August 18, 2025 Biodexa Announces Enrolment of First Patients into Pivotal Phase 3 Serenta Trial in Familial Adenomatous Polyposis (FAP) Opportunity to be First Mover in $7.3Bn Addressable Market Biodexa Pharmaceuticals PLC (“Biodexa” or “the Company”), (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, is pleased to announce the enrolment of the first two patients by the Pan American Center for Onc ...

Core Viewpoint - Biodexa Pharmaceuticals has filed a Clinical Trial Application (CTA) with the European Medicines Agency (EMA) for its Phase 3 Serenta trial targeting familial adenomatous polyposis (FAP), a hereditary condition that significantly increases the risk of colorectal cancer if untreated [2][4]. Company Overview - Biodexa Pharmaceuticals PLC is a clinical stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, including eRapa for FAP and other conditions [2][11]. - The company has received Fast Track Designation from the FDA and Orphan Drug designation in Europe for its eRapa program [4]. Clinical Trial Details - The Serenta trial (NCT06950385) is a randomized, double-blind, placebo-controlled Phase 3 study aimed at evaluating the safety and efficacy of eRapa in FAP patients [5]. - The trial will initially cover clinical sites in Denmark, Germany, Netherlands, and Spain, with Italy expected to be added later [3]. Funding and Support - The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded $20 million in grant funding to support the eRapa program [4]. - CPRIT has a history of significant investment in cancer research, having awarded $2.9 billion in grants to date [9]. Disease Context - Familial adenomatous polyposis (FAP) is characterized by the development of hundreds to thousands of colorectal polyps, leading to a near-100% lifetime risk of colorectal cancer if untreated [7]. - There is a significant unmet need for effective therapies for FAP, as current treatment options primarily involve surgical resection [7]. Product Information - eRapa is a proprietary oral formulation of rapamycin (sirolimus), an mTOR inhibitor, designed to improve bioavailability and reduce toxicity compared to existing formulations [8][12]. - The product is protected by multiple patents extending through 2035, with additional applications pending [8].

Core Viewpoint - Biodexa Pharmaceuticals has filed a Clinical Trial Application (CTA) with the European Medicines Agency (EMA) for its Phase 3 Serenta trial targeting familial adenomatous polyposis (FAP), a hereditary condition that significantly increases the risk of colorectal cancer if untreated [2][4]. Company Overview - Biodexa Pharmaceuticals PLC is a clinical stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, including eRapa for FAP and other conditions [2][11]. - The company has received Fast Track Designation from the FDA and Orphan Drug designation in Europe for its eRapa program [4]. Clinical Trial Details - The Serenta trial (NCT06950385) is a randomized, double-blind, placebo-controlled Phase 3 study aimed at evaluating the safety and efficacy of eRapa in FAP patients [5]. - The trial will initially cover clinical sites in Denmark, Germany, Netherlands, and Spain, with Italy expected to be added later [3]. Funding and Support - The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded $20 million in grant funding to support the eRapa program, contributing to the advancement of the trial [4][9]. Disease Context - Familial adenomatous polyposis (FAP) is characterized by the development of hundreds to thousands of colorectal polyps, leading to a near-100% lifetime risk of colorectal cancer if untreated [7]. - There is a significant unmet need for effective and less invasive therapies for FAP patients, as current treatment options primarily involve surgical resection [7]. Product Information - eRapa is a proprietary oral formulation of rapamycin (sirolimus), an mTOR inhibitor, designed to improve bioavailability and reduce toxicity compared to existing formulations [8][12]. - The product is protected by multiple patents extending through 2035, with additional applications potentially providing further protection [8].

Core Viewpoint - Biodexa Pharmaceuticals has activated the first clinical study site for its Phase 3 Serenta trial targeting familial adenomatous polyposis (FAP), marking a significant milestone in developing a new treatment option for this condition [1][3]. Group 1: Clinical Trial Details - The Serenta trial (NCT06950385) is a randomized, double-blind, placebo-controlled study aimed at evaluating the safety and efficacy of eRapa in individuals diagnosed with FAP [2]. - The first clinical site in the US is now open and actively screening eligible participants for the trial [2]. Group 2: Company Achievements and Support - Biodexa's CEO, Stephen Stamp, highlighted the importance of the first clinical site activation following the Fast Track Designation and a positive Type C Meeting, emphasizing the collaborative efforts with Emtora Biosciences and LumaBridge [3]. - The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded $20 million in grant funding to support the eRapa program, showcasing significant financial backing for the initiative [3][6]. Group 3: Background on Familial Adenomatous Polyposis (FAP) - FAP is a rare inherited disorder characterized by the development of hundreds to thousands of colorectal polyps, with a near-100% lifetime risk of colorectal cancer if untreated [4]. - There is a significant unmet need for effective and less invasive therapies for FAP patients, as current standard care involves active surveillance and surgical resection [4]. Group 4: About eRapa - eRapa is a proprietary oral formulation of rapamycin (sirolimus), an mTOR inhibitor, which plays a crucial role in regulating cellular metabolism, growth, and proliferation [5][8]. - The formulation is designed to improve bioavailability and reduce toxicity associated with existing rapamycin forms, with patent protection extending through 2035 [5][11].

Core Insights - Biodexa Pharmaceuticals PLC has announced the brand name "Serenta" for its upcoming Phase 3 clinical study in Familial Adenomatous Polyposis (FAP) [1][2] - The company has launched a dedicated website, www.serentatrial.com, to provide information and resources for patients, caregivers, and healthcare professionals [1][2] Company Overview - Biodexa is a clinical stage biopharmaceutical company focused on developing innovative products for diseases with unmet medical needs, with its lead programs including eRapa for FAP and Non-Muscle Invasive Bladder Cancer, tolimidone for type 1 diabetes, and MTX110 for aggressive rare/orphan brain cancer [4][8] - eRapa is a proprietary oral tablet formulation of rapamycin, an mTOR inhibitor, designed to improve bioavailability and reduce toxicity associated with existing rapamycin formulations [3][5] - The company utilizes proprietary drug delivery technologies to enhance the bio-distribution of its medicines [8]

Core Viewpoint - Biodexa Pharmaceuticals has received Orphan Drug Designation in Europe for eRapa in familial adenomatous polyposis (FAP), following a similar designation from the FDA in 2019, and is preparing to initiate a Phase 3 study targeting a market opportunity of approximately $7.3 billion [1][2][5]. Company Overview - Biodexa Pharmaceuticals PLC is a clinical stage biopharmaceutical company focused on developing innovative treatments for diseases with unmet medical needs, including eRapa for FAP and other products for type 1 diabetes and rare brain cancers [9][13]. Orphan Drug Designation - The Orphan Drug Designation in the EU is granted by the European Commission based on a positive opinion from the EMA Committee for Orphan Medicinal Products, aimed at encouraging the development of drugs for rare, life-threatening diseases [2]. Phase 3 Study Details - The Phase 3 study of eRapa in FAP will be a double-blind placebo-controlled trial involving 168 patients, with a 2:1 randomization of drug to placebo, conducted across approximately 30 clinical sites in the US and Europe [3]. Market Opportunity - The addressable market for eRapa in FAP is estimated at $7.3 billion, based on the prevalence of FAP and the adult populations in the US and Europe, with a median annual cost of approved non-biologic orphan drugs in the US being $206,176 [5]. FAP Disease Overview - Familial adenomatous polyposis (FAP) is characterized by the proliferation of polyps in the colon and rectum, typically diagnosed in mid-teens, with no approved therapeutic options currently available [4]. eRapa Product Information - eRapa is a proprietary oral formulation of rapamycin, an mTOR inhibitor, designed to improve bioavailability and reduce toxicity compared to existing forms of rapamycin [6][10]. Clinical Study Results - Data from the Phase 2 study of eRapa indicated a median 17% reduction in total polyp burden at 12 months and a 75% non-progression rate, with cohort 2 showing an 89% non-progression rate and a 29% median reduction in polyp burden [7].