Cover Page
Cover Page - shares | 9 Months Ended | |
Sep. 30, 2020 | Oct. 29, 2020 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2020 | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q3 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity Registrant Name | ZIOPHARM ONCOLOGY INC | |
Entity Central Index Key | 0001107421 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Trading Symbol | ZIOP | |
Entity Emerging Growth Company | false | |
Entity Small Business | false | |
Entity Common Stock, Shares Outstanding | 214,291,057 | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-33038 | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 84-1475642 | |
Entity Address, Address Line One | One First Avenue | |
Entity Address, Address Line Two | Parris Building 34 | |
Entity Address, Address Line Three | Navy Yard Plaza | |
Entity Address, City or Town | Boston | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02129 | |
City Area Code | 617 | |
Local Phone Number | 259-1970 |
BALANCE SHEETS
BALANCE SHEETS - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 135,471 | $ 79,741 |
Receivables | 5,428 | 3,330 |
Prepaid expenses and other current assets | 14,172 | 22,421 |
Total current assets | 155,071 | 105,492 |
Property and equipment, net | 7,577 | 1,110 |
Right of use asset | 2,370 | 2,272 |
Deposits | 130 | 130 |
Other non-current assets | 746 | 110 |
Total assets | 165,894 | 109,114 |
Current liabilities: | ||
Accounts payable | 2,619 | 906 |
Accrued expenses | 15,836 | 10,846 |
Lease liability - current portion | 866 | 774 |
Total current liabilities | 19,321 | 12,526 |
Lease liability - noncurrent portion | 1,655 | 1,578 |
Total liabilities | 20,976 | 14,104 |
Commitments and contingencies (Note 6) | ||
Stockholders' equity: | ||
Common stock, $0.001 par value; 250,000,000 shares authorized; 214,165,690 and 181,803,320 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively | 214 | 182 |
Additional paid-in capital | 886,033 | 778,953 |
Accumulated deficit | (741,329) | (684,125) |
Total stockholders' equity | 144,918 | 95,010 |
Total liabilities and stockholders' equity | $ 165,894 | $ 109,114 |
BALANCE SHEETS (Parenthetical)
BALANCE SHEETS (Parenthetical) - $ / shares | Sep. 30, 2020 | Dec. 31, 2019 |
Statement of Financial Position [Abstract] | ||
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 250,000,000 | 250,000,000 |
Common stock, shares issued | 214,165,690 | 181,803,320 |
Common stock, shares outstanding | 214,165,690 | 181,803,320 |
STATEMENTS OF OPERATIONS
STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Operating expenses: | ||||
Research and development | $ 13,968 | $ 8,641 | $ 38,725 | $ 28,115 |
General and administrative | 6,353 | 4,807 | 18,862 | 13,707 |
Total operating expenses | 20,321 | 13,448 | 57,587 | 41,822 |
Loss from operations | (20,321) | (13,448) | (57,587) | (41,822) |
Other income, net | 6 | 203 | 383 | 523 |
Non-cash inducement warrant expense | (60,751) | (60,751) | ||
Net loss | $ (20,315) | $ (73,996) | $ (57,204) | $ (102,050) |
Basic and diluted net loss per share | $ (0.10) | $ (0.43) | $ (0.27) | $ (0.62) |
Weighted average common shares outstanding used to compute basic and diluted net loss per share | 212,837,367 | 170,613,712 | 208,497,410 | 164,053,029 |
STATEMENTS OF CHANGES IN STOCKH
STATEMENTS OF CHANGES IN STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Common Stock | Additional Paid In Capital | Accumulated Deficit |
Balance at Dec. 31, 2018 | $ 85,564 | $ 161 | $ 651,732 | $ (566,329) |
Balance (in shares) at Dec. 31, 2018 | 161,066,136 | |||
Stock-based compensation | 4,741 | 4,741 | ||
Exercise of employee stock options | 1,020 | 1,020 | ||
Exercise of employee stock options (in shares) | 352,652 | |||
Restricted stock awards | 1,000 | $ 1 | 999 | |
Restricted stock awards (in shares) | 1,408,536 | |||
Issuance of common stock upon exercise of warrants, net | 52,499 | $ 18 | 52,481 | |
Issuance of common stock upon exercise of warrants, net (in shares) | 17,803,031 | |||
Issuance of inducement warrants | 60,751 | 60,751 | ||
Cancelled restricted common stock (in shares) | (74,599) | |||
Restricted stock buy-back at vesting to cover taxes | (370) | (370) | ||
Restricted stock buy-back at vesting to cover taxes (in shares) | (165,178) | |||
Issuance of common stock in connection with at the market offering, net | 2,971 | $ 1 | 2,970 | |
Issuance of common stock in connection with at the market offering, net (in shares) | 639,442 | |||
Net loss | (102,050) | (102,050) | ||
Balance at Sep. 30, 2019 | 106,126 | $ 181 | 774,324 | (668,379) |
Balance (in shares) at Sep. 30, 2019 | 181,030,020 | |||
Balance at Jun. 30, 2019 | 61,995 | $ 162 | 656,216 | (594,383) |
Balance (in shares) at Jun. 30, 2019 | 162,477,963 | |||
Stock-based compensation | 1,486 | 1,486 | ||
Exercise of employee stock options | 420 | 420 | ||
Exercise of employee stock options (in shares) | 94,584 | |||
Restricted stock awards (in shares) | 15,000 | |||
Issuance of common stock upon exercise of warrants, net | 52,499 | $ 18 | 52,481 | |
Issuance of common stock upon exercise of warrants, net (in shares) | 17,803,031 | |||
Issuance of inducement warrants | 60,751 | 60,751 | ||
Issuance of common stock in connection with at the market offering, net | 2,971 | $ 1 | 2,970 | |
Issuance of common stock in connection with at the market offering, net (in shares) | 639,442 | |||
Net loss | (73,996) | (73,996) | ||
Balance at Sep. 30, 2019 | 106,126 | $ 181 | 774,324 | (668,379) |
Balance (in shares) at Sep. 30, 2019 | 181,030,020 | |||
Balance at Dec. 31, 2019 | 95,010 | $ 182 | 778,953 | (684,125) |
Balance (in shares) at Dec. 31, 2019 | 181,803,320 | |||
Stock-based compensation | 5,393 | 5,393 | ||
Exercise of employee stock options | $ 43 | 43 | ||
Exercise of employee stock options (in shares) | 22,916 | 22,916 | ||
Restricted stock awards | $ 1 | (1) | ||
Restricted stock awards (in shares) | 555,900 | |||
Cancelled restricted common stock (in shares) | (141,230) | |||
Issuance of common stock in connection with a public offering, net | $ 88,661 | $ 29 | 88,632 | |
Issuance of common stock in connection with a public offering, net (in shares) | 29,110,111 | |||
Issuance of common stock in connection with at the market offering, net | 13,015 | $ 2 | 13,013 | |
Issuance of common stock in connection with at the market offering, net (in shares) | 2,814,673 | |||
Net loss | (57,204) | (57,204) | ||
Balance at Sep. 30, 2020 | 144,918 | $ 214 | 886,033 | (741,329) |
Balance (in shares) at Sep. 30, 2020 | 214,165,690 | |||
Balance at Jun. 30, 2020 | 163,414 | $ 214 | 884,214 | (721,014) |
Balance (in shares) at Jun. 30, 2020 | 214,150,940 | |||
Stock-based compensation | 1,792 | 1,792 | ||
Exercise of employee stock options | 27 | 27 | ||
Exercise of employee stock options (in shares) | 14,750 | |||
Net loss | (20,315) | (20,315) | ||
Balance at Sep. 30, 2020 | $ 144,918 | $ 214 | $ 886,033 | $ (741,329) |
Balance (in shares) at Sep. 30, 2020 | 214,165,690 |
STATEMENTS OF CHANGES IN STOC_2
STATEMENTS OF CHANGES IN STOCKHOLDERS' EQUITY (Parenthetical) $ in Millions | 9 Months Ended |
Sep. 30, 2020USD ($) | |
Market Offering [Member] | |
Issuance of common stock, commissions and expenses | $ 0.4 |
Public Offering [Member] | |
Issuance of common stock, commissions and expenses | $ 5.9 |
STATEMENTS OF CASH FLOWS
STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
Cash flows from operating activities: | ||
Net loss | $ (57,204) | $ (102,050) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation | 708 | 453 |
Stock-based compensation | 5,393 | 5,741 |
Non-cash inducement warrant expense | 60,751 | |
(Increase) decrease in: | ||
Receivables | (2,098) | (1,577) |
Prepaid expenses and other current assets | 8,249 | (3,047) |
Right of use asset | (98) | |
Deposits | (2) | |
Other noncurrent assets | (636) | 9,432 |
Increase (decrease) in: | ||
Accounts payable | 1,713 | 219 |
Accrued expenses | 3,828 | 810 |
Deferred rent | 17 | |
Lease liabilities | 168 | 7 |
Net cash used in operating activities | (39,977) | (29,246) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (6,012) | (184) |
Net cash used in investing activities | (6,012) | (184) |
Cash flows from financing activities: | ||
Repurchase of common stock | (370) | |
Proceeds from exercise of stock options | 43 | 1,020 |
Issuance of common stock in connection with a public offering, net | 88,661 | 2,971 |
Issuance of common stock in connection with at the market offerings, net | 13,015 | 52,499 |
Net cash provided by financing activities | 101,719 | 56,120 |
Net increase in cash and cash equivalents, and restricted cash | 55,730 | 26,690 |
Cash and cash equivalents, and restricted cash, beginning of period | 79,741 | 61,729 |
Cash and cash equivalents, and restricted cash, end of period | 135,471 | 88,419 |
Supplementary disclosure of cash flow information: | ||
Compensation paid in restricted stock, gross | $ 1,000 | |
Accounts included in accounts payable and accrued expenses related to property and equipment | $ 1,163 |
Business
Business | 9 Months Ended |
Sep. 30, 2020 | |
Accounting Policies [Abstract] | |
Business | 1. Business Overview ZIOPHARM Oncology, Inc., which is referred to herein as “ZIOPHARM,” or the “Company,” is a biopharmaceutical company seeking to develop, acquire, and commercialize, on its own or with partners, a diverse portfolio of immuno-oncology therapies. The Company’s operations to date have consisted primarily of conducting research and development and raising capital to fund those efforts. The Company’s fiscal year ends on December 31. The Company has operated at a loss since its inception in 2003 and has no recurring revenues from operations. The Company anticipates that losses will continue for the foreseeable future. As of September 30, 2020, the Company had approximately $135.5 million of cash and cash equivalents and the Company’s accumulated deficit was approximately $741.3 million. Given its current development plans, the Company anticipates cash resources will be sufficient to fund operations into mid-2022. The Basis of Presentation The accompanying unaudited interim financial statements have been prepared in accordance with the instructions to Form 10-Q It is management’s opinion that the accompanying unaudited interim financial statements reflect all adjustments (which are normal and recurring) that are necessary for a fair statement of the results for the interim periods. The unaudited interim financial statements should be read in conjunction with the audited financial statements and the notes thereto for the year ended December 31, 2019, included in the Company’s Annual Report on Form 10-K The year-end The results disclosed in the statements of operations for the three and nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for the full fiscal year 2020 Use of Estimates The preparation of financial statements in conformity with accounting principles generally accepted in the United States requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. Although the Company regularly assesses these estimates, actual results could differ from those estimates. Changes in estimates are recorded in the period in which they become known. The Company’s most significant estimates and judgments used in the preparation of its financial statements are: • Clinical trial expenses; • Collaboration agreements; • Fair value measurements of stock-based compensation; and • Income taxes Impact of COVID-19 Pandemic With the global spread of the ongoing COVID-19 , COVID-19 business and operations . The extent to which the COVID-19 pandemic impacts the Company’s business, clinical development and regulatory efforts and the value of its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements, and the effectiveness of actions taken globally to contain and treat the disease. The global economic slowdown, the overall disruption of global healthcare systems and the other risks and uncertainties associated with the COVID-19 pandemic could have a material adverse effect on the Company’s business, financial condition, results of operations and growth prospects. In addition, the Company is subject to other challenges and risks specific to its business and its ability to execute on its business plan and strategy, as well as risks and uncertainties common to companies in the biopharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of its product candidates; delays or problems in obtaining clinical supply, loss of single source suppliers or failure to comply with manufacturing regulations; identifying, acquiring or in-licensing COVID-19 Subsequent Events The Company evaluated all events and transactions that occurred after the balance sheet date through the date of this filing. During this period, the Company did not have any material subsequent events that impacted its financial statements or disclosures. |
Financings
Financings | 9 Months Ended |
Sep. 30, 2020 | |
Text Block [Abstract] | |
Financings | 2. Financings February 2020 Public Offering On February 5, 2020, the Company entered into an underwriting agreement with Jefferies, as representative of the several underwriters named therein, relating to the issuance and sale of 27,826,086 shares of its common stock. The price to the public in the offering was $3.25 per share, and the underwriters agreed to purchase the shares from the Company pursuant to the underwriting agreement at a purchase price of $3.055 per share. Under the terms of the underwriting agreement, the Company also granted the underwriters an option, exercisable for 30 days, to purchase up to an additional 4,173,912 shares of common stock at a purchase price of $3.055 per share. The offering was made pursuant to the Company’s effective registration statement on Form S-3ASR No. 333-232283) On March 10, 2020, the underwriters exercised their option to purchase an additional 1,284,025 shares. The net proceeds were approximately $3.9 million after deducting underwriting discounts and offering expenses paid by the Company. At-the-market In June 2019, the Company entered into an Open Market Sale Agreement, or sales agreement, with Jefferies LLC, as agent, or (“Jefferies”), pursuant to which the Company may offer and sell, from time to time through Jefferies, shares of its common stock having an aggregate offering price of up to $ million. Shares will be sold pursuant to the Company’s effective registration statement on Form S-3ASR (File . as previously filed with the SEC. During the nine months ended September 30, 2019, the Company sold an aggregate of 639,442 shares of its common stock. The offering was made pursuant to the Company’s effective registration statement on Form S-3ASR No. 333-232283) During the three months ended September 30, 2020, there were no at-the-market sales. During the nine months ended September 30, 2020, the Company issued and sold an shares of its common stock under the sales agreement for aggregate net proceeds of m November 2018 Private Placement and 2019 Inducement Warrants On November 11, 2018, the Company entered into a securities purchase agreement with certain institutional and accredited investors pursuant to which it sold an aggregate of 18,939,394 immediately separable units at a price per unit of $2.64 to such investors, for net proceeds of approximately $47.1 million. Each unit was comprised of (i) one share of our common stock, par value $0.001 per share and (ii) a warrant to purchase one share of common stock. The securities issued by the Company pursuant to the securities purchase agreement and to be issued upon exercise of the warrants were not registered under the Securities Act and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements. When issuing the units, the Company relied on the private placement exemption from registration provided by Section 4(a)(2) of the Securities Act and by Rule 506 of Registration D, promulgated thereunder and on similar exemptions under applicable state laws and filed a Form D with the SEC on November 19, 2018. On February 7, 2019, the Company filed a registration statement on Form S-3 On July 26, 2019 and September 12, 2019, the Company entered into agreements for the exercise of the warrants issued in November 2018 to purchase common stock in a private placement. Pursuant to the terms of the agreements, investors exercised warrants for an aggregate of 17,803,031 shares of common stock, at an exercise price of $3.01 per share. The Company issued new warrants to purchase up to 17,803,031 additional shares of common stock as an inducement for warrant holders to exercise their 2018 warrants early. The new warrants will become exercisable six months following the date of issuance, will expire on the fifth anniversary of the initial exercise date, and have an exercise price of $7.00 (Note 9). Proceeds from the exercise of the warrants, before deducting placement agent fees and other related expenses of $1.1 million were approximately $52.5 million. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2020 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 3. Summary of Significant Accounting Policies The Company’s significant accounting policies were identified in the Company’s Annual Report. There have been no material changes in those policies since the filing of its Annual Report except as noted below. New Accounting Pronouncements In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes 2019-12 In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement 2018-03. |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2020 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 4. Fair Value Measurements The Company accounts for its financial assets and liabilities using fair value measurements. The authoritative accounting guidance defines fair value, establishes a framework for measuring fair value under generally accepted accounting principles and enhances disclosures about fair value measurements. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. The fair value hierarchy is based on three levels of inputs, of which the first two are considered observable and the last unobservable, that may be used to measure fair value as follows: • Level 1 - Quoted prices in active markets for identical assets or liabilities. • Level 2 - Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. • Level 3 - Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. Assets and liabilities measured at fair value on a recurring basis as of September 30, 2020 and December 31, 2019 were as follows: ($ in thousands) Fair Value Measurements at Reporting Date Using Description Balance as of Quoted Prices in Significant Other Significant Assets: Cash equivalents $ 75,300 $ 75,300 $ — $ — ($ in thousands) Fair Value Measurements at Reporting Date Using Description Balance as of Quoted Prices in Significant Other Significant Assets: Cash equivalents $ 68,031 $ 68,031 $ — $ — |
Net Loss per Share
Net Loss per Share | 9 Months Ended |
Sep. 30, 2020 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 5. Net Loss per Share Basic net loss per share is computed by dividing net loss by the weighted average number of shares of common stock outstanding for the period. The Company’s potentially dilutive shares, which include outstanding common stock options, unvested restricted stock and preferred stock, have not been included in the computation of diluted net loss per share for any of the periods presented as the result would be anti-dilutive. Such potentially dilutive shares of common stock at September 30, 2020 and 2019 consisted of the following: September 30, 2020 2019 Stock options 6,572,191 4,995,549 Inducement stock options 863,333 965,000 Unvested restricted stock 1,289,389 1,569,579 Warrants 22,272,727 18,939,394 30,997,640 26,469,522 |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2020 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies License Agreements Exclusive License Agreement with PGEN Therapeutics On October 5, 2018, the Company entered into the License Agreement, with PGEN Therapeutics, Inc. or PGEN, a wholly owned subsidiary of Precigen Inc., or Precigen, which was formerly known as Intrexon Corporation. As between the Company and PGEN, the terms of the License Agreement replace and supersede the terms of: (a) that certain Exclusive Channel Partner Agreement by and between the Company and Precigen, dated January 6, 2011, as amended by the First Amendment to Exclusive Channel Partner Agreement effective September 13, 2011, the Second Amendment to the Exclusive Channel Partner Agreement effective March 27, 2015, and the Third Amendment to Exclusive Channel Partner Agreement effective June 29, 2016, which was subsequently assigned by Precigen to PGEN; (b) certain rights and obligations pursuant to that certain License and Collaboration Agreement effective March 27, 2015 between ZIOPHARM, Precigen and ARES TRADING S.A., or Ares Trading, a subsidiary of Merck KGaA, or Merck, as assigned by Precigen to PGEN, or the Ares Trading Agreement; (c) that certain License Agreement between the Company, Precigen, and MD Anderson, with an effective date of January 13, 2015, or the MD Anderson License, which was subsequently assigned by Precigen and assumed by PGEN effective as of January 1, 2018; and (d) that certain Research and Development Agreement between the Company, Precigen and MD Anderson with an effective date of August 17, 2015, or the Research and Development Agreement, and any amendments or statements of work thereto. Pursuant to the terms of the License Agreement, PGEN has granted the Company exclusive, worldwide rights to research, develop and commercialize (i) products utilizing PGEN’s RheoSwitch ® ® IL-12 (iii) T-cell sub-licensable Sleeping Beauty The Company is solely responsible for all aspects of the research, development and commercialization of the exclusively licensed products for the treatment of cancer. The Company is required to use commercially reasonable efforts to develop and commercialize IL-12 , In consideration of the licenses and other rights granted by PGEN, the Company pay s As of September 30, 2020, the Company had in prepaid expenses and other current assets. The Company did have any annual license expenses for the three and nine months ended September 2020 and The Company will also make milestone payments totaling up to an additional $52.5 million for each exclusively licensed program upon the initiation of later stage clinical trials and upon the approval of exclusively licensed products in various jurisdictions. In addition, the Company will pay PGEN tiered royalties ranging from low-single IL-12 low-single mid-single The Company is responsible for all development costs associated with each of the licensed products, other than Gorilla IL-12 PGEN will pay the Company royalties ranging from low-single mid-single During the three and nine months ended September there were no expenses for services performed by PGEN. As of September 2020 and December 31, 2019, the Company had $0 and , respectively, in accrued expenses for amounts due to PGEN. During the three and nine months ended the Company recorded an expense of $0.3 million and million, respectively, for services performed by PGEN License Agreement—The University of Texas MD Anderson Cancer Center On January 13, 2015, the Company, together with Precigen, entered into the MD Anderson License with MD Anderson (which Precigen subsequently assigned to PGEN). Pursuant to the MD Anderson License, the Company, together with Precigen, holds an exclusive, worldwide license to certain technologies owned and licensed by MD Anderson including technologies relating to novel CAR T-cell non-viral The term of the MD Anderson License expires on the later of (a) the expiration of all patents licensed thereunder, or (b) the twentieth anniversary of the date of the MD Anderson License; provided, however, that following the expiration of the term of the MD Anderson License, the Company, together with PGEN, shall have a fully-paid up, royalty free, perpetual, irrevocable and sublicensable license to use the licensed intellectual property thereunder. After ten years from the date of the MD Anderson License and subject to a 90-day non-exclusive case-by-case 180-day On August 17, 2015, the Company, Precigen and MD Anderson entered into the Research and Development, or the 2015 Agreement, to formalize the scope and process for the transfer by MD Anderson, pursuant to the terms of the MD Anderson License, of certain existing research programs and related technology rights, as well as the terms and conditions for future collaborative research and development of new and ongoing research programs. Pursuant to the 2015 Agreement, the Company, Precigen and MD Anderson formed a joint steering committee to oversee and manage the new and ongoing research programs. Under the License Agreement with PGEN, the Company and PGEN agreed that PGEN would no longer participate on the joint steering committee after the date of the License Agreement. As provided under the MD Anderson License, the Company provided funding for research and development activities in support of the research programs under the Research and Development Agreement for a period of three years and in an amount of no less than $15.0 million and no greater than $20.0 million per year. On October 22, 2019, the Company entered into an amendment to the Research and Development Agreement extending its term until December 31, 2026. During the nine months ended September 30, 2020 and 2019, the Company made no payments to MD Anderson. The net balance of cash resources on hand at MD Anderson available to offset expenses and future costs is $11.2 million, which is included in prepaid expenses and other current assets on the Company’s balance sheet at September 30, 2020. On October 22, 2019, the Company entered into the 2019 Research and Development Agreement, or the 2019 Agreement, with MD Anderson, pursuant to which the parties agreed to collaborate with respect to the Company’s Sleeping Beauty non-viral The Company will own all intellectual property developed under the 2019 Agreement and will retain all rights to intellectual property for oncology products manufactured using non-viral Sleeping Beauty non-exclusive The Company has agreed, beginning on January 1, 2021, to reimburse MD Anderson up to a total of $20.0 million for development costs incurred starting after January 1, 2021 under the 2019 Agreement. In addition, the Company will pay MD Anderson royalties on net sales of its TCR products at rates in the low single digits. The Company is required to make performance-based payments upon the successful completion of clinical and regulatory benchmarks relating to its TCR products. The aggregate potential benchmark payments are $36.5 million, of which only $3.0 million will be due prior to the first marketing approval of the Company’s TCR products. The royalty rates and benchmark payments owed to MD Anderson may be reduced upon the occurrence of certain events. The Company also agreed that it will sell the Company’s TCR products to MD Anderson at preferential prices and will sell its TCR products in Texas exclusively to MD Anderson for a limited period of time following the first commercial sale of the Company’s TCR products. In connection with the execution of the 2019 Agreement, the Company issued MD Anderson a warrant to purchase 3,333,333 shares of common stock. Refer to Note 9 – Warrants License Agreement with the National Cancer Institute On May 28, 2019, the Company entered into a patent license agreement, or the Patent License, with the National Cancer Institute, or the NCI. Pursuant to the Patent License, the Company holds an exclusive, worldwide license to certain intellectual property to develop and commercialize patient-derived (autologous), peripheral blood T-cell T-cell non-viral non-exclusive, Pursuant to the terms of the Patent License, the Company is required to pay the NCI a cash payment in the aggregate amount of $1.5 million payable in $0.5 million installments within sixty days, six-months, paid as of 20 20 On January 8, 2020, the Company entered into an amendment to the patent license agreement which expanded the TCR library to include additional TCR’s reactive to mutated KRAS and TP53. Under the amendment, the Company paid $0.6 million during the nine months ending September 30, 2020. The Company recognized $0 and $0.6 million of expense for the three and nine months ended September 30, 2020, respectively. The terms of the Patent License also require the Company to pay the NCI minimum annual royalties in the amount of $0.3 million, which amount will be reduced to $0.1 million once the aggregate minimum annual royalties paid by the Company equals $1.5 million. The first minimum annual royalty payment is payable on the date that is eighteen months following the date of the Patent License. As of September 30, 2020, the Company included a prepayment of $0.3 million related to the Patent License as prepaid expenses and other current assets on the Company’s b s On September 28, 2020, the Company entered into a second amendment to the patent license agreement which expanded the TCR library to include additional TCR’s receptors. Under the second amendment, the Company will pay the NCI an additional $0.4 million. The Company recorded $0.4 million as an accrued expense as of September 30, 2020. The Company is also required to make performance-based payments upon successful completion of clinical and regulatory benchmarks relating to the licensed products. The aggregate potential benchmark payments are $4.3 million, of which aggregate payments of $3.0 million are due only after marketing approval in the United States or in Europe, Japan, Australia, China or India. The first benchmark payment of $0.1 million will be due upon the initiation of the Company’s first sponsored Phase 1 clinical trial of a licensed product or licensed process in the field of use licensed under the Patent License. In addition, the Company is required to pay the NCI one-time mid-single The Patent License will expire upon expiration of the last patent contained in the licensed patent rights, unless terminated earlier. The NCI may terminate or modify the Patent License in the event of a material breach, including if the Company does not meet certain milestones by certain dates, or upon certain insolvency events that remain uncured following the date that is 90 days following written notice of such breach or insolvency event. The Company may terminate the Patent License, or any portion thereof, in the Company’s sole discretion at any time upon 60 days’ written notice to the NCI. In addition, the NCI has the right to: (i) require the Company to sublicense the rights to the product candidates covered by the Patent License upon certain conditions, including if the Company is not reasonably satisfying required health and safety needs and (ii) terminate or modify the Patent License, including if the Company is not satisfying requirements for public use as specified by federal regulations. Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute On January 10, 2017, the Company announced the signing of a CRADA, with the NCI for the development of adoptive cell transfer, or ACT,-based immunotherapies genetically modified using the Sleeping Beauty non-viral Sleeping Beauty recorded $0.6 million and million of expense for the three and nine months ended September 30, 2020 and 2019, respectively. Ares Trading License and Collaboration Agreement On March 27, 2015, the Company, together with PGEN, signed the Ares Trading Agreement, with Ares Trading S.A., a subsidiary of the biopharmaceutical business of Merck KGaA, Darmstadt, Germany, through which the parties established a collaboration for the research and development and commercialization of certain products for the prophylactic, therapeutic, palliative or diagnostic use for cancer in humans. PGEN was entitled to receive $5.0 million, from Ares Trading, payable in equal quarterly installments over two years for each identified product candidate, which will be used to fund discovery work. The Company was responsible for costs exceeding the quarterly installments and all other costs of the preclinical research and development. For the nine months ended September 30, 2020 and 2019, the Company incurred no expenses under the Ares Trading Agreement. Ares Trading paid a non-refundable Under the License Agreement, PGEN agreed to perform all future obligations of the Company under the Ares Trading Agreement other than certain payment obligations. Accordingly, the Company recognized the remaining deferred revenue as part of the settlement of a related party relationship in 2018. Patent and Technology License Agreement—The University of Texas MD Anderson Cancer Center and the Texas A&M University System On August 24, 2004, the Company entered into a patent and technology license agreement with MD Anderson and the Texas A&M University System, which the Company refers to, collectively, as the Licensors. Under this agreement, the Company was granted an exclusive, worldwide license to rights (including rights to U.S. and foreign patent and patent applications and related improvements and know-how) Under the terms of the agreement, the Company may be required to make additional payments to the Licensors upon achievement of certain other milestones in varying amounts which, on a cumulative basis could total up to an additional $4.5 million. In addition, the Licensors are entitled to receive single digit percentage royalty payments on sales from a licensed product and will also be entitled to receive a portion of any fees that the Company may receive from a possible sublicense under certain circumstances. Collaboration Agreement with Solasia Pharma K.K. On March 7, 2011, the Company entered into a License and Collaboration Agreement with Solasia Pharma K.K., or Solasia, which was amended on July 31, 2014 to include an exclusive worldwide license. Pursuant to the License and Collaboration Agreement, the Company granted Solasia an exclusive license to develop and commercialize darinaparsin in both intravenous and oral forms and related organic arsenic molecules, in all indications for human use. As consideration for the license, the Company is eligible to receive from Solasia development- and sales-based milestones, a royalty on net sales of darinaparsin, once commercialized, and a percentage of any sublicense revenues generated by Solasia. Solasia will be responsible for all costs related to the development, manufacturing and commercialization of darinaparsin. The Company’s Licensors, as defined in the agreement, will receive a portion of all milestone and royalty payments made by Solasia to the Company in accordance with the terms of the license agreement with the Licensors. |
Leases
Leases | 9 Months Ended |
Sep. 30, 2020 | |
Leases, Operating [Abstract] | |
Leases | 7. Leases In June 2012, the Company entered into a master lease for the Company’s corporate office headquarters in Boston, which was originally set to expire in August 2016, but renewed through August 31, 2021. As of September 30, 2020 and December 31, 2019, a total security deposit of $0.1 million is included in deposits on the Company’s balance sheet. On January 30, 2018, the Company entered into a lease agreement for office space in Houston at MD Anderson. Under the terms of the Houston lease agreement, the Company leased approximately two hundred and ten On March 12, 2019, the Company entered into a lease agreement for office space in Houston. Under the terms of the Houston lease agreement, the Company leases approximately one thousand and thirty-eight eight thousand four hundred and forty-three Effective April 13, 2020, the Company leased an additional five thousand five hundred eighty-four All future rent expense incurred in Houston, will be deducted from the Company’s prepayments at MD Anderson. Effective June 1, 2020, the Company entered into a noncancelable lease for a period of less than a year with monthly payments of approximately $10 thousand . Effective September 1, 2020, the Company added additional space to the noncancelable lease for a period of less than a year with monthly payments now totaling approximately $15 thousand The components of lease expense were as follows: Three Months Ended Nine Months Ended (in thousands) 2020 2020 Operating lease cost $ 267 $ 768 Total lease cost $ 267 $ 768 Weighted-average remaining lease term (years) 4.99 4.99 Weighted-average discount rate 8.00 % 8.00 % Three Months Ended Nine Months Ended (in thousands) 2019 2019 Operating lease cost $ 184 $ 585 Total lease cost $ 184 $ 585 Weighted-average remaining lease term (years) 2.15 2.15 Weighted-average discount rate 8.00 % 8.00 % As of September 30, 2020, the maturities of the Company’s operating lease liabilities for the years ended December 31, were as follows (in thousands): 2020 (excluding the nine September $ 318 2021 896 2022 353 2023 364 2024 375 Thereafter 851 Total lease payments 3,157 Less: Imputed interest and adjustments (637 ) Present value of lease payments $ 2,520 |
Stock-Based Compensation
Stock-Based Compensation | 9 Months Ended |
Sep. 30, 2020 | |
Text Block [Abstract] | |
Stock-Based Compensation | 8. Stock-Based Compensation The Company recognized stock-based compensation expense on all employee and non-employee For the three months ended September 30, For the nine months ended September 30, (in thousands) 2020 2019 2020 2019 Research and development $ 522 $ 339 $ 1,587 $ 1,068 General and administrative 1,270 1,147 3,806 3,673 Stock-based compensation expense $ 1,792 $ 1,486 $ 5,393 $ 4,741 The Company granted an aggregate of 203,178 and 1,252,178 stock options during the three and nine months ended September 30, 2020 with a weighted-average grant date fair value of $1.96 and $2.45 per share, respectively. The Company granted an aggregate of 140,000 and 1,835,755 stock options during the three and nine months ended September 30, 2019 with a weighted average grant date fair value of $3.05 and $1.90 per share, respectively. On January 6, 2019, the Company sett l d On May 26, 2020 , , , thousand , On September 18, 2020 , , , a director of the Company . These extensions and acceleration of vesting resulted in additional stock compensation expense of $(250) in the three and nine months ended September 30, 2020. For the three months ended September 30, 2020 and 2019 , For the three months ended September 30, 2020 2019 Risk-free interest rate 0.36 - 0.39% 1.39 - 1.92% Expected life in years 5.75 - 6.25 6.25 Expected volatility 73.59 - 72.87 - 78.34% Expected dividend yield 0% 0% Stock option activity under the Company’s stock option plans for the nine months ended September 30, 2020 is as follows: (in thousands, except share and per share data) Number of Weighted- Weighted- Aggregate Outstanding, December 31, 2019 5,842,879 $ 3.21 Granted 1,252,178 3.89 Exercised (22,916 ) 1.87 Cancelled (499,950 ) 3.84 Outstanding, September 30, 2020 6,572,191 $ 3.93 7.82 $ 978 Options exercisable, September 30, 2020 3,711,684 $ 4.10 6.96 $ 704 Options exercisable, December 31, 2019 2,765,357 $ 4.39 6.70 $ 3,603 Options available for future grant 6,986,610 At September 30, 2020, total unrecognized compensation costs related to unvested stock options outstanding amounted to $8.0 million. The cost is expected to be recognized over a weighted-average period of 1.82 years. A summary of the status of unvested restricted stock for the nine months ended September 30, 2020 is as follows: Number of Shares Weighted-Average Non-vested, 939,636 $ 2.93 Granted 555,900 4.21 Vested (64,917 ) 3.42 Cancelled (141,230 ) 3.15 Non-vested, 1,289,389 $ 3.43 At September 30, 2020, total unrecognized compensation costs related to unvested restricted stock outstanding amounted to $3.0 million. The cost is expected to be recognized over a weighted-average period of 1.57 years. At the Company’s annual meeting held on June 29, 2020, the shareholders approved the 2020 Equity Incentive Plan, or the 2020 Plan, which is a successor to and continuation of the Company’s 2012 Equity Incentive Plan, or the 2012 plan. The 2020 Plan ha d authorized, additional awards can be granted from the 2012 Plan or the Company’s 2003 Stock Option Plan. |
Warrants
Warrants | 9 Months Ended |
Sep. 30, 2020 | |
Warrants Disclosure [Abstract] | |
Warrant | 9. Warrants In connection with the Company’s November 2018 private placement which provided net proceeds of approximately $47.1 million, the Company issued warrants to purchase an aggregate of 18,939,394 shares of common stock, or the 2018 warrants, which became exercisable six months after the closing of the private placement. The warrants have an exercise price of $3.01 per share and have a five-year term. The relative fair value of the warrants was estimated at $18.4 million using a Black-Scholes model with the following assumptions: expected volatility of 71%, risk free interest rate of 2.99%, expected life of five years and no dividends. The Company assessed whether the warrants require accounting as derivatives. The Company determined that the warrants were (1) indexed to the Company’s own stock and (2) classified in stockholders’ equity in accordance with Financial Accounting Standards Board , or Derivatives and Hedging On July 26, 2019 and September 12, 2019, the Company entered into agreements with existing investors for the exercise of previously issued warrants to purchase common stock in the private placement. Pursuant to the terms of the agreements, investors exercised their 2018 warrants for an aggregate of 17,803,031 shares of common stock, at an exercise price of $3.01 per share. Proceeds from the warrant exercise, after deducting placement agent fees and other related expenses of $1.1 million were approximately $52.5 million. The Company issued participating investors new warrants to purchase up to 17,803,031 additional shares of common stock, or the 2019 warrants, as an inducement for the warrant holders to exercise their 2018 warrants. The 2019 warrants will expire on the fifth anniversary of the initial exercise date and have an exercise price of $7.00. The 2019 warrants were valued using a Black-Scholes valuation model and resulted in a $60.8 million non-cash On October 22, 2019, the Company entered into the 2019 Agreement with MD Anderson. Refer to Note 6 – Commitments and Contingencies As of |
Joint Venture
Joint Venture | 9 Months Ended |
Sep. 30, 2020 | |
Equity Method Investments and Joint Ventures [Abstract] | |
Joint Venture | 10. Joint Venture On December 18, 2018, the Company entered into a Framework Agreement with TriArm Therapeutics, Ltd., or TriArm, pursuant to which the parties agreed to launch Eden BioCell, Ltd., or Eden BioCell, to lead clinical development and commercialization of certain Sleeping Beauty- CAR-T On January 3, 2019, Eden BioCell was incorporated in Hong Kong as a private company. Eden BioCell, the Company and TriArm entered into a Share Subscription Agreement on January 23, 2019, where the Company and TriArm agreed to contribute certain intellectual property, services and cash (only with respect to TriArm) to Eden BioCell to subscribe for a certain number of newly issued ordinary shares in the share capital of Eden BioCell. On the closing date, upon the issuance and subscription of the shares, in respect of the aforementioned consideration, 10,000,000 ordinary shares were issued to the Company and 10,000,000 ordinary shares were issued to TriArm. The closing of the transaction occurred on July 5, 2019. The Framework Agreement and Share Subscription Agreements were each respectively amended to be effective as of this date. Upon consummation of the joint venture, Eden BioCell and the Company also entered into a license agreement, pursuant to which the Company licensed the rights to Eden BioCell for third-generation Sleeping Beauty CAR-T see certain spec ified As of July 5, 2019, as a result of the design and purpose of Eden BioCell, the Company determined that Eden BioCell was considered a variable interest entity, or VIE, and concluded that it is not the primary beneficiary of the VIE as it did not have the power to direct the activities of the VIE that most significantly impact its performance. Rather, the Company accounts for the equity interest in Eden BioCell under the equity method of accounting as it has the ability to exercise significant influence over the operations of Eden BioCell. The Company determined that Eden BioCell was not a customer and therefore, accounted for the transaction as the transfer of nonfinancial assets to be recognized at their fair value on the contribution date. The fair value of the intellectual property contributed to Eden BioCell had a de minimis value due to the early stage of the technology and the likelihood of clinical success. Due to the de minimis fair value of the intellectual property contributed, the Company did not record a gain or loss on this transaction and recognized no value for its equity-method investment. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2020 | |
Accounting Policies [Abstract] | |
New Accounting Pronouncements | New Accounting Pronouncements In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes 2019-12 In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement 2018-03. |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured at Fair Value on Recurring Basis | Assets and liabilities measured at fair value on a recurring basis as of September 30, 2020 and December 31, 2019 were as follows: ($ in thousands) Fair Value Measurements at Reporting Date Using Description Balance as of Quoted Prices in Significant Other Significant Assets: Cash equivalents $ 75,300 $ 75,300 $ — $ — ($ in thousands) Fair Value Measurements at Reporting Date Using Description Balance as of Quoted Prices in Significant Other Significant Assets: Cash equivalents $ 68,031 $ 68,031 $ — $ — |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Earnings Per Share [Abstract] | |
Potential Dilutive Shares Excluded from Computation of Diluted Net Loss Per Share | Such potentially dilutive shares of common stock at September 30, 2020 and 2019 consisted of the following: September 30, 2020 2019 Stock options 6,572,191 4,995,549 Inducement stock options 863,333 965,000 Unvested restricted stock 1,289,389 1,569,579 Warrants 22,272,727 18,939,394 30,997,640 26,469,522 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Leases, Operating [Abstract] | |
Components of Lease Expense | The components of lease expense were as follows: Three Months Ended Nine Months Ended (in thousands) 2020 2020 Operating lease cost $ 267 $ 768 Total lease cost $ 267 $ 768 Weighted-average remaining lease term (years) 4.99 4.99 Weighted-average discount rate 8.00 % 8.00 % Three Months Ended Nine Months Ended (in thousands) 2019 2019 Operating lease cost $ 184 $ 585 Total lease cost $ 184 $ 585 Weighted-average remaining lease term (years) 2.15 2.15 Weighted-average discount rate 8.00 % 8.00 % |
Lessee, Operating Lease, Liability, Maturity | As of September 30, 2020, the maturities of the Company’s operating lease liabilities for the years ended December 31, were as follows (in thousands): 2020 (excluding the nine September $ 318 2021 896 2022 353 2023 364 2024 375 Thereafter 851 Total lease payments 3,157 Less: Imputed interest and adjustments (637 ) Present value of lease payments $ 2,520 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2020 | |
Text Block [Abstract] | |
Stock-Based Compensation Expense on All Employee and Non-Employee Awards | The Company recognized stock-based compensation expense on all employee and non-employee For the three months ended September 30, For the nine months ended September 30, (in thousands) 2020 2019 2020 2019 Research and development $ 522 $ 339 $ 1,587 $ 1,068 General and administrative 1,270 1,147 3,806 3,673 Stock-based compensation expense $ 1,792 $ 1,486 $ 5,393 $ 4,741 |
Fair Value of Stock Options Assumptions Using Black-Scholes Option Valuation Model | For the three months ended September 30, 2020 and 2019 , For the three months ended September 30, 2020 2019 Risk-free interest rate 0.36 - 0.39% 1.39 - 1.92% Expected life in years 5.75 - 6.25 6.25 Expected volatility 73.59 - 72.87 - 78.34% Expected dividend yield 0% 0% |
Stock Option Activity under Stock Option Plan | Stock option activity under the Company’s stock option plans for the nine months ended September 30, 2020 is as follows: (in thousands, except share and per share data) Number of Weighted- Weighted- Aggregate Outstanding, December 31, 2019 5,842,879 $ 3.21 Granted 1,252,178 3.89 Exercised (22,916 ) 1.87 Cancelled (499,950 ) 3.84 Outstanding, September 30, 2020 6,572,191 $ 3.93 7.82 $ 978 Options exercisable, September 30, 2020 3,711,684 $ 4.10 6.96 $ 704 Options exercisable, December 31, 2019 2,765,357 $ 4.39 6.70 $ 3,603 Options available for future grant 6,986,610 |
Summary of Unvested Restricted Stock | A summary of the status of unvested restricted stock for the nine months ended September 30, 2020 is as follows: Number of Shares Weighted-Average Non-vested, 939,636 $ 2.93 Granted 555,900 4.21 Vested (64,917 ) 3.42 Cancelled (141,230 ) 3.15 Non-vested, 1,289,389 $ 3.43 |
Business - Additional Informati
Business - Additional Information (Detail) - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Cash and cash equivalents | $ 135,471 | $ 79,741 |
Accumulated deficit | $ 741,329 | $ 684,125 |
Financings - Additional Informa
Financings - Additional Information (Detail) - USD ($) | Mar. 10, 2020 | Feb. 05, 2020 | Sep. 12, 2019 | Jun. 30, 2019 | Nov. 11, 2018 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 |
Class of Stock [Line Items] | |||||||||
Sale of stock consideration received on transaction | $ 88,661,000 | $ 2,971,000 | |||||||
Common stock, par value | $ 0.001 | $ 0.001 | |||||||
Gross proceeds received | $ 2,971,000 | $ 13,015,000 | $ 2,971,000 | ||||||
Proceeds from private placement | $ 52,500,000 | ||||||||
At The Market Offering [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Stock issued during period | 2,814,673 | 639,442 | |||||||
Sale of stock consideration received on transaction | $ 13,000,000 | $ 3,000,000 | |||||||
Underwriting offering cost | $ 13,000,000 | ||||||||
Private Placement [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Class of warrant or right, number of securities called by warrants or rights | 17,803,031 | 18,939,394 | |||||||
Warrant Exercise Price per share | $ 3.01 | $ 3.01 | |||||||
Placement agent fees and other expenses | $ 1,100,000 | ||||||||
Private Placement [Member] | New Warrants [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Class of warrant or right, number of securities called by warrants or rights | 17,803,031 | ||||||||
Warrant Exercise Price per share | $ 7 | ||||||||
Securities Purchase Agreement [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Stock issued during period | 18,939,394 | ||||||||
Common stock, par value | $ 0.001 | ||||||||
par value per share | $ 2.64 | ||||||||
Gross proceeds received | $ 47.1 | $ 47,100,000 | |||||||
Underwriting Agreement [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Stock issued during period | 27,826,086 | ||||||||
Issuance & sale of common stock in public offering price per share | $ 3.25 | ||||||||
Sale of stock consideration received on transaction | $ 84,800,000 | ||||||||
par value per share | $ 3.055 | ||||||||
Underwriting Agreement [Member] | Maximum | |||||||||
Class of Stock [Line Items] | |||||||||
Stock issued during period | 4,173,912 | ||||||||
par value per share | $ 3.055 | ||||||||
Underwriting Agreement [Member] | Stock Option [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Stock issued during period | 1,284,025 | ||||||||
Sale of stock consideration received on transaction | $ 3,900,000 | ||||||||
Open Market Sale Agreement [Member] | At The Market Offering [Member] | |||||||||
Class of Stock [Line Items] | |||||||||
Sale of stock consideration received on transaction | $ 100,000,000 |
Assets and Liabilities Measured
Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value, Measurements, Recurring - USD ($) $ in Thousands | Sep. 30, 2020 | Dec. 31, 2019 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents | $ 75,300 | $ 68,031 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents | $ 75,300 | $ 68,031 |
Potential Dilutive Shares Exclu
Potential Dilutive Shares Excluded from Computation of Diluted Net Loss Per Share (Detail) - shares | 9 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount | 30,997,640 | 26,469,522 |
Warrants | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount | 22,272,727 | 18,939,394 |
Stock Options | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount | 6,572,191 | 4,995,549 |
Stock Options | Two Thousand Twelve Stock Option Plan [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount | 863,333 | 965,000 |
Unvested Restricted Stock | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount | 1,289,389 | 1,569,579 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Thousands | May 28, 2019 | Aug. 17, 2015 | Jan. 13, 2015 | Jul. 31, 2015 | Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Jan. 01, 2021 | Nov. 28, 2020 | Sep. 28, 2020 | Dec. 31, 2019 | Oct. 22, 2019 | Feb. 19, 2019 | Oct. 05, 2018 | Aug. 24, 2004 |
Accrued Payments | ||||||||||||||||
Research and development expense | $ 13,968 | $ 8,641 | $ 38,725 | $ 28,115 | ||||||||||||
Cash balance | 135,471 | 135,471 | $ 79,741 | |||||||||||||
Agreement commencement date | 2015-05 | |||||||||||||||
MD Anderson License and the Research and Development Agreement Member [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Research and development service agreement aggregate quarterly payments | 15,000 | |||||||||||||||
Reimbursement of historical costs | $ 20,000 | |||||||||||||||
Accrued Payments | 3,000 | |||||||||||||||
Number of Warrants | 3,333,333 | |||||||||||||||
CRADA Agreement [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Obligations due under contract | $ 5,000 | |||||||||||||||
Quarterly payments under contract | 600 | 600 | 1,300 | 1,300 | ||||||||||||
Aggregate potential benchmark payments | $ 36,500 | |||||||||||||||
CAR Products [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Amount of royalties receivable | $ 50,000 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Expected cash payment payable per installments | $ 500 | |||||||||||||||
The University of Texas MD Anderson Cancer Center and The Texas A & M University System | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Milestone maximum payment | $ 4,500 | |||||||||||||||
Obligations due under contract | 400 | 400 | $ 400 | |||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Expected Cash Payment Payable | 1,500 | |||||||||||||||
Minimum Royalties Amount Payable | $ 300 | |||||||||||||||
Description Of First Annual Royalty Payable | The first minimum annual royalty payment is payable on the date that is eighteen months following the date of the Patent License. | |||||||||||||||
Description Of First Benchmark Payable | The first benchmark payment of $0.1 million will be due upon the initiation of the Company’s first sponsored Phase 1 clinical trial of a licensed product or licensed process in the field of use licensed under the Patent License. | |||||||||||||||
Description Of option To terminate Agreement | The NCI may terminate or modify the Patent License in the event of a material breach, including if the Company does not meet certain milestones by certain dates, or upon certain insolvency events that remain uncured following the date that is 90 days following written notice of such breach or insolvency event. | |||||||||||||||
Agreement termination, notice period | 60 days | |||||||||||||||
Annual Licensing fee paid | 0 | 0 | 0 | 0 | ||||||||||||
Payments under patent license | 1,500 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | Performance Based Payments Member [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Aggregate Benchmark Payments Payable | $ 4,300 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | One Time Benchmark Payments [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Potential Benchmark Payments Payable | 12,000 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | Scenario, Forecast [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Expected Cash Payment Payable | $ 1,500 | |||||||||||||||
Minimum Royalties Amount Payable | $ 100 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | Post Marketing Approval [Member] | Performance Based Payments Member [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Aggregate Benchmark Payments Payable | 3,000 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | License [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Licensing fee | 0 | 600 | ||||||||||||||
Payments under patent license | 600 | |||||||||||||||
ZIOP License Agreement With The National Cancer Institute [Member] | licensed products [Member] | One Time Benchmark Payments [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Maximum Sales Revenue On Which Benchmark Payments Payable | $ 1,000,000 | |||||||||||||||
Prepaid Expenses and Other Current Assets | MD Anderson License | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Cash balance | 11,200 | 11,200 | ||||||||||||||
Prepaid Expenses and Other Current Assets | ZIOP License Agreement With The National Cancer Institute [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Prepaid Royalties | 300 | 300 | ||||||||||||||
Intrexon Corporation | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Licensing fee | $ 115,000 | |||||||||||||||
Research and development expense | $ 300 | $ 1,700 | ||||||||||||||
Milestone payment receivable | 5,000 | 5,000 | ||||||||||||||
Upfront payment received | $ 57,500 | |||||||||||||||
Percentage of upfront fee Payable | 50.00% | |||||||||||||||
Annual Licensing fee | 100 | |||||||||||||||
Expected additional milestones payable | 52,500 | |||||||||||||||
Expected Cash Payment Payable | $ 0 | 0 | $ 100 | |||||||||||||
Annual Licensing fee paid | $ 100 | |||||||||||||||
Intrexon Corporation | T-cell receptor | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Maximum royalty amount | $ 100,000 | |||||||||||||||
Portion of income payable to related party | 20.00% | |||||||||||||||
Maximum | MD Anderson License and the Research and Development Agreement Member [Member] | ||||||||||||||||
Accrued Payments | ||||||||||||||||
Research and development expense | $ 20,000 |
Lease expense (Detail)
Lease expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Operating lease cost | $ 267 | $ 184 | $ 768 | $ 585 |
Total lease cost | $ 267 | $ 184 | $ 768 | $ 585 |
Weighted-average remaining lease term (years) | 4 years 11 months 26 days | 2 years 1 month 24 days | 4 years 11 months 26 days | 2 years 1 month 24 days |
Weighted-average discount rate | 8.00% | 8.00% | 8.00% | 8.00% |
Operating lease liabilities (De
Operating lease liabilities (Detail) $ in Thousands | Sep. 30, 2020USD ($) |
2020 (excluding the nine months ended September 30, 2020) | $ 318 |
2021 | 896 |
2022 | 353 |
2023 | 364 |
2024 | 375 |
Thereafter | 851 |
Total lease payments | 3,157 |
Less: Imputed interest and adjustments | (637) |
Present value of lease payments | $ 2,520 |
Leases - Additional Information
Leases - Additional Information (Details) $ in Thousands | Sep. 01, 2020USD ($) | Jun. 01, 2020USD ($) | Apr. 13, 2020USD ($) | Mar. 12, 2019USD ($)ft² | Sep. 30, 2020USD ($) | Sep. 30, 2020USD ($) | Dec. 31, 2019USD ($) | Oct. 15, 2019USD ($)ft² | Jan. 30, 2018USD ($)ft² |
Land Subject to Ground Leases | ft² | 1,038 | ||||||||
Operating Lease Monthly Rental Payment | $ 10 | $ 2 | |||||||
Boston, MA | |||||||||
Security Deposit | $ 100 | $ 100 | $ 100 | ||||||
Sublease term amendment | Aug. 31, 2021 | ||||||||
Operating lease expiration month and year | 2016-08 | ||||||||
Houston, TX | |||||||||
Land Subject to Ground Leases | ft² | 8,443 | ||||||||
Operating Lease Area | ft² | 210 | ||||||||
Operating Leases Future Minimum Monthly Payment Due Through Year 2021 | $ 12 | $ 17 | $ 1 | ||||||
Annual Base Rent | 3.00% | 3.00% | |||||||
Lessee Operating Lease Monthly Rental Payment | $ 15 |
Stock-Based Compensation Expens
Stock-Based Compensation Expense Included in Statement of Operations (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation | $ 1,792 | $ 1,486 | $ 5,393 | $ 4,741 |
Research and Development Expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation | 522 | 339 | 1,587 | 1,068 |
General and Administrative Expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Stock-based compensation | $ 1,270 | $ 1,147 | $ 3,806 | $ 3,673 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) | Sep. 18, 2020 | Jun. 30, 2020 | May 26, 2020 | Jan. 06, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Jun. 29, 2020 | Dec. 31, 2019 |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Outstanding options issued | 6,572,191 | 6,572,191 | 5,842,879 | |||||||
Stock options, granted | 203,178 | 140,000 | 1,252,178 | 1,835,755 | ||||||
Options available for future grant | 6,986,610 | 6,986,610 | ||||||||
Weighted-average grant date fair value | $ 1.96 | $ 3.05 | $ 2.45 | $ 1.90 | ||||||
Number of shares issued for accrued annual performance bonus | 446,428 | |||||||||
Number of options vested | 113,350 | 31,220 | 448,130 | |||||||
Adiitional stock compensation expenses | $ 65,000 | $ 154,000 | ||||||||
Acceleration Of Vesting [Member] | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Adiitional stock compensation expenses | (250) | (250) | ||||||||
Director | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Number of options vested | 25,281 | |||||||||
Restricted Stock | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Number of shares vested | 15,890 | 45,277 | ||||||||
Unvested Stock | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Unrecognized compensation costs related to unvested restricted stock outstanding | 8,000,000 | $ 8,000,000 | ||||||||
Expected recognition period | 1 year 9 months 25 days | |||||||||
Unvested Restricted Common Stock | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Unrecognized compensation costs related to unvested restricted stock outstanding | $ 3,000,000 | $ 3,000,000 | ||||||||
Expected recognition period | 1 year 6 months 25 days | |||||||||
Number of shares vested | 64,917 | |||||||||
Outside 2012 Plan | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Outstanding options issued | 863,333 | 863,333 | ||||||||
2020 Equity Incentive Plan [Member] | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Shares available for new issue | 21,000,000 | |||||||||
2012 Equity Incentive Plan [Member] | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Options available for future grant | 0 | |||||||||
2003 Equity Incentive Plan [Member] | ||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||
Options available for future grant | 0 |
Fair Value of Stock Options Ass
Fair Value of Stock Options Assumptions Using Black-Scholes Option Valuation Model (Detail) | 3 Months Ended | |
Sep. 30, 2020 | Sep. 30, 2019 | |
Risk-free interest rate, Minimum | 0.36% | 1.39% |
Risk-free interest rate, Maximum | 0.39% | 1.92% |
Expected life in years | 6 years 3 months | |
Expected volatility, Minimum | 73.59% | 72.87% |
Expected volatility, Maximum | 74.18% | 78.34% |
Expected dividend yield | 0.00% | 0.00% |
Maximum [Member] | ||
Expected life in years | 6 years 3 months | |
Minimum [Member] | ||
Expected life in years | 5 years 9 months |
Stock Option Activity Under Sto
Stock Option Activity Under Stock Option Plan (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 | Dec. 31, 2019 | |
Number of Shares | |||||
Beginning Balance | 5,842,879 | ||||
Granted | 203,178 | 140,000 | 1,252,178 | 1,835,755 | |
Exercised | (22,916) | ||||
Cancelled | (499,950) | ||||
Ending Balance | 6,572,191 | 6,572,191 | 5,842,879 | ||
Options exercisable, at end of period | 3,711,684 | 3,711,684 | 2,765,357 | ||
Options available for future grant | 6,986,610 | 6,986,610 | |||
Weighted Average Exercise Price | |||||
Beginning Balance | $ 3.21 | ||||
Granted | 3.89 | ||||
Exercised | 1.87 | ||||
Cancelled | 3.84 | ||||
Ending Balance | $ 3.93 | 3.93 | $ 3.21 | ||
Options exercisable, at end of period | $ 4.10 | $ 4.10 | $ 4.39 | ||
Weighted Average Contractual Term (Years) | |||||
Outstanding, at end of period | 7 years 9 months 25 days | ||||
Options exercisable, at end of period | 6 years 11 months 15 days | 6 years 8 months 12 days | |||
Aggregate Intrinsic Value | |||||
Outstanding, at end of period | $ 978 | $ 978 | |||
Options exercisable, at end of period | $ 704 | $ 704 | $ 3,603 |
Summary of Non-Vested Restricte
Summary of Non-Vested Restricted Stock (Detail) - Unvested Restricted Common Stock | 9 Months Ended |
Sep. 30, 2020$ / sharesshares | |
Number of Shares | |
Beginning Balance | shares | 939,636 |
Granted | shares | 555,900 |
Vested | shares | (64,917) |
Cancelled | shares | (141,230) |
Ending Balance | shares | 1,289,389 |
Weighted Average Grant Date Fair Value | |
Beginning Balance | $ / shares | $ 2.93 |
Granted | $ / shares | 4.21 |
Vested | $ / shares | 3.42 |
Cancelled | $ / shares | 3.15 |
Ending Balance | $ / shares | $ 3.43 |
Warrants - Additional Informati
Warrants - Additional Information (Detail) - USD ($) | Sep. 12, 2019 | Nov. 11, 2018 | Sep. 30, 2020 | Sep. 30, 2019 | Sep. 30, 2020 | Sep. 30, 2019 |
Fair Value Assumptions Expected volatility Rate Maximum | 74.18% | 78.34% | ||||
Fair Value Assumptions Risk Free Interest Rate Maximum | 0.39% | 1.92% | ||||
Fair Value Assumptions Expected Term1 | 6 years 3 months | |||||
Fair Value Assumptions Expected Dividend Rate | 0.00% | 0.00% | ||||
Net Proceeds from Underwriting | $ 2,971,000 | $ 13,015,000 | $ 2,971,000 | |||
Proceeds from private placement | $ 52,500,000 | |||||
Non-cash inducement warrant expense | $ 60,751,000 | $ 60,751,000 | ||||
MD Anderson Warrant [Member] | ||||||
Number of securities into which the class of warrant converted | 3,333,333 | 3,333,333 | ||||
Warrant Exercise Per share | $ 0.001 | $ 0.001 | ||||
fair value of adjustment of warrants | $ 14,500,000 | |||||
Warrant Expiry date | Dec. 31, 2026 | Dec. 31, 2026 | ||||
Securities Purchase Agreement [Member] | ||||||
Net Proceeds from Underwriting | $ 47.1 | $ 47,100,000 | ||||
Private Placement [Member] | ||||||
Number of securities into which the class of warrant converted | 17,803,031 | 18,939,394 | 18,939,394 | |||
Warrant Exercise Per share | $ 3.01 | $ 3.01 | $ 3.01 | |||
fair value of adjustment of warrants | $ 18,400,000 | |||||
Fair Value Assumptions Expected volatility Rate Maximum | 71.00% | |||||
Fair Value Assumptions Risk Free Interest Rate Maximum | 2.99% | |||||
Fair Value Assumptions Expected Term1 | 5 years | |||||
Fair Value Assumptions Expected Dividend Rate | 0.00% | |||||
Placement agent fees and other expenses | $ 1,100,000 | |||||
Private Placement [Member] | New Warrants [Member] | ||||||
Number of securities into which the class of warrant converted | 17,803,031 | |||||
Warrant Exercise Per share | $ 7 | |||||
Non-cash inducement warrant expense | $ 60,800,000 |
Joint Venture - Additional Inf
Joint Venture - Additional Information (Detail) - USD ($) $ in Millions | Jul. 05, 2019 | Sep. 30, 2020 | Dec. 31, 2019 | Jan. 03, 2019 |
Subsidiary or Equity Method Investee [Line Items] | ||||
Common stock, shares issued | 214,165,690 | 181,803,320 | ||
Eden Bio Cell [Member] | ||||
Subsidiary or Equity Method Investee [Line Items] | ||||
Equity method investments | $ 0 | |||
Ziopharm [Member] | Eden Bio Cell [Member] | ||||
Subsidiary or Equity Method Investee [Line Items] | ||||
Equity interest in affilated entity | 50.00% | |||
Tri Arm [Member] | Eden Bio Cell [Member] | ||||
Subsidiary or Equity Method Investee [Line Items] | ||||
Equity interest in affilated entity | 50.00% | |||
Tri Arm [Member] | License Agreement Terms [Member] | Eden Bio Cell [Member] | ||||
Subsidiary or Equity Method Investee [Line Items] | ||||
Milestone payment made | $ 10 | |||
Conditional milestone amount payable | $ 25 | |||
Share Subscription Agreement [Member] | Ziopharm [Member] | Eden Bio Cell [Member] | ||||
Subsidiary or Equity Method Investee [Line Items] | ||||
Common stock, shares issued | 10,000,000 | |||
Share Subscription Agreement [Member] | Tri Arm [Member] | Eden Bio Cell [Member] | ||||
Subsidiary or Equity Method Investee [Line Items] | ||||
Common stock, shares issued | 10,000,000 |