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On Track To Be First Mover In $7Bn Addressable Market With No Current Therapeutic Options

By: Benzinga Staff Writer

CARDIFF, UK / ACCESS Newswire / September 8, 2025 / Biodexa Pharmaceuticals PLC. , a late clinical-stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, has launched its registrational Phase 3 trial of eRapa - its proprietary encapsulated form of rapamycin being developed for the treatment of familial adenomatous polyposis (FAP), a debilitating disease of the lower GI tract.

This post was written and published as a collaboration between the in-house editorial team at Benzinga and Biodexa Pharmaceuticals PLC. with financial support from Biodexa. The two organizations work to ensure that any and all information contained within is true and accurate as of the date hereof to the best of their knowledge and research. This content is for informational purposes only and not intended to be investing advice.

Multiple Key Milestones So Far in 2025

The company just enrolled the first patients in the Phase 3 trial. Earlier this year, its collaboration partner, Rapamycin Holdings, Inc., which does business as Emtora Biosciences, was awarded an additional $3 million grant by the Cancer Prevention & Research Institute of Texas bringing the total non-dilutive funding for the Phase 3 trial to $20 million. Together with the match already paid into escrow by Biodexa, the Phase 3 trial is basically funded. The company also recorded two other important milestones earlier this year: Fast Track status from the U.S. Food and Drug Administration and Orphan Drug designation from the European Commission.

All three milestones put the company in a clear leadership position in advancing a drug that could bring relief to people suffering from this largely genetic disease.

A Rare Disease But A $7Bn Addressable Market

FAP causes hundreds or thousands of precancerous polyps to grow throughout the lower 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 rectum removed. If left untreated, there is a near 100% chance the patient will develop colorectal cancer.

There are reported prevalence's of FAP of one in 5,000 to 10,000 population in the U.S. and one in 11,300 to 37,600 population in Europe. Based on the lowest estimates of prevalence of 1/10,000 and 1/37,600 in the US and Europe, respectively, the adult populations in each territory of approximately 258 million and 358 million and the median annual cost of approved non-biologic orphan drugs in the US of $206,176, the combined US / European addressable market for eRapa in FAP is approximately $7.3Bn.

eRapa Could Have First Mover Advantage

Treating FAP has proven difficult over the years. While Pfizer Inc.'s (PFE) Celecoxib (Celebrex), a Cox-2 inhibitor, was approved to reduce the number of polyps for FAP about eight years ago, it failed a post approval study and the label was pulled. Recursion Pharmaceuticals, Inc. (RXRX) is developing REC-4881 and Tempest Therapeutics Inc (TPST) is developing TPST-1495, both for FAP and both are in Phase 2. Biodexa, with compelling Phase 2 data in hand, is already in Phase 3 and could have first mover advantage in a significant market. Orphan drug designation provides market exclusivity for eRapa of 7 years and 10 years from launch in the U.S. and Europe, respectively.

Phase 3 Trial Fueled By Additional Grant On Back Of Compelling Phase 2 Data

Biodexa's Phase 3 trial is a double-blind, placebo-controlled trial in 168 patients, randomized 2:1 drug to placebo. It is expected that the study will be conducted in approximately 30 clinical sites across the U.S. and Europe. The U.S. component of the study is being managed by LumaBridge, and the European component by Precision for Medicine LLC.

The Phase 3 trial advanced after Biodexa completed a Phase 2 trial that demonstrated a 17% median decrease in overall polyp burden and an overall non-progression rate of 75% at 12 months. In cohort 2, which deployed the dosage regimen selected for the Phase 3 trial, the median decrease in polyp burden was 29% with a non-progression rate of 89% at 12 months. Following that study Biodexa held a Type C meeting with FDA that included a discussion of the statistical plan, the safety database and, most importantly, a composite endpoint for the Phase 3 study. FDA representatives from both the gastroenterology and oncology divisions provided valuable input into the proposed program.

On the back of the Phase 2 data, the Cancer Prevention & Research Institute of Texas (CPRIT) initially awarded a $17 million grant to support the Phase 3 program. This was increased to $20 million with the most recent award. Overall, CPRIT has awarded $2.9 billion in grants to Texas research institutions and organizations focused on treating and preventing cancer. CPRIT funding has advanced scientific and clinical knowledge and provided 7.4 million life-saving cancer prevention and early detection services reaching Texans from all 254 counties.

Featured image from Shutterstock.

This post contains sponsored content. This content is for informational purposes only and is not intended to be investing advice.

Click here for more information on Biodexa Pharmaceuticals.

Contact:

Stephen Stamp, CEO, CFO

ir@biodexapharma.com

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. We do not undertake to advise you as to any change in this information. 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. This is not a solicitation of any offer to buy or sell. Redington, Inc. is paid by Biodexa Pharmaceuticals PLC to provide investor relations services, and its employees or members of their families may from time to time own an equity interest in companies mentioned herein.

SOURCE: Biodexa Pharmaceuticals

View the original press release on ACCESS Newswire

CARDIFF, UK / ACCESS Newswire / July 28, 2025 / Familial Adenomatous Polyposis (FAP) is an inherited condition that puts people at a much greater risk of developing colon cancer. If left untreated, there is a near 100% likelihood that the person will develop colon or rectum cancer. Despite the seriousness, there is no approved therapeutic option for treating FAP patients, and surgical resection of the colon and/or rectum remains the standard of care.

That could change if one company has its way.

Biodexa Pharmaceuticals PLC. , a clinical-stage biopharmaceutical company, reports making significant progress in developing eRapa, an oral tablet formulation of rapamycin, to treat FAP non-surgically. eRapa inhibits the mTOR (mammalian Target Of Rapamycin) protein. Too much mTOR has been linked to cancer and has been shown to be over-expressed in FAP polyps, thereby underscoring the rationale for using an mTOR inhibitor like eRapa to treat FAP.

Key Milestones Met

Since the start of the year, Biodexa has hit several milestones with eRapa as it works to bring its treatment to the market in the U.S. and Europe. The company says it already has key opinion leaders engaged, vendors contracted, 30 sites in the U.S. and Europe identified for its Phase 3 study and funding in place, putting it ahead of its potential rivals including Recursion Pharmaceuticals Inc. (RXRX), which is currently in a Phase 1b/2 study of its treatment for FAP and Tempest Therapeutics Inc. (TPST), which in May received the go-ahead from the FDA for a Phase 2 trial of its FAP drug.

If Biodexa's eRapa treatment is shown to be successful in its Phase 3 study and the treatment gets approved, the company believes it would have first-mover advantage in what it estimates is a $7 billion addressable market opportunity.

Solid Regulatory Footing

In February, Biodexa received Fast Track status from the U.S. Food and Drug Administration (FDA) for eRapa. Fast Track status is designed to facilitate the development and expedite the review of drugs to treat serious conditions where there is an unmet medical need. The FDA awarded Biodexa Fast Track status after Phase 2 data showed patients experienced an 89% non-progression rate and a 29% median reduction in polyp burden at 12 months compared with baseline.

In conjunction with its Fast Track Status in the U.S., eRapa received Orphan Drug status in Europe, adding to the Orphan Drug designation in the U.S. that had been awarded some time ago. Both are designed to speed up the development of drugs that could provide significant benefits to patients who are suffering from rare, life-threatening diseases. The European Orphan Drug status also gives the drug company 10 years of marketing exclusivity and provides special incentives, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees, reports Biodexa.

Those designations were quickly followed by Biodexa holding a Type C meeting with the FDA that included a discussion of the statistical plan, the safety database and most importantly, a composite endpoint for a Phase 3 study. FDA representatives from both the gastroenterology and oncology divisions provided valuable input into the proposed program, which is important for the success of the Phase 3 study. In Europe, Biodexa has already named Precision for Medicine LLC as the clinical research organization to conduct the European component of the Phase 3 study of eRapa.

Trials Getting Underway

The trials are now getting underway. In June, Biodexa initiated its first clinical site in the U.S. with its collaboration partner, Rapamycin Holdings Inc., which does business as Emtora Biosciences. Emtora Biosciences was awarded an additional $3 million grant (bringing the total to $20 million) by the Cancer Prevention & Research Institute of Texas to support the Phase 3 FAP trial. Biodexa has branded its Phase 3 program the 'Serenta' trial. The Serenta trial (NCT06950385) is a randomized, double-blind, placebo-controlled study designed to evaluate the safety and efficacy of eRapa in individuals diagnosed with FAP. The first site is actively screening eligible participants.

In Europe, Biodexa just filed a Clinical Trial Application (CTA) with the European Medicines Agency (EMA) for its Serenta trial. A CTA is the formal regulatory submission required to obtain approval to begin a clinical trial in Europe and is similar to the Investigational New Drug (IND) application process in the United States. If approved, it would permit the Serenta trial to proceed in Europe, initially covering clinical sites in Denmark, Germany, the Netherlands and Spain, with Italy expected to be added in due course. Investors should expect more announcements from Biodexa as the Phase 3 trial hits more milestones in the weeks and months to come.

Biodexa is optimistic about its future prospects and aims to replicate the success of Palvella Therapeutics Inc. (PVLA), a biotechnology company also developing a rapamycin product for patients with rare dermatological diseases that sports a $425 million market cap (enterprise value of around $350 million excluding $75 million cash at March 31, 2025). Following a similar strategy, Biodexa is also going after rare diseases that don't have proven therapies and have big addressable markets, with ambitious plans to improve the quality of life for suffering patients around the world.

Featured image from Biodexa

This post contains sponsored content. This content is for informational purposes only and is not intended to be investing advice.

Click here for more information on Biodexa Pharmaceuticals.

Contact:

Stephen Stamp, CEO, CFO

ir@biodexapharma.com

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. We do not undertake to advise you as to any change in this information. 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. This is not a solicitation of any offer to buy or sell. Redington, Inc. is paid by Biodexa Pharmaceuticals PLC to provide investor relations services, and its employees or members of their families may from time to time own an equity interest in companies mentioned herein.

SOURCE: Biodexa Pharmaceuticals

View the original press release on ACCESS Newswire

European equities traded in the US as American depositary receipts were tracking higher late Tuesday morning, rising 0.56% to 1,478.37 on the S&P Europe Select ADR Index.

From continental Europe, the gainers were led by pharmaceutical company Novo Nordisk and telecommunications company Nokia , which rose 2.5% and 1.4% respectively. They were followed by medical device maker EDAP TMS and brewing company Anheuser-Busch InBev , which were up 1.3% and 1.2% respectively.

The decliners from continental Europe were led by semiconductor company Sequans Communications and biopharmaceutical company Cellectis , which fell 3.9% and 3.2% respectively. They were followed by biopharmaceutical companies Genfit and Grifols , which lost 2.8% and 1.6% respectively.

From the UK and Ireland, the gainers were led by biopharmaceutical companies Biodexa Pharmaceuticals and NuCana , which advanced 7% and 4.1% respectively. They were followed by communications company WPP and alcoholic beverage company Diageo , which increased 2.4% and 2.2% respectively.

The decliners from the UK and Ireland were led by biopharmaceutical companies Adaptimmune Therapeutics and Amarin , which dropped 3.5% and 1.7% respectively. They were followed by biotech firm Trinity Biotech and cruise line operator Carnival 1.1% and 0.7%.

European equities traded in the US as American depositary receipts were slightly higher late Friday morning, up 0.11% to 1,475.45 on the S&P Europe Select ADR Index, which ended the week more than 1% lower.

From continental Europe, the gainers were led by biopharmaceutical companies Genfit and DBV Technologies , which advanced 2.6% and 5% respectively. They were followed by biopharmaceutical company Grifols and oil and gas company TotalEnergies , which were up 0.1% and 0.6% respectively.

The decliners from continental Europe were led by semiconductor company Sequans Communications and internet browser company Opera (OPRA), which fell 7% and 0.9% respectively. They were followed by biopharmaceutical company Cellectis and internet advertising firm Criteo , which were down 1.6% and 0.5% respectively.

From the UK and Ireland, the gainers were led by biotech firm Trinity Biotech and biopharmaceutical company Mereo BioPharma Group , which increased 2.5% and 3.1% respectively. They were followed by biopharmaceutical company Biodexa Pharmaceuticals and utilities company National Grid , which rose 1.5% and 0.7% respectively.

The decliners from the UK and Ireland were led by biopharmaceutical company NuCana , which lost 4.9%. They were followed by biopharmaceutical company Akari Therapeutics and financial services company Barclays , which were off 0.1% and 0.8% respectively.