Larimar Therapeutics (LRMR) Corporate presentation summary

Disease background and unmet need

• Friedreich's ataxia (FA) is a rare, progressive neurodegenerative disease affecting about 5,000 patients in the U.S. and 20,000 globally, with most cases presenting before age 14.

• FA is caused by a genetic defect that lowers frataxin (FXN) levels, leading to severe symptoms and early mortality, typically from heart disease.

• Current approved treatments do not address frataxin deficiency, leaving a high unmet medical need.

• Clinician surveys show 98% believe a treatment targeting frataxin is needed, and 98% agree addressing the root cause is the next step in FA treatment.

Nomlabofusp mechanism and preclinical data

• Nomlabofusp is a first-in-class mitochondrial protein replacement therapy designed to deliver mature human frataxin to mitochondria.

• Preclinical studies show nomlabofusp extends survival, prevents ataxic gait, and preserves cardiac function in FXN-deficient mouse models.

• In vitro, nomlabofusp transduces cells and localizes frataxin to mitochondria.

Clinical development and efficacy

• Four completed studies (Phase 1 SAD/MAD, Phase 2 dose-exploration, adolescent PK) and an ongoing open-label (OL) study support dose-dependent increases in tissue FXN.

• In the OL study, 100% of participants at Day 180 achieved skin FXN levels >50% of healthy volunteers, similar to asymptomatic carriers.

• Consistent improvements in clinical outcomes (mFARS, FARS-ADL, 9-HPT, MFIS) were observed after 1 year of treatment compared to worsening in a natural history reference group.

• Dose-dependent increases in FXN were confirmed in Phase 1 and 2 studies, with 50 mg daily predicted to achieve ≥50% of healthy control FXN levels in most patients.