$RARE
Ultragenyx Pharmaceutical Inc.

2024 Q2 Form 10-Q Financial Statement

#000095017024052346 Filed on May 03, 2024

View on sec.gov

Income Statement

Concept 2024 Q2 2024 Q1 2023 Q1
Revenue $147.0M $108.8M $100.5M
YoY Change 35.75% 8.3% 25.72%
Cost Of Revenue $21.28M $17.53M $12.26M
YoY Change 114.65% 43.04% 100.93%
Gross Profit $125.7M $91.30M $88.24M
YoY Change 27.8% 3.47% 19.51%
Gross Profit Margin 85.53% 83.89% 87.8%
Selling, General & Admin $80.60M $78.16M $76.65M
YoY Change -0.98% 1.98% 13.87%
% of Gross Profit 64.1% 85.61% 86.86%
Research & Development $161.5M $178.5M $165.7M
YoY Change -2.09% 7.72% 15.75%
% of Gross Profit 128.44% 195.5% 187.78%
Depreciation & Amortization $8.862M $8.845M $5.236M
YoY Change 65.3% 68.93% 28.08%
% of Gross Profit 7.05% 9.69% 5.93%
Operating Expenses $242.1M $256.6M $242.3M
YoY Change -1.72% 5.9% 15.15%
Operating Profit -$116.4M -$165.3M -$154.1M
YoY Change -21.35% 7.3% 12.79%
Interest Expense $3.410M $12.57M $5.956M
YoY Change -45.22% 111.05% -167.41%
% of Operating Profit
Other Income/Expense, Net -$1.829M -$1.605M $308.0K
YoY Change -89.47% -621.1% 6.57%
Pretax Income -$130.7M -$170.2M -$163.5M
YoY Change -17.82% 4.13% 7.72%
Income Tax $858.0K $455.0K $495.0K
% Of Pretax Income
Net Earnings -$131.6M -$170.7M -$164.0M
YoY Change -17.66% 4.09% 7.65%
Net Earnings / Revenue -89.51% -156.83% -163.16%
Basic Earnings Per Share -$1.52 -$2.03 -$2.33
Diluted Earnings Per Share -$1.52 -$2.03 -$2.33
COMMON SHARES
Basic Shares Outstanding 83.13M 82.34M 70.22M
Diluted Shares Outstanding 86.58M 84.29M 70.37M

Balance Sheet

Concept 2024 Q2 2024 Q1 2023 Q1
SHORT-TERM ASSETS
Cash & Short-Term Investments $763.8M $412.1M $629.4M
YoY Change 38.38% -34.52% 5.19%
Cash & Equivalents $480.7M $112.3M $85.77M
Short-Term Investments $283.1M $299.8M $543.6M
Other Short-Term Assets $54.70M $47.73M $67.38M
YoY Change 6.47% -29.16% 6.91%
Inventory $40.14M $35.91M $26.62M
Prepaid Expenses
Receivables $106.0M $100.3M $42.99M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $964.6M $596.0M $766.3M
YoY Change 37.39% -22.23% 8.31%
LONG-TERM ASSETS
Property, Plant & Equipment $278.7M $285.1M $277.9M
YoY Change -9.57% 2.6% 56.93%
Goodwill $44.41M $44.41M $44.41M
YoY Change 0.0% 0.0% 0.0%
Intangibles $166.2M $165.0M $159.2M
YoY Change 5.06% 3.69% -0.51%
Long-Term Investments $110.7M $156.6M $90.41M
YoY Change 54.07% 73.19% -62.5%
Other Assets $53.77M $59.97M $22.26M
YoY Change 103.0% 169.42% 4.91%
Total Long-Term Assets $653.8M $711.1M $617.1M
YoY Change 7.33% 15.24% -8.79%
TOTAL ASSETS
Total Short-Term Assets $964.6M $596.0M $766.3M
Total Long-Term Assets $653.8M $711.1M $617.1M
Total Assets $1.618B $1.307B $1.383B
YoY Change 23.42% -5.52% -0.05%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $59.81M $40.97M $58.98M
YoY Change 42.01% -30.53% 149.02%
Accrued Expenses $154.4M $149.1M $144.4M
YoY Change -13.51% 3.27% 14.28%
Deferred Revenue
YoY Change
Short-Term Debt $0.00 $0.00 $0.00
YoY Change
Long-Term Debt Due
YoY Change
Total Short-Term Liabilities $272.9M $245.6M $218.2M
YoY Change 11.66% 12.56% 30.47%
LONG-TERM LIABILITIES
Long-Term Debt $0.00 $0.00 $0.00
YoY Change
Other Long-Term Liabilities $15.20M $14.48M $9.257M
YoY Change -98.32% 56.41% 172.91%
Total Long-Term Liabilities $15.20M $14.48M $9.257M
YoY Change -98.32% 56.41% 172.91%
TOTAL LIABILITIES
Total Short-Term Liabilities $272.9M $245.6M $218.2M
Total Long-Term Liabilities $15.20M $14.48M $9.257M
Total Liabilities $1.186B $1.167B $1.163B
YoY Change 0.32% 0.33% 97.91%
SHAREHOLDERS EQUITY
Retained Earnings -$3.690B -$3.558B -$2.945B
YoY Change 18.85% 20.83% 32.3%
Common Stock $92.00K $83.00K $71.00K
YoY Change 29.58% 16.9% 1.43%
Preferred Stock
YoY Change
Treasury Stock (at cost) $3.395M $3.332M $356.0K
YoY Change 791.08% 835.96%
Treasury Stock Shares $7.812K
Shareholders Equity $432.4M $140.3M $220.5M
YoY Change
Total Liabilities & Shareholders Equity $1.618B $1.307B $1.383B
YoY Change 23.42% -5.52% -0.05%

Cashflow Statement

Concept 2024 Q2 2024 Q1 2023 Q1
OPERATING ACTIVITIES
Net Income -$131.6M -$170.7M -$164.0M
YoY Change -17.66% 4.09% 7.65%
Depreciation, Depletion And Amortization $8.862M $8.845M $5.236M
YoY Change 65.3% 68.93% 28.08%
Cash From Operating Activities -$76.96M -$190.7M -$156.9M
YoY Change -33.74% 21.59% 33.48%
INVESTING ACTIVITIES
Capital Expenditures $4.509M $3.211M $25.03M
YoY Change -72.57% -87.17% -22.22%
Acquisitions
YoY Change
Other Investing Activities $65.07M $2.065M $3.951M
YoY Change -43.83% -47.73% -253.26%
Cash From Investing Activities $60.56M $94.44M $111.3M
YoY Change -39.08% -15.16% -397.91%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 384.9M -58.00K -722.0K
YoY Change 1065.78% -91.97% -144.79%
NET CHANGE
Cash From Operating Activities -76.96M -190.7M -156.9M
Cash From Investing Activities 60.56M 94.44M 111.3M
Cash From Financing Activities 384.9M -58.00K -722.0K
Net Change In Cash 368.5M -97.03M -46.07M
YoY Change 2164.18% 110.59% -69.96%
FREE CASH FLOW
Cash From Operating Activities -$76.96M -$190.7M -$156.9M
Capital Expenditures $4.509M $3.211M $25.03M
Free Cash Flow -$81.47M -$193.9M -$181.9M
YoY Change -38.56% 6.62% 21.51%

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CY2024Q1 us-gaap Nature Of Operations
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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 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 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 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 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 imperfect, or OI;</span></div></div><div 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 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 March 31, 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>
CY2024Q1 us-gaap Number Of Reportable Segments
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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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P4Y
CY2024Q1 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-477000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-606000
CY2024Q1 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
46000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
1253000
CY2024Q1 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
-431000
CY2023Q4 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
647000

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