Pharmaceuticals Company Announces Positive Results of Familial Adenomatous Polyposis Treatment
Biodexa Pharmaceuticals: Promising Advances in the Treatment of Precancerous Polyps with eRapa.
Disclaimer: This article is intended for informational purposes only and should not be considered medical advice. The information provided is based on available data as of the publication date. Readers should consult healthcare professionals for specific medical advice or treatment. Neither the author nor the publisher takes responsibility for any consequences arising from the use of this information.
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Biodexa Pharmaceuticals PLC (NASDAQ:BDRX) recently released additional positive results from a phase 2 clinical trial of eRapaâ„¢, its investigational drug designed to treat Familial Adenomatous Polyposis (FAP). FAP is a predominantly inherited condition that increases the likelihood of developing colorectal cancer. The encouraging 12-month data were presented at the 2024 InSIGHT biannual meeting held in Barcelona, shedding light on the potential future of FAP treatment.
Understanding Familial Adenomatous Polyposis (FAP)
Familial Adenomatous Polyposis is a genetic disorder characterized by the growth of hundreds to thousands of precancerous polyps throughout the gastrointestinal tract. Typically manifesting during the patient's teenage years, FAP necessitates rigorous surveillance and often requires surgical resection of the colon and/or rectum. Unfortunately, there are currently no approved therapeutic options to manage this condition. Consequently, individuals with FAP face the inevitability of having their entire colon and/or rectum removed, which usually results in lifelong use of a colostomy bag. If left unmanaged, FAP carries a 100% risk of progressing to colorectal cancer. Biodexa Pharmaceuticals aims to revolutionize the treatment landscape for FAP patients with eRapa, which could potentially become the first therapeutic option for this precancerous condition. The results from the 12-month phase 2 clinical trial of eRapa have been promising. Key findings include:
An overall 17% median reduction in polyp burden.
A non-progression rate of 75%.
More notably, among patients in Cohort 2 (who received daily treatment on alternate weeks), 89% were classified as non-progressors at the 12-month mark, with a median reduction in polyp burden of 29%. These results suggest that eRapa may decrease the need for surgical interventions, thereby enhancing the quality of life for FAP patients by reducing the frequency of surgeries. The open-label phase 2 study involved 30 adult participants with FAP across seven centers of excellence in the United States. The median age of participants was 43 years, encompassing individuals with either an intact colon or one partially resected, along with at least ten noncancerous tumors in the rectal remnant. Patients were divided into three dosing cohorts: every other day, daily every other week, and daily.
The primary endpoints of the study were safety and tolerability of eRapa, along with the percentage change from baseline in polyp burden at six months. However, the trial extended to 12 months, resulting in the latest data. eRapa, a proprietary oral tablet formulation of rapamycin (also known as sirolimus), functions by inhibiting the mTOR (mammalian Target Of Rapamycin) protein—a protein linked to cancer progression when overactive.
The Findings
Stephen Stamp, CEO of Biodexa, emphasized the significance of the phase 2 results, stating, "The promising phase 2 results, if confirmed in a registrational phase 3 study, may delay or potentially obviate the need for resection of the colon and/or rectum in FAP patients." He further elaborated that the accumulating data from both six-month and 12-month studies, combined with the apparent tolerability of eRapa, suggest that long-term use may be feasible. This could potentially forestall surgical resection and substantially improve the quality of life for FAP patients, which impacts approximately 40,000 individuals in the United States and up to 60,000 in Europe.
eRapa's active ingredient, rapamycin, is well-known for its role in slowing down the mTOR protein. mTOR is crucial for cell growth and proliferation, and its overactivity has been linked to various cancers, including colorectal cancer. By inhibiting this protein, eRapa may help control the growth of precancerous polyps in FAP patients. The path forward for eRapa involves further clinical validation in a phase 3 registrational study. If the results from phase 2 are replicated, eRapa could be a groundbreaking therapy for FAP patients, offering them a viable alternative to the current standard of care, which heavily relies on surgery. The release of the 12-month phase 2 trial results for eRapa marks a milestone in the quest to find effective treatments for Familial Adenomatous Polyposis. Biodexa Pharmaceuticals' innovative approach could potentially transform the treatment paradigm for this devastating condition, providing hope for improved patient outcomes and quality of life.
As always, ongoing research and clinical trials are essential to confirm these promising early results. Should eRapa prove successful in subsequent studies, it may represent a major advancement in the fight against colorectal cancer for those with FAP.
Disclaimer: This article is intended for informational purposes only and should not be considered medical advice. The information provided is based on available data as of the publication date. Readers should consult healthcare professionals for specific medical advice or treatment. Neither the author nor the publisher takes responsibility for any consequences arising from the use of this information.
Real-time information is available daily at https://stockregion.net