2023 Q2 Form 10-Q Financial Statement

#000095017023018017 Filed on May 05, 2023

View on sec.gov

Income Statement

Concept 2023 Q2 2023 Q1 2022 Q4
Revenue $108.3M $100.5M $103.3M
YoY Change 21.23% 25.72% 23.93%
Cost Of Revenue $9.914M $12.26M $5.319M
YoY Change 19.88% 100.93% 51.58%
Gross Profit $98.40M $88.24M $98.03M
YoY Change 21.37% 19.51% 22.72%
Gross Profit Margin 90.85% 87.8% 94.85%
Selling, General & Admin $81.40M $76.65M $72.85M
YoY Change 19.47% 13.87% 22.58%
% of Gross Profit 82.73% 86.86% 74.31%
Research & Development $164.9M $165.7M $170.8M
YoY Change 6.74% 15.75% 38.85%
% of Gross Profit 167.64% 187.78% 174.24%
Depreciation & Amortization $5.361M $5.236M $4.957M
YoY Change 19.64% 28.08% 40.27%
% of Gross Profit 5.45% 5.93% 5.06%
Operating Expenses $246.4M $242.3M $243.7M
YoY Change 10.64% 15.15% 33.55%
Operating Profit -$148.0M -$154.1M -$145.6M
YoY Change 4.49% 12.79% 41.99%
Interest Expense $6.225M $5.956M $8.038M
YoY Change -167.04% -167.41% -151.33%
% of Operating Profit
Other Income/Expense, Net -$17.36M $308.0K -$15.69M
YoY Change 1767.1% 6.57% 272.43%
Pretax Income -$159.1M -$163.5M -$153.3M
YoY Change 0.78% 7.72% 25.19%
Income Tax $732.0K $495.0K -$1.451M
% Of Pretax Income
Net Earnings -$159.8M -$164.0M -$151.8M
YoY Change 1.05% 7.65% 23.99%
Net Earnings / Revenue -147.57% -163.16% -146.91%
Basic Earnings Per Share -$2.25 -$2.33
Diluted Earnings Per Share -$2.25 -$2.33 -$2.16
COMMON SHARES
Basic Shares Outstanding 70.81M 70.22M 70.11M
Diluted Shares Outstanding 70.90M 70.37M

Balance Sheet

Concept 2023 Q2 2023 Q1 2022 Q4
SHORT-TERM ASSETS
Cash & Short-Term Investments $552.0M $629.4M $747.8M
YoY Change 1.69% 5.19% 1.02%
Cash & Equivalents $102.1M $85.77M $132.9M
Short-Term Investments $449.9M $543.6M $614.8M
Other Short-Term Assets $51.38M $67.38M $68.93M
YoY Change -33.67% 6.91% -3.93%
Inventory $28.26M $26.62M $26.77M
Prepaid Expenses
Receivables $70.52M $42.99M $40.45M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $702.1M $766.3M $883.9M
YoY Change 3.48% 8.31% 3.19%
LONG-TERM ASSETS
Property, Plant & Equipment $308.2M $277.9M $259.7M
YoY Change 48.62% 56.93% 83.88%
Goodwill $44.41M $44.41M $44.41M
YoY Change 0.0% 0.0% 0.0%
Intangibles $158.2M $159.2M $160.1M
YoY Change -0.6% -0.51% 22.42%
Long-Term Investments $71.83M $90.41M $154.5M
YoY Change -59.81% -62.5% -47.42%
Other Assets $26.49M $22.26M $16.85M
YoY Change 25.28% 4.91% -18.06%
Total Long-Term Assets $609.2M $617.1M $661.5M
YoY Change -5.13% -8.79% -0.64%
TOTAL ASSETS
Total Short-Term Assets $702.1M $766.3M $883.9M
Total Long-Term Assets $609.2M $617.1M $661.5M
Total Assets $1.311B $1.383B $1.545B
YoY Change -0.71% -0.05% 1.51%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $42.12M $58.98M $43.27M
YoY Change 23.1% 149.02% 152.5%
Accrued Expenses $178.5M $144.4M $204.7M
YoY Change 4.8% 14.28% 40.62%
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 $244.4M $218.2M $261.2M
YoY Change 10.76% 30.47% 44.02%
LONG-TERM LIABILITIES
Long-Term Debt $0.00 $0.00 $0.00
YoY Change -100.0%
Other Long-Term Liabilities $906.2M $9.257M $4.820M
YoY Change 23950.8% 172.91% 379.6%
Total Long-Term Liabilities $906.2M $9.257M $4.820M
YoY Change 23950.8% 172.91% 379.6%
TOTAL LIABILITIES
Total Short-Term Liabilities $244.4M $218.2M $261.2M
Total Long-Term Liabilities $906.2M $9.257M $4.820M
Total Liabilities $1.182B $1.163B $1.193B
YoY Change 84.0% 97.91% 98.88%
SHAREHOLDERS EQUITY
Retained Earnings -$3.105B -$2.945B -$2.781B
YoY Change 30.23% 32.3% 34.12%
Common Stock $71.00K $71.00K $70.00K
YoY Change 1.43% 1.43% 1.45%
Preferred Stock
YoY Change
Treasury Stock (at cost) $381.0K $356.0K $0.00
YoY Change
Treasury Stock Shares $7.812K $0.00
Shareholders Equity $129.0M $220.5M $352.5M
YoY Change
Total Liabilities & Shareholders Equity $1.311B $1.383B $1.545B
YoY Change -0.71% -0.05% 1.51%

Cashflow Statement

Concept 2023 Q2 2023 Q1 2022 Q4
OPERATING ACTIVITIES
Net Income -$159.8M -$164.0M -$151.8M
YoY Change 1.05% 7.65% 23.99%
Depreciation, Depletion And Amortization $5.361M $5.236M $4.957M
YoY Change 19.64% 28.08% 40.27%
Cash From Operating Activities -$116.2M -$156.9M -$90.34M
YoY Change 50.36% 33.48% 66.24%
INVESTING ACTIVITIES
Capital Expenditures $16.44M $25.03M $26.97M
YoY Change -153.16% -22.22% -268.06%
Acquisitions
YoY Change
Other Investing Activities $115.9M $3.951M -$144.6M
YoY Change 5.24% -253.26% -269.94%
Cash From Investing Activities $99.42M $111.3M -$171.6M
YoY Change 25.58% -397.91% -348.49%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 33.02M -722.0K 1.882M
YoY Change 465.7% -144.79% -97.8%
NET CHANGE
Cash From Operating Activities -116.2M -156.9M -90.34M
Cash From Investing Activities 99.42M 111.3M -171.6M
Cash From Financing Activities 33.02M -722.0K 1.882M
Net Change In Cash 16.28M -46.07M -260.1M
YoY Change 110.08% -69.96% -358.92%
FREE CASH FLOW
Cash From Operating Activities -$116.2M -$156.9M -$90.34M
Capital Expenditures $16.44M $25.03M $26.97M
Free Cash Flow -$132.6M -$181.9M -$117.3M
YoY Change 186.18% 21.51% 206.33%

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<p style="text-indent:-4.749%;padding-left:4.533%;font-size:10.0pt;margin-top:0.0pt;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;">1. Organization</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, 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:5.0%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;">The </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:&quot;Calibri&quot;, 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 ultra-rare genetic diseases. The Company operates as </span><span style="font-size:10.0pt;font-family:&quot;Calibri&quot;, sans-serif;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:&quot;Calibri&quot;, sans-serif;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;"> reportable segment and has four commercially approved products.</span></p><p style="text-indent:5.0%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, 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:5.0%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, 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><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:&quot;Calibri&quot;, sans-serif;min-width:fit-content;"> </span></p><p style="text-indent:5.0%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, 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><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:&quot;Calibri&quot;, sans-serif;min-width:fit-content;"> </span></p><p style="text-indent:5.0%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, 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:5.0%;font-size:10.0pt;margin-top:4.5pt;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:&quot;Calibri&quot;, 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:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:&quot;Calibri&quot;, sans-serif;min-width:fit-content;"> </span></p><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, 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:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, 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:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, 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:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;">UX143 (setrusumab), which is subject to the Company</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:8.0pt;font-family:&quot;Calibri&quot;, sans-serif;min-width:fit-content;">’</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:&quot;Calibri&quot;, 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:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, 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;</span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, 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; and</span></div></div><div style="margin-left:3.333%;text-indent:0.0%;display:flex;margin-top:6.0pt;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:&quot;Times New Roman&quot;, serif;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;">UX053 is a messenger RNA, or mRNA, product candidate designed for the treatment of patients with Glycogen Storage Disease Type III, or GSDIII, a disease caused by a glycogen debranching enzyme, or 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;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:&quot;Calibri&quot;, 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’s ultimate success depends on the outcome of its research and development and commercialization activities. Management recognizes that the Company will likely need to raise additional capital to fully implement its business plans. Through March 31, 2023, 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;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;">The Company expects it will need to 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>
CY2023Q1 us-gaap Number Of Reportable Segments
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CY2023Q1 us-gaap Use Of Estimates
UseOfEstimates
<p style="text-indent:0.0%;font-size:10.0pt;margin-top:13.5pt;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:&quot;Calibri&quot;, sans-serif;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:&quot;Calibri&quot;, sans-serif;min-width:fit-content;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.0pt;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:&quot;Calibri&quot;, 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><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:&quot;Calibri&quot;, sans-serif;min-width:fit-content;"> </span></p>
CY2023Q1 us-gaap Cash And Cash Equivalents At Carrying Value
CashAndCashEquivalentsAtCarryingValue
85768000
CY2022Q1 us-gaap Cash And Cash Equivalents At Carrying Value
CashAndCashEquivalentsAtCarryingValue
153988000
CY2023Q1 us-gaap Restricted Cash Current
RestrictedCashCurrent
1964000
CY2022Q1 us-gaap Restricted Cash Current
RestrictedCashCurrent
217000
CY2023Q1 us-gaap Restricted Cash Noncurrent
RestrictedCashNoncurrent
3795000
CY2022Q1 us-gaap Restricted Cash Noncurrent
RestrictedCashNoncurrent
2001000
CY2023Q1 us-gaap Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations
CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations
91527000
CY2022Q1 us-gaap Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations
CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations
156206000
CY2023Q1 us-gaap Inventory Finished Goods
InventoryFinishedGoods
11525000
CY2022Q4 us-gaap Inventory Finished Goods
InventoryFinishedGoods
9280000
CY2023Q1 us-gaap Investments Fair Value Disclosure
InvestmentsFairValueDisclosure
701004000
CY2022Q4 us-gaap Investments Fair Value Disclosure
InvestmentsFairValueDisclosure
884017000
CY2023Q1 us-gaap Available For Sale Debt Securities Amortized Cost Basis
AvailableForSaleDebtSecuritiesAmortizedCostBasis
693251000
CY2023Q1 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
723000
CY2023Q1 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
3989000
CY2023Q1 us-gaap Available For Sale Securities Debt Securities
AvailableForSaleSecuritiesDebtSecurities
689985000
CY2022Q4 us-gaap Available For Sale Debt Securities Amortized Cost Basis
AvailableForSaleDebtSecuritiesAmortizedCostBasis
882363000
CY2022Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
407000
CY2022Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
6135000
CY2022Q4 us-gaap Available For Sale Securities Debt Securities
AvailableForSaleSecuritiesDebtSecurities
876635000
CY2023Q1 us-gaap Inventory Work In Process
InventoryWorkInProcess
15091000
CY2022Q4 us-gaap Inventory Work In Process
InventoryWorkInProcess
17486000
CY2022Q4 us-gaap Inventory Net
InventoryNet
26766000
CY2023Q1 rare Research Clinical Study And Manufacturing Expenses
ResearchClinicalStudyAndManufacturingExpenses
42725000
CY2022Q4 rare Research Clinical Study And Manufacturing Expenses
ResearchClinicalStudyAndManufacturingExpenses
73558000
CY2023Q1 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
55796000
CY2022Q4 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
78938000
CY2023Q1 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
45883000
CY2022Q4 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
52182000
CY2023Q1 us-gaap Accrued Liabilities Current
AccruedLiabilitiesCurrent
144404000
CY2022Q4 us-gaap Accrued Liabilities Current
AccruedLiabilitiesCurrent
204678000
CY2023Q1 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
100496000
CY2022Q1 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
79935000
CY2023Q1 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
100496000
CY2022Q1 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
79935000
CY2022Q4 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
1479000
CY2021Q4 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
9076000
CY2023Q1 rare Contract With Customer Liability Additions During Period
ContractWithCustomerLiabilityAdditionsDuringPeriod
0
CY2022Q1 rare Contract With Customer Liability Additions During Period
ContractWithCustomerLiabilityAdditionsDuringPeriod
40000
CY2023Q1 rare Contract With Customer Liability Deductions During Period
ContractWithCustomerLiabilityDeductionsDuringPeriod
1479000
CY2022Q1 rare Contract With Customer Liability Deductions During Period
ContractWithCustomerLiabilityDeductionsDuringPeriod
3249000
CY2023Q1 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
0
CY2022Q1 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
5867000
CY2021Q4 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
351786000
CY2022 rare Proceeds From Royalties Received Financing Activities
ProceedsFromRoyaltiesReceivedFinancingActivities
490950000
CY2022 rare Royalty Revenue
RoyaltyRevenue
-21692000
CY2022 rare Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
NonCashInterestExpenseOnLiabilityForSaleOfFutureRoyaltiesAndCapitalizedToPropertyPlantAndEquipment
54395000
CY2022Q4 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
875439000
CY2023Q1 rare Royalty Revenue
RoyaltyRevenue
-4882000
CY2023Q1 rare Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
NonCashInterestExpenseOnLiabilityForSaleOfFutureRoyaltiesAndCapitalizedToPropertyPlantAndEquipment
19456000
CY2023Q1 rare Liability For Sale Of Future Royalties
LiabilityForSaleOfFutureRoyalties
890013000
CY2023Q1 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
31939000
CY2022Q1 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
29387000
CY2023Q1 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
12923444
CY2022Q1 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
9386957
CY2023Q1 rare Stock Issued During Period Shares At Market Offering
StockIssuedDuringPeriodSharesAtMarketOffering
1050372
CY2023Q1 rare Proceeds From Issuance Of Common Stock At Market Offering
ProceedsFromIssuanceOfCommonStockAtMarketOffering
78900000
CY2023Q1 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-701000
CY2022Q4 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
-845000
CY2023Q1 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
-3266000
CY2022Q4 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
-5728000
CY2023Q1 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
-3967000
CY2022Q4 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
-6573000

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