EMA Grants Orphan Drug Designation to LSTA1 for Pancreatic Cancer

Kristen K. Buck, MD

The European Medicines Agency’s (EMA) Committee for Orphan Medical Products has granted orphan drug designation to LSTA1 (formerly CEND-1) for the treatment of patients with pancreatic cancer.¹

LSTA1 is being evaluated in several ongoing clinical trials in a variety of solid tumors. In pancreatic cancer, the agent is being evaluated vs placebo in combination with gemcitabine plus nab-paclitaxel (Abraxane) in patients with newly diagnosed, metastatic disease in the phase 2 ASCEND trial (NCT05042128).²

“Pancreatic cancer has one of the highest mortality rates of all cancers and affects hundreds of thousands of patients worldwide each year. Although progress has been made in understanding and treating pancreatic cancer, there remains significant unmet medical need,” Kristen K. Buck, MD, executive vice president of R&D and chief medical officer of Lisata Therapeutics, stated in a news release.

“To date, LSTA1 has demonstrated favorable safety, tolerability, and activity to enhance delivery of standard-of-care chemotherapy for patients with metastatic pancreatic cancer. Obtaining orphan drug designation from the EMA reinforces our belief that LSTA1 offers major improvement in treating patients with this terrible disease. We are excited by the promise of LSTA1 for the treatment of pancreatic cancer and other solid tumors and are committed to advancing our development programs with the goal of providing a benefit to patients.”

On August 8, 2023, the FDA granted orphan drug designation to the agent for the treatment of patients with malignant glioma.³

LSTA1 is an investigational drug designed to activate the transport system, a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target solid tumors more effectively. LSTA1 is also designed to modify the tumor microenvironment, making tumors more susceptible to treatment with immunotherapy.

Previously, preclinical data from Lisata and their partners have shown improved delivery of anti-cancer therapies, including chemotherapy, immunotherapy, and RNA-based treatments. Additionally, LSTA1 has proved safe, tolerable, and active in clinical trials seeking to improve the delivery of standard chemotherapy for patients with pancreatic cancer.¹

To be eligible for enrollment in ASCEND, patients at least 18 years of age must have histologically confirmed metastatic pancreatic ductal adenocarcinoma or poorly differentiated carcinoma; measurable disease per RECIST v1.1 criteria; archival tumor tissue for correlative study; an ECOG performance status of 0 or 1; and adequate renal, hematologic, and hepatic function.²

Patients are being randomly assigned to receive 3.2 mg/kg of LSTA1 plus 125 mg/m2 of nab-paclitaxel and 1000 mg/m2 of gemcitabine on days 1, 8, and 15 of each 28-day cycle, or the same regimen of nab-paclitaxel and gemcitabine plus placebo.

Progression-free survival will be evaluated as the primary end point. Secondary end points will include overall survival, objective response rate, patient-reported outcomes, and safety.

References

1. Lisata Therapeutics announces European Medicines Agency has granted LSTA1 orphan drug designation for the treatment of pancreatic cancer. News release. Lisata Therapeutics. October 17, 2023. Accessed October 17, 2023. https://ir.lisata.com/news-releases/news-release-details/lisata-therapeutics-announces-european-medicines-agency-has
2. The ASCEND study: gemcitabine and nab-paclitaxel with CEND-1 or placebo in patients with untreated metastatic pancreatic ductal adenocarcinoma (ASCEND). ClinicalTrials.gov. Updated January 26, 2023. Accessed October 17, 2023. https://www.clinicaltrials.gov/study/NCT05042128
3. Lisata Therapeutics announces US FDA orphan drug designation granted to LSTA1 for the treatment of malignant glioma. News release. August 8, 2023. Accessed August 8, 2023. https://ir.lisata.com/news-releases/news-release-details/lisata-therapeutics-announces-us-fda-orphan-drug-designation