Lisata Therapeutics Inc (NASDAQ:LSTA) announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its investigational product, certepetide, for the treatment of cholangiocarcinoma.
Cholangiocarcinoma, a rare and aggressive form of cancer affecting the bile ducts, presents a significant challenge due to the limited effectiveness of current treatment options, particularly in advanced stages.
The Orphan Drug Designation is intended to encourage the development of treatments for rare diseases by providing certain benefits, including tax credits for clinical research and eligibility for market exclusivity.
“Receiving Orphan Drug Designation for our investigational product, certepetide, is a pivotal step toward addressing the unmet need for cholangiocarcinoma therapies and providing patients with new, innovative treatment options,” said Kristen Buck, Lisata’s chief medical officer in a statement.
Certepetide is an investigational drug that enhances the delivery and effectiveness of anti-cancer therapies by activating the C-end rule active transport mechanism, allowing for improved tumor penetration and accumulation. It also modifies the tumor microenvironment to reduce immunosuppression and inhibit metastasis, showing favorable safety and clinical activity in trials for pancreatic cancer and other solid tumors.
Lisata is currently advancing certepetide in its BOLSTER trial, a Phase 2a study designed to evaluate the efficacy and safety of the drug. The BOLSTER trial is a double-blind, placebo-controlled, multi-center, randomized study conducted in the US to assess certepetide in both first- and second-line treatment settings for cholangiocarcinoma.
Investors welcomed the news, sending shares 4.3% higher on Thursday morning in New York.