Pilatus Biosciences Receives FDA Orphan Drug Designation for PLT012 in Liver Cancer

Pilatus Biosciences Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its leading molecule, PLT012, for treating liver and intrahepatic bile duct cancer (HCC/ICCA). Achieved in November 2024, this designation marks a crucial advancement in developing innovative therapies for patients facing these challenging cancers. Dr. Raven Lin, CEO, emphasized the company’s commitment to addressing urgent medical needs, while Prof. Ping-Chih Ho highlighted PLT012’s unique therapeutic approach targeting the tumor microenvironment. Pilatus is actively working with regulatory authorities to expedite PLT012’s development and availability.

Pilatus Biosciences, a preclinical-stage biopharmaceutical company spun out from the Ludwig Institute for Cancer Research (Lausanne), is leading the development of first-in-class biologics targeting metabolic checkpoints. Supported by the Cancer Research Institute (New York), the company employs a pioneering approach to immunometabolism, reprogramming the immune microenvironment to combat cancer effectively.

“We are honored to receive Orphan Drug Designation for PLT012, a milestone that reflects our dedication to addressing the urgent need for innovative therapies in liver and intrahepatic bile duct cancer,” said Dr. Raven Lin, CEO and Co-founder of Pilatus Biosciences. Prof. Ping-Chih Ho, Chair of the Scientific Advisory Board and Co-founder of Pilatus Biosciences, added, “PLT012 leverages metabolic checkpoint targeting to reprogram the tumor microenvironment (TME), offering a unique therapeutic approach. This designation highlights the promising scientific discoveries and results we have achieved in addressing the underserved area. It further motivates us to accelerate PLT012’s development and collaborate globally to bring this promising treatment to patients with limited options.”

Currently, Pilatus Biosciences is advancing the development of PLT012, actively engaging with regulatory authorities and stakeholders to expedite the availability of this promising therapy.