On August 15, 2022 aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported second quarter 2022 results and provided a corporate update (Press release, aTyr Pharma, AUG 15, 2022, View Source [SID1234618433]).

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"We are pleased with our second quarter progress as we announced our plans to initiate EFZO-FIT, a Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "This global pivotal study is a major milestone for the sarcoidosis community and is projected to be the largest interventional study for patients with sarcoidosis to date. We are on track to enroll the first patient in this study this quarter."

Second Quarter 2022 and Subsequent Period Highlights

In May 2022, announced plans to initiate the global pivotal EFZO-FIT study, a global pivotal Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously once a month for a total of 12 doses. The study intends to enroll 264 subjects with pulmonary sarcoidosis at multiple centers in North America, Europe and Japan. The trial design will incorporate a forced steroid taper. The primary endpoint of the study is steroid reduction. Secondary endpoints include measures of lung function and sarcoidosis symptoms.
Accomplished several operational milestones for the EFZO-FITTM study since its announcement in May 2022, including multiple interactions with regulatory authorities in the United States, European Union and Japan along with the submission of study protocol and clinical trial applications to regulatory authorities, ethics committees and institutional review boards. Site selection, qualification and initiations for several sites have occurred, as well as an investigator meeting for U.S. sites. The company is on track to enroll the first patient in the study in the third quarter of 2022.
Received U.S. Food and Drug Administration (FDA) Fast Track designation for efzofitimod for the treatment of pulmonary sarcoidosis. Fast Track designation helps facilitate development and expedite the review of drugs to treat serious or life-threatening diseases with unmet medical need. Fast Track designation provides certain benefits, including more frequent interactions with the FDA throughout the development program, as well as eligibility for accelerated approval, priority review and rolling review.
Presented clinical data from the recently completed Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis at the American Thoracic Society (ATS) 2022 International Conference in San Francisco, California.
Announced fibroblast growth receptor 4 (FGFR4) as the target receptor for a fragment of the Alanyl-tRNA Synthetase (AARS) in a poster presented at the Keystone Symposia on Tissue Fibrosis and Repair: Mechanisms, Human Disease and Therapeutics. FGFR4 is known to play a role in diseases related to inflammation and fibrosis, including conditions where unchecked fibrosis can precede the development of certain cancers. The company intends to interrogate the interaction between this fragment of AARS and FGFR4 and the implications for disease in order to explore this synthetase fragment as a potential pipeline candidate.
Second Quarter 2022 Financial Highlights and Cash Position

Cash & Investment Position: Cash, restricted cash, cash equivalents and investments as of June 30, 2022, were $89.3 million.
R&D Expenses: Research and development expenses were $9.1 million for the second quarter of 2022, which consisted of product development and manufacturing costs for the efzofitimod and ATYR2810 programs, as well as startup costs for the Phase 3 EFZO-FITTM study.
G&A Expenses: General and administrative expenses were $3.4 million for the second quarter of 2022.
Shares Outstanding: Common shares outstanding were 28,127,458 as of June 30, 2022.
Conference Call and Webcast Details

aTyr will host a conference call and webcast today at 5:00 p.m. EDT / 2:00 p.m. PDT to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: View Source An audio replay will be available for at least 90 days following the event.

About Efzofitimod

aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease. Efzofitimod, a fusion protein comprised of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates innate and adaptive immune response in inflammatory disease states. aTyr’s lead indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease. Clinical proof-of-concept for efzofitimod was recently established in a Phase 1b/2a multiple-ascending dose, placebo-controlled study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated safety and a consistent dose response and trends of benefit of efzofitimod compared to placebo on key efficacy endpoints, including steroid reduction, lung function, clinical symptoms and inflammatory biomarkers. aTyr is currently conducting EFZO-FIT, a Phase 3 study of efzofitimod in pulmonary sarcoidosis patients.

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.