On Monday, Larimar Therapeutics, Inc. (NASDAQ: LRMR ) released initial data from the ongoing long-term OLE study evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver in participants with Friedreich’s Ataxia .
Friedreich’s Ataxia is caused by a mutation in the FXN gene, which carries the code for the frataxin protein. Frataxin is important for the normal function of mitochondria, the energy-producing parts of cells
At the time of data cut off for the OLE study, 14 patients were included with up to 260 days (mean 99 days) of long-term daily treatment of 25 mg of nomlabofusp.
More than 50% of these patients were non-ambulatory (bedridden).
Tissue FXN levels showed a mean change from baseline of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90
25 mg of nomlabofusp increased and maintained tissue FXN levels over time, increasing from a mean level of 15% of healthy volunteers at baseline to 30% in buccal cells and from 16% to 72% in skin cells at Day 90
Tissue FXN levels appear to reach steady-state levels by Day 30 in buccal cells (inside of the mouth and lips).
Nomlabofusp was generally well tolerated with two participants who had serious adverse events that resolved within 24 hours and withdrew from the study.
The most common adverse events were injection site reactions, with most being mild, brief, and self-limited.
The company said dose escalation to 50 mg daily in the OLE has been initiated in 6 participants.
Screening of adolescents with FA is ongoing for the pediatric PK run-in study, with dosing expected in early 2025; adolescents who complete study participation will transition into the OLE study after assessment of safety and PK data.
Price Action: LRMR stock is down 21.5% at $4.85 at last check Monday.
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