What changed in this filing

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Item 1. Financial Statements

2025-08-14 compared with 2025-04-30 · 180 added, 147 removed, 414 unchanged (44% of the section changed)

Financial Statements (unaudited)

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

Condensed Consolidated Statements of Operations and Comprehensive Loss for the three months ended March 31, 2025 and 2024

Condensed Consolidated Statements of Changes in Stockholders' Equity for the three months ended March 31, 2025 and 2024

Condensed Consolidated Statements of Cash Flows for the three months ended March 31, 2025 and 2024

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

Notes to Condensed Consolidated Financial Statements

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Cash and cash equivalents

Marketable securities

Short-term marketable securities

Prepaid expenses and other current assets

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5,000,000 shares authorized

as of March 31, 2025 and December 31, 2024;

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

no shares issued and

outstanding as of March 31, 2025 and December 31, 2024

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

Common stock, $ 0.001 par value per share;

115,000,000 shares

authorized as of March 31, 2025 and December 31, 2024;

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

64,027,892 and 63,815,065 shares issued and outstanding as of

March 31, 2025 and December 31, 2024, respectively

June 30, 2025 and December 31, 2024, respectively

Additional paid-in capital

Accumulated deficit

Accumulated other comprehensive gain

Accumulated other comprehensive gain (loss)

Total stockholders’ equity

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(In thousands, except share and per share data)

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Operating expenses:

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Balances as of March 31, 2025

Stock-based compensation expense

Unrealized loss on marketable debt securities

Balances as of June 30, 2025

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

LARIMAR THERAPEUTICS, INC.

CONDENSED CONSOLIDATED STATEMENTS OF CHANGES IN

STOCKHOLDERS’ EQUITY

(In thousands, except share data)

Comprehensive

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Balances as of March 31, 2024

Stock-based compensation expense

Exercise of stock options

Unrealized loss on marketable debt securities

Balances as of June 30, 2024

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

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(In thousands)

Three Months Ended March 31,

Six Months Ended June 30,

Cash flows from operating activities:

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Purchases of marketable securities

Maturities and sales of marketable securities

Maturities of marketable securities

Net cash provided by (used in) investing activities

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Proceeds from issuance of equity securities, net of issuance costs

Proceeds from exercise of stock options and warrants

Net cash provided by financing activities

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

Offering costs included in accounts payable and accrued expense

Offering costs included in 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.

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

Larimar has completed two Phase 1 clinical trials and a Phase 2 dose exploration trial, and has an ongoing Phase 2 OLE trial in patients with FA and an ongoing run-in study in adolescent patients with FA.

In May 2021, the Company reported positive top-line data from its Phase 1 FA program after completing dosing of the single ascending dose ("SAD") trial in December 2020 and of the multiple ascending dose ("MAD") trial in March 2021.

Data from these trials demonstrated proof-of-concept by showing that daily subcutaneous injections of nomlabofusp for up to 13 days resulted in dose-dependent increases in FXN levels from baseline compared to placebo in all evaluated tissues (buccal cells, skin, and platelets).

There were no serious adverse events associated with either the MAD or SAD trials.

In May 2023, the Company reported preliminary unblinded top-line data from the 25 mg cohort of the Phase 2 four-week, placebo-controlled, dose exploration trial of nomlabofusp in FA patients.

Data from the cohort indicated nomlabofusp was generally well tolerated and showed increases in FXN levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells) at day 14.

In July 2023, the FDA cleared initiation of a second cohort (50 mg) of the four-week, placebo-controlled, Phase 2 dose exploration trial of nomlabofusp in patients with FA and the initiation of the OLE trial with daily dosing of 25 mg.

In February 2024, the Company reported positive top-line data and successful completion of the Phase 2 dose exploration study.

Nomlabofusp was generally well tolerated throughout the four-week treatment periods, had a predictable pharmacokinetic profile and led to dose dependent increases in FXN levels in all evaluated tissues (skin and buccal cells) after daily dosing of 14 days followed by every other day dosing until day 28 in the 25 mg and 50 mg cohorts.

Participants in the 25 mg (n=13) and 50 mg (n=15) cohorts were randomized 2:1 to receive subcutaneous injections of nomlabofusp or placebo.

Patients who completed treatment in the Phase 2 dose exploration study or who previously completed a prior clinical trial of nomlabofusp are eligible to screen and possibly participate in the OLE study.

In March 2024, Larimar dosed the first patient in the OLE trial, evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver.

The OLE study will evaluate the safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues as well as other exploratory pharmacodynamic markers (lipid profiles and gene expression data) following long-term subcutaneous administration of nomlabofusp.

In addition, following the completion of enrollment, as well as at least one participant completing one year of dosing, clinical assessments collected during the study will be compared to data from a matched control arm derived from participants in the Friedreich’s Ataxia Clinical Outcome Measures Study ("FACOMS") database.

On May 30, 2024, Larimar announced that the FDA's Center for Drug Evaluation and Research ("CDER") had selected nomlabofusp as one of a few programs for participation in the Support for Clinical Trials Advancing Rare Disease Therapeutics ("START") Pilot Program.

The objective of the 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 of biologics and drugs.

In September 2024, the Company received access to the Medicines and Healthcare Regulatory Agency ("MHRA”) Innovative Licensing and Access Pathway (“ILAP”) for the treatment of adults and children with FA.

The ILAP is facilitating patient access to novel treatments by accelerating time to market through opportunities for enhanced engagements with UK regulatory authorities and other stakeholders.

Along with the receipt of the ILAP designation, nomlabofusp has already been granted orphan drug designations in the U.S.

and the European Union (the “EU”), and access to the PRIME scheme in the EU.

In December 2024, the Company reported positive initial data from the ongoing OLE study.

This data included safety, FXN levels, clinical, pharmacokinetic data and dose escalation:

• Nomlabofusp was generally well tolerated for up to 260 days in subjects.

The most common adverse events were injection site reactions, with most being mild, brief in duration, and self-limited.

Two participants had serious adverse events (one seizure and one anaphylactic reaction) that resolved within 24 hours and withdrew from the study;

• Long-term 25 mg tissue FXN levels showed positive mean change from baseline of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90 and that 25 mg of nomlabofusp increased and maintained tissue FXN levels over time, increasing from a mean level of 15 % of HV at baseline to 30 % in buccal cells and from 16 % to 72 % in skin cells at Day 90;

• Tissue FXN levels appear to reach steady-state levels by Day 30 in buccal cells;

• Early clinical data showed trends toward improvement across a number of clinical outcomes for long-term 25 mg daily nomlabofusp including:

(1) decreased values indicating early trends towards improvement in the Modified Friedreich's Ataxia Rating Scale (mFARS) and the FARS-Activities of Daily Living ("ADL"), Modified Fatigue Impact Scale and 9 hole peg test at 90 days relative to baseline;

• Key pharmacokinetic data showed rapid absorption after subcutaneous administration with exposure appearing to reach steady state in plasma by day 30 with no further accumulation, which is consistent with data from our Phase 1 and Phase 2 studies.

Also in December 2024, Larimar announced that it was increasing the dose in the OLE to 50 mg of nomlabofusp daily for then currently enrolled and all future OLE study participants.

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, Larimar expects to see additional reactions.

To reduce the risk of allergic reactions, including anaphylaxis, Larimar amended the OLE protocol to administer premedication for the first month of dosing.

The OLE study is ongoing with seven sites activated and participants continuing to enroll.

Active study participants are currently receiving the 50 mg dose of nomlabofusp.

The Company plans to provide an update on OLE data on at least 30 to 40 study participants, some of whom have been receiving nomlabofusp for more than a year, in September 2025.

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

Study participants in the PK run-in study 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.

Dosing of this adolescent PK run-in study was completed in March 2025.

Following assessment of safety and PK data, participants will be eligible to screen for the OLE study.

The data from this cohort is expected to be presented in the nomlabofusp program update in September 2025.

In February 2025, FDA accepted the data supporting the comparability of the lyophilized drug product to the frozen solution and agreed with Larimar’s plans to introduce the lyophilized product into its clinical development program in mid-2025.

The frozen solution is the dosage form currently being used in the OLE.

The lyophilized drug product is the formulation that Larimar intends to commercialize.

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’s concentration as an RLSE to support approval will be a matter of review in a future marketing application.

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).

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.

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 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”).

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 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.

• 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 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.

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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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, including seeking feedback on the adequacy of the safety data set for a BLA submission seeking accelerated approval targeted for the end of 2025.

Additionally, the Company has obtained feedback from both FDA and EMA on the global Phase 3 study protocol, and the Company is on track to initiate the study by mid-2025 with potential sites in the U.S., E.U., U.K, Canada, and Australia.

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 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.

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.

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.

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.

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Generally Accepted Accounting Principles ("GAAP").

The condensed 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 March 31, 2025 and for the three months ended March 31, 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 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.

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 March 31, 2025, condensed consolidated results of operations for the three months ended March 31, 2025 and condensed consolidated statement of cash flows for the three months ended March 31, 2025 have been made.

The results of operations for the three months ended March 31, 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 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 .

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 $ 29.3 million and $ 14.7 million for the three months ended March 31, 2025 and 2024, respectively.

In addition, as of March 31, 2025, the Company had an accumulated deficit of $ 298.4 million .

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

As of March 31, 2025, the Company had approximately $ 157.5 million of cash, cash equivalents and marketable securities available for use to fund its operations and capital requirements.

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.

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.

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 second 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 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.

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 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.

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

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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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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 9,564,550 and 6,555,145 common stock equivalents outstanding as of March 31, 2025 and 2024, respectively, from the computation of diluted net loss per share for the three months ended March 31, 2025 and 2024 because they had an anti-dilutive impact due to the net loss incurred for the periods presented.

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.

Segment Information

Operating segments are defined as components of an enterprise for which separate discrete information is available for evaluation by the chief operating decision maker, or decision making group, in deciding how to

allocate resources in assessing performance.

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.

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

The table below summarizes the significant expense categories regularly provided to the CODM for the three months ended March 31, 2025 and 2024:

For the Three Months Ended March 31,

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

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Operating expenses:

Technical operations

Development (a)

Development (a)(b)

Nomlabofusp support

General and administrative (b)

General and administrative (c)(d)

Total operating expenses

Other income, net

(a) Development expenses include research and development related stock compensation expense of approximately $ 0.9 million for the three months ended March 31, 2025 and 2024, respectively.

(b) General and administrative expenses include general and administrative related stock compensation expense of approximately $ 0.9 million and $ 1.2 million for the three months ended March 31, 2025 and 2024 , respectively.

(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.

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 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 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):

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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.

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

The Company has determined it will adopt this ASU on January 1, 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 adoption of which is not expected to have a material impact on the Company’s consolidated results of operations or cash flows.

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

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The Company will evaluate the impact adopting ASU 2024-03 will have on the Company's consolidated financial statements and disclosures.

We recognize deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the financial reporting and tax basis of assets and liabilities, as well as for operating loss and tax credit carryforwards.

Deferred tax assets and liabilities are measured using the tax rates that are expected to apply to taxable income for the years in which those tax assets and liabilities are expected to be realized or settled.

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

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 .

Fair Value Measurements and Marketable Securities

Fair Value Measurements

The Company’s assets and liabilities that are measured at fair value on a recurring basis as of March 31, 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 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 valuation hierarchy is based on the transparency of inputs to the valuation of an asset or liability as of the measurement date.

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

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

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:

(in thousands)

March 31, 2025

June 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 $ 1.1 million as of March 31, 2025 and December 31, 2024, respectively, and is included in prepaid expenses and other current assets on the condensed consolidated balance sheets.

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 Company classifies its money market funds and U.S.

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

As of March 31, 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 March 31, 2025 and December 31, 2024 , no allowances for credit losses for the Company’s

investments were recorded.

During the three months ended March 31, 2025 and 2024 , the Company did no t recognize any impairment losses related to investments.

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

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.

government money market fund, U.S.

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Marketable Securities

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

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

(in thousands)

March 31, 2025

June 30, 2025

Treasury Bills

5 unchanged sentences

Total marketable securities

As of March 31, 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 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.

Prepaid Expenses and Other Current Assets

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Accumulated depreciation

Depreciation expense was $ 0.1 million for each of the three months ended March 31, 2025 and 2024.

In addition, for the three months ended March 31, 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 $ 0.2 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.

Accrued Expenses

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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 March 31, 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 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.

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

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In 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.

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 $ 65.1 million.

ATM Agreement

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

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

restricted stock units, and cash or other stock-based awards.

ISOs may be granted only to the Company’s employees, including the Company’s officers, and the employees of the Company’s affiliates.

4 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 March 31, 2025 , 537,542 shares of common stock were available for grant under the 2020 Plan.

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

No such options were cancelled in the three months ended March 31, 2025.

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.

Stock Option Valuation

The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees during the three months ended March 31, 2025:

Risk-free interest rate

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Stock Options

The following table summarizes the Company’s stock option activity for the three months ended March 31, 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 six months ended June 30, 2025 (amounts in millions, except for share, contractual term, and per share data):

Weighted Average

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Options forfeited/expired

Outstanding as of March 31, 2025

Exercisable as of March 31, 2025

Vested and expected to vest as of March 31, 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 March 31, 2025 .

Outstanding as of June 30, 2025

Exercisable as of June 30, 2025

Vested and expected to vest as of June 30, 2025

Option Grants

During the three months ended March 31, 2025 , the Company granted options to purchase 2,130,437 shares of common stock to employees 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, 2025 was $ 2.74 .

As of March 31, 2025 , 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 2.91 years.

Inducement Stock Option Grants

There were no inducement awards granted in the three months ended March 31, 2025.

As of March 31, 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 2.04 years.

Restricted Stock Units

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.

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

Of the 2,344,137 options granted, 2,249,137 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 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.

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.

1 unchanged sentence

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

Activity with respect to the Company's RSUs and performance-based RSUs during the three months ended March 31, 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 six months ended June 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 March 31, 2025

Unvested and expected to vest as of March 31, 2025

Outstanding as of June 30, 2025

Unvested and expected to vest as of June 30, 2025

Restricted Stock Unit Grants

During the three months ended March 31, 2025 , the Company granted 344,988 shares of RSUs, which vest annually over four years to employees under the 2020 Plan.

The RSUs have a weighted-average grant date fair value of $ 3.46 per unit.

As of March 31, 2025 , total unrecognized compensation expense for RSUs was $ 3.2 million, which is expected to be recognized over a weighted-average period of 2.76 years.

During the six months ended June 30, 2025 , the Company granted 544,988 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.

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

As of June 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 March 31, 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 months ended March 31, 2025.

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.

Stock-Based Compensation

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

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Research and development

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 of these leases are reflected in the financial statements for three months ended March 31, 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 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.

Boston Office Lease

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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 alternate lab space in King of Prussia, Pennsylvania for a period of four year s.

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 .

Due to required tenant improvements to be completed by the landlord, the Company did not take immediate possession of the leased property.

2 unchanged sentences

Lease Expense

Expense arising from operating leases was $ 0.1 million during each of the three months ended March 31, 2025 and 2024.

For operating leases, the weighted-average remaining lease term for leases at March 31, 2025 and December 31, 2024 was 4.2 and 4.5 years, respectively.

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

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 %.

The Company has not entered into any financing leases.

Maturities of lease liabilities due under these lease agreements as of March 31, 2025 are as follows:

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

(in thousands)

Nine months ending December 31, 2025

Six months ending December 31, 2025

Year ended December 31, 2026

14 unchanged sentences

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

During the three months ended March 31, 2025 , the Company incurred less than $ 0.1 million of costs related to the Track-FA program.

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.

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