Following our Phase 1 trial in healthy volunteers with Resolaris, we embarked on a clinical development program consisting of Phase 1b/2 clinical trials to treat patients with Resolaris for multiple rare myopathies with an immune component; including
- Adult Limb girdle muscular dystrophy 2B;
- Adult Facioscapulohumeral muscular dystrophy (FSHD); and
- Early onset FSHD
The planning and design of our exploratory Phase 1b/2 trials were guided by principles related to safety and tolerability. In addition, the studies also evaluated certain clinical assessments and the utility of exploratory pharmacodynamic (PD) markers (including MRI measurements to quantitate areas of potential muscle inflammation).
Ongoing Clinical Trials
Early Onset FSHD (003) Trial: Phase 1b/2 »
In the fourth quarter of 2015, we initiated a multinational, multi-center Phase 1b/2 clinical trial of Resolaris in patients with early onset FSHD. This international Phase 1b/2 clinical trial is an open-label, intra-patient dose escalation study designed to assess the safety, tolerability, immunogenicity and biological activity of Resolaris at weekly doses of 0.3, 1.0 and 3.0 mg/kg to patients with early onset FSHD.
Study protocol is to enroll 8 patients between the ages of 16 and 25 and 8 patients between the ages of 12 and 15.
Stage 1 completed enrollment in August 2016. We announced interim data from our first four patients enrolled in Stage 1 in the fourth quarter of 2016.
LGMD2B/FSHD (004) Trial: Phase 1b/2 »
In the fourth quarter of 2015, we initiated a multinational, multi-center Phase 1b/2 clinical trial of Resolaris in adult patients with LGMD2B or FSHD. This international Phase 1b/2 clinical trial is an open-label, intra-patient dose escalation study designed to assess the safety, tolerability, immunogenicity and biological activity of weekly and twice weekly intravenous infusions of Resolaris in adults with either LGMD2B or FSHD.
We completed enrollment ahead of schedule in May of 2016 with 18 total patients (8 with FSHD and 10 with LGMD), exceeding our stated target enrollment of 16 patients.
We announced results in the fourth quarter of 2016.
FSHD (002) Trial: Phase 1b/2 »
In March of 2016, we announced results from our multi-national exploratory Phase 1b/2 clinical trial of Resolaris in adult patients with FSHD in the United States and European Union. This randomized, double-blind, placebo-controlled trial was designed to evaluate the safety, tolerability, pharmacokinetics and exploratory pharmacodynamics markers and clinical assessments of multiple intravenous doses of Resolaris in adults with FSHD.
Resolaris was studied in three dose escalation cohorts (0.3, 1.0 and 3.0 mg/kg) across four sites and 20 patients. In each cohort, patients were randomized at a ratio of 3:1 to receive Resolaris or placebo. Patients in the first two cohorts were dosed weekly over a period of one month, and patients in the third cohort were dosed weekly over a period of three months.
We believe the safety, tolerability, immunogenicity and activity profile of Resolaris as demonstrated in this study warrants advancing our program in adult FSHD patients and potentially other rare diseases.