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The use of the optical imaging agent FG001 led to the detection of additional cancer by optical guidance in patients with high-grade glioma, meeting the primary end point of the phase 2b FG001-CT-001 trial.
The use of the optical imaging agent FG001 led to the detection of additional cancer by optical guidance in patients with high-grade glioma, meeting the primary end point of the phase 2b FG001-CT-001 trial (EudraCT 2020-003089-38).1
Findings showed that all patients who received FG001 (n = 12) had additional cancer detected, and FG001 lit up all patients’ cancer with a tumor-to-background ratio greater than 2.
Additionally, no statistically significant differences were reported between FG001 and 5-ALA in terms of sensitivity, specificity, negative predictive value, positive predictive value, or gross total resection measured on MRI. Notably, all patients who received 5-ALA (n = 12) also had additional cancer detected by optical guidance.
Findings also showed that FG001 was well tolerated. Two grade 1 adverse effects were reported.
“These results are in line with our expectations of a safe and effective drug and the data generated will be used for the optimal design of the next stage clinical development. We have positive phase 2 data on FG001 in 3 indications—aggressive brain, head and neck, and lung cancers—which provide multiple options for routes to market," Andreas Kjaer, chief scientific officer of FluoGuide, stated in a news release.
FG001 is a fluorophore designed to target uPAR, a cancer-specific target expressed in the majority of solid tumors. The agent is injected into a patient's vein before surgery and is intended to light up the tumor during surgery to guide the surgeon in its removal and spare healthy tissue.
In October 2023, the FDA granted an orphan drug designation to FG001 as an optical imaging agent for the visualization of malignant tissue during surgery for patients with high-grade glioma.2
The phase 2b trial is being conducted in Copenhagen, Denmark, and it is enrolling patients at least 18 years of age with primary malignant glioblastoma scheduled for neurosurgery.3 Prior FG001 is not permitted. Patients must have surgery planned that will be guided by 5-ALA with the objective of gross total resection, and they must also have adequate organ and bone marrow function.
Key exclusion criteria include any known allergy or hypersensitivity to indocyanine green; a performance status or co-morbidity that would make a patient unfit for participation in the opinion of the investigator; pre-existing hepatic and/or renal insufficiency; and prior history of serious gastrointestinal perforation, diverticulitis, and/or peptic ulcer disease.
Patients are being randomly assigned 1:1 to receive FG001 or 5-ALA. Fluorescence-guided surgery using FG001 or 5-ALA is being evaluated vs white light surgery with each patient serving as their own control. Notably, the trial is not powered to demonstrate statistical significance or non-inferiority.1
In phase 2 of the study, the primary end point is the proportion of patients with at least 1 undetermined tissue or unexpected fluorescent tissue at the end of surgery. Secondary end points include pharmacokinetics; percentage of patients with gross total resection by post-surgery MRI with either FG001 or 5-ALA; positive and negative predictive values for FG001 and 5-ALA; and safety.
“I am pleased with this first result of FG001 efficacy in aggressive brain cancer. Here is a promising drug and we should now define the best use of it for the benefit of our patients,” Jane Skjøth-Rasmussen, MD, PhD, chief physician in the Department of Neurosurgery at Rigshospitalet, Copenhagen, Denmark, and principal investigator of FG001-CT-001, said in a news release.