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
Biodexa Pharmaceuticals (Nasdaq: BDRX) announced positive Phase 2 clinical trial results of its new drug eRapa for treating Familial Adenomatous Polyposis (FAP). The trial, involving 30 adult patients at seven U.S. centers, showed an 83% non-progression rate at six months and a statistically significant 24% reduction in overall mean polyp burden (p=0.04). The study was partially funded by a $20M grant from the Cancer Prevention and Research Institute of Texas. The results will be presented at the 2024 Digestive Disease Week annual meeting. The drug appeared safe and well-tolerated, with 97% of patients remaining on treatment at six months.
- 83% non-progression rate at six months.
- Statistically significant 24% reduction in overall mean polyp burden (p=0.04).
- 97% of patients remained on treatment at six months.
- Only two drug-related Grade 3 Serious Adverse Events occurred.
- $20M grant funding from the Cancer Prevention and Research Institute of Texas.
- Scheduled presentation at the prestigious 2024 Digestive Disease Week annual meeting.
- Only 30 patients were involved in the trial, which may limit generalizability.
- Two drug-related Grade 3 Serious Adverse Events occurred.
- Pending 12-month data, which may change the overall outcome.
Insights
eRapa™'s Phase 2 clinical trial results are quite promising for patients with Familial Adenomatous Polyposis (FAP), a condition that significantly increases colorectal cancer risk. The standout data point is the 83% non-progression rate at six months, including a significant 24% reduction in total polyp burden. This is particularly encouraging because FAP patients face a high baseline risk of cancer development and typically require aggressive surveillance and intervention.
Given that 61% and 67% of patients in the duodenum and colorectum cohorts, respectively, demonstrated a partial response (> 20% reduction in polyp burden), these results are highly relevant. The trial's safety profile is also noteworthy with only two Grade 3 serious adverse events and no Grade 4 or 5 events reported, suggesting that eRapa™ is well-tolerated.
Why does this matter? It indicates that eRapa™ could potentially become a key therapeutic option for managing FAP, reducing the need for invasive procedures like colectomies. This could drastically improve the quality of life and long-term health outcomes for these patients. Moreover, the significant grant funding from the Cancer Prevention and Research Institute of Texas adds another layer of credibility to this research.
The positive Phase 2 results for Biodexa's eRapa™ are likely to have a noticeable impact on the company's stock. As an acquisition-focused biopharmaceutical company, Biodexa's success in bringing a statistically significant product through clinical trials could attract interest from larger pharmaceutical companies, potentially driving up its market valuation.
From a financial perspective, the support from a
Short-term vs. Long-term: In the short term, these results could lead to a stock price surge due to positive market sentiment. Over the long term, successful commercialization of eRapa™ could provide a stable revenue stream and significantly improve Biodexa's financial stability. However, investors should remain cautious about potential risks, including the outcomes of further trials and regulatory approvals, which could affect long-term projections.
May 21, 2024
Biodexa Pharmaceuticals PLC
(“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
Statistically Significant decrease in overall mean polyp burden at 6 months (p=0.04)
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 pleased to announce that results of a Phase 2 clinical trial of eRapa in Familial Adenomatous Polyposis (“FAP”) (NCT04230499) are scheduled for presentation at the prestigious 2024 Digestive Disease Week annual meeting in Washington D.C.. Carol Burke, MD, the Principal Investigator, will present the six month data in a podium presentation at the meeting.
The study was partially supported by
Design of the Phase 2 study
The open-label study was conducted in seven U.S. centers of excellence in 30 adult patients with median age of 43 years with intact colon (n=6) or post-colectomy and ileo-rectal anastomosis and at least 10 adenomas in the rectal remanent (n=24).
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: >
Results of the Phase 2 study at six months
In the duodenum, 14/18 (
In summary, eRapa appeared safe and well-tolerated with a significant
Polyp burden data at 12 months compared with baseline will be presented at the InSIGHT scientific conference in Barcelona on June 19-22, 2024.
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 Europe3. eRapa has received Orphan Designation in the US with plans to seek such designation in Europe. Importantly, mTOR has been shown to be over-expressed in FAP polyps – thereby underscoring the rationale for using a potent and safe mTOR inhibitor like eRapa to treat FAP.
About the Cancer Prevention and Research Institute of Texas
CPRIT was created by the Texas Legislature and approved by a statewide vote in 2007 to lead the Lone Star State’s fight against cancer. In 2019, Texas voters again voted overwhelmingly to continue CPRIT with an additional
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 April 26, 2024, Biodexa announced that it had entered into a definitive agreement with Emtora Biosciences, Inc. for the rights to eRapa under an exclusive, worldwide license (with the ability to grant sublicenses) to develop, manufacture, commercialize and otherwise advance the clinical potential of eRapa.
For more information, please contact:
Biodexa Pharmaceuticals PLC |
| Stephen Stamp, CEO, CFO Tel: +44 (0)29 20480 180 www.biodexapharma.com |
About Biodexa Pharmaceuticals PLC
Biodexa Pharmaceuticals PLC (listed on NASDAQ: BDRX) is a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs. The Company’s lead development programs include eRapa, under development for Familial Adenomatous Polyposis and Non Muscle Invasive Blader Cancer: tolimidone, under development as a for the treatment of type 1 diabetes; and MTX110, which is being studied in aggressive rare/orphan brain cancer indications.
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 Cardiff, UK. For more information visit www.biodexapharma.com.
Forward-Looking Statements
Certain statements in this announcement may constitute “forward-looking statements” within the meaning of legislation in the United Kingdom and/or United States. Such statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and are based on management’s belief or interpretation. All statements contained in this announcement that do not relate to matters of historical fact should be considered forward-looking statements. In certain cases, forward-looking statements can be identified by the use of words such as “plans”, “expects” or “does not anticipate”, or “believes”, or variations of such words and phrases or statements that certain actions, events or results “may”, “could”, “would”, “might” or “will be taken”, “occur” or “be achieved.” Forward-looking statements and information are subject to various known and unknown risks and uncertainties, many of which are beyond the ability of the Company to control or predict, that may cause their actual results, performance or achievements to be materially different from those expressed or implied thereby, and are developed based on assumptions about such risks, uncertainties and other factors set out herein.
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 SEC, which contain and identify other important factors that could cause actual results to differ materially from those contained in any projections or forward-looking statements. These forward-looking statements speak only as of the date of this announcement. All subsequent written and oral forward-looking statements by or concerning Biodexa are expressly qualified in their entirety by the cautionary statements above. Except as may be required under relevant laws in the United States, Biodexa does not undertake any obligation to publicly update or revise any forward-looking statements because of new information, future events or events otherwise arising.
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