Larimar (LRMR) Up on Lifting of Partial Clinical Hold on FA Study

Core Viewpoint - Larimar Therapeutics, Inc. has received FDA approval to lift the partial clinical hold on its nomlabofusp (CTI-1601) clinical program, leading to an 8.5% increase in share price on May 21 [1]. Company Overview - Larimar's lead candidate, nomlabofusp, is being developed for treating Friedreich's ataxia (FA), a rare and fatal nervous system disease caused by frataxin deficiency [2]. - The company has no marketed products currently, making the successful development of nomlabofusp its primary focus [7]. Clinical Study Details - In February 2024, Larimar completed a four-week, placebo-controlled phase II dose exploration study for nomlabofusp, which was well-tolerated [3]. - The FDA's review included data from 25 mg and 50 mg cohorts, with patients receiving daily dosing for 14 days, followed by alternate day dosing until day 28 [3]. Efficacy Results - At day 14, patients treated with 50 mg of nomlabofusp achieved frataxin levels exceeding 33% of the average levels seen in healthy volunteers, with three patients exceeding 50% [4]. Ongoing Studies - Nomlabofusp (25 mg) is currently being evaluated in an open label extension (OLE) study, which will initially investigate daily subcutaneous injections [5]. - The company plans to escalate dosing to 50 mg in the OLE study after further pharmacodynamics characterization at the 25 mg dose, with interim data expected in Q4 2024 [6]. Market Context - Biogen currently markets Skyclarys, the first approved treatment for FA, which was approved by the FDA in February 2023 and in Europe in February 2024 [8]. - The acquisition of Reata Pharmaceuticals by Biogen in September 2023 added this treatment to its portfolio, with ongoing launches in the U.S. and EU [8]. Stock Performance - Year-to-date, Larimar's shares have increased by 73.4%, contrasting with a 6.5% decline in the industry [4].