2022 Q3 Form 10-Q Financial Statement

#000095017022013411 Filed on July 29, 2022

View on sec.gov

Income Statement

Concept 2022 Q3 2022 Q2 2022 Q1
Revenue $90.70M $89.34M $79.94M
YoY Change 11.09% 2.72% -19.58%
Cost Of Revenue $8.631M $8.270M $6.100M
YoY Change 106.73% 163.71% 17.53%
Gross Profit $82.07M $81.07M $73.84M
YoY Change 5.94% -3.3% -21.63%
Gross Profit Margin 90.48% 90.74% 92.37%
Selling, General & Admin $69.84M $68.14M $67.31M
YoY Change 29.62% 27.57% 26.39%
% of Gross Profit 85.1% 84.04% 91.17%
Research & Development $237.3M $154.5M $143.2M
YoY Change 109.23% 36.5% -2.96%
% of Gross Profit 289.13% 190.6% 193.89%
Depreciation & Amortization $4.694M $4.481M $4.088M
YoY Change 45.91% 43.71% 21.41%
% of Gross Profit 5.72% 5.53% 5.54%
Operating Expenses $307.1M $222.7M $210.5M
YoY Change 83.59% 33.64% 4.83%
Operating Profit -$225.1M -$141.6M -$136.6M
YoY Change 150.55% 71.06% 28.21%
Interest Expense $1.857M -$9.285M -$8.835M
YoY Change -92.89% -69.66% -55.78%
% of Operating Profit
Other Income/Expense, Net -$1.105M -$930.0K $289.0K
YoY Change 166.27% 1288.06% -136.35%
Pretax Income -$238.8M -$157.9M -$151.8M
YoY Change 227.98% 29.43% 11.79%
Income Tax $6.287M $302.0K $558.0K
% Of Pretax Income
Net Earnings -$245.1M -$158.2M -$152.3M
YoY Change 235.77% 29.19% 11.88%
Net Earnings / Revenue -270.23% -177.03% -190.55%
Basic Earnings Per Share -$3.50 -$2.26 -$2.19
Diluted Earnings Per Share -$3.50 -$2.262M -$2.191M
COMMON SHARES
Basic Shares Outstanding 70.04M 69.93M 69.38M
Diluted Shares Outstanding 70.05M 69.93M 69.52M

Balance Sheet

Concept 2022 Q3 2022 Q2 2022 Q1
SHORT-TERM ASSETS
Cash & Short-Term Investments $888.7M $542.8M $598.3M
YoY Change 20.56% -35.82% -38.02%
Cash & Equivalents $391.7M $159.9M $154.0M
Short-Term Investments $497.0M $382.9M $444.3M
Other Short-Term Assets $70.98M $77.46M $63.02M
YoY Change 2.58% 21.16% -7.32%
Inventory $21.85M $21.08M $17.81M
Prepaid Expenses
Receivables $31.16M $37.21M $28.37M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $1.013B $678.5M $707.5M
YoY Change 19.58% -28.54% -33.93%
LONG-TERM ASSETS
Property, Plant & Equipment $234.6M $207.4M $177.1M
YoY Change 97.04% 44.83% 40.42%
Goodwill $44.41M $44.41M $44.41M
YoY Change 0.0%
Intangibles $158.4M $159.2M $160.0M
YoY Change 21.04%
Long-Term Investments $121.3M $178.7M $241.1M
YoY Change -58.22% -22.88% 11.42%
Other Assets $18.29M $21.14M $21.22M
YoY Change 11.36% 66.91% 138.4%
Total Long-Term Assets $605.8M $642.1M $676.5M
YoY Change -5.03% 14.06% 28.39%
TOTAL ASSETS
Total Short-Term Assets $1.013B $678.5M $707.5M
Total Long-Term Assets $605.8M $642.1M $676.5M
Total Assets $1.618B $1.321B $1.384B
YoY Change 9.0% -12.68% -13.38%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $23.53M $34.21M $23.69M
YoY Change 51.09% 57.01% 50.86%
Accrued Expenses $189.1M $170.4M $126.4M
YoY Change 72.4% 87.08% 52.79%
Deferred Revenue $4.437M $5.867M
YoY Change 13.42% -76.62%
Short-Term Debt $0.00 $0.00 $0.00
YoY Change
Long-Term Debt Due
YoY Change
Total Short-Term Liabilities $227.4M $220.6M $167.2M
YoY Change 67.26% 73.64% 31.68%
LONG-TERM LIABILITIES
Long-Term Debt $0.00 $0.00 $0.00
YoY Change
Other Long-Term Liabilities $4.050M $3.768M $3.392M
YoY Change 759.87% -99.01% -99.11%
Total Long-Term Liabilities $4.050M $3.768M $3.392M
YoY Change 759.87% -99.01% -99.11%
TOTAL LIABILITIES
Total Short-Term Liabilities $227.4M $220.6M $167.2M
Total Long-Term Liabilities $4.050M $3.768M $3.392M
Total Liabilities $1.150B $642.6M $587.6M
YoY Change 108.6% 18.93% 8.17%
SHAREHOLDERS EQUITY
Retained Earnings -$2.629B -$2.384B -$2.226B
YoY Change 34.75%
Common Stock $70.00K $70.00K $70.00K
YoY Change 2.94%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity $468.8M $678.1M $796.4M
YoY Change
Total Liabilities & Shareholders Equity $1.618B $1.321B $1.384B
YoY Change 9.0% -12.68% -13.38%

Cashflow Statement

Concept 2022 Q3 2022 Q2 2022 Q1
OPERATING ACTIVITIES
Net Income -$245.1M -$158.2M -$152.3M
YoY Change 235.77% 29.19% 11.88%
Depreciation, Depletion And Amortization $4.694M $4.481M $4.088M
YoY Change 45.91% 43.71% 21.41%
Cash From Operating Activities -$95.35M -$77.26M -$117.5M
YoY Change 59.84% 18.22% -26.25%
INVESTING ACTIVITIES
Capital Expenditures -$26.05M -$30.92M $32.18M
YoY Change 26.68% 54.04% 96.08%
Acquisitions
YoY Change
Other Investing Activities -$135.8M $110.1M -$2.578M
YoY Change 572.37% -619.91% -98.45%
Cash From Investing Activities -$161.9M $79.17M -$37.36M
YoY Change 297.11% -291.92% -79.52%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 491.9M 5.837M 1.612M
YoY Change 6414.93% -53.77% -87.26%
NET CHANGE
Cash From Operating Activities -95.35M -77.26M -117.5M
Cash From Investing Activities -161.9M 79.17M -37.36M
Cash From Financing Activities 491.9M 5.837M 1.612M
Net Change In Cash 234.7M 7.748M -153.4M
YoY Change -352.72% -108.24% -53.51%
FREE CASH FLOW
Cash From Operating Activities -$95.35M -$77.26M -$117.5M
Capital Expenditures -$26.05M -$30.92M $32.18M
Free Cash Flow -$69.30M -$46.33M -$149.7M
YoY Change 77.29% 2.33% -14.82%

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<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;">UX111 (formerly ABO-102) is an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease. In May 2022, the Company announced an exclusive license agreement with Abeona Therapeutics Inc. (Abeona) for UX111 whereby the Company assumed responsibility for the UX111 program, as further described in Note 6;</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;">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 developing through 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. In July 2022, the Company executed its option to acquire GeneTx as further described in Note 12;</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 adeno-associated virus 9, (AAV9) gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease; and</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 June 30, 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>
us-gaap Use Of Estimates
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<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>
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CY2021 rare Percentage Of Gross Accounts Receivables From Different Customer
PercentageOfGrossAccountsReceivablesFromDifferentCustomer
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