Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Subjects With Systemic Sclerosis With Interstitial Lung Disease

Based upon interim analysis data from the Phase 2 proof of concept study in subjects with
systemic sclerosis with interstitial lung disease (CC-4047-SSC-001), the study did not meet
its primary endpoint of improvement in Forced Vital Capacity (FVC) nor for improvement in the
modified Rodnan skin score at 24 and 52 weeks, for subjects who had completed blinded
treatment. In this study, pomalidomide was well tolerated with an adverse event profile
comparable to the risk safety information known for pomalidomide in other diseases. Treatment
has stopped, but patients will remain in follow up.

Inclusion Criteria:

- Male or females between 18 and 80 years of age (inclusive) at the time of consent

- Diagnosis of SSC as defined by ACR criteria

- Onset of the first non-Raynaud's manifestation of SSC within 7 years of Screening

- Subjects are required to meet at least one of the following 2 pulmonary-related
criteria to be eligible for the study:

- Repeat FVC at Baseline (Visit 2) within 5% of the FVC measured at Screening

- Carbon monoxide diffusing capacity (DLco) ≥ 35% and ≤ 80% of predicted value at
Screening

- Abnormalities on high resolution computed tomography consistent with sclerodermatous
involvement of the lung (eg, ground glass, honeycombing)

FVC ≥ 45% and <70% at Screening and Baseline (Visit 2) [with or without a documented
pre-specified FVC decline or fibrosis score]

OR

FVC readings ≥ 70% and ≤ 80% at Screening and Baseline (Visit 2) with a documented history
of either or both of:

1. A ≥ 5% decrease (expressed as percent predicted or in liters) in FVC in the 24-month
period prior to Baseline (Visit 2) based on 3 or more assessments. Two assessments may
be done during the Screening phase provided the assessments are completed at least 2
weeks apart.

2. An HRCT fibrosis score > 20%

Exclusion Criteria:

- Oxygen saturation (SpO2) < 92% (room air [sea level] at rest) at Screening or Baseline

- Known diagnosis of obstructive lung disease as defined by forced expiratory volume
(FEV1)/FVC ratio < 0.7

- Diagnosis of pulmonary arterial hypertension (PAH) requiring treatment

- Known diagnosis of other significant respiratory disorders (e.g., asthma,
tuberculosis, sarcoidosis, aspergillosis, chronic bronchitis, neoplastic disease,
cystic fibrosis, etc.)

- Current clinical diagnosis of another inflammatory connective tissue disease (e.g.,
systemic lupus erythematosus, rheumatoid arthritis, Sjogren's syndrome, etc.)

- Use of melphalan within 52 weeks of Screening

- Additional concomitant medications which prolong the QT/QTc interval (measure of
heart's electrical cycle) during the course of the study

- Use of any anti-coagulant or anti-thrombotic medications (other than low dose-aspirin
[(≤ 100 mg/day)

- Use of any cytotoxic/immunosuppressive agent (other than prednisone ≤ 10 mg/day [mean
dose] or equivalent), including but not limited to azathioprine, cyclophosphamide,
methotrexate, mycophenolate and cyclosporine within 28 days (4 weeks) of Screening

- Use of any biologic agent within 84 days (12 weeks) or 5 half-lives of Screening. In
the case of rituximab, use within 168 days (24 weeks) of Screening or no recovery of
CD20-positive B lymphocytes if the last dose of rituximab has been more than 24 weeks
prior to Screening

- Use of bosentan, ambrisentan, sildenafil, tadalafil and macitentan for PAH within 28
days (4 weeks) of Screening

- Use of medications (e.g., D-penicillamine, Potaba) with putative scleroderma
disease-modifying properties within 4 weeks of Screening

- Use of any investigational drug within 4 weeks of Screening or 5
pharmacodynamic/pharmacokinetic half-lives if known (whichever is longer)

- Smoking of cigars, pipes or cigarettes within 24 weeks of Screening