Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (May 2026)
54 slides from Larimar Therapeutics's investor presentation, May 14, 2026
Slide 1: Larimar Therapeutics Corporate Deck May 2026
pro May 2026 Larimar Therapeutics Corporate Deck Exhibit 99.2
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this presentation other than statements of historical fact are forward-l
Slide 3: Nomlabofusp Granted FDA Breakthrough Therapy Designation for Friedreich's Ataxia
Nomlabofusp Program Granted FDA Breakthrough Therapy Designation for the Treatment of Adults and Children with Friedreich’s Ataxia Designed to expedite the development and regulatory review of a drug for a serious condition Eligibility requires preliminary clinical evidence which indicates that the
Slide 4: Nomlabofusp Awarded Multiple Global Regulatory Designations
Nomlabofusp Awarded Multiple Global Regulatory Designations Intended to Expedite and Incentivize the Development Program US Designations Breakthrough Therapy Designation START Pilot Program Orphan Drug Designation Fast Track Designation Rare Pediatric Disease Designation Priority review voucher prog
Slide 5: FDA Engagement on Planned BLA Submission for Nomlabofusp Program
FDA Engagement on Planned BLA Submission for Nomlabofusp Program Intend to initiate rolling BLA* seeking accelerated approval with submission of nonclinical and clinical modules in June 2026; submission of the final modules, including the CMC module, expected in 2H 2026 Continued alignment with FDA
Slide 6: Multiple Planned Near-term Milestones for Potential Registration
Multiple Planned Near-term Milestones for Potential Registration Open Label Study Global Phase 3 *Rolling BLA Submission U.S. Approval/Launch Topline data Q2 2026 Initiate dosing of first patient Mid-2026 1H 2027 $200.4 M in cash & investments as of March 31, 2026, with projected cash runway into Q2
Slide 7: Friedreich's Ataxia (FA): A rare, debilitating and progressive disease
Friedreich’s Ataxia (FA): A rare, debilitating and progressive disease 7 * E.C. Deutsch et al. Molecular Genetics and Metabolism 101 (2010) 238–245. Larimar is developing nomlabofusp as the first potential disease modifying therapy for FA. Designed to potentially save patients from enormous sufferin
Slide 8: Nomlabofusp is Designed to Target the Root Cause of FA, FXN Deficiency
Nomlabofusp is Designed to Target the Root Cause of FA, FXN Deficiency 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 hum
Slide 9: 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 10: Robust Clinical Development Program to Support Planned BLA Submission
Robust Clinical Development Program to Support Nomlabofusp Planned BLA Submission Seeking Accelerated Approval Consistent, dose-dependent increases in FXN levels in skin and buccal cells across the development program Ongoing OL Study Open label study Assessing the long-term safety, efficacy, PK, PD
Slide 11: Larimar Technology is Supported by a Strong IP Portfolio
2040 Nomlabofusp Composition of Matter and Methods of Treatment US 11,459,363, US 12,180,253, EP 4004022B1 (Exclusive license from Indiana University) US continuation, EP divisional, and foreign applications pending Expiration July 2040 Composition of Matter Larimar Technology is Supported by a Stro
Slide 12: Nomlabofusp Long-term Open Label Study
Nomlabofusp Long-term Open Label Study
Slide 13: Expanded Open Label Study: Now Includes Adolescents and New Participants
Expanded Open Label Study*: Now Includes Adolescents and Participants not in Prior Nomlabofusp Studies Initially, adult 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 Adult and adolescent participants no
Slide 14: Increases in Skin FXN Levels were Sustained Over Time
Change from Baseline in Skin FXN Levels* \ Skin FXN Levels* Increases in Skin FXN Levels* were Sustained Over Time 100% of Participants at Day 180 had Skin FXN Levels >50% of Healthy Volunteers Dotted Line indicates 50% of the average FXN concentrations of healthy volunteers. *FXN levels measured vi
Slide 15: Nomlabofusp Increased FXN to Levels Similar to Asymptomatic Carriers
Nomlabofusp Increased FXN to Levels Similar to Asymptomatic Carriers Over Time in the Open Label Study % of Participants* with Skin FXN Levels > 8.2 pg/µg** (50% of the average FXN concentration levels of healthy volunteers which is similar to levels in asymptomatic carriers) Baseline Day 30 Day 90
Slide 16: Nomlabofusp Safety Summary with Long-term Treatment
Nomlabofusp Safety Summary* with Long-term Treatment ~8,000 doses of nomlabofusp have been administered throughout the clinical development program Most common AEs were mild/moderate local ISRs and did not lead to any discontinuations 65 total participants received at least 1 dose of nomlabofusp acr
Slide 17: 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 18: Improvements Across Clinical Outcomes Relative to FACOMS Reference Group
Improvements Across Clinical Outcomes with Nomlabofusp Relative to Worsening in FACOMS Reference Group Supports Potential Clinical Benefits mFARS [0- 93] FARS-ADL [0- 36] 9-HPT Dominant Hand [Seconds] MFIS [0- 84] Statistic Nomlabofusp FACOMS1 Nomlabofusp FACOMS1 Nomlabofusp FACOMS1 Nomlabofusp FACO
Slide 19: Global Confirmatory Phase 3 Double-blind Placebo-controlled Study
Global Confirmatory Phase 3 Double-blind Placebo-controlled Study Plan to initiate screening in Q2 2026; dosing of first patient expected mid-2026 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 tolerabil
Slide 20: Nomlabofusp Program Advances Towards BLA Submission
Nomlabofusp Program for the Treatment of Adults and Children with FA Advances Towards BLA Submission Seeking Accelerated Approval Safety and Exposure Dataset will be a matter of review at time of BLA submission Anticipated Near-term Registrational Milestones Topline OL data in Q2 2026 FDA Type B mee
Slide 21: Appendix
Appendix Larimar Therapeutics
Slide 22: Mitochondrial Localization and Preclinical Data
Mitochondrial Localization and Preclinical Data
Slide 23: Nomlabofusp Cell Transduction In Vitro Leads to hFXN in Mitochondria
Nomlabofusp Cell Transduction In Vitro Leads to hFXN 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 immunofluorescence microscopy
Slide 24: Nomlabofusp Extends Survival in FXN-deficient KO Mice
Nomlabofusp Extends Survival in FXN-deficient KO Mice Initial proof-of-concept for FXN replacement therapy in cardiac mouse model of FA 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 mea
Slide 25: 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 26: Nomlabofusp Delivers hFXN to Mitochondria and Restores SDH Activity
Nomlabofusp Delivers hFXN to Mitochondria and Restores SDH Activity in KO Mice Study Design – Cardiac and skeletal muscle FXN knockout mice (MCK-CRE) were treated at varying SQ doses of nomlabofusp every other day for two weeks at Jackson Laboratories (Bar Harbor, ME). After dosing, animals were sac
Slide 27: Nomlabofusp Prevents Left Ventricle Dilation in KO Mice
Nomlabofusp Prevents Left Ventricle Dilation in KO Mice Study Design – Cardiac and skeletal muscle FXN knockout mice (MCK-CRE) were treated at 10 mg/kg every other day at Jackson Laboratories (Bar Harbor, ME). Echocardiograms were performed pre-dose and post dose. Left ventricular (LV) volume increa
Slide 28: Nomlabofusp Preserves Left Ventricle Function in KO Mice
Nomlabofusp Preserves Left Ventricle Function in KO Mice Study Design – Cardiac and skeletal muscle FXN knockout mice (MCK-CRE) were treated at 10 mg/kg every other day at Jackson Laboratories (Bar Harbor, ME). Echocardiograms were performed pre-dose and post dose. Percent Change Age in Weeks Left V
Slide 29: Phase 1 Clinical Data
Phase 1 Clinical Data o \.
Slide 30: CTI-1601: Phase 1 Clinical Program in Patients with FA
CTI-1601: Phase 1 Clinical Program in Patients with FA Program consisted of double-blind, placebo controlled single- and multiple-ascending dose trials Phase 1 Development Plan Two double-blind, placebo-controlled dosing trials in patients with FA Patient dosing began December 2019 Safety Review Com
Slide 31: 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 32: Dose Dependent Increases in FXN Levels in Skin and Buccal Cells in Phase 1
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 33: 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 34: 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 35: 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 36: Phase 2 Dose Exploration Data
Phase 2 Dose Exploration Data
Slide 37: 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 38: 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 39: 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 40: 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 41: Elevated TGs in FA Decreased with Nomlabofusp and Correlated with FXN Increases
Elevated TGs in FA Decreased with Nomlabofusp and Correlated with FXN Increases In patients with FA from Phase 2 dose exploration study after treatment with nomlabofusp r : -0.8735, P <0.0001 r : -0.5765, P= 0.0194 TGs, Avg % (Change from Baseline at D28) FXN, pg/ug (Change from Baseline at D14) 25
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 Open Label Data
Additional Open Label Data o \.
Slide 44: Skin FXN Levels Achieved Higher % of Healthy Volunteers' FXN Levels
Skin FXN Levels Achieved 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 FXN level in healthy volunteers (HV); FXN level is calculated by dividing each participant's FXN level by the average FXN lev
Slide 45: 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 46: 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 47: Pooled Data Confirms Disease & FXN Relationships Consistent with Literature
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 48: 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 49: Non-Interventional Study Data
Non-Interventional Study Data
Slide 50: 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 51: FDA START Pilot Program
FDA START Pilot Program o \.
Slide 52: 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 53: FARA
FARA
Slide 54: Strong Relationship with FARA – TRACK-FA Neuroimaging Consortium Industry Partner
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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