TransCode Therapeutics, Inc. is a clinical-stage RNA oncology company committed to more effectively treating cancer using innovative RNA therapeutics. The company’s mission is to develop transformative therapies that address the underlying drivers of metastatic cancer, a leading cause of cancer-related deaths worldwide.
Company History and Business Overview
TransCode Therapeutics, Inc. was incorporated on January 11, 2016 under the laws of the State of Delaware. From its founding until mid-2021, the company was primarily engaged in organizational activities, including raising capital, and limited research and development (R&D) activities. During this period, TransCode faced challenges such as limited funding and a small team, which restricted its ability to advance its research programs rapidly.
A significant turning point came in April 2021 when the company received a Fast-Track Small Business Innovation Research (SBIR) Award from the National Cancer Institute of the National Institutes of Health. This award, worth up to $2.39 million over three years, provided non-dilutive funding to support the company’s research partnership with Massachusetts General Hospital and its efforts to advance TTX-MC138 into clinical trials.
In July 2021, TransCode achieved a major milestone by completing its initial public offering (IPO), raising $28.75 million in gross proceeds. This capital infusion marked a transformative moment for the company, enabling it to significantly expand its R&D activities, hire additional employees to support its operations, and accelerate the development of its lead therapeutic candidate, TTX-MC138.
The successful IPO allowed TransCode to overcome many of its initial obstacles and make substantial progress in advancing its pipeline of RNA-based therapeutic candidates. However, the company has continued to face challenges. In 2023, TransCode received several notifications from the Nasdaq Stock Market that it was not in compliance with certain listing requirements. This led the company to appeal the Nasdaq Staff’s determination, and in November 2024, the Nasdaq Hearings Panel granted the company a formal extension to regain compliance.
TransCode’s proprietary technology platform, TTX, leverages an iron oxide nanoparticle as the physical carrier of therapeutic oligonucleotides, designed to overcome the challenge of effective delivery to tumors and metastatic sites. The company’s flagship program, TTX-MC138, is an antisense oligonucleotide conjugated to the TTX delivery system, designed to inhibit microRNA-10b, a known driver of metastasis in multiple cancer types.
In 2023, TransCode received an exploratory Investigational New Drug (IND) Study May Proceed notification from the U.S. Food and Drug Administration (FDA), allowing it to conduct a Phase 0 clinical trial with radiolabeled TTX-MC138. The trial, which dosed its first patient in 2023, was designed to demonstrate the delivery of TTX-MC138 to metastatic lesions and quantify the amount of the drug candidate reaching those sites. Preliminary results showed that radioactivity consistent with accumulation of TTX-MC138 was detected in the regions of the metastatic lesions, and pharmacokinetic behavior of the radiolabeled TTX-MC138 was consistent with non-clinical IND-enabling studies.
In April 2024, TransCode received an IND Study May Proceed notification from the FDA to conduct a Phase 1/2 clinical trial with TTX-MC138, which commenced in the third quarter of 2024. The trial is designed to evaluate the safety and preliminary anti-tumor activity of TTX-MC138 in patients with advanced solid tumors.
In addition to TTX-MC138, TransCode has a pipeline of other solid tumor programs in the preclinical stage, including TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1 (PD-L1), and TTX-RIGA, an RNA-based agonist of the retinoic acid-inducible gene I (RIG-I), targeting activation of innate immunity in the tumor microenvironment.
Financial Performance and Liquidity
Financial Performance
TransCode is still in the early stages of its clinical development journey and, as a result, has not generated any revenue from product sales to date. The company has relied on a combination of equity financings, including its IPO, and government grants to fund its operations.
For the year ended December 31, 2023, TransCode reported a net loss of $18.55 million, with no revenue generated. The company’s operating expenses for the year were $18.50 million, primarily consisting of $12.26 million in research and development costs and $7.15 million in general and administrative expenses.
In the most recent quarter (Q3 2024), TransCode reported a net loss of $2.32 million, compared to a larger net loss in the same quarter of the previous year. The company’s operating cash flow (OCF) for Q3 2024 was negative $3.91 million, and its free cash flow (FCF) was negative $3.93 million. Both OCF and FCF decreased year-over-year due to continued investment in research and development and clinical trials for TTX-MC138.
Liquidity
As of September 30, 2024, TransCode had cash of approximately $1.90 million. The company believes this cash balance will be sufficient to fund its operating expenses and capital expenditure requirements into late 2024. However, TransCode will need to raise additional capital to continue its operations and advance its clinical development programs beyond that point.
TransCode’s financial position as of September 30, 2024, showed a current ratio and quick ratio of 1.58, indicating that the company had sufficient short-term assets to cover its short-term liabilities. The company reported no outstanding debt, resulting in a debt-to-equity ratio of 0.
In the third quarter of 2024, TransCode received three letters from the Nasdaq Stock Market notifying the company that it was not in compliance with certain Nasdaq listing rules, including the bid price requirement and the shareholder approval rule. As a result, the company’s shares are subject to delisting from the Nasdaq Capital Market.
On October 1, 2024, TransCode appealed the Nasdaq Staff’s determination to a Nasdaq Hearings Panel, and on November 4, 2024, the company was informed that the Panel granted it a formal extension to regain compliance with the Nasdaq Listing Rules. The company must take several actions, including obtaining shareholder approval for a reverse stock split, to maintain its Nasdaq listing.
Management Changes
In January 2024, TransCode experienced a significant management change when Michael Dudley, the company’s then-President, CEO, and Director, resigned from his positions. Following this departure, Thomas A. Fitzgerald, who was serving as the company’s Chief Financial Officer, was appointed as Interim CEO.
Product Pipeline and Clinical Progress
TransCode’s lead therapeutic candidate, TTX-MC138, has shown promising results in preclinical studies. The drug candidate has demonstrated complete regression of metastatic disease in several mouse models of pancreatic and breast cancer. Additionally, TTX-MC138 was successfully delivered and showed bioactivity in a case study of spontaneous feline mammary carcinoma, supporting the company’s decision to advance it into clinical development.
In August 2023, TransCode initiated its first clinical trial, a Phase 0 study designed as an open-label, single-center trial. The study aimed to demonstrate the delivery of a single microdose of radiolabeled TTX-MC138 to radiographically-confirmed metastases in subjects with advanced solid tumors. Preliminary data from the single patient dosed in this trial showed encouraging results, with radioactivity consistent with TTX-MC138 accumulation detected in the regions of metastatic lesions. The pharmacokinetic behavior of the radiolabeled TTX-MC138 was in line with expectations based on non-clinical studies. Importantly, the study quantified the amount of drug candidate delivered to metastatic lesions, providing further evidence of TTX-MC138’s ability to accumulate in metastatic tumors. The patient tolerated the dosing with no reported adverse reactions.
Building on these positive results, TransCode received FDA notification in April 2024 to proceed with a Phase 1 clinical trial of TTX-MC138. This trial, which began in the third quarter of 2024, is designed as a multicenter, open-label, dose-escalation and dose-expansion study in patients with advanced solid tumors. The initial stage of the trial, Phase 1a, involves the administration of escalating therapeutic dose levels of TTX-MC138 to up to six cohorts of approximately three patients per cohort, with the first cohort receiving the lowest therapeutic dose level.
In addition to TTX-MC138, TransCode is developing other solid tumor programs in the preclinical stage. These include TTX-siPDL1, an siRNA-based modulator of programmed death-ligand 1, and TTX-RIGA, an RNA-based agonist of the retinoic acid-inducible gene I, which targets the activation of innate immunity in the tumor microenvironment.
The company is also exploring the development of TTX-CRISPR, a CRISPR-Cas9-based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells, and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines that activate cytotoxic immune responses against tumor cells. However, the advancement of these additional programs is subject to available funding.
Risks and Challenges
TransCode faces several risks and challenges common to early-stage biopharmaceutical companies, including:
Regulatory approval hurdles: The company must navigate the complex regulatory landscape and obtain necessary approvals from the FDA and other regulatory bodies to commercialize its product candidates.
Competitive landscape: TransCode operates in a highly competitive oncology market, with established players and other emerging therapies targeting similar pathways.
Funding and liquidity concerns: As a clinical-stage company, TransCode will need to continue raising significant capital to fund its operations and development activities, which may be challenging, especially in the current market environment.
Intellectual property protection: The company’s success is dependent on its ability to protect its proprietary technology and intellectual property rights.
Reliance on third-party vendors: TransCode relies on contract research organizations, contract manufacturing organizations, and other third-party vendors to support its operations, which introduces additional risks.
Potential for technology and platform failures: There is no guarantee that TransCode’s TTX delivery platform and the underlying technologies will be successful in overcoming the challenges of effective drug delivery to tumors and metastatic sites.
Nasdaq compliance: The company must take necessary actions to regain compliance with Nasdaq listing requirements to maintain its listing on the Nasdaq Capital Market.
Outlook and Conclusion
TransCode Therapeutics is at a critical juncture in its development, with its lead candidate, TTX-MC138, now in Phase 1/2 clinical trials. The company’s pioneering work in RNA oncology and its proprietary TTX delivery platform have the potential to address a significant unmet need in the treatment of metastatic cancer.
While TransCode faces the usual risks and challenges associated with a clinical-stage biopharmaceutical company, its recent progress, including the successful completion of the Phase 0 trial and the FDA clearance to initiate the Phase 1/2 trial, demonstrate the company’s ability to navigate these obstacles.
The upcoming results from the Phase 1/2 trial of TTX-MC138 will be a critical milestone for TransCode, as they will provide important insights into the safety and preliminary anti-tumor activity of the drug candidate. Additionally, the company’s efforts to regain compliance with Nasdaq listing requirements will be crucial in maintaining access to the public capital markets and supporting its long-term growth.
TransCode’s focus on the US market and its status as a clinical-stage company mean that it does not yet have geographic market performance data to report. However, the company’s progress in clinical development and its innovative approach to RNA therapeutics position it as a potential leader in the field of oncology, particularly in addressing the challenges of metastatic cancer.
Overall, TransCode Therapeutics is a compelling story in the rapidly evolving field of RNA therapeutics, with the potential to make a significant impact on the lives of cancer patients. As the company continues to execute on its strategic priorities, it will be essential to monitor its progress, clinical data, and ability to navigate the regulatory and financial challenges that lie ahead. The success of its ongoing clinical trials, particularly for TTX-MC138, will be crucial in determining the company’s future trajectory and its potential to revolutionize cancer treatment.
Disclaimer: This article is for informational purposes only. It does not constitute financial, legal, or other types of advice. While every effort has been made to ensure the accuracy of the information presented here, the author and the publisher do not make any guarantees about the completeness, reliability, and accuracy of this information.