Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (January 2026)
54 slides from Larimar Therapeutics's investor presentation, Jan 12, 2026
Slide 1: Larimar Therapeutics Corporate Deck
pro January 2026 Larimar Therapeutics Corporate Deck
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: Friedreich's ataxia (FA): A rare, debilitating and progressive disease
Friedreich’s ataxia (FA): A rare, debilitating and progressive disease 3 * 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 4: 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 5: FA-Treating Clinicians' Perspectives on Importance of FXN Replacement
FA-Treating Clinicians’ Perspectives on Importance of FXN Replacement A recent national survey* of clinicians treating patients with FA (n = 50) Reinforces the ongoing need for additional therapeutic options for the FA patient 98% believe there is a need for a treatment that targets frataxin levels
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: Advancing Nomlabofusp Towards Registration as the First Potential Disease Modifying Therapy
Advancing Nomlabofusp Towards Registration as the First Potential Disease Modifying Therapy for Friedreich’s Ataxia Nomlabofusp is a first-in-class mitochondrial protein replacement therapy designed to directly address systemic frataxin deficiency in patients with FA, a rare neurodegenerative diseas
Slide 8: 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, US 12,180,253, EP 4004022B1 (Exclusive license from Indiana University) US continuation, EP divisional, and foreign applications pending Expiration July 2040 Composition of Mat
Slide 9: Nomlabofusp Long-term Open Label Study
Nomlabofusp Long-term Open Label Study
Slide 10: 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 11: 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 12: 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 13: 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 14: Nomlabofusp Safety Observations with Long-term Treatment
Nomlabofusp Safety Observations* with Long-term Treatment 65 total participants received at least 1 dose of nomlabofusp across all studies with 39 participants in OL study Most common AEs were mild/moderate local ISRs and did not lead to any withdrawals 7 of 39 participants in OL study experienced a
Slide 15: Education and Management Measures for Risk Mitigation
Education and Management Measures for Risk Mitigation Education and Awareness Management Information on anaphylaxis and potential risks is included in the investigator brochure, study protocols, and informed consent forms Investigators’ guide informs how to recognize, manage, and treat anaphylaxis P
Slide 16: OL Study: Most Withdrawals Occurred in the First Days of Treatment
OL Study: Most Withdrawals Occurred in the First 90 Days of Treatment* Reason for Withdraw Total Study Day 1 Study Day 2- 90 Study Day 91-180 Study Day 181-360 Study Day 360+ Anaphylaxis 7 4 3 0 0 0 Generalized Urticaria 3 0 2 1 0 0 Other Adverse Event** 3 0 1 1 0 1 Other*** 2 0 2 0 0 0 Total Withdr
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 Worsening in FACOMS
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: Benefit-Risk Profile Underpins the Potential of Nomlabofusp
Benefit-Risk Profile Underpins the Potential of Nomlabofusp Based on FDA October 2023 Guidance on Benefit-Risk Assessment Framework for Biological Products Clinical data suggests increases in frataxin levels may result in slowing or halting disease progression 2 Anaphylaxis occurred in some patients
Slide 20: Path to Planned BLA Submission in Q2 2026 Seeking Accelerated Approval
Path to Planned BLA Submission in Q2 2026 Seeking Accelerated Approval Expected Elements of BLA Submission Use of Skin FXN Levels as a Surrogate Endpoint Justification for using increases in skin FXN concentrations from participants in the OL study as a reasonably likely surrogate endpoint (RLSE) FD
Slide 21: 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
Slide 22: 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 novel surrogate endpoint Positive long-term data Increased skin FXN levels sim
Slide 23: Appendix
Appendix Larimar Therapeutics
Slide 24: Mitochondrial Localization and Preclinical Data
Mitochondrial Localization and Preclinical Data
Slide 25: 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 26: 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 27: Nomlabofusp Prevents Development of Ataxic Gait in Neurologic 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 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 29: 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 30: 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 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 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 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 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 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 Ph 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: 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 43: 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 44: 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 45: Additional Phase 1 and 2 Data Presented at the International Congress for Ataxia Research, November 2024
Additional Phase 1 and 2 Data Presented at the International Congress for Ataxia Research, November 2024
Slide 46: 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 47: Pooled Data from Completed Phase 1 & 2 Studies Confirms Disease & FXN Relationships
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 the Clinical and Regulatory 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 – Joined 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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