Advanced Accelerator Applications SA

Pictured: Illustration of cancer cells being held in the crosshairs of a scope/iStock, wildpixel The final big M&A deal of 2023—BMS’s purchase of RayzeBio for $4.1 billion—centers on the development of radiopharmaceuticals, and with good reason. While targeted cancer treatment approaches such as immunotherapies have taken off, experts say they don’t work for all patients and, in some cases, lead to severe toxicities. Radiopharmaceuticals, a targeted therapy in which radiation-emitting isotopes are delivered to cancer cells, may be the answer to some of these challenges. “Trial after trial has shown the benefits of radiopharmaceuticals, which is driving the excitement in this field,” Delphine Chen, director of molecular imaging and therapy at the Fred Hutchinson Cancer Center told BioSpace. “We are seeing good responses even in patients who have undergone multiple prior therapies, something we don’t see with other therapies.” Unlike traditional radiotherapy, where the radiation is administered from outside the patient’s body, radiopharmaceutical therapy targets cancer cells using specific delivery vehicles such as monoclonal antibodies carrying radioactive isotopes. The isotopes emit radioactive β-particles or highly concentrated α particles, which cause irreparable damage to the DNA of cancer cells. Investment Accelerates Radiopharmaceuticals have been around for nearly a century, and in the 1940s, radioactive iodine was used to treat thyroid cancers. “It’s not like it’s a completely novel space,” Dominik Ruettinger, senior vice president and global head of Research & Early Development for Oncology at Bayer, told BioSpace. “But earlier, it didn’t quite work. It was mainly side effects that affected the kidneys and bone marrow that stopped these super early programs.” While still used for thyroid cancers, radiopharmaceuticals didn’t gain traction in other indications, due in part to complicated treatment protocols. But over the past two decades, this began to change. In the early 2000s, the FDA approved two β-particles emitting radiopharmaceutical therapies, Zevalin (Ibritumomab) and Bexxar (tositumomab), to treat some people with non-Hodgkin lymphoma, a type of blood cancer. Increased investment followed. In 2014, Bayer acquired Algeta, a Norwegian biotech company focused on developing α particle–emitting radiopharmaceuticals, for $2.9 billion. In 2018, Novartis spent $6 billion to acquire two radiopharmaceutical companies, Endocyte and Advanced Accelerator Application S.A. Novartis’ Pluvicto, approved in 2022 for metastatic castration-resistant prostate cancers, and Lutathera, approved in 2018 to treat gastroenteropancreatic neuroendocrine tumors, stemmed from these acquisitions. Pluvicto’s approval as the first targeted radioligand therapy for advanced prostate cancer followed successful Phase III data, which showed the drug extended overall survival by four months compared to standard-of-care for relapsed prostate cancer patients. Pluvicto has raked in more than $700 million, and Novartis expects it to become a multibillion-dollar drug, CEO Vas Narasimhan said during an investor call in October 2023. Also, in June 2021, Bayer agreed to acquire Noria Therapeutics and PSMA Therapeutics. Through this transaction, the German multinational obtained exclusive rights to a differentiated α radionuclide investigational compound based on actinium-225 and a small molecule directed toward prostate-specific membrane antigen (PSMA). More recently, Eli Lilly entered the space, acquiring Point Biopharma and its PSMA-targeted treatment, Lu-PNT2002, for $1.4 billion last October. Point and partner Lantheus Holdings revealed topline data from a Phase III trial in December, showing that the drug elicited a 3.5-month progression-free survival advantage over the control. A Targeted Advantage Of the myriad targeted cancer therapies that have been developed over the last decades, 97% have failed. One reason, research shows, is that the drugs selected for clinical investigation target the wrong pathway. Radiopharmaceuticals work independently of signaling pathways, Ruettinger said. “Radiopharmaceutical therapies work regardless of the mutational status of cancer, and [regardless] of therapies a patient may have been exposed to before,” he told BioSpace. “That is what people are so excited about.” While the clinical data looks promising, there are no head-to-head comparisons between radiopharmaceutical and other modalities, Ken Song, president and CEO of RayzeBio told BioSpace in an email. “But the level of efficacy and safety seen thus far with [radiopharmaceutical therapies] highlights the potential of it as a modality that effectively treats patients across multiple cancer types.” Song added that because radiopharmaceuticals have shown benefits across two diverse cancer types—prostate cancer and neuroendocrine tumors—he is hopeful they may work for many other cancer types as well. Seeking ‘Irreversible Damage’ Most of the current radiopharmaceuticals on the market, including Novartis’ Pluvicto and Lutathera, use an isotope called lutetium-177a β-emitting particle, which generally creates a single-stranded DNA break. These emissions travel long distances, and over a period of three days patients and their excretions emit radiations, posing a hazard to those who come into contact with them. However, RayzeBio—along with several other companies, including Perspective Therapeutics and ArtBio—is developing α-emitting radiopharmaceuticals for solid tumors, which do not require patient isolation. In contrast to β particle–emitting isotopes, α-particle isotopes don’t travel long distances, negating the need for isolation. In addition, they deliver a more concentrated dose of toxic radiation than α particles and create double-stranded DNA breaks. “With double-stranded breaks in the DNA, the damage is pretty much irreversible and there are no known mechanisms for cancer cells to repair such damage, which makes it so compelling,” Ruettinger noted. Overcoming Roadblocks Despite all these developments, two of the biggest challenges in scaling radiotherapies have been manufacturing and supply. Due to the short half-life of the radioisotopes, radiopharmaceuticals need to be delivered to a patient within 10 days of manufacturing, Ruettinger said, making the distribution of the drug a big challenge from a logistics point of view. The other problem, he said, is the supply of isotopes. In April 2023, Pluvicto reportedly experienced a supply shortage amidst rising demand. The company subsequently resolved the shortage following FDA approval for commercial production of the drug at the Novartis RLT manufacturing facility in Millburn, NJ, in 2023. The drug is made in small batches and must be given to patients within five days, making it nearly impossible to stockpile, Ruettinger pointed out. A company spokesperson told BioSpace in an email that Novartis is aiming for a production capacity of 250,000 doses in 2024 and beyond. Fred Hutch’s Chen said she believes these challenges will be resolved with time. “I agree that radiopharmaceutical therapy is poised to become big in the coming years.” Aayushi Pratap is a New York-based health and science journalist.

Agreement provides Quanterix a license to extensively studied p-Tau 217 antibodies to enable global access to plasma-based testing for Alzheimer’s Disease research and diagnostics BILLERICA, Mass.--(BUSINESS WIRE)-- Quanterix Corporation (NASDAQ: QTRX), a leading provider of ultra-sensitive research products and high-definition diagnostics, today announced the signing of a license agreement with Janssen Sciences Ireland UC (Janssen), a Johnson & Johnson (J&J) Company. Under this agreement, Quanterix will receive worldwide, non-exclusive rights to J&J’s extensively studied p-Tau 217 antibodies and assay designs for potential use in clinical research and diagnostic products, further strengthening Quanterix’s position at the forefront of the Alzheimer's Disease (AD) biomarker field. Under the terms of the license agreement, Janssen will grant Quanterix a worldwide non-exclusive license for J&J-developed technology to produce Simoa p-Tau 217 research-use only (RUO) assay kits for global distribution. Furthermore, under the agreement Quanterix will launch a Laboratory Developed Test (LDT) based on the J&J p-Tau 217 antibodies and assay, offered through Accelerator under the Lucent Diagnostics brand. These advances mark a significant milestone for Quanterix's continued leadership in advancing Alzheimer's Disease diagnosis and treatment, providing a path for the first scalable immunoassay-based p-Tau 217 test to potentially become widely available to researchers and clinicians. p-Tau 217 has emerged as a top performing biomarker for AD, enabling clinical sensitivity and specificity in blood1. Highly sensitive and specific blood-based biomarker tests may improve diagnosis by expanding access to treatments for millions of individuals with early Alzheimer’s Disease, which is currently detected using positron emission tomography (PET) or lumbar puncture for CSF biomarkers. These methods are expensive, lack wide availability, and are invasive. There is growing consensus that high performing blood-based biomarker tests may be an appropriate alternative for patient care. "This collaboration demonstrates the scientific advances that are possible when Simoa’s ultra-sensitive detection is combined with clinically validated antibodies and critical reagents,” said Masoud Toloue, CEO of Quanterix. “Providing access to high-performing blood-based p-Tau 217 assays are an important step as we work with several partners to build the global infrastructure for non-invasive testing of Alzheimer’s Disease.” For more information about Quanterix’s work in neurology, visit: . About Quanterix From discovery to diagnostics, Quanterix’s ultrasensitive biomarker detection is fueling breakthroughs only made possible through its unparalleled sensitivity and flexibility. The Company’s Simoa® technology has delivered the gold standard for earlier biomarker detection in blood, serum or plasma, with the ability to quantify proteins that are far lower than the Limit of Quantification (LoQ) of conventional analog methods. Its industry-leading precision instruments, digital immunoassay technology and CLIA-certified Accelerator laboratory have supported research that advances disease understanding and management in neurology, oncology, immunology, cardiology and infectious disease. Quanterix has been a trusted partner of the scientific community for nearly two decades, powering research published in more than 2,000 peer-reviewed journals. Find additional information about the Billerica, Massachusetts-based company at or follow us on Twitter and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “expect,” “plan,” “anticipate,” “estimate,” “intend” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements in this news release are based on Quanterix’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Factors that may cause Quanterix’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Quanterix’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” sections contained therein. Except as required by law, Quanterix assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. 1Brum WS, Cullen NC, Janelidze S, et al. A two-step workflow based on plasma p-tau217 to screen for amyloid β positivity with further confirmatory testing only in uncertain cases. Nat Aging. 2023;3(9):1079-1090

The Therapeutics Accelerator (the 'Accelerator') will identify, fund and advance innovative projects from academic partners in the U.K. and U.S. with the goal to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years, targeting multiple approvals in key markets. The Accelerator will operate a unique non-profit/for-profit model with the intention to commit up to £200 million ($250 million). The Accelerator will receive support from a new partnership between University Hospitals in Cleveland, Ohio, University of Oxford and investment company Oxford Science Enterprises. Former U.K. Prime Minister David Cameron to lead the international efforts of the Accelerator as Chair of the Oxford-Harrington Rare Disease Centre Advisory Board. OXFORD, United Kingdom and CLEVELAND, Oct. 3, 2023 /PRNewswire/ -- The University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland, Ohio announced the launch of the Oxford-Harrington Rare Disease Centre Therapeutics Accelerator with an official signing event at the University of Oxford. The event was hosted by the Oxford-Harrington Rare Disease Centre (OHC), a partnership established in 2019 between the University of Oxford and Harrington Discovery Institute. Attendance at the signing included leaders from OHC, the University of Oxford, the Harrington family, University Hospitals, and Oxford Science Enterprises. The OHC unites world-leading research across the U.K. and U.S. with expertise in therapeutics development by combining University of Oxford's scientific leadership and resources (250+ principal investigator scientists working on over 350 rare diseases) with Harrington Discovery Institute's track record of advancing breakthrough medicines into clinical development (177 medicines funded, 36 companies launched, 19 medicines into the clinic, and 13 licenses to pharma companies since its founding in 2012). The Accelerator is a first-in-kind, transatlantic initiative to identify, fund and advance breakthrough academic discoveries to deliver new treatments for the 400 million people worldwide who suffer from rare diseases. Its goal is to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years and target multiple approvals from regulators in key markets including the U.S., the U.K. and Europe. David Cameron, the former U.K. Prime Minister, who was present at the launch event, will lead the international efforts of the Accelerator under his role as Chair of the Oxford-Harrington Rare Disease Centre Advisory Board. David Cameron said: "I know from my own experience of having a son born with an incredibly rare and incurable disease, how little we really know about rare diseases. Although individually they may only affect small numbers of patients like my son, Ivan, collectively they represent a major global health crisis that affects millions of patients and their families in life-changing ways. As Prime Minister and since, I have been fortunate to follow the enormous scientific breakthroughs that have taken place over the last decade, such as the use of genomics, which opened my eyes to how much we could know about rare diseases. That's why I set up Genomics England and personally championed the 100,000 Genomes Project - now expanded to sequence 5 million adults across the UK - and that is why today I am so pleased to back this unique Rare Disease Therapeutics Accelerator, designed to make a transformational, global impact on the treatment of these devastating and often neglected diseases. I have long said that beating these diseases is within our grasp. This Accelerator will deliver new, real and tangible therapies for patients and, by doing so, offer huge benefits for patients, their families and wider society. I am delighted to play my part in making it a reality." The OHC prioritizes rare genetic diseases affecting children or adults where there is urgent need and opportunity for greatest impact and that fall into the areas of rare neurological diseases, cancers and developmental diseases. Projects will be sourced from academic labs across the U.K. and U.S. The Accelerator will operate a unique non-profit/for-profit model with the intention to commit up to £200 million into new projects. Project funding will be paired with research, drug development, commercial strategy and business development expertise from pharma-experienced industry leaders with a track record of bringing new drugs to market. The Accelerator will receive support from a new co-funding and co-investment agreement between University Hospitals in Cleveland, Ohio, University of Oxford and investment company Oxford Science Enterprises, and aims to ensure that capital is deployed efficiently to advance a sustainable pipeline of new therapies either through academia or via the creation of venture-backed spinout companies. The first investment by the Accelerator was made in September 2023 in conjunction with the creation of AlveoGene, a new U.K. company launched to develop innovative inhaled gene therapies and transform outcomes in patients with rare respiratory diseases. The partners are also collaborating to create a dedicated Rare Disease Impact Fund to enable investments in mission-aligned projects and accelerate the development of rare disease therapeutics. Sir John Bell, Regius Professor of Medicine at the University of Oxford and member of the OHC Steering Committee, said: "It requires true innovation, collaboration, vision and commitment to make meaningful progress in changing the global rare disease landscape for patients, their families and society. Since the creation of the OHC in 2019 we have made good progress along this path – and the hope is that by building on this foundation with this exciting new initiative, we can really create momentum to drive the translation of cutting-edge science into life-changing therapies targeting some of the most debilitating diseases." Jonathan Stamler, MD, President and Founder of Harrington Discovery Institute, said: "Harrington has been supporting the advancement of treatments for rare and other diseases for more than a decade, committing significant funds and resources to this global challenge with considerable success. Our commitment is to major unmet needs in society to better the world. This new Accelerator and extended collaboration with Oxford and Oxford Science Enterprises represents a major step forward towards finding the best medicines that improve outcomes for the millions of individuals and families affected by rare diseases." Matthew Wood, Professor of Neuroscience and Director of the OHC, said: "OHC was set up with a bold vision and ambitious goals to make an impact on the treatment of rare diseases. This new Accelerator initiative takes this a step further with its unique collaboration and for-profit and not-for-profit model. It is only by combining resources and adopting innovative approaches that we can truly accelerate the development of new drugs for patients in need. Already we have seen many exciting opportunities and look forward to engaging with scientists at the forefront of rare disease research." Georg Holländer, Head of Department of Paediatrics at the University of Oxford, said: "This is a great moment for Paediatrics at Oxford as it expands its commitment to childhood diseases across the U.K. and the U.S. There is so much great research being done on these diseases and only through exciting multi-organizational collaborations like the OHC and the Accelerator can we make the progress we need to bring hope to patients and their families around the world." Ed Bussey, CEO at Oxford Science Enterprises, said: "As a company focused on translating scientific breakthroughs into commercially successful businesses, we are thrilled to partner with OHC on the introduction of a Rare Disease Therapeutics Accelerator. Through this initiative we hope to uncover the most promising opportunities, creating and building innovative new companies with the goal of improving patient outcomes for people impacted by rare diseases globally." Ronald G. Harrington, Entrepreneur and Philanthropist, said: "Our family is thrilled to see the extraordinary progress that Harrington Discovery Institute has made since we launched in 2012. It is humbling and gratifying that our efforts are recognized and supported by so many participants in the industry from disease foundations to prestigious universities and innovative biopharmaceutical companies. The launch of this new rare disease initiative and extension of our relationship with Oxford is a further example of our ambition to make a meaningful positive impact on global health challenges affecting millions of people around the world." Cliff A. Megerian, MD, FACS, CEO, University Hospitals, said: "Every day, through the work we do at University Hospitals, we are inspired by the courage and determination of patients with rare diseases and their families. This gives us a real sense of urgency and motivation to make a difference in the lives of these families and we are fully committed to supporting this new Accelerator and the efforts of our transatlantic team and partners in their mission." Daniel I. Simon, MD, President, Academic & External Affairs and Chief Scientific Officer, University Hospitals, said: "Our health system is focused on translating discoveries to address unmet clinical needs. Leveraging the unique Harrington model in the Oxford-Harrington Rare Disease Centre will catalyze the development of new medicines for hundreds of thousands of patients across the globe." For academics and researchers to find out more about the Therapeutics Accelerator at the Oxford-Harrington Rare Disease Centre and how to apply, please go to OxfordHarrington.org. Notes to Editors: About Rare Diseases More than 400 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are about 7,000 known rare diseases, with new diseases being discovered every day. A rare disease affects one in 10 Americans, or 10 percent of the US population. Similarly, Europe has approximately 30 million people who suffer from a rare disease. The majority of all rare diseases are genetic in origin, which means they are present throughout a person's life. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA) and similar estimates have been made for treatments approved by the European Medicine Agency (EMA). Therefore, someone with a rare disease today faces a lifelong, often life-threatening, condition with little hope for a cure, or even an effective treatment option. About Oxford-Harrington Rare Disease Centre The Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, U.K. and Harrington Discovery Institute at University Hospitals, Cleveland, OH, USA. OHC combines world-leading strengths in research and therapeutics development from across these organizations, to set the science and innovation agenda, driving cutting-edge rare disease breakthroughs to address the unmet need in rare disease across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | Twitter About the University of Oxford Medical Sciences Division Oxford University's Medical Sciences Division is one of the largest biomedical research centres in Europe, with over 2,500 people involved in research and more than 2,800 students. The University is rated the best in the world for medicine and life sciences, and it is home to the UK's top-ranked medical school. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. About Oxford Science Enterprises Oxford Science Enterprises (OSE) is an independent, billion-pound investment company, created in 2015 to found, fund and build transformational businesses via its unique partnership with the University of Oxford, the world's #1 research university. This partnership enables OSE to work with the brightest academic minds tackling the world's toughest challenges and guarantees unrivalled access to their scientific research. In collaboration with its global network of entrepreneurs and advisers, OSE goes well beyond funding to shape and nurture these complex ideas into successful businesses. Actively focused on a core portfolio of around 40 companies spanning three high-growth, high-impact sectors – Life Sciences, Health Tech, and Deep Tech – the company adopts a flexible, long-term investment approach, recognising the path from ground-breaking research to global markets takes time and resilience. To date, OSE has invested £0.6 billion in over 80 ambitious companies built on Oxford science. For more information, visit: oxfordscienceenterprises.com | Twitter | LinkedIn About Harrington Discovery Institute The Harrington Discovery Institute at University Hospitals in Cleveland, OH – part of The Harrington Project for Discovery & Development – is a translational accelerator that advances promising discoveries from academic labs into the clinic for the benefit of patients and society. The institute was created in 2012 with a $50 million founding gift from the Harrington family and is a prime example of the commitment they share with University Hospitals to a Vision for a 'Better World'. For more information, visit: HarringtonDiscovery.org | LinkedIn |Twitter About University Hospitals, Cleveland, Ohio Founded in 1866, University Hospitals serves the needs of patients through an integrated network of 21 hospitals (including five joint ventures), more than 50 health centers and outpatient facilities, and over 200 physician offices in 16 counties throughout northern Ohio. The system's flagship quaternary care, academic medical center, University Hospitals Cleveland Medical Center, is affiliated with Case Western Reserve University School of Medicine, Northeast Ohio Medical University, Oxford University, the Technion Israel Institute of Technology and National Taiwan University College of Medicine. The main campus also includes the UH Rainbow Babies & Children's Hospital, ranked among the top children's hospitals in the nation; UH MacDonald Women's Hospital, Ohio's only hospital for women; and UH Seidman Cancer Center, part of the NCI-designated Case Comprehensive Cancer Center. UH is home to some of the most prestigious clinical and research programs in the nation, with more than 3,000 active clinical trials and research studies underway. UH Cleveland Medical Center is perennially among the highest performers in national ranking surveys, including "America's Best Hospitals" from U.S. News & World Report. UH is also home to 19 Clinical Care Delivery and Research Institutes. UH is one of the largest employers in Northeast Ohio with more than 30,000 employees. Follow UH on LinkedIn, Facebook and Twitter. For more information, visit UHhospitals.org. SOURCE Harrington Discovery Institute