LARIMAR THERAPEUTICS INC. overview

FDA stated in written correspondence for 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 FDA recommended measuring skin FXN concentrations to support evidence of effectiveness for accelerated approval pathway and acknowledged submitted data appear sufficient to support relationship between increased skin FXN concentrations and relevant tissues such as heart, dorsal root ganglia and...

BALA CYNWYD, Pa., March 03, 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 members of the company’s management team will present and participate in 1x1 investor meetings at the Leerink Partners Global Healthcare Conference, taking place in Miami Beach, FL from March 10 – 12, 2025. Details on the presentation can be found below. Date: Monday,...

Adolescents receive a weight-based dose equivalent to the 50 mg adult dose Adolescents 12-17 years old who complete participation in the pharmacokinetic (PK) run-in study will be eligible to screen in the ongoing open label extension (OLE) study Larimar is continuing to enroll adolescent patients and plans to initiate a cohort of children 2-11 years old in 1H 2025 Long-term 50 mg data from adults in OLE study and available adolescent PK run-in data expected mid-2025 BALA CYNWYD, Pa., Jan. 23,...

Daily subcutaneous injections of 25 mg nomlabofusp in 14 participants were generally well tolerated for up to 260 days in the ongoing open label extension (OLE) study Tissue frataxin (FXN) levels showed mean change from baseline of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90 Tissue FXN levels increased and were maintained over time, with mean levels increasing from 15% of healthy volunteers (HV) at baseline to 30% in buccal cells and from 16% to 72% in skin cells at Day...

Treatment with nomlabofusp modified gene expression and lipid profiles in addition to increasing frataxin (FXN) levels in study participants with Friedreich’s ataxia (FA) Modeling and simulation predict that, in most patients with FA, 50 mg of nomlabofusp administered daily is likely to achieve FXN levels that are ≥ 50% of levels observed in healthy controls and similar to mean FXN levels reported in asymptomatic heterozygous carriers Disease characteristics of adult participants in the...

Nomlabofusp program update expected mid-December to include available safety, pharmacokinetic (PK) and frataxin data, as well as available clinical outcomes observations from patients with Friedreich’s ataxia (FA) receiving 25 mg of nomlabofusp daily for 30-180 days in ongoing open label extension (OLE) study Initiation of PK run-in study in adolescents on track by year-end 2024 Initiation of global confirmatory/registration study planned mid-2025 Biologics License Application (BLA) submission...

BALA CYNWYD, Pa., Sept. 19, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that data from the Company’s nomlabofusp Phase 1 studies and Phase 2 dose exploration study, some of which has been previously disclosed, will be presented at the annual International Congress for Ataxia Research (ICAR) being held November 12-15, 2024 in London, U.K....

BALA CYNWYD, Pa., Aug. 27, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that members of the company’s management team will participate in 1x1 investor meetings at the Wells Fargo Healthcare Conference, taking place September 4-6, 2024 in Boston, MA and the H.C. Wainwright 26th Annual Global Investment Conference, taking place September 9-11, 2024...