MEDICAL, INDUSTRY OUTLOOK
Businesswire | May 11, 2023
Bayer and Bicycle Therapeutics plc a biotechnology company pioneering a new and differentiated class of therapeutics by utilizing proprietary bicyclic peptides technology (Bicycle®), announced that they have entered into a strategic collaboration agreement to discover, develop, manufacture, and commercialize Bicycle radioconjugates for multiple agreed upon oncology targets.
Bicyclic peptides are peptides consisting of 9-20 amino acids that can be synthetically manufactured and bind to targets with high affinity and selectivity, affording high tumor penetration and fast excretion from healthy organs. They are chemically synthesizable, featuring a low molecular weight and tunable pharmacokinetics, with a large surface-area for molecular interactions that allows protein-protein interactions to be targeted. The two companies will jointly use Bicycle’s peptide technology to develop bicyclic peptides for several undisclosed oncology targets. Targeted radiotherapies are an innovative class of cancer therapies. Due to their unique mode of action, they have the potential to unlock a broad opportunity space and serve patients in high-unmet medical need indications.
“At Bayer, we enter strategic collaborations to expand our access to innovation,” said Christian Rommel, Ph.D., Global Head of Research and Development and Member of the Executive Committee, Pharmaceuticals Division, Bayer. “With Bicycle’s proprietary peptide-based technology, we continue to strengthen our oncology development pipeline by adding next-generation targeted radiotherapeutics to address high unmet medical needs of cancer patients.”
“Bayer is a pioneer in the radiopharmaceuticals space and provides new and additional validation of our unique technology,” said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. “We believe our bicyclic peptide platform, coupled with Bayer’s scale and expertise in developing radiopharmaceuticals, has the potential to deliver improved clinical outcomes for patients with cancer. We look forward to collaborating with Bayer to bring forth new potential first-in-class radiopharmaceutical treatments based on Bicycles.”
Under the terms of the agreement, Bayer and Bicycle will collaborate on the development of bicyclic peptides for multiple oncology targets. Bicycle will utilize its proprietary phage platform to discover and develop bicyclic peptides and Bayer will be responsible for, and fully fund, all further preclinical and clinical development, manufacturing, and commercialization activities. Bicycle will receive a $45 million upfront payment and with potential development and commercial-based milestone fees, payments to Bicycle could total up to $1.7 billion. Bicycle will also be eligible to receive mid-single to double-digit tiered royalties on Bicycle-based medicines commercialized by Bayer. The closing of the transaction is subject to clearance of the transaction under the U.K. National Security and Investment Act 2021.
About Bicycle Therapeutics
Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, referred to as Bicycles, for diseases that are underserved by existing therapeutics. Bicycles are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycles attractive candidates for drug development. Bicycle is evaluating BT5528, a second-generation Bicycle Toxin Conjugate (BTC™) targeting EphA2; BT8009, a second-generation BTC targeting Nectin-4, a well-validated tumor antigen; and BT7480, a Bicycle TICA™ targeting Nectin-4 and agonizing CD137, in company-sponsored Phase I/II trials. In addition, BT1718, a BTC that targets MT1-MMP, is being investigated in an ongoing Phase I/IIa clinical trial sponsored by the Cancer Research UK Centre for Drug Development. Bicycle is headquartered in Cambridge, UK, with many key functions and members of its leadership team located in Cambridge, MA.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Prnewswire | April 12, 2023
Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced that it has entered into an agreement with Fulgent Genetics a technology-based company with a well-established clinical diagnostic business and a therapeutic development business. Fulgent Genetics is now able to offer single-cell multi-omics on the Tapestri Platform on its fully customizable menu of services, including whole genome, whole exome, RNA sequencing, tumor profiling, methylation sequencing, liquid biopsy, single cell sequencing, spatial biology, and pathology services to support its growing pharma client demands. Additionally, Fulgent Genetics will evaluate the Tapestri Platform for applications in clinical development to streamline the drug development and approval process.
Over the last ten to fifteen years, there has been a paradigm shift in clinical trials towards precision oncology by using biomarkers to select or enrich trial cohorts. The Tapestri Platform's ability to do single-cell analysis of genotype and phenotype from the same cell can provide the sensitivity and resolution for biomarker discovery unattainable by current bulk methods. Single-cell resolution can reveal treatment resistance mechanisms that may help to better stratify patients in clinical trials. While on treatment, residual levels of disease can be detected with the high sensitivity of single-cell analysis. Characterizing clonal heterogeneity and tracking clonal evolution during treatment can potentially guide adaptive and rational combination therapies for better patient stratification and better clinical trial outcomes.
"The increasing adoption of the Tapestri Platform for translational research and clinical development by many cancer centers and global pharma companies demonstrates the necessity of single-cell multi-omics," said Dr. Hanlin (Harry) Gao M.D., Ph.D., D.A.B.M.G., F.A.C.M.G., Chief Scientific Officer of Fulgent Genetics. "The relationship between Fulgent Genetics and Mission Bio will make it easier and faster for biopharmaceutical customers to generate data for more precise clinical trials."
"Fulgent Genetics' full scope, end-to-end service and comprehensive genomic portfolio is the perfect one-stop shop for pharma customers," said Todd Druley, M.D., Ph.D., Chief Medical Officer of Mission Bio. "While some pharma customers can and still do partner with us through our internal Pharma Assay Development (PAD) services, this agreement provides another option for them to leverage the single-cell multi-omics expertise and full suite of solutions offered by Fulgent Genetics, particularly as they complete the validation of the Tapestri Platform in their CAP CLIA lab in the near future."
Customers today have access to the Tapestri Platform and its full breadth of single-cell multi-omics and scMRD assays through Fulgent Genetics' labs.
About Mission Bio
Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others.
The company's Tapestri Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution.
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MEDICAL, INDUSTRY OUTLOOK
PRNewswire | May 17, 2023
REGENXBIO Inc. announced that preclinical research in Mucopolysaccharidosis type IVA also known as Morquio syndrome, was selected for inclusion in the Foundation for the National Institutes of Health Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), clinical trial portfolio. The Consortium brings together partners such as NIH and FDA, as well as partners from private and non-profit sectors.
Sponsored by Nemours Children's Hospital, MPS IVA is one of eight programs selected as part of AMP® BGTC's first clinical portfolio to help accelerate the development of bespoke gene therapies, with the goal of streamlining the regulatory approval process. MPS IVA is a metabolic condition that primarily affects the skeleton, and is estimated to impact 1 in 200,000 to 300,000 individuals.
"I am proud of our scientists who led this important clinical research work for REGENXBIO. Our mission to deliver the curative potential of gene therapy reflects not only patients that can be impacted by our clinical pipeline, but also all patients who should have the opportunity to be positively impacted by gene therapy," said Kenneth T. Mills, President and CEO REGENXBIO. "As a partner of the BGTC, REGENXBIO is pleased to see this program advancing important science into the clinic."
Criteria for selection included the adequacy of the gene for insertion into an adeno-associated virus (AAV) vector, sufficient proof of concept and natural history data, the existence of an established disease model, a lack of available treatment and an overall readiness for entering into a clinical trial. REGENXBIO will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases. Use of the NAV® Technology Platform will help address the goal of making gene therapy more accessible by creating a platform approach with standardized processes to deliver novel therapies for many different genetic disorders. These programs together further validate the versatility of NAV® vectors and will provide additional data that collectively drive the advancement of the AAV gene therapy field.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
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