Item 1. |
FINANCIAL STATEMENTS |
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3 |
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4 |
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5 |
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6 |
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7 |
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Item 2. |
23 |
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Item 3. |
34 |
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Item 4. |
35 |
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PART II. OTHER INFORMATION |
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Item 1. |
35 |
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Item 1A. |
36 |
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Item 2. |
36 |
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Item 3. |
36 |
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Item 4. |
36 |
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Item 5. |
36 |
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Item 6. |
36 |
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37 |
September 30, 2020 |
December 31, 2019 |
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(unaudited) |
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ASSETS |
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Current Assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Short-term investments |
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Accounts receivable, net |
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Inventory |
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Prepaid expenses and other current assets |
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Total current assets |
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Deferred tax assets |
— | |||||||
Operating lease right-of-use |
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Property and equipment, net |
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Deposits |
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Total assets |
$ | $ | ||||||
LIABILITIES AND STOCKHOLDERS’ EQUITY |
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Current Liabilities: |
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Accounts payable |
$ | $ | ||||||
Accrued expenses and other liabilities |
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Total current liabilities |
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Operating lease liability, net of current portion |
— | |||||||
Total liabilities |
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Commitments and contingencies |
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Stockholders’ equity: |
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Preferred stock, $ |
— | |||||||
Common stock, $ |
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Additional paid-in capital |
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Accumulated deficit |
( |
) | ( |
) | ||||
Accumulated other comprehensive income (loss) |
( |
) | ||||||
$ | $ |
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For the Three Months Ended September 30, |
For the Nine Months Ended September 30, |
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2020 |
2019 |
2020 |
2019 |
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Revenues: |
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t |
$ | $ | $ | $ | ||||||||||||
Revenues from collaborative arrangements |
— | — | ||||||||||||||
Total revenues |
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Operating costs and expenses: |
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s |
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Research and development |
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Selling, general and administrative |
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Total operating costs and expenses |
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Operating income (loss) |
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Other income, net |
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Net income (loss) before income taxes |
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Income tax provision (benefit) |
( |
) | ( |
) | ||||||||||||
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Net income (loss) |
$ | $ | $ | $ | ||||||||||||
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Net income (loss) per share: |
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Basic |
$ | $ | $ | $ | ||||||||||||
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Diluted |
$ | $ | $ | $ | ||||||||||||
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Weighted average shares outstanding: |
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Basic |
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Diluted |
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Net income (loss) |
$ | $ | $ | $ | ||||||||||||
Other comprehensive income (loss): |
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Unrealized gain (loss) on available-for-sale |
( |
) | ( |
) | ( |
) | ||||||||||
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Comprehensive income (loss) |
$ | $ | $ | $ | ||||||||||||
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Preferred |
Common Stock |
Additional Paid-in |
Accumulated |
Accumulated Other Comprehensive |
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Stock |
Shares |
Amount |
Capital |
Deficit |
Gain (Loss) |
Total |
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Balance at December 31, 2019 |
$ | — | $ | $ | $ | ( |
) | $ | $ | |||||||||||||||||||
Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
Exercise of stock options for common stock |
— | — | — | |||||||||||||||||||||||||
Amortization of restricted stock for services |
— | — | — | — | — | |||||||||||||||||||||||
Other comprehensive gain (loss) |
— | — | — | — | — | |||||||||||||||||||||||
Net income (loss) |
— | — | — | — | — | |||||||||||||||||||||||
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Balance at March 31, 2020 |
— | ( |
) | |||||||||||||||||||||||||
Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
Exercise of stock options for common stock |
— | — | — | |||||||||||||||||||||||||
Amortization of restricted stock for services |
— | — | — | — | — | |||||||||||||||||||||||
Other comprehensive gain (loss) |
— | — | — | — | — | ( |
) | ( |
) | |||||||||||||||||||
Net income (loss) |
— | — | — | — | — | |||||||||||||||||||||||
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Balance at June 30, 2020 |
— | ( |
) | ( |
) | |||||||||||||||||||||||
Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
Exercise of stock options for common stock |
— | — | — | |||||||||||||||||||||||||
Amortization of restricted stock for services |
— | — | — | — | — | |||||||||||||||||||||||
Issuance of common stock upon vesting of restricted stock |
— | ( |
) | — | — | ( |
) | |||||||||||||||||||||
Other comprehensive gain (loss) |
— | — | — | — | — | ( |
) | ( |
) | |||||||||||||||||||
Net income (loss) |
— | — | — | — | — | |||||||||||||||||||||||
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Balance at September 30, 2020 |
$ | — | $ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||||
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Preferred |
Common Stock |
Additional Paid-in |
Accumulated |
Accumulated Other Comprehensive |
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Stock |
Shares |
Amount |
Capital |
Deficit |
Gain (Loss) |
Total |
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Balance at December 31, 2018 |
$ | — | $ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||||
Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
Exercise of stock options for common stock |
— | — | — | |||||||||||||||||||||||||
Other comprehensive gain (loss) |
— | — | — | — | — | |||||||||||||||||||||||
Net income (loss) |
— | — | — | — | ( |
) | — | ( |
) | |||||||||||||||||||
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Balance at March 31, 2019 |
— | ( |
) | ( |
) | |||||||||||||||||||||||
Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
Exercise of stock options for common stock |
— | — | — | |||||||||||||||||||||||||
Other comprehensive gain (loss) |
— | — | — | — | — | |||||||||||||||||||||||
Net income (loss) |
— | — | — | — | — | |||||||||||||||||||||||
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Balance at June 30, 2019 |
— | ( |
) | |||||||||||||||||||||||||
Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
Exercise of stock options for common stock |
— | — | — | |||||||||||||||||||||||||
Other comprehensive gain (loss) |
— | — | — | — | — | ( |
) | ( |
) | |||||||||||||||||||
Net income (loss) |
— | — | — | — | — | |||||||||||||||||||||||
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Balance at September 30, 2019 |
$ | — | $ | $ | $ | ( |
) | $ | $ | |||||||||||||||||||
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For the Nine Months Ended September 30, |
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2020 |
2019 |
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Operating Activities: |
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Net income (loss) |
$ | $ | ||||||
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: |
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Depreciation |
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Amortization of right-of-use |
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Stock-based compensation |
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Deferred taxes |
( |
) | — | |||||
Change in accrued interest and accretion of discount on investments |
( |
) | ( |
) | ||||
(Increase) decrease in: |
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Accounts receivable, net |
( |
) | ||||||
Inventory |
( |
) | ( |
) | ||||
Prepaid expenses and other current assets and deposits |
( |
) | ( |
) | ||||
Increase (decrease) in: |
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Accounts payable |
( |
) | ||||||
Accrued expenses and other liabilities |
( |
) | ||||||
Operating lease liability |
( |
) | ( |
) | ||||
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Net cash provided by (used in) operating activities |
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Investing Activities: |
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Purchases of property and equipment |
( |
) | ( |
) | ||||
Purchases of investments |
( |
) | ( |
) | ||||
Proceeds from maturities and sales of investments |
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Net cash provided by (used in) investing activities |
( |
) | ||||||
Financing Activities: |
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Payment of employee withholding tax related to stock-based compensation |
( |
) | — | |||||
Proceeds from exercise of stock options |
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Net cash provided by (used in) financing activities |
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Net increase (decrease) in cash and cash equivalents |
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Cash and cash equivalents—beginning of period |
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Cash and cash equivalents—end of period |
$ | $ | ||||||
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Supplemental disclosures of cash flow information: |
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Cash paid for income taxes |
$ | $ | — | |||||
Non-cash investing and financing activities: |
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Unrealized gain (loss) on available-for-sale |
$ | ( |
) | $ |
1. |
Organization and Description of Business. |
2. |
Basis of Presentation and Significant Accounting Policies. |
a. |
INTERIM FINANCIAL STATEMENTS. 10-Q was derived from the audited financial statements and does not include all disclosures required by U.S. GAAP. |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
b. |
PRINCIPLES OF CONSOLIDATION |
c. |
USE OF ESTIMATES. |
d. |
CASH AND CASH EQUIVALENTS. |
e. |
INVESTMENTS |
f. |
ACCOUNTS RECEIVABLE, NET. |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
g. |
INVENTORY. first-in-first-out work-in-process ® on November 28, 2018 as the related costs were expected to be recoverable through the commercialization of the product. Costs incurred prior to the FDA approval of Firdapse® were recorded as research and development expenses in prior years’ consolidated statements of operations and comprehensive income (loss). If information becomes available that suggests that inventories may not be realizable, the Company may be required to expense a portion or all of the previously capitalized inventories. As of September 30, 2020 and December 31, 2019, inventory consisted mainly of work-in-process |
h. |
PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-clinical studies, clinical trials and studies, regulatory affairs and consulting. Prepaid manufacturing consists of advances for the Company’s drug manufacturing activities. Such advances are recorded as expense as the related goods are received or the related services are performed. |
i. |
FAIR VALUE OF FINANCIAL INSTRUMENTS. |
j. |
FAIR VALUE MEASUREMENTS. |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
Fair Value Measurements at Reporting Date Using |
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Balances as of September 30, 2020 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) |
Significant Other Observable Inputs (Level 2) |
Significant Unobservable Inputs (Level 3) |
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Cash and cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | $ | — | $ | — | ||||||||||
U.S. Treasuries |
$ | $ | — | $ | $ | — | ||||||||||
Short-term investments: |
||||||||||||||||
Short-term bond funds |
$ | $ | $ | — | $ | — | ||||||||||
Balances as of December 31, 2019 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) |
Significant Other Observable Inputs (Level 2) |
Significant Unobservable Inputs (Level 3) |
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Cash and cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | $ | — | $ | — | ||||||||||
U.S. Treasuries |
$ | $ | — | $ | $ | — | ||||||||||
Short-term investments: |
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U.S. Treasuries |
$ | $ | — | $ | $ | — | ||||||||||
k. |
OPERATING LEASES. right-of-use non-lease components, which are generally accounted for separately. |
l. |
REVENUE RECOGNITION. ® in November 2018. Subsequent to receiving FDA approval, the Company entered into an arrangement with one distributor (the “Customer”), who is the exclusive distributor of Firdapse® in the United States. The Customer subsequently resells Firdapse® to a small group of exclusive specialty pharmacies (“SPs”) whose dispensing activities for patients with specific payors may result in government-mandated or privately negotiated rebate obligations for the Company with respect to the purchase of Firdapse® . |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
m. |
RESEARCH AND DEVELOPMENT. |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
n. |
STOCK-BASED COMPENSATION. |
o. |
CONCENTRATION OF RISK. |
p. |
ROYALTIES. |
q. |
INCOME TAXES. |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
r. |
COMPREHENSIVE INCOME (LOSS). available-for-sale |
s. |
NET INCOME (LOSS) PER COMMON SHARE. |
For the Three Months Ended September 30, |
For the Nine Months Ended September 30, |
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2020 |
2019 |
2020 |
2019 |
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Basic weighted average common shares outstanding |
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Effect of dilutive securities |
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Dilutive weighted average common shares outstanding |
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|
t. |
RECLASSIFICATIONS. |
u. |
RECENTLY ISSUED ACCOUNTING STANDARDS. 2018-18, Collaborative Arrangements (Topic 808), which amends ASC 808 to clarify when transactions between participants in a collaborative arrangement under ASC 808 are within the scope of the FASB’s new revenue standard, ASU 2014-09 (codified in ASC 606). The amendments require the application of ASC 606 existing guidance to determine the units of account that are distinct in a collaborative arrangement for purposes of identifying transactions with customers. If a unit of account within the collaborative arrangement is distinct and is with a customer, an entity shall apply the guidance in Topic 606 to that unit of account. In a transaction between collaborative participants, an entity is precluded by ASU 2018-18 from presenting a transaction together with “revenue from contracts with customers” unless the unit of account is within the scope of ASC 606 and the entity applies the guidance in ASC 606 to such unit of account. The Company adopted the new standard on January 1, 2020. The Company has a collaboration agreement with Endo Ventures Limited (Endo). See Note 7 (Collaborative Arrangement). However, these amendments did not have an impact on the Company’s consolidated financial statements, as Endo does not meet the definition of a customer. |
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
3. |
Investments. |
Estimated Fair Value |
Gross Unrealized Gains |
Gross Unrealized Losses |
Amortized Cost |
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At September 30, 2020: |
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U.S. Treasuries – Cash equivalents |
$ | $ | — | $ | ( |
) | $ | |||||||||
Bond Funds – ST |
— | ( |
) |
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|
|
|
|
|
|
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Total |
$ | $ | — | $ | ( |
) | $ | |||||||||
|
|
|
|
|
|
|
|
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At December 31, 2019 : |
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U.S. Treasuries – Cash equivalents |
$ | $ | $ | — | $ | |||||||||||
U.S. Treasuries – ST |
— | |||||||||||||||
|
|
|
|
|
|
|
|
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Total |
$ | $ | $ | — | $ | |||||||||||
|
|
|
|
|
|
|
|
September 30, 2020 |
||||
Due in one year or less |
$ | |||
|
|
4. |
Prepaid Expenses and Other Current Assets. |
September 30, 2020 |
December 31, 2019 |
|||||||
Prepaid manufacturing costs |
$ | $ | ||||||
Prepaid insurance |
||||||||
Prepaid subscription fees |
||||||||
Prepaid research fees |
||||||||
Prepaid commercialization expenses |
||||||||
Due from collaborative arrangements |
— | |||||||
Other |
||||||||
|
|
|
|
|||||
Total prepaid expenses and other current assets |
$ | $ | ||||||
|
|
|
|
5. |
Operating Leases. |
For the Three Months Ended September 30, 2020 |
For the Nine Months Ended September 30, 2020 |
|||||||
Operating lease cost |
$ | $ |
September 30, 2020 |
||||
Cash paid for amounts included in the measurement of lease liabilities: |
||||
Operating cash flows |
$ | |||
Right-of-use |
||||
Operating leases |
$ |
September 30, 2020 |
||||
Operating lease right-of-use |
$ | |||
Other current liabilities |
$ | |||
Operating lease liabilities, net of current portion |
||||
Total operating lease liabilities |
$ | |||
Weighted average remaining lease term |
||||
Weighted average discount rate |
% |
2020 (remaining three months) |
$ | |||
2021 |
||||
2022 |
||||
Total lease payments |
||||
Less imputed interest |
( |
) | ||
Total |
$ | |||
6. |
Accrued Expenses and Other Liabilities. |
September 30, 2020 |
December 31, 2019 |
|||||||
Accrued preclinical and clinical trial expenses |
$ | $ | ||||||
Accrued professional fees |
||||||||
Accrued compensation and benefits |
||||||||
Accrued license fees |
||||||||
Accrued purchases |
||||||||
Accrued contributions |
— | |||||||
Operating lease liability |
||||||||
Accrued variable consideration |
||||||||
Accrued income tax |
||||||||
Other |
||||||||
Current accrued expenses and other liabilities |
||||||||
Lease liability—non-current |
— | |||||||
Non-current accrued expenses and other liabilities |
— | |||||||
Total accrued expenses and other liabilities |
$ | $ | ||||||
7. |
Collaborative Arrangements. |
7. |
Collaborative Arrangement s (continued). |
8. |
Commitments and Contingencies. |
9. |
Agreements. |
a. |
LICENSE AGREEMENT WITH BIOMARIN (FIRDAPSE ® ) ® . Under the license agreement, the Company pays: (i) royalties to the licensor for ® equal to ® equal to |
b. |
AGREEMENTS FOR DRUG MANUFACTURING, DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. |
10. |
Income Taxes. |
11. |
Stockholders’ Equity. |
12. |
Stock Compensation. |
Three months ended September 30, |
Nine months ended September 30, |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||
Research and development |
$ | $ | $ | $ | ||||||||||||
Selling, general and administrative |
||||||||||||||||
Total stock-based compensation |
$ | $ | $ | $ | ||||||||||||
13. |
Subsequent Events. |
• | Overview. |
• | Basis of Presentation. |
• | Critical Accounting Policies and Estimates. |
• | Results of Operations. |
• | Liquidity and Capital Resources. off-balance sheet arrangements and our outstanding commitments, if any. |
• | Caution Concerning Forward-Looking Statements. |
Three months ended September 30, |
Change |
|||||||||||||||
2020 |
2019 |
$ |
% |
|||||||||||||
Research and development expenses |
$ | 3,349,986 | $ | 4,354,172 | (1,004,186 | ) | (23.1 | ) | ||||||||
Employee stock-based compensation |
399,247 | 242,867 | 156,380 | 64.4 | ||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total research and development expenses |
$ | 3,749,233 | $ | 4,597,039 | (847,806 | ) | (18.4 | ) | ||||||||
|
|
|
|
|
|
|
|
Nine months ended September 30, |
Change |
|||||||||||||||
2020 |
2019 |
$ |
% |
|||||||||||||
Research and development expenses |
$ | 11,083,166 | $ | 11,730,562 | (647,396 | ) | (5.5 | ) | ||||||||
Employee stock-based compensation |
1,238,521 | 803,800 | 434,721 | 54.1 | ||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total research and development expenses |
$ | 12,321,687 | $ | 12,534,362 | (212,675 | ) | (1.7 | ) | ||||||||
|
|
|
|
|
|
|
|
• | decreases in medical and regulatory affairs and quality assurance expenses and expenses from our ongoing clinical trials evaluating Firdapse ® for the treatment of MuSK-MG as we close out that trial, and our proof-of-concept ® for the treatment of SMA Type 3; and |
• | increases in employee stock-based compensation which is non-cash and relates to the expense of stock options awards to certain employees and stock option awards due to headcount increases. |
Three months ended September 30, |
Change |
|||||||||||||||
2020 |
2019 |
$ |
% |
|||||||||||||
Selling |
$ | 5,578,521 | $ | 4,670,568 | 907,953 | 19.4 | ||||||||||
General and administrative |
3,327,841 | 2,823,031 | 504,810 | 17.9 | ||||||||||||
Employee stock-based compensation |
1,078,599 | 574,193 | 504,406 | 87.8 | ||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total selling, general and administrative expenses |
$ | 9,984,961 | $ | 8,067,792 | 1,917,169 | 23.8 | ||||||||||
|
|
|
|
|
|
|
|
Nine months ended September 30, |
Change |
|||||||||||||||
2020 |
2019 |
$ |
% |
|||||||||||||
Selling |
$ | 17,004,447 | $ | 14,655,613 | 2,348,834 | 16.0 | ||||||||||
General and administrative |
10,323,782 | 8,944,694 | 1,379,088 | 15.4 | ||||||||||||
Employee stock-based compensation |
3,553,138 | 1,871,667 | 1,681,471 | 89.8 | ||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total selling, general and administrative expenses |
$ | 30,881,367 | $ | 25,471,974 | 5,409,393 | 21.2 | ||||||||||
|
|
|
|
|
|
|
|
• | increases in selling (commercialization) expenses, which consist primarily of the costs of our expansion of the sales force and the cost of contracting with a rare-disease experience inside sales agency; |
• | increases in general and administrative expenses, which are primarily due to the expansion of our operations and headcount to support our ongoing efforts to expand our net revenues from sales of Firdapse ® ; and |
• | increases in employee stock-based compensation which is non-cash and relates to the expense of stock option awards to certain employees and directors and stock option awards due to headcount increases. |
• | the scope, rate of progress and cost of our clinical trials and other product development activities; |
• | future clinical trial results; |
• | the terms and timing of any collaborative, licensing and other arrangements that we may establish; |
• | the cost and timing of regulatory approvals; |
• | the cost and delays in product development as a result of any changes in regulatory oversight applicable to our products; |
• | the level of revenues that we report from sales of Firdapse ® ; |
• | the effect of competition and market developments; |
• | the cost of filing and potentially prosecuting, defending and enforcing any patent claims and other intellectual property rights; and |
• | the extent to which we acquire or invest in other products. |
• | Payments under our license agreement ® equal to 7% of net sales (as defined in the license agreement) in North America for any calendar year for sales up to $100 million, and 10% of net sales in North America in any calendar year in excess of $100 million; and (ii) royalties to the third-party licensor of the rights sublicensed to us for seven years from the first commercial sale of Firdapse® equal to 7% of net sales (as defined in the license agreement between BioMarin and the third-party licensor) in any calendar year. For the three and nine-months ended September 30, 2020, we recognized approximately $3.9 million and $11.8 million, respectively, of royalties, which is included in cost of sales in the accompanying consolidated statement of operations. |
• | Purchase commitments |
• | Employment agreements |
• | Lease for office space |
• | The impact of the recent outbreak of a novel strain of coronavirus on our business or on the economy generally; |
• | Whether we will be able to continue to successfully market Firdapse ® while maintaining full compliance with applicable federal and state laws, rules and regulations; |
• | Whether our estimates of the size of the market for Firdapse ® for the treatment of Lambert-Eaton Myasthenic Syndrome (“LEMS”) will turn out to be accurate; |
• | Whether we will be able to locate LEMS patients who are undiagnosed or are misdiagnosed with other diseases; |
• | Whether patients will discontinue from the use of our drug at rates that are higher than historically experienced or are higher than we project; |
• | If the average daily dose taken by patients changes over time, it could affect our results of operations; |
• | Whether Firdapse ® patients can be successfully titrated to stable therapy; |
• | Whether we can continue to market Firdapse ® on a profitable and cash flow positive basis; |
• | Whether any revenue guidance that we provide to the public market will turn out to be accurate; |
• | Whether payors will continue to reimburse for our product at the price that we charge for the product; |
• | The ability of our third-party suppliers and contract manufacturers to maintain compliance with current Good Manufacturing Practices (cGMP); |
• | The ability of our distributor and the specialty pharmacies that distribute our product to maintain compliance with applicable law; |
• | Our ability to maintain compliance with applicable rules relating to our patient assistance programs and our contributions to 501(c)(3) organizations that support LEMS patients; |
• | The scope of our intellectual property and the outcome of any future challenges or opposition to our intellectual property, and, conversely, whether any third-party intellectual property presents unanticipated obstacles for Firdapse ® ; |
• | Whether our lawsuits against Jacobus and the specialty pharmacy distributing its product for patent infringement will be successful; |
• | The effect on our business and future results of operations arising from the approval by the FDA of Ruzurgi ® for the treatment of pediatric LEMS patients (ages 6 to under 17); |
• | Whether our suit against the United States FDA seeking to vacate the FDA’s approval of Ruzurgi ® will ultimately be successful; |
• | Whether we can continue to compete successfully if the approval of Ruzurgi ® is not overturned and Ruzurgi® continues to be prescribed for off-label use by adult LEMS patients; |
• | Whether, because of the lower price of Ruzurgi ® , payors will require that patients try off-label Ruzurgi® first before they approve Firdapse® as a treatment for adult LEMS patients; |
• | The impact on Firdapse ® of adverse changes in potential reimbursement and coverage policies from government and private payors such as Medicare, Medicaid, insurance companies, health maintenance organizations and other plan administrators, or the impact of pricing pressures enacted by industry organization, the federal government or the government of any state, including as a result of increased scrutiny over pharmaceutical pricing or otherwise; |
• | The impact on our business and results of operations of public statements by politicians and a vocal group of LEMS patients and doctors who object to our pricing of Firdapse ® ; |
• | Changes in the healthcare industry and the effect of political pressure from and actions by President Trump, Congress and/or medical professionals seeking to reduce prescription drug costs; |
• | The state of the economy generally and its impact on our business; |
• | Changes to the healthcare industry occasioned by any future repeal and replacement of the Affordable Care Act, in laws relating to the pricing of drug products, or changes in the healthcare industry generally; |
• | The scope, rate of progress and expense of our clinical trials and studies, pre-clinical studies, proof-of-concept |
• | Our ability to complete our trials and studies on a timely basis and within the budgets we establish for such trials and studies; |
• | Whether the recent coronavirus outbreak will further affect the timing of our currently ongoing clinical trials; |
• | Whether the trial that we are currently undertaking to evaluate Firdapse ® for the treatment of Spinal Muscular Atrophy (SMA) Type 3, or any other trials that we may undertake in the future, will be successful; |
• | Whether Firdapse ® will ever be approved for the treatment of MuSK-MG, SMA Type 3, or any other neuromuscular disease; |
• | Whether we can successfully commercialize Firdapse ® in Canada on a profitable basis; |
• | Whether our suit to overturn the approval of Ruzurgi ® in Canada will be successful; |
• | The impact on sales of Firdapse ® in the United States if an amifampridine product is purchased in Canada for use in the United States; |
• | Whether we will be able to successfully complete the clinical trial in Japan that will be required to seek approval to commercialize Firdapse ® in Japan; |
• | Whether we will be able to obtain approval to commercialize Firdapse ® in Japan; |
• | Whether we can successfully develop, obtain approval of and successfully market a long-acting version of Firdapse ® ; |
• | Whether our efforts to grow our business beyond Firdapse ® through acquisitions of companies or in-licensing of product opportunities in the neuromuscular or neurology therapeutic areas will be successful; |
• | Whether we will have sufficient capital to finance any such acquisitions; |
• | Whether our version of generic vigabatrin tablets will ever be approved by the FDA; |
• | Even if our version of vigabatrin tablets is approved for commercialization, whether Endo Ventures/Par Pharmaceutical (our collaborator in this venture) will be successful in marketing the product; and |
• | Whether we will earn milestone payments on the first commercial sale of vigabatrin tablets and royalties on sales of generic vigabatrin tablets. |
a. |
We have carried out an evaluation, under the supervision and with the participation of our management, including our principal executive officer and principal financial officer, of the effectiveness of the design and operation of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Based on such evaluation, our principal executive officer and principal financial officer have concluded that as of September 30, 2020, our disclosure controls and procedures were effective to ensure that the information required to be disclosed by us in the reports filed or submitted by us under the Exchange Act, was recorded, processed, summarized or reported within the time periods specified in the rules and regulations of the SEC, and include controls and procedures designed to ensure that information required to be disclosed by us in such reports was accumulated and communicated to management, including our principal executive officer and principal financial officer, as appropriate to allow timely decisions regarding required disclosures. |
b. |
During the three months ended September 30, 2020, there were no changes in our internal controls or in other factors that could have a material effect, or are reasonably likely to have a material effect, on our internal control over financial reporting. |
31.1 |
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31.2 |
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32.1 |
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32.2 |
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101.INS |
Inline XBRL Instance Document – the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document. | |
101.SCH |
Inline XBRL Taxonomy Extension Schema Document | |
101.CAL |
Inline XBRL Taxonomy Extension Calculation Linkbase Document | |
101.DEF |
Inline XBRL Taxonomy Extension Definition Linkbase Document | |
101.LAB |
Inline XBRL Taxonomy Extension Label Linkbase Document | |
101.PRE |
Inline XBRL Taxonomy Extension Presentation Linkbase Document | |
104 |
The cover page for the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, has been formatted in Inline XBRL. |
Catalyst Pharmaceuticals, Inc. | ||
By: | /s/ Alicia Grande | |
Alicia Grande | ||
Vice President, Treasurer and Chief Financial Officer |
Exhibit 31.1
Certification of Principal Executive Officer
I, Patrick J. McEnany, certify that:
1. | I have reviewed this quarterly report on Form 10-Q of Catalyst Pharmaceuticals, Inc.; |
2. | Based on my knowledge, this quarterly report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report; |
3. | Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; |
4. | The registrants other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15(d)-15(f)) for the registrant and have: |
a) | Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiary, is made known to us by others within those entities, particularly during the period in which this report is being prepared; |
b) | Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles; |
c) | Evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and |
d) | Disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; and |
5. | The registrants other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrants auditors and the audit committee of registrants board of directors (or persons performing the equivalent functions): |
a) | all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and |
b) | any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting. |
Date: November 9, 2020
/s/ Patrick J. McEnany |
Patrick J. McEnany |
Chief Executive Officer |
(Principal Executive Officer) |
Exhibit 31.2
Certification of Principal Financial Officer
I, Alicia Grande, certify that:
1. | I have reviewed this quarterly report on Form 10-Q of Catalyst Pharmaceuticals, Inc.; |
2. | Based on my knowledge, this quarterly report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report; |
3. | Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; |
4. | The registrants other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15(d)-15(f)) for the registrant and have: |
a) | Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiary, is made known to us by others within those entities, particularly during the period in which this report is being prepared; |
b) | Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles; |
c) | Evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and |
d) | Disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; and |
5. | The registrants other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrants auditors and the audit committee of registrants board of directors (or persons performing the equivalent functions): |
a) | all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and |
b) | any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting. |
Date: November 9, 2020
/s/ Alicia Grande |
Alicia Grande |
Chief Financial Officer |
(Principal Financial Officer) |
Exhibit 32.1
Certification Required by 18 U.S.C. Section 1350
(as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002)
I, Patrick J. McEnany as Principal Executive Officer of Catalyst Pharmaceuticals, Inc. (the Company), certify, pursuant to 18 U.S.C. Section 1350 (as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002), that to my knowledge:
1. | the accompanying Quarterly Report on Form 10-Q of the Company for the quarterly period ended September 30, 2020 (the Report), filed with the U.S. Securities and Exchange Commission, fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934, as amended; and |
2. | the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company. |
Date: November 9, 2020 | /s/ Patrick J. McEnany | |||||
Patrick J. McEnany | ||||||
Chief Executive Officer | ||||||
(Principal Executive Officer) |
Exhibit 32.2
Certification Required by 18 U.S.C. Section 1350
(as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002)
I, Alicia Grande as Principal Financial Officer of Catalyst Pharmaceuticals, Inc. (the Company), certify, pursuant to 18 U.S.C. Section 1350 (as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002), that to my knowledge:
1. | the accompanying Quarterly Report on Form 10-Q of the Company for the quarterly period ended September 30, 2020 (the Report), filed with the U.S. Securities and Exchange Commission, fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934, as amended; and |
2. | the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company. |
Date: November 9, 2020 | /s/ Alicia Grande | |||||
Alicia Grande | ||||||
Chief Financial Officer | ||||||
(Principal Financial Officer) |
Consolidated Balance Sheets (Parenthetical) - $ / shares |
Sep. 30, 2020 |
Dec. 31, 2019 |
---|---|---|
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 200,000,000 | 150,000,000 |
Common stock, shares issued | 103,648,224 | 103,397,033 |
Common stock, shares outstanding | 103,648,224 | 103,397,033 |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (unaudited) - USD ($) |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2020 |
Sep. 30, 2019 |
Sep. 30, 2020 |
Sep. 30, 2019 |
|
Revenues: | ||||
Product revenue, net | $ 29,166,658 | $ 30,897,444 | $ 87,907,894 | $ 72,183,782 |
Revenue, Product and Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Revenues from collaborative arrangements | $ 150,000 | $ 150,000 | ||
Total revenues | 29,316,658 | $ 30,897,444 | 88,057,894 | $ 72,183,782 |
Operating costs and expenses: | ||||
Cost of sales | $ 3,878,760 | $ 4,387,461 | $ 12,169,499 | $ 10,360,874 |
Cost, Product and Service [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Research and development | $ 3,749,233 | $ 4,597,039 | $ 12,321,687 | $ 12,534,362 |
Selling, general and administrative | 9,984,961 | 8,067,792 | 30,881,367 | 25,471,974 |
Total operating costs and expenses | 17,612,954 | 17,052,292 | 55,372,553 | 48,367,210 |
Operating income (loss) | 11,703,704 | 13,845,152 | 32,685,341 | 23,816,572 |
Other income, net | 33,567 | 393,415 | 481,069 | 1,187,091 |
Net income (loss) before income taxes | 11,737,271 | 14,238,567 | 33,166,410 | 25,003,663 |
Income tax provision (benefit) | (31,602,596) | 608,388 | (30,379,459) | 1,058,039 |
Net income (loss) | $ 43,339,867 | $ 13,630,179 | $ 63,545,869 | $ 23,945,624 |
Net income (loss) per share: | ||||
Basic | $ 0.42 | $ 0.13 | $ 0.61 | $ 0.23 |
Diluted | $ 0.41 | $ 0.13 | $ 0.60 | $ 0.23 |
Weighted average shares outstanding: | ||||
Basic | 103,535,431 | 102,974,105 | 103,452,025 | 102,864,571 |
Diluted | 106,316,241 | 107,045,234 | 106,386,617 | 105,821,609 |
Net income (loss) | $ 43,339,867 | $ 13,630,179 | $ 63,545,869 | $ 23,945,624 |
Other comprehensive income (loss): | ||||
Unrealized gain (loss) on available-for-sale securities | (5,280) | (2,330) | (15,976) | 39,676 |
Comprehensive income (loss) | $ 43,334,587 | $ 13,627,849 | $ 63,529,893 | $ 23,985,300 |
Organization and Description of Business |
9 Months Ended | ||
---|---|---|---|
Sep. 30, 2020 | |||
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Organization and Description of Business |
Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”) is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), Anti-MuSK antibody positive myasthenia gravis (MuSK-MG), and Spinal Muscular Atrophy (SMA) Type 3. On November 28, 2018, the U.S. Food and Drug Administration ( FDA) granted approval of Firdapse® for the treatment of adults with LEMS (ages 17 and above). On January 15, 2019, the Company launched its first product, Firdapse® , in the United States for the treatment of adults with LEMS. On August 6, 2020, the Company announced that Canada’s national healthcare regulatory agency, Health Canada, has approved Firdapse ® for the treatment of patients in Canada with LEMS. Since inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets, raising capital, and selling its product. The Company incurred operating losses in each period from inception, and started reporting operating income during the year ended December 31, 2019. The Company has been able to fund its cash needs to date through several public and private offerings of its securities and from revenues from product sales. See Note 11 (Stockholders’ Equity). Capital Resources While there can be no assurance, based on currently available information, the Company estimates that it has sufficient resources to support its operations for at least the next 12 months from the issuance date of this Form 10-Q. The Company may raise required funds in the future through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional product development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business. Risks and Uncertainties There are many uncertainties regarding the novel coronavirus
(COVID-19) pandemic, and the Company is closely monitoring the impact of the pandemic on all aspects of its business, including how the pandemic is impacting its patients, employees, suppliers, vendors, business partners, clinical trials, and distribution channels. The Company is unable to predict the impact that COVID-19 will have on its financial position and operating results in future periods due to numerous uncertainties. The Company will continue to assess the evolving impact of the COVID-19 pandemic and make adjustments to its operations as necessary. |
Basis of Presentation and Significant Accounting Policies |
9 Months Ended | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Accounting Policies [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Basis of Presentation and Significant Accounting Policies |
In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2019 included in the 2019 Annual Report on Form 10-K filed by the Company with the SEC. The results of operations for the nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for any future period or for the full 2020 fiscal year.
The short-term bond funds and U.S. Treasuries held at September 30, 2020 are classified as available-for-sale non-current investments in its consolidated balance sheets. The Company records available-for-sale available-for-sale available-for-sale The Company previously owned a short-term bond fund that was classified as trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognized realized gains and losses and unrealized gains and losses to earnings. At September 30, 2020 and December 31, 2019, there were no investments classified as trading securities, as the Company sold its interest in the short-term bond fund classified as trading in 2019. There was no realized or unrealized gain (loss) on trading securities for the three and nine months ended September 30, 2020. Realized losses on trading securities during the three and nine months ended September 30, 2019 were $0 and $4,980, respectively. Unrealized gain (loss) on trading securities was $0 and $89,405 for the three and nine months ended September 30, 2019 , respectively, and is included in other income, net in the accompanying consolidated statements of operations.
Products that have been approved by the FDA or other regulatory authorities, such as Firdapse ® , are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of Firdapse® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”. The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage.
Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability.
To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to arrangements that meet the definition of a contract under Topic 606, including when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below. The Company also may generate revenues from payments received under a collaborative agreement. Collaborative agreement payments may include nonrefundable fees at the inception of the agreements, milestone and event-based payments for specific achievements designated in the collaborative agreements, and/or royalties on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, see Revenues from Collaborative Arrangements below. Product Revenue, Net: ® to the Customer (its exclusive distributor) who subsequently resells Firdapse® to both a small group of SPs who have exclusive contracts with the Company to distribute the Company’s products to patients and potentially to medical centers or hospitals on an emergency basis. In addition to the distribution agreement with its Customer, the Company enters into arrangements with health care providers and payors that provide for government-mandated and/or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of the Company’s products.The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days. Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods, and are recorded in cost of sales. If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three and nine month periods ended September 30, 2020 and 2019. During the three and nine months ended September 30, 2020 and 2019, all of the Company’s sales were to its Customer. Reserves for Variable Consideration: These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts. The amount of variable consideration which is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of September 30, 2020 and, therefore, the transaction price was not reduced further during the three and nine months ended September 30, 2020 and 2019. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. Trade Discounts and Allowances: ® to the Customer, these payments are classified in selling, general and administrative expenses in the Company’s consolidated statement of operations and comprehensive income (loss). However, if the Company has determined such services received to date are not distinct from the Company’s sale of products to the Customer, these payments have been recorded as a reduction of revenue within the consolidated statement of operations and comprehensive income (loss) through September 30, 2020 and 2019, as well as a reduction to accounts receivable, net on the consolidated balance sheets.Funded Co-pay Assistance Program:co-pay assistance program intended to provide financial assistance to qualified commercially-insured patients. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that the Company expects to receive associated with Firdapse® that has been recognized as revenue, but remains in the distribution channel at the end of each reporting period. These payments are considered payable to the Customer and the related reserve is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities in the consolidated balance sheets.Product Returns: Provider Chargebacks and Discounts: Government Rebates: Bridge and Patient Assistance Programs: ® to uninsured patients who satisfy pre-established criteria for either the Bridge Program or the Patient Assistance Program. Patients who meet the Bridge Program eligibility criteria and are transitioning from investigational product while they are waiting for a coveragedetermination, or later, for patients whose access is threatened by the complications arising from a change of insurer may receive a temporary supply of free Firdapse ® while the Company is determining the patient’s third-party insurance, prescription drug benefit or other third-party coverage for Firdapse® . The Patient Assistance Program provides free Firdapse® for longer periods of time for those who are uninsured or functionally uninsured with respect to Firdapse® because they are unable to obtain coverage from their payor despite having health insurance, to the extent allowed by applicable law. The Company does not recognize any revenue related to these free products and the associated costs are classified in selling, general and administrative expenses in the Company’s consolidated statements of operations and comprehensive income (loss). Revenues from Collaborative Arrangements: ® (vigabatrin) tablets as well as the commercialization of Firdapse® in Canada. Pursuant to the terms of these agreements, collaborators could be required to make various payments to the Company, including upfront license fees, milestone payments based on achievement of regulatory approvals, and royalties on sales of products resulting from the collaborative agreement. Nonrefundable upfront license fees are recognized upon receipt as persuasive evidence of an arrangement exists, the price to the collaborator is fixed or determinable and collectability is reasonably assured. In the third quarter of 2020, an upfront fee was earned under the collaboration agreement for the commercialization of Firdapse ® in Canada. The collaborative agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments in accordance with the provisions of Accounting Standards Update (ASU) No. 2010-17, Revenue Recognition – Milestone Method (“Milestone Method of Accounting”). The Company recognizes consideration that is contingent upon the achievement of a milestone in its entirety as revenue in the period in which the milestone is achieved only if the milestone is substantive in its entirety. A milestone is considered substantive when it meets all of the following criteria:1. The consideration is commensurate with either the entity’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone; 2. The consideration relates solely to past performance; and 3. The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. A milestone is defined as an event (i) that can only be achieved based in whole or in part on either the entity’s performance or on the occurrence of a specific outcome resulting from the entity’s performance, (ii) for which there is substantive uncertainty at the date the arrangement is entered into that the event will be achieved, and (iii) that would result in additional payments being due to the vendor. The Company believes that achievement of the milestones will be substantive and there will be no substantive uncertainty once the milestones are achieved. No milestones were achieved in the three and nine months ended September 30, 2020 and 2019. In arrangements where the Company does not deem the collaborator to be a customer, payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments. Under the arrangements, the Company will receive royalty reports 60 days after quarter end from one collaborator, and within nine days after quarter end from the other collaborator. Since the Company will receive royalty reports 60 days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. In instances where royalty reports are received within nine days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter in which the sales occurred. For the three and nine months ended September 30, 2020 and 2019, there was no royalty revenue from sales of the collaborative product. Refer to Note 7 (Collaborative Arrangement), for further discussion on the Company’s collaborative arrangement.
The Company sells its product in the United States through an exclusive distributor (its Customer) to specialty pharmacies. Therefore, its distributor and specialty pharmacies account for all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions. The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse ® , and expects Firdapse® to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to effectively commercialize Firdapse® . The Company relies exclusively on third parties to formulate and manufacture Firdapse ® and its drug candidates. The commercialization of Firdapse® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and for the commercialization of Firdapse® . If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of its drugs.
The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-than-not threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50 percent likelihood of being realized upon ultimate settlement with the relevant tax authority. The Company is subject to income taxes in the U.S. federal jurisdiction and various state jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for years before 2017 . If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.On March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed into law making several changes to the Internal Revenue Code. The changes include, but are not limited to: increasing the limitation on the amount of deductible interest expense, allowing companies to carryback certain net operating losses, and increasing the amount of net operating loss carryforwards that corporations can use to offset taxable income. The tax law changes in the CARES Act did not have a material impact on the Company’s income tax provision.
Diluted net income (loss) per common share is computed by dividing net income (loss) by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period. The following table reconciles basic and diluted weighted average common shares:
Outstanding common stock equivalents totaling approximately 5.4 million and 4.8 million, were excluded from the calculation of diluted net income (loss) per common share for the three and nine months ended September 30, 2020 as their effect would be anti-dilutive. For the three and nine months ended September 30, 2019, approximately 0.4 million and 2.9 million shares of outstanding stock options were excluded from the calculation of diluted net income (loss) per common share as their effect would be anti-dilutive.
In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments. available-for-sale In August 2018, the FASB issued ASU 2018-15, Intangibles – Goodwill and Other – Internal-Use Software (Subtopic 350-40), Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract.internal-use software (and hosting arrangements that include an internal-use software license). Accordingly, the amendments in this update require an entity in a hosting arrangement that is a service contract to follow the guidance in Subtopic 350-40 to determine which implementation costs to capitalize as an asset related to the service contract and which costs to expense. The Company adopted the new standard on January 1, 2020 and applied prospectively to all implementation costs incurred after the date of adoption. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements. |
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Available-for-sale
There were no realized gains or losses from available-for-sale The estimated fair values of available-for-sale September
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Prepaid Expenses and Other Current Assets |
Prepaid expenses and other current assets consist of the following:
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Operating Leases |
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Disclosure of Operating Leases [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Operating Leases |
The Company has operating lease agreements for its corporate office. The leases include options to extend the leases for up to 1 year and options to terminate the lease within 1 year. There are no obligations under finance leases. The Company entered into an agreement in May 2020 that amended its lease for its office facilities. Under the amended lease, the Company’s leased space will increase from approximately 7,800 square feet of space to approximately 10,700 square feet of space. The lessor is currently building this space. The lease is expected to commence in early 2021 when construction of the asset is completed and available for use. The lease disclosures for the three and nine months periods ended September 30, 2020 in these financial statements have been adjusted for the modification of the current lease. The components of lease expense were as follows:
Supplemental cash flow information related to leases was as follows:
Supplemental balance sheet information related to leases was as follows:
Remaining payments of lease liabilities as of September 30, 2020 were as follows:
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Accrued Expenses and Other Liabilities |
Accrued expenses and other liabilities consist of the following:
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Collaborative Arrangements |
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Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Collaborative Arrangements |
In December 2018, the Company entered into a collaboration and license agreement (Collaboration) with Endo, for the further development and commercialization of generic Sabril ® (vigabatrin) tablets through Endo’s U.S. Generic Pharmaceuticals segment, doing business as Par Pharmaceutical. Under the Collaboration, Endo assumes all development, manufacturing, clinical, regulatory, sales and marketing costs under the collaboration, while the Company is responsible for exercising commercially reasonable efforts to develop, or cause the development of, a final finished, stable dosage form of generic Sabril ® tablets. Under the terms of the Collaboration, the Company has received an up-front payment, and will receive a milestone payment, and a sharing of defined net profits upon commercialization from Endo consisting of a mid-double-digit percent of net sales of generic Sabril® . The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is ten years following the commercial launch of the product.The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of September 30, 2020 and 2019, no milestone payments have been earned. There were no revenues from collaborative arrangement with Endo for the three or nine months ended September 30, 2020 and 2019. Total expenses incurred, net, in connection with the collaborative agreement with Endo for three and nine months ended September 30, 2020 were approximately $0 and $4,200, respectively. Total expenses incurred, net, in connection with the collaborative agreement with Endo for three and nine months ended September 30, 2019 were approximately $32,000 and $70,000, respectively. These expenses have been included in research and development expenses in the accompanying consolidated statements of operations. In August 2020, the Company entered into a collaboration and license agreement with KYE Pharmaceuticals Inc (KYE), for the commercialization of Firdapse ® in Canada. Under the agreement, KYE assumes all selling and marketing costs under the collaboration, while the Company is responsible for supply of Firdapse ® based on the collaboration partner’s purchase orders. Under the terms of the agreement, the Company has earned an up-front payment, will receive milestone payments, and a sharing of defined net profits upon commercialization from KYE consisting of a mid-double-digit percent of net sales of Firdapse® . The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is ten years following the commercial launch of the product in Canada. As of September 30, 2020, a $150,000 upfront fee was recognized in connection with the collaborative agreement with KYE. Total expenses incurred, net, in connection with the collaborative agreement with KYE for three and nine months ended September 30, 2020 were approximately $161,000. These expenses have been included in selling, general and administrative expenses in the accompanying consolidated statements of operations. The collaborative agreement provides for event-based payments subject to achievement of specified development, regulatory and sales-based milestones. As of September 30, 2020, no milestone payments have been earned. |
Commitments and Contingencies |
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Commitments and Contingencies Disclosure [Abstract] | |||
Commitments and Contingencies |
In 2018, the Company became aware that certain patents granted to Northwestern University (which patents have been licensed by Northwestern to a third party) for a new GABA aminotransferase inhibitor were developed from CPP-115, which had previously been licensed to the Company by Northwestern. As a result, on October 26, 2018, the Company terminated the license agreement for CPP-115 and commenced an arbitration proceeding against Northwestern seeking damages for alleged breaches of the license agreement. Shortly thereafter, Northwestern filed counterclaims against the Company in the arbitration action seeking damages for alleged breaches by the Company of the license agreement. On May 21, 2019, the Company entered into a settlement agreement with Northwestern that resolved all pending disputes between the parties with no admission of liability by either party, released all claims of liability or wrongdoing between the Company and Northwestern, and dismissed the pending arbitration. Under the settlement agreement, the Company received a $100,000 payment on May 21, 2019, which is reported as income in other income, net in the consolidated statement of operations. The Company is also entitled to receive certain contingent compensation that will be reported when and if received.In May 2019, the FDA approved an NDA for Jacobus Pharmaceuticals (“Jacobus”) for Ruzurgi ® , their version of amifampridine (3,4-DAP), for the treatment of pediatric LEMS patients (ages 6 to under 17). The Company believes that Jacobus is offering Ruzurgi® at a lower price than the Company is offering Firdapse® . In addition, while the NDA for Ruzurgi® only covers pediatric patients, the Company believes Ruzurgi® is being prescribed off label to adult LEMS patients. If Jacobus is able to successfully sell Ruzurgi® off-label to adult LEMS patients, it could have a material adverse effect on the Company’s business, financial condition and results of operations. The Company believes that the FDA’s approval of Ruzurgi ® violated its statutory rights and was in multiple other respects arbitrary, capricious and contrary to law. As a result, in June 2019 the Company filed suit against the FDA and several related parties challenging this approval and related drug labeling. The Company’s complaint, which was filed in the federal district court for the Southern District of Florida, alleged that the FDA’s approval of Ruzurgi® violated multiple provisions of FDA regulations regarding labeling, resulting in misbranding in violation of the Federal Food, Drug, and Cosmetic Act (FDCA); violated its statutory rights to Orphan Drug Exclusivity and New Chemical Entity Exclusivity under the FDCA; and was in multiple other respects arbitrary, capricious, and contrary to law, in violation of the Administrative Procedure Act. Among other remedies, the suit sought an order vacating the FDA’s approval of Ruzurgi® . Jacobus intervened in the case and each party filed a cross motion for summary judgement. On July 30, 2020, the Magistrate Judge considering the Company’s lawsuit against the FDA filed a Report and Recommendation in which she recommended to the District Judge handling the case that she grant the FDA’s and Jacobus’ motions for summary judgement and deny the Company’s motion for summary judgement. On September 29, 2020, the District Judge adopted the Report and Recommendation of the Magistrate Judge, granted the FDA’s and Jacobus’s motions for summary judgement, and dismissed the Company’s case. The Company has appealed the decision to the Eleventh Circuit Court of Appeals. There can be no assurance as to the outcome of this lawsuit. On August 10, 2020, Health Canada issued a Notice of Compliance (NOC) to Medunik for Ruzurgi ® for the treatment of LEMS. The Company has since initiated a legal proceeding in Canada seeking judicial review of Health Canada’s decision to issue the NOC for Ruzurgi® as incorrect and unreasonable under Canadian law. Data protection, per Health Canada regulations, is supposed to prevent Health Canada from issuing a NOC to a drug that directly or indirectly references an innovative drug’s data, for eight years from the date of the innovative drug’s approval. The Ruzurgi® Product Monograph clearly references pivotal nonclinical carcinogenicity and reproductive toxicity data for amifampridine phosphate developed by Catalyst. As such, the Company believes that our data was relied upon to establish the nonclinical safety profile of Ruzurgi® needed to meet the standards of the Canadian Food and Drugs Act. There can be no assurance of the results of this proceeding. Additionally, from time to time the Company may become involved in legal proceedings arising in the ordinary course of business. Except as set forth above, the Company believes that there is no other litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on its results of operations, financial condition or cash flows. |
Agreements |
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Agreements |
On May 29, 2019, the Company entered into an amendment to its license agreement for Firdapse ® . Under the amendment, the Company has expanded its commercial territory for Firdapse® , which originally was comprised of North America, to include Japan. Additionally, the Company has an option to further expand its territory under the license agreement to include most of Asia, as well as Central and South America, upon the achievement of certain milestones in Japan. Under the amendment, the Company will pay royalties on net sales in Japan of a similar percentage to the royalties that the Company is currently paying under its original license agreement for North America. During January 2020, the Company was advised that BioMarin has transferred certain rights under the license agreement to SERB S.A.
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Income Taxes |
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Income Tax Disclosure [Abstract] | |||
Income Taxes |
The Company is subject to income taxes in the U.S. federal jurisdiction and various states jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for any years before 2017 . If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense. The Company has evaluated the positive and negative e v idence bearing upon the realizability of its deferred tax assets. As of September 30, 2020, the Company determined that there is sufficient positive evidence to conclude that it is more likely than not that additional deferred taxes of approximately $31.3 million are realizable. The Company, therefore, reduced the valuation allowance accordingly. As of December 31, 2019, the Company provided a full valuation allowance for deferred tax assets including NOL and tax credit carryovers. |
Stockholders' Equity |
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Equity [Abstract] | |||
Stockholders' Equity |
Preferred Stock The Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share, at September 30, 2020 and December 31, 2019. No shares of preferred stock were outstanding at September 30, 2020 and December 31, 2019. Common Stock On August 20, 2020, the Company’s stockholders approved an increase in the Company’s authorized common stock par value $0.001 per share, from 150,000,000 shares to 200,000,000 shares. At September 30, 2020 and December 31, 2019, 103,648,224 and 103,397,033 shares, respectively, of common stock were issued and outstanding. Each holder of common stock is entitled to one vote of each share of common stock held of record on all matters on which stockholders generally are entitled to vote. 2020 Shelf Registration Statement On July 23, 2020, the Company filed a shelf registration statement with the SEC to sell up to $200 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the “2020 Shelf Registration Statement”). The 2020 Shelf Registration Statement (file no.
333-240052) was declared effective by the SEC on July 31, 2020. As of the date of this report, no offerings have been completed under the Company’s 2020 Shelf Registration Statement. |
Stock Compensation |
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Share-based Payment Arrangement [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Stock Compensation |
For the three and nine-month periods ended September 30, 2020 and 2019, the Company recorded stock-based compensation expense as follows:
Stock Options As of September 30, 2020, there were outstanding stock options to purchase 11,790,335 shares of common stock, of which stock options to purchase 6,833,809 shares of common stock were exercisable as of September 30, 2020. During the three and nine-month periods ended September 30, 2020, the Company granted seven-year term options to purchase an aggregate of 10,000 and 1,005,000 shares, respectively, of the Company’s common stock to employees and directors. The Company recorded stock-based compensation related to stock options totaling $1,345,962 and $4,356,739, respectively, during the three and nine-month periods ended September 30, 2020. During the three and nine-month periods ended September 30, 2020, respectively, 348,163 and 1,542,992 options vested. During the three and nine-month periods ended September 30, 2019, the Company granted seven-year term options to purchase an aggregate of 172,500 and 484,500 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $817,060 and $2,675,467, respectively, during the three and nine-month periods ended September 30, 2019. During the three and nine-month periods ended September 30, 2019, respectively, 74,998 and 1,365,827 options vested. During the three and nine-month periods ended September 30, 2020, options to purchase 215,097 shares and 240,096 shares, respectively, of the Company’s common stock were exercised, with proceeds of $631,048 and $693,398 respectively, to the Company. During the three and nine-month periods ended September 30, 2019, options to purchase 108,332 shares and 298,332 shares, respectively, of the Company’s common stock were exercised, with proceeds of $256,062 and $537,962 respectively, to the Company. During both the three and nine-month periods ended September 30, 2019, options to purchase 6,666 shares of the Company’s common stock were exercised on a “cashless” basis , resulting in the issuance of an aggregate of 3,444 shares of the Company’s common stock. As of September 30, 2020, there was approximately $7.8 million of unrecognized compensation expense related to non-vested stock option awards granted under the 2014 and 2018 Stock Incentive Plans. The cost is expected to be recognized over a weighted average period of approximately 2.1 years.Restricted Stock Units The Company granted zero and 30,000 restricted stock units during the three and nine-month periods ended September 30, 2020, respectively. There were no restricted stock units granted during the three and nine-month periods ended September 30, 2019. During the three and nine-month periods ended September 30, 2020, the Company recorded non-cash stock-based compensation expense related to restricted stock units totaling $131,884 and $434,920, respectively. No stock-based compensation related to restricted stock units was recorded during the three and nine-month periods ended September 30, 2019.As of September 30, 2020, there was approximately $1.2 million of unrecognized compensation expense related to
non-vested restricted stock units granted under the 2018 Stock Incentive Plan. The cost is expected to be recognized over a weighted average period of approximately 2.2 years. |
Subsequent Events |
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Subsequent Events [Abstract] | |||
Subsequent Events |
On October 6, 2020, the United States Patent and Trademark Office issued U.S. Patent 10,793,893 (the ‘893 patent). The ’893 patent is exclusively licensed to the Company and covers certain methods for treating disease using amifampridine drug products, including Catalyst’s Firdapse ® product, in patients who are slow metabolizers of amifampridine. On October 19, 2020, the Company announced that it has filed a lawsuit in the U.S. District Court for New Jersey against Jacobus, and a lawsuit in the U.S. District Court for the Western District of Pennsylvania against PantherRx Rare LLC (PantherRx) for infringement of the ’893 patent. The lawsuit arises from Jacobus’ and PantherRx’s sales and marketing of Ruzurgi ® (amifampridine, 10 mg). The lawsuit alleges that the Ruzurgi® product infringes the ‘893 patent when administered in accordance with its product labeling. The lawsuit seeks damages and injunctive relief to prevent further marketing of Ruzurgi® in violation of the ‘893 patent. There can be no assurance as to the outcome of this matter. |
Basis of Presentation and Significant Accounting Policies (Policies) |
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INTERIM FINANCIAL STATEMENTS |
In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2019 included in the 2019 Annual Report on Form
10-K filed by the Company with the SEC. The results of operations for the nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for any future period or for the full 2020 fiscal year. |
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PRINCIPLES OF CONSOLIDATION. |
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USE OF ESTIMATES |
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CASH AND CASH EQUIVALENTS |
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INVESTMENTS |
The short-term bond funds and U.S. Treasuries held at September 30, 2020 are classified as available-for-sale non-current investments in its consolidated balance sheets. The Company records available-for-sale available-for-sale available-for-sale The Company previously owned a short-term bond fund that was classified as trading securities. Trading securities are recorded at fair value based on the closing market price of the security. For trading securities, the Company recognized realized gains and losses and unrealized gains and losses to earnings. At September 30, 2020 and December 31, 2019, there were no investments classified as trading securities, as the Company sold its interest in the short-term bond fund classified as trading in 2019. There was no realized or unrealized gain (loss) on trading securities for the three and nine months ended September 30, 2020. Realized losses on trading securities during the three and nine months ended September 30, 2019 were $0 and $4,980, respectively. Unrealized gain (loss) on trading securities was $0 and $89,405 for the three and nine months ended September 30, 2019 , respectively, and is included in other income, net in the accompanying consolidated statements of operations. |
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ACCOUNTS RECEIVABLE, NET |
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INVENTORY |
Products that have been approved by the FDA or other regulatory authorities, such as Firdapse ® , are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of Firdapse® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”. The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage. |
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PREPAID EXPENSES AND OTHER CURRENT ASSETS |
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FAIR VALUE OF FINANCIAL INSTRUMENTS |
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FAIR VALUE MEASUREMENTS |
Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment, and considers factors specific to the asset or liability.
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OPERATING LEASES |
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REVENUE RECOGNITION |
To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to arrangements that meet the definition of a contract under Topic 606, including when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below. The Company also may generate revenues from payments received under a collaborative agreement. Collaborative agreement payments may include nonrefundable fees at the inception of the agreements, milestone and event-based payments for specific achievements designated in the collaborative agreements, and/or royalties on sales of products resulting from a collaborative arrangement. For a complete discussion of accounting for collaborative arrangements, see Revenues from Collaborative Arrangements below. Product Revenue, Net: ® to the Customer (its exclusive distributor) who subsequently resells Firdapse® to both a small group of SPs who have exclusive contracts with the Company to distribute the Company’s products to patients and potentially to medical centers or hospitals on an emergency basis. In addition to the distribution agreement with its Customer, the Company enters into arrangements with health care providers and payors that provide for government-mandated and/or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of the Company’s products.The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days. Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods, and are recorded in cost of sales. If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three and nine month periods ended September 30, 2020 and 2019. During the three and nine months ended September 30, 2020 and 2019, all of the Company’s sales were to its Customer. Reserves for Variable Consideration: These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts. The amount of variable consideration which is included in the transaction price may be constrained, and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplated application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of September 30, 2020 and, therefore, the transaction price was not reduced further during the three and nine months ended September 30, 2020 and 2019. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. Trade Discounts and Allowances: ® to the Customer, these payments are classified in selling, general and administrative expenses in the Company’s consolidated statement of operations and comprehensive income (loss). However, if the Company has determined such services received to date are not distinct from the Company’s sale of products to the Customer, these payments have been recorded as a reduction of revenue within the consolidated statement of operations and comprehensive income (loss) through September 30, 2020 and 2019, as well as a reduction to accounts receivable, net on the consolidated balance sheets.Funded Co-pay Assistance Program:co-pay assistance program intended to provide financial assistance to qualified commercially-insured patients. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that the Company expects to receive associated with Firdapse® that has been recognized as revenue, but remains in the distribution channel at the end of each reporting period. These payments are considered payable to the Customer and the related reserve is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability which is included in accrued expenses and other current liabilities in the consolidated balance sheets.Product Returns: Provider Chargebacks and Discounts: Government Rebates: Bridge and Patient Assistance Programs: ® to uninsured patients who satisfy pre-established criteria for either the Bridge Program or the Patient Assistance Program. Patients who meet the Bridge Program eligibility criteria and are transitioning from investigational product while they are waiting for a coveragedetermination, or later, for patients whose access is threatened by the complications arising from a change of insurer may receive a temporary supply of free Firdapse ® while the Company is determining the patient’s third-party insurance, prescription drug benefit or other third-party coverage for Firdapse® . The Patient Assistance Program provides free Firdapse® for longer periods of time for those who are uninsured or functionally uninsured with respect to Firdapse® because they are unable to obtain coverage from their payor despite having health insurance, to the extent allowed by applicable law. The Company does not recognize any revenue related to these free products and the associated costs are classified in selling, general and administrative expenses in the Company’s consolidated statements of operations and comprehensive income (loss). Revenues from Collaborative Arrangements: ® (vigabatrin) tablets as well as the commercialization of Firdapse® in Canada. Pursuant to the terms of these agreements, collaborators could be required to make various payments to the Company, including upfront license fees, milestone payments based on achievement of regulatory approvals, and royalties on sales of products resulting from the collaborative agreement. Nonrefundable upfront license fees are recognized upon receipt as persuasive evidence of an arrangement exists, the price to the collaborator is fixed or determinable and collectability is reasonably assured. In the third quarter of 2020, an upfront fee was earned under the collaboration agreement for the commercialization of Firdapse ® in Canada. The collaborative agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments in accordance with the provisions of Accounting Standards Update (ASU) No. 2010-17, Revenue Recognition – Milestone Method (“Milestone Method of Accounting”). The Company recognizes consideration that is contingent upon the achievement of a milestone in its entirety as revenue in the period in which the milestone is achieved only if the milestone is substantive in its entirety. A milestone is considered substantive when it meets all of the following criteria:1. The consideration is commensurate with either the entity’s performance to achieve the milestone or the enhancement of the value of the delivered item(s) as a result of a specific outcome resulting from the entity’s performance to achieve the milestone; 2. The consideration relates solely to past performance; and 3. The consideration is reasonable relative to all of the deliverables and payment terms within the arrangement. A milestone is defined as an event (i) that can only be achieved based in whole or in part on either the entity’s performance or on the occurrence of a specific outcome resulting from the entity’s performance, (ii) for which there is substantive uncertainty at the date the arrangement is entered into that the event will be achieved, and (iii) that would result in additional payments being due to the vendor. The Company believes that achievement of the milestones will be substantive and there will be no substantive uncertainty once the milestones are achieved. No milestones were achieved in the three and nine months ended September 30, 2020 and 2019. In arrangements where the Company does not deem the collaborator to be a customer, payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments. Under the arrangements, the Company will receive royalty reports 60 days after quarter end from one collaborator, and within nine days after quarter end from the other collaborator. Since the Company will receive royalty reports 60 days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter following the quarter in which the corresponding sales occurred. In instances where royalty reports are received within nine days after quarter end, royalty revenue from sales of collaboration products by our collaborator will be recognized in the quarter in which the sales occurred. For the three and nine months ended September 30, 2020 and 2019, there was no royalty revenue from sales of the collaborative product. Refer to Note 7 (Collaborative Arrangement), for further discussion on the Company’s collaborative arrangement. |
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RESEARCH AND DEVELOPMENT |
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STOCK-BASED COMPENSATION |
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CONCENTRATION OF CREDIT RISK |
The Company sells its product in the United States through an exclusive distributor (its Customer) to specialty pharmacies. Therefore, its distributor and specialty pharmacies account for all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions. The Company currently has a single product with limited commercial sales experience, which makes it difficult to evaluate its current business, predict its future prospects and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, Firdapse ® , and expects Firdapse® to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to effectively commercialize Firdapse® . The Company relies exclusively on third parties to formulate and manufacture Firdapse ® and its drug candidates. The commercialization of Firdapse® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and for the commercialization of Firdapse® . If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of its drugs. |
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ROYALTIES |
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INCOME TAXES |
The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-than-not threshold, the amount recognized in the financial statements is the largest benefit that has a greater than 50 percent likelihood of being realized upon ultimate settlement with the relevant tax authority. The Company is subject to income taxes in the U.S. federal jurisdiction and various state jurisdictions. Tax regulations within each jurisdiction are subject to the interpretation of the related tax laws and regulations and require significant judgment to apply. The Company is not subject to U.S. federal, state and local tax examinations by tax authorities for years before 2017 . If the Company were to subsequently record an unrecognized tax benefit, associated penalties and tax related interest expense would be reported as a component of income tax expense.On March 27, 2020, the Coronavirus Aid, Relief, and Economic Security (CARES) Act was signed into law making several changes to the Internal Revenue Code. The changes include, but are not limited to: increasing the limitation on the amount of deductible interest expense, allowing companies to carryback certain net operating losses, and increasing the amount of net operating loss carryforwards that corporations can use to offset taxable income. The tax law changes in the CARES Act did not have a material impact on the Company’s income tax provision. |
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COMPREHENSIVE INCOME (LOSS) |
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NET INCOME (LOSS) PER COMMON SHARE |
Diluted net income (loss) per common share is computed by dividing net income (loss) by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period. The following table reconciles basic and diluted weighted average common shares:
Outstanding common stock equivalents totaling approximately 5.4 million and 4.8 million, were excluded from the calculation of diluted net income (loss) per common share for the three and nine months ended September 30, 2020 as their effect would be anti-dilutive. For the three and nine months ended September 30, 2019, approximately 0.4 million and 2.9 million shares of outstanding stock options were excluded from the calculation of diluted net income (loss) per common share as their effect would be anti-dilutive. |
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RECLASSIFICATIONS |
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RECENTLY ISSUED ACCOUNTING STANDARDS |
In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments — Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments. available-for-sale In August 2018, the FASB issued ASU 2018-15, Intangibles – Goodwill and Other – Internal-Use Software (Subtopic 350-40), Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract.internal-use software (and hosting arrangements that include an internal-use software license). Accordingly, the amendments in this update require an entity in a hosting arrangement that is a service contract to follow the guidance in Subtopic 350-40 to determine which implementation costs to capitalize as an asset related to the service contract and which costs to expense. The Company adopted the new standard on January 1, 2020 and applied prospectively to all implementation costs incurred after the date of adoption. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements. |
Basis of Presentation and Significant Accounting Policies (Tables) |
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Accounting Policies [Abstract] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Fair Value Measurement Specific to Assets or Liability |
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Basic and Dilutive Weighted Average Common Shares | The following table reconciles basic and diluted weighted average common shares:
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Investments (Tables) |
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Investments, Debt and Equity Securities [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Summary of Available-for-Sale Investments by Security type | Available-for-sale
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Estimated Fair Values of Available for Sale Securities | The estimated fair values of
available-for-sale September
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Prepaid Expenses and Other Current Assets (Tables) |
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Prepaid expenses and other current assets | Prepaid expenses and other current assets consist of the following:
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Operating Leases (Tables) |
9 Months Ended | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Sep. 30, 2020 | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Disclosure of Operating Leases [Abstract] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Lease, Cost | The components of lease expense were as follows:
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Schedule of Supplemental Cash Flow Information Related To Lease | Supplemental cash flow information related to leases was as follows:
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Schedule of Supplemental Balance Sheet related To Lease | Supplemental balance sheet information related to leases was as follows:
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Lessee, Operating Lease, Liability, Maturity | Remaining payments of lease liabilities as of September 30, 2020 were as follows:
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Accrued Expenses and Other Liabilities (Tables) |
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Payables and Accruals [Abstract] | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following:
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Stock Compensation (Tables) |
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Share-based Payment Arrangement [Abstract] | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Stock-Based Compensation Expense | For the three and nine-month periods ended September 30, 2020 and 2019, the Company recorded stock-based compensation expense as follows:
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Basis of Presentation and Significant Accounting Policies - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares |
3 Months Ended | 9 Months Ended | ||
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Sep. 30, 2020 |
Sep. 30, 2019 |
Sep. 30, 2020 |
Sep. 30, 2019 |
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Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | ||||
Basic weighted average common shares outstanding | 103,535,431 | 102,974,105 | 103,452,025 | 102,864,571 |
Effect of dilutive securities | 2,780,810 | 4,071,129 | 2,934,592 | 2,957,038 |
Dilutive weighted average common shares outstanding | 106,316,241 | 107,045,234 | 106,386,617 | 105,821,609 |
Investments - Additional Information (Detail) - USD ($) |
3 Months Ended | 9 Months Ended | ||
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Sep. 30, 2020 |
Sep. 30, 2019 |
Sep. 30, 2020 |
Sep. 30, 2019 |
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Investments, Debt and Equity Securities [Abstract] | ||||
Realized gains losses from available for sale securities | $ 0 | $ 0 | $ 0 | $ 0 |
Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($) |
9 Months Ended | 12 Months Ended |
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Sep. 30, 2020 |
Dec. 31, 2019 |
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Net Investment Income [Line Items] | ||
Amortized cost | $ 105,001,320 | $ 64,929,745 |
Gross Unrealized Gains | 9,505 | |
Gross Unrealized Losses | (6,471) | |
Estimated Fair Value | 104,994,849 | 64,939,250 |
U.S. Treasuries - ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 4,999,587 | |
Gross Unrealized Gains | 7,463 | |
Estimated Fair Value | 5,007,050 | |
U.S Treasury Bond Securities Cash Equivalents [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 94,993,295 | 59,930,158 |
Gross Unrealized Gains | 2,042 | |
Gross Unrealized Losses | (1,195) | |
Estimated Fair Value | 94,992,100 | $ 59,932,200 |
Bond Funds – ST [Member] | ||
Net Investment Income [Line Items] | ||
Amortized cost | 10,008,025 | |
Gross Unrealized Losses | (5,276) | |
Estimated Fair Value | $ 10,002,749 |
Investment - Estimated Fair Values of Available for Sale Securities (Detail) - USD ($) |
Sep. 30, 2020 |
Dec. 31, 2019 |
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Investments, Debt and Equity Securities [Abstract] | ||
Estimated Fair Value | $ 104,994,849 | $ 64,939,250 |
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) |
Sep. 30, 2020 |
Dec. 31, 2019 |
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Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid manufacturing costs | $ 3,268,032 | $ 1,526,013 |
Prepaid insurance | 247,610 | 1,263,129 |
Prepaid subscriptions fees | 561,430 | 501,251 |
Prepaid research fees | 472,093 | 481,057 |
Prepaid commercialization expenses | 168,611 | 62,959 |
Due from collaborative arrangements | 306,678 | |
Other | 589,598 | 516,665 |
Total prepaid expenses and other current assets | $ 5,614,052 | $ 4,351,074 |
Operating Leases - Operating Leases (Detail) - USD ($) |
3 Months Ended | 9 Months Ended |
---|---|---|
Sep. 30, 2020 |
Sep. 30, 2020 |
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Disclosure of Operating Leases [Abstract] | ||
Operating lease cost | $ 61,111 | $ 200,624 |
Operating Leases - Schedule of Supplemental Cash Flow Information Related To Lease (Detail) |
9 Months Ended |
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Sep. 30, 2020
USD ($)
| |
Cash paid for amounts included in the measurement of lease liabilities: | |
Operating cash flows | $ 253,024 |
Right-of-use assets obtained in exchange for lease obligations: | |
Operating leases | $ 37,528 |
Operating Leases -Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($) |
Sep. 30, 2020 |
Dec. 31, 2019 |
---|---|---|
Disclosure of Operating Leases [Line Items] | ||
Operating lease right-of-use assets | $ 12,167 | $ 793,252 |
Other current liabilities | 114,563 | 300,518 |
Operating lease liabilities, net of current portion | $ 647,532 | |
Total operating lease liabilities | $ 114,563 | |
Weighted average remaining lease term | 3 months 18 days | |
Weighted average discount rate | 3.68% |
Operating Leases -Lessee, Operating Lease, Liability, Maturity (Detail) |
Sep. 30, 2020
USD ($)
|
---|---|
Disclosure of Operating Leases [Line Items] | |
2020 (remaining three months) | $ 86,582 |
2021 | 28,860 |
2022 | |
Total lease payments | 115,442 |
Less imputed interest | (879) |
Total | $ 114,563 |
Operating Leases - Additional Information (Detail) |
9 Months Ended |
---|---|
Sep. 30, 2020
USD ($)
ft²
| |
Disclosure of Operating Leases [Line Items] | |
Finance Lease Obligations | $ | $ 0 |
Lessor, Operating Lease, Option to Extend | 1 year |
Lessor, Operating Lease, Option to Terminate | 1 year |
Before agreement of company leased spaces | 7,800 |
After agreement of company leased spaces | 10,700 |
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) |
Sep. 30, 2020 |
Dec. 31, 2019 |
---|---|---|
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 643,578 | $ 1,183,513 |
Accrued professional fees | 1,923,164 | 1,241,526 |
Accrued compensation and benefits | 2,877,077 | 3,064,645 |
Accrued license fees | 7,840,955 | 8,751,991 |
Accrued purchases | 1,332,190 | 1,313,310 |
Accrued contributions | 1,535,000 | |
Operating lease liability | 114,563 | 300,518 |
Accrued variable consideration | 859,399 | 884,764 |
Accrued income tax | 566,075 | 1,533,696 |
Other | 69,608 | 172,332 |
Current accrued expenses and other liabilities | 16,226,609 | 19,981,295 |
Lease liability - non-current | 647,532 | |
Non-current accrued expenses and other liabilities | 647,532 | |
Total accrued expenses and other liabilities | $ 16,226,609 | $ 20,628,827 |
Commitments and Contingencies - Additional Information (Detail) |
1 Months Ended |
---|---|
May 21, 2019
USD ($)
| |
Settlement Agreement [Member] | Other Income [Member] | |
Commitments [Line Items] | |
Litigation Settlement Received | $ 100,000 |
Agreements - Additional Information (Detail) - License Agreement with BioMarin [Member] $ in Millions |
9 Months Ended |
---|---|
Sep. 30, 2020
USD ($)
| |
License Agreement [Line Items] | |
Date on which strategic collaboration is entered into | Oct. 26, 2012 |
Royalty agreement period | 7 years |
Net sales royalty threshold | $ 100 |
Minimum [Member] | |
License Agreement [Line Items] | |
Percentage of royalty on net sales | 7.00% |
Maximum [Member] | |
License Agreement [Line Items] | |
Percentage of royalty on net sales | 10.00% |
Income Taxes - Additional Information (Detail) |
9 Months Ended |
---|---|
Sep. 30, 2020
USD ($)
| |
Income Tax Disclosure [Abstract] | |
Deferred tax assets | $ 31,347,442 |
Deferred tax assets recognised in income statement | $ 31,300,000 |
Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail) |
Sep. 30, 2020
Vote
$ / shares
shares
|
Aug. 20, 2020
$ / shares
shares
|
Dec. 31, 2019
$ / shares
shares
|
---|---|---|---|
Stockholders Equity [Line Items] | |||
Preferred stock, shares authorized | 5,000,000 | 5,000,000 | |
Preferred stock, par value | $ / shares | $ 0.001 | $ 0.001 | |
Preferred stock, shares outstanding | 0 | 0 | |
Common stock, shares authorized | 200,000,000 | 150,000,000 | |
Common stock, par value | $ / shares | $ 0.001 | $ 0.001 | $ 0.001 |
Common stock, shares issued | 103,648,224 | 103,397,033 | |
Common stock, shares outstanding | 103,648,224 | 103,397,033 | |
Number of votes entitled for each share of common stock | Vote | 1 | ||
Maximum [Member] | |||
Stockholders Equity [Line Items] | |||
Common stock, shares authorized | 150,000,000 | ||
Minimum [Member] | |||
Stockholders Equity [Line Items] | |||
Common stock, shares authorized | 200,000,000 |
Stockholders' Equity (2020 Shelf Registration Statement) - Additional Information (Detail) $ in Millions |
Jul. 23, 2020
USD ($)
|
---|---|
2020 Shelf Registration Statement [Member] | |
Stockholders' Equity [Line Items] | |
Maximum dollar amount of common stock to be issued under shelf registration statement | $ 200 |
Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($) |
3 Months Ended | 9 Months Ended | ||
---|---|---|---|---|
Sep. 30, 2020 |
Sep. 30, 2019 |
Sep. 30, 2020 |
Sep. 30, 2019 |
|
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 1,477,846 | $ 817,060 | $ 4,791,659 | $ 2,675,467 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | 399,247 | 242,867 | 1,238,521 | 803,800 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 1,078,599 | $ 574,193 | $ 3,553,138 | $ 1,871,667 |
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