Achondroplasia - Pipeline Review, H2 2018

Global Markets Direct’s, ‘Achondroplasia - Pipeline Review, H2 2018’, provides an overview of the Achondroplasia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Achondroplasia, 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 Achondroplasia 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 Achondroplasia

- The report reviews pipeline therapeutics for Achondroplasia 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 Achondroplasia therapeutics and enlists all their major and minor projects

- The report assesses Achondroplasia 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 Achondroplasia

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 Achondroplasia

- 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 Achondroplasia 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

Ascendis Pharma A/S

BioClin Therapeutics Inc

BioMarin Pharmaceutical Inc

Ribomic Inc

Ascendis Pharma A/S

BioClin Therapeutics Inc

BioMarin Pharmaceutical Inc

Ribomic Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Achondroplasia – Overview

Achondroplasia – Therapeutics Development

Pipeline Overview

Pipeline by Companies ...

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Achondroplasia – Overview

Achondroplasia – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Products under Development by Companies

Achondroplasia – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Achondroplasia – Companies Involved in Therapeutics Development

Ascendis Pharma A/S

BioClin Therapeutics Inc

BioMarin Pharmaceutical Inc

Ribomic Inc

Achondroplasia – Drug Profiles

ACP-015 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

B-701 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

infigratinib – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RBM-007 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

TA-46 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

vosoritide – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Achondroplasia – Dormant Projects

Achondroplasia – Product Development Milestones

Featured News & Press Releases

Jun 21, 2018: Therachon Announces Start of Natural History Study in Children with Achondroplasia

Jun 14, 2018: BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia

May 08, 2018: Ascendis Pharma Announces Dosing of First Subjects in Phase 1 Trial of TransCon CNP

Feb 14, 2018: Therachon Announces Dosing of First Subject in Phase 1 Clinical Trial Evaluating TA-46, a Novel Investigational Therapy for the Potential Treatment of Achondroplasia

Jan 08, 2018: Ascendis Pharma Provides Update on TransCon CNP

Sep 05, 2017: Ascendis Pharma Announces Presentations on TransCon CNP at Upcoming Medical Conferences

Jun 20, 2017: Therachon Receives EMA and FDA Orphan Drug Designation for TA-46 for Achondroplasia

Apr 03, 2017: Ascendis Pharma Presents Data on TransCon CNP at ENDO 2017

Feb 28, 2017: Ascendis Pharma Announces Data on TransCon CNP at ENDO 2017

Dec 12, 2016: BioMarin Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia

Oct 19, 2016: BioMarin Presents Vosoritide Data in Achondroplasia at American Society of Human Genetics 2016 Meeting

Apr 20, 2016: BioMarin Provides Program Update on Vosoritide in Achondroplasia

Oct 12, 2015: BioMarin’s Initial 6-Month Data From Phase 2 Study of Vosoritide in Children With Achondroplasia Presented at the American Society for Bone and Mineral Research Annual 2015 Meeting

Jun 17, 2015: BMN 111 Improves Growth Velocity in Children With Achondroplasia in Phase 2 Study

Jan 15, 2014: BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia

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 Achondroplasia, H2 2018

Number of Products under Development by Companies, H2 2018

Products under Development by ...

List of Tables

Number of Products under Development for Achondroplasia, H2 2018

Number of Products under Development by Companies, H2 2018

Products under Development by Companies, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Achondroplasia – Pipeline by Ascendis Pharma A/S, H2 2018

Achondroplasia – Pipeline by BioClin Therapeutics Inc, H2 2018

Achondroplasia – Pipeline by BioMarin Pharmaceutical Inc, H2 2018

Achondroplasia – Pipeline by Ribomic Inc, H2 2018

Achondroplasia – Dormant Projects, H2 2018

List of Figures

List of Figures

Number of Products under Development for Achondroplasia, H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products by ...

List of Figures

Number of Products under Development for Achondroplasia, H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products by Targets, H2 2018

Number of Products by Stage and Targets, H2 2018

Number of Products by Mechanism of Actions, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

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