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 83% non-progression rate at 6
Months
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 $20M in
grant funding from the Cancer Prevention and Research Institute of
Texas under product development awards DP22053 and DP190069.
Design of the Phase 2 studyThe
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: >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
monthsIn 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
Barcelona on June 19-22, 2024.
Figure 1.Waterfall plots showing % change in
duodenal and colorectal polyp burden after six months
About eRapaeRapa 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 FAPFAP 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 TexasCPRIT 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 $3
billion for a total $6 billion investment in cancer research and
prevention. To date, CPRIT has awarded over $3 billion in grants to
Texas research institutions and organizations through its academic
research, prevention and product development research programs.
CPRIT has also recruited more than 281 distinguished researchers to
Texas, supported the establishment, expansion or relocation of 51
companies to Texas and generated over $7.66 billion in additional
public and private investment. CPRIT funding has advanced
scientific and clinical knowledge and provided over 8.1 million
life-saving cancer prevention and early detection services to
Texans in all 254 counties. Learn more
at https://cprit.texas.gov.
1. Tian et al., mTOR
Signalling in Cancer and mTOR Inhibitors in Solid Tumor Targeting
Therapy, Int J Mol Sci. 2019 Feb; 20(3):
7552. www.rarediseases.org3. www.orpha.net
eRapa licenseOn 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, CFOTel: +44 (0)29 20480
180www.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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