What changed in this filing

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Item 1. Financial Statements

2025-11-05 compared with 2025-08-14 · 150 added, 149 removed, 445 unchanged (40% of the section changed)

Financial Statements (unaudited)

Condensed Consolidated Balance Sheets as of June 30, 2025 and December 31, 2024

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three and six months ended June 30, 2025 and 2024

Condensed Consolidated Statements of Changes in Stockholders' Equity for the three and six months ended June 30, 2025 and 2024

Condensed Consolidated Statements of Cash Flows for the six months ended June 30, 2025 and 2024

Condensed Consolidated Balance Sheets as of September 30, 2025 and December 31, 2024

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three and nine months ended September 30, 2025 and 2024

Condensed Consolidated Statements of Changes in Stockholders' Equity for the three and nine months ended September 30, 2025 and 2024

Condensed Consolidated Statements of Cash Flows for the nine months ended September 30, 2025 and 2024

Notes to Condensed Consolidated Financial Statements

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(In thousands, except share and per share data)

September 30,

Current assets:

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5,000,000 shares authorized

as of June 30, 2025 and December 31, 2024;

as of September 30, 2025 and December 31, 2024;

no shares issued and

outstanding as of June 30, 2025 and December 31, 2024

outstanding as of September 30, 2025 and December 31, 2024

Common stock, $ 0.001 par value per share;

115,000,000 shares

authorized as of June 30, 2025 and December 31, 2024;

authorized as of September 30, 2025 and December 31, 2024;

85,590,392 and 63,815,065 shares issued and outstanding as of

June 30, 2025 and December 31, 2024, respectively

September 30, 2025 and December 31, 2024, respectively

Additional paid-in capital

Accumulated deficit

Accumulated other comprehensive gain (loss)

Accumulated other comprehensive gain

Total stockholders’ equity

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(In thousands, except share and per share data)

Three Months Ended June 30,

Six Months Ended June 30,

Three Months Ended September 30,

Nine Months Ended September 30,

Operating expenses:

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Other comprehensive loss:

Unrealized loss on marketable securities

Total other comprehensive loss

Unrealized gain (loss) on marketable securities

Total other comprehensive gain (loss)

Total comprehensive loss

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Balances as of June 30, 2025

Issuance of common stock, net

Stock-based compensation expense

Unrealized gain on marketable debt securities

Balances as of September 30, 2025

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

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Balances as of June 30, 2024

Stock-based compensation expense

Exercise of stock options

Unrealized gain on marketable debt securities

Balances as of September 30, 2024

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

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(In thousands)

Six Months Ended June 30,

Cash flows from operating activities:

Nine Months Ended September 30,

Operating activities:

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

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Net cash used in operating activities

Cash flows from investing activities:

Investing activities:

Purchases of property and equipment

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Net cash provided by (used in) investing activities

Cash flows from financing activities:

Proceeds from issuance of equity securities, net of issuance costs

Financing activities:

Proceeds from issuance of common stock, net of issuance costs

Proceeds from exercise of stock options and warrants

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Supplemental disclosure of non-cash investing and financing activities:

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

Offering costs included in accrued expense

Offering costs included in accounts payable and accrued expense

Leased assets obtained in exchange for new operating lease liabilities

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Larimar initiated human clinical studies of nomlabofusp in 2019.

Since then, the Company has completed two Phase 1 clinical studies, a Phase 2 dose exploration study and a PK run-in study in adolescents (12-17 years old).

Since then, the Company has completed four clinical studies:

(i) two Phase 1 clinical studies, (ii) a Phase 2 dose exploration study and (iii) a PK run-in study in adolescents (12-17 years old).

In 2024, the Company initiated its open label extension study in patients with FA.

The open label extension study's eligibility criteria only allowed patients who previously participated in the Company’s prior nomlabofusp Phase 1 or Phase 2 studies.

In 2025, the Company changed the name of the open label extension study to the open label study, due to the inclusion of patients who had not participated in prior nomlabofusp clinical studies.

The Company's ongoing open label study is currently screening and enrolling eligible adolescents that completed dosing in the recently completed PK run-in study into the open label study.

In addition, the Company is also screening and enrolling patients with FA who have not participated in prior nomlabofusp clinical studies.

The Company is also planning to enroll children (2-11 years of age) directly into the open label study.

In 2025, the Company amended the study protocol to include adolescent and adult patients who had not participated in a prior nomlabofusp study and the study name was changed to the open label study.

The Company has engaged in multiple discussions and interactions with the U.S.

Food and Drug Administration (“FDA”) in connection with its clinical development of nomlabofusp and such communications remain ongoing.

The Company currently participates in the FDA’s Center for Drug Evaluation and Research ("CDER”) Support for Clinical Trials Advancing Rare Disease Therapeutics (“START”) pilot program.

The objective of this program is to accelerate the development of drugs for rare diseases that lead to significant disability or death by facilitating frequent advice and regular communication with the FDA staff to expedite the review process.

The Company was selected for, and currently participates in the FDA’s Center for Drug Evaluation and Research (“CDER”) Support for Clinical Trials Advancing Rare Disease Therapeutics (“START”) pilot program.

The Company has also had numerous interactions with the European Medicines Agency (“EMA”) and the United Kingdom’s Medicines and Healthcare Regulatory Agency (“MHRA”) regarding the clinical development of nomlabofusp.

The Company has received Orphan Drug Designation and Fast Track Designation from the FDA.

The Company has been granted orphan drug designation in the European Union (the ”EU”) and access to the EU’s Priority Medicines Program (“PRIME”) Scheme.

The Company has also received access to the EMA’s Innovative Licensing and Access Pathway (“ILAP”).

The Company has been granted orphan drug designation by the EMA and access to the EMA's Priority Medicines Program (“PRIME”) scheme.

The Company has also received access to the MHRA's Innovative Licensing and Access Pathway (“ILAP”).

All these programs are designed to facilitate drug development of therapeutics for rare and/or orphan indications.

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This data included safety, FXN levels, clinical and pharmacokinetic data.

Also in December 2024, the Company announced that they were increasing the dose in the open label study to 50 mg of nomlabofusp daily for then currently enrolled participants.

All newly enrolled participants will start dosing at 50 mg nomlabofusp daily.

In March 2025, the Company announced that its Safety Monitoring Team has deemed anaphylaxis as an adverse drug reaction likely associated with nomlabofusp and therefore they expect to see additional reactions.

To reduce the risk of allergic reactions, including anaphylaxis, the Company amended the open label study protocol to administer premedication with antihistamines for the first month of dosing.

Also in December 2024, the Company announced that it was increasing the dose in the open label study to 50 mg of nomlabofusp daily for then currently enrolled participants.

All newly enrolled participants start dosing at 50 mg nomlabofusp daily.

In March 2025, the Company announced that its Safety Monitoring Team has deemed anaphylaxis as an adverse drug reaction likely associated with nomlabofusp had identified anaphylaxis as an adverse drug reaction likely associated with nomlabofusp.

• In January 2025, the Company initiated dosing of adolescents (12-17 years old) in its PK run-in study for patients with FA.

Study participants were randomized 2:1 to receive either nomlabofusp at a

weight-base dose expected to match the PK of adults receiving the 50 mg dose, or placebo, daily for seven days.

Study participants were randomized 2:1 to receive either nomlabofusp at a weight-base dose expected to match the PK of adults receiving the 50 mg dose, or placebo, daily for seven days.

The Company completed dosing of 14 adolescents in March 2025.

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The frozen solution is the dosage form currently being used in the open label study.

In May 2025, the Company began to introduce the lyophilized product formulation, which is intended for commercialization, into the open label study with new study participants.

• In March 2025, the Company announced that FDA stated as part of a START pilot program that it is open to considering the use of FXN concentration as a reasonably likely surrogate endpoint (“RLSE”) and the acceptability of FXN concentration as an RLSE to support accelerated approval will be a matter of review in a future marketing application.

In July 2025, the Company began to introduce the lyophilized product formulation, which is intended for commercialization, into the open label study with new study participants.

• In March 2025, the Company announced that FDA stated as part of a START pilot program meeting that it is open to considering the use of FXN concentration as a reasonably likely surrogate endpoint (“RLSE”) and the acceptability of FXN concentration as an RLSE to support accelerated approval will be a matter of review in a future marketing application.

FDA recommended focusing on assessments of skin FXN concentrations rather than buccal FXN concentrations due to more consistent sampling and less variability.

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• In June 2025, the FDA provided safety database recommendations for a Biologics License Application, (“BLA”), seeking accelerated approval, which included evaluating safety in at least 30 participants with continuous study drug exposure for six months and a subset of at least 10 of those participants with continuous study drug exposure for one year, with the large majority of data coming from participants receiving the 50 mg dose.

In a prior communication received in March, FDA stated that it is open to the use of skin FXN concentrations as a RLSE and acknowledged the submitted data appear to support a relationship between increased skin FXN and relevant tissues such as the heart, dorsal root ganglion, and skeletal muscle.

Acceptability of increases in skin FXN for accelerated approval will be decided during future BLA review.

• In July 2025, the Company announced the publication of nonclinical data evaluating the mechanism of action, pharmacodynamics and pharmacology of nomlabofusp as a novel FXN protein replacement therapy designed to address the underlying cause of FA in two peer-reviewed articles.

• In July 2025, the Company announced the publication of two peer-reviewed articles with nonclinical data evaluating the mechanism of action, pharmacodynamics and pharmacology of nomlabofusp as a novel FXN protein replacement therapy designed to address the underlying cause of FA.

These data were included in the briefing package reviewed by the FDA in support of using skin FXN concentrations as a RLSE for the Company's registrational program seeking accelerated approval for nomlabofusp.

Nomlabofusp plans and milestones include the following:

• The ongoing open label study continues to enroll, and active study participants are currently receiving the 50 mg dose of nomlabofusp.

The Company plans to provide an update in September 2025 on open label data on at least 30 to 40 study participants who received at least one dose of nomlabofusp.

Safety and PK data from the recently completed adolescent PK run-in study are also expected as part of the September 2025 update.

• Adolescent participants from the PK run-in study are currently screening and enrolling in the open label study.

Larimar is also screening and enrolling into the open label study patients with FA who have not participated in prior nomlabofusp clinical studies.

In addition, Larimar plans to enroll children (2 to 11 years of age) directly into the open label study.

• In the second quarter of 2026, the Company plans to submit a BLA seeking accelerated approval.

• Following feedback from FDA and EMA on the study protocol, global Phase 3 study sites in the U.S., European Union, U.K., Canada, and Australia were identified and are currently being qualified and initiated.

The Company expects to begin patient recruitment later this year.

• In September 2025, the Company announced data from its ongoing long-term open label study:

o In four completed studies and its ongoing open label study, 65 participants received at least one dose of nomlabofusp, including 39 in the open label study, with 14 on treatment for at least six months and eight for over one year in the open label study.

o Consistent directional improvements across four key clinical outcomes were observed in the open label study relative to a Friedreich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population and the observed increase in skin FXN levels.

The Company believes these new data, as well as the improvement in abnormal lipid profiles observed in prior completed studies, provide support that nomlabofusp increases FXN in patients with FA and that the strategy of FXN replacement has the potential to result in a clinical benefit.

o Changes observed in skin FXN levels and clinical outcomes after nomlabofusp administration across diverse participants with FA, including individuals with advanced disease, are all directionally consistent and suggest a potential treatment effect.

o Of the 39 participants in the open label study (and of 65 total participants who received at least 1 dose in all nomlabofusp studies), 7 experienced anaphylaxis in the first 6 weeks of dosing and returned to their usual state of health after standard treatment.

Anaphylaxis is more common upon re-exposure to a drug after a gap in dosing;

in the open label study, of the 10 participants who had not had prior exposure to nomlabofusp only 1 experienced anaphylaxis (this reaction was one of the 7 events discussed above).

Excluding these events, long term dosing of nomlabofusp was generally well tolerated including 14 on treatment for at least 6 months and 8 on treatment for over 1 year.

The Company is implementing a modified starting dose regimen designed to mitigate the risk of anaphylaxis events as agreed to by the FDA.

The Company's nomlabofusp plans and milestones include the following:

• The Company plans to provide an update on regulatory discussions and open label study status in the first quarter of 2026.

• BLA submission seeking accelerated approval targeted in the second quarter of 2026.

• The modified starting dose regimen is also being incorporated into the global phase 3 protocol.

The Company continues to qualify sites globally and prepare for study initiation and patient enrollment.

The Company is subject to risks and uncertainties common to pre-commercial companies in the biotechnology industry, including, but not limited to, development and commercialization by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with governmental regulations, failure to secure regulatory approval for its drug candidates or any other product candidates and the ability to secure additional capital to fund its operations.

Product candidates under development will require

extensive non-clinical and clinical testing and regulatory approval prior to commercialization.

Product candidates under development will require extensive non-clinical and clinical testing and regulatory approval prior to commercialization.

These efforts require significant amounts of additional capital, adequate personnel, infrastructure and extensive compliance-reporting capabilities.

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The consolidated balance sheet as of December 31, 2024 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 June 30, 2025 and for the three and six months ended June 30, 2025 and 2024, 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 September 30, 2025 and for the three and nine months ended September 30, 2025 and 2024, 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, 2024 included in the Company’s Annual Report on Form 10-K filed with the SEC on March 24, 2025.

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, 2025, condensed consolidated results of operations for the three and six months ended June 30, 2025 and condensed consolidated statement of cash flows for the six months ended June 30, 2025 have been made.

The results of operations for the three and six months ended June 30, 2025 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2025 .

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 September 30, 2025, condensed consolidated results of operations for the three and nine months ended September 30, 2025 and condensed consolidated statement of cash flows for the nine months ended September 30, 2025 have been made.

The results of operations for the three and nine months ended September 30, 2025 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2025 .

Liquidity and Capital Resources

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

The Company has incurred net losses of $ 55.5 million and $ 36.3 million for the six months ended June 30, 2025 and 2024, respectively.

In addition, as of June 30, 2025, the Company had an accumulated deficit of $ 324.6 million .

The Company has incurred net losses of $ 103.2 million and $ 51.8 million for the nine months ended September 30, 2025 and 2024, respectively.

In addition, as of September 30, 2025, the Company had an accumulated deficit of $ 372.3 million .

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

As of June 30, 2025, the Company had approximately $ 138.5 million of cash, cash equivalents and marketable securities available for use to fund its operations and capital requirements.

As of September 30, 2025, the Company had approximately $ 175.4 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.

(“Zafgen”) and, prior to the 2020 merger with Zafgen, capital contributions from Chondrial Holdings, LLC.

Subsequent to June 30, 2025, the Company completed an underwritten public offering of common stock raising net proceeds of approximately $ 65.1 million.

On July 31, 2025, the Company completed an underwritten public offering of common stock raising net proceeds of approximately $ 65.0 million.

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 combined with the net proceeds of approximately $ 65.1 million from the Company's July 2025 public offering 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, into the fourth quarter of 2026.

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.

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, into the fourth quarter of 2026.

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 commercial and other expenditures in order to further extend cash resources.

The Company continually evaluates various financing alternatives further as discussed below.

If the Company is unable to raise sufficient additional funding prior to the issuance of its 2025 financial statements for the fiscal year ending December 31, 2025, the Company may conclude there is substantial doubt about our ability to continue as a going concern for the twelve months following the filing of its annual report on 2025 Form 10K for the fiscal year ending December 31, 2025.

The Company has not yet commercialized any products and does not expect to generate revenue from the commercial sale of any products for several years, if at all.

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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, volatility of capital markets, and other adverse macroeconomic events, including those due to inflationary pressures, rising interest rates, 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.

Summary of Significant Accounting Policies

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Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, non-clinical and clinical development activities and clinical trials as well as to manufacture clinical trial materials, and other costs.

The Company recognizes external research and development

costs based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its key service providers.

The Company recognizes external research and development costs based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its key service providers.

Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses.

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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.

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

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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For periods in which the Company has reported net losses (all periods since inception), diluted net loss per common share attributable to common stockholders is the same as basic net loss per common share attributable to common stockholders, since dilutive common stock equivalents are not assumed to have been issued if their effect is antidilutive.

The Company excluded 9,593,248 and 6,900,232 common stock equivalents outstanding as of June 30, 2025 and 2024, respectively, from the computation of diluted net loss per share for the three and six months ended

June 30, 2025 and 2024 because they had an anti-dilutive impact due to the net loss incurred for the periods presented.

The Company excluded 9,618,132 and 6,990,827 common stock equivalents outstanding as of September 30, 2025 and 2024, respectively, from the computation of diluted net loss per share for the three and nine months ended September 30, 2025 and 2024 because they had an anti-dilutive impact due to the net loss incurred for the periods presented.

Segment Information

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The Company is managed on a consolidated basis and has one operating and reportable segment related to the development of clinical and preclinical product candidates for the development of the Company’s proprietary new therapies, primarily nomlabofusp, the Company's life science segment.

The Company’s chief operating decision maker (“CODM”) is the Company's chief executive officer ("CEO").

The Company’s chief operating decision maker (“COPM”) is the Company's chief executive officer (“CEO”).

The accounting policies of the life science segment are the same as those described in the summary of significant accounting policies.

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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 and six months ended June 30, 2025 and 2024:

For the Three Months Ended June 30,

For the Six Months Ended June 30,

The table below summarizes the significant expense categories regularly provided to the CODM for the three and nine months ended September 30, 2025 and 2024:

For the Three Months Ended September 30,

For the Nine Months Ended September 30,

Operating expenses:

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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 $ 0.9 million and $ 1.0 million for the three months ended June 30, 2025 and 2024, respectively.

(b) Development expenses include research and development related stock compensation expense of approximately $ 1.8 million and $ 1.9 million for the six months ended June 30, 2025 and 2024, respectively.

(c) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 0.9 million and $ 1.3 million for the three months ended June 30, 2025 and 2024, respectively.

(d) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 1.8 million and $ 2.5 million for the six months ended June 30, 2025 and 2024 , respectively.

(a) Development expenses include research and development related stock compensation expense of approximately $ 0.9 million and $ 0.9 million for the three months ended September 30, 2025 and 2024, respectively.

(b) Development expenses include research and development related stock compensation expense of approximately $ 2.7 million and $ 2.8 million for the nine months ended September 30, 2025 and 2024, respectively.

(c) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 0.9 million and $ 1.0 million for the three months ended September 30, 2025 and 2024, respectively.

(d) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 2.7 million and $ 3.6 million for the nine months ended September 30, 2025 and 2024, respectively.

(e) Commercial expenses relate to commercial readiness activities.

Recently Issued and Adopted Accounting Pronouncements

From time to time, new accounting guidance is issued by the FASB or other standard setting bodies that is adopted by us as of the effective date or, in some cases where early adoption is permitted, in advance of the effective

We have assessed the recently issued guidance that is not yet effective and believe the new guidance will not have a material impact on the condensed consolidated results of operations, cash flows or financial position.

In November 2023, the Financial Accounting Standards Board (“FASB”) issued ASU 2023-07, Segment Reporting (Topic 280):

Improvements to Reportable Segment Disclosures (“ASU 2023-07”).

ASU 2023-07 requires disclosure of incremental segment information on an annual and interim basis.

The amendments also require companies with a single reportable segment to provide all disclosures required by this amendment and all existing segment disclosures in ASC 280, Segment Reporting.

The amendments are effective for fiscal years beginning after December 15, 2023, and interim periods beginning after December 15, 2024.

The Company adopted ASU 2023-07 , effective December 31, 2024 , in these consolidated financial statements.

ASU 2023-07 only impacted the disclosures and did not impact the consolidated financial statements.

See Note 2, Segment Information, for disclosures related to the adoption of ASU 2023-07.

Recently Issued Accounting Pronouncements Not Yet Adopted

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The ASU is effective for fiscal years beginning after December 15, 2024, with early adoption permitted.

The Company will adopt ASU-203-09 beginning with our annual reporting period ending December 31, 2025, the adoption of which is not expected to have a material impact on the Company’s consolidated results of operations or cash flows.

The Company will adopt ASU-203-09 beginning with our annual reporting period ending December 31, 2025.

The Company is currently evaluating the impact that this standard may have on its year-end financial statements.

In November 2024, the FASB issued ASU 2024-03, “Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40):

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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 and six months ended June 30, 2025 and June 30, 2024, 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 nine months ended September 30, 2025 and 2024 , 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.

On July 4, 2025, Public Law 119-21 (“The Law”) became effective.

The Law includes significant provisions, such as the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act, modifications to the international tax framework and the restoration of favorable tax treatment for certain business provisions.

The legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented through 2027.

The Company is currently assessing The Law's impact on its consolidated financial statements;

however, it does not expect The Law to have a material impact on its estimated annual effective tax rate or cash flows in 2025 .

legislation has multiple effective dates, with certain provisions effective in 2025 and others implemented through 2027.

The Law did not have a material impact on its estimated annual effective tax rate or cash flows in 2025 .

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 June 30, 2025 and December 31, 2024 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 September 30, 2025 and December 31, 2024 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 June 30, 2025 and December 31, 2024 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, 2025 and December 31, 2024:

For accounts payable and accrued liabilities, the carrying amounts of these financial instruments as of September 30, 2025 and December 31, 2024 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 September 30, 2025 and December 31, 2024:

(in thousands)

June 30, 2025

September 30, 2025

Cash equivalents:

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Total cash equivalents and marketable securities

The accrued interest receivable related to the Company’s investments was $ 1.0 million and $ 1.1 million as of June 30, 2025 and December 31, 2024, 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 $ 1.0 million and $ 1.1 million as of September 30, 2025 and December 31, 2024, respectively, and 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 June 30, 2025 and December 31, 2024, 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, 2025 and December 31, 2024 , no allowances for credit losses for the Company’s investments were recorded.

During the three and six months ended June 30, 2025 and 2024 , the Company did no t recognize any impairment losses related to investments.

As of June 30, 2025 and December 31, 2024, the Company's cash equivalents and marketable securities consisted of a U.S.

As of September 30, 2025 and December 31, 2024, 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 September 30, 2025 and December 31, 2024 , no allowances for credit losses for the Company’s investments were recorded.

During the three and nine months ended September 30, 2025 and 2024 , the Company did no t recognize any impairment losses related to investments.

As of September 30, 2025 and December 31, 2024, the Company's cash equivalents and marketable securities consisted of a U.S.

government money market fund, U.S.

4 unchanged sentences

Marketable Securities

The following table summarizes the Company's marketable securities as of June 30, 2025 and December 31, 2024:

The following table summarizes the Company's marketable securities as of September 30, 2025 and December 31, 2024:

(in thousands)

June 30, 2025

September 30, 2025

Treasury Bills

5 unchanged sentences

Total marketable securities

As of June 30, 2025 and December 31, 2024 , the Company held no investments that have been in a continuous loss position for 12 months or longer.

As of September 30, 2025 and December 31, 2024 , the Company held no investments that have been in a continuous loss position for 12 months or longer.

Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consisted of the following:

September 30,

(in thousands)

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Fixed assets, net consisted of the following:

September 30,

(in thousands)

4 unchanged sentences

Accumulated depreciation

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

Depreciation expense was $ 0.1 million for the three and six months ended June 30, 2024.

In addition, for the three and six months ended June 30, 2025 and 2024 , there was less than $ 0.1 million of depreciation related to sublet assets recorded as other expense.

Depreciation expense was $ 0.1 million and $ 0.3 million for the three and nine months ended September 30, 2025 , respectively.

Depreciation expense was $ 0.1 million and $ 0.2 million for the three and nine months ended September 30, 2024, respectively.

In addition, for the three and nine months ended September 30, 2025 and 2024 , there was less than $ 0.1 million of depreciation related to sublet assets recorded as other expense.

Accrued Expenses

Accrued expenses consisted of the following:

September 30,

(in thousands)

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The aggregate gross proceeds for the issuance and sale of the common stock and prefunded warrants were $ 80.0 million, transaction costs totaled $ 4.6 million and resulted in net proceeds of $ 75.4 million.

The Company’s Registration Statement on Form S-3, filed with the SEC on June 26, 2020, registered the resale of 6,105,359 shares of common stock sold and the 628,403

shares of common stock underlying the prefunded warrants.

The Company’s Registration Statement on Form S-3, filed with the SEC on June 26, 2020, registered the resale of 6,105,359 shares of common stock sold and the 628,403 shares of common stock underlying the prefunded warrants.

MTS Health Partners served as placement agent to the Company in connection with the private placement.

As partial compensation for these services, the Company issued MTS Health Partners 35,260 shares of common stock.

As of June 30, 2025 , 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 64,027,892 shares were issued and outstanding, and up to 5,000,000 shares of undesignated preferred stock, par value $ 0.001 per share, of which no shares were issued or outstanding.

As of September 30, 2025 , 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 85,590,392 shares were issued and outstanding, and up to 5,000,000 shares of undesignated preferred stock, par value $ 0.001 per share, of which no shares were issued or 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.

5 unchanged sentences

In July 2025, the Company completed an underwritten public offering in which the Company issued and sold 21,562,500 shares of its common stock including the exercise in full of the underwriters’ option to purchase additional shares of common stock, at a public offering price of $ 3.20 per share.

The Company received net proceeds of approximately $ 65.1 million.

The Company received net proceeds of approximately $ 65.0 million, after deducting underwriting discounts, commissions and other offering expenses.

ATM Agreement

16 unchanged sentences

As permitted by the 2020 Plan, the Company added 2,552,603 and 1,756,363 shares available for grant to the 2020 Plan on January 1, 2025 and January 1, 2024, respectively.

As of June 30, 2025 , 508,844 shares of common stock were available for grant under the 2020 Plan.

During the six months ended June 30, 2025 and twelve months ended December 31, 2024 , options to purchase 333 and 1,242 shares, respectively, issued under the Prior Plans were cancelled and became available for grant under the 2020 Plan.

As of September 30, 2025 , 483,960 shares of common stock were available for grant under the 2020 Plan.

During the nine months ended September 30, 2025 and twelve months ended December 31, 2024 , options to purchase 1,333 and 1,242 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 six months ended June 30, 2025:

September 30,

Risk-free interest rate

3 unchanged sentences

Stock Options

The following table summarizes the Company’s stock option activity for the six months ended June 30, 2025 (amounts in millions, except for share, contractual term, and per share data):

The following table summarizes the Company’s stock option activity for the nine months ended September 30, 2025 (amounts in millions, except for share, contractual term, and per share data):

Weighted Average

4 unchanged sentences

Options forfeited/expired

Outstanding as of June 30, 2025

Exercisable as of June 30, 2025

Vested and expected to vest as of June 30, 2025

(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" at June 30, 2025 .

Outstanding as of September 30, 2025

Exercisable as of September 30, 2025

Vested and expected to vest as of September 30, 2025

Option Grants

During the six months ended June 30, 2025 , the Company granted options to purchase 2,344,137 shares of common stock to employees and directors under the 2020 Plan.

During the nine months ended September 30, 2025 , the Company granted options to purchase 2,417,137 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.

2 unchanged sentences

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

The weighted-average grant date fair value of options granted under the 2020 Plan during the six months ended June 30, 2025 was $ 2.63 .

As of June 30, 2025 , total unrecognized compensation expense related to unvested stock options granted under the 2020 Plan was $ 13.3 million, which is expected to be recognized over a weighted average period of 2.72 years.

The weighted-average grant date fair value of options granted under the 2020 Plan during the nine months ended September 30, 2025 was $ 2.64 .

As of September 30, 2025 , total unrecognized compensation expense related to unvested stock options granted under the 2020 Plan was $ 12.2 million, which is expected to be recognized over a weighted average period of 2.53 years.

Inducement Stock Option Grant

There were no inducement awards granted in the six months ended June 30, 2025.

As of June 30, 2025 , total unrecognized compensation expense related to unvested inducement options granted was $ 0.6 million, which is expected to be recognized over a weighted average period of 1.79 years.

Restricted Stock Units Grants

In January 2025, RSUs were granted under the 2020 Plan to certain of the Company's employees in order to maintain retention of key employees.

There were no inducement awards granted in the nine months ended September 30, 2025.

As of September 30, 2025 , total unrecognized compensation expense related to unvested inducement options granted was $ 0.5 million, which is expected to be recognized over a weighted average period of 1.54 years.

Restricted Stock Units

In January 2025, RSUs were granted under the 2020 Plan to certain of the Company's employees in order to retain key employees.

The value of an RSU award is based on the Company's stock price on the date of grant.

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 six months ended June 30, 2025 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 nine months ended September 30, 2025 was as follows (in millions, except share, contractual term, and per share data):

Weighted Average

5 unchanged sentences

Restricted stock units forfeited

Outstanding as of June 30, 2025

Unvested and expected to vest as of June 30, 2025

Outstanding as of September 30, 2025

Unvested and expected to vest as of September 30, 2025

Restricted Stock Unit Grants

During the six months ended June 30, 2025 , the Company granted 544,988 shares of RSUs to employees under the 2020 Plan.

During the nine months ended September 30, 2025 , the Company granted 344,988 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 $ 3.46 per unit.

As of June 30, 2025 , total unrecognized compensation expense for RSUs was $ 2.8 million, which is expected to be recognized over a weighted average period of 2.53 years.

As of September 30, 2025 , total unrecognized compensation expense for RSUs was $ 2.5 million, which is expected to be recognized over a weighted average period of 2.32 years.

Performance-Based Restricted Stock Unit Grants

In January 2025, the Company granted performance-based RSUs (the "January 2025 PSU Awards") to each of the executive officers of the Company under the 2020 Plan.

In January 2025, the Company granted performance-based RSUs (the “January 2025 PSU Awards”) " to each of the four executive officers of the Company under the 2020 Plan.

Each award was expressed as a target number of RSUs.

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

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

As of September 30, 2025 , 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 nine months ended September 30, 2025.

Stock-Based Compensation

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

Three Months Ended June 30,

Six Months Ended June 30,

Three Months Ended September 30,

Nine Months Ended September 30,

Research and development

3 unchanged sentences

The Company is party to an exclusive License Agreement (the “WFUHS License”), dated November 30, 2016, as amended, with Wake Forest University Health Sciences (“WFUHS”) and an exclusive License Agreement (the “IU License”), dated November 30, 2016, as amended, with Indiana University (“IU”).

Such agreements provide for a transferable, worldwide license to certain patent rights regarding technology used by the Company with respect to the development of nomlabofusp.

Such agreements provide for a transferable, worldwide license to certain patent rights regarding technology used by the Company

with respect to the development of nomlabofusp.

Both agreements continue from their effective date through the last to date of expiration of the licensed patents, unless earlier terminated by either party in accordance with their terms.

17 unchanged sentences

The new lease on 3,462 additional square footage commenced on October 1, 2023 and the Company recorded a right of use asset and lease liability of $ 0.3 million as of that date.

The right of use assets and lease liabilities with both these leases are reflected in the financial statements for six months ended June 30, 2025 as are the right of use asset and lease liability of the Company's Boston office space discussed below.

The right of use assets and lease liabilities with both these leases are reflected in the financial statements for nine months ended September 30, 2025 as are the right of use asset and lease liability of the Company's Boston office space discussed below.

Boston Office Lease

26 unchanged sentences

Lease Expense

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

Expense arising from operating leases was $ 0.1 million and $ 0.3 million during the three and six months ended June 30, 2024, respectively.

For operating leases, the weighted-average remaining lease term for leases at June 30, 2025 and December 31, 2024 was 4.0 and 4.5 years, respectively.

For operating leases, the weighted average discount rate for leases at June 30, 2025 and December 31, 2024 was 11.0 %.

Expense arising from operating leases was $ 0.1 million and $ 0.4 million during the three and nine months ended September 30, 2025 , respectively.

Expense arising from operating leases was $ 0.1 million and $ 0.4 million during the three and nine months ended September 30, 2024, respectively.

For operating leases, the weighted-average remaining lease term for leases at September 30, 2025 and December 31, 2024 was 3.8 and 4.5 years, respectively.

For operating leases, the weighted average discount rate for leases at September 30, 2025 and December 31, 2024 was 11.0 %.

The Company has not entered into any financing leases.

Maturities of lease liabilities due under these lease agreements as of June 30, 2025 are as follows:

Maturities of lease liabilities due under these lease agreements as of September 30, 2025 are as follows:

(in thousands)

Six months ending December 31, 2025

Three months ending December 31, 2025

Year ended December 31, 2026

9 unchanged sentences

In May 2024, the Company entered into an agreement with the Friedreich’s Ataxia Research Alliance (“FARA”) to join the TRACK-FA Neuroimaging Consortium that includes pharmaceutical, biotechnology, academic and clinical partners.

The consortium will conduct a natural history study designed to establish disease-specific neuroimaging biomarkers to track disease progression in the brain and spinal cord and provide a basis for utilizing these biomarkers in clinical trials.

As an industry partner, the Company will help fund the study and contribute to the study design, research activities, and analysis.

The Company will have access to all study data for use in its regulatory filings, as appropriate.

The consortium is conducting a natural history study designed to establish disease-specific neuroimaging biomarkers to track disease progression in the brain and spinal cord and provide a basis for utilizing these biomarkers in clinical trials.

As an industry partner, the Company is helping to fund the study and contribute to the study design, research activities, and analysis.

The Company has access to all study data for use in its regulatory filings, as appropriate.

During the twelve months ended December 31, 2024, the Company incurred $ 0.9 million of costs related to the Track-FA program and will fund future costs going forward.

One of the Company’s Directors is also a director of FARA.

During the six months ended June 30, 2025 , the Company incurred less than $ 0.1 million of costs related to the Track-FA program.

Additionally, during the twelve months ended December 31, 2024, the Company sponsored patient and caregiver awareness events held by FARA for a cumulative $ 0.1 million.

During the nine months ended September 30, 2025 , the Company incurred less than $ 0.1 million of costs related to the Track-FA program.

Additionally, during three and nine months ended September 30, 2025 , the Company sponsored patient and caregiver awareness events held by FARA for a cumulative of less than $ 0.1 million and $ 0.1 million, respectively.

During the twelve months ended December 31, 2024, the Company sponsored patient and caregiver awareness events held by FARA for a cumulative $ 0.1 million.

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