Lisata Therapeutics' certepetide, combined with standard-of-care chemotherapy, has demonstrated encouraging preliminary results in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC). The data, from Cohort A of the Phase 2 ASCEND trial, revealed a positive trend in overall survival (OS) and complete response rates compared to placebo, potentially offering a new treatment avenue for this aggressive cancer. The findings will be presented at the 2025 American Society of Clinical Oncology Gastrointestinal Cancers Symposium.
The ASCEND trial (NCT05042128), led by the Australasian Gastro-Intestinal Trials Group (AGITG), is a Phase 2, double-blind, randomized, placebo-controlled study conducted across 25 sites in Australia and New Zealand. It evaluates certepetide in combination with gemcitabine and nab-paclitaxel, the standard chemotherapy regimen for mPDAC. The trial was amended post Lisata's acquisition of Cend Therapeutics to align with international regulatory standards and optimize data collection.
Improved Overall Survival and Complete Response Rates
Preliminary data from Cohort A, which included 95 patients, showed a median overall survival (mOS) of 12.68 months in the certepetide-treated group compared to 9.72 months in the placebo-treated group. While median progression-free survival (mPFS) was similar in both groups (5.5 months), the objective response rate (ORR) was notably better in the certepetide arm, with 4 out of 65 patients (6.2%) achieving complete responses, compared to 0 out of 28 in the placebo arm.
"The data from Cohort A are as we expected and corroborate our decision to add Cohort B to the ASCEND protocol," stated Kristen K. Buck, M.D., Executive Vice President of Research and Development and Chief Medical Officer of Lisata. "The increase in overall survival and the observation of 4 complete responses in the certepetide-treated group compared to none in the placebo group for Cohort A, coupled with our expectation of even better outcomes in Cohort B, where we believe early indications show a strong separation in mPFS benefiting patients treated with certepetide, support our plans to advance certepetide development to Phase 3 in early 2026."
Certepetide's Mechanism of Action
Certepetide (formerly LSTA1) is an investigational iRGD cyclic peptide designed to enhance drug delivery to solid tumors. It activates an uptake pathway that allows co-administered anti-cancer drugs to more effectively target and penetrate tumor tissue. Additionally, certepetide has demonstrated the ability to modify the tumor microenvironment, potentially increasing susceptibility to immunotherapies. The drug has received Fast Track designation and Orphan Drug Designation for pancreatic cancer in the U.S. and E.U., as well as Orphan Drug Designation for glioma and osteosarcoma in the U.S.
Next Steps
Lisata anticipates data from Cohort B of the ASCEND trial in the coming months, with full data from both cohorts to be reported thereafter. The company plans to initiate Phase 3 development of certepetide in early 2026, contingent on these results.
