Biodexa Secures Exclusive License for Phase 3 Drug eRapa™ to Treat FAP

On April 26, 2024, Biodexa Pharmaceuticals PLC made an important announcement regarding the acquisition of eRapa, a promising treatment for Familial Adenomatous Polyposis (FAP). FAP is a precancerous condition characterized by numerous polyps in the colon and rectum, affecting an estimated 100,000 individuals in the U.S. and Europe. This condition often leads to the surgical removal of the colon or rectum unless an effective treatment is available. Currently, the standard care involves regular surveillance and eventual surgery, as there are no approved pharmacological treatments.

The agreement with Rapamycin Holdings Inc. (doing business as Emtora Biosciences) grants Biodexa the exclusive worldwide rights to develop, manufacture, and commercialize eRapa. This drug is poised to enter a Phase 3 clinical trial for FAP, supported by a generous $17 million non-dilutive grant from the Cancer Prevention and Research Institute of Texas (CPRIT). Stephen Stamp, CEO and CFO of Biodexa, emphasized the significant enhancement this brings to their oncology pipeline, noting the potential for eRapa to delay or prevent the need for invasive surgery in FAP patients.

eRapa stands out due to its formulation of rapamycin, an mTOR inhibitor commonly used to prevent organ rejection in kidney transplants. Leveraging nanotechnology and pH-sensitive polymers, eRapa aims to overcome the usual issues associated with rapamycin, such as poor bioavailability and variable pharmacokinetics. The drug's promising potential extends beyond FAP to other indications, including bladder and prostate cancers. For FAP, several genetic factors contribute to its prevalence, occurring in roughly 1 in 5,000 to 10,000 individuals in the U.S. and varying figures in Europe. eRapa has already received Orphan Designation in the U.S., with similar plans in Europe.

Stephen Dufilho, Executive Chairman of Emtora, highlighted the decade-long journey of eRapa’s development, beginning at the University of Texas and bolstered by grants from CPRIT. Emtora is on the cusp of completing a Phase 2 study with positive preliminary results to be presented soon. This open-label, multi-center study focused on 30 patients with confirmed FAP, prioritizing the safety, tolerability, and reduction of polyp burden over six months.

Looking beyond FAP, eRapa is being tested in Non-Muscle Invasive Bladder Cancer (NMIBC), characterized by tumors in the bladder lining. This Phase 2 study, supported by a $3 million grant from the National Cancer Institute, aims to evaluate the drug's efficacy and safety, with outcomes expected by late 2025. NMIBC has a significant prevalence rate, particularly among men.

Further opportunities for eRapa include exploring its application in other rare gastrointestinal diseases. Biodexa plans to leverage low-cost trials to explore these indications and gather initial proof-of-concept data.