Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Corporate Update (September 2025)
18 slides from Larimar Therapeutics's corporate update, Sep 29, 2025
Slide 1: Larimar Therapeutics Program Update
pro September 2025 Larimar Therapeutics Program Update Exhibit 99.2
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements that are based on the beliefs and assumptions of Larimar Therapeutics, Inc. ( “Company”) and on information currently available to management. All statements contained in this presentation other than statements of histo
Slide 3: Initial 50 mg OL Study Data Supports Therapeutic Potential of Nomlabofusp
Initial 50 mg OL Study Data Supports Therapeutic Potential of Nomlabofusp Increased skin FXN levels and consistent directional improvement in clinical outcomes Long-term safety observations with daily nomlabofusp 100% of participants with data at 6 months achieved skin FXN levels over 50% of median
Slide 4: Friedreich's Ataxia (FA): A rare and progressive disease
Friedreich’s Ataxia (FA): A rare and progressive disease * E.C. Deutsch et al. Molecular Genetics and Metabolism 101 (2010) 238–245. Genetic defect on both alleles lowers frataxin levels Affects ~20,000 patients globally Progressive disease Unmet Medical Need Larimar is developing nomlabofusp, the f
Slide 5: Nomlabofusp is Designed to Deliver Additional Frataxin
Nomlabofusp is Designed to Deliver Additional Frataxin Nomlabofusp (CTI-1601) maintains the cleavage site between the MTS and mature human frataxin (FXN) The presence of the cleavage site allows the CPP and MTS to be removed by mitochondrial processing peptidase to produce mature human FXN in the mi
Slide 6: FXN Levels Clearly Predict Disease Progression in FA
FXN Levels Clearly Predict Disease Progression in FA Lower FXN levels are associated with earlier onset of disease, faster rate of disease progression, and shorter time to loss of ambulation Adapted from H.L.Plasterer et al. PLoS ONE 2013 8(5):e63958 Age of Onset (Years) Median Time to Loss of Ambul
Slide 7: Expanding Open Label Study: Now Includes Adolescents and Participants not in Prior Studies
Expanding Open Label Study*: Now Includes Adolescents and Participants not in Prior Nomlabofusp Studies Initially, participation in a prior Phase 1 or Phase 2 trial required Expanded study criteria to include: Adolescents (12-17 yrs) from the PK run-in study Participants not in prior studies Plan to
Slide 8: Median Change from Baseline in Skin FXN Levels
Median Change from Baseline in Skin FXN Levels* \ * Median Skin FXN Levels* Increases in Skin FXN Levels are Sustained Over Time 100% of Participants at Day 180 had Skin FXN Levels >50% of Healthy Volunteers Dotted Line indicates 50% of healthy volunteers FXN level *FXN levels measured via detection
Slide 9: 100% of OL Study Participants Achieved FXN Levels by Day 180 Over 50% of Healthy Volunteers
100% of OL Study Participants on Nomlabofusp Achieved FXN Levels by Day 180 that are Over 50% of Healthy Volunteers Percentage of Participants* with Skin FXN Levels > 8.2 pg/µg** (50% of the median FXN concentration found in healthy volunteers) Baseline Day 30 Day 90 Day 180 0% 0/18 33% 6/18 43% 6/1
Slide 10: Skin FXN Levels Achieve Higher % of Healthy Volunteers' FXN Levels
Skin FXN Levels Achieve Higher % of Healthy Volunteers’ FXN Levels* Following Daily Nomlabofusp % of Average HV Baseline < 12.5% 12.5 ≤ 25% 25% ≤ 37.5% > 37.5% *% of average healthy volunteers (HV) FXN level is calculated by dividing each participant's FXN level by the average FXN level (16.34 pg/µg
Slide 11: Absolute Skin FXN Levels Increase Over Time with Nomlabofusp Treatment
Absolute Skin FXN Levels Increase Over Time with Nomlabofusp* Treatment in the Open Label Study Statistic Baseline Day 30 Day 90 Day 180 N 18 18 14 10 Median (IQR) 2.70 (2.14, 4.13) 6.87 (5.34, 10.37) 7.50 (6.66, 13.73) 13.44 (10.10, 26.71) (Min, Max) (1.5, 6.3) (1.5, 76.4) (5.6, 37.1) (8.7, 92.9) F
Slide 12: Disease Characteristics – OL Study & FACOMS Reference Population
Disease Characteristics – OL Study & FACOMS Reference Population Nomlabofusp* FACOMS Age of screening (years) n 38 370 Mean (SD) 30.2 (10.94) 27.5 (9.30) Min, Max 12, 55 12, 54 Age of symptom onset (years) n 38 370 Mean (SD) 12.7 (6.09) 13.8 (5.50) Min, Max 5, 30 5,30 Baseline mFARS Total Score n 38
Slide 13: Improvements Across Clinical Outcomes with Nomlabofusp Relative to Worsening in FACOMS
Improvements Across Clinical Outcomes with Nomlabofusp Relative to Worsening in FACOMS Study Supports Potential Clinical Benefits mFARS [0- 93] FARS-ADL [0- 36] 9-HPT Dominant Hand MFIS [0- 84] Statistic Nomlabofusp FACOMS1 Nomlabofusp FACOMS1 Nomlabofusp FACOMS1 Nomlabofusp FACOMS1 Baseline Median
Slide 14: Nomlabofusp Safety Observations with Long-term Treatment
Nomlabofusp Safety Observations with Long-term Treatment Following the 2 most recent cases of anaphylaxis, Larimar is modifying the starting dose regimen to include: Antihistamines 5 days prior to first dose and for 90 days after first dose 5 mg test dose followed by a 25 mg dose 1 hour later and th
Slide 15: Nomlabofusp PK Profile Consistent Across Studies
Nomlabofusp PK Profile Consistent Across Studies Rapid absorption after subcutaneous administration Steady state reached by Day 30 at both the 25 mg and 50 mg doses with no further accumulation Pharmacokinetic profile consistent with Phase 1 and Phase 2 studies Long-term PK Profile Consistent with P
Slide 16: Global Phase 3 Double-blind Placebo-controlled Study
Global Phase 3 Double-blind Placebo-controlled Study Qualifying sites in U.S., Europe, U.K., Canada, and Australia 18 months of treatment Ambulatory participants 2 - 40 years of age (~2/3 under 21 years of age) n = 100 – 150 Key Study Objectives Safety and tolerability Upright stability (U.S.) and m
Slide 17: Potential Path to Bring Nomlabofusp to Patients Worldwide
Potential Path to Bring Nomlabofusp to Patients Worldwide Received feedback from FDA and EMA on study protocol Sites in the U.S., Europe, U.K., Canada, and Australia currently being qualified Global Phase 3 Study* BLA submission seeking accelerated approval targeted in Q2 2026 U.S. launch targeted f
Slide 18: Nomlabofusp Advancing Towards BLA Submission for FA
Nomlabofusp Advancing Towards BLA Submission for FA First potential disease modifying therapy Designed to systemically address FXN deficiency in FA FDA clarity on key BLA elements BLA based on skin FXN levels as potential surrogate endpoint Positive long-term data Increased skin FXN levels similar t
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