Lisata Therapeutics Gets EMA Waiver for Certepetide in Pediatric Pancreatic Cancer

Lisata Therapeutics, Inc., a clinical-stage pharmaceutical company listed on Nasdaq under the symbol LSTA, has announced a significant milestone regarding its lead investigational product, certepetide (formerly known as LSTA1), for pancreatic cancer. The European Medicines Agency (EMA) has approved a Paediatric Investigation Plan (PIP) for certepetide and granted a full waiver for pediatric studies in pancreatic cancer.

The EMA's Paediatric Committee (PDCO) recommended and the EMA approved this waiver on the basis that "pancreatic cancer occurs only in adults." This decision means that Lisata is not required to conduct clinical trials of certepetide in children for the treatment of pancreatic cancer. Consequently, the company can now allocate resources more efficiently toward developing certepetide for adult patients with pancreatic cancer. The waiver is expected to significantly reduce the clinical trial burden and overall costs associated with bringing the therapy to market in Europe.

Kristen K. Buck, M.D., Executive Vice President of Research and Development and Chief Medical Officer of Lisata, expressed satisfaction with the EMA's decision. She highlighted that the waiver would streamline the company's development program and facilitate the registration process for certepetide in Europe. She also noted that this waiver pertains only to pancreatic cancer and does not affect Lisata's commitment to exploring certepetide's efficacy in other pediatric solid tumors.

The waiver aligns with the 2007 Paediatric Regulation of the European Union, which aims to boost the development of medicines for children. This regulation established the PDCO to oversee pediatric medicine development, ensuring that necessary data is collected through studies in children to support the authorization of medicines for pediatric use. In certain situations, a waiver is recommended when pediatric studies are deemed infeasible or inappropriate.

Certepetide is an investigational drug designed to activate a unique uptake pathway that enhances the penetration of co-administered or tethered anti-cancer drugs into solid tumors. It works by modifying the tumor microenvironment, reducing its immunosuppressive properties, and inhibiting metastatic processes. Preclinical data and ongoing clinical trials have shown that certepetide can improve the delivery and efficacy of various emerging anti-cancer therapies, including immunotherapies and RNA-based treatments. The drug has demonstrated safety, tolerability, and clinical activity in trials focused on enhancing standard chemotherapy for pancreatic cancer.

Lisata Therapeutics is committed to advancing certepetide to treat various solid tumors. The drug has received multiple designations, including Fast Track status in the U.S., Orphan Drug Designation for pancreatic cancer in both the U.S. and the E.U., and Orphan Drug Designation for glioma and osteosarcoma in the U.S. Additionally, it has been granted Rare Pediatric Disease Designation for osteosarcoma in the U.S.

Lisata is a clinical-stage pharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for advanced solid tumors and other serious diseases. The company's lead product candidate, certepetide, leverages its proprietary CendR Platform® technology to selectively target and penetrate solid tumors. Lisata has established notable commercial and R&D partnerships and anticipates several key milestones over the next two years. The company is confident that its current capital will support operations into early 2026, covering anticipated data milestones from ongoing and planned clinical trials. Lisata continues to be at the forefront of developing transformative therapies for cancer patients.