Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (October 2025)
52 slides from Larimar Therapeutics's investor presentation, Oct 14, 2025
Slide 1: Larimar Therapeutics Corporate Deck
pro October 2025 Larimar Therapeutics Corporate Deck
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: Nomlabofusp – First Potential Disease Modifying Therapy for Friedreich's Ataxia
Nomlabofusp – First Potential Disease Modifying Therapy for Friedreich’s Ataxia Initial 50 mg Open Label Study Data Supports Therapeutic Potential of Nomlabofusp Increased skin FXN levels and consistent directional improvement in clinical outcomes 100% of participants with data at 6 months achieved
Slide 4: Friedreich's Ataxia (FA): A rare and progressive disease
Friedreich’s Ataxia (FA): A rare and progressive disease 4 * 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
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: Nomlabofusp Long-term Open Label Study
Nomlabofusp Long-term Open Label Study
Slide 8: Expanding Open Label Study: Now Includes Adolescents and New Participants
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 9: 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 10: 100% of OL Study Participants Achieved FXN Levels 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 11: 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 12: Absolute Skin FXN Levels Increase Over Time with Nomlabofusp
Absolute Skin FXN Levels Increase Over Time with Nomlabofusp* Treatment in the Open Label Study FXN = frataxin; IQR = interquartile range Note: Median skin FXN levels in Larimar’s noninterventional healthy volunteer study= 16.34 pg/µg * Data include all participants with quantifiable FXN levels at e
Slide 13: 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 14: Improvements Across Clinical Outcomes 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 15: 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 16: Key Clinical Data From Prior Studies
Key Clinical Data From Prior Studies
Slide 17: Interest from FDA in Correlation Between Lipids and FXN Concentrations
Interest from FDA in Exploring the Correlation Between Lipids and FXN Concentrations Decreases in elevated lipids in adults with FA* observed after nomlabofusp treatment *Samples from Phase 2 dose exploration study evaluating nomlabofusp 25 mg (Cohort 1) and 50 mg (Cohort 2) or placebo via subcutane
Slide 18: Nomlabofusp PK Profile Consistent Across Studies
Nomlabofusp PK Profile Consistent Across Studies
Slide 19: 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 20: 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* Update on open label study status and regulatory discussions in Q1 2026 BLA submission
Slide 21: 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
Slide 22: Appendix Larimar Therapeutics
Appendix Larimar Therapeutics
Slide 23: Larimar Technology is Supported by a Strong IP Portfolio
2015 2020 2030 2035 2040 2045 2050 2055 2010 2025 Nomlabofusp Composition of Matter and Methods of Treatment US 11,459,363 and US 12,180,253 (Exclusive license from Indiana University) US continuation and foreign applications pending Expiration July 2040 Composition of Matter Larimar Technology is S
Slide 24: Mitochondrial Localization and Preclinical Data
Mitochondrial Localization and Preclinical Data
Slide 25: Nomlabofusp Transduction Leads to hFXN Located in Mitochondria
Nomlabofusp Transduction of Cells In Vitro Leads to hFXN Located in Mitochondria FXN DAPI TOMM20 DAPI FXN TOMM20 DAPI FXN co-localizes with TOMM20 FXN staining TOMM20 (mitochondria) staining Rat cardiomyocytes (H9C2) were transduced with nomlabofusp Cells were fixed and analyzed by immunofluorescenc
Slide 26: Nomlabofusp Extends Survival in FXN-deficient KO Mice
Nomlabofusp Extends Survival in FXN-deficient KO Mice Median survival of MCK-Cre FXN-KO mice 166 days (nomlabofusp) vs. 98 days (Vehicle) Nomlabofusp administered 10 mg/kg SC every other day Survival beyond vehicle mean (107.5 days) 87.5% (nomlabofusp) vs. 33% (Vehicle) Demonstrates that nomlabofusp
Slide 27: Nomlabofusp Prevents Development of Ataxic Gait in KO Mouse Model
Nomlabofusp Prevents Development of Ataxic Gait in Neurologic KO Mouse Model hFXN replacement with nomlabofusp prevents development of ataxic gait Nomlabofusp-treated mice survive longer than untreated mice Human frataxin present in brain, dorsal root ganglia and spinal cord demonstrating central ne
Slide 28: Nomlabofusp Delivers hFXN to Mitochondria and Restores SDH Activity
Nomlabofusp Delivers hFXN to Mitochondria and Restores SDH Activity in KO Mice Mitochondria hFXN concentration increases dose-dependently Given subcutaneously, nomlabofusp functionally replaces hFXN in mitochondria of KO mice MPK = mg/kg MPK = mg/kg Mitochondrial FXN (Heart) SDH Activity (Muscle) Su
Slide 29: Nomlabofusp Prevents Left Ventricle Dilation in KO Mice
Nomlabofusp Prevents Left Ventricle Dilation in KO Mice Left ventricular (LV) volume increases in systole in untreated mice by 8 weeks (after 4 weeks of dosing with vehicle), but remains similar to wildtype when treated with nomlabofusp (10 mg/kg every other day) Diameter (mm) Age in Weeks Age in We
Slide 30: Nomlabofusp Preserves Left Ventricle Function in KO Mice
Nomlabofusp Preserves Left Ventricle Function in KO Mice Percent Change Age in Weeks Left Ventricle Ejection Function Left Ventricle Fractional Shortening Percent Change Age in Weeks KO: CTI-1601 Wild-type: Vehicle KO: Vehicle Study Design – Cardiac and skeletal muscle FXN knockout mice (MCK-CRE) we
Slide 31: Phase 1 Clinical Data
Phase 1 Clinical Data o \.
Slide 32: CTI-1601: Phase 1 Clinical Program in Patients with FA
CTI-1601: Phase 1 Clinical Program in Patients with FA Phase 1 Development Plan Two double-blind, placebo-controlled dosing trials in patients with FA Patient dosing began December 2019 Safety Review Committee assessed all blinded data between each cohort to ensure patient safety Number of subjects:
Slide 33: Completed Phase 1 Multiple Ascending Dose Study Treatment Schedules
Completed Phase 1 Multiple Ascending Dose Study Treatment Schedules for Each Cohort- nomlabofusp (CTI-1601) or placebo 13-day Treatment Period Cohort 2 (50 mg; n = 9) 2 3 4 5 1 6 7 8 9 10 11 12 13 14 = Administration of nomlabofusp or placebo = No Administration 13-day Treatment Period Cohort 1 (25
Slide 34: Dose Dependent Increases in FXN Levels in Skin and Buccal Cells
Dose Dependent Increases in FXN Levels Observed in Skin and Buccal Cells in Phase 1 *FXN levels measured via detection of peptide derived from mature FXN; FXN concentrations are normalized to total cellular protein content in each sample; Data represent median and 25th and 75th percentiles; FXN leve
Slide 35: MAD Trial Patient Demographics
MAD Trial Patient Demographics Parameter Statistic All placebo (n=7) 25 mg CTI-1601 (n=6) 50 mg CTI-1601 (n=7) 100 mg CTI-1601 (n=7) All CTI-1601 (n=20) Overall (n=27) Sex Male n (%) 5 (71.4) 3 ( 50.0) 4 ( 57.1) 3 ( 42.9) 10 ( 50.0) 15 (55.6) Female n (%) 2 (28.6) 3 ( 50.0) 3 ( 42.9) 4 ( 57.1) 10 (
Slide 36: MAD Trial Patient Disease Characteristics
MAD Trial Patient Disease Characteristics Parameter Statistic All placebo (n=7) 25 mg CTI-1601 (n=6) 50 mg CTI-1601 (n=7) 100 mg CTI-1601 (n=7) All CTI-1601 (n=20) Overall (n=27) Age at Symptom Onset Mean 14.1 24.0 19.3 11.9 18.1 17.1 SD 5.34 14.48 6.21 6.72 10.37 9.39 Median 15.0 18.0 19.0 10.0 18.
Slide 37: Summary of MAD Trial PK Analyses
Summary of MAD Trial PK Analyses CTI-1601 was quickly absorbed after subcutaneous administration Dose-proportional increases in exposure observed with increasing doses of CTI-1601 Mean half life of CTI-1601 in plasma was approximately 11 hours CTI-1601 appeared to be at or close to steady state expo
Slide 38: Phase 2 Dose Exploration Data
Phase 2 Dose Exploration Data
Slide 39: Completed Phase 2 Dose Exploration Study (25 & 50 mg Cohorts)
Completed Ph 2 Dose Exploration Study (25 & 50 mg Cohorts) Goal: Further characterize PK/PD and assess safety to inform long-term dose and dose regimen 28-day Treatment Period - nomlabofusp (CTI-1601) or placebo 16 17 18 19 15 20 21 22 23 24 25 26 27 28 2 3 4 5 1 6 7 8 9 10 11 12 13 14 = Subcutaneou
Slide 40: Dose-Dependent Increase in FXN Levels in Skin Cells
Dose-Dependent Increase in FXN Levels in Skin Cells Participants dosed daily for 14 days, then every other day until day 28 FXN Levels* in Skin Cells Change from Baseline** FXN Levels* in Skin Cells Change from Baseline at Day 14 *FXN levels measured via detection of peptide derived from mature FXN;
Slide 41: Skin Cell FXN Levels Achieve Higher % of Healthy Volunteers
Skin Cell FXN Levels Achieve Higher % of Healthy Volunteers* Following 14 days of Daily Nomlabofusp Only participants with quantifiable levels at baseline and day 14 are included in the figures. *% of healthy volunteer FXN level is calculated by dividing each participant's FXN level by the average F
Slide 42: Increase Towards Normal Gene Expression in Adults with FA
Increase Towards Normal Gene Expression in Adults with FA* Observed After Nomlabofusp Treatment Select Baseline Gene Expression Patients with FA* vs. Healthy Volunteers (HV)** Post-treatment Changes in Gene Expression From Baseline Data presented at the International Congress for Ataxia Research, No
Slide 43: Additional Phase 2 Data Presented at the International Congress for Ataxia Research
Additional Phase 1 and 2 Data Presented at the International Congress for Ataxia Research, November 2024
Slide 44: Nomlabofusp Clinical Studies Included a Broad, Representative Population of Adults with FA
Nomlabofusp Clinical Studies Included a Broad, Representative Population of Adults with FA N* Median Mean Min Max Age 61 28.0 31.9 19 69 Age of Onset 61 15.0 15.9 5 60 Age of Diagnosis 61 19.0 21.0 5 64 Shorter GAA (GAA1) 60 550.0 555.8 99 1000 Longer GAA (GAA2) 60 900.0 890.2 265 1300 Frataxin, % o
Slide 45: Pooled Data from Completed Phase 1 & 2 Studies
Pooled Data from Completed Phase 1 & 2 Studies Confirms Disease & FXN Relationships are Consistent with Literature Quartile FXN Concentration* (pg/mcg) Age at Symptom Onset** Age at Diagnosis** GAA1** GAA2** Q1 (N=14) < 1.31 10.5 14.5 616.5 899.5 Q2 (N=14) 1.31 - <1.95 13.5 23.0 486.0 866.0 Q3 (N=14
Slide 46: Modeling/Simulation Predicts 50mg Daily Can Achieve Skin FXN Levels ≥50% of Healthy Controls
Modeling/Simulation Predicts* 50mg Daily Can Achieve Skin FXN Levels ≥50% of Healthy Controls in Most Patients Dashed red line – 50% the average skin FXN/protein ratio (8.17 pg/ug) in a non-interventional study in healthy controls (HC) Blue line – median of simulated values across trials Red lines –
Slide 47: Non-Interventional Study Data
Non-Interventional Study Data
Slide 48: CLIN-1601-002: Top-line Non-interventional Study Results
CLIN-1601-002: Top-line Non-interventional Study Results Non-interventional study measured FXN in homozygous healthy volunteers FXN concentrations were measured in skin and buccal cells from 60 homozygous healthy volunteers utilizing the same sampling technique and assay as clinical trials of nomlab
Slide 49: FDA START Pilot Program
FDA START Pilot Program o \.
Slide 50: START Pilot Program Continues to Expedite Development of Nomlabofusp
START Pilot Program Continues to Expedite the Clinical and Regulatory Development of Nomlabofusp START Pilot Program Support for Clinical Trials Advancing Rare Disease Therapeutics 1 of 7 novel drugs development programs selected by FDA A new milestone-driven program launched by the FDA in September
Slide 51: FARA
FARA
Slide 52: Strong Relationship with FARA – TRACK-FA Neuroimaging Consortium
Strong Relationship with FARA – Joined FARA’s TRACK-FA Neuroimaging Consortium as an Industry Partner National, non-profit organization dedicated to the pursuit of scientific research leading to treatments and a cure for FA FARA provides industry with several key items Assistance with patient recrui
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