Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Corporate Update (May 2023)
15 slides from Larimar Therapeutics's corporate update, May 15, 2023
Slide 1: CTI-1601 Program Update
May 2023 Larimar Therapeutics CTI-1601 Program Update Exhibit 99.2
Slide 2: Forward-Looking Statements
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 histo
Slide 3: Preliminary Top-line Data from 25 mg cohort of Ph 2 Trial
Preliminary Top-line Data from 25 mg cohort of Ph 2 Trial Frataxin (FXN) Levels A meeting between Larimar and FDA is scheduled to discuss the information needed to gain clearance to initiate a 50 mg cohort in the Phase 2 trial Update on CTI-1601 program expected in Q3 2023 Next Steps Participants do
Slide 4: Investment Highlights
Investment Highlights Double-blind, placebo-controlled Phase 1 proof-of-concept trials in individuals with FA complete Data show that CTI-1601 was generally well tolerated when dosed daily for up to 13 days & dose dependent increases in frataxin (FXN) levels from baseline compared to placebo in all
Slide 5: Friedreich's ataxia caused by genetic defect resulting in low frataxin
Caused by genetic defect resulting in low levels of frataxin Patients with FA only produce ~20-40% of normal frataxin levels depending on the tissue, sampling technique, and assay considered1 Affects ~20,000 patients globally, with ~5,000 patients in the U.S. and majority of the remaining patients i
Slide 6: Dose Dependent Increases in FXN Levels Observed 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 7: Increases in FXN Correlated with Increasing CTI-1601 Dose in Phase 1 Trial
Increases in FXN Correlated with Increasing CTI-1601 Dose in Phase 1 Trial Skin Biopsies Day 13 FXN* Change from Baseline# Buccal Cells Day 4/7 FXN* Change from Baseline◊ *FXN levels measured via detection of peptide derived from mature FXN. FXN concentrations are normalized to total cellular protei
Slide 8: Ongoing Phase 2, Four-week Dose Exploration Study
Ongoing Phase 2, Four-week Dose Exploration Study Goal: Further characterize PK/PD and assess safety to inform long-term dose and dose regimen Treatment Schedule 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 14 = Administration of CTI-1601 or placebo
Slide 9: Demographics
Demographics Demographics similar between Phase 1 and Phase 2 trials of CTI-1601 Parameter, n (%) Placebo (N=4) CTI-1601 25 mg (N=9) Overall (N=13) Mean Age (SD) (Years) 34.0 (9.20) 37.8 (14.93) 36.6 (13.16) Male 2 (50.0%) 5 (55.6%) 7 (53.8%) White 4 (100.0%) 8 (88.9%) 12 (92.3%) Other 0 1 (11.1%) 1
Slide 10: Disease Characteristics
Disease Characteristics Parameter Statistic Placebo (N=4) CTI-1601 (n=9) Overall (n=13) Age at Symptom Onset (years) n 4 8 12 Mean (SD) 14.5 (4.93) 13.0 (10.47) 13.5 (8.77) Median 14.5 10.0 11.0 Q1, Q3 11, 19 8, 13 9, 15 Min, Max 9, 20 5, 38 5, 38 Age at Diagnosis (years) n 4 9 13 Mean (SD) 17.5 (5.
Slide 11: CTI-1601 Generally Well Tolerated in Phase 2 25 mg Cohort
CTI-1601 appeared to be generally well tolerated in Phase 2 trial’s 25 mg cohort Pharmacokinetic Data Suggest steady state achieved by day 14 Summary of Phase 2 trial safety data (25 mg cohort): 25 mg CTI-1601 or placebo were administered subcutaneously daily for 14 days and then every other day unt
Slide 12: Increases in FXN Levels Observed in Skin Biopsies
Increases in FXN Levels Observed in Skin Biopsies Median placebo-adjusted increase from baseline of 3.5 pg/µg in FXN levels in skin with 14 days daily dosing *FXN levels measured via detection of peptide derived from mature FXN; FXN concentrations are normalized to total cellular protein content in
Slide 13: Increases in FXN Levels Observed in Buccal Cells
Increases in FXN Levels Observed in Buccal Cells Median placebo-adjusted increase from baseline of 0.9 pg/µg in FXN levels in buccal cells with 14 days daily dosing *FXN levels measured via detection of peptide derived from mature FXN; FXN concentrations are normalized to total cellular protein cont
Slide 14: CTI-1601 Clinical Development Plan
CTI-1601 Clinical Development Plan Planned Trials Include: Global double-blind placebo-controlled pivotal trial. Jive open-label extension trial for eligible patients who participated in SAD, MAD, and/or four-week dose exploration studies. MAD trial in patients 2 to 17 years of age. Participants eli
Slide 15: CTI-1601: First Therapeutic Intended to Increase FXN
15 Phase 2 Generally well tolerated; Dose-dependent increases in FXN levels observed in all evaluated tissues with 7 days of daily dosing at 50 mg and 100 mg Phase 1 Generally well tolerated Median placebo-adjusted increase from baseline of 3.5 pg/µg in FXN levels in skin with 14 days daily dosing M