Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Corporate Update (February 2024)
22 slides from Larimar Therapeutics's corporate update, Feb 12, 2024
Slide 1: Nomlabofusp (CTI-1601) Program Update
February 2024 Larimar Therapeutics Nomlabofusp (CTI-1601) Program Update Exhibit 99.3
Slide 2: 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 historical fact are forward-look
Slide 3: Positive Topline Phase 2 Data for Nomlabofusp (CTI-1601)
Positive Topline Phase 2 Data for Nomlabofusp (CTI-1601) Dose-dependent increases in tissue frataxin (FXN) levels in skin and buccal cells Nomlabofusp was generally well-tolerated following repeated subcutaneous injections up to 28 days Participants treated with 50 mg for 14 days and then every othe
Slide 4: Clinical-Stage Novel Protein Replacement Therapy Platform
Clinical-Stage Novel Protein Replacement Therapy Platform Lead candidate nomlabofusp* (CTI-1601) is a recombinant fusion protein designed to directly address frataxin deficiency in patients with Friedreich's ataxia (FA) by delivering the protein to mitochondria. Granted Orphan Drug (US & EU), Rare P
Slide 5: Friedreich's Ataxia (FA): A rare and progressive disease
Friedreich’s Ataxia (FA): A rare and progressive disease 5 * E.C. Deutsch et al. Molecular Genetics and Metabolism 101 (2010) 238–245. Most patients with FA only produce ~20-40% of normal frataxin levels depending on the tissue, sampling technique, and assay considered* Genetic defect on both allele
Slide 6: FXN Levels Predict Disease Progression in FA
FXN Levels 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 Ambulation (Y
Slide 7: Phase 2 Dose Exploration Study for 25 mg and 50 mg Cohorts
Phase 2 Dose Exploration Study for 25 and 50 mg Cohorts Goal: Further characterize PK/PD and assess safety to inform long-term dose and dose regimen Treatment Schedule - nomlabofusp (CTI-1601) or placebo 28-day Treatment Period 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
Slide 8: Demographics – Phase 2 Trial
Demographics – Phase 2 Trial 25 mg Cohort 50 mg Cohort Placebo N = 4 Nomlabofusp N = 9 Overall N = 13 Placebo N = 5 Nomlabofusp N = 10 Overall N = 15 Age at Screening (Years) Mean (SD) 34.0 (9.20) 37.8 (14.93) 36.6 (13.16) 28.6 (4.67) 28.1 (11.00) 28.3 (9.17) Median 33 31 31 27 24 26 Q1, Q3 27, 42 2
Slide 9: Disease Characteristics – Phase 2 Study
Disease Characteristics – Phase 2 Study 25 mg Cohort 50 mg Cohort Placebo N = 4 Nomlabofusp N = 9 Overall N = 13 Placebo N = 5 Nomlabofusp N = 10 Overall N = 15 Age at Symptom Onset (Years) Mean (SD) 14.5 (4.93) 13.0 (10.47) 13.5 (8.77) 15.2 (7.26) 13.7 (8.37) 14.2 (7.78) Median 14.5 10 11 14 12.5 1
Slide 10: Dose-Dependent Increase in FXN Levels in Skin Cells
Dose-Dependent Increase in FXN Levels in Skin Cells Skin Cells FXN Levels* Change from Baseline** FXN Levels* in Skin Cells Change from Baseline at Day 14 Participants dosed daily for 14 days, then every other day until day 28 *FXN levels measured via detection of peptide derived from mature FXN; FX
Slide 11: Dose-Dependent Increase in FXN Levels in Buccal Cells
Dose-Dependent Increase in FXN Levels in Buccal Cells Buccal Cells FXN Levels* Change from Baseline** FXN Levels* in Buccal Cells Change from Baseline at Day 14 Participants dosed daily for 14 days, then every other day until day 28 *FXN levels measured via detection of peptide derived from mature F
Slide 12: Absolute Increases in Skin FXN Levels
Absolute Increases in Skin FXN Levels Day 14 Skin FXN Levels Dose Visit Absolute Values (pg/µg) Median Mean 25 mg Baseline 3.70 3.38 Day 14 5.53 6.40 Change from Baseline 2.81 3.02 50 mg Baseline 2.12 2.08 Day 14 7.40 7.32 Change from Baseline 5.57 5.24 Dose response in tissue FXN concentrations and
Slide 13: Absolute Increases in Buccal FXN Levels
Absolute Increases in Buccal FXN Levels Day 14 Buccal FXN Levels Dose Visit Absolute Values (pg/µg) Median Mean 25 mg Baseline 1.78 1.80 Day 14 2.24 2.22 Change from Baseline 0.56 0.42 50 mg Baseline 1.61 1.69 Day 14 2.44 2.38 Change from Baseline 0.72 0.69 Dose response in tissue FXN concentrations
Slide 14: 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 15: Buccal Cell FXN Levels Achieve Higher % of Healthy Volunteers
Buccal 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
Slide 16: Increasing FXN Levels May Slow Disease Progression
Increasing FXN Levels May Slow Disease Progression *FARS: Friedreich’s ataxia rating scale, measures disease progression with a higher score indicating a greater level of disability. **FXN levels measured in peripheral blood mononuclear cells (PBMCs). FXN levels as measured by % of normal demonstrat
Slide 17: Encouraging Therapeutic Potential for Nomlabofusp
Encouraging Therapeutic Potential for Nomlabofusp Continue nomlabofusp clinical development Dose-dependent increases in frataxin levels with nomlabofusp in several studies Frataxin deficiency is the root cause of the disease Lower levels of frataxin correlate with disease burden Animal models show t
Slide 18: Nomlabofusp: Predictable Pharmacokinetics
Nomlabofusp: Predictable Pharmacokinetics Quick absorption after subcutaneous administration 1 2 3 Dose-proportional increases in exposure observed cc Pharmacokinetic profile consistent with Phase 1 studies
Slide 19: Ph1 & Ph2 Data: Nomlabofusp is Generally Well Tolerated
Ph1 & Ph2 Data: Nomlabofusp is Generally Well Tolerated 44 of 46 clinical trial participants dosed with nomlabofusp completed their respective study One Phase 2 participant in the 25 mg cohort withdrew due to allergic reaction that resolved with standard treatment One Phase 1 participant in the 50 m
Slide 20: Open-label Extension Study: Initiated Q1 2024
Open-label Extension Study: Initiated Q1 2024 Preliminary interim data expected in Q4 2024 Key Eligibility Criteria Previous participation in Phase 1 or Phase 2 trials Key Study Objectives Safety and tolerability Long-term PK Tissue FXN concentrations and potential use as surrogate endpoint to suppo
Slide 21: Nomlabofusp Clinical Development Plan
Nomlabofusp Clinical Development Plan Planned global double-blind placebo-controlled registration/confirmatory study** Planned pediatric MAD trial in patients 2 to 17 years of age* Intend to pursue accelerated approval pathway with potential BLA submission targeted for 2H 2025 *Company is discussing
Slide 22: Regulatory Updates
Regulatory Updates Q1 2024: Dosing of first patient in OLE study Q4 2024: Initial data from OLE study; initiated in Q1 2024 2H 2024: Final Phase 2 data planned to be presented at a conference 2H 2025: BLA submission Expected Milestones Consistent Ph 1 and Ph 2 Findings Initiated discussions with FDA
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