Table of Contents
Table of Contents 3
List of Tables 5
List of Figures 5
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 6
AveXis Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 7
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 8
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 9
AveXis Inc, Medical Devices Deals, 2012 to YTD 2018 10
AveXis Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 11
AveXis Inc, Pharmaceuticals & Healthcare, Deal Details 13
Venture Financing 13
AveXis Raises USD65 Million in Venture Financing 13
AveXis Raises USD10 Million in Series C Financing Round 15
Partnerships 16
AveXis Enters into Licensing Agreement with Genethon and Centre National de la Recherche Scientifique 16
Licensing Agreements 17
AveXis Enters into Licensing Agreement with Regenxbio 17
AveXis Enters into Licensing Agreement with Nationwide Children’s Hospital for Development of Rett syndrome 19
AveXis Enters into Licensing Agreement with Nationwide Children’s Hospital for Development of Amyotrophic Lateral Sclerosis 20
Asklepios BioPharma Enters into Licensing Agreement with AveXis 21
AveXis Amends Licensing Agreement With ReGenX Biosciences 22
AveXis Amends Licensing Agreement with Nationwide Children’s Hospital 23
Equity Offering 24
AveXis Raises USD460 Million in Public Offering of Shares 24
AveXis Raises USD287.8 Million in Public Offering of Shares 26
AveXis Raises USD146.6 Million in Public Offering of Shares 28
AveXis Completes Underwriter’s Exercise of Over-Allotment Option of IPO 30
AveXis Raises USD2 Million in Private Placement of Shares and Warrants 31
Debt Offering 32
AveXis Raises USD0.5 Million in Private Placement of 8% Notes 32
Acquisition 33
Novartis Acquires AveXis for USD8.7 Billion 33
AveXis Inc – Key Competitors 35
AveXis Inc – Key Employees 36
AveXis Inc – Locations And Subsidiaries 37
Head Office 37
Other Locations & Subsidiaries 37
Recent Developments 38
Financial Announcements 38
May 03, 2018: AveXis Reports First Quarter 2018 Financial and Operating Results 38
Feb 27, 2018: AveXis Reports Fourth Quarter and Full Year 2017 Financial and Operating Results 40
Nov 09, 2017: AveXis Reports Third Quarter 2017 Financial and Operating Results 42
Aug 10, 2017: AveXis Reports Second Quarter 2017 Financial and Operating Results 44
May 11, 2017: AveXis Reports First Quarter 2017 Financial and Operating Results 46
Corporate Communications 48
Feb 15, 2017: AveXis Appoints Rick Modi as Senior Vice President and Chief Business Officer 48
Product Approvals 49
Mar 27, 2018: AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1 49
Jan 04, 2018: AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1 50
Jun 14, 2017: AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101 51
Jan 31, 2017: AveXis Gene Therapy AVXS-101 Granted Access into EMA PRIME Program for Spinal Muscular Atrophy Type 1 53
Clinical Trials 54
May 08, 2018: AveXis Issues Community Statement on the SPR1NT Trial 54
Apr 25, 2018: AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3 55
Apr 24, 2018: AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology 57
Apr 19, 2018: AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology 59
Jan 30, 2018: AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients 60
Jan 16, 2018: AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy 61
Dec 13, 2017: AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101 62
Nov 01, 2017: AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1 64
Oct 03, 2017: AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society 66
Sep 29, 2017: AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process 67
Apr 25, 2017: AveXis Presents Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology 69
Apr 18, 2017: AveXis to Host Webcast of Preclinical and Clinical Studies of AVXS-101 as Presented at the Annual Meeting of the American Academy of Neurology 71
Mar 16, 2017: AveXis Reports Topline Results from Phase 1 Trial of AVXS-101 in SMA Type 1 and Fourth Quarter and Full Year 2016 Financial and Operating Results 72
Mar 09, 2017: AveXis to Report Topline Phase 1 Trial Results in SMA Type 1 75
Mar 02, 2017: AveXis to Present Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at AAN Annual Meeting 76
Feb 06, 2017: AveXis Announces Single-Arm Design for European Pivotal Study of AVXS-101 in SMA Type 1 Patients 77
Appendix 78
Methodology 78
About GlobalData 78
Contact Us 78
Disclaimer 78
AveXis Inc, Pharmaceuticals & Healthcare, Key Facts, 2017 2
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 6
AveXis Inc, Pharmaceuticals & Healthcare Deals By Type, 2012 to YTD 2018 7
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 8
AveXis Inc, Deals By Therapy Area, 2012 to YTD 2018 9
AveXis Inc, Medical Devices Deals, 2012 to YTD 2018 10
AveXis Inc, Pharmaceuticals & Healthcare, Deals Summary, 2012 to YTD 2018 11
AveXis Raises USD65 Million in Venture Financing 13
AveXis Raises USD10 Million in Series C Financing Round 15
AveXis Enters into Licensing Agreement with Genethon and Centre National de la Recherche Scientifique 16
AveXis Enters into Licensing Agreement with Regenxbio 17
AveXis Enters into Licensing Agreement with Nationwide Children’s Hospital for Development of Rett syndrome 19
AveXis Enters into Licensing Agreement with Nationwide Children’s Hospital for Development of Amyotrophic Lateral Sclerosis 20
Asklepios BioPharma Enters into Licensing Agreement with AveXis 21
AveXis Amends Licensing Agreement With ReGenX Biosciences 22
AveXis Amends Licensing Agreement with Nationwide Children's Hospital 23
AveXis Raises USD460 Million in Public Offering of Shares 24
AveXis Raises USD287.8 Million in Public Offering of Shares 26
AveXis Raises USD146.6 Million in Public Offering of Shares 28
AveXis Completes Underwriter’s Exercise of Over-Allotment Option of IPO 30
AveXis Raises USD2 Million in Private Placement of Shares and Warrants 31
AveXis Raises USD0.5 Million in Private Placement of 8% Notes 32
Novartis Acquires AveXis for USD8.7 Billion 33
AveXis Inc, Key Competitors 35
AveXis Inc, Key Employees 36
AveXis Inc, Subsidiaries 37
List of Figures
AveXis Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 2
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 2
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 2
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 2
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Year, 2012 to YTD 2018 6
AveXis Inc, Pharmaceuticals & Healthcare, Deals by Type, 2012 to YTD 2018 7
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Region, 2012 to YTD 2018 8
AveXis Inc, Pharmaceuticals & Healthcare, Deals By Therapy Area, 2012 to YTD 2018 9
AveXis Inc, Medical Devices Deals, 2012 to YTD 2018 10