SNDX 10-Q
Syndax Pharmaceuticals Inc (SNDX)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
(Mark One)
For the quarterly period ended
or
For the transition period from to
Commission File Number:
(Exact Name of Registrant as Specified in Its Charter)
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(State or Other Jurisdiction of Incorporation or Organization) |
(IRS Employer Identification No.) |
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(Address of Principal Executive Offices) |
(Zip Code) |
(
(Registrant’s Telephone Number, Including Area Code)
Securities registered pursuant to Section 12(b) of the Act:
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered |
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.
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 (Section 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.:
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Accelerated filer |
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Non-accelerated filer |
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Smaller reporting company |
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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 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 31, 2025, there were
g
FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q, or this Quarterly Report, contains forward-looking statements and information within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the "safe harbor" created by those sections. All statements other than statements of historical fact are “forward-looking statements” for purposes of this Quarterly Report on Form 10-Q. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “could,” “estimate,” “expects,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative or plural of those terms, and similar expressions.
Forward-looking statements include, but are not limited to, statements about:
These statements are only current predictions and are subject to known and unknown risks, uncertainties and other factors that may cause our or our industry’s actual results, levels of activity, performance, or achievements to be materially different from those anticipated by the forward-looking statements. We discuss many of these risks in this report in greater detail in the section titled “Risk Factors” and elsewhere in this report. You should not rely upon forward-looking statements as predictions of future events.
Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. Except as required by law, we are under no duty to update or revise any of the forward-looking statements, whether as a result of new information, future events or otherwise.
ii
TABLE OF CONTENTS
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Item 1. |
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Consolidated Balance Sheets as of September 30, 2025 and December 31, 2024 |
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2 |
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3 |
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Consolidated Statements of Cash Flows for the nine months ended September 30, 2025 and 2024 |
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5 |
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Item 2. |
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Management’s Discussion and Analysis of Financial Condition and Results of Operations |
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Item 3. |
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24 |
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Item 4. |
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Item 1. |
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Item 1A. |
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Item 5. |
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28 |
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Item 6. |
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29 |
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c
iii
Part I: FINANCIAL INFORMATION
Item 1: Financial Statements
SYNDAX PHARMACEUTICALS, INC.
(unaudited)
CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share data)
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September 30, 2025 |
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December 31, 2024 |
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ASSETS |
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Current assets: |
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Cash and cash equivalents |
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$ |
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$ |
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Short-term investments |
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Accounts receivable, net |
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Inventory |
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Short-term deposits |
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Other receivable, net |
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Collaboration receivable, net |
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Prepaid expenses and other current assets |
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Total current assets |
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Long-term investments |
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Property and equipment, net |
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Right-of-use asset, net |
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Restricted cash |
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Total assets |
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$ |
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$ |
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LIABILITIES AND STOCKHOLDERS’ EQUITY |
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Current liabilities: |
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Accounts payable |
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$ |
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$ |
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Collaboration payable, net |
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Accrued expenses and other current liabilities |
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Current portion of royalty interest financing liability |
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Current portion of right-of-use liability |
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Current portion of capital lease |
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Total current liabilities |
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Long-term liabilities: |
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Royalty interest financing liability, less current portion |
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Right-of-use liability, less current portion |
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Total long-term liabilities |
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Total liabilities |
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Stockholders’ equity: |
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Preferred stock, $ |
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Common stock, $ |
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Additional paid-in capital |
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Accumulated other comprehensive gain |
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Accumulated deficit |
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( |
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( |
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Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
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$ |
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$ |
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The accompanying notes are an integral part of these unaudited consolidated financial statements.
1
SYNDAX PHARMACEUTICALS, INC.
(unaudited)
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands, except share and per share data)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2025 |
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2024 |
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2025 |
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2024 |
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Revenue: |
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Product revenue, net |
$ |
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$ |
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$ |
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$ |
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Collaboration revenue, net |
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Milestone and license revenue |
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Total revenues |
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Operating expenses: |
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Cost of product sales |
$ |
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$ |
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$ |
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$ |
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Research and development |
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Selling, general and administrative |
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Total operating expenses |
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Loss from operations |
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( |
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( |
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( |
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( |
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Other (expense) income, net: |
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Royalty interest expense |
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( |
) |
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( |
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Other interest expense |
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( |
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( |
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( |
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Interest income |
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Other (expense) income |
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( |
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( |
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( |
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Total other (expense) income, net |
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( |
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( |
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Net loss |
$ |
( |
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$ |
( |
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$ |
( |
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$ |
( |
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Other Comprehensive loss: |
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Unrealized gain on marketable securities |
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Other Comprehensive loss |
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( |
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( |
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( |
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( |
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Net loss per share: |
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Basic and diluted loss per share attributable to common stockholders |
$ |
( |
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$ |
( |
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$ |
( |
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$ |
( |
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Weighted-average common shares used in calculating: |
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Basic and diluted loss per share attributable to common stockholders |
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The accompanying notes are an integral part of these unaudited consolidated financial statements.
2
SYNDAX PHARMACEUTICALS, INC.
(unaudited)
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY
(In thousands, except share and per share data)
Nine months ended September 30, 2025 |
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(In thousands, except share data) |
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Common Stock |
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Additional |
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Accumulated |
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Accumulated |
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Total |
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Shares |
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Amount |
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Balance as of December 31, 2024 |
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$ |
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$ |
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$ |
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$ |
( |
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$ |
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Stock purchase under ESPP |
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— |
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— |
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— |
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— |
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— |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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Unrealized loss on investments |
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— |
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— |
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— |
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— |
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Vesting of RSUs |
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— |
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— |
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— |
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— |
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— |
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Employee withholdings ESPP |
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— |
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— |
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— |
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— |
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Proceeds from exercise of stock options |
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— |
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— |
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— |
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Net loss |
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— |
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— |
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— |
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— |
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( |
) |
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( |
) |
Balance as of March 31, 2025 |
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$ |
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$ |
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$ |
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$ |
( |
) |
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$ |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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Unrealized loss on investments |
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— |
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— |
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— |
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( |
) |
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— |
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( |
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Vesting of RSUs |
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— |
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— |
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— |
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— |
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— |
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Employee withholdings ESPP |
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— |
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— |
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— |
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— |
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Proceeds from exercise of stock options |
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— |
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— |
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— |
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Net loss |
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— |
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— |
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— |
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— |
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( |
) |
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( |
) |
Balance as of June 30, 2025 |
|
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$ |
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$ |
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$ |
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$ |
( |
) |
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$ |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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Unrealized gains on investments |
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— |
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— |
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— |
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— |
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Stock purchase under ESPP |
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— |
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— |
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— |
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— |
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— |
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Employee withholdings ESPP |
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— |
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— |
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( |
) |
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— |
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— |
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( |
) |
Vesting of RSUs |
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— |
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— |
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— |
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— |
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— |
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Proceeds from exercise of stock options |
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— |
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— |
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— |
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Net loss |
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— |
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— |
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— |
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— |
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( |
) |
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( |
) |
Balance as of September 30, 2025 |
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$ |
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$ |
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$ |
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$ |
( |
) |
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$ |
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The accompanying notes are an integral part of these unaudited consolidated financial statements.
3
SYNDAX PHARMACEUTICALS, INC.
(unaudited)
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY
(In thousands, except share and per share data)
(In thousands, except share data) |
|
Common Stock |
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Additional |
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Accumulated |
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Accumulated |
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Total |
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Shares |
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Amount |
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Balance as of December 31, 2023 |
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$ |
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$ |
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$ |
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$ |
( |
) |
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$ |
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Stock purchase under ESPP |
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— |
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— |
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— |
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— |
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— |
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Stock-based compensation expense |
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— |
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— |
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— |
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— |
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Unrealized loss on investments |
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— |
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— |
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— |
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( |
) |
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— |
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( |
) |
Vesting of RSUs |
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— |
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— |
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— |
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— |
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— |
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Employee withholdings ESPP |
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— |
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— |
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— |
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— |
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Proceeds from exercise of stock options |
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— |
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— |
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— |
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|||
Net loss |
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— |
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— |
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— |
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— |
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( |
) |
|
|
( |
) |
Balance as of March 31, 2024 |
|
|
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|
$ |
|
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$ |
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$ |
( |
) |
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$ |
( |
) |
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$ |
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||||
Stock-based compensation expense |
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— |
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— |
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— |
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— |
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||
Unrealized loss on investments |
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— |
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— |
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— |
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( |
) |
|
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— |
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|
( |
) |
Vesting of RSUs |
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— |
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— |
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— |
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— |
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— |
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|
Employee withholdings ESPP |
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— |
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— |
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— |
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— |
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||
Proceeds from exercise of stock options |
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— |
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— |
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— |
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|||
Par value adjustment |
|
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— |
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1 |
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— |
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— |
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— |
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Net loss |
|
|
— |
|
|
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— |
|
|
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— |
|
|
|
— |
|
|
|
( |
) |
|
|
( |
) |
Balance as of June 30, 2024 |
|
|
|
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
|
||||
Stock-based compensation expense |
|
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— |
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— |
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|
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— |
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— |
|
|
|
|
||
Unrealized gains on investments |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
|
||
Stock purchase under ESPP |
|
|
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
Employee withholdings ESPP |
|
|
— |
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
||
Vesting of RSUs |
|
|
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
Proceeds from exercise of stock options |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
|||
Net loss |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
( |
) |
|
|
( |
) |
Balance as of September 30, 2024 |
|
|
|
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||||
The accompanying notes are an integral part of these unaudited consolidated financial statements.
4
SYNDAX PHARMACEUTICALS, INC.
(unaudited)
CONSOLIDATED STATEMENTS OF CASH FLOWS
(In thousands)
|
|
Nine Months Ended September 30, |
|
|||||
|
|
2025 |
|
|
2024 |
|
||
CASH FLOWS FROM OPERATING ACTIVITIES: |
|
|
|
|
|
|
||
Net loss |
|
$ |
( |
) |
|
$ |
( |
) |
Adjustments to reconcile net loss to net cash used in operating activities: |
|
|
|
|
|
|
||
Depreciation |
|
|
|
|
|
|
||
Accretion of investments |
|
|
( |
) |
|
|
( |
) |
Non-cash operating lease expense |
|
|
|
|
|
|
||
Stock-based compensation |
|
|
|
|
|
|
||
Amortization of debt issuance costs |
|
|
|
|
|
|
||
Provision for credit losses |
|
|
|
|
|
|
||
Changes in operating assets and liabilities: |
|
|
|
|
|
|
||
Accounts receivable |
|
|
( |
) |
|
|
|
|
Inventory |
|
|
( |
) |
|
|
|
|
Prepaid expenses and other assets |
|
|
( |
) |
|
|
( |
) |
Collaboration (payable) receivable, net |
|
|
( |
) |
|
|
( |
) |
Other receivable |
|
|
( |
) |
|
|
( |
) |
Other assets |
|
|
|
|
|
|
||
Accounts payable |
|
|
|
|
|
( |
) |
|
Accrued expenses and other liabilities |
|
|
|
|
|
|
||
Net cash used in operating activities |
|
|
( |
) |
|
|
( |
) |
CASH FLOWS FROM INVESTING ACTIVITIES: |
|
|
|
|
|
|
||
Purchases of property and equipment |
|
|
( |
) |
|
|
|
|
Purchases of short and long-term investments |
|
|
( |
) |
|
|
( |
) |
Proceeds from sales and maturities of short-term investments |
|
|
|
|
|
|
||
Net cash provided by investing activities |
|
|
|
|
|
|
||
CASH FLOWS FROM FINANCING ACTIVITIES: |
|
|
|
|
|
|
||
Proceeds from Employee Stock Purchase Plan |
|
|
|
|
|
|
||
Proceeds from stock option exercises |
|
|
|
|
|
|
||
Net cash provided by financing activities |
|
|
|
|
|
|
||
NET DECREASE CASH, CASH EQUIVALENTS AND RESTRICTED CASH |
|
|
( |
) |
|
|
( |
) |
CASH, CASH EQUIVALENTS AND RESTRICTED CASH—beginning of period |
|
|
|
|
|
|
||
CASH, CASH EQUIVALENTS AND RESTRICTED CASH —end of period |
|
$ |
|
|
$ |
|
||
The accompanying notes are an integral part of these unaudited consolidated financial statements.
5
SYNDAX PHARMACEUTICALS, INC.
(unaudited)
NOTES TO CONSOLIDATED FINANCIAL STATEMENTS
1. Nature of Business
Syndax Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. We currently have two commercially approved products and a robust slate of clinical development programs. We were incorporated in
We are subject to challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: revenue generation from Revuforj and Niktimvo; obtaining regulatory approval of additional indications for our approved products; delays or problems in the supply of our products, loss of single source suppliers or failure to comply with manufacturing regulations; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements.
2. Basis of Presentation
The accompanying unaudited consolidated financial statements have been prepared in conformity with U.S. generally accepted accounting principles, or U.S. GAAP. Any reference in these notes to applicable guidance is meant to refer to U.S. GAAP as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates, or ASU, of the Financial Accounting Standards Board, or FASB.
Certain prior amounts reported in the accompanying consolidated financial statements and notes thereto have been reclassified to conform to the current year presentation. Specifically, the change in royalty payable presented within the consolidated balance sheet for the period ended December 31, 2024, of $
In the opinion of management, the accompanying unaudited interim financial statements include all normal and recurring adjustments (which consist primarily of accruals and estimates that impact the financial statements) considered necessary to present fairly the Company’s financial position as of September 30, 2025 and its results of operations for the three and nine months ended September 30, 2025 and 2024 and cash flows for the nine months ended September 30, 2025 and 2024. Operating results for the three and nine months ended September 30, 2025 are not necessarily indicative of the results that may be expected for the year ending December 31, 2025. The unaudited interim financial statements presented herein do not contain the required disclosures under U.S. GAAP for annual financial statements. The accompanying unaudited interim financial statements should be read in conjunction with the annual audited financial statements and related notes as of and for the year ended December 31, 2024 contained in the Company’s annual report on Form 10-K, filed with the Securities and Exchange Commission, or SEC, on March 3, 2025.
In 2011, the Company established a wholly owned subsidiary in the United Kingdom, which the Company dissolved in June 2024. In 2014, the Company established a wholly owned U.S. subsidiary, which the Company dissolved in July 2025. In 2021, the Company established a wholly owned subsidiary in the Netherlands. To date, there have been no material activities for these entities. All intercompany balances and transactions have been eliminated in consolidation.
3. Summary of Significant Accounting Policies
The Company’s significant accounting policies, which are disclosed in the audited consolidated financial statements for the year ended December 31, 2024, and the notes thereto are included in the Company’s Annual Report on Form 10-K that was filed with the SEC on March 3, 2025. Since the date of filing, there have been no material changes to the Company’s significant accounting policies, except as noted below.
6
Use of Estimates
The preparation of the unaudited consolidated financial statements in conformity with U.S. 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 unaudited consolidated financial statements and the reported amounts of costs and expenses during the reporting period. The Company bases estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. The Company evaluates its estimates and assumptions on an ongoing basis.
Estimates and assumptions about future events and their effects cannot be determined with certainty and therefore require the exercise of judgment. As of the date of issuance of these unaudited consolidated financial statements, the Company is not aware of any specific event or circumstance that would require the Company to update its estimates, assumptions and judgments or revise the carrying value of its assets or liabilities. These estimates may change as new events occur and additional information is obtained and are recognized in the unaudited consolidated financial statements as soon as they become known. Actual results could differ from those estimates and any such differences may be material to the Company’s unaudited consolidated financial statements.
Income taxes
In accordance with Topic ASC 270, Interim Reporting, and Topic ASC 740, Income Taxes, the Company is required at the end of each interim period to determine the best estimate of its annual effective tax rate and then apply that rate in providing for income taxes on a current year-to-date (interim period) basis. For the nine months ended September 30, 2025 and 2024, the Company recorded
On July 4, 2025, the One Big Beautiful Bill Act, or OBBBA, was enacted in the United States. The OBBBA includes significant changes to the Internal Revenue Code of 1986, or the Tax Code, such as the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act of 2017, or the TCJA, modifications to certain aspects of the international tax framework and the restoration of favorable tax treatment for certain business expense provisions. The OBBBA introduced several U.S. tax law changes, including the ability to immediately expense domestic research and experimental, or R&E, expenditures starting in 2025, and an election to accelerate any unamortized domestic R&E expenditures over a one or two year period beginning with the 2025 tax year. In accordance with ASC 740, Accounting for Income Taxes, the impacts of the OBBBA did not affect the Company’s U.S. deferred tax assets or liabilities, as the Company continues to maintain a full valuation allowance against those balances.
Other comprehensive income
Comprehensive loss consists of two components, net loss and other comprehensive loss, net of tax. The Company’s other comprehensive loss, net of tax, consists of unrealized gains (losses) on the Company’s investments.
Collaboration revenue, net
In September 2021, the Company entered into the Incyte License and Collaboration Agreement, or the Incyte License, with Incyte Corporation, or Incyte, covering the worldwide development and commercialization of axatilimab. In August 2024, the U.S. Food and Drug Administration, or FDA, approved Niktimvo for the treatment of chronic graft-versus host disease, or cGVHD, after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). See Note 4 — Significant Collaborative Research and License Agreements — Incyte Collaboration for further details on the Incyte License and Collaboration Agreement.
In accordance with Topic ASC 808, Collaboration Arrangements, Incyte has been identified as the principal in product sales, therefore, the Company will recognize its share of any profits or losses in the amount of net product sales less cost of goods sold and shared commercial costs, royalties and other expenses, in the period in which the underlying sales and costs are recognized. The Company’s share of net profits in connection with commercialization of products will be presented as “Collaboration revenue, net” and its share of net losses will be presented as “Collaboration loss” within operating expenses. The year to date “Collaboration revenue, net” or “Collaboration loss” will be presented as the net cumulative amount for all periods reported on in the financial statements. The Company will continue to recognize the costs associated with ongoing development services in the R&D operating expense line, including any cost-sharing components with Incyte.
7
Recently Issued and Adopted Accounting Pronouncements
From time to time, new accounting pronouncements are issued by the FASB or other accounting standard setting bodies that we adopt as of the specified effective date. Unless otherwise discussed below, we do not believe that the adoption of recently issued standards have or may have a material impact on our unaudited consolidated financial statements or disclosures.
In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses (ASU “2024-03”). Among other items, the requirements include expanded disclosures around employee compensation and selling expenses. ASU 2024-03 will be effective for the Company for the year ending December 31, 2027. The Company is still evaluating the impact of this new guidance on its unaudited consolidated financial statements but expects the adoption to result in disclosure changes only.
4. Significant Collaborative Research and License Agreements
Incyte Collaboration
In September 2021, the Company entered into the Incyte License with Incyte, covering the worldwide development and commercialization of axatilimab. Also in September 2021, the Company entered into a share purchase agreement with Incyte, or the Incyte Share Purchase Agreement. These agreements are collectively referred to as the Incyte Agreements. Under the terms of the Incyte Agreements, Incyte received exclusive commercialization rights outside of the United States, subject to certain royalty payment obligations set forth below. In the United States, Incyte and the Company are co-commercializing and co-promoting axatilimab as Niktimvo™ (axatilimab-csfr). The Company and Incyte share equally the profits and losses from co-commercialization efforts in the United States.
The Company and Incyte have agreed to continue to co-develop axatilimab and to share development costs associated with global and additional U.S.-specific clinical trials, with Incyte responsible for
Under the terms of the Incyte Agreements, in December 2021, Incyte paid the Company a non-refundable cash payment of $
In August 2024, the FDA approved Niktimvo for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).
For the three and nine months ended September 30, 2025, the Company has recognized collaboration revenue of $
Vitae Pharmaceuticals, Inc.
In October 2017, the Company entered into a license agreement, or the Vitae License Agreement, with Vitae Pharmaceuticals, Inc., or Vitae, a subsidiary of AbbVie, Inc., or AbbVie, under which the Company was granted an exclusive, sublicensable, worldwide license to a portfolio of preclinical, orally available, small molecule inhibitors of the Menin–KMT2A binding interaction, or the Menin Assets. Upon execution of the Vitae License Agreement, the Company agreed to pay Allergan (the predecessor in interest to AbbVie) up to $
8
one-time, sales-based milestone payments based on achievement of certain annual sales thresholds. The Company is solely responsible for the development and commercialization of the Menin Assets. Each party may terminate the Vitae License Agreement for the other party’s uncured material breach or insolvency, and the Company may terminate the Vitae License Agreement at any time upon advance written notice to Vitae. Vitae may terminate the Vitae License Agreement if the Company or any of its affiliates or sublicensees institutes a legal challenge to the validity, enforceability, or patentability of the licensed patent rights. Unless terminated earlier in accordance with its terms, the Vitae License Agreement will continue on a country-by-country and product-by-product basis until the later of: (i) the expiration of all of the licensed patent rights in such country; (ii) the expiration of all regulatory exclusivity applicable to the product in such country; and (iii)
As of the date of the Vitae License Agreement, the asset acquired had no alternative future use nor had it reached a stage of technological feasibility. As the processes or activities that were acquired along with the license do not constitute a “business,” the transaction has been accounted for as an asset acquisition. In the second quarter of 2025 the Company submitted a supplemental New Drug Application to the FDA for a second indication for Revuforj. As a result, an expense of $
UCB Biopharma Sprl
In 2016, the Company entered into a license agreement, or the UCB License Agreement, as amended from time to time, with UCB Biopharma Sprl, or UCB, under which UCB granted to the Company a worldwide, sublicensable, exclusive license to UCB6352, which the Company refers to as axatilimab, an anti-CSF-1R monoclonal antibody. Upon execution of the agreement, the Company agreed to pay UCB up to $
As of the date of the UCB License Agreement, the asset acquired had no alternative future use nor had it reached a stage of technological feasibility. As the processes or activities that were acquired along with the license do not constitute a “business,” the transaction has been accounted for as an asset acquisition. As a result, in 2016, the upfront payment of $
9
5. Net Loss per Share Attributable to Common Stockholders
Basic net loss per share attributable to common stockholders is computed by dividing the net loss attributable to common stockholders by the weighted-average number of common shares outstanding for the period. Because the Company has reported a net loss for all periods presented, diluted net loss per common share is the same as basic net loss per common share for those periods.
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
|
2025 |
|
|
2024 |
|
|
2025 |
|
|
2024 |
|
||||
|
(In thousands, except share and per |
|
|
(In thousands, except share and per |
|
||||||||||
Numerator—basic and diluted: |
|
|
|
|
|
|
|
|
|
|
|
||||
Net loss |
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
Net loss attributable to common |
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
Net loss per share attributable to common |
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
Denominator—basic and diluted: |
|
|
|
|
|
|
|
|
|
|
|
||||
Weighted-average number of common shares |
|
|
|
|
|
|
|
|
|
|
|
||||
The following potentially dilutive securities have been excluded from the computation of diluted weighted-average shares outstanding because such securities have an antidilutive impact due to losses reported (in common stock equivalent shares):
|
|
September 30, |
|
|||||
|
|
2025 |
|
|
2024 |
|
||
Options to purchase common stock |
|
|
|
|
|
|
||
Employee Stock Purchase Plan |
|
|
|
|
|
|
||
Non-vested restricted stock units (RSUs) |
|
|
|
|
|
|
||
For additional information related to the Company’s common stock see Note 12 — Stock Based Compensation.
6. Other Receivables, net
In April 2024, entinostat received marketing approval in China, and as a result, the Company recorded $
7. Fair Value Measurements
The carrying amounts of cash and cash equivalents, restricted cash, accounts payable, and accrued expenses approximated their estimated fair values due to the short-term nature of these financial instruments. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. The accounting standard describes a fair value hierarchy based on three levels of inputs, of which the first two are considered observable and the last unobservable, that may be used to measure fair value, which are the following:
Level 1— Quoted prices (unadjusted) in active markets that are accessible at the market date for identical unrestricted assets or liabilities.
Level 2— Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs for which all significant inputs 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.
10
The table below presents information about the Company’s assets and liabilities that are regularly measured and carried at fair value and indicate the level within the fair value hierarchy of valuation techniques the Company utilized to determine such fair values (in thousands):
|
|
Fair Value Measurements Using |
|
|||||||||||||
|
|
|
|
|
Quoted |
|
|
|
|
|
|
|
||||
|
|
|
|
|
Prices |
|
|
Significant |
|
|
|
|
||||
|
|
|
|
|
(unadjusted) |
|
|
Other |
|
|
Significant |
|
||||
|
|
Total |
|
|
in Active |
|
|
Observable |
|
|
Unobservable |
|
||||
|
|
Carrying |
|
|
Markets |
|
|
Inputs |
|
|
Inputs |
|
||||
|
|
Value |
|
|
(Level 1) |
|
|
(Level 2) |
|
|
(Level 3) |
|
||||
|
|
(In thousands) |
|
|||||||||||||
September 30, 2025 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Assets: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash and cash equivalents |
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
||
Short-term investments |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Long-term investments |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Total assets |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
— |
|
|||
December 31, 2024 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Assets: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cash and cash equivalents |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
— |
|
|||
Short-term investments |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Long-term investments |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
Total assets |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
— |
|
|||
There have been no material impairments of our assets measured and carried at fair value during the periods ended September 30, 2025 and 2024. In addition, there have been no changes in valuation techniques during the periods ended September 30, 2025 and 2024. The fair value of Level 1 instruments classified as cash equivalents are valued using quoted market prices in active markets. The fair value of Level 2 instruments classified as short and long-term investments are determined based on quoted prices in active markets, which are either directly or indirectly observable as of the reporting date with fair value being determined using models or other valuation methodologies.
The Company’s short and long-term investments are classified as available-for-sale securities. As of September 30, 2025, the remaining contractual maturities of the available-for-sale securities were 1 to 14 months, and the balance in the Company’s accumulated other comprehensive gain was comprised solely of activity related to the Company’s available-for-sale securities. There were
The following table summarizes the available-for-sale securities:
|
|
Amortized |
|
|
Unrealized |
|
|
Unrealized |
|
|
|
|
||||
|
|
Cost |
|
|
Gains |
|
|
Losses |
|
|
Fair Value |
|
||||
|
|
(In thousands) |
|
|||||||||||||
September 30, 2025 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Commercial paper |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Corporate bonds |
|
|
|
|
|
|
|
|
— |
|
|
|
|
|||
US Treasury |
|
|
|
|
|
|
|
|
— |
|
|
|
|
|||
|
|
$ |
|
|
$ |
|
|
$ |
— |
|
|
$ |
|
|||
December 31, 2024 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Commercial paper |
|
$ |
|
|
$ |
— |
|
|
$ |
( |
) |
|
$ |
|
||
Corporate bonds |
|
|
|
|
|
— |
|
|
|
— |
|
|
|
|
||
US Treasury |
|
|
|
|
|
|
|
|
— |
|
|
|
|
|||
|
|
$ |
|
|
$ |
|
|
$ |
( |
) |
|
$ |
|
|||
11
8. Prepaid Expenses and Other Current Assets
Prepaid expenses and other current assets consisted of the following (in thousands):
|
|
September 30, 2025 |
|
|
December 31, 2024 |
|
||
Prepaid insurance |
|
$ |
|
|
$ |
|
||
Interest receivable on investments |
|
|
|
|
|
|
||
Prepaid subscription |
|
|
|
|
|
|
||
Prepaid state and local taxes |
|
|
|
|
|
|
||
Prepaid rent |
|
|
|
|
|
|
||
Prepaid inventory |
|
|
|
|
|
|
||
Other |
|
|
|
|
|
|
||
Total prepaid expenses and other current assets |
|
$ |
|
|
$ |
|
||
9. Inventory
Inventory consisted of the following (in thousands):
|
|
September 30, 2025 |
|
|
December 31, 2024 |
|
||
Raw materials |
|
$ |
|
|
$ |
|
||
Work-in-process |
|
|
|
|
|
|
||
Finished goods |
|
|
|
|
|
|
||
Total Inventory |
|
$ |
|
|
$ |
|
||
Inventories are stated at the lower of cost or net realizable value, as determined on a first-in, first-out basis.
10. Accrued Expenses and Other Current Liabilities
Accrued expenses and other current liabilities consisted of the following (in thousands):
|
|
September 30, 2025 |
|
|
December 31, 2024 |
|
||
Product revenue allowances |
|
$ |
|
|
$ |
|
||
Accrued clinical study and trial costs |
|
|
|
|
|
|
||
Accrued selling, general, and administrative costs |
|
|
|
|
|
|
||
Accrued compensation and related costs |
|
|
|
|
|
|
||
Accrued professional fees |
|
|
|
|
|
|
||
Accrued milestone costs |
|
|
|
|
|
|
||
Accrued royalty payable |
|
|
|
|
|
|
||
Accrued royalty interest financing expense |
|
|
|
|
|
|
||
Other |
|
|
|
|
|
|
||
Total accrued expenses and other current liabilities |
|
$ |
|
|
$ |
|
||
11. Royalty Interest Financing Liability
On November 4, 2024, the Company entered into a Purchase and Sale Agreement, or the Purchase and Sale Agreement, with Royalty Pharma Development Funding, LLC, or Royalty Pharma, pursuant to which Royalty Pharma purchased rights to certain revenue streams from net sales of products comprising or containing axatilimab (including Niktimvo™) by the Company, its affiliates and its licensees in the United States and its respective territories, districts, commonwealths and possessions (including Guam and Puerto Rico) in exchange for an upfront fee of $
Pursuant to the Purchase and Sale Agreement, Royalty Pharma purchased the right to receive a percentage of net sales equal to a royalty rate of
The Purchase and Sale Agreement contains customary representations, warranties and indemnities of the Company and Royalty Pharma and customary covenants relating to the royalty payments, including the grant of a back-up security interest in the purchased royalties and certain assets related to the product and restrictions on the incurrence of additional indebtedness and on the existence of liens on the Company’s assets related to the product.
12
Upon a change of control, the Company will have the right, but not the obligation, to repurchase the Revenue Participation Right at a repurchase price set forth in the Purchase and Sale Agreement. In addition, the Purchase and Sale Agreement provides that if certain events of default occur, including certain bankruptcy events or certain termination events with respect to the Company’s license agreement with UCB Biopharma Srl, Royalty Pharma may require the Company to repurchase Royalty Pharma’s interests in the Revenue Participation Right at a repurchase price equal to the Royalty Cap.
The Company assessed the Purchase and Sale Agreement and identified it as a sale of future revenue in the form of a debt instrument to be accounted for as a liability under Topic ASC 470, Borrower’s Accounting for Debt Modifications. The Company has elected to use the prospective method in its calculation of its effective interest rate and will update this calculation quarterly when there are changes in the projected sales. The debt is allocated on the balance sheet as short term and long term. The short term portion represents the royalty payments owed over the next 12 months. The long term portion is recorded on the balance sheet as net of issuance costs. Issuance costs pursuant to the Purchase and Sale Agreement consisted primarily of bank and legal fees and totaled $
|
|
September 30, 2025 |
|
|
December 31, 2024 |
|
||
Current portion of royalty interest financing liability |
|
$ |
|
|
$ |
|
||
Royalty interest financing liability, less current portion |
|
|
|
|
|
|
||
Debt issuance costs |
|
|
( |
) |
|
|
( |
) |
Total royalty interest financing liability, net |
|
$ |
|
|
$ |
|
||
12. Stock-Based Compensation
In January 2025, the number of shares of common stock available for issuance under the Company’s 2015 Omnibus Incentive Plan, or the 2015 Plan, was increased by
As of September 30, 2025, there were
The Company recognized stock-based compensation expense related to the issuance of stock option awards and restricted stock units to employees and non-employees and related to the Company’s 2015 Employee Stock Purchase Plan, or ESPP, in the consolidated statements of comprehensive loss as follows:
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
|
2025 |
|
|
2024 |
|
|
2025 |
|
|
2024 |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
||||
Research and development |
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Selling, general and administrative |
|
|
|
|
|
|
|
|
|
|
|
||||
Total |
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Compensation expense by type of award in the three and nine months ended September 30, 2025 and 2024 was as follows:
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
|
2025 |
|
|
2024 |
|
|
2025 |
|
|
2024 |
|
||||
|
|
|
|
|
|
|
|
|
|
|
|
||||
Stock options |
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
RSUs |
|
|
|
|
|
|
|
|
|
|
|
||||
ESPP |
|
|
|
|
|
|
|
|
|
|
|
||||
Total |
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
13
In addition, stock-based compensation expense of $
As of September 30, 2025, there were $
Employee Benefit Plan
The Company maintains a defined contribution 401(k) retirement plan. For the three and nine months ended September 30, 2025, the Company made $
13. Stockholders’ Equity
Pre-Funded Warrants
In December 2021, the Company sold pre-funded warrants to purchase
14. Commitments and Contingencies
License Agreements
The Company is obligated to pay royalties pursuant to the Vitae License Agreement and the UCB License Agreement as a percentage of net product sales for direct licensed products, such as Revuforj and Niktimvo. The obligation to pay royalties expires, on a country-by-country basis and licensed product-by-licensed product basis at the later of (i) the expiration of all of the licensed patent rights in such country; (ii) the expiration of all regulatory exclusivity applicable to the product in such country; and (iii) 10 years from the date of the first commercial sale of the product in such country. These fees were recorded as cost of product sales.
15. Segment Reporting
The Company manages its business activities on a consolidated basis and operate as a single operating and reportable segment: Syndax Pharmaceuticals. The Company primarily derives revenue in the United States through milestone revenue and product sales on the approved products, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr). The accounting policies of the segment are the same as those described in Note 3 – Summary of Significant Accounting Policies.
To assess performance, the Company’s Chief Operating Decision Maker, or CODM, Michael Metzger, uses consolidated net loss as the segment’s measure of segment profit or loss. The CODM uses net loss in the budget and forecasting process and considers budget-to actual variances on a quarterly basis when making decisions about the allocation of operating and capital resources.
14
The following table provides the operating financial results of our biopharmaceutical cancer therapeutics segment:
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
||||||||||
|
|
2025 |
|
|
2024 |
|
|
2025 |
|
|
2024 |
|
||||
September 30, 2025 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Total Revenue |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
Less: Significant and other segment expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cost of product sales |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Research and development expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Revumenib-related costs |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Axatilimab-related costs |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Other R&D programs |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Personnel cost and other expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
General and administrative expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Commercial-related expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Personnel cost and other expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Other SG&A expenses |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Stock-based compensation |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Royalty interest expense |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Interest (income) expense, net |
|
|
( |
) |
|
|
( |
) |
|
|
( |
) |
|
|
( |
) |
Other expense (income), net |
|
|
|
|
|
( |
) |
|
|
|
|
|
|
|||
Segment net loss |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
16. Subsequent Events
On October 24, 2025, the Company announced FDA approval of Revuforj® (revumenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. As a result of the approval, a development and regulatory milestone of $
15
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
The following information should be read in conjunction with the unaudited financial information and the notes thereto included in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K that was filed with the Securities and Exchange Commission, or SEC, on March 3, 2025.
Company Overview
We are a commercial-stage biopharmaceutical company advancing innovative cancer therapies. We currently have two commercially approved products, Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr), and a robust slate of clinical development programs. Revuforj is our first-in-class menin inhibitor that was approved by the U.S. Food and Drug Administration, or FDA, in November 2024 for the treatment of relapsed or refractory, or R/R, acute leukemia with a lysine methyltransferase 2A gene, or KMT2A, translocation in adult and pediatric patients one year and older. In October 2025, Revuforj received a second approval from the FDA for the treatment of R/R acute myeloid leukemia, or AML, with a susceptible nucleophosmin 1 mutation, or NPM1m, in adult and pediatric patients one year and older who have no satisfactory alternative treatment option. We are also studying revumenib in combination with standard-of-care agents in NPM1m AML or KMT2A-rearranged acute leukemia across the treatment landscape, including in newly diagnosed patients. Additionally, we are exploring the use of revumenib as a treatment in solid tumors, specifically its activity in metastatic colorectal cancer. Niktimvo is our first-in-class colony stimulating factor-1 receptor, or CSF-1R, blocking antibody that was approved by the FDA in August 2024 for the treatment of chronic graft-versus-host disease, or cGVHD, after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Axatilimab is in development for the treatment of newly diagnosed cGVHD patients in combination with standard-of-care therapies, as well for the treatment of idiopathic pulmonary fibrosis, or IPF. We plan to continue to leverage the technical and business expertise of our management team and scientific collaborators to license, acquire and develop additional therapeutics to expand our pipeline.
We have begun generating product revenue from sales of Revuforj and collaboration revenue from sales of Niktimvo. We continue to incur significant research and development and other expenses related to our ongoing operations. Except for 2021, we have not been profitable and have incurred losses in each period since our inception in 2005. For the nine months ended September 30, 2025 and 2024, we reported a net loss of $217.4 million and $224.6 million, respectively. As of September 30, 2025, we had an accumulated deficit of $1.4 billion. As of September 30, 2025, we had cash, cash equivalents and short- and long-term investments of $456.1 million.
Business Update
Revuforj® (revumenib)
16
Niktimvo™ (axatilimab-csfr)
Financial Operations Overview
Product Revenue, net
Our second FDA-approved product, Revuforj, was approved by the FDA for commercial sale in the U.S. on November 15, 2024. In accordance with GAAP, we determine net product revenue for Revuforj, with specific assumptions for variable consideration components including, but not limited to, trade discounts and allowances, co-pay assistance programs and payor rebates.
We generated no product revenue during the three and nine months ended September 30, 2024.
Collaboration revenue, net
In September 2021, we entered into the Incyte License and Collaboration Agreement, or the Incyte License, with Incyte covering the worldwide development and commercialization of axatilimab. In August 2024, the FDA approved Niktimvo for the
17
treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).
In accordance with Topic ASC 808, Collaboration Arrangements, Incyte has been identified as the principal in product sales, therefore, we will recognize its 50% share of any profits or losses in the amount of net product sales less cost of goods sold and shared commercial and other expenses, in the period in which the underlying sales and costs are recognized. Our share of net profits in connection with commercialization of Niktimvo will be presented as “Collaboration revenue, net” and our share of net losses will be presented as “Collaboration loss” within operating expenses. Collaboration revenue or expense is made up of our share of the 50% profit with Incyte. We record collaboration revenue net of commercial expenses, including any royalties owed on license agreements. We will continue to recognize the costs associated with ongoing development services in the R&D operating expense line, including any cost-sharing components with Incyte.
Cost of Product Sales
Our cost of product sales includes the cost of goods sold and royalties associated with Revuforj sales in the United States.
Research and Development
Since our inception, we have primarily focused on our clinical development programs. Research and development expenses consist primarily of costs incurred for the development of our product candidates and include:
Internal and external research and development costs are expensed as they are incurred. Cost-sharing amounts received by us are recorded as reductions to research and development expense. Costs for certain development activities, such as clinical trials, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations or other information provided to us by our vendors.
Research and development activities are central to our business model. Product candidates in late 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 late-stage clinical trials. We plan to continue to spend a significant amount of our resources on research and development activities for the foreseeable future as we continue to advance the development of our product candidates. The amount of research and development expenses allocated to external spending will continue to grow, while we expect our internal spending to grow at a slower and more controlled pace.
It is difficult to determine, with certainty, the duration and completion costs of our current or future preclinical programs, research studies and clinical trials of our product candidates. The duration, costs and timing of research studies and clinical trials of our product candidates will depend on a variety of factors that include, but are not limited to, the following:
18
In addition, the probability of success for each product candidate will depend on numerous factors, including competition, manufacturing capability and commercial viability. The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate the nature, timing or costs of the efforts that will be necessary to complete the remainder of the development of our product candidates for the period, if any, in which material net cash inflows from these potential drug candidates may commence. Clinical development timelines, the probability of success and development costs can differ materially from expectations.
Selling, General and Administrative
Selling, general and administrative expenses consist primarily of commercialization costs as well as employee-related expenses, including salaries, benefits, non-cash stock-based compensation and travel expenses, for our employees in executive, finance, human resources, information technology, business development and support functions, as well as sales and marketing expenses to support the launch and commercialization of Revuforj and Niktimvo. Other selling, general and administrative expenses include facility-related costs not otherwise allocated to research and development expenses and accounting, tax, legal, information technology and consulting services. We anticipate that our selling, general and administrative expenses will remain stable in the next few years.
Royalty Interest Expense
Royalty interest expense consists of interest expense related to the Purchase and Sale Agreement with Royalty Pharma.
Other Interest Expense
Other interest expense consists of interest expense related to our operational and capital leases.
Interest Income
Interest income consists of income earned on our cash, cash equivalents and short- and long-term investment balances.
Other Expense
Other expense includes expense consisting of revaluation of foreign currency related to trade payables and amortization of debt issuance costs.
Recent Accounting Pronouncements
For a discussion of new accounting pronouncements please read Note 3 - Summary of Significant Accounting Policies to our unaudited consolidated financial statements included in this Quarterly Report.
Critical Accounting Policies and Estimates
Our management’s discussion and analysis of financial condition and results of operations are based on our financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States, or U.S. GAAP. The preparation of these financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenue and expenses and related disclosures of contingent assets and liabilities. We base our estimates on historical experience, known trends and events and various other factors that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. In making estimates and judgments, management employs critical accounting policies.
There have been no material changes to our critical accounting estimates described in the “Management’s Discussion and Analysis of Financial Condition and Results of Operations” contained in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.
19
Results of Operations
Comparison of the three and nine months ended September 30, 2025 and 2024:
|
|
Three Months Ended September 30, |
|
|
Change |
|
|
Nine Months Ended September 30, |
|
|
Change |
|
||||||||||||
|
|
2025 |
|
|
2024 |
|
|
$ |
|
|
2025 |
|
|
2024 |
|
|
$ |
|
||||||
|
|
(In thousands) |
|
|
(In thousands) |
|
||||||||||||||||||
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Product revenue, net |
|
$ |
32,007 |
|
|
$ |
— |
|
|
$ |
32,007 |
|
|
$ |
80,649 |
|
|
$ |
— |
|
|
|
80,649 |
|
Collaboration revenue, net |
|
|
13,864 |
|
|
|
— |
|
|
|
13,864 |
|
|
|
22,975 |
|
|
|
— |
|
|
|
22,975 |
|
Milestone and license revenue |
|
|
— |
|
|
|
12,500 |
|
|
|
(12,500 |
) |
|
|
— |
|
|
|
16,000 |
|
|
|
(16,000 |
) |
Total revenues |
|
|
45,871 |
|
|
|
12,500 |
|
|
|
33,371 |
|
|
|
103,624 |
|
|
|
16,000 |
|
|
|
87,624 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Cost of goods sold |
|
$ |
2,100 |
|
|
$ |
— |
|
|
$ |
2,100 |
|
|
$ |
4,264 |
|
|
$ |
— |
|
|
|
4,264 |
|
Research and development |
|
|
56,280 |
|
|
|
70,971 |
|
|
|
(14,691 |
) |
|
|
180,143 |
|
|
|
176,118 |
|
|
|
4,025 |
|
Selling, general and administrative |
|
|
44,917 |
|
|
|
31,106 |
|
|
|
13,811 |
|
|
|
129,753 |
|
|
|
83,189 |
|
|
|
46,564 |
|
Total operating expenses |
|
|
103,297 |
|
|
|
102,077 |
|
|
|
1,220 |
|
|
|
314,160 |
|
|
|
259,307 |
|
|
|
54,853 |
|
Loss from operations |
|
|
(57,426 |
) |
|
|
(89,577 |
) |
|
|
(32,151 |
) |
|
|
(210,536 |
) |
|
|
(243,307 |
) |
|
|
(32,771 |
) |
Other (expense) income, net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Royalty interest expense |
|
|
(8,283 |
) |
|
|
— |
|
|
|
(8,283 |
) |
|
|
(24,186 |
) |
|
|
— |
|
|
|
(24,186 |
) |
Other interest expense |
|
|
— |
|
|
|
(23 |
) |
|
|
23 |
|
|
|
(6 |
) |
|
|
(123 |
) |
|
|
117 |
|
Interest income |
|
|
5,269 |
|
|
|
5,442 |
|
|
|
(173 |
) |
|
|
18,402 |
|
|
|
18,982 |
|
|
|
(580 |
) |
Other (expense) income |
|
|
(275 |
) |
|
|
32 |
|
|
|
(307 |
) |
|
|
(1,082 |
) |
|
|
(141 |
) |
|
|
(941 |
) |
Total other (expense) income, net |
|
|
(3,289 |
) |
|
|
5,451 |
|
|
|
(8,740 |
) |
|
|
(6,872 |
) |
|
|
18,718 |
|
|
|
(25,590 |
) |
Net loss |
|
$ |
(60,715 |
) |
|
$ |
(84,126 |
) |
|
$ |
(23,411 |
) |
|
$ |
(217,408 |
) |
|
$ |
(224,589 |
) |
|
$ |
(7,181 |
) |
Product Revenue, net
In November 2024, we began to generate product revenue from sales of Revuforj in the United States. We record product revenue net of estimated discounts, chargebacks, rebates, product returns, and other gross-to-net revenue deductions. Product revenue, net from sales of Revuforj was $32.0 million and $80.6 million for the three and nine months ended September 30, 2025, respectively.
Collaboration Revenue, net
In February 2025, we began to generate sales of Niktimvo in the United States in collaboration with our partner, Incyte. Our Collaboration revenue, net was $13.9 million for the three months ended September 30, 2025. Our Collaboration revenue, net was $23.0 million for the nine months ended September 30, 2025, and includes the $0.2 million of Collaboration loss recognized in the first quarter.
Cost of Product Sales
Our cost of product sales were $2.1 million and $4.3 million for the three and nine months ended September 30, 2025, respectively, resulting from increased sales of Revuforj. Included in cost of product sales are royalties owed to AbbVie on Revuforj sales as part of the Vitae License Agreement.
Research and Development
The following table summarizes the research and development expenses for the three and nine months ended September 30, 2025 and 2024:
20
|
|
Three Months Ended September 30, |
|
|
Change |
|
|
Nine Months Ended September 30, |
|
|
Change |
|
||||||||||||
|
|
2025 |
|
|
2024 |
|
|
$ |
|
|
2025 |
|
|
2024 |
|
|
$ |
|
||||||
|
|
(In thousands) |
|
|
(In thousands) |
|
||||||||||||||||||
Revumenib-related costs |
|
$ |
21,747 |
|
|
$ |
24,454 |
|
|
$ |
(2,707 |
) |
|
$ |
70,707 |
|
|
$ |
76,421 |
|
|
$ |
(5,714 |
) |
Axatilimab-related costs |
|
|
9,447 |
|
|
|
24,204 |
|
|
|
(14,757 |
) |
|
|
38,211 |
|
|
|
38,309 |
|
|
|
(98 |
) |
Other research and development programs |
|
|
694 |
|
|
|
669 |
|
|
|
25 |
|
|
|
3,441 |
|
|
|
2,169 |
|
|
|
1,272 |
|
Personnel cost and other expenses |
|
|
20,114 |
|
|
|
15,807 |
|
|
|
4,307 |
|
|
|
55,642 |
|
|
|
44,922 |
|
|
|
10,720 |
|
Stock-based compensation |
|
|
4,278 |
|
|
|
5,837 |
|
|
|
(1,559 |
) |
|
|
12,142 |
|
|
|
14,297 |
|
|
|
(2,155 |
) |
Total research and development expenses |
|
$ |
56,280 |
|
|
$ |
70,971 |
|
|
$ |
(14,691 |
) |
|
$ |
180,143 |
|
|
$ |
176,118 |
|
|
$ |
4,025 |
|
For the three months ended September 30, 2025, our total research and development expenses decreased by $14.7 million from the comparable prior year period. The decrease was primarily due to:
For the nine months ended September 30, 2025, total research and development expenses increased $4.0 million from the comparable prior year period. The increase was primarily due to:
Selling, General and Administrative
The following tables summarizes the selling, general and administrative expenses for the three and nine months ended September 30, 2025 and 2024:
|
|
Three Months Ended September 30, |
|
|
Change |
|
|
Nine Months Ended September 30, |
|
|
Change |
|
||||||||||||
|
|
2025 |
|
|
2024 |
|
|
$ |
|
|
2025 |
|
|
2024 |
|
|
$ |
|
||||||
|
|
(In thousands) |
|
|
(In thousands) |
|
||||||||||||||||||
Commercial related expenses |
|
$ |
12,210 |
|
|
$ |
7,620 |
|
|
$ |
4,590 |
|
|
$ |
32,949 |
|
|
$ |
21,012 |
|
|
$ |
11,937 |
|
Other selling, general and administrative expenses |
|
|
9,106 |
|
|
|
3,854 |
|
|
|
5,252 |
|
|
|
18,511 |
|
|
|
12,111 |
|
|
|
6,400 |
|
Personnel cost and other expenses |
|
|
15,797 |
|
|
|
13,535 |
|
|
|
2,262 |
|
|
|
54,186 |
|
|
|
33,635 |
|
|
|
20,551 |
|
Stock-based compensation |
|
|
7,804 |
|
|
|
6,097 |
|
|
|
1,707 |
|
|
|
24,107 |
|
|
|
16,431 |
|
|
|
7,676 |
|
Total selling, general and administrative expenses |
|
$ |
44,917 |
|
|
$ |
31,106 |
|
|
$ |
13,811 |
|
|
$ |
129,753 |
|
|
$ |
83,189 |
|
|
$ |
46,564 |
|
For the three and nine months ended September 30, 2025, our total selling, general and administrative expenses increased $13.8 million and $46.6 million, respectively, from the comparable prior year period. The increases were primarily due to commercial-related costs, driven by ongoing activities in 2025 associated with the commercial launches for Revuforj and Niktimvo. The increases in personnel and stock-based compensation costs were primarily related to higher costs in headcount in 2025 to support the commercialization of Revuforj and Niktimvo.
21
Royalty Interest Expense
For the three and nine months ended September 30, 2025, royalty interest expense increased due to the interest expense recognized related to the Royalty Pharma Purchase and Sale Agreement signed in November 2024.
Other Interest Expense
For the three and nine months ended September 30, 2025, other interest expense decreased from the comparable period due to less interest expense recognized related to capital leases.
Interest Income
For the three and nine months ended September 30, 2025, interest income decreased from the comparable period. The decrease of interest income was primarily due to the fluctuation of interest rates and the average balance of cash equivalents and short and long-term investments.
Other Expense
For the three and nine months ended September 30, 2025, other expense increased from the comparable period primarily related to the current period amortization of debt issuance costs related to the Royalty Pharma Purchase and Sale Agreement.
Liquidity and Capital Resources
Cash Flows
The following table sets forth the primary sources and uses of cash and cash equivalents for each period set forth below:
|
|
Nine Months Ended September 30, |
|
|||||
|
|
2025 |
|
|
2024 |
|
||
|
|
(In thousands) |
|
|||||
Net cash used in operating activities |
|
$ |
(253,492 |
) |
|
$ |
(217,515 |
) |
Net cash provided by investing activities |
|
|
208,458 |
|
|
|
49,198 |
|
Net cash provided by financing activities |
|
|
7,759 |
|
|
|
5,942 |
|
Net decrease cash, cash equivalents and restricted cash |
|
$ |
(37,275 |
) |
|
$ |
(162,375 |
) |
Net Cash Used in Operating Activities
Net cash used in operating activities increased from $217.5 million for the nine months ended September 30, 2024 to $253.5 million for the nine months ended September 30, 2025, primarily due to an increase in accounts receivable, inventory, collaboration receivable, and short term deposits, and a decrease in collaboration payable.
Net Cash Provided by Investing Activities
Net cash provided by investing activities for the nine months ended September 30, 2025, was $208.5 million and was related to $341.3 million from the maturities of available-for-sale securities, offset by the purchase of $132.7 million of available-for-sale securities.
Net cash provided by investing activities for the nine months ended September 30, 2024, was $49.2 million and was related to $229.9 million from the maturities of available-for-sale securities, offset by the purchase of $180.7 million of available-for-sale securities.
Net Cash Provided by Financing Activities
Net cash provided by financing activities for the nine months ended September 30, 2025, increased by $1.8 million from the comparable prior year period primarily due to an increase in proceeds from the exercise of stock options in the current period.
Purchase and Sale Agreement
In October 2024, we entered into a purchase and sale agreement with Royalty Pharma, pursuant to which Royalty Pharma purchased the right to receive 13.8% on quarterly net sales of Niktimvo in the United States and its respective territories, districts, commonwealths and possessions (including Guam and Puerto Rico) in exchange for an upfront payment of $350.0 million (gross) at closing, received in November 2024. Aggregate payments to Royalty Pharma pursuant to the Royalty Agreement will be capped at $822.5 million or 2.35 times the funded amount.
22
For additional details on our purchase and sale agreement with Royalty Pharma, see Note 11 - “Royalty Interest Financing Liability” to our consolidated financial statements in this Quarterly Report.
Future Funding Requirements
We believe that the combination of our available cash, cash equivalents, short-term and long-term investments, as well as our expected Revuforj gross contribution and Niktimvo collaboration revenue, is sufficient to fund existing and planned cash requirements. Our primary uses of capital are, and we expect will continue to be, compensation and related expenses, third-party clinical research and development services, clinical costs, commercialization costs, legal and other regulatory expenses and general overhead costs. We have based our estimates on assumptions that may prove to be incorrect, and we could use our capital resources sooner than we currently expect.
Additionally, the process of testing product candidates in clinical trials is costly, and the timing, progress and outcomes in these trials is uncertain. We cannot estimate the actual amounts necessary to successfully complete the development and commercialization of our product candidates or whether, or when, we may achieve profitability.
Our future capital requirements will depend on many factors, including:
We expect to continue to support our future cash needs through a combination of equity offerings, debt financings and additional funding from license and collaboration arrangements. Except for any obligations of our collaborators to reimburse us for research and development expenses or to make milestone or royalty payments under our agreements with them, we will not have any committed external source of liquidity.
Our material contractual obligations and commitments as of September 30, 2025, primarily relate to our maturities of operating leases for office space and equipment and capital leases for office equipment. As of September 30, 2025, we have $0.3 million payable within 12 months.
Except as disclosed above, we have no material non-cancelable purchase commitments with service providers, as we have generally contracted on a cancelable, purchase-order basis. We enter into contracts in the normal course of business with equipment and reagent vendors, CROs, contract manufacturing organizations, and other third parties for clinical trials, preclinical research studies and testing and manufacturing services. These contracts are cancelable by us upon prior notice. Payments due upon cancellation
23
consist only of payments for services provided or expenses incurred, including noncancelable obligations of our service providers, up to the date of cancellation. These payments are not determinable.
We have incurred losses and cumulative negative cash flows from operations since our inception, excluding the year ended December 31, 2021. As of September 30, 2025, we had an accumulated deficit of $1.4 billion. We anticipate that we will likely continue to incur significant losses for at least the next couple years. To the extent that we raise additional capital through the future sale of equity or debt, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing common stockholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
At-the-Market Offering Program
In May 2023, we entered into a sales agreement with Cowen and Company, LLC, or TD Cowen, under which we could, from time to time, issue and sell shares of our common stock having aggregate sales proceeds of up to $200.0 million, in a series of one or more at-the-market equity offerings, or the 2023 ATM Program. TD Cowen is not required to sell any specific share amounts but acts as the Company’s sales agent, using commercially reasonable efforts consistent with its normal trading and sales practices. Pursuant to the sales agreement, shares will be sold under the shelf registration statement on Form S-3ASR (Registration No. 333-277424), which became automatically effective upon filing on February 27, 2024. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. For the three and nine months ended September 30, 2025, we did not sell any shares of common stock under the 2023 ATM Program. As of September 30, 2025, we had $157.9 million available under the 2023 ATM Program.
Significant Risks and Uncertainties
Unfavorable interest rates and geo-political unrest could result in further economic uncertainty and volatility in the capital markets in the near term and, as a result, could negatively affect our operations and could make it difficult for us to obtain traditional financing on acceptable terms, if at all. Furthermore, such economic conditions have produced downward pressure on share prices. Although we do not believe that inflation has had a material impact on our financial position or results of operations to date, the return of a high inflationary environment could increase our operating costs, including our labor costs and research and development costs. These costs may also be negatively impacted due to supply chain constraints, geopolitical tensions, including tariffs, the ongoing wars between Russia and Ukraine and Israel and Hamas as well as other conflicts in the Middle East, including between Israel and Hezbollah, worsening macroeconomic conditions and employee availability and wage increases, which may result in additional stress on our working capital.
Additionally, we are subject to other challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of additional indications for our approved products; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements. See the section titled “Risk Factors” located elsewhere in this report for additional information.
Item 3. Quantitative and Qualitative Disclosures about Market Risk
The market risk inherent in our financial instruments and in our financial position represents the potential loss arising from adverse changes in interest rates. As of September 30, 2025, we had cash and cash equivalents of $116.8 million, consisting of overnight investments, interest-bearing money market funds and commercial paper, and short and long-term investments of $339.3 million, consisting of commercial paper, highly rated corporate bonds and treasuries. Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates. The primary objectives of our investment activities are to ensure liquidity and to preserve principal while at the same time maximizing the interest income, we receive from our marketable securities without significantly increasing risk. We have established guidelines regarding approved investments and maturities of investments, which are designed to maintain safety and liquidity. Due to the relative short-term maturities of our cash equivalents and the low risk profile of our short and long-term investments, an immediate 100 basis point change in interest rates would not have a material effect on the fair market value of our cash equivalents and short and long-term investments. We have the
24
ability to hold our investments until maturity, and therefore, we would not expect our operating results or cash flows to be affected to any significant degree by the effect of a change in market interest rates on our investment portfolio.
We do not believe that inflation and changing prices had a significant impact on our results of operations for any periods presented herein.
Item 4. Controls and Procedures
Disclosure Controls and Procedures and Internal Control over Financial Reporting
Controls and Procedures
We have carried out an evaluation, under the supervision and with the participation of our management, including our principal executive officer and principal financial officer, of the effectiveness of the design and operation of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act), as of September 30, 2025. Based upon that evaluation, our principal executive officer and principal financial officer concluded that, as of the end of the period covered by this report, our disclosure controls and procedures are effective in ensuring that:
In designing and evaluating our disclosure controls and procedures, our management recognized that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and our management necessarily was required to apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures.
Changes in Internal Control over Financial Reporting
There was no change in our internal control over financial reporting during the quarter ended September 30, 2025, that has materially affected, or is reasonably likely to materially affect, our internal control over financial reporting.
25
PART II. OTHER INFORMATION
Item 1. Legal Proceedings
In the ordinary course of business, we are from time to time involved in lawsuits, claims, investigations, proceedings, and threats of litigation relating to intellectual property, commercial arrangements and other matters. While the outcome of these proceedings and claims cannot be predicted with certainty, as of September 30, 2025, we were not party to any material legal or arbitration proceedings. No governmental proceedings are pending or, to our knowledge, contemplated against us.
Item 1A. Risk Factors
In addition to the other information contained elsewhere in this report, you should carefully consider the risks and uncertainties described in “Part I, Item 1A—Risk Factors” of our Annual Report on Form 10-K for the year ended December 31, 2024, or 2024 Form 10-K, filed with the Securities and Exchange Commission, or SEC, on March 3, 2025, and in “Part II, Item 1A—Risk Factors” of our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, or Q1 2025 Form 10-Q, filed with the SEC on May 5, 2025, which could materially and adversely affect our business, prospects, financial condition and results of operations. New risk factors can emerge from time to time, and it is not possible to predict the impact that any factor or combination of factors may have on our business, prospects, financial condition and results of operations. The risk factors disclosure in our 2024 Form 10-K and Q1 2025 Form 10-Q is qualified by the information that is described in this Quarterly Report on Form 10-Q. The risks described in our 2024 Form 10-K and Q1 2025 Form 10-Q are not our only risks. Additional risks and uncertainties not currently known to us or that we currently deem to be immaterial also may materially adversely affect our business, financial condition or future results. There have been no material changes in our risk factors previously disclosed in our 2024 Form 10-K and Q1 2025 Form 10-Q, except as indicated below.
Our products and product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit the commercial scope of their approved use, or result in significant negative consequences following any marketing approval.
Undesirable side effects caused by our products and product candidates could cause the interruption, delay or halting of the trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other foreign regulatory authorities. For example, the Revuforj U.S. Prescribing Information label contains a boxed warning for differentiation syndrome, which can be fatal, as well as QTc prolongation and Torsades de Pointes. Results of the clinical trials may reveal a high and unacceptable severity and prevalence of side effects or other unexpected characteristics. In such event, the trials could be suspended or terminated, or the FDA or foreign regulatory authorities could deny approval of our product candidates for any or all targeted indications. Drug-related side effects could affect patient recruitment or the ability of enrolled subjects to complete the trial or result in potential product liability claims. Any of these occurrences may harm our business, financial condition and prospects.
Additionally, if our product candidates receive marketing approval, and we or others later identify undesirable side effects, a number of potentially significant negative consequences could result, including:
Any of these events could prevent us from achieving or maintaining market acceptance of our product candidates for use in targeted indications or otherwise materially harm its commercial prospects, if approved, and could harm our business, results of operations and prospects.
We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
26
Revuforj, Niktimvo and any product candidate that receives approval in the future would face significant competition from other therapies in the relevant indication. For example, cGVHD has historically been managed by off-label treatments. However, in the past several years, the FDA has approved three drugs, ibrutinib (Imbruvica®), belomosidil (Rezurock®) and ruxolitinib (Jakafi®), for use in patients with cGVHD after failure of one or more lines of systemic therapy. All three of these drugs may compete with Niktimvo in patients diagnosed with cGVHD.
We initially developed revumenib for the treatment of R/R adult and pediatric patients with KMT2Ar ALL, KMT2Ar AML and NPM1 mutant AML. At this time, other than Revuforj, there are no drugs approved for these defined populations and patients are managed using the standard of care treatment regimens developed for general AML and ALL populations. However, there are other agents in development for similar populations that may in the future be approved for these defined populations and that may prove to be more effective than Revuforj. We are also advancing a pipeline of frontline trials of revumenib in combination with standard of care therapies to support potential additional listings in the National Comprehensive Cancer Network, or NCCN, Guidelines® and/or label expansion opportunities.
Existing or potential competitors have or may have substantially greater financial, technical and human resources than we do and significantly greater experience in the discovery and development of product candidates, obtaining FDA and other regulatory approvals of product candidates and the commercialization of those products. Our competitors may be more successful than us in obtaining FDA approval for drugs and achieving widespread market acceptance. Our competitors’ drugs may be more effective or more effectively marketed and sold than any drug we may commercialize and may render our product candidates obsolete or non-competitive before we can recover the expenses of developing and commercializing any of our product candidates. Our competitors may also obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours. We anticipate that we will face intense and increasing competition as new drugs enter the market and advanced technologies become available.
We believe that our ability to successfully compete will depend on, among other things:
Even if we obtain regulatory approval of our other product candidates, the availability, commercial formulary placement, and price of our competitors’ products could limit the demand and the price we are able to charge. We may not be able to implement our business plan if the acceptance of our product candidates is inhibited by price competition or the reluctance of physicians to switch from existing methods of treatment, or if physicians switch to other new drug or biologic products or choose to reserve our drugs for use in limited circumstances.
Off-label use or misuse of our products may harm our reputation in the marketplace or result in injuries that lead to costly product liability suits.
We have received regulatory approval to market Revuforj in the U.S. for the treatment of (i) R/R acute leukemia with a KMT2A translocation in adult and pediatric patients one year old and older and (ii) R/R AML with a susceptible NPM1 mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options, and regulatory approval to market Niktimvo for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. We may only promote or market Revuforj, Niktimvo, and our other product candidates, if approved by the FDA, for their specifically approved indications and in a manner consistent with the approved labeling. We train our marketing and sales force against promoting our product candidates for uses outside of the approved indications for use, known as “off-label uses.” We cannot, however, prevent a physician from using our products off-label, when in the physician’s independent professional medical judgment, he or she deems it appropriate. Furthermore, the use of our products for indications other than those approved by the FDA may not effectively treat such conditions. Any such off-label use of our product candidates could harm our reputation in the marketplace among physicians and patients. There may also be increased risk of injury to patients if physicians attempt to use our products for any off-label uses, which could lead to product liability lawsuits that might require significant financial and management resources and that could harm our reputation. Additionally, the FDA imposes stringent restrictions on manufacturers’ communications
27
regarding off-label uses and if we, or our collaborators, do not promote our products in a manner consistent with the approved labeling, we, or they, may be subject to warnings or enforcement action for off-label marketing. Violation of the Federal Food, Drug, and Cosmetic Act, or FCA, and other statutes, including the FCA, relating to the promotion and advertising of prescription drugs may lead to investigations or allegations of violations of federal and state healthcare fraud and abuse laws and state consumer protection laws.
Disruptions at the FDA, the SEC and other government agencies and regulatory authorities caused by funding shortages or a government shutdown could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
The ability of the FDA and comparable foreign regulatory authorities to review and approve new products can be affected by a variety of factors, including government budget and funding levels, government shutdowns, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory, and policy changes. Average review times at the agency have fluctuated in recent years as a result. In addition, government funding of the SEC and other government agencies on which our operations may rely, including those that fund research and development activities, is subject to the political process, which is inherently fluid and unpredictable.
Disruptions at the FDA and other agencies and comparable regulatory authorities may also slow the time necessary for new drugs or biologics to be reviewed and/or approved by necessary government agencies and regulatory authorities, which would adversely affect our business. For example, over the last several years, including the ongoing government shutdown that began on October 1, 2025, and previously from December 22, 2018 to January 25, 2019, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities. If the current government shutdown continues or a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business
Item 5. Other Information
Trading Arrangements
During the three months ended September 30, 2025, none of our directors and officers (as defined in Rule 16a-1 (f) under the Exchange Act)
28
Item 6. Exhibits
Exhibit No. |
|
Description |
|
|
|
3.1 |
|
|
|
|
|
3.2 |
|
|
|
|
|
3.3 |
|
|
|
|
|
31.1 |
|
|
|
|
|
31.2 |
|
|
|
|
|
32.1* |
|
|
|
|
|
101.INS |
|
Inline XBRL Instance Document - the instance document does not appear in the Interactive Data File as its XBRL tags are embedded within the Inline XBRL Document. |
|
|
|
101.SCH |
|
Inline XBRL Taxonomy Extension Schema With Embedded Linkbase Documents |
|
|
|
104 |
|
Cover page formatted as Inline XBRL and contained in Exhibit 101. |
* Furnished herewith and not deemed to be “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liability of that section, and shall not be deemed to be incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act (whether made before or after the date of the Form 10-Q), irrespective of any general incorporation language contained in such filing.
29
SIGNATURES
Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
Date: November 3, 2025
By: |
|
/s/ Michael A. Metzger |
|
|
Michael A. Metzger |
|
|
Chief Executive Officer |
|
|
(Principal Executive Officer) |
By: |
|
/s/ Keith A. Goldan |
|
|
Keith A. Goldan |
|
|
Chief Financial Officer and Treasurer |
|
|
(Principal Financial Officer and Principal Accounting Officer) |
30