0000950170-23-018017 | 10-Q | XBRL Viewer | Ultragenyx Pharmaceutical INC

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CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 85,768	$ 132,944
Marketable debt securities	543,586	614,818
Accounts receivable, net	42,991	40,445
Inventory	26,616	26,766
Prepaid expenses and other current assets	67,375	68,926
Total current assets	766,336	883,899
Property, plant, and equipment, net	277,873	259,726
Equity investments	5,197	5,531
Marketable debt securities	85,213	148,970
Right-of-use assets	22,949	25,961
Intangible assets, net	159,171	160,105
Goodwill	44,406	44,406
Other assets	22,259	16,846
Total assets	1,383,404	1,545,444
Current liabilities:
Accounts payable	58,981	43,274
Accrued liabilities	144,404	204,678
Contract liabilities	0	1,479
Lease liabilities	11,261	11,779
Liabilities for sales of future royalties	3,533	0
Total current liabilities	218,179	261,210
Lease liabilities	17,340	19,814
Deferred tax liabilities	31,667	31,667
Liabilities for sales of future royalties	886,480	875,439
Other liabilities	9,257	4,820
Total liabilities	1,162,923	1,192,950
Stockholders’ equity:
Preferred stock - 25,000,000 shares authorized; nil outstanding as of March 31, 2023 and December 31, 2022	0	0
Common stock - 250,000,000 shares authorized; 70,660,249 and 70,197,297 shares issued and outstanding as of March 31, 2023 and December 31, 2022, respectively	71	70
Treasury stock, at cost, 7,812 and nil shares held as of March 31, 2023 and December 31, 2022, respectively	(356)	0
Deferred compensation obligation	356	0
Additional paid-in capital	3,169,371	3,140,019
Accumulated other comprehensive loss	(3,967)	(6,573)
Accumulated deficit	(2,944,994)	(2,781,022)
Total stockholders’ equity	220,481	352,494
Total liabilities and stockholders’ equity	$ 1,383,404	$ 1,545,444

Net Loss Per Share (Tables)

3 Months Ended

Mar. 31, 2023

Earnings Per Share [Abstract]

Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share

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 March 31,
	2023		2022
Options to purchase common stock, restricted stock units, and performance stock units		12,825,527		9,363,681
Employee stock purchase plan		97,917		23,276
		12,923,444		9,386,957

Balance Sheet Components - Accrued Liabilities (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Payables and Accruals [Abstract]
Research, clinical study, and manufacturing expenses	$ 42,725	$ 73,558
Payroll and related expenses	55,796	78,938
Other	45,883	52,182
Total accrued liabilities	$ 144,404	$ 204,678

License and Research Agreements - Share of Collaboration and Royalty Revenue Related to Crysvita (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	$ 100,496	$ 79,935
Product Revenue
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	44,229	26,684
Product Revenue \| Kyowa Kirin Collaboration
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	21,234	9,394
Collaboration and Royalty \| Kyowa Kirin Collaboration
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	76,022	59,396
Collaboration and Royalty \| Kyowa Kirin Collaboration \| Profit Share Territory
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	49,906	45,164
Non-cash Collaboration Royalty Revenue
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	4,882	4,838
Non-cash Collaboration Royalty Revenue \| Kyowa Kirin Collaboration \| Royalty Revenue in European Territory
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	$ 4,882	$ 4,838

- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

+ References

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+ Details

License and Research Agreements - Additional Information (Details) - USD ($)		1 Months Ended				3 Months Ended		113 Months Ended
	Jul. 01, 2022	Apr. 30, 2023	May 31, 2022	Jan. 31, 2022	Jan. 31, 2021	Mar. 31, 2023	Mar. 31, 2022	Dec. 31, 2022
Daiichi Sankyo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Additional upfront payments received						$ 0		$ 1,500,000
Revenue recognized						$ 1,500,000	$ 3,200,000
Regeneron Pharmaceuticals, Inc.
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Upfront payments of collaboration				$ 30,000,000.0
Intangible assets, remaining amortization period						10 years 6 months
Eligible future milestone payments				$ 63,000.0
Cost of Goods and Services Sold						$ 800,000	700,000
Costs of goods sold related to amortization of intangible asset						700,000	700,000
Development costs						3,200,000	$ 600,000
Regeneron Pharmaceuticals, Inc. \| Maximum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Profit loss sharing percentage on net sales				40.00%
Regeneron Pharmaceuticals, Inc. \| Minimum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Profit loss sharing percentage on net sales				20.00%
Abeona \| Maximum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Eligible future milestone payments						$ 1,000,000.0
License Agreement \| Profit Share Territory \| Minimum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Tiered double-digit revenue share percentage entitled to receive		20.00%
License and Collaboration Agreement \| Mereo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Payments made under agreement					$ 50,000,000.0
License and Collaboration Agreement \| Maximum \| Mereo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Future contingent milestone payments					$ 254,000,000.0
License and Collaboration Agreement \| Abeona
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Percentage of royalty on net sales receives						10.00%
Commercial milestone payments			$ 30,000,000.0
Kyowa Kirin Collaboration \| License Agreement
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Profit loss sharing percentage on net sales						30.00%		35.00%
Kyowa Kirin Collaboration \| License Agreement \| Maximum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Tiered double-digit revenue share percentage entitled to receive		30.00%
Kyowa Kirin Collaboration \| License Agreement \| Prepaid Expenses and Other Current Assets
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
License agreement other receivables						$ 12,700,000		$ 6,400,000
Kyowa Kirin Collaboration \| License Agreement \| Profit Share Revenue
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
License agreement accounts receivable						17,400,000		27,500,000
Kyowa Kirin Collaboration \| License Agreement \| Commercial and Development Activity Reimbursements
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
License agreement accrued liabilities						$ 6,900,000		$ 3,100
Kyowa Kirin Collaboration \| License Agreement \| Profit Share Territory
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Remaining profit or loss share percentage on commercializing products						50.00%
Percentage of right to receive royalty payments on net sales	30.00%
Kyowa Kirin Collaboration \| License Agreement \| Royalty Revenue in European Territory
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Percentage of royalty on net sales receives						10.00%
License and Technology Access Agreement \| Daiichi Sankyo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Upfront payment received						$ 125,000,000.0
Additional upfront payments received						25,000,000.0
License and Technology Access Agreement \| License Agreement \| Daiichi Sankyo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Revenue recognized						$ 183,300,000

Document and Entity Information - shares	3 Months Ended
Document and Entity Information - shares	Mar. 31, 2023	May 01, 2023
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Mar. 31, 2023
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q1
Trading Symbol	RARE
Entity Registrant Name	ULTRAGENYX PHARMACEUTICAL INC.
Entity Central Index Key	0001515673
Entity Current Reporting Status	Yes
Current Fiscal Year End Date	--12-31
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Common Stock, Shares Outstanding		70,814,462
Entity Shell Company	false
Entity File Number	001-36276
Entity Tax Identification Number	27-2546083
Entity Address, Address Line One	60 Leveroni Court
Entity Address, City or Town	Novato
Entity Address, State or Province	CA
Entity Address, Postal Zip Code	94949
City Area Code	415
Local Phone Number	483-8800
Entity Interactive Data Current	Yes
Title of 12(b) Security	Common Stock, $0.001 par value
Security Exchange Name	NASDAQ
Entity Incorporation, State or Country Code	DE
Document Quarterly Report	true
Document Transition Report	false

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Revenues:
Total revenues	$ 100,496	$ 79,935
Operating expenses:
Cost of sales	12,257	6,100
Research and development	165,698	143,155
Selling, general and administrative	76,646	67,312
Total operating expenses	254,601	216,567
Loss from operations	(154,105)	(136,632)
Interest income	6,290	494
Change in fair value of equity investments	(334)	(9,329)
Non-cash interest expense on liability for sale of future royalties	(15,636)	(6,584)
Other income	308	289
Loss before income taxes	(163,477)	(151,762)
Provision for income taxes	(495)	(558)
Net loss	$ (163,972)	$ (152,320)
Net loss per share, basic	$ (2.33)	$ (2.19)
Net loss per share, diluted	$ (2.33)	$ (2.19)
Weighted-average shares used in computing net loss per share, basic	70,368,478	69,516,668
Weighted-average shares used in computing net loss per share, diluted	70,368,478	69,516,668
Collaboration and License
Revenues:
Total revenues	$ 51,385	$ 48,413
Product Sales
Revenues:
Total revenues	44,229	26,684
Royalty Revenue
Revenues:
Total revenues	$ 4,882	$ 4,838

Accumulated Other Comprehensive Loss (Tables)

3 Months Ended

Mar. 31, 2023

Other Comprehensive Income (Loss), Net of Tax [Abstract]

Schedule of Total Accumulated Other Comprehensive Loss

Total accumulated other comprehensive loss consisted of the following (in thousands):


	March 31,			December 31,
	2023			2022
Foreign currency translation adjustments	$	(701	)	$	(845	)
Unrealized loss on available-for-sale securities		(3,266	)		(5,728	)
Total accumulated other comprehensive loss	$	(3,967	)	$	(6,573	)

Equity Transactions - Additional Information (Details) - USD ($) $ in Millions	1 Months Ended	3 Months Ended
	May 31, 2021	Mar. 31, 2023
Stockholders Equity [Line Items]
Stock sold in at-the-market offering		1,050,372
Net proceeds from at-the-market offering		$ 78.9
Open Market Sale
Stockholders Equity [Line Items]
Common stock shares sold		0
Option to sell common stock aggregate offering price	$ 350.0

Revenue - Summary of Changes in Contract Liabilities (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Disaggregation of Revenue [Abstract]
Balance of contract liabilities at beginning of period	$ 1,479	$ 9,076
Additions	0	40
Deductions	(1,479)	(3,249)
Balance of contract liabilities at end of period, net	$ 0	$ 5,867

Liabilities for Sales of Future Royalties

3 Months Ended

Mar. 31, 2023

Deferred Revenue Disclosure [Abstract]

Liabilities for Sales of Future Royalties

8. Liabilities for Sales 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 EU, the U.K., and Switzerland under the terms of the Company’s Collaboration and License Agreement

with KKC dated August 29, 2013, as amended, or the KKC Collaboration Agreement. 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, or when aggregate royalty payments received by RPI are equal to $800.0 million.

In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS. Pursuant to the agreement, OMERS paid $500.0 million to the Company in consideration for the right to receive 30% of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $725.0 million or the date the final royalty payment is made to the Company under the KKC Collaboration Agreement.

Proceeds from these transactions were recorded as liabilities for sales of future royalties on the Condensed Consolidated Balance Sheets. Upon inception of the respective arrangements, the Company recorded $320.0 million and $500.0 million, net of transaction costs of $5.8 million and $9.1 million for RPI and OMERS, respectively, using the effective interest method over the estimated life of the applicable arrangement. In order to determine the amortization of the liabilities, the Company is required to estimate the total amount of future royalty payments to be received by the Company and paid to RPI and OMERS, subject to the capped amount, over the life of the arrangements. The excess of future estimated royalty payments (subject to the capped amount), over the $314.2 million and $491.0 million, respectively, of net proceeds, is recorded as non-cash interest expense over the life of the arrangements. Consequently, the Company estimates an imputed interest on the unamortized portion of the liabilities and records interest expense relating to the transactions. The Company records the royalty revenue arising from the net sales of Crysvita in the applicable territories as non-cash royalty revenue in the Consolidated Statements of Operations over the term of the arrangements. Royalties earned under the RPI and OMERS arrangements from inception to March 31, 2023 have been $57.5 million and nil, respectively.

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 liabilities and the effective interest rate. The Company’s effective annual interest rate was approximately 9.3% and 8.4%, for RPI and OMERS, respectively, as of March 31, 2023.

There are a number of factors that could materially affect the amount and timing of royalty payments from KKC in the applicable territories, most of which are not within the Company’s control. Such factors include, but are not limited to, the success of KKC’s sales and promotion of Crysvita, changing standards of care, macroeconomic and inflationary pressures, the introduction of competing products, pricing for reimbursement in various 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, or USD, while significant portions of the underlying sales of Crysvita are made in currencies other than USD, and other events or circumstances that could result in reduced royalty payments from 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 the relevant territories 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 arrangements.

The following table shows the activity within the liability account (in thousands):


	Liabilities for Sales of Future Royalties
	RPI			OMERS		Total
December 31, 2021	$	351,786		$	—	$	351,786
Net proceeds from sale of future royalties		—			490,950		490,950
Royalty revenue		(21,692	)		—		(21,692	)
Non-cash interest expense		35,095			19,300		54,395
December 31, 2022		365,189			510,250		875,439
Royalty revenue		(4,882	)		—		(4,882	)
Non-cash interest expense		8,726			10,730		19,456
March 31, 2023	$	369,033		$	520,980	$	890,013

Revenue (Tables)

3 Months Ended

Mar. 31, 2023

Revenue from Contract with Customer [Abstract]

Summary of Disaggregation of Total Revenues

The following table disaggregates total revenues from external customers by collaboration and license revenue and product sales (in thousands):


	Three Months Ended
	March 31,
	2023		2022
Collaboration and license revenue:
Crysvita collaboration revenue in profit- share territory	$	49,906	$	45,164
Daiichi Sankyo		1,479		3,249
Total collaboration and license revenue		51,385		48,413
Product sales:
Crysvita		21,234		9,394
Mepsevii		8,480		4,861
Dojolvi		14,303		12,429
Evkeeza		212		—
Total product sales		44,229		26,684
Crysvita royalty revenue		4,882		4,838
Total revenues	$	100,496	$	79,935

The following table disaggregates total revenues based on geographic location (in thousands):


	Three Months Ended
	March 31,
	2023		2022
North America	$	67,132	$	61,917
Latin America		22,799		9,791
Europe		9,387		8,227
Japan		1,178		—
Total revenues	$	100,496	$	79,935

Summary of Changes in Contract Liabilities


	Three Months Ended March 31,
	2023			2022
Balance of contract liabilities at beginning of period	$	1,479		$	9,076
Additions		—			40
Deductions		(1,479	)		(3,249	)
Balance of contract liabilities at end of period, net	$	—		$	5,867

Balance Sheet Components (Tables)

3 Months Ended

Mar. 31, 2023

Investments, Debt and Equity Securities [Abstract]

Summary of Cash Equivalents and Investments Classified as Available For Sale Securities

The fair values of cash equivalents and investments classified as available-for-sale securities consisted of the following (in thousands):


	March 31, 2023
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	51,187	$	—	$	—		$	51,187
Certificates of deposit and time deposits			13,848		—		—			13,848
Corporate bonds			315,735		140		(3,221	)		312,654
Commercial paper			161,958		—		—			161,958
Asset-backed securities			11,774		—		(56	)		11,718
U.S. Government Treasury and agency securities			128,749		583		(685	)		128,647
Debt securities in government-sponsored entities			10,000		—		(27	)		9,973
Total		$	693,251	$	723	$	(3,989	)	$	689,985


	December 31, 2022
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	102,847	$	—	$	—		$	102,847
Certificates of deposit and time deposits			25,972		—		—			25,972
Corporate bonds			432,211		87		(4,700	)		427,598
Commercial paper			135,393		—		—			135,393
Asset-backed securities			12,002		—		(22	)		11,980
U.S. Government Treasury and agency securities			157,933		320		(1,263	)		156,990
Debt securities in government-sponsored entities			16,005		—		(150	)		15,855
Total		$	882,363	$	407	$	(6,135	)	$	876,635

Summary of Inventory

Inventory consists of the following (in thousands):


	March 31,		December 31,
	2023		2022
Work-in-process	$	15,091	$	17,486
Finished goods		11,525		9,280
Total inventory	$	26,616	$	26,766

Accrued Liabilities

Accrued liabilities consist of the following (in thousands):


	March 31,		December 31,
	2023		2022
Research, clinical study, and manufacturing expenses	$	42,725	$	73,558
Payroll and related expenses		55,796		78,938
Other		45,883		52,182
Total accrued liabilities	$	144,404	$	204,678

GeneTx Acquisition - Additional Information (Details) - USD ($) $ in Millions	1 Months Ended			12 Months Ended
	Jul. 31, 2022	Apr. 30, 2022	Aug. 31, 2019	Dec. 31, 2022
GeneTx [Member]
Business Acquisition [Line Items]
Business acquisition, consideration transferred	$ 75.0
Outstanding Cash and Cash Equivalents Acquired	15.6
Amount on adjustments for working capital and transaction expenses	0.6
Payments to Acquire Businesses, Gross	91.2
Regulatory Approval Milestones	85.0
GeneTx [Member] \| Maximum
Business Acquisition [Line Items]
Future contingent milestone payments	190.0
Additional milestone payments	30.0
Commercial milestone payment	$ 75.0
Program Agreement And Unitholder Option Agreement [Member] \| Gene Tx Biotherapeutics L L C [Member]
Business Acquisition [Line Items]
Business acquisition option exercise period, description			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.
Upfront payment for exclusive option to acquisition			$ 20.0
Option extension payment			$ 25.0
Acquisition cost		$ 75.0
In Process Research and Development [Member] \| GeneTx [Member]
Business Acquisition [Line Items]
Acquired in-process research and development				$ 75.0

Net Loss Per Share

3 Months Ended

Mar. 31, 2023

Earnings Per Share [Abstract]

Net Loss Per Share

10. 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 and the weighted average treasury stock for deferred compensation obligations required to be settled in shares of common stock. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of shares of common stock, the weighted average treasury stock for deferred compensation obligations required to be settled in shares of common stock, and potential dilutive securities outstanding during the period.


	Three Months Ended March 31,
	2023		2022
Options to purchase common stock, restricted stock units, and performance stock units		12,825,527		9,363,681
Employee stock purchase plan		97,917		23,276
		12,923,444		9,386,957

Stock-Based Awards

3 Months Ended

Mar. 31, 2023

Share-Based Payment Arrangement [Abstract]

Stock-Based Awards

9. Stock-Based Awards

The 2014 Incentive Plan, or the 2014 Plan, provides for automatic annual increases in shares available for grant, beginning on January 1, 2015 through January 1, 2024. As of March 31, 2023, there were 861,763 shares reserved under the 2014 Plan for the

future issuance of equity awards, 5,405,257 shares reserved for under the 2014 Employee Stock Purchase Plan, and 247,369 shares reserved for under the Employment Inducement Plan.

The table below sets forth the stock-based compensation expense for the periods presented (in thousands):


	Three Months Ended March 31,
	2023		2022
Cost of sales	$	346	$	175
Research and development		18,120		16,907
Selling, general and administrative		13,473		12,305
Total stock-based compensation expense	$	31,939	$	29,387

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Operating activities:
Net loss	$ (163,972)	$ (152,320)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation	31,952	29,396
Amortization of (discount) premium on marketable debt securities, net	(2,994)	1,894
Depreciation and amortization	5,236	4,088
Change in fair value of equity investments	334	9,329
Non-cash royalty revenue	(4,882)	(4,838)
Non-cash interest expense on liabilities for sales of future royalties	15,636	6,584
Other	187	35
Changes in operating assets and liabilities:
Accounts receivable	(2,508)	33
Inventory	150	(1,475)
Prepaid expenses and other assets	(519)	9,126
Accounts payable, accrued, and other liabilities	(34,008)	(16,164)
Contract liabilities	(1,479)	(3,209)
Net cash used in operating activities	(156,867)	(117,521)
Investing activities:
Purchase of property, plant, and equipment	(25,034)	(32,184)
Purchase of marketable debt securities	(62,024)	(203,710)
Proceeds from sale of marketable debt securities	10,715	39,422
Proceeds from maturities of marketable debt securities	191,598	188,990
Payment for intangible asset	0	(30,000)
Other	(3,951)	120
Net cash provided by (used in) investing activities	111,304	(37,362)
Financing activities:
Proceeds from the issuance of common stock under equity plan awards, net of tax	750	(1,755)
Other	28	(143)
Net cash (used in) provided by financing activities	(722)	1,612
Effect of exchange rate changes on cash	211	(108)
Net decrease in cash, cash equivalents and restricted cash	(46,074)	(153,379)
Cash, cash equivalents and restricted cash at beginning of period	137,601	309,585
Cash, cash equivalents and restricted cash at end of period	91,527	156,206
Supplemental disclosures of non-cash information:
Acquired lease liabilities arising from obtaining right-of-use assets	0	132
Stock-based compensation capitalized into ending inventory	2,276	1,581
Costs of PP&E included in AP, accrued, and other liabilities	9,137	24,782
Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment	$ 3,820	$ 2,101

Organization - Additional Information (Details)	3 Months Ended
Organization - Additional Information (Details)	Mar. 31, 2023 Segment
Organization And Nature Of Business [Line Items]
Number of reportable segments	1

Balance Sheet Components - Summary of Cash Equivalents and Investments Classified as Available For Sale Securities (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	$ 693,251	$ 882,363
Gross Unrealized Gains	723	407
Gross Unrealized Losses	(3,989)	(6,135)
Estimated Fair Value	689,985	876,635
Money Market Funds
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	51,187	102,847
Estimated Fair Value	51,187	102,847
Asset-backed Securities
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	11,774	12,002
Gross Unrealized Gains		0
Gross Unrealized Losses	(56)	(22)
Estimated Fair Value	11,718	11,980
Certificate of Deposits and Time Deposits
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	13,848	25,972
Estimated Fair Value	13,848	25,972
Corporate Bonds
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	315,735	432,211
Gross Unrealized Gains	140	87
Gross Unrealized Losses	(3,221)	(4,700)
Estimated Fair Value	312,654	427,598
Commercial Paper
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	161,958	135,393
Gross Unrealized Losses	0
Estimated Fair Value	161,958	135,393
U.S. Government Treasury and Agency Securities
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	128,749	157,933
Gross Unrealized Gains	583	320
Gross Unrealized Losses	(685)	(1,263)
Estimated Fair Value	128,647	156,990
Debt Securities in Government Sponsored Entities
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	10,000	16,005
Gross Unrealized Losses	(27)	(150)
Estimated Fair Value	$ 9,973	$ 15,855

Balance Sheet Components - Summary of Inventory (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Inventory, Net [Abstract]
Work-in-process	$ 15,091	$ 17,486
Finished goods	11,525	9,280
Total inventory	$ 26,616	$ 26,766

Financial Instruments

3 Months Ended

Mar. 31, 2023

Fair Value Disclosures [Abstract]

Financial Instruments

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.

The Company determines the fair value of its equity investment in Solid Biosciences Inc., or Solid, by using the quoted market prices, which are Level 1 fair value measurements.

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):


	March 31, 2023
	Level 1		Level 2		Level 3		Total
Money market funds	$	51,187	$	—	$	—	$	51,187
Certificate of deposits and time deposits		—		13,848		—		13,848
Corporate bonds		—		312,654		—		312,654
Commercial paper		—		161,958		—		161,958
Asset-backed securities		—		11,718		—		11,718
U.S. Government Treasury and agency securities		15,706		112,941		—		128,647
Debt securities in government-sponsored entities		—		9,973		—		9,973
Investment in Solid common stock		2,473		—		—		2,473
Other		—		8,546		—		8,546
Total	$	69,366	$	631,638	$	—	$	701,004


	December 31, 2022
	Level 1		Level 2		Level 3		Total
Money market funds	$	102,847	$	—	$	—	$	102,847
Certificates of deposits and time deposits		—		25,972		—		25,972
Corporate bonds		—		427,598		—		427,598
Commercial paper		—		135,393		—		135,393
Asset-backed securities		—		11,980		—		11,980
U.S. Government Treasury and agency securities		27,645		129,345		—		156,990
Debt securities in government-sponsored entities		—		15,855		—		15,855
Investment in Solid common stock		2,807		—		—		2,807
Other		—		4,575		—		4,575
Total	$	133,299	$	750,718	$	—	$	884,017

Stock-Based Awards - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	$ 31,939	$ 29,387
Cost of sales
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	346	175
Research and development
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	18,120	16,907
Selling, general and administrative
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	$ 13,473	$ 12,305

Liabilities for Sales of Future Royalties - Additional Information (Details) - USD ($) $ in Thousands	1 Months Ended		3 Months Ended		12 Months Ended
	Jul. 31, 2022	Dec. 31, 2019	Mar. 31, 2023	Mar. 31, 2022	Dec. 31, 2022
Liability For Sale Of Future Royalties [Line Items]
Net proceeds from sale of future royalties					$ 490,950
RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Net proceeds from sale of future royalties					0
OMERS
Liability For Sale Of Future Royalties [Line Items]
Net proceeds from sale of future royalties					$ 490,950
Royalty Purchase Agreement
Liability For Sale Of Future Royalties [Line Items]
Profit loss sharing percentage on net sales	30.00%
Royalty Purchase Agreement \| RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Non-cash collaboration royalty revenue			$ 57,500
Proceeds from sale of future royalties		$ 320,000
Royalty payment termination description			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, or when aggregate royalty payments received by RPI are equal to $800.0 million.
Net proceeds from sale of future royalties		314,200
Effective annual interest rate			9.30%
Royalties transaction costs net		5,800
Royalty Purchase Agreement \| OMERS
Liability For Sale Of Future Royalties [Line Items]
Non-cash collaboration royalty revenue			$ 0
Proceeds from sale of future royalties			500,000
Royalty payment termination description	In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS. Pursuant to the agreement, OMERS paid $500.0 million to the Company in consideration for the right to receive 30% of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $725.0 million or the date the final royalty payment is made to the Company under the KKC Collaboration Agreement.
Royalty agreement termination threshold amount	$ 725,000
Payment from sale of future royalties	$ 500,000
Net proceeds from sale of future royalties			$ 491,000
Effective annual interest rate			8.40%
Royalties transaction costs net				$ 9,100
Royalty Purchase Agreement \| Minimum \| RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Royalty agreement termination threshold amount		$ 608,000	$ 608,000
Royalty Purchase Agreement \| Maximum \| RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Royalty agreement termination threshold amount			$ 800,000

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Statement of Comprehensive Income [Abstract]
Net loss	$ (163,972)	$ (152,320)
Other comprehensive income (loss):
Foreign currency translation adjustments	144	(60)
Unrealized gain (loss) on available-for-sale securities	2,462	(5,090)
Other comprehensive loss:	2,606	(5,150)
Total comprehensive loss	$ (161,366)	$ (157,470)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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Amount after tax and reclassification adjustments of other comprehensive income (loss).

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+ Details

Summary of Significant Accounting Policies (Tables)

3 Months Ended

Mar. 31, 2023

Accounting Policies [Abstract]

Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands):


	March 31,
	2023		2022
Cash and cash equivalents	$	85,768	$	153,988
Restricted cash included in prepaid expenses and other current assets		1,964		217
Restricted cash included in other assets		3,795		2,001
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows	$	91,527	$	156,206

GeneTx Acquisition

3 Months Ended

Mar. 31, 2023

Business Combinations [Abstract]

GeneTx Acquisition

6. GeneTx Acquisition

In August 2019, the Company entered into a Program Agreement and a Unitholder Option Agreement with GeneTx Biotherapeutics LLC, or GeneTx, to collaborate on the development of GeneTx’s GTX-102, an 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 during the quarter ended March 31, 2020, which was recorded as an in-process research and development expense. In April 2022, the parties entered into an amendment to the

Unitholder Option Agreement, or the Amendment, which provided the Company with an additional, earlier option to acquire GeneTx for an option exercise price of $75.0 million based on the earlier of receipt of interim data in the Phase 1/2 study or a specified date, such option, the Interim Option.

In July 2022, the Company exercised the Interim Option to acquire GeneTx and entered into a Unit Purchase Agreement, or the Purchase Agreement, pursuant to which the Company purchased all the outstanding units of GeneTx. In accordance with the terms of the Purchase Agreement, the Company paid the option exercise price of $75.0 million and an additional $15.6 million to acquire the outstanding cash of GeneTx, and adjustments for working capital and transaction expenses of $0.6 million, for a total purchase consideration of $91.2 million. Additionally, the Company may make payments of up to $190.0 million upon the achievement of certain milestones, including up to $30.0 million in milestone payments upon achievement of the earlier of initiation of a Phase 3 clinical study or product approvals in Canada and the U.K., up to $85.0 million in additional regulatory approval milestones for the achievement of U.S. and EU product approvals, and up to $75.0 million in commercial milestone payments based on annual worldwide net product sales. In addition, the Company will also pay tiered mid- to high single-digit percentage royalties based on licensed product annual net sales. If the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, GeneTx is entitled to receive a portion of proceeds from the sale or a cash payment from the Company if the Company choses to retain the PRV.

The transaction was accounted as an asset acquisition, as substantially all of the fair value of the gross assets acquired was concentrated in a single identifiable in-process research and development intangible asset. Prior to the achievement of certain development and regulatory milestones, the acquired in-process research and development intangible asset has not yet reached technological feasibility and has no alternative future use. Accordingly, the Company recorded the acquisition price of $75.0 million, net of cash and working capital acquired, as in-process research and development expense during the year ended December 31, 2022.

License and Research Agreements (Tables)

3 Months Ended

Mar. 31, 2023

Share of Collaboration and Royalty Revenue Related to Crysvita

Total Crysvita revenue was as follows (in thousands):


	Three Months Ended March 31,
	2023		2022
Revenue in profit-share territory	$	49,906	$	45,164
Product revenue		21,234		9,394
Non-cash royalty revenue in European territory		4,882		4,838
Total Crysvita revenue	$	76,022	$	59,396

Schedule of 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):


	Three Months Ended March 31,
	2023		2022
Research and development	$	2,272	$	4,619
Selling, general and administrative		9,080		9,196
Total	$	11,352	$	13,815

Solid and Arcturus

Schedule of Changes in Fair Value of Equity Investment

The changes in the fair value of the Company’s equity investments in the common stock of Solid and Arcturus were as follows (in thousands):


	Common stock
As of December 31, 2021	$	32,200
Change in fair value		(19,299	)
Sale of shares		(10,094	)
December 31, 2022		2,807
Change in fair value		(334	)
March 31, 2023	$	2,473

Liabilities for Sales of Future Royalties - Schedule of Activity Within Liability For Sale of Future Royalties (Details) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2023	Dec. 31, 2022
Liability For Sale Of Future Royalties [Line Items]
Liability for sale of future royalties at beginning of year	$ 875,439	$ 351,786
Net proceeds from sale of future royalties		490,950
Royalty revenue	(4,882)	(21,692)
Non-cash interest expense	19,456	54,395
Liability for sale of future royalties at end of year	890,013	875,439
RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Liability for sale of future royalties at beginning of year	365,189	351,786
Net proceeds from sale of future royalties		0
Royalty revenue	(4,882)	(21,692)
Non-cash interest expense	8,726	35,095
Liability for sale of future royalties at end of year	369,033	365,189
OMERS
Liability For Sale Of Future Royalties [Line Items]
Liability for sale of future royalties at beginning of year	510,250	0
Net proceeds from sale of future royalties		490,950
Royalty revenue	0	0
Non-cash interest expense	10,730	19,300
Liability for sale of future royalties at end of year	$ 520,980	$ 510,250

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Unaudited) - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit	Treasury Stock	Deferred Compensation On Obligation
Beginning balance at Dec. 31, 2021	$ 922,561	$ 69	$ 2,997,497	$ (1,404)	$ (2,073,601)	$ 0	$ 0
Beginning balance, shares at Dec. 31, 2021		69,344,998
Stock-based compensation	29,578		29,578
Issuance of common stock under equity plan awards, net of tax	1,755	$ 1	1,754
Issuance of common stock under equity plan awards, net of tax, shares		451,942
Other comprehensive income (loss)	(5,150)			(5,150)
Net loss	(152,320)				(152,320)
Ending balance at Mar. 31, 2022	796,424	$ 70	3,028,829	(6,554)	(2,225,921)	0	0
Ending balance, shares at Mar. 31, 2022		69,796,940
Beginning balance at Dec. 31, 2022	$ 352,494	$ 70	3,140,019	(6,573)	(2,781,022)	0	0
Beginning balance, shares at Dec. 31, 2022	70,197,297	70,197,297
Stock-based compensation	$ 30,103		30,103
Issuance of common stock under equity plan awards, net of tax	(750)	$ 1	(751)
Issuance of common stock under equity plan awards, net of tax, shares		462,952
Deferred compensation						(356)	356
Other comprehensive income (loss)	2,606			2,606
Net loss	(163,972)				(163,972)
Ending balance at Mar. 31, 2023	$ 220,481	$ 71	$ 3,169,371	$ (3,967)	$ (2,944,994)	$ (356)	$ 356
Ending balance, shares at Mar. 31, 2023	70,660,249	70,660,249

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

+ Details

- Definition

Amount after tax and reclassification adjustments of other comprehensive income (loss).

+ References

+ Details

- Definition

Total of all stockholders' equity (deficit) items, net of receivables from officers, directors, owners, and affiliates of the entity which are attributable to the parent. The amount of the economic entity's stockholders' equity attributable to the parent excludes the amount of stockholders' equity which is allocable to that ownership interest in subsidiary equity which is not attributable to the parent (noncontrolling interest, minority interest). This excludes temporary equity and is sometimes called permanent equity.

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
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+ Details

Stock-Based Awards - Additional Information (Details)	3 Months Ended
Stock-Based Awards - Additional Information (Details)	Mar. 31, 2023 shares
2014 Incentive Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share reserved for future issuance	861,763
Automatic increases in shares available for grant effective date	Jan. 01, 2015
Shares available for grant, ending date	Jan. 01, 2024
2014 Employee Stock Purchase Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share reserved for future issuance	5,405,257
Employment Inducement Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share reserved for future issuance	247,369

Organization

3 Months Ended

Mar. 31, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Organization

1. Organization

Ultragenyx Pharmaceutical Inc., or the Company, is a biopharmaceutical company incorporated in Delaware.

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 operates as one reportable segment and has four commercially approved products.

Crysvita® (burosumab) is approved in the United States, or U.S., the European Union, or EU, and certain other regions for the treatment of X-linked hypophosphatemia, or XLH, in adult and pediatric patients one year of age and older. Crysvita is also approved in the U.S. and certain other regions for the treatment of fibroblast growth factor 23, or FGF23,-related hypophosphatemia in tumor-induced osteomalacia, or TIO, associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.

Mepsevii® (vestronidase alfa) is approved in the U.S., the EU and certain other regions, as the first medicine for the treatment of children and adults with mucopolysaccharidosis VII, or MPS VII, also known as Sly syndrome.

Dojolvi® (triheptanoin) is approved in the U.S. and certain other regions for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders, or LC-FAOD.

Evkeeza® (evinacumab) is approved in the U.S. and the European Economic Area, or EEA, for the treatment of homozygous familial hypercholesterolemia, or HoFH. The Company has exclusive rights to commercialize Evkeeza® (evinacumab) outside of the U.S.

In addition to the approved products, the Company has the following ongoing clinical development programs:

•

UX111 (formerly ABO-102) is an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease;

•

DTX401 is an adeno-associated virus 8, or AAV8, gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia, or GSDIa;

•

DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase, or OTC deficiency, the most common urea cycle disorder;

•

UX143 (setrusumab), which is subject to the Company’s collaboration agreement with Mereo BioPharma 3, or Mereo, is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells for the treatment of patients with osteogenesis imperfect, or OI;

•

GTX-102 is an antisense oligonucleotide, or ASO, for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene;

•

UX701 is an adeno-associated virus 9, or AAV9, gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease; and

•

UX053 is a messenger RNA, or mRNA, product candidate designed for the treatment of patients with Glycogen Storage Disease Type III, or GSDIII, a disease caused by a glycogen debranching enzyme, or AGL, deficiency that results in glycogen accumulation in the liver and muscle.

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. Management recognizes that the Company will likely need to raise additional capital to fully implement its business plans. Through March 31, 2023, the Company has relied primarily on its sale of equity securities, its revenues from commercial products, its sale of future royalties, and strategic collaboration arrangements to finance its operations.

The Company expects it will need to 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.

License and Research Agreements

3 Months Ended

Mar. 31, 2023

Research Grant Agreement [Abstract]

License and Research Agreements

7. License and Research Agreements

Kyowa Kirin Co., Ltd.

In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd., or KKC (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 U.S. 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 shared 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. In April 2023, which was the transition date for the profit-share territory, KKC became the lead party and is 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.

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 the Profit-share Territory

The Company and KKC shared 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 a transfer price of 30% of net sales. The transfer price on these sales was 35% prior to December 31, 2022. The remaining profit or loss after supply costs from commercializing products in the profit-share territory was shared between the Company and KKC on a 50/50 basis until April 2023. In April 2023, commercialization responsibilities for Crysvita in the profit-share territory transitioned to KKC and KKC assumed responsibility for the commercialization of Crysvita in the territory at and after April 2023. Thereafter, the Company is entitled to receive a tiered double-digit revenue share from the mid-20% range up to a maximum rate of 30%.

In September 2022, the Company entered into an amendment to the collaboration agreement which clarified the scope of increased participation by KKC in support of the Company’s commercial activities prior to April 2023 and granted the Company the right to continue to support KKC in commercial field activities in the U.S. through April 2024, subject to the limitations and conditions set forth in the amendment. As a result, the Company will continue to support commercial field and marketing efforts

through a cost share arrangement through April 2024, subject to the limits and conditions set forth in the amendment. After April 2024, the Company’s rights to promote Crysvita in the U.S. will be limited to medical geneticists and the Company will solely bear its expenses related to the promotion of Crysvita in the profit-share territory.

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 prior to April 2023 was analogous to a royalty and therefore recorded its share as collaboration revenue, similar to a royalty. Starting in April 2023, the Company began to record the royalty revenue as the underlying sales occur.

In July 2022, the Company sold to OMERS its right to receive 30% of the future royalty payments due to the Company based on net sales of Crysvita in the U.S. and Canada, subject to a cap, beginning in April 2023, as further described in Note 8.

Royalty Revenue Related to Sales in the 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 8. Liabilities for Sales of Future Royalties.” 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. The Company records this revenue as royalty revenue.

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. 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 Latin American territories and charges the Company a transfer price of 30% of net sales. The transfer price on these sales was 35% prior to December 31, 2022. The Company also pays to KKC a low single-digit royalty on net sales in Latin America.

Total Crysvita revenue was as follows (in thousands):


	Three Months Ended March 31,
	2023		2022
Revenue in profit-share territory	$	49,906	$	45,164
Product revenue		21,234		9,394
Non-cash royalty revenue in European territory		4,882		4,838
Total Crysvita revenue	$	76,022	$	59,396

Cost Sharing Payments


	Three Months Ended March 31,
	2023		2022
Research and development	$	2,272	$	4,619
Selling, general and administrative		9,080		9,196
Total	$	11,352	$	13,815

Collaboration Receivable and Payable

The Company had accounts receivable from KKC in the amount of $17.4 million and $27.5 million from profit-share revenue and royalties and other receivables recorded in prepaid expenses and other current assets of $12.7 million and $6.4 million and accrued liabilities of $6.9 million and $3.1 million from commercial and development activity reimbursements, as of March 31, 2023 and December 31, 2022, respectively.

Daiichi Sankyo

In March 2020, the Company executed a License and Technology Access Agreement, or the License Agreement, with Daiichi Sankyo Co., Ltd., or 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 Pinnacle PCLTM producer cell line platform, or Pinnacle PCL Platform, and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products.

Under the terms of the License Agreement, Daiichi Sankyo made an upfront payment of $125.0 million and an additional $25.0 million upon completion of the technology transfer of the Pinnacle PCL Platform and HEK293 platform. Daiichi Sankyo reimbursed the Company for all costs associated with the transfer of the manufacturing technology.

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 was recognized over an initial period which was completed during the first quarter of 2022, 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 was recognized on a straight-line basis over the remaining technology transfer period, which ended in March 2023, as it was expected that Daiichi Sankyo would receive and consume the benefits consistently throughout the period. The license agreement and the sublicense agreement were terminated following the conclusion of the technology transfer period. Total revenue recognized under the license agreement through March 31, 2023 was $183.3 million.

The Company recognized $1.5 million and $3.2 million for the three months ended March 31, 2023 and 2022, respectively, in revenue related to this arrangement. The Company recorded nil and $1.5 million of contract liabilities as of March 31, 2023 and December 31, 2022, respectively.

Mereo

In December 2020, the Company entered into a License and Collaboration Agreement with Mereo to collaborate on the development of setrusumab. Under the terms of the agreement, the Company will lead future global development of setrusumab in both pediatric and adult patients with Osteogenesis Imperfecta, or OI. The Company was granted an exclusive license to develop and commercialize setrusumab in the U.S., Turkey, and the rest of the world, excluding the European Economic Area, United Kingdom, and Switzerland, or the Mereo territory, where Mereo retains commercial rights. Each party will be responsible for post-marketing commitments and commercial supply in their respective territories.

Upon the closing of the transaction in January 2021, the Company made a payment of $50.0 million to Mereo and will be required to make payments of up to $254.0 million upon the achievement of certain clinical, regulatory, and commercial milestones. The Company will pay for all global development costs as well as tiered double-digit percentage royalties to Mereo on net sales in the U.S., Turkey, and the rest of the world (excluding the Mereo Territory), and Mereo will pay the Company a fixed double-digit percentage royalty on net sales in the Mereo Territory.

Although Mereo is a variable interest entity, the Company is not the primary beneficiary as it does not have the power to direct the activities that would most significantly impact the economic performance of Mereo. Prior to the achievement of certain development milestones, all consideration paid to Mereo represents rights to potential future benefits associated with Mereo’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use.

Regeneron

In January 2022, the Company announced a collaboration with Regeneron Pharmaceuticals, or Regeneron, to commercialize Evkeeza for HoFH outside of the U.S. Evkeeza is approved in the U.S., where it is marketed by Regeneron, and in the EU and U.K. as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol-lowering therapies to treat adults and adolescents aged 12 years and older with HoFH. Pursuant to the terms of the agreement, the Company received the rights to develop, commercialize and distribute the product for HoFH in countries outside of the U.S. The Company is obligated to pay up to $63.0 million in future milestone payments, contingent upon the achievement of certain regulatory and sales milestones. The Company may share in certain costs for global trials led by Regeneron and also received the right to opt into other potential indications.

The collaboration agreement is within the scope of ASC 808 which provides guidance on the presentation and disclosure of collaborative arrangements. As the Company would be the principal in future sale transactions with the customer, the Company will recognize product sales and cost of sales in the period the related sales occur and the related revenue recognition criteria are met. Under the collaboration agreement, Regeneron supplies the product and charges the Company a transfer price from the low 20%

range up to 40% on net sales, which is recognized as cost of sales in the Company’s Condensed Consolidated Statement of Operations.

Upon the closing of the transaction in January 2022, the Company paid Regeneron a $30.0 million upfront payment. As the upfront payment was related to the Company’s usage of intellectual property related to Evkeeza for HoFH, the upfront payment was recorded as an intangible asset, which is amortized over its useful life of 10.5 years.

The Company recorded costs of goods sold of $0.8 million and $0.7 million for the three months ended March 31, 2023 and 2022, respectively, of which $0.7 million and $0.7 million, respectively related to the amortization of the intangible asset. Further, the Company reimbursed Regeneron for certain research and development costs of $3.2 million and $0.6 million for the three months ended March 31, 2023 and 2022, respectively.

Abeona

In May 2022, the Company announced an exclusive license agreement for the AAV gene therapy for UX111 with Abeona Therapeutics, Inc., or Abeona, for the treatment of MPS IIIA. Under the terms of the agreement, the Company assumed responsibility for the UX111 program and in return, Abeona is eligible to receive tiered royalties of up to 10% on net sales and commercial milestone payments of up to $30.0 million following regulatory approval of the product. Additionally, the Company entered into an Assignment and Assumption Agreement with Abeona to transfer and assign to the Company the exclusive license agreement between Nationwide Children’s Hospital, or NCH, and Abeona for certain rights related to UX111. Under the NCH agreement, NCH is eligible to receive from the Company up to $1.0 million in development and regulatory milestones as well as royalties in the low single-digits of net sales.

The Company is obligated to pay Abeona certain prior development costs and other transition costs related to UX111. Prior to product regulatory approval, all consideration paid to Abeona represents rights to potential future benefits associated with Abeona’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use.

Other Arrangements

The Company has also entered into several collaborations and/or licensing arrangements in prior periods. Except as disclosed above, there have been no material changes in these arrangements during the three months ended March 31, 2023 as compared to those disclosed in "Note 8 License and Research Agreements" to the Consolidated Financial Statements in the Annual Report.

Under the financial terms of these arrangements, the Company may be required to make payments upon achievement of developmental, regulatory, and commercial milestones, which could be significant. Future milestone payments, if any, will be reflected in the Condensed Consolidated Statements of Operations upon the occurrence of the contingent event. In addition, the Company may be required to pay royalties on future sales if products related to these arrangements are commercialized. The payment of these amounts, however, is contingent upon the occurrence of various future events, which have a high degree of uncertainty of occurrence.

As described in the Annual Report, in connection with the Company’s collaborations with Solid and Arcturus Therapeutics Holdings, Inc., or Arcturus, the Company previously held certain equity interests in each entity. As of December 31, 2022, the Company held no shares of Arcturus common stock. The changes in the fair value of the Company’s equity investments in the common stock of Solid and Arcturus were as follows (in thousands):


	Common stock
As of December 31, 2021	$	32,200
Change in fair value		(19,299	)
Sale of shares		(10,094	)
December 31, 2022		2,807
Change in fair value		(334	)
March 31, 2023	$	2,473

Equity Transactions

3 Months Ended

Mar. 31, 2023

Equity [Abstract]

Equity Transactions

11. Equity Transactions

In May 2021, the Company entered into an Open Market Sale Agreement with Jefferies LLC, or Jefferies, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in at-the-market, or ATM, offerings through Jefferies. As of March 31, 2023, the Company has sold 1,050,372 shares under the arrangement resulting in net proceeds of approximately $78.9 million. No shares were sold under the arrangement for the three months ended March 31, 2023.

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-URI https://asc.fasb.org/topic&trid=2208762

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Topic 505
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-Section 50
-Paragraph 13
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496158-112644

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
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Reference 14: http://www.xbrl.org/2003/role/disclosureRef
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Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) (Unaudited) - shares	Mar. 31, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Preferred stock, shares authorized	25,000,000	25,000,000
Preferred stock, shares outstanding	0	0
Common stock, shares authorized	250,000,000	250,000,000
Common stock, shares issued	70,660,249	70,197,297
Common stock, shares outstanding	70,660,249	70,197,297
Treasury stock , shares	7,812	0

Financial Instruments - Summary of Financial Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	$ 701,004	$ 884,017
Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	69,366	133,299
Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	631,638	750,718
Money Market Funds
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	51,187	102,847
Money Market Funds \| Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	51,187	102,847
Asset-backed Securities
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	11,718	11,980
Asset-backed Securities \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	11,718	11,980
Certificate of Deposits and Time Deposits
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	13,848	25,972
Certificate of Deposits and Time Deposits \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	13,848	25,972
Corporate Bonds
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	312,654	427,598
Corporate Bonds \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	312,654	427,598
Commercial Paper
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	161,958	135,393
Commercial Paper \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	161,958	135,393
U.S. Government Treasury and Agency Securities
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	128,647	156,990
U.S. Government Treasury and Agency Securities \| Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	15,706	27,645
U.S. Government Treasury and Agency Securities \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	112,941	129,345
Debt Securities in Government Sponsored Entities
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	9,973	15,855
Debt Securities in Government Sponsored Entities \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	9,973	15,855
Investments in Arcturus and Solid Common Stock
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	2,473	2,807
Investments in Arcturus and Solid Common Stock \| Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	2,473	2,807
Other
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	8,546	4,575
Other \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	$ 8,546	$ 4,575

Revenue - Additional Information (Details)	Mar. 31, 2023	Dec. 31, 2022
Customer One
Disaggregation of Revenue [Line Items]
Percentage of gross accounts receivable balance	40.00%	68.00%
Customer Two
Disaggregation of Revenue [Line Items]
Percentage of gross accounts receivable balance	26.00%	0.00%

License and Research Agreements - Schedule of Cost Sharing Payments (Details) - Kyowa Kirin Collaboration - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Cost Sharing Payments [Line Items]
Research and development	$ 2,272	$ 4,619
Selling, general and administrative	9,080	9,196
Total	$ 11,352	$ 13,815

Liabilities for Sales of Future Royalties (Tables)

3 Months Ended

Mar. 31, 2023

Deferred Revenue Disclosure [Abstract]

Schedule of activity within liability for sale of future royalties

The following table shows the activity within the liability account (in thousands):


	Liabilities for Sales of Future Royalties
	RPI			OMERS		Total
December 31, 2021	$	351,786		$	—	$	351,786
Net proceeds from sale of future royalties		—			490,950		490,950
Royalty revenue		(21,692	)		—		(21,692	)
Non-cash interest expense		35,095			19,300		54,395
December 31, 2022		365,189			510,250		875,439
Royalty revenue		(4,882	)		—		(4,882	)
Non-cash interest expense		8,726			10,730		19,456
March 31, 2023	$	369,033		$	520,980	$	890,013

Accumulated Other Comprehensive Loss

3 Months Ended

Mar. 31, 2023

Other Comprehensive Income (Loss), Net of Tax [Abstract]

Accumulated Other Comprehensive Loss

12. Accumulated Other Comprehensive Loss

Total accumulated other comprehensive loss consisted of the following (in thousands):


	March 31,			December 31,
	2023			2022
Foreign currency translation adjustments	$	(701	)	$	(845	)
Unrealized loss on available-for-sale securities		(3,266	)		(5,728	)
Total accumulated other comprehensive loss	$	(3,967	)	$	(6,573	)

Balance Sheet Components

3 Months Ended

Mar. 31, 2023

Investments, Debt and Equity Securities [Abstract]

Balance Sheet Components

4. Balance Sheet Components

Cash Equivalents and Marketable Debt Securities

The fair values of cash equivalents and investments classified as available-for-sale securities consisted of the following (in thousands):


	March 31, 2023
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	51,187	$	—	$	—		$	51,187
Certificates of deposit and time deposits			13,848		—		—			13,848
Corporate bonds			315,735		140		(3,221	)		312,654
Commercial paper			161,958		—		—			161,958
Asset-backed securities			11,774		—		(56	)		11,718
U.S. Government Treasury and agency securities			128,749		583		(685	)		128,647
Debt securities in government-sponsored entities			10,000		—		(27	)		9,973
Total		$	693,251	$	723	$	(3,989	)	$	689,985


	December 31, 2022
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	102,847	$	—	$	—		$	102,847
Certificates of deposit and time deposits			25,972		—		—			25,972
Corporate bonds			432,211		87		(4,700	)		427,598
Commercial paper			135,393		—		—			135,393
Asset-backed securities			12,002		—		(22	)		11,980
U.S. Government Treasury and agency securities			157,933		320		(1,263	)		156,990
Debt securities in government-sponsored entities			16,005		—		(150	)		15,855
Total		$	882,363	$	407	$	(6,135	)	$	876,635

At March 31, 2023, the remaining contractual maturities of available-for-sale securities were less than three years. There have been no significant realized gains or losses on available-for-sale securities for the periods presented. For investments with unrealized losses, the Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis.

Inventory

Inventory consists of the following (in thousands):


	March 31,		December 31,
	2023		2022
Work-in-process	$	15,091	$	17,486
Finished goods		11,525		9,280
Total inventory	$	26,616	$	26,766

Accrued Liabilities

Accrued liabilities consist of the following (in thousands):


	March 31,		December 31,
	2023		2022
Research, clinical study, and manufacturing expenses	$	42,725	$	73,558
Payroll and related expenses		55,796		78,938
Other		45,883		52,182
Total accrued liabilities	$	144,404	$	204,678

Financial Instruments (Tables)

3 Months Ended

Mar. 31, 2023

Fair Value Disclosures [Abstract]

Summary of Financial Assets Measured on Recurring Basis

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):


	March 31, 2023
	Level 1		Level 2		Level 3		Total
Money market funds	$	51,187	$	—	$	—	$	51,187
Certificate of deposits and time deposits		—		13,848		—		13,848
Corporate bonds		—		312,654		—		312,654
Commercial paper		—		161,958		—		161,958
Asset-backed securities		—		11,718		—		11,718
U.S. Government Treasury and agency securities		15,706		112,941		—		128,647
Debt securities in government-sponsored entities		—		9,973		—		9,973
Investment in Solid common stock		2,473		—		—		2,473
Other		—		8,546		—		8,546
Total	$	69,366	$	631,638	$	—	$	701,004


	December 31, 2022
	Level 1		Level 2		Level 3		Total
Money market funds	$	102,847	$	—	$	—	$	102,847
Certificates of deposits and time deposits		—		25,972		—		25,972
Corporate bonds		—		427,598		—		427,598
Commercial paper		—		135,393		—		135,393
Asset-backed securities		—		11,980		—		11,980
U.S. Government Treasury and agency securities		27,645		129,345		—		156,990
Debt securities in government-sponsored entities		—		15,855		—		15,855
Investment in Solid common stock		2,807		—		—		2,807
Other		—		4,575		—		4,575
Total	$	133,299	$	750,718	$	—	$	884,017

Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022	Mar. 31, 2022	Dec. 31, 2021
Accounting Policies [Abstract]
Cash and cash equivalents	$ 85,768	$ 132,944	$ 153,988
Restricted cash included in prepaid expenses and other current assets	$ 1,964		$ 217
Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List]	Prepaid expenses and other current assets		Prepaid expenses and other current assets
Restricted cash included in other assets	$ 3,795		$ 2,001
Restricted Cash and Cash Equivalents, Noncurrent, Asset, Statement of Financial Position [Extensible List]	Other assets		Other assets
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows	$ 91,527	$ 137,601	$ 156,206	$ 309,585

Balance Sheet Components - Additional Information (Details)	3 Months Ended
	Mar. 31, 2022
Maximum
Schedule Of Available For Sale Securities [Line Items]
Available-for-sale securities remaining contractual maturities	3 years

Net Loss Per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) - shares	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Common stock equivalents excluded from computation of diluted net loss per share	12,923,444	9,386,957
Options to Purchase Common Stock, RSUs, and PSUs
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Common stock equivalents excluded from computation of diluted net loss per share	12,825,527	9,363,681
Employee Stock Purchase Plan
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Common stock equivalents excluded from computation of diluted net loss per share	97,917	23,276

Revenue - Summary of Disaggregation of Total Revenues from Customers (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Total revenues
Total revenues	$ 100,496	$ 79,935
Collaboration and License
Total revenues
Total revenues	51,385	48,413
Collaboration and License \| Crysvita
Total revenues
Total revenues	49,906	45,164
Collaboration and License \| Daiichi Sankyo
Total revenues
Total revenues	1,479	3,249
Product Revenue
Total revenues
Total revenues	44,229	26,684
Product Revenue \| Crysvita
Total revenues
Total revenues	21,234	9,394
Product Revenue \| Mepsevii
Total revenues
Total revenues	8,480	4,861
Product Revenue \| Dojolvi
Total revenues
Total revenues	14,303	12,429
Product Revenue \| Evkeeza
Total revenues
Total revenues	212	0
Royalty Revenue
Total revenues
Total revenues	4,882	4,838
Royalty Revenue \| Crysvita
Total revenues
Total revenues	$ 4,882	$ 4,838

Revenue

3 Months Ended

Mar. 31, 2023

Revenue from Contract with Customer [Abstract]

Revenue

5. Revenue

The following table disaggregates total revenues from external customers by collaboration and license revenue and product sales (in thousands):


	Three Months Ended
	March 31,
	2023		2022
Collaboration and license revenue:
Crysvita collaboration revenue in profit- share territory	$	49,906	$	45,164
Daiichi Sankyo		1,479		3,249
Total collaboration and license revenue		51,385		48,413
Product sales:
Crysvita		21,234		9,394
Mepsevii		8,480		4,861
Dojolvi		14,303		12,429
Evkeeza		212		—
Total product sales		44,229		26,684
Crysvita royalty revenue		4,882		4,838
Total revenues	$	100,496	$	79,935

The following table disaggregates total revenues based on geographic location (in thousands):


	Three Months Ended
	March 31,
	2023		2022
North America	$	67,132	$	61,917
Latin America		22,799		9,791
Europe		9,387		8,227
Japan		1,178		—
Total revenues	$	100,496	$	79,935

The following table presents changes in the contract liabilities (in thousands):


	Three Months Ended March 31,
	2023			2022
Balance of contract liabilities at beginning of period	$	1,479		$	9,076
Additions		—			40
Deductions		(1,479	)		(3,249	)
Balance of contract liabilities at end of period, net	$	—		$	5,867

See Note 7 for additional details on contract liabilities activities.

The Company’s largest accounts receivable balance was from a collaboration partner and accounted for 40% and 68% of the total accounts receivable balance as of March 31, 2023 and December 31, 2022, respectively. The accounts receivable balance from a different customer accounted for 26% and nil of the total accounts receivable balance as of March 31, 2023 and December 31, 2022, respectively.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-URI https://asc.fasb.org/topic&trid=49130388

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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-Publisher FASB
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-Name Accounting Standards Codification
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+ Details

Revenue - Summary of Disaggregation of Total Revenues (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Disaggregation Of Revenue [Line Items]
Total revenues	$ 100,496	$ 79,935
North America
Disaggregation Of Revenue [Line Items]
Total revenues	67,132	61,917
Latin America
Disaggregation Of Revenue [Line Items]
Total revenues	22,799	9,791
Europe
Disaggregation Of Revenue [Line Items]
Total revenues	9,387	8,227
Japan
Disaggregation Of Revenue [Line Items]
Total revenues	$ 1,178	$ 0

- Definition

+ References

+ Details

Summary of Significant Accounting Policies

3 Months Ended

Mar. 31, 2023

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

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, or 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 Condensed 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 17, 2023, or Annual Report, with the United States Securities and Exchange Commission, or the SEC.

The results of operations for the three months ended March 31, 2023 are not necessarily indicative of the results to be expected for the year ending December 31, 2023. The Condensed Consolidated Balance Sheet as of December 31, 2022 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.

Use of Estimates

The accompanying Condensed Consolidated Financial Statements have been prepared in accordance with GAAP. The preparation of the Condensed 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 Condensed 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, revenue recognition, and the liabilities for sales 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 used 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 Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands):


	March 31,
	2023		2022
Cash and cash equivalents	$	85,768	$	153,988
Restricted cash included in prepaid expenses and other current assets		1,964		217
Restricted cash included in other assets		3,795		2,001
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows	$	91,527	$	156,206

Credit Losses

The Company is exposed to credit losses primarily through receivables from customers and collaborators and through its available-for-sale debt securities. For trade receivables and other instruments, the Company uses a 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’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. There were no material credit losses recorded for receivables and available-for-sale debt securities for the three months ended March 31, 2023 and 2022.

Revenue Recognition

Collaboration and License Revenue

The Company has certain license and collaboration agreements that are within the scope of Accounting Standards Codification, or 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 Condensed Consolidated Statements 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.

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 Company also records 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.

The Company sold the right to receive certain royalty payments from net sales of Crysvita in certain territories to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERS, as further described in “Note 8 Liabilities for Sales of Future Royalties”. The Company records the royalty revenue from the net sales of Crysvita in the applicable territories on a prospective basis as non-cash royalty revenue in the Condensed Consolidated Statements of Operations over the term of the applicable arrangement.

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, or 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. The Company’s estimates of government mandated rebates, chargebacks, estimated product returns, and other deductions depends on the identification of key customer contract terms and conditions, as well as estimates of sales volumes to different classes of payors. If actual results vary, the Company may need to adjust these estimates, which could have a material effect on earnings in the period of the adjustment.

Deferred Compensation Plan

The Company maintains a nonqualified deferred compensation plan whereby certain employees and members of the board of directors are able to defer certain equity awards and other compensation. Amounts deferred are invested into various mutual funds. The plan complies with the provisions of Section 409A of the Internal Revenue Code. All of the various mutual funds held in the plan are classified as trading securities and recorded at fair value in other non-current assets in the Condensed Consolidated Balance Sheets with changes in fair value recognized as earnings in the period they occur. The corresponding liability for the plan is included in other non-current liabilities in the Condensed Consolidated Balance Sheets. Certain equity awards deferred under the plan are required to be settled through the issuance of Company stock. These awards are recorded as treasury stock and deferred compensation obligation within stockholders’ equity.

Stock-Based Awards (Tables)

3 Months Ended

Mar. 31, 2023

Share-Based Payment Arrangement [Abstract]

Summary of Stock-Based Compensation Expense

The table below sets forth the stock-based compensation expense for the periods presented (in thousands):


	Three Months Ended March 31,
	2023		2022
Cost of sales	$	346	$	175
Research and development		18,120		16,907
Selling, general and administrative		13,473		12,305
Total stock-based compensation expense	$	31,939	$	29,387

License and Research Agreements - Schedule of Changes in Fair Value of Equity Investment (Details) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2023	Dec. 31, 2022
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Common stock, Beginning balance	$ 884,017
Common stock, Ending balance	701,004	$ 884,017
Solid and Arcturus \| Common Stock
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Common stock, Beginning balance	2,807	32,200
Common stock, Change in fair value	(334)	(19,299)
Common stock, Sale of shares		(10,094)
Common stock, Ending balance	$ 2,473	$ 2,807

Summary of Significant Accounting Policies (Policies)

3 Months Ended

Mar. 31, 2023

Accounting Policies [Abstract]

Basis of Presentation

Use of Estimates

Cash, Cash Equivalents, and Restricted Cash

Cash, Cash Equivalents and Restricted Cash


	March 31,
	2023		2022
Cash and cash equivalents	$	85,768	$	153,988
Restricted cash included in prepaid expenses and other current assets		1,964		217
Restricted cash included in other assets		3,795		2,001
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows	$	91,527	$	156,206

Credit Losses

Revenue Recognition

Collaboration and License Revenue

Product Sales

Deferred Compensation Plan

- Definition

Disclosure of accounting policy for credit loss on financial instrument measured at amortized cost basis, net investment in lease, off-balance sheet credit exposure, and available-for-sale debt security. Includes, but is not limited to, methodology used to estimate allowance for credit loss, how writeoff of uncollectible amount is recognized, and determination of past due status and nonaccrual status.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
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+ Details

Accumulated Other Comprehensive Loss - Schedule of Total Accumulated Other Comprehensive Loss (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract]
Foreign currency translation adjustments	$ (701)	$ (845)
Unrealized loss on available-for-sale securities	(3,266)	(5,728)
Total accumulated other comprehensive loss	$ (3,967)	$ (6,573)

- Definition

Accumulated change in equity from transactions and other events and circumstances from non-owner sources, net of tax effect, at period end. Excludes Net Income (Loss), and accumulated changes in equity from transactions resulting from investments by owners and distributions to owners. Includes foreign currency translation items, certain pension adjustments, unrealized gains and losses on certain investments in debt and equity securities, other than temporary impairment (OTTI) losses related to factors other than credit losses on available-for-sale and held-to-maturity debt securities that an entity does not intend to sell and it is not more likely than not that the entity will be required to sell before recovery of the amortized cost basis, as well as changes in the fair value of derivatives related to the effective portion of a designated cash flow hedge.

+ References + Details