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)是一家以收购为重点的临床阶段生物制药公司,开发一系列针对未满足医疗需求病症的创新产品管线,正在取得eRapa药物治疗家族性腺瘤性息肉病(FAP)方面的良好进展。
FAP是一种大多数为遗传状况的疾病,会让人们更容易罹患结肠癌。最近,来自第二期临床试验的12个月积极数据在2024年巴塞罗那的InSIGHt双年会上发表。
在FAP中,数百甚至数千个形成癌前病变的息肉在胃肠道内生长。目前还没有批准用于治疗FAP患者的治疗方法——对于FAP患者来说,积极监测和结肠/直肠的手术切除仍然是标准治疗方法。FAP通常在患者的中年期出现,患者最终需要切除全部结肠/直肠,并在余生中佩戴结肠造口袋。如果不治疗,这种疾病将导致百分之百的结肠癌风险。
治疗FAP的更好方法
虽然对于患有FAP的全球10万多人来说,多次筛查和手术已经成为他们的生活方式,但它不必一直保持这种方式。
Biodexa认为,eRapa是治疗这种癌前病变的第一种治疗方法,其迄今为止的数据支持了这一评估。eRapa 12个月第二期临床试验结果显示,总息肉负荷中位数降低17%,总非进展率为75%。更为引人注目的是,第2组(每个星期交替治疗每日一次的患者)中,12个月中被评定为非进展患者的患者达到89%,息肉负荷中位数降低29%。如果这意味着FAP患者需要更少的手术且生活质量得到显著提高,那么这将可为他们改变局面。12个月的数据表明了eRapa的持续效应。
“如果注册第三期研究证实了这一令人充满期望的第二期结果,将可能推迟或潜在的省去对FAP患者结肠/直肠的切除,” Biodexa首席执行官Stephen Stamp表示,“eRapa的六个月及现在的12个月数据,连同其明显的耐受性表明,似乎可以长期使用eRapa,从而有助于推迟结肠/直肠的切除,并显著提高FAP患者的生活质量。这种疾病影响了美国高达4万名患者和欧洲高达6万名患者。”
eRapa是异维甲酸的专有口服片剂,也称为西罗莫司,它可以减缓哺乳动物靶蛋白(mTOR)m哺乳动物T目标O的R西罗莫司蛋白。mTOR表达过多与癌症有关。
阻止癌症的进展
针对30名FAP成年患者的第二期开放标签研究在美国七个卓越中心进行。试验参与者的中位年龄为43岁,拥有未切除或部分切除结肠,并在直肠残留物中至少发现10个非癌性肿瘤。患者被分为3个剂量组,包括每隔一天、每周隔一天和每天给药。虽然主要终点是eRapa的安全性和耐受性以及6个月基线息肉负荷的百分比变化,但患者继续接受治疗和监测12个月,因此产生了新的数据。
Biodexa表示,对于Cohort 2(每周交替治疗每日一次)给予的剂量将很可能成为其即将启动的第III期试验的首选药量。该研究计划招募约140名患有生殖系或表现型FAP的高风险患者,并设计为双盲安慰剂对照。
Biodexa的第2期结果由卡罗尔·伯克(Carol Burke)博士在InSIGHt上发表,她是克利夫兰诊所的专业胃肠疾病学家,也是FAP的领先权威。伯克是第2期研究和即将开始的第3期研究的主要研究员。第二期试验得到德克萨斯州癌症预防和研究协会(CPRIT)300万美元的资助。CPRIt为第3期试验提供了1700万美元的资助。
尽管FAP可能很少见,但对于成千上万的患病人士来说,必须有更好的治疗方法。 Biodexa认为eRapa就是这样一种方法。第2期试验结果似乎支持了这一点,随着第3期试验的开始,FAP患者可能有了希望。
联系方式:
Stephen Stamp,首席执行官和财务官
ir@biodexapharma.com
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SOURCE: Biodexa Pharmaceuticals PLC