Lisata Therapeutics, Inc. announced promising preclinical results for its investigational candidate, certepetide (formerly LSTA1). The data, presented in a poster by Dr. Dan G. Duda of Massachusetts General Hospital at the 2024 Cholangiocarcinoma Foundation Annual Conference, showed that certepetide combined with standard-of-care chemotherapy and immunotherapy improved survival in mice with intrahepatic cholangiocarcinoma. These findings suggest potential benefits for human patients with this aggressive cancer and support advancing clinical development efforts for certepetide in intrahepatic cholangiocarcinoma.

Intrahepatic cholangiocarcinoma, also known as intrahepatic bile duct cancer, is a rare, aggressive cancer that starts in the bile ducts within the liver. It is the second most common liver malignancy, making up about 10% of all cholangiocarcinoma. Intrahepatic cholangiocarcinoma has an overall poor prognosis, and symptoms are usually nonspecific, contributing to an advanced tumor stage at diagnosis.

Certepetide (formerly LSTA1) is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. Certepetide actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. Certepetide has also been shown to modify the tumor microenvironment, diminishing its immunosuppressive nature and inhibiting the metastatic cascade.

To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to test its ability to enhance the effectiveness of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of certepetide to enable a variety of treatment modalities to treat a range of solid tumors more effectively. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.) as well as Orphan Drug Designation for glioma (U.S.) and osteosarcoma (U.S.).

Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.).