Biodexa Pharmaceuticals Plc announced that 12 month results of a Phase 2 clinical trial of eRapa? in Familial Adenomatous Polyposis (NCT04230499) were reported in an oral presentation at the prestigious 2024 InSIGHT bi-annual meeting in Barcelona by Carol Burke, M.D., a specialist gastroenterologist at the Cleveland Clinic and a leading authority in FAP, a mostly genetic disease of precancerous polyps for which the only current remedy is surgical removal of the colon and/or rectum. The Phase 2 study was partially supported by $3 million in grant funding from the Cancer Prevention and Research Institute of Texas (CPRIT) under product development awards DP22053 and DP190069.

The Phase 3 study of eRapa in FAP is supported by a $17 million grant from CPRIT. Dr. Burke is the Principal Investigator for the both the Phase 2 study and the upcoming Phase 3 registrational study. The open-label study was conducted in seven U.S. centers of excellence in 30 adult patients with median age of 43 years with intact colon (n=6) or post-colectomy and ileo-rectal anastomosis and at least 10 adenomas in the rectal remanent (n=24).

Patients were sequentially enrolled into three dosing cohorts of 10 patients each for a 12-month treatment period: 0.5mg every other day (Cohort 1), 0.5mg daily every other week (Cohort 2), and 0.5mg daily (Cohort 3). Upper and lower endoscopic surveillance occurred at baseline and after six months. Although the primary endpoints were safety and tolerability of eRapa and percentage change from baseline in polyp burden at six months, as measured by the aggregate of all polyp diameters, patients continued to receive treatment and monitoring for 12 months (secondary endpoint).

Results of the Phase 2 study at 12 months Overall, 21 of 28 (75%) patients were deemed to be non-progressors at 12 months with a median reduction in polyp burden of 17%. In Cohort 2, eight of nine (89)% of patients were deemed non-progressors at 12 months with a median reduction in polyp burden of 9%. Over the course of 12 months, there were four related Grade 3 or higher and one related Serious Adverse Event reported during the trial and 95% compliance rate at 12 months.

One patient was removed from the trial due to non-compliance. The Phase 3 registrational study 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. eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus. Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor.

mTOR has been shown to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorgenesis1. Rapamycin is approved in the US for organ rejection in renal transplantation as Rapamune®(Pfizer). Through the use of nanotechnology and pH sensitive polymers, eRapa is designed to address the poor bioavailability, variable pharmacokinetics and toxicity generally associated with the currently available forms of rapamycin.

eRapa is protected by a number of issued patents which extend through 2035, with other pending applications potentially providing further protection beyond 2035.