TransCode Therapeutics Announces Publication of Preclinical Data Supporting Therapeutic Potential of TTX-MC138 in Glioblastoma

Rhea-AI Summary

TransCode Therapeutics (NASDAQ: RNAZ) reported preclinical data showing its lead candidate TTX-MC138 reaches orthotopic glioblastoma tumors after intravenous dosing and produces sustained target engagement against miR-10b.

The study in murine models reported a 5-fold increase in apoptotic activity in tumors and a statistically significant survival benefit. IND‑enabling studies, PK/biodistribution, and toxicity work have been completed, and Phase 1 safety data exist in non-CNS patients; a Phase 2a trial is anticipated in H1 2026.

Positive

• Systemic delivery of TTX-MC138 to brain tumors demonstrated
• Apoptotic activity in tumors increased by 5-fold
• Treatment produced a statistically significant survival benefit
• IND-enabling PK, biodistribution, and toxicity studies completed
• Phase 1 safety data observed in non-CNS patients

Negative

• Efficacy shown only in murine orthotopic GBM models, not humans
• No clinical efficacy data yet in glioblastoma patients
• Phase 2a in GBM is anticipated but not yet initiated

Key Figures

Median survival under two years Glioblastoma standard-of-care outcome described in article

Apoptotic activity increase five-fold TTX-MC138-induced apoptotic activity in GBM tumors in murine models

Journal volume/issue Volume 17, Issue 1 Journal of Functional Biomaterials publication details

Phase 2a start timing first half of 2026 Anticipated start of Phase 2a trial for TTX-MC138 in metastatic disease

Phase 2a enrollment up to 45 patients Planned colorectal minimal residual disease Phase 2a trial (Dec 11, 2025 news)

Stable disease rate 44% (7/16 patients) Phase 1a TTX-MC138 metastatic disease results (Oct 14, 2025 news)

Current price $7.31 Price before publication of GBM preclinical data

52-week range $6.0829–$468.44 52-week low and high prior to this news

Market Reality Check

$8.01 Last Close

Volume Volume 21,427 is below the 20-day average of 29,728, indicating subdued trading ahead of this news. normal

Technical Price at $7.31 is trading below the 200-day MA of $10.36 and far below the 52-week high of $468.44.

Peers on Argus 1 Up

Biotech peers show mixed moves, with names like INAB, CYCCP, and BOLT down while APRE and EPIX are up. Momentum scanner only flags AEON up 5.2% with no news, suggesting RNAZ trading is more company-specific than sector-driven.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 22	Board appointment	Neutral	-4.1%	Added experienced director and committee member to strengthen governance and oversight.
Dec 11	Clinical collaboration	Positive	+20.7%	Launched Phase 2a PRE-I-SPY trial with TTX-MC138 in colorectal minimal residual disease.
Nov 17	Executive hire	Positive	+1.8%	Appointed Senior Development Officer to guide TTX-MC138 and seviprotimut-L toward late-stage trials.
Oct 14	Phase 1a results	Positive	+5.6%	Reported Phase 1a TTX-MC138 completion with primary safety endpoint met and stable disease cases.
Oct 08	Acquisition & financing	Positive	+21.1%	Acquired Polynoma plus $25M equity investment, adding Phase 3–ready melanoma vaccine asset.

Pattern Detected

RNAZ has mostly reacted positively to strategic and clinical catalysts, with four of the last five news events seeing price gains, while one board-related announcement coincided with a decline.

Recent Company History

Over the past six months, TransCode reported several milestones. On Oct 8, 2025, it acquired Polynoma and secured a $25 million equity investment, adding Phase 3–ready seviprotimut‑L and lifting the stock. A completed Phase 1a trial of TTX‑MC138 with no dose‑limiting toxicities and stable disease in 44% of patients preceded a planned Phase 2a trial. Management additions in Nov and Dec 2025 supported late‑stage development. December’s Phase 2a colorectal collaboration reinforced TTX‑MC138’s importance, and today’s GBM preclinical data further extends this program’s scope.

Market Pulse Summary

This announcement adds preclinical GBM data to the existing TTX-MC138 clinical narrative, showing systemic delivery to brain tumors, a five-fold increase in apoptotic activity, and a statistically significant survival benefit in murine models. It complements prior Phase 1a safety and pharmacodynamic findings and the planned Phase 2a trial in colorectal minimal residual disease. With shares trading near the $6.08 52-week low and below the $10.36 200-day MA, investors may watch for future GBM trial initiation and additional human efficacy readouts.

Key Terms

glioblastoma multiforme medical

"supporting the application of its lead candidate, TTX-MC138, for the treatment of glioblastoma multiforme (GBM)"

An aggressive form of brain cancer that grows quickly and spreads into nearby brain tissue, often causing severe symptoms and limited treatment options. For investors, glioblastoma matters because it defines a high unmet medical need and a potentially large market for new drugs, devices, or diagnostics; success or failure in clinical trials and regulatory reviews for glioblastoma treatments can dramatically affect the valuation and risk profile of healthcare companies, like a make-or-break product for a small biotech.

microRNA10-b medical

"The molecular target of TTX-MC138, microRNA10-b (miR-10b), is highly expressed in GBM cells"

microRNA-10b is a small non-coding RNA molecule that helps control which genes are turned up or down inside cells, acting a bit like a dimmer switch for specific cellular programs. It is often studied as a marker and potential target in diseases such as cancer because changes in its level can signal disease progression or affect how tumors spread; investors watch it because therapies or tests that target or measure it can drive drug development, clinical trials, and diagnostic markets.

antisense oligonucleotides medical

"capability of TransCode's TTX platform to systemically deliver antisense oligonucleotides (ASOs) to brain neoplasms"

Antisense oligonucleotides are short, synthetic pieces of genetic material designed to stick to specific messenger RNA molecules inside cells and block or alter the production of a particular protein. For investors, they matter because they represent a targeted drug approach with potential to treat diseases that other medicines cannot, but they also carry development, safety and regulatory risks that can lead to large swings in a biotech company’s value.

pharmacokinetics medical

"Considering that investigational new drug (IND) enabling studies as well as pharmacokinetics, biodistribution, and required toxicity studies"

Pharmacokinetics is the study of how a substance, such as a drug or chemical, moves through and is processed by the body over time. It tracks how it is absorbed, distributed, broken down, and eventually eliminated. For investors, understanding pharmacokinetics helps gauge the effectiveness, safety, and potential risks of new medications or treatments, which can influence a company’s success and valuation in the healthcare industry.

biodistribution medical

"enabling studies as well as pharmacokinetics, biodistribution, and required toxicity studies for TTX-MC138"

Biodistribution is the map of where a drug, vaccine, or diagnostic agent travels and accumulates inside the body after administration. Investors care because where a product ends up affects how well it works, what side effects it may cause, and whether regulators will approve it—similar to tracking dye in a plumbing system to find leaks or blockages; unexpected destinations can raise safety, cost, and market-adoption risks.

apoptotic medical

"TTX-MC138 also induced apoptotic activity in tumors by five-fold"

Apoptotic describes cells undergoing apoptosis, a programmed, orderly process in which a cell essentially dismantles and disposes of itself—like a worn appliance being safely decommissioned to avoid causing damage. For investors, labeling a drug or biomarker as apoptotic signals a specific mechanism of action that can mean effective removal of diseased cells (important for cancer treatments) or possible harm to healthy tissue, affecting trial outcomes, regulatory review and commercial prospects.

orthotopic medical

"Nanotherapy Targeting miR-10b Improves Survival in Orthotopic Glioblastoma Models"

Orthotopic describes placing tissue, cells, an organ, or a disease model in its normal anatomical location—for example, transplanting a liver into the liver bed or studying a tumor in the organ where it naturally grows. Investors watch for orthotopic approaches because they tend to produce results that better predict real-world safety and effectiveness than unnatural models, so they can improve the credibility of clinical data and influence regulatory and commercial prospects.

investigational new drug regulatory

"Considering that investigational new drug (IND) enabling studies as well as pharmacokinetics"

An investigational new drug is a medication that is still being tested in clinical trials to determine if it is safe and effective for treating a specific condition. For investors, it represents a potential breakthrough that could lead to a new treatment and significant financial gains if successful, but also carries risks since it has not yet been approved for widespread use.

AI-generated analysis. Not financial advice.

• TTX-MC138 is delivered to glioblastoma tumors in murine models of the disease, following intravenous injection
• TTX-MC138 demonstrated suppression of miR-10b target and extended survival in aggressive glioblastoma models
• Study supports future clinical development of TTX-MC138 for patients with glioblastoma

BOSTON, Jan. 6, 2026 /PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical stage company pioneering immuno-oncology and RNA therapeutics for the treatment of high risk and advanced cancer, today announced the publication of preclinical research supporting the application of its lead candidate, TTX-MC138, for the treatment of glioblastoma multiforme (GBM). The article was published in the peer-reviewed Journal of Functional Biomaterials (Volume 17, Issue 1). The study, entitled "Nanotherapy Targeting miR-10b Improves Survival in Orthotopic Glioblastoma Models," resulted from a collaboration between TransCode and Michigan State University. The study was led by Dr. Anna Moore, Professor, Director of the Precision Health Program, and Associate Dean for Research Development at the College of Human Medicine at Michigan State University and scientific co-founder of TransCode.

Glioblastoma is the most aggressive primary brain cancer, with median survival under two years from diagnosis despite current standard-of-care interventions. The molecular target of TTX-MC138, microRNA10-b (miR-10b), is highly expressed in GBM cells where it drives tumor survival, growth and invasiveness.

The study demonstrated delivery of TTX-MC138 to human GBM tumors implanted into the brains of murine models after intravenous injection, resulting in sustained target engagement within the tumor. TTX-MC138 also induced apoptotic activity in tumors by five-fold, consistent with observed induction of tumor cell death. Importantly, treatment with TTX-MC138 resulted in a statistically significant increase in survival.

These findings demonstrate the capability of TransCode's TTX platform to systemically deliver antisense oligonucleotides (ASOs) to brain neoplasms and further supports its potential utility in overcoming key delivery barriers, including nucleic acid degradation and limited tumor penetration. Considering that investigational new drug (IND) enabling studies as well as pharmacokinetics, biodistribution, and required toxicity studies for TTX-MC138 have already been completed and that the formulation has shown appreciable safety in Phase I clinical trials in patients with non-central nervous system (CNS) cancers, these results support advancing TTX-MC138 to future clinical evaluation in patients with GBM.

"This research represents an important step forward in targeting one of the most treatment-resistant forms of cancer," said Dr. Zdravka Medarova, CSO of TransCode. "By pairing our differentiated delivery approach with robust biological support, we are broadening the potential reach of our RNA-based therapeutics beyond metastatic solid tumors," added Dr. Medarova. 

TTX-MC138 is currently evaluated in metastatic disease in a Phase 1a clinical trial, with a Phase 2a clinical trial anticipated to begin in the first half of 2026, underscoring the translational relevance of this approach.

About TransCode Therapeutics 

TransCode Therapeutics is a clinical stage company pioneering immuno-oncology and RNA therapeutic treatments of high risk and advanced cancers. The company's lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors that overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode has a portfolio of other first-in-class therapeutic candidates designed to mobilize the immune system to recognize and destroy cancer cells. For more information, visit www.transcodetherapeutics.com.

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 value of TTX-MC138 for the treatment of GBM, statements concerning the timing, conduct and results of TransCode's collaborations, statements concerning TransCode's clinical trials, statements concerning the timing of clinical trials, statements about microRNAs and their involvement in cancer, statements concerning the therapeutic potential of TransCode's TTX delivery platform and statements concerning the therapeutic potential of TransCode's TTXMC138 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.

FAQ

What did TransCode announce about TTX-MC138 for glioblastoma (RNAZ) on January 6, 2026?

Published preclinical results showing IV delivery to brain tumors, 5-fold tumor apoptosis increase, and a statistically significant survival benefit in murine GBM models.

How did TTX-MC138 perform in preclinical glioblastoma models for RNAZ?

TTX-MC138 reached orthotopic GBM tumors after IV dosing, engaged miR-10b, induced apoptosis five-fold, and extended survival.

Has TransCode started clinical trials of TTX-MC138 in glioblastoma (RNAZ)?

Not yet; TTX-MC138 is in a Phase 1a trial for metastatic disease and a Phase 2a in GBM is anticipated in H1 2026.

What preclinical and regulatory milestones has RNAZ completed for TTX-MC138?

IND-enabling studies plus pharmacokinetics, biodistribution, and required toxicity studies have been completed.

Does the published study prove TTX-MC138 will work in glioblastoma patients (RNAZ)?

No; the publication reports positive murine-model data but does not provide human GBM efficacy data.

What target does TTX-MC138 engage in glioblastoma and why is it relevant to RNAZ investors?

TTX-MC138 targets miR-10b, which is highly expressed in GBM and linked to tumor survival, growth and invasiveness.