New world-first pilot study for Cholangiocarcinoma

In Patient Speak

Our liver is made up of 300 billion cells, each functioning like its own city—’Cell Cities.’

In any city, things can occasionally spiral out of control. Cancer, in this example, is like a rogue cluster of buildings growing uncontrollably within the city, disrupting its infrastructure and operations.

On the surface of the cell city, there’s a sneaky recruitment station (CD36) that helps these rogue buildings thrive by:

  • Bringing in fuel (like fats) to keep them growing.
  • Recruiting guards (immune-suppressing cells like Tregs and TAMs) to shield the CD36 stations from the body’s patrol officers (T-cells), which are tasked with eliminating unregulated stations.

PLT012 is a humanised anti-CD36 antibody inhibitor that targets and shuts down this recruitment station. By blocking CD36, PLT012:

  • Starves the rogue buildings.
  • Dismantles their defences.
  • Boosts the T-cells’ ability to attack.

This multi-pronged approach makes PLT012 a pioneering treatment, offering hope even for tumours that resist traditional therapies.

Please Share

Pilatus Biosciences Inc. Secures FDA Orphan Drug Designation for PLT012: A Breakthrough in Liver and Intrahepatic Bile Duct Cancer Treatment

News provided by
Pilatus Biosciences Dec 10, 2024, 22:04 ET

DOVER, Del. and EPALINGES, Switzerland, Dec. 10, 2024 /PRNewswire/ — 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.

About Orphan Drug Designation

The FDA’s Orphan Drug Designation (ODD) program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. ODD qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and 7 years marketing exclusivity upon FDA approval.

Join Us And Light Australia Green 2025

Light Australia Green is Celebrating World Cholangiocarcinoma Day
Raising awareness and funds: Ensuring Today’s Science Benefits Today’s Patient Survival.

Visit Our Social Platforms

Navigator Journal and Digital Toolkits

Do you have your free Patient Navigator?