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Larimar Therapeutics, Inc. (LRMR) on Monday announced positive data from an ongoing long-term open-label study evaluating daily subcutaneous injections of Nomlabofusp in participants with Friedreich’s Ataxia.
Friedreich's Ataxia (FA) is a rare, inherited disorder causing progressive damage to the nervous system, leading to symptoms like poor coordination, difficulty walking, and sensory loss, as well as heart disease and scoliosis. In the study, an increase in skin frataxin (FXN) levels was observed with short- and long-term daily Nomlabofusp.
“These new data, as well as the improvement in abnormal lipid profiles observed in prior completed studies, provide support that nomlabofusp increases FXN in patients with FA and that the strategy of FXN replacement has the potential to result in a clinical benefit,” said CEO Carole Ben-Maimon.
Shares of the company, however, traded 31% lower in the pre-market session after the company said that anaphylaxis or a severe allergic reaction was reported in seven participants in the open-label study.
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