SEC Filings

Form 10-K
AVEXIS, INC. filed this Form 10-K on 03/18/2016
Document Outline
Entire Document (2412.7 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
Page 5 - PART I
Page 6 - N/A
Page 7 - Our Strategy
Page 8 - Continue to invest in and develop robust and sustainable manufacturing processes and multiple supply
Page 9 - N/A
Page 10 - SMA Type 1
Page 11 - Existing Treatments for SMA
Page 12 - Components of AVXS-101
Page 13 - Preliminary Clinical Results
Page 14 - N/A
Page 15 - N/A
Page 16 - Preclinical Studies
Page 17 - Future SMA Type 1 Clinical Development
Page 18 - Alternative splicing
Page 19 - Licensed patents and patent applications
Page 20 - N/A
Page 21 - The Research Institute at Nationwide Children's Hospital
Page 22 - N/A
Page 23 - Asklepios Biopharmaceutical, Inc.
Page 24 - Government Regulation and Product Approval
Page 25 - U.S. Biologic Products Development Process
Page 26 - Human Clinical Trials Under an IND
Page 27 - Phase 1.
Page 28 - Compliance with cGMP Requirements
Page 29 - N/A
Page 30 - Orphan Drug Designation
Page 31 - Breakthrough therapy designation.
Page 32 - U.S. Patent Term Restoration and Marketing Exclusivity
Page 33 - Other Healthcare Laws and Regulations
Page 34 - Coverage and Reimbursement
Page 35 - Health Reform
Page 36 - Additional Regulation
Page 37 - Corporate Information
Page 38 - We have never generated revenue from product sales and may never be profitable.
Page 39 - Our limited operating history may make it difficult for you to evaluate the success of our business
Page 40 - We will need to raise additional funding, which may not be available on acceptable terms, or at all.
Page 41 - Risks related to the development of our current product candidate
Page 42 - AVXS-101 is based on a novel technology, which makes it difficult to predict the time and cost of de
Page 43 - N/A
Page 44 - Success in preclinical studies or early clinical trials, including in our ongoing Phase 1 clinical t
Page 45 - We may find it difficult to enroll patients in our clinical trials, which could delay or prevent us
Page 46 - Because the number of subjects in our clinical trial to date is small and have all been treated at o
Page 47 - N/A
Page 48 - As we evaluate and develop manufacturing process improvements to AVXS-101, we may be required to con
Page 49 - N/A
Page 50 - As an organization, we are in the process of conducting our first Phase 1 clinical trial, and have n
Page 51 - Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain reg
Page 52 - Even if we obtain regulatory approval for a product candidate, our product candidates will remain su
Page 53 - We face significant competition in an environment of rapid technological change and the possibility
Page 54 - Even if we obtain and maintain approval for AVXS-101 from the FDA, we may never obtain approval for
Page 55 - We may in the future enter into collaborations with third parties to develop AVXS-101. If these coll
Page 56 - N/A
Page 57 - We may not be successful in finding strategic collaborators for continuing development of AVXS-101 o
Page 58 - We are in the process of changing our third-party manufacturer of AVXS-101 and, although we intend t
Page 59 - To the extent we rely on a third-party manufacturing facility for commercial supply, that third part
Page 60 - Risks related to the commercialization of AVXS-101
Page 61 - If we are unable to establish sales, medical affairs and marketing capabilities or enter into agreem
Page 62 - If the market opportunities for AVXS-101 are smaller than we believe they are, our product revenues
Page 63 - Delays in obtaining regulatory approval of our manufacturing process and facility or disruptions in
Page 64 - Failure to comply with ongoing regulatory requirements could cause us to suspend production or put i
Page 65 - The insurance coverage and reimbursement status of newly-approved products is uncertain. Failure to
Page 66 - N/A
Page 67 - If we obtain approval to commercialize AVXS-101 outside of the United States, in particular in the E
Page 68 - If AVXS-101 becomes subject to a product recall it could harm our reputation, business and financial
Page 69 - We may fail to capitalize on other potential product candidates that may be a greater commercial opp
Page 70 - If we are unable to manage expected growth in the scale and complexity of our operations, our perfor
Page 71 - Healthcare legislative reform measures may have a material adverse effect on our business and result
Page 72 - Our relationships with customers, physicians, and third-party payors will be subject, directly or in
Page 73 - N/A
Page 74 - Product liability lawsuits against us could cause us to incur substantial liabilities and could limi
Page 75 - Third parties on which we rely and we may be adversely affected by natural disasters and our busines
Page 76 - Risks related to our intellectual property
Page 77 - If we are unable to obtain and maintain patent protection for our current product candidate, any fut
Page 78 - N/A
Page 79 - N/A
Page 80 - Our intellectual property licenses with third parties may be subject to disagreements over contract
Page 81 - We may not be successful in obtaining necessary rights to AVXS-101 through acquisitions and in-licen
Page 82 - Obtaining and maintaining our patent protection depends on compliance with various procedural, docum
Page 83 - We may not be able to protect our intellectual property rights throughout the world.
Page 84 - N/A
Page 85 - Third parties may initiate legal proceedings alleging that we are infringing their intellectual prop
Page 86 - Intellectual property litigation could cause us to spend substantial resources and distract our pers
Page 87 - We may be subject to claims asserting that our employees, consultants or advisors have wrongfully us
Page 88 - N/A
Page 89 - If we do not obtain patent term extension for AVXS-101, our business may be materially harmed.
Page 90 - Risks related to ownership of our common stock
Page 91 - Concentration of ownership of our common stock among our executive officers and directors and their
Page 92 - A significant portion of our total outstanding shares are restricted from immediate resale but may b
Page 93 - We have broad discretion in the use of our cash and cash equivalents, including the net proceeds fro
Page 94 - If we engage in future acquisitions or strategic partnerships, this may increase our capital require
Page 95 - We will incur increased costs as a result of operating as a public company, and our management will
Page 96 - Provisions in our corporate charter documents and under Delaware law could make an acquisition of us
Page 97 - Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable futur
Page 98 - PART II
Page 99 - N/A
Page 100 - Item 6. Selected Financial Data.
Page 101 - Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations.
Page 102 - Licensing Agreements
Page 103 - Asklepios Biopharmaceutical, Inc.
Page 104 - General and Administrative Expense
Page 105 - Critical Accounting Policies and Significant Judgments and Estimates
Page 106 - N/A
Page 107 - N/A
Page 108 - Determination of the Fair Value of Stock-Based Compensation Grants
Page 109 - Common Stock Valuation Methodology
Page 110 - Tax Indemnity
Page 111 - Emerging Growth Company Status
Page 112 - Results of Operations
Page 113 - General and Administrative
Page 114 - Comparison of the Years Ended December 31, 2014 and 2013
Page 115 - Interest Expense
Page 116 - Cash Flows
Page 117 - Investing Activities
Page 118 - N/A
Page 119 - Contractual Obligations, Commitments and Contingencies
Page 120 - Item 8. Financial Statements and Supplementary Data.
Page 121 - Consolidated Balance Sheets
Page 122 - N/A
Page 123 - N/A
Page 124 - AveXis, Inc.
Page 125 - N/A
Page 126 - 1. Background
Page 127 - Notes to Consolidated Financial Statements (Continued)
Page 128 - Notes to Consolidated Financial Statements (Continued)
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 - Notes to Consolidated Financial Statements (Continued)
Page 160 - Notes to Consolidated Financial Statements (Continued)
Page 161 - Notes to Consolidated Financial Statements (Continued)
Page 162 - Notes to Consolidated Financial Statements (Continued)
Page 163 - Notes to Consolidated Financial Statements (Continued)
Page 164 - Notes to Consolidated Financial Statements (Continued)
Page 165 - Notes to Consolidated Financial Statements (Continued)
Page 166 - Notes to Consolidated Financial Statements (Continued)
Page 167 - Notes to Consolidated Financial Statements (Continued)
Page 168 - Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure.
Page 169 - Item 9B. Other Information.
Page 170 - PART III
Page 171 - Thomas J. Dee
Page 172 - Andrew F. Knudten
Page 173 - Jonathan Leff
Page 174 - Section 16(a) Beneficial Ownership Reporting Compliance
Page 175 - Compensation Committee
Page 176 - Summary Compensation Table
Page 177 - Narrative to Summary Compensation Table
Page 178 - Annual Bonus and Non-Equity Incentive Plan Compensation
Page 179 - Exchange Agreement
Page 180 - Outstanding Equity Awards at December 31, 2015
Page 181 - Employment Agreements
Page 182 - Dr. Nagendran
Page 183 - Dr. Kaspar
Page 184 - Mr. Nolan
Page 185 - Mr. Knudten
Page 186 - Reversion of Shares.
Page 187 - Tax Limitations on Incentive Stock Options.
Page 188 - Stock Appreciation Rights.
Page 189 - N/A
Page 190 - Other Stock Awards.
Page 191 - Stock Awards.
Page 192 - Amendment and Termination
Page 193 - Other Terms
Page 194 - N/A
Page 195 - Compensation Committee Interlocks and Insider Participation
Page 196 - N/A
Page 197 - Equity Compensation Plan Information
Page 198 - Participation in our Initial Public Offering
Page 199 - Private Placements of our Securities
Page 200 - Investor Rights Agreement
Page 201 - Indemnification Agreements
Page 202 - Director Independence
Page 203 - PART IV
Page 204 - N/A
Page 205 - N/A
Page 206 - SIGNATURES
Page 207 - N/A
Page 208 - Exhibit Index
Page 209 - N/A
Page 210 - N/A
Subdocument 2 - EX-21.1 - EX-21.1
Page 1 - Exhibit 21.1
Subdocument 3 - EX-31.1 - EX-31.1
Page 1 - Exhibit 31.1
Subdocument 4 - EX-31.2 - EX-31.2
Page 1 - Exhibit 31.2
Subdocument 5 - EX-32.1 - EX-32.1
Page 1 - Exhibit 32.1
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