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 September 30, 2020
OR
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the transition period from to
Commission file number: 001-37539
(Exact name of registrant as specified in its charter)
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) |
(Address of principal executive offices)
(650 ) 741-7700
(Registrant’s telephone number, including area code)
Securities registered pursuant to Section 12(b) of the Act:
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
The |
Indicate by check mark 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 reports), 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 Yes ☒ No ☐
Regulation S-T
(§232.405 of this chapter) 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, smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2
of the Exchange Act.| ☒ | 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 provided 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 As of October 30, 2020, there were 61,796,443 shares of the registrant’s Common Stock, par value $0.001 per share, outstanding.
TABLE OF CONTENTS
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| Item 1. |
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| Item 3. |
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| Item 1. |
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| Item 1A. |
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| Item 5. |
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| Item 6. |
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2
PART I. – FINANCIAL INFORMATION
Item 1. |
Financial Statements |
GLOBAL BLOOD THERAPEUTICS, INC.
Condensed Consolidated Balance Sheets
(In thousands, except share and per share amounts)
September 30, 2020 |
December 31, 2019 |
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(Unaudited) |
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| Assets |
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| Current assets: |
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| Cash and cash equivalents |
$ | $ | ||||||
| Short-term marketable securities |
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| Accounts receivable, net |
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| Inventories |
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| Prepaid expenses and other current assets |
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| Total current assets |
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| Property and equipment, net |
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| Long-term marketable securities |
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| Operating lease right-of-use |
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| Restricted cash |
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| Other assets, noncurrent |
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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 liabilities |
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| Accrued compensation |
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| Other liabilities, current |
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| Total current liabilities |
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| Long-term debt |
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| Operating lease liabilities, noncurrent |
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| Other liabilities, noncurrent |
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| Total liabilities |
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| Commitments and contingencies |
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| Stockholders’ equity: |
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| Preferred stock, $ |
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| Common stock, $ |
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| Additional paid-in capital |
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| Accumulated other comprehensive income |
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| Accumulated deficit |
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| Total stockholders’ equity |
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| Total liabilities and stockholders’ equity |
$ | $ | ||||||
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See accompanying notes to unaudited condensed consolidated financial statements.
3
GLOBAL BLOOD THERAPEUTICS, INC.
Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)
(In thousands, except share and per share amounts)
Three Months Ended September 30, |
Nine Months Ended September 30, |
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2020 |
2019 |
2020 |
2019 |
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| Product sales, net |
$ | $ | — | $ | $ | — | ||||||||||
| Costs and operating expenses: |
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| Cost of sales |
— | — | ||||||||||||||
| Research and development |
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| Selling, general and administrative |
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| Total costs and operating expenses |
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| Loss from operations |
( |
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| Other income (expense): |
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| Interest income |
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| Interest expense |
( |
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| Other expenses, net |
( |
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| Total other income (expense), net |
( |
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| Net loss |
( |
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| Other comprehensive loss: |
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| Net unrealized gain (loss) on marketable securities, net of tax |
( |
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| Comprehensive loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
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| Basic and diluted net loss per common share |
$ | ( |
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) | $ | ( |
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| Weighted-average number of shares used in computing basic and diluted net loss per common share |
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See accompanying notes to unaudited condensed consolidated financial statements.
4
GLOBAL BLOOD THERAPEUTICS, INC.
Condensed Consolidated Statements of Stockholders’ Equity
(Unaudited)
(In thousands, except share amounts)
Common Stock |
Accumulated |
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Shares |
Amount |
Additional Paid- In Capital |
Other Comprehensive Income |
Accumulated Deficit |
Total Stockholders’ Equity |
|||||||||||||||||||
| Balance at December 31, 2019 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
| Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes |
— | ( |
) | — | — | ( |
) | |||||||||||||||||
| Issuance of common stock pursuant to ESPP purchases |
— | — | — | |||||||||||||||||||||
| Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
| Net unrealized gain on marketable securities |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | — | ( |
) | ( |
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| Balance at March 31, 2020 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
| Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes |
— | ( |
) | — | — | ( |
) | |||||||||||||||||
| Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
| Net unrealized gain on marketable securities |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | — | ( |
) | ( |
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| Balance at June 30, 2020 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
| Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes |
— | ( |
) | — | — | ( |
) | |||||||||||||||||
| Issuance of common stock pursuant to ESPP purchases |
— | — | — | |||||||||||||||||||||
| Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
| Net unrealized loss on marketable securities |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
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| Balance at September 30, 2020 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
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5
Common Stock |
Accumulated |
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Shares |
Amount |
Additional Paid- In Capital |
Other Comprehensive Income (Loss) |
Accumulated Deficit |
Total Stockholders’ Equity |
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| Balance at December 31, 2018 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||
| Issuance of common stock upon equity offerings, net of issuance costs |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes |
— | ( |
) | — | — | ( |
) | |||||||||||||||||
| Issuance of common stock pursuant to ESPP purchases |
— | — | — | |||||||||||||||||||||
| Vesting of restricted stock purchases |
— | — | — | |||||||||||||||||||||
| Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
| Net unrealized gain on marketable securities |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | — | ( |
) | ( |
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| Balance at March 31, 2019 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
| Issuance of common stock upon equity offerings, net of issuance costs |
— | — | ||||||||||||||||||||||
| Issuance of common stock upon exercise of stock options |
— | — | ||||||||||||||||||||||
| Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes |
— | ( |
) | — | — | ( |
) | |||||||||||||||||
| Vesting of restricted stock purchases |
— | — | — | |||||||||||||||||||||
| Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
| Net unrealized gain on marketable securities |
— | — | — | — | ||||||||||||||||||||
| Net loss |
— | — | — | — | ( |
) | ( |
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| Balance at June 30, 2019 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
| Issuance of common stock upon equity offerings, net of issuance costs |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
| Issuance of common stock upon vesting of restricted share units, net of shares withheld for employee taxes |
— | ( |
) | — | — | ( |
) | |||||||||||||||||
| Issuance of common stock pursuant to ESPP purchases |
— | — | — | |||||||||||||||||||||
| Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
| Net unrealized loss on marketable securities |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
| Net loss |
— | — | — | — | ( |
) | ( |
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| Balance at September 30, 2019 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
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See accompanying notes to unaudited condensed consolidated financial statements.
6
GLOBAL BLOOD THERAPEUTICS, INC.
Condensed Consolidated Statements of Cash Flows
(Unaudited)
(In thousands)
Nine Months Ended September 30, |
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2020 |
2019 |
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CASH FLOWS FROM OPERATING ACTIVITIES: |
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Net loss |
$ | ( |
) | $ | ( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
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Depreciation and amortization |
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Amortization (accretion) of premium (discount) on marketable securities |
( |
) | ||||||
Non-cash interest expense |
— | |||||||
Amortization of operating lease right-of-use |
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Stock-based compensation |
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Changes in operating assets and liabilities: |
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Accounts receivables |
( |
) | — | |||||
Inventories |
( |
) | — | |||||
Prepaid expenses and other assets |
( |
) | ( |
) | ||||
Accounts payable |
( |
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Accrued liabilities |
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Accrued compensation |
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Other liabilities, current |
( |
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) | ||||
Operating lease liabilities |
( |
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— |
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| ( |
) | ( |
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CASH FLOWS FROM INVESTING ACTIVITIES: |
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Purchase of property and equipment |
( |
) | ( |
) | ||||
Purchase of marketable securities |
( |
) | ( |
) | ||||
Maturities of marketable securities |
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| ( |
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CASH FLOWS FROM FINANCING ACTIVITIES: |
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Proceeds from issuance of common stock in public offering, net of issuance costs |
— | |||||||
Proceeds from issuance of common stock in settlement of employee stock purchase plan and exercise of stock options |
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Payments of debt issuance costs |
( |
) | — | |||||
Tax paid related to net share settlement of equity awards |
( |
) | ( |
) | ||||
Net increase (decrease) in cash, cash equivalents and restricted cash |
( |
) | ||||||
Cash, cash equivalents and restricted cash at beginning of period |
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Cash, cash equivalents and restricted cash at end of period |
$ | $ | ||||||
SUPPLEMENTAL DISCLOSURES OF CASH FLOW INFORMATION: |
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Cash paid for interest |
$ | $ | — | |||||
SUPPLEMENTAL DISCLOSURES OF NON-CASH INVESTING AND FINANCING INFORMATION: |
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Leasehold improvements paid for by landlord |
$ | $ | ||||||
Accrued purchase of property and equipment |
$ | $ | ||||||
Accrued issuance costs |
$ | ( |
) | $ | — | |||
Operating lease liabilities arising from obtaining right-of-use |
$ | $ | ||||||
RECONCILIATION OF CASH, CASH EQUIVALENTS, AND RESTRICTED CASH TO THE CONDENSED CONSOLIDATED BALANCE SHEETS |
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| $ | $ | |||||||
| $ | $ | |||||||
See accompanying notes to unaudited condensed consolidated financial statements.
7
GLOBAL BLOOD THERAPEUTICS, INC.
Notes to Unaudited Condensed Consolidated Financial Statements
1. Organization and Basis of Presentation
Global Blood Therapeutics, Inc., or the Company, we, us, or our, was incorporated in Delaware in February 2011 and commenced operations in May 2012. We are a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. In late November 2019, we received U.S. Food and Drug Administration, or FDA, accelerated approval for our first medicine, Oxbryta
®
(voxelotor) tablets for the treatment of sickle cell disease, or SCD, in adults and children 12 years of age and older. In early December 2019, we began to make Oxbryta available to patients through our specialty
pharmacy partner network. Our principal operations are based in South San Francisco, California, and we operate in one segment. Need for Additional Capital
In the course of our development activities, we have sustained operating losses, and we expect such losses to continue over the next several years. Our ultimate success depends on the outcome of our commercial launch of Oxbryta, research and development and business development activities. Since inception through September 30, 2020, we have incurred cumulative net losses of $924.7 million. We expect to incur additional losses for the foreseeable future to commercialize Oxbryta and conduct product research and development, and expect to potentially raise additional capital to fully implement our business plan. If needed, we intend to raise such capital through borrowings, the issuance of additional equity, and potentially through strategic alliances with partner companies or other transactions. However, if such financing is not available at adequate levels, we will need to
re-evaluate
our operating plans. We believe that our existing cash and cash equivalents and marketable securities will be sufficient to fund our cash requirements for at least twelve months subsequent to the issuance of these financial statements.2. Summary of Significant Accounting Policies
Basis of Preparation
The accompanying unaudited interim condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP, and applicable rules and regulations of the Securities and Exchange Commission, or SEC, regarding interim financial reporting. As permitted under those rules, certain footnotes or other financial information that are normally required by U.S. GAAP have been condensed or omitted, and accordingly the balance sheet as of December 31, 2019 has been derived from audited consolidated financial statements at that date but does not include all of the information required by U.S. GAAP for complete financial statements. These unaudited interim condensed consolidated financial statements have been prepared on the same basis as our annual consolidated financial statements and, in the opinion of management, reflect all adjustments (consisting only of normal recurring adjustments) that are necessary for a fair presentation of our financial information. The results of operations for the nine months ended September 30, 2020 are not necessarily indicative of the results to be expected for the year ending December 31, 2020 or for any other interim period or for any other future year.
The accompanying unaudited interim condensed consolidated financial statements and related financial information should be read in conjunction with the audited financial statements and the related notes thereto for the year ended December 31, 2019 included in our Annual Report on Form
10-K,
filed with the SEC on February 26, 2020.Use of Estimates
The preparation of the accompanying consolidated financial statements in accordance with U.S. GAAP requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements, and the reported amounts of revenues and expenses during the reporting period. We base our estimates and assumptions on historical experience when available and on various factors that we believe to be reasonable under the circumstances. We evaluate our estimates and assumptions on an ongoing basis. Our actual results could differ from these estimates under different assumptions or conditions.
Concentration of Risk
Credit Risk
We invest in a variety of financial instruments and, by our Board approved investment policy, limit the amount of credit exposure with any one issuer, industry or geographic area for investments other than instruments backed by the U.S. federal government.
8
Major Customers
We have entered into distribution agreements with certain limited specialty pharmacies and specialty distributors. For the nine months ended September 30, 2020, our two largest customers represented approximately 94 % of our product revenue and approximately 86 % of our accounts receivable balance at September 30, 2020.
Major Suppliers
We do not currently have any of our own manufacturing facilities, and therefore depend on an outsourced manufacturing strategy for the production of Oxbryta for commercial use and for the production of our product candidates for clinical trials. We have contracts in place with one third-party manufacturer that is approved for the commercial production of Oxbryta and one third-party supplier that is approved for Oxbryta’s active pharmaceutical ingredient. Although there are potential sources of supply other than our existing manufacturers and suppliers, any new supplier would be required to qualify under applicable regulatory requirements.
Principles of Consolidation
The accompanying unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany transactions and balances have been eliminated upon consolidation.
Significant Accounting Policies
Except as noted below, there have been no material revisions in our significant accounting policies described in Note 2 to the consolidated financial statements included in our Annual Report on Form
10-K
for the year ended December 31, 2019.Accounting Pronouncements Adopted
In August 2018, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, , or ASU
No. 2018-15,
Intangibles—Goodwill and
Other—Internal-Use
Software (Subtopic 350-40),
Customer’s Accounting for Implementation Costs Incurred in a Cloud ComputingArrangement That Is a Service Contract
2018-15.
ASU No. 2018-15
requires a customer that is a party to a cloud computing service contract to follow the internal-use
software guidance in Subtopic 350-40
to determine which implementation costs to capitalize and which costs to expense. The amendments in this update are effective for annual reporting periods beginning after December 15, 2019, and interim periods within those fiscal years. The amendments in this update should be applied either retrospectively or prospectively to all implementation costs incurred after the date of adoption. We adopted ASU No. 2018-15
in the first quarter of 2020 and applied the guidance prospectively to the implementation costs incurred in our implementations of various cloud computing arrangements that are service contracts. The adoption of this new standard did not have a material impact on our condensed consolidated financial statements.In August 2018, the FASB issued ASU . The new standard modifies the disclosure requirements on fair value measurements in Topic 820, Fair Value Measurement, including removals of, modification to, and additional disclosure requirements from Topic 820. The amendment of ASU
No. 2018-13,
Fair Value Measurement (Topic 820)
No. 2018-13
removes disclosure requirements from Topic 820 in the areas of (1) the amount of and reasons for transfers between Level 1 and Level 2 of the fair value hierarchy, (2) the policy for timing of transfers between levels, and (3) the valuation processes for Level 3 fair value measurements. The amendments in this update are effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. Except for certain amendments related to Level 3 fair value measurements, all the other amendments should be applied retrospectively to all periods presented upon their effective date. We adopted ASU No. 2018-13
in the first quarter of 2020 and applied the guidance retrospectively. The adoption of this new standard did not have a material impact on our condensed consolidated financial statements.In June 2016, the FASB issued ASU , which amends the guidance on the impairment of financial instruments. The new standard adds to U.S. GAAP an impairment model that is based on expected losses rather than incurred losses, which is known as the current expected credit loss, or CECL model. The CECL model applies to most debt instruments (other than those measured at fair value), trade and other receivables, financial guarantee contracts, and loan commitments. debt securities are scoped out of this guidance. Our investment portfolio primarily consists of debt securities carried at fair value. Our accounts
No. 2016-13,
Measurement of Credit Losses on Financial Instruments (Topic 326)
Available-for-sale
available-for-sale
receivable are not long
term
in nature
and we do not expect to write off accounts receivable. The amendments in this update are effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. We adopted ASU No. 2016-13
in the first quarter of 2020 and applied the guidance prospectively. The adoption of this new standard did not have a material impact on our condensed consolidated financial statements.9
3. Fair Value Measurements
Fair value accounting is applied for all financial assets and liabilities that are recognized or disclosed at fair value in the consolidated financial statements on a recurring basis (at least annually). Our financial instruments consist of cash and cash equivalents, marketable securities, accounts receivables, accounts payable and accrued liabilities. Cash and cash equivalents, marketable securities and restricted cash are reported at their respective fair values on our condensed consolidated balance sheets. The remaining financial instruments are reported on our condensed consolidated balance sheets at cost that approximate current fair values due to their relatively short maturities.
Assets and liabilities recorded at fair value on a recurring basis in the condensed consolidated balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:
Level
1
Level
2
Level
3
The following table summarizes our financial assets measured at fair value on a recurring basis (in thousands):
September 30, 2020 |
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Total |
Level 1 |
Level 2 |
Level 3 |
|||||||||||||
| Financial Assets: |
||||||||||||||||
| Money market funds |
$ | $ | $ | — | $ | — | ||||||||||
| Corporate debt securities |
— | — | ||||||||||||||
| U.S. government agency securities |
— | — | ||||||||||||||
| Certificates of deposits |
— | — | ||||||||||||||
| U.S. government securities |
— | — | ||||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total financial assets |
$ | $ | $ | $ | — | |||||||||||
| |
|
|
|
|
|
|
|
|||||||||
December 31, 2019 |
||||||||||||||||
Total |
Level 1 |
Level 2 |
Level 3 |
|||||||||||||
| Financial Assets: |
||||||||||||||||
| Money market funds |
$ | $ | $ | — | $ | — | ||||||||||
| Corporate debt securities |
— | — | ||||||||||||||
| U.S. government agency securities |
— | — | ||||||||||||||
| Certificates of deposits |
— | — | ||||||||||||||
| U.S. government securities |
— | — | ||||||||||||||
| |
|
|
|
|
|
|
|
|||||||||
| Total financial assets |
$ | $ | $ | $ | — | |||||||||||
| |
|
|
|
|
|
|
|
|||||||||
We estimate the fair values of our investments in corporate debt securities, government and government related securities and certificates of deposits by taking into consideration valuations obtained from third-party pricing services. The fair value of our marketable securities classified within Level 2 is based upon observable inputs that may include benchmark yields, reported trades, broker/dealer quotes, issuer spreads, or A/A2, or higher, by Moody’s and Standard and Poor’s.
two-sided
markets, benchmark securities, bids, offers and reference data including market research publications. At September 30, 2020 and December 31, 2019, the weighted average remaining contractual maturities of our Level 2 investments was less than one year and all of these investments are rated A-1/P-1
10
4. Securities
Available-for-Sale
Estimated fair values of securities are generally based on prices obtained from commercial pricing services. The following table is a summary of securities recorded in cash and cash equivalents, restricted cash, or marketable securities in our condensed consolidated balance sheets (in thousands):
available-for-sale
available-for-sale
September 30, 2020 |
December 31, 2019 |
|||||||||||||||||||||||||||||||
Amortized Cost |
Unrealized Gains |
Unrealized (Losses) |
Estimated Fair Value |
Amortized Cost |
Unrealized Gains |
Unrealized (Losses) |
Estimated Fair Value |
|||||||||||||||||||||||||
| Financial Assets: |
||||||||||||||||||||||||||||||||
| Money market funds |
$ | $ | — | $ | — | $ | $ | $ | — | $ | — | $ | ||||||||||||||||||||
| Corporate debt securities |
— | ( |
) | |||||||||||||||||||||||||||||
| U.S. government agency securities |
— | ( |
) | |||||||||||||||||||||||||||||
| Certificates of deposits |
— | |||||||||||||||||||||||||||||||
| U.S. government securities |
— | |||||||||||||||||||||||||||||||
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
| Total |
$ | $ | $ | — | $ | $ | $ | $ | ( |
) | $ | |||||||||||||||||||||
| |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||||
The following table summarizes the classification of the securities on our condensed consolidated balance sheets (in thousands):
available-for-sale
September 30, 2020 |
December 31, 2019 |
|||||||
| Cash and cash equivalents |
$ | $ | ||||||
| Short-term marketable securities |
||||||||
| Long-term marketable securities |
||||||||
| |
|
|
|
|||||
| Total |
$ | $ | ||||||
| |
|
|
|
|||||
We do not intend to sell the investments that are in an unrealized loss position, and it is unlikely that we will be required to sell the investments before recovery of their amortized cost basis, which may be maturity. We have determined that the gross unrealized losses on our marketable securities were temporary in nature during the periods presented.
5. Balance Sheet Components
Inventories
We began capitalizing inventories in November 2019 once the FDA approved Oxbryta. Inventories consist of the following (in thousands):
September 30, 2020 |
December 31, 2019 |
|||||||
| Raw materials |
$ | $ | ||||||
| Work-in-process |
||||||||
| Finished goods |
||||||||
| |
|
|
|
|||||
| Total inventories |
$ | $ | ||||||
| |
|
|
|
|||||
11
Property and Equipment, Net
Property and equipment consists of the following (in thousands):
September 30, 2020 |
December 31, 2019 |
|||||||
Laboratory equipment |
$ | $ | ||||||
Computer equipment |
||||||||
Leasehold improvements |
||||||||
Construction-in-progress |
||||||||
Total property and equipment |
||||||||
Less: accumulated depreciation and amortization |
( |
) | ( |
) | ||||
Property and equipment, net |
$ | $ | ||||||
Accrued liabilities
Accrued liabilities consist of the following (in thousands):
September 30, 2020 |
December 31, 2019 |
|||||||
Accrued research and development costs |
$ | $ | ||||||
Accrued manufacturing costs |
||||||||
Accrued professional and consulting services |
||||||||
Accrued sales deductions |
||||||||
Other |
||||||||
Total accrued liabilities |
$ | $ | ||||||
Other liabilities, current
Other liabilities consist of the following (in thousands):
September 30, 2020 |
December 31, 2019 |
|||||||
Operating lease liabilities, current |
$ | $ | ||||||
Other payable |
||||||||
Total other liabilities, current |
$ | $ | ||||||
6. Long-term Debt
Term Loan
On December 17, 2019, we entered into the Loan Agreement, or Term Loan, with funds managed by Pharmakon Advisors LP, which are BioPharma Credit PLC, as collateral agent, Biopharma Credit Investments V (Master) LP, as a lender, and BPCR Limited Partnership, as a lender, and collectively, the Lenders, for a senior secured credit facility consisting of an initial tranche of $75.0 million and the option to draw an additional $75.0 million until December 31, 2020. The first tranche, in the amount of $75.0 million, was funded in connection with the closing date of the Term Loan in December 2019. In June 2020, in accordance with the terms of the Loan Agreement, we provided notification to Lenders of our intention to draw the second tranche of the Term Loan, in the amount of $75.0 million, prior to December 31, 2020, subject to certain closing conditions. As of September 30, 2020, the second tranche of the Term Loan has not been drawn.
The Term Loan carries a 72-month term. The Term Loan bears interest at a floating per annum interest rate equal to 7.00 % plus the greater of (a) the 2 %. In the event we default, the interest rate would be 3 % above the rate that is otherwise applicable thereto. Interest on amounts outstanding are payable quarterly in arrears . The Term Loan repayment schedule provides for interest only payments for the first 39 months , followed by consecutive equal quarterly payments of principal and interest commencing in March 2023 and continuing through the maturity of December 2025.
3-month
LIBOR rate and (b) We have the option to prepay all or a portion of the borrowed amounts under the Term Loan. If we exercise this option, we must pay a prepayment fee between 1 % and 3 % of the principal amount being prepaid depending on the timing of the prepayment, or Prepayment Fee. If the prepayment occurs before December 2022, we must also pay an amount equal to the sum of all interest that would have accrued and been payable from date of prepayment through December 2022, or Make Whole Amount. We are obligated to pay an additional fee to the Lenders determined by multiplying the principal amount being paid or prepaid multiplied by 2 %, or Paydown Fee, when such payments are made.
12
In the event of default or change in control, all unpaid principal and all accrued and unpaid interest amounts (if any) become immediately due and payable, at which point, we will be subject to the Prepayment Fee, the Make Whole Amount (if any) and the Paydown Fee. Events of default include, but are not limited to, a payment default, a material adverse change, and insolvency. The obligations under the Term Loan are secured by a first priority security interest in and a lien on substantially all of our assets, subject to certain exceptions.
Debt issuance costs paid directly to the Lenders of $1.1 million and the other debt issuance costs of $0.4 million were accounted for as discounts on the Term Loan. These debt discounts along with the Paydown Fee are being amortized or accreted to interest expenses throughout the life of the Term Loan using the effective interest rate method. As of September 30, 2020, there were unamortized issuance costs and debt discounts of $1.4 million, which were recorded as a direct deduction from the Term Loan on the condensed consolidated balance sheet. In addition, we paid the Lenders $1.1 million for the option to draw the additional $75.0 million, which was capitalized as a deferred asset and which is included in other assets, current and amortized on a straight-line basis through December 31, 2020. As of September 30, 2020, the unamortized fee for the option to draw the additional funds was $0.3 million.
Future payments of principal and interest on the Term Loan as of September 30, 2020 were as follows (in thousands):
Year ending December 31, |
Amount |
|||
2020 (three months) |
$ | |||
2021 |
||||
2022 |
||||
2023 |
||||
2024 |
||||
2025 |
||||
Total minimum payments |
||||
Less amount representing interest |
( |
) | ||
Less amount representing Paydown Fee |
( |
) | ||
Long-term debt, gross |
||||
Discount on notes payable |
( |
) | ||
Accretion of Paydown Fee |
||||
Long-term debt |
$ | |||
7. Commitments and Contingencies
Leases
We have operating leases for our headquarters, where we have office and research and development laboratory facilities, and equipment. Our leases have remaining lease terms of 1 to 10 years. Most of these leases require monthly lease payments that may be subject to annual increases throughout the lease term. Certain of these leases include renewal options at our election, with renewal terms that can extend the lease term from 1 to 10 years. These optional periods have not been considered in the determination of the or ROU, assets or lease liabilities associated with these leases as we did not consider it reasonably certain that we would exercise the options.
right-of-use,
Lease costs included in operating expenses in the consolidated statement of operations in relation to these operating leases were $3.1 million and $1.4 million for the three months ended September 30, 2020 and 2019, respectively; and $9.3 million and $4.6 million for the nine months ended September 30, 2020 and 2019, respectively. Included in these lease costs were variable lease costs, which were not included within the measurement of our operating ROU assets and operating lease liabilities in the amount of $0.7 million and $0.4 million for the three months ended September 30, 2020 and 2019, respectively; and $2.0 million and $1.8 million for the nine months ended September 30, 2020 and 2019, respectively. The variable lease cost is comprised primarily of our proportionate share of operating expenses, property taxes, and insurance in relation with our facility lease. These costs are classified as operating lease expense due to our election to not separate lease and
non-lease
components.13
Supplemental cash flow information related to leases for the period reported is as follows (in thousands, except weighted-average remaining lease term and weighted-average discount rate):
Nine Months Ended September 30, |
||||||||
2020 |
2019 |
|||||||
ROU assets obtained in exchange for new operating lease |
$ | $ | ||||||
Cash paid for amounts included in the measurement of lease liabilities |
||||||||
Weighted-average remaining lease term of operating leases (in years) |
||||||||
Weighted-average discount rate of operating leases |
% | % | ||||||
The majority of our lease costs are driven by our operating lease for our headquarters in South San Francisco, where we have office and research and development laboratory facilities.
In March 2017, we entered into a noncancelable operating lease, or Original Lease, for approximately 67,185 square feet of space in South San Francisco, California, or Prior Premises. The Original Lease term commenced in November 2017 as we gained control over physical access to the Prior Premises. We acquired $11.1 million of leasehold improvements at the Prior Premises with the tenant inducement allowance provided under the Original Lease. We are required to repay $1.7 million of the tenant inducement allowance, or Initial Allowance, to the landlord in the form of additional monthly rent with interest applied over the term of the Original Lease.
In August 2018, we entered into an amendment to the Original Lease, or Lease Amendment, to relocate the leased premises from the Prior Premises to a building consisting of approximately 164,150 rentable square feet of space, or Substitute Premises. The date on which we became responsible for paying rent under the Lease Amendment was March 13, 2020, or the Substitute Premises Payment Commencement Date. The Lease Amendment has a contractual term, or Substitute Premises Term, of 10 years from the Substitute Premises Payment Commencement Date, which was subsequently determined to be in March 2020. The Lease Amendment grants us an option to extend the Lease Amendment for an additional 10 -year period. Future minimum rental payments under the Lease Amendment during the 10 -year term are $121.5 million in the aggregate. Under the Lease Amendment, we are obligated to pay to the landlord certain costs, including taxes and operating expenses. On October 1, 2019, we determined that the Lease Amendment for the Substitute Premises had commenced as we had the right to control the Substitute Premises. The Lease Amendment also provides a tenant inducement allowance of up to $27.9 million, of which $4.1 million, if utilized, would be repaid to the landlord in the form of additional monthly rent with interest applied. As of September 30, 2020, we have capitalized $32.3 million of costs in leasehold improvements within property and equipment, net for construction of leasehold improvements at the Substitute Premises, which were mostly acquired with the tenant inducement provided under the Lease Amendment.
to-be-constructed
We vacated the Prior Premises and surrendered and delivered the Prior Premises to the landlord in May 2020, upon which time we had no further obligations with respect to the Prior Premises other than with respect to the Initial Allowance, which we will repay to the landlord in the form of additional monthly rent with interest applied over the term of the Original Lease. Upon signing of the Lease Amendment, we 3.8 million and $5.2 million for the nine months ended September 30, 2020 and 2019, respectively.
re-evaluated
the remaining useful life of the leasehold improvements at the Prior Premises and started to amortize the leasehold improvements over the remaining period of expected use, resulting in an acceleration of depreciation expenses for approximately $As of September 30, 2020, the maturities of our operating lease liabilities were as follows (in thousands):
Year ending December 31, |
Amount |
|||
2020 (three months) |
$ | |||
2021 |
||||
2022 |
||||
2023 |
||||
2024 |
||||
2025 |
||||
Thereafter |
||||
Total lease payments |
||||
Less: Imputed interest |
( |
) | ||
Present value of operating lease liabilities |
$ | |||
14
Contingencies
In the ordinary course of business, we may be subject to legal claims and regulatory actions that could have a material adverse effect on our business or financial position. We assess our potential liability in such situations by analyzing potential outcomes, assuming various litigation, regulatory and settlement strategies. If we determine a loss is probable and its amount can be reasonably estimated, we accrue an amount equal to the estimated loss.
8. Stock-Based Compensation
We have three stock-based compensation plans – the Amended and Restated 2017 Inducement Equity Plan, or 2017 Inducement Plan, the Amended and Restated 2015 Stock Option and Incentive Plan, or 2015 Plan, and the 2012 Stock Option and Grant Plan, or 2012 Plan. As of September 30, 2020, there were 1,521,896 shares reserved under the 2017 Inducement Plan and 4,626,324 shares reserved under the 2015 Plan for the future issuance of equity awards. Upon adoption of the 2015 Plan in July 2015, no new awards or grants are permitted under the 2012 Plan. See Note 10 to the Consolidated Financial Statements included in our Annual Report on Form
10-K
for the year ended December 31, 2019 for additional information related to these stock-based compensation plans.Stock Options
The following summarizes option activity under the 2017 Inducement Plan, 2015 Plan and 2012 Plan:
Number of Options |
Weighted- Average Exercise Price |
|||||||
Outstanding — December 31, 2019 |
$ | |||||||
Options granted |
||||||||
Options exercised |
( |
) | ||||||
Options canceled |
( |
) | ||||||
Outstanding — September 30, 2020 |
$ | |||||||
The fair values of stock options granted to employees were calculated using the following assumptions:
Three Months Ended September 30, |
Nine Months Ended September 30, | |||||||
2020 |
2019 |
2020 |
2019 | |||||
Expected term (in years) |
||||||||
Volatility |
||||||||
Risk-free interest rate |
||||||||
Dividend yield |
||||||||
Restricted Stock Units
The following table summarizes activity of restricted stock units, or RSUs, granted to employees with service-based vesting under the 2017 Inducement Plan and 2015 Plan and related information:
Number of RSUs |
Weighted- Average Grant Date Fair Value |
|||||||
Non-vested units — December 31, 2019 |
$ | |||||||
RSUs granted |
||||||||
RSUs vested |
( |
) | ||||||
RSUs forfeited |
( |
) | ||||||
Non-vested units — September 30, 2020 |
$ | |||||||
15
Market-Condition RSUs Granted to Employees
The Compensation Committee of our Board of Directors granted, effective June 1, 2020, awards of up to an aggregate of 414,700 RSUs to certain of our senior management, including our executive officers, under the 2015 Plan, the vesting of which is contingent upon the achievement of three escalating stock price targets. Upon the achievement of the respective stock price targets,
fifty percent of the RSUs allotted to that tranche will vest, while the remaining fifty percent will vest on the first anniversary of the date the stock price target was achieved, subject to the employee’s continued employment or other service relationship with the Company through such vesting date. Under the terms of the awards, if the stock price targets are not achieved for all or some of the tranches on or before June 30, 2024, the unvested awards will be automatically terminated and forfeited. The compensation cost for the RSUs with a market condition is not reversed when the market condition is not satisfied. The target prices and
vesting
tranches are set forth in the table below:Stock Price Targets |
Number of Units Allotted | |
$ |
||
$ |
||
$ |
The grant date fair value of the market-condition awards was estimated using a Monte Carlo simulation model, which includes variables such as the expected volatility of the Company’s share price and interest rates to generate potential future outcomes. We recognize the related compensation expense on a straight-line basis over the applicable derived service periods, which are the estimated periods of time that would be required to satisfy the market conditions.
The following table summarizes activity of the market-condition awards under the 2015 Plan and related information (in thousands, except share, per share amounts and vesting period):
Number of Units |
Weighted- Average Grant Date Fair Value |
Weighted- Average Remaining Vesting Period (years) |
Aggregate Intrinsic Value |
|||||||||||||
Non-vested market-condition awards — December 31, 2019 |
$ | — | — | |||||||||||||
Granted |
||||||||||||||||
Vested |
— | — | ||||||||||||||
Forfeited |
( |
) | ||||||||||||||
Non-vested market-condition awards — September 30, 2020 |
$ | $ | ||||||||||||||
The following table summarizes the assumptions used to estimate the fair value of the market-condition awards as of the grant date:
Valuation date stock price |
$ | |||
Volatility |
% | |||
Risk-free interest rate |
% | |||
Dividend yield |
At September 30, 2020, total unrecognized compensation expense related to 16.4 million, which is expected to be recognized over their respective remaining derived service periods. The weighted average derived service period is 1.79 years. For the three months and nine months ended September 30, 2020, we recognized $3.3 million and $4.4 million, respectively, in stock-based compensation expense related to the market-condition awards.
non-vested
market-condition awards was $16
Stock-Based Compensation Expense
Total stock-based compensation recognized by function included in the condensed consolidated statements of operations and comprehensive loss was as follows (in thousands):
Three Months Ended September 30, |
Nine Months Ended September 30, |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||
Research and development |
$ | $ | $ | $ | ||||||||||||
Selling, general and administrative |
||||||||||||||||
Total stock-based compensation expense |
$ | $ | $ | $ | ||||||||||||
9. Net Loss per Share
Basic net loss per
share
is computed by dividing net loss by the weighted-average number of common shares outstanding for the period. Since we were in a loss position for all periods presented, diluted net loss per share is the same as basic net loss per share for all periods as the inclusion of all potential common shares outstanding would have been anti-dilutive. The following securities were not included in the diluted net loss per share calculations because their effect was anti-dilutive:
Three Months Ended September 30, |
Nine Months Ended September 30, |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||
Options to purchase common stock |
||||||||||||||||
Restricted stock units |
||||||||||||||||
Total |
||||||||||||||||
17
Item 2. |
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS |
You should read the following discussion and analysis of our financial condition and results of operations in conjunction with our unaudited condensed consolidated financial statements and related notes included in Part I, Item 1 of this Quarterly Report on Form
10-Q
and with our audited consolidated financial statements and related notes thereto for the year ended December 31, 2019, included in our Annual Report on Form 10-K
filed with the Securities and Exchange Commission on February 26, 2020, or our Annual Report. This discussion and other parts of this Quarterly Report on Form
10-Q
contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended, that involve risks and uncertainties, such as statements of our plans, objectives, expectations and intentions. In some cases you can identify forward-looking statements by terms such as “may,” “will,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “predict,” “potential,” “believe,” “should” and similar expressions. Our actual results could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the section of this Quarterly Report on Form 10-Q
titled “Risk Factors.” We caution readers not to place undue reliance on any forward-looking statements made by us, which speak only as of the date they are made. Except as may be required by law, we assume no obligation to update these forward-looking statements or the reasons that results could differ from these forward-looking statements. Overview
We are a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, we are delivering on our goal to transform the treatment and care of sickle cell disease, or SCD, a lifelong, devastating inherited blood disorder that is marked by red blood cell, or RBC, destruction and occluded blood flow and hypoxia, leading to anemia, stroke, multi-organ failure, severe pain crises, and shortened patient life span. As a result of the historic lack of treatment options, patients with SCD suffer serious morbidity and premature mortality.
It is estimated the prevalence of SCD is approximately 100,000 individuals in the United States, where newborn screening is mandatory, more than 100,000 people age 12 years and older in the Gulf Cooperation region, and approximately 52,000 individuals in Europe. The global incidence of SCD is estimated to be 250,000 to 300,000 births annually, and SCD is concentrated in populations of African, Middle Eastern and South Asian descent.
In November 2019, we received U.S. Food and Drug Administration, or FDA, accelerated approval for our first medicine, Oxbryta
®
(voxelotor) tablets for the treatment of SCD in adults and children 12 years of age and older. Oxbryta, an oral therapy taken once daily, is the first FDA-approved
treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD.The accelerated approval of Oxbryta is based on clinically meaningful and statistically significant improvements in hemoglobin levels, accompanied by reductions in RBC destruction (hemolysis). Data from the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study of 274 patients 12 years of age and older with SCD showed that, after 24 weeks of treatment, 51.1% of patients receiving Oxbryta achieved a greater than 1 g/dL increase in hemoglobin compared with 6.5% receiving placebo (p<0.001). The HOPE data also demonstrated corresponding improvements in other markers of hemolysis as well as a favorable safety and tolerability profile for Oxbryta.
In December 2019, we began to make Oxbryta available to patients through our specialty pharmacy partner network. As part of the product launch, we are focused on securing reimbursement and expanding patient access, and we established GBT Source Solutions
TM
, a comprehensive program for patients who are prescribed Oxbryta that provides a wide range of practical, educational and financial support customized to each patient’s needs. We are conducting and plan to conduct additional studies of Oxbryta, including our Phase 2a HOPE-KIDS 1 Study (an open-label, single- and multiple-dose Phase 2a study that is evaluating the safety, tolerability, pharmacokinetics and exploratory treatment effect of Oxbryta in pediatric patients aged 4 to 17 years with SCD) and, as a condition of accelerated approval, our Phase 3 HOPE-KIDS 2 Study (a post-approval confirmatory study we initiated in December 2019 that is using transcranial Doppler, or TCD, flow velocity to seek to demonstrate a decrease in stroke risk in children 2 to 15 years of age). We also expect to conduct additional clinical studies of Oxbryta, including to seek to expand the potential approved product label into younger pediatric populations, as well as other post-marketing studies.
In June 2020, we announced plans to submit by
mid-2021
a New Drug Application, or NDA, for Oxbryta, which will include a new age-appropriate
formulation, for the treatment of SCD in children ages 4 to 11 years under the FDA’s accelerated approval pathway. The NDA will include clinical data from our Phase 2a HOPE-KIDS 1 study. The FDA’s accelerated approval pathway can18
allow for earlier approval of drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint. In addition, we are initiating a multi-center Early Access Program, or EAP, in the United States for pediatric patients to provide access to treatment with Oxbryta prior to potential market authorization for children ages 4 to 11 who, in the judgment of their treating physician, have an urgent need for treatment for their SCD, have no comparable or satisfactory treatment options and are unable to participate in our clinical trials.
In June 2020, we announced plans to submit by
mid-2021
a Marketing Authorization Application, or MAA, to the European Medicines Agency, or EMA, seeking full marketing authorization of Oxbryta to treat hemolytic anemia (which is low hemoglobin due to red blood cell destruction) in SCD patients ages 12 years and older. The planned MAA will include data from our Phase 3 HOPE Study and our Phase 2 HOPE-KIDS 1 Study, both of which enrolled patients at clinical sites in Europe. In addition, we announced our intention to initiate an EAP in Europe for patients and physicians who may need access to Oxbryta prior to potential marketing authorization.In September 2020, we announced our entry into an exclusive agreement with Biopharma-Middle East and Africa to distribute Oxbryta in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and the United Arab Emirates, collectively known as the Gulf Cooperation Council, or GCC, region. In the GCC region, the U.S. approval of Oxbryta can be referenced to allow for access to the medicine while health authorities conduct their reviews.
Beyond Oxbryta, we are also engaged in other research and development activities, all of which are currently in earlier development stages, including working on new targets to develop the next generation of treatments for SCD. As part of our efforts to build our pipeline, we regularly evaluate opportunities to
in-license,
acquire or invest in new business, technology or assets or engage in related discussions with other business entities.In August 2018, we entered into the License Agreement, or Roche Agreement, with F.
Hoffmann-La
Roche Ltd. and Hoffmann-La
Roche Inc. (together, “Roche”) pursuant to which Roche granted us an exclusive and sublicensable worldwide license under certain patent rights and know-how
to develop and commercialize inclacumab, a p-selectin
inhibitor in development to address pain crises associated with the disease, including any modified compounds targeting p-selectin
and derived from inclacumab, for all indications and uses, except diagnostic use. Roche retained a non-exclusive,
worldwide, perpetual, royalty-free license to inclacumab solely for any diagnostic use. We are developing inclacumab as a treatment for vaso-occlusive crises, or VOCs, in patients with SCD, and we expect to be able to leverage the safety data from Roche’s prior clinical studies, which were not in patients with SCD, as we proceed with our development of inclacumab. We expect to initiate a pivotal clinical study by the end of the first half of 2021.In December 2019, we entered into the License and Collaboration Agreement, or Syros Agreement, with Syros Pharmaceuticals, Inc., or Syros, to discover, develop and commercialize novel therapies for SCD and beta thalassemia. Under the agreement, Syros will use its leading gene control platform to identify therapeutic targets and discover drugs that potentially induce fetal hemoglobin, and we have an option to obtain an exclusive worldwide license to develop, manufacture and commercialize any compounds or products resulting from the collaboration, subject to Syros’ option to
co-promote
the first product in the United States.The outbreak of the novel coronavirus, which causes coronavirus disease 2019
SARS-CoV-2,
(COVID-19),
has evolved into a global pandemic that has impacted our business, including our commercialization of Oxbryta and our research and development activities. For example, we have implemented a temporary work from home policy, temporarily suspended our field team from most in-person
interactions, including visits to physician offices, clinics and hospitals as well as in-person
meetings with payors, and temporarily delayed or paused certain research and development activities, including screening and enrollment in all GBT-sponsored
studies, including our Phase 2a HOPE-KIDS 1 Study, our Phase 3 HOPE-KIDS 2 Study and our dose optimization study that is intended to assess doses of Oxbryta of up to 3,000 mg per day. As we continue to monitor and work toward resumption of all trial activities, we are continuing with administrative trial-start up activities (such as contracting and IRB/EC approvals). Notably, the COVID-19
pandemic has not significantly impacted our supply of Oxbryta. We continue to believe we have an adequate supply of Oxbryta to sustain estimated patient need through the remainder of this year and into late 2021, and we are continuing to produce Oxbryta tablets. In addition, we are continuing to engage with healthcare professionals and payors through increased use of digital and internet-based education and outreach. We have seen a significant decrease in weekly new patient prescriptions for Oxbryta from a peak in early March, and we expect the rate of new patient prescriptions to remain lower, possibly through the fourth quarter of 2020, and potentially longer depending on the course of the pandemic. While we intend to resume normal operations as soon as practicable, we do not know for certain the extent or duration of these and other disruptions or the long-term impact on our business.We own or jointly own and have exclusively licensed rights to Oxbryta and our product candidates in the United States, Europe and other major markets. We are the sole owner of issued U.S. patents covering Oxbryta, including its composition of matter, methods of use, formulations and polymorphs of Oxbryta. These issued U.S. patents covering Oxbryta will expire between 2032 and 2037, absent any applicable patent term extensions. We own or
co-own
additional pending patent applications in the United States and multiple foreign countries relating to Oxbryta.19
We are not profitable and have incurred losses and negative cash flows from operations each year since our inception. Our net losses were $185.7 million and $170.8 million for the nine months ended September 30, 2020 and 2019, respectively. As of September 30, 2020, we had an accumulated deficit of $924.7 million. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations. We had $418.4 million in cash and cash equivalents and $116.8 million in marketable securities as of September 30, 2020.
Critical Accounting Policies and Estimates
Our management’s discussion and analysis of our financial condition and results of operations is based on our financial statements, which have been prepared in accordance with United States generally accepted accounting principles, or U.S. GAAP. The preparation of these financial statements requires us to make 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 financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the 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 under different assumptions or conditions.
There have been no material changes to our critical accounting policies from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report.
Results of Operations
Comparison of the Three Months Ended September 30, 2020 and 2019
Three Months Ended September 30, |
$ Change |
% Change |
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2020 |
2019 |
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(in thousands, except percentages) |
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| Product sales, net |
$ | 36,889 | $ | — | $ | 36,889 | * | |||||||||
| Costs and operating expenses: |
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| Cost of sales |
513 | — | 513 | * | ||||||||||||
| Research and development |
40,196 | 39,088 | 1,108 | 3 | % | |||||||||||
| Selling, general and administrative |
54,491 | 29,654 | 24,837 | 84 | % | |||||||||||
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| Total costs and operating expenses |
95,200 | 68,742 | 26,458 | 38 | % | |||||||||||
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| Loss from operations |
(58,311 | ) | (68,742 | ) | 10,431 | |||||||||||