Lisata Therapeutics announced positive preliminary results from Cohort B of the ASCEND Phase 2b trial, demonstrating that certepetide combined with standard-of-care chemotherapy significantly improved clinical outcomes in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC). The findings, scheduled for presentation at the European Society for Medical Oncology Gastrointestinal Cancers Congress on July 2, 2025, represent a potential breakthrough in treating one of the most challenging cancers.
Trial Design and Patient Population
The ASCEND trial is a 158-patient, double-blind, randomized, placebo-controlled Phase 2b clinical trial evaluating standard-of-care gemcitabine and nab-paclitaxel versus the same regimen plus certepetide or placebo in patients with mPDAC. Participants were enrolled from 24 sites across Australia and New Zealand from May 2022 to December 2023.
The study comprises two sequentially enrolled dosing regimens. Cohort A employed one 3.2 mg/kg dose of certepetide administered as an IV push over 1 minute immediately after gemcitabine infusion and before nab-paclitaxel infusion. Cohort B used the same initial dosing but added a second dose of certepetide or placebo administered 4 hours after the first dose.
Significant Clinical Improvements
The preliminary Cohort B data revealed substantial improvements across multiple efficacy endpoints. Six-month progression-free survival reached 60.8% for the certepetide-treated group compared to 25% for the placebo-treated group. Median progression-free survival was 7.5 months for certepetide patients versus 4.7 months for placebo patients.
Objective response rate demonstrated a more than two-fold improvement, with 45.2% of certepetide-treated patients responding compared to 19% of placebo-treated patients. Median overall survival was 10.32 months for the certepetide group compared to 9.23 months for the placebo group.
Corroborating Earlier Results
These Cohort B findings build upon promising Cohort A data presented at the 2025 American Society of Clinical Oncology Gastrointestinal Cancers Symposium in January. The earlier cohort showed a positive trend in overall survival, including four complete responses in the certepetide-treated group compared to none in the placebo-treated group.
Comparison of data from both cohorts indicates that the addition of two doses of certepetide to standard-of-care chemotherapy resulted in clinically meaningful improvements in both progression-free survival and objective response rate for patients with mPDAC.
Addressing Critical Medical Need
Pancreatic cancer represents one of the most challenging oncological conditions, ranking as the 6th leading cause of cancer mortality worldwide. In Australia, pancreatic cancer is the 3rd leading cause of cancer-related deaths. With a five-year survival rate of just 13%, there is considerable need for new treatment options.
"The data from ASCEND provides us with critical new knowledge that will significantly enhance our understanding of how to optimally treat patients battling pancreatic cancer," commented Dr. Andrew Dean, Study Chair. "We are excited by the evidence of certepetide's therapeutic effect and encourage the continued development of this potentially treatment paradigm-changing compound."
Novel Mechanism of Action
Certepetide, formerly known as LSTA1, is an internalizing RGD cyclic peptide designed to activate a novel uptake pathway that allows co-administered anti-cancer drugs to target and penetrate solid tumors more effectively. The drug actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in tumors.
The compound has also been shown to modify the tumor microenvironment, making tumors more susceptible to immunotherapies. Certepetide has received multiple regulatory designations, including Fast Track designation and Orphan Drug Designation for pancreatic cancer in both the U.S. and E.U., as well as Orphan Drug Designation for glioma and osteosarcoma in the U.S.
Future Development Plans
David J. Mazzo, PhD, President and Chief Executive Officer of Lisata, stated, "We are pleased with the promising Cohort B data of the ASCEND trial. These data, taken with those previously reported for Cohort A, reinforce our confidence in the therapeutic promise of certepetide. Along with its attractive safety profile, we continue to believe that certepetide has the potential to transform the treatment landscape for mPDAC and many other devastating solid tumors."
Final data and key findings from both cohorts of the ASCEND study are anticipated to be available later this year. The clinically significant findings provide compelling support for the continued and expedited investigation of certepetide as a novel therapeutic agent for the treatment of metastatic pancreatic cancer.
