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ARCA biopharma Announces FDA Approval of IND Application for AB201 as a Potential Treatment for COVID-19

Dr. Bristow

Dr. Michael Bristow, President and CEO, ARCA biopharma, Inc.
Dr. Michael Bristow, President and CEO, ARCA biopharma, Inc.
Dr. Michael Bristow, President and CEO, ARCA biopharma, Inc.
  • Initiation of Phase 2b clinical trial anticipated in Q4 2020

  • Trial to enroll approximately 100 patients hospitalized with COVID-19

  • Topline data anticipated Q2 2021

WESTMINSTER, Colo., Oct. 07, 2020 (GLOBE NEWSWIRE) — ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically targeted therapies for cardiovascular diseases, today announced the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for AB201 (rNAPc2) as a potential treatment for patients hospitalized with COVID-19. ARCA anticipates initiating a Phase 2b/3 sequential clinical trial, ASPEN-COVID-19, of AB201 in approximately 100 patients hospitalized with COVID-19 in the fourth quarter of this year, with Phase 2b followed by a contiguous Phase 3 study that is dependent on Phase 2 results. The Company anticipates topline data from the trial in the second quarter of 2021.

The planned Phase 2b trial is anticipated to be a randomized comparison of two dose regimens of AB201 versus heparin prescribed per local standard of care. The clinical course of some patients with COVID-19 is complicated by a virus-triggered coagulopathy that includes thrombotic events and inflammatory processes, thought to be mediated in part by tissue factor production. AB201 is a potent tissue factor inhibitor anticoagulant with anti-inflammatory and antiviral properties. The primary endpoint of the trial will be change in D-dimer level from baseline to Day 8. D-dimer is a biomarker commonly used for assessing coagulation activation, which is elevated in approximately 50% of hospitalized COVID-19 patients and is directly associated with adverse clinical outcomes.   If Phase 2b indicates a favorable effect on D-dimer levels, following FDA review of the data and identification of the proposed Phase 3 AB201 dose, the Company anticipates that clinical investigative sites will begin enrolling in the planned Phase 3 clinical trial. The primary endpoint of Phase 3 will be clinical recovery as measured by the Adaptive COVID-19 Treatment Trial (ACTT-1) ordinal scale, with secondary endpoints that include D-dimer levels and the number of thrombotic events. Phase 3 will be event driven, with an estimated requirement of 450 patients. The Phase 2b and Phase 3 trials are described in a common protocol and use identical entry criteria and the same heparin regimen control.

Dr. Michael Bristow, ARCA’s President and Chief Executive Officer, who is also an American Heart Association (AHA) funded COVID-19 investigator, commented, “The ASPEN-COVID-19 trial will use the coagulopathy biomarker D-dimer to identify an optimal dose from AB201 regimens that are both within the therapeutic range as determined from Phase 2 trials investigating cardiovascular thrombosis prophylaxis. If successful, we anticipate using this dose in a planned Phase 3 trial to evaluate potential improvement in clinical outcomes. We believe that the combination of anticoagulant, anti-inflammatory and antiviral effects of AB201 may favorably impact clinical recovery of patients hospitalized with COVID-19.”

The trial is being managed in collaboration with the Colorado Prevention Center (CPC),

Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ

  • Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ

  • LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy

Diamond Pharma Services (“Diamond”), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (“GenSight”), including the preparation, authoring support, agency communication and submission of GenSight’s first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ®. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: “We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamond’s broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals – Glybera® and Yescarta®.”

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005339/en/

Contacts

Katie Odgaard
Zyme Communications
Tel: +44 7787 502 947
E-mail: katie.odgaard@zymecommunications.com

Source Article

Global Precision Medicine Market 2020-2030: Focus on Ecosystem, Technology, Application, and Competitive Landscape

The “Global Precision Medicine Market: Focus on Ecosystem, Technology, Application, Country Data (21 Countries), and Competitive Landscape – Analysis and Forecast, 2020-2030” report has been added to ResearchAndMarkets.com’s offering.

Global Precision Medicine Market to Reach $278.61 Billion by 2030

Precision medicine refers to the medicine developed as per an individual’s genetic profile. It provides guidance regarding the prevention, diagnosis, and treatment of diseases. The segmentation of the population is done depending on the genome structure of the individuals and their compatibility with a specific drug molecule.

In the precision medicine market, the application of molecular biology is to study the cause of a patient’s disease at the molecular level, so that target-based therapies or individualized therapies can be applied to cure the patient’s health-related problems.

This industry is gaining traction due to the increasing awareness about healthcare among individuals, integration of smart devices such as smartphones and tablets into healthcare, and increasing collaborations and agreements of IT firms with the diagnostics and biopharmaceutical companies for the development of precision diagnostic tools.

Within the research report, the market is segmented on the basis of product type, ecosystem application, and region, which highlight value propositions and business models useful for industry leaders and stakeholders. The research also comprises country-level analysis, go-to-market strategies of leading players, future opportunities, among others, to detail the scope and provide 360-degree coverage of the domain.

Key Topics Covered:

1 Product Definition

2 Research Scope

3 Research Methodology

4 Global Precision Medicine Market Overview

4.1 Market Definition

4.2 Precision Medicine: A Frontier in the Genesis of Patient-centric Medicine

4.3 Precision Medicine: Remodeling the One-Size-Fits-All Theory to Individually Tailored Therapy

4.4 Initiatives and Programs

4.5 Precision Medicine: Enabling Technologies and Applications

4.5.1 Innovators

4.5.1.1 3D DNA Printing

4.5.1.1.1 Introduction

4.5.1.1.2 Role of 3D DNA Printing

4.5.1.2 RNA-Seq

4.5.1.2.1 Introduction

4.5.1.2.2 Role of RNA-Seq in Precision Medicine

4.5.1.2.3 Key Players

4.5.1.3 4D Molecular Imaging

4.5.1.3.1 Introduction

4.5.1.3.2 Role of 4D Molecular Imaging in Precision Medicine

4.5.1.3.3 Key Players

4.5.2 Early Adopters

4.5.2.1 CRISPR

4.5.2.1.1 Introduction

4.5.2.1.2 Role of CRISPR in Precision Medicine

4.5.2.1.3 Key Players

4.5.2.2 Blockchain

4.5.2.3 Imaging Informatics

4.5.3 Early Majority

4.5.3.1 Artificial Intelligence (AI)

4.5.3.2 Circulating Free DNA (cfDNA)

4.5.3.3 Big Data

4.5.3.4 Next-Generation Sequencing (NGS)

4.5.3.5 Health Informatics

4.5.3.6 Bioinformatics

4.5.4 Late Majority

4.5.4.1 Polymerase Chain Reactions (PCR)

4.5.4.2 Microarray

4.6 COVID-19 Impact on the Global Precision Medicine Market

5 Market Dynamics

5.1 Overview

5.2 Market Drivers

5.2.1 Advancement of Sequencing Technologies

5.2.2 Rising Prevalence of Chronic Diseases

5.2.3 Growing Demand for Preventive Care

5.2.4 Shifting the Significance in Medicine, from Reaction to Prevention

5.2.5 Reducing Adverse Drug Reactions Through Pharmacogenomics Test

5.2.6 Potential to Reduce the Overall Healthcare Cost Across the Globe

5.3 Market Restraints

5.3.1 Unified Framework for Data Integration

5.3.2 Limited Knowledge about Molecular Mechanism/ Interaction

5.3.3 Lack of Robust Reimbursement Landscape

5.3.4 Regulatory Hurdles

5.4 Market Opportunities

5.4.1 Targeted Gene Therapy

5.4.2 Expansion into the Emerging Markets

5.4.3 Collaboration and Partnerships Across Value Chain to Accelerate the Market Entry

6 Industry Insights

Global Precision Medicine Market 2020 with (COVID-19) Impact Analysis, Product Type, Application, Key Manufacturers, Regions and Forecast to 2025

The MarketWatch News Department was not involved in the creation of this content.

Sep 29, 2020 (CDN Newswire via Comtex) —
A wide-ranging analysis report titled Global Precision Medicine Market 2020 by Company, Regions, Type and Application, Forecast to 2025 provides a brief overview of the market covering the scope, size, disposition, and growth of the industry. The report presents an estimate of the current market scenario and data related to the competitive landscape of the industry. It delivers five-year industry forecasts, growth rates, and an analysis of the industry key players and their market shares. The report shows information regards to several regions that have successfully established its position in the global Precision Medicine market. The geographical and competitive dynamics of this global market will help you get a comprehensive picture of the market.

An industrial chain, market measurements regarding revenue, sales, value, capacity, regional market examination, section insightful information, and the market forecast is provided. The report gives global Precision Medicine market share analysis, as well as analyzes market position, market share, and segmented revenue. Further company and financial overview, product portfolio, new project launched, recent development analysis are the parameters added with this report.

NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

DOWNLOAD FREE SAMPLE REPORT:https://www.marketquest.biz/sample-request/8778

The report investigates the development, trends, and new entrants in the sector, with elaborate profiles of the leading companies operating in the market, including: Johnson & Johnson, Novartis, Illumina, IBM, Thermo Fisher Scientific, GE Healthcare, Almac Group, Laboratory Corporation of America Holdings, Roche, Abbott Laboratories, Randox Laboratories, Intel Corporation, Healthcore, Qiagen, Biomrieux Sa, Cepheid

In market segmentation by types, the report covers: Diagnostics, Therapies

In market segmentation by applications, the report covers the following uses: Oncology, Neurosciences, Immunology, Respiratory, Others

The report provides a 5-year forecast (2020-2025) assessed based on how the global Precision Medicine market is predicted to grow in major regions like: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina), MENA (Saudi Arabia, UAE, Turkey and South Africa)

Industry Size & Forecast: The report offers estimations on the global Precision Medicine market industry size on the basis of value and volume are provided in this part of the report. This report offers deep insights into the prevailing and upcoming market trends. Then it has examined the high-growth segments including product type, application, and end-users, taking into account their CAGR, share, and size. Market developments and future opportunities estimated to emerge in the market industry are looked into in this study. The forecasts are provided taking into consideration product, application, and regional segments of the market.

ACCESS FULL REPORT:https://www.marketquest.biz/report/8778/global-precision-medicine-market-2020-by-company-regions-type-and-application-forecast-to-2025

A Peek At Over the Highlights of the Report:

  • The study provides a synopsis