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.

On August 15, 2022 Shanghai’s Bennu Biotherapeutics reported that closed a $15 million Angel+ financing round to support its novel T-cell development programs (Press release, BennuBio, AUG 15, 2022, View Source [SID1234618405]). Founded in 2021, Bennu plans to take cell therapy beyond CAR-T drugs to include T cell types such as TIL, TCR-T and Treg. Although CAR-T has had great success in hematological tumors, it faces several clinical challenges overall. Bennu expects to aim its T cell drugs at solid tumor cancers and autoimmune diseases. The financing was led by INCE Capital with Life Science Park Innovation Fund and Witruth Capital participating.

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On August 15, 2022 GenoImmune Therapeutics, a Wuhan company founded by China’s gene sequencing company, BGI, reported that it closed a funding round to support its immuno-cell therapy products (Press release, Genoimmune Therapeutics, AUG 15, 2022, View Source [SID1234618404]). The company’s core technologies include its AI-driven neoantigen technology, a GMP cell preparation platform and mRNA tumor vaccines aimed at solid tumors including melanoma, lung cancer and colorectal cancer. Using its AI-based screening, GenoImmune can identify novel tumor antigens that are used in T-Cell and Neo-T cell therapies. In previous $15+ million rounds, the company attracted Hillhouse, ForeBright and Wuhan East-lake Venture Capital as investors.

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On August 15, 2022 Anixa Biosciences, Inc. (NASDAQ: ANIX) ("Anixa") a biotechnology company focused on the treatment and prevention of cancer and infectious diseases, reported that, in conjunction with its partner Moffitt Cancer Center, it has commenced treatment of the first patient in the clinical trial of its novel chimeric antigen receptor T-cell (CAR-T) therapy for ovarian cancer (Press release, Anixa Biosciences, AUG 15, 2022, View Source [SID1234618393]).

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The study is a dose-escalation Phase 1 trial to determine safety and the maximum tolerated dose of follicle stimulating hormone receptor T-cells and to preliminarily assess efficacy. The study is being conducted at Moffitt Cancer Center and will consist of up to 48 patients.

The CAR-T approach used for Anixa’s therapy is known as chimeric endocrine receptor T-cell (CER-T) since the target of the engineered T-cells is an endocrine receptor. While CAR-T therapy has shown efficacy in some hematological tumors, reproducing the same results with solid tumors, such as ovarian cancer, has proven challenging. One of the reasons for this difficulty is that effective CAR-T therapy needs a specific antigen to recognize that is only present on target cancer cells in order to avoid negatively affecting healthy cells. The CER-T therapy being evaluated in Anixa’s Phase 1 study differs from traditional CAR-T in that it targets the follicle stimulating hormone receptor (FSHR), which research indicates is exclusively expressed on ovarian cells in healthy adult females.

"We are pleased that the first patient has been treated in our ovarian cancer CAR-T clinical study," stated Amit Kumar, Ph.D., Chairman and CEO of Anixa Biosciences. "This is truly an exciting time for Anixa, as we have now begun treating patients in our second clinical trial. With our CAR-T study, we hope to determine whether our unique targeting approach will work in solid tumors—a difficult challenge for traditional CAR-T therapies."

Robert Wenham, M.D., MS, FACOG, FACS, the trial’s lead investigator and Chair of the Department of Gynecologic Oncology at Moffitt Cancer Center, added, "With limited treatment options for recurrent, chemo-resistant ovarian cancer, I am hopeful that this program can provide a unique opportunity to make a meaningful impact on patients of this devastating disease."

Jose R. Conejo-Garcia, M.D., Ph.D., Chair of the Department of Immunology at Moffitt Cancer Center and co-inventor of the CER-T technology, added, "It is exciting to see our novel FSHR-mediated CAR-T technology reaching patients, and if our unique CAR-T approach is successful, it could serve as a model for future targeted CAR-T therapies in other cancer types."

Dr. Conejo-Garcia and his research team developed the FSHR-mediated CAR-T technology when he was at the Wistar Institute where he contributed to report for the first time on the role of T-cell responses in the outcome of ovarian cancer patients. The clinical trial being conducted today is based on this pre-clinical work, originally published in Clinical Cancer Research. Anixa has an exclusive, world-wide license to this technology.

About Anixa’s CER-T Approach (Follicle Stimulating Hormone Receptor-Mediated CAR-T technology)
Anixa’s chimeric antigen receptor T-cell (CAR-T) technology approach is an autologous cell therapy comprised of engineered T-cells that target the follicle stimulating hormone receptor (FSHR). FSHR is found at immunologically relevant levels exclusively on the granulosa cells of the ovaries. Since the target is a hormone (chimeric endocrine) receptor, and the target-binding domain is derived from its natural ligand, this technology is known as CER-T (chimeric endocrine receptor T-cell) therapy, a new type of CAR-T.

On August 15, 2022 Lytix Biopharma AS ("Lytix" or the "Company"), a clinical-stage company with an in situ vaccination technology platform, reported data from its ATLAS-IT-04 trial in patients with progressive metastatic soft tissue sarcoma (STS) (Press release, Lytix Biopharma, AUG 15, 2022, View Source [SID1234618392]). The data from this Phase II proof of concept study shows that LTX-315 in combination with Adoptive Cell Therapy (ACT) was able to stabilize the disease in 3 out of 4 fully treated patients in this hard-to-treat patient population, and that the LTX-315 treatment generated tumor-specific T cells.

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The data is presented June 5th, 2022, as a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting, Chicago, IL, U.S.A.

The ATLAS-IT-04 trial was an open label, exploratory, Phase II trial assessing the effect of LTX-315 when used in combination with ACT in patients with metastatic STS. ACT with tumor infiltrating lymphocytes (TILs) is a potent treatment that can induce complete and durable tumor regression as documented in patients with melanoma. The use of ACT with TILs for patients with advanced STS has not previously been reported.

Patients with advanced stages of STS have few effective treatment options and respond poorly to current treatment as well as to immunotherapy tested in clinical trials. The trial design of ATLAS-IT-04 included intratumoral injections of LTX-315 ahead of surgical removal of tumors, followed by in vitro expansion of T cells as the first step. In a second step, the expanded T cells were infused back to the patients and the effect of LTX-315 on the tumor microenvironment was assessed.

LTX-315 is a first-in-class non-viral oncolytic molecule, representing a new and superior in situ therapeutic vaccination principle to boost the clonal expansion of T cells that kill tumor cells through a targeted immune response. In a recent Phase I/II study LTX-315 has been shown to increase TILs in malignant solid tumors after intratumoral injection.

The immune response data from the ATLAS-IT-04 trial demonstrates that the treatment induce both new and tumor-specific T cells which provides proof to the concept that LTX-315 generates an immune response that targets the tumor. Moreover, the data shows that LTX-315 induce expansion of a heterogenous pool of T-cell clones in blood, and a pool of these are also present in tumor tissue after treatment.

"This trial demonstrates that the combination of LTX-315 and ACT is not only feasible and tolerable, but that tumor-specific T cells can be expanded in vitro from tumors that have been pretreated with the oncolytic molecule LTX-315", Inge Marie Svane, PI and Professor at the National Center for Cancer Immune Therapy, Department of Oncology, Copenhagen University Hospital, comments.

She adds: "This combination therapy invokes tumor specific T cells that can be cultured and infused as part of an adoptive transfer regimen for several subtypes of soft tissue sarcoma, and its treatment schedule should be further optimized to achieve superior signs of efficacy."

The poster is presented June 5th at the ASCO (Free ASCO Whitepaper) 2022 Annual Meeting.