Larimar’s Pediatric Friedreich’s Ataxia Trial Termination: What Investors Should Know

Larimar Therapeutics, Inc. (LRMR) announced an update on their ongoing clinical study.

Study Overview
This early-stage clinical trial, officially titled “A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Nomlabofusp in Adolescents and Children With Friedreich’s Ataxia,” was designed to test Larimar Therapeutics’ lead drug in younger patients with Friedreich’s ataxia, a rare inherited nerve disorder. The main goal was to assess safety and how the body processes the drug, a key step before larger, longer studies, and an important proof point for Larimar’s platform targeting this serious unmet medical need.

Intervention/Treatment
The study tested nomlabofusp (also known as CTI-1601), a drug given as a shot under the skin. It is a lab-made fusion protein meant to replace frataxin, a protein lacking in people with Friedreich’s ataxia. Patients were assigned to receive either nomlabofusp or a placebo injection, allowing researchers to compare safety and early signals of benefit between active treatment and control.

Study Design
This was an interventional Phase 1 trial with patients randomly assigned to different groups. One group received nomlabofusp and the other received placebo, in parallel, for seven days. The study was “quadruple blind,” meaning patients, their care teams, the investigators, and those measuring outcomes did not know who received the drug or placebo. The primary purpose was treatment-focused, but in this early phase the emphasis was on safety rather than clear measures of clinical improvement.

Study Timeline
The study was first submitted in November 2024, signaling the formal start of the regulatory process for this pediatric program. The most recent update to the record was filed on January 26, 2026, showing that key information on the trial has been reviewed and refreshed. Notably, the trial status is listed as “terminated,” indicating that the study was stopped early and will not reach a planned primary or final completion date; reasons for termination are not detailed in the registry entry.

Market Implications
The termination of this Phase 1 pediatric study is likely to weigh on Larimar Therapeutics’ stock in the near term, as it raises questions about the path forward for nomlabofusp in younger patients and possibly the broader program. Investors will focus on whether the stop was driven by safety signals, strategic reprioritization, or operational issues, as each scenario carries very different valuation implications. In the rare disease space, where companies such as Reata (now part of Biogen) and others have pursued Friedreich’s ataxia therapies, pipeline setbacks can quickly shift sentiment and funding toward competitors with cleaner development paths. For Larimar, clarity from management on remaining adult data, alternative dosing, or new indications will be critical to stabilizing expectations and determining whether the market views this as a temporary setback or a more fundamental challenge to the company’s platform. The study record confirms the trial has been terminated and recently updated, with further details available on the ClinicalTrials portal.

To learn more about LRMR’s potential, visit the Larimar Therapeutics, Inc. drug pipeline page.