Biodexa Launches EU Trial for First Pill to Prevent Genetic Cancer

CARDIFF, UK – November 24, 2025 – Biodexa Pharmaceuticals has activated its first European clinical trial site at the University of Bonn, Germany, marking a critical step forward in the development of a potential first-in-class oral therapy for Familial Adenomatous Polyposis (FAP). The registrational Phase 3 Serenta trial will evaluate eRapa, a novel formulation of the drug rapamycin, as a non-surgical intervention to halt disease progression in a patient population that currently has no approved pharmaceutical alternatives.

The trial's expansion into Europe, following its U.S. launch in August 2025, represents a significant milestone for FAP patients who face a near-100% lifetime risk of developing colorectal cancer. The activation comes shortly after Biodexa received approval for its Clinical Trial Application from the European Medicines Agency (EMA), signaling a clear regulatory path forward on the continent.

“Coming shortly after approval of our Clinical Trial Application by the European Medicines Agency, opening of the first site in Europe for our Serenta trial in FAP is another major milestone,” said Gary Shangold MD, Chief Medical Officer of Biodexa, in a statement. He credited the support of collaborators Emtora Biosciences, the European contract research organization (CRO) Precision for Medicine, and a substantial grant from the Cancer Prevention and Research Institute of Texas (CPRIT).

The Burden of a Near-Certain Cancer

Familial Adenomatous Polyposis is a rare, inherited genetic disorder that fundamentally alters a patient's life from adolescence. The condition is characterized by the relentless growth of hundreds to thousands of polyps in the colon and rectum, typically beginning in a patient's mid-teens. If left untreated, the progression from benign polyps to malignant colorectal cancer is considered almost inevitable, often by age 50.

Currently, the standard of care is a brutal regimen of constant surveillance and prophylactic surgery. Patients undergo frequent endoscopies to monitor polyp growth, ultimately facing the necessity of a colectomy (removal of the colon) and potentially a proctectomy (removal of the rectum) to prevent cancer. While life-saving, these sequential surgeries carry significant lifelong consequences, impacting digestive function, fertility, and overall quality of life. The unmet medical need for a less invasive, effective therapy is profound, affecting an estimated one in 5,000 to 10,000 people in the U.S. and one in 11,300 to 37,600 in Europe.

A New Use for a Known Drug

The scientific rationale for the Serenta trial is rooted in repurposing a well-understood biological mechanism. The drug at the center of the study, eRapa, is a proprietary oral formulation of rapamycin (also known as sirolimus). Rapamycin is a potent inhibitor of the mammalian Target of Rapamycin (mTOR) pathway, a critical signaling cascade that regulates cell metabolism, growth, and proliferation. Crucially, research has shown that the mTOR pathway is over-expressed in FAP polyps, making it a prime therapeutic target.

While rapamycin is already FDA-approved for preventing organ rejection in renal transplants, its standard formulations suffer from poor bioavailability, variable absorption, and potential toxicity. Biodexa’s eRapa is engineered to overcome these limitations. It uses nanotechnology and pH-sensitive polymers to create a capsule designed for controlled absorption, aiming to deliver the drug more effectively while improving its safety profile.

The decision to advance eRapa into a pivotal Phase 3 study is backed by compelling Phase 2 data presented at major medical conferences in 2024. In a 30-patient open-label study (NCT04230499), eRapa demonstrated a statistically significant 24% mean reduction in total polyp burden after six months. More importantly, data from a specific cohort receiving the dose now used in the Phase 3 trial (0.5 mg daily, every other week) showed a remarkable 89% non-progression rate and a 29% median reduction in polyp burden after 12 months. These strong results provide the foundational evidence for the larger, placebo-controlled Serenta trial.

A High-Stakes Bet for a Micro-Cap Biotech

For Biodexa, a UK-based micro-cap biopharmaceutical company, the Serenta trial is a defining endeavor. With a market capitalization hovering under $3 million and a history of net losses typical of pre-revenue biotechs, the company's future is heavily tied to the success of its late-stage pipeline. The estimated $7.3 billion addressable market for FAP in the U.S. and Europe presents a substantial commercial opportunity, amplified by the drug's Orphan Drug Designation in Europe and Fast Track status in the U.S., which offer market exclusivity and expedited reviews.

The financial burden of a global Phase 3 trial, however, is immense. This is where a critical partnership comes into play. The trial is substantially funded by a $20 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT), originally awarded to eRapa's developer, Emtora Biosciences. Biodexa, which licensed the drug from Emtora in April 2024, has fulfilled its matching fund requirements, unlocking the full grant. This non-dilutive funding allows Biodexa to pursue this late-stage program without placing an existential financial strain on the company, a crucial advantage for a small firm in a capital-intensive industry.

Navigating a Developing Competitive Landscape

While Biodexa highlights that no approved non-surgical therapies exist for FAP, eRapa is not the only drug vying to fill this void. The competitive landscape includes several other investigational therapies. Cancer Prevention Pharmaceuticals' combination of eflornithine and sulindac, for instance, failed to meet its primary endpoint in a Phase 3 trial but is still being pursued for regulatory approval based on positive signals in a patient subgroup.

Other notable competitors include Recursion Pharmaceuticals' REC-4881, a MEK inhibitor that showed a promising 43% median polyp reduction in early Phase 2 data, and S.L.A. Pharma's ALFA, an oral formulation of an omega-3 fatty acid also in Phase 3 testing. This developing pipeline underscores the intense scientific and commercial interest in finding a medical solution for FAP.

The Serenta trial (NCT06950385) is designed to provide a definitive answer on eRapa's efficacy. The randomized, double-blind, placebo-controlled study aims to enroll 168 patients across approximately 30 sites in the U.S. and Europe. Patients will be randomized on a 2:1 basis to receive either eRapa or a placebo, with the primary goal of determining if the drug can prevent or delay disease progression. Following the activation of the University of Bonn, Biodexa plans to bring nine additional European sites online in the Netherlands, Spain, Denmark, and Italy over the next few months, rapidly expanding the trial's global footprint.