TransCode Therapeutics, Inc. (NASDAQ:RNAZ) Advancing Targeted RNA Therapeutics Through Innovative Delivery

Published on September 24, 2024 | Ticker: RNAZ

TransCode Therapeutics, Inc. (NASDAQ:RNAZ) is a biopharmaceutical company focused on developing and commercializing innovative drugs and diagnostics for treating and identifying cancer. The company's lead therapeutic candidate, TTX-MC138, is designed to inhibit the ability of metastatic tumor cells to survive, with the goal of achieving durable disease regression and long-term patient survival.

Business Overview

TransCode was founded in 2016 and has developed a proprietary TTX delivery platform that leverages an iron-oxide nanoparticle to safely and effectively deliver oligonucleotide-based therapeutics to tumors and metastases. The company's modular approach allows it to adapt the size, charge, and surface chemistry of the nanoparticle core to optimize delivery of various therapeutic payloads, including small interfering RNAs, antisense oligonucleotides, non-coding RNA mimics, mRNA-based cancer vaccines, CRISPR-based gene repair and replacement platforms, and pattern recognition receptors.

In 2023, TransCode received exploratory Investigational New Drug (IND) Study May Proceed notification from the FDA for TTX-MC138, allowing the company to conduct a Phase 0 clinical trial to demonstrate quantitative delivery of radiolabeled TTX-MC138 to metastatic lesions in subjects with advanced solid tumors. The company treated one patient in this Phase 0 trial. In April 2024, TransCode received IND Study May Proceed notification from the FDA to conduct a Phase I/II clinical trial with TTX-MC138, which it expects to commence in mid-2024.

In addition to TTX-MC138, TransCode has other solid tumor programs in the preclinical stage, including 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 targeting activation of innate immunity in the tumor microenvironment. The company is also developing 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.

Financials

For the three months ended March 31, 2024, TransCode reported a net loss of $3,326,812, compared to a net loss of $4,816,934 in the same period of the prior year. The company's research and development expenses decreased from $2,591,350 in the first quarter of 2023 to $1,759,020 in the first quarter of 2024, while general and administrative expenses decreased from $2,296,334 to $1,529,962 over the same period.

For the full year 2023, TransCode reported an annual net loss of $18,546,095 and had no revenue. The company's annual operating cash flow was -$18,074,535, and its annual free cash flow was -$18,110,144.

As of March 31, 2024, TransCode had cash of approximately $4.9 million, which the company believes will be sufficient to fund its operating expenses and capital expenditure requirements into the third quarter of 2025. However, the company will need to raise additional capital to continue its operations and advance its pipeline beyond that timeframe.

Liquidity

TransCode's primary sources of capital have been the sale of equity securities, including its initial public offering in 2021, subsequent equity financings, and funds received under a Small Business Innovation Research (SBIR) award from the National Cancer Institute. Through March 31, 2024, the company had received net cash proceeds of approximately $51.7 million from these sources.

The company believes its current cash position will be sufficient to fund its operations and capital requirements into the third quarter of 2025. However, TransCode will need to raise additional capital to continue its operations and advance its pipeline of product candidates, including the planned Phase I/II clinical trial for TTX-MC138. The company may seek to raise additional funds through the sale of equity or debt securities, collaborations, or other strategic transactions.

Risks and Challenges

TransCode faces several risks and challenges in its pursuit of developing and commercializing its product candidates. These include the inherent risks of drug development, such as the uncertainty of clinical trial outcomes, regulatory approval, and commercial viability. The company also faces the challenge of effectively delivering its oligonucleotide-based therapeutics to tumors and metastases, which is a key focus of its TTX delivery platform.

Additionally, TransCode may be unable to raise sufficient capital to fund its operations and pipeline development, which could force the company to amend, delay, limit, reduce, or terminate its development programs. The company also faces the risk of potential delisting from the Nasdaq Capital Market if it is unable to regain compliance with the exchange's minimum stockholders' equity or minimum bid price requirements.

Outlook

In the near-term, TransCode's key focus will be on advancing TTX-MC138 through its planned Phase I/II clinical trial, which is expected to commence in mid-2024. The company also plans to continue the development of its other solid tumor programs, including TTX-siPDL1 and TTX-RIGA, as well as its CRISPR-based and mRNA-based platforms.

TransCode has also recently entered into a collaboration agreement with Debiopharm, a Swiss-based biopharmaceutical company, to test the development of new targeted nucleic acid delivery modalities using the company's TTX delivery platform. This collaboration could provide additional validation of TransCode's technology and potentially lead to future strategic partnerships.

Additionally, the company has applied for a Direct-to-Phase II SBIR award from the National Cancer Institute to support the clinical development of TTX-MC138, as well as a Fast-Track SBIR award to support the clinical development of TTX-siPDL1. If awarded, these non-dilutive funding sources could provide important resources to advance TransCode's pipeline.

Conclusion

TransCode Therapeutics is a promising biopharmaceutical company that is leveraging its innovative TTX delivery platform to advance a pipeline of oligonucleotide-based therapeutics for the treatment of cancer. With its lead candidate, TTX-MC138, poised to enter a Phase I/II clinical trial, and a robust preclinical pipeline, TransCode is well-positioned to make significant strides in the field of targeted RNA therapeutics. However, the company faces several risks and challenges, including the need to raise additional capital to fund its operations and pipeline development. Investors should closely monitor TransCode's progress as it works to unlock the potential of its technology and deliver innovative solutions for cancer patients.

You may be interested in...

Perspective Therapeutics: Pioneering Targeted Alpha Therapies for Cancer Treatment

Perspective Therapeutics, Inc. (NYSE AMERICAN: CATX) is a small-cap radiopharmaceutical company that is pioneering advanced treatment applications for cancers throughout the body. The company has developed a proprietary platform technology that utilizes precision-targeted alpha therapies (TATs) to deliver potent radioactive payloads directly to tumor cells, sparing healthy tissue. Perspective Therapeutics' unique approach has the potential to transform the treatment landscape for a wide range of solid tumors, offering hope to patients who have exhausted traditional options.

PTC Therapeutics (PTCT): Pioneering Innovative Therapies for Rare Diseases

Business Overview and Company History: PTC Therapeutics, Inc. (NASDAQ:PTCT) is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. The company's diversified therapeutic portfolio pipeline includes several commercial products and product candidates in various stages of development, positioning PTC as a leader in the rare disease space.

IMNN: Unlocking the Power of DNA-Mediated Immunotherapy in Ovarian Cancer

Imunon, Inc. (NASDAQ:IMNN) is a clinical-stage biotechnology company pioneering the development of innovative DNA-based immunotherapies and next-generation vaccines. The company’s lead clinical program, IMNN-001, has demonstrated remarkable promise in the treatment of advanced ovarian cancer, a devastating disease with limited treatment options. Imunon’s cutting-edge approach has the potential to transform the landscape of ovarian cancer care and deliver meaningful improvements in patient outcomes.

TransCode Therapeutics (NASDAQ:RNAZ): A Pioneer in RNA Oncology Tackling Metastatic Cancer

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.

Tango Therapeutics (TNGX): Pioneering the Next Generation of Precision Cancer Medicines

Tango Therapeutics, a clinical-stage biotechnology company, is at the forefront of developing innovative precision oncology therapies. Founded in 2017, the company's mission is to revolutionize cancer treatment by leveraging its state-of-the-art target discovery platform to uncover novel targets and create highly selective small molecule drugs.