Larimar Therapeutics Inc. (LRMR) announced Monday positive top-line data from phase 2 dose exploration study from 25 mg and 50 mg cohorts of Nomlabofusp in patients with Friedreich's Ataxia or FA.
In pre-market activity on Nasdaq, Larimar shares were losing more than 37 percent to trade at $4.87.
The shares had gained around 24 percent on last Friday to close at $7.76.
The clinical-stage biotechnology company focused on complex rare diseases said it has successfully completed its four-week, placebo-controlled Phase 2 dose exploration study of nomlabofusp (CTI-1601) in participants with FA.
In the trial, Nomlabofusp was generally well tolerated and demonstrated dose dependent increases in frataxin levels in all evaluated tissues following repeated subcutaneous injections in patients in the 25 and 50 mg cohorts with no serious adverse events.
The company noted that dose dependent increases in frataxin levels were observed in skin and buccal cells.
The Open Label Extension or OLE study was initiated in January 2024 to dose 25 mg daily of nomlabofusp, with dosing anticipated to be starting later this quarter. The company expects initial data in the fourth quarter of fiscal 2024.
The company has initiated discussions with the FDA on use of tissue frataxin levels as a novel surrogate endpoint to support a potential Biologics License Application or BLA submission for accelerated approval targeted for second half of 2025.
Carole Ben-Maimon, President, and Chief Executive Officer of Larimar, said, "We believe the dose-response and increases in FXN levels seen in peripheral tissues further reinforce the therapeutic potential of nomlabofusp to address FXN deficiency, the known root cause of disease in patients with FA. ….Together with the consistent results seen across our Phase 2 and Phase 1 studies, we believe these findings suggest that nomlabofusp can achieve tissue FXN levels that may have a clinically meaningful effect on disease progression in patients with FA."
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