New five-year data on Agamree in patients with DMD

Swiss specialty drugmaker Santhera Pharmaceuticals (SIX: SANN) saw its shares rise 5.8% to 10.90 francs today, after it reported positive top-line results from an analysis of long-term data, including first assessments from the ongoing, open-label, multicenter GUARDIAN study evaluating Agamree (vamorolone) in patients with Duchenne muscular dystrophy (DMD).

The long-term analysis included corticosteroid-naïve patients who began Agamree treatment between four and seven years of age in clinical studies and continued through various access programs, including the GUARDIAN study. Data from up to 110 patients were analyzed, with patient numbers varying by analysis depending on data availability. In this long-term analysis, patients had received Agamree for up to eight years, with a median follow-up of about five years. Most patients remained on higher doses (4–6 mg/kg/day) in real-world clinical settings over the observation period.

Patients treated with Agamree maintained motor function over extended follow-up, demonstrating durable efficacy as measured by time to loss of ambulation comparable to standard of care corticosteroids (p=0.91). In pre-specified subgroup analyses, no differences were observed compared with either daily deflazacort or prednisone.