Biodexa's eRapa Receives US FDA Fast Track Designation for Familial Adenomatous Polyposis

Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) has received a significant boost with the US Food and Drug Administration (FDA) granting Fast Track Designation to its proprietary oral tablet formulation of rapamycin, eRapa™, for the treatment of Familial Adenomatous Polyposis (FAP). This designation is a testament to the drug's potential to address an unmet medical need and offers Biodexa several advantages in its development and regulatory pathway.

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 delay or even obviate the need for surgical resection of the colon and/or rectum in FAP patients.



The Phase 2 study of eRapa™ in FAP, partially supported by a $3 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT), demonstrated promising results at 12 months. Overall, 75% of patients were deemed non-progressors, with a median reduction in polyp burden of 17%. In Cohort 2, 89% of patients were non-progressors, with a median reduction in polyp burden of 29%. These results suggest that eRapa™ may be a valuable treatment option for FAP patients, potentially delaying or even preventing the need for surgery.

The Fast Track Designation is often accompanied by Orphan Drug Designation, which provides additional benefits such as clinical trial design assistance, FDA staff support, and market exclusivity if the drug is approved. In the case of eRapa™, it has already received Orphan Drug Designation in the US and plans to seek similar designation in Europe. This designation can help Biodexa navigate the regulatory pathway more efficiently and potentially reduce the time it takes to bring eRapa™ to market.



The Phase 3 registrational study of eRapa™ in FAP is planned to be a double-blind placebo-controlled design recruiting approximately 140 high-risk patients diagnosed with germline or phenotypic FAP. The primary clinical endpoint is likely to be the first progression-free survival event, which will include major surgery (or referral thereto), polypectomy for advanced neoplasia, advancement of Spigelman stage, diagnosis of high-grade dysplasia or cancer, and death from any cause. A $17 million grant from CPRIT will support this study.

In summary, the US FDA's Fast Track Designation for eRapa™ in Familial Adenomatous Polyposis (FAP) significantly impacts Biodexa's regulatory pathway and timelines. This designation allows for an expedited review process, offers market exclusivity, and provides additional support for clinical trials. The promising results from the Phase 2 study and the potential for eRapa™ to address an unmet medical need create a substantial market opportunity for Biodexa. With its unique formulation and potential advantages, eRapa™ could become a valuable treatment option for FAP patients and establish a strong market position for Biodexa.