Delaware | 71-0869350 | |
(State or Other Jurisdiction of | (I.R.S. Employer | |
Incorporation or Organization) | Identification Number) | |
1 Cedar Brook Drive, Cranbury, NJ | 08512 | |
(Address of Principal Executive Offices) | (Zip Code) | |
(609) 662-2000 | ||
(Registrant’s Telephone Number, Including Area Code) |
Large accelerated filer x | Accelerated filer o | |
Non-accelerated filer o | Smaller reporting company o | |
Emerging growth company o |
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• | the progress and results of our preclinical and clinical trials of our drug candidates; |
• | the cost of manufacturing drug supply for our clinical and preclinical studies, including the cost of manufacturing Pompe Enzyme Replacement Therapy (“ERT”); |
• | the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates including those testing the use of pharmacological chaperones co-formulated and co-administered with ERT and for the treatment of lysosomal storage disorders; |
• | the future results of on-going preclinical research and subsequent clinical trials for cyclin-dependent kinase-like 5 (“CDKL5”) deficiency, including our ability to obtain regulatory approvals and commercialize CDKL5 therapies and obtain market acceptance for such therapies; |
• | the costs, timing and outcome of regulatory review of our product candidates; |
• | the number and development requirements of other product candidates that we pursue; |
• | the costs of commercialization activities, including product marketing, sales and distribution; |
• | the emergence of competing technologies and other adverse market developments; |
• | our ability to successfully commercialize migalastat HCl ; |
• | our ability to manufacture or supply sufficient clinical or commercial products; |
• | our ability to obtain reimbursement for migalastat HCl; |
• | our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of migalastat HCl; |
• | our ability to obtain market acceptance of migalastat HCl; |
• | the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing and defending intellectual property-related claims; |
• | the extent to which we acquire or invest in businesses, products and technologies; |
• | our ability to successfully integrate our acquired products and technologies into our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected; |
• | our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators; |
• | fluctuations in foreign currency exchange rates; and |
• | changes in accounting standards. |
September 30, | December 31, | ||||||
2018 | 2017 | ||||||
Assets | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 201,827 | $ | 49,060 | |||
Investments in marketable securities | 362,556 | 309,502 | |||||
Accounts receivable | 14,189 | 9,464 | |||||
Inventories | 6,311 | 4,623 | |||||
Prepaid expenses and other current assets | 16,151 | 19,316 | |||||
Total current assets | 601,034 | 391,965 | |||||
Property and equipment, less accumulated depreciation of $15,483 and $12,515 at September 30, 2018 and December 31, 2017, respectively | 10,659 | 9,062 | |||||
In-process research & development | 23,000 | 23,000 | |||||
Goodwill | 197,797 | 197,797 | |||||
Other non-current assets | 6,099 | 5,200 | |||||
Total Assets | $ | 838,589 | $ | 627,024 | |||
Liabilities and Stockholders’ Equity | |||||||
Current liabilities: | |||||||
Accounts payable, accrued expenses, and other current liabilities | $ | 54,330 | $ | 53,890 | |||
Deferred reimbursements | 2,750 | 7,750 | |||||
Contingent consideration payable | 8,800 | 8,400 | |||||
Total current liabilities | 65,880 | 70,040 | |||||
Deferred reimbursements | 14,156 | 14,156 | |||||
Convertible notes | 172,186 | 164,167 | |||||
Senior secured term loan | 146,622 | — | |||||
Contingent consideration payable | 19,300 | 17,000 | |||||
Deferred income taxes | 6,465 | 6,465 | |||||
Other non-current liabilities | 3,029 | 2,346 | |||||
Total liabilities | 427,638 | 274,174 | |||||
Commitments and contingencies | |||||||
Stockholders’ equity: | |||||||
Common stock, $0.01 par value, 500,000,000 and 250,000,000 shares authorized, 189,254,341 and 166,989,790 shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively | 1,941 | 1,721 | |||||
Additional paid-in capital | 1,731,174 | 1,400,758 | |||||
Accumulated other comprehensive loss: | |||||||
Foreign currency translation adjustment | (875 | ) | (1,659 | ) | |||
Unrealized gain on available-for-sale securities | (211 | ) | (436 | ) | |||
Warrants | 13,063 | 16,076 | |||||
Accumulated deficit | (1,334,141 | ) | (1,063,610 | ) | |||
Total stockholders’ equity | 410,951 | 352,850 | |||||
Total Liabilities and Stockholders’ Equity | $ | 838,589 | $ | 627,024 |
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Revenue: | |||||||||||||||
Net product sales | $ | 20,596 | $ | 10,874 | $ | 58,601 | $ | 22,201 | |||||||
Cost of goods sold | 4,310 | 1,790 | 10,060 | 3,626 | |||||||||||
Gross profit | 16,286 | 9,084 | 48,541 | 18,575 | |||||||||||
Operating expenses: | |||||||||||||||
Research and development | 138,227 | 40,641 | 213,685 | 103,502 | |||||||||||
Selling, general and administrative | 31,867 | 21,647 | 88,435 | 60,090 | |||||||||||
Changes in fair value of contingent consideration payable | 1,300 | (244,250 | ) | 2,700 | (238,622 | ) | |||||||||
Loss on impairment of assets | — | 465,427 | — | 465,427 | |||||||||||
Depreciation | 1,073 | 851 | 3,015 | 2,486 | |||||||||||
Total operating expenses | 172,467 | 284,316 | 307,835 | 392,883 | |||||||||||
Loss from operations | (156,181 | ) | (275,232 | ) | (259,294 | ) | (374,308 | ) | |||||||
Other income (expense): | |||||||||||||||
Interest income | 2,721 | 1,190 | 7,371 | 2,702 | |||||||||||
Interest expense | (4,715 | ) | (4,351 | ) | (13,763 | ) | (12,820 | ) | |||||||
Change in fair value of derivatives | — | — | (2,739 | ) | 163 | ||||||||||
Other (expense) income | (1,039 | ) | 2,044 | (3,593 | ) | 4,891 | |||||||||
Loss before income tax | (159,214 | ) | (276,349 | ) | (272,018 | ) | (379,372 | ) | |||||||
Income tax benefit | 51 | 164,683 | 1,104 | 164,578 | |||||||||||
Net loss attributable to common stockholders | $ | (159,163 | ) | $ | (111,666 | ) | $ | (270,914 | ) | $ | (214,794 | ) | |||
Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.84 | ) | $ | (0.69 | ) | $ | (1.47 | ) | $ | (1.44 | ) | |||
Weighted-average common shares outstanding — basic and diluted | 189,162,841 | 160,796,841 | 184,606,790 | 148,963,864 |
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Net loss | $ | (159,163 | ) | $ | (111,666 | ) | $ | (270,914 | ) | $ | (214,794 | ) | |||
Other comprehensive (loss) gain: | |||||||||||||||
Foreign currency translation adjustment gain (loss), net of tax impact of $59, $0, $168, $0, respectively | 665 | (1,176 | ) | 1,167 | (3,312 | ) | |||||||||
Unrealized gain (loss) on available-for-sale securities | 244 | (131 | ) | 225 | (203 | ) | |||||||||
Other comprehensive income (loss) | $ | 909 | $ | (1,307 | ) | $ | 1,392 | $ | (3,515 | ) | |||||
Comprehensive loss | $ | (158,254 | ) | $ | (112,973 | ) | $ | (269,522 | ) | $ | (218,309 | ) |
Nine Months Ended September 30, | |||||||
2018 | 2017 | ||||||
Operating activities | |||||||
Net loss | $ | (270,914 | ) | $ | (214,794 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||
Amortization of debt discount and deferred financing | 7,868 | 7,179 | |||||
Depreciation | 3,015 | 2,486 | |||||
Stock-based compensation | 20,873 | 17,067 | |||||
Loss on impairment | — | 465,427 | |||||
Gain on disposal of asset | — | (8 | ) | ||||
Change in fair value of derivatives | 2,739 | (265 | ) | ||||
Non-cash changes in the fair value of contingent consideration payable | 2,700 | (238,622 | ) | ||||
Foreign currency remeasurement (gain)/ loss | 775 | (4,932 | ) | ||||
Non-cash deferred taxes | — | (164,585 | ) | ||||
Changes in operating assets and liabilities: | |||||||
Accounts receivable | (5,182 | ) | (4,288 | ) | |||
Inventories | (2,049 | ) | (3,386 | ) | |||
Prepaid expenses and other current assets | 2,633 | (3,358 | ) | ||||
Other non-current assets | (1,101 | ) | 344 | ||||
Account payable and accrued expenses | 3,684 | 13,037 | |||||
Non-current liabilities | 681 | 637 | |||||
Deferred reimbursement | (5,000 | ) | (12,600 | ) | |||
Net cash used in operating activities | $ | (239,278 | ) | $ | (140,661 | ) | |
Investing activities | |||||||
Sale and redemption of marketable securities | 388,135 | 230,981 | |||||
Purchases of marketable securities | (440,963 | ) | (450,358 | ) | |||
Capital expenditures | (4,571 | ) | (3,398 | ) | |||
Net cash used in investing activities | $ | (57,399 | ) | $ | (222,775 | ) | |
Financing activities | |||||||
Proceeds from issuance of common stock, net of issuance costs | 294,584 | 243,036 | |||||
Proceeds from senior secured term loan, net of issuance costs | 146,622 | — | |||||
Payment of capital leases | (218 | ) | (212 | ) | |||
Payment of contingent consideration | — | (10,000 | ) | ||||
Purchase of vested restricted stock units | (2,681 | ) | (1,067 | ) | |||
Proceeds from exercise of stock options | 8,492 | 8,841 | |||||
Payment of deferred financing fees | — | (28 | ) | ||||
Proceeds from exercise of warrants | 3,617 | — | |||||
Net cash provided by financing activities | $ | 450,416 | $ | 240,570 | |||
Effect of exchange rate changes on cash, cash equivalents and restricted cash | $ | (1,146 | ) | $ | 1,030 | ||
Net increase (decrease) in cash and cash equivalents and restricted cash | 152,593 | (121,836 | ) | ||||
Cash and cash equivalents and restricted cash at beginning of period | $ | 51,237 | $ | 187,413 | |||
Cash and cash equivalents and restricted cash at end of period | $ | 203,830 | $ | 65,577 | |||
Supplemental disclosures of cash flow information | |||||||
Cash paid during the period for interest | $ | 3,787 | $ | 3,662 | |||
Capital expenditures, unpaid | $ | 538 | $ | — | |||
Capital expenditures funded by capital lease | $ | 80 | $ | — |
As of September 30, 2018 | |||||||||||||||
(in thousands) | Cost | Gross unrealized Gain | Gross unrealized Loss | Fair Value | |||||||||||
Cash and cash equivalents | $ | 201,827 | $ | — | $ | — | $ | 201,827 | |||||||
Corporate debt securities, current portion | 227,517 | 5 | (123 | ) | 227,399 | ||||||||||
Commercial paper | 82,704 | — | (49 | ) | 82,655 | ||||||||||
Asset-backed securities | 52,145 | — | (44 | ) | 52,101 | ||||||||||
Money market | 350 | — | — | 350 | |||||||||||
Certificates of deposit | 51 | — | — | 51 | |||||||||||
$ | 564,594 | $ | 5 | $ | (216 | ) | $ | 564,383 | |||||||
Included in cash and cash equivalents | $ | 201,827 | $ | — | $ | — | $ | 201,827 | |||||||
Included in marketable securities, current and non-current | 362,767 | 5 | (216 | ) | 362,556 | ||||||||||
Total cash, cash equivalents and marketable securities | $ | 564,594 | $ | 5 | $ | (216 | ) | $ | 564,383 |
As of December 31, 2017 | |||||||||||||||
(in thousands) | Cost | Gross unrealized Gain | Gross unrealized Loss | Fair Value | |||||||||||
Cash and cash equivalents | $ | 49,060 | $ | — | $ | — | $ | 49,060 | |||||||
Corporate debt securities, current portion | 199,314 | 1 | (303 | ) | 199,012 | ||||||||||
Commercial paper | 79,878 | — | (75 | ) | 79,803 | ||||||||||
Asset-backed securities | 30,346 | — | (59 | ) | 30,287 | ||||||||||
Money market | 350 | — | — | 350 | |||||||||||
Certificates of deposit | 50 | — | — | 50 | |||||||||||
$ | 358,998 | $ | 1 | $ | (437 | ) | $ | 358,562 | |||||||
Included in cash and cash equivalents | $ | 49,060 | $ | — | $ | — | $ | 49,060 | |||||||
Included in marketable securities | 309,938 | 1 | (437 | ) | 309,502 | ||||||||||
Total cash, cash equivalents and marketable securities | $ | 358,998 | $ | 1 | $ | (437 | ) | $ | 358,562 |
(in thousands) | September 30, 2018 | December 31, 2017 | September 30, 2017 | December 31, 2016 | |||||||||||
Cash and cash equivalents | $ | 201,827 | $ | 49,060 | $ | 64,133 | $ | 187,026 | |||||||
Restricted cash | 2,003 | 2,177 | 1,444 | 387 | |||||||||||
Cash and cash equivalents and restricted cash shown in the statement of cash flows | $ | 203,830 | $ | 51,237 | $ | 65,577 | $ | 187,413 |
(in thousands) | September 30, 2018 | December 31, 2017 | |||||
Raw materials | $ | 2,954 | $ | 2,394 | |||
Work-in-process | $ | 1,458 | $ | 1,449 | |||
Finished goods | 1,899 | 780 | |||||
Total inventories | $ | 6,311 | $ | 4,623 |
Liability component | September 30, 2018 | December 31, 2017 | ||||||
Principal | $ | 400,000 | $ | 250,000 | ||||
Less: debt discount (1) | (76,946 | ) | (81,566 | ) | ||||
Less: deferred financing (1) | (4,246 | ) | (4,267 | ) | ||||
Net carrying value of the debt | $ | 318,808 | $ | 164,167 |
Components (In thousands) | Three Months Ended September 30, 2018 | Nine Months Ended September 30, 2018 | ||||||
Contractual interest expense | $ | 1,969 | $ | 5,744 | ||||
Amortization of debt discount | 2,609 | 7,620 | ||||||
Amortization of deferred financing | 137 | 399 | ||||||
Total | $ | 4,715 | $ | 13,763 |
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2018 | 2017 | 2018 | 2017 | ||||||||||||
Expected stock price volatility | 75.5 | % | 82.5 | % | 80.7 | % | 83.1 | % | |||||||
Risk free interest rate | 2.7 | % | 1.8 | % | 2.4 | % | 2.0 | % | |||||||
Expected life of options (years) | 5.62 | 5.89 | 5.62 | 6.21 | |||||||||||
Expected annual dividend per share | $ | — | $ | — | $ | — | $ | — |
Number of Shares | Weighted Average Exercise Price | Weighted Average Remaining Contractual Life | Aggregate Intrinsic Value | |||||||||
(in thousands) | (in millions) | |||||||||||
Options outstanding, December 31, 2017 | 15,181 | $ | 7.48 | |||||||||
Granted | 2,077 | $ | 15.36 | |||||||||
Exercised | (1,282 | ) | $ | 6.62 | ||||||||
Forfeited | (200 | ) | $ | 9.75 | ||||||||
Expired | (7 | ) | 10.45 | |||||||||
Options outstanding, September 30, 2018 | 15,769 | $ | 8.56 | 7.0 years | $ | 65.8 | ||||||
Vested and unvested expected to vest, September 30, 2018 | 15,003 | $ | 8.43 | 6.9 years | $ | 63.9 | ||||||
Exercisable at September 30, 2018 | 9,406 | $ | 7.26 | 6.0 years | $ | 47.4 |
Number of Shares | Weighted Average Grant Date Fair Value | Weighted Average Remaining Years | Aggregate Intrinsic Value | |||||||||
(in thousands) | (in millions) | |||||||||||
Non-vested units as of December 31, 2017 | 2,575 | $ | 5.85 | |||||||||
Granted | 1,635 | $ | 16.57 | |||||||||
Vested | (517 | ) | $ | 5.91 | ||||||||
Forfeited | (58 | ) | $ | 10.23 | ||||||||
Non-vested units as of September 30, 2018 | 3,635 | $ | 10.49 | 2.7 years | $ | 44.0 |
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
(in thousands) | 2018 | 2017 | 2018 | 2017 | ||||||||||||
Equity compensation expense recognized in: | ||||||||||||||||
Research and development expense | $ | 2,905 | $ | 2,390 | $ | 8,603 | $ | 7,456 | ||||||||
Selling, general and administrative expense | 4,149 | 3,110 | 12,270 | 9,611 | ||||||||||||
Total equity compensation expense | $ | 7,054 | $ | 5,500 | $ | 20,873 | $ | 17,067 |
(in thousands) | Level 2 | Total | ||||||
Assets: | ||||||||
Commercial paper | $ | 82,655 | $ | 82,655 | ||||
Asset-backed securities | 52,101 | 52,101 | ||||||
Corporate debt securities | 227,399 | 227,399 | ||||||
Money market funds | 3,335 | 3,335 | ||||||
$ | 365,490 | $ | 365,490 |
Level 2 | Level 3 | Total | ||||||||||
Liabilities: | ||||||||||||
Contingent consideration payable | $ | — | $ | 28,100 | $ | 28,100 | ||||||
Deferred compensation plan liability | 2,996 | — | 2,996 | |||||||||
$ | 2,996 | $ | 28,100 | $ | 31,096 |
(in thousands) | Level 2 | Total | |||||
Assets: | |||||||
Commercial paper | $ | 79,803 | $ | 79,803 | |||
Asset-backed securities | 30,287 | 30,287 | |||||
Corporate debt securities | 199,012 | 199,012 | |||||
Money market funds | 2,598 | 2,598 | |||||
$ | 311,700 | $ | 311,700 |
Level 2 | Level 3 | Total | |||||||||
Liabilities: | |||||||||||
Contingent consideration payable | $ | — | $ | 25,400 | $ | 25,400 | |||||
Deferred compensation plan liability | 2,258 | — | 2,258 | ||||||||
$ | 2,258 | $ | 25,400 | $ | 27,658 |
Contingent Consideration Liability | Fair Value as of September 30, 2018 | Valuation Technique | Unobservable Input | Range | ||||||
Discount rate | 10.0% | |||||||||
Clinical and regulatory milestones | $ | 27.6 | million | Probability weighted discounted cash flow | Probability of achievement of milestones | 71.0%-100.0% | ||||
Projected year of payments | 2018-2022 |
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
(in thousands) | 2018 | 2017 | 2018 | 2017 | |||||||||||
Balance, beginning of the period | $ | 26,800 | $ | 265,350 | 25,400 | 269,722 | |||||||||
Payment of contingent consideration in cash | — | — | — | (10,000 | ) | ||||||||||
Changes in fair value during the period, included in statement of operations | 1,300 | (244,250 | ) | 2,700 | (238,622 | ) | |||||||||
Balance, end of the period | $ | 28,100 | $ | 21,100 | 28,100 | $ | 21,100 |
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
(in thousands, except per share amounts) | 2018 | 2017 | 2018 | 2017 | ||||||||||||
Numerator: | ||||||||||||||||
Net loss attributable to common stockholders | $ | (159,163 | ) | $ | (111,666 | ) | $ | (270,914 | ) | $ | (214,794 | ) | ||||
Denominator: | ||||||||||||||||
Weighted average common shares outstanding — basic and diluted | 189,162,841 | 160,796,841 | 184,606,790 | 148,963,864 |
As of September 30, | ||||||
(in thousands) | 2018 | 2017 | ||||
Options to purchase common stock | 15,769 | 16,212 | ||||
Convertible notes | 40,850 | 40,850 | ||||
Outstanding warrants, convertible to common stock | 2,657 | 3,110 | ||||
Unvested restricted stock units | 3,635 | 2,690 | ||||
Vested restricted stock units, unissued | 103 | 50 | ||||
Total number of potentially issuable shares | 63,014 | 62,912 |
• | Commercial success. In the nine months ended September 30, 2018, Galafold® revenue totaled approximately $58.6 million. Revenue has been generated primarily in the EU. |
• | Regulatory progress. We received approval for migalastat in the U.S. and Japan. |
• | Pompe clinical study. We have reported positive data from a Phase 1/2 clinical study to evaluate Pompe disease patients treated with our novel treatment paradigm AT-GAA. |
• | Pipeline Growth: With 14 new gene therapy programs for LSDs, we have established a leading portfolio of medicines for people living with rare metabolic disorders. We acquired worldwide development and commercial rights for ten gene therapy programs in rare, neurologic LSDs with lead programs in CLN6, CLN3, and CLN8 Batten disease. An additional four programs were added to the pipeline through the collaboration with Penn to pursue research and development of novel gene therapies for Pompe disease, Fabry disease, CDD and one additional undisclosed rare metabolic disorder. |
• | Manufacturing. We successfully scaled manufacture of Pompe biologic engineering batches to commercial scale (1,000L) with capacity plans to enable us to produce sufficient quantities to serve the entire Pompe population as quickly as possible after receipt of applicable regulatory approvals. Through our collaborations with NCH and Penn, we also gain access to their preclinical manufacturing capabilities, clinical supply and CMO relationships for those programs. |
• | Financial strength. Total cash, cash equivalents and marketable securities of $564.4 million at September 30, 2018 compared to $358.6 million at December 31, 2017. The current cash position, including expected Galafold® revenues, is sufficient to fund ongoing Fabry, Pompe and gene therapy program operations into at least 2021. Potential future business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements. |
Three Months Ended September 30, | ||||||||||||
(in thousands) | 2018 | 2017 | Change | |||||||||
Net product sales | $ | 20,596 | $ | 10,874 | $ | 9,722 | ||||||
Cost of goods sold | 4,310 | 1,790 | 2,520 | |||||||||
Cost of goods sold as a percentage of net product sales | 20.9 | % | 16.5 | % | 4.4 | % | ||||||
Operating expenses: | ||||||||||||
Research and development | 138,227 | 40,641 | 97,586 | |||||||||
Selling, general and administrative | 31,867 | 21,647 | 10,220 | |||||||||
Changes in fair value of contingent consideration payable | 1,300 | (244,250 | ) | 245,550 | ||||||||
Loss on impairment of asset | — | 465,427 | (465,427 | ) | ||||||||
Depreciation | 1,073 | 851 | 222 | |||||||||
Other income (expense): | ||||||||||||
Interest income | 2,721 | 1,190 | 1,531 | |||||||||
Interest expense | (4,715 | ) | (4,351 | ) | (364 | ) | ||||||
Change in fair value of derivatives | — | — | — | |||||||||
Other (expense) income | (1,039 | ) | 2,044 | (3,083 | ) | |||||||
Income tax benefit | 51 | 164,683 | (164,632 | ) | ||||||||
Net loss attributable to common stockholders | $ | (159,163 | ) | $ | (111,666 | ) | $ | (47,497 | ) |
(in thousands) | Three Months Ended September 30, | |||||||
Projects | 2018 | 2017 | ||||||
Third party direct project expenses | ||||||||
Migalastat (Fabry Disease) | $ | 2,610 | $ | 3,265 | ||||
AT-GAA (Pompe Disease) | 12,312 | 10,456 | ||||||
SD-101 (EB-Epidermolysis Bullosa) | — | 9,660 | ||||||
Pre-clinical programs | 254 | 192 | ||||||
Total third party direct project expenses | 15,176 | 23,573 | ||||||
Other project costs | ||||||||
Personnel costs | 15,584 | 11,410 | ||||||
Other costs | 7,467 | 5,658 | ||||||
Total other project costs | 23,051 | 17,068 | ||||||
Business development transactions | 100,000 | — | ||||||
Total research and development costs | $ | 138,227 | $ | 40,641 |
Nine Months Ended September 30, | ||||||||||||
(in thousands) | 2018 | 2017 | Change | |||||||||
Net product sales | $ | 58,601 | $ | 22,201 | 36,400 | |||||||
Cost of goods sold | 10,060 | 3,626 | 6,434 | |||||||||
Cost of goods sold as a percentage of net product sales | 17.2 | % | 16.3 | % | 0.9 | % | ||||||
Operating expenses: | ||||||||||||
Research and development | 213,685 | 103,502 | 110,183 | |||||||||
Selling, general and administrative | 88,435 | 60,090 | 28,345 | |||||||||
Changes in fair value of contingent consideration payable | 2,700 | (238,622 | ) | 241,322 | ||||||||
Loss on impairment of assets | — | 465,427 | (465,427 | ) | ||||||||
Depreciation | 3,015 | 2,486 | 529 | |||||||||
Other income (expense): | ||||||||||||
Interest income | 7,371 | 2,702 | 4,669 | |||||||||
Interest expense | (13,763 | ) | (12,820 | ) | (943 | ) | ||||||
Change in fair value of derivatives | (2,739 | ) | 163 | (2,902 | ) | |||||||
Other (expense) income | (3,593 | ) | 4,891 | (8,484 | ) | |||||||
Income tax benefit | 1,104 | 164,578 | (163,474 | ) | ||||||||
Net loss attributable to common stockholders | $ | (270,914 | ) | $ | (214,794 | ) | $ | (56,120 | ) |
(in thousands) | Nine Months Ended September 30, | |||||||
Projects | 2018 | 2017 | ||||||
Third party direct project expenses | ||||||||
Migalastat (Fabry Disease) | $ | 10,044 | $ | 8,644 | ||||
AT-GAA (Pompe Disease) | 37,364 | 29,594 | ||||||
SD-101 (EB-Epidermolysis Bullosa) | — | 15,424 | ||||||
Pre-clinical programs | 1,054 | 430 | ||||||
Total third party direct project expenses | 48,462 | 54,092 | ||||||
Other project costs | ||||||||
Personnel costs | 44,501 | 34,162 | ||||||
Other costs | 20,722 | 15,248 | ||||||
Total other project costs | 65,223 | 49,410 | ||||||
Business development transactions | 100,000 | — | ||||||
Total research and development costs | $ | 213,685 | $ | 103,502 |
• | the progress and results of our clinical trials of our drug candidates; |
• | the cost of manufacturing drug supply for our clinical and preclinical studies, including the cost of manufacturing Pompe ERT; |
• | the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates; |
• | the costs, timing and outcome of regulatory review of our product candidates; |
• | the number and development requirements of other product candidates that we pursue; |
• | the costs of commercialization activities, including product marketing, sales and distribution; |
• | the emergence of competing technologies and other adverse market developments; |
• | our ability to obtain reimbursement for migalastat HCl; |
• | our ability to obtain market acceptance of migalastat HCl; |
• | the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing and defending intellectual property-related claims; |
• | the extent to which we acquire or invest in businesses, products and technologies; |
• | our ability to successfully integrate our acquired products and technologies into our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected; and |
• | our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators. |
Exhibit Number | Description | |
2.1 | ||
10.1 | ||
31.1 | ||
31.2 | ||
32.1 | ||
101 | The following financial information from this Quarterly Report on Form 10-Q for the nine months ended September 30, 2018, formatted in XBRL (Extensible Business Reporting Language) and filed electronically herewith: (i) the Consolidated Balance Sheets; (ii) the Consolidated Statements of Operations; (iii) the Consolidated Statements of Comprehensive Loss; (iv) the Consolidated Statements of Cash Flows; (v) and the Notes to the Consolidated Financial Statements |
AMICUS THERAPEUTICS, INC. | ||
Date: November 5, 2018 | By: | /s/ John F. Crowley |
John F. Crowley | ||
Chairman and Chief Executive Officer | ||
(Principal Executive Officer) | ||
Date: November 5, 2018 | By: | /s/ William D. Baird III |
William D. Baird III | ||
Chief Financial Officer | ||
(Principal Financial Officer) |
1. | I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.; |
2. | Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report; |
3. | Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; |
4. | The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have: |
5. | The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions): |
Date: November 5, 2018 | /s/ John F. Crowley |
John F. Crowley | |
Chairman and Chief Executive Officer |
1. | I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.; |
2. | Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report; |
3. | Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; |
4. | The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have: |
5. | The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions): |
Date: November 5, 2018 | /s/ William D. Baird III |
William D. Baird III | |
Chief Financial Officer |
Date: November 5, 2018 | By: | /s/ John F. Crowley |
John F. Crowley | ||
Chairman and Chief Executive Officer | ||
Date: November 5, 2018 | By: | /s/ William D. Baird III |
William D. Baird III | ||
Chief Financial Officer |
Document and Entity Information - shares |
9 Months Ended | |
---|---|---|
Sep. 30, 2018 |
Oct. 29, 2018 |
|
Document And Entity Information [Abstract] | ||
Entity Registrant Name | AMICUS THERAPEUTICS INC | |
Entity Central Index Key | 0001178879 | |
Document Type | 10-Q | |
Document Period End Date | Sep. 30, 2018 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Entity Current Reporting Status | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Common Stock, Shares Outstanding | 189,289,881 | |
Document Fiscal Year Focus | 2018 | |
Document Fiscal Period Focus | Q3 | |
Entity Emerging Growth Company | false | |
Entity Small Business | false |
Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands |
Sep. 30, 2018 |
Dec. 31, 2017 |
---|---|---|
Statement of Financial Position [Abstract] | ||
Accumulated depreciation of property and equipment | $ 15,483 | $ 12,515 |
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Common stock, shares authorized | 500,000,000 | 250,000,000 |
Common stock, shares issued | 189,254,341 | 166,989,790 |
Common stock, shares outstanding | 189,254,341 | 166,989,790 |
Consolidated Statements of Operations - USD ($) $ in Thousands |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Sep. 30, 2018 |
Sep. 30, 2017 |
|
Revenue: | ||||
Net product sales | $ 20,596 | $ 10,874 | $ 58,601 | $ 22,201 |
Cost of goods sold | 4,310 | 1,790 | 10,060 | 3,626 |
Gross profit | 16,286 | 9,084 | 48,541 | 18,575 |
Operating expenses: | ||||
Research and development | 138,227 | 40,641 | 213,685 | 103,502 |
Selling, general and administrative | 31,867 | 21,647 | 88,435 | 60,090 |
Changes in fair value of contingent consideration payable | 1,300 | (244,250) | 2,700 | (238,622) |
Loss on impairment of assets | 0 | 465,427 | 0 | 465,427 |
Depreciation | 1,073 | 851 | 3,015 | 2,486 |
Total operating expenses | 172,467 | 284,316 | 307,835 | 392,883 |
Loss from operations | (156,181) | (275,232) | (259,294) | (374,308) |
Other income (expense): | ||||
Interest income | 2,721 | 1,190 | 7,371 | 2,702 |
Interest expense | (4,715) | (4,351) | (13,763) | (12,820) |
Change in fair value of derivatives | 0 | 0 | (2,739) | 163 |
Other (expense) income | (1,039) | 2,044 | (3,593) | 4,891 |
Loss before income tax | (159,214) | (276,349) | (272,018) | (379,372) |
Income tax benefit | 51 | 164,683 | 1,104 | 164,578 |
Net loss attributable to common stockholders | $ (159,163) | $ (111,666) | $ (270,914) | $ (214,794) |
Net loss attributable to common stockholders per common share - basic and diluted (in dollars per share) | $ (0.84) | $ (0.69) | $ (1.47) | $ (1.44) |
Weighted-average common shares outstanding - basic and diluted (in shares) | 189,162,841 | 160,796,841 | 184,606,790 | 148,963,864 |
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Sep. 30, 2018 |
Sep. 30, 2017 |
|
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (159,163) | $ (111,666) | $ (270,914) | $ (214,794) |
Other comprehensive (loss) gain: | ||||
Foreign currency translation adjustment gain (loss), net of tax impact of $59, $0, $168, $0, respectively | 665 | (1,176) | 1,167 | (3,312) |
Unrealized gain (loss) on available-for-sale securities | 244 | (131) | 225 | (203) |
Other comprehensive income (loss) | 909 | (1,307) | 1,392 | (3,515) |
Comprehensive loss | $ (158,254) | $ (112,973) | $ (269,522) | $ (218,309) |
Consolidated Statements of Comprehensive Loss (Parenthetical) - USD ($) $ in Thousands |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Sep. 30, 2018 |
Sep. 30, 2017 |
|
Statement of Comprehensive Income [Abstract] | ||||
Foreign currency translation adjustment gain (loss), tax (benefit) expense | $ 59 | $ 0 | $ 168 | $ 0 |
Description of Business |
9 Months Ended |
---|---|
Sep. 30, 2018 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Description of Business | Description of Business Amicus Therapeutics, Inc. (the “Company”) is a global patient-centric biotechnology company engaged in the discovery, development and commercialization of a diverse set of novel treatments for patients living with rare metabolic diseases. With one medicine for Fabry disease that has achieved widespread global approval, a differentiated biologic for Pompe disease in the clinic and the recent addition of fourteen new gene therapy programs into the pipeline, including two clinical stage gene therapies for Batten disease, the Company has a leading portfolio of medicines for lysosomal storage disorders (“LSDs”). The cornerstone of the Company’s portfolio is migalastat HCl (also referred to as “migalastat”), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the United States (“U.S.”), European Union (“EU”) and Japan, with additional approvals granted and applications pending in several other geographies. During the third quarter of 2018, the Company initiated the commercial launch of Galafold® in the U.S. for the treatment of adult patients with a confirmed diagnosis of Fabry disease and an amenable genetic variant. The lead biologics program of the Company’s pipeline is Amicus Therapeutics GAA (“AT-GAA”, also known as ATB200/AT2221), a novel, clinical-stage, potential best-in-class treatment paradigm for Pompe disease. The Company’s Chaperone-Advanced Replacement Therapy (“CHART®”) platform technology is leveraged to develop novel products for Pompe disease and potentially future other LSDs. During the second half of 2018, the Company has expanded its portfolio to include fourteen new gene therapy programs. In September 2018, the Company acquired worldwide development and commercial rights for ten gene therapy programs for neurologic LSDs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University through the acquisition of Celenex, Inc., (“Celenex”) a private, clinical stage gene therapy company, for cash consideration of $100.0 million and additional consideration payable upon the achievement of certain development and approval milestones. The acquisition establishes Amicus as a leading company in neurologic LSDs. The lead programs in CLN6, CLN3, and CLN8 Batten disease are potential first-to-market curative therapies for these rare, devastating diseases. For additional information see "—Note 3. Acquisitions." In October 2018, the Company further expanded its gene therapy portfolio through a collaboration agreement with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (“Penn”) to pursue the research and development of novel gene therapies for four additional indications, including Pompe disease, Fabry disease, CDKL5 deficiency disorder (“CDD”) and one additional undisclosed rare metabolic disorder. This relationship will combine the Company's protein engineering and glycobiology expertise with Penn’s adeno associated virus (“AAV”) gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability. The Company believes that its platform technologies and its product pipeline uniquely positions it and drives its commitment to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. During the third quarter of 2018, the Company entered into a loan agreement with BioPharma Credit PLC, as the lender, for a $150.0 million non-dilutive senior secured term loan (the “Senior Secured Term Loan”) with an interest rate equal to 3-month LIBOR plus 7.50% per annum, subject to a floor and ceiling on the rate, which matures in five years. The Company received net proceeds of $146.6 million in September 2018, after deducting fees and estimated expenses payable by the Company. There are no warrants or any equity conversion features associated with the Senior Secured Term Loan. The proceeds from this financing will be used to support the cost of the Celenex acquisition, its related development costs and other general corporate purposes. For additional information, see " —Note 6. Debt." During the first quarter of 2018, the Company issued 20,239,839 shares of its common stock through an underwritten offering resulting in net proceeds of $294.6 million after deducting underwriting discounts and commissions and offering expenses payable by the Company. The Company expects to use the net proceeds of the offering for investment in the U.S. and international commercial infrastructure for migalastat HCl, investment in manufacturing capabilities for the ERT ATB200, the continued clinical development of its product candidates, research and development expenditures, clinical and pre-clinical trial expenditures, commercialization expenditures and for other general corporate purposes. For additional information, see “—Note 7. Stockholders’ Equity.” The Company had an accumulated deficit of approximately $1.3 billion as of September 30, 2018 and anticipates incurring losses through the fiscal year ending December 31, 2018 and beyond. The Company has been able to fund its operating losses to date through stock offerings, debt issuances, payments from partners during the terms of the collaboration agreements, and other financing arrangements. The current cash position, including expected Galafold® revenues, is sufficient to fund ongoing Fabry, Pompe and gene therapy program operations into at least 2021. Potential future business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company’s future capital requirements. |
Summary of Significant Accounting Policies |
9 Months Ended |
---|---|
Sep. 30, 2018 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Basis of Presentation The Company has prepared the accompanying unaudited consolidated financial statements in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited financial statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s interim financial information. The accompanying unaudited consolidated financial statements and related notes should be read in conjunction with the Company’s financial statements and related notes as contained in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2017. For a complete description of the Company’s accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2017. Consolidation The consolidated financial statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions have been eliminated in consolidation. Foreign Currency Transactions The functional currency for most of the Company’s foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company’s foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders’ equity. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. Reclassification Certain prior year amounts have been reclassified for comparative purposes. The reclassifications did not affect results of operations, net assets or cash flows. Cash, Cash Equivalents, Restricted Cash and Marketable Securities The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition, to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company’s consolidated balance sheet. Unrealized holding gains and losses are reported within comprehensive income (loss) in the statements of comprehensive loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations or other observable inputs. Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in non-current assets on the Company’s consolidated balance sheet. Concentration of Credit Risk The Company’s financial instruments that are exposed to concentration of credit risk consist primarily of cash and cash equivalents and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on cash and cash equivalents or its marketable securities. The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company’s accounts receivable at September 30, 2018 have arisen from product sales primarily in the EU. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined and the Company evaluates the creditworthiness of each customer on a regular basis. To date, the Company has not incurred any credit losses. Revenue Recognition The Company’s net product sales consist of sales of Galafold® for the treatment of Fabry disease primarily in the EU. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program (“EAP”). Orders for Galafold® are generally received from pharmacies and the ultimate payor is typically a government authority. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company’s customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenues from sales of Galafold® are recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes. The Company elected the portfolio approach practical expedient in applying ASC Topic 606 to its identified revenue streams. Contracts within each revenue stream have similar characteristics and the Company believes this approach would not differ materially from applying ASC Topic 606 to each individual contract. Recent Accounting Developments - Guidance Adopted in 2018 ASU 2018-07 — In June 2018, the Financial Accounting Standards Board (the “FASB”) issued Accounting Standards Update (“ASU”) 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting (“ASU 2018-07”).These amendments expand the scope of Topic 718, Compensation—Stock Compensation (which currently only includes share-based payments to employees) to include share-based payments issued to non-employees for goods or services. Consequently, the accounting for share-based payments to non-employees and employees will be substantially aligned. ASU 2018-07 supersedes Subtopic 505-50, Equity—Equity-Based Payments to Non-Employees, and is effective for all public entities for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. Early adoption is permitted, but no earlier than a company’s adoption date of Topic 606, Revenue from Contracts with Customers. The Company early adopted ASU 2018-07 in the second quarter of 2018 and there was no material impact on its consolidated financial statements from the adoption. ASU 2018-02 — In February 2018, the FASB issued ASU 2018-02, Income Statement - Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income (“ASU 2018-02”). Prior to ASU 2018-02, U.S. GAAP required the remeasurement of deferred tax assets and liabilities as a result of a change in tax laws or rates to be presented in net income from continuing operations, even in situations in which the related income tax effects of items in accumulated other comprehensive income were originally recognized in other comprehensive income. As a result, such items, referred to as stranded tax effects, did not reflect the appropriate tax rate. Under ASU 2018-02, entities are permitted, but not required, to reclassify from accumulated other comprehensive income to retained earnings those stranded tax effects resulting from the Tax Cuts and Jobs Act of 2017. ASU 2018-02 is effective for all entities for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. Early adoption is permitted. The Company adopted the new standard effective January 1, 2018. As a result of the adoption, the Company reclassified a gain of $383,000 from the foreign currency translation adjustment in accumulated other comprehensive loss to accumulated deficit in the consolidated balance sheet as of September 30, 2018. ASU 2017-09 — In May 2017, the FASB issued ASU 2017-09, Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting (“ASU 2017-09”). The amendments in ASU 2017-09 provide guidance on determining which changes to the terms and conditions of share-based payment awards require an entity to apply modification accounting under ASU 2017-09. An entity should account for the effects of a modification unless all the following are met: (1) The fair value (or calculated value or intrinsic value, if such an alternative measurement method is used) of the modified award is the same as the fair value (or calculated value or intrinsic value, if such an alternative measurement method is used) of the original award immediately before the original award is modified. If the modification does not affect any of the inputs to the valuation technique that the entity uses to value the award, the entity is not required to estimate the value immediately before and after the modification; (2) The vesting conditions of the modified award are the same as the vesting conditions of the original award immediately before the original award is modified; and (3) The classification of the modified award as an equity instrument or a liability instrument is the same as the classification of the original award immediately before the original award is modified. ASU 2017-09 is effective for all entities for annual periods, including interim periods within those annual periods, beginning after December 15, 2017. The Company adopted this standard on January 1, 2018 and as the Company did not have any modification of awards, the adoption of the standard did not have a material impact on its consolidated financial statements. ASU 2017-01 — In January 2017, the FASB issued ASU 2017-01, Business Combinations (Topic 805): Clarifying the Definition of a Business (“ASU 2017-01”). This ASU clarifies the definition of a business. The amendments affect all companies and other reporting organizations that must determine whether they have acquired or sold a business. The amendments in ASU 2017-01 are effective for public companies for annual periods beginning after December 15, 2017, including interim periods within those periods. The amendments should be applied prospectively as of the beginning of the period of adoption. The Company adopted ASU 2017-01 on January 1, 2018. The adoption of the standard did not have a material impact on its consolidated financial statements. For additional information see "—Note 3. Acquisitions." ASU 2016-18 — In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”). The amendments of ASU 2016-18 require an entity to include amounts generally described as restricted cash and restricted cash equivalents with cash and cash equivalents when reconciling the beginning of period and end of period total amounts on the statement of cash flows. The amendments of ASU 2016-18 are effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual periods. The Company adopted the guidance in ASU 2016-18 effective January 1, 2018. In connection with the adoption of the standard, the Company applied the guidance retrospectively which resulted in an increase in cash flows from operations of $1.1 million on the statement of cash flows for the nine months ended September 30, 2017. ASU 2016-16 — In October 2016, the FASB issued ASU 2016-16, Income Taxes (Topic 740): Intra-Entity Transfers of Assets Other Than Inventory (“ASU 2016-16”). ASU 2016-16 requires an entity to recognize the income tax consequences of an intra-entity transfer of an asset other than inventory when the transfer occurs. The amendments eliminate the exception for an intra-entity transfer of an asset other than inventory. The amendments in ASU 2016-16 are effective for public business entities for annual periods beginning after December 15, 2017, including interim reporting periods within those annual reporting periods. The Company adopted ASU 2016-16 on January 1, 2018 and there was no material impact from the adoption. ASU 2016-01 — In January 2016, the FASB issued ASU No. 2016-01, Financial Instruments-Overall: Recognition and Measurement of Financial Assets and Financial Liabilities (“ASU 2016-01”). ASU 2016-01 changes accounting for equity investments, financial liabilities under the fair value option, and presentation and disclosure requirements for financial instruments. ASU 2016-01 does not apply to equity investments in consolidated subsidiaries or those accounted for under the equity method of accounting. In addition, the FASB clarified guidance related to the valuation allowance assessment when recognizing deferred tax assets resulting from unrealized losses on available-for-sale debt securities. Equity investments with readily determinable fair values will be measured at fair value with changes in fair value recognized in net income. Companies have the option to measure equity investments without readily determinable fair values at either fair value or at cost adjusted for changes in observable prices minus impairment. Companies that elect the fair value option for financial liabilities must recognize changes in fair value related to instrument-specific credit risk in other comprehensive income. Companies must assess valuation allowances for deferred tax assets related to available-for-sale debt securities in combination with their other deferred tax assets. ASU 2016-01 is effective beginning in the first quarter of 2018 and the Company adopted it in the first quarter of 2018. There was no impact on the Company’s consolidated financial statements and related disclosures upon adoption, as the Company does not have equity investments or liabilities with credit risk. In addition, the guidance relating to deferred tax assets did not result in a change in accounting treatment for the Company. ASU 2014-09 — In May 2014, the FASB issued ASU 2014-09, Revenue from Contracts with Customers (Topic 606) which along with amendments issued in 2015 and 2016, replaced substantially all current U.S. GAAP guidance on this topic and eliminated industry-specific guidance. The new revenue recognition standard requires entities to recognize revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. Effective January 1, 2018, the Company adopted the new revenue recognition standard using the modified retrospective approach and applied this approach only to contracts that were not completed as of January 1, 2018. The timing of revenue recognition and treatment of contract costs remains unchanged under the new revenue recognition standard. As such, the adoption of the new revenue recognition standard did not have a material impact on the Company's consolidated financial statements. The information presented for the periods prior to January 1, 2018 has not been restated and is reported under the accounting standard in effect for those periods. Recent Accounting Developments - Guidance Not Yet Adopted In August 2018, the Securities Exchange Commission (“SEC”) issued Final Rule 33-10532, Disclosure Update and Simplification, which amends certain disclosure requirements that were redundant, duplicative, overlapping or superseded by other SEC disclosure requirements. The amendments generally eliminated or otherwise reduced certain disclosure requirements of various SEC rules and regulations. However, in some cases, the amendments require additional information to be disclosed, including changes in stockholders’ equity in interim periods. The rule is effective 30 days after its publication in the Federal Register. The rule was posted on October 4, 2018. On September 25, 2018, the SEC released guidance advising it will not object to a registrant adopting the requirement to include changes in stockholders’ equity in the Form 10-Q for the first quarter beginning after the effective date of the rule. The Company is currently assessing the impact that this standard will have on its consolidated financial statements upon adoption and expects to adopt the guidance in it's Form 10-Q for the period ended March 31, 2019. ASU 2018-13 — In August 2018, the FASB issued ASU 2018-03, Fair Value Measurement (Topic 820): Disclosure Framework-Changes to the Disclosure Requirements for Fair Value Measurement (“ASU 2018-13”). The amendments modify the disclosure requirements in Topic 820. ASU 2018-13 is effective for all entities for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. The amendments on changes in unrealized gains and losses, the range and weighted average of significant unobservable inputs used to develop Level 3 fair value measurements, and the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. Early adoption is permitted. An entity is permitted to early adopt any removed or modified disclosures upon issuance of ASU 2018-13 and delay adoption of the additional disclosures until their effective date. The Company is currently assessing the impact that this standard will have on its consolidated financial statements upon adoption. ASU 2017-12 — In August 2017, the FASB issued ASU 2017-12, Derivatives and Hedging (Topic 815): Targeted Improvements to Accounting for Hedging Activities (“ASU 2017-12”). The amendments in this ASU 2017-12 better align an entity’s risk management activities and financial reporting for hedging relationships through changes to both the designation and measurement guidance for qualifying hedging relationships and the presentation of hedge results. To meet that objective, the amendments expand and refine hedge accounting for both nonfinancial and financial risk components and align the recognition and presentation of the effects of the hedging instrument and the hedged item in the financial statements. The amendments in ASU 2017-12 also make certain targeted improvements to simplify the application of hedge accounting guidance and ease the administrative burden of hedge documentation requirements and assessing hedge effectiveness. For public business entities, the amendment is effective for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. Early adoption, including adoption in an interim period, is permitted. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2017-11 — In July 2017, the FASB issued ASU 2017-11, Earnings Per Share (Topic 260); Distinguishing Liabilities from Equity (Topic 480); Derivatives and Hedging (Topic 815): (Part I) Accounting for Certain Financial Instruments with Down Round Features, (Part II) Replacement of the Indefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable Noncontrolling Interests with a Scope Exception (“ASU 2017-11”). Part I of ASU 2017-11 addresses the complexity of accounting for certain financial instruments with down round features. Down round features are features of certain equity-linked instruments (or embedded features) that result in the strike price being reduced on the basis of the pricing of future equity offerings. When determining whether certain financial instruments should be classified as liabilities or equity instruments, a down round feature no longer precludes equity classification when assessing whether the instrument is indexed to an entity’s own stock. Part II of ASU 2017-11 addresses the difficulty of navigating Topic 480, Distinguishing Liabilities from Equity, because of the existence of extensive pending content in the FASB Accounting Standards Codification®. For public business entities, the amendments in Part I of ASU 2017-11 are effective for fiscal years beginning after December 15, 2018. Early adoption is permitted for all entities, including adoption in an interim period. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2017-08 — In March 2017, the FASB issued ASU 2017-08, Receivables—Nonrefundable Fees and Other Costs (Subtopic 310-20), Premium Amortization on Purchased Callable Debt Securities (“ASU 2017-08”). The amendments in ASU 2017-08 shorten the amortization period for certain callable debt securities held at a premium. Specifically, the amendments require the premium to be amortized to the earliest call date. The amendments do not require an accounting change for securities held at a discount; the discount continues to be amortized to maturity. ASU 2017-08 is effective for public business entities for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2018. Early adoption is permitted, including adoption in an interim period. If an entity early adopts in an interim period, any adjustments should be reflected as of the beginning of the fiscal year that includes that interim period. The amendments should be applied on a modified retrospective basis, with a cumulative-effect adjustment directly to retained earnings as of the beginning of the period of adoption. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2017-04 — In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment (“ASU 2017-04”). To simplify the subsequent measurement of goodwill, ASU 2017-04 eliminates Step 2 from the goodwill impairment test. The annual, or interim, goodwill impairment test is performed by comparing the fair value of a reporting unit with its carrying amount. An impairment charge should be recognized for the amount by which the carrying amount exceeds the reporting unit’s fair value; however, the loss recognized should not exceed the total amount of goodwill allocated to that reporting unit. In addition, income tax effects from any tax deductible goodwill on the carrying amount of the reporting unit should be considered when measuring the goodwill impairment loss, if applicable. ASU 2017-04 also eliminates the requirements for any reporting unit with a zero or negative carrying amount to perform a qualitative assessment and, if it fails that qualitative test, to perform Step 2 of the goodwill impairment test. An entity still has the option to perform the qualitative assessment for a reporting unit to determine if the quantitative impairment test is necessary. ASU 2017-04 should be applied on a prospective basis. The nature of and reason for the change in accounting principle should be disclosed upon transition. A public business entity that is a U.S. SEC filer should adopt ASU 2017-04 for its annual or any interim goodwill impairment tests in fiscal years beginning after December 15, 2019. Early adoption is permitted for interim or annual goodwill impairment tests performed on testing dates after January 1, 2017. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2016-02 — In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842) (“ASU 2016-02”). ASU 2016-02 requires the recognition of lease assets and lease liabilities on the balance sheet for all lease obligations and disclosing key information about leasing arrangements. ASU 2016-02 requires the recognition of lease assets and lease liabilities by lessees for those leases classified as operating leases under previous generally accepted accounting principles. ASU 2016-02 will be effective for the Company for all annual and interim periods beginning after December 15, 2018, including interim periods within those fiscal years. In August 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements, (“ASU 2018-11”). ASU 2018-11 provide entities with an additional transition method for adoption, whereby, an entity initially applies the new leases standard at the adoption date and recognizes a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. The Company will adopt these standards effective January 1, 2019, and expects to elect certain of the practical expedients permitted, including the expedient that permits the Company to retain its existing lease assessment and classification. The Company also expects to elect the transition method in ASU 2018-11. The Company is currently working through an adoption plan which includes the evaluation of lease contracts compared to the new standard. While the Company is currently assessing the impact that this standard will have on its consolidated financial statements, the Company expects to recognize lease liabilities and right of use assets. |
Acquisitions |
9 Months Ended |
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Sep. 30, 2018 | |
Business Combinations [Abstract] | |
Acquisitions | Acquisitions In September 2018, the Company expanded its pipeline by acquiring the rights and related intellectual property of ten gene therapy programs through its acquisition of Celenex. Celenex is a private, clinical stage gene therapy company whose lead programs are ten gene therapy programs including CLN6 and CLN3, which are in clinical stage, and several programs in pre-clinical stage. Pursuant to the terms of the agreement, the Company acquired Celenex for cash consideration of $100 million. The Company has also agreed to pay up to an additional $15 million in connection with the achievement of certain development milestones, $262 million in connection with the achievement of certain regulatory approval milestones across multiple programs and up to $75 million in tiered sales milestone payments. Celenex has an exclusive license agreement with Nationwide Children’s Hospital (“NCH”). Under this license agreement, NCH is eligible to receive development and sales based milestones of up to $7.8 million for each product. The Company evaluated the Celenex transaction based on the guidance in Business Combinations (Topic 805) and concluded that the transaction did not meet the definition of a business and was an asset acquisition. Given the fact that the license has no alternative future use, the $100.0 million upfront payment was expensed to research and development expense in the Consolidated Statements of Operations for the three and nine months ended September 30, 2018 in accordance with ASC Topic 730, Research and Development. |
Cash, Cash Equivalents, Marketable Securities and Restricted Cash |
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Cash, Cash Equivalents, and Short-term Investments [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Cash, Cash Equivalents, Marketable Securities and Restricted Cash | Cash, Cash Equivalents, Marketable Securities and Restricted Cash As of September 30, 2018, the Company held $201.8 million in cash and cash equivalents and $362.6 million of available-for-sale debt securities which are reported at fair value on the Company’s consolidated balance sheets. Unrealized holding gains and losses are reported within accumulated other comprehensive loss in the statements of comprehensive loss. If a decline in the fair value of a marketable security below the Company’s cost basis is determined to be other than temporary, such marketable security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in earnings as an impairment charge. The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as many of these securities are either government backed or of the highest credit rating. Investments that have original maturities greater than three months but less than one year are classified as current, while investments that have maturities greater than one year are classified as long-term. Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:
For the nine months ended September 30, 2018 there were nominal realized gains. For the fiscal year ended December 31, 2017, there were no realized gains or losses. The cost of securities sold is based on the specific identification method. Unrealized loss positions in the available-for-sale debt securities as of September 30, 2018 and December 31, 2017 reflect temporary impairments that have been in a loss position for less than twelve months and as such are recognized in other comprehensive gain (loss). The fair value of these available-for-sale debt securities in unrealized loss positions was $315.3 million and $295.1 million as of September 30, 2018 and December 31, 2017, respectively. The following table provides a reconciliation of cash and cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same such amounts shown in the statement of cash flows.
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Inventories |
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Inventories | Inventories Inventories consist of raw materials, work-in-process and finished goods related to the manufacture of Galafold®. The following table summarizes the components of inventories:
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Debt |
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Debt Disclosure [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Debt | Debt Senior Secured Term Loan due 2023 In September 2018, the Company entered into a loan agreement with BioPharma Credit PLC as the lender. The loan agreement provides for a $150 million senior secured term loan (“Senior Secured Term Loan”) with an interest rate equal to the 3-month LIBOR plus 7.50% per annum and matures five years from the maturity date. The Senior Secured Term Loan will be repaid in four quarterly payments equal to 12.50% thereof starting on the forty-eight month anniversary of the date of the first credit extension with the balance due on the Maturity Date. Interest is payable quarterly in arrears. The Senior Secured Term Loan contains certain customary representations and warranties, affirmative and negative covenants and events of default applicable to the Company and certain of its subsidiaries, but does not include any financial covenants relating to the achievement or maintenance of revenue or cash flow. If an event of default occurs and is continuing, the lender may declare all amounts outstanding under the Senior Secured Term Loan to be immediately due and payable. The Company received net proceeds of $146.6 million in September 2018, after deducting fees and estimated expenses payable by the Company. Convertible Notes due 2023 In December 2016, the Company issued at par value $250 million aggregate principal amount of unsecured Convertible Senior Notes due 2023 (the “Convertible Notes”), which included the exercise in full of the $25 million over-allotment option granted to the initial purchasers of the Convertible Notes in a private offering to qualified institutional buyers pursuant to Rule 144A under the Securities Act (the “Note Offering”). Interest is payable semiannually on June 15 and December 15 of each year, beginning on June 15, 2017. The Convertible Notes will mature on December 15, 2023, unless earlier repurchased, redeemed, or converted in accordance with their terms. The Convertible Notes are convertible at the option of the holders, under certain circumstances and during certain periods, into cash, shares of the Company’s common stock or a combination thereof. The net proceeds from the Note Offering were $243.0 million, after deducting fees and estimated expenses payable by the Company. In addition, the Company used approximately $13.5 million of the net proceeds from the issuance of the Convertible Notes to pay the cost of the capped call transactions (“Capped Call Confirmations”) that the Company entered into in connection with the issuance of the Convertible Notes. In accounting for the issuance of the Convertible Notes, the Company separated the Convertible Notes into liability and equity components based on their relative values. The Convertible Notes are initially convertible into approximately 40,849,675 shares of the Company’s common stock under certain circumstances prior to maturity at a conversion rate of 163.3987 shares per $1,000 principal amount of Convertible Notes, which represents a conversion price of approximately $6.12 per share of the Company's common stock, subject to adjustment under certain conditions. The last reported sale price of the Company’s common stock was equal to or more than 130% of the conversion price of the Convertible Notes for at least 20 trading days of the 30 consecutive trading days ending on the last day of the second quarter. As a result, the Convertible Notes are currently convertible into the Company’s common stock. As further described in “—Note 7. Stockholders’ Equity,” on February 15, 2018, the Company entered into an underwriting agreement relating to an underwritten public offering of 19,354,839 shares of the Company’s common stock. Under the terms of the underwriting agreement, the Company granted the underwriters an option, exercisable for 30 days after February 16, 2018, to purchase up to an additional 2,903,225 shares of the Company’s common stock, which was exercised with respect to 885,000 shares of the Company’s common stock. Subsequent to the underwritten public offering on February 15, 2018, the Company did not have sufficient unissued authorized shares to cover a conversion of the Convertible Notes. As a result, the Company accounted for the portion of the bifurcated conversion feature and of the Capped Call Confirmations that would not be able to be net share settled as a current derivative liability and as a derivative asset, respectively. The fair value of the derivative liability for the conversion feature and derivative asset for the Capped Call Confirmations at February 15, 2018 was determined to be $507.4 million and $13.6 million, respectively, of which the portion that was determined to not be able to be net share settled was recorded with a corresponding impact to additional-paid-in-capital. Subsequent changes to fair value of the derivatives were recorded in the second quarter of 2018 through earnings on the Company’s consolidated statements of operations resulting in a change in fair value of derivatives for the nine months ended September 30, 2018 of $2.7 million. Following the approval by the stockholders of the Company on June 7, 2018, to increase the authorized shares of common stock to 500,000,000, the Company has sufficient unissued authorized shares to cover a conversion of the Convertible Notes. As a result, the derivative liability and derivative asset were reclassified into additional-paid-in-capital. The fair value of the derivative liability for the conversion feature and derivative asset for the Capped Call Confirmations at June 7, 2018 was determined to be $88.3 million and $2.4 million, respectively. The Convertible Notes and Senior Secured Term Loan consist of the following (in thousands):
______________________________________ (1) Included in the consolidated balance sheets within convertible notes and senior secured term loan and amortized to interest expense over the remaining life of the Convertible Notes and Senior Secured Term Loan using the effective interest rate method. The following table sets forth total interest expense recognized related to the Convertible Notes and Senior Secured Term Loan for the three and nine months ended September 30, 2018, respectively:
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Stockholders' Equity |
9 Months Ended |
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Sep. 30, 2018 | |
Equity [Abstract] | |
Stockholders' Equity | Stockholders’ Equity Common Stock and Warrants On February 15, 2018, the Company announced the pricing of an underwritten offering of 19,354,839 shares of its common stock at $15.50 per share, resulting in gross proceeds of $300.0 million. Under the terms of the underwriting agreement, the Company granted the underwriters an option, exercisable for 30 days after February 16, 2018, to purchase up to an additional 2,903,225 shares of the Company’s common stock, which was exercised with respect to 885,000 shares of the Company’s common stock at a purchase price of $15.50 per share. The Company received net proceeds of $294.6 million from these offerings, after deducting underwriting discounts and commissions and offering expenses payable by the Company. In April 2018, 453,214 warrants were exercised at $7.98 per share of common stock resulting in gross cash proceeds of $3.6 million. On June 7, 2018, the Company’s stockholders approved an amendment to the Company’s Restated Certificate of Incorporation to increase the number of shares of common stock, par value $0.01 per share, that the Company is authorized to issue from 250,000,000 shares to 500,000,000 shares. |
Share based Compensation |
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Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Share based Compensation | Share based Compensation The Company’s Equity Incentive Plans consist of the Amended and Restated 2007 Equity Incentive Plan (the “Plan”) and the 2007 Director Option Plan (the “2007 Director Plan”). The Plan provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, advisors and consultants at a price to be determined by the Company’s Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company’s business. The 2007 Director Plan is intended to promote the recruiting and retention of highly qualified eligible directors and strengthen the commonality of interest between directors and stockholders by encouraging ownership of common stock of the Company. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option. Stock Option Grants The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
Beginning in the third quarter of 2017, the average expected life was determined using our actual historical data versus a “simplified” method used in prior quarters. The “simplified” method of estimating the expected exercise term uses the mid-point between the vesting date and the end of the contractual term. In earlier quarters, we did not have sufficient reliable exercise data to justify a change from the use of the “simplified” method of estimating the expected exercise term of employee stock option grants. The impact from this change was not material. A summary of the Company’s stock options for the nine months ended September 30, 2018 were as follows:
As of September 30, 2018, the total unrecognized compensation cost related to non-vested stock options granted was $36.0 million and is expected to be recognized over a weighted average period of three years. Restricted Stock Units (“RSUs”) and Performance-Based Restricted Stock Units RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee’s continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2018 is as follows:
On December 30, 2016, the Compensation Committee approved a form of Performance-Based Restricted Stock Unit Award Agreement (the “Performance-Based RSU Agreement”), to be used for performance-based RSUs granted to participants under the Amended and Restated 2007 Equity Incentive Plan, including named executive officers. Certain awards under the Performance-Based RSU Agreement were granted in January 2017 and 2018. The 2018 grants include 187,222 market performance-based restricted stock units (“MPRSUs”) granted to executives. Vesting of these awards is contingent upon the Company meeting certain total shareholder return (“TSR”) levels as compared to a select peer group over the next three years. The MPRSUs cliff vest at the end of the three-year period and have a maximum potential to vest at 200% (374,444 shares) based on TSR performance. The related share-based compensation expense is determined based on the estimated fair value of the underlying shares on the date of grant and is recognized on a straight-line basis over the vesting term. The estimated fair value per share of the MPRSUs was $25.44 and was calculated using a Monte Carlo simulation model. The awards also include 187,211 performance based awards that will vest over the next three years based on the Company achieving certain clinical milestones. During the nine months ended September 30, 2018, none of the clinical milestones for the performance based RSUs awarded in 2017 or 2018 were reached. For the nine months ended September 30, 2018, 516,940 RSUs have vested and all non-vested units are expected to vest over their normal term. As of September 30, 2018, there was $25.6 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of three years. Compensation Expense Related to Equity Awards The following table summarizes information related to compensation expense recognized in the statements of operations related to the equity awards:
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Assets and Liabilities Measured at Fair Value |
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Fair Value Disclosures [Abstract] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assets and Liabilities Measured at Fair Value | Assets and Liabilities Measured at Fair Value The Company’s financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows: Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly. Level 3 — Inputs that are unobservable for the asset or liability. A summary of the fair value of the Company’s recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2018 are identified in the following table:
A summary of the fair value of the Company’s recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2017 are identified in the following table:
The Company’s Convertible Notes fall into the Level 2 category within the fair value level hierarchy. The fair value was determined using broker quotes in a non-active market for valuation. The fair value of the debt at September 30, 2018 was approximately $531.2 million. The Company’s Senior Secured Term Loan fall into the Level 2 category within the fair value level hierarchy and the fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. The carrying value of the Senior Secured Term Loan approximates the fair value. The Company did not have any Level 3 assets as of September 30, 2018 or December 31, 2017. Cash, Money Market Funds and Marketable Securities The Company classifies its cash within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available for sale debt securities and classifies these assets and the money market funds within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. No changes in valuation techniques or inputs occurred during the nine months ended September 30, 2018. No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the nine months ended September 30, 2018. Contingent Consideration Payable The contingent consideration payable resulted from the acquisition of Callidus Biopharma, Inc. (“Callidus”) in November 2013. The most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Using this approach, expected future cash flows are calculated over the expected life of the agreement, are discounted, and then exercise scenario probabilities are applied. The valuation is performed quarterly. Gains and losses are included in the statement of operations. The contingent consideration payable for Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approach, the estimated fair value could be significantly higher or lower than the fair value the Company determined. The Company may be required to record losses in future periods. The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe program:
Contingent consideration liabilities are remeasured to fair value each reporting period using discount rates, probabilities of payment and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met. The following table shows the change in the balance of contingent consideration payable for the three and nine months ended September 30, 2018 and 2017, respectively:
Deferred Compensation Plan - Investment and Liability The Deferred Compensation Plan (the “Deferral Plan”) provides certain key employees and members of the Board of Directors with an opportunity to defer the receipt of such participant’s base salary, bonus and director’s fees, as applicable. Deferral Plan assets are classified as trading securities and recorded at fair value with changes in the investment’s fair value recognized in the period they occur. The asset investments consist of market exchanged mutual funds. The Company considers its investments in marketable securities as available-for-sale and classifies these assets and related liability within the fair value hierarchy as Level 2, primarily utilizing broker quotes in a non-active market for valuation of these securities. |
Basic and Diluted Net Loss per Common Share |
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Basic and Diluted Net Loss per Common Share | Basic and Diluted Net Loss per Common Share The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
Dilutive common stock equivalents would include the dilutive effect of common stock options, convertible debt units, RSUs and warrants for common stock equivalents. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:
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Collaborations |
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Collaborative Arrangement [Abstract] | |
Collaboratives | Collaborations In October 2018, the Company further expanded its gene therapy portfolio through a collaboration agreement with Penn to pursue research and development of novel gene therapies for four additional indications, including Pompe disease, Fabry disease, CDD and one additional undisclosed rare metabolic disorder. This relationship will combine the Company's protein engineering and glycobiology expertise with Penn’s AAV gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability. In connection with the collaboration agreement, the Company made an upfront payment of $7 million in cash to Penn in October 2018 and agreed to certain milestone payments following the achievement of certain developmental and commercial milestone events by a licensed product in each indication up to an aggregate of $86.5 million per indication. |
Summary of Significant Accounting Policies (Policies) |
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Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The Company has prepared the accompanying unaudited consolidated financial statements in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited financial statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s interim financial information. The accompanying unaudited consolidated financial statements and related notes should be read in conjunction with the Company’s financial statements and related notes as contained in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2017. For a complete description of the Company’s accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2017. |
Consolidation | Consolidation The consolidated financial statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions have been eliminated in consolidation. |
Foreign Currency Transactions | Foreign Currency Transactions The functional currency for most of the Company’s foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company’s foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders’ equity. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. |
Cash, Cash Equivalents, Restricted Cash and Marketable Securities | Cash, Cash Equivalents, Restricted Cash and Marketable Securities The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition, to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company’s consolidated balance sheet. Unrealized holding gains and losses are reported within comprehensive income (loss) in the statements of comprehensive loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations or other observable inputs. Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in non-current assets on the Company’s consolidated balance sheet. |
Concentration of Credit Risk | Concentration of Credit Risk The Company’s financial instruments that are exposed to concentration of credit risk consist primarily of cash and cash equivalents and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on cash and cash equivalents or its marketable securities. The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company’s accounts receivable at September 30, 2018 have arisen from product sales primarily in the EU. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined and the Company evaluates the creditworthiness of each customer on a regular basis. |
Revenue Recognition | Revenue Recognition The Company’s net product sales consist of sales of Galafold® for the treatment of Fabry disease primarily in the EU. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program (“EAP”). Orders for Galafold® are generally received from pharmacies and the ultimate payor is typically a government authority. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company’s customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenues from sales of Galafold® are recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes. The Company elected the portfolio approach practical expedient in applying ASC Topic 606 to its identified revenue streams. Contracts within each revenue stream have similar characteristics and the Company believes this approach would not differ materially from applying ASC Topic 606 to each individual contract. |
Recent Accounting Developments | Recent Accounting Developments - Guidance Adopted in 2018 ASU 2018-07 — In June 2018, the Financial Accounting Standards Board (the “FASB”) issued Accounting Standards Update (“ASU”) 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting (“ASU 2018-07”).These amendments expand the scope of Topic 718, Compensation—Stock Compensation (which currently only includes share-based payments to employees) to include share-based payments issued to non-employees for goods or services. Consequently, the accounting for share-based payments to non-employees and employees will be substantially aligned. ASU 2018-07 supersedes Subtopic 505-50, Equity—Equity-Based Payments to Non-Employees, and is effective for all public entities for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. Early adoption is permitted, but no earlier than a company’s adoption date of Topic 606, Revenue from Contracts with Customers. The Company early adopted ASU 2018-07 in the second quarter of 2018 and there was no material impact on its consolidated financial statements from the adoption. ASU 2018-02 — In February 2018, the FASB issued ASU 2018-02, Income Statement - Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income (“ASU 2018-02”). Prior to ASU 2018-02, U.S. GAAP required the remeasurement of deferred tax assets and liabilities as a result of a change in tax laws or rates to be presented in net income from continuing operations, even in situations in which the related income tax effects of items in accumulated other comprehensive income were originally recognized in other comprehensive income. As a result, such items, referred to as stranded tax effects, did not reflect the appropriate tax rate. Under ASU 2018-02, entities are permitted, but not required, to reclassify from accumulated other comprehensive income to retained earnings those stranded tax effects resulting from the Tax Cuts and Jobs Act of 2017. ASU 2018-02 is effective for all entities for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. Early adoption is permitted. The Company adopted the new standard effective January 1, 2018. As a result of the adoption, the Company reclassified a gain of $383,000 from the foreign currency translation adjustment in accumulated other comprehensive loss to accumulated deficit in the consolidated balance sheet as of September 30, 2018. ASU 2017-09 — In May 2017, the FASB issued ASU 2017-09, Compensation—Stock Compensation (Topic 718): Scope of Modification Accounting (“ASU 2017-09”). The amendments in ASU 2017-09 provide guidance on determining which changes to the terms and conditions of share-based payment awards require an entity to apply modification accounting under ASU 2017-09. An entity should account for the effects of a modification unless all the following are met: (1) The fair value (or calculated value or intrinsic value, if such an alternative measurement method is used) of the modified award is the same as the fair value (or calculated value or intrinsic value, if such an alternative measurement method is used) of the original award immediately before the original award is modified. If the modification does not affect any of the inputs to the valuation technique that the entity uses to value the award, the entity is not required to estimate the value immediately before and after the modification; (2) The vesting conditions of the modified award are the same as the vesting conditions of the original award immediately before the original award is modified; and (3) The classification of the modified award as an equity instrument or a liability instrument is the same as the classification of the original award immediately before the original award is modified. ASU 2017-09 is effective for all entities for annual periods, including interim periods within those annual periods, beginning after December 15, 2017. The Company adopted this standard on January 1, 2018 and as the Company did not have any modification of awards, the adoption of the standard did not have a material impact on its consolidated financial statements. ASU 2017-01 — In January 2017, the FASB issued ASU 2017-01, Business Combinations (Topic 805): Clarifying the Definition of a Business (“ASU 2017-01”). This ASU clarifies the definition of a business. The amendments affect all companies and other reporting organizations that must determine whether they have acquired or sold a business. The amendments in ASU 2017-01 are effective for public companies for annual periods beginning after December 15, 2017, including interim periods within those periods. The amendments should be applied prospectively as of the beginning of the period of adoption. The Company adopted ASU 2017-01 on January 1, 2018. The adoption of the standard did not have a material impact on its consolidated financial statements. For additional information see "—Note 3. Acquisitions." ASU 2016-18 — In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”). The amendments of ASU 2016-18 require an entity to include amounts generally described as restricted cash and restricted cash equivalents with cash and cash equivalents when reconciling the beginning of period and end of period total amounts on the statement of cash flows. The amendments of ASU 2016-18 are effective for annual reporting periods beginning after December 15, 2017, and interim periods within those annual periods. The Company adopted the guidance in ASU 2016-18 effective January 1, 2018. In connection with the adoption of the standard, the Company applied the guidance retrospectively which resulted in an increase in cash flows from operations of $1.1 million on the statement of cash flows for the nine months ended September 30, 2017. ASU 2016-16 — In October 2016, the FASB issued ASU 2016-16, Income Taxes (Topic 740): Intra-Entity Transfers of Assets Other Than Inventory (“ASU 2016-16”). ASU 2016-16 requires an entity to recognize the income tax consequences of an intra-entity transfer of an asset other than inventory when the transfer occurs. The amendments eliminate the exception for an intra-entity transfer of an asset other than inventory. The amendments in ASU 2016-16 are effective for public business entities for annual periods beginning after December 15, 2017, including interim reporting periods within those annual reporting periods. The Company adopted ASU 2016-16 on January 1, 2018 and there was no material impact from the adoption. ASU 2016-01 — In January 2016, the FASB issued ASU No. 2016-01, Financial Instruments-Overall: Recognition and Measurement of Financial Assets and Financial Liabilities (“ASU 2016-01”). ASU 2016-01 changes accounting for equity investments, financial liabilities under the fair value option, and presentation and disclosure requirements for financial instruments. ASU 2016-01 does not apply to equity investments in consolidated subsidiaries or those accounted for under the equity method of accounting. In addition, the FASB clarified guidance related to the valuation allowance assessment when recognizing deferred tax assets resulting from unrealized losses on available-for-sale debt securities. Equity investments with readily determinable fair values will be measured at fair value with changes in fair value recognized in net income. Companies have the option to measure equity investments without readily determinable fair values at either fair value or at cost adjusted for changes in observable prices minus impairment. Companies that elect the fair value option for financial liabilities must recognize changes in fair value related to instrument-specific credit risk in other comprehensive income. Companies must assess valuation allowances for deferred tax assets related to available-for-sale debt securities in combination with their other deferred tax assets. ASU 2016-01 is effective beginning in the first quarter of 2018 and the Company adopted it in the first quarter of 2018. There was no impact on the Company’s consolidated financial statements and related disclosures upon adoption, as the Company does not have equity investments or liabilities with credit risk. In addition, the guidance relating to deferred tax assets did not result in a change in accounting treatment for the Company. ASU 2014-09 — In May 2014, the FASB issued ASU 2014-09, Revenue from Contracts with Customers (Topic 606) which along with amendments issued in 2015 and 2016, replaced substantially all current U.S. GAAP guidance on this topic and eliminated industry-specific guidance. The new revenue recognition standard requires entities to recognize revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. Effective January 1, 2018, the Company adopted the new revenue recognition standard using the modified retrospective approach and applied this approach only to contracts that were not completed as of January 1, 2018. The timing of revenue recognition and treatment of contract costs remains unchanged under the new revenue recognition standard. As such, the adoption of the new revenue recognition standard did not have a material impact on the Company's consolidated financial statements. The information presented for the periods prior to January 1, 2018 has not been restated and is reported under the accounting standard in effect for those periods. Recent Accounting Developments - Guidance Not Yet Adopted In August 2018, the Securities Exchange Commission (“SEC”) issued Final Rule 33-10532, Disclosure Update and Simplification, which amends certain disclosure requirements that were redundant, duplicative, overlapping or superseded by other SEC disclosure requirements. The amendments generally eliminated or otherwise reduced certain disclosure requirements of various SEC rules and regulations. However, in some cases, the amendments require additional information to be disclosed, including changes in stockholders’ equity in interim periods. The rule is effective 30 days after its publication in the Federal Register. The rule was posted on October 4, 2018. On September 25, 2018, the SEC released guidance advising it will not object to a registrant adopting the requirement to include changes in stockholders’ equity in the Form 10-Q for the first quarter beginning after the effective date of the rule. The Company is currently assessing the impact that this standard will have on its consolidated financial statements upon adoption and expects to adopt the guidance in it's Form 10-Q for the period ended March 31, 2019. ASU 2018-13 — In August 2018, the FASB issued ASU 2018-03, Fair Value Measurement (Topic 820): Disclosure Framework-Changes to the Disclosure Requirements for Fair Value Measurement (“ASU 2018-13”). The amendments modify the disclosure requirements in Topic 820. ASU 2018-13 is effective for all entities for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. The amendments on changes in unrealized gains and losses, the range and weighted average of significant unobservable inputs used to develop Level 3 fair value measurements, and the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. Early adoption is permitted. An entity is permitted to early adopt any removed or modified disclosures upon issuance of ASU 2018-13 and delay adoption of the additional disclosures until their effective date. The Company is currently assessing the impact that this standard will have on its consolidated financial statements upon adoption. ASU 2017-12 — In August 2017, the FASB issued ASU 2017-12, Derivatives and Hedging (Topic 815): Targeted Improvements to Accounting for Hedging Activities (“ASU 2017-12”). The amendments in this ASU 2017-12 better align an entity’s risk management activities and financial reporting for hedging relationships through changes to both the designation and measurement guidance for qualifying hedging relationships and the presentation of hedge results. To meet that objective, the amendments expand and refine hedge accounting for both nonfinancial and financial risk components and align the recognition and presentation of the effects of the hedging instrument and the hedged item in the financial statements. The amendments in ASU 2017-12 also make certain targeted improvements to simplify the application of hedge accounting guidance and ease the administrative burden of hedge documentation requirements and assessing hedge effectiveness. For public business entities, the amendment is effective for fiscal years beginning after December 15, 2018, and interim periods within those fiscal years. Early adoption, including adoption in an interim period, is permitted. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2017-11 — In July 2017, the FASB issued ASU 2017-11, Earnings Per Share (Topic 260); Distinguishing Liabilities from Equity (Topic 480); Derivatives and Hedging (Topic 815): (Part I) Accounting for Certain Financial Instruments with Down Round Features, (Part II) Replacement of the Indefinite Deferral for Mandatorily Redeemable Financial Instruments of Certain Nonpublic Entities and Certain Mandatorily Redeemable Noncontrolling Interests with a Scope Exception (“ASU 2017-11”). Part I of ASU 2017-11 addresses the complexity of accounting for certain financial instruments with down round features. Down round features are features of certain equity-linked instruments (or embedded features) that result in the strike price being reduced on the basis of the pricing of future equity offerings. When determining whether certain financial instruments should be classified as liabilities or equity instruments, a down round feature no longer precludes equity classification when assessing whether the instrument is indexed to an entity’s own stock. Part II of ASU 2017-11 addresses the difficulty of navigating Topic 480, Distinguishing Liabilities from Equity, because of the existence of extensive pending content in the FASB Accounting Standards Codification®. For public business entities, the amendments in Part I of ASU 2017-11 are effective for fiscal years beginning after December 15, 2018. Early adoption is permitted for all entities, including adoption in an interim period. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2017-08 — In March 2017, the FASB issued ASU 2017-08, Receivables—Nonrefundable Fees and Other Costs (Subtopic 310-20), Premium Amortization on Purchased Callable Debt Securities (“ASU 2017-08”). The amendments in ASU 2017-08 shorten the amortization period for certain callable debt securities held at a premium. Specifically, the amendments require the premium to be amortized to the earliest call date. The amendments do not require an accounting change for securities held at a discount; the discount continues to be amortized to maturity. ASU 2017-08 is effective for public business entities for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2018. Early adoption is permitted, including adoption in an interim period. If an entity early adopts in an interim period, any adjustments should be reflected as of the beginning of the fiscal year that includes that interim period. The amendments should be applied on a modified retrospective basis, with a cumulative-effect adjustment directly to retained earnings as of the beginning of the period of adoption. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2017-04 — In January 2017, the FASB issued ASU 2017-04, Intangibles - Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment (“ASU 2017-04”). To simplify the subsequent measurement of goodwill, ASU 2017-04 eliminates Step 2 from the goodwill impairment test. The annual, or interim, goodwill impairment test is performed by comparing the fair value of a reporting unit with its carrying amount. An impairment charge should be recognized for the amount by which the carrying amount exceeds the reporting unit’s fair value; however, the loss recognized should not exceed the total amount of goodwill allocated to that reporting unit. In addition, income tax effects from any tax deductible goodwill on the carrying amount of the reporting unit should be considered when measuring the goodwill impairment loss, if applicable. ASU 2017-04 also eliminates the requirements for any reporting unit with a zero or negative carrying amount to perform a qualitative assessment and, if it fails that qualitative test, to perform Step 2 of the goodwill impairment test. An entity still has the option to perform the qualitative assessment for a reporting unit to determine if the quantitative impairment test is necessary. ASU 2017-04 should be applied on a prospective basis. The nature of and reason for the change in accounting principle should be disclosed upon transition. A public business entity that is a U.S. SEC filer should adopt ASU 2017-04 for its annual or any interim goodwill impairment tests in fiscal years beginning after December 15, 2019. Early adoption is permitted for interim or annual goodwill impairment tests performed on testing dates after January 1, 2017. The Company is currently assessing the impact that this standard will have on its consolidated financial statements. ASU 2016-02 — In February 2016, the FASB issued ASU 2016-02, Leases (Topic 842) (“ASU 2016-02”). ASU 2016-02 requires the recognition of lease assets and lease liabilities on the balance sheet for all lease obligations and disclosing key information about leasing arrangements. ASU 2016-02 requires the recognition of lease assets and lease liabilities by lessees for those leases classified as operating leases under previous generally accepted accounting principles. ASU 2016-02 will be effective for the Company for all annual and interim periods beginning after December 15, 2018, including interim periods within those fiscal years. In August 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements, (“ASU 2018-11”). ASU 2018-11 provide entities with an additional transition method for adoption, whereby, an entity initially applies the new leases standard at the adoption date and recognizes a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. The Company will adopt these standards effective January 1, 2019, and expects to elect certain of the practical expedients permitted, including the expedient that permits the Company to retain its existing lease assessment and classification. The Company also expects to elect the transition method in ASU 2018-11. The Company is currently working through an adoption plan which includes the evaluation of lease contracts compared to the new standard. While the Company is currently assessing the impact that this standard will have on its consolidated financial statements, the Company expects to recognize lease liabilities and right of use assets. |
Cash, Cash Equivalents, Marketable Securities and Restricted Cash (Tables) |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2018 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Cash, Cash Equivalents, and Short-term Investments [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of cash and available-for-sale securities | Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:
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Schedule of restricted cash and cash equivalents | The following table provides a reconciliation of cash and cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same such amounts shown in the statement of cash flows.
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Inventories (Tables) |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2018 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Inventory Disclosure [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of inventories for the period | The following table summarizes the components of inventories:
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Debt (Tables) |
9 Months Ended | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2018 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Debt Disclosure [Abstract] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of liability components of the Convertible Notes | The Convertible Notes and Senior Secured Term Loan consist of the following (in thousands):
______________________________________ (1) Included in the consolidated balance sheets within convertible notes and senior secured term loan and amortized to interest expense over the remaining life of the Convertible Notes and Senior Secured Term Loan using the effective interest rate method. |
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Components of total interest expense recognized related to the Convertible Notes | The following table sets forth total interest expense recognized related to the Convertible Notes and Senior Secured Term Loan for the three and nine months ended September 30, 2018, respectively:
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Share based Compensation (Tables) |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2018 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of Fair Value of Options | The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:
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Schedule of Stock Options Activity | A summary of the Company’s stock options for the nine months ended September 30, 2018 were as follows:
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Schedule of Non-Vested RSU Activity under the Plan | A summary of non-vested RSU activity under the Plan for the nine months ended September 30, 2018 is as follows:
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Schedule of Equity Compensation Expenses | The following table summarizes information related to compensation expense recognized in the statements of operations related to the equity awards:
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Assets and Liabilities Measured at Fair Value (Tables) |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2018 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Summary of assets and liabilities subject to fair value measurements | A summary of the fair value of the Company’s recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of September 30, 2018 are identified in the following table:
A summary of the fair value of the Company’s recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2017 are identified in the following table:
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Schedule of changes in contingent consideration payable | The following table shows the change in the balance of contingent consideration payable for the three and nine months ended September 30, 2018 and 2017, respectively:
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Callidus Biopharma Inc | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of significant unobservable inputs used in the valuation of the contingent consideration payable | The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe program:
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Basic and Diluted Net Loss per Common Share (Tables) |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2018 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Earnings Per Share [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of reconciliation of the numerator and denominator used in computing basic and diluted net loss per common share | The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:
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Schedule of potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method | The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:
|
Summary of Significant Accounting Policies - Foreign Currency Transactions (Details) - USD ($) $ in Thousands |
9 Months Ended | ||
---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Dec. 31, 2017 |
|
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Total stockholders’ equity | $ 410,951 | $ 352,850 | |
Retained earnings (accumulated deficit) | (1,334,141) | $ (1,063,610) | |
Net cash used in operating activities | (239,278) | $ (140,661) | |
Accounting Standards Update 2018-02 | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Retained earnings (accumulated deficit) | 383 | ||
Accounting Standards Update 2016-18 | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Net cash used in operating activities | $ 1,100 | ||
Accumulated Foreign Currency Adjustment Attributable | Accounting Standards Update 2018-02 | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Total stockholders’ equity | $ (383) |
Acquisitions (Details) - Celenex $ in Millions |
1 Months Ended |
---|---|
Sep. 30, 2018
USD ($)
| |
Business Acquisition [Line Items] | |
Upfront cash payment to acquire assets | $ 100.0 |
Development milestones | 15.0 |
Submission and approval milestones | 262.0 |
Maximum expected payment over next four years | 75.0 |
Research and development expense | 100.0 |
Nationwide Children's Hospital | |
Business Acquisition [Line Items] | |
Development milestones | $ 7.8 |
Cash, Cash Equivalents, Marketable Securities and Restricted Cash - Additional Information (Details) - USD ($) |
9 Months Ended | 12 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Dec. 31, 2017 |
Sep. 30, 2017 |
Dec. 31, 2016 |
|
Cash, Cash Equivalents, and Short-term Investments [Abstract] | ||||
Realized gain (loss) on debt securities | $ 0 | $ 0 | ||
Fair value of available-for-sale debt securities in unrealized loss positions | 315,300,000 | 295,100,000 | ||
Cash and cash equivalents | 201,827,000 | 49,060,000 | $ 64,133,000 | $ 187,026,000 |
Restricted cash | 2,003,000 | 2,177,000 | 1,444,000 | 387,000 |
Cash and cash equivalents and restricted cash shown in the statement of cash flows | $ 203,830,000 | $ 51,237,000 | $ 65,577,000 | $ 187,413,000 |
Inventories (Details) - USD ($) $ in Thousands |
Sep. 30, 2018 |
Dec. 31, 2017 |
---|---|---|
Inventory Disclosure [Abstract] | ||
Raw materials | $ 2,954 | $ 2,394 |
Work-in-process | 1,458 | 1,449 |
Finished goods | 1,899 | 780 |
Total inventories | $ 6,311 | $ 4,623 |
Share based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Sep. 30, 2018 |
Sep. 30, 2017 |
|
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected stock price volatility | 75.50% | 82.50% | 80.70% | 83.10% |
Risk free interest rate | 2.70% | 1.80% | 2.40% | 2.00% |
Expected life of options (years) | 5 years 7 months 15 days | 5 years 10 months 20 days | 5 years 7 months 15 days | 6 years 2 months 16 days |
Expected annual dividend per share (in dollars per share) | $ 0 | $ 0 | $ 0 | $ 0 |
Share based Compensation - Expense Summary (Details) - USD ($) $ in Thousands |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Sep. 30, 2018 |
Sep. 30, 2017 |
|
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total equity compensation expense | $ 7,054 | $ 5,500 | $ 20,873 | $ 17,067 |
Research and development expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total equity compensation expense | 2,905 | 2,390 | 8,603 | 7,456 |
Selling, general and administrative expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total equity compensation expense | $ 4,149 | $ 3,110 | $ 12,270 | $ 9,611 |
Assets and Liabilities Measured at Fair Value - Level 3 Rollforward (Details) - USD ($) $ in Thousands |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2018 |
Sep. 30, 2017 |
Sep. 30, 2018 |
Sep. 30, 2017 |
|
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | ||||
Balance, beginning of the period | $ 26,800 | $ 265,350 | $ 25,400 | $ 269,722 |
Payment of contingent consideration in cash | 0 | 0 | 0 | (10,000) |
Changes in fair value during the period, included in statement of operations | 1,300 | (244,250) | 2,700 | (238,622) |
Balance, end of the period | $ 28,100 | $ 21,100 | $ 28,100 | $ 21,100 |
Collaborations (Details) - Collaborative Arrangement - Subsequent Event $ in Millions |
1 Months Ended |
---|---|
Oct. 31, 2018
USD ($)
| |
Collaborative Agreements | |
Payments for collaborative arrangement | $ 7.0 |
Conditional aggregate payments per indication | $ 86.5 |
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