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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DC 20549
FORM 10-Q
(Mark One)
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☒ |
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the quarterly period ended June 30, 2024
OR
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☐ |
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: 001-41199
Amylyx Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)
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Delaware |
46-4600503 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer
Identification No.) |
43 Thorndike St. Cambridge, Massachusetts |
02141 |
(Address of principal executive offices) |
(Zip Code) |
(617) 682-0917
(Registrant’s telephone number, including area code)
Not Applicable
(Former name, former address and former fiscal year, if changed since last report)
Securities registered pursuant to Section 12(b) of the Act:
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Title of each class |
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Trading Symbol(s) |
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Name of each exchange on which registered |
Common Stock, $0.0001 par value per share |
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AMLX |
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Nasdaq Global Select Stock Market |
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 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). Yes ☒ No ☐
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.
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Large accelerated filer |
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Accelerated filer |
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Non-accelerated filer |
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☐ |
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Smaller reporting company |
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☒ |
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Emerging growth company |
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☐ |
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 July 31, 2024, the registrant had 68,082,468 shares of common stock, $0.0001 par value per share, outstanding.
AMYLYX PHARMACEUTICALS, INC.
QUARTERLY REPORT ON FORM 10-Q
FOR THE QUARTERLY PERIOD ENDED JUNE 30, 2024
Table of Contents
From time to time, we may use our website or our LinkedIn profile at www.linkedin.com/company/amylyx to distribute material information. Our financial and other material information is routinely posted to and accessible on the Investors section of our website, available at www.amylyx.com. Investors are encouraged to review the Investors section of our website because we may post material information on that site that is not otherwise disseminated by us. Information that is contained in and can be accessed through our website or our LinkedIn page is not incorporated into, and does not form a part of, this Quarterly Report on Form 10-Q.
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q, or Quarterly Report, contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act. 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 “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” or the negative of these terms or other comparable terminology. These statements are not guarantees of future results or performance and involve substantial risks and uncertainties. Forward-looking statements in this Quarterly Report include, but are not limited to, express or implied statements about:
•our ability to obtain regulatory approvals of AMX0035 in Wolfram syndrome, progressive supranuclear palsy, or PSP, or any other indications, avexitide, or any other current or future product candidates;
•the timing, progress and results of our research and development activities, preclinical studies and clinical trials, including our Phase 2b/3 global clinical trial of AMX0035 for the treatment of PSP known as the ORION trial, our Phase 2 clinical trial of AMX0035 for the treatment of Wolfram syndrome known as the HELIOS trial, and the anticipated Phase 3 clinical program for avexitide in post-bariatric hypoglycemia, or PBH, as well as any other development efforts, preclinical studies and clinical trials for our current and any future product candidates;
•our ability to successfully commercialize and market our product candidates, if approved, and the timing of any commercialization and marketing efforts;
•our ability to contract with third-party suppliers, manufacturers and other service providers and their ability to perform adequately and to produce sufficient quantities of clinical and, if needed, commercial supplies;
•the market size, opportunity, demand and growth potential for our current and any future product candidates, if approved;
•our ability to build and maintain our own sales and marketing capabilities, or seek collaborative partners, to commercialize our current and any future product candidates, if approved;
•our ability to obtain funding for our operations;
•the timing to implement our restructuring plan and our ability to refocus our operations on our revised priorities;
•our ability to retain the continued service of our key executives and to identify, hire and retain additional qualified professionals;
•our ability to successfully complete our ongoing or planned clinical trials of AMX0035 and avexitide and to advance any other current or future product candidates into, and successfully complete, preclinical studies and clinical trials;
•our ability to successfully recruit and enroll suitable patients in our clinical trials;
•the timing or likelihood of the accomplishment of various scientific, clinical, regulatory filings and approvals and other product development objectives;
•the rate and degree of market acceptance of AMX0035, avexitide and any other current or future product candidates, if approved, by physicians, patients, third-party payors and others in the medical community;
•the implementation of our business model and strategic plans for our business, products, product candidates and technology;
•our ability to identify, evaluate, in-license and develop additional products or product candidates to complement our existing pipeline and ability to successfully incorporate acquired assets into our existing pipeline;
•the scope of protection we are able to establish and maintain for intellectual property rights covering our products, product candidates and technology;
•developments relating to our competitors and our industry, including any regulatory developments;
•our estimates regarding expenses, revenue, capital requirements, cash runway and future needs for additional financing;
•fluctuations of our quarterly and annual operating results and the related effects on our stock price;
•the effect of global financial and economic conditions and geopolitical events, including heightened and fluctuating interest rates and inflation, foreign exchange fluctuations, particularly the Pound Sterling to U.S. Dollar, the risk of
economic slowdown or recession in the United States, instability in the banking system, overall market volatility in the United States or the United Kingdom, including as a result of, among other factors, the ongoing war between Russia and Ukraine, the Israel-Hamas war and escalating conflict in the Middle East, the upcoming presidential election in the United States, including political unrest, or similar events, on our business; and
•other statements about future events, including those listed under the section titled “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 the section titled “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.
All of our forward-looking statements are as of the date of this Quarterly Report only. In each case, actual results may differ materially from such forward-looking information. We can give no assurance that such expectations or forward-looking statements will prove to be correct. An occurrence of or any material adverse change in one or more of the risk factors or risks and uncertainties referred to in this Quarterly Report or included in our other public disclosures or our other periodic reports or other documents or filings filed with or furnished to the Securities and Exchange Commission, or the SEC, could materially and adversely affect our business, prospects, financial condition and results of operations. Except as required by law, we do not undertake or plan to update or revise any such forward-looking statements to reflect actual results, changes in plans, assumptions, estimates or projections or other circumstances affecting such forward-looking statements occurring after the date of this Quarterly Report, even if such results, changes or circumstances make it clear that any forward-looking information will not be realized. Any public statements or disclosures by us following this Quarterly Report that modify or impact any of the forward-looking statements contained in this Quarterly Report will be deemed to modify or supersede such statements in this Quarterly Report.
We may from time to time provide 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.
TRADEMARKS
Solely for convenience, our trademarks and trade names in this report are referred to without the ® and ™ symbols, but such references should not be construed as any indicator that we will not assert, to the fullest extent under applicable law, our rights thereto.
PART I—FINANCIAL INFORMATION
Item 1. Financial Statements.
AMYLYX PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share data)
(Unaudited)
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June 30, 2024 |
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December 31, 2023 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
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$ |
74,999 |
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$ |
170,201 |
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Short-term investments |
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234,813 |
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201,161 |
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Accounts receivable, net |
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4,091 |
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40,050 |
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Inventories |
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— |
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38,323 |
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Prepaid expenses and other current assets |
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12,819 |
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14,931 |
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Total current assets |
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326,722 |
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464,666 |
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Property and equipment, net |
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1,473 |
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2,686 |
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Restricted cash equivalents |
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719 |
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719 |
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Operating lease right-of-use assets |
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2,769 |
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3,725 |
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Long-term inventories |
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— |
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44,957 |
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Other assets |
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479 |
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701 |
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Total assets |
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$ |
332,162 |
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$ |
517,454 |
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Liabilities and Stockholders’ Equity |
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Current liabilities: |
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Accounts payable |
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$ |
4,955 |
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$ |
22,061 |
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Accrued expenses |
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62,757 |
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57,724 |
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Operating lease liabilities, current portion |
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2,369 |
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2,257 |
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Total current liabilities |
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70,081 |
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82,042 |
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Operating lease liabilities, net of current portion |
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772 |
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1,980 |
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Total liabilities |
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70,853 |
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84,022 |
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Commitments and contingencies (Note 10) |
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Stockholders’ equity: |
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Preferred stock, $0.0001 par value; 10,000,000 shares authorized |
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— |
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— |
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Common stock, $0.0001 par value; 300,000,000 shares authorized; 68,079,057 and 67,707,432 shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively |
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7 |
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7 |
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Additional paid-in capital |
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757,841 |
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738,177 |
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Accumulated deficit |
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(496,442 |
) |
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(304,949 |
) |
Accumulated other comprehensive (loss) income |
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(97 |
) |
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197 |
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Total stockholders’ equity |
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261,309 |
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433,432 |
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Total liabilities and stockholders’ equity |
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$ |
332,162 |
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$ |
517,454 |
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The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
AMYLYX PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share data)
(Unaudited)
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Three Months Ended June 30, |
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Six Months Ended June 30, |
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2024 |
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2023 |
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2024 |
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2023 |
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Product revenue, net |
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$ |
(1,023 |
) |
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$ |
98,216 |
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$ |
87,620 |
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$ |
169,644 |
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Operating expenses: |
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Cost of sales |
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8 |
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5,580 |
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5,953 |
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10,863 |
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Cost of sales - inventory impairment and loss on firm purchase commitments |
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7,410 |
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— |
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117,871 |
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— |
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Research and development |
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23,347 |
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29,044 |
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59,955 |
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53,236 |
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Selling, general and administrative |
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21,647 |
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43,391 |
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79,406 |
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87,397 |
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Restructuring expenses |
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22,851 |
|
|
|
— |
|
|
|
22,851 |
|
|
|
— |
|
Total operating expenses |
|
|
75,263 |
|
|
|
78,015 |
|
|
|
286,036 |
|
|
|
151,496 |
|
(Loss) income from operations |
|
|
(76,286 |
) |
|
|
20,201 |
|
|
|
(198,416 |
) |
|
|
18,148 |
|
Other income, net: |
|
|
|
|
|
|
|
|
|
|
|
|
Interest income |
|
|
4,069 |
|
|
|
3,887 |
|
|
|
8,395 |
|
|
|
7,605 |
|
Other expense, net |
|
|
(483 |
) |
|
|
(81 |
) |
|
|
(1,230 |
) |
|
|
(343 |
) |
Total other income, net |
|
|
3,586 |
|
|
|
3,806 |
|
|
|
7,165 |
|
|
|
7,262 |
|
(Loss) income before income taxes |
|
|
(72,700 |
) |
|
|
24,007 |
|
|
|
(191,251 |
) |
|
|
25,410 |
|
Provision for income taxes |
|
|
— |
|
|
|
1,933 |
|
|
|
242 |
|
|
|
1,763 |
|
Net (loss) income |
|
$ |
(72,700 |
) |
|
$ |
22,074 |
|
|
$ |
(191,493 |
) |
|
$ |
23,647 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net (loss) income per share |
|
|
|
|
|
|
|
|
|
|
|
|
Basic |
|
$ |
(1.07 |
) |
|
$ |
0.33 |
|
|
$ |
(2.82 |
) |
|
$ |
0.35 |
|
Diluted |
|
$ |
(1.07 |
) |
|
$ |
0.31 |
|
|
$ |
(2.82 |
) |
|
$ |
0.34 |
|
Weighted-average shares used in computing net (loss) income per share |
|
|
|
|
|
|
|
|
|
|
|
|
Basic |
|
|
68,024,929 |
|
|
|
67,233,617 |
|
|
|
67,939,642 |
|
|
|
66,976,871 |
|
Diluted |
|
|
68,024,929 |
|
|
|
70,132,040 |
|
|
|
67,939,642 |
|
|
|
70,471,821 |
|
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
AMYLYX PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE (LOSS) INCOME
(in thousands)
(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
|
Six Months Ended June 30, |
|
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
Net (loss) income |
|
$ |
(72,700 |
) |
|
$ |
22,074 |
|
|
$ |
(191,493 |
) |
|
$ |
23,647 |
|
Other comprehensive (loss) income |
|
|
|
|
|
|
|
|
|
|
|
|
Foreign currency translation (loss) gain |
|
|
(43 |
) |
|
|
(18 |
) |
|
|
(201 |
) |
|
|
60 |
|
Net unrealized gain (loss) on available-for-sale securities |
|
|
23 |
|
|
|
(79 |
) |
|
|
(93 |
) |
|
|
(46 |
) |
Other comprehensive (loss) income |
|
|
(20 |
) |
|
|
(97 |
) |
|
|
(294 |
) |
|
|
14 |
|
Comprehensive (loss) income |
|
$ |
(72,720 |
) |
|
$ |
21,977 |
|
|
$ |
(191,787 |
) |
|
$ |
23,661 |
|
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
AMYLYX PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY
(in thousands, except share data)
(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Common Stock |
|
|
Additional
Paid-In |
|
|
Accumulated
Other
Comprehensive |
|
|
Accumulated |
|
|
Total
Stockholders’ |
|
|
|
Shares |
|
|
Amount |
|
|
Capital |
|
|
Income (Loss) |
|
|
Deficit |
|
|
Equity |
|
Balance as of December 31, 2023 |
|
|
67,707,432 |
|
|
$ |
7 |
|
|
$ |
738,177 |
|
|
$ |
197 |
|
|
$ |
(304,949 |
) |
|
$ |
433,432 |
|
Issuance of common stock upon exercise of stock options |
|
|
49,618 |
|
|
|
— |
|
|
|
147 |
|
|
|
— |
|
|
|
— |
|
|
|
147 |
|
Issuance of common stock upon vesting of RSUs |
|
|
218,537 |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
Stock-based compensation expense |
|
|
— |
|
|
|
— |
|
|
|
9,924 |
|
|
|
— |
|
|
|
— |
|
|
|
9,924 |
|
Other comprehensive loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(274 |
) |
|
|
— |
|
|
|
(274 |
) |
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(118,793 |
) |
|
|
(118,793 |
) |
Balance as of March 31, 2024 |
|
|
67,975,587 |
|
|
$ |
7 |
|
|
$ |
748,248 |
|
|
$ |
(77 |
) |
|
$ |
(423,742 |
) |
|
$ |
324,436 |
|
Issuance of common stock upon exercise of stock options |
|
|
59,981 |
|
|
|
— |
|
|
|
23 |
|
|
|
— |
|
|
|
— |
|
|
|
23 |
|
Issuance of common stock upon vesting of RSUs |
|
|
43,489 |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
Stock-based compensation expense |
|
|
— |
|
|
|
— |
|
|
|
9,570 |
|
|
|
— |
|
|
|
— |
|
|
|
9,570 |
|
Other comprehensive loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(20 |
) |
|
|
— |
|
|
|
(20 |
) |
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(72,700 |
) |
|
|
(72,700 |
) |
Balance as of June 30, 2024 |
|
|
68,079,057 |
|
|
$ |
7 |
|
|
$ |
757,841 |
|
|
$ |
(97 |
) |
|
$ |
(496,442 |
) |
|
$ |
261,309 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Common Stock |
|
|
Additional
Paid-In |
|
|
Accumulated
Other
Comprehensive |
|
|
Accumulated |
|
|
Total
Stockholders’ |
|
|
|
Shares |
|
|
Amount |
|
|
Capital |
|
|
Income (Loss) |
|
|
Deficit |
|
|
Equity |
|
Balance as of December 31, 2022 |
|
|
66,512,011 |
|
|
$ |
7 |
|
|
$ |
694,906 |
|
|
$ |
(86 |
) |
|
$ |
(354,220 |
) |
|
$ |
340,607 |
|
Issuance of common stock upon exercise of stock options |
|
|
451,298 |
|
|
|
— |
|
|
|
2,777 |
|
|
|
— |
|
|
|
— |
|
|
|
2,777 |
|
Issuance of common stock upon vesting of RSUs |
|
|
132,294 |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
Stock-based compensation expense |
|
|
— |
|
|
|
— |
|
|
|
7,580 |
|
|
|
— |
|
|
|
— |
|
|
|
7,580 |
|
Other comprehensive loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
111 |
|
|
|
— |
|
|
|
111 |
|
Net income |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
1,573 |
|
|
|
1,573 |
|
Balance as of March 31, 2023 |
|
|
67,095,603 |
|
|
$ |
7 |
|
|
$ |
705,263 |
|
|
$ |
25 |
|
|
$ |
(352,647 |
) |
|
$ |
352,648 |
|
Issuance of common stock upon exercise of stock options |
|
|
226,138 |
|
|
|
— |
|
|
|
1,687 |
|
|
|
— |
|
|
|
— |
|
|
|
1,687 |
|
Issuance of common stock upon vesting of RSUs |
|
|
32,676 |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
Stock-based compensation expense |
|
|
— |
|
|
|
— |
|
|
|
10,053 |
|
|
|
— |
|
|
|
— |
|
|
|
10,053 |
|
Other comprehensive loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(97 |
) |
|
|
— |
|
|
|
(97 |
) |
Net income |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
22,074 |
|
|
|
22,074 |
|
Balance as of June 30, 2023 |
|
$ |
67,354,417 |
|
|
$ |
7 |
|
|
$ |
717,003 |
|
|
$ |
(72 |
) |
|
$ |
(330,573 |
) |
|
$ |
386,365 |
|
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
AMYLYX PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
Six Months Ended June 30, |
|
|
|
2024 |
|
|
2023 |
|
Cash flows (used in) provided by operating activities: |
|
|
|
|
|
|
Net (loss) income |
|
$ |
(191,493 |
) |
|
$ |
23,647 |
|
Adjustments to reconcile net (loss) income to net cash (used in) provided by operating activities: |
|
|
|
|
|
|
Stock-based compensation expense |
|
|
19,494 |
|
|
|
17,451 |
|
Depreciation expense |
|
|
523 |
|
|
|
485 |
|
Accretion of investment discounts, net |
|
|
(5,760 |
) |
|
|
(4,886 |
) |
Inventory impairment and loss on firm purchase commitments |
|
|
117,871 |
|
|
|
— |
|
Other non-cash items |
|
|
827 |
|
|
|
— |
|
Changes in operating assets and liabilities: |
|
|
|
|
|
|
Accounts receivable, net |
|
|
35,958 |
|
|
|
(18,167 |
) |
Inventories |
|
|
(9,253 |
) |
|
|
(32,636 |
) |
Interest receivable |
|
|
421 |
|
|
|
(356 |
) |
Prepaid expenses and other current assets |
|
|
1,657 |
|
|
|
(1,318 |
) |
Operating lease right-of-use assets |
|
|
957 |
|
|
|
881 |
|
Other assets |
|
|
217 |
|
|
— |
|
Accounts payable |
|
|
(17,087 |
) |
|
|
18,455 |
|
Accrued expenses |
|
|
(20,305 |
) |
|
|
971 |
|
Operating lease liabilities |
|
|
(1,096 |
) |
|
|
(990 |
) |
Net cash (used in) provided by operating activities |
|
|
(67,069 |
) |
|
|
3,537 |
|
Cash flows (used in) provided by investing activities: |
|
|
|
|
|
|
Purchases of property and equipment |
|
|
(156 |
) |
|
|
(528 |
) |
Purchases of investments |
|
|
(231,986 |
) |
|
|
(9,756 |
) |
Proceeds from maturities of short-term investments |
|
|
204,000 |
|
|
|
189,500 |
|
Net cash (used in) provided by investing activities |
|
|
(28,142 |
) |
|
|
179,216 |
|
Cash flows provided by financing activities: |
|
|
|
|
|
|
Follow-on offering costs paid |
|
|
— |
|
|
|
(136 |
) |
Proceeds from exercise of stock options and RSUs vesting |
|
|
1,493 |
|
|
|
5,663 |
|
Withholding taxes paid on stock-based awards |
|
|
(1,335 |
) |
|
|
(3,103 |
) |
Net cash provided by financing activities |
|
|
158 |
|
|
|
2,424 |
|
Effect of exchange rate changes on cash, cash equivalents and restricted cash equivalents |
|
|
(149 |
) |
|
|
57 |
|
Net (decrease) increase in cash, cash equivalents and restricted cash equivalents |
|
|
(95,202 |
) |
|
|
185,234 |
|
Cash, cash equivalents and restricted cash equivalents, beginning of period |
|
|
170,920 |
|
|
|
63,245 |
|
Cash, cash equivalents and restricted cash equivalents, end of period |
|
$ |
75,718 |
|
|
$ |
248,479 |
|
Supplemental disclosure of cash flow information: |
|
|
|
|
|
|
Taxes withheld on stock-based awards included in accrued expenses |
|
$ |
12 |
|
|
$ |
67 |
|
Purchases of property and equipment included in accounts payable |
|
$ |
— |
|
|
$ |
81 |
|
Income taxes paid |
|
$ |
281 |
|
|
$ |
2,562 |
|
The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.
AMYLYX PHARMACEUTICALS, INC.
NOTES TO CONDENSED Consolidated FINANCIAL STATEMENTS
(unaudited)
1. Nature of the Business
Amylyx Pharmaceuticals, Inc., together with its wholly owned subsidiaries, known as Amylyx or the Company, is a biotechnology company that is committed to the discovery and development of new treatment options for communities with high unmet needs, including people living with serious and fatal diseases. The Company has preclinical or clinical development programs underway in neurodegenerative, neuroendocrine and endocrine diseases.
On July 9, 2024, Amylyx completed the acquisition of avexitide for the potential treatment of hyperinsulinemic hypoglycemia, a disease of the endocrine system. Avexitide is an investigational, first-in-class glucagon-like peptide-1, or GLP-1, receptor antagonist that has been evaluated in five clinical trials for post-bariatric hypoglycemia, or PBH, and has also been studied in congenital hyperinsulinism, or HI, two indications characterized by hyperinsulinemic hypoglycemia. The U.S. Food and Drug Administration, or FDA, has granted avexitide breakthrough therapy designation for both indications, rare pediatric disease designation in congenital HI, and orphan drug designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). Amylyx expects to begin the Phase 3 program for avexitide in PBH in the first quarter of 2025.
The Company is also investigating AMX0035, developed by Amylyx, in both neurodegenerative and neuroendocrine diseases where endoplasmic reticulum, or ER, stress and mitochondrial dysfunction are implicated, including progressive supranuclear palsy, or PSP, and Wolfram syndrome. The Company dosed the first participant in the HELIOS trial, a Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome, in April 2023. Amylyx plans to present the HELIOS topline data for all 12 participants at Week 24, including all of the longer-term data available for participants who have reached their Week 36 or Week 48 visit at that time, at a medical meeting this fall. The Company is engaging with stakeholders, including the FDA, and planning for a single Phase 3 clinical trial and will share additional details on the clinical trial design once finalized. The Company dosed the first participant in the ORION trial, a Phase 2b/3 global clinical trial of AMX0035 for the treatment of PSP, in December 2023. Data from an interim analysis of the ORION are expected in mid-2025. The Company is also advancing additional drug candidates developed by Amylyx for neurodegenerative diseases including AMX0114, a potent antisense oligonucleotide targeting inhibition of Calpain-2, a key contributor to the axonal (also known as Wallerian) degeneration pathway. The Company is preparing to initiate a multiple ascending dose clinical trial of AMX0114 in people living with ALS in the second half of 2024.
In April 2024, the Company announced it had started a process with the FDA and Heath Canada to voluntarily discontinue the marketing authorizations for RELYVRIO®/ALBRIOZA™ (AMX0035) for amyotrophic lateral sclerosis, or ALS, and remove the product from the market in the U.S. and Canada based on topline results from the global Phase 3 PHOENIX trial, which failed to meet its prespecified primary and secondary endpoints. The Company will continue to collect available data on survival and to share learnings from PHOENIX to help inform future ALS research. Amylyx is planning to wind down the Open Label Extension by the end of 2024. Separately, the PHOENIX survival data collection efforts will continue at the encouragement of ALS specialists.
Risks and Uncertainties
The Company is subject to risks and uncertainties common to companies in the biotechnology industry, including, but not limited to, the outcome of preclinical studies and clinical trials, potential difficulties with or delays in timing with respect to regulatory approval processes, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations, ability to secure additional capital to fund operations, and risks associated with the economic challenges caused by economic uncertainty in various global markets caused by geopolitical instability and conflict. The Company and its contractors may experience disruptions in supply of items that are essential for its research and development activities, including, for example, raw materials and bulk drug substances that the Company imports from Europe and Canada used in the manufacturing of AMX0035, avexitide and any additional or future product candidates.
2. Summary of Significant Accounting Policies
Significant Accounting Policies
The Company’s significant accounting policies are disclosed in the audited consolidated financial statements for the year ended December 31, 2023 and the notes thereto, which are included in the Company’s most recent Annual Report on Form 10-K. Since the date of those consolidated financial statements, there have been no material changes to its significant accounting policies.
Basis of Presentation and Consolidation
The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the U.S., or GAAP, and include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. In the opinion of the Company's management, all normal and recurring adjustments necessary for a fair presentation have been reflected. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the ASC, and Accounting Standards Update, or ASU, of the Financial Accounting Standards Board, or FASB.
The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates.
New Accounting Pronouncements Not Yet Adopted
In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvement to Income Tax Disclosures, or ASU 2023-09, to enhance the transparency and decision usefulness of income tax disclosures. ASU 2023-09 is effective for annual periods beginning after December 15, 2024, on a prospective basis. Early adoption and retrospective application are permitted. The Company is currently evaluating the impact of this ASU on its consolidated financial statements and related disclosures.
In November 2023, the FASB issued ASU No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures, or ASU 2023-09, which requires public entities to disclose information about their reportable segments’ significant expenses on an interim and annual basis. ASU 2023-07 is effective for the Company beginning the year ended December 31, 2024. The Company is currently evaluating the impact of this ASU on its consolidated financial statements and related disclosures.
3. PRODUCT REVENUE, NET
To date, the Company’s only source of product revenue had been from sales of RELYVRIO, known as ALBRIOZA in Canada. Significant judgment was required in estimating GTN adjustments considering historical experience, payer channel mix (e.g., Medicare or Medicaid), current contract prices under applicable programs, unbilled claims and processing time lags and inventory levels in the distribution channel. In April 2024, the Company announced it had started a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for RELYVRIO®/ALBRIOZA™ and remove the product from the market in the U.S. and Canada based on topline results from the Phase 3 PHOENIX trial. As a result, the Company did not generate significant revenue from product sales for the three months ended June 30, 2024. The following table reconciles gross product revenue to net product revenue (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
|
Six Months Ended June 30, |
|
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
Product revenue, gross |
|
$ |
605 |
|
|
$ |
108,405 |
|
|
$ |
114,265 |
|
|
$ |
192,958 |
|
GTN adjustments |
|
|
(1,628 |
) |
|
|
(10,189 |
) |
|
|
(26,645 |
) |
|
|
(23,314 |
) |
Product revenue, net |
|
$ |
(1,023 |
) |
|
$ |
98,216 |
|
|
$ |
87,620 |
|
|
$ |
169,644 |
|
The activity and ending reserve balance for GTN adjustments were as follows for the six months ended June 30, 2024 and 2023 (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Chargebacks and Cash Discounts |
|
|
Medicaid and Medicare Rebates |
|
|
Other Rebates, Returns, Discounts and Adjustments |
|
|
Total |
|
Ending balance at December 31, 2023 |
|
$ |
3,143 |
|
|
$ |
4,946 |
|
|
$ |
11,073 |
|
|
$ |
19,162 |
|
Provision related to sales in the current year |
|
|
4,983 |
|
|
|
3,504 |
|
|
|
20,916 |
|
|
|
29,403 |
|
Adjustments related to prior period sales |
|
|
(1,696 |
) |
|
|
— |
|
|
|
(1,062 |
) |
|
|
(2,758 |
) |
Credits and payments made |
|
|
(6,430 |
) |
|
|
(7,664 |
) |
|
|
(8,828 |
) |
|
|
(22,922 |
) |
Ending balance at June 30, 2024 |
|
$ |
— |
|
|
$ |
786 |
|
|
$ |
22,099 |
|
|
$ |
22,885 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Chargebacks and Cash Discounts |
|
|
Medicaid and Medicare Rebates |
|
|
Other Rebates, Returns, Discounts and Adjustments |
|
|
Total |
|
Ending balance at December 31, 2022 |
|
$ |
648 |
|
|
$ |
1,992 |
|
|
$ |
1,664 |
|
|
$ |
4,304 |
|
Provision related to sales in the current year |
|
|
10,053 |
|
|
|
6,516 |
|
|
|
7,147 |
|
|
|
23,716 |
|
Adjustments related to prior period sales |
|
|
— |
|
|
|
(236 |
) |
|
|
(166 |
) |
|
|
(402 |
) |
Credits and payments made |
|
|
(5,831 |
) |
|
|
(4,294 |
) |
|
|
(5,988 |
) |
|
|
(16,113 |
) |
Ending balance at June 30, 2023 |
|
$ |
4,870 |
|
|
$ |
3,978 |
|
|
$ |
2,657 |
|
|
$ |
11,505 |
|
Included in the ending reserve balance for GTN adjustments are chargebacks resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to customers who directly purchase the product from the Company, discounts to customers for prompt payment and estimates for product returns. Chargebacks, discounts and returns are recorded as reductions of accounts receivable, net on the condensed consolidated balance sheets to the extent there are receivable balances to reduce. If there are net balance owed to customers, they are recorded as a component of accrued expenses on the condensed consolidated balance sheets. In addition, included in the ending reserve balance for GTN adjustments are Medicaid and Medicare rebates, other rebates for obligations under voluntary patient assistance programs, and accrued fees payable to customers. Medicaid and Medicare rebates, other rebates and fees are recorded as a component of accrued expenses on the condensed consolidated balance sheets.
4. SHORT-TERM INVESTMENTS
The Company has classified all of its marketable securities at June 30, 2024 as “available-for-sale”. The Company records available-for-sale securities at fair value, with the unrealized gains and losses included as a separate component of other accumulated comprehensive income (loss). There were no realized gains or losses recognized during the three and six months ended June 30, 2024 and 2023.
The Company adjusts the cost of available-for-sale debt securities for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion are included in interest income. The cost of securities sold is based on the specific identification method. The Company includes interest and dividends on securities classified as available-for-sale in interest income. Accrued interest receivable relating to the Company's available-for-sale securities is presented within prepaid expenses and other current assets in the accompanying condensed consolidated balance sheets, and amounted to less than $0.1 million and $0.5 million at June 30, 2024 and December 31, 2023, respectively.
As of June 30, 2024, there were no securities in an unrealized loss position for greater than 12 months. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost bases. The Company did not record an allowance for credit losses as of June 30, 2024.
Short-term investments, which are classified as available-for-sale, consisted of the following (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at June 30, 2024: |
|
Amortized
Cost Basis |
|
|
Unrealized
Gain |
|
|
Unrealized
Loss |
|
|
Fair
Value |
|
Treasury bills |
|
$ |
234,842 |
|
|
$ |
1 |
|
|
$ |
(30 |
) |
|
$ |
234,813 |
|
Total short-term investments |
|
$ |
234,842 |
|
|
$ |
1 |
|
|
$ |
(30 |
) |
|
$ |
234,813 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at December 31, 2023: |
|
Amortized
Cost Basis |
|
|
Unrealized
Gain |
|
|
Unrealized
Loss |
|
|
Fair
Value |
|
Treasury bills |
|
$ |
196,098 |
|
|
$ |
67 |
|
|
$ |
— |
|
|
$ |
196,165 |
|
U.S. agency bonds |
|
|
4,999 |
|
|
|
— |
|
|
|
(3 |
) |
|
|
4,996 |
|
Total short-term investments |
|
$ |
201,097 |
|
|
$ |
67 |
|
|
$ |
(3 |
) |
|
$ |
201,161 |
|
5. INVENTORIES
Inventories consisted of the following (in thousands):
|
|
|
|
|
|
|
|
|
|
|
June 30, 2024 |
|
|
December 31, 2023 |
|
Raw materials |
|
$ |
— |
|
|
$ |
53,144 |
|
Work in process |
|
|
— |
|
|
|
18,945 |
|
Finished goods |
|
|
— |
|
|
|
11,191 |
|
Total inventories |
|
$ |
— |
|
|
$ |
83,280 |
|
In April, 2024, the Company announced it had started a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for RELYVRIO®/ALBRIOZA™ and remove the product from the market in the U.S. and Canada based on topline results from the global Phase 3 PHOENIX trial. As a result, the Company recorded approximately $92.5 million of charges associated with the write-down of inventory for the six months ended June 30, 2024. Inventory write-downs for the six months ended June 30, 2023 were immaterial.
6. Accrued Expenses
Accrued expenses consisted of the following (in thousands):
|
|
|
|
|
|
|
|
|
|
|
June 30, 2024 |
|
|
December 31, 2023 |
|
Accrued external research and development |
|
$ |
7,466 |
|
|
$ |
12,625 |
|
Accrued benefits and incentive compensation |
|
|
6,067 |
|
|
|
16,790 |
|
Accrued manufacturing |
|
|
9,008 |
|
|
|
1,652 |
|
Accrued consulting and other professional fees |
|
|
2,424 |
|
|
|
6,506 |
|
Accrued returns, rebates and co-pay assistance |
|
|
22,059 |
|
|
|
16,063 |
|
Accrued royalties |
|
|
— |
|
|
|
3,111 |
|
Accrued loss on future purchase commitments |
|
|
12,604 |
|
|
|
— |
|
Accrued restructuring expenses |
|
|
2,648 |
|
|
|
— |
|
Other accrued expenses |
|
|
481 |
|
|
|
977 |
|
Total accrued expenses |
|
$ |
62,757 |
|
|
$ |
57,724 |
|
7. FAIR VALUE MEASUREMENTS
The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values (in thousands):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June 30, 2024 |
|
|
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
|
Total |
|
Assets: |
|
|
|
|
|
|
|
|
|
|
|
|
Cash equivalents |
|
$ |
536 |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
536 |
|
Short-term investments: |
|
|
|
|
|
|
|
|
|
|
|
|
Treasury bills |
|
|
234,813 |
|
|
|
— |
|
|
|
— |
|
|
|
234,813 |
|
Total short-term investments |
|
|
234,813 |
|
|
|
— |
|
|
|
— |
|
|
|
234,813 |
|
Restricted cash equivalents |
|
|
719 |
|
|
|
— |
|
|
|
— |
|
|
|
719 |
|
Total financial assets |
|
$ |
236,068 |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
236,068 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, 2023 |
|
|
|
Level 1 |
|
|
Level 2 |
|
|
Level 3 |
|
|
Total |
|
Assets: |
|
|
|
|
|
|
|
|
|
|
|
|
Cash equivalents |
|
$ |
76,710 |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
76,710 |
|
Short-term investments: |
|
|
|
|
|
|
|
|
|
|
|
|
Treasury bills |
|
|
196,165 |
|
|
|
— |
|
|
|
— |
|
|
|
196,165 |
|
U.S. agency bonds |
|
|
— |
|
|
|
4,996 |
|
|
|
— |
|
|
|
4,996 |
|
Total short-term investments |
|
|