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Item 1. Financial Statements

2026-08-04 compared with 2026-05-14 · 137 added, 109 removed, 392 unchanged (39% of the section changed)

Financial Statements (unaudited)

Condensed Consolidated Balance Sheets as of March 31, 2026 and December 31, 2025

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three months ended March 31, 2026 and 2025

Condensed Consolidated Statements of Changes in Stockholders' Equity for the three months ended March 31, 2026 and 2025

Condensed Consolidated Statements of Cash Flows for the three months ended March 31, 2026 and 2025

Condensed Consolidated Balance Sheets as of June 30, 2026 and December 31, 2025

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three and six months ended June 30, 2026 and 2025

Condensed Consolidated Statements of Changes in Stockholders' Equity for the three and six months ended June 30, 2026 and 2025

Condensed Consolidated Statements of Cash Flows for the six months ended June 30, 2026 and 2025

Notes to Condensed Consolidated Financial Statements

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5,000,000 shares authorized

as of March 31, 2026 and December 31, 2025;

as of June 30, 2026 and December 31, 2025;

500,000 and 250,000

shares issued and outstanding as of March 31, 2026

and December 31, 2025, respectively

shares issued and outstanding as of June 30, 2026 and

December 31, 2025, respectively

Common stock, $ 0.001 par value per share;

115,000,000 shares

authorized as of March 31, 2026 and December 31, 2025;

103,882,937 and 83,090,392 shares issued and outstanding as of

March 31, 2026 and December 31, 2025, respectively

215,000,000 and 115,000,000

shares authorized as of June 30, 2026 and December 31, 2025,

respectively;

103,882,937 and 83,090,392 shares issued and

outstanding as of June 30, 2026 and December 31, 2025, respectively

Additional paid-in capital

Accumulated deficit

Accumulated other comprehensive gain

Accumulated other comprehensive gain (loss)

Total stockholders’ equity

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(In thousands, except share and per share data)

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Operating expenses:

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Preferred stock

Weighted-average shares used in computing basic and diluted net loss per share:

Weighted-average shares used in computing basic and

diluted net loss per share:

Preferred stock

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Balances as of March 31, 2026

Stock-based compensation expense

Unrealized loss on marketable debt securities

Balances as of June 30, 2026

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

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Balances as of March 31, 2025

Vesting of restricted stock units

Stock-based compensation expense

Unrealized loss on marketable securities

Balances as of June 30, 2025

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

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(In thousands)

Three Months Ended March 31,

Six Months Ended June 30,

Cash flows from operating activities:

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

Offering costs included in accounts payable and accrued expense

Offering costs included in accounts payable and accrued expenses

Leased assets obtained in exchange for new operating lease liabilities

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

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The consolidated balance sheet as of December 31, 2025 was derived from the Company’s audited financial statements but does not include all disclosures required by GAAP.

The accompanying unaudited condensed consolidated financial statements as of March 31, 2026 and for the three months ended March 31, 2026 and 2025, have been prepared by the Company pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements.

The accompanying unaudited condensed consolidated financial statements as of June 30, 2026 and for the three and six months ended June 30, 2026 and 2025, have been prepared by the Company pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements.

Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to such rules and regulations.

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These condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and the notes thereto for the year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K filed with the SEC on March 19, 2026.

In the opinion of management, all adjustments, consisting only of normal recurring adjustments necessary for a fair statement of the Company’s condensed consolidated financial position as of March 31, 2026, condensed consolidated results of operations for the three months ended March 31, 2026 and condensed consolidated statement of cash flows for the three months ended March 31, 2026 have been made.

The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2026 .

In the opinion of management, all adjustments, consisting only of normal recurring adjustments necessary for a fair statement of the Company’s condensed consolidated financial position as of June 30, 2026, condensed consolidated results of operations for the three and six months ended June 30, 2026 and condensed consolidated statement of cash flows for the six months ended June 30, 2026 have been made.

The results of operations for the three and six months ended June 30, 2026 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2026 .

Liquidity and Capital Resources

The Company’s condensed consolidated financial statements have been presented on the basis that it will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business.

Since its inception, the Company has incurred significant recurring operating losses and negative cash flows from operations.

The Company has incurred net losses of $ 29.6 million and $ 29.3 million for the three months ended March 31, 2026 and 2025, respectively.

In addition, as of March 31, 2026, the Company had an accumulated deficit of $ 464.4 million .

The Company has incurred net losses of $ 62.4 million and $ 55.5 million for the six months ended June 30, 2026 and 2025, respectively.

In addition, as of June 30, 2026, the Company had an accumulated deficit of $ 497.2 million .

The Company expects to continue to generate operating losses for the foreseeable future.

As of March 31, 2026, the Company had approximately $ 200.4 million of cash, cash equivalents and marketable securities available for use to fund its operations and capital requirements.

As of June 30, 2026, the Company had approximately $ 156.3 million of cash, cash equivalents and marketable securities available for use to fund its operations and capital requirements.

The Company has funded its operations to date primarily with proceeds from sales of common stock and proceeds from the sale of prefunded warrants for the purchase of common stock, the acquisition in 2020 of cash, cash equivalents and marketable securities upon the merger with Zafgen, Inc.

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In July 2025, the Company completed an underwritten public offering of common stock raising net proceeds of approximately $ 65.0 million.

In February 2026, the Company completed an underwritten public offering of common stock raising net proceeds of approximately $ 107.6 million.

In February 2026, the Company completed another underwritten public offering of common stock raising net proceeds of approximately $ 107.6 million.

Going Concern

In accordance with Accounting Standards Update (“ASU”) No.

2014-15, “Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern”, the Company has evaluated whether there are certain conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that these condensed consolidated financial statements are issued.

As of the issuance date of these condensed consolidated financial statements, the Company expects its cash, cash equivalents and marketable securities will be sufficient to fund its forecasted operating expenses and capital expenditure requirements for at least twelve months from the date these financial statements are issued.

The Company has not yet commercialized any products and its ability to commercialize nomlabofusp is dependent on receiving regulatory approval in the United States and/or other countries.

The Company expects that its research and development, general and administrative and commercial expenses will continue to increase and, as a result, that it may need additional capital to fund its future operating and capital requirements.

Unless and until the Company can generate substantial revenue, management continuously evaluates different strategies to obtain the required funding for future operations.

These strategies include seeking additional funding through a combination of public or private equity offerings, debt or royalty financings, collaborations and licensing arrangements, strategic partnerships with pharmaceutical and/or larger biotechnology companies, or other sources.

The incurrence of indebtedness would result in increased payment obligations and the Company may be required to agree to certain restrictive covenants, such as limitations on its ability to incur additional debt, limitations on its ability to acquire, sell or license intellectual property rights, minimum required cash balances and other operating restrictions that could adversely impact the Company's ability to conduct its business.

Any additional fundraising efforts may divert the Company's management from their day-to-day activities, which may adversely affect its ability to develop and commercialize its product candidates.

There can be no assurance that the Company would be able to raise sufficient additional capital on acceptable terms, if at all.

If such additional financing is not available on satisfactory terms, or is not available in sufficient amounts, or if the Company does not have sufficient authorized shares, the Company may be required to delay, limit, or eliminate the development of business opportunities and its ability to achieve its business objectives, its competitiveness, and its business, financial condition, and results of operations will be materially adversely affected.

The Company could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and it may be required to relinquish rights to some of its technologies or product candidates or otherwise agree to terms unfavorable to it, any of which may have a material adverse effect on the Company's business, operating results and prospects.

In addition, geopolitical tensions (including the war in the Middle East and other regional conflicts around the world), volatility of capital markets, and other adverse macroeconomic events, including those due to inflationary pressures, rising interest rates, tariff or trade protection measures that have been or may in the future be imposed by the U.S.

or other countries, bank instability and the ability of the U.S.

government to manage federal debt limits as well as the potential impact of other health crises on the global financial markets may reduce the Company's ability to access capital, which could negatively affect its liquidity and ability to continue as a going concern.

If the Company is unable to obtain sufficient funding when needed and/or on acceptable terms, the Company may be required to significantly curtail, delay or discontinue one or more of its research and development programs, the manufacture of clinical and commercial supplies, product portfolio expansion, commercialization efforts and/or commercial operations, which could adversely affect its business prospects, or the Company may be unable to continue operations.

If the timing of the Company's clinical assumptions are delayed or if there are other forecasted assumption changes that negatively impact its operating plan, the Company would reduce expenditures in order to further extend cash resources.

2014-15, “Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern”, the Company has evaluated whether there are certain conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that these unaudited condensed consolidated financial statements are issued.

While the Company expects its cash, cash equivalents and marketable securities will fund its forecasted operating expenses and capital expenditure requirements into the third quarter of 2027, these cash resources are not sufficient to fund the Company’s planned operations for a period of at least one year from the date these unaudited condensed consolidated financial statements are issued.

The Company will need to raise additional capital to fund its ongoing operations.

The Company continues to evaluate opportunities to further finance its operating cash needs through a combination of some, or all, of the following:

equity or debt offerings, royalty financings, collaborations, strategic alliances, and/or marketing, distribution or licensing arrangements.

In addition, the Company believes it will be eligible for the FDA's rare pediatric disease priority review voucher which could then be sold if nomlabofusp receives FDA approval.

There is no assurance, however, that additional financing will be available when needed or that the Company will be able to obtain financing on terms acceptable to the Company.

If the Company is unable to obtain funding when required in the future, the Company could be required to delay, reduce, or eliminate research and development programs, or commercialization efforts, which could adversely affect its business prospects.

These conditions raise substantial doubt about the Company’s ability to continue as a going concern.

The accompanying unaudited condensed consolidated financial statements have been prepared assuming the Company will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of the business.

The unaudited condensed consolidated financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classifications of liabilities that might result from the outcome of this uncertainty.

Summary of Significant Accounting Policies

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Stock-Based Compensation

The Company measures all stock-based awards granted to employees and directors based on the fair value on the date of grant using the Black-Scholes option-pricing model.

Compensation expense of those awards is recognized over the requisite service period, which is the vesting period of the respective award.

The Company accounts for its stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation-Stock Compensation (ASC 718).

ASC 718 requires all stock-based payments to employees, including grants of employee stock options, restricted stock units, modifications to existing stock options, and equity classified warrants to be recognized in the consolidated statements of operations based on their grant date fair values.

The grant-date fair value of stock options is estimated using the Black-Scholes option pricing model.

The grant-date fair value of restricted stock awards and performance-based restricted stock awards are determined based on the closing price of the Company’s common stock on the date of grant (or, if the grant date is not a business day, the closing price on the preceding business day).

Compensation expense of stock options and restricted stock units is recognized over the requisite service period, which is the vesting period of the respective award.

Typically, the Company issues awards with only service-based vesting conditions and records the expense for these awards using the straight-line method.

The Company accounts for forfeitures as they occur.

For performance-based restricted stock awards, the Company recognizes the expense over the estimated period in which the awards are expected to be earned;

such period begins once achievement of the performance objective is determined to be probable.

The Company classifies stock-based compensation expense in its consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified.

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The expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options.

risk-free interest rate is determined by reference to the U.S.

The risk-free interest rate is determined by reference to the U.S.

Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award.

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The CODM uses consolidated financial information, including consolidated net loss, to evaluate performance, forecast future period financial results, allocate resources for the company by, among other things, comparing budgeted to actual results.

The table below summarizes the significant expense categories regularly provided to the CODM for the three months ended March 31, 2026 and 2025:

For the Three Months Ended March 31,

The table below summarizes the significant expense categories regularly provided to the CODM for the three and six months ended June 30, 2026 and 2025:

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Operating expenses:

Technical operations

Development (a)

Development (a)(b)

Nomlabofusp support

General and administrative (b)

Commercial (c)

General and administrative (c)(d)

Commercial (e)

Total operating expenses

Other income, net

(a) Development expenses include research and development related stock compensation expense of approximately $ 1.0 million and $ 0.9 million for the three months ended March 31, 2026 and 2025, respectively.

(b) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 1.0 million and $ 0.9 million for the three months ended March 31, 2026 and 2025, respectively.

(a) Development expenses include research and development related stock compensation expense of approximately $ 1.1 million and $ 0.9 million for the three months ended June 30, 2026 and 2025, respectively.

(b) Development expenses include research and development related stock compensation expense of approximately $ 2.1 million and $ 1.8 million for the six months ended June 30, 2026 and 2025, respectively.

(c) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 1.1 million and $ 0.9 million for the three months ended June 30, 2026 and 2025, respectively.

(d) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 2.0 million and $ 1.8 million for the six months ended June 30, 2026 and 2025, respectively.

(e) Commercial expenses relate to commercial readiness activities.

Recently Issued and Adopted Accounting Pronouncements

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We record valuation allowances to reduce deferred tax assets to the amount we believe is more likely than not to be realized.

During the three months ended March 31, 2026 and 2025 , the Company recorded no income tax benefits for the net operating losses incurred in each period due to the uncertainty of realizing a benefit from those items.

During the three and six months ended June 30, 2026 and 2025 , the Company recorded no income tax benefits for the net operating losses incurred in each period due to the uncertainty of realizing a benefit from those items.

Fair Value Measurements and Marketable Securities

Fair Value Measurements

The Company’s assets and liabilities that are measured at fair value on a recurring basis as of March 31, 2026 and December 31, 2025 are measured in accordance with the standards of ASC 820,“ Fair Value Measurements and Disclosures ”, which establishes a three-level valuation hierarchy for measuring fair value and expands financial statement disclosures about fair value measurements.

The Company’s assets and liabilities that are measured at fair value on a recurring basis as of June 30, 2026 and December 31, 2025 are measured in accordance with the standards of ASC 820,“ Fair Value Measurements and Disclosures ”, which establishes a three-level valuation hierarchy for measuring fair value and expands financial statement disclosures about fair value measurements.

The valuation hierarchy is based on the transparency of inputs to the valuation of an asset or liability as of the measurement date.

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The Company’s financial instruments consist primarily of cash, cash equivalents, marketable securities, accounts payable and accrued liabilities.

For accounts payable and accrued liabilities, the carrying amounts of these financial instruments as of March 31, 2026 and December 31, 2025 were considered representative of their fair values due to their short term to maturity.

The following tables summarize the Company’s cash equivalents and marketable securities as of March 31, 2026 and December 31, 2025:

For accounts payable and accrued liabilities, the carrying amounts of these financial instruments as of June 30, 2026 and December 31, 2025 were considered representative of their fair values due to their short term to maturity.

The following tables summarize the Company’s cash equivalents and marketable securities as of June 30, 2026 and December 31, 2025:

(in thousands)

March 31, 2026

June 30, 2026

Cash equivalents:

Money market funds invested in government securities

Treasury Bills

Total cash equivalents

Marketable securities:

Treasury Bills

Government securities

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Total cash equivalents and marketable securities

The accrued interest receivable related to the Company’s investments was $ 0.8 million and $ 0.7 million as of March 31, 2026 and December 31, 2025, respectively, and is included in prepaid expenses and other current assets on the condensed consolidated balance sheet.

The accrued interest receivable related to the Company’s investments was $ 0.3 million and $ 0.7 million as of June 30, 2026 and December 31, 2025, respectively, and it is included in prepaid expenses and other current assets on the condensed consolidated balance sheet.

The Company classifies its money market funds and U.S.

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These inputs include reported trades of and broker/dealer quotes on the same or similar securities, issuer credit spreads, benchmark securities, prepayment/default projections based on historical data and other observable inputs.

As of March 31, 2026 and December 31, 2025, the unrealized losses for available-for-sale investments were non-credit related, and the Company does not intend to sell the investments that were in an unrealized loss position, nor will it be required to sell those investments before recovery of their amortized cost basis, which may be maturity.

As of March 31, 2026 and December 31, 2025 , no allowances for credit losses for the Company’s investments were recorded.

During the three months ended March 31, 2026 and 2025 , the Company did no t recognize any impairment losses related to investments.

As of March 31, 2026 and December 31, 2025, the Company's cash equivalents and marketable securities consisted of a U.S.

As of June 30, 2026 and December 31, 2025, the unrealized losses for available-for-sale investments were non-credit related, and the Company does not intend to sell the investments that were in an unrealized loss position, nor will it be required to sell those investments before recovery of their amortized cost basis, which may be maturity.

As of June 30, 2026 and December 31, 2025 , no allowances for credit losses for the Company’s investments were recorded.

During the three and six months ended June 30, 2026 and 2025 , the Company did no t recognize any impairment losses related to investments.

As of June 30, 2026 and December 31, 2025, the Company's cash equivalents and marketable securities consisted of a U.S.

government money market fund, U.S.

Treasury Bills, and U.S.

government and agency securities,

all held in our name in a separate custody account with U.S.

government and agency securities, all held in our name in a separate custody account with U.S.

government money market fund has same-day liquidity access and the U.S.

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Marketable Securities

The following table summarizes the Company's marketable securities as of March 31, 2026 and December 31, 2025:

The following table summarizes the Company's marketable securities as of June 30, 2026 and December 31, 2025:

(in thousands)

March 31, 2026

June 30, 2026

Treasury Bills

Government securities

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Total marketable securities

As of March 31, 2026 and December 31, 2025 , the Company held no investments that have been in a continuous loss position for 12 months or longer.

As of June 30, 2026 and December 31, 2025 , the Company held no investments that have been in a continuous loss position for 12 months or longer.

Prepaid Expenses and Other Current Assets

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Accumulated depreciation

Depreciation expense was $ 0.1 million for the three months ended March 31, 2026 and 2025, respectively.

In addition, for the three months ended March 31, 2026 and 2025 , there was less than $ 0.1 million of depreciation related to sublet assets recorded as other expense.

Depreciation expense was $ 0.1 million for the three and six months ended June 30, 2026 , respectively.

Depreciation expense was $ 0.1 million and $ 0.2 million for the three and six months ended June 30, 2025, respectively.

In addition, for the three and six months ended June 30, 2026 and 2025 , there was less than $ 0.1 million of depreciation related to sublet assets recorded as other expense.

Accrued Expenses

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Stockholders’ Equity and Stock Options

As of March 31, 2026 , the Company’s Ninth Amended and Restated Certificate of Incorporation, as amended, authorized the Company to issue up to 115,000,000 shares of common stock, par value $ 0.001 per share, of which 103,882,937 shares were issued and outstanding, and up to 5,000,000 shares of undesignated preferred stock, par value $ 0.001 per share, of which 500,000 shares were issued or outstanding.

At the Company's annual meeting of stockholder on May 19, 2026, the Company's stockholders approved and increase in the number of authorized shares of common stock, par value $ 0.001 per share from 115,000,000 shares to 215,000,000 shares.

As of June 30, 2026 , the Company’s Ninth Amended and Restated Certificate of Incorporation, as amended, authorized the Company to issue up to 215,000,000 shares of common stock, par value

$ 0.001 per share, of which 103,882,937 shares were issued and outstanding, and up to 5,000,000 shares of undesignated preferred stock, par value $ 0.001 per share, of which 500,000 shares were issued and outstanding.

The voting, dividend and liquidation rights of the holders of the Company’s common stock are subject to and qualified by the rights, powers and preferences of the holders of the preferred stock.

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In the event of the Company’s liquidation, dissolution or winding up, holders of Series A Preferred Stock will participate pari passu with any distribution of proceeds to holders of common stock, calculated on an as-converted basis.

Holders of preferred stock are entitled to receive when, as, and if dividends are

declared and paid on the common stock, an equivalent dividend, calculated on an as-converted basis.

Holders of preferred stock are entitled to receive when, as, and if dividends are declared and paid on the common stock, an equivalent dividend, calculated on an as-converted basis.

Shares of Series A Preferred Stock are otherwise not entitled to dividends.

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Options outstanding under the Prior Plans will remain outstanding, unchanged, and subject to the terms of the Prior Plans and the respective award agreements, and no further awards will be made under the Prior Plans.

However, if any award previously granted under the Prior Plans, expires, terminates, is canceled, or is forfeited for any reason after the approval of the 2020 Plan, the shares subject to that award will be added to the 2020 Plan share pool so that they can be utilized for new grants under the 2020 Plan.

However, if any award previously granted under the Prior Plans, expires, terminates, is canceled, or is forfeited for any reason after the

approval of the 2020 Plan, the shares subject to that award will be added to the 2020 Plan share pool so that they can be utilized for new grants under the 2020 Plan.

The 2020 Plan provides for the grant of incentive stock options (“ISOs”), nonstatutory stock options (“NSOs”), stock appreciation rights, restricted stock awards, restricted stock unit awards, performance-based restricted stock units, and cash or other stock-based awards.

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(i) 1,700,000 shares plus (ii) an annual increase on January 1, 2021 and each anniversary of such date thereafter through January 1, 2030, equal to the lesser of (A) 4 % of the shares issued and outstanding on the last day of the immediately preceding fiscal year, or (B) such smaller number of shares as determined by the Board (collectively, the “Plan Limit”).

The maximum aggregate number of shares that may be issued under the 2020 Plan is 8,000,000 over the ten-year term of the 2020 Plan.

The maximum aggregate number of shares that may be issued under the 2020 Plan in respect of incentive stock options is 8,000,000 over the ten-year term of the 2020 Plan.

As permitted by the 2020 Plan, the Company added 3,323,616 and 2,552,603 shares available for grant to the 2020 Plan on January 1, 2026 and January 1, 2025, respectively.

As of March 31, 2026 , 1,326,303 shares of common stock were available for grant under the 2020 Plan.

During the three months ended March 31, 2026 and twelve months ended December 31, 2025 , options to purchase 416 and 1,333 shares, respectively, issued under the Prior Plans were cancelled and became available for grant under the 2020 Plan.

As of June 30, 2026 , 848,841 shares of common stock were available for grant under the 2020 Plan.

During the six months ended June 30, 2026 and twelve months ended December 31, 2025 , options to purchase 1,623 and 1,333 shares, respectively, issued under the Prior Plans were cancelled and became available for grant under the 2020 Plan.

Stock Option Valuation

The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees during the three months ended March 31, 2026:

Risk-free interest rate

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Stock Options

The following table summarizes the Company’s stock option activity for the three months ended March 31, 2026 (amounts in millions, except for share, contractual term, and per share data):

The following table summarizes the Company’s stock option activity for the six months ended June 30, 2026 (amounts in millions, except for share, contractual term, and per share data):

Weighted Average

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Options forfeited/expired

Outstanding as of March 31, 2026

Exercisable as of March 31, 2026

Vested and expected to vest as of March 31, 2026

(a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were (“In the money”) as of March 31, 2026 .

Outstanding as of June 30, 2026

Exercisable as of June 30, 2026

Vested and expected to vest as of June 30, 2026

Option Grants

During the three months ended March 31, 2026 , the Company granted options to purchase 2,146,290 shares of common stock to employees and directors under the 2020 Plan.

The options have an exercise price equal to the closing stock price as of the grant date and vest over four years , with 25 % vesting on the first anniversary of the grant and the remainder vesting in equal monthly installments thereafter.

The weighted-average grant date fair value of options granted under the 2020 Plan during the three months ended March 31, 2026 was $ 2.89 .

As of March 31, 2026 , total unrecognized compensation expense related to unvested stock options granted under the 2020 Plan was $ 15.0 million, which is expected to be recognized over a weighted average period of 2.58 years.

During the six months ended June 30, 2026 , the Company granted options to purchase 2,636,040 shares of common stock to employees and directors under the 2020 Plan.

The options have an exercise price equal to the closing stock price as of the grant date.

Of the 2,636,040 options granted, 2,360,290 were granted to employees and vest over four years , with 25 % vesting on the first anniversary of the grant and the remainder vesting in equal monthly installments thereafter.

The remaining 275,750 options were annual grants to the Company's directors and vest on the earlier of (i) one year from the grant date or (ii) the date of the Company's next annual meeting of stockholders.

The weighted-average grant date fair value of options granted under the 2020 Plan during the six months ended June 30, 2026 was $ 2.87 .

As of June 30, 2026 , total unrecognized compensation expense related to unvested stock options granted under the 2020 Plan was $ 14.7 million, which is expected to be recognized over a weighted average period of 2.40 years.

Inducement Stock Option Grant

There were no inducement awards granted in the three months ended March 31, 2026.

As of March 31, 2026 , total unrecognized compensation expense related to unvested inducement options granted was $ 0.3 million, which is expected to be recognized over a weighted average period of 1.06 years.

There were no inducement awards granted in the six months ended June 30, 2026.

As of June 30, 2026 , total unrecognized compensation expense related to unvested inducement options granted was $ 0.2 million, which is expected to be recognized over a weighted average period of 0.82 years.

Restricted Stock Units

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The shares underlying the RSUs are not issued until the RSUs vest.

Activity with respect to the Company's RSUs and performance-based RSUs during the three months ended March 31, 2026 was as follows (in millions, except share, contractual term, and per share data):

Activity with respect to the Company's RSUs and performance-based RSUs during the six months ended June 30, 2026 was as follows (in millions, except share, contractual term, and per share data):

Weighted Average

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Restricted stock units forfeited

Outstanding as of March 31, 2026

Unvested and expected to vest as of March 31, 2026

Outstanding as of June 30, 2026

Unvested and expected to vest as of June 30, 2026

Restricted Stock Unit Grants

During the three months ended March 31, 2026 , the Company granted 527,190 shares of RSUs to employees under the 2020 Plan.

During the six months ended June 30, 2026 , the Company granted 527,190 shares of RSUs to employees under the 2020 Plan.

The RSUs vest annually over four years and have a weighted-average grant date fair value of $ 3.60 per unit.

As of March 31, 2026 , total unrecognized compensation expense for RSUs was $ 3.6 million, which is expected to be recognized over a weighted average period of 2.86 years.

As of June 30, 2026 , total unrecognized compensation expense for RSUs was $ 3.2 million, which is expected to be recognized over a weighted average period of 2.70 years.

Performance-Based Restricted Stock Unit Grants

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With respect to the January 2025 PSU Awards, the Board established specified regulatory-related performance criteria and a corresponding performance period over which such performance criteria must be achieved, the satisfaction of which are conditions to earning the January 2025 PSU Awards and vesting of the underlying RSUs.

As of March 31, 2026 , the January 2025 PSU Awards were outstanding covering 200,000 common stock underlying RSUs.

As of June 30, 2026 , the January 2025 PSU Awards were outstanding covering 200,000 common stock underlying RSUs.

The grant date fair value of the performance-based RSUs was $ 3.46 per unit based on the grant date closing price per share.

As of March 31, 2026 , the underlying performance criteria of the January 2025 PSU Awards were determined to be not probable of achievement for accounting purposes, and no stock-based compensation expense was recognized for the three months ended March 31, 2026 and 2025.

As of June 30, 2026 , the underlying performance criteria of the January 2025 PSU Awards were determined to be not probable of achievement for accounting purposes, and no stock-based compensation expense was recognized for the three and six months ended June 30, 2026 and 2025.

Stock-Based Compensation

Stock-based compensation expense was classified in the condensed consolidated statements of operations as follows:

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Research and development

23 unchanged sentences

On November 24, 2025, the Company executed the second amendment to the lease on the current 8,104 square footage of office space in Bala Cynwyd, Pennsylvania which was set to expire in August 2026 that extended the lease term on the then current 8,104 square footage through January 31, 2030 and agreed to lease an additional 7,107 square feet of space from the same landlord.

As of March 31, 2026, the property had not been made available for use due to the tenant improvement work being performed by the landlord.

Consequently, the Company determined that the lease has not yet commenced for accounting purposes.

No right of use asset or lease liability will be recorded until the lease commencement date which shall occur in Q2 2026.

The right of use assets and lease liabilities with both these leases are reflected in the financial statements for three months ended March 31, 2026 as are the right of use asset and lease liability of the Company's Boston office space discussed below.

The lease on the additional space commenced on April 14, 2026 and the Company recorded a right of use asset and lease liability of $ 1.5 million.

The right of use assets and lease liabilities with this lease are reflected in the financial statements for six months ended June 30, 2026 as are the right of use asset and lease liability of the Company's Boston office space discussed below.

Boston Office Lease

2 unchanged sentences

As part of the agreement, the Company was initially required to maintain a letter of credit, of $ 1.3 million.

In October of 2024, upon the agreement with the landlord that we had achieved certain clinical development milestones required in the lease, this letter of credit was reduced to $ 0.6 million.

In October 2024, pursuant to an agreement with the landlord acknowledging that we had achieved certain clinical development milestones required under the lease, this letter of credit was reduced to $ 0.6 million.

In June 2026, upon the agreement with the landlord that we had achieved certain clinical development milestones required in the lease, this letter of credit was reduced to $ 0.5 million.

During both periods presented, this cash deposit is classified as restricted cash within the condensed consolidated financial statements.

16 unchanged sentences

Lease Expense

Expense arising from operating leases was $ 0.1 million during the three months ended March 31, 2026 and 2025, respectively.

For operating leases, the weighted-average remaining lease term for leases as of March 31, 2026 and December 31, 2025 was 3.4 and 3.6 years, respectively.

For operating leases, the weighted average discount rate for leases as of March 31, 2026 and December 31, 2025 was 11.0 %.

Expense arising from operating leases was $ 0.2 million and $ 0.3 million during the three and six months ended June 30, 2026 , respectively.

Expense arising from operating leases was $ 0.1 million and $ 0.3 million during the three and six months ended June 30, 2025, respectively.

For operating leases, the weighted-average remaining lease term for leases as of June 30, 2026 and December 31, 2025 was 3.3 and 3.6 years, respectively.

For operating

leases, the weighted average discount rate for leases as of June 30, 2026 and December 31, 2025 was 11.0 %.

The Company has not entered into any financing leases.

Maturities of lease liabilities due under these lease agreements as of March 31, 2026 are as follows:

Maturities of lease liabilities due under these lease agreements as of June 30, 2026 are as follows:

(in thousands)

Nine months ending December 31, 2026

Six months ending December 31, 2026

Year ended December 31, 2027

6 unchanged sentences

The Company is not currently a party to any litigation, nor is management aware of any pending or threatened litigation against the Company, that it believes would materially affect the Company's business, operating results, financial condition or cash flows.

Net Loss Per Share Giving Effect to Conversion of Common Stock to Series A Preferred Stock

The following table sets forth the computation of basic and diluted net loss per share for the three months ended March 31, 2026 (in thousands, except share and per share amounts).

Net Loss Per Share Giving Effect to Exchange of Common Stock to Series A Preferred Stock

The following table sets forth the computation of basic and diluted net loss per share for the three and six months ended June 30, 2026 (in thousands, except share and per share amounts).

For purposes of earnings per share, the Series A Preferred Stock have the same characteristics as common stock and have no liquidation or other material preferential rights over common stock and accordingly, have been considered as a second class of common stock in the computation of net loss per share regardless of their legal form.

Losses are allocated between the common shares and the Series A Preferred Stock on a pro rata basis as they share equally in losses and residual net assets on an as-converted basis.

Three Months Ended June 30,

Six Months Ended June 30,

Allocation of net loss to common

5 unchanged sentences

The weighted average number of Series A Preferred Stock reflects only the period of time that this class of securities has been newly designated and issued.

The Company excluded the options below to purchase common stock, unreleased restricted stock units, and performance-based restricted stock units outstanding as of March 31, 2026 and 2025, respectively, from the computation of diluted net loss per share for the three months ended March 31, 2026 and 2025, respectively, because they had an anti-dilutive impact due to the net loss incurred for the periods.

The Company excluded the options below to purchase common stock, unreleased restricted stock units, and performance-based restricted stock units outstanding as of June 30, 2026 and 2025, respectively, from the computation of diluted net loss per share for the six months ended June 30, 2026 and 2025, respectively, because they had an anti-dilutive impact due to the net loss incurred for the periods.

Additionally, the as-converted preferred shares have been excluded from diluted earnings per share as the effect would not be dilutive.

As of March 31,

As of June 30,

Options to purchase common stock

1 unchanged sentence

Unvested performance-based restricted common stock

Related Party

During the six months ended June 30, 2026, the Company sponsored patient and caregiver awareness events held by Friedreich’s Ataxia Research Alliance ("FARA") for a cumulative $ 0.2 million.

One of the Company’s directors is a director of FARA.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1. Financial Statements