Conatus Pharmaceuticals Initiates Phase 2 Trial In Patients With Liver Cirrhosis And Separate Open Label Phase 2 Trial In Cirrhosis Patients With Portal Hypertension

Conatus Pharmaceuticals Inc.

, a biotechnology company focused on the development and commercialization of novel medicines to treat liver disease, today announced the initiation of a Phase 2 clinical trial of its lead drug candidate, emricasan, in patients with liver cirrhosis (LC), and the initiation of a separate exploratory Phase 2 trial in cirrhosis patients with portal hypertension (PH). The Phase 2 LC clinical trial is designed to consist of two phases and enroll approximately 80 subjects at approximately 20 sites in the United States. In the first phase, which is double-blind and placebo-controlled, subjects will be randomized 1:1 to receive either 25 mg of emricasan or placebo orally twice daily for three months. In the second phase, which will be open label, subjects who complete the first phase of the trial, either on treatment or placebo, may receive emricasan for up to an additional three months. Top-line data from the first phase of this trial are expected to be available in the second half of 2015. Inclusion criteria will include clinical, radiological, or biochemical evidence of liver cirrhosis and a Model for End-Stage Liver Disease (MELD) score of 11 to 18 during the screening period. The primary endpoint will be change from baseline in cleaved cytokeratin 18 (cCK18), a mechanism-specific biomarker of apoptosis associated with excessive cell death, which contributes to chronic inflammation. Secondary endpoints will include change from baseline in MELD score and change from baseline in Child-Pugh score, an assessment of the prognosis of chronic liver disease, primarily cirrhosis. The clinical trial will also evaluate other key serum biomarkers and the safety and tolerability of emricasan in the target patient population. The exploratory, open label Phase 2 clinical trial in PH patients is designed to enroll approximately 20 subjects at approximately eight sites in the United States. Inclusion criteria will include clinical, radiological, or biochemical evidence of liver cirrhosis and portal hypertension confirmed by hepatic venous pressure gradient (HVPG) procedure prior to enrollment. Subjects will receive 25 mg of emricasan orally twice daily for 28 days. The primary endpoint will be change from baseline in cCK18 and HVPG. Secondary endpoints will include change from baseline in MELD score and change from baseline in Child-Pugh score. The clinical trial will also evaluate other key serum biomarkers and the safety and tolerability of emricasan in the target patient population. Top-line data from this trial are expected to be available in the third quarter of 2015 and this open label trial may allow for interim data evaluations. "Liver cirrhosis represents a high medical need segment of the liver disease spectrum," said Conatus President and Chief Executive Officer, Steven J. Mento, Ph.D. "Current treatment options are limited and designed to control symptoms in an attempt to keep a patient healthy enough until a suitable organ becomes available and the patient undergoes a liver transplant. We believe our new trials will provide important information regarding emricasan's potential to benefit cirrhosis patients and help us prioritize opportunities for Phase 3 clinical development and ultimately the pathway for the initial commercialization of emricasan."