Biodexa Announces Positive Phase 2 Clinical Trial Results of eRapa™ at 12 months in Familial Adenomatous Polyposis (FAP)
Overall 75% Non-progression Rate at 12 Months
Median Decrease in Overall Polyp Burden at 12 Months of 17%
Cohort 2 Non-progression Rate at 12 months of 81% and Median Decrease in Poly Burden of 29% Informs Phase 3 Dosage Regimen
Data Reported in Oral Presentation at 2024 Bi-Annual InSIGHT Meeting
(DATELINE)
The Phase 2 study was partially supported by
Design of the Phase 2 study
The open-label study was conducted in seven
Patients were sequentially enrolled into three dosing cohorts of 10 patients each for a 12-month treatment period: 0.5mg every other day (Cohort 1), 0.5mg daily every other week (Cohort 2), and 0.5mg daily (Cohort 3). Upper and lower endoscopic surveillance occurred at baseline and after six months. Although the primary endpoints were safety and tolerability of eRapa and percentage change from baseline in polyp burden at six months, as measured by the aggregate of all polyp diameters, patients continued to receive treatment and monitoring for 12 months (secondary endpoint).
Results of the Phase 2 study at 12 months
Overall, 21 of 28 (75%) patients were deemed to be non-progressors at 12 months with a median reduction in polyp burden of 17%.
In Cohort 2, eight of nine (89)% of patients were deemed non-progressors at 12 months with a median reduction in polyp burden of 29%.
Over the course of 12 months, there were four related Grade 3 or higher and one related Serious Adverse Event reported during the trial and 95% compliance rate at 12 months. One patient was removed from the trial due to non-compliance.
In summary, eRapa appeared safe and well-tolerated with a median 17% reduction in the total polyp burden at 12 months compared with baseline and an overall 75% non-progression rate. The 89% non-progression rate and 29% median reduction in polyp burden in Cohort 2 is likely to be the preferred dosage regimen for Phase 3.
Polyp burden data at six months compared with baseline was presented at the
Planned Phase 3 registrational study of eRapa in FAP
The Phase 3 registrational study is planned to be a double-blind placebo-controlled design recruiting approximately 140 high risk patients diagnosed with germline or phenotypic FAP. The primary clinical endpoint is likely to be the first progression free survival event which will include major surgery (or referral thereto), polypectomy for advanced neoplasia, advancement of Spigelman stage, diagnosis of high-grade dysplasia or cancer and death from any cause. A
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 tumorgenesis1. 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.
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 typically leads to cancer of the colon and/or rectum. There is a significant hereditary component to FAP with a reported incidence of one in 5,000 to 10,000 in the US2 and one in 11,300 to 37,600 in
About the
CPRIT was created by the
1. Tian et al., mTOR Signalling in Cancer and mTOR Inhibitors in Solid Tumor Targeting Therapy, Int J Mol Sci. 2019 Feb; 20(3): 755
2. www.rarediseases.org
3. www.orpha.net
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About
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 tumorgenesis.
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 glycemic 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 solubilised 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
Forward-Looking Statements
Certain statements in this announcement may constitute “forward-looking statements” within the meaning of legislation in the
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
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