Larimar Therapeutics (LRMR) The Citizens Life Sciences Conference 2026 summary

Program overview and disease background

• Developing a protein replacement therapy targeting Friedreich's ataxia, a rare, progressive neurodegenerative disease caused by frataxin deficiency.

• The therapy aims to deliver frataxin into mitochondria via subcutaneous injection, potentially slowing or reversing disease progression.

• Friedreich's ataxia affects about 5,000 patients in the US and 20,000 globally, with higher prevalence in Europe.

• Existing approved therapy (SKYCLARYS) addresses oxidative stress but not the root protein deficiency.

Clinical data and efficacy

• Four clinical trials completed, including dose exploration and PK studies; ongoing open-label extension study with patients out over a year.

• Demonstrated increased frataxin levels in patients, with 100% of patients above 50% of normal at six months.

• Clinical outcomes showed a 2.25-point improvement in mFARS, surpassing the 1.5-point improvement seen with the approved drug.

• Improvements also observed in activities of daily living, nine-hole peg test, and fatigue scale.

• Correlation found between frataxin levels and functional improvements; further data cut planned for Q2.

Biomarker and safety insights

• Skin and buccal cells used for frataxin measurement due to accessibility and correlation with disease burden.

• Animal studies confirmed frataxin increases in key tissues, supporting use of surrogate endpoints.

• Dose-dependent normalization of lipid profiles observed, correlating with frataxin levels.

• Most common adverse events are mild to moderate injection site reactions; seven cases of anaphylaxis, mainly after drug hiatus and re-exposure.

• No dropouts due to injection site reactions; anaphylaxis managed by discontinuation.