Larimar Therapeutics Reports First Quarter 2025 Financial Results
Larimar Therapeutics reported Q1 2025 financial results with significant progress in their nomlabofusp clinical development program for Friedreich's Ataxia (FA). The FDA has shown openness to considering skin FXN concentration as a surrogate endpoint for accelerated approval, with a BLA submission planned for year-end 2025.
Key developments include completion of adolescent dosing in the PK run-in study, plans for a global Phase 3 study in mid-2025, and an upcoming program update in September 2025 featuring OLE study data. The company maintains a strong financial position with $157.5 million in cash and equivalents, providing runway into Q2 2026.
Financial highlights show a Q1 2025 net loss of $29.3 million ($0.46 per share), compared to $14.7 million in Q1 2024. R&D expenses increased to $26.6 million, while G&A expenses rose to $4.6 million. The company is advancing toward potential registration of the first disease-modifying therapy for FA patients.
Larimar Therapeutics ha comunicato i risultati finanziari del primo trimestre 2025, segnando progressi significativi nel loro programma di sviluppo clinico di nomlabofusp per l'atassia di Friedreich (FA). La FDA ha mostrato apertura nel considerare la concentrazione cutanea di FXN come endpoint surrogato per un'approvazione accelerata, con la presentazione della BLA prevista per la fine del 2025.
Tra gli sviluppi principali vi sono il completamento della somministrazione negli adolescenti nello studio PK run-in, i piani per uno studio globale di Fase 3 a metà 2025 e un aggiornamento del programma previsto per settembre 2025, che includerà dati dallo studio OLE. L'azienda mantiene una solida posizione finanziaria con 157,5 milioni di dollari in contanti e equivalenti, garantendo risorse fino al secondo trimestre 2026.
I dati finanziari evidenziano una perdita netta di 29,3 milioni di dollari nel primo trimestre 2025 (0,46 dollari per azione), rispetto a 14,7 milioni nel primo trimestre 2024. Le spese di R&S sono aumentate a 26,6 milioni, mentre le spese generali e amministrative sono salite a 4,6 milioni. L'azienda sta avanzando verso la possibile registrazione della prima terapia modificante la malattia per i pazienti con FA.
Larimar Therapeutics informó los resultados financieros del primer trimestre de 2025, con avances significativos en su programa clínico de desarrollo de nomlabofusp para la Ataxia de Friedreich (FA). La FDA ha mostrado disposición para considerar la concentración de FXN en la piel como un endpoint sustituto para la aprobación acelerada, con la presentación de la BLA planeada para finales de 2025.
Los desarrollos clave incluyen la finalización de la dosificación en adolescentes en el estudio PK run-in, planes para un estudio global de Fase 3 a mediados de 2025 y una próxima actualización del programa en septiembre de 2025 con datos del estudio OLE. La compañía mantiene una sólida posición financiera con 157,5 millones de dólares en efectivo y equivalentes, asegurando recursos hasta el segundo trimestre de 2026.
Los aspectos financieros muestran una pérdida neta en el primer trimestre de 2025 de 29,3 millones de dólares (0,46 dólares por acción), en comparación con 14,7 millones en el primer trimestre de 2024. Los gastos de I+D aumentaron a 26,6 millones, mientras que los gastos generales y administrativos crecieron a 4,6 millones. La compañía avanza hacia el posible registro de la primera terapia modificadora de la enfermedad para pacientes con FA.
라리마 테라퓨틱스는 2025년 1분기 재무 결과를 발표하며 프리드라이히 운동실조증(FA)을 위한 놈라보푸스프 임상 개발 프로그램에서 상당한 진전을 이루었다고 밝혔다. FDA는 가속 승인용 대체 평가변수로 피부 내 FXN 농도를 고려하는 데 열린 태도를 보였으며, 2025년 말까지 BLA 제출을 계획하고 있다.
주요 진전 사항으로는 PK 런인 연구에서 청소년 투약 완료, 2025년 중반 글로벌 3상 시험 계획, 2025년 9월 OLE 연구 데이터를 포함한 프로그램 업데이트 예정 등이 있다. 회사는 현금 및 현금성 자산 1억 5750만 달러를 보유하여 2026년 2분기까지 재정적 안정성을 유지하고 있다.
재무 하이라이트는 2025년 1분기 순손실이 2930만 달러(주당 0.46달러)로, 2024년 1분기 1470만 달러에 비해 증가했음을 보여준다. 연구개발비는 2660만 달러로 증가했고, 일반관리비는 460만 달러로 상승했다. 회사는 FA 환자를 위한 최초의 질병 변형 치료제 등록을 향해 나아가고 있다.
Larimar Therapeutics a annoncé ses résultats financiers du premier trimestre 2025, marquant des progrès significatifs dans leur programme de développement clinique de nomlabofusp pour l'ataxie de Friedreich (FA). La FDA s'est montrée ouverte à considérer la concentration cutanée de FXN comme un critère de substitution pour une approbation accélérée, avec une soumission de BLA prévue pour la fin de l'année 2025.
Les développements clés incluent la fin de la phase de dosage chez les adolescents dans l'étude PK run-in, des plans pour une étude mondiale de phase 3 à la mi-2025, et une mise à jour du programme prévue en septembre 2025 présentant les données de l'étude OLE. L'entreprise conserve une solide position financière avec 157,5 millions de dollars en liquidités et équivalents, assurant un financement jusqu'au deuxième trimestre 2026.
Les points financiers montrent une perte nette de 29,3 millions de dollars au premier trimestre 2025 (0,46 dollar par action), contre 14,7 millions au premier trimestre 2024. Les dépenses de R&D ont augmenté à 26,6 millions, tandis que les frais généraux et administratifs ont grimpé à 4,6 millions. L'entreprise progresse vers l'enregistrement potentiel du premier traitement modifiant la maladie pour les patients atteints de FA.
Larimar Therapeutics veröffentlichte die Finanzergebnisse für das erste Quartal 2025 und meldete bedeutende Fortschritte in ihrem klinischen Entwicklungsprogramm für Nomlabofusp bei Friedreich-Ataxie (FA). Die FDA zeigte sich offen dafür, die FXN-Konzentration in der Haut als Surrogat-Endpunkt für eine beschleunigte Zulassung zu berücksichtigen, mit einer geplanten BLA-Einreichung Ende 2025.
Wichtige Entwicklungen umfassen den Abschluss der Dosierung bei Jugendlichen in der PK-Run-in-Studie, Pläne für eine globale Phase-3-Studie Mitte 2025 sowie ein bevorstehendes Programm-Update im September 2025 mit Daten aus der OLE-Studie. Das Unternehmen verfügt über eine starke Finanzlage mit 157,5 Millionen US-Dollar an liquiden Mitteln und Äquivalenten, was eine Finanzierung bis ins zweite Quartal 2026 sichert.
Finanzielle Highlights zeigen einen Nettoverlust von 29,3 Millionen US-Dollar im ersten Quartal 2025 (0,46 US-Dollar pro Aktie), verglichen mit 14,7 Millionen im ersten Quartal 2024. Die F&E-Ausgaben stiegen auf 26,6 Millionen, während die Verwaltungs- und Gemeinkosten auf 4,6 Millionen zunahmen. Das Unternehmen arbeitet auf die mögliche Zulassung der ersten krankheitsmodifizierenden Therapie für FA-Patienten hin.
- Strong cash position of $157.5M as of March 31, 2025
- FDA open to considering skin FXN concentration for accelerated approval pathway
- Cash runway extends into Q2 2026
- On track for BLA submission by end of 2025
- Global Phase 3 study expansion to multiple regions (US, Europe, UK, Canada, Australia)
- Net loss increased to $29.3M in Q1 2025 vs $14.7M in Q1 2024
- R&D expenses doubled to $26.6M from $12.9M year-over-year
- G&A expenses increased to $4.6M from $3.8M year-over-year
- Manufacturing costs increased by $7.1M
Insights
Larimar shows solid cash position despite doubled R&D spend, funding key regulatory milestones through Q2 2026.
Larimar's Q1 2025 financial results reveal a strategic acceleration in R&D investment balanced by a strong cash position. With
The quarterly net loss doubled to
This accelerated spending aligns with Larimar's transition to late-stage development. The increased manufacturing investment supports the planned mid-2025 introduction of the commercial lyophilized formulation - a critical step toward market readiness.
G&A expenses grew modestly to
FDA openness to surrogate endpoint for accelerated approval creates clearer regulatory pathway for Larimar's rare disease therapy.
The FDA's feedback represents a significant regulatory milestone for Larimar's nomlabofusp program. The agency's willingness to consider skin FXN concentration as a reasonably likely surrogate endpoint could substantially streamline the approval pathway for this Friedreich's ataxia treatment.
This regulatory approach acknowledges the challenges of rare disease drug development. The FDA specifically recognized the relationship between increased FXN concentrations in skin cells and relevant tissues such as heart and dorsal root ganglion - validating Larimar's biomarker strategy.
Larimar has established a well-defined regulatory roadmap: BLA submission by year-end 2025 seeking accelerated approval, coupled with a global Phase 3 study initiating mid-2025 across multiple regions including the U.S., Europe, and Australia.
The completion of adolescent dosing in their PK run-in study represents important progress toward expanding the potential patient population. The planned September 2025 data update from both the 50 mg open-label extension study and adolescent cohort will provide critical supporting evidence for their regulatory submissions.
- FDA stated as part of a START pilot program meeting that it is open to considering skin FXN concentration as a reasonably likely surrogate endpoint in support of an accelerated approval
- BLA seeking accelerated approval planned to be submitted by year-end 2025; global Phase 3 study planned to initiate in mid-2025
- Completed dosing in adolescent PK run-in study; topline 50 mg dose data from the OLE study and data from adolescent cohort planned for program update in September 2025
- Strong balance sheet of
$157.5 million cash, cash equivalents and marketable securities as of March 31, 2025, with projected cash runway into second quarter of 2026
BALA CYNWYD, Pa., April 30, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its first quarter 2025 operating and financial results.
“Our strong execution across our nomlabofusp clinical development program continues and we are focused on delivering the first potential disease modifying therapy to patients with FA. Importantly, with the robust preclinical and clinical data package we have in hand and the additional clinical data readouts coming later this year, coupled with the positive FDA feedback and recommendations in line with our current approach, we are on track to submit our planned Biologics License Application (BLA) by the end of 2025 to seek accelerated approval,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “We continue to have frequent communication with the FDA via our START pilot program participation including interactions regarding the adequacy of the safety data set required to support BLA submission. In mid-2025 we expect to initiate our global Phase 3 study following global regulatory feedback on the study protocol, and we also expect to transition to the lyophilized formulation of nomlabofusp. We completed dosing of adolescents in our pharmacokinetic (PK) run-in study and expect to report adolescent data from that study, along with data from participants receiving the 50 mg dose of nomlabofusp daily in our long-term open label extension (OLE) study. This program update will be in September 2025. We look forward to these important near-term catalysts as we work to bring nomlabofusp towards potential registration.”
Recent Highlights
- Completed Dosing of Adolescents in Pharmacokinetic (PK) Run-In Study: Today Larimar announced completion of dosing of adolescents (12-17 years of age) in a PK run-in study for pediatric patients with FA. Adolescents received a weight-based dose expected to match PK of adults receiving the 50 mg dose. Following assessment of safety and PK data, participants will be eligible to screen for the OLE study. The data from this cohort are expected to be presented during the nomlabofusp program update in September 2025.
- Potential for Accelerated Approval Pathway Based on Skin FXN Concentrations: In March 2025, Larimar announced that the FDA is open to the use of FXN concentrations as a reasonably likely surrogate endpoint (RLSE) and the acceptability of FXN concentrations as an RLSE to support approval will be a matter of review in a future marketing application. FDA recommended focusing on assessments of skin FXN concentrations and acknowledged that submitted data 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 we submitted were performed at relevant human doses. FDA suggested exploring the relationship between increases in FXN in skin and changes in pharmacodynamic markers such as lipid profiles and/or clinical measures to provide additional support for the use of FXN as a RLSE.
- Planned Upcoming Regulatory Discussions: Larimar continues to interact with FDA under the START pilot program, including seeking feedback on the adequacy of the safety data set required to support BLA submission.
- 50 mg OLE Data Expected in September 2025: The ongoing OLE study continues to enroll, and active study participants are currently receiving the 50 mg dose of nomlabofusp. Larimar plans to provide an update on OLE data on at least 30 to 40 study participants, including 50 mg dose data, in September 2025.
- Planned Transition to Lyophilized Form of Nomlabofusp: Larimar plans to introduce the lyophilized product formulation intended for commercialization into the clinical development program in mid-2025.
- BLA Submission and Initiation of Global Phase 3 Study on Track: Global Phase 3 study is on track to initiate in mid-2025 with potential sites in the U.S., Europe, U.K., Canada, and Australia. Larimar plans to submit the BLA submission seeking accelerated approval by the end of 2025 to seek accelerated approval.
First Quarter 2025 Financial Results
As of March 31, 2025, the Company had cash, cash equivalents and marketable securities totaling
The Company reported a net loss for the first quarter of 2025 of
Research and development expenses for the first quarter of 2025 were
General and administrative expenses were
About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.
Forward-Looking Statements
This press release contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimar’s ability to develop and commercialize nomlabofusp and any other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials, interactions and filings with the FDA, expectations regarding potential for accelerated approval or accelerated access and time to market and overall development plans and other matters regarding Larimar’s business strategies, ability to raise capital, use of capital, results of operations and financial position, and plans and objectives for future operations.
In some cases, you can identify forward-looking statements by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical milestones and continued interactions with the FDA; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later clinical trials, and assessments; that the FDA may not ultimately agree with Larimar’s nomlabofusp development strategy; the potential impact of public health crises on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale nomlabofusp’s manufacturing process; Larimar’s ability to obtain regulatory approvals for nomlabofusp and future product candidates; Larimar’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent Larimar’s management’s views only as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law.
Investor Contact:
Joyce Allaire
LifeSci Advisors
jallaire@lifesciadvisors.com
(212) 915-2569
Company Contact:
Michael Celano
Chief Financial Officer
mcelano@larimartx.com
(484) 414-2715
| Larimar Therapeutics, Inc. | ||||||||
| Condensed Consolidated Balance Sheets | ||||||||
| (unaudited) | ||||||||
| March 31, | December 31, | |||||||
| 2025 | 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 21,126 | $ | 33,218 | ||||
| Marketable securities | 136,400 | 150,236 | ||||||
| Prepaid expenses and other current assets | 7,959 | 11,850 | ||||||
| Total current assets | 165,485 | 195,304 | ||||||
| Long-term marketable securities | — | |||||||
| Property and equipment, net | 855 | 881 | ||||||
| Operating lease right-of-use assets | 2,647 | 2,838 | ||||||
| Restricted cash | 606 | 606 | ||||||
| Other assets | 582 | 596 | ||||||
| Total assets | $ | 170,175 | $ | 200,225 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,274 | $ | 2,424 | ||||
| Accrued expenses | 18,764 | 20,872 | ||||||
| Operating lease liabilities, current | 1,096 | 1,060 | ||||||
| Total current liabilities | 22,134 | 24,356 | ||||||
| Operating lease liabilities | 3,770 | 4,057 | ||||||
| Total liabilities | 25,904 | 28,413 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Preferred stock; | — | — | ||||||
| Common stock, | 64 | 64 | ||||||
| Additional paid-in capital | 442,592 | 440,758 | ||||||
| Accumulated deficit | (298,439 | ) | (269,158 | ) | ||||
| Accumulated other comprehensive gain | 54 | 148 | ||||||
| Total stockholders’ equity | 144,271 | 171,812 | ||||||
| Total liabilities and stockholders’ equity | $ | 170,175 | $ | 200,225 | ||||
| Larimar Therapeutics, Inc. | ||||||||
| Consolidated Statements of Operations | ||||||||
| (In thousands, except share and per share data) | ||||||||
| (unaudited) | ||||||||
| Three Months Ended March 31, | ||||||||
| 2025 | 2024 | |||||||
| Operating expenses: | ||||||||
| Research and development | $ | 26,552 | $ | 12,939 | ||||
| General and administrative | 4,636 | 3,795 | ||||||
| Total operating expenses | 31,188 | 16,734 | ||||||
| Loss from operations | (31,188 | ) | (16,734 | ) | ||||
| Other income, net | 1,907 | 2,080 | ||||||
| Net loss | $ | (29,281 | ) | $ | (14,654 | ) | ||
| Net loss per share, basic and diluted | $ | (0.46 | ) | $ | (0.27 | ) | ||
| Weighted average common shares outstanding, basic and diluted | 63,964,008 | 53,553,707 | ||||||
| Comprehensive loss: | ||||||||
| Net loss | $ | (29,281 | ) | $ | (14,654 | ) | ||
| Other comprehensive (loss): | ||||||||
| Unrealized (loss) on marketable securities | (94 | ) | (106 | ) | ||||
| Total other comprehensive (loss) | (94 | ) | (106 | ) | ||||
| Total comprehensive loss | $ | (29,375 | ) | $ | (14,760 | ) | ||
FAQ
What is the FDA's response to Larimar's (LRMR) skin FXN concentration endpoint for accelerated approval in 2025?
When will Larimar (LRMR) release the 50mg nomlabofusp OLE study data?
What is Larimar's (LRMR) cash position and runway as of Q1 2025?
What are the key milestones for Larimar's (LRMR) Phase 3 study and BLA submission in 2025?
How did Larimar's (LRMR) Q1 2025 R&D expenses compare to Q1 2024?
