Approval given after Safety Review Committee (SRC) review of
safety data from the three patients comprising Cohort 2
- No significant safety or dose limiting toxicities reported in
Cohort 2
- New patients currently being evaluated for eligibility in
Cohort 3
- PK and PD data from Cohort 1 patients consistent with
preclinical and Phase 0 trial results
BOSTON, Dec. 18,
2024 /PRNewswire/ -- TransCode Therapeutics, Inc.
(NASDAQ: RNAZ), the RNA oncology company committed to more
effectively treating cancer using RNA therapeutics, today announced
that the Safety Review Committee (SRC) monitoring its Phase 1
clinical trial has unanimously approved opening of the third cohort
of patients based on its favorable review of Cohort 2 safety data.
The therapeutic candidate being evaluated, TTX-MC138, is
TransCode's lead candidate designed to inhibit microRNA-10b, or
miR-10b, a microRNA critical to the emergence and progression of
many metastatic cancers. The dose administered to the third
cohort will be approximately double the dose administered to the
second cohort.
Several patients in the first and second cohort remain on study
for continued treatment. No significant safety or dose
limiting toxicities have been reported. Analysis of Cohort 1
data for pharmacokinetic (PK) and pharmacodynamic (PD) activity is
ongoing and to date suggests that TTX-MC138 demonstrates a PK/PD
profile consistent with preclinical results and results from the
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. Additionally, the
concentration of TTX-MC138 in blood plasma as a function of dose in
humans was found to be higher than achieved in nonclinical studies,
suggesting a favorable pharmacokinetic profile.
"An SRC is a group of clinicians and experts that oversee
patient safety during the conduct of 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. The recommendations of the SRC are used to
decide whether a clinical trial should be continued as designed,
changed, or terminated," commented Sue
Duggan, TransCode's Senior Vice President of Operations.
Duggan added, "Enrollment into the study continues based on the
cumulative safety data review. Eligible patients may now be
screened and scheduled in Cohort 3 for treatment with the next dose
level of TTX-MC138 while preliminary data analysis
continues."
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate that targets
microRNA-10b, a microRNA widely believed to be a driver of
metastatic disease. 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 phase followed by a dose-expansion phase.
The primary objective of the dose-escalation phase is to evaluate
the safety and tolerability of escalating dose levels of TTX-MC138.
In the dose-expansion phase, 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 is developing 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. 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.
For more information, please contact:
TransCode Therapeutics, Inc.
Tania Montgomery-Hammon, VP of
Business Development
tania.montgomery@transcodetherapeutics.com
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SOURCE TransCode Therapeutics, Inc.