$RARE
Ultragenyx Pharmaceutical Inc.

2024 Q4 Form 10-Q Financial Statement

#000095017024121679 Filed on November 06, 2024

View on sec.gov

Income Statement

Concept 2024 Q4 2024 Q3
Revenue $139.5M
YoY Change 42.27%
Cost Of Revenue $21.02M
YoY Change 91.1%
Gross Profit $118.5M
YoY Change 36.02%
Gross Profit Margin 84.93%
Selling, General & Admin $80.35M
YoY Change 7.25%
% of Gross Profit 67.82%
Research & Development $170.1M
YoY Change 8.18%
% of Gross Profit 143.58%
Depreciation & Amortization $8.757M
YoY Change 21.79%
% of Gross Profit 7.39%
Operating Expenses $250.5M
YoY Change 7.88%
Operating Profit -$132.0M
YoY Change -9.04%
Interest Expense $11.41M
YoY Change 153.53%
% of Operating Profit
Other Income/Expense, Net $3.077M
YoY Change -540.2%
Pretax Income -$133.2M
YoY Change -16.22%
Income Tax $303.0K
% Of Pretax Income
Net Earnings -$133.5M
YoY Change -16.37%
Net Earnings / Revenue -95.71%
Basic Earnings Per Share -$1.40
Diluted Earnings Per Share -$1.40
COMMON SHARES
Basic Shares Outstanding 92.34M 92.17M
Diluted Shares Outstanding 95.49M

Balance Sheet

Concept 2024 Q4 2024 Q3
SHORT-TERM ASSETS
Cash & Short-Term Investments $621.6M
YoY Change 40.61%
Cash & Equivalents $150.6M
Short-Term Investments $471.0M
Other Short-Term Assets $41.37M
YoY Change -10.27%
Inventory $43.33M
Prepaid Expenses
Receivables $93.30M
Other Receivables $0.00
Total Short-Term Assets $799.6M
YoY Change 33.45%
LONG-TERM ASSETS
Property, Plant & Equipment $272.4M
YoY Change -8.21%
Goodwill $44.41M
YoY Change 0.0%
Intangibles $164.9M
YoY Change 4.86%
Long-Term Investments $203.1M
YoY Change 135.87%
Other Assets $53.87M
YoY Change 93.12%
Total Long-Term Assets $738.8M
YoY Change 15.62%
TOTAL ASSETS
Total Short-Term Assets $799.6M
Total Long-Term Assets $738.8M
Total Assets $1.538B
YoY Change 24.25%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $35.99M
YoY Change 7.89%
Accrued Expenses $185.0M
YoY Change 3.59%
Deferred Revenue
YoY Change
Short-Term Debt $0.00
YoY Change
Long-Term Debt Due
YoY Change
Total Short-Term Liabilities $285.0M
YoY Change 12.22%
LONG-TERM LIABILITIES
Long-Term Debt $0.00
YoY Change
Other Long-Term Liabilities $16.13M
YoY Change 57.64%
Total Long-Term Liabilities $16.13M
YoY Change 57.64%
TOTAL LIABILITIES
Total Short-Term Liabilities $285.0M
Total Long-Term Liabilities $16.13M
Total Liabilities $1.185B
YoY Change -1.81%
SHAREHOLDERS EQUITY
Retained Earnings -$3.823B
YoY Change 17.12%
Common Stock $92.00K
YoY Change 27.78%
Preferred Stock
YoY Change
Treasury Stock (at cost) $3.555M
YoY Change 833.07%
Treasury Stock Shares
Shareholders Equity $346.8M
YoY Change
Total Liabilities & Shareholders Equity $1.538B
YoY Change 24.25%

Cashflow Statement

Concept 2024 Q4 2024 Q3
OPERATING ACTIVITIES
Net Income -$133.5M
YoY Change -16.37%
Depreciation, Depletion And Amortization $8.757M
YoY Change 21.79%
Cash From Operating Activities -$67.00M
YoY Change -43.16%
INVESTING ACTIVITIES
Capital Expenditures $1.617M
YoY Change -56.3%
Acquisitions
YoY Change
Other Investing Activities -$273.9M
YoY Change -502.83%
Cash From Investing Activities -$275.5M
YoY Change -528.46%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 11.17M
YoY Change -55.71%
NET CHANGE
Cash From Operating Activities -67.00M
Cash From Investing Activities -275.5M
Cash From Financing Activities 11.17M
Net Change In Cash -331.3M
YoY Change 1069.1%
FREE CASH FLOW
Cash From Operating Activities -$67.00M
Capital Expenditures $1.617M
Free Cash Flow -$68.61M
YoY Change -43.56%

Facts In Submission

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<p style="text-indent:-4.749%;padding-left:4.533%;font-size:10pt;margin-top:0;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-weight:bold;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">1. Organization</span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">Ultragenyx Pharmaceutical Inc., or the Company, is a biopharmaceutical company incorporated in Delaware.</span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">The </span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. The Company operates as </span><span style="font-size:10pt;font-family:'Calibri',sans-serif;color:#000000;white-space:pre-wrap;min-width:fit-content;">one</span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;"> reportable segment and has four commercially approved products.</span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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.</span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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.</span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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.</span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">Evkeeza® (evinacumab) is approved in the U.S. and the European Economic Area, or EEA, and Japan for the treatment of homozygous familial hypercholesterolemia, or HoFH. The Company has exclusive rights to commercialize Evkeeza® (evinacumab) outside of the U.S.</span></p><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">In addition to the approved products, the Company has the following ongoing clinical development programs:</span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;"> </span></p><div class="item-list-element-wrapper" style="margin-left:4.528%;display:flex;margin-top:6pt;justify-content:flex-start;align-items:baseline;margin-bottom:0;min-width:4.537%;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:Times New Roman;min-width:4.7521786492374725%;word-break:keep-all;display:inline-flex;justify-content:flex-start;">•</span><div style="width:100%;display:inline;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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;</span></div></div><div class="item-list-element-wrapper" style="margin-left:4.528%;display:flex;margin-top:6pt;justify-content:flex-start;align-items:baseline;margin-bottom:0;min-width:4.537%;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:Times New Roman;min-width:4.7521786492374725%;word-break:keep-all;display:inline-flex;justify-content:flex-start;">•</span><div style="width:100%;display:inline;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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;</span></div></div><div class="item-list-element-wrapper" style="margin-left:4.528%;display:flex;margin-top:6pt;justify-content:flex-start;align-items:baseline;margin-bottom:0;min-width:4.537%;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:Times New Roman;min-width:4.7521786492374725%;word-break:keep-all;display:inline-flex;justify-content:flex-start;">•</span><div style="width:100%;display:inline;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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;</span></div></div><div class="item-list-element-wrapper" style="margin-left:4.528%;display:flex;margin-top:6pt;justify-content:flex-start;align-items:baseline;margin-bottom:0;min-width:4.537%;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:Times New Roman;min-width:4.7521786492374725%;word-break:keep-all;display:inline-flex;justify-content:flex-start;">•</span><div style="width:100%;display:inline;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">UX143 (setrusumab), which is subject to the Company</span><span style="color:#000000;white-space:pre-wrap;font-weight:bold;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">’</span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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 Imperfecta, or OI;</span></div></div><div class="item-list-element-wrapper" style="margin-left:4.528%;display:flex;margin-top:6pt;justify-content:flex-start;align-items:baseline;margin-bottom:0;min-width:4.537%;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:Times New Roman;min-width:4.7521786492374725%;word-break:keep-all;display:inline-flex;justify-content:flex-start;">•</span><div style="width:100%;display:inline;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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; and</span></div></div><div class="item-list-element-wrapper" style="margin-left:4.528%;display:flex;margin-top:6pt;justify-content:flex-start;align-items:baseline;margin-bottom:0;min-width:4.537%;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:Times New Roman;min-width:4.7521786492374725%;word-break:keep-all;display:inline-flex;justify-content:flex-start;">•</span><div style="width:100%;display:inline;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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.</span></div></div><p style="text-indent:4.533%;font-size:10pt;margin-top:4.5pt;font-family:Times New Roman;margin-bottom:0;text-align:left;"><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">The Company has sustained operating losses and expects such annual losses to continue over the next several years. The Company</span><span style="color:#000000;white-space:pre-wrap;font-weight:bold;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">’</span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">s ultimate success depends on the outcome of its research and development and commercialization activities. Through September 30, 2024, </span><span style="color:#000000;white-space:pre-wrap;font-size:10pt;font-family:'Calibri',sans-serif;min-width:fit-content;">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 may need to raise additional capital to fully implement its business plans 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>
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AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
1693000
CY2023Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
440000
CY2023Q4 us-gaap Available For Sale Securities Debt Securities
AvailableForSaleSecuritiesDebtSecurities
735815000
CY2024Q3 us-gaap Inventory Work In Process
InventoryWorkInProcess
20281000
CY2023Q4 us-gaap Inventory Work In Process
InventoryWorkInProcess
18859000
CY2024Q3 us-gaap Inventory Finished Goods
InventoryFinishedGoods
23053000
CY2023Q4 us-gaap Inventory Finished Goods
InventoryFinishedGoods
15110000
CY2024Q3 us-gaap Inventory Net
InventoryNet
43334000
CY2023Q4 us-gaap Inventory Net
InventoryNet
33969000
CY2024Q3 rare Research Clinical Study And Manufacturing Expenses
ResearchClinicalStudyAndManufacturingExpenses
39752000
CY2023Q4 rare Research Clinical Study And Manufacturing Expenses
ResearchClinicalStudyAndManufacturingExpenses
65326000
CY2024Q3 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
82924000
CY2023Q4 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
82936000
CY2024Q3 us-gaap Allowance For Doubtful Accounts Receivable
AllowanceForDoubtfulAccountsReceivable
32928000
CY2023Q4 us-gaap Allowance For Doubtful Accounts Receivable
AllowanceForDoubtfulAccountsReceivable
17029000
CY2024Q3 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
29424000
CY2023Q4 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
31195000
CY2024Q3 us-gaap Accrued Liabilities Current
AccruedLiabilitiesCurrent
185028000
CY2023Q4 us-gaap Accrued Liabilities Current
AccruedLiabilitiesCurrent
196486000
CY2024Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
139494000
CY2023Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
98052000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
395353000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
306857000
CY2024Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
139494000
CY2023Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
98052000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
395353000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
306857000
CY2023Q4 us-gaap Temporary Equity Carrying Amount Including Portion Attributable To Noncontrolling Interests
TemporaryEquityCarryingAmountIncludingPortionAttributableToNoncontrollingInterests
0
us-gaap Noncontrolling Interest Increase From Subsidiary Equity Issuance
NoncontrollingInterestIncreaseFromSubsidiaryEquityIssuance
7000000
CY2024Q3 us-gaap Temporary Equity Carrying Amount Including Portion Attributable To Noncontrolling Interests
TemporaryEquityCarryingAmountIncludingPortionAttributableToNoncontrollingInterests
7000000
CY2022Q4 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
875439000
CY2023 rare Royalty Revenue
RoyaltyRevenue
59307000
CY2023 rare Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
NonCashInterestExpenseOnLiabilityForSaleOfFutureRoyaltiesAndCapitalizedToPropertyPlantAndEquipment
75435000
CY2023Q4 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
891567000
rare Royalty Revenue
RoyaltyRevenue
60669000
rare Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
NonCashInterestExpenseOnLiabilityForSaleOfFutureRoyaltiesAndCapitalizedToPropertyPlantAndEquipment
47519000
CY2024Q3 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
878417000
CY2024Q3 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
41569000
CY2023Q3 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
34877000
us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
117866000
us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
101469000
CY2024Q3 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
16732694
CY2023Q3 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
14651749
us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
16271672
us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
14154897
CY2022Q3 rare Total Contract Value Of Related Party Contribution
TotalContractValueOfRelatedPartyContribution
1000000
CY2022Q3 rare Related Party Transaction Contribution Period
RelatedPartyTransactionContributionPeriod
P4Y
CY2024Q3 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-2177000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-606000
CY2024Q3 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
3073000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
1253000
CY2024Q3 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
896000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
647000
CY2024Q3 ecd Trd Arr Adoption Date
TrdArrAdoptionDate
September 9, 2024
CY2024Q3 ecd Trd Arr Ind Name
TrdArrIndName
Michael Narachi
CY2024Q3 ecd Trd Arr Ind Title
TrdArrIndTitle
member of the Company’s Board of Directors
CY2024Q3 ecd Rule10b51 Arr Adopted Flag
Rule10b51ArrAdoptedFlag
true
CY2024Q3 ecd Trd Arr Securities Agg Avail Amt
TrdArrSecuritiesAggAvailAmt
16250
CY2024Q3 ecd Trd Arr Expiration Date
TrdArrExpirationDate
June 17, 2025

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