INTERIM ANALYSIS

A multicenter, nationwide, retrospective registry of 24 patients who were refractory despite previous treatment¹

Background and Study Design

• The objectives of this observational study were:
  • Primary: Determine the effect of Acthar Gel treatment on the clinical outcomes of patients with DM/PM who were refractory to some combination of corticosteroids, immunosuppressants, rituximab, and intravenous immunoglobulin (IVIG)
  • Secondary: Determine if distinct subgroups of patients, as defined by myopathology or autoantibody status, have different responses to Acthar Gel treatment
• Patient variables assessed for association with responsiveness to Acthar Gel treatment included:
  • DM/PM diagnosis
  • Sex
  • Age
  • Presence of autoantibodies at baseline
  • Extramuscular symptoms (rash) at baseline
  • Administration of concomitant medications
• Outcomes were determined by an independent review of the prescribing physician’s notes, clinical parameters, and laboratory data for each patient
  • Patient laboratory results included myositis-specific antibodies, glycated hemoglobin (HbA1c), and creatine phosphokinase (CPK)

Acthar Regimen

• Acthar Gel was administered via subcutaneous injection*
  • 80 U twice weekly (n=22)
  • 40 U twice weekly (n=1)
  • 80 U once weekly (n=1)
• Treatment ranged from 2 to 18 months, with a median of 6 months
• 22 of 24 patients (92%) were treated concomitantly with other medications

*Initial dosing was 80 U twice weekly via subcutaneous injection. Physicians adjusted doses at their discretion based on disease severity, concomitant health issues, and response to therapy.

Study Limitations

• These results are based on an interim observational study and may not be fully representative of outcomes in the overall patient population. 22 patients were on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel
• While this is the largest observational trial to date, the sample size (N=24) was small. Treatment and assessment of a larger population for a longer time period may provide further insight into predictors of responsiveness
• The population in this study is primarily female and therefore it cannot be determined if gender may have a significant influence on the efficacy of Acthar Gel in DM/PM patients
• This study is uncontrolled and, as such, all patients were not receiving a uniform standard of care. Patients received different classes of medications and combinations of these medications at the time of intervention with Acthar Gel, without a washout period or standardization of their concomitant treatments. No patient cohort was established to receive a standard of care for DM/PM without concomitant Acthar Gel for comparison

58% of patients (n=14) had clinically significant improvement with Acthar Gel^‖,¶ 

Primary Objective

• 57% (n=4) DM patients showed response
• 59% (n-10) PM patients showed response
• There was no association between responsiveness and DM vs PM

^‖Outcomes were determined by an independent review of the prescribing physician’s notes, clinical parameters, and laboratory data for each patient.

^¶Improvement in the inflammatory neuropathy cause and treatment score by at least 1 point, MMT scores of more than 20%, or improvement in myositis activities profile scores by 2 or more points were considered as patient response to Acthar Gel treatment.

Relationship Between Treatment and Response

• Patients who responded had a higher mean duration of treatment (9.7 months) vs nonresponders, whose mean treatment duration was 3.5 months (P<.0001)
• Acthar Gel treatments of longer duration also correlated with a larger percent change reduction in CPK levels from baseline (P=.0123)