$TVTX
Travere Therapeutics Inc

2015 Q4 Form 10-K Financial Statement

#000162828016011857 Filed on February 26, 2016

View on sec.gov

Income Statement

Concept 2015 Q4 2014 Q4 2014 Q3
Revenue $30.45M $14.09M $8.348M
YoY Change 116.17%
Cost Of Revenue $760.0K $340.0K $198.0K
YoY Change 123.53%
Gross Profit $29.69M $13.75M $8.150M
YoY Change 115.93%
Gross Profit Margin 97.51% 97.62% 97.63%
Selling, General & Admin $18.95M $18.46M $17.37M
YoY Change 2.65% 152.88% 350.05%
% of Gross Profit 63.83% 134.25% 213.15%
Research & Development $15.37M $14.19M $12.65M
YoY Change 8.32% 185.51% 803.29%
% of Gross Profit 51.77% 103.2% 155.17%
Depreciation & Amortization $3.970M $1.860M $1.850M
YoY Change 113.44% 3000.0% 3600.0%
% of Gross Profit 13.37% 13.53% 22.7%
Operating Expenses $45.65M $32.78M $30.22M
YoY Change 39.26% 176.12% 401.01%
Operating Profit -$15.20M -$18.70M -$21.87M
YoY Change -18.68% 57.49% 262.59%
Interest Expense -$4.210M -$10.33M -$2.629M
YoY Change -59.24% 549.69% -54.2%
% of Operating Profit
Other Income/Expense, Net $2.210M -$10.33M $3.887M
YoY Change -121.39% 1081.52% -165.0%
Pretax Income -$12.99M -$29.03M -$17.98M
YoY Change -55.23% 127.73% 49.69%
Income Tax -$10.53M $0.00 $0.00
% Of Pretax Income
Net Earnings -$2.469M -$29.03M -$17.98M
YoY Change -91.49% 126.38% 49.69%
Net Earnings / Revenue -8.11% -206.08% -215.38%
Basic Earnings Per Share -$0.07 -$1.10 -$0.67
Diluted Earnings Per Share -$0.14 -$1.10 -$0.84
COMMON SHARES
Basic Shares Outstanding 36.15M shares 26.68M shares
Diluted Shares Outstanding 28.21M shares

Balance Sheet

Concept 2015 Q4 2014 Q4 2014 Q3
SHORT-TERM ASSETS
Cash & Short-Term Investments $229.6M $27.76M $37.80M
YoY Change 727.1% 355.08% 130.49%
Cash & Equivalents $37.81M $18.20M $25.86M
Short-Term Investments $191.8M $9.556M $11.90M
Other Short-Term Assets $10.50M $800.0K $2.300M
YoY Change 1212.5% -42.86% 360.0%
Inventory $2.536M $801.0K $700.0K
Prepaid Expenses
Receivables $12.46M $7.960M $4.700M
Other Receivables $46.80M $0.00 $0.00
Total Short-Term Assets $301.9M $37.33M $45.40M
YoY Change 708.73% 397.7% 170.24%
LONG-TERM ASSETS
Property, Plant & Equipment $428.0K $671.0K $600.0K
YoY Change -36.21% 426.58%
Goodwill $936.0K $936.0K
YoY Change 0.0%
Intangibles $161.5M
YoY Change
Long-Term Investments $400.0K
YoY Change
Other Assets $1.859M $2.265M $2.300M
YoY Change -17.92% 655.0% -4.17%
Total Long-Term Assets $210.3M $98.14M $100.5M
YoY Change 114.32% 655.04% 2084.78%
TOTAL ASSETS
Total Short-Term Assets $301.9M $37.33M $45.40M
Total Long-Term Assets $210.3M $98.14M $100.5M
Total Assets $512.3M $135.5M $145.9M
YoY Change 278.14% 560.87% 581.78%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $7.639M $7.124M $11.30M
YoY Change 7.23% 100.47% 232.35%
Accrued Expenses $23.82M $27.88M $11.70M
YoY Change -14.57% 471.21% 680.0%
Deferred Revenue
YoY Change
Short-Term Debt $0.00 $40.50M $0.00
YoY Change -100.0%
Long-Term Debt Due $1.000M $1.500M
YoY Change -37.5%
Total Short-Term Liabilities $86.98M $107.5M $49.10M
YoY Change -19.12% 194.1% 80.51%
LONG-TERM LIABILITIES
Long-Term Debt $43.80M $43.30M $43.10M
YoY Change 1.15%
Other Long-Term Liabilities $3.066M $1.617M $63.70M
YoY Change 89.61% 61.7%
Total Long-Term Liabilities $3.066M $1.617M $106.8M
YoY Change 89.61% 61.7%
TOTAL LIABILITIES
Total Short-Term Liabilities $86.98M $107.5M $49.10M
Total Long-Term Liabilities $3.066M $1.617M $106.8M
Total Liabilities $212.3M $172.7M $156.1M
YoY Change 22.91% 330.02% 473.9%
SHAREHOLDERS EQUITY
Retained Earnings -$65.15M -$179.2M
YoY Change -63.64% 162.58%
Common Stock $365.8M $140.9M
YoY Change 159.71% 7593107.55%
Preferred Stock
YoY Change
Treasury Stock (at cost) $0.00 $3.215M
YoY Change -100.0% 235.85%
Treasury Stock Shares 0.000 shares 379.6K shares
Shareholders Equity $300.0M -$37.25M -$10.20M
YoY Change
Total Liabilities & Shareholders Equity $512.3M $135.5M $145.9M
YoY Change 278.14% 560.87% 581.78%

Cashflow Statement

Concept 2015 Q4 2014 Q4 2014 Q3
OPERATING ACTIVITIES
Net Income -$2.469M -$29.03M -$17.98M
YoY Change -91.49% 126.38% 49.69%
Depreciation, Depletion And Amortization $3.970M $1.860M $1.850M
YoY Change 113.44% 3000.0% 3600.0%
Cash From Operating Activities -$3.550M -$9.090M -$15.50M
YoY Change -60.95% 11.53% 484.91%
INVESTING ACTIVITIES
Capital Expenditures -$1.720M -$80.00K -$150.0K
YoY Change 2050.0% -33.33% 1400.0%
Acquisitions
YoY Change
Other Investing Activities -$98.74M $2.050M -$1.900M
YoY Change -4916.59% 48.55% -64.42%
Cash From Investing Activities -$100.5M $1.970M -$2.060M
YoY Change -5199.49% 56.35% -61.5%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 1.230M -530.0K 3.540M
YoY Change -332.08% 0.0% -83.25%
NET CHANGE
Cash From Operating Activities -3.550M -9.090M -15.50M
Cash From Investing Activities -100.5M 1.970M -2.060M
Cash From Financing Activities 1.230M -530.0K 3.540M
Net Change In Cash -102.8M -7.650M -14.02M
YoY Change 1243.53% 3.1% -206.78%
FREE CASH FLOW
Cash From Operating Activities -$3.550M -$9.090M -$15.50M
Capital Expenditures -$1.720M -$80.00K -$150.0K
Free Cash Flow -$1.830M -$9.010M -$15.35M
YoY Change -79.69% 12.2% 481.44%

Facts In Submission

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CY2014 rtrx Reversal Of Registration Payment Obligation Liability
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CY2015 rtrx Reversal Of Registration Payment Obligation Liability
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CY2014 rtrx Securities Sold Not Yet Purchased1
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CY2015 rtrx Securities Sold Not Yet Purchased1
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CY2014 rtrx Sharebasedpaymentmadetoinvestorsforinducementtoparticipateinfinancing
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CY2015 rtrx Sharebasedpaymentmadetoinvestorsforinducementtoparticipateinfinancing
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CY2014 rtrx Shares Issued Related To Acquisition
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CY2015 rtrx Shares Issued Related To Acquisition
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CY2014Q4 rtrx Technology License Liability Current
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0 USD
CY2015 rtrx Unrealized Loss On Securities Sold Not Yet Purchased
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0 USD
CY2015 rtrx Unrecognized Tax Benefits Decrease Resultingfrom Deferred Tax Assets
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1800000 USD
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CY2013 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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CY2014 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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CY2015 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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CY2013 us-gaap Amortization Of Intangible Assets
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324000 USD
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732000 USD
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817000 USD
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0 USD
CY2014 us-gaap Amortization Of Debt Discount Premium
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0 USD
CY2015 us-gaap Amortization Of Debt Discount Premium
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398000 USD
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0 USD
CY2014 us-gaap Amortization Of Financing Costs
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0 USD
CY2013 us-gaap Amortization Of Financing Costs And Discounts
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0 USD
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1340000 USD
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4499000 USD
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8000 USD
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103000 USD
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651000 USD
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374000 USD
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2349000 USD
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0 USD
CY2014 us-gaap Business Combination Contingent Consideration Arrangements Change In Amount Of Contingent Consideration Liability1
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0 USD
CY2015 us-gaap Business Combination Contingent Consideration Arrangements Change In Amount Of Contingent Consideration Liability1
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4000 USD
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0 USD
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571000 USD
CY2015 us-gaap Cost Of Goods Sold
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CY2013 us-gaap Current Federal Tax Expense Benefit
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0 USD
CY2014 us-gaap Current Federal Tax Expense Benefit
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0 USD
CY2015 us-gaap Current Federal Tax Expense Benefit
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2094000 USD
CY2013 us-gaap Current Income Tax Expense Benefit
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0 USD
CY2014 us-gaap Current Income Tax Expense Benefit
CurrentIncomeTaxExpenseBenefit
0 USD
CY2015 us-gaap Current Income Tax Expense Benefit
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CY2013 us-gaap Current State And Local Tax Expense Benefit
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0 USD
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2300000 USD
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3160000 USD
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7830000 USD
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25124000 USD
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216000 USD
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0 USD
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1881000 USD
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1.78
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3.17
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0 USD
CY2014 us-gaap Effect Of Exchange Rate On Cash And Cash Equivalents
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0 USD
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0.2900
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0.2663
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-0.2702
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0.0230
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0.0551
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0.0000
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0.0368
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0.0153
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5602000 USD
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900000 USD
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0 USD
CY2015 us-gaap Excess Tax Benefit From Share Based Compensation Financing Activities
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2425000 USD
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10100000 USD
CY2014 us-gaap Fair Value Adjustment Of Warrants
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23786000 USD
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33307000 USD
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0
CY2015 us-gaap Fair Value Assumptions Expected Dividend Rate
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0
CY2014 us-gaap Fair Value Assumptions Expected Term
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P3M29D
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0.85
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17560000 USD
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84121000 USD
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15517000 USD
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15476000 USD
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15474000 USD
CY2015Q4 us-gaap Finite Lived Intangible Assets Amortization Expense Year Three
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15474000 USD
CY2015Q4 us-gaap Finite Lived Intangible Assets Amortization Expense Year Two
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15474000 USD
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99867000 USD
CY2015Q4 us-gaap Finite Lived Intangible Assets Gross
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179096000 USD
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94265000 USD
CY2015Q4 us-gaap Finite Lived Intangible Assets Net
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161536000 USD
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P11Y0M22D
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0 USD
CY2014 us-gaap Foreign Currency Transaction Gain Loss Before Tax
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0 USD
CY2015 us-gaap Foreign Currency Transaction Gain Loss Before Tax
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140004000 USD
CY2013 us-gaap Foreign Currency Transaction Gain Loss Realized
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0 USD
CY2014 us-gaap Foreign Currency Transaction Gain Loss Realized
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0 USD
CY2015 us-gaap Foreign Currency Transaction Gain Loss Realized
ForeignCurrencyTransactionGainLossRealized
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CY2014 us-gaap Gain Loss On Sale Of Property Plant Equipment
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0 USD
CY2015 us-gaap Gain Loss On Sale Of Property Plant Equipment
GainLossOnSaleOfPropertyPlantEquipment
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CY2013 us-gaap Gain Loss Related To Litigation Settlement
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0 USD
CY2014 us-gaap Gain Loss Related To Litigation Settlement
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0 USD
CY2015 us-gaap Gain Loss Related To Litigation Settlement
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15500000 USD
CY2013 us-gaap Gains Losses On Extinguishment Of Debt
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0 USD
CY2014 us-gaap Gains Losses On Extinguishment Of Debt
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0 USD
CY2015 us-gaap Gains Losses On Extinguishment Of Debt
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CY2014Q4 us-gaap Goodwill
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CY2015Q4 us-gaap Goodwill
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0 USD
CY2014 us-gaap Impairment Of Intangible Assets Finitelived
ImpairmentOfIntangibleAssetsFinitelived
0 USD
CY2015 us-gaap Impairment Of Intangible Assets Finitelived
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4710000 USD
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CY2014Q1 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2014Q2 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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9280000 USD
CY2014Q3 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2014Q4 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2014 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2015Q1 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2015Q2 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2015Q3 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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144339000 USD
CY2015Q4 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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CY2015 us-gaap Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest
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105467000 USD
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65000 USD
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IncomeTaxExpenseBenefit
0 USD
CY2014Q4 us-gaap Income Tax Expense Benefit
IncomeTaxExpenseBenefit
0 USD
CY2014 us-gaap Income Tax Expense Benefit
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CY2015Q1 us-gaap Income Tax Expense Benefit
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IncomeTaxExpenseBenefit
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CY2015Q3 us-gaap Income Tax Expense Benefit
IncomeTaxExpenseBenefit
38761000 USD
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CY2015 us-gaap Income Tax Expense Benefit
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CY2014 us-gaap Income Taxes Paid
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5000 USD
CY2015 us-gaap Income Taxes Paid
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9610000 USD
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2842000 USD
CY2014 us-gaap Increase Decrease In Accounts Payable And Accrued Liabilities
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20604000 USD
CY2015 us-gaap Increase Decrease In Accounts Payable And Accrued Liabilities
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3379000 USD
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0 USD
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CY2015 us-gaap Increase Decrease In Accounts Receivable
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4504000 USD
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0 USD
CY2014 us-gaap Increase Decrease In Inventories
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282000 USD
CY2015 us-gaap Increase Decrease In Inventories
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1174000 USD
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1349000 USD
CY2014 us-gaap Increase Decrease In Prepaid Deferred Expense And Other Assets
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CY2015 us-gaap Increase Decrease In Prepaid Deferred Expense And Other Assets
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966000 USD
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0 USD
CY2014 us-gaap Increase Decrease In Prepaid Taxes
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0 USD
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8107000 USD
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40000 USD
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0 USD
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0 USD
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CY2014 us-gaap Interest Expense
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CY2015 us-gaap Interest Expense
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4080000 USD
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259000 USD
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2536000 USD
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315000 USD
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289000 USD
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100000 USD
CY2015Q4 us-gaap Inventory Valuation Reserves
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300000 USD
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CY2014 us-gaap Operating Expenses
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OperatingExpenses
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OperatingExpenses
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0 USD
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0 USD
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1645000 USD
CY2015Q4 us-gaap Operating Leases Future Minimum Payments Next Rolling Twelve Months
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1026000 USD
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2265000 USD
CY2015Q4 us-gaap Other Assets Noncurrent
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1995000 USD
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4396000 USD
CY2015 us-gaap Other Comprehensive Income Availableforsale Securities Adjustment Net Of Tax Portion Attributable To Parent
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4395000 USD
CY2015 us-gaap Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
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300000 USD
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CY2015Q4 us-gaap Other Liabilities Noncurrent
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CY2015Q4 us-gaap Preferred Stock Shares Issued
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CY2015Q4 us-gaap Preferred Stock Shares Outstanding
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CY2015Q4 us-gaap Preferred Stock Value
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CY2015 us-gaap Other Nonoperating Income Expense
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OtherSignificantNoncashTransactionValueOfConsiderationGiven1
0 USD
CY2014 us-gaap Other Significant Noncash Transaction Value Of Consideration Given1
OtherSignificantNoncashTransactionValueOfConsiderationGiven1
0 USD
CY2015 us-gaap Other Significant Noncash Transaction Value Of Consideration Given1
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CY2013 us-gaap Payment Of Financing And Stock Issuance Costs
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0 USD
CY2014 us-gaap Payment Of Financing And Stock Issuance Costs
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CY2015 us-gaap Payment Of Financing And Stock Issuance Costs
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9500000 USD
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CY2013 us-gaap Payments For Repurchase Of Common Stock
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958000 USD
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2257000 USD
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CY2013 us-gaap Payments To Acquire Businesses Net Of Cash Acquired
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0 USD
CY2014 us-gaap Payments To Acquire Businesses Net Of Cash Acquired
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29150000 USD
CY2015 us-gaap Payments To Acquire Businesses Net Of Cash Acquired
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33430000 USD
CY2013 us-gaap Payments To Acquire Intangible Assets
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5433000 USD
CY2014 us-gaap Payments To Acquire Intangible Assets
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3301000 USD
CY2015 us-gaap Payments To Acquire Intangible Assets
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7008000 USD
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0 USD
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400000 USD
CY2014 us-gaap Payments To Acquire Marketable Securities
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10149000 USD
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198530000 USD
CY2013 us-gaap Payments To Acquire Property Plant And Equipment
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117000 USD
CY2014 us-gaap Payments To Acquire Property Plant And Equipment
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663000 USD
CY2015 us-gaap Payments To Acquire Property Plant And Equipment
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22000 USD
CY2014Q4 us-gaap Preferred Stock Par Or Stated Value Per Share
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CY2015Q4 us-gaap Preferred Stock Shares Authorized
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40000000 USD
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149487000 USD
CY2013 us-gaap Proceeds From Issuance Of Warrants
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0 USD
CY2014 us-gaap Proceeds From Issuance Of Warrants
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CY2015 us-gaap Proceeds From Issuance Of Warrants
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0 USD
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42366000 USD
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0 USD
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0 USD
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0 USD
CY2013 us-gaap Proceeds From Sale And Maturity Of Marketable Securities
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4385000 USD
CY2014 us-gaap Proceeds From Sale And Maturity Of Marketable Securities
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6493000 USD
CY2015 us-gaap Proceeds From Sale And Maturity Of Marketable Securities
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9977000 USD
CY2013 us-gaap Proceeds From Sale Of Productive Assets
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0 USD
CY2014 us-gaap Proceeds From Sale Of Productive Assets
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0 USD
CY2015 us-gaap Proceeds From Sale Of Productive Assets
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148411000 USD
CY2013 us-gaap Proceeds From Stock Options Exercised
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0 USD
CY2014 us-gaap Proceeds From Stock Options Exercised
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0 USD
CY2015 us-gaap Proceeds From Stock Options Exercised
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6818000 USD
CY2014Q4 us-gaap Property Plant And Equipment Net
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671000 USD
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428000 USD
CY2014 us-gaap Provision For Doubtful Accounts
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100000 USD
CY2015 us-gaap Provision For Doubtful Accounts
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0 USD
CY2015Q4 us-gaap Purchase Obligation
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596000 USD
CY2013 us-gaap Repayments Of Lines Of Credit
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0 USD
CY2014 us-gaap Repayments Of Lines Of Credit
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0 USD
CY2015 us-gaap Repayments Of Lines Of Credit
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45000000 USD
CY2013 us-gaap Repayments Of Notes Payable
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0 USD
CY2014 us-gaap Repayments Of Notes Payable
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31283000 USD
CY2015 us-gaap Repayments Of Notes Payable
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0 USD
CY2013 us-gaap Repayments Of Other Debt
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0 USD
CY2014 us-gaap Repayments Of Other Debt
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500000 USD
CY2015 us-gaap Repayments Of Other Debt
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2000000 USD
CY2013 us-gaap Repayments Of Related Party Debt
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<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:13px;padding-top:12px;font-size:13pt;"><font style="font-family:Arial;font-size:13pt;color:#4c4c4c;font-style:normal;font-weight:bold;text-decoration:none;">DESCRIPTION OF BUSINESS</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:16px;font-size:12pt;"><font style="font-family:Arial;font-size:12pt;color:#006ebf;font-style:normal;font-weight:bold;text-decoration:none;">Organization and Description of Business</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">Retrophin, Inc. (&#8220;we&#8221;, &#8220;our&#8221;, &#8220;us&#8221;, &#8220;Retrophin&#8221; and the &#8220;Company&#8221;) refers to Retrophin, Inc., a Delaware corporation, as well as our direct and indirect subsidiaries. Retrophin is a fully integrated biopharmaceutical company headquartered in San Diego, California focused on the development, acquisition and commercialization of therapies for the treatment of serious, catastrophic or rare diseases. We regularly evaluate and, where appropriate, act on opportunities to expand our product pipeline through licenses and acquisitions of products in areas that will serve patients with serious, catastrophic or rare diseases and that we believe offer attractive growth characteristics. </font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">During the first quarter of 2015, the Company completed the acquisition of all worldwide rights, titles, and ownership of Cholbam (cholic acid), the first FDA approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders). The Company generated the first sales from Cholbam (known as Kolbam in the European Union) in April 2015. See Note 3. for further discussion.</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:12px;font-size:10.5pt;"><font style="font-family:Arial;font-size:10.5pt;color:#666666;font-style:italic;font-weight:bold;text-decoration:none;">We currently sell the following three products:</font></div><table cellpadding="0" cellspacing="0" style="padding-top:4px;padding-bottom:8px;font-family:Times New Roman; font-size:10pt;"><tr><td style="width:48px;" rowspan="1" colspan="1"></td><td rowspan="1" colspan="1"></td></tr><tr><td style="vertical-align:top" rowspan="1" colspan="1"><div style="line-height:120%;font-size:9.5pt;padding-left:24px;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">&#8226;</font></div></td><td style="vertical-align:top;" rowspan="1" colspan="1"><div style="line-height:120%;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">Chenodal</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;"><sup style="vertical-align:top;line-height:120%;font-size:pt">&#174;</sup></font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;"> (chenodeoxycholic acid) is approved in the United States for the treatment of patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age. Chenodal has been the standard of care for cerebrotendinous xanthomatosis (&#8220;CTX&#8221;) patients for more than three decades and the Company is currently pursuing adding this indication to the label.</font></div></td></tr></table><table cellpadding="0" cellspacing="0" style="padding-top:4px;padding-bottom:8px;font-family:Times New Roman; font-size:10pt;"><tr><td style="width:48px;" rowspan="1" colspan="1"></td><td rowspan="1" colspan="1"></td></tr><tr><td style="vertical-align:top" rowspan="1" colspan="1"><div style="line-height:120%;font-size:9.5pt;padding-left:24px;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">&#8226;</font></div></td><td style="vertical-align:top;" rowspan="1" colspan="1"><div style="line-height:120%;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">Cholbam (cholic acid) is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders.</font></div></td></tr></table><table cellpadding="0" cellspacing="0" style="padding-top:4px;padding-bottom:8px;font-family:Times New Roman; font-size:10pt;"><tr><td style="width:48px;" rowspan="1" colspan="1"></td><td rowspan="1" colspan="1"></td></tr><tr><td style="vertical-align:top" rowspan="1" colspan="1"><div style="line-height:120%;font-size:9.5pt;padding-left:24px;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">&#8226;</font></div></td><td style="vertical-align:top;" rowspan="1" colspan="1"><div style="line-height:120%;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">Thiola (tiopronin) is approved in the United States for the </font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria.</font></div></td></tr></table><div style="line-height:120%;padding-bottom:8px;padding-top:12px;font-size:10.5pt;"><font style="font-family:Arial;font-size:10.5pt;color:#666666;font-style:italic;font-weight:bold;text-decoration:none;">We are currently developing the following pipeline products:</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">Sparsentan</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;font-style:italic;">,</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;"> also known as RE-021, is an investigational therapeutic agent which acts as both a potent angiotensin receptor blocker (&#8220;ARB&#8221;), as well as a selective endothelin receptor antagonist (&#8220;ERA&#8221;), with selectivity toward endothelin receptor type A. We have secured a license to sparsentan from Ligand and Bristol-Myers Squibb (who referred to it as DARA). We are developing sparsentan as a treatment for FSGS, which is a leading cause of end-stage renal disease. We are currently enrolling patients for the DUET Phase 2 clinical study of sparsentan for the treatment of FSGS and we anticipate having a top line data read out in the third quarter of 2016. Depending on the data obtained in the DUET study, we may be able to support an application for accelerated approval for sparsentan on the basis of proteinuria as a surrogate endpoint. Sparsentan was granted fast track designation in June 2015 and orphan drug designation in the U.S. and EU in January and November 2015, respectively.</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">RE-024, a novel small molecule, is being developed as a potential treatment for pantothenate kinase-associated neurodegeneration (&#8220;PKAN&#8221;). PKAN is a genetic neurodegenerative disorder that is typically diagnosed in the first decade of life. Consequences of PKAN include parkinsonism, dystonia, and other severe systemic manifestations. There are currently no viable treatment options for patients with PKAN.&#160; RE-024 is a phosphopantothenate prodrug therapy that aims to restore levels of this key substrate in PKAN patients.&#160; Certain international health regulators have approved the initiation of dosing RE-024 in PKAN under physician-initiated studies in accordance with local regulations in their respective countries.&#160;&#160; The Company filed a U.S. IND for RE-024 with the FDA in the first quarter of 2015 to support the commencement of a Company-sponsored Phase 1 study, which initiated in April 2015. RE-024 was granted orphan drug designation from the FDA in May 2015 and was granted fast track designation in June 2015.</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">RE-034 is a synthetic hormone analog of the first 24 amino acids of the 39 amino acids contained in adrenocorticotropic hormone (&#8220;ACTH&#8221;) incorporated into a novel formulation developed by the Company. &#160;RE-034 exhibits similar physiological actions as endogenous ACTH by binding to melanocortin receptors, resulting in its anti-inflammatory and immunomodulatory effects. The Company has successfully manufactured RE-034 at proof-of-concept scale using a novel formulation that allows modulation of the release of the active ingredient from the site of administration. &#160;The Company intends to continue preclinical development of RE-034 to enable multiple strategic options.</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:12px;font-size:10.5pt;"><font style="font-family:Arial;font-size:10.5pt;color:#666666;font-style:italic;font-weight:bold;text-decoration:none;">2015 Public Offering</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">On March 24, 2015, we completed a public offering of </font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">7,866,000</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;"> shares of common stock at a price of </font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">$19.00</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;"> per share. We received net proceeds from the offering of </font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">$140.0 million</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">, after deducting underwriting fees and other offering costs of </font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">$9.5 million</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">. The shares of common stock were offered by us pursuant to a shelf registration statement that was declared effective by the SEC on March 13, 2015.</font></div></div>
CY2015 us-gaap Prior Period Reclassification Adjustment Description
PriorPeriodReclassificationAdjustmentDescription
<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:8px;padding-top:12px;text-align:justify;font-size:10pt;"><font style="font-family:Arial Narrow;font-size:10pt;color:#006ebf;font-style:normal;font-weight:bold;text-decoration:none;">Reclassifications</font><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;font-style:italic;">&#160;</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">Certain reclassifications have been made to the prior year financial statements in order to conform to the current year&#8217;s presentation.</font></div></div>
CY2015 us-gaap Revenue Recognition Services Licensing Fees
RevenueRecognitionServicesLicensingFees
<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:8px;padding-top:12px;text-align:justify;font-size:10pt;"><font style="font-family:Arial Narrow;font-size:10pt;color:#006ebf;font-style:normal;font-weight:bold;text-decoration:none;">Patents</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">The Company expenses external costs, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company also expenses costs associated with maintaining and defending patents subsequent to their issuance in the period incurred.</font></div></div>
CY2015 us-gaap Use Of Estimates
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<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:8px;padding-top:12px;font-size:10pt;"><font style="font-family:Arial Narrow;font-size:10pt;color:#006ebf;font-style:normal;font-weight:bold;text-decoration:none;">Use of Estimates</font></div><div style="line-height:120%;padding-bottom:8px;padding-top:4px;text-align:justify;font-size:9.5pt;"><font style="font-family:Arial Narrow;font-size:9.5pt;color:#000000;">In preparing financial statements in conformity with U.S. GAAP, management is required to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reporting period. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. These estimates and assumptions include revenue recognition, valuing equity securities in share-based payments, estimating fair value of equity instruments recorded as derivative liabilities, estimating the fair value of net assets acquired in business combinations, estimating the useful lives of depreciable and amortizable assets, goodwill impairment, estimating of contingent consideration, estimating of valuation allowances and uncertain tax positions, and estimating the fair value of long-lived assets to assess whether impairment charges may apply.</font></div></div>

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