Larimar Therapeutics Aktie

Interactions with FDA over the past year have provided clear expectations for the path to submission of the nomlabofusp BLA Written FDA recommendations for safety database include a total of at least 30 participants with continuous exposure for 6 months including a subset of at least 10 with 1-year; large majority of the exposure should be on the 50 mg dose BLA submission seeking accelerated approval planned in the second quarter of 2026 to allow for inclusion of the recommended safety data for adults and children OLE data expected in September 2025 from 30-40 participants who received at least one dose of nomlabofusp; data will include participants on the 50 mg dose Adolescent PK run-in data expected in September 2025 from 14 participants (some on placebo); participants currently screening and enrolling into OLE Global Phase 3 study activities are ongoing with identification and qualification of sites in U.S., Europe, U.K., Canada, and Australia Company management to host webcast and conference call today at 8:00 a.m. ET BALA CYNWYD, Pa.» Mehr auf globenewswire.com

BALA CYNWYD, Pa., June 20, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the Company will host a conference call and webcast to discuss regulatory updates for the Company's nomlabofusp clinical development program for the treatment of Friedreich's Ataxia on Monday, June 23, 2025 at 8:00 am EDT.» Mehr auf globenewswire.com

FDA stated as part of a START pilot program meeting that it is open to considering skin FXN concentration as a reasonably likely surrogate endpoint in support of an accelerated approval BLA seeking accelerated approval planned to be submitted by year-end 2025; global Phase 3 study planned to initiate in mid-2025 Completed dosing in adolescent PK run-in study; topline 50 mg dose data from the OLE study and data from adolescent cohort planned for program update in September 2025 Strong balance sheet of $157.5 million cash, cash equivalents and marketable securities as of March 31, 2025, with projected cash runway into second quarter of 2026 BALA CYNWYD, Pa., April 30, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its first quarter 2025 operating and financial results.» Mehr auf globenewswire.com