(“Biodexa” or the “Company”)
Positive Statistically Significant Phase 2 Clinical Trial Results of Biodexa’s Newly-licensed eRapa™ in Familial Adenomatous Polyposis (FAP) Scheduled for Presentation at the
2024 Digestive Disease Week Annual Meeting
Overall 83% non-progression rate at 6 Months
Statistically Significant decrease in overall mean polyp burden at 6 months (p=0.04)
The 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. 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. Polyp outcomes were classified into Progressive Disease (PD: >20% increase in polyp burden), Stable Disease (SD: ± 20% change in polyp burden) or Partial Response (PR: >20% reduction in polyp burden). A combination endpoint of non-progressors included SD and PR.
Results of the Phase 2 study at six months
In the duodenum, 14/18 (78%) patients were non-progressors with 11/18 (61%) of these patients with PR. In the colorectum, 25/29 (86%) patients were non progressors including all with an intact colon; of these 15/29 (67%) patients demonstrated PR including 4 with an intact colon (see Figure 1 below). Only two drug-related Grade 3 Serious Adverse Events occurred during the trial (with no Grade 4 or 5 reported), and 97% of patients remained on treatment at six months.
In summary, eRapa appeared safe and well-tolerated with a significant 24% reduction in the total polyp burden at six months compared with baseline (p=0.04) and an overall 83% non-progression rate.
Polyp burden data at 12 months compared with baseline will be presented at the InSIGHT scientific conference in
Figure 1.
Waterfall plots showing % change in duodenal and colorectal polyp burden after six months
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
eRapa license
On
For more information, please contact:
Tel: +44 (0)29 20480 180 www.biodexapharma.com |
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
Source:
2024 GlobeNewswire, Inc., source