AFYX Therapeutics A/S Secures DKK 25 million in Funding

On December 19, 2024 AFYX Therapeutics A/S, a pioneering Danish biopharmaceutical company specializing in reversed innovation and the repurposing of established pharmaceutical substances, reported it has successfully raised DKK 25 million in its latest funding round (Press release, AFYX Therapeutics, DEC 19, 2024, View Source [SID1234649209]). This milestone will support the company’s efforts to advance its product pipeline, scale operations, and expand its footprint across the European Union and other global markets.

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The funding round was led by existing investors and employees and highlights the robust internal and external trust in AFYX’s mission and strategic direction. The new funds will accelerate the development of the company’s leading products, including its clobetasol patch for oral lichen planus and esketamine therapy for cluster headaches. Additionally, the capital will fuel AFYX’s commercial expansion into new markets, organizational growth, and broader international partnerships.

"We are thrilled to have secured this significant investment, which will enable us to advance our mission of developing transformative biopharmaceutical solutions," said Dr. Claus Møller San Pedro, CEO and co-founder of AFYX Therapeutics. "The continued trust from our investors reflects their confidence in our team and vision. With their ongoing support, we’re well-positioned to address critical unmet medical needs and deliver groundbreaking therapies that improve patient outcomes."

Since its formation in January 2024, through the merger of three highly experienced pharmaceutical innovators, AFYX Therapeutics has made remarkable progress. Over its first year, the company has accelerated its research and development activities, expanded its commercial reach, and secured new international distribution partnerships, paving the way for sustained growth and innovation.

Vivesto strengthens Cantrixil program with new preclinical results and patent application

On December 19, 2024 Vivesto AB, an oncology-focused development company, reported that positive results were obtained from preclinical studies with combination treatments within the company’s Cantrixil program, supporting continued development in hematological cancer (Press release, Vivesto, DEC 19, 2024, View Source [SID1234649208]). Vivesto also announced that a new patent application covering the treatment of hematological cancer with Cantrixil was filed, with the aim to strengthen the IP position.

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The drug candidate Cantrixil has been evaluated in further combination treatments with other anti-cancer drugs generating new in vitro data in hematological cancer cell lines. The results demonstrate clear positive effects of Cantrixil in combination with other anti-cancer drugs. The positive results confirm previous preclinical efficacy results and support continued development in hematological cancer. New results from hematological cancer models are expected to be presented throughout 2025.

"Hematological cancer is one of Vivesto’s priority focus areas, and we are pleased to report successful results from yet another preclinical study supporting the further development of the Cantrixil program. We believe that Cantrixil can play a key role in the treatment of hematological cancer and look forward to reporting new data from additional ongoing studies early next year that will be of great importance as the program is developed towards clinical phase," said Erik Kinnman, CEO of Vivesto. "Vivesto also filed a new patent application for the treatment of hematological cancer with Cantrixil as a robust patent portfolio is fundamental for value creation in clinical development, and eventually, commercialization of the product."

Cantrixil has previously shown strong cytotoxic effects at low doses in cell lines derived from patients with hematological cancer. The recently generated data provides important input to the dosing selection and treatment regime in upcoming preclinical and clinical studies.

Sprint Bioscience adds new cancer drug program to portfolio

On December 18, 2024 Sprint Bioscience AB (publ) reported that the company is broadening its portfolio with a drug development program for the treatment of acute myeloid leukemia (AML). The program targets the decapping scavenger enzyme (DCPS), a target protein that has been validated both in scientific literature and by Sprint Bioscience in collaboration with Associate Professor Julian Walfridsson at Karolinska Institutet.

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The initiation of Sprint Bioscience’s DCPS program is part of the company’s strategy to capitalize on its platform for small-molecule drug discovery to broaden the portfolio in the cancer area and to bring more opportunities for future licensing deals.

The DCPS program focuses on a target protein that degrades a metabolite resulting from the processing of mRNA. Both small-molecule inhibition and genetic knock-down of DCPS affects the differentiation and proliferation of several AML cell lines as well as patient-derived samples. Furthermore, healthy tissue seems to be insensitive to DCPS inhibition, indicating that DCPS inhibitors could offer a safe and effective treatment option for AML patients. Sprint Bioscience has also identified biomarkers to predict response to DCPS inhibition, enabling patient selection and clinical success for patients with unmet therapeutic needs.

"We have identified a target protein that constitutes a very attractive approach for the treatment of AML. We aim to advance the project to a stage where it can be licensed to an international pharmaceutical company, enabling further clinical development and progression towards the market to benefit AML patients in need of new treatment options," said, Johan Emilsson, CEO of Sprint Bioscience.

AML is a severe type of blood cancer. The Global Burden of Disease study estimates that about 140,000 people around the world are diagnosed with AML on a yearly basis. There is an urgent medical need to identify safe and effective therapies to improve treatment outcomes.

Sprint Bioscience’s portfolio now consists of six internal drug development programs (five of which are in cancer) and one program that has been licensed to Day One Biopharmaceuticals.

(Press release, Sprint Bioscience, DEC 18, 2024, View Source [SID1234660961])

TigaTx Announces up to $35.5M in Funding from ARPA-H and NIH Awards to Advance First-in-Class Engineered IgA Monoclonal Antibody for Cancer and Infectious Diseases

On December 18, 2024 TigaTx, Inc., a biotechnology company developing a therapeutic platform technology of engineered Immunoglobulin A (IgA) monoclonal antibodies to address a broad range of diseases, reported that the Advanced Research Projects Agency for Health (ARPA-H) has awarded TigaTx up to $33.5 million in funding (Press release, TigaTx, DEC 18, 2024, View Source [SID1234654374]). Separately, TigaTx was awarded a two-year, $2 million Direct to Phase II Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) of the U.S. National Institutes of Health under award number R44CA291266.

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Many immune cell types, such as T cells, NK cells, and macrophages, are currently engaged by anti-cancer therapies, resulting in successful outcomes for cancer patients. However, neutrophils, the first line of defense in infection and inflammation, are the most abundant immune cells in human circulation and have not yet been harnessed by cancer therapies. Beyond their abundance, neutrophils possess other ideal characteristics as anti-cancer effector cells. They are innate killer cells, and they cross talk with other cells of the innate and adaptive immune system, thereby, further propagating the anti-tumor cascade.

TigaTx’s novel, proprietary platform technology leverages engineered monomeric IgA to bind to and potently activate neutrophils, unleashing their powerful anti-tumor killing ability to treat cancer. TigaTx’s technology yields IgA molecules that have drug-like properties and overcomes historical challenges associated with manufacturing IgA.

The ARPA-H and SBIR funding will allow TigaTx to advance its lead program, TIGA-001, an IgA anti-EGFR neutrophil engager, into the clinic to generate clinical proof-of-concept data for the engineered IgA platform. While anti-EGFR IgGs and tyrosine kinase inhibitors are approved for colon, lung, and head and neck cancers, less than 25% of patients respond, and even for those patients who do respond, their cancer will typically recur. TIGA-001’s distinct neutrophil activation-targeted mechanism of action has the potential to benefit patients with resistance or intolerance to approved anti-EGFR therapies.

In parallel, the ARPA-H award will enable TigaTx to expand its engineered IgA platform to treat infectious diseases. With increasing emergence of antibiotic-resistant strains of bacteria and persistent and emergent viral threats, TigaTx will develop secretory, dimeric IgA as a new therapeutic class for infectious diseases.

"We’re thrilled to announce this funding from ARPA-H and NIH, which provides important external validation of the breakthrough potential of our IgA monoclonal antibody platform and its first-in-class therapeutic applications," said Anne Altmeyer, Ph.D., President and CEO of TigaTx. "This funding will allow us to validate our platform by generating clinical proof-of-concept data for our lead oncology program, TIGA-001. The funding will also enable us to expand our IgA platform technology to other high-unmet-need indications both in oncology and infectious diseases."

Howard Stern, M.D., Ph.D., Chief Scientific Officer of TigaTx, commented, "Engineered IgA neutrophil engagers have the potential to revolutionize the treatment of cancer by harnessing neutrophils’ innate killing power. Further expanding our IgA platform to dimeric IgA opens a new frontier to address the growing threat of communicable diseases."

Under the ARPA-H funding, TigaTx will collaborate with several prestigious academic institutions and world-renowned oncology and immunology researchers to further characterize engineered IgA’s mechanism of action and develop additional proof-of-concept in cutting-edge preclinical models. Collaborating institutions include:

Weill Cornell Medicine

Taha Merghoub, Ph.D., Deputy Director of the Sandra and Edward Meyer Cancer Center; Margaret and Herman Sokol Professor of Oncology Research; Professor of Pharmacology and Professor of Immunology Research in Medicine; Co-Director of the Ludwig Collaborative and Swim Across America Laboratory; Co-Director of the Parker Institute for Cancer Immunotherapy at Weill Cornell Medicine

Jedd D. Wolchok, M.D., Ph.D., FAACR, FASCO, Meyer Director of the Sandra and Edward Meyer Cancer Center; Professor of Medicine; Co-Director of the Ludwig Collaborative and Swim Across America Laboratory; Director of the Parker Institute for Cancer Immunotherapy at Weill Cornell Medicine; Oncologist at New York Presbyterian/Weill Cornell Medical Center

Daniel Hirschhorn, Ph.D., Assistant Professor of Research in Pharmacology

Yale School of Medicine

Richard Flavell, Ph.D., FRS, Sterling Professor of Immunobiology; Investigator, Howard Hughes Medical Institute

Esen Sefik, Ph.D., Assistant Professor, Immunobiology; Howard Hughes Medical Institute Fellow

Hadassah Hebrew University Medical Center (Jerusalem, Israel)

Zvi Fridlender, M.D. M.Sc, Head, Department of Internal Medicine D, Head Laboratory of Lung Cancer Research

The content of this release is solely the responsibility of the authors and does not necessarily represent the official views of the Advanced Research Projects Agency for Health (ARPA-H) or the National Institutes of Health.

Applied DNA Customer ÚHKT Initiates Phase I Clinical Trial for Rapidly Manufacturable CAR T-cell Therapy Produced from LineaDNA

On December 18, 2024 Applied DNA Sciences, Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company"), a leader in PCR-based DNA technologies, reported that the State Institute for Drug Control of the Czech Republic (SÚKL) approved an application for a Phase I clinical trial of an investigational CD123-specific autologous CAR T-cell therapy by the Institute of Hematology and Blood Transfusion (ÚHKT/Eng: IHBT) in Prague for the treatment of relapsed and/or refractory acute myeloid leukemia (AML) (Press release, Applied DNA Sciences, DEC 18, 2024, View Source [SID1234650780]). UHKT-CAR123-01 utilizes Applied DNA’s synthetic DNA, Linea DNA, as a critical component in its manufacture.

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AML is a hematologic malignancy with a high rate of treatment failure for which current treatment options are often restricted to palliative approaches. Novel emerging methods leveraging advancements in genetic medicines, such as CAR T-cell therapies, can potentially improve outcomes of patients after relapse but have been difficult to establish for clinical use due to high costs and long manufacturing times predominantly attributable to the use of viral vectors.

UHKT-CAR123 seeks to address these issues by generating CD123-specific CAR T-cells in a non-viral workflow utilizing Linea DNA to reduce manufacturing costs and timelines. Preclinical data showed that ÚHKT’s Linea DNA-empowered non-viral workflow resulted in the rapid production of substantial and cost-efficient CAR T-cell yields with high potency[1].

"The use of Linea DNA illustrates our innovative approach to finding new and best-in-class treatments for patients with relapsed or refractory AML," stated Dr. Jan Vydra, principal investigator of the UHKT-CAR123 clinical trial.

Added Pavel Otáhal, Ph.D., scientific project leader at ÚHKT, "The Linea DNA platform enables the very rapid abiotic production of expression vectors usable for highly effective electroporation-based CAR-T manufacturing compared to plasmid-based vectors. The decreases in complexities and costs of developing autologous CAR-T technologies offer an innovative approach for the rapid clinical testing of novel types of CAR-T products. This is an incredible milestone for ÚHKT and one that we could not have achieved without the commitment of the Applied DNA team."

Applied DNA CEO Dr. James A. Hayward, said, "We congratulate ÚHKT on their progress into the clinic. Their accomplishment is also a significant milestone for Linea DNA as we look to support additional customers expected to initiate clinical trials in calendar 2025."

About Linea DNA
Linea DNA is an enzymatically produced, linear DNA manufactured by the Company’s proprietary, large-scale polymerase chain reaction ("PCR") based manufacturing platform, the Linea DNA platform. As an alternative to plasmid-based DNA, Linea DNA can be used to manufacture of a wide range of nucleic acid-based therapies and in vitro diagnostics, including mRNA therapies, DNA vaccines, cell and gene therapies, and molecular and genetic diagnostic tests.