Woman, aged 53 years, with DM1
This patient did reach the primary endpoint of IMACS DOI.*
Image not of an actual patient.
This patient did reach the primary endpoint of IMACS DOI.*
Image not of an actual patient.
This patient did reach the primary endpoint of IMACS DOI.*
DM patient presented with severe rashes and mild muscle weakness1
| Disease duration |
| 1.3 years |
| Treatment history |
| Prednisone (15 months, 60 mg taper) Tacrolimus (3 months, 3 mg) Azathioprine (3 months, 100 mg) Mycophenolate mofetil (5 months, 3 g) Methotrexate (9 months, 25 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (7.5 mg) Tacrolimus (3 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1.3 years | Prednisone (15 months, 60 mg taper) Tacrolimus (3 months, 3 mg) Azathioprine (3 months, 100 mg) Mycophenolate mofetil (5 months, 3 g) Methotrexate (9 months, 25 mg) |
Prednisone (7.5 mg) Tacrolimus (3 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
Treatments and Outcomes
| CSM | Start | End | Change |
| MD global | 5.5 | 0.5 | -5.0 |
| MMT | 146 | 150 | +4 |
| CPK levels | 66.0 | 84.0 | +18.0 |
| HAQ-DI | 0 | 0 | 0 |
| Patient global | 2.0 | 1.0 | -1.0 |
| Extra-muscular global | 5.5 | 0 | -5.5 |
| CSM | Start | End | Change |
| MD global | 5.5 | 0.5 | -5.0 |
| MMT | 146 | 150 | +4 |
| CPK levels | 66.0 | 84.0 | +18.0 |
| HAQ-DI | 0 | 0 | 0 |
| Patient global | 2.0 | 1.0 | -1.0 |
| Extra-muscular global | 5.5 | 0 | -5.5 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire-Disability Index;
MD global=physician global disease activity; MMT=Manual Muscle Testing.
Safety Findings from Full Study
| Adverse events | Patients with event (n) | Severity |
| Serious adverse events | ||
| Herpes zoster | 1 | Moderate |
| Disseminated herpes zoster | 1 | Severe |
| Avascular necrosis | 1 | Severe |
| Chest pain | 1 | Mild |
| Heart block | 1 | Severe |
| Nonserious adverse events | ||
| Injection site bruising and rash | 4 | Mild |
| Diarrhea | 1 | Mild |
| Anxiety | 1 | Mild |
| Insomnia | 2 | Mild |
| Calcinosis | 2 | Moderate |
| Depression | 1 | Mild |
| Agitation | 1 | Mild |
| Herpes pneumonitis | 1 | Moderate |
| Sinus tachycardia | 1 | Moderate |
| High cholesterol | 1 | Mild |
| Hyperglycemia | 3 | Mild |
| Infection (sinusitis and upper respiratory tract infection) | 2 | Mild |
| Hypertension | 2 | Mild |
| Adverse events | Patients with event (n) | Severity |
| Serious adverse events | ||
| Herpes zoster | 1 | Moderate |
| Disseminated herpes zoster | 1 | Severe |
| Avascular necrosis | 1 | Severe |
| Chest pain | 1 | Mild |
| Heart block | 1 | Severe |
| Nonserious adverse events | ||
| Injection site bruising and rash | 4 | Mild |
| Diarrhea | 1 | Mild |
| Anxiety | 1 | Mild |
| Insomnia | 2 | Mild |
| Calcinosis | 2 | Moderate |
| Depression | 1 | Mild |
| Agitation | 1 | Mild |
| Herpes pneumonitis | 1 | Moderate |
| Sinus tachycardia | 1 | Moderate |
| High cholesterol | 1 | Mild |
| Hyperglycemia | 3 | Mild |
| Infection (sinusitis and upper respiratory tract infection) | 2 | Mild |
| Hypertension | 2 | Mild |
- All adverse events except heart block were study-drug related
- Infections should be considered as a potential risk of Acthar Gel
- No patient developed significant weight gain, cushingoid features, diabetes, persistent hypertension or hyperglycemia, or an increase in A1C
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Additional patients from this study
Woman, aged 37 years, with DM1
DM patient presented with moderate to severe rashes and mild muscle weakness
| Disease duration |
| 27 years |
| Treatment history |
| Prednisone (30 months, 60 mg taper) Methotrexate (30 months, 15 mg) Mycophenolate mofetil (15 months, 2 g) |
| Concomitant treatments (baseline dose) |
| Prednisone (7.5 mg) Methotrexate (15 g) Mycophenolate mofetil (3 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 27 years | Prednisone (30 months, 60 mg taper) Methotrexate (30 months, 15 mg) Mycophenolate mofetil (15 months, 2 g) |
Prednisone (7.5 mg) Methotrexate (15 mg) Mycophenolate mofetil (3 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 4.5 | 0.5 | -4 |
| MMT | 138 | 150 | +12 |
| CPK levels | 66 | 28 | -38 |
| HAQ-DI | 1.375 | 1 | -0.375 |
| Patient global | 9.5 | 7 | -1.5 |
| Extra-muscular global | 3 | 1 | -2 |
| CSM | Start | End | Change |
| MD global | 4.5 | 0.5 | -4 |
| MMT | 138 | 150 | +12 |
| CPK levels | 66 | 28 | -38 |
| HAQ-DI | 1.375 | 1 | -0.375 |
| Patient global | 9.5 | 7 | -1.5 |
| Extra-muscular global | 3 | 1 | -2 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire-Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
Extra-muscular global score was predominantly due to cutaneous disease activity.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Man, aged 27 years, with PM1
PM patient presented with severe muscle weakness
| Disease duration |
| 1.4 years |
| Treatment history |
| Prednisone (17 months, 60 mg taper) Methotrexate (16 months, 20 mg) Azathioprine (14 months, 100 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (42.5 mg) Methotrexate (15 mg) Azathioprine (100 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1.4 years | Prednisone (17 months, 60 mg taper) Methotrexate (16 months, 20 mg) Azathioprine (14 months, 100 mg) |
Prednisone (42.5 mg) Methotrexate (15 mg) Azathioprine (100 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 8 | 2 | -6 |
| MMT | 93 | 132 | +39 |
| CPK levels | 6124 | 934 | -5190 |
| HAQ-DI | 1.375 | 1 | -0.375 |
| Patient global | 7.5 | 3 | -3.5 |
| Extra-muscular global | 1 | 0 | -1 |
| CSM | Start | End | Change |
| MD global | 8 | 2 | -6 |
| MMT | 93 | 132 | +39 |
| CPK levels | 6124 | 934 | -5190 |
| HAQ-DI | 1.375 | 1 | -0.375 |
| Patient global | 7.5 | 3 | -3.5 |
| Extra-muscular global | 1 | 0 | -1 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Woman, aged 58 years, with PM1
PM patient presented with severe muscle weakness
| Disease duration |
| 1.4 years |
| Treatment history |
| Prednisone (14 months, 80 mg taper) Methotrexate (14 months, 25 mg) Mycophenolate mofetil (2 months, 2 g) Azathioprine (6 months, 100 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (10 mg) Mycophenolate mofetil (2 g) Azathioprine (100 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1.4 years | Prednisone (14 months, 80 mg taper) Methotrexate (14 months, 25 mg) Mycophenolate mofetil (2 months, 2 g) Azathioprine (6 months, 100 mg) |
Prednisone (10 mg) Mycophenolate mofetil (2 g) Azathioprine (100 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 6.5 | 1.5 | -5 |
| MMT | 116 | 139 | +23 |
| CPK levels | 289 | 343 | +54 |
| HAQ-DI | 1.375 | 0.875 | -0.5 |
| Patient global | 5 | 2.5 | -2.5 |
| Extra-muscular global | 2 | 0.5 | -1.5 |
| CSM | Start | End | Change |
| MD global | 6.5 | 1.5 | -5 |
| MMT | 116 | 139 | +23 |
| CPK levels | 289 | 343 | +54 |
| HAQ-DI | 1.375 | 0.875 | -0.5 |
| Patient global | 5 | 2.5 | -2.5 |
| Extra-muscular global | 2 | 0.5 | -1.5 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Woman, aged 75 years, with DM1
DM patient presented with moderate-to-severe rashes and severe muscle weakness
| Disease duration |
| 1 year |
| Treatment history |
| Prednisone (11 months, 60 mg taper) Methotrexate (5 months, 20 mg) Mycophenolate mofetil (3 months, 3 g) Azathioprine (3 months, 100 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (50 mg) Mycophenolate mofetil (3 g) Azathioprine (100 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1 year | Prednisone (11 months, 60 mg taper) Methotrexate (5 months, 20 mg) Mycophenolate mofetil (3 months, 3 g) Azathioprine (3 months, 100 mg) |
Prednisone (50 mg) Mycophenolate mofetil (3 g) Azathioprine (100 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 5 | 0.7 | -4.3 |
| MMT | 118 | 148 | +30 |
| CPK levels | 43 | 27 | -16 |
| HAQ-DI | 1.25 | 0.125 | -1.125 |
| Patient global | 5 | 0 | -5 |
| Extra-muscular global | 4 | 0 | -4 |
| CSM | Start | End | Change |
| MD global | 5 | 0.7 | -4.3 |
| MMT | 118 | 148 | +30 |
| CPK levels | 43 | 27 | -16 |
| HAQ-DI | 1.25 | 0.125 | -1.125 |
| Patient global | 5 | 0 | -5 |
| Extra-muscular global | 4 | 0 | -4 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
Extra-muscular global score was predominantly due to cutaneous disease activity.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Woman, aged 54 years, with DM1
DM patient presented with moderate-to-severe rashes and severe muscle weakness
| Disease duration |
| 1 year |
| Treatment history |
| Prednisone (18 months, 60 mg taper) Intravenous immunoglobulin (6 months) Mycophenolate mofetil (7 months, 3 g) Methotrexate (18 months, 22.5 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (10 mg) Methotrexate (22.5 mg) Mycophenolate mofetil (3 g) Hydroxychloroquine (400) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1 year | Prednisone (18 months, 60 mg taper) Intravenous immunoglobulin (6 months) Mycophenolate mofetil (7 months, 3 g) Methotrexate (18 months, 22.5 mg) |
Prednisone (10 mg) Methotrexate (22.5 mg) Mycophenolate mofetil (3 g) Hydroxychloroquine (400) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 4.5 | 0.2 | -4.3 |
| MMT | 119 | 142 | +23 |
| CPK levels | 955 | 326 | -629 |
| HAQ-DI | 1.125 | 1 | -0.125 |
| Patient global | 5 | 2 | -3 |
| Extra-muscular global | 2.2 | 0.3 | -0.9 |
| CSM | Start | End | Change |
| MD global | 4.5 | 0.2 | -4.3 |
| MMT | 119 | 142 | +23 |
| CPK levels | 955 | 326 | -629 |
| HAQ-DI | 1.125 | 1 | -0.125 |
| Patient global | 5 | 2 | -3 |
| Extra-muscular global | 2.2 | 0.3 | -0.9 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
Extra-muscular global score was predominantly due to cutaneous disease activity.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Woman, aged 51 years, with PM1
PM patient presented with severe muscle weakness and dysphagia
| Disease duration |
| 1 year |
| Treatment history |
| Prednisone (24 months, 40 mg taper) Methotrexate (8 months, 17.5 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (2.5 mg) Methotrexate (17.5 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1 year | Prednisone (24 months, 40 mg taper) Methotrexate (8 months, 17.5 mg) |
Prednisone (2.5 mg) Methotrexate (17.5 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 0.8 | 0.1 | -0.7 |
| MMT | 123 | 143 | +20 |
| CPK levels | 2460 | 645 | -1815 |
| HAQ-DI | 0 | 0 | 0 |
| Patient global | 2 | 0 | -2 |
| Extra-muscular global | 0.4 | 0 | -0.4 |
| CSM | Start | End | Change |
| MD global | 0.8 | 0.1 | -0.7 |
| MMT | 123 | 143 | +20 |
| CPK levels | 2460 | 645 | -1815 |
| HAQ-DI | 0 | 0 | 0 |
| Patient global | 2 | 0 | -2 |
| Extra-muscular global | 0.4 | 0 | -0.4 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Woman, aged 45 years, with PM1
PM patient presented with severe muscle weakness and dysphagia
| Disease duration |
| 1.7 years |
| Treatment history |
| Prednisone (24 months, 60 mg taper) Intravenous immunoglobulin (8 months, 2 g/kg) Rituximab (12 months ago, 2 g) Methotrexate (18 months, unknown) |
| Concomitant treatments (baseline dose) |
| Prednisone (20 mg) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 1.7 years | Prednisone (24 months, 60 mg taper) Intravenous immunoglobulin (8 months, 2 g/kg) Rituximab (12 months ago, 2 g) Methotrexate (18 months, unknown) |
Prednisone (20 mg) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 6.5 | 5 | -1.5 |
| MMT | 106 | 113 | +7 |
| CPK levels | 15580 | 14482 | -1098 |
| HAQ-DI | 1.375 | 1.5 | +0.125 |
| Patient global | 5 | 5.5 | +0.5 |
| Extra-muscular global | 0.5 | 0.1 | -0.4 |
| CSM | Start | End | Change |
| MD global | 6.5 | 5 | -1.5 |
| MMT | 106 | 113 | +7 |
| CPK levels | 15580 | 14482 | -1098 |
| HAQ-DI | 1.375 | 1.5 | +0.125 |
| Patient global | 5 | 5.5 | +0.5 |
| Extra-muscular global | 0.5 | 0.1 | -0.4 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
Patient, aged 64 years, with DM1
DM patient presented with severe muscle weakness and mild arthritis
| Disease duration |
| 0.35 years |
| Treatment history |
| Prednisone (6 months, 60 mg taper) Methotrexate (5 months, 25 mg) |
| Concomitant treatments (baseline dose) |
| Prednisone (15 mg) Methotrexate (dosage not available) |
| Disease duration | Treatment history | Concomitant treatments (baseline dose) |
| 0.35 years | Prednisone (6 months, 60 mg taper) Methotrexate (5 months, 25 mg) |
Prednisone (15 mg) Methotrexate (dosage not available) |
These results are based on a single patient and may not be fully representative of outcomes in the overall patient population. The patient was on multiple therapies. The clinical outcomes may not be solely attributable to Acthar Gel.
| CSM | Start | End | Change |
| MD global | 7.2 | 2.1 | -5.1 |
| MMT | 96 | 107 | +11 |
| CPK levels | 483 | 5886 | +5403 |
| HAQ-DI | 1.5 | 1.5 | 0 |
| Patient global | 7.5 | 7 | -0.5 |
| Extra-muscular global | 0.8 | 1.5 | +0.7 |
| CSM | Start | End | Change |
| MD global | 7.2 | 2.1 | -5.1 |
| MMT | 96 | 107 | +11 |
| CPK levels | 483 | 5886 | +5403 |
| HAQ-DI | 1.5 | 1.5 | 0 |
| Patient global | 7.5 | 7 | -0.5 |
| Extra-muscular global | 0.8 | 1.5 | +0.7 |
CPK=creatine phosphokinase; CSM=core set measure; HAQ-DI=Health Assessment Questionnaire Disability Index; MD global=Physician Global Assessment of Change; MMT=Manual muscle testing.
Decrease in MD global, CPK, HAQ-DI, patient global, and extra-muscular global is an improvement.
Increase in MMT is an improvement.
*IMACS DOI: 3 of any of the 6 CSMs improved by 20%, with no more than 2 CSMs worsening by ≥25% (worsening measure cannot include the MMT). Patients who met DOI at any visit must have continued to meet DOI on subsequent visits until study completion.
Adapted from Aggarwal R, Marder G, Koontz DC, Nandkumar P, Qi Z, Oddis CV. Ann Rheum Dis. 2018
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