Larimar Therapeutics (LRMR) investor relations material

Study background and disease overview

• Friedreich's Ataxia (FA) is a rare, progressive disease caused by genetic defects lowering frataxin (FXN) levels, affecting about 20,000 patients globally, with significant unmet medical need.

• Lower FXN levels are linked to earlier onset, faster progression, and shorter time to loss of ambulation in FA.

• Nomlabofusp is designed to deliver additional frataxin to mitochondria, aiming to address the underlying FXN deficiency.

Study design and objectives

• Ongoing long-term open-label study evaluates daily subcutaneous injections of nomlabofusp in FA patients, focusing on safety, pharmacokinetics, and ability to increase tissue frataxin levels compared to a natural history reference population.

• Participants initially received 25 mg, with transition to 50 mg after 30 days; adolescents receive weight-based equivalent dosing.

• Study includes both adults and adolescents, with plans to enroll children aged 2–11 years.

• Global phase 3, double-blind, placebo-controlled study is planned, targeting 100–150 ambulatory patients aged 2–40 years, with sites in the US, Europe, UK, Canada, and Australia.

• Study population includes about 50% non-ambulatory participants at baseline, with over half wheelchair-bound, indicating advanced disease.

Efficacy and biomarker results

• 100% of participants with 6 months of treatment achieved skin FXN levels over 50% of healthy volunteer median, similar to asymptomatic carriers.

• Median skin FXN levels increased from 2.70 pg/μg at baseline to 13.44 pg/μg at 6 months, approaching healthy volunteer levels (16.34 pg/μg).

• Consistent improvements observed in mFARS, FARS-ADL, nine-hole peg test, and MFIS over one year, with a median 2.25-point improvement in mFARS, compared to a 1-point worsening in the reference population.

• Improvements in activities of daily living, fine motor coordination, and fatigue exceeded minimal important differences.

• Data suggest potential for actual clinical improvement, even in late-stage patients.