RESEARCH, PHARMA TECH
OM1 | February 06, 2023
OM1, a leading real-world data, results, and technology firm focused on chronic diseases, recently announced the release of its Reasons for Discontinuation (RfD) Analytic Reports. Utilizing the OM1 Real-World Data CloudTM, Immunology Real-World Data Network, and proprietary artificial intelligence (AI) technology, the RfD reports provide difficult-to-obtain, extremely valuable discontinuation trends for cDMARDs, bDMARDs, and tsDMARDs in primary dermatologic and rheumatologic conditions.
Each year, millions of Americans cease their prescriptions prematurely, costing the pharmaceutical industry in the United States an estimated $250 billion in lost income and can affect the health of these patients. Therefore, it is crucial for pharmaceutical businesses to comprehend patient discontinuation patterns so they may act on information regarding tolerability, costs, and emerging contraindications to enhance the patient experience. Prior to this point, it was extremely difficult to extract these insights due to the fact that crucial information was not reflected in claims or any other sort of structured data but was only available in clinical notes.
OM1 is elevating the clinical narrative into actionable insights that pharmaceutical companies can rely on to comprehend patient adherence and clinician prescribing behavior, thanks to its data cloud built on billions of data points from over 300 million patients and unique AI and modeling capabilities. The analyses, which are conducted across multiple strata, such as demographic groups, patient characteristics, medical histories, comorbidities and care pathways, extract insights from the extensive clinical data sets available to OM1 and notes from prescribing clinicians dating back to 2013.
Currently, accessible RfD reports include psoriatic arthritis, rheumatoid arthritis, ankylosing spondylitis, psoriasis, and atopic dermatitis, with more to come, including schizophrenia, depression, and bipolar illness.
Founded in 2015, OM1 is a renowned health outcomes, and registries firm focused on treatment outcome measurement, comparison, and prediction. OM1 created the first intelligent data cloud for healthcare, leveraging big data, standardized outcomes measurement, and artificial intelligence technologies to provide more precise information and improve decision-making for stakeholders across the healthcare ecosystem. The company is reimagining real-world data and evidence in immunology, cardiometabolic, mental health and neurosciences, respiratory and ENT specialty areas by establishing vast electronically connected networks of clinicians and health data.
RESEARCH, PHARMA TECH
Asklepios BioPharmaceutical, Inc. (AskBio) | March 01, 2023
Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, recently announced that the European Commission (EC) has granted orphan drug designation to AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is an investigational recombinant adeno-associated virus (AAV) based gene therapy that is being developed as a one-time intravenous infusion for patients with LGMD type 2I/R9.
The disease affects 4.5 people per million worldwide, with more than 5,000 people affected in the EU and the US. The EC decision was received through AskBio's EU-based subsidiary BrainVectis, following a positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP)
AB-1003 is currently being investigated in a Phase 1/2 multicenter study in the US to evaluate its tolerability, safety, and efficacy in adult subjects with genotypically confirmed LGMD2I/R9.
The EC for medicinal products grants orphan drug designation developed to treat a life-threatening disease that affects no more than five people in 10,000 in the EU, provided there is no other adequate treatment option or the medicine can bring significant value to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible reductions or exemptions in certain regulatory fees. Additionally, if the medicine is approved for marketing, this designation will provide ten years of marketing exclusivity.
Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause progressive weakening and wasting of the muscles in the arms and legs. The severity, features of LGMD and age of onset vary among the many subtypes of the condition and are often inconsistent, even within the same family. Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD caused by mutations in the FKRP gene. Signs and symptoms of LGMD2I/R9 often appear in late childhood and may include difficulty walking and running. The symptoms intensify with time, eventually leading to considerable disability, and afflicted persons typically require a wheelchair for movement roughly 23-26 years after beginning. Unfortunately, there is presently no treatment that slows the advancement of the disease, and management is based on the indications and symptoms that each individual exhibits.
About Asklepios BioPharmaceutical, Inc. (AskBio)
AskBio is a leading, fully integrated gene therapy company dedicated to developing innovative gene therapeutics and life-saving medicines. With operations worldwide, its pipeline includes gene therapy candidates for various diseases, including the central nervous system, neuromuscular, metabolic, and cardiovascular diseases. AskBio's global headquarters are located in RTP, NC, with European headquarters in Edinburgh. In addition, the company operates AAV research centers in Philadelphia, PA; Columbus, OH; and Paris, and modern clinical and commercial facilities in San Sebastian, Spain.
BUSINESS INSIGHTS, PHARMACY MARKET
VERO Biotech | February 03, 2023
On February 2, 2023, VERO Biotech Inc., a commercial-stage biotechnology company focused on neonatal intensive care and the acute care hospital community, announced that FDA has approved its innovative second-generation GENOSYL® Inhaled Nitric Oxide (iNO) delivery system for use in the operating room with rebreathing anesthesia. The second generation GENOSYL® DS is now the first and only iNO delivery device approved for use in both rebreathing and non-rebreathing anesthesia procedures, enhancing patient care, saving money for the hospital, and minimizing waste anesthetic gas pollution.
GENOSYL® DS is the first tankless inhaled nitric oxide delivery system. Nitric oxide, when inhaled, dilates the pulmonary blood vessels and can improve oxygenation in neonates suffering from hypoxic respiratory failure and pulmonary hypertension. GENOSYL® DS, unlike tank-based systems, generates and delivers iNO at the bedside via a small disposable cassette. This helps eliminate the need for hospitals to manage large, cumbersome tanks while also aiding in the simplification of clinical workflow.
The following are the expected advantages of the second generation GENOSYL® DS
Ability to employ rebreathing anesthesia: Rebreathing allows reduced gas flows, resulting in less usage of expensive anesthetic drugs, thereby reducing hospital costs; enhanced patient comfort (by preserving patient body temperature and moisture).
Smoother care process: seamless iNO delivery from the ICU to surgery and post-operative care leads to process and workflow enhancements for the healthcare organization, thus lowering the hospital's overall cost.
Set and forget: It allows anesthesiologists to continue using rebreathing anesthesia, thereby preventing potentially dangerous, time-consuming and cumbersome workarounds.
Reduced ecological consequences of anesthesia delivery: less anesthetic is released into the environment.
It is important to note that FDA has only approved the second generation GENOSYL® DS for use with rebreathing anesthesia in the surgical suite. The third generation GENOSYL® DS, which was recently approved, has not been tested with rebreathing anesthesia. VERO Biotech is currently conducting similar validations and expects to have data available in the first quarter of 2023.
About VERO Biotech
Headquartered in Atlanta, GA, VERO Biotech is an emerging biotechnology firm that focuses on designing, developing, and commercializing next-generation inhaled nitric oxide (NO) delivery systems to tackle unmet medical needs in patients with cardiopulmonary conditions. The company's mission is to improve patients' lives by pioneering innovative technologies for inhaled NO delivery in acute care hospitals and beyond, wherever nitric oxide treatment is required. By focusing on the science, development, and commercialization of its innovations, it aims to exceed customer expectations regarding safety, efficacy, and growth.