Item 1. Financial Statements
Item 1. Financial Statements
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(Unaudited)
(In thousands, except share amounts)
September 30,
December 31,
2020
2019
Assets
Current assets:
Cash and cash equivalents
$
197,486
$
433,584
Short-term investments
568,034
321,646
Accounts receivable, net
27,762
32,844
Inventory
14,457
11,546
Prepaid expenses and other current assets
64,264
51,397
Total current assets
872,003
851,017
Property and equipment, net
48,773
44,348
Investments in equity securities
129,320
27,752
Long-term investments
—
5,174
Right-of-use assets
37,597
30,328
Intangible assets, net
131,194
129,000
Goodwill
44,406
44,406
Other assets
5,092
3,471
Total assets
$
1,268,385
$
1,135,496
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable
$
10,850
$
12,871
Accrued liabilities
79,653
83,194
Contract liabilities, net
102,898
—
Short-term lease liabilities
7,785
7,235
Total current liabilities
201,186
103,300
Long-term lease liabilities
36,668
29,757
Deferred tax liabilities
33,306
33,306
Liability related to the sale of future royalties
331,034
315,369
Total liabilities
602,194
481,732
Stockholders’ equity:
Preferred stock — 25,000,000 shares authorized; nil outstanding as of September 30, 2020 and
December 31, 2019
—
—
Common stock — 250,000,000 shares authorized; 60,815,690 and 57,838,220 shares issued
and outstanding as of September 30, 2020 and December 31, 2019, respectively
61
58
Additional paid-in capital
2,260,682
2,086,863
Accumulated other comprehensive income (loss)
1,013
( 147
)
Accumulated deficit
( 1,595,565
)
( 1,433,010
)
Total stockholders’ equity
666,191
653,764
Total liabilities and stockholders’ equity
$
1,268,385
$
1,135,496
See accompanying notes.
2
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
(In thousands, except share and per share amounts)
Three Months Ended September 30,
Nine Months Ended September 30,
2020
2019
2020
2019
Revenues:
Collaboration and license
$
66,924
$
21,585
$
144,300
$
55,070
Product sales
11,215
4,215
25,760
13,051
Non-cash collaboration royalty revenue
3,331
—
9,428
—
Total revenues
81,470
25,800
179,488
68,121
Operating expenses:
Cost of sales
2,348
2,683
648
3,901
Research and development
87,314
100,144
280,984
274,294
Selling, general and administrative
42,123
41,006
131,891
119,647
Total operating expenses
131,785
143,833
413,523
397,842
Loss from operations
( 50,315
)
( 118,033
)
( 234,035
)
( 329,721
)
Interest income
1,338
3,319
6,054
10,468
Change in fair value of investments in equity securities
( 11,520
)
2,166
91,348
11,994
Non-cash interest expense on liability related to the sale of future
royalties
( 8,582
)
—
( 25,093
)
—
Other income (expense)
547
( 153
)
308
( 941
)
Loss before income taxes
( 68,532
)
( 112,701
)
( 161,418
)
( 308,200
)
Provision for income taxes
( 313
)
( 293
)
( 1,137
)
( 722
)
Net loss
$
( 68,845
)
$
( 112,994
)
$
( 162,555
)
$
( 308,922
)
Net loss per share, basic and diluted
$
( 1.13
)
$
( 1.96
)
$
( 2.73
)
$
( 5.50
)
Weighted-average shares used in computing net loss per share,
basic and diluted
60,687,177
57,707,694
59,564,163
56,161,996
See accompanying notes.
3
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(Unaudited)
(In thousands)
Three Months Ended September 30,
Nine Months Ended September 30,
2020
2019
2020
2019
Net loss
$
( 68,845
)
$
( 112,994
)
$
( 162,555
)
$
( 308,922
)
Other comprehensive income:
Foreign currency translation adjustments
374
( 234
)
414
( 79
)
Unrealized gain (loss) on available-for-sale securities
( 645
)
( 92
)
746
641
Other comprehensive income (loss)
( 271
)
( 326
)
1,160
562
Total comprehensive loss
$
( 69,116
)
$
( 113,320
)
$
( 161,395
)
$
( 308,360
)
See accompanying notes.
4
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY
(Unaudited)
(In thousands, except share amounts)
Common Stock
Additional
Paid-In
Accumulated
Other
Comprehensive
Accumulated
Total
Stockholders'
Shares
Amount
Capital
Income (Loss)
Deficit
Equity
Balance as of June 30, 2020
60,413,756
$
60
$
2,220,963
$
1,284
$
( 1,526,720
)
$
695,587
Employee stock-based compensation
—
—
20,527
—
—
20,527
Issuance of common stock under equity
plan awards, net of tax
401,934
1
19,192
—
—
19,193
Other comprehensive loss
—
—
—
( 271
)
—
( 271
)
Net loss
—
—
—
—
( 68,845
)
( 68,845
)
Balance as of September 30, 2020
60,815,690
$
61
$
2,260,682
$
1,013
$
( 1,595,565
)
$
666,191
Common Stock
Additional
Paid-In
Accumulated
Other
Comprehensive
Accumulated
Total
Stockholders'
Shares
Amount
Capital
Income (Loss)
Deficit
Equity
Balance as of December 31, 2019
57,838,220
$
58
$
2,086,863
$
( 147
)
$
( 1,433,010
)
$
653,764
Issuance of common stock in connection
with license agreement, net of issuance
costs
1,243,913
1
55,267
—
—
55,268
Issuance of common stock in connection with
at-the-market offering, net of issuance costs
283,333
—
20,391
—
—
20,391
Employee stock-based compensation
—
—
63,112
—
—
63,112
Issuance of common stock upon exercise of
warrants and under equity plan awards, net
of tax
1,450,224
2
35,049
—
—
35,051
Other comprehensive income
—
—
—
1,160
—
1,160
Net loss
—
—
—
—
( 162,555
)
( 162,555
)
Balance as of September 30, 2020
60,815,690
$
61
$
2,260,682
$
1,013
$
( 1,595,565
)
$
666,191
Common Stock
Additional
Paid-In
Accumulated
Other
Comprehensive
Accumulated
Total
Stockholders'
Shares
Amount
Capital
Income (Loss)
Deficit
Equity
Balance as of June 30, 2019
57,665,375
$
58
$
2,045,685
$
255
$
( 1,226,211
)
$
819,787
Employee stock-based compensation
—
—
19,894
—
—
19,894
Issuance of common stock under equity plan
awards, net of tax
84,515
—
904
—
—
904
Other comprehensive loss
—
—
—
( 326
)
—
( 326
)
Net loss
—
—
—
—
( 112,994
)
( 112,994
)
Balance as of September 30, 2019
57,749,890
$
58
$
2,066,483
$
( 71
)
$
( 1,339,205
)
$
727,265
5
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY
(Unaudited)
(In thousands, except share amounts)
Common Stock
Additional
Paid-In
Accumulated
Other
Comprehensive
Accumulated
Total
Stockholders'
Shares
Amount
Capital
Income (Loss)
Deficit
Equity
Balance as of December 31, 2018
50,860,588
$
51
$
1,639,773
$
( 633
)
$
( 1,030,283
)
$
608,908
Issuance of common stock in connection with
underwritten public offering, net of
issuance costs
5,833,333
6
330,409
—
—
330,415
Issuance of common stock in connection with
at-the-market offering, net of issuance costs
468,685
—
24,828
—
—
24,828
Employee stock-based compensation
—
—
62,854
—
—
62,854
Issuance of common stock under equity plan
awards, net of tax
587,284
1
8,619
—
—
8,620
Other comprehensive income
—
—
—
562
—
562
Net loss
—
—
—
—
( 308,922
)
( 308,922
)
Balance as of September 30, 2019
57,749,890
$
58
$
2,066,483
$
( 71
)
$
( 1,339,205
)
$
727,265
See accompanying notes.
6
ULTRAGENYX PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(Unaudited)
(In thousands)
Nine Months Ended September 30,
2020
2019
Operating activities:
Net loss
$
( 162,555
)
$
( 308,922
)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation
62,897
62,335
Amortization of premium (discount) on investment securities, net
217
( 4,931
)
Depreciation and amortization
9,128
6,370
Foreign currency remeasurement loss
167
925
Change in fair value of investments in equity securities
( 91,348
)
( 11,994
)
Non-cash collaboration royalty revenue
( 9,428
)
—
Non-cash interest expense on liability related to the sale of future royalties
25,093
—
Other
( 28
)
220
Changes in operating assets and liabilities:
Accounts receivable
5,126
( 10,402
)
Inventory
( 2,635
)
( 6,604
)
Prepaid expenses and other assets
( 12,461
)
( 5,874
)
Right-of-use assets
( 7,261
)
( 15,581
)
Accounts payable, accrued, and other liabilities
2,822
4,043
Contract liabilities, net
102,898
—
Lease liabilities
7,600
17,071
Net cash used in operating activities
( 69,768
)
( 273,344
)
Investing activities:
Purchase of property and equipment
( 21,959
)
( 13,332
)
Purchase of investments
( 648,524
)
( 610,267
)
Purchase of investments in equity securities
( 10,220
)
( 14,339
)
Purchase of convertible notes receivable
( 1,880
)
—
Proceeds from the sale of investments
40,390
35,100
Proceeds from maturities of investments
367,446
522,533
Payment for intangible assets
( 2,275
)
—
Net cash used in investing activities
( 277,022
)
( 80,305
)
Financing activities:
Proceeds from the issuance of common stock in connection with the license agreement, net
55,268
—
Proceeds from the issuance of common stock in connection with underwritten public
offerings, net
—
330,415
Proceeds from the issuance of common stock in connection with at-the-market offering, net
20,391
24,828
Proceeds from the issuance of common stock from exercise of warrants and equity plan
awards, net
35,051
8,620
Principal repayments of financing leases
( 149
)
—
Net cash provided by financing activities
110,561
363,863
Effect of exchange rate changes on cash
239
( 502
)
Net increase (decrease) in cash, cash equivalents and restricted cash
( 235,990
)
9,712
Cash, cash equivalents and restricted cash at beginning of period
436,244
115,525
Cash, cash equivalents and restricted cash at end of period
$
200,254
$
125,237
Supplemental disclosures of non-cash information:
Acquired lease liabilities arising from obtaining right-of-use assets
$
13,195
$
21,515
See accompanying notes.
7
ULTRAGENYX PHARMACEUTICAL INC.
Notes to Condensed Consolidated Financial Statements
1.
Organization
Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company incorporated in California on April 22, 2010. The Company subsequently reincorporated in the state of Delaware in June 2011.
The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare genetic diseases. The Company has four approved therapies. Crysvita ® (burosumab) is approved in the United States by the U.S. Food and Drug Administration (FDA) and in Canada for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older, and has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. In October 2020, this authorization was expanded to include older adolescents and adults. In Brazil, Crysvita is approved for treatment of XLH in adult and pediatric patients one year of age and older. Crysvita was approved by the FDA on June 18, 2020 for the treatment of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO), associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.
The Company has also received FDA approval for Mepsevii™ (vestronidase alfa), the first medicine approved for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. In the European Union and the United Kingdom, Mepsevii is approved under exceptional circumstances for patients of all ages for the treatment of non-neurological manifestations of MPS VII. In Brazil, Mepsevii is approved for the treatment of MPS VII for patients of all ages.
Dojolvi, formerly known as UX007, was approved by FDA on June 30, 2020 for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD).
In addition to the approved treatments, the Company has two ongoing clinical development programs. DTX301 is an adeno-associated virus 8 (AAV8) gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder; and DTX401 is an AAV8 gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia (GSDIa). The Company operates as one reportable segment.
The Company has sustained operating losses and expects such annual losses to continue over the next several years. The Company’s ultimate success depends on the outcome of its research and development and commercialization activities, for which it expects to incur additional losses in the future. Management recognizes that the Company will likely need to raise additional capital to fully implement its business plans. Through September 30, 2020, the Company has relied primarily on the proceeds from equity offerings and its sale of future royalties to finance its operations.
The Company will likely raise additional capital through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company would need to reevaluate its operating plans.
2.
Summary of Significant Accounting Policies
Basis of Presentation
The accompanying unaudited Condensed Consolidated Financial Statements include the accounts of the Company and its wholly-owned subsidiaries and have been prepared in accordance with U.S. generally accepted accounting principles (GAAP) for interim financial information and in accordance with the instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. The unaudited interim consolidated financial statements have been prepared on the same basis as the annual financial statements. In the opinion of management, the accompanying unaudited Condensed Consolidated financial Statements reflect all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation. These financial statements should be read in conjunction with the audited financial statements and notes thereto for the preceding fiscal year contained in the Company’s Annual Report on Form 10-K filed on February 14, 2020 with the United States Securities and Exchange Commission (SEC).
The results of operations for the three and nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for the year ending December 31, 2020. The Condensed Consolidated Balance Sheet as of December 31, 2019 has been derived from audited financial statements at that date, but does not include all of the information required by GAAP for complete financial statements.
8
Use of Estimates
The accompanying consolidated financial statements have been prepared in accordance with GAAP. The preparation of the consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the consolidated financial statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trial accruals, fair value of assets and liabilities, income taxes, stock-based compensation, and the liability related to the sale of future royalties. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates.
Cash, Cash Equivalents and Restricted Cash
Restricted cash primarily consists of money market accounts as collateral for the Company’s obligations under its facility leases. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheets that sum to the total of the same such amounts shown in the consolidated statement of cash flows (in thousands):
September 30,
2020
2019
Cash and cash equivalents
$
197,486
$
122,577
Restricted cash included in prepaid expenses and
other current assets
847
161
Restricted cash included in other assets
1,921
2,499
Total cash, cash equivalents, and restricted cash
shown in the statements of cash flows
$
200,254
$
125,237
Credit Losses
Effective January 1, 2020, the Company adopted Accounting Standards Update (ASU) 2016-13, Financial Instruments — Credit Losses, (Topic 326): Measurement of Credit Losses on Financial Instruments, which changed the impairment model for most financial assets and certain other instruments. For trade receivables and other instruments, the Company uses a new forward-looking expected loss model that generally results in the earlier recognition of allowances for losses. For available-for-sale debt securities with unrealized losses, the losses are recognized as allowances rather than as reductions in the amortized cost of the securities.
The Company is exposed to credit losses primarily through receivables from customers and collaborators and through its available-for-sale debt securities. The Company’s expected loss allowance methodology for the receivables is developed using historical collection experience, current and future economic market conditions, a review of the current aging status and financial condition of the entities. Specific allowance amounts are established to record the appropriate allowance for customers that have a higher probability of default. Balances are written off when determined to be uncollectible. The Company’s expected loss allowance methodology for the debt securities is developed by reviewing the extent of the unrealized loss, the size, term, geographical location, and industry of the issuer, the issuers’ credit ratings and any changes in those ratings, as well as reviewing current and future economic market conditions and the issuers’ current status and financial condition. The Company considered the current and expected future economic and market conditions surrounding the novel coronavirus (COVID-19) pandemic and determined that the estimate of credit losses was not significantly impacted. The adoption of ASU 2016-13 did not have a material impact on the Condensed Consolidated Financial Statements and related disclosures and there was no allowance for losses on available-for-sale debt securities which were attributable to credit risk for the three and nine months ended September 30, 2020.
Revenue Recognition
Collaboration and license revenue
The Company has certain license and collaboration agreements that are within the scope of Accounting Standards Codification (ASC) 808, Collaborative Agreements , which provides guidance on the presentation and disclosure of collaborative arrangements. Generally, the classification of the transactions under the collaborative arrangements is determined based on the nature of contractual terms of the arrangement, along with the nature of the operations of the participants. The Company records its share of collaboration revenue, net of transfer pricing related to net sales in the period in which such sales occur, if the Company is considered as an agent in the arrangement. The Company is considered an agent when the collaboration partner controls the product before transfer to the customers and has the ability to direct the use of and obtain substantially all of the remaining benefits from the product. Funding received related to research and development services and commercialization costs is generally classified as a reduction of research and development expenses and selling, general and administrative expenses, respectively, in the consolidated statement of operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations.
9
The Company also re cords royalty revenues under certain of the Company’s license or collaboration agreements in exchange for license of intellectual property. If the Company does not have any future performance obligations for these license or collaboration agreements, royalty revenue is recorded as the underlying sales occur.
In order to record collaboration revenue, the Company utilizes certain information from its collaboration partners , including revenue from the sale of the product, associated reserves on revenue, and costs incurred for development and sales activities. For the periods covered in the financial statements presented, there have been no material changes to prior period estimates of revenues and expenses.
The terms of the Company’s collaboration and license agreements may contain multiple performance obligations, which may include licenses and research and development activities. The Company evaluates these agreements under ASC 606, Revenue from Contracts with Customers (ASC 606), to determine the distinct performance obligations. The Company analogizes to ASC 606 for the accounting for distinct performance obligations for which there is a customer relationship. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Total consideration may include nonrefundable upfront license fees, payments for research and development activities, reimbursement of certain third-party costs, payments based upon the achievement of specified milestones, and royalty payments based on product sales derived from the collaboration.
If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined based on the prices charged to customers or using expected cost-plus margin. The Company estimates the efforts needed to complete the performance obligations and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligations using input measures.
Product sales
The Company sells its approved products through a limited number of distributors. Under ASC 606, revenue from product sales is recognized at the point in time when the delivery is made and when title and risk of loss transfers to these distributors. The Company also recognizes revenue from sales of certain products on a “named patient” basis, which are allowed in certain countries prior to the commercial approval of the product. Prior to recognizing revenue, the Company makes estimates of the transaction price, including any variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Product sales are recorded net of estimated government-mandated rebates and chargebacks, estimated product returns, and other deductions.
Provisions for returns and other adjustments are provided for in the period the related revenue is recorded, as estimated by management. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are reviewed periodically and adjusted as necessary. If actual results vary, the Company may need to adjust these estimates, which could have an effect on earnings in the period of the adjustment.
Non-cash collaboration royalty revenue
Effective January 1, 2020, the Company sold the right to receive certain royalty payments from net sales of Crysvita to RPI Finance Trust (RPI), an affiliate of Royalty Pharma, as further described in Note 7. The Company records the royalty revenue from the net sales of Crysvita in the applicable European territories on a prospective basis as non-cash royalty revenue in the Consolidated Statements of Operations over the term of the arrangement.
3.
Financial Instruments
Financial assets and liabilities are recorded at fair value. The carrying amount of certain financial instruments, including cash and cash equivalents, accounts receivable, accounts payable and accrued liabilities approximate fair value due to their relatively short maturities. Assets and liabilities recorded at fair value on a recurring basis in the balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:
Level 1 —Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date;
Level 2 —Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities; and
Level 3 —Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data.
10
The following tables set forth the fair value of the Company’s financial assets remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands):
September 30, 2020
Level 1
Level 2
Level 3
Total
Money market funds
$
135,764
$
—
$
—
$
135,764
Repurchase agreements
—
33,000
—
33,000
Time deposits
—
10,000
—
10,000
Corporate bonds
—
193,677
—
193,677
Commercial paper
—
102,891
—
102,891
Asset-backed securities
—
8,715
—
8,715
U.S. Government Treasury and agency securities
214,923
47,827
—
262,750
Investment in Arcturus common stock
128,700
—
—
128,700
Total
$
479,387
$
396,110
$
—
$
875,497
December 31, 2019
Level 1
Level 2
Level 3
Total
Money market funds
$
293,309
$
—
$
—
$
293,309
Repurchase agreements
—
100,000
—
100,000
Time deposits
—
10,000
—
10,000
Corporate bonds
—
77,026
—
77,026
Commercial paper
—
80,119
—
80,119
Asset-backed securities
—
30,406
—
30,406
U.S. Government Treasury and agency securities
96,329
53,979
—
150,308
Investment in Arcturus equity securities
26,088
—
1,664
27,752
Total
$
415,726
$
351,530
$
1,664
$
768,920
The Company determined the fair value of the Arcturus Therapeutics Holdings Inc. (Arcturus) common stock by using the quoted market price on September 30, 2020, which is a Level 1 fair value measurement. See Note 6 for additional details on the Arcturus transaction.
In July 2020, the Company invested $ 2.5 million in DiaCarta Ltd. (DiaCarta), a private diagnostic company, in the form of a convertible promissory note that matures in two years , if not converted earlier. The Company was also issued a warrant to purchase up to $ 1.0 million of DiaCarta preferred stock. The fair value of the warrant to purchase shares of DiaCarta was based on unobservable inputs that are significant to the measurement of the fair value of the asset and is supported by little or no market data; accordingly, the warrant is considered a Level 3 financial asset and is remeasured on a nonrecurring basis. The Company measured the fair value of the warrant by applying the Black-Scholes option pricing method and utilizing the following inputs: stock price, strike price, volatility, risk-free interest rate, and expected term. The Company will recognize interest income on the convertible promissory note based on the effective interest rate method over the life of the note. As of September 30, 2020, the balance of the convertible promissory note was $ 2.0 million, including $ 0.1 million in interest receivable, and was recorded in other assets, and the allocated fair value of the warrant was $ 0.6 million and was recorded in investments in equity securities.
11
4.
Balance Sheet Components
Cash Equivalents and Investments
The fair values of cash equivalents and investments classified as available-for-sale securities consisted of the following (in thousands):
September 30, 2020
Gross Unrealized
Amortized
Cost
Gains
Losses
Estimated
Fair Value
Money market funds
$
135,764
$
—
$
—
$
135,764
Repurchase agreements
33,000
—
—
33,000
Time deposits
10,000
—
—
10,000
Corporate bonds
192,943
741
( 7
)
193,677
Commercial paper
102,890
1
—
102,891
Asset-backed securities
8,712
3
—
8,715
U.S. Government Treasury and agency securities
262,583
167
—
262,750
Total
$
745,892
$
912
$
( 7
)
$
746,797
December 31, 2019
Gross Unrealized
Amortized
Cost
Gains
Losses
Estimated
Fair Value
Money market funds
$
293,309
$
—
$
—
$
293,309
Repurchase agreements
100,000
—
—
100,000
Time deposits
10,000
—
—
10,000
Corporate bonds
77,022
17
( 13
)
77,026
Commercial paper
80,119
—
—
80,119
Asset-backed securities
30,375
31
—
30,406
U.S. Government Treasury and agency securities
150,184
124
—
150,308
Total
$
741,009
$
172
$
( 13
)
$
741,168
At September 30, 2020, the remaining contractual maturities of available-for-sale securities were less than one year . There have been no realized gains or losses on available-for-sale securities for the three and nine months ended September 30, 2020 and 2019. All marketable securities with unrealized losses at September 30, 2020 have been in a loss position for less than twelve months. Based on the Company’s application of its expected loss allowance methodology, it is probable that the principal and interest will be collected in accordance with the contractual terms, and that the unrealized loss on these securities were not attributable to credit risk.
Inventory
Inventory consists of the following (in thousands):
September 30,
December 31,
2020
2019
Work-in-process
$
10,211
$
8,191
Finished goods
4,246
3,355
Total inventory
$
14,457
$
11,546
Accrued Liabilities
Accrued liabilities consist of the following (in thousands):
September 30,
December 31,
2020
2019
Research, clinical study, and manufacturing expenses
$
20,486
$
22,894
Payroll and related expenses
43,008
41,324
Other
16,159
18,976
Total accrued liabilities
$
79,653
$
83,194
12
5.
Revenue
The following table disaggregates total revenues from customers (in thousands):
Three Months Ended
September 30,
Nine Months Ended September 30,
2020
2019
2020
2019
Collaboration and license revenue:
Crysvita collaboration revenue in profit-
share territory
$
34,058
$
19,534
$
91,079
$
48,721
Crysvita royalty revenue in European
territory
—
1,950
1,498
5,896
Daiichi Sankyo
32,866
—
51,723
—
Bayer
—
101
—
453
Total collaboration and license revenue
66,924
21,585
144,300
55,070
Product sales:
Crysvita
3,277
1,140
7,436
2,734
Mepsevii
4,076
2,361
11,686
8,274
Dojolvi
3,862
714
6,638
2,043
Total product sales
11,215
4,215
25,760
13,051
Crysvita non-cash collaboration royalty
revenue
3,331
—
9,428
—
Total revenues
$
81,470
$
25,800
$
179,488
$
68,121
The following table disaggregates total revenues based on geographic location (in thousands):
Three Months Ended
September 30,
Nine Months Ended September 30,
2020
2019
2020
2019
North America
$
72,651
$
21,700
$
154,939
$
56,318
Europe
5,002
2,790
15,707
8,522
All other
3,817
1,310
8,842
3,281
Total revenues
$
81,470
$
25,800
$
179,488
$
68,121
The following table presents changes in the contract assets (liabilities) (in thousands):
Nine Months Ended September 30,
2020
2019
Balance of contract assets at beginning of period
$
—
$
2,979
Additions
( 154,621
)
452
Deductions
51,723
( 3,447
)
Balance of contract liabilities at end of period, net
$
( 102,898
)
$
( 16
)
See Note 6 for additional details on contract assets (liabilities) activities.
The Company’s largest accounts receivable balance accounted for 83 % and 87 % of the total accounts receivable balance as of September 30, 2020 and December 31, 2019, respectively, and was due from a collaboration partner.
6.
License and Research Agreements
Kyowa Kirin Collaboration and License Agreement
In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd. (KKC or formerly Kyowa Hakko Kirin Co., Ltd. or KHK). Under the terms of this collaboration and license agreement, as amended, the Company and KKC collaborate on the development and commercialization of Crysvita in the field of orphan diseases in the United States and Canada, or the profit-share territory, and in the European Union, United Kingdom, and Switzerland, or the European territory, and the Company has the right to develop and commercialize such products in the field of orphan diseases in Mexico and Central and South America, or Latin America.
Development Activities
In the field of orphan diseases, and except for ongoing studies being conducted by KKC, the Company is the lead party for development activities in the profit-share territory and in the European territory until the applicable transition date; the Company is
13
also the lead party for core development activities conducted in Japan and Korea, for which the core development plan is limited to clinical trials mutually agreed to by the Company and KKC. The Company shares the costs for development activities in the profit-share territory and the European territory conducted pursuant to the development plan before the applicable transition date equally with KKC. KKC is responsible for 100 % of the costs for development activities in Japan and Korea. In April 2023, which is the transition date for the profit-share territory, and on the applicable transition date for the European territory, KKC will become the lead party and be responsible for the costs of the development activities. However, the Company will continue to share the costs of the studies commenced prior to the applicable transition date equally with KKC. Crysvita was approved in the European Union and United Kingdom in February 2018 and was approved by the FDA in April 2018.
The collaboration and license agreements are within the scope of ASC 808, which provides guidance on the presentation and disclosure of collaborative arrangements.
Collaboration revenue related to sales in profit-share territory
The Company and KKC share commercial responsibilities and profits in the profit-share territory until April 2023. Under the collaboration agreement, KKC manufactures and supplies Crysvita for commercial use in the profit-share territory and charges the Company the transfer price of 35 % of net sales through December 31, 2022, and 30 % thereafter. The remaining profit or loss after supply costs from commercializing products in the profit-share territory are shared between the Company and KKC on a 50 /50 basis until April 2023. Thereafter, the Company will be entitled to receive a tiered double-digit revenue share in the mid-to-high 20 % range.
As KKC is the principal in the sale transaction with the customer, the Company recognizes a pro-rata share of collaboration revenue, net of transfer pricing, in the period the sale occurs. The Company concluded that its portion of KKC’s sales in the profit-share territory is analogous to a royalty and therefore recorded its share as collaboration revenue, similar to a royalty.
Royalty revenue related to sales in European territory
KKC has the commercial responsibility for Crysvita in the European territory. In December 2019, the Company sold its right to receive royalty payments based on sales in the European territory to Royalty Pharma, effective January 1, 2020, as further described in Note 7. Prior to the Company’s sale of the royalty, the Company received a royalty of up to 10 % on net sales in the European territory, which was recognized as the underlying sales occur. Beginning in 2020, the Company records the royalty revenue as non-cash royalty revenues. During the nine months ended September 30, 2020, there was a change in estimate of the revenue reserves related to sales made prior to January 1, 2020, as a result of which, the Company recorded $ 1.5 million as royalty revenue in European territory.
The Company’s share of collaboration and royalty revenue related to Crysvita was as follows (in thousands):
Three Months Ended September 30,
Nine Months Ended September 30,
2020
2019
2020
2019
Company's share of revenue in profit-
share territory
$
34,058
$
19,534
$
91,079
$
48,721
Royalty revenue in European territory
—
1,950
1,498
5,896
Non-cash royalty revenue in European
territory
3,331
—
9,428
—
Total
$
37,389
$
21,484
$
102,005
$
54,617
Product revenue related to sales in other territories
The Company is responsible for commercializing Crysvita in Latin America and Turkey. The Company is considered the principal in these territories as the Company controls the product before it is transferred to the customer. Accordingly, the Company records revenue on a gross basis related to the sale of Crysvita once the product is delivered and the risk and title of the product is transferred to the distributor. The Company recorded product sales of $ 3.3 million and $ 7.4 million for the three and nine months ended September 30, 2020, respectively, and $ 1.1 million and $ 2.7 million for the three and nine months ended September 30, 2019, respectively, net of estimated product returns and other deductions. KKC has the option to assume responsibility for commercialization efforts in Turkey from the Company, after a certain minimum period.
Under the collaboration agreement, KKC manufactures and supplies Crysvita, which is purchased by the Company for sales in the above territories and is based on 35 % of the net sales through December 31, 2022 and 30 % thereafter. The Company also pays to KKC a low single-digit royalty on net sales in Latin America .
Cost sharing payments
Under the collaboration agreement, KKC and the Company share certain development and commercialization costs. As a result, the Company was reimbursed for these costs and operating expenses were reduced as follows (in thousands):
14
Three Months Ended September 30,
Nine Months Ended September 30,
2020
2019
2020
2019
Research and development
$
5,156
$
7,124
$
15,814
$
20,118
Selling, general and administrative
5,492
5,633
18,509
16,009
Total
$
10,648
$
12,757
$
34,323
$
36,127
Collaboration receivable
The Company had accounts receivable from KKC in the amount of $ 23.2 million and $ 28.5 million from profit-share revenue and royalties and other receivables recorded in prepaid and other current assets of $ 12.9 million and $ 17.8 million and accrued liabilities of $ 1.6 million and $ 0.9 million from commercial and development activity reimbursements, as of September 30, 2020 and December 31, 2019, respectively.
Bayer HealthCare LLC
The Company has an agreement with Bayer Healthcare LLC (Bayer) to research, develop and commercialize AAV gene therapy products for the treatment of hemophilia A (DTX 201). Under this agreement, Bayer has been granted an exclusive license to develop and commercialize one or more novel gene therapies for hemophilia A. The agreement requires that Bayer use commercially reasonable efforts to conduct and fund a proof-of-concept (POC) clinical trial and any subsequent clinical trials and commercialization of gene therapy products for treatment of hemophilia A. Bayer will have worldwide rights to commercialize the potential future product.
Bayer is responsible for funding certain research and development services performed by the Company in the performance of its obligations under the annual research plan and budget. Under the terms of the agreement with Bayer, the Company is eligible to receive development and commercialization milestone payments of up to $ 232.0 million, as well as, royalty payments ranging in the high single-digit to low double-digit percentages, not exceeding the mid-teens, of net sales of licensed products. The Company achieved the first milestone in December 2017, the second milestone in April 2018, and has received $ 15.0 million for such milestones to date.
The Company’s obligations under the contract were completed by end of December 31, 2019 and no revenue was recorded for the three and nine months ended September 30, 2020. The Company recorded revenue of $ 0.1 million and $ 0.5 million for the three and nine months ended September 30, 2019, respectively. The Company may record future milestone payments as revenue, if it becomes probable that a significant reversal in the amount of revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved.
Arcturus
The Company has a Research Collaboration and License Agreement with Arcturus to research and develop therapies for select rare diseases. Pursuant to the agreement, the Company incurred none and $ 0.7 million for the three and nine months ended September 30, 2019, respectively, in research and development expense for the funding of certain research services received from Arcturus. No research and development expenses were recognized in 2020 related to the Arcturus agreement.
In May 2020, the Company exercised an option to purchase 600,000 shares of Arcturus’ common stock at $ 16.00 per share, or a total purchase price of $ 9.6 million. Accordingly, after the exercise of the option, the Company owned 3,000,000 shares, or 14.6 %, of Arcturus’ then outstanding common stock. The purchase was made pursuant to the equity purchase agreement between the parties entered into in June 2019 in connection with the amendment to the research collaboration and license agreement. The Arcturus common stock is restricted from sale or transfer by the Company for six months from the exercise date, subject to certain conditions. The Company has elected to apply the fair value option to account for the equity investment in Arcturus. The Company had accounted for the option to purchase additional shares of Arcturus common stock at fair value based on the Black-Scholes option pricing method.
The changes in the fair value of the Company’s investment in Arcturus securities were as follows (in thousands):
Arcturus common stock
Fair value of option to purchase additional shares of Arcturus common stock
Total
Acquisition of investment in Arcturus securities
in June 2019
$
13,872
$
467
$
14,339
Change in fair value
12,216
1,197
13,413
December 31, 2019
26,088
1,664
27,752
Change in fair value
67,400
23,948
91,348
Transfer of value upon option exercise
35,212
( 25,612
)
9,600
September 30, 2020
$
128,700
$
—
$
128,700
15
The Company recorded $ 11.5 million in unrealized loss for the three months ended September 2020 from the fair value adjustment on the investment in Arcturus common stock.
GeneTx
In August 2019, the Company entered into a Program Agreement and a Unitholder Option Agreement with GeneTx Biotherapeutics, LLC (GeneTx) to collaborate on the development of GeneTx’s GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome.
Pursuant to the terms of the Unitholder Option Agreement, the Company made an upfront payment of $ 20.0 million for an exclusive option to acquire GeneTx, which was exercisable any time prior to 30 days following FDA acceptance of the IND for GTX-102. Pursuant to the agreement, upon acceptance of the IND, which occurred in January 2020, the Company elected to extend the option period by paying an option extension payment of $ 25.0 million (option extension premium) during the prior quarter ended March 31, 2020. The Company has a right to acquire GeneTx for a payment of $ 125.0 million, at any time, until the earlier of 30 months from the first dosing of a patient in a planned Phase 1/2 study (subject to extensions) or 90 days after results are available from that study. This exclusive option to acquire GeneTx can be extended under certain circumstances, by up to four additional three-month periods, by paying an additional extension fee for each three-month period.
During the exclusive option period, GeneTx is responsible for conducting the program based on the development plan agreed between the parties and, subject to the terms in the Program Agreement, has the decision-making authority on all matters in connection with the research, development, manufacturing and regulatory activities with respect to the Program. The Company will provide support, at its discretion, including strategic guidance and clinical expertise. The Company and GeneTx will collaborate on the management of the Phase 1/2 study in patients with Angelman syndrome. If the Company acquires GeneTx, the Company will then be responsible for all development and commercialization activities from the date of acquisition. The Company would also be required to make payments upon achievement of certain development and commercial milestones, as well as royalties, depending upon the success of the program.
Although GeneTx is a variable interest entity, the Company is not the primary beneficiary as it currently does not have the power to direct the activities that would most significantly impact the economic performance of GeneTx. Prior to product regulatory approval, all consideration paid to GeneTx represents rights to potential future benefits associated with GeneTx’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use. Accordingly, for the nine months ended September 30, 2020, the Company recorded the option extension payment of $ 25.0 million as an in-process research and development expense.
REGENXBIO, Inc.
In March 2020, the Company executed a License Agreement with REGENXBIO, Inc. (REGENEX), for an exclusive, sublicensable, worldwide license to REGENX’s NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder. In return for these rights, the Company made an upfront payment of $ 7.0 million, which was recorded as an in-process research and development expense during the prior quarter ended March 31, 2020. The Company will pay certain annual fees of $ 0.1 million, milestone payments of up to $ 14.0 million, and royalties on any net sales of products incorporating the licensed intellectual property that range from a high single-digit to low double-digit royalty .
Daiichi Sankyo
In March 2020, the Company executed a License and Technology Access Agreement (the License Agreement) with Daiichi Sankyo Co., Ltd. (Daiichi Sankyo). Pursuant to the License Agreement, the Company granted Daiichi Sankyo a non-exclusive license to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. The Company retains the exclusive right to use the manufacturing technology for its current target indications and additional indications identified now and in the future. The Company will provide certain technical assistance and technology transfer services during the technology transfer period of three years to enable Daiichi Sankyo to use the technologies for its internal gene therapy programs. Daiichi Sankyo has an option to extend the technology transfer period including know-how improvements by two additional one-year periods by paying a fixed amount for each additional year. Daiichi Sankyo will be responsible for the manufacturing, development, and commercialization of products manufactured with the licensed technology; however, the Company has the option to co-develop and co-commercialize rare disease products at the IND stage. Ultragenyx may also provide strategic consultation to Daiichi Sankyo on the development of both AAV-based gene therapy products and other products for rare diseases.
Under the terms of the License Agreement, Daiichi Sankyo made an upfront payment of $ 125.0 million and will pay an additional $ 25.0 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms, as well as single-digit royalties on net sales of products manufactured in either system. Daiichi Sankyo will reimburse the Company for all costs associated with the transfer of the manufacturing technology.
16
The Company also entered into a Stock Purchase Agreement (SPA) with Daiichi Sankyo, pursuant to which Daiichi Sankyo purchased 1,243,913 shares in exchange for $ 75.0 million in cash during the first quarter of 2020. The fair market value of the common stock issued to Daiichi Sankyo was $ 55.3 million based on the stock price of $ 44.43 per share on the date of issuance, resulting in a $ 19.7 million premium on the SPA. Daiichi Sankyo is also subject to a three-year standstill and restrictions on sale of the shares (subject to customary exceptions or release).
In June 2020, the Company executed a subsequent license agreement (the Sublicense Agreement) with Daiichi Sankyo for transfer of certain technology in consideration for an upfront payment of $ 8.0 million and annual maintenance fees, milestone payments, and royalties on any net sales of products incorporating the licensed intellectual property.
The License Agreement, the Sublicense Agreement, and the SPA are being accounted for as one arrangement because they were entered into at or near the same time and negotiated in contemplation of one another. The Company evaluated the License Agreement and the Sublicense Agreement under ASC 606 and determined that the performance obligations under the agreements are (i) intellectual property with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms together with the initial technical assistance and technology transfer services, which are expected to be completed over a period of 18 to 21 months, and (ii) the transfer of any know-how and improvements after the completion of the initial technology transfer through the end of the three year technology transfer period.
The Company determined that the total transaction price of the License Agreement was $ 183.6 million which was comprised of the $ 19.7 million premium from the SPA, the $ 125.0 million upfront payment, the $ 25.0 million in unconstrained milestone payments, $ 8.0 million from the Sublicense Agreement, and the $ 5.9 million estimated reimbursement amount for delivering the license and technology services.
The Company allocated the total transaction price to the two performance obligations on a relative stand-alone selling price basis. Revenue allocated to the intellectual property and the technology transfer services will be recognized over an initial estimated period of 18 to 21 months, measuring the progress toward complete satisfaction of the individual performance obligation using an input measure. Revenue for know-how and improvements after the completion of technology transfer will be recognized over the remaining technology transfer period (i.e., months 19-36) on a straight-line basis, as it is expected that Daiichi Sankyo will receive and consume the benefits consistently throughout the period. The performance obligations are estimated to be substantially complete by March 2023. The estimated period to complete the technology transfer services and the related milestones payments, if any, are subject to revised estimates which could be impacted by limitations or delays from the COVID-19 pandemic, successful scale-up of the manufacturing, and other changes that may impact timing. Royalties from commercial sales will be accounted for as revenue upon achievement of such sales, assuming all other revenue recognition criteria are met.
For the three and nine months ended September 30, 2020, the Company recognized $ 32.9 million and $ 51.7 million, respectively, in revenue related to this arrangement. Accordingly, the Company had recorded $ 102.9 million as contract liabilities, net, as of September 30, 2020. The Company recorded a receivable related to the above agreements of $ 0.9 million as of September 30, 2020 in prepaid expenses and other current assets.
7. Liability Related to the Sale of Future Royalties
In December 2019, the Company entered into a Royalty Purchase Agreement with RPI. Pursuant to the agreement, RPI paid $ 320.0 million to the Company in consideration for the right to receive royalty payments effective January 1, 2020, arising from the net sales of Crysvita in the European Union, the United Kingdom, and Switzerland under the terms of the Company’s Collaboration and License Agreement with KKC dated August 29, 2013, as amended. The agreement with RPI will automatically terminate, and the payment of royalties to RPI will cease, in the event aggregate royalty payments received by RPI are equal to or greater than $ 608.0 million prior to December 31, 2030, or in the event aggregate royalty payments received by RPI are less than $ 608.0 million prior to December 31, 2030, when aggregate royalty payments received by RPI are equal to $ 800.0 million.
As RPI’s rate of return is explicitly limited due to the cap on royalties they may receive, proceeds from the transaction were recorded as a liability (liability related to sale of future royalties on the Consolidated Balance Sheets). The Company amortizes $ 320.0 million, net of transaction cost of $ 5.8 million using the effective interest method over the estimated life of the arrangement. In order to determine the amortization of the liability, the Company is required to estimate the total amount of future royalty payments to be received by the Company and paid to RPI, subject to the capped amount, over the life of the arrangement. The excess of future estimated royalty payments (subject to the capped amount), over the $ 314.2 million of net proceeds, is recorded as non-cash interest expense over the life of the arrangement. Consequently, the Company estimates an imputed interest on the unamortized portion of the liability and records interest expense relating to the transaction. The Company records the royalty revenue from the net sales of Crysvita in the applicable European territories as non-cash royalty revenue in the Consolidated Statements of Operations.
The Company periodically assesses the expected royalty payments using a combination of historical results, internal projections and forecasts from external sources. To the extent such payments are greater or less than the Company’s initial estimates or the timing of such payments is materially different than its original estimates, the Company will prospectively adjust the amortization of the liability and the effective interest rate. The Company’s effective annual interest rate was approximately 10.3 % and 10.1 % as of September 30, 2020 and December 31, 2019, respectively.
There are a number of factors that could materially affect the amount and timing of royalty payments from KKC in the applicable European territories, most of which are not within the Company’s control. Such factors include, but are not limited to, the
17
success of KKC’s sales and promotion of Crysvita, changing standards of care, delays or disruptions related to the COVID-19 pandemic, the introduction of competing products, pricing for reimbursement in various European territories, manufacturing or other delays, intellectual property matters, adverse events that result in governmental health authority imposed restrictions on the use of Crysvita, significant changes in foreign exchange rates as the royalty payments are made in U.S. dollars (USD) while significant portions of the underlying European sales of Crysvita are made in currencies other than USD, and other events or circumstances that could result in reduced royalty payments from European sales of Crysvita, all of which would result in a reduction of non-cash royalty revenue and the non-cash interest expense over the life of the arrangement. Conversely, if sales of Crysvita in Europe are more than expected, the non-cash royalty revenue and the non-cash interest expense recorded by the Company would be greater over the term of the arrangement.
The following table shows the activity within the liability account (in thousands):
Liability related to the sale of future royalties
Proceeds from sale of future royalties in
December 2019
$
314,234
Non-cash interest expense
1,135
December 31, 2019
315,369
Non-cash collaboration royalty revenue
( 9,428
)
Non-cash interest expense
25,093
September 30, 2020
$
331,034
8 .
Stock-Based Awards
The 2014 Incentive Plan (the 2014 Plan) provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024 . As of September 30, 2020 , there were 3,186,920 shares reserved under the 2014 Plan for the future issuance of equity awards and 3,287,825 shares reserved for the 2014 Employee Stock Purchase Plan.
The table below sets forth the stock-based compensation expense for the periods presented (in thousands):
Three Months Ended
September 30,
Nine Months Ended
September 30,
2020
2019
2020
2019
Cost of sales
$
133
$
319
$
313
$
404
Research and development
11,533
10,629
35,318
33,891
Selling, general and administrative
8,675
8,959
27,291
28,040
Total stock-based compensation expense
$
20,341
$
19,907
$
62,922
$
62,335
9 .
Net Loss Per Share
Basic net loss per share has been computed by dividing the net loss by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of shares of common stock and potential dilutive securities outstanding during the period.
The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive:
Three Months Ended
September 30,
Nine Months Ended
September 30,
2020
2019
2020
2019
Options to purchase common stock and
restricted stock units
8,627,419
8,155,406
8,637,904
7,936,664
Employee stock purchase plan
35,282
33,094
19,701
18,547
Common stock warrants
—
149,700
39,337
149,700
8,662,701
8,338,200
8,696,942
8,104,911
10 .
Equity Transactions
In July 2017, the Company entered into an At-The-Market, or ATM, sales agreement with Cowen and Company, LLC (Cowen), whereby the Company could sell up to $ 150.0 million in aggregate proceeds of common stock from time to time, with Cowen as its sales agent. During the three months ended June 30, 2020, the Company sold 283,333 shares of common stock, resulting in net proceeds of $ 20.4 million, after commissions and other offering costs. As of June 30, 2020, the Company had completed the sale of all
18
available amounts under the ATM facility. During the three and nine months ended September 30, 2019, the Company sold none and 468,685 shares of common stock, respectively, resulting in net proceeds of approximately none and $ 24.8 million, respectively, after commissions and other offering costs.
In February 2019, the Company completed an underwritten public offering in which 5,833,333 shares of common stock were sold, which included 760,869 shares purchased by the underwriters pursuant to an option granted to them in connection with the offering, at a public offering price of $ 60.00 per share. The total proceeds that the Company received from the offering were approximately $ 330.4 million, net of underwriting discounts and commissions.
11 .
Accumulated Other Comprehensive Income (Loss)
Total accumulated other comprehensive income (loss) consisted of the following (in thousands):
September 30,
December 31,
2020
2019
Foreign currency translation adjustments
$
108
$
( 306
)
Unrealized gain on available-for-sale securities
905
159
Total accumulated other comprehensive income
(loss)
$
1,013
$
( 147
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12. Subsequent Event
On October 23, 2020, the Company announced that it had entered into a strategic collaboration and license agreement (the collaboration agreement) with Solid Biosciences, Inc. (Solid) to focus on the development and commercialization of new gene therapies for Duchenne muscular dystrophy (Duchenne) using its HeLa producer cell line (PCL) manufacturing platform. Under the terms of the collaboration agreement, Solid granted the Company an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin. The Company also entered into a stock purchase agreement and investor agreement with Solid in connection with the collaboration agreement, and purchased 7,825,797 shares of Solid’s common stock for an aggregate purchase price of $ 40.0 million. The Company will pay Solid up to $ 255.0 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double-digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.
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