Fujirebio, Inc.

A protein called alpha-synuclein that accumulates and ravages the brains of some people with Parkinson’s disease is the target of several experimental drugs. Until recently, the only way to detect it was by opening someone’s brain when they died. That’s starting to change. Amprion, a 30-person biotech based in San Diego, has developed a way to detect small amounts of alpha-synuclein from spinal fluid. A study published last year in The Lancet Neurology suggests the method could help confirm suspected diagnoses and even predict Parkinson’s before common symptoms arise. And now Amprion is scaling up to commercialize the test. CEO and co-founder Russell Lebovitz told Endpoints News in an exclusive interview that Amprion has raised $6 million in the first portion of its Series B, which will help it hire 15 more employees and expand its salesforce and operations throughout the US. The round was led by Formation Venture Engineering, with participation from Eli Lilly. Lilly’s interest is notable because the pharma company helped pioneer the use of diagnostics to detect amyloid in the brains of people with Alzheimer’s disease. The drugmaker is also developing drugs for Parkinson’s, and the company’s scientific leaders have mentioned alpha-synuclein as an area of interest for future neurodegenerative research at the company. “They have recognized, as have a lot of the drug companies, that this is going to be important in the future,” Lebovitz said. The technology underlying Amprion’s test was forged in the early 2000s in the aftermath of the mad cow disease crisis. Claudio Soto, a biochemist and professor of neurology at the UT Health Houston McGovern Medical School in Houston, developed a way to detect prions, the misfolded proteins that cause bovine spongiform encephalopathy in cattle and Creutzfeldt-Jakob disease in humans. The infectious proteins cause natural versions of the proteins to misfold, leaving tainted and toxic prions in their wake as they spread through the brain. That natural amplification formed the basis of Soto’s test. He added highly purified and properly folded proteins to spinal fluid or blood from a patient suspected of having the disease. If prions were present in the sample, they would quickly convert the normal proteins into diseased ones. He compared the method to PCR, the technique used to make many copies of DNA for easier sequencing. Soto formed a startup with Lebovitz in 2007 that aimed to refine the technique, now dubbed a seed amplification assay. The mad cow crisis abated, but the duo believed the technique would be valuable to help detect other misfolded proteins, such as amyloid and tau in Alzheimer’s and alpha-synuclein in Parkinson’s. The first ten years were slow going. The test originally took nearly two weeks to run, but they got it down to 20 hours. And a fluorescent dye that only lights up in the presence of misfolded proteins made the results easy to interpret. The tests are currently run out of its CLIA-certified lab in San Diego. The company is developing a simplified kit of the test that other labs can run, and hopes to submit it to the FDA in about 18 months. By 2019, Amprion had developed an alpha-synuclein test for Parkinson’s that it believed would be scalable. The company raised a $13.7 million Series A over the next three years and began offering the test to doctors at major medical centers and research institutions in 2023, Lebovitz said. The study published last year revealed that the test had a sensitivity of about 88%, meaning that about 12 in 100 people with Parkinson’s had a false negative result. Among the 1,100 healthy volunteers, the test had a 97% specificity, meaning about 3 in 100 people without the disease had a false positive. The test performed more poorly for certain subsets of Parkinson’s disease, including those with mutations in the LRRK2 gene linked to the disease. Drugmakers and regulators alike are increasingly turning to biomarkers to decide which patients to include in a clinical trial and how to assess whether the drug is working. In August, the FDA issued a letter of support encouraging “further study and use of” seed amplification assays for alpha-synuclein. Amprion has worked on similar amplification assays for amyloid and tau, but these tests are still experimental. The company offers clinical tests for amyloid, tau and another brain biomarker called neurofilament using machines sold by Fujirebio. With the new funding, Leboviz said “we can hit every major medical center doing cognitive and motor disorders in the US.” He’s hoping to raise another $9 million in the Series B round this year to help the company open a lab in Europe. But Amprion’s efforts to scale up come at a challenging time. About two weeks ago, an investigation published in Science cast doubt on several research papers supporting the role of alpha-synuclein in Parkinson’s disease. More than 130 papers published by National Institutes of Health neuroscientist Eliezer Masliah had “suspect images,” including doctored and duplicated results, according to the report. Some of the questionable studies were cited as evidence underpinning the alpha-synuclein-targeting antibody prasinezumab, which is being developed by Prothena Biosciences and Roche. Lebovitz said that other scientists have consistently demonstrated alpha-synuclein’s role in Parkinson’s and other neurodegenerative diseases irrespective of Masliah’s studies. “None of Amprion’s data relies on any work ever performed or published by Eliezer Masliah,” Lebovitz said. “We have never met with Dr. Masliah, and he has never provided any advice or other counsel.”

New investment supports DxA's mission to advance the development of scalable and universally accessible diagnostics. Blood tests will be key to further evolving Alzheimer's diagnostics, care, and research. NEW YORK, Sept. 25, 2024 /PRNewswire/ -- The Alzheimer's Drug Discovery Foundation (ADDF) today announced a new investment in C2N Diagnostics. The $7 million investment is the largest to date from the ADDF's Diagnostics Accelerator (DxA) and underscores the DxA's commitment to developing and deploying novel biomarkers, including blood tests, that will transform the Alzheimer's diagnostic landscape, a core tenet of the ADDF's mission. Scalable, accessible blood-based biomarkers will be critical to enabling a combination therapy and precision medicine approach in Alzheimer's, like in cancer care. "Blood tests are transforming Alzheimer's disease detection and will continue to play a critical role in the future of Alzheimer's treatment and research," notes Howard Fillit, MD, Co-Founder and Chief Science Officer at the ADDF. "PTau217 has emerged as the blood-based biomarker gold standard for diagnostic purposes and studies show that C2N's PrecivityAD2® test has industry-leading sensitivity and accuracy, positioning these tests to replace more invasive PET scans and spinal taps. This investment will improve this critical tool's accessibility, a pillar of the DxA's mission to develop diagnostics for the millions of patients living with this disease." C2N's pioneering blood tests are rooted in scientific rigor with a history of breakthroughs. Notably, the company's PrecivityAD® blood test was first-to-market with the help of ADDF seed-funding. With this new investment, C2N intends to develop a deployable version of its blood biomarker detection platform, like well-known diagnostics platforms such as Roche's Elecsys and Fujirebio's Lumipulse, that will enable global access to the test. This investment builds upon the ADDF and C2N's longstanding partnership. "C2N Diagnostics gratefully appreciates the Alzheimer's Drug Discovery Foundation's DxA for their trust and confidence in our approach and for providing this critical funding. We recognize the significance of being the largest recipient to date of DxA funding and take that responsibility very seriously," says Joel Braunstein, MD, CEO and Co-Founder of C2N. "We're intensely focused on our goal to develop an end-to-end clinical mass spectrometry solution. This platform will enable high-performance Alzheimer's disease testing on a global scale. C2N believes a decentralized process for high-performance testing, developed and qualified in a regulatory-compliant manner, is a requisite to address the growing global demand for early and accurate disease detection." Blood-based biomarkers are one part of the DxA's diverse portfolio, which also includes ocular tests and digital tools. To date, the DxA has invested more than $70 million in over 60 projects. Along with blood tests, the future of Alzheimer's diagnostics will incorporate digital and ocular tools that will not only provide patients with early and accurate diagnoses, but also have the potential to be used for screening, prognostic, and prevention efforts. "Sensitive and accurate biomarkers are crucial to support early diagnosis and intervention, which studies show can have considerable impact on disease progression," says Niranjan Bose, PhD, B Pharm, Managing Director of Health & Life Sciences at Gates Ventures. "The true measure of the value of a biomarker is whether it's accessible and scalable enough to promote adoption and subsequently transform the diagnostic landscape—which is what we hope to do here." Advancements in Alzheimer's blood tests will help accelerate drug development and clinical trials by providing faster and more cost-effective ways to enroll patients into trials and will aid in monitoring the target engagement of new drugs. Ultimately, blood tests and novel biomarkers will support the future of combination therapy and precision medicine—the holy grail of Alzheimer's treatment. ABOUT THE ALZHEIMER'S DRUG DISCOVERY FOUNDATION (ADDF) Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer's Drug Discovery Foundation is dedicated to rapidly accelerating the discovery of drugs to prevent, treat and cure Alzheimer's disease. The ADDF is the only public charity solely focused on funding the development of drugs for Alzheimer's, employing a venture philanthropy model to support research in academia and the biotech industry. The ADDF's leadership and contributions to the field have played a pivotal role in bringing the first Alzheimer's PET scan (Amyvid®) and blood test (PrecivityAD®) to market, as well as fueling the current robust and diverse drug pipeline. Through the generosity of its donors, the ADDF has awarded more than $290 million to fund over 750 Alzheimer's drug discovery programs, biomarker programs and clinical trials in 20 countries. To learn more, please visit: . ABOUT THE DIAGNOSTICS ACCELERATOR (DxA) The Diagnostics Accelerator, created in July 2018, is a $100 million global research initiative from partners including ADDF Co-Founder Leonard A. Lauder, Bill Gates, Jeff Bezos, MacKenzie Scott, the Dolby family, the Charles and Helen Schwab Foundation, The Association for Frontotemporal Degeneration, among others, to develop novel biomarkers for the early detection of Alzheimer's disease and related dementias. This research initiative is dedicated to accelerating the development of affordable and accessible biomarkers to diagnose Alzheimer's disease and related dementias and advance the clinical development of more targeted treatments. Through translational research awards and access to consulting support from industry experts, this program will challenge, assist and fund the research community in both academia and industry to develop novel peripheral and digital biomarkers. ABOUT C2N DIAGNOSTICS, LLC C2N Diagnostics is a specialty diagnostics company with a vision to bring Clarity Through Innovation®. C2N strives to provide exceptional clinical laboratory services and advanced diagnostic solutions in the field of brain health. C2N's high-resolution mass spectrometry-based biomarker services and products are used for: clinical decision-making to improve patient care, including diagnosis and treatment monitoring; maximizing the quality and efficiency of clinical trials that test novel treatments for neurodegeneration; and providing innovative tools to help healthcare researchers better understand novel mechanisms of disease, identify new treatment targets, and conduct important epidemiologic studies to improve global public health. C2N assays have been used in over 150 Alzheimer's disease and other research studies throughout the U.S. and the world. This includes landmark treatment and prevention trials involving disease-modifying therapies (DMTs) that are changing the trajectory of Alzheimer's disease. C2N has ongoing collaborations with multi-national pharmaceutical and biotech companies, leading academic institutions, National Institute on Aging, Alzheimer's Association, and other non-profits and consortiums. Over 30,000 Precivity™-related biomarker measures have been reported through peer-reviewed publications, with many more manuscripts currently under review. C2N has partnered with numerous leading clinical diagnostic labs throughout the world to offer expanded Precivity™ testing access to providers and patients, including Unilabs across 75 countries, Grupo Fleury in Brazil and Healius Pathology in Australia for clinical use, and with Mediford Corporation in Japan for research purposes. C2N also recently announced it has entered into a non-exclusive agreement with Mayo Clinic Laboratories. The company acknowledges generous support from National Institute on Aging, GHR Foundation, Alzheimer's Drug Discovery Foundation, Alzheimer's Association and BrightFocus Foundation. For more information visit . ABOUT PRECIVITY™ TESTS The Precivity™ tests are innovative new blood tests intended for use in patients 55 and older with signs or symptoms of mild cognitive impairment or dementia. These tests aid healthcare providers in the detection of amyloid plaques in the brain, a pathological hallmark of Alzheimer's disease, and inform medical management and treatment decisions. ABOUT MASS SPECTROMETRY A mass spectrometer is a device that produces charged particles (ions) from biological substances and biomarkers present in complex tissues and fluids like blood. The platform then uses electric and magnetic fields to measure the mass of the charged particles. Liquid chromatography-tandem mass spectrometry (LC-MS/MS) offers advantages of true multiplexing, quantitative accuracy, high sensitivity, high specificity, and wide dynamic range, enabling definitive identification and quantification of Alzheimer's disease biomarkers at both low and high abundance. High resolution LC-MS/MS is an ideal platform to evaluate multiple complex biomarkers simultaneously to help optimize diagnostic accuracy. SOURCE Alzheimer's Drug Discovery Foundation WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

MALVERN, Pa. & TOKYO--( BUSINESS WIRE )--Fujirebio today announced that its wholly-owned subsidiary Fujirebio Diagnostics, Inc. has filed its Lumipulse ® G pTau 217/β-Amyloid 1-42 Plasma Ratio in-vitro diagnostic (IVD) test with the U.S. Food and Drug Administration (FDA). The new plasma test is expected to be the first commercially available blood-based IVD test in the U.S. to help in the assessment of Alzheimer’s disease (AD). The Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio, which received Breakthrough Device Designation from the FDA, is an IVD test using measurable pTau 217 and β-Amyloid 1-42 concentrations found in human plasma. The test combines these concentrations into a numerical ratio of pTau 217/β-Amyloid 1-42 to identify patients with amyloid pathology associated with AD. β-Amyloid neuritic plaque pathology in the brain is believed to contribute to the loss of cognitive function that characterizes AD. AD is a devastating condition that afflicts more than 6 million Americans and is a leading cause of disability and death. Access to reliable diagnostic tools is currently limited. Diagnosis involves a variety of subjective measures and costly or invasive procedures, such as amyloid positron emission tomography (PET) scans and cerebral spinal fluid (CSF) tests. As a result, many patients are not diagnosed until their disease has advanced, limiting the benefits of available treatment options. The Lumipulse pTau 217/β-Amyloid 1-42 Plasma Ratio test uses Fujirebio’s fully automated Lumipulse G1200 instrument system, which is widely available in clinical laboratories throughout the U.S. The new ratio test complements the Lumipulse G β-Amyloid Ratio (1-42/1-40), authorized by the FDA for use in CSF in May 2022. “The lack of effective and accessible diagnostics for AD contributes to its late diagnosis and inadequate treatment,” says Monte Wiltse, President and CEO at Fujirebio Diagnostics, Inc. “We designed our Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio test to help physicians and patients with signs and symptoms of cognitive decline obtain an AD diagnosis much earlier when interventions are more effective. An early and accurate diagnosis will also facilitate the development of new drug therapies, which are urgently needed as the prevalence of AD increases with a rapidly aging population globally. As part of our worldwide commitment to improving the diagnosis and treatment of AD, Fujirebio is developing additional assays, which will increase the availability of diagnostic tools and expand the foundation for early, more effective treatment.” About Fujirebio Fujirebio, a member of H.U. Group Holdings Inc., is a global leader in the field of high-quality in vitro diagnostics (IVD) testing. It has more than 50 years’ accumulated experience in the conception, development, production, and worldwide commercialization of robust IVD products. Fujirebio was the first company to develop and market CSF biomarkers under the Innogenetics brand over 25 years ago. Fujirebio remains the only company with such a comprehensive line-up of manual and fully automated neurodegenerative disease assays and consistently partners with organizations and clinical experts across the world to develop new pathways for earlier, easier and more complete neurodegenerative diagnostic tools. More information can be found at www.fujirebio.com/alzheimer . About Fujirebio Diagnostics, Inc. Fujirebio Diagnostics, Inc., a wholly-owned subsidiary of Fujirebio Holdings, Inc., is the premier cancer diagnostics company and the industry leader in cancer biomarker assays. The company pioneered and introduced the CA125 test, the first FDA-approved ovarian cancer biomarker, over 25 years ago. Fujirebio Diagnostics specializes in the clinical development, manufacturing and commercialization of in-vitro diagnostic products for the management of human disease states, with an emphasis in oncology. For more information about Fujirebio Diagnostics, please call +1 610-240-3800 or visit us at www.fujirebio.com .