SEC Filings

Form 10-K
AVEXIS, INC. filed this Form 10-K on 03/16/2017
Document Outline
Entire Document (6128.4 KB)
Subdocument 1 - 10-K - 10-K
Page 1 - UNITED STATES
Page 2 - SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
Page 3 - N/A
Page 4 - TABLE OF CONTENTS [Check later]
Page 5 - PART I
Page 6 - SMN2
Page 7 - in vivo
Page 8 - Our Strategy
Page 9 - Advance the development of AVXS-101 outside of the United States.
Page 10 - Background on Spinal Muscular Atrophy
Page 11 - SMN1
Page 12 - Existing Treatments for SMA
Page 13 - Components of AVXS-101
Page 14 - Clinical Development of AVXS-101
Page 15 - N/A
Page 16 - Neuromuscular Disorders
Page 17 - N/A
Page 18 - Preclinical Studies
Page 19 - Future SMA Type 1 Clinical Development
Page 20 - Competition
Page 21 - Licensed Patents and Patent Applications
Page 22 - Nationwide Children's Hospital
Page 23 - The Research Institute at Nationwide Children's Hospital
Page 24 - REGENXBIO
Page 25 - Asklepios Biopharmaceutical, Inc.
Page 26 - Government Regulation and Product Approval
Page 27 - U.S. Biologic Products Development Process
Page 28 - Human Clinical Trials Under an IND
Page 29 - Phase 1.
Page 30 - Additional Regulation for Gene Therapy Clinical Trials
Page 31 - U.S. Review and Approval Processes
Page 32 - Orphan Drug Designation
Page 33 - Expedited Development and Review Programs
Page 34 - U.S. Patent Term Restoration and Marketing Exclusivity
Page 35 - Other Healthcare Laws and Regulations
Page 36 - Coverage and Reimbursement
Page 37 - Health Reform
Page 38 - N/A
Page 39 - Additional Regulation
Page 40 - Information about Segments
Page 41 - Item 1A. Risk Factors
Page 42 - We have never generated revenue from product sales and may never be profitable.
Page 43 - Our limited operating history may make it difficult for you to evaluate the success of our business
Page 44 - N/A
Page 45 - Risks related to the development of our current product candidate
Page 46 - AVXS-101 is based on a novel technology, which makes it difficult to predict the time and cost of de
Page 47 - Breakthrough therapy designation by the FDA and access to Priority Medicines, or PRIME, by the EMA m
Page 48 - Success in preclinical studies or early clinical trials, including in our Phase 1 clinical trial, ma
Page 49 - We may find it difficult to enroll patients in our clinical trials, which could delay or prevent us
Page 50 - N/A
Page 51 - Because the number of subjects in our clinical trial to date is small and have all been treated at o
Page 52 - As we evaluate and develop manufacturing process improvements to AVXS-101, we may be required to con
Page 53 - AVXS-101 may cause undesirable side effects or have other properties that could delay or prevent its
Page 54 - As an organization, we recently completed our first Phase 1 clinical trial, and have never conducted
Page 55 - Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain reg
Page 56 - We face significant competition in an environment of rapid technological change and the possibility
Page 57 - SMN2
Page 58 - Even if we obtain and maintain approval for AVXS-101 from the FDA, we may never obtain approval for
Page 59 - We may in the future enter into collaborations with third parties to develop AVXS-101. If these coll
Page 60 - N/A
Page 61 - We may not be successful in finding strategic collaborators for continuing development of AVXS-101 o
Page 62 - We are in the process of changing our third-party manufacturer of AVXS-101 and, although we have beg
Page 63 - We are simultaneously building our own manufacturing facility, and working with third-party manufact
Page 64 - Risks related to the commercialization of AVXS-101
Page 65 - If we are unable to establish sales, medical affairs and marketing capabilities or enter into agreem
Page 66 - If the market opportunities for AVXS-101 are smaller than we believe they are, our product revenues
Page 67 - Delays in obtaining regulatory approval of our manufacturing process and facility or disruptions in
Page 68 - Failure to comply with ongoing regulatory requirements could cause us to suspend production or put i
Page 69 - Any contamination in our manufacturing process, shortages of raw materials or failure of any of our
Page 70 - N/A
Page 71 - If we obtain approval to commercialize AVXS-101 outside of the United States, in particular in the E
Page 72 - If AVXS-101 becomes subject to a product recall it could harm our reputation, business and financial
Page 73 - We may fail to capitalize on other potential product candidates that may be a greater commercial opp
Page 74 - If we are unable to manage expected growth in the scale and complexity of our operations, our perfor
Page 75 - Healthcare legislative reform measures may have a material adverse effect on our business and result
Page 76 - Our relationships with customers, physicians, and third-party payors will be subject, directly or in
Page 77 - N/A
Page 78 - Product liability lawsuits against us could cause us to incur substantial liabilities and could limi
Page 79 - Third parties on which we rely and we may be adversely affected by natural disasters and our busines
Page 80 - Risks related to our intellectual property
Page 81 - If we are unable to obtain and maintain patent protection for our current product candidate, any fut
Page 82 - N/A
Page 83 - N/A
Page 84 - Our intellectual property licenses with third parties may be subject to disagreements over contract
Page 85 - We may not be successful in obtaining necessary rights to AVXS-101 through acquisitions and in-licen
Page 86 - Obtaining and maintaining our patent protection depends on compliance with various procedural, docum
Page 87 - We may not be able to protect our intellectual property rights throughout the world.
Page 88 - Issued patents covering AVXS-101 could be found invalid or unenforceable if challenged in court. We
Page 89 - Third parties may initiate legal proceedings alleging that we are infringing their intellectual prop
Page 90 - Intellectual property litigation could cause us to spend substantial resources and distract our pers
Page 91 - We may be subject to claims asserting that our employees, consultants or advisors have wrongfully us
Page 92 - N/A
Page 93 - If we do not obtain patent term extension for AVXS-101, our business may be materially harmed.
Page 94 - Intellectual property rights and regulatory exclusivity rights do not necessarily address all potent
Page 95 - N/A
Page 96 - Our executive officers, directors and principal stockholders will maintain the ability to control al
Page 97 - If we engage in future acquisitions or strategic partnerships, this may increase our capital require
Page 98 - Raising additional capital may cause dilution to our existing stockholders, restrict our operations
Page 99 - We will incur increased costs as a result of operating as a public company, and our management will
Page 100 - Our ability to utilize our net operating loss carryforwards and certain other tax attributes may be
Page 101 - Item 1B. Unresolved Staff Comments
Page 102 - PART II
Page 103 - Stockholders
Page 104 - Item 6. Selected Financial Data.
Page 105 - N/A
Page 106 - Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations.
Page 107 - Licensing Agreements
Page 108 - Asklepios Biopharmaceutical, Inc.
Page 109 - General and Administrative Expense
Page 110 - Research and Development Costs
Page 111 - N/A
Page 112 - Determination of the Fair Value of Stock-Based Compensation Grants
Page 113 - Common Stock Valuation Methodology
Page 114 - Emerging Growth Company Status
Page 115 - N/A
Page 116 - Results of Operations
Page 117 - Research and Development
Page 118 - General and Administrative
Page 119 - Cash Flows
Page 120 - Investing Activities
Page 121 - N/A
Page 122 - Contractual Obligations, Commitments and Contingencies
Page 123 - Item 8. Financial Statements and Supplementary Data.
Page 124 - N/A
Page 125 - N/A
Page 126 - AveXis, Inc.
Page 127 - N/A
Page 128 - 1. Background
Page 129 - Notes to Consolidated Financial Statements (Continued)
Page 130 - Notes to Consolidated Financial Statements (Continued)
Page 131 - Notes to Consolidated Financial Statements (Continued)
Page 132 - Notes to Consolidated Financial Statements (Continued)
Page 133 - Notes to Consolidated Financial Statements (Continued)
Page 134 - Notes to Consolidated Financial Statements (Continued)
Page 135 - Notes to Consolidated Financial Statements (Continued)
Page 136 - Notes to Consolidated Financial Statements (Continued)
Page 137 - Notes to Consolidated Financial Statements (Continued)
Page 138 - Notes to Consolidated Financial Statements (Continued)
Page 139 - Notes to Consolidated Financial Statements (Continued)
Page 140 - Notes to Consolidated Financial Statements (Continued)
Page 141 - Notes to Consolidated Financial Statements (Continued)
Page 142 - Notes to Consolidated Financial Statements (Continued)
Page 143 - Notes to Consolidated Financial Statements (Continued)
Page 144 - Notes to Consolidated Financial Statements (Continued)
Page 145 - Notes to Consolidated Financial Statements (Continued)
Page 146 - Notes to Consolidated Financial Statements (Continued)
Page 147 - Notes to Consolidated Financial Statements (Continued)
Page 148 - Notes to Consolidated Financial Statements (Continued)
Page 149 - Notes to Consolidated Financial Statements (Continued)
Page 150 - Notes to Consolidated Financial Statements (Continued)
Page 151 - Notes to Consolidated Financial Statements (Continued)
Page 152 - Notes to Consolidated Financial Statements (Continued)
Page 153 - Notes to Consolidated Financial Statements (Continued)
Page 154 - Notes to Consolidated Financial Statements (Continued)
Page 155 - Notes to Consolidated Financial Statements (Continued)
Page 156 - Notes to Consolidated Financial Statements (Continued)
Page 157 - Notes to Consolidated Financial Statements (Continued)
Page 158 - Notes to Consolidated Financial Statements (Continued)
Page 159 - Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure.
Page 160 - Remediation Efforts
Page 161 - Changes in Internal Control over Financial Reporting
Page 162 - PART III
Page 163 - Item 13. Certain Relationships and Related Transactions, and Director Independence.
Page 164 - PART IV
Page 165 - N/A
Page 166 - N/A
Page 167 - SIGNATURES
Page 168 - N/A
Page 169 - Exhibit Index
Page 170 - N/A
Page 171 - N/A
Subdocument 2 - EX-21.1 - EX-21.1
Page 1 - Exhibit 21.1
Page 2 - N/A
Subdocument 3 - EX-23.1 - EX-23.1
Page 1 - Exhibit 23.1
Page 2 - N/A
Subdocument 4 - EX-31.1 - EX-31.1
Page 1 - Exhibit 31.1
Page 2 - N/A
Subdocument 5 - EX-31.2 - EX-31.2
Page 1 - Exhibit 31.2
Page 2 - N/A
Subdocument 6 - EX-32.1 - EX-32.1
Page 1 - Exhibit 32.1
Page 2 - N/A
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