$RARE
Ultragenyx Pharmaceutical Inc.

2024 Q3 Form 10-Q Financial Statement

#000095017024089488 Filed on August 02, 2024

View on sec.gov

Income Statement

Concept 2024 Q3 2024 Q2
Revenue $139.5M $147.0M
YoY Change 42.27% 35.75%
Cost Of Revenue $21.02M $21.28M
YoY Change 91.1% 114.65%
Gross Profit $118.5M $125.7M
YoY Change 36.02% 27.8%
Gross Profit Margin 84.93% 85.53%
Selling, General & Admin $80.35M $80.60M
YoY Change 7.25% -0.98%
% of Gross Profit 67.82% 64.1%
Research & Development $170.1M $161.5M
YoY Change 8.18% -2.09%
% of Gross Profit 143.58% 128.44%
Depreciation & Amortization $8.757M $8.862M
YoY Change 21.79% 65.3%
% of Gross Profit 7.39% 7.05%
Operating Expenses $250.5M $242.1M
YoY Change 7.88% -1.72%
Operating Profit -$132.0M -$116.4M
YoY Change -9.04% -21.35%
Interest Expense $11.41M $3.410M
YoY Change 153.53% -45.22%
% of Operating Profit
Other Income/Expense, Net $3.077M -$1.829M
YoY Change -540.2% -89.47%
Pretax Income -$133.2M -$130.7M
YoY Change -16.22% -17.82%
Income Tax $303.0K $858.0K
% Of Pretax Income
Net Earnings -$133.5M -$131.6M
YoY Change -16.37% -17.66%
Net Earnings / Revenue -95.71% -89.51%
Basic Earnings Per Share -$1.40 -$1.52
Diluted Earnings Per Share -$1.40 -$1.52
COMMON SHARES
Basic Shares Outstanding 92.17M 83.13M
Diluted Shares Outstanding 95.49M 86.58M

Balance Sheet

Concept 2024 Q3 2024 Q2
SHORT-TERM ASSETS
Cash & Short-Term Investments $621.6M $763.8M
YoY Change 40.61% 38.38%
Cash & Equivalents $150.6M $480.7M
Short-Term Investments $471.0M $283.1M
Other Short-Term Assets $41.37M $54.70M
YoY Change -10.27% 6.47%
Inventory $43.33M $40.14M
Prepaid Expenses
Receivables $93.30M $106.0M
Other Receivables $0.00 $0.00
Total Short-Term Assets $799.6M $964.6M
YoY Change 33.45% 37.39%
LONG-TERM ASSETS
Property, Plant & Equipment $272.4M $278.7M
YoY Change -8.21% -9.57%
Goodwill $44.41M $44.41M
YoY Change 0.0% 0.0%
Intangibles $164.9M $166.2M
YoY Change 4.86% 5.06%
Long-Term Investments $203.1M $110.7M
YoY Change 135.87% 54.07%
Other Assets $53.87M $53.77M
YoY Change 93.12% 103.0%
Total Long-Term Assets $738.8M $653.8M
YoY Change 15.62% 7.33%
TOTAL ASSETS
Total Short-Term Assets $799.6M $964.6M
Total Long-Term Assets $738.8M $653.8M
Total Assets $1.538B $1.618B
YoY Change 24.25% 23.42%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $35.99M $59.81M
YoY Change 7.89% 42.01%
Accrued Expenses $185.0M $154.4M
YoY Change 3.59% -13.51%
Deferred Revenue
YoY Change
Short-Term Debt $0.00 $0.00
YoY Change
Long-Term Debt Due
YoY Change
Total Short-Term Liabilities $285.0M $272.9M
YoY Change 12.22% 11.66%
LONG-TERM LIABILITIES
Long-Term Debt $0.00 $0.00
YoY Change
Other Long-Term Liabilities $16.13M $15.20M
YoY Change 57.64% -98.32%
Total Long-Term Liabilities $16.13M $15.20M
YoY Change 57.64% -98.32%
TOTAL LIABILITIES
Total Short-Term Liabilities $285.0M $272.9M
Total Long-Term Liabilities $16.13M $15.20M
Total Liabilities $1.185B $1.186B
YoY Change -1.81% 0.32%
SHAREHOLDERS EQUITY
Retained Earnings -$3.823B -$3.690B
YoY Change 17.12% 18.85%
Common Stock $92.00K $92.00K
YoY Change 27.78% 29.58%
Preferred Stock
YoY Change
Treasury Stock (at cost) $3.555M $3.395M
YoY Change 833.07% 791.08%
Treasury Stock Shares
Shareholders Equity $346.8M $432.4M
YoY Change
Total Liabilities & Shareholders Equity $1.538B $1.618B
YoY Change 24.25% 23.42%

Cashflow Statement

Concept 2024 Q3 2024 Q2
OPERATING ACTIVITIES
Net Income -$133.5M -$131.6M
YoY Change -16.37% -17.66%
Depreciation, Depletion And Amortization $8.757M $8.862M
YoY Change 21.79% 65.3%
Cash From Operating Activities -$67.00M -$76.96M
YoY Change -43.16% -33.74%
INVESTING ACTIVITIES
Capital Expenditures $1.617M $4.509M
YoY Change -56.3% -72.57%
Acquisitions
YoY Change
Other Investing Activities -$273.9M $65.07M
YoY Change -502.83% -43.83%
Cash From Investing Activities -$275.5M $60.56M
YoY Change -528.46% -39.08%
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 384.9M
YoY Change -55.71% 1065.78%
NET CHANGE
Cash From Operating Activities -67.00M -76.96M
Cash From Investing Activities -275.5M 60.56M
Cash From Financing Activities 11.17M 384.9M
Net Change In Cash -331.3M 368.5M
YoY Change 1069.1% 2164.18%
FREE CASH FLOW
Cash From Operating Activities -$67.00M -$76.96M
Capital Expenditures $1.617M $4.509M
Free Cash Flow -$68.61M -$81.47M
YoY Change -43.56% -38.56%

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, 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%;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%;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%;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%;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%;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%;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 June 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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<p style="font-size:10pt;margin-top:13.5pt;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;">Use of Estimates</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></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 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.</span></p>
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255859000
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CY2022Q4 rare Liability For Sale Of Future Royalties
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59307000
CY2023 rare Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
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75435000
CY2023Q4 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
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rare Royalty Revenue
RoyaltyRevenue
34296000
rare Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
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31807000
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39363000
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AllocatedShareBasedCompensationExpense
34653000
us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
76297000
us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
66592000
CY2024Q2 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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17016900
CY2023Q2 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
14943528
us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
16043787
us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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13888375
CY2022Q3 rare Related Party Payment Of Contribution
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1000000
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P4Y
CY2024Q2 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
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-696000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-606000
CY2024Q2 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
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-399000
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1253000
CY2024Q2 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
-1095000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
647000

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