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
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