The clinical trial evaluated 25 adults with HAE type 1 or 2, comparing three distinct dosing schedules: 600 mg every 24 weeks, 300 mg every 24 weeks, and 300 mg every 12 weeks. Data collected through day 169 indicates that the treatment effectively silences plasma prekallikrein messenger RNA, which is a key driver of the condition. In the primary endpoint analysis, patients experienced dramatic decreases in monthly attack rates, with reductions reaching as high as 96% in the 300 mg Q24W cohort.
Beyond frequency, the therapeutic impact is characterized by both efficacy and durability. A significant portion of participants remained attack-free throughout the observation period, and plasma prekallikrein levels showed sustained suppression of up to 94%. Dr. Dongxu Shu, CEO of Argo Biopharma, noted that the trial results support the program's advancement toward addressing the industry-wide need for prophylactic options that do not require frequent administration. Safety assessments revealed no treatment-related discontinuations or deaths, with observed adverse events limited mostly to mild, transient injection-site reactions.

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