Setemelanotide in a Single Patient With Partial Lipodystrophy

This is a single-patient study to assess the safety and efficacy of setmelanotide in a unique
patient with partal LD and severe metabolic abnormalities, most notably refractory
hypertriglyceridemia leading to recurrent pancreatitis despite ongoing plasmapheresis
treatment.

Inclusion Criteria:

1. Signed written informed consent

2. Ability to comply with visits and procedures required by program.

3. Has physician-confirmed partial lipodystrophy and the following characteristics:

- Has atypical lipodystrophy.

- Has presence of neutralizing antibody to metreleptin

- Patient has life threatening hypertriglyceridemia and has had >8 episodes of
pancreatitis, requiring weekly plasmapaheresis

- Has Type 1 diabetes melltus with HbA1c > 10%. Age and Reproductive Status

4. Female, under the the age of 18 years • The patient has primary amenorrhea at this
time

Exclusion Criteria:

- 1. Diagnosis of schizophrenia, bipolar disorder, personality disorder or other
Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the
investigator believes will interfere significantly with study compliance.
Neurocognitive disorders affecting ability to consent will not be disqualifying as
long as an appropriate guardian able to give consent has been appointed.

2. History or close family history (parents or siblings) of skin cancer or melanoma,
or patient history of ocular-cutaneous albinism.

3. Significant dermatologic findings relating to melanoma or pre-melanoma skin
lesions, determined as part of a screening comprehensive skin evaluation performed by
a qualified dermatologist. Any concerning lesions identified during the screening
period will be biopsied and results known to be benign prior to enrollment. If the
pre-treatment biopsy results are of concern, the patient may need to be excluded from
the study.

4. Significant hypersensitivity to study drug. 5. Inability to comply with QD
injection regimen.