Larimar Therapeutics Expects to Launch Pharmacokinetic Study for Pediatric Friedreich Ataxia by Year-End

Carole Ben-Maimon, MD

(Credit: Larimar)

In a recent company update, Larimar Therapeutics announced that it is planning to initiate a pharmacokinetic (PK) run-in study in adolescents 12 to 17 years of age and children 2 to 11 years of age with Friedreich ataxia (FA) by year-end, initially aiming to enroll 12 to 15 adolescent patients who will be randomized 2:1 to receive either the company's investigational therapy nomlabofusp or placebo daily.¹ The company noted that participants can transition to the open-label extension study (NCT06447025) after completing the PK run-in study, which continues to progress with interim data to be reported in the fourth quarter of the year.

In May 2024, the FDA selected the company’s nomlabofusp development program as one of few programs able to take part in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.² The selection was based on development program readiness, which included the potential of nomlabofusp to address serious and unmet clinical needs in a rare neurodegenerative condition. Additionally, the alignment of chemistry, manufacturing, and controls development timelines with clinical development plans, and a proposed communications plan to accelerate pivotal study initiation and path to potential biologics license applications (BLA) submission was considered for the selection.

“We made significant achievements in our nomlabofusp program this quarter that strongly position us for successful execution across important catalysts over the next 12 months. We were honored to be selected by the FDA to participate in the START pilot program which may be invaluable in helping us achieve our timeline for BLA submission targeted for the second half of 2025 to support accelerated approval,” Carole Ben-Maimon, MD, president, and chief executive officer at Larimar, said in a statement.¹ “We are actively pursuing clinical sites in the U.S., Europe, U.K. Canada, and Australia in anticipation of initiating a global confirmatory study in mid-2025.”

In June 2024, the company entered an agreement with the Friedreich’s Ataxia Research Alliance to join the TRACK-FA Neuroimaging Consortium which includes pharmaceutical, biotechnology, academic and clinical partners.¹ Larimar noted in its company update that the consortium will perform a natural history study aimed to establish disease-specific neuroimaging biomarkers to track disease progression in the brain and spinal cord as well as offer a basis for using these biomarkers in studies. Utilizing longitudinal data from large cohorts of patients compared with controls, the study will investigate the changes in areas formally shown to be compromised among patients with FA. As a partner in industry, Larimar announced that it will assist with funding the study and help with the study design, research activities, and analysis.

“We are excited to have recently joined the TRACK-FA Neuroimaging Consortium as an industry partner to support research to define disease-specific neuroimaging biomarkers for potential use in clinical trials,” Ben-Maimon addressed in a statement.¹ “Our OLE study continues to progress with all 7 sites now activated and interim data planned for the fourth quarter of this year. We plan to initiate a PK run-in study in adolescents with FA by year-end, with option for study participants to transition to the OLE study after completing the run-in study. Expanding our clinical program into younger patients will allow us to evaluate the effect of nomlabofusp earlier in the disease process which may help further address the effect of the underlying frataxin deficiency in patients with FA.”

REFERENCES
1. Larimar Therapeutics Reports Second Quarter 2024 Operating and Financial Results. News Release. August 7, 2024. Accessed August 15, 2024. https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-reports-second-quarter-2024-operating-and
2. Larimar Therapeutics Selected by FDA to Participate in START Pilot Program for Nomlabofusp in Friedreich’s Ataxia. News Release. May 30, 2024. Accessed August 15, 2024. https://investors.larimartx.com/news-releases/news-release-details/larimar-therapeutics-selected-fda-participate-start-pilot