As of December 31, 2023, the Company had cash, cash equivalents and marketable securities totaling $86.8 million. “This year we made tremendous progress across key clinical and regulatory milestones for our nomlabofusp program. We were thrilled to recently report positive top-line data and successful completion of our Phase 2 dose exploration study. Nomlabofusp was generally well-tolerated and demonstrated dose-dependent increases in skin and buccal cell frataxin levels. Importantly, skin frataxin levels as a percentage of levels in healthy volunteers more than doubled in all patients after 14 days of daily treatment with 50 mg. The clear dose-response and the magnitude of increase in tissue frataxin levels further reinforces the therapeutic potential of nomlabofusp to address frataxin deficiency, the known root cause of disease in patients with Friedreich’s ataxia (FA),” said Carole Ben-Maimon, president, and CEO of Larimar. “Additionally, the first patient in our OLE study has been dosed. The OLE study will inform on the long-term safety profile and long-term tissue frataxin levels. We remain on track to report interim data from the OLE study in the fourth quarter of 2024.”
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