The Longview: Fireside Chat with Biodexa CEO Stephen Stamp
CARDIFF, UK / ACCESS Newswire / September 23, 2026 /Biotech in the News: The Longview
1. What is Familial Adenomatous Polyposis?
FAP is a mostly inherited rare disease caused by mutation of the APC gene and is characterized by hundreds, or even thousands, of polyps in the GI tract. Not all countries maintain registries but prevalence is somewhere between one in 5,000 to 10,000 in the US and one in 11,000 to 37,000 in Europe. Symptoms include intestinal blockages, rectal bleeding, diarrhea and abdominal pain and, if untreated, FAP almost always eventually leads to colorectal cancer.
2. What attracted you to the eRapa opportunity?
Several factors. Most importantly, unmet need - there are no approved products for the treatment of FAP. Currently, the only option is multiple life-altering surgeries ending with an ostomy bag. Second, the molecule. The active ingredient in eRapa, rapamycin, itself has a 25-year history of safety and the rapalog class of compounds, or mTOR inhibitors, are well characterized inhibitors of cell proliferation and cell cycle progression. Third, the data.
"The active ingredient in eRapa, rapamycin, itself has a 25-year history of safety. "
Our partners and licensors at Emtora Biosciences had compelling Phase 2 data in 30 patients which demonstrated reductions in polyp burden, an indicator of disease progression, over six and 12 months. Fourth, orphan drug designation, or ODD. ODD confers several regulatory advantages including seven and 10 years market exclusivity in the US and Europe, respectively. And last but by no means least, grant funding. The Cancer Prevention and Research Institute of Texas, or CPRIT, awarded a $17 million grant, subsequently increased to $20 million, to support the Phase 3 program for eRapa - a game-changing amount of grant money and it speaks to the potential of the opportunity.
3. Where are you with your program and when might we see more data?
With more than 90 participants enrolled, we are over halfway in our planned recruitment of 168 participants. We have 29 of the leading clinical centers in the US and five EU countries working for us. The trial, which we have branded Serenta, should be fully recruited in the first half of next year. Serenta is event-driven which means it will be
"We are planning a futility analysis after 25 events which...could be early next year. "
unblinded after 75 progression events, as defined in the protocol. We are planning a futility analysis after 25 events which, based on current trends, could be early next year. With only a dozen or so confirmed events to date, it is too early to precisely predict when Serenta will complete - it could be as early as mid-2028 or as late as end of 2029. We think we are probably two plus years ahead of the competition, none of which have started a Phase 3 FAP program according to clinicaltrials.gov.
4. What have been the challenges in the development of eRapa so far?
Both FDA and EMA were consistent in their views that change in polyp burden would not, on its own, be considered a clinically meaningful endpoint for a registrational trial. So we designed a detailed, and somewhat complex, composite endpoint for the definition of disease progression with 15 criteria assigned across three anatomical compartments of the GI tract, those being the duodenum, colon and rectum / sigmoid.
"...we have been able to sign up most of the leading FAP treatment centers and, along with that, most of the leading KOLs "
As with most orphan indications, recruiting patients is challenging. In Serenta, recruitment is even more demanding because the trial is event-driven, and we need to reach progression endpoints within a reasonable timeframe. For that reason, we enrol only higher-risk participants with more extensive disease at baseline, based on minimum eligibility criteria for numbers of polyps. Having said that, being ahead of the competition, we have been able to sign up most of the leading FAP treatment centers and, along with that, most of the leading KOLs. Our Phase 3 protocol does not always conform to historical clinical practice. Getting alignment with some clinicians has been challenging whose standard of care has been to surgically resect GI segments, sometimes prophylactically, on the basis there is no available chemopreventative intervention, and the patients will need surgery eventually - so why take the risk of colorectal cancer? We hope the data from our Phase 3 trial of eRapa will change that mindset and reduce or eliminate the need for these life-altering surgeries.
5. How do you view the competition, all of whom are better capitalized than Biodexa?
Let me first say we are not privy to everything that 's going on in our competitor 's programs, so this is based on what we have seen online and cross-referenced with the literature. And bear in mind, FAP is a lifelong disease so safety, tolerability and convenience are primary considerations. The leading competitors are targeting inhibition of the cell proliferation across several pathways.
One approach seems designed to block ERK pathway activation through MEK inhibition. Because it works upstream of mTOR, it may prove disease modifying but other MEK inhibitors have shown side effects including retinopathy and decreased left ventricular ejection fraction, as well as some transient, less serious issues such as acne-like rash, but all of which may be problematic with chronic administration. We reported one SAE in our Phase 2 trial and one so far in the current Phase 3 trial, both pancreatitis and both resolved without change of drug regimen.
A second approach is designed to inhibit signaling in the wnt / beta catenin pathway, right at the root cause biology of errant cell proliferation. Again, this approach may prove disease modifying in terms of cell proliferation and polyp growth - maybe even more than the MEK inhibitor approach. But addressing the wnt / beta catenin signaling pathway is challenging because that pathway also plays important roles in normal healthy functions such as liver metabolism, lung tissue repair, hair follicle renewal, and osteoblast maturation in bone formation.
"FAP is a lifelong disease so safety, tolerability and convenience are primary considerations. "
Past attempts to develop wnt / beta catenin inhibitors to treat cancer have failed to successfully "thread the therapeutic index needle " - by that I mean broad pathway inhibition could not separate oncogenic signaling in tumors from wnt 's essential roles in normal stem cell turnover, leading to troubling off-target toxicity. Moreover, the latest generation of wnt / beta catenin inhibitors are peptides requiring parenteral administration which is not preferable for chronic therapy and patient compliance. eRapa is an oral capsule with a roughly two-year lead in clinical development.
The good news for long-suffering FAP patients is that there are products in development which could meaningfully alter the course of their disease, short term and/or long term, and delay or obviate the need for life-altering surgeries and attendant co-morbidities.
Click here for more information on Biodexa Pharmaceuticals.
Contact:
Stephen Stamp, CEO
ir@biodexapharma.com
This post contains sponsored content prepared by a third party. This content is for informational purposes only and is not intended to be investing advice.
Important notice, please read:The information and statistical data contained herein may contain forward-looking statements that reflect the company 's intentions, expectations, assumptions, or beliefs concerning future events, including, but not limited to, expectations with respect to FDA and other regulatory bodies approval of new products, technology, and product development milestones, the ability of the company to leverage its product development and negotiate favorable collaborative agreements, the commencement of sales, the size of market opportunities with respect to the company 's product candidates and sufficiency of the company 's cash flow for future liquidity and capital resource needs and other risks identified in the Risk Factor Section of the company 's Annual Report and any subsequent reports filed with the SEC. The forward-looking statements are qualified by important factors that could cause actual results to differ materially from those in the forward-looking statements. In addition, significant fluctuations in quarterly results may occur as a result of varying milestone payments and the timing of costs and expenses related to the company 's research and development programs. Biodexa does not undertake to advise you as to any change in this information.
SOURCE: Biodexa Pharmaceuticals PLC
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