TransCode Therapeutics Announces Preliminary Clinical Results in First Patient in Phase 0 Clinical Study with Lead Therapeutic Candidate, TTX-MC138
24 10월 2023 - 9:00PM
TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology
company committed to more effectively treating cancer using RNA
therapeutics, today announced preliminary results with its lead
therapeutic candidate, TTX-MC138, in the first patient enrolled in
its Phase 0 clinical trial aimed at demonstrating delivery of
TTX-MC138 to metastatic cancer, including metastases beyond those
found in the liver. These preliminary data showed that
radioactivity consistent with accumulation of TTX-MC138 was
detected by noninvasive imaging in the regions of the metastatic
lesions previously identified by fluorodeoxyglucose (FDG)/positron
emission tomography (PET) (FDG/PET). In addition, radiolabeled
TTX-MC138 had pharmacokinetic behavior consistent with that
expected based on non-clinical IND-enabling studies. The patient
tolerated the dosing with no reported adverse reactions. Metabolite
analysis indicated circulation of intact radiolabeled TTX-MC138 for
more than 20 hours, equivalent to that predicted by Drug Metabolism
and Pharmacokinetics (DMPK) modelling, and that the drug candidate
analyzed in the blood was identical to that of the manufactured
drug candidate, demonstrating in vivo stability. Complete analysis
of data from this first patient is in process and will be included
in the final report for all patients enrolled in the study.
TransCode’s Chief Technology Officer, Zdravka
Medarova, PhD, commented, “We believe these preliminary clinical
data support our thesis that TTX-MC138 can be delivered
successfully to metastatic lesions for the potential treatment of
metastatic cancer. Preclinical evidence pointing towards
miRNA-10b’s critical role in metastatic progression across a number
of major cancer types suggests that inhibition of miRNA-10b in
patients with advanced disease could have a dramatic impact on
their disease.”
The Phase 0 trial is an open-label,
single-center, microdose study intended to demonstrate delivery of
the radiolabeled version of TTX-MC138 to radiographically-confirmed
metastases in subjects with advanced solid tumors. Up to 12
subjects may be enrolled in this clinical study, each of which is
expected to receive a single microdose of radiolabeled TTX-MC138
followed by positron emission tomography/magnetic resonance imaging
(PET-MRI) and blood analyses. The trial is intended to quantify the
amount of TTX-MC138 delivered to metastatic lesions, especially
beyond the liver, and the pharmacokinetics of the therapeutic
candidate in those patients. The trial is intended to yield
important data regarding TTX-MC138 delivery to clinical metastases
that could inform dose selection and frequency, for further
clinical development. The trial is not intended to demonstrate a
therapeutic effect.
In the earlier IND-enabling studies conducted in
non-human primates (NHP), TTX-MC138 demonstrated long circulation
and tissue distribution consistent with hepatic clearance. Data
from the NHP study were incorporated into a DMPK model, intended to
model the pharmacokinetics and tissue distribution of TTX-MC138 in
humans. The model predicted circulation and tissue distribution in
humans consistent with results from TransCode’s nonclinical studies
in which numerous complete regressions of metastatic disease were
observed.
TTX-MC138 consists of an iron oxide nanocarrier
conjugated to a nucleic acid specifically designed to inhibit the
oncogenic RNA, microRNA-10b. MiRNA-10b has been described as the
master regulator of cancer progression in a number of advanced
solid tumors. TransCode believes that TTX-MC138 has the potential
to become a treatment for many of these cancers. Administration of
TTX-MC138 has demonstrated complete regression of metastatic
disease in a number of mouse models of pancreatic and breast
cancer. In addition, TTX-MC138 was successfully delivered and
demonstrated bioactivity in a case study of spontaneous feline
mammary carcinoma.
“Our Phase 0 trial involves a single microdose
of radiolabeled TTX-MC138 followed by noninvasive PET-MRI imaging
and metabolite analysis. Given the similarities between humans and
non-human primates relative to anatomy, physiology, and molecular
biology, we anticipated results in trial patients comparable to
those observed in the DMPK model based on our NHP studies, as
evidenced by the preliminary data we announced today,” added
Michael Dudley, Chief Executive Officer of TransCode.
This study was done in collaboration with
Andreas Varkaris, MD, PhD, an attending physician and investigator
for the Termeer Center for Targeted Therapies at Massachusetts
General Hospital and the principal investigator of TransCode’s
study.
About TransCode
Therapeutics
TransCode is an RNA oncology company created on
the belief that cancer can be more effectively treated using RNA
therapeutics. Using its iron oxide nanoparticle delivery platform,
the Company has created a portfolio of drug candidates designed to
target a variety of tumor types with the objective of significantly
improving patient outcomes. The Company’s lead therapeutic
candidate, TTX-MC138, is focused on treating metastatic cancer,
which is believed to cause approximately 90% of all cancer deaths
totaling over nine million per year worldwide. The Company believes
that TTX-MC138 has the potential to dramatically improve clinical
outcomes in a range of cancers, including breast, pancreatic,
ovarian and colon cancer, glioblastomas and others. Another of the
Company’s drug candidates, TTX-siPDL1, focuses on treating tumors
by targeting a protein called Programmed death-ligand 1 (PD-L1).
TransCode also has three cancer-agnostic programs: TTX-RIGA, an
RNA–based agonist of the retinoic acid-inducible gene I designed to
drive an immune response in the tumor microenvironment; TTX-CRISPR,
a CRISPR/Cas9–based therapy platform for the repair or elimination
of cancer-causing genes inside tumor cells; and TTX-mRNA, an
mRNA-based platform for the development of cancer vaccines designed
to activate cytotoxic immune responses against tumor cells.
Forward-Looking Statements
This release contains “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995, including, without limitation, statements
concerning preliminary first patient results of the Phase 0
clinical trial of radiolabeled TTX-MC138, statements concerning
expected clinical results of TransCode’s therapeutic candidates,
statements concerning the results of RNA research, statements
concerning the potential for treating cancer with RNA therapeutics,
statements concerning the timing and outcome of expected regulatory
filings and clinical trials, including the current first-in-human
study of TTX-MC138, and whether this study will demonstrate
proof-of-mechanism, and statements concerning TransCode’s portfolio
of drug candidates and TTX technology platform generally. Of note,
a Phase 0 clinical trial is an exploratory study, conducted under
an exploratory Investigational New Drug (eIND) application.
Exploratory IND studies usually involve very limited human exposure
to a therapeutic candidate to evaluate mechanism of action in order
to inform potential clinical evaluation in future clinical studies,
but otherwise have no therapeutic intent. Further, caution should
be taken when interpreting the preliminary results of the Phase 0
trial. This data may differ from future results of this study, or
different conclusions or considerations may qualify such results,
once additional data have been received and fully evaluated.
Preliminary data also remains subject to audit and verification
procedures that may result in the final data being materially
different from the preliminary data previously published. Any
forward-looking statements in this press release are based on
management’s current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements. These risks and
uncertainties include, but are not limited to: the risk associated
with drug discovery and development; the risk that the results of
clinical trials we conduct will not be consistent with our
pre-clinical studies or expectations; risks associated with the
timing and outcome of TransCode’s planned regulatory submissions;
risks associated with TransCode’s planned clinical trials for its
product candidates; risks associated with obtaining, maintaining
and protecting intellectual property; risks associated with
TransCode’s ability to enforce its patents against infringers and
defend its patent portfolio against challenges from third parties;
the risk of competition from other companies developing products
for similar uses; risks associated with TransCode’s financial
condition and its need to obtain additional funding to support its
business activities, including TransCode’s ability to continue as a
going concern; risks associated with TransCode’s dependence on
third parties; and risks associated with the COVID-19 coronavirus.
For a discussion of these and other risks and uncertainties, and
other important factors, any of which could cause TransCode’s
actual results to differ from those contained in or implied by the
forward-looking statements, see the section entitled “Risk Factors”
in TransCode’s Annual Report on Form 10-K for the year ended
December 31, 2022, as well as discussions of potential risks,
uncertainties and other important factors in any subsequent
TransCode filings with the Securities and Exchange Commission. All
information in this press release is as of the date of the release;
TransCode undertakes no duty to update this information unless
required by law.
For more information, please
contact:
TransCode Therapeutics, Inc.Alan Freidman, VP Investor
Relationsalan.freidman@transcodetherapeutics.com
TransCode Therapeutics (NASDAQ:RNAZ)
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TransCode Therapeutics (NASDAQ:RNAZ)
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