Familial Adenomatous Polyposis (FAP) is characterized as a proliferation of polyps in the colon and/or rectum, usually occurring in the mid-teens. If left untreated, it universally leads to colorectal cancer.
There is a significant hereditary component to FAP with a reported prevalence of one in 5,000 to 10,000 in the US and one in 11,300 to 37,600 in Europe.
There is no approved therapeutic option for treating FAP patients, for whom active surveillance and surgical resection of the colon and/or rectum remain the standard of care.
Biodexa (based in Cardiff, UK) has received US FDA Fast Track Designation for eRapa, a proprietary encapsulated form of rapamycin being developed for the treatment of familial adenomatous polyposis. Rapamycin has been shown to have a significant role in the signaling pathway that regulates cellular metabolism, growth, and proliferation and is activated during the genesis of tumors. mTOR (mammalian target of Rapamycin) has been shown to be over-expressed in FAP polyps – thereby underscoring the rationale for using a potent and safe mTOR inhibitor like eRapa to treat FAP. As Rapamycin is already approved for transplants and other conditions, it enabled the FDA to approve eRap for Fast Track.
Through the use of nanotechnology and pH sensitive polymers, eRapa is designed to address the poor bioavailability, variable pharmacokinetics and toxicity generally associated with the currently available forms of rapamycin.
Biodexa is an up-and-coming biotech company that is developing a pipeline of innovative products for the treatment of diseases with unmet medical needs. They underscored the unmet need for a therapeutic alternative with the potential to delay or prevent surgical removal of the colon and/or rectum.
The company recently showed this to be an alternative to surgical resection of the colon and/or rectum. Data from their Phase 2 study of eRapa in FAP showed it to be safe and well-tolerated with a 17% average reduction in total polyp burden at 12 months and an overall 75% non-progression rate compared with baseline. A second group of patients fared even better with 29% average reduction in polyp burden at 12 months and an 89% non-progression rate compared with baseline. The dosing given to the second group – daily every other week — is the preferred dosage regimen for the upcoming Phase 3 study.
Biodexa has now received US FDA Orphan Drug designation for eRapa in FAP and plans to seek a similar designation in Europe.
