Larimar Therapeutics Provides Nomlabofusp Development Update and Reports Fourth Quarter and Full Year 2024 Financial Results

Rhea-AI Summary

Larimar Therapeutics (NASDAQ: LRMR) has reported significant progress in its nomlabofusp development program for Friedreich's ataxia (FA). The FDA has indicated openness to considering skin frataxin (FXN) concentration as a surrogate endpoint for accelerated approval, acknowledging data supporting the relationship between skin FXN and relevant tissues.

Key developments include:

• BLA submission targeted for year-end 2025
• Global Phase 3 study planned for mid-2025
• Open Label Extension study progressing with 50mg dose
• Pediatric PK run-in study advancing for adolescents

Financial highlights:

• Q4 2024 net loss of $28.8 million ($0.45 per share)
• Full year 2024 net loss of $80.6 million ($1.32 per share)
• Strong cash position of $183.5 million as of December 31, 2024
• Cash runway projected into Q2 2026

Positive

• FDA openness to skin FXN concentration as surrogate endpoint for accelerated approval
• Strong cash position of $183.5M with runway into Q2 2026
• FDA acceptance of lyophilized drug product transition
• Successful protocol feedback from both FDA and EMA for Phase 3 study

Negative

• Net loss increased to $80.6M in 2024 from $36.9M in 2023
• R&D expenses significantly increased to $73.3M from $27.7M year-over-year
• Anaphylaxis identified as adverse drug reaction requiring protocol amendment
• Manufacturing costs increased by $36.1M

Insights

Biotech Regulatory Analyst

Larimar's regulatory strategy for nomlabofusp is gaining significant traction with the FDA. The agency's openness to considering skin frataxin (FXN) concentration as a surrogate endpoint for accelerated approval represents a pivotal development that could meaningfully accelerate the path to market for this Friedreich's ataxia (FA) treatment.

The FDA's acknowledgment that Larimar's data supports a relationship between skin FXN levels and critical tissues (heart, dorsal root ganglia, skeletal muscle) strengthens the company's surrogate endpoint strategy. This validation of the biomarker approach is particularly valuable for rare disease drug development where traditional endpoints may be challenging to achieve in reasonably sized trials.

Two aspects deserve careful attention: First, the FDA qualified that final acceptance of FXN as a surrogate endpoint "would ultimately be a matter of review of the data in a future marketing application," indicating some remaining regulatory uncertainty. Second, the protocol amendment adding premedication due to anaphylaxis risk signals a meaningful safety concern that requires management but doesn't appear to derail development plans.

The company's participation in the START pilot program is facilitating productive regulatory interactions, with both FDA and EMA providing protocol feedback for the global Phase 3 study. The timeline targeting BLA submission by year-end 2025 appears realistic given the current development pace, particularly with the company transitioning to the commercial lyophilized formulation this year.

The integrated regulatory approach - pursuing accelerated approval while simultaneously initiating Phase 3 to confirm clinical benefit - represents a well-structured strategy for bringing this potential first disease-modifying therapy to FA patients.

Financial Analyst

Larimar's $183.5 million cash position provides a substantial runway into Q2 2026, comfortably supporting their planned BLA submission and Phase 3 initiation. This strong financial foundation is critical given the company's accelerating burn rate, with Q4 2024 net loss increasing to $28.8 million from $13.0 million in Q4 2023.

The 152% year-over-year increase in R&D expenses to $26.7 million for Q4 2024 reflects appropriate investment in manufacturing scale-up, including crucial lyophilization development for the commercial formulation. This significant manufacturing investment ($15.0 million increase) demonstrates management's confidence in the program's advancement and preparation for potential commercialization.

G&A expenses rose 31% to $4.6 million, primarily driven by consulting costs related to commercial activities, signaling appropriate pre-launch preparation. The 108% increase in interest income year-over-year reflects efficient capital management in the higher interest rate environment.

For the full year 2024, the $80.6 million net loss ($1.32 per share) versus $36.9 million in 2023 illustrates the increased investment as nomlabofusp advances toward pivotal trials and potential approval. The 164% increase in annual R&D expenses to $73.3 million indicates substantial program advancement.

With current cash supporting operations well beyond the targeted BLA submission, Larimar is financially positioned to execute their clinical and regulatory strategy without immediate capital needs. The concentrated investment in manufacturing and clinical development aligns with the company's stage and regulatory strategy, reflecting disciplined resource allocation toward value-creating milestones.

• FDA stated in written correspondence for 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
  
• FDA recommended measuring skin FXN concentrations to support evidence of effectiveness for accelerated approval pathway and acknowledged submitted data appear sufficient to support relationship between increased skin FXN concentrations and relevant tissues such as heart, dorsal root ganglia and skeletal muscle
  
• Larimar is continuing discussions with FDA on the adequacy of the safety data set to support BLA submission
  
• BLA seeking accelerated approval targeted for submission by year-end 2025
  
• FDA and EMA feedback obtained on global Phase 3 study protocol; study planned to initiate in mid-2025
  
• Topline 50 mg dose data from the OLE study and available data from the adolescent cohort of the PK run-in study planned for program update in September 2025
  
• Strong balance sheet of $183.5 million cash, cash equivalents and marketable securities as of December 31, 2024, with projected cash runway into second quarter of 2026
  

BALA CYNWYD, Pa., March 24, 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 fourth quarter and full year 2024 operating and financial results.

“The strong clinical and regulatory progress across our nomlabofusp program reinforces the timing of our planned Biologics License Application (BLA) submission expected by the end of 2025 to seek accelerated approval. Importantly, we are enthusiastic about the recent FDA interactions and their openness to consider skin frataxin (FXN) concentrations as a potential novel surrogate endpoint reasonably likely to predict clinical benefit in patients with Friedreich’s ataxia (FA). Acceptability of FXN as a novel surrogate endpoint will be based on review of the data by the Food and Drug Administration (FDA) in the future BLA. As we previously disclosed, we have been collecting skin FXN concentration data throughout the development program,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “Overall, FDA’s recommendations are in line with our current approach, and we appreciate the frequent dialogue via our participation in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program as we further refine our registrational plan.”

Dr. Ben-Maimon continued, “Our open label extension (OLE) study continues to advance, with some participants on daily treatment for up to one year. We continue to actively enroll study participants, and all participants are currently receiving the 50 mg dose. The 25 mg initial data we presented in December showed increased and sustained FXN levels over time and early trends in improved clinical outcomes in patients with FA. Our pediatric pharmacokinetic (PK) run-in study continues to progress, and we expect to complete the dosing of the adolescent cohort by the end of this month and report available data in September 2025. We also plan to enroll children 2-11 years of age in the clinical program. We have received feedback from both FDA and European Medicines Agency (EMA) on the protocol for our global Phase 3 trial and are on track to initiate this trial mid-2025. We believe our near-term data package strongly positions us to bring the first potential disease modifying therapy to patients with FA.”

Recent Highlights

• Potential for Accelerated Approval Pathway Based on FXN Concentrations as a Reasonably Likely Surrogate Endpoint (RSLE): FDA stated in written correspondence associated with a meeting through the START pilot program that they are open to considering the use of FXN concentration as a RLSE and the acceptability of FXN’s use as an RLSE would ultimately be a matter of review of the data in a future marketing application.
  
  
• Increases in Skin FXN as Evidence of Effectiveness: 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 recently 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 were performed at relevant human doses.
  
  
• Additional Analyses Under Consideration: FDA also suggested that Larimar consider 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 is continuing discussions with FDA regarding 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 all 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, some of whom have been receiving nomlabofusp for over one year, in September 2025.
  
  
• OLE Protocol Amended to Include Premedication: Larimar’s 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 has amended the OLE protocol to administer premedication for the first month of dosing.
  
  
• Continuing Dosing of Adolescents in Pharmacokinetic (PK) Run-In Study: In January 2025, Larimar initiated dosing of adolescents (12-17 years of age) in a PK run-in study for pediatric patients with FA. In this study, adolescents are receiving a weight-based dose expected to match the 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. Larimar anticipates the dosing of this cohort will be completed by the end of this month. The data from this cohort is expected to be presented during the nomlabofusp program update in September 2025.
  
  
• FDA Acceptance to Transition to Lyophilized Form of Nomlabofusp: 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 our 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.
  
  
• BLA Submission and Initiation of Global Phase 3 Study on Track: Larimar has obtained feedback from both FDA and EMA on the global Phase 3 study protocol and is on track to initiate the study by mid-2025 with potential sites in the U.S., Europe, U.K., Canada, and Australia. Larimar is targeting the BLA submission to seek accelerated approval by the end of 2025.
  

Fourth Quarter and Full Year 2024 Financial Results 

As of December 31, 2024, the Company had cash, cash equivalents and marketable securities totaling $183.5 million.

The Company reported a net loss for the fourth quarter of 2024 of $28.8 million, or $0.45 per share, compared to a net loss of $13.0 million, or $0.30 per share, for the fourth quarter of 2023.

Research and development expenses for the fourth quarter of 2024 were $26.7 million compared to $10.6 million for the fourth quarter of 2023. The increase in research and development expenses was primarily driven by an increase of $15.0 million in nomlabofusp manufacturing costs including lyophilization development, production scaling costs and manufacturing costs related to producing doses to be used in ongoing and planned clinical trials, an increase of $1.3 million in personnel expense due to increased headcount, an increase of $1.0 million of professional fees related to consulting costs, and an increase of $0.5 million in stock compensation costs associated with 2024 grants, partially offset by a decrease of $1.7 million in clinical trial costs primarily associated with decreased activity in the dose exploration study in 2024.

General and administrative expenses for the fourth quarter of 2024 were $4.6 million compared to $3.5 million for the fourth quarter of 2023. The increase in general and administrative expenses was primarily driven by an increase of $0.6 million in professional fees primarily related to consulting costs related to commercial activity and other public company related expenses, and an increase of $0.4 million in personnel expense due to increased headcount.

Other income (expense), net was $2.5 million of income in the three months ended December 31, 2024 compared to $1.2 million of income in the three months ended December 31, 2023. The increase primarily relates to interest income on a higher investment base and higher investment yields on that base during the current period.

For the full year 2024, the Company reported a net loss of $80.6 million, or $1.32 per share, compared to a net loss of $36.9 million, or $0.84 per share, for the same period in 2023.

Research and development expenses for the full year 2024 were $73.3 million compared to $27.7 million for the same period in 2023. The increase in research and development expenses was primarily driven by an increase of $36.1 million in nomlabofusp manufacturing costs including lyophilization development, production scaling costs and manufacturing costs related to producing doses to be used in ongoing and planned clinical trials, an increase of $4.5 million in personnel expense due to increased headcount, an increase of $1.3 million of professional fees related to consulting costs, an increase of $1.2 million in clinical costs primarily associated with the ongoing OLE study which began dosing participants in the first quarter of 2024, an increase of $1.0 million in stock compensation costs associated with 2024 equity compensation grants, an increase of $0.9 million related to the Friedreich’s Ataxia Research Alliance (FARA) Track-FA program, an increase of $0.3 million in internal lab costs and an increase of $0.2 million in facility costs associated with the new lab space.

General and administrative expenses for the full year 2024 were $17.6 million compared to $14.1 million for 2023. The increase in general and administrative expenses was primarily driven by an increase of $1.5 million in professional fees primarily related to consulting costs related to commercial activity and other public company related expenses, an increase of $1.4 million in personnel expense due to increased headcount, an increase of $0.4 million of other expense related to computer software, information technology services and recruiting and an increase of $0.2 million in stock compensation costs associated with 2024 equity compensation grants.

Other income (expense), net was $10.3 million of income in the twelve months ended December 31, 2024 compared to $4.8 million of income in the twelve months ended December 31, 2023. The increase primarily relates to increased interest income on a higher investment base and higher investment yields on that base during 2024.

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; delays in patient recruitment, including as a result of changes in clinical protocols and adverse events; 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.
Consolidated Balance Sheets
		December 31,				December 31,
			2024				2023
Assets
Current assets:
Cash and cash equivalents		$	33,218			$	26,749
Marketable securities			150,236				60,041
Prepaid expenses and other current assets			11,850				3,385
Total current assets			195,304				90,175
Property and equipment, net			881				684
Operating lease right-of-use assets			2,838				3,078
Restricted cash			606				1,339
Other assets			596				659
Total assets		$	200,225			$	95,935
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable		$	2,424			$	1,283
Accrued expenses			20,872				7,386
Operating lease liabilities, current			1,060				837
Total current liabilities			24,356				9,506
Operating lease liabilities			4,057				4,709
Total liabilities			28,413				14,215
Commitments and contingencies (See Note 8)
Stockholders’ equity:
Preferred stock; $0.001 par value per share; 5,000,000 shares authorized as of December 31, 2024 and December 31, 2023; no shares issued and outstanding as of December 31, 2024 and December 31, 2023			—				—
Common stock, $0.001 par value per share; 115,000,000 shares authorized as of December 31, 2024 and December 31, 2023; 63,815,065 and 43,909,069 shares issued and outstanding as of December 31, 2024 and December 31, 2023, respectively			64				43
Additional paid-in capital			440,758				270,150
Accumulated deficit			(269,158	)			(188,554	)
Accumulated other comprehensive gain (loss)			148				81
Total stockholders’ equity			171,812				81,720
Total liabilities and stockholders’ equity		$	200,225			$	95,935

                        

Larimar Therapeutics, Inc.
Consolidated Statements of Operations
(In thousands, except share and per share data)
		Three Months Ended December 31,								Three Months Ended December 31,
		2024				2023				2024				2023
Operating expenses:
Research and development		$	26,738			$	10,648			$	73,278			$	27,670
General and administrative		4,555				3,514				17,612				14,088
Total operating expenses		31,293				14,162				90,890				41,758
Loss from operations		(31,293		)		(14,162		)		(90,890		)		(41,758		)
Other income, net		2,469				1,169				10,286				4,809
Net loss		$	(28,824	)		$	(12,993	)		$	(80,604	)		$	(36,949	)
Net loss per share, basic and diluted		$	(0.45	)		$	(0.30	)		$	(1.32	)		$	(0.84	)
Weighted average common shares outstanding, basic and diluted		63,810,823				43,906,281				61,256,084				43,901,241

FAQ

What is the timeline for Larimar's (LRMR) BLA submission for nomlabofusp?

Larimar plans to submit the Biologics License Application (BLA) for accelerated approval by the end of 2025.

How much cash does Larimar (LRMR) have and what is their runway?

As of December 31, 2024, Larimar has $183.5M in cash, with projected runway into Q2 2026.

When will Larimar (LRMR) start their Phase 3 trial for nomlabofusp?

The global Phase 3 trial is planned to initiate in mid-2025 across multiple countries including the U.S., Europe, U.K., Canada, and Australia.

What are the latest developments in LRMR's Open Label Extension study?

All participants are receiving 50mg doses, with some on treatment for over a year. Data update expected September 2025 for 30-40 participants.

What safety concerns have emerged in Larimar's (LRMR) nomlabofusp trials?

Anaphylaxis has been identified as an adverse drug reaction, leading to protocol amendment requiring premedication during the first month of dosing.