On May 16, 2022 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to defeating cancer using RNA therapeutics, reported financial results for the first quarter ended March 31, 2022, and recent business progress (Press release, TransCode Therapeutics, MAY 16, 2022, View Source [SID1234614635]).

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TransCode co-founder and Chief Technology Officer, Dr. Zdravka Medarova, indicated, "We believe we remain on track to submit an exploratory Investigational New Drug Application (eIND) this year to test our lead therapeutic candidate, TTX-MC138, in a Phase 0 study in cancer patients with advanced solid tumors. We believe this study has the potential to establish proof-of-mechanism for our platform which ultimately could help us build a broad and diverse pipeline of therapeutics and diagnostics that have the potential to reach previously undruggable genetic targets."

"Additionally, we believe we achieved the first milestone related to our Small Business Innovation Research (SBIR) grant during the first quarter and anticipate receiving the next tranche of award funding during the second quarter," said Michael Dudley, co-founder, president and CEO of TransCode. "This non-dilutive funding, combined with capital from our 2021 IPO, provide the resources to drive continued progress across the organization, including key additions to our team who bring valuable expertise, and continued advancement of our lead therapeutic candidate and preclinical development of our other therapeutic candidates. As we move toward the Phase 0 proof-of-mechanism clinical trial, we continue to focus on using the power and versatility of our TTX platform to solve the challenges of RNA delivery in oncology."

Other First Quarter 2022 Highlights

Expanded global RNA oncology patent portfolio with filing of an International PCT Application (PCT/US21/65580) entitled TEMPLATE DIRECTED IMMUNOMODULATION FOR CANCER THERAPY (the ‘580 application). The ‘580 application, expected to be published in June 2022, represents an extension of TransCode’s use of its patented RNA therapeutic platform to include using pattern recognition receptors (PRR) to target tumor cells by activating the RIG-I signaling pathway. Once inside a cell, the selected PRR-activating oligonucleotide encounters a microRNA specific to that tumor where it is expected to activate a type I interferon-driven immune response, leading to programmed tumor cell death.
Published article in the journal "Cancers" titled, Clinical Applications of Short Non-Coding RNA-Based Therapies in the Era of Precision Medicine, in collaboration with teams from Massachusetts General Hospital (MGH), Michigan State University, and Northeastern University. The article describes the development, challenges, and clinical successes of short non-coding RNA-based drugs and details several examples of how these RNA drugs are designed, chemically modified, and delivered to treat cancer, cardiovascular disease, and rare genetic disorders. In addition, the article highlights key similarities and differences between various short non-coding RNA platforms and discusses considerations to maximize treatment efficacy of RNA-based therapeutics. TransCode Co-Founder and scientific advisor, Dr. Anna Moore, was a contributing author to the article. The article was published on March 21, 2022.
Planned Milestones

TransCode’s goals to continue to advance its portfolio include:

TTX-MC138
Submission to FDA of an eIND application for its First-in-Human (FIH) clinical trial.
Completion of a FIH Phase 0 clinical study intended to demonstrate quantifiable evidence of delivery of radiolabeled TTX-MC138 to metastatic lesions in advanced solid tumors; measure pharmacokinetics and biodistribution in vital organs and other tissues; potentially inform therapeutic dose levels based on microdose results; and validate delivery for the TTX pipeline more broadly, potentially opening-up additional relevant RNA targets that have been previously undruggable due to challenges with RNA delivery.
Concurrent completion of IND-enabling studies to support filing an IND application for a Phase I clinical trial of TTX-MC138.
Publication of preclinical results supporting the lead therapeutic candidate, TTX-MC138, in pancreatic cancer and glioblastoma multiforme.
Publication of preclinical results supporting therapeutic candidate, TTX-RIGA.
Continuation of preclinical studies for therapeutic candidates TTX-RIGA, TTX-siPDL1, and TTX-siLIN28B.
Continuation of discussions regarding potential partnerships.
File for Orphan Drug Designation for its lead therapeutic candidate in additional tumor indications.
First Quarter 2022 Financial Highlights

Cash and cash equivalents were $16.9 million at March 31, 2022, compared to $20.8 million at December 31, 2021.
Research and development expense was $1.9 million in the first quarter of 2022, compared to $0.3 million in the first quarter of 2021.
General and administrative expense was $1.6 million in the first quarter of 2022, compared to $0.2 million in the first quarter of 2021.
Operating loss for the three months ended March 31, 2022, was $3.5 million, compared to an operating loss of $0.4 million in the prior year period.
Financial Guidance

TransCode expects that its cash of $16.9 million as of March 31, 2022, together with additional funding expected from the April 2021 SBIR award, are sufficient to fund planned operations into the first quarter 2023 but not for a full 12 months from the date of its financial statements.