BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) announced third-quarter 2025 financial results and a clinical update highlighting continued progress in its nomlabofusp development program for Friedreich’s ataxia (FA), a rare genetic disorder with no approved disease-modifying therapies.
In its open-label study, Larimar reported that after six months of daily nomlabofusp administration, all participants (n=10) achieved skin frataxin (FXN) levels similar to those seen in asymptomatic carriers. After one year, participants showed consistent directional improvement across multiple clinical measures—including modified Friedreich Ataxia Rating Scale (mFARS), FARS-Activities of Daily Living (ADL), 9-Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS)—compared with a worsening seen in a natural history reference population from the FACOMS study.
“We were pleased to recently share exciting long-term data from our open-label study showing consistent directional improvement across four key clinical outcome measures relative to a Friedrich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population, and increased skin frataxin (FXN) levels similar to asymptomatic carriers,” said Carole Ben-Maimon, MD, president and CEO of Larimar. “These findings underscore the potential of daily nomlabofusp treatment to help change the disease course of patients living with Friedreich’s ataxia, including those with advanced disease.”
The company reported that nomlabofusp was generally well tolerated in long-term dosing, with eight participants receiving treatment for over a year. Seven participants experienced anaphylaxis during the first six weeks of dosing; all recovered following standard treatment. The company noted that such events are more common upon re-exposure to a therapy after a dosing gap. To mitigate future risk, Larimar is introducing a modified starting dose regimen, which will also be incorporated into its Phase 3 study design.
“With this positive data in hand, and a targeted path to registration, we continue to target our Biologics License Application (BLA) submission in the second quarter of 2026 seeking accelerated approval,” Ben-Maimon added. “We are focused on execution of our near-term milestones that will advance nomlabofusp as the first potential disease-modifying therapy designed to address the root cause of FA.”
The company continues to prepare for its Phase 3 confirmatory study and has begun qualifying global sites for enrollment. Larimar plans to provide an update on its regulatory discussions and open-label study status in the first quarter of 2026, with a potential U.S. launch targeted for early 2027 pending regulatory approval.
Financially, Larimar reported $175.4 million in cash, cash equivalents, and marketable securities as of September 30, 2025—enough to fund operations into the fourth quarter of 2026. The company posted a net loss of $47.7 million, or $0.61 per share, for the third quarter, compared to a net loss of $15.5 million, or $0.24 per share, in the same quarter last year.
Research and development expenses rose to $44.9 million from $13.9 million a year ago, primarily due to increased manufacturing and clinical costs for nomlabofusp, while general and administrative expenses rose modestly to $4.6 million.
Larimar’s data reinforces growing optimism for nomlabofusp as a potential first-in-class treatment capable of addressing the genetic cause of Friedreich’s ataxia. With a pivotal trial on the horizon and regulatory engagement underway, the company remains positioned to advance one of the most promising therapeutic candidates in rare neurogenetic disease.
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