Following Positive Phase 2 Results and Orphan Drug Designation, Biodexa's FAP Drug Receives FDA Fast Track Status
Biodexa Pharmaceuticals (NASDAQ:BDRX) has received FDA Fast Track status for eRapa, its drug treating familial adenomatous polyposis (FAP), following positive Phase 2 results and Orphan Drug designation. The Phase 2 trial showed promising results with a 17% median decrease in overall polyp burden and 75% non-progression rate. Notably, cohort 2 demonstrated an 89% non-progression rate and 29% median reduction in polyp burden at 12 months.
FAP is an inherited condition affecting 1 in 5,000-10,000 people in the U.S. and 1 in 11,300-37,600 in Europe, leading to a nearly 100% lifetime risk of colorectal cancer. Currently, there are no approved therapeutic options, with surgery being the standard treatment. The upcoming Phase 3 study will be double-blind placebo-controlled, recruiting approximately 168 high-risk FAP patients.
Biodexa Pharmaceuticals (NASDAQ:BDRX) ha ricevuto lo status di Fast Track dalla FDA per eRapa, il suo farmaco per il trattamento della poliposi adenomatosa familiare (FAP), dopo risultati positivi nella fase 2 e la designazione di Farmaco Orfano. La sperimentazione di fase 2 ha mostrato risultati promettenti con una riduzione mediana del 17% del carico polipoide e un tasso di non progressione del 75%. In particolare, il gruppo 2 ha dimostrato un tasso di non progressione dell'89% e una riduzione mediana del 29% del carico polipoide a 12 mesi.
La FAP è una condizione ereditaria che colpisce 1 persona ogni 5.000-10.000 negli Stati Uniti e 1 ogni 11.300-37.600 in Europa, con un rischio di quasi il 100% di cancro colorettale nel corso della vita. Attualmente, non ci sono opzioni terapeutiche approvate, con la chirurgia che rappresenta il trattamento standard. Il prossimo studio di fase 3 sarà in doppio cieco e controllato con placebo, reclutando circa 168 pazienti ad alto rischio di FAP.
Biodexa Pharmaceuticals (NASDAQ:BDRX) ha recibido el estatus de Fast Track de la FDA para eRapa, su medicamento que trata la poliposis adenomatosa familiar (FAP), tras resultados positivos en la fase 2 y la designación de Medicamento Huérfano. El ensayo de fase 2 mostró resultados prometedores con una disminución mediana del 17% en la carga total de pólipos y una tasa de no progresión del 75%. Notablemente, el grupo 2 demostró una tasa de no progresión del 89% y una reducción mediana del 29% en la carga de pólipos a los 12 meses.
La FAP es una condición hereditaria que afecta a 1 de cada 5.000-10.000 personas en EE. UU. y a 1 de cada 11.300-37.600 en Europa, lo que lleva a un riesgo casi del 100% de cáncer colorrectal a lo largo de la vida. Actualmente, no hay opciones terapéuticas aprobadas, siendo la cirugía el tratamiento estándar. El próximo estudio de fase 3 será doble ciego y controlado con placebo, reclutando aproximadamente a 168 pacientes de alto riesgo de FAP.
바이오덱사 제약 (NASDAQ:BDRX)는 가족성 선종성 용종증(FAP) 치료 약물인 eRapa에 대해 긍정적인 2상 결과와 고아약 지정에 따라 FDA의 패스트 트랙 지위를 받았습니다. 2상 시험은 전체 용종 부담에서 17%의 중앙 감소와 75%의 비진행률을 보이며 유망한 결과를 보여주었습니다. 특히, 코호트 2는 12개월 동안 89%의 비진행률과 29%의 중앙 용종 부담 감소를 보여주었습니다.
FAP는 미국에서 5,000-10,000명 중 1명, 유럽에서는 11,300-37,600명 중 1명에게 영향을 미치는 유전적 질환으로, 평생 대장암 발생 위험이 거의 100%에 이릅니다. 현재 승인된 치료 옵션이 없으며, 수술이 표준 치료입니다. 다가오는 3상 연구는 이중 맹검 위약 대조로 진행되며, 약 168명의 고위험 FAP 환자를 모집할 예정입니다.
Biodexa Pharmaceuticals (NASDAQ:BDRX) a reçu le statut Fast Track de la FDA pour eRapa, son médicament traitant la polypose adénomateuse familiale (FAP), suite à des résultats positifs en phase 2 et à la désignation de médicament orphelin. L'essai de phase 2 a montré des résultats prometteurs avec une diminution médiane de 17% de la charge totale de polypes et un taux de non-progression de 75%. Notamment, le groupe 2 a démontré un taux de non-progression de 89% et une réduction médiane de 29% de la charge de polypes à 12 mois.
La FAP est une condition héréditaire qui touche 1 personne sur 5.000-10.000 aux États-Unis et 1 sur 11.300-37.600 en Europe, entraînant un risque de cancer colorectal presque à 100% au cours de la vie. Actuellement, il n'existe aucune option thérapeutique approuvée, la chirurgie étant le traitement standard. La prochaine étude de phase 3 sera en double aveugle et contrôlée par placebo, recrutant environ 168 patients à haut risque de FAP.
Biodexa Pharmaceuticals (NASDAQ:BDRX) hat den Fast Track-Status der FDA für eRapa erhalten, ein Medikament zur Behandlung der familiären adenomatösen Polyposis (FAP), nach positiven Ergebnissen der Phase 2 und der Orphan Drug-Designation. Die Phase-2-Studie zeigte vielversprechende Ergebnisse mit einer medianen Reduktion der gesamten Polypenlast um 17% und einer Nicht-Progressionsrate von 75%. Besonders Kohorte 2 zeigte eine Nicht-Progressionsrate von 89% und eine mediane Reduktion der Polypenlast um 29% nach 12 Monaten.
FAP ist eine erbliche Erkrankung, die 1 von 5.000-10.000 Menschen in den USA und 1 von 11.300-37.600 in Europa betrifft, was zu einem nahezu 100%igen lebenslangen Risiko für kolorektalen Krebs führt. Derzeit gibt es keine zugelassenen Therapieoptionen, wobei die Chirurgie die Standardbehandlung darstellt. Die bevorstehende Phase-3-Studie wird doppelblind und placebo-kontrolliert sein und etwa 168 Hochrisiko-FAP-Patienten rekrutieren.
- FDA Fast Track designation received for eRapa
- Phase 2 trial showed 17% median decrease in polyp burden
- Cohort 2 achieved 89% non-progression rate
- Patent protection extends through 2035
- Large market potential with up to 40,000 U.S. and 60,000 European patients
- Phase 3 trials still required for approval
- Current lack of approved therapeutic options indicates challenging regulatory pathway
Insights
The FDA Fast Track designation for eRapa represents a significant milestone in addressing the critical unmet need in FAP treatment. The current standard of care - prophylactic colectomy - is highly invasive and life-altering, particularly considering FAP's early onset in teenagers. eRapa's mechanism of action through mTOR inhibition is particularly noteworthy, as mTOR overexpression in FAP polyps provides a clear biological rationale for the treatment approach.
The Phase 2 results are particularly compelling from a clinical perspective. The 89% non-progression rate in cohort 2 is remarkable for a disease that typically progresses relentlessly. The 29% median reduction in polyp burden suggests not just disease stabilization but actual regression, which is unprecedented in FAP treatment. The alternate-week dosing regimen selected for Phase 3 appears to optimize both efficacy and tolerability, important for a treatment that may require long-term administration.
The market opportunity, while modest in size with 40,000 patients in the U.S. and 60,000 in Europe, represents a concentrated patient population with a clear therapeutic need. The Fast Track designation, combined with the previous Orphan Drug status, provides significant regulatory advantages that could accelerate the path to market. The patent protection through 2035 ensures a substantial period of market exclusivity if approved.
The upcoming Phase 3 trial's design with 168 high-risk patients is well-powered to demonstrate efficacy, particularly given the strong Phase 2 signals. If the results confirm the Phase 2 findings, eRapa could fundamentally transform FAP treatment from a surgical to a medical paradigm, potentially delaying or preventing the need for colectomy. This would represent a revolutionary improvement in patient quality of life, particularly considering the young age at which FAP typically manifests.
CARDIFF, UK / ACCESS Newswire / February 12, 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, received Fast Track status from the U.S. Food and Drug Administration for eRapa, a proprietary encapsulated form of rapamycin being developed for the treatment of familial adenomatous polyposis (FAP). The news follows Biodexa reporting positive 12-month data from a phase 2 clinical trial in July 2024, as previously covered by Benzinga.
The FDA's Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions where there is an unmet medical need. FAP, an inherited condition that puts people at a much greater risk of developing colon cancer, falls into that category. The condition is typically diagnosed in the early teenage years and results in a nearly
To be considered a "serious" disease by the FDA, a condition must have a substantial impact on survival and daily functioning or the likelihood that it will worsen over time if left untreated. An unmet medical need is defined as the absence of an existing treatment or the availability of a potentially more effective treatment. Companies granted Fast Track status are able to have more frequent meetings and written communications with the FDA to discuss the drug's developmental plan, the design of proposed clinical trials and the use of biomarkers. This designation may also lead to faster drug approval.
Meeting An Unmet Need
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.
There's a significant hereditary component to FAP, with a reported incidence of one in 5,000 to 10,000 in the U.S. and one in 11,300 to 37,600 in Europe. Biodexa, which has already received U.S. FDA Orphan Drug designation for eRapa in FAP, plans to seek a similar designation in Europe.
eRapa is a proprietary oral tablet formulation of rapamycin, also known as sirolimus, which slows down the mammalian Target Of Rapamycin (mTOR) 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. eRapa has a number of patents that the company says extend through 2035.
Promising Clinical Trials
It doesn't seem surprising that Biodexa received Fast Track designation for eRapa for FAP. Data from the company's phase 2 study of eRapa in FAP showed eRapa demonstrated a
"The promising phase 2 results, if confirmed in a registrational phase 3 study, may delay or potentially obviate the need for resection of the colon and/or rectum in FAP patients," said Stephen Stamp, CEO of Biodexa. "The six-month and 12-month data together with its apparent tolerability suggest longer-term use of eRapa may be possible, with the potential to forestall resection and substantially increase the quality of life of patients with this devastating precancerous condition impacting up to 40,000 patients in the U.S. and up to 60,000 in Europe."
People suffering from FAP deserve better options than screening and surgeries. Biodexa wants to deliver that with eRapa, and with the FDA Fast Track designation, it is getting closer to giving the thousands of people suffering from this precancerous disease a better alternative.
Featured photo by farland9 on stock.adobe.com.
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 PLC
View the original press release on ACCESS Newswire
FAQ
What were the key results of BDRX's Phase 2 trial for eRapa?
How many patients will be enrolled in BDRX's Phase 3 trial for eRapa?
What is the market size for BDRX's FAP treatment?
How long does BDRX's patent protection for eRapa extend?
What benefits does FDA Fast Track status provide to BDRX?
