Document and Entity Information
| Document and Entity Information - shares | 3 Months Ended | |
| Mar. 31, 2016 | May. 12, 2016 | |
| Document and Entity Information [Abstract] | ||
| Entity Registrant Name | AveXis, Inc. | |
| Entity Central Index Key | 1,652,923 | |
| Document Type | 10-Q | |
| Document Period End Date | Mar. 31, 2016 | |
| Amendment Flag | false | |
| Current Fiscal Year End Date | --12-31 | |
| Entity Current Reporting Status | Yes | |
| Entity Filer Category | Non-accelerated Filer | |
| Entity Common Stock, Shares Outstanding | 23,013,838 | |
| Document Fiscal Year Focus | 2,016 | |
| Document Fiscal Period Focus | Q1 |
Consolidated Balance Sheets
| Consolidated Balance Sheets - USD ($) | Mar. 31, 2016 | Dec. 31, 2015 |
| Current assets: | ||
| Cash and cash equivalents | $ 148,237,433 | $ 62,251,860 |
| Prepaid expenses and other current assets | 1,461,287 | 909,629 |
| Total current assets | 149,698,720 | 63,161,489 |
| Property and equipment, net | 5,944,656 | 235,590 |
| Other long-term assets | 400,134 | 1,687,212 |
| Total assets | 156,043,510 | 65,084,291 |
| Current liabilities: | ||
| Accounts payable | 2,852,827 | 359,787 |
| Accrued expenses | 2,806,316 | 2,437,017 |
| Accrued indemnification obligation | 4,080,500 | 4,080,500 |
| Total current liabilities | 9,739,643 | 6,877,304 |
| Total liabilities | $ 9,739,643 | $ 6,877,304 |
| Commitments and contingencies (Note 10) | ||
| Redeemable common stock; par value $0.0001 per share, no shares issued and outstanding at March 31, 2016; 456.043 shares issued and outstanding at December 31, 2015 | $ 1,032,909 | |
| Stockholders' equity: | ||
| Common stock; par value $0.0001 per share, 100,000,000 shares authorized, 23,013,838 shares issued and outstanding at March 31, 2016; 22,080,000 shares authorized, 6,817,093 shares issued and outstanding at December 31, 2015 | $ 2,301 | 682 |
| Additional paid-in capital | 225,688,193 | 115,723,046 |
| Accumulated deficit | (79,386,627) | (58,550,520) |
| Total stockholder's equity | 146,303,867 | 57,174,078 |
| Total liabilities, redeemable common stock and stockholders' equity | $ 156,043,510 | 65,084,291 |
| Class D Preferred Stock | ||
| Stockholders' equity: | ||
| Preferred stock | 309 | |
| Class C Preferred Stock | ||
| Stockholders' equity: | ||
| Preferred stock | 237 | |
| Class B-1 Preferred Stock | ||
| Stockholders' equity: | ||
| Preferred stock | $ 324 |
Consolidated Balance Sheets (Pa
| Consolidated Balance Sheets (Parenthetical) - USD ($) | Mar. 31, 2016 | Dec. 31, 2015 |
| Redeemable common stock | ||
| Redeemable common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Redeemable common stock, shares issued (in shares) | 0 | 456,043 |
| Redeemable common stock, shares outstanding (in shares) | 0 | 456,043 |
| Common stock | ||
| Common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Common stock, shares authorized (in shares) | 100,000,000 | 22,080,000 |
| Common stock, shares issued | 23,013,838 | 6,817,093 |
| Common stock, shares outstanding | 23,013,838 | 6,817,093 |
| Class D Preferred Stock | ||
| Preferred stock | ||
| Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Preferred stock, shares authorized (in shares) | 0 | 3,105,000 |
| Preferred stock, shares issued (in shares) | 0 | 3,093,092 |
| Preferred stock, shares outstanding (in shares) | 0 | 3,093,092 |
| Class C Preferred Stock | ||
| Preferred stock | ||
| Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Preferred stock, shares authorized (in shares) | 0 | 2,365,020 |
| Preferred stock, shares issued (in shares) | 0 | 2,365,020 |
| Preferred stock, shares outstanding (in shares) | 0 | 2,365,020 |
| Preferred stock, aggregate liquidation preference (in dollars) | $ 5,003,492 | |
| Class B-2 Preferred Stock | ||
| Preferred stock | ||
| Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Preferred stock, shares authorized (in shares) | 326,557 | 326,557 |
| Preferred stock, shares issued (in shares) | 0 | 0 |
| Preferred stock, shares outstanding (in shares) | 0 | 0 |
| Class B-1 Preferred Stock | ||
| Preferred stock | ||
| Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Preferred stock, shares authorized (in shares) | 0 | 3,278,938 |
| Preferred stock, shares issued (in shares) | 0 | 3,237,528 |
| Preferred stock, shares outstanding (in shares) | 0 | 3,237,528 |
| Preferred stock, aggregate liquidation preference (in dollars) | $ 8,000,003 | |
| Undesignated Preferred Stock | ||
| Preferred stock | ||
| Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
| Preferred stock, shares authorized (in shares) | 10,000,000 | 1,000,000 |
| Preferred stock, shares issued (in shares) | 0 | 0 |
| Preferred stock, shares outstanding (in shares) | 0 | 0 |
Consolidated Statements of Oper
| Consolidated Statements of Operations and Comprehensive Loss - USD ($) | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Operating expenses: | ||
| General and administrative | $ 4,823,913 | $ 642,634 |
| Research and development | 16,065,089 | 4,812,025 |
| Total operating expenses | 20,889,002 | 5,454,659 |
| Loss from operations | (20,889,002) | (5,454,659) |
| Interest income | 52,895 | 1,455 |
| Net loss | (20,836,107) | (5,453,204) |
| Comprehensive loss | $ (20,836,107) | $ (5,453,204) |
| Basic and diluted net loss per common share (in dollars per share) | $ (1.24) | $ (0.79) |
| Weighted-average basic and diluted common shares outstanding (in shares) | 16,774,718 | 6,895,185 |
Consolidated Statements of Cash
| Consolidated Statements of Cash Flows - USD ($) | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Cash flows from operating activities | ||
| Net loss | $ (20,836,107) | $ (5,453,204) |
| Adjustments to reconcile net loss to net cash used in operating activities: | ||
| Depreciation and amortization | 11,045 | 1,350 |
| Stock-based compensation | 13,543,547 | 95,230 |
| Stock-based third-party research and development | 4,067,201 | |
| Noncash research and development | 412,554 | |
| Changes in operating assets and liabilities | ||
| Prepaid expenses and other current assets | (420,180) | (39,749) |
| Other long-term assets | (731,663) | |
| Accounts payable | 580,688 | 236,691 |
| Accrued expenses | 169,971 | (138,569) |
| Net cash used in operating activities | (7,682,699) | (818,496) |
| Cash flows from investing activities | ||
| Capital expenditures | (3,659,718) | (4,803) |
| Net cash used in investing activities | (3,659,718) | (4,803) |
| Cash flows from financing activities | ||
| Proceeds from issuance of common stock, net of issuance costs | 98,169,703 | |
| Payments of deferred offering costs | (883,713) | |
| Proceeds from exercise of stock warrants | 42,000 | |
| Net cash provided by financing activities | 97,327,990 | 5,000,020 |
| Net increase in cash and cash equivalents | 85,985,573 | 4,176,721 |
| Cash and cash equivalents, beginning of period | 62,251,860 | 3,119,713 |
| Cash and cash equivalents, end of period | 148,237,433 | 7,296,434 |
| Supplemental Cash Flow Information | ||
| Cash paid for interest | 275 | |
| Purchases of property and equipment in accounts payable and accrued liabilities | 2,060,392 | |
| Deferred issuance costs for planned initial public offering in accrued expenses | $ 290,848 | |
| Class C Preferred Stock | ||
| Cash flows from financing activities | ||
| Proceeds from issuance of common stock | $ 5,000,020 | |
Background
| Background | 3 Months Ended |
| Mar. 31, 2016 | |
| Background | |
| Background | 1. Background AveXis, Inc. was formed on March 8, 2010 in the state of Delaware as Biolife Cell Bank, LLC. In January 2012, the Company converted from a limited liability company to a corporation, Biolife Cell Bank, Inc. In January 2014, the Company amended and restated its Certificate of Incorporation to change its name to AveXis, Inc. (“AveXis” or “the Company”). The Company is a clinical-stage gene therapy company dedicated to developing and commercializing gene therapy treatments for patients suffering from rare and life-threatening neurological genetic diseases. The Company’s initial product candidate, AVXS-101, is a gene therapy product candidate currently in a Phase 1 clinical trial for the treatment of spinal muscular atrophy (“SMA”), Type 1, the leading genetic cause of infant mortality. Initial Public Offering On February 17, 2016, the Company completed an initial public offering (“IPO”), which resulted in the issuance and sale of 4,750,000 shares of its common stock at a public offering price of $20.00 per share, resulting in net proceeds of approximately $88,350,000 after deducting underwriting discounts and other estimated offering costs. Upon the closing of the IPO, the 3,278,938 shares of Class B-1 preferred stock, 326,557 shares of Class B-2 preferred stock, 2,365,020 shares of Class C preferred stock and 3,105,000 of Class D preferred stock were automatically converted into shares of the Company’s common stock. On March 3, 2016, the underwriters of the Company’s IPO exercised their over-allotment option to purchase an additional 527,941 shares of the Company’s common stock at the initial public offering price of $20.00 per share, less underwriting discounts, resulting in net proceeds of approximately $9,800,000. |
Summary of Significant Accounti
| Summary of Significant Accounting Policies | 3 Months Ended |
| Mar. 31, 2016 | |
| Summary of Significant Accounting Policies | |
| Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) for interim financial reporting and the rules and regulations of the U.S. Securities and Exchange Commission (“SEC”) and, therefore, do not include all information and footnote disclosures normally included in the annual consolidated financial statements. In the opinion of management, the unaudited condensed consolidated financial statements reflect all adjustments of a normal recurring nature that are necessary for a fair presentation of the results for the interim periods presented. Interim results are not necessarily indicative of results for a full year. The December 31, 2015 condensed consolidated balance sheet data contained within this Form 10-Q was derived from audited consolidated financial statements included in the Company’s Form 10-K for the year ended December 31, 2015 (“Annual Report”), but does not include all disclosures required by accounting principles generally accepted in the United States. Significant Accounting Policies There have been no material changes in the Company’s significant accounting policies as of and for the three months ended March 31, 2016, as compared with the significant accounting policies described in the Company’s Annual Report. Use of Estimates The preparation of the condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. In the Company’s opinion, the accompanying unaudited condensed consolidated financial statements include all normal recurring adjustments necessary for a fair presentation of the Company’s financial position, results of operations and cash flows for the interim periods presented. Fourth Amended and Restated Certificate of Incorporation On February 1, 2016, the Company amended its certificate of incorporation such that the total authorized capital stock of the Company consisted of 30,000,000 shares of common stock, par value $0.0001 per share, 3,278,938 shares of Class B-1 preferred stock, $0.0001 par value per share, 326,557 shares of Class B-2 preferred stock, $0.0001 par value per share, 2,365,020 shares of Class C preferred stock, $0.0001 par value per share, 3,105,000 shares of Class D preferred stock, $0.0001 par value per share and 1,000,000 shares of preferred stock, $0.0001 par value per share. Additionally, the Company effected a stock split whereby each outstanding share of common stock and Class B-1, B-2, C and D preferred stock was converted into 1.38 shares of common stock and Class B-1, B-2, C and D preferred stock, respectively. All share and per share information presented in these condensed consolidated financial statements and accompanying footnotes has been retroactively adjusted to reflect the stock split. Fifth Amended and Restated Certificate of Incorporation On February 17, 2016, the Company amended its certificate of incorporation such that the total authorized capital stock of the Company consisted of 100,000,000 shares of common stock, par value $0.0001 per share, and 10,000,000 shares of preferred stock, $0.0001 par value per share. Recent Accounting Pronouncements In August 2014, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) 2014-15, Presentation of Financial Statements — Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern , requiring management to evaluate whether events or conditions could impact an entity’s ability to continue as a going concern for at least one year after the date that the financial statements are issued and to provide disclosures if necessary. Disclosures will be required if conditions give rise to substantial doubt and the type of disclosure will be determined based on whether management’s plans will be able to alleviate the substantial doubt. The ASU will be effective for the first annual period ending after December 15, 2016. The Company does not believe the adoption of this ASU will have a material effect on the Company’s consolidated financial statements. In June 2014, the FASB issued ASU No. 2014-12, Compensation—Stock Compensation (Topic 718): Accounting for Share-Based Payments When the Terms of an Award Provide That a Performance Target Could be Achieved after the Requisite Service Period , which requires the Company to assess share-based awards with performance targets that could be achieved after the requisite service period for potential treatment as performance conditions. Under the ASU, compensation expense is to be recognized when the performance target is deemed probable and should represent the compensation expense attributable to the periods for which service has already been rendered. If the performance target is reached prior to achievement of the service period, the remaining unrecognized compensation cost should be recognized over the remaining service period. The ASU is effective for annual and interim periods beginning after December 15, 2015 with early adoption permitted. The Company has determined that adopting this ASU will not have a material effect on the Company’s consolidated financial statements. In November 2014, the FASB issued ASU No. 2014-16, Determining Whether the Host Contract in a Hybrid Financial Instrument Issued in the Form of a Share Is More Akin to Debt or to Equity (“ASU 2014-16”). The guidance requires an entity to determine the nature of the host contract by considering all stated and implied substantive terms and features of the hybrid financial instrument, weighing each term and feature on the basis of the relevant facts and circumstances (commonly referred to as the whole-instrument approach). ASU 2014-16 applies to all entities and is effective for annual periods beginning after December 15, 2015, and interim periods thereafter. Early adoption is permitted. The Company has determined that adopting this ASU will not have a material effect on the Company’s consolidated financial statements. In November 2015, the FASB issued Accounting Standards Update 2015-17, Balance Sheet Classification of Deferred Taxes . To simplify presentation, the new guidance requires that all deferred tax assets and liabilities, along with any related valuation allowance, be classified as noncurrent on the balance sheet. As a result, each jurisdiction will now only have one net noncurrent deferred tax asset or liability. The new guidance will be effective for public business entities in fiscal years beginning after December 15, 2016, including interim periods within those years. Early adoption is permitted for all entities as of the beginning of an interim or annual reporting period. The Company does not believe the adoption of this ASU will have a material effect on the Company’s consolidated financial statements. In February 2015, the FASB issued ASU 2015-2, Consolidation (Topic 810): Amendments to the Consolidation Analysis , which provides clarification regarding the guidance surrounding consolidation of certain legal entities. This guidance is effective for annual and interim periods beginning after December 15, 2015. The Company has determined that adopting this ASU will not have a material effect on the Company’s consolidated financial statements. In February 2016, the FASB issued Accounting Standards Codification (“ASC”) No. 2016-02, Leases (“ASC 842”). The guidance requires lessees to recognize almost all leases on their balance sheet as a right-of-use asset and a lease liability. For income statement purposes, the FASB retained a dual model, requiring leases to be classified as either operating or finance. Lessor accounting is similar to the current model, but updated to align with certain changes to the lessee model and the new revenue recognition standard. Existing sale-leaseback guidance, including guidance for real estate, is replaced with a new model applicable to both lessees and lessors. ASC 842 is effective for fiscal years beginning after December 15, 2018. The Company is evaluating the adoption of ASC 842, but has not determined the effects it may have on the Company’s consolidated financial statements. In March 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting (“ASU 2016-09”) , which requires the Company to recognize the income tax effects of awards in the income statement when the awards vest or are settled. ASU 2016-09 is effective for annual and interim periods beginning after December 15, 2016 with early adoption permitted. The Company is evaluating the adoption of ASU 2016-09, but has not determined the effects it may have on the Company’s consolidated financial statements. |
Collaboration and License Agree
| Collaboration and License Agreements | 3 Months Ended |
| Mar. 31, 2016 | |
| Collaboration and License Agreements | |
| Collaboration and License Agreements | 3. Collaboration and License Agreements Nationwide Children’s Hospital In October 2013 (the “Effective Date”), the Company entered into an Exclusive License Agreement (the “Nationwide License”), which agreement was amended and restated in its entirety (the “Amended and Restated Nationwide License”) on January 13, 2016 (the “Amended and Restated Nationwide License Effective Date”), with Nationwide Children’s Hospital (“NCH”). Under the terms of the agreement, NCH granted the Company an exclusive, non-transferable, worldwide license to certain patents held by NCH for the therapy and treatment of SMA. The Company was also provided a license to the Investigational New Drug application (“IND”) for AVXS-101 (the “Product Candidate”) and was provided the right to become sponsor of the IND after completion of the Phase 1 clinical trial. On October 14, 2015, the Company exercised the option and, as of November 6, 2015, the Company became the sponsor of the IND. Additionally, the Company was provided the U.S. marketing rights to the product upon receipt of regulatory approval. The Company is responsible for all clinical trial costs incurred by NCH that are not covered by third party research grants and the Company committed to spend not less than $9,400,000 for the development of the Product Candidate during the first eight years of the Nationwide License. As of March 31, 2016, this obligation was achieved, and was acknowledged by NCH to have been achieved as of the date of the Amended and Restated Nationwide License. As consideration for the Nationwide License, on the Effective Date, the Company agreed to issue 331,053 shares of its common stock to NCH (the “Up-front Shares”), which represented 3% of the Company’s outstanding capital stock on a fully-diluted basis. Additionally, the Company agreed to make certain future milestone payments totaling $125,000 upon achievement of certain regulatory milestones and agreed to reimburse NCH, upon the successful completion of an additional financing round, the amount of $83,163 to cover past patent costs and expenses incurred by NCH prior to the Effective Date. The Nationwide License provided that for the 30 day period immediately following FDA approval of the Biologics License Application (“BLA”), NCH had the option (if it owns at least 50% of the shares issued to it pursuant to the agreement) to sell all, but not less than all, of the Up-front Shares back to the Company at a per share price equal to two times the price per share of preferred stock sold by the Company in its Class B Financing ($2.47 per share), with such consideration to be paid by the Company in four equal quarterly installments (the “Royalty Option”).The Royalty Option expired upon the effectiveness of the Amended and Restated Nationwide Agreement. Accordingly, NCH no longer has the right to sell the Up-front Shares issued upon the Effective Date of the original NCH License back to the Company under any circumstances. Prior to the expiration of the Royalty Option, because NCH could require the Company to repurchase the Up-front Shares upon exercise of the Royalty Option, the fair value of the Up-front Shares as of the Effective Date of the Nationwide License was reflected as redeemable common stock on the Company’s consolidated balance sheet as of December 31, 2015. The rights granted to the Company under the Nationwide License represent distinct components that need to be combined with other licensed intellectual property and know how in order to complete the clinical development of AVXS-101 and have no alternative future use. Additionally, the Company did not acquire any employees in connection with the Nationwide License. As a result of the above, and the early-stage nature of the licensed technology, the Company concluded that the acquired rights did not meet the definition of a business, and therefore the Company accounted for the Nationwide License as an asset acquisition and expensed such amounts as research and development expense. In addition to the above, the Nationwide License granted NCH anti-dilution protection on its 3% equity ownership of the Company’s outstanding capital stock on a fully-diluted basis until such time that the Company achieved a $50,000,000 market capitalization, and required that the Company file a registration statement for an initial public offering of its common stock within ninety days of the Effective Date. Failure to do so would constitute a material breach of the agreement and would allow NCH to terminate the Nationwide License. On January 13, 2014, the Nationwide License was amended to delay the requirement to file a registration statement to within 30 calendar days of NCH providing written notice to the Company that NCH had dosed the seventh patient in the Phase 1 clinical trial, and the anti-dilution protection afforded to NCH was extended until such time as the Company achieved a $100,000,000 market capitalization. In consideration for this amendment the Company agreed to pay to NCH an aggregate of $50,000, with $20,000 payable on the amendment date, and three $10,000 payments payable within ten days of the dosing of each of the first, second and fourth patients in the Phase 1 clinical trial. In August 2014, the Company issued an additional 86,725 common shares to NCH pursuant to the anti-dilution provisions of the Nationwide License. The Company recognized additional research and development expense of $214,298, in its consolidated financial statements for the year ended December 31, 2014, representing the fair value of the additional common shares that were granted. In March 2015, the Company issued an additional 34,463 common shares to NCH, and in May 2015, the Company issued an additional 3,802 common shares to NCH in each case pursuant to the anti-dilution provisions of the Nationwide License. The Company recognized additional research and development expense of $473,164 in its consolidated financial statements for the year ended December 31, 2015, representing the fair value of the additional common shares that were granted. NCH’s anti-dilution protection right expired on May 29, 2015 upon achievement by the Company of a $100,000,000 market capitalization. On April 23, 2015, the Nationwide License was again amended to further extend the filing deadline for a registration statement to December 31, 2015 in exchange for a $100,000 payment by the Company to NCH. Such amount is included in research and development expense for the year ended December 31, 2015. On October 14, 2015, the Company and NCH entered into an amendment to the Nationwide License. The amendment permits the Company to submit to the FDA for the transfer of the IND and associated regulatory filing to the Company and for the Company to become the sponsor of such IND. Contemporaneous with the execution of this amendment, the Company and NCH submitted the requisite documents to the FDA to initiate the transfer process. On November 6, 2015, the FDA approved the Company’s sponsorship of such IND. The Amended and Restated Nationwide License commenced on the Amended and Restated Nationwide License Effective Date and terminates upon the expiration of the royalty term for the Product Candidate in each country in which it is sold. The Amended and Restated Nationwide License can also be terminated (i) by the Company for convenience at any time after the first anniversary of the Effective Date upon six months prior written notice, (ii) by either party in the event of a material uncured breach upon thirty days written notice, (iii) by NCH upon the bankruptcy/insolvency of the Company, and (iv) by NCH if it is sued by the Company for anything other than a suit brought in response to any suit brought by NCH regarding the validity or enforceability of the NCH patents. REGENXBIO Inc. License On March 21, 2014, the Company entered into a License Agreement (the “ReGenX License”) with ReGenX Biosciences, LLC, predecessor to REGENXBIO Inc. (“ReGenX”). Under the terms of the agreement, ReGenX granted the Company an exclusive, non-transferable, worldwide license to utilize ReGenX’s proprietary adeno-associated virus (“AAV”) gene delivery platform for the treatment of SMA, by in vivo gene therapy, using ReGenX’s AAV9 gene delivery vector. As consideration for the ReGenX License, the Company agreed to make a $2,000,000 up-front payment (the “ReGenX Up-front Payment”). $1,000,000 of such amount was paid in March 2014 and the remaining $1,000,000 was paid by June 30, 2014. Additionally, the Company agreed to pay potential future milestones aggregating $12,250,000, and a mid-single to low-double digit royalty on net sales, if any, of the Company’s Product Candidate, subject to reduction in specified circumstances; and lower mid-double digit percentages of any sublicense fees the Company receives from sublicenses of the licensed intellectual property rights. The Company also agreed to pay an annual maintenance fee on each anniversary of the effective date of the ReGenX License. Annual maintenance fee of $12,500 are included in research and development expenses for the three months ended March 31, 2016. The rights granted to the Company under the ReGenX License represent distinct components that need to be combined with other licensed intellectual property and know how in order to complete the clinical development of AVXS-101. Additionally, the Company did not acquire any employees or manufacturing capabilities in connection with the ReGenX License. As a result, the Company accounted for the ReGenX License as an asset acquisition. The ReGenX License term continues until the last valid patent claim expires or lapses in all countries of the world. Additionally, the Company may terminate the ReGenX License at any time upon a specified period notice and ReGenX may terminate upon the breach or insolvency of the Company, if the Company is greater than a specified number of days late in paying money due under the agreement or if the Company, its affiliates, or sublicensees challenges the ReGenX patents subject to the ReGenX License. Either party may terminate the ReGenX License for material breach if such breach is not cured within a specified number of days. Asklepios Biopharmaceutical, Inc. License On May 29, 2015, the Company and Asklepios Biopharmaceutical, Inc. (“AskBio”) entered into a Non-Exclusive License Agreement (the “AskBio License”). Under the terms of the AskBio License, AskBio granted the Company a non-exclusive, non-transferable, worldwide license to certain patents and know how held by AskBio. As consideration for the AskBio License, the Company agreed to make a $1,000,000 up-front payment (the “AskBio Up-front Payment”), with $300,000 of such amount payable within thirty days of the effective date of the AskBio License, $300,000 payable within thirty days following the dosing of the first patient in the ongoing Phase 1 clinical trial of the Product Candidate and $400,000 payable upon the dosing of the ninth patient in the clinical trial, as well as potential future milestone payments aggregating $9,600,000, and a tiered royalty on net sales, if any, of the Company’s Product Candidate, on a country-by-country basis, starting at percentages in the low-single digits and increasing to mid-single digits. These royalty rates are subject to potential reduction in specified circumstances, including, in the event the Company exercises its option to make a specified one-time royalty option fee payment to AskBio. The Company must also pay AskBio a low double digit percentage of all consideration the Company receives from any sublicense of the licensed technology. Additionally, the Company agreed to pay an annual maintenance fee of $50,000 on each anniversary of the effective date of the AskBio License. The rights granted to the Company under the AskBio License represent distinct components that need to be combined with other licensed intellectual property and know-how in order to complete the clinical development of AVXS-101. Additionally, the Company did not acquire any employees or manufacturing capabilities in connection with the AskBio License. As a result, the Company accounted for the AskBio License as an asset acquisition. Annual maintenance fees of $12,500 are included in research and development expense for the three months ended March 31, 2016. The AskBio License term continues until the last valid patent claim expires or lapses in all countries of the world. Additionally, the Company may terminate the AskBio License at any time upon six months’ notice and AskBio may terminate upon the breach or insolvency of the Company. Additionally, AskBio may terminate the AskBio License in the event the Company (i) researches, develops or commercializes any AAV-based treatment for hemophilia or (ii) undergoes a change in control or is otherwise acquired by a third party that researches, develops or commercializes any AAV-based treatment for hemophilia, in each case, until April 1, 2019 unless such change in control is first approved by AskBio, such approval not to be unreasonably withheld if the party in control following such a change of control event agrees to additional restrictive measures as reasonably proposed by AskBio in its sole discretion prior to such change of control respecting the use of the AskBio licensed technology. |
Property and Equipment, Net
| Property and Equipment, Net | 3 Months Ended |
| Mar. 31, 2016 | |
| Property and Equipment, Net | |
| Property and Equipment, Net | 4. Property and Equipment, Net Property and equipment, net, consists of the following: March 31, 2016 December 31, 2015 Office furniture and equipment $ $ Construction in progress — Property and equipment, gross Less: accumulated depreciation ) ) Property and equipment, net $ $ Depreciation expense was $11,045 and $1,350 for the three months ended March 31, 2016 and 2015, respectively. |
Accrued Expenses
| Accrued Expenses | 3 Months Ended |
| Mar. 31, 2016 | |
| Accrued Expenses | |
| Accrued Expenses | 5. Accrued Expenses Accrued expenses consist of the following: March 31, 2016 December 31, 2015 Accrued manufacturing development costs $ $ Accrued payroll, bonus and deferred compensation Accrued professional fees Accrued issuance costs for planned initial public offering Accrued employee expenses — Accrued clinical trial costs Accrued license maintenance fees Accrued severance — Other Accrued Expenses $ $ |
Accrued Indemnification Obligat
| Accrued Indemnification Obligation | 3 Months Ended |
| Mar. 31, 2016 | |
| Accrued Indemnification Obligation | |
| Accrued Indemnification Obligation | 6. Accrued Indemnification Obligation In January 2014, the Company issued 2,334,391 shares of restricted common stock to a member of its Board of Directors pursuant to a consulting agreement for scientific advisory services to be performed by the director on behalf of the Company. In connection with the restricted stock purchase agreement, the Company agreed to indemnify this consultant for any taxes, interest, fines, penalties or other costs and expenses that the consultant may incur in the future should the Internal Revenue Service (“IRS”) succeed in a tax determination that the stock price paid by consultant (which was par value) was lower than the fair market value of the stock on the date of grant. The indemnification term is in effect for six years after the due date of the tax return for the year in which the stock was issued (April 15, 2021). In connection with the preparation of the Company’s audited consolidated financial statements for the year ended December 31, 2014, the Company determined that the per share fair value of the Company’s common stock on January 28, 2014, the grant date, was $1.51. As a result, the Company intends to issue the consultant an amended Form 1099 for the 2014 tax year reflecting an aggregate fair value of the restricted stock grant of $3,535,419. Due to the indemnity obligation contained in the consultant’s restricted stock purchase agreement, the Company will ultimately be required to reimburse the consultant for the taxes he will pay following receipt of the amended Form 1099 and the amendment of the consultant’s 2014 personal income tax return. As a result, the Company has concluded that payment of such indemnity is probable as of December 31, 2015 and March 31, 2016. Additionally, the Company intends to gross-up such indemnification payment for the tax that will be payable by the consultant on the indemnity payment. As a result, the Company has accrued $4,080,500 at March 31, 2016 and December 31, 2015, representing the Company’s best estimate of the ultimate tax indemnification and gross-up payment to be made to the consultant. |
Stock-Based Compensation
| Stock-Based Compensation | 3 Months Ended |
| Mar. 31, 2016 | |
| Stock-Based Compensation | |
| Stock-Based Compensation | 7. Stock-Based Compensation In May 2014, the Company’s Board of Directors adopted the 2014 Stock Plan (the “2014 Plan”), and in January 2016 the Company adopted the 2016 Equity Incentive Plan (the “2016 Plan” and, together with the 2014 Plan, the “Plans”). The 2016 Plan became effective on February 10, 2016, or the IPO Date. On and after the IPO Date, no additional stock awards will be granted under the 2014 Plan. The Board may amend or suspend the 2016 Plan at any time, although no such action may materially impair the rights under any then-outstanding award without the holder’s consent. The Company will obtain stockholder approval for any amendments to the 2016 Plan as required by law. No incentive stock options may be granted under the 2016 Plan after the tenth anniversary of the effective date of the 2016 Plan. Initially, the aggregate number of shares of the Company’s common stock that may be issued pursuant to stock awards under the 2016 Plan is 4,339,451 shares, which is the sum of (1) 2,400,000 new shares, plus (2) the number of shares reserved for issuance under the 2014 Plan on the IPO Date, plus (3) any shares subject to outstanding stock awards that would have otherwise been returned to the 2014 Plan. Additionally, the number of shares of the Company’s common stock reserved for issuance under the 2016 Plan will automatically increase on January 1 of each year, beginning on January 1, 2017 and continuing through and including January 1, 2026, by 4.0% of the total number of shares of the Company’s capital stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares determined by the Board. The maximum number of shares that may be issued pursuant to the exercise of incentive stock options under the 2016 Plan is 8,678,902 shares. The following table summarizes stock option activity under the Plans for the three months ended March 31, 2016: Weighted Weighted Average Number of Shares Average Exercise Price Remaining Contractual Life (Years) Aggregate Intrinsic Value (a) Outstanding at December 31, 2015 $ $ Granted $ Exercised — $ — Cancelled or forfeited — $ — Outstanding at March 31, 2016 $ $ Exercisable at March 31, 2016 $ $ Exercisable and expected to vest at March 31, 2016 $ $ (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the estimated fair value of the common stock for the options that were in-the-money at March 31, 2016 and December 31, 2015. During the three months ended March 31, 2016 and 2015, there were no stock options exercised. As of March 31, 2016 and December 31, 2015, there was $14,228,435 and $12,942,684, respectively, of unrecognized stock-based compensation expense related to stock option awards that is expected to be recognized over a weighted-average period of 1.5 and 1.9 years, respectively. The Company has recorded aggregate stock-based compensation expense related to the issuance of stock option awards under the Plans in the consolidated statements of operations and comprehensive loss as follows: Three Months Ended March 31, 2016 2015 Research and development $ $ General and administrative $ $ Stock Options Granted to Employees The weighted-average grant date fair value of options granted during the three months ended March 31, 2016 and 2015 was $11.16 and $1.47, respectively, on the date of grant using the Black-Scholes option-pricing model with the following weighted-average assumptions: Three Months Ended March 31, 2016 2015 Expected volatility % % Risk-free interest rate % % Expected terms (in years) Expected dividend yield % % Valuation of Common Stock. Prior to the IPO, the Company estimated the fair value of common stock underlying stock option awards at the grant date of the award. Valuation estimates were prepared by management in accordance with the framework of the AICPA Practice Guide, with the assistance of independent third party valuations, and are approved by the Company’s Board of Directors. Prior to the IPO, the Company’s valuations of its common stock as of October 9, 2013, January 28, 2014, March 7, 2014, March 31, 2014, June 30, 2014, August 11, 2014, September 30, 2014, December 31, 2014, March 31, 2015, June 30, 2015, September 30, 2015 and December 31, 2015 were based on a number of objective and subjective factors, including external market conditions affecting the Company’s industry sector, the prices at which the Company sold shares of its common and preferred stock, and the likelihood of achieving a liquidity event such as an initial public offering. Refer to the Annual Report for details of each valuation. Restricted Stock Granted to Non-Employees In January 2014, the Company issued 2,334,391 shares of restricted common stock to Dr. Brian Kaspar pursuant to a consulting agreement for scientific advisory services. Of these shares, 583,597 common shares were vested at the time of grant and the remaining restricted shares are scheduled to vest in the amount of 25% per year on the second, third and fourth anniversary of the grant date pursuant to a restricted stock purchase agreement, which became effective upon the effectiveness of the consulting agreement. In January 2016, the Company entered into an employment agreement with Dr. Kaspar. Upon the effectiveness of the employment agreement, Dr. Kaspar’s 1,759,794 unvested shares granted pursuant to the restricted stock purchase agreement vested in full. As a result of the vesting in full of the remainder of this award in January 2016 the Company recorded $10,370,762 for the three months ended March 31, 2016 to research and development expense as compared to $4,036,564 recognized related to the grant for the three months ended March, 31, 2015. |
Net Loss Per Common Share
| Net Loss Per Common Share | 3 Months Ended |
| Mar. 31, 2016 | |
| Net Loss Per Common Share | |
| Net Loss Per Common Share | 8. Net Loss Per Common Share Net Loss per Common Share Basic net loss per common share is computed using the weighted average number of common shares outstanding during the period. Diluted net loss per common share is computed using the sum of the weighted average number of common shares outstanding during the period and, if dilutive, the weighted average number of potential shares of common stock, including the assumed exercise of stock options, stock warrants and unvested restricted common stock. The Company applies the two-class method to calculate its basic and diluted net loss per share attributable to common stockholders, as its preferred stock and common stock are participating securities. The two-class method is an earnings allocation formula that treats a participating security as having rights to earnings that otherwise would have been available to common stockholders. However, the two-class method does not impact the net loss per share of common stock as the Company was in a net loss position for each of the periods presented and preferred stockholders do not participate in losses. For the three months ended March 31, 2016 and 2015, the following potentially dilutive securities have been excluded from the computations of diluted weighted-average shares outstanding because the effect would be anti-dilutive: Three Months Ended March 31, 2016 2015 Stock options issued and outstanding Stock warrants Unvested restricted common stock — Amounts in the table above reflect the common stock equivalents of the noted instruments. The following table summarizes the calculation of the basic and diluted net loss per common share: Three Months Ended March 31, 2016 2015 Numerator: Net loss $ ) $ ) Denominator: Weighted-average basic and diluted common shares Basic and diluted net loss per common share from continuing operations $ ) $ ) |
Separation Agreement
| Separation Agreement | 3 Months Ended |
| Mar. 31, 2016 | |
| Separation Agreement | |
| Separation Agreement | 9. Separation Agreement On April 22, 2015, the Company’s prior chief executive officer (the “Prior CEO”), ceased to be an employee of the Company. In connection with the termination of his employment, the Company agreed to pay the Prior CEO the amount of $535,000, consisting of a $500,000 severance benefit (the “Severance”) and $35,000 of accrued and unused vacation (the “Vacation Pay”). The Severance is to be paid over a 12-month period in equal monthly installments. However, under the terms of the separation agreement, in the event the Prior CEO were to resign, or be removed from, his service on the Company’s Board, then (i) 50% of the then unpaid portion of Severance due to the Prior CEO would be paid to him in a lump sum within 30 days from the termination of his service on the Board and (ii) the remaining 50% of the unpaid Severance due to the Prior CEO would be paid in equal installments over the lesser of (a) six months or (b) the remainder of the original 12-month period. The Vacation Pay was paid in a lump sum in May 2015. In June 2015, the Prior CEO’s service on the Board terminated. In connection with the transactions described above, the Company recorded a charge of $535,000 in its consolidated statements of operations for the year ended December 31, 2015. Such amount is included within general and administrative expense in the consolidated statement of operations. Additionally, the Company agreed to fully accelerate the vesting of 53,820 unvested stock options held by the Prior CEO at the time of the termination of his employment. The Company determined that the acceleration of vesting was a Type III modification pursuant to ASC 718. Therefore, the Company recognized the incremental fair value of the awards as of the modification date and recognized the amount immediately since the awards did not require further service. In connection with this modification, the Company recorded a charge of $503,502 in its consolidated statement of operations for the year ended December 31, 2015. Such amount is included within general and administrative expense. At December 31, 2015, the Company had $153,846 related to the Severance recorded as an accrued liability, which was paid in the first quarter of 2016. |
Commitments and Contingencies
| Commitments and Contingencies | 3 Months Ended |
| Mar. 31, 2016 | |
| Commitments and Contingencies | |
| Commitments and Contingencies | 10. Commitments and Contingencies Operating Lease In March 2016, the Company entered into a lease agreement, which expires in September 2026, for approximately 48,529 square feet of warehouse and office space Libertyville, Illinois. A portion of the warehouse space will house the Company’s manufacturing operation. The lease agreement provides for annual escalation in rent payments during the lease term. The lease agreement provides the Company with a one-time right to terminate the lease effective as of the last day of the ninety-sixth full calendar month of the lease subject to a termination fee. The Company is amortizing the escalation in rental payments on a straight-line basis over the term of the lease. Future minimum lease payments are as follows: Year ending December 31, 2016 $ 2017 2018 Thereafter Total $ License Agreements See Note 3 for information regarding licenses entered into by the Company. These agreements may require the Company to make future payments relating to sublicense fees, milestone fees and royalties on future sales, if any, of the Product Candidate. Guarantees and Indemnifications The Company has accrued $4,080,500 at March 31, 2016 and December 31, 2015, representing the Company’s best estimate of the ultimate tax indemnification and gross-up payment to be made to a former consultant pursuant to a tax indemnification granted to such consultant in connection with a restricted common stock grant. Additionally, in the normal course of business, the Company has entered into agreements that contain a variety of representations and provide for general indemnification. The Company’s exposure under these agreements is unknown because it involves claims that may be made against the Company in the future. To date, the Company has not paid any claims or been required to defend any action related to these indemnification obligations. As of March 31, 2016 and December 31, 2015, the Company did not have any material indemnification claims related to these agreements that were probable or reasonably possible and consequently has not recorded any related liabilities. Litigation Lawsuits may be asserted against the Company in the normal course of business. Based on information currently available, management believes that the disposition of any matters will not have a materially adverse effect on the financial position, results of operations or cash flows of the Company. |
Taxes
| Taxes | 3 Months Ended |
| Mar. 31, 2016 | |
| Taxes | |
| Taxes | 11. Taxes Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Valuation allowances are provided if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. We have evaluated the positive and negative evidence bearing upon the realizability of our deferred tax assets, including our net operating losses. Based on our history of operating losses, we believe that it is more likely than not that the benefit of our deferred tax assets will not be realized. Accordingly, we have provided a full valuation allowance for deferred tax assets as of March 31, 2016 and December 31, 2015. |
Summary of Significant Accoun17
| Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
| Mar. 31, 2016 | |
| Summary of Significant Accounting Policies | |
| Basis of Presentation | Basis of Presentation The condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) for interim financial reporting and the rules and regulations of the U.S. Securities and Exchange Commission (“SEC”) and, therefore, do not include all information and footnote disclosures normally included in the annual consolidated financial statements. In the opinion of management, the unaudited condensed consolidated financial statements reflect all adjustments of a normal recurring nature that are necessary for a fair presentation of the results for the interim periods presented. Interim results are not necessarily indicative of results for a full year. The December 31, 2015 condensed consolidated balance sheet data contained within this Form 10-Q was derived from audited consolidated financial statements included in the Company’s Form 10-K for the year ended December 31, 2015 (“Annual Report”), but does not include all disclosures required by accounting principles generally accepted in the United States. |
| Use of Estimates | Use of Estimates The preparation of the condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. In the Company’s opinion, the accompanying unaudited condensed consolidated financial statements include all normal recurring adjustments necessary for a fair presentation of the Company’s financial position, results of operations and cash flows for the interim periods presented. |
| Recent Accounting Pronouncements | Recent Accounting Pronouncements In August 2014, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) 2014-15, Presentation of Financial Statements — Going Concern (Subtopic 205-40): Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern , requiring management to evaluate whether events or conditions could impact an entity’s ability to continue as a going concern for at least one year after the date that the financial statements are issued and to provide disclosures if necessary. Disclosures will be required if conditions give rise to substantial doubt and the type of disclosure will be determined based on whether management’s plans will be able to alleviate the substantial doubt. The ASU will be effective for the first annual period ending after December 15, 2016. The Company does not believe the adoption of this ASU will have a material effect on the Company’s consolidated financial statements. In June 2014, the FASB issued ASU No. 2014-12, Compensation—Stock Compensation (Topic 718): Accounting for Share-Based Payments When the Terms of an Award Provide That a Performance Target Could be Achieved after the Requisite Service Period , which requires the Company to assess share-based awards with performance targets that could be achieved after the requisite service period for potential treatment as performance conditions. Under the ASU, compensation expense is to be recognized when the performance target is deemed probable and should represent the compensation expense attributable to the periods for which service has already been rendered. If the performance target is reached prior to achievement of the service period, the remaining unrecognized compensation cost should be recognized over the remaining service period. The ASU is effective for annual and interim periods beginning after December 15, 2015 with early adoption permitted. The Company has determined that adopting this ASU will not have a material effect on the Company’s consolidated financial statements. In November 2014, the FASB issued ASU No. 2014-16, Determining Whether the Host Contract in a Hybrid Financial Instrument Issued in the Form of a Share Is More Akin to Debt or to Equity (“ASU 2014-16”). The guidance requires an entity to determine the nature of the host contract by considering all stated and implied substantive terms and features of the hybrid financial instrument, weighing each term and feature on the basis of the relevant facts and circumstances (commonly referred to as the whole-instrument approach). ASU 2014-16 applies to all entities and is effective for annual periods beginning after December 15, 2015, and interim periods thereafter. Early adoption is permitted. The Company has determined that adopting this ASU will not have a material effect on the Company’s consolidated financial statements. In November 2015, the FASB issued Accounting Standards Update 2015-17, Balance Sheet Classification of Deferred Taxes . To simplify presentation, the new guidance requires that all deferred tax assets and liabilities, along with any related valuation allowance, be classified as noncurrent on the balance sheet. As a result, each jurisdiction will now only have one net noncurrent deferred tax asset or liability. The new guidance will be effective for public business entities in fiscal years beginning after December 15, 2016, including interim periods within those years. Early adoption is permitted for all entities as of the beginning of an interim or annual reporting period. The Company does not believe the adoption of this ASU will have a material effect on the Company’s consolidated financial statements. In February 2015, the FASB issued ASU 2015-2, Consolidation (Topic 810): Amendments to the Consolidation Analysis , which provides clarification regarding the guidance surrounding consolidation of certain legal entities. This guidance is effective for annual and interim periods beginning after December 15, 2015. The Company has determined that adopting this ASU will not have a material effect on the Company’s consolidated financial statements. In February 2016, the FASB issued Accounting Standards Codification (“ASC”) No. 2016-02, Leases (“ASC 842”). The guidance requires lessees to recognize almost all leases on their balance sheet as a right-of-use asset and a lease liability. For income statement purposes, the FASB retained a dual model, requiring leases to be classified as either operating or finance. Lessor accounting is similar to the current model, but updated to align with certain changes to the lessee model and the new revenue recognition standard. Existing sale-leaseback guidance, including guidance for real estate, is replaced with a new model applicable to both lessees and lessors. ASC 842 is effective for fiscal years beginning after December 15, 2018. The Company is evaluating the adoption of ASC 842, but has not determined the effects it may have on the Company’s consolidated financial statements. In March 2016, the FASB issued ASU No. 2016-09, Compensation—Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting (“ASU 2016-09”) , which requires the Company to recognize the income tax effects of awards in the income statement when the awards vest or are settled. ASU 2016-09 is effective for annual and interim periods beginning after December 15, 2016 with early adoption permitted. The Company is evaluating the adoption of ASU 2016-09, but has not determined the effects it may have on the Company’s consolidated financial statements. |
Property and Equipment, Net (Ta
| Property and Equipment, Net (Tables) | 3 Months Ended |
| Mar. 31, 2016 | |
| Property and Equipment, Net | |
| Summary of property and equipment, net | March 31, 2016 December 31, 2015 Office furniture and equipment $ $ Construction in progress — Property and equipment, gross Less: accumulated depreciation ) ) Property and equipment, net $ $ |
Accrued Expenses (Tables)
| Accrued Expenses (Tables) | 3 Months Ended |
| Mar. 31, 2016 | |
| Accrued Expenses | |
| Schedule of accrued expenses | March 31, 2016 December 31, 2015 Accrued manufacturing development costs $ $ Accrued payroll, bonus and deferred compensation Accrued professional fees Accrued issuance costs for planned initial public offering Accrued employee expenses — Accrued clinical trial costs Accrued license maintenance fees Accrued severance — Other Accrued Expenses $ $ |
Stock-Based Compensation (Table
| Stock-Based Compensation (Tables) | 3 Months Ended |
| Mar. 31, 2016 | |
| Stock-Based Compensation | |
| Summary of Company's stock option activity | Weighted Weighted Average Number of Shares Average Exercise Price Remaining Contractual Life (Years) Aggregate Intrinsic Value (a) Outstanding at December 31, 2015 $ $ Granted $ Exercised — $ — Cancelled or forfeited — $ — Outstanding at March 31, 2016 $ $ Exercisable at March 31, 2016 $ $ Exercisable and expected to vest at March 31, 2016 $ $ (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the estimated fair value of the common stock for the options that were in-the-money at March 31, 2016 and December 31, 2015. |
| Summary of aggregate stock-based compensation recorded in the consolidated statements of operations and comprehensive loss | Three Months Ended March 31, 2016 2015 Research and development $ $ General and administrative $ $ |
| Summary of Company's grant date fair value and assumptions used in determining fair value of stock options | Three Months Ended March 31, 2016 2015 Expected volatility % % Risk-free interest rate % % Expected terms (in years) Expected dividend yield % % |
Net Loss Per Common Share (Tabl
| Net Loss Per Common Share (Tables) | 3 Months Ended |
| Mar. 31, 2016 | |
| Net Loss Per Common Share | |
| Potentially dilutive securities excluded from computations of diluted weighted-average shares outstanding | Three Months Ended March 31, 2016 2015 Stock options issued and outstanding Stock warrants Unvested restricted common stock — |
| Summary of the basic and diluted net loss per common share | Three Months Ended March 31, 2016 2015 Numerator: Net loss $ ) $ ) Denominator: Weighted-average basic and diluted common shares Basic and diluted net loss per common share from continuing operations $ ) $ ) |
Commitments and Contingencies (
| Commitments and Contingencies (Tables) | 3 Months Ended |
| Mar. 31, 2016 | |
| Commitments and Contingencies | |
| Future minimum lease payments | Year ending December 31, 2016 $ 2017 2018 Thereafter Total $ |
Background (Details)
| Background (Details) - USD ($) | Mar. 03, 2016 | Feb. 17, 2016 |
| Class B-1 Preferred Stock | ||
| Initial Public Offering | ||
| Shares converted (in shares) | 3,278,938 | |
| Class B-2 Preferred Stock | ||
| Initial Public Offering | ||
| Shares converted (in shares) | 326,557 | |
| Class C Preferred Stock | ||
| Initial Public Offering | ||
| Shares converted (in shares) | 2,365,020 | |
| Class D Preferred Stock | ||
| Initial Public Offering | ||
| Shares converted (in shares) | 3,105,000 | |
| Common Stock | IPO | ||
| Initial Public Offering | ||
| Shares issued (in shares) | 4,750,000 | |
| Share price (in dollars per share) | $ 20 | |
| Net proceeds | $ 88,350,000 | |
| Common Stock | Over-Allotment Option | ||
| Initial Public Offering | ||
| Shares issued (in shares) | 527,941 | |
| Share price (in dollars per share) | $ 20 | |
| Net proceeds | $ 9,800,000 |
Summary of Significant Accoun24
| Summary of Significant Accounting Policies (Details) | Feb. 01, 2016$ / sharesshares | Mar. 31, 2016$ / sharesshares | Feb. 17, 2016$ / sharesshares | Dec. 31, 2015$ / sharesshares |
| Commitments and Contingencies | ||||
| Common stock, shares authorized (in shares) | shares | 30,000,000 | 100,000,000 | 100,000,000 | 22,080,000 |
| Common stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | $ 0.0001 |
| Class B-1 Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Preferred stock, shares authorized (in shares) | shares | 3,278,938 | 0 | 3,278,938 | |
| Preferred stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | |
| Class B-2 Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Preferred stock, shares authorized (in shares) | shares | 326,557 | 326,557 | 326,557 | |
| Preferred stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | |
| Class C Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Preferred stock, shares authorized (in shares) | shares | 2,365,020 | 0 | 2,365,020 | |
| Preferred stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | |
| Class D Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Preferred stock, shares authorized (in shares) | shares | 3,105,000 | 0 | 3,105,000 | |
| Preferred stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | |
| Undesignated Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Preferred stock, shares authorized (in shares) | shares | 1,000,000 | 10,000,000 | 10,000,000 | 1,000,000 |
| Preferred stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | $ 0.0001 |
| Common Stock | ||||
| Commitments and Contingencies | ||||
| Stock split ratio | 1.38 | |||
| Preferred Stock | Class B-1 Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Stock split ratio | 1.38 | |||
| Preferred Stock | Class B-2 Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Stock split ratio | 1.38 | |||
| Preferred Stock | Class C Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Stock split ratio | 1.38 | |||
| Preferred Stock | Class D Preferred Stock | ||||
| Commitments and Contingencies | ||||
| Stock split ratio | 1.38 |
Collaboration and License Agr25
| Collaboration and License Agreements - Nationwide Children's Hospital (Details) | Jan. 13, 2016 | Jan. 13, 2014USD ($) | May. 31, 2015shares | Mar. 31, 2015shares | Aug. 31, 2014USD ($)shares | Oct. 31, 2013USD ($)installmentshares | May. 31, 2015USD ($) | Dec. 31, 2015USD ($) | Dec. 31, 2014USD ($) | May. 29, 2015USD ($) | Apr. 23, 2015USD ($) | Mar. 07, 2014$ / shares |
| Collaborative Arrangement | Nationwide Children's Hospital | ||||||||||||
| Collaboration and License Agreements | ||||||||||||
| Minimum commitment to spend | $ 9,400,000 | |||||||||||
| Minimum commitment to spend, period ( in years) | 8 years | |||||||||||
| Shares issued (in shares) | shares | 3,802 | 34,463 | 86,725 | 331,053 | ||||||||
| Ownership percentage (as a percent) | 3.00% | |||||||||||
| Aggregate potential future milestones | $ 125,000 | |||||||||||
| Patent costs | $ 83,163 | |||||||||||
| Period following FDA approval of BLA to sell shares (in days) | 30 days | |||||||||||
| Minimum ownership percentage of shares issued pursuant to the agreement (as a percent) | 50.00% | |||||||||||
| Ratio of the per share sale price to the price per share of preferred stock sold in Class B Financing | 2 | |||||||||||
| Quarterly installments, number | installment | 4 | |||||||||||
| Market capitalization, threshold | $ 100,000,000 | $ 50,000,000 | ||||||||||
| Maximum period to file registration statement upon effective date (in days) | 90 days | |||||||||||
| Maximum period to file registration statement upon written notice of dosing seventh patient (in days) | 30 days | |||||||||||
| Amendment, consideration, payable | $ 50,000 | $ 100,000 | ||||||||||
| Amendment, consideration, amount due on amendment date | 20,000 | |||||||||||
| Amendment, consideration, amount due within ten days of the first patient dosing | 10,000 | |||||||||||
| Amendment, consideration, amount due within ten days of the second patient dosing | 10,000 | |||||||||||
| Amendment, consideration, amount due within ten days of the fourth patient dosing | $ 10,000 | |||||||||||
| Fair value of shares granted | $ 214,298 | $ 473,164 | ||||||||||
| Market capitalization, achieved | $ 100,000,000 | |||||||||||
| Termination, notice period (in months) | 6 months | |||||||||||
| Termination, event of a material uncured breach, notice period (in days) | 30 days | |||||||||||
| Research and development expenses | Collaborative Arrangement | Nationwide Children's Hospital | ||||||||||||
| Collaboration and License Agreements | ||||||||||||
| Amendment, consideration, expensed | $ 100,000 | |||||||||||
| Stock issuance expense | $ 473,164 | $ 214,298 | ||||||||||
| Class B-1 Preferred Stock | Preferred Stock | ||||||||||||
| Collaboration and License Agreements | ||||||||||||
| Share price (in dollars per share) | $ / shares | $ 2.47 |
Collaboration and License Agr26
| Collaboration and License Agreements - REGENXBIO Inc. License (Details) - Collaborative Arrangement - ReGenX - USD ($) | 1 Months Ended | 3 Months Ended | |||
| Jun. 30, 2014 | Mar. 31, 2014 | Mar. 31, 2016 | Mar. 24, 2014 | Mar. 21, 2014 | |
| Collaboration and License Agreements | |||||
| Up-front payment, agreed-upon amount | $ 2,000,000 | ||||
| Up-front payment, paid | $ 1,000,000 | $ 1,000,000 | |||
| Aggregate potential future milestones | $ 12,250,000 | ||||
| Research and development expenses | |||||
| Collaboration and License Agreements | |||||
| Maintenance fee | $ 12,500 | ||||
Collaboration and License Agr27
| Collaboration and License Agreements - Asklepios Biopharmaceutical, Inc. License (Details) - Collaborative Arrangement - Asklepios Biopharmaceutical, Inc. - USD ($) | May. 29, 2015 | Mar. 31, 2016 |
| Collaboration and License Agreements | ||
| Up-front payment, agreed-upon amount | $ 1,000,000 | |
| Up-front payment, portion payable within 30 days of the effective date | 300,000 | |
| Up-front payment, portion payable within 30 days following dosing of the first patient | 300,000 | |
| Up-front payment, portion payable upon dosing of the ninth patient in a clinical trial | 400,000 | |
| Aggregate potential future milestones | 9,600,000 | |
| Annual maintenance fee | $ 50,000 | |
| Termination, notice period (in months) | 6 months | |
| Research and development expenses | ||
| Collaboration and License Agreements | ||
| Maintenance fee | $ 12,500 |
Property and Equipment, Net - T
| Property and Equipment, Net - Tabular Disclosure (Details) - USD ($) | Mar. 31, 2016 | Dec. 31, 2015 |
| Property and Equipment, Net | ||
| Property and equipment, gross | $ 5,972,625 | $ 252,514 |
| Less: accumulated depreciation | (27,969) | (16,924) |
| Property and equipment, net | 5,944,656 | 235,590 |
| Office furniture and equipment | ||
| Property and Equipment, Net | ||
| Property and equipment, gross | 350,637 | $ 252,514 |
| Construction in progress | ||
| Property and Equipment, Net | ||
| Property and equipment, gross | $ 5,621,988 |
Property and Equipment, Net - D
| Property and Equipment, Net - Depreciation Expense (Details) - USD ($) | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Property and Equipment, Net | ||
| Depreciation expense | $ 11,045 | $ 1,350 |
Accrued Expenses (Details)
| Accrued Expenses (Details) - USD ($) | Mar. 31, 2016 | Dec. 31, 2015 |
| Accrued Expenses | ||
| Accrued manufacturing development costs | $ 892,756 | $ 513,606 |
| Accrued payroll, bonus and deferred compensation | 794,676 | 761,556 |
| Accrued professional fees | 395,759 | 348,165 |
| Accrued issuance costs for planned initial public offering | 290,848 | 371,043 |
| Accrued employee expenses | 102,536 | |
| Accrued clinical trial costs | 103,093 | 93,621 |
| Accrued license maintenance fees | 43,311 | 68,311 |
| Accrued severance | 153,846 | |
| Other | 183,337 | 126,869 |
| Accrued Expenses | $ 2,806,316 | $ 2,437,017 |
Accrued Indemnification Oblig31
| Accrued Indemnification Obligation (Details) - USD ($) | Jan. 28, 2014 | Mar. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 |
| Accrued Indemnification Obligation | ||||
| Accrued indemnification obligation | $ 4,080,500 | $ 4,080,500 | ||
| Restricted Stock | Director | ||||
| Accrued Indemnification Obligation | ||||
| Shares granted (in shares) | 2,334,391 | |||
| Share price (in dollars per share) | $ 1.51 | |||
| Aggregate fair value | $ 3,535,419 | |||
| Tax indemnification | Director | ||||
| Accrued Indemnification Obligation | ||||
| Indemnification term (in years) | 6 years | |||
| Accrued indemnification obligation | $ 4,080,500 | $ 4,080,500 |
Stock-Based Compensation - Gene
| Stock-Based Compensation - General Information (Details) - 2016 Equity Incentive Plan - shares | Feb. 10, 2016 | Jan. 01, 2026 |
| Stock-Based Compensation | ||
| Plan expiration period (in years) | 10 years | |
| Common stock that may be issued pursuant to stock awards (in shares) | 4,339,451 | |
| Common stock that may be issued pursuant to stock awards, new shares (in shares) | 2,400,000 | |
| Annual automatic increase percentage (as a percent) | 4.00% | |
| Forecast | ||
| Stock-Based Compensation | ||
| Common stock that may be issued pursuant to stock awards (in shares) | 8,678,902 |
Stock-Based Compensation - Stoc
| Stock-Based Compensation - Stock Option Activity (Details) - Employee Stock Options - USD ($) | 3 Months Ended | 12 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | Dec. 31, 2015 | |
| Number of Shares | |||
| Outstanding at the beginning of the period (in shares) | 1,748,877 | ||
| Granted (in shares) | 338,740 | ||
| Exercised (in shares) | 0 | 0 | |
| Outstanding at the end of the period (in shares) | 2,087,617 | 1,748,877 | |
| Weighted Average Exercise Price | |||
| Outstanding at the beginning of the period (in dollars per share) | $ 15.04 | ||
| Granted (in dollars per share) | 19.27 | ||
| Outstanding at the end of the period (in dollars per share) | $ 15.72 | $ 15.04 | |
| Weighted Average | |||
| Remaining Contractual Life (Years) | 9 years 3 months | 9 years 4 months 17 days | |
| Aggregate Intrinsic Value (in dollars) | $ 24,058,862 | $ 8,224,947 | |
| Exercisable and Vested and Expected to Vest | |||
| Number of Shares - exercisable (in shares) | 173,521 | ||
| Weighted Average Exercise Price - exercisable (in dollars per share) | $ 2.92 | ||
| Remaining Contractual Life (Years) - exercisable | 8 years 11 months 5 days | ||
| Aggregate Intrinsic Value - exercisable (in dollars) | $ 4,922,093 | ||
| Number of Shares - vested and expected to vest (in shares) | 2,037,937 | ||
| Weighted Average Exercise Price - vested and expected to vest (in dollars per share) | $ 16.04 | ||
| Remaining Contractual Life (Years) - vested and expected to vest | 9 years 3 months | ||
| Aggregate Intrinsic Value - vested and expected to vest (in dollars) | $ 22,828,336 | ||
Stock-Based Compensation - St34
| Stock-Based Compensation - Stock Options Exercised (Details) - shares | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Employee Stock Options | ||
| Stock-Based Compensation | ||
| Exercised (in shares) | 0 | 0 |
Stock-Based Compensation - Unre
| Stock-Based Compensation - Unrecognized Stock-based Compensation Expense (Details) - Employee Stock Options - USD ($) | 3 Months Ended | 12 Months Ended |
| Mar. 31, 2016 | Dec. 31, 2015 | |
| Stock-Based Compensation | ||
| Unrecognized stock-based compensation expense | $ 14,228,435 | $ 12,942,684 |
| Unrecognized stock-based compensation expense, weighted average recognition period (in years) | 1 year 6 months | 1 year 10 months 24 days |
Stock-Based Compensation - Aggr
| Stock-Based Compensation - Aggregate Stock-based Compensation Expense (Details) - Employee Stock Options - USD ($) | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Stock-Based Compensation | ||
| Aggregate stock-based compensation expense | $ 3,172,785 | $ 95,230 |
| Research and development expenses | ||
| Stock-Based Compensation | ||
| Aggregate stock-based compensation expense | 1,089,580 | 42,942 |
| General and administrative expenses | ||
| Stock-Based Compensation | ||
| Aggregate stock-based compensation expense | $ 2,083,205 | $ 52,288 |
Stock-Based Compensation - Weig
| Stock-Based Compensation - Weighted Average Grant Date Fair Value (Details) - $ / shares | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Employee Stock Options | ||
| Stock-Based Compensation | ||
| Weighted-average grant date fair value of options granted during the period (in dollars per share) | $ 11.16 | $ 1.47 |
Stock-Based Compensation - We38
| Stock-Based Compensation - Weighted-average Assumptions (Details) | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Stock-Based Compensation | ||
| Expected volatility (as a percent) | 90.00% | 86.94% |
| Risk-free interest rate (as a percent) | 1.57% | 1.16% |
| Expected term (in years) | 6 years 29 days | 4 years 2 months 9 days |
| Expected dividend yield (as a percent) | 0.00% | 0.00% |
Stock-Based Compensation - Rest
| Stock-Based Compensation - Restricted Stock Granted to Non-Employees - Board Member (Details) - Restricted Stock - shares | Jan. 28, 2014 | Jan. 31, 2016 |
| Dr. Brian Kaspar | ||
| Stock-Based Compensation | ||
| Shares granted (in shares) | 2,334,391 | 1,759,794 |
| Shares vested (in shares) | 583,597 | 1,759,794 |
| Vesting period (in years) | 4 years | |
| Dr. Brian Kaspar | Shares vesting upon grant | ||
| Stock-Based Compensation | ||
| Vesting percentage (as a percent) | 25.00% | |
| Dr. Brian Kaspar | Shares vesting on second anniversary | ||
| Stock-Based Compensation | ||
| Vesting percentage (as a percent) | 25.00% | |
| Dr. Brian Kaspar | Shares vesting on third anniversary | ||
| Stock-Based Compensation | ||
| Vesting percentage (as a percent) | 25.00% | |
| Dr. Brian Kaspar | Shares vesting on fourth anniversary | ||
| Stock-Based Compensation | ||
| Vesting percentage (as a percent) | 25.00% | |
| Director | ||
| Stock-Based Compensation | ||
| Shares granted (in shares) | 2,334,391 |
Stock-Based Compensation - Re40
| Stock-Based Compensation - Restricted Stock Granted to Non-Employees - Chief Scientific Officer (Details) - Dr. Brian Kaspar - Restricted Stock - USD ($) | Jan. 28, 2014 | Jan. 31, 2016 | Mar. 31, 2016 | Mar. 31, 2015 |
| Stock-Based Compensation | ||||
| Shares granted (in shares) | 2,334,391 | 1,759,794 | ||
| Shares vested (in shares) | 583,597 | 1,759,794 | ||
| Research and development expenses | ||||
| Stock-Based Compensation | ||||
| Aggregate stock-based compensation expense | $ 10,370,762 | $ 4,036,564 |
Net Loss Per Common Share - Dil
| Net Loss Per Common Share - Dilutive Securities (Details) - shares | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Antidilutive securities not included: | ||
| Antidilutive securities | 2,397,837 | 2,369,467 |
| Employee Stock Options | ||
| Antidilutive securities not included: | ||
| Antidilutive securities | 2,087,617 | 455,400 |
| Stock warrants | ||
| Antidilutive securities not included: | ||
| Antidilutive securities | 310,220 | 163,273 |
| Restricted Stock | ||
| Antidilutive securities not included: | ||
| Antidilutive securities | 1,750,794 | |
Net Loss Per Common Share - Cal
| Net Loss Per Common Share - Calculation (Details) - USD ($) | 3 Months Ended | |
| Mar. 31, 2016 | Mar. 31, 2015 | |
| Numerator: | ||
| Net loss | $ (20,836,107) | $ (5,453,204) |
| Denominator: | ||
| Weighted-average basic and diluted common shares (in shares) | 16,774,718 | 6,895,185 |
| Basic and diluted net loss per common share from continuing operations (in dollars per share) | $ (1.24) | $ (0.79) |
Separation Agreement - Severanc
| Separation Agreement - Severance and Vacation Pay (Details) - USD ($) | Apr. 22, 2015 | May. 31, 2015 | Dec. 31, 2015 |
| Separation Agreement | |||
| Accrued severance | $ 153,846 | ||
| Chief Executive Officer | |||
| Separation Agreement | |||
| Accrued severance and vacation | $ 535,000 | ||
| Accrued severance | 500,000 | 153,846 | |
| Accrued vacation | $ 35,000 | ||
| Severance payment period (in months) | 12 months | 12 months | |
| Lump sum payment percentage (as a percent) | 50.00% | ||
| Lump sum payment period after termination (in days) | 30 days | ||
| Equal installments payment percentage (as a percent) | 50.00% | ||
| Equal installments payment period after termination (in months) | 6 months | ||
| General and administrative expenses | Chief Executive Officer | |||
| Separation Agreement | |||
| Severance benefit and vacation pay charge | $ 535,000 |
Separation Agreement - Unvested
| Separation Agreement - Unvested Stock Options (Details) - USD ($) | Apr. 22, 2015 | Dec. 31, 2015 |
| Separation Agreement | ||
| Accrued severance | $ 153,846 | |
| Chief Executive Officer | ||
| Separation Agreement | ||
| Accrued severance | $ 500,000 | 153,846 |
| Chief Executive Officer | Employee Stock Options | ||
| Separation Agreement | ||
| Accelerated vesting (in shares) | 53,820 | |
| Chief Executive Officer | Employee Stock Options | General and administrative expenses | ||
| Separation Agreement | ||
| Accelerated vesting charge (in dollars) | $ 503,502 |
Commitments and Contingencies -
| Commitments and Contingencies - Operating Lease - Area Leased (Details) | Mar. 31, 2016ft² |
| Office and warehouse space in Libertyville, Illinois | |
| Operating Leases | |
| Approximate size of office space (in square feet) | 48,529 |
Commitments and Contingencies46
| Commitments and Contingencies - Operating Lease - Minimum Lease Payments (Details) - Office and warehouse space in Libertyville, Illinois | Mar. 31, 2016USD ($) |
| Operating Leases | |
| 2,016 | $ 252,007 |
| 2,017 | 258,307 |
| 2,018 | 264,765 |
| Thereafter (2019 and thereafter) | 2,024,919 |
| Total | $ 2,799,998 |
Commitments and Contingencies47
| Commitments and Contingencies - Guarantees and Indemnifications (Details) - USD ($) | Mar. 31, 2016 | Dec. 31, 2015 |
| Accrued Indemnification Obligation | ||
| Accrued indemnification obligation | $ 4,080,500 | $ 4,080,500 |
| Director | Tax indemnification | ||
| Accrued Indemnification Obligation | ||
| Accrued indemnification obligation | $ 4,080,500 | $ 4,080,500 |