Global Program Offers New Hope for Rare Genetic Cancer Syndrome

CHARLOTTE, NC & CARDIFF, UK – March 31, 2026 – For individuals diagnosed with Familial Adenomatous Polyposis (FAP), a rare genetic condition with a near-certain progression to cancer, the path forward has historically been limited and life-altering. Today, a new global initiative offers a ray of hope, providing access to an investigational therapy outside the confines of a clinical trial.

Tanner Pharma Group, a specialist in global medicine access, and Biodexa Pharmaceuticals PLC, a clinical-stage biopharmaceutical company, have launched a strategic partnership to create a global Early Access Program (EAP) for eRapa. This program allows clinicians worldwide to request the investigational drug for patients with FAP in countries where it is not yet commercially available, marking a significant step in addressing a profound unmet medical need.

A Lifeline Beyond Surgery

Familial Adenomatous Polyposis is a devastating inherited disorder affecting an estimated 1 in 5,000 to 1 in 18,000 people. It is characterized by the relentless growth of hundreds to thousands of polyps in the colon and rectum, typically beginning during a person's teenage years. If left untreated, the lifetime risk of these polyps developing into colorectal cancer is nearly 100%, often before the age of 40.

To date, there are no approved pharmaceutical treatments for FAP. The standard of care is rigorous surveillance followed by a prophylactic colectomy—the complete surgical removal of the colon—and sometimes the rectum as well. While this major surgery drastically reduces the risk of cancer, it comes with significant lifelong consequences, including altered bowel function, potential complications, and a diminished quality of life. Furthermore, even after surgery, patients require continued monitoring for polyp growth in remaining gastrointestinal tissue and for other FAP-related cancers.

The search for a non-surgical intervention has been a long-standing goal for researchers and a desperate hope for patients and their families. While some drugs, like the COX-2 inhibitors sulindac and celecoxib, have been used off-label to help manage polyp burden, their efficacy is limited and side effects can be a concern. This new Early Access Program for eRapa represents the first time a targeted investigational therapy is being made widely available to this patient population for therapeutic use.

Unpacking the Science of eRapa

The scientific rationale behind eRapa is rooted in the specific biology of FAP. eRapa is a proprietary oral capsule formulation of rapamycin (also known as sirolimus), a drug that inhibits a key cellular regulator called mTOR (mammalian Target Of Rapamycin). The mTOR pathway plays a crucial role in cell metabolism, growth, and proliferation. In FAP, this pathway has been shown to be over-expressed in the polyps, driving their uncontrolled growth.

By inhibiting mTOR, eRapa aims to directly interrupt the biological engine that fuels polyp development and progression. Biodexa's unique formulation was designed to improve upon the bioavailability and pharmacokinetic profile of existing rapamycin products, potentially enhancing its therapeutic effect while managing its safety profile.

The promise of this approach has been borne out in recent clinical findings. In June 2024, Biodexa announced positive 12-month results from a Phase 2 trial. The study demonstrated that eRapa was generally safe and well-tolerated, achieving an overall non-progression rate of 75% among patients. A specific cohort receiving what may be an optimal dose showed even more striking results: an 89% non-progression rate and a median reduction in overall polyp burden of 29%.

Building on this success, Biodexa has initiated a pivotal Phase 3 registrational trial, named Serenta, to definitively evaluate the drug's safety and efficacy. The global trial aims to enroll 168 patients and is supported by a significant $17 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT). The therapy has also received both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration, underscoring its potential to address a serious condition lacking adequate treatment options.

Navigating the Path to Early Access

The partnership leverages the specialized expertise of Tanner Pharma Group to navigate the complex regulatory and logistical landscape of providing pre-approval access to medicines. Early Access Programs, also known as compassionate use or named patient programs, are highly regulated pathways designed for patients with life-threatening conditions who have exhausted all available treatment options and are unable to participate in a clinical trial.

Through this program, a treating physician can request eRapa on behalf of a specific, eligible patient. Tanner Pharma will manage the process, ensuring compliance with local laws and regulations in each country. Access is not guaranteed and depends on several factors, including the patient's medical eligibility, the regulatory framework of the host country, and the availability of government or private funding to cover the cost of the treatment.

It is a critical distinction that these programs are for treatment, not research, and are not a substitute for the rigorous scientific evaluation of a clinical trial. They exist to bridge the gap between the completion of clinical development and commercial approval for patients with no other recourse. This initiative carefully balances the ethical imperative to provide compassionate care with the need to maintain the integrity of the ongoing Phase 3 trial, which remains the definitive pathway to regulatory approval.

A New Blueprint for Rare Disease Drug Development

Beyond the immediate benefit to patients, this collaboration represents an innovative and increasingly vital strategy in modern drug development, particularly for rare diseases. A key component of the EAP is Biodexa's plan to generate Real World Data (RWD) from the program's participants. This data, collected during routine clinical care, provides invaluable insights into how a drug performs in a broader, more diverse patient population than is typically found in a controlled clinical trial.

RWD can supplement the evidence gathered in registrational trials, offering a more complete picture of a drug’s long-term safety, effectiveness, and impact on patient quality of life. For regulators and payers, this information can be crucial in assessing a new therapy's overall value and place in the treatment landscape. For a company like Biodexa, it helps build a robust evidence package that can support regulatory submissions and facilitate market access upon approval.

This model—partnering with an access specialist to provide an investigational drug early while systematically gathering RWD—is becoming a new blueprint for biopharmaceutical companies. It allows them to meet an urgent patient need, build awareness and experience with their product among clinicians, and de-risk the path to commercialization. In the field of rare diseases like FAP, where patient populations are small and the need for new options is immense, such forward-thinking strategies are not just good business; they are an essential part of advancing medicine.