On August 15, 2022 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported financial results for the second quarter ended June 30, 2022, and recent business progress (Press release, TransCode Therapeutics, AUG 15, 2022, View Source [SID1234618407]).

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"We continued to advance our therapeutic programs during the second quarter," said Michael Dudley, co-founder, president and CEO of TransCode. "We remain on track to submit an exploratory Investigational New Drug Application (eIND) this year for our planned Phase 0 clinical trial with TTX-MC138 in cancer patients with advanced solid tumors. In addition, we were granted orphan drug designation by the US Food and Drug Administration (FDA) for TTX-siPDL1 for pancreatic cancer, and we continued preclinical studies with our other therapeutic candidates in a variety of tumor indications. In May, we were awarded the second tranche of additional non-dilutive funding from our NIH grant. We believe that these funds, along with our existing cash, provide sufficient runway to carry us into the first quarter of 2023."

"Subsequent to the quarter end, we announced a strategic alliance with MD Anderson Cancer Center to further advance our pipeline of RNA-targeted oncology therapeutic and diagnostic candidates. Under the alliance, scientists from both organizations will collaborate on preclinical studies and clinical trials seeking to further validate our pipeline candidates and expand the reach of our TTX discovery engine," added Dudley.

TransCode co-founder and Chief Technology Officer, Dr. Zdravka Medarova, commented, "We believe our unique RNA delivery platform, TTX, has the potential to enable the development of a wide array of safe and effective RNA therapeutics for cancer by addressing the challenge of delivery to tumor cells anywhere in the body. We seek to demonstrate TTX’s ability to reach metastatic lesions in cancer patients in our planned Phase 0 clinical trial. We believe the trial has the potential to establish proof of delivery for our TTX platform, which ultimately could help enable us to build a broad and diverse pipeline of therapeutics and diagnostics to reach previously undruggable genetic targets."

Recent Business Highlights

In June 2022, the FDA granted our request for orphan drug designation (ODD) for TTX-siPDL1 for the treatment of pancreatic cancer. ODD status for TTX-siPDL1 provides several potential benefits including seven years of marketing exclusivity if the designated candidate receives FDA marketing approval, tax credits for qualified R&D expenses, and an exemption from payment of the Prescription Drug User Fee Amendment (PDUFA) filing fee, a savings estimated to be more than $3 million.
In May 2022, received notice of the availability of the second tranche of funding from a Fast-Track Small Business Innovation Research (SBIR) grant we were awarded by the National Cancer Institute of the NIH (Award Number R44CA257093). The SBIR award, totaling $2.3 million, supports the continued clinical evaluation of TTX-MC138. The Company achieved the first milestone related to the SBIR grant during the first quarter of 2022.
In May 2022, acquired an option giving TransCode the right to negotiate an exclusive, worldwide, royalty-bearing license related to a radiotheranostic technology disclosed in patent application PCT/US2021/057912 entitled THERAPEUTIC, RADIOLABLED NANOPARTICLES AND METHODS OF USE THEREOF. Invented by Dr. Medarova and colleagues at Massachusetts General Hospital, the technology represents another potential advancement in the diagnosis and treatment of cancer. The use of radioisotopes is expected to enable further advancement of our pipeline of RNA therapeutics by expanding their scope to include theranostic applications.
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 trial 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 for future trials 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.
Further progress toward completion of IND-enabling studies to support filing an IND application for a Phase I clinical trial of TTX-MC138.
Completion of preclinical in vivo studies supporting the lead therapeutic candidate, TTX-MC138, in pancreatic cancer and glioblastoma multiforme.
Completion of preclinical in vivo studies supporting therapeutic candidates, TTX-RIGA, TTX-siPDL1 and TTX-siLIN28B.
Continuation of discussions regarding potential partnerships.
File for orphan drug designation for TTX-MC138 in additional tumor indications.
Second Quarter 2022 Financial Highlights

Cash was $13.4 million at June 30, 2022, compared to $20.8 million at December 31, 2021.
Research and development expense was $2.6 million in the second quarter of 2022, compared to $0.2 million in the second quarter of 2021.
General and administrative expense was $2.1 million in the second quarter of 2022, compared to $0.1 million in the second quarter of 2021.
Operating loss for the three months ended June 30, 2022, was $4.7 million, compared to an operating loss of $0.4 million in the prior year period.
Financial Guidance

TransCode expects that its cash of $13.4 million as of June 30, 2022, is sufficient to fund planned operations into the first quarter 2023 but not for a full 12 months from the date of its financial statements.