Lisata Therapeutics Announces EMA Has Granted LSTA1 Orphan Drug Designation for the Treatment of Pancreatic Cancer 

Lisata Therapeutics, Inc. recently announced the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted orphan drug designation to LSTA1, the company’s lead product candidate for the treatment of pancreatic cancer. Currently, LSTA1 is the subject of multiple ongoing and planned clinical studies being conducted globally in a variety of solid tumor types, including pancreatic cancer, in combination with a variety of anti-cancer regimens.

“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,” said Kristen K. Buck, MD, Executive Vice President of R&D and Chief Medical Officer of Lisata. “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.”

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion adopted by the EMA Committee for Orphan Medicinal Products. The EMA’s orphan designation is given to drugs and biologics in the EU for conditions defined as rare diseases, which affect no greater than 5 in 10,000 people in the EU. Companies that meet the EMA’s orphan designation criteria are eligible for incentives that may include protocol assistance from the EMA, potential fee reductions, and EU marketing exclusivity of the particular indication for 10 years after approval.

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. LSTA1 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. LSTA1 also has the potential to modify the tumor microenvironment, making tumors more susceptible to immunotherapies. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies and RNA-based therapeutics. Additionally, LSTA1 has demonstrated favorable safety, tolerability and activity in clinical trials to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively.

Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s lead product candidate, LSTA1, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to target and penetrate solid tumors more effectively. Based on Lisata’s CendR Platform Technology, Lisata has already established noteworthy commercial and R&D partnerships. The company expects to announce numerous clinical study and business milestones over the next 2 years and has projected that its current business and development plan is funded with available capital through these milestones and into early 2026. For more information, visit www.lisata.com.