Twelve month Phase 2 Clinical Trial Results of eRapa™ in Familial Adenomatous Polyposis (FAP) to be Presented at Prestigious Biennial InSIGHT 2024 Meeting in Barcelona
June 10 2024 - 8:30AM
June 10, 2024
Biodexa Pharmaceuticals
PLC(“Biodexa” or the “Company”)
Twelve month Phase 2 Clinical Trial
Results of eRapa™ in Familial Adenomatous Polyposis (FAP) to be
Presented at Prestigious Biennial InSIGHT 2024 Meeting in
Barcelona
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, announces that 12 month data of
a Phase 2 clinical trial of eRapa in Familial Adenomatous Polyposis
(“FAP”) are to be presented at the prestigious biennial InSIGHT
2024 meeting in Barcelona, Spain on June 22, 2024. Carol Burke, MD,
the Principal Investigator, will present the six and 12 month data
(NCT04230499) in an oral presentation at the meeting. Dr Burke
presented the six-month data at the Digestive Disease Weekly
meeting in Washington D.C. on May 21, 2024.
eRapa in FAPFAP is
characterized as a proliferation of polyps in the GI tract, 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.
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. 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.
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.
1. Tian et al., mTOR
Signaling 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
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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