Any drug used for brain cancer must cross the blood-brain barrier.
Both LMP744 and LMP400 easily cross the BBB, are not pumped out by drug transporters, and accumulate in the brain for up to 24 hours after a single dose.
Requirement #2
The drug doses used for brain tumors must be demonstrated to be safe in clinical trials:
LMP744 and LMP400 have been shown to be safe in multiple phase 1 clinical trials done in solid tumors and lymphomas.
Requirement #3
The drug uses must have mechanisms of action known to be important in brain cancer:
With the combination of reducing the over expression of cMyc oncogene and inhibition of TOP-1, both of which are well known cancer targets in brain cancer, LMP744 offers new hope against a deadly disease.
Requirement #4
The mechanisms of action must be impressive:
The demonstration of these effects to the FDA resulted in the orphan drug designation (ODD) for Gibson being more broad than was requested by the company.
Requirement #5
There must be very strong preclinical evidence of activity:
GBM Activity: Extensive NIH studies with LMP744 resulted in an upcoming Phase II trial of LMP744 in GBM patients who have failed one drug.
Requirement # 6
There should be evidence of effectiveness in multiple brain tumors:
In addition to GBM, studies at the NIH have shown evidence of effect in multiple other brain tumors.
Requirement # 7
There must be very strong preclinical evidence of activity:
Duke University has shown in subcutaneous IDH mutant astrocytoma models that LMP400 slows tumor growth.
Conclusion
Documented activity in brain tumors from Gibson drugs that have gone through Phase I trials gives strong evidence of the potential.
