☒ | 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
Large accelerated filer | ☒ | Accelerated filer | ☐ | |||
Non-accelerated filer | ☐ | Smaller reporting company | ☐ | |||
Emerging growth company | ☐ |
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 |
As
Item 1. Item 2. Item 3. Item Item 3. Item 4. Item 5. Item 6. 3 3 3 4 5 6 7 15 21Item 4.Controls and Procedures2121Item 1.Legal Proceedings21Item 1A.Risk Factors 22 2. 22 22 Item 1. 22 Item 1A. 23 Item 2. 5960 5960 5960 5960 5960 6162
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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June 30, 2019 | December 31, 2018 | |||||||
(Unaudited) | ||||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 338,454 | $ | 275,357 | ||||
Short-term marketable securities | 320,098 | 202,177 | ||||||
Prepaid expenses and other current assets | 8,207 | 8,246 | ||||||
Total current assets | 666,759 | 485,780 | ||||||
Property and equipment, net | 12,345 | 14,981 | ||||||
Long-term marketable securities | 73,176 | 114,281 | ||||||
Operating lease r ight-of-use assets | 13,964 | — | ||||||
Restricted cash | 2,395 | 2,395 | ||||||
Other assets, noncurrent | 193 | 206 | ||||||
Total assets | $ | 768,832 | $ | 617,643 | ||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 6,755 | $ | 6,046 | ||||
Accrued liabilities | 23,369 | 16,792 | ||||||
Accrued compensation | 8,241 | 10,036 | ||||||
Other liabilities, current | 1,348 | 899 | ||||||
Total current liabilities | 39,713 | 33,773 | ||||||
Operating lease liabilities, noncurrent | 24,048 | — | ||||||
Other liabilities, noncurrent | 881 | 11,071 | ||||||
Total liabilities | 64,642 | 44,844 | ||||||
Commitments and contingencies | ||||||||
Stockholders’ equity: | ||||||||
Preferred stock, $0.001 par value, 5,000,000 shares authorized as of June 30, 2019 (unaudited) and December 31, 2018; no shares issued and outstanding | — | — | ||||||
Common stock, $0.001 par value, 150,000,000 shares authorized as of June 30, 2019 (unaudited) and December 31, 2018, respectively; 59,906,199 and 55,640,299 shares issued and outstanding as of June 30, 2019 (unaudited) and December 31, 2018, respectively | 60 | 56 | ||||||
Additional paid-in capital | 1,281,333 | 1,044,941 | ||||||
Accumulated other comprehensive income (loss) | 1,191 | (48 | ) | |||||
Accumulated deficit | (578,394 | ) | (472,150 | ) | ||||
Total stockholders’ equity | 704,190 | 572,799 | ||||||
Total liabilities and stockholders’ equity | $ | 768,832 | $ | 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 June 30, | Six Months Ended June 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 36,010 | $ | 31,573 | $ | 70,476 | $ | 61,517 | ||||||||
General and administrative | 24,794 | 10,914 | 42,849 | 23,665 | ||||||||||||
Total operating expenses | 60,804 | 42,487 | 113,325 | 85,182 | ||||||||||||
Loss from operations | (60,804 | ) | (42,487 | ) | (113,325 | ) | (85,182 | ) | ||||||||
Other income (expense): | ||||||||||||||||
Interest income, net | 3,546 | 2,115 | 7,196 | 3,287 | ||||||||||||
Other income (expenses), net | (63 | ) | 4 | (115 | ) | (29 | ) | |||||||||
Total other income, net | 3,483 | 2,119 | 7,081 | 3,258 | ||||||||||||
Net loss | (57,321 | ) | (40,368 | ) | (106,244 | ) | (81,924 | ) | ||||||||
Other comprehensive loss: | ||||||||||||||||
Net unrealized gain on marketable securities, net of tax | 615 | 82 | 1,239 | 94 | ||||||||||||
Comprehensive loss | $ | (56,706 | ) | $ | (40,286 | ) | $ | (105,005 | ) | $ | (81,830 | ) | ||||
Basic and diluted net loss per common share | $ | (1.01 | ) | $ | (0.78 | ) | $ | (1.88 | ) | $ | (1.65 | ) | ||||
Weighted-average number of shares used in computing basic and diluted net loss per common share | 56,539,760 | 51,742,904 | 56,386,560 | 49,767,633 | ||||||||||||
4
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 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 | ) | |||||||||||||||||
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 | |||||||||||||
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 | ) | |||||||||||||||||
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 | ||||||||||||
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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Six Months Ended June 30, | ||||||||
2019 | 2018 | |||||||
CASH FLOWS FROM OPERATING ACTIVITIES: | ||||||||
Net loss | $ | (106,244 | ) | $ | (81,924 | ) | ||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||
Depreciation and amortization | 4,187 | 1,342 | ||||||
Amortization (accretion) of premium (discount) on marketable securities | (1,204 | ) | 107 | |||||
Amortization of operating lease right-of-use assets | 212 | — | ||||||
Stock-based compensation | 20,401 | 15,584 | ||||||
Changes in operating assets and liabilities: | ||||||||
Prepaid expenses and other assets | 59 | (2,419 | ) | |||||
Accounts payable | 558 | (3,717 | ) | |||||
Accrued liabilities | 6,136 | 1,894 | ||||||
Accrued compensation | (1,795 | ) | (2,339 | ) | ||||
Operating lease liabilities | (563 | ) | — | |||||
Other liabilities | — | 572 | ||||||
Net cash used in operating activities | (78,253 | ) | (70,900 | ) | ||||
CASH FLOWS FROM INVESTING ACTIVITIES: | ||||||||
Purchase of property and equipment | (548 | ) | (3,300 | ) | ||||
Purchase of marketable securities | (195,297 | ) | (112,558 | ) | ||||
Maturities of marketable securities | 120,923 | 76,778 | ||||||
Net cash used in investing activities | (74,922 | ) | (39,080 | ) | ||||
CASH FLOWS FROM FINANCING ACTIVITIES: | ||||||||
Proceeds from issuance of common stock in public offering, net of issuance costs | 213,989 | 255,119 | ||||||
Proceeds from issuance of common stock in settlement of employee stock purchase plan and exercise of stock options | 4,266 | 3,896 | ||||||
Tax paid related to net share settlement of equity awards | (1,983 | ) | (5,404 | ) | ||||
Net cash provided by financing activities | 216,272 | 253,611 | ||||||
Net increase in cash, cash equivalents and restricted cash | 63,097 | 143,631 | ||||||
Cash, cash equivalents and restricted cash at beginning of period | 277,752 | 199,378 | ||||||
Cash, cash equivalents and restricted cash at end of period | $ | 340,849 | $ | 343,009 | ||||
SUPPLEMENTAL DISCLOSURES OF NON-CASH INVESTING AND FINANCING INFORMATION: | ||||||||
Leasehold improvements paid for by landlord | $ | 851 | $ | — | ||||
Accrued purchase of property and equipment | $ | 147 | $ | (1,401 | ) | |||
Accrued offering costs | $ | 446 | $ | — | ||||
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 | — | ||||||||||||
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Total financial assets | $ | 574,986 | $ | 145,413 | $ | 429,573 | $ | — | ||||||||
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June 30, 2019 | ||||||||||||||||
Total | Level 1 | Level 2 | Level 3 | |||||||||||||
Financial Assets: | ||||||||||||||||
Money market funds | $ | 333,452 | $ | 333,452 | $ | — | $ | — | ||||||||
Corporate debt securities | 134,311 | — | 134,311 | — | ||||||||||||
U.S. government agency securities | 112,367 | — | 112,367 | — | ||||||||||||
Certificates of deposits | 6,758 | — | 6,758 | — | ||||||||||||
U.S. government securities | 144,838 | — | 144,838 | — | ||||||||||||
Total financial assets | $ | 731,726 | $ | 333,452 | $ | 398,274 | $ | — | ||||||||
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 | ||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June 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 | $ | 333,452 | $ | — | $ | — | $ | 333,452 | $ | 275,234 | $ | — | $ | — | $ | 275,234 | ||||||||||||||||
Corporate debt securities | 133,775 | 536 | — | 134,311 | 110,053 | 69 | (95 | ) | 110,027 | |||||||||||||||||||||||
U.S. government agency securities | 112,169 | 200 | (2 | ) | 112,367 | 88,042 | 40 | (54 | ) | 88,028 | ||||||||||||||||||||||
Certificates of deposits | 6,714 | 44 | — | 6,758 | 6,681 | 1 | (7 | ) | 6,675 | |||||||||||||||||||||||
U.S. government securities | 144,425 | 413 | — | 144,838 | 111,730 | 60 | (62 | ) | 111,728 | |||||||||||||||||||||||
Total | $ | 730,535 | $ | 1,193 | $ | (2 | ) | $ | 731,726 | $ | 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 | ||||
|
|
|
|
June 30, 2019 | December 31, 2018 | |||||||
Cash and cash equivalents | $ | 338,452 | $ | 275,234 | ||||
Short-term marketable securities | 320,098 | 202,177 | ||||||
Long-term marketable securities | 73,176 | 114,281 | ||||||
Total | $ | 731,726 | $ | 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 | ||||
|
|
|
|
June 30, 2019 | December 31, 2018 | |||||||
Laboratory equipment | $ | 7,935 | $ | 7,363 | ||||
Computer equipment | 1,501 | 1,501 | ||||||
Leasehold improvements | 13,785 | 13,785 | ||||||
Construction-in-progress | 1,213 | 239 | ||||||
Total property and equipment | 24,434 | 22,888 | ||||||
Less: accumulated depreciation and amortization | (12,089 | ) | (7,907 | ) | ||||
Property and equipment, net | $ | 12,345 | $ | 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 | ||||
|
|
|
|
June 30, 2019 | December 31, 2018 | |||||||
Accrued clinical and manufacturing expenses | $ | 18,602 | $ | 15,121 | ||||
Accrued professional and consulting services | 4,336 | 1,016 | ||||||
Other | 431 | 655 | ||||||
Total accrued liabilities | $ | 23,369 | $ | 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 | ||||
|
|
|
|
June 30, 2019 | December 31, 2018 | |||||||
Operating lease liabilities, current | $ | 1,318 | $ | — | ||||
Restricted shares subject to repurchase, current | — | 157 | ||||||
Deferred rent, current | — | 712 | ||||||
Other payable | 30 | 30 | ||||||
Total other liabilities, current | $ | 1,348 | $ | 899 | ||||
Deferred rent, noncurrent | $ | — | $ | 11,041 | ||||
Accrued lease liabilities | 851 | — | ||||||
Other liabilities, noncurrent | 30 | 30 | ||||||
Total other liabilities, noncurrent | $ | 881 | $ | 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 | 807,965 | 50.15 | ||||||
Options exercised | (175,801 | ) | 17.91 | |||||
Options canceled | (116,869 | ) | 41.01 | |||||
Outstanding — June 30, 2019 | 3,758,846 | $ | 34.33 | |||||
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 June 30, | Six Months Ended June 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Expected term (in years) | 5.3-6.1 | 5.3-6.1 | 5.3-6.1 | 5.3-6.1 | ||||||||||||
Volatility | 70.5%-71.7% | 68.7%-70.8% | 70.5%-72.2% | 68.7%-70.8% | ||||||||||||
Risk-free interest rate | 1.8%-2.4% | 2.6%-2.9% | 1.8%-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 | 995,385 | 50.77 | ||||||
RSUs vested | (116,389 | ) | 41.74 | |||||
RSUs forfeited | (75,317 | ) | 46.01 | |||||
Non-vested units — June 30, 2019 | 1,619,848 | $ | 47.90 | |||||
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 June 30, | Six Months Ended June 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Research and development | $ | 4,715 | $ | 3,784 | $ | 8,738 | $ | 6,752 | ||||||||
General and administrative | 6,233 | 4,016 | 11,663 | 8,832 | ||||||||||||
Total stock-based compensation expense | $ | 10,948 | $ | 7,800 | $ | 20,401 | $ | 15,584 | ||||||||
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 June 30, | Six Months Ended June 30, | |||||||||||||||
2019 | 2018 | 2019 | 2018 | |||||||||||||
Options to purchase common stock | 3,758,846 | 3,297,839 | 3,758,846 | 3,297,839 | ||||||||||||
Restricted stock subject to future vesting | — | 97,037 | — | 97,037 | ||||||||||||
Restricted stock units | 1,730,348 | 1,002,766 | 1,730,348 | 1,002,766 | ||||||||||||
Total | 5,489,194 | 4,397,642 | 5,489,194 | 4,397,642 | ||||||||||||
As of June 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 $1.0 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 (six months) | $ | 2,247 | ||
2020 | 4,576 | |||
2021 | 4,701 | |||
2022 | 4,856 | |||
2023 | 5,017 | |||
2024 | 5,183 | |||
Thereafter | 16,114 | |||
Total lease payments | 42,694 | |||
Less: Imputed interest (b) | (17,328 | ) | ||
Present value of operating lease liabilities | $ | 25,366 | ||
(a) | Operating lease payments exclude $121.5 million of legally binding minimum lease payments for Lease Amendment, which has been signed by us but has not yet commenced. |
(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) | The table above is prepared under the assumption that the Substitute Premises Commencement Date at the Substitute Premises starts on June 30, 2020. |
Item 2. | MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
(“SCD”). If we succeed in obtaining FDA review and approval of our NDA for voxelotor, we expect to begin commercialization of voxelotor in the first half of 2020.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 plan 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,In June 2019, we announced 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 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 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 second half of 2019. Previously, we disclosed that we completed aforwith the voxelotor programFDA, and that we expect to file our NDA infor the treatment of SCD no later than the second half of 2019.evaluatingcontinuing to evaluate the safety and pharmacokinetics of single and multiple doses of voxelotor in adolescent and pediatric patients with SCD in a Phase 2a clinical trial, which we call the HOPE-KIDS 1 Study.
In August 2018, we entered into a license agreement (the “License Agreement”) with F.sickle cell diseaseSCD indication, including up to $40.5 million based on achievement of certain clinical development and regulatory milestones for inclacumab in the sickle cell disease indication, and up to $85.0 million based on achievement of certain thresholds for annual net sales of inclacumab. We will also pay Roche up to an additional $5.5 million in milestone payments, which are owed to a third party, based on achievement of such clinical development and regulatory milestones for inclacumab. We will also pay Roche up to $19.25 million in milestone payments based on achievement of certain clinical development and regulatory milestones for inclacumab for any other indication than the sickle cell diseaseSCD indication. We have the right to sublicense our rights under the License Agreement to our affiliates without Roche’s consent. Subject to certain conditions and limitations, including Roche’s right of first negotiation described below, we will also have the right to sublicense our rights under the License Agreement tobe ablesubmit an Investigational New Drug application to leverage the safety data from Roche’s prior clinical studies, which were notFDA for inclacumab in patients with SCD, as we proceed with our development of inclacumab.
2021.
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 June 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
(in thousands, except percentages) | ||||||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 36,010 | $ | 31,573 | $ | 4,437 | 14 | % | ||||||||
General and administrative | 24,794 | 10,914 | 13,880 | 127 | % | |||||||||||
Total operating expenses | 60,804 | 42,487 | 18,317 | 43 | % | |||||||||||
Loss from operations | (60,804 | ) | (42,487 | ) | (18,317 | ) | 43 | % | ||||||||
Interest income, net | 3,546 | 2,115 | 1,431 | 68 | % | |||||||||||
Other income (expenses), net | (63 | ) | 4 | (67 | ) | * | ||||||||||
Net loss | $ | (57,321 | ) | $ | (40,368 | ) | $ | (16,953 | ) | 42 | % | |||||
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 June 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
Costs incurred by development program: | ||||||||||||||||
Voxelotor for the treatment of SCD | $ | 29,490 | $ | 24,888 | $ | 4,602 | 18 | % | ||||||||
Other preclinical programs and voxelotor for the treatment of hypoxemic pulmonary disorders | 5,165 | 6,127 | (962 | ) | (16 | )% | ||||||||||
Inclacumab for the treatment of SCD | 1,355 | 558 | 797 | 143 | % | |||||||||||
Total research and development expenses | $ | 36,010 | $ | 31,573 | $ | 4,437 | 14 | % | ||||||||
personnel.
Six Months Ended June 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
(in thousands, except percentages) | ||||||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | $ | 70,476 | $ | 61,517 | $ | 8,959 | 15 | % | ||||||||
General and administrative | 42,849 | 23,665 | 19,184 | 81 | % | |||||||||||
Total operating expenses | 113,325 | 85,182 | 28,143 | 33 | % | |||||||||||
Loss from operations | (113,325 | ) | (85,182 | ) | (28,143 | ) | 33 | % | ||||||||
Interest income, net | 7,196 | 3,287 | 3,909 | 119 | % | |||||||||||
Other expenses, net | (115 | ) | (29 | ) | (86 | ) | 297 | % | ||||||||
Net loss | $ | (106,244 | ) | $ | (81,924 | ) | $ | (24,320 | ) | 30 | % | |||||
Six Months Ended June 30, | $ Change | % Change | ||||||||||||||
2019 | 2018 | |||||||||||||||
Costs incurred by development program: | ||||||||||||||||
Voxelotor for the treatment of SCD | $ | 57,214 | $ | 48,019 | $ | 9,195 | 19 | % | ||||||||
Other preclinical programs and voxelotor for the treatment of hypoxemic pulmonary disorders | 10,527 | 12,940 | (2,413 | ) | (19 | )% | ||||||||||
Inclacumab for the treatment of SCD | 2,735 | 558 | 2,177 | 390 | % | |||||||||||
Total research and development expenses | $ | 70,476 | $ | 61,517 | $ | 8,959 | 15 | % | ||||||||
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 | |||
|
|
|
|
Six Months Ended June 30, | ||||||||
2019 | 2018 | |||||||
Cash used in operating activities | $ | (78,253 | ) | $ | (70,900 | ) | ||
Cash used in investing activities | (74,922 | ) | (39,080 | ) | ||||
Cash provided by financing activities | 216,272 | 253,611 | ||||||
Net increase in cash, cash equivalents and restricted cash | $ | 63,097 | $ | 143,631 | ||||
bonuses.
bonus payments. These were partially offset by an increase of $1.9 million in accrued liabilities due to the growth of 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,June 30, 2019. Based on the evaluation of our disclosure controls and procedures as of March 31,June 30, 2019, our Chief Executive Officer and Chief Financial Officer have concluded that, as of March 31,June 30, 2019, our disclosure controls and procedures were effective at the reasonable assurance level.March 31,June 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,June 30, 2019 and December 31, 2018 and the notes accompanying those consolidated financial statements.threesix months ended March 31,June 30, 2019 and 2018 were $48.9$106.2 million and $41.6$81.9 million, respectively. As of March 31,June 30, 2019, we had an accumulated deficit of $521.1$578.4 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 agedU.S. Food and Drug Administration (“FDA”)FDA agreed with our proposal to use an accelerated regulatory approval pathway for voxelotor for the treatment of SCD under Subpart H. We expect to file our NDA for voxelotor for the FDA’s Subpart H regulations (“Subpart H”),treatment of SCD no later than the second half of 2019. If we succeed in obtaining FDA review and thatapproval of our NDA for voxelotor, we planexpect to submit an NDA under this pathway.
begin commercialization of voxelotor in the first half of 2020.
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.
In addition, in connection with our NDA for voxelotor for the treatment of SCD,
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
noa limited sales organization. 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.ourvoxelotor or any other product candidates.candidates, if any. As such, we may be required to hire substantially more sales representatives to adequately support the commercialization of our product candidates or we may incur excess costs as a result of hiring more sales representatives than necessary. With respect to certain geographical markets, we may enter into collaborations with other entities to utilize their local marketing and distribution capabilities, but we may be unable to enter into such agreements on favorable terms, if at all. If our future collaborators do not commit sufficient resources to commercialize our future products, if any, and we are unable to develop the necessary marketing capabilities on our own, we will be unable to generate sufficient product revenue to sustain our business. We may be competing with companies that currently have extensive and well-funded marketing and sales operations. For example, Novartis has announced that the FDA has accepted for review an approval application for the biologic crizanlizumab for the potential treatment of patients with SCD, with a potential approval in the United States on or before January 15, 2020, and that they have submitted crizanlizumab for a conditional approval by the EMA, for potential approval in the third quarter of 2020. Without an internal team or the support of a third party to perform marketing and sales functions, we would be unable to compete successfully against more established companies.
willfully falsifying, concealing or covering up a material fact or making any materially false statement, in connection with the delivery of, or payment for, healthcare benefits, items or services; similar to the U.S. federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; |
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.
Item 2. | Unregistered Sales of Equity Securities and Use of Proceeds |
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b) |
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c) |
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Item 3. | Defaults Upon Senior Securities |
Item 4. | Mine Safety Disclosures |
Item 5. | Other Information |
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 management compensation plan, contract or arrangement. |
* | The certification attached as Exhibit 32.1 that accompanies this Quarterly Report on Form |
Global Blood Therapeutics, Inc. | ||||||||
Date: August 7, 2019 | By: | /s/ Ted W. Love, M.D. | ||||||
Ted W. Love, M.D. President and Chief Executive Officer (Principal Executive Officer) | ||||||||
Date: August 7, 2019 | By: | /s/ Jeffrey Farrow | ||||||
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Jeffrey Farrow | ||||||||
Chief Financial Officer (Principal Financial Officer) |
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