$RARE
Ultragenyx Pharmaceutical Inc. Stock $rare

$rare Overview Financials Valuation SEC Filings Company Website

2022 Q2 Form 10-Q Financial Statement

#000095017022007940 Filed on May 06, 2022

Income Statement

Concept	2022 Q2	2022 Q1	2021 Q1
Revenue	$89.34M	$79.94M	$99.40M
YoY Change	2.72%	-19.58%	173.75%
Cost Of Revenue	$8.270M	$6.100M	$5.190M
YoY Change	163.71%	17.53%	-248.29%
Gross Profit	$81.07M	$73.84M	$94.21M
YoY Change	-3.3%	-21.63%	136.65%
Gross Profit Margin	90.74%	92.37%	94.78%
Selling, General & Admin	$68.14M	$67.31M	$53.26M
YoY Change	27.57%	26.39%	12.08%
% of Gross Profit	84.04%	91.17%	56.53%
Research & Development	$154.5M	$143.2M	$147.5M
YoY Change	36.5%	-2.96%	30.59%
% of Gross Profit	190.6%	193.89%	156.58%
Depreciation & Amortization	$4.481M	$4.088M	$3.367M
YoY Change	43.71%	21.41%	18.14%
% of Gross Profit	5.53%	5.54%	3.57%
Operating Expenses	$222.7M	$210.5M	$200.8M
YoY Change	33.64%	4.83%	27.9%
Operating Profit	-$141.6M	-$136.6M	-$106.6M
YoY Change	71.06%	28.21%	-11.68%
Interest Expense	-$9.285M	-$8.835M	-$19.98M
YoY Change	-69.66%	-55.78%	-288.67%
% of Operating Profit
Other Income/Expense, Net	-$930.0K	$289.0K	-$795.0K
YoY Change	1288.06%	-136.35%	74.34%
Pretax Income	-$157.9M	-$151.8M	-$135.8M
YoY Change	29.43%	11.79%	14.46%
Income Tax	$302.0K	$558.0K	$379.0K
% Of Pretax Income
Net Earnings	-$158.2M	-$152.3M	-$136.1M
YoY Change	29.19%	11.88%	14.38%
Net Earnings / Revenue	-177.03%	-190.55%	-136.97%
Basic Earnings Per Share	-$2.26	-$2.19
Diluted Earnings Per Share	-$2.262M	-$2.191M	-$2.029M
COMMON SHARES
Basic Shares Outstanding	69.93M	69.38M	66.95M
Diluted Shares Outstanding	69.93M	69.52M

Balance Sheet

Concept	2022 Q2	2022 Q1	2021 Q1
SHORT-TERM ASSETS
Cash & Short-Term Investments	$542.8M	$598.3M	$965.4M
YoY Change	-35.82%	-38.02%	45.55%
Cash & Equivalents	$159.9M	$154.0M	$383.8M
Short-Term Investments	$382.9M	$444.3M	$581.6M
Other Short-Term Assets	$77.46M	$63.02M	$68.00M
YoY Change	21.16%	-7.32%	4.94%
Inventory	$21.08M	$17.81M	$12.46M
Prepaid Expenses
Receivables	$37.21M	$28.37M	$25.00M
Other Receivables	$0.00	$0.00	$0.00
Total Short-Term Assets	$678.5M	$707.5M	$1.071B
YoY Change	-28.54%	-33.93%	19.75%
LONG-TERM ASSETS
Property, Plant & Equipment	$207.4M	$177.1M	$126.1M
YoY Change	44.83%	40.42%	51.2%
Goodwill	$44.41M	$44.41M
YoY Change
Intangibles	$159.2M	$160.0M
YoY Change
Long-Term Investments	$178.7M	$241.1M	$216.4M
YoY Change	-22.88%	11.42%	180.67%
Other Assets	$21.14M	$21.22M	$8.900M
YoY Change	66.91%	138.4%	81.63%
Total Long-Term Assets	$642.1M	$676.5M	$526.9M
YoY Change	14.06%	28.39%	55.52%
TOTAL ASSETS
Total Short-Term Assets	$678.5M	$707.5M	$1.071B
Total Long-Term Assets	$642.1M	$676.5M	$526.9M
Total Assets	$1.321B	$1.384B	$1.598B
YoY Change	-12.68%	-13.38%	29.58%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable	$34.21M	$23.69M	$15.70M
YoY Change	57.01%	50.86%	9.03%
Accrued Expenses	$170.4M	$126.4M	$82.70M
YoY Change	87.08%	52.79%	22.12%
Deferred Revenue	$4.437M	$5.867M	$25.09M
YoY Change	13.42%	-76.62%	-82.73%
Short-Term Debt	$0.00	$0.00	$0.00
YoY Change
Long-Term Debt Due
YoY Change
Total Short-Term Liabilities	$220.6M	$167.2M	$127.0M
YoY Change	73.64%	31.68%	-35.5%
LONG-TERM LIABILITIES
Long-Term Debt	$0.00	$0.00	$0.00
YoY Change
Other Long-Term Liabilities	$3.768M	$3.392M	$382.8M
YoY Change	-99.01%	-99.11%	-2.79%
Total Long-Term Liabilities	$3.768M	$3.392M	$382.8M
YoY Change	-99.01%	-99.11%	-2.79%
TOTAL LIABILITIES
Total Short-Term Liabilities	$220.6M	$167.2M	$127.0M
Total Long-Term Liabilities	$3.768M	$3.392M	$382.8M
Total Liabilities	$642.6M	$587.6M	$543.2M
YoY Change	18.93%	8.17%	-12.95%
SHAREHOLDERS EQUITY
Retained Earnings	-$2.384B	-$2.226B
YoY Change
Common Stock	$70.00K	$70.00K
YoY Change
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity	$678.1M	$796.4M	$1.055B
YoY Change
Total Liabilities & Shareholders Equity	$1.321B	$1.384B	$1.598B
YoY Change	-12.68%	-13.38%	29.58%

Cashflow Statement

Concept	2022 Q2	2022 Q1	2021 Q1
OPERATING ACTIVITIES
Net Income	-$158.2M	-$152.3M	-$136.1M
YoY Change	29.19%	11.88%	14.38%
Depreciation, Depletion And Amortization	$4.481M	$4.088M	$3.367M
YoY Change	43.71%	21.41%	18.14%
Cash From Operating Activities	-$77.26M	-$117.5M	-$159.3M
YoY Change	18.22%	-26.25%	67.46%
INVESTING ACTIVITIES
Capital Expenditures	-$30.92M	$32.18M	$16.41M
YoY Change	54.04%	96.08%	16.44%
Acquisitions
YoY Change
Other Investing Activities	$110.1M	-$2.578M	-$166.0M
YoY Change	-619.91%	-98.45%	104.87%
Cash From Investing Activities	$79.17M	-$37.36M	-$182.4M
YoY Change	-291.92%	-79.52%	91.77%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities	5.837M	1.612M	12.65M
YoY Change	-53.77%	-87.26%	-77.26%
NET CHANGE
Cash From Operating Activities	-77.26M	-117.5M	-159.3M
Cash From Investing Activities	79.17M	-37.36M	-182.4M
Cash From Financing Activities	5.837M	1.612M	12.65M
Net Change In Cash	7.748M	-153.4M	-329.9M
YoY Change	-108.24%	-53.51%	143.85%
FREE CASH FLOW
Cash From Operating Activities	-$77.26M	-$117.5M	-$159.3M
Capital Expenditures	-$30.92M	$32.18M	$16.41M
Free Cash Flow	-$46.33M	-$149.7M	-$175.8M
YoY Change	2.33%	-14.82%	60.88%

Facts In Submission

Frame	Concept Type	Concept / XBRL Key	Value
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CY2022Q1	dei	Document Fiscal Period Focus DocumentFiscalPeriodFocus	Q1
CY2022Q1	dei	Entity Central Index Key EntityCentralIndexKey	0001515673
CY2022Q1	dei	Document Type DocumentType	10-Q
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CY2022Q1	dei	Document Period End Date DocumentPeriodEndDate	2022-03-31
CY2022Q1	dei	Document Fiscal Year Focus DocumentFiscalYearFocus	2022
CY2022Q1	dei	Document Transition Report DocumentTransitionReport	false
CY2022Q1	dei	Entity File Number EntityFileNumber	001-36276
CY2022Q1	dei	Entity Registrant Name EntityRegistrantName	ULTRAGENYX PHARMACEUTICAL INC.
CY2022Q1	dei	Entity Incorporation State Country Code EntityIncorporationStateCountryCode	DE
CY2022Q1	dei	Entity Tax Identification Number EntityTaxIdentificationNumber	27-2546083
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CY2022Q1	us-gaap	Equity Securities Fv Ni Gain Loss EquitySecuritiesFvNiGainLoss	-9329000
CY2021Q1	us-gaap	Equity Securities Fv Ni Gain Loss EquitySecuritiesFvNiGainLoss	-20619000
CY2022Q1	rare	Non Cash Collaboration Royalty Revenue NonCashCollaborationRoyaltyRevenue	4838000
CY2021Q1	rare	Non Cash Collaboration Royalty Revenue NonCashCollaborationRoyaltyRevenue	3872000
CY2022Q1	rare	Non Cash Interest Expense On Liability Related To Sale Of Future Royalties NonCashInterestExpenseOnLiabilityRelatedToSaleOfFutureRoyalties	6584000
CY2021Q1	rare	Non Cash Interest Expense On Liability Related To Sale Of Future Royalties NonCashInterestExpenseOnLiabilityRelatedToSaleOfFutureRoyalties	8418000
CY2022Q1	us-gaap	Other Noncash Income Expense OtherNoncashIncomeExpense	-35000
CY2021Q1	us-gaap	Other Noncash Income Expense OtherNoncashIncomeExpense	-325000
CY2022Q1	us-gaap	Increase Decrease In Accounts Receivable IncreaseDecreaseInAccountsReceivable	-33000
CY2021Q1	us-gaap	Increase Decrease In Accounts Receivable IncreaseDecreaseInAccountsReceivable	1993000
CY2022Q1	us-gaap	Increase Decrease In Inventories IncreaseDecreaseInInventories	1475000
CY2021Q1	us-gaap	Increase Decrease In Inventories IncreaseDecreaseInInventories	-474000
CY2022Q1	us-gaap	Increase Decrease In Prepaid Deferred Expense And Other Assets IncreaseDecreaseInPrepaidDeferredExpenseAndOtherAssets	-9126000
CY2021Q1	us-gaap	Increase Decrease In Prepaid Deferred Expense And Other Assets IncreaseDecreaseInPrepaidDeferredExpenseAndOtherAssets	10656000
CY2022Q1	rare	Increase Decrease In Accounts Payable Accrued Liabilities And Other Operating Liabilities IncreaseDecreaseInAccountsPayableAccruedLiabilitiesAndOtherOperatingLiabilities	-16164000
CY2021Q1	rare	Increase Decrease In Accounts Payable Accrued Liabilities And Other Operating Liabilities IncreaseDecreaseInAccountsPayableAccruedLiabilitiesAndOtherOperatingLiabilities	-23602000
CY2022Q1	rare	Increase Decrease In Contract Liabilities IncreaseDecreaseInContractLiabilities	-3209000
CY2021Q1	rare	Increase Decrease In Contract Liabilities IncreaseDecreaseInContractLiabilities	-41479000
CY2022Q1	us-gaap	Net Cash Provided By Used In Operating Activities NetCashProvidedByUsedInOperatingActivities	-117521000
CY2021Q1	us-gaap	Net Cash Provided By Used In Operating Activities NetCashProvidedByUsedInOperatingActivities	-159346000
CY2022Q1	us-gaap	Payments To Acquire Property Plant And Equipment PaymentsToAcquirePropertyPlantAndEquipment	32184000
CY2021Q1	us-gaap	Payments To Acquire Property Plant And Equipment PaymentsToAcquirePropertyPlantAndEquipment	16414000
CY2022Q1	us-gaap	Payments To Acquire Available For Sale Securities Debt PaymentsToAcquireAvailableForSaleSecuritiesDebt	203710000
CY2021Q1	us-gaap	Payments To Acquire Available For Sale Securities Debt PaymentsToAcquireAvailableForSaleSecuritiesDebt	405230000
CY2022Q1	us-gaap	Proceeds From Sale Of Available For Sale Securities Debt ProceedsFromSaleOfAvailableForSaleSecuritiesDebt	39422000
CY2021Q1	us-gaap	Proceeds From Sale Of Available For Sale Securities Debt ProceedsFromSaleOfAvailableForSaleSecuritiesDebt	14980000
CY2022Q1	us-gaap	Proceeds From Maturities Prepayments And Calls Of Available For Sale Securities ProceedsFromMaturitiesPrepaymentsAndCallsOfAvailableForSaleSecurities	188990000
CY2021Q1	us-gaap	Proceeds From Maturities Prepayments And Calls Of Available For Sale Securities ProceedsFromMaturitiesPrepaymentsAndCallsOfAvailableForSaleSecurities	224240000
CY2022Q1	rare	Purchase Of Mutual Funds Related To Nonqualified Deferred Compensation Plan PurchaseOfMutualFundsRelatedToNonqualifiedDeferredCompensationPlan	-2458000
CY2022Q1	us-gaap	Payments To Acquire Intangible Assets PaymentsToAcquireIntangibleAssets	30000000
CY2022Q1	us-gaap	Payments For Proceeds From Other Investing Activities PaymentsForProceedsFromOtherInvestingActivities	-2578000
CY2022Q1	us-gaap	Net Cash Provided By Used In Investing Activities NetCashProvidedByUsedInInvestingActivities	-37362000
CY2021Q1	us-gaap	Net Cash Provided By Used In Investing Activities NetCashProvidedByUsedInInvestingActivities	-182424000
CY2022Q1	us-gaap	Proceeds From Issuance Of Shares Under Incentive And Share Based Compensation Plans Including Stock Options ProceedsFromIssuanceOfSharesUnderIncentiveAndShareBasedCompensationPlansIncludingStockOptions	1755000
CY2021Q1	us-gaap	Proceeds From Issuance Of Shares Under Incentive And Share Based Compensation Plans Including Stock Options ProceedsFromIssuanceOfSharesUnderIncentiveAndShareBasedCompensationPlansIncludingStockOptions	12761000
CY2022Q1	us-gaap	Proceeds From Payments For Other Financing Activities ProceedsFromPaymentsForOtherFinancingActivities	-143000
CY2021Q1	us-gaap	Proceeds From Payments For Other Financing Activities ProceedsFromPaymentsForOtherFinancingActivities	-112000
CY2022Q1	us-gaap	Net Cash Provided By Used In Financing Activities NetCashProvidedByUsedInFinancingActivities	1612000
CY2021Q1	us-gaap	Net Cash Provided By Used In Financing Activities NetCashProvidedByUsedInFinancingActivities	12649000
CY2022Q1	us-gaap	Effect Of Exchange Rate On Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations EffectOfExchangeRateOnCashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	-108000
CY2021Q1	us-gaap	Effect Of Exchange Rate On Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations EffectOfExchangeRateOnCashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	-771000
CY2022Q1	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Period Increase Decrease Including Exchange Rate Effect CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsPeriodIncreaseDecreaseIncludingExchangeRateEffect	-153379000
CY2021Q1	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Period Increase Decrease Including Exchange Rate Effect CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsPeriodIncreaseDecreaseIncludingExchangeRateEffect	-329892000
CY2021Q4	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	309585000
CY2020Q4	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	726294000
CY2022Q1	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	156206000
CY2021Q1	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	396402000
CY2022Q1	rare	Operating And Finance Lease Liability From Obtaining Right Of Use Assets OperatingAndFinanceLeaseLiabilityFromObtainingRightOfUseAssets	132000
CY2022Q1	rare	Non Cash Interest Expense On Liability Related To The Sale Of Future Royalties Capitalized Into Ending Property Plant And Equipment NonCashInterestExpenseOnLiabilityRelatedToTheSaleOfFutureRoyaltiesCapitalizedIntoEndingPropertyPlantAndEquipment	2101000
CY2022Q1	us-gaap	Nature Of Operations NatureOfOperations	<p style="text-indent:-4.749%;padding-left:4.533%;font-size:10.0pt;margin-top:0.0pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">1. Organization</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company incorporated in Delaware.</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">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 </span><span style="font-size:10.0pt;font-family:Calibri;background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">one</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> reportable segment. The Company has four commercially approved products.</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">Crysvita® (burosumab) is approved in the United States (U.S.) by the U.S. Food and Drug Administration (FDA) and in Canada for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older, and is approved in the European Union (EU) and the United Kingdom, for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, adolescents, and adults. In Brazil, Colombia, and Mexico, Crysvita is approved for treatment of XLH in adult and pediatric patients one year of age and older. Crysvita is also approved in the U.S. by the FDA for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO), associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">Mepsevii® (vestronidase alfa) is approved by the FDA as the first medicine for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. In the European Union and the United Kingdom, Mepsevii is approved under exceptional circumstances for patients of all ages for the treatment of non-neurological manifestations of MPS VII. In Brazil, Mepsevii is approved for the treatment of MPS VII for patients of all ages.</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">Dojolvi® (triheptanoin) is approved in the U.S., Canada, and Brazil for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD).</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">On January 7, 2022, the Company announced a collaboration with Regeneron Pharmaceuticals (Regeneron) to commercialize Evkeeza® (evinacumab) outside of the U.S. Evkeeza is approved in the U.S. and the European Economic Area (EEA) for the treatment of homozygous familial hypercholesterolemia (HoFH).</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">In addition to the approved products, the Company has the following ongoing clinical development programs:</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:4.5pt;line-height:1.15;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:3.333%;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;min-width:fit-content;text-indent:0;display:inline-flex;font-size:10.0pt;font-family:Times New Roman;justify-content:flex-start;min-width:3.447919145106397%;">•</span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">DTX401 is an adeno-associated virus 8 (AAV8) gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia (GSDIa);</span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:4.5pt;line-height:1.15;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:3.333%;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;min-width:fit-content;text-indent:0;display:inline-flex;font-size:10.0pt;font-family:Times New Roman;justify-content:flex-start;min-width:3.447919145106397%;">•</span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder;</span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:4.5pt;line-height:1.15;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:3.333%;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;min-width:fit-content;text-indent:0;display:inline-flex;font-size:10.0pt;font-family:Times New Roman;justify-content:flex-start;min-width:3.447919145106397%;">•</span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">UX143 (setrusumab), which is subject to the Company's collaboration agreement with Mereo BioPharma 3 (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 (OI); </span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:4.5pt;line-height:1.15;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:3.333%;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;min-width:fit-content;text-indent:0;display:inline-flex;font-size:10.0pt;font-family:Times New Roman;justify-content:flex-start;min-width:3.447919145106397%;">•</span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">GTX-102 is an antisense oligonucleotide (ASO), which the Company is collaborating on the development with GeneTx Biotherapeutics LLC (GeneTx) 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;</span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:4.5pt;line-height:1.15;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:3.333%;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;min-width:fit-content;text-indent:0;display:inline-flex;font-size:10.0pt;font-family:Times New Roman;justify-content:flex-start;min-width:3.447919145106397%;">•</span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">UX701 is an AAV type 9 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</span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:4.5pt;line-height:1.15;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:3.333%;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;min-width:fit-content;text-indent:0;display:inline-flex;font-size:10.0pt;font-family:Times New Roman;justify-content:flex-start;min-width:3.447919145106397%;">•</span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">UX053 is a messenger RNA (mRNA) product candidate designed for the treatment of patients with Glycogen Storage Disease Type III (GSDIII), a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. </span></div></div><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">The Company has sustained operating losses and expects such annual losses to continue over the next several years. The Company’s ultimate success depends on the outcome of its research and development and commercialization activities, for which it expects to incur additional losses in the future. Management recognizes that the Company will likely need to raise additional capital to fully implement its business plans. Through March 31, 2022, 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.</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">The Company will likely raise additional capital through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company would need to reevaluate its operating plans.</span></p>
CY2022Q1	us-gaap	Use Of Estimates UseOfEstimates	<p style="text-indent:0.0%;font-size:10.0pt;margin-top:13.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">Use of Estimates</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.15;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;">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 liability related to the sale of future royalties. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates.</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Calibri;min-width:fit-content;"> </span></p>
CY2022Q1	us-gaap	Cash And Cash Equivalents At Carrying Value CashAndCashEquivalentsAtCarryingValue	153988000
CY2021Q1	us-gaap	Cash And Cash Equivalents At Carrying Value CashAndCashEquivalentsAtCarryingValue	383794000
CY2022Q1	us-gaap	Restricted Cash Current RestrictedCashCurrent	217000
CY2021Q1	us-gaap	Restricted Cash Current RestrictedCashCurrent	10687000
CY2022Q1	us-gaap	Restricted Cash Noncurrent RestrictedCashNoncurrent	2001000
CY2021Q1	us-gaap	Restricted Cash Noncurrent RestrictedCashNoncurrent	1921000
CY2022Q1	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	156206000
CY2021Q1	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations	396402000
CY2022Q1	us-gaap	Investments Fair Value Disclosure InvestmentsFairValueDisclosure	817850000
CY2021Q4	us-gaap	Investments Fair Value Disclosure InvestmentsFairValueDisclosure	1001451000
CY2022Q1	us-gaap	Available For Sale Debt Securities Amortized Cost Basis AvailableForSaleDebtSecuritiesAmortizedCostBasis	797952000
CY2022Q1	us-gaap	Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax	30000
CY2022Q1	us-gaap	Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax	6403000
CY2022Q1	us-gaap	Available For Sale Securities Debt Securities AvailableForSaleSecuritiesDebtSecurities	791579000
CY2021Q4	us-gaap	Available For Sale Debt Securities Amortized Cost Basis AvailableForSaleDebtSecuritiesAmortizedCostBasis	969592000
CY2021Q4	us-gaap	Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax	5000
CY2021Q4	us-gaap	Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax	1288000
CY2021Q4	us-gaap	Available For Sale Securities Debt Securities AvailableForSaleSecuritiesDebtSecurities	968309000
CY2022Q1	us-gaap	Inventory Work In Process InventoryWorkInProcess	11989000
CY2021Q4	us-gaap	Inventory Work In Process InventoryWorkInProcess	10504000
CY2022Q1	us-gaap	Inventory Finished Goods InventoryFinishedGoods	5820000
CY2021Q4	us-gaap	Inventory Finished Goods InventoryFinishedGoods	5727000
CY2022Q1	us-gaap	Inventory Net InventoryNet	17809000
CY2021Q4	us-gaap	Inventory Net InventoryNet	16231000
CY2022Q1	rare	Research Clinical Study And Manufacturing Expenses ResearchClinicalStudyAndManufacturingExpenses	40092000
CY2021Q4	rare	Research Clinical Study And Manufacturing Expenses ResearchClinicalStudyAndManufacturingExpenses	40880000
CY2022Q1	us-gaap	Employee Related Liabilities Current EmployeeRelatedLiabilitiesCurrent	41638000
CY2021Q4	us-gaap	Employee Related Liabilities Current EmployeeRelatedLiabilitiesCurrent	62591000
CY2022Q1	us-gaap	Other Accrued Liabilities Current OtherAccruedLiabilitiesCurrent	44626000
CY2021Q4	us-gaap	Other Accrued Liabilities Current OtherAccruedLiabilitiesCurrent	42084000
CY2022Q1	us-gaap	Accrued Liabilities Current AccruedLiabilitiesCurrent	126356000
CY2021Q4	us-gaap	Accrued Liabilities Current AccruedLiabilitiesCurrent	145555000
CY2022Q1	us-gaap	Revenue From Contract With Customer Excluding Assessed Tax RevenueFromContractWithCustomerExcludingAssessedTax	79935000
CY2021Q1	us-gaap	Revenue From Contract With Customer Excluding Assessed Tax RevenueFromContractWithCustomerExcludingAssessedTax	99395000
CY2022Q1	us-gaap	Revenue From Contract With Customer Excluding Assessed Tax RevenueFromContractWithCustomerExcludingAssessedTax	79935000
CY2021Q1	us-gaap	Revenue From Contract With Customer Excluding Assessed Tax RevenueFromContractWithCustomerExcludingAssessedTax	99395000
CY2021Q4	us-gaap	Contract With Customer Liability Current ContractWithCustomerLiabilityCurrent	9076000
CY2020Q4	us-gaap	Contract With Customer Liability Current ContractWithCustomerLiabilityCurrent	66568000
CY2022Q1	rare	Contract With Customer Assets Liability Additions During Period ContractWithCustomerAssetsLiabilityAdditionsDuringPeriod	-40000
CY2021Q1	rare	Contract With Customer Assets Liability Additions During Period ContractWithCustomerAssetsLiabilityAdditionsDuringPeriod	-1271000
CY2022Q1	rare	Contract With Customer Assets Liability Deductions During Period ContractWithCustomerAssetsLiabilityDeductionsDuringPeriod	3249000
CY2021Q1	rare	Contract With Customer Assets Liability Deductions During Period ContractWithCustomerAssetsLiabilityDeductionsDuringPeriod	42750000
CY2022Q1	us-gaap	Contract With Customer Liability Current ContractWithCustomerLiabilityCurrent	5867000
CY2021Q1	us-gaap	Contract With Customer Liability Current ContractWithCustomerLiabilityCurrent	25089000
CY2022Q1	rare	Percentage Of Gross Accounts Receivable Balance PercentageOfGrossAccountsReceivableBalance	0.61
CY2021Q4	rare	Percentage Of Gross Accounts Receivable Balance PercentageOfGrossAccountsReceivableBalance	0.71
CY2020Q4	rare	Liability Related To Sale Of Future Royalties LiabilityRelatedToSaleOfFutureRoyalties	335665000
CY2021	rare	Non Cash Collaboration Royalty Revenue NonCashCollaborationRoyaltyRevenue	17951000
CY2021	rare	Non Cash Interest Expense On Liability Related To Sale Of Future Royalties And Capitalized To Property Plant And Equipment NonCashInterestExpenseOnLiabilityRelatedToSaleOfFutureRoyaltiesAndCapitalizedToPropertyPlantAndEquipment	34072000
CY2021Q4	rare	Liability Related To Sale Of Future Royalties LiabilityRelatedToSaleOfFutureRoyalties	351786000
CY2022Q1	rare	Non Cash Collaboration Royalty Revenue NonCashCollaborationRoyaltyRevenue	4838000
CY2022Q1	rare	Non Cash Interest Expense On Liability Related To Sale Of Future Royalties And Capitalized To Property Plant And Equipment NonCashInterestExpenseOnLiabilityRelatedToSaleOfFutureRoyaltiesAndCapitalizedToPropertyPlantAndEquipment	8685000
CY2022Q1	rare	Liability Related To Sale Of Future Royalties LiabilityRelatedToSaleOfFutureRoyalties	355633000
CY2022Q1	us-gaap	Allocated Share Based Compensation Expense AllocatedShareBasedCompensationExpense	29387000
CY2021Q1	us-gaap	Allocated Share Based Compensation Expense AllocatedShareBasedCompensationExpense	24298000
CY2022Q1	us-gaap	Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount	9386957
CY2021Q1	us-gaap	Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount	8118221
CY2022Q1	rare	Stock Issued During Period Shares At Market Offering StockIssuedDuringPeriodSharesAtMarketOffering	1050372
CY2022Q1	rare	Proceeds From Issuance Of Common Stock At Market Offering ProceedsFromIssuanceOfCommonStockAtMarketOffering	78900000
CY2022Q1	us-gaap	Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax	-181000
CY2021Q4	us-gaap	Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax	-121000
CY2022Q1	us-gaap	Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax	-6373000
CY2021Q4	us-gaap	Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax	-1283000
CY2022Q1	us-gaap	Accumulated Other Comprehensive Income Loss Net Of Tax AccumulatedOtherComprehensiveIncomeLossNetOfTax	-6554000
CY2021Q4	us-gaap	Accumulated Other Comprehensive Income Loss Net Of Tax AccumulatedOtherComprehensiveIncomeLossNetOfTax	-1404000

Files In Submission

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0000950170-22-007940-index.html	Edgar Link	pending
0000950170-22-007940.txt	Edgar Link	pending
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rare-20220331_htm.xml	Edgar Link	completed
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rare-20220331_pre.xml	Edgar Link	unprocessable
rare-ex10_1.htm	Edgar Link	pending
rare-ex31_1.htm	Edgar Link	pending
rare-ex31_2.htm	Edgar Link	pending
rare-ex32_1.htm	Edgar Link	pending
report.css	Edgar Link	pending
Show.js	Edgar Link	pending

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Ultragenyx Pharmaceutical Inc. Stock $rare

2022 Q2 Form 10-Q Financial Statement

#000095017022007940 Filed on May 06, 2022

Income Statement

Balance Sheet

Cashflow Statement

Facts In Submission

Files In Submission

$RARE Ultragenyx Pharmaceutical Inc. Stock $rare

2022 Q2 Form 10-Q Financial Statement

#000095017022007940 Filed on May 06, 2022

Income Statement

Balance Sheet

Cashflow Statement

Facts In Submission

Files In Submission

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Ultragenyx Pharmaceutical Inc. Stock $rare