Stockholders' Equity | 5. Stockholders’ Equity Preferred Stock The Company is authorized to issue up to 10,000,000 shares of preferred stock, $0.00001 par value per share, with no shares outstanding as of September 30, 2018 and December 31, 2017. The Board of Directors is authorized to designate the terms and conditions of any preferred stock the Company may issue without further action by the stockholders of the Company. Common Stock The Company is authorized to issue up to 300,000,000 shares of common stock, $0.00001 par value per share. In February 2014, the Company entered into a stock purchase agreement with one of its founders. The agreement provided for the purchase of 1,000,000 shares of the Company’s common stock at a price per share of $0.01 in exchange for future services to be rendered to the Company as measured by certain performance criteria. The shares were subject to a repurchase option and were to vest in two tranches of 500,000 shares each, upon achievement of the performance target or upon a triggering event as defined. The Company determined that the fair value of the unrecognized expense was $168,000 at February 20, 2014, the grant date. In May 2015, the Company repurchased 633,810 of these shares at a purchase price of $0.00001 per share. In connection with the repurchase, the Company entered into an amendment to the stock purchase agreement to provide that the remaining 366,190 shares will continue to vest in two tranches of 183,095 shares each, upon achievement of the performance target or upon a triggering event as defined. The pro rata grant date fair value of the unrecognized expense is $62,000. In October 2015, a triggering event became probable of occurrence and was deemed achieved in October 2016; therefore, the Company recorded $31,000 of stock-based compensation expense through December 31, 2016. No similar expense was recognized during the three and nine months ended September 30, 2018 and 2017. The Company will continue to reassess at each reporting period whether it is probable that the performance target will be achieved, and if and when it is deemed probable, the Company will begin to record compensation expense using the fair value to determine stock-based compensation expense in its financial statements over the period the Company estimates the performance target will actually be achieved. On June 20, 2016, the Company entered into the Equity Distribution Agreement (the “Distribution Agreement”) with Maxim Group LLC (“Maxim”) pursuant to which the Company was able to offer and sell, from time to time, through Maxim up to 3,748,726 shares of its common stock (the “Maxim Offering”). Effective July 26, 2017, the Distribution Agreement terminated automatically in accordance with the terms thereof. All shares of common stock offered and sold in the Maxim Offering were issued pursuant to the Company’s shelf registration statement on Form S-3 (File No. 333-212134) filed with the SEC on June 20, 2016, as amended by Amendment No. 1 thereto filed with the SEC on July 26, 2016, and declared effective on July 26, 2016 (the “Shelf Registration Statement”) and the prospectus relating to the Maxim Offering that forms a part of the registration statement on Form S-3. The registration statement on Form S-3 was declared effective by the SEC on July 26, 2016. From the inception of the Distribution Agreement through July 26, 2017, the Company sold 1,267,237 shares of its common stock under the Distribution Agreement resulting in net proceeds to the Company of $1.6 million, after deducting the sales agent’s commission. On August 24, 2016, the Company entered into a common stock purchase agreement (the “Purchase Agreement”), pursuant to which Aspire Capital Fund, LLC (“Aspire Capital”) was committed to purchase up to an aggregate of $12.5 million of shares of the Company’s common stock over the 30-month term of the Purchase Agreement. Upon execution of the Purchase Agreement, the Company issued and sold 333,333 shares of common stock (the “Initial Shares”) a t a price per share of $1.50, for an aggregate purchase price of $500,000 440,000, which has been recorded as deferred financing costs, with a prorated portion charged to additional paid-in-capital for the Initial Shares, with the remainder of the costs amortized over the term of the Purchase Agreement as there was no guarantee that additional shares would be sold under the Purchase Agreement. Pursuant to the terms of the Purchase Agreement, the Company was able to, from time to time and subject to certain limitations, direct Aspire Capital to purchase shares of the Company’s common stock using pricing formulas based on average prevailing market prices around the time of each sale. Additionally, the Company and Aspire may not effect any sales of shares of the Company's common stock under the Purchase Agreement during the continuance of an event of default or on any trading day that the closing sale price of its common stock is less than $0.40 per share (subject to adjustment for stock dividends, splits, combinations or similar transactions or events). Effective June 19, 2017, the Company terminated the Purchase Agreement, resulting in a write off of the remaining capitalized deferred financing costs relating to the Purchase Agreement of $337,000. From the inception of the Purchase Agreement through June 19, 2017, the Company sold 1,476,991 shares of its common stock under the Purchase Agreement resulting in aggregate gross proceeds to the Company of $2.0 million, in addition to the Initial Shares and the Commitment Shares. On February 8, 2017, the Company entered into a Stock Purchase Agreement (the “SPA”) with PoC Capital, LLC (“PoC”), pursuant to which, among other things, the Company issued to PoC 1,286,173 shares of its common stock. Under the terms of the SPA, PoC has agreed to fund $1.8 million in study costs associated with certain clinical studies, including a planned proof-of-concept trial in patients with Glycogen Storage Disease type Ia. Any study costs in excess of that amount will be the Company’s sole responsibility. The Company has accounted for the $1.8 million as a prepaid expense on the balance sheet. During the three and nine months ended September 30, 2018, the Company recorded amortization expense of $64,000 and $541,000, respectively, in clinical study costs related to the SPA with PoC. During the three and nine months ended September 30, 2017, the Company recorded amortization expense of $249,000 and $648,000, respectively. The remaining prepaid balance of $265,000 was recorded on the balance sheet as of September 30, 2018. On June 14, 2017, the Company entered into a securities purchase agreement (the “Securities Purchase Agreement”) with certain accredited investors (the “Purchasers”) pursuant to which the Company sold an aggregate of 3,749,783 shares (the “Shares”) of its common stock in a registered direct offering (the “Registered Direct Offering”). The Shares were offered by the Company pursuant to the Shelf Registration Statement. In a concurrent private placement, the Company also agreed, pursuant to the Securities Purchase Agreement, to issue and sell to each of the Purchasers a warrant to purchase 0.75 shares of common stock (the “Warrants”) for each share of common stock purchased by a Purchaser in the Registered Direct Offering (the “Private Placement” and, together with the Registered Direct Offering, the “Offerings”). The exercise price of the Warrants is $1.30 per share, subject to adjustment as provided therein, and will be exercisable beginning on December 19, 2017 through December 19, 2022. The combined purchase price for one Share and one Warrant to purchase 0.75 shares of common stock in the Offerings was $1.15. The closing of the Offerings occurred on June 19, 2017. The aggregate net proceeds from the Offerings, after deducting the placement agents’ fees and offering expenses, were $3.9 million. On September 28, 2017, the Company entered into a purchase agreement (the “Registered Offering Purchase Agreement”) pursuant to which, on September 29, 2017, the Company sold to Lincoln Park Capital Fund, LLC (“LPC”) 701,282 shares of common stock (the “LPC Initial Shares”) at a price of approximately $1.78 per share for an aggregate purchase price of $1.3 million, pursuant to the Shelf Registration Statement and the prospectus supplement thereto dated September 28, 2017. On September 28, 2017, the Company also entered a purchase agreement (the “Commitment Purchase Agreement”) and a registration rights agreement (the “Registration Rights Agreement”) with LPC, pursuant to which the Company has the right to sell to LPC up to $15.0 million in shares of common stock, subject to certain limitations and conditions set forth in the Commitment Purchase Agreement. Upon the satisfaction of the conditions in the Commitment Purchase Agreement (the “Commencement”) the Company will have the right, from time to time at its sole discretion over the 30-month period from and after the Commencement, to direct LPC to purchase up to 75,000 shares of common stock on any business day (subject to certain limitations contained in the Commitment Purchase Agreement), with such amounts increasing based on certain threshold prices set forth in the Commitment Purchase Agreement; however, not to exceed $1.0 million in total purchase proceeds per purchase date. The purchase price of shares of common stock that the Company elects to sell to LPC pursuant to the Commitment Purchase Agreement will be based on the market prices of the common stock at the time of such purchases as set forth in the Commitment Purchase Agreement. In addition to regular purchases, as described above, the Company may also direct LPC to purchase additional amounts as accelerated purchases or as additional purchases if the closing sale price of the common stock is not below certain threshold prices, as set forth in the Commitment Purchase Agreement. In all instances, the Company may not sell shares of its common stock to LPC under the Commitment Purchase Agreement if it would result in LPC beneficially owning more than 4.99% of the Common Stock. As consideration for LPC’s commitment to purchase shares of common stock pursuant to the Commitment Purchase Agreement, the Company issued to LPC 100,000 shares of common stock (the “LPC Commitment Shares”) From inception of the Commitment Purchase Agreement through December 31, 2017, Commitment Purchase On December 11, 2017, the Company closed an underwritten public offering of 5,900,000 shares of its common stock, including 769,565 shares sold pursuant to the underwriters’ full exercise of their option to purchase additional shares to cover over-allotments, pursuant to the Shelf Registration Statement at a public offering price of $2.50 per share, for total net proceeds of $13.4 million after deducting underwriting discounts and commissions and other offering expenses. On February 6, 2018, the Company completed an underwritten public offering of common stock pursuant to the Shelf Registration Statement (the “February 2018 Offering”). In the February 2018 Offering, the Company sold 12,650,000 shares of the Company’s common stock at a public offering price of $5.00 per share of common stock. Upon the closing of the February 2018 Offering on February 6, 2018, the Company received net proceeds of $58.7 million, after deducting underwriting discounts, commissions and other offering expenses. On June 11, 2018, the Company completed an underwritten public offering of common stock (the “June 2018 Offering”) pursuant to the Shelf Registration Statement and a registration statement on Form S-3MEF (File No. 333-225479) filed pursuant to Rule 462(b) of the Securities Act of 1933, as amended. In the June 2018 Offering, the Company sold 8,625,000 shares of the Company’s common stock at a public offering price of $9.00 per share of common stock. Upon the closing of the June 2018 Offering on June 11, 2018, the Company received net proceeds of $72.3 million, after deducting underwriting discounts, commissions and other offering expenses. On September 25, 2018, the Company completed an underwritten public offering of common stock (the “September 2018 Offering”) pursuant to the Company’s universal shelf registration statement on Form S-3 (File No. 333-226133), filed with the SEC on July 11, 2018 and declared effective on July 19, 2018. In the September 2018 Offering, the Company sold 9,500,000 shares of the Company’s common stock at a public offering price of $18.50 per share. Upon the closing of the September 2018 Offering on September 25, 2018, the Company received net proceeds of $165.0 million, after deducting underwriting discounts, commissions and other offering expenses. During the nine months ended September 30, 2018 and 2017, and in accordance with the Company’s 2014 Employee Stock Purchase Plan (the “ESPP”), the Company issued an aggregate of 72,597 and 19,887 shares of its common stock to certain employees, respectively. |