Lisata's Certepetide Promising for Cholangiocarcinoma Treatment in Preclinical Model

Lisata Therapeutics, Inc., a company at the forefront of developing innovative treatments for advanced solid tumors, has unveiled promising preclinical data for its investigational candidate, certepetide, formerly known as LSTA1. These findings were showcased in a poster by Dr. Dan G. Duda from Massachusetts General Hospital during the 2024 Cholangiocarcinoma Foundation Annual Conference. The data indicate that certepetide, when used in conjunction with standard chemotherapy and immunotherapy, enhances survival rates in mice afflicted with intrahepatic cholangiocarcinoma. This development suggests potential therapeutic benefits for human patients suffering from this aggressive cancer, underpinning the need for further clinical investigations into certepetide's efficacy.

Kristen K. Buck, M.D., Executive Vice President of Research and Development and Chief Medical Officer at Lisata, highlighted the encouraging nature of previous preclinical and early clinical results for certepetide. Lisata is currently running a Phase 2a clinical trial, named the BOLSTER trial, to assess certepetide combined with standard chemotherapy as a first-line treatment for cholangiocarcinoma. The BOLSTER trial is expanding to include a new arm that will test certepetide with standard chemoimmunotherapy, driven by recommendations from trial investigators and the pressing need for second-line treatments. Results for both trial arms are anticipated in 2025, with regular updates expected throughout the year.

Dr. Duda's research, titled "Enhancing the Efficacy of Standard Therapy in Intrahepatic Cholangiocarcinoma Using LSTA1 (certepetide), a Novel Tumor Targeting and Penetration Agent," provided critical insights. The study demonstrated that certepetide, combined with anti-PD-1 immunotherapy and gemcitabine/cisplatin chemotherapy, significantly prolonged survival in an aggressive mouse model of intrahepatic cholangiocarcinoma. The treatment also resulted in reduced disease morbidity and lung metastasis while lowering the risk of death by 76%. Moreover, it was well tolerated in mice, presenting no overt additional toxicities. Preliminary data suggest that certepetide facilitates immune cell infiltration into tumor tissues by opening collapsed tumor vessels, thus enhancing immune response.

Prof. Dan G. Duda emphasized the significance of these preclinical findings, noting the substantial need for new and more effective treatment options for intrahepatic cholangiocarcinoma, a disease known for its poor prognosis. The promising data from animal models support continued development of certepetide as a potential breakthrough therapy for this severe condition.

Intrahepatic cholangiocarcinoma, also referred to as intrahepatic bile duct cancer, originates in the bile ducts within the liver and is characterized by its aggressive nature. It is the second most common type of liver cancer, accounting for roughly 10% of all cholangiocarcinomas. The overall prognosis for this cancer type is dismal, often due to nonspecific symptoms leading to late-stage diagnoses.

Certepetide aims to enhance the effectiveness of anti-cancer drugs in penetrating solid tumors through a novel uptake pathway. This investigational drug not only facilitates better drug delivery to tumors but also modifies the tumor microenvironment, reducing its immunosuppressive properties and inhibiting metastasis. Non-clinical data have shown that certepetide improves the delivery of various emerging therapies, including immunotherapies and RNA-based treatments. Certepetide has already shown a favorable safety profile in completed and ongoing clinical trials for pancreatic cancer treatment and holds several designations, including Fast Track and Orphan Drug Designations for multiple cancers.

Lisata Therapeutics is committed to discovering, developing, and commercializing cutting-edge therapies for advanced solid tumors and other serious conditions. With certepetide as its lead candidate, Lisata has formed significant commercial and research partnerships based on its proprietary CendR Platform® technology. The company expects to achieve several milestones in the next two years and is financially positioned to support its operations until early 2026, which includes the anticipated results from its clinical trials.