Biodexa Receives US FDA Fast Track Designation for eRapa in Familial Adenomatous Polyposis
Biodexa Pharmaceuticals (Nasdaq: BDRX) has received FDA Fast Track designation for eRapa, their proprietary encapsulated rapamycin treatment for familial adenomatous polyposis (FAP). This designation aims to expedite development and review of drugs addressing serious unmet medical needs.
The Phase 2 study of eRapa demonstrated promising results with a median 17% reduction in total polyp burden at 12 months and a 75% non-progression rate. Notably, cohort 2 showed even better outcomes with an 89% non-progression rate and 29% median reduction in polyp burden using an alternate-week dosing regimen, which will be used in the upcoming Phase 3 study.
FAP, affecting 1 in 5,000-10,000 people in the US and 1 in 11,300-37,600 in Europe, currently has no approved therapeutic options besides surgical removal of the colon/rectum. eRapa has also received FDA Orphan Drug designation for FAP treatment.
Biodexa Pharmaceuticals (Nasdaq: BDRX) ha ricevuto la designazione Fast Track della FDA per eRapa, il loro trattamento proprietario a base di rapamicina incapsulata per la poliposi adenomatosa familiare (FAP). Questa designazione mira ad accelerare lo sviluppo e la revisione di farmaci che affrontano gravi esigenze mediche non soddisfatte.
Lo studio di Fase 2 di eRapa ha dimostrato risultati promettenti con una riduzione mediana del 17% del carico poliposo totale a 12 mesi e un tasso di non progressione del 75%. Vale la pena notare che il campione 2 ha mostrato risultati ancora migliori con un tasso di non progressione dell'89% e una riduzione mediana del 29% del carico poliposo utilizzando un regime di dosaggio alternato ogni settimana, che sarà utilizzato nel prossimo studio di Fase 3.
La FAP, che colpisce 1 persona su 5.000-10.000 negli Stati Uniti e 1 su 11.300-37.600 in Europa, attualmente non ha opzioni terapeutiche approvate oltre alla rimozione chirurgica del colon/retto. eRapa ha anche ricevuto la designazione di farmaco orfano della FDA per il trattamento della FAP.
Biodexa Pharmaceuticals (Nasdaq: BDRX) ha recibido la designación Fast Track de la FDA para eRapa, su tratamiento propietario de rapamicina encapsulada para la poliposis adenomatosa familiar (FAP). Esta designación tiene como objetivo acelerar el desarrollo y la revisión de medicamentos que abordan serias necesidades médicas no satisfechas.
El estudio de Fase 2 de eRapa mostró resultados prometedores con una reducción mediana del 17% en la carga total de pólipos a 12 meses y una tasa de no progresión del 75%. Notablemente, el cohorte 2 mostró resultados aún mejores con una tasa de no progresión del 89% y una reducción mediana del 29% en la carga de pólipos utilizando un régimen de dosificación cada dos semanas, que se utilizará en el próximo estudio de Fase 3.
La FAP, que afecta a 1 de cada 5.000-10.000 personas en EE.UU. y 1 de cada 11.300-37.600 en Europa, actualmente no tiene opciones terapéuticas aprobadas además de la extirpación quirúrgica del colon/recto. eRapa también ha recibido la designación de medicamento huérfano de la FDA para el tratamiento de la FAP.
바이오덱사 제약 (Nasdaq: BDRX)는 가족성 선종성 용종증(FAP) 치료를 위한 독자적인 캡슐화된 라파마이신 치료제인 eRapa에 대해 FDA의 패스트 트랙 지정을 받았습니다. 이 지정은 심각한 unmet medical needs를 해결하는 약물의 개발과 검토를 가속화하는 것을 목표로 합니다.
eRapa의 2상 연구는 12개월 기준 총 용종 부담의 중앙값 17% 감소와 75%의 비진행률을 보이며 유망한 결과를 나타냈습니다. 특히, 코호트 2는 대체 주간 투여 요법을 사용하여 89% 비진행률과 29%의 중앙값 용종 부담 감소라는 더 나은 결과를 나타냈는데, 이는 곧 있을 3상 연구에서 사용될 예정입니다.
FAP는 미국에서 5,000-10,000명 중 1명, 유럽에서 11,300-37,600명 중 1명에게 영향을 미치며, 현재 대장/직장 절제 외에 승인된 치료 옵션이 없습니다. eRapa는 FAP 치료를 위한 FDA의 오르판 약물 지정을 받기도 했습니다.
Biodexa Pharmaceuticals (Nasdaq: BDRX) a reçu la dénomination Fast Track de la FDA pour eRapa, son traitement propriétaire encapsulé à base de rapamycine pour la polypose adénomateuse familiale (FAP). Cette dénomination vise à accélérer le développement et l'examen des médicaments répondant à des besoins médicaux non satisfaits graves.
La phase 2 de l'étude sur eRapa a montré des résultats prometteurs avec une réduction médiane de 17 % de la charge totale en polypes à 12 mois et un taux de non-progression de 75 %. Notamment, le groupe 2 a montré des résultats encore meilleurs avec un taux de non-progression de 89 % et une réduction médiane de 29 % de la charge en polypes utilisant un schéma posologique en alternance, qui sera utilisé dans le prochain essai de phase 3.
La FAP, qui touche 1 personne sur 5.000 à 10.000 aux États-Unis et 1 sur 11.300 à 37.600 en Europe, n'a actuellement pas d'options thérapeutiques approuvées en dehors de l'ablation chirurgicale du côlon/rectum. eRapa a également reçu la désignation de médicament orphelin de la FDA pour le traitement de la FAP.
Biodexa Pharmaceuticals (Nasdaq: BDRX) hat die Fast-Track-Zulassung der FDA für eRapa erhalten, ihre proprietäre encapsulierte Rapamycin-Behandlung für die familiäre adenomatöse Polyposis (FAP). Diese Zulassung zielt darauf ab, die Entwicklung und Überprüfung von Medikamenten, die schweren ungedeckten medizinischen Bedürfnissen entsprechen, zu beschleunigen.
Die Phase 2-Studie zu eRapa zeigte vielversprechende Ergebnisse mit einer medianen Reduzierung des gesamten Polypenlast um 17% nach 12 Monaten und einer Non-Progressionsrate von 75%. Besonders cohort 2 zeigte noch bessere Ergebnisse mit einer Non-Progressionsrate von 89% und einer medianen Reduzierung der Polypenlast um 29% bei Anwendung eines alternierenden Dosierungsschemas, das in der bevorstehenden Phase 3-Studie verwendet werden wird.
FAP, das 1 von 5.000-10.000 Menschen in den USA und 1 von 11.300-37.600 in Europa betrifft, hat derzeit keine genehmigten Therapieoptionen außer der chirurgischen Entfernung von Colon/Rektum. eRapa hat auch die Orphan-Drug-Zulassung der FDA für die FAP-Behandlung erhalten.
- FDA Fast Track designation received for eRapa
- Phase 2 results showed 17% median reduction in polyp burden
- Cohort 2 achieved 89% non-progression rate and 29% polyp reduction
- FDA Orphan Drug designation already obtained
- None.
Insights
The FDA Fast Track designation for eRapa marks a pivotal development for both Biodexa and FAP patients. This designation typically accelerates the development timeline by 2-3 years and enables more frequent FDA interactions, rolling reviews and potential priority review qualification. The Phase 2 results are particularly compelling, with cohort 2's
The market opportunity is substantial despite FAP's rare disease status. With current treatment to surgical intervention costing
Three key factors make this development particularly significant:
- eRapa is positioned to be the first FDA-approved therapeutic option for FAP, potentially establishing market dominance
- The hereditary nature of FAP ensures a stable, identifiable patient population
- The combination of Orphan Drug status and Fast Track designation provides enhanced market protection and accelerated development pathways
This progress could catalyze broader interest in Biodexa's pipeline and technology platform, particularly in rare disease applications where their drug delivery expertise could be leveraged for other indications.
February 10, 2025
Biodexa Receives US FDA Fast Track Designation for eRapa in Familial Adenomatous Polyposis
Underscores unmet need for a therapeutic alternative with the potential to delay or prevent surgical removal of the colon and/or rectum
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, announced today that the US Food and Drug Administration (“FDA”) has granted Fast Track designation for eRapa, a proprietary encapsulated form of rapamycin being developed for the treatment of familial adenomatous polyposis (FAP). Fast track designation is intended to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
The designation was requested based on the potential for eRapa to address an unmet medical need for FAP, a condition which left untreated universally leads to colorectal cancer. Today, the only treatment option is surgical resection of the colon and/or rectum. Data from the Phase 2 study of eRapa in FAP showed eRapa to be safe and well-tolerated with a median
eRapa in FAP
FAP is characterized as a proliferation of polyps in the colon and/or rectum, usually occurring in mid-teens. 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. If untreated, FAP universally leads to cancer of the colon and/or rectum. There is a significant hereditary component to FAP with a reported prevalence of one in 5,000 to 10,000 in the US1 and one in 11,300 to 37,600 in Europe2. Biodexa has received US FDA Orphan Drug designation for eRapa in FAP and plans to seek a similar designation in Europe.
About eRapa
eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus. Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor. mTOR has been shown to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorgenesis3. Importantly, mTOR has been shown to be over-expressed in FAP polyps – thereby underscoring the rationale for using a potent and safe mTOR inhibitor like eRapa to treat FAP. Rapamycin is approved in the US for organ rejection in renal transplantation as Rapamune®(Pfizer). Through the use of nanotechnology and pH sensitive polymers, eRapa is designed to address the poor bioavailability, variable pharmacokinetics and toxicity generally associated with the currently available forms of rapamycin. eRapa is protected by a number of issued patents which extend through 2035, with other pending applications potentially providing further protection beyond 2035.
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1. www.rarediseases.org
2. www.orpha.net
3. Tian et al., mTOR Signaling in Cancer and mTOR Inhibitors in Solid Tumor Targeting Therapy, Int J Mol Sci. 2019 Feb; 20(3): 755
For more information, please contact:
Biodexa Pharmaceuticals PLC |
| Stephen Stamp, CEO, CFO |
| Tel: +44 (0)29 20480 180 |
| www.biodexapharma.com |
About Biodexa Pharmaceuticals PLC
Biodexa Pharmaceuticals PLC (listed on NASDAQ: BDRX) is a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs. The Company’s lead development programs include eRapa, under development for Familial Adenomatous Polyposis and Non-Muscle Invasive Blader Cancer; tolimidone, under development for the treatment of type 1 diabetes; and MTX110, which is being studied in aggressive rare/orphan brain cancer indications.
eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus. Rapamycin is an mTOR (mammalian Target Of Rapamycin) inhibitor. mTOR has been shown to have a significant role in the signalling pathway that regulates cellular metabolism, growth and proliferation and is activated during tumorigenesis.
Tolimidone is an orally delivered, potent and selective inhibitor of Lyn kinase. Lyn is a member of the Src family of protein tyrosine kinases, which is mainly expressed in hematopoietic cells, in neural tissues, liver, and adipose tissue. Tolimidone demonstrates glycaemic control via insulin sensitization in animal models of diabetes and has the potential to become a first in class blood glucose modulating agent.
MTX110 is a solubilized formulation of the histone deacetylase (HDAC) inhibitor, panobinostat. This proprietary formulation enables delivery of the product via convection-enhanced delivery (CED) at chemotherapeutic doses directly to the site of the tumor, by-passing the blood-brain barrier and potentially avoiding systemic toxicity.
Biodexa is supported by three proprietary drug delivery technologies focused on improving the bio-delivery and bio-distribution of medicines. Biodexa’s headquarters and R&D facility is in Cardiff, UK. For more information visit www.biodexapharma.com.
Forward-Looking Statements
Certain statements in this announcement may constitute “forward-looking statements” within the meaning of legislation in the United Kingdom and/or United States. Such statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and are based on management’s belief or interpretation. All statements contained in this announcement that do not relate to matters of historical fact should be considered forward-looking statements. In certain cases, forward-looking statements can be identified by the use of words such as “plans”, “expects” or “does not anticipate”, or “believes”, or variations of such words and phrases or statements that certain actions, events or results “may”, “could”, “would”, “might” or “will be taken”, “occur” or “be achieved.” Forward-looking statements and information are subject to various known and unknown risks and uncertainties, many of which are beyond the ability of the Company to control or predict, that may cause their actual results, performance or achievements to be materially different from those expressed or implied thereby, and are developed based on assumptions about such risks, uncertainties and other factors set out herein.
Reference should be made to those documents that Biodexa shall file from time to time or announcements that may be made by Biodexa in accordance with the rules and regulations promulgated by the SEC, which contain and identify other important factors that could cause actual results to differ materially from those contained in any projections or forward-looking statements. These forward-looking statements speak only as of the date of this announcement. All subsequent written and oral forward-looking statements by or concerning Biodexa are expressly qualified in their entirety by the cautionary statements above. Except as may be required under relevant laws in the United States, Biodexa does not undertake any obligation to publicly update or revise any forward-looking statements because of new information, future events or events otherwise arising.
FAQ
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