The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to lixudebart (ALE.F02) for the treatment of idiopathic pulmonary fibrosis (IPF). The announcement was made in May 2024 by Alentis Therapeutics, a clinical-stage biotechnology company based in Switzerland.
“The Orphan Drug designation for lixudebart underscores the urgent medical need for an effective IPF treatment,” said Alentis Chief Medical Officer Luigi Manenti, MD. “We have completed IND-enabling studies for our IPF program, and we strongly believe that targeting CLDN1 in fibrotic lungs with our highly specific antibody has the potential to reverse the course of the disease.”
Lixudebart is a first-in-class monoclonal antibody developed for liver, lung and kidney fibrosis. The investigational antibody is designed to reverse organ fibrosis by specifically targeting a unique Claudin-1 (CLDN1) epitope exposed in fibrotic tissue. CLDN1 is a previously unexploited target that plays a key role in the pathology of cancer and fibrotic disease.
In phase 1 single- and multiple-ascending dose studies in healthy volunteers, lixudebart was observed to be well tolerated, with no serious safety concerns. Lixudebart is currently being assessed in clinical trials for advanced liver fibrosis (NCT05939947) and ANCA-associated vasculitis (NCT06047171).
“IPF patients do not have transformative treatment options and often experience tolerability challenges with the current standard of care drugs. Alentis is doing important work on developing lixudebart as a treatment targeting the root causes of disease,” said Steven Nathan, MD, medical director of the Advanced Lung Disease and Transplant Program at Inova Fairfax Hospital. “This molecule has shown a very favorable safety profile in phase 1, and I would be thrilled to see lixudebart tested in IPF patients in the future.”
