Core Insights - Biodexa Pharmaceuticals has received Fast Track designation from the US FDA for eRapa, a treatment for familial adenomatous polyposis (FAP), highlighting the urgent need for alternatives to surgical intervention [1][2] Group 1: eRapa and Its Development - eRapa is a proprietary encapsulated form of rapamycin, designed to address the unmet medical need for FAP, a condition that can lead to colorectal cancer if untreated [1][2] - The Phase 2 study of eRapa demonstrated a median 17% reduction in total polyp burden at 12 months compared to baseline, with a 75% non-progression rate overall [2] - In cohort 2 of the study, patients experienced an 89% non-progression rate and a 29% median reduction in polyp burden at 12 months, with a preferred dosing regimen of daily every other week for the upcoming Phase 3 study [2] Group 2: Familial Adenomatous Polyposis (FAP) - FAP is characterized by a proliferation of polyps in the colon and/or rectum, typically beginning in the mid-teens, with no approved therapeutic options currently available [3] - The prevalence of FAP is reported to be one in 5,000 to 10,000 in the US and one in 11,300 to 37,600 in Europe, indicating a significant hereditary component [3] Group 3: Mechanism and Technology - eRapa functions as an mTOR inhibitor, which plays a crucial role in cellular metabolism, growth, and proliferation, and is over-expressed in FAP polyps [4][8] - The formulation of eRapa utilizes nanotechnology and pH-sensitive polymers to improve bioavailability and reduce toxicity associated with existing rapamycin forms [4] Group 4: Company Overview - Biodexa Pharmaceuticals is a clinical stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, including eRapa for FAP and other programs targeting type 1 diabetes and rare brain cancers [7]
Biodexa Receives US FDA Fast Track Designation for eRapa in Familial Adenomatous Polyposis