Lisata Therapeutics Receives Paediatric Investigation Plan Waiver From EMA for Certepetide in Pancreatic Cancer

Lisata Therapeutics, Inc. recently announced it has reached agreement with the European Medicines Agency (EMA) on a Paediatric Investigation Plan (PIP) for Lisata’s lead investigational product, certepetide (formerly LSTA1), in pancreatic cancer. The EMA has agreed to a product-specific full pediatric waiver for certepetide in pancreatic cancer.

The Paediatric Committee (PDCO) of the EMA recommended and the EMA granted a waiver for certepetide for all subsets of the pediatric population for the treatment of pancreatic cancer on the grounds that “pancreatic cancer occurs only in adults.” This waiver removes any requirement for Lisata to conduct clinical studies of certepetide in children in support of an overall marketing authorization application in Europe for pancreatic cancer. It also allows Lisata to focus its development efforts on bringing this therapy to relevant patients with pancreatic cancer as quickly as possible. By eliminating any requirement for pediatric clinical studies in pancreatic cancer, the waiver significantly reduces the clinical trial burden and overall cost of completing development and achieving market registration in Europe.

“We are pleased that the European Medicines Agency has confirmed that pediatric studies evaluating certepetide are not needed to support a market authorization application in pancreatic cancer,” said Kristen K. Buck, MD, Executive Vice President of Research and Development and Chief Medical Officer of Lisata. “With a waiver in hand, we are now able to streamline our development program as we work toward certepetide’s product registration in Europe. This full waiver is specific to pancreatic cancer and will not affect our commitment to evaluate certepetide in other pediatric solid tumors as required.”

In 2007, the European Union implemented the Paediatric Regulation, a landmark change that significantly impacted the development of medicines for children. This regulation aims to increase the research and development of medicinal products; specifically, those applicable to children. A key part of the regulation was the establishment of the PDCO. The PDCO coordinates the EMA’s work on pediatric medicines by determining the study(ies) companies must conduct in children as part of their overall development program for Europe. A PIP is a development plan designed to ensure that the necessary data are obtained through studies in children to support the authorization of a medicine for children. In some instances, a PIP waiver by the PDCO for certain conditions is recommended and then granted by the EMA when development of a medicine for use in children is not feasible or appropriate.

Certepetide (formerly 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. Certepetide 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. Certepetide also has been shown to modify the tumor microenvironment diminishing the immunosuppressive nature of the tumor microenvironment and inhibiting the metastatic cascade. We and our collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of emerging anti-cancer therapies, including immunotherapies and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to test its ability to enhance the effectiveness of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of certepetide to enable a variety of treatment modalities to treat a range of solid tumors more effectively. Certepetide has been awarded Fast Track designation (US) and Orphan Drug Designation for pancreatic cancer (US and EU) as well as Orphan Drug Designation for glioma (US) and osteosarcoma (US). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (US).

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, certepetide (formerly LSTA1), is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to selectively target and penetrate solid tumors more effectively. Lisata has already established noteworthy commercial and R&D partnerships based on its CendR Platform technology. The company expects to announce numerous milestones over the next two years and believes that its projected capital will fund operations into early 2026, encompassing anticipated data milestones from its ongoing and planned clinical trials. For more information, visit www.lisata.com.