Lixudebart

Agomab is now eligible for market exclusivity and a range of financial incentives. Image credit: Shutterstock / HammadKhn. Six months after kicking off a Phase I trial for idiopathic pulmonary fibrosis (IPF) treatment AGMB-447, Agomab Therapeutics has now secured a US Food and Drug Administration (FDA) orphan drug designation for the asset. Recommended Buyer's Guides Buyer's Guide Leading Guide to Packaging Materials, Containers and Containment Services for the Pharmaceutical Industry Buyer's Guide Leading Guide to Compliance Software for the Pharmaceutical Industry The Belgium-based biotech will now be in line for tax credits for US-based clinical trials and, if the therapy is approved, the potential for seven years of market exclusivity in the designated indication. IPF is a rare, chronic lung disease that affects the air sacs – called alveoli – in the lungs. The condition is characterised by thickening of lung tissue that becomes stiff, causing breathing difficulties. It is not known what causes the disease, though the risk is increased with smoking and a family history of IPF. There are currently two FDA-approved drugs for IPF, Boehringer Ingelheim’s Ofev (nintedanib) and Genentech’s Esbriet (pirfenidone). Ofev is a tyrosine kinase inhibitor whilst Esbriet targets TGF-beta, a chemical mediator that plays a role in development of fibrosis. Agomab has gone down a similar route to Genentech, with AGMB-447 blocking the action of the TGFβ type 1 receptor. Agomab’s USP is that AGMB-447, which is inhaled, inhibits the TGFβ type 1 receptor, also known as ALK5, only in the lung. The biotech has achieved this by designing the drug to be rapidly metabolised through hydrolysis in the plasma, preventing exposure throughout the whole body. See Also: The tiny pills that may send shockwaves across immunology Roche’s PI3K inhibitor secures breakthrough status in breast cancer Agomab is evaluating AGMB-447 in a placebo-controlled Phase I trial that aims to enrol 76 patients with IPF. Participants will either receive a single dose or multiple doses of AGMB-447, the latter treatment will occur in two cohorts over seven days or 14 days. The randomised trial has primary endpoints comprising safety assessments up to eight weeks after therapy. Secondary endpoints include pharmacokinetic measurements. The biotech dosed the first patient in December 2023. Agomab’s chief medical officer Philippe Wiesel said: “As we progress through our ongoing first-in-human Phase I trial, we look forward to evaluating the data from the single ascending dose and multiple ascending dose evaluation of AGMB-447 in healthy subjects and IPF patients.” Agomab secured $100m in Series C financing in October 2023. In a statement at the time, the biotech said it would use part of the funds to progress Crohn’s disease candidate AGMB-129 – also an ALK5 inhibitor – through Phase II trials. Agomab’s designation comes only a few weeks after Alentis Therapeutics won orphan drug status for its candidate lixudebart in IPF. In the same announcement as its orphan drug designation, Alentis reported positive safety results from a Phase I trial evaluating lixudebart in IPF patients.

Lixudebart (ALE.F02) is a first-in-class monoclonal antibody in clinical development for multi-organ fibrosis BASEL, Switzerland--(BUSINESS WIRE)-- Alentis Therapeutics (“Alentis”), the clinical-stage biotechnology company developing treatments for Claudin-1 positive (CLDN1+) tumors and organ fibrosis, announced today that the U.S. Food and Drug Administration (FDA) has granted lixudebart (ALE.F02) Orphan Drug designation for the treatment of Idiopathic Pulmonary Fibrosis (IPF). Dr. Luigi Manenti, Chief Medical Officer of Alentis said, “The Orphan Drug designation for lixudebart underscores the urgent medical need for an effective IPF treatment. 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.” “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,” added Prof. Steven Nathan, Medical Director of the Advanced Lung Disease and Transplant Program at Inova Fairfax Hospital. “This molecule has shown a very favorable safety pro Phase 1, and I would be thrilled to see lixudebart tested in IPF patients in the future.” The FDA provides orphan status to drugs or biologics to prevent, diagnose or treat a rare disease or condition. Orphan Drug designation qualifies sponsors for certain incentives, including tax credits for qualified clinical trials, user-fee exemptions and a potential seven years of market exclusivity after approval. About lixudebart (ALE.F02) 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 CLDN1 epitope exposed in fibrotic tissue. 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 tested in clinical trials for advanced liver fibrosis (NCT05939947) and ANCA-associated vasculitis (NCT06047171). About IPF Idiopathic pulmonary fibrosis, or IPF, is a rare and serious chronic disease affecting tissue surrounding the air sacs or alveoli in the lungs, resulting in the thickening of lung tissue. Over time, this thickening causes permanent scarring of the lungs, called fibrosis, which makes breathing increasingly difficult. There is currently no cure for this condition with treatments only slowing the progression of IPF. About Alentis Therapeutics Alentis Therapeutics, the CLDN1 company, is a clinical-stage biotech developing breakthrough treatments for CLDN1+ tumors and organ fibrosis. CLDN1 is a previously unexploited target that plays a key role in the pathology of cancer and fibrotic disease. Alentis is the leading company pioneering anti-CLDN1 antibodies and ADCs to modify and reverse the course of disease. Alentis was founded based on ground-breaking research in the laboratory of Prof. Thomas Baumert, MD at the University of Strasbourg and the French National Institute of Health and Medical Research (Inserm). Alentis is headquartered at the pharma-biotech hub in Basel, Switzerland with an R&D subsidiary in Strasbourg, France and clinical operations in the US. Visit