Biodexa Announces Additional Positive Results Of Phase 2 Trial Of eRapa in Treatment Of Precancerous Polyps in the GI Tract - Now 12-Month Data
Biodexa Pharmaceuticals PLC (NASDAQ:BDRX) has announced positive 12-month data from its Phase 2 clinical trial of eRapa™ for the treatment of Familial Adenomatous Polyposis (FAP), a condition that significantly increases the risk of colorectal cancer. Presented at the 2024 InSIGHT biannual meeting, results indicate a 17% median reduction in polyp burden and a non-progression rate of 75%. Notably, 89% of patients in Cohort 2 (treated daily on alternate weeks) were deemed non-progressors, with a 29% median reduction in polyp burden.
FAP patients currently undergo continuous surveillance and surgeries, often resulting in complete resection of the colon or rectum. Biodexa aims for eRapa to be the first therapeutic alternative, potentially reducing the need for surgical interventions and improving patients' quality of life. The Phase 3 trial, planned to start soon, will involve 140 high-risk FAP patients and be double-blind placebo-controlled. This trial is partially funded by a $17 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT).
- Positive 12-month data from Phase 2 trial of eRapa show a 17% median decrease in polyp burden and a 75% non-progression rate.
- Cohort 2 of the trial indicated 89% of patients were non-progressors with a 29% median reduction in polyp burden.
- Biodexa plans a Phase 3 trial, supported by a $17 million grant from CPRIT.
- None.
Insights
The latest 12-month data from Biodexa's phase 2 trial of eRapa in treating Familial Adenomatous Polyposis (FAP) signals promising advancements in tackling this rare and devastating pre-cancerous condition. The trial data indicates a 17% median decrease in overall polyp burden and a 75% non-progression rate, with particularly strong results from cohort 2 where 89% were non-progressors and a 29% median reduction in polyp burden. Given that FAP often leads to mandatory colon resection, these results could represent a substantial improvement in patient quality of life.
eRapa's mechanism, which involves slowing the mTOR (mammalian Target Of Rapamycin) protein, aligns with emerging scientific understanding of mTOR's role in cancer progression. The promising efficacy coupled with the drug's tolerability over 12 months suggests it could potentially delay or even avoid the need for surgical resection in FAP patients. If these results are confirmed in a phase 3 trial, eRapa could become the first therapeutic option for treating FAP, significantly altering the treatment landscape.
The transition from phase 2 to phase 3 is a critical juncture for any drug and the positive results from Biodexa’s phase 2 trial of eRapa are encouraging. The study's double-blind placebo-controlled design for phase 3, involving around 140 high-risk patients, will provide robust data necessary to confirm eRapa’s efficacy and safety profile. The $17 million grant from the Cancer Prevention and Research Institute of Texas for the phase 3 trial also underscores the medical community’s belief in the potential of eRapa.
From a clinical trial design perspective, the choice of daily every-other-week dosing for cohort 2, which showed the most promising results, addresses both efficacy and patient compliance concerns. This dosing regimen will likely be critical to the success of the phase 3 trial. Should these findings hold up, eRapa might not only reduce the need for invasive surgeries but also set a new standard in the management of FAP.
The market implications of Biodexa’s successful phase 2 trial results can be significant. Currently, no approved therapeutic options exist for FAP, meaning that eRapa could capture a unique market niche if it progresses to approval. With an estimated 100,000 people worldwide affected by FAP and no existing treatments, the commercial potential is notable. Moreover, the potential for eRapa to improve quality of life by reducing the need for surgical interventions can position the drug as a highly sought-after option in both the U.S. and Europe, home to the majority of FAP patients.
The $17 million grant for the phase 3 trial also mitigates financial risks and highlights institutional support for eRapa’s development. Investors should monitor the progress of the phase 3 trial closely, as positive results could lead to rapid market adoption and significant revenue streams for Biodexa. However, it is important to remain cautious until phase 3 results are available, as clinical trials can be unpredictable.
CARDIFF, UK / ACCESSWIRE / July 11, 2024 / Biodexa Pharmaceuticals PLC (NASDAQ:BDRX), an acquisition-focused clinical-stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, is making good progress with eRapa™, its drug to treat Familial Adenomatous Polyposis (FAP).
FAP is a mostly inherited condition that puts people at a much greater risk of developing colorectal cancer. Positive 12-month data from a phase 2 clinical trial were recently presented at the 2024 InSIGHT biannual meeting in Barcelona.
With FAP, hundreds or thousands of precancerous polyps grow throughout the gastrointestinal tract. There is no approved therapeutic option for treating FAP patients - for whom active surveillance and surgical resection of the colon and/or rectum remain the standard of care. People with FAP, which usually appears in the patient's mid-teens, end up eventually having their entire colon and/or rectum resected and having to carry a colostomy bag for the remainder of their lives . If left untreated, there is a
A Better Way Of Treating FAP
While multiple screenings and surgeries are a way of life for the more than 100,000 people worldwide who suffer from this condition, it doesn't have to remain that way.
Biodexa believes eRapa could be the first therapeutic option to treat this precancerous condition, and its data so far backs up that assessment. Results of a 12-month phase 2 clinical trial of eRapa demonstrated an overall
"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," said Stephen Stamp, CEO of Biodexa. "The six-month, and now 12-month, data together with its apparent tolerability suggest longer-term use of eRapa may be possible, with the potential to forestall resection and substantially increase the quality of life of patients with this devastating precancerous condition impacting up to 40,000 patients in the U.S. and up to 60,000 in Europe."
eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus, which slows down the mTOR (mammalian Target Of Rapamycin) protein. Too much mTOR has been linked to cancer.
Stopping It In Its Tracks
The phase 2 open-label study of 30 adults with FAP was conducted in seven U.S. centers of excellence. The median age of the trial participants was 43 years, with either an intact colon or one with a portion removed and at least ten noncancerous tumors in the rectal remnant. Patients were enrolled in three dosing cohorts, including every other day, daily every other week and daily. Although the primary endpoints were safety and tolerability of eRapa and percentage change from baseline in polyp burden at six months, patients continued to receive treatment and monitoring for 12 months, leading to the new data.
Biodexa said the dosing given to cohort 2 - daily every other week - will likely be the preferred dosage regime for its phase 3 trial, which the company is gearing up to launch soon. That study is planned to be a double-blind placebo-controlled design recruiting approximately 140 high-risk patients diagnosed with germline or phenotypic FAP.
Biodexa's phase 2 results were presented at InSIGHT by Carol Burke, M.D., a specialist gastroenterologist at the Cleveland Clinic and a leading authority in FAP. Burke is the Principal Investigator for both the phase 2 study and the upcoming phase 3 study. The phase 2 trial was partially supported by
FAP may be rare, but for the tens of thousands of people suffering from this precancerous condition, there's got to be a better treatment. Biodexa believes it has that with eRapa. The phase 2 trial results seem to lean that way, and with a phase 3 study about to kick off, there may be hope on the horizon for FAP patients.
Contact:
Stephen Stamp, CEO, CFO
ir@biodexapharma.com
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SOURCE: Biodexa Pharmaceuticals PLC
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