FG001 Gains FDA Orphan Drug Designation for High-Grade Glioma
Top-line results from a phase 2b trial evaluating FG001 for patients with high-grade glioma are expected by the end of November.
- FG001 is a fluorophore targeting uPAR, a cancer-specific target found in various solid tumors.
- FG001 has previously shown positive results in preclinical studies and interim results from a phase 2a trial in patients with head and neck cancer.
- Researchers are also discussing plans for the next stage of clinical development of FG001 with regulatory authorities to ensure effective approval and necessary data.
The FDA has 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.1
“The granting of orphan designation by the FDA is important and may simplify FG001's path toward market approval and can provide significant benefits in conducting clinical trials, as well as during commercialization,” said Morten Albrechtsen, chief executive officer of FluoGuide, in a press release. “We have completed enrollment and treatment in our phase 2b clinical trial of FG001 and are now looking forward to top line results, which we expect by the end of November.”
3d illustration of human body organ(brain anatomy): © PIC4U - stock.adobe.com
FG001 is a fluorophore which targets uPAR, a cancer-specific target expressed in a variety of solid tumors. FG001 has the same spectral specifications as indocyanine green, which is already approved, suggesting that it can be used on current imaging equipment without the need to make any changes.
Previously, FG001 has been evaluated in several preclinical studies, the latest of which was conducted by a human surgeon using human equipment on human cancer transplanted into mice, and FG001 performed as intended. Then in June 2023, positive interim results from a phase 2a trial assessing FG001 in patients with head and neck cancer were reported, showing FG001 to successfully illuminate tumors in biopsies from 100% of the patients.1,2
Now, FG001 is being evaluated in a controlled, randomized, multicenter, phase 2b trial (FG001-CT-001; 2020-003089-38) where investigators will examine the effect of FG001 in guiding surgery of patients with aggressive brain cancer. The effect of FG001 will also be compared with 5-ALA and white light.1,4
In the phase 2b trial being conducted out of Copenhagen, Denmark, patients aged 18 years and older who have primary malignant glioblastoma scheduled for neurosurgery will be enrolled.3 Patients will be included if their surgery is planned to be guided by 5-ALA, and patients must have anticipated gross total resection and adequate organ and bone marrow function to be eligible.
The study will randomly assign patients in a 1:1 fashion to receive FG001 and 5-ALA. In the respective arm, FG001 and 5-ALA will be compared with white light. To note, the trial is not powered to demonstrate statistical significance between FG001, white light or 5-ALA.
Investigators are assessing the efficacy of the agent in patients undergoing surgery as the primary end point, with pharmacokinetics, efficacy of FG001 or 5-ALA after surgery by determination of gross total resection, positive predicted value of FG001 and 5-ALA fluorescence tumor tissue obtained from each trial patient, contrast of FG001 to differentiate tumor tissue from normal tissue, negative predictive value from each trial patient for FG001 vs 5-ALA, and safety and tolerability of the optimal dose of FG001 as secondary end points of the phase 2 portion of the study.4
The trial is ongoing with topline results of the phase 2b portion expected to be announced before the end of November 2023.
“We are now discussing plans for next-stage clinical development of FG001 in aggressive brain cancer with regulators, so we can ensure the most effective route to approval, supported by the necessary data," said Albrechtsen.1
REFERENCES:
FluoGuide receives FDA orphan drug designation for FG001 in high-grade glioma. News Release. FluoGuide. October 3, 2023. Accessed October 4, 2023. https://tinyurl.com/53c2jj53
Pipeline. FluoGuide. Accessed October 4, 2023. https://tinyurl.com/53c2jj53
FluoGuide announces positive interim results from phase IIa trial of FG001 in head & neck cancer. Modular Finance. June 20, 2023. Accessed October 4, 2023. https://tinyurl.com/2w3dmdjk
An open-label, dose escalation, single-dose administration, multi-center phase I/II trial of FG001 (an imaging agent), in patients with glioblastoma scheduled for neurosurgery. ClinicalTrialsRegister.eu. June 10, 2021. Accessed October 4, 2023. https://tinyurl.com/mvv2c2my
