Biodexa's eRapa Gains FDA Fast Track for Familial Adenomatous Polyposis

Biodexa Pharmaceuticals PLC has announced that the US Food and Drug Administration (FDA) has awarded Fast Track designation to its drug, eRapa, aimed at treating familial adenomatous polyposis (FAP). This designation is crucial as it accelerates the development and review process for drugs that address serious health conditions and fill unmet medical needs. FAP is a condition that, if untreated, almost invariably leads to colorectal cancer, with current treatment options being limited to surgical removal of the colon and/or rectum.

The decision to grant Fast Track status to eRapa was influenced by data from a Phase 2 study that showed the drug's potential to meet an unmet medical need in FAP treatment. The study revealed that eRapa was safe and well-tolerated, demonstrating a median reduction of 17% in total polyp burden over twelve months and a non-progression rate of 75%. Notably, patients in one study cohort achieved an 89% non-progression rate and a 29% reduction in polyp burden using a dosing regimen that will be applied in the upcoming Phase 3 study.

FAP typically manifests in adolescence with the growth of numerous polyps in the colon and/or rectum. Without treatment, it almost certainly progresses to colorectal cancer. Currently, no approved non-surgical therapeutic options exist for FAP, making the development of eRapa particularly significant. FAP is also known to be hereditary, affecting between 1 in 5,000 to 10,000 individuals in the United States and 1 in 11,300 to 37,600 in Europe. Biodexa has already received Orphan Drug designation for eRapa from the FDA and plans to seek similar recognition in Europe.

The drug eRapa is an encapsulated form of rapamycin, a known mTOR (mammalian Target Of Rapamycin) inhibitor, which plays a critical role in controlling cellular growth, metabolism, and proliferation. mTOR is often over-expressed in FAP polyps, providing a rationale for using eRapa to combat the condition. Traditional forms of rapamycin, such as Rapamune® (Pfizer), are used primarily to prevent organ rejection in kidney transplants. However, eRapa employs advanced nanotechnology and pH-sensitive polymers to overcome the challenges of poor bioavailability and variable pharmacokinetics, which are common with existing rapamycin formulations. The protective patents for eRapa extend until 2035, with additional patent applications potentially extending beyond that year.

Biodexa, a clinical-stage biopharmaceutical enterprise listed on NASDAQ as BDRX, is committed to developing groundbreaking treatments for diseases with unmet medical needs. In addition to eRapa, their pipeline includes tolimidone, aimed at treating type 1 diabetes, and MTX110, which is targeted at aggressive, rare brain cancers. Tolimidone works by inhibiting Lyn kinase to enhance insulin sensitivity, showing promise as a novel agent for blood glucose control. MTX110 is an innovative formulation of panobinostat, a histone deacetylase inhibitor, designed to deliver chemotherapy directly to brain tumors, minimizing systemic side effects.

The company is supported by proprietary drug delivery technologies that focus on enhancing the bio-delivery and distribution of medications. Based in Cardiff, UK, Biodexa continues to lead in the research and development of solutions for complex medical conditions.