Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Corporate Update (July 2023)
12 slides from Larimar Therapeutics's corporate update, Jul 25, 2023
Slide 1: Larimar Therapeutics CTI-1601 Program Update
July 2023 Larimar Therapeutics CTI-1601 Program Update
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: CTI-1601 Cleared by FDA to Advance Clinical Development
CTI-1601 Cleared by FDA to Advance Clinical Development FDA review of Larimar Complete Response to its partial clinical hold clears initiation of both 50 mg cohort in Phase 2 dose exploration trial and OLE trial Phase 2 Trial Proceeding to 50 mg Cohort OLE Trial Cleared for Initiation Next Steps FDA
Slide 4: Investment Highlights
Investment Highlights Clinical-stage biotechnology company focused on addressing unmet needs in Friedreich's ataxia (FA) and potentially other complex rare diseases based on a platform technology backed by a strong intellectual property portfolio Lead candidate CTI-1601 is a recombinant fusion prote
Slide 5: Friedreich's ataxia caused by genetic defect resulting in low frataxin levels
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 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 7: Increases in FXN Levels in Phase 2 Trial's 25 mg Cohort
Increases in FXN Levels in Phase 2 Trial’s 25 mg Cohort FXN* Change from Baseline (Skin Cells) *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 percentile
Slide 8: Clinical Data Indicate CTI-1601 is Generally Well Tolerated
Clinical Data Indicate CTI-1601 is Generally Well Tolerated CTI-1601 administered to 37 different adults with FA in multiple studies 35 of 37 clinical trial participants dosed with CTI-1601 completed their respective study One Phase 2 participant withdrew due to an allergic reaction that resolved wi
Slide 9: Phase 2 Dose Exploration Study Advancing to 50 mg Cohort
Phase 2 Dose Exploration Study Advancing to 50 mg Cohort 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
Slide 10: Open-label Extension Trial: Initiation Expected in Q1 2024
Open-label Extension Trial: Initiation Expected in Q1 2024 Preliminary interim data expected in Q4 2024 Screening Period ≤ 42 days Key Eligibility Criteria Previous participation in Phase 1 or Phase 2 trials Subcutaneous Injections of 25 mg CTI-1601 Treatment Period: Planned for ≥ 1 year with any ne
Slide 11: CTI-1601 Clinical Development Plan
CTI-1601 Clinical Development Plan Ongoing and Planned Trials* Include: Global double-blind placebo-controlled pivotal trial*** Open-label extension trial with 25 mg daily dosing for eligible patients who participated in SAD, MAD, and/or four-week dose exploration studies. Initiation expected Q1 202
Slide 12: Regulatory Updates and Expected Milestones
Regulatory Updates Q3 2023: Initiation of 50 mg cohort in Phase 2 dose exploration trial Q1 2024: Initiation of open-label extension trial 1H 2024: Top-line data from Phase 2 trial’s 50 mg cohort Q4 2024: Interim data from open-label extension trial Expected Milestones CTI-1601 Phase 2 dose explorat