- 15 patients treated across four escalating dose levels of
TTX-MC138
- No significant safety or dose limiting toxicities reported
- 10 patients remain on study with no evidence of disease
progression
- PD analysis at 24 hours post-dosing provides evidence of
miR-10b target engagement
BOSTON, May 8, 2025
/PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the
RNA oncology company committed to more effectively treating cancer
using RNA therapeutics, today announced that the third patient in
Cohort 4 of its Phase 1a clinical trial has received their initial
dose of TTX-MC138. All cohorts have enrolled at least three
patients who have been dosed with TTX-MC138 at least once. The
Safety Review Committee monitoring the clinical trial unanimously
approved opening the fourth cohort based on its review of available
safety and pharmacokinetic (PK) data. Additionally, the Safety
Review Committee approved expanded enrollment in Cohort 3 to obtain
additional safety data. To date, 15 patients have received at least
one dose of TTX-MC138 at 4 separate dose levels ranging from 0.8
mg/kg to 4.8 mg/kg. Three patients have been treated in the
expanded enrollment.
Ten patients remain on study for continued treatment, receiving
an additional dose of TTX-MC138 during each treatment cycle every
28 days, and may remain on study absent any significant safety
observations or disease progression. Two patients who have remained
on study the longest have received so far seven doses of TTX-MC138
over approximately seven months and have demonstrated stable
disease. The 10 patients currently on study have shown no disease
progression. Further, no significant safety or dose limiting
toxicities have been reported in any of the trial's 15 patients.
Analysis of PK activity from Cohorts 1, 2 and 3 is ongoing and
suggests that TTX-MC138 demonstrates a PK/PD profile consistent
with preclinical results and results from TransCode's Phase 0
clinical trial. Specifically, preliminary PK and pharmacodynamic
(PD) data follow a predictable dose-response relationship. Analysis
of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated
with doses of 0.8 mg/kg and 1.6 mg/kg, respectively, demonstrates
miR-10b target engagement at 24 hours post-infusion.
The observed tolerability profile and the available PK/PD
results thus far support advancement of the clinical trial to
further evaluate safety and potential anti-tumor activity of
TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial.
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 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 and at a certain dose level 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 TransCode's 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, 2024,
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.
