Biodexa Reports Positive 12-Month Phase 2 Results for eRapa™ in FAP

Biodexa Pharmaceuticals PLC has announced promising 12-month results from its Phase 2 clinical trial of eRapa™ for treating Familial Adenomatous Polyposis (FAP). The results, presented by Dr. Carol Burke at the 2024 InSIGHT bi-annual meeting in Barcelona, highlighted a 75% non-progression rate and a median decrease in overall polyp burden of 17% at the one-year mark. FAP, predominantly a genetic disorder characterized by precancerous polyps, currently has no treatment other than surgical removal of the colon and/or rectum.

The study, partially funded by $3 million from the Cancer Prevention and Research Institute of Texas (CPRIT), involved 30 adult patients with a median age of 43 years. Participants were enrolled across three dosing cohorts: 0.5mg every other day, 0.5mg daily every other week, and 0.5mg daily. The primary endpoints were safety and tolerability, along with the percentage change in polyp burden from baseline at six months, with continued monitoring up to 12 months.

Key findings at the 12-month juncture include a 75% overall non-progression rate among 28 patients and a median polyp burden reduction of 17%. Cohort 2 showed particularly promising results with an 89% non-progression rate and a median reduction in polyp burden of 29%. The study reported four related Grade 3 or higher adverse events and one serious adverse event, with a 95% compliance rate.

The study's design and outcomes suggest that Cohort 2's dosing regimen may be optimal for the upcoming Phase 3 trial. This next phase, supported by a $17 million grant from CPRIT, will be a double-blind, placebo-controlled study involving 140 high-risk FAP patients. It aims to assess progression-free survival, considering factors like major surgery, advanced neoplasia, and diagnosis of high-grade dysplasia or cancer.

eRapa, a proprietary oral formulation of rapamycin, targets the mTOR pathway, which is significant in cellular metabolism and growth regulation. Rapamycin, approved in the US for organ rejection prevention, faces challenges such as poor bioavailability and variable pharmacokinetics. eRapa addresses these issues using nanotechnology and pH-sensitive polymers. The drug is protected by several patents extending through 2035.

FAP typically manifests in mid-teens and can lead to colorectal cancer if untreated. It has a notable hereditary component, with an incidence of 1 in 5,000 to 10,000 in the US and 1 in 11,300 to 37,600 in Europe. Current management involves active surveillance and surgical intervention, with no approved therapeutic options. eRapa, which has received Orphan Designation in the US and is seeking similar status in Europe, offers a potential non-surgical treatment by inhibiting the over-expressed mTOR pathway in FAP polyps.

Stephen Stamp, CEO of Biodexa, remarked on the potential of eRapa to delay or even eliminate the need for major surgical interventions, thereby significantly improving the quality of life for FAP patients.

The successful Phase 2 results pave the way for the Phase 3 study, aiming to confirm eRapa's efficacy and safety in a larger patient cohort. If successful, this could mark a significant advancement in the management of FAP, providing a non-surgical treatment option for patients worldwide.