Health Canada Approves Expansion of Biodexa's Serenta Phase 3 Trial for Familial Adenomatous Polyposis
Key Insights
Health Canada has approved the expansion of Biodexa's registrational Phase 3 Serenta trial for familial adenomatous polyposis (search) (FAP) into Canada, adding three to four new clinical centers.
The double-blind, placebo-controlled trial is currently active across 29 centers in the US and five European countries, with 73 of a planned 168 patients enrolled to date.
eRapa, an oral mTOR (search) inhibitor formulation of rapamycin, targets mTOR overexpression in FAP polyps and has received Orphan Drug Designation in the US.
Biodexa Pharmaceuticals PLC has received approval from Health Canada to expand its registrational Phase 3 trial of eRapa, branded Serenta, into Canada for the treatment of familial adenomatous polyposis (search) (FAP). The expansion is expected to add three or four new clinical centers to the ongoing study, which is already operational across 29 active recruiting sites in the United States and five European countries.
The Serenta trial is a double-blind, placebo-controlled study designed to enroll 168 patients randomized 2:1 to receive either eRapa or placebo. As of the announcement date, 73 patients have been enrolled. The Phase 3 program is supported by a $20 million grant from the Cancer Prevention and Research Institute of Texas (search) (CPRIT).
"We have seen an acceleration in enrolment in the last few months as the European centers have opened. The addition of Canada underpins our enrolment goals," said Stephen Stamp, Chief Executive Officer of Biodexa. "Serenta has been received positively by clinicians and FAP patients for whom there is currently no approved therapeutic option and life-altering surgery remains standard of care."
eRapa and the mTOR Rationale
eRapa is a proprietary oral capsule formulation of rapamycin, also known as sirolimus, which functions as an mTOR (search) (mammalian Target Of Rapamycin) inhibitor. mTOR plays a significant role in the signaling pathway that regulates cellular metabolism, growth, and proliferation and is activated during tumorigenesis. Critically, mTOR has been shown to be over-expressed in FAP polyps, providing the mechanistic rationale for deploying a potent mTOR inhibitor in this patient population.
Data from an open-label Phase 2 trial of eRapa were presented at Digestive Disease Week and InSIGHT 2024 in May and June 2024, respectively. Based on those findings, Biodexa initiated the current registrational Phase 3 trial. eRapa has already received Orphan Drug Designation in the United States, and the company has indicated plans to seek similar designation in Europe.
Familial Adenomatous Polyposis: Unmet Medical Need
FAP is characterized by a proliferation of polyps in the colon and/or rectum, typically manifesting in the mid-teenage years. The condition carries a significant hereditary component, with a reported incidence of one in 5,000 to 10,000 in the US and one in 11,300 to 37,600 in Europe. If left untreated, FAP typically progresses to cancer of the colon and/or rectum.
Currently, no approved pharmacological therapy exists for FAP. The standard of care consists of active surveillance and surgical resection of the colon and/or rectum — interventions that are life-altering for patients, many of whom are young at the time of diagnosis.
Biodexa's Broader Pipeline
Beyond eRapa, Biodexa is advancing several other programs. MTX240 (search), a molecular glue designed to bring PDE3a (search) and SLFN12 (search) intracellular proteins into close proximity, is under development for gastrointestinal stromal tumors (GIST). The compound drives RNase-mediated apoptosis in GIST cells through a mechanism independent of KIT signaling. Tolimidone, an orally delivered Lyn kinase (search) inhibitor, is being developed for type 1 diabetes and has demonstrated glycemic control via insulin sensitization in animal models, with potential to become a first-in-class blood glucose modulating agent. eRapa is also under investigation for non-muscle invasive bladder cancer.
Biodexa Pharmaceuticals PLC is headquartered in Cardiff, UK, and trades on Nasdaq under the ticker BDRX.