Marinus Pharmaceuticals Earns Rare Pediatric Disease Designation from FDA for Ganaxolone Treating CDKL5 Deficiency Disorder (CDD)

Marinus Pharmaceuticals | July 31, 2020

Marinus Pharmaceuticals, Inc., a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.

“We are pleased that ganaxolone has received Rare Pediatric Disease Designation from the FDA and are enthusiastic that the timing of this news aligns with our continued expectation to report top line data this quarter from our pivotal Phase 3 Marigold Study evaluating oral ganaxolone in patients with CDD,” said Scott Braunstein, M.D., Chief Executive Officer at Marinus. “This designation for ganaxolone for CDD underscores the significant unmet medical need for children and young adults with this serious and rare genetic disease, which causes early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Our goal is to advance a pipeline of programs that can treat rare seizure disorders, including CDD, tuberous sclerosis complex and PCDH19-related epilepsy, and bring much-needed medicines to patients as soon as possible.”

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The Complete List and Analysis of the 10 Best Pharmaceutical Companies in 2018. 7 criteria have been considered including revenue, growth, R&D investments, and others.

Spotlight

The Complete List and Analysis of the 10 Best Pharmaceutical Companies in 2018. 7 criteria have been considered including revenue, growth, R&D investments, and others.

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BUSINESS INSIGHTS

Ascential Digital Commerce and GSK Consumer Healthcare Announce Launch of Ascential Digital Commerce Connect at Cannes Lions

Ascential Digital Commerce and GSK Consumer Healthcare | June 15, 2022

Ascential Digital Commerce, whose technology driven products power brands to win in digital commerce, and GSK Consumer Healthcare, a science-led healthcare company, will join forces on June 20th during the Cannes Lions International Festival of Creativity to launch Ascential Digital Commerce Connect, a first-of-its-kind, data-rich, digital commerce platform that provides C-suite executives with visibility of brand sales and performance from multiple data sources. Ascential Digital Commerce Connect improves reaction time to market dynamics, increasing a brand's performance in the rapidly growing eCommerce retail market. Ascential Digital Commerce and GSK Consumer Healthcare partnered together to develop Ascential Digital Commerce Connect to provide visibility and clarity of GSK Consumer Healthcare's eCommerce brands performance, improve reaction time to market dynamics and position the brand for further growth. GSK Consumer Healthcare anticipates eCommerce growth to reach the mid-teens percentage of the business by 2025, thereby making eCommerce a priority channel to drive overall sales. According to Nasdaq research, 95% of all purchases by 2040 will be conducted via eCommerce and a 2021 IDC study revealed that the digital commerce applications market will see double-digit growth through 2025. “Visibility of data is becoming increasingly vital to businesses every day. Especially as eCommerce has become a core business capability for businesses around the globe, managing data streams for those businesses has become increasingly challenging.” Filippo Battaini, Research Manager at IDC Retail Insights The Ascential Digital Commerce Connect web-based platform captures, analyzes, visualizes, and displays critical marketing data about each brand and competitor to monitor key activities and answer marketers' key questions when it comes to Understanding the current brand/sales performance in the eCommerce marketplace – standalone vs. key competitors and owned channels vs. major online retailers like Amazon and Alibaba Dissecting what brand health looks like regionally throughout EMEA, US/North America, Asia-Pacific, and Latin America Navigating potential challenges and outages around supply chain, research and development, disruptive markets, and other key metrics. More accurately calculating marketing spending as a percentage of gross revenue During Cannes Lions 2022, Ascential Digital Commerce and GSK Consumer Healthcare will host a panel discussion to discuss the catalyst for developing Ascential Digital Commerce Connect and how GSK Consumer Healthcare currently leverages the platform for brand success. Product demos will also be available to showcase the platform. "The next five years represent a tipping point in the retailing industry," says Duncan Painter, CEO at Ascential. "Retailers and brands with the deepest, most recent insights into the digital performance of their products and services will gain an informed, competitive edge. The Connect platform turns data into actionable insights, and GSK Consumer Healthcare is on a strong path to further realize eCommerce growth." "Healthcare today is driven by the digital economy, whether consumers and patients are searching for healthcare information and services online, logging into exercise classes, ordering health and beauty products from their favorite online retailers, or managing prescription medications online," says Taryn Marella, Global Head of Digital Commerce, GSK Consumer Healthcare. "Ascential Digital Commerce Connect delivers actionable insights that help us monitor the health of our business and identify growth opportunities, update messaging and enhance creativity in the digital environment." About Ascential Digital Commerce Ascential Digital Commerce powers the global eCommerce ecosystem. Our platform allows brands, businesses and their agency partners to connect to consumers in every touchpoint of their journey to purchase goods and products. We transform data into actionable insights, allowing brands to build their competitive edge and drive growth with better, faster decisions. We solve the most complex challenges that global brands and businesses face in the digital commerce landscape with technology and human expertise to drive sales growth on the leading digital eCommerce marketplaces globally. About Ascential Ascential delivers specialist information, analytics, and eCommerce optimization platforms to the world's leading consumer brands and their ecosystems. Our world-class businesses improve performance and solve problems for our customers by delivering immediately actionable information combined with visionary longer-term thinking across Digital Commerce, Product Design, and Marketing. We also serve customers across Retail & Financial Services. With over 2,800 employees across five continents, we combine local expertise with a global footprint for clients in over 120 countries. Ascential is listed on the London Stock Exchange. About GSK Consumer Healthcare GSK’s Consumer Healthcare business is on track to become an independent company through a proposed demerger from GSK, expected in July 2022. The new business, to be named Haleon, is expected to be a world-leader in consumer healthcare. Led by its purpose to deliver better everyday health with humanity, it will be strongly positioned to play a vital role in a growing sector that is more relevant than ever. Haleon will have a world-class portfolio of category-leading global brands, including Sensodyne, Voltaren, Panadol and Centrum, brands that are trusted by healthcare professionals, customers and people worldwide to improve the health and wellbeing of individuals and their communities.

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PHARMA TECH

Nucleai Announces a Partnership with Sirona Dx for AI-Driven Discovery of Novel Spatial Biomarkers in Solid Tumors

Nucleai and Sirona Dx | July 14, 2022

Nucleai, a leader in AI-powered spatial biology transforming precision medicine by unlocking the power of pathology data, and Sirona Dx, a leading provider of multiomic single-cell analytical services, today announced a collaboration to advance the discovery of novel spatial biomarkers in solid tumors. The partnership combines Nucleai’s powerful AI spatial analytics platform with Sirona Dx’s leading multiplex assay development expertise to identify novel spatial signatures in high-plex assays that predict response to therapy and inform treatment decisions. Through this strategic collaboration, Sirona Dx will provide Nucleai and its customers with a CRO partner that will enable the deployment of AI-based diagnostic algorithms to support biopharma clinical trials. Nucleai’s robust computational AI-powered spatial biology algorithm supports various image modalities and cancer indications, offering a highly customizable, deployable solution across the drug development ecosystem. These capabilities earned Nucleai the position of one of the top 10 spatial biology companies in 2022. “Multiplex imaging technologies have improved rapidly over the years; however, identifying actionable insights from all the data collected remains immensely challenging. This powerful synergy between Sirona Dx and Nucleai is very exciting as it will allow us to identify actionable spatial signatures that can predict response to immunotherapy.” Avi Veidman, Co-Founder & Chief Executive Officer of Nucleai Sirona Dx is recognized as the original pioneers of spatial biology, having launched the first spatial-omics CRO service to biopharma in 2018. Today, their technology-agnostic, spatial biology suite includes the leading multiplexed imaging platforms and combines deep technical expertise required to develop high-performance multiplexed assays as large as 50 markers. Sophisticated multiplexed tissue imaging capabilities contributed to their selection as a Top 10 CRO of 2021 by Medhealth Outlook. “We are delighted to announce a partnership with Nucleai,” said Andrew Brown, Ph.D. Chief Commercial Officer at Sirona Dx. “Nucleai’s AI-driven analytical capabilities are exceptional. Our combined expertise in multiplexed tissue assay development, data generation and state-of-the-art analysis will unlock new spatial insights and accelerate the spatial biology revolution in precision medicine.” About Nucleai Nucleai is an AI-powered spatial biology company with a mission to transform drug development and clinical treatment decisions by unlocking the power of pathology data. Nucleai provides pharmaceutical companies, contract research organizations, and diagnostics laboratories with a state-of-the-art AI platform to improve clinical trials and clinical decision-making. About Sirona Dx Sirona Dx is a technical, CLIA accredited CRO, founded in 2016 to accelerate the pace of immunotherapy and targeted therapy development. With a leading-edge menu of specialized high complexity, single-cell proteomics and genomics services, Sirona Dx supports exploratory biomarker programs and advanced precision medicine initiatives to deliver safer and more effective treatment solutions.

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BUSINESS INSIGHTS

DiMe Releases Toolkits to Improve Sensor Data Integration and Power Better, Faster Global Healthcare and Research

Digital Medicine Society | July 18, 2022

Today, the Digital Medicine Society released the Sensor Data Integrations Toolkits, four comprehensive toolkits to guide data producers, processors, and consumers to use the influx of data from the increased use of wearables and digital sensing products at scale. These resources are based on the Sensor Data Integrations project, comprised of healthcare leaders from Amazon Web Services Oracle, the Moffitt Cancer Center, Takeda, the US Department of Veterans Affairs and more. These toolkits will help realize the promise of sensor generated data to drive better decisions, faster, to improve healthcare delivery and research. The project team will conduct a demo of the toolkits during a live launch event on July 18 at 10:30 am ET, featuring Micky Tripathi, the National Coordinator for Health Information Technology at the US Department of Health and Human Services. The surge of data from sensor technologies is far outpacing the industry's ability to collect, store, analyze, protect, and use this data effectively for patient care and research. The number of US patients using remote patient monitoring devices is expected to surpass 70 million by 2025. Between 2022 and 2028, the global market for wearable technology is expected to grow around 18.5 percent, reaching $380.5 billion. The number of unique digital endpoints being used in industry sponsored trials of new medical products increased by over 950% between October 2019 and May 2022. The number of sponsors using these products in medical product development increased from 12 to 96 in the same timespan. And while the ability to discern high-quality sensor data suitable for clinical decision making is increasing rapidly, the ability to access these data is constrained by a current dependence on individual point solutions. "Sensor generated data, captured during people's daily lives, offer the opportunity to redefine how we measure health and disease. This opportunity powers the possibility of using high quality, high resolution flows of data to reimagine our approach to healthcare and research, leveraging more complete information to improve individual clinical decisions, decisions about the effectiveness of new medical products, and broader policy and public health decisions. DiMe's new Sensor Data Integrations Toolkits provide action-oriented resources to help data producers, processors, and consumers come together to create a sensor data ecosystem suitable for scale." DiMe CEO Jennifer Goldsack AWS has contributed customer feedback and technical expertise to ensure the toolkit meets the needs of today's modern healthcare system. "Across every industry, we see companies trying to accelerate their path to the cloud," said Lita Sands, Head of Solutions Life Sciences at AWS. "Timelines are getting quicker while the data collected is increasing exponentially – there is a clear and significant need for how to effectively and securely collect and use this information at scale. DiMe's new toolkits are a lifeline to organizations working with sensor data. They offer a comprehensive starting point for data producers, processors, and consumers to help build an integrated pipeline to support better and faster decision making." The development of these toolkits builds on DiMe's previous pre-competitive initiatives, such as The Playbook, the essential guide for developing and deploying digital clinical measures to advance patient care, clinical research, and public health. The Sensor Data Integrations Toolkits go further to ensure that high quality sensor data can be used at scale to improve patient care and speed efficient medical product development. The DiMe community is currently working on additional projects related to taking digitally generated data to scale and will be releasing additional resources over the coming months. DiMe is not only committed to creating and disseminating new digital health approaches and tools, but also sharing user experiences with the broader community. We encourage Sensor Data Integrations Toolkits users to contribute to Dime's "Resources in Action" case study hub by sharing how you are using resources to further the safe, effective, equitable, and ethical use of digital medicine to redefine healthcare and improve human health. The leading organizations from across the global healthcare, research, and digital health innovation sectors that collaborated with DiMe to create these open-access resources are AWS, Elevance Health, Evidation, US Food and Drug Administration (FDA), Human First, Institute of Electrical and Electronics Engineers, Medable, Moffitt Cancer Center, Open mHealth, Oracle, Savvy, Takeda, and US Department of Veterans Affairs (VA). About the Digital Medicine Society DiMe is a global non-profit and the professional home for all members of the digital medicine community. Together, we tackle the toughest digital medicine challenges, develop clinical-quality resources on a technology timeline, and deliver these actionable resources to the field via open-source channels and educational programs. Join us to advance the ethical, effective, equitable, and safe use of digital medicine to redefine healthcare and improve lives.

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