Follows Safety Review Committee (SRC) approval based on safety
data from patients in Cohorts 1 and 2
- No significant safety or dose limiting toxicities reported
in Cohorts 1 and 2
- PK data from Cohorts 1 and 2 consistent with preclinical and
Phase 0 trial results
BOSTON, Jan. 14,
2025 /PRNewswire/ -- TransCode Therapeutics, Inc.
(NASDAQ: RNAZ), the RNA oncology company committed to more
effectively treating cancer using RNA therapeutics, today announced
that the first patient in Cohort 3 of its Phase 1 clinical trial
has been dosed with TTX-MC138, its lead candidate. The Safety
Review Committee monitoring the clinical trial unanimously approved
opening of the third cohort based on its favorable review of Cohort
1 and 2 safety and pharmacokinetic (PK) data. Additional
Cohort 3 patients have been scheduled. The dose administered to
patients in the third cohort is approximately double the dose
administered to those in the second cohort.
Several patients in the first and second cohort remain on study
for continued treatment, receiving additional doses of TTX-MC138.
No significant safety or dose limiting toxicities have been
reported. Analyses of PK data and pharmacodynamic (PD)
activity from Cohorts 1 and 2 is ongoing. To date, the analyses
suggest that TTX-MC138 demonstrates a PK/PD profile consistent with
preclinical results and results from TransCode's previous Phase 0
clinical trial. Specifically, results from Cohort 1 confirmed
the Phase 0 observation that TTX-MC138 shows evidence of
pharmacodynamic activity in the presence of high baseline
expression of miR-10b, reaching a 66% inhibition at 24 hours after
infusion, similar to that seen in the Phase 0 trial. Additionally,
TTX-MC138 activity increased with the escalated dose administered
in Cohort 2 and was consistent at subsequent administrations of
TTX-MC138, suggesting a favorable pharmacokinetic profile.
"An SRC is a group of clinicians and other experts that
oversee patient safety during a clinical trial. The SRC
determines whether and how a study should proceed, including dose
escalation and de-escalation decisions in accordance with the study
design. Enrollment into this study continues based on the SRC's
cumulative safety data review. We are very pleased with the
commitment from our clinical sites which may enable quick
completion of the third cohort," commented Sue Duggan, TransCode's Senior Vice President of
Operations.
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate designed to
inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be
critical to the emergence and progression of many metastatic
cancers. TransCode's 2023 Phase 0 clinical trial produced evidence
of delivery of a radiolabeled version of TTX-MC138 to metastatic
lesions and pharmacodynamic activity, even at a microdose of the
drug candidate, suggesting a broad therapeutic window for
TTX-MC138.
About the Trial
TransCode's Phase 1 clinical trial is a multicenter, open-label,
dose-escalation and dose-expansion study designed to generate
critical data to support evaluation of the safety and tolerability
of TTX-MC138 in patients with a variety of metastatic solid
cancers. While not an endpoint, the trial may provide early
evidence of clinical activity of TTX-MC138. The trial comprises an
initial dose-escalation stage followed by a dose-expansion stage.
The primary objective of the dose-escalation stage is to evaluate
the safety and tolerability of escalating dose levels of TTX-MC138.
In the dose-expansion stage, the safety, tolerability and
anti-tumor activity of TTX-MC138 will be further evaluated in
certain tumor types selected based on preliminary results from the
dose-escalation phase.
Further information is available at www.clinicaltrials.gov
NCT Identifier: (NCT06260774).
About TransCode Therapeutics
TransCode is a clinical-stage oncology company focused on
treating metastatic disease. The company is committed to defeating
cancer through the intelligent design and effective delivery of RNA
therapeutics based on its proprietary TTX nanoparticle platform.
The company's lead therapeutic candidate, TTX-MC138, is focused on
treating metastatic tumors which overexpress microRNA-10b, a
unique, well-documented biomarker of metastasis. In addition,
TransCode has a portfolio of other first-in-class RNA therapeutic
candidates designed to overcome the challenges of RNA delivery and
thus unlock therapeutic access to a variety of novel genetic
targets that could be relevant to treating a variety of
cancers.
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 the timing,
conduct and results of the Phase 1 clinical trial, statements about
microRNAs and their involvement in cancer, and statements
concerning the therapeutic potential of TransCode's TTX-MC138 and
other therapeutic candidates. 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 risks associated with drug discovery
and development; the risk that the results of clinical trials will
not be consistent with TransCode's preclinical studies or
expectations or with results from previous clinical trials; risks
associated with the conduct of clinical trials; 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 the timing and outcome of TransCode's planned
regulatory submissions; 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; risks of competition from other companies developing
products for similar uses; risks associated with TransCode's
dependence on third parties; and risks associated with geopolitical
events and pandemics, including the COVID-19 coronavirus and
military actions. 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, 2023,
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 this release; TransCode undertakes no
duty to update this information unless required by law.
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SOURCE TransCode Therapeutics, Inc.