Larimar Therapeutics (LRMR) Company Report

Larimar Therapeutics is developing nomlabofusp for Friedreich's ataxia (FA), a devastating neurodegenerative disease, aiming to be the first disease-modifying therapy by restoring frataxin levels.
The company plans to submit a Biologics License Application (BLA) in Q2 2026 for accelerated approval, using frataxin levels as a novel surrogate endpoint, with a targeted launch in early 2027.
Clinical data from the ongoing open-label study shows treated patients achieved frataxin levels above the 50% mark by day 180 and demonstrated an improvement of 2.25 points in mFARS, which is considered clinically meaningful.
While generally well-tolerated, seven patients experienced anaphylaxis, primarily early in treatment, which was treatable with standard therapy; mitigation strategies are in place.
A confirmatory Phase 3 trial for ambulatory patients (2-40 years old) is being initiated, with regulatory authorities having reviewed and approved the protocol.

7 days ago

Transcript

Larimar Therapeutics Provides Update on Friedreich's Ataxia Program

LRMR

Product Launch

New Projects/Investments

Larimar Therapeutics is developing nomlabofusp as a potential first disease-modifying therapy for Friedreich's ataxia (FA), a rare neurodegenerative disease caused by frataxin deficiency.
The company plans to submit a Biologics License Application (BLA) for accelerated approval in Q2 2026, targeting a launch in early 2027. The FDA has agreed to use frataxin levels as a novel surrogate endpoint for review.
Clinical data indicates that nomlabofusp led to an improvement of 2.25 points in mFARS, an outcome measure for FA, while a matched reference population worsened by one point. This improvement is considered clinically meaningful.
The safety profile includes common injection site reactions and seven cases of anaphylaxis among 65 exposed patients, all of which were treatable with EpiPens and resolved without long-term issues.
A confirmatory Phase III trial for 100-150 ambulatory patients is being initiated, with the first subject expected to be dosed early in 2026.

7 days ago

Transcript

Larimar Therapeutics Details Nomlabofusp Development and Regulatory Strategy

LRMR

Product Launch

New Projects/Investments

Guidance Update

Larimar Therapeutics is developing nomlabofusp for Friedreich's ataxia (FA), a rare neurodegenerative disease affecting approximately 5,000 patients in the US and 20,000 worldwide.
The company is pursuing accelerated approval for nomlabofusp, with a BLA filing in Q2 2026 based on frataxin levels as a novel surrogate endpoint, targeting a launch in early 2027. A regulatory update is also planned for Q1 2026.
Clinical data from an open-label study showed that treated patients achieved skin frataxin levels above the 50% mark by day 180 and demonstrated a 2.25-point improvement in mFARS score, considered clinically meaningful, compared to worsening in a natural history study.
While the most common adverse events are injection site reactions, seven patients experienced anaphylaxis which was treatable with EpiPens, and mitigation strategies are in place.
A Phase III confirmatory study is being initiated, enrolling 100-150 ambulatory patients for 18 months, with first dosing expected early in 2026.

7 days ago

Transcript

Larimar Therapeutics Provides Update on Nomlabofusp Regulatory Pathway and Financials

LRMR

New Projects/Investments

Guidance Update

Product Launch

Larimar Therapeutics is advancing Nomlabofusp (CTI-1601) for Friedreich’s Ataxia (FA), targeting a Biologics License Application (BLA) submission for accelerated approval in Q2 2026 and a U.S. launch in early 2027.
The BLA submission will utilize skin frataxin (FXN) levels as a novel surrogate endpoint, supported by data from multiple studies showing dose-dependent increases in tissue FXN and consistent directional improvements across key clinical outcomes.
Nomlabofusp holds several regulatory designations, including Orphan Drug and Fast Track, and was selected for the FDA's START pilot program, underscoring regulatory support for its development.
While generally well-tolerated, anaphylaxis occurred in 7 of 39 participants in the open-label study, which was successfully managed with epinephrine with no further complications.
The company reported an estimated $136.9 million in cash and investments as of December 31, 2025, providing a projected financial runway into Q4 2026.

Jan 12, 2026, 9:18 PM

8-K

Larimar Therapeutics, Inc. Completes Exchange of Common Stock for Series A Convertible Preferred Stock

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Convertible Preferred Issuance

On December 16, 2025, Larimar Therapeutics, Inc. entered into an exchange agreement with Blue Owl Healthcare Opportunities IV Public Investments LP, where the stockholder exchanged 2,500,000 shares of common stock for 250,000 shares of newly designated Series A convertible preferred stock.
Each share of the Series A Preferred Stock is convertible into 10 shares of Common Stock, subject to a beneficial ownership conversion limitation initially set at 9.99% of the total outstanding Common Stock, which can be increased to 19.99%.
The Series A Preferred Stock generally has no voting rights, except as legally required or for amendments to its terms, which require majority holder consent.
In a liquidation event, Preferred Stock holders will participate pari passu with Common Stock holders on an as-converted basis.

Dec 17, 2025, 1:00 PM

8-K

Larimar Therapeutics Reports Q3 2025 Financial Results and Operational Highlights

LRMR

Earnings

Guidance Update

Product Launch

Larimar Therapeutics reported a net loss of $47.7 million, or $0.61 per share, for the third quarter ended September 30, 2025. Research and development expenses for the quarter increased to $44.9 million, primarily driven by a $25.8 million increase in nomlabofusp manufacturing costs.
As of September 30, 2025, the company held $175.4 million in cash, cash equivalents, and marketable securities, with a projected cash runway into the fourth quarter of 2026.
The company is targeting a Biologics License Application (BLA) submission for nomlabofusp in the second quarter of 2026, seeking accelerated approval, with a U.S. launch targeted for early 2027.
A modified starting dose regimen for nomlabofusp, designed to mitigate the risk of anaphylaxis events, has been agreed to by the FDA and is being incorporated into the Phase 3 protocol.

Nov 5, 2025, 12:03 PM

8-K

Larimar Therapeutics Updates on Nomlabofusp Clinical Program and Financial Outlook

LRMR

Guidance Update

New Projects/Investments

Product Launch

Larimar Therapeutics reported positive open-label study data for nomlabofusp in Friedreich’s Ataxia, showing 100% of participants achieved skin FXN levels over 50% of healthy volunteers at 6 months and a median mFARS score improvement of 2.25 after one year, indicating therapeutic potential.
The company plans to submit a Biologics License Application (BLA) for accelerated approval in Q2 2026, with a targeted U.S. launch for early 2027. The FDA has agreed with Larimar's modified dosing regimen following 7 cases of anaphylaxis in the open-label study.
As of September 30, 2025, Larimar estimates $175.7 million in cash and investments, projecting a cash runway into Q4 2026.

Oct 14, 2025, 11:00 AM

8-K

Larimar Therapeutics Provides Nomlabofusp Clinical Data and Regulatory Update

LRMR

Guidance Update

New Projects/Investments

Larimar Therapeutics announced new data from its long-term open-label study of nomlabofusp for Friedreich's ataxia, demonstrating consistent directional improvements in key clinical outcome measures including mFARS, FARS ADL, Nine-Hole Peg Test, and MFIS after one year of dosing in eight participants.
Participants treated with nomlabofusp for one year showed a median 2.25-point improvement in mFARS, contrasting with a median one-point worsening in the FACOMS reference population.
All participants achieved frataxin levels exceeding 50% of healthy volunteers by six months, reaching levels similar to asymptomatic carriers.
While seven participants experienced anaphylaxis in the open-label study, Larimar has modified its dosing regimen with FDA agreement, and the company remains on track for a Biologics License Application (BLA) submission in the second quarter of 2026 seeking accelerated approval.
Larimar reported a pro forma cash balance of $203.6 million as of June 30, 2025, with a projected cash runway into Q4 2026.

Sep 29, 2025, 12:00 PM

Transcript

Larimar Therapeutics Reports Positive Nomlabofusp Data and BLA Submission Update

LRMR

New Projects/Investments

Guidance Update

Larimar Therapeutics reported positive 25-milligram and 50-milligram data from its ongoing long-term open-label study of nomlabofusp for Friedreich's ataxia.
100% of participants achieved frataxin levels greater than 50% of healthy volunteers by six months.
Participants treated with nomlabofusp for one year showed a median 2.25-point improvement in MFARS, while the FACOMS reference population worsened by one point.
Safety data indicated anaphylaxis in seven participants in the open-label study, mostly on the initial day of administration, with all returning to usual health after standard treatment.
The company continues to target BLA submission for nomlabofusp in the second quarter of 2026, seeking accelerated approval.

Sep 29, 2025, 12:00 PM

Transcript

Larimar Therapeutics Announces Encouraging Nonlobofus Open-Label Study Results and 2026 BLA Target

LRMR

New Projects/Investments

Guidance Update

Larimar Therapeutics reported positive long-term open-label study data for nonlobofus in Friedreich's ataxia (FA), demonstrating consistent directional improvements in key clinical outcome measures, including a median 2.25 improvement in mFARS after one year of dosing compared to a median 1.0 point worsening in a reference population.
The study also showed that 100% of participants achieved skin frataxin levels greater than 50% of healthy volunteers by six months.
While anaphylaxis was reported in 7 participants, mostly on the initial day of administration, all recovered after standard treatment, and Larimar's modified dosing regimen was agreed upon by the FDA.
Larimar Therapeutics is targeting a BLA submission in 2026 for nonlobofus, seeking accelerated approval based on skin frataxin levels.
The company has $203.6 million in pro forma cash as of June 30, 2025, with a projected cash runway into Q4 2026.