INDUSTRIAL IMPACT
QIAGEN and Neuron23™ | September 15, 2022
QIAGEN and Neuron23 Inc., an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, announced the signing of an agreement to develop a companion diagnostic for Neuron23’s brain penetrant leucine-rich repeat kinase inhibitor for Parkinson’s disease.
Under the new Master Collaboration Agreement, QIAGEN will develop and validate a clinical trial assay that will detect a combination of biomarkers discovered by Neuron23 that together predict the responsiveness of Parkinson’s disease to a LRRK2 inhibitor. The partnership will support the clinical development of Neuron23’s drug candidate that is currently in the late stages of preclinical development. Subject to further clinical development, the agreement also covers options for the future development of additional companion diagnostics.
Neuron23 joins a group of more than 25 leading pharmaceutical and biotechnology companies who have reached master collaboration agreements with QIAGEN to develop and commercialize companion diagnostic tests for their drug candidates – a deep pipeline of potential future products to advance precision medicine for the benefit of patients around the world.
The assay for this collaboration will be integrated into a next-generation sequencing workflow that leverages QIAGEN’s Sample to Insight capabilities, including instrumentation, IVD sample preparation, library preparation and bioinformatics. The workflow is planned to be developed using the NextSeqTM 500 System as part of the NGS strategic collaboration between QIAGEN and Illumina. Based on Neuron23’s artificial intelligence (AI)-enabled drug discovery and biomarker platform, it will target a complex signature of 50 single nucleic polymorphisms for U.S. and European populations and eventually additional SNPs that are prevalent in Asian populations.
“The collaboration with Neuron23 shows the rapid momentum precision medicine is gaining in disease areas outside oncology. Our expertise in blood- and NGS-based molecular testing from Sample to Insight will enable Neuron23 to run a clinical trial for a drug candidate that may have the potential to modify the course of an inexorable neurodegenerative disease in a genetically defined population.”
Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics, at QIAGEN
“This collaboration combines the leading expertise of Neuron23 in drug discovery, data science, and machine learning with QIAGEN’s long-standing experience and global leadership in companion diagnostic development. QIAGEN’s blood-based test will help to identify patients with Parkinson’s disease who are likely to respond to Neuron23’s LRRK2 inhibitor. The development of a companion diagnostic identifying this sub-population of Parkinson’s disease patients will de-risk the clinical development of Neuron23’s LRRK2 inhibitor and help identify individuals who may benefit from this disease-modifying therapy. We are excited to be working with an industry leader on the first companion diagnostic developed for Parkinson’s disease,” said Nancy Stagliano, Ph.D., CEO of Neuron23.
No laboratory tests are currently available for the diagnosis of non-genetic cases for Parkinson’s disease. Usually, the disease is diagnosed based on medical history and neurological examination. Although no cure currently exists for Parkinson’s disease, therapies are used to alleviate some symptoms.
LRRK2 is a complex, multidomain protein found in neurons and many tissues and cell types throughout the body. Mutations in the LRRK2 gene are one of the most common causes of familial Parkinson’s disease and individuals who inherit gain of function mutations in LRRK2 are clearly at higher risk to develop the disease in later life. Additionally, there is emerging evidence that LRRK2 may play a role in a subset of the larger population of patients with non-familial Parkinson’s disease. Recent investigations have shown that small-molecule LRRK2 inhibitors can be neuroprotective, suggesting that therapies targeting LRRK2 could be beneficial in a larger population of patients.
QIAGEN is a pioneer in precision medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from NGS to polymerase chain reaction and digital PCR for companion diagnostic development. QIAGEN has ten PCR based companion diagnostic indications that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer and NSCLC, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer.
About QIAGEN
QIAGEN N.V., a Netherlands-based holding company, is the leading global provider of Sample to Insight solutions that enable customers to gain valuable molecular insights from samples containing the building blocks of life. Our sample technologies isolate and process DNA, RNA and proteins from blood, tissue and other materials. Assay technologies make these biomolecules visible and ready for analysis. Bioinformatics software and knowledge bases interpret data to report relevant, actionable insights. Automation solutions tie these together in seamless and cost-effective workflows. QIAGEN provides solutions to more than 500,000 customers around the world in Molecular Diagnostics, Applied Testing, Pharma and Academia. As of June 30, 2022, QIAGEN employed more than 6,100 people in over 35 locations worldwide.
About Neuron23™
Neuron23™ Inc. is an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases. Neuron23 combines recent advances in human genetics with a state-of-the-art artificial intelligence (AI)-enabled drug discovery and biomarker platform to advance therapeutics for devastating diseases. The Company’s focus areas are neurodegenerative diseases, neuroinflammatory diseases, and systemic autoimmune and inflammatory diseases. Founded in 2018, Neuron23 has assembled a world-class team of experts and entrepreneurs located in South San Francisco, CA.
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CELL AND GENE THERAPY
TEMBEXA and Smallpox | September 27, 2022
Emergent BioSolutions Inc. announced that it has completed its acquisition of exclusive worldwide rights to TEMBEXA® the first oral antiviral approved by the U.S. Food and Drug Administration for all age groups for the treatment of smallpox, from Chimerix. TEMBEXA was approved in June 2021 and is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates.
The completion of the acquisition follows the satisfaction or waiver by the parties, as applicable, of all closing conditions, including expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act), as amended, and receipt of consent from the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, for a sub-contract agreement between Chimerix and Emergent.
“The addition of TEMBEXA to our smallpox medical countermeasure franchise, which consists of our smallpox vaccine and therapeutic for smallpox vaccine complications, creates a more comprehensive offering to combat this deadly public health threat. We look forward to supporting the U.S. government’s smallpox preparedness strategy on a broader scale by executing on this BARDA contract.”
Paul Williams, SVP government/MCM business at Emergent
The 10-year contract valued at up to $680 million, to supply up to 1.7 million treatment courses of tablet and suspension formulations of TEMBEXA® to the U.S. government, was awarded to Chimerix on August 29, 2022. The contract includes an initial product procurement valued at approximately $115 million, with optional future procurement, valued at up to approximately $551 million, exercisable at the sole discretion of BARDA. In addition to product procurement, the contract includes reimbursed post marketing activities of approximately $12 million.
Financial Terms
Based on the terms of the final BARDA agreement, Emergent is expected to pay Chimerix
An upfront payment of $238 million;
Potential milestone payments of up to $124 million contingent on the potential exercise by the U.S. government of procurement options following the base period;
15% royalty on gross profit from sales of TEMBEXA outside the U.S.;
20% royalty on gross profit from sales of TEMBEXA in the U.S. that are in excess of the 1.7 million treatment courses as contemplated in the existing BARDA contract; and
Up to an additional $12.5 million upon achievement of certain development-based milestones.
ABOUT TEMBEXA
TEMBEXA is an oral antiviral approved by the FDA in June 2021 for the treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates. TEMBEXA is formulated as 100 mg tablets and 10 mg/mL oral suspension dosed once weekly for two weeks. The oral suspension formulation is particularly important for patients who have difficulty swallowing due to age or medical status. TEMBEXA is not indicated for the treatment of diseases other than human smallpox disease.
The effectiveness of TEMBEXA for the treatment of smallpox disease has not been determined in humans because adequate and well-controlled field trials have not been feasible and inducing smallpox disease in humans to study the drug’s efficacy is not ethical. TEMBEXA efficacy may be reduced in immunocompromised patients based on studies in immune deficient animals. TEMBEXA has a BOXED WARNING for increased risk for mortality when used for longer duration.
About Smallpox
Smallpox is a highly contagious disease caused by the variola virus. Historically, smallpox was one of the deadliest diseases in history with a case fatality rate of approximately 30%. Despite successful eradication of smallpox in the 1970s, there is considerable concern that variola virus could reappear, either through accidental release or as a weapon of bioterrorism. According to the U.S. Centers for Disease Control and Prevention variola virus is ranked in the highest risk category for bioterrorism agents due to its ease of transmission, high mortality rate, and potential to cause public panic and social disruption. Based on a recent report – The Department of Health and Human Services Fiscal Year 2023 Public Health and Social Services Emergency Fund Justification of Estimates for Appropriations Committee – smallpox remains a threat of high concern to both the domestic and international community.
About Emergent BioSolutions
At Emergent, our mission is to protect and enhance life. For over 20 years, we’ve been at work defending people from things we hope will never happen—so we are prepared just in case they ever do. We provide solutions for complex and urgent public health threats through a portfolio of vaccines and therapeutics that we develop and manufacture for governments and consumers. We also offer a range of integrated contract development and manufacturing services for pharmaceutical and biotechnology customers.
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MEDTECH
NeuroSense and NeuraLight | July 29, 2022
NeuroSense Therapeutics Ltd. a company developing treatments for severe neurodegenerative diseases, and NeuraLight, a company developing objective and sensitive biomarkers for neurological disorders, today announced a collaboration to advance the science of digital biomarkers in the detection and monitoring of neurological diseases including amyotrophic lateral sclerosis.
NeuraLight's platform, uses proprietary computer vision and deep learning algorithms to extract over 100 occulometic markers from facial videos captured with a standard webcam. NeuroSense is conducting a Phase IIb PARADIGM trial, which is a double-blind, placebo-controlled study evaluating the efficacy of NeuroSense's lead combination drug candidate, PrimeC, in the treatment of ALS. The NeuroSense-NeuraLight collaboration entails sharing and tracking patient data to advance the identification and use of ALS digital biomarkers in a parallel study conducted by NeuraLight. This collaboration marks NeuraLight's first clinical trial and comes on the heels of NeuraLight's $25M Series A raise.
"There is compelling value in the use of precise biomarkers to diagnose and track the progression of neurodegenerative diseases. We are very pleased to work with the stellar team at NeuraLight who are pioneering the development of digital oculometric biomarkers, which complement our extensive evaluation of biological markers of ALS. We believe these tools have the potential to enable patient stratification and increase the likelihood of success in a future NeuroSense pivotal trial of PrimeC in the treatment of ALS. This is a first step in what we envision as a long-term collaboration with NeuraLight in ALS as well as future studies we plan for Alzheimer's disease and Parkinson's disease."
NeuroSense CEO Alon Ben-Noon
Dr. Rivka Kreitman, NeuraLight's Chief Innovation Officer, commented, "Along with advancing a promising therapy for ALS, we look forward to demonstrating that oculometrics serve as a robust proxy for the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) through this partnership. The validation of our technology's efficacy will improve success rates of trials moving forward – ensuring objectivity, reliability, and replicability, as well as reducing costs and friction."
About ALS
Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years of diagnosis. In the US alone, over 5,000 patients are diagnosed yearly with ALS, and researchers anticipate the number of ALS patients in the US to grow 24 percent by 2040. The cause of ALS is unknown, making it difficult for researchers to develop disease-modifying therapies for the neurodegenerative disease.
About PrimeC
PrimeC, NeuroSense's lead drug candidate is a combination therapy that was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). NeuroSense completed a Phase IIa clinical study which successfully met its safety and efficacy endpoints including reducing functional and respiratory deterioration and statistically significant changes in ALS-related biological markers indicating PrimeC's biological activity. Through a collaboration with Massachusetts General Hospital in Boston on novel Neuron-Derived Exosomes (NDEs), NeuroSense is working to further determine the biological changes in ALS-related pathologies and the effect of PrimeC on relevant targets. Results from this study are expected Q2 2022.
About NeuraLight
NeuraLight is on a mission to transform the lives of billions of people impacted by neurological disorders by digitizing neurological evaluation and care. Our AI-driven platform integrates multiple digital markers to accelerate and improve drug development, monitoring, and precision care for patients with neurological disorders. The technology driving the platform includes proprietary Deep Learning algorithms which automatically extract a host of digital oculometric markers from facial videos captured with a standard webcam or smartphone. Our founders are repeat entrepreneurs and industry veterans (including both the co-founder of Chorus.ai and the founding CTO of Flatiron health) leading a 25-strong team, supported by renowned neurologists and 2 Nobel laureates as well as a stellar Scientific Advisory Board, and have raised $30.5M to date.
About NeuroSense
NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing treatments for patients suffering from debilitating neurodegenerative diseases. NeuroSense believes that these diseases, which include amyotrophic lateral sclerosis (ALS), Alzheimer's disease and Parkinson's disease, among others, represent one of the most significant unmet medical needs of our time, with limited effective therapeutic options available for patients to date. Due to the complexity of neurodegenerative diseases and based on strong scientific research on a large panel of related biomarkers, NeuroSense's strategy is to develop combined therapies targeting multiple pathways associated with these diseases.
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