Larimar Therapeutics(LRMR) Newsfilter·2024-12-16 12:00Core 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][4] Group 1: Study Results - Daily subcutaneous injections of 25 mg nomlabofusp were administered to 14 participants for up to 260 days, with a mean treatment duration of 99 days [4] - Tissue frataxin (FXN) levels increased significantly, with buccal cells showing a rise from 15% of healthy volunteers at baseline to 30% at Day 90, and skin cells from 16% to 72% [1][5] - Early trends towards improvement in clinical outcomes were observed, including decreased values in modified Friedreich Ataxia Rating Scale (mFARS) and other clinical measures at Day 90 [5][6] 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][5] - The treatment was generally well tolerated, with serious adverse events occurring in two participants that resolved within 24 hours [5][6] Group 3: Future Plans and Developments - The company has initiated dose escalation to 50 mg daily in the OLE study for six participants, with plans to increase the dose for all participants [1][6] - A pediatric pharmacokinetic run-in study is ongoing, with dosing expected to start early 2025, and adolescents completing the study will transition into the OLE [1][6] - 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][7] 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 projected runway into the second quarter of 2026 [1][6]