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Pictured: Novartis' logo outside its building in San Diego, California/iStock, JHVEPhoto Novartis on Tuesday expanded its peptide discovery collaboration with Japanese biotech PeptiDream in a deal worth more than $2.7 billion in milestone payments, making it the latest move in the red hot radiopharma sector. Under the agreement, PeptiDream use its technology–the Peptide Discovery Platform System—to find new macrocyclic peptides against targets that Novartis has selected. These targets can be conjugated into radioligand therapies (RLTs), which may have other therapeutic or diagnostic uses. Novartis is paying PeptiDream $180 million upfront and is also eligible to receive $2.71 billion in milestone payments. The biotech may also net tiered royalties on any product sales. PeptiDream’s stock price jumped 24% on the news in Tuesday trading. Novartis and PeptiDream have been working together on various deals since 2010. In 2019, the two companies agreed to find new peptide-drug conjugates. The terms of that partnership were not disclosed. “We are delighted to further expand our long-standing macrocyclic peptide discovery collaboration with Novartis, adding additional peptide RLT and other programs to our strong partnership,” PeptiDream CEO Patrick Reid said in a statement. “As macrocyclic peptides continue to be the ideal vectors for the targeted deliver of a variety of therapeutic payloads, PeptiDream continues to build an extensive pipeline of partnered as well as internal peptide-conjugate programs.” PeptiDream has also been active in the deal space as the biotech received $40 million upfront from Roche’s Genentech last year to discover radioisotope drug conjugates. The agreement is also worth up to $1 billion in milestones. The biotech has also previously initiated discovery deals with Eli Lilly and Merck, among others. “Our expanded partnership reflects our shared commitment to pioneering science that broadens the scope of transformative therapeutic approaches such as RLT,” Shiva Malek, global head of oncology research at Novartis, said in a statement. Radiopharmaceuticals have been a big investment area for Novartis. In 2018, the pharma spent over $6 billion to acquire two companies, Advanced Accelerator Application S.A. and Endocyte. Out of those deals, Novartis gained access to the prostate cancer treatment Pluvicto and Lutathera. These radioligands have been good earners for Novartis, as Pluvicto raked in $310 million in the first quarter of 2024 while Lutathera brought in $169 million. The radiopharma deal space has been active in the past year as AstraZeneca inked a potential $2.4 billion deal in March 2024 to acquire Fusion Pharmaceuticals, gaining access to its radio-conjugate candidate FPI-2265, a treatment for prostate cancer. Last year, Bristol Myers Squibb spent $4.1 billion to purchase RayzeBio, securing access to its lead radiopharma program. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.

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