Lisata Therapeutics Secures FDA Orphan Drug Designation for Certepetide in Treating Aggressive Bile Duct Cancer

Lisata Therapeutics, Inc. (Nasdaq: LSTA), a clinical-stage pharmaceutical company developing therapies for advanced solid tumors, has announced that its investigational drug, certepetide, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of cholangiocarcinoma, a rare and aggressive form of cancer. Cholangiocarcinoma, or bile duct cancer, forms in the bile ducts, essential for digestion. It is difficult to diagnose and often misclassified, leading to underreported cases. While around 8,000 people are diagnosed annually in the U.S., the actual number may be higher. With a five-year survival rate of under 5%, there is an urgent need for new treatments.

Key Highlights:

• The FDA has granted Orphan Drug status to certepetide, offering incentives like seven years of exclusive marketing rights post-approval and potential tax credits for qualified clinical trials.
• Certepetide is being evaluated in the BOLSTER trial, a Phase 2a study for first- and second-line cholangiocarcinoma treatments.
• Cholangiocarcinoma has limited treatment options, and the five-year survival rate is below 5%, making new therapies like certepetide crucial for improving outcomes.

About Lisata Therapeutics

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 product candidate, certepetide , 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 1.5 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 on the Company, please visit www.lisata.com .