- Clinical trial Safety Review Committee (SRC) approved opening
of Cohort 4 based on favorable safety data from the three patients
comprising Cohort 3
- No significant safety or dose limiting toxicities reported in
Cohorts 1, 2, or 3
- Patients currently being evaluated for Cohort 4
eligibility
- PK and PD data from patients in Cohort 1 and Cohort 2
consistent with preclinical results and results from Phase 0
clinical trial
BOSTON, March 13,
2025 /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 I
clinical trial has unanimously approved opening of the fourth
cohort of patients based on the SRC's favorable review of Cohort 3
safety data. The therapeutic candidate being evaluated, TTX-MC138,
is a first-in-class therapeutic designed to treat multiple
metastatic cancers using antisense technology. The dose
administered to the fourth cohort, as originally planned in the
clinical protocol, will be approximately fifty percent higher than
the dose administered in the third cohort.
Under the clinical protocol, patients may remain on study absent
safety events or disease progression. Out of 9 patients treated
with TTX-MC138 in the first three cohorts, 6 remain on study for
continued treatment since there have been no dose limiting
toxicities or disease progression with these patients. The patient
that has remained on study the longest has, to date, received 7
doses each approximately 28 days apart over the approximately 7
months that this patient has been on study. In addition to
approving opening the fourth cohort, the SRC approved enrollment of
additional patients in Cohort 3 to build upon the safety profile of
TTX-MC138. Further, Cohort 1 and 2 data analysis for both
pharmacokinetic (PK) and pharmacodynamic (PD) activity 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. Preliminary results from Cohort 2 confirmed
observations from the Phase 0 trial and from Cohort 1 that the drug
candidate shows evidence of activity in patient blood. Preliminary
PK analysis suggests that dose levels 0.8-1.6 mg/kg, the levels
administered to Cohorts 1 and 2, could represent an efficacious
range.
"SRC approval to open the fourth cohort and expand enrollment in
Cohort 3 is an important advancement for the clinical trial. It
will provide an opportunity to obtain additional safety and PK/PD
data, inform the dose expansion stage of the clinical trial and may
allow us to obtain initial evidence of clinical activity" 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
subjects may now be screened and scheduled in Cohort 4 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 micro-RNA 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 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 stage.
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. 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
pre-clinical 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.
