Larimar Therapeutics (LRMR) 44th Annual J.P. Morgan Healthcare Conference summary

Disease overview and unmet need

• Friedreich's ataxia is a rare, devastating neurodegenerative disease with early onset and systemic complications, leading to early death, primarily from hypertrophic cardiomyopathy.

• The disease is caused by frataxin deficiency; current approved therapy does not address the root cause.

• Lower frataxin levels correlate with earlier onset and faster progression; increasing levels can delay onset and slow progression.

• There is strong demand for new therapies, with high patient and family interest in clinical trials.

Therapeutic approach and clinical development

• The investigational therapy, nomlabofusp, is designed to restore frataxin by delivering it into mitochondria, mimicking endogenous protein processing.

• Clinical studies show treated patients achieve frataxin levels above the 50% threshold associated with normal function by day 180.

• The open-label study has expanded to include adolescents and will soon enroll children aged 2-11.

• Accelerated approval is being pursued, with a BLA submission planned for Q2 and a launch targeted for early 2027.

Safety and risk management

• Most common adverse events are mild to moderate injection site reactions, which decrease over time and do not cause withdrawal.

• Anaphylaxis occurred in some patients, mainly those with prior exposure; all cases resolved with standard therapy, and risk mitigation includes test dosing, antihistamines, and epinephrine autoinjectors.

• Long-term safety data show continued tolerability, with over 8,000 doses administered and patients on therapy for up to two years.