Larimar Therapeutics announced Monday that its Friedreich’s ataxia treatment increased median levels of a key protein called frataxin in a mid-stage study.
The biotech is in talks with the FDA to use frataxin biomarker data to support a potential accelerated approval submission in 2025, though its treatment is still under partial clinical hold.
In the Phase II study, patients with Friedreich’s ataxia received either 25 mg or 50 mg of nomlabofusp or placebo. Their skin cells were biopsied and buccal cells (the cells inside the cheek) were swabbed to measure levels of frataxin. When people lack frataxin, a mitochondrial protein, it results in muscle weakness and a loss of balance and coordination, among other symptoms.
Two weeks after being treated every day, 10 patients who received 50 mg of nomlabofusp saw the median level of frataxin in their skin cells increase by 5.57 pg frataxin/µg of total protein compared to…
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