FDA Grants Orphan Drug Status to Alentis' Lixudebart for Idiopathic Pulmonary Fibrosis

In a significant advancement for the treatment of Idiopathic Pulmonary Fibrosis (IPF), Alentis Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational drug lixudebart (ALE.F02). Alentis Therapeutics is a clinical-stage biotechnology firm dedicated to developing treatments targeting Claudin-1 positive (CLDN1+) tumors and organ fibrosis.

Dr. Luigi Manenti, Chief Medical Officer at Alentis, emphasized the critical need for new IPF treatments, noting that the Orphan Drug status highlights the potential of lixudebart to address this unmet medical need. According to Dr. Manenti, the company has completed the necessary IND-enabling studies and believes that their highly specific antibody, which targets CLDN1 in fibrotic lungs, could potentially alter the disease's trajectory.

Prof. Steven Nathan, Medical Director of the Advanced Lung Disease and Transplant Program at Inova Fairfax Hospital, added that current IPF treatments often come with significant tolerability issues and do not substantially transform patient outcomes. Expressing optimism about lixudebart, he pointed out that the molecule had demonstrated a favorable safety profile in Phase 1 trials. Prof. Nathan is hopeful that the drug will be tested in IPF patients in future clinical trials.

The FDA's Orphan Drug designation is awarded to drugs or biologics aimed at preventing, diagnosing, or treating rare diseases or conditions. This designation offers several incentives to sponsors, such as tax credits for eligible clinical trials, exemptions from user fees, and potentially seven years of market exclusivity following approval.

Lixudebart (ALE.F02) is a pioneering monoclonal antibody developed for the treatment of fibrosis in the liver, lungs, and kidneys. This investigational drug is designed to specifically target a unique CLDN1 epitope present in fibrotic tissues, with the aim of reversing organ fibrosis. In Phase 1 studies involving single- and multiple-ascending doses in healthy volunteers, lixudebart was found to be well tolerated, with no significant safety concerns. The drug is currently undergoing clinical trials for advanced liver fibrosis (NCT05939947) and ANCA-associated vasculitis (NCT06047171).

Idiopathic Pulmonary Fibrosis (IPF) is a rare, severe chronic disease that affects the tissue surrounding the alveoli, or air sacs, in the lungs. This leads to the thickening of lung tissue and eventually results in permanent lung scarring, known as fibrosis, which makes breathing increasingly difficult. Currently, there is no cure for IPF, and available treatments only aim to slow the disease's progression.

Alentis Therapeutics, known as the CLDN1 company, is a clinical-stage biotech firm focused on developing groundbreaking treatments for CLDN1+ tumors and organ fibrosis. CLDN1 is a previously untapped target that plays a significant role in the pathology of both cancer and fibrotic diseases. Alentis is pioneering the development of anti-CLDN1 antibodies and antibody-drug conjugates (ADCs) to modify and potentially reverse the course of these diseases.

The foundation of Alentis Therapeutics is rooted in pioneering research conducted in the laboratory of Prof. Thomas Baumert, MD, at the University of Strasbourg and the French National Institute of Health and Medical Research (Inserm). The company is headquartered in Basel, Switzerland, a major hub for pharma-biotech innovation, with a research and development subsidiary in Strasbourg, France, and clinical operations based in the United States.

In conclusion, the FDA's Orphan Drug designation for lixudebart represents a promising step forward for Alentis Therapeutics and patients suffering from IPF, offering hope for a more effective treatment in the future.

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