Santhera Announces Publication of Efficacy, Safety and Tolerability Data with Vamorolone (AGAMREE) in Patients with Duchenne Muscular Dystrophy in Neurology

• Results from 112 patients with DMD who completed the study confirm maintenance of efficacy and benefits in safety and tolerability of treatment with vamorolone over 48 weeks
• AGAMREE has shown safety benefits in patients switching from standard of care corticosteroids in terms of recovery of bone health and growth
• More than 200 patients have now been treated with vamorolone for up to 84 months across clinical studies and access programs
• AGAMREE is the only approved medication in the European Union (EU) for treating all patients from age 4 years with DMD [1], and the first DMD treatment approved across the U.S., EU and UK

Pratteln, Switzerland, February 14, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the publication of the paper “Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy” in the peer-reviewed journal Neurology [2]. The publication reports the results of the 48-week treatment with vamorolone in patients with DMD in the VISION-DMD study, supporting the long-term efficacy and safety profile of vamorolone and concluding that vamorolone was generally well tolerated, consistent with the 24-week study findings, as published previously in JAMA Neurology [3].

The Neurology publication states:

“Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD) [3]. This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone).

A total of 121 participants with DMD were randomized. Vamorolone at a dose of 6 mg/kg/d showed maintenance of improvement for all motor outcomes to week 48 (e.g., for primary outcome, time to stand from supine [TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052 [0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48 weeks, vamorolone at a dose of 2 mg/kg/d showed similar improvements as 6 mg/kg/d for North Star Ambulatory Assessment (NSAA) (vamorolone 6 mg/kg/d–vamorolone 2 mg/kg/d LSM [SE] 0.49 [1.14]; 95% CI −1.80 to 2.78, p = 0.67), but less improvement for other motor outcomes. The placebo to vamorolone 6 mg/kg/d group showed rapid improvements after 20 weeks of treatment approaching benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for TTSTAND, time to run/walk 10 m, and NSAA. There was significant improvement in linear growth after crossover in the prednisone to vamorolone 6 mg/kg/d group, and rapid reversal of prednisone-induced decline in bone turnover biomarkers in both crossover groups. There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups.