Versantis, a clinical-stage biotech company founded by scientists from ETH Zurich and industry experts, is focused on the development of orphan drugs in liver and pediatric diseases for which today there is still a large unmet medical need. Globally 850 million people live with liver disease and 2 million die every year, primarily due to cirrhosis. Most cirrhotic patients eventually decompensate, requiring hospitalisation due to complications, such as hepatic encephalopathy (HE) or acute-on-chronic liver failure (ACLF). As of today, no specific treatments are approved to support such cases, which are associated with high mortality if not medically managed early.
The company’s lead candidate, VS-01 is a lipid-based, potentially lifesaving, multi-organ support therapy that aims to reverse Acute-on-Chronic Liver Failure (ACLF) by enhancing the clearance of ammonia and other toxins from the body. By reversing HE and the multi-organ failure cascade of ACLF, VS-01 aims to urgently fill the outstanding medical gap. VS-01 was granted orphan drug designations from both the US FDA and EMA.
Today, versants announced positive results from the Phase 1b clinical trial of its lead candidate VS-01 in patients with decompensated cirrhosis. The results showed that VS-01 was safe and well-tolerated, with no dose-limiting toxicities or unexpected safety signals. No serious adverse events (AE) were reported, and no patients discontinued because of an AE. Patients receiving multiple doses of VS-01 showed improvements in selected biomarkers and clinical cognitive tests, which support the clinical potential of VS-01 and encourage its further investigation in a Phase 2a study in the target indication of ACLF.
Meriam Kabbaj, COO and co-Founder of Versantis commented: “The successful completion of Versantis’ first-in-human study is another great achievement I am extremely proud of. Beyond the safety of VS-01, this study desacralises the i.p. route of administration in cirrhotic patients and this will certainly open up new therapeutic avenues bringing new hope to patients with ACLF.”
The promising data supports the advancement of VS-01 into Phase 2 starting early 2022 to determine efficacy. Versantis hopes to soon provide a meaningful new treatment option to patients with acute liver diseases, for which today there is still a large unmet medical need.