Study of Effectiveness and Safety of SD-101 in Subjects With Epidermolysis Bullosa

Epidermolysis Bullosa (EB) is a rare group of inherited disorders that manifest as blistering
or erosion of the skin and in some cases, the epithelial liming of other organs, in response
to little or no apparent trauma. There is a lack of effective agents for skin disorders
involving blistering and lesion formation. Current approved therapies are minimally effective
and have safety issues. Scioderm has developed SD-101 Dermal Cream [0% (vehicle), 3% and 6%]
for the treatment of lesions associated with EB.

The purpose of this study is to assess the safety and efficacy of SD-101 cream (3% or 6%) vs.
vehicle in the treatment of lesions in 48 subjects with Simplex, Recessive Dystrophic, or
Junctional Epidermolysis Bullosa.

SD-101 cream [containing 3% or 6% or vehicle (0%)] will be applied topically, once a day to
the entire body for a period of 90 days. Subjects will come into the study site to have a
target wound assessed at baseline. Selected target wound must be at least a certain age and
within a prespecified size range at study entry. Photographic confirmation of the target
wound location will be collected at baseline, and the picture saved from the first visit will
be used to confirm location of the target wound at subsequent visits. The subject will return
to the study site for visits 2,3,4 and 5 (at 14, 30, 60 and 90 days respectively) to have the
target wound previously identified at baseline, re-assessed for the level of healing. In
addition, changes in itching, pain, Body Surface Area (BSA) coverage of blisters and lesions,
and scaring of the healed target wound will also be assessed at each visit.

Inclusion Criteria:

- Informed Consent form signed by the subject or the subject's legal representative; if
the subject is under the age of 18 but capable of providing assent, signed assent from
the subject.

- Subject (or caretaker) must be willing to comply with all protocol requirements.

- Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

- Subjects must be 6 months of age and older.

- Subjects must have 1 target wound within a prespecified size range at study entry

- Target wound must be of at least a certain age

Exclusion Criteria:

- Subjects who do not meet the entry criteria outlined in inclusion criteria.

- Selected target wound cannot have clinical evidence of local infection.

- Use of any investigational drug within the 30 days before enrollment.

- Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.

- Use of systemic or topical steroidal therapy within the 30 days before enrollment.
(Inhaled steroids and ophthalmic drops containing steroids are allowed).

- Use of systemic antibiotics within the 7 days before enrollment.

- Current or former malignancy.

- Arterial or venous disorder resulting in ulcerated lesions.

- Diabetes mellitus.

- Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed
ar screening for female subjects of childbearing potential)

- Females of childbearing potential who are not abstinent and not practicing a medically
acceptable method of contraception.

- Known history of cardiac, hepatic, or renal disease.