effort with potential for disease modification in PAH,” Zamanian said in a press release. “While currently approved vasoactive agents have utility in the clinical management of the symptoms of PAH, they do not address the underlying inflammation which is an important signature of this cardiovascular disease. We have arrived at a moment of shift of therapeutic paradigm, where we may have a chance to realize a potentially disease modifying approach.”
Added Nocolls: “Inflammation is now recognized as an important component of PAH which is not addressed by currently available therapies. Our recently published preclinical studies suggest that elevated LTB4 levels may play a role in the inflammatory component of PAH, which can lead to obstructed arterioles, vasoconstriction and worsening cardiac function. Targeted LTB4 blockade may represent an important new therapeutic approach to PAH disease.”
Eiger, headquartered in Palo Alto, California, is a clinical-stage biopharmaceutical company committed to developing and commercializing novel products for the treatment of orphan diseases.