With Fast Track Designation in Hand, a Successful Protocol Discussion with FDA, and CROs in Place, Biodexa is on Track to Initiate its Funded Phase 3 Trial in FAP Next Quarter
Biodexa Pharmaceuticals (NASDAQ:BDRX) is advancing towards initiating a Phase 3 trial for eRapa, its proprietary encapsulated rapamycin formulation for treating familial adenomatous polyposis (FAP). The company recently achieved key milestones including FDA Fast Track designation and a successful Type C meeting.
The Phase 3 study will be a double-blind placebo-controlled trial involving approximately 168 high-risk FAP patients across 30 clinical sites in the U.S. and Europe. The trial is substantially funded with a $17 million grant from the Cancer Prevention Research Institute of Texas, matched by $8.5 million from Biodexa.
The company's Phase 2 trial demonstrated promising results, with cohort 2 showing an 89% non-progression rate and 29% median reduction in polyp burden at 12 months. Biodexa has appointed LumaBridge and Precision for Medicine as CROs for the U.S. and European components respectively.
Biodexa Pharmaceuticals (NASDAQ:BDRX) sta avanzando verso l'avvio di uno studio di Fase 3 per eRapa, la sua formulazione proprietaria di rapamicina incapsulata per il trattamento della poliposi adenomatosa familiare (FAP). L'azienda ha recentemente raggiunto traguardi chiave, tra cui la designazione Fast Track della FDA e un incontro di tipo C di successo.
Lo studio di Fase 3 sarà uno studio controllato, in doppio cieco e con placebo, che coinvolgerà circa 168 pazienti ad alto rischio di FAP in 30 siti clinici negli Stati Uniti e in Europa. La sperimentazione è finanziata in gran parte da una sovvenzione di 17 milioni di dollari del Cancer Prevention Research Institute of Texas, integrata da 8,5 milioni di dollari da Biodexa.
Il trial di Fase 2 dell'azienda ha mostrato risultati promettenti, con il secondo gruppo che ha evidenziato una percentuale di non progressione dell'89% e una riduzione mediana del carico di polipi del 29% a 12 mesi. Biodexa ha nominato LumaBridge e Precision for Medicine come CRO per le componenti statunitensi ed europee, rispettivamente.
Biodexa Pharmaceuticals (NASDAQ:BDRX) está avanzando hacia el inicio de un ensayo de Fase 3 para eRapa, su formulación de rapamicina encapsulada para el tratamiento de la poliposis adenomatosa familiar (FAP). La empresa logró recientemente hitos clave, incluyendo la designación de Vía Rápida de la FDA y una reunión tipo C exitosa.
El estudio de Fase 3 será un ensayo controlado, doble ciego y con placebo que involucrará aproximadamente a 168 pacientes de alto riesgo con FAP en 30 sitios clínicos en EE. UU. y Europa. El ensayo está financiado en gran parte con una subvención de 17 millones de dólares del Cancer Prevention Research Institute of Texas, complementada con 8,5 millones de dólares de Biodexa.
El ensayo de Fase 2 de la empresa mostró resultados prometedores, con el grupo 2 mostrando una tasa de no progresión del 89% y una reducción mediana del 29% en la carga de pólipos a los 12 meses. Biodexa ha designado a LumaBridge y Precision for Medicine como CRO para las partes de EE. UU. y Europa, respectivamente.
바이오덱사 제약회사 (NASDAQ:BDRX)는 가족성 선종성 폴립증(FAP) 치료를 위한 독점적인 캡슐화된 라파마이신 제형인 eRapa의 3상 시험 개시를 향해 나아가고 있습니다. 이 회사는 최근 FDA의 패스트 트랙 지정 및 성공적인 타입 C 회의와 같은 주요 이정표를 달성했습니다.
3상 연구는 미국과 유럽의 30개 임상 사이트에서 약 168명의 고위험 FAP 환자를 대상으로 하는 이중 맹검 위약 대조 시험이 될 것입니다. 이 시험은 텍사스 암 예방 연구소의 1,700만 달러 보조금으로 상당 부분 자금을 지원받고 있으며, 바이오덱사에서 850만 달러를 추가로 지원합니다.
회사의 2상 시험은 유망한 결과를 보여주었으며, 2군에서는 89%의 비진행률과 12개월 동안 폴립 부담의 중간 29% 감소가 나타났습니다. 바이오덱사는 미국 및 유럽 부문의 CRO로 LumaBridge와 Precision for Medicine을 각각 임명했습니다.
Biodexa Pharmaceuticals (NASDAQ:BDRX) avance vers le lancement d'un essai de Phase 3 pour eRapa, sa formulation de rapamycine encapsulée pour le traitement de la polypose adénomateuse familiale (FAP). L'entreprise a récemment atteint des jalons clés, y compris la désignation Fast Track de la FDA et une réunion de type C réussie.
L'étude de Phase 3 sera un essai contrôlé, en double aveugle et avec placebo, impliquant environ 168 patients à haut risque de FAP dans 30 sites cliniques aux États-Unis et en Europe. L'essai est largement financé par une subvention de 17 millions de dollars du Cancer Prevention Research Institute of Texas, complétée par 8,5 millions de dollars de Biodexa.
L'essai de Phase 2 de l'entreprise a montré des résultats prometteurs, le groupe 2 affichant un taux de non-progression de 89 % et une réduction médiane de 29 % de la charge en polypes après 12 mois. Biodexa a nommé LumaBridge et Precision for Medicine comme CRO pour les composantes américaines et européennes, respectivement.
Biodexa Pharmaceuticals (NASDAQ:BDRX) schreitet voran, um eine Phase-3-Studie für eRapa, seine proprietäre, verkapselte Rapamycin-Formulierung zur Behandlung der familiären adenomatösen Polyposis (FAP), zu starten. Das Unternehmen hat kürzlich wichtige Meilensteine erreicht, darunter die Fast-Track-Bezeichnung der FDA und ein erfolgreiches Typ-C-Meeting.
Die Phase-3-Studie wird eine doppelblinde, placebo-kontrollierte Studie sein, die etwa 168 Hochrisiko-FAP-Patienten an 30 klinischen Standorten in den USA und Europa einbezieht. Die Studie wird größtenteils durch einen Zuschuss von 17 Millionen Dollar des Cancer Prevention Research Institute of Texas finanziert, ergänzt durch 8,5 Millionen Dollar von Biodexa.
Die Phase-2-Studie des Unternehmens zeigte vielversprechende Ergebnisse, wobei Kohorte 2 eine Nicht-Progressionsrate von 89 % und eine mediane Reduktion der Polypenlast um 29 % nach 12 Monaten aufwies. Biodexa hat LumaBridge und Precision for Medicine als CROs für die US-amerikanischen und europäischen Komponenten ernannt.
- Received FDA Fast Track designation for eRapa
- Successfully completed Phase 2 trial with 89% non-progression rate in cohort 2
- Secured substantial funding of $25.5M for Phase 3 trial
- Appointed experienced CROs for both U.S. and European trial components
- Clear regulatory pathway established after successful FDA Type C meeting
- No currently approved products or revenue stream
- Will require additional funding beyond Phase 3 trial for commercialization
Insights
Biodexa's regulatory progress for eRapa represents meaningful advancement in the drug development pathway. The Fast Track designation grants several important advantages, including more frequent FDA communications, potential eligibility for Accelerated Approval, and rolling submission review - all potentially shortening the timeline to market by months. The successful Type C meeting specifically addressing statistical plan, safety database, and composite endpoint resolution removes significant regulatory uncertainty for the Phase 3 program.
The double-blind, placebo-controlled Phase 3 design with 168 patients across 30 sites represents an appropriately powered and designed registrational study for this rare indication. The Phase 2 efficacy signals are particularly encouraging, with the selected Phase 3 dosing regimen demonstrating an
The fully-funded status of the program (
The advancement of eRapa into Phase 3 represents a potential paradigm shift for FAP patients who currently face no FDA-approved therapeutic options beyond invasive surgical management. FAP profoundly impacts quality of life, with polyp development typically beginning in adolescence and inevitable progression to colorectal cancer without intervention. The current standard of care - complete colectomy - carries substantial morbidity and lifelong consequences.
The mechanistic rationale for eRapa is scientifically sound, targeting overexpressed mTOR signaling in FAP polyps. The Phase 2 data showing meaningful polyp burden reduction and high non-progression rates suggests potential to delay or reduce surgical interventions - a transformative prospect for these patients.
The composite endpoint agreement with FDA likely addresses both polyp burden and progression metrics, appropriate for capturing clinically meaningful outcomes in this population. While surgical prevention will remain necessary for many patients, an effective pharmaceutical intervention could potentially delay surgery or reduce its extent, preserving organ function longer.
The involvement of specialized rare disease CROs increases confidence in appropriate site selection and patient recruitment strategies - critical factors in rare disease trials that often fail due to enrollment challenges. With thousands of new FAP cases annually and no approved alternatives, eRapa addresses a clear unmet medical need with potential first-to-market advantage in this orphan indication.
CARDIFF, UNITED KINGDOM / ACCESS Newswire / March 19, 2025 / Biodexa Pharmaceuticals PLC. (NASDAQ:BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, is making progress in readying the launch of a phase 3 trial for eRapa, its proprietary encapsulated form of rapamycin being developed for the treatment of familial adenomatous polyposis (FAP).
In just the last few weeks, Biodexa received Fast Track designation from the FDA, conducted a successful Type C (pre-Phase 3 protocol finalization) meeting with the FDA and appointed a clinical research organization (CRO) to conduct the European component of its registrational Phase 3 trial. A CRO for the U.S. component was appointed earlier.
FAP is an inherited condition that puts people at a much greater risk of developing colon cancer. 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 removed. If left untreated there is a high likelihood the person will develop colon or rectum cancer.
Biodexa hopes to help with eRapa, 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 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", noted Stephen Stamp, Biodexa's CEO and CFO.
Phase 3 Study Commencing Next Quarter
Biodexa, which has already received Fast Track designation by the FDA for the drug, completed a successful Phase 2 trial of eRapa, demonstrating a
The Phase 3 study will be a double-blind placebo-controlled design recruiting approximately 168 high-risk patients diagnosed with germline or phenotypic FAP. It is expected the study will be conducted in about 30 clinical sites across the U.S. and Europe.
That Phase 3 trial is getting closer to a launch following on the heels of what the company says was a successful Type C meeting with the FDA. During the meeting with FDA representatives from both the gastroenterology and oncology divisions, Biodexa and the FDA discussed the company's statistical plan, the safety database and a composite endpoint for the Phase 3 study. As a result of that meeting, Biodexa believes it has a clear path forward for the initiation of the U.S. Phase 3 study next quarter.
"With no approved products for FAP, we were pleased to collaborate with FDA and our U.S. CRO, LumaBridge, to define the regulatory pathway for eRapa in FAP," said Gary Shangold, MD, Chief Medical Officer of Biodexa. "Agreement on the composite endpoint, in particular, clears the path to finalize the protocol, recruit the U.S. sites and begin patient enrollment."
The Phase 3 program is substantially funded by a
U.S. and European CROs On Board
Biodexa previously appointed LumaBridge to conduct the study in the U.S. and just tapped Precision for Medicine as the CRO to conduct the European component of the upcoming registrational Phase 3 study of eRapa in FAP.
LumaBridge was founded in 2014 to help advance the development of novel immunotherapies in the fight against cancer. Building on the founders' 3 decades of combined experience in academic and military research, LumaBridge offers full outsourced clinical trial support across the entire timeline of clinical development, including consultation on clinical development strategy, as well as special capabilities in military research. Over the 11 years since its founding, LumaBridge has made a significant impact in the advancement of immuno-oncology therapies, supporting a substantial number of trials and projects for over 30 clients.
Precision for Medicine is focused on rare diseases and has a stated mission to accelerate the pathway for complex drug development. Precision for Medicine has been conducting studies for over twenty years and according to Biodexa is known for its high-caliber, therapeutically specialized staff, experienced scientists and physicians, advanced specialty laboratories and problem-solving capabilities. Precision for Medicine has conducted 333 clinical trials in rare diseases and employs over 700 team members in Europe across 11 locations, Biodexa shared in a press release.
With thousands of new cases of FAP each year, people in Europe and America suffering from this disease need relief. Biodexa is aiming to deliver that, and with its phase 3 study about to get underway, that may happen sooner rather than later.
Featured photo by National Cancer InstituteonUnsplash.
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Stephen Stamp, CEO, CFO
ir@biodexapharma.com
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SOURCE: Biodexa Pharmaceuticals PLC
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