Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (August 2026)
40 slides from Larimar Therapeutics's investor presentation, Aug 4, 2026
Slide 1: Larimar Therapeutics Corporate Deck
pro August 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: Advancing Nomlabofusp Towards Registration as the First Potential Disease Modifying Therapy for FA
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 4: Friedreich's Ataxia (FA): A rare, debilitating and progressive disease
Friedreich’s Ataxia (FA): A rare, debilitating and progressive disease 4 * 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. Intended to help patients avoid profound suffering and maint
Slide 5: 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 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: Alignment with FDA for Rolling Submission of BLA Package for Accelerated Approval
Alignment with FDA for Rolling Submission of BLA Package for Accelerated Approval After a Multi-disciplinary Type B Pre-BLA Meeting FXN as Novel Surrogate Endpoint FDA reaffirmed willingness to consider FXN as a novel surrogate endpoint and that Larimar's exposure-response analysis linking nomlabofu
Slide 8: Comprehensive and Long-Term Data Package Across All Studies
Comprehensive and Long-Term Data Package* Across All Studies Open Label Study 76 Participants >10,000 Doses administered to date in the OL study 66 Received at least one dose of nomlabofusp 43 Participants dosed 22 Active Maximum duration >800 days Sustained increases in tissue FXN levels Continued
Slide 9: Additional Patients with Longer Exposure in OL Study Reinforces Disease Modifying Potential
Additional Patients with Longer Exposure in OL Study* Reinforces the Disease Modifying Potential of Nomlabofusp 13 participants completed 1 year of dosing, 7 of the 13 completed 18 months, and 3 of the 13 completed 2 years Well-Characterized Safety Profile Increased Tissue FXN Levels Observed Improv
Slide 10: Nomlabofusp Long-term Open Label Study (Ongoing)
Nomlabofusp Long-term Open Label Study (Ongoing)
Slide 11: Expanded Open Label Study: Now Includes Adolescents and Participants not in Prior Studies
Expanded Open Label Study*: Now Includes Adolescents and Participants not in Prior Nomlabofusp Studies Initially, adults who had participated 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 partici
Slide 12: Participant Characteristics Represent a Broad Range of Disease Severity
Participant Characteristics Represent a Broad Range of Disease Severity *As of June 2026, there were 43 participants including 2 dosed after the March 2026 cutoff. OL Study Nomlabofusp* N = 41 Age of Screening (Years) Mean (SD) 29.0 (11.30) Min, Max 12, 55 Age Group ≥12 to <18 8 (19.5) ≥18 33 (80.5)
Slide 13: Change from Baseline in Skin FXN Levels
Change from Baseline in Skin FXN Levels* Skin FXN Levels* Increases in Skin FXN Levels* Reached Steady State by 3 Months and were Sustained Over Time *FXN levels measured via detection of peptide derived from mature FXN; FXN concentrations are normalized to total cellular protein content in each sam
Slide 14: Nomlabofusp Treatment Increased and Sustained FXN Levels to Range Expected in Asymptomatic Carriers
Nomlabofusp Treatment Increased and Sustained FXN Levels in Open Label Study to Range Expected in Asymptomatic Carriers % of Participants with Skin FXN Levels in the Range of Asymptomatic Heterozygous Carriers (> 8.2 pg/µg; ~50% of Mean Healthy Volunteer FXN Concentration) Baseline 1 month 3 months
Slide 15: Safety Profile of Nomlabofusp Administration is Well-Characterized
Safety Profile of Nomlabofusp Administration* is Well-Characterized Nomlabofusp is generally well-tolerated long-term; >10,000 doses have been administered in the OL study Most common adverse events were local injection site reactions that were mild to moderate, decreased in frequency over time, and
Slide 16: Baseline Characteristics Used to Generate FACOMS Reference Group
Baseline Characteristics Used to Generate FACOMS Reference Group Methodology for generating reference group was reviewed by FDA and recommendations were incorporated FACOMS longitudinal natural history study (N = 955) includes participants with confirmed FA diagnosis Larimar identified participants
Slide 17: Improvements Across Clinical Outcomes Relative to Worsening in FACOMS Reference Group
Improvements Across Clinical Outcomes with Nomlabofusp Relative to Worsening in FACOMS Reference Group Supports Potential Clinical Benefits Range = min, max. Data are presented as of the March 2026 cutoff date. 1 Based on the range of baseline characteristics of participants in the OL study, Larimar
Slide 18: 2.6-point Difference in Mean mFARS Score at 1 year
2.6-point Difference in Mean mFARS Score for Nomlabofusp Treated Participants and FACOMS Reference Group at 1 year Change in mFARS Change from baseline after treatment with nomlabofusp in OL study: Mean 1.0-point improvement at 1 year in 13 participants Mean 2.3-point improvement at 18 months in 7 p
Slide 19: Patients Who Worsened Between Prior Study and OL Study Improved on Key Outcome Measures
Patients Who Worsened Between Prior Nomlabofusp Study and OL Study* Improved On Key Clinical Outcome Measures During Treatment Average Gap = 2.5 years Average Gap = 2.5 years Average Gap = 2.5 years Average Gap = 2.5 years FXN Levels mFARS FARS-ADL 9-HPT OL study: N at 1 year = 13; N at 18 months =
Slide 20: Different Clinical Outcome Measures May Be More Or Less Sensitive At Different Points In Disease Course
Different Clinical Outcome Measures May Be More Or Less Sensitive At Different Points In A Patient’s Disease Course Average Gap = 2.5 years Average Gap = 2.5 years Average Gap = 2.5 years Average Gap = 2.5 years FXN Levels mFARS FARS-ADL 9-HPT < 12.5 Years Duration (N = 4) ≥ 12.5 – <25 Years Duratio
Slide 21: Majority Achieved Improvement at Year With Certain Outcome Measures More Sensitive at Different Times
Majority Achieved Improvement at 1 Year With Certain Outcome Measures More Sensitive at Different Times in Disease Course mFARS Improved Worsened MFIS 9-HPT 61.5% 38.5% 100% 50% 50% 40% 60% 100% 100% 61.5% 61.5% 38.5% 60% 40% 50% 50% 80% 20% 38.5% 75% 25% 25% 25% 20% 75% 75% 80% 91.7% 8.3% ≥ 12.5 –
Slide 22: Global Confirmatory Phase 3 Double-blind Placebo-Controlled Study
Global Confirmatory Phase 3 Double-blind Placebo-Controlled Study Dosing of first patient expected Q3 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 tolerability Upright stability (U.S.) and mFARS (E
Slide 23: Rolling BLA Initiated and First Module Submitted; Final Modules Expected 2H 2026
Rolling BLA Initiated and First Module Submitted; Final Modules Expected 2H 2026 Rolling BLA Submission Global Phase 3 Rolling BLA Submission Completion Potential U.S. Launch Pursue path towards approvals in EU, UK, Canada and Australia Initial Module June 2026 Initiate dosing Q3 2026 Remaining modu
Slide 24: Nomlabofusp Advancing as the First Potential Disease Modifying Therapy for Broad FA Population
Nomlabofusp Advancing as the First Potential Disease Modifying Therapy for Broad FA Population Rolling BLA submission initiated First module submitted Completion expected 2H 2026 Dosing of first patient in global Phase 3 trial expected Q3 2026 Sustained FXN levels up to 18 months; Improvements in cl
Slide 25: Appendix Larimar Therapeutics
Appendix Larimar Therapeutics
Slide 26: 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 27: Nomlabofusp Awarded Multiple U.S., EU, and U.K. Regulatory Designations
Nomlabofusp Awarded Multiple U.S., EU, and U.K. Regulatory Designations Intended to Expedite the Development Program Orphan Drug Designation (EU) PRIME Designation (EU) Innovative Licensing and Access Pathway (ILAP) (UK) Breakthrough Therapy Designation START Pilot Program Orphan Drug Designation Fa
Slide 28: Mitochondrial Localization and Preclinical Data
Mitochondrial Localization and Preclinical Data
Slide 29: 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 30: 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 31: 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 32: Nomlabofusp Delivers hFXN to Mitochondria and Restores SDH Activity in KO Mice
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 33: 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 34: 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 35: Non-Interventional Study Data
Non-Interventional Study Data
Slide 36: 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 37: FDA START Pilot Program
FDA START Pilot Program o \.
Slide 38: START Pilot Program Continues to Expedite 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 39: FARA
FARA
Slide 40: 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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