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Description of Business and Basis of Presentation | 9 Months Ended
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Sep. 30, 2025
Organization, Consolidation and Presentation of Financial Statements [Abstract] |
Description of Business and Basis of Presentation | 1. Description of Business and Basis of Presentation 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”). 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 The condensed consolidated financial statements include the accounts of Larimar and its wholly owned subsidiary. All intercompany balances and transactions have been eliminated. The accompanying condensed consolidated financial statements have been prepared in conformity with U.S. Generally Accepted Accounting Principles (“GAAP”). 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 September 30, 2025 and for the three and nine months ended September 30, 2025 and 2024, have been prepared by the Company pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to such rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. 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 September 30, 2025, condensed consolidated results of operations for the three and nine months ended September 30, 2025 and condensed consolidated statement of cash flows for the nine months ended September 30, 2025 have been made. The results of operations for the three and nine months ended September 30, 2025 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2025. Liquidity and Capital Resources The Company’s condensed consolidated financial statements have been presented on the basis that it will continue as a going concern, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. Since its inception, the Company has incurred significant recurring operating losses and negative cash flows from operations. The Company has incurred net losses of $103.2 million and $51.8 million for the nine months ended September 30, 2025 and 2024, respectively. In addition, as of September 30, 2025, the Company had an accumulated deficit of $372.3 million. The Company expects to continue to generate operating losses for the foreseeable future. As of September 30, 2025, the Company had approximately $175.4 million of cash, cash equivalents and marketable securities available for use to fund its operations and capital requirements. The Company has funded its operations to date primarily with proceeds from sales of common stock and proceeds from the sale of prefunded warrants for the purchase of common stock, the acquisition in 2020 of cash, cash equivalents and marketable securities upon the merger with Zafgen, Inc. (“Zafgen”) and, prior to the 2020 merger with Zafgen, capital contributions from Chondrial Holdings, LLC. On July 31, 2025, the Company completed an underwritten public offering of common stock raising net proceeds of approximately $65.0 million. In accordance with Accounting Standards Update (“ASU”) No. 2014-15, “Disclosure of Uncertainties about an Entity’s Ability to Continue as a Going Concern”, the Company has evaluated whether there are certain conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that these condensed consolidated financial statements are issued. As of the issuance date of these condensed consolidated financial statements, the Company expects its cash, cash equivalents and marketable securities 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. 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. 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. 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.

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