Larimar publishes data supporting skin FXN as surrogate endpoint for FA

BALA CYNWYD, Pa. - Larimar Therapeutics, Inc. (NASDAQ:LRMR), a $205 million market cap biotech company showing strong momentum with an 11% gain last week, announced Tuesday the publication of two peer-reviewed articles highlighting nonclinical data on nomlabofusp, its frataxin (FXN) protein replacement therapy for Friedreich’s ataxia (FA). According to InvestingPro data, the company maintains a healthy balance sheet with more cash than debt.

The published research provides evidence of nomlabofusp’s mechanism of action and its ability to increase FXN levels in disease-relevant tissues including dorsal root ganglia, heart, and skeletal muscle.

The nonclinical findings were included in materials reviewed by the U.S. Food and Drug Administration (FDA) in support of potentially using skin FXN concentrations as a reasonably likely surrogate endpoint for Larimar’s registrational program seeking accelerated approval.

The company plans to submit a Biologics License Application (BLA) for nomlabofusp in the second quarter of 2026. If approved, nomlabofusp would be the first potential disease-modifying therapy for FA patients.

"These encouraging data contributed to FDA’s openness to consider the use of skin FXN concentrations as a reasonably likely surrogate endpoint in support of an accelerated approval," said Carole Ben-Maimon, President and Chief Executive Officer of Larimar, in a press release statement.

The two articles now available online are titled "Pharmacokinetics and Pharmacodynamics of Nomlabofusp in Non-clinical Studies of Friedreich’s Ataxia" and "Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria."

Friedreich’s ataxia is a rare genetic disease characterized by FXN deficiency that leads to progressive nervous system damage and movement problems. With its next earnings report due on August 7, investors seeking deeper insights into Larimar’s development pipeline and financial metrics can access comprehensive analysis through InvestingPro’s detailed research reports, which cover over 1,400 US stocks with expert analysis and actionable intelligence.

In other recent news, Larimar Therapeutics has announced an adjusted timeline for its Biologics License Application (BLA) submission for the Friedreich’s Ataxia treatment, nomlabofusp, now set for the second quarter of 2026. This change follows recommendations from the FDA regarding safety database requirements and the use of skin frataxin concentrations as a surrogate endpoint. Leerink Partners has reiterated an Outperform rating with a $25 price target, highlighting the potential for accelerated approval. Larimar reported $158 million in cash and marketable securities, which is expected to sustain operations until the new submission date. Additionally, Citizens JMP maintains a Market Outperform rating with a $21 target, emphasizing key milestones for 2025, including data from ongoing trials and the initiation of a pivotal confirmatory study. The FDA has shown openness to using skin frataxin concentrations as a surrogate endpoint, which could facilitate accelerated approval. Larimar is preparing for a global Phase 3 study, with site identification underway across multiple countries. These developments mark significant steps in Larimar’s efforts to advance treatment options for Friedreich’s Ataxia.

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