RESEARCH, PHARMA TECH
Ionis Pharmaceuticals, Inc. | March 10, 2023
Ionis Pharmaceuticals, a leading RNA-targeted therapy firm, recently announced that the USFDA has accepted for review a new drug application (NDA) for eplontersen. Eplontersen is an investigational antisense medicine designed to reduce the production of transthyretin (TTR) protein and treat hereditary and non-hereditary forms of ATTR amyloidosis (ATTR).
The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA has declared that no review concerns were detected and made no new data requests. The FDA also stated that it does not intend to conduct a meeting of the advisory committee to discuss the application.
ATTR amyloidosis is a systemic disease caused by the accumulation of TTR protein in organs, leading to progressive organ damage and eventually death. Patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) experience ongoing debilitating nerve damage throughout their bodies, resulting in the progressive loss of motor function.
The NDA for eplontersen is based on results from the global Phase 3 NEURO-TTRansform study showcased at the International Symposium on Amyloidosis (ISA). In the study, eplontersen showcased a statistically significant and clinically meaningful change relative to the baseline for its co-primary and critical secondary endpoints compared to the external placebo group. In addition, Eplontersen also demonstrated a favorable safety and tolerability profile.
Ionis and AstraZeneca announced a strategic partnership to develop and commercialize eplontersen in December 2021. Eplontersen is being developed and commercialized jointly by both businesses in the United States and will be created and commercialized by AstraZeneca throughout the rest of the globe, except Latin America.
The FDA granted eplontersen Orphan Drug Designation in the United States in January 2022. Eplontersen is also being studied in the worldwide Phase 3 CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and deadly disorder leading to progressive heart failure and death within 3 to 5 years after disease start.
About Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. is a leading biopharmaceutical company headquartered in Carlsbad, California. Its unique antisense technology platform allows them to develop RNA-targeted therapies that modify the production of disease-causing proteins, leading to the development of treatments for rare genetic disorders, cardiovascular diseases, and neurodegenerative diseases. The company's most significant achievement is developing and commercializing Spinraza, the first approved treatment for spinal muscular atrophy. Ionis Pharmaceuticals has also developed drugs for other rare diseases, including transthyretin amyloidosis, Huntington's disease, and myotonic dystrophy type 1.
Businesswire | June 02, 2023
REALM IDx announced the launch of REALM Pharma Services, an end-to-end discovery, translational research, and clinical trial service for biotechnology and pharmaceutical partners. Through a strategic integration, REALM Pharma Services brings together the research arms of subsidiaries, Ambry Genetics and Invicro, to offer cutting-edge genomics, traditional and molecular pathology, and advanced imaging and spatial biology. Combined with machine learning and artificial intelligence (AI) from REALM’s informatics organization, the company believes this end-to-end service can help reveal new insights and develop multi-dimensional models for drug discovery and validation.
“We are excited to offer a comprehensive multiomic and multimodal program that has been tailored to support the needs of our biopharma partners,” said Aaron Elliott, PhD, CEO of REALM IDx. “We believe the integrated approach offered through REALM Pharma Services is a transformative shift in research and will bring new, data-driven insights to precision medicine.”
Use of advanced omics, including single-cell omics, is increasingly being incorporated by biopharma into discovery and research efforts, particularly in the field of cancer. This approach can support improved understanding of biological complexity in immuno-oncology and a system-wide path to drug efficacy. However, a significant challenge has been the lack of a consolidated infrastructure to not only support analytical testing and data collection, but also computational analysis of unique data sets to find novel associations for translational insights.
“In supporting our biopharma partners, we see ourselves as a powerful connecting force,” said Jonathan Usuaka, PhD, Chief Strategy and Informatics Officer for REALM IDx. “Along with world-class genomics, pathology, spatial imaging and analysis services, we support real-world data and advanced analytics to inform drug development at every stage.”
The company will introduce REALM Pharma Services while exhibiting at the upcoming American Society of Clinical Oncology annual conference in Chicago, June 3 – 5. While at the conference, the company will also share details of a new comprehensive genomic profiling test (CGP) for cancer developed in collaboration with its partners in Japan and set to launch this summer. Additional information about REALM Pharma Services may be found on REALM’s website.
About REALM IDx
REALM IDx, Inc. is a health care company pioneering in the field of Integrated Diagnostics (IDx), an advanced field of clinical science that brings together laboratory medicine, radiology, pathology and sophisticated artificial intelligence to derive actionable insights to predict, diagnose and treat disease. Powered by proprietary software platforms, industry-leading genomics technology from Ambry Genetics Corporation and radiology and pathology services from Invicro, LLC, the company is equipped to collect, analyze and report on multi-modal precision diagnostic data sets.
BUSINESS INSIGHTS, PHARMA TECH
Businesswire | April 26, 2023
Global healthcare and business consulting services provider, Terebellum, has chosen to adopt the name of its global parent, AscellaHealth, to reflect the scale of its operations and breadth of expertise.
The business specialises in providing end-to-end solutions to pharmaceutical manufacturers and other industry stakeholders to streamline product commercialisation, improve medication access, reduce costs and enhance patient outcomes for specialty and rare disease patients.
Based in Dublin, Ireland and Manchester, UK, former Terebellum provides pre-commercialisation and market access support, supply chain logistics, distribution and fulfilment and unique pharmaceutical financial services to UK and European pharmaceutical manufacturers and healthcare organisations.
Adopting the brand name of its parent company, AscellaHealth, will enable the specialty pharma expert to demonstrate its full range of expertise, international insight and distinctiveness of offer. Upon Terebellum’s transition to the AscellaHealth brand identity in its market-facing activities, the company will continue to maintain current business operations, client relationships and dedication to patient-centric care.
Craig Caceci, Managing Director at AscellaHealth EU/UK, said: “It’s our strong belief that to optimise outcomes in specialty healthcare a specialised approach is needed, due to the specific challenges associated with rare conditions.
“The AscellaHealth name provides an opportunity to highlight how we are perfectly positioned to offer uniquely tailored solutions across the globe to ensure streamlined medication access and the best possible treatment journey for rare and orphan disease patients.”
In a recent customer survey carried out by AscellaHealth, 100 percent of respondents agreed that the organisation presents unique value in the market and is uniquely qualified to address their needs.
Craig adds: “Supporting patients, life science manufacturers, payers and providers, we bring expertise and care to every requirement to develop and implement novel approaches that ensure patients with rare diseases can get the treatment they need, when they need it most.
“Our new brand tagline, ‘a rare and special perspective’, captures our dedication to applying knowledge and expertise to improve health outcomes of rare disease and specialty patients, perfectly.”
AscellaHealth, co-founded by Dea Belazi, President & CEO and Bill Oldham, Chairman & CFO in 2013, employs nearly 200 employees worldwide to provide integrated end-to-end solutions that span the entire pharmaceutical lifecycle and beyond, supporting all segments of the specialty pharma industry.
Former Terebellum joins US-based pharmacy distribution and patient-management organisation, Optime Care, in adopting the name of parent AscellaHealth, to form one globally-recognised brand for specialty pharma expertise.
About AscellaHealth LLC
AscellaHealth, a global Healthcare & Specialty Pharmacy solutions organisation, serving patients, life sciences manufacturers, payers, and providers, offers a comprehensive portfolio of uniquely tailored, tech-enabled services supporting complex, chronic conditions or rare diseases that require specialty medications and/or cell and gene therapies. A recipient of numerous industry awards for innovation, and a NASP Strategic Channel Partner of the Year award winner, AscellaHealth’s best-in-class, patient-centric approach is built upon proprietary technology processes for novel programs and services to support the launch of specialty medications and proactively address multiple challenges, optimise clinical health outcomes, and improve quality of life for this patient population. AscellaHealth brings a rare and special perspective to all stakeholders.