Cash, cash equivalents and marketable securities totaled $915.9M as of March 31. The company said, “As we move forward with the planned initiation of our global Phase 3 HARBOR(TM) study for del-desiran this quarter, we continue to work diligently to improve people’s lives by advancing our AOC platform. In March, we announced new long-term del-desiran data from our MARINA-OLE study showing reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT), muscle strength and activities of daily living when compared to END-DM1 natural history data. Importantly, we also secured agreement with global regulators on the inclusion of these endpoints in the HARBOR study, including vHOT as the primary endpoint. We look forward to sharing preliminary data in approximately half of the study participants in our Phase 1/2 FORTITUDE(TM) trial in people living with FSHD this quarter and 5 mg/kg cohort data from our Phase 1/2 EXPLORE44 trial of people living with DMD44 in the second half of this year.”
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