Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Corporate Update (June 2025)
14 slides from Larimar Therapeutics's corporate update, Jun 23, 2025
Slide 1: Nomlabofusp Regulatory Update
pro June 2025 Larimar Therapeutics Nomlabofusp Regulatory Update Exhibit 99.2
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: Written FDA Recommendations on Safety Database for the Nomlabofusp BLA Submission
Written FDA Recommendations on Safety Database for the Nomlabofusp BLA Submission for Friedreich’s Ataxia Program Interactions with FDA over the past year have provided clear expectations for the path to BLA submission FA: Friedreich’s ataxia; OLE: Open-label extension; BLA: Biologics License Applic
Slide 4: Clear FDA Expectations for Path to BLA Submission Seeking Accelerated Approval
Clear FDA Expectations for Path to BLA Submission Seeking Accelerated Approval Planned in Q2 2026 Elements of BLA Submission FDA Recommendations Use of Skin FXN Concentrations as a Surrogate Endpoint Open to use of increases in skin FXN concentrations as a RLSE Acknowledged submitted data appears to
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 (FXN) levels depending on the tissue, sampling technique, and assay considered* Initial symptoms includ
Slide 6: Nomlabofusp is Designed to Deliver Additional Frataxin
Nomlabofusp is Designed to Deliver Additional Frataxin 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 human FXN in the mi
Slide 7: Open-label Extension: Dosing Continues
Open-label Extension: 25 mg Completed, Dosing at 50 mg Continues Long-term data from 50 mg dose expected September 2025 Key Eligibility Criteria Previous participation in Phase 1 or Phase 2 trials Nomlabofusp or placebo arms; most received nomlabofusp Key Study Objectives Safety and tolerability Lon
Slide 8: Nomlabofusp Daily Increased Skin FXN Levels in the OLE Study
Nomlabofusp 25 mg Daily Increased Skin FXN Levels in the OLE Study 72% at Day 90 (43.86 % min, 160.61 % max) 16% at baseline (9.33% min, 24.48% max) Mean % of healthy volunteers FXN levels measured via detection of peptide derived from mature FXN; FXN concentrations are normalized to total cellular
Slide 9: Observed Trends Towards Improvement in Clinical Outcomes at Day 90 in OLE
Observed Trends Towards Improvement in Clinical Outcomes at Day 90 in OLE After Daily 25 mg Nomlabofusp Visit Statistic mFARS 93-Point Scale FARS-ADL 36-Point Scale Modified Fatigue Impact Scale 84-Point Scale 9 Hole Peg Test Dominant Hand Time (Seconds) N = 8 N = 8 N = 8 N = 8 Baseline Mean (SD) 55
Slide 10: Nomlabofusp is Generally Well-Tolerated with Long-Term Treatment
Nomlabofusp is Generally Well-Tolerated with Long-Term Treatment First potential disease modifying therapy to treat FA, a rare and progressive neurodegenerative disease Anaphylaxis has been deemed an adverse drug reaction likely related to nomlabofusp by the Larimar Safety Team Participants with pri
Slide 11: Recent Updates to the OLE Study Design
Recent Updates to the OLE Study Design Commercial Formulation Antihistamine Premedication Adolescent Expansion Program Expansion Premedication with antihistamines starting 5 days prior to the first dose and continuing for the first month in participants with prior exposure who have been off treatmen
Slide 12: START Pilot Program Continues to Expedite 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 13: Potential Path to Bring Nomlabofusp to Patients Worldwide
Potential Path to Bring Nomlabofusp to Patients Worldwide Received feedback from FDA and EMA on study protocol Potential sites in the U.S., Europe, U.K., Canada, and Australia Study Design* Double-blind placebo-controlled study Ambulatory patients (n = 100 – 150) weighted to younger patients Include
Slide 14: Nomlabofusp Advancing Towards BLA Submission for FA
Nomlabofusp Advancing Towards BLA Submission for FA First potential disease modifying therapy Designed to systemically address FXN deficiency in FA FDA clarity on key BLA elements Skin FXN concentrations as a surrogate endpoint Safety database of at least 30 for 6-mos, and a subset of at least 10 fo