CARDIFF, UK / ACCESSWIRE / July 11, 2024 / Biodexa Pharmaceuticals PLC (NASDAQ:BDRX), an acquisition-focused clinical-stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, is making good progress with eRapa, its drug to treat Familial Adenomatous Polyposis (FAP).
FAP is a mostly inherited condition that puts people at a much greater risk of developing colorectal cancer. Positive 12-month data from a phase 2 clinical trial were recently presented at the 2024 InSIGHT biannual meeting in Barcelona.
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 and/or rectum resected and having to carry a colostomy bag for the remainder of their lives . If left untreated, there is a 100% chance the person will develop colorectal cancer.
A Better Way Of Treating FAP
While multiple screenings and surgeries are a way of life for the more than 100,000 people worldwide who suffer from this condition, it doesn't have to remain that way.
Biodexa believes eRapa could be the first therapeutic option to treat this precancerous condition, and its data so far backs up that assessment. Results of a 12-month phase 2 clinical trial of eRapa demonstrated an overall 17% median decrease in overall polyp burden and an overall non-progression rate of 75%. Even more compelling, of patients in Cohort 2 (treated daily, alternate weeks), 89% of patients were deemed non-progressors at 12 months, with a median reduction in polyp burden of 29%. That could be gaming-changing for FAP patients if it means fewer surgeries with much improved quality of life. The 12-month data demonstrate a longevity of effect of eRapa.
"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 now 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."
eRapa is 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.
Stopping It In Its Tracks
The phase 2 open-label study of 30 adults with FAP was conducted in seven U.S. centers of excellence. The median age of the trial participants was 43 years, with either an intact colon or one with a portion removed and at least ten noncancerous tumors in the rectal remnant. Patients were enrolled in three dosing cohorts, including every other day, daily every other week and daily. Although the primary endpoints were safety and tolerability of eRapa and percentage change from baseline in polyp burden at six months, patients continued to receive treatment and monitoring for 12 months, leading to the new data.
Biodexa said the dosing given to cohort 2 - daily every other week - will likely be the preferred dosage regime for its phase 3 trial, which the company is gearing up to launch soon. That study is planned to be a double-blind placebo-controlled design recruiting approximately 140 high-risk patients diagnosed with germline or phenotypic FAP.
Biodexa's phase 2 results were presented at InSIGHT by Carol Burke, M.D., a specialist gastroenterologist at the Cleveland Clinic and a leading authority in FAP. Burke is the Principal Investigator for both the phase 2 study and the upcoming phase 3 study. The phase 2 trial was partially supported by $3 million in grant funding from the Cancer Prevention and Research Institute of Texas (CPRIT). CPRIT is providing a $17 million grant for the phase 3 trial.
FAP may be rare, but for the tens of thousands of people suffering from this precancerous condition, there's got to be a better treatment. Biodexa believes it has that with eRapa. The phase 2 trial results seem to lean that way, and with a phase 3 study about to kick off, there may be hope on the horizon for FAP patients.
Contact:
Stephen Stamp, CEO, CFO
ir@biodexapharma.com
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SOURCE: Biodexa Pharmaceuticals PLC
Biodexa Pharmaceuticals PLC(NASDAQ:BDRX)是一家以收購爲重點的臨床階段生物製藥公司,開發一系列針對未滿足醫療需求病症的創新產品管線,正在取得eRapa藥物治療家族性腺瘤性息肉病(FAP)方面的良好進展。
FAP是一種大多數爲遺傳狀況的疾病,會讓人們更容易罹患結腸癌。最近,來自第二期臨床試驗的12個月積極數據在2024年巴塞羅那的InSIGHt雙年會上發表。
在FAP中,數百甚至數千個形成癌前病變的息肉在胃腸道內生長。目前還沒有批准用於治療FAP患者的治療方法——對於FAP患者來說,積極監測和結腸/直腸的手術切除仍然是標準治療方法。FAP通常在患者的中年期出現,患者最終需要切除全部結腸/直腸,並在餘生中佩戴結腸造口袋。如果不治療,這種疾病將導致百分之百的結腸癌風險。
治療FAP的更好方法
雖然對於患有FAP的全球10萬多人來說,多次篩查和手術已經成爲他們的生活方式,但它不必一直保持這種方式。
Biodexa認爲,eRapa是治療這種癌前病變的第一種治療方法,其迄今爲止的數據支持了這一評估。eRapa 12個月第二期臨床試驗結果顯示,總息肉負荷中位數降低17%,總非進展率爲75%。更爲引人注目的是,第2組(每個星期交替治療每日一次的患者)中,12個月中被評定爲非進展患者的患者達到89%,息肉負荷中位數降低29%。如果這意味着FAP患者需要更少的手術且生活質量得到顯著提高,那麼這將可爲他們改變局面。12個月的數據表明了eRapa的持續效應。
“如果註冊第三期研究證實了這一令人充滿期望的第二期結果,將可能推遲或潛在的省去對FAP患者結腸/直腸的切除,” Biodexa首席執行官Stephen Stamp表示,“eRapa的六個月及現在的12個月數據,連同其明顯的耐受性表明,似乎可以長期使用eRapa,從而有助於推遲結腸/直腸的切除,並顯著提高FAP患者的生活質量。這種疾病影響了美國高達4萬名患者和歐洲高達6萬名患者。”
eRapa是異維甲酸的專有口服片劑,也稱爲西羅莫司,它可以減緩哺乳動物靶蛋白(mTOR)m哺乳動物T目標O的R西羅莫司蛋白。mTOR表達過多與癌症有關。
阻止癌症的進展
針對30名FAP成年患者的第二期開放標籤研究在美國七個卓越中心進行。試驗參與者的中位年齡爲43歲,擁有未切除或部分切除結腸,並在直腸殘留物中至少發現10個非癌性腫瘤。患者被分爲3個劑量組,包括每隔一天、每週隔一天和每天給藥。雖然主要終點是eRapa的安全性和耐受性以及6個月基線息肉負荷的百分比變化,但患者繼續接受治療和監測12個月,因此產生了新的數據。
Biodexa表示,對於Cohort 2(每週交替治療每日一次)給予的劑量將很可能成爲其即將啓動的第III期試驗的首選藥量。該研究計劃招募約140名患有生殖系或表現型FAP的高風險患者,並設計爲雙盲安慰劑對照。
Biodexa的第2期結果由卡羅爾·伯克(Carol Burke)博士在InSIGHt上發表,她是克利夫蘭診所的專業胃腸疾病學家,也是FAP的領先權威。伯克是第2期研究和即將開始的第3期研究的主要研究員。第二期試驗得到德克薩斯州癌症預防和研究協會(CPRIT)300萬美元的資助。CPRIt爲第3期試驗提供了1700萬美元的資助。
儘管FAP可能很少見,但對於成千上萬的患病人士來說,必須有更好的治療方法。 Biodexa認爲eRapa就是這樣一種方法。第2期試驗結果似乎支持了這一點,隨着第3期試驗的開始,FAP患者可能有了希望。
聯繫方式:
Stephen Stamp,首席執行官和財務官
ir@biodexapharma.com
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SOURCE: Biodexa Pharmaceuticals PLC