A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Adult Patients With Transfusion-Dependent Beta Thalassemia

Inclusion Criteria:

- Patients ≥ 18 years of age with transfusion-dependent beta thalassemia.

- Patients must have increased iron blood levels as measured by transferrin saturation
(TSAT)

- Patients must have increased iron levels in the heart as measured by magnetic
resonance imaging (MRI)

- Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a
stable dose prior to study and expected to remain stable during study.

- Female patients of childbearing potential must not be pregnant and must use an
effective birth control method during the study.

- Male patients must be either surgically sterile or use an effective birth control
method during the study.

- Patient must be willing and able to provide written informed consent.

Exclusion Criteria:

- Patients must not have a medical condition that would interfere with the conduct of
the clinical study.

- Pregnant or lactating women.

- Patients taking an immunosuppressive agent (except topical over-the-counter steroids)
or have a planned surgery (except dental surgery or simple dermatologic procedures).

- Patients participating in an investigational clinical trial within 30 days of this
study.

- Patients who are unwilling or unable to comply with the study requirements.

- History of hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.

- History of allergic reaction to hepcidin or excipients.

- Unable to undergo MRI.