Primary Hyperoxaluria - Pipeline Review, H1 2019

Global Markets Direct’s, ‘Primary Hyperoxaluria - Pipeline Review, H1 2019’, provides an overview of the Primary Hyperoxaluria pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Primary Hyperoxaluria, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Primary Hyperoxaluria and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape of Primary Hyperoxaluria

- The report reviews pipeline therapeutics for Primary Hyperoxaluria by companies and universities/research institutes based on information derived from company and industry-specific sources

- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

- The report reviews key players involved Primary Hyperoxaluria therapeutics and enlists all their major and minor projects

- The report assesses Primary Hyperoxaluria therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

- The report summarizes all the dormant and discontinued pipeline projects

- The report reviews latest news related to pipeline therapeutics for Primary Hyperoxaluria

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Primary Hyperoxaluria

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Primary Hyperoxaluria pipeline depth and focus of Indication therapeutics

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Allena Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

BridgeBio Pharma LLC

Dicerna Pharmaceuticals Inc

Intellia Therapeutics Inc

OxThera AB

Precision Biosciences Inc

Allena Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

BridgeBio Pharma LLC

Dicerna Pharmaceuticals Inc

Intellia Therapeutics Inc

OxThera AB

Precision Biosciences Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Primary Hyperoxaluria – Overview

Primary Hyperoxaluria – Therapeutics Development

Pipeline Overview

Pipeline ...

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Primary Hyperoxaluria – Overview

Primary Hyperoxaluria – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Products under Development by Companies

Primary Hyperoxaluria – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Primary Hyperoxaluria – Companies Involved in Therapeutics Development

Allena Pharmaceuticals Inc

Alnylam Pharmaceuticals Inc

BridgeBio Pharma LLC

Dicerna Pharmaceuticals Inc

Intellia Therapeutics Inc

OxThera AB

Precision Biosciences Inc

Primary Hyperoxaluria – Drug Profiles

BBP-711 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DCR-PHXC – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Inhibit HAO1 for Primary Hyperaxuluria – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Inhibit HAO1 for Primary Hyperoxaluria Type I – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

lumasiran – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Oxabact – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

reloxaliase – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Primary Hyperoxaluria – Dormant Projects

Primary Hyperoxaluria – Discontinued Products

Primary Hyperoxaluria – Product Development Milestones

Featured News & Press Releases

May 02, 2019: Dicerna submits updated IND application for DCR-PHXC for treatment of primary hyperoxaluria (PH) for the PHYOX 2 pivotal trial

May 02, 2019: Dicerna submits updated IND application for DCR-PHXC for treatment of primary hyperoxaluria (PH) for the PHYOX 2 pivotal trial

Apr 16, 2019: Alnylam begins late-stage paediatric trial of lumasiran

Apr 15, 2019: Intellia Therapeutics announces Oral Presentation on Gene Therapy to inhibit HAO1 for Primary Hyperoxaluria Type I at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy

Mar 29, 2019: Dicerna announces the presentation of updated data demonstrating utility of its lead compound DCR-PHXC in treating primary hyperoxaluria type 1 (PH1) and type 2 (PH2)

Mar 29, 2019: Dicerna announces the presentation of updated data demonstrating utility of its lead compound DCR-PHXC in treating primary hyperoxaluria type 1 (PH1) and type 2 (PH2)

Nov 15, 2018: OxThera is being granted US patent for oxalobacter secretagogues

Oct 31, 2018: OxThera presents encouraging 52-week efficacy and safety data for Oxabact in primary hyperoxaluria type 1 (PH1) patients with ESRD

Oct 25, 2018: Dicerna Announces Late-Breaking Data Supporting Use of DCR-PHXC in Adults with Primary Hyperoxaluria Types 1 and 2 (PH1 and PH2)

Oct 25, 2018: Dicerna Announces Late-Breaking Data Supporting Use of DCR-PHXC in Adults with Primary Hyperoxaluria Types 1 and 2 (PH1 and PH2)

Oct 25, 2018: Alnylam expands Alnylam Act program to improve diagnosis of Primary Hyperoxaluria Type 1 and aligns with FDA on trial design for ILLUMINATE-B Phase 3 pediatric study of Lumasiran

Oct 08, 2018: Alnylam reports new positive Phase l/ll results from lumasiran trial

Oct 05, 2018: Dicerna Announces Late-Breaking Data from the PHYOX Phase 1 Clinical Trial of DCR-PHXC Will Be Presented at ASN Kidney Week 2018

Oct 05, 2018: Dicerna Announces Late-Breaking Data from the PHYOX Phase 1 Clinical Trial of DCR-PHXC Will Be Presented at ASN Kidney Week 2018

Sep 05, 2018: Dicerna announces proof of concept for DCR-PHXC in the treatment of primary hyperoxaluria

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Primary Hyperoxaluria, H1 2019

Number of Products under Development by Companies, H1 2019

Products under Development ...

List of Tables

Number of Products under Development for Primary Hyperoxaluria, H1 2019

Number of Products under Development by Companies, H1 2019

Products under Development by Companies, H1 2019

Number of Products by Stage and Target, H1 2019

Number of Products by Stage and Mechanism of Action, H1 2019

Number of Products by Stage and Route of Administration, H1 2019

Number of Products by Stage and Molecule Type, H1 2019

Primary Hyperoxaluria – Pipeline by Allena Pharmaceuticals Inc, H1 2019

Primary Hyperoxaluria – Pipeline by Alnylam Pharmaceuticals Inc, H1 2019

Primary Hyperoxaluria – Pipeline by BridgeBio Pharma LLC, H1 2019

Primary Hyperoxaluria – Pipeline by Dicerna Pharmaceuticals Inc, H1 2019

Primary Hyperoxaluria – Pipeline by Intellia Therapeutics Inc, H1 2019

Primary Hyperoxaluria – Pipeline by OxThera AB, H1 2019

Primary Hyperoxaluria – Pipeline by Precision Biosciences Inc, H1 2019

Primary Hyperoxaluria – Dormant Projects, H1 2019

Primary Hyperoxaluria – Discontinued Products, H1 2019

List of Figures

List of Figures

Number of Products under Development for Primary Hyperoxaluria, H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products ...

List of Figures

Number of Products under Development for Primary Hyperoxaluria, H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products by Stage and Targets, H1 2019

Number of Products by Stage and Mechanism of Actions, H1 2019

Number of Products by Routes of Administration, H1 2019

Number of Products by Stage and Routes of Administration, H1 2019

Number of Products by Molecule Types, H1 2019

Number of Products by Stage and Molecule Types, H1 2019

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