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NEWS
Larimar Therapeutics Announces Positive Initial Data from Ongoing Long-term Open Label Extension Study & Progress Across Nomlabofusp Program for Friedreich's Ataxia
Monday, 16th December at 7:00 am
• 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 90
• Early trends towards improvement in clinical outcomes were observed at Day 90, supporting the potential that nomlabofusp administration may result in a clinical benefit across a broad spectrum of patients with Friedreich's ataxia (FA)
• Pharmacokinetic (PK) data suggest that nomlabofusp levels in plasma appeared to reach steady state by Day 30 with no further accumulation following long-term daily administration
• Dose escalation to 50 mg daily in the OLE has initiated in 6 participants to date
• Screening of adolescents with FA is ongoing for the pediatric PK run-in study with dosing expected early 2025; adolescents who complete study participation will transition into OLE study after assessment of safety and PK data
• Initiation of global confirmatory/registration study planned mid-2025
• Biologics License Application (BLA) submission targeted for 2H 2025 to support potential accelerated approval
• Strong balance sheet with $203.7 million of cash and investments as of September 30, 2024, with projected runway into the second quarter of 2026
• Company management to host webcast and conference call today at 8:00 a.m. ET
Monday, 16th December at 7:00 am
• 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 90
• Early trends towards improvement in clinical outcomes were observed at Day 90, supporting the potential that nomlabofusp administration may result in a clinical benefit across a broad spectrum of patients with Friedreich's ataxia (FA)
• Pharmacokinetic (PK) data suggest that nomlabofusp levels in plasma appeared to reach steady state by Day 30 with no further accumulation following long-term daily administration
• Dose escalation to 50 mg daily in the OLE has initiated in 6 participants to date
• Screening of adolescents with FA is ongoing for the pediatric PK run-in study with dosing expected early 2025; adolescents who complete study participation will transition into OLE study after assessment of safety and PK data
• Initiation of global confirmatory/registration study planned mid-2025
• Biologics License Application (BLA) submission targeted for 2H 2025 to support potential accelerated approval
• Strong balance sheet with $203.7 million of cash and investments as of September 30, 2024, with projected runway into the second quarter of 2026
• Company management to host webcast and conference call today at 8:00 a.m. ET
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