Certepetide Wins FDA Orphan Drug Designation in Cholangiocarcinoma

• The FDA has granted orphan drug designation to certepetide (formerly LSTA1) for the treatment of cholangiocarcinoma.
• Certepetide is an investigational agent that assists co-administered anticancer drugs to more effectively penetrate tumors.
• The agent is being investigated in the phase 2a BOLSTER study (NCT05712356).

Certepetide has been granted FDA orphan drug designation for the treatment of cholangiocarcinoma.¹

Certepetide is an investigational drug that selectively targets and enhances the uptake of anticancer drugs into solid tumors. Additionally, in preclinical models, certepetide showed the ability to modify the tumor microenvironment.

Orphan drug designation is granted to agents that treat a rare disease or condition affecting fewer than 200,000 people in the US. With this designation, Lisata Therapeutics, the sponsor, could be eligible for up to 7 years of market exclusivity, exemption from user fees, and tax credits for qualified clinical trials.

“Cholangiocarcinoma is a rare and aggressive form of cancer that presents a significant challenge for patients due to limited treatment options, especially after initial therapy,” said Kristen K. Buck, MD,executive vice president of research and development and Chief medical officer of Lisata, in a press release. “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.”

About the Phase 2a BOLSTER Study of Certepetide in Cholangiocarcinoma

The BOLSTER trial has an estimated enrollment of 80 patients and is currently recruiting across locations in Arizona, Florida, Kentucky, Minnesota, North Carolina, Ohio, South Carolina, Texas, and Virginia. The study’s primary end point is incidence of adverse events.² 

Patients are randomized to 1 of 4 arms:

• An experimental arm where patients with untreated cholangiocarcinoma receive certepetide, durvalumab (Imfinzi), cisplatin, and gemcitabine;
• An experimental arm where patients with second-line cholangiocarcinoma receive certepetide with oxaliplatin and 5-fluorouracil (FOLFOX);
• A placebo arm where patients with untreated cholangiocarcinoma receive durvalumab, cisplatin, gemcitabine, and placebo; and
• A placebo arm where patients with second-line cholangiocarcinoma receive FOLFOX and placebo.

Patients with an ECOG performance status of 0 or 1, a life expectancy of at least 3 months, adequate organ and marrow function, and adequate contraception are eligible for enrollment in the study. Those with a history or clinical evidence of symptomatic central nervous system metastases, active autoimmune disease, or any comorbidity that could interfere with study treatment or patient safety in the opinion of the investigator are not eligible for study participation.

REFERENCES:

1. Lisata Therapeutics’ certepetide granted FDA orphan drug designation for the treatment of cholangiocarcinoma. News release. Lisata Therapeutics. September 5, 2024. Accessed September 6, 2024. https://tinyurl.com/4mkfftbw 

2. A study of LSTA1 when added to standard of care versus standard of care alone in patients with advanced solid tumors (BOLSTER). ClinicalTrials.gov. Updated September 3, 2024. Accessed September 6, 2024. https://clinicaltrials.gov/study/NCT05712356