Larimar Therapeutics Announces Positive Initial Data from Ongoing Long-term Open Label Extension Study & Progress Across Nomlabofusp Program for Friedreich’s Ataxia

Core Insights - Larimar Therapeutics announced positive initial data from the ongoing open label extension (OLE) study of nomlabofusp, indicating it is generally well tolerated and shows potential clinical benefits for patients with Friedreich's ataxia (FA) [1][2] Group 1: Study Results - Daily subcutaneous injections of 25 mg nomlabofusp were well tolerated for up to 260 days in 14 participants [1] - Tissue frataxin (FXN) levels increased significantly, with buccal cells rising from 15% to 30% and skin cells from 16% to 72% at Day 90 [1][2] - Early trends towards improvement in clinical outcomes were observed at Day 90, suggesting potential clinical benefits across a broad spectrum of FA patients [1][4] Group 2: Pharmacokinetics and Safety - Pharmacokinetic data indicated that nomlabofusp levels in plasma reached a steady state by Day 30, with no further accumulation noted [1][4] - The most common adverse events were mild injection site reactions, with serious adverse events occurring in two participants that resolved within 24 hours [3][2] Group 3: Development Plans - The dose of nomlabofusp has been escalated to 50 mg daily for six participants, with plans to increase the dose for all current participants [5][2] - Screening for adolescents in a pediatric pharmacokinetic study is ongoing, with dosing expected to start in early 2025 [1][5] - A global confirmatory/registration study is planned to initiate in mid-2025, with a Biologics License Application (BLA) submission targeted for the second half of 2025 [1][6] Group 4: Financial Position - As of September 30, 2024, the company reported a strong balance sheet with $203.7 million in cash and investments, providing a runway into the second quarter of 2026 [1]