Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)

Inclusion Criteria:

- Must give written informed consent to participate in the study

- Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes
mellitus and hypertriglyceridemia.

- Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with
HbA1c ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study
protocol

- Hypertriglyceridemia as defined by fasting TG levels ≥ 500 mg/dL at both Screening and
Qualification visits. Patients with the clinical diagnosis of FPL and with fasting TG
levels ≥ 200 (≥ 2.26 mmol/L) to < 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or
family history criteria for study inclusion may be further screened and enrolled in
the study.

- Presence of hepatosteatosis (fatty liver), as evidenced by a Screening MRI indicating
a hepatic fat fraction (HFF) ≥ 6.4%

Exclusion Criteria:

- Diagnosis of generalized lipodystrophy

- Diagnosis of acquired partial lipodystrophy (APL)

- Acute pancreatitis within 4 weeks of Screening

- Acute Coronary Syndrome within 6 months of screening

- Major surgery within 3 months of screening

- Have any other conditions in the opinion of the investigator which could interfere
with the patient participating in or completing the study