Niigata University

OSAKA, Japan, Oct. 9, 2024 /PRNewswire/ -- Alpha Fusion, Inc. (CEO: Sunao Fujioka, headquartered in Kita-ku, Osaka), has raised a total of ¥1.02 billion through a Series B funding round. The round was led by SBI Investment Co., Ltd. and OSAKA University Venture Capital, with participation from several new investors. This funding will enable Alpha Fusion to accelerate its research and development efforts, enhance its supply chain, and deliver Japan's cutting-edge cancer therapies to the global market as quickly as possible. Alpha Fusion Leading the World in Astatine-based Drug Discovery Alpha Fusion is a pioneering startup dedicated to bringing "Targeted Alpha Therapy (TAT) using Astatine (At-211)" to cancer patients worldwide. As a leader in the clinical development of Astatine-based therapeutics, Alpha Fusion is advancing its pipeline and building a highly efficient supply chain using cyclotrons in collaboration with partners across the globe. TAT is highly anticipated to become a foundational drug discovery platform in oncology, offering the potential to create numerous novel treatments. In the past year, several major pharmaceutical companies in Europe and the U.S. have acquired startups developing Actinium (Ac-225) -based radiopharmaceuticals, signaling a surge of interest in this field. Astatine (At-211) is garnering attention for its short half-life, which anticipates clinical safety, its halogen properties that allow it to be directly labeled into low-mid sized-molecule ligands, its ease of sourcing raw material, and the simplicity of its production using cyclotrons. Business Progress and the Impact of Series B Funding Since our Series A funding round in May 2023, Alpha Fusion has made significant strides, as detailed below: 1. 1st Pipeline (Thyroid Cancer, Phase 1 ongoing): Progress in high-dose administration, approaching the determination of recommended dosages. 2. 2nd Pipeline (Prostate Cancer, Phase 1 ongoing): Successful completion of the world's first administration of [211At]PSMA-5 to a patient in June. 3. Additional Pipelines: Multiple pipeline developments underway through both in-house R&D and collaborations. July 2023: Initiated joint research with QST Chiba for tumor-specific targeting. August 2023: Began joint research with QST Takasaki/Niigata University on novel Astatine-labeling platform technologies. 4. Supply Chain: Progress in manufacturing preparations for clinical trial drugs both domestically and internationally, shaping the future of our supply chain. First half of 2024: Completion of Osaka University's TAT Cyclotron building to enable efficient Astatine production. First half of 2024: Set up GMP facilities for clinical trial drug production in Japan and advanced collaboration with overseas CDMOs. 5. Intellectual Property: Received patent approvals covering manufacturing methods, materials, and applications related to our pipeline. 6. Research Centers: Expanded domestic research operations, including a new center in Tsukuba (SakuLab-Tsukuba), in addition to Osaka University's research hub. 7. Team Expansion: Strengthened the advisory board and enhanced functional capabilities critical to pharmaceutical development (e.g., supply chain, CMC, quality assurance, clinical development, regulatory affairs). With this funding, we will accelerate our R&D efforts and prepare for company-sponsored clinical trials, enhance our supply chain for GMP-grade clinical trial drug production domestically and internationally, and further expand our team. We are committed to demonstrating the unique advantages of Astatine-based drug discovery with tangible business results and clinical evidence. Investors in Series B Round (in no particular order): New Investors: SBI Investment Co., Ltd. Mitsui Mining & Smelting Co., Ltd. Kobe University Capital Joyo Capital Partners Existing Investors (Follow-on Investment): OSAKA University Venture Capital D3 LLC JGC Corporation Comments from Alpha Fusion CEO, Sun ao Fujioka "In the past year, Alpha Fusion has made great progress. We are strategically advancing pipeline R&D and supply chain construction by leveraging the unique characteristics of Astatine: its short half-life, the potential for pipeline expansion due to its halogen properties, and the stable supply enabled by cyclotron production. The market is moving rapidly, with ongoing M&A and large-scale funding in the TAT sector globally. As we continue developing TAT pipelines, this funding round serves as a critical step toward company-sponsored clinical trials and GMP-grade Astatine drug production. We will push forward, engaging more stakeholders to realize our mission of advancing cancer treatment." About Alpha Fusion Alpha Fusion, Inc. was established to commercialize Astatine (At-211) drug discovery, based on research outcomes from Osaka University and the Japan Science and Technology Agency (JST) OPERA QiSS program. The company is dedicated to unleashing the therapeutic potential of Alpha (Astatine) therapy in the fast-emerging field of Targeted Alpha Therapy. Through world-class R&D and business development, Alpha Fusion aims to bring this innovative modality to the forefront of cancer treatment. Contact Information For further inquiries, please contact: Email: [email protected] SOURCE Alpha Fusion Inc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

In their pursuit to discover the mechanism behind how RVCL does its damage, researchers found some clues to the DNA damage theory of aging. TREX1 is a gene that is supposed to direct the maintenance of the entire body's DNA, but new research shows that when people are born with mutated TREX1, it causes catastrophic damage to the DNA over time, resulting in a deadly rare disease called retinal vasculopathy with cerebral leukoencephalopathy (RVCL). Published in Nature Communications, the research was led by teams at the Perelman School of Medicine at the University of Pennsylvania and the Brain Research Institute at Niigata University in Japan. While it was already known that a mutation in TREX1 was behind RVCL, the mechanism by which it did its damage was not known. In discovering that TREX1 speeds up the typical process of DNA damage -- a process some theorize is tied to every animal's aging process -- the researchers may not have only discovered the weapon TREX1 uses on RVCL patients, but also offered insight beyond this rare disease population. "It seems that accelerated DNA damage in RVCL causes the premature aging of certain cells, including the cells in the blood vessel wall," said the study's lead author Jonathan Miner, MD, PhD, an associate professor of Rheumatology at the Perelman School of Medicine. "If this is the case, then targeting TREX1 could have very broad implications for the treatment of many human diseases linked to aging, including cardiovascular diseases, autoimmune disorders, and cancer." RVCL impacts about 200 people worldwide and is often misdiagnosed as lupus, multiple sclerosis, or cancer. The disease causes the breakdown of small blood vessels in the body, which can affect many organs, including the brain, eyes, kidneys, liver, and bones. Patients with the condition typically do not begin to have symptoms -- such as memory loss, partial loss of vision, and small strokes -- until their 40s or 50s. Eventually, the breakdowns can cause organ damage and failure, including brain atrophy and blindness. There is no cure or treatment, and many patients die within five to ten years of symptoms beginning. "We're hopeful that our work will put us on the path toward improving the lives of patients with RVCL," said the other lead author of this study Taisuke Kato, PhD, an associate professor of Molecular Neuroscience at Niigata University. "With our discoveries, we feel we will be much better equipped to address what is happening within their bodies." Studying RVCL models in animal and human cells, Miner and his colleagues explored their hypothesis that the TREX1 mutation, which shortens the gene, was causing instability within cells and damage that appears similar to breakdowns seen in radiation injuries. They found that the mutation was interfering with a DNA repair process , which occurs when there is a break in both strands of DNA. This interruption of the process allowed DNA to be deleted, and cells prematurely aged and stopped dividing, which leads to overall premature aging and organ damage. In addition to uncovering RVCL's primary mechanism of damage, the researchers also found that the TREX1 mutation also displayed a cell-level vulnerability that mirrors those seen in people with mutations to the BRCA1 and BRCA2 genes, which cause breast cancer. Sure enough, the authors found similar elevated rates of risk of breast cancer in patients with TREX1 mutations as in patients with mutations in the BRCA1 and BRCA2 genes. But on top of that, the TREX1 mutation's effect on DNA damage also makes people with it more susceptible to damage from chemotherapy, the team found. "I do worry that certain treatments may have accelerated the progression of disease in some patients," Miner said. "In many cases, chemotherapy was prescribed as a way of treating suspected 'autoimmunity' since certain chemotherapeutic agents can also be used to treat patients with systemic lupus. This was frequently employed in the treatment of RVCL in the past, and even recently by some, and we are concerned that this would actually make the disease worse." The study's findings provide a clearer picture for the types of treatments and medicines that could be pursued for people with RVCL. They could involve lowering levels of TREX1 in the body, correcting the mutation, or just blocking the gene's DNA-damaging effects. "Until those therapies are developed, we are working to figure out whether certain mediations already FDA-approved for the treatment of other diseases might be repurposed for RVCL or potentially impact TREX1 levels in the body overall," Miner said. "TREX1 levels increase with age in multiple tissues in all humans -- even in healthy individuals without RVCL -- and we need to understand the processes linked to this." To that end, the study's findings extend well beyond the population of patients with RVCL because of what it may add to the DNA damage theory of aging. "One hope is that understanding the role of TREX1 in RVCL might help us uncover mechanisms that could link the TREX1 gene to a wide variety of human conditions that could also include normal aging," Miner said. This study was funded by the National Institutes of Health (K08AR070918, R01AI143982, R01NS131480, T32 GM007170), the Rheumatology Research Foundation, the Colton Center for Autoimmunity, the Clayco Foundation, the Penn RVCL Sisters Fund, the Japan Agency for Medical Research and Development (22ek0109424h0003), the Japan Society for the Promotion of Science (22H00466), and the Japan MHLW for Research on Intractable Disease (JP21FC1007).

The trial assessed Leukine in 64 subjects with mild to moderate autoimmune pulmonary alveolar proteinosis. Credit: mi_viri / Shutterstock.com. The Japanese Pharmaceuticals and Medical Device Agency (PMDA) has granted approval for Partner Therapeutics’ Leukine (sargramostim) for inhaled use in treating autoimmune pulmonary alveolar proteinosis (aPAP). This is the first regulatory approval of an inhaled recombinant granulocyte-macrophage colony-stimulating factor (GM-CSF) product for this indication. In Japan, Leukine is branded as Sargmalin and was licensed to Nobelpharma in 2022 for certain indications. Leukine is a glycosylated recombinant human GM-CSF produced by recombinant DNA technology in yeast. Partner Therapeutics will execute the manufacturing of Sargmalin for Nobelpharma at its facility in Lynnwood, US. See Also: GSK expands Asia-Pacific reach for Trelegy Ellipta with Singapore approval UK MHRA approves Advanz Pharma antibiotic for complex infections The approval is based on the findings from the multicentre, randomised, double-blind, placebo-controlled Phase II PAGE study. It assessed Leukine in 64 subjects with mild to moderate aPAP. Subjects were randomised in a 1:1 ratio to receive either Leukine 125μg or a placebo twice a day for seven days, followed by seven days off, over 12 cycles of two weeks. The trial demonstrated a significant improvement on the primary endpoint, which was the improvement in the alveolar-arterial oxygen gradient from baseline to week 25. Subjects treated with Leukine also showed greater improvements in serum biomarker secondary endpoints, including KL-6, CEA and monocyte chemoattractant protein-1. Partner Therapeutics CEO Robert Mulroy stated: “We are grateful to Nobelpharma for their partnership, as well as their deep commitment to making this treatment available to aPAP patients in Japan. “It has been a pleasure working with their team and we look forward to ensuring the availability of Sargmalin. We also deeply appreciate the commitment of the many patients and doctors who participated in the investigator-initiated clinical trial led by Dr Nakata of Niigata University.”