Biodexa Reports More Positive 12-Month Phase 2 Results for eRapa in GI Precancerous Polyps Treatment

Biodexa Pharmaceuticals PLC, a biopharmaceutical company based in Cardiff, UK, has been making notable advancements with its drug, eRapa™, aimed at treating Familial Adenomatous Polyposis (FAP). This condition, often hereditary, significantly increases the likelihood of developing colorectal cancer. Recent positive 12-month data from a phase 2 clinical trial were showcased at the 2024 InSIGHT meeting in Barcelona.

FAP is characterized by the growth of hundreds or thousands of precancerous polyps throughout the gastrointestinal tract. Currently, no approved therapeutic options exist for FAP patients, who typically rely on active surveillance and surgical removal of the colon and/or rectum. This often results in patients needing to use a colostomy bag for the rest of their lives. Without treatment, there is a certainty of developing colorectal cancer.

Biodexa's eRapa offers a promising new approach to treating FAP. The company’s data indicates that eRapa could become the first therapeutic option for this precancerous condition. Findings from a 12-month phase 2 clinical trial showed a 17% median decrease in overall polyp burden and a 75% non-progression rate. Among patients treated daily on alternate weeks (Cohort 2), 89% were non-progressors at 12 months, with a median reduction in polyp burden of 29%. These results suggest that eRapa could significantly reduce the need for surgeries, thereby improving the quality of life for FAP patients.

Stephen Stamp, CEO of Biodexa, expressed optimism about the phase 2 results, which, if confirmed in a phase 3 study, could delay or even eliminate the need for colon and/or rectum resection in FAP patients. The data indicates that longer-term use of eRapa may be possible, with the potential to prevent resection and greatly enhance the quality of life for patients.

eRapa is an oral tablet formulation of rapamycin, also known as sirolimus, which inhibits the mTOR protein, a protein linked to cancer when present in excess. The phase 2 open-label study, conducted at seven U.S. centers of excellence, involved 30 adults with FAP. The median age of participants was 43, with either an intact or partially removed colon and at least ten noncancerous tumors in the rectal remnant. Participants were divided into three dosing cohorts: every other day, daily every other week, and daily. The primary endpoints were safety and tolerability of eRapa, and the percentage change in polyp burden at six months, with treatment and monitoring extending to 12 months.

Biodexa is planning a phase 3 trial with a double-blind placebo-controlled design, recruiting around 140 high-risk patients diagnosed with germline or phenotypic FAP. The preferred dosage for this trial will likely be the regimen used in Cohort 2 of the phase 2 trial.

Carol Burke, M.D., a gastroenterologist at the Cleveland Clinic and a leading expert in FAP, presented the phase 2 results at InSIGHT. Dr. Burke is also the Principal Investigator for both the phase 2 and the upcoming phase 3 trials. The phase 2 study received partial funding from the Cancer Prevention and Research Institute of Texas (CPRIT), which is also providing a $17 million grant for the phase 3 trial.

Though FAP is rare, it affects tens of thousands of people who currently face limited treatment options. Biodexa's eRapa could provide a much-needed therapeutic alternative, as indicated by the promising phase 2 trial results. The upcoming phase 3 study offers hope for even more substantial progress in treating this condition.