Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Corporate Update (June 2026)
26 slides from Larimar Therapeutics's corporate update, Jun 29, 2026
Slide 1: Larimar Therapeutics Nomlabofusp Program Update
pro June 2026 Larimar Therapeutics Nomlabofusp Program Update
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: Initiated Rolling BLA Submission for Accelerated Approval; Positive Open Label Study Data
Initiated Rolling BLA Submission for Accelerated Approval; Positive Open Label Study Data Positive Longer-Term OL Data Skin FXN levels continue to be similar to those of asymptomatic carriers at 6, 12 and 18 months Improvement in clinical outcome measures with 13 participants at 1 year Long term dos
Slide 4: Alignment with FDA for Submission of BLA Package for Accelerated Approval
Alignment with FDA for Submission of BLA Package for Accelerated Approval Following 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 nomlabofusp
Slide 5: 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 21 Discontinued* Maximum duration >800 days ~11 Adults and adolescents in scree
Slide 6: Additional Patients with Longer Exposure Reinforces Disease Modifying Potential of Nomlabofusp
Additional Patients with Longer Exposure Reinforces the Disease Modifying Potential of Nomlabofusp 13 participants completed 1 year of dosing, 7 completed 18 months, and 3 completed 2 years Well-Characterized Safety Profile Increased Tissue FXN Levels Observed Improvement of Clinical Outcomes Daily
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. Intended to help patients avoid profound suffering and maint
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: 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 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 in Open Label Study
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 with Nomlabofusp Relative to 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 Outcomes
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 = 7
Slide 20: Different Clinical Outcome Measures May Be More Or Less Sensitive At Different Points
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 ≥ 12.5 – <25 Years Duration ≥ 25 Y
Slide 21: Majority Achieved Improvement at Year With Certain Outcome Measures More Sensitive
Majority Achieved Improvement at 1 Year With Certain Outcome Measures More Sensitive at Different Times in Disease Course mFARS FARS-ADL 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%
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 (
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
Appendix
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