On April 3, 2024 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported financial results for 2023 and recent business progress (Press release, TransCode Therapeutics, APR 3, 2024, View Source [SID1234641817]).

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"We believe 2023 was extremely productive and pivotal for TransCode. We are proud to have advanced our lead therapeutic candidate, TTX-MC138, into the clinic as a first-in-class drug candidate against metastatic cancer. The year was highlighted by preliminary clinical results from our Phase 0 clinical trial with radiolabeled TTX-MC138," said Tom Fitzgerald, interim CEO and CFO of TransCode. "Further, despite very challenging financial markets, we raised over $25 million in equity financings, streamlined our operations for higher efficiency, and signed two strategic collaborations aimed at demonstrating the value of our TTX platform in additional applications. We also continued to achieve other important milestones, with the ultimate objective of fulfilling the promise of RNA therapeutics for oncology applications. We now look forward to our Phase 1 clinical trial of TTX-MC138 which, subject to FDA authorization, is planned to begin in mid-2024."

Key Highlights (2023 and Q1 2024)

· IRB approval for FDA-cleared first-in-human Phase 0 clinical trial with TTX-MC138.
· First patient dosed and preliminary clinical results with radiolabeled TTX-MC138 in the Phase 0 clinical trial.
· Signed a co-research agreement with Debiopharm to develop nucleic acid therapeutics for cancer treatment.
· Signed a joint research and development agreement with Akribion Genomics to develop a CRISPR-derived technology platform for cancer treatment.
· Raised over $25 million in equity financings in an extremely challenging financial environment.
· Refocused our development strategy, prioritizing advancing TTX-MC138 into a Phase 1 clinical trial and reducing cash burn.
· Appointed CFO Tom Fitzgerald as interim CEO and director Philippe Calais as Executive Chairman following the resignation of Michael Dudley as CEO.
· Appointed Daniel Vlock, M.D., as Chief Medical Officer (part-time).
· Regained compliance with Nasdaq’s stockholders’ equity requirement.
· Presented TransCode technology at leading cancer conferences including San Antonio Breast Cancer Symposium, AACR (Free AACR Whitepaper), OTS, and TIDES Europe.

TTX-MC138

· Reported preliminary clinical results suggesting delivery of TTX-MC138 to metastatic lesions in a patient with breast cancer metastatic to lungs, bone, and liver.
· Reported positive preclinical results in a model of glioblastoma showing successful delivery and pharmacodynamic activity in brain tumors.
· Reported positive preclinical results in a model of metastatic pancreatic adenocarcinoma showing 50% inhibition of metastatic progression compared to standard-of-care chemotherapy.
· Received second Orphan Drug Designation from FDA, for TTX-MC138 in pancreatic cancer.

TTX-siPDL1

· Reported positive preclinical results in pancreatic cancer, showing extended survival relative to standard-of-care chemotherapy.

TTX-RIGA

· Reported successful completion of animal studies in a model of melanoma showing arrest of secondary tumor progression.

2024 Corporate and R&D Objectives

With what we expect will be a very exciting year ahead, our 2024 corporate goals, none of which are assured, are to strengthen our organization while raising sufficient capital to continue to advance our research. We will also seek to expand collaborations with strategic partners to further enhance the value of our pipeline.

2024 will also be a critical year for the clinical development of TTX-MC138 and our preclinical portfolio, as we seek to:

· Initiate a Phase 1 clinical trial with TTX-MC138 in patients with advanced solid tumors as soon as we obtain FDA authorization and relevant institutional review board approvals from investigational sites.
· Report preliminary results from the planned Phase 1 trial later this year.
· Publish preclinical results in the second half of the year supporting TTX-MC138 in glioblastoma and pancreatic cancer, TTX-siPDL1 in pancreatic cancer, and TTX-RIGA in melanoma.
· Continue preclinical studies for therapeutic candidates TTX-mRNA and TTX-CRISPR.
· Advance existing strategic partnerships and sign others around TransCode’s TTX delivery platform, TTX-CRISPR, TTX-siRNA, TTX-MC138, and TTX-mRNA candidates.
· Further develop an exploratory test for miRNA-10b.
· If capital resources permit, initiate CMC development to support future IND-enabling studies with TTX-siPDL1 or TTX-RIGA candidates.

2023 Financial Highlights (amounts are approximate)

· Cash was $2.8 million on December 31, 2023, compared to $4.97 million on December 31, 2022.
· Research and development expenses were $12.3 million in 2023 compared to $10.2 million in 2022.
· General and administrative expenses were $7.2 million in 2023 compared to $8.4 million in 2022.
· Operating loss was $19.4 million in 2023 compared to $18.6 million in 2022.

Financial Guidance

TransCode expects that its cash of approximately $2.8 million as of December 31, 2023, together with approximately $6.2 million in net proceeds received from the sale of common stock and warrants in a January 2024 registered direct offering, will be sufficient to fund planned operations into late third quarter or early fourth quarter of 2024.