Pilatus Biosciences' PLT012 Gains FDA Orphan Drug Status for Liver and Bile Duct Cancer

DOVER, Del. and EPALINGES, Switzerland, Dec. 10, 2024 -- Pilatus Biosciences Inc. has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for its primary drug candidate, PLT012, intended for the treatment of liver and intrahepatic bile duct cancers (HCC/ICCA). This achievement, secured in November 2024, is a significant step in the company's mission to develop innovative treatments for these difficult-to-treat cancers.

Pilatus Biosciences, a preclinical-stage biopharmaceutical company, emerged from the Ludwig Institute for Cancer Research and is at the forefront of creating first-of-its-kind biologics aimed at metabolic checkpoints. With backing from the Cancer Research Institute, Pilatus utilizes an innovative approach to immunometabolism, focusing on reprogramming the immune microenvironment to fight cancer more effectively.

Dr. Raven Lin, CEO and Co-founder of Pilatus Biosciences, expressed the company's honor in receiving the ODD for PLT012, emphasizing the importance of addressing the urgent need for new therapies in the field of liver and intrahepatic bile duct cancer. Prof. Ping-Chih Ho, Chair of the Scientific Advisory Board and Co-founder, highlighted that PLT012 uses metabolic checkpoint targeting to reprogram the tumor microenvironment (TME), offering a distinctive therapeutic method. This designation underscores the promising scientific advancements made in this area and motivates further acceleration of PLT012's development.

Pilatus Biosciences is currently advancing PLT012, working closely with regulatory bodies to fast-track the availability of this treatment. PLT012 is a humanized anti-CD36 antibody with a unique dual mechanism of action: it disarms immunosuppressive cell populations and enhances effector T cell functions. The molecule has demonstrated potential against various tumors with unmet medical needs and is on track to submit its first U.S. IND and begin patient dosing in 2025. As a standalone treatment, PLT012 has shown significant anti-tumor efficacy in both immune 'hot' and 'cold' tumor models, improving CD8+ T cell activity and reducing pro-tumorigenic macrophages.

The FDA's Orphan Drug Designation program grants orphan status to drugs designed to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 people in the United States. This status provides sponsors with development incentives, including tax credits for clinical testing, exemptions from prescription drug user fees, and seven years of marketing exclusivity upon FDA approval.

Liver and intrahepatic bile duct cancer, primarily hepatocellular carcinoma (HCC) and intrahepatic cholangiocarcinoma (ICCA), often require complex treatment strategies. Metabolic reprogramming plays a critical role in these cancers by modifying the TME to support tumor growth. The incidence of recurrence in these cancers is notably high, necessitating multiple lines of treatment.

PLT012 shows promise as a therapeutic candidate with its dual mechanism that complements existing treatments and potentially enhances therapeutic outcomes through immune stimulation in the TME. Pilatus Biosciences is committed to discovering and developing innovative antibodies and bifunctional proteins targeting metabolic checkpoints, aiming to reprogram the immuno-microenvironment to fight cancer.

The company has established a laboratory in Taiwan to bolster its research and development efforts, focusing on early clinical development and biomarker discovery. Pilatus operates globally, leveraging an international team and fostering collaborations to drive the development of groundbreaking therapies in oncology.