Biogen, DDN, Qlucore, And More: News From December 2018

December 19, 2018 | December featured exciting new, products, and partnerships from around the bio-IT community from innovating companies, organizations, and universities, including Biogen, DDN, Qlucore, and more.

Biogen and Ionis announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB067 (IONIS-SOD1_RX), an investigational treatment for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations. ALS with SOD1 mutations is a subtype of familial ALS and accounts for approximately 2% of all ALS cases. As a part of the option exercise, Biogen made a one-time $35 million payment to Ionis. Future payments may include potential post-licensing milestone payments of up to $55 million and royalties in the low to mid-teen percentages on annual worldwide net sales. Biogen will be solely responsible for the costs and expenses related to the development, manufacturing and commercialization of BIIB067 following the option exercise. The decision to exercise the option was based on a positive interim analysis of a randomized, placebo controlled single- and multiple-ascending dose Phase 1 study (n=70) that achieved proof-of-biology and proof-of-concept for BIIB067. At the highest dose tested (n=10), treatment with BIIB067 over a three month period resulted in a statistically significant lowering of SOD1 protein levels in the cerebrospinal fluid (p=0.002) and a numerical trend towards slowing of clinical decline as measured by the ALS Functional Rating Scale-Revised, both compared to placebo (n=12). The safety and tolerability profile in this analysis supports continued development of BIIB067 in ALS. Based on these results, Biogen plans to advance BIIB067 to a pivotal clinical study. “Biogen and Ionis have a shared goal of bringing innovative therapies to those living with severe neurological diseases where there is significant unmet medical need,” Michael Ehlers, executive vice president, research and development at Biogen, said in a press release. “The progress made to date in the BIIB067 clinical program is a positive step forward. We are committed to our goal of bringing forward a therapy for patients with ALS with SOD1 mutations, who currently have limited or no treatment options.” Press release

DataDirect Networks (DDN) announced a breakthrough in microscopy workflows that enables real-time analysis for improved resolution of images. Comprehensive testing of DDN AI200 shared parallel storage appliance, a NVIDIA DGX-1 system and the Microvolution advanced processing software demonstrates significant experimental and economic benefits, delivering 1600-fold better yield compared with traditional microscopy workflows, and 8X faster time to insight than using cloud-based infrastructure. Lattice LightSheet microscopes and other high-data-rate instruments are revolutionizing biological research in areas such as neuroscience, developmental biology, cancer research, and biomedical engineering. To enable the incredible resolution of these high-end instruments, researchers employ sophisticated deconvolution algorithms to increase signal-to-noise and to remove blurring in these 3D images. However, researchers have historically faced efficiency challenges when storing, managing and processing the large amounts of data generated, and the sheer amount of processing required limited these techniques to a post-processing step rather than being applicable in real-time during imaging capture. “Real-time analysis and imaging provide unprecedented flexibility for experiments,” George Vacek, global director for life sciences, healthcare and machine learning at DDN, said in a press release. “The platform critically enables the increased temporal resolution that these instruments offer. Efficient detection allows for imaging of dim and fast phenomenon that were not previously possible to resolve.” Press release

Qlucore has announced Qlucore Omics Explorer 3.5 of its data analysis software. Key features of the new version include the addition of Templates and an extensive upgrade of plot configuration and options giving improved visualizations. Templates, based on Python, is a new functionality in the program that makes it possible to create a script of commands that are executed by Qlucore Omics Explorer (QOE). The program provides a number of templates for basic functionality and demonstration purposes and allows the user to write new templates, with a user interface. The addition of Templates will add great value to organizations since they can create standardized analysis templates for standard analysis. The integration with Python also opens a whole new world of possibilities for customization. The addition of Templates lets the user run exactly the same analysis many times, integrate QOE in existing work-flows and expand them, configure a favorite analysis for a quick start, get started with pre-configured templates for standard analysis, and use templates to explore the program and get a feeling for functionality. Press release

amfAR, The Foundation for AIDS Research, has awarded $800,000 in new funding to researchers developing an ambitious gene therapy-based approach to curing HIV. The award launches a critical new phase in a study initiated in 2017 with $2.3 million in grants awarded to seven teams of researchers. Six of the teams will move forward with the next phase of the project. “This is an ambitious and complex project with very exciting potential,” said amfAR Chief Executive Officer Kevin Robert Frost, in a press release. “It’s a carefully constructed strategy that involves some of the most talented and innovative scientists in the field. We think it holds great promise for developing the scientific basis of a cure by the end of 2020, which is the aim of our Countdown to a Cure for AIDS initiative.” The researchers will test an approach that combines CAR stem cells that secrete broadly neutralizing antibodies, together with an enzyme (Brec1) that targets HIV DNA in the cell it has infected (while leaving other DNA intact), and long-term secretion of a broadly neutralizing antibody from the liver. The goal is to 1) induce CAR stem cells to kill reservoir cells; 2) to express two different antibodies to neutralize virions (virus that exists outside of cells) in the blood and tissues, and; 3) to use Brec1 to remove the provirus (virus that has been integrated into a cell’s DNA) from infected cells. The investigators are: Hildegard Büning (co-principal investigator, molecular biologist, Hannover Medical School, Hannover, Germany), who was recently named president of the European Society of Gene and Cell Therapy; Keith Jerome (co-principal investigator, virologist, University of Washington, Seattle); Hans-Peter Kiem (transplantation biologist, Fred Hutchinson Cancer Research Center, Seattle); Scott Kitchen (molecular biologist, UCLA, Los Angeles); Drew Weissman (Immunologist, University of Pennsylvania, Philadelphia); and Richard Wyatt (immunologist, The Scripps Research Institute, La Jolla). Press release

EpiVario announced it has raised $445K in a seed round to support the development of a new treatment for a wide range of memory-related psychiatric disorders at the source, including Post-Traumatic Stress Disorder (PTSD), Alzheimer's, and alcohol and drug addiction. Founded by University of Pennsylvania epigeneticists, the company's novel approach works by targeting the non-essential gene, ACSS2, to prevent the process of negative memory formation or reconsolidation, a key contributor to PTSD and other memory-related conditions. The funds will be used to hire additional research staff and support the first phase of preclinical research milestones. EpiVario's proof-of-concept has already been established in animals. Research on ACSS2 knockdown mice, conducted by EpiVario co-founders Philipp Mews was published in Nature in June of 2017. EpiVario plans to begin its Phase 1 study in 2020. Press release

Labstep announced an institute-wide trial of their cloud-based technology at Medical Research Council Unit The Gambia at the London School of Hygiene & Tropical Medicine. The trial of Labstep.com at MRC Unit The Gambia at LSHTM is the first institute-wide assessment of the cloud-based, mobile-optimised web app that connects groups of researchers who can share version-controlled experimental procedures. Labstep allows researchers to run experimental procedures at the workbench, capturing real-time data on scientific processes, and uses this to build a record of research activity. The lack of legacy infrastructure provides a unique opportunity for local researchers to leap directly towards use of cloud data storage and synchronisation over mobile networks, bypassing WiFi or ethernet source complications. Jake Schofield, Labstep co-founder and CEO, said in a press release: “We built Labstep with the pain points of the individual researcher in mind. With Labstep, we are streamlining scientific data recording and subsequent processing, supporting the individual as well as answering the global call for enhanced research documentation. Institute-wide use of Labstep will be a huge improvement to the researcher’s day-to-day work and brilliantly supports collaboration within and between research labs.” Article

New England Biolabs (NEB) announced the first automated, web-based application, GlycanAnalyzer, which enables routine use of enzymatic digestions to improve N-glycan analyses. This software not only saves precious times for glycobiologists who are more attuned to traditional methods, but also provides the most accurate interpretation of glycan enzymatic digestion data on the market. N-glycan structure plays a critical role in numerous physiological and pathological processes, making N-glycan profiling imperative to biotherapeutic quality and development. Yet, until now, the only way to characterize them has been through manual analysis, which can take anywhere from several days to several weeks to complete. The new GlycanAnalyzer software pattern matches UPLC-FLR-MS peak shifts of released N-glycans following exoglycosidase digestions and automates structure assignments, which no other commercial offerings can do at the moment. It then presents this data visually and links to published reference databases. NEB performed a quality test of GlycanAnalyzer using a monoclonal antibody and four glycan standards and found that 100% of peaks were correctly assigned using the technology, compared to only 81% with the leading competitor, and, using GlycanAnalyzer’s “Assign Top 5” option (which returns the top 5 peak movements of the sample), all assignments were returned in only 20 minutes.