☒ | QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
☐ | TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
Delaware
Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||
Common Stock, par value $0.001 per share | GBT | The NASDAQ Global Select Market |
Large accelerated filer | ☒ | Accelerated filer | ☐ | |||
Non-accelerated filer | ☐ | Smaller reporting company | ☐ | |||
Emerging growth company | ☐ |
Securities registered pursuant to Section 12(b) of the Act:
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Item 1. | 25 | |||||
Item 1A. | 25 | |||||
Item 2. | 62 | |||||
Item 3. | 63 | |||||
Item 4. | 63 | |||||
Item 5. | 63 | |||||
Item 6. | 64 | |||||
65 |
Item 1. | Financial Statements |
March 31, 2019 | December 31, 2018 | |||||||
(Unaudited) | ||||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 156,836 | $ | 275,357 | ||||
Short-term marketable securities | 300,431 | 202,177 | ||||||
Prepaid expenses and other current assets | 8,222 | 8,246 | ||||||
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Total current assets | 465,489 | 485,780 | ||||||
Property and equipment, net | 13,090 | 14,981 | ||||||
Long-term marketable securities | 117,811 | 114,281 | ||||||
Operating leaseright-of-use assets | 14,075 | — | ||||||
Restricted cash | 2,395 | 2,395 | ||||||
Other assets, noncurrent | 200 | 206 | ||||||
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Total assets | $ | 613,060 | $ | 617,643 | ||||
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Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 7,346 | $ | 6,046 | ||||
Accrued liabilities | 17,757 | 16,792 | ||||||
Accrued compensation | 5,629 | 10,036 | ||||||
Other liabilities, current | 1,359 | 899 | ||||||
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Total current liabilities | 32,091 | 33,773 | ||||||
Operating lease liabilities, noncurrent | 24,401 | — | ||||||
Other liabilities, noncurrent | 30 | 11,071 | ||||||
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Total liabilities | 56,522 | 44,844 | ||||||
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Commitments and contingencies | ||||||||
Stockholders’ equity: | ||||||||
Preferred stock, $0.001 par value, 5,000,000 shares authorized as of March 31, 2019 and December 31, 2018; no shares issued and outstanding | — | — | ||||||
Common stock, $0.001 par value, 150,000,000 shares authorized as of March 31, 2019 (unaudited) and December 31, 2018, respectively; 56,337,045 and 55,640,299 shares issued and outstanding as of March 31, 2019 (unaudited) and December 31, 2018, respectively | 56 | 56 | ||||||
Additionalpaid-in capital | 1,076,979 | 1,044,941 | ||||||
Accumulated other comprehensive income (loss) | 576 | (48 | ) | |||||
Accumulated deficit | (521,073 | ) | (472,150 | ) | ||||
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Total stockholders’ equity | 556,538 | 572,799 | ||||||
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Total liabilities and stockholders’ equity | $ | 613,060 | $ | 617,643 | ||||
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September 30, 2019 | December 31, 2018 | |||||||
(Unaudited) | ||||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 267,604 | $ | 275,357 | ||||
Short-term marketable securities | 335,500 | 202,177 | ||||||
Prepaid expenses and other current assets | 12,699 | 8,246 | ||||||
Total current assets | 615,803 | 485,780 | ||||||
Property and equipment, net | 14,337 | 14,981 | ||||||
Long-term marketable securities | 80,000 | 114,281 | ||||||
Operating lease right-of-use assets | 13,845 | — | ||||||
Restricted cash | 2,395 | 2,395 | ||||||
Other assets, noncurrent | 968 | 206 | ||||||
Total assets | $ | 727,348 | $ | 617,643 | ||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 7,135 | $ | 6,046 | ||||
Accrued liabilities | 22,198 | 16,792 | ||||||
Accrued compensation | 10,450 | 10,036 | ||||||
Other liabilities, current | 1,410 | 899 | ||||||
Total current liabilities | 41,193 | 33,773 | ||||||
Operating lease liabilities, noncurrent | 23,691 | — | ||||||
Other liabilities, noncurrent | 4,367 | 11,071 | ||||||
Total liabilities | 69,251 | 44,844 | ||||||
Commitments and contingencies | ||||||||
Stockholders’ equity: | ||||||||
Preferred stock, $0.001 par value, 5,000,000 shares authorized as of September 30, 2019 (unaudited) and December 31, 2018; 0 shares issued and outstanding | — | — | ||||||
Common stock, $0.001 par value, 150,000,000 shares authorized as of September 30, 2019 (unaudited) and December 31, 2018, respectively; 60,208,293 and 55,640,299 shares issued and outstanding as of September 30, 2019 (unaudited) and December 31, 2018, respectively | 60 | 56 | ||||||
Additional paid-in capital | 1,300,000 | 1,044,941 | ||||||
Accumulated other comprehensive income (loss) | 978 | (48 | ) | |||||
Accumulated deficit | (642,941 | ) | (472,150 | ) | ||||
Total stockholders’ equity | 658,097 | 572,799 | ||||||
Total liabilities and stockholders’ equity | $ | 727,348 | $ | 617,643 |
Three Months Ended March 31, | ||||||||
2019 | 2018 | |||||||
Operating expenses: | ||||||||
Research and development | $ | 34,468 | $ | 29,944 | ||||
General and administrative | 18,055 | 12,751 | ||||||
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Total operating expenses | 52,523 | 42,695 | ||||||
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Loss from operations | (52,523 | ) | (42,695 | ) | ||||
Other income (expense): | ||||||||
Interest income, net | 3,650 | 1,173 | ||||||
Other expenses, net | (50 | ) | (34 | ) | ||||
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Total other income, net | 3,600 | 1,139 | ||||||
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Net loss | (48,923 | ) | (41,556 | ) | ||||
Other comprehensive income: | ||||||||
Net unrealized gain on marketable securities, net of tax | 624 | 11 | ||||||
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Comprehensive loss | $ | (48,299 | ) | $ | (41,545 | ) | ||
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Basic and diluted net loss per common share | $ | (0.87 | ) | $ | (0.87 | ) | ||
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Weighted-average number of shares used in computing basic and diluted net loss per common share | 56,231,587 | 47,770,023 | ||||||
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Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 39,088 | $ | 33,026 | $ | 109,564 | $ | 94,543 | ||||||||
General and administrative | 29,654 | 12,450 | 72,503 | 36,115 | ||||||||||||
Total operating expenses | 68,742 | 45,476 | 182,067 | 130,658 | ||||||||||||
Loss from operations | (68,742 | ) | (45,476 | ) | (182,067 | ) | (130,658 | ) | ||||||||
Other income (expense): | ||||||||||||||||
Interest income, net | 4,226 | 2,480 | 11,422 | 5,768 | ||||||||||||
Other expenses, net | (31 | ) | (72 | ) | (146 | ) | (101 | ) | ||||||||
Total other income, net | 4,195 | 2,408 | 11,276 | 5,667 | ||||||||||||
Net loss | (64,547 | ) | (43,068 | ) | (170,791 | ) | (124,991 | ) | ||||||||
Other comprehensive loss: | ||||||||||||||||
Net unrealized gain (loss) on marketable securities, net of tax | (213 | ) | (139 | ) | 1,026 | (45 | ) | |||||||||
Comprehensive loss | $ | (64,760 | ) | $ | (43,207 | ) | $ | (169,765 | ) | $ | (125,036 | ) | ||||
Basic and diluted net loss per common share | $ | (1.07 | ) | $ | (0.83 | ) | $ | (2.96 | ) | $ | (2.47 | ) | ||||
Weighted-average number of shares used in computing basic and diluted net loss per common share | 60,098,093 | 52,050,232 | 57,637,318 | 50,536,860 |
Common Stock | Additional Paid- In Capital | Accumulated Other Comprehensive Loss | Accumulated Deficit | Total Stockholders’ Equity | ||||||||||||||||||||
Shares | Amount | |||||||||||||||||||||||
Balance at December 31, 2018 | 55,640,299 | $ | 56 | $ | 1,044,941 | $ | (48 | ) | $ | (472,150 | ) | $ | 572,799 | |||||||||||
Issuance of common stock upon equity offerings, net of issuance costs | 511,363 | — | 21,246 | — | — | 21,246 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 52,288 | — | 817 | — | — | 817 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 78,155 | — | (686 | ) | — | — | (686 | ) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases | 30,745 | — | 1,128 | — | — | 1,128 | ||||||||||||||||||
Vesting of restricted stock purchases | 24,195 | — | 80 | — | — | 80 | ||||||||||||||||||
Stock-based compensation expense | — | — | 9,453 | — | — | 9,453 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | 624 | — | 624 | ||||||||||||||||||
Net loss | — | — | — | — | (48,923 | ) | (48,923 | ) | ||||||||||||||||
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Balance at March 31, 2019 | 56,337,045 | $ | 56 | $ | 1,076,979 | $ | 576 | $ | (521,073 | ) | $ | 556,538 | ||||||||||||
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Common Stock | Additional Paid- In Capital | Accumulated Other Comprehensive Loss | Accumulated Deficit | Total Stockholders’ Equity | ||||||||||||||||||||
Shares | Amount | |||||||||||||||||||||||
Balance at December 31, 2017 | 46,131,723 | $ | 46 | $ | 617,051 | $ | (336 | ) | $ | (297,957 | ) | $ | 318,804 | |||||||||||
Issuance of common stock upon equity offerings, net of issuance costs | 4,994,736 | 5 | 255,062 | — | — | 255,067 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 259,150 | 1 | 2,168 | — | — | 2,169 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 143,400 | — | (5,404 | ) | — | — | (5,404 | ) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases | 36,748 | — | 784 | — | — | 784 | ||||||||||||||||||
Vesting of restricted stock purchases | 71,246 | — | 105 | — | — | 105 | ||||||||||||||||||
Stock-based compensation expense | — | — | 7,784 | — | — | 7,784 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | 11 | — | 11 | ||||||||||||||||||
Net loss | — | — | — | — | (41,556 | ) | (41,556 | ) | ||||||||||||||||
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Balance at March 31, 2018 | 51,637,003 | $ | 52 | $ | 877,550 | $ | (325 | ) | $ | (339,513 | ) | $ | 537,764 | |||||||||||
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Common Stock | Additional | Accumulated Other | Total | |||||||||||||||||||||
Shares | Amount | Paid- In Capital | Comprehensive Loss | Accumulated Deficit | Stockholders ’ Equity | |||||||||||||||||||
Balance at December 31, 2018 | 55,640,299 | $ | 56 | $ | 1,044,941 | $ | (48 | ) | $ | (472,150 | ) | $ | 572,799 | |||||||||||
Issuance of common stock upon equity offerings, net of issuance costs | 511,363 | — | 21,246 | — | — | 21,246 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 52,288 | — | 817 | — | — | 817 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 78,155 | — | (686 | ) | — | — | (686 | ) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases | 30,745 | — | 1,128 | — | — | 1,128 | ||||||||||||||||||
Vesting of restricted stock purchases | 24,195 | — | 80 | — | — | 80 | ||||||||||||||||||
Stock-based compensation expense | — | — | 9,453 | — | — | 9,453 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | 624 | — | 624 | ||||||||||||||||||
Net loss | — | — | — | — | (48,923 | ) | (48,923 | ) | ||||||||||||||||
Balance at March 31, 2019 | 56,337,045 | $ | 56 | $ | 1,076,979 | $ | 576 | $ | (521,073 | ) | $ | 556,538 | ||||||||||||
Issuance of common stock upon equity offerings, net of issuance costs | 3,375,527 | 3 | 192,294 | — | — | 192,297 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 123,513 | 1 | 2,331 | — | — | 2,332 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 47,258 | — | (1,297 | ) | — | — | (1,297 | ) | ||||||||||||||||
Vesting of restricted stock purchases | 22,856 | — | 78 | — | — | 78 | ||||||||||||||||||
Stock-based compensation expense | — | — | 10,948 | — | — | 10,948 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | 615 | — | 615 | ||||||||||||||||||
Net loss | — | — | — | — | (57,321 | ) | (57,321 | ) | ||||||||||||||||
Balance at June 30, 2019 | 59,906,199 | $ | 60 | $ | 1,281,333 | $ | 1,191 | $ | (578,394 | ) | $ | 704,190 | ||||||||||||
Issuance of common stock upon equity offerings, net of issuance costs | 100,000 | — | 6,126 | — | — | 6,126 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 18,439 | — | 386 | — | — | 386 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 151,120 | — | (1,532 | ) | — | — | (1,532 | ) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases | 32,535 | — | 1,234 | — | — | 1,234 | ||||||||||||||||||
Stock-based compensation expense | — | — | 12,453 | — | — | 12,453 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | (213 | ) | — | (213 | ) | ||||||||||||||||
Net loss | — | — | — | — | (64,547 | ) | (64,547 | ) | ||||||||||||||||
Balance at September 30, 2019 | 60,208,293 | $ | 60 | $ | 1,300,000 | $ | 978 | $ | (642,941 | ) | $ | 658,097 |
Common Stock | Additional | Accumulated Other | Total | |||||||||||||||||||||
Shares | Amount | Paid- In Capital | Comprehensive Loss | Accumulated Deficit | Stockholders ’ Equity | |||||||||||||||||||
Balance at December 31, 2017 | 46,131,723 | $ | 46 | $ | 617,051 | $ | (336 | ) | $ | (297,957 | ) | $ | 318,804 | |||||||||||
Issuance of common stock upon equity offerings, net of issuance costs | 4,994,736 | 5 | 255,062 | — | — | 255,067 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 259,150 | 1 | 2,168 | — | — | 2,169 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 143,400 | — | (5,404 | ) | — | — | (5,404 | ) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases | 36,748 | — | 784 | — | — | 784 | ||||||||||||||||||
Vesting of restricted stock purchases | 71,246 | — | 105 | — | — | 105 | ||||||||||||||||||
Stock-based compensation expense | — | — | 7,784 | — | — | 7,784 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | 11 | — | 11 | ||||||||||||||||||
Net loss | — | — | — | — | (41,556 | ) | (41,556 | ) | ||||||||||||||||
Balance at March 31, 2018 | 51,637,003 | $ | 52 | $ | 877,550 | $ | (325 | ) | $ | (339,513 | ) | $ | 537,764 | |||||||||||
Offering cost related to the December 2017 follow-on offerings | — | — | 52 | — | — | 52 | ||||||||||||||||||
Issuance of common stock upon exercise of stock options | 148,954 | — | 1,118 | — | — | 1,118 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 10,123 | — | — | — | — | — | ||||||||||||||||||
Vesting of restricted stock purchases | 71,014 | — | 104 | — | — | 104 | ||||||||||||||||||
Stock-based compensation expense | — | — | 7,800 | — | — | 7,800 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | 83 | — | 83 | ||||||||||||||||||
Net loss | — | — | — | — | (40,368 | ) | (40,368 | ) | ||||||||||||||||
Balance at June 30, 2018 | 51,867,094 | $ | 52 | $ | 886,624 | $ | (242 | ) | $ | (379,881 | ) | $ | 506,553 | |||||||||||
Issuance of common stock upon exercise of stock options | 119,174 | — | 1,792 | — | — | 1,792 | ||||||||||||||||||
Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes | 84,045 | — | (849 | ) | — | — | (849 | ) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases | 24,283 | — | 863 | — | — | 863 | ||||||||||||||||||
Vesting of restricted stock purchases | 25,791 | — | 80 | — | — | 80 | ||||||||||||||||||
Stock-based compensation expense | — | — | 6,968 | — | — | 6,968 | ||||||||||||||||||
Net unrealized gain on marketable securities | — | — | — | (139 | ) | — | (139 | ) | ||||||||||||||||
Net loss | — | — | — | — | (43,068 | ) | (43,068 | ) | ||||||||||||||||
Balance at September 30, 2018 | 52,120,387 | $ | 52 | $ | 895,478 | $ | (381 | ) | $ | (422,949 | ) | $ | 472,200 | |||||||||||
Three Months Ended March 31, | ||||||||
2019 | 2018 | |||||||
CASH FLOWS FROM OPERATING ACTIVITIES: | ||||||||
Net loss | $ | (48,923 | ) | $ | (41,556 | ) | ||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||
Depreciation and amortization | 2,094 | 670 | ||||||
Amortization (accretion) of premium (discount) on marketable securities | (565 | ) | 93 | |||||
Amortization of operating leaseright-of-use assets | 100 | — | ||||||
Stock-based compensation | 9,453 | 7,784 | ||||||
Changes in operating assets and liabilities: | ||||||||
Prepaid expenses and other assets | 49 | (677 | ) | |||||
Accounts payable | 1,272 | 262 | ||||||
Accrued liabilities | 887 | 125 | ||||||
Accrued compensation | (4,407 | ) | (3,856 | ) | ||||
Operating lease liabilities | (276 | ) | — | |||||
Other liabilities | — | 389 | ||||||
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Net cash used in operating activities | (40,316 | ) | (36,766 | ) | ||||
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CASH FLOWS FROM INVESTING ACTIVITIES: | ||||||||
Purchase of property and equipment | (56 | ) | (502 | ) | ||||
Purchase of marketable securities | (164,177 | ) | — | |||||
Maturities of marketable securities | 63,582 | 42,076 | ||||||
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Net cash provided by (used in) investing activities | (100,651 | ) | 41,574 | |||||
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CASH FLOWS FROM FINANCING ACTIVITIES: | ||||||||
Proceeds from issuance of common stock in public offering, net | 21,207 | 254,940 | ||||||
Proceeds from issuance of common stock in settlement of employee stock purchase plan and exercise of stock options | 1,925 | 2,821 | ||||||
Tax paid related to net share settlement of equity awards | (686 | ) | (5,404 | ) | ||||
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Net cash provided by financing activities | 22,446 | 252,357 | ||||||
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Net increase (decrease) in cash, cash equivalents and restricted cash | (118,521 | ) | 257,165 | |||||
Cash, cash equivalents and restricted cash at beginning of period | 277,752 | 199,378 | ||||||
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Cash, cash equivalents and restricted cash at end of period | $ | 159,231 | $ | 456,543 | ||||
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SUPPLEMENTAL DISCLOSURES OFNON-CASH INVESTING AND FINANCING INFORMATION: | ||||||||
Accrued purchase of property and equipment | $ | 146 | $ | 1,087 | ||||
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Accrued offering costs | $ | (41 | ) | $ | 127 | |||
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Nine Months Ended September 30, | ||||||||
2019 | 2018 | |||||||
CASH FLOWS FROM OPERATING ACTIVITIES: | ||||||||
Net loss | $ | (170,791 | ) | $ | (124,991 | ) | ||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||
Depreciation and amortization | 6,294 | 2,509 | ||||||
Accretion of discount on marketable securities | (1,724 | ) | (226 | ) | ||||
Amortization of operating lease right-of-use assets | 332 | — | ||||||
Stock-based compensation | 32,854 | 22,552 | ||||||
Changes in operating assets and liabilities: | ||||||||
Prepaid expenses and other assets | (5,217 | ) | (4,496 | ) | ||||
Accounts payable | 1,088 | (2,131 | ) | |||||
Accrued liabilities | 5,557 | 5,882 | ||||||
Accrued compensation | 415 | (1,400 | ) | |||||
Other current liabilities | (30 | ) | — | |||||
Operating lease liabilities | (858 | ) | — | |||||
Other liabilities | — | 499 | ||||||
Net cash used in operating activities | (132,080 | ) | (101,802 | ) | ||||
CASH FLOWS FROM INVESTING ACTIVITIES: | ||||||||
Purchase of property and equipment | (1,198 | ) | (4,946 | ) | ||||
Purchase of marketable securities | (330,130 | ) | (259,631 | ) | ||||
Maturities of marketable securities | 233,837 | 108,707 | ||||||
Net cash used in investing activities | (97,491 | ) | (155,870 | ) | ||||
CASH FLOWS FROM FINANCING ACTIVITIES: | ||||||||
Proceeds from issuance of common stock in public offering, net of issuance costs | 219,443 | 255,119 | ||||||
Proceeds from issuance of common stock in settlement of employee stock purchase plan and exercise of stock options | 5,890 | 6,722 | ||||||
Repurchases of unvested restricted stock | — | (8 | ) | |||||
Tax paid related to net share settlement of equity awards | (3,515 | ) | (6,253 | ) | ||||
Net cash provided by financing activities | 221,818 | 255,580 | ||||||
Net decrease in cash, cash equivalents and restricted cash | (7,753 | ) | (2,092 | ) | ||||
Cash, cash equivalents and restricted cash at beginning of period | 277,752 | 199,378 | ||||||
Cash, cash equivalents and restricted cash at end of period | $ | 269,999 | $ | 197,286 | ||||
SUPPLEMENTAL DISCLOSURES OF NON-CASH INVESTING AND FINANCING INFORMATION: | ||||||||
Leasehold improvements paid for by landlord | $ | 4,367 | $ | — | ||||
Accrued purchase of property and equipment | $ | 76 | $ | (2,173 | ) | |||
December 31, 2018 | Adjustments Due to our Adoption of Topic 842 | January 1, 2019 | ||||||||||
Assets: | ||||||||||||
Operating lease ROU assets | $ | — | $ | 14,177 | $ | 14,177 | ||||||
Liabilities: | ||||||||||||
Operating lease liabilities, current as included in other liabilities, current | — | 1,176 | 1,176 | |||||||||
Deferred rent, current as included in other liabilities, current | 712 | (712 | ) | — | ||||||||
Operating lease liabilities, noncurrent | — | 24,754 | 24,754 | |||||||||
Deferred rent, noncurrent as included in other liabilities, noncurrent | 11,041 | (11,041 | ) | — |
December 31, 2018 | Adjustments Due to our Adoption of Topic 842 | January 1, 2019 | ||||||||||
Assets: | ||||||||||||
Operating Lease ROU assets | $ | — | $ | 14,177 | $ | 14,177 | ||||||
Liabilities: | ||||||||||||
Operating lease liabilities, current as included in other liabilities, current | — | 1,176 | 1,176 | |||||||||
Deferred rent, current as included in other liabilities, current | 712 | (712 | ) | — | ||||||||
Operating lease liabilities, noncurrent | — | 24,754 | 24,754 | |||||||||
Deferred rent, noncurrent as included in other liabilities, noncurrent | 11,041 | (11,041 | ) | — |
March 31, 2019 | ||||||||||||||||
Total | Level 1 | Level 2 | Level 3 | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 145,413 | $ | 145,413 | $ | — | $ | — | ||||||||
Corporate debt securities | 143,927 | — | 143,927 | — | ||||||||||||
U.S. government agency securities | 109,458 | — | 109,458 | — | ||||||||||||
Certificates of deposits | 6,957 | — | 6,957 | — | ||||||||||||
U.S. government securities | 169,231 | — | 169,231 | — | ||||||||||||
|
|
|
|
|
|
|
| |||||||||
Total financial assets | $ | 574,986 | $ | 145,413 | $ | 429,573 | $ | — | ||||||||
|
|
|
|
|
|
|
|
September 30, 2019 | ||||||||||||||||
Total | Level 1 | Level 2 | Level 3 | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 237,318 | $ | 237,318 | $ | — | $ | — | ||||||||
Corporate debt securities | 117,188 | — | 117,188 | — | ||||||||||||
U.S. government agency securities | 146,933 | — | 146,933 | — | ||||||||||||
Certificates of deposits | 6,530 | — | 6,530 | — | ||||||||||||
U.S. government securities | 144,849 | — | 144,849 | — | ||||||||||||
Total financial assets | $ | 652,818 | $ | 237,318 | $ | 415,500 | $ | — | ||||||||
December 31, 2018 | ||||||||||||||||
Total | Level 1 | Level 2 | Level 3 | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 275,234 | $ | 275,234 | $ | — | $ | — | ||||||||
Corporate debt securities | 110,027 | — | 110,027 | — | ||||||||||||
U.S. government agency securities | 88,028 | — | 88,028 | — | ||||||||||||
Certificates of deposits | 6,675 | — | 6,675 | — | ||||||||||||
U.S. government securities | 111,728 | — | 111,728 | — | ||||||||||||
Total financial assets | $ | 591,692 | $ | 275,234 | $ | 316,458 | $ | — | ||||||||
March 31, 2019 | December 31, 2018 | |||||||||||||||||||||||||||||||
Amortized Cost | Unrealized Gains | Unrealized (Losses) | Estimated Fair Value | Amortized Cost | Unrealized Gains | Unrealized (Losses) | Estimated Fair Value | |||||||||||||||||||||||||
Financial Assets: | ||||||||||||||||||||||||||||||||
Money market funds | $ | 145,413 | $ | — | $ | — | $ | 145,413 | $ | 275,234 | $ | — | $ | — | $ | 275,234 | ||||||||||||||||
Corporate debt securities | 143,664 | 278 | (15 | ) | 143,927 | 110,053 | 69 | (95 | ) | 110,027 | ||||||||||||||||||||||
U.S. government agency securities | 109,364 | 98 | (4 | ) | 109,458 | 88,042 | 40 | (54 | ) | 88,028 | ||||||||||||||||||||||
Certificates of deposits | 6,926 | 32 | (1 | ) | 6,957 | 6,681 | 1 | (7 | ) | 6,675 | ||||||||||||||||||||||
U.S. government securities | 169,043 | 194 | (6 | ) | 169,231 | 111,730 | 60 | (62 | ) | 111,728 | ||||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| |||||||||||||||||
Total | $ | 574,410 | $ | 602 | $ | (26 | ) | $ | 574,986 | $ | 591,740 | $ | 170 | $ | (218 | ) | $ | 591,692 | ||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30, 2019 | December 31, 2018 | |||||||||||||||||||||||||||||||
Amortized Cost | Unrealized Gains | Unrealized (Losses) | Estimated Fair Value | Amortized Cost | Unrealized Gains | Unrealized (Losses) | Estimated Fair Value | |||||||||||||||||||||||||
Financial Assets: | ||||||||||||||||||||||||||||||||
Money market funds | $ | 237,318 | $ | — | $ | — | $ | 237,318 | $ | 275,234 | $ | — | $ | — | $ | 275,234 | ||||||||||||||||
Corporate debt securities | 116,660 | 528 | — | 117,188 | 110,053 | 69 | (95 | ) | 110,027 | |||||||||||||||||||||||
U.S. government agency securities | 146,856 | 131 | (54 | ) | 146,933 | 88,042 | 40 | (54 | ) | 88,028 | ||||||||||||||||||||||
Certificates of deposits | 6,478 | 52 | — | 6,530 | 6,681 | 1 | (7 | ) | 6,675 | |||||||||||||||||||||||
U.S. government securities | 144,528 | 321 | — | 144,849 | 111,730 | 60 | (62 | ) | 111,728 | |||||||||||||||||||||||
Total | $ | 651,840 | $ | 1,032 | $ | (54 | ) | $ | 652,818 | $ | 591,740 | $ | 170 | $ | (218 | ) | $ | 591,692 | ||||||||||||||
March 31, 2019 | December 31, 2018 | |||||||
Cash and cash equivalents | $ | 156,744 | $ | 275,234 | ||||
Short-term marketable securities | 300,431 | 202,177 | ||||||
Long-term marketable securities | 117,811 | 114,281 | ||||||
|
|
|
| |||||
Total | $ | 574,986 | $ | 591,692 | ||||
|
|
|
|
September 30, 2019 | December 31, 2018 | |||||||
Cash and cash equivalents | $ | 237,318 | $ | 275,234 | ||||
Short-term marketable securities | 335,500 | 202,177 | ||||||
Long-term marketable securities | 80,000 | 114,281 | ||||||
Total | $ | 652,818 | $ | 591,692 | ||||
March 31, 2019 | December 31, 2018 | |||||||
Laboratory equipment | $ | 7,551 | $ | 7,363 | ||||
Computer equipment | 1,501 | 1,501 | ||||||
Leasehold improvements | 13,785 | 13,785 | ||||||
Construction-in-progress | 253 | 239 | ||||||
|
|
|
| |||||
Total property and equipment | 23,090 | 22,888 | ||||||
Less: accumulated depreciation and | (10,000 | ) | (7,907 | ) | ||||
|
|
|
| |||||
Property and equipment, net | $ | 13,090 | $ | 14,981 | ||||
|
|
|
|
September 30, 2019 | December 31, 2018 | |||||||
Laboratory equipment | $ | 8,191 | $ | 7,363 | ||||
Computer equipment | 1,979 | 1,501 | ||||||
Leasehold improvements | 13,785 | 13,785 | ||||||
Construction-in-progress | 4,575 | 239 | ||||||
Total property and equipment | 28,530 | 22,888 | ||||||
Less: accumulated depreciation and amortization | (14,193 | ) | (7,907 | ) | ||||
Property and equipment, net | $ | 14,337 | $ | 14,981 | ||||
March 31, 2019 | December 31, 2018 | |||||||
Accrued clinical and manufacturing expenses | $ | 15,126 | $ | 15,121 | ||||
Accrued professional and consulting services | 2,104 | 1,016 | ||||||
Other | 527 | 655 | ||||||
|
|
|
| |||||
Total accrued liabilities | $ | 17,757 | $ | 16,792 | ||||
|
|
|
|
September 30, 2019 | December 31, 2018 | |||||||
Accrued clinical and manufacturing expenses | $ | 17,843 | $ | 15,121 | ||||
Accrued professional and consulting services | 3,850 | 1,016 | ||||||
Other | 505 | 655 | ||||||
Total accrued liabilities | $ | 22,198 | $ | 16,792 | ||||
March 31, 2019 | December 31, 2018 | |||||||
Operating lease liabilities, current | $ | 1,252 | $ | — | ||||
Restricted shares subject to repurchase, current | 77 | 157 | ||||||
Deferred rent, current | — | 712 | ||||||
Other payable | 30 | 30 | ||||||
|
|
|
| |||||
Total other liabilities, current | $ | 1,359 | $ | 899 | ||||
|
|
|
| |||||
Deferred rent, noncurrent | $ | — | $ | 11,041 | ||||
Other liabilities, noncurrent | 30 | 30 | ||||||
|
|
|
| |||||
Total other liabilities, noncurrent | $ | 30 | $ | 11,071 | ||||
|
|
|
|
September 30, 2019 | December 31, 2018 | |||||||
Operating lease liabilities, current | $ | 1,380 | | $ | — | |||
Restricted shares subject to repurchase, current | — | 157 | ||||||
Deferred rent, current | — | 712 | ||||||
Other payable | 30 | 30 | ||||||
Total other liabilities, current | $ | 1,410 | $ | 899 | ||||
Deferred rent, noncurrent | $ | — | $ | 11,041 | ||||
Accrued lease liabilities | 4,367 | — | ||||||
Other liabilities, noncurrent | — | 30 | ||||||
Total other liabilities, noncurrent | $ | 4,367 | $ | 11,071 | ||||
Number of Options | Weighted- Average Exercise Price | |||||||
Outstanding — December 31, 2018 | 3,243,551 | $ | 29.74 | |||||
Options granted | 685,765 | 48.93 | ||||||
Options exercised | (52,288 | ) | 15.62 | |||||
Options canceled | (63,033 | ) | 37.22 | |||||
|
|
|
| |||||
Outstanding — March 31, 2019 | 3,813,995 | $ | 33.26 | |||||
|
|
|
|
Number of Options | Weighted- Average Exercise Price | |||||||
Outstanding — December 31, 2018 | 3,243,551 | $ | 29.74 | |||||
Options granted | 885,765 | 50.06 | ||||||
Options exercised | (194,239 | ) | 18.20 | |||||
Options canceled | (142,577 | ) | 41.92 | |||||
Outstanding — September 30, 2019 | 3,792,500 | $ | 34.62 | |||||
Three Months Ended March 31, | ||||
2019 | 2018 | |||
Expected term (in years) | 6.1 | 6.1 | ||
Volatility | 71.6%-72.2% | 69.1%-69.3% | ||
Risk-free interest rate | 2.5%-2.6% | 2.6%-2.7% | ||
Dividend yield | — | — |
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Expected term (in years) | 6.1 | 6.1 | 5.3-6.1 | 5.3-6.1 | ||||||||||||
Volatility | 70.4%-71.2% | 69.7%-70.3% | 70.4%-72.2% | 68.7%-70.8% | ||||||||||||
Risk-free interest rate | 1.4%-1.9% | 2.8%-2.9% | 1.4%-2.6% | 2.6%-2.9% | ||||||||||||
Dividend yield | — | — | — | — |
Number of RSUs | Weighted- Average Grant Date Fair Value | |||||||
Non-vested units — December 31, 2018 | 816,169 | $ | 43.34 | |||||
RSUs granted | 772,050 | 48.90 | ||||||
RSUs vested | (92,359 | ) | 41.16 | |||||
RSUs forfeited | (28,983 | ) | 45.94 | |||||
|
| |||||||
Non-vested units — March 31, 2019 | 1,466,877 | $ | 46.36 | |||||
|
|
Number of RSUs | Weighted- Average Grant Date Fair Value | |||||||
Non-vested units — December 31, 2018 | 816,169 | $ | 43.34 | |||||
RSUs granted | 1,150,960 | 50.84 | ||||||
RSUs vested | (295,764 | ) | 43.67 | |||||
RSUs forfeited | (96,088 | ) | 46.57 | |||||
Non-vested units — September 30, 2019 | 1,575,277 | $ | 48.56 | |||||
Three Months Ended March 31, | ||||||||
2019 | 2018 | |||||||
Research and development | $ | 4,023 | $ | 2,968 | ||||
General and administrative | 5,430 | 4,816 | ||||||
|
|
|
| |||||
Total stock-based compensation expense | $ | 9,453 | $ | 7,784 | ||||
|
|
|
|
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Research and development | $ | 5,109 | $ | 2,835 | $ | 13,847 | $ | 9,587 | ||||||||
General and administrative | 7,344 | 4,133 | 19,007 | 12,965 | ||||||||||||
Total stock-based compensation expense | $ | 12,453 | $ | 6,968 | $ | 32,854 | $ | 22,552 | ||||||||
Three Months Ended March 31, | ||||||||
2019 | 2018 | |||||||
Options to purchase common stock | 3,813,995 | 3,379,550 | ||||||
Restricted stock subject to future vesting | 22,856 | 1,040,806 | ||||||
Restricted stock units | 1,626,127 | 170,371 | ||||||
|
|
|
| |||||
Total | 5,462,978 | 4,590,727 | ||||||
|
|
|
|
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Options to purchase common stock | 3,792,500 | 3,200,529 | 3,792,500 | 3,200,529 | ||||||||||||
Restricted stock subject to future vesting | — | 71,246 | — | 71,246 | ||||||||||||
Restricted stock units | 1,685,777 | 926,244 | 1,685,777 | 926,244 | ||||||||||||
Total | 5,478,277 | 4,198,019 | 5,478,277 | 4,198,019 | ||||||||||||
As of September 30, 2019, the Substitute Premises Term has not commenced as we did not have the right to use or control physical access to the Substitute Premises. We have capitalized $4.5 million of costs in
Year ending December 31, | Amount (a) | |||
2019 (nine months) | $ | 3,364 | ||
2020 | 4,576 | |||
2021 | 4,701 | |||
2022 | 4,856 | |||
2023 | 5,017 | |||
2024 | 5,183 | |||
Thereafter | 16,114 | |||
|
| |||
Total lease payments | 43,811 | |||
Less: Imputed interest(b) | (18,158 | ) | ||
|
| |||
Present value of operating lease liabilities | $ | 25,653 | ||
|
|
Year ending December 31, | Amount (a) | |||
2019 (three months) | $ | 1,129 | ||
2020 | 4,576 | |||
2021 | 4,701 | |||
2022 | 4,856 | |||
2023 | 5,017 | |||
2024 | 5,183 | |||
Thereafter | 16,114 | |||
Total lease payments | 41,576 | |||
Less: Imputed interest (b) | (16,505 | ) | ||
Present value of operating lease liabilities | $ | 25,071 | ||
(a) |
|
(b) | Imputed interest is calculated using the interest rate for each lease. |
Year ending December 31, | Amount (c) | |||
2019 | $ | 4,406 | ||
2020 | 6,513 | |||
2021 | 11,642 | |||
2022 | 12,020 | |||
2023 | 12,409 | |||
Thereafter | 90,367 | |||
|
| |||
Total | $ | 137,357 | ||
|
|
Year ending December 31, | Amount (c) | |||
2019 | $ | 4,406 | ||
2020 | 6,513 | |||
2021 | 11,642 | |||
2022 | 12,020 | |||
2023 | 12,409 | |||
Thereafter | 90,367 | |||
Total | $ | 137,357 | ||
(c) |
|
Item 2. | MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
(“SCD”).or SCD.red blood cellRBC destruction, improvements in anemia, improvements in markers of tissue oxygenation, and reduced numbers of sickled RBCs.or FDA(“FDA”) about seeking an accelerated approval pathway for voxelotor for SCD. In December 2018, we announced that the FDA agreed with our proposal to use the accelerated regulatory approval pathway under the FDA’s Subpart H regulations, or Subpart H, for voxelotor for the treatment of SCD, and that we planplanned to submit a new drug application or NDA,(“NDA”) under this pathway to the FDA. The FDA grants accelerated approval under Subpart H for new drugs that address serious or life-threatening illnesses and that provide meaningful therapeutic benefit. Additionally, in December 2018, we announced updated efficacy and safety results from Part A of the Phase 3 HOPE Study of voxelotor at both the 1500 mg and 900 mg doses after 24 weeks of treatment versus placebo. These results, from approximately 150 patients with SCD treated with voxelotor for 24 weeks at both doses versus placebo, showed a statistically significant increase in the primary endpoint and showed improvements in other hemolysis measures. Voxelotor also continued to show a favorable safety and tolerability profile at 24 weeks. Recently,completedannounced updated results from the ongoing Phase 3 HOPE Study of voxelotor in patients ages 12 and older with SCD. The findings from 274 adolescents and adults treated with voxelotor showed the HOPE Study met its primary endpoint of an improvement in hemoglobin greater than 1 g/dL at 24 weeks with voxelotor 1500 mg compared with placebo, with apre-NDA meeting favorable safety and tolerability profile. In the study, voxelotor provided a rapid, statistically significant and sustained improvement in hemoglobin levels and reduced the incidence of worsening anemia and hemolysis. In June 2019, we also announced that we have reached final agreement with the FDA on the design of our transcranial doppler (“TCD”), post-approval confirmatory study. We expect to initiate this study in the fourth quarter of 2019.
voxelotor shortly thereafter.
In August 2018, we entered into a license agreement (the “License Agreement”) with F.the companyus an exclusive and sublicensable worldwide license under certain patent rights and
Three Months Ended March 31, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
(in thousands, except percentages) | ||||||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 34,468 | $ | 29,944 | $ | 4,524 | 15 | % | ||||||||
General and administrative | 18,055 | 12,751 | 5,304 | 42 | % | |||||||||||
|
|
|
|
|
| |||||||||||
Total operating expenses | 52,523 | 42,695 | 9,828 | 23 | % | |||||||||||
|
|
|
|
|
| |||||||||||
Loss from operations | (52,523 | ) | (42,695 | ) | (9,828 | ) | 23 | % | ||||||||
Interest income, net | 3,650 | 1,173 | 2,477 | 211 | % | |||||||||||
Other income (expenses), net | (50 | ) | (34 | ) | (16 | ) | 47 | % | ||||||||
|
|
|
|
|
| |||||||||||
Net loss | $ | (48,923 | ) | $ | (41,556 | ) | $ | (7,367 | ) | 18 | % | |||||
|
|
|
|
|
|
Three Months Ended September 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
(in thousands, except percentages) | ||||||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 39,088 | $ | 33,026 | $ | 6,062 | 18 | % | ||||||||
General and administrative | 29,654 | 12,450 | 17,204 | 138 | % | |||||||||||
Total operating expenses | 68,742 | 45,476 | 23,266 | 51 | % | |||||||||||
Loss from operations | (68,742 | ) | (45,476 | ) | (23,266 | ) | 51 | % | ||||||||
Interest income, net | 4,226 | 2,480 | 1,746 | 70 | % | |||||||||||
Other expenses, net | (31 | ) | (72 | ) | 41 | (57 | )% | |||||||||
Net loss | $ | (64,547 | ) | $ | (43,068 | ) | $ | (21,479 | ) | 50 | % | |||||
Three Months Ended March 31, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
Costs incurred by development program: | ||||||||||||||||
Voxelotor for the treatment of SCD | $ | 27,725 | $ | 23,132 | $ | 4,593 | 20 | % | ||||||||
Other preclinical programs and voxelotor for the treatment of hypoxemic pulmonary disorders | 5,363 | 6,812 | (1,449 | ) | (21 | )% | ||||||||||
Inclacumab for the treatment of SCD | 1,380 | — | 1,380 | 100 | % | |||||||||||
|
|
|
|
|
| |||||||||||
Total research and development expenses | $ | 34,468 | $ | 29,944 | $ | 4,524 | 15 | % | ||||||||
|
|
|
|
|
|
Three Months Ended September 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
Costs incurred by development program: | ||||||||||||||||
Voxelotor for the treatment of SCD | $ | 29,294 | $ | 24,656 | $ | 4,638 | 19 | % | ||||||||
Other preclinical programs | 7,664 | 5,901 | 1,763 | 30 | % | |||||||||||
Inclacumab for the treatment of SCD | 2,130 | 2,469 | (339 | ) | (14 | )% | ||||||||||
Total research and development expenses | $ | 39,088 | $ | 33,026 | $ | 6,062 | 18 | % | ||||||||
Nine Months Ended September 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
(in thousands, except percentages) | ||||||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 109,564 | $ | 94,543 | $ | 15,021 | 16 | % | ||||||||
General and administrative | 72,503 | 36,115 | 36,388 | 101 | % | |||||||||||
Total operating expenses | 182,067 | 130,658 | 51,409 | 39 | % | |||||||||||
Loss from operations | (182,067 | ) | (130,658 | ) | (51,409 | ) | 39 | % | ||||||||
Interest income, net | 11,422 | 5,768 | 5,654 | 98 | % | |||||||||||
Other expenses, net | (146 | ) | (101 | ) | (45 | ) | 45 | % | ||||||||
Net loss | $ | (170,791 | ) | $ | (124,991 | ) | $ | (45,800 | ) | 37 | % | |||||
Nine Months Ended September 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
Costs incurred by development program: | ||||||||||||||||
Voxelotor for the treatment of SCD | $ | 86,516 | $ | 72,940 | $ | 12,576 | 17 | % | ||||||||
Other preclinical programs | 18,183 | 18,575 | (392 | ) | (2 | )% | ||||||||||
Inclacumab for the treatment of SCD | 4,865 | 3,028 | 1,837 | 61 | % | |||||||||||
Total research and development expenses | $ | 109,564 | $ | 94,543 | $ | 15,021 | 16 | % | ||||||||
personnel.
General and administrative, or
the costs of obtaining clinical and commercial supplies of voxelotor, inclacumab and any other product candidates we may identify and develop;
�� | the costs of obtaining clinical and commercial supplies of voxelotor, inclacumab and any other product candidates we may identify and develop; |
Three Months Ended March 31, | ||||||||
2019 | 2018 | |||||||
Cash used in operating activities | $ | (40,316 | ) | $ | (36,766 | ) | ||
Cash provided by (used in) investing activities | (100,651 | ) | 41,574 | |||||
Cash provided by financing activities | 22,446 | 252,357 | ||||||
|
|
|
| |||||
Net increase (decrease) in cash, cash equivalents and restricted cash | $ | (118,521 | ) | $ | 257,165 | |||
|
|
|
|
Nine Months Ended September 30, | ||||||||
2019 | 2018 | |||||||
Cash used in operating activities | $ | (132,080 | ) | $ | (101,802 | ) | ||
Cash used in investing activities | (97,491 | ) | (155,870 | ) | ||||
Cash provided by financing activities | 221,818 | 255,580 | ||||||
Net decrease in cash, cash equivalents and restricted cash | $ | (7,753 | ) | $ | (2,092 | ) | ||
Cash used in operating activities for the three months ended March 31, 2018 was $36.8 million, consisting of a net loss of $41.6 million, which wasbonus payments. These were partially offset bynon-cash charges an increase of $7.8 million for stock-based compensation and $0.8 million for depreciation and amortization expense. The change in our net operating assets and liabilities was due primarily to a decrease of $3.9$5.9 million in accrued compensation primarilyliabilities due to the paymentgrowth of annual employee bonus.
our operations.
December 31, 2018 | Adjustments Due to our Adoption of Topic 842 | January 1, 2019 | ||||||||||
Assets: | ||||||||||||
Operating Lease ROU assets | $ | — | $ | 14,177 | $ | 14,177 | ||||||
Liabilities: | ||||||||||||
Operating lease liabilities, current as included in other liabilities, current | — | 1,176 | 1,176 | |||||||||
Deferred rent, current as included in other liabilities, current | 712 | (712 | ) | — | ||||||||
Operating lease liabilities, noncurrent | — | 24,754 | 24,754 | |||||||||
Deferred rent, noncurrent as included in other liabilities, noncurrent | 11,041 | (11,041 | ) | — |
December 31, 2018 | Adjustments Due to our Adoption of Topic 842 | January 1, 2019 | ||||||||||
Assets: | ||||||||||||
Operating lease ROU assets | $ | — | $ | 14,177 | $ | 14,177 | ||||||
Liabilities: | ||||||||||||
Operating lease liabilities, current as included in other liabilities, current | — | 1,176 | 1,176 | |||||||||
Deferred rent, current as included in other liabilities, current | 712 | (712 | ) | — | ||||||||
Operating lease liabilities, noncurrent | — | 24,754 | 24,754 | |||||||||
Deferred rent, noncurrent as included in other liabilities, noncurrent | 11,041 | (11,041 | ) | — |
Item 3. | Quantitative and Qualitative Disclosures about Market Risk |
Item 4. | Controls and Procedures |
Legal ProceedingsMarch 31,September 30, 2019. Based on the evaluation of our disclosure controls and procedures as of March 31,September 30, 2019, our Chief Executive Officer and Chief Financial Officer have concluded that, as of March 31,September 30, 2019, our disclosure controls and procedures were effective at the reasonable assurance level.threenine months ended March 31,September 30, 2019 that have materially affected, or are reasonably likely to materially affect, our internal controls over financial reporting.Item 1.
Item 1A. | Risk Factors. |
March 31,September 30, 2019 and December 31, 2018 and the notes accompanying those consolidated financial statements.threenine months ended March 31,September 30, 2019 and 2018 were $48.9$170.8 million and $41.6$125.0 million, respectively. As of March 31,September 30, 2019, we had an accumulated deficit of $521.1$642.9 million. We have not generated any revenue since our inception, and have financed our operations primarily through the sale of equity securities. We continue to incur significant research and development and other expenses related to our ongoing operations and expect to incur losses for the foreseeable future. We anticipate these losses will increase as we:ongoing orof voxelotor we may conduct in the future in SCD patients;patients or for any other indications for voxelotor, inclacumab or any other product candidates if any;
with SCD, which we expanded to include a new single-dose cohort in children aged
begin commercialization of voxelotor shortly thereafter.
jurisdictions (including the EMA’s PRIME program).
clinical studies and toxicology studies, and possibly additional future nonclinical studies and clinical trials to demonstrate safety and efficacy of voxelotor for SCD or any other potential indication we may pursue)Study). In addition, we will need to seek and obtain and maintain regulatory approval for SCD or any other potential indication, secure an adequate manufacturing supply to support larger clinical trials and commercial sales and build a commercial organization. Further, the success of voxelotor as a potential commercial product will also depend on patent and trade secret protection, acceptance of voxelotor by patients, the medical community and third-party payors, its ability to compete with other therapies, the status and availability of healthcare coverage and adequate reimbursement, and maintenance of an acceptable safety and efficacy profile following approval, among other factors. If we do not achieve all of these factors in a timely manner or at all, we could experience significant delays or an inability to successfully commercialize voxelotor, which would materially harm our business.
We are also generating additional clinical data regarding voxelotor in SCD patients in our OLE studies and our EAP.
In June 2019, we announced new results from the HOPE Study from 274 adolescents and adults with SCD treated with voxelotor. These findings showed the HOPE Study met its primary endpoint of an improvement in hemoglobin greater than 1 g/dL at 24 weeks with voxelotor 1500 mg compared with placebo, with a favorable safety and tolerability profile. In the study, voxelotor provided a rapid, statistically significant and sustained improvement in hemoglobin levels and reduced the incidence of worsening anemia and hemolysis.
In addition, data and results from later studies or programs may conflict with earlier findings.
delays or failures to receive approval for conduct of clinical studies in one or more geographies, which could result in delays in enrollment and availability of data and results;
directly with us, without payment to us, or result in our inability to manufacture or commercialize drugs without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize voxelotor, inclacumab or any future product candidates.
patent rights covering voxelotor and certain voxelotor analogs, some of which patent rights we jointly own with the Regents. Additionally, in the United States, each
candidates, and we currently have no sales organization.candidates. To successfully commercialize voxelotor or any other products that may result from our development programs, we will need to develop commercial capabilities, either on our own or with others. We are building the infrastructure required for commercialization of our lead product candidate voxelotor to prepare for potential launch.commercialization in the first half of 2020. This includes establishing a sales and marketing organization with technical expertise and supporting distribution capabilities to commercialize our product candidatesvoxelotor in major markets,the United States, which will beis expensive, difficult, risky and time consuming. Any failure or delay in the development of our internal sales, marketing, and distribution capabilities would adversely impact the commercialization of ourvoxelotor or any other products, if any are approved.
retail prices, the reporting of prices used to calculate Medicaid rebate information and other information affecting federal, state and third-party product reimbursement are subject to scrutiny under this law. The civil False Claims Act has been used to assert liability on the basis of, among other things, kickbacks and other improper referrals, improperly reported government pricing metrics such as Best Price
off-label uses not expressly approved by the FDA in a product’s label), and allegations as to misrepresentations with respect to the services rendered. Intent to deceive is not required to establish liability under the civil False Claims Act. Over time, False Claims Act lawsuits against biopharmaceutical companies have increased significantly in volume and breadth, leading to multiple substantial civil and criminal settlements regarding sales practices and promoting off label uses. Further, the government may further prosecute conduct constituting a false claim under the criminal False Claims Act; |
purchasers of the manufacturers’ products are appropriately licensed. Further, manufacturers have product investigation, quarantine, disposition, and FDA and trading partner notification responsibilities related to counterfeit, diverted, stolen, and intentionally adulterated products that would result in serious adverse health consequences or death to humans, as well as products that are the subject of fraudulent transactions or which are otherwise unfit for distribution such that they would be reasonably likely to result in serious health consequences or death.
individuals in the United States as a result of unemployment, underemployment or the potential repeal of certain provisions of the ACA, may decrease the demand for healthcare services and pharmaceuticals. If fewer patients are seeking medical care because they do not have insurance coverage, we may experience difficulties in any eventual commercialization of our product candidates and our business, results of operations, financial condition and cash flows could be adversely affected.
example, negative publicity regarding drug pricing and price increases by pharmaceutical companies has negatively impacted, and may continue to negatively impact, the markets for biotechnology and pharmaceutical stocks. In the past, following periods of volatility in the market, securities class-action litigation has often been instituted against companies. Such litigation, if instituted against us, could result in substantial costs and diversion of management’s attention and resources, which could materially and adversely affect our business, financial condition, results of operations and growth prospects.
prior calendar year, subject to the ability of our board of directors or compensation committee to take action to reduce the size of the increase in any given year. In addition, in January 2017 our board of directors approved our 2017 Inducement Equity Plan (“2017 Inducement Plan”). The 2017 Inducement Plan enables us and our subsidiaries to grantMarch 31,September 30, 2019, the number of shares reserved for grant under the 2017 Inducement Plan was 243,325190,775 shares, subject to adjustment for reorganization, recapitalization, stock dividend, stock split, or similar changes in our capital stock. In addition, we have reserved shares of common stock for issuance pursuant to our 2015 Employee Stock Purchase Plan (“2015 ESPP”), which number of shares will automatically increase each year on January 1, from January 1, 2016 to January 1, 2025, by the lesser of (i) 3,000,000 shares of common stock, (ii) 1% of all shares of our capital stock outstanding as of December 31 of the prior calendar year, or (iii) such lesser number of shares as determined by the administrator of our 2015 ESPP. Currently, we plan to register the increased number of shares available for issuance under the 2015 Plan and the 2015 ESPP each year. If our board of directors elects to increase the number of shares available for future grant under the 2015 Plan, the 2017 Inducement Plan or the 2015 ESPP, our stockholders may experience additional dilution, and our stock price may fall.
Item 2. | Unregistered Sales of Equity Securities and Use of Proceeds |
a) |
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b) |
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Item 6. | Exhibits |
Exhibit Number | Exhibit Description | Incorporated by Reference | Filed Herewith | |||||||||||||||||
Form | Date | Number | ||||||||||||||||||
3.1 | S-1/A | 7/31/2015 | 3.2 | |||||||||||||||||
3.2 | S-1/A | 7/31/2015 | 3.4 | |||||||||||||||||
4.1 | S-1/A | 7/31/2015 | 4.1 | |||||||||||||||||
10.1# | X | |||||||||||||||||||
31.1 | X | |||||||||||||||||||
31.2 | X | |||||||||||||||||||
32.1* | X | |||||||||||||||||||
101.SCH | Inline XBRL Taxonomy Extension Schema Document | X | ||||||||||||||||||
101.CAL | Inline XBRL Taxonomy Extension Calculation Linkbase Document | X | ||||||||||||||||||
101.DEF | Inline XBRL Taxonomy Extension Definition Linkbase Document. | X | ||||||||||||||||||
101.LAB | Inline XBRL Taxonomy Extension Label Linkbase Document. | X | ||||||||||||||||||
101.PRE | Inline XBRL Taxonomy Extension Presentation Linkbase Document. | X | ||||||||||||||||||
104 | Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101.) | X |
# | Represents a management compensation plan, contract or arrangement. Senior Management Team Members have been designated by the Compensation Committee of the Company’s Board of Directors and include all of the Company’s executive officers (other than the Chief Executive Officer), who shall each continue to be considered Senior Management Team Members for purposes of Change in Control Benefits so long as they are employed with the Company in any capacity. |
* | The certification attached as Exhibit 32.1 that accompanies this Quarterly Report on Form |
Global Blood Therapeutics, Inc. | ||||||
Date: | By: | /s/ Ted W. Love, M.D. | ||||
Ted W. Love, M.D. President and Chief Executive Officer (Principal Executive Officer) | ||||||
Date: | By: | /s/ Jeffrey Farrow | ||||
Jeffrey Farrow Chief Financial Officer (Principal Financial Officer) |
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