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Clinical Trials

Vamorolone is an investigational drug being developed for Duchenne muscular dystrophy. While vamorolone may be found to show improved efficacy and safety profiles in many other disorders, we chose DMD as the initial indication due to commitment of the founders for this disease, and the effective partnerships with muscular dystrophy foundations worldwide.

Vamorolone was tested in 86 healthy adult men in 2015. In addition to studying the pharmacokinetics of the drug, doses were escalated safely through 20mg/kg/day for 14 days. The results of this Phase 1 trial are published here.

A sequential series of three clinical trials of 48 DMD boys (ages 4 to <7 years) has completed enrollment (VBP15-002; VBP15-003; VBP15-LTE). VBP15-002 (Phase 2a) was an open-label multiple ascending dose study with four dose groups of vamorolone (0.25 mg/kg/day, 0.75 mg/kg/day, 2.0 mg/kg/day, 6.0 mg/kg/day; 12 patients per dose group). In VBP15-002 (now completed), patients received two weeks of vamorolone, followed by a two week wash-out period, with the primary goals of safety, pharmacokinetics, and pharmacodynamic biomarker studies. VBP15-003 (now completed) was a 24 week extension study where patients continued on the same dose of vamorolone. The primary goals were safety and efficacy (improvement in strength and function). VBP15-LTE is a 2 year long-term extension study, where patients dose escalate (e.g. the patients in the 0.25 mg/kg/day group in the
VBP15-002/VBP15-003 studies can increase dose to the highest dose – 6.0 mg/kg/day – at the wishes of the family and their physician). VBP15-LTE is ongoing, with expected completion ~May 2020.

An additional trial of vamorolone in 120 DMD boys, VBP15-004, began enrollment in August 2018. This is a placebo-controlled, prednisone-controlled, double blind study of two doses of vamorolone (2.0 and 6.0 mg/kg/day). The trial has two 24 week periods, for a total of 48 weeks. Period 1 has randomization of patients to one of four groups (2.0 mg/kg/day vamorolone; 6.0 mg/kg/day vamorolone; 0.75 mg/kg/day prednisone; placebo). After the 24 week Period 1 has ended, the prednisone and placebo patients begin treatment with vamorolone for 24 week Period 2. Thus, in Period 2 all patients are treated with vamorolone. This study is currently recruiting (NCT03439670); read it here.

The DMD clinical trial program was developed with guidance from the FDA (Phase 1 – Phase 2 transition meeting), EMA CHMP (two rounds of scientific guidance), and the EMA Paediatric Investigation Plan (PIP).