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Ultragenyx Pharmaceutical Inc (RARE)：製薬・医療：M&Aディール及び事業提携情報

◆英語タイトル：Ultragenyx Pharmaceutical Inc (RARE) - Pharmaceuticals & Healthcare - Deals and Alliances Profile
◆商品コード：DATA904C9926
◆発行会社（調査会社）：GlobalData
◆発行日：2018年11月
◆ページ数：106
◆レポート言語：英語
◆レポート形式：PDF
◆納品方法：Eメール
◆対象地域：アメリカ
◆産業分野：製薬・医療
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【レポートの概要】

Summary
Ultragenyx Pharmaceutical Inc (Ultragenyx) is a biotechnology company that develops life-enhancing therapeutics for rare and ultra-rare genetic diseases. Its only marketed product, Mepsevii (vestronidase alfa) is the first indication approved for the treatment of adults and children with Mucopolysaccharidosis (MPS) VII (Sly syndrome). The company’s pipeline comprises Burosumab (KRN23 or UX023), a fully human monoclonal antibody in Phase 3 for the treatment of X-linked hypophosphatemia (XLH); and Burosumab in Phase 2 for the treatment of tumor-induced osteomalacia (TIO); UX007 in Phase 3 for patients with Glut1 DS; UX007 as an oral administration in Phase 2 for the treatment of LC-FAOD. DTX301, in Phase 2 for the treatment of ornithine transcarbamylase (OTC) deficiency; DTX401 in Phase 1 for the treatment of GSDIa, and DTX201 in Phase 1 for the treatment of hemophilia A. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.

Ultragenyx Pharmaceutical Inc (RARE) – Pharmaceuticals & Healthcare – Deals and Alliances Profile provides you comprehensive data and trend analysis of the company’s Mergers and Acquisitions (M&As), partnerships and financings. The report provides detailed information on Mergers and Acquisitions, Equity/Debt Offerings, Private Equity, Venture Financing and Partnership transactions recorded by the company over a five year period. The report offers detailed comparative data on the number of deals and their value categorized into deal types, sub-sector and regions.

GlobalData derived the data presented in this report from proprietary in-house Pharma eTrack deals database, and primary and secondary research.

Scope

- Financial Deals – Analysis of the company’s financial deals including Mergers and Acquisitions, Equity/Debt Offerings, Private Equity, Venture Financing and Partnerships.
- Deals by Year – Chart and table displaying information encompassing the number of deals and value reported by the company by year, for a five year period.
- Deals by Type – Chart and table depicting information including the number of deals and value reported by the company by type such as Mergers and Acquisitions, Equity/Debt Offering etc.
- Deals by Region – Chart and table presenting information on the number of deals and value reported by the company by region, which includes North America, Europe, Asia Pacific, the Middle East and Africa and South and Central America.
- Deals by Sub-sector – Chart and table showing information on the number of deals and value reported by the company, by sub-sector.
- Major Deals – Information on the company’s major financial deals. Each such deal has a brief summary, deal type, deal rationale; and deal financials and target Company’s (major public companies) key financial metrics and ratios.
- Business Description – A brief description of the company’s operations.
- Key Employees – A list of the key executives of the company.
- Important Locations and Subsidiaries – A list and contact details of key centers of operation and subsidiaries of the company.
- Key Competitors – A list of the key competitors of the company.
- Key Recent Developments – A brief on recent news about the company.

Reasons to Buy

Get detailed information on the company’s financial deals that enable you to understand the company’s expansion/divestiture and fund requirements
- The profile enables you to analyze the company’s financial deals by region, by year, by business segments and by type, for a five year period.

Understand the company’s business segments’ expansion / divestiture strategy
- The profile presents deals from the company’s core business segments’ perspective to help you understand its corporate strategy.

Access elaborate information on the company’s recent financial deals that enable you to understand the key deals which have shaped the company
- Detailed information on major recent deals includes a summary of each deal, deal type, deal rationale, deal financials and Target Company’s key financial metrics and ratios.

Equip yourself with detailed information about the company’s operations to identify potential customers and suppliers.
- The profile analyzes the company’s business structure, locations and subsidiaries, key executives and key competitors.

Stay up-to-date on the major developments affecting the company
- Recent developments concerning the company presented in the profile help you track important events.

Gain key insights into the company for academic or business research
- Key elements such as break up of deals into categories and information on detailed major deals are incorporated into the profile to assist your academic or business research needs.

Note*: Some sections may be missing if data is unavailable for the company.

【レポートの目次】

Table of Contents
Table of Contents 3
List of Tables 5
List of Figures 5
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 8
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 10
Ultragenyx Pharmaceutical Inc, Medical Devices Deals, 2012 to YTD 2018 11
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 12
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deal Details 15
Venture Financing 15
Dimension Therapeutics Raises USD65 Million in Series B Financing 15
Dimension Therapeutics Raises USD30 Million in Venture Financing 17
Dimension Therapeutics Raises USD5 Million in Series A Financing 18
Ultragenyx Pharma Raises US$75 Million In Series B Financing 19
Ultragenyx Pharma Raises US$15.1 Million In Venture Financing 21
Partnerships 22
Aptuit Enters into Agreement with Dimension Therapeutics 22
Takeda Pharma Enters into Co-Development Agreement with Ultragenyx Pharma 23
Ultragenyx Pharma Enters into Research Agreement with Saint Louis University 24
Dimension Therapeutics Enters into R&D Agreement with Eunice Kennedy Shriver National Institute 25
Dimension Therapeutics Amends Research Agreement with University of Pennsylvania 26
Licensing Agreements 27
Ultragenyx Pharma Exercises Option for Licensing Agreement with Regenxbio 27
Dimension Therapeutics Enters into Licensing Agreement with Perelman School of Medicine at the University of Pennsylvania 28
Ultragenyx Pharma Enters into Licensing Agreement with Arcturus Therapeutics 29
ReGenX Biosciences Enters into Option And Licensing Agreement with Dimension Therapeutics 30
Ultragenyx Pharma Enters into Licensing Agreement with Inserm Transfert and Institut du Cerveau et de la Moelle Epiniere 31
Bayer HealthCare Enters into Licensing Agreement with Dimension Therapeutics for Hemophilia A 33
ReGenX Biosciences Amends Licensing Agreement With Dimension Therapeutics 34
Ultragenyx Pharma Enters Into Licensing Agreement With Kyowa Hakko Kirin For KRN23 35
Ultragenyx Pharma Expands Licensing Agreement With Baylor Research Institute For Triheptanoin 37
Ultragenyx Pharmaceutical Amends its Licensing Agreement with St. Jude Children’s Research Hospital 39
Ultragenyx Pharma Enters into Licensing Agreement with HIBM Research 40
Ultragenyx Pharma Enters into Licensing Agreement with UniQuest for Triheptanoin 41
Ultragenyx Pharma Enters Into Licensing Agreement With Saint Louis University 42
Equity Offering 43
Ultragenyx Pharma Plans to Raise up to USD72.6 Million in Public Offering of Shares 43
Ultragenyx Pharma Raises USD287.5 Million in Public Offering of Shares 44
Ultragenyx Pharma Plans to Raise up to USD150 Million in Public Offering of Shares 46
Dimension Therapeutics Plans to Raise up to USD35 Million in Public Offering of Shares 47
Ultragenyx Pharma Raises up to USD65 Million in Private Placement of Shares 48
Dimension Therapeutics Prices IPO for USD72.86 Million 50
Ultragenyx Pharma Completes Public Offering of Shares for USD303.6 Million 52
Ultragenyx Pharma Raises USD162 Million in Public Offering of Shares 54
Ultragenyx Pharma Raises USD76.7 Million in Public Offering of Shares 56
Ultragenyx Pharma Completes IPO For US$139 Million 58
Asset Transactions 59
Novartis Acquires Rare Pediatric Disease Priority Review Voucher from Ultragenyx Pharma for USD130 Million 59
Acquisition 60
Ultragenyx Pharma Acquires Dimension Therapeutics for USD152.3 Million 60
RegenxBio to Acquire Dimension Therapeutics 62
Ultragenyx Pharmaceutical Inc – Key Competitors 64
Ultragenyx Pharmaceutical Inc – Key Employees 65
Ultragenyx Pharmaceutical Inc – Locations And Subsidiaries 66
Head Office 66
Other Locations & Subsidiaries 66
Recent Developments 67
Financial Announcements 67
Nov 05, 2018: Ultragenyx reports third quarter 2018 financial results and corporate update 67
Aug 02, 2018: Ultragenyx reports second quarter 2018 financial results and corporate update 69
May 07, 2018: Ultragenyx Reports First Quarter 2018 Financial Results 71
Feb 20, 2018: Ultragenyx Reports Fourth Quarter and Full Year 2017 Financial Results and Corporate Update 73
Nov 02, 2017: Ultragenyx Reports Third Quarter 2017 Financial Results and Corporate Update 76
Jul 27, 2017: Ultragenyx Reports Second Quarter 2017 Financial Results and Corporate Update 78
May 04, 2017: Ultragenyx Reports First Quarter 2017 Financial Results and Corporate Update 80
Feb 16, 2017: Ultragenyx Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Update 82
Corporate Communications 84
Sep 17, 2018: Ultragenyx Pharmaceutical appoints Dr Wladimir Hogenhuis as chief operating officer 84
Jan 29, 2018: Ultragenyx Names Camille L. Bedrosian as Chief Medical Officer and Executive Vice President 85
Apr 10, 2017: Ultragenyx Appoints Deborah Dunsire, M.D., to Board of Directors 86
Legal and Regulatory 87
Aug 02, 2018: Certara Software or Services supported 95% of the US FDA novel drug approvals in first half of 2018 87
Product News 88
11/16/2017: Ultragenyx Announces Completion of Patient Dosing in First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency 88
08/31/2017: Dimension Therapeutics Commences Patient Dosing in Global, Multi-Center Phase 1/2 Clinical Trial of DTX301 in Ornithine Transcarbamylase Deficiency 89
07/30/2018: Ultragenyx doses first patient in DTX401 Phase 1/2 study to treat GSDIa 90
04/24/2017: Dimension Therapeutics Announces Presentations at the Upcoming American Society for Gene and Cell Therapy Annual Meeting 91
04/23/2018: Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia 93
03/09/2017: Dimension Therapeutics Provides Update on DTX-101 94
03/07/2018: Ultragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigational Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency 96
01/31/2017: Dimension Announces Interim Topline Results from Ongoing Phase 1/2 Clinical Program for DTX101, Dimension’s Lead AAV Product Candidate in Development for Adult Patients with Moderate/Severe to Severe Hemophilia B 98
01/07/2018: Ultragenyx Announces Positive Interim Topline Results From First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency 100
Product Approvals 101
Aug 29, 2018: Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders 101
Clinical Trials 102
Jan 04, 2018: Ultragenyx Announces Update to UX007 Development Program in Long-Chain Fatty Acid Oxidation Disorder Patients 102
Apr 27, 2017: Ultragenyx Initiates Global Phase 3 Study of UX007 in Glut1 DS Patients with Disabling Movement Disorders 103
Mar 22, 2017: Ultragenyx Announces Topline Data from Phase 2 UX007 Glucose Transporter Type-1 Deficiency Syndrome Seizure Study 104
Appendix 106
Methodology 106
About GlobalData 106
Contact Us 106
Disclaimer 106

List of Tables
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Key Facts, 2017 2
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 8
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
Ultragenyx Pharmaceutical Inc, Deals By Therapy Area, 2012 to YTD 2018 10
Ultragenyx Pharmaceutical Inc, Medical Devices Deals, 2012 to YTD 2018 11
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 12
Dimension Therapeutics Raises USD65 Million in Series B Financing 15
Dimension Therapeutics Raises USD30 Million in Venture Financing 17
Dimension Therapeutics Raises USD5 Million in Series A Financing 18
Ultragenyx Pharma Raises US$75 Million In Series B Financing 19
Ultragenyx Pharma Raises US$15.1 Million In Venture Financing 21
Aptuit Enters into Agreement with Dimension Therapeutics 22
Takeda Pharma Enters into Co-Development Agreement with Ultragenyx Pharma 23
Ultragenyx Pharma Enters into Research Agreement with Saint Louis University 24
Dimension Therapeutics Enters into R&D Agreement with Eunice Kennedy Shriver National Institute 25
Dimension Therapeutics Amends Research Agreement with University of Pennsylvania 26
Ultragenyx Pharma Exercises Option for Licensing Agreement with Regenxbio 27
Dimension Therapeutics Enters into Licensing Agreement with Perelman School of Medicine at the University of Pennsylvania 28
Ultragenyx Pharma Enters into Licensing Agreement with Arcturus Therapeutics 29
ReGenX Biosciences Enters into Option And Licensing Agreement with Dimension Therapeutics 30
Ultragenyx Pharma Enters into Licensing Agreement with Inserm Transfert and Institut du Cerveau et de la Moelle Epiniere 31
Bayer HealthCare Enters into Licensing Agreement with Dimension Therapeutics for Hemophilia A 33
ReGenX Biosciences Amends Licensing Agreement With Dimension Therapeutics 34
Ultragenyx Pharma Enters Into Licensing Agreement With Kyowa Hakko Kirin For KRN23 35
Ultragenyx Pharma Expands Licensing Agreement With Baylor Research Institute For Triheptanoin 37
Ultragenyx Pharmaceutical Amends its Licensing Agreement with St. Jude Children’s Research Hospital 39
Ultragenyx Pharma Enters into Licensing Agreement with HIBM Research 40
Ultragenyx Pharma Enters into Licensing Agreement with UniQuest for Triheptanoin 41
Ultragenyx Pharma Enters Into Licensing Agreement With Saint Louis University 42
Ultragenyx Pharma Plans to Raise up to USD72.6 Million in Public Offering of Shares 43
Ultragenyx Pharma Raises USD287.5 Million in Public Offering of Shares 44
Ultragenyx Pharma Plans to Raise up to USD150 Million in Public Offering of Shares 46
Dimension Therapeutics Plans to Raise up to USD35 Million in Public Offering of Shares 47
Ultragenyx Pharma Raises up to USD65 Million in Private Placement of Shares 48
Dimension Therapeutics Prices IPO for USD72.86 Million 50
Ultragenyx Pharma Completes Public Offering of Shares for USD303.6 Million 52
Ultragenyx Pharma Raises USD162 Million in Public Offering of Shares 54
Ultragenyx Pharma Raises USD76.7 Million in Public Offering of Shares 56
Ultragenyx Pharma Completes IPO For US$139 Million 58
Novartis Acquires Rare Pediatric Disease Priority Review Voucher from Ultragenyx Pharma for USD130 Million 59
Ultragenyx Pharma Acquires Dimension Therapeutics for USD152.3 Million 60
RegenxBio to Acquire Dimension Therapeutics 62
Ultragenyx Pharmaceutical Inc, Key Competitors 64
Ultragenyx Pharmaceutical Inc, Key Employees 65
Ultragenyx Pharmaceutical Inc, Subsidiaries 66

List of Figures
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 2
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 2
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 2
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 2
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 7
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 8
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 9
Ultragenyx Pharmaceutical Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 10
Ultragenyx Pharmaceutical Inc, Medical Devices Deals, 2012 to YTD 2018 11

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