Biodexa's FAP Drug Receives FDA Fast Track Status Following Positive Phase 2 Results and Orphan Drug Designation

Biodexa Pharmaceuticals (NASDAQ: BDRX) has received a significant boost with the U.S. Food and Drug Administration (FDA) granting Fast Track designation for its proprietary oral tablet formulation of rapamycin, eRapa™, for the treatment of Familial Adenomatous Polyposis (FAP). This designation highlights the urgent need for effective treatment options for FAP, a condition that without intervention leads to colorectal cancer, with current standard care being surgical removal of the colon and rectum.

The Phase 2 study of eRapa™ showed promising results, including a median 17% reduction in polyp burden and a 75% non-progression rate over 12 months. Notably, cohort 2 demonstrated even better outcomes with an 89% non-progression rate and a 29% median reduction in polyp burden using an alternate-week dosing regimen, which will be used in the upcoming Phase 3 study. FAP has a hereditary prevalence, affecting roughly 1 in 5,000 to 10,000 individuals in the US. Biodexa is also pursuing Orphan Drug designation for eRapa™ in Europe.

The FDA's Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions where there is an unmet medical need. FAP, an inherited condition that puts people at a much greater risk of developing colon cancer, falls into that category. The condition is typically diagnosed in the early teenage years and results in a nearly 100% lifetime risk of colorectal cancer.



The Fast Track designation allows for an expedited review process, which can help Biodexa bring eRapa™ to market more quickly. This designation is intended for drugs that treat serious conditions and have the potential to address unmet medical needs. In the case of eRapa™, the FDA has recognized its potential to address the critical unmet need in FAP treatment, which currently lacks approved therapeutic options, and the only available treatment is surgical resection of the colon and/or rectum.

The Phase 2 results are particularly compelling from a clinical perspective. The 89% non-progression rate in cohort 2 is remarkable for a disease that typically progresses relentlessly. The 29% median reduction in polyp burden suggests not just disease stabilization but actual regression, which is unprecedented in FAP treatment. The alternate-week dosing regimen selected for Phase 3 appears to optimize both efficacy and tolerability, important for a treatment that may require long-term administration.

The market opportunity is substantial despite FAP's rare disease status. With current treatment to surgical intervention costing $50,000-$100,000 per procedure, eRapa™ could potentially capture a significant portion of a market estimated at $500M-$1B annually. The 29% median reduction in polyp burden observed in cohort 2 could translate to meaningful delays in surgical intervention, providing both clinical and economic benefits.

Three key factors make this development particularly significant:

1. eRapa™ is positioned to be the first FDA-approved therapeutic option for FAP, potentially establishing market dominance.
2. The hereditary nature of FAP ensures a stable, identifiable patient population.
3. The combination of Orphan Drug status and Fast Track designation provides enhanced market protection and accelerated development pathways.

This progress could catalyze broader interest in Biodexa's pipeline and technology platform, particularly in rare disease applications where their drug delivery expertise could be leveraged for other indications.