Table of Contents
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 10-Q
[Mark One]
| QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the Quarterly Period Ended June 30, 2021
OR
| TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
Commission File
No. 001-33057
(Exact name of registrant as specified in its charter)
| (State or other jurisdiction of incorporation or organization) |
(IRS Employer Identification No.) | |
| |
||
(Address of principal executive offices) |
(Zip Code) |
Registrant’s telephone number, including area code: (305 ) 420-3200
Securities registered pursuant to Section 12(b) of the Act:
| Title of Each Class |
Ticker Symbol |
Name of Exchange on Which Registered |
Indicate by checkmark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such report(s), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation Yes ☒ No ☐
S-T
during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a
non-accelerated
filer, a smaller reporting company or an emerging growth company. See definitions of “accelerated filer”, “large accelerated filer”, “smaller reporting company” and “emerging growth company” in Rule 12b-2
of the Exchange Act: | Large accelerated filer | ☐ | ☒ | ||||
Non-accelerated filer |
☐ | Smaller reporting company | ||||
| Emerging growth company | ||||||
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule ☒
12b-2
of the Exchange Act). Yes ☐ No
Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date 102,978,461 shares of common stock, $0.001 par value per share, were outstanding as of August 5, 2021.
CATALYST PHARMACEUTICALS, INC.
INDEX
PART I. FINANCIAL INFORMATION |
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| 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. |
24 |
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| Item 3. |
35 |
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| Item 4. |
36 |
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PART II. OTHER INFORMATION |
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| Item 1. |
36 |
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| Item 1A. |
37 |
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| Item 2. |
38 |
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| Item 3. |
38 |
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| Item 4. |
38 |
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| Item 5. |
38 |
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| Item 6. |
38 |
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39 |
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2
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED BALANCE SHEETS
(in thousands, except share data)
June 30, 2021 |
December 31, 2020 |
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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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| Operating lease right-of-use |
— | |||||||
| Property and equipment, net |
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| Deferred tax assets |
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| Deposits |
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| Total assets |
$ | $ | ||||||
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| 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 |
— | |||||||
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| Total liabilities |
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| Commitments and contingencies (Note 10) |
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| Stockholders’ equity: |
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| Preferred stock, $ |
— | |||||||
| Common stock, $ shares outstanding at June 30, 2021 and December 31, 2020, respectively |
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| Additional paid-in capital |
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| Accumulated deficit |
( |
) | ( |
) | ||||
| Accumulated other comprehensive income (loss) |
( |
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| Total stockholders’ equity |
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| Total liabilities and stockholders’ equity |
$ | $ | ||||||
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The accompanying notes are an integral part of these consolidated financial statements.
3
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (unaudited)
(in thousands, except share data)
For the Three Months Ended June 30, |
For the Six Months Ended June 30, |
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2021 |
2020 |
2021 |
2020 |
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| Revenues: |
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| Product revenue, net |
$ | $ | $ | $ | ||||||||||||
| License and other revenue |
— | — | ||||||||||||||
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| 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 |
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| Other income, net |
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| Net income before income taxes |
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| Provision for income taxes |
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| Net income |
$ | $ | $ | $ | ||||||||||||
| Net income 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 |
$ | $ | $ | $ | ||||||||||||
| Other comprehensive income: |
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| Unrealized gain (loss) on available-for-sale |
( |
) | ( |
) | ( |
) | ||||||||||
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| Comprehensive income |
$ | $ | $ | $ | ||||||||||||
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The accompanying notes are an integral part of these consolidated financial statements.
4
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY (unaudited)
For the three and six months ended June 30, 2021 and 2020
(in thousands)
Preferred Stock |
Common Stock |
Additional Paid-in Capital |
Accumulated Deficit |
Accumulated Other Comprehensive Gain (Loss) |
Total |
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Shares |
Amount |
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| Balance at December 31, 2020 |
$ | — | $ | $ | $ | ( |
) | $ | $ | |||||||||||||||||||
| Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
| Exercise of stock options for common stock |
— | — | — | — | ||||||||||||||||||||||||
| Amortization of restricted stock for services |
— | — | — | — | — | |||||||||||||||||||||||
| Repurchase of common stock |
— | ( |
) | — | — | ( |
) | — | ( |
) | ||||||||||||||||||
| Other comprehensive gain (loss) |
— | — | — | — | — | ( |
) | ( |
) | |||||||||||||||||||
| Net income |
— | — | — | — | — | |||||||||||||||||||||||
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| Balance at March 31, 2021 |
— | ( |
) | ( |
) | |||||||||||||||||||||||
| Issuance of stock options for services |
— | — | — | — | — | |||||||||||||||||||||||
| Exercise of stock options for common stock |
— | — | — | — | ||||||||||||||||||||||||
| Amortization of restricted stock for services |
— | — | — | — | — | |||||||||||||||||||||||
| Repurchase of common stock |
— | ( |
) | ( |
) | — | ( |
) | — | ( |
) | |||||||||||||||||
| Issuance of common stock upon vesting of restricted stock units, net |
— | — | ( |
) | — | — | ( |
) | ||||||||||||||||||||
| Other comprehensive gain (loss) |
— | — | — | — | — | |||||||||||||||||||||||
| Net income |
— | — | — | — | — | |||||||||||||||||||||||
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| Balance at June 30, 2021 |
$ | — | $ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||||
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Preferred Stock |
Common Stock |
Additional Paid-in Capital |
Accumulated Deficit |
Accumulated Other Comprehensive Gain (Loss) |
Total |
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Shares |
Amount |
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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 |
— | — | — | — | — | |||||||||||||||||||||||
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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 |
— | — | — | — | — | |||||||||||||||||||||||
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| Balance at June 30, 2020 |
$ | — | $ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||||
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The accompanying notes are an integral part of these consolidated financial statements.
5
CATALYST PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited)
(in thousands)
For the Six Months Ended June 30, |
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2021 |
2020 |
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| Operating Activities: |
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| Net income |
$ | $ | ||||||
| Adjustments to reconcile net income to net cash provided by (used in) operating activities: |
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| Depreciation |
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| Stock-based compensation |
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| Deferred taxes |
— | |||||||
| Change in accrued interest and accretion of discount on investments |
( |
) | ( |
) | ||||
| Reduction in the carrying amount of right-of-use asset |
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| (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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| Repurchase of common stock |
( |
) | — | |||||
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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 |
$ | ( |
) | $ | ( |
) | ||
| Operating lease liabilities arising from obtaining right-of-use |
$ | $ | — | |||||
The accompanying notes are an integral part of these consolidated financial statements.
6
CATALYST PHARMACEUTICALS, INC.
NOTES TO UNAUDITED CONSOLIDATED FINANCIAL STATEMENTS
1. |
Organization and Description |
Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”) is a commercial-stage patient-centric biopharmaceutical company focused on
in-licensing,
developing and commercializing novel high-quality medicines for patients living with rare diseases. On November 28, 2018, the U.S. Food and Drug Administration, or 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 adult patients in Canada with LEMS. On October 28, 2020, the Company launched Firdapse®
in Canada for the treatment of patients with LEMS through a license and supply agreement with KYE Pharmaceuticals. 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 until the second quarter of 2019, when it started reporting net income. The Company has been able to fund its cash needs to date through offerings of its securities and revenues from sales of its product. See Note 13 (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 planned operations for at least the next 12 months from the issuance date of this Form
10-Q.
The Company may raise 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 drug 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 and if 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 numerous aspects of the coronavirus
(COVID-19)
pandemic that have adversely affected the Company’s business since the beginning of the pandemic. The Company closely monitors the impact of the pandemic on all aspects of its business and takes steps, wherever possible, to lessen those impacts. However, the Company is unable to predict the impact that the coronavirus pandemic will have on its business in future periods. 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. |
7
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
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, 2020 included in the 2020 Annual Report on
Form 10-K
filed by the Company with the SEC. The results of operations for the six months ended June 30, 2021 are not necessarily indicative of the results to be expected for any future period or for the full 2021 fiscal year. b. |
PRINCIPLES OF CONSOLIDATION |
c. |
USE OF ESTIMATES. |
| d. |
CASH AND CASH EQUIVALENTS. |
e. |
INVESTMENTS |
The short-term bond funds and U.S. Treasuries held at June 30, 2021 are classified as securities. The short-term bond funds are classified as current assets, which reflects management’s intention to use the proceeds from the sale of these investments to fund the Company’s operations, as necessary. The Company classifies U.S. Treasuries with stated maturities of greater than three months and less than one year in short-term investments, U.S. Treasuries with stated maturities greater than one year are classified as no
available-for-sale
non-current
investments in its consolidated balance sheets. There are non-current
investments as of June 30, 2021 and December 31, 2020. The Company records securities at fair value with unrealized gains and losses reported in accumulated other comprehensive income (loss) in stockholders’ equity. Realized gains and losses are included in other income, net and are derived using the specific identification method for determining the cost of securities sold. Interest income is recognized when earned and is included in other income, net in the consolidated statements of operations and comprehensive income. The Company recognizes a charge when the declines in the fair value below the amortized cost basis of its securities are judged to be other-than-temporary. The Company considers various factors in determining whether to recognize an other-than-temporary charge, including whether the Company intends to sell the security or whether it is more likely than not that the Company would be required to sell the security before recovery of the amortized cost basis. The Company has not recorded any other-than-temporary impairment charges on its securities. See Note 3 (Investments).
available-for-sale
available-for-sale
available-for-sale
f. |
ACCOUNTS RECEIVABLE, NET. |
8
2. |
Basis of Presentation and Significant |
| |
g. |
INVENTORY. work-in-process first-in, first out (FIFO) flow of goods. The Company began capitalizing inventories post FDA approval of Firdapse® 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. 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 June 30, 2021 inventory consisted of raw materials, work-in process and finished goods. As of December 31, 2020, inventory consisted mainly of work-in-process |
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.
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. |
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.
9
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
Fair Value Measurements at Reporting Date Using (in thousands) |
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Balances as of June 30, 2021 |
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: |
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| Money market funds |
$ | $ | $ | — | $ | — | ||||||||||
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| U.S. Treasuries |
$ | $ | $ | — | $ | — | ||||||||||
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| Short-term investments: |
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| Short-term bond funds |
$ | $ | $ | — | $ | — | ||||||||||
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Balances as of December 31, 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: |
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| Money market funds |
$ | $ | $ | — | $ | — | ||||||||||
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| U.S. Treasuries |
$ | $ | — | $ | $ | — | ||||||||||
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| Short-term investments: |
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| Short-term bond funds |
$ | $ | $ | — | $ | — | ||||||||||
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k. |
OPERATING LEASES. right-of-use non-lease components, which are accounted for separately. |
l. |
SHARE REPURCHASES. |
The Company accounts for share repurchases by charging the excess of
the
repurchase price over the repurchased common stock’s par value entirely to accumulated deficit. All repurchased shares are retired and become authorized but unissued shares. The Company accrues for the shares purchased under the share repurchase plan based on the trade date. The Company may terminate or modify its share repurchase program at any time. m. |
REVENUE RECOGNITION. |
Product Revenues:
The Company recognizes revenue when its customer obtains title of the promised goods, in an amount that reflects the consideration to which the Company expects to be entitled in exchange for these goods. The Company had no contracts with customers until the FDA approved Firdapse
®
in November 2018. Subsequent to receiving FDA approval, the Company entered into an arrangement with one distributor (the “Customer”), which 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®
. 10
2. |
Basis of Presentation and |
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 collaborative and license agreements. Collaborative and license agreement payments may include nonrefundable fees at the inception of the agreements, contingent payments for specific achievements designated in the agreements, and/or net profit-sharing payments on sales of products resulting from the collaborative and license arrangements. For a complete discussion of accounting for collaborative and licensing arrangements, see Revenues from Collaboration and Licensing 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 or upon dispense to patient). 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 six months ended June 30, 2021 and 2020.
During the three and six months ended June 30, 2021 and 2020, all of the Company’s sales of Firdapse
®
in the United States 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.
11
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
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 contemplates 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 June 30, 2021 and, therefore, the transaction price was not reduced further during the three and six months ended June 30, 2021 and 2020. 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:
®
June 30,
2020, as well as a reduction to accounts receivable, net on the consolidated balance sheets. Prompt Payment Discounts:
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 third-party vendor 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:
12
2. |
Basis of Presentation |
Government Rebates:
Bridge and Patient Assistance Programs:
®
free of charge 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 coverage determination, 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®
®
. The Patient Assistance Program provides 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. Revenues from Collaboration and Licensing Arrangements:
The Company analyzes license and collaboration arrangements pursuant to FASB ASC Topic 808, Collaborative Arrangement Guidance and Consideration, (“ASC 808”) to assess whether such arrangements, or transactions between arrangement participants, involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities or are more akin to a vendor-customer relationship. In making this evaluation, the Company considers whether the activities of the collaboration are considered to be distinct and deemed to be within the scope of the collaborative arrangement guidance or if they are more reflective of a vendor-customer relationship and, therefore, within the scope of FASB ASC Topic 606, Revenue from Contracts with Customers. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement.
For elements of collaboration arrangements that are not accounted for pursuant to guidance in
Topic
606, an appropriate recognition method is determined and applied consistently, generally by analogy to the revenue from contracts with customers guidance. Pursuant to
Topic
606, for arrangements or transactions between arrangement participants determined to be within the scope of the contracts with customers guidance, the Company performs the following steps to determine the appropriate amount of revenue to be recognized as the Company fulfills its obligations: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company evaluates the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determines whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract. Goods or services that meet these criteria are considered distinct performance obligations. The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration.
13
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
The agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments as variable consideration in accordance with Topic 606. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential transaction price and the likelihood that the transaction price will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and, if so, these options are considered performance obligations.
Revenue is recognized when, or as, the Company satisfies a performance obligation by transferring a promised good or service to a customer. An asset is transferred when, or as, the customer obtains control of that asset.
After contract inception, the transaction price is reassessed at every period end and updated for changes such as resolution of uncertain events. Any change in the overall transaction price is allocated to the performance obligations based on the same methodology used at contract inception.
The Company recognizes sales-based royalties or net profit-sharing when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty or net profit-sharing has been satisfied.
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.
Refer to Note 9 (Collaboration and Licensing Arrangements), for further discussion on the Company’s collaborative and licensing arrangements.
n. |
RESEARCH AND DEVELOPMENT. |
o. |
STOCK-BASED COMPENSATION. |
p. |
CONCENTRATION OF RISK. |
The Company sells its product in the United States through an exclusive distributor (its Customer) to SPs. Therefore, its distributor and SPs 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®
. 14
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
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. q. |
ROYALTIES. |
r. |
INCOME |
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.s. |
COMPREHENSIVE INCOME. available-for-sale |
t. |
NET INCOME PER COMMON SHARE. |
Diluted net income per common share is computed by dividing net income 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:
For the Three Months Ended June 30, |
For the Six Months Ended June 30, |
|||||||||||||||
2021 |
2020 |
2021 |
2020 |
|||||||||||||
| Basic weighted average common shares outstanding |
||||||||||||||||
| Effect of dilutive securities |
||||||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Diluted weighted average common shares outstanding |
||||||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
Outstanding common stock equivalents totaling approximately 4.9 million and 5.1 million, were excluded from the calculation of diluted net income per common share for the three and six months ended June 30, 2021 as their effect would be anti-dilutive. For the three and six months ended June 30, 2020, approximately 3.5 million and 4.8 million shares of common stock equivalents were excluded from the calculation of diluted net income per common share as their effect would be anti-dilutive.
15
2. |
Basis of Presentation and Significant Accounting Policies (continued). |
u. |
RECLASSIFICATIONS. |
v. |
RECENTLY ISSUED ACCOUNTING STANDARDS. 2019-12, Income Taxes: Simplifying the Accounting for Income Taxes step-up in the tax basis of goodwill. The standard was effective for annual periods beginning after December 15, 2020 and interim periods within, with early adoption permitted. Adoption of the standard required certain changes to be made prospectively, with some changes to be made retrospectively. The Company adopted the new standard on January 1, 2021. The adoption of this standard did not have a material impact on the Company’s consolidated financial statements. |
3. |
Investments. |
Available-for-sale
Estimated Fair Value |
Gross Unrealized Gains |
Gross Unrealized Losses |
Amortized Cost |
|||||||||||||
| At June 30, 2021: |
||||||||||||||||
| U.S. Treasuries – Cash equivalents |
$ | $ | $ | ( |
) |
$ | ||||||||||
| Short-term bond funds |
— | ( |
) | |||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total |
$ | $ | $ | ( |
) | $ | ||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| At December 31, 2020: |
||||||||||||||||
| U.S. Treasuries – Cash equivalents |
$ | $ | $ | — | $ | |||||||||||
| Short-term bond funds |
— | |||||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total |
$ | $ | $ | — | $ | |||||||||||
| |
|
|
|
|
|
|
|
|||||||||
There were no
available-for-sale
The estimated fair values of securities at June 30, 2021, by contractual maturity, are summarized as follows (in thousands):
available-for-sale
June 30, 2021 |
||||
| Due in one year or less |
$ | |||
4. |
Inventory, net. |
Inventory, net consists of the following (in thousands):
June 30, 2021 |
December 31, 2020 |
|||||||
| Raw materials |
$ | $ | — | |||||
| Work-in-process |
||||||||
| Finished goods |
||||||||
| |
|
|
|
|||||
| Total inventory, net |
|
$ |
|
$ | ||||
| |
|
|
|
|||||
16
5. |
Prepaid Expenses and |
Prepaid expenses and other current assets consist of the following (in thousands):
June 30, 2021 |
December 31, 2020 |
|||||||
| Prepaid manufacturing costs |
$ | $ | ||||||
| Prepaid tax |
||||||||
| Prepaid insurance |
||||||||
| Prepaid subscription fees |
||||||||
| Prepaid research fees |
||||||||
| Prepaid commercialization expenses |
||||||||
| Due from collaborative and licensing arrangements |
||||||||
| Other |
||||||||
| |
|
|
|
|||||
| Total prepaid expenses and other current assets |
$ | $ | ||||||
| |
|
|
|
|||||
6. |
Operating Lease. |
The Company has an operating lease agreement for its corporate office. The lease includes an option to extend the lease for up to 5 years and options to terminate the lease within 6 and 7.6 years. 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 increased from approximately 7,800 square feet of space to approximately 10,700 square feet of space. The amended lease commenced in March 2021 when construction of the asset was completed and space became available for use. Consequently, the Company recorded the effects of the amended lease during Q1 2021.
The components of lease expense were as follows (in thousands):
For the three months ended June 30, 2021 |
For the six months ended June 30, 2021 |
|||||||
| Operating lease cost |
$ | $ | ||||||
Supplemental cash flow information related to leases was as follows:
June 30, 2021 |
||||
| Cash paid for amounts included in the measurement of lease liabilities: |
||||
| Operating cash flows |
$ | |||
| Right-of-use |
||||
| Operating leases |
$ | |||
Supplemental balance sheet information related to leases was as follows:
| Operating lease right-of-use |
$ |
|||
| |
|
|||
| Other current liabilities |
$ | |||
| Operating lease liabilities, net of current portion |
||||
| |
|
|||
| Total operating lease liabilities |
$ | |||
| |
|
|||
| Remaining lease term |
|
|||
| Discount rate |
% | |||
17
6. |
Operating Lease (continued). |
Remaining payments of lease liabilities as of June 30, 2021 were as follows (in thousands):
| 2021 (remaining six months) |
$ | |||
| 2022 |
||||
| 2023 |
||||
| 2024 |
||||
| 2025 |
||||
| Thereafter |
||||
| |
|
|||
| Total lease payments |
||||
| Less imputed interest |
( |
) | ||
| |
|
|||
| Total |
$ | |||
| |
|
Rent expense was approximately $0.1 million and $0.2 million for the three and six months ended June 30, 20210.1 million and $0.2 million for the three and six months ended June 30, 2020
, respec
and $tively,
, respectively.
7. |
Property and Equipment, net. |
Property and equipment, net consists of the following (in thousands):
June 30, 2021 |
December 31, 2020 |
|||||||
| Computer equipment |
$ | $ | ||||||
| Furniture and equipment |
||||||||
| Leasehold improvements |
||||||||
| Less: Accumulated depreciation |
( |
) | ( |
) | ||||
| |
|
|
|
|||||
| Total property and equipment, net |
$ | $ | ||||||
| |
|
|
|
|||||
8. |
Accrued Expenses and Other Liabilities. |
Accrued expenses and other liabilities consist of the following (in thousands):
June 30, 2021 |
December 31, 2020 |
|||||||
| 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 |
|
$ |
|
$ | ||||
| |
|
|
|
|||||
18
9. |
Collaboration |
Endo
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 ten years following the commercial launch of the product.
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 The Company evaluated the license agreement with Endo to determine whether it is a collaborative arrangement for purposes of ASC 808. As the Company shares in the significant risks and rewards, the Company has concluded that this is a collaborative arrangement. As developing a final finished dosage form of a generic product in exchange for consideration is not an output of the Company’s ongoing activities, Endo does not represent a contract with a customer. However, Topic 808 does not provide guidance on the recognition of consideration exchanged or accounting for the obligations that may arise between the parties. The Company concluded that ASC Topic 730, , should be applied by analogy to payments between the parties during the development activities and ASC 606 for the milestone payment and sharing of defined net profits upon commercialization.
Research and Development
The collaborative agreement included a nonrefundable upfront license fee that was recognized upon receipt following execution of the collaborative arrangement for vigabatrin tablets.
The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Par. As of June 30, 2021 and 2020, no
There were no
expenses incurred, net, in connection with the collaborative agreement for the three and six months ended June 30, 2021 were approximately $45,000
$8,554 and $4,206 ,
respectively. These expenses have been included in research and development expenses in the accompanying consolidated statements of operations and comprehensive income.
KYE Pharmaceuticals
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, Catalyst granted KYE an exclusive license to commercialize and market Firdapse
®
in Canada. 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 will receive an
up-front
payment, received payment upon transfer of Marketing Authorization and delivery of commercial product, received payment for supply of Firdapse®
, 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. Although this agreement is in form identified as a collaborative agreement, the Company has concluded for accounting purposes that it represents a contract with a customer. This is because the Company grants to KYE a license and provides supply of Firdapse
®
in exchange for consideration, which are outputs of the Company’s ongoing activities. Accordingly, the Company has concluded that this collaborative arrangement will be accounted for pursuant to ASC 606. The collaborative agreement included a nonrefundable upfront license fee that was recognized upon transfer of the license based on a determination that the right is provided as the intellectual property exists at the point in time in which the license is granted.
19
9. |
Collaboration and Licensing Arrangements (continued). |
Under the arrangement, the Company will receive profit-sharing reports within nine days after quarter end from the collaborator. Revenue from sales of collaboration products by the Company’s collaborator will be recognized in the quarter in which the sales occurred.
Revenues
from the arrangement with KYE for the three or six months ended June 30, 2021 were no
Total expenses incurred, net, in connection with the agreement with KYE for the three and six months ended June 30, 2021 were approximately $DyDo Pharma, Inc.
On June 28, 2021, the Company entered into a license agreement with DyDo Pharma, Inc. (DyDo), for the development and commercialization of Firdapse
®
in Japan. Under the agreement, DyDo will have joint rights to develop Firdapse
®
, and exclusive rights to commercialize the product, in Japan. DyDo will be responsible for funding all clinical, regulatory, marketing and commercialization activities in Japan, while the Company is responsible for clinical and commercial supply based on purchase orders, as well as providing support to DyDo in its efforts to obtain regulatory approval for the product from the Japa
nese regulatory authorities. Under the terms of the agreement, the Company has earned an
up-front
payment and may earn further development and sales milestones for Firdapse®
, as well as revenue on product supplied to DyDo. The Company has concluded that this license agreement will be accounted for pursuant to ASC 606. The agreement included a nonrefundable upfront license fee that was recognized upon the effective date of the agreement as the intellectual property exists at the point in time in which the right to the license is granted. The Company determined the granting of the right to the license is distinct from the supply of Firdapse
®
and represents a separate performance obligation in the agreement. Additionally, the agreement includes sales-based milestones in which the license is deemed to be the predominant item to which these milestones relate. Revenue will be recognized when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty has been satisfied. Variable consideration related to regulatory milestones is fully constrained and only recognized when the uncertainty is subsequently resolved. For clinical and commercial supply of the product, the Company will recognize revenue when the Customer obtains control of the Company’s product, which will occur at a point in time which is generally at time of shipment.
Revenue from the arrangement with DyDo for the three and six months ended June 30, 2021 was approximately $2.7
relating to the nonrefundable upfront license fee, and
is included in license and other revenue in the accompanying consolidated statements of operations and comprehensive income. As of June 30, 2021, no milestone payments have been earned and there has been no clinical or commercial supply of Firdapse
®
. 10. |
Commitments and Contingencies. |
In May 2019, the FDA approved a New Drug Application (NDA) for Jacobus Pharmaceuticals for Ruzurgi at a lower price than the Company is offering Firdapse
®
, 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®
®
. While the NDA for Ruzurgi®
only covers pediatric patients, the Company believes that 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, and Jacobus intervened in the case. 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®
. 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 judgment and deny the Company’s motion for summary judgment. 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 judgment, and dismissed the Company’s case.
20
10. |
Commitments and |
The Company has appealed the District Court’s decision to the Eleventh Circuit Court of Appeals. There can be no assurance as to the outcome of the Company’s appeal.
On August 10, 2020, Health Canada issued a Notice of Compliance (NOC) to Medunik 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
®
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®
®
Product Monograph clearly references pivotal nonclinical carcinogenicity and reproductive toxicity data for amifampridine phosphate developed by the Company. As such, the Company believes that its data was relied upon to establish the nonclinical safety profile of Ruzurgi®
needed to meet the standards of the Canadian Food and Drugs Act. On June 3, 2021, the Company announced a positive decision in this proceeding that quashed the NOC previously issued for Ruzurgi
®
and remanded the matter to the Minister of Health to redetermine its decision to grant marketing authorization to Ruzurgi®
in spite of Firdapse®
’s data protection rights. However, on June 28, 2021, the Company announced that Health Canada had re-issued
a NOC for Ruzurgi®
, once again allowing the product to be marketed in Canada for patients with LEMS. There can be no assurance of the result of this proceeding. See Note 15 (Subsequent Events). 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.
11. |
Agreements. |
a. |
LICENSE AGREEMENT FOR FIRDAPSE ® .On ® . Under the license agreement, the Company pays: (i) royalties to the licensor for ® equal to ® equal to |
On May 29, 2019, the Company and BioMarin entered into an amendment to the Company’s 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. In January 2020, the Company was advised that BioMarin has transferred certain rights under the license agreement to SERB S.A.
b. |
AGREEMENTS FOR DRUG MANUFACTURING, DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. |
21
12. |
Income Taxes. |
The Company’s effective income tax rate was 23.0 % and 5.7 % for the 21 %
six
months ended June 30, 2021 and 2020, respectively. The difference in the effective rates between periods is driven by the release of the Company’s valuation allowance against deferred taxes in the third quarter of 2020. Differences in the effective tax and the statutory federal income tax rate of
are driven by state income taxes and anticipated annual permanent differences, and offset by the orphan drug credit claimed.
The Company had no
13. |
Stockholders’ Equity. |
Preferred Stock
The Company has 5,000,000 0.001 No
Common Stock
The Company has 200,000,000 0.001 103,162,041 103,781,641 one vote of each share of common stock held of record on all matters on which stockholders generally are entitled to vote.
Share Repurchases
In March 2021, the Company’s Board of Directors approved a share repurchase program that authorizes the repurchase of up to $40 million of the Company’s common stock, pursuant to a repurchase plan under Rule 732,951 and 800,000 shares were repurchased for an aggregate purchase price of approximately $3.7 million ($5.00 average price per share) and $4.0 million ($4.95 average price per share), respectively.
10b-18
of the Securities Act. The share repurchase program commenced on March 22, 2021 and, during the three and six months ended June 30, 2021, See Note 15 (Subsequent Events).
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. 14. |
Stock Compensation. |
For the three and six months ended June 30, 2021 and 2020, the Company recorded stock-based compensation expense as follows (in thousands):
Three months ended June 30, |
Six months ended June 30, |
|||||||||||||||
2021 |
2020 |
2021 |
2020 |
|||||||||||||
| Research and development |
$ | $ | $ | $ | ||||||||||||
| Selling, general and administrative |
||||||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total stock-based compensation |
$ | $ | $ | $ | ||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
22
14. |
Stock Compensation (continued). |
Stock Options
As of June 30, 2021, there were outstanding stock options to purchase 13,759,670 shares of common stock, of which stock options to purchase 8,765,623 shares of common stock were exercisable as of June 30, 2021.
During the three and six months ended June 30, 2021, the Company granted seven-year 70,000 and 630,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $1.4 million and $2.8 million, respectively, during the three and six months ended June 30, 2021.
During the three and six months ended June 30, 2020, the Company granted seven-year 260,000 and 995,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $1.6 million and $3.0 million, respectively, during the three and six months ended June 30, 2020.
During the three and six months ended June 30, 2021, options to purchase 83,332 shares and 173,330 shares, respectively, of the Company’s common stock were exercised, with proceeds of $270,523 and $458,818 respectively, to the Company.
During the three and six months ended June 30, 2020, options to purchase 13,333 shares and 24,999 shares, respectively, of the Company’s common stock were exercised, with proceeds of $36,201 and $62,350 respectively, to the Company.
As of June 30, 2021, there was approximately $8.3 million of unrecognized compensation expense related to 2.08 years.
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 Restricted Stock Units
There were no 30,000 0.1 million and $0.3 million, respectively. During the three and six months ended June 30, 2020, the Company recorded 0.2 million and $0.3 million, respectively.
non-cash
stock-based compensation expense related to restricted stock units totaling $non-cash
stock-based compensation expense related to restricted stock units totaling $As of June 30, 2021, there was approximately $0.8 million of unrecognized compensation expense related to 1.49 years.
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 15. |
Subsequent |
Subsequent to June 30, 2021, 199,746 shares of the Company’s common stock were repurchased under the Company’s stock repurchase plan. The shares were purchased at an average price of $5.48 per share for a total cost of $1.1 million.
During July 2021, as a result of Health Canada
re-issuing
an NOC for Ruzurgi®
allowing the product to be marketed again in Canada for patients with LEMS, the Company along with KYE, filed a suit against Health Canada to overturn the Minister’s recent decision to approve Ruzurgi®
. There can be no assurance as to the outcome of this proceeding. 23
ITEM 2. |
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
Introduction
Management’s Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to provide an understanding of our financial condition, changes in financial condition and results of operations. The discussion and analysis is organized as follows:
• |
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. |
OVERVIEW
We are a commercial-stage patient centric biopharmaceutical company focused on
in-licensing,
developing and commercializing novel high-quality medicines for patients living with rare diseases. We are currently focusing our efforts on products that treat diseases in the neuromuscular and neurological space, but recently, our Board of Directors approved an expansion in our company’s strategic focus to include potentially acquiring or in-licensing
innovative technology platforms and earlier stage programs in other rare disease therapeutic categories outside of neuromuscular diseases. To accomplish these new priorities, we are prepared to invest more heavily in research and development, including acquiring earlier stage opportunities and innovative technology. We believe that this strategic expansion will better position our company to build out a broader more diversified portfolio of drug candidates that we believe will add greater value to our company over the near and long-term. However, there can be no assurance that whatever product candidates or technology platforms we acquire, if any, will be successfully developed. To spearhead this investment, we recently hired Dr. Preethi Sundaram as our Chief Product Development Officer. In that position, Dr. Sundaram will lead the strategy and direct the development of programs from early-stage R&D assets through late-stage clinical programs that will be focusing on developing therapies to treat rare diseases. We are also currently exploring several potential opportunities to acquire companies with drug products in development or to
in-license
drug products in development. However, no definitive agreements have been entered into to date. We are dedicated to making a meaningful impact on the lives of those suffering from rare diseases, and we believe in putting patients first in everything we do.
Impact of the
COVID-19
pandemic on our business The
COVID-19
pandemic has resulted, and is expected to continue to result, in significant economic disruption, and has adversely affected and will likely continue to adversely affect our business. We are actively monitoring the situation and are taking those actions that may be required by federal, state or local authorities or that we determine are in the best interests of our patients, investigators, employees and stockholders. In March 2020, in light of worsening conditions as a result of the pandemic, we implemented a number of safety related initiatives among our employees, including a travel ban and a work from home policy for all employees. This included our customer-facing employees, who began working remotely and utilizing telephone and
web-based
technologies to provide support to patients and their healthcare providers. Recently, in light of decreasing COVID-19
case numbers and increasing vaccination, we have begun to relax these restrictions, including a return to in-person
work. However, the continually changing nature of the pandemic means that these restrictions may return. 24
We believe that because many healthcare providers have delayed seeing new patients because of the pandemic, there has been a delay in the diagnosis of new LEMS patients and their initiating therapy, which has slowed our efforts to locate new patients who could benefit from our therapy. However, we believe that as more healthcare providers resume seeing new patients on a regular basis, the impact of this aspect of the
COVID-19
pandemic on our business will lessen. Firdapse
®
On November 28, 2018, we received approval from the FDA for Firdapse
®
Tablets, 10 mg for the treatment of adult LEMS patients (ages 17 and above). In January 2019, we launched Firdapse®
in the United States, selling through a field force experienced in neurologic, central nervous system or rare disease products consisting at the time of approximately 20 field personnel, including sales (Regional Account Managers), patient assistance and insurance navigation support (Patient Access Liaisons), and payor reimbursement (National Account Managers). We also have a field-based force of six medical science liaisons who are helping educate the medical communities and patients about LEMS and about our ongoing clinical trial activities evaluating Firdapse®
for other ultra-orphan, neuromuscular diseases. Finally, we are working with several rare disease advocacy organizations (including Global Genes, the National Organization for Rare Disorders (NORD), and the Myasthenia Gravis Foundation of America) to help increase awareness and level of support for patients living with LEMS, Anti-MuSK antibody positive myasthenia gravis, or MuSK-MG,
and other neurological diseases, and to provide education for the physicians who treat these rare diseases and the patients they treat. In early 2020, we expanded our field sales group by almost one hundred percent and contracted with an experienced inside sales agency generating leads through telemarketing to targeted physicians. We made these changes to expand our sales efforts beyond the neuromuscular specialists who regularly treat LEMS patients to reach the roughly 9,000 neurology and neuromuscular healthcare providers that may be treating an adult LEMS patient who can benefit from Firdapse
®
. We also continue to make available at no-cost
a LEMS voltage gated calcium channel (VGCC) antibody testing program for use by physicians who suspect that one of their patients may have LEMS and wish to reach a definitive diagnosis. Further, we are continuing to expand our digital and social media activities in order to introduce our product and services to potential patients and their healthcare providers. We are supporting the distribution of Firdapse
®
through Catalyst Pathways®
, our personalized treatment support program. Catalyst Pathways®
is a single source for personalized treatment support, education and guidance through the challenging dosing and titration regimen to an effective therapeutic dose. It also includes distributing the drug through a very small group of exclusive specialty pharmacies (primarily AnovoRx), which is consistent with the way that most pharmaceutical products for ultra-orphan diseases are distributed and dispensed to patients. We believe that by using specialty pharmacies in this way, the difficult task of navigating the health care system is far better for the patient needing treatment for their rare disease and the health care community in general. In order to help adult LEMS patients afford their medication, we, like other pharmaceutical companies which are marketing drugs for ultra-orphan conditions, have developed an array of financial assistance programs that are available to reduce patient costs to not more than $10.00 per month (currently $0.00 per month) available for all LEMS patients prescribed Firdapse
co-pays
and deductibles to a nominal affordable amount. For eligible patients with commercial coverage, a co-pay
assistance program designed to keep out-of-pocket
®
. We are also donating, and committing to continue to donate, money to qualified, independent charitable foundations dedicated to providing assistance to any U.S. LEMS patients in financial need. Subject to compliance with regulatory requirements, our goal is that no LEMS patient is ever denied access to their medication for financial reasons. In May 2019, the FDA approved a New Drug Application (NDA) for Ruzurgi
®
, another version of amifampridine (3,4-DAP),
for the treatment of pediatric LEMS patients (ages 6 to under 17). While the NDA for Ruzurgi®
only covers pediatric patients, we believe that Ruzurgi®
is regularly being prescribed off-label
to adult LEMS patients. We believe that under applicable law, Jacobus Pharmaceuticals (“Jacobus”) is not permitted to market its amifampridine product, Ruzurgi®
, to adult LEMS patients in the United States, and we are continuing to aggressively take all steps available to us to protect Firdapse’s®
exclusivity under the Orphan Drug Act. There can be no assurance, however, that we will be able to stop the off-label
prescribing of Ruzurgi®
to adult LEMS patients. If Jacobus is able to successfully continue to sell Ruzurgi®
off-label
to additional adult LEMS patients, it could have a material adverse effect on our business, financial condition and results of operations. We also believe that the FDA’s approval of Ruzurgi
®
violated our statutory rights and was in multiple other respects arbitrary, capricious and contrary to law. As a result, in June 2019 we filed suit against the FDA and several related parties challenging this approval and related drug labeling, and Jacobus intervened in our case. Our 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 our 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 setting aside the FDA’s approval of Ruzurgi®
. 25
On July 30, 2020, the Magistrate Judge considering our 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 judgment and deny our motion for summary judgment. On September 29, 2020, the District Judge adopted the Report and Recommendation of the Magistrate Judge, granted the FDA’s and Jacobus’ motions for summary judgment, and dismissed our case.
We believe that the District Judge’s decision was incorrect as a matter of law and contrary to the plain language of the Orphan Drug Act. We believe that if the District Judge’s decision to grant summary judgment is correct on the law, it means that the FDA has the authority to effectively eliminate the benefits of exclusivity under the Orphan Drug Act, which we believe will chill the incentive for drug companies to spend the millions of dollars necessary to develop an orphan drug. As a result, we have appealed the District Court’s decision to the Eleventh Circuit Court of Appeals. The case has been fully briefed and oral argument was held in March 2021. However, no decision has been received to date, and there can be no assurance as to the result of such proceeding.
We are currently developing a long-acting formulation of amifampridine phosphate. A number of candidate formulations have been prepared, and three of the most promising formulations were evaluated in a pharmacokinetic (PK) study completed during the fourth quarter of 2020. The results of this first PK study will be used to inform the design and refinement of future product formulations and additional PK work to be conducted. We have also completed a number of advisory board meetings with both patients and doctors in order to establish the optimum target characteristics of the long-acting formulation of amifampridine phosphate that are desired by the LEMS patient community and treating physicians. There can be no assurance that we will be able to successfully develop a long-acting formulation of amifampridine phosphate, that any such formulation will be approved by the FDA for marketing, or that any such formulation will be commercially viable.
On August 10, 2020, we announced the trial. We found in our analysis that there was a large degree of symptom variability during the double-blind withdrawal period. We believe that sources of such variability can be addressed in a redesigned study that may better demonstrate the efficacy of our drug for the treatment of
top-line
results from our Phase 3 clinical trial (MSK-002)
evaluating Firdapse®
for the treatment of adults with MuSK-MG.
Unfortunately, the MSK-002
trial did not achieve statistical significance on its primary endpoint or its secondary endpoint. However, we have recently concluded a detailed analysis of the data from this trial in an effort to understand why the MuSK-MG
Phase 3 trial did not meet statistical significance on its endpoints, even though clinical improvement was observed by patients and investigators during the initial dose-titration period of the trial and in our previous proof-of-concept
MuSK-MG.
We recently presented our hypotheses and a revised protocol to the FDA with respect to the
MuSK-MG
indication. The FDA provided written comments that were unfavorable towards a revised study design and further questioned the ability of the initial MuSK-MG
pilot study to be supportive. These remarks make it unlikely that a single study similar design to MSK-002
would be sufficient for potential approval of the MuSK-MG
indication. We are preparing an appropriate expert panel to discuss options and review the likelihood of success for a MuSK-MG
indication for Firdapse®
. We intend to conduct a study evaluating Firdapse
proof-of-concept
®
as a treatment for Hereditary Neuropathy with Liability to Pressure Palsies (HNPP). The scientific basis for considering this indication is that leakage of neuron potassium channels is observed in HNPP. Since Firdapse®
is a potassium channel blocker, it may mitigate the pathological effects of the potassium channel leakage in HNPP patients. The FDA requested that a new, patient centric endpoint be researched and used for our proposed study, without assurance that it would be acceptable for approval. Based upon the uncertainty of such an endpoint, we have decided not to conduct this as a company sponsored study. The proposed investigator is considering whether to seek Company support to conduct the study as an investigator-initiated study. There can be no assurance that clinical trials of Firdapse
®
that we undertake in the future will be successful. Further, there can be no assurance that we will ever be granted the right to commercialize Firdapse®
for any additional indications. Our NDS filing for Firdapse
®
for the symptomatic treatment of LEMS was approved by Health Canada on July 31, 2020. In August 2020, we entered into a license agreement with KYE Pharmaceuticals (KYE), pursuant to which we licensed the Canadian rights for Firdapse®
for the treatment of LEMS to KYE. Pursuant to the license agreement, KYE is obligated to pay us an up-front
payment based on approval and product supply, data protection milestones based on achievements of sales and regulatory milestones, and a sharing of defined net sales upon commercialization. On August 10, 2020, Health Canada issued a Notice of Compliance (NOC) to Medunik for Ruzurgi
®
for the treatment of LEMS. We 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 us. As such, we believe 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. 26
On June 3, 2021, we announced a positive decision in this proceeding that quashed the NOC previously issued for Ruzurgi
®
and remanded the matter to the Minister of Health to redetermine its decision to grant marketing authorization to Ruzurgi®
in spite of Firdapse®
’s data protection rights. However, on June 28, 2021, we announced that Health Canada had re-issued
an NOC for Ruzurgi®
, once again allowing the product to be marketed in Canada for patients with LEMS. As a result, in early July 2021, we, along with our partner KYE, filed a second suit against Health Canada to overturn their most recent decision. There can be no assurance as to the outcome of this proceeding. In May 2019, we entered into an amendment to our license agreement for Firdapse
®
. Under the amendment, we have expanded our commercial territory for Firdapse®
, which originally was comprised of North America, to include Japan. Additionally, we have an option to further expand our 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, we will pay royalties on net sales in Japan of a similar percentage to the royalties that we are currently paying under our original license agreement for North America. We have reached an agreement with Japanese regulatory authorities as to the scope of the clinical trial that we will be required to undertake in Japan before we will be permitted to submit an application to the Japanese regulatory authorities to seek to commercialize Firdapse
®
for the treatment of LEMS in Japan. We also recently been granted orphan drug designation in Japan for the symptomatic treatment of LEMS. There can be no assurance that we will successfully obtain the right to commercialize Firdapse®
in Japan. On June 28, 2021, we entered into a license agreement with DyDo Pharma, Inc. (DyDo), pursuant to which we
sub-licensed
to DyDo the Japanese rights for Firdapse®
for the treatment of LEMS. Under the terms of the Agreement, DyDo will have joint rights to develop Firdapse®
, and exclusive rights to commercialize the product, in Japan. DyDo will be responsible for funding all clinical, regulatory, marketing and commercialization activities in Japan. We will be responsible for clinical and commercial supply, as well as providing support to DyDo in its efforts to obtain regulatory approval for the product from the Japanese regulatory authorities. Subject to the satisfaction of terms and conditions as set forth in the Agreement, we have earned an upfront payment and are eligible to receive further development and sales milestones for Firdapse®
, as well as revenue on product supplied to DyDo. All of our patent rights for Firdapse
®
are derived from our license agreement. In August 2020, the United States Patent and Trademark Office (USPTO) allowed Patent No. 10,798,893 (the ’893 patent) to our licensor and thereby to us, and the patent issued on October 6, 2020. The patent is directed to the use of suitable doses of amifampridine to treat patients, regardless of the therapeutic indication, that are slow metabolizers of amifampridine. Any drug product containing amifampridine with a label that states the patented dosing regimens and doses in the Dosing and Administration section prior to April 7, 2034, the expiration date of the patent, could possibly infringe this patent. Generic drug product labels would necessarily have to do this, and we intend to take all appropriate actions to protect our intellectual property. In that regard, in October 2020, we 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 the specialty pharmacy marketing Ruzurgi
®
, PantherRx Rare LLC (PantherRx), for infringement of the ‘893 patent. The suits have since been combined in the U.S. District Court for New Jersey. The lawsuit arises from Jacobus’ and PantherRx’s sales and marketing of Ruzurgi®
(amifampridine) Tablets, 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 our patent rights. The lawsuit is in the discovery stage and there can be no assurance as to the results of these proceedings. In April 2021, t
he USPTO allowed Patent No. 11,060,128 (the ’128 patent) to our licensor and thereby to us, and the patent issued on July 13, 2021. The patent is directed to the use of suitable doses of amifampridine to treat patients suffering with LEMS, that are slow metabolizers of amifampridine. Any drug product containing amifampridine with a label for the treatment of LEMS, that states the patented dosing regimens and doses in the Dosing and Administration section of a product label could possibly infringe this patent.
We are also pursuing additional patent applications for Firdapse
®
in an effort to further protect our drug product. There can be no assurance that any additional patents will be issued which provide additional intellectual property protection for our drug product. There can be no assurance that we do not or will not infringe on patents held by third parties or that third parties in the future will not claim that we have infringed on their patents. In the event that our products or technologies infringe or violate the patent or other proprietary rights of third parties, there is a possibility we may be prevented from pursuing product development, manufacturing or commercialization of our products that utilize such technologies until the underlying patent dispute is resolved. For example, there may be patents or patent applications held by others that contain claims that our products or operations might be determined to infringe or that may be broader than we believe them to be. Given the complexities and uncertainties of patent laws, there can be no assurance as to the impact that future patent claims against us may have on our business, financial condition, results of operations, or prospects.
27
Generic Sabril
®
In December 2018, we entered into a definitive agreement with Endo International plc’s subsidiary, Endo Ventures Limited (“Endo”), for the further development and commercialization of generic Sabril
®
tablets through Endo’s United States Generic Pharmaceuticals segment, Par Pharmaceutical. Pursuant to the agreement, in December 2018, we received an up-front
payment of $500,000. We will be entitled to receive a milestone payment of $2.0 million on the commercial launch of the product. Further, we will receive a sharing of defined net profits upon commercialization and we are obligated to share the costs of certain development expenses. There can be no assurance that our collaboration with Endo for the development of generic Sabril
®
(vigabatrin) tablets will be successful and that if an abbreviated new drug application (ANDA) is approved for vigabatrin tablets in the future, that it will be profitable to us. Capital Resources
At June 30, 2021, we had cash and investments of approximately $155.3 million and no funded debt. Based on our current financial condition and forecasts of available cash, we believe that we have sufficient funds to support our operations for at least the next 12 months from the date of this Form
10-Q.
There can be no assurance that we will continue to be successful in commercializing Firdapse®
or will continue to be profitable and cash flow positive. Further, there can be no assurance that if we need additional funding in the future, whether such funding will be available to us. See “Liquidity and Capital Resources” below for further information on our liquidity and cash flow. Basis of Presentation
Revenues.
During the fiscal quarter ended June 30, 2021, we continued to generate revenues from product sales of Firdapse
®
in the U.S. We expect these revenues to fluctuate in future periods based on our sales of Firdapse®
. We received approval from Health Canada on July 31, 2020, for Firdapse®
for the symptomatic treatment of LEMS in adults and as of December 31, 2020, we had launched Firdapse®
in Canada. For the three and six months ended June 30, 2021 and 2020, revenues generated under our collaborative agreement with KYE Pharmaceuticals were immaterial. We expect our revenues from the KYE collaborative agreement to fluctuate in future periods based on our collaborator’s ability to sell Firdapse®
in Canada. For the three and six months ended June 30, 2021, we did not generate revenues under our collaborative agreement with Endo. We expect our revenues from the Endo collaborative agreement to fluctuate in future periods based on our collaborator’s ability to meet various regulatory milestones set forth in such agreement.
For the three and six months ended June 30, 2021, we earned approximately $2.7 million in revenue under our licensing agreement with DyDo. We expect our revenues from the DyDo license agreement to fluctuate in future periods based on DyDo’s ability to meet various regulatory milestones set forth in such agreement.
Cost of Sales.
Cost of sales consists of third-party manufacturing costs, freight, royalties, and indirect overhead costs associated with sales of Firdapse
®
. Cost of sales may also include period costs related to certain inventory manufacturing services, inventory adjustments charges, unabsorbed manufacturing and overhead costs, and manufacturing variances. 28
Research and Development Expenses.
Our research and development expenses consist of costs incurred for company-sponsored research and development activities, as well as support for selected investigator-sponsored research. The major components of research and development costs include preclinical study costs, clinical manufacturing costs, clinical study and trial expenses, insurance coverage for clinical trials, consulting, and other third-party costs, salaries and employee benefits, stock-based compensation expense, supplies and materials, and allocations of various overhead costs related to our product development efforts. To date, all of our research and development resources have been devoted to the development of Firdapse
®
, CPP-109
(our version of vigabatrin), and formerly CPP-115,
and we currently expect that our future development costs will be attributable principally to the continued development of Firdapse®
. However, as noted in the Overview above, we are prepared to invest in future research and development, including earlier stage opportunities and innovative technology. Our cost accruals for clinical studies and trials are based on estimates of the services received and efforts expended pursuant to contracts with numerous clinical study and trial sites and clinical research organizations (CROs). In the normal course of our business we contract with third parties to perform various clinical study and trial activities in the
on-going
development of potential products. The financial terms of these agreements are subject to negotiation and vary from contract to contract and may result in uneven payment flows. Payments under the contracts depend on factors such as the achievement of certain events or milestones, the successful enrollment of patients, the allocation of responsibilities among the parties to the agreement, and the completion of portions of the clinical study or trial or similar conditions. The objective of our accrual policy is to match the recording of expenses in our consolidated financial statements to the actual services received and efforts expended. As such, expense accruals related to preclinical and clinical studies or trials are recognized based on our estimate of the degree of completion of the event or events specified in the specific study or trial contract. We monitor service provider activities to the extent possible; however, if we underestimate activity levels associated with various studies or trials at a given point in time, we could be required to record significant additional research and development expenses in future periods. Preclinical and clinical study and trial activities require significant up-front
expenditures. We anticipate paying significant portions of a study or trial’s cost before they begin and incurring additional expenditures as the study or trial progresses and reaches certain milestones. Selling, General and Administrative Expenses.
During 2019, we actively committed funds to developing our commercialization program for Firdapse
®
and we have continued to incur substantial commercialization expenses, including sales, marketing, patient services, patient advocacy and other commercialization related expenses as we have continued our sales program for Firdapse®
. Our general and administrative expenses consist primarily of salaries and personnel expenses for accounting, corporate, compliance, and administrative functions. Other costs include administrative facility costs, regulatory fees, insurance, and professional fees for legal including litigation cost, information technology, accounting, and consulting services.
Stock-Based Compensation.
We recognize expense for the fair value of all stock-based awards to employees, directors, and consultants in accordance with accounting principles generally accepted in the U.S. (U.S. GAAP). For stock options, we use the Black-Scholes option valuation model in calculating the fair value of the awards.
Income Taxes.
Our effective income tax rate is the ratio of income tax expense (benefit) over our income before income taxes.
Recently Issued Accounting Standards.
For discussion of recently issued accounting standards, please see Note 2, “Basis of Presentation and Significant Accounting Policies,” in the interim consolidated financial statements included in this report.
29
Critical Accounting Policies and Estimates
Our discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which have been prepared in accordance with U.S. GAAP. The preparation of these consolidated financial statements requires us to make judgments, estimates, and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the consolidated financial statements, as well as the reported revenues and expenses during the reporting periods. For a full discussion of our accounting policies, please refer to Note 2 on the Financial Statements included in our 2020 Annual Report on Form included in our 2020 Annual Report on
10-K
that we filed with the SEC on March 15, 2021. Our most critical accounting policies and estimates include: accounting for revenue recognition, leases, preclinical study and clinical trial expenses, stock-based compensation and valuation allowance for deferred tax assets. We continually evaluate our judgments, estimates and assumptions. We base our estimates on the terms of underlying agreements, our expected course of development, historical experience and other factors that we believe are reasonable based on the circumstances, the results of which form our management’s basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates. There have been no material changes to our critical accounting policies and estimates from the information provided in Part II, Item 7, Management’s Discussion and Analysis of Financial Condition and Results of Operations
Form 10-K.
Results of Operations
Revenues.
For the three and six months ended June 30, 2021, we recognized approximately $33.6 million and $63.8 million, respectively, in net revenue from product sales from Firdapse
®
compared to approximately $29.6 million and $58.7 million for the three and six months ended June 30, 2020. We recognized approximately $2.7 million for the three and six months ended June 30, 2021 in license and other revenue. We had no revenues from our collaborative or license arrangements for the three and six months ended June 30, 2020. Cost of Sales.
Cost of sales was approximately $4.5 million and $9.2 million for the three and six months ended June 30, 2021 compared to approximately $4.1 million and $8.3 million for the three and six months ended June 30, 2020. Cost of sales consists principally of royalty payments which are based on net revenue as defined in the applicable license agreement.
Research and Development Expenses.
Research and development expenses for the three months ended June 30, 2021 and 2020 were approximately $4.5 million and $4.4 million, respectively, and represented approximately 21.7% and 22.5% of total operating costs and expenses for the three months ended June 30, 2021 and 2020, respectively. Research and development expenses for the three months ended June 30, 2021 and 2020 were as follows (in thousands):
Three months ended June 30, |
Change |
|||||||||||||||
2021 |
2020 |
$ |
% |
|||||||||||||
| Research and development expenses |
$ | 4,074 | $ | 3,929 | 145 | 3.7 | % | |||||||||
| Employee stock-based compensation |
376 | 421 | (45 | ) | (10.7 | )% | ||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total research and development expenses |
$ | 4,450 | $ | 4,350 | 100 | 2.3 | % | |||||||||
| |
|
|
|
|
|
|
|
|||||||||
Research and development expenses for the six months ended June 30, 2021 and 2020 were approximately $7.5 million and $8.6 million, respectively, and represented approximately 18.2% and 22.7% of total operating costs and expenses for the six months ended June 30, 2021 and 2020, respectively. Research and development expenses for the six months ended June 30, 2021 and 2020 were as follows (in thousands):
Six months ended June 30, |
Change |
|||||||||||||||
2021 |
2020 |
$ |
% |
|||||||||||||
| Research and development expenses |
$ | 6,692 | $ | 7,734 | (1,042 | ) | (13.5 | )% | ||||||||
| Employee stock-based compensation |
765 | 839 | (74 | ) | (8.8 | )% | ||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total research and development expenses |
$ | 7,457 | $ | 8,573 | (1,116 | ) | (13.0 | )% | ||||||||
| |
|
|
|
|
|
|
|
|||||||||
Research and development expenses stayed relatively consistent for the three and six month periods ended June 30, 2021 when compared to the same periods in 2020. For the three and six months ended June 30, 2020, research and development expenses included costs relating to our trial, both of which were completed in the second half of 2020. For the three and six months ended June 30, 2021, research and development expenses included costs relating to winding down of the sites for both the MuSK-MG clinical trial and SMA type 3 proof-of-concept trial. Research and development costs in both 2020 and 2021 periods included expenses relating to medical and regulatory affairs, our expanded access programs, and our efforts to develop a long-acting formulation of amifampridine phosphate.
MuSK-MG
clinical trial and our SMA type 3 proof-of-concept
30
We expect that research and development expenses will continue to be substantial in 2021 and beyond as we continue our Expanded Access Program, take steps to continue the development of a long-acting formulation of amifampridine phosphate and evaluate Firdapse as a treatment for other neuromuscular diseases. Research and development expenses will also increase if we elect to further the development of Firdapse for
®
®
MuSK-MG
or if we successfully execute on our strategic initiative to acquire or in-license
innovative technology platforms and/or earlier stage programs in other therapeutic categories outside of neuromuscular diseases. Selling, General and Administrative Expenses.
Selling, general and administrative expenses for the three months ended June 30, 2021 and 2020 were approximately $11.5 million and $10.8 million, respectively, and represented 56.2% and 56.1% of total operating costs and expenses for the three months ended June 30, 2021 and 2020, respectively. Selling, general and administrative expenses for the three months ended June 30, 2021 and 2020 were as follows (in thousands):
Three months ended June 30, |
Change |
|||||||||||||||
2021 |
2020 |
$ |
% |
|||||||||||||
| Selling |
$ | 5,981 | $ | 5,622 | 359 | 6.4 | % | |||||||||
| General and administrative |
4,409 | 3,838 | 571 | 14.9 | % | |||||||||||
| Employee stock-based compensation |
1,142 | 1,373 | (231 | ) | (16.8 | )% | ||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total selling, general and administrative expenses |
$ | 11,532 | $ | 10,833 | 699 | 6.5 | % | |||||||||
| |
|
|
|
|
|
|
|
|||||||||
Selling, general and administrative expenses for the six months ended June 30, 2021 and 2020 were approximately $24.2 million and $20.9 million, respectively, and represented 59.2% and 55.3% of total operating costs and expenses for the six months ended June 30, 2021 and 2020, respectively. Selling, general and administrative expenses for the six months ended June 30, 2021 and 2020 were as follows (in thousands):
Six months ended June 30, |
Change |
|||||||||||||||
2021 |
2020 |
$ |
% |
|||||||||||||
| Selling |
$ | 11,657 | $ | 11,425 | 232 | 2.0 | % | |||||||||
| General and administrative |
10,267 | 6,996 | 3,271 | 46.8 | % | |||||||||||
| Employee stock-based compensation |
2,324 | 2,475 | (151 | ) | (6.1 | )% | ||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total selling, general and administrative expenses |
$ | 24,248 | $ | 20,896 | 3,352 | 16.0 | % | |||||||||
| |
|
|
|
|
|
|
|
|||||||||
For the three and six months ended June 30, 2021, selling, general and administrative expenses increased approximately $0.7 million and $3.4 million, respectively, when compared to the same periods in 2020. The increase for the six months ended June 30, 2021 was primarily attributable to the timing of commitments to make contributions to 501(c)(3) organizations supporting LEMS patients of approximately $2.0 million, increases in litigation fees of approximately $1.1 million and increased costs due to the expansion of our operations and headcount required to support our ongoing efforts to commercialize Firdapse
®
. We expect that selling, general and administrative expenses will continue to be substantial in future periods as we continue our efforts to increase our revenues from Firdapse
®
and take steps to expand our business. Stock-Based Compensation.
Total stock-based compensation for the three and six months ended June 30, 2021 were $1.5 million and $3.1 million, respectively, and for the three and six months ended June 30, 2020 were $1.8 million and $3.3 million, respectively. In the first half of 2021, grants were principally for stock options relating to 2020
year-end
bonus awards. In the first half of 2020, grants were principally for stock options relating to 2019 year-end
bonus awards. Other Income, Net.
We reported other income, net in all periods relating to our investment of our cash and cash equivalents and investments. The decrease in other income, net for the three and six months ended June 30, 2021 when compared to the same periods in 2020 is primarily due to lower yields on investments, despite higher invested balances. Other income, net, consists primarily of interest and dividend income.
31
Income Taxes.
Our effective income tax rate was 23.0% and 5.7% for the six months ended June 30, 2021 and 2020, respectively. The difference in the effective rates between periods is driven by the release of the valuation allowance against deferred taxes in the third quarter of 2020. Differences in the effective tax and the statutory federal income tax rate of 21% are driven by state income taxes and anticipated annual permanent differences, and offset by the orphan drug credit claimed.
We had no uncertain tax positions as of June 30, 2021 and December 31, 2020.
Net Income.
Our net income was approximately $12.2 million and $19.8 million, respectively, for the three and six months ended June 30, 2021 ($0.12 and $0.19, respectively, per basic and $0.11 and $0.18, respectively, per diluted share) as compared to net income of approximately $9.8 million and $20.2 million, respectively, for the three and six months ended June 30, 2020 ($0.09 and $0.20, respectively, per basic and $0.09 and $0.19, respectively, per diluted share).
Liquidity and Capital Resources
Since our inception, we have financed our operations primarily through multiple public and private offering of our securities and, since January 2019, from revenues from product sales. At June 30, 2021, we had cash and cash equivalents and investments aggregating approximately $155.3 million and working capital of approximately $160.2 million. At December 31, 2020, we had cash and cash equivalents and investments aggregating $140.3 million and working capital of $136.5 million. At June 30, 2021, substantially all of our cash and cash equivalents was deposited with one financial institution, and such balances were in excess of federally insured limits. Further, as of such date, substantially all such funds were invested in money market accounts, short-term interest-bearing obligations and U.S. Treasuries.
We incurred operating losses through the quarter ended March 31, 2019 and reported net income for the first time during the three and six month periods ended June 30, 2019. We expect to continue to spend substantial dollars on our current and future drug development programs.
Based on forecasts of available cash, we believe that we have sufficient resources to support our currently anticipated operations for at least the next 12 months from the date of this report. There can be no assurance that we will remain profitable or that we will be able to obtain any additional funding that we may require in the future.
In the future, we may require additional working capital to support our operations depending on our future success with Firdapse
®
sales and whether our results continue to be profitable and cash flow positive. There can be no assurance as to the amount of any such funding that will be required for these purposes or whether any such funding will be available to us when it is required. In that regard, our future funding requirements will depend on many factors, including:
| • | the extent to which we seek to acquire or in-license additional drug development candidates; |
| • | 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; and |
| • | the cost of filing and potentially prosecuting, defending and enforcing any patent claims and other intellectual property rights. |
We may raise additional funds if required in the future through public or private equity offerings, debt financings, corporate collaborations or other means. We also may seek governmental grants for a portion of the required funding for our clinical trials and preclinical trials. We may further seek to raise capital to fund additional product development efforts or product acquisitions, even if we have sufficient funds for our planned operations. Any sale by us of additional equity or convertible debt securities could result in dilution to our stockholders. There can be no assurance that any such required additional funding will be available to us at all or available on terms acceptable to us. Further, to the extent that we raise additional funds through collaborative arrangements, it may be necessary to relinquish some rights to our technologies or grant sublicenses on terms that are not favorable to us. If we are not able to secure additional funding when needed, we may have to delay, reduce the scope of or eliminate one or more research and development programs, which could have an adverse effect on our business.
32
On July 23, 2020, we 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 2020 Shelf Registration Statement. Cash Flows.
Net cash provided by operating activities was $19.5 million and $20.5 million, respectively, for the
six-month
periods ended June 30, 2021 and 2020. During the six months ended June 30, 2021 net cash provided by operating activities was primarily attributable to our net income of $19.8 million, increases of $0.7 million in prepaid expenses and other current and non-current
assets, $0.9 million in lease liability and of $7.6 million of non-cash
expenses. This was partially offset by increases of $0.3 million in accounts receivable, $2.4 million in inventory and decreases of $1.3 million in accounts payable and $5.5 million in accrued expenses and other liabilities. During the six months ended June 30, 2020 net cash provided by operating activities was primarily attributable to our net income of $20.2 million, increases of $3.8 million in accounts receivable, net and $1.7 million in accounts payable and decreases of $0.1 million in inventory and $4.1 million of non-cash
expenses. This was partially offset by an increase of $3.2 million in prepaid expenses and other current and non-current
assets and decreases of $5.5 million in accrued expenses and other liabilities and $0.8 million in operating lease liability. Net cash used in investing activities was $10.9 million, for the
six-month
period ended June 30, 2021, consisting primarily of purchases of investments. Net cash provided by investing activities was $5.0 million for the six-month
period ended June 30, 2020, consisting of proceeds from maturities and sales of investments. Net cash used in financing activities during the
six-month
period ended June 30, 2021 was $3.5 million and consisted primarily of the repurchase of common stock. Net cash provided by financing activities during the six-month
period ended June 30, 2020 was $62 thousand, consisting of proceeds from the exercise of options to purchase common stock. Contractual Obligations and Arrangements.
We have entered into the following contractual arrangements:
| • | Payments under our license agreement |
| • | Royalties to our licensor for seven years from the first commercial sale of Firdapse ® 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 |
| • | Royalties to the third-party licensor of the rights sublicensed to us 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 duration of regulatory exclusivity within a territory and 3.5% for territories in any calendar year in territories without regulatory exclusivity. |
For the three and six months ended June 30, 2021, we recognized an aggregate of approximately $4.3 million and $8.4 million, respectively, of royalties, which is included in cost of sales in the accompanying consolidated statement of operations and comprehensive income.
| • | Employment agreements |
| • | Purchase commitment. |
| • | Lease for office space |
33
Off-Balance
Sheet Arrangements. We currently have no debt or finance leases. We have an operating lease for our office facilities. We do not have any
off-balance
sheet arrangements as such term is defined in rules promulgated by the SEC. Caution Concerning Forward-Looking Statements
This Current Report on Form
10-Q
contains “forward-looking statements”, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding our expectations, beliefs, plans or objectives for future operations and anticipated results of operations. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, “believes”, “anticipates”, “proposes”, “plans”, “expects”, “intends”, “may”, and other similar expressions are intended to identify forward-looking statements. Such statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other achievements to be materially different from any future results, performances or achievements expressed or implied by such forward-looking statements. Factors that might cause such differences include, but are not limited to, those discussed in the section entitled “Item 1A – Risk Factors” in our 2020 Annual Report on Form 10-K.
The continued successful commercialization of Firdapse
®
and the development of additional indications for Firdapse®
is highly uncertain. Factors that will affect our success include the uncertainty of: | • | The impact of the COVID-19 pandemic 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; |
| • | Whether the daily dose taken by patients changes over time and affects 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 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 appeal of the District Court’s decision in our suit against the United States FDA seeking to vacate the FDA’s approval of Ruzurgi ® will 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; |
34
| • | 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 Biden, 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 changes 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 any clinical trials and studies that we may undertake on a timely basis and within the budgets we establish for such trials and studies; |
| • | Whether COVID-19 will further affect the timing and costs of our currently ongoing and contemplated clinical trials; |
| • | Whether Firdapse ® will ever be approved for the treatment of any neuromuscular disease other than LEMS; |
| • | Whether Firdapse ® can be successfully commercialized 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 amifampridine phosphate; |
| • | Whether our efforts to grow our business beyond Firdapse ® through acquisitions of companies or in-licensing of product opportunities 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. |
Our current plans and objectives are based on assumptions relating to the continued commercialization of Firdapse
®
and the development of additional indications for Firdapse®
. Although we believe that our assumptions are reasonable, any of our assumptions could prove inaccurate. In light of the significant uncertainties inherent in the forward-looking statements we have made herein, which reflect our views only as of the date of this report, you should not place undue reliance upon such statements. We undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. ITEM 3. |
QUANTITATIVE AND QUALITATIVE DISCLOSURE ABOUT MARKET RISK |
Market risk represents the risk of changes in the value of market risk-sensitive instruments caused by fluctuations in interest rates, foreign exchange rates and commodity prices. Changes in these factors could cause fluctuations in our results of operations and cash flows.
Our exposure to interest rate risk is currently confined to our cash and short-term investments that are from time to time invested in highly liquid money market funds, U.S. Treasuries and short-term bond funds. The primary objective of our investment activities is to preserve our capital to fund operations. We also seek to maximize income from our investments without assuming significant risk. We do not use derivative financial instruments in our investment portfolio. Our cash and investments policy emphasizes liquidity and preservation of principal over other portfolio considerations.
35
ITEM 4. |
CONTROLS AND PROCEDURES |
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 June 30, 2021, 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 June 30, 2021, 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. |
PART II. OTHER INFORMATION
ITEM 1. |
LEGAL PROCEEDINGS |
Ruzurgi
®
We believe that the FDA’s approval of Ruzurgi
®
violated our statutory rights and was in multiple other respects arbitrary, capricious and contrary to law. As a result, in June 2019 we filed suit against the FDA and several related parties challenging this approval and related drug labeling, and Jacobus intervened in the litigation. Our 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 our 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 setting aside the FDA’s approval of Ruzurgi®
. On July 30, 2020, the Magistrate Judge considering our 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 judgment and deny our motion for summary judgment. On September 29, 2020, the District Judge adopted the Report and Recommendation of the Magistrate Judge, granted the FDA’s and Jacobus’ motions for summary judgment, and dismissed our case.
We believe that the District Judge’s decision is incorrect as a matter of law and contrary to the plain language of the Orphan Drug Act. We believe that if the District Judge’s decision to grant summary judgment is correct on the law, it means that the FDA has the authority to effectively eliminate the benefits of exclusivity under the Orphan Drug Act, which we believe will chill the incentive for drug companies to spend the millions of dollars necessary to develop an orphan drug. As a result, we have appealed the District Court’s decision to the Eleventh Circuit Court of Appeals. There can be no assurance of the result of such proceeding.
On August 10, 2020, Health Canada issued a Notice of Compliance (NOC) to Medunik for Ruzurgi needed to meet the standards of the Canadian Food and Drugs Act.
®
for the treatment of LEMS. We have 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 us. As such, we believe that our data was relied upon to establish the nonclinical safety profile of Ruzurgi®
On June 3, 2021, we announced a positive decision in this proceeding that quashed the NOC previously issued for Ruzurgi
®
and remanded the matter to the Minister of Health to redetermine its decision to grant marketing authorization to Ruzurgi®
in spite of Firdapse®
’s data protection rights. However, on June 28, 2021, we announced that Health Canada had re-issued
an NOC for Ruzurgi®
, once again allowing the product to be marketed in Canada for patients with LEMS. As a result, in early July 2021, we, along with our partner KYE, filed a second suit against Health Canada to overturn their most recent decision. There can be no assurance as to the outcome of this proceeding. 36
Patent Litigation
All of our patent rights for Firdapse
®
are derived from our license agreement. In August 2020, the United States Patent and Trademark Office (USPTO) allowed U.S. Patent No. 10,793,893 (the ’893 patent) to our licensor and thereby to us, and the patent issued on October 6, 2020. The patent is directed to the use of suitable doses of amifampridine to treat patients, regardless of the therapeutic indication, that are slow metabolizers of amifampridine. Any drug product containing amifampridine with a label that states the patented dosing regimens and doses in the Dosing and Administration section prior to April 7, 2034, the expiration date of the patent, could possibly infringe this patent. Generic drug product labels would necessarily have to do this, and we intend to take all appropriate actions to protect our intellectual property. In that regard, in October 19, 2020, we 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 the specialty pharmacy marketing Ruzurgi
®
, PantherRx Rare LLC (PantherRx), for infringement of the ‘893 Patent. The suits have since been combined in the U.S. District Court for New Jersey. The lawsuit arises from Jacobus’ and PantherRx’s sales and marketing of Ruzurgi®
(amifampridine) Tablets, 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 our patent rights. The lawsuit is in the discovery stage and there can be no assurance as to the results of this proceeding.
Other Litigation
From time to time we may become involved in legal proceedings arising in the ordinary course of business. Other than as set forth above, we believe that there is no litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on our results of operations, financial condition or cash flows.
ITEM 1A. |
RISK FACTORS |
There are many factors that affect our business, our financial condition, and the results of our operations. In addition to the information set forth in this quarterly report, you should carefully read and consider “Item 1A. Risk Factors” in Part I, and “Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part II, of our 2020 Annual Report on Form
10-K
filed with the SEC, which contain a description of significant factors that might cause our actual results of operations in future periods to differ materially from those currently expected or desired. 37
ITEM 2. |
UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS |
Issuer Purchases of Equity Securities
In March 2021, the Company’s Board of Directors approved a share repurchase program that authorizes the repurchase of up to $40 million of the Company’s common stock, pursuant to a repurchase plan under Rule
10b-18
of the Securities Act. The share repurchase program commenced on March 22, 2021. | Period |
Total Number of Shares Purchased |
Average Price Paid Per Share |
Total Number of Shares Purchased as Part of Publicly Announced Program |
Dollar Value of Shares that May Yet Be Purchased (in thousands) |
||||||||||||
| April 1, 2021 – April 30, 2021 |
432,951 | $ | 4.53 | 432,951 | $ | 37,748 | ||||||||||
| May 1, 2021 – May 31, 2021 |
9,260 | $ | 4.62 | 9,260 | $ | 37,705 | ||||||||||
| June 1, 2021 – June 30, 2021 |
290,740 | $ | 5.73 | 290,740 | $ | 36,040 | ||||||||||
ITEM 3. |
DEFAULTS UPON SENIOR SECURITIES |
None
ITEM 4. |
MINE SAFETY DISCLOSURE |
Not applicable
ITEM 5. |
OTHER INFORMATION |
None
ITEM 6. |
EXHIBITS |
| 31.1 | Certification of Principal Executive Officer under Section 302 of the Sarbanes-Oxley Act of 2002 | |
| 31.2 | Certification of Principal Financial Officer under Section 302 of the Sarbanes-Oxley Act of 2002 | |
| 32.1 | Certification of Principal Executive Officer under Section 906 of the Sarbanes-Oxley Act of 2002 | |
| 32.2 | Certification of Principal Financial Officer under Section 906 of the Sarbanes-Oxley Act of 2002 | |
| 101.INS | XBRL Instance Document | |
| 101.SCH | XBRL Taxonomy Extension Schema | |
| 101.CAL | XBRL Taxonomy Extension Calculation Linkbase | |
| 101.DEF | XBRL Taxonomy Extension Definition Linkbase | |
| 101.LAB | XBRL Taxonomy Extension Label Linkbase | |
| 101.PRE | XBRL Taxonomy Extension Presentation Linkbase | |
| 104 | Cover Page Interactive Data File (formatted as Inline XBRL and contained in Exhibit 101) | |
38
SIGNATURES
Pursuant to the Securities Exchange Act of 1934, the Registrant has caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| Catalyst Pharmaceuticals, Inc. | ||
| By: | /s/ Alicia Grande | |
| Alicia Grande | ||
| Vice President, Treasurer and Chief Financial Officer | ||
Date: August 9, 2021
39
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 registrant’s 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 registrant’s 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 registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and |
| 5. | The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of registrant’s 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 registrant’s 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 registrant’s internal control over financial reporting. |
Date: August 9, 2021
| /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 registrant’s 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 registrant’s 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 registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and |
| 5. | The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of registrant’s 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 registrant’s 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 registrant’s internal control over financial reporting. |
Date: August 9, 2021
| /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 June 30, 2021 (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: August 9, 2021 | /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 June 30, 2021 (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: August 9, 2021 | /s/ Alicia Grande | |||
| Alicia Grande | ||||
| Chief Financial Officer | ||||
| (Principal Financial Officer) |