Treatment of Proteinuria Due to Treatment Resistant or Treatment Intolerant Idiopathic FSGS

Inclusion Criteria

Subjects must meet all of the following criteria for inclusion in the study at the
Screening Visit/Visit 1 and the Baseline Visit/Visit 2:

1. Must be adequately informed and understand the nature and risks of the study and must
be able to provide a signature and date on the ICF.

2. Must be at least 18 years of age at Screening Visit/Visit 1 and can be male or female.

3. Female subjects must be of nonchildbearing potential (history of hysterectomy,
bilateral oophorectomy, or are postmenopausal with no history of menstrual flow in the
12 months prior to Screening Visit/Visit 1), or if of childbearing potential must be
nonpregnant, nonlactating and agree to use effective contraception with a male partner
throughout study participation and for 4 weeks after ending study participation.
Acceptable forms of contraception include hormonal measures (oral contraceptive pills,
contraceptive patch, contraceptive ring, injections), intrauterine devices, double
barrier method (condom plus diaphragm, condom or diaphragm plus spermicidal gel or
foam), and abstinence.

4. Must have a history of primary FSGS confirmed by a renal biopsy.

5. Must be willing to undergo a renal biopsy unless the most recent renal biopsy is
within 3 years of the Screening Visit/Visit 1.

6. Must have a total urine protein creatinine ratio (uPCR) greater than 3.0 mg/mg at
Screening Visit/Visit 1.

7. Must have an estimated glomerular filtration rate (eGFR) at least 30 mL/min/1.73 m2 at
the Screening Visit/Visit 1 as calculated by the 4-variable modification of diet in
renal disease (MDRD) study equation.

8. Must have failed to achieve sustained CR or PR of proteinuria as determined by the
investigator with at least 1 prior immunosuppressive therapy, or must have intolerance
to at least 1 prior immunosuppressive therapy.

9. Must have been treated with an angiotensin converting enzyme inhibitor (ACEi) or
angiotensin receptor blocker (ARB) for at least 4 weeks prior to Screening Visit/Visit
1 and on stable maintenance dose(s) for at least 14 days prior to Screening
Visit/Visit 1 and are expected to remain on that dose for the duration of the study or
have a documented intolerance to ACEi or ARB with approval from the MM prior to
enrollment. Subjects taking other antihypertensive medication(s), must be on a stable
dose of for at least 4 weeks prior to the Screening Visit/Visit 1.

10. Must have a mean systolic blood pressure no more than 150 mm Hg and a diastolic blood
pressure of no more than 90 mm Hg determined by the average of at least 3 seated
readings taken at least 5 minutes apart at Screening Visit/Visit 1 and at the Baseline
Visit/Visit 2.

11. Must be able to communicate effectively with study personnel.

12. Must be able and willing to follow all protocol requirements and study restrictions.

13. Must be able and willing to return for all study visits.

Exclusion Criteria

Subjects are ineligible for study participation if they meet any of the following criteria
at the Screening Visit/Visit 1 or Baseline Visit/Visit 2:

1. Is from a vulnerable population, as defined by the US CFR Title 45, Part 46, Section
46.111(b) and other local and national regulations, including but not limited to,
employees (temporary, part-time, full time, etc) or a family member of the research
staff conducting the study, or of the sponsor, or of the clinical research
organization, or of the IRB/IEC.

2. Is unwilling to receive, or is intolerant of, subcutaneous injections.

3. Has any history of use of ACTH preparations for treatment of nephrotic syndrome
(including but not limited to Acthar and Synacthen®).

4. Has a history of sensitivity to ACTH preparations (including but not limited to Acthar
and Synacthen).

5. Has a history of sensitivity to porcine protein products.

6. Has a body mass index (BMI) greater than 45 kg/m2 at Screening Visit/Visit 1.

7. Has secondary FSGS, including but not limited to, obesity, known genetic cause,
reduced kidney mass, Human Immunodeficiency Virus (HIV), intravenous heroin abuse, or
sickle cell anemia.

8. Cannot have failed to achieve sustained CR or PR of proteinuria, as determined by the
investigator, on more than 2 prior immunosuppressive therapies with different
mechanisms of action.

9. Has had a renal biopsy demonstrating greater than 50% global glomerular sclerosis, or
greater than 50% cortical interstitial fibrosis, or collapsing FSGS.

10. Has any known contraindication(s) to Acthar including, but not limited to:

11. Any known history of scleroderma, osteoporosis, or ocular herpes simplex.

12. Any current uncontrolled hypertension, primary adrenocortical insufficiency, or
adrenal cortical hyperfunction.

13. Any current congestive heart failure (defined as New York Heart Association Functional
Class III to IV).

14. Peptic ulcer (within 6 months prior to Screening Visit/Visit 1).

15. Recent major surgery (within 6 months prior to Screening Visit/Visit 1, renal biopsy
is not considered an exclusionary surgical procedure).

16. Has a history of chronic active hepatitis; acute or chronic hepatitis B; or acute or
chronic hepatitis C.

17. Has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close
contact with an individual with an active TB infection.

18. Has a clinically significant infection requiring intravenous administration of
antibiotics and hospitalization in the 4 weeks prior to Screening Visit/Visit 1.

19. Has known immune compromised status, including but not limited to, individuals who
have undergone organ transplantation or who are known to be positive HIV.

20. Has Type 1 or Type 2 diabetes mellitus (prior diagnosis of gestational diabetes
mellitus is not exclusionary).

21. Has had any of the following in the 12 weeks prior to Screening Visit 1/Visit 1:
unstable angina confirmed by a cardiologist, myocardial infarction, coronary artery
bypass graft, percutaneous trans-luminal coronary angioplasty, transient ischemic
attack or cerebrovascular disease, unstable cardiac arrhythmia, or resuscitated sudden
cardiac death.

22. Has any solid tumor malignancy currently diagnosed or undergoing therapy, or has
received therapy for any solid tumor malignancy in the 5 years prior to Screening
Visit/Visit 1, with the exception of treated and cured basal cell carcinoma, treated
and cured squamous cell carcinoma of the skin, and treated and cured carcinoma in situ
of the cervix.

23. Has a diagnosis of, is undergoing therapy for, or has received therapy for a
hematologic malignancy in the 5 years prior to Screening Visit/Visit 1.

24. Has been treated with certain medications within specified timeframes prior to the
Screening Visit/Visit 1 (Rituximab, Investigational treatment for FSGS,
Intravenous/intramuscular corticosteroids,Therapeutic drug/device trial (except FSGS),
Cyclophosphamide, Chronic oral, corticosteroids

25. Has been treated with certain medications within specified timeframes prior to
Baseline Visit/Visit 2 (Corticosteroids , Mycophenolate, Mofetil).

26. Has current or recent (within 6 months of the Screening Visit/Visit 1) drug or alcohol
abuse as defined in Diagnostic and Statistical Manual of Mental Disorders, Fifth
Edition, Diagnostic Criteria for Drug and Alcohol Abuse.

27. Has any of the following laboratory abnormalities at the Screening Visit/Visit 1:

- Hemoglobin less than 8.0 g/dL.

- Platelets less than 50,000 cells/μL.

- Absolute neutrophil count (ANC) greater than 1000 cells/μL.

- Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total
bilirubin greater than 2 times the upper limit of normal (ULN).

- Glycosylated hemoglobin (HbA1c) greater than 6.5%.

- Positive Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody
(HBcAb).

- Positive Hepatitis C virus antibody (HCV) and HCV polymerase chain reaction (PCR)
at least 25 IU/mL (HCV PCR will be automatically analyzed if HCV is positive).

- Positive or indeterminate interferon gamma release assay (IGRA).

28. Has any other clinically significant disease, disorder or laboratory abnormality
which, in the opinion of the investigator (by its nature or by being inadequately
controlled), might put the patient at risk due to participation in the study, or may
influence the results of the study or the subject's ability to complete the study.