Larimar Therapeutics Begins Dosing Adolescents in Friedreich's Ataxia Treatment Study

MT Newswires Live01-23

Larimar Therapeutics (LRMR) said Thursday it has begun dosing adolescents aged 12 to 17 in its pediatric pharmacokinetic, or PK, run-in study of nomlabofusp, a treatment for Friedreich's ataxia.

In the study, participants will receive weight-based doses matching the 50mg adult dose and are randomized 2:1 to receive either nomlabofusp or a placebo daily for seven days, the biotechnology company said. Adolescents who complete the PK run-in study will be eligible to join the ongoing open-label extension, or OLE study.

The OLE study assesses safety, tolerability, PK, and Tissue frataxin levels in buccal and skin cells, as well as exploratory pharmacodynamic markers and clinical outcomes during long-term subcutaneous administration of nomlabofusp.

Larimar shares were up 1% in recent after-hours activity.

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