What changed in this filing

/stocks — the workspaceLOADING

Item 1. Financial Statements

2026-05-14 compared with 2025-11-05 · 155 added, 249 removed, 346 unchanged (54% of the section changed)

Financial Statements (unaudited)

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

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

Notes to Condensed Consolidated Financial Statements

13 unchanged sentences

(In thousands, except share and per share data)

September 30,

Current assets:

Cash and cash equivalents

Short-term marketable securities

Marketable securities

Prepaid expenses and other current assets

16 unchanged sentences

5,000,000 shares authorized

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

no shares issued and

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

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

500,000 and 250,000

shares issued and outstanding as of March 31, 2026

and December 31, 2025, respectively

Common stock, $ 0.001 par value per share;

115,000,000 shares

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

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

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

September 30, 2025 and December 31, 2024, respectively

March 31, 2026 and December 31, 2025, respectively

Additional paid-in capital

7 unchanged sentences

(In thousands, except share and per share data)

Three Months Ended September 30,

Nine Months Ended September 30,

Three Months Ended March 31,

Operating expenses:

4 unchanged sentences

Other income, net

Net loss per share, basic and diluted

Weighted average common shares outstanding, basic and diluted

Comprehensive loss:

Other comprehensive loss:

Unrealized gain (loss) on marketable securities

Total other comprehensive gain (loss)

Unrealized loss on marketable securities

Total other comprehensive loss

Total comprehensive loss

Basic and diluted net loss per share:

Preferred stock

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

Preferred stock

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

3 unchanged sentences

(In thousands, except share data)

Preferred Stock

Comprehensive

1 unchanged sentence

Balances as of December 31, 2025

Issuance of common stock, net of issuance costs

Issuance of preferred stock in exchange for common stock

Vesting of restricted stock units

Stock-based compensation expense

Unrealized loss on marketable securities

Balances as of March 31, 2025

Exercise of stock options

Stock-based compensation expense

Unrealized loss on marketable debt securities

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

Balances as of March 31, 2026

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

6 unchanged sentences

Balances as of December 31, 2024

Issuance of common stock, net

Vesting of restricted stock units

Exercise of stock options

Stock-based compensation expense

1 unchanged sentence

Balances as of March 31, 2025

Stock-based compensation expense

Exercise of stock options

Unrealized loss on marketable debt securities

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.

2 unchanged sentences

(In thousands)

Nine Months Ended September 30,

Operating activities:

Three Months Ended March 31,

Cash flows from operating activities:

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

8 unchanged sentences

Net cash used in operating activities:

Investing activities:

Cash flows from investing activities:

Purchases of property and equipment

2 unchanged sentences

Net cash provided by (used in) investing activities

Financing activities:

Proceeds from issuance of common stock, net of issuance costs

Proceeds from exercise of stock options and warrants

Cash flows from financing activities:

Proceeds from issuance of equity securities

Proceeds from exercise of stock options

Net cash provided by financing activities

3 unchanged sentences

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 accounts payable and accrued expense

Leased assets obtained in exchange for new operating lease liabilities

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

3 unchanged sentences

Larimar Therapeutics, Inc., together with its subsidiary (the “Company” or “Larimar”), is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using its novel cell penetrating peptide technology platform.

Larimar's lead product candidate, nomlabofusp (nomlabofusp is the International Nonproprietary Name and the United States Adopted Name for CTI-1601), is a subcutaneously administered, recombinant fusion protein intended to deliver human frataxin (“FXN”), an essential protein, to the mitochondria of patients with Friedreich’s ataxia (“FA”).

Larimar's lead product candidate, nomlabofusp, is a subcutaneously administered, recombinant fusion protein intended to deliver human frataxin (“FXN”), an essential protein, to the mitochondria of patients with Friedreich's Ataxia (“FA”).

FA is a rare, progressive, and fatal disease in which patients are unable to produce sufficient FXN due to a genetic abnormality.

Larimar initiated human clinical studies of nomlabofusp in 2019.

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 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 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 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 to expedite the review process.

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 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.

Recent significant developments are as follows:

• In December 2024, the Company reported positive initial data from its ongoing open label study.

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

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.

The Company completed dosing of 14 adolescents in March 2025.

• In February 2025, FDA accepted the data supporting the comparability of the lyophilized drug product to the frozen solution and agreed with the Company's plans to introduce the lyophilized product into its clinical development program.

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

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.

FDA acknowledged that data the Company recently submitted appear to support a relationship between increased FXN concentrations in skin cells and relevant tissues such as the heart, dorsal root ganglion and skeletal muscle.

FDA also acknowledged that the nonclinical studies the Company submitted were performed at relevant human doses.

FDA also suggested that the Company consider exploring the relationship between increases in FXN in skin and changes in pharmacodynamic (“PD”) markers such as lipid profiles and/or clinical measures to provide additional support for the use of FXN as an RLSE.

The Company continues to interact with the FDA under the START pilot program.

• 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 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.

• 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.

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

Even if the Company's drug development efforts are successful, it is uncertain when, if ever, it will realize significant revenue from product sales.

Basis of Presentation

4 unchanged sentences

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 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.

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.

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.

1 unchanged sentence

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 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 .

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 .

Liquidity and Capital Resources

1 unchanged sentence

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

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

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.

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.

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.

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

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 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, 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.

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

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 fixed 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.

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 will be able to raise sufficient additional capital on acceptable terms, if at all.

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.

Summary of Significant Accounting Policies

27 unchanged sentences

The expected dividend yield considers the fact that the Company has never paid cash dividends on common stock and does not expect to pay any cash dividends in the foreseeable future.

Net Loss Per Share

Basic net loss per share is computed by dividing the net loss attributable to common stockholders by the weighted average number of common shares outstanding for the period.

Diluted net loss per share attributable to common stockholders is computed by dividing the diluted net loss attributable to common stockholders by the weighted average number of common shares, including potentially dilutive common stock equivalents assuming the dilutive effect of outstanding stock options, outstanding restricted stock units, and unvested restricted common shares, as determined using the treasury stock method.

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 9,618,132 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

1 unchanged sentence

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 (“COPM”) is the Company's chief executive officer (“CEO”).

The Company’s chief operating decision maker (“CODM”) 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.

5 unchanged sentences

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 nine months ended September 30, 2025 and 2024:

For the Three Months Ended September 30,

For the Nine Months Ended September 30,

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,

Operating expenses:

Technical operations

Development (a)(b)

Development (a)

Nomlabofusp support

General and administrative (c)(d)

Commercial (e)

General and administrative (b)

Commercial (c)

Total operating expenses

Other income, net

(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.

(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.

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 date.

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.

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 consolidated results of operations, cash flows or financial position.

Recently Issued Accounting Pronouncements Not Yet Adopted

In December 2023, the FASB issued ASU No.

2023-09, “Improvements to Income Tax Disclosures (Topic 740),” which requires entities to disclose in their rate reconciliation table additional categories of information about federal, state and foreign income taxes as well as additional information about reconciling items if certain quantitative thresholds are met.

This ASU will require all entities to disclose income taxes paid, net of refunds, disaggregated by federal (national), state and foreign taxes for annual periods and to disaggregate the information by jurisdiction based on a quantitative threshold.

All entities are required to apply the guidance prospectively, with the option to apply it retrospectively.

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 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):

6 unchanged sentences

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 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.

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 .

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.

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

4 unchanged sentences

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 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:

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:

(in thousands)

September 30, 2025

March 31, 2026

Cash equivalents:

2 unchanged sentences

Marketable securities:

Treasury Bills

Government securities

6 unchanged sentences

Marketable securities:

Treasury Bills

Government securities

1 unchanged sentence

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 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 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 Company classifies its money market funds and U.S.

5 unchanged sentences

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 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.

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.

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.

1 unchanged sentence

Marketable Securities

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

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

(in thousands)

September 30, 2025

Treasury Bills

March 31, 2026

Government securities

1 unchanged sentence

December 31, 2025

Treasury Bills

Government securities

Total marketable securities

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.

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.

Prepaid Expenses and Other Current Assets

Prepaid expenses and other current assets consisted of the following:

September 30,

(in thousands)

4 unchanged sentences

Fixed assets, net consisted of the following:

September 30,

(in thousands)

4 unchanged sentences

Accumulated depreciation

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.

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.

Accrued Expenses

Accrued expenses consisted of the following:

September 30,

(in thousands)

3 unchanged sentences

Stockholders’ Equity and Stock Options

Common Stock and Prefunded Warrants

On May 28, 2020, the Company entered into a securities purchase agreement with certain accredited investors (the “Purchasers”) for the sale by the Company in a private placement of 6,105,359 shares of the Company’s common stock and prefunded warrants to purchase an aggregate of 628,403 shares of the Company’s common stock, for a price of $ 11.88 per share of the common stock and $ 11.87 per prefunded warrant.

The prefunded warrants were exercisable at an exercise price of $ 0.01 and were exercisable indefinitely.

In August 2023, the 628,403 shares of prefunded warrants were exercised and the Company received cash proceeds of six thousand two hundred and eighty-four dollars .

The private placement closed on June 1, 2020.

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.

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 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.

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.

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.

2 unchanged sentences

No cash dividends have been declared or paid to date.

In February 2024, the Company completed an underwritten public offering in which the Company issued and sold 19,736,842 shares of its common stock at a public offering price of $ 8.74 per share.

The Company received net proceeds of approximately $ 161.8 million after deducting underwriting discounts, commissions and other offering expenses.

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 $ 65.0 million, after deducting underwriting discounts, commissions and other offering expenses.

In July 2025, the Company sold 21,562,500 shares of its common stock in an underwritten public offering at a price of $ 3.20 per share and received net proceeds of approximately $ 65.0 million.

In February 2026, the Company sold 23,000,000 shares of its common stock in an underwritten public offering at a price of $ 5.00 per share and received net proceeds of approximately $ 107.6 million.

ATM Agreement

In November 2022, the Company entered into a sales agreement (the “2022 ATM Agreement”) with Guggenheim Securities, LLC in connection with the establishment of an “at-the-market” offering program under which the Company could sell up to an aggregate of $ 50.0 million of shares of common stock (the “ATM Shares”) from time to time.

In February 2024, in connection with the underwritten public offering described above, the Company terminated the 2022 ATM Agreement.

No ATM Shares were ever sold pursuant to the 2022 ATM Agreement.

In May 2024, the Company entered into a sales agreement (the "ATM Agreement") with Guggenheim Securities, LLC in connection with the establishment of an “at-the-market” offering program under which the Company could sell up to an aggregate of $ 100 million of shares of common stock (the “ATM Shares”) from time to time.

To date, no sales of common stock have been made under the 2024 ATM Agreement.

To date, no sales of common stock have been made under this ATM Agreement.

Preferred Stock

In December 2025, the Company entered into an exchange agreement (the “Exchange Agreement”) with Blue Owl Healthcare Opportunities IV Public Investments LP (the “Stockholder”) pursuant to which the Stockholder exchanged 2,500,000 shares of the Company’s common stock for 250,000 shares of newly designated Series A Convertible Preferred Stock of par value $ 0.001 per share ( the “Series A Preferred Stock”).

In January 2026, the Stockholder exchanged an additional 2,500,000 shares of the Company’s common stock for an additional 250,000 shares of Series A Preferred Stock.

Each share of the Series A Preferred Stock will be convertible into ten shares of common stock at the option of the holder at any time, subject to certain limitations, including that the holder will be prohibited from converting preferred stock into common stock if, as a result of such conversion, the holder, together with its affiliates, would beneficially own a number of shares of common stock more than 9.99 % of the total common stock then issued and outstanding immediately following the conversion of such shares of Series A Preferred Stock.

Holders of the Series A Preferred Stock are permitted to increase this percentage to an amount not to exceed 19.99 % upon 60 days’ notice.

Shares of Series A Preferred Stock will generally have no voting rights , except as required by law and except that the consent of a majority of the holders of the outstanding Series A Preferred Stock will be required to amend the terms of the preferred stock.

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.

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

The Series A Preferred Stock ranks on parity with the common stock and any class or series of capital stock of the Company created specifically ranking by its terms on parity with the preferred stock.

Additionally, it ranks senior to any class or series of capital stock of the Company hereafter created specifically ranking by its terms junior to the preferred stock and junior to any class or series of capital stock of Larimar created specifically ranking by its terms senior to any preferred stock, in each case, as to distributions of assets upon liquidation, dissolution or winding up of the Company, whether voluntarily or involuntarily.

2020 Equity Incentive Plan

10 unchanged sentences

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 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.

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.

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 nine months ended September 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 nine months ended September 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 three months ended March 31, 2026 (amounts in millions, except for share, contractual term, and per share data):

Weighted Average

3 unchanged sentences

Options granted

Options exercised

Options forfeited/expired

Outstanding as of September 30, 2025

Exercisable as of September 30, 2025

Vested and expected to vest as of September 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 September 30, 2025 .

Outstanding as of March 31, 2026

Exercisable as of March 31, 2026

Vested and expected to vest as of March 31, 2026

Option Grants

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.

Of the 2,417,137 options granted, 2,322,137 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 95,000 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 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 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.

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.

Inducement Stock Option Grant

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.

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.

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.

RSUs are 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 nine months ended September 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 three months ended March 31, 2026 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 September 30, 2025

Unvested and expected to vest as of September 30, 2025

Outstanding as of March 31, 2026

Unvested and expected to vest as of March 31, 2026

Restricted Stock Unit Grants

During the nine months ended September 30, 2025 , the Company granted 344,988 shares of RSUs to employees under the 2020 Plan.

During the three months ended March 31, 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 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.

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.

Performance-Based Restricted Stock Unit Grants

2 unchanged sentences

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

As of March 31, 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 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.

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.

Stock-Based Compensation

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

Three Months Ended September 30,

Nine Months Ended September 30,

Three Months Ended March 31,

Research and development

2 unchanged sentences

Intellectual Property Licenses

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.

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.

In partial consideration for the right and license granted under these agreements, the Company will pay each of WFUHS and IU a royalty of a low single digit percentage of net sales of licensed products depending on whether there is a valid patent covering such products.

As additional consideration for these agreements, the Company is obligated to pay each of WFUHS and IU certain milestone payments of up to $ 2.6 million in the aggregate upon the achievement of certain developmental milestones, which commenced with the enrollment of the first patient in a Phase 1 clinical trial.

The Company enrolled the first patient in its SAD trial on December 11, 2019 and paid WFUHS and IU less than $ 0.1 million.

The Company will also pay each of WFUHS and IU sublicensing fees ranging from a high-single digit to a low double-digit percentage of sublicense consideration depending on the Company’s achievement of certain regulatory milestones as of the time of receipt of the sublicense consideration.

The Company is also obligated to reimburse WFUHS and IU for patent-related expenses.

The Company is party to an exclusive License Agreement (the "IU License"), dated November 30, 2016, as amended, with Indiana University (“IU”).

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

In partial consideration for the right and license granted under the agreement, the Company will pay IU a royalty of a low single digit percentage of net sales of licensed products depending on whether there is a valid patent covering such products.

As additional consideration for these agreements, the Company is obligated to pay IU certain milestone payments of up to $ 2.0 million in the aggregate upon the achievement of certain developmental milestones, which commenced with the enrollment of the first patient in a Phase 1 clinical trial.

The Company enrolled the first patient in its SAD trial on December 11, 2019 and paid IU less than $ 0.1 million.

The Company will also pay IU sublicensing fees ranging from a high-single digit to a low double-digit percentage of sublicense consideration depending on the Company’s achievement of certain regulatory milestones as of the time of receipt of the sublicense consideration.

The Company is also obligated to reimburse IU for patent-related expenses.

In the event that the Company disputes the validity of any of the licensed patents, the royalty rate would be tripled during such dispute.

The Company is also obligated to pay to IU a minimum annual royalty of less than $ 0.1 million per annum.

In the event that the Company is required to pay IU consideration, then the Company may deduct 20 % of such IU consideration on a dollar-for-dollar basis from the consideration due to WFUHS.

In the event that the Company is required to pay WFUHS consideration, then the Company may deduct 60 % of such WFUHS consideration on a dollar-for-dollar basis from the consideration due to IU.

In October 2022, the Company initiated dosing of a Phase 2 study.

Pursuant to the terms of both the WFUHS License and the IU License, the company recognized milestone expense of $ 0.3 million within research and development expenses.

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

Pursuant to the terms of the IU License, the company recognized milestone expense of $ 0.1 million within research and development expenses.

The agreement continues from its effective date through the last to date of expiration of the licensed patent, unless earlier terminated by either party in accordance with the terms of the agreement.

We were previously a party to the License Agreement dated November 30, 2016, with Wake Forest University Health Sciences (“WFUHS”), which licensed certain patent rights regarding technology used by us with respect to the development of nomlabofusp in consideration for an obligation to pay WFUHS a royalty of a low single digit percentage of net sales of licensed products, certain milestones, and certain sublicensing revenue.

The WFUHS license expired in December 2025 with the last to expire licensed patent.

Bala Cynwyd Office Space

On August 8, 2019, the Company entered into an operating lease for office space in Bala Cynwyd, Pennsylvania, effective as of December 15, 2019, for a period of three years and six months with an option to extend the lease for three additional years.

On August 8, 2019, the Company entered into an operating lease for 4,642 square footage of office space in Bala Cynwyd, Pennsylvania, effective as of December 15, 2019, for a period of three years and six months with an option to extend the lease for three additional years.

Due to required tenant improvements to be completed by the landlord, the Company did not take immediate possession of the leased property and the lease term commenced on February 15, 2020 .

2 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 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.

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.

Boston Office Lease

16 unchanged sentences

The Company records sublease income on this sublease on a straight-line basis as a component of other income/(expense).

On November 5, 2018, the Company entered into an operating lease for office and lab space in Philadelphia, Pennsylvania, effective as of January 1, 2019 , and expiring on December 31, 2020 with an option to extend the lease for two additional years.

On August 4, 2020, the Company executed the first option to extend the lease for an additional year, expiring on December 31, 2021 .

On August 9, 2021, the Company executed the remaining option to extend the lease for an additional year, expiring on December 31, 2022 .

In September 2023, the Company extended this lease for an additional year with the option to terminate with four months' notice.

On March 28, 2024, the Company gave the requisite notice and vacated the property in May 2024.

On October 16, 2023, the Company entered into an operating lease for lab space in King of Prussia, Pennsylvania for a period of four years .

3 unchanged sentences

Lease Expense

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 %.

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 %.

The Company has not entered into any financing leases.

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

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

(in thousands)

Three months ending December 31, 2025

Year ended December 31, 2026

Nine 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.

Related Party

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 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 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.

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).

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.

Allocation of net loss to common

Allocation of net loss to Series A Preferred

Weighted average shares of common stock outstanding, basic and diluted

Weighted average shares of Series A Preferred outstanding, basic and diluted

Net loss per share of common stock, basic and diluted

Net loss per share of Series A Preferred, basic and diluted

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.

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,

Options to purchase common stock

Unvested restricted common stock

Unvested performance-based restricted common stock

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