sage-10q_20170930.htm

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

Form 10-Q

 

QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2017

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: 001-36544

 

Sage Therapeutics, Inc.

(Exact name of registrant as specified in its charter)

 

 

Delaware

27-4486580

(State or other jurisdiction of

incorporation or organization)

(I.R.S. Employer

Identification No.)

215 First Street

Cambridge, Massachusetts 02142

(Address of principal executive office) (Zip Code)

Registrant’s telephone number, including area code: (617) 299-8380

 

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 and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of 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 and post such files).    Yes      No  

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 the definitions of “large accelerated filer”, “accelerated filer”, “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

 

Large accelerated filer

Accelerated filer

 

 

 

 

Non-accelerated filer

  (Do not check if a smaller reporting company)

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, 2017, there were 37,537,655 shares of the registrant’s Common Stock, $0.0001 par value per share, outstanding.

 

 

 

 

 


 

Cautionary Note Regarding Forward-Looking Statements

This Quarterly Report on Form 10-Q, or Quarterly Report, contains forward-looking statements that involve risks and uncertainties. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may”, “will”, “should”, “expects”, “intends”, “plans”, “anticipates”, “believes”, “estimates”, “predicts”, “potential”, “continue” or the negative of these terms or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:

 

our plans to develop and commercialize our product candidates in the central nervous system, or CNS, disorders we discuss in this Quarterly Report, and potentially in other indications;

 

our ability, within the expected timeframes, to complete our ongoing clinical trials and non-clinical studies; to announce the results of such studies and trials; to advance our product candidates into additional clinical trials, including pivotal clinical trials; and to successfully complete such clinical trials;

 

our expectations as to the sufficiency of the planned clinical development programs for our product candidates, if successful, to support regulatory approval; our plans with respect to filing for regulatory approval of our product candidates and the timing of filings, if clinical development is successful; and our expectations with respect to the anticipated regulatory review pathway for our product candidates and the potential to obtain regulatory approval and to commercialize any product, if approved;

 

our estimates regarding expenses; use of cash; timing of future cash needs; and capital requirements;

 

our potential to achieve future revenues;

 

our expectations with respect to the availability of supplies of our product candidates, and the expected performance of our third-party manufacturers;

 

our ability to obtain and maintain intellectual property protection for our proprietary assets and other forms of exclusivity relevant to our business;

 

the estimated number of patients in indications of interest to us; the potential for our product candidates in those indications, if approved; the size of the potential markets for our product candidates; and our ability to serve those markets;

 

the anticipated rate and degree of market acceptance, and expectations regarding the pricing and potential availability and level of reimbursement, of our product candidates in any indication and in any country if approved;

 

our plans for expanding our activities, including outside the U.S., and the potential for future collaborations, partnerships or other types of contractual relationships, if appropriate, for accomplishing our strategic objectives;

 

the level of costs we may incur in connection with our activities, the possible timing and sources of future financings, and our ability to obtain additional financing when needed to fund future operations;

 

the potential for success of competing products that are or become available for the indications that we are pursuing or may pursue in the future;

 

the potential risk of loss of key scientific or management personnel; and

 

other risks and uncertainties, including those listed under Part II, Item 1A, Risk Factors.

Any forward-looking statements in this Quarterly Report reflect our current views with respect to future events and with respect to our future financial performance, and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, among other things, those described under Part II, Item 1A, Risk Factors and elsewhere in this Quarterly Report. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future.

2


 

This Quarterly Report contains estimates, projections and other information concerning our industry, the general business environment, and the markets for certain diseases, including estimates regarding the potential size of those markets and the estimated incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties, and actual events, circumstances or numbers, including actual disease prevalence rates and market size, may differ materially from the information reflected in this Quarterly Report. Unless otherwise expressly stated, we obtained this industry, business information, market data, prevalence information and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data, and similar sources, in some cases applying our own assumptions and analysis that may, in the future, prove not to have been accurate.

 

 

3


 

Sage Therapeutics, Inc.

INDEX

 

 

 

 

 

Page

PART I – FINANCIAL INFORMATION

 

 

 

 

 

 

 

Item 1.

 

Financial Statements (Unaudited)

 

5

 

 

Condensed Consolidated Balance Sheets as of September 30, 2017 and December 31, 2016

 

5

 

 

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three and nine months ended September 30, 2017 and 2016  

 

6

 

 

Condensed Consolidated Statements of Cash Flows for the nine months ended September 30, 2017 and 2016

 

7

 

 

Notes to Condensed Consolidated Financial Statements

 

8

Item 2.

 

Management’s Discussion and Analysis of Financial Condition and Results of Operations

 

19

Item 3.

 

Quantitative and Qualitative Disclosures About Market Risk

 

31

Item 4.

 

Controls and Procedures

 

31

 

 

 

PART II – OTHER INFORMATION

 

 

 

 

 

 

 

Item 1.

 

Legal Proceedings

 

32

Item 1A.

 

Risk Factors

 

32

Item 6.

 

Exhibits

 

60

 

 

Signatures

 

61

 

 

4


 

PART I — FINANCIAL INFORMATION

Item 1.

Financial Statements

Sage Therapeutics, Inc. and Subsidiaries

Condensed Consolidated Balance Sheets

(in thousands, except share and per share data)

(Unaudited)

 

 

 

September 30,

2017

 

 

December 31,

2016

 

Assets

 

 

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

 

 

Cash and cash equivalents

 

$

134,916

 

 

$

168,517

 

Marketable securities

 

 

108,535

 

 

 

228,962

 

Prepaid expenses and other current assets

 

 

4,892

 

 

 

5,100

 

Total current assets

 

 

248,343

 

 

 

402,579

 

Property and equipment, net

 

 

1,893

 

 

 

1,388

 

Restricted cash

 

 

849

 

 

 

564

 

Total assets

 

$

251,085

 

 

$

404,531

 

Liabilities and Stockholders’ Equity

 

 

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

 

 

Accounts payable

 

$

13,030

 

 

$

12,817

 

Accrued expenses

 

 

36,807

 

 

 

22,352

 

Total current liabilities

 

 

49,837

 

 

 

35,169

 

Other liabilities

 

 

1,196

 

 

 

845

 

Total liabilities

 

 

51,033

 

 

 

36,014

 

Commitments and contingencies (Note 5)

 

 

 

 

 

 

 

 

Stockholders’ equity:

 

 

 

 

 

 

 

 

Preferred stock, $0.0001 par value per share; 5,000,000 shares authorized at

   September 30, 2017 and December 31, 2016; no shares issued or

   outstanding at September 30, 2017 and December 31, 2016

 

 

 

 

 

 

Common stock, $0.0001 par value per share; 120,000,000 shares authorized at

   September 30, 2017 and December 31, 2016; 37,524,984 and 37,222,518 shares

   issued at September 30, 2017 and December 31, 2016, respectively; 37,524,638

   and 37,222,172 shares outstanding at September 30, 2017 and December 31, 2016,

   respectively

 

 

4

 

 

 

4

 

Treasury stock, at cost, 346 shares at September 30, 2017 and December 31, 2016

 

 

(17

)

 

 

(17

)

Additional paid-in capital

 

 

721,105

 

 

 

688,959

 

Accumulated deficit

 

 

(521,025

)

 

 

(320,327

)

Accumulated other comprehensive loss

 

 

(15

)

 

 

(102

)

Total stockholders’ equity

 

 

200,052

 

 

 

368,517

 

Total liabilities and stockholders’ equity

 

$

251,085

 

 

$

404,531

 

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

 

 

5


 

Sage Therapeutics, Inc. and Subsidiaries

Condensed Consolidated Statements of Operations and Comprehensive Loss

(in thousands, except share and per share data)

(Unaudited)

 

 

 

Three months ended September 30,

 

 

Nine months ended September 30,

 

 

 

2017

 

 

2016

 

 

2017

 

 

2016

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

58,286

 

 

$

29,075

 

 

$

159,386

 

 

$

78,752

 

General and administrative

 

 

16,087

 

 

 

8,989

 

 

 

43,320

 

 

 

25,033

 

Total operating expenses

 

 

74,373

 

 

 

38,064

 

 

 

202,706

 

 

 

103,785

 

Loss from operations

 

 

(74,373

)

 

 

(38,064

)

 

 

(202,706

)

 

 

(103,785

)

Interest income, net

 

 

677

 

 

 

275

 

 

 

2,056

 

 

 

717

 

Other expense, net

 

 

(23

)

 

 

(7

)

 

 

(48

)

 

 

(18

)

Net loss

 

$

(73,719

)

 

$

(37,796

)

 

$

(200,698

)

 

$

(103,086

)

Net loss per share—basic and diluted

 

$

(1.97

)

 

$

(1.15

)

 

$

(5.37

)

 

$

(3.20

)

Weighted average common shares outstanding—basic and

   diluted

 

 

37,470,912

 

 

 

32,975,897

 

 

 

37,367,802

 

 

 

32,218,204

 

Comprehensive loss:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Net loss

 

$

(73,719

)

 

$

(37,796

)

 

$

(200,698

)

 

$

(103,086

)

Other comprehensive items:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Unrealized gain (loss) on marketable securities

 

 

52

 

 

 

(74

)

 

 

87

 

 

 

(35

)

Total other comprehensive gain

 

 

52

 

 

 

(74

)

 

 

87

 

 

 

(35

)

Total comprehensive loss

 

$

(73,667

)

 

$

(37,870

)

 

$

(200,611

)

 

$

(103,121

)

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

 

 

6


 

Sage Therapeutics, Inc. and Subsidiaries

Condensed Consolidated Statements of Cash Flows

(in thousands)

(Unaudited)

 

 

 

Nine months ended September 30,

 

 

 

2017

 

 

2016

 

Cash flows from operating activities

 

 

 

 

 

 

 

 

Net loss

 

$

(200,698

)

 

$

(103,086

)

Adjustments to reconcile net loss to net cash used in operating activities:

 

 

 

 

 

 

 

 

Stock-based compensation expense

 

 

25,289

 

 

 

12,927

 

Premium on marketable securities

 

 

(49

)

 

 

(663

)

Amortization of premium on marketable securities

 

 

(57

)

 

 

87

 

Depreciation

 

 

396

 

 

 

199

 

Changes in operating assets and liabilities:

 

 

 

 

 

 

 

 

Prepaid expenses and other current assets

 

 

208

 

 

 

(1,680

)

Accounts payable

 

 

221

 

 

 

116

 

Accrued expenses and other liabilities

 

 

14,312

 

 

 

7,198

 

Net cash used in operating activities

 

 

(160,378

)

 

 

(84,902

)

Cash flows from investing activities

 

 

 

 

 

 

 

 

Proceeds from sales and maturities of marketable securities

 

 

214,436

 

 

 

7,999

 

Purchases of marketable securities

 

 

(93,816

)

 

 

(118,650

)

Purchases of property and equipment

 

 

(556

)

 

 

(901

)

Increase in restricted cash

 

 

(285

)

 

 

(525

)

Net cash provided by (used in) investing activities

 

 

119,779

 

 

 

(112,077

)

Cash flows from financing activities

 

 

 

 

 

 

 

 

Proceeds from stock option exercises and employee stock purchase plan issuances

 

 

6,998

 

 

 

728

 

Payments of offering costs

 

 

 

 

 

(599

)

Proceeds from public offerings of common stock, net of commissions and underwriting

   discounts

 

 

 

 

 

330,175

 

Net cash provided by financing activities

 

 

6,998

 

 

 

330,304

 

Net increase (decrease) in cash and cash equivalents

 

 

(33,601

)

 

 

133,325

 

Cash and cash equivalents at beginning of period

 

 

168,517

 

 

 

186,753

 

Cash and cash equivalents at end of period

 

$

134,916

 

 

$

320,078

 

Supplemental disclosure of non-cash financing activities

 

 

 

 

 

 

 

 

Purchases of property and equipment financed with landlord tenant incentive

 

$

353

 

 

$

 

Purchases of property and equipment included in accounts payable or accrued expenses

 

$

 

 

$

106

 

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

 

 

7


 

SAGE THERAPEUTICS, INC. AND SUBSIDIARIES

Notes to Condensed Consolidated Financial Statements

(Unaudited)

 

1. Nature of Operations

Sage Therapeutics, Inc. (“Sage” or the “Company”) is a clinical-stage biopharmaceutical company committed to developing and commercializing novel medicines to treat life-altering central nervous system, or CNS, disorders, where there are no approved therapies or existing therapies are inadequate.  The Company has a portfolio of product candidates with a current focus on modulating two critical CNS receptor systems, GABA and NMDA.  The GABA receptor family, which is recognized as the major inhibitory neurotransmitter in the CNS, mediates downstream neurologic and bodily function via activation of GABAA receptors.  The NMDA-type receptors of the glutamate receptor system are a major excitatory receptor system in the CNS.  Dysfunction in these systems is implicated in a broad range of CNS disorders.  The Company is targeting CNS indications where patient populations are easily identified, clinical endpoints are well-defined, and development pathways are feasible.

The Company was incorporated under the laws of the State of Delaware on April 16, 2010, and commenced operations on January 19, 2011 as Sterogen Biopharma, Inc. On September 13, 2011, the Company changed its name to Sage Therapeutics, Inc.

The Company is subject to risks and uncertainties common to companies in the biotech industry, including, but not limited to, the risks associated with developing product candidates at each stage of non-clinical and clinical development; the challenges associated with gaining regulatory approval of such product candidates; the risks associated with commercializing pharmaceutical products, if approved for marketing and sale; the potential for development by third parties of new technological innovations that may compete with the Company’s products; the dependence on key personnel; the challenges of protecting proprietary technology; the need to comply with government regulations; the high costs of drug development; and the uncertainty of being able to secure additional capital when needed to fund operations.

The Company has incurred losses and negative cash flows from operations since its inception. As of September 30, 2017, the Company had an accumulated deficit of $521.0 million. From its inception through September 30, 2017, the Company received net proceeds of $643.3 million from the sales of redeemable convertible preferred stock, the issuance of convertible notes, and the proceeds from its initial public offering (“IPO”) in July 2014 and follow-on underwritten public offerings in April 2015, January 2016 and September 2016. Until such time, if ever, as the Company can generate substantial product revenue, the Company expects to finance its cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements and other sources of funding.  If the Company is unable to raise additional funds through equity or debt financings when needed, the Company may be required to delay, limit, reduce or terminate product development or future commercialization efforts or grant rights to develop and market products or product candidates that the Company would otherwise prefer to develop and market itself.

Based on its current operating plans, the Company believes its cash, cash equivalents and marketable securities of $243.5 million as of September 30, 2017 will be sufficient to fund its anticipated level of operations and capital expenditures into the second quarter of 2018.

 

Under Accounting Standards Update, or ASU, 2014-15, Presentation of Financial Statements—Going Concern (Subtopic 205-40), or ASC 205-40, the Company has the responsibility to evaluate whether conditions and/or events raise substantial doubt about its ability to meet its future financial obligations as they become due within one year after the date that the financial statements are issued. As required by ASC 205-40, this evaluation shall initially not take into consideration the potential mitigating effects of plans that have not been fully implemented as of the date the financial statements are issued.  

 

The Company anticipates that its current capital resources will enable it to meet its anticipated operational expenses and capital expenditures into the second quarter of calendar year 2018. As of September 30, 2017, management has assessed the Company’s ability to continue as a going concern in accordance with the requirements of ASC 205-40.  Management has determined that the Company’s accumulated deficit, history of losses, and future expected losses meet the ASC 205-40 standard for raising substantial doubt about the Company’s ability to continue as a going concern within one year of the issuance date of these condensed consolidated financial statements. As of September 30, 2017, the Company had an accumulated deficit of $521.0 million and cash, cash equivalents and short-term investments on hand of $243.5 million. The Company is currently forecasting a significant increase in expenditures to support a potential filing of a New Drug Application, or NDA, for brexanolone in postpartum depression, or PPD, in the U.S. and future potential regulatory filings in the European Union, or EU, if the results of the Phase 3 clinical trials in PPD are positive and support such filings, continued preparations for a potential future commercial launch of brexanolone, if successfully developed and approved, and spending on other clinical programs in development. The Company’s current financial resources would not be sufficient to fund the Company’s currently forecasted operating plan for a one year period from the issuance of these condensed consolidated financial statements.  The Company has developed plans to mitigate this risk, which primarily consist of raising additional capital through a combination of equity or debt financings, and, depending on the availability and level of additional financings, potentially new collaborations and reducing cash expenditures.

8


 

 

The Company currently expects to seek additional funding. While the Company has raised capital in the past, the ability to raise capital in future periods is not considered probable, as defined under the accounting standards. As such, under the requirements of ASC 205-40, management may not consider the potential for future capital raises in their assessment of the Company’s ability to meet its obligations for the next 12 months.

 

If the Company is not able to secure adequate additional funding, the Company plans to make reductions in spending. In that event, the Company may have to delay, reduce the scope of, suspend or eliminate one or more research and development programs or reduce spending and hiring in connection with the Company’s commercialization efforts. The actions necessary to reduce spending under this plan at a level that mitigates the factors described above is not considered probable, as defined in the accounting standards; as such, under the requirements of ASC 205-40, the full extent to which management may extend the Company’s funds through these actions may not be considered in management’s assessment of the Company’s ability to continue as a going concern for the next 12 months as defined by ASC 205-40.

 

As a result, in accordance with the requirements of ASC 205-40, management has concluded that it is required to disclose that substantial doubt exists about the Company’s ability to continue as a going concern for one year from the date these financial statements are issued. While management has plans in place to mitigate these actions, they are not considered probable, as defined in the accounting standards, and a failure to raise the additional funding or to effectively implement cost reductions could harm the Company’s business, results of operations and future prospects.

 

The accompanying unaudited interim condensed consolidated financial statements of the Company have been prepared on a going-concern basis, which contemplates the realization of assets and the satisfaction of liabilities in the normal course of business. The accompanying unaudited interim condensed consolidated financial statements do not include any adjustments to reflect the possible future effects on the recoverability and classification of assets or the amounts and classification of liabilities that may result from uncertainty related to the ability to continue as a going concern.

 

 

2. Summary of Significant Accounting Policies

Basis of Presentation

The unaudited interim condensed consolidated financial statements of the Company included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (the “SEC”). Certain information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements as of and for the year ended December 31, 2016.

The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited consolidated financial statements. In the opinion of the Company’s management, the accompanying unaudited condensed consolidated financial statements contain all adjustments, consisting of only normal recurring adjustments, necessary for a fair statement of its financial position as of September 30, 2017, its results of operations and comprehensive loss for the three and nine months ended September 30, 2017 and 2016, and its cash flows for the nine months ended September 30, 2017 and 2016. The consolidated balance sheet at December 31, 2016 was derived from audited financial statements, but does not include all disclosures required by GAAP. The results for the three and nine months ended September 30, 2017 are not necessarily indicative of the results for the year ending December 31, 2017, or for any future period.

Principles of Consolidation

The unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries as disclosed in Note 2, Summary of Significant Accounting Policies, within the “Notes to Consolidated Financial Statements” accompanying its Annual Report on Form 10-K for the fiscal year ended December 31, 2016. Intercompany accounts and transactions have been eliminated.

Use of Estimates

The preparation of consolidated financial statements in conformity with GAAP requires management 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 consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates.

9


 

Research and Development

Research and development expenses are comprised of costs incurred in performing research and development activities, including salaries and benefits, overhead costs, depreciation, contract services and other related costs. Research and development costs are expensed to operations as the related obligation is incurred.

The Company has entered into various research and development contracts with research institutions and other companies both inside and outside of the U.S. These agreements are generally cancelable, and related payments are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates may be made in determining the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. The Company’s historical accrual estimates have not been materially different from the actual costs.

Stock-Based Compensation

The Company recognizes compensation expense for stock-based awards made to employees and nonemployee directors, including grants of stock options and restricted stock, based on the estimated fair value on the date of grant, over the requisite service period.

For stock-based options and restricted stock units with time-based vesting, issued to nonemployee consultants, the Company recognizes the fair value of the award as an expense over the period in which the related services are received. The fair value of the awards and measurement of related stock-based compensation is subject to periodic adjustments as the awards vest.

For awards that vest upon achievement of a performance condition, the Company recognizes compensation expense when achievement of the performance condition is deemed probable over the implicit service period.

The fair value of each option grant is estimated using the Black-Scholes option-pricing model. Through December 31, 2015, the Company lacked sufficient Company-specific historical and implied volatility information, and as a result, the Company used the volatility of a group of publicly-traded peer companies in the Black-Scholes calculations. Beginning in 2016, the Company estimated its expected volatility using a weighted average of the historical volatility of publicly-traded peer companies and the volatility of its common stock, and expects to continue to do so until such time as it has adequate historical data regarding the volatility of its traded stock price for the duration of the expected term. The expected term of the Company’s options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options, while the expected term of its options granted to consultants and nonemployees has been determined based on the contractual term of the options. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. The expected dividend yield is based on the fact that the Company never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future.

The Company also applies a forfeiture rate in order to calculate stock-based compensation expense. Expected forfeitures are based on the Company’s historical experience and management’s expectations of future forfeitures. To the extent actual forfeitures differ from the estimates, the difference will be recorded as a cumulative adjustment in the period in which the estimates are revised. The Company recognizes stock-based compensation expense for only the portion of awards that are expected to vest.

Marketable securities

Marketable securities consist of investments with original maturities greater than 90 days. The Company considers its investment portfolio of investments to be available-for-sale. Accordingly, these investments are recorded at fair value, which is based on quoted market prices. Unrealized gains and losses are reported as a component of accumulated other comprehensive items in stockholders’ equity. Realized gains and losses and declines in value judged to be other than temporary are included as a component of other expense, net, based on the specific identification method. When determining whether a decline in value is other than temporary, the Company considers various factors, including whether the Company has the intent to sell the security, and whether it is more likely than not that the Company will be required to sell the security prior to recovery of its amortized cost basis.  No declines in value were deemed to be other than temporary during the three and nine months ended September 30, 2017.

10


 

Fair Value Measurements

Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. Financial assets and liabilities carried at fair value are classified and disclosed in one of the following three categories:

 

Level 1

 

 

Quoted market prices in active markets for identical assets or liabilities.

 

 

 

 

 

Level 2

 

 

Observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities.

 

 

 

 

 

Level 3

 

 

Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities.

 

The Company’s cash equivalents and marketable securities at September 30, 2017 and December 31, 2016 were carried at fair value, determined according to the fair value hierarchy.

The carrying amounts reflected in the unaudited consolidated balance sheets for accounts payable and accrued expenses approximate their fair values due to their short-term maturities at September 30, 2017 and December 31, 2016, respectively.

Recently Issued Accounting Pronouncements

In May 2014, the Financial Accounting Standards Board (the “FASB”) issued ASU No. 2014-09, Revenue from Contracts with Customers (Topic 606), which supersedes all existing revenue recognition requirements, including most industry-specific guidance. The new standard requires a company to recognize revenue when it transfers goods or services to customers in an amount that reflects the consideration that the company expects to receive for those goods or services. The FASB has continued to issue accounting standards updates to clarify and provide implementation guidance related to Revenue from Contracts with Customers, including ASU 2016-08, Revenue from Contract with Customers: Principal versus Agent Considerations, ASU 2016-10, Revenue from Contracts with Customers: Identifying Performance Obligations and Licensing, and ASU 2016-12, Revenue from Contracts with Customers: Narrow-Scope Improvements and Practical Expedients. These amendments address a number of areas, including the entity’s identification of its performance obligations in a contract, collectability, non-cash consideration, presentation of sales tax and an entity’s evaluation of the nature of its promise to grant a license of intellectual property and whether or not that revenue is recognized over time or at a point in time. These new standards will be effective for the Company beginning January 1, 2018. The Company had the option to early adopt the standard for the year ending December 31, 2017. The Company early adopted the standard as of January 1, 2017, although there is no impact of this new guidance on its consolidated financial statements as it does not currently have any revenue generating arrangements.

In February 2016, the FASB issued ASU No. 2016-02, Leases, which will replace the existing guidance in ASC 840, “Leases.” The updated standard aims to increase transparency and comparability among organizations by requiring lessees to recognize leased assets and leased liabilities on the consolidated balance sheets and requiring disclosure of key information about leasing arrangements. The standard will be effective on January 1, 2019, with early adoption permitted. The Company is in the process of evaluating the impact that this new guidance will have on its consolidated financial statements.

 

In March 2016, the FASB issued ASU No. 2016-09, Improvements to Employee Share-Based Payment Accounting, which intends to simplify several aspects of accounting for share-based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities, an option to recognize gross stock compensation expense with actual forfeitures recognized as they occur, as well as certain classifications on the statement of cash flows.  The standard is effective for annual periods that began after December 15, 2016 and for interim periods within those fiscal years. The Company adopted ASU 2016-09 on the required effective date of January 1, 2017. The Company elected to maintain its existing policy to estimate forfeitures when determining periodic stock-based compensation expense. The adoption of the other provisions of ASU 2016-09 had no impact on the Company’s financial position, results of operations or cash flows.

In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments, which introduces a new methodology for accounting for credit losses on financial instruments, including available-for-sale debt securities. The guidance establishes a new "expected loss model" that requires entities to estimate current expected credit losses on financial instruments by using all practical and relevant information. Any expected credit losses are to be reflected as allowances rather than reductions in the amortized cost of available-for-sale debt securities. Early adoption is permitted for annual periods beginning after December 15, 2018, and interim periods therein. The Company is in the process of evaluating the impact that this new guidance will have on its consolidated financial statements.

11


 

In August 2016, the FASB issued ASU No. 2016-15, Statement of Cash Flows (Topic 230): Classification of Certain Cash Receipts and Cash Payments. The standard reduces the diversity in practice in how certain cash receipts and cash payments are presented and classified in the statement of cash flows. The standard will be effective on January 1, 2018. The Company is in the process of evaluating the impact that this new guidance will have on its consolidated financial statements.

In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash that changes the presentation of restricted cash and cash equivalents in the statement of cash flows. Restricted cash and restricted cash equivalents will be included with cash and cash equivalents when reconciling the beginning-of-period and end-of-period total amounts shown on the statement of cash flows. This standard is effective for the Company in the fiscal year beginning January 1, 2018, but early adoption is permissible. After adopting the standard, the amounts of restricted cash shown on the consolidated balance sheets of the Company would be included in cash and cash equivalents in the statement of cash flows. The Company is in the process of evaluating the impact that this new guidance will have on its consolidated financial statements.  

In May 2017, the FASB issued ASU No. 2017-09, Compensation—Stock Compensation (Topic 718) — Scope of Modification Accounting, which applies to entities that change the terms or conditions of a share-based payment award. The amendments in this standard include guidance on determining whether changes to the terms and conditions of share-based payment awards require an entity to apply modification accounting under Topic 718 unless all of the following conditions are met: (1) the fair value of the modified award is the same as the fair value of the original award immediately before the original award is modified, and if the modification does not affect any of the inputs to the valuation technique that the entity uses to value the award, then the entity is not required to estimate the value immediately before and after the modification; (2) the vesting conditions of the modified award are the same as the vesting conditions of the original award immediately before the original award is modified; and (3) the classification of the modified award as an equity instrument or a liability instrument is the same as the classification of the original award immediately before the original award is modified. The amendments are effective for annual periods, and interim periods within those annual periods, beginning after December 15, 2017 and should be applied prospectively to an award modified on or after the adoption date. The Company is in the process of evaluating the impact that this new guidance will have on its consolidated financial statements.

Other accounting standards that have been issued or proposed by the FASB or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on the Company’s consolidated financial statements upon adoption.

 

 

3. Fair Value Measurements

The Company’s cash equivalents are classified within Level 1 of the fair value hierarchy.  The Company’s investments in marketable securities are classified within Level 2 of the fair value hierarchy.

The fair values of the Company’s marketable securities are based on prices obtained from independent pricing sources.  Consistent with the fair value hierarchy described above, securities with validated quotes from pricing services are reflected within Level 2, as they are primarily based on observable pricing for similar assets or other market observable inputs.  Typical inputs used by these pricing services include, but are not limited to, reported trades, benchmark yields, issuer spreads, bids, offers or estimates of cash flow, prepayment spreads and default rates.

12


 

The following tables summarize the Company’s money market funds and marketable securities as of September 30, 2017 and December 31, 2016:

 

 

 

September 30, 2017

 

 

 

Total

 

 

Quoted

Prices in

Active

Markets

(Level 1)

 

 

Significant

Other

Observable

Inputs

(Level 2)

 

 

Significant

Unobservable

Inputs

(Level 3)

 

 

 

(in thousands)

 

Cash equivalents:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

134,916

 

 

$

134,916

 

 

$

 

 

$

 

Total cash equivalents

 

 

134,916

 

 

 

134,916

 

 

 

 

 

 

 

Marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

U.S. government securities

 

 

37,992

 

 

 

 

 

 

37,992

 

 

 

 

U.S. corporate bonds

 

 

16,317

 

 

 

 

 

 

16,317

 

 

 

 

U.S. commercial paper

 

 

24,710

 

 

 

 

 

 

24,710

 

 

 

 

International commercial paper

 

 

29,516

 

 

 

 

 

 

29,516

 

 

 

 

Total marketable securities

 

 

108,535

 

 

 

 

 

 

108,535

 

 

 

 

Total cash equivalents and marketable securities

 

$

243,451

 

 

$

134,916

 

 

$

108,535

 

 

$

 

 

 

 

December 31, 2016

 

 

 

Total

 

 

Quoted

Prices in

Active

Markets

(Level 1)

 

 

Significant

Other

Observable

Inputs

(Level 2)

 

 

Significant

Unobservable

Inputs

(Level 3)

 

 

 

(in thousands)

 

Cash equivalents:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

168,517

 

 

$

168,517

 

 

$

 

 

$

 

Total cash equivalents

 

 

168,517

 

 

 

168,517

 

 

 

 

 

 

 

Marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

U.S. government securities

 

 

100,031

 

 

 

 

 

 

100,031

 

 

 

 

U.S. corporate bonds

 

 

58,452

 

 

 

 

 

 

58,452

 

 

 

 

International corporate bonds

 

 

24,190

 

 

 

 

 

 

24,190

 

 

 

 

U.S. commercial paper

 

 

30,351

 

 

 

 

 

 

30,351

 

 

 

 

International commercial paper

 

 

15,938

 

 

 

 

 

 

15,938

 

 

 

 

Total marketable securities

 

 

228,962

 

 

 

 

 

 

228,962

 

 

 

 

Total cash equivalents and marketable securities

 

$

397,479

 

 

$

168,517

 

 

$

228,962

 

 

$

 

 

During the nine months ended September 30, 2017 and 2016, there were no transfers among the Level 1, Level 2 and Level 3 categories.

Marketable Securities

The following tables summarize the gross unrealized gains and losses of the Company’s marketable securities as of September 30, 2017 and December 31, 2016:

 

 

 

September 30, 2017

 

 

 

Amortized Cost

 

 

Gross Unrealized

Gains

 

 

Gross Unrealized

Losses

 

 

Fair Value

 

 

 

(in thousands)

 

Assets:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

U.S. government securities

 

$

38,003

 

 

$

 

 

$

(11

)

 

$

37,992

 

U.S. corporate bonds

 

 

16,321

 

 

 

1

 

 

 

(5

)

 

 

16,317

 

U.S. commercial paper

 

 

24,710

 

 

 

 

 

 

 

 

 

24,710

 

International commercial paper

 

 

29,516

 

 

 

 

 

 

 

 

 

29,516

 

 

 

$

108,550

 

 

$

1

 

 

$

(16

)

 

$

108,535

 

13


 

 

 

 

December 31, 2016

 

 

 

Amortized Cost

 

 

Gross Unrealized

Gains

 

 

Gross Unrealized

Losses

 

 

Fair Value

 

 

 

(in thousands)

 

Assets:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

U.S. government securities

 

$

100,055

 

 

$

2

 

 

$

(26

)

 

$

100,031

 

U.S. corporate bonds

 

 

58,508

 

 

 

 

 

 

(56

)

 

 

58,452

 

International corporate bonds

 

 

24,212

 

 

 

 

 

 

(22

)

 

 

24,190

 

U.S. commercial paper

 

 

30,351

 

 

 

 

 

 

 

 

 

30,351

 

International commercial paper

 

 

15,938

 

 

 

 

 

 

 

 

 

15,938

 

 

 

$

229,064

 

 

$

2

 

 

$

(104

)

 

$

228,962

 

 

As of September 30, 2017, all marketable securities held by the Company had remaining contractual maturities of one year or less.

 

There have been no impairments of the Company’s assets measured and carried at fair value during the three and nine months ended September 30, 2017 and the year ended December 31, 2016.

 

 

4. Accrued Expenses

Accrued expenses consist of the following:

 

 

 

September 30, 2017

 

 

December 31, 2016

 

 

 

(in thousands)

 

Development costs

 

$

27,326

 

 

$

14,541

 

Employee-related expenses

 

 

6,299

 

 

 

5,948

 

Professional services

 

 

3,179

 

 

 

1,751

 

Other accrued expenses

 

 

3

 

 

 

112

 

 

 

$

36,807

 

 

$

22,352

 

 

 

5. Commitments and contingencies

Operating Leases

In May 2017, the Company entered into the Sixth Amendment to the Lease with ARE-MA Region No. 38 (“Sixth Amendment”) to increase the amount of square feet of office space that the Company leases in a multi-tenant building located at 215 First Street, Cambridge, Massachusetts.  Prior to entering the Sixth Amendment, the Company rented 22,067 square feet of office space in this building under an operating lease that was scheduled to expire in February 2022. The Sixth Amendment increases the amount of leased space at this location by 32,876 square feet, with the additional space consisting of (i) 8,200 square feet beginning on August 15, 2017, and (ii) 24,676 square feet beginning on or around January 1, 2018.  The term for this additional space will expire no later than August 15, 2024.  Additionally, the term of the existing lease has been extended from March 1, 2022 until the expiration date of the Sixth Amendment on August 15, 2024.

In May 2016, the Company entered into an operating lease with Jamestown Premier 245 First, LLC under which, beginning in September 2016, the Company rents 19,805 square feet of office space in a multi-tenant building located at 245 First St., Cambridge, Massachusetts.  The lease has been assigned by the lessor to CLPF-Cambridge Science Center, LLC. The lease is accounted for as an operating lease and is scheduled to expire in February 2022.

 

 

14


 

Future minimum lease payments under non-cancelable operating leases are as follows at September 30, 2017:

 

Years Ending December 31,

 

(in thousands)

 

2017

 

$

782

 

2018

 

 

4,444

 

2019

 

 

4,537

 

2020

 

 

4,632

 

2021

 

 

4,728

 

Thereafter

 

 

8,935

 

 

 

$

28,058

 

 

CyDex License Agreement

In September 2015, the Company and CyDex Pharmaceuticals, Inc. (“CyDex”) amended and restated their existing commercial license agreement. Under the terms of the commercial license agreement as amended and restated, CyDex has granted to the Company an exclusive license to CyDex’s Captisol drug formulation technology and related intellectual property for the manufacture of pharmaceutical products incorporating the Company’s compounds known as brexanolone (SAGE-547) and SAGE-689, and the development and commercialization of the resulting products in the treatment, prevention or diagnosis of any disease or symptom in humans or animals other than (i) the ocular treatment of any disease or condition with a formulation, including a hormone; (ii) topical ocular treatment of inflammatory conditions; (iii) treatment and prophylaxis of fungal infections in humans; and (iv) any ocular treatment for retinal degeneration. As consideration for the inclusion of SAGE-689 in the license granted by CyDex, the Company paid to CyDex $0.1 million, which was recorded as research and development expense for the year ended December 31, 2015 in connection with the execution of the amended and restated license agreement.

The Company is obligated to make milestone payments under the amended and restated license agreement with CyDex based on the achievement of clinical development and regulatory milestones in the amount of up to $0.8 million in clinical milestones and up to $3.8 million in regulatory milestones for each of the first two fields with respect to brexanolone; up to $1.3 million in clinical milestones and up to $8.5 million in regulatory milestones for each of the third and fourth fields with respect to brexanolone; and up to $0.8 million in clinical milestones and up to $1.8 million in regulatory milestones for one field with respect to SAGE-689.

In August 2016, an additional clinical development milestone was met for the brexanolone program under the license agreement with CyDex, and accordingly, the Company recorded research and development expense for the three months ended September 30, 2016 of $0.3 million. 

In December 2016, an additional clinical development milestone was met for the brexanolone program under the license agreement with CyDex, and accordingly, the Company recorded research and development expense for the three months ended December 31, 2016 of $0.5 million. 

For the three and nine months ended September 30, 2017, the Company did not record any expense or make any milestone payments under the license agreement with CyDex.

University of California License Agreements

In October 2013, the Company entered into a non-exclusive license agreement with The Regents of the University of California under which the Company was granted a non-exclusive license to certain clinical data and clinical material for use in the development and commercialization of biopharmaceutical products in the licensed field, including status epilepticus and postpartum depression. In May 2014, the license agreement was amended to add the treatment of essential tremor to the licensed field of use, materials and milestone fee provisions of the agreement. As of December 31, 2015, the Company paid to The Regents of the University of California clinical development milestones of $0.1 million and will be required to pay royalties of less than 1% on net sales for a period of fifteen years following the sale of the first product developed using the data and materials. The license will terminate on the earlier to occur of (i) 27 years after the effective date or (ii) 15 years after the last-derived product is first commercially sold.

In June 2015, the Company entered into an exclusive license agreement with The Regents of the University of California whereby the Company was granted an exclusive license to certain patent rights related to the use of allopregnanolone to treat various diseases. In exchange for such license, the Company paid an upfront payment of $50,000 and will make payments of $15,000 for annual maintenance fees until the calendar year following the first sale, if any, of a licensed product. The Company is obligated to make milestone payments following the achievement of specified regulatory and sales milestones of up to $0.7 million and $2.0 million in the aggregate, respectively, of which none have been paid to date. Following the first sale, if any, of a licensed product, the Company is obligated to pay royalties at a low single digit percentage of net sales, if any, of licensed products, subject to specified

15


 

minimum annual royalty amounts. Unless terminated by operation of law or by acts of the parties under the terms of the agreement, the license agreement will terminate when the last-to-expire patents or last-to-be abandoned patent applications expire, whichever is later.

For the year ended December 31, 2016 and for the three and nine months ended September 30, 2017, the Company did not record any expense or make any milestone or royalty payments under either license agreement with The Regents of the University of California.

Consulting Agreement

In January 2014, the Company entered into a consulting agreement with a non-employee advisor whereby the Company is obligated to make cash payments of up to $2.0 million and to issue up to 126,984 shares of common stock upon attainment of certain clinical development and regulatory milestones, of which $0.5 million has been paid and 39,681 shares of common stock have been issued to date.

For the year ended December 31, 2016 and the three and nine months ended September 30, 2017, the Company did not record any expense or make any milestone payments under the consulting agreement with the non-employee advisor.

 

 

6. Sale of Equity Securities

On January 12, 2016, the Company completed the sale of 3,157,894 shares of its common stock at a price to the public of $47.50 per share, resulting in net proceeds to the Company of $140.4 million after deducting underwriting discounts and commissions and offering expenses paid by the Company.

On September 14, 2016, the Company completed the sale of 5,062,892 shares of its common stock at a price to the public of $39.75 per share, resulting in net proceeds to the Company of $189.2 million after deducting underwriting discounts and commissions paid by the Company.

 

 

7. Stock-Based Compensation

Restricted Stock Units

During the nine months ended September 30, 2017, the Company granted 32,500 restricted stock units to employees of the Company. The Company did not grant restricted stock units prior to January 1, 2017.  During the three and nine months ended September 30, 2017, the Company recorded $0.2 million and $0.4 million, respectively, of stock-based compensation expense related to its restricted stock units. These restricted stock units vest ratably over two years, with cliff vesting of 50% at both the one-year and two-year anniversary of the grant, which will be in February 2018 and February 2019, respectively. 

Stock Option Plans

On December 15, 2016, the Board of Directors of the Company approved the 2016 Inducement Equity Plan (the “2016 Stock Option Plan”). The 2016 Stock Option Plan provides for the grant of equity awards to individuals who have not previously been an employee or a non-employee director of the Company to induce them to accept employment and to provide them with a proprietary interest in the Company.

As of September 30, 2017, under the 2014 Stock Option and Incentive Plan (the “2014 Stock Option Plan”) and the 2016 Stock Option Plan, an aggregate of 8,094,060 shares were reserved and 2,125,783 shares were available for future issuance.

The 2014 Stock Option Plan provides for an annual increase, to be added on the first day of each year, of up to 4% of the Company’s issued and outstanding shares of common stock on the last day of the prior year. On January 1, 2017, 1,488,886 shares of common stock, representing 4% of the Company’s issued and outstanding shares of common stock as of December 31, 2016, were added to the 2014 Stock Option Plan.

During the nine months ended September 30, 2017 and 2016, the Company granted 449,208 and 74,039 options, respectively, to employees to purchase shares of common stock that contain performance-based vesting criteria, primarily related to the achievement of certain clinical and regulatory development milestones related to product candidates. Recognition of stock-based compensation expense associated with these performance-based stock options commences when the performance condition is considered probable of achievement, using management’s best estimates.

16


 

During the nine months ended September 30, 2017 and 2016, the achievement of the remaining milestones that are the criteria for vesting of performance-based stock options was considered not probable, nor met, and therefore no expense has been recognized related to these awards for the nine months ended September 30, 2017 and 2016.

Stock-based compensation expense for stock options and restricted stock units recognized during the three and nine months ended September 30, 2017 and 2016 was as follows:

 

 

 

Three months ended September 30,

 

 

Nine months ended September 30,

 

 

 

2017

 

 

2016

 

 

2017

 

 

2016

 

 

 

(in thousands)

 

 

 

 

 

 

 

 

 

Research and development

 

$

5,399

 

 

$

2,530

 

 

$

14,239

 

 

$

6,179

 

General and administrative

 

 

4,332

 

 

 

2,218

 

 

 

11,050

 

 

 

6,748

 

 

 

$

9,731

 

 

$

4,748

 

 

$

25,289

 

 

$

12,927

 

 

The weighted average grant date fair value per share relating to outstanding stock options granted under the Company’s stock option plans during the nine months ended September 30, 2017 and 2016 was $40.43 and $23.32, respectively.

The table below summarizes activity related to stock options:

 

 

 

Shares

 

 

Weighted

Average Exercise

Price

 

 

Weighted Average

Remaining Life

(in years)

 

 

Aggregate

Intrinsic Value

(in thousands)

 

Outstanding as of December 31, 2016

 

 

4,231,807

 

 

$

29.99

 

 

 

8.24

 

 

$

92,843

 

Granted

 

 

2,169,250

 

 

 

58.33

 

 

 

 

 

 

 

 

 

Exercised

 

 

(286,748

)

 

 

22.20

 

 

 

 

 

 

 

 

 

Forfeited

 

 

(176,132

)

 

 

42.57

 

 

 

 

 

 

 

 

 

Outstanding as of September 30, 2017

 

 

5,938,177

 

 

$

40.35

 

 

 

8.19

 

 

$

140,390

 

Exercisable as of September 30, 2017

 

 

2,301,343

 

 

$

25.30

 

 

 

7.10

 

 

$

85,819

 

 

At September 30, 2017, the Company had unrecognized stock-based compensation expense related to its unvested service-based stock option awards of $90.2 million, which is expected to be recognized over the remaining weighted average vesting period of 2.68 years. The total fair value of options vested for the nine months ended September 30, 2017 and 2016 was $18.0 million and $14.9 million, respectively.

At September 30, 2017, 678,252 performance-based stock options were both outstanding and unvested, and the total unrecognized stock-based compensation expense related to those awards was $13.7 million.

 

 

8. Net Loss Per Share

Basic and diluted net loss per share was calculated as follows for the three and nine months ended September 30, 2017 and 2016:

 

 

 

Three months ended September 30,

 

 

Nine months ended September 30,

 

 

 

2017

 

 

2016

 

 

2017

 

 

2016

 

Basic net loss per share:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Numerator:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Net loss (in thousands)

 

$

(73,719

)

 

$

(37,796

)

 

$

(200,698

)

 

$

(103,086

)

Denominator:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Weighted average common stock

   outstanding—basic

 

 

37,470,912

 

 

 

32,975,897

 

 

 

37,367,802

 

 

 

32,218,204

 

Dilutive effect of shares of common stock

   equivalents resulting from common stock

   options and restricted stock units

 

 

 

 

 

 

 

 

 

 

 

 

Weighted average common stock

   outstanding—diluted

 

 

37,470,912

 

 

 

32,975,897

 

 

 

37,367,802

 

 

 

32,218,204

 

Net loss per share—basic and diluted

 

$

(1.97

)

 

$

(1.15

)

 

$

(5.37

)

 

$

(3.20

)

 

17


 

The following common stock equivalents outstanding as of September 30, 2017 and 2016 were excluded from the computation of diluted net loss per share for the periods presented because including them would have been anti-dilutive:

 

 

 

Three months ended September 30,

 

 

Nine months ended September 30,

 

 

 

2017

 

 

2016

 

 

2017

 

 

2016

 

Stock options

 

 

5,259,925

 

 

 

3,631,465

 

 

 

5,259,925

 

 

 

3,631,465

 

Restricted stock units

 

 

30,100

 

 

 

 

 

 

30,100

 

 

 

 

Employee stock purchase plan

 

 

6,500

 

 

 

4,368

 

 

 

6,500

 

 

 

4,368

 

Restricted stock awards

 

 

 

 

 

2,313

 

 

 

 

 

 

2,313

 

 

 

 

5,296,525

 

 

 

3,638,146

 

 

 

5,296,525

 

 

 

3,638,146

 

 

 

18


 

Item 2.

Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q, or Quarterly Report, and our Annual Report on Form 10-K for the year ended December 31, 2016, or Annual Report, and the audited financial information and the notes thereto.

We caution you that forward-looking statements are not guarantees of future performance, and that our actual results of operations, financial condition and liquidity, and the developments in our business and the industry in which we operate, may differ materially from the results discussed or projected in the forward-looking statements contained in this Quarterly Report. In addition, even if our results of operations, financial condition and liquidity, and the developments in our business and the industry in which we operate are consistent with the forward-looking statements contained in this Quarterly Report, they may not be predictive of results or developments in future periods. 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.

The following information and any forward-looking statements should be considered in light of factors discussed elsewhere in this Quarterly Report, including those risks identified under Part II, Item 1A, Risk Factors, and in the Annual Report.

We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any forward-looking statements to reflect any change in our expectations or in events, conditions or circumstances under which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

Overview

We are a clinical-stage biopharmaceutical company committed to developing and commercializing novel medicines to treat life-altering central nervous system, or CNS, disorders, where there are no approved therapies or existing therapies are inadequate. We have a portfolio of product candidates with a current focus on modulating two critical CNS receptor systems, GABA and NMDA.  The GABA receptor family, which is recognized as the major inhibitory neurotransmitter in the CNS, mediates downstream neurologic and bodily function via activation of GABAA receptors.  The NMDA-type receptors of the glutamate receptor system are a major excitatory receptor system in the CNS.  Dysfunction in these systems is implicated in a broad range of CNS disorders.  We are targeting CNS indications where patient populations are easily identified, clinical endpoints are well-defined, and development pathways are feasible.

The following table summarizes the status of our development programs as of the date of this Quarterly Report.

 

 

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Our lead product candidate, brexanolone (USAN) for intravenous, or IV, use, is a proprietary formulation of allopregnanolone, a naturally occurring neuroactive steroid that acts as a positive allosteric modulator of GABAA receptors, including both synaptic and extrasynaptic populations. We are currently developing brexanolone as a potential treatment for postpartum depression, or PPD.  PPD is a distinct and readily identified depressive disorder that is a biological complication of childbirth, affecting a subset of women typically commencing in the third trimester of pregnancy or within four weeks after giving birth.  We anticipate announcing top-line data from the Phase 3 clinical program, known as the Hummingbird Study, in the fourth quarter of 2017.  The Hummingbird Study is comprised of two placebo-controlled Phase 3 clinical trials of brexanolone, one in severe PPD and one in moderate PPD.  We have received Breakthrough Therapy designation from the U.S. Food and Drug Administration, or FDA, for brexanolone as a potential treatment for PPD. Based on input we received from the FDA during a Breakthrough Therapy meeting, we believe that, if successful, the results of the Phase 3 clinical program, together with the results of prior clinical studies of brexanolone in PPD and non-clinical studies, will be sufficient to support the submission of a New Drug Application, or NDA, to the FDA seeking approval for brexanolone in PPD in the U.S.   We have also received PRIority MEdicines (PRIME) designation from the European Medicines Agency, or EMA, for brexanolone in the treatment of PPD in the EU.  Incorporating scientific advice we recently received from the EMA, we believe our current Phase 3 clinical program in PPD, if successful, will be sufficient to support submission of a marketing authorization application, or MAA, to the EMA seeking approval of brexanolone for PPD in the EU.  We anticipate that the outcome of the ongoing Phase 3 clinical trials in PPD, if supportive of an MAA filing, will inform any future regulatory discussions and potential post-marketing clinical development obligations.

In the third quarter of 2017, we announced results of a Phase 3 clinical trial of brexanolone in super-refractory status epilepticus, or SRSE.  The trial did not meet the primary endpoint of the trial comparing success in weaning of third-line agents and resolution of status epilepticus in SRSE patients treated with brexanolone vs. placebo when added to standard-of-care.  We do not currently plan to pursue brexanolone as a treatment for SRSE.

Our most advanced next-generation product candidate is SAGE-217, a novel neuroactive steroid that, like brexanolone, is a positive allosteric modulator of GABAA receptors, targeting both synaptic and extrasynaptic GABAA receptors. We are currently developing SAGE-217 as a potential treatment for two mood disorders, major depressive disorder, or MDD, and PPD, and two movement disorders, Parkinson’s disease and essential tremor.  

We have completed dosing in our first blinded, placebo-controlled Phase 2 clinical trial of SAGE-217 in MDD, and expect to report top-line results from this trial in the fourth quarter of 2017.  

In November 2017, we reported results from an open-label exploratory Phase 2 clinical trial evaluating SAGE-217 as an adjunctive treatment to anti-Parkinsonian agents in 14 patients with tremor-predominant Parkinson’s disease.  In the clinical trial, SAGE-217 improved tremor symptoms, as assessed by the MDS-UPDRS - Part II/III tremor score, by a mean change of 7.7 points (40.0%) by Day 8 from a mean baseline of 19.1 points, which was the primary efficacy endpoint in the trial.  The most common adverse events observed in the trial were dizziness, sedation and somnolence, each occurring in two patients. We believe the results of this open-label Phase 2 trial will provide guidance on methodology, dosing, and design for potential future placebo-controlled Phase 2 clinical trial in Parkinson’s disease.  

We are currently conducting Part C of an exploratory Phase 2 clinical trial of SAGE-217 in essential tremor.  Part C, an open-label trial, was initiated in conjunction with changes in study design to enable us to study higher doses and longer duration of dosing.  As a result of the changes, enrollment in Parts A and B was discontinued prior to completion. In November 2017, we announced results of Part A of the Phase 2 trial generated prior to discontinuation of enrollment.  Part A was an open-label trial with morning dosing of SAGE-217 for 7 days, enrolling 16 patients diagnosed with essential tremor, defined as visible and persistent bilateral postural tremor and kinetic tremor, involving hands and forearms, with a duration greater than five years prior to screening. In Part A, SAGE-217 improved tremor symptoms, as assessed by the TETRAS upper limb combined kinetic score, by at least 30% on Day 7 in 8/12 patients (67%) who received SAGE-217 oral capsule in the study for the entire 7 days, which was the pre-established success criteria for moving to the next part of the study.  Administration of SAGE-217 in the morning was generally well-tolerated. The most common adverse events were somnolence, dizziness, and sedation. There were no serious adverse events reported in the 14 patients receiving SAGE-217 oral capsule. There was one serious adverse event reported as confusion in one of the two patients who received SAGE-217 oral solution.  Part B was discontinued prior to enrollment of a sufficient number of patients to generate meaningful data.  We anticipate reporting top-line results from Part C of the Phase 2 clinical trial of SAGE-217 in essential tremor in the fourth quarter of 2017.  

We also anticipate completing enrollment of a blinded, placebo-controlled Phase 2 clinical trial of SAGE-217 in PPD in the fourth quarter of 2017, and expect to report top-line results from this trial in the first quarter of 2018.  

We have a portfolio of other novel compounds that target GABAA receptors, including SAGE-324 and SAGE-689, which are at earlier stages of development with a focus on both acute and chronic CNS disorders.

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Our second area of focus is the development of novel compounds that target the NMDA receptor. The first product candidate selected for development from this program is SAGE-718, an oxysterol-based positive allosteric modulator of the NMDA receptor.  Our initial areas of focus for development of SAGE-718 is expected to be cerebrosterol deficit disorders, Anti-NMDA Receptor Encephalitis, and other indications involving NMDA receptor hypofunction. We believe measuring levels of anti-NMDA receptor antibodies or decreased levels of cerebrosterol, a naturally occurring oxysterol, may represent a biomarker to identify, for future study, broader patient populations characterized by cognitive dysfunction and neuropsychiatric symptoms resulting from NMDA receptor dysfunction or hypofunction. Examples of these potential areas for future evaluation include certain types, aspects or subpopulations of a number of diseases such as depression, Alzheimer’s disease, attention deficit hyperactivity disorder, schizophrenia, Huntington’s disease, and neuropathic pain. We commenced the Phase 1 clinical program for SAGE-718 in the second quarter of 2017.  In November 2017, we reported results from a Phase 1 single ascending dose (SAD) trial of SAGE-718 in healthy volunteers. The primary objectives of the study were to assess the safety, tolerability, and pharmacokinetics of SAGE-718.  In the single ascending dose trial, SAGE-718 was administered as an oral solution in four double-blind placebo-controlled cohorts (randomized 6:2) enrolling a total of 32 healthy volunteers.  SAGE-718 was well-tolerated in the trial with no serious adverse events reported.  The pharmacokinetics of SAGE-718 observed in the trial were highly predictable with low variability.  Based on these results, we plan to initiate a Phase 1 multiple ascending dose trial along with further dose finding studies subject to further regulatory agreement on proceeding with additional dose levels. We are also investigating the effects of SAGE-718 on pharmacodynamic biomarkers.

We expect to continue our focus on allosteric modulation of the GABAA and NMDA receptor systems in the brain. The GABAA and NMDA receptor systems are broadly accepted as impacting many psychiatric and neurological disorders, spanning disorders of mood, seizure, cognition, anxiety, sleep, pain, epilepsy, and movement disorders, among others. We believe that we will have the opportunity to develop molecules from our internal portfolio with the goal of addressing a number of these disorders in the future. Our ability to identify and develop such novel CNS therapies is enabled by our proprietary chemistry platform that is centered, as a starting point, on knowledge of the chemical scaffolds of certain endogenous neuroactive steroids. We believe our knowledge of the chemistry and activity of allosteric modulators allows us to efficiently design molecules with different characteristics.  This diversity enables us to regulate important properties such as half-life, brain penetration and receptor pharmacology, and to select for development product candidates that may have the potential for better selectivity, increased tolerability, and fewer off-target side effects than either current CNS therapies or previous therapies which have failed in development.  

We have not generated any revenue to date. We have incurred net losses in each year since our inception, and we have an accumulated deficit of $521.0 million as of September 30, 2017. Our net losses were $200.7 million for the nine months ended September 30, 2017 and $159.0 million for the year ended December 31, 2016. These losses have resulted principally from costs incurred in connection with research and development activities and general and administrative costs associated with our operations. We expect to incur significant expenses and increasing operating losses for the foreseeable future.

We expect that our expenses will increase substantially in connection with our ongoing activities, as we:

 

advance regulatory activities focused on potential filing of an NDA for brexanolone in PPD in the U.S., and a future potential regulatory filing in the EU, if the results of our Phase 3 clinical trials in PPD are positive and support such filings;

 

initiate an open-label follow-on study of brexanolone in adult women with PPD and a placebo-controlled clinical trial of brexanolone in adolescent females with PPD, if the results of the Phase 3 clinical trials of brexanolone in PPD are positive;

 

advance SAGE-217 further in development, potentially in one or more of MDD, essential tremor, Parkinson’s disease, and PPD, depending on the outcome of the ongoing trials and strategic assessments;  

 

continue to advance SAGE-718, our early-stage novel allosteric modulator for NMDA, including continuing the ongoing Phase 1 clinical program;

 

continue non-clinical studies of SAGE-324 with a potential focus on orphan epilepsies and indications involving GABA hypofunction;

 

continue our research and development efforts to evaluate the potential for our product candidates in the treatment of additional indications or in new formulations, and to identify new drug candidates in the treatment of CNS disorders;

 

seek regulatory approvals for any product candidates that successfully complete clinical development;

 

continue preparations for a potential future commercial launch of brexanolone, if our development efforts are successful;

 

complete validation work and other supply chain activities related to brexanolone to be ready for commercial supply if our development efforts are successful; refine and scale-up the manufacturing process for SAGE-217 to be ready for late stage clinical trials if we are able to proceed to that stage; improve the manufacturing process for our other product candidates; and manufacture clinical supplies as development progresses;

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add personnel, including personnel to support our product development and future commercialization efforts and potential expansion of EU activities, and incur increases in stock compensation expense related to existing and new personnel with respect to both service-based and performance-based awards;

 

add operational, financial and management information systems; and

 

maintain, leverage and expand our intellectual property portfolio.

As a result, we will need additional financing in the future to support our continuing operations. Until such time that we can generate significant revenue from product sales, if ever, we expect to finance our operations through a combination of equity or debt financings or other sources, which may include collaborations with third parties. We may never successfully complete development of any of our product candidates; obtain adequate patent protection or other exclusivity for our product candidates; obtain necessary regulatory approval for our product candidates; or achieve commercial viability for any approved product. Adequate additional financing may not be available to us on acceptable terms, or at all. Our inability to raise capital as and when needed would have a negative impact on our financial condition and on our ability to pursue our business strategy. Arrangements with collaborators or others may require us to relinquish rights to certain of our technologies or product candidates. We will need to generate significant revenue to achieve profitability, and we may never do so.

We expect that our existing cash, cash equivalents and marketable securities as of September 30, 2017 will enable us to fund our operating expenses and capital expenditure requirements, based on our current operating plan, into the second quarter of 2018. See “—Liquidity and Capital Resources”.

Financial Operations Overview

Revenue

We have not generated any revenue from product sales since our inception, and do not expect to generate any revenue from the sale of products in the near future. If our development efforts result in clinical success and regulatory approval or collaboration agreements with third parties for our product candidates, we may generate revenue from those product candidates.

Operating Expenses

Our operating expenses since inception have consisted primarily of costs associated with research and development activities and general and administrative activities.

Research and Development Expenses

Research and development expenses, which consist primarily of costs associated with our product research and development efforts, are expensed as incurred. Research and development expenses consist primarily of:

 

personnel costs, including salaries, benefits, stock-based compensation and travel expenses, for employees engaged in research and development functions;

 

expenses incurred under agreements with contract research organizations, or CROs, and sites that conduct our non-clinical studies and clinical trials;

 

expenses associated with manufacturing materials for use in clinical trials, developing external manufacturing capabilities and validating our manufacturing processes;

 

costs of outside consultants engaged in research and development activities, including their fees, stock-based compensation and travel expenses;

 

other expenses related to our non-clinical studies and clinical trials and expenses related to our regulatory activities; and

 

payments made under our third-party license agreements.

Costs for certain development activities are recognized based on an evaluation of the progress to completion of specific tasks using information and data provided to us by our vendors and our clinical sites.

We have been developing our product candidates and focusing on other research and development programs, including exploratory efforts to identify new compounds, target validation for identified compounds and lead optimization for our earlier-validated programs. Our direct research and development expenses are tracked on a program-by-program basis, and consist primarily of external costs, such as fees paid to investigators, central laboratories, CROs and contract manufacturing organizations, or CMOs, in

22


 

connection with our non-clinical studies and clinical trials; third-party license fees related to our product candidates; and fees paid to outside consultants who perform work on our programs. We do not allocate employee-related costs and other indirect costs to specific research and development programs because these costs are deployed across multiple product programs under research and development and, as such, are separately classified as unallocated research and development expenses.

Research and development activities are central to our business. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will continue to increase in the foreseeable future as we continue or initiate clinical trials and non-clinical studies for certain product candidates, pursue later stages of clinical development of our product candidates and continue our earlier stage exploratory efforts.

We cannot determine with certainty the duration and costs of the current or future clinical trials of our product candidates or if, when, or to what extent we will generate revenue from the commercialization and sale of any of our product candidates, if approved for marketing and sale. The duration, costs, and timing of clinical trials and development of our product candidates will depend on a variety of factors, including:

 

the scope, size, rate of progress, and expense of our ongoing as well as any additional clinical trials, non-clinical studies,  and other research and development activities;

 

future clinical trial and non-clinical study results;

 

decisions by regulatory authorities related to our product candidates;

 

uncertainties in clinical trial enrollment rate or design;